Edited Transcript of FLXN earnings conference call or presentation 7-May-20 8:30pm GMT – Yahoo Finance

BURLINGTON May 8, 2020 (Thomson StreetEvents) -- Edited Transcript of Flexion Therapeutics Inc earnings conference call or presentation Thursday, May 7, 2020 at 8:30:00pm GMT

* David A. Arkowitz

Flexion Therapeutics, Inc. - CFO

Flexion Therapeutics, Inc. - Chief Commercial Officer

* Michael D. Clayman

Flexion Therapeutics, Inc. - Co-Founder, President, CEO & Director

Flexion Therapeutics, Inc. - VP of Corporate Communications & IR

* Randall S. Stanicky

RBC Capital Markets, Research Division - MD of Global Equity Research & Lead Analyst

Scott Young, Flexion Therapeutics, Inc. - VP of Corporate Communications & IR [1]

Good afternoon. This is Scott Young, Vice President of Corporate Communications and Investor Relations.

Before we begin, I'd call your attention to the metrics slides that we will discuss in today's presentation. Those slides can be viewed directly via the webcast or under the Investors tab on flexiontherapeutics.com. In addition, the press release we issued this afternoon and an archive of this conference call can also be found there.

Today's conference call will be led by Flexion's Chief Executive Officer, Dr. Michael Clayman; and he is joined by David Arkowitz, Chief Financial Officer; and Melissa Layman, Chief Commercial Officer.

On today's call, we will be making forward-looking statements that include commercial, financial, clinical and regulatory projections. Statements relating to future financial or business performance, conditions or strategies and other business matters, including expectations regarding net sales, operating expenses, cash utilization, clinical, regulatory and commercial developments and anticipated milestones are forward-looking statements within the meaning of the Private Securities Litigation Reform Act. Flexion cautions that these forward-looking statements are subject to various assumptions, risks and uncertainties which change over time. Additional information on the factors and risks that could affect Flexion's business, financial conditions and results of operations are contained in Flexion's Form 10-Q for the quarter ended March 31, 2020, filed with the SEC today and other filings which are available at http://www.sec.gov as well as Flexion's website. These forward-looking statements speak only as of the date of this call and Flexion assumes no duty to update such statements.

I will now turn the call over to Flexion's CEO, Mike Clayman.

Michael D. Clayman, Flexion Therapeutics, Inc. - Co-Founder, President, CEO & Director [2]

Thank you, Scott, and thank you all for joining us today.

It is clear that in the only 8 weeks since our fourth quarter earnings call, the world has changed dramatically as a result of COVID-19. On today's call, we'll talk about what we've been seeing in doing both commercially and operationally during the unprecedented public health crisis, but we'll begin with a focus on our first quarter performance and provide color on why we remain enthusiastically bullish about ZILRETTA's future.

Today, we reported net ZILRETTA sales of $20.1 million for the first quarter of 2020. We had previously indicated that we believed our Q1 sales would be roughly flat versus Q4 sales of $23.7 million, and we were on track to deliver sales in this range prior to the COVID-19 outbreak. We also said that at that point, it was simply impossible to predict how the outbreak would evolve or what the effects of more aggressive social distancing or other containment efforts might have on patients or practices. Since then, the picture has become much clearer, and we can speak now to the effects of the pandemic on ZILRETTA sales and later in the call, on our development activities.

Beginning in late Q1, we saw most practices substantially reduce patient visits or even suspend these altogether. And as a result, utilization of ZILRETTA dropped significantly. We believe it will take time to get to the new normal and the cadence of the recovery will vary practice by practice, region by region and even person by person. Thus far, we have seen decreased demand for ZILRETTA in the second quarter and believe COVID-19 will adversely impact ZILRETTA revenue for the remainder of the year.

That said, we do take note of a number of external circumstances that have the potential to increase demand for ZILRETTA over time. These all relate to the delay, postponement or cancellation of total knee arthroplasties or TKAs, and the intense medical need to provide rapid, substantial, durable non-opioid analgesia for patients whose knee pain drove them to consider TKA in the first place. Three factors contribute to this dynamic and these were all brought to our attention by orthopedic key opinion leaders.

First, while elective surgeries are beginning to happen around the country, in most cases, there will be a gradual return to full capacity, and many TKAs will be further delayed. Second, particularly for those of Medicare age, who are all considered in the vulnerable COVID-19 population, there may be reluctance to enter a hospital to have surgery until these patients have greater confidence they can avoid infectious risk. Finally, for those who are younger than 65 and were previously employed, many may prioritize income generation over surgery and rehab.

Recent survey data indicate that almost 50% of orthopedic surgeons expect that procedure volume will not be back to normal until some time in 2021 and 63% expect anywhere between 25% and 75% of their patients will continue to delay their surgery for fear of COVID-19.

Of course, Flexion is adapting, too. Since instructing our employees to work from home in mid-March, our MBMs have adopted various technologies to engage in meaningful conversations with prescribing physicians. Melissa can provide more color on what we are seeing in the field, but since health care practitioners have more time on their hands, our MBMs have experienced greater access to prescribers who have historically been very difficult to see, and their response to the ZILRETTA clinical data has been highly encouraging. And even with COVID-19, we continue to hear stories of patients who have received unprecedented OA knee pain relief from ZILRETTA.

The bottom line is that our confidence in ZILRETTA's clinical performance and long-term potential is undiminished. We are not the only company to believe that ZILRETTA holds tremendous potential for millions of patients with OA knee pain. In April, in the midst of the pandemic, we announced that we entered into an exclusive license agreement with HK Tainuo and Jiangsu Tainuo, a subsidiary of CSPC, a leading Chinese biopharmaceutical company. Under the terms of the agreement, we are entitled to receive an upfront payment of $10 million and up to $32.5 million in development, regulatory and commercial sales milestone payments. And Flexion will be solely responsible for the manufacturing and supply of ZILRETTA for all clinical and commercial activities.

While our confidence in ZILRETTA is resolute, it is impossible for us to predict how long the pandemic will affect sales. And as we announced in our press release this afternoon, we have taken meaningful actions to reduce our operating expenses in the near term. David will discuss these steps in more detail, but we are committed to ensuring that we come out of the pandemic in a strong financial position.

Regarding clinical development, recognizing that many clinical trial sites had shut down, wanting to ensure the safety of our research participants and in consideration of guidance issued by the FDA, in early April we announced that we would temporarily suspend our Phase II clinical trial evaluating the efficacy of ZILRETTA in patients with shoulder OA or adhesive capsulitis and also our Phase I clinical trial evaluating the safety and tolerability of FX201, our intra-articular gene therapy candidate for OA.

Subsequently, we made the strategic decision to discontinue our Phase II trial investigating ZILRETTA in shoulder OA and in adhesive capsulitis. Given the small number of patients enrolled in that trial, the uncertainty of when we would be able to restart it, as well as the costs required to maintain the study in an inactive status, we believe terminating the trial is the most responsible action at this time. Moving forward, we will look to leverage our learnings from the study and potentially use these to refine the trial design to advance ZILRETTA in these indications.

We also aim to reinitiate the FX201 single ascending dose trial and recommence enrollment when conditions and staffing at our trial sites safely permit the return of patients.

Turning to FX301, our NaV1.7 inhibitor formulated within a thermosensitive hydrogel for administration as a peripheral nerve block for controlled postoperative pain, we continue to advance this product candidate. FX301 is a liquid at room temperature and gels following injection at body temperature to create a controlled release depot that can provide persistent therapeutic concentrations of drug locally at the selected nerve. We recently unveiled new preclinical data in a virtual poster presentation now available on the American Society of Regional Anesthesia and Acute Pain Medicine website. These data show that compared to liposomal bupivacaine and placebo, FX301 provided sustained postoperative analgesic effect with no impairment in motor function. In addition, high local concentrations of funapide, the active ingredient in FX301, were measured at the site of administration for the duration of the study, consistent with the creation of the depot providing controlled drug release.

While it is still early days for the program, these findings support our belief that unlike typical local anesthetics, the selective pharmacology of FX301 has the potential to deliver substantial and persistent pain relief while preserving motor function which could enable earlier rehabilitation following musculoskeletal surgery and potentially more rapid discharge from the hospital. We are completing GLP tox studies for FX301, and we remain on track to initiate clinical trials in 2021.

Before I turn it over to Melissa, I would like to make just a few final comments. While there are many uncertainties about the shape and pace of the recovery, there are some key facts we know right now. We know that there are millions of patients for whom ZILRETTA can provide substantial and extended relief from OA knee pain. We know that at least many tens of thousands of elective knee replacements have been deferred due to COVID-19 and those patients will need effective pain relief while they wait for surgery. We know that our commercial organization is poised for the recovery at whatever pace or shape it takes. Finally, we know that everyone at Flexion stands ready to adapt to any circumstances we face, and we remain confident in our future prospects.

At this point, I would like to turn it over to Melissa to discuss our commercial operations.

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Melissa Layman, Flexion Therapeutics, Inc. - Chief Commercial Officer [3]

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Thank you, Mike.

I've been with Flexion now for just under 2 months, my first day marked by our Q4 earnings call and my second day by the work-from-home directive resulting from the rise of the COVID-19 pandemic. Despite having yet to set foot in the field, I've met virtually with dozens of physicians and members of our sales organization. Each of these meetings has only confirmed what I believed about ZILRETTA before joining the team and bolstered my perceptions of the Flexion organization, particularly in light of our response to COVID-19 and the enhanced role I know ZILRETTA can play in effective, durable pain management for patients all along the OA knee pain treatment continuum.

Mike discussed some of what we have experienced in the field during recent weeks. I'll provide some additional color on how we've been engaging with prescribers during the crisis and how we plan to emerge from the crisis, restore ZILRETTA's prior sales trajectory and continue to grow from there. Before I talk about what we are seeing now, I'd like to start by walking through the commercial metrics for the first quarter.

As Mike mentioned, we were pleased with the way the first quarter was progressing prior to the onset of COVID-19 and believe ZILRETTA was tracking to meet our expectations. You can see in Slide 2 that we recorded ZILRETTA net sales of $20.1 million, reflecting the pandemic's material impact on March sales. We expanded our list of target accounts in the fourth quarter to 5,000 and by March 31, 2020, our reach had extended to almost all of them. And by the end of the first quarter, 3,672 accounts had purchased ZILRETTA, which was an increase of 184 purchasing accounts compared to the fourth quarter of 2019. As of the end of March, 2,832 or 77% of accounts had reordered ZILRETTA. Notably, this increase in purchasers occurred off a customer base that continued to grow even in light of the pandemic.

On Slide 3, you can see how our sales have progressed since launch. And as we've mentioned, we were pleased with the performance in the first part of Q1 prior to the onset of COVID-19, when we were on track to deliver sales in line with fourth quarter of last year, consistent with our previous guidance.

The next 3 slides reflect ZILRETTA purchasing patterns across our growing account base comprised of practices, clinics and hospitals of various sizes and potential.

Slide 4 describes the cumulative number of accounts that have purchased ZILRETTA since launch as well as the distribution of quarterly purchase volumes across our account base. In Q1, the trend continued with a growing number of accounts purchasing within and across the purchase break of 1 to 10 units, 11 to 50 units and 51 or more units with significant growth to 916 accounts in the 51-plus category.

Slide 5 flips the perspective, looking at the cumulative distribution of ZILRETTA purchases by accounts. For example, those 916 accounts in the 51-plus purchase group shown on the previous slide have been responsible for 177,664 of the total units purchased since launch and roughly 35,000 more units than were purchased from this purchase volume group in Q4 2019. This clearly illustrates that purchasing accounts continue to move through the ZILRETTA utilization continuum described on previous calls.

As we've also previously highlighted, even our largest accounts remain underpenetrated, and we continue to believe that there is tremendous opportunity to increase ZILRETTA utilization within and across each of these groups.

It is also important to point out that while total ZILRETTA purchases in the first quarter were approximately 36,500 units, which is slightly lower than the 37,500 units purchased in the fourth quarter, through the middle of March, we were pleased with our progress and believe we were well on track to meet or exceed the fourth quarter purchase volume until the effects of COVID-19 began to impact our business and ZILRETTA purchases dropped precipitously.

In our final slide, #6, we break out ZILRETTA purchasing by new and existing accounts. In Q1, new accounts purchased 12,504 units, which further illustrates our strong start to 2020 prior to the onset of COVID-19 across the U.S.

As Mike mentioned, COVID-19 has had significant impact on our customers. Throughout the COVID-19 impacted window, overall clinician access to ZILRETTA-eligible patients has been limited, variable and practice dependent. Some practices have shut down in-person patient visits altogether, while others are open to high priority critical or acute cases while implementing social distancing measures. While the circumstances vary, one common denominator has been the presence and tenacity of our sales force. While not a replacement for in-person exchanges, our reps have been successful in targeted remote detailing, in some cases gaining access to prescribers that exceeds pre-COVID-19 levels. They have effectively employed various audiovisual technologies to engage with both existing and new accounts to discuss the important role ZILRETTA can play in the OA treatment paradigm, including for those patients who are awaiting or opt to decline surgery once COVID-19 restrictions are lifted.

To better understand the current impact of COVID-19 on orthopedic practice and what the recovery might look like, we held a series of focus groups with specialists from across the nation. The insight from those exchanges support our assumptions that the return to new normalized practice patient flow will be gradual and driven by ongoing social distancing requirements and safety requirements as well as patients' comfort in returning to their doctors' offices. However, we also see the potential for increased use of ZILRETTA in a wider variety of patient types as both existing and new prescribers look to manage the growing backlog of surgical candidates, including those previously scheduled for TKR who, for various COVID-19-related reasons, choose to defer treatment in favor of less invasive options like ZILRETTA.

As Mike aptly described, we cannot predict the timing or shape of the recovery. But as it occurs, our commercial team will be there to support prescribers and ensure they understand the even more important role ZILRETTA can play in effectively managing their patients' OA knee pain prior to or in avoidance of surgery.

With that, I'll now turn it over to David for the financial update.

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David A. Arkowitz, Flexion Therapeutics, Inc. - CFO [4]

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Thank you, Melissa.

Before I provide an overview of our financial performance in the first quarter, I'd like to share some details on our fiscal response to the economic and business disruption caused by COVID-19.

As Mike mentioned and as we described in our first quarter earnings press release, we undertook a series of prudent and disciplined steps to reduce expenses across the organization in order to enhance our financial flexibility and liquidity. In total, we believe these steps will deliver between $43 million and $53 million in savings this year. As a result, our full year 2020 total operating expenses, which include the cost of sales, research and development, and selling, general and administrative expenses, are expected to be in the range of $167 million to $177 million.

We undertook cost savings initiatives across every area of the business, but the majority of the savings will be realized through the following: hiring and travel freezes, suspension and/or termination of active clinical trials, elimination of live presence at medical and industry conferences, reductions in in-person physician speaker programs and reductions in market research and select marketing programs and materials as well as elimination of nonessential operating expenses.

In addition, given the impact of COVID-19 and the uncertainty surrounding ZILRETTA sales for the remainder of this year, we are temporarily suspending manufacturing activities for ZILRETTA to avoid excessive inventory buildup and to reduce costs. We believe our current inventory of finished goods will be sufficient to meet demand for ZILRETTA through at least the remainder of this year. And unlike typical contract manufacturing agreements, our condominium model at Patheon provides us with the ability to scale up production based on market dynamics. And once we determine that additional supply will be needed, we can reinitiate manufacturing.

With respect to our first quarter financial performance, we reported a net loss of $36.8 million for the first quarter of 2020 compared to a net loss of $41.5 million for the same period of 2019.

Net sales of ZILRETTA were $20.1 million and $10.6 million for the 3 months ended March 31, 2020 and 2019, respectively. Cost of sales were $2.3 million and $1.8 million for the 3 months ended March 31, 2020 and 2019, respectively. The first quarter 2020 net sales reflect a gross to net reduction of 11%. The gross to net reduction is primarily comprised of distributor and service fees, returns reserve, health care provider rebates and mandatory government discounts and rebates, such as Medicaid, 340B institutions, Veterans Administration and Department of Defense. The rebates to health care providers are variable based on the volume of product purchased and contribute 2% of the first quarter total gross to net reduction of 11%.

As Melissa mentioned, starting in the middle of March, purchases of ZILRETTA by accounts, meaning physician practices, clinics and hospitals, dropped materially due to COVID-19. As a result, the inventory levels held by our distributors at the end of the first quarter were higher than our target level of 1 to 3 weeks. While purchases of ZILRETTA by accounts continue at a decreased rate, it will take time for our distributors to work through their current inventory. Since we recognize revenue upon sales to our distributors, a combination of reduced purchases in the second quarter by account and higher-than-normal distributor inventory levels have the potential to particularly impact our second quarter revenue negatively.

Research and development expenses were $21.1 million and $15.4 million for the 3 months ended March 31, 2020 and 2019, respectively. The increase in research and development expenses of $5.7 million was primarily due to an increase of $2.2 million in salary and other employee-related costs for additional headcount and stock compensation expense; an increase in expenses related to FX201, including the $2.5 million milestone to GeneQuine for dosing the first human patient in the Phase I clinical trial; and an increase in other portfolio expenses primarily related to a $0.5 million milestone to Xenon Pharmaceuticals, offset by a decrease of $0.5 million in development expenses for ZILRETTA due to lower clinical trial expenses during the period.

Selling, general and administrative expenses were $29.3 million and $32.2 million for the 3 months ended March 31, 2020 and 2019, respectively. Selling expenses were $20.5 million and $23.8 million for the 3 months ended March 31, 2020 and 2019, respectively. The year-over-year decrease in selling expenses of $3.3 million was primarily due to a reduction in physician and patient marketing activities. General and administrative expenses were $8.8 million and $8.4 million for the 3 months ended March 31, 2020 and 2019, respectively, which represents an increase of $0.4 million.

Interest expense was $0.4 million and $1 million for the 3 months ended March 31, 2020 and 2019, respectively. Interest expense was $4.7 million and $3.9 million for the 3 months ended March 31, 2020 and 2019, respectively.

As of March 31, 2020, we had approximately $125.2 million in cash, cash equivalents and marketable securities compared with $136.7 million as of December 31, 2019.

At this point in time, we cannot forecast with any precision the duration of the pandemic or its full impact on purchasing patterns or utilization of ZILRETTA. Therefore, it would be imprudent for us to provide specific guidance with respect to our future sales of ZILRETTA or our cash runway. We will continue to review and reassess our assumptions and provide additional color when possible. As Mike mentioned, our confidence in ZILRETTA is as strong as ever, and we will take the steps necessary to ensure we are well positioned from a financial perspective to maximize ZILRETTA's value in a post-COVID-19 environment.

At this point, I would ask the operator to please open the line for questions.

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Questions and Answers

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Operator [1]

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(Operator Instructions) Our first question or comment comes from the line of Randall Stanicky from RBC Capital Markets.

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Randall S. Stanicky, RBC Capital Markets, Research Division - MD of Global Equity Research & Lead Analyst [2]

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Great. Do you have a sense from any of the regions or the large practices that have reopened as to what their ordering patterns look like? Because presumably, that would give you a sense for at least physician or practice thinking around any pent-up demand and then where they intend to focus on from a procedure perspective kind of channel inventory aside. And then I have a follow-up.

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Michael D. Clayman, Flexion Therapeutics, Inc. - Co-Founder, President, CEO & Director [3]

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Melissa, do you want to take that?

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Melissa Layman, Flexion Therapeutics, Inc. - Chief Commercial Officer [4]

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Yes. I think that's me. We have been sort of following the basis for which practices have been reopening and it is not geographically specific. We believe that the return to normal patient volume is going to vary by region, by practice and even by patient. And the impact to existing office-based patient flow that we've seen don't necessarily correlate to the areas where COVID-19 has been more prevalent.

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Michael D. Clayman, Flexion Therapeutics, Inc. - Co-Founder, President, CEO & Director [5]

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And just building on Melissa. I mean, Randall, I just think it's too early for us to declare that we're seeing patterns. We're seeing some accounts order substantial quantities, other accounts ordering lesser amounts, but it's far from us having an update and then say, we have clarity on ordering pattern.

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Randall S. Stanicky, RBC Capital Markets, Research Division - MD of Global Equity Research & Lead Analyst [6]

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Let me ask a follow-up. I mean, during this shutdown period, which in some regions, regions like New York is probably going to continue, have you guys been able to alter your outreach via DTC? I mean, you have captive patients who are homebound, who are probably in pain, maybe thinking about the timing of elective surgeries. Have you figured out a way to access those patients to get ZILRETTA as an option in front of them?

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Melissa Layman, Flexion Therapeutics, Inc. - Chief Commercial Officer [7]

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So with regard to accessing patients specifically, our efforts haven't been focused there during this period. They have been focused specifically with our health care providers. Our sales force has been working from home since the middle of March, and they have been fully audiovisual equipped to continue to engage physicians remotely. And in fact, many of their conversations with our users and some of our nonusers have focused around that very point, which is that with surgeries having been rescheduled, postponed, canceled altogether, the wait times to get to those surgeries are mounting and that ZILRETTA offers an alternative to managing the intractable pain that these patients are continuing to suffer with and therefore is being more widely considered by our customers as that bridging tool that they can use until those patients are able to be put back on to the surgical schedule.

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Operator [8]

See the article here:
Edited Transcript of FLXN earnings conference call or presentation 7-May-20 8:30pm GMT - Yahoo Finance

The medicine of music: Tim Vallillee turns to song as therapy for cystic fibrosis and for life – TheChronicleHerald.ca

YARMOUTH, N.S.

For Tim Vallillee, music is medicine.

Something he can reach out to when hes hurting and when hes not. Something he can tap into for his physical health and his soul.

Something, most importantly,he can share with others.

Ive always felt that the fact I sing so much has been added therapy for me, says Vallillee, 52, who has cystic fibrosis.

Vallillee grew up in Yarmouth and lives in the Valley with his wife Agatha and son Isaiah. When COVID-19 came into play, he lost his gigs as venues were no longer open to the public.

When theyre taken away your musical brain says, this really sucks. But my CF brain says I cant do my thing, how am I going to tell how Im feeling?

Singing is a barometer that I use for the level of my health, he explains, likening it to an early warning device that lets him know how hes feeling inside especially when hes reaching for those high notes.

Ive got a pretty powerful voice but you cant do that without air in your lungs, he says. If that air is not in there it shows in my voice. It can be very subtle, but I notice it. I know something is brewing.

But where theres a will, theres a way.

Vallillee has been performing and sharing music Thursday evenings via Facebook live on his personal social media page. For his first session he put his iPhone in a tripod, sat on his bed and played for a couple of hours. Its now a highlight of his week.

Its great because I get to do what I like to do and its all part of my life therapy to have music in my life.

And hes happy to have that connection with others again.

In addition to Facebook you can also hear his music on YouTube. He also has a website to connect with others http://www.timothyv.ca.

Vallillee learned to play the guitar when he was 18. But it was something that happened when he was 16 that chartered his musical course.

My dad had a massive heart attack and when he was in the hospital . . . I promised my dad that if he wouldnt die I would learn to play an instrument, he says. He always wanted one of his four kids to learn an instrument. He always joked the best thing we could play was the record player.

His father recovered and a couple of years later reminded Vallillee of his promise. So the son grabbed a guitar and taught himself to play.

He ended up playing in many bands with friends. One of his first gigs was a variety show during Yarmouth high schools winter carnival in the 1980s.

Vallillee turns to music to express himself when hes feeling happy, sad, joyful, fearful sometimes bored and, more recently, heartbroken. His most recent original song is called Home To Me.

Its a song he wrote about what a special place Nova Scotia is. He wrote the song following the April mass shooting tragedy in the province. In the lyrics he picked out places and experiences in Nova Scotiathat he connects with and hopes others will as well.

I tried to encompass as many people as I could from Yarmouth to Cape Breton, he says.

Vallillee didnt want the song to just be about healing right now but a song people can listen to at any point in their life, whether next week, next month, or next year, and it will make them feel good.

Folks from this part of the country, are different from all the rest. We've got a special something deep down in our chest.

Nova Scotia is where I roam...Nova Scotia, that's my home. Nova Scotia is where I'll be ... Nova Scotia, is home to me.

Vallillee'swife, Agatha Bourassa, says not only is music where her husband shines, its a gift he gives to others.

This great song idea came about like every one of Tims songs have over the past two decades. Nobody really knows how he writes but he sparks an idea. Sometimes he pulls off on the side of the road and all of a sudden he regurgitates a song, she says. We just lived through this horrific tragedy in Nova Scotia and maybe no one feels like celebrating but Tim truly wrote that song to try and uplift every Nova Scotian. Lets celebrate life because every breath is worth it.

Indeed. Breaths are something those with cystic fibrosis cant take for granted. Which makes life challenging during the COVID-19 pandemic. People with immune deficiencies and underlying health issues are especially at risk.

Still, Vallillee isnt doing anything drastically different now than he does in normal times. Thats because, in a sense, hes always living in a COVID reality.

I used to say Im one bug away from getting sick and ending up in the hospital and dying in a week Ive always lived that perspective, he says, adding those with CF are very used to avoiding germs.

He calls himself a germ ninja.

Its not like I normally go around in a mask, but I will in a hospital. And Im vigilantabout always trying to wash my hands and avoid germs.

My wife, God love her, if she hears anybody coughing or sneezing in my direction even if were sitting a movie theatre and she hears somebody cough behind or near us its like weve got to move, he says. Shes seen me knock on deaths door quite a number of times. Thankfully we survived that.

Fortunately for Vallillee and his family, his health has been good in recent years. He hasnt been in the hospital in abouteight years and he attributes this to Kalydeco, a new medication he received six years ago. Its been a game-changer.

It basically goes in and changes CF genes inside my body on a genetic level. Because of that my lung function jumped about 20 per cent when I first got on it, he says. It virtually saved my life. I havent been in the hospital since. Ive had a couple of rounds of being sick, but never to the point that I was hospitalized.

The health of others with cystic fibrosis is also never far from Vallillees thoughts. Hes been promoting an online House of Commons petition aimed at helping Canadians with cystic fibrosis, cancer and other life-threatening diseases to have access to medicines and clinical trials that could save, prolong and/or improve their lives.

And then there is fundraising that has always been a special cause for Vallillees family, especially during May, which is Cystic Fibrosis Month.

COVID-19 will have an impact in terms of preventing people from coming together physically in large numbers to fundraise. In some areas that held traditional walks each May, virtual walks are being held instead.

In other words, COVID wont silence efforts in more ways than one.

On Saturday, May 16, from noonto 12 a.m. he and others will take part in an event called the 12-Hour Sing-A-Thon for Cystic Fibrosis via Facebook live. Darrin Harvey of 89.3 K-Rock will the celebrity host for the kickoff that day and Vallillee and Eben Higgins (both living with CF) will perform and also present numerous East Coast musicians from the region. The goal is to entertain, raise awareness and fundraise. Information about 12-Hour Sing-A-Thon for Cystic Fibrosis can be found on Facebook.

Vallillee is the second oldest person that he knows of in Nova Scotia living with cystic fibrosis.

Every cystic has this timeline, but we dont know what the timeline is, its all dependent on how healthy we are, if we take care of ourselves, if we get exposed to some bug, he says.

But Vallillee doesnt focus on getting sick. Instead, his focus is always on staying well.

And thats where his songs help.

Its the medicine of music, he says.

And he hopes its contagious.

Cystic fibrosis (CF) is the most common fatal genetic disease affecting Canadian children and young adults. At present, there is no cure.

CF causes various effects on the body, but mainly affects the digestive system and lungs. The degree of CF severity differs from person to person, however, the persistence and ongoing infection in the lungs, with the destruction of lungs and loss of lung function, will eventually lead to death in the majority of people with CF.

Typical complications caused by cystic fibrosis are:

It is estimatedone in every 3,600 children born in Canada has CF. More than 4,300 Canadian children, adolescents, and adults with cystic fibrosis attend specialized CF clinics.

Visit the Cystic Fibrosis Canada website to learn more and to see how you can donate.

You can also visit the Cystic Fibrosis Atlantic Canada website

CF is a multi-system disorder that produces a variety of symptoms including:

CF is a genetic disease that occurs when a child inherits two defective copies of the gene responsible for cystic fibrosis, one from each parent. Approximately, one in 25 Canadians carry one defective copy of the CF gene. Carriers do not have CF, nor do they exhibit any of the related symptoms.

When two CF carriers have a child, there is a 25 percent chance that the child will be born with CF. There is also a 50 per cent chance that the child will be a carrier, and a 25 per cent chance that the child will not be a carrier, nor have CF.

RELATED:

Link:
The medicine of music: Tim Vallillee turns to song as therapy for cystic fibrosis and for life - TheChronicleHerald.ca

COVID-19 Stem Cell Therapies Pipeline, 2020 Report – ResearchAndMarkets.com – Business Wire

DUBLIN--(BUSINESS WIRE)--The "COVID-19 Stem Cell Therapies Pipeline" report has been added to ResearchAndMarkets.com's offering.

Complementing the slew of vaccines in development are the upcoming stem cell therapies, aimed at boosting patients' immune systems and eliminating pathogens. The COVID-19 Stem Cell Therapies Pipeline report provides comprehensive data analysis of 22 organizations developing stem cell therapies for COVID-19 globally from the Americas, Europe, the Middle East and Africa (EMEA), and Asia-Pacific region (APAC).

Some report features include:

The report covers stem cell therapy market with detailed review of their cell therapy research and development including the stages of production (pre-clinical, clinical and commercial). In addition, it pulls together insights from interviews and surveys with key opinion leaders. It seeks to holistically inform the community on the current status of COVID-19 stem cell therapy development and discover future opportunities for collaboration, technology transfer and co-development.

Key Topics Covered

Section 1

Section 2: COVID-19 Stem Cell Therapies Pipeline in the Americas

Section 3: COVID-19 Stem Cell Therapies Pipeline in EMEA

Section 4: COVID-19 Stem Cell Therapies Pipeline in APAC

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/kpp6tj

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Role of science highlighted in pandemic fight – Chinadaily.com.cn –

Members of the medical team from Beijing's China-Japan Friendship Hospital visit a novel coronavirus pneumonia patient in an ICU ward at Tongji Hospital in Wuhan, Central China's Hubei province, on March 25, 2020. [Photo by Zhu Xingxin/chinadaily.com.cn]

Experts exploring new, innovative approaches to tackle novel coronavirus

Science and technology have played, and will continue to play, a decisive role in mitigating the pandemic, whether it is by discovering new features about the novel coronavirus, looking for new treatment or vaccines or offering expertise in psychological services, experts said.

But science does not always proceed in an unambiguous straight line toward progress. These undertakings, especially those involve pushing boundaries deeper into the unknown, are time-consuming, complicated and unpredictable. So it is important for the public to understand the scientific process to fully respect and appreciate these efforts, they said.

As President Xi Jinping has said, the COVID-19 epidemic is the "fastest spreading, most infectious and most challenging public health emergency since the birth of New China". He has also stressed that epidemic control efforts require the support of science and technology and urged scientists who are working on treatment and a vaccine to accelerate their research while upholding rigorous scientific practices and ensuring their products are safe.

With the leadership of Xi and joint efforts by the whole of society, the epidemic is now under control in China, said Huai Jinpeng, executive vice-president of the China Association for Science and Technology.

"But the disease is still spreading across the globe, and there is a strong downward pressure for the world economy and a noticeable spike in instability and uncertainty," he said at a meeting with the nation's science officials on April 30.

During this critical juncture, Chinese scientists need to be even more hardworking and pragmatic, and make a greater contribution to the nation's post-epidemic socioeconomic recovery with science and innovation, Huai said.

At the same time, they also need to expand their network of cooperation at home and abroad. Science officials and workers should maintain high ethical and professional standards, and be a role model for society, he added.

Wan Gang, president of the China Association for Science and Technology, said the nation's science workers were immediately mobilized to tackle the epidemic when the outbreak began, and have provided crucial scientific support in controlling the disease and assisting the socioeconomic recovery.

Communication is also a key aspect of the overall disease prevention and control effort, he said, adding that the various COVID-19 related information platforms under the association have attracted over 7 billion views in the past few months.

When Chinese microbiologist Wang Jun volunteered to go to Wuhan, Hubei province, to help the city's hospitals research the novel coronavirus, he said he felt like he was heading into a "battlefield".

The motive behind his action was simple. "Our institute (the Institute of Microbiology of the Chinese Academy of Sciences) has been researching the virus since the outbreak began," Wang said on April 20.

"With Wuhan being the first place to have reported the disease, I had a gut feeling that there must have been many questions that our front-line medical staff didn't even know existed, so we had to go to the battlefront to learn about the situation and their needs," he said.

Since the outbreak began, the academy has sent dozens of researchers to Wuhan. Their work has played a major role in the overall epidemic control effort. Their five main objectives were viral research, creating new diagnostic tools, testing clinical treatments, health evaluation for recovered patients and psychological counseling.

Wang said his team had discovered that children, who were believed to be less susceptible to COVID-19, could still spread the disease even when their symptoms were mild, making them potential asymptomatic carriers that might float under the diagnostic radar.

The virus also has some very intricate immunological effects that would make case tracing via antibody tests more difficult, so "more research is definitely needed", he added.

Jin Qi, director of the Chinese Academy of Medical Sciences' Institute of Pathogen Biology, said that scientists' understanding of the novel coronavirus remains limited and is constantly expanding, and with new information unearthed, new questions would emerge.

For example, most researchers agreed that a 14-day quarantine is generally sufficient for a patient to show symptoms, but there are now rare cases in which patients experience the onset of symptoms well after the two-week period, Jin said.

Drugs and vaccines

Wang Guiqiang, head of Peking University First Hospital's department of infectious diseases, said at a seminar in late April that drugs and vaccines are crucial for stopping the pandemic for good, but this will require time and effort by scientists around the globe.

China has three vaccines, one vectorwhich uses just a gene from the coronavirusand two inactivated, currently in Phase II clinical trials. The vector vaccine is spearheaded by Chen Wei, a senior preventive medical expert, and the results for the Phase II trial are set to be published in May, according to official sources.

The two inactivated vaccines were developed respectively by the Wuhan Institute of Biological Products Co Ltd and Sinovac Research & Development Co Ltd.

Zhong Nanshan, a renowned respiratory expert, told People's Daily last month that although there has not been a wonder cure found for COVID-19, some drugs have proved to be effective to some extent.

"We're testing a variety of drugs, such as chloroquine, and experiment results have shown the drug is definitely effective," he said, adding that scientists are analyzing the data and would publish their findings soon.

Some traditional Chinese medicines, including Lianhua Qingwen Capsules, are also being studied. For the capsule, Zhong said although its anti-viral effect against COVID-19 isn't that pronounced, it does have a "remarkable anti-inflammatory effect" that can help patients recover quicker.

A major component of all scientific work is about testing available knowledge and methods, but not all tests can return positive results. Discovering what works, and, sometimes more importantly, what doesn't work and why, is crucial in expanding humanity's knowledge of the disease.

Cao Bin, vice-president of China-Japan Friendship Hospital, said at a seminar last month that they had found Lopinavir/Ritonavir, a combination of anti-HIV drugs that showed potential in treating COVID-19 in the early days of the outbreak, did not produce desirable results.

In late April, the Lancet medical journal published a study by Cao on his clinical trials on remdesivir in China. The study said the experimental drug from the United States did not significantly speed up the recovery of critically ill patients compared with the control group.

The authors warn that interpretation of their study is limited because it only recruited 237 adults, rather than the target of 453 patients, due to the rapid decline of COVID-19 cases in China. They concluded that more research is needed.

Pushing boundaries

Through strong government support and hard work, Chinese scientists are also exploring new and innovative ways to tackle the novel coronavirus.

Zhang Linqi, a professor at Tsinghua University School of Medicine in Beijing, said his team has been using antibodies to "drive a wedge" between the virus' spike proteinits "key" for entering cellsand the receptor that it binds to.

That would effectively block the virus from entry. It has been very effective in animal tests, and may serve to inspire new vaccine candidates, he said at an online seminar in late April.

Scientists have discovered that there is a small but potent section of the spike protein that does most of the work called the receptor-binding domain, or RBD.

Knowing that, Zhang's team, along with scientists from Shenzhen Third People's Hospital, found two antibodies that, together, can insert themselves at the junction of the RBD and the cell's receptor, blocking the virus from latching onto the cell.

Zhang said they are testing the blocking effect in possible vaccines, and early results are "really encouraging". But research is still in its early stages and more rigorous studies and tests are needed, he added.

Hu Baoyang, executive president of the Chinese Academy of Sciences' Institute of Stem Cell and Regenerative Medicine, said since arriving in Wuhan on March 1, his team had been busy testing stem cell therapy to calm the overreacting immune system and repair the lung tissue of severe and critically ill patients.

In the 46 days that followed, Hu and his team traveled to 13 hospitals and screened over 650 candidate patients for this innovative treatment. At a news briefing on April 16, Sun Yanrong, deputy director of the China National Center for Biotechnology Development, said over 200 patients in Wuhan had received stem cell therapy, and current results show the treatment can improve the recovery rate of severely ill patients and is generally safe.

However, stem cell therapy is far from perfect. Stem cells can differentiate into various types of cells, and some might turn cancerous, according to the University of Nebraska Medical Center. Some stem cells are also difficult to isolate and cultivate in large quantities, so more research and testing are also needed.

"Labs are our bastions, and our scientific research is the weapon against the epidemic," Hu said.

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UB investigators uncover cellular mechanism involved in Krabbe disease – UB Now: News and views for UB faculty and staff – University at Buffalo…

A group of UB researchers have published a paper that clarifies certain cellular mechanisms that could lead to improved outcomes in patients with globoid cell leukodystrophy, commonly known as Krabbe disease.

The paper, titled Macrophages Expressing GALC Improve Peripheral Krabbe Disease by a Mechanism Independent of Cross-Correction, was published May 5 in the journal Neuron.

The research was led by Lawrence Wrabetz and M. Laura Feltri. Wrabetz and Feltri head the Hunter James Kelly Research Institute and both are professors in the departments of Biochemistry and Neurology in the Jacobs School of Medicine and Biomedical Sciences at UB.

The institute is named for the son of former Buffalo Bills quarterback Jim Kelly. Hunter Kelly died at age 8 in 2005 from complications of Krabbe disease.

Krabbe disease is a progressive and fatal neurologic disorder that usually affects newborns and causes death before a child reaches the age of 2 or 3.

Traditionally, hematopoietic stem cell transplantation, also known as a bone marrow transplant, has improved the long-term survival and quality of life of patients with Krabbe disease, but it is not a cure.

It has long been assumed that the bone marrow transplant works by a process calledcross-correction, in which an enzyme called GALC is transferred from healthy cells to sick cells.

Using a new Krabbe disease animal model and patient samples, the UB researchers determinedthatin reality cross-correctiondoes not occur. Rather, the bone marrow transplant helps patients through a different mechanism.

The researchers first determined which cells are involved in Krabbe disease and by which mechanism. They discovered that both myelin-forming cells, or Schwann cells, and macrophages require the GALC enzyme, which is missing in Krabbe patients due to genetic mutation.

Schwann cells require GALC to prevent the formation of a toxic lipid called psychosine, which causes myelin destruction and damage to neurons. Macrophages require GALC to aid with the degradation of myelin debris produced by the disease.

The research showed that hematopoietic stem cell transplantation does not work bycross-correction, but by providing healthy macrophages with GALC.

According to Feltri, the data reveal that improvingcross-correctionwould be a way to makebone marrow transplants and other experimental therapies such as gene therapy more effective.

Bone marrow transplantation and other treatments for lysosomal storage disorders, such as enzyme replacement therapy, have historically had encouraging but limited therapeutic benefit, says study first author Nadav I. Weinstock, an MD-PhD student in the Jacobs School. Our work defined the precise cellular and mechanistic benefit of bone marrow transplantation in Krabbe disease, while also shedding light on previously unrecognized limitations of this approach.

Future studies, using genetically engineered bone marrow transplantation or other novelapproaches,may one day build on our findings and eventually bridge the gap for effectively treating patients with lysosomal disease, he continues.

UB investigators included Daesung Shin, research assistant professor at the Hunter James Kelly Research Institute; Nicholas Silvestri, clinical associate professor of neurology, Jacobs School; Narayan Dhimal, PhD student; Chelsey B. Reed, MD-PhD student; and undergraduate student Oliver Sampson.

Also participating in the research were Eric E. Irons, MD-PhD student, and Joseph T.Y. Lau, a distinguished faculty member from the Department of Molecular and Cellular Biology at Roswell Park Comprehensive Cancer Center.

The research was funded by multiple grants from the National Institutes of Health awarded to Weinstock, Shin, Wrabetz and Feltri, and also supported by Hunters Hope.

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Gilbert Paterson band students to participate in worldwide documentary – Medicine Hat News

By Jensen, Randy on May 8, 2020.

LETHBRIDGE HERALD

A group of students from Gilbert Paterson Middle Schools band class are working at being part of the worlds largest band.

Paterson band teacher Karly Lewis, who continues to teach her students online, became aware of a feature documentary that tells the story of world renowned trumpeter Ryan Anthony, who is proving to the world that art is essential for survival while battling his own terminal cancer.

Anyone can participate and theyve created band parts from beginner level to professional, said Lewis. Kids will be submitting videos of their parts for the documentary. Out of 225 band members I have about 40 so far and the due date is May 15.

According to the documentary webpage, Anthony was at the top of his game when eight years ago he was diagnosed with Multiple Myeloma. Despite crippling treatments, he continued to play his music and could often be found suffering the after effects of chemotherapy backstage moments before he was due to perform. Despite what he was going through, Anthony continued to play every note as if it were his last.

Anthonys family organized a charity Cancer Blows which has produced concerts featuring renowned musicians to raise awareness and funds to find a cure.

However, at the beginning of this year, despite stem cell transplants, Anthony learned his cancer had aggressively returned and he had likely six to 12 months to live.

A crew of Los Angeles-based documentary filmmakers have decided to capture Anthonys battle against terminal cancer. A huge part of the story is how he took the musical piece Song of Hope by Peter Meechan and used it in his fight. It has become an anthem of strength, togetherness and hope that has been featured on concert programs around the world.

The filmmakers then reached out to as many musicians as possible, no matter what their level, to unite and perform Song of Hope alongside Anthony. Each musician downloads the music from the website, performs their instrumental part and sends in their video which will then be stitched together into one music video.

This documentary will involve players worldwide and I will have Grade 6 kids taking part which is amazing, said Lewis. Just their images of them being part of this documentary will be exciting for them.

For more information or to participate visit songforhopemovie.com.

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Rituximab Offers No Extra Benefit to Induction Chemo in ALL – Medscape

Adding the anti-CD20 monoclonal antibody rituximab (various brands) to induction chemotherapy in patients with B-precursor acute lymphoblastic leukaemia (B-ALL) does not improve outcomes, UK researchers have found in a primary analysis of phase 3 trial data.

However, a separate examination of findings from the same study may nevertheless point to an update to the genetic classification for the disease that could help in creating an overall combined clinical and genetic risk score.

The research was published as an abstract from the British Society for Haematology 60th Annual Scientific Meeting, which was cancelled due to the COVID-19 pandemic.

UKALL14 involved patients with B-ALL aged 2565 years, regardless of Philadelphia chromosome (Ph) status or CD20 expression, who were randomised to standard induction chemotherapy (SOC) with or without 4 doses of rituximab (SOC+R).

Focusing on patients recruited after an amendment to the SOC regimen in April 2012, the team conducted an intention-to-treat analysis in 288 SOC patients and 289 given SOC+R, of whom 95.5% received all 4 doses of the immunotherapy.

Adele Fielding, professor of haematology at University College London Cancer Institute, London, and colleagues report that complete remission rates, at 92.7% with SOC and 94.8% with SOC+R, were similar in the two treatment arms.

There was also no difference in minimal residual disease (MRD) rates, with 42.2% and 41.8%, respectively, negative for residual disease.

Adverse, including severe, event rates were similar between the two cohorts, and there was no difference in non-relapse mortality.

After a median follow-up of 50.5 months, the researchers calculate that the 3-year event-free survival (EFS) for patients given SOC was 41.9% versus 48.7% for those receiving SOC+R, at a hazard ratio of 0.88 (p=0.28).

This contrasts with the French GRAALL-2005/R study, in which adults aged 1859 years with CD20-positive, Phnegative ALL were randomised to chemotherapy with or without rituximab, with a total of 16 to 18 infusions given across all treatment phases.

Their results indicated that adding rituximab to the ALL chemotherapy protocol improved outcomes, increasing EFS by 33% versus chemotherapy alone (p=0.04).

Prof Fielding told Medscape News UK that, for UKALL14, they had "hypothesised that giving rituximab early would make the difference, namely in helping to eliminate MRD early on".

"We were anxious not to give too much in case of toxicity from infections. It turned out that it is not toxic and doesnt seem to work to eliminate MRD early on."

She added that, in fact, "the French data showed that too," which prompts her to wonder at the mechanism of action of rituximab in B-ALL.

"Maybe you need more doses at times when patients have functional neutrophils or macrophages, or natural killer cells."

Prof Fielding also pointed out that, in the French study, they focused on patients with Ph-negative disease and in those in whom more than 20% of blasts expressed CD20.

"An important finding from our workis that the level of CD20 expression does not correlate with response to rituximab."

Approached for comment, Rachel Kahn, research communications manager at Blood Cancer UK, said that, "the immunotherapy drug rituximab remains a vital treatment for many types of blood cancer".

She told Medscape News UK, however, that "this interesting research suggests that there may not be any additional benefit of taking this drug for people with ALL".

She highlighted that the results nevertheless suggested that patients who underwent myeloablative allogeneic stem cell transplant (MAallaSCT) appeared to derive a significant benefit from adding rituximab to SOC.

Three-year EFS was 50.7% among MAallaSCT patients given SOC alone versus 72.2% in those receiving SOC+R, at a hazard ratio of 0.47 (p=0.03), which was related to a reduction in relapse risk.

This effect was not seen in patients given reduced intensity allogeneic stem cell transplantation or in maintenance groups, prompting Rachel Kahn to call for further research to identify which patients with ALL "may benefit from taking rituximab".

Prof Fielding said that, as they "do not have any plausible biological explanation" for the finding, the team is "going to be cautious about interpreting" it.

Overall, she feels that, as rituximab is "safe, its probably better to give it to everyone", as "our ability to do that is greater than our ability to do proper flow cytometry in local centres to accurately quantify CD20".

In a separate analysis, Prof Fielding and colleagues looked at all 653 patients who started treatment both before and after the SOC regimen amendment, of whom 49% were found to have high-risk chromosomal abnormalities.

These included 31% with BCR-ABL1, 8% with KMT2A-AFF1, 9% with HoL and 5% with CK abnormalities.

CK and HoL patients had lower 3-year overall survival than the overall cohort, at 24% and 19%, respectively, versus 52%, while patients with KMT2A-AFF1 fusion had an overall survival of 44% and BCR-ABL1 patients had a similar survival to the overall group.

The team also identified a series of other chromosomal abnormalities, including 1.3% with ABL-class fusions and the same proportion with JAK-STAT abnormalities, the latter had reduced 3-year overall survival, at 35%.

In contrast, among the 3% of patients with ZNF384 fusions, only two relapsed and none died.

Having found that secondary copy number alterations affecting key genes had no impact on outcomes, the team proposed "an amendment to the genetic risk classification for adult ALL", consisting of:

very high risk: CK, HoL or JAK-STAT abnormalities

high risk: all KMT2A fusions

tyrosine kinase sensitive: BCR-ABL1 and ABL-class fusions

low-risk: ZNF384 fusions

standard risk: all other patients

The team writes: "The integration of these primary genetic risk factors with other risk factors such as age, white cell count and MRD into an overall risk score is a key goal of our current work."

Prof Fielding said that the "immediate goal" of the team is "evaluating an overall risk score in our next trial, UKALL15, which has been submitted to Cancer Research UK for funding".

Rachel Kahn commented that "this research is a key example of how important it is to continue developing risk scores based on the make-up of the cancer, which can help clinicians understand how likely someone is to respond to treatment".

"This study shows that further clues can be found based on changes to a patients chromosomes."

She continued: "The more we know about how abnormalities influence how risky a cancer is thought to be, the closer we get to being able to personalise treatment to each individual to give them the most effective treatments and the best possible chance of survival."

The study was funded by Cancer Research UK.

No conflicts of interest declared.

[However, Fielding declared to ASH : Amgen: Consultancy; Novartis: Consultancy; Pfizer: Consultancy; Incyte: Consultancy.

BSH 2020: Abstracts BSH2020-OR-001 & BSH2020-OR-004

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Rituximab Offers No Extra Benefit to Induction Chemo in ALL - Medscape

ROCKET PHARMACEUTICALS : Management’s Discussion and Analysis of Financial Condition and Results of Operations (form 10-Q) – marketscreener.com

You should read the following discussion and analysis of our financial conditionand results of operations together with the condensed consolidated financialstatements and related notes that are included elsewhere in this QuarterlyReport on Form 10-Q and our Annual Report on Form 10-K for the fiscal year endedDecember 31, 2019 filed with the U.S. Securities and Exchange Commission, or theSEC, on March 6, 2020, or our 2019 Form 10-K. This discussion containsforward-looking statements based upon current plans, expectations and beliefsthat involve risks and uncertainties. Our actual results may differ materiallyfrom those anticipated in these forward-looking statements as a result ofvarious factors, including, but not limited to, those discussed in the sectionentitled "Risk Factors" and elsewhere in this Quarterly Report on Form 10-Q. Inpreparing this MD&A, we presume that readers have access to and have read theMD&A in our 2019 Form 10-K, pursuant to Instruction 2 to paragraph (b) of Item303 of Regulation S-K. Unless stated otherwise, references in this QuarterlyReport on Form 10-Q to "us," "we," "our," or our "Company" and similar termsrefer to Rocket Pharmaceuticals, Inc.

We are a clinical-stage, multi-platform biotechnology company focused on thedevelopment of first, only and best-in-class gene therapies, with directon-target mechanism of action and clear clinical endpoints, for rare anddevastating diseases. We currently have three clinical-stage ex vivo lentiviralvector ("LVV") programs currently enrolling patients in the US and EU forFanconi Anemia ("FA"), a genetic defect in the bone marrow that reducesproduction of blood cells or promotes the production of faulty blood cells,Leukocyte Adhesion Deficiency-I ("LAD-I"), a genetic disorder that causes theimmune system to malfunction and Pyruvate Kinase Deficiency ("PKD"), a rare redblood cell autosomal recessive disorder that results in chronic non-spherocytichemolytic anemia. Of these, both the Phase 2 FA program and the Phase 1/2 LAD-Iprogram are in registration-enabling studies in the US and EU. In addition, inthe US we have a clinical stage in vivo adeno-associated virus ("AAV") programfor Danon disease, a multi-organ lysosomal-associated disorder leading to earlydeath due to heart failure. Finally, we have a pre-clinical stage LVV programfor Infantile Malignant Osteopetrosis ("IMO"), a genetic disorder characterizedby increased bone density and bone mass secondary to impaired bone resorption -this program is anticipated to enter the clinic in 2020. We have globalcommercialization and development rights to all of these product candidatesunder royalty-bearing license agreements. Additional work in the discovery stagefor an FA CRISPR/CAS9 program as well as a gene therapy program for the lesscommon FA subtypes C and G is ongoing.

Recent Developments

On February 20, 2020, we entered into separate, privately negotiated exchangeagreements (the "Exchange Agreements") with certain holders of our outstanding5.75% Convertible Senior Notes due 2021 (the "2021 Convertible Notes") to extendthe maturity date by one year. Pursuant to the Exchange Agreements, we exchangedapproximately $39.35 million aggregate principal amount of the 2021 ConvertibleNotes (which represents approximately 76% of the aggregate outstanding principalamount of the 2021 Convertible Notes) for (a) approximately $39.35 millionaggregate principal amount of 6.25% Convertible Senior Notes due August 2022(the "2022 Convertible Notes") (an exchange ratio equal to 1.00 2022 ConvertibleNote per exchanged 2021 Convertible Note) and (b) $119,416 in cash to pay theaccrued and unpaid interest on the exchanged 2021 Convertible Notes from, andincluding, February 1, 2020 to February 20, 2020. The 2022 Convertible Noteswere issued in private placements exempt from registration in reliance onSection 4(a) (2) of the Securities Act of 1933, as amended (the "SecuritiesAct"). Upon completion of the exchange transactions, approximately $12.65million aggregate principal amount of 2021 Convertible Notes remainedoutstanding.

Gene Therapy Overview

Genes are composed of sequences of deoxyribonucleic acid ("DNA"), which code forproteins that perform a broad range of physiologic functions in all livingorganisms. Although genes are passed on from generation to generation, geneticchanges, also known as mutations, can occur in this process. These changes canresult in the lack of production of proteins or the production of alteredproteins with reduced or abnormal function, which can in turn result in disease.

Gene therapy is a therapeutic approach in which an isolated gene sequence orsegment of DNA is administered to a patient, most commonly for the purpose oftreating a genetic disease that is caused by genetic mutations. Currentlyavailable therapies for many genetic diseases focus on administration of largeproteins or enzymes and typically address only the symptoms of the disease. Genetherapy aims to address the disease-causing effects of absent or dysfunctionalgenes by delivering functional copies of the gene sequence directly into thepatient's cells, offering the potential for curing the genetic disease, ratherthan simply addressing symptoms.

We are using modified non-pathogenic viruses for the development of our genetherapy treatments. Viruses are particularly well suited as delivery vehiclesbecause they are adept at penetrating cells and delivering genetic materialinside a cell. In creating our viral delivery vehicles, the viral (pathogenic)genes are removed and are replaced with a functional form of the missing ormutant gene that is the cause of the patient's genetic disease. The functionalform of a missing or mutant gene is called a therapeutic gene, or the"transgene." The process of inserting the transgene is called "transduction."Once a virus is modified by replacement of the viral genes with a transgene, themodified virus is called a "viral vector." The viral vector delivers thetransgene into the targeted tissue or organ (such as the cells inside apatient's bone marrow). We have two types of viral vectors in development, LVVand AAV. We believe that our LVV and AAV-based programs have the potential tooffer a long-lasting and significant therapeutic benefit to patients.

Gene therapies can be delivered either (1) ex vivo (outside the body), in whichcase the patient's cells are extracted and the vector is delivered to thesecells in a controlled, safe laboratory setting, with the modified cells thenbeing reinserted into the patient, or (2) in vivo (inside the body), in whichcase the vector is injected directly into the patient, either intravenously("IV") or directly into a specific tissue at a targeted site, with the aim ofthe vector delivering the transgene to the targeted cells.

We believe that scientific advances, clinical progress, and the greaterregulatory acceptance of gene therapy have created a promising environment toadvance gene therapy products as these products are being designed to restorecell function and improve clinical outcomes, which in many cases includeprevention of death at an early age.

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The chart below shows the current phases of development of Rocket's programs andproduct candidates:

LVV Programs. Rocket's LVV-based programs utilize third-generation,self-inactivating lentiviral vectors to target selected rare diseases.Currently, Rocket is developing LVV programs to treat FA, LAD-I, PKD, and IMO.

Fanconi Anemia Complementation Group A (FANCA):

FA, a rare and life-threatening DNA-repair disorder, generally arises from amutation in a single FA gene. An estimated 60 to 70% of cases arise frommutations in the Fanconi-A ("FANCA") gene, which is the focus of our program. FAresults in bone marrow failure, developmental abnormalities, myeloid leukemiaand other malignancies, often during the early years and decades of life. Bonemarrow aplasia, which is bone marrow that no longer produces any or very few redand white blood cells and platelets leading to infections and bleeding, is themost frequent cause of early morbidity and mortality in FA, with a median onsetbefore 10 years of age. Leukemia is the next most common cause of mortality,ultimately occurring in about 20% of patients later in life. Solid organmalignancies, such as head and neck cancers, can also occur, although at lowerrates during the first two to three decades of life.

Although improvements in allogeneic (donor-mediated) hematopoietic stem celltransplant ("HSCT"), currently the most frequently utilized therapy for FA, haveresulted in more frequent hematologic correction of the disorder, HSCT isassociated with both acute and long-term risks, including transplant-relatedmortality, graft versus host disease ("GVHD"), a sometimes fatal side effect ofallogeneic transplant characterized by painful ulcers in the GI tract, livertoxicity and skin rashes, as well as increased risk of subsequent cancers. Ourgene therapy program in FA is designed to enable a minimally toxic hematologiccorrection using a patient's own stem cells during the early years of life. Webelieve that the development of a broadly applicable autologous gene therapy canbe transformative for these patients.

Each of our LVV-based programs utilize third-generation, self-inactivatinglentiviral vectors to correct defects in patients' HSCs, which are the cellsfound in bone marrow that are capable of generating blood cells over a patient'slifetime. Defects in the genetic coding of HSCs can result in severe, andpotentially life-threatening anemia, which is when a patient's blood lacksenough properly functioning red blood cells to carry oxygen throughout the body.Stem cell defects can also result in severe and potentially life-threateningdecreases in white blood cells resulting in susceptibility to infections, and inplatelets responsible for blood clotting, which may result in severe andpotentially life-threatening bleeding episodes. Patients with FA have a geneticdefect that prevents the normal repair of genes and chromosomes within bloodcells in the bone marrow, which frequently results in the development of acutemyeloid leukemia ("AML"), a type of blood cancer, as well as bone marrow failureand congenital defects. The average lifespan of an FA patient is estimated to be30 to 40 years. The prevalence of FA in the US and EU is estimated to be about4,000, and given the efficacy seen in non-conditioned patients, the addressableannual market opportunity is now thought to be in the 400 to 500 range.

We currently have one LVV-based program targeting FA, RP-L102. RP-L102 is ourlead lentiviral vector based program that we in-licensed from Centro deInvestigaciones Energticas, Medioambientales y Tecnolgicas ("CIEMAT"), whichis a leading research institute in Madrid, Spain. RP-L102 is currently beingstudied in our sponsored Phase 2 registrational enabling clinical trialstreating FA patients initially at the Center for Definitive and CurativeMedicine at Stanford University School of Medicine ("Stanford") and HospitalInfantil de Nino Jesus ("HNJ") in Spain. The Phase 2 portion of the trial isexpected to enroll ten patients total from the U.S. and EU. Patients willreceive a single IV infusion of RP-L102 that utilizes fresh cells and "ProcessB" which incorporates a modified stem cell enrichment process, transductionenhancers, as well as commercial-grade vector and final drug product.

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Table of ContentsIn October 2019, at the European Society of Cell and Gene Therapy ("ESGCT") 2019Annual Congress, long-term Phase 1/2 clinical data of RP-L102, from the clinicaltrial sponsored by CIEMAT, for FA "Process A", without the use of myeloablativeconditioning was presented demonstrating evidence of increasing and durableengraftment leading to bone marrow restoration exceeding the 10% thresholdagreed to by the FDA and EMA for the ongoing registration-enabling Phase 2trial. In patient 02002, who received what we consider adequate drug product,hemoglobin levels are now similar to those in the first year after birth,suggesting hematologic correction over the long term.

During the third quarter of 2019, we received alignment from the FDA on thetrial design and the primary endpoint. This alignment was similar to thatpreviously received from the European Medicines Agency ("EMA"). Resistance tomitomycin-C, a DNA damaging agent, in bone marrow stem cells at a minimum timepoint of one year to serve as the primary endpoint for our Phase II study. InDecember 2019, we announced that the first patient of the global Phase 2 studyfor RP-L102 "Process B" for FA received investigational therapy. There will betotal of 10 patients enrolled in the global Phase 2 studies.

In December 2019, we also announced preliminary results from two pediatricpatients treated with "Process B" RP-L102 prior to development of severe bonemarrow failure in our Phase 1 trial of RP-L102 for FA. To evaluate transductionefficiency, an analysis of the proportion of the MMC-resistant colony formingcells was conducted and both patients have thus far exhibited early signs ofengraftment, including increases in blood cell lineages in one patient. Nodrug-related safety or tolerability issues have been reported.

Leukocyte Adhesion Deficiency-I (LAD-I):

LAD-I is a rare autosomal recessive disorder of white blood cell adhesion andmigration, resulting from mutations in the ITGB2 gene encoding for the Beta-2Integrin component, CD18. Deficiencies in CD18 result in an impaired ability forneutrophils (a subset of infection-fighting white blood cells) to leave bloodvessels and enter into tissues where these cells are needed to combatinfections. As is the case with many rare diseases, true estimates of incidenceare difficult; however, several hundred cases have been reported to date.

Most LAD-I patients are believed to have the severe form of the disease. SevereLAD-I is notable for recurrent, life-threatening infections and substantialinfant mortality in patients who do not receive an allogeneic HSCT. Mortalityfor severe LAD-I has been reported as 60 to 75% by age two in the absence ofallogeneic HCST.

We currently have one program targeting LAD-I, RP-L201. RP-L201 is a clinicalprogram that we in-licensed from CIEMAT. We have partnered with UCLA to leadU.S. clinical development efforts for the LAD-I program. UCLA and its Eli andEdythe Broad Center of Regenerative Medicine and Stem Cell Research is servingas the lead U.S. clinical research center for the registrational clinical trialfor LAD-I, and HNJ is serving as the lead clinical site in Spain.

The ongoing open-label, single-arm, Phase 1/2 registration enabling clinicaltrial of RP-L201 has dosed one severe LAD-I patient in the U.S. to assess thesafety and tolerability of RP-L201. The first patient was treated with RP-L201in third quarter 2019. This study has received $6.5 million CLIN2 grant awardfrom the California Institute for Regenerative Medicine ("CIRM") to support theclinical development of gene therapy for LAD-I.

In December 2019, we announced initial results from the first pediatric patienttreated with RP-L201, demonstrating early evidence of safety. Analyses ofperipheral vector copy number ("VCN"), and CD18-expressing neutrophils wereperformed through three months after infusion of RP-L201 to evaluate engraftmentand phenotypic correction. The patient exhibited early signs of engraftment withVCN myeloid levels at 1.5 at three months and CD-18 expression of 45%. No safetyor tolerability issues related to RP-L201 administration (or investigationalproduct) had been identified as of that date. The study is expected to enrollnine patients globally.

Pyruvate Kinase Deficiency (PKD):

Red blood cell PKD is a rare autosomal recessive disorder resulting frommutations in the pyruvate kinase L/R ("PKLR") gene encoding for a component ofthe red blood cell ("RBC") glycolytic pathway. PKD is characterized by chronicnon-spherocytic hemolytic anemia, a disorder in which RBCs do not assume anormal spherical shape and are broken down, leading to decreased ability tocarry oxygen to cells, with anemia severity that can range from mild(asymptomatic) to severe forms that may result in childhood mortality or arequirement for frequent, lifelong RBC transfusions. The pediatric population isthe most commonly and severely affected subgroup of patients with PKD, and PKDoften results in splenomegaly (abnormal enlargement of the spleen), jaundice andchronic iron overload which is likely the result of both chronic hemolysis andthe RBC transfusions used to treat the disease. The variability in anemiaseverity is believed to arise in part from the large number of diverse mutationsthat may affect the PKLR gene. Estimates of disease incidence have rangedbetween 3.2 and 51 cases per million in the white U.S. and EU population.Industry estimates suggest at least 2,500 cases in the U.S. and EU have alreadybeen diagnosed despite the lack of FDA-approved molecularly targeted therapies.Enrollment is currently ongoing and we anticipate treating the first patient inthe third quarter of 2020.

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Table of ContentsWe currently have one LVV-based program targeting PKD, RP-L301. RP-L301 is aclinical stage program that we in-licensed from CIEMAT. The IND for RP-L301 toinitiate a global Phase 1 study was cleared by the FDA in October 2019. Thisprogram has been granted EMA orphan drug disease designation and FDA orphan drugdisease designation ("ODD").

This global Phase 1 open-label, single-arm, clinical trial is expected to enrollsix adult and pediatric transfusion-dependent PKD patients in the U.S. andEurope. Lucile Packard Children's Hospital Stanford will serve as the lead sitein the U.S. for adult and pediatric patients, and Hospital InfantilUniversitario Nio Jess will serve as the lead site in Europe for pediatricsand Hospital Universitario Fundacin Jimnez Daz will serve as the lead site inEurope for adult patients.

Infantile Malignant Osteopetrosis (IMO):

IMO is a genetic disorder characterized by increased bone density and bone masssecondary to impaired bone resorption. Normally, small areas of bone areconstantly being broken down by special cells called osteoclasts, then madeagain by cells called osteoblasts. In IMO, the cells that break down bone(osteoclasts) do not work properly, which leads to the bones becoming thickerand not as healthy. Untreated IMO patients may suffer from a compression of thebone-marrow space, which results in bone marrow failure, anemia and increasedinfection risk due to the lack of production of white blood cells. Untreated IMOpatients may also suffer from a compression of cranial nerves, which transmitsignals between vital organs and the brain, resulting in blindness, hearing lossand other neurologic deficits.

We currently have one LVV-based program targeting IMO, RP-L401. RP-L401 is apreclinical program that we in-licensed from Lund University, Sweden. Thisprogram has been granted ODD and Rare Pediatric Disease designation from theFDA. The FDA defines a "rare pediatric disease" as a serious andlife-threatening disease that affects less than 200,000 people in the U.S. thatare aged between birth to 18 years. The Rare Pediatric Disease designationprogram allows for a sponsor who receives an approval for a product topotentially qualify for a voucher that can be redeemed to receive a priorityreview of a subsequent marketing application for a different product. We havepartnered with UCLA to lead U.S. clinical development efforts for the IMOprogram and anticipate that UCLA will serve as the lead U.S. clinical site forIMO. We intend to file an IND for IMO and commence our clinical trial in thefourth quarter of 2020.

Danon disease is a multi-organ lysosomal-associated disorder leading to earlydeath due to heart failure. Danon disease is caused by mutations in the geneencoding lysosome-associated membrane protein 2 ("LAMP-2"), a mediator ofautophagy. This mutation results in the accumulation of autophagic vacuoles,predominantly in cardiac and skeletal muscle. Male patients often require hearttransplantation and typically die in their teens or twenties from progressiveheart failure. Along with severe cardiomyopathy, other Danon disease symptomscan include skeletal muscle weakness, liver disease, and intellectualimpairment. There are no specific therapies available for the treatment of Danondisease. RP-A501 is in clinical trials as an in vivo therapy for Danon disease,which is estimated to have a prevalence of 15,000 to 30,000 patients in the U.S.and the EU, however new market research is being performed and the prevalence ofpatients may be updated in the future.

In January 2019, we announced the clearance of our IND application by the FDAfor RP-A501, and in February 2019, we were notified by the FDA that we weregranted Fast Track designation for RP-A501. University of California San DiegoHealth is the initial and lead center for our Phase 1 clinical trial.

On May 2, 2019, we presented additional preclinical data at the ASCGT annualmeeting, indicating that high VCN, in Danon disease-relevant organs in both miceand non-human primates ("NHN's"), with high concentrations in heart and livertissue (for NHP, cardiac VCN was approximately 10 times higher on average thanin skeletal muscle and central nervous system), which is consistent withreported results in several studies of heart tissue across different species.There were no treatment-related adverse events or safety issues up to thehighest dose. We have dosed three patients in the RP-A501 phase 1 clinicaltrial. We will continue further enrollment with clinical data read-outs in thefourth quarter of 2020.

As of March 2020, we have dosed three patients in the RP-A501 phase 1 clinicaltrial. This completes the first low dose cohort of the Phase 1 study. Based onthe preliminary safety and efficacy data review of this completed cohort, boththe FDA and IDMC has provided clearance to advance to a higher dose cohort inPhase 1 Trial of RP-A501 for Danon Disease. We will continue further enrollmentwith clinical data read-outs in the second half of 2020.

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In addition to its LVV and AAV programs, we also have a program evaluatingCRISPR/Cas9-based gene editing for FA. This program is currently in thediscovery phase. CRISPR/Cas9-based gene editing is a different method ofcorrecting the defective genes in a patient, where the editing is very specificand targeted to a particular gene sequence. "CRISPR/Cas9" stands for Clustered,Regularly Interspaced Short Palindromic Repeats ("CRISPR") Associated protein-9.The CRISPR/Cas9 technology can be used to make "cuts" in DNA at specific sitesof targeted genes, making it potentially more precise in delivering genetherapies than traditional vector-based delivery approaches. CRISPR/Cas9 canalso be adapted to regulate the activity of an existing gene without modifyingthe actual DNA sequence, which is referred to as gene regulation.

Strategy

We seek to bring hope and relief to patients with devastating, undertreated,rare pediatric diseases through the development and commercialization ofpotentially curative first-in-class gene therapies. To achieve these objectives,we intend to develop into a fully-integrated biotechnology company. In the near-and medium-term, we intend to develop our first-in-class product candidates,which are targeting devastating diseases with substantial unmet need, developproprietary in-house analytics and manufacturing capabilities and continue tocommence registration trials for our currently planned programs. In the mediumand long-term, we expect to submit our first biologics license applications("BLAs"), and establish our gene therapy platform and expand our pipeline totarget additional indications that we believe to be potentially compatible withour gene therapy technologies. In addition, during that time, we believe thatour currently planned programs will become eligible for priority review vouchersfrom the FDA that provide for expedited review. We have assembled a leadershipand research team with expertise in cell and gene therapy, rare disease drugdevelopment and commercialization.

We believe that our competitive advantage lies in our disease-based selectionapproach, a rigorous process with defined criteria to identify target diseases.We believe that this approach to asset development differentiates us as a genetherapy company and potentially provides us with a first-mover advantage.

Financial Overview

Since our inception, we have devoted substantially all of our resources toorganizing and staffing the Company, business planning, raising capital,acquiring or discovering product candidates and securing related intellectualproperty rights, conducting discovery, research and development activities forthe programs and planning for potential commercialization. We do not have anyproducts approved for sale and have not generated revenue from product sales.From inception through March 31, 2020, we raised net cash proceeds ofapproximately $373.1 million from investors through both equity and convertibledebt financing to fund operating activities. As of March 31, 2020, we had cash,cash equivalents and investments of $275.9 million.

Since inception, we have incurred significant operating losses. Our ability togenerate product revenue sufficient to achieve profitability will depend heavilyon the successful development and eventual commercialization of one or more ofthe current or future product candidates and programs. We had net losses of$24.7 million for the three months ended March 31, 2020 and $77.3 million forthe year ended December 31, 2019. As of March 31, 2020, we had an accumulateddeficit of $207.8 million. We expect to continue to incur significant expensesand higher operating losses for the foreseeable future as we advance our currentproduct candidates from discovery through preclinical development and clinicaltrials and seek regulatory approval of our product candidates. In addition, ifwe obtain marketing approval for any of their product candidates, we expect toincur significant commercialization expenses related to product manufacturing,marketing, sales and distribution. Furthermore, we expect to incur additionalcosts as a public company. Accordingly, we will need additional financing tosupport continuing operations and potential acquisitions of licensing or otherrights for product candidates.

Until such a time as we can generate significant revenue from product sales, ifever, we will seek to fund our operations through public or private equity ordebt financings or other sources, which may include collaborations with thirdparties and government programs or grants. Adequate additional financing may notbe available to us on acceptable terms, or at all. We can make no assurancesthat we will be able to raise the cash needed to fund our operations and, if wefail to raise capital when needed, we may have to significantly delay, scaleback or discontinue the development and commercialization of one or more productcandidates or delay pursuit of potential in-licenses or acquisitions.

Because of the numerous risks and uncertainties associated with productdevelopment, we are unable to predict the timing or amount of increased expensesor when or if we will be able to achieve or maintain profitability. Even if weare able to generate product sales, we may not become profitable. If we fail tobecome profitable or are unable to sustain profitability on a continuing basis,then we may be unable to continue our operations at planned levels and be forcedto reduce or terminate our operations.

Revenue

To date, we have not generated any revenue from any sources, including fromproduct sales, and we do not expect to generate any revenue from the sale ofproducts in the near future. If our development efforts for product candidatesare successful and result in regulatory approval or license agreements withthird parties, we may generate revenue in the future from product sales.

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Research and Development Expenses

Our research and development program ("R&D") expenses consist primarily ofexternal costs incurred for the development of our product candidates. Theseexpenses include:

expenses incurred under agreements with research institutions that conduct

research and development activities including, process development,

preclinical, and clinical activities on Rocket's behalf;

costs related to process development, production of preclinical and clinical

materials, including fees paid to contract manufacturers and manufacturing

input costs for use in internal manufacturing processes;

consultants supporting process development and regulatory activities; and

costs related to in-licensing of rights to develop and commercialize our

product candidate portfolio.

We recognize external development costs based on contractual payment schedulesaligned with program activities, invoices for work incurred, and milestoneswhich correspond with costs incurred by the third parties. Nonrefundable advancepayments for goods or services to be received in the future for use in researchand development activities are recorded as prepaid expenses.

Our direct research and development expenses are tracked on a program-by-programbasis for product candidates and consist primarily of external costs, such asresearch collaborations and third party manufacturing agreements associated withour preclinical research, process development, manufacturing, and clinicaldevelopment activities. Our direct research and development expenses by programalso include fees incurred under license agreements. Our personnel, non-programand unallocated program expenses include costs associated with activitiesperformed by our internal research and development organization and generallybenefit multiple programs. These costs are not separately allocated by productcandidate and consist primarily of:

Our research and development activities are central to our business model.Product candidates in later stages of clinical development generally have higherdevelopment costs than those in earlier stages of clinical development. As aresult, we expect that research and development expenses will increasesubstantially over the next several years as we increase personnel costs,including stock-based compensation, support ongoing clinical studies, seek toachieve proof-of-concept in one or more product candidates, advance preclinicalprograms to clinical programs, and prepare regulatory filings for productcandidates.

We cannot determine with certainty the duration and costs to complete current orfuture clinical studies of product candidates or if, when, or to what extent wewill generate revenues from the commercialization and sale of any of our productcandidates that obtain regulatory approval. We may never succeed in achievingregulatory approval for any of our product candidates. The duration, costs, andtiming of clinical studies and development of product candidates will depend ona variety of factors, including:

the scope, rate of progress, and expense of ongoing as well as any future

clinical studies and other research and development activities that we

undertake;

future clinical trial results;

uncertainties in clinical trial enrollment rates;

changing standards for regulatory approval; and

the timing and receipt of any regulatory approvals.

We expect research and development expenses to increase for the foreseeablefuture as we continue to invest in research and development activities relatedto developing product candidates, including investments in manufacturing, as ourprograms advance into later stages of development and as we conduct additionalclinical trials. The process of conducting the necessary clinical research toobtain regulatory approval is costly and time-consuming, and the successfuldevelopment of product candidates is highly uncertain. As a result, we areunable to determine the duration and completion costs of research anddevelopment projects or when and to what extent we will generate revenue fromthe commercialization and sale of any of our product candidates.

Our future research and development expenses will depend on the clinical successof our product candidates, as well as ongoing assessments of the commercialpotential of such product candidates. In addition, we cannot forecast with anydegree of certainty which product candidates may be subject to futurecollaborations, when such arrangements will be secured, if at all, and to whatdegree such arrangements would affect our development plans and capitalrequirements. We expect our research and development expenses to increase infuture periods for the foreseeable future as we seek to complete development ofour product candidates.

The successful development and commercialization of our product candidates ishighly uncertain. This is due to the numerous risks and uncertainties associatedwith product development and commercialization, including the uncertainty of:

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Table of Contents

the scope, progress, outcome and costs of our clinical trials and other

research and development activities;

the efficacy and potential advantages of our product candidates compared to

alternative treatments, including any standard of care;

the market acceptance of our product candidates;

obtaining, maintaining, defending and enforcing patent claims and other

intellectual property rights;

significant and changing government regulation; and

the timing, receipt and terms of any marketing approvals.

A change in the outcome of any of these variables with respect to thedevelopment of our product candidates that we may develop could mean asignificant change in the costs and timing associated with the development ofour product candidates. For example, if the FDA or another regulatory authoritywere to require us to conduct clinical trials or other testing beyond those thatwe currently contemplate for the completion of clinical development of any ofour product candidates that we may develop or if we experience significantdelays in enrollment in any of our clinical trials, we could be required toexpend significant additional financial resources and time on the completion ofclinical development of that product candidate.

General and Administrative Expenses

General and administrative ("G&A") expenses consist primarily of salaries andrelated benefit costs for personnel, including stock-based compensation andtravel expenses for our employees in executive, operational, finance, legal,business development, and human resource functions. In addition, othersignificant general and administrative expenses include professional fees forlegal, patents, consulting, investor and public relations, auditing and taxservices as well as other expenses for rent and maintenance of facilities,insurance and other supplies used in general and administrative activities. Weexpect general and administrative expenses to increase for the foreseeablefuture due to anticipated increases in headcount to support the continuedadvancement of our product candidates. We also anticipate that we will incurincreased accounting, audit, legal, regulatory, compliance and director andofficer insurance costs as well as investor and public relations expenses.

Interest Expense

Interest expense is related to the 2021 Convertible Notes, which mature inAugust 2021, and the 2022 Convertible Notes, which mature in August 2022.

Interest Income

Interest income is related to interest earned from investments.

Critical Accounting Policies and Significant Judgments and Estimates

Our consolidated financial statements are prepared in accordance with generallyaccepted accounting principles in the U.S. The preparation of our financialstatements and related disclosures requires us to make estimates and judgmentsthat affect the reported amounts of assets, liabilities, costs and expenses, andthe disclosure of contingent assets and liabilities in our financial statements.We base our estimates on historical experience, known trends and events andvarious other factors that we believe are reasonable under the circumstances,the results of which form the basis for making judgments about the carryingvalues of assets and liabilities that are not readily apparent from othersources. We evaluate estimates and assumptions on an ongoing basis. Actualresults may differ from these estimates under different assumptions orconditions.

Our significant accounting policies are described in more detail in our 2019Form 10-K, except as otherwise described below.

Results of Operations

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ROCKET PHARMACEUTICALS : Management's Discussion and Analysis of Financial Condition and Results of Operations (form 10-Q) - marketscreener.com

Stem Cell Therapy Market To Boom In Near Future By 2027 Scrutinized In New Research – Cole of Duty

The Covid-19 (coronavirus) pandemic is impacting society and the overall economy across the world. The impact of this pandemic is growing day by day as well as affecting the supply chain. The COVID-19 crisis is creating uncertainty in the stock market, massive slowing of supply chain, falling business confidence, and increasing panic among the customer segments. The overall effect of the pandemic is impacting the production process of several industries including medical devices, pharmaceuticals, healthcare, biotechnology, and many more. Trade barriers are further restraining the demand- supply outlook. As government of different regions have already announced total lockdown and temporarily shutdown of industries, the overall production process being adversely affected; thus, hinder the overall Stem Cell Therapy Market globally. This report on Stem Cell Therapy Market provides the analysis on impact on Covid-19 on various business segments and country markets. The report also showcases market trends and forecast to 2027, factoring the impact of Covid -19 Situation.

The stem cell therapy marketwas valued at US$ 1,534.55 million in 2019 and is expected to grow at a CAGR of 16.7% from 2020to 2027 to reach US$ 5,129.66 million by 2027.

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What is Stem Cell Therapy Market?

Stem cells are preliminary body cells from which all other cells with specialized functions are generated. Under controlled environment in the body or a clinical laboratory, these cells divide to form more cells called daughter cells. Due to the advent of modern health science, these cells play a major role in understanding the occurrence of diseases, generation of advanced regenerative medicines, and drug discovery. There are certain sources such as embryo, bone marrow, body fats, and umbilical cord blood amongst others, where stem cells are generated. The global stem cell therapy market is driven by factors such asincreasing awareness related to the stem cells therapy in effective disease management and growing demand for regenerative medicines. However, high cost related with stem cell therapy is likely to obstruct the growth of the stem cell therapymarket during the forecast period. The growing research and development activities in Asia Pacific region is expected to offer huge growth opportunity for stem cell therapy market.

Researchers are further investigating stem cell therapy in autoimmune disorder. Other adult stem cells based treatments are under clinical trials. Hematopoietic stem cells are currently used for treating more than 80 medical diseases, which include diseases of the immune system, blood disorders, neurological disorders, metabolic disorders, genetic disorders, and several types of cancers like leukemia, lymphoma, etc.Emerging Players in the Stem Cell Therapy Market Research include:

A factor which can be a restraint for Stem Cell Therapy Market can be some companies do not collaborate with service providers or they dont take advantage of digitization as they dont have awareness for the same. Nevertheless, digitization in services is opting by an online company to know more exactly about consumer behavior plus it makes business policies flexible to adopt changes as per the market condition on which success and growth of an organization depend which will give more growth opportunities in coming years.

This report will help you determine and analyze your portfolio of key market players with information such as company profile, components and services offered, financial information from the past three years, and key developments it helps you to develop a strategy to gain a competitive edge in the past 5 years. The market payers from Stem Cell Therapy Market are anticipated to lucrative growth opportunities in the future with the rising demand for Stem Cell Therapy Market in the global market.

Key questions answered by this report:

Global Stem Cell Therapy Market By Type

Global Stem Cell Therapy Market By Treatment

Global Stem Cell Therapy Market ByApplication

Global Stem Cell Therapy Market By End User

Global Stem Cell TherapyMarket By Geography

Stem Cell Therapy Market Table of Contents:

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Stem Cell Therapy Market To Boom In Near Future By 2027 Scrutinized In New Research - Cole of Duty

FDA approves Tabrecta, first targeted therapy to treat metastatic NSCLC – The Cancer Letter

publication date: May. 8, 2020

FDA has granted accelerated approval to Tabrecta (capmatinib) for adult patients with metastatic non-small cell lung cancer whose tumors have a mutation that leads to mesenchymal-epithelial transition exon 14 skipping as detected by an FDA-approved test.

Tabrecta is the first FDA-approved therapy to treat NSCLC with specific mutations (those that lead to mesenchymal-epithelial transition or MET exon 14 skipping).

Tabrecta is sponsored by Novartis.

FDA also approved the FoundationOne CDx assay (Foundation Medicine, Inc.) as a companion diagnostic for Tabrecta. Most patients had tumor samples that were tested for mutations that lead to MET exon 14 skipping using local tests and confirmed with the F1CDx, which is a next-generation sequencing based in vitro diagnostic device capable of detecting several mutations, including mutations that lead to MET exon 14 skipping.

Lung cancer is increasingly being divided into multiple subsets of molecularly defined populations with drugs being developed to target these specific groups, Richard Pazdur, director of the FDA Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDAs Center for Drug Evaluation and Research, said in a statement. Tabrecta is the first approval specifically for the treatment of patients with non-small cell lung cancer whose tumors have mutations that lead to MET exon 14 skipping. This patient population now has an option for a targeted therapy, which they didnt have prior to today.

Efficacy was demonstrated in the GEOMETRY mono-1 trial (NCT02414139), a multicenter, non-randomized, open-label, multicohort study enrolling 97 patients with metastatic NSCLC with confirmed MET exon 14 skipping. Patients received Tabrecta 400 mg orally twice daily until disease progression or unacceptable toxicity.

The main efficacy outcome measures were overall response rate (ORR) determined by a blinded independent review committee using RECIST 1.1 and response duration. Among the 28 treatment-nave patients, the ORR was 68% (95% CI: 48, 84) with a response duration of 12.6 months (95% CI: 5.5, 25.3). Among the 69 previously treated patients, the ORR was 41% (95% CI: 29, 53) with a response duration of 9.7 months (95% CI: 5.5, 13.0).

FDA approves daratumumab and hyaluronidase-fihj for multiple myeloma

FDA has approved daratumumab and hyaluronidase-fihj (Darzalex Faspro) for adult patients with newly diagnosed or relapsed/refractory multiple myeloma. This new product allows for subcutaneous dosing of daratumumab.

Darzalex Faspro is sponsored by Janssen Biotech Inc.

Daratumumab and hyaluronidase-fihj is approved for the following indications that intravenous daratumumab had previously received:

in combination with bortezomib, melphalan and prednisone in newly diagnosed patients who are ineligible for autologous stem cell transplant,

in combination with lenalidomide and dexamethasone in newly diagnosed patients who are ineligible for autologous stem cell transplant and in patients with relapsed or refractory multiple myeloma who have received at least one prior therapy,

in combination with bortezomib and dexamethasone in patients who have received at least one prior therapy,

as monotherapy, in patients who have received at least three prior lines of therapy including a proteasome inhibitor and an immunomodulatory agent or who are double-refractory to a PI and an immunomodulatory agent.

Efficacy of daratumumab and hyaluronidase-fihji (monotherapy) was evaluated in COLUMBA (NCT03277105), an open-label non-inferiority trial randomizing 263 patients to daratumumab and hyaluronidase-fihj and 259 to intravenous daratumumab (daratumumab IV). The trials co-primary endpoints were overall response rate and pharmacokinetic endpoint of the maximum Ctrough on cycle 3, day 1 pre-dose. Daratumumab and hyaluronidase-fihj was non-inferior to daratumumab IV in evaluating these two endpoints.

The ORR was 41.1% for daratumumab and hyaluronidase-fihj and 37.1% for daratumumab IV with a risk ratio of 1.11 (95% CI: 0.89, 1.37). The geometric mean ratio comparing daratumumab and hyaluronidase-fihj to daratumumab IV for maximum Ctrough was 108% (90% CI: 96,122).

Efficacy of daratumumab and hyaluronidase-fihj in combination with VMP (D-VMP) was evaluated in a single-arm cohort of PLEIADES (NCT03412565), a multi-cohort, openlabel trial. Eligible patients were required to have newly diagnosed multiple myeloma and were ineligible for transplant. The major efficacy outcome measure, ORR, was 88.1% (95% CI: 77.8, 94.7).

Efficacy of daratumumab and hyaluronidase-fihj in combination with Rd (D-Rd) was evaluated in a single-arm cohort of this trial. Eligible patients had received at least one prior line of therapy. ORR was 90.8% (95% CI: 81.0, 96.5).

FDA accepts NDA for CC-486 in AML indication

FDA has accepted a New Drug Application for CC-486, an investigational oral hypomethylating agent, for the maintenance treatment of adult patients with acute myeloid leukemia who achieved complete remission, or CR with incomplete blood count recovery, following induction therapy with or without consolidation treatment, and who are not candidates for, or who choose not to proceed to, hematopoietic stem cell transplantation.

CC-486 is sponsored by Bristol Myers Squibb. FDA granted the application Priority Review and set a Prescription Drug User Fee Act goal date of Sept. 3, 2020.

The NDA submission was based on the efficacy and safety results of the phase III QUAZAR AML-001 study, which met the primary endpoint of improved overall survival for patients receiving AML maintenance treatment with CC-486 versus placebo.

Often, newly diagnosed adult patients with AML achieve a complete response with induction therapy, however many patients will relapse and experience a poor outcome. Patients in remission are seeking treatment options that decrease the likelihood of relapse and extend overall survival, Noah Berkowitz, senior vice president of Global Clinical Development, Hematology, at Bristol Myers Squibb, said in a statement.

CC-486 is an investigational therapy that is not approved for any use in any country.

Caris Life Sciences submits two PMA applications to FDA for whole exome and whole transcriptome sequencing

Caris Life Sciences has submitted two Pre-Market Approval applications for MI Exome CDx and MI Transcriptome CDx to FDA.

MI Exome CDx, whole exome sequencing (DNA), and MI Transcriptome CDx, whole transcriptome sequencing (RNA), are precision medicine assays that include key companion diagnostic biomarkers with therapy claims, and detect all classes of alterations including genomic signatures for microsatellite instability, tumor mutation burden, and loss of heterozygosity.

MI Exome CDx is a next-generation sequencing-based test utilizing DNA isolated from formalin-fixed paraffin embedded tumor tissue specimens for the qualitative detection of genomic alterations. MI Exome CDx can identify genetic variants (single nucleotide variants, insertions and deletions), copy number alterations, MSI, TMB and LOH.

MI Transcriptome CDx is a next-generation sequencing-based test that utilizes RNA isolated from formalin-fixed paraffin embedded tumor tissue specimens for the qualitative detection of genomic and transcriptomic alterations. MI Transcriptome CDx is a broad, multi-gene panel utilized to identify gene fusions, transcript variants, genetic variants (single nucleotide variants, insertions and deletions), and gene expression changes. FDA granted MI Transcriptome CDx received Breakthrough Device designation in 2019.

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FDA approves Tabrecta, first targeted therapy to treat metastatic NSCLC - The Cancer Letter