Category Archives: Gene Therapy Clinics

Edited Transcript of FLXN earnings conference call or presentation 12-Mar-20 8:30pm GMT – Yahoo Finance

By adminMarch 17, 2020Gene Therapy Clinics

Woburn Mar 16, 2020 (Thomson StreetEvents) -- Edited Transcript of Flexion Therapeutics Inc earnings conference call or presentation Thursday, March 12, 2020 at 8:30:00pm GMT

* David A. Arkowitz

Flexion Therapeutics, Inc. - CFO

* Michael D. Clayman

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

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

The Benchmark Company, LLC, Research Division - Senior Healthcare Technology Research Analyst

Joh. Berenberg, Gossler & Co. KG, Research Division - Analyst

* Serge D. Belanger

Good afternoon, ladies and gentlemen. And welcome to the Flexion Therapeutics Fourth Quarter and Full Year 2019 Financial Results Conference Call. My name is Daniel, and I will be your coordinator for today. (Operator Instructions)

I'll now turn the call over to the company.

Scott Young, Flexion Therapeutics, Inc. - VP of Corporate Communications & IR [2]

Thank you, Dan. Good afternoon. This is Scott Young, Vice President for Corporate Communications and Investor Relations. Before we begin, I would call your attention to the metrics slide that we will discuss in today's presentation. Those slides can be viewed directly via the webcast, in the 8-K we issued this afternoon or under the Investors tab on flexiontherapeutics.com.

In addition, our Q4 earnings press release and an archive of this conference call, can also be found there. Today's call will be led by Flexion's Chief Executive Officer, Dr. Michael Clayman; and he is joined by David Arkowitz, Flexion's Chief Financial Officer; and Melissa Layman, Flexion's newly appointed 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

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-K for the year ended December 31, 2019, which was filed with the SEC 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 [3]

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Thanks, Scott, and thank you all for joining. Today, I'll recap our commercial progress in 2019, review our life cycle management activities, provide an update on our pipeline and discuss our priorities for this year. After that, I'll turn it over to David for a deeper review of our commercial metrics and a summary of our financial performance, and then we will open the line and take questions.

To begin, as we reported today, we recorded full year ZILRETTA net sales of $73 million for 2019, which is fully in line with the preliminary unaudited revenue estimate we provided in early January. We are very pleased with our sales performance in 2019, which represents growth of more than 220%, compared to our 2018 full year net sales of $22.5 million. Those numbers tell a very compelling story, and they speak to the outstanding work of our field-based teams, the excellent coverage we have for ZILRETTA, the impact from our judicious use of volume-based rebates, but most importantly, our sales performance speaks to the remarkable experience that patients and clinicians are having with ZILRETTA. As you will see in the commercial metrics that David will review, by the end of 2019, our customers had purchased 175,000 units of ZILRETTA. While our data are limited to the account level, that number provides a reasonable surrogate for the number of patients who have been treated with ZILRETTA since it was introduced to the market in late 2017. And we know that many of those patients have received unprecedented pain relief from ZILRETTA. I say this based on real-world feedback from the countless conversations I've had with grateful ZILRETTA patients and their physicians, feedback that is wholly consistent with our compelling clinical trial data.

In fact, last year, we became aware of several professional athletes who received ZILRETTA, namely Rod Woodson, the NFL Hall of Fame Defensive back named as one of the top 100 players of all time. Michael Eruzione, the captain of the 1980 Gold medal winning U.S. Olympic Hockey team and the player who scored the game winning goal against the Russians during the Miracle On Ice; and Chris Dickerson, a former outfielder who played from the New York Yankees and other major league baseball teams. From snow blowing a driveway to playing with their children and grandchildren, to jogging for the first time in years, they all have profoundly moving stories about how ZILRETTA has helped each of them manage their knee OA pain, and thereby, improve their ability to participate in regular everyday activities. As part of our ongoing physician marketing initiatives, we brought all 3 athletes together at a major orthopedic conference last December, where they and their treating physicians, spoke to a standing room crowd of some of the country's leading sports medicine experts. They shared their stories of how ZILRETTA has helped them, and the best words I can use to describe that session are inspiring and humbling.

While all are elite athletes, their ZILRETTA stories echo the scores of e-mails and letters I've received from patients who are so grateful to reengage in things they love doing with less pain. From gardening, playing a round of golf, walking on the beach, hiking, each of these stories serves as a reminder to all of us that while we have impacted more than 175,000 patients, there are 15 million people who see their doctor every year for OA knee pain. And 5 million of them receive an intra-articular injection. The opportunity for ZILRETTA and Flexion is truly massive, which brings me back to our performance in 2019 and our goals for 2020.

Throughout the course of 2019, we saw existing practices increase their use of ZILRETTA and more than 1,600 new accounts start using the product. Today, with the benefit of 2 years in the market, we have actual claims [data that show some slight] (added by company after the call) changes in the payer mix versus our assumptions prior to launch. We now see that the actual payer mix is skewed slightly more toward Medicare, which accounts for roughly 55% of the market versus previous estimates of 50%. 40% is still commercial, but Medicaid, VA and 340B plans are making up roughly 5% of the mix. We view this modest increase in Medicare patients to be incrementally positive as Medicare patients can be injected the same-day they visit without any need for prior authorization. In addition, it is important to point out that commercial coverage for ZILRETTA remains excellent. As we discussed in December, the recent approval of our sNDA resulted in a significantly improved label, which most importantly, removed the onerous not intended for repeat administration wording in the limitation of use statement. Within days of the approval, we developed materials to help our MBMs communicate the label update and we have anecdotal feedback from the field that tells us that the changes have been very well received. While we clearly view the label update as a tailwind. We have always said that we continue to believe that the meaningful impact will be seen over the following quarters and years. From a commercial perspective, our progress in 2019 strengthens our belief that ZILRETTA can become the new standard of care for the intra-articular treatment for OA knee pain in the years ahead. While under normal circumstances, these factors would give us confidence that our 2020 ZILRETTA net sales guidance of $120 million to $135 million is both credible and achievable. Like all businesses, we are acutely attuned to the potential impacts of the coronavirus global pandemic, and we are monitoring this dynamic situation very closely. To date, we have not seen any material impacts on ZILRETTA sales, our ability to access customers or to initiate our clinical trials. However, it is impossible to predict how the outbreak could evolve in the months ahead or what impacts more aggressive social distancing or other containment efforts might have on patients or practices. Regarding our supply chain, we believe we are in a very strong position. We do not source any of our key materials from China, and we presently have approximately 10 months worth of finished product inventory in our warehouses in the U.S. Furthermore, we have an additional 12 months of API, triamcinolone acetonide at our manufacturing facility in the U.K. Again, it is impossible to predict the long-term impacts of the outbreak. But we feel very good about our ability to provide ZILRETTA to patients over the quarters ahead.

Shifting to our clinical development activities, our Phase II trial to investigate the safety and efficacy of ZILRETTA in shoulder OA and adhesive capsulitis, also known as frozen shoulder syndrome continues to advance, and we anticipate data from that trial in 2021.

As we've previously discussed, these 2 conditions combined account for roughly 800,000 injections, and they present an opportunity for us to expand the use of ZILRETTA with a subset of orthopedists, who primarily focus on sports medicine and commonly treat these conditions with steroid injections.

With respect to our pipeline, we've also been making progress with our 2 drug candidates, FX201 and FX301. FX201 is our gene therapy, which holds the potential to provide OA pain relief for at least a year, improve function and potentially modify disease. As we announced last year, the IND for FX201 was accepted by the FDA, and we recently treated the first 2 patients in our Phase I dose-ranging study. We anticipate treating approximately 15 to 24 patients, who will be followed for 104 weeks with initial readout in 2021.

Now we'll move to FX301. Our NAV 1.7 inhibitor, formulated within a thermosensitive hydrogel for administration as a peripheral nerve block for control of postoperative pain. We've held our pre-IND meeting with FDA, and we remain on track to initiate our first FX301 clinical trial in 2021.

Unlike typical local anesthetics, we believe the selective pharmacology of FX301 has the potential to deliver at least 3 to 5 days of effective pain relief, while preserving motor function, which could enable ambulation, rapid discharge from the hospital and early rehabilitation following musculoskeletal surgery.

Finally, regarding our search for a Chief Commercial Officer. We were very excited to announce the appointment of Melissa Layman earlier this week, and I'm delighted that she is able to join us for today's call. As we've said repeatedly, in our search for a CCO, we were looking for someone who had had success leading an entire commercial enterprise, who had deep experience and expertise in each of the key commercial functional domains, who had a track record as a very strong leader and who would positively contribute to our already strong culture. Because Melissa fulfills all of these criteria and is simply a terrific person to interact with. We could not be more pleased to have her take the helm of our commercial organization. Before David summarizes our fourth quarter financials and walks through the commercial metrics, I'd like to give Melissa the opportunity to make a few remarks. Melissa?

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Melissa Layman, [4]

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Thank you, Mike, and thank you for the kind words. It is such a pleasure to be here today and such an honor to join Flexion. While I've barely been in my role for a day, I can already see what an amazing group of people work here. The team has been welcoming, engaging and candid, and that was consistent throughout the entire interview process from the Executive Committee to the Board members. The commitment to patients and passion for winning has been universal, and that was one of the many reasons I wanted to join. Put simply, I can't begin to convey how excited I am about the opportunity that's ahead of us. The strong foundation that's been laid by Mike, David, Steve Meyers, and the rest of the commercial leadership team is truly impressive, and I'm looking forward to working together to grow ZILRETTA's market share and help make it the leading IA treatment in this space. Over the next few months, I'll be working intensively with our commercial organization, and I look forward to representing our commercial effort on future calls. At this point, I'll turn it over to David.

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David A. Arkowitz, Flexion Therapeutics, Inc. - CFO [5]

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Thank you, Melissa. I'll start by walking through our commercial metrics which, as Scott mentioned, can be found on our website and in the 8-K we issued today.

If you look at Slide 2, you can see that we recorded ZILRETTA net sales of $23.7 million in the fourth quarter of 2019, bringing full year 2019 net sales to $73 million. As Mike mentioned, year-over-year growth topped [220%] (corrected by company after the call). As in previous quarters, we expanded our list of target accounts in the fourth quarter to 4,972, and by December 31, 2019, we've called on almost all of them. At the end of the fourth quarter, 3,488 accounts had purchased ZILRETTA, which is an increase of nearly 360 purchasing accounts compared to the end of the third quarter.

As of the end of December, we had 2,642 accounts or 76% of all purchasing accounts place at least 1 reorder for ZILRETTA. Notably, we saw our reorder rate increase in each quarter throughout 2019, and this occurred on a successively growing customer base. Slide 3 charts our quarterly sales from launch through the fourth quarter of 2019, which provides a very compelling view of ZILRETTA's growth in the market, especially since the introduction of the permanent J Code on January 1, 2019. We do not provide quarterly guidance, but based on routine seasonality impacts, directionally, we anticipate first quarter net sales to be roughly flat versus the fourth quarter of last year.

Moving to Slide 4. This slide and the remaining 2 slides reflect purchases of ZILRETTA by accounts, which represent physician practices, clinics and hospitals of various sizes and purchasing potential. As we look at the distribution of accounts that have purchased ZILRETTA since launch, we stayed with the same groupings that we've used in previous quarters, accounts that have purchased 1 to 10 units, purchased 11 to 50 units or purchased more than 50 units. We continue to see a significant number of accounts with purchases of 1 to 10 units. And as of December 31, 2019, roughly 1,670 accounts had made purchases in this range. While approximately 1,030 accounts had purchased 11 of 50 units. In addition, 794 accounts had purchased more than 50 units, which represents growth of 150% as compared to Q1 2019 when 313 accounts had purchased in this category.

Looking at Slide number 5. You can clearly see the distribution of purchases by accounts. Those 794 accounts that have each purchased more than 50 units are in total, responsible for approximately 143,000 units or roughly 81% of all units purchased since launch. As we have mentioned previously, accounts generally move along a utilization continuum from 1 to 10 units to 11 to 50 units and then to more than 50 units. Importantly, none of our purchasing accounts has fully incorporated ZILRETTA into their practice. And this holds true for even the highest utilizers. As a result, we believe there is tremendous opportunity for us to increase utilization across each of these groups. Before I leave this slide, I would like to point out that the total ZILRETTA purchases by accounts in the fourth quarter were approximately 37,500 units, which is lower than the 48,600 units purchased in the third quarter. We believe this quarter-over-quarter reduction was primarily the result of the broad-based rebate program that we introduced in the third quarter. There was a strong amount of enthusiasm and pent-up demand for this program, and we believe that some purchases that otherwise would have occurred in the fourth quarter instead occurred in the third quarter. To a much lesser extent, we believe that the holidays in the fourth quarter had an impact on the sequential quarter reduction as well. Nevertheless, we saw total ZILRETTA purchases by accounts increased by more than 70% in the second half of 2019 versus the first half of 2019.

Moving to Slide 6. Here, we break out ZILRETTA purchases by new and existing accounts. And in the fourth quarter, we added about 350 new purchasing accounts. While we expect to eventually see a slowing in the number of new accounts coming on board each quarter, we continue to be pleased with the progress we are making with new accounts as they typically work their way through the ZILRETTA utilization continuum.

So at this point, I will briefly walk through the fourth quarter and full year 2019 financial results, which we included in the press release issued this afternoon and in our 10-K. We reported a net loss of $149.8 million for full year 2019 as compared to a net loss of $169.7 million for full year 2018. Net sales of ZILRETTA were $23.7 million for the fourth quarter of 2019 and totaled $73 million for full-year 2019. The cost of sales for full-year 2019 was $10 million. The fourth quarter 2019 net sales reflect a gross to net reduction of 11%. A 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, and Veterans Administration, Department of Defense. As we previously mentioned, in the third quarter, we started offering rebates to eligible health care providers that are variable based on the volume of product purchased. These provider rebates contributed 4% of the fourth quarter total gross to net reduction of 11%. Research and development expenses were $69.6 million, and $53.1 million for the years ended December 31, 2019 and 2018, respectively. The increase in research and development expenses year-over-year of $16.5 million was primarily due to an increase in salary and other related costs for additional headcount and stock-based compensation expense, an increase in expenses related to portfolio expansion and other program costs, including an upfront payment to Xenon Pharmaceuticals related to FX301, and an increase in development expenses for ZILRETTA. Selling, general and administrative expenses were $129.7 million and $121.3 million for the years ended December 31, 2019, and 2018, respectively. Selling expenses were $96.3 million and $87.3 million for the years ended December 31, 2019 and 2018, respectively. The year-over-year increase in selling expenses of $9 million was primarily due to salary and other employee-related costs and external costs related to marketing and reimbursement support activities.

General and administrative expenses were $33.4 million and $34 million for the years ended December 31, 2019 and 2018, respectively, which represents a decrease of $0.6 million year-over-year. Interest expense was $17.1 million and $15.7 million for the years ended December 31, 2019 and 2018, respectively. We expect that while our operating expenses will continue to increase in the near term, primarily driven by commercial activities in support of ZILRETTA, line extension clinical trials for ZILRETTA, continued development of FX201 and FX301 and development activities associated with future additions to the pipeline. We believe we will be able to increasingly leverage our infrastructure in support of these efforts. As of December 31, 2019, we had approximately $136.7 million in cash, cash equivalents and marketable securities compared with $258.8 million as of December 31, 2018. In addition, earlier this quarter, we fully drew down $20 million from our revolving credit facility, which is secured by our accounts receivables. We believe that our current cash balance with the expected future sales [of ZILRETTA] (added by company after the call) and the ongoing prudent management of our expenses will enable us to reach profitability. However, our projections are based on certain market assumptions, which may or may not be affected by the coronavirus pandemic. As a result, we will continue to review and reassess our [assumptions] (added by company after the call)

in light of those factors. In addition, we will remain opportunistic as it relates to potential funding decisions, and we will do what we believe is in the best long-term interest of Flexion and our shareholders.

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 comes from Randall Stanicky with RBC Capital Markets.

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Daniel James Busby, RBC Capital Markets, Research Division - Senior Associate [2]

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This is Dan Busby on for Randall. A couple of questions. First, among the high-prescribing accounts, in particular, can you give us a sense of how much more room there is to grow within those practices? I think I heard you mentioned that you haven't fully penetrated any of those accounts yet. And of the physicians in those accounts who aren't using it, what's the pushback you're hearing?

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David A. Arkowitz, Flexion Therapeutics, Inc. - CFO [3]

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Yes, Dan, this is David. So as we shared in our prepared remarks and in the deck, we've got almost 800 accounts that have purchased 50 units, more than 50 units of ZILRETTA launch to date. In the vast, vast majority of those accounts, ZILRETTA has not been fully adopted, incorporated into their practices and even there's a subset within those accounts of almost 50 accounts that have purchased more than 500 of units of ZILRETTA launch to date. So there's room to run with those almost 800 accounts as well as the other accounts that are less than 50 units of ZILRETTA purchased launch to date. And in terms of the other part of your question, in terms of why have they not fully incorporated ZILRETTA at this juncture. The way this will typically work is take a practice with 5 or 6 physicians [1 or] (added by company after the call) 2 of those physicians have started to use ZILRETTA and are using ZILRETTA for their patient -- appropriate patient population. But there just hasn't been awareness and experience with ZILRETTA for the other 3 or 4 docs in the practice. So it's a process. It's a process for the docs that are treating the existing patients to talk to their colleagues or our representatives to be making those physicians that are not yet using ZILRETTA, aware of ZILRETTA, getting them comfortable with reimbursement. So that's really what is going on.

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Daniel James Busby, RBC Capital Markets, Research Division - Senior Associate [4]

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Okay, that's helpful. And I guess, as a follow-up to that, if 1 practice, if 1 or 2 physicians within a practice, have secured reimbursement? Is it typically the case that other doctors who aren't using it, but may in the future, they would have reimbursement set up already? Or is that more kind of doctor by doctor?

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David A. Arkowitz, Flexion Therapeutics, Inc. - CFO [5]

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No, it's typically at the office level, at the clinic level. But the -- it's an issue of just getting familiar and comfortable with reimbursement, experiencing reimbursement, and that takes those docs that have not achieved that to just go through the process.

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Operator [6]

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Our next question comes from Elliot Wilbur with Raymond James.

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Elliot Henry Wilbur, Raymond James & Associates, Inc., Research Division - Senior Research Analyst [7]

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A couple of questions. First, Mike, I believe you mentioned in your commentary that you expect 1Q 2020 ZILRETTA sales to be essentially flat versus 4Q '19. Just want to get maybe a little bit more color behind that? How much of that you think is attributable to high deductible plans, perhaps influencing utilization versus other factors such as seasonality or just sort of overall company conservatism kind of in light of potentially increased macro uncertainty here in the short term?

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Michael D. Clayman, Flexion Therapeutics, Inc. - Co-Founder, President, CEO & Director [8]

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Yes. So I think there are a few things, Elliot. So it's a good and important question. First, just to recognize, historically, in this space, the hyaluronic acids are typically down 10% in the first quarter, with the driver being, as you point out, deductible. That is a key driver. I think that you also have to look at an older population may be less active in the winter months. And as a result, have less need to go to their physicians. So there are a couple of reasons why there is a basis for, relatively speaking, the first quarter being softer than other quarters and why we have guided to flat in the first quarter.

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Elliot Henry Wilbur, Raymond James & Associates, Inc., Research Division - Senior Research Analyst [9]

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Okay. And then with respect to your commentary around some of the data points that emerge from the claims data that you're referring to in terms of the payer mix with Medicare representing now a higher proportion. How should we think about the relative opportunity with respect to additional growth levers such as repeat administration, bilateral administration within the context of a greater Medicare book of business versus commercial?

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Michael D. Clayman, Flexion Therapeutics, Inc. - Co-Founder, President, CEO & Director [10]

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Yes, it's a good question. We -- as you know, the Medicare population has the opportunity to benefit most directly in a sense from ZILRETTA and that physicians can use same-day without any hesitancy and proceed with confidence that they will be reimbursed. They can repeat dose without any concern about reimbursement. They can do bilateral injections, et cetera. So we like the Medicare population. We also like the commercial population. It's very straightforward with Medicare. And I would say directionally, as a modest bump up in the representation of Medicare is, wind in our sails and will improve our circumstances. As we often say, this is not a light switch, but it's directionally encouraging.

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Elliot Henry Wilbur, Raymond James & Associates, Inc., Research Division - Senior Research Analyst [11]

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Okay. And then just a couple of financial questions for David as well. With respect to SG&A trends, they've held relatively flat in the $32 million, $33 million range over the last 4 quarters, not sure kind of directionally would expect it to increase, but obviously, you've been able to get a lot more leverage out of that line than probably what we would have expected at the beginning of the year. So how should we think about progression of that number over the course of 2020?

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David A. Arkowitz, Flexion Therapeutics, Inc. - CFO [12]

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Yes, Elliot, great question. So to your point, it has been relatively flat quarter-over-quarter in 2019 the G&A component of that. So if you just look at quarterly SG&A for fourth quarter about 20 -- excuse me, about $32 million, about $8 million of it is G&A, that will stay flat in the ensuing quarters. $24 million of it is commercial. And as we've talked about before, slightly more than half of that is related to headcount and support of spend, and we are right-size from a headcount standpoint. So increases in that portion will be minimal, where we're going to see some increases over time are external marketing activities in support of increasing ZILRETTA sales. But because that's only less than half of the selling and marketing spend, you're not going to see SG&A go up by meaningful amounts quarter-over-quarter. And on top of all that, as you might imagine, we're very focused on leveraging as best we can, our SG&A infrastructure that we've built up over the past 4 quarters.

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Edited Transcript of FLXN earnings conference call or presentation 12-Mar-20 8:30pm GMT - Yahoo Finance

Why soap works – even on coronavirus – and how its discovery altered human history – Chattanooga Times Free Press

By adminMarch 16, 2020Gene Therapy Clinics

It probably began with an accident thousands of years ago. According to one legend, rain washed the fat and ash from frequent animal sacrifices into a nearby river, where they formed a lather with a remarkable ability to clean skin and clothes. Perhaps the inspiration had a vegetal origin in the frothy solutions produced by boiling or mashing certain plants. However it happened, the ancient discovery of soap altered human history. Although our ancestors could not have foreseen it, soap would ultimately become one of our most effective defenses against invisible pathogens.

People typically think of soap as gentle and soothing, but from the perspective of microorganisms, it is often extremely destructive. A drop of ordinary soap diluted in water is sufficient to rupture and kill many types of bacteria and viruses, including the new coronavirus that is currently circling the globe. The secret to soap's impressive might is its hybrid structure.

Soap is made of pin-shaped molecules, each of which has a hydrophilic head -- it readily bonds with water -- and a hydrophobic tail, which shuns water and prefers to link up with oils and fats. These molecules, when suspended in water, alternately float about as solitary units, interact with other molecules in the solution and assemble themselves into little bubbles called micelles, with heads pointing outward and tails tucked inside.

Some bacteria and viruses have lipid membranes that resemble double-layered micelles with two bands of hydrophobic tails sandwiched between two rings of hydrophilic heads. These membranes are studded with important proteins that allow viruses to infect cells and perform vital tasks that keep bacteria alive. Pathogens wrapped in lipid membranes include coronaviruses, HIV, the viruses that cause hepatitis B and C, herpes, Ebola, Zika, dengue, and numerous bacteria that attack the intestines and respiratory tract.

When you wash your hands with soap and water, you surround any microorganisms on your skin with soap molecules. The hydrophobic tails of the free-floating soap molecules attempt to evade water; in the process, they wedge themselves into the lipid envelopes of certain microbes and viruses, prying them apart.

"They act like crowbars and destabilize the whole system," said professor Pall Thordarson, acting head of chemistry at the University of New South Wales. Essential proteins spill from the ruptured membranes into the surrounding water, killing the bacteria and rendering the viruses useless.

In tandem, some soap molecules disrupt the chemical bonds that allow bacteria, viruses and grime to stick to surfaces, lifting them off the skin. Micelles can also form around particles of dirt and fragments of viruses and bacteria, suspending them in floating cages. When you rinse your hands, all the microorganisms that have been damaged, trapped and killed by soap molecules are washed away.

On the whole, hand sanitizers are not as reliable as soap. Sanitizers with at least 60% ethanol do act similarly, defeating bacteria and viruses by destabilizing their lipid membranes. But they cannot easily remove microorganisms from the skin. There are also viruses that do not depend on lipid membranes to infect cells, as well as bacteria that protect their delicate membranes with sturdy shields of protein and sugar. Examples include bacteria that can cause meningitis, pneumonia, diarrhea and skin infections, as well as the hepatitis A virus, poliovirus, rhinoviruses and adenoviruses (frequent causes of the common cold).

These more resilient microbes are generally less susceptible to the chemical onslaught of ethanol and soap. But vigorous scrubbing with soap and water can still expunge these microbes from the skin, which is partly why hand-washing is more effective than sanitizer. Alcohol-based sanitizer is a good backup when soap and water are not accessible.

In an age of robotic surgery and gene therapy, it is all the more wondrous that a bit of soap in water, an ancient and fundamentally unaltered recipe, remains one of our most valuable medical interventions. Throughout the course of a day, we pick up all sorts of viruses and microorganisms from the objects and people in the environment. When we absentmindedly touch our eyes, nose and mouth -- a habit, one study suggests, that recurs as often as every 2 1/2 minutes -- we offer potentially dangerous microbes a portal to our internal organs.

Washing with soap and water is one of the key public health practices that can significantly slow the rate of a pandemic and limit the number of infections, preventing a disastrous overburdening of hospitals and clinics. But the technique works only if everyone washes their hands frequently and thoroughly: Work up a good lather, scrub your palms and the back of your hands, interlace your fingers, rub your fingertips against your palms, and twist a soapy fist around your thumbs.

Or as the Canadian health officer Bonnie Henry said recently, "Wash your hands like you've been chopping jalapeos and you need to change your contacts." Even people who are relatively young and healthy should regularly wash their hands, especially during a pandemic, because they can spread the disease to those who are more vulnerable.

Soap is more than a personal protectant; when used properly, it becomes part of a communal safety net. At the molecular level, soap works by breaking things apart, but at the level of society, it helps hold everything together. Remember this the next time you have the impulse to bypass the sink: Other people's lives are in your hands.

The New York Times

Why Soap Works – The New York Times

By adminMarch 14, 2020Gene Therapy Clinics

In an age of robotic surgery and gene therapy, it is all the more wondrous that a bit of soap in water, an ancient and fundamentally unaltered recipe, remains one of our most valuable medical interventions. Throughout the course of a day, we pick up all sorts of viruses and microorganisms from the objects and people in the environment. When we absentmindedly touch our eyes, nose and mouth a habit, one study suggests, that recurs as often as every two and a half minutes we offer potentially dangerous microbes a portal to our internal organs.

As a foundation of everyday hygiene, hand-washing was broadly adopted relatively recently. In the 1840s Dr. Ignaz Semmelweis, a Hungarian physician, discovered that if doctors washed their hands, far fewer women died after childbirth. At the time, microbes were not widely recognized as vectors of disease, and many doctors ridiculed the notion that a lack of personal cleanliness could be responsible for their patients deaths. Ostracized by his colleagues, Dr. Semmelweis was eventually committed to an asylum, where he was severely beaten by guards and died from infected wounds.

Florence Nightingale, the English nurse and statistician, also promoted hand-washing in the mid-1800s, but it was not until the 1980s that the Centers for Disease Control and Prevention issued the worlds first nationally endorsed hand hygiene guidelines.

Or as the Canadian health officer Bonnie Henry said recently, Wash your hands like youve been chopping jalapeos and you need to change your contacts. Even people who are relatively young and healthy should regularly wash their hands, especially during a pandemic, because they can spread the disease to those who are more vulnerable.

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Why Soap Works - The New York Times

Flexion Therapeutics Inc (FLXN) Q4 2019 Earnings Call Transcript – Motley Fool

By adminMarch 14, 2020Gene Therapy Clinics

Image source: The Motley Fool.

Flexion Therapeutics Inc(NASDAQ:FLXN)Q42019 Earnings CallMar 12, 2020, 4:30 p.m. ET

Operator

Good afternoon, ladies and gentlemen. And welcome to the Flexion Therapeutics Fourth Quarter and Full Year 2019 Financial Results Conference Call. My name is Daniel and I will be your coordinator for today. At this time all participants are in listen-only mode. We will be facilitating a question-and-answer session at the end of today's call. [Operator Instructions]

I will now turn the call over to the company.

Scott Young -- Vice President, Corporate Communications & Investor Relations

Thank you, Dan. Good afternoon. This is Scott Young, Vice President for Corporate Communications and Investor Relations. Before we begin, I would call your attention to the metric slides that we will discuss in today's presentations. Those slides can be viewed directly via the webcast, in the 8-K we issued this afternoon or under the Investors tab on flexiontherapeutics.com. In addition, our Q4 earnings press release and an archive of this conference call can also be found there.

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

On today's call we will be making forward-looking statements 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-K for the year ended December 31, 2019 which was filed with the SEC 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.

Mike Clayman -- Chief Executive Officer & Co-Founder

Thanks, Scott, and thank you all for joining. Today, I'll recap our commercial progress in 2019, review our life cycle management activities, provide an update on our pipeline and discuss our priorities for this year. After that, I'll turn it over to David for a deeper review of our commercial metrics and a summary of our financial performance and then we will open the line and take questions.

As you will see in the commercial metrics that David will review, by the end of 2019, our customers had purchased 175,000 units of ZILRETTA. While our data are limited to the account level, that number provides a reasonable surrogate for the number of patients who have been treated with ZILRETTA since it was introduced to the market in late 2017. And we know that many of those patients have received unprecedented pain relief from ZILRETTA. I say it is based on real world feedback from the countless conversation I've had with grateful ZILRETTA patients and their physicians, feedback that is wholly consistent with our compelling clinical trial data.

In fact, last year, we became aware of several professional athletes who received ZILRETTA, namely Rod Woodson, the NFL Hall of Fame Defensive back, who was named as one of the top 100 players of all time. Michael Eruzione, the captain of 1980 Gold medal winning US Olympic Hockey team and the player who scored the game winning goal against the Russians during the Miracle On Ice; and Chris Dickerson, a former outfielder who played from the New York Yankees and other major league baseball teams. From snow blowing a driveway to playing with their children and grandchildren, to jogging for the first time in years, they all have profoundly moving stories about how ZILRETTA has helped each of them manage their knee OA pain and thereby improve their ability to participate in regular everyday activities.

As part of our ongoing physician marketing initiatives, we brought all three athletes together at a major orthopedic conference last December, where they and their treating physicians, spoke to a standing room crowd of some of the country's leading sports medicine experts. They share their stories of how ZILRETTA has helped them, and the best words I can use to describe that session are inspiring and humbling. While all are elite athletes, their ZILRETTA stories echo the scores of emails and letters I've received from patients who are so grateful to reengage in things they love doing with less pain. From gardening, playing a round of golf, walking on the beach, hiking, each of these stories serves as a reminder to all of us that while we have impacted more than 175,000 patients, there are 15 million people who see their doctor every year for OA knee pain and 5 million of them receive an intra-articular injection. The opportunity for ZILRETTA inflection is truly massive, which brings me back to our performance in 2019 and our goals for 2020.

Throughout the course of 2019, we saw existing practices increase their use of ZILRETTA and more than 1,600 new accounts start using the product. Today, with the benefit of two years in the market, we have actual claims data that shows some slight changes in the payer mix versus our assumptions prior to launch. We now see that the actual payer mix is skewed slightly more toward Medicare, which accounts for roughly 55% of the market versus previous estimates of 50%. 40% is still commercial, but Medicaid, VA and 340B plans are making up roughly 5% of the mix. We view this modest increase in Medicare patients to be incrementally positive as Medicare patients can be injected the same-day they visit without any need for prior authorization.

In addition, it is important to point out that commercial coverage for ZILRETTA remains excellent. As we discussed in December, the recent approval of our sNDA result in a significantly improved label, which most importantly, removed the onerous not intended for repeat administration wording in the limitation of use statement. Within days of the approval, we developed materials to help our MBMs communicate the label update and we have anecdotal feedback from the field that tells us that the changes have been very well received. While we clearly view the label update as a tailwind, we have always said that we continue to believe that the meaningful impact will be seen over the following quarters and years.

From a commercial perspective, our progress in 2019 strengthens our belief that ZILRETTA can become the new standard of care for the intra-articular treatment for OA knee pain in the years ahead. While under normal circumstances, these factors would give us confidence that our 2020 ZILRETTA net sales guidance of $120 million to $135 million is both credible and achievable.

Like all businesses, we are acutely attuned to the potential impacts of the coronavirus global pandemic, and we are monitoring this dynamic situation very closely. To date, we have not seen any material impacts on ZILRETTA sales, our ability to access customers or to initiate our clinical trials. However, it is impossible to predict how the outbreak could evolve in the months ahead or what impacts more aggressive social distancing or other containment efforts might have on patients or practices. Regarding our supply chain, we believe we are in a very strong position. We do not source any of our key materials from China and we presently have approximately 10 months worth of finished product inventory in our warehouses in the US. Furthermore, we have an additional 12 months of API, triamcinolone acetonide at our manufacturing facility in the UK. Again, it is impossible to predict the long term impacts of the outbreak, but we feel very good about our ability to provide ZILRETTA to patients over the quarters ahead.

Shifting to our clinical development activities, our Phase 2 trial to investigate the safety and efficacy of ZILRETTA in shoulder OA and adhesive capsulitis, also known as frozen shoulder syndrome continues to advance and we anticipate data from that trial in 2020. As we've previously discussed, these two conditions combined account for roughly 800,000 injections and they present an opportunity for us to expand the use of ZILRETTA with a subset of orthopedics who primarily focus on sports medicine and commonly treat these conditions with steroid injections.

With respect to our pipeline, we've also been making progress with our two drug candidates FX201 and FX301. FX201 is our gene therapy, which holds the potential to provide OA pain relief for at least a year, improve function and potentially modify disease. As we announced last year, the IND for FX201 was accepted by the FDA and we recently treated the first two patients in our Phase 1 dose ranging study. We anticipate treating approximately 15 to 24 patients, who will be followed for 104 weeks with initial readout in 2021.

Now we'll move to FX301. Our NaV1.7 inhibitor formulated within a thermosensitive hydrogel for administration as a peripheral nerve block for control of post-operative pain. We held our pre-IND meeting with FDA and we remain on track to initiate our first FX301 clinical trial in 2021. Unlike typical local anesthetics, we believe the selective pharmacology of FX301 has the potential to deliver at least three days to five days of effective pain relief, while preserving motor function, which could enable ambulation, rapid discharge from the hospital and early rehabilitation following musculoskeletal surgery.

Finally, regarding our search for a Chief Commercial Officer. We were very excited to announce the appointment of Melissa Layman earlier this week, and I'm delighted that she is able to join us for today's call. As we've said repeatedly, in our search for a CCO, we were looking for someone who have had success leading an entire commercial enterprise, who had deep experience and expertise in each of the key commercial functional domains, who had a track record as a very strong leader and who would positively contribute to our already strong culture. Because Melissa fulfills all of these criteria and is simply a terrific person to interact with, we could not be more pleased to have her take the helm of our commercial organization.

Before David summarizes our fourth quarter financials and walks through the commercial metrics, I'd like to give Melissa the opportunity to make a few remarks. Melissa?

Melissa Layman -- Chief Commercial Officer

The commitment to patients and passion for winning has been universal and that was one of the many reasons I wanted to join. Put simply, I can't begin to convey how excited I am about the opportunity that's ahead of us. The strong foundation that's been laid by Mike, David, Steve Meyers, and the rest of the commercial leadership team is truly impressive and I'm looking forward to working together to grow ZILRETTA's market share and help make it the leading IA treatment in this space. Over the next few months, I'll be working intensively with our commercial organization, and I look forward to representing our commercial effort on future calls.

At this point, I'll turn it over to David.

David Arkowitz -- Chief Financial Officer

Thank you, Melissa. I'll start by walking through our commercial metrics, which as Scott mentioned can be found on our website and in the 8-K we issued today.

If you look at Slide 2, you can see that we recorded ZILRETTA net sales of $23.7 million in the fourth quarter of 2019, bringing full year 2019 net sales to $73 million. As Mike mentioned, year-over-year growth topped 220%. As in previous quarters, we expanded our list of target accounts in the fourth quarter to 4,972 and by December 31, 2019 we had called on almost all of them. At the end of the fourth quarter, 3,488 accounts had purchased ZILRETTA, which is an increase of nearly 360 purchasing accounts compared to the end of the third quarter. As of the end of December, we had 2,642 accounts or 76% of all purchasing accounts placed at least one reorder for ZILRETTA. Notably, we saw our reorder rate increase in each quarter throughout 2019 and this occurred on a successively growing customer base.

Slide 3 charts our quarterly sales from launch through the fourth quarter of 2019, which provides a very compelling view of ZILRETTA's growth in the market, especially since the introduction of the permanent J Code on January 1, 2019. We do not provide quarterly guidance, but based on routine seasonality impacts, directionally we anticipate first quarter net sales to be roughly flat versus the fourth quarter of last year.

Moving to Slide 4. This slide and the remaining two slides reflect purchases of ZILRETTA by accounts, which represent physician practices, clinics and hospitals of various sizes and purchasing potential. As we look at the distribution of accounts that have purchased the ZILRETTA since launch, we stayed with the same groupings that we've used in previous quarters, accounts that have purchased one units to 10 units, purchased 11 units to 50 units or purchased more than 50 units. We continue to see a significant number of accounts with purchases of one unit to 10 units. And as of December 31, 2019, roughly 1,670 accounts had made purchases in this range, while approximately 1,030 accounts had purchased 11 units to 50 units. In addition, 794 accounts had purchased more than 50 units, which represents growth of 150% as compared to Q1 2019 when 313 accounts had purchased in this category.

Before I leave this slide, I would like to point out that the total ZILRETTA purchases by accounts in the fourth quarter were approximately 37,500 units, which is lower than the 48,600 units purchased in the third quarter. We believe this quarter-over-quarter reduction was primarily the result of the broad-based rebate program that we introduced in the third quarter. There was a strong amount of enthusiasm and pent-up demand for this program and we believe that some purchases that otherwise would have occurred in the fourth quarter instead occurred in the third quarter. To a much lesser extent, we believe that the holidays in the fourth quarter had an impact on the sequential quarter reduction as well. Nevertheless, we saw total ZILRETTA purchases by accounts increased by more than 70% in the second half of 2019 versus the first half of 2019.

Moving to Slide 6. Here we break out ZILRETTA purchases by new and existing accounts and in the fourth quarter, we added about 350 new purchasing accounts. While we expect to eventually see a slowing in the number of new accounts coming on board each quarter, we continue to be pleased with the progress we are making with new accounts as they typically work their way through the ZILRETTA utilization continuum.

Research and development expenses were $69.6 million and $53.1 million for the years ended December 31, 2019 and 2018, respectively. The increase in research and development expenses year-over-year of $16.5 million was primarily due to an increase in salary and other related costs for additional headcount and stock based compensation expense, an increase in expenses related to portfolio expansion and other program costs, including an upfront payment to Xenon Pharmaceuticals related to FX301 and an increase in development expenses for ZILRETTA.

Selling, general and administrative expenses were $129.7 million and $121.3 million for the year ended December 31, 2019 and 2018, respectively. Selling expenses were $96.3 million and $87.3 million for the years ended December 31, 2019 and 2018, respectively. The year-over-year increase in selling expenses of $9 million was primarily due to salary and other employee related costs and external costs related to marketing and reimbursement support activities.

General and administrative expenses were $33.4 million and $34 million for the years ended December 31, 2019 and 2018, respectively, which represents a decrease of $0.6 million year-over-year.

Interest expense was $17.1 million and $15.7 million for the years ended December 31, 2019 and 2018, respectively. We expect that while our operating expenses will continue to increase in the near term, primarily driven by commercial activities in support of ZILRETTA, line extension clinical trials for ZILRETTA, continued development of FX201 and FX301 and development activities associated with future additions to the pipeline. We believe we will be able to increasingly leverage our infrastructure in support of these efforts.

As of December 31, 2019, and we had approximately $136.7 million in cash, cash equivalents and marketable securities compared with $258.8 million as of December 31, 2018. In addition, earlier this quarter, we fully drew down $20 million from our revolving credit facility, which is secured by our accounts receivables. We believe that our current cash balance with the expected future sales of ZILRETTA and the ongoing prudent management of our expenses will enable us to reach profitability. However, our projections are based on certain market assumptions, which may or may not be affected by the coronavirus pandemic. As a result, we will continue to review and reassess our assumptions in light of those factors. In addition, we will remain opportunistic as it relates to potential funding decisions, and we will do what we believe is in the best long-term interest of Flexion and our shareholders.

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

Operator

Thank you. That concludes our prepared remarks. We'll now open the call for questions. [Operator Instructions] Our first question comes from Randall Stanicky with RBC Capital Markets. Your line is now open.

Dan Busby -- RBC Capital Markets -- Analyst

Good evening, this is Dan Busby on for Randall. A couple of questions. First, among the high prescribing accounts in particular, can you give us a sense of how much more room there is to grow within those practices? I think I heard you mentioned that you haven't fully penetrated any of those accounts yet. And of the physicians in those accounts who aren't using it, what's the pushback you're hearing?

David Arkowitz -- Chief Financial Officer

Yeah Dan, this is David. So as we shared in our prepared remarks and in the deck, we've got almost 800 accounts that have purchased 50 units, more than 50 units of ZILRETTA launch to date. The vast, vast majority of those accounts, ZILRETTA has not been fully adopted, incorporated into their practices and even there is a subset even within those accounts of almost 50 accounts that have purchased more than 500 of units of ZILRETTA launch to date. So there's room to run with those almost 800 accounts as well as the other accounts that are less than 50 units of ZILRETTA purchased launch to date.

And in terms of the other part of your question, in terms of why have they not fully incorporated ZILRETTA at this juncture. The way this will typically work is take a practice with five physicians or six physicians one or two of those physicians have started to use ZILRETTA and are using ZILRETTA for their patient -- appropriate patient population but there just hasn't been awareness and experience with ZILRETTA for the other three or four docs in the practice. So it's a process, it's a process for the docs that are treating the existing patients to talk to their colleagues, for our representatives to be making those physicians that are not yet using ZILRETTA aware of ZILRETTA, getting them comfortable with reimbursement. So that's really what is going on.

Dan Busby -- RBC Capital Markets -- Analyst

Okay, that's helpful. And I guess as a follow-up to that, if one practice -- if one or two physicians within a practice have secured reimbursement, is it typically the case that other doctors who aren't using it, but may in the future, they would have reimbursement set up already or is that more kind of doctor by doctor?

Mike Clayman -- Chief Executive Officer & Co-Founder

No, it's typically at the office level, at the clinical level. But the -- it's an issue of just getting familiar and comfortable with reimbursement, experiencing reimbursement and that takes those docs that have not achieved that to just go through the process.

Dan Busby -- RBC Capital Markets -- Analyst

Got it, understood. Thank you.

David Arkowitz -- Chief Financial Officer

Thanks, Dan. Sure.

Operator

Thank you. Our next question comes from Elliot Wilbur with Raymond James. Your line is now open.

Elliot Wilbur -- Raymond James and Associates -- Analyst

Thanks, good afternoon. Couple of questions. First, Mike, I believe you mentioned in your commentary that you expect 1Q 2020 ZILRETTA sales to be essentially flat versus 4Q '19. Just want to get maybe a little bit more color behind that? How much of that you think is attributable to high deductible plans, perhaps influencing utilization versus other factors such as seasonality or just sort of overall company conservatism kind of in light of potentially increased macro uncertainty here in the short term?

Mike Clayman -- Chief Executive Officer & Co-Founder

Yeah, so I think there are a few things, Elliot. So it's a good and important question. First just to recognize, historically, in this space, the hyaluronic acids are typically down 10% in the first quarter, with the driver being as you point out, deductible. That is a key driver. I think that you also have to look at an older population may be less active in the winter months. And as a result, have less need to go to their physicians. So there are a couple of reasons why there is a basis for relatively speaking the first quarter being [Indecipherable] other quarters and why we have guided to flat in the first quarter.

Elliot Wilbur -- Raymond James and Associates -- Analyst

Mike Clayman -- Chief Executive Officer & Co-Founder

Yeah, it's a good question. We -- as you know the Medicare population has the opportunity to benefit most directly in a sense from ZILRETTA and that physicians can use same day without any hesitancy and proceed with confidence that they will be reimbursed. They can repeat dose without any concern about reimbursement. They can do bilateral injections etc. So we like the Medicare population, we also like the commercial population. It's very straightforward with Medicare. And I would say directionally as a modest bump up in the representation of Medicare is wind in our sales and will improve our circumstances. As we often say this is not a light switch, but it's directionally encouraging.

Elliot Wilbur -- Raymond James and Associates -- Analyst

Okay. And then just a couple of financial questions for David as well. With respect to SG&A trends, they've held relatively flat in the $32 million, $33 million range over the last four quarters, not sure kind of directionally would expect it to increase, but obviously, you've been able to get a lot more leverage out of that line than probably what we would have expected at the beginning of the year. So how should we think about progression of that number over the course of 2020?

David Arkowitz -- Chief Financial Officer

Yeah, Elliot, great question. So to your point it has been relatively flat quarter-over-quarter in 2019, the G&A component of that. So if you just look at quarterly SG&A for fourth quarter about 20 -- about excuse me, about $32 million, about $8 million of it is G&A, that will stay flat in the ensuing quarters. $24 million of it's is commercial. And as we've talked about before, slightly more than half of that is related to headcount and support of spend and we are right sized from a headcount standpoint. So increases in that portion will be minimal, where we're going to see some increases over time are external marketing activities in support of increasing ZILRETTA sales. But because that's only less than half of the selling and marketing spend, you're not going to see SG&A go up by meaningful amounts quarter-over-quarter. And on top of all that, as you might imagine, we're very focused on leveraging as best we can, our SG&A infrastructure that we've built up over the past four quarters.

Elliot Wilbur -- Raymond James and Associates -- Analyst

Fair enough, one last question for you, David, just doing some simple back of the envelope math here, yield is a much higher net price per unit in the fourth quarter versus what we've seen during the course of the year. Wondering how much of that may be attributable to simply payer mix? Or is there some sort of Medicare true-up or other reserve adjustments that may have led to that?

David Arkowitz -- Chief Financial Officer

Yeah, so Elliot, the gross to net reductions in the third quarter that was 14%. That would have generated a net price per unit of about $490. In the fourth quarter, our gross to net, as I talked about earlier, was 11% and that generates net price per unit of about $507 per unit. And really the difference between Q4 and Q3, why the gross to net reduction is a bit less, relates to the provider rebates those were obviously less in the fourth quarter than they were in the third quarter on a per unit basis.

Elliot Wilbur -- Raymond James and Associates -- Analyst

All right, thank you.

David Arkowitz -- Chief Financial Officer

Thank you.

Operator

Thank you. Our next question comes from Gary Nachman with BMO Capital Markets. Your line is now open.

Gary Nachman -- BMO Capital Markets -- Analyst

Hi, good afternoon. Mike, first, I just want to confirm, if not for all the coronavirus uncertainty, would you be reiterating ZILRETTA guidance of $120 million to $135 million that you provided in January, based on everything that you had been seeing thus far? And David, how much flexibility do you have with expenses? If revenue ends up trailing your guidance, what sort of levers do you have -- in both SG&A and R&D?

Mike Clayman -- Chief Executive Officer & Co-Founder

Yes, good questions Gary, let me say that. We do reiterate guidance in the script. We do indicate our confidence in achieving full year revenue $120 million to $135 million. What we do is qualify that by the unknowns associated with COVID-19 and inability to predict with any sense of reality, what the situation will be in the coming weeks and months. So I think it would be imprudent for us to guide to $120 million to $135 million without that qualifier, because there are just too many unknowns. Bottom line is take COVID off the table and our confidence in the guidance continues at the highest level.

David Arkowitz -- Chief Financial Officer

And Gary, this is David. So operating expenses on a quarterly basis around $50 million. We do have a certain amount of operational flexibility to dial expenses up and dial them down. Obviously, we want to ensure that we're appropriately resourcing ZILRETTA from a commercialization standpoint from a line extension standpoint and then we're appropriately resourcing FX201 and 301 in their respective development. But with all that said, we are sitting here in Burlington, Mass in rented space that's not all that expensive and we don't have a whole lot of fixed cost. So we have a certain amount of flexibility, but we're obviously going to do what makes the most sense to the business.

Gary Nachman -- BMO Capital Markets -- Analyst

Okay, great. And then Mike, just a little bit more on what sort of impact have you been seeing or hearing so far in the label update. How have you changed your promotional materials and messaging since that label update? I know that it's pretty early, but -- and it takes some time for you to really see the impact there. But any color on the anecdotal feedback that you've been getting on that? And then just -- yes, go ahead, I'll have a follow up after.

Mike Clayman -- Chief Executive Officer & Co-Founder

Okay. I'd just say, it's a good question. We're very happy with our ability and the ability of our sales reps or MBMs to share the reprint and reprint cover that includes information about the trial, data from the trial and to be able to do that proactively.

Operator

Ladies and gentlemen, please standby your conference will resume momentarily. Once again, thank you for your patience and please continue to standby.

Mike Clayman -- Chief Executive Officer & Co-Founder

[Speech Overlap] Apologies, we had a technical snafu. So what we're hearing anecdotally is very positive, but it's too early to quantify the effect. But directionally, it is -- we're absolutely confident it will be positive.

Gary Nachman -- BMO Capital Markets -- Analyst

Okay. And then the last question is just what sort of initiatives might you have in place to try and shift an acceleration with the targeted physicians to the higher utilization buckets.

And Melissa, welcome to you, maybe it's a chance for you to comment just a little bit, based on your prior experience, where do you see the greatest opportunity to accelerate ZILRETTA? And then at what point do you think the sales force might be ready to take on some new products in the bag and then leverage that infrastructure a little bit better? Thanks. Hello?

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Flexion Therapeutics Inc (FLXN) Q4 2019 Earnings Call Transcript - Motley Fool

Scarborough brother and sister, aged 11 and 12, with the genetic cholesterol condition FH are helped by new clinic – The Scarborough News

By adminMarch 14, 2020Gene Therapy Clinics

The clinic helps children at risk of developing heart disease in the future due to high cholesterol.

They can now attend a ground-breaking clinic run by the familial hypercholesterolemia (FH) service at York Teaching Hospital NHS Foundation Trust.

FH is an inherited condition which can lead to extremely high cholesterol levels and is passed down through families in the genes.

The FH service, led by Dr Chandrajay, Consultant in Chemical Pathology and Metabolic Medicine, and Claire Tuson, Familial Hypercholesterolaemia Specialist Nurse, has recently extended their service to include children and adolescents.

Claire explained: Research has shown that children with FH start to develop a build-up of fatty plaque in their arteries before the age of 10. Once diagnosed, FH is easy to treat so it makes sense to work with families as soon as possible.

Last year, with the support of Consultant Paediatrician Dr Dominic Smith, we extended gene testing to all children aged 10 years old and over, who have a parent affected with FH. Testing children for FH could prevent a potentially fatal heart attack or stroke.

The first six children from York and Scarborough that were identified with FH have recently attended our new Yorkshire and Humber joint paediatric clinic for children and their families, which launched at the end of January.

FH is estimated to affect 1 in 250 people in the UK, including over 56,000 children.

It is an inherited disorder of cholesterol and lipid metabolism, caused by an alteration in a single gene where people have higher levels of bad cholesterol levels from birth. If left undetected and untreated FH can lead to the early development of heart and circulatory problems.

Kiera Pickering, aged 12, and her brother Connor, aged 11, from Scarborough, were two of the first children to attend the clinic.

Claire added: Its a real breakthrough to be able to identify and treat children with FH so early. Alongside dietary and lifestyle advice to maintain a healthy body weight, children can be considered for statin therapy from as young as 10 years old.

"Statin treatment can not only prevent, but potentially reverse, the build-up of cholesterol and allow children and young people to live a perfectly healthy life.

Despite the availability of genetic testing, more than 85 percent of people with FH in the UK are undiagnosed.

The British Heart Foundation estimates that currently only around 600 children in the UK have been diagnosed with FH, meaning that thousands more are not on treatment and remain unaware of their future risk of heart disease.

For more information about the FH clinics contact claire.tuson@york.nhs.uk

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Scarborough brother and sister, aged 11 and 12, with the genetic cholesterol condition FH are helped by new clinic - The Scarborough News

Spinal Muscular Atrophy Treatment Market to Exhibit a Spectacular CAGR of 28.9%; Growing Initiatives by Government Authorities for Pre-Diagnosis to…

By adminMarch 7, 2020Gene Therapy Clinics

Pune, March 03, 2020 (GLOBE NEWSWIRE) -- The global Spinal Muscular Atrophy Treatment Market size is expected to reach USD 14.49 billion by 2026, exhibiting a CAGR of 28.9% during the forecast period. The rising prevalence of rare diseases around the world will fuel demand for SMA treatment in the forthcoming years, which in turn will aid the growth of the market. As per the National Policy for the treatment of rare diseases, globally, around 6000 to 8000 rare diseases are estimated to exist with new rare diseases reported on a regular basis. Furthermore, 80% of all the rare diseases are genetically originated and therefore impact children inexplicably. The survey also revealed that 50% of new cases are in children and are responsible for 35% of deaths before the age of 1 year, 10% between the ages of 1 and 5 years and 12% between 5 and 15 years. Nonetheless, the growing initiatives by government authorities for pre-diagnosis will impact the Spinal Muscular Atrophy Treatment Market share positively during the forecast period, predicts our lead analysts at Fortune Business Insights.

For more information in the analysis of this report, visit: https://www.fortunebusinessinsights.com/industry-reports/spinal-muscular-atrophy-treatment-market-100576

According to the report, published by Fortune Business Insights, titled Spinal Muscular Atrophy Treatment Market Size, Share and Global Trend By Product (Nusinersen and Onasemnogen Abeparvovec), By Disease Type (Type 1 SMA, Type 2 SMA and Others), By Distribution Channel (Hospital Pharmacies, Retail Pharmacies and Others), and Geography Forecast till 2026 the market size stood at USD 1.72 billion. The SMA Treatment Market report executes a PESTEL study and SWOT analysis to reveal the stability, restrictions, openings, and threats in the smart building market. Combined with the market analysis capabilities and knowledge integration with the relevant findings, the report has foretold the robust future growth of the SMA treatment market, and all articulated with geographical and merchandise segments. Moreover, it also shows different procedures and strategies, benefactors and dealers working in the market, explores components convincing market development, generation patterns, and following systems. Additionally, the figures and topics covered in this report are both all-inclusive and reliable for the readers.

Market Driver:

R&D Initiatives by Key Players to Spur Sales Opportunities

The surge in research and development activities for the improvement of therapies and treatment options by key players will aid the Spinal Muscular Atrophy Treatment Market growth during the forecast period. Various drug pipeline for advanced stages of clinical trials by major pharmaceutical companies will augment the healthy growth of the market. For instance, Genentech/Roches pipeline candidate of Risdiplam, which recently received a priority review from the FDA and is expected to receive a decision on approval from the FDA by May 2020. Furthermore, the growing initiatives for pre-diagnosis and positive reimbursement policies will boost the Spinal Muscular Atrophy Treatment Market trends in the foreseeable future. Moreover, the growing awareness regarding pivotal treatment options will create new opportunities for the market.

Request a Sample Copy of the Research Report:https://www.fortunebusinessinsights.com/enquiry/request-sample-pdf/spinal-muscular-atrophy-treatment-market-100576

Market Restraint:

High Cost of Products to Impede Market Expansion

The cost-intensive products and high prices associated with the rare disease therapies will subsequently obstruct the growth of the market. For instance, spinraza is expected to cost US$ 750,000 for the first year and will be repriced at US$ 375,000 after that. Apart from that, Novartis rare gene therapy, Zolgensma will come at a price of US$ 2.1 million for a one-time treatment. The expensive cost of therapies will restrict the adoption of treatment for many patients, which in turn will act as a restraint for the Spinal Muscular Atrophy Treatment Market revenue.

Regional Insight:

Presence of Major Players to Influence Growth in North America

The market in North America stood at USD 854 million in 2018 and is likely to remain dominant during the forecast period. The growth in the region is attributed to the presence of prominent players in the region. The growing awareness regarding the prevalence of rare disease and pre-treatment initiatives will bolster accelerate the Spinal Muscular Atrophy Treatment Market growth in North America.

List of the Major Players Operating in the Global SMA Treatment Market Include:

Have Any Query? Ask Our Experts:https://www.fortunebusinessinsights.com/enquiry/speak-to-analyst/spinal-muscular-atrophy-treatment-market-100576

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Study maps landmarks of peripheral artery disease to guide treatment development – University of Illinois News

By adminMarch 5, 2020Gene Therapy Clinics

CHAMPAIGN, Ill. Novel biomedical advances that show promise in the lab often fall short in clinical trials. For researchers studying peripheral artery disease, this is made more difficult by a lack of standardized metrics for what recovery looks like. A new study from University of Illinois at Urbana-Champaign researchers identifies major landmarks of PAD recovery, creating signposts for researchers seeking to understand the disease and develop treatments.

Having these landmarks could aid in more optimal approaches to treatment, identifying what kind of treatment could work best for an individual patient and when it would be most effective, said Illinois bioengineering professor Wawrzyniec L. Dobrucki, who led the study. He also is affiliated with the Carle Illinois College of Medicine.

PAD is a narrowing of the arteries in the limbs, most commonly the legs, so they dont receive enough blood flow. It often isnt diagnosed until walking becomes painful, when the disease is already fairly advanced. Diabetes, obesity, smoking and age increase the risk for PAD and can mask the symptoms, making PAD difficult to diagnose. Once diagnosed, there is no standard treatment, and doctors may struggle to find the right approach for a patient or to tell whether a patient is improving, Dobrucki said.

The researchers used multiple imaging methods to create a holistic picture of the changes in muscle tissue, blood vessels and gene expression through four stages of recovery after mice had the arteries in their legs surgically narrowed to mimic the narrowing found in PAD patients. They published their results in the journal Theranostics.

There are a lot of people who study PAD, so there are all these potential new therapies, but we dont see them in the clinics, said postdoctoral researcher Jamila Hedhli, the first author of the paper. So the main goal of this paper is utilizing these landmarks to standardize our practice as researchers. How can we see if the benefit of certain therapies is really comparable if we are not measuring the same thing?

The cross-disiplinary collaboration identified landmarks over four stages of disease recovery. Pictured, from left: senior research scientist Iwona Dobrucka, professor Jefferson Chan, postdoctoral researcher Jamila Hedhli, graduate student Hailey Knox, professor Wawrzyniec Dobrucki, professor Michael Insana, adjunct John Cole.

Photo by Fred Zwicky

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Dobruckis group collaborated with bioengineering professor Michael Insana, chemistry professor Jefferson Chan and senior research scientist Iwona Dobrucka, the director of the Molecular Imaging Laboratory in the Beckman Institute for Advanced Science and Technology, to monitor the mice with a suite of imaging technologies that could be found in hospitals or clinics, including ultrasound, laser speckle contrast, photoacoustics, PET and more. Each method documented a different aspect of the mouses response to the artery narrowing anatomy, metabolism, muscle function, the formation of new blood vessels, oxygen perfusion and genetic activity.

By serially imaging the mice over time, the researchers identified key features and events over four phases of recovery.

Each imaging method gives us a different aspect of the recovery of PAD that the other tools will not. So instead of looking at only one thing, now were looking at a whole spectrum of the recovery, Hedhli said. By looking at these landmarks, were allowing scientists to use them as a tool to say At this point, I should see this happening, and if we add this kind of therapy, there should be an enhancement in recovery.

Though mice are an imperfect model for human PAD, each of the imaging platforms the researchers used can translate to human PAD patients, as well as to other diseases, Dobrucki said. Next, the researchers plan to map the landmarks of PAD in larger animals often used in preclinical studies, such as pigs, and ultimately in human patients.

We are very interested in improving diagnosis and treatment, Hedhli said. Many people are working to develop early diagnosis and treatment options for patients. Having standard landmarks for researchers to refer to can facilitate all of these findings, move them forward to clinic and, we hope, result in successful clinical trials.

The National Institutes of Health, the American Heart Association, and the Ministry of Science and Higher Education of Poland supported this work. Chan, Dobrucki, Hedhli and Insana also are affiliated with the Beckman Institute. Hedhli was supported by a Beckman-Brown Postdoctoral Fellowship.

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Study maps landmarks of peripheral artery disease to guide treatment development - University of Illinois News

Misophonia: A Neurologic, Psychologic, and Audiologic… : The Hearing Journal – LWW Journals

By adminMarch 5, 2020Gene Therapy Clinics

iStock/nicoletaionescu, misophonia, pain, health.

Resources on Misphonia.

Misophonia1 is a newly described condition that is still not thoroughly understood. To her credit, Marsha Johnson, AuD, was the first to informally discuss sensitivity to specific sounds in online support groups for patients with hyperacusis, and documented about 500 cases of similar sound sensitivity problems in the late 1990s (personal communication, Dec. 3, 2019). She designed the term selective sound sensitivity syndrome (S4), which is still used today to describe misophonia. Later, Margaret Jasterboff, PhD, and Pawel Jastreboff, PhD, coined the term misophonia (which conservatively means dislike of sound),1,2 and other scientists such as Tyler, et al., refer to essentially the same condition as annoyance hyperacusis.3

Misophonia is considered sound intolerance and oversensitivity to certain sounds, which can result in distraction and annoyance that may limit a person's ability to concentrate, think, and learn.4 Some of the trigger sounds include gum popping, lip-smacking, food chewing or crunching, throat clearing, nose sniffing, breathing, tapping, and clicking.3, 5-8 Misophonia has yet to be classified in the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-V); and unfortunately, the scientific community has not reached a consensus to categorize this condition as an auditory, psychiatric/psychologic, or neurologic ailment. This lack of recognition not only prevents health care providers from officially classifying the disorder but also hinders affected individuals from seeking help.

Misophonia can be associated with high levels of anxiety9 and comorbid factors, including post-traumatic stress disorder (PTSD), obsessive-compulsive disorder (OCD), and anorexia.10,11 Autonomous sensory meridian response (ASMR), usually triggered by auditory and visual stimuli, has been associated with heightened levels of misophonia. ASMR is described as a relaxing tingling sensation that starts from the head and neck and spreads through the body.12 Although sound sensitivity and hyperacusis have been investigated in autism spectrum disorder (ASD),13,14 misophonia has not been explored in children and adults with ASD. Misophonia can also cause academic difficulties. Connie Porcaro, PhD, a university professor, was concerned about misophonia awareness among her peers in the academic community. In 2019, she designed a study to see how much the university instructors knew about misophonia and what they would do to accommodate students with this condition in their classrooms. Of the 686 survey participants, only 18.4 percent self-reported having knowledge of misophonia. Instructors who knew about this condition indicated that they would be willing to provide classroom accommodations for those who suffer from misophonia.4

The neural correlates of misophonia have been explored, with areas of interest that include the non-classical auditory pathway, limbic system, and auditory cortices.7, 15-18 Kumar and colleagues employed fMRI in individuals with misophonia while they were exposed to unpleasant trigger sounds.16 The researchers found increased neuronal activities in an area referred to as the anterior insular cortex (AIC). Although the findings of this study have been questioned by some researchers,19 it still is considered as one of the strongest pieces of evidence for a neurological origin of misophonia. It is believed that AIC, in general, is a major player in emotional awareness,20 and one can simply expect the increased responses in this area in individuals with misophonia when they are exposed to trigger sounds. Schrder and colleagues provided additional evidence for a neurobiological basis for misophonia. They studied the response of the N1 component of late auditory evoked potentials to oddball stimuli in a group of subjects with misophonia. Their findings indicated a reduced amplitude in N1 to oddball stimuli. They concluded that a neurobiological deficit in those with misophonia could potentially impair auditory processing of incoming stimuli.18 Another fMRI study has shown some correlations between misophonia and OCD. fMRI data have indicated that those with misophonia have perfectionist and compulsive behaviors.21 These findings may support the clinical observation of annoyance at chewing sounds in those with misophonia.

Additionally, genetics may play a role in misophonia. Although no strong research supports a genetic basis of misophonia, Sanchez and Silva from Brazil have reported a family with 15 members with misophonia and a possible autosomal dominant inheritance.22 Anecdotally, some of our patients have reported a mutation in chromosome 5 when they checked their genetic makeup using commercially available kits. Interestingly, a gene by the name of TENM2, which is involved in brain development, has a cytogenic location at 5q34 (in chromosome 5, longarm region 3 band 4).23

No standard questionnaire has been developed for misophonia. During the evaluation, patients are usually asked to complete a set of surveys, such as the Misophonia Questionnaire,24 which has three subscales: the Misophonia Symptom Scale, Misophonia Emotions and Behaviors Scale, and Misophonia Severity Scale. Another useful tool is the Amsterdam Misophonia Scale (A-MISO-S),25 which is widely used in many clinics. There are no universally accepted protocols for misophonia evaluation. Dozier, et al., have proposed a set of diagnostic criteria for misophonia that employs a variety of misophonic stimuli, including both auditory and visual modalities.26 Some clinics may measure loudness discomfort levels, particularly if misophonia is associated with hyperacusis. Aazh and colleagues described the loudness level measures in a group of children and adolescents with tinnitus and hyperacusis, and noticed that the uncomfortable loudness levels (ULLs) were at least 20 dB lower at 8 kHz than at 250 Hz.27 These findings also provide important information on the assessment of misophonic triggers, such as high-pitched screeching, that are reported by some patients.

Misophonia can be managed through tinnitus retraining therapy (TRT), cognitive behavioral therapy (CBT), compassion training, distress tolerance, mindfulness, and acceptance-based treatment, among others.28 Comorbid conditions such as depression and anxiety could be treated with antidepressants and anxiolytics. Brout and colleagues recommended a multidisciplinary care pathway to develop coping skills through CBT, mindfulness, and behavioral change.29 In general, it is understood that mental health and comorbid factors such as anxiety and depression can exacerbate misophonic responses. Parental mental health has also been explored, and the effectiveness of treatment for sound sensitivity disorders has been shown to influence the treatment of people with a history of parental mental health illness.30, 31

TRT. TRT employs extensive counseling and sound therapy. In a study by Margaret and Pawel Jastreboff, on a relatively large number of patients with decreased sound tolerance disorders, 152 out of 184 patients with both misophonia and hyperacusis and 139 out of 167 patients with misophonia alone experienced improvements after TRT.32 In the clinical practice of Ali Danesh, PhD, the use of ear-level sound generators with pleasant signals such as Zen fractal music or other sounds streamed via a smartphone to a Bluetooth hearing device, combined with extensive counseling, were shown to be helpful in managing those with misophonia. However, no solid evidence-based data are available for this observation.

CBT. Since the models describing hyperacusis and tinnitus have significant commonalities with misophonia, it can be presumed that the methods used to treat tinnitus and hyperacusis can also be effective for misophonia. The evidence-based and research-proven protocols for hyperacusis and tinnitus management support CBT.33-35 The CBT protocol for misophonia is designed to identify negative automatic thoughts (NATs) and examine the validity and truth behind those negative thoughts. After a few sessions, patients realize that most NATs such as anger, isolation, fear, or poor relationships are false perceptions. Aazh and colleagues provide a case formulation model for a CBT plan to manage misophonia.36 This model starts with the exploration of misophonia patients initial emotional responses, such as anger and irritation, and their physical complaints, such as tightening of the stomach, static-like tingling sensations on the skin, or pain, when exposed to trigger sounds. These physical sensations and initial emotional reactions to trigger sounds generate a series of negative thoughts that lead to further emotional and physical responses, which in turn result in evaluative thoughts, hence generating a vicious circle of thoughts (Fig. 1).36 The CBT-based interventional model for misophonia aims to break this cycle by assisting the patients in examining and exploring their negative thoughts so they can process and modify them.

Although there is no quick fix or magic pill for misophonia, the use of sound therapies, behavioral modifications, and CBT seems to be promising. Further research will be able to show the effectiveness of other treatment methods, such as electrical and magnetic stimulations, in the search for misophonia management.

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Misophonia: A Neurologic, Psychologic, and Audiologic... : The Hearing Journal - LWW Journals

Precision medicine in 2020: what barriers remain for drug developers? – pharmaceutical-technology.com

By adminMarch 4, 2020Gene Therapy Clinics

High costs, data management issues and recruitment problems are some of the main challenges for personalised medicine in clinical development. Credit: US Air Force/Kemberly Groue Genome sequencing costs are falling fast, opening the playing field for developing highly personalised drug candidates. Personalised medicine in the 21st century offers the promise of therapies customised based on the study of what truly makes us unique: our DNA.

The importance of the individual has been widely established in medicine since time immemorial. The well-worn adage that physicians should treat the patient, not the disease has been around since the 19th century, and the awareness of that message is far older than that. Even Hippocrates, the father of Western medicine who treated patients in the fifth century BC, stressed the importance of treating each patient as an individual.

For the sweet [medicines] do not benefit everyone, nor do the astringent ones, nor are all patients able to drink the same things, Hippocrates wrote.

Hippocrates might have tailored his rudimentary treatments based on the patients age, physique and other easily observable factors, but personalised medicine in the 21st century offers the promise of therapies customised based on the study of what truly makes us unique: our DNA.

Advancements in genomics, proteomics, data analysis and other fields both medical and technical are gradually facilitating the development of laser-focused drugs, as well as the ability to predict peoples personal risk factors for particular diseases and how individual responses to various treatments might differ.

After years of anticipation, there is now evidence that governments around the world have clocked the importance of personalised medicine and are driving efforts to the build the genetic data sets and biobanks that are required to push the science forward. Former US President Barack Obama launched the Precision Medicine Initiative to great fanfare in 2015; the scheme has since evolved into the All of Us research programme, which aims to gather health data from more than a million US volunteer-citizens to unlock new insights.

In the UK, the 100,000 Genomes Project reached its goal of sequencing 100,000 whole genomes from 85,000 NHS patients with cancer or rare diseases. Genomics England has noted that so far, analysis of this data has revealed actionable findings in around one in four rare disease patients, while about 50% of cancer cases suggest the potential for a therapy or clinical trial.

You can match a blood transfusion to a blood type that was an important discovery, said Obama at the launch of the Precision Medicines Initiative, summarising the broad appeal of personalised therapies and diagnostics. What if matching a cancer cure to our genetic code was just as easy, just as standard? What if figuring out the right dose of medicine was as simple as taking our temperature?

The stage might be set for personalised healthcare to dramatically transform public health, but few in the medical field would deny that the world is hardly ready yet. Transitioning from the traditional one-cure-fits-all treatment model to new processes that leverage patients genetics, lifestyles and environmental risk factors is an immense task that presents challenges in both the laboratory and the clinic.

Oncology is, by a landslide, the field that has been most impacted by developments in precision medicine; around 90% of the top-marketed precision treatments approved in 2018 were cancer therapies, while other therapeutic areas have lagged far behind. The majority of approved precision medicines in oncology achieve something of a halfway house between the old way and the new they fall short of being tailored to a specific individual, but they allow for more detailed stratification of patients by the oncogenic mutations of their tumours, which may be driving cancer cell survival and growth.

Common examples of these mutations are HER-2 in certain breast and stomach cancers, BRAF in melanoma and EGFR in lung cancer. High expression of these proteins at cancer sites can be targeted by precision treatments, such as Roches monoclonal antibody Herceptin (trastuzumab) for HER-2, Genentechs BRAF inhibitor Zelboraf (vemurafenib), and Roches EGFR inhibitor Tagrisso (osimertinib). Regulators such as the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) are also increasingly approving tumour-agnostic treatments the first and most famous of which is Mercks immunotherapy Keytruda (pembrolizumab) which target specific biomarkers regardless of tumour location.

But despite the availability of a growing menu of personalised cancer treatments, actually matching patients up to the right therapy can be difficult. According to a survey of US acute care organisations conducted by Definitive Healthcare and published in December 2019, just over 20% had established precision medicine programmes. Investment in genomic testing is vital to quickly get patients on the best treatment course, but financial and operational barriers remain.

The foremost among these is the cost associated with genomic sequencing and the use of companion diagnostic devices, cited by 28% of Definitive Healthcares respondents as the biggest challenge for already-established precision medicine schemes. Lack of expertise is another obstacle, as many physicians may struggle to accurately interpret test results without specialist assistance another major cost driver for clinics and hospital departments trying to build pathology teams that are up-to-date with the newest tests. A 2018 survey of 160 oncologists by Cardinal Health found that 60% of physicians who dont use genomic tests avoid them because of the difficulty of interpreting the data.

In clinical research and development, too, there are growing pains associated with moving the pharmaceutical pipeline towards drugs targeting smaller patient sub-groups. Again, cost is a central issue companion diagnostics dont come cheap, finding and validating biomarkers to guide targeted therapies is a lengthy task, and analysing vast amounts of data often requires new teams with specialised knowledge.

The expense of incorporating a host of new processes into innovative trial designs not to mention the cost of manufacturing cell and gene therapies obviously has an impact on the list price of personalised drugs that win approval. This is most clearly seen in the eye-watering prices of some of the worlds first truly individualised cancer treatments, chimeric antigen receptor T-cell (CAR-T) therapies.

Treatments such as Novartiss Kymriah and Gileads Yescarta remove T-cells from the patients blood, modify them to target tumour cell antigens and then infuse them back into the blood stream. These therapies have achieved impressive results in rare and advanced cancers, but cost upwards of $400,000 per patient, limiting their reimbursement options among both private and public payers. Promising advances in CAR-T manufacturing and potential off-the-shelf T-cell production could help bring these costs down in the years to come, but for now the problem remains.

As for the broader clinical trial eco-system, these studies have been historically set up to assess a drug candidates safety and efficacy in an increasingly large segment of the patient population, building evidence towards the regulatory approval process. Bringing a personalised medicine through the clinical development process is a new paradigm in a number of ways; as well as the aforementioned cost drivers, there can be an extra enrolment burden to identify and recruit patients this is already a common cause of trial failure, but its all the more difficult when youre looking to access a small patient sub-group with the appropriate biological profile.

The difficulty of providing sufficient evidence of safety and efficacy can also present issues where current regulations struggle to accommodate new innovations in personalised medicine. Smaller trial designs present statistical problems in terms of understanding a drugs definitive risk-benefit profile, and while some personalised applications can be discovered as part of larger trials that fail to meet their endpoints outside of a select patient group with particular biomarkers, many current regulations dont accept post hoc analysis and would require an entirely new trial.

Personalised medicine developers desire better guidance on how best to design a successful clinical trial for a personalised therapy, because absent guidance, they risk presenting suboptimal evidence regarding stratification options, reads a 2017 study on personalised medicine barriers, published in the Journal of Law and Biosciences. Designing clinical trials for differently responding subgroups (for example, biomarker-positive and biomarker-negative groups) requires additional time and resources. Companies are reluctant to make this investment without a commensurate increase in the certainty of regulatory approval.

The increasing use of surrogate endpoints, conditional approvals and real-world data is helping to address these issues, but theyre not yet an ideal solution. Conditional approvals rely on very careful post-marketing observation and analysis, while the value of surrogate endpoints has been questioned, adding to the tension between accelerating approvals and ensuring patient safety.

The ultimate benefits of creating more personalised treatments are clear, and their advantages for human health could, in the long-term, be matched by their economic returns. After all, quickly treating patients with the right therapy for them or, even better, using knowledge of a patients genetic risk profile to prevent illness in the first place would be a huge financial gain for overburdened health systems.

Todays costs are gradually falling, as NIH data on DNA sequencing costs demonstrate. But there is still a long way to go before we can wave goodbye to the blanket drug development that has dominated modern pharma for decades, even in the advanced field of oncology, let alone other therapeutic areas. Only a sustained and holistic push from regulators, drug developers, clinicians, governments and others will be enough to bring us over the line.

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Precision medicine in 2020: what barriers remain for drug developers? - pharmaceutical-technology.com

Hemophilia Treatment Market outlook with industry review and forecasts – Technology Magazine

By adminMarch 4, 2020Gene Therapy Clinics

Hemophilia Treatment Market will exceed USD 14.8 billion by 2025; as per a new research report.

Increasing burden of bleeding disorders including hemophilia will stimulate global hemophilia treatment market growth in upcoming years. According to the World Federation of Hemophilia, the global prevalence of hemophilia was depicted to be around 184,723 in the year 2016. Currently, the number of individuals in the U.S. suffering from hemophilia is predicted to be about 20,000. Individuals with hemophilia A is anticipated to be around 149,764 and individuals with hemophilia B are around 29,712. According to the Center for Disease Control and Prevention, average age for diagnosis of hemophilia is 36 months for mild hemophilia, 8 months for moderate hemophilia and 1 month for severe hemophilia. Thus, rise in prevalence of hemophilia will boost the hemophilia treatment business growth in the forthcoming period.

Rise in initiatives and educational programs by the government will further boost the hemophilia treatment market. Organizations such as World Federation of Hemophilia (WFH) along with government of several countries carry out diagnosis program. One such program is Cornerstone initiative by WFH for underprivileged people. Furthermore, increase in R&D activities along with novel drug launches will upsurge the growth of hemophilia treatment market; for instance, gene therapy is under clinical investigation.

Request sample copy of this report @ https://www.decresearch.com/request-sample/detail/2772

Additionally, increasing adoption of prophylaxis treatment by individuals suffering from hemophilia is contributing to the growth of the market. However, high-costs associated with hemophilia drugs will limit the growth of hemophilia treatment market. Furthermore, lack of awareness and adequate treatment in developing and under-developed countries will have negative impact on the market growth.

Hemophilia A segment will hold a notable revenue size of USD 6,743.7 million in the year 2018. The prevalence rate is high in developing and underdeveloped economies. According to the National Hemophilia Foundation, hemophilia A is four times more common than hemophilia B. Thus, increase in number of individuals suffering from hemophilia A along with rise in awareness regarding treatment of hemophilia will positively impact the growth of segment.

Recombinant coagulation factor concentrates market is estimated to dominate the market in the coming years and grow at 5% over the estimation period. Additionally, recombinant coagulation factor concentrates have a low risk of transmitting an infectious disease and are used to prevent viral infection. Also, recombinant coagulation factor concentrates increases the safety of replacement therapy, increasing its adoption rate leading to increased business growth over the forecast period.

Prophylaxis treatment market segment will hold a significant revenue size of USD 7,219.9 million in 2018 and is estimated to hold majority market share over the forthcoming years owing to rise in adoption of prophylaxis treatment. Patients with severe hemophilia are recommended prophylactic treatment to avoid bleeding episodes. Furthermore, some recent studies have proven prophylactic treatment to be quite effective with incidence of joint pain getting reduced by prophylactic treatment. Thus, better efficacy and merits over other treatment will increase its adoption rate, thereby leading to upsurge in prophylaxis segment revenue.

Make Inquiry about this report @ https://www.decresearch.com/inquiry-before-buying/2772

Factor replacement therapy market will grow at a significant pace owing to its preference over other therapies. Recombinant factor replacement therapy is the first line treatment for patients with moderate to severe hemophilia. Increasing R&D investments and new drug launch in replacement therapy is estimated to drive the growth of segment. Furthermore, increasing awareness amongst the patients regarding factor replacement therapy will boost the market growth.

Coagulation factors market held a significant market share and was valued at USD 9,948.3 million in the year 2018. Coagulation factors are used in replacement therapy. The rise in prevalence of moderate to severe hemophilia will spur the demand of replacement therapy, thereby boosting the demand for coagulation factors.

Injectables route of administration is projected to witness growth at 5% over the estimation period owing to majority of medications that are dispensed in injectable form. Also, bioavailability of intravenously administered drug is 100%, thus, intravenous route is preferable for quick action. Nasal spray segment is anticipated to foresee momentous growth in the coming years due to various advantages offered by nasal route over injectables such as ease of administration.

Hemophilia treatment in adult will dominate the patient segment and was valued at USD 6,778.2 million in 2018, due to rise in prevalence of hemophilia in adults. According to one recent article, around 65% of the individuals suffering from hemophilia are adults. Also, the chances of developing moderate to severe hemophilia are high in adults. Thus, growing age results in high occurrence of hemophilia, thereby impacting the growth of segment.

Hemophilia treatment centers (HTCs) is expected to grow at 4.6% in the coming years owing to increase preference of these centers over hospitals and clinics. Hemophilia treatment centers are primarily aimed to provide comprehensive hemophilia treatment to the underprivileged patients. Additionally, HTCs focus on organizing initiatives for spreading awareness regarding hemophilia in developing and under-developed countries. Also, special care provided by HTC's will increase its preference, thereby fueling its growth.

U.S. hemophilia treatment market was valued at USD 3,539.3 million in the year 2018. According to the National Hemophilia Foundation, the number of individuals suffering with hemophilia are estimated to be around 20,000. Furthermore, favorable reimbursement policies provided by hemophilia treatment centers and insurance in the country will raise the adoption rates of hemophilia treatment.

India hemophilia treatment market is expected to grow at 9.2% in the coming years owing to rise in awareness regarding hemophilia treatment in developing economies. Furthermore, rise in government initiatives, increase in healthcare expenditure and educational programs conducted by government will further increase the market growth.

Some of the major players involved in the global hemophilia treatment market are Sanofi SA, Swedeish Orphan Biovitrum AB, Biotest AG., Genentech, among others. Companies are implementing several business strategies such as strategic collaborations, mergers and acquisition to increase their product portfolio and market footprint. For instance, in June 2018, Biotest opened two plasma collection centers in Czech Republic to complement its plasma collection capacity.

Original post:
Hemophilia Treatment Market outlook with industry review and forecasts - Technology Magazine

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Edited Transcript of FLXN earnings conference call or presentation 12-Mar-20 8:30pm GMT – Yahoo Finance

Why soap works – even on coronavirus – and how its discovery altered human history – Chattanooga Times Free Press

Why Soap Works – The New York Times

Flexion Therapeutics Inc (FLXN) Q4 2019 Earnings Call Transcript – Motley Fool

Scarborough brother and sister, aged 11 and 12, with the genetic cholesterol condition FH are helped by new clinic – The Scarborough News

Spinal Muscular Atrophy Treatment Market to Exhibit a Spectacular CAGR of 28.9%; Growing Initiatives by Government Authorities for Pre-Diagnosis to…

Study maps landmarks of peripheral artery disease to guide treatment development – University of Illinois News

Misophonia: A Neurologic, Psychologic, and Audiologic… : The Hearing Journal – LWW Journals

Precision medicine in 2020: what barriers remain for drug developers? – pharmaceutical-technology.com

Hemophilia Treatment Market outlook with industry review and forecasts – Technology Magazine