Stem Cell Clinic

Edited Transcript of BMRN earnings conference call or presentation 29-Apr-20 8:15pm GMT – Yahoo Finance

By Gene Therapy Clinics

NOVATO Apr 30, 2020 (Thomson StreetEvents) -- Edited Transcript of Biomarin Pharmaceutical Inc earnings conference call or presentation Wednesday, April 29, 2020 at 8:15:00pm GMT

* Brian R. Mueller

BioMarin Pharmaceutical Inc. - Senior VP of Finance, Acting CFO & CAO

* Henry J. Fuchs

BioMarin Pharmaceutical Inc. - President of Worldwide Research & Development

BioMarin Pharmaceutical Inc. - Chairman & CEO

BioMarin Pharmaceutical Inc. - Executive VP & Chief Commercial Officer

* Robert A. Baffi

BioMarin Pharmaceutical Inc. - President of Global Manufacturing & Technical Operations

BioMarin Pharmaceutical Inc. - VP of IR

Evercore ISI Institutional Equities, Research Division - Senior MD & Equity Analyst

* Kennen B. MacKay

RBC Capital Markets, Research Division - MD & Co-Head of US Biotechnology Research

* Peter B. Kim

* Philip M. Nadeau

William Blair & Company L.L.C., Research Division - Co-Group Head of Biopharma Equity Research

Sanford C. Bernstein & Co., LLC., Research Division - VP

Welcome to the BioMarin First Quarter 2020 Financial Results Conference Call. Hosting the conference call today from BioMarin is Traci McCarty, Vice President of Investor Relations. Please go ahead, Traci.

Traci McCarty, BioMarin Pharmaceutical Inc. - VP of IR [2]

Thank you, May, and thank you, everyone, for joining us today. To remind you, this nonconfidential presentation contains forward-looking statements about the business prospects of BioMarin, including expectations regarding BioMarin's financial performance, commercial products and potential future products in different areas of therapeutic research and development.

Results may differ materially depending on the progress of BioMarin's product programs, actions of regulatory authorities, availability of capital, future actions in the pharmaceutical market and developments by competitors, and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, such as 10-Q, 10-K and 8-K reports.

On the call remotely from BioMarin management today are J.J. Bienaim, Chairman and Chief Executive Officer; Jeff Ajer, Executive Vice President, Chief Commercial Officer; Robert Baffi, President, Global Manufacturing and Technical Operations; Hank Fuchs, President, Worldwide Research and Development; and Brian Mueller, acting Chief Financial Officer. We hope to keep this call to 1 hour and also give everyone the opportunity to ask a question today, so we request that you limit yourself to one during the Q&A portion of the call. Thank you for your understanding. I will now turn the call over to our Chairman and CEO, J.J. Bienaim.

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Jean-Jacques Bienaim, BioMarin Pharmaceutical Inc. - Chairman & CEO [3]

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Thank you, Traci. Good afternoon, and thank you for joining us on today's call. We hope you and your families are healthy and managing through these unusual circumstances brought about by the COVID-19 virus. So these are unprecedented times, but the essential nature of our medicines to the patients who need them has enabled BioMarin to weather the challenge of COVID-19 quite well. Equally as important, I want to underscore the extraordinary dedication of our employees who have kept operations running smoothly in order to maintain access to our therapies around the world.

Our first quarter record results of $502 million of total revenues or 25% growth over last year, the testament to the importance of our therapies and our diversified product base and commercial footprint. Due in part to the sale of Firdapse, GAAP net income in the first quarter was $81.4 million, exceeding our current full year guidance range of $20 million to $80 million.

In the first quarter, we experienced minimal interruptions due to COVID-19, but we do anticipate the potential for more meaningful business disruptions for the remainder of 2020 due to the pandemic. As a result, we have chosen to reduce our full year total revenue guidance by around 5% or a total of $100 million, while maintaining both GAAP and non-GAAP income fully estimated provided earlier this year. Despite potential near-term impacts to our commercial business on COVID-19, our next blockbusters, BMN 270, valoctocogene roxaparvovec, (inaudible) valrox for hemophilia A and vosoritide for achondroplasia continuing to advance, and Jeff will reveal our recently approved brand name for BMN 270 in a moment.

Briefly on vosoritide for achondroplasia. In the quarter, we announced that based on recent meetings, a successful meeting with health authorities in the U.S. and Europe, we plan to submit marketing applications to the FDA and EMA in the third quarter of this year. If approved, vosoritide will be the first medicine for the treatment of achondroplasia in the U.S. and Europe. So we are delighted that this potential therapy proceeds a step closer to regulatory.

In conclusion, BioMarin employees have risen to the evolving challenges of the COVID-19 pandemic, demonstrating a high level of commitment and dedication to the patients we serve. The underlying fundamentals of our business remains strong and our manufacturing and supply chain resilience. We have built a durable base business with essential medicines transition the pipeline to address larger rare indications, diversified risk and positions ourselves for substantial success in both the near term and the long term. We are confident in our ability to manage through this ongoing global health crisis, while staying grounded in our long-term strategy for success.

I'd like to say a few words about Robert Baffi, who has made tremendous contributions to the organization over the last 20 years. During this time at BioMarin, he has manufactured the most complex biological products in the world and visits the most advanced commercial scale gene therapy manufacturing capability. His leadership, technical expertise, foresight and dedication has played a key role in where we stand today, and we want to acknowledge his many contributions. Thank you, Robert. And we are pleased that he will remain with BioMarin through the review of BMN 270 valrox and the vosoritide marketing applications to ensure manufacturing continuity as the successor, Greg Guyer, begins his journey with us in May, coming from Bristol-Myers Squibb.

Thank you all for your continued support. And now I would like to turn the call over to Robert to say a few words. Robert?

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Robert A. Baffi, BioMarin Pharmaceutical Inc. - President of Global Manufacturing & Technical Operations [4]

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Thank you, J.J. Innovation has always been at the core of BioMarin's success. During my 20 years, as the Head of Technical Operations, and has instill in the company a few guiding principles to foster a culture of innovation: first, let science inform and lead decision-making; second, let compliance focus our efforts on patient safety and clinical outcomes; and third, let ingenuity create adaptivity and resiliency in our approach to drug development. These 3 tenants, infused with the talents of the most dedicated people I've ever worked with, have consistently enabled us to take research ideas rapidly through development, navigating the complexity of the regulatory approval process in a highly effective and differentiated manner to meet the needs of patients.

BioMarin's leadership team has shared and supported the vision for creating a fully integrated company with technical operations, powers clinical studies and commercial demand, and is an integral component of strategic technology development paradigms for assuring the timely delivery of an uninterrupted supply of product.

Furthermore, innovative and appropriately implemented CMC strategies linked to a faster clinical design allows for rapid development and high success rates that benefit both patients and shareholders alike. As a company, we are going through multiple transitions simultaneously, challenging in some ways, invigorating in others. Our transition to profitability this year provides the resources to develop more innovative therapies. Our transition to gene therapy product leverages our clinical manufacturing and commercial capabilities and place us squarely at the forefront of the emerging technological advancement in precision medicine. Our transition and technical operation leadership provides the opportunity to build an innovative approach to drug development to fuel our growth.

When I first saw that to Dr. Greg Guyer's CV, I could not help but be impressed with the scope of his responsibility and the experiential variety and diversity of his career. In many ways, while a different journey, it shared a lot of commonality with my own and that is not to let us vote to BioMarin. I am confident and committed that the transition in technical operations at BioMarin from me to Greg will build on the legacy of science, compliance and ingenuity, for our patients that will benefit from the products that will emerge from our efforts, for our employees and their careers, and for our shareholders as we become profitable.

In terms of licensure of our gene therapy manufacturing facility in support of BMN 270 approval, I'm pleased to share that the Health Products Regulatory Authority of Ireland conducted, on behalf of the European Medicines Agency, a pre-approval inspection in Q1. This inspection involves a detailed review of the facility, equipment, process, and analytical studies and relevant documentations generated in support of validation, production and testing. Following this inspection, a CGMP certification was granted, allowing for commercial production and distribution of BMN 270 in the EU when the product is approved.

At present, the inspection of the facility by FDA is expected to be completed during Q2, allowing full licensure in the U.S. of the facility consistent with the August 21 PDUFA action date. We have more than 400 doses of commercial BMN 270 ready for potential launch later this year, and remain very enthusiastic about the prospects for introducing the first gene therapy product for a bleeding disorder to the hemophilia community as soon as possible.

I thank you for your support throughout my time at BioMarin. And now I'd like to turn the call over to Jeff to discuss the commercial business update. Jeff?

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Jeffrey Robert Ajer, BioMarin Pharmaceutical Inc. - Executive VP & Chief Commercial Officer [5]

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Thank you, Robert. As we begin 2020, I'm very pleased with the team's performance across all brands and all regions during the quarter. As J.J. mentioned, we achieved our highest quarterly revenue on record, with total revenues of $502 million in the first quarter with net product revenues marketed by BioMarin, up 24% to $433 million. This achievement reflects the fundamental strength and growth of our business despite near-term challenges related to COVID-19, which I will address in a moment.

On to results in the quarter and starting with Palynziq. In the U.S., the trend of increasing revenue based on a steadily growing base of patients on commercial therapy, including progression from induction and titration to daily maintenance dosing, continued in Q1. In the early part of the quarter, we did experience a seasonal slowing of new patient enrollments and patient starts, somewhat mirroring our historical experience with Kuvan in the United States. We are reporting $35 million in Palynziq revenue for the first quarter, with the majority of that revenue coming from the U.S.

In Europe, in the first quarter, multiple clinics across Germany continued to actively treat patients with Palynziq and early uptake signals are encouraging. During the quarter, we made significant progress in Germany, adding clinics that now have some experience prescribing Palynziq and managing patients through the induction and titration phase to daily maintenance dosing. As the number of commercial patients in Germany steadily grows, we anticipate meaningful revenue contribution from the EU starting this year. We anticipate finalizing price and reimbursement negotiations in Germany by mid this year, an important step toward getting price and reimbursement approvals in other high priority European markets.

Kuvan contributed $122 million in revenues in the quarter or 14% growth year-over-year, with most of that growth coming from the United States. Vimizim revenues grew 9% year-over-year, contributing $137 million in the first quarter, driven by an 11% increase in patients year-over-year. This is reflective of the continued anticipated growth potential we expect for Vimizim.

Turning to Naglazyme. Revenues totaled $114 million, a 32% year-over-year growth for the well-established brand. As with Vimizim, the impact from uneven large order patterns makes the quarterly comparison difficult. The number of commercial patients on Naglazyme grew by 6% in the past year, and is indicative of the ongoing growth potential for this brand, nearly 15 years since being approved.

And finally, Brineura contributed $24 million in net product revenues, which represented 97% year-over-year growth. These revenues were essentially flat over Q4, and that was driven by a modest year-end inventory build in the EMEA region in Q4. Importantly, the growth in Brineura revenues compared to prior year reflects an underlying growth of 86% in commercial patients. We are seeing a net increase in patients benefiting from Brineura treatment due to the success of our disease awareness and patient identification programs.

Taken together, we are pleased with first quarter results and demand for our products. And while we experienced minimal financial impact in the first quarter due to COVID-19, we anticipate the potential for a higher degree of impact during the remainder of 2020 as disruptions of day-to-day operations of clinics and hospitals flow through our business.

Our global commercial teams will continue to adjust to implement innovative approaches to engage with clinics and patients to ensure continuity of access to our medicines. Where possible, we are supporting home infusion efforts to help mitigate impact. However, some COVID-19 disruption, the new patient starts as well as to ongoing infusion center visits from existing patients are expected to continue. As a result, we are reducing total revenue guidance by 5% at the midpoint to between $1,850 million to $1,950 million for the full year 2020. The vast majority of today's updated total revenue guidance reflects adjustments to in line brands, including Vimizim, Naglazyme and Palynziq. And assumes our business will return to normalized demand patterns in the second half of 2020. Although we did not give BMN 270 2020 revenue guidance in February, our 2020 total revenue guidance did assume some contribution from BMN 270 in Europe.

And now I'd like to end my remarks with an update on our hemophilia gene therapy program and introduce you to the intended brand name, ROCTAVIAN. In previous calls, you've heard references to valrox, which was an abbreviated form of our INN or international nonproprietary name, valoctocogene roxaparvovec or alternatively, our program identifier, BMN 270. Both the FDA and the EMA have accepted ROCTAVIAN as our brand name, and we look forward to adopting ROCTAVIAN as we get closer to launch. In the meantime, we will cease to use valrox, so as to not confuse it with our intended brand name.

Other key launch readiness activities have continued to progress. We have essentially built out the commercial team in the United States, and have added key individuals to support priority in markets in the EU. The majority of these new employees have substantial and diverse experience in hemophilia. Our brand campaigns also continue to develop as anticipated and teams have pivoted to virtual and digital platforms, allowing for ongoing engagement with the marketplace in lieu of face-to-face interactions. Obviously, certain activities are more amenable to virtual engagements than others, and where COVID-19 is challenging us most in the short-term is with gene therapy educational programs and site readiness.

Fortunately, with the team already on board, we anticipate being well prepared to launch if we receive regulatory approvals. In the meantime, we have recently finished some very positive pricing research, which validated payer willingness to embrace ROCTAVIAN with the current data set, and we'll look forward to providing you with updates on our pricing at launch.

Thank you for your attention, and I will now turn the call over to Hank to provide an R&D update. Hank?

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Henry J. Fuchs, BioMarin Pharmaceutical Inc. - President of Worldwide Research & Development [6]

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Thanks, Jeff. I'd also like to echo J.J.'s expression of deep and heartfelt appreciation for the time Robert has spent with us. And also to welcome Greg Guyer, to the organization where the secret sauce is bottled.

The R&D organization is delighted that our next significant product opportunities continue to progress, particularly under the circumstances brought about by COVID-19. The ability to come to work mostly virtually and focus on the advancement of our innovative products to date, ROCTAVIAN and vosoritide has been especially gratifying and a welcome distraction from the ongoing pandemic. I want to acknowledge and thank our teams for their commitment and contributions during these challenging times. I have been impressed by your flexibility and your ability to keep the story going while we are dealing with the pandemic.

Starting with ROCTAVIAN, and with a strong and memorable brand name, congratulations, Jeff, the FDA is committed to meet the August 21 PDUFA action date. In Europe, our marketing authorization application filing remains on accelerated assessment at this time. However, the review procedure is to be extended by at least 3 months due to COVID-19 delays. Further, as is the case with most filings that initially receive accelerated assessment, we believe there is a high possibility that our M&A will revert to a standard review procedure from accelerated assessment. Based on these assumptions, we expect the CHMP opinion by late '20 or early '21.

We continue to plan to share our 3 -- our 4-year update of the 16, 13 vector genome per kilo dose as well as the 3-year update on the 40, 13 vector genome per kilo dose in the middle of the year, but the form is as yet to be defined given the changing environment for medical meetings. We have a data analysis plan in place. We'll move forward with business as usual, but the venue and method for providing the update is still fluid at this point. So thanks for bearing with us.

Importantly, we do not expect COVID-19 to impact the time lines for completion of the ROCTAVIAN Phase III trial. Enrollment was completed in November of last year, and one of the benefits of being a onetime treatment is that patients do not need to receive therapy on a chronic basis. We're also confident that the integrity of the ongoing data collection for the study -- for this pivotal study is being sufficiently maintained as home health care solutions align nicely with the collection of the primary endpoint annualized bleed rate data.

Turning now to vosoritide for the treatment of achondroplasia. As J.J. mentioned, we plan to submit a global marketing applications in the third quarter of this year. Our multipronged development program, including a long-term Phase II clinical results in 5 to 18-year-old children, comprehensive natural history data, the ongoing study of newborns through 5 years and the highly statistically significant placebo-controlled Phase III trial makes for a very comprehensive data package spanning more than 5 years of treatment with children with achondroplasia. Again, we're the beneficiary of fortunate timing and that our pivotal submission data read out prior to the pandemic, and now much of the work can be concluded remotely. If approved, vosoritide would be the first and only medicine designated for the treatment of achondroplasia in the U.S. and in European Union.

We continue to look forward to publishing the full data from the Phase III study later this year, and we're pleased to let you know that our late-breaker has been accepted in an upcoming medical Congress. The presentation will include 1 year growth velocity, height Z scores, body proportionality, safety and subgroup analyses. So stay tuned for more specifics as to when and where those data will appear.

The Phase II study of vosoritide in 0 to 5 year olds, referred to as study 206, is proceeding well, and we are very pleased that safety data from children ages 6 months to 5 years participating in that study will be available as part of our registration package. We're grateful that the timing of key studies has aligned well with our pre-COVID-19 plans.

Moving to BMN 307, our investigational gene therapy for phenylketonuria. We're continuing to prepare new sites to open in order to enroll patients when it is safely do so given the COVID-19 circumstances. We're excited about the prospect of BMN 307 as it represents a third treatment for phenylketonuria in our PKU franchise and a second gene therapy development program, leveraging our learnings and capabilities from ROCTAVIAN. Currently, we expect the study to start later -- we expect to start the study later in 2020.

The R&D organization is energized by the opportunities before us in 2020, with both ROCTAVIAN for severe hemophilia A and vosoritide for children with achondroplasia advancing towards potential approvals. We are hopeful that these innovative treatments will be available in the very near future. We look forward to updating you on our progress over the coming quarters, and thank you for your continued support.

And I'll now turn the call over to Brian to review the financials.

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Brian R. Mueller, BioMarin Pharmaceutical Inc. - Senior VP of Finance, Acting CFO & CAO [7]

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Thank you, Hank. Please refer to today's press release summarizing our financial results for full details on the first quarter of 2020, and as usual, our comprehensive report on the quarter will be available in our upcoming Form 10-Q, which we are on track to file over the next couple of days.

As Jeff mentioned, we are experiencing some modest impacts from the COVID-19 pandemic and as a result, we have updated full year total revenue guidance to between $1.85 billion to $1.95 billion. As Jeff noted,our updated revenue guidance is based on the assumption that our business will return to normalized demand patterns in the second half of the year.

Importantly, while we lowered our revenue guidance due to the impact of COVID-19 on our commercial business, we were able to analyze our 2020 spending projections and make adjustment that allowed us to maintain our prior GAAP and non-GAAP income guidance despite the lower revenue.

Moving to operating expenses. R&D expense for the first quarter of 2020 was $142 million and lower compared to R&D expense for the first quarter of 2019 of $184 million, mostly due to less R&D activity for ROCTAVIAN, given its late stage of development as well as Palynziq following its approvals in the U.S. and Europe.

SG&A expense for the first quarter of '20 was $187 million, which was higher than SG&A expense for the first quarter of 2019 of $162 million. The year-over-year increase was expected with the single largest driver, being the commercial preparation for the launch of ROCTAVIAN and the continued global launch of Palynziq. We also incurred some unpredicted foreign currency exchange losses during the month of March as the COVID pandemic negatively affected some of our assets denominated and some of the more volatile global currencies.

During the bottom line results, we reported GAAP net income of $81 million in the first quarter of 2020, compared to a GAAP net loss of $56.5 million in the first quarter of 2019. The improvement in GAAP income was primarily due to higher revenue, lower R&D expenses and the gain on the sale of the deferred tax assets. With higher revenues and lower R&D expenses, non-GAAP income of $117 million in the first quarter of 2020 grew substantially as compared to Q1 2019 non-GAAP income of $25 million. Both of these first quarter 2020 bottom line results gives us a great start towards achieving our 2020 goals of GAAP net income on an annual basis for the first time in the company's history, a considerable growth in non-GAAP income.

I'd also like to touch on the potential tax benefit that we mentioned last quarter that may be recognized in the second half of this year. Our current 2020 GAAP net income guidance of between $20 million to $80 million, excludes the potential impact of intra-entity intangible asset transfers between BioMarin entities. If these intangible asset transfers occur, we estimate that the tax effect could result in a onetime noncash income tax benefit of greater than $500 million. As I mentioned previously, you may have seen similar transactions completed by some of our larger peers in recent quarters.

Speaking to total cash and investments, we ended the first quarter of 2020 with $1.15 billion compared to $1.17 billion at the end of December 2019. The modest decrease in total cash and investments during Q1 2020 was largely due to some timing of operating cash flows. However, the significant improvement over the first quarter of 2019 where total cash and investments decreased by $105 million. This valid cash position, coupled with vibrant business fundamentals, put us in good standing to manage through the continued uncertainty related to COVID-19.

In closing, the stronger performance of the business during the first quarter of 2020, plus our positive financial outlook for the rest of the year, indicate that 2020 should be a transformational year for the company. And the prospects of value to come from ROCTAVIAN and vosoritide, if they are approved commercially, give us an enthusiasm about our future.

Thank you for your support, and we will now open the call to your questions. Operator?

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Questions and Answers

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Operator [1]

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(Operator Instructions) Our first question is from the line of Robyn Karnauskas from Suntrust.

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Robyn Kay Shelton Karnauskas, SunTrust Robinson Humphrey, Inc., Research Division - Research Analyst [2]

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And just first off, congratulations to Greg. But to Rob, it was lovely working with you. I think you brought a breath of fresh air to working with management teams in all of my buyers (inaudible). So thank you so much. I learned a lot. I guess I'm going to start-up with some questions on ROCTAVIAN, and I hope I get that correctly. So first of all, what gives you confidence that in the United States, that there won't be any more delays? People ask me this nonstop. And then when you talk about assuming normal operations go -- resume in the second half, is that in the beginning of the second half? Do you have a time line for that? If it goes into fourth quarter, could we see further delays?

And the third question is, what are you hearing as far as like people willing to have gene therapy procedures done in the COVID environment as early as fourth quarter? Are people open to it? Is it separate from the hospitals? Or what are you hearing from the ground?

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Jean-Jacques Bienaim, BioMarin Pharmaceutical Inc. - Chairman & CEO [3]

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Okay. Hi. (inaudible) do you want to answer the question on the no delays with the FDA? Hank?

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Edited Transcript of BMRN earnings conference call or presentation 29-Apr-20 8:15pm GMT - Yahoo Finance

FDA grants orphan drug designation to multi-antigen T-cell therapy for AML – Healio

By Stem Cell Treatment

FDA News

April 30, 2020

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The FDA granted orphan drug designation to MT-401, an allogeneic T-cell therapy for the treatment of acute myeloid leukemia, according to the agents manufacturer.

MT-401 (Marker Therapeutics) an investigational, multi-tumor-associated antigen (MultiTAA)-specific T-cell therapy will be tested in a phase 2 clinical trial of patients with AML after allogeneic stem cell transplantation.

T cells are taken from healthy doors and put through a two-step cell culture process. The result is a CD4/CD8 cell mixture that can recognize multiple tumor-specific antigens.

We are pleased that the FDA has granted orphan designation to MT-401 and believe it is supportive of its potential to treat post-allogeneic stem cell transplant patients with AML a devastating and pervasive blood disease with a high medical need for a treatment, Peter L. Hoang, president and CEO of Marker Therapeutics, said in a company-issued press release.

In investigator-sponsored trials, our MultiTAA-specific T-cell product candidate was well-tolerated, and we have observed clinical benefit across various liquid and solid tumors, suggesting the product candidates ability to induce a patients own T cells to expand for a more durable antitumor effect. We look forward to initiating our company-sponsored phase 2 study, he added.

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and upon regulatory approval 7 years of market exclusivity.

The FDA granted orphan drug designation to MT-401, an allogeneic T-cell therapy for the treatment of acute myeloid leukemia, according to the agents manufacturer.

MT-401 (Marker Therapeutics) an investigational, multi-tumor-associated antigen (MultiTAA)-specific T-cell therapy will be tested in a phase 2 clinical trial of patients with AML after allogeneic stem cell transplantation.

T cells are taken from healthy doors and put through a two-step cell culture process. The result is a CD4/CD8 cell mixture that can recognize multiple tumor-specific antigens.

We are pleased that the FDA has granted orphan designation to MT-401 and believe it is supportive of its potential to treat post-allogeneic stem cell transplant patients with AML a devastating and pervasive blood disease with a high medical need for a treatment, Peter L. Hoang, president and CEO of Marker Therapeutics, said in a company-issued press release.

In investigator-sponsored trials, our MultiTAA-specific T-cell product candidate was well-tolerated, and we have observed clinical benefit across various liquid and solid tumors, suggesting the product candidates ability to induce a patients own T cells to expand for a more durable antitumor effect. We look forward to initiating our company-sponsored phase 2 study, he added.

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and upon regulatory approval 7 years of market exclusivity.

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FDA grants orphan drug designation to multi-antigen T-cell therapy for AML - Healio

CHMP Issues Positive Opinion Recommending Subcutaneous Formulation of Daratumumab for the Treatment of Patients with Multiple Myeloma – Yahoo Finance

By Stem Cell Treatment

Company Announcement

Copenhagen, Denmark; April 30, 2020 Genmab A/S (GMAB) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a Positive Opinion recommending the use of the subcutaneous formulation of daratumumab for the treatment of adult patients with multiple myeloma in frontline and relapsed / refractory settings. The CHMPs Positive Opinion for the subcutaneous formulation of daratumumab applies to all currently approved daratumumab indications in frontline and relapsed / refractory multiple myeloma settings. In August 2012, Genmab granted Janssen Biotech, Inc. (Janssen) an exclusive worldwide license to develop, manufacture and commercialize daratumumab.

We are very pleased with this Positive Opinion from the CHMP as it potentially brings the convenient dosing of subcutaneous daratumumab closer to becoming available for multiple myeloma patients in Europe, said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

The Marketing Authorization Application for this formulation was submitted to the EMA by Janssen Pharmaceutica NV in July 2019 based on data from two studies: the Phase III non-inferiority COLUMBA (MMY3012) study, which compared the subcutaneous formulation of daratumumab to the intravenous formulation in patients with relapsed or refractory multiple myeloma, and data from the Phase II PLEIADES (MMY2040) study, which is evaluating subcutaneous daratumumab in combination with certain standard multiple myeloma regimens. The topline results from the COLUMBA data were announced in February 2019 and subsequently presented in oral sessions at the 2019 American Society of Clinical Oncology (ASCO) Annual Meeting and the 24th European Hematology Association (EHA) Annual Congress. Updated data of the COLUMBA and the PLEIADES studies were presented during poster sessions at the 61st American Society of Hematology (ASH) Annual Meeting in December 2019.

About the COLUMBA (MMY3012) studyThe Phase III trial (NCT03277105) is a randomized, open-label, parallel assignment study that included 522 adults diagnosed with relapsed and refractory multiple myeloma. Patients were randomized to receive either: subcutaneous (SC) daratumumab, as 1800 mg daratumumab with rHuPH20 2000 U/mL once weekly in Cycle 1 and 2, every two weeks in Cycles 3 to 6, every 4 weeks in Cycle 7 and thereafter until disease progression, unacceptable toxicity or the end of study; or 16 mg/kg IV daratumumab once weekly in Cycle 1 and 2, every two weeks in Cycles 3 to 6, every 4 weeks in Cycle 7 and thereafter until disease progression, unacceptable toxicity or the end of study. The co-primary endpoints of the study are overall response rate and Maximum trough concentration of daratumumab (Ctrough; defined as the serum pre-dose concentration of daratumumab on Cycle 3 Day 1).

About the PLEIADES (MMY2040) studyThe Phase II trial (NCT03412565) is a non-randomized, open-label, parallel assignment study that includes 265 adults either newly diagnosed or with relapsed or refractory multiple myeloma. Patients with newly diagnosed multiple myeloma are being treated with 1,800 mg SC daratumumab in combination with either bortezomib, lenalidomide and dexamethasone (D-VRd) or bortezomib, melphalan and prednisone (D-VMP). Patients with relapsed or refractory multiple myeloma are being treated with 1,800 mg SC daratumumab plus lenalidomide and dexamethasone (D-Rd). An additional cohort of patients with relapsed and refractory multiple myeloma treated with daratumumab plus carfilzomib and dexamethasone (D-Kd) was subsequently added to the study. The primary endpoint for the D-VMP, D-Kd and D-Rd cohorts is overall response rate. The primary endpoint for the D-VRd cohort is very good partial response or better rate.

About DARZALEX (daratumumab)DARZALEX (daratumumab) intravenous infusion is indicated for the treatment of adult patients in the United States: in combination with bortezomib, thalidomide and dexamethasone as treatment for patients newly diagnosed with multiple myeloma who are eligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of patients with multiple myeloma who have received at least one prior therapy; in combination with pomalidomide and dexamethasone for the treatment of patients with multiple myeloma who have received at least two prior therapies, including lenalidomide and a proteasome inhibitor (PI); and as a monotherapy for the treatment of patients with multiple myeloma who have received at least three prior lines of therapy, including a PI and an immunomodulatory agent, or who are double-refractory to a PI and an immunomodulatory agent.1 DARZALEX is the first monoclonal antibody (mAb) to receive U.S. Food and Drug Administration (U.S. FDA) approval to treat multiple myeloma. DARZALEX intravenous infusion is indicated for the treatment of adult patients in Europe: in combination with bortezomib, thalidomide and dexamethasone as treatment for patients newly diagnosed with multiple myeloma who are eligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with bortezomib, melphalan and prednisone for the treatment of adult patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; for use in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least one prior therapy; and as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, whose prior therapy included a PI and an immunomodulatory agent and who have demonstrated disease progression on the last therapy2. The option to split the first infusion of DARZALEX over two consecutive days has been approved in both Europe and the U.S. In Japan, DARZALEX intravenous infusion is approved for the treatment of adult patients: in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone for the treatment of relapsed or refractory multiple myeloma; in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant. DARZALEX is the first human CD38 monoclonal antibody to reach the market in the United States, Europe and Japan. For more information, visit http://www.DARZALEX.com.

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Daratumumab is a human IgG1k monoclonal antibody (mAb) that binds with high affinity to the CD38 molecule, which is highly expressed on the surface of multiple myeloma cells. Daratumumab triggers a persons own immune system to attack the cancer cells, resulting in rapid tumor cell death through multiple immune-mediated mechanisms of action and through immunomodulatory effects, in addition to direct tumor cell death, via apoptosis (programmed cell death).1,2,3,4,5,6

Daratumumab is being developed by Janssen Biotech, Inc. under an exclusive worldwide license to develop, manufacture and commercialize daratumumab from Genmab. A comprehensive clinical development program for daratumumab is ongoing, including multiple Phase III studies in smoldering, relapsed and refractory and frontline multiple myeloma settings. Additional studies are ongoing or planned to assess the potential of daratumumab in other malignant and pre-malignant diseases in which CD38 is expressed, such as amyloidosis, NKT-cell lymphoma and T-cell ALL. Daratumumab has received two Breakthrough Therapy Designations from the U.S. FDA for certain indications of multiple myeloma, including as a monotherapy for heavily pretreated multiple myeloma and in combination with certain other therapies for second-line treatment of multiple myeloma.

About Genmab Genmab is a publicly traded, international biotechnology company specializing in the creation and development of differentiated antibody therapeutics for the treatment of cancer. Founded in 1999, the company is the creator of three approved antibodies: DARZALEX (daratumumab, under agreement with Janssen Biotech, Inc.) for the treatment of certain multiple myeloma indications in territories including the U.S., Europe and Japan, Arzerra (ofatumumab, under agreement with Novartis AG), for the treatment of certain chronic lymphocytic leukemia indications in the U.S., Japan and certain other territories and TEPEZZA (teprotumumab, under agreement with Roche granting sublicense to Horizon Therapeutics plc) for the treatment of thyroid eye disease in the U.S. Daratumumab is in clinical development by Janssen for the treatment of additional multiple myeloma indications, other blood cancers and amyloidosis. A subcutaneous formulation of ofatumumab is in development by Novartis for the treatment of relapsing multiple sclerosis. Genmab also has a broad clinical and pre-clinical product pipeline. Genmab's technology base consists of validated and proprietary next generation antibody technologies - the DuoBody platform for generation of bispecific antibodies, the HexaBody platform, which creates effector function enhanced antibodies, the HexElect platform, which combines two co-dependently acting HexaBody molecules to introduce selectivity while maximizing therapeutic potency and the DuoHexaBody platform, which enhances the potential potency of bispecific antibodies through hexamerization. The company intends to leverage these technologies to create opportunities for full or co-ownership of future products. Genmab has alliances with top tier pharmaceutical and biotechnology companies. Genmab is headquartered in Copenhagen, Denmark with sites in Utrecht, the Netherlands, Princeton, New Jersey, U.S. and Tokyo, Japan.

Contact: Marisol Peron, Corporate Vice President, Communications & Investor Relations T: +1 609 524 0065; E: mmp@genmab.com

For Investor Relations: Andrew Carlsen, Senior Director, Investor RelationsT: +45 3377 9558; E: acn@genmab.com

This Company Announcement contains forward looking statements. The words believe, expect, anticipate, intend and plan and similar expressions identify forward looking statements. Actual results or performance may differ materially from any future results or performance expressed or implied by such statements. The important factors that could cause our actual results or performance to differ materially include, among others, risks associated with pre-clinical and clinical development of products, uncertainties related to the outcome and conduct of clinical trials including unforeseen safety issues, uncertainties related to product manufacturing, the lack of market acceptance of our products, our inability to manage growth, the competitive environment in relation to our business area and markets, our inability to attract and retain suitably qualified personnel, the unenforceability or lack of protection of our patents and proprietary rights, our relationships with affiliated entities, changes and developments in technology which may render our products or technologies obsolete, and other factors. For a further discussion of these risks, please refer to the risk management sections in Genmabs most recent financial reports, which are available on http://www.genmab.com and the risk factors included in Genmabs most recent Annual Report on Form 20-F and other filings with the U.S. Securities and Exchange Commission (SEC), which are available at http://www.sec.gov. Genmab does not undertake any obligation to update or revise forward looking statements in this Company Announcement nor to confirm such statements to reflect subsequent events or circumstances after the date made or in relation to actual results, unless required by law.

Genmab A/S and/or its subsidiaries own the following trademarks: Genmab; the Y-shaped Genmab logo; Genmab in combination with the Y-shaped Genmab logo; HuMax; DuoBody; DuoBody in combination with the DuoBody logo; HexaBody; HexaBody in combination with the HexaBody logo; DuoHexaBody; HexElect; and UniBody. Arzerra is a trademark of Novartis AG or its affiliates. DARZALEX is a trademark of Janssen Pharmaceutica NV. TEPEZZA is a trademark of Horizon Therapeutics plc.

1 DARZALEX Prescribing information, September 2019. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2019/761036s024lbl.pdf Last accessed September 20192 DARZALEX Summary of Product Characteristics, available at https://www.ema.europa.eu/en/medicines/human/EPAR/darzalex Last accessed October 20193 De Weers, M et al. Daratumumab, a Novel Therapeutic Human CD38 Monoclonal Antibody, Induces Killing of Multiple Myeloma and Other Hematological Tumors. The Journal of Immunology. 2011; 186: 1840-1848.4 Overdijk, MB, et al. Antibody-mediated phagocytosis contributes to the anti-tumor activity of the therapeutic antibody daratumumab in lymphoma and multiple myeloma. MAbs. 2015; 7: 311-21.5 Krejcik, MD et al. Daratumumab Depletes CD38+ Immune-regulatory Cells, Promotes T-cell Expansion, and Skews T-cell Repertoire in Multiple Myeloma. Blood. 2016; 128: 384-94.6 Jansen, JH et al. Daratumumab, a human CD38 antibody induces apoptosis of myeloma tumor cells via Fc receptor-mediated crosslinking.Blood. 2012; 120(21): abstract 2974

Company Announcement no. 18CVR no. 2102 3884LEI Code 529900MTJPDPE4MHJ122

Genmab A/SKalvebod Brygge 431560 Copenhagen VDenmark

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CHMP Issues Positive Opinion Recommending Subcutaneous Formulation of Daratumumab for the Treatment of Patients with Multiple Myeloma - Yahoo Finance

Freezing Life: The Current Trends in Cryopreservation – Technology Networks

By Stem Cell Treatment

Cryopreservation has become an indispensable step in the daily routine of scientific research as well as in a number of medical applications, ranging from assisted reproduction and transplantations to cell-based therapies and biomarker identification. It is hardly possible to picture todays scientific and medical advancements without this technique.The successful development and implementation of all the therapeutic and scientific discoveries involving cryopreservation relies on the correct and safe translation of the method from the laboratory to the clinical and manufacturing scale.

With the need to correctly use this technique, more research is focusing on optimizing cryopreservation methods and investigating what the long-term effects and consequences are on the physiology of the cryopreserved material.

An important part of cell therapy research is focused on adult stem cells (ASCs). ASCs can be derived from different sources such as peripheral blood, bone marrow or adipose tissue and display strong promises because of their capacity to differentiate into any cell type of the human body.In recent work3, the team of Michael Pepper at the Institute for Cellular and Molecular Medicine in Pretoria, South Africa, explored the effects of cryopreservation on the differentiation ability of adipose tissue-derived stem cells (ADSCs). After analyzing gene expression of key adipogenic genes and the degree of differentiating cells, characterized with high levels of CD36 and intracellular lipid droplets, the scientists reported that slow freeze cryopreservation of cells shortly after their isolation causes no alterations on their ability to differentiate. Pepper is convinced of the necessity to perform such analysis when cryopreserving important cell pools: It is critical to do a post-thaw analysis of cell function to determine how the cryopreservation may have affected the cells.His team is analyzing the effects of cryopreservation on other cell types largely used in cell-based therapies such as hematological stem cells and peripheral blood mononuclear cells (PBMCs). Although they didnt observe major alterations in terms of immunophenotyping or the post-thaw proliferation of the cells, Pepper expresses his concern that more subtle characteristics might be affected.

Correct cryopreservation of cells intended for therapeutic use is crucial. This is very important particularly as cells may persist for a long time in the recipient. This area of cell therapy research definitely requires more attention, Pepper says. Moreover, his words reflect on the need to evaluate not only the direct post-thaw recovery, but to look deeper into the late-onset effects cryopreservation might have and ensure that transplanted cells have preserved their therapeutic properties.

In contrast to slow freezing, vitrification relies on the fast freezing of the material by putting it in high concentration of cryoprotectant and in contact with liquid nitrogen. This method allows the direct transition of water from liquid to solid state without crystal formation. The highly concentrated cryoprotectant prevents ice formation and therefore there is no need for slow cooling.

Although vitrification has a great potential, there are a couple of parameters that are a point of concern. The quick and drastic freeze is possible thanks to the high concentration of cryoprotectant, but the latter is also associated with higher toxicity. In some cases, an additional limitation is the direct contact of the sample with liquid nitrogen which is a predisposition for viral or bacterial contamination.The team of Christiani Amorim at the Institute for Experimental and Clinical Research in Louvain, Belgium, is approaching the challenges of vitrification in the context of ovarian auto-transplantation. Ovarian auto-transplantation consists of preserving a piece of ovarian tissue with active follicles from the pre-therapeutic ovary of a cancer patient, as chemotherapy often has damaging effects on the reproductive organs. This tissue sample will be conserved and auto-transplanted onto the patients ovary when she has recovered and wishes to become pregnant.In their recent research4, the authors used stepped vitrification, in which the concentration of the cryoprotectant is gradually increased while simultaneously temperature decreases. This avoids ice crystal formation and also prevents cryoprotectant toxicity.Although stepped vitrification has previously given good results in bovine ovarian tissue5, this was not the case for human ovarian tissue. The scientists didnt detect normal follicles following thawing and linked this to high cryoprotectant toxicity. Indeed, they observed all signs of dimethyl sulfoxide (DMSO)-related cell membrane damage: significant organelle damage, cell membrane disintegration and apoptosis. These observations imply on the variability of outcomes that the method could give when applied to the same type of tissue but from a different organism.Amorim is positive about the future of their method and recognizes the need for further research on the topic: I can see a great potential in the stepped vitrification approach, but I also believe that there is a lot we still need to learn before thinking about using it as method of choice for human ovarian tissue cryopreservation. The high cryoprotectant concentration that should be applied in this approach is my first concern. () Our study clearly showed that 50% DMSO is too high, so we need to try lower concentrations or combine it with other cryoprotectants.

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Freezing Life: The Current Trends in Cryopreservation - Technology Networks

Needs way to measure pain besides the scale of 1-10 – Coastal Courier

By Stem Cell Doctors

By Keith Roach, M.D.

DEAR DR. ROACH: Why is pain measured by numbers? I have severe pain, and doctors do not know what to do when I say it is an 11 on a scale of 1-10. My pain cant be measured by numbers. It depends on the time of day, what I have done during the day and the weather.

I have arthritis in most of my joints, specifically my spine and hips. Having had five spine surgeries, epidurals and hip shots, I have pain every day. There is not much more that can be done but to take opioids. It can be hard to make the decision either to take an opioid and go out shopping or for coffee feeling like I am in a vacuum, or to go out in pain.

There is no chronic pain support group in my area, and no one can understand how I feel, even the professionals, unless they have gone through it. So when asked how I am, I say fine. Other people dont want to hear about my pain.

Why is there not another way the doctor can measure your pain? I have given up everything I love to do in life because of pain. Theres no way to get better from pain. -- M.L.D.

ANSWER: I am very sorry to hear your story, as it is similar to those I have heard before from people with chronic pain due to many different causes. It is disappointing for me to hear that you havent found a pain specialist in your area who seems to care about helping you.

Although the 1-10 pain scale is thoroughly entrenched, it has its flaws. The biggest one is that what one person might consider a two, another person might consider a nine. Ive seen people with horrifying injuries gritting their teeth and saying their pain is a 3 while other people claim their pain from what seems to be a minor condition is a 10 (I had one person tell me the pain from getting his blood pressure taken was a 10). Because pain is subjective, there is no way of standardizing what a person means with their pain rating. However, a 10 on a scale of 1-10 is supposed to be the worst pain imaginable.

DEAR DR. ROACH: My father, 90, has neuropathy in his feet and legs, and it is very painful. He recently talked to a clinic that is offering stem cell treatment to relieve the pain. The clinic says it helps 85% of those who get the treatment; however, because of HIPAA laws, they dont provide any referrals.

The treatments are very expensive ($16,000), and results are seen in six weeks to six months. Are you familiar with this treatment, and is it effective for most people? Is this something you can recommend? -- D.B.

ANSWER: I also have seen advertisements for stem cell treatments for many conditions. For neuropathy in particular, there are no good studies that give an estimate of effectiveness. It may be the case that 85% of people treated at the clinic report improvement. But the risk of a placebo effect is very high with this kind of procedure, and I could not recommend stem cell treatments for neuropathy without better information about the risks, the benefits and how long those benefits might last.

Dr. Roach regrets that he is unable to answer individual questions, but will incorporate them in the column whenever possible. Readers may email questions to ToYourGoodHealth@med.cornell.edu.

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Needs way to measure pain besides the scale of 1-10 - Coastal Courier

Brave intensive care nurse, 27, diagnosed with rare cancer just weeks after dream wedding dies of disease – The Sun

By Stem Cell Doctors

A BRAVE intensive care nurse diagnosed with rare cancer just weeks after her dream wedding has died of the disease.

Julia Cullen, 27, from Hartlepool, won plaudits for her no-holds-barred insight into life battling a rare form of leukaemia.

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The fit and healthy nurse fell ill just weeks after marrying her beloved fiance, Peter, 29, in October 2018.

She underwent gruelling treatment programmes to combat the disease - only for it to return aggressively in February.

Julia died at home in her sleep last week surrounded by her devastated family.

She lost her mum, Cath, who was also a nurse, to the cruel disease four years ago.

Heartbroken Peter wrote on Facebook: I need to let everybody who knew my beautiful wife Julia Cullen know that tonight she unfortunately passed away after a long battle with leukaemia.

She went peacefully in her sleep surrounded by her family. Thank you for all for your support during this difficult past year.

Goodnight darling be at peace I will never forget you.

Julias dad Graham, 57, sister Louisa, 28, brother Ste, 41, and twin Jack tragically lost their mum, Cath, 61, also a nurse, to bowel cancer in May 2016.

The family has now launched an appeal in support of Anthony Nolan and encouraged people to sign up as stem cell donors.

Louisa said: My best friend, my soul, my rock, my beautiful brave sister, took her last breath last night.

Its been such a hard journey for her and us all over the last year from her diagnosis.

Goodnight darling be at peace I will never forget you.

Julia, we always said we could get through anything as long as we have each other, so Im a bit lost what to do in life now.

Julia had shared blunt photos on Instagram from her 15-month fight to show how cancer does not discriminate.

She told her followers after being diagnosed aged 26: If theres anything I want to do after going through this experience of cancer, its to raise awareness that its happening.

Its happening to the young and old, the fit and unfit, the employed and the unemployed.

Its happening now and it could happen to you. Yes. You.

It isnt glamorous, it isnt a story - its hard, its painful, its gruelling, its terrifying.

In her Instagram story, Julia told how she then spotted spots on her legs and messaged Louisa: Ive Googled it, Ive got leukaemia.

Julia was admitted to a hospital ward in January 2019, where doctors dropped the bombshell that she had Acute Lymphoblastic Leukaemia - a blood cancer rare in adults and mainly found in children.

In Julias Instagram story, she tells how she went pink after an allergic reaction to antibiotics and how shaving her head was freeing yet upsetting as f***.

She shared the highs of spending time with her friends and the lows of having to have a plastic tube - known as a Hickman line - fed through her chest.

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In February, she wrote on Instagram of her determination to win her fight, and said: You will get through it.

You wont fight it as people say, youll face it. Youll face it with whoever and whatever you have to. Because life is precious. So be thankful. Always thankful.

Brother Jack, a hospital porter, said: Words cant describe how heartbroken and devastated I am to lose my twin sister.

I love you so much and I will continue to look after our wonderful family.

To support and donate to the family's appeal visit: https://www.justgiving.com/crowdfunding/louisa-siddle-1

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Brave intensive care nurse, 27, diagnosed with rare cancer just weeks after dream wedding dies of disease - The Sun

RIP Rishi Kapoor! Succumbing to Leukemia, Veteran Actor Dies at 67 in Mumbai, Here is all You Need to Know Ab – India.com

By Stem Cell Doctors

Veteran actor Rishi Kapoor passed away today morning at around 8:45 am. He was suffering from leukemia, a type of cancer. The 67-year old actor was admitted to H N Reliance hospital in Mumbai on Wednesday morning after his health deteriorated. Rishi Kapoor took his last breath in the hospital surrounded by his family. Also Read - Twin Tragedies: After Irrfan Khan, Veteran Actor Rishi Kapoor Dies at 67; Twitter Overwhelmed With Sadness

Kapoor & Sons actor was diagnosed with leukemia in 2018 and was in New York for a long time for his treatment. Indias one of the finest actors, Rishi Kapoor lost his battle with cancer and left the world a day after actor Irrfan Khans demise. While we mourn his death and pray for his souls peace, here we tell you all about the condition he was suffering from. Also Read - Rishi Kapoor Dies at 67 (1952-2020) in Mumbai HN Reliance Foundation Hospital

Leukemia is a blood cancer in which there is an uncontrollable growth of the white blood cells (WBCs), which are responsible for protecting your body from invasion of harmful pathogens and abnormal cells. When the WBCs start dividing too quickly, they do not work normally and crowd out the red blood cells and blood platelets. Notably, WBCs are produced in the bone marrow. In case of leukemia, this spongy tissue produces abnormal WBCs. Also Read - Rishi Kapoor Fans Pray For Speedy Recovery After Actor is Shifted to ICU

Leukemia is characterised by symptoms including fever, persistent fatigue, unexplained weight loss, enlarged spleen, swollen lymph nodes, and recurrent nosebleed. People suffering from this deadly condition also experience excessive sweating, bone pain, and tiny red spots on skin.

Though the exact cause behind leukemia is unknown, doctors believe, it occurs when the WBCs undergo mutation in their DNA. This leads to rapid and abnormal growth of these cells.

Doctors conduct a physical exam to check for signs and symptoms of leukemia. They also recommend blood tests to determine if you have abnormal level of WBCs. Bone marrow test is also performed to confirm the condition. As a part of this test, a sample of bone marrow is removed from the hipbone of the patient and is sent to a lab for test.

As far as treatment of leukemia is concerned, options like chemotherapy, biological therapy, targeted therapy, radiation therapy, and stem cell transplant are currently available.

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RIP Rishi Kapoor! Succumbing to Leukemia, Veteran Actor Dies at 67 in Mumbai, Here is all You Need to Know Ab - India.com

The teenager who survived cancer, sepsis and a collapsed lung – Wales Online

By Stem Cell Doctors

Teenager Libby Waite first noticed something was wrong with her health on a family holiday to Florida. She felt exhausted the entire time and struggled to walk the shortest of diatances.

And once she returned home and her friends spotted a lump on her neck while admiring her necklace, she knew something more sinister was going on.

After seeing her GP, who sent Libby for blood tests, a private scan and biopsy revealed she had cancer.

She was immediately referred to the Noah's Ark Children's Hospital for Wales in Cardiff where she was diagnosed with the blood cancer Hodgkin lymphoma.

"As a teenage girl, my first concern was about how it would affect my appearance. I was scared I would lose my hair," Libby said.

Her mum Joanne added: "As a mother it's your worst nightmare. I felt like I had my guts wrenched out, it was awful."

Unfortunately, Libby's first few weeks in hospital took a dramatic turn when she contracted sepsis because of her suppressed immune system caused by the first dose of chemotherapy.

"I was so shocked. The doctors had first thought she would have very little side-effects, so no-one expected that reaction," mum Joanne explained.

"One minute she was sat having breakfast, the next she went quiet and unresponsive. It was frightening."

As a result of the sepsis, Libby's lung also collapsed and she had to spend two weeks in intensive care while she recovered.

Libby was eventually able to leave hospital on Christmas Eve 2015.

"Although it had been scary it was so nice to be able to get home for Christmas," said Libby.

Libby, now 18, from Pontypool, returned to hospital where she was able to continue with a different type of chemotherapy.

Despite having four doses, tests revealed that the cancer had not gone away, so she was referred to a specialist consultant in London who recommended immunotherapy - a treatment which uses a persons own modified cells to treat cancer.

It is thought that Libby was one of the first patients in Wales to receive the relatively new treatment back in 2016.

Libby had a stem cell transplant where her own cells were harvested, treated and planted back in to her body to fight the cancer.

This required a three-week isolation period where her immune system was completely supressed.

Unfortunately, Libby did lose her hair as a result of the earlier chemotherapy treatment, but she said: "It actually wasn't as bad as I thought it would be. I had a really cool wig and I was able to use make-up for my eyebrows."

Thankfully the treatment was successful and Libby is now in remission after a long recuperation period at home while she gathered her strength.

She recalled: "I felt so weak, I was confined to the sofa for weeks and I had to rebuild my strength to walk again"

Eventually Libby said she was was able to return to school and sit her GCSEs and go to her year 11 prom.

"Going to prom was the first time I finally felt like a young person again," she admitted.

"I just wanted to go out with my friends and do everything they were doing."

Libby, who is currently applying for university, is now helping Cancer Research UK launch Race for Life at Home in Wales to help carry on the fight against the disease in these unprecedented times.

She took part in her local Race for Life 5k event in Cwmbran in 2017 after her treatment.

The Cwmbran event is among many which the organisers have postponed this spring and summer to protect the countrys health during the coronavirus outbreak.

But as the nation continues on lockdown, undeterred women and men are already vowing to carry on and complete a Race for Life at Home challenge at home, in their garden or their nearest green space.

Many of the scientists and researchers funded by Cancer Research UK are currently being redeployed to help in the fight against Covid-19, including assisting with testing.

By helping to beat coronavirus, the charity said it can lessen the impact it is having on the care of cancer patients.

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Cassandra Miles, Cancer Research UKs spokeswoman for Wales, said: "At a time when it feels like everything is at a standstill, there is one thing that hasnt stopped, cancer.

"Our priority as a charity is ensuring that people affected by cancer are getting the support they need right now.

"But we are already getting people asking about doing Race for Life at Home because they dont want to see the charity lose out on vital funding. Its truly humbling to see the response.

"So from their homes, wed love for supporters to join us and Race for Life at Home in these challenging times.

"From a run or 5K walk around the garden to limbo in the living room, there is no wrong way to Race for Life at Home. With no entry fee, people might choose to twerk, limbo, star jump, squat, skip, dance, or come up with their own novel way of taking part and share it with friends. The message is very much that while we might be apart, were doing this together. There is no wrong way to get involved and join our community.

"Those lucky enough to have a garden may choose to make use of it but whatever people decide to do, we are immensely grateful for the support, now more than ever. If the idea takes off, we could be looking at hundreds of people in Wales stepping forward to Race for Life at Home and perhaps collecting sponsorship to do so."

People can visit raceforlife.org and sign up free for ideas on how they can create their own Race for Life at Home challenge.

And the Cancer Research UK Race for Life Facebook page will help people feel energised with weekly live workout sessions.

Organisers are also inviting participants to join the Race for Life at Home community by sharing photos and videos on social media using the hashtag, #RaceForLifeAtHome.

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The teenager who survived cancer, sepsis and a collapsed lung - Wales Online

Four UTSW Researchers Named to The National Academy of Sciences – D Magazine

By Stem Cell Medical Center

Four UT Southwestern Medical Center scientists have been elected to the The National Academy of Sciences, one of the top honors for American scientists.

Peer scientists selected Sean Morrison, Kim Orth, Michael Rosen, and Sandra Schmid for their original research and achievements. UT Southwestern now has 25 members of the academy, the most of any institution in Texas.

Election to the prestigious National Academy of Sciences recognizes the pioneering contributions these scientists have made to advance our understanding of basic cellular function and molecular processes with application to addressing a broad spectrum of unmet medical needs including cancer and treatments for bacterial infections, said Dr. Daniel K. Podolsky, President of UT Southwestern Medical Center via release. Their election enriches the National Academy of Sciences efforts to provide data and advice on the nations most critical issues in science, health, and medicine.

Morrison is the Director of the Childrens Medical Center Research Institute (CRI) at UT Southwestern and Professor of Pediatrics and has worked in the fields of stem cell biology and cancer, and has created new methods to purify stem cells and allow them to persist and regenerate after injury. This recognizes, first and foremost, the work of many talented people over the years in my lab, most of whom have now gone on to their own laboratories at UT Southwestern and other institutions. Many of the key insights for the important discoveries that were made came from them so this really recognizes their work. Id also like to acknowledge all my colleagues, all of you at UT Southwestern and at Childrens Health, for the incredible environment that you created for science, Morrison said via release.

Orth is a Professor of Molecular Biology and Biochemistry and has discovered biochemical mechanisms behind many bacterial infections, revealing how pathogens use host cells for their own benefit. I want to thank you all for this wonderful celebration, even though we have to Zoom . Thanks to this amazing institution, UT Southwestern, the wonderful administration including Drs. (Daniel) Podolsky and (David) Russell and the other administrators and staff. As (Chair of Molecular Biology) Eric Olson said, I have moved up the ranks here, starting as a technician, to a student, a postdoc, and now Professor, Orth said via release. And this path has driven my success. Another major key to my success is all of the talented people that have worked in my lab and my mentors, friends, collaborators, and, of course, my family.

Rosen is the Chair of Biophysics and Professor in the Cecil H. and Ida Green Comprehensive Center for Molecular, Computational, and Systems Biology, and investigates how cells compartmentalize processes without the use of membranes. When we began our work on phase separation about a decade ago, it really was not obvious at all whether this was going to be some weird, esoteric little thing that a few proteins did or (if) it was going to become a more general principle in biology. So it wasa tremendous risk that many of us took in making a move in this new direction. More than anything, I want to thank the various people whojoined me in taking this great risk a decade ago that I think has proved to be very much worthwhile, Rosen said via release.

Schmid is the Professor and Chair of Cell Biology and is recognized for her work on endocytosis, or how cells absorb nutrients and other molecules, including the major pathway for uptake within the cell. Ive been lucky to start and end my academic career at two unique institutions, Schmid said via release. As a PhD student in the early 80s, I was supported and challenged by my peers and faculty in the Biochemistry department at Stanford to ask important questions and do the most impactful research. Over decades, the leadership at UT Southwestern has inspired, supported and celebrated the very best research creating a collegial culture that breeds success.

This important recognition by their peers reflects the breadth and quality of research underway at UT Southwestern, and serves as inspiration for new generations of trainees and scientists to carry on the tradition of discovery that is the hallmark of distinguished academic medical centers, said Dr. W. P. Andrew Lee., Executive Vice President for Academic Affairs, Provost and Dean of UT Southwestern Medical School via release.

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Four UTSW Researchers Named to The National Academy of Sciences - D Magazine

CHMP Grants Positive Opinion for DARZALEX (daratumumab) Subcutaneous Formulation for the Treatment of Patients with Multiple Myeloma – Yahoo Finance

By Stem Cell Medical Center

The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a Positive Opinion recommending approval for DARZALEX (daratumumab) subcutaneous (SC) formulation for the treatment of adult patients with multiple myeloma in frontline and relapsed/refractory settings. The novel SC formulation of daratumumab is co-formulated with recombinant human hyaluronidase PH20 (rHuPH20) [Halozyme's ENHANZE drug delivery technology] and reduces treatment time from hours to approximately three to five minutes, with similar efficacy, and fewer infusion-related reactions compared to intravenous (IV) administration.1,2 The CHMPs Positive Opinion for daratumumab SC formulation applies to all current daratumumab indications including newly diagnosed and transplant-ineligible patients, as well as relapsed or refractory patients.

"Despite therapeutic advances in the treatment of multiple myeloma, the time taken for administration of most intravenous treatments is relatively long and there have been few significant improvements over the years," said Maria-Victoria Mateos, M.D., Ph.D., COLUMBA primary investigator and Director of the Myeloma Unit at University Hospital of Salamanca-IBSAL, Salamanca, Spain. "The daratumumab subcutaneous formulation has the potential to transform the treatment experience for patients and physicians as it reduces time in the chair from hours to minutes, and, because it is administered as a fixed dose from the first treatment, it reduces preparation time and chances of error by eliminating the need for dose calculations."

The Positive Opinion is supported by data from the Phase 3 COLUMBA (MMY3012) and Phase 2 PLEIADES (MMY2040) studies presented at the 2019 American Society of Clinical Oncology (ASCO) Annual Meeting and 62nd American Society of Hematology (ASH) Annual Meeting, respectively.1,2 The COLUMBA presentation included a non-inferiority comparison of daratumumab SC formulation to daratumumab IV formulation for co-primary endpoints of overall response rate and maximum Ctrough concentration.1 Furthermore, in a subsequent paper published in The Lancet Haematology, patient-reported treatment satisfaction scores with daratumumab SC versus daratumumab IV were reported using the modified-Cancer Therapy Satisfaction Questionnaire.3 The PLEIADES study evaluated the daratumumab SC formulation in different combination regimens in patients with newly diagnosed multiple myeloma or with relapsed/refractory disease.2

"The subcutaneous formulation of daratumumab showed similar efficacy and fewer infusion-related reactions compared to intravenous daratumumab, and, overall, patients expressed satisfaction with subcutaneous therapy. If approved, we are hopeful this new formulation could offer improved quality of life for patients with multiple myeloma," said Patrick Laroche, M.D., Haematology Therapy Area Lead, Europe, Middle East and Africa (EMEA), Janssen-Cilag. "Janssen is proud to have developed a new formulation to meet the needs of our patients and continue to make a meaningful difference to the lives of those living with multiple myeloma."

"Since its first European approval in 2016, intravenous daratumumab has been used in the treatment of more than 100,000 patients worldwide and, if approved, both new and existing patients with multiple myeloma will be able to start or switch to the subcutaneous formulation as part of their multiple myeloma daratumumab-based treatment regimens," adds Craig Tendler, M.D., Vice President, Clinical Development and Global Medical Affairs, Oncology at Janssen Research & Development, LLC. "Todays Positive Opinion represents Janssens commitment to continuing to improve the treatment experience for patients living with multiple myeloma."

#ENDS#

In Europe, daratumumab is indicated:4

About the COLUMBA Study (MMY3012)3,5The randomised, open-label, multicentre Phase 3 study included 522 patients with multiple myeloma who had received at least three prior lines of therapy including a proteasome inhibitor (PI) and an immunomodulatory drug (IMiD), or whose disease was refractory to both a PI and an IMiD. In the arm that received the subcutaneously (SC) administered formulation of daratumumab (n=263), patients (median age of 65) received a fixed dose of daratumumab 1,800 milligrams (mg) co-formulated with recombinant human hyaluronidase PH20 (rHuPH20) 2,000 Units per millilitre (U/mL), SC weekly for cycles 1 2, every two weeks for cycles 3 6, and every four weeks for cycle 7 and thereafter. In the daratumumab IV arm (n=259), patients (median age of 67) received daratumumab for intravenous infusion 16 milligrams per kilogram (mg/kg) weekly for cycles 1 2, every two weeks for cycles 3 6, and every four weeks for cycle 7 and thereafter. Each cycle was 28 days. Patients in both treatment arms continued until disease progression or unacceptable toxicity. Co-primary endpoints were overall response rate (ORR) (non-inferiority = 60 percent retention of the lower bound [208%] of the 95% CI of the SIRIUS trial, with relative risk [RR] analysed by Farrington-Manning test) and pre-dose cycle 3, day 1 (C3D1) daratumumab Ctrough (non-inferiority = lower bound of 90 percent confidence interval (CI) for the ratio of the geometric means [GM] 80%).

Story continues

About the PLEIADES Study (MMY2040)6 The non-randomised, open-label, parallel assignment study Phase 2 PLEIADES trial included 240 adults either newly diagnosed or with relapsed or refractory multiple myeloma. Patients with newly diagnosed multiple myeloma were treated with 1,800 mg of the subcutaneous formulation in combination with either bortezomib, lenalidomide and dexamethasone (D-VRd) or bortezomib, melphalan and prednisone (D-VMP). Patients with relapsed or refractory disease were treated with 1,800 mg of the subcutaneous formulation plus lenalidomide and dexamethasone (D-Rd). The primary endpoint for the D-VMP and D-Rd cohorts was overall response rate. The primary endpoint for the D-VRd cohort was very good partial response or better rate. An additional cohort of patients with relapsed and refractory multiple myeloma treated with daratumumab plus carfilzomib and dexamethasone was subsequently added to the study.

About daratumumabDaratumumab is a first-in-class7 biologic targeting CD38, a surface protein that is highly expressed across multiple myeloma cells, regardless of disease stage.8 Daratumumab is believed to induce tumour cell death through multiple immune-mediated mechanisms of action, including complement-dependent cytotoxicity (CDC), antibody-dependent cell-mediated cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP), as well as through apoptosis, in which a series of molecular steps in a cell lead to its death.4 A subset of myeloid derived suppressor cells (CD38+ MDSCs), CD38+ regulatory T cells (Tregs) and CD38+ B cells (Bregs) were decreased by daratumumab.4 Since launch, it is estimated that 100,000 patients have been treated with daratumumab worldwide.9 Daratumumab is being evaluated in a comprehensive clinical development programme across a range of treatment settings in multiple myeloma, such as in frontline and relapsed settings.10,11,12,13,14,15,16,17 Additional studies are ongoing or planned to assess its potential in other malignant and pre-malignant haematologic diseases in which CD38 is expressed, such as smouldering myeloma.18,19 For more information, please see https://www.clinicaltrials.gov/.

For further information on daratumumab, please see the Summary of Product Characteristics at https://www.ema.europa.eu/en/medicines/human/EPAR/darzalex.

In August 2012, Janssen Biotech, Inc. and Genmab A/S entered a worldwide agreement, which granted Janssen an exclusive licence to develop, manufacture and commercialise daratumumab.20

About Multiple MyelomaMultiple myeloma (MM) is an incurable blood cancer that starts in the bone marrow and is characterised by an excessive proliferation of plasma cells.21 In Europe, more than 48,200 people were diagnosed with MM in 2018, and more than 30,800 patients died.22 Almost 60 percent of patients with MM do not survive more than five years after diagnosis.23

Although treatment may result in remission, unfortunately, patients will most likely relapse as there is currently no cure.24 Refractory MM is when a patients disease progresses within 60 days of their last therapy.25,26 Relapsed cancer is when the disease has returned after a period of initial, partial or complete remission.27 While some patients with MM have no symptoms at all, most patients are diagnosed due to symptoms that can include bone problems, low blood counts, calcium elevation, kidney problems or infections.28 Patients who relapse after treatment with standard therapies, including proteasome inhibitors and immunomodulatory agents, have poor prognoses and few treatment options available.29

About the Janssen Pharmaceutical Companies of Johnson & JohnsonAt Janssen, were creating a future where disease is a thing of the past. Were the Pharmaceutical Companies of Johnson & Johnson, working tirelessly to make that future a reality for patients everywhere by fighting sickness with science, improving access with ingenuity, and healing hopelessness with heart. We focus on areas of medicine where we can make the biggest difference: Cardiovascular & Metabolism, Immunology, Infectious Diseases & Vaccines, Neuroscience, Oncology, and Pulmonary Hypertension.

Learn more at http://www.janssen.com/emea. Follow us at http://www.twitter.com/janssenEMEA for our latest news. Janssen-Cilag, Janssen Research & Development, LLC and Janssen Biotech, Inc. are part of the Janssen Pharmaceutical Companies of Johnson & Johnson.

Cautions Concerning Forward-Looking StatementsThis press release contains "forward-looking statements" as defined in the Private Securities Litigation Reform Act of 1995 regarding the benefits of daratumumab for the treatment of patients with multiple myeloma. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialise, actual results could vary materially from the expectations and projections of Janssen Pharmaceutical Companies and/or Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; manufacturing difficulties and delays; competition, including technological advances, new products and patents attained by competitors; challenges to patents; product efficacy or safety concerns resulting in product recalls or regulatory action; changes in behaviour and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson's Annual Report on Form 10-K for the fiscal year ended December 29, 2019, including in the sections captioned "Cautionary Note Regarding Forward-Looking Statements" and "Item 1A. Risk Factors," and in the companys most recently filed Quarterly Report on Form 10-Q, and the companys subsequent filings with the Securities and Exchange Commission. Copies of these filings are available online at http://www.sec.gov, http://www.jnj.com or on request from Johnson & Johnson. None of the Janssen Pharmaceutical Companies nor Johnson & Johnson undertakes to update any forward-looking statement as a result of new information or future events or developments.

ENHANZE is a registered trademark of Halozyme.

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References

1 Mateos MV, Nahi H, Legiec W, et al. Efficacy and safety of the randomized, open-label, non-inferiority, phase 3 study of subcutaneous (SC) versus intravenous (IV) daratumumab (DARA) administration in patients (pts) with relapsed or refractory multiple myeloma (RRMM): COLUMBA. J Clin Oncol. 2019;37(Suppl.): abstract 8005.2 Chari A, San-Miguel J, McCarthy H, et al. Subcutaneous daratumumab plus standard treatment regimens in patients with multiple myeloma across lines of therapy: Pleiades study update. Blood. 2019;134(Suppl.1):abstract 3152.3 Mateos MV, Nahi H, Legiec W, et al. Subcutaneous versus intravenous daratumumab in patients with relapsed or refractory multiple myeloma (COLUMBA): a multicentre, open-label, non-inferiority, randomised, phase 3 trial. Lancet Haematol. 2020 Mar 23 [epub ahead of print].4 European Medicines Agency. DARZALEX summary of product characteristics. Available at:https://www.ema.europa.eu/documents/product-information/darzalex-epar-productinformation_en.pdf Last accessed April 2020.5 ClinicalTrials.gov. A Study of Subcutaneous Versus (vs.) Intravenous Administration of Daratumumab in Participants With Relapsed or Refractory Multiple Myeloma. NCT03277105. Available at: https://clinicaltrials.gov/ct2/show/NCT03277105 Last accessed April 2020.6 ClinicalTrials.gov. A Study to Evaluate Subcutaneous Daratumumab in Combination With Standard Multiple Myeloma Treatment Regimens. NCT03412565. Available at: https://clinicaltrials.gov/ct2/show/NCT03412565 Last accessed April 2020.7 Sanchez L, Wang Y, Siegel DS, Wang ML. Daratumumab: a first-in-class CD38 monoclonal antibody for the treatment of multiple myeloma. J Hematol Oncol. 2016;9:51.8 Fedele G, di Girolamo M, Recine U, et al. CD38 ligation in peripheral blood mononuclear cells of myeloma patients induces release of protumorigenic IL-6 and impaired secretion of IFNgamma cytokines and proliferation. Mediat Inflamm. 2013;2013:564687.9 [Data on file]. DARZALEX: New Patient Starts Launch to Date. RF-8220310 ClinicalTrials.gov. A study to evaluate daratumumab in transplant eligible participants with previously untreated multiple myeloma (Cassiopeia). NCT02541383. Available at: https://clinicaltrials.gov/ct2/show/NCT02541383 Last accessed April 202011 ClinicalTrials.gov. A study comparing daratumumab, lenalidomide, and dexamethasone with lenalidomide and dexamethasone in relapsed or refractory multiple myeloma. NCT02076009. Available at: https://clinicaltrials.gov/ct2/show/NCT02076009 Last accessed April 2020.12 ClinicalTrials.gov. Addition of daratumumab to combination of bortezomib and dexamethasone in participants with relapsed or refractory multiple myeloma. NCT02136134. Available at: https://clinicaltrials.gov/ct2/show/NCT02136134 Last accessed April 2020.13 ClinicalTrials.gov. A study of combination of daratumumab and Velcade (bortezomib) melphalan-prednisone (DVMP) compared to Velcade melphalan-prednisone (VMP) in participants with previously untreated multiple myeloma. NCT02195479. Available at: https://clinicaltrials.gov/ct2/show/NCT02195479 Last accessed April 2020.14 ClinicalTrials.gov. Study comparing daratumumab, lenalidomide, and dexamethasone with lenalidomide and dexamethasone in participants with previously untreated multiple myeloma. NCT02252172. Available at: https://clinicaltrials.gov/ct2/show/NCT02252172 Last accessed April 2020.15 ClinicalTrials.gov. A study of Velcade (bortezomib) melphalan-prednisone (VMP) compared to daratumumab in combination with VMP (D-VMP), in participants with previously untreated multiple myeloma who are ineligible for high-dose therapy (Asia Pacific region). NCT03217812. Available at: https://clinicaltrials.gov/ct2/show/NCT03217812 Last accessed April 2020.16 ClinicalTrials.gov. Comparison of pomalidomide and dexamethasone with or without daratumumab in subjects with relapsed or refractory multiple myeloma previously treated with lenalidomide and a proteasome inhibitor daratumumab/pomalidomide/dexamethasone vs pomalidomide/dexamethasone (EMN14). NCT03180736. Available at: https://clinicaltrials.gov/ct2/show/NCT03180736 Last accessed April 2020.17 ClinicalTrials.gov. Study of carfilzomib, daratumumab and dexamethasone for patients with relapsed and/or refractory multiple myeloma (CANDOR). NCT03158688. Available at: https://clinicaltrials.gov/ct2/show/NCT03158688 Last accessed April 2020.18 ClinicalTrials.gov. A study to evaluate 3 dose schedules of daratumumab in participants with smoldering multiple myeloma. NCT02316106. Available at: https://clinicaltrials.gov/ct2/show/NCT02316106 Last accessed April 2020.19 ClinicalTrials.gov. An efficacy and safety proof of concept study of daratumumab in relapsed/refractory mantle cell lymphoma, diffuse large B-cell lymphoma, and follicular lymphoma. NCT02413489. Available at: https://clinicaltrials.gov/ct2/show/NCT02413489 Last accessed April 2020.20 Johnson & Johnson. Janssen Biotech announces global license and development agreement for investigational anti-cancer agent daratumumab. Press release August 30, 2012. Available at: https://www.jnj.com/media-center/press-releases/janssen-biotech-announces-global-license-and-development-agreement-for-investigational-anti-cancer-agent-daratumumab Last accessed April 2020.21 American Society of Clinical Oncology. Multiple myeloma: introduction. Available at: https://www.cancer.net/cancer-types/multiple-myeloma/introduction Last accessed April 2020.22 GLOBOCAN 2018. Cancer Today Population Factsheets: Europe Region. Available at: https://gco.iarc.fr/today/data/factsheets/populations/908-europe-fact-sheets.pdf Last accessed April 2020.23 De Angelis R, Minicozzi P, Sant M, et al. Survival variations by country and age for lymphoid and myeloid malignancies in Europe 2000-2007: results of EUROCARE-5 population-based study. Eur J Cancer. 2015;51:2254-68.24 Abdi J, Chen G, Chang H, et al. Drug resistance in multiple myeloma: latest findings and new concepts on molecular mechanisms. Oncotarget. 2013;4:2186207.25 National Cancer Institute. NCI dictionary of cancer terms: refractory. Available at: https://www.cancer.gov/publications/dictionaries/cancer-terms?CdrID=350245 Last accessed April 2020.26 Richardson P, Mitsiades C, Schlossman R, et al. The treatment of relapsed and refractory multiple myeloma. Hematology Am Soc Hematol Educ Program. 2007:317-23.27 National Cancer Institute. NCI dictionary of cancer terms: relapsed. Available at: https://www.cancer.gov/publications/dictionaries/cancer-terms?CdrID=45866 Last accessed April 2020.28 American Cancer Society. Multiple myeloma: early detection, diagnosis and staging. Available at: https://www.cancer.org/content/dam/CRC/PDF/Public/8740.00.pdf Last accessed April 2020.29 Kumar SK, Lee JH, Lahuerta JJ, et al. Risk of progression and survival in multiple myeloma relapsing after therapy with IMiDs and bortezomib: a multicenter international myeloma working group study. Leukemia. 2012;26:149-57.

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CHMP Grants Positive Opinion for DARZALEX (daratumumab) Subcutaneous Formulation for the Treatment of Patients with Multiple Myeloma - Yahoo Finance

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