Sarepta Therapeutics Announces Partnership with Roche

Sarepta Therapeutics Announces Partnership with Roche in Territories Outside the United States for its Investigational Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy, SRP-9001

Press release here

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  • Roche obtains the exclusive right to launch and commercialize SRP-9001 outside the United States 
  • At closing, Sarepta will receive an upfront payment of $1.15 billion, comprising $750 million in cash and $400 million in Sarepta stock, priced at $158.59 per share of common stock 
  • Additionally, Sarepta is eligible to receive up to $1.7 billion in regulatory and sales milestones, plus royalties on net sales 
  • Sarepta will continue to be responsible for clinical development and manufacturing of SRP-9001 with global clinical development costs shared equally with Roche 

Sarepta Therapeutics, Inc. announced that Sarepta and Roche have entered into a licensing agreement providing Roche exclusive commercial rights to SRP-9001 (AAVrh74.MHCK7.micro-dystrophin), Sarepta’s investigational gene therapy for Duchenne muscular dystrophy (DMD), outside the United States. Under the agreement, Sarepta will receive $1.15 billion in an upfront payment and an equity investment; up to $1.7 billion in regulatory and sales milestones; and royalties on net sales, anticipated to be in the mid-teens. In addition, Roche and Sarepta will equally share global development expenses. Sarepta retains all rights to SRP-9001 in the United States.

The collaboration combines Sarepta’s leading gene therapy candidate for DMD with Roche’s global reach, commercial presence and regulatory expertise to accelerate access to SRP-9001 for patients outside the United States. DMD is an X-linked rare degenerative neuromuscular disorder causing severe progressive muscle loss and premature death. SRP-9001, currently in clinical development for DMD, is designed to deliver the micro-dystrophin-encoding gene directly to the muscle tissue for the targeted production of the micro-dystrophin protein.ç

 

Doug Ingram, president and chief executive officer, Sarepta – “As a mission-driven organization, we are inspired to partner with Roche with the goal of bringing SRP-9001 to patients outside the United States. This collaboration will not only increase the speed with which SRP-9001 could benefit DMD patients outside the United States but will also greatly expand the scope of territories within which we could potentially launch SRP-9001 and improve and save lives. In addition to the validation that comes from joining forces with Roche, this licensing agreement – one of the most significant ex-U.S. licensing transactions in biopharma – will provide Sarepta with the resources and focus to accelerate our gene therapy engine and, if successful, bring SRP-9001 to patients as quickly as possible, potentially transforming the lives of countless DMD patients across the globe.”

 

James Sabry, Head of Roche Pharma Partnering –  “We are excited to enter this licensing agreement with Sarepta. By working together to provide SRP-9001 to patients, we hope to fundamentally transform the lives of patients and families living with this devastating disorder for which there are currently only limited treatment options.”

As part of the agreement, Sarepta will continue to be responsible for the global development plan and manufacturing build-out for SRP-9001. Through its leading hybrid manufacturing platform, Sarepta will remain responsible for manufacturing of clinical and commercial supplies. Sarepta has also granted Roche an option to acquire ex-U.S. rights to certain future DMD-specific programs, in exchange for separate milestone and royalty considerations, and cost-sharing.

About Sarepta

Sarepta Therapeutics, Inc., a biopharmaceutical company, is working to unlock the potential of RNA-based and gene therapy technologies for the treatment of serious and life-threatening diseases like Duchenne muscular dystrophy (DMD). Sarepta’s primary focus is to rapidly advance new treatments for DMD.

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Sarepta Therapeutics, Inc.

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FDA grants accelerated approval to Vyondys 53

Sarepta Therapeutics Announces FDA Approval of VYONDYS 53™ (golodirsen) Injection for the Treatment of Duchenne Muscular Dystrophy (DMD) in Patients Amenable to Skipping Exon 53

Source: Sarepta Therapeutics, Inc., Dec 12, 2019 – Read the original news here– 

The U.S. Food and Drug Administration granted accelerated approval to Vyondys 53 (golodirsen) injection to treat Duchenne muscular dystrophy (DMD) patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping. It is estimated that about 8% of patients with DMD have this mutation.

 

Billy Dunn, M.D., acting director of the Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research – “The FDA recognizes the urgent need for new medical treatments for serious neurological disorders and we have a long-standing commitment to working with researchers, drug companies and patients to facilitate the development and approval of treatments for rare diseases. With today’s accelerated approval, patients with Duchenne muscular dystrophy who have a confirmed mutation of the dystrophin gene amenable to exon 53 skipping will now have available the first treatment targeted specifically for this disease subtype. Use of the accelerated approval pathway will make Vyondys 53 available to patients based on initial data and we look forward to learning more about the drug’s clinical benefit from the ongoing confirmatory clinical trial.”

 

Doug Ingram, president and chief executive officer, Sarepta – “Today is monumental for Sarepta and, more importantly, for the DMD community. VYONDYS 53, our second approved exon-skipping RNA therapy for DMD, may treat up to 8% of the DMD community, representing those patients who have a confirmed exon 53 amenable mutations.  Along with EXONDYS 51® (eteplirsen), we now offer treatment options for approximately 20% of those with DMD in the U.S.”

 

Pat Furlong, founding president and chief executive officer, Parent Project Muscular Dystrophy (PPMD link here) – “With the approval of VYONDYS 53, up to another 8% of Duchenne families will have a therapy to treat this devastating disease. For 25 years, PPMD has been working with researchers, clinicians, industry, and the Duchenne community to find treatments for all people living with Duchenne. And while we need to ensure that these approved therapies are accessible for patients, today we celebrate this approval and thank Sarepta for their continued leadership in the fight to end Duchenne.”

 

More about Vyondys 53 (golodirsen)

Like Exondys 51, golodirsen, which Sarepta hopes to sell under the name Vyondys 53, is designed to treat a group of Duchenne patients with a particular type of mutation. Exondys 51 works for about 13% of DMD patients—those whose disease is amenable to exon 51 skipping. If approved, golodirsen would offer treatment to patients with a mutation in exon 53—about 8% of the DMD population.

VYONDYS 53 is priced at parity to EXONDYS 51, the price of which has not increased since its launch in 2016. Patients and physicians can access more information at www.SareptAssist.com or by calling 1-888-727-3782.

Important Safety Information for VYONDYS 53

Approval of Vyondys 53

Vyondys 53 was approved under the accelerated approval pathway, which provides for the approval of drugs that treat serious or life-threatening diseases and generally offer a meaningful advantage over existing treatments. Approval under this pathway can be based on adequate and well-controlled studies showing the drug affects a surrogate endpoint that is reasonably likely to predict clinical benefit to patients. This pathway provides earlier patient access to promising new drugs while the company conducts clinical trials to verify the predicted clinical benefit.

The accelerated approval of Vyondys 53 is based on the surrogate endpoint of an increase in dystrophin production in the skeletal muscle observed in some patients treated with the drug. The FDA has concluded that the data submitted by the applicant demonstrated an increase in dystrophin production that is reasonably likely to predict clinical benefit in patients with DMD who have a confirmed mutation of the dystrophin gene amenable to exon 53 skipping. A clinical benefit of the drug, including improved motor function, has not been established. In making this decision, the FDA considered the potential risks associated with the drug, the life-threatening and debilitating nature of the disease and the lack of available therapy. Read more here.

The Status

  • VYONDYS 53 is approved under accelerated review based on an increase in dystrophin production in skeletal muscle of patients amenable to exon 53 skipping. Continued approval may be contingent upon verification of a clinical benefit in confirmatory trials.
  • VYONDYS 53 has met the full statutory standards for safety and effectiveness and as such is not considered investigational or experimental.
  • The commercial distribution of VYONDYS 53 in the U.S. will commence immediately
  • Information for patients and clinicians is available at www.SareptAssist.com

What is exon skipping?

Mutations in the dystrophin gene are one cause of DMD. Most commonly, one or more exons (a portion of a gene) are missing, and the remaining exons don’t fit together correctly. (Think of a zipper that doesn’t work properly, because teeth are missing.)

Because of this error, cells cannot make the dystrophin protein that muscles need to work properly. Without it, muscle cells become damaged and, over time, are replaced with scar tissue and fat.

To fix the broken genetic machinery, scientists are developing drugs that skip over parts that contain missing or defective exons. In this way, the machinery can produce a less imperfect dystrophin protein, which may improve muscle function in children with exon mutations. > Pipeline exon-skipping

What about Canada?

At this moment, Vyondys 53 is not available in Canada. We hope that shortly, Sarepta Therapeutics will file a request for marketing approval with Health Canada.

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DEFEAT DUCHENNE FAMILY FORUM – MONTREAL

We are excited to share that the next Defeat Duchenne Family Forum will take place in Montréal, Québec, on Saturday, May 2, 2020.

Presented by PTC Therapeutics and in partnership with Jesse’s Journey, this unique educational event provides the opportunity for you – families and caregivers navigating the Duchenne journey, to come together with researchers, clinicians, and industry professionals for a day of education and inspiration. More than that, the Defeat Duchenne Family Forum provides a support network. A connection opportunity for you to know that they are not alone and together, we can defeat Duchenne. Registration is free, and the day includes take-away resources and all meals (breakfast, lunch, and snacks). Event page here + Facebook event page here

  • What: Defeat Duchenne Family Forum
  • When: Saturday, May 2, 2020 | 9 a.m. – 4 p.m
  • Followed by: Social & Vendor Fair from 4 – 6 p.m
  • Where: Shriners Hospital for Children (1003 Décarie Blvd, Montréal, Québec H4A 0A9)

Stay tuned as we announce registration and full program details in early 2020.

 

The first-ever Duchenne Family Forum In London, Ontario

May 25, 2019, in London, Ontario

This unique event – tailored to families in Canada affected by Duchenne muscular dystrophy – featured updates about the latest research, clinical trials recruiting in Canada, and more. Read more here.

Defeat Duchenne Family Forum in Calgary, Alberta

November 2, 2019, in Calgary, Alberta

More than 75 Canadians came together for the inaugural Defeat Duchenne Family Forum presented by PTC Therapeutics on Saturday, November 2, 2019, in Calgary, Alberta. Families from across Western Canada affected by Duchenne muscular dystrophy came together with world-renowned researchers, clinicians, and industry professionals for a day of education, inspiration, and hope. Read more here.

More links

Learn more about Jesse’s Journey here.

Watch our video about John Davidson, the godfather of the Canadian Duchenne community here.