Ultragenyx and Solid Biosciences Announce Strategic Collaboration to Develop and Commercialize New Gene Therapies for Duchenne Muscular Dystrophy

Collaboration combines Solid’s differentiated microdystrophin construct and Ultragenyx’s HeLa PCL manufacturing platform for use with AAV8 and variants

Solid receives $40 million upfront via equity investment at a premium; up to $255 million in milestones plus royalty payments

Solid retains exclusive rights to all other uses of its microdystrophins, including its existing SGT-001 program

NOVATO, Calif. and CAMBRIDGE, Mass., Oct. 23, 2020 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (Nasdaq: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare diseases, and Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company focused on advancing meaningful therapies for Duchenne muscular dystrophy (Duchenne), today announced a strategic collaboration and license agreement to focus on the development and commercialization of new gene therapies for Duchenne. The parties will collaborate to develop products that combine Solid’s differentiated microdystrophin construct, Ultragenyx’s HeLa producer cell line (PCL) manufacturing platform, and AAV8 variants. The collaboration also brings together Solid’s expertise in muscle biology and Ultragenyx’s expertise in bringing novel therapies to patients with rare diseases.

Under the terms of the collaboration, Solid granted Ultragenyx an exclusive license for any pharmaceutical product that expresses Solid’s proprietary microdystrophin construct from AAV8 and variants thereof in clade E for use in the treatment of Duchenne and other diseases resulting from lack of functional dystrophin, including Becker muscular dystrophy. Ultragenyx has made a $40 million investment in Solid and has agreed to pay up to $255 million in cumulative milestone payments per product upon achievement of specified milestone events, and tiered royalties on worldwide net sales at low double digit to mid-teens percentages. Upon achievement of proof-of-concept, Solid has the right to opt-in to co-fund collaboration programs in return for participation in a profit share or increased royalty payments.

“We believe that Solid’s microdystrophin is best-in-class with its unique neuronal nitric oxide synthase binding domain,” said Emil D. Kakkis, MD, PhD, Chief Executive Officer and President of Ultragenyx. “By using an AAV8 variant validated in prior human and other studies combined with our scalable, efficient HeLa producer cell line platform, we believe we can leverage our mutual strengths to develop a high-quality AAV-based treatment alternative for Duchenne.”

“Ultragenyx has a demonstrated track record of success in developing and commercializing innovative therapies for rare diseases,” said Ilan Ganot, Co-Founder, President and Chief Executive Officer at Solid Biosciences. “We believe it is the partner of choice for exploring new gene therapy opportunities for patients with Duchenne.”

Solid’s proprietary microdystrophin construct has exhibited functional benefit in preclinical models. In preclinical studies, animals expressing a microdystrophin capable of restoring neuronal nitric oxide synthase (nNOS) resisted fatigue better than those expressing a microdystrophin that does not. Patients dosed with Solid’s proprietary microdystrophin construct at the 2E14 vg/kg dose in Solid’s ongoing IGNITE DMD clinical trial have also preliminarily demonstrated nNOS activity and function, further validating these preclinical results. Solid expects to dose the next patient in the IGNITE DMD clinical

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Roche announces collaboration with Atea Pharmaceuticals to develop a potential oral treatment for COVID-19 patients

  • Roche and Atea partner to jointly develop AT-527, an orally administered direct-acting antiviral (DAA) currently in Phase 2 clinical trials

  • AT-527 has the potential to be the first novel oral antiviral to treat COVID-19 patients outside the hospital setting as well as in the hospital and may also be used in post-exposure prophylactic settings

  • Oral, small-molecule DAAs for COVID-19 patients allow for large-scale manufacturing and facilitate broad patient access

  • If approved, Atea will distribute AT-527 in the United States and Roche will be responsible for global manufacturing and distribution outside the United States

Basel, 22 October 2020 – Roche (SIX: RO, ROG; OTCQX: RHHBY) and Atea Pharmaceuticals, Inc. announced today that they are joining forces in the fight against COVID-19 to develop, manufacture and distribute AT-527, Atea’s investigational oral direct-acting antiviral, to people around the globe. AT-527 acts by blocking the viral RNA polymerase enzyme needed for viral replication, and is currently being studied in a Phase 2 clinical trial for hospitalised patients with moderate COVID-19. A Phase 3 clinical trial, expected to start in Q1 2021, will explore the potential use in patients outside of the hospital setting. In addition, AT-527 may be developed for post-exposure prophylactic settings.

AT-527, while being a potential oral treatment option for hospitalised patients, also holds the potential to be the first oral treatment option for COVID-19 patients that are not hospitalised.  Additionally, the manufacturing process of small-molecule DAAs allows the ability to produce large quantities of a much needed treatment. If successful, AT-527 could help treat patients early, reduce the progression of the infection, and contribute to decreasing the overall burden on health systems.

The collaboration aims to accelerate the clinical development and manufacturing of AT-527, to investigate its safety and efficacy, and to provide this potential treatment option to patients around the world as quickly as possible. If AT-527 proves safe and effective in clinical trials and regulatory approvals are granted, Atea will be responsible for distributing this treatment option in the U.S, with the option to request Genentech’s support, and Roche will be responsible for distribution outside the United States.

“The ongoing complexities of COVID-19 require multiple lines of defence. By joining forces with Atea, we hope to offer an additional treatment option for hospitalised and non-hospitalised COVID-19 patients, and to ease the burden on hospitals during a global pandemic.” said Bill Anderson, Chief Executive Officer of Roche Pharmaceuticals. “In jointly developing and manufacturing AT-527 at scale, we seek to make this treatment option available to as many people around the world as we possibly can.”

“Roche shares our passion for delivering innovative new medicines to address great unmet medical needs. The COVID-19 pandemic has highlighted the urgent need for a novel, oral antiviral to treat this highly infectious and often deadly virus,” said Jean-Pierre Sommadossi, Ph.D., Chief Executive Officer and Founder of Atea Pharmaceuticals. “AT-527 is expected to be ideally suited to combat COVID-19 as it inhibits viral replication by interfering with viral RNA polymerase, a key component in the

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ImmunoGen and Huadong Medicine Announce Strategic Collaboration to Develop and Commercialize Mirvetuximab Soravtansine in Greater China

Partnership Accelerates Development Path for Mirvetuximab in Greater China and Expands Huadong Medicine’s Oncology Portfolio with Innovative ADC

Combines ImmunoGen’s Lead Clinical Program with Huadong’s Regional Oncology Expertise

ImmunoGen to Receive $40 Million Upfront Payment and is Eligible to Receive Up to $265 Million in Potential Development, Regulatory, and Commercial Milestone Payments

ImmunoGen, Inc. (Nasdaq: IMGN), a leader in the expanding field of antibody-drug conjugates (ADCs) for the treatment of cancer, and Hangzhou Zhongmei Huadong Pharmaceutical Co., Ltd., a wholly-owned subsidiary of Huadong Medicine Co., Ltd., today announced that the companies have entered into an exclusive collaboration to develop and commercialize mirvetuximab soravtansine in mainland China, Hong Kong, Macau, and Taiwan (Greater China). ImmunoGen will retain all rights to mirvetuximab in the rest of the world.

This collaboration provides ImmunoGen with access to the second largest pharmaceutical market in the world via Huadong Medicine’s development, regulatory, and commercial capabilities, while supporting Huadong Medicine’s growth strategy to build a deep portfolio of oncology, endocrinology, and autoimmunology candidates. Mirvetuximab adds a compelling late-stage oncology asset to Huadong Medicine’s portfolio.

“With extensive regional experience, the right development and regulatory capabilities, and access to a deep local network of hospitals and clinics across Greater China, Huadong Medicine is an ideal partner for us,” said Mark Enyedy, ImmunoGen’s President and Chief Executive Officer. “This collaboration reflects mirvetuximab’s potential to deliver meaningful value to ovarian cancer patients as well as our ability to translate our work in ADCs into long-term relationships that create sustainable value for ImmunoGen and our partners. We look forward to working closely with Huadong Medicine to develop and commercialize mirvetuximab in Greater China as we advance the mirvetuximab program and prepare for the first potential commercial launch in the United States in 2022.”

“ImmunoGen is a leader in the development of ADCs for the treatment of cancer and this partnership provides us with a late-stage asset that will enable us to further expand our pipeline of innovative oncology programs,” said Liang Lu, Chairman of Huadong Medicine. “The compelling clinical data generated to date highlights mirvetuximab’s potential to be a promising therapy for an extremely difficult to treat disease and we look forward to beginning its development as we seek to meet the growing needs of ovarian cancer patients in Greater China.”

Under the terms of the agreement, ImmunoGen will receive an upfront payment of $40 million and is eligible to receive additional milestone payments of up to $265 million as certain development, regulatory, and commercial objectives are achieved. ImmunoGen is also eligible to receive low double digit to high teen royalties as a percentage of mirvetuximab commercial sales by Huadong Medicine in Greater China.

Huadong Medicine will be responsible for the development as well as potential regulatory submissions and commercialization of mirvetuximab in Greater China pursuant to input from a joint steering committee comprised of individuals from both companies. Huadong Medicine will also have the opportunity to participate in global clinical studies of mirvetuximab conducted by ImmunoGen. ImmunoGen will continue

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