Families of Spinal Muscular Atrophy and Repligen partner to develop a treatment for SMA

Families of Spinal Muscular Atrophy (FSMA) announced today that it has entered into a groundbreaking exclusive license agreement with Repligen Corporation for the development of a potential treatment of Spinal Muscular Atrophy (SMA).

SMA, the leading genetic killer of children under the age of two, is typically marked by the degeneration of muscle movement including the muscles that control crawling, walking, swallowing or breathing. There are no approved therapies for the treatment of SMA, which affects one in every 6,000 babies. One in every 40 people carries the gene that causes SMA, indicating approximately 7.5 million carriers in the United States.

Families of SMA was started by a small group of parents in 1984 who wanted to raise funds for SMA research and support all affected families. FSMA has funded $50 million for SMA research, raised from individual donations and through fundraising events held by volunteer families and Chapters.

FSMA made investments of $13 million during the last decade to bring this specific program to the cusp of clinical development. Through FSMA's leadership, the research has resulted in a drug candidate that treats the underlying cause of SMA. In preclinical studies, the drug has been shown to efficiently cross the blood brain barrier - a critical feature for a neurological drug - and prolong survival significantly in two different mouse models of SMA.

Often an "Orphan Disease," such as SMA, will not be "adopted" by the pharmaceutical industry because there is limited financial incentive to make new medications to treat smaller patient populations. This license agreement marks a significant milestone for the SMA community by securing the commitment of an industrial partner to develop potential treatments through the highly expensive human clinical trial phases.

"At this point in the program, joining forces with a corporate partner to advance into clinical studies is the best way to meet our objective of accelerating drug development for SMA," said Kenneth Hobby, President of Families of SMA. "Repligen is an ideal partner for this program with the necessary resources and expertise to invest in and focus on successfully developing this new treatment for SMA. We are very excited to partner with Repligen and look forward to making our combined efforts successful in delivering an effective treatment to our patients."

"Families of SMA has made remarkable progress in defining a series of highly potent compounds which may be clinical candidates for SMA," stated Walter C. Herlihy, President and Chief Executive Officer of Repligen Corporation. "We look forward to working with FSMA and their collaborators in the development of what we hope will be an important new treatment for SMA."

Through the agreement with Repligen, Families of SMA has the potential to recover its investments through a series of milestone payments if the program successfully progresses through clinical stages and eventually reaches market approval.


Families of Spinal Muscular Atrophy


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