Families of SMA funded and directed the preclinical development of RG3039 with an investment of more than $13 million. This was the first drug discovery program ever conducted specifically for SMA. Repligen licensed RG3039 in 2009 from Families of SMA.
“We believe this collaboration with Pfizer, a leading pharmaceutical company with specialized efforts in orphan and genetic diseases, has the potential to accelerate the development of therapies for SMA,” said Walter C. Herlihy, Ph.D., President and Chief Executive Officer of Repligen.
“There is a critical need to expedite potential treatment solutions for rare diseases such as spinal muscular atrophy, where patients have such limited options,” said Jose Carlos Gutierrez-Ramos, Senior Vice President, Pfizer BioTherapeutics R&D. “This partnership will combine our expert capabilities in advancing molecules for genetic diseases with Repligen’s leading SMA program.”
“This licensing deal demonstrates the innovative collaborations that Families of SMA has successfully implemented between non-profit, biotech and big pharma,” stated Jill Jarecki, Ph.D., Research Director for Families of SMA. “These partnerships are critical for the development of new treatments for an orphan disease such as SMA. We are extremely pleased to see Pfizer taking the lead on the development and commercialization of the SMA program, following Repligen’s development work and FSMA’s original investment.”
FSMA began the Quinazoline/
Through FSMA’s leadership and research funding of $13 million, a drug candidate has been created 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.
The FSMA research model is to fund early stage drug discovery programs for SMA and then partner with companies for later stages and accelerated clinical development. At the earliest stages of drug development, programs have less than a 1% chance of success. This inherent risk along with small patient populations has traditionally hindered industry from working on orphan diseases.
This new license agreement with Pfizer marks a significant advance for the SMA community by securing the commitment of one of the worlds largest pharmaceutical companies to develop potential treatments for the disease.
The SMA program includes RG3039, a small molecule drug candidate in clinical development for SMA, as well as backup compounds and enabling technologies. Under the terms of the agreement, Repligen is responsible for completing the first two cohorts of an active Phase 1 trial evaluating RG3039 in healthy volunteers, which it anticipates will occur during the first quarter of 2013. Repligen will also provide certain technology transfer services to Pfizer who will then assume full responsibility for the SMA program moving forward, including the conduct of any registration trials necessary for product approval. Repligen has previously received U.S. Orphan Drug and Fast Track designations for RG3039 for the treatment of SMA, as well as Orphan Medicinal Product designation in the EU.
Families of SMA
Families of SMA is the world’s leader focused on funding SMA research to develop a treatment and cure for the disease. The successful results and progress that the organization has delivered, from basic research to drug discovery to clinical trials, provide real hope for families and patients impacted by the disease. The charity has invested over $55 million in research and has been involved in funding half of all the ongoing novel drug programs for SMA. Families of SMA is a nonprofit 501(c)3 organization, with 31 Chapters and 90,000 members and supporters throughout the United States. The organization’