Pfizer Licenses Families of Spinal Muscular Atrophy Quinazoline Drug Program From Repligen

Pfizer Licenses Families of Spinal Muscular Atrophy Quinazoline Drug Program
From Repligen

ELK GROVE VILLAGE, Ill., Jan. 3, 2013 (GLOBE NEWSWIRE) -- Repligen Corporation
announced today that it has entered into an exclusive worldwide licensing
agreement with Pfizer Inc. to advance Repligen's spinal muscular atrophy (SMA)
program, originally in-licensed from Families of SMA (FSMA).

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/RG3039 program in 2000 at the very initial stages
of drug development, when risk is the highest. It was the very first
industrial drug program for SMA ever conducted. The direction from FSMA
provided the positive results necessary to license the program to Repligen and
now Pfizer to leverage resources and expertise for clinical development.

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

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's work has produced major discoveries, including identification
of the underlying cause and a back-up gene for the disease, which provides a
clearly defined target for disease altering therapies. The organization is
also dedicated to supporting SMA families through networking, information and
services and to improving care for all SMA patients.

Please contact Kenneth Hobby, President, at or call (800)
886-1762 if you have any questions.

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