Repligen Announces Licensing Agreement with Pfizer for Spinal Muscular Atrophy Program

  Repligen Announces Licensing Agreement with Pfizer for Spinal Muscular
  Atrophy Program

Business Wire

WALTHAM, Mass. -- January 3, 2013

Repligen Corporation (NASDAQ:RGEN) 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). 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 entitled to
receive up to $70 million from Pfizer, commencing with an upfront payment of
$5 million and total potential future milestone payments of up to $65 million
as well as royalties on any future sales of SMA compounds developed under the
agreement. SMA is an orphan neurodegenerative genetic disease that presents
early in life.

“This agreement is consistent with the strategic decision we announced in
August 2012 to focus Repligen’s internal efforts on the growth of our
bioprocessing business, while seeking external partners for our therapeutic
development programs,” said Walter C. Herlihy, Ph.D., President and Chief
Executive Officer of Repligen. “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

“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.”

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.

“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.”

Families of SMA, a patient organization dedicated to funding research to
advance therapies for 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’s research and
clinical efforts, including the current Phase 1b trial, have been partially
supported by a $1.4 million grant from the Muscular Dystrophy Association.

About Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease
in which a defect in the SMN1 (survival motor neuron) gene results in low
levels of the protein SMN and leads to progressive damage to motor neurons. It
is the leading cause of infant mortality and the second most common inherited
neuromuscular disease, with symptoms that typically emerge before the age of
two. SMA is characterized by progressive muscle weakness leading to severe
physical disability and, often, early loss of life due to respiratory

About 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. For more information:

About the Muscular Dystrophy Association

The Muscular Dystrophy Association (MDA) is the leading nonprofit health
agency dedicated to finding treatments and cures for more than 40
neuromuscular diseases, including SMA, by funding worldwide research. MDA also
funds comprehensive health care and support services, advocacy, information
and education, and accessible summer camp for thousands of youngsters fighting
progressive muscle diseases.To date, MDA has invested more than $41.6 million
in SMA research, funding basic research and clinical trials of therapeutic
strategies such asgene-based therapies, small-molecule development, and stem
cells. MDA, along with other SMA patient advocacy groups, has been working
with policymakers to explore the potential of expanding newborn screening
panels to include SMA.For more information, visitmda.organd follow MDA on
Facebook ( and Twitter (@MDAnews).

Repligen Corporation

Repligen Corporation is a life sciences company focused on the development,
production and commercialization of high-value consumable products used in the
process of manufacturing biological drugs. Our bioprocessing products are sold
to major life sciences and biopharmaceutical companies worldwide. We are a
leading manufacturer of Protein A, a critical reagent used during the
production of monoclonal antibody therapeutics. We also supply several growth
factor products used to increase cell culture productivity during
fermentation. In addition, we have developed and market a series of
chromatography products used in the purification of biologics, and sell test
kits to ensure final product quality. Aside from our core bioprocessing
business, we have a portfolio of clinical-stage partnering assets, including a
pancreatic imaging agent in Phase 3 development and two central nervous system
orphan drug candidates. Repligen’s corporate headquarters are located in
Waltham, MA, USA; we have an additional manufacturing facility in Lund,
Sweden. For more information, please visit our website at

This press release contains forward-looking statements, which are made
pursuant to the safe harbor provisions of Section 27A of the Securities Act of
1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as
amended. Investors are cautioned that statements in this press release which
are not strictly historical statements, including, without limitation, express
or implied statements regarding the potential utility of RG3039 for the
treatment of SMA, the clinical success of RG3039 and its further clinical
development and our receipt of any future payments under the terms of our
agreement with Pfizer, Pfizer’s ability to terminate the license agreement for
convenience, our strategic decision to focus on the growth of our
bioprocessing business, the future demand for our bioprocessing, growth factor
and chromatography products, plans and objectives for future operations, our
ability to successfully negotiate and consummate partnering transactions for
our clinical-stage assets, specifically RG1068, RG3039 and RG2833, plans and
objectives for product development and acquisitions, plans and objectives for
regulatory approval, product development, our market share and product sales
and other statements identified by words like “believe,” “expect,” “may,”
“will,” “should,” “seek,” or “could” and similar expressions, constitute
forward-looking statements. Such forward-looking statements are subject to a
number of risks and uncertainties that could cause actual results to differ
materially from those anticipated, including, without limitation, risks
associated with: the success of our clinical trials, including our Phase 1b
clinical trial of RG3039 in patients with SMA; our ability to successfully
grow our bioprocessing business, including as a result of acquisition,
commercialization or partnership opportunities; our ability to successfully
negotiate and consummate development and commercialization partnerships for
our portfolio of clinical-stage assets on acceptable terms, if at all; our
ability to develop and commercialize products and the market acceptance of our
products; reduced demand for our products that adversely impacts our future
revenues, cash flows, results of operations and financial condition; the
ability to obtain, and the timing and receipt of, FDA approval for our NDA for
RG3039; our ability to obtain other required regulatory approvals; the success
of current and future collaborative or supply relationships, including our
agreement with Pfizer; our ability to compete with larger, better-financed
bioprocessing, pharmaceutical and biotechnology companies; new approaches to
the treatment of our targeted diseases; our compliance with all Food and Drug
Administration and EMEA regulations; our ability to obtain, maintain and
protect intellectual property rights for our products; the risk of litigation
regarding our intellectual property rights; our limited sales capabilities;
our volatile stock price; and other risks detailed in Repligen’s Annual Report
on Form 10-K on file with the Securities and Exchange Commission and the other
reports that Repligen periodically files with the Securities and Exchange
Commission. Actual results may differ materially from those Repligen
contemplated by these forward-looking statements. These forward-looking
statements reflect management’s current views and Repligen does not undertake
to update any of these forward-looking statements to reflect a change in its
views or events or circumstances that occur after the date hereof except as
required by law.


Repligen Corporation
Sondra S. Newman, 781-419-1881
Director Investor Relations
Press spacebar to pause and continue. Press esc to stop.