Repligen Receives First Milestone Payment From Pfizer Under Licensing Agreement for Spinal Muscular Atrophy Program

Repligen Receives First Milestone Payment From Pfizer Under Licensing
Agreement for Spinal Muscular Atrophy Program

WALTHAM, Mass., Sept. 4, 2013 (GLOBE NEWSWIRE) -- Repligen Corporation
(Nasdaq:RGEN) announced today that it has received a $1 million milestone
payment from Pfizer, Inc. under the terms of the companies' exclusive
worldwide licensing agreement (the "Agreement") for the development of
compounds to treat spinal muscular atrophy (SMA). This first milestone payment
was triggered by completion of specific program activities and coincides with
the successful completion of all transition obligations by Repligen. Repligen
announced the Agreement in January of this year, at which time it received an
upfront payment of $5 million. Repligen remains eligible to receive up to $64
million in additional success-based milestone payments, as well as royalties
on any future sales of compounds developed under the Agreement.

"Consistent with our strategic decision last August to focus on building
Repligen's bioprocessing business while scaling back our investment in
therapeutics, we out-licensed the SMA program and have successfully completed
its transition to Pfizer," said Walter C. Herlihy, Ph.D., President and CEO of
Repligen. "We believe the Agreement preserves the potential for the SMA
program to deliver significant long-term upside for our shareholders."

Repligen originally in-licensed the SMA program from Families of SMA (FSMA), a
patient organization dedicated to supporting research to advance therapies for
SMA. FSMA funded and directed the preclinical development of the program's
lead compound, RG3039, with an investment of more than $13 million. This was
the first drug discovery program ever conducted specifically for SMA. The
Muscular Dystrophy Association also provided critical support to Repligen's
research and clinical efforts, including the conduct of a Phase 1b trial.

About Spinal Muscular Atrophy

Spinal muscular atrophy 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 genetic 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 Repligen Corporation

Repligen Corporation (Nasdaq:RGEN) 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 to separate and purify monoclonal antibody therapeutics. We also
supply several growth factor products used to increase cell culture
productivity during the fermentation stage of drug manufacturing. In addition,
we have developed and market our OPUS^® line of pre-packed "plug-and-play"
chromatography columns, and we provide 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 an orphan drug candidate in Phase 1 development.
Repligen's corporate headquarters are located in Waltham, MA (USA) and our
manufacturing facilities are located in Waltham, MA and Lund, Sweden.

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, constitute forward-looking statements,
including, without limitation, express or implied statements regarding the
potential utility of RG3039 for the treatment of SMA, the safety and
tolerability profile of RG3039 as well as its ability to achieve specific
levels of target enzyme inhibition in future clinical trials, the potential
for the SMA program to deliver significant long-term upside for our
shareholders, 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, our ability to meet future milestones under the
agreement with Pfizer, Pfizer's ability to terminate the license 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, plans and objectives for product development and
acquisitions, our market share and product sales and other statements
identified by words like "believe," "expect," "may," "will," "should," "seek,"
"anticipate," or "could" and similar expressions. 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 of
RG3039 in patients with SMA and in healthy volunteers, 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 impact of the expiration of Bristol-Meyers Squibb royalty
payments from U.S. sales of Orencia^®; 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; our ability to successfully
integrate Repligen Sweden AB, including achieving manufacturing efficiencies
at Repligen Sweden AB; 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.

CONTACT: Sondra S. Newman
         Director Investor Relations
         Repligen Corporation
         (781) 419-1881
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