WALTHAM, Mass.--(BUSINESS WIRE)--
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 SMA.”
“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 insufficiency.
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: www.curesma.org.
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 as gene-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, visit mda.org and
follow MDA on Facebook (facebook.com/MDAnational)
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 www.repligen.com.
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
snewman@repligen.com
Director
Investor Relations
Source: Repligen Corporation