Friday, January 4, 2013



You may remember that last January we had a couple of meetings with Pfizer , the drug company, to discuss the fact that they produced SMA milk. At the time we said that they should realise the effect that the name has on parents who have children dying from Spinal Muscular Atrophy. When they said that they would not change the name we suggested that they should be contributing to SMA research. They contributed £ 25,000 which we gave to the Jennifer Trust.

One year on and Pfizer have now announced that they will contribute $ 70 Million to SMA research through the company REPLIGEN who are working on an SMA medicine. In fact clinical trials of a new drug are starting in the first quarter of this year.

We could not be more pleased with this development and recognise that Pfizer actually do listen to people. They even sold the SMA milk brand.

We are , today, stopping the Shame on Pfizer campaign in recognition of the fact that they are now so instrumental in looking for a cure for SMA. Well done to everybody who contacted them last year

Government launching a consultation on screening for SMA and now this major funding development. I like the look of this 2013 !

Repligen Announces Licensing Agreement with Pfizer
for Spinal Muscular Atrophy Program

 January 03, 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 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.

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
Repligen Announces Licensing Agreement with Pfizer for Spinal Muscular Atrophy Program,
January 03, 2013
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, smallmolecule
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 and 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 clinicalstage
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


  1. What fabulous news!!! Much love to you all

    Louise Hawkesworth (@ouisey) xx

  2. Fantastic news. Well done!!!