Updates on Pharmaceutical Companies Investing in SMA Drug Development

By Cure SMA | Published On February 3, 2015

Over the past decade, one of the most important advances in SMA drug development has been the number of pharmaceutical companies investing in treatments for SMA. Ten years ago, we had just one company. Now, we have a dozen. These additional companies bring additional resources and expertise that will get us to treatments and a cure faster.

In the past several weeks, there have been several notable developments in this area:

  1. Astellas invested in Cytokinetics to advance CK-2127107, a muscle drug, into Phase 2 clinical trials.
  2. Roche agreed to purchase Trophos. Roche, the third largest pharmaceutical company in the world, is now using its resources to help move olesoxime forward.
  3. Roche Venture Capital (VC) invested in AveXis, which is currently working on a gene therapy treatment. AveXis also received funding from Deerfield Management, a healthcare investment firm, as part of that same round of financing.
  4. Pfizer and Repligen announced plans to dissolve their partnership effective the end of April. Cure SMA is currently working with the groups involved to learn more about future plans and options for the program. A further update will be provided on this as soon as we have definitive information, which will likely happen in late April.

Our SMA drug pipeline tracks the progress of all the individual drug programs, as well as the different companies who are investing in those programs. The most recent version of this pipeline was released at the end of last year. We’ll be releasing an updated version later this spring, reflecting these developments in the SMA drug programs.

Questions on the drug pipeline or on how we’re working with these pharmaceutical partners? Send us an email at info@curesma.org.

Topics: Clinical Trials, Front Page News

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