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Boston, MA and New York, NY - VENTURE WIRE - September 13, 2004
CombinatoRx Signs Discovery Deal With Spinal Muscular Atrophy Foundation
CombinatoRx Inc., a biopharmaceutical company that has developed a process to screen combinations of approved compounds at high throughput in order to create new drugs with novel biological activity, said it has initiated a research and development collaboration with the Spinal Muscular Atrophy Foundation, a nonprofit organization.
The collaboration will focus on identifying novel combination drugs for the treatment of spinal muscular atrophy, or SMA, a genetic, motor neuron disease characterized by the wasting away of skeletal muscles and caused by a defect in the Survival Motor Neuron Gene, or SMN.
SMA is incurable, untreatable and eventually fatal. The disease is often compared to a genetic form of polio because it attacks the same spinal cord cells, and is a lead genetic cause of death among infants and toddlers. In severe cases, the weakness it causes is so great that death results either in the womb, or within the first two years of life.
The new collaboration "expands the business, and takes us into a therapeutic area that we've not been in before," said Daniel Grau, who serves as CombinatoRx's vice president of corporate development.
"It's effectively our first therapeutic effort in CNS (or central nervous system)," Grau noted.
Using a screening process that enables two or more already-approved drug molecules to be screened against a validated disease target, the company's scientists have been able to develop combination medicines that act on multiple targets in a disease network.
Because it is focused on screening libraries of existing Food and Drug Administration and globally approved drugs with known profiles, "in many cases you can move rapidly, and with less cost, into clinical development," Grau said of the platform, which the firm has previously used to establish its clinical and pre-clinical pipeline in cancer and immuno-inflammatory diseases.
That factor, said Grau, "is obviously attractive from an investor perspective, but is also very attractive to the SMA foundation, because they have an urgent focus on getting new therapies to patients as quickly as possible."
As part of the sponsored research CombinatoRx will apply its flagship high throughput screening platform to discover drug combinations that upregulate SMN protein levels.
Under the terms of the agreement, CombinatoRx will receive two years of research and development funding, the ownership of all intellectual property, and commercial rights to all discovered products. In addition, the start-up will also receive milestone payments as programs advance to the investigational new drug application stage, and is also eligible for increased funding to support clinical development of new therapeutics for SMA.
Founded in 2000 by scientists from Harvard and MIT, Boston-based CombinatoRx has raised $90M in venture capital, most recently taking in a $32 million Series D round which closed in March. Shareholders in the start-up include Boston Millennia Partners, Easton Hunt Capital Partners, Yasuda Enterprise Development, Global Life Science Ventures, Canaan Partners, Flagship Ventures and TL Ventures.
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