Milford biotech firm may see therapy advance via new partnership
Milford biotech startup Prothelia Inc. announced it has entered into an agreement with a Connecticut-based drug company to further develop Prothelia’s clinical-stage treatment for the rare form of muscular dystrophy known as MCD1A.
Alexion Pharmaceuticals Inc., which specializes in treatments for rare diseases, will have an option to acquire Prothelia and license its clinical-state treatment, Laminin-111, after entering into a “strategic agreement” with Prothelia and its research partner, the University of Nevada Reno (UNR), according to a statement from Prothelia.
A “significant step” for potential treatment
The option to purchase the company and bring the treatment to market depends on the ability of Prothelia and UNR to meet the required development milestones, according to the two organizations.
The patented Laminin-111 therapy is based on the work of Dr. Dean Burkin, a professor at the UNR school of medicine. The patented therapy was licensed to Prothelia in 2008 but has yet to undergo clinical trials, according to Irving Adler, a spokesman for Alexion.
Prothelia CEO Richard Cloud said the agreement with Alexion is a “significant step” toward further investigating Laminin-111 as a potential treatment for MDC1A, a rare, life-threatening disease that causes respiratory insufficiency, poor muscle tone, muscle atrophy, scoliosis and joint problems as well as delayed development of motor skills.
"We are confident that Alexion is the right partner, given their commitment to patients suffering from devastating, ultra-rare diseases like MDC1A,” Cloud said.
There is currently no treatment for MCD1A, and Laminin-111 is still years away from being market-ready, according to Adler, the Alexion spokesman.
But Adler said Alexion, which has the capability to manufacture drugs on a large scale that Prothelia does not, is excited about the potential of Laminin-111.
Soliris success sets the bar for future therapies
Alexion’s commercial drug Soliris, which transformed the startup into a global drug company when it was approved by the U.S. Food and Drug Administration in 2007, treats the rare genetic kidney disease called paroxysmal nocturnal hemoglobinuria.
Adler said patients treated with Soliris have, in many cases, come off dialysis and seen debilitating effects of the disease reversed. In partnering with, and potentially acquiring Prothelia, Alexion hopes it will change MCD1A patients’ lives in the same way.
“That’s the type of transformative impact we are looking for,” Adler said.
Adler did not have information about the size of Prothelia’s operations, or how Alexion might absorb the startup if a deal is finalized.
A spokeswoman for Prothelia referred comment to Alexion.
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