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With Early Data, Alkermes Plots Quick Jump to Pivotal Study For MS Drug

Xconomy Boston — 

Alkermes’ transformation from a drug-delivery specialist to a pharma company with its own pipeline has been well-documented. Shares are now worth more than triple what they were a few years ago as the Dublin, and Waltham, MA-based company has advanced in-house drugs for depression and schizophrenia.

Now you can add a prospective multiple sclerosis treatment—one that Alkermes aims to pit against Biogen Idec’s blockbuster MS pill dimethyl fumarate (Tecfidera)—to the pack.

Alkermes (NASDAQ: ALKS) said today that the results from a Phase 1 trial of ALKS 8700, its prospective MS pill, came back positive. This is an early study, of course, geared to test a drug’s safety in healthy volunteers, not how well it impacts disease in MS patients. Alkermes has a long way to go. But the company designed its trial to compare ALKS 8700 to Biogen’s (NASDAQ: BIIB) drug in a small group of volunteers to see if its hypothesis—that its drug might cause fewer side effects than Biogen’s drug—holds water.

Alkermes believes it is seeing signs that ALKS 8700 does make an impact on safety. The company is reporting that the volunteers taking its drug had fewer GI-related side effects than those taking dimethyl fumarate (the most common side effect was flushing). Alkermes is encouraged enough by the results that it aims to talk to the FDA about moving ALKS 8700 into a pivotal study later this year. While Alkermes says it’s continuing to pursue once-daily dosing options, it would test a twice-daily dose in the pivotal study (dimethyl fumarate is also taken twice a day).

Alkermes’ ALKS 8700 is a prodrug—an inactive substance that enzymes or other chemicals in the body turn into a drug—-that yields monomethyl fumarate, a product that Alkermes is trying to show is better than Biogen’s drug.

The key for Alkermes is for this to translate into a significant safety advantage. In a recent research note, analyst Ken Cacciatore from Cowen & Co. noted that based on discussions with experts in the field, a “sizable number of patients are having tolerability issues with [dimethyl fumarate], especially when initiating therapy,” leading many to discontinue treatment. Still, the bar would be high for Alkermes to barge into the market.

“A methyl fumarate prodrug would be appealing for the treatment of MS, but would have to demonstrate at least a 50 percent decrease in side effects to be clinically compelling and justify use over [Biogen’s drug],” Cacciatore wrote, citing discussions with experts.

The prize is substantial: dimethyl fumarate quickly became a blockbuster. It was approved in March 2013 and generated $2.9 billion during the 2014 fiscal year. But Alkermes is also facing some competition. Xenoport (NASDAQ: XNPT), of Santa Clara, CA, is developing a similar drug, and according to a note from RBC Capital Markets’ Michael Yee, Biogen has said it’s working on updated formulations of dimethyl fumarate in Phase 1 testing as well. Yee added today that Biogen believes dimethyl fumarate “has a different efficacy profile” than monomethyl fumarate, so it’s worth watching how effective these drugs ultimately prove to be in patients relative to Biogen’s drug.

“It would make sense that [Biogen] would suggest this, although there is a debate as to whether [dimethyl fumarate] or [monomethyl fumarate] have enough clinical efficacy differences,” Yee wrote.

Clearly, for its part, Alkermes believes today’s results make ALKS 8700 a threat.

“The results from this study demonstrated ALKS 8700 converts efficiently into MMF after oral administration with the potential to offer improved GI tolerability for patients with MS,” said Alkermes chief medical officer Elliot Ehrich, in a statement. “This highly informative clinical study provided clear data regarding dose selection and supports our decision to advance ALKS 8700 twice-daily into pivotal development later this year.”