In fresh setback, Alnylam's most advanced drug flunks PhIIb RSV test

Alnylam's most advanced RNAi drug program has flunked a Phase IIb test for efficacy in treating respiratory syncytial virus (RSV). Just four months after axing a third of its staff, the Cambridge, MA-based biotech ($ALNY) says that ALN-RSV01--one of its partnered programs--failed to achieve the primary endpoint: a drop in the rate of new or progressive bronchiolitis obliterans syndrome at 180 days after RSV infection. But Alnylam did claim a "statistically significant reduction in the incidence of day 180 BOS" (blocked airways) in two of four analyses of patients.

This morning, Alnylam was sticking to the sunny side of the data, downplaying a clinical failure at a time the biotech clearly needs to establish a record of success. Its stock price is down 24% in three months, but this morning shares barely budged on the news.

"We believe that these data provide important evidence that ALN-RSV01 reduces the incidence of new or progressive BOS in RSV-infected lung transplant patients, replicating the findings from our Phase IIa study of this agent in the same clinical setting. We plan to discuss the results of this study with U.S. and European regulatory authorities later this year and, thereafter, determine appropriate next steps, if any, on our ALN-RSV program," said Akshay K. Vaishnaw, the senior vice president and CMO of Alnylam. 

Over the last two years Alnylam has changed dramatically. Once a leader in a hot field, the biotech has felt a growing chill as leading companies exited the arena, concerned of the risk and lengthy development timelines. And instead of growing fast in the wake of new deals, Alnylam is now shrinking as it circles the wagons around the existing pipeline.

The RSV program is partnered with Kyowa Hakko Kirin in Asia and Cubist Pharmaceuticals in the rest of the world. The company has been focused heavily on an effort to get five of its own programs into the clinic, with a special emphasis on programs for transthyretin-mediated amyloidosis and hemophilia.

- here's the press release
- here's the Reuters report

Related Article:
Alnylam cutting third of jobs to devote cash to lead RNAi programs
Countering skeptics, Alnylam claims pioneering first in RNAi cholesterol study