Alnylam misses mark in potential lung treatment study - Mass High Tech

Alnylam Pharmaceuticals Inc. (Nasdaq: ALNY) of Cambridge said today that it missed its goal but saw a “statistically significant reduction” in the incidence of a life-threatening lung disease in lung transplant patients with respiratory syncytial virus (RSV) infection in a Phase 2b trial of a drug candidate.

Alnylam’s stock was trading up 1 percent at around $10.23 per share as of 11 a.m. Wednesday, hours after the company announced the results of the trial of ALN-RSV01.

The company said the study missed the primary endpoint of reduced incidence of new or progressive bronchiolitis obliterans syndrome (BOS) within six months after RSV infection in an “intent-to-treat” analysis of all 87 patients who participated. However, it said in a statement that “in all analyses, ALN-RSV01 treatment was associated with a clinically meaningful treatment effect, with a reduction of over 50 percent in the incidence of day 180 BOS as compared with placebo.”

“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 2a 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, senior vice president and chief medical officer of Alnylam in the statement.

The company said that RSV infection in lung transplant patients represents “a significant unmet medical need” since BOS has about a 50 percent mortality rate within five years.

More than three years ago, Alnylam received an upfront payment of $20 million from Cubist Pharmaceuticals Inc. in a partnership to develop Alnylam’s ALN-RSV program. The deal was said to be potentially worth a total of $102.5 million to Alnylam.