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Oppenheimer healthcare equity strategist Jared Holz described the results as one of the most anticipated “binary” events in biotech for the second half of the year.
Dreamstime
A highly anticipated late-stage trial of
Alnylam Pharmaceutical
s’ drug Onpattro in a rare heart condition returned positive results, the company said, sending shares of the biotech up more than 50% in premarket trading.
The results for the condition, called transthyretin-mediated amyloidosis with cardiomyopathy, seem likely to open a far larger market to Onpattro, which was responsible for $475 million in revenue last year. A
Pfizer
(ticker: PFE) drug that treats the same condition sold $2 billion last year.
Alnylam (ALNY) reported only top-line results from the 360-patient, Phase 3 trial on Wednesday. It said full results will follow at a scientific conference next month, and that it will file for approval of the drug in the new indication late this year.
The company said that patients who received the treatment performed better after a year on a six-minute walk test than patients who received a placebo. Patients who received the treatment also showed improvement over those who received a placebo on a metric designed to measure quality of life.
Alnylam said that there were five deaths in the group that received the treatment, compared with eight in the groups that received a placebo.
Onpattro is currently approved by the FDA to treat polyneuropathy caused by transthyretin-mediated amyloidosis. The drug is an RNAi-based therapeutic, delivered using the same lipid nanoparticle approach used in the mRNA-based vaccines. Its initial approval in 2018 was a major validation of lipid nanoparticles-based medicines, and a key milestone in the development of the mRNA-based vaccines.
Alnylam shares were down 16.3% so far this year as of the close of trading on Tuesday. The results of the trial had been a much-anticipated event for investors this summer.
“This had been one of the most highly anticipated ‘binary’ events in Biotech for the second half of the year and will likely appease investors that were looking at this trial to spark some renewed enthusiasm across therapeutics,”
Oppenheimer
healthcare equity strategist Jared Holz wrote in a note to investors on Wednesday morning.
Biotech stocks are still down this year, despite a strong summer. The
SPDR S&P Biotech ETF
(XBI) has fallen 27.7% so far in 2022, though it is up 9% since the start of July.
In late July, Alnylam reported second-quarter revenue of $214 million. At the time, analysts said that investors were focused on the Onpattro trail.
Write to Josh Nathan-Kazis at josh.nathan-kazis@barrons.com
