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Shares in Alnylam Pharmaceuticals jumped after the company said a drug to treat a rare genetic form of heart disease met its goals in a late-stage trial, opening up the possibility of billions of dollars in additional sales for one of the most valuable US biotechs.
The company said its quarterly Vutrisiran injection cut the risk of mortality and adverse cardiovascular events by at least 28 per cent in a phase-three trial. It also improved capacity for physical exertion among patients with a rare protein disorder that affects heart function.
The disease, called transthyretin-mediated amyloidosis cardiomyopathy, or ATTR-CM, affects around 100,000 patients in the US and up to 500,000 globally, most of whom are men aged 60 and over. However, many of the cases go undiagnosed.
Vutrisiran is already approved to treat a more rare but related heart condition under the brand name Amvuttra. But Alnylam now anticipates that the US Food and Drug Administration could greenlight a wider rollout of the drug early next year.
The approval would be a huge boon for Alnylam, which specialises in a treatment method called RNA interference, or RNAi, that works by silencing genetic mutations affecting patients with rare diseases.
Shares in Alnylam, which has long been considered a takeover target for major pharmaceutical groups, were up 28 per cent shortly after Wall Street’s opening bell on Monday, giving the biotech a market value of more than $26bn.
Crucially, Vutrisiran boosted the health outcomes of trial participants already using Tafamidis, a rival drug sold by Pfizer under the brand Vyndamax, which is currently the most common treatment option for ATTR-CM patients. Analysts have said Vutrisiran’s efficacy among this subgroup of patients would be key to justifying its use as a first-line therapy.
“There really is the potential that Vutrisiran will become the new standard of care for [ATTR-CM patients],” said Yvonne Greenstreet, Alnylam’s chief executive. “We have in our hands a multibillion-dollar opportunity and what this will do to us is accelerate our transition to becoming a leading biotech company.”
Greenstreet compared the positive trial data to having “finally reached the top of mountain” after the stock dropped 8 per cent in a single day earlier this year when investors responded negatively to the biotech’s decision to change the way in which it was assessing its closely watched late-stage trial.
“I think this is the moment that will stand out as an inflection point in the company’s history and really allow us to take the power of RNAi and point it to so many more unmet medical needs,” added Greenstreet.
Alnylam is also developing a hypertension drug with Swiss pharma group Roche, as well as deploying its RNAi technology against Alzheimer’s, diabetes and obesity in early-stage trials.
Vutrisiran reduced the risk of mortality from all causes compared with a placebo group by 28 per cent among patients already using Tafamidis as a stabiliser and by 33 per cent among patients on no other treatments.
Speaking at a JPMorgan investor event earlier this month, Carlos Santos-Gallego, a leading cardiologist at New York’s Mount Sinai Hospital, said an improvement of 25 per cent or more among patients on dual therapies on Alnylam’s trial would be “very clinically meaningful” and lead him to prescribe Vutrisiran to his patients.
