Heart disease drugmaker Alnylam (NASDAQ:ALNY) stock rose sharply on the success of a Phase 3 study of its heart drug, vutrisiran. The drug is also sold as Amvuttra.
The drug cut the risk of death from a heart disease called ATTR-CM by up to 36%.
ATTR-CM is a rare hereditary condition. The standard treatment has been a drug called Vyndaqel, which is also sold by Pfizer (NYSE:PFE).
As trading opened this morning, ALNY stock was trading at $222 per share, up 34% from its June 21 close. At that price, the market capitalization was over $28 billion.
Heart Disease Hope
Alnylam CEO Yvonne Greenstreet called the result “a multi-billion dollar opportunity.” FirstWord Pharma called the study results a “home run.”
Vutrisiran is an RNAi therapeutic that inhibits the genetic expression of specific molecules by neutralizing targeted mRNA. In this case, the target is prealbumin, a protein made in the liver that carries thyroid hormones and vitamin A.
The study, called HELIOS-B, examined 655 adults with ATTR-CM who received a 25 mg shot of either vutrisiran or a placebo every three months.
Vindaqel was first approved in 2019 and given an orphan drug designation used for rare diseases. Fewer than 9,000 patients were identified in the last decade but the condition is considered underdiagnosed. AstraZeneca (NYSE:AZN) has estimated there may be as many as 500,000 people worldwide with the condition.
Patients with suspected ATTR-CM often feel worse when taking standard medication for high blood pressure. They may have an irregular heartbeat, suffer fatigue and shortness of breath, and have other symptoms. Diagnosis is done by a cardiologist. In addition to standard tests like an electrocardiogram (ECG), this can include genetic tests.
ALNY Stock: What Happens Next?
The number of people with ATTR-CM is still unknown. An available treatment option is likely to help find some who are otherwise not being diagnosed.
On the date of publication, Dana Blankenhorn did not hold (either directly or indirectly) any positions in the securities mentioned in this article. The opinions expressed in this article are those of the writer, subject to the InvestorPlace.com Publishing Guidelines.
