Alnylam reported a successful Phase 3 trial of its heart drug butrisiran, also known as Ambutra.
Source: Pavel Kapiš / Shutterstock.com
Cardiac medicine manufacturer Alnilam (Nasdaq:Arnie) shares soared following the success of a Phase 3 trial of its heart drug, butrisiran, which is also sold as Ambutra.
The drug reduced the risk of death from a type of heart disease called ATTR-CM by up to 36%.
ATTR-CM is a rare genetic disease. The standard treatment is a drug called Vyndaqel, which Pfizer (New York Stock Exchange:Personal consumption tax).
As of the start of trading this morning, ALNY stock was trading at $222 per share, up 34% from its closing price on June 21. At this price, the company has a market cap of over $28 billion.
Hope for Heart Disease
Alnylam CEO Yvonne Greenstreet called the results a “billion-dollar opportunity.” First Ward Pharma He called the findings a “home run.”
Vutriciran is an RNAi therapeutic that blocks the gene expression of specific molecules by neutralizing their target mRNA — in this case, prealbumin, a protein made in the liver that carries thyroid hormone and vitamin A.
The study, called HELIOS-B, looked at 655 adults with ATTR-CM who received injections of 25 mg of butrisiran or a placebo every three months.
Vyndaqel was first approved in 2019 and received orphan drug designation for use in treating a rare disease, which is considered underdiagnosed, with fewer than 9,000 confirmed cases in the past decade. AstraZeneca (New York Stock Exchange:Azun) estimates that as many as 500,000 people worldwide have the disease.
Patients with suspected ATTR-CM often experience worsening symptoms when taking standard medications for high blood pressure. They may experience irregular heartbeats, fatigue, shortness of breath, and other symptoms. The diagnosis is made by a cardiologist.electro-cardiogram), which may include genetic testing.
ALNY Stock: What happens next?
The number of people with ATTR-CM is still unknown, and available treatments could help find patients who would otherwise go undiagnosed.
On the date of publication, Dana Blankenhorn did not have (either directly or indirectly) any positions in the securities mentioned in this article. Opinions expressed in this article are solely those of the author, copyright InvestorPlace.com. Publication Guidelines.