What happened
Shares of BridgeBio Pharma (BBIO 0.94%) were up 90% for the week as of 10:25 a.m. ET on Friday, according to data provided by S&P Global Market Intelligence. The biotech company, which focuses on therapies for oncology and genetic diseases, reported positive phase 3 trial data regarding one of its therapies.
NASDAQ: BBIO
Key Data Points
So what
On Monday, BridgeBio said its therapy, acoramidis, showed promising results in a phase 3 study to treat transthyretin amyloid cardiomyopathy (ATTR-CM), a serious disease of the heart muscle that can lead to heart failure. In ATTR-CM, the transthyretin protein becomes misshapen in the bloodstream and can build up in the heart, nerves, and other organs.
Acoramidis is a pill that helps modulate transthyretin. In the trial, the company said patients given the drug saw an 81% on-treatment survival rate, compared to a 74% rate with a placebo. Patients given the therapy were also 50% less likely to be hospitalized for cardiovascular reasons, BridgeBio said.
The company said the drug met its primary endpoint and showed a good safety profile. Because of the positive trial news, BridgeBio said it plans to submit its New Drug Application (NDA) with the Food and Drug Administration (FDA) by the end of the year, with regulatory filings in other countries to follow next year.
Now what
The strong showing for acoramidis means it should be a strong competitor to two other ATTR-CM drugs: Alnylam Pharmaceuticals' therapy, patisiran, which is under review for cardiomyopathy, and Pfizer drug Vyndamax, which has been approved in the U.S. to treat ATTR-CM.
Investors will want to see how BridgeBio's other lead pipeline candidate, infigratinib, which is already approved to treat bile duct cancer, fares in a late-stage trial to treat children with achondroplasia, a common type of dwarfism. In the first quarter, the company reported revenue of $1.826 million, up 7.7%, year over year, and an earnings-per-share (EPS) loss of $0.92, compared to an EPS loss of $1.35 in the same period last year. As of the first quarter, the company had $467 million in cash.
