February 13, 2024

The need-to-know this morning

Biogen reported earnings for the fourth quarter and 2023.
Regulators in Europe approved Casgevy, a CRISPR-based gene-editing treatment, for people with sickle cell disease and beta thalassemia. Casgevy is made by Vertex Pharmaceuticals and CRISPR Therapeutics.
The FDA extended a review of Rocket Pharmaceuticals' gene therapy, called Kresladi, by three months to June 30, to allow additional time for the review of manufacturing information. Kresladi is being developed to treat leukocyte adhesion deficiency-I, a rare, genetic disease that causes immune deficiency in children.
Roivant and Incyte also reported earnings.
AI drug discovery firm Exscientia fired CEO Andrew Hopkins for cause following an investigation that found he had "inappropriate" relationships with two company employees. David Nicholson, chairman of Exscientia's board, also resigned because he had prior knowledge of Hopkins' relationships but didn't notify the company.

obesity

BioAge's $170 million Series D for GLP-1 combo pill

BioAge, a California biotech developing a combination therapy with the blockbuster obesity drug Zepbound, just raised a $170 million Series D round. Its lead candidate, azelaprag, is meant to help optimize weight loss from GLP-1 drugs so that muscle mass is preserved. BioAge is developing drugs for neuroinflammation as well.

This funding round includes Sofinnova Investments, Eli Lilly and Amgen's venture arms, and previous investor Andreessen Horowitz. Although BioAge has several programs, it was the azelaprag-Zepbound combination that sparked investor interest.

Azelaprag mimics the hormone apelin, which is secreted in response to exercise. It plays a role in regulating metabolism and muscle biology. Apelin levels dwindle with age — so BioAge, whose drugs aim to keep the effects of aging at bay — is interested.

rare diseases

KalVista's swelling drug performs well in phase 3

KalVista Pharmaceuticals' treatment for the acute swelling episodes seen in hereditary angioedema performed well in Phase 3 studies — meaning that the company will submit a marketing application for the drug, called sebetralstat, by the middle of the year. Symptoms began to abate between 1.6 and 1.8 hours after patients took the oral pill, compared to 6.7 hours with placebo. There were no serious side effects from the medicine.

Right now, the most common way to treat a swelling episode from hereditary angioedema is a generic medicine called icatibant, which is injected subcutaneously. It takes about two hours for symptom relief to begin.

Although the market is small, sales for sebetralstat could hit $600 million in the U.S. and Europe, Leerink analysts say. Notably, Intellia Therapeutics is developing a CRISPR-based treatment for hereditary angioedema that has the potential to be curative.

ebola

Ebola vaccine works even in people who had already been infected

A new study shows that people vaccinated against Ebola with a single dose of Merck's Ervebo are far less likely to die than those who aren't vaccinated, even if they do develop the disease.

The research, published in Lancet Infectious Diseases, is based on data from the 2018-2020 Ebola Zaire outbreak in the Democratic Republic of the Congo. It showed that the inoculation halved the risk of dying among people who had received the vaccine before developing symptoms — including those who only received the vaccine just a day or two before getting sick. About 27% of people who had been vaccinated within two days of becoming symptomatic died, compared to 56% in those who were unvaccinated.

"That's a huge finding," the study's first author told STAT. "We know now that vaccination is better late than never. So this is one more reason to use the Ebola vaccine."

rebranding

Gilead acquiring CymaBay for $4.3 billion

Gilead Sciences will acquire CymaBay Therapeutics for $4.3 billion, based primarily on the latter's lead candidate, seladelpar, which aims to treat a rare autoimmune liver disease called primary biliary cholangitis. The FDA is reviewing a marketing application for seladelpar, with an approval expected by Aug. 14.

This acquisition boosts Gilead's liver disease portfolio, which includes its treatments for hepatitis C. It also represents a redemption for CymaBay, which experienced some serious setbacks in 2019 when seladelpar failed to treat MASH, formerly known as NASH. In 2022, CymaBay's stock was about $2 a share. The deal with Gilead places shares for CymaBay at $32.50 — 27% higher than they were just last week.

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