July 17, 2023

The need-to-know this morning

A drug developed by the biotech firm BridgeBio to treat an increasingly common heart condition succeeded in its main goal in a clinical trial — reducing the risk of death by 25% compared to a placebo. The results may give the medicine, acoramidis, a path to the market after a failure that led its maker's stock to plunge in December 2021.
The Federal Trade Commission made a second request for information to Seagen and Pfizer regarding their proposed $43 billion merger.
Shares of Apellis Pharmaceuticals fell in pre-market trading due to a safety bulletin distributed to eye doctors. The notice warned of six cases of occlusive retinal vasculitis, a serious inflammatory side effect, reported in people injected with Syfovre, the company's recently approved eye disease treatment.

autoimmune disease

Argenx antibody drug performs well in new indication

Belgium-based Argenx said that its antibody drug, Vyvgart, substantially delayed the progression of an autoimmune nerve disorder called chronic demyelinating polyneuropathy, or CIDP. A pivotal trial showed a 61% reduction in the risk of relapse compared to placebo: "We hit the bullseye," Argenx CEO Tim Van Hauwermeiren told STAT. The company plans on sharing the CIDP data with the FDA and submit a marketing application by the end of the year.

Vyvgart has already been approved for myasthenia gravis, another autoimmune disease. The drug's performing well commercially, and Argenx thinks the drug could help treat about a dozen other autoimmune diseases.

alzheimer's

The new CRISPR approaches to Alzheimer's disease

Researchers are looking at CRISPR as a possible means to treat Alzheimer's disease. The work, presented at the Alzheimer's Association International Conference in Amsterdam, is in its infancy — tested only in mice and lab-made organoids made from brain cells. But the research exemplifies how scientists are trying to approach Alzheimer's from a variety of different angles — not just the amyloid approach, as in years previous.

In the aforementioned work, scientists at Duke tried to use CRISPR to dampen the effects of the APOE4 gene — using powerful editing tools to trim its epigenome. When they attacked the right spot of the epigenome, they were able to essentially shut off the APOE4 gene, limiting its expression in mice. No matter the results of that individual study, the thinking is that Alzheimer's is a complex disease that will need multiple approaches to treat it.

"There is no magic bullet for this disease," one of the researchers told STAT.

Venture capital

Westlake Village BioPartners raises $450 million

Westlake Village BioPartners just raised $450 million in its third funding round. The West Coast firm was launched five years ago by Amgen vets Sean Harper and Beth Seidenberg. This new fund was raised in part by two former investors from 5AM Ventures, Mira Chaurushiya and David Allison, who are now joining Westlake.

The initial aim of Westlake had been to support the fledgling biotech scene in Los Angeles. At the time, the only biopharma players there were Amgen, Kite Pharma, and a handful of others. Since 2018, Westlake has launched eight companies in L.A., and more than a dozen elsewhere in the U.S. About 40% of the new fund will go to companies in the Los Angeles area.

patent wars

Under pressure, J&J expanding access to TB pill

The United Nations-affiliated group STOP TB scored a victory in a long-standing patent battle with Johnson & Johnson. The advocacy group has been fighting the pharma giant over the patent on a lifesaving tuberculosis pill called bedaquiline. The medicine was approved in 2012, and was the first in more than four decades to treat the disease — but it's hard to access in low- and middle-income countries. Tuberculosis still kills about 1.5 million people each year — and J&J was initially charging $900 per course in low-income countries. "It cost a bloody fortune when they first released it," one TB expert told STAT, and though it now costs about $300 it "often remains out of reach."

The initial patent for bedaquiline is expiring this month — and STOP TB just announced an agreement to make cheaper generic versions of the drug available in dozens of low- and middle-income countries with high rates of the disease. Interestingly, novelist and YouTube star John Green has been one of the most vocal proponents of widening access to the drug, and the pressure campaign seems to have worked.

That said, J&J has a secondary patent that only expires in 2027, which can optimize the original molecule. If J&J chooses to enforce the patent, it could delay the arrival of generics in countries where drug-resistant forms of the disease are rampant.

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