August 1, 2024

The need-to-know this morning

Biotech earnings are bountiful: Moderna, Biogen, Apellis Pharmaceuticals, Neurocrine Biosciences, Ionis Pharmaceuticals, Blueprint Medicines, Agios Pharmaceuticals, Regeneron, and Alnylam Pharmaceuticals.
Otsuka Pharmaceuticals said it is paying $800 million to acquire Jnana Therapeutics, developers of a drug for PKU.

vaccines

What the 40-year quest to fight malaria teaches us about vaccine development

For decades, pharma companies and academic researchers have struggled to develop vaccines that could confer protection against malaria, fueling doubts about whether such a product was even possible. But scientists have finally succeeded — there are now two products being rolled out, one developed by GSK and partners, and the other by scientists at the University of Oxford.

It's a story of both "scientific grit and fundraising ingenuity," my colleague Drew Joseph writes. Researchers not only had to figure out how to take on a sophisticated biological adversary, but they also had to find ways to advance the testing of products that much of the biopharma industry was not interested in.

This piece explains not only how the malaria vaccines came to be, but also how commercial prospects can influence vaccine development. Experts also propose alternative ways of funding research on neglected diseases going forward.

Please take some time to read the story here. And if you want a quick summary of it for now (though I still recommend the full story), here are four key takeaways.

GLP-1s

Lilly's Zepbound shows benefits in heart failure

Eli Lilly reported this morning that Zepbound cut the risk of major complications and improved symptoms in patients who have heart failure with preserved ejection fraction (HFpEF). This follows positive results from Novo Nordisk's Wegovy in this condition.

The trials by the two companies were designed very differently, so it's difficult to compare the results at this point. (I get into the data from both in the full story.) But this development shows how the pharma giants continue to compete not just on the amount of weight loss their GLP-1s can cause, but also how well the treatments perform in disease areas beyond obesity and diabetes.

health tech

Wojcicki files proposal to take 23andMe private

A trust controlled by 23andMe co-founder Anne Wojcicki has filed to take the genetic testing firm private, offering to purchase all shares of the firm for the current stock price of 40 cents per share, according to an SEC filing.

In a letter accompanying the filing, Wojcicki wrote, "the short-term focus of the public markets has led me to believe that the Company will be best equipped to execute against this mission as a private entity, allowing us to remove certain public company costs and distractions." She had previously telegraphed her intention to do this.

The proposal would still need to be approved a special committee of the company's board and by shareholders.

gene therapy

Regenxbio's Duchenne therapy could be next on the market

Regenxbio said today that its experimental gene therapy for Duchenne muscular dystrophy was given to two more boys in a Phase 1/2 trial and produced high levels of microdystrophin, a miniaturized version of the protein needed for muscles to function properly.

The results mean that Regenxbio's medicine, called RGX-202, could become the second gene therapy for Duchenne to reach the market, after the FDA's controversial approval of Sarepta's Elevidys in June.

It's not known if more microdystrophin necessarily leads to greater improvements in muscle function. Still, the FDA has shown that it will grant accelerated approval to a gene therapy based largely on microdystrophin production alone.

venture capital

venBio raises $528 million for its fifth fund

From Allison DeAngelis: San Francisco VC firm venBio raised $528 million for its fifth fund, passing the firm's initial target but coming in slightly less than the $550 million it raised for its 2021 fund.

Managing Partner Aaron Royston said that the firm's management settled on the fund size for a few reasons: Raising a larger pool of capital would mean the firm would either need to invest in more startups — which would require more manpower — or put more money into each startup it invests in, which not every biotech needs. And one of the partners is eyeing retirement in a few years and didn't want to sign on for another fund.

VenBio has invested in more than 40 companies since it launched in 2011. It was an early investor in RayzeBio, the radiopharmaceuticals startup that went public last year and was ultimately acquired by Bristol Myers Squibb. "Rayze, when we invested in the series A, it was an idea on a napkin. Within five years, we were able to drive to clinical data," Royston said. VenBio is a product-focused firm, he noted, and the team likes to stick with companies until they can prove their scientific concept in human trials, even if that means investing in multiple financing rounds.

VenBio plans to use its newest fund to invest in 11 or 12 companies. Royston's interest areas? "The cardiometabolic area, unsurprisingly," he commented, as well as autoimmune and inflammatory disease treatments and RNA suppression technology.

politics

What Harris' potential VP picks say on health care

Kamala Harris is expected to announce her pick for vice president as early as this week. My colleague Sarah Owermohle takes us through the leading candidates and their track record on health policies.

One potential pick is Minnesota Gov. Tim Walz, whose administration implemented drug price transparency requirements and championed legislation that capped insulin prices for patients in emergency situations. This year, Minnesota reached a settlement with Eli Lilly that would cap insulin prices in the state for at least five years.

Read on to learn more about other top choices and their records.

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