May 15, 2023

The need-to-know this morning

Sarepta Therapeutics' stock price rose 26% to $152 in pre-market trading, following the decision Friday by an FDA advisory panel to recommend the accelerated approval of the company's gene therapy for Duchenne muscular dystrophy.
Athenex, a developer of cell therapies for cancer treatment, filed for Chapter 11 bankruptcy protection. The company has reached an agreement with its lenders to pursue a sale of its assets.
Verve Therapeutics said VERVE-101, its PCSK9-targeted gene-editing treatment for a common cause of heart disease, remains on an FDA clinical hold, although an early-stage study conducted outside the U.S. continues. The company also unveiled a second gene-editing treatment, called VERVE-102, against the same PCSK9 target.

vaccines

A struggling Novavax wants governments to pay up

These days, vaccines for Covid-19 just aren't much in demand. That's problematic for companies like Novavax, which dedicated vast resources to creating jabs that might help curb the pandemic. Now, Novavax's new CEO, John Jacobs, is urging governments to honor vaccine purchase commitments, as it struggles to stay afloat, Financial Times writes.

"We had to invest billions … to deliver a vaccine that helped to stem the tide of a global pandemic that was killing millions of people," Jacobs said. "And then to all of a sudden say: 'Well you spent all your money, you committed to protect our citizens. Now we don't think we need it as much. Sorry, you're out of luck.' That's probably not good for long-term relationships and that type of thing and to benefit public health."

Countries have set up $2.1 billion in advanced purchase agreements with Novavax, Jacobs said, stretching to 2025 — but there's no guarantee that governments will honor these deals. The company's also in the midst of a $700 million arbitration process linked to it canceling a vaccine contract with Gavi, and said it would repay the U.K. government $112.5 million after it decided not to buy additional vaccine doses. The company's current liabilities are around $2 billion, and it only has about $637 million cash in hand.

nonprofits

Eric Lander's ambitious plan with Science for America

Last year, the storied scientist Eric Lander quietly launched Science for America, a nonprofit that unites researchers and technologists so they can work together on the biggest issues that threaten humanity. Its mission is to address existential issues like cancer, climate change, pandemic preparedness, and how research is conducted in the U.S.

"I think it cuts to the heart of what scientists want to do, which is to go after big problems, but they often don't always get a chance to do that in traditional industrial or academic or government settings," Lander told STAT.

SfA, as it's known, has been granted $30 million over two years from philanthropic bigwigs like Gates Ventures, Emerson Collective, Bloomberg Philanthropies, and Schmidt Futures. The money is meant to help great minds collaborate on solving specific problems. For instance, one focus is retooling how drugs are developed for cancer patients — addressing the bottlenecks in clinical trials that make testing difficult.

approvals

A new, nonhormonal drug approved for hot flashes

Veozah, a menopause drug developed by Astellas, has been approved by the FDA. It's the first nonhormonal drug approved to treat hot flashes and night sweats. That's key, because many people undergoing menopause can't take hormonal therapies — and few treatments have been available for women who experience these symptoms.

"When you think about the impact of vasomotor symptoms on work, on cognitive function, on sleep, on quality of life — the availability of another option is exciting," Lauren Streicher, medical director of the Northwestern Medicine Center for Menopause, told the New York Times. "This is something we've been anticipating for a long time."

The approval is based on three Phase 3 trials that tested Veozah in more than 3,000 women with moderate or severe hot flashes. The pill, when taken once daily, significantly reduced these symptoms compared to placebo. Certain neurons in the brain run amok when women enter menopause and their estrogen levels fall. The drug contains a compound called fezolinetant, which binds to these neurons and "calms them down," Streicher said.

shortages

Can compounding pharmacies fill supply chain gaps?

When medical supplies were dwindling at the start of the Covid pandemic, the FDA temporarily allowed compounding pharmacies to swoop in and fill in the gaps — creating 13 drugs for the virus to meet hospitals' needs. Now, there are other gaps in the medicinal supply chain. For instance, over-the-counter ibuprofen and acetaminophen for children are in short supply.

This could be another instance where compounding pharmacies could save the day, opines Scott Brunner, CEO of the Alliance for Pharmacy Compounding. But they're awaiting special action and guidance from the FDA, which hasn't been issued.

"Changes to federal law are required so that when shortage drugs are urgently needed, compounders may assist immediately, without bureaucratic delays and impediments," he writes.