October 14, 2025

The need-to-know this morning

Biocryst Pharmaceuticals is acquiring Astria Therapeutics in a cash-and-stock deal with a $700 million enterprise value. The centerpiece of the transaction is Astria's long-acting injectable drug in late-stage clinical development to treat hereditary angioedema, or HAE, a serious tissue-swelling disorder. Biocryst markets a pill for HAE.
Kailera Therapeutics, a privately held developer of drugs for obesity, raised $600 million — one of the largest private financings this year.
Denali Therapeutics said the FDA extended by three months a review of its treatment for Hunter syndrome, a rare genetic disorder. The approval decision date is now April 5, 2026.
Shah Capital, the second-largest shareholder of Covid vaccine maker Novavax, sent a letter to the company's board, urging them to considering selling the company in order to boost shareholder value.
Johnson & Johnson kicked off third-quarter earnings season and announced a plan to separate its orthopedics business, but the diversified healthcare giant did not announce a rumored acquisition of Protagonist Therapeutics.

EXCLUSIVE

FDA scientists say politics are increasingly overshadowing data

Tension are escalating between career scientists and political leaders at the Food and Drug Administration as health secretary Robert F. Kennedy Jr. works through his agenda.

My colleague Lizzy Lawrence spoke with more than 20 current and former agency and officials, as well as legal experts, for a deeply reported look at the issue. The conversations offered new insights into the political pressure shaping some of the FDA’s most important actions this year, from tackling autism to revisiting vaccine safety.

Among the findings: FDA career scientists resisted when officials proposed GSK's shelved drug leucovorin be approved as a treatment for autism. Eventually, the FDA's top drug regulator, George Tidmarsh, came to a compromise with staff — agreeing to ask GSK to instead submit an application for cerebral folate deficiency, a rare neurological disorder that can have overlapping symptoms with autism. But still, when talking publicly about leucovorin, FDA Commissioner Marty Makary touted it as an autism treatment.

Read more.

regulation

Novo's Indiana plant deemed out of compliance

A troubled contract manufacturing plant recently acquired by Novo Nordisk is considered to not be in compliance with the FDA, a serious designation that could further delay the approval of drugs made in the facility, including some from Scholar Rock and Regeneron Pharmaceuticals.

STAT previously reported that an FDA inspection of the plant over the summer found a litany of issues, some related to cat hair, pests, and equipment failures. Last week, the agency told Novo that the plant is classified as “official action required,” a regulatory term meaning the site is in an unacceptable state of compliance.

The plant was previously owned by Catalent, but Novo acquired it as part of its parent company’s acquisition of Catalent last year.

biotech

Regeneron to submit gene therapy for hearing loss after positive data

Over the weekend, Regeneron published updated data on its investigational gene therapy for a type hearing loss that supports its plan to submit the treatment for approval later this year.

In a trial of 12 participants with hearing loss due to variants of the otoferlin gene, nine patients reached a level of hearing typically deemed to no longer require cochlear implants. Three patients achieved normal hearing levels, according to the results, published in the New England Journal of Medicine.

politics

AstraZenca is second pharma company to announce drug-pricing deal with Trump

Late last Friday, President Trump announced his second "most-favored nations" deal, this time with AstraZeneca.

As was the case with his first deal, with Pfizer, the British pharma company agreed to give the U.S. Medicaid program drug prices that are in line with what it offers other major developed countries and to launch new drugs at so-called most-favored nation prices. In exchange, AstraZeneca will also get a three-year reprieve on national security-related pharmaceutical tariffs.

Though Trump officials and AstraZeneca CEO Pascal Soriot touted the agreements a way to create huge savings for patients and the government, drug pricing experts say these kinds of deals will not meaningfully affect drug costs and budgets.

AstraZeneca will also list some primary care drugs on Trump’s new direct-to-consumer site, TrumpRx.gov. In a graphic (below) shown at the White House event announcing the deal, Trump's team said that there will be discounts up to 654% — a mathematical impossibility considering patients are not being paid to take the drugs. Meanwhile, AstraZeneca's press release said it will offer the drugs at cash prices up to 80% lower than the list prices.

Daniel Payne

M&A

J&J reportedly in talks to buy Protagonist

The Wall Street Journal reported on Friday that Johnson & Johnson is in talks to buy Protagonist Therapeutics.

The biotech's stock surged 30% on Friday after the report and the company closed out the week with a market cap of $5.4 billion.

J&J is already partnered with Protagonist to develop an oral treatment for immune diseases including plaque psoriasis and ulcerative colitis. Protagonist also has another drug in its pipeline, rusfertide, to treat a rare blood cancer. The drug, which the biotech is working with Takeda on, showed positive results in a pivotal trial.

gene therapy

Sarepta to seek approval of drug for ultra-rare form of muscular dystrophy

An experimental gene therapy from Sarepta Therapeutics increased levels of the gene missing in an ultra-rare form of muscular dystrophy called limb-girdle muscular dystrophy (LGMD) 2E, according to data the company presented last Friday.

Sarepta now plans to submit the therapy to regulators for review. If cleared, it would be the first approved treatment in LGMD, a broad collection of highly rare muscular diseases. But the company is likely to face a significant uphill battle.

The therapy relies on the same gene-ferrying virus that Sarepta uses in its other treatments, including its controversial approved treatment for Duchenne muscular dystrophy.