Pfizer's Lyme data has an awkward statistical miss

Pfizer and Valneva's Lyme disease vaccine looks meaningfully effective, cutting cases by about 73% in a large Phase 3 trial. But the inoculation failed its primary endpoint: The confidence interval came in below the pre-specified threshold, a miss that could complicate regulatory review despite the overall data and safety profile looking solid, STAT's Matthew Herper writes.

"I'd like them to explain the difference," said Paul Offit, the director of the Vaccine Education Center at Children's Hospital of Philadelphia. "It's odd."

With no Lyme vaccine currently available and prior efforts derailed more by perception than data, regulators now face a familiar dilemma: how strictly to enforce statistical rules when the clinical signal is strong, especially amid broader turmoil at FDA and CDC advisory bodies that could make this one of the more closely watched vaccine decisions in the near term.

supply chain

Middle East conflict could strain drug supply chains

The escalating conflict in the Middle East is threatening to rattle global pharmaceutical supply chains. While the region accounts for less than 1% of global drug output, key shipping lanes and air corridors are increasingly being disrupted, write STAT's Ed Silverman and Annalisa Merelli. The war has also sparked rising fuel costs, while new pressures are being brought to bear on cargo capacity.

Manufacturers in India and the EU, which are particularly those reliant on transit through the Strait of Hormuz, may face higher logistics costs that could ultimately be passed on to patients, especially for low-margin generics that India supplies at scale.

The drugs that are "most at risk are biologics, such as blood products and live vaccines, which often have extremely short shelf lives and must be transported under tightly controlled temperature conditions," one supply chain expert told STAT. "Any delay or break in the cold chain can render them unusable."

dermatology

Apogee eczema drug seems more durable than existing blockbusters

Apogee Therapeutics said this morning that its experimental eczema antibody, zumilokibart, delivered skin and itch relief on par with blockbuster rivals while requiring far fewer injections, mid-stage data show. The results look broadly comparable to Regeneron and Sanofi's Dupixent and Eli Lilly's Ebglyss, STAT's Adam Feuerstein writes, except that both of those drugs are dosed more frequently.

Phase 3 trials for Apogee's medicine, an IL-13 blocker engineered for longer activity, are planned later this year.

"These results are kind of a home run," Apogee CEO Michael Henderson told STAT, adding that "the open question was: Could we convert non-responders to become responders as well? That's something Dupixent cannot do. What we saw there was pretty eye-popping."

chronic disease

Ron Cohen to helm startup focused on Parkinson's

From STAT's Jason Mast: Ron Cohen, the longtime CEO of Acorda Therapeutics, is back at the helm of a new startup: Oryon Cell Therapies. Built out of research from Harvard and Mass General Brigham, Oryon is developing stem cell-derived neurons designed to replace those lost in patients with Parkinson's. The company says it has received $42 million in funding between investments and grants.

It presented data Saturday at the International Conference on Alzheimer's and Parkinson's Diseases in Copenhagen, showing encouraging results from the first five patients. Cohen, who oversaw the development of neurological drugs at Acorda for nearly three decades before selling its assets for $185 million as part of a 2024 bankruptcy sale, will have his work cut out for him.

Two other companies, BlueRock Therapeutics and Aspen Neuroscience, have started or are planning pivotal trials for neuron-replacement treatments in Parkinson's. But Cohen said he sees a chance for a superior treatment. Oryon creates its cells from a different source — a patient's own T cells' — and develops them into more mature neurons than Aspen and BlueRock.

lung disease

Insmed's Arikayce works earlier in bacterial lung disease

Phase 3 data from Insmed suggests its drug Arikayce can improve respiratory symptoms in patients with a rare, bacterial lung disease called mycobacterium avium complex who haven't received antibiotics. Arikayce was granted accelerated approval in 2018 for patients with this disease who were unresponsive to antibiotics. The company said it'll submit the new results to the FDA to convert the accelerated approval into a full one, STAT's Adam Feuerstein writes.

If that happens, Leerink analysts said, that could boost peak sales of the drug over $1 billion.

podcast

A conversation with the 'godfather' of biotech

On the most recent episode of "The Readout LOUD," we bring you a special conversation with Stelios Papadopolous, also know as the "godfather" of biotech. He sat down for an on-stage chat with our colleague Damian Garde Thursday afternoon during STAT's Breakthrough Summit East event in New York City.

We also chat about the latest biotech news, including new weight loss data from Structure Therapeutics and Eli Lilly, plus recent staff departures from the Food and Drug Administration.

Listen here.

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