November 5, 2024

The need-to-know this morning

Syndax Pharmaceuticals raised $350 million by selling a royalty on sales of its approved medicine Niktimvo to Royalty Pharma.
Vertex Pharmaceuticals said the first patient with sickle cell disease was treated with Casgevy, its CRISPR-based therapy, since the medicine was approved last December.
Merus said the FDA decision date for zenocutuzumab, a drug to treat lung and pancreatic cancer caused by a specific genetic mutation, was delayed to Feb. 4 from December. The agency requested extra time to review new manufacturing information.

gene editing

Patient dies in Beam trial of CRISPR sickle cell therapy

A patient with sickle cell disease died while participating in a clinical trial of a CRISPR-based treatment from Beam Therapeutics, the company said this morning.

The patient succumbed to respiratory failure, deemed to be "likely caused" by a regimen of chemotherapy required to prepare the patient for the treatment, called BEAM-101, which uses Beam's new, more precise form of CRISPR gene editing called base editing.

The fatality underscores the risks of one-time, genetic treatments for blood disorders like sickle cell and beta thalassemia, including those already marketed by Bluebird Bio and Vertex. The gene editing is 21st century, but it remains shackled to a highly toxic chemotherapy, called busulfan, approved in the 1950s.

Read more from STAT's Adam Feuerstein and Jason Mast.

politics

How the election results could affect biopharma

Ahead of the results rolling in, here are some resources on how each candidate could impact issues of concern to the biopharma industry.

The Stifel Biopharmaceutical Sector newsletter out yesterday predicts that Kamala Harris would be less likely to walk back Medicare's drug pricing authority granted through the Inflation Reduction Act, but stronger than Donald Trump on maintaining an independent FDA and blocking the exercise of march-in rights.

STIFEL BIOPHARMACEUTICAL SECTOR NEWSLETTER

I also recommend this piece from my colleague Adam Feuerstein last week. He wrote that if Harris wins, she'll likely maintain much of the status quo. But a Trump victory would open up a wide range of chaotic possibilities for the industry, especially if he actually allows Robert F. Kennedy Jr. to influence decisions about the FDA.

biotech

Vertex advances stem cell therapy for type 1 diabetes

Vertex said that following meetings with regulators in the U.S., U.K., and Europe, it's decided to advance its stem cell therapy for type 1 diabetes, converting an ongoing Phase 1/2 study to a Phase 1/2/3 study.

The trial will include 50 patients infused with a single dose of the treatment, called VX-880, Vertex said while reporting earnings yesterday. The primary endpoint is the proportion of patients who reach insulin independence without severe episodes of low blood sugar, or hypoglycemia.

In an update earlier this year on the Phase 1/2 study, Vertex said that 12 patients were able to produce enough of their own insulin to maintain healthy blood glucose levels 90 days later. All but one reduced or eliminated the need to inject insulin over those three months and three people followed for one year no longer needed to inject insulin at all.

Meanwhile, Sana also said yesterday that it will increase its investment in its type 1 diabetes stem cell therapy as it suspends the development of other programs.

obesity

Investors reevaluate this year's obesity darling

Viking Therapeutics, whose stock has soared this year on promising obesity results and its potential to be takeover target, reported new data at the Obesity Week conference yesterday. Detailed results of a Phase 1 trial of its oral peptide candidate showed that at the highest dose of 100 mg, patients experienced 6.8% placebo-adjusted weight loss in just one month.

(The treatment, oral VK2735, activates receptors of the GLP-1 and GIP hormones, similar to the mechanism of Eli Lilly's Zepbound.)

Shares of Viking initially spiked, but ended down for the day by over 13%. Though the treatment showed substantial and rapid weight loss, observers noted that it would be costly and time-consuming to manufacture large amounts of the drug at the 100-mg dose. Companies like Eli Lilly that are developing small molecule obesity pills have an advantage, as they're cheaper and simpler to manufacture than oral peptides.

vTFET-viking-falls-as-investors-reevaluate-obesity-darling(6)

Viking also showed detailed data of a Phase 2 trial of an injectable version of VK2735. In a subset of participants, people who received treatment maintained the majority of their weight loss four weeks after their final dose, supporting the possibility that the treatment could be dosed monthly for maintenance. If Viking can prove that in further studies, that could threaten the competitiveness of Amgen's monthly injectable obesity candidate.

GLP-1s

Hims & Hers sticks with compounding strategy

From my colleague Katie Palmer: Since Hims & Hers started prescribing compounded GLP-1s last May, the company has signed up roughly 50,000 subscribers for the weight loss drugs, according to the company's quarterly earnings. The direct-to-consumer telehealth company has been assertive that it will be able to continue selling the compounded versions of the on-patent drugs even when the shortages of branded versions come to an end — a strategy that could land the company in court, as we've previously reported.

That message didn't change on Monday, with CEO Andrew Dudum claiming that millions of patients could benefit from "the ability to hyper-personalize" doses and titration schedules to avoid side effects. (The company also said it would start offering liraglutide as its first generic GLP-1 in 2025.) As the FDA continues to assess whether Novo Nordisk's semaglutide and Eli Lilly's tirzepatide are in shortage, Dudum said the company is "actively communicating with federal and state legislative and regulatory bodies" to share customers' challenges trying to access the branded medications.

infectious disease

Peter Marks addresses recent vaccine skepticism

Amid a fresh wave of vaccine disinformation and skepticism, my colleague Helen Branswell spoke with Peter Marks, the top vaccine regulator for the FDA.

He discussed the extensive safety data that drug manufacturers have to collect before bringing vaccines to market, trial designs for vaccines, and post-marketing surveillance.

Being transparent "is the right thing to do," he said. "And probably the most important thing to do is to try to continue to educate people that there are small risks with vaccines but those small risks are greatly outweighed by their potential benefits," he said.