November 14, 2024

The need-to-know this morning

In a move to safeguard the company’s dominant position in cancer, Merck said it will license a new cancer drug from LaNova Medicines, a Shanghai-based firm, for $588 million upfront and as much as $2.7 billion in potential milestone payments.

politics

GOP-controlled Congress gives Trump broad power

Now that it's been determined that Republicans will have full control of the House and Senate, President-elect Trump will have broad power to assert his will over health care policies.

Major health care issues are potentially at stake, including subsidies for Affordable Care Act plans, Medicaid funding, restructuring of massive federal agencies like the FDA and CDC, access to telehealth, drug middlemen reforms, drug price negotiations, and China’s rise in biotechnology.

Democrats are in a weak position to push for their preferences on many of these issues, though Congress’ arcane rules and splits within the GOP could potentially give the party at least some openings. In the Senate, 60 votes are needed to overcome a filibuster, and Republicans are short of that number.

biotech

Looking ahead to Vertex's high-stakes pain readout

Vertex Pharmaceuticals' next quest is to transform the treatment of pain, and a pivotal milestone is fast approaching.

The biotech plans to announce results by the end of the year from a mid-stage trial of an experimental treatment for patients with lumbosacral radicular syndrome, more commonly known as sciatica.

Analysts have framed this trial as a major moment for the company, and the stakes are high. My colleague Jonathan Wosen breaks down the background and design of the trial, as well as expectations for the results.

politics

CDC, FDA officials warn of anti-vaccine consequences

Two Biden administration officials — CDC Director Mandy Cohen and the FDA's top vaccine regulator, Peter Marks — warned yesterday that there could be serious consequences for the country's children if anti-vaccine views prevail.

The comments, made at separate conferences yesterday, came as the country waits to see who will fill key health positions in the new Trump administration and how much sway anti-vaccine figures like Robert F. Kennedy Jr. might exert.

“The natural consequences of not believing in science or the potential benefit of these vaccines may be that we have unnecessary deaths,” Marks said. “I’m sorry to say that. I hope it doesn’t have to come to that, but it seems like that is where we are.

Read more from STAT's Helen Branswell and Anil Oza.

gene therapy

Tune Therapeutics to launch HBV gene-editing trial

From my colleague Jason Mast: Tune Therapeutics will soon begin human testing of a gene-editing therapy for hepatitis B, the startup announced today. It’s the second company to obtain regulatory clearance for such a study, after Precision Biosciences said in October it would start the first ever trial of a gene editing treatment for HBV.

These efforts come over a decade after researchers first proposed that gene editing could eventually provide a cure for a chronic virus that still infects over 300 million people worldwide. The virus has evaded other treatment attempts by forming stable DNA loops in liver cells or integrating into human DNA, beyond the reach of conventional approaches.

Both Intellia Therapeutics and Beam Therapeutics toyed with HBV programs before ultimately shelving them. Precision Biosciences is trying to cut out of patients’ liver a sliver of DNA, using a DNA-cutting enzyme found in algae. Tune is not cutting DNA at all. Instead, the company uses an approach called epigenetic editing, where researchers can use certain enzymes to suppress or activate genes without breaking them.

Neither Precision nor Tune are starting their trials in the U.S., where CRISPR companies have often complained regulators are too stringent in their preclinical requirements. Tune is beginning in New Zealand. Precision has decided to study its therapy in Moldova. If successful, both are likely to then apply to begin studies in the U.S.

obesity

Amgen defends obesity drug amid bone concerns

And to close the loop on this — if you recall, on Tuesday, a research note raising potential safety concerns with Amgen's lead obesity candidate triggered the company's shares to plummet, erasing $12 billion in market value.

The note was based on data found in hidden tabs of a file attached to a publication of early trial results for the drug, called MariTide. The tabs contained what appeared to be data showing study participants experiencing loss of bone mineral density, the note said.

Amgen's head of development yesterday defended the drug at an investor conference, saying the data tables referenced by the note were not finalized and were not subject to standard review, so the company has asked the journal to issue a correction and add in the finalized data.

There were overlapping margins of error between treatment and placebo groups, leading researchers to conclude there was no association between the MariTide and bone density changes, he added.

Observers have hypothesized that MariTide may risk bone density loss due to its mechanism of blocking receptors of the GIP hormone. But the Amgen executive said the company has studied genetic data, and it did not find a signal that genetic variants associated with reduced GIP receptor activity were linked to bone density reduction or bone diseases.

Read all about this saga here.

Adam's Biotech Scorecard

Neurogene and the race to the bottom of gene therapy

From my colleague Adam Feuerstein: Pardon the cranky rant, but I believe companies developing genetic medicines for even the rarest and most severe diseases should be conducting randomized, placebo-controlled clinical trials. Anything less is scientifically irresponsible and shows a lack of respect for patients and their families. These therapies are irreversible and carry substantial risks, so the benefits better be clearly demonstrated.

Yet at Neurogene, the developer of a gene therapy for Rett syndrome, expediency seems to be the priority. The company is collecting the least amount of data possible from single-arm, open-label studies that, by definition, will not demonstrate convincing proof of efficacy or safety.

Randomized, placebo-controlled studies are the gold standard for determining conclusively the benefits and risks of an experimental treatment. Yet, gene therapy developers are carving out an exception, claiming it’s too difficult, logistically or ethically, to ask patients to participate in such scientifically rigorous trials.