Alnylam's Amvuttra enters ATTR-CM drug race

The FDA has approved Alynylam's RNAi therapy Amvuttra for treating transthyretin amyloid cardiomyopathy (ATTR-CM), positioning it to compete with Pfizer's Vyndamax and BridgeBio's Attruby. Priced at $476,000 annually, Amvuttra is significantly more expensive than BridgeBio's pill and may face access hurdles, particularly with Medicare Advantage.

Unlike its rivals, Amvuttra silences the gene producing unstable proteins rather than stabilizing them. Despite trial data showing efficacy, cross-trial comparisos remain inconclusive. Alnylam sees the launch as a turning point toward profitability, aiming to turn Amvuttra into its flagship product.

gene therapy

Is the gene therapy field in an overcorrection?

Gene therapy pioneer Jim Wilson argues that the field is suffering from "irrational pessimism," he said during STAT's Breakthrough Summit East. Companies are abandoning ship and approved therapies are going unprescribed, but that's an overrcorrection for the irrational exuberance for gene therapies a few years back, he said.

"I never would have thought that we would have been in a situation where there were FDA-approved products that were priced several million per dose, and no one's prescribing them. No one," Wilson said, referring to hemophilia therapies.

He has transitioned from academia to lead GemmaBio, focusing on cost reduction and partnerships with middle-income countries. And the biggest similarity between university labs and the biotech startup hustle? Chasing funding, he said.

glp-1 drugs

Cost and access hinder GLP-1 adoption

Although GLP-1 drugs offer tremendous potential for obesity and other health conditions, there's subtantial concern from employers over cost and patient adherence — and that's limiting broader use. Manufacturers like Novo Nordisk and Eli Lilly are lowering prices, but systemic issues in U.S. health care like inequity and coverage restrictions are major roadblocks, STAT's Ed Silverman writes.

"People don't stay on these medications and, partially, it's because they don't know how to stay on them," Included Health CEO Owen Tripp said, speaking at STAT's Breakthrough Summit East. "…That's why you hear me talk about an integrated approach [to health care], because it's only through an integrated approach that we can achieve the sorts of outcomes that we talk about that make these drugs really wonderful."

podcast

How obesity biotechs compete against Novo and Lilly

Why isn't Immunovant advancing the drug it just reported positive data on? And what did Jim Wilson have to say about that gene therapy pessimism?

We discuss all that and more on this week's episode of "The Readout LOUD," STAT's biotech podcast. We chat about positive data from Immunovant's autoimmune drug candidate, a patient death linked to Sarepta's gene therapy, and dispatches from our STAT Breakthrough Summit East in New York.

We also bring on Zealand Pharma CEO Adam Steensberg to talk about his company's amylin-targeting obesity treatment, the partnership deal it recently signed with Roche, and his thoughts on the industry's intense focus on weight loss.

Listen here.

china

Expedition Therapeutics brings Chinese drugs to U.S.

Expedition Therapeutics, a new startup backed by Venrock and BVF Partners, aims to license promising Chinese drug candidates for immune and inflammatory diseases and bring them to the U.S. Led by biotech veteran Yi Larson, the company plans to create independent subsidiaries around different batches of drugs, mirroring Roivant Sciences' strategy.

"China's really big. It is really hard to be truly comprehensive… I think one of the biggest challenges is making sure you find an asset that's truly differentiated, because generally, there are a lot of them," Yi told STAT's Allison DeAngelis.

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