Morning! Immediately, we now have loads of fascinating content material from yesterday’s STAT Breakthrough Summit East in New York. Additionally, Alnylam wins an essential approval for ATTR-CM.
Alnylam’s Amvuttra enters ATTR-CM drug race
The FDA has authorized Alynylam’s RNAi remedy Amvuttra for treating transthyretin amyloid cardiomyopathy (ATTR-CM), positioning it to compete with Pfizer’s Vyndamax and BridgeBio’s Attruby. Priced at $476,000 yearly, Amvuttra is considerably costlier than BridgeBio’s capsule and should face entry hurdles, significantly with Medicare Benefit.
Not like its rivals, Amvuttra silences the gene producing unstable proteins somewhat than stabilizing them. Regardless of trial knowledge displaying efficacy, cross-trial comparisos stay inconclusive. Alnylam sees the launch as a turning level towards profitability, aiming to show Amvuttra into its flagship product.
Is the gene remedy subject in an overcorrection?
Gene remedy pioneer Jim Wilson argues that the sector is affected by “irrational pessimism,” he stated throughout STAT’s Breakthrough Summit East. Firms are abandoning ship and authorized therapies are going unprescribed, however that’s an overrcorrection for the irrational exuberance for gene therapies a number of years again, he stated.
“I by no means would have thought that we’d have been in a state of affairs the place there have been FDA-approved merchandise that had been priced a number of million per dose, and nobody’s prescribing them. Nobody,” Wilson stated, referring to hemophilia therapies.
He has transitioned from academia to guide GemmaBio, specializing in value discount and partnerships with middle-income international locations. And the largest similarity between college labs and the biotech startup hustle? Chasing funding, he stated.
Value and entry hinder GLP-1 adoption
Though GLP-1 medication provide large potential for weight problems and different well being situations, there’s subtantial concern from employers over value and affected person adherence — and that’s limiting broader use. Producers like Novo Nordisk and Eli Lilly are decreasing costs, however systemic points in U.S. well being care like inequity and protection restrictions are main roadblocks, STAT’s Ed Silverman writes.
“Folks don’t keep on these drugs and, partially, it’s as a result of they don’t know how one can keep on them,” Included Well being CEO Owen Tripp stated, talking at STAT’s Breakthrough Summit East. “…That’s why you hear me discuss an built-in method [to health care], as a result of it’s solely by an built-in method that we are able to obtain the types of outcomes that we discuss that make these medication actually fantastic.”
How weight problems biotechs compete in opposition to Novo and Lilly
Why isn’t Immunovant advancing the drug it simply reported constructive knowledge on? And what did Jim Wilson should say about that gene remedy pessimism?
We talk about all that and extra on this week’s episode of “The Readout LOUD,” STAT’s biotech podcast. We chat about constructive knowledge from Immunovant’s autoimmune drug candidate, a affected person loss of life linked to Sarepta’s gene remedy, and dispatches from our STAT Breakthrough Summit East in New York.
We additionally convey on Zealand Pharma CEO Adam Steensberg to speak about his firm’s amylin-targeting weight problems remedy, the partnership deal it lately signed with Roche, and his ideas on the trade’s intense give attention to weight reduction.
Expedition Therapeutics seeks to convey Chinese language medication to U.S.
Expedition Therapeutics, a brand new startup backed by Venrock and BVF Companions, goals to license promising Chinese language drug candidates for immune and inflammatory ailments and produce them to the U.S. Led by biotech veteran Yi Larson, the corporate plans to create impartial subsidiaries round totally different batches of medication, mirroring Roivant Sciences’ technique.
“China’s actually large. It’s actually laborious to be really complete… I feel one of many greatest challenges is ensuring you discover an asset that’s really differentiated, as a result of usually, there are plenty of them,” Yi instructed STAT’s Allison DeAngelis.
Extra reads
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Blood take a look at for ovarian most cancers misses some Black and Native American sufferers, research finds, STAT
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Eli Lilly launches weight-loss drug Mounjaro in India, beats Novo Nordisk to main market, Reuters
- Elevation drops sole clinical-stage ADC over poor section 1 knowledge, lays off 70% of employees, FierceBiotech
- Former CDC director Tom Frieden shares warning for RFK Jr. on measles outbreak, STAT
