With approval, Alnylam enters the ATTR-CM drug race
Morning! As we speak, we’ve got loads of attention-grabbing content material from yesterday’s STAT Breakthrough Summit East in New York. Additionally, Alnylam wins an vital approval for ATTR-CM.
Alnylam’s Amvuttra enters ATTR-CM drug race
The FDA has permitted 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 tablet and will face entry hurdles, notably with Medicare Benefit.
In contrast to its rivals, Amvuttra silences the gene producing unstable proteins reasonably than stabilizing them. Regardless of trial knowledge exhibiting 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 discipline 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 permitted therapies are going unprescribed, however that’s an overrcorrection for the irrational exuberance for gene therapies just a few 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 price discount and partnerships with middle-income nations. And the most important 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 super potential for weight problems and different well being situations, there’s subtantial concern from employers over price 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.
“Individuals don’t keep on these drugs and, partially, it’s as a result of they don’t know methods to keep on them,” Included Well being CEO Owen Tripp stated, talking at STAT’s Breakthrough Summit East. “…That’s why you hear me speak about an built-in strategy [to health care], as a result of it’s solely via an built-in strategy that we will obtain the types of outcomes that we speak about that make these medication actually great.”
How weight problems biotechs compete towards Novo and Lilly
Why isn’t Immunovant advancing the drug it simply reported optimistic knowledge on? And what did Jim Wilson must say about that gene remedy pessimism?
We focus on all that and extra on this week’s episode of “The Readout LOUD,” STAT’s biotech podcast. We chat about optimistic 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 carry on Zealand Pharma CEO Adam Steensberg to speak about his firm’s amylin-targeting weight problems remedy, the partnership deal it not too long ago signed with Roche, and his ideas on the business’s intense deal with weight reduction.
Expedition Therapeutics seeks to carry 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 illnesses and produce them to the U.S. Led by biotech veteran Yi Larson, the corporate plans to create unbiased subsidiaries round totally different batches of medicine, mirroring Roivant Sciences’ technique.
“China’s actually large. It’s actually onerous to be really complete… I feel one of many largest challenges is ensuring you discover an asset that’s really differentiated, as a result of typically, there are a number of them,” Yi advised STAT’s Allison DeAngelis.
Extra reads
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Blood check 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 part 1 knowledge, lays off 70% of workers, FierceBiotech
- Former CDC director Tom Frieden shares warning for RFK Jr. on measles outbreak, STAT
