Morning! As we speak, we now have loads of attention-grabbing 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 dearer than BridgeBio’s capsule and will face entry hurdles, notably with Medicare Benefit.
Not like its rivals, Amvuttra silences the gene producing unstable proteins somewhat 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 mentioned throughout STAT’s Breakthrough Summit East. Corporations 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 mentioned.
“I by no means would have thought that we might 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 mentioned, referring to hemophilia therapies.
He has transitioned from academia to steer 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 mentioned.
Value and entry hinder GLP-1 adoption
Though GLP-1 medication provide great potential for weight problems and different well being circumstances, 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.
“Individuals don’t keep on these drugs and, partially, it’s as a result of they don’t know how you can keep on them,” Included Well being CEO Owen Tripp mentioned, talking at STAT’s Breakthrough Summit East. “…That’s why you hear me discuss 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 discuss that make these medication actually great.”
How weight problems biotechs compete in opposition to Novo and Lilly
Why isn’t Immunovant advancing the drug it simply reported optimistic knowledge on? And what did Jim Wilson should 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 convey on Zealand Pharma CEO Adam Steensberg to speak about his firm’s amylin-targeting weight problems therapy, the partnership deal it not too long ago signed with Roche, and his ideas on the business’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 convey them to the U.S. Led by biotech veteran Yi Larson, the corporate plans to create unbiased subsidiaries round completely different batches of medication, mirroring Roivant Sciences’ technique.
“China’s actually huge. It’s actually arduous to be actually complete… I believe one of many greatest challenges is ensuring you discover an asset that’s actually differentiated, as a result of typically, there are plenty of them,” Yi informed STAT’s Allison DeAngelis.
Extra reads
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Blood check for ovarian most cancers misses some Black and Native American sufferers, examine 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