387: Challenges for a crucial sickle cell treatment and the latest outlook for GLP-1s
Episode
28 min
Read time
2 min
Topics
Investing, Fundraising & VC, Leadership
AI-Generated Summary
Key Takeaways
- ✓GLP-1 Market Divergence: Novo Nordisk forecasts 5-13% sales decline versus analyst expectations of 3% decline, while Eli Lilly projects 23-27% growth and added nearly $100 billion in market cap in one day. Lilly's Zepbound shows superior effectiveness and gains market share, while Novo faces patent expirations outside the US and pricing pressure from direct-to-consumer channels requiring significant price cuts.
- ✓Oral GLP-1 Competition: Novo's oral semaglutide pill generates 50,000 prescriptions weekly at launch, primarily through direct-to-consumer channels priced below Lilly's planned oral offering. Despite food and drink restrictions, patient demand remains strong, with most users switching from compounded versions rather than expanding the total patient population. HIMS announces $50 monthly compounded oral Wegovy, forcing further pricing pressure.
- ✓Psychedelic Drug Approval Politics: FDA initially included Compass Pathways' psilocybin treatment for treatment-resistant depression on the national priority voucher list, but the White House removed it hours before announcement despite RFK's public support for psychedelics. Compass negotiates alternative rolling submission pathway with FDA, allowing faster review than traditional process while avoiding political controversy that could undermine field credibility.
- ✓Kascevi Manufacturing Crisis: Only 60 patients globally receive Vertex's CRISPR sickle cell therapy two years post-approval because patients require three to five hospital visits for stem cell collection, with some needing two-week hospitalizations during sickle cell crises. Abnormally shaped sickle cells disrupt centrifuge color indicators used for cell selection, and harsh gene-editing process damages cells, requiring multiple collection cycles.
- ✓CRISPR Competitive Threat: Beam Therapeutics develops competing sickle cell therapy launching next year with easier cell collection, gentler manufacturing process, and potentially superior effectiveness. Genetics (formerly Bluebird Bio) already treats more patients than Vertex using less harsh manufacturing. Analyst Eric Schmidt predicts Beam will capture market leadership, threatening Vertex's decade-long investment in CRISPR technology development.
What It Covers
This episode examines three major biotech developments: Novo Nordisk forecasts sales decline while Eli Lilly projects 23-27% growth in GLP-1 obesity drugs, Compass Pathways' psychedelic depression treatment gets removed from Trump's priority voucher program, and Vertex's CRISPR sickle cell therapy Kascevi faces severe manufacturing delays due to difficult stem cell collection.
Key Questions Answered
- •GLP-1 Market Divergence: Novo Nordisk forecasts 5-13% sales decline versus analyst expectations of 3% decline, while Eli Lilly projects 23-27% growth and added nearly $100 billion in market cap in one day. Lilly's Zepbound shows superior effectiveness and gains market share, while Novo faces patent expirations outside the US and pricing pressure from direct-to-consumer channels requiring significant price cuts.
- •Oral GLP-1 Competition: Novo's oral semaglutide pill generates 50,000 prescriptions weekly at launch, primarily through direct-to-consumer channels priced below Lilly's planned oral offering. Despite food and drink restrictions, patient demand remains strong, with most users switching from compounded versions rather than expanding the total patient population. HIMS announces $50 monthly compounded oral Wegovy, forcing further pricing pressure.
- •Psychedelic Drug Approval Politics: FDA initially included Compass Pathways' psilocybin treatment for treatment-resistant depression on the national priority voucher list, but the White House removed it hours before announcement despite RFK's public support for psychedelics. Compass negotiates alternative rolling submission pathway with FDA, allowing faster review than traditional process while avoiding political controversy that could undermine field credibility.
- •Kascevi Manufacturing Crisis: Only 60 patients globally receive Vertex's CRISPR sickle cell therapy two years post-approval because patients require three to five hospital visits for stem cell collection, with some needing two-week hospitalizations during sickle cell crises. Abnormally shaped sickle cells disrupt centrifuge color indicators used for cell selection, and harsh gene-editing process damages cells, requiring multiple collection cycles.
- •CRISPR Competitive Threat: Beam Therapeutics develops competing sickle cell therapy launching next year with easier cell collection, gentler manufacturing process, and potentially superior effectiveness. Genetics (formerly Bluebird Bio) already treats more patients than Vertex using less harsh manufacturing. Analyst Eric Schmidt predicts Beam will capture market leadership, threatening Vertex's decade-long investment in CRISPR technology development.
Notable Moment
The FDA placed Compass Pathways' psilocybin depression treatment on the priority voucher list, recognizing its potential for treatment-resistant depression affecting veterans, but conservative Trump advisers vetoed it just hours before the public announcement. This reveals tension between RFK's pro-psychedelic stance and political concerns about midterm elections, forcing the psychedelics field through traditional approval pathways.
Episode Transcript
Welcome to this week's episode of the Read Out Loud, a weekly biotech podcast from STAT. I'm Allison DeAngelis. I'm Adam Voorstein. And I'm Elaine Chen. It is Thursday, February 5. Adam's birthday for listeners. Two days. Mark your calendars. And on this week's show, we'll talk about a psychedelic drug from compass pathways that encountered a roadblock with the Trump White House. Two starkly different outlooks from Novo Nordisk and Eli Lilly. And a closer look at why manufacturing problems have slowed the rollout of a crucial sickle cell treatment. Hey, Read Out Loud listeners. Bob Herman here, STATS business of health care reporter and the writer behind the newsletter, HealthCare Inc. HealthCare Inc is a weekly newsletter devoted to unpacking the business and secret inner workings of The US health care industry. If you're someone who has ever received a medical bill or craves in-depth policy explainers or loves a playful meme now and again, I highly recommend you check this newsletter out. Learn more at the link in this episode's description. And now back to our regularly scheduled programming. Thanks. What what's everyone doing for the Super Bowl this weekend? I am probably going to my brother in law's house to watch it on his very, very large television, hopefully. It's bigger than mine. What are we eating? I don't know yet, actually. I need to figure that out. I've been planning because not only do we have guys the Super Bowl this weekend, but Olympic opening ceremonies are Friday. So I've got events for both of that. I need to figure out what food I'm bringing. I wanna know. Elaine, who are you rooting for in the Super Bowl? Definitely not rooting for the Patriots. Wow. I'm sorry. I'm sorry. But part of it is, because I'm rooting for my conference, and part of it is also because I, like to be on the other side of the game as you guys. Mhmm. Well, you know, I mean, I am in a house divided, essentially. Yeah. Because you're from Seattle, Allison. You have to support You have to support your hometown. Well So what are you so how are you dealing with is it that I grew up in the Seattle area, but also my partner's brother-in-law is from Seattle. So but then he, my partner, and his family are from New England. So we really are it's things are gonna get explosive this weekend. I don't know. I'm I'm I'm rooting for the halftime show in Bad Bunny. That's what I'm rooting for. You're not gonna watch the Turning Point USA alternative halftime show? You know, I have not followed Kid Rock's career. You're not a big you're not a big Kid Rock fan? You're not you're not, like, you're not craving another Kid Rock concert? No comment. Who do you guys think I should look for in the Super Bowl? The day that the Bears and the Patriots make the Super Bowl will be, …
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