The Readout Loud

We bring on UniQure CEO Matt Kapusta to discuss the company's announcement this week that its one-time treatment for Huntington’s disease significantly slowed down the neurological condition in a key study. We also chat about the Trump's administration initiatives on autism. Officials this week warned pregnant women not to take Tylenol, saying that it may cause autism, and they announced an FDA decision to make a decades-old drug called leucovorin available as a treatment for people with certain autism symptoms. To unpack the implications of these statements, we bring on by Ari Ne'eman, an assistant professor of health policy and management at Harvard’s school of public health who researches how policies affect people with disabilities.

Andrew Joseph, STAT's European correspondent, sheds light on significant pharma pullbacks from the U.K., revealing why giants like Merck and GSK are shifting their investments elsewhere. He analyzes the pricing tensions and the impact of U.S. investment strategies on the U.K. life sciences landscape. The discussion also covers Roche's bold $3.5 billion acquisition of 89bio, reflecting a trend of steady biotech M&A activity and improving market sentiment, providing listeners with a clear perspective on industry dynamics.

Mike Rea, a pharma consultant and head of Idea Pharma, shares insights from his personal journey through medical setbacks that reshaped his career. The conversation shifts to President Trump's proposed drug policies targeting China, stirring a debate on innovation versus protectionism. They also dive into the game-changing potential of the PD-1 VEGF inhibitor Ivanesimab, and discuss the leadership changes at Novo Nordisk, with implications for Alzheimer's research. Health challenges and the importance of patient advocacy in epilepsy treatment add a deeply personal touch.

The podcast dives into the swirling uncertainty surrounding COVID vaccine effectiveness and political narratives. It highlights the delicate balance health officials must strike amid public discontent and changing FDA guidelines. Meanwhile, there’s a noticeable rebound in the biotech sector, with optimism around upcoming IPOs and successful clinical trials. Enthusiastic discussions also spotlight significant advancements in sleep and lung disorder treatments, particularly at the World Sleep Congress and lung cancer meetings.

Chelsea Cirruzzo, Washington correspondent at STAT, joins the conversation to dissect the abrupt ouster of CDC Director Susan Monarez and the wave of resignations among senior officials amid vaccine policy tensions. They discuss the impact on public trust in health institutions and the shifting landscape of health policies. The dialogue also covers STAT's VC rankings report, highlighting the challenges and successes within biotech venture capital, including intriguing insights about fund performance relative to size.

This week, the podcast dives into the dramatic flop of Viking Therapeutics’ obesity drug trial, raising concerns about its treatment effectiveness and the biotech industry's fierce competition. The discussion shifts to the controversial 'pharma to table' trend, which promises direct sales to consumers but is unlikely to lower drug prices as hoped. Insights into cash pay pricing reveal the challenges faced by uninsured patients, while the impact of shifting to direct sales models on pharmaceutical pricing strategies is explored. Tune in for a fascinating take on these pivotal health industry topics!

Marissa Russo, a STAT fellow and PhD candidate at the Mayo Clinic focusing on glioblastoma, joins to delve into the hotly debated topic of alternatives to animal testing. She explores the innovative methodologies like organ-on-chip models and the push from the FDA and NIH towards more humane research practices. The conversation also touches on the complexities of these new approaches and the challenges in ensuring their reliability. Additionally, insights on the shifting landscape of mRNA technology add to the discussion's depth.

In this episode, Natalie Holles, former CEO of Third Harmonic Bio, shares her insights on the tough decisions behind dissolving the company and returning cash to shareholders. She discusses the challenges of biotech leadership amid failures, including Eli Lilly's disappointing obesity drug results and Replimmune's FDA rejection. Holles emphasizes the importance of transparency and data-driven approaches in the industry. The conversation wraps up with a humorous take on the challenges of balancing personal life with professional aspirations in biotech.

In this discussion, guests Brian Skorney, a senior research analyst at Baird, Ned Sharpless, former acting FDA commissioner, and Robert Califf, former FDA commissioner, delve into the impact of Vinay Prasad's departure from the FDA. They examine the political turbulence influencing FDA operations and the biotech sector, especially regarding drug approvals and leadership changes. The conversation highlights the challenges of regulatory integrity, the evolving landscape of gene therapies, and the urgent need for improved data collection in post-marketing drug safety.

Kaylin West, mother of 7-year-old Cooper with Duchenne muscular dystrophy, shares her powerful insights on Sarepta's decision to halt its gene therapy, Elevidys. She reflects on her family's journey through the hopeful yet tumultuous world of gene treatments. The podcast also delves into the emotional impact of such medical developments, highlighting the importance of community support for families navigating similar challenges. Alongside biotech innovations and new drug developments, Kaylin's personal story adds a poignant depth to the discussion.