BioSpace

In this episode, you'll be listening to  Akshay Rai, principal, Healthcare & Biotech Investments at Premji Invest and Viswa Colluru, CEO and founder, Enveda. They discuss how AI platforms must now prove themselves through data, focused pipelines and clinical readouts and that promises of faster, cheaper drug discovery are not enough to entice strong investor engagement. 
 
Host
Jennifer Smith-Parker, Director of Insights, BioSpace

Guests
Viswa Colluru, CEO & Founder, Enveda
Akshay Rai, Principal, Healthcare & Biotech Investments, Premji Invest

Disclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Biopharma’s weight loss warriors have led the news this week, with Structure Therapeutics and Rhythm Pharmaceuticals announcing new data. Structure delivered a “competitive profile” for its investigational GLP-1 pill compared to Eli Lilly’s orforglipron and Novo Nordisk’s oral Wegovy, BMO Capital Markets wrote to investors, after the candidate elicited 16.3% weight loss after 44 weeks in a Phase 2 trial. Rhythm was not as successful, announcing that Imcivree failed in a Phase 3 basket trial of various genetically driven obesities.
 
Meanwhile, Lilly and Novo made headlines outside of the clinical realm, as Lilly warned the public of the potential safety risks of taking compounded versions of tirzepatide—marketed as Zepbound for obesity and Mounjaro for type 2 diabetes. And Novo was hit with an FDA warning letter for failing to investigate reported adverse events—including three deaths—potentially linked to its own diabetes drug Ozempic. The letter only adds to what has been a challenging recent run for Novo, one that led to a 34% drop in total assets under management for its controlling shareholder Novo Holdings.
 
On the regulatory front, Robert F. Kennedy Jr.’s vaccine agenda is stalled after Massachusetts District Court Judge Brian Murphy found that his efforts to overhaul policies in this area were likely unlawful. Murphy specifically cited Kennedy’s move last June to empty the CDC’s Advisory Committee on Immunization Practices (ACIP). This comes on the heels of a White House crackdown in which officials would like the health secretary to tone down his vaccine skepticism, according to a report by The Wall Street Journal.
 
At the FDA, tensions continue to escalate, with Sen. Ron Johnson (R-WI) launching an investigation into recent rejections of rare disease drugs. And transparency issues have come to light once again regarding the circumstances around biologics chief Vinay Prasad’s imminent departure and recent request to remain anonymous during a media briefing about uniQure’s gene therapy for Huntington’s disease.

In this episode of Denatured, you'll listen to Oxana Iliach, senior director of regulatory strategy at Certara and Vera Pomerantseva, director of product management for risk-based quality management at eClinical Solutions. We speak about how the FDA's latest decision to have one, rather than two pivotal studies, for new drug applications raises the bar for data collection and risk-based management.

Host

⁠Jennifer C. Smith-Parker⁠, Director of Insights, BioSpace

Guests

Oxana Iliach, Senior Director of Regulatory Strategy, Certara

Vera Pomerantseva, Director of Product Management for RBQM, eClinical Solutions

Disclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

After Friday’s news that Center for Biologics Evaluation & Research Chief Vinay Prasad will leave the FDA—again—at the end of April, stocks for several rare disease drug developers popped. UniQure, in particular, was up 51% in premarket trading on Monday. Prasad in a meeting last Thursday with select journalists called the biotech’s Huntington’s treatment AMT-130 a “failed” therapy, according to STAT News. Shares of Replimune and REGENXBIO—which have suffered rejections during the past year—also rose.

One person who is not impressed with the plethora of rare disease drug rejections of late—H.C. Wainwright said in a note Tuesday that there have been at least five cell and gene therapies they believe could have been approved under prior FDA officials—is Wisconsin Senator Ron Johnson. Tuesday, Bloomberg News reported that Johnson has launched an investigation into these recent denials.

Johnson called the FDA’s request that uniQure conduct a sham surgery-controlled trial of AMT-130 “bureaucratic idiocy,” according to the publication. Meanwhile, uniQure and the FDA appear to be on different pages regarding the design of this prospective trial, with uniQure Chief Medical Officer Walid Abi-Saab referring to a 10-12 hour surgery during which [burr] holes would be drilled in patients’ skulls and Prasad claiming on a media call last week that it would require only “one to three nicks in the scalp.”

In other news, no episode of The Weekly would be complete without our weekly weight loss segment. Roche and Zealand Pharma’s amylin analog fell short of Eli Lilly’s rival candidate eloralintide; AbbVie reported what analysts called “competitive” results, with its amylin analog eliciting nearly 10% weight loss at 13 weeks in a Phase 1 trial; and Regeneron touted a much-needed Phase 3 win for Hansoh-partnered dual GLP-1/GIPR agonist olatorepatide in China. Beyond data, Novo Nordisk and Hims & Hers are together again, with Novo striking a deal to sell its injectable and oral GLP-1 medicines through the telehealth provider.

Elsewhere on the business side of biopharma, experts are reporting a cut-throat atmosphere behind doors on the M&A front as the supply of companies available to buy dwindles.

Stylus Medicine CEO Emile Nuwaysir and Chief Scientific Officer Jason Fontenot speak with BioSpace about the biotech's work on next-generation genetic medicines—and why they think it’s the perfect time to be in the cell therapy game.
Stylus Medicine is featured in BioSpace's  list, the top startups to watch in the U.S.

Host
Annalee Armstrong, Senior Editor, BioSpace

Guests
Emile Nuwaysir, CEO, Stylus Medicine
Jason Fontenot, Chief Scientific Officer, Stylus Medicine

Disclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.