From Two Trials to One, Sponsors Face a Higher Standard

In this episode of Denatured, you'll listen to Dr. Marc Hedrick, President and CEO of Plus Therapeutics and Dr. Phil Kantoff, CEO & Co-founder of Convergent Therapeutics. They discuss breakthroughs like alpha-emitting isotopes, supply chain solutions and why radiotherapeutics is biotech's next big frontier.Host Jennifer C. Smith-Parker, Director of Insights, BioSpace Guests Marc Hedrick, President & CEO, Plus Therapeutics Philip Kantoff, Co-founder & CEO, Convergent TherapeuticsDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations. 

Biogen, Eli Lilly and Merck spent more than $20 billion in the past week to swallow biotechs with approved products or promising drug candidates—representing three of this year’s four biggest takeovers. First, Merck picked up Terns Pharmaceuticals and its mid-stage leukemia drug for $6.7 billion. Then, on Tuesday, Lilly and Biogen struck, acquiring Centessa Pharmaceuticals and Apellis Pharmaceuticals respectively.Those big-ticket deals aren’t the only recent transactions, however. Others include Novartis’ up to $2 billion pick up of Excellergy and Gilead’s $2.1 billion purchase of Ouro Medicines. Meanwhile, Kevin Tang—the newly minted CEO of Aurinia Pharmaceuticals—again has his sights set on Kezar Life Sciences, which he previously targeted in 2024. This time, biopharma’s “clean-up” man is offering $50 million for the chronic disease–focused biotech.Last week also saw nods for Denali Therapeutics’ Avlayah, the first treatment for Hunter syndrome to target the disease’s neurological complications, and Rocket Pharmaceuticals’ Kresladi for leukocyte adhesion deficiency-I. And it’s been a big week for Biogen, which besides moving on Apellis, won approval of a high-dose formulation of spinal muscular atrophy drug Spinraza and scored a Phase 2 win for lupus candidate litifilimab in cutaneous lupus erythematosus.On the weight loss front, Wave Life Sciences’ stock was cut in half after its obesity candidate WVE-007 failed to impress investors in a Phase 1 trial.

In this episode, you'll be listening to Sergey Jakimov, managing partner at LongeVC and Artem Trotsyuk, operating partner, US, LongeVC. We discuss how developers in the longevity space should stop chasing aging as an abstract target and concentrate on specific mechanisms that can clearly tackle age-related conditions. HostJennifer C. Smith-Parker, Director of Insights, BioSpaceGuestsSergey Jakimov, Managing Partner, LongeVCArtem Trotsyuk, Operating Partner-US, LongeVC 

Immunology & inflammation stole the show this week, as Gilead dropped up to $2.1 billion for Ouro Medicines and its T cell engager OM336—an investment it hopes to split with longtime partner Galapagos. Meanwhile, Sanofi also added a T cell engager in a licensing deal with Kali Therapeutics worth $180 million upfront.One I&I partnership that bore fruit last week was that of Johnson & Johnson and Protagonist Therapeutics which got their IL-23 receptor blocker Icotyde across the FDA finish line for plaque psoriasis. Icotyde is the first commercial product for Protagonist.Even the drama this week came from the I&I space, as Kevin Tang took the reins as CEO of Aurinia Pharmaceuticals, which you may remember was the company at the heart of former Center for Drug Evaluation and Research Director George Tidmarsh’s exit from the FDA.Outside of I&I, the CDC’s Advisory Committee on Immunization Practices made headlines once again as Vice Chair Robert Malone posted to social media that the group was being disbanded. The Department of Health and Human Services quickly corrected the record, but Malone posted a few hours later that “dissolving and reforming remains one of options being considered.”In FDA policy news, BioSpace recaps the now nine-month-old Commissioner’s National Priority Voucher pilot program and unpacks new draft guidance on animal testing alternatives. Plus, check out an opinion article on the need for precision ALS drugs and features on gene therapy for hearing loss and Big Pharma’s overall R&D spending in 2025.

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.  HostJennifer Smith-Parker, Director of Insights, BioSpaceGuestsViswa Colluru, CEO & Founder, EnvedaAkshay Rai, Principal, Healthcare & Biotech Investments, Premji InvestDisclaimer: 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.Host⁠Jennifer C. Smith-Parker⁠, Director of Insights, BioSpaceGuestsOxana Iliach, Senior Director of Regulatory Strategy, CertaraVera Pomerantseva, Director of Product Management for RBQM, eClinical SolutionsDisclaimer: 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.HostAnnalee Armstrong, Senior Editor, BioSpaceGuestsEmile Nuwaysir, CEO, Stylus MedicineJason Fontenot, Chief Scientific Officer, Stylus MedicineDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

In this Denatured episode, Jennifer C. Smith-Parker speaks to Ram May-Ron, managing partner at FreeMind Group, and Ravi Kiron, managing director at Biopharma Strategy Advisors. We'll discuss how best to tailor an investment approach of both nondilutive funding and family offices to overcome the drug development valley of death. Host⁠Jennifer Smith-Parker⁠, Director of Insights, BioSpaceGuestsRam May-Ron, Managing Partner, FreeMind GroupRavi Kiron, Managing Director, Biopharma Strategy AdvisorsDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.