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.  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.

The FDA is dominating the headlines once again thisweek.  Days after FDA Commissioner Marty Makary appeared to question uniQure’s gene therapy candidate for Huntington’s disease, the company revealed that the agency will require it to conduct a randomized, double-blind, sham surgery–controlled Phase 3 study. The FDA also published anothercomplete response letter (CRL), this one for REGENXBIO’s gene therapy for Hunter syndrome. The rejection, sustained by the biotech early last month, was driven by issues with the study’s population, controls and use of surrogate markers to measure efficacy, according to the document.  Meanwhile, regulatory experts have expressed concernsthat the FDA’s circle of trust is shrinking, making many decisions feel like “fiat”—both in terms of individual drug applications and policy. The FDA has reportedly initiated a probe into complaints that a toxic workplace is fostered by CBER director Vinay Prasad, who is at the heart of many of these decisions. Finally, the biopharma industry continues to react to the agency’s pivot from a requirement of two pivotal trials to one for approval, asking why now, what are the risks and what exactly the FDA expects from this one trial.   Still on the gene therapy front, Sarepta Therapeutics CEO Doug Ingram stepped down last week to spend more time with family as the company’s muscular dystrophy mission hits home. Also during the company’s fourth quarter earnings call, Sarepta projected that sales of its embattled Duchenne muscular dystrophy gene therapy Elevidys will be flat or down as far as 15% in 2026.  On the obesity front, Eli Lilly topped Novo Nordisk again in a weight loss trial, this time in a Lilly-sponsored study of patients with type 2 diabetes. But don’t count Novo out yet. The company is actively seeking out new obesity assets, according to business development executive Tamara Darsow. Just last week, Novo linked with Boston’sVivtex to advance novel weight loss pills.Finally, check out BioPham Executive this week for a rundown of 2025’s top-selling assets—spoiler: Merck’s Keytruda held onto its crown as number one—and a story on former2seventy exec Chip Baird’s new role as CEO of recently launched Poplar Therapeutics, which secured a $45 million series A extension this week.

In this episode, you'll be listening to Indu Navar, CEO and founder of EverythingALS and Dr. Olga Uspenskaya, chief medical officer at VectorY Therapeutics. We'll be speaking about patient-pharma collaborations accelerating trials and hope, advances in ALS biology understanding and biomarker-driven endpoints.HostJennifer Smith-Parker, Director of Insights, BioSpaceGuests⁠Indu Navar⁠, CEO and Founder, EverythingALSDr. Olga Uspenskaya, Chief Medical Officer, VectorY TherapeuticsDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Eli Lilly notches another win over Novo Nordisk, as Zepbound bests CagriSema in a head-to-head trial sponsored by Novo; The FDA kicked off Rare Disease Week, providing draft guidance on its new plausible mechanism pathway, while a bipartisan senate hearing on Thursday will focus on the authorization process for rare conditions; Another leadership change shakes up CDC; and Gilead acquires CAR T partner Arcellx for nearly $8 billion.  Everything is coming up Lilly. The Indianapolis-based pharma bested its chief rival, Novo Nordisk in a head-to-head test. In a Phase 3 trial initiated by Novo itself, Lilly’s Zepbound generated 25.5% weight loss while the Danish pharma’s CagriSema elicited 23%. The results sent Novo’s shares plummeting by an unprecedented 20% to a pre-Wegovy valuation while Lilly’s market cap continues to climb.   Novo attempted a comeback on Tuesday, announcing that its triple-G agonist UBT251 scored almost 20% weight loss after 24 weeks in a Phase 2 trial in China. By comparison, Lilly’s own triple-G competitor retatrutide led to 17.5% weight loss over the same timeframe, according to BMO Capital Markets analysts. Novo also sweetened the pot, announcing that it would slash the prices for all three of its GLP-1 medicines starting in 2027.    Meanwhile, the FDA kicked off Rare Disease Week with draft guidance on the new Plausible Mechanism Pathway for personalized therapies that was first teased in November. Jumping off last summer’s Baby KJ success story, the new pathway is aimed at advancing treatments for ultra-rare diseases. And a bipartisan senate hearing on Thursday will focus on the authorization process for rare disease therapies.  While the rare disease space has enjoyed recent regulatory progress, funding these vital therapies remains a challenge. Companies like the Orphan Therapeutics Accelerator (OTXL), a non-profit biotech, are trying to change this with creative approaches including tax exempt status and unique partnerships with CDMOs and CMOs. Finally, in a move that also has implication for the rare disease space, the FDA’s official pivot from a two clinical trial requirement to just one for new drug applications is lighting up biopharma social media.   And over at the CDC, there is more upheaval on the leadership front as National Institutes of Health Director Jay Bhattacharya replaces acting director Jim O’Neill as head of the agency, and principal deputy director Ralph Abraham steps down, citing “unforeseen family obligations.”    On the business front, Gilead inked the biggest M&A deal of the year so far, acquiring CAR T partner Arcellx for nearly $8B. And Merck’s Keytruda should have a few extra years of dominance thanks to a web of patents, with billions on the line. Check it out in BioPharm Executive, in your inboxes Wednesday.  HostsJef Akst, Managing Editor, BioSpaceHeather McKenzie, Senior Editor, BioSpaceAnnalee Armstrong, Senior Editor, BioSpace

Corsera Health’s Chief Operating Officer Rena Denoncourt and CFO Meredith Kaya speak with BioSpace about the biotech’s mission and vision for the next generation of cardiovascular care in this special bonus episode. Corsera Health is featured in BioSpace's NextGen: Class of 2026 list, the top startups to watch in the U.S.HostAnnalee Armstrong, Senior Editor, BioSpaceGuestsRena Denoncourt, Chief Operating Officer, Corsera HealthMeredith Kaya, Chief Financial Officer, Corsera HealthDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

In this episode, you'll be listening to Jane Hughes, President of R&D and Co Founder of Verdiva Bio, and Jon Rees, CEO and cofounder of MitoRx Therapeutics. We'll discuss next-generation obesity solutions tackling GLP-1's muscle loss and adherence challenges, through innovative muscle preservation, oral administration and combination therapy.HostJennifer Smith-Parker, Director of Insights, BioSpaceGuestsJane Hughes, President of R&D and Co-founder, Verdiva BioJon Rees, CEO and Co-founder, MitoRx TherapeuticsDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Last week, the FDA declined to review Moderna’s mRNA-based flu vaccine, with Center for Biologics Evaluation and Research Director Vinay Prasad himself signing the refuse-to-file (RTF) letter. Prasad said Moderna’s comparator group “does notreflect the best-available standard of care,” while Moderna pushed back, saying CBER had previously found a standard-dose flu shot to be an “acceptable” comparator. Despite the shocking turn of events, HHS backed Prasad, saying Moderna didn’t “follow very clear FDA guidance from 2024.”Regardless of the reasons, the development imperilsModerna’s breakeven plans, affecting not just the company’s investigational flu vaccine but also a flu-COVID combo shot and other parts of the company’s pipeline. And for the broader industry, it highlights the regulatory uncertainty that persists into 2026, even as other countries move full speed ahead with novel modalities.That regulatory uncertainty extended this week to DiscMedicine, whose rare disease drug bitopertin was rejectedby the FDA. The company tried to convince investors that its planned Phase 3 trial would pave the way toward a refiling, but analysts raised concerns that the study was developed with oldFDA leadership. Disc’s situation also raises questions about the FDA’s Commissioner’s National Priority Voucher, which bitopertin had received.Meanwhile, Compass Pathways dropped long-awaited data from two Phase 3 trials of its psilocybin-based COMP360, showing strong durability data that “clearly met the Street’s bar for success,” according to Stifel analysts. The company plans to complete a rolling new drug application by the end of the year.Finally, the CDC has been left leaderless, again, with the departure of Jim O’Neill, who had been servingas acting director after the ouster of Susan Monarez last summer. And Sanofi’s Paul Hudson was removed as CEO last week. He’ll be replaced by Merck KGaA’s Belén Garijo, who becomes just the second woman in charge of a Big pharmacompany.

Last year, the FDA announced it would be phasingout animal testing requirements for some therapies. The NIHfollowed suit. According to Thomas Hartung, professor and chair at Johns Hopkins Bloomberg School of Public Health, these policy shifts are an “overdue adaptation to scientific progress.” In this special edition of The Weekly, Hartung discusses howartificial intelligence (AI) and various non-animal models such as human organoids will transform drug development and delivery, especially now that the regulatory side is catching up. The FDA Modernization Act, now making its way through Congress, codifies the FDA’s stated goal of starting to move away from animal testing. And in making that announcement last year, the FDA put out a roadmap for how to do this. In May, Hartung will attend the National Biotechnology Conference in San Diego as a keynote speaker. BioSpace is a media partner of the meeting, and Managing Editor Jef Akst will also attend to moderate the executive track. The 2026 National Biotechnology Conference runs May 11–14.You can find the agenda here.

In this episode of Denatured, Jennifer C. Smith-Parker speaks with Dr. Rob Monroe, Vice President and Chief Scientific Officer of Oncology at Danaher Corporation and Chief Medical Officer at Leica Biosystems, and Jennifer Fakish, Vice President and Franchise Head of Oncology at Danaher Corporation. We'll be discussing how antibody drug conjugates (ADCs) are transforming cancer care and with AI-powered pathology, doctors can now measure her HER2 more precisely to match patients with the best treatments. HostJennifer Smith-Parker, Director of Insights, BioSpaceGuestsDr. Rob Monroe, Vice President and Chief Scientific Officer, Oncology, Danaher Corporation; Chief Medical Officer, Leica BiosystemsJennifer Faikish, Vice President and Franchise Head, Oncology, Danaher CorporationDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Last Thursday, Hims & Hers launched a compounded versionof Novo’s oral Wegovy, approved just before Christmas and launched in early January. Novo was, not surprisingly, none too pleased. FDA Commissioner Marty Makary denounced“illegal copycat drugs” in an X post the same day without naming Hims, which then said it would notsell the oral weight loss drug. Novo sued the consumer healthcare company for patent infringement, seeking potentially hundreds of millions in damages. Thenit emerged that FDA hit Hims with a warning letter last year about infestations of vermin at a facility it owns, including rodents, birds and insects, as well as a live spider in a production area.The timing of all the Hims drama is interesting, because BioSpace was already covering Novo, Lilly and their blockbuster GLP weight loss drugs as both companies—and others—reported 2025 fourth quarter and full year earnings. EliLilly and Novo both reported last Wednesday, and both spoke of Novo’s oral Wegovy launch positively. But the calls had two very different tones, as Lilly beat Q4 2025 analyst consensus by more than a billion dollars while Novo projected sales to decline by 5% in 2026.In other recent earnings calls, AbbVie touted strong Skyriziand Rinvoq sales, and the company continues to follow Johnson & Johnson’s lead into the psychedelics space. And Biogen discussed the highly anticipated readout of its tau-targetingtherapy for Alzheimer’s.Finally, in gene therapy, Sarepta struggles to recover from last year’s patient deaths as other DMD contenders near the market; uniQure pauses two arms of a clinical trial for Fabry disease; and Ultragenyx resubmits its in vivo gene therapy UX111 for the treatment of Sanfilippo syndrome type A.

In this episode of Denatured, Jennifer C. Smith-Parker speaks to Erik Digman Wiklud, CEO of Circio and Jacob Becraft, CO-founder and CEO of Strand Therapeutics. Since the mRNA vaccine breakthroughs of the COVID-19 era, attention has turned to what's next for programmable medicines. While first- generation mRNA prove the power of transient genetic instruction, its instability, immune reactivity, and short-lived expression have limited its use mainly to vaccines. Emerging platforms like circular and logic circuit RNA are expanding the field's therapeutic horizons.HostJennifer Smith-Parker, Director of Insights, BioSpaceGuestsErik Digman Wiklund⁠, CEO, CircioJacob Becraft, Co-founder and CEO, Strand TherapeuticsDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Pfizer reported the first data from its new obesity pipeline, picked up in the nearly $10 billion acquisition of Metsera last fall. While BMO Capital Markets said in a Tuesday note that the data “look competitive,” analysts clamored for more details on Pfizer’s earnings call the same morning—and were left wanting more. Meawhile, Merck batted away accusations of “modest growth” from analysts on its own earnings call, as CEO Robert Davis touted “probably the broadest and widest pipelinewe’ve had in years.” These calls followed Roche last week and Johnson & Johnson before that, but earnings season is just getting started. On the docket today is Eli Lilly, which has been acquisitive of late, plus Novo Nordisk, Novartis, AbbVie and more.  On the regulatory front, the FDA is expected to decideon eight products this month, including REGENXBIO’s Hunter syndrome gene therapy RGX-121. The biotech ran into a regulatory snag last week, however, as the FDA placed a clinical hold on two of its programs, including RGX-121. The agency also launched its much-anticipated PreCheck pilot program, which aims to make it easier for companies to build manufacturing plants in the U.S. And President Donald Trump’s TrumpRx platform is delayed, potentially amid anti-kickback concerns.  In ClinicaSpace this week, we took a deep dive intothe resurgent psychedelics space, which could see two companies—Compass Pathways and Definium—submit FDAapplications this year. H.C. Wainwright analyst Patrick Trucchio told BioSpace 2026 is set to be “the biggest year from a clinical data standpoint,” since the firm began covering Compass in 2018. And check your inboxes Wednesday for BioPharmExecutive, where we take a look back at the banner IPO year that was 2021: Where are these companies now?

In this episode of Denatured, Jennifer C. Smith-Parker speaks with RTW’s Rod Wong and Stephanie Sirota how shifting JPM deal timing masks record M&A potential; why oncology, obesity, psychedelics, and neuroscience are attracting fresh capital; and how “alpha stacking” shapes their investment edge in an age of chronic uncertainty. They cover topics discussed in RTW's new book, "Innovation is the Best Medicine."Hosts⁠⁠⁠⁠Jennifer Smith-Parker⁠⁠, Director of Insights, BioSpaceGuestsRoderick Wong, Managing Partner, Chief Investment Officer, RTW InvestmentsStephanie Sirota, Partner, Chief Business Officer, RTW InvestmentsDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Roche made the biggest splash this week so far, announcing on Tuesday that GLP-1/GIP injectable CT-388 led to 22.5%weight loss in a Phase II trial. These numbers appear to put CT-388, which Roche acquired in its $2.7 billion Carmot buy, in line with Eli Lilly’s Zepbound, according to William Blair analysts. Roche plans to start a Phase III study of CT-388 in the first half of this year and is also pairing the drug with a therapy from Zealand Pharma, with the aim of offering a weight lossoption with fewer gastrointestinal side effects. Meanwhile, Baseline Therapeutics debuted to challenge Lilly with a Phase III–ready GLP-1 for alcohol use disorder.  In the vaccines sector, Moderna took perhaps the biggest action to date amid Health Secretary Robert F. Kennedy’s anti-vaccine policies and rhetoric, last week announcing that the company will no longer run late-stage vaccine trials for infectious diseases. “You cannot make a return on investment if you don’t have access to the U.S. market,” CEO Stéphane Bancel saidthe World Economic Forum at Davos, Switzerland. Pfizer CEO Albert Bourla, also speaking at Davos, called RFK Jr.’s rhetoric and policies on vaccines “anti-science.”  Finally, Sarepta released new data on Monday for Elevidys, the company’s embattled gene therapy for neuromuscular disease Duchenne muscular dystrophy. Plus, check out up-and-coming treatments for Alzheimer’s and Parkinson’s.

In this episode of Denatured, BioSpace editorial team members, Senior Editor, Annalee Armstrong, and News Editor, Dan Samorodnitsky, discuss their post-JPM takeaways and 2026 forecasts after speaking to a range of pharma and biotech executives and investors last week.Hosts⁠Jennifer Smith-Parker⁠, Director of Insights, BioSpace⁠Guests⁠Annalee Armstrong, Senior Editor, BioSpace⁠Dan Samorodnitsky, News Editor, BioSpaceDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.