Ep. 301 - ASCO’s First-in-Human Trials, Crossover Investors & FDA's Rare Disease Plans

The Trump administration’s overhaul of FDA is still underway, but the agency has already veered away from its decades-long trajectory in ways that will profoundly reshape medical product development and the lives of U.S. patients. On the latest BioCentury This Week podcast, BioCentury’s analysts assess who the winners and losers are likely to be as FDA changes shape.The analysts discuss a pair of in vivo CAR T companies: Esobiotec, whose story offers a case study in product-focused strategy, and Capstan Therapeutics, whose in vivo CAR T platform for immunological disorders attracted a buyer in Abbvie. The team also analyzes why data from Compass Pathways for psilocybin therapy COMP360 disappointed investors despite hitting the endpoint, and how molecular glues could be the future of selective CDK2 targeting. This episode of BioCentury This Week was sponsored by ICON Biotech.View full story: https://www.biocentury.com/article/656341#biotech #biopharma #pharma #lifescience #RandD #drugapproval00:01 - Sponsor Message: ICON Biotech02:26 - BioCentury Grand Rounds Europe05:02 - FDA's Future15:03 - Psychedelics22:53 - Esobiotech's Fast Exit31:37 - Molecular Glues and CDK234:43 - Abbvie's Capstan BuyTo submit a question to BioCentury’s editors, email the BioCentury This Week team at [email protected] us by sending a text

Publicly traded U.K. investment firm and company builder Syncona is restructuring its fund amid ongoing market challenges in the biopharma industry. On the latest BioCentury This Week podcast, BioCentury’s analysts discuss how the firm will steer its portfolio toward returns for shareholders, while aiming to build a new fund away from public markets. The analysts then assess Eli Lilly's takeout of cardiovascular base editing company Verve, and what Washington Editor Steve Usdin calls FDA’s new “two-track” future for evaluating new therapies for approval — those with clear-cut benefits and those with ambiguous efficacy safety and efficacy profiles — in light of the many departures of senior FDA staff. They also discuss NASDAQ’s largest biopharma IPO — by Caris —  in two years, the latest obesity readouts, FDA Commissioner Marty Makary’s priority pathway and Usdin’s Q&A with new BIO Chair Fritz Bittenbender. This episode of BioCentury This Week was sponsored by ICON Biotech.View full story: https://www.biocentury.com/article/656266#biotech #biopharma #pharma #lifescience #finance #CV #FDA00:01 - Sponsor Message: ICON Biotech02:14 - Syncona Restructures Fund10:08 - Lilly's $1B Verve Takeout21:45 - FDA's 2-Track FutureTo submit a question to BioCentury’s editors, email the BioCentury This Week team at [email protected] us by sending a text

The tragic death of a second non-ambulatory DMD patient treated with Sarepta's Elevidys gene therapy marks a turning point for the field and should drive all stakeholders to come together to figure out how to safely treat patients with this new modality. On the latest BioCentury This Week podcast, BioCentury’s analysts discuss the opportunity FDA, companies and patient advocates have to share data and identify a safe path forward for the gene therapy field, both in DMD and beyond.The analysts also review promising early data for amylin agonists to treat obesity from Metsera and Eli Lilly, and discuss the vision for FDA’s future laid out by Commissioner Marty Makary and CBER director Vinay Prasad. This episode of BioCentury This Week was sponsored by ICON Biotech.View full story: https://www.biocentury.com/article/656214#biotech #biopharma #pharma #lifescience #obesity #FDA #DMD00:01 - Sponsor Message: ICON Biotech01:19 - Gene Therapy Safety11:55 - Amylin in Obesity18:17 - FDA's FutureTo submit a question to BioCentury’s editors, email the BioCentury This Week team at [email protected] us by sending a text

With federal funding at risk and VCs increasingly focused on de-risked assets, academia is facing unprecedented headwinds despite rapid advancements in innovation. One potential bright spot: Pharma, which is turning to academia to access first-in-class innovation. But many in pharma complain it is difficult to work with academia.On a special Grand Rounds edition of the BioCentury This Week podcast, BioCentury analysts and special guests discuss a new way of thinking and a new era of collaboration between academia and industry.View full story: https://www.biocentury.com/article/656177#biotech #biopharma #pharma #lifescience #Discovery #Translation #BioCenturyGrandRounds00:00 - Introduction02:38 - Key Findings07:42 - Collaboration and Opportunities 25:52 - BioCentury Grand Rounds EuropeTo submit a question to BioCentury’s editors, email the BioCentury This Week team at [email protected] us by sending a text

Translational trends at this year’s ASCO meeting featured new and selective ways to target cell surface receptors on solid tumors. On the latest BioCentury This Week podcast, BioCentury’s analysts discuss the findings from Executive Director of Biopharma Intelligence Lauren Martz’s deep dive into first-in-human studies at the American Society of Clinical Oncology meeting, including how immunocytokines, solid tumor CAR Ts and Chinese innovation are thriving in early trials.The analysts also examine the signs of strain and resilience in biotech’s crossover investors, as well as FDA’s plans for revamping rare disease regulation. This episode of BioCentury This Week was sponsored by ICON Biotech.View full story: https://www.biocentury.com/article/656139#biotech #biopharma #pharma #lifescience #RandD #DrugDevelopment00:01 - Sponsor Message: ICON Biotech02:11 - ASCO’s First-in-Human Trials12:46 - Crossover Investor Health Check22:54 - FDA's Rare Disease PlansTo submit a question to BioCentury’s editors, email the BioCentury This Week team at [email protected] us by sending a text

Dealmaking by a pair of pharmas has given the biotech industry its best day of transactions in months, tallying nearly $13 billion in guaranteed payments across two deals. On the latest BioCentury This Week podcast, BioCentury’s analysts discuss how the takeout of Blueprint Medicines for $9.1 billion up front gives Sanofi a drug for a rare immunological disorder and bolsters the French pharma’s already strong presence in immunology. The analysts also assess the $3.5 billion partnership between BioNTech and Bristol Myers Squibb for an asset targeting cancer’s hottest target, PD-(L)1 x VEGF, and underwhelming data from the leading asset against the target, PD-1 x VEGF bispecific ivonescimab, from Summit and Akeso Inc. Those data coincided with the kick-off of the American Society of Clinical Oncology (ASCO) meeting in Chicago, where almost a dozen companies were presenting readouts for another hot target, CLDN18.2. Evopoint is among the companies; its program recently attracted Astellas as a partner. Meanwhile, the biopharma industry is racing to counter the White House’s most favored nation drug pricing strategy. BioCentury’s Washington analyst, Steve Usdin, explains the urgency and details some of industry’s options.View full story: https://www.biocentury.com/article/656097#biotech #biopharma #pharma #lifescience #deals00:00 - Introduction04:39 - Sanofi Buys Blueprint09:22 - BMS-BioNTech20:01 - Hot Targets23:40 - Drug PricingTo submit a question to BioCentury’s editors, email the BioCentury This Week team at [email protected] us by sending a text

Dutch biotech Merus’ EGFR x LGR5 bispecific antibody has caught investors’ attention in the run-up to ASCO as a new approach to block EGFR signaling. On the latest BioCentury This Week podcast, BioCentury’s analysts discuss Phase II data from Merus for petosemtamab as they preview the American Society of Clinical Oncology’s upcoming annual meeting. The analysts also assess a setback in a gene therapy trial for Rocket Pharmaceuticals, renewed interest in cancer target EpCAM, and a flurry of biopharma activity on the Hong Kong stock exchange. Finally, the team previews BioCentury’s second annual Grand Rounds R&D meeting, which takes place next week in Chicago. This episode was sponsored by Jeito Capital.View full story: https://www.biocentury.com/article/656038#biotech #biopharma #pharma #lifescience #RandD #DrugDevelopment00:01 - Sponsor Message: Jeito Capital24:52 - HK IPOs07:12 - ASCO16:37 - EpCAM20:16 - RocketTo submit a question to BioCentury’s editors, email the BioCentury This Week team at [email protected] us by sending a text

Political and market turmoil has raised questions whether the U.S. can retain its biopharma leadership position, and in turn presents Europe with an opportunity to attract talent back to the continent with an offer of a more stable biomedical funding and regulatory environment. Jeito Capital’s Ksenija Pavletic and Cambridge Innovation Capital’s Anne Horgan join BioCentury’s analyst to wrap up the 25th annual Bio€quity Europe conference in Bruges, Belgium, by discussing their key takeaways from the two-day event. This episode was sponsored by Jeito Capital.View full story: https://www.biocentury.com/article/655998#biotech #biopharma #pharma #lifescience #networking00:01 - Sponsor Message: Jeito Capital02:10 - Key Takeaways09:13 - Challenges and Opportunities for Europe19:23 - Capital Efficiency and Success Stories28:27 - Leadership, Teams & TalentTo submit a question to BioCentury’s editors, email the BioCentury This Week team at [email protected] us by sending a text

The turbulence that has come with the Trump administration’s policies related to the U.S. biopharmaceutical industry is creating an opening for Europe to bolster its life sciences industry. On the latest BioCentury This Week podcast, BioCentury’s editors look at how Europe can capitalize on staffing cuts at FDA and NIH and an uncertain policy environment to lure back talent to bolster regulatory agencies and biotech R&D engines and attract assets and partners from China.The editors also discuss two biotech deals driven by former leaders of BD at Roche, Biomarin's James Sabry and Sophie Kornowski at Boston Pharmaceuticals. Under the leadership of Sabry and CEO Alexander Hardy, Biomarin delivered its first takeout in a decade by acquiring Inozyme as it positions itself to take advantage of a regulatory and policy environment that they believe is favorable to their rare disease strategy. CEO Kornowski, meanwhile, executed on a plan to focus her company on a single liver disease asset that GSK acquired for $1.2 billion up front. Finally, BioCentury’s editors discuss the management shake-up at obesity company Novo Nordisk, where Lars Fruergaard Jørgensen, the leader who spearheaded Novo Nordisk’s transformation into a dominant player in obesity, is stepping down.Chan Zuckerberg Chicago Biohub is hosting an exclusive, invite-only reception on June 3 — the eve of BioCentury's Grand Rounds conference — gathering top voices in biotech innovation and investment to exchange bold ideas, spark new collaborations and channel the energy of Chicago’s thriving innovation ecosystem. If you’re interested in attending, please register here. This episode was sponsored by Jeito Capital.View full story: https://www.biocentury.com/article/655964#biotech #biopharma #pharma #lifescience #politics #policy #law00:01 - Sponsor Message: Jeito Capital05:05 - European Biotech's Moment?19:00 - Boston Pharma, Biomarin Deals29:41 - Novo Nordisk CEOTo submit a question to BioCentury’s editors, email the BioCentury This Week team at [email protected] us by sending a text

Biopharmas developing therapies for obesity are looking to a future where the quality of weight loss matters as much as the quantity — what targets and programs could help realize that vision? On the latest BioCentury This Week podcast, BioCentury’s editors discuss the signaling pathways that may exclusively target fat mass while preserving, or even increasing, lean mass — and which companies are playing in the space.The editors also assess what Vinay Prasad’s past comments say about how he might lead FDA’s Center for Biologics Evaluation and Research (CBER) — and examine how investors are evaluating the possibility that he might move the bar for regulatory approvals. The team then introduces the first in a new BioCentury series, with a look at a Literature Dive into ovarian cancer “atlases” — large-scale molecular profiling studies that point to predictive signatures and patient stratification strategies. This episode was sponsored by Jeito Capital.View full story: https://www.biocentury.com/article/655898#biotech #biopharma #pharma #obesity #FDA #CBER #LifeScience #OvarianCancer00:01 - Sponsor Message: Jeito Capital03:22 - Obesity: Quality over Quantity15:47 - Prasad at FDA27:58 - Ovarian Cancer AtlasesTo submit a question to BioCentury’s editors, email the BioCentury This Week team at [email protected] us by sending a text