This Week in Biotech #51
Catch up on the latest biotech breakthroughs and upcoming trends (May 2-8, 2025).
Hello and welcome to This Week in Biotech by Biotech Blueprint, edition 51.
THIS WEEK’S KEY TAKEAWAYS 🔑
CRISPR Therapeutics made headlines this week with promising phase 1 results for its gene therapy that lowers cholesterol, CTX310. The treatment, which targets the ANGPTL3 gene, showed reductions of up to 82% in triglycerides and 81% in LDL cholesterol. This is an early but potentially major step toward expanding gene editing into broader cardiovascular disease. Investors welcomed the news, reinforcing optimism around CRISPR’s growing pipeline beyond sickle cell and beta thalassemia.
Markets reacted sharply to the appointment of Vinay Prasad as head of the FDA’s Center for Biologics Evaluation and Research (CBER). Prasad, a UCSF oncologist and vocal critic of pandemic era public health policies, replaces Peter Marks, who resigned due to pressure from Health and Human Services Secretary Robert F. Kennedy Jr. Biotech stocks fell across the board following the announcement, with the S&P Biotech ETF (XBI) dropping 6%, and vaccine developers like Moderna (-12%), BioNTech (-5.6%), and Vaxcyte (-14%) all trading lower. The FDA’s Vaccines Advisory Committee is scheduled to meet virtually on May 22 to determine the formula for the 2025–2026 COVID-19 season, offering the first glimpse into how the new CBER leadership may approach vaccine policy.
Meanwhile, the NIH faces existential pressure. Trump’s proposed budget would slash its funding from $48.5B to $27B, consolidate its 27 institutes into five, and redirect focus toward epidemic-scale threats. It’s not policy yet, but the proposal reflects a shift that could reshape U.S. biomedical research, both in academia but also the industry.
Charles River Laboratories, the world’s largest provider of lab animals, also came under pressure as the FDA continues its push toward replacing animal testing with new methods such as AI-guided simulations and organ-on-chip models. The company announced a major board shake up and a strategic review in response to shifting regulatory priorities. I’ll be digging into this shift and what it means for biotech and animal research in an upcoming deep dive and podcast.
This week was a reminder that biotech lives at the intersection of science and policy. As gene editing moves forward, and Washington rethinks everything from vaccine regulation to lab animal use, companies will need to stay nimble.
For more details, keep reading or check out our AI-generated podcast or Symvol video, a <5 min visual rundown of this week’s biotech breakthroughs.
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MARKET UPDATES
🔹 Vertex Pharmaceuticals fell as much as 13% (its steepest drop since 2020) after Q1 earnings missed expectations. Revenue came in at $2.77B (vs. $2.85B expected), and adjusted EPS at $4.06 (vs. $4.32 expected). Although the company raised the lower end of 2025 guidance, concerns mounted over slow insurance uptake for its new non-opioid painkiller, Journavx. Only 45% of covered patients can currently access it without prior authorization. Analysts were split on the outlook.
Despite recent volatility, Vertex and Gilead have been seen as tariff-resilient outliers thanks to their U.S.-based operations, high domestic tax rates, and local IP holdings. While not immune to policy shocks, both have outperformed much of the pharma sector in 2025, with Gilead up 24% and Vertex up 7.8% year-to-date. Gilead further solidified its U.S. footprint this week with $11B in new investment commitments.
🔹 Capricor Therapeutics’ stock dramatically dropped by 42% this week following the announcement of the first FDA advisory committee meeting under the newly appointed commissioner, Marty Makary. The external committee is slated to evaluate Capricor’s cell therapy, deramiocel, for the treatment of children and young adults with Duchenne Muscular Dystrophy (DMD). Despite investor hesitation, Capricor’s CEO views the panel as just another step toward potential approval of the first-in-class therapy. Controversial approvals in the DMD space are not unheard of- previous approvals include Exondys 51 and Serepta’s gene therapy - and likely spurred the formation of this external advisory committee. Capricor’s PDUFA is set for Aug. 31, 2025 and the advisory committee will evaluate deramiocel before the deadline.
BIOTECH/PHARMA NEWS
🔹 Blood cancer biotech Vor Bio is winding down its clinical and manufacturing operations and exploring strategic alternatives, including a potential sale, merger, or licensing deal, due to limited fundraising prospects and current clinical data. Approximately 95% of staff will be laid off, with a small team retained to manage the transition and regulatory obligations.
🔹 The FDA’s Vaccines and Related Biological Products Advisory Committee will meet virtually on May 22, 2025, to recommend the 2025-2026 Covid-19 vaccine formula for use in the U.S. The panel will review data on Moderna, Pfizer/BioNTech, and potentially Novavax, whose vaccine remains under Emergency Use Authorization pending further study.
🔹 Valneva announced that the European Medicines Agency has temporarily suspended the use of its chikungunya vaccine, Ixchiq, in individuals 65+ following reports of 17 serious adverse events, including 2 deaths, among elderly patients with underlying conditions. The suspension will remain in place while a safety review is conducted. Use in individuals aged 12-64 remains unchanged. U.S. and French health authorities have also issued precautionary guidance for older adults.
🔹❗Vinay Prasad, a UCSF oncologist and critic of pandemic-era health policy, has been appointed to lead the FDA’s Center for Biologics Evaluation and Research (CBER), overseeing vaccines and gene therapies. He replaces Peter Marks, who resigned last month citing pressure from HHS Secretary RFK Jr. Prasad has called for stricter evidence standards and more randomized trials for vaccines, raising uncertainty for biopharma developers. Following the announcement, the S&P Biotech ETF (XBI) fell 6%, with vaccine stocks like Moderna (-12%), Vaxcyte (-14%), and BioNTech (-5.6%) trading sharply lower.
🔹 President Trump signed a new executive order aimed at accelerating domestic pharmaceutical production by streamlining FDA plant approvals and fast-tracking Environmental Protection Agency permits for new facilities. The order also calls for stricter enforcement of foreign ingredient sourcing and potential public disclosure of non-compliant overseas plants. As part of a broader strategy to shift drug manufacturing back to the U.S., the administration has floated tariffs on pharmaceutical imports, framing the issue as a national security concern. While full tariff details remain pending, major drugmakers like J&J, Eli Lilly, and Roche have already announced increased U.S. manufacturing investments.
🔹 Alvotech and Teva have received FDA approval for Selarsdi, a biosimilar to J&J’s Stelara, with full interchangeability across all approved indications. This includes treatment for plaque psoriasis, psoriatic arthritis, Crohn’s disease, and ulcerative colitis in both adults and children. The milestone solidifies Alvotech’s U.S. presence with two interchangeable biosimilars now on the market and further expands Teva’s biosimilar leadership strategy. With three more biologics under FDA review, the Teva-Alvotech partnership is gaining momentum as a key player in the effort to lower costs and expand access to biologic therapies.
🔹 The future of the National Institute of Health (NIH) has been thrown into uncertainty following President Trump’s proposed 2026 budget to cut the agency’s funding from $48.5B in 2025 to $27B in 2026. Citing a “loss of trust” and what he called an “unfocused” research agenda, Trump’s plan would consolidate the NIH’s 27 centers and institutes into just five broad entities, prioritizing chronic diseases and large-scale epidemics. The proposal has sparked strong backlash from science organizations, which warn that that if Congress approves the cuts, US risks losing its edge as a global scientific leader.
CLINICAL TRIAL UPDATES
🔹❗CRISPR Therapeutics reported positive phase 1 results for CTX310, showing up to 82% reduction in triglycerides and up to 81% reduction in LDL in high-risk patients, with a favorable safety profile. The company also highlighted momentum for Casgevy, its approved CRISPR-based therapy for sickle cell and beta thalassemia, with over 65 treatment centers activated and 90+ patients enrolled. Additional updates from programs including CTX320 (LPA), CTX112 (CD19 CAR-T), and CTX131 (CD70 CAR-T) are expected throughout 2025.
🔹 Tiziana Life Sciences announced positive data from an open-label study evaluating nasal foralumab in patients with non-active secondary progressive multiple sclerosis. The treatment led to meaningful reductions in fatigue, stabilization of disability progression, and significantly reduced microglial activation. Notably, no new lesions were seen on MRI, and no serious treatment-related adverse events were reported. These results highlight the potential of nasal foralumab, the only fully human anti-CD3 monoclonal antibody in development, as a novel, non-invasive therapy for progressive MS. A phase 2 randomized controlled trial is currently underway, with top-line data expected by year-end 2025.
🔹 Actuate Therapeutics announced statistically significant topline results from its global phase 2 trial evaluating elraglusib in combination with gemcitabine/nab-paclitaxel (GnP) as a first-line treatment for metastatic pancreatic cancer. The combination arm showed a marked improvement in median overall survival and a favorable risk-benefit profile compared to GnP alone. The trial met its primary endpoint. Actuate plans to engage with U.S. and EU regulators in the second half of 2025 to expedite potential approval and commercialization of elraglusib.
ON THE HORIZON
🔹 May 2025 FDA PDUFAs:
May 22: The FDA is expected to decide on Arcutis Biotherapeutics’ application for Zoryve (roflumilast) foam, a daily topical treatment for scalp and body psoriasis in patients 12+.
May 23: The FDA is set to decide on Sanofi’s sBLA for MenQuadfi, a quadrivalent meningococcal vaccine, which could expand its use to infants as young as 6 weeks for protection against Neisseria meningitidis serogroups A, C, W, and Y.
May 24: The FDA is expected to decide on Liquidia’s resubmitted NDA for Yutrepia, an inhaled treprostinil powder for treating pulmonary arterial hypertension, following tentative approval last year.
May 26: Decision on Merck’s supplemental application for Welireg (belzutifan) to treat advanced pheochromocytoma and paraganglioma (rare neuroendocrine tumors).
May 28: The FDA has extended the PDUFA goal date for Eton Pharmaceuticals’ ET-400, a therapy for a rare pediatric disease, to allow more time to review supplemental data submitted in Dec. 2024.
May 31: Moderna’s next-generation COVID-19 vaccine (mRNA-1283).
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DISCLAIMER: This content is for informational purposes only. It should not be taken as legal, tax, investment, financial, or other advice. The views expressed here are my own and do not reflect the opinions of any company or institution.
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