This Week in Biotech #54
Catch up on the latest biotech breakthroughs and upcoming trends (May 23-29, 2025).
Hi and welcome to This Week in Biotech by Biotech Blueprint, edition 54.
Biotech Blueprint is now a podcast. You can find us on YouTube, Spotify, and Apple Podcasts.
THIS WEEK’S KEY TAKEAWAYS 🔑
Gene editing hit a rough patch this week, with safety concerns slowing progress in a few major trials. On the policy side, pandemic era funding and guidance continued to shift.
Intellia’s stock plummeted after a patient in its gene-editing trial showed signs of liver toxicity. This adverse event renewed some concerns about the risks that may come with editing DNA inside the body, even when the company says the issue is resolving. Rocket Pharma faced a harsher reality as a patient death led to a clinical hold and a sharp drop in the stock.
On a more positive note, GSK & Spero delivered phase 3 success for their oral UTI antibiotic. Strand Therapeutics debuted early human data showing systemic immune responses from localized mRNA therapy. This is a potential validation for the next generation of IL-12-based immunotherapy. Beam Therapeutics gained orphan drug status for its gene base editing candidate in liver disease. And Eli Lilly continued its pivot toward next-gen pain treatments with a $1B deal to acquire SiteOne Therapeutics.
On the political front, CDC reversed guidance recommending COVID-19 vaccination for children & pregnant women, a move welcomed by vaccine skeptics but sharply criticized by the broader medical community. In another major shift, HHS pulled a $590M grant from Moderna for its bird flu vaccine, signaling that pandemic-era funding pipelines are closing, and perhaps setting a troubling precedent for future public health preparedness.
🎙️ AI SUMMARY
Listen to an AI-generated summary of this episode below.
BIOTECH/PHARMA NEWS
🔹 The Make America Healthy Again (MAHA) report, released by the U.S. Department of Health and Human Services (HHS) under Secretary Robert F. Kennedy Jr., is facing scrutiny following multiple reports that some of its cited studies do not exist and others may have been misrepresented. Investigations by news outlet NOTUS found at least seven references in the report that could not be verified, including citations attributed to researchers who deny authoring the work. Additionally, several cited studies reportedly contain broken links, incorrect author listings, or conclusions inconsistent with the original research.
The White House responded by affirming its confidence in Secretary Kennedy and attributed the issues to “formatting problems,” stating the report will be updated. The MAHA report, which addresses topics such as childhood health, overmedicalization, and vaccine schedules, is the first publication from the MAHA Commission. As of now, HHS has not formally addressed the specific citation inconsistencies.
🔹 Intellia Therapeutics shares plunged on May 29 after the company disclosed a serious liver-related side effect in a patient enrolled in its phase 3 trial for nexiguran ziclumeran (nex-z), a gene-editing therapy developed with Regeneron for transthyretin amyloid cardiomyopathy (ATTR-CM). The patient experienced Grade 4 liver enzyme elevations, though remained asymptomatic and the issue is reportedly resolving without major intervention. The trial has dosed over 200 of 765 patients, and while Intellia noted the overall safety profile is consistent with earlier studies, the news adds to broader concerns about safety risks in gene therapy, leading to a sharp drop in shares.
🔹 Beam Therapeutics announced that the FDA has granted Orphan Drug Designation to its investigational therapy BEAM-302 for alpha-1 antitrypsin deficiency (AATD), a rare genetic disorder affecting the lungs and liver. BEAM-302 is a liver-targeting lipid nanoparticle (LNP) formulation designed to correct the PiZ mutation using gene base editing technology.
🔹 Moderna reported strong interim results from its phase 1/2 trial of mRNA-1018, an investigational H5 avian flu vaccine, showing that nearly 98% of participants achieved protective antibody levels after two doses, with a favorable safety profile. However, the update was overshadowed by news that the U.S. Department of Health and Human Services has terminated its $590M contract with Moderna for late-stage development and procurement of pre-pandemic flu vaccines. The funding loss raises uncertainty around the vaccine’s future. Moderna says it remains committed to pandemic preparedness and is exploring alternative paths for advancing the program.
🔹 Prothena has shut down its Birtamimab program for the second time after the anti-amyloid antibody failed to meet its primary endpoint in a phase 3 trial. The study enrolled 207 newly diagnosed Mayo Stage IV AL amyloidosis patients but showed no improvement in time to all-cause mortality, despite earlier post hoc analyses suggesting a potential benefit in this high-risk group. In response, the company announced plans to reduce operating costs and significantly downsize staff. Prothena will continue to advance other clinical assets, including its Alzheimer’s candidate PRX012 and partnered programs with Roche, Novo Nordisk, and Bristol Myers Squibb.
🔹 Eli Lilly announced its acquisition of SiteOne Therapeutics, a biotech firm developing non-opioid pain treatments, in a deal worth up to $1B. The move brings STC-004, a phase 2–ready Nav1.8 inhibitor targeting chronic pain, into Lilly’s pipeline. The deal strengthens Lilly’s push to develop safer, addiction-free therapies amid growing demand for alternatives to opioids. SiteOne’s platform also includes additional ion channel inhibitors for conditions like chronic cough and ocular pain.
🔹 Keros Therapeutics is discontinuing the development of cibotercept (KER-012) in patients with pulmonary arterial hypertension (PAH) after safety concerns were observed in its phase 2 TROPOS trial that caused the company to voluntarily halt the trial earlier this year. Keros announced they would undergo a corporate restructuring and reduce its workforce by 45% to cut costs. They are continuing to evaluate cibotercept in other indications as well as push forward their other programs in neuromuscular diseases and hematological disorders. An update on other strategic alternatives, including a potential sale or business combination is expected on Jun. 9, 2025.
🔹 iTeos Therapeutics announced plans to wind down operations in an effort to maximize shareholder value. The decision follows the recent termination of its partnership with GSK, which came after their anti-TIGIT antibody, belrestotug, failed to meet key endpoints in a phase 2 clinical trial. The company is exploring additional sales revenue from its remaining assets including two phase 1 oncology programs, ENT1 inhibitor EOS-984 and anti-TREM2 antibody EOS-215, as well as a their preclinical obesity program targeting ENT1.
🔹 Biogen and City Therapeutics have announced a strategic collaboration to develop RNA interference (RNAi) therapies, initially targeting a single pathway linked to central nervous system (CNS) disorders. The deal includes a $46M upfront commitment from Biogen consisting of a $16M cash payment and a $30M convertible note investment that would give Biogen minority equity interest. The partnership leverages Biogen’s proprietary drug delivery technologies alongside City’s next-generation RNAi engineering platform. Biogen also holds the option to expand the collaboration to additional targets with further payments. The partnership underscores Biogen’s ongoing shift toward external innovation and expanding their R&D portfolio.
🔹 The FDA’s Vaccines and Related Biological Products Advisory Committee voted unanimously on May 22, 2025, to recommend that COVID-19 vaccines for the 2025–2026 season be updated to target the JN.1 lineage, with a preference for the LP.8.1 strain. This marks the second year in a row that the committee has supported a JN.1-based formula. Moderna has already submitted its updated Spikevax for review, while Novavax expressed concerns about manufacturing timelines if LP.8.1 remains the preferred strain. Despite these concerns, Pfizer, Moderna, and other major vaccine makers say they are ready to deliver updated shots in time for the season.
CLINICAL TRIAL UPDATES
🔹 Veru’s phase 2b QUALITY trial showed that adding enobosarm (3 mg) to semaglutide (Wegovy) led to greater fat loss, preservation of muscle, improved physical function, and fewer GI side effects compared to semaglutide alone in older adults with obesity. The combo resulted in 99% of weight loss coming from fat while nearly eliminating lean mass loss. Based on these results, Veru plans to advance the 3mg dose into phase 3. A follow-up study is also underway to test whether enobosarm can prevent fat regain after GLP-1 discontinuation.
🔹 Strand Therapeutics announced promising early phase 1 data for its lead mRNA therapy, STX-001, in patients with advanced solid tumors at the 2025 ASCO Annual Meeting. STX-001, a self-replicating mRNA therapy encoding IL-12, showed a favorable safety profile and early signs of anti-tumor activity, including a confirmed complete response and systemic immune effects, even in non-injected lesions. The trial enrolled 22 patients with checkpoint inhibitor–refractory tumors and observed immune activation, tumor infiltration by immune cells, and dose-dependent cytokine responses. These results support further development of STX-001 both as a monotherapy and in combination with checkpoint inhibitors.
🔹 GSK and Spero Therapeutics announced that their oral antibiotic candidate tebipenem HBr met the primary endpoint in the phase 3 trial, demonstrating comparable effectiveness to IV antibiotics in treating hospitalized adults with complicated urinary tract infections (cUTIs). The Independent Data Monitoring Committee recommended stopping the trial early due to clear efficacy. If approved, tebipenem HBr would be the first oral carbapenem antibiotic available in the U.S., offering a much needed treatment option for patients with drug-resistant cUTIs. GSK plans to submit the data to the FDA in the second half of 2025. The news sent Spero’s stock soaring over 250%.
🔹 Rocket Pharmaceuticals saw its stock plunge over 60% this week after a patient death in its phase 2 gene therapy trial for Danon disease. The therapy, RP-A501, uses an AAV9 vector to deliver the LAMP2B gene to heart cells. The FDA placed the trial on clinical hold after the patient developed capillary leak syndrome following administration of a new immunosuppressive agent, ultimately leading to fatal complications. The market reaction underscores persistent investor concerns over the safety risks tied to gene therapies.
🔹 Gilead announced that its drug Trodelvy significantly improved progression-free survival in the phase 3 ASCENT-03 trial for patients with first-line metastatic triple-negative breast cancer (mTNBC) who are not eligible for immunotherapy. This marks the second major positive trial for Trodelvy in first-line mTNBC, reinforcing its potential to become a foundational treatment across this aggressive cancer subtype. With consistent safety data and strong results in both PD-L1-negative and PD-L1-positive patients, Trodelvy could soon be the first antibody-drug conjugate approved for all first-line mTNBC patients. Full results will be presented at ASCO.
PUBLIC HEALTH SPOTLIGHT
🔹 On May 27, HHS Secretary Robert F. Kennedy Jr. announced that the CDC will no longer recommend COVID-19 vaccines for healthy children and pregnant women, reversing long-standing guidance. The move, framed by Kennedy as “common sense and good science,” has sparked controversy among public health experts who warn it could hinder vaccine access and increase risks for vulnerable groups. Critics argue the decision bypasses traditional scientific review and undermines trust in federal health agencies, while supporters view it as a correction to overreach in prior vaccine policy.
ON THE HORIZON
🔹 May 2025 FDA PDUFAs:
May 22: Arcutis Biotherapeutics’ Zoryve (roflumilast). ✅ APPROVED
May 23: Sanofi’s sBLA for MenQuadfi. ✅ APPROVED
May 24: Liquidia’s Yutrepia. ✅ APPROVED
May 26: Merck’s sBLA for Welireg (belzutifan). ✅ APPROVED
May 28: Eton Pharmaceuticals’ ET-400. ✅ APPROVED
May 31: Moderna’s next-generation COVID-19 vaccine (mRNA-1283).
🔹 June 2025 FDA PDUFAs:
June 10: Merck’s BLA for clesrovimab, an investigational, long-acting monoclonal antibody designed to protect infants from RSV during their first season.
June 12: UroGen’s UGN-102, a novel, non-surgical intravesical therapy for low-grade intermediate-risk non-muscle invasive bladder cancer.
June 17: KalVista’s sebetralstat, an oral on-demand treatment for hereditary angioedema (HAE). If approved, it would be the first non-injectable option for managing acute HAE attacks.
June 19: Gilead’s lenacapavir, a twice-yearly injectable for HIV prevention. If approved, it would be the first long-acting PrEP option administered only twice a year.
June 23: Nuvation Bio’s taletrectinib, a next-generation ROS1 inhibitor for advanced ROS1-positive non-small cell lung cancer. If approved, it would offer a potential best-in-class treatment with activity against resistance mutations and brain metastases.
June 28: Unicycive’s oxylanthanum carbonate, a next-generation phosphate binder for hyperphosphatemia in dialysis patients. If approved, it could offer a lower pill burden and more convenient dosing compared to existing treatments.
June 30: Verastem’s avutometinib + defactinib combo in recurrent KRAS-mutant low-grade serous ovarian cancer. If approved, it would be the first FDA-approved treatment specifically for this population.
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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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Your Substack is great! Can you recommend a good place to find clinical trial data? Are there any free resources or databases?