This Week in Biotech #48
Catch up on the latest biotech breakthroughs and upcoming trends (Apr 11-17, 2025).
Welcome or welcome back to This Week in Biotech by Biotech Blueprint, edition 48.
THIS WEEK’S KEY TAKEAWAYS 🔑
For a quick overview, here are the main developments across biotech this week.
For more, check out the full edition or listen to our AI-generated podcast. We’re also introducing ▶️ Biotech Briefing, a new way to explore key insights through short, dynamic video content powered by Symvol.
🐁 AI replacing mice: The FDA announced it will phase out animal testing in favor of AI-based models, boosting stocks like RXRX, ABSI, SDGR, and ABCL. CROs like CRL fell. Antibody developers stand to benefit. We are planning a podcast and a mini series on this topic so stay tuned!
💊 Lilly’s oral GLP-1 impresses: Orforglipron showed strong A1C and weight loss results in phase 3, positioning it as a future blockbuster. Peer firms GPCR and MTSR surged.
🚫 Pfizer exits oral GLP-1 race: Safety concerns halt danuglipron’s development. Competitors like VKTX, ALT, and GPCR gained on the news.
🦟 CDC backs chikungunya vaccine: Bavarian Nordic’s Vimkunya gets U.S. recommendation as global cases rise. Single-dose VLP platform could see rapid uptake.
🧬 uniQure gains on Huntington’s news: AMT-130 received FDA breakthrough therapy status after slowing disease progression in early data. Shares jumped >40%.
🇪🇺 EU approves Leqembi: Eisai and Biogen’s Alzheimer’s drug becomes the first EU-approved therapy to slow clinical decline by clearing amyloid.
🧪 Verve cuts LDL-C via gene editing: VERVE-102 showed up to 69% LDL-C (“bad cholesterol”) reduction in phase 1b with no serious adverse events. Stock up ~40%.
🫁 Amgen sets new SCLC standard: Imdelltra outperformed chemo in phase 3 for small cell lung cancer. A potential first-line treatment shift.
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▶️ BIOTECH BRIEFING
This Week in Biotech as a video. Created using Symvol.
MARKET UPDATES
🔹 AI-driven drug discovery stocks surged after the FDA announced last week it will begin phasing out animal testing for investigational new drug applications, encouraging the use of AI-based computational models and simulations instead. The move, hailed as a paradigm shift by FDA Commissioner Martin Makary, is expected to accelerate drug development while reducing reliance on animal models. Beneficiaries included firms like Recursion (RXRX), Absci (ABSI), Schrödinger (SDGR), and AbCellera (ABCL), while contract research organizations like Charles River (CRL) fell sharply. Analysts see the shift as a net positive for antibody developers such as Amgen, Regeneron, and Xencor.
BIOTECH NEWS
🔹 Eli Lilly announced positive phase 3 results for orforglipron, its once-daily oral small molecule GLP-1 receptor agonist, showing significant A1C reductions (1.3–1.6%) and weight loss of up to 16 lbs (7.9%) over 40 weeks in adults with type 2 diabetes. The trial also found that over 65% of participants on the highest dose reached A1C levels ≤6.5%, below the ADA-defined diabetes threshold. Orforglipron’s safety profile was consistent with injectable GLP-1 therapies, with mild/moderate GI side effects. As the first non-peptide oral GLP-1 to succeed in a phase 3 trial, orforglipron’s strong performance positions it as a potential blockbuster, with Lilly expecting global launches for weight management and diabetes starting in 2025 and 2026, respectively. The results boosted shares of peer companies Structure Therapeutics (GPCR) and Metsera (MTSR), which are developing their own oral GLP-1 candidates currently in phase 2b.
🔹 Bavarian Nordic announced that its single dose chikungunya vaccine, Vimkunya, has received a recommendation from the U.S. CDC’s Advisory Committee on Immunization Practices (ACIP) for individuals aged 12+. The recommendation applies to U.S. travelers visiting regions experiencing a chikungunya outbreak or those at elevated risk. Vimkunya, a virus-like particle (VLP)-based recombinant vaccine, was approved by the FDA and European Commission earlier in 2025 and is currently under review in the UK and Canada. Chikungunya, a mosquito-borne virus, has spread to more than 110 countries and caused 620,000 reported cases in 2024 (a 20% increase from 2023). The disease often presents with fever and severe joint pain, and 30-40% of cases may lead to chronic arthritis.
🔹 uniQure’s stock jumped over 40% after the company announced that its investigational gene therapy AMT-130 received breakthrough therapy designation from the FDA for the treatment of Huntington’s disease. The designation was based on promising phase 1/2 clinical data showing a meaningful slowing of disease progression in treated patients. AMT-130 has already been granted RMAT, orphan drug, and fast track designations, and uniQure expects to provide additional regulatory updates and guidance on its Biologics License application submission in Q2 2025. There are currently no approved therapies that modify the course of Huntington’s disease.
🔹 Eisai and Biogen’s Leqembi (lecanemab) has become the first medicine authorized in the European Union to slow the progression of early Alzheimer’s disease. The approval follows positive results from the phase 3 trial, which showed lecanemab reduced clinical decline by 31% over 18 months. This decision comes after a 2 year review and positions Leqembi as the first EU-approved therapy to target an underlying cause of Alzheimer’s by clearing toxic amyloid protofibrils. Leqembi is now authorized across all 27 EU member states, as well as Iceland, Liechtenstein, and Norway. The drug is already approved in the US, Japan, China, the UK.
🔹 Pfizer announced on Apr. 14 it will discontinue development of its once-daily oral GLP-1 receptor agonist, danuglipron, after a case of potential drug-induced liver injury and feedback from regulators. While the compound had shown promising pharmacokinetic results, Pfizer concluded that the risk outweighed the benefit. In response, shares of rival oral obesity drug developers Viking Therapeutics (VKTX), Metsera (MTSR), and Structure Therapeutics (GPCR) surged, as did Terns Pharmaceuticals (TERN), Ventyx Biosciences (VTYX), and Altimmune (ALT), with analysts calling Pfizer’s exit a major boost to smaller competitors. Pfizer says it remains committed to advancing other metabolic programs, including its oral GIPR antagonist.
CLINICAL TRIAL UPDATES
🔹 Verve Therapeutics announced encouraging initial results from its Heart-2 phase 1b trial of VERVE-102, an in vivo base editing therapy targeting PCSK9 for patients with heterozygous familial hypercholesterolemia (premature coronary artery disease). A single infusion of VERVE-102 led to dose dependent reductions in LDL-C, with an average drop of 53% and a maximum of 69% in the highest dose group. The therapy was well-tolerated, with no treatment-related serious adverse events reported. The company expects to launch a phase 2 trial later this year and anticipates a decision from Eli Lilly on its opt-in rights. Following the announcement, Verve’s stock jumped nearly 40%.
🔹 Amgen announced that its phase 3 trial of Imdelltra (tarlatamab-dlle) demonstrated a significant overall survival benefit over standard chemotherapy in patients with small cell lung cancer. Imdelltra, a first-in-class bispecific T cell engager targeting DLL3, showed both statistical and clinical superiority. The drug’s safety profile remained consistent with prior findings. These results mark the first global phase 3 study to demonstrate a survival advantage in this setting, positioning Imdelltra as a potential new standard of care.
ON THE HORIZON
🔹 April 2025 FDA PDUFAs:
Apr. 2: Reproxalap is an investigational drug developed by Aldeyra Therapeutics for the treatment of dry eye disease. REJECTED ❌
Apr. 3: Cabozantinib, marketed by Exelixis under the brand name Cabometyx, is an oral tyrosine kinase inhibitor approved for treating various cancers. APPROVED ✅
Apr. 3: Amgen’s (Horizon Therapeutics) Inebilizumab (Uplizna) for IgG4-related disease (supplemental BLA). APPROVED ✅
Apr. 18: Dupilumab (Dupixent), developed by Regeneron and Sanofi, is an anti-inflammatory biologic already approved for several conditions like atopic dermatitis, asthma, and eosinophilic esophagitis. The FDA is currently reviewing two supplemental applications: one for bullous pemphigoid (PDUFA date: June 20, 2025) and another for chronic spontaneous urticaria (PDUFA date: April 18, 2025), which could further expand its use.
Apr. 26: Telix Pharma’s TLX101-CDx (Pixclara), imaging agent for recurrent glioma (new NDA for F-18 FET diagnostic tracer) awaiting its decision on Apr. 26.
Apr. 29: Elamipretide is an investigational peptide developed by Stealth BioTherapeutics, designed to target mitochondrial dysfunction by binding to cardiolipin in the inner mitochondrial membrane, thereby enhancing mitochondrial function. It is primarily being developed for the treatment of Barth syndrome, an ultra-rare genetic disorder characterized by cardiac abnormalities, muscle weakness, and reduced life expectancy.
Apr. 29: J&J’s Nipocalimab for generalized Myasthenia Gravis (gMG) in adults who are anti-AChR, anti-MuSK or anti-LRP4 antibody-positive (original BLA, anti-FcRn monoclonal antibody). The product has orphan designation and priority review.
Apr. 30: Dihydroergotamine (DHE) is used for the acute treatment of migraine with or without aura in adults. Satsuma Pharmaceuticals is developing STS101, a novel DHE nasal powder formulation designed for fast, easy self-administration and rapid absorption.
Have a great rest of your week and thanks for reading Biotech Blueprint.
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