This Week in Biotech #49
Catch up on the latest biotech breakthroughs and upcoming trends (Apr 18-24, 2025).
Welcome to This Week in Biotech by Biotech Blueprint, edition 49.
THIS WEEK’S KEY TAKEAWAYS 🔑
Here’s a quick snapshot of the biggest biotech developments from the past few days:
💉 Novavax shares jumped ~20% after the FDA requested more data on its COVID-19 vaccine, signaling possible full approval despite prior criticism from HHS Secretary RFK Jr.
🧬 Gene therapy stocks rallied following FDA Commissioner Martin Makary’s supportive comments on a new pathway for rare disease treatments.
💰 Merck is nearing a $3.5B deal to acquire SpringWorks, gaining approved and emerging rare disease drugs, bolstering its oncology pipeline.
⚖️ Viking Therapeutics reported strong results from its obesity and NASH programs, including up to 14.7% weight loss in phase 2.
🧪 Gilead’s Trodelvy + Merck’s Keytruda combo met its phase 3 goal, showing significant progression-free survival improvement in PD-L1+ metastatic breast cancer.
AstraZeneca and Daiichi Sankyo’s Enhertu + pertuzumab combo showed superior efficacy in HER2+ metastatic breast cancer, potentially redefining 1st-line treatment.
Exact Sciences launched Oncodetect, a highly sensitive minimal residual disease test for early cancer recurrence, now available nationwide with Medicare coverage pending.
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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▶️ BIOTECH BRIEFING
This Week in Biotech now as a video. Created using Symvol.
MARKET UPDATES
🔹 Novavax (NVAX) shares jumped ~20% on Apr. 23 after the FDA requested additional clinical data on its COVID-19 vaccine, a move investors viewed as a positive signal toward eventual full approval. Although the vaccine currently holds conditional approval, Novavax had anticipated full approval by Apr. 1. The FDA’s request came as part of a post-marketing commitment, which involves further safety and efficacy studies. This development follows comments from U.S. Health and Human Services Secretary Robert F. Kennedy Jr., who criticized the vaccine’s approach and cast doubt on its effectiveness.
🔹 Gene therapy stocks surged after FDA Commissioner Martin Makary made encouraging remarks about the sector during a SiriusXM interview on The Megyn Kelly Show. Makary highlighted a potential “new pathway” for treatments targeting rare diseases, emphasizing the promise of gene therapies and the agency’s openness to modernizing approval processes. He noted that this mechanism would apply primarily to conditions affecting small patient populations. Companies such as Beam Therapeutics, Intellia Therapeutics, uniQure, Regenxbio, Avidity Biosciences, and Dyne Therapeutics all saw gains following the comments.
BIOTECH NEWS
🔹 Merck is in advanced talks to acquire U.S-based SpringWorks Therapeutics for approximately $3.5B, aiming to expand its rare disease and oncology portfolio. The deal, based on a price of around $47 per share, would give Merck access to SpringWorks’ approved drug Ogsiveo for desmoid tumors and its newly approved treatment for a genetic disorder, Gomekli. This potential acquisition comes as Merck faces declining sales for its existing therapies and recent pipeline setbacks. If finalized, it would mark one of Merck’s largest recent biotech deals and strengthen its position in targeted rare disease and cancer therapies.
🔹 AbbVie has submitted a biologics license application to the FDA for TrenibotulinumtoxinE (TrenibotE), a first in class botox developed for the treatment of moderate to severe frown lines. TrenibotE is designed to deliver a rapid onset of action (as early as 8 hours post-injection) and a shorter duration of effect lasting 2 to 3 weeks. If approved, it would be the first neurotoxin of its kind on the market, providing patients with a shorter-term alternative that could address common concerns about unnatural or long-lasting effects from aesthetic treatments. The submission is supported by clinical data from more than 2,100 patients.
🔹 Halozyme Therapeutics has filed a patent infringement lawsuit against Merck in the U.S. District Court of New Jersey, alleging that Merck’s injectable formulation of Keytruda violates multiple Halozyme patents related to its technology, which have been in place since 2011. Halozyme claims that Merck has knowingly used its proprietary technology to develop injectable Keytruda and intends to launch it later this year despite the infringement. The company is seeking both damages and an injunction to halt the alleged patent violation.
🔹 On Apr. 23, Viking Therapeutics provided a promising update on its clinical pipeline in its Q1 2025 corporate report. Lead candidate VK2735, a dual GLP-1/GIP receptor agonist for obesity, showed compelling results in the phase 2 VENTURE trial, delivering up to 14.7% mean body weight reduction over 13 weeks with a favorable safety profile. The oral version of VK2735 also showed encouraging weight loss, up to 5.3% over 28 days, in a phase 1 study, suggesting strong potential for an oral obesity treatment. For non-alcoholic steatohepatitis (NASH), VK2809 met its primary endpoint in the phase 2b VOYAGE study, demonstrating significant liver fat reduction; key histology data from the ongoing 52 week portion are expected in Q2 2025. Viking is also advancing VK0214 for X-linked adrenoleukodystrophy (X-ALD), with results from a phase 1b study anticipated by mid 2025.
🔹 Vanda Pharmaceuticals claims that the FDA has unlawfully delayed a hearing on the approval of their drug tradipitant for gastroparesis, and have wrongly blamed the delay on staffing cuts and a new FDA Commissioner, Martin Makary. Vanda asserts that the FDA’s Center for Drug Evaluation and Research (CDER) used internal delays and excuses, such as needing more time to review, to avoid scheduling the hearing. The company highlights that this pattern of delays is institutional, with no hearings granted for new drug approvals in the past decade. Vanda criticizes the FDA’s legal strategy of avoiding judicial review and calls on Makary and Secretary Kennedy to uphold transparency and lawful process. CEO Mihael Polymeropoulos urges a shift toward innovation-friendly, transparent decision-making at the FDA.
🔹 Exact Sciences has launched Oncodetect, a highly sensitive, tumor-informed molecular residual disease test designed to detect signs of cancer recurrence across multiple solid tumors, with a focus on colorectal cancer. Oncodetect identifies up to 200 tumor-specific ctDNA variants and can detect recurrence up to two years earlier than imaging by finding trace amounts of ctDNA, down to 1 in 20,000 cfDNA molecules. Backed by long-term data from the Alpha-CORRECT study, the test provides clear “detected” or “not detected” results, supporting personalized treatment decisions and earlier interventions. Now available nationwide, Exact Sciences is working toward Medicare reimbursement and expanding across additional tumor types.
🔹 Renovaro has expanded its strategic partnership with AI cloud infrastructure firm Nebul to accelerate its biomarker discovery and next gen diagnostics programs. Leveraging Nebul’s high performance computing and private AI infrastructure, Renovaro aims to develop ultra sensitive diagnostics for early cancer detection, with a focus on identifying minimal residual disease in lung cancer. The collaboration supports Renovaro’s deep learning models with scalable, secure computing resources, which are critical for clinical AI applications.
🔹 Novo Nordisk announced they would seek approval for their oral GLP-1 (semaglutide) to treat obesity. Novo’s oral semaglutide is currently approved for treatment of type 2 diabetes. Their announcement to pursue approval for treating obesity comes after Eli Lilly’s competing oral GLP-1 (orfoglipron) showed positive phase 3 data in lowering body weight and increases the competition for weight loss drugs between the two companies.
🔹 As tariffs loom, several pharmaceutical companies are preparing to relocate drug production to the United States. Roche recently announced a $50B project, including plans for a new research and development center and drug manufacturing facilities. This investment from the Swiss company follows several similar moves by Novartis, J&J, Eli Lilly, and Regeneron. However, many of these projects were already underway before the proposed pharmaceutical tariffs.
CLINICAL TRIAL UPDATES
🔹 In the Q1 2025 financial results, Gilead Sciences highlighted significant developments in its pipeline. The company anticipates a pivotal FDA decision by June 19 on lenacapavir, a twice-yearly injectable for HIV prevention, which could become a key growth driver. Additionally, Gilead reported progress in its oncology pipeline, including a successful trial of Trodelvy for treating an aggressive form of breast cancer, potentially boosting future demand. The company also continues to expand its cell therapy products and has new options in development.
🔹 Coya Therapeutics reported positive interim results from a study evaluating low dose IL-2 and CTLA4-Ig in 5 patients with mild to moderate frontotemporal dementia (FTD). The treatment significantly increased regulatory T cell numbers and function, with patients showing minimal to no cognitive decline over 22 weeks. The therapy was well tolerated, with no serious adverse events. These findings support Coya’s strategy of targeting immune dysregulation in neurodegenerative diseases and suggest potential for COYA 302 as a novel treatment approach for FTD, which currently has no approved therapies.
🔹 Sanofi reported that their oral TNF inhibitor, balinatunfib, fell short of phase 2 primary endpoints for the treatment of psoriasis. They reported that the drug was well tolerated and showed clinically relevant responses, but it did not significantly reduce psoriasis compared to placebo. They plan to investigate if balinatunfib could be used in combination therapies for psoriasis treatment. This news came alongside their otherwise positive Q1 earnings report where they presented a sales growth of 9.7% led by their lead immunology drug candidate (Dupixent), their hemophilia A drug (Altuviiio), and their vaccine portfolio (including RSV, polio, meningitis).
🔹 In the phase 3 trial, AstraZeneca and Daiichi Sankyo’s Enhertu (trastuzumab deruxtecan) combined with pertuzumab showed a highly statistically significant and clinically meaningful improvement in progression free survival compared to the standard of care for patients with HER2-positive metastatic breast cancer. This marks the first trial in over a decade to demonstrate superior efficacy in this patient population. These results suggest Enhertu plus pertuzumab could redefine first line therapy for HER2-positive metastatic breast cancer.
🔹 Gilead Sciences has announced that the phase 3 trial met its primary endpoint, showing a statistically significant and clinically meaningful improvement in progression free survival for patients with previously untreated PD-L1+ metastatic triple negative breast cancer treated with Trodelvy + Merck’s Keytruda, compared to the standard of care. This marks the first pivotal trial to demonstrate the superiority of an antibody-drug conjugate in combination with immunotherapy in PD-L1+ metastatic breast cancer. The safety profile of the combination was consistent with prior data. These results suggest a potential new frontline treatment option for a patient population with historically limited therapeutic choices.
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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Oral pill GLP1s will be a game changer I wonder what adherence might look like