This Week in Biotech #50
Catch up on the latest biotech breakthroughs and upcoming trends (Apr 25 - May 1, 2025).
Welcome to This Week in Biotech by Biotech Blueprint, edition 50 🎉. Thanks for your continued support and here’s to adding another zero to our newsletter count!
THIS WEEK’S KEY TAKEAWAYS 🔑
🔬 FDA approved Satsuma’s Atzumi, the first ever dihydroergotamine nasal powder for acute migraine.
💉 HHS is shifting away from COVID funding, investing $500M into “Generation Gold Standard” to develop universal flu and coronavirus vaccines.
🧠 FDA approved J&J’s Imaavy (nipocalimab) for rare disease myasthenia gravis in adults and children 12+, showing rapid symptom relief and up to 20 months of disease control.
⚖️ Following safety issues with its oral GLP-1, Pfizer scrapped danuglipron and is now eyeing acquisitions to rebuild its obesity pipeline.
🧬 FDA approved Abeona’s Zevaskyn, the first-ever cell-based gene therapy for wounds in recessive dystrophic epidermolysis bullosa.
🩸 EU approved Regeneron’s Lynozyfic for multiple myeloma. The bispecific antibody showed a 71% response rate and monthly dosing flexibility.
💊 Novartis acquired Regulus for $800M+ to expand its kidney disease pipeline.
🫁 Boehringer’s Zongertinib achieved a 71% response rate in non-small cell lung cancer.
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.
🎙️ PODCAST
Listen to our AI-generated podcast celebrating the 50th edition of today's newsletter:
I also recently launched a new podcast series where I sit down with industry experts and professionals to explore the challenges, trends, and innovations shaping biotech today. In the first episode, I sat down with Hartaj, a longtime biotech analyst and fellow newsletter writer, for a candid and conversation about the biotech industry. We reflected on our career paths, both my transition out of academia and the launch of Biotech Blueprint, and his move from Wall Street to founding Biotech Capital Compass. We explored topics including the potential of mRNA vaccines in cancer, the role of AI in drug discovery, and how recent layoffs in both government and industry could influence the next chapter of biotech innovation. We also discussed our approaches to curating biotech news, the realities of career transitions, and the importance of community-building in this space. It was an honest, engaging conversation between two people passionate about the future of biotech, and the first of what we hope will be many more.
▶️ BIOTECH BRIEFING
This Week in Biotech now as a video. Created using Symvol.
MARKET UPDATES
🔹 Following a recent webinar addressing disappointing mid-stage phase 2 clinical trial results for vidofludimus calcium in multiple sclerosis, Immunic’s stock (IMUX) is slowly rebounding. The failure to meet primary endpoints led to a sharp decline on Tuesday, but reassurances from CEO Daniel Vitt highlight promising secondary endpoints that support the drug’s continued development.
🔹 Biohaven (BHVN) shares jumped 11% after securing an investment of up to $600M from Oberland Capital to support clinical development and commercialization, including its lead asset, troriluzole, for spinocerebellar ataxia. The first $250M will be funded immediately with additional tranches tied to FDA milestones and strategic acquisitions.
BIOTECH NEWS
🔹 Satsuma Pharmaceuticals has received FDA approval for Atzumi, the first and only dihydroergotamine (DHE; derived from fungus) nasal powder for the acute treatment of migraine. Delivered via a portable, patient-friendly device, Atzumi offers fast, sustained relief and convenient administration. Clinical trials showed rapid DHE absorption and consistent plasma levels with a favorable safety profile. The approval marks a milestone for migraine care, offering an easier-to-use alternative to injectable or nasal spray DHE options that have previously limited adoption.
🔹 The U.S. Department of Health and Human Services (HHS), under Health Secretary Robert F. Kennedy Jr., is shifting vaccine priorities with a $500M investment in the Generation Gold Standard project, aimed at developing universal vaccines, including flu and coronavirus vaccines that protect against multiple strains. This move redirects funding away from COVID-19 efforts like the Biden-era Project NextGen, which HHS has called “wasteful.” The flagship vaccine, BPL-1357, developed by NIH scientists now in senior roles, has completed early safety testing. Critics question the scale of the funding amid agency-wide budget cuts, while HHS defends the project as transparent and free from commercial conflicts.
🔹 The FDA has approved J&J’s Imaavy for generalized myasthenia gravis (gMG) in adults and children 12+. In trials, Imaavy rapidly reduced harmful IgG antibodies, improved daily function, and provided up to 20 months of disease control with a favorable safety profile. A support program, Imaavy withMe, may allow eligible patients to start treatment quickly and pay as little as $0 per infusion.
🔹 Pfizer is pivoting its cardiometabolic strategy after halting development of its oral obesity drug danuglipron due to safety concerns. Despite a Q1 earnings beat on earnings per share, revenue fell 8% year over year to $13.7B, missing expectations due to sharply lower Paxlovid sales. CEO Albert Bourla confirmed Pfizer will pursue acquisitions and partnerships to rebuild its obesity pipeline, while launching a $5.7B cost-cutting initiative to fund R&D and improve margins. Investors are now focused on whether Pfizer’s external dealmaking can offset declining covid product sales and reignite long term growth.
🔹 Hims & Hers and Novo Nordisk have launched a partnership to offer of Novo’s Wegovy available on the Hims & Hers platform for $599/month, including all dosage strengths, 24/7 clinical support, and nutrition guidance. The partnership aims to integrate Novo’s treatments with Hims & Hers’ digital care model to improve long-term outcomes and broaden access to obesity care.
🔹 Abeona’s topical cell gene therapy, Zevaskyn, obtained FDA approval for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). RDEB is a rare disease caused by mutations in COL7A1 and causes patients have chronic blistering and severe wounds. Abeona has developed a first of its kind autologous cell-based gene therapy that is genetically modified to contain functional copies of COL7A1. In phase 3 trials, Zevaskyn showed 50% more healing of in large chronic RDEB wounds in 81% of patients compared to 16% wound healing in patients receiving the current standard of care. Zevaskyn is expected to be available to patients in Q3 2025.
🔹 Regeneron’s Lynozyfic (linvoseltamab) has received conditional EU approval for treating relapsed/refractory multiple myeloma in adults who have undergone at least three prior therapies. This BCMAxCD3 bispecific antibody showed a 71% response rate and 50% complete response rate in the pivotal LINKER-MM1 trial, with a manageable safety profile and convenient response-adapted dosing every four weeks. The approval adds a differentiated treatment option for patients with limited alternatives and reinforces Regeneron’s growing presence in hematologic cancers. An FDA decision in the U.S. is expected by July 2025.
🔹 Vaxart has received approval from the Department of Health and Human Services (HHS) to resume enrollment for its phase 2 trials of an oral COVID-19 vaccine. The company was awarded $456M in government funding during the Biden administration to develop the vaccine pill as part of the next generation of COVID-19 vaccines. Preliminary safety data in 400 participants was considered favorable for continuation of the planned trial. However, the trials were paused in February after the HHS determined more time was needed to review the initial findings. During the pause, Vaxart was unable to continue enrolling the planned 10,000 participants for its phase 2 trial, and caused the company to lay off 10% of its workforce. With the go ahead now granted, Vaxart can proceed with its plans to test their COVID-19 vaccine pill against current mRNA vaccines and is also focusing on its norovirus vaccine pill trials.
🔹 Hoping to expand its kidney disease portfolio, Novartis has struck a deal to acquire Regulus Therapeutics and their autosomal dominant polycystic kidney disease (ADPKD) therapy, farabursen. Farabursen is an oligonucleotide that inhibits miR-17 in kidney tissue to improve kidney function. Phase 1b data improved kidney volume and urinary PC1/PC2 levels as markers of disease severity across 4 separate cohorts of patients. This deal is for $800M upfront with a potential maximum payment of $1.7B if certain clinical endpoints are met.
🔹 Eli Lilly signed a deal with Creyon for global licensing and multi-target research collaboration focused on discovery and development of RNA oligonucleotides in multiple diseases. Creyon, an AI based oligonucleotide development company, aims to produce safe and effective oligonucleotide drug candidates at a faster rate. Lilly put $13M down upfront with the potential to pay $1B in development and sales. Specific lead candidates and disease indications were not discussed.
🔹 Akeso received its first ever FDA approval for penpulimab-kcqx, a PD-1 monoclonal antibody, for both first line and later line treatment of advanced non-keratinizing nasopharyngeal carcinoma (NPC), marking a major regulatory milestone for the Chinese biotech. The approvals were supported by strong safety and efficacy data from global phase 3 trials. This not only expands treatment options for U.S. patients with metastatic NPC but also demonstrates the growing global reach of Chinese innovation in oncology. The announcement coincided with interim survival data for a different Akeso-partnered drug, ivonescimab, developed with Summit Therapeutics, which plunged 32% after the data failed to meet statistical significance in a head-to-head trial against Merck’s Keytruda, triggering multiple trading halts.
🔹 Veracyte has expanded the availability of its Decipher Prostate Genomic Classifier to include patients with metastatic prostate cancer, making it the only Medicare-covered gene expression test available across the full spectrum of prostate cancer. Previously used in localized disease, the test helps clinicians tailor treatment intensity based on tumor aggressiveness. Backed by multiple phase 3 studies, the test is now being rolled out through early access sites, with broader availability expected by June 2025. This launch underscores Veracyte’s continued push to personalize cancer care using genomic insights.
CLINICAL TRIAL UPDATES
🔹 Xencor announced positive interim results from its study of XmAb942, an extended half-life anti-TL1A antibody in development for inflammatory bowel disease (IBD). The therapy was well tolerated in healthy volunteers, with no serious adverse events and a long half-life supporting 12 week maintenance dosing. Based on these findings, Xencor will launch a phase 2b trial in ulcerative colitis in Q3/Q4 2025.
🔹 ALX Oncology announced that its phase 2 ASPEN-03 and ASPEN-04 trials evaluating evorpacept in combination with Merck’s Keytruda for advanced head and neck cancer did not meet their primary endpoints. While the combinations showed manageable safety, the data did not support further pursuit in this indication. The company will instead continue advancing evorpacept in other cancers where it is paired with anti-cancer antibodies like Herceptin and Rituaxan, supported by promising clinical responses and a strong safety profile. Additional data in breast and colorectal cancer are expected soon.
🔹 Boehringer Ingelheim’s investigational oral therapy zongertinib has shown durable and clinically meaningful results in the phase 1b Beamion LUNG-1 trial for patients with HER2 (ERBB2)-mutant advanced non-small cell lung cancer (NSCLC). Presented at AACR 2025 and published in The New England Journal of Medicine, the data revealed a 71% objective response rate, 7% complete responses, and a 96% disease control rate. Median duration of response reached 14.1 months, and median progression-free survival was 12.4 months. Zongertinib also demonstrated activity in brain metastases and maintained a manageable safety profile, with a low incidence of severe adverse events and no drug-related deaths or cardiotoxicity.
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).
Have a great rest of your week and thanks for reading Biotech Blueprint!
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