Biotech
Alethio Therapeutics has raised $90 million in Series A funding to advance its treatments for rare blood cancers, specifically myeloproliferative neoplasms (MPNs). The company's lead program, ATX-011, is a monoclonal antibody targeting G6b, a receptor on platelets and certain bone marrow cells, with the aim of providing a more precise approach than existing therapies. Preclinical studies suggest ATX-011 is well-behaved, and it is expected to enter clinical testing in the first half of 2028. The funding round was co-led by Sofinnova Partners and RA Capital Management, with backing from Cancer Research Horizons, Oxford Science Enterprises, and the University of Oxford. Alethio spun out of research at the University of Oxford and was co-founded by Adam Mead and Beth Psaila. The company also has a second drug prospect, an antibody-drug conjugate, in development for myelofibrosis.
Democrats regained majorities in the House and Senate in the midterm elections, a shift that analysts say will escalate the biopharmaceutical sector's broader recovery. Drug pricing may not be a hot-button issue given existing programs under Biden and Trump. Analysts also expect greater scrutiny of FDA and HHS leadership, vaccine policy, and China-related biotech collaborations, but not a blanket ban on data or M&A. Early stock performance suggests investor optimism.
Vertex Pharmaceuticals reported third-quarter revenue of $3.44 billion, a 12% increase year-over-year, with over 95% coming from its cystic fibrosis portfolio. The company slightly raised its annual revenue guidance to $13.15-$13.25 billion. Despite mixed performance in non-CF drugs and a 3% stock dip, executives expressed strong confidence in their ability to launch povetacicept, a kidney disease treatment awaiting an FDA decision by the end of the month. Analysts view the reassurances as encouraging, highlighting the potential for a renal franchise exceeding $10 billion in peak sales.
The FDA has agreed to review UniQure's approval application for its Huntington's disease gene therapy and is expected to decide by May 1. Bright Minds Biosciences' application for a mid-stage trial of oral antiseizure drug BMB-101 was placed on partial clinical hold due to mouse toxicity. Specific Biologics closed a $35 million Series A round led by Hatteras Venture Partners. Teva Pharmaceuticals will stop early a mid-stage study of emrusolmin in multiple system atrophy after an interim futility analysis. CinRx Pharma and Graviton BioScience formed a joint venture, CinRock Pharma, to develop ROCK2-inhibiting drugs for cardiovascular conditions.
Karyopharm Therapeutics, a cash-strapped cancer drugmaker, has filed for Chapter 11 bankruptcy to sell itself and reorganize. Through the filing, Karyopharm intends to hold an auction with Ayrmid, parent of cell therapy maker Gamida Cell, as the lead bidder. Karyopharm will continue operating during the proceedings with a $55 million bankruptcy loan from its lenders, and an acquisition should close in early 2027. Karyopharm sells the blood cancer drug Xpovio, approved for multiple myeloma in 2019 and later for a common lymphoma, with potential myelofibrosis approval pending. Despite bringing a drug to market, Karyopharm has not turned a profit and reported $115 million in 2025 net Xpovio sales, $196 million in losses, and a $1.8 billion accumulated deficit. The company warned it only had cash to last through mid-2026 and faces $15.8 million in debt due. Ayrmid CEO Joe Wiley said adding Xpovio to Gamida Cell's blood cancer cell therapies could build a broader hematology business. Karyopharm shares fell 83% to 11 cents, giving a market value of $2.5 million, with $55 million in cash as of June 30.
AstraZeneca reiterated its $80 billion revenue target by 2030 and unveiled a $1.5 billion annual cost-savings program, 'Reshaping for Sustainable Growth,' which includes a $2 billion restructuring charge and reinvestment in R&D and AI. Alnylam Pharmaceuticals reported $1.13 billion in third-quarter Amvuttra revenue, in line with expectations, but shares fell more than 20% on concerns about Medicare reimbursement changes and UnitedHealth's restrictive policy, raising questions about the drug's growth outlook. Regeneron Pharmaceuticals shares rose 13% after reporting record quarterly sales: Dupixent at $6.5 billion (up 35%), high-dose Eylea at $700 million, and Libtayo at $550 million, all beating estimates. Analysts highlighted Regeneron's successful defense against biosimilar competition and noted pipeline prospects, including an RNA-based medicine licensed from Alnylam for MASH.
At the European Society for Medical Oncology (ESMO) meeting, Moderna and Merck's individualized neoantigen therapy intismeran autogene met high expectations in melanoma but Moderna's shares fell double digits on commercial and broader-tumor doubts. Tango Therapeutics' vopimetostat combo showed strong response rates in pancreatic cancer, yet its stock dropped about 20% amid layoff news and competition concerns. Xencor's XmAb819 posted a 46% response rate in kidney cancer, making it a top Wall Street winner.
Two months after Actio Biosciences sold some assets to Jazz Pharmaceuticals for $1.3 billion, its team has launched Setara Biosciences with $90 million in financing. Setara aims to advance a drug for Charcot-Marie-Tooth disease and overactive bladder, along with discovery-stage programs in rare diseases, epilepsy, and migraines. The lead program, STAR-0871, an oral TRPV4 ion channel inhibitor, is preparing for a Phase 1b study in severe Charcot-Marie-Tooth by year-end and a Phase 2 study in overactive bladder. Actio alumni David Goldstein (CEO), David Breckenridge (CSO), and Cayce Denton (CFO) lead Setara, backed by investors including Deerfield Management, Canaan, and Jazz.
Aspen Neuroscience raised $140.6 million in its initial public offering, selling roughly 9.4 million shares at $15 each, the low end of its proposed range. The company's lead candidate, sasineprocel, is a one-time personalized cell therapy designed to replenish dopamine-producing neurons in Parkinson's disease patients. Early Phase 1/2a data suggest potential functional improvements and a favorable safety profile, with long-term data expected in 2028 and a Phase 3 trial planned by the end of next year. Aspen is one of several companies pursuing cell therapies for Parkinson's, including Bayer subsidiary BlueRock Therapeutics. The IPO adds to a growing number of biotech offerings in 2026, with 29 biotechs having gone public on U.S. exchanges so far this year.
Flagship Pioneering has launched Etcelera, a new biotechnology company formed by merging three of its portfolio companies: Etiome, Cellarity, and Empress Therapeutics. Led by Adrian "Ad" Rawcliffe, former CEO of Adaptimmune, Etcelera aims to combine artificial intelligence with diverse drug discovery tools to better predict clinical success of experimental medicines. The company will leverage perturbation data and machine learning to identify novel disease drivers and develop small molecule drugs, initially targeting hematological conditions, immune diseases, and metabolic-fibrotic disorders. No new funding was announced, but the three predecessor companies had raised $374 million in venture capital. Etcelera retains a 2022 partnership with Novo and is pursuing another alliance with an unnamed large pharmaceutical firm. Flagship will move Cellarity's oral sickle cell disease drug into a separate standalone company.