BHVN News & Events

Executives from 15 rare disease-focused nonprofits met on Wednesday with acting FDA Commissioner Kyle Diamantas and senior staff. In the meeting, Diamantas pledged to prevent political interference, Endpoint News' Zachary Brennan reports. Publicly traded large-cap drugmakers include AstraZeneca (AZN), Bristol Myers (BMY), Eli Lilly (LLY), GSK (GSK), Johnson & Johnson (JNJ), Merck (MRK), Novartis (NVS), Pfizer (PFE), Roche (RHHBY) and Sanofi (SNY). Biotech stocks include Replimune (REPL), Moderna (MRNA), Sarepta (SRPT), Xencor (XNCR), PTC Therapeutics (PTCT), Biohaven (BHVN), Edgewise Therapeutics (EWTX), Design Therapeutics (DSGN), Ultragenyx (RARE), Dyne Therapeutics (DYN), Wave Life Sciences (WVE), MeiraGTx (MGTX), uniQure (QURE), Regenxbio (RGNX), Novavax (NVAX) and Lexeo Therapeutics (LXEO).

The U.S. Food and Drug Administration said it has issued draft guidance to help developers bring promising gene therapies to patients more efficiently by making greater use of existing scientific and regulatory knowledge. When finalized, the guidance will outline how sponsors can use publicly available information and established platform knowledge, including chemistry, manufacturing and controls data, nonclinical study results and clinical information, to streamline regulatory submissions for human gene therapy products that use genome editing in human somatic cells. "Today's action reflects the FDA's commitment to get safe and effective cell and gene therapies to patients faster, particularly those living with rare and life-threatening diseases who have few or no other treatment options," said Karim Mikhail, B. Pharm., M.S., Acting Director of the Center for Biologics Evaluation and Research. "By providing information on how companies may build on what is already known we are accelerating innovation without compromising the rigorous scientific standards that patients and the public depend on.Ultimately, this is about making sure that the promise of gene therapy reaches the patients who need it most, as quickly and safely as possible." This draft guidance supports the development of a wide range of cell and gene therapy products, including those that use genome editing, and is part of a broader set of complementary FDA actions in this area. Publicly traded large-cap drugmakers include AstraZeneca (AZN), Bristol Myers (BMY), Eli Lilly (LLY), GSK (GSK), Johnson & Johnson (JNJ), Merck (MRK), Novartis (NVS), Pfizer (PFE), Roche (RHHBY) and Sanofi (SNY). Biotech stocks include Replimune (REPL), Moderna (MRNA), Sarepta (SRPT), Xencor (XNCR), PTC Therapeutics (PTCT), Biohaven (BHVN), Edgewise Therapeutics (EWTX), Design Therapeutics (DSGN), Ultragenyx (RARE), Dyne Therapeutics (DYN), Wave Life Sciences (WVE), MeiraGTx (MGTX), uniQure (QURE), Regenxbio (RGNX), Novavax (NVAX) and Lexeo Therapeutics (LXEO).

Biohaven announced the initiation of first-in-human dosing for BHV-8100, its oral, brain-penetrant pyruvate kinase M2 isoform modulator. Dr. Bruce Car, Chief Scientific Officer of Biohaven, stated, "BHV-8100 represents a potential fundamental breakthrough in a new class of medicines with potential therapeutic effects in both systemic and brain disorders that involve metabolic and immune dysfunction. PKM2 acts as a bridge between metabolism and immune function. By modulating PKM2, we are striving to correct the core energy deficit known to underpin a number of inflammatory and neurological disorders including atopic dermatitis, Alzheimer's disease, and retinitis pigmentosa. Achieving this important milestone to advance a convenient oral, once-daily and brain-penetrant PKM2 modulator reflects Biohaven's mission to deliver innovative and potentially transformative therapies in areas of profound unmet medical need."

Biohaven reported "compelling" results regarding its proprietary extracellular MoDE - BHV-1300 - and TRAP - BHV-1400 - degrader platform. Key highlights from the Phase 1b Graves' Disease patient experience with BHV-1300 include: weekly administration of BHV-1300 1000 mg achieved mean reductions of pathogenic TSHR-IgG1 of greater than 80% by week 12 in patients with Graves' hyperthyroidism, a deeper reduction than demonstrated with the competition; patients with elevated thyroid hormones experienced a normalization of free T4 at a median of 3 weeks and a normalization of free T3 within a median of 5 weeks, with reported improvements in classic symptoms of hyperthyroidism. BHV-1300 has been safe and well-tolerated through 12 weeks of dosing. BHV-1300 preserved the immune protection that FcRn inhibitors have not demonstrated. Biohaven is advancing BHV-1300 to a pivotal trial in Graves' disease in the coming weeks. Key highlights from the Phase 1b IgA nephropathy disease patient experience with BHV-1400 include: administration of BHV-1400 achieved deep, rapid, and sustained mean reductions of pathogenic Gd-IgA1 of greater than 60% within 48 hours of subcutaneous administration and 70% within the one month of dosing in patients with IgAN; participants also subjectively reported improvements in fatigue. BHV-1400 has been safe and well-tolerated throughout one month of dosing. Biohaven plans to study BHV-1400 in patients with IgAN, initiating a pivotal trial in mid-2026 designed to support regulatory approval.