M&A, Deals, Partnerships:
Quince Therapeutics (SF) Merges with Private Pulmonary Startup to Stay Alive on Nasdaq
After its lead drug for a rare childhood neurological disease failed in a Phase 3 trial earlier this year, cash-strapped Quince Therapeutics is merging with private pulmonary startup Orphai Therapeutics in an all-stock deal that will keep the combined company listed on Nasdaq — though existing Quince shareholders will be left with just 6.9% of the new company.
The merger gives Orphai a public listing and fresh start, with its lead asset being a mid-stage drug for pulmonary hypertension associated with lung disease, effectively transforming Quince into an entirely different company.
Other Interesting News:
Kyverna Therapeutics (SF) Files for FDA Approval of Potentially First-Ever Autoimmune CAR-T Therapy
Kyverna has begun its FDA application for miv-cel, a CAR-T cell therapy for stiff person syndrome — a rare and debilitating neurological disease with no currently approved treatments — putting it on track to potentially become the first CAR-T therapy ever approved for an autoimmune disease.
Early trial data showed striking results, with patients achieving a median 46% improvement in walking ability after 16 weeks, and 67% of patients who needed a walking aid at the start of the trial no longer requiring one — with full one-year data expected later this year.
BioAge Labs (SF) Pivots to Inflammation Drug After Scrapping Obesity Candidate
After ditching its obesity drug last year due to safety concerns, BioAge is now focused on BGE-102, an inflammation-targeting pill that showed an 85% reduction in a key inflammatory marker in early trials — with potential applications across heart disease, cognitive decline, and even eye disease.
The company is backed by a $550 million Novartis partnership and plans to kick off two new mid-stage trials in 2026, positioning BGE-102 as a broad anti-aging and inflammation platform rather than a single-disease drug.
Alumis (SF) Abandons Thyroid Eye Disease Drug, Seeks Buyer or Partner
Alumis is walking away from lonigutamab, a drug it inherited through its merger with Acelyrin last year that was originally positioned as a more convenient alternative to Amgen’s blockbuster Tepezza — citing an increasingly crowded market and a preference to focus resources on its core drug envudeucitinib.
The company is now exploring strategic options for the asset, which could include a sale or partnership, while also potentially facing a $51 million write-down on its books for the shelved program.
Aardvark Therapeutics (SD) Hits Major Setback as FDA Halts Rare Disease Drug Trials
The FDA has placed a full clinical hold on all trials of Aardvark’s ARD-101 — a drug for Prader-Willi syndrome, a rare genetic disorder causing extreme, uncontrollable hunger — after cardiac safety signals were detected, halting a Phase 3 study that was expected to deliver results this summer.
With its stock down over 50% since the initial pause in February and a cash runway only extending to mid-2027, Aardvark is now unblinding its trial data to assess its options — making the next few months critical for the company’s survival.
Model Medicines (SD) Advances AI-Discovered Drug Candidates Toward Human Trials
Model Medicines, a La Jolla-based AI drug discovery company, has virtually screened 325 billion molecules and is preparing to bring two drug candidates into human trials in 2026 — one targeting cancer and cardiovascular disease, and another a broad-spectrum antiviral that showed stronger preclinical results than key ingredients in both Veklury and Paxlovid.
Unlike most AI drug companies, Model trained its models on small but diverse datasets to discover entirely novel chemistry rather than variations of existing drugs — a contrarian approach the founder believes is key to finding true breakthroughs.
BioMarin Pharmaceutical (SF) Hits Setback with $270 Million Rare Disease Drug
BioMarin’s BMN 401, a treatment for a rare genetic disorder that causes abnormal bone development in children, only hit one of two key goals in a Phase 3 trial — improving a key biomarker but failing to show meaningful skeletal healing on X-rays or improvement across any secondary endpoints.
The disappointing result is a significant blow to BioMarin, which paid $270 million to acquire the drug just over a year ago — though the company says it is still evaluating next steps for the program given the severe unmet need in this condition.
Amgen (LA) Faces Growing Global Scrutiny Over Rare Disease Drug Tavneos
Amgen is under fire from multiple directions over Tavneos — the FDA wants to pull its approval over clinical trial manipulation concerns, European regulators are investigating data integrity, and a Japanese pharma partner is now warning doctors to stop prescribing it after reporting 20 patient deaths linked to severe liver injury.
Amgen is standing firm, pointing out that no US deaths have been linked to the drug despite treating a similar number of patients as Japan, and continues to call Tavneos safe and effective for the rare immune disorder it treats.
Design Therapeutics (SD) Reports Promising Early Data for Friedreich Ataxia Drug
Design Therapeutics’ drug DT-216P2 showed meaningful improvements in movement, balance, and fatigue in patients with Friedreich ataxia — a rare degenerative genetic disease — after just four weeks of treatment, marking what the company believes is the first time a drug has both increased a key deficient protein and produced measurable clinical improvements in this condition.
The drug was well-tolerated with no serious side effects, and the company plans to move toward a registration trial — the final step before seeking FDA approval — with a program update expected in Q4 2026.
