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77 Years of Nothing. Then Zanvastro.
Alexander disease has had exactly zero approved treatments since it was first described in 1949. That changed this week when the FDA approved Ionis Pharmaceuticals' Zanvastro (zilganersen), an antisense drug injected into the spinal fluid every 12 weeks. In the pivotal trial, Zanvastro demonstrated a least-squares mean difference of 33.3% in gait speed preservation versus pooled control at week 61. That's the difference between walking to the kitchen and needing a wheelchair. The drug also showed benefits across communication, swallowing, and seizure measures. With roughly 500 cases ever reported worldwide, Alexander disease won't be a blockbuster market, but the approval is the third neurological win for Ionis's antisense platform, validating a playbook the company is now running against Angelman syndrome and beyond.
Why it matters: Each approved antisense drug for a genetically defined brain disease builds the regulatory, manufacturing, and commercial infrastructure that makes the next one easier. Zanvastro isn't just a win for 500 patients; it's a proof point for an entire therapeutic class now accelerating across rare neurology.
Read more →Oncology Shakeups
Keytruda Just Lost a Survival Fight for the First Time
A bispecific antibody from Chinese biotech Akeso beat Keytruda on overall survival in a head-to-head Phase 3 lung cancer trial. That's a first for the $32 billion franchise. Earlier data showed ivonescimab nearly doubled progression-free survival (11.14 vs. 5.82 months). The catch: it was a China-only study. Summit Therapeutics is running a global confirmatory trial, with an FDA decision on a different ivonescimab indication due November 14.
Read more →AbbVie Shows Up Late to the Myeloma Party With the Best Data in the Room
AbbVie's bispecific T-cell engager etentamig posted a 74% response rate and cut progression risk by 60% in relapsed myeloma, outperforming standard care across both endpoints. The problem: J&J, Pfizer, and Regeneron already have approved competitors on pharmacy shelves. A convenient once-monthly dosing schedule could be AbbVie's differentiator, with a regulatory filing planned for the end of 2026.
Read more →Pfizer Sells a Cancer Drug for Less Than a Malibu Condo
A clinical-stage ADC inherited from the $43 billion Seagen acquisition just left Pfizer's building for $12 million, landing at tiny Canadian biotech Medicus Pharma. The drug wasn't killed by bad data; it was killed by portfolio triage. Pfizer retains milestone rights exceeding $1 billion if the drug works, but the fire-sale price adds to growing skepticism about whether the Seagen deal will ever pay off.
Read more →A China-India Tag Team Is Coming for Keytruda's Crown
Indian pharma giant Cipla licensed U.S. rights to a Chinese-developed Keytruda biosimilar from Qilu Pharmaceutical, creating an unconventional supply chain aimed at oncology's biggest cash cow. The biosimilar is in Phase III trials with Keytruda's core patent expiring around December 2028. Cipla plans to build a portfolio of roughly 10 U.S. biosimilars over the next five to six years.
Read more →Clinical Trials & Safety Signals
Three Autoimmune Patients Died. Now CAR-T's Biggest Bet Is on Pause.
Novartis halted eight CAR-T trials for autoimmune diseases after three patient deaths from a severe immune overreaction. These patients had lupus and arthritis, not terminal cancer, which makes the risk calculus fundamentally different. Bristol Myers Squibb also paused its own autoimmune CAR-T studies. The entire field now faces a hard question: can a therapy built for dying cancer patients ever be safe enough for chronic disease?
Read more →Four Years of Data Suggest This Drug Might Rewire Kids' Brains
Stoke and Biogen's zorevunersen showed durable seizure reduction and cognitive improvement across four years of treatment in Dravet syndrome, a severe childhood epilepsy. Of 75 patients, 77% stayed in the study at the four-year mark. The disease-modifying signal (seizure control plus functional gains) is unusual for this condition. A pivotal Phase 3 readout is expected in the second half of 2027.
Read more →Science & Discovery
Roche Paid $190M for a Weight-Loss Drug That Might Spare Your Muscles
Up to 40% of weight lost on GLP-1 drugs comes from muscle, not fat. Roche licensed Hanmi's HM17321, a completely different class of obesity drug (a UCN2 analog), after preclinical data showed it cut fat by 33.6% while actually increasing lean mass by 11.9% in mice. It's still years from human efficacy data, but it slots into Roche's growing three-pronged obesity strategy alongside its GLP-1 and amylin programs.
Read more →A Single Dose of Psilocybin Blocked Chemo Nerve Pain for 8 Months in Mice
A significant proportion of chemo patients develop nerve damage (chemotherapy-induced peripheral neuropathy), and there are zero approved drugs to prevent it. A study in Science found that two doses of psilocybin given before chemotherapy prevented neuropathic pain in mice for up to eight months by preserving mitochondrial transport in nerve cells. Clinical trials in human patients are reportedly in the works.
Read more →Regulatory & Funding
The FDA Is Letting AI Devices Treat Patients Before Proving They Work
Select digital health devices can now reach real patients before full FDA authorization under a new pilot called TEMPO. Selected products include an AI blood pressure co-pilot and an AI-powered cognitive behavioral therapy agent. The FDA explicitly has not evaluated their effectiveness yet. Companies must collect real-world data while treating patients, with full authorization expected to follow.
Read more →The MyoKardia Team Just Raised $400M for Their Cardiac Comeback
Kardigan, founded by the executives who built MyoKardia (sold to BMS for $13.1 billion), priced its upsized IPO at $16 per share, raising $400 million. The company has three cardiac programs, with two expected to deliver data readouts in the first half of 2027. It's among the largest biotech IPOs of 2026, signaling that investors are willing to pay a premium for proven teams in a recovering market.
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