SNAPSHOT

Kyverna’s CAR-T therapy shows “unprecedented” results in rare autoimmune disease, eyes first FDA approval

Kyverna Therapeutics reported strong phase 2 data for its CAR-T therapy miv-cel in stiff person syndrome (SPS), showing major functional improvements and positioning it for potential FDA approval as the first CAR-T for autoimmune disease.

Why it matters: SPS has no approved treatments, affects ~6,000 people in the U.S. and progressively worsens over time. Miv-cel’s ability to both improve symptoms and reset the immune system could redefine care, not just for SPS, but for a range of autoimmune diseases.

Backstory: CAR-T therapies are typically used in cancer, but Kyverna is applying the approach to autoimmune diseases by targeting CD19-positive B cells that produce harmful autoantibodies.

Big picture: If approved, miv-cel could open the door for CAR-T therapies across autoimmune conditions like lupus, multiple sclerosis and myasthenia gravis, potentially shifting treatment from chronic management to one-time interventions.

Zoom in: All patients discontinued immunosuppressive therapies as part of the study. The data showed a 46% improvement in walking ability at 16 weeks (primary endpoint). Out of all patients, 81% of patients achieved clinically meaningful improvement and 67% of patients using walking aids no longer needed them. Secondary measures (disability, stiffness, sensitivity) also improved significantly.

What’s next: The safety profile was manageable, with mostly mild cytokine release syndrome and limited neurotoxicity. This undoubtedly helps Kyverna as it plans to file for FDA approval, though uncertainty remains over whether a single-arm trial will suffice. The company is also advancing trials in myasthenia gravis and other autoimmune diseases.

SNAP AGAIN

Revolution Medicines’ RAS inhibitors show strong early results, signaling a broader breakthrough in “undruggable” cancer targets

Revolution Medicines unveiled new data showing its RAS inhibitor daraxonrasib is effective in pancreatic cancer, with promising response rates in early-line treatment and potential to expand across multiple cancers.

Why it matters: RAS mutations drive many hard-to-treat cancers and were long considered “undruggable.” These results suggest a new class of therapies could significantly improve outcomes in pancreatic and lung cancers.

Backstory: RAS was one of the first cancer-causing genes discovered but lacked a targetable structure for decades. Only two RAS-targeting drugs are currently approved, and options remain limited, especially in pancreatic cancer.

Zoom in: Daraxonrasib showed a 47% response rate as a first-line monotherapy in pancreatic cancer and the response rate jumped to 58% when combined with chemotherapy. This is compared to a 52% response rate in lung cancer from Zoldonrasib, a related drug. With the monotherapy, the survival rate was 83% and the progression-free rate was 71% at six months. Prior phase 3 data showed a 6-month survival benefit vs. chemotherapy.

What’s next: The company is advancing phase 3 trials for daraxonrasib. There is also interest on the horizon for a new preclinical candidate (RM-055) that aims to overcome resistance by inactivating RAS directly.

SNIPPETS

What’s happening in biotech today?

💊 Blockbuster bruised: Merck’s kidney cancer drug Welireg suffered a setback after failing in a Phase 3 trial to significantly delay tumor progression or improve survival when combined with Keytruda and Lenvima as a first-line treatment for clear cell renal cell carcinoma. The result undermines Merck’s plans to expand Welireg’s use and reduces its projected revenue potential, which had been expected to help offset future losses from Keytruda’s patent expiration. While the outcome does not affect other ongoing studies, analysts say it limits the drug’s blockbuster potential and may create an opportunity for rival therapies, including Arcus Biosciences’ experimental HIF-2 alpha inhibitor, which is advancing toward late-stage trials.

🔥 Inflammation tamed: BioAge Labs reported encouraging early data for its NLRP3 inhibitor BGE-102, suggesting “best-in-class” potential for reducing inflammation linked to cardiovascular risk, including new Phase 1 results for a lower 60-milligram dose that align with earlier findings from a higher dose. The drug appears well-tolerated and capable of crossing the blood-brain barrier, supporting its potential use across multiple conditions. BioAge is advancing to a Phase 2 trial focused on cardiovascular outcomes, with results expected later this year and a possible Phase 3 start in 2027. Growing industry interest in NLRP3 inhibitors, including major investments and acquisitions, highlights the drug class’s broad therapeutic and commercial promise.

✂️ Relapse slashed: Roche reported strong Phase III results for its investigational BTK inhibitor fenebrutinib in multiple sclerosis, showing significant reductions in relapse rates of over 50% compared to teriflunomide, along with improvements in MRI markers and encouraging trends in disability progression. These findings support its potential as a high-efficacy oral treatment for both relapsing and primary progressive forms of the disease, and Roche plans to submit the data to regulators. However, the results are tempered by a safety concern, as more deaths occurred in the fenebrutinib groups than in the control arm, creating uncertainty that will require careful regulatory review despite otherwise promising efficacy.

🛑 Trial halted: Novartis has halted two Phase 3 trials of its anticoagulant abelacimab in cancer-associated thrombosis after data indicated it would not outperform Eliquis, leading the company to discontinue development in that indication, including a related study in certain cancer-linked blood clots. Despite the setback, Novartis will continue advancing abelacimab in a Phase 3 trial for atrial fibrillation, its primary focus, with plans for a potential approval filing in 2028. The decision follows mixed results across the factor XI inhibitor class, where several competitors have faced failures, though some late-stage successes suggest the approach may still hold promise in select settings.

SPEED READ

More news

• Moderna has launched a Phase 3 trial of its mRNA-based bird flu vaccine after losing U.S. funding, with new support from a global health coalition.

• The FDA approved Merck’s once-daily HIV drug Idvynso, a two-drug, non-INSTI regimen that maintains viral suppression.

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