SNAPSHOT

Kyverna aims for first-ever CAR-T approval in autoimmune disease after strong trial results

Kyverna Therapeutics plans to seek FDA approval in early 2026 for miv-cel, a CAR-T cell therapy that showed promising results in a trial for stiff person syndrome (SPS), a rare, disabling autoimmune disease with no approved treatments. Kyverna’s stock rose 25% following the news.

Why it matters: This could become the first FDA-approved cell therapy for any autoimmune condition, opening a new frontier in treatment for millions living with such diseases.

Backstory: Originally focused on Treg cell therapy, Kyverna pivoted to conventional CAR-T technology and developed miv-cel, securing major investments. Despite early setbacks and leadership changes, the company refocused on SPS, targeting a faster clinical path in a less crowded space.

Zoom in: In a 26-person Phase 2 study, miv-cel delivered a 46% median improvement on a timed walking test at 4 months and most patients no longer required walking aids or immunotherapy in the same amount of time. 80%+ of patients exhibited a clinically meaningful improvement of 20% or more. The trial showed no high-grade immune or neuro side effects and manageable cases of low white blood cell counts.

Big picture: Kyverna's success could validate CAR-T's potential in autoimmune diseases beyond cancer and reshape the treatment landscape for conditions like lupus, multiple sclerosis, and others. Miv-cel's success may reignite investor confidence in autoimmune cell therapy after recent skepticism.

What's next: Some analysts called the results a "best-case scenario" with potential to "eliminate chronic treatment burden" for patients. Kyverna will present full results at a medical conference in 2026 and submit a Biologics License Application to the FDA in the first half of the year.

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SNIPPETS

What’s happening in biotech today?

🎯 Tumor takedown: Immunome’s investigational therapy varegacestat demonstrated a significant 84% reduction in risk of disease progression or death in patients with desmoid tumors during the phase 3 Ringside trial, meeting its primary endpoint and showing strong secondary outcomes, including a 56% objective response rate compared to 9% for placebo. The once-daily oral gamma secretase inhibitor also significantly reduced tumor volume and pain, outperforming current standard Ogsiveo, and was generally well tolerated with mostly mild side effects. Immunome plans to file for FDA approval in the second quarter of 2026, following its $50 million acquisition of varegacestat from Ayala Pharmaceuticals in 2024.

 💼 Deal spree: Sanofi has entered a new partnership with Dren Bio worth up to $1.7 billion to develop therapies for multiple autoimmune diseases using Dren’s Targeted Myeloid Engager and Phagocytosis Platform. Dren will receive $100 million upfront and lead early discovery, after which Sanofi will handle development, manufacturing, and commercialization, with Dren retaining the option to co-fund further development. This deal follows Sanofi’s $600 million acquisition earlier this year of Dren’s bispecific monoclonal antibody DR-0201 (now called SAR448501), currently in two Phase I trials for undisclosed inflammatory conditions. Dren also holds major partnerships with Novartis and Pfizer, valued at up to $3 billion and $1 billion, respectively.

💸 Payday: Adaptive Biotechnologies has signed two non-exclusive agreements with Pfizer worth up to $890 million to support research in rheumatoid arthritis and other immune-related diseases. Under the deals, Adaptive will receive upfront payments and milestone-based compensation tied to research, development, and sales of a rheumatoid arthritis asset. Pfizer will use Adaptive's immune cell analysis technology to identify T-cell receptors involved in disease. Additionally, Pfizer licensed access to Adaptive's extensive T-cell receptor database to train AI models for drug discovery, with undisclosed financial terms for this multi-year data agreement.

💰 Gout grab: Swedish biopharma Sobi has acquired San Diego-based Arthrosi Therapeutics for up to $1.5 billion, including $950 million upfront and up to $550 million in milestone payments, securing rights to the company’s lead asset, pozdeutinurad. This next-generation, once-daily oral URAT1 inhibitor is in phase 3 trials for progressive and tophaceous gout, with initial data expected in 2026. The acquisition strengthens Sobi’s pipeline in gout, a condition marked by severe joint pain and limited treatment options. Pozdeutinurad aims to improve on older URAT1 inhibitors by offering greater specificity and fewer side effects than earlier drugs like probenecid and AstraZeneca’s now-withdrawn Zurampic.

💥 Trial flop: Argenx will discontinue two Phase 3 trials of its autoimmune drug Vyvgart in thyroid eye disease after an interim analysis showed the treatment was no more effective than placebo, meeting futility criteria. The setback halts efforts to expand Vyvgart’s use beyond its current approvals in myasthenia gravis and another autoimmune disorder, despite generating nearly $3 billion in sales this year. The trials, each enrolling 123 patients, failed to show significant improvement in reducing eye bulging compared to placebo. While this outcome was unexpected given related drug successes in Graves’ disease, Argenx continues to pursue broader applications with additional readouts expected through 2027.

SNAP AGAIN

Sanofi’s multiple sclerosis drug fumbles in key trial, faces FDA delay

Sanofi's experimental multiple sclerosis (MS) drug, tolebrutinib, failed a Phase 3 trial for primary progressive multiple sclerosis and is now also facing a delayed FDA decision for another MS form.

Why it matters: Tolebrutinib was a cornerstone of Sanofi’s $3.7B acquisition of Principia Biopharma and represented one of its most advanced efforts in MS, a highly competitive, high-stakes drug market.

Backstory: Sanofi bought Principia in 2020, betting big on BTK inhibitors to reshape MS treatment. Tolebrutinib was meant to be a flagship product, but regulatory delays and prior safety concerns, including liver toxicity risks, have dogged its progress.

Zoom in: The 767-person PERSEUS trial in primary progressive MS failed its main goal of slowing disability progression. Consequently, Sanofi will not seek approval for this indication. The FDA’s review of tolebrutinib for non-relapsing secondary progressive MS has been delayed beyond the Dec. 28 deadline due to a major amendment. Sanofi has submitted an expanded access protocol, signaling FDA’s openness to limited use despite formal approval delays.

Big picture: The failures cast doubt on Sanofi's MS pipeline and raise pressure on its leadership as rivals like Roche move ahead with their own BTK inhibitors showing stronger results in MS.

What's next: Despite the stumble, analysts caution against writing off tolebrutinib entirely, citing FDA interest in patient access. Sanofi expects more clarity from the FDA in early 2026.

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