Kyverna Submits BLA for First-in-Class Autoimmune CAR-T Therapy; ImmunityBio and Vanda Report Positive Late-Stage Trial Results

CAR-T Therapy for Autoimmune Diseases Expected to Submit for Marketing Approval Next Year

Kyverna Therapeutics announced positive topline results from the registrational Phase 2 clinical trial, KYSA-8, of its investigational therapy mivocabtagene autoleucel (miv-cel, formerly known as KYV-101), for the treatment of Stiff Person Syndrome (SPS).Miv-cel is a fully human, autologous, CD19-targeted CAR-T cell therapy incorporating the CD28 co-stimulatory domain.SPS is a disabling, progressive autoimmune disease with no treatments currently approved by the U.S. FDA.

The research results show that,Miv-cel achieved statistically significant clinical benefits across all primary and secondary endpoints, with a single dose capable of reversing disability status and enabling patients to discontinue prior immunotherapy.In terms of motor function, miv-cel brought significant and lasting improvements in the Timed 25-Foot Walk (T25FW) test.Compared with the baseline, the median improvement at week 16 reached 46% (p=0.0002), with 81% of patients showing an improvement in walking ability of more than 20%, which is considered the threshold for clinical significance.In terms of safety, miv-cel was generally well-tolerated. Kyverna plans to submit miv-cel for the SPS indication in the first half of 2026.Biologics License Application (BLA). According to the press release,This therapy is expected to become the first CAR-T therapy approved by the FDA for autoimmune diseases.

Phase 2/3 Trial of Breakthrough Cancer Therapy Reports Positive Results Again

ImmunityBio Announces Positive Efficacy of BCG Combined with Anktiva (Nogapendekin Alfa Inbakicept) in High-Risk, High-Grade Papillary-Only Non-Muscle-Invasive Bladder Cancer (NMIBC) Patients Unresponsive to BCG

The QUILT-3.032 Phase 2/3 clinical study announced this time included a total of 80 subjects;At 36 months, the disease-specific survival rate was 96%, and the progression-free survival rate was 83%, demonstrating the long-term efficacy persistence of Anktiva in the high-grade papillary NMIBC population without carcinoma in situ (CIS).In addition,The bladder removal surgery avoidance rates at 12 months and 36 months reached 92% and 82%, respectively., further supporting its durable response and bladder preservation effects. In terms of safety, Anktiva combined with BCG was well-tolerated, with a safety profile consistent with BCG alone.

Anktiva is an IL-15 superagonist. The therapy was granted Breakthrough Therapy Designation by the FDA in 2019., for use in combination with BCG to treat patients with non-muscle-invasive carcinoma in situ of the bladder who previously responded poorly to BCG. In April 2024, the U.S. FDA approved Anktiva for use with BCG to treat adult patients with BCG-unresponsive non-muscle-invasive bladder cancer with carcinoma in situ, with or without papillary tumors. Notably, the industry media Evaluate listed this therapy as one of the 10 potential blockbuster treatments expected to be approved in 2024.

Innovative Monoclonal Antibody Immunotherapy Submits Application for FDA Approval

Vanda Pharmaceuticals announced that it has submitted a Biologics License Application (BLA) to the U.S. FDA for its investigational therapy imsidolimab, intended for the treatment of Generalized Pustular Psoriasis (GPP). GPP is a rare, chronic, and potentially life-threatening autoinflammatory skin disease, typically characterized by sudden widespread outbreaks of pustules and erythema, accompanied by systemic symptoms such as fever and fatigue; its primary driver isIL36RNLoss-of-function mutation.Imsidolimab is a novel IgG4 antibody that targets the IL-36 receptor to inhibit the IL-36 receptor signaling pathway.This mechanism of action aims to address the common endogenous IL-36RA regulatory defect in GPP patients, which is usually associated withIL36RNRelated to gene mutations.

This BLA submission is primarily based on the positive results of the global Phase 3 GEMINI-1 and GEMINI-2 studies. The analysis shows,A single intravenous administration can achieve rapid disease clearance, enabling patients to reach a skin state of "clear" or "almost clear" lesions.; and in the subsequent approximately 2-year maintenance study, the efficacy was sustained through monthly dosing. In terms of safety, imsidolimab demonstrated a favorable safety profile, with no clinically significant safety signals observed.

References:

[1] Vanda Announces Submission of Biologics License Application to the FDA for Imsidolimab for the Treatment of Generalized Pustular Psoriasis. Retrieved December 16, 2025 from https://www.prnewswire.com/news-releases/vanda-announces-submission-of-biologics-license-application-to-the-fda-for-imsidolimab-for-the-treatment-of-generalized-pustular-psoriasis-302641858.html

[2] Kyverna Therapeutics Announces Positive Topline Data from Registrational KYSA-8 Trial of Miv-cel (KYV-101) in Stiff Person Syndrome. Retrieved December 16, 2025 from https://ir.kyvernatx.com/news-releases/news-release-details/kyverna-therapeutics-announces-positive-topline-data

[3] ANKTIVA® with BCG Demonstrates 96% Survival from Bladder Cancer at Three Years with Median Survival Not Yet Reached in BCG-Unresponsive High-Grade Papillary-Only Non-Muscle Invasive Bladder Cancer. Retrieved December 16, 2025 from https://www.businesswire.com/news/home/20251216185945/en/ANKTIVA-with-BCG-Demonstrates-96-Survival-from-Bladder-Cancer-at-Three-Years-with-Median-Survival-Not-Yet-Reached-in-BCG-Unresponsive-High-Grade-Papillary-Only-Non-Muscle-Invasive-Bladder-Cancer

Disclaimer: This article is for information exchange purposes only. The views expressed in the article do not represent the position of WuXi AppTec, nor does WuXi AppTec support or oppose the views mentioned in the article. This article is not a recommendation for treatment plans. For guidance on treatment options, please visit a正规 hospital.

Copyright Statement: Individuals are welcome to share this article on their Moments. Unauthorized reproduction by media or institutions in any form to other platforms is prohibited. For authorization to reproduce, please reply "reprint" on the WeChat Official Account of "WuXi AppTec" to obtain reprint guidelines.