AstraZeneca's Anselamimab Fails to Meet Primary Endpoint in AL Amyloidosis Trial

AstraZeneca's Anselamimab did not meet the primary endpoint in overall patients with AL amyloidosis. The treatment area breakdown for net sales includes oncology (39.8%), cardiovascular, renal and metabolic diseases (24.4%), respiratory and autoimmune diseases (14.6%), and other diseases (21.2%). Net sales are distributed across the UK, Europe, the US, Americas, and Africa/Asia/Australia.

Title: Immix Biopharma Advances NXC-201 Toward Biologics License Application Submission

Immix Biopharma, Inc. (IMMX) has reported a significant milestone in its ongoing development of NXC-201, a CAR-T cell therapy for relapsed/refractory AL Amyloidosis. The company announced that its lead candidate has demonstrated a class-leading safety profile, including the absence of neurotoxicity in patients with low-volume disease. This development is a crucial step toward the submission of a Biologics License Application (BLA) for NXC-201, which would mark the first approval for a cell therapy in the orphan indication of AL Amyloidosis [1].

The interim results from the ongoing NEXICART-2 study, presented at the 2025 ASCO conference, highlighted the favorable safety profile of NXC-201. The therapy's "digital filter" technology minimizes non-specific activation, enhancing its safety in patients with low-volume disease. This advancement is particularly notable as neurotoxicity is a significant concern in other CAR-T therapies. The company's Chief Executive Officer, Ilya Rachman, M.D., Ph.D., emphasized the importance of this milestone, stating, "We are singularly focused on completing NEXICART-2 for BLA submission, where we are accelerating progress" [1].

Immix Biopharma has also received recognition for NXC-201, including Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug Designation (ODD) from the FDA and EMA. These designations may expedite the development and approval process for NXC-201. The company plans to explore additional indications for NXC-201 post-BLA submission, potentially expanding its market reach and long-term growth [1].

However, the company faces potential challenges. The interim trial data for NXC-201 may not be consistent with future results, raising concerns about the therapy's efficacy. Additionally, Immix Biopharma has not yet received FDA pre-market approval for any drug product, indicating a lack of commercialized success despite ongoing clinical trials. Funding and capital requirements may hinder the company's ability to continue necessary clinical trials and advance product candidates as planned [1].

In the broader context, the AL Amyloidosis market is growing, with an estimated prevalence of 33,277 patients in 2024 and a projected value of $6 billion in 2025. AstraZeneca's Anselamimab, a treatment for AL amyloidosis, did not meet its primary endpoint in overall patients, highlighting the competitive landscape and the need for effective therapies [1].

References
[1] https://www.nasdaq.com/articles/immix-biopharma-reports-class-leading-safety-profile-car-t-nxc-201-low-volume-al