CARsgen’s CT0596 CAR-T shows strong early results in refractory myeloma

CARsgen Therapeutics Holdings Limited (HKEX: 2171.HK), a biopharmaceutical firm developing advanced CAR T-cell therapies, has reported favorable early clinical data for CT0596—an allogeneic BCMA-directed CAR-T product developed on its THANK-u Plus platform. The preliminary results come from an ongoing exploratory study evaluating safety and efficacy in relapsed/refractory multiple myeloma (R/R MM) and plasma cell leukemia (R/R PCL), adding further momentum to the company’s growing pipeline in immune-oncology.

As of May 6, 2025, eight patients with advanced R/R MM, each of whom had previously failed at least three treatment regimens, were enrolled and infused with CT0596 following FC-based lymphodepletion chemotherapy. CARsgen confirmed that CT0596 was well tolerated with no dose-limiting toxicities (DLTs), high-grade cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), or graft-versus-host disease (GVHD) reported.

What Did the CT0596 Trial Show in Early Results?

The most striking efficacy signals were observed among the first five patients who reached the week 4 response evaluation window. Of these, 60% achieved a stringent complete response (sCR) or complete response (CR), and 80% demonstrated MRD-negative status in the bone marrow—a key clinical indicator in myeloma prognosis. Among two additional patients assessed at day 14, lesion volume reductions of 92% and 65% were documented, while one subject awaits further evaluation under the trial’s efficacy protocol. Importantly, all sCR/CR patients continue to maintain responses, with one monitored through four months post-infusion.

The favorable safety profile and signs of durable remission underscore CT0596’s potential as a new-generation allogeneic therapy capable of overcoming manufacturing and cost limitations inherent in autologous CAR-T treatments.

How Is THANK-u Plus Transforming Allogeneic CAR-T Engineering?

CARsgen’s THANK-u Plus is a refined version of its earlier THANK-uCAR platform. Designed to address the inhibitory effects of NKG2A receptor expression on NK cells, THANK-u Plus enables sustained CAR-T expansion irrespective of NKG2A variability. Preclinical evaluations show enhanced expansion and superior antitumor activity against BCMA and dual CD19/CD20 targets in NK-cell rich environments—suggesting broader application beyond multiple myeloma, including solid tumors and autoimmune conditions.

By deploying this platform, CARsgen aims to reduce production complexity and increase persistence of allogeneic CAR-T cells, enhancing treatment access and scalability.

What Are CARsgen’s Next Clinical Steps?

Following the promising early readouts, CARsgen announced plans to file an Investigational New Drug (IND) application for CT0596 in the second half of 2025. The company intends to present more comprehensive trial results at upcoming oncology and cell therapy conferences to reinforce clinical confidence in its allogeneic pipeline.

The trial’s trajectory also hints at potential expansion into other indications where autoreactive plasma cells are implicated. Further cohort expansion and dose optimization studies are expected to inform CARsgen’s regulatory strategy and next-phase trial design.

How Is CARsgen Positioned in the Global CAR-T Ecosystem?

Based in Shanghai and listed on the Hong Kong Stock Exchange, CARsgen Therapeutics is one of the few China-origin biotechs with a globally integrated CAR-T pipeline spanning hematologic malignancies, solid tumors, and autoimmune diseases. The company has full-stack capabilities from target discovery through commercial-scale GMP manufacturing, enabling rapid clinical translation and scalability.

CARsgen retains global rights over most of its assets, giving it optionality in licensing or co-development deals with multinational pharmaceutical companies. Its focus on in-house technology platforms like THANK-u Plus, designed to improve efficacy, safety, and cost-efficiency, sets it apart in the crowded immunotherapy space.

What Does Market Sentiment Say About CARsgen’s Stock Performance?

CARsgen Therapeutics (HKEX: 2171.HK) surged 9.04% on May 9, 2025, closing at HKD 16.88, following its CT0596 data disclosure. The stock has climbed approximately 181.8% over the past year, reflecting sustained retail and institutional interest in the company’s novel CAR-T programs. Despite this rally, the current price remains 14.66% below its 52-week high of HKD 19.78 recorded in late February.

The company’s market capitalization now stands around HKD 9.67 billion, with a daily volume exceeding 2.5 million shares—marking renewed trading activity around clinical catalysts. However, analyst sentiment remains cautiously optimistic. The average one-year price target hovers at HKD 9.24, implying potential downside risk due to concerns over profitability and high financial leverage. CARsgen reported a net loss of HKD 857.8 million in the trailing twelve months, with a negative EPS of -1.45 and a debt-to-equity ratio of 8.4.

Institutional Flow and Buy/Hold Sentiment: What Are the Signals?

Institutional ownership of CARsgen stock has increased significantly, with a 62.5% rise in institutional holders over the past quarter. Key institutional investors include funds affiliated with Vanguard, which collectively own more than 10 million shares—suggesting growing confidence in CARsgen’s clinical trajectory despite its pre-revenue status.

On balance, the short-term investor consensus remains “Hold,” reflecting both the clinical upside and financial caution. Analysts suggest awaiting further data disclosures and IND filing progress before initiating or expanding positions. With upcoming clinical milestones and potential regulatory interactions on the horizon, any confirmation of durability and expansion potential could serve as a bullish catalyst for the stock.

Is CARsgen Preparing for Global Expansion in Allogeneic Cell Therapy?

CARsgen’s long-term vision includes expanding its CAR-T footprint beyond China and positioning THANK-u Plus as a global standard for scalable, off-the-shelf immunotherapy. This includes potential partnerships, co-development ventures, and eventual commercialization strategies aimed at oncology centers in North America, Europe, and the Asia-Pacific region.

If CT0596 continues to demonstrate efficacy and tolerability across expanded cohorts, CARsgen could become a pivotal player in the next wave of allogeneic CAR-T development. The company’s technology-driven approach, combined with strong clinical signals and a willingness to iterate across platforms, may position it at the forefront of cell therapy innovation.

Early Clinical Promise, Measured Optimism in the Market

CT0596 represents a compelling case study in allogeneic CAR-T development—balancing early efficacy with safety in a notoriously difficult-to-treat patient population. While market sentiment reflects caution due to financial metrics and development risks, CARsgen’s progress offers a tangible glimpse at the future of “off-the-shelf” cell therapies.

Investors, clinicians, and biotech watchers will be closely following the IND submission timeline and next-phase trial data. With institutional interest rising and scientific validation mounting, CARsgen appears to be entering a pivotal stage in its evolution as a global cell therapy innovator.