Three World-First Therapies Approved on the Same Day: How to Navigate Commercialization After Solid Tumor CAR-T Is Priced at 990,000 RMB

Data from a confirmatory Phase II randomized controlled trial in China showed that, among treated patients, the median progression-free survival (PFS) was 4.37 months in the Kailimei® group versus 1.84 months in the control group, representing a nearly 70% reduction in the risk of disease progression or death. In terms of median overall survival (OS), the Kailimei group achieved 9.49 months, compared with 5.49 months in the control group.

However, CARsgen Therapeutics specifically pointed out that there are differences in efficacy evaluation between randomized controlled trials (RCTs) and single-arm trials for autologous CAR-T products. Single-arm trials use pre-lymphodepletion imaging as the baseline, which intuitively reflects efficacy, whereas RCTs use pre-randomization imaging as the baseline. During the cell manufacturing period, more than half of the patients experienced increased tumor burden, leading to an underestimation of actual efficacy. Meanwhile, some patients randomized to the CAR-T group failed to receive infusion due to disease progression but were still included in the efficacy analysis. Additionally, patient willingness to enroll was delayed due to concerns about being randomized to the control group, resulting in a worse disease status at trial entry.

A biotech executive deeply involved in the R&D of cell therapy products once told VCBeat, "Approval of  Satricabtagene autoleucel is not an issue; it all depends on how well commercialization can be executed."

Prior to the announcement of the pricing, market estimates suggested it would likely be no less than RMB 1 million.

Saikaize (zevorcabtagene autoleucel) received NMPA approval for market launch in March 2024 as a fourth-line therapy for patients with relapsed or refractory multiple myeloma (R/R MM). The commercialization challenges of CAR-T therapies for solid tumors are not on the same scale as those for hematologic malignancies.

Although CARsgen Therapeutics stands at a new starting point, the sales trajectory of its autologous BCMA CAR-T product, Saikaize, can serve as a reference.

According to the annual report, as CARsgen Therapeutics' first commercialized product, this BCMA CAR-T therapy received 218 valid orders from its partner Huadong Medicine throughout 2025 after being approved in 2024.

The volume ramp-up pace of Saikaize may have given CARsgen Therapeutics more concrete expectations for Kailimei.

To this end, CARsgen Therapeutics has assembled a sales force of nearly 30 people, more than 80% of whom have experience in promoting First-in-Class products.

Xie Xiaocheng, Head of Sales for Kailimei at CARsgen Therapeutics, previously worked at CTTQ and Akeso Biopharma, where he was deeply involved in the commercialization of products such as anlotinib and cadonilimab. Liu Yangyang, Head of Marketing, formerly held senior oncology marketing and oncology business unit director roles at major domestic and multinational pharmaceutical companies, including Bayer, AstraZeneca, and Qilu Pharmaceutical.

Following the inclusion of Saikaize in the commercial health insurance innovative drug list, Kailimei has also initiated the relevant process for commercial insurance access. During an investor briefing, CARsgen Therapeutics explicitly stated that it had already applied for negotiations to be included in the commercial insurance formulary. In particular, for products priced at 990,000 RMB, the extent of commercial insurance coverage may be more critical than the size of the sales team.

Meanwhile, the primary commercialization strategy of CARsgen Therapeutics is to start at the high end and achieve precise coverage.

During the initial commercialization phase, the focus was on 100 leading oncology hospitals and cell therapy centers within comprehensive Grade A tertiary hospitals, with access approvals completed for 55 centers prior to launch. For overseas expansion, CARsgen Therapeutics is advancing in a tiered manner. The first tier includes Singapore, Hong Kong (China), and Saudi Arabia, where registration applications are being rapidly advanced by leveraging core clinical data from mainland China. The second tier comprises ICH member countries such as Australia, South Korea, and Japan, where small-sample confirmatory clinical trials compliant with local regulations will be conducted based on Chinese data. The third tier targets core markets including the United States, Europe, and Canada, where plans are in place to engage with regulatory authorities and initiate pivotal registration clinical trials meeting local standards, capitalizing on the EMA PRIME designation and FDA RMAT designation already obtained.

Currently, only two drugs targeting Claudin18.2 have been approved: Kailimei from CARsgen Therapeutics and Zolbetuximab (Vyloy) from Astellas.

From the perspective of CARsgen Therapeutics, Zolbetuximab was approved for a first-line indication, essentially constituting targeted therapy in combination with chemotherapy; whereas Kailimei was approved for a last-line indication following the failure of second-line or subsequent therapies. The two do not directly overlap in terms of treatment lines, and neither existing product covers the other's indicated scope.

Furthermore, CARsgen Therapeutics is actively expanding the application of Kailimei in early-line cancer treatment and perioperative settings. This includes several investigator-initiated clinical trials, such as adjuvant therapy for pancreatic cancer post-surgery, consolidation therapy following adjuvant treatment for gastric or gastroesophageal junction adenocarcinoma after radical resection, and sequential therapy after first-line treatment. These efforts aim to advance CAR-T therapy from later-line use to earlier stages of treatment.

Undoubtedly, CAR-T therapy for solid tumors offers a new treatment option. However, in recent years, the core of industry discussions has centered on how to transform CAR-T from a treatment accessible to only a few into an affordable therapy for a broader patient population.

Currently, there are two common answers to this question: Allogeneic CAR-T and In Vivo CAR-T.

Li Zonghai also stated candidly at the conference that autologous CAR-T therapy currently remains accessible only to a subset of patients with high paying capacity, while the goal of technological iteration is to make it affordable for a broader patient population.

In theory, universal CAR-T can achieve off-the-shelf availability, eliminating the need to wait for patient blood collection and cell preparation. However, the industry widely considers this a more challenging path than CAR-T for solid tumors.

Li Zonghai even described it as follows: "The challenges of universal CAR-T are truly immense, essentially requiring my utmost effort. Autologous CAR-T for solid tumors is already a ten-star difficulty, while universal CAR-T adds another five stars."

Over the past year, in vivo CAR-T has nearly become the hottest track in global cell therapy. The industry widely considers this to be the core direction for cell therapy over the next decade. 

In Li Zonghai's view, the key competitive factor for the future of in vivo CAR-T therapy will be transduction efficiency. Those who can achieve a higher proportion of T-cell transduction with lower doses are likely to be the first to cross the threshold for industrialization.

"In vivo transduction efficiency is the yardstick that determines life or death." In light of the clinical data recently released in the industry, some practitioners also believe that "clinical response is only observed when lentiviral vectors are administered at high doses reaching the 10^9 level. What does this mean? It indicates poor delivery efficiency."

Currently, industry attention on in vivo CAR-T therapy is largely focused on the field of hematologic malignancies, whereas the treatment of solid tumors presents challenges that are an order of magnitude greater. The tumor microenvironment is more complex, there is a lack of B cell-mediated stimulatory expansion mechanisms as seen in peripheral blood, and the required viral vector doses are expected to rise further. Under these circumstances, the application of in vivo CAR-T therapy in solid tumors still faces significant uncertainty.

In fact, autologous, universal, and in vivo CAR-T products each have their own specific application scenarios. For instance, in patients with T-cell exhaustion following bispecific antibody exposure, the efficacy of autologous or in vivo CAR-T therapies may be compromised, whereas universal CAR-T therapies utilizing T cells from healthy donors could potentially address this issue.

From the perspective of its pipeline under development, CARsgen Therapeutics continues to pursue a three-pronged strategy involving autologous, universal (off-the-shelf), and in vivo CAR-T therapies. 

Notably, CARsgen Therapeutics disclosed that it will no longer develop new pipelines in the autologous CAR-T field. In the field of universal CAR-T therapies, CARsgen Therapeutics has an R&D pipeline targeting BCMA, CD19/CD20, CLL1, NKG2DL, Claudin18.2, and GPC3. In the area of in vivo CAR-T therapies, CARsgen Therapeutics has also initiated projects targeting Claudin18.2, GPRC5D, and other antigens. The dual-target CD19/CD20 in vivo CAR-T product is currently in clinical trials, with initial clinical data expected to be available by the end of 2026. The in vivo CAR-T product for solid tumor indications is planned to submit an IND application in the second half of 2027.

Furthermore, regarding licensing strategy, Li Zonghai revealed in an interview with VCBeat that multiple domestic and international companies are currently in discussions with CARsgen Therapeutics, covering universal and in vivo CAR-T technology platforms and products. The collaboration models vary by project: "For highly competitive areas, we aim to license out early; for less competitive ones, we can take our time to refine the partnership."