December 2, 2025 — Hopstem Therapeutics, a leader in iPSC-derived neural cell therapeutics, announced that its global first-in-class allogeneic forebrain neural progenitor cell therapy, hNPC01 injection, was granted Fast Track Designation (FTD) by the U.S. Food and Drug Administration (FDA) for treating chronic motor dysfunction caused by ischemic stroke. This designation marks global authoritative regulatory recognition of hNPC01’s clinical value and innovation, laying a critical foundation for accelerating the product’s international clinical development and future commercialization.

FDA Fast Track Designation: Accelerated Pathway for Unmet Medical Needs

According to Section 112 of the Food and Drug Administration Modernization Act of 1997 (FDAMA), Fast Track Designation is intended to facilitate the development and expedite the review of drugs/biologics for treating "serious or life-threatening conditions" and that "demonstrate the potential to address unmet medical needs." For hNPC01 Injection, this designation means:

1. More frequent interactions with the FDA and tailored guidance covering key aspects such as clinical trial design and data requirements, helping to optimize the development pathway.

2. Eligibility for Rolling Review of a Biologics License Application (BLA), allowing the submission of completed sections of the application for review as they are ready, rather than waiting until all sections are complete, significantly shortening the review timeline.

3. Potential eligibility for Priority Review or Accelerated Approval in the future, further expediting the path to market.

4. Eligibility for the Expanded Access (often referred to as "compassionate use") pathway, allowing more eligible patients with no other treatment options access to the potential therapy.

Notably, the FDA’s decision was primarily based on the "significant unmet medical need" in the disease area targeted by hNPC01 and the "potential of hNPC01 to address this need." Taking stroke sequelae as an example, there are nearly 100 million stroke survivors globally, over 20 million in China, and more than 3 million new stroke cases annually in China alone. Due to the lack of effective treatments for neural injury, patients often face lifelong disability. Similarly, other forebrain neural injuries or degenerative diseases, such as traumatic brain injury and cerebral palsy, also lack plausible cause-targeting and effective treatments. hNPC01, as a neural progenitor cell therapy, offers a novel therapeutic approach for such diseases through dual mechanisms: replacing damaged neuronal cells via in vivo differentiation and promoting the repair of existing neural circuits.

About hNPC01, the global first-in-class allogeneic cell therapy for cerebral neural injuries

hNPC01 Injection is an allogeneic forebrain neural progenitor cell therapy derived from human induced pluripotent stem cells (iPSCs), featuring the following core strengths:

High Purity, Consistency, and Safety: Hopstem’s unique second-generation RONA differentiation method, enables stable batch production of neural progenitor cells capable of differentiating into cells consistent with human forebrain tissue. The forebrain-fated neural cells in hNPC01 exceeds 99%, with no detectable residual iPSCs or other tissue cell types. The manufacturing process has demonstrated high batch-to-batch consistency and stability. The safety of hNPC01 was strongly supported by nonclinical studies and phase 1 study human data to date.

Plausible Cell Replacement Mechanism: hNPC01 can further differentiate into functional cortical neurons both in vitro and in vivo. Mechanism studies have shown that electrophysiological activities were detected in 90% of differentiated human neurons starting at one month after hNPC01 transplantation in rat models of cerebral ischemic stroke. These human neurons were able to form neural circuit connections with rat thalamus and other brain regions, and significant improvement in motor function recovery was observed^[1].

Advanced Clinical Development with Significant Motor Function Improvement: Following hNPC01's Investigational New Drug (IND) approvals in China and the U.S. in June, 2023 and March, 2024, a total of 23 ischemic stroke participants with chronic motor dysfunction were enrolled in the Phase I clinical studies in China, all have completed 12-18 months of follow-up except 3 lost-to-follows. These studies demonstrate a favorable safety profile and therapeutic potential ^[2], laying a solid foundation for future pivotal studies:

1. All dose groups reached statistically significant differences at 12 months after treatment comparing to baseline in Fugl-Meyer Motor Scale (FMMS, total 100 points for measuring upper and lower limb motor functions). The combined target subgroup improvement reached statistically significant differences at 12 months comparing to historical randomized rehabilitation control group or sham surgery group in FMMS or FMA-UE (Fugl-Meyer Motor Scale for Upper Extremity) with p value less than 0.001 or 0.005.

2. The average upper limb improvement (FMA-UE) is approximately 11.3 points in the low-dose group and approximately 13.7 points in the high-dose group after hNPC01 treatment at 12 months. The responder rate (proportion of participants achieved a clinically significant improvement of FMA-UE > 5 points) in the target population is 92%. Improvements in lower limb motor function and language function were also observed with this one-time causative-targeting treatment. In contrast, the only approved therapy for treating chronic upper-limb dysfunction due to stroke in global, Vagus Nerve Stimulator (VNS), requires 3 months of rehabilitation, was approved with an average upper limb improvement of 5.8 points, a responder rate of 47%, and no reported improvements in lower limb or language function ^[3].

3. The majority of participants showed continuous improvement in upper and lower limb motor function up to 12 or 18 months after treatment. Nine participants who completed 18 months of follow-up showed further FMMS improvements ranging from 1 to 10 points on top of their 12-month scores.

4. To date, 9 participants have 1 grade improvement in the modified Rankin Scale (mRS, measuring disability), which is nearly 70% in the target population, indicating hNPC01’s potential to bring benefits to these disable chronic stroke participants and improving their quality of life.

Company Statement and Future Outlook

Dr. Shuning Zhang, Vice President of Medical and Clinical Affairs from Hopstem Biotechnology, stated: "As the world's first forebrain neural cell therapy product, hNPC01's receipt of FDA Fast Track Designation is not only a recognition of the product's innovation and clinical value but also an affirmation of China's compliant clinical data supporting the accelerated overseas development of global first-in-class products. We will fully leverage this regulatory support, maintain close communication with FDA, optimize the design of late-stage clinical trials, and accelerate the product approval in regions including China and the US, while strictly adhering to the compliance requirements to ensure product consistency, stability, safety, and efficacy. We will remain patient-orientated and are committed to bringing this innovative therapy to market as soon as possible, offering new hope to neural injury patients worldwide."

It is reported that Dr. Jing Fan, Founder and CEO of Hopstem Biotechnology, has been invited to deliver a keynote presentation on the clinical progress of hNPC01 for treating stroke sequelae at the International Society for Stem Cell Research (ISSCR) forum "Accelerating Pluripotent Stem Cell-Derived Cell Therapies - Starting with the End in Mind" in Boston on December 11-12 this year. Hopstem plan to proceed to a multi-region pivotal clinical trial of hNPC01 treating chronic motor dysfunction due to ischemic stroke, to further validate the existing clinical findings and support priority review and market approval in multiple regions. The company also plans to initiate clinical studies for hemorrhagic stroke, traumatic brain injury, cerebral palsy, and other conditions to address the significant unmet clinical needs in these neural injury diseases. Additionally, in accordance with FDA Fast Track requirements, Hopstem will soon announce its expanded access policy and eligible indications for hNPC01, providing options to clinicians for patients suffering from severe diseases lacking treatment alternatives.

Disclaimer

1.Fast Track Designation (FTD) is an FDA regulatory pathway established to expedite the development of products for serious conditions. It does not mean the product has received marketing approval, nor does it guarantee ultimate marketing approval.

2.hNPC01 Injection is currently still in the clinical trial stage. Its safety and efficacy have not been fully validated. Relevant clinical outcomes are subject to the final clinical trial data.

3.This press release is for informational purposes only and does not constitute any medical advice or product promotion. Patients should choose treatment options under the guidance of physicians.

4.This press release contains certain forward-looking statements. These statements are based on the current expectations and beliefs of the company's management team and are subject to inherent uncertainties, risks, and changes in assumptions that may cause actual results to differ from those described in the forward-looking statements.

References:

1. Xiao H, et al. Forebrain neural progenitors effectively integrate into host brain circuits and improve neural function after ischemic stroke. Nature Communications. 2025; 16:5132.

2. [Breakthrough Progress in iPSC Cell Therapy] Hopstem's hNPC01 Injection Reached 12-Month Endpoint in Phase I Clinical Study for Ischemic Stroke Hemiplegia, Showing Significant Clinical Improvement.

3. Dawson J, et al. Vagus nerve stimulation paired with rehabilitation for upper limb motor function after ischemic stroke (VNS-REHAB): a randomized, blinded, pivotal, device trial. Lancet. 2021;397(10284):1545-1553.