hNPC01 Fast Track Designation by FDA

As the project partner, BLA congratulates Hopstem Therapeutics on receiving FDA Fast Track Designation for hNPC01 injection– the global first-in-class allogeneic human forebrain neural progenitor cell therapy

Gaithersburg, MD (December 3, 2025) – BLA Regulatory LLC announced that, as a partner of Hopstem Therapeutics, it has successfully supported hNPC01 injection– the global first-in-class allogeneic human forebrain neural progenitor cell therapy – in obtaining the Fast Track Designation (FTD) granted by the U.S. Food and Drug Administration (FDA) for the treatment of chronic motor dysfunction caused by ischemic stroke.

About hNPC01

The Phase 1 clinical trial of hNPC01 for the treatment of sequelae of ischemic stroke has demonstrated its breakthrough advantages.

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

1. 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.
2. 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].
3. 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.

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.”

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.

For more information on regulatory pathways such as Fast Track and Breakthrough Therapy, please refer to our reports:

• Fast Track vs. Breakthrough Therapy: Clinical Strategy Differences Explained
• Accelerated Approval Pathway 2025 Q4

About HopStem

Hopstem Biotechnology Inc. (abbreviated as Hopstem) was founded in January 2017. It is a globally innovative biotechnology company dedicated to the development of iPSC-derived off-the-shelf cell therapy products, boasting a total of 10,000 square meters of facilities in Qiantang District, Hangzhou, including a cell drug R&D center, GMP cell production workshop, quality control center, and office space. Hopstem possesses leading global patents and innovative technological advantages in iPSC reprogramming, pluripotent stem cell neural differentiation, and various cell engineering technologies. It has established an iPSC cell product CMC platform, a fully suspension-based automated production process, and a variety of innovative analytical methods.

The company has developed GMP-compliant iPSC cell lines available for commercial licensing both domestically and internationally, along with a pipeline of investigational products (including various neural cells, ophthalmic cells, etc.). Among them, its lead product hNPC01, a human forebrain neural progenitor cell injection, is undergoing Phase I registered clinical trials in China and has obtained FDA clinical trial clearance. It is currently the fastest-developing iPSC-derived cell product of its kind globally, targeting major unmet clinical needs such as stroke, craniocerebral injury sequelae, cerebral palsy, and epilepsy.

Since its establishment, Hopstem has completed multiple rounds of financing totaling hundreds of millions of RMB, led and continuously supported by renowned investment institutions including Qiantang Venture Capital, DT Capital Partners, Longmen Capital, BiVenture, Hillhouse Capital, and Lilly Asia Ventures. In late 2022, Qiantang Venture Capital further injected nearly 100 million RMB in a Series B+ round.

About BLA Regulatory, LLC

BLA Regulatory is a Maryland-based consultancy, conveniently located near the FDA headquarters, offering U.S. agent services for a wide range of FDA-regulated products. Our team provides expert guidance through the regulatory, application, and registration processes, helping clients navigate FDA requirements with confidence. With extensive experience across therapeutic areas—including oncology, respiratory, autoimmune, inflammatory, metabolic, and cardiovascular diseases—BLA Regulatory ensures compliance and strategic support at every stage of drug development. Visit our website for more information and assistance.

For further information contact:

info@bla-regulatory.com

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.

Reference

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 ischaemic stroke (VNS-REHAB): a randomised, blinded, pivotal, device trial. Lancet. 2021;397(10284):1545-1553.