FDA Grants Fast Track Designation to Xellsmart Off-The-Shelf Cell Therapy for Parkinson's Disease

XellSmart's proprietary off-the-shelf iPSC-derived cell therapy XS411 receives Fast Track Designation from the US FDA, marking a key milestone for its global clinical development across China and the United States.

SHANGHAI and SUZHOU, China, Aug. 4, 2026 /PRNewswire/ -- On July 28, 2026, XellSmart announced that XS411, the company's independently developed allogeneic, off-the-shelf injectable iPSC-derived cell therapy candidate indicated for Parkinson's disease—the world's second most prevalent neurodegenerative disorder — has been officially awarded Fast Track Designation (FTD) by the US Food and Drug Administration (FDA). This regulatory designation underscores the US health authority's recognition of XS411's clinical value and potential to address the profound unmet clinical needs in Parkinson's disease treatment, representing a pivotal milestone in its worldwide clinical development and future market access.

FTD Accelerates Sino-U.S. Clinical Development

FTD is an expedited review pathway for investigational medicines intended to treat serious diseases with unmet clinical needs. By securing this designation, XS411 qualifies for a full set of preferential regulatory incentives throughout its US development and commercialization journey.

First, the team can conduct regular, intensive discussions with FDA reviewers throughout drug development to refine clinical trial designs and adapt R&D strategies. Second, XS411 qualifies for rolling BLA submission, which can greatly cut down regulatory review timelines. Third, XS411 can receive FDA Priority Review upon fulfilling specified clinical endpoints to further expedite its US marketing approval. Collectively, these regulatory advantages will accelerate XS411's clinical progression, regulatory review and commercial launch in the US market.

This FTD achievement builds on XS411's robust cross-border clinical progress since 2025 — the product secured FDA IND approval with special exemptions in January 2025 and obtained China NMPA registrational clinical trial clearance in April 2025. Now the China clinical program has advanced to Phase II and completed enrollment, driven by positive Phase I results. The newly conferred FTD is a further vote of confidence to validate XS411's therapeutic potential to resolve unmet medical needs for global Parkinson's patients.

Clinical Progress: XS411 Completed Full Enrollment in China Multicenter Phase II Trial

In July 2026, XellSmart announced the full completion of patient enrollment in China's first "National Center for Neurological Disorders"-led multicenter Phase II registrational trial of XS411 for the treatment of Parkinson's disease. The trial employs the globally acknowledged PROBE design (Prospective, Randomized, Open-label, Blinded Endpoint Assessment), which will yield stronger clinical evidence than traditional single-arm open-label studies. The trial design not only meets the demanding clinical delivery requirements of cell therapy, but could also reduce study bias and improve overall trial rigor with randomization and blinded endpoint evaluation. This optimized design is more likely to produce solid, credible efficacy data to support the program's subsequent Phase III registrational development and approval.

Phase II advancement is built on XS411's encouraging Phase I results. Upon NMPA clearance in 2025, this registrational clinical trial was initiated in the same year, led by Beijing Tiantan Hospital, Capital Medical University — China's leading National Center for Neurological Disorders. Cumulative long-term follow-up data have demonstrated favorable safety and tolerability profiles for the cell therapy product. After transplantation, participants achieved statistically significant improvements in OFF-state MDS-UPDRS Part III motor scores, prolonged ON-time duration, and enhanced quality-of-life metrics compared with baseline. Molecular imaging confirmed transplanted cells successfully engrafted, survived, differentiated within the brain parenchyma, and acquired functional capacity to synthesize and secrete dopamine neurotransmitters.

Building upon positive Phase I outcomes, China multicenter Phase II registrational trial was rapidly rolled out at Beijing Tiantan Hospital, also led by Principal Investigator Professor Feng Tao, a leading Chinese Parkinson's disease specialist, in collaboration with China's top-tier medical institutions: Peking Union Medical College Hospital, the Second Affiliated Hospital of Soochow University, Jiangsu Provincial People's Hospital, and West China Hospital, Sichuan University.

To date, XellSmart has secured nine INDs for registrational clinical trials across China and the United States. The company holds a global leading position in developing iPSC-derived cell therapies for neurological conditions including Parkinson's disease and amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig's disease). With steady advancement of Sino-US registrational programs, this homegrown Chinese iPSC-derived cell therapy, with the potential to both mitigate symptoms and modify underlying disease progression, is poised to deliver an innovative therapy for 10 million+ Parkinson's patients globally.

About XellSmart

XellSmart is a clinical-stage, regenerative medicine company focusing on treating CNS diseases with clinical-grade, allogeneic, off-the-shelf, iPSC-derived cell therapies.

Founded by Dr. Xiang Li following his return to China, XellSmart has curated a world-leading full-time expert team possessing core capabilities in iPSC-derived cell therapy innovation and translational industrialization. The team, recruited from renowned global academic institutions and top international cell therapy enterprises, oversees the full spectrum of work covering R&D, process development and commercial manufacturing of iPSC-derived cell therapeutics.

Backed by this seasoned interdisciplinary talent pool, the company has built and operates a complete in-house infrastructure, consisting of a 5,000 m² R&D center, a B+A grade GMP production plant and a dedicated QC laboratory. It has completed CMC development for several clinical-grade iPSC cell candidates, and established end-to-end clinical cell production workflows alongside strict quality management systems. Leveraging this mature platform, XellSmart has manufactured GMP-compliant iPSC-derived neural progenitor cell products for registrational trials and successfully administered these investigational therapeutics to patients across multiple clinical study sites.

With robust manufacturing and clinical execution capabilities in place, XellSmart has further forged a portfolio of proprietary, pioneering technological platforms and established a comprehensive global intellectual property ecosystem driven by in-house innovation. The company holds nine INDs cleared by NMPA and FDA for registrational clinical trials. Its pipeline includes first-in-China and world-first clinical-grade products targeting severe, life-threatening central nervous system (CNS) diseases, such as Parkinson's disease, multiple system atrophy-Parkinsonian type, spinal cord injury and ALS:

• Primary Parkinson's Disease: now in China Phase II & US Phase I registrational clinical trials. The China trial, led by Beijing Tiantan Hospital, with Peking Union Medical College Hospital as a collaborative site, is supported by China's national key research initiatives.
• Early-onset Parkinson's Disease: now in China's first, randomized, double-blind, controlled, Phase II registrational trial, led by Huashan Hospital, Fudan University, partnered with the Second Affiliated Hospital of Soochow University, and backed by major grants from China's National Brain Science Project.
• Multiple System Atrophy-Parkinsonian Type: now in China Phase I/II + US Phase I trial registrational clinical trials. The China trial is led by Beijing Tiantan Hospital.
• Spinal Cord Injury: a First-in-Class IND, now in a Phase I registrational clinical trial in China and the US. The China trial is led by the Third Affiliated Hospital of Sun Yat-sen University.
• Amyotrophic Lateral Sclerosis (ALS / Lou Gehrig's Disease): a First-in-Class IND & the first China-developed iPSC-derived cell therapy granted the US FDA Orphan Drug Designation in 2023, now in combined Phase I/II trial in China + Phase I trial in the US. The China trial is led by Peking University Third Hospital.

XellSmart's rapid clinical advancement has been fueled by multiple rounds of market-driven financing. It has secured lead investments and follow-on capital injections from top-tier venture capital institutions and industrial strategic investors, including FreeS Fund, Qiming Venture Partners, Lilly Asia Ventures (LAV), Sequoia China, and 3SBIO, fully endorsing XellSmart's cutting-edge technology and long-term development potential.

About Parkinson's Disease and XS411

Parkinson's disease (PD) ranks as the second-leading neurodegenerative disease globally. Epidemiological statistics indicate more than 10 million patients worldwide, including approximately 4 million cases across China. PD is characterized by progressive degeneration and loss of dopaminergic neurons within the substantia nigra of the midbrain, manifesting clinically as resting tremor, rigidity, bradykinesia, postural and gait disturbances, which drastically erode patients' quality of life.

To date, no approved pharmacotherapy can halt or reverse the progressive neurodegenerative process. All current treatments merely alleviate symptomatic manifestations, leaving an enormous global unmet clinical demand.

XS411 is engineered to target this root pathological mechanism. The therapeutic modality works via intracerebral transplantation of healthy induced pluripotent stem cell (iPSC)-derived dopaminergic neural progenitor cells, which replace degenerated endogenous dopaminergic neurons and restore impaired neurological function. Named after World Parkinson's Day (April 11), XS411 embodies XellSmart's patient-centric R&D philosophy.

To sum up, combining its proprietary functional cell-generation mechanism, FDA Fast Track Designation and fully enrolled Chinese Phase II study, XellSmart stands ready to pioneer transformative therapies for neurodegenerative diseases and benefit global patients at the earliest opportunity.