FDA Grants Orphan Drug Designation to Cellenkos' CK0803 Tregs for Treatment of Amyotrophic Lateral Sclerosis

• Orphan Drug Designation granted to CK0803 for Amyotrophic Lateral Sclerosis
• CK0803 is CNS Homing Tregs expressing CXCR3^hi, CXCR7⁺, LFA1⁺
• CK0803 is planned for Randomized Placebo Control Trial in 2026

HOUSTON, Oct. 20, 2025 /PRNewswire/ -- Cellenkos^® Inc., a clinical-stage biotechnology company focused on developing allogeneic, off-the-shelf, T regulatory (Treg) cell therapies for autoimmune diseases and inflammatory disorders especially in the areas of high unmet needs, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its novel cell therapy product, CK0803, which consists of neurotropic CNS Homing Tregs expressing CXCR3^hi, CXCR7⁺, LFA1⁺. CK0803 Tregs preferentially migrate to the sites of neuroinflammation, neutralize activated microglia and restore homeostasis.

This designation supports the development of medicines for rare disorders that affect fewer than 200,000 people in the U.S, and qualifies a company for incentives, including tax credits, exemptions from certain FDA fees for clinical trials, and the potential for seven years of market exclusivity following drug approval. Amyotrophic Lateral Sclerosis (ALS) has an incidence of 1.0-2.6 per million in the U.S and a projected prevalence of 35,000 patients.

"Receiving Orphan Drug Designation for CK0803 in ALS underscores the importance of bringing novel, transformative treatment options to patients suffering from this rare disease", said Dr. Simrit Parmar, MD, Founder of Cellenkos. Dr. Parmar is also faculty at Texas A&M School of Engineering Medicine. "We aim to deliver a transformative therapeutic that can slow disease progression in ALS, and decrease levels of plasma neurofilament, a biomarker of active neurodegeneration and neuroinflammation."

The results of a Proof-of-Concept compassionate use clinical study with 6 ALS patients was published earlier this year in NEJM Evidence, demonstrating that Cellenkos' Tregs can lead to durable slowing of the ALS disease progression.

Preliminary data from the Phase 1 trial of CK0803 (NCT05695521), shows disease slowing for both spinal and bulbar onset ALS, especially in patients that started treatment early on in their disease.

Key Study Findings

• Durable slowing of ALSFRS-R score: Early-stage Spinal-Onset patients progressed minimally during the treatment period for over 196 days and over 50 days after treatment stopped. Bulbar-Onset patients saw a benefit late in treatment.
• Safety Profile: CK0803 was well tolerated where no dose-limiting toxicities were observed. CK0803 does not require any additional conditioning regimen and/ or IL-2 supplementation. Multiple infusions of cryopreserved CK803 Tregs were administered intravenously in the ambulatory setting, where patients were able to go home the same day.
• Decrease in Plasma Neurofilament: Observation showed a consistent decrease in subset of patients correlating to slowing of ALSFRS-R decline

Broader Potential Across Neuroinflammatory Diseases
Beyond ALS, CK0803 has potential to have activity in other neuroinflammatory / neurodegenerative diseases including Multiple Sclerosis, Frontotemporal Dementia, Alzheimer's Dementia, Parkinson's Disease, in addition to other rare neurodegenerative conditions which have a driving neuroinflammatory component.

About Amyotrophic Lateral Sclerosis
ALS is a progressive neurodegenerative disease that affects nerve cells responsible for voluntary muscle movement. As both upper and lower motor neurons deteriorate, leading to muscle weakness, paralysis, and eventual respiratory failure. Most individuals with ALS succumb to the disease within 3 to 5 years of diagnosis. Currently, there is no cure.

About Cellenkos, Inc.
Cellenkos is a clinical-stage biotechnology company based in Houston, Texas, focused on developing off-the-shelf Treg cell therapies for rare inflammatory diseases and autoimmune disorders. Cellenkos' Treg therapies, derived from umbilical cord blood, are designed to provide powerful anti-inflammatory effects and long-lasting immune modulation without the need for donor matching. The company is committed to advancing the development of these promising therapies to improve the lives of patients with rare and underserved conditions.

For more information, please visit www.cellenkosinc.com.

Contact:
For Cellenkos: [email protected] 

SOURCE Cellenkos, Inc.