Cellenkos’ Treg Cell Therapy Granted FDA Orphan Drug Status for ALS

HOUSTON, TX — October 20, 2025 — Cellenkos Inc., a clinical-stage biotechnology company, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its novel allogeneic T regulatory (Treg) cell therapy product, CK0803, for the treatment of Amyotrophic Lateral Sclerosis (ALS).

The ODD supports the development of therapies for rare disorders, such as ALS, which affects approximately 35,000 patients in the U.S. It qualifies Cellenkos for incentives, including tax credits and potential market exclusivity for seven years upon approval.

Targeted Approach to Neuroinflammation

CK0803 consists of proprietary neurotropic CNS Homing Tregs engineered to express markers like CXCR3+ hi, CXCR7+ hi, and LFA1+. This design allows the cells to preferentially migrate to sites of neuroinflammation in the central nervous system (CNS), where they work to neutralize activated microglia and restore cellular balance.

“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. “We aim to deliver a therapeutic that can slow disease progression in ALS, and decrease levels of plasma neurofilament, a biomarker of active neurodegeneration and neuroinflammation.”

Preliminary Data Shows Durable Disease Slowing

The ODD application was supported by evidence from both a compassionate use study published in NEJM Evidence and preliminary data from the ongoing Phase 1 trial of CK0803 (NCT05695521).

Key findings from the studies indicate:

• Disease Slowing: Data showed durable slowing of the decline in ALSFRS-R score (a measure of disease progression). Early-stage spinal-onset patients progressed minimally during treatment (over 196 days) and for over 50 days post-treatment.
• Biomarker Response: A consistent decrease in plasma neurofilament—a key measure of neurodegeneration—was observed in a subset of patients, correlating to the slower decline in clinical scores.
• Safety and Dosing: CK0803 was well tolerated, with no dose-limiting toxicities. The off-the-shelf, cryopreserved Tregs were administered intravenously in the ambulatory setting, requiring no additional conditioning regimen or IL-2 supplementation.

Cellenkos is positioning CK0803 for potential activity across a broader spectrum of neuroinflammatory and neurodegenerative diseases, including Multiple Sclerosis, Alzheimer’s Dementia, and Parkinson’s Disease. ALS remains a progressive, fatal condition with no current cure, typically leading to respiratory failure within 3 to 5 years of diagnosis.