Celon Pharma's PDE10A Inhibitor Shows Promise in Parkinson's Disease Trial

Celon Pharma, a Polish biotech company, has reported positive results from a phase 2 clinical trial of its PDE10A inhibitor, CPL'36, in patients with Parkinson's disease experiencing levodopa-induced dyskinesia (LID). The study, which involved 105 adults, demonstrated significant reductions in involuntary movements, a common side effect of long-term levodopa treatment.

Efficacy and Safety Profile

The trial evaluated two daily doses of CPL'36 (20 mg and 30 mg) against placebo. By the fourth week of treatment, patients receiving the 20 mg dose showed an improvement of 12.30 units on the Unified Dyskinesia Rating Scale, while those on the 30 mg dose improved by 13.58 units, meeting the study's primary endpoint.

Safety data revealed that no patients in the 20 mg cohort experienced severe adverse events, compared to 5.7% in the 40 mg group and 8.8% in the placebo group. However, treatment discontinuation rates due to adverse events were 11.1%, 8.6%, and 2.9% for the 20 mg, 40 mg, and placebo groups, respectively.

Mechanism of Action and Market Potential

CPL'36 targets PDE10A, an enzyme expressed in brain regions associated with various cognitive disorders. Joanna Sierzputowska-Prarat, Celon's clinical neuropsychiatry lead, highlighted the drug's "unique pharmacodynamic profile, characterized by rapid enzyme dissociation," which she believes distinguishes it from other PDE10A inhibitors and contributes to its positive clinical outcomes.

Celon Pharma's CEO, Maciej Wieczorek, Ph.D., expressed optimism about CPL'36's potential to "significantly contribute to the expansion and advancement of the global market for Parkinson's disease pharmacotherapy." He emphasized the drug's promise in addressing the unmet needs of LID patients who are currently underserved by existing treatments.

Competitive Landscape

Celon is not alone in exploring PDE10A inhibition for neurological disorders. Swiss biotech Noema Pharma recently reported positive results for its PDE10A inhibitor, gemlapodect, in a small phase 2 trial for Tourette syndrome. Additionally, EuMentis Therapeutics aims to advance its own PDE10A inhibitor to phase 2 trials in early 2025, despite previous setbacks in meeting program targets.

The success of CPL'36 in this Parkinson's disease trial follows a previous "statistically significant" benefit demonstrated in a separate phase 2 trial for schizophrenia last year, further validating the potential of PDE10A inhibition across multiple neurological indications.