- FDA clears path for Phase 3 testing following positive Type B meeting
- Regulatory alignment confirms preclinical data sufficient for pivotal initiation
- Phase 3 to include two 28-day trials and a 12-month safety extension
- CPL'36 demonstrated strong efficacy and safety in prior Phase 2 study
- Program expansion planned into additional CNS indications, including Parkinson's disease
WARSAW, Poland, Oct. 08, 2025 (GLOBE NEWSWIRE) -- Celon Pharma S.A. (WSE: CLN) announced today it has secured a key regulatory milestone for its lead neuroscience program. The U.S. Food and Drug Administration (FDA) provided positive feedback clearing the path for Phase 3 testing of CPL'36, the company's novel second-generation PDE10A inhibitor for schizophrenia.
The FDA confirmed that Celon Pharma's existing preclinical data are sufficient to begin pivotal studies and endorsed the proposed Phase 3 program, which includes two 28-day randomized, double-blind trials in patients with acute schizophrenia episodes and a 12-month open-label extension to establish long-term safety.
"This validates both the strength of our scientific foundation and the robustness of our clinical program," said Maciej Wieczorek, Ph.D., Chief Executive Officer of Celon Pharma. "With FDA alignment in place, we are advancing CPL'36 into Phase 3 development and expanding its potential across CNS disorders, including Levodopa-Induced Dyskinesia in Parkinson's disease."
The FDA also agreed that "treatment of schizophrenia" is the appropriate indication for CPL'36, confirming the compound's potential for broad labeling at registration. The Agency supported Celon Pharma's plan to conduct required supportive studies-including drug-drug interaction (DDI), QTc, and mass balance-concurrently with the pivotal trials, provided key safety measures are in place. This parallel development plan is expected to streamline timelines and maintain strategic flexibility.
Strong Efficacy in Phase 2 Sets the Stage
CPL'36 previously demonstrated robust and clinically meaningful efficacy in a 189-patient Phase 2 trial, with both the 20 mg and 40 mg doses producing statistically significant improvements versus placebo across positive and total PANSS scores. The 40 mg dose also showed early signs of cognitive benefit.
At Week 4, the positive PANSS subscale improved by 3.7 points for the 20 mg dose (p<0.001; Cohen's d=0.73) and 6.3 points for the 40 mg dose (p<0.001; Cohen's d=1.38). For total PANSS score-a key secondary endpoint-reductions were 9.7 and 16.4 points, respectively (both p<0.001). These results underscore CPL'36's differentiated mechanism of action, which modulates dopaminergic and glutamatergic neurotransmission to address the broad symptom spectrum of schizophrenia.
Next Steps
Celon Pharma plans to finalize its Phase 3 protocols incorporating FDA feedback and expects to initiate pivotal studies in 2026. The company is also advancing regulatory preparations and exploring global partnerships to support development and potential commercialization.
About CPL'36
CPL'36 is a selective phosphodiesterase 10A (PDE10A) inhibitor with an improved fast-off pharmacodynamic profile designed to modulate dopaminergic and glutamatergic neurotransmission. It has demonstrated potential to address positive, negative, and cognitive symptoms of schizophrenia and is also being evaluated for Levodopa-Induced Dyskinesia (LiD) in Parkinson's disease.
About Celon Pharma S.A.
Celon Pharma S.A. is an integrated biopharmaceutical company dedicated to the discovery, development, and commercialization of innovative therapies in neuroscience, oncology, and metabolic diseases. The Company combines advanced research capabilities with in-house GMP manufacturing to accelerate the development of high-value therapeutic candidates.
For more information, visit www.celonpharma.com.
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