Strong Proof-of-Concept Data from Phase 2 Trial of PDE10A Inhibitor (CPL’36), a Novel Oral Antipsychotic

-Statistically significant benefit demonstrated in primary endpoint of positive subscale of the Positive and Negative Syndrome Scale for Schizophrenia (PANSS) at Week 4-

-Dose-response was demonstrated, consistent with higher efficacy in the 40 mg dose-

-Favorable tolerability profile demonstrated relative to standard-of-care atypical antipsychotics-

-Investor call to be held 10^th of July at 3 PM CEST with details provided below:

• MS Teams: https://teams.microsoft.com/l/meetup-join/19%3ameeting_NTA4ZDg0NzEtYjU2ZS00N2NjLWI4MjAtYzAwMzQxMDBhNDlj%40thread.v2/0?context=%7b%22Tid%22%3a%22dc816c94-0313-4f34-8ed1-1bff167d7003%22%2c%22Oid%22%3a%22640e4061-4b30-4b4f-ab73-ea6aee635b80%22%7d
• Telephone: +48 22 161 67 63, 522919119# Poland, Warsaw. 522 919 119#

WARSAW, Poland, July 09, 2024 (GLOBE NEWSWIRE) — Celon Pharma S.A. (CLN.WA) announces today robust and positive Phase 2 clinical trial results for its PDE10A inhibitor (CPL’36), a novel, oral, once-daily antipsychotic. Statistically significant and clinically meaningful improvements in the primary endpoint of positive subscale of the PANSS were seen in both tested doses of CPL’36, with a dose-response effect. Management plans to discuss these highly encouraging results with regulatory agencies to advance CPL’36 towards registrational trials and global marketing approvals. Additionally, CPL’36 is being investigated as a potential treatment for levodopa-induced dyskinesia in Parkinson’s disease, with Phase 2 results expected to be reported in 4Q 2024.

“The results from this Phase 2 trial suggest a beneficial effect of CPL’36 across multiple aspects of schizophrenia pathophysiology. Clinical efficacy was deemed strong, and moreover, the drug tolerability was quite favorable,” confirmed Maciej Wieczorek, Ph.D., CEO of Celon Pharma S.A. “We believe that these results could herald a breakthrough in the treatment of this disease that impacts over 24 million patients world-wide, by identifying an innovative drug class with a new mechanism of action that may be used to replace or complement the current standard of care. These results represent a lengthy and satisfying journey for Celon as we have been closely analyzing the competitive environment in schizophrenia and the clinical trial designs of major international companies working on PDE10A inhibitors. We are therefore proud to have developed a drug characterized by unprecedented efficacy, with global potential to help underserved patients,” he concluded.

Trial description

The CPL’36 Phase 2 study was an international, multicenter, randomized, placebo-controlled clinical trial conducted on a group of 189 adult patients hospitalized due to acute schizophrenia.

CPL’36 was administered for 4 weeks in two doses of 20 and 40 mg once daily and was placebo-controlled at a ratio of 1:1:1. Patient baseline severity was moderate-severe to severe, as characterized by a PANSS total score of approximately 106. Patients were screened for up to 10 days and then randomized and treated over a four-week period, at which time the primary endpoint assessment was conducted at Day 28.

At Week 4 of treatment, the reduction in positive PANSS subscale score which was the primary endpoint in the trial was 3.7 units from baseline in the 20 mg dose (LS mean difference from placebo, p<0.001, Cohen’s d: 0.73), and 6.3 units in the 40 mg dose (LS Mean difference from placebo, p<0.001, Cohen’s d: 1.38).

For total PANSS score at week 4 of treatment (a key secondary endpoint), the 20 mg dose of CPL’36 demonstrated a 9.7 unit reduction from baseline compared to placebo (LS mean difference from placebo, p<0.001, Cohen’s d: 0.77), and 16.4 units in the 40 mg dose (LS mean difference from placebo, p<0.001, Cohen’s d: 1.47).

Other secondary endpoints in the trial included the effects of CPL’36 on overall clinical improvement cognitive performance and functioning such as Clinical Global Impression Scale Improvement (CGI-I), Brief Assessment of Cognition in Schizophrenia (BACS), and number of participants who withdraw due to adverse events (AEs). Results across all of these endpoints were also positive.

Drug tolerability was favorable with most treatment emergent adverse events characterized as mild. Exacerbations of schizophrenia represented the most common severe adverse events that were potentially related to the drug (1.5% in the placebo group, 1.8% in the 20 mg group and 3.1% in the 40 mg group). Treatment discontinuation due to adverse events likely related to the drug occurred in 3.1% patients in the placebo group, 0% patients in the 20 mg group and 7.7% patients in the 40 mg group.

Investor Call Information:

Małgorzata Siewierska
inwestorzy@celonpharma.com
+48519066531

ABOUT CELON PHARMA

Celon Pharma is an integrated biopharmaceutical company which designs, develops, manufactures and distributes pharmaceutical products. It was founded in 2002 by Maciej Wieczorek, PhD. The Company currently has approx. 500 employees. It employs approx. 160 scientists responsible for research and development, half of whom either have a PhD or are in the process of acquiring their PhD. The Company’s pipeline includes more than a dozen projects for the development of innovative drugs with therapeutic groups such as oncology, neuro-psychiatry, autoimmunity and metabolism. The Company has its own research and development laboratories, which allow it to develop its own pharmaceutical technologies by using extensive laboratory equipment resources, as well as the experience and expertise of its staff. It also has a modern manufacturing facility where dry pharmaceutical forms are manufactured. Over the last few years, the Company has introduced products into the market in the following therapeutic areas: oncology, central nervous system diseases, cardiology, respiratory diseases. The Company has been developing the technology for manufacturing inhalation drugs and several projects of innovative drugs since 2007. It has been listed on the Warsaw Stock Exchange since 2016. www.celonpharma.com

Disclaimer
This press release (“Press release”) has been prepared by Celon Pharma S.A. (the “Company”). By reading the Press release, you agree to be bound by the following limitations.

This Press release is strictly confidential to the recipient. Neither this Press release or any part hereof nor the information contained herein may be reproduced or redistributed, passed on, or the contents otherwise divulged, directly or indirectly, to any other person or published, in whole or in part.

If you gain access to this Press release by mistake, or you are not an addressee of this Press release or a person authorized to use this Press release, please bear in mind the confidential nature of this Press release and immediately contact the Company in order to return it to the Company.

The Press release does not constitute an offer to sell or subscribe for or a solicitation of an offer to purchase or subscribe for securities. This Press release is provided for informational purposes only. This Press release does not constitute or form part of and should not be construed as an offer, solicitation or invitation to sell or issue, or an offer, solicitation or invitation to, subscribe for, underwrite, buy or otherwise acquire, securities of the Company or any of its subsidiaries in any jurisdiction, or an inducement/recommendation to enter into investment activity in any jurisdiction. Neither this Press release nor any part hereof, nor the fact of its distribution or issuance, shall form the basis of, or be relied on in connection with, any contract, commitment or investment decision whatsoever.

The information contained herein is only preliminary and indicative and does not purport to contain the information that would be required to evaluate the Company, its financial position and/or any investment decision. This Press release is not intended to provide, and should not be relied upon for, accounting, legal or tax advice nor does it constitute an investment recommendation. This Press release is given in conjunction with an oral press release and should not be taken out of context.

No information included in this Press release may be considered as investment advice or investment recommendation. The information contained in the Press release has not been independently verified. No repress release, warranty or undertaking, expressed or implied, is made as to, and no reliance should be placed on, the fairness, accuracy, completeness or correctness of the information or the opinions contained herein.

Matters discussed in this Press release may constitute forward-looking statements. Forward-looking statements constitute statements that are other than statements of historical fact. Statements which include the words “expects”, “intends”, “plans”, “believes”, “projects”, “anticipates”, “will”, “targets”, “aims”, “may”, “would”, “could”, “continue” and similar statements of a future or forward-looking nature identify such forward-looking statements. Forward-looking statements include in particular statements regarding the financial performance, business strategy, plans and objectives of the Company for future operations (including growth potential). All forward-looking statements included in this Press release address matters that involve known and unknown risks, uncertainties and other factors which could cause the Company’s actual results, performance or achievements to differ materially from those indicated in such forward-looking statements and from past results, performance or achievements of the Company. Such forward-looking statements are based upon various assumptions and estimates regarding future events, including numerous assumptions regarding the Company’s present and future business strategies and future operating environment. Although the Company believes that these estimates and assumptions are reasonable, they may prove to be incorrect.

The information, opinions and forward-looking statements contained in this Press release speak only as at the date of this Press release and are subject to change without notice. The Company, its directors, agents, employees and advisors do not intend to, and expressly disclaim any duty, undertaking or obligation to, make or disseminate any supplement, amendment, update or revision to any of the information, opinions or forward-looking statements contained in this Press release to reflect any change in events, conditions or circumstances. To the extent permitted under the applicable provisions of law, neither the Company nor any of their affiliates, advisers or representatives shall have any liability whatsoever (in negligence or otherwise) for any loss however arising from any use of this Press release or its contents or otherwise arising in connection with this Press release.

This Press release is not for distribution or use by any person or entity in any jurisdiction where such distribution or use would be contrary to local law or regulation or which would subject the Company or any of its affiliates to authorization, notification, licensing or other registration requirements under applicable laws. Any failure to comply with this restriction may constitute a violation of United States securities laws. Persons into whose possession this Press release comes should observe all such restrictions.

Obtain more information by contacting:
Małgorzata Siewierska
media@celonpharma.com