FDA Clears Centessa Pharmaceuticals for Narcolepsy Trial

By Bobby Samwell • Monday, April 22 10:19 • Permanent Link

Centessa Pharmaceuticals plc (NASDAQ: CNTA) has just received clearance from the U.S. Food and Drug Administration (FDA) to initiate a phase 1 first-in-human clinical trial of ORX750 for the treatment of narcolepsy. This marks a significant development for the clinical-stage pharmaceutical company as it aims to discover and develop transformational medicines for patients.

ORX750 is an investigational, orally administered, highly potent, and selective orexin receptor 2 (OX2R) agonist designed to directly target the underlying pathophysiology of orexin neuron loss in narcolepsy type 1 (NT1), with potential applicability to narcolepsy type 2 (NT2), idiopathic hypersomnia (IH), and other sleep-wake disorders with normal orexin levels.

The phase 1 study will evaluate the safety, tolerability, and pharmacokinetics of single-ascending doses (SAD) and multiple-ascending doses (MAD) of ORX750 in healthy adult subjects. It will also include a cross-over pharmacodynamic (PD) assessment utilizing the maintenance of wakefulness test (MWT) and Karolinska Sleepiness Scale (KSS) in acutely sleep-deprived healthy adult subjects, with proof-of-concept data anticipated in the second half of 2024.

CEO Saurabh Saha, MD, PhD, expressed excitement over the milestone, highlighting the company's adaptive phase 1 study aimed at generating early proof-of-concept data for ORX750. This data is expected to enable dose selection for planned studies evaluating ORX750 in patients with NT1 and in patient populations with normal orexin levels, including NT2 and IH.

ORX750 has shown potent activation of the OX2R with an in vitro EC50 of 0.11 nm and 9,800-fold selectivity over the human orexin receptor (HOX1R), making it a promising candidate for the treatment of narcolepsy and other sleep-wake disorders.

Centessa Pharmaceuticals is also involved in other advanced programs, including a hemophilia program and an immuno-oncology program focused on their LockBody® technology platform. The company operates with the conviction that each of its programs has the potential to change the current treatment paradigm and establish a new standard of care. As a result of these announcements, the company's shares have moved 1.7% on the market, and are now trading at a price of $10.74. For more information, read the company's full 8-K submission here.

The above analysis is intended for educational purposes only and was performed on the basis of publicly available data. It is not to be construed as a recommendation to buy or sell any security. Any buy, sell, or other recommendations mentioned in the article are direct quotations of consensus recommendations from the analysts covering the stock, and do not represent the opinions of Market Inference or its writers. Past performance, accounting data, and inferences about market position and corporate valuation are not reliable indicators of future price movements. Market Inference does not provide financial advice. Investors should conduct their own review and analysis of any company of interest before making an investment decision.