BOSTON and LONDON, April 22, 2024 (GLOBE NEWSWIRE) -- Centessa Pharmaceuticals
plc (http://www.centessa.com/) (Nasdaq: CNTA), a clinical-stage pharmaceutical
company that aims to discover and develop medicines that are transformational
for patients, today announced that the U.S. Food and Drug Administration (FDA)
has cleared the Investigational New Drug application (IND) to initiate a Phase
1 first-in-human, clinical trial of ORX750 for the treatment of narcolepsy.
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. In parallel to the SAD, a cross-over pharmacodynamic
(PD) assessment will be performed utilizing the Maintenance of Wakefulness Test
(MWT) and Karolinska Sleepiness Scale (KSS) in acutely sleep-deprived healthy
adult subjects which is intended to provide proof-of-concept data to enable dose
selection for NT1, NT2 and IH indications. The study has a maximum exposure
limit specified by the FDA which the Company believes significantly exceeds the
predicted efficacious doses of ORX750 in indications associated with or without
orexin loss; therefore, the Company does not expect this limit to affect any of
the planned clinical development activities for ORX750. The Company expects to
commence dosing of the Phase 1 study in healthy volunteers imminently, and
proof-of-concept data are anticipated in the second half of 2024.
The Phase 1 study design of ORX750 includes SAD combined with PoC cohorts to
assess PD effects of ORX750 by measuring sleep latency with the MWT and
subjective sleepiness with the KSS in acutely sleep-deprived healthy subjects.
"This is a significant milestone for the development of our potential best-in-
class OX2R agonist, ORX750, for the treatment of narcolepsy and other sleep-wake
disorders," said Saurabh Saha MD PhD, Chief Executive Officer of Centessa. "We
are excited to begin executing what we believe is an elegant, adaptive Phase 1
study aimed at generating early proof-of-concept data for ORX750 in acutely
sleep-deprived healthy volunteers in the second half of this year. We expect
this study 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."
About ORX750
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). ORX750 has
been shown to potently activate the OX2R with an in vitro EC50 of 0.11 nM and
9,800-fold selectivity over the human orexin receptor (hOX1R). ORX750 is
Centessa's first orexin product candidate being developed for the treatment of
narcolepsy with potential expansion into narcolepsy type 2 (NT2), idiopathic
hypersomnia (IH) and other sleep-wake disorders.
About Centessa Pharmaceuticals
Centessa Pharmaceuticals plc is a clinical-stage pharmaceutical company that
aims to discover and develop transformational medicines for patients. Our most
advanced programs include a hemophilia program, an orexin agonist program for
the treatment of narcolepsy and other sleep-wake disorders and an immuno-
oncology program focused on our LockBody® technology platform. We operate with
the conviction that each of our programs has the potential to change the current
treatment paradigm and establish a new standard of care. For more information,
visit www.centessa.com (http://centessa.com/), which does not form part of this
release.
Forward Looking Statements
This press release contains forward-looking statements. These statements may be
identified by words such as "may," "might," "will," "could," "would," "should,"
"expect," "intend," "plan," "objective," "anticipate," "believe," "estimate,"
"predict," "potential," "continue," "ongoing," "aim," "seek," and variations of
these words or similar expressions that are intended to identify forward-looking
statements. Any such statements in this press release that are not statements of
historical fact may be deemed to be forward-looking statements, including
statements related to the Company's ability to discover and develop
transformational medicines for patients; the timing of commencement of new
studies or clinical trials of ORX750 and other potential orexin agonist
candidates; research and clinical development plans and the timing thereof; the
Company's ability to differentiate ORX750 and other potential orexin agonist
candidates from other treatment options; the development and therapeutic
potential of ORX750 and other potential orexin agonist candidates; predicted
efficacious doses of ORX750; the Company's ability to successfully conduct its
clinical development of ORX750 below the maximum exposure limit set by the FDA
or, in the event the Company plans to exceed the maximum exposure limit, the
Company's ability to successfully have the maximum exposure limit removed; and
other regulatory matters, including the timing and likelihood of success of
obtaining authorizations to initiate or continue clinical trials. Any forward-
looking statements in this press release are based on our current expectations,
estimates and projections only as of the date of this release and are subject to
a number of risks and uncertainties that could cause actual results to differ
materially and adversely from those set forth in or implied by such forward-
looking statements. These risks and uncertainties include, but are not limited
to, risks related to the safety and tolerability profile of our product
candidates; our ability to protect and maintain our intellectual property
position; business (including commercial viability), regulatory, economic and
competitive risks, uncertainties, contingencies and assumptions about the
Company; risks inherent in developing product candidates and technologies;
future results from our ongoing and planned clinical trials; our ability to
obtain adequate financing, including through our financing facility with
Oberland, to fund our planned clinical trials and other expenses; trends in the
industry; the legal and regulatory framework for the industry, including the
receipt and maintenance of clearances to conduct or continue clinical testing;
future expenditures risks related to our asset-centric corporate model; the risk
that any one or more of our product candidates will not be successfully
developed and/or commercialized; the risk that the results of preclinical
studies or clinical studies will not be predictive of future results in
connection with future studies; geo-political risks such as the Russia-Ukraine
war and conflicts in the Middle East. These and other risks concerning our
programs and operations are described in additional detail in our Annual Report
on Form 10-K, Quarterly Reports on Form 10-Q, and our other reports, which are
on file with the U.S. Securities and Exchange Commission (SEC). We explicitly
disclaim any obligation to update any forward-looking statements except to the
extent required by law.
Contact:
Kristen K. Sheppard, Esq.
SVP of Investor Relations
investors@centessa.com (mailto:investors@centessa.com)
A photo accompanying this announcement is available at
https://www.globenewswire.com/NewsRoom/AttachmentNg/b64204a9-7c64-47fd-b834-
598ca0e45978
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