Centessa Pharmaceuticals Announces Orphan Drug Designation Granted to SerpinPC for the Treatment of Hemophilia B
September 14 2022 - 8:00AM
Centessa Pharmaceuticals plc (Nasdaq: CNTA), a clinical-stage
pharmaceutical company with a Research & Development
(“R&D”) innovation engine that aims to discover, develop, and
ultimately deliver impactful medicines to patients, today
announced that the U.S. Food and Drug Administration (FDA) has
granted Orphan Drug Designation to SerpinPC, a novel inhibitor of
activated protein C (“APC”), for the treatment of hemophilia B.
Centessa plans to begin registrational studies of SerpinPC in the
fourth quarter of 2022.
SerpinPC is a biological drug candidate, based on the serpin
family of proteins that is designed to allow more thrombin to be
generated by inhibiting APC, thus rebalancing coagulation in
hemophilia patients. In September 2021, Centessa announced positive
topline data from its proof-of-concept Phase 2a study of SerpinPC
in severe hemophilia A and B patients not on prophylaxis. In the
highest dose cohort, SerpinPC demonstrated an 88% reduction in
median Annualized Bleeding Rate (ABR) for all bleeds and a 94%
reduction in median ABR for spontaneous joint bleeds. SerpinPC was
observed to be well-tolerated with no thrombosis and no instances
of sustained D-dimer elevations.
“We believe SerpinPC has the potential to offer patients with
hemophilia B a convenient subcutaneous option that is designed to
prevent and reduce bleeds without the risk of thrombosis,” said
Saurabh Saha, MD, PhD, Chief Executive Officer of Centessa. “This
designation from the FDA is an important milestone in the
development of SerpinPC and underscores the need for new,
innovative treatment options for patients with hemophilia B. We
look forward to initiating registrational studies for SerpinPC
later this year, as well as reporting the two-year follow-up data
from the SerpinPC Phase 2a open label extension study in the fourth
quarter.”
Orphan Drug Designation is granted by the FDA to drugs or
biologics intended to treat a rare disease or condition, defined as
one that affects fewer than 200,000 people in the U.S. Orphan Drug
Designation provides certain financial incentives to support
clinical development, and the potential for up to seven years of
marketing exclusivity for the product for the designated orphan
indication in the U.S. if the product is ultimately approved for
its designated indication.
About Centessa
Pharmaceuticals
Centessa Pharmaceuticals plc is a clinical-stage pharmaceutical
company with an R&D innovation engine that aims to discover,
develop, and ultimately deliver impactful medicines to patients.
Our programs span discovery-stage to late-stage development and
cover a range of high-value indications in rare diseases and
immuno-oncology. We are led by a management team with extensive
R&D experience, providing direct guidance to our program teams
to rapidly advance our candidates from research through all stages
of development. For more information, visit www.centessa.com,
which does not form part of this release.
Forward Looking StatementsThis press release
contains forward-looking statements. These statements may be
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be forward-looking statements, including statements related to the
Company’s ability to deliver impactful medicines to patients; the
ability of our management team and board to drive execution of the
Company’s portfolio of programs; our asset-centric business model
and the intended advantages and benefits thereof; research and
clinical development plans and the timing thereof; the scope,
progress, results and costs of developing our product candidates or
any other future product candidates; the development and
therapeutic potential of our product candidates, including
SerpinPC; strategy; regulatory matters, including the timing and
likelihood of initiating clinical trials, reporting clinical trial
results, submitting an IND and the success of obtaining
authorizations to initiate or continue clinical trials or market
any products; and the market size and opportunity for our product
candidates. 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 non-clinical 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 risks related to the ongoing COVID-19
pandemic including the effects of the Delta, Omicron and any other
variants. 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
Relationsinvestors@centessa.com
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