Forma Therapeutics Holdings, Inc. (Nasdaq: FMTX), a
clinical-stage biopharmaceutical company focused on rare
hematologic diseases and cancers, today announced the European
Commission granted Orphan Drug designation to Forma’s FT-4202 for
the treatment of sickle cell disease (SCD), based on a positive
opinion from the Committee for Orphan Medicinal Products of the
European Medicines Agency (EMA). FT-4202 was previously granted
Fast Track, Rare Pediatric Disease and Orphan Drug designations
from the U.S. Food and Drug Administration (FDA) for the treatment
of patients with SCD.
“I am pleased to see the regulatory community recognize the
urgent need to bring therapies to people living with sickle cell
disease,” said Frank Lee, president and chief executive officer of
Forma. “Without effective treatment, sickle cell can affect all
organs over time and lead to substantial suffering for those born
with this genetic disease. We embrace the potential this
designation may provide as we prepare to initiate a global
registrational Phase 2/3 trial with FT-4202 in the first quarter of
2021.”
Forma is currently enrolling patients with SCD in a randomized,
placebo-controlled, multi-center Phase 1 study to evaluate the
safety and pharmacokinetics/pharmacodynamics (PK/PD) of FT-4202.
For more information on eligibility and study sites for the open
Phase 1 study, please visit clinicaltrials.gov/NCT03815695.
About Orphan Drug Designation in the European Union
(EU)
The European Commission grants Orphan Drug designation (ODD) to
investigational drugs intended to diagnose, prevent or treat a rare
disease. To qualify for ODD, the potential therapeutic must target
a life-threatening or chronically debilitating disease that affects
fewer than five (5) in 10,000 persons in the EU. In addition, the
investigational drug must either provide a significant benefit over
existing therapies or provide a treatment for patients for whom
existing therapies do not work or exist. The designation provides
financial and regulatory incentives to the sponsor company such as
reduced fees, tax waivers, dedicated funds to reimbursement and 10
years of market exclusivity.
About Sickle Cell Disease
Sickle cell disease (SCD) is one of the most common single-gene
disorders and is estimated to affect more than 70,000 in the EU-27,
as well as approximately 100,000 people in the United States. The
National Institutes of Health (NIH) reports that prevalence is
estimated at more than 20 million individuals globally. From 2010
to 2050, the annual number of newborns with SCD is expected to rise
globally by approximately one-third.1 Despite recent advances in
treatment, most patients with SCD still suffer from pain crises,
lifelong disability, significant morbidity and reduced quality of
life.
About FT-4202
FT-4202 is a novel selective red blood cell (RBC) pyruvate
kinase-R (PKR) activator designed to be a disease-modifying therapy
for the treatment of sickle cell disease (SCD). Employing a
multimodal approach, FT-4202 works upstream by activating the RBCs’
natural PKR activity to decrease 2,3-DPG levels, which leads
hemoglobin to hold on to oxygen molecules longer to reduce RBC
sickling. The downstream activity of FT-4202 increases ATP levels,
the fuel that provides energy to cells, to improve RBC health and
survival. Together, these effects are anticipated to increase
hemoglobin levels and decrease painful vaso-occlusive crises. In
preclinical safety studies, FT-4202 did not inhibit aromatase
activity or affect steroidogenesis, important biological processes
responsible for sexual development.
About Forma Therapeutics
Forma Therapeutics is a clinical-stage biopharmaceutical company
focused on the research, development and commercialization of novel
therapeutics to transform the lives of patients with rare
hematologic diseases and cancers. Our R&D engine combines deep
biology insight, chemistry expertise and clinical development
capabilities to create drug candidates with differentiated
mechanisms of action focused on indications with high unmet need.
Our work has generated a broad proprietary portfolio of programs
with the potential to provide profound patient benefit. For more
information, please visit www.FormaTherapeutics.com or follow us on
Twitter @FORMAInc and LinkedIn.
Forward-looking Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995, as amended, including, without limitation, express or implied
statements regarding the potential regulatory, exclusivity and
marketing advantages of Orphan Drug designation by the EU
Commission and previously granted Fast Track, Rare Pediatric
Disease and Orphan Drug designations from the U.S. Food and Drug
Administration (FDA) of FT-4202 for the treatment of patients with
SCD,the advancement of our sickle cell disease program, our
expectations of the therapeutic benefits related thereto, the
timing and success of ongoing clinical trials, and our growth as a
company and the anticipated contribution of our executives and
employees to our operations and progress. The words “may,” “will,”
“could,” “would,” “should,” “expect,” “plan,” “anticipate,”
“intend,” “believe,” “estimate,” “predict,” “project,” “potential,”
“continue,” “target” and similar expressions are intended to
identify forward-looking statements, although not all
forward-looking statements contain these identifying words.
Any forward-looking statements in this press release are based
on management’s current expectations and beliefs and are subject to
a number of risks, uncertainties and important factors that may
cause actual events or results to differ materially from those
expressed or implied by any forward-looking statements contained in
this press release, including, without limitation, those risks and
uncertainties related to the advancement of our clinical programs
and other risks identified in our SEC filings, including those
risks discussed under the heading “Risk Factors” in our Quarterly
Report on Form 10-Q for the quarter ended June 30, 2020, as well as
other risks detailed in our subsequent filings with the SEC. We
caution you not to place undue reliance on any forward-looking
statements, which speak only as of the date they are made. We
disclaim any obligation to publicly update or revise any such
statements to reflect any change in expectations or in events,
conditions or circumstances on which any such statements may be
based, or that may affect the likelihood that actual results will
differ from those set forth in the forward-looking statements. Any
forward-looking statements contained in this press release
represent our views only as of the date hereof and should not be
relied upon as representing its views as of any subsequent date. We
explicitly disclaim any obligation to update any forward-looking
statements.
____________________
1Piel, F. B., Hay, S. I., Gupta, S., Weatherall, D. J., &
Williams, T. N. (2013). Global burden of sickle cell anaemia in
children under five, 2010-2015: Modelling based on demographics,
excess mortality, and interventions. PLOS Medicine, 10(7).
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Media Contact: Kari Watson, +1 781-235-3060 MacDougall
kwatson@macbiocom.com Investor Contacts: Mario Corso, +1
781-366-5726 Forma Therapeutics mcorso@formatherapeutics.com
Stephanie Ascher, +1 212-362-1200 Stern Investor Relations
stephanie.ascher@sternir.com
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