FORMA Therapeutics Announces FT-4202 Granted Fast Track Designation & Rare Pediatric Disease Designation for Treatment of Sic...
February 20 2020 - 7:05AM
Business Wire
FORMA Therapeutics, Inc., a clinical-stage biopharmaceutical
company focused on rare hematologic diseases and cancers, today
announced the U.S. Food and Drug Administration (FDA) has granted
Fast Track designation and Rare Pediatric Disease designation for
its lead clinical asset, FT-4202, in development as a
disease-modifying treatment for sickle cell disease (SCD). FT-4202
is a selective pyruvate kinase-R (PKR) activator that exhibits the
potential to beneficially impact both anemia and vaso-occlusive
crises for people living with SCD.
“These designations for FORMA’s lead clinical asset underscore
the FDA’s continued recognition of the needs of patients with SCD,”
said Frank Lee, chief executive officer of FORMA Therapeutics.
“With more than 100,000 individuals living with SCD in the U.S.,
and with relatively few treatment options, we believe significant
unmet medical needs persist. These FDA designations give FORMA the
opportunity to accelerate the development of a new treatment for
people impacted by SCD.”
FORMA is currently enrolling patients with SCD in a Phase 1
study to evaluate the safety and pharmacokinetics/pharmacodynamics
(PK/PD) of FT-4202. FORMA plans to initiate a registrational trial
within the next year. For more information on eligibility and study
sites for the open Phase 1 study, please visit
clinicaltrials.gov/NCT03815695.
About Fast Track Designation
The FDA’s Fast Track designation is a process designed to
facilitate the development and expedite the review of drugs to
treat serious conditions and fill an unmet medical need. Fast Track
designation allows for early and frequent communication with the
FDA throughout the entire drug development and review process. It
also enables eligibility for Accelerated Approval and Priority
Review, as well as a rolling review of a company’s New Drug
Application, if relevant criteria are met.1
About Rare Pediatric Disease Designation
Under the FDA’s Rare Pediatric Disease (RPD) program, a sponsor
who receives marketing approval for a product with an RPD
designation may be eligible for a voucher that can be redeemed to
obtain priority review for any subsequent marketing application.
The FDA defines a "rare pediatric disease" as a rare disease in
which the serious or life-threatening manifestations primarily
affect individuals from age zero to 18.2
About Sickle Cell Disease
Sickle cell disease (SCD) is the most common disorder caused by
a single gene mutation. Worldwide, an estimated 300,000 children
are born with SCD each year. In the U.S., prevalence of SCD is
approximately 100,000 people. In people living with SCD, red blood
cells, or RBCs, spontaneously deform in low oxygen conditions,
taking on a sickle-like shape. Sickled cells are stiff and have
damaged membranes, causing the RBCs to clump and burst in small
blood vessels, resulting in inflammation and vaso-occlusive crises.
Repeated deformation also depletes the RBC energy supply, called
ATP. One important consequence of this energy depletion is
increased levels of a metabolite, 2,3-DPG, that further reduces the
RBCs’ affinity for oxygen and exacerbates the cycle of repeated
deformation and anemia.
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 focused on the discovery, development and
commercialization of transformative medicines for patients with
rare hematologic diseases and cancers. A fully integrated
biopharmaceutical company, FORMA’s validated, proprietary R&D
engine combines deep biology insight, chemistry expertise and
clinical development capabilities to create differentiated drug
candidates with best-in-class or first-in-class potential. FORMA
has delivered high-value clinical candidates to its partners and
generated a broad proprietary portfolio of programs, ranging from
preclinical to pivotal-stage, with the potential to provide
profound patient benefit. For more information, please visit the
company website at www.formatherapeutics.com or follow us on
Twitter @FORMAInc and LinkedIn.
1https://www.fda.gov/forpatients/approvals/fast/ucm405399.htm.
Accessed February 2020.
2https://www.fda.gov/news-events/fda-brief/fda-brief-fda-updates-draft-guidance-rare-pediatric-disease-priority-review-voucher-program.
Accessed February 2020.
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