Certa Therapeutics Receives EU Orphan Designation for FT011, a GPR68 Inhibitor Being Developed as an Anti-Fibrotic Treatment for Systemic Sclerosis
2024年7月22日 - 4:00PM
Certa Therapeutics (Certa), a biotechnology company developing
innovative precision therapies for patients with inflammatory and
fibrotic diseases, today announces that the European Medicines
Agency (EMA) has granted Orphan Drug Designation (ODD) for its lead
drug candidate, FT011, as a potential treatment of Systemic
Sclerosis (SSc). Certa has previously also received ODD and Fast
Track designation for FT011 as a potential treatment of SSc from
the U.S. Food and Drug Administration (FDA).
FT011 is a novel, first-in-class oral therapy for the treatment
of chronic fibrosis in multiple organs. It targets an important but
previously undrugged membrane GPCR receptor, GPR68, a master switch
of fibrosis. An extensive body of data demonstrates promising
efficacy in multiple in vitro and in vivo models of
inflammatory and fibrotic disease. Phase I and IIa clinical studies
in patients with SSc have also demonstrated favourable efficacy,
safety and pharmacokinetics.
SSc is a chronic, progressive, autoimmune disease characterised
by inflammation and fibrosis (scar tissue formation) in the skin
and in various internal organs - commonly lungs, kidneys and heart.
Skin fibrosis is the distinguishing feature of SSc and is
associated with significantly reduced function and disability in
patients. Currently, there are no treatments on the market that
effectively stop or reverse scarring in the skin and organs.
Professor Darren Kelly Certa Therapeutics CEO and
founder said, “GPR68 inhibition modulates the biological
pathways causing inflammation and fibrosis to the skin in patients
with SSc. With limited treatment options available for patients
with SSc, the EMA Orphan Drug Designation and FDA Orphan Drug and
Fast Track Designations reflect the potential for FT011 to address
a critical need for people living with this debilitating and
life-threatening condition.
“These are significant validations for the company and highlight
the unmet need for novel medicines to reduce inflammation and
scarring in chronic fibrotic diseases, which are often progressive
and can have a poor prognosis.”
Certa is planning for a Phase IIb confirmatory SSc clinical
trial with FT011, bringing the company closer to providing better
treatment options for patients with fibrosis. It is also developing
biomarkers and gene signatures to identify patients most likely to
respond to treatment to ensure the best outcomes for those with
fibrosis.
The FDA and EMA grant orphan status to products intended to
treat, diagnose, or prevent life-threatening rare diseases or
conditions that affect fewer than five in 10,000 people in Europe,
or under 200,000 people in the US, and with either no currently
approved method of diagnosis, prevention, or treatment, or with
significant benefit to those affected by the disease. Orphan drug
designation provides certain benefits, including the potential for
extensive marketing exclusivity following regulatory approval,
reduction in regulatory fees and, in the case of EU, a centralised
approval process.
For further information please contact
Media – AustraliaKirrily Davis, E:
kdavis@bcpvc.com M: +61 (0)401 220228
Media - InternationalSue Charles, Charles
Consultants, E: sue.charles@charles-consultants.com M: +44 (0)7968
726585
About Systemic Sclerosis (Scleroderma)
Scleroderma is an extremely debilitating, potentially
life-threatening autoimmune condition characterised by inflammation
and fibrosis of the skin and other organs (commonly the lungs,
kidneys, and heart). This condition results in high morbidity with
substantial detriment on quality of life, with patients commonly
experiencing loss of mobility and function, pain, fatigue, often
accompanied with a significant impact to their mental health.
Scleroderma has the highest mortality among rheumatic diseases.
About Certa Therapeutics Certa
Therapeutics is a clinical-stage biotechnology company focused on
improving lives by treating patients with debilitating diseases via
novel targeted therapies.
Certa Therapeutics has designed a platform of candidate drugs
and validated the role of GPR68, a defined G protein-coupled
receptor (GPCR) receptor which mediates signalling pathways
associated with inflammation and fibrosis. GPR68 is silent in
healthy tissue but activated following injury or disease. Evidence
demonstrates the role of GPR68 on multiple downstream pathways
causing inflammation and fibrosis. These targeted drug candidates
have established proof of concept as potential treatments for
multiple fibrotic diseases including serious and chronic conditions
impacting the kidney, lung, eye, skin, and heart. The morbidity and
mortality impact of fibrotic diseases is substantial, ultimately
causing 45% of all deaths globally. Significant breakthroughs are
urgently needed in this field, addressing a market worth more than
US$15B annually. The company is seeking to combine its innovative
therapeutics with biomarkers and genetic analysis to identify those
patients most likely to benefit from treatment, providing potential
for a precision therapy.
FT011 is an investigational product which has not received
marketing authorisation or approval by any regulatory agency,
including the US Food and Drug Administration, the European
Medicines Agency, or the Australian Therapeutic Goods Agency. The
investigational drug products being developed by Certa Therapeutics
are undergoing clinical studies to evaluate the safety and
effectiveness in humans. In the company’s lead program, FT011 is
being developed as a novel, first-in-class oral therapy for the
treatment of systemic sclerosis (scleroderma) and has successfully
completed a multi-national, double-blinded randomised controlled
trial. In addition to being granted EMA Orphan Drug Designation,
FT011 has been awarded Orphan Drug Designation and Fast Track
Designation from the FDA.
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