Certa Therapeutics' FT011 Granted US FDA Orphan Drug Designation for the Treatment of Systemic Sclerosis

MELBOURNE, Australia, Oct. 23, 2023 /PRNewswire/ -- Certa Therapeutics (Certa), a biotechnology company developing innovative precision therapies for patients with inflammatory and fibrotic diseases, today announces that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for its investigational therapy FT011 for the treatment of systemic sclerosis (scleroderma).

Orphan Drug Designation is granted by the FDA to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the U.S. Orphan drug status provides benefits to drug developers, including assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees and seven years of post-approval marketing exclusivity.¹ 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).

"We are very pleased that the FDA has granted Orphan Drug Designation to FT011 which we believe highlights the urgent need for innovation and new therapeutic options for scleroderma patients" said Professor Darren Kelly, Certa Therapeutics CEO and founder. "This designation represents an important milestone in the development of FT011, which has the potential to establish first-in-class clinical benefits by precisely targeting the root cause of fibrosis and offer treatment across multiple organs within these patients."

Certa previously announced positive top-line results from a Phase 2 study which indicated that treatment with FT011 for 12 weeks resulted in a clinically meaningful improvement in 60% of patients treated with FT011 400mg (p = 0.019 vs. placebo) and 20% of patients in the FT011 200mg group compared with 10% in the placebo group.² Three patients in the pooled FT011 groups achieved a maximum  CRISS score of 1.0, representing the greatest probability of clinical improvement.³ The study safety profile demonstrated that FT011 was safe and well tolerated, with no differences in adverse event (AE) rates between the treatment arms. There were no serious AEs reported in the study, nor any AEs resulting in study drug interruption, withdrawal, or discontinuation.

Data from the Phase 2 study will be presented at the American College of Rheumatology Annual Meeting 2023 (ACR Convergence), taking place between 10-15 November 2023, in San Diego CA.

1. U.S. Food and Drug Administration Orphan Drug Designation. Available at: https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/designating-orphan-product-drugs-and-biological-products Accessed October 9, 2022.
2. https://certatherapeutics.com/ground-breaking-results-in-phase-2-scleroderma-study-by-certa-therapeutics-demonstrates-improvement-in-more-than-60-of-patients/ 
3. A formal sample size calculation for efficacy endpoints was not conducted for this study.
4. D Khanna, et al., Arthritis Rheumatol. 2016; 68(2): 299–311. doi:10.1002/art.39501
5. S Eddy, et al., "Identification of Non-Invasive Surrogates as Predictors of Response to FT011 in Kidney Disease" ASN Kidney Week 2022, abstract 3767783

About the Phase 2 studyCerta's Phase II trial was a randomised, double blind, placebo-controlled study of the pharmacokinetics, pharmacodynamic effects and safety of oral FT011 doses in participants with diffuse systemic sclerosis (SSc).

ClinicalTrials.gov Identifier: NCT04647890

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 associated with a 2.5-fold higher risk of mortality than the general population.

About Certa Therapeutics & FT011Certa Therapeutics is a biotechnology company focused on improving lives by treating patients with debilitating diseases via novel targeted therapies. Since its inception in 2018, Certa has secured over $28m from Australian investors Brandon Biocatalyst and Uniseed.

FT011 is a novel, first-in-class oral therapy for the treatment of chronic fibrosis in multiple organs advancing through clinical development for scleroderma (systemic sclerosis). It targets a previously important but undrugged membrane GPCR receptor with an extensive body of data demonstrating promising efficacy in multiple models of fibrotic disease. Transcriptomic research has demonstrated that treatment with FT011 results in reversal in the activation of genetic markers associated with fibrosis, providing potential for a precision therapy.⁵

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.

Certa Therapeutics is seeking to combine these innovative therapeutics with biomarkers and genetic analysis to identify those patients most likely to benefit from treatment. Significant breakthroughs are urgently needed in this field, addressing a market worth more than US$15B annually.

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.

https://certatherapeutics.com/

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