SteroTherapeutics Granted Orphan Drug Designation to ST-003, a Novel Drug Candidate for the Treatment of Primary Sclerosing Cholangitis
31 May, 2022, 08:00 ET
- Designation provides fast track reviews and a 30% fungible tax credit for all future clinical development expenses
- Company on track to submit IND in 2023
DOYLESTOWN, Pa., May 31, 2022 /PRNewswire/ -- SteroTherapeutics LLC, a clinical-stage company targeting metabolic and rare diseases, today announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for ST-003, a novel galanin receptor inhibitor, for the treatment of primary sclerosing cholangitis (PSC). PSC is a rare, heterogeneous, idiopathic, inflammatory disorder of the bile ducts resulting in strictures and scarring of the ducts that can gradually cause serious liver damage. ST-003 is designed to halt the impact of excessive galanin concentrations that increase cholangiocyte proliferation and fibrogenesis, as demonstrated in humans and in specific mouse models.
"This important designation is a milestone in the development of ST-003 and highlights the need for potential new treatment options for patients with PSC," said Manohar Katakam, Ph.D., Chief Executive Officer of SteroTherapeutics. "Orphan designation for ST-003 is a critical step toward clinical development as we now have fast track reviews and a 30% fungible tax credit for all future clinical development expenses. ST-003 has the potential to be an important treatment by addressing a host of disease factors found in the PSC disease state. We are especially pleased that this is the second program in our pipeline to receive this designation, as it validates and adds value to our programs."
SteroTherapeutics expects to submit an Investigational New Drug (IND) application to the FDA for ST-003 in 2023 and marks the second program, following ST-002 for the treatment of Cushing's syndrome, to receive Orphan Drug Designation by the FDA.
FDA Orphan Drug Designation is granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States. 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.
About Primary Sclerosing Cholangitis:
Primary sclerosing cholangitis (PSC) is a chronic liver disease in which the bile ducts inside and outside the liver become inflamed and scarred, and eventually narrowed or blocked. When this happens, bile builds up in the liver and causes further liver damage.
SteroTherapeutics, a clinical stage company is focused on developing novel orphan disease therapeutics with significant unmet needs as well as potentially larger indications affecting large patient populations. SteroTherapeutics' lead programs, ST-002 to treat Cushing's syndrome, ST-003 to treat primary sclerosing cholangitis and ST-004 to treat multiple myeloma, have been proven in previous animal and human studies to possess a strong safety profile and established mechanisms of action. Both ST-002 and ST-003 have received Orphan Drug Designation from the U.S. FDA. The company's strategic intent is to focus on platform technologies and corresponding disease pathways in disease states with significant unmet or underserved medical needs. In each individual disease target SteroTherapeutics embraces the opportunity to provide safe, effective treatments and restore an optimal quality of life. Learn more at sterotx.com
SOURCE Sterotherapeutics LLC
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