Nuvectis Pharma has secured the US Food and Drug Administration (FDA) Orphan Drug Designation for NXP800 to treat ARID1a-deficient ovarian, fallopian tube, and primary peritoneal cancers.
ARID1a (AT-rich interactive domain-containing protein 1a) is a tumour suppressor that collaborates with p53 to regulate tumour growth in gynecologic cancers.
NXP800 is an orally bioavailable small molecule activator of the GCN2 kinase, which works by chronic activation of GCN2 to induce cancer cell death.
The drug is currently in a Phase 1b clinical trial for the treatment of platinum-resistant, ARID1a-mutated ovarian carcinoma and in a clinical trial for the treatment of cholangiocarcinoma.
The US health regulator grants Orphan Drug Designation to drugs that are intended to treat a rare disease or condition, which affects fewer than 200,000 people in the US.
The designation provides certain financial incentives to support the clinical development, and up to seven years of marketing exclusivity for the product in the US if approved.
Nuvectis chairman and CEO Ron Bentsur said: “We are very pleased to have received this designation from the FDA for NXP800.
“The prevalence of ovarian cancer, which is comprised of ovarian, fallopian tube and primary peritoneal cancers, exceeds the 200,000-patient threshold below which drugs may be eligible to receive Orphan Drug Designation in the United States and in ovarian cancer it has been uncommon to receive this designation for the treatment of a subset of the disease.
“We, therefore, believe that this Orphan Drug Designation granted by the FDA for NXP800 for the treatment of a subset of ovarian cancer, specifically for patients with an ARID1a deficiency, provides further validation for NXP800’s mechanism of action and the target patient population in our ongoing Phase 1b clinical trial in patients with platinum-resistant, ARID1a-mutated ovarian cancer. We expect to provide a data update from this study this coming fall.”
Nuvectis Pharma is a US-based biopharmaceutical company developing advanced precision medicines for the treatment of serious conditions of unmet medical need in oncology.
The company is currently developing two candidates, NXP800 and NXP900.
The US Food and Drug Administration granted Fast Track Designation for the NXP800 development in platinum-resistant, ARID1a-mutated ovarian carcinoma.
NXP900 is an oral small molecule inhibitor of the SRC Family of Kinases, currently developed in a Phase 1a dose escalation study.
The drug works with a unique mechanism which inhibits both the catalytic and scaffolding functions of the SRC kinase, to completely close the signaling pathway.
