Nuvectis' NXP800 Secures FDA Orphan Drug Status For Cancer Treatment

Nuvectis Pharma has received Orphan Drug Designation from the US Food and Drug Administration (FDA) for its drug NXP800, aimed at treating ARID1a-deficient cancers of the ovary, fallopian tube, and primary peritoneum. ARID1a, a crucial tumor suppressor protein, works alongside p53 to manage tumor growth in gynecologic cancers. NXP800 is an oral small molecule that activates the GCN2 kinase, leading to the death of cancer cells through prolonged GCN2 activation.

Currently, NXP800 is undergoing a Phase 1b clinical trial for platinum-resistant ovarian carcinoma with ARID1a mutations and is also being tested for cholangiocarcinoma. The Orphan Drug Designation from the FDA is awarded to therapies for rare diseases affecting fewer than 200,000 people in the US. This status provides financial benefits for development and, if approved, grants up to seven years of exclusive marketing rights in the US.

Ron Bentsur, Nuvectis Chairman and CEO, said in a statement, “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.”

He further added, “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, a US-based biopharmaceutical firm, specializes in creating advanced precision treatments for severe and unmet medical needs in oncology. The company is advancing two key drug candidates: NXP800 and NXP900. The FDA has awarded Fast Track Designation to NXP800 for its development against platinum-resistant ovarian carcinoma with ARID1a mutations. Meanwhile, NXP900, an oral small molecule targeting the SRC Family of Kinases, is currently undergoing a Phase 1a dose escalation trial. This drug employs a distinctive mechanism to block both the catalytic and scaffolding functions of SRC kinase, effectively shutting down the entire signaling pathway.