On August 17, 2023 Nuvectis Pharma, Inc. (NASDAQ: NVCT) ("Nuvectis" or the "Company"), a clinical-stage biopharmaceutical company focused on the development of innovative precision medicines for the treatment of serious conditions of unmet medical need in oncology, reported that NXP800 was granted Orphan Drug Designation by the United States Food and Drug Administration ("FDA") for the treatment of cholangiocarcinoma (Press release, Nuvectis Pharma, AUG 17, 2023, View Source [SID1234634478]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Ron Bentsur, Chairman and Chief Executive Officer of Nuvectis, commented, "We are pleased to have received this designation from the FDA for NXP800, which emphasizes the need for new, innovative therapies for the treatment of cholangiocarcinoma, a deadly malignancy with only limited treatment options available. NXP800 is an oral small molecule with a novel mechanism of action that has demonstrated robust activity in several preclinical cancer models, including ARID1a-mutated ovarian, endometrial and gastric carcinomas, as well as cholangiocarcinoma. The clinical activity of NXP800 is currently being evaluated in a Phase 1b clinical trial in patients with ARID1a-mutated ovarian carcinoma, with additional diseases planned for clinical investigations. This orphan drug designation is an important milestone in our journey toward our mission of developing NXP800 for the treatment of serious conditions of unmet medical need in oncology."

About Orphan Drug Designation

Orphan Drug Designation is granted by the FDA to drugs or biologics intended to treat a rare disease or condition, defined as one that affects fewer than 200,000 people in the United States. Orphan Drug Designation provides certain financial incentives to support clinical development, and the potential for up to seven years of marketing exclusivity for the product for the designated orphan indication in the United States if the product is approved for its designated indication.

About Cholangiocarcinoma

Cholangiocarcinoma is a cancer of the biliary tract originating in the epithelium of the biliary tree accounting for approximately 3% of all gastrointestinal malignancies, with an annual incidence of approximately 8,000 – 10,000 in the United States. Surgical resection is the only potentially curative treatment for cholangiocarcinoma, but the disease is often diagnosed as unresectable because of local extension and/or metastases. While several targeted therapies have been approved in recent years for subsets of patients with cholangiocarcinoma based on specific tumor genetics, the overall prognosis remains poor with 5-year survival rates of 20-50% after resection and almost 0% in unresectable tumors.