Skip to main content
Premium Trial:

Request an Annual Quote

Onconova Therapeutics Begins Rigosertib Study in Rare Squamous Cell Cancer Subgroup

NEW YORK – Onconova Therapeutics has enrolled the first patient in a Phase II trial evaluating the efficacy and safety of rigosertib in patients with recessive dystrophic epidermolysis bullosa-associated, locally advanced or metastatic squamous cell carcinoma.

The patient has received a dose of the investigational targeted drug at EB House Austria within the University Hospital in Salzburg, which specializes in epidermolysis bullosa, an incurable skin blistering condition due to a lack of collagen in the skin that occurs in individuals who inherit two mutated copies of the COL7A1 gene. Young patients with this condition have a very high risk of squamous cell carcinoma that tends to be aggressive and can be fatal.

"Recessive dystrophic epidermolysis bullosa is a genetic skin blistering disease that often results in squamous cell carcinoma in severe subtypes," Onconova CEO Steven Fruchtman said in a statement. "In this patient cohort, squamous cell carcinoma is the leading cause of death ... We hope rigosertib can prove beneficial to this rare patient population with a tremendous unmet medical need."

Onconova's open-label investigator-initiated study will enroll a dozen patients who have recessive dystrophic epidermolysis bullosa-associated advanced squamous cell carcinoma and failed standard treatments. As a primary endpoint, researchers will track their response to oral or intravenous rigosertib. They will also evaluate biomarkers in archival tissue samples from all patients, as well as their quality of life on the drug. Patients will receive rigosertib for up to a year, and the trial will be ongoing for two-and-a-half years.

This study is being supported by Onconova and DEBRA International, the leading advocacy network for epidermolysis bullosa. Pennsylvania-headquartered Onconova is also studying rigosertib in combination with nivolumab (Bristol Myers Squibb's Opdivo) in a trial that began enrolling KRAS-mutated lung adenocarcinoma patients last year.