Soligenix, a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, has received US Food and Drug Administration (FDA) clearance to advance a pivotal phase 3 clinical trial evaluating SGX942 (dusquetide) for the treatment of oral mucositis in head and neck cancer (HNC) patients being treated with chemoradiation (CRT). Soligenix plans to begin this study in the second quarter of 2017.

Based on positive phase 2 results (Study IDR-OM-01), the upcoming pivotal phase 3 clinical trial (Study IDR-OM-02) will be a highly powered, double-blind, randomized, placebo-controlled, multinational trial that will seek to enroll approximately 190 subjects with squamous cell carcinoma of the oral cavity and oropharynx who are scheduled to receive a minimum total cumulative radiation dose of 55 Gy fractionated as 2.0-2.2 Gy per day with concomitant cisplatin chemotherapy given as a dose of 80-100 mg/m2 every third week. Subjects will be randomized to receive either 1.5 mg/kg SGX942 or placebo given twice a week during and for 2 weeks following completion of CRT. The primary endpoint for the study will be the median duration of severe oral mucositis, which will be assessed by oral examination at each treatment visit and then through 6 weeks following completion of CRT. Oral mucositis will be evaluated using the WHO Grading system. Severe oral mucositis is defined as a WHO Grade of =3. Subjects will be followed for an additional 12 months after the completion of treatment.

The study design incorporates feedback from the FDA as well as from the European Medicines Agency (EMA) via the Scientific Advice process.  The Scientific Advice from the EMA indicates that a single, double-blind, placebo-controlled, multinational, phase 3 pivotal study, if successful, in conjunction with results from the phase 2 dose-ranging study, generally will be considered sufficient to support a marketing authorization application (MAA) for potential licensure in Europe.

"We are pleased to have a pivotal phase 3 study design that incorporates feedback from both the FDA and EMA, and that has the potential to support marketing approval in both the US and the European Union," stated Christopher J. Schaber, PhD, president and chief executive officer of Soligenix.  "Study initiation later this year will represent a significant step forward in our oral mucositis development program.  We look forward to advancing this pivotal trial in an effort to address the significant unmet medical need that currently exists in this patient population."