Soligenix’s self-described “DOM–INNATE” study has initiated patient enrollment. The late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, announced that patient enrollment has been opened for its Phase 3, multinational, randomized, double-blind, placebo-controlled study evaluating SGX942 (dusquetide) as a treatment for severe oral mucositis in patients with head and neck cancer receiving chemoradiation therapy (CRT).
Mucositis is the clinical term for damage done to the mucosa by anticancer therapies. It can occur in any mucosal region, but is most commonly associated with the mouth, followed by the small intestine. It is estimated that mucositis affects approximately 500,000 people in the US per year and occurs in 40% of patients receiving chemotherapy.
Mucositis can be severely debilitating and can lead to infection, sepsis, the need for parenteral nutrition and narcotic analgesia. The gastrointestinal damage causes severe diarrhea. These symptoms can limit the doses and duration of cancer treatment, leading to sub-optimal treatment outcomes. Oral mucositis in HNC remains an area of unmet medical need where there are currently no approved drug therapies.
Soligenix has been working with leading oncology centers, a number of which participated in the Phase 2 study, to advance this Phase 3 clinical trial referred to as the “DOM–INNATE” study (Dusquetide treatment in Oral Mucositis – by modulating INNATE immunity). 
Based on the positive and previously published Phase 2 results (Study IDR-OM-01), the 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 two 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 six 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 US Food and Drug Administration (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 for potential licensure in Europe.
“Oral mucositis remains a debilitating side effect of cancer treatments and is particularly severe and prevalent in head and neck cancer patients, where there is currently no approved drug therapy,” stated Stephen T. Sonis, DMD, DMSc, Clinical Professor of Oral Medicine at Harvard School of Dental Medicine and Chair of the Soligenix Oral Mucositis Medical Advisory Board. “Our current understanding of oral mucositis highlights the role of the innate immune response in exacerbating the damage done by CRT. As SGX942 is the first innate defense regulator in development for oral mucositis and has previously demonstrated positive results in a Phase 2 clinical trial, it may have the potential to offer a promising option to treat the extremely devastating consequences of CRT.”
“We are pleased to initiate the pivotal Phase 3 study 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 Richard C. Straube, MD, Senior Vice President and Chief Medical Officer of Soligenix. “In an effort to better maintain study quality and more effectively manage clinical expense, we intend to begin with a controlled roll-out of US study sites, followed by the addition of European centers in early 2018. This will allow us to first ensure protocol adherence in the US before expansion of the study to centers outside the US. 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.”
The Phase 2 oral mucositis clinical study was partially funded with a grant from the National Institute of Dental and Craniofacial Research Small Business Innovation Research grant #1R43 DE024032-01 (Soligenix, Inc.).
Soligenix’s BioTherapeutics business segment is also developing SGX301 as a novel photodynamic therapy, utilizing safe visible light for the treatment of cutaneous T-cell lymphoma, a first-in-class innate defense regulator (IDR) technology, dusquetide (SGX942) for the treatment of oral mucositis in head and neck cancer, and proprietary formulations of oral beclomethasone 17, 21-dipropionate (BDP) for the prevention/treatment of gastrointestinal (GI) disorders characterized by severe inflammation including pediatric Crohn’s disease (SGX203) and acute radiation enteritis (SGX201).
Soligenix’s Vaccines/BioDefense business segment includes active development programs for RiVax®, a ricin toxin vaccine candidate, OrbeShield®, a GI acute radiation syndrome therapeutic candidate and SGX943, the therapeutic candidate for antibiotic resistant and emerging infectious disease. The development of the vaccine programs incorporates the use of the proprietary heat stabilization platform technology, known as ThermoVax®. To date, this business segment has been supported with government grant and contract funding from the National Institute of Allergy and Infectious Diseases (NIAID) and the Biomedical Advanced Research and Development Authority (BARDA).
For further information regarding Soligenix, Inc., please visit the company’s website.