Soligenix, Inc. (OTCQB: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, announced today positive results in its Phase 2 clinical trial, in which SGX942, a first-in-class Innate Defense Regulator (IDR), at a dose of 1.5 mg/kg, successfully reduced the median duration of severe oral mucositis by 50% in all patients and by 67% in patients receiving the most aggressive chemoradiation therapy (CRT) for treatment of their head and neck cancer.
This exploratory study achieved all objectives, including identifying a best dose of 1.5 mg/kg. The study enrolled 111 patients across three SGX942 dose groups (i.e., 1.5, 3.0, and 6.0 mg/kg) and a placebo group and evaluated patients undergoing CRT for head and neck cancer. In the 1.5 mg/kg treatment group, the median duration of severe oral mucositis was decreased by 50%, from 18 days to 9 days (p=0.099), meeting the prospectively defined statistical threshold of p<0.1 in the study protocol. Further, patients receiving the most aggressive CRT in this dose group had even more striking reductions in their median duration of severe oral mucositis of 67%, from 30 days to 10 days (p=0.040). Clinicians are most concerned about severe oral mucositis, which includes patients who cannot eat and/or drink due to their mouth ulcers.
In addition to the oral mucositis findings, a trend towards increased incidence of "complete response" of tumor at the one month follow up visit was observed (47% in placebo vs. 63% in SGX942 at 1.5 mg/kg). Decreases in mortality and significant decreases in infection rate were also observed with SGX942 treatment and are being further evaluated. In the preliminary analysis, SGX942 was found to be generally safe and well tolerated, consistent with the safety profile observed in the prior Phase 1 study conducted in 84 healthy volunteers. Long-term follow-up evaluations are ongoing with final results expected in the fourth quarter of 2016. Data from this Phase 2 trial is expected to be submitted for future presentation and publication.
One of the leading investigators in oral mucositis, Stephen T. Sonis, DMD, DMSc, Professor of Oral Medicine at Harvard School of Dental Medicine and Member of the Soligenix Oral Mucositis Medical Advisory Board noted, "Oral mucositis is a devastating side effect of CRT used for the treatment of head and neck cancers, which compromises patients' tolerability of therapy and quality of life, and adds to health and economic burdens associated with care. Despite intense efforts to develop effective interventions for CRT-induced mucositis, there are currently no approved drugs. SGX942 is the first Innate Defense Regulator in development for oral mucositis. The favorable results noted in this trial demonstrate the validity of a novel treatment approach which targets critical biological events leading to mucositis and strongly support further trials. The clinical results are consistent with preclinical animal studies in which SGX942 reduced duration of oral mucositis by approximately 50% following either chemotherapy or radiation therapy."
"We are extremely pleased with the findings from the first Phase 2 study of SGX942. The study met all of its objectives of defining the best dose, the most appropriate clinical endpoint, and patient population to use in future pivotal trials," stated Richard Straube, MD, Senior Vice President and Chief Medical Officer of Soligenix. "The positive and clinically meaningful effect observed on the duration of ulcerative and severe oral mucositis clearly demonstrates the significant biologic activity that SGX942 exerts on this catastrophic side-effect of cancer treatment. The impact of SGX942 on other clinically beneficial outcomes (e.g., tumor control and infection rates) confirms the animal model data that, in treating patients' oral mucositis, SGX942 may also prove to have a positive impact on their head and neck cancer."
"After years of development, it is truly gratifying to witness this transformational event, in which the potential of the IDR technology begins to be translated into the clinical setting," stated B. Brett Finlay, PhD, University of British Columbia, Peter Wall Distinguished Professor, CIFAR Senior Fellow Michael Smith Laboratories and co-founder of the IDR technology. "With their unique mechanism of action, IDRs have the potential to not only mitigate oral mucositis, but also address the increasing problem of emerging and antibiotic resistant bacterial infections."
"We are very excited with the outcome of this Phase 2 clinical study," stated Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. "Given the positive and compelling preliminary data generated in oral mucositis, where there is substantial market potential, we will now look to engage the US Food and Drug Administration (FDA) on the size and scope of a pivotal Phase 2/3 clinical program since the data suggest that a pivotal trial could potentially be designed of a size roughly similar to the completed trial. Simultaneous with this activity, we will be aggressively pursuing opportunities for partnership to support subsequent clinical trials with SGX942. With the biologic activity of the IDR technology now confirmed in humans, we will also look to expand its potential utility in the infectious disease space."
Dr. Schaber continued, "With the successful completion of the Phase 2 oral mucositis study, we now direct clinical efforts to the quality execution of our pivotal Phase 3 clinical trial in cutaneous T-cell lymphoma (CTCL) with SGX301, which was recently opened for enrollment. CTCL is a high priority disease indication, having received fast track and orphan designation from the FDA, and has the potential to support marketing registration in the US and the rest of the world."