On February 3, 2020 Soligenix, Inc. (Nasdaq: 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, reported that the Japanese Patent Office has granted the patent titled "Novel Peptides and Analogs for Use in the Treatment of Oral Mucositis (Press release, Soligenix, FEB 3, 2020, View Source [SID1234553785])." This allowance builds on similar intellectual property in the United States (US), New Zealand, Australia and Singapore and patent applications pending in other jurisdictions worldwide. The new claims cover therapeutic use of dusquetide (active ingredient in SGX942) and related innate defense regulator (IDR) analogs, and add to composition of matter claims for dusquetide and related analogs that have been granted in the US and worldwide. Dusquetide previously demonstrated positive results in a Phase 2 oral mucositis clinical trial and a pivotal Phase 3 study is ongoing, with a positive interim analysis completed in August 2019 and final topline results expected in Q2 2020.
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Based on the positive and previously published Phase 2 results, the pivotal Phase 3 clinical trial is a highly powered, double-blind, randomized, placebo-controlled, multinational trial. The study, called DOM-INNATE (Dusquetide treatment in Oral Mucositis – by modulating INNATE immunity), is enrolling approximately 260 subjects with head and neck cancer (HNC) undergoing standard chemoradiation therapy (CRT) and who are therefore at high risk of developing severe oral mucositis. Enrollment is ongoing in the US and across Europe with enrollment completion expected in Q1 2020 and final topline primary endpoint results anticipated in Q2 2020.
"Soligenix continues to pursue broad patent coverage for its IDR technology, including dusquetide, first with composition of matter claims followed by therapeutic use claims in oral mucositis," stated Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. "Having successfully pursued composition of matter claims in jurisdictions worldwide, we continue to pursue therapeutic use claims like those issued in the US previously and in Japan with this most recent patent. The therapeutic use claims are expected to be generally valid until 2034, which provides significant patent protection and life to dusquetide and our other IDRs. This allowance is timely as we continue to have discussions with potential strategic partners and pursue all options to advance our pipeline and plan for commercial activities."
About Oral Mucositis
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, based upon review of historic published studies and reports and an interpolation of data on the incidence of mucositis, 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.
The mechanisms of mucositis have been extensively studied and have been recently linked to the interaction of chemotherapy and/or radiation therapy with the innate defense system. Bacterial infection of the ulcerative lesions is now regarded as a secondary consequence of dysregulated local inflammation triggered by therapy-induced cell death, rather than as the primary cause of the lesions.
It is estimated, based upon review of historic published studies and reports and an interpolation of data on the incidence of oral mucositis, that oral mucositis in HNC is a subpopulation of approximately 90,000 patients in the US, with a comparable number in Europe. Oral mucositis almost always occurs in patients with HNC treated with CRT and is severe, causing inability to eat and/or drink, in >80% of patients. It is common (40-100% incidence) in patients undergoing high dose chemotherapy and hematopoietic cell transplantation, where the incidence and severity of oral mucositis depends greatly on the nature of the conditioning regimen used for myeloablation.
In the pediatric population, head and neck cancer is a rarer occurrence and is caused by different underlying pathologies. The major types of HNC in children are lymphoma, sarcomas (including rhabdomyosarcomas), and neuroblastoma rather than squamous cell carcinoma, the major type of adult HNC cancers. Hematopoietic stem cell transplantation (HSCT), especially allogeneic transplantation with higher risk of oral mucositis, is more frequently used in the pediatric population than in adults when treating a number of primary tumor types, as seen in leukemia and lymphoma. Both treatment of HNC and HSCT are associated with high risk of oral mucositis in the pediatric population.
Oral mucositis remains an area of unmet medical need where there are currently no approved drug therapies in the context of any solid tissue tumors.
About Dusquetide
Dusquetide (the active ingredient in SGX942) is an IDR, a new class of short, synthetic peptides. It has a novel mechanism of action whereby it modulates the body’s reaction to both injury and infection towards an anti-inflammatory, anti-infective and tissue healing response. IDRs have no direct antibiotic activity but, by modulating the host’s innate immune system responses, increase survival after infections caused by a broad range of bacterial Gram-negative and Gram-positive pathogens. It also accelerates resolution of tissue damage following exposure to a variety of agents including bacterial pathogens, trauma and chemo- and/or radiation therapy. Preclinical efficacy and safety has been demonstrated in numerous animal disease models including mucositis, colitis, macrophage activation syndrome (MAS) as well as bacterial infections, including melioidosis.
SGX942 has demonstrated safety and tolerability in a Phase 1 clinical study in 84 healthy human volunteers. Positive efficacy results were demonstrated in an exploratory Phase 2 clinical study (Study IDR-OM-01) in 111 patients with oral mucositis due to CRT for HNC, including potential long-term ancillary benefits. Soligenix is working with leading oncology centers in the US and Europe to advance SGX942 in oral mucositis with the conduct of a pivotal Phase 3 clinical trial (Study IDR-OM-02) referred to as the "DOM–INNATE" study (Dusquetide treatment in Oral Mucositis – by modulating INNATE immunity). The multinational, placebo-controlled, randomized Phase 3 study is targeted to enroll approximately 260 subjects with squamous cell carcinoma of the oral cavity and oropharynx, 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 are 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 is the median duration of severe oral mucositis, assessed by oral examination at each treatment visit and then through six weeks following completion of CRT. Oral mucositis is evaluated using the WHO (World Health Organization) Grading system. Severe oral mucositis is defined as a WHO Grade of ≥3. Subjects are to be followed for an additional 12 months after the completion of treatment. An interim analysis for this study has been completed and identified a promising signal.
SGX942 has received Fast Track Designation from the FDA for the treatment of oral mucositis as a result of radiation and/or chemotherapy treatment in HNC patients, as well as Promising Innovative Medicine designation in the United Kingdom by the Medicines and Healthcare Products Regulatory Agency for the treatment of severe oral mucositis in HNC patients receiving CRT. In addition, products containing the same active ingredient, dusquetide, have been granted Fast Track Designation as an adjunctive therapy with other antibacterial drugs, for the treatment of melioidosis and Orphan Drug Designations in the treatment of MAS and the treatment of acute radiation syndrome.
Soligenix has a strong intellectual property position in the IDR technology platform, including composition of matter for dusquetide and related analogs. Dusquetide was developed pursuant to discoveries made by Professors B. Brett Finlay, PhD and Robert Hancock, PhD of the University of British Columbia, Canada. Soligenix has received partial funding from NIH for its oral mucositis clinical studies. The Phase 2 study was supported with a Phase I SBIR grant (#R43DE024032) award, with the Phase 3 study being supported by a Phase II SBIR grant (#R44DE024032) award.