SNGX: Data Review Committee Recommends SNGX to Continue on its Phase II Clinical Trial of SGX942 for the Treatment of Oral Mucositis in Head and Neck Cancer Patients
OBB:SNGX The Background In December, 2013, SNGX (SNGX) initiated the Phase II study of SGX942 for the treatment of oral mucositis in head and neck cancer patients. This Phase II trial is a randomized, double-blind, dose-ranging, placebo-controlled trial, initially set to enroll approximately 75 subjects across three SGX942 dose groups and placebo, focused on demonstrating the safety and biologic activity of SGX942 in patients with tumors of the mouth and oropharynx who often experience debilitating oral mucositis as a consequence of tumor treatment with CRT. The primary efficacy assessment is the comparison of the incidence and/or duration of both ulcerative and severe oral mucositis throughout the subjects' 7 week course of CRT and for an additional 4 weeks thereafter. Other key efficacy measures will assess patient reported outcomes, pharmacoeconomic parameters such as hospitalization and radiation-associated side effects including mouth stiffness and dryness. The Positive Recommendation Earlier today (March 3), Soligenix (SNGX) announced that it has received a positive recommendation from the Data Review Committee (DRC) to continue enrolling into the company's Phase II clinical trial evaluating SGX942 as a treatment for oral mucositis in patients undergoing chemoradiation (CRT) therapy for head and neck cancer. Following DRC review of available data on the subjects enrolled in the trial, the committee recommended that enrollment include an additional 20 subjects randomized into either a single SGX942 dose group or the placebo group to allow for a more targeted assessment of the drug's potential effect and to inform final dose selection in this patient population. The study blind will remain in effect until completion of the trial. We are pleased with the positive recommendation from the DRC, which will allow for a sufficient number of subjects to be enrolled into the most promising dose group to increase the potential of demonstrating a positive effect. With the positive recommendation, we are further convinced the favorable safety profile for SGX942. Given the additional subjects to be enrolled and the primary endpoint assessment through 11 weeks, we estimate top-line results will be available in 2H2015. The Unique Mechanism of Action of SGX942 SGX942 belongs to a new class of compounds called Innate Defense Regulators (IDRs). SGX942 represents a novel and innovative approach to therapeutically modulating the immune system by targeting the innate immune system and has the potential to target multiple indications, especially for the treatment of infections and tissue damages. IDRs provide a novel approach to the control of infection and tissue damage via highly selective binding to an intracellular adaptor protein, sequestosome-1, also known as p62, which has a pivotal function in signal transduction during activation and control of the innate defense system. Sequestosome is a recently identified target for modulation of innate defenses and is expressed in most cell types. IDRs have no direct antibiotic activity but modulate host responses, increasing survival after infections with a broad range of bacterial Gram-negative and Gram-positive pathogens including both antibiotic sensitive and resistant strains, as well as accelerating resolution of tissue damage following exposure to a variety of agents including bacterial pathogens, trauma and chemo- or radiation-therapy. Since IDRs target the host (and not the pathogen), IDRs do not engender resistance and are active against resistant pathogens. In vitro data indicate that the endothelium plays a significant role in SGX94 activity and animal studies show that IDRs selectively promote monocyte and macrophage recruitment to disease sites and accelerate resolution of disease. Though IDR action depends on monocytes and macrophages, there is no dependence on either the adaptive immune system (e.g., T cells and B cells) or neutrophils. This suggests that IDRs may be effective in immunosuppressed patients. Moreover, p62 functions downstream of signaling receptors (TLRs, NODs) responsible for sensing both infection and tissue damage, which gives it a role in innate immune modulation relevant to a wide range of diseases from infection (pathogen sensing) to colitis and mucositis (damage sensing). Market Opportunity SGX942 has the potential to provide adjunctive or stand-alone therapies for the broader antimicrobial/antibiotic, antiviral, anti-inflammatory, and anti-cancer markets. Each of the above mentioned market is a multi- billion dollar market. More narrowly, the tissue damage (mucositis) opportunity alone represents a $1B+ market. Mucositis is a debilitating condition involving extensive ulceration of the oral cavity that frequently affects cancer patients undergoing radiation and chemotherapy treatment. Roughly 90% of patients on radiation (43% severe) and 40% of patients receiving chemotherapy get mucositis. There is an estimated 500,000 cancer patients getting mucositis annually in the United States alone. World-wide, the potential market for mucositis will exceed $1 billion in the next few years. 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. We believe any treatment that accelerates healing and/or diminishes the rate of appearance of mucositis would have a significant beneficial impact on the quality of life of these patients and may allow for more aggressive chemotherapy. 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. As a modulator of the innate immune system, SGX942 has the potential to target both primary and secondary causes of mucositis. Oral mucositis is an area of unmet medical need where there are only limited treatment options. Ccurrently, no drug has been approved for oral mucositis in head and neck cancer. We noticed that there are a few products already on the market for oral mucositis. But the competitive landscape favors SGX942 in our view. Amgen’s Kepivance (Palifermin) is the only approved drug for oral mucositis in transplantation; but is contra-indicated for solid tumor patients. Kepivance is a recombinant form of human keratinocyte growth factor (KGF), a protein that is naturally produced by the body. Kepivance is indicated to decrease the incidence and duration of severe oral mucositis in patients with hematologic malignancies receiving myelotoxic therapy requiring hematopoietic stem cell support. The safety and efficacy of Kepivance have not been established in patients with non-hematologic malignancies. Kepivance must be IV injected for 3 consecutive days before and 3 consecutive days after treatment for a total of 6 injections. Also, Kepivance is expensive at about $10,000 per patient. Access Pharma’s MuGard is approved as a medical device and is dispensed in a ready to use mouth rinse. MuGard is indicated for the management of oral mucositis/stomatitis that may be caused by radiotherapy and/or chemotherapy. Three other medical devices on the market are DARA Biosciences, Gelclair, Edward Pharma’s Mucotrol, and EUSA Pharma’s Caphosol. Gelclair is a prescription mouth gel that is designed and approved for the management and relief of pain caused by mouth sores. Gelclair is established by mixing the powder in a glass of water to form the rinse and patients gargle and spit out. Mucotrol is concentrated oral gel wafer which is indicated for the management and relief of pain from oral lesions associated with oral mucositis/stomatitis. Caphosol is similar to Gelclair. Patients must mix the contents of two ampoules to form a rinse and then swish/spit out. These devices attempt to create a protective barrier around the oral ulceration; however, none of these devices are biologically based. Apparently, most of the above treatment options for mucositis only address the symptoms and do not address the cause of mucositis, while Soligenix’s SGX942 has a new mechanism of action which can address both the primary and the secondary causes of mucositis. We believe that, if approved, SGX942 will command a significant market share of the mucositis market, which could reach $1 billion in the next few years. We believe SGX942 will have peak sales of $300 million and that the potential worldwide market for SGX942 is in excess of $500 million for all applications, including oral mucositis. READ THE LATEST FULL RESEARCH REPORT HERE SUBSCRIBE TO ZACKS SMALL CAP RESEARCH to receive our articles and reports emailed directly to you each morning. Please visit our website for additional information on Zacks SCR and to view our disclaimer.
