Soligenix, Inc.,  a late-stage biopharmaceutical company, announced SGX942 (dusquetide) has been granted Promising Innovative Medicine (PIM) designation in the United Kingdom (UK) by the Medicines and Healthcare Products Regulatory Agency (MHRA) for the treatment of severe oral mucositis in head and neck cancer patients receiving chemoradiation therapy.

The PIM designation is the first step towards inclusion in the Early Access to Medicines Scheme (EAMS).  Launched in April 2014, EAMS offers severely ill patients with life-threatening and seriously debilitating conditions the lifeline of trying ground-breaking new medicines much earlier than they would normally be accessible.

PIM status, the first phase of EAMS, which is awarded following an assessment of early nonclinical and clinical data by the MHRA, has been created as an early signal to companies that the development plan is appropriate and indicates that a product could be a candidate for the second phase of the EAMS scheme, once further development work has been conducted.  In this second phase, the product is made available to UK patients before a marketing authorization is approved.  This early boost to a drug's potential is expected to be beneficial to companies, especially small and medium-sized enterprises.

The criteria products must meet to obtain the PIM designation are: Criterion 1 – The condition should be life-threatening or seriously debilitating with a high unmet medical need (i.e., there is no method of treatment, diagnosis or prevention available or existing methods have serious limitations); Criterion 2 – The medicinal product is likely to offer major advantage over methods currently used in the UK; Criterion 3 – The potential adverse effects of the medicinal product are likely to be outweighed by the benefits, allowing for the reasonable expectation of a positive benefit risk balance.  A positive benefit risk balance should be based on preliminary scientific evidence, as justified by the applicant, that the safety profile of the medicinal product is likely to be manageable and acceptable in relation to the estimated benefits.

"We are excited that the MHRA agrees that dusquetide meets the specified criteria for PIM designation based on the phase 2 oral mucositis clinical data, in conjunction with the consistency that has been observed in previous preclinical and phase 1 clinical studies," stated Christopher J. Schaber, PhD, president and chief executive officer of Soligenix.  "We look forward to working with the MHRA to advance the program and leverage the potential benefits of the EAMS scheme to make this important product available to patients and physicians facing the challenges of severe oral mucositis."

Dusquetide (the active ingredient in SGX942) is an innate defense regulator (IDR), a new class of short, synthetic peptides.  It has a novel mechanism of action in that it modulates the body's reaction to both injury and infection towards an anti-inflammatory and an anti-infective response.  IDRs have no direct antibiotic activity but, by modulating the host's innate immune system responses, increase survival after infections with 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, melioidosis, macrophage activation syndrome (MAS) and other bacterial infections.  

SGX942 has demonstrated safety in a phase 1 clinical study in 84 healthy human volunteers.  Recently, SGX942 has demonstrated preliminary efficacy and safety in an exploratory phase 2 clinical study in 111 patients with oral mucositis due to chemoradiation (CRT) therapy for head and neck cancer.  Consistent with preclinical findings, SGX942 at a dose of 1.5 mg/kg demonstrated positive improvements in decreasing the duration of severe oral mucositis by 50% overall compared to the placebo group, from 18 days to 9 days (p=0.099).  In patients at highest risk of oral mucositis (e.g., those exposed to the most aggressive concomitant chemotherapy), the reduction in the duration of severe oral mucositis was even more significant at 67% when treated with SGX942 1.5 mg/kg, from 30 days to 10 days (p=0.04).  The p-values meet the prospectively defined statistical threshold of p<0.1 in the study protocol.  Additional observations included an improved tumor response to CRT therapy at the one month follow-up visit, as well as decreases in infection rate.  The study results are reviewed in "Dusquetide: A Novel Innate Defense Regulator Demonstrating a Significant and Consistent Reduction in the Duration of Oral Mucositis in Preclinical Data and a Randomized, Placebo-Controlled Phase 2a Clinical Study" published online in the Journal of Biotechnology and are available at the following link: http://dx.doi.org/10.1016/j.jbiotec.2016.10.010. Long-term (12 month) follow-up data further indicated the safety and tolerability of SGX942 treatment, with a trend towards reduced mortality and increased tumor resolution in the 1.5 mg/kg SGX942 treatment group.  Opioid pain medication use was also seen to decrease over the course of CRT in the 1.5 mg/kg SGX942 treatment group at the point of highest oral mucositis risk, while it increased in the placebo group.

Dusquetide and related analogs have a strong intellectual property position, including composition of matter.  Dusquetide was developed pursuant to discoveries made by Professors B. Brett Finlay, PhD and Robert Hancock, PhD of the University of British Columbia, Canada.

Drug products containing dusquetide have also received Fast Track Designations from the FDA for the treatment of oral mucositis as a result of radiation and/or chemotherapy treatment in head and neck cancer patients, and as an adjunctive therapy with other antibacterial drugs, for the treatment of melioidosis. Orphan Drug Designations for use of dusquetide in the treatment of MAS as well as for the treatment of acute radiation syndrome have also been granted.

Soligenix is a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need.