PRINCETON - 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, announced an interim update on the open-label, investigator-initiated study (IIS) evaluating extended HyBryte treatment for up to 12 months in patients with early-stage cutaneous T-cell lymphoma (CTCL).
The trial is sponsored by Ellen Kim, MD, Director, Penn Cutaneous Lymphoma Program, Vice Chair of Clinical Operations, Dermatology Department, and Professor of Dermatology at the Hospital of the University of Pennsylvania who was a leading enroller in the Phase 3 FLASH (Fluorescent Light Activated Synthetic Hypericin) study for the treatment of early-stage CTCL. To date six patients have been enrolled and treated with HyBryte over a time period ranging up to 44 weeks. Patients have responded positively to HyBryte therapy with 75% (3 of the 4 subjects who have completed at least 12 weeks of therapy) already achieving 'Treatment Success', as predefined in the study's protocol as 50% improvement in their cumulative mCAILS (modified Composite Assessment of Index Lesion Severity) score compared to Baseline. Of the three Treatment Successes, two were achieved within the first 12 weeks of treatment and the third within 18 weeks. Of the remaining three patients, two have only recently started HyBryte therapy and have not yet reached their first efficacy evaluation visit (i.e., at Week 6) and the other had a substantial improvement documented at the Week 18 visit, but has not yet achieved the success threshold. In addition, HyBryte appears to be safe and well tolerated in all patients, with no treatment-related adverse events reported to date.
'In the Phase 3 FLASH study, HyBryte was shown to be efficacious with a promising safety profile. With limited treatment options, especially in the early stages of their disease, CTCL patients are often searching for alternative treatments. In our U.S. Food and Drug Administration (FDA)-funded study, initial results evaluating the expanded use of HyBryte in a 'real world' treatment setting are promising, further supporting and extending results from the previous positive Phase 2 and 3 clinical trials,' noted Dr. Kim, Principal Investigator of the IIS. 'We look forward to continuing to work with the FDA to complete this study and to participating in the upcoming confirmatory Phase 3 placebo-controlled study.'
'We are pleased with the initial study results and with the FDA's support of the IIS that provides CTCL patients an opportunity to access HyBryte in an open-label setting,' stated Christopher J. Schaber, President and CEO of Soligenix, Inc. 'CTCL is an incredibly difficult to treat orphan disease and remains an area of unmet medical need with a very limited number of safe and effective treatment options. Following the initial Phase 3 FLASH study which demonstrated the safety and efficacy of shorter courses of HyBryte therapy, we are pleased to see that continuing treatment for longer time periods is resulting in the anticipated improved outcomes for patients. As the body of compelling data continues to grow in support of this novel therapy, we look forward to working with Dr. Kim on this important study as well as embarking on our confirmatory Phase 3 replication study later this year. We will continue to provide additional updates on the IIS as data becomes available.'
The clinical study RW-HPN-MF-01, 'Assessment of Treatment with Visible Light Activated Synthetic Hypericin Ointment in Mycosis Fungoides Patients' is designed as an open-label, multicenter clinical trial enrolling up to 50 patients at select U.S. clinical centers. Patients have the potential to be treated for up to 12 months with twice a week dosing (visible light activation following ointment application by 24 +/- 6 hours). The study also allows for potential transition to a 'real-world' setting with home-use. The primary endpoint for the study is evaluating the number of treatment successes defined as 50% reduction in the cumulative mCAILS score from Baseline to end of the treatment.
About HyBryte
HyBryte (research name SGX301) is a novel, first-in-class, photodynamic therapy utilizing safe, visible light for activation. The active ingredient in HyBryte is synthetic hypericin, a potent photosensitizer that is topically applied to skin lesions that is taken up by the malignant T-cells, and then activated by safe, visible light approximately 24 hours later. The use of visible light in the red-yellow spectrum has the advantage of penetrating more deeply into the skin (much more so than ultraviolet light) and therefore potentially treating deeper skin disease and thicker plaques and lesions. This treatment approach avoids the risk of secondary malignancies (including melanoma) inherent with the frequently employed DNA-damaging drugs and other phototherapy that are dependent on ultraviolet exposure. Combined with photoactivation, hypericin has demonstrated significant anti-proliferative effects on activated normal human lymphoid cells and inhibited growth of malignant T-cells isolated from CTCL patients. In a published Phase 2 clinical study in CTCL, patients experienced a statistically significant (p=0.04) improvement with topical hypericin treatment whereas the placebo was ineffective. HyBryte has received orphan drug and fast track designations from the U.S. Food and Drug Administration (FDA), as well as orphan designation from the European Medicines Agency (EMA).
The published Phase 3 FLASH trial enrolled a total of 169 patients (166 evaluable) with Stage IA, IB or IIA CTCL. The trial consisted of three treatment cycles. Treatments were administered twice weekly for the first 6 weeks and treatment response was determined at the end of the 8th week of each cycle. In the first double-blind treatment cycle (Cycle 1), 116 patients received HyBryte treatment (0.25% synthetic hypericin) and 50 received placebo treatment of their index lesions. A total of 16% of the patients receiving HyBryte achieved at least a 50% reduction in their lesions (graded using a standard measurement of dermatologic lesions, the CAILS score) compared to only 4% of patients in the placebo group at 8 weeks (p=0.04) during the first treatment cycle (primary endpoint). HyBryte treatment in this cycle was safe and well tolerated.
In the second open-label treatment cycle (Cycle 2), all patients received HyBryte treatment of their index lesions. Evaluation of 155 patients in this cycle (110 receiving 12 weeks of HyBryte treatment and 45 receiving 6 weeks of placebo treatment followed by 6 weeks of HyBryte treatment), demonstrated that the response rate among the 12-week treatment group was 40% (p
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