Expanded HyBryte™ Treatment Demonstrating Positive Outcomes in Early-Stage Cutaneous T-Cell Lymphoma
Soligenix, a late-stage biopharmaceutical company, announced promising interim results for its HyBryte™ treatment in an open-label, investigator-initiated study for early-stage cutaneous T-cell lymphoma (CTCL). Supported by a $2.6 million FDA Orphan Products Development Grant, the study evaluated HyBryte™ treatment extended up to 12 months. Six patients have been enrolled, with 75% of patients who completed at least 12 weeks of therapy achieving 'Treatment Success,' defined as a ≥50% improvement in their cumulative mCAILS score. The treatment has been well-tolerated with no adverse events reported. The study aims to enroll up to 50 patients and will provide additional data as it progresses.
- 75% of patients who completed at least 12 weeks of HyBryte™ treatment achieved 'Treatment Success'.
- The study received a $2.6 million FDA Orphan Products Development Grant.
- HyBryte™ was well-tolerated with no treatment-related adverse events reported.
- The study supports the safety and efficacy of extended HyBryte™ treatment in early-stage CTCL, potentially offering an effective option for a disease with treatments.
- None.
The preliminary outcomes of the HyBryte™ treatment in early-stage cutaneous T-cell lymphoma (CTCL) patients provide promising insights for both patients and investors. CTCL represents a niche area with significant unmet medical needs and successful treatment options are limited. The interim results show that 75% of the patients who have completed at least 12 weeks of therapy achieved substantial improvement, highlighting HyBryte™'s potential efficacy.
Investors should note that the FDA's support, manifested through a
However, it’s essential to consider that these results are interim and based on a small sample size of six patients. Thus, while the initial data is encouraging, more extensive data from the ongoing confirmatory Phase 3 trial will be important to fully ascertain the treatment’s efficacy and safety. The transition to a 'real-world' setting with potential home-use underscores the flexibility and applicability of HyBryte™, potentially making it more attractive from a commercial and patient convenience perspective.
The financial implications of the positive interim results for HyBryte™ are significant for Soligenix, Inc. From a business perspective, the company's focus on treating rare diseases like CTCL puts them in a favorable position to benefit from orphan drug pricing and market exclusivity. This can lead to lucrative revenue streams if HyBryte™ is approved and commercialized successfully.
The FDA's support through a
However, investors should be mindful of the fact that this is an interim update. The final outcomes, especially from the upcoming Phase 3 placebo-controlled study, will be pivotal in determining the actual market potential of HyBryte™. Additionally, the financial projections should consider the costs involved in bringing the therapy to market, including the confirmatory trials and potential commercialization expenses.
Study Supported by
"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
"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
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
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 (
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
The third (optional) treatment cycle (Cycle 3) was focused on safety and all patients could elect to receive HyBryte™ treatment of all their lesions. Of note,
Overall safety of HyBryte™ is a critical attribute of this treatment and was monitored throughout the three treatment cycles (Cycles 1, 2 and 3) and the 6-month follow-up period. HyBryte's™ mechanism of action is not associated with DNA damage, making it a safer alternative than currently available therapies, all of which are associated with significant, and sometimes fatal, side effects. Predominantly these include the risk of melanoma and other malignancies, as well as the risk of significant skin damage and premature skin aging. Currently available treatments are only approved in the context of previous treatment failure with other modalities and there is no approved front-line therapy available. Within this landscape, treatment of CTCL is strongly motivated by the safety risk of each product. HyBryte™ potentially represents the safest available efficacious treatment for CTCL. With very limited systemic absorption, a compound that is not mutagenic and a light source that is not carcinogenic, there is no evidence to date of any potential safety issues.
Following the first Phase 3 study of HyBryte™ for the treatment of CTCL, the FDA and the EMA indicated that they would require a second successful Phase 3 trial to support marketing approval. With agreement from the EMA on the key design components, the second, confirmatory study, called FLASH2, is expected to be initiated before the end of 2024. This study is a randomized, double-blind, placebo-controlled, multicenter study that will enroll approximately 80 subjects with early-stage CTCL. The FLASH2 study replicates the double-blind, placebo-controlled design used in the first successful Phase 3 FLASH study that consisted of three 6-week treatment cycles (18 weeks total), with the primary efficacy assessment occurring at the end of the initial 6-week double-blind, placebo-controlled treatment cycle (Cycle 1). However, this second study extends the double-blind, placebo-controlled assessment to 18 weeks of continuous treatment (no "between-Cycle" treatment breaks) with the primary endpoint assessment occurring at the end of the 18-week timepoint. In the first Phase 3 study, a treatment response of
In addition, the FDA awarded an Orphan Products Development grant to support the evaluation of HyBryte™ for expanded treatment in patients with early-stage CTCL, including in the home use setting. The grant, totaling
About Cutaneous T-Cell Lymphoma (CTCL)
CTCL is a class of non-Hodgkin's lymphoma (NHL), a type of cancer of the white blood cells that are an integral part of the immune system. Unlike most NHLs which generally involve B-cell lymphocytes (involved in producing antibodies), CTCL is caused by an expansion of malignant T-cell lymphocytes (involved in cell-mediated immunity) normally programmed to migrate to the skin. These malignant cells migrate to the skin where they form various lesions, typically beginning as patches and may progress to raised plaques and tumors. Mortality is related to the stage of CTCL, with median survival generally ranging from about 12 years in the early stages to only 2.5 years when the disease has advanced. There is currently no cure for CTCL. Typically, CTCL lesions are treated and regress but usually return either in the same part of the body or in new areas.
CTCL constitutes a rare group of NHLs, occurring in about
About Soligenix
Soligenix is a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need. Our Specialized BioTherapeutics business segment is developing and moving toward potential commercialization of HyBryte™ (SGX301 or synthetic hypericin sodium) as a novel photodynamic therapy utilizing safe visible light for the treatment of cutaneous T-cell lymphoma (CTCL). With successful completion of the second Phase 3 study, regulatory approvals will be sought to support potential commercialization worldwide. Development programs in this business segment also include expansion of synthetic hypericin (SGX302) into psoriasis, our first-in-class innate defense regulator (IDR) technology, dusquetide (SGX942) for the treatment of inflammatory diseases, including oral mucositis in head and neck cancer, and (SGX945) in Behçet's Disease.
Our Public Health Solutions business segment includes development programs for RiVax®, our ricin toxin vaccine candidate, as well as our vaccine programs targeting filoviruses (such as Marburg and Ebola) and CiVax™, our vaccine candidate for the prevention of COVID-19 (caused by SARS-CoV-2). The development of our vaccine programs incorporates the use of our proprietary heat stabilization platform technology, known as ThermoVax®. To date, this business segment has been supported with government grant and contract funding from the National Institute of Allergy and Infectious Diseases (NIAID), the Defense Threat Reduction Agency (DTRA) and the Biomedical Advanced Research and Development Authority (BARDA).
For further information regarding Soligenix, Inc., please visit the Company's website at https://www.soligenix.com and follow us on LinkedIn and Twitter at @Soligenix_Inc.
This press release may contain forward-looking statements that reflect Soligenix's current expectations about its future results, performance, prospects and opportunities, including but not limited to, potential market sizes, patient populations, clinical trial enrollment, the expected timing for closing the offering described herein and the intended use of proceeds therefrom. Statements that are not historical facts, such as "anticipates," "estimates," "believes," "hopes," "intends," "plans," "expects," "goal," "may," "suggest," "will," "potential," or similar expressions, are forward-looking statements. These statements are subject to a number of risks, uncertainties and other factors that could cause actual events or results in future periods to differ materially from what is expressed in, or implied by, these statements, and include the expected amount and use of proceeds from the offering and the expected closing date of the offering. Soligenix cannot assure you that it will be able to successfully develop, achieve regulatory approval for or commercialize products based on its technologies, particularly in light of the significant uncertainty inherent in developing therapeutics and vaccines against bioterror threats, conducting preclinical and clinical trials of therapeutics and vaccines, obtaining regulatory approvals and manufacturing therapeutics and vaccines, that product development and commercialization efforts will not be reduced or discontinued due to difficulties or delays in clinical trials or due to lack of progress or positive results from research and development efforts, that it will be able to successfully obtain any further funding to support product development and commercialization efforts, including grants and awards, maintain its existing grants which are subject to performance requirements, enter into any biodefense procurement contracts with the
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FAQ
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