SQSTM1

Novel p62-based treatment accelerates wound healing and reduces complications BOSTON, June 27, 2024 /PRNewswire/ -- CureLab Oncology, a clinical-stage biotech company, has filed a patent application with worldwide potential for a revolutionary wound-healing therapy based on its p62/SQSTM1 DNA plasmid. This innovative approach, leveraging CureLab's chronic inflammation therapeutic, Elenagen™, addresses a critical unmet need in the treatment of both acute and chronic wounds. The therapy offers the potential to significantly improve patient outcomes, reduce healthcare costs, and transform the standard of care in this field. The patented therapy leverages the unique properties of the p62 plasmid to accelerate the natural healing process, reduce inflammation, and prevent complications such as sepsis, infection, tissue necrosis, and scarring. Preclinical studies in animal models have demonstrated the therapy's effectiveness in a variety of wound types, including cuts, burns, diabetic ulcers, and pressure sores. "This patent application is a major milestone for CureLab Oncology and underscores our commitment to developing innovative therapies that address significant medical challenges," said Alexander Shneider, Ph.D., CEO of CureLab Oncology. "Chronic and acute wounds affect millions of people worldwide, and our p62-based therapy has the potential to revolutionize how we treat these debilitating conditions." The global wound care market is projected to reach USD 37.08 billion by 2032, growing at a compound annual growth rate (CAGR) of 7.5% from 2024 to 2032. This growth is fueled by the rising prevalence of chronic diseases like diabetes, an aging population, and advancements in wound care technologies. "Our patenting strategy for this wound-healing product mirrors our successful approach with other therapies," said Ilya Lapshin, general counsel for CureLab Oncology. "By initially filing a provisional patent application in the USA and subsequently seeking worldwide protection, we aim to secure comprehensive intellectual property rights for this groundbreaking innovation." About Elenagen CureLab's lead investigational compound is code-named Elenagen, an experimental DNA therapy that consists of a circular piece of DNA called a plasmid that includes a gene for a human protein called p62/SQSTM1. In clinical studies conducted ex-US, Elenagen demonstrated desirable safety profile and statistically significant clinical benefit for cancer patients enhancing the anti-cancer effects of chemotherapy. Experimental results also demonstrate mitigation of chronic inflammation, anti-aging effects, and stimulation of an immune response to the tumor. About CureLab Oncology CureLab Oncology Inc. is a pre-IPO, clinical-stage biotech company headquartered in the greater Boston area, Massachusetts. CureLab is dedicated to advancing new and safer therapeutics for solid tumors and other oncology and inflammatory indications. To learn more, visit curelab.com. Media contact: Tim Cox, ZingPR, [email protected] SOURCE CureLab Oncology Inc.

Pipeline Expansion of Novel Innate Defense Regulator Technology PRINCETON, N.J., Nov. 30, 2023 /PRNewswire/ -- 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 today that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for a Phase 2a clinical trial entitled, "Pilot Study of SGX945 (Dusquetide) in the Treatment of Aphthous Ulcers in Behçet's Disease." The study is designed to evaluate the safety and efficacy of SGX945 (dusquetide) and is expected to begin patient enrollment in the second half of 2024. "We are pleased to have received FDA clearance on our SGX945 Phase 2 pilot trial in aphthous ulcers of Behçet's disease," stated Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. "Our previous studies with dusquetide in oral mucositis have clearly validated the biologic activity in aphthous ulcers induced by chemotherapy and radiation. Given the role of the innate immune system in ulcers associated with Behçet's Disease, and the unmet medical need particularly for more severe ulcers such as genital and leg ulcers, we believe that dusquetide may offer significant relief to patients. We are excited to expand dusquetide's development into different innate immune-related inflammatory conditions such as Behçet's disease, as a component of our long-term strategy to enhance the value of this unique compound. Behçet's disease is an unmet medical need, with up to 18,000 people in the U.S., 80,000 in Europe, 350,000 people in Turkey and as many as 1 million people worldwide affected by this incurable disease. Given our promising results with aphthous ulcers in oral mucositis, we are hopeful dusquetide will have a role to play in helping underserved patients suffering from this difficult to treat and chronic disease." Under this IND, the pilot clinical trial of SGX945 will be an open-label study that will enroll approximately 25 patients age 18 years or older with mild to moderate Behçet's disease active oral and/or genital ulcers. Patients will receive SGX945 as a twice weekly 4-minute intravenous (IV) infusion for 4 weeks. Efficacy endpoints will include the extent of lesion clearance, timeline to lesion clearance, and patient reported quality of life indices. About Dusquetide Dusquetide (the active ingredient in SGX945 and SGX942 in development for oral mucositis) is an innate defense regulator (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 have been demonstrated in numerous animal disease models including mucositis, colitis, macrophage activation syndrome (MAS) as well as bacterial infections, including melioidosis. In addition, potential anti-tumor activity has been demonstrated in multiple in vitro and in vivo xenograft studies. Dusquetide has demonstrated safety and tolerability in a Phase 1 clinical study in 84 healthy human volunteers. In Phase 2 and 3 clinical studies with SGX942 in over 350 subjects with oral mucositis due to chemoradiation therapy for head and neck cancer, positive efficacy results were demonstrated, including potential long-term ancillary benefits. 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. About Behçet's Disease Behçet's Disease (BD) is commonly known as an inflammatory disorder of the blood vessels (vasculitis). Often first diagnosed in young adults, it's effects and severity will wax and wane over time. Major signs and symptoms usually include mouth sores (approximately 95% of patients), skin rashes and lesions (approximately 50% of patients), genital sores (approximately 50% of patients), leg ulcers (approximately 40% of patients) and eye inflammation (approximately 15% of patients). It is a painful disease, directly impacting the patient's quality of life and ability to productively engage in life activities, including work. BD is thought to be an auto-immune disease with both genetic and environmental factors. It is most common along the "Silk Road" in the Middle East and East Asia, including Turkey, Iran, Japan and China. There are approximately 18,000 known cases of BD in the U.S. and 80,000 in Europe. There are as many as 1,000,000 people worldwide living with BD. There is no cure for BD, rather treatments are prescribed to manage symptoms. Treatments may include both maintenance therapies and those specifically addressing flares (e.g., mouth ulcers, genital ulcers and leg ulcers). Corticosteroids are generally applied topically to sores and as eyedrops and may also be given systemically to reduce inflammation. Although used frequently, they have limited efficacy over the long-term and have significant side effects that become more concerning with more chronic use. Genital ulcers are often associated with significant genital scarring while leg ulcers can result in a post-thrombotic syndrome. Other treatments for BD flares involve suppressing the immune system with drugs (e.g., cyclosporine or cyclophosphamide). These drugs come with a higher risk of infection, liver and kidney problems, low blood counts and high blood pressure. Finally, anti-inflammatory drugs are also used, including anti-TNF medications. The only approved drug in BD is apremilast, which is used as a maintenance therapy to prevent formation of oral ulcers. Unfortunately, apremilast is associated with both high cost and side effects including diarrhea, nausea, upper respiratory tract infection and headache. About Soligenix, Inc. 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 a successful Phase 3 study completed, regulatory approval is being sought and commercialization activities for this product candidate are being advanced initially in the U.S. 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. The Company also is developing proprietary formulations of oral beclomethasone 17,21-dipropionate (BDP) for the prevention/treatment of gastrointestinal (GI) disorders characterized by severe inflammation such as pediatric Crohn's disease (SGX203). Our Public Health Solutions business segment includes active 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 and follow us on LinkedIn and Twitter at @Soligenix_Inc. This press release may contain forward-looking statements that reflect Soligenix, Inc.'s current expectations about its future results, performance, prospects and opportunities, including but not limited to, potential market sizes, patient populations and clinical trial enrollment. 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 U.S. Government or other countries, that it will be able to compete with larger and better financed competitors in the biotechnology industry, that changes in health care practice, third party reimbursement limitations and Federal and/or state health care reform initiatives will not negatively affect its business, or that the U.S. Congress may not pass any legislation that would provide additional funding for the Project BioShield program. In addition, there can be no assurance as to the timing or success of any of its clinical/preclinical trials. Despite the statistically significant result achieved in the HyBryte™ (SGX301) Phase 3 clinical trial for the treatment of cutaneous T-cell lymphoma, there can be no assurance that a marketing authorization from the FDA or EMA will be successful. Notwithstanding the result in the HyBryte™ (SGX301) Phase 3 clinical trial for the treatment of cutaneous T-cell lymphoma and the Phase 2a clinical trial of SGX302 for the treatment of psoriasis, there can be no assurance as to the timing or success of the clinical trials of SGX302 for the treatment of psoriasis. Despite the positive efficacy results demonstrated in the Phase 2 and 3 clinical studies of SGX942 for the treatment of oral mucositis due to chemoradiation therapy for head and neck cancer, there can be no assurance as to the timing or success of the clinical trials of SGX945 for the treatment of Behçet's Disease. Further, there can be no assurance that RiVax® will qualify for a biodefense Priority Review Voucher (PRV) or that the prior sales of PRVs will be indicative of any potential sales price for a PRV for RiVax®. Also, no assurance can be provided that the Company will receive or continue to receive non-dilutive government funding from grants and contracts that have been or may be awarded or for which the Company will apply in the future. These and other risk factors are described from time to time in filings with the Securities and Exchange Commission (the "SEC"), including, but not limited to, the Company's preliminary prospectus (Registration No. 333-271049) filed with the SEC on May 4, 2023, and Soligenix's reports on Forms 10-Q and 10-K. Unless required by law, Soligenix assumes no obligation to update or revise any forward-looking statements as a result of new information or future events. SOURCE SOLIGENIX, INC.

In a preprint paper published on MedRxiv, a team of scientists at CureLab Oncology and their international collaborators have demonstrated a clear clinical benefit of a new biological agent against the deadliest form of ovarian cancer. The results were derived from a clinical study that evaluated the safety and efficacy of Elenagen, a novel anticancer therapeutic (plasmid DNA encoding p62/SQSTM1 protein), as an adjuvant to platinum-based chemotherapy, gemcitabine, in patients with advanced platinum-resistant ovarian cancer BOSTON, Nov. 30, 2023 /PRNewswire/ -- Major scientific breakthroughs are sometimes the result of a combination of serendipity and human error. One such example is platinum-based chemotherapy, which was discovered by accident. Using a special device, researchers were testing how cancer cells responded to electricity. During the study, they observed that the cancer cells were indeed affected — but it was only later that they realized that they had forgotten to plug in the device. What they had stumbled upon is that the cancer cells were in fact responding to the platinum electrodes and platinum ions in the media. Today, the majority of newly diagnosed ovarian cancer patients are initially treated with platinum-based chemotherapy (such as cisplatin and carboplatin), a therapy that keeps many of them free of disease progression over a prolonged period. Sooner or later, however, most patients stop responding to platinum therapies, joining those who had a platinum-resistant form of ovarian cancer from the very beginning. Treatment options for these patients are limited, which makes it especially important to improve the efficacy of platinum-based chemotherapies. "Almost 1 in 80 women will be diagnosed with ovarian cancer during her lifetime, and almost 1 in 110 will die of it," said Dr. Alex Shneider, founder and CEO of CureLab Oncology. "I doubt that we can significantly change these numbers anytime soon. But these numbers would be less tragic if we could say to a diagnosed woman: 'We can give you an extended period of a high-quality life.' Our team has been focused on giving patients diagnosed with platinum-resistant ovarian cancer additional life — a life full of energy and free of pain." One of the commonly used treatments for platinum-resistant ovarian cancer (PROC) patients is gemcitabine. Originally, it was thought that gemcitabine 'tricks' rapidly dividing cancer cells because the molecule looks very similar to the natural nucleotides required for cell propagation while making cell division impossible. Later, it turned out that gemcitabine also works through a patient's immune system, and possibly other mechanisms that are yet to be discovered. In a study published on MedRxiv, a team of scientists from CureLab Oncology, the N.N. Alexandrov National Cancer Center, and Minsk City Oncology Center set out to establish whether an investigational new drug, Elenagen, can safely enhance gemcitabine PROC treatment. Elenagen is a plasmid, a circular DNA that encodes a gene known as p62/SQSTM1 (p62). Originally, Dr. Shneider and his team decided to inject this plasmid into cancer patients because elevated levels of p62 were reported for cancer cells, distinguishing them from normal cells. Could injecting p62-encoding DNA into a muscle could result in a specific immune response against overly abundant p62, thereby selectively eliminating cancer cells? In other words, a classic vaccine mechanism of action. However, to their great surprise, CureLab researchers and their academic collaborators observed Elenagen effects that could not be explained solely by their original hypothesis. For example, in response to an intramuscular injection of Elenagen, tumors changed their internal structure, permitting immune cells to enter the tumors more efficiently while making it harder for metastatic cells to exit. Importantly, Elenagen also reduced systemic chronic inflammation in the organism. Chronic inflammation plays a detrimental role in many diseases, including cancer. "When we began our study, we were concerned that the mechanism of action of this novel drug is not clear," said co-author of the study, Professor Sergey Krasny, M.D., Ph.D., Sc.D. "In fact, it may be years or even decades before it is entirely understood. Then we thought, what if our predecessors had waited for the mechanism of action of penicillin to be entirely clear in the lab before testing in the clinic? We were also compelled by the strong published evidence of Elenagen's effects obtained during Phase I clinical studies and in laboratory dogs." In a clinical trial, 40 PROC patients were randomly divided into two groups. One received standard gemcitabine regimen while another received a combination of the same gemcitabine treatment coupled with weekly injections of Elenagen. No serious adverse events (SAE) due to Elenagen were reported. Note: SAE have often observed during immune oncology studies, which sets Elenagen apart. In addition to safety, the study used the standard primary endpoint of progression-free survival (PFS). PFS is the duration of time between the moment when a patient is enrolled in a study and the moment of relapse of a primary tumor and/or metastatic lesions, as confirmed by radiographic scans. As in other published reports, the average PFS for gemcitabine-treated group was just 2.8 months. However, the group receiving the chemotherapy supplemented with Elenagen demonstrated a PFS of 7.2 months, a statistically significant increase. Importantly, 9 out of 20 patients receiving Elenagen did not demonstrate any disease progression, with a longest recorded observation of 24 months. At the same time, all patients receiving only the chemotherapy had disease progression within less than one year. Although the fact that 45% of patients in the Elenagen-treated group did not show a disease progression is itself remarkable, additional truly counterintuitive data appeared through a subgroup analysis. CA 125 is a protein circulating in blood and is used by oncologists as an ovarian oncomarker. The higher the level of CA 125, the worse the prognosis. One might expect that Elenagen + gemcitabine treatment would show reduced benefits for patients with elevated CA 125, since their disease is more severe and is thus harder to treat. However, compared to the control group, the patients with elevated CA 125 patients were the most responsive to Elenagen regimens. Another prognosis factor for a patient is the number of platinum chemotherapy courses received prior to becoming platinum resistant. Indeed, if disease progresses after just one course of platinum treatment, it is often more severe and harder to treat than in cases where the patient remains progression-free over multiple courses. Also of note: the most obvious effect of Elenagen treatment was observed in patients whose disease progressed after just one course of platinum chemotherapy. Encouraged by these ex-US results, CureLab Oncology has submitted its pre-IND package to the FDA. The company plans to expand the study in the USA as Phase II/III study. The principal investigator for the study will be Prof. Bhavana Pothuri, MD of NYU. The same combination of Elenagen with gemcitabine will be explored. Meanwhile, non-pivotal studies will continue abroad to test if Elenagen also enhances anti-PROC effects of paclitaxel, doxil, and other treatments. About CureLab Oncology CureLab Oncology Inc. is a pre-IPO, clinical-stage biotech company headquartered in the greater Boston area, Massachusetts. CureLab is dedicated to advancing new and safer therapeutics for solid tumors and other oncology and inflammatory indications. CureLab's lead investigational compound is code-named Elenagen, an experimental DNA therapy that consists of a circular piece of DNA called a plasmid that includes a gene for a human protein called p62/SQSTM1. To learn more, visit curelab.com. Media contact: Tim Cox | ZingPR | [email protected] SOURCE CureLab Oncology Inc.