SQSTM1

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.

While the disease has progressed in all patients receiving standard of care, it did not progress in 40% of the patients receiving standard chemotherapy together with the experimental drug, Elenagen — and for half of the patients in the group, Elenagen has increased the time to disease progression by more than 2.5 times BOSTON, Sept. 9, 2022 /PRNewswire/ -- CureLab Oncology, a clinical-stage, pre-IPO biotech company, is presenting the results of its ongoing ex-US clinical trial at this year's meeting of the European Society for Medical Oncology (ESMO). The trial is assessing an experimental DNA plasmid encoding the human protein, p62 (affectionately codenamed Elenagen), at the N.N. Alexandrov National Cancer Centre, one of the leading cancer centers in Eastern Europe. Continue Reading CureLab Oncology, a clinical-stage, pre-IPO biotech company, is presenting the results of its ongoing ex-US clinical trial at this year’s meeting of the European Society for Medical Oncology (ESMO). Platinum-resistant ovarian cancer (PROC) is one of the deadliest forms of ovarian cancer and has a very poor prognosis. The FDA approved other novel treatments for PROC based on statistically significant improvements for the delay of progression of the cancer, assessed by the increasing size of a primary tumor and/or the appearance of new metastatic lesions or growth of the existing ones; i.e., progression-free survival (PFS). A statistically significant prolongation of PFS was clearly demonstrated in the trial and continues to extend as the trial continues. Summary of what CureLab Oncology is presenting at ESMO 2022 In the ex-US clinical trial, CureLab Oncology is assessing its experimental plasmid DNA therapeutic, Elenagen, for its ability to extend progression-free survival (PFS) in patients with PROC receiving standard chemotherapy, gemcitabine. For all patients in the trial receiving the standard of care only, the disease progressed during the first year of observations, with 50% of patients progressing within 2.7 months. In contrast, the disease did not progress at all for almost 40% of patients receiving the same gemcitabine in combination with weekly intramuscular injections of Elenagen. In 50% of patients receiving standard of care only, the disease has progressed within 2.7 months, while the time to progression for 50% of patients in the "chemo plus Elenagen" group has increased to 7.2 months. To date, no patient receiving Elenagen demonstrated any adverse event (AE) or significant adverse event (SAE) greater than grade 1, and all resolved quickly without impact. So far, the longest PFS in the gemcitabine + Elenagen treatment group is 27 months (and continues to grow), while the disease has progressed in all patients in the gemcitabine-only group within less than 12 months. "While our data on increasing progression-free survival are already statistically significant (P=0.01), it will take a little longer to reach a statistical significance on overall survival of the patients," said Professor Sergey Krasny, M.D., D.Sc., co-author of the ESMO presentation, and a medical director of the study. "However, so far, no single patient receiving gemcitabine alone has lived longer than 18 months, while over 50 percent of patients receiving gemcitabine and Elenagen live longer than that. Also of note is that no person who survived to live 18 months has died at this point in the study." "Our presentation at ESMO 2022 is a great step forward compared to what we presented at ESMO 2021. Now, we are certain that Elenagen is increasing progression-free survival for platinum-resistant ovarian cancer patients in the trial," said Alexander Shneider, Ph.D., CEO of CureLab Oncology. 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 animal studies and Phase I/II human trials conducted ex-US, Elenagen demonstrated promise in reversing tumor grade, changing the tumor microenvironment, and enhancing the anti-cancer effects of chemotherapy. Experimental results also indicate mitigation of chronic inflammation and stimulation of an immune response to the tumor. About CureLab Oncology CureLab Oncology Inc. is a pre-IPO, clinical-stage immuno-oncology 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.

PRINCETON, N.J., Aug. 12, 2022 /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 its recent accomplishments and financial results for the quarter ended June 30, 2022. "We have accomplished a number of important milestones thus far in 2022, with more significant milestones anticipated before year-end," stated Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. "Under our Specialized Biotherapeutics business segment, the results of our successful Phase 3 FLASH (Fluorescent Light Activated Synthetic Hypericin) study evaluating HyBryte™ (synthetic hypericin) for the treatment of cutaneous T-cell lymphoma (CTCL) were published in the prestigious JAMA Dermatology. We received agreement from the U.S. Food & Drug Administration (FDA) on an initial pediatric study plan for HyBryte™ which stipulates our intention to request a full waiver of pediatric studies upon submission of the HyBryte™ new drug application (NDA) in the fourth quarter of this year. Additionally, we are expanding into new disease indications with synthetic hypericin (SGX302) having received FDA investigational new drug (IND) clearance for a Phase 2a clinical trial in mild-to-moderate psoriasis, also expected to begin in the fourth quarter of this year. Our Public Health Solutions business segment, supported by funding from the National Institute of Allergy and Infectious Diseases, continues to achieve key objectives that have the potential to play an important role in the Biden-Harris Administration's initiatives for pandemic preparedness. Recently, we demonstrated 100% protection of non-human primates against both lethal Sudan ebolavirus and Marburg marburgvirus challenge using a bivalent, thermostabilized vaccine formulated in a single vial. This same heat stable subunit vaccine platform technology has been applied to the development of our COVID-19 vaccine, CiVax™, which has demonstrated rapid and broadly functional vaccine and booster responses, including against delta and omicron variants. We also entered into a strategic partnership with SERB Pharmaceuticals to supply our novel ricin antigen in support of their early stage ricin therapeutic development program." Dr. Schaber continued, "With approximately $20.2 million in cash, not including our non-dilutive government funding, we anticipate having the capital required to achieve our near-term milestones, including NDA filing and initiation of the Phase 2a psoriasis study, while we continue to evaluate various strategic options, including but not limited to, partnership and merger and acquisition opportunities." Soligenix Recent Accomplishments On July 27, 2022, the Company announced it had received agreement from the FDA on an initial pediatric study plan for HyBryte™ for the treatment of CTCL. To view this press release, please click here. On July 25, 2022, the Company announced it had signed a worldwide exclusive license to supply its ricin antigen to SERB Pharmaceuticals, for development of a novel therapeutic treatment against ricin toxin poisoning. To view this press release, please click here. On July 20, 2022, the Company announced that the results of its successful Phase 3 FLASH study evaluating HyBryte™ for the treatment of CTCL has been published in the Journal of the American Medical Association (JAMA) Dermatology. To view this press release, please click here. On June 28, 2022, the Company announced that the FDA had cleared the IND application for its Phase 2a clinical trial titled, "Phase 2 Study Evaluating SGX302 in the Treatment of Mild-to-Moderate Psoriasis." To view this press release, please click here. On June 23, 2022, the Company announced it had achieved 100% protection of non-human primates against lethal Marburg marburgvirus challenge using a bivalent, thermostabilized vaccine formulated in a single vial, reconstituted only with sterile water immediately prior to use. To view this press release, please click here. On June 1, 2022, the Company announced a publication describing key binding characteristics of its Innate Defense Regulator, dusquetide, to the p62 protein. To view this press release, please click here. On May 23, 2022, the Company announced the United States Patent and Trademark Office had issued a Notice of Allowance for the patent application titled "Compositions and Methods of Manufacturing Trivalent Filovirus Vaccines." To view this press release, please click here. Financial Results – Year Ended June 30, 2022 Soligenix's revenues for the quarter ended June 30, 2022 were $0.4 million as compared to $0.2 million for the quarter ended June 30, 2021. Revenues primarily included licensing revenue from the Company's strategic partnership with SERB Pharmaceuticals and payments on government contracts and grants. The Company has received government funding to support the development of: RiVax®, its ricin toxin vaccine candidate; SGX943, for treatment of emerging and/or antibiotic-resistant infectious diseases; ThermoVax®, its thermostabilization platform technology; and CiVax™, its vaccine candidate for the prevention of COVID-19. Soligenix's basic net loss was $2.4 million, or ($0.06) per share, for the quarter ended June 30, 2022, as compared to $1.9 million, or ($0.05) per share, for the three months ended June 30, 2021. The increase in net loss was primarily attributed to an increase in legal and consulting services associated with the arbitration against Emergent BioSolutions, Inc. and certain of its subsidiaries as well as an increase in research and development expenses associated with preparation of the upcoming HyBryte™ NDA filing. Research and development expenses were $2.0 million as compared to $1.9 million for the quarter ended June 30, 2022 and 2021, respectively. The increase in research and development spending for the quarter ended June 30, 2022 was primarily attributable to the increased expenses associated with preparation of the upcoming HyBryte™ NDA filing. General and administrative expenses were $1.4 million and $1.1 million for the quarter ended June 30, 2022 and 2021, respectively. This increase in general and administrative expenses is primarily attributable to an increase in legal and consulting services associated with the arbitration against Emergent BioSolutions, Inc. and certain of its subsidiaries. As of June 30, 2022, the Company's cash position was approximately $20.2 million. 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) 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 proprietary formulations of oral beclomethasone 17,21-dipropionate (BDP) for the prevention/treatment of gastrointestinal (GI) disorders characterized by severe inflammation including pediatric Crohn's disease (SGX203). Our Public Health Solutions business segment includes active development programs for RiVax®, our ricin toxin vaccine candidate, and SGX943, our therapeutic candidate for antibiotic resistant and emerging infectious disease, and 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, such as experienced with the COVID-19 outbreak. 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 1/2 proof-of-concept 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. 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, including, but not limited to, 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. Company Codes: NASDAQ-SMALL:SNGX