Primary Malignant Liver Neoplasm

SARASOTA, Fla. and MIAMI, Oct. 24, 2024 (GLOBE NEWSWIRE) -- NAYA Biosciences (“NAYA”) (NASDAQ: NAYA), a life science portfolio company dedicated to bringing breakthrough treatments to patients in oncology, autoimmune diseases, and fertility, today announced an update regarding its clinic trial plans. The Company received regulatory approval from the Israeli Ministry of Health in July 2024 and subsequent institutional review board clearance to initiate patient enrollment in up to 7 academic centers for its clinical trial evaluating the safety and efficacy of NY-303, its GPC3-targeting NK Engager bispecific antibody, as a monotherapy for the treatment of hepatocellular carcinoma in patients not responding to first-line immunotherapy. “With the completion of the recent merger, we can now begin our focus on planning for the initiation of clinical trials with our lead GPC3-targeting NK engager bispecific antibody, which is a significant milestone for NAYA Biosciences,” commented NAYA Biosciences President and NAYA Therapeutics CEO Dr. Daniel Teper. “We are passionate about bringing new options to patients not responding to first line immunotherapy and expect that positive Phase I safety and preliminary efficacy data will support accelerated clinical development in HCC as well as exploration in additional solid and pediatric tumors.” The Phase 1 part of the trial will consist of a dose-escalation where patients responding to treatment will continue weekly administration as long as no disease progression is observed. Key endpoints include safety, pharmacokinetics, activity markers, preliminary clinical efficacy, and time-to-progression. NAYA is planning to start patient recruitment in early 2025 at leading medical centers in Israel including Hadassah Hospital, Sheba Medical Center, and Sourasky Medical Center. The Phase 2a part of the trial is anticipated to be expanded to the United States and Europe subject to additional regulatory approvals and will evaluate NY-303 at two dose levels selected in Phase I. Key efficacy endpoints will include objective response rate and progression-free survival. “Hepatocellular carcinoma is a leading cause of cancer death worldwide with a rapidly increasing global trend,” commented NAYA Therapeutics Chief Medical Officer Dan Chiche, MD. “Currently, less than 30% of patients treated with current standard of care respond to immunotherapy (confirmed objective response) and mean progression-free survival is less than 7 months. NY-303’s dual targeting of NK cells and GPC3 is a unique, novel mechanism of action supported by scientific data showing potential to unlock non-response to checkpoint inhibitors.” About HCC Hepatocellular carcinoma (HCC) is the most common form of primary liver cancer, often developing in individuals with chronic liver conditions like hepatitis B or C, cirrhosis, and Metabolic Dysfunction-Associated SteatoHepatitis (MASH). HCC is a global health challenge, with over 900,000 new cases annually, making it the sixth most common cancer worldwide. The incidence of HCC in the United States has been steadily rising. Survival rates for HCC are often low due to late detection. The five-year survival rate for liver cancer in the United States is around 20%, but this varies widely depending on the stage at diagnosis. For early-stage HCC, where surgical resection or liver transplantation is possible, survival rates can improve dramatically, while advanced stages have far poorer outcomes. Globally, survival rates are similarly low, with wide variation based on healthcare access and screening programs. Recent advances in immunotherapy such as immune checkpoint inhibitors are offering new hope for patients with advanced HCC but new treatment approaches are still critical for improving survival and quality of life for patients. About NY-303 NY-303 is a first-in-class therapeutic candidate that leverages the engagement and activation of natural killer (NK) cells using a bispecific antibody targeting both GPC3, an oncofetal protein uniquely expressed on liver cancer cells, and NKp46, a cell surface receptor on natural killer (NK) cells that plays a central role in NK cell activation and the elimination of target cells. The unique mechanism enables NY-303 to turn the tumor form a “cold” into a ‘hot’ stage making liver cancer cells susceptible against immunotherapy to target and destroy cancer cells. This innovative approach is especially promising for patients with liver cancer, such as hepatocellular carcinoma (HCC), who face limited treatment options and poor survival rates but also for other GPC3-expressing tumors like lung squamous cell carcinoma, ovarian cancer, and pediatric cancer. Currently, liver cancer therapies include surgery, liver transplantation, targeted drugs, and immunotherapies like checkpoint inhibitors, but the prognosis remains grim, especially for advanced-stage disease. With a global five-year survival rate of around 20%, and even lower in more advanced cases, there is a critical need for more effective therapies. NY-303 represents a new opportunity for liver cancer patients by harnessing the body’s immune system to specifically attack tumor cells and engage NK-cells. Unlike traditional treatments, this therapy is designed to offer potent anti-cancer effects with a potentially safer profile, reducing the side effects often associated with chemotherapy and other conventional treatments and overcoming non-responding tumors against immunotherapies. For patients with HCC, NY-303 could significantly improve outcomes by offering a more precise, targeted approach to eradicating cancer, which is critical given the high recurrence rates and challenges in treating advanced liver cancer. As part of the next generation of immunotherapies, NY-303 may redefine treatment possibilities and offer renewed hope to those facing this difficult diagnosis. About NAYA BiosciencesNAYA Biosciences (NASDAQ: NAYA) is a life science portfolio company dedicated to bringing breakthrough treatments to patients in oncology, autoimmune diseases, and fertility. Our hub & spoke model harnesses the shared resources of a parent company and agility of lean strategic franchises, enabling efficient acquisition, development, and partnering of assets and allowing for optimized return on investment by combining scalable, profitable commercial revenues with the upside of innovative clinical-stage therapeutics. NAYA’s expanding portfolio of assets currently includes NY-303, a GPC3 and NKp46 targeting bispecific antibody for the treatment of hepatocellular carcinoma (HCC) with a unique mode of action targeting non-responders to the current immunotherapy standard of care (approximately 70% of the current treatable market) cleared to enroll patients in a Phase 1/2a monotherapy trial in early 2025, NY-338, a CD38 and NKp46 targeting bispecific antibody for the treatment of multiple myeloma and autoimmune diseases with a differentiated safety and efficacy profile, and INVOcell®, an FDA-approved fertility device which has demonstrated comparable success rates to traditional In Vitro Fertilization (IVF). Safe Harbor StatementThis release includes forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. The Company invokes the protections of the Private Securities Litigation Reform Act of 1995. All statements regarding our expected future financial position, results of operations, cash flows, financing plans, business strategies, products and services, competitive positions, growth opportunities, plans and objectives of management for future operations, as well as statements that include words such as "anticipate," "if," "believe," "plan," "estimate," "expect," "intend," "may," "could," "should," "will," and other similar expressions are forward-looking statements. All forward-looking statements involve risks, uncertainties, and contingencies, many of which are beyond our control, which may cause actual results, performance, or achievements to differ materially from anticipated results, performance, or achievements. Factors that may cause actual results to differ materially from those in the forward-looking statements include those set forth in our filings at www.sec.gov. We are under no obligation to (and expressly disclaim any such obligation to) update or alter our forward-looking statements, whether as a result of new information, future events or otherwise. NAYA Investor & Media ContactAnna Baran-DjokovicSVP, Investor Relations+1-305-615-9162anna@nayabiosciences.com

Treatment with OTX-2002 led to controlled downregulation of MYC gene expression and resulted in significant inhibition of tumor growth as a monotherapy and in combination with standard of care agents in preclinical models of HCCProof-of-concept data for OTX-2002 support potential of epigenomic controllers to effectively target and modulate expression of nearly any human gene, including historically undruggable targets Data further validate the OMEGA platform’s potential to rapidly and prospectively design, develop and advance programmable epigenomic mRNA medicines for the treatment of a broad range of diseases CAMBRIDGE, Mass., Sept. 17, 2024 (GLOBE NEWSWIRE) -- Omega Therapeutics, Inc. (Nasdaq: OMGA) (“Omega”), a clinical-stage biotechnology company pioneering the development of a new class of programmable epigenomic mRNA medicines, today announced the publication of preclinical data from studies of OTX-2002 in Nature Communications. The article highlights the potential of Omega’s approach to precision epigenomic control and the ability of OTX-2002 to controllably regulate the historically undruggable MYC gene in multiple models of hepatocellular carcinoma (HCC), the most common type of primary liver cancer. “These proof-of-concept data demonstrate that by combining cutting edge understanding of DNA architecture and epigenomic regulation with our OMEGA platform, we can rapidly and prospectively design an epigenomic controller to lay intended epigenetic marks to controllably target nearly any gene and tune its expression,” said Mahesh Karande, President and Chief Executive Officer of Omega Therapeutics. “These preclinical data show OTX-2002’s exquisitely specific engagement of c-MYC, a master oncogene that has proven challenging to regulate by other modalities, resulting in its downregulation at the mRNA and protein level and robust anti-tumor effects across multiple preclinical HCC models. We believe this publication, along with the promising preliminary clinical data generated from our ongoing MYCHELANGELO™ I trial, further validates the capabilities of the OMEGA platform to develop programmable epigenomic mRNA therapeutics to potentially address a broad spectrum of diseases.” OTX-2002 is a first-in-class, bicistronic mRNA-encoded epigenomic controller (EC) delivered via liver-targeting lipid nanoparticles that is designed to durably downregulate MYC expression pre-transcriptionally via two different epigenetic modifications at two distinct genomic loci within the MYC gene. MYC is a master transcription factor that regulates cell proliferation, differentiation and apoptosis and plays a significant role in more than 50% of all human cancers. Key Findings The publication, titled “Targeted Transcriptional Downregulation of MYC Using Epigenomic Controllers Demonstrates Antitumor Activity in Hepatocellular Carcinoma Models,” describes OTX-2002 and its effects on tumor growth in cellular and animal models of HCC. Treatment with OTX-2002 resulted in the following: Rapid reduction in both MYC mRNA and protein levels Decreased viability of multiple HCC cell linesDose-dependent reduction in tumor size and growth rate in preclinical models of HCCAntitumor activity and mechanism of action that is synergistic when combined with either tyrosine kinase inhibitors or immune checkpoint inhibitor agentsBeneficial impact on the tumor microenvironment in immune-competent mice bearing HCC tumors as evidenced by a decrease in inhibitory regulatory T cells OTX-2002 is currently under clinical evaluation in the Phase 1/2 MYCHELANGELO™ I trial in adult patients with HCC and other MYC-associated solid tumors (NCT05497453). About the OMEGA platformThe OMEGA platform leverages the Company’s deep understanding of gene regulation, genomic architecture and epigenetic mechanisms to design programmable epigenomic mRNA medicines that precisely target and modulate gene expression at the pre-transcriptional level. Combining world-class data science capabilities with rational drug design and customized delivery, the OMEGA platform enables control of fundamental epigenetic processes and reprogramming of cellular physiology to address the root cause of disease. Omega’s modular and programmable mRNA medicines, called epigenomic controllers, target specific genomic loci within insulated genomic domains with high specificity to durably tune single or multiple genes to treat and cure diseases through unprecedented precision epigenomic control. About Omega TherapeuticsOmega Therapeutics is a clinical-stage biotechnology company pioneering the development of a new class of programmable epigenomic mRNA medicines to treat or cure a broad range of diseases. By pre-transcriptionally modulating gene expression, Omega’s approach enables precision epigenomic control of nearly all human genes, including historically undruggable and difficult-to-treat targets, without altering native nucleic acid sequences. Founded in 2017 by Flagship Pioneering following breakthrough research by world-renowned experts in the field of epigenetics, Omega is led by a seasoned and accomplished leadership team with a track record of innovation and operational excellence. The Company is committed to revolutionizing genomic medicine and has a pipeline of therapeutic candidates derived from its OMEGA platform spanning oncology, regenerative medicine, and multigenic diseases including inflammatory and cardiometabolic conditions. For more information, visit omegatherapeutics.com, or follow us on X and LinkedIn. Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the versatile capabilities and power of the OMEGA platform, the potential of Omega’s approach to precision epigenomic control and the ability of OTX-2002 to controllably regulate the historically undruggable MYC gene in multiple models of HCC, the wide spectrum of possibilities to apply precision epigenomic control as a novel therapeutic modality to meaningfully address key drivers of many diseases, and the potential of the Company’s pipeline of therapeutic candidates. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: the novel technology on which our product candidates are based makes it difficult to predict the time and cost of preclinical and clinical development and subsequently obtaining regulatory approval, if at all; the substantial development and regulatory risks associated with epigenomic controllers due to the novel and unprecedented nature of this new category of medicines; our limited operating history; the incurrence of significant losses and the fact that we expect to continue to incur significant additional losses for the foreseeable future; our need for substantial additional financing; volatility in capital markets and general economic conditions; our investments in research and development efforts that further enhance the OMEGA platform, and their impact on our results; uncertainty regarding preclinical development, especially for a new class of medicines such as epigenomic controllers; potential delays in and unforeseen costs arising from our clinical trials; the fact that our product candidates may be associated with serious adverse events, undesirable side effects or have other properties that could halt their regulatory development, prevent their regulatory approval, limit their commercial potential, or result in significant negative consequences; difficulties manufacturing the novel technology on which our epigenomic controller candidates are based; our ability to adapt to rapid and significant technological change; our reliance on third parties for the manufacture of materials; our ability to successfully acquire and establish our own manufacturing facilities and infrastructure; our reliance on a limited number of suppliers for lipid excipients used in our product candidates; our ability to advance our product candidates to clinical development; and our ability to obtain, maintain, enforce and adequately protect our intellectual property rights. These and other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2024, and our other filings with the SEC, could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management's estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.

Mature results from Medivir's phase 1b / 2a open label trial of fostrox + Lenvima® confirm improved outcomes in second-line advanced liver cancer with a median time to progression (TTP) of 10.9 months1 (4.1 – 18.1). The results showed an objective response (ORR) of 24%, and a median duration of response of 7.0 months. Detailed safety update reinforces the ability to combine fostrox and Lenvima long-term, only 1 patient discontinuing fostrox due to adverse events. Medivir's fostrox (fostroxacitabine bralpamide) is the only orally-administered, liver-targeted inhibitor of DNA replication. Its unique mechanism for the treatment of liver cancer delivers the cell-killing compound selectively to tumor cells locally in the liver while minimizing harm to healthy cells. STOCKHOLM, Sept. 16, 2024 /PRNewswire/ -- Medivir AB (Nasdaq Stockholm: MVIR), a pharmaceutical company focused on developing innovative treatments for cancer in areas of high unmet medical need, today presented positive, mature data from its ongoing phase 1b / 2a study of fostroxacitabine bralpamide (fostrox) + Lenvima® in advanced liver cancer (hepatocellular carcinoma/HCC) at the ESMO (European Society of Medical Oncology) Congress in Barcelona, Spain. Today's ESMO update, poster number 986P, presented by Dr Hong Jae Chon on Monday September 16, shows promising duration of benefit with 19% of patients continuing treatment for more than a year and the longest running patient remaining on treatment for over 2 years, with sustained partial response. The patients in the study had disease control on fostrox + Lenvima independent if they benefitted from previous line of therapy, showing potential for all second-line patients to benefit from the combination. The safety and tolerability profile continues to be encouraging with no unexpected adverse events. While hematological adverse events were common, they were temporary in nature. Decreases in neutrophil & platelet counts showed a cyclic pattern with recovery before next cycle of treatment, enabling patients to remain on treatment long-term. Importantly, no patient experienced febrile neutropenia or low platelet count with bleeding and there were no fostrox-related serious adverse events. Results come despite very poor prognosis for most second-line HCC patients today, with just 5–10% responding to current standard of care treatment, and a typical TTP of only 3–4 months. Dr. Pia Baumann, Chief Medical Officer at Medivir, said: - "With three patients still remaining on study treatment, all of whom treated for more than a year, this data-set is now quite mature. At a median follow-up of 10.5 months, fostrox + Lenvima have clearly shown promise of improved outcomes beyond current alternatives for second-line liver cancer patients. Fostrox is designed to only target tumor cells locally in the liver, without harming healthy cells. It is therefore reassuring to see the tolerability profile of fostrox enabling the combination of two highly potent treatments, fostrox + Lenvima, without compromising patient safety. Patients were able to stay on treatment long-term, which evidently contributes to the extended duration of benefit and a median time to progression of 10.9 months, substantially longer than previously seen in second-line liver cancer. It is with reinforced confidence we continue our preparations for the initiation of the planned phase 2b study comparing fostrox + Lenvima with Lenvima alone in a randomized setting to confirm the benefit of the combination." Dr Hong Jae Chon, Professor at CHA Bundang Hospital in Korea, and investigator in the fostrox + Lenvima study, commented: "Treatment outcomes have improved in first-line with the use of immunotherapy combinations, resulting in more patients fit enough to receive second-line treatment. But with no treatments approved in second-line after immunotherapy, there is a significant unmet medical need for new treatments options for these patients. The phase 1b/2a data for fostrox + Lenvima show highly encouraging clinical benefits for patients, indicating that when adding fostrox to Lenvima, efficacy is better than expected from Lenvima alone. It is especially encouraging that in addition to patients experiencing benefit for an extended period of time, patients also responded to the treatment independent of outcome in previous line of therapy. I look forward to evaluating the efficacy of fostrox plus Lenvima in a randomized, controlled trial." The data are from Medivir's ongoing phase 1b/2a open-label, multi-center, dose-escalation and dose-expansion study, evaluating the safety and efficacy of fostrox in combination with Lenvima in patients for whom current first- or second-line treatment has proven ineffective or is not tolerable. HCC is the most common type of liver cancer, accounting for more than 80% of cases worldwide.2 There are approximately 660,000 patients diagnosed with HCC per year globally and current five-year survival is less than 20 percent3. Medivir will host a webcast where Dr Chon and Dr Pia Baumann will present the data and answer questions. The webcast will take place Today, September 16, at 13.45 CET, and will be streamed via a link on the website: . The poster and the presentation from the webcast will also be available on Medivir's website after the presentation. For additional information, please contact; Magnus Christensen, CFO, Medivir AB Telephone: +46 8 5468 3100 E-mail: [email protected] About fostrox Fostrox is a liver-targeted inhibitor of DNA replication that delivers the cell-killing compound selectively to the tumor while minimizing the harmful effect on normal cells. This is achieved by coupling an active chemotherapy (troxacitabine) with a prodrug tail. This design enables fostrox to be administered orally and travel directly to the liver where the active substance is released locally in the liver. With this unique mechanism, fostrox has the potential to become the first liver-targeted, orally administered drug that can help patients with various types of liver cancer. A phase 1b monotherapy study with fostrox has been completed and a phase 1b/2a combination study in HCC is ongoing where it has shown encouraging anti-cancer efficacy with a good safety and tolerability profile. About primary liver cancer Primary liver cancer is the third leading cause of cancer-related deaths worldwide. Hepatocellular carcinoma (HCC) is the most common cancer that arises in the liver and it is the fastest growing cancer in the USA. Although existing therapies for advanced HCC can extend the lives of patients, treatment benefits are insufficient and death rates remain high. There are approximately 660,000 patients diagnosed with primary liver cancer per year globally and current five-year survival is less than 20 percent2,3. HCC is a heterogeneous disease with diverse etiologies, and lacks defining mutations observed in many other cancers. This has contributed to the lack of success of molecularly targeted agents in HCC. The limited overall benefit, taken together with the poor overall prognosis for patients with intermediate and advanced HCC, results in a large unmet medical need. About Medivir Medivir develops innovative drugs with a focus on cancer where the unmet medical needs are high. The drug candidates are directed toward indication areas where available therapies are limited or missing and there are great opportunities to offer significant improvements to patients. Medivir is focusing on the development of fostroxacitabine bralpamide (fostrox), a drug candidate designed to selectively treat cancer cells in the liver and to minimize side effects. Collaborations and partnerships are important parts of Medivir's business model, and the drug development is conducted either by Medivir or in partnership. Medivir's share (ticker: MVIR) is listed on Nasdaq Stockholm's Small Cap list. . Data cut-off 19 August, 2024 Rumgay et al.,European Journal of Cancer 2022 vol.161, 108-118. Yang, J.D., Hainaut, P., Gores, G.J. et al. A global view of hepatocellular carcinoma: trends, risk, prevention and management. Nat Rev Gastroenterol Hepatol 16, 589–604 (2019). This information was brought to you by Cision The following files are available for download: SOURCE Medivir WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED