Encephalomyelitis, Acute Disseminated

LOS ANGELES, Oct. 15, 2024 (GLOBE NEWSWIRE) -- Trethera Corporation (“Trethera”), a clinical stage biopharmaceutical company committed to developing novel drugs targeting nucleotide metabolism for the treatment of cancer and autoimmune diseases, announces an upcoming presentation at the 7 th Annual Meeting of the Rare Neuroimmune Disorders Symposium (RNDS). Trethera’s CEO, Dr. Ken Schultz, will present research highlighting the use of Trethera’s deoxycytidine kinase (dCK) inhibitor, TRE-515, as a treatment for optic neuritis and acute disseminated encephalomyelitis (ADEM). The RNDS meeting is a specialized gathering of patients and translational science experts highlighting notable, novel and rigorous scientific discoveries made in rare neuroimmune disorders that advances understanding of the clinical care of patients with these diseases. Dr. Schultz’s presentation focuses on Trethera advancements in the treatment of optic neuritis, a rare neurologic disease which affects the optic nerve causing sudden blindness or visual impairment. The presentation also emphasizes ADEM, a severe autoimmune disease occurring mainly in 6 to 8 year-old children that can lead to loss of consciousness, coma, and death. Figure 1: PET scans showing mouse brain before (left) and after (right) disease induction (brain encircled in a dashed white line). The enzyme dCK becomes upregulated during disease. Trethera’s clinical stage and first-in-class drug, TRE-515, holds FDA Orphan Drug status for both optic neuritis and ADEM. FDA Orphan Drug designation confers substantial advantages, including a faster path to market approval, FDA assistance in designing clinical trials, exemption from the $4M drug approval application fee, and eligibility for seven years of marketing exclusivity. Should the FDA approve TRE-515 for commercial use in ADEM, Trethera would be eligible for a pediatric priority review voucher. TRE-515 is currently being evaluated in a Phase 1 dose escalation solid tumors clinical trial. Presentation Details: Presentation Title: Advancements in ADEM & optic neuritis treatment with TRE-515: A clinical stage, dual orphan drug designated, first-in-class, deoxycytidine kinase inhibitor Presenter: Kenneth A. Schultz, MD Session: Research on Rare Neuroimmune Disorders, Part I Date: Saturday, October 19, 2024; 9:00AM – 11:15AM Location: Main Ballroom, Hyatt Regency; 2334 N International Pkwy, Dallas, Texas About Trethera Trethera is a clinical stage, privately held, biopharmaceutical company dedicated to pioneering the development of novel treatments for autoimmune diseases and cancers. Founded by prominent UCLA scientists, Trethera is led by experienced management and board members. Trethera’s innovative approach to targeting nucleotide metabolism led to the development of TRE-515, an orally administered capsule twice designated by the FDA as an Orphan Drug. TRE-515 is a first-in-class clinical stage drug that inhibits deoxycytidine kinase (dCK), the rate-limiting enzyme in the nucleoside salvage pathway, one of two biosynthetic pathways that generate DNA precursors. It is believed that some forms of cancer may be preferentially dependent on the salvage pathway to support tumor growth, and certain autoimmune diseases might also respond to TRE-515 treatment. Trethera is developing TRE-515 for use as a monotherapy or in combination to precisely target a metabolic vulnerability of cancer or autoimmune diseases that will transform outcomes for patients. For more information, please visit us at trethera.com or e-mail Investor Relations at ir@trethera.com . Note on Forward-Looking Statements All statements other than statements of historical facts included in this press release that address activities, events or developments that Trethera believes or anticipates will or may occur in the future are “forward-looking statements,” which may often, but not always, be identified by the use of such words as “may,” “might,” “will,” “will likely result,” “would,” “should,” “estimate,” “plan,” “project,” “forecast,” “intend,” “expect,” “anticipate,” “believe,” “seek,” “continue,” “target” or the negative of such terms or other similar expressions. Although Trethera has a reasonable basis for the forward-looking statements contained herein, Trethera cautions that such statements are based on current expectations about future events and are subject to risks, uncertainties and factors relating to medical and scientific research, all of which are difficult to predict and many of which are beyond Trethera’s control, that may cause actual results to differ materially from those expressed or implied by the forward-looking statements in this press release. These potential risks and uncertainties include, without limitation: the extent to which development of any novel cancer therapies or therapies for autoimmune diseases succeeds; whether Trethera would obtain the necessary regulatory approvals to commence human trials or commercialize TRE-515 or any novel therapies resulting from such research; Trethera successfully implementing its growth strategy, including that relating to its disease therapies; the effects of the global Covid-19 pandemic; changes in economic conditions; competition; and risks and uncertainties applicable to the business of Trethera. The statements in this press release speak only as of the date hereof and Trethera does not undertake any obligation to update, amend or clarify these forward-looking statements whether as a result of new information, future events or otherwise. The Company intends that all forward-looking statements be subject to the safe-harbor provisions of the Private Securities Litigation Reform Act of 1995. A photo accompanying this announcement is available at

LOS ANGELES, July 16, 2024 (GLOBE NEWSWIRE) -- Trethera Corporation, a clinical stage biopharmaceutical company committed to developing novel drugs targeting nucleotide metabolism for the treatment of cancer and autoimmune diseases, announced today that it was awarded a $2 million NIH Small Business Innovation Research (SBIR) grant for preclinical studies treating acute disseminated encephalomyelitis (ADEM), a neurologic disease principally of children. Trethera’s clinical stage and first-in-class drug, TRE-515, holds the only FDA Orphan Drug designation for ADEM. ADEM is an autoimmune disease that can present with fever and difficulty walking as well as loss of consciousness and coma. Viral infections, such as influenza, mumps, or COVID, frequently precede the disease. ADEM is a rare disease, affecting 12,000 to 15,000 patients a year in the United States, with most cases occurring in 6 to 8 year-old children. The grant will advance preclinical studies to enable clinic entry for a disease where no approved therapies currently exist. TRE-515 is the only drug to receive an FDA Orphan Drug designation for the treatment of ADEM, a designation that confers substantial advantages, including FDA assistance in designing clinical trials, access to the FDA Orphan Drug Grants Program, exemption from the $4M drug approval application fee, and eligibility for seven years of marketing exclusivity. Furthermore, should the FDA approve TRE-515 for commercial use in ADEM, Trethera would be eligible for a pediatric priority review voucher. Combining the study data generated through this grant and the Orphan Drug designation, Trethera plans to collaborate with the FDA to define the most expedient clinical trial path to commercialization. “Achieving both NIH funding and FDA Orphan Drug designation confers multiple external validations of our potential to treat ADEM,” said Dr. Ken Schultz, principal investigator and Trethera CEO. “Our team is highly motivated by the potential to develop the first FDA approved treatment to save children’s lives and improve outcomes in ADEM. Our strategy of combining TRE-515 commercial development for a rare disease with ongoing efforts to treat more common diseases, such as solid tumors and Crohn’s, provides multiple FDA approval opportunities, thereby reducing overall development risk.” “Approximately half of ADEM patients recover with hospitalized intensive care. For others, ADEM can be fatal or lead to lifelong disability. Any drug that could improve these outcomes for patients would be groundbreaking. TRE-515 could significantly benefit ADEM patients beyond the available therapeutic options,” said Trethera Scientific Advisory Board member Dr. Larry Steinman. Dr. Steinman is a distinguished immunologist and pediatric neurologist at Stanford University. “The pathology of ADEM involves a severe bout of inflammation in the central nervous system that can include the brain, spinal cord, and sometimes the optic nerves. The inflammation damages myelin, the protective substance that coats nerve fibers throughout the central nervous system. No medications have been specifically approved by the FDA to treat ADEM,” said UCLA’s Dr. Peter Clark, member of the Trethera Scientific Advisory Board and grant co-investigator. Figure 1: Representative stained spinal cord sections from a mouse ADEM model. Arrows point to regions of leukocyte infiltration. A discussion summary from the independent panel of NIH scientists and physicians that reviewed Trethera’s proposal noted the “strong scientific premise and rigor…. established safety in Phase I clinical trial for solid tumors… and commercialization potential is high.” Harvard’s Research Director of Neuroimmunology and Trethera Scientific Advisory Board member, Dr. Michael Levy, noted “The clinical treatment for an ADEM patient remains complex on multiple levels. Treating a disease with a real mortality risk in a vulnerable pediatric population creates a challenging role for the attending physician especially given the lack of FDA approved therapies. I look forward to the day TRE-515 can be FDA approved to provide confidence to ADEM patients and their parents.” Sources: J Neurol. 2020 Oct;267(10); Int Care Med. 2008 Mar;34(3); Neuro. 2001 May 22;56(10) About Trethera Trethera is a clinical stage, privately held, biopharmaceutical company dedicated to pioneering the development of novel treatments for autoimmune diseases and cancers. Founded by prominent UCLA scientists, Trethera is led by experienced management and board members. Trethera's innovative approach to targeting nucleotide metabolism led to the development of TRE-515, an orally administered capsule twice designated by the FDA as an Orphan Drug. TRE-515 is a first-in-class clinical stage drug that inhibits deoxycytidine kinase (dCK), the rate-limiting enzyme in the nucleoside salvage pathway, one of two biosynthetic pathways that generate DNA precursors. It is believed that some forms of cancer may be preferentially dependent on the salvage pathway to support tumor growth, and certain autoimmune diseases might also respond to TRE-515 treatment. Trethera is developing TRE-515 for use as a monotherapy or in combination to precisely target a metabolic vulnerability of cancer or autoimmune diseases that will transform outcomes for patients. For more information, please visit us at trethera.com or e-mail Investor Relations at ir@trethera.com. Note on Forward-Looking Statements All statements other than statements of historical facts included in this press release that address activities, events or developments that Trethera believes or anticipates will or may occur in the future are “forward-looking statements,” which may often, but not always, be identified by the use of such words as "may," "might," "will," "will likely result," "would," "should," "estimate," "plan," "project," "forecast," "intend," "expect," "anticipate," "believe," "seek," "continue," "target" or the negative of such terms or other similar expressions. Although Trethera has a reasonable basis for the forward-looking statements contained herein, Trethera cautions that such statements are based on current expectations about future events and are subject to risks, uncertainties and factors relating to medical and scientific research, all of which are difficult to predict and many of which are beyond Trethera’s control, that may cause actual results to differ materially from those expressed or implied by the forward-looking statements in this press release. These potential risks and uncertainties include, without limitation: the extent to which development of any novel cancer therapies or therapies for autoimmune diseases succeeds; whether Trethera would obtain the necessary regulatory approvals to commence human trials or commercialize TRE-515 or any novel therapies resulting from such research; Trethera successfully implementing its growth strategy, including that relating to its disease therapies; the effects of the global Covid-19 pandemic; changes in economic conditions; competition; and risks and uncertainties applicable to the business of Trethera. The statements in this press release speak only as of the date hereof and Trethera does not undertake any obligation to update, amend or clarify these forward-looking statements whether as a result of new information, future events or otherwise. The Company intends that all forward-looking statements be subject to the safe-harbor provisions of the Private Securities Litigation Reform Act of 1995. The content of this press release is solely the views of its authors and does not represent the official views of the NIH. A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/bfbdd798-3243-4875-addb-25512db370d7

LOS ANGELES, Sept. 05, 2023 (GLOBE NEWSWIRE) -- Trethera Corporation, a clinical stage biopharmaceutical company committed to developing novel drugs targeting nucleotide metabolism for the treatment of cancer and autoimmune diseases, announced today the appointment of Michael Levy, MD, PhD, to its Scientific Advisory Board. Dr. Levy will have a particular focus on evaluating the clinical development of Trethera’s lead asset, TRE-515, in demyelinating autoimmune diseases. A demyelinating disease is any condition that causes damage to the protective covering (myelin sheath) that surrounds nerve fibers in the brain, spinal cord, and peripheral nervous system. When the myelin sheath is damaged, nerve impulses slow or even stop, causing neurological problems. Dr. Levy is currently an Associate Professor of Neurology at Harvard Medical School and Research Director for the Division of Neuroimmunology at Massachusetts General Hospital, where he leads a team focused on rare autoimmune diseases of the central nervous system. His efforts focus on seeking to understand the causes and to develop treatments for rare autoimmune demyelinating diseases, including optic neuritis, a condition in which inflammation of the optic nerve affects vision, and acute disseminated encephalomyelitis (ADEM), a neural inflammation that primarily impacts children. Dr. Levy will advise Trethera regarding the potential of its first-in-class compound, TRE-515, to treat optic neuritis and ADEM as well as other more common neurologic diseases such as multiple sclerosis (MS). “We are honored to have Dr. Levy join our advisory board,” said Dr. Ken Schultz, Chairman and CEO of Trethera. “He is a leading authority on rare neurologic autoimmune diseases and has expertise in both early and late-stage clinical trials. His knowledge is especially well suited and valuable given that TRE-515 has achieved FDA Orphan Drug Designations for both optic neuritis and ADEM.” “I am delighted to support Trethera as it advances TRE-515 as a treatment option for patients with optic neuritis and ADEM,” said Dr. Levy. “Supporting these efforts is especially compelling given the high unmet need. Both conditions are frequently treated with high-dose corticosteroids, which can be associated with significant side effects and incomplete symptom resolution. With no approved therapies, and a real mortality risk in a vulnerable pediatric patient population, treating ADEM remains complex on multiple levels.” Dr. Levy received his Doctor of Medicine and Philosophy (MD, PhD) from Baylor College of Medicine, completing his neurology residency training at Johns Hopkins Hospital. He also holds a Bachelor of Arts (BA) degree from the University of Pennsylvania. He is a fellow of the American Academy of Neurology. About Trethera Trethera is a clinical stage privately held biopharmaceutical company dedicated to pioneering the development of novel treatments for autoimmune diseases and cancers. Founded by prominent UCLA scientists, Trethera is led by experienced management and board members. Trethera's innovative approach to targeting nucleotide metabolism led to the development of TRE-515, an orally administered capsule twice designated by the FDA as an Orphan Drug. TRE-515 is a first-in-class clinical stage drug that inhibits deoxycytidine kinase (dCK), the rate-limiting enzyme in the nucleoside salvage pathway, one of two biosynthetic pathways that generate DNA precursors. It is believed that some forms of cancer may be preferentially dependent on the salvage pathway to support tumor growth, and certain autoimmune diseases, such as multiple sclerosis, might also respond to TRE-515 treatment. Trethera is developing TRE-515 for use as a monotherapy or in combination, to precisely target a metabolic vulnerability of cancer or autoimmune diseases that will transform outcomes for patients. For more information, please visit us at trethera.com or e-mail Investor Relations at ir@trethera.com. About TRE-515 TRE-515 is an orally delivered, once daily, therapeutic engineered to inhibit dCK, the key enzyme in the nucleoside salvage pathway. A common characteristic of tumor cells in solid malignancies and pathological immune cells in autoimmune diseases is the requirement for elevated nucleotide levels to support abnormal and accelerated cell division. In contrast, dCK activity is not required in most healthy adult human cells. Mediated by the rate limiting enzyme, dCK, the nucleoside salvage pathway may play a pivotal role in enabling the rapid cell proliferation of cancer cells and aberrant activated lymphocytes, suggesting dCK as a potential therapeutic target with expected enhanced safety. Note on Forward-Looking Statements All statements other than statements of historical facts included in this press release that address activities, events or developments that Trethera believes or anticipates will or may occur in the future are “forward-looking statements,” which may often, but not always, be identified by the use of such words as "may," "might," "will," "will likely result," "would," "should," "estimate," "plan," "project," "forecast," "intend," "expect," "anticipate," "believe," "seek," "continue," "target" or the negative of such terms or other similar expressions. Although Trethera has a reasonable basis for the forward-looking statements contained herein, Trethera cautions that such statements are based on current expectations about future events and are subject to risks, uncertainties and factors relating to medical and scientific research, all of which are difficult to predict and many of which are beyond Trethera’s control, that may cause actual results to differ materially from those expressed or implied by the forward-looking statements in this press release. These potential risks and uncertainties include, without limitation: the extent to which development of any novel cancer therapies or therapies for autoimmune diseases succeeds; whether Trethera would obtain the necessary regulatory approvals to commence human trials or commercialize TRE-515 or any novel therapies resulting from such research; Trethera successfully implementing its growth strategy, including that relating to its disease therapies; the effects of the global Covid-19 pandemic; changes in economic conditions; competition; and risks and uncertainties applicable to the business of Trethera. The statements in this press release speak only as of the date hereof and Trethera does not undertake any obligation to update, amend or clarify these forward-looking statements whether as a result of new information, future events or otherwise. The Company intends that all forward-looking statements be subject to the safe-harbor provisions of the Private Securities Litigation Reform Act of 1995.