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FARMINGDALE, N.Y., Oct. 02, 2024 (GLOBE NEWSWIRE) -- DepYmed, Inc. (“DepYmed” or the “Company”) is a clinical-stage pharmaceutical company focused on developing transformative medicines for many poorly treated diseases that may benefit significantly by targeting the protein tyrosine phosphatase (PTP) family of enzymes. Today, DepYmed announces co-founder and research advisor Nicholas Tonks, Ph.D. will present data at the 9th World Rett Syndrome Congress in Queensland, Australia being held October 2-5, 2024. Dr. Tonks’ presentation, titled, “PTP1B inhibition – a clinic-ready, mechanism-based approach to the treatment of Rett syndrome” will occur on October 3, 2024 at 4:30PM local time. In the presentation, Dr. Tonks discusses the nature of Rett syndrome as an X-linked neurological disorder caused primarily by mutations in a transcriptional regulator, methyl CpG binding protein 2 (MECP2). He then outlines the preclinical work completed for the Company’s PTP1B inhibitor, DPM-1003, which improved a wide range of Rett syndrome phenotypes in RTT mice. He concludes that these data provide validation of PTP1B as a mechanism-based, therapeutic target for Rett syndrome, suggesting DPM-1003 as a new approach to therapeutic intervention in the disease. DPM-1003 is currently poised to enter clinical development and has been cleared by the FDA for clinical testing in the United States under an IND. At this time, the Company is actively raising funds to support this clinical initiative. The 9th World Rett Syndrome Congress includes the latest in research, clinical innovations, and drugs. Seminars will be presented by world-renowned experts in Rett syndrome. The Conference is hosted by the Rett Syndrome Association of Australia, a not-for-profit organization managed by a committee of volunteers most of whom are parents of Rett syndrome children. The Association seeks to enhance the quality of life for persons with the syndrome and their families, and to be of assistance to anyone interested in the disorder. About DPM-1003 Protein tyrosine phosphatases are major players in the control of cell signaling pathways that are disrupted in many diseases, yet to date no drug modulators of these enzymes have been successfully developed. DepYmed is the first company to develop a new class of orally bioavailable drug candidates that act by allosterically inhibiting PTP1B, one of the most important PTP drug targets. One of its lead compounds has shown promising efficacy in preclinical models of Rett syndrome, and the Company hopes to initiate a phase 1 clinical trial in 2024. DPM-1003 has been granted Orphan Drug designation by the U.S. Food and Drug Administration and conditionally designated under the FDA’s Rare Pediatric Disease program. About Rett Syndrome Rett syndrome is a progressive neurodevelopmental disorder that almost exclusively affects females. Infants with Rett syndrome generally develop normally for 6 to 18 months after birth. At this point, they lose previously acquired skills (developmental regression) such as purposeful hand movements and the ability to communicate. Affected children often develop autistic-like behaviors, breathing irregularities, feeding and swallowing difficulties, growth retardation, and seizures. Most Rett syndrome cases are caused by mutations of the MECP2 gene on the X chromosome and can present with a wide range of disability ranging from mild to severe. The expression of PTP1B, DepYmed’s target in Rett syndrome, is regulated by MECP2; in turn, PTP1B controls important cell functions that are disrupted in the disease. About DepYmed, Inc. DepYmed Inc., is a New York based rare disease and cancer therapeutic development company that was founded to capitalize on the scientific discoveries of the Tonks lab at Cold Spring Harbor Laboratory in the physiological function of PTP1B and ways to modulate its role in various human diseases. DepYmed is currently developing a new class of potent, orally bioavailable small molecule allosteric inhibitors of the enzyme PTP1B as novel therapeutics for Rett Syndrome, different types of cancer, metabolic diseases, obesity, and Alzheimer’s Disease where the PTP1B target has shown promise. In addition, DepYmed has also discovered a novel class of small molecules with copper chelating properties that it is developing as potential therapeutic agents for diseases such as Wilson disease and various cancers. The company is actively developing a deep pipeline of new compounds with broad therapeutic potential in these emerging drug classes in collaboration with Cold Spring Harbor Laboratory. For more information, please visit the Company’s website at: www.depymed.com. For Media Inquiries please contact: Jules AbrahamJQA Partners, Inc.jabraham@jqapartners.com 917-885-7378 DepYmed:info@depymedinc.com

CAMBRIDGE, MA / ACCESSWIRE / April 3, 2024 / Moderna, Inc. (NASDAQ:MRNA) today announced that interim data for a first-in-human, phase 1/2, open-label, dose optimization study and extension study, evaluating the safety and efficacy of mRNA-3927, an investigational mRNA therapy for propionic acidemia (PA), has been published in Nature. "We are excited to share the first published clinical data utilizing an mRNA therapy for intracellular protein replacement," said Kyle Holen, M.D., Moderna's Senior Vice President and Head of Development, Therapeutics and Oncology. "PA is a rare, inherited metabolic disorder that results from the body's inability to process certain parts of proteins and lipids due to a specific enzyme deficiency. For people with PA, harmful amounts of toxic metabolites can build up in the body and lead to metabolic decompensation events (MDEs) and multisystemic complications. These interim data indicate early signs of potential clinical benefit with mRNA-3927, and importantly also demonstrate that mRNA-3927 has infrequent treatment-limiting side effects. I'm particularly proud of these results given that there are currently no therapeutic treatments approved for patients with this disease." The ongoing global Phase 1/2 clinical trial (ClinicalTrials.gov Identifier: NCT04159103) is a multicenter, open-label study designed to assess the safety, pharmacodynamics, and pharmacokinetics of mRNA-3927 in participants aged one year and older with genetically confirmed PA. Data from the study was previously presented at the 2023 American Society of Gene & Cell Therapy (ASGCT) Annual Meeting. As of the latest data cut (May 31, 2023), 16 participants received investigational therapy mRNA-3927 across five cohorts as part of the dose optimization and extension studies. Of these, 12 participants completed the study and enrolled in the open-label extension study. Over 340 intravenous doses were administered, accounting for over 15 person-years of treatment. To date, mRNA-3927 has been well tolerated in participants at the doses administered, with no dose-limiting toxicities observed. Fifteen participants reported treatment-emergent adverse events (TEAEs), while nine participants experienced drug-related TEAEs. Serious adverse events (SAEs) were reported in eight participants. Most SAEs were related to PA and unrelated to mRNA-3927. Five participants had mild infusion-related (IRR) TEAEs; however, most events occurred at the first doses. A reduction in the number of MDEs was observed. The relative risk for MDEs was reduced by 70% during the treatment period of the dose optimization study. No MDEs were observed with higher doses of mRNA-3927. Additional participants are now being enrolled into the study as part of a dose expansion phase to allow for further characterization of the efficacy, safety, and pharmacodynamic activity of mRNA-3927. About Propionic Acidemia Propionic acidemia is a rare, serious, inherited metabolic disorder with significant morbidity and mortality, affecting one in 100,000-150,000 individuals worldwide. PA is caused by pathogenic variants in the propionyl-coenzyme A carboxylase (PCC) α or β subunits (PCCA and PCCB genes, respectively), leading to PCC deficiency and subsequent accumulation of toxic metabolites. PA is characterized by recurrent life-threatening MDEs and multisystemic complications. Some of the most common multisystemic complications include neurological manifestations, cardiomyopathy, arrhythmia, growth retardation, recurrent pancreatitis, bone marrow suppression, and predisposition to infection. Long-term, insults by toxic metabolites cause multiorgan complications and are correlated with recurrent MDEs. Currently, there are no effective therapies for PA that target the underlying root cause of the disease. About mRNA-3927 mRNA-3927 is a novel, IV-administered, lipid nanoparticle (LNP)-encapsulated dual investigational mRNA therapy that encodes for PCCA and PCCB subunit proteins to restore functional PCC enzyme activity in the liver. By encoding for the PCC enzyme, mRNA-3927 has the potential to replace a missing or dysfunctional enzyme. About Moderna Moderna is a leader in the creation of the field of mRNA medicine. Through the advancement of mRNA technology, Moderna is reimagining how medicines are made and transforming how we treat and prevent disease for everyone. By working at the intersection of science, technology and health for more than a decade, the company has developed medicines at unprecedented speed and efficiency, including one of the earliest and most effective COVID-19 vaccines. Moderna's mRNA platform has enabled the development of therapeutics and vaccines for infectious diseases, immuno-oncology, rare diseases and autoimmune diseases. With a unique culture and a global team driven by the Moderna values and mindsets to responsibly change the future of human health, Moderna strives to deliver the greatest possible impact to people through mRNA medicines. For more information about Moderna, please visit modernatx.com and connect with us on X (formerly Twitter), Facebook, Instagram, YouTube and LinkedIn. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding: the potential clinical benefits of mRNA-3927 for the treatment of propionic acidemia; the safety and tolerability of mRNA-3927; and plans for future clinical studies of mRNA-3927. In some cases, forward-looking statements can be identified by terminology such as "will," "may," "should," "could," "expects," "intends," "plans," "aims," "anticipates," "believes," "estimates," "predicts," "potential," "continue," or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. The forward-looking statements in this press release are neither promises nor guarantees, and you should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Moderna's control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements. These risks, uncertainties, and other factors include, among others, those risks and uncertainties described under the heading "Risk Factors" in Moderna's Annual Report on Form 10-K for the fiscal year ended December 31, 2023, filed with the U.S. Securities and Exchange Commission (SEC), and in subsequent filings made by Moderna with the SEC, which are available on the SEC's website at . Except as required by law, Moderna disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Moderna's current expectations and speak only as of the date of this press release. Moderna Contacts Media: Elise Meyer Senior Director, Corporate Communications +1 617-852-7041 Elise.Meyer@modernatx.com Investors: Lavina Talukdar Senior Vice President & Head of Investor Relations +1 617-209-5834 Lavina.Talukdar@modernatx.com SOURCE: Moderna, Inc. View the original press release on accesswire.com

FARMINGDALE, N.Y., March 19, 2024 (GLOBE NEWSWIRE) -- Depymed, Inc. (“DepYmed” or the “Company”) is a clinical-stage pharmaceutical company focused on developing transformative medicines for many poorly treated diseases that may benefit significantly by targeting the protein tyrosine phosphatase (PTP) family of enzymes. Today, DepYmed announces it has received clearance from the U.S. Food and Drug Administration to initiate its phase 1 clinical trial for DPM-1003 for the treatment of Rett Syndrome. “This is a foundational achievement for DepYmed. It allows us to transition into a clinical-stage company with a transformational new drug candidate that has the possibility to provide significant benefit for patients,” said Andreas Grill, DepYmed’s President and CEO. “As an IND for the first drug of its kind, we believe this milestone opens a pathway to the development of important new treatments for a wide range of diseases where inhibiting PTP enzymes holds promise. This includes Rett Syndrome, cancer, metabolic disease and obesity, and Alzheimer’s Disease. We look forward to beginning to assess the safety and tolerability of DPM-1003 and continue our development of this compound for Rett Syndrome and our other ongoing programs.” About DPM-1003 Protein tyrosine phosphatases are major players in the control of cell signaling pathways that are disrupted in many diseases, yet to date no drug modulators of these enzymes have been successfully developed. DepYmed is the first company to develop a new class of orally bioavailable drug candidates that act by inhibiting PTP1B, one of the most important PTP drug targets. One of its lead compounds has shown promising efficacy in preclinical models of Rett syndrome, and the Company hopes to initiate a phase 1 clinical trial in 2024. DPM-1003 has been granted Orphan Drug designation by the U.S. Food and Drug Administration and conditionally designated under the FDA’s Rare Pediatric Disease program. About Rett Syndrome Rett syndrome is a progressive neurodevelopmental disorder that almost exclusively affects females. Infants with Rett syndrome generally develop normally for 6 to 18 months after birth. At this point, they lose previously acquired skills (developmental regression) such as purposeful hand movements and the ability to communicate. Affected children often develop autistic-like behaviors, breathing irregularities, feeding and swallowing difficulties, growth retardation, and seizures. Most Rett syndrome cases are caused by mutations of the MECP2 gene on the X chromosome and can present with a wide range of disability ranging from mild to severe. The expression of PTP1B, DepYmed’s target in Rett syndrome, is regulated by MECP2; in turn, PTP1B controls important cell functions that are disrupted in the disease. About DepYmed, Inc. DepYmed, Inc. is a New York-based rare disease and cancer therapeutic development company that was founded to capitalize on the scientific discoveries of the Tonks lab at Cold Spring Harbor Laboratory in the physiological function of PTP1B and ways to modulate its role in various human diseases. DepYmed is currently developing a new class of potent, orally bioavailable small molecule inhibitors of the enzyme PTP1B as novel therapeutics for Rett Syndrome, different types of cancer, metabolic diseases, obesity, and Alzheimer’s Disease where the PTP1B target has shown promise. In addition, DepYmed has also discovered a novel class of small molecules with copper chelating properties that it is developing as potential therapeutic agents for diseases such as Wilson disease and various cancers. The company is actively developing a deep pipeline of new compounds with broad therapeutic potential in these emerging drug classes in collaboration with Cold Spring Harbor Laboratory. For more information, please visit the Company’s website at: . For Media Inquiries please contact: Jules Abraham JQA Partners, Inc. jabraham@jqapartners.com 917-885-7378 DepYmed: info@depymedinc.com