Growth retardation

The FDA has granted orphan drug designation for IBT’s product IBP-1016 for gastroschisis. Infants suffering from gastroschisis suffer from risks of growth retardation, sepsis and NEC which leads to increased mortality. Hospital stays for these infants are commonly long and costly. Orphan drugs are either drugs or biologics intended for the treatment, diagnosis or prevention of rare diseases or disorders affecting less than 200 000 patients in the USA per year. An orphan drug designation qualifies the company applying for it to receive certain benefits from the US government, such as tax reductions and long term market exclusivity, in exchange for developing the drug. The approval does not change the standard regulatory requirements and processes for obtaining marketing approval for a product. Consequently, all aspects of the development must be investigated, including the clinical safety and efficacy documentation required for a market authorisation. “This is the first time the FDA has granted orphan drug designation for gastroschisis. This is important as there are no treatments available for this serious disease. We are now investigating the optimum development pathway to obtain market authorization”, says Staffan Strömberg, Chief Executive Officer, IBT. About Infant Bacterial Therapeutics AB Infant Bacterial Therapeutics AB (“IBT”) is a public company domiciled in Stockholm. The company’s Class B shares are since September 10, 2018, listed on Nasdaq Stockholm (IBT B). IBT is a pharmaceutical company whose purpose is to develop and market drugs targeting diseases affecting prematurely born infants or caused by antibiotic-resistant bacteria. IBT’s main focus is on its drug candidate IBP-9414, whose development program is designed to demonstrate a reduced incidence of necrotizing enterocolitis (NEC) and whether prematurely born infants achieve improved sustained feeding tolerance (SFT) when treated with the active substance Lactobacillus reuteri, a bacterial strain naturally found in human breast milk. IBP-9414 is currently in an ongoing registration-enabling pivotal Phase III study and is the company’s most advanced development project. The portfolio includes drug candidates, IBP-1016, IBP-1118, and IBP-1122. IBP-1016 is for the treatment of gastroschisis, a life-threatening and rare condition where the child is born with externalized abdominal organs. IBP-1118 aims to prevent ROP (retinopathy of prematurity), a leading cause of blindness in premature infants, while IBP-1122 aims to eliminate vancomycin-resistant enterococci (VRE), which cause antibiotic-resistant hospital acquired infections. By developing these drugs, IBT has the opportunity to address medical needs where no available treatments currently exist. The content above comes from the network. if any infringement, please contact us to modify.

Data were presented to demonstrate that DPM-1003 provides a mechanism-based approach to the treatment of Rett Syndrome and is poised to enter clinical development in 2024FARMINGDALE, N.Y., June 15, 2023 (GLOBE NEWSWIRE) -- DepYmed, Inc. (“DepYmed” or the “Company”) is a preclinical stage pharmaceutical company focused on developing transformative treatments for rare diseases and oncology that target the protein tyrosine phosphatase (PTP) family of enzymes. Today DepYmed announces the presentation of a development update for its small molecule PTP1B inhibitor program and its lead clinical candidate, DPM-1003, at the 2023 International Rett Syndrome Foundation (IRSF) Rett Syndrome Scientific Meeting. The Company presented a poster outlining the development of DPM-1003 and Dr. Nicholas Tonks, DepYmed’s founding scientist, delivered a podium presentation demonstrating the potential of this new treatment of Rett Syndrome. The conference was held June 5-7, 2023 at the Nashville Marriott at Vanderbilt University in Nashville, Tenn. The data illustrated the Company’s mechanism-based approach and provided an overview of DPM-1003 preclinical efficacy and safety moving the compound through IND-enabling studies to initiate clinical studies in 2024. The data demonstrate that DPM-1003 is a potent, specific inhibitor of PTP1B and a potential once-daily orally administered drug to treat Rett Syndrome. “We believe these data, which were presented for the first time in a public forum, validate our continued development of DPM-1003 toward treating Rett Syndrome,” said Andreas Grill, DepYmed’s President and CEO. “We are diligently continuing our IND-enabling studies and intend to enter clinical development under an IND submission to the FDA this year. We will continue to provide relevant updates on our progress.” About IRSF In 1983, a small, dedicated group of parents whose children had Rett syndrome formed the first non-profit to focus exclusively on Rett syndrome – the International Rett Syndrome Association (IRSA). In 2007, IRSA and Rett Syndrome Research Foundation (RSRF) consolidated resources to better serve families and maximize research investments toward a cure. The International Rett Syndrome Foundation (IRSF) emerged with a mission to accelerate research and empower families that builds upon these foundations’ pioneering work toward care and cure. empower families with information, knowledge, and connectivity. Today, the International Rett Syndrome Foundation continues its almost 40-year commitment to creating a world without Rett. It remains focused on accelerating research to treat and cure Rett syndrome as it seeks to empower families with information, knowledge, and connectivity. About DPM-1003 Protein tyrosine phosphatases are major players in the control of cell signaling pathways that are disrupted in many diseases, yet no drug modulators of these enzymes have been successfully developed to date. 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 and different types of cancer. 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 such diseases 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

FARMINGDALE, New York, May 31, 2023 (GLOBE NEWSWIRE) -- DepYmed, Inc. (“DepYmed” or the “Company”) is a preclinical stage pharmaceutical company focused on developing transformative treatments for rare diseases and oncology that target the protein tyrosine phosphatase (PTP) family of enzymes. Today DepYmed announces it will be presenting two abstracts on its small molecule PTP1B inhibitor program at the 2023 International Rett Syndrome Foundation (IRSF) Rett Syndrome Scientific Meeting. The conference will be held June 5-7, 2023 at the Nashville Marriott at Vanderbilt University in Nashville, Tenn. “This is a unique opportunity for us to present our mechanism-based approach to treating Rett Syndrome and to report progress on our lead clinical candidate at this important scientific forum. We believe our data continue to validate the potential of our compound for this devastating disease,” said Andreas Grill, DepYmed’s President and CEO. “We look forward to sharing these early data at the conference and with the wider scientific community.” About IRSFIn 1983, a small, dedicated group of parents whose children had Rett syndrome formed the first non-profit to focus exclusively on Rett syndrome – the International Rett Syndrome Association (IRSA). In 2007, IRSA and Rett Syndrome Research Foundation (RSRF) consolidated resources to better serve families and maximize research investments toward a cure. The International Rett Syndrome Foundation (IRSF) emerged with a mission to accelerate research and empower families that builds upon these foundations’ pioneering work toward care and cure. empower families with information, knowledge, and connectivity. Today, the International Rett Syndrome Foundation continues its almost 40-year commitment to creating a world without Rett. It remains focused on accelerating research to treat and cure Rett syndrome as it seeks to empower families with information, knowledge, and connectivity. About DPM-1003Protein tyrosine phosphatases are major players in the control of cell signaling pathways that are disrupted in many diseases, yet no drug modulators of these enzymes have been successfully developed to date. 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 SyndromeRett 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 and different types of cancer. 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 such diseases 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