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New Trial to Study Progerinin Treatment for Ultra-Rare Condition BUSAN, KOREA; PEABODY, MA; and GERMANTOWN, MD / ACCESSWIRE / October 2, 2024 / The United States Food and Drug Administration (FDA) has authorized the start of patient enrollment for a Phase 2a clinical trial of Progerinin, an investigational drug being developed for the treatment of Progeria. Advancing the development of Progerinin could potentially provide an additional treatment for this fatal disease. PRG Science & Technology logo Hutchinson-Gilford Progeria Syndrome (“Progeria”) is an ultra-rare, genetic, “rapid-aging” condition affecting approximately 1 in 18 million people. Without treatment, children with Progeria die of heart disease at an average age of 14.5 years old. Progeria is caused by a random genetic mutation that produces an overabundance of the toxic protein, progerin. The accumulation of progerin occurs with aging in the general population, in the cardiovascular system and elsewhere in the body, such as the skin. In children and young adults with Progeria, the rate of progerin accumulation is highly accelerated, resulting in progressive cellular damage and atherosclerotic heart disease. Progerinin was developed by Korean-based biotech company PRG Science & Technology Co., Ltd. (PRG S&T) with research funding from The Progeria Research Foundation (PRF). The trial is a collaborative effort between PRG S&T, PRF, and trial site Boston Children’s Hospital, and will be managed by contract research organization Amarex. In the Phase 2a randomized open label trial, Progerinin will be administered in combination with the current standard-of-care, Zokinvy, to 10 patients with Progeria and Progeroid Laminopathies (PL) who are one year of age and older. Zokinvy has been shown to increase lifespan in a Progeria mouse model by 25%, and data published in the journal Cells in April 2023 indicated that Progerinin showed a potential increase in Progeria mouse lifespan by 50%. Zokinvy works by blocking the toxic, disease-causing protein, progerin, from developing and attacking normal cells, while Progerinin appears to reduce the level of progerin that accumulates in the body. Currently, Zokinvy is the only treatment for Progeria and PL approved by the FDA (in 2020), the European Medicines Agency (EMA, in 2022) and the Japanese Ministry of Health, Labor and Welfare (MHLW, in 2024). “Since its establishment, PRG S&T has focused on developing rare genetic disease treatments. This clinical trial demonstrates PRG S&T’s R&D capabilities to improve access to new drugs and expand treatment opportunities for children with progeria worldwide,” said Bumjoon Park, president of PRG S&T. “PRG S&T will focus on R&D and technological innovation to accelerate the development of treatments, including preparation for follow-up clinical trials, and lead to the development of new drugs.” “This new trial represents another step forward in our mission to treat and cure these amazing children and young adults with Progeria,” said PRF Executive Director Audrey Gordon, Esq. “Each Progeria clinical trial builds on learnings from our previous trials, so that we are continually making progress toward even better treatments and the cure. Most importantly, we are grateful for the courage and resilience of these children and young adults with Progeria and their families for participating in research programs and contributing to this important mission.” “Patients with Progeria and their families deserve more options,” said Dr. Kush Dhody, President, Amarex Clinical Research. “This is an especially devastating diagnosis given the shortened lifespan it causes. We’re honored to support a study that brings more hope to these patients.” About Progeria Progeria, also known as Hutchinson-Gilford Progeria Syndrome (HGPS), and PL are ultra-rare, multisystemic, premature aging diseases that accelerate mortality in young patients. Progeria is caused by a genetic mutation in the LMNA (“lamin A”) gene, and results in a disease-causing abnormal protein called progerin. There are approximately 400-450 children and young adults worldwide with Progeria. Thanks to PRF-funded research, we now know that progerin is produced in all of us as we age, but at a much lower rate than in children with Progeria. Due to this discovery of the biological connection between Progeria, heart disease and aging, finding the cure for one of the rarest diseases on earth could provide keys for treating millions of adults with heart disease and stroke associated with the natural aging process, as well as help the entire aging population. About PRG Science & Technology Co., Ltd. PRG S&T is a research & development company specializing in the treatment of rare genetic diseases. PRG S&T has been developing therapeutics for rare genetic diseases (laminopathies, neurodegenerative diseases, neuro-oncology etc.) with small molecules, which are derived from target sites on aberrant Protein-Protein Interactions (PPI). All four pipelines currently in PRG S&T’s possession have been designated as orphan drugs by the US FDA. Progerinin, the investigational treatment for Progeria has been approved by the FDA to enter phase 2 clinical trials, and is about to enter Phase2 at Boston Children’s Hospital. The non-clinical study of PRG-N-01, the treatment for neurofibromatosis type2, has been completed. IND for Phase I/II of PRG-N-01 has been approved by the Korea FDA and has been administered to patients at Asan Medical Center (South Korea) in June 2024. If these two drugs, Progerinin and PRG-N-01, are developed successfully, they are expected to become first-in-class innovative new drugs. For more information, please visit . About The Progeria Research Foundation The Progeria Research Foundation (PRF) was established in 1999 by the family of Sam Berns, a child with Progeria. Within four years of its founding, the PRF Genetics Consortium discovered the Progeria gene, a collaboration led by Dr. Francis Collins, former Director of the National Institutes of Health (NIH). PRF has funded and co-coordinated all lonafarnib-associated clinical trials for Progeria and Progeroid Laminopathies, conducted at Boston Children’s Hospital, and supports scientists who conduct Progeria research worldwide. PRF’s International Patient Registry includes over 370 children with Progeria in 70 countries. PRF is the only non-profit organization solely dedicated to finding treatments and the cure for Progeria and its aging-related conditions, including heart disease. The organization fills a void, putting these children and Progeria at the forefront of scientific efforts. For more information and to support PRF’s mission, please visit . About Amarex Clinical Research, LLC, an NSF company Amarex Clinical Research, LLC, is a global, full-service Contract Research Organization (CRO) with significant expertise conducting biomedical research. The leadership team’s combined experience includes the design and conduct of several hundred clinical research projects in many therapeutic indications. Amarex provides services in Project Management: Phase I-IV, BE/BA, PK/PD; Regulatory Affairs: FDA Applications and meetings, applications to International Health Authorities, GxP Compliance Audits; Clinical Operations; Adaptive Study Designs; Statistical Analysis; Data Management; Medical Monitoring; Safety and Pharmacovigilance; and General Consulting. Amarex can take your product through the entire approval process, from creating the regulatory approval strategy, to conducting trials, to writing the marketing approval application. Join our growing list of clients with approved products assisted by quality, cost-efficient services. For more information visit . Contact Information Eleanor Maillie PRF emaillie@progeriaresearch.org 978.879.9244 Sunghee Kim PRG S&T shkim@prgst.com Kalli DeWeese Amarex kdeweese@nsf.org Related Images PRG Science & Technology logo Amarex Clinical Research logo PRF logo SOURCE: The Progeria Research Foundation View the original press release on newswire.com.

Dr. Brown is a world-leading expert in Neurofibromatosis (NF) and the Director of the Neurofibromatosis clinic at The Mount Sinai Hospital, NYMIAMI, Sept. 03, 2024 (GLOBE NEWSWIRE) -- Pasithea Therapeutics Corp. (NASDAQ: KTTA) (“Pasithea” or the “Company”), a clinical-stage biotechnology company developing PAS-004, a next-generation macrocyclic MEK inhibitor, for the treatment of neurofibromatosis type 1 (NF1) and other indications, announced today that Rebecca Brown, M.D., Ph.D. has been appointed as a member of the Company’s Scientific Advisory Board. Dr. Brown is currently Director of the Neurofibromatosis Clinic at The Mount Sinai Hospital and Assistant Professor in the Department of Neurology (Division of Neuro-Oncology), Internal Medicine, and Neurosurgery at the institution. Dr. Brown will contribute scientific insights and valuable clinical perspectives to Pasithea's development of PAS-004. Dr. Brown has been an investigator in dozens of NF1 research studies including basic, translational, and clinical trials, with a focus on cutaneous and plexiform neurofibromas in NF1. Her research has been published extensively in major scientific journals and regularly serves as faculty at national and international meetings. “We are honored to have Dr. Brown, an internationally recognized NF1 expert, join our Scientific Advisory Board,” commented Dr. Tiago Reis Marques, Chief Executive Officer of Pasithea. “As we plan the initiation of our Phase 1/2a clinical trial in NF1 Dr. Brown’s extensive experience in the research and treatment of both plexiform and cutaneous neurofibromas will provide an important contribution to our PAS-004 development efforts going forward.” Dr. Rebecca Brown stated “I look forward to assisting with the development of PAS-004, a next-generation macrocyclic MEK inhibitor with promising preclinical data for the treatment of NF1.This product addresses an unmet need and potentially distinguishes itself from other drugs within the same class. The long half-life adds to other desirable features including a potentially superior therapeutic index, a cleaner safety profile, and easier compliance via once per day dosing. There have been tremendous achievements over the past 5 years in drug development for plexiform neurofibromas associated with NF1, and I believe that PAS-004 may provide the next big leap in treatment efficacy and compliance.” Dr. Rebecca Brown’s Biography Dr. Rebecca Brown (M.D., Ph.D.) is a board-certified adult neuro-oncologist who specializes in Neurofibromatosis (NF) and Schwannomatosis (SWN) genetic nerve tumor predisposition syndromes. She earned her Ph.D. from The University of Texas at Austin (UT Austin) in Neuroscience studying the molecular genomics and behavioral outcomes of endocrine-disrupting pollutants on females across multiple generations. Dr. Brown completed a post-doctoral fellowship at the Center for Strategic and Innovative Technologies at UT Austin in human performance research and then earned her M.D. from UT Southwestern in 2013. She completed her intern year at East Tennessee State University in 2014 and her neurology residency at Mount Sinai Hospital in NYC in 2017. She specialized in neuro-oncology during a fellowship at Memorial Sloan Kettering Cancer Center (MSKCC) completed in 2019. She worked as an instructor at MSKCC for 18 months prior to accepting a position as Assistant Professor and Director of the all-ages NF and SWN Clinic at The Mount Sinai Health System in January 2021. Dr. Brown has experience on both sides of the bench in NF laboratory research involving the RAS-RAF-MEK-ERK (MAPK) pathway, including genome editing, cell culture, xenografts, and clinical trials. Her particular interest is in developing treatments for NF1-associated dermal tumors called cutaneous neurofibromas. About PAS-004 PAS-004 is a small molecule allosteric inhibitor of MEK 1/2, which are dual-specificity protein kinases, in the MAPK signaling pathway. The MAPK pathway has been implicated in a variety of diseases, as it functions to drive cell proliferation, differentiation, survival, and a variety of other cellular functions that, when abnormally activated, are critical for the formation and progression of tumors, fibrosis, and other diseases. MEK inhibitors block phosphorylation (activation) of extracellular signal-regulated kinases (ERK), which can lead to cell death and inhibition of tumor growth. Existing FDA-approved MEK inhibitors are marketed for a range of diseases, including certain cancers and neurofibromatosis type 1 (NF1). We believe these MEK inhibitors suffer from certain limitations, including known toxicities. Unlike current FDA-approved MEK inhibitors, PAS-004 is macrocyclic, which we believe may lead to improved pharmacokinetic and safety (tolerability) profiles. Cyclization offers rigidity for stronger binding with drug target receptors. PAS-004 was designed to provide a longer half-life with what we believe is a better therapeutic window. Further, we believe the potency and safety profile that PAS-004 has demonstrated in preclinical studies may also lead to stronger and more durable response rates and efficacy, as well as better dosing schedules. PAS-004 has been tested in a range of mouse models of various diseases and has completed preclinical testing and animal toxicology studies. Additionally, PAS-004 has received orphan-drug designation from the FDA for the treatment of NF1, which may provide seven years of marketing exclusivity upon approval of an NDA. About Pasithea Therapeutics Corp. Pasithea is a clinical-stage biotechnology company focused on the discovery, research and development of innovative treatments for central nervous system (CNS) disorders and RASopathies. With an experienced team of experts in the fields of neuroscience, translational medicine, and drug development, Pasithea is developing new molecular entities for the treatment of neurological disorders, including Neurofibromatosis type 1 (NF1), Solid Tumors, and Amyotrophic Lateral Sclerosis (ALS). Forward Looking Statements This press release contains statements that constitute “forward-looking statements” made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include all statements, other than statements of historical fact, regarding the Company’s current views and assumptions with respect to future events regarding its business, as well as other statements with respect to the Company’s plans, assumptions, expectations, beliefs and objectives, the success of the Company’s current and future business strategies, product development, preclinical studies clinical studies, clinical and regulatory timelines, market opportunity, competitive position, business strategies, potential growth opportunities and other statements that are predictive in nature. Forward-looking statements are subject to numerous conditions, many of which are beyond the control of the Company. While the Company believes these forward-looking statements are reasonable, undue reliance should not be placed on any such forward-looking statements, which are based on information available to the Company on the date of this release. These forward-looking statements are based upon current estimates and assumptions and are subject to various risks and uncertainties, including factors set forth in the Company’s most recent Annual Report on Form 10-K, Quarterly Report on Form 10-Q and other filings made with the U.S. Securities and Exchange Commission (SEC). Thus, actual results could be materially different. The Company undertakes no obligation to update these statements whether as a result of new information, future events or otherwise, after the date of this release, except as required by law. Pasithea Therapeutics Contact Patrick GaynesCorporate Communicationspgaynes@pasithea.com

TUESDAY, Sept. 3, 2024 -- Fecal immunochemical tests (FITs) have variable sensitivity and specificity for detecting advanced colorectal neoplasia (ACN), according to a study published online Sept. 3 in the Annals of Internal Medicine . Barcey T. Levy, M.D., Ph.D., from the University of Iowa Carver College of Medicine in Iowa City, and colleagues compared the performance characteristics of five commonly used FITs (with colonoscopy as the reference standard) in a cross-sectional study in three U.S. academic medical centers and affiliated endoscopy units. Participants aged 50 to 85 years completed five different FITs before colonoscopy: four qualitative tests (Hemoccult ICT, Hemosure iFOB, OC-Light S FIT, and QuickVue iFOB) and one quantitative test (OC-Auto FIT). Test performance for ACN, defined as advanced polyps or colorectal cancer (CRC), was the primary outcome. A total of 3,761 participants were enrolled in the study. The researchers found that 320 participants had ACN (8.5 percent), including nine (0.2 percent) with CRC. Test positivity varied from 3.9 to 16.4 percent across FITs. There was variation observed in the rates of unevaluable FITs, from 0.2 to 2.5 percent. For ACN, the sensitivity and specificity varied from 10.1 to 36.7 percent and from 85.5 to 96.6 percent, respectively. All differences in sensitivity between FITs were statistically significant apart from the difference between Hemosure iFOB and QuickVue iFOB, while all differences in specificity were statically significant. Distal location of ACN was also associated with higher FIT sensitivity. "Our findings have implications for the benefits and cost-effectiveness of CRC screening using FIT," the authors write.