Neurofibroma

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.

MIAMI, Sept. 26, 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 cancer indications, today announced that it has entered into definitive agreements for the issuance and sale of an aggregate of 1,219,513 shares of its common stock (or pre-funded warrants in lieu thereof), accompanying Series A warrants to purchase up to 1,219,513 shares of common stock and accompanying short-term Series B warrants to purchase up to 1,219,513 shares of common stock at a purchase price of $4.10 per share (or per pre-funded warrant in lieu thereof) and accompanying warrants in a private placement priced at-the-market under the rules of the Nasdaq Stock Market. The Series A and the short-term Series B warrants will have an exercise price of $3.85 per share and will be exercisable immediately upon issuance. The Series A warrants will expire five years from the issuance date and the short-term Series B warrants will expire 18 months from the issuance date. The closing of the offering is expected to occur on or about September 30, 2024, subject to the satisfaction of customary closing conditions. H.C. Wainwright & Co. is acting as the exclusive placement agent for the offering. The gross proceeds from the offering are expected to be approximately $5 million, prior to deducting placement agent’s fees and other offering expenses payable by the Company. Pasithea intends to use the net proceeds from the offering for working capital and other general corporate purposes. The securities described above are being offered in a private placement under Section 4(a)(2) of the Securities Act of 1933, as amended (the “Securities Act”), and/or Regulation D promulgated thereunder and, along with the shares of common stock underlying the warrants, have not been registered under the Securities Act, or applicable state securities laws. Accordingly, the warrants and underlying shares of common stock may not be offered or sold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Securities Act and such applicable state securities laws. Pursuant to a registration rights agreement with investors, the Company has agreed to file a resale registration statement covering the securities described above. This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction. About Pasithea Therapeutics Corp. Pasithea is a 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 statements regarding the ability of the Company to consummation of the private placement, the satisfaction of the closing conditions of the private placement and the use of proceeds therefrom, the Company’s ongoing Phase 1 clinical trial and the safety, tolerability, pharmacokinetic (PK) and preliminary efficacy of PAS-004, as well as all other 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 risks that future clinical trial results may not match results observed to date, may be negative or ambiguous, or may not reach the level of statistical significance required for regulatory approval, as well as other 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

-- Single patient in 2mg cohort with stage 3 colon cancer who received 4 prior lines of therapy achieves prolonged stable disease and remains on drug into 6th dosing cycle -- -- No treatment-related adverse events (TRAEs) or dose-limiting toxicities (DLTs) observed to date, including no rash or gastrointestinal (GI) AEs -- -- Systemic exposure at steady-state enables constant target inhibition while avoiding peak plasma toxicities -- -- Half-life of approximately 70 hours supports once-daily or less frequent oral dosing -- -- Distinctive MEK inhibitor profile for the treatment of both NF1-related plexiform and cutaneous neurofibromas, cancer, and other opportunities -- MIAMI, Sept. 26, 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 cancer indications, today announced safety, tolerability, pharmacokinetic (PK) and preliminary efficacy data from the first 2 cohorts of patients (n=6) in its Phase 1 clinical trial of PAS-004, being conducted at four clinical sites in the United States. The Phase 1 clinical trial is a multi-center, open-label, dose escalation 3+3 study design to evaluate the safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and preliminary efficacy of PAS-004 in patients with MAPK pathway driven advanced solid tumors with a documented RAS, NF1 or RAF mutation or patients who have failed BRAF/MEK inhibition (NCT06299839). “We are very pleased to share the PK, safety, and preliminary efficacy data from the 2 mg and 4 mg cohorts in our first-in-human Phase 1 clinical trial of PAS-004. We believe these data demonstrate a PK and safety profile that differentiates PAS-004 as a next-generation MEK inhibitor. We have already achieved significant PAS-004 exposures with a favorable safety profile and have not seen adverse side effects such as rash or GI toxicity, which are typical for MEK inhibitors even at low doses. The long half-life at approximately 70 hours, and the ability to achieve a flat PK curve at steady-state, aim to provide a constant target inhibition while avoiding peak plasma toxicities, which is a unique PK profile among MEK inhibitors used for the treatment of Neurofibromatosis type 1 (NF1),” stated Dr. Tiago Reis Marques, Chief Executive Officer of Pasithea. “In addition, we are encouraged to see early potential signs of efficacy, with a heavily pre-treated patient with colorectal cancer showing prolonged stable disease. Colorectal cancer is known to not provide a RECIST response when treated with single-agent MEK inhibitors. This patient has a BRAF K601E mutation, a mutational status with no approved therapies. We are encouraged that this patient has been treated continuously into the 6th 28-day dosing cycle with no toxicities or AEs observed. While still early in clinical development, we believe PAS-004 is showing early signs of differentiation, indicating PAS-004 has the potential to outperform current MEK inhibitors in terms of safety, reduced administration frequency, and potentially efficacy. Our goal is to provide a once-daily or less frequent dosing treatment with broader application, not only for NF1 but also for other indications.” Interim Phase 1 Results Pharmacokinetics (PK) Plasma exposure increased with an increase in dose and linear PK is observedLong half-life of approximately 70 hours will allow for once daily dosing or longer intervalsProlonged systemic exposure with minimal fluctuation in PAS-004 plasma concentration at steady state (Cmax/Cmin ratio of 1.2) indicates a potential to achieve constant target inhibition At steady-state, drug levels peaked at about 5 hours with a geometric mean maximum concentration (Cmax) of 16.2 and 61.3 ng/mL for the 2 mg and 4 mg dose groups, respectively. The mean elimination half-life was 67.9 hours supporting once-daily or less frequent oral dosing. “PAS-004 has demonstrated distinct properties that we believe are significant advantages for an oral MEK inhibitor. PAS-004 has a significantly longer half-life compared to early-generation MEK inhibitors, particularly those used for the treatment of NF1, which have half-lives of less than 8 hours. The ability to achieve prolonged plasma exposures, as reflected in stable plasma concentrations at steady state, may potentially allow PAS-004 to achieve efficacious doses with a favorable safety profile,” stated Dr. Tiago Reis Marques, Chief Executive Officer of Pasithea. Safety & Tolerability No treatment-related adverse events (TRAEs) or dose limiting toxicities (DLTs) observed to date In the first 2 dosing cohorts (n=6), PAS-004 was shown to be well-tolerated with a favorable safety profile with no drug-related dose interruptions, reductions or discontinuations. There were no drug-related serious AEs (SAE) in any dose arm and no protocol-defined stopping criteria were met. Importantly, at the 2 and 4 mg dose levels no rash or skin toxicity, gastro-intestinal (GI) toxicity, or ocular toxicity have been observed to date. The study independent Safety Review Committee has completed its safety review of data from the second dose cohort of 4 mg and the Company has initiated cohort 3 dosing at an increased dose of 8 mg in capsules and has filed a protocol amendment to increase dosing schedule. PAS-004 Demonstrates a Differentiated MEK Inhibitor Profile Unlike first-generation MEK inhibitors for the treatment of NF1 that require twice-daily dosing (BID) and exhibit short half-lives (<8 hours), PAS-004 has the potential to achieve prolonged target inhibition due to its long half-life of approximately 70 hours with once-daily dosing (QD). The PK profile shows consistent plasma levels at steady-state, as reflected by a low Cmax to Cmin ratio, potentially reducing the risks for Cmax-related toxicity. These findings provide a compelling rationale for the advancement of PAS-004 into clinical trials for both the treatment of cutaneous and plexiform neurofibromas in NF1, cancer and other MAPK-driven opportunities. The company expects to provide additional trial updates on a periodic basis as the trial progresses. About Pasithea Therapeutics Corp. Pasithea is a 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 statements regarding the Company’s ongoing Phase 1 clinical trial and the safety, tolerability, pharmacokinetic (PK) and preliminary efficacy of PAS-004, as well as all other 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 risks that future clinical trial results may not match results observed to date, may be negative or ambiguous, or may not reach the level of statistical significance required for regulatory approval, as well as other 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 Photos accompanying this announcement are available at https://www.globenewswire.com/NewsRoom/AttachmentNg/4765238d-9961-438e-b459-509a5c917974 https://www.globenewswire.com/NewsRoom/AttachmentNg/0e484fc9-7068-4c8f-8060-f4109228d013