Pasithea Therapeutics宣布FDA授予PAS-004用于治疗肌萎缩侧索硬化症（ALS）的孤儿药资格认定

MIAMI, June 02, 2026 (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, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to PAS-004 for the treatment of Amyotrophic Lateral Sclerosis (ALS).

迈阿密，2026年6月02日（GLOBE NEWSWIRE）-- Pasithea Therapeutics Corp.（NASDAQ: KTTA）（“Pasithea”或“公司”）是一家临床阶段生物技术公司，正在开发PAS-004这一下一代大环MEK抑制剂。该公司今日宣布，美国食品药品监督管理局（FDA）已授予PAS-004用于治疗肌萎缩侧索硬化症（ALS）的孤儿药资格认定。

“ALS remains a devastating neurodegenerative disease with limited treatment options and significant unmet medical need,” said Dr. Tiago Reis Marques, Chief Executive Officer of Pasithea. “This designation further supports our efforts to explore the potential of PAS-004 in ALS and other serious diseases where dysregulation of the MAPK pathway may play an important role.”

“ALS仍是一种破坏性极大的神经退行性疾病，治疗选择有限，存在重大的未满足医疗需求，”Pasithea首席执行官Tiago Reis Marques博士表示。“这一认定进一步支持我们探索PAS-004在ALS以及其他严重疾病中的潜力，在这些疾病中，MAPK通路失调可能发挥重要作用。”

The FDA grants Orphan Drug Designation to therapies intended for the treatment, prevention, or diagnosis of diseases or conditions affecting fewer than 200,000 people in the United States. Orphan Drug Designation provides several potential benefits to drug developers, including eligibility for tax credits for qualified clinical trials, exemption from certain FDA fees, and the potential for seven years of market exclusivity following approval.

FDA向用于治疗、预防或诊断在美国影响少于200,000人的疾病或状况的疗法授予孤儿药资格认定。孤儿药资格认定为药物开发者提供若干潜在益处，包括符合条件的临床试验可享有税收抵免、免除某些FDA费用，以及获批后可能获得七年的市场独占权。

In November 2025, the Company announced a $1 million grant award from ALS Association to study the efficacy, safety, and tolerability of PAS-004 for the treatment of ALS.

2025年11月，公司宣布获得ALS Association提供的100万美元资助，用于研究PAS-004治疗ALS的疗效、安全性和耐受性。

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord. Over the course of the disease, people lose the ability to move, to speak, and eventually, to breathe. The disease is always fatal, usually within five years of diagnosis. Few treatment options exist, resulting in a high unmet need for new therapies to address functional deficits and disease progression.

肌萎缩侧索硬化症（ALS）是一种进展性神经退行性疾病，会影响大脑和脊髓中的神经细胞。随着疾病进展，患者会失去运动、说话并最终呼吸的能力。该病总是致命的，通常在确诊后五年内死亡。现有治疗选择很少，因此迫切需要新疗法来应对功能缺损和疾病进展。

Pasithea is a clinical-stage biotechnology company primarily focused on the research and development of its lead drug candidate, PAS-004, a next-generation macrocyclic MEK inhibitor intended for the treatment of RASopathies, MAPK pathway-driven tumors, and other diseases. The Company is currently testing PAS-004 in a Phase 1 clinical trial in patients with advanced cancer (NCT06299839), and a Phase 1/1b clinical trial in patients with neurofibromatosis type 1 (NF1)-associated plexiform neurofibromas with symptomatic and inoperable, incompletely resected, or recurrent PN (NCT06961565).

Pasithea是一家临床阶段生物技术公司，主要专注于其主要候选药物PAS-004的研究与开发。PAS-004是一种下一代大环MEK抑制剂，拟用于治疗RASopathies、MAPK通路驱动型肿瘤以及其他疾病。公司目前正在晚期癌症患者中开展PAS-004的1期临床试验（NCT06299839），并在患有症状性且不可手术、未完全切除或复发性PN的1型神经纤维瘤病（NF1）相关丛状神经纤维瘤患者中开展1/1b期临床试验（NCT06961565）。

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 of PAS-004 in advanced cancer patients, the Company’s ongoing Phase 1/1b clinical trial of PAS-004 in adult NF1 patients, and the safety, tolerability, pharmacokinetic (PK), pharmacodynamics (PD) 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, pre-clinical studies, clinical studies, clinical and regulatory timelines, market opportunity, competitive position, business strategies, potential growth and financing 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 Reports on Form 10-Q and other filings made with the U.S. Securities and Exchange Commission. 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.

本新闻稿包含根据1995年《私人证券诉讼改革法》安全港条款作出的构成“前瞻性陈述”的陈述。这些前瞻性陈述包括有关公司正在晚期癌症患者中开展的PAS-004 1期临床试验、公司正在成年NF1患者中开展的PAS-004 1/1b期临床试验，以及PAS-004的安全性、耐受性、药代动力学（PK）、药效学（PD）和初步疗效的陈述；还包括除历史事实陈述之外的所有其他陈述，涉及公司当前关于其业务未来事件的观点和假设，以及有关公司计划、假设、预期、信念和目标，公司当前及未来业务战略的成功，产品开发，临床前研究，临床研究，临床和监管时间表，市场机会，竞争地位，业务战略，潜在增长和融资机会，以及其他具有预测性质的陈述。前瞻性陈述受到众多条件的影响，其中许多并非公司所能控制。尽管公司认为这些前瞻性陈述是合理的，但不应过度依赖任何此类前瞻性陈述；这些陈述基于截至本新闻稿发布之日公司可获得的信息。这些前瞻性陈述基于当前估计和假设，并受到各种风险和不确定性的影响，包括未来临床试验结果可能与迄今观察到的结果不一致、可能为阴性或模糊，或可能达不到监管批准所要求的统计学显著性水平的风险，以及公司最新Form 10-K年度报告、Form 10-Q季度报告和向美国证券交易委员会提交的其他文件中列明的其他因素。因此，实际结果可能存在重大差异。除法律要求外，公司不承担在本新闻稿发布之日后因新信息、未来事件或其他原因更新这些陈述的义务。