Pasithea Therapeutics Announces FDA Clearance of IND for PAS-004 in Neurofibromatosis Type 1 (NF1)-Associated Cutaneous Neurofibromas (cNF)

The clearance extends PAS-004 development into a second indication for which Pasithea intends to seek regulatory approval.

Rhea-AI Summary

Pasithea Therapeutics (Nasdaq: KTTA) received FDA clearance to study PAS-004 in a Phase 2 trial for NF1-associated cutaneous neurofibromas.

The randomized, placebo-controlled trial will evaluate the oral drug candidate in patients with skin tumors associated with neurofibromatosis type 1 (NF1). The active investigational new drug application permits clinical development in these patients in the United States, under the FDA Division of Dermatology and Dentistry.

Pasithea intends to seek regulatory approval for this second indication. There are currently no FDA-approved systemic therapies for NF1-associated cutaneous neurofibromas. The application was supported by nonclinical safety data and clinical safety, tolerability, drug-exposure data and early treatment-response signals from ongoing studies in adults with NF1-associated plexiform neurofibromas and advanced cancer.

3 points · 0 major

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Hollow bars mark forward-looking points. How the balance works

Positive

• Moderate pointFDA clearance permits a randomized, placebo-controlled Phase 2 trial of PAS-004 in NF1-associated cutaneous neurofibromas.
• Minor pointEarly treatment-response signals from ongoing NF1-associated plexiform neurofibroma and advanced cancer studies supported the application.
• Minor point. Forward-looking: it has not happened yet and may not happen.Second indication: Pasithea intends to seek regulatory approval for PAS-004 in NF1-associated cutaneous neurofibromas.

Negative

• None.

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Key Figures

NF1 prevalence of cNF: over 95% Moderate-to-severe cNF population: more than 80,000 patients Trial phase: Phase 2

NF1 prevalence of cNF

over 95%

People with NF1 affected by cutaneous neurofibromas

Moderate-to-severe cNF population

more than 80,000 patients

Estimated NF1 patients in the United States

Trial phase

Phase 2

Randomized, placebo-controlled trial for NF1-associated cNF

Previous Clinical trial Reports

1 past event · Latest: Oct 23

Same Type 1 event

1. Oct 23

   Phase 1 clinical data

   24h Move

   -9.1%

   Phase 1 data reported prolonged stable disease and mostly Grade 1–2 treatment-related adverse events.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Key Terms

investigational new drug, placebo-controlled, mek inhibitor, pharmacokinetic

4 terms

investigational new drug regulatory

"cleared the Company’s Investigational New Drug (IND) application"

An investigational new drug is a medication that is still being tested in clinical trials to determine if it is safe and effective for treating a specific condition. For investors, it represents a potential breakthrough that could lead to a new treatment and significant financial gains if successful, but also carries risks since it has not yet been approved for widespread use.

placebo-controlled medical

"for a randomized, placebo-controlled Phase 2 clinical trial"

"Placebo-controlled" describes a testing method where one group receives the actual treatment or intervention, while another group receives a harmless, inactive version called a placebo. This approach helps determine whether the real treatment has genuine effects beyond psychological expectations. For investors, understanding this ensures confidence that reported benefits are real and not influenced by bias or false perceptions.

mek inhibitor medical

"a next-generation oral macrocyclic MEK inhibitor"

A MEK inhibitor is a drug that blocks MEK enzymes, which act like a relay in a cell’s growth-signaling pathway; by cutting that relay these drugs can slow or stop uncontrolled cell growth seen in some cancers and other proliferative conditions. Investors care because clinical trial outcomes, safety data, and regulatory approvals determine whether a MEK inhibitor becomes a marketable therapy, directly affecting a developer’s revenue prospects and valuation — like a product passing a crucial safety and sales test.

pharmacokinetic medical

"pharmacokinetic data and early signals of treatment response"

Pharmacokinetic describes how a drug moves through and leaves the body — how it is absorbed, spread to tissues, broken down and excreted — like tracking a package from pickup to delivery and disposal. For investors, these properties determine effective dose, safety risks, how often a medicine must be taken, and how reliably it works, which in turn influence clinical trial success, regulatory approval chances, production complexity and a drug’s commercial value.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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— FDA clearance allows Pasithea to advance PAS-004 for NF1-associated cNF, a condition affecting over 95% of people with NF1 and for which there are currently no FDA-approved systemic therapies. —

MIAMI, Nov. 03, 2026 (GLOBE NEWSWIRE) -- Pasithea Therapeutics Corp. (Nasdaq: KTTA) (“Pasithea” or the “Company”), a clinical-stage biotechnology company developing PAS-004, a next-generation oral macrocyclic MEK inhibitor for the treatment of chronic diseases, including neurofibromatosis type 1 (NF1)-associated neurofibromas, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) application for a randomized, placebo-controlled Phase 2 clinical trial for PAS-004 in patients with NF1 associated cutaneous neurofibromas (cNF). The Phase 2 study will be conducted under an active IND overseen by the FDA Division of Dermatology and Dentistry (DDD).

“FDA clearance of our IND for PAS-004 in cutaneous neurofibromas is an important milestone for Pasithea and a significant step forward for people living with NF1 who have cNFs,” said Kartik Krishnan, MD, PhD, Chief Medical Officer of Pasithea. “Cutaneous neurofibromas are among the most visible and burdensome manifestations of NF1, and there are currently no approved systemic therapies for them. PAS-004 is designed to address this unmet need as a once-daily oral MEK inhibitor whose long half-life and flat pharmacokinetic profile may support chronic dosing.”

“We are pleased to expand the clinical development of PAS-004 into NF1-associated cNF, the second indication for which we intend to seek regulatory approval. PAS-004 has the potential to become the first FDA-approved systemic treatment for NF1-associated cNF, addressing a significant unmet medical need and what we believe represents a multibillion-dollar market opportunity,” said Dr. Tiago Reis Marques, MD, PhD, CEO of Pasithea. “PAS-004 is intended for the more than 80,000 NF1 patients in the United States estimated to have moderate to severe cNF disease burden.”

Pasithea supported its IND with nonclinical safety, clinical safety, tolerability and pharmacokinetic data and early signals of treatment response emerging from its ongoing Phase 1/1b study in adults with NF1-associated plexiform neurofibromas and its ongoing Phase 1 study in adults with advanced cancer.

With the IND now in effect, Pasithea may proceed with clinical development of PAS-004 in patients with NF1-cNFs in the United States.

About Cutaneous Neurofibromas

NF1-associated cutaneous neurofibromas (cNF) are benign tumors that grow in or just beneath the skin at virtually any location in the body and are a hallmark of NF1 It is a serious condition and associated with significant morbidity that can have substantial impact on day-to-day functioning, leading patients to rate this as the most significant burden of NF1 despite other potentially life-threatening complications. cNF typically begin to appear in adolescence, generally increase in number and volume with age. The number of cNF on an individual patient can range from a few to tens of thousands. Although malignant transformation does not occur with NF1-associated cNF, cNF are present in over 95% of individuals with NF1 and have a significant negative impact both physically, with disfigurement, and on health-related quality of life (QoL). A greater cNF burden, particularly volume and number of cNF and facial involvement, are associated with poorer health-related QoL. Patients have rated cutaneous manifestations of NF1 as the most significant burden of NF1 largely owing to cNF-associated disfigurement despite other potentially life-threatening complications. In a Patient Listening Session organized by the Children’s Tumor Foundation (CTF), four of the six representatives who were selected to collectively represent the NF1 community described how the appearance of the cNF and associated stigma consistently interferes with their activities of daily living, including being denied employment opportunities, harassed and in fear of being out in public. Current management relies largely on procedural approaches such as surgical excision, electrodesiccation, or laser therapy. There are no FDA approved therapies approved for the treatment of NF1-associated cNF.

About Pasithea Therapeutics Corp.

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 advanced cancer patients (NCT06299839), and a Phase 1/1b clinical trial in adult patients with neurofibromatosis type 1 (NF1)-associated plexiform neurofibromas (NCT06961565).

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 Investigational New Drug (IND) application for PAS-004 in cutaneous neurofibroma (cNF), the Company’s plans for and the design, timing, size, enrollment, and conduct of a Phase 2 clinical trial of PAS-004 in patients with NF1-associated cNF, the potential of PAS-004 as a treatment for cNF, the unmet medical need in cNF, the Company’s clinical data from its Phase 1 dose-escalation study of PAS-004 in advanced solid tumors, the Company’s Phase 1/1b clinical trial of PAS-004 in adult NF1 patients, and the safety, tolerability, pharmacokinetic (PK), pharmacodynamic (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 related to the initiation, design, timing, enrollment, and conduct of clinical trials and interactions with regulatory authorities, and 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.

Contact

Company Contact:
Patrick Gaynes
Corporate Communications
pgaynes@pasithea.com

For Investor Inquiries:
Mike Moyer
Managing Director, LifeSci Advisors, LLC
mmoyer@lifesciadvisors.com

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

What did the FDA clear for Pasithea’s PAS-004 in cutaneous neurofibromas?

The FDA cleared an investigational new drug application for a randomized, placebo-controlled Phase 2 trial of PAS-004 in patients with NF1-associated cutaneous neurofibromas. The clearance allows clinical development in these patients in the United States; it is not marketing approval.