Duyuru • Jun 30
Pasithea Therapeutics Reports Positive Interim Phase 1 Advanced Cancer Study Data And Protocol Expansion Pasithea Therapeutics Corp. announced updated interim safety and clinical activity data from its ongoing first-in-human trial evaluating PAS-004 in patients with MAPK pathway-driven advanced solid tumors with a documented RAS, NF1 or RAF mutation, including patients who have failed prior BRAF/MEK inhibitor treatment. PAS-004 has been well-tolerated, with all treatment-related adverse events (TRAEs) being Grade 1 or 2, no observed cardiac or retinal treatment-related events, and low rates of rash and gastrointestinal toxicities. Protocol amendment extends dose escalation to higher levels: continued dose escalation using tablet formulation to explore 18mg, 24mg, 30mg, 40mg, and up to 52mg dose levels to further characterize safety, pharmacokinetics (PK), and early efficacy signals at higher doses. Interim PK and safety data at pharmacologically active doses support PAS-004 as a differentiated candidate with potential for long-term chronic dosing with favorable tolerability. An optional pilot food effect assessment has been introduced to evaluate the impact of fed vs. fasted administration on PAS-004 pharmacokinetics, to optimize long-term dosing strategy. 34 patients have been enrolled and dosed. Heavily pre-treated population with a median of 3 prior lines of therapy (range 1 – 8). Difficult to treat diagnoses (e.g., refractory pancreatic cancer and ovarian cancer), including 12 patients with BRAF V600E mutations, 10 of whom previously progressed on BRAFi and/or BRAFi/MEKi combinations. Well-tolerated safety profile with long-term, once daily dosing. No dose-limiting toxicities (DLTs), and no discontinuations due to TRAEs with 10 patients on study for >90 days, of which 6 patients >150 days, 4 patients >300 days, and 2 patients >365 days. All TRAEs were Grade 1 or Grade 2; low cumulative rates of rash (12%) and diarrhea (6%) across all patients at all doses throughout the study, including no (0%) rash or diarrhea in the 4 patients on study >300 days and 2 patients >365 days. No events of retinal or cardiac toxicity. Disease stabilization in the refractory population (N=12). Multiple patients demonstrating durable clinical benefit and tumor shrinkage, including several MEK/BRAF-pretreated patients who have remained on study for greater than six months and two patients on study for over one year – exceeding the approximately 5-month median progression-free survival reported in the literature for BRAF/MEK rechallenge in this setting. The final progression-free survival data will not be known until the end of the trial. Pharmacokinetics support daily dosing and exploration of higher dose levels. Long half-life (approximately 60 hours). Linear, dose proportional PK. Cmax/Cmin ratio. Duyuru • Jun 16
Pasithea Therapeutics Amends Clinical Study Protocol for Phase 1/1B Nf1 Clinical Trial Pasithea Therapeutics Corp. has amended the clinical study protocol for the Phase 1/1b trial of PAS-004 in neurofibromatosis type 1 (NF1) patients with symptomatic inoperable, incompletely resected, or recurrent plexiform neurofibroma (PN). The amendments include an update to the dose escalation part of the study (Part A) to allow for the enrollment of additional participants at two additional higher dose levels (24mg and 32mg), the ability to backfill completed dose cohorts (4mg, 8mg, 12mg and 18mg) with up to two participants, and the evaluation of intermediate dose levels. In addition, the protocol amendments allow patients to remain on treatment for up to 18 months in Part A, and include additional MRI scans to comprehensively evaluate PN, as well as adding more detailed cutaneous neurofibroma (CN) measurements, including tumor height and volume. Pasithea has completed enrollment and multi-cycle dosing of the initial 4mg, 8mg, 12mg and 18mg cohorts, and enrolled the 24mg cohort and an intermediate 15mg cohort. This multicenter, phase 1/1b, open-label study is dividend into two parts: a dose-escalation phase (part A) and an expansion cohort phase (part B). To date, the dose-escalation phase has enrolled and dosed 18 patients with NF1. Duyuru • May 05
Pasithea Therapeutics Corp. Appoints Kartik Krishnan as Chief Medical Officer, Effective May 1, 2026 Pasithea Therapeutics Corp. announced the appointment of Kartik Krishnan, M.D., Ph.D. as Chief Medical Officer (CMO) of the Company, effective May 1, 2026. Dr. Krishnan will oversee all clinical development and medical strategy as the Company advances PAS-004 through the clinic for the treatment of neurofibromatosis type 1 (NF1) associated plexiform and cutaneous neurofibromas. Dr. Krishnan has over 20 years of experience in clinical development, pharmacovigilance, clinical operations, regulatory affairs, and R&D strategy. Prior to joining Pasithea, Dr. Krishnan was Chief Executive Officer at OncoNano Medicines, a privately held company developing anti-cancer assets. Prior to that, Dr. Krishnan was Chief Medical Officer at Arcus Biosciences, a discovery and clinical development company focusing on combination therapies in immuno-oncology. Earlier in his career, he held various clinical development and medical director roles of increasing responsibility at companies including, Astex Pharmaceuticals, Genentech, Five Prime Therapeutics, BioMarin, and Amgen. Prior to joining industry, Dr. Krishnan held a faculty position in the Department of Pediatrics at the University of Arizona, with both clinical and primary research responsibilities. While at Genentech, Dr. Krishnan was an integral part of the clinical team for cobimetanib (Cotellic), contributing to the approval in the United States and Europe in 2015, for treatment of BRAF V600E or BRAF V600K melanoma in combination with vemurafenib (Zelboraf). In addition, Dr. Krishnan worked to establish development opportunities for this MEK inhibitor beyond melanoma, including monotherapy work in diseases such as Langerhans cell histiocytosis (for which cobimetinib was approved in 2022) and novel combinations in breast and colon cancer. Dr. Krishnan received his B.A. in History (with Distinction) from the University of Virginia. He completed his M.D. and Ph.D. in Molecular, Biochemical and Biophysical Studies at Columbia University. His Ph.D. studies were completed in the lab of Dr. John Krolewski, focusing on JAK/STAT signaling in the interferon pathway. Dr. Krishnan trained in pediatrics at UCLA and in pediatric hematology and oncology at the Johns Hopkins University and the National Cancer Institute. Duyuru • Apr 21
Pasithea Therapeutics Corp Announces Grant of Rare Pediatric Disease Designation by FDA to Pas-004 for Treatment of Neurofibromatosis Type 1 Pasithea Therapeutics Corp. announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to PAS-004 for treatment of Neurofibromatosis type-1 (NF1). The FDA grants RPDD for serious or life-threatening diseases in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years and affect fewer than 200,000 people in the U.S. There are approximately 115,000 individuals in the U.S living with NF1. Under the FDA's Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives an approval of a new drug application (NDA) or biologics license application (BLA) for a rare pediatric disease may be eligible for a Priority Review Voucher (PRV) which can be redeemed to obtain priority review for a subsequent marketing application for a different product. The PRV may be sold or transferred to another sponsor. In the last 12 months, disclosed PRV sales have ranged from $150–$205 million. PAS-004 has so far been granted the following FDA regulatory designations: Orphan Drug Designation, Fast Track Designation and Rare Pediatric Disease Designation. The Company is currently conducting a Phase 1/1b multicenter, open-label, dose escalation trial of PAS-004 in adult participants with symptomatic, inoperable, incompletely resected, or recurrent NF1-PN (NCT06961565). Plexiform neurofibromas (PN) are tumors originating from the nerve sheath that grow through and around nerves and may involve multiple nerve branches. Thirty to fifty(30-50%) of patients with NF1 will harbor PNs, which can undergo malignant transformation. PN-related morbidities are primarily caused by the direct impact of the tumor on surrounding structures and can be life-threatening when they compress vital organs or when they become malignant. Pasithea 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). Duyuru • Apr 01
Pasithea Therapeutics Corp Announces Grant of Fast Track Designation by Fda to Pas-004 for Treatment of Neurofibromatosis Type 1 Associated Plexiform Neurofibromas Causing Significant Morbidity Pasithea Therapeutics Corp. a clinical-stage biotechnology company developing PAS-004, a next-generation macrocyclic MEK inhibitor, announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to PAS-004 for the treatment of NF1-associated PN causing significant morbidity. Fast Track designation includes the following opportunities to facilitate Fast Track product development: Frequent communications with the FDA review team throughout the product development process. Rolling Review, which allows portions of a marketing application to be reviewed and assessed by the FDA before the company submits the complete application. In addition, a Fast Track designation product may potentially be eligible for accelerated approval and/or priority review if relevant criteria are met. The Company is currently conducting a Phase 1/1b multicenter, open-label, dose escalation trial of PAS-004 in adult participants with symptomatic, inoperable, incompletely resected, or recurrent NF1-PN (NCT06961565). Plexiform neurofibromas (PN) are tumors originating from the nerve sheath that grow through and around nerves and may involve multiple nerve branches. Thirty to fifty percent (30-50%) of patients with NF1 will harbor PNs, which can undergo malignant transformation. PN-related morbidities are primarily caused by the direct impact of the tumor on surrounding structures and can be life-threatening when they compress vital organs or when they become malignant. 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). 
