Pasithea Therapeutics has announced the start of a Phase I/Ib open-label trial of macrocyclic mitogen-activated protein kinase (MEK) inhibitor, PAS-004, for treating adults with neurofibromatosis type 1 (NF1) who have symptomatic and inoperable plexiform neurofibromas.
The trial is also intended for those with incompletely resected or recurrent plexiform neurofibromas.
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It aims to evaluate the therapy’s tolerability, safety, pharmacodynamics (PD), and pharmacokinetics (PK) to also determine preliminary anti-tumour activity and a recommended dose for the following Phase II trials.
Royal North Shore Hospital in Sydney, Australia, is the initial active trial site, with subject enrolment anticipated to start in the second quarter of this year.
Pasithea plans to open additional trial sites in Australia, the US, and South Korea in the upcoming months.
Australia’s Novotech has been selected as the clinical research organisation for the trial.
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By GlobalDataPasithea is carrying out the trial via Pasithea MacroMEK, the company’s wholly owned subsidiary in Australia.
The trial’s primary aim is to assess the tolerability and safety of PAS-004 over a 28-day treatment cycle in this patient population.
Secondary objectives include identifying the recommended Part B dose or maximum tolerated dose, characterising the PK and PD profiles, and assessing preliminary efficacy on target plexiform neurofibroma volume and cutaneous neurofibromas.
Part A of the trial will enrol up to 24 subjects who will be given one of four planned PAS-004 tablet doses, 4mg, 8mg, 12mg, 18mg, in a modified three plus three design to identify the recommended Part B dose.
In Part B, subjects will be dosed at the recommended Part B dose level and a lower dose for up to six continuous cycles of 28-day treatment to detect the recommended Phase II dose.
Pasithea Therapeutics CEO Dr Tiago Reis Marques said: “We are encouraged by the safety and clinical data observed to date in oncology patients and are optimistic that PAS-004’s tolerability profile will extend to the NF1 population.
“Importantly, our existing cancer data has enabled us to begin the NF1 trial at a higher dose than originally contemplated. In addition, we anticipate meaningful cash rebates of eligible trial costs through the Australian research and development (R&D) Tax Incentive, further enhancing the efficiency of this programme.”
