FDA Grants Fast Track Status to PHST001 in Ovarian Cancer
The FDA granted fast track designation to PHST001 for the treatment of advanced ovarian cancer.
US FDA
- PHST001, an investigational anti-CD24 monoclonal antibody, received FDA fast track designation for advanced platinum-resistant ovarian cancer.
- This includes its use as a monotherapy for platinum-resistant disease and in combination with chemotherapy for platinum-sensitive disease.
- PHST001 is currently in a phase 1 trial (NCT06840886) evaluating its safety, tolerability, and preliminary efficacy in patients with advanced relapsed/refractory solid tumors, with dose escalation and planned expansion cohorts.
The FDA recently granted fast track designation to PHST001, an investigational anti-CD24 monoclonal antibody, for the treatment of advanced ovarian cancer. This designation applies to both platinum-resistant ovarian cancer as a monotherapy and in combination with chemotherapy for platinum-sensitive disease.1
This accelerated pathway underscores the significant unmet need in ovarian cancer treatment and the potential of PHST001 to address it.
“Receiving fast track designation from the FDA reinforces the promise of CD24 as a next-generation immuno-oncology target and highlights the potential of PHST001 to address a critical unmet need in the treatment of ovarian cancer,” said Raphaël Rousseau, MD, PhD, chief medical officer of Pheast Therapeutics, in a press release. “We are committed to advancing PHST001 through the clinic and accelerating its development for cancer patients in urgent need of more effective treatments.”
PHST001 is designed to bolster innate immune recognition and antitumor activity in solid tumors. The rationale behind its mechanism of action centers on CD24, a cell surface protein highly expressed across various tumor types, including ovarian and triple-negative breast cancers. CD24 interacts with Siglec-10, an inhibitory receptor on macrophages, thereby promoting tumor immune evasion. High CD24 expression is also recognized as a negative prognostic factor in several malignancies. By targeting CD24, PHST001 aims to disrupt this immunosuppressive axis and unleash a more robust antitumor immune response.
PHST001 is currently being evaluated in a multicenter, open-label, phase 1 trial.2 This study is assessing the safety, tolerability, and preliminary efficacy of PHST001 in patients with advanced relapsed/refractory solid tumors.
The study is enrolling patients aged 18 and older with histologically or cytologically confirmed advanced solid tumors that have relapsed or are refractory to all available standard therapies. Key inclusion criteria include adequate hepatic, renal, and bone marrow function.
In the dose-escalation phase, patients will receive PHST001 at 1 of 7 dose levels, administered once every 3 weeks on day 1 of each 21-day cycle. Approximately 40 to 80 patients are anticipated for this initial phase, with subsequent expansion cohorts planned for the second part of the trial.
The primary end points of the study are safety and the incidence of dose-limiting toxicities. Secondary end points include preliminary antitumor activity per RECIST 1.1 criteria, pharmacokinetics, and pharmacodynamics.
