US-based drug discovery and development firm Vivace Therapeutics has received the US Food and Drug Administration (FDA) Orphan Drug Designation for VT3989 in mesothelioma.
VT3989 is an investigational small-molecule cancer therapeutic designed to target the Hippo pathway by inhibiting palmitoylation of TEAD protein family members.
The TEAD autopalmitoylation inhibitor has been assessed in over 200 patients in an ongoing, open-label Phase 1 clinical trial.
It is reportedly the first TEAD autopalmitoylation inhibitor with publicly available compelling clinical efficacy data.
Clinical findings for VT3989 have shown promise in mesothelioma patients who have not responded to chemotherapy and immuno-oncology combination treatments.
Vivace Therapeutics president and CEO Sofie Qiao said: “The granting of Orphan Drug Designation to VT3989 underscores the critical need for new, effective therapies for mesothelioma, an aggressive cancer with limited treatment options.
“The benefits provided by this important designation will support our continued advancement of VT3989, which has already generated compelling clinical trial data, a first for this promising therapeutic class.
“We are committed to continuing clinical development of VT3989 and discussing a move into a registrational Phase 3 study in mesothelioma with FDA by the end of 2025.”
The FDA’s Orphan Drug Designation is intended to support the development of treatments for rare diseases affecting fewer than 200,000 people in the US.
The designation offers benefits such as tax credits for clinical trials, exemptions from certain FDA fees, and potentially seven years of market exclusivity post-approval.
The Phase 1 study evaluated the safety, tolerability, pharmacokinetics, and biological activity of VT3989 in patients with refractory metastatic solid tumours, including refractory pleural and non-pleural malignant mesothelioma.
The multi-centre, open-label trial has demonstrated a positive safety profile, reinforcing its potential as a best-in-class therapeutic option.
Based in the San Francisco Bay Area, Vivace Therapeutics is focused on small-molecule drug discovery targeting the Hippo pathway.
Recently, the company has secured $35m in a Series D financing round, led by RA Capital Management, alongside existing investors Canaan Partners and Cenova Capital.
Vivace intends to use the funds to advance the clinical development of VT3989.
