Patients diagnosed with pleural mesothelioma have limited treatment options, but the FDA’s recent approval of fast-track designation for an investigational small molecule therapeutic called VT3989 offers reason for hope. The new treatment, which is specifically designed to treat unresectable malignant pleural or nonpleural mesothelioma that has progressed after prior immune checkpoint inhibitor therapy and platinum-based chemotherapy, has shown promising results in clinical trials.
Mesothelioma Patients Need New Therapeutic Options
Patients diagnosed with pleural mesothelioma are often treated using traditional cancer therapies, but once their disease has progressed to an advanced stage, there is little that can be done. Researchers are working to address this need, and innovative drugs that deliver promising results in clinical tests are often granted the FDA’s fast track designation so that they can be brought to market more quickly. Vivace Therapeutics, a small molecule discovery and development company, recently announced that its drug VT3989 has received this designation. The therapy targets the Hippo pathway, inhibiting a process known as transcriptional enhanced associate domain autopalmitoylation.
According to Sofie Qiao, president and chief executive officer of Vivace Therapeutics, the desperate need for new and effective treatment for rare illnesses like mesothelioma, known as orphan drugs, is what drives the FDA’s fast track approval. When companies can demonstrate a drug’s progress in treating one of these challenging illnesses, the program process facilitates development and expedites its review, allowing the drugs to reach patients earlier than through standard approval pathways.
Mesothelioma Drug Targets Critical Hippo Pathway Dysfunction
Mesothelioma tumors frequently show signs of malfunctions in the signaling pathway called the HIPPO pathway, which, when functioning well, regulates transcription factors YAP and TAZ. Unfortunately, when this pathway malfunctions in tumors, YAP and TAZ are relocated to the cell nucleus, where they interact with certain proteins, activating genes. This leads to uncontrolled proliferation and the growth of the deadly tumors. VT3989 is designed to restore control over these ca mechanisms, which are particularly common in mesothelioma.
Patients diagnosed with mesothelioma and other metastatic solid tumors are being recruited for an ongoing open-label phase 1 clinical study. The study, which is assessing the safety, tolerability, pharmacokinetics, and biological activity of VT3989, has already evaluated its impact in more than 200 patients at a dozen locations throughout the United States and Australia. The trial is anticipated to eventually enroll 336 patients, with an estimated primary completion date of December 24, 2026, and a final study completion date of June 2, 2027.
If you or someone you love has been diagnosed with mesothelioma, the Patient Advocates at Mesothelioma.net are here to help. Contact us today at 1-800-692-8608 to learn more about the resources available to you.
