At ASCO’s 2023 convention for patients diagnosed with glioma and a specific genetic alteration, Servier Pharmaceuticals announced that vorasidenib, the drug being investigated in its Phase III trial (INDIGO) slowed tumor growth by a median of 61%. Vorasidenib is an oral inhibitor of mutant IDH1/2 enzymes.
Additionally, the vorasidenib group showed a 27.7-month period before a progression in their tumors against 11.1 months for patients receiving a placebo. The FDA granted vorasidenib Fast Track designation in March 2023. The results of the study were published in the NEJM.
About the Participants
A total of 331 patients who were between the ages of 16 and 71 were enrolled. Each patient had grade 2 gliomas, a brain cancer, with IDH mutations or prior surgery with either unsatisfactory results or the patient had relapsed.
It is estimated that this form of glioma impacts approximately four thousand individuals in the United States annually.
Study investigator Katherine Peters at Duke’s Cancer Center commented that this therapy can avoid patients having to endure aggressive treatment such as chemotherapy, radiation, or additional surgery. Dr. Peters added that it is a development we have not seen in more than two decades.
The Secondary Endpoint
The time to the next intervention, the secondary endpoint, was met. At the time of this analysis, the vorasidenib group had not indicated the need for intervention but the placebo group did require intervention at 17.8 months.
Servier, a private biopharma company and the developer of vorasidenib, reported that alanine aminotransferase (ALT) an enzyme that indicates liver function, showed a 9.6% increase in patients receiving the drug. The company said the toxicity (Grade 3) was reversible and manageable. It is believed that it can be resolved given proper medical attention. Servier has full confidence in the safety profile exhibited in the trial.
The company began its U.S. operations by setting up a subsidiary in Boston about five years ago. It started with several significant acquisitions such as $2.4 billion for Shire’s oncology products with Vorasidenib as the lead product.
The second major acquisition was the $1.8 billion acquisition of Agios Pharmaceutical’s AG 270 under development for NSCLC and pancreatic cancer.