Final Results of Phase 3 Trial for INQOVI® in MDS and CMML Patients

Taiho Oncology, Inc. has published the conclusive findings from the ASCERTAIN clinical trial, which evaluated the oral combination of decitabine and cedazuridine, known as INQOVI®. The trial aimed at adults with certain types of myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML). A first of its kind, the Phase 3 study established the pharmacological equivalence of an oral formulation to an intravenous one for hypomethylating agents used in treating MDS or CMML. The research showed a median overall survival rate of about 32 months and an overall response rate of 62% among the patients who intended to be treated.

The safety profile of INQOVI® was found to be on par with that of intravenous decitabine, with common side effects being thrombocytopenia, neutropenia, and anemia—aligning with expectations for such treatments. These results underpinned the FDA and Health Canada's approval of INQOVI® in July 2020 for MDS treatment, encompassing both previously untreated and treated cases, as well as various subtypes and risk groups.

Historically, hypomethylating agents such as azacitidine and decitabine were accessible only in injectable forms, necessitating regular visits to healthcare facilities for infusions. However, the ASCERTAIN study indicated that the oral decitabine and cedazuridine combination offers a feasible alternative, potentially minimizing the need for frequent medical center visits. The study's lead author, Dr. Guillermo Garcia-Manero, highlighted the significance of these findings, noting the encouraging median overall survival rate exceeding historical data.

Dr. Tehseen Salimi, Senior Vice President, and Head of Medical Affairs at Taiho Oncology, Inc., emphasized the benefits of INQOVI®, particularly its convenience for patients with MDS and CMML, possibly reducing office visits and travel.

The ASCERTAIN trial was a multicenter, randomized, open-label study that compared the oral decitabine and cedazuridine combination to intravenous decitabine, administered over the initial two cycles. From the third cycle onwards, patients continued to receive the oral combination. The study's main goal was to confirm the equivalence of the 5-day area-under-the-curve (AUC) for the oral and intravenous formulations.

INQOVI® represents a breakthrough as the first and only oral hypomethylating agent to receive approval from the FDA and Health Canada for treating intermediate and high-risk MDS, including CMML. Taiho Oncology, Inc., and Taiho Pharma Canada, Inc., are responsible for its commercialization in the U.S. and Canada, respectively.

The product's safety information warns of potential myelosuppression and embryo-fetal toxicity, advising pregnant women of the risks and recommending contraception during and after treatment. Common adverse reactions and laboratory abnormalities were also detailed, underscoring the necessity for blood cell count monitoring and appropriate therapeutic adjustments.

Taiho Oncology, Inc., is dedicated to enhancing the lives of cancer patients and their support systems, focusing on developing and marketing oral anti-cancer drugs. With a strong pipeline of small molecule clinical candidates for various cancers, the company operates under the umbrella of Taiho Pharmaceutical Co., Ltd., a subsidiary of Otsuka Holdings Co., Ltd. Headquartered in Princeton, New Jersey, Taiho Oncology also manages its parent company's operations in Europe and Canada.