Takeda targets leukemia competitor to Novartis' Scemblix in $1.3B Ascentage deal

Takeda Pharmaceutical has entered into an agreement with Ascentage Pharma that could be worth up to $1.3 billion. This deal includes a $100 million signing bonus for Ascentage and potential milestone payments totaling $1.2 billion if Takeda exercises its licensing option. The agreement centers around Ascentage’s olverembatinib, a third-generation BCR-ABL tyrosine kinase inhibitor (TKI) being developed for chronic myeloid leukemia (CML) and other hematological cancers.

If Takeda chooses to exercise the licensing option, it would gain global development and commercialization rights for olverembatinib, excluding Greater China and Russia. Additionally, Takeda will make a $75 million minority equity investment in Ascentage.

Olverembatinib, already approved in China, is the first third-generation BCR-ABL1 inhibitor on the market there, specifically for CML patients with T315I mutations. It is approved for TKI-resistant chronic-phase CML or accelerated-phase CML patients who have the T315I mutation. Outside of China, the drug has received an FDA fast-track designation and an orphan drug designation from the European Medicines Agency.

Despite the availability of various TKIs, there remains an unmet need for CML patients who have developed resistance or refractory conditions to existing treatments. Takeda’s president of the global oncology business unit, Teresa Bitetti, highlighted the company's history of advancing treatments for hematological cancers and expressed optimism about olverembatinib's potential.

Ascentage's CEO, Dajun Yang, emphasized the importance of leveraging Takeda’s global commercial expertise to further develop and market olverembatinib. The drug is set to enter a global phase 3 trial, coded POLARIS-2, for previously treated adult patients with chronic-phase CML with or without the T315I mutation. This trial was approved by the FDA in February and is expected to begin in the first half of the year.

Novartis, another major player in the TKI space, is also addressing the unmet need left by traditional TKIs. Its drug Scemblix has received conditional FDA approval for third-line CML treatment and is now being tested as a first-line treatment. In a head-to-head phase 3 study, Scemblix showed better and faster molecular response rates compared to its predecessor, Gleevac.

TKIs have significantly improved CML treatment since Novartis’ Gleevac was first approved, transforming a once fatal diagnosis into a manageable condition. However, treatment resistance and difficult-to-treat mutations still pose challenges, prompting ongoing development and innovation in this field.