RARE Daily

Takeda Reports Phase 3 Trial of Iclusig Met Primary Endpoint in Rare ALL

November 18, 2022

Takeda reported that the randomized, phase 3 PhALLCON trial met its primary endpoint, demonstrating that adult patients with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia treated with Iclusig plus reduced-intensity chemotherapy achieved higher rates of minimal residual disease-negative complete remission compared to imatinib.

Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) is a rare form of ALL that affects approximately 25 percent of adult ALL patients in the U.S. and is characterized by the presence of an abnormal gene, known as the Philadelphia chromosome. In patients who are Philadelphia chromosome-positive (Ph+), an abnormal chromosome is formed when pieces of chromosomes 9 and 22 switch with each other. This forms a longer chromosome 9 and a shorter chromosome 22, which leads to the development of BCR::ABL1 and is associated with Ph+ ALL.

Minimal residual disease (MRD)-negativity is associated with improvement in long-term outcomes for patients, as reported in literature. Iclusig is the only pan-mutational and third-generation tyrosine kinase inhibitor targeting BCR::ABL1 and all known single, treatment-resistant mutations, including the most resistant T315I mutation.

“Ph+ ALL is a fast-progressing disease with no targeted treatments currently approved in the frontline for patients in the U.S. There is an urgent need for an effective treatment that can suppress the development of difficult-to-treat mutations, which are associated with poor long-term outcomes,” said Awny Farajallah, head of Global Medical Affairs Oncology at Takeda. “We are excited to see how Iclusig may be able to address this gap in care for these patients and look forward to sharing the results.”

The PhALLCON study is a Phase 3 randomized, international, open-label multicenter trial evaluating the efficacy and safety of ICLUSIG versus imatinib in combination with reduced-intensity chemotherapy as a frontline therapy for adult patients with newly diagnosed Ph+ ALL. In the trial, no new safety signals were observed. Data from this trial will be discussed with regulatory agencies and shared with the scientific community in the future.

The U.S. Food and Drug Administration approved Iclusig in November 2016 for the treatment of adult patients with chronic-phase CML with resistance or intolerance to at least two prior kinase inhibitors, accelerated-phase or blast-phase CML or Ph+ ALL for whom no other kinase inhibitor is indicated, and T315I+ CML or T315I-positive Ph+ ALL.

Photo: Awny Farajallah, head of Global Medical Affairs Oncology at Takeda

Author: Rare Daily Staff

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