Precision AML Trial Adds Pharma Candidates To The Mix

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Includes: ALXN, AMGN, BAYRY, CELG, GILD, JAZZ, SGEN
by: EP Vantage

The ambitious multi-armed acute myeloid leukaemia trial Beat AML will test as many as 10 clinical-stage pharma projects, a substantial expansion from the four candidates originally announced, the study’s chief investigators revealed at Ash on Sunday.

The additional projects have not been disclosed because the contracts are being negotiated, but the trial’s coordinators, the Leukemia and Lymphoma Society (LLS), said they would be added along with new AML subtypes. A focus on subgroup identification and collaboration with US FDA reviewers has the potential to speed clinical projects to an approval decision, so it is no wonder that more pharma groups want to be included.

Passing lane

Beat AML is intended to accelerate targeted agents into a disease that has not seen innovation in 40 years, since the introduction of cytarabine and daunorubicin chemotherapy – a cocktail that is being incrementally improved upon by Jazz Pharmaceuticals’ (NASDAQ:JAZZ) Vyxeos (Collaborative leukaemia trial orbits moonshot initiative, October 19, 2016).

Study coordinators so far have named four “baskets”: patients with core binding factor AML, patients with partial tandem duplication or chromosomal translocations on the MLL gene, those with IDH2 mutations, and those with hypermethylation – these patients will be identified within seven days of screening via genetic testing.

The first group will be treated with Alexion Pharmaceuticals’ (NASDAQ:ALXN) back-from-the-dead anti-CD200 antibody samalizumab, the second with Gilead Sciences’ (NASDAQ:GILD) kinase inhibitor entospletinib, the third with Celgene’s (NASDAQ:CELG) enasidenib, and the last with Boehringer Ingelheim’s anti-CD33 antibody BI 836858.

Unlike many trials focusing on subtypes, those without any of these biomarkers will have a targeted treatment option in the form of a FLT3 inhibitor – the researchers did not name the FLT3, but Novartis’s midostaurin is the most advanced in this class and currently awaits an FDA decision.

In a presentation at Ash, investigator Dr Brian Druker of the Oregon Health & Science University said additional biomarker-driven arms will be NPM1, p53/complex karyotype, FLT3 and IDH1 mutation.

Small numbers, big differences

In a press event to discuss the trial yesterday, the chief investigators said Beat AML would be designed as a nimble trial, able to add and expand arms if treatment regimens seem to be working to develop the knowledge base, and likewise discontinue others if the early signs are not positive.

The researchers have involved FDA reviewers in the design and hold out the hope of, in the event of clearly efficacious treatments, beginning the dialogue on a submission on as few as 25-50 patients.

“For small groups when they see very big differences with good safety, they’re not going to require trials,” Dr Ross Levine, of the Memorial Sloan Kettering haematological malignancy centre, said of the FDA dialogue.

As a potential shortcut to the market, this could prove attractive for players like Amgen (NASDAQ:AMGN), Bayer (OTCPK:BAYRY) and Seattle Genetics (NASDAQ:SGEN), to name three companies with targeted AML projects.

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