Earlier this month, during the American Society of Clinical Oncology (ASCO) annual meeting in Chicago, Daiichi Sankyo Co. Ltd. provided a sneak peek at data from the pivotal phase III QUANTUM-R study of single-agent quizartinib in patients with relapsed/refractory acute myeloid leukemia (r/rAML) with FLT3-ITD mutations. But the Tokyo-based company saved details for the 23rd Congress of the European Hematology Association (EHA) in Stockholm, where the data on the oral selective FLT3 inhibitor were presented as a late-breaking oral presentation during the plenary program.

As Agios Pharmaceuticals Inc. awaits an FDA decision on ivosidenib (AD-120) in relapsed or refractory acute myeloid leukemia (r/r AML) – the isocitrate dehydrogenase 1 mutant, or IDH1m, inhibitor is under priority review with an Aug. 21 PDUFA date – the company moved AG-348, its selective small-molecule activator of the pyruvate kinase-R (PKR) enzyme, into a second phase III trial that will lay the groundwork for another potential filing. The randomized, double-blind, placebo-controlled trial is expected to enroll approximately 80 adults with pyruvate kinase (PK) deficiency who do not receive regular blood transfusions, seeking to show that AG-348 produces sustained increase in hemoglobin (Hb) in those individuals.

For the treatment of breast cancer, the biggest newsmaker at the 2018 American Society of Clinical Oncology (ASCO) annual meeting in Chicago had nothing to do with drug development. Instead, the findings came from a National Cancer Institute (NCI)-sponsored study directing physicians to use less treatment, not more, in most women with a common form of early stage disease.

Earlier this month, during the American Society of Clinical Oncology (ASCO) annual meeting in Chicago, Daiichi Sankyo Co. Ltd. provided a sneak peek at data from the pivotal phase III QUANTUM-R study of single-agent quizartinib in patients with relapsed/refractory acute myeloid leukemia (r/rAML) with FLT3-ITD mutations. But the Tokyo-based company saved details for the 23rd Congress of the European Hematology Association (EHA) in Stockholm, where the data on the oral selective FLT3 inhibitor were presented as a late-breaking oral presentation during the plenary program.

CoA Therapeutics represents the 19th subsidiary in three years for parent company Bridgebio Pharma Inc., adding a small-molecule approach to increase levels of coenzyme-A, or CoA – thus, the startup's intuitive name – in genetic disorders associated with CoA deficiency. Through its model of housing each therapeutic effort in an individual subsidiary supported by shared central resources, Bridgebio committed funds to drive CoA's lead program through human proof of concept. 

On the heels of an FDA orphan drug designation for lead compound PU-H71 in myelofibrosis, privately held Samus Therapeutics Inc. moved the small molecule into a phase Ib dose-escalation study in patients with the bone marrow disorder. PU-H71 is designed to target the epichaperome, a protein complex present in more than half of the cancers tested by the company, a spinout of Memorial Sloan Kettering Cancer Center (MSKCC).

Existing investors stepped up with enthusiasm to an oversubscribed $65 million internal round at Nimbus Therapeutics that will enable the Cambridge, Mass.-based company to accelerate pipeline programs and examine additional targets in metabolic, immuno-oncology (I-O) and autoimmune disease. Each of the company's current investors participated in the round, including Atlas Venture, SR One, Lilly Ventures, Bill Gates, Pfizer Venture Investments, Lightstone Ventures and Schrödinger.