May 17 Clinical Quick Takes: Achillion Phase III plans for PNH combo; plus NCI, MorphoSys

Achillion to start Phase III of PNH combo in 1H20
Achillion Pharmaceuticals Inc. (NASDAQ:ACHN) said it will start in 1H20 a Phase III trial evaluating oral CFD inhibitor ACH-4471 in combination with C5 inhibitors to treat paroxysmal nocturnal hemoglobinuria. The company reported its plans along with interim data from 11 PNH patients in a 24-week Phase II showing that ACH-4471 plus IV Soliris eculizumab increased mean hemoglobin by about 2 g/dL at week 4; the combo also reduced the need for blood transfusions from 34 transfusions in the 24 weeks prior to screening to only one transfusion during the trial (see "Achillion Down After Discontinuing PNH Monotherapy").

Pediatric MATCH trial links quarter of patients to targeted therapy
An early analysis of the Phase II NCI-COG Pediatric Molecular Analysis for Therapy Choice (Pediatric MATCH) trial showed that 24% of patients whose solid tumors were evaluated for genetic alterations were eligible to receive one of 10 targeted therapies being tested in the trial; the researchers had anticipated a 10% hit rate. The trial is enrolling up to 1,500 cancer patients ages 1-21 to determine whether cancer therapies can be targeted effectively to a predefined set of genetic mutations independent of cancer type or site (see "FDA’s Pediatric Target Picks")...