Drugs

Agios Pharma Stops Drug Development After FDA Hold

Agios Pharmaceuticals has pulled the plug on its development program involving its second pyruvate kinase-R activator, AG-519, and withdrew its investigational new drug application 12/15 following a verbal notification of an FDA clinical hold. “These decisions do not affect the company’s ongoing global Phase 2 study (DRIVE PK) for AG-348, a novel, first-in-class activator of both wild-type (normal) and mutated PKR enzymes,” it says. Agios is advancing AG-348 into pivotal trials as the first potential disease-modifying treatment for pyruvate kinase (PK) deficiency. “Based on our clinical experience with DRIVE PK, we are developing a registration path for AG-348 in adult PK deficiency patients and plan to discuss this strategy with regulators,” Agios adds.

DRIVE PK is an ongoing global Phase 2, open-label safety and efficacy trial and the first study evaluating AG-348 in adult, transfusion-independent patients with PK deficiency. The study target enrollment has been reached with a total of 52 patients, according to the company. In total, 124 patients and healthy volunteers have been treated with AG-348 since studies were initiated in 2014. “As of the 9/23/2016 data cut-off, AG-348 was well-tolerated with the majority of treatment-related adverse events Grade 1-2; the most frequent being headache, nausea and insomnia,” it says. “AG-348 also demonstrated clinically relevant, rapid and sustained hemoglobin increases in 15 of 26 patients with at least one missense mutation, and in 15 out of 32 patients overall.”

As for AG-519, the drug was evaluated in a Phase 1 study in the United Kingdom to assess safety, tolerability, pharmacokinetics, pharmacodynamics and bioavailability. “A previously disclosed case of drug-induced cholestatic hepatitis occurred in the bioavailability portion of the study and this volunteer continues to be monitored,” Agios says. “No additional adverse events have been reported. In addition, AG-519 was undergoing a palatability (taste test) study in volunteers in the United States to develop a formulation for potential future development. In total, 98 volunteers have received AG-519 and no volunteers or patients are currently receiving the drug.”

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome