An anonymous director reports

TRANSITION THERAPEUTICS' ELND005 SHOWED NO PROLONGATION OF QT INTERVAL

Transition Therapeutics Inc. has released results from a thorough QT study in which no QT effects were observed at supra-therapeutic single doses of neuropsychiatric drug candidate, ELND005. A tQT study is a specialized clinical trial required by the U.S. Food and Drug Administration for the approval of most drugs in development. Transition's wholly owned subsidiary, Transition Therapeutics Ireland Ltd., presented the tQT study data at the Clinical Trials in Alzheimer's Disease conference on Nov. 21, 2014.

From a safety perspective, drugs that have no QT prolongation effects are particularly desirable for administration to an elderly Alzheimer's disease population. AD patients are especially challenging for novel drug development, since they typically have multiple medical co-morbidities and receive multiple medications. The use of some anti-psychotics and anti-depressants may increase the risk of QT prolongation, arrhythmias and sudden death in AD patients.

Single doses, 2,000 milligrams and 7,000 milligrams of ELND005, appeared to be safe and well tolerated by healthy subjects in this study. ELND005 exposures in this study are fivefold higher than the loading dose of 1,000 milligrams BID in the current agitation and aggression in AD clinical study (AG201). These ELND005 doses did not have any clinically relevant effects on electrocardiogram parameters and the study represents a clearly negative thorough QT study.

About the ELND005 tQT study

The QT interval represents the amount of time the heart's electrical system takes to repolarize, or recharge, after each beat. As prolongation of the QT interval may increase the risk for cardiac arrhythmias, the FDA requires a tQT study for most new drugs in development. A tQT study is a specialized clinical trial designed to assess whether an investigational medication has the potential to prolong the QT interval.

Study design

The tQT study was a partially double-blind (in regard to ELND005 treatment), randomized, placebo- and active controlled, four-arm crossover trial in 52 healthy male and female subjects using single doses during separate treatment periods, and undergoing intensive ECG monitoring. Each subject received a single oral dose of the following four treatments: 2,000 milligrams ELND005, 7,000 milligrams ELND005, placebo as a negative control and 400 milligrams moxifloxacin as a positive control. The central ECG laboratory was blinded to subjects, time and all treatments (including positive control) when performing the ECG reading.

About ELND005

ELND005 is an orally bioavailable small molecule that is being investigated for multiple neuropsychiatric indications on the basis of its proposed dual mechanism of action, which includes beta-amyloid anti-aggregation and regulation of brain myo-inositol levels. An extensive clinical program of phase 1 and phase 2 studies has been completed with ELND005 to support clinical development, including the published phase 2 study ELND005-AD201 in Alzheimer's disease. ELND005 is also being studied as a potential treatment of agitation and aggression in Alzheimer's disease (study ELND005-AG201), and as a therapy for those with Down syndrome (study ELND005-DS-201). ELND005 has received fast-track designation from the psychiatry division of the U.S. Food and Drug Administration for its potential as a treatment of neuropsychiatric symptoms (including agitation) in AD.

We seek Safe Harbor.