Pipeline Therapeutics has initiated a phase 1/2a trial of PIPE-505, which is a small molecule gamma secretase inhibitor (GSI), in patients who have sensorineural hearing loss (SNHL) associated with hearing speech in noisy environments.

The phase 1/2a trial is anticipated to take in 24 adult patients, and will be carried out as a randomized, double-blind, placebo-controlled, safety, pharmacokinetic, and preliminary efficacy study of unilateral intratympanic PIPE-505 in patients having SNHL associated with speech-in-noise impairment.

The primary outcome measure of the clinical trial is safety, which has been defined to be the occurrence of treatment-emergent adverse events from baseline to a follow-up of three months.

According to the US biopharma company, preliminary efficacy will be evaluated by audiometry and other audiological tests for studying the impact of a single unilateral intratympanic injection of PIPE-505 on processing of signal-to-noise, hearing thresholds, and also electrophysiological measures of cochlear function.

Pipeline Therapeutics will look to release topline results from the early-stage clinical trial in early 2021.

Carmine Stengone – President and CEO of Pipeline Therapeutics said: “At Pipeline, I believe we have assembled the world’s foremost experts in synaptogenesis, remyelination and axonal repair, with PIPE-505 being just the first of many planned drug candidates intended to regenerate critical CNS pathways in large patient populations with hitherto untreatable neurological conditions.

“The dual mechanism of action for PIPE-505, involving repair of the cochlear synapse and the regeneration of outer hair cells critical for hearing quality and sensitivity, uniquely positions this small molecule to address two of the main cochlear elements commonly lost in SNHL. As such, we believe PIPE-505 holds significant promise as a potential therapy for patients suffering from this form of hearing loss, of which we estimate there to be some 10 million in the U.S. alone.”

  Carmine Stengone, PIPE-505, Pipeline Therapeutics, sensorineural hearing loss, USA