Drug intelligence / Profile preview

PIPE-505

Development stage
Phase 2
Lead developer
Contineum Therapeutics
Modality
Small Molecules
Administration
Intratympanic
01

Overview

PIPE-505 is a small molecule gamma secretase inhibitor (GSI) developed by Contineum Therapeutics (formerly Pipeline Therapeutics) for the treatment of sensorineural hearing loss (SNHL), particularly associated with speech-in-noise impairment. The drug acts through dual mechanisms involving the repair of cochlear synapses and regeneration of outer hair cells, both critical for hearing quality and sensitivity. Mechanistically, it inhibits gamma secretase substrates DCC and Notch, facilitating spiral ganglion neurite growth via the Netrin/DCC pathway to regenerate inner hair cell ribbon synapses, and increasing Atoh1 expression via reduced Notch signaling to promote outer hair cell formation. This regenerative approach aims to restore auditory function in patients with SNHL[1][3][4][6].

Other names
PIPE 505PIPE505PIPE-505
02

Targets

NOTCH (Neurogenic locus notch homolog protein 3)GSEC (Gamma-Secretase Complex)DCC (Deleted in colorectal carcinoma protein)

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