Trophos SA, a clinical stage pharmaceutical company developing innovative therapeutics for indications with under-served needs in neurology and cardiology, announced today that the company is increasing the focus of its development programs on neuroprotection and cardioprotection. The Company has a novel and proprietary cholesterol-oxime based pipeline of drug candidates that enhance the function and survival of stressed cells via modulation of dysfunctional mitochondria, through interactions at the permeability transition pore (mPTP). The announcement follows the award of nearly USD 9 million in grants associated with its lead drug candidate TRO19622

Advancing programs include:

MitoTarget: a Trophos led consortium has been awarded a EUR 6 million grant to study restorative approaches for therapy of neurodegenerative diseases, notably including support for a clinical efficacy study of TRO19622 in Amyotrophic Lateral Sclerosis (ALS) patients.

TRO19622: development in Spinal Muscular Atrophy (SMA), an orphan neurodegenerative disorder, continues following successful phase Ib study in SMA patients.

The Company will continue its development programmed in SMA with TRO19622 following a phase Ib PK and tolerability study in SMA patients ranging from 6-25 years old, which demonstrated good tolerability and established the pharmacokinetic characteristics of the molecule in children. Discussions are ongoing with the EMEA regarding appropriate trial design to demonstrate efficacy and allow registration in this indication. This program has received financial support from the Association Française contre les Myopathies (AFM).

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