Developing drugs to protect nerve cells in neurodegenerative diseases
Neurodegenerative diseases involve protein misfolding, cellular stress and impaired cellular waste disposal. Rare inherited neuropathies such as Charcot-Marie-Tooth disease cause progressive nerve damage, muscle weakness and sensory loss, yet lack disease-modifying treatments.
Enemsa Pharma is developing icerguastat, alone and in drug combinations, to protect nerve cells in neurodegenerative diseases. Its combination with ambroxol targets complementary cellular pathways, with development focused on Parkinson’s disease linked to GBA1 mutations.
What stands out about Enemsa Pharma
Secured exclusive US rights from the University of Chicago to develop icerguastat for demyelinating diseases, including CMT.
Source ↗Holds FDA orphan drug designations for icerguastat in amyotrophic lateral sclerosis and Charcot-Marie-Tooth disease.
Source ↗Reports preclinical synergy between icerguastat and ambroxol in in-vitro models of ALS and Parkinson’s disease.
Source ↗Led InFlectis BioScience as COO and CEO before co-founding Enemsa Pharma; Pierre Miniou also served on Vivalis’s executive committee.
Source ↗Co-founders previously built other biotech companies: Pierre Miniou co-founded InFlectis BioScience in 2013, and Philippe Jaffré co-founded Xenothera.
Source ↗Co-founder Emmanuelle Abgueguen holds a biology PhD and led IFB-088 development from preclinical proof of concept to an ALS phase 2 trial.
Source ↗Co-founder Philippe Jaffré held financial planning and analysis roles in Novartis’s Medical Nutrition division.
Source ↗Icerguastat was evaluated with riluzole in InFlectis BioScience’s completed exploratory phase 2 trial in 51 people with bulbar-onset ALS.
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