Perha Pharmaceuticals

www.perha-pharma.com

Perha Pharmaceuticals is a biotech company identifying, optimizing and characterizing innovative, low molecular weight kinase inhibitors and developing them up to proof-of-concept in man. Perha Pharma focuses its research on two main diseases: cognitive disorders associated with Down syndrome (DS) and Alzheimer’s disease (AD), and hearing loss (HL) induced by ototoxic products. The first proof-of-concept in man should be achieved in both pathologies through niche indications: cognitive deficits in DS and hearing loss induced by cis-platin or aminoglycoside antibiotics. Perha Pharma was co-founded in April 2019 by Dr Laurent MEIJER, Chairman & CSO, former distinguished Director of Research at the CNRS, and Dr Philippe de LAVENNE, CEO, former Vice-President at Actelion Pharmaceuticals. For 40 years, Dr. Meijer dedicated his research to the study of protein kinases and their inhibitors, mostly at the CNRS. Perha Pharma focuses its research & development on two main programs: - Correction of cognitive deficits associated with Down syndrome: Trisomy 21 and Alzheimer's disease share a common therapeutic target involved in cognitive disorders, the DYRK1A protein kinase. The pharmacological inhibitors of this kinase optimized by Perha Pharma are derived from Leucettamine B, extracted from the Leucetta microraphis marine sponge. These molecules correct cognitive impairments observed in three mouse models of DS and three rodent models of AD. Leucettinib 21, our preclinical drug candidate, has been selected among over 500 synthesized Leucettinibs following a stringent multi-parameter go / no go decision tree. Perha Pharma is now preparing for regulatory preclinical studies. -Prevention of hearing loss induced by ototoxic drugs or noise: Perha Pharma-optimized molecules prevent hair cell apoptosis of the inner ear in cell models exposed to cisplatin. Perha Pharma is working at a proof-of-concept using rodents exposed to cisplatin or acoustic trauma.

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Perha Pharmaceuticals is a biotech company identifying, optimizing and characterizing innovative, low molecular weight kinase inhibitors and developing them up to proof-of-concept in man. Perha Pharma focuses its research on two main diseases: cognitive disorders associated with Down syndrome (DS) and Alzheimer’s disease (AD), and hearing loss (HL) induced by ototoxic products. The first proof-of-concept in man should be achieved in both pathologies through niche indications: cognitive deficits in DS and hearing loss induced by cis-platin or aminoglycoside antibiotics. Perha Pharma was co-founded in April 2019 by Dr Laurent MEIJER, Chairman & CSO, former distinguished Director of Research at the CNRS, and Dr Philippe de LAVENNE, CEO, former Vice-President at Actelion Pharmaceuticals. For 40 years, Dr. Meijer dedicated his research to the study of protein kinases and their inhibitors, mostly at the CNRS. Perha Pharma focuses its research & development on two main programs: - Correction of cognitive deficits associated with Down syndrome: Trisomy 21 and Alzheimer's disease share a common therapeutic target involved in cognitive disorders, the DYRK1A protein kinase. The pharmacological inhibitors of this kinase optimized by Perha Pharma are derived from Leucettamine B, extracted from the Leucetta microraphis marine sponge. These molecules correct cognitive impairments observed in three mouse models of DS and three rodent models of AD. Leucettinib 21, our preclinical drug candidate, has been selected among over 500 synthesized Leucettinibs following a stringent multi-parameter go / no go decision tree. Perha Pharma is now preparing for regulatory preclinical studies. -Prevention of hearing loss induced by ototoxic drugs or noise: Perha Pharma-optimized molecules prevent hair cell apoptosis of the inner ear in cell models exposed to cisplatin. Perha Pharma is working at a proof-of-concept using rodents exposed to cisplatin or acoustic trauma.

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