Roche, Basel, Switzerland
Drug Discovery & Development - October 04, 2010
According to the latest statistics from the WHO, there are currently around 35 million people diagnosed with Alzheimer’s disease. Parkinson’s disease is the most common motor disorder, typically affecting the aged (65+) population. At present, about 0.1% to 0.2% of the western population suffer from Parkinson’s disease.
With these numbers in mind, Roche is expanding its CNS portfolio to treat a range of unmet medical needs including Alzheimer’s disease, multiple sclerosis, Parkinson’s disease, treatment-resistant depression, and schizophrenia. In a recent deal with Belgium-based reMYND, Roche licensed a series of novel compounds that act as alpha-synuclein and tau toxicity modulators, with potential for the treatment of Parkinson’s disease (PD) and Alzheimer’s disease (AD).
The compounds are unique in that they have the potential to be disease modifying, while most currently available treatments treat only the symptoms of disease.
Mode of action
The technology platform utilizes cell-based methodologies to identify relevant areas of therapeutic intervention within the lysosomal protein-degradation pathway. This approach is a highly promising as it counteracts toxicity arising from misfolded proteins in the brain and has potential applications in a host of neurodegenerative diseases.
The compounds licensed from reMYND inhibit alpha-synuclein and tau mediated neurotoxicities strongly implicated in PD and AD, respectively. Aggregated alpha-synuclein is the most abundant protein in Lewy bodies, a characteristic pathology in PD, and mutations in alpha-synuclein have been found to be causally associated with this devastating disease. Tau proteins are abundant in neurons in the central nervous system. When tau proteins are misfolded and/or hyperphosphorylated they clump together and form large intracellular aggregates. These so-called tau tangles together with amyloid plaques are pathological hallmarks in Alzheimer's-type dementias.
In preclinical studies, the most advanced compound for the treatment of PD fully inhibited disease progression. This lead PD program could become the first disease-modifying treatment of Parkinson’s disease targeting noxious alpha-synuclein pathology to be taken into clinical development. The reMYND-Roche collaboration aims to further optimize the profile of this compound series including follow-up series to enable further development and clinical testing. Similarly, testing of tau-directed compounds in preclinical models showed very promising results.
With these numbers in mind, Roche is expanding its CNS portfolio to treat a range of unmet medical needs including Alzheimer’s disease, multiple sclerosis, Parkinson’s disease, treatment-resistant depression, and schizophrenia. In a recent deal with Belgium-based reMYND, Roche licensed a series of novel compounds that act as alpha-synuclein and tau toxicity modulators, with potential for the treatment of Parkinson’s disease (PD) and Alzheimer’s disease (AD).
The compounds are unique in that they have the potential to be disease modifying, while most currently available treatments treat only the symptoms of disease.
Mode of action
The technology platform utilizes cell-based methodologies to identify relevant areas of therapeutic intervention within the lysosomal protein-degradation pathway. This approach is a highly promising as it counteracts toxicity arising from misfolded proteins in the brain and has potential applications in a host of neurodegenerative diseases.
The compounds licensed from reMYND inhibit alpha-synuclein and tau mediated neurotoxicities strongly implicated in PD and AD, respectively. Aggregated alpha-synuclein is the most abundant protein in Lewy bodies, a characteristic pathology in PD, and mutations in alpha-synuclein have been found to be causally associated with this devastating disease. Tau proteins are abundant in neurons in the central nervous system. When tau proteins are misfolded and/or hyperphosphorylated they clump together and form large intracellular aggregates. These so-called tau tangles together with amyloid plaques are pathological hallmarks in Alzheimer's-type dementias.
In preclinical studies, the most advanced compound for the treatment of PD fully inhibited disease progression. This lead PD program could become the first disease-modifying treatment of Parkinson’s disease targeting noxious alpha-synuclein pathology to be taken into clinical development. The reMYND-Roche collaboration aims to further optimize the profile of this compound series including follow-up series to enable further development and clinical testing. Similarly, testing of tau-directed compounds in preclinical models showed very promising results.
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