Alpha Cognition Announces Positive Data from Pre-Clinical Studies of ALPHA-0602 (Progranulin) for Amyotrophic Lateral Sclerosis (ALS)
Alpha Cognition Inc. (ACOGF) has announced encouraging preclinical results for its ALPHA-0602 ALS gene therapy program. The AAV-based therapy showed significant PGRN expression in motor neurons, reduction in cell death, and improved motor neuron health in both in vitro and in vivo models. Key outcomes include increased cerebrospinal fluid PGRN levels and persistent weight gain in treated TDP-43 transgenic mice. The company plans to advance clinical trials to further validate these findings, marking a critical milestone in addressing ALS treatment.
- Positive preclinical data for ALPHA-0602 in ALS therapy.
- Demonstrated robust PGRN expression and reduced motor neuron cell death.
- Successful transduction of CNS cells in ALS models.
- Treated mice showed persistent weight gain, indicating therapeutic benefits.
- None.
Highlights of the positive proof of concept pre-clinical results demonstrated with ALPHA-0602 in vitro in motor neurons and in vivo in models of ALS, include:
- ALPHA-0602 demonstrated abundant PGRN expression in motor neurons, suggesting a neurotrophic role for PGRN. ALPHA-0602 further increased PGRN levels and decreased motor neuron cell death in in vitro models.
- Using an in vivo model of ALS to further assess the neurotrophic effects of PGRN, ALPHA-0602 reversed the motor neuron toxicity resulting from both decreased levels of TDP-43 and FUS, and expression of ALS related toxic forms of these proteins.
- In an ALS transgenic mouse model caused by a toxic form of TDP-43, ALPHA-0602 administered via adeno-associated virus, resulted in successful viral transduction of CNS cells and substantially increased cerebrospinal fluid (CSF) levels of PGRN.
- ALPHA-0602 treated TDP-43 transgenic mice persistently gained weight throughout the 10-week study, in contrast to untreated transgenic animals who failed to gain weight. Continued weight gain in the face of a significant and sustained toxic insult, is indicative of a therapeutic benefit of ALPHA-0602 expression.
“Collectively, these new insights from our preclinical research further support the development of ALPHA-0602 for the treatment of the motor neuron degeneration associated with ALS,” said
About
ALPHA-1062, is a patented new chemical entity being developed as a new generation acetylcholinesterase inhibitor for the treatment of Alzheimer's disease, with expected minimal gastrointestinal side effects. ALPHA-1062's active metabolites is differentiated from donepezil and rivastigmine in that it binds neuronal nicotinic receptors, most notably the alpha-7 subtype, which is known to have a positive effect on cognition. ALPHA-1062 is also being developed in combination with memantine to treat moderate to severe Alzheimer's dementia and as an intranasal formulation for traumatic brain injury.
ALPHA-0602 (Progranulin) is expressed in several cell types in the central nervous system and in peripheral tissues, it promotes cell survival, regulates certain inflammatory processes, and plays a significant role in regulating lysosomal function and microglial responses to disease. Its intended use for the treatment of neurodegenerative diseases has been patented by the Company and ALPHA-0602 has been granted an Orphan Drug Designation for the treatment of ALS by the FDA.
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https://www.alphacognition.com/
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