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Alpha Cognition Announces Positive Data from Pre-Clinical Studies of ALPHA-0602 (Progranulin) for Amyotrophic Lateral Sclerosis (ALS)

Alpha Cognition Inc. (TSX-V: ACOG) (OTCQB: ACOGF) (“Alpha Cognition” (ACI), or the “Company”), a biopharmaceutical company committed to developing novel therapies with the potential to transform the lives of people with debilitating neurodegenerative disorders, is pleased to announce positive preclinical data from their ALPHA-0602 ALS gene therapy program. These data underscore the robust preclinical evidence supporting Alpha Cognition’s AAV-based gene therapy approach to treating ALS and highlight the Company’s strategy to validate these data in planned clinical trials.

Denis Kay, the Company’s Chief Scientific Officer, commented: “ALS is a devastating disease with patients in urgent need of effective therapies. ALPHA-0602 is designed to increase brain progranulin (PGRN) levels and provide support for the motor system through the reduction of TDP-43 and FUS pathology, as well as reducing neuroinflammation and the effects of oxidative stress, associated with ALS. These benefits have been observed in preclinical studies and strongly support the continued development of ALPHA-0602 for the treatment of ALS.”

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 Michael McFadden, Alpha Cognition’s Chief Executive Officer. “This represents an important milestone for our company, and we plan to assess the effect of progranulin expression on the neuropathology associated with this animal model as a next step in our development program.”

About Alpha Cognition Inc.

Alpha Cognition Inc. is a clinical stage, biopharmaceutical company dedicated to developing treatments for patients suffering from neurodegenerative diseases, such as Alzheimer's disease and Amyotrophic Lateral Sclerosis (ALS), for which there are limited treatment options.

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.

Neither TSX Venture Exchange (the “TSX-V”), OTC Markets Group, nor the TSX-V’s Regulation Services Provider (as that term is defined in policies of the TSX-V) accepts responsibility for the adequacy or accuracy of this release.

Forward-looking Statements

This news release is not, and under no circumstances is to be construed as, an advertisement or a public offering of securities. No securities commission or similar authority in Canada or in any other jurisdiction has reviewed or in any way passed upon this news release or the merits of the securities described herein and any representation to the contrary is an offence.

This news release includes forward-looking statements within the meaning of applicable securities laws. Except for statements of historical fact, any information contained in this news release may be a forward‐looking statement that reflects the Company’s current views about future events and are subject to known and unknown risks, uncertainties, assumptions and other factors that may cause the actual results, levels of activity, performance or achievements to be materially different from the information expressed or implied by these forward-looking statements. Forward‐looking statements can be identified by the words “may,” “might,” “will,” “could,” “would,” “should,” “expect,” “intend,” “plan,” “objective,” “anticipate,” “believe,” “estimate,” “predict,” “project,” “potential,” “target,” “seek,” “contemplate,” “continue” and “ongoing,” or the negative of these terms, or other comparable terminology intended to identify statements about the future. Forward‐looking statements in this news release include statements regarding the Company’s business strategy, market size, potential growth opportunities, capital requirements, clinical development activities, the timing and results of clinical trials, regulatory submissions, potential regulatory approval and commercialization of the technology. Although the Company believes that we have a reasonable basis for each forward-looking statement, we caution you that these statements are based on a combination of facts and factors currently known by us and our expectations of the future, about which we cannot be certain. The Company cannot assure that the actual results will be consistent with these forward-looking statements as a result of known and unknown risks, uncertainties, assumptions and other factors. These risks, uncertainties, assumptions and other factors include those associated with clinical studies and manufacturing, as well as development and commercialization of the Company’s products; the need for additional financing to maintain operations; risks posed by the economic and political environments in which the Company operates and intends to operate; market instability due to the COVID-19 pandemic; the potential for losses arising from the expansion of operations into new markets; increased competition; assumptions regarding market trends and the expected demand and desires for the Company’s products and proposed products; reliance on industry manufacturers, suppliers and key personnel; the failure to adequately protect intellectual property; a failure to adequately manage future growth; adverse market conditions; and failure to satisfy ongoing regulatory requirements or obtain regulatory approvals. These forward‐looking statements speak only as of the date of this news release and, other than as required by applicable securities laws, the Company undertakes no obligation to revise or update any forward‐looking statements, even if new information becomes available in the future.

This news release may also contain estimates and other statistical, market and industry data from independent parties or made by the Company relating to our industry. This data involves a number of assumptions and limitations, and you are cautioned not to give undue weight to such estimates. We cannot guarantee the accuracy and completeness of information from third party sources.


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