ALS: ALPHA-602

The Alpha-602 product originated almost a decade ago when it was discovered by two professors at McGill University in Montreal that a protein called progranulin seemed to show activity for several neurological disorders. Progranulin is a large protein that was found to be present in virtually all living animals and seemed to be used by the body for multiple tasks. Upon further investigation, scientists discovered that the large molecule was made of smaller polypeptides or subunits, referred to as Granulin Epithelin Modules (“GEMs”). The Company’s Alpha-602 development program is at a relatively early stage, and operations are currently focused on Alpha-1062.

Current treatments for ALS are relatively new from an approval perspective and appear to have limited effectiveness. It is the Company’s view that all current approved treatments are considered to be extremely expensive and not well received by patients, payers (insurance companies) or caregivers.

Alpha-602 Pre-Clinical Development

To investigate Alpha-602’s potential as the intact molecule, ACI designed a program that would stimulate the overproduction of the protein in animals with specific neurological disorders. Initial work with animal models of ALS were completed in 2013 and indicated that progranulin did in fact reverse the disease process. However, an approach using methods of upregulating the protein would require significant investment and ultimately the use of gene therapy. As a result, ACI began to investigate an alternative route to development.

Alpha-602 Regulatory development

Once a GEM formulation has been identified and validated in pre-clinical testing in relevant animal models, the pre-IND document package would be prepared by the Company for FDA submission. At this point in the Alpha-602 development program the focus would be on a GEM formulation to treat ALS. In March 2019 the Company was granted an Orphan Drug Designation by the FDA for the use of Alpha-602 in the treatment of ALS. The Orphan Drug Designation has a number of significant benefits including:

  1. tax credits of 50% off the clinical drug testing cost awarded upon approval;
  2. eligibility for market exclusivity for seven years post approval; and
  3. waiver of New Drug Application and biologics license application fees, which would be approximately US$2,200,000.

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