Manufacturing of AS-202, an Antisense oligonucleotides for a Phase 1/2 Clinical Trial for Amyotrophic Lateral Sclerosis

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Grant Award Details

Grant Number:
CLIN1-14933
Investigator(s):
Award Value:
$0
Status:
Closed

Grant Application Details

Application Title:

Manufacturing of AS-202, an Antisense oligonucleotides for a Phase 1/2 Clinical Trial for Amyotrophic Lateral Sclerosis

Public Abstract:
Therapeutic Candidate or Device

AS-202, an antisense oligonucleotide

Indication

Amyotrophic Lateral Sclerosis

Therapeutic Mechanism

AS-202 targets PIKFYVE, a lipid kinase. By inhibiting PIKFYVE activity, it induces exosomal secretion, which robustly clears misfolded proteins including C9ORF72 dipeptide repeat proteins (DPRs), TDP-43 and tau from neurons.

Unmet Medical Need

To date, therapeutic options for ALS have been limited, and disease-modifying drugs remain to be developed. Current FDA approved drugs Riluzole and edaravone are not effective.

Project Objective

Complete cGMP manufacturing of AS-202

Major Proposed Activities

  • cGMP Manufacturing of sufficient AS-202 drug substance for the phase I/II clinical trial
  • Analytic method development and qualification for AS-202 drug substance and stability studies
  • cGMP manufacturing of placebo and AS-202 drug product
Statement of Benefit to California:
Being one of the most popular States, California has many ALS patients. We have met many of them during the annual ALS walk. Most of them suffer ALS from unknown causes with only a small percentage have known genetic mutation. AS-202 is designed to help all ALS patients regardless of the cause of the disease. Our data from animal studies showed that it is safe and likely to be effective. This proposal would help us make the drug product to be used for our planned Phase I/II clinical trials.