Manufacturing of AS-202, an Antisense oligonucleotides for a Phase 1/2 Clinical Trial for Amyotrophic Lateral Sclerosis
Grant Award Details
Grant Type:
Grant Number:
CLIN1-14933
Investigator(s):
Disease Focus:
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
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.