Phase 1 Safety Run-in Study and Phase 1b Randomized, Double Blinded, Placebo Controlled Trial

Minimum Age:

18 Years

Maximum Age:

95 Years

Inclusion Criteria:

- Ability of the subject or his/her legally authorized representative to provide informed consent.

- Adult ALS subjects (≥18 years of age)

- Diagnosis of ALS, according to the Revised El Escorial Criteria for ALS

- Subjects with disease onset ≤ 5 years

- Upright (sitting position) Slow Vital Capacity (SVC) as adjusted for sex, age and height ≥ 50% predicted

- Subjects must have documented ALSFRSR score of 36-45 at baseline.

- Subjects taking concomitant Riluzole or Edaravone or Albrioza at study entry must be on a stable dose for ≥ 30 days prior to the first dose of study treatment (Day 1).

- Screening values of coagulation parameters including platelet count, international normalized ratio (INR), prothrombin time (PT), and activated partial thromboplastin time (APTT) should be within normal ranges.

- Agree to practice highly effective contraception during the study and continue contraception for 90 days after their last dose of study treatment.

Exclusion Criteria:

- Uncontrolled infection, not responding to appropriate antimicrobial agents after seven days of therapy. The Protocol medical monitor is the final arbiter of eligibility.

- Antiplatelet or anticoagulant therapy within the 14 days prior to Day 1 or anticipated use during the study, including but not limited to daily aspirin including low dose aspirin (defined as ≤ 150 mg/day), clopidogrel, dipyridamole, warfarin, dabigatran, rivaroxaban and apixaban

- Clinically significant low platelet count (defined as < 100,000/mm3), coagulation tests, or laboratory abnormalities that would render a subject unsuitable for inclusion

- Unwillingness to comply with study procedures, including follow-up, as specified by this protocol, or unwillingness to cooperate fully with the Investigator

- Have any other conditions, which, in the opinion of the Investigator would make the subject unsuitable for inclusion, or could interfere with the subject participating in or completing the study

- Concurrent participation in any other interventional clinical study

- Treatment with another investigational drug, biological agent, or device, including, but not limited to sodium phenylbutyrate, within 1 month of Screening, or 5 half-lives of investigational agent, whichever is longer

- Treatment of cancer in the last 5 years (except in situ carcinoma of the cervix or basal cell carcinoma)

- Female subjects who are pregnant or currently breastfeeding

- Other unspecified reasons that, in the opinion of the Investigator or Sponsor, make the subject unsuitable for enrollment.