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This study is a placebo-controlled Phase I study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of the antisense oligonucleotide (ASO) AMX0114 in adult participants with amyotrophic lateral sclerosis (ALS).

The purpose of this study is to determine how safe and tolerable the investigational drug, AMX0114, is for the treatment of amyotrophic lateral sclerosis (ALS).

AMX0114 is given by intrathecal injection, an injection in the lower back into the spinal canal, also known as lumbar puncture. This clinical trial is designed to test if the treatment is safe and tolerable by monitoring the incidence of adverse events, serious adverse events, dose limiting toxicities (DLTs), and incidence of abnormalities in clinical laboratory assessments, vital signs, physical and neurological examinations, and electrocardiograms (ECGs). This trial will also assess the effects of AMX0114 on biomarkers of ALS, including markers of neuronal death and neuroinflammation.

Inclusion Criteria:

1. Ability to understand the purpose and risks of this study, willingness to comply with the study and to provide informed consent in accordance with local laws and regulations.

2. Male or female, at least 18 years of age.

3. Diagnosis of clinically definite or clinically probable ALS, made by a physician who is experienced with management of ALS.

4. Time since onset of first symptom of ALS should be 3 times the upper limit of normal (ULN) and/or total bilirubin > 1.5 times the ULN (obtained within 4 weeks of first dose) except when a result of Gilbert syndrome.

5. Abnormal renal function defined as estimated glomerular filtration rate (eGFR) < 60 mL/min/1.73m2.

6. Other laboratory abnormalities, including abnormalities in platelet count, international normalized ratio, prothrombin time, and activated partial thromboplastin time.

7. Pregnant women (confirmed by a pregnancy test within 7 days prior to first dose) or women currently breastfeeding.

8. Current or previous clinically significant, unstable medical condition (other than ALS), that in the opinion of the Investigator could affect a participant's safety or ability to comply with the study.

9. Significant abnormalities in physical/neurological examination, vital signs, or electrocardiogram (ECG), which in the opinion of the Investigator could affect the safety of the participant.

10. Presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that could affect the participant's ability to provide informed consent or comply with study procedures.

11. Current or previous enrollment in another trial involving use of an investigational

therapy, in most cases within 30 days after the last dose of the study drug, prior

to starting this study.

1. Current or previous treatment with small interfering ribonucleic acid, stem cell

therapy, any ASO or gene therapy.

1. Any contraindications for lumbar puncture or repeated intrathecal injection and/or

underlying disorders that could be affected by intrathecal injections.

1. Prior severe reaction or known hypersensitivity to any part of the Study Drug.