A Study to Evaluate Efficacy, Safety and Tolerability of CK-2127107 in Patients With Amyotrophic Lateral Sclerosis (ALS)

Enrolled participants will be dosed with CK-2127107 150, 300, 450 mg or placebo twice daily
(300, 600, 900 mg/day or placebo) for a period of 12 weeks.

Inclusion Criteria:

- Diagnosis of familial or sporadic ALS ≤ 24 months prior to screening

- Upright Slow Vital Capacity (SVC) ≥ 60% of predicted for age, height and sex at
screening

- Able to swallow tablets

- A caregiver (if one is needed)

- Able to perform reproducible pulmonary function tests

- Pre-study clinical laboratory findings within the normal range or, if outside the
normal range, deemed not clinically significant by the Investigator

- Male patients who have not had a vasectomy and confirmed zero sperm count must agree
after receiving the first dose of study drug until 10 weeks after the last dose to
either use acceptable methods of contraception or abstain from sex

- Female patients must be post-menopausal or sterilized or must not be breastfeeding,
have a negative pregnancy test, have no intention to become pregnant during the study
and use acceptable methods of contraception or abstain from heterosexual intercourse
from Screening until 10 weeks after last dose of study drug

- Patients must be either on riluzole for at least 30 days prior to screening or have
not taken riluzole for at least 30 days prior to screening and not planning to start
riluzole during the course of the study.

Exclusion Criteria:

- At the time of screening, any use of non-invasive ventilation (NIV), e.g. continuous
positive airway pressure [CPAP], noninvasive bi-level positive airway pressure [NPPV]
or noninvasive volume ventilation [NVV]for any portion of the day, or mechanical
ventilation via tracheostomy, or on any form of oxygen supplementation

- Neurological impairment due to a condition other than ALS

- Presence at screening of any medically significant cardiac, pulmonary, GI,
musculoskeletal, or psychiatric illness that might interfere with the patient's
ability to comply with study procedures or that might confound the interpretation of
clinical safety or efficacy data

- Has taken any investigational study drug within 30 days or five half-lives of the
prior agent, whichever is longer, prior to dosing

- Known to have received CK-2127107 or tirasemtiv in any previous clinical trial

- Has received or is considering receiving during the course of the study any form of
stem cell therapy for the treatment of ALS

- Has received or is considering receiving during the course of the study any form of
gene therapy for the treatment of ALS

- Has received or is considering obtaining during the course of the study a
diaphragmatic pacing system

- History of substance abuse within the past 2 years

- Use of certain medications