Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Tirasemtiv · 2 trials · 2 indications
The number of participants with adverse events was used as the measure for the long-term safety and tolerability of tirasemtiv.
SVC was measured using a spirometer (in units of liters). Following 3 to 5 breaths at rest, the patients were instructed to take as deep an inspiration as possible followed by a maximum exhalation (blowing out all the air in their lungs). Values obtained were converted to % predicted values (ie, the test result as a percent of predicted values for patients of similar demographic and baseline characteristics \[eg, height, age, sex\], based on Knudson 83 normative values).
| Arm | Type | Description |
|---|---|---|
| Delayed Start Treatment | EXPERIMENTAL | The Delayed Start Treatment group consisted of patients who received placebo in CY 4031 and tirasemtiv in CY 4033. |
| Early Start Treatment | EXPERIMENTAL | The Early Start Treatment group consisted of patients who received tirasemtiv in both CY 4031 and CY 4033. |
| Group 1 - Placebo | PLACEBO_COMPARATOR | Day 1 through Week 48: 2 placebo tablets twice daily |
| Group 2 - 250 mg tirasemtiv | EXPERIMENTAL | Day 1 through Week 48: 1 tablet of tirasemtiv (125 mg) and 1 tablet of matching placebo in the AM and 1 tablet of tirasemtiv (125 mg) and 1 tablet of matching placebo in the PM |
| Group 3 - 375 mg tirasemtiv | EXPERIMENTAL | Day 1 through Week 2: 1 tablet of tirasemtiv (125 mg) and 1 tablet of matching placebo in the AM and 1 tablet of tirasemtiv (125 mg) and 1 tablet of matching placebo in the PM; Weeks 3 through 48: 1 tablet of tirasemtiv (125 mg) and 1 tablet of matching placebo in the AM and 2 tablets of tirasemtiv (250 mg) in the PM |
| Group 4 - 500 mg tirasemtiv | EXPERIMENTAL | Day 1 through Week 2: 1 tablet of tirasemtiv (125 mg) and 1 tablet of matching placebo in the AM and 1 tablet of tirasemtiv (125 mg) and 1 tablet of matching placebo in PM; Weeks 3 and 4: 1 tablet of tirasemtiv (125 mg) and 1 tablet of matching placebo in the AM and 2 tablets of tirasemtiv (250 mg) in the PM; Weeks 5 through 48: 2 tablets of tirasemtiv (250 mg) in the AM and 2 tablets of tirasemtiv (250 mg) in the PM |
| Name | Type | Description |
|---|---|---|
| tirasemtiv | DRUG | Oral |
| Placebo tablets | DRUG | - |
Inclusion Criteria: * Able to comprehend and willing to sign an Informed Consent Form (ICF). If verbal consent is given, a Legal Designee of the patient must sign the ICF form * Completed participation on study drug and the Follow-Up Visit in the CY 4031 study * Male patients, who have not had a va...
Tirasemtiv is an investigational small molecule being developed for the treatment of Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's disease. It is intended for use in patients with this progressive neurodegenerative condition that affects nerve cells in the brain and spinal cord.
Tirasemtiv is being developed by Cytokinetics, Incorporated, a biopharmaceutical company focused on muscle biology. The company's stock is traded under the ticker symbol CYTK on the NASDAQ stock exchange.
Tirasemtiv is in Phase 3 clinical development. It has completed a Phase 3 trial known as VITALITY-ALS (NCT02496767), which enrolled 744 participants with ALS. The drug is still investigational and has not been approved by regulatory authorities.
Tirasemtiv has been studied in two completed Phase 3 clinical trials. The primary trial, VITALITY-ALS (NCT02496767), enrolled 744 patients with ALS across multiple countries. A follow-up study (NCT02936635) enrolled 280 patients who had completed the VITALITY-ALS trial.
Tirasemtiv is a small molecule that targets the sarcomere, the fundamental contractile unit of skeletal muscle. By sensitizing the sarcomere to calcium, it is designed to increase muscle force in response to nerve signals, potentially counteracting the muscle weakness seen in ALS.