Update: 30 June 2021
- Preclinical data with FTX-6058 showed an increase in HbF levels up to approximately 30% of total haemoglobin.
- Fulcrum has initiated a Phase 1 trial with FTX-6058 in healthy adult volunteers. Dosing has been initiated in the randomized, double-blind, placebo-controlled, multiple ascending dose (MAD) cohorts of the trial. Dosing continues in the single ascending dose (SAD) portion.
- The company anticipates sharing data from this Phase 1 trial in mid-2021 and initiating a clinical trial in sickle cell patients by the end of 2021.
Update: 08 January 2021
No update available
Update: 05 November 2020
An Investigational New Drug application is now in effect in the USA for a Phase 1 trial in healthy adult volunteers to investigate FTX-6058 for sickle cell disease patients.
FTX-6058 is a small molecule designed to increase fetal haemoglobin.
The Phase 1 trial will evaluate the safety, tolerability, and pharmacokinetics of FTX-6058 and will be comprised of four parts.
- Part A will be a randomized, double-blind, placebo-controlled, single ascending dose (SAD) study in up to six cohorts.
- Part B will be a randomized, double-blind, placebo-controlled, multiple ascending dose (MAD) study in up to four cohorts dosed once daily for 14 days.
- Part C will be an open-label pilot food effect study in subjects randomized to take FTX-6058 with and without a high-fat meal, and
- Part D will be an open-label study to evaluate the potential of FTX-6058 to induce CYP3A – an enzyme that helps humans metabolize or digest drugs.