BioJiva Reports Results of Pilot Phase 2 Clinical Trial of RT001 in Patients with Amyotrophic Lateral Sclerosis (ALS)
Signals of Clinical Benefit Detected in Small Phase 2 Study, Suggesting Potential to Show Statistically Significant Therapeutic Benefit in Larger, Targeted Trial
Consistent Benefit vs. Placebo in Primary and Secondary Endpoints, With Greater Benefit Witnessed in More Severe Patients
Excerpt from the Press Release:
LOS ALTOS, Calif., Sept. 15, 2022 (GLOBE NEWSWIRE) — BioJiva, a clinical-stage biopharmaceutical company focused on the development of novel, first-in-class therapies for degenerative diseases, today reported data from its completed multicenter, randomized, double-blind, placebo-controlled pilot Phase 2 clinical trial evaluating RT001, the company’s lead development candidate, in patients with amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease). The trial’s prespecified primary endpoint was change from baseline in the revised ALS Functional Rating Scale (ALSFRS-R) following 24 weeks of treatment. At 24 weeks, data demonstrated that patients treated with RT001 experienced less worsening (a 3.3-point reduction from baseline) in ALSFRS-R score as compared to greater worsening (4.6-point reduction from baseline) for the placebo group. Similarly, patients treated with RT001 experienced less worsening (a 13.3-point increase from baseline) in their score on the 40-item ALS assessment questionnaire (ALSAQ40) as compared to the placebo group (a 17.2-point increase from baseline). While suggesting a signal of directional improvement with RT001 treatment, these findings did not achieve statistical significance due to the small size of the study.
This six-month randomized Phase 2 pilot study (NCT04762589), which was followed by a six-month open-label extension during which all patients received RT001, was conducted at four ALS Centers of Excellence in Europe. Investigators enrolled 43 patients with ALS who had symptom duration of less than three years. Enrolled patients had baseline ALSFRS-R scores ranging from 25 to 44, with a mean of 38.2 (+/-5.0.).
Thirty-nine of the 43 enrolled subjects completed the initial 24-week randomized phase, with 32 of those also completing the open-label extension for a full year of treatment.
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