Woolsey Pharmaceuticals Begins Treating First Patient with Higher-Dose BRAVYL in ALS Study

Woolsey Pharmaceuticals, a clinical-stage drug development company based in New York, has achieved a significant milestone in its ongoing research on Amyotrophic Lateral Sclerosis (ALS). The company recently announced the commencement of treatment for the first patient at a dosage of 300 mg/day in its REAL study, which is dedicated to evaluating the effects of BRAVYL on ALS.

Previous findings from the REAL study have shown promising results with a dosage of 180 mg/day of BRAVYL. Participants on this regimen experienced a 15.5% decrease in Neurofilament light chain (NfL) levels from baseline over a six-month period, with a statistical significance of p<0.001. Given that the ALS population typically sees an 11% increase in NfL over six months, this suggests that the 180 mg/day dosage of BRAVYL might potentially reduce NfL by up to 26% compared to a control group.

Furthermore, the study found a correlation between greater decreases in NfL and less deterioration in the ALS Functional Rating Scale-Revised (ALSFRS-R), with a Spearman's coefficient of -0.45 (p=0.028). This implies that the reduction in NfL could be associated with early clinical benefits in ALS patients.

A "propensity matched" analysis was conducted to compare the 12-month clinical outcomes of the REAL study participants with a matched cohort from the Ceftriaxone ALS Study database. The matched cohort consisted of 31 patients selected based on factors such as pre-treatment age, ALSFRS-R scores, and time from onset of ALS. The results showed that the rate of deterioration in ALSFRS-R was improved by 28% (p=0.12) in the REAL study participants compared to the matched controls. Additionally, there was a 42% (p=0.05) slower decline in Slow Vital Capacity (SVC), which measures breathing, and a 50% (p=0.06) slower rate of muscle strength decline. Notably, the lower limb muscle weakening was reduced by 71% (p=0.04), while the upper limb muscle weakening saw a more modest reduction of 37% (p=0.22).

An analysis matching REAL study participants in Australia to the Australian Motor Neurone Disease (MND) Registry, a longitudinal registry, yielded similar findings for ALSFRS-R and SVC. However, muscle strength data could not be analyzed due to the lack of such data in the registry.

Sven Jacobson, CEO of Woolsey Pharmaceuticals, highlighted the importance of this milestone, stating, "The dosing of the first participant at 300 mg is another important program milestone. This next phase will allow us to evaluate the safety and tolerability of a higher dose, in preparation for our planned phase 2b study. Our goal is to continue to advance development of BRAVYL for people living with ALS, in hopes that we can slow the progression of the disease and improve their quality of life."

ALS is a severe neurodegenerative disease characterized by rapid and inevitable decline in patients, with a mean survival time of only two to five years. Any progress in slowing the progression of ALS represents a significant advancement in improving the prognosis and quality of life for those affected by this devastating condition.

Woolsey Pharmaceuticals is committed to pushing the boundaries of medical science, particularly in the treatment of ALS. The company's mission is to usher in a new era of treatment for neurodegenerative diseases, aiming to save and improve the lives of patients in need.

How to obtain the latest research advancements in the field of biopharmaceuticals?

In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!