Woolsey Pharmaceuticals Announces Treatment Of First Patient with Higher-Dose BRAVYL in The REAL Amyotrophic Lateral Sclerosis (ALS) Study (NCT05218668)

NEW YORK, July 8, 2024 /PRNewswire/ -- Woolsey Pharmaceuticals, a clinical-stage drug development company, announced that the first patient has started treatment at 300 mg/day in the REAL study, which is evaluating the effects of BRAVYL in Amyotrophic Lateral Sclerosis (ALS).

As recently announced, participants treated with 180 mg/day BRAVYL in the REAL study experienced a 15.5% decrease (improvement) in NfL from baseline to 6 months (p<0.001). Since NfL in the ALS population tends to increase by a mean of 11% in 6 months, this suggests that 180 mg/day BRAVYL could potentially reduce NfL up to 26% versus a control group.

Moreover, greater decreases in NfL were correlated with less deterioration on the ALSFRS-R (Spearman's coefficient = -0.45, p=0.028) implying that early clinical benefits of reducing NfL may be seen contemporaneously with the NfL reductions by BRAVYL.

A "propensity matched" analysis was performed wherein 12-month clinical outcomes from REAL were compared with those from a matched cohort selected by identifying 31 patients from the Ceftriaxone ALS Study database that were most similar (based on pre-treatment age, ALSFRS-R, time from onset of ALS, and other important parameters) to each REAL patient.

The rate of deterioration in ALSFRS-R was improved in REAL vs. the matched controls by 28% (p=0.12). REAL patients also had a 42% (p=0.05) slower deterioration in SVC (breathing) and a 50% (p=0.06) slower rate of muscle strength decline, driven mostly by lower limb muscles with a 71% (p=0.04) reduction in weakening and a more modest 37% (p=0.22) reduction in weakening of upper limb muscles. An analysis that matched REAL patients recruited in Australia to the Australian MND Registry (a longitudinal registry, not a study) yielded directionally similar ALSFRS-R and SVC findings while muscle strength could not be analyzed as the registry does not contain such data.

"The dosing of the first participant at 300 mg is another important program milestone," notes Sven Jacobson, CEO of Woolsey. "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."

ABOUT ALS

ALS is a fatal neurodegenerative disease characterized by inevitable, and often rapid, decline in patients as the disorder advances (mean survival time is only two to five years). Accordingly, the ability to impede any worsening represents a meaningful advancement in efforts to enhance the prognosis and quality of life of individuals impacted by this devastating condition.

ABOUT WOOLSEY PHARMACEUTICALS

Woolsey Pharmaceuticals is expanding the boundaries of medical science. Our lead indication is Amyotrophic Lateral Sclerosis (ALS), a progressive and debilitating disease that ultimately is fatal. Our mission is to help usher in a new era of neurodegenerative disease treatment, saving and improving the lives of patients in need.

SOURCE Woolsey Pharmaceuticals, Inc.