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Woolsey Pharmaceuticals Emerges from Stealth Mode to Announce Patients Enrolled in Two New CNS Studies

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One trial seeks to reduce wandering in dementia patients; the other targets two rare, deadly neurodegenerative diseases.

NEW YORK–(BUSINESS WIRE)–Woolsey Pharmaceuticals, a clinical-stage company developing treatments for neurodegenerative diseases, today announced dosing of the first patient with BRAVYL in its FOUND (Fasudil fOr Uncontrolled waNDering) study for Alzheimer’s and Vascular Dementia patients who have wandering behaviors of elopement and/or getting lost. The Company also announced the first patient in its ROCKIT-1 (ROCK Inhibitor in Tauopathies – 1) trial of BRAVYL in Progressive Supranuclear Palsy (PSP) and Corticobasal Syndrome (CBS) patients has commenced treatment.

ABOUT WANDERING IN DEMENTIA PATIENTS

Dementia is the leading cause of dependency and disability amongst older adults. Wandering is one of the most dangerous dementia-related behaviors. Earlier this month, in the midst of a massive winter storm that slammed the Northeast, a 67-year-old Allentown, PA woman who had Alzheimer’s disease, wandered away from her home. After a frantic search, she was found four blocks away, lying in a snowbank. She was pronounced dead from hypothermia a few hours later.

The stress experienced by families and caregivers when a person living with dementia wanders and becomes lost is significant. Wandering represents the “tipping point” for many caregivers, as they are more likely to report significant depression and burden — which, in turn, can lead to the gut-wrenching decision to place a loved one in a nursing home or locked memory care unit.

BRAVYL (oral fasudil), is a potent inhibitor of Rho-kinase (ROCK) and is believed to reduce wandering behaviors by improving blood flow in the hippocampus, a part of the brain that plays a major role in navigation, learning, and memory. BRAVYL is non-sedating.

BRAVYL has been tested in a small study of dementia patients who had symptoms of wandering. The disappearance of wandering symptoms in these patients was observed shortly after the start of treatment. Not long after discontinuation of treatment, patients returned to their pretreatment (wandering) state. This on-off effect was replicated more than once in these subjects.

THE FOUND STUDY

FOUND is a phase 2a study that will evaluate whether BRAVYL improves wandering behaviors of excess movement and pacing, cognition, memory, neuropsychiatric symptomatology, and caregiver/nursing staff burden, among other endpoints. Participation will be for up to 26 weeks. FOUND is currently recruiting at multiple sites in the United States and Australia.

“Wandering away from home or a care facility is dangerous for people with dementia and stressful for families and caregivers who must continually monitor the person’s movement 24 hours a day,” said Dr. David Thomas, professor at Eastern Michigan University and noted expert on dementia-related wandering. “If results from an earlier study could be replicated here, it would certainly represent a historic and momentous step forward in the quality of life and care of people who wander.”

“We are pleased to enroll the first patient in the FOUND trial, which will further our understanding of the potential role of BRAVYL for the treatment of dementia patients who wander,” said David Geliebter, Co-Founder and Executive Chairman of Woolsey Pharmaceuticals. “Today’s announcement is an important milestone in bringing more than just hope to these patients and their loved ones.”

Data readout in the study is anticipated in 2022.

ABOUT PSP and CBS

Progressive supranuclear palsy (PSP) and corticobasal syndrome (CBS) are rare neurodegenerative disorders associated with the accumulation of insoluble deposits of predominantly 4 microtubule binding domain repeat (4R) tau protein in specific brain regions.

PSP affects movement, gait and balance, speech, swallowing, vision, mood and behavior, and thinking. One of the classic signs of the disease is an inability to aim and move the eyes properly, which individuals may experience as blurring of vision. CBS patients present with bizarre rigidity and dystonia (a condition in which a person’s muscles contract uncontrollably). There are currently no effective therapies for either disorder. From the onset of PSP and CBS, the lifespan of patients averages 7 years.

Levels of Rho-kinase (ROCK) are elevated in both PSP and CBS patients, and is thought to be involved in the accumulation of tau aggregates. It is hypothesized that BRAVYL, a ROCK inhibitor, will reduce levels of ROCK activity in the brains of PSP and CBS patients, reducing 4R tau phosphorylation and aggregation, as well as inducing clearance of existing 4R tau aggregates that are the hallmark of the two diseases.

THE ROCKIT-1 STUDY

ROCKIT-1 is a phase 2a open-label study to assess preliminary safety, tolerability, and effect on neurodegeneration biomarkers of BRAVYL in patients with 4-Repeat (4R) Tauopathies to assess whether the drug improves clinical features of the disease. The study will enroll approximately 15 patients, all of whom will be treated with BRAVYL for 48 weeks.

ROCKIT-1 is being conducted at the University of California, San Francisco (UCSF), under the direction of Dr. Adam Boxer. Dr. Boxer is the Endowed Professor in Memory and Aging in the Department of Neurology. He directs UCSF’s Neurosciences Clinical Research Unit and the Alzheimer’s Disease and Frontotemporal Degeneration Clinical Trials Program at the UCSF Memory and Aging Center.

“Studies of progressive supranuclear palsy and corticobasal syndrome suggest that mitigating pathogenic tau levels is a rational strategy for tauopathy treatment,” notes Dr. Boxer. “BRAVYL, a ROCK 1 and ROCK 2 inhibitor, mitigated pathogenic tau levels in animal models. ROCK 1 and ROCK 2 have been shown to be upregulated in brain tissue of PSP and CBS patients. Any therapeutic that can demonstrate biological proof of concept in this patient population would be a major advancement in this field.”

“Dosing the first patient in our ROCKIT-1 clinical trial marks a critical turning point in our efforts to advance a potentially disease-modifying treatment for patients with PSP and CBS,” said Sven Jacobson, Co-Founder and Chief Executive Officer of Woolsey. “We are excited to progress clinical development of BRAVYL and to bring forward a much-needed therapy for these rapidly progressing neurodegenerative diseases.”

BRAVYL has been granted “orphan drug” designation (ODD) by the Food and Drug Administration (FDA) for both PSP and CBS. The FDA’s Office of Orphan Drug Products awards orphan status to support development of medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the U.S. ODD provides certain benefits, including seven years market exclusivity upon regulatory approval.

Data readout in the study is anticipated in 2022.

ABOUT WOOLSEY

Woolsey Pharmaceuticals does not discover new drugs. We are an indications discovery company. We discover new ways to use existing drugs for other diseases and conditions for which patients in need have no therapeutic solutions.

In late 2019, Woolsey licensed exclusive rights to BRAVYL from a leading Japanese pharmaceutical company. The drug’s active ingredient, fasudil, is approved in Japan and China in an IV formulation for the treatment of cerebral vasospasm. Woolsey’s drug, BRAVYL, is an oral version of fasudil.

Woolsey Pharmaceuticals is a portfolio company of Embark Healthcare (embarkhc.com).

Contacts

Sven Jacobson

[email protected]

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