Woolsey Pharmaceuticals Completes B Round Extension To Advance Its ALS Program

NEW YORK, Feb. 27, 2025 /PRNewswire/ -- Woolsey Pharmaceuticals today announced the successful completion of an extension to its Series B Preferred financing round by existing investors.

"We are immensely grateful for the continued support of our investors and remain steadfast and confident in our mission to advance BRAVYL® for the treatment of ALS," said Sven Jacobson, CEO of Woolsey Pharmaceuticals. "With these funds, we are well-positioned to complete critical milestones."

In the fourth quarter of 2024, the company completed enrollment into the high-dose (300 mg/day) cohort of the REAL study, which is assessing BRAVYL® (oral fasudil) for the treatment of Amyotrophic Lateral Sclerosis (ALS). This follows promising biomarker and clinical findings from the study's standard-dose cohort. A data readout is expected in June of this year.

At the 180 mg/day dose (n=31), the biomarker Neurofilament Light (NfL), a sensitive indicator of neuronal damage and rate of ALS progression, decreased by 15% from baseline to 6 months (p=0.0006). Moreover, greater decreases in NfL were correlated with less deterioration in function as measured by the ALSFRS-R (p=0.028). There were directionally positive clinical improvements compared to a matched historical control — a 17% lower decline in ALSFRS-R, a 37% lower decline in slow vital capacity (SVC), and a 56% lower decline in muscle strength.

In December 2024, Woolsey announced that treatment with BRAVYL significantly reduced TDP-43 aggregation and cytoplasmic redistribution. In ALS, TDP-43 pathology is understood to spread via extracellular vesicles, including neuronal exosomes, which are secreted by sick neurons and can be taken up by healthy ones. TDP-43 pathology is characterized by the translocation of TDP-43 from the nucleus, where it normally resides, to the cytoplasm and the formation of aggregates.

Key Findings Include:

• Mouse motor-neuron cells treated with baseline neuronal derived exosomes (NDEs) from ALS patients in the REAL study showed marked aggregation and redistribution of TDP-43 into the cytoplasm, indicating that NDEs from ALS patients can confer TDP-43 pathology in this system.
• In contrast, when exposed to NDEs from the same patients treated for 6 months with BRAVYL, TDP-43 aggregation and cytoplasmic redistribution were significantly reduced by 60% (p=0.0006) and 61% (p<0.0001) respectively.

Woolsey has substantial patent coverage — 36 patent families have been filed for BRAVYL (oral fasudil), a number of which have already issued.

US patent 11,779,588 protects the company's proprietary position in using oral fasudil to target disease progression in ALS patients, and includes a claim "A method of treating a patient with sporadic amyotrophic lateral sclerosis (ALS), comprising orally administering a therapeutically effective amount of a fasudil or hydroxyfasudil (M3), or a pharmaceutically acceptable salt thereof, to the patient." 

US patents 11,944, 633 and 12,115,167 cover, among other innovations, solid and liquid dysphagia-friendly formulations of fasudil. Dysphagia, a common swallowing disorder affecting many patients, including those with neurodegenerative diseases such as ALS, is a major barrier to administration of solid pills and capsules. Woolsey's advanced formulations are tailored to make medication easier to swallow, without compromising the efficacy or stability of the active ingredients. These patents support the next generation of its ALS drug BRAVYL, to be named BRAVYL-DF.

On January 10, 2025, Woolsey announced that the U.S. Patent and Trademark Office (USPTO) granted additional claims for the use of BRAVYL in treating ALS.  This latest patent allowance covers treatment of possible, probable laboratory-supported, probable, or definite ALS, as defined by El Escorial Revised ALS diagnostic criteria, using fasudil or its active metabolite.  Further claims are directed to reducing neurofilament light (NfL).

Woolsey also has Orphan Drug Designations for the use of BRAVYL in ALS for both the U.S. and Europe.

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 worsening represents a meaningful advancement in efforts to enhance the prognosis and quality of life of individuals impacted by the devastating condition.

About Woolsey Pharmaceuticals

Woolsey Pharmaceuticals' 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.