Fulcrum Therapeutics® Announces Multiple Presentations on FSHD at the American Academy of Neurology’s Annual Meeting

Presentations highlight potential of losmapimod to slow or stop progression of FSHD

Company on track to initiate Phase 3 REACH trial in 2Q 2022

CAMBRIDGE, Mass., April 01, 2022 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced multiple oral and poster presentations on losmapimod for facioscapulohumeral muscular dystrophy (FSHD) at the American Academy of Neurology’s Annual Meeting, taking place April 2-7 in Seattle, WA. These presentations highlight clinical data supporting the potential of losmapimod as well as the design of the Phase 3 REACH trial of losmapimod in FSHD.

“FSHD is a progressive, relentless, and debilitating disease for which there are no approved therapies,” said Christopher Morabito, MD, Fulcrum’s chief medical officer. “The clinical data being presented at AAN demonstrate losmapimod’s potential to slow or stop disease progression, while providing important insights into optimal measures of disease progression in FSHD. As we prepare to begin enrolling patients in our Phase 3 REACH trial this quarter, we remain focused on serving the FSHD community by working to bring a much-needed therapy to patients.”

Oral Presentations:
The following presentations will take place during the S23 – Therapeutics for Muscle Diseases Scientific Platform Session

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, 48-Week Study of the Efficacy and Safety of Losmapimod in Subjects with FSHD: ReDUX4

• Date/Time: Tuesday, April 5^th, 4:42pm – 4:54pm PDT
• Presenter: Christopher Morabito, MD—Fulcrum Therapeutics

An Open-Label Study of Losmapimod to Evaluate the Safety, Tolerability, and Biomarker and Clinical Outcome Assessment Changes in Subjects with FSHD1

• Date/Time: Tuesday, April 5^th, 4:54pm – 5:06pm PDT
• Presenter: Jennifer Shoskes, PharmD —Fulcrum Therapeutics

Whole Body MRI Quantitative muscle analysis to evaluate Efficacy of Losmapimod in a Phase 2 Placebo-Controlled Study in Subjects with FSHD (ReDUX4)

• Date/Time: Tuesday, April 5^th, 5:06pm – 5:18pm PDT
• Presenter: Christopher Morabito, MD—Fulcrum Therapeutics

Poster Presentations:
The following poster will be available during the Poster Session 4 on Sunday, April 3^rd from 8:00am to 9:00am PDT.

Reachable Workspace to Evaluate Efficacy of Losmapimod in Subjects with FSHD in Two Phase 2 Studies

• Presenter: Jennifer Shoskes, PharmD —Fulcrum Therapeutics
• Poster ID: 13-008

The following poster will be available during the Poster Session 12 on Tuesday, April 5^th from 5:30pm – 6:30pm PDT

Quantitative muscle analysis in FSHD using Whole-Body MRI: Composite Muscle Measurements for Cross-Sectional Analysis

• Presenter: Jennifer Shoskes, PharmD —Fulcrum Therapeutics
• Poster ID: 2715                

The presentations will be available to registered conference attendees as well as on the company website at https://www.fulcrumtx.com/pipeline/#publications. The presentations will also be available for viewing to registered conference attendees during the virtual AAN 2022 Annual Meeting taking place April 24-26.

About FSHD
FSHD is a serious, rare, progressive and debilitating disease for which there are no approved treatments. It is characterized by fat infiltration of skeletal muscle leading to muscular atrophy involving primarily the face, scapula and shoulders, upper arms, and abdomen. Impact on patients includes profound decreases in the ability to perform activities of daily living, loss of upper limb function, loss of mobility and independence and chronic pain. FSHD is one of the most common forms of muscular dystrophy and has an estimated patient population of 16,000 to 38,000 in the United States alone.

About Losmapimod
Losmapimod is an investigational, selective p38α/β mitogen activated protein kinase (MAPK) inhibitor. Fulcrum exclusively in-licensed losmapimod from GSK following Fulcrum’s discovery of the role of p38α/β inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Results reported from the ReDUX4 trial demonstrated slowed disease progression and improved function, including positive impacts on upper extremity strength, supporting losmapimod’s potential to be a transformative therapy for the treatment of FSHD. Although losmapimod had never previously been explored in muscular dystrophies, it had been evaluated in more than 3,500 subjects in clinical trials across multiple other indications, and has been generally well tolerated. Losmapimod has been granted U.S. Food and Drug Administration (FDA) Fast Track designation and Orphan Drug Designation for the treatment of FSHD.

About REACH
Fulcrum plans to begin enrolling patients in its Phase 3 REACH clinical trial in the second quarter of 2022. REACH will be a randomized, double-blind, placebo-controlled, multi-national trial to evaluate the efficacy and safety of losmapimod for the treatment of FSHD. The trial is expected to enroll approximately 230 adults with FSHD. Patients will be randomized 1:1 to receive either losmapimod, administered orally as a 15 mg tablet twice a day, or placebo, and evaluated over a 48-week treatment period. RWS is the primary endpoint of the trial. Secondary endpoints include muscle fat infiltration (MFI) measured by MRI, Patient Global Impression of Change (PGIC), and Quality of Life in Neurological Disorders of the upper extremity (Neuro QOL-UE). REACH will also include patient-centered assessments of healthcare utilization.

About Fulcrum Therapeutics
Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum’s two lead programs in clinical development are losmapimod, a small molecule for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and other hemoglobinopathies, including beta-thalassemia. The company’s proprietary product engine, FulcrumSeek™, identifies drug targets that can modulate gene expression to treat the known root cause of gene mis-expression.

Please visit www.fulcrumtx.com.

Forward-Looking Statements

This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this press release are forward-looking statements, including statements regarding the planned REACH trial including its expected start date and enrollment target, losmapimod’s potential as a therapy for FSHD, the ability of the selected endpoints to support regulatory approval and the sufficiency of Fulcrum’s cash resources. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with Fulcrum’s ability to continue to advance its product candidates in clinical trials; initiate and enroll clinical trials on the timeline expected or at all; obtain and maintain necessary approvals from the FDA and other regulatory authorities; replicate in clinical trials positive results found in preclinical studies and/or earlier-stage clinical trials of losmapimod, FTX-6058 and its other product candidates; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause Fulcrum’s actual results to differ from those contained in the forward-looking statements, see the “Risk Factors” section, as well as discussions of potential risks, uncertainties, and other important factors, in Fulcrum’s most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent Fulcrum’s views as of the date hereof and should not be relied upon as representing Fulcrum’s views as of any date subsequent to the date hereof. Fulcrum anticipates that subsequent events and developments will cause Fulcrum’s views to change. However, while Fulcrum may elect to update these forward-looking statements at some point in the future, Fulcrum specifically disclaims any obligation to do so.

Contact:

Please direct all patient inquiries to: clinicaltrials@fulcrumtx.com.

Investors and Media:

Naomi Aoki
Senior Vice President, Corporate Communications and Investor Relations
naoki@fulcrumtx.com 

Stephanie Ascher
Stern Investor Relations, Inc.
stephanie.ascher@sternir.com 
212-362-1200

Doug Haslam
Berry & Company Public Relations
dhaslam@berrypr.com 
212-253-8881