GlobeNewswire Inc | Sep 20, 2021 07:00AM EDT

September 20, 2021

Presentations highlight progress in the development of losmapimod for FSHD

CAMBRIDGE, Mass., Sept. 20, 2021 (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 it will present multiple posters and presentations on losmapimod for facioscapulohumeral muscular dystrophy (FSHD) during the Virtual Congress of the World Muscle Society. These results highlight progress made in the development of losmapimod for the treatment of FSHD as well as imaging modalities for the management of FSHD.

FSHD is a serious, relentless and debilitating disease for which there are currently no approved therapies, saidChris Morabito, MD, Fulcrums chief medical officer. The results from the Phase 2b ReDUX4 trial show clinically relevant benefit across multiple structural, functional and patient reported endpoints with losmapimod and represent a major advance for the potential treatment of FSHD. These data being presented support the benefit/risk of losmapimod as a disease-modifying therapy and its continued development. We remain on track to meet with regulators in the fourth quarter of this year and to provide an update on the clinical path forward in the first quarter of 2022.

Fulcrum-Sponsored Symposium:Advances in Assessment of FSHD and Clinical Trial Results with LosmapimodDate/Time: Monday, September 20, 12:00pm 1:30pm EDTPresenters:

-- Jeffrey Statland, MD, PhD University of Kansas Medical Center -- Michelle Mellion, MD Fulcrum Therapeutics -- Rabi Tawil, MD University of Rochester Medical Center -- Baziel van Engelen & Joost Kools Radboud University Medical Center -- Chris Morabito, MD Fulcrum Therapeutics

Oral Presentation:A Phase 2, Randomized, Double-blind, Placebo-controlled, 48-Week Study of the Efficacy and Safety of Losmapimod in Subjects with FSHD: ReDUX4Date/Time: Thursday, September 23rd, 10:30am 10:40am EDTPresenter:

-- Rabi Tawil, MD University of Rochester Medical Center

Poster Presentations:Date/Time: Thursday, September 23rd, Available for viewing starting at 11:30am EDTPosters will be available to registered conference attendees and will also be available in the Publications section of fulcrumtx.com.

Title: An Open-Label Study of Losmapimod to Evaluate the Safety, Tolerability, and Biomarker and Clinical Outcome Assessment Changes in Subjects with FSHD1Author: Joost Kools Radboud University Medical CenterPoster ID: EP 168

Title:Use of snRNA-seq to Characterize the Pathogenic Skeletal Muscle Microenvironment Author: Anu Raman, Ph.D. Fulcrum TherapeuticsPoster ID: EP 170

Title: Whole body MRI Quantitative Muscle Analysis to Evaluate Efficacy of Losmapimod in a Phase 2 Placebo-controlled Study in Subjects with FSHD (ReDUX4)Author: Michelle Mellion, MD Fulcrum TherapeuticsPoster ID: EP 330

Title: Quantitative Muscle Analysis in FSHD Using Whole-Body MRI: Composite Muscle Measurements for Cross-Sectional AnalysisAuthor: Michelle Mellion, MD Fulcrum TherapeuticsPoster ID: EP 331

Title: Revolutionizing Drug Discovery in Genetically Defined Muscle Disease Using Single-Cell and High Dimensional DatasetsAuthor: Alejandro Rojas, Ph.D. Fulcrum TherapeuticsPoster ID: EP 321

About FSHD FSHD is a serious, rare, progressive and disabling disease for which there are no approved treatments. FSHD is characterized by muscle degeneration and fat infiltration, initially affecting movement of the face and eventually the arms, trunk and legs. Disease progression results in accumulation of disability, with many patients ultimately becoming dependent upon the use of a wheelchair for daily mobility. Impact on patients includes decreased ability to perform activities of daily living, maintain independence, and lost ability to function or work.

FSHD is caused by mis-expression of DUX4 in skeletal muscle, resulting in the presence of DUX4 proteins that are toxic to muscle tissue. Normally, DUX4-driven gene expression is limited to early embryonic development, after which time the DUX4 gene is silenced. In people with FSHD, the DUX4 gene is turned on as a result of a genetic mutation. The result is death of muscle and its replacement by fat, leading to skeletal muscle weakness and progressive disability. There are no approved therapies for FSHD, one of the most common forms of muscular dystrophy, with an estimated patient population of 16,000 to 38,000 in the United States alone.

About Losmapimod Losmapimod is a selective p38/ mitogen activated protein kinase (MAPK) inhibitor that was exclusively in-licensed from GSK by Fulcrum Therapeutics following Fulcrums discovery of the role of p38/ inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Utilizing its proprietary product engine, FulcrumSeek, Fulcrum discovered that inhibition of p38/ reduced expression of the DUX4 gene in muscle cells derived from patients with FSHD. Although losmapimod has never previously been explored in muscular dystrophies, it has been evaluated in more than 3,600 subjects in clinical trials across multiple other indications, including in several Phase 2 trials and a Phase 3 trial. No safety signals were attributed to losmapimod in any of these trials. Losmapimod has been granted U.S. Food and Drug Administration (FDA) Fast Track designation and Orphan Drug Designation for the treatment of FSHD.

About Fulcrum TherapeuticsFulcrum 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. Fulcrums proprietary product engine, FulcrumSeek, identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta-thalassemia into Phase 1 clinical development.

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, including statements regarding the development status of the Companys product candidates, the potential advantages and therapeutic potential of the Companys product candidates and planned meetings with regulatory agencies. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Companys strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. 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 managements 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 Fulcrums ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in clinical trials; initiate and enroll clinical trials on the timeline expected or at all; correctly estimate the potential patient population and/or market for the Companys product candidates; replicate in clinical trials positive results found in preclinical studies and/or earlier-stage clinical trials of losmapimod 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 the Companys 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 the Companys most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Companys views as of the date hereof and should not be relied upon as representing the Companys views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Companys views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

Contact:

Investors: Christi Waarich Director, Investor Relations and Corporate Communications617-651-8664cwaarich@fulcrumtx.com

Stephanie Ascher Stern Investor Relations, Inc.stephanie.ascher@sternir.com212-362-1200

Media: Kaitlin GallagherBerry & Company Public Relationskgallagher@berrypr.com212-253-8881