GlobeNewswire Inc | Nov 4, 2020 04:01PM EST

November 04, 2020

CAMBRIDGE, Mass., Nov. 04, 2020 (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 on the companys IND-supporting preclinical studies with its Embryonic Ectoderm Development (EED) inhibitor, FTX-6058, for hemoglobinopathies such as sickle cell disease and -thalassemia during the virtual 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, December 5-8, 2020.

We believe FTX-6058 has the potential to offer a differentiated approach for the treatment of hemoglobinopathies like sickle cell disease and -thalassemia, saidOwen B. Wallace, Ph.D., Fulcrums chief scientific officer. By demonstrating clinically desirable elevations of fetal hemoglobin up to approximately 30% of total hemoglobin in preclinical studies, these data, and those from our completed IND-enabling studies with FTX-6058, are incredibly exciting. We are pleased to present our preclinical proof-of-concept findings at this important scientific meeting and look forward to our clinical trial of FTX-6058 in healthy volunteers.

Fulcrum will present three posters describing the potential of FTX-6058 to treat hemoglobinopathies during the following virtual sessions:

Poster Title:In vivo characterization of FTX-6058, a novel small molecule fetal hemoglobin inducer for sickle cell disease Presenter: Keqiang Xie, Ph.D., Senior Scientist at Fulcrum TherapeuticsSession Name: 113. Hemoglobinopathies, Excluding ThalassemiaNew Genetic Approaches to Sickle Cell Disease: Poster IDate and Time Saturday, December 5, 2020 from 7:00 a.m. 3:30 p.m. PT

Poster Title: Induction of fetal hemoglobin by FTX6058, a novel small molecule development candidatePresenter: Christopher Moxham, Ph.D., Senior Vice President Discovery Research at Fulcrum TherapeuticsSession Name: 802. Chemical Biology and Experimental Therapeutics: Poster IDate and Time Saturday, December 5, 2020 from 7:00 a.m. 3:30 p.m. PT

Poster Title:In vitro characterization of FTX-6058, a novel small molecule fetal hemoglobin inducer for sickle cell diseasePresenter: Billy Stuart, Scientist II, Fulcrum TherapeuticsSession Name: 113. Hemoglobinopathies, Excluding ThalassemiaBasic and Translational Science: Poster IIIDate and Time Monday, December 7, 2020 from 7:00 a.m. 3:30 p.m. PT

The poster sessions will be available to registered conference attendees and the presentations will be published online in the November 2020 supplemental issue of Blood. The posters will also be made available in the Publications section of fulcrumtx.com following the sessions.

About FTX-6058FTX-6058 is a highly potent small molecule inhibitor of Embryonic Ectoderm Development (EED) capable of inducing robust HbF protein expression in cell and murine models. Fulcrum believes the pharmacokinetics and human dose simulations support that FTX-6058 may be given as a once daily oral compound. The validation of EED as a target for sickle cell disease and the discovery of FTX-6058 as a novel HbF-inducing small molecule were conducted using Fulcrums proprietary Product Engine. Preclinical data with FTX-6058 showed an increase in HbF levels up to approximately 30% of total hemoglobin. Fulcrum has initiated a Phase 1 trial with FTX-6058 in healthy volunteers.

About Sickle Cell DiseaseSickle cell disease (SCD) is a genetic disorder of the red blood cells caused by a mutation in the HBB gene. This gene encodes a protein that is a key component of hemoglobin, a protein complex whose function is to transport oxygen in the body. The result of the mutation is less efficient oxygen transport and the formation of red blood cells that have a sickle shape. These sickle shaped cells are much less flexible than healthy cells and can block blood vessels or rupture leading to anemia. SCD patients typically suffer from serious clinical consequences, which may include anemia, pain, infections, stroke, heart disease, pulmonary hypertension, kidney failure, liver disease and reduced life expectancy.

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 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) and Phase 3 for the treatment of COVID-19. 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 StatementsThis 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 and the potential advantages and therapeutic potential of the Companys product candidates. 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 later clinical trials positive results found in preclinical studies and/or earlier-stage clinical trials of losmapimod and its other product candidates; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; 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