Fulcrum Therapeutics Announces Multiple Presentations During the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition

Rhea-AI Summary

Fulcrum Therapeutics (Nasdaq: FULC) has initiated the screening of healthy volunteers in its Phase 1 clinical trial for FTX-6058, a small molecule aimed at treating sickle cell disease and β-thalassemia. The company will also present preclinical data on FTX-6058 at the upcoming ASH Annual Meeting from December 5-8, 2020. Preclinical studies indicate potential elevations of fetal hemoglobin by up to 30%. The results will support the ongoing clinical trial, highlighting FTX-6058's promise for addressing hemoglobinopathies.

Positive

• Initiation of Phase 1 clinical trial with FTX-6058 in healthy volunteers.
• Preclinical studies suggest significant fetal hemoglobin elevation of up to 30%.
• Upcoming presentations at the ASH Annual Meeting showcasing preclinical data.

Negative

• None.

Screening of healthy volunteers initiated in Phase 1 clinical trial of FTX-6058

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 company’s IND-supporting preclinical studies with its Embryonic Ectoderm Development (EED) inhibitor, FTX-6058, for hemoglobinopathies such as sickle cell disease and β-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 β-thalassemia,” said Owen B. Wallace, Ph.D., Fulcrum’s 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 Therapeutics
Session Name: 113. Hemoglobinopathies, Excluding Thalassemia—New Genetic Approaches to Sickle Cell Disease: Poster I
Date 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 candidate
Presenter: Christopher Moxham, Ph.D., Senior Vice President Discovery Research at Fulcrum Therapeutics
Session Name: 802. Chemical Biology and Experimental Therapeutics: Poster I
Date 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 disease
Presenter: Billy Stuart, Scientist II, Fulcrum Therapeutics
Session Name: 113. Hemoglobinopathies, Excluding Thalassemia—Basic and Translational Science: Poster III
Date 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-6058
FTX-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 Fulcrum’s 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 Disease
Sickle 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 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 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 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 Company’s product candidates and the potential advantages and therapeutic potential of the Company’s product candidates. All statements, other than statements of historical facts, contained in this press release, including statements regarding the Company’s 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 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 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 Company’s 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 Company’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 the Company’s most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company’s views as of the date hereof and should not be relied upon as representing the Company’s views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company’s 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 Communications
617-651-8664
cwaarich@fulcrumtx.com

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

Media:
Kaitlin Gallagher
Berry & Company Public Relations
kgallagher@berrypr.com
212-253-8881

FAQ

What is the significance of the Phase 1 clinical trial for FTX-6058?

The Phase 1 clinical trial for FTX-6058 represents a critical step in assessing its safety and efficacy for treating sickle cell disease and β-thalassemia.

When will Fulcrum Therapeutics present data on FTX-6058?

Fulcrum Therapeutics will present data on FTX-6058 at the ASH Annual Meeting, scheduled for December 5-8, 2020.

What are the expected benefits of FTX-6058?

FTX-6058 aims to significantly increase fetal hemoglobin levels, which could alleviate the symptoms of sickle cell disease and β-thalassemia.

What is the current status of FTX-6058?

FTX-6058 is currently in Phase 1 clinical trials with healthy volunteers, following promising preclinical results.