Fulcrum Therapeutics to Present Initial Data from Phase 1b Trial of FTX-6058 in Adults with Sickle Cell Disease at European Hematology Association (EHA) Hybrid Congress in Vienna, Austria

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Fulcrum Therapeutics, Inc.

– FTX-6058 is the only oral hemoglobin F (HbF) inducer in clinical development

-Data will include baseline HbF changes, safety, tolerability, pharmacokinetics and other pharmacodynamic parameters measurements of the ongoing dose cohort of 6 mg FTX-6058 in adults with sickle cell disease (SCD)

– Webcast for investors on June 10and 8 a.m. EDT

CAMBRIDGE, Mass., May 12, 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 that it will present two posters at the European Association of Hematology (EHA) which will take place June 9 -12, 2022, in Vienna, Austria. A third abstract has also been accepted for publication. Accepted abstracts are now available online on the EHA website at www.ehaweb.org.

“HbF is the only mechanism that has been shown to broadly improve clinical outcomes for patients with sickle cell disease, including anemia, vaso-occlusive crises, pain, fatigue, and acute chest syndrome,” said Judy Dunn, Ph.D., President of Research and Development at Fulcrum. “This Phase 1b study was designed to provide proof of concept that FTX-6058 produces increases in HbF and could potentially be the first oral HbF inducer to address the critical unmet needs of this population.”

  • FTX-6058 is an experimental oral small molecule embryonic ectoderm development inhibitor (EEDI) that has demonstrated robust induction of fetal hemoglobin (HbF) in human cells and animal models of sickle cell disease (SCD) .

  • Clinically, in a phase 1 multiple ascending dose (MAD) study in healthy volunteers, FTX-6058 induced HBG (hemoglobin γ-subunit) mRNA in a dose-dependent manner. Translation of HBG mRNA is responsible for production of HbF protein.

In addition to the poster presentation, Fulcrum will host a virtual investor event on June 10and at 8 a.m. EDT to review this initial FTX-6058 data and provide a program update.

Information on the presentation of the poster
Date: June 10, 2022
Time: 4:30 p.m. CEST/10:30 a.m. EDT

Title: Interim Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Results of a
open-label study of FTX-6058 in adults living with sickle cell disease
Presenter: Julie Kanter, MD, Director of the UAB Adult Sickle Cell Clinic and Associate Professor of Hematology and Oncology

Title: Inhibition of the repressive polycomb 2 complex by EED induces fetal hemoglobin in healthy subjects.
and models of sickle cell disease
Presenter: Billy Stuart, Ph.D., Fulcrum Therapeutics

Publication Information

Title: A systematic review of the literature describing the protective effects of HbF in sickle cell disease outcomes
Authors: Olga Mitelman, J. Barry, J. Bernhard, P. Bruno, C. Morabito, S. Snedecor, S. Ronnebaum

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 primary clinical development programs are losmapimod, a small molecule for the treatment of facioscapulohumeral muscular dystrophy (FSHD), and FTX-6058, a small molecule designed to increase the expression of fetal hemoglobin for the treatment of sickle cell disease and other haemoglobinopathies, including beta-thalassemia. Fulcrum’s proprietary product engine, FulcrumSeek™, identifies drug targets capable of modulating gene expression to address the known root cause of malgene expression. For more information, visit www.fulcrumtx.com and follow us on Twitter @FulcrumTx and LinkedIn.

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 fact, contained in this press release are forward-looking statements, including statements regarding the planned REACH trial, including its expected start date and enrollment goal, presentation data from the first dose cohort in the FTX-6058 Phase 1b trial and the second dose cohort, the clinical development plan for FTX-6058 as well as the timeline for expansion into other hemoglobinopathies and the initiation of the registrational trial for sickle cell disease, the appointment of additional development candidates and the timing of the fourth IND, the sufficiency of Fulcrum’s treatment, the financial resources, the potential of losmapimod as a therapy for FSHD and the Ability of selected REACH trial endpoints to support regulatory approval. The words “anticipate”, “believe”, “continue”, “could”, “estimate”, “expect”, “intend”, “may”, “plan”, “potential”, ” predict”, “project”, “should”, “target”, “shall”, “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain such identifying words. All forward-looking statements are based on management’s current expectations regarding future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those stated or implied. by these 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 through clinical trials; initiate and recruit clinical trials on schedule or not 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, retain or protect intellectual property rights in 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, each 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. Further, the forward-looking statements included in this press release represent the views of Fulcrum as of the date hereof and should not be relied upon as representing the views of Fulcrum as of any date subsequent to the date hereof. Fulcrum anticipates that subsequent events and developments will cause Fulcrum to change its mind. However, although Fulcrum may choose to update these forward-looking statements at some time in the future, Fulcrum expressly disclaims any obligation to do so.

Contact:

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

Media:
Dee Smith
Executive Director, Corporate Communications
dsmith@fulcrumtx.com
202-746-1324

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