First patient dosed in Phase 1b trial of FTX-6058 in sickle cell disease; Initial data expected in 2Q 2022
Submitted IND for FTX-6058 to support initiation of Phase 1b trial in select hemoglobinopathies in 2Q 2022
On track to provide update on losmapimod in FSHD in 1Q 2022
“Fulcrum made substantial progress in 2021, positioning us for a transformative 2022,” said
“As we enter 2022, we are building on this progress, with multiple milestones expected across our clinical and discovery-stage pipeline,”
Recent Accomplishments
- Dosed first sickle cell disease patient in the Phase 1b clinical trial of FTX-6058, an oral fetal hemoglobin (HbF) inducer.
- Completed three-month preclinical toxicology studies and initiated chronic toxicology studies to advance FTX-6058 in multiple indications.
- Submitted Investigational New Drug (IND) application to initiate clinical development of FTX-6058 in select hemoglobinopathies, including beta-thalassemia.
- Announced the appointment of
Esther Rajavelu as Chief Financial Officer.
Expected Milestones
Losmapimod
- Provide an update in the first quarter of 2022 on plans for a Phase 3 clinical trial of losmapimod in facioscapulohumeral muscular dystrophy (FSHD).
FTX-6058
- Report initial data, including measures of HbF protein induction, from the Phase 1b trial in people with sickle cell disease in the second quarter of 2022.
- Initiate Phase 1b trial in select hemoglobinopathies, including beta-thalassemia, in the second quarter of 2022.
- Initiate registrational trial in sickle cell disease in early 2023.
Preclinical Pipeline
- Nominate next development candidate by end of 2022 to support the company’s fourth IND by the end of the first quarter of 2023.
Company Update
Today, Fulcrum also announced that
“I’m delighted that Kate will continue on our board in this new capacity,”
“It’s an exciting time at Fulcrum as the losmapimod and FTX-6058 programs advance in the clinic and the research pipeline continues to accelerate,” said
2022 Financial Outlook
As of
Fulcrum to Present at the virtual 40th Annual
Fulcrum will present at the virtual 40th annual
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, with no safety signals attributed to losmapimod. Losmapimod has been granted
About FSHD
FSHD is one of the most common forms of muscular dystrophy. It is a serious, rare, progressive and disabling disease for which there are no approved treatments and has an estimated patient population of 16,000 to 38,000 in
About FTX-6058
FTX-6058, an EED inhibitor, is an investigational oral HbF inducer being developed for the treatment of sickle cell disease and other hemoglobinopathies, such as beta-thalassemia. The validation of EED as a target for sickle cell disease and the discovery of FTX-6058 was conducted using FulcrumSeek™. Results from a Phase 1 healthy volunteer trial demonstrated proof of biology and proof of mechanism, including robust induction of HBG mRNA after 14 days of dosing. To date, FTX-6058 has been generally well-tolerated with no serious adverse events reported.
About Sickle Cell Disease
Sickle cell disease 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 cells. Sickle cell disease 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
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 timing of data readouts and other clinical updates regarding Fulcrum’s product candidates, the potential advantages and therapeutic potential of Fulcrum’s product candidates, the initiation and enrollment of clinical trials and the timing and design of planned clinical trials and submission of INDs, and its 2022 financial outlook among others. 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
Contact:
Investors:
Director, Investor Relations and Corporate Communications
cwaarich@fulcrumtx.com
617-651-8664
Stern Investor Relations, Inc.
stephanie.ascher@sternir.com
212-362-1200
Media:
Senior Vice President, Corporate Communications and Investor Relations
naoki@fulcrumtx.com
Berry & Company Public Relations
kgallagher@berrypr.com
212-253-8881
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