Full Press Release Details
Fulcrum Therapeutics Announces Upcoming Milestones
to Support Its Mission of Treating the Root Cause of Rare Genetic Diseases
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
to provide update on losmapimod in FSHD in 1Q 2022
Kate Haviland appointed Chair of board of directors
CAMBRIDGE, Mass., January 10, 2022 Fulcrum Therapeutics, Inc. (Nasdaq: FULC), a clinical-stage
biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today outlined its recent accomplishments and expected upcoming milestones. Fulcrum will present at the virtual 40th Annual J.P. Morgan Healthcare Conference on Thursday, January 13 at 12:00 p.m. ET. A live webcast will be available on the Investor Relations section of Fulcrum s website.
Fulcrum made substantial progress in 2021, positioning us for a transformative 2022, said Bryan Stuart, president and chief executive officer.
Our clinical results with losmapimod highlight its potential to slow or stop the progression of FSHD, a relentless form of muscular dystrophy that leads to loss of upper body function and mobility and for which there are no treatments. We also
reported results from our clinical program with FTX-6058, supporting its potential to be a functional cure for sickle cell disease. Based on these results, we are intently focused on advancing this oral HbF
inducer for people with sickle cell disease as well as other hemoglobinopathies.
As we enter 2022, we are building on this progress, with
multiple milestones expected across our clinical and discovery-stage pipeline, Mr. Stuart continued. We plan to provide an update on losmapimod in the first quarter. I m also pleased to share that we enrolled the first patient
with sickle cell disease in our Phase 1b trial, putting us on track to report initial data in the second quarter of 2022. We also submitted an IND for FTX-6058 in select hemoglobinopathies and expect to start
a Phase 1b trial in the second quarter of 2022. In parallel, we continue to advance our FulcrumSeek product engine and plan to nominate our next development candidate this year to support an
IND by the end of the first quarter of 2023.
Recent Accomplishments
Preclinical Pipeline
Today, Fulcrum also announced that Kate Haviland, chief operating officer and incoming chief executive officer of Blueprint Medicines, has been named chair of
Fulcrum s board of directors. Ms. Haviland has been a member of Fulcrum s board since 2018. As part of a planned transition, she is succeeding Mark Levin. Mr. Levin is a partner at Third Rock Ventures and has served on
Fulcrum s board of directors since the company s founding in 2015 and as chair of the board since 2016. Mr. Levin will remain on the board through April 18, 2022.
I m delighted that Kate will continue on our board in this new capacity, Mr. Stuart said. Kate has an outstanding record in the
biotechnology industry and has been a tremendous asset to Fulcrum. We are extremely grateful to Mark for his leadership as chair and for his contributions to our growth and evolution from a start-up to a
publicly traded company with three potentially disease-modifying programs in clinical development.
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 Ms. Haviland. I m very pleased to be part of this team and honored to
lead this board in my new role. As we head into 2022 and beyond, I believe we are well positioned to advance our mission to bring life-changing therapies to patients with genetically defined rare diseases.
2022 Financial Outlook
2021, Fulcrum s cash, cash equivalents and marketable securities were $218.2 million (unaudited). Based on its current operating plans, the company believes this is sufficient to fund its anticipated operating expenses and capital
expenditure requirements into 2024.
Fulcrum to Present at the virtual 40th Annual J.P. Morgan Healthcare Conference
Fulcrum will present at the virtual 40th annual J.P. Morgan Healthcare Conference on Thursday,
January 13, 2022, at 12:00 p.m. ET followed by a Q&A session. A live webcast will be accessible in the Investor Relations section of the company s website, www.fulcrumtx.com and will be archived for 30 days
following the event.
Losmapimod is an investigational, selective p38 / mitogen activated protein kinase (MAPK) inhibitor. Fulcrum
exclusively in-licensed losmapimod from GSK following Fulcrum s discovery of the role of p38 / 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 U.S. Food and Drug Administration (FDA) Fast Track designation and Orphan Drug Designation for the treatment of 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 the United States alone. 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.
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 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 lead programs in clinical development are losmapimod, a small molecule for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and FTX-6058, a small
molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and other hemoglobinopathies, including beta-thalassemia. The company s proprietary product engine, FulcrumSeek , identifies drug targets that can modulate gene expression to treat the known root cause of gene mis-expression.
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 Securities and Exchange Commission. In addition, the forward-looking statements included in
this press release represent Fulcrum s views as of the date hereof and should not be relied upon as representing Fulcrum s views as of any date subsequent to the date hereof. Fulcrum anticipates that subsequent events and developments will
cause Fulcrum s views to change. However, while Fulcrum may elect to update these forward-looking statements at some point in the future, Fulcrum specifically disclaims any obligation to do so.
Director, Investor Relations and Corporate
Stern Investor Relations, Inc.
Senior Vice President, Corporate Communications and
Berry & Company Public Relations