Full Press Release Details
Fulcrum Therapeutics Reports Recent Business Highlights and
Fourth Quarter and Full Year 2020 Financial Results
Company on track to present data from Phase 2b ReDUX4 trial with losmapimod in facioscapulohumeral muscular
dystrophy (FSHD) in late-2Q 2021
On track to report results from Phase 1 trial in healthy adult volunteers with
FTX-6058 for sickle cell disease in mid-2021
Company to discontinue Phase 3 COVID-19 trial (LOSVID)
Extended cash runway into 4Q 2022; raised $50.6 million in gross proceeds from January 2021 public offering
Conference call scheduled for 8:00 a.m. ET today
CAMBRIDGE, Mass. March 4, 2021 Fulcrum Therapeutics, Inc. (Nasdaq: FULC), a clinical-stage biopharmaceutical
company focused on improving the lives of patients with genetically defined rare diseases, today provided a business update and reported financial results for the fourth quarter and full year of 2020.
In 2020, we made meaningful progress in advancing our pipeline despite the extraordinary challenges brought on by
COVID-19, said Robert J. Gould, Ph.D., president and chief executive officer. We have laid the foundation to achieve several key milestones in 2021, including a comprehensive assessment of our
Phase 2 ReDUX4 trial with losmapimod in facioscapulohumeral muscular dystrophy late in the second quarter and completing our Phase 1 trial in healthy adult volunteers with FTX-6058, a highly potent small
molecule EED inhibitor in development for the treatment of select hemoglobinopaties, including sickle cell disease and beta-thalassemia. Additionally, after careful consideration and a strategic review of the
COVID-19 landscape, we are discontinuing our LOSVID trial. This enables us to focus on rare diseases. I would like to thank the patients and investigators who participated in this trial and the Fulcrum team
who worked tirelessly to rapidly design and launch the LOSVID trial during a global pandemic.
Furthermore, we have made great progress with
our next-generation product engine including new levels of validation in our internal research efforts and externally through our strategic collaborations, continued Dr. Gould. With the additional capital from our recent public
offering, we have extended our cash runway into the fourth quarter of 2022 and we believe that we are well positioned to continue progress on our goal to advance therapies to improve the lives of patients with genetically defined rare
Recent Business Highlights
Fourth Quarter and Full Year 2020 Financial Results
Collaboration revenue was $8.8 million for the year ended December 31, 2020, as compared
to no revenue recognized during the year ended December 31, 2019. The increase in collaboration revenue was due to the execution of the collaboration and license agreements with Acceleron and MyoKardia in December 2019 and July 2020,
Research and development expenses were $59.0 million for the year ended December 31, 2020, as compared to $71.1 million for the
year ended December 31, 2019. Research and development expenses for the year ended December 31, 2019 include $25.6 million of one-time costs incurred associated with the issuance of Series B
convertible preferred stock under the company s license agreement with GSK for losmapimod and $2.5 million of one-time costs incurred
associated with the achievement of a milestone under the company s license agreement with GSK for losmapimod. Excluding these one-time costs, the
increase of $16.0 million was primarily due to increased costs to support the company s ongoing and planned clinical trials and increased personnel-related costs to support the growth of Fulcrum s research and development
organization, including increased stock-based compensation expense.
General and administrative expenses were
$21.4 million for the year ended December 31, 2020, as compared to $13.1 million for the year ended December 31, 2019. The increase of $8.3 million was primarily due to increased costs associated with operating as a public
company and increased personnel-related costs to support the growth of the organization, including increased stock-based compensation expense.
Net loss was $70.8 million for the year ended December 31,
2020, as compared to $82.7 million for the year ended December 31, 2019.
Conference Call and Webcast
Fulcrum Therapeutics, Inc. will host a conference call and webcast today at 8:00 a.m. ET to discuss the company s fourth quarter and full year 2020
financial results and recent business highlights. The webcast will be accessible through the Investor Relations section of Fulcrum s website at www.fulcrumtx.com. Following the live webcast, an archived replay will also be
U.S./Canada Dial-in Number:
International Dial-in
Number: 470-495-9162
Conference ID: 6376419
Replay Dial-in Number: 855-859-2056
Replay International Dial-in Number:
Conference ID: 6376419
FSHD is characterized by progressive skeletal muscle loss that initially causes weakness in muscles in the face, shoulders, arms and trunk, and progresses to
weakness throughout the lower body. Skeletal muscle weakness results in significant physical limitations, including an inability to smile and difficulty using arms for activities, with many patients ultimately becoming dependent upon the use of a
wheelchair for daily mobility.
FSHD is caused by mis-expression of DUX4 in skeletal muscle, resulting in the presence of DUX4 proteins that are toxic to
muscle tissue. Normally, DUX4-driven gene expression is limited to early embryonic development, after which time the DUX4 gene is silenced. In people with FSHD, the DUX4 gene is turned on as a result of a genetic mutation. The result is
death of muscle and its replacement by fat, leading to skeletal muscle weakness and progressive disability. There are no approved therapies for FSHD, one of the most common forms of muscular dystrophy, with an estimated patient population of 16,000
to 38,000 in the United States alone.
Losmapimod is a selective p38 / mitogen activated protein kinase
(MAPK) inhibitor that was exclusively in-licensed from GSK by Fulcrum Therapeutics following Fulcrum s discovery of the role of p38 / inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Utilizing its internal product engine, Fulcrum discovered that inhibition of
p38 / reduced expression of the DUX4 gene in muscle cells derived from patients with FSHD. Losmapimod has been evaluated in more than 3,600 subjects in
clinical research across multiple indications, including in several Phase 2 trials and a large Phase 3 trial in acute myocardial infarction. No safety signals were attributed to losmapimod in any of these trials. In 2020, the company received U.S.
and European Orphan Drug Designation for losmapimod for the treatment of FSHD. Fulcrum is currently conducting Phase 2 trials investigating the safety, tolerability, and efficacy of losmapimod to treat the root cause of FSHD.
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 cells. 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.
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 adult volunteers.
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). 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, the potential advantages and therapeutic potential of Fulcrum s product candidates, initiation and enrollment
of clinical trials and availability of clinical trial data, and the Company s ability to fund its operations with cash on hand. 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 clinical trials positive results found in preclinical
studies and/or earlier-stage clinical trials of losmapimod, FTX-6058 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.
Fulcrum Therapeutics, Inc.
Selected Consolidated Balance Sheet Data
| December 31, 2020 | December 31, 2019 | |||||||
| Cash, cash equivalents, and marketable securities | $ | 112,914 | $ | 96,713 | ||||
| Working capital (1) | 92,785 | 87,943 | ||||||
| Total assets | 129,577 | 110,439 | ||||||
| Total stockholders equity | 95,181 | 87,153 |
Fulcrum Therapeutics, Inc.
Consolidated Statements of Operations
(In thousands, except per share data)
| Three Months Ended December 31, | Year Ended December 31, | |||||||||||||||
| 2020 | 2019 | 2020 | 2019 | |||||||||||||
| Collaboration revenue | $ | 4,225 | $ | $ | 8,823 | $ | ||||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | 16,145 | 12,087 | 59,042 | 71,072 | ||||||||||||
| General and administrative | 5,867 | 4,403 | 21,392 | 13,145 | ||||||||||||
| Total operating expenses | 22,012 | 16,490 | 80,434 | 84,217 | ||||||||||||
| Loss from operations | (17,787 | ) | (16,490 | ) | (71,611 | ) | (84,217 | ) | ||||||||
| Other income, net | 67 | 367 | 792 | 1,540 | ||||||||||||
| Net loss | $ | (17,720 | ) | $ | (16,123 | ) | $ | (70,819 | ) | $ | (82,677 | ) | ||||
| Cumulative convertible preferred stock dividends | (7,128 | ) | ||||||||||||||
| Net loss attributable to common stockholders | $ | (17,720 | ) | $ | (16,123 | ) | $ | (70,819 | ) | $ | (89,805 | ) | ||||
| Net loss per share attributable to common stockholders, basic and diluted | $ | (0.64 | ) | $ | (0.71 | ) | $ | (2.79 | ) | $ | (8.13 | ) | ||||
| Weighted average number of common shares used in net loss per share attributable to common stockholders, basic and diluted | 27,537 | 22,610 | 25,354 | 11,046 |
Director, Investor Relations and Corporate
Stern Investor Relations, Inc.
Berry & Company Public Relations