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Fulcrum Therapeutics, Inc. (FULC)
Chart Events
Performance Outlook
| Previous Close | 11.52 |
| Open | 11.45 |
| Bid | 0.00 x 1000 |
| Ask | 0.00 x 900 |
| Day's Range | 11.35 - 12.00 |
| 52 Week Range | 7.01 - 22.39 |
| Volume | 301,194 |
| Avg. Volume | 118,913 |
| Market Cap | 379.324M |
| Beta (5Y Monthly) | N/A |
| PE Ratio (TTM) | N/A |
| EPS (TTM) | -14.92 |
| Earnings Date | Mar 04, 2021 |
| Forward Dividend & Yield | N/A (N/A) |
| Ex-Dividend Date | N/A |
| 1y Target Est | 19.00 |
Fair Value
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- GlobeNewswire
Fulcrum Therapeutics Presents Published Structure of Investigational Small Molecule FTX-6058 at the American Chemical Society (ACS) Spring 2021 Virtual Conference
Company initiates dosing in Phase 1 healthy volunteer multiple ascending dose (MAD) cohortCAMBRIDGE, Mass., April 09, 2021 (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 presented the medicinal chemistry strategy for FTX-6058 at the First Time Disclosure Session at the American Chemical Society (ACS) Spring 2021 National Meeting. FTX-6058 is a highly potent orally bioavailable small molecule EED inhibitor for the potential treatment of select hemoglobinopathies, including sickle cell disease and β-thalassemia. The validation of EED as a fetal hemoglobin (HbF) inducer target for sickle cell disease was conducted using FulcrumSeek, Fulcrum’s proprietary product engine. “We are pleased to report progress on our development of FTX-6058 including the first publication of the structure of this compelling EED inhibitor,” said Chris Moxham, Ph.D., Fulcrum’s chief scientific officer. “We believe that this oral, once-a-day therapy with an impressive preclinical pharmacological profile has the potential to provide a meaningful therapeutic benefit to patients with sickle cell disease and β-thalassemia. We are also excited to report initial PK results from the SAD cohort and that our Phase 1 trial in healthy volunteers continues to progress with initiation of the multiple ascending dose cohorts. We expect to report the full data from this Phase 1 trial mid-year.” FTX-6058 inhibits PRC2 via binding to EED, which induces robust HbF protein expression in both cell and murine models. Increasing HbF has the potential to prevent or reduce disease-related pathophysiology and reduce the risk of recurring events such as vaso-occlusive crises and hemolysis. Preclinical data with FTX-6058 showed an increase in HbF levels up to approximately 30% of total hemoglobin, demonstrating the potential to have a significant impact in patients with sickle cell disease. Human genetic data further indicate that individuals with the sickle cell mutation and high HbF levels may have asymptomatic disease, underscoring the protective effect of increased HbF. Fulcrum’s Phase 1 trial in healthy volunteers is evaluating the safety, tolerability and pharmacokinetics of FTX-6058. Dosing has been initiated in the randomized, double-blind, placebo-controlled, multiple ascending dose (MAD) cohorts of the trial. Dosing continues in the single ascending dose (SAD) portion. The company anticipates sharing data from this Phase 1 trial in mid-2021 and initiating a clinical trial in sickle cell patients by the end of 2021. Today’s presentation, titled “Discovery of clinical candidate FTX-6058: a potent, orally bioavailable upregulator of fetal hemoglobin for treatment of sickle cell disease”, will be available in the “Publications” section of fulcrumtx.com. About Sickle Cell DiseaseSickle 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. 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 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 StatementsThis 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; 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 Communicationscwaarich@fulcrumtx.com 617-651-8664 Stephanie Ascher Stern Investor Relations, Inc.stephanie.ascher@sternir.com 212-362-1200 Media: Kaitlin GallagherBerry & Company Public Relationskgallagher@berrypr.com212-253-8881
- GlobeNewswire
Fulcrum Therapeutics Appoints Judith A. Dunn, Ph.D. as President of Research and Development
CAMBRIDGE, Mass., March 24, 2021 (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 Judith A. Dunn, Ph.D. has been appointed President of Research and Development. Dr. Dunn has held multiple leadership roles in global research and drug development spanning from discovery through commercialization. Most recently, she supported the development of new companies targeting areas of unmet need in neuroscience as an Entrepreneur in Residence at Atlas Venture. “We are honored to have Dr. Dunn join Fulcrum to expand our R&D leadership team, particularly as we move toward significant advancements with our losmapimod and FTX-6058 development programs,” said Bryan E. Stuart, Fulcrum’s incoming president and chief executive officer. “Our product engine and industry collaborations position us to advance many new opportunities in discovery and drug development in the months and years ahead. Judy’s outstanding experience with over 25 years in drug discovery and clinical development will be an important resource for us as we work to advance these programs.” “I am thrilled to take on this role at Fulcrum,” said Dr. Dunn. “I was very impressed with the productivity and potential of Fulcrum’s product engine, which has already generated two important programs for FSHD and select hemoglobinopathies, including sickle cell disease. I’m also excited for the advances in research and development, including bringing forward two INDs over the next 24 months. FulcrumSeek’s ability to generate and integrate functional, morphological and transcriptional data with great precision enables rapid identification of novel targets across a wide variety of diseases. I look forward to working with the team to continue to advance programs to treat the root causes of genetically defined rare diseases.” Prior to Atlas, Dr. Dunn served as Vice President of Clinical Development at Roche and was previously head of the Roche Innovation Center in New York City. She also supported the establishment of the Empire Discovery Institute, a New York State initiative to support progression of early stage research to clinical application. She has additionally held positions in both the research and commercial divisions of Pfizer and led clinical programs in psychiatry at Sunovion (Sepracor). Dr. Dunn received a postdoctoral fellowship in new drug development from Pfizer, a training fellowship in neuropharmacology from the Center for Brain Research, a Doctor of Philosophy, Developmental Neurobiology from Wesleyan University and a Bachelor of Science, Neurobiology from the University of Rochester. 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. 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. 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. Contact: Investors: Christi Waarich Director, Investor Relations and Corporate Communications617-651-8664cwaarich@fulcrumtx.com Stephanie Ascher Stern Investor Relations, Inc.stephanie.ascher@sternir.com 212-362-1200 Media: Kaitlin GallagherBerry & Company Public Relationskgallagher@berrypr.com212-253-8881
- GlobeNewswire
Fulcrum Therapeutics Presents Data for Potential FSHD Biomarker and Clinical Outcome Assessments at 2021 Muscular Dystrophy Association (MDA) Virtual Clinical & Scientific Conference
– Demonstrated Whole-Body MRI captures heterogeneity and provides key disease severity and progression information correlated with FSHD clinical endpoints – – Demonstrated potential of FSHD-TUG and Emerald in-home assessments as accurate, low-burden clinical assessments of mobility for FSHD patients – – Company on track to report data from Phase 2b ReDUX4 trial with losmapimod in FSHD in late-2Q 2021 – CAMBRIDGE, Mass., March 18, 2021 (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 the presentation of new data related to the use of imaging biomarkers and clinical outcome assessments for facioscapulohumeral muscular dystrophy (FSHD) at the 2021 Muscular Dystrophy Association (MDA) virtual Clinical and Scientific Conference. Presentations included evaluation of disease severity and progression with whole body musculoskeletal magnetic resonance imaging (WB-MSK-MRI), FSHD-TUG, a modified Timed Up and Go (TUG) assessment for FSHD patients, and in-home passive measurements of mobility and sleep. The presentation and posters can be found on Fulcrum’s website at https://www.fulcrumtx.com/pipeline/#publications. “There is a critical need for accurate, low patient burden assessments that can effectively track disease severity and progression, and correlate with clinical outcomes in FSHD,” said Michelle Mellion, M.D., Fulcrum’s senior medical director. “The Whole Body-MSK MRI can capture the heterogeneity and provide important information about disease severity as it correlates with FSHD relevant clinical endpoints. This protocol is currently being used in our Phase 2 clinical trials of losmapimod. WB-MSK-MRI also may enable an individualized assessment of disease progression, offering a more efficient screening of potential therapies and better facilitate decisions in the development of new treatments. Additionally, Emerald and FSHD-TUG help capture key metrics in FSHD patients.” Fulcrum and AMRA Medical have developed a quantitative WB-MSK-MRI protocol and analysis algorithms to volumetrically measure fat replacement of skeletal muscle in FSHD to use in multi-site clinical trials. WB-MSK-MRI is non-invasive and captures a holistic evaluation of the skeletal musculature, identifying small quantitative changes in muscle health that correlate with functional measures in FSHD patients and enable an assessment of disease heterogeneity. In the study being presented, the protocol was performed and standardized at six sites where patients were screened, biopsies were taken between 1-4 weeks and 5-12 weeks, and a final MRI scan was conducted between weeks 5-12. WB-MSK-MRI was shown to capture heterogeneity and provide important information about disease severity and progression in 17 patients. Of 618 muscles, 478 were analyzed. Good reproducibility was found across all muscles, with higher reproducibility in larger muscles. Results also showed strong cross-sectional correlation between Regional Composite Measurement, TUG, FSHD-TUG and Reachable Work Space (RWS) assessments. Fulcrum has also identified FSHD-TUG as a potential clinical outcome assessment of mobility in FSHD patients. In a separate study presented at the MDA meeting, FSHD-TUG demonstrated a correlation with clinical severity and patient reported physical function and lower extremity function. Existing assessments largely focus on walking parameters as a test of function, but most FSHD patients report difficulty getting up from lying down position. The FSHD-TUG was optimized to also include evaluation of sit to supine (laying on back) and the reverse. The study was conducted to determine the reliability and validity of TUG, a traditional measure of mobility, and FSHD-TUG, over a one-year period. Twenty-two FSHD patients and twenty healthy volunteers were enrolled. Patients were screened and stratified into groups, and each group performed two trials of the classic TUG and FSHD TUG on two separate visits one week apart. A total of four trials over two separate visits were recorded for each participant. On average, FSHD subjects took approximately twice the time to complete TUG, FSHD-TUG, and components of the FSHD-TUG compared to healthy volunteers. These results support the reliability and validity of FSHD-TUG as a potential clinical assessment of mobility for ongoing and future clinical trials. Study results also highlight the use of in-home monitoring from Emerald, a contactless radio-wave-based home monitoring system, to enable a large number of passively derived measurements of clinical progression including gait speed, time in bed, sleep and vital signs. Ten FSHD patients were observed in their homes for three months. In addition to in-home gait speed, novel metrics including assessments of sleep schedule variability and eTUG (the time from motion initiation within the bed to moving two meters away from the bed edge) were derived. Emerald’s in-home measurements were strongly correlated with clinical metrics. As the number of measurements increased, Emerald’s metrics became increasingly sensitive and were shown to detect smaller fluctuations in disease progression. “As we advance our clinical development program for losmapimod for the treatment of FSHD, we are also continually working to improve our ability to assess disease progression based on the most clear and effective outcome measures,” said Chris Moxham, Ph.D., Fulcrum’s chief scientific officer. “Assessments based on musculoskeletal MRI and clinical outcomes may be key to demonstrating patient benefit in this population. We expect full data from our Phase 2b ReDux4 trial late in the second quarter of this year, which will provide additional insights to inform the path forward for losmapimod in the treatment of FSHD.” About FSHDFSHD 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. About Losmapimod Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor that was exclusively in-licensed from GSK by Fulcrum Therapeutics following Fulcrum’s discovery of the role of p38α/β inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Utilizing its internal product engine, Fulcrum discovered that inhibition of p38α/β reduced expression of the DUX4 gene in muscle cells derived from patients with FSHD. Although losmapimod has never previously been explored in muscular dystrophies, it has been evaluated in more than 3,500 subjects in clinical trials across multiple other indications, including in several Phase 2 trials and a Phase 3 trial. No safety signals were attributed to losmapimod in any of these trials. Fulcrum is currently conducting Phase 2 trials investigating the safety, tolerability, and efficacy of losmapimod to treat the root cause of FSHD. 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 StatementsThis 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; 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 Communicationscwaarich@fulcrumtx.com 617-651-8664 Stephanie Ascher Stern Investor Relations, Inc.stephanie.ascher@sternir.com 212-362-1200 Media: Kaitlin GallagherBerry & Company Public Relationskgallagher@berrypr.com212-253-8881
