Fulcrum Therapeutics Inc. (FULC) Presents Data for Potential FSHD Biomarker and Clinical Outcome Assessments at MDA

March 18, 2021 11:31 AM EDT

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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.”

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