Springbok Analytics Announces Phase 1 Clinical Trial Evaluating Clenbuterol as FSHD Treatment

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Jun 12, 2024, 09:00 ET

This dose escalation study for safety and tolerability of clenbuterol incorporates Springbok's precision muscle MRI analysis and molecular outcome measures

CHARLOTTESVILLE, Va., June 12, 2024 /PRNewswire/ - Springbok Analytics (www.springbokanalytics.com), a life sciences muscle analytics company, announced today its participation in a Phase 1 Clinical Trial to evaluate safe dosage for clenbuterol as a treatment for Facioscapulohumeral muscular dystrophy (FSHD). Springbok's MRI-based muscle analysis helps researchers to better understand and identify disease progression of FSHD by providing individual muscle volume and fat fraction metrics in trial participants. Beyond studying the safety and tolerability of clenbuterol, Springbok is focused on strengthening and extending the foundations for future FSHD clinical trial design and execution. Springbok is also supporting the 31^st Annual FSHD Society International Research Congress held this week in Denver.

This trial is part of a larger NIH Wellstone grant led by Jeff Chamberlain, MD and Stephen Tapscott, MD, PhD, with trial activities coordinated by Jeffrey M. Statland, MD and Rabi Tawil, MD, who direct the FSHD Clinical Trials Research Network (CTRN) out of the University of Kansas and the University of Rochester, respectively. Seth Friedman, PhD at Seattle Children's is overseeing imaging implementation. "We are excited to expand the scope of our work with Springbok Analytics. This technology can benefit an FSHD-focused clinical trial by giving us new perspectives on what can be modulated by drug therapy," said Dr. Friedman. "The shorter exam duration will improve patient tolerability and Springbok's comprehensive individual muscle analytics get us closer to deriving personalized progression models."

FSHD is one of the most prevalent muscular dystrophies, affecting an estimated 20,000 people in the United States alone. There are currently no FDA approved therapies for FSHD, which represents both a large and unmet medical need. Clenbuterol has been shown to decrease DUX4-related targets in patient-derived FSHD cell assays, and several small studies in neuromuscular disorders have demonstrated improved strength or function with clenbuterol.

Springbok's AI advancements have also led to improvements in both imaging and processing time. A recent study focused on the disease burden of FSHD clinical trial participants¹ highlighted a strong preference for imaging over invasive muscle biopsies, which often remove healthy muscle tissue, as well as the need to reducing imaging time. 85 percent of the FSHD patients surveyed said that they would be willing to take part in MRI, compared to just 44 percent for an open muscle biopsy. For participants that indicated they would be willing to undergo an MRI, just 30 percent said they would be willing to sit in an MRI machine for two hours or more. Springbok's full body analysis can be done in under 40 minutes, and its protocols have been designed with scalability in mind, knowing that flexibility and ease-of-deployment are requirements across global multi-site studies.

"The rationale for this trial is that if clenbuterol is safe and well tolerated, and can increase muscle mass and reduce DUX4 levels, it can be repurposed to both stop disease progression and restore motor function," said Jeffrey M. Statland, MD, professor of neurology at the University of Kansas Medical Center. "Springbok's precision and sensitivity are so important in studying a disease like FSHD, which impacts the form and function of individuals, and their specific muscles, so differently."

Springbok scans are also accompanied by patient-facing reports with 3D muscle visualizations, empowering individuals to better manage their muscle health and disease progression. "The patient experience in clinical trials is always important, especially when there are accessibility issues. From what we have heard from many FSHD patients, length of time in the scanner and lack of reporting are factors of consideration in trial participation," said Scott Magargee, Springbok Analytics Co-Founder and CEO. "We are helping researchers and pharmaceutical companies to run more effective clinical trials by generating more precise and personalized insights, supporting those on the leading edge of FSHD research pursuing improved treatments, and ultimately, a cure."

Springbok's flexible imaging protocol has been implemented at hundreds of imaging centers around the world and is compatible across all major scanner platforms. The company is also developing standardized guidance for how to use muscle MRI as a trial passport to screen for drug trial enrollment. The FSHD Clinical Trial Research Network (CTRN) is being leveraged as part of this trial, and the data is intended to ultimately support a Phase 3 trial to confirm the efficacy of Clenbuterol in FSHD. To learn more about Springbok's muscle analysis capabilities for clinical trials, visit here.

About Springbok Analytics:

Springbok is a life sciences muscle analytics company that drives better health and performance outcomes.

Its technology analyzes MRI data and creates personalized 3D visualizations of muscle health, enhancing the assessment, treatment monitoring, research and diagnostic value of advanced imaging. Springbok's rapid imaging sequence and AI-based analysis reveals a complete view of musculoskeletal health, precisely quantified individual muscle volume and quality, fat infiltration, left-right asymmetries, as well as scar tissue, edema, and tendon morphology.

To learn more about how Springbok is creating a better view of health, please visit www.springbokanalytics.com.