Clenbuterol Hydrochloride

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 (), 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 31st 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 . SOURCE Springbok Analytics

Drug Combination’s Unique Mechanism of Action Enables Reactivation of Brain Adrenergic Function, Showing Improvements in Multiple Areas of Cognition SAN CARLOS, Calif. & LISBON, Portugal--(BUSINESS WIRE)-- CuraSen Therapeutics, Inc., a clinical-stage company developing small molecule therapies to treat neurodegenerative disease, announced that it will present additional positive Phase 2a data with its combination adrenergic activator, CST-2032/CST-107, in patients with mild cognitive impairment (MCI) or mild dementia from either Alzheimer’s or Parkinson’s disease. The oral presentation will take place at AD/PD™ 2024: The International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders, being held March 5-9, 2024, in Lisbon, Portugal. CST-2032 is one of CuraSen’s two lead product candidates being tested in patients with Alzheimer’s and Parkinson’s disease. Both CST-2032 and CST-103, the company’s other lead clinical candidate, are oral, brain-permeant, beta2 adrenoceptor (β2-AR) agonists delivered in combination with CST-107 (nadolol, a brain-sparing beta blocker) to inhibit peripheral effects of β2-AR agonism. Both CST-2032 and CST-103 work by enabling reactivation of brain adrenergic function lost early in the neurodegenerative disease process. The updated Phase 2a data with CST-2032/CST-107 showed statistically and clinically important effects across several cognitive domains in patients with Alzheimer’s and Parkinson’s disease, such as memory, attention, executive function, facial recognition and impulse control — areas known to be impacted by adrenergic function. Target effect sizes of 0.2-0.3 or higher, as seen in this study, predict success in larger, longer studies. “These encouraging data build on earlier results presented at October 2023’s CTAD conference, reinforcing that treatment with CST 2032/CST-107 can produce meaningful quality-of-life cognitive improvements for Alzheimer’s and Parkinson’s patients, particularly those with mild cognitive impairment, rapidly and safely,” said Anthony Ford, PhD, chief executive officer of CuraSen. “The robust, collective data set from both CST-2032 and CST-103 demonstrate that this novel mechanism of action for restoring adrenergic function to the brain is a highly compelling strategy to address unmet symptoms of neurodegeneration. We plan to evaluate additional doses and refine optimal patient profiles in our upcoming Phase 2 studies, with the goal to significantly increase cognitive performance and ultimately, halt disease progression.” The randomized, placebo-controlled, double-blinded crossover study evaluated 64 patients with MCI or mild dementia from either Alzheimer’s disease or Parkinson’s diseases: AD with MCI = 30 patients randomized; 25 completed AD with dementia = 6 patients randomized; 5 completed PD with MCI = 22 patients randomized; 21 completed PD with dementia = 6 patients randomized; 4 completed Data highlights included: Digit Symbol Substitution test, which measures complex attention, memory and executive function: AD patients with MCI = 0.36 effect size; p=0.02 on Day 14 Facial Expression Recognition response time to accurately recognize facial expressions, which measures social cognition: AD patients with MCI = 0.66 effect size; p=0.04 on Day 14 for happy expression and 0.91 effect size; p = 0.01 on Day 14 for surprise expression Stop Signal test, which measures impulse control PD patients with MCI = 0.52 effect size; p=0.04 on Day 14 Excellent safety and tolerability pro /> No serious adverse events: no increases in heart rate observed one to four hours after dose The company plans to initiate longer studies with both CST-2032/CST-107 and CST-103/CST-107 in AD and PD patients with MCI later this year, as well as develop fixed-dose combination tablets for each of these drug candidates. Study Objectives and Design The study objectives were to establish safety and tolerability of CST-2032/CST-107, identify optimal dosing, and identify cognitive tests responsive to agonist stimulus within the two-week dosing period. Measures of cognition included the digit symbol substitution test, Cambridge neuropsychological test battery, and the facial expression recognition task. The study took place at multiple sites in the United States and New Zealand. Patients received either daily 3 mg of CST-2032 with 3 mg of nadolol for 14 days, or matching placebo, followed by a wash-out period (≥7-d) before crossing over to the other treatment arm. Blood samples are being analyzed for AD biomarkers, and DNA samples for AD and PD genotyping. Abstract Details Title: “CST-2032, a Novel Beta-2 Adrenoceptor Agonist, Improves Cognition in Patients with Impairment Due to Alzheimer’s or Parkinson’s Disease” Abstract #: PO34 Presenter: Gabriel Vargas, MD, PhD, chief medical officer, CuraSen Therapeutics Session: Translational Drug Discovery and Experimental Models 05 Date: Saturday, March 9, 2024 Time: 17:25-17:40 UTC, session time,16:40 - 18:40 UTC Location: Auditorium VII About CuraSen Therapeutics CuraSen is focused on the development of new treatments for neurodegenerative diseases, including Parkinson’s disease, Alzheimer’s disease and other related orphan conditions. CuraSen’s drugs are designed to activate certain receptor populations in the brain to compensate for critical neuronal and glial functions that have otherwise been lost due to degeneration and represent a unique approach in the field. The company is evaluating CST-103 and CST-2032, both selective β2-AR agonists, in combination with CST-107, a β-AR blocker, in multiple Phase 2 clinical studies. CST-3056, an α1A-AR agonist, is in preclinical development for the treatment of Alzheimer’s disease. For more information, please visit .