Breaking News
April 20, 2018 - FDA Alert: Certain Kratom-Containing Powder Products by Viable Solutions: Recall -Possible Salmonella Contamination
April 20, 2018 - What is heart failure?
April 20, 2018 - Inactivity of astronauts during spaceflights may have more pronounced effect on skeletal muscle than hypoxia
April 20, 2018 - New SIDS Info app seeks to reduce infant sleep-related deaths
April 20, 2018 - Wide-scale distribution of naloxone effectively prevents overdose deaths, study finds
April 20, 2018 - Triple-negative breast cancer found to be chemoresistant prior to treatment
April 20, 2018 - ACL tears occur the same way in women and men, study finds
April 20, 2018 - UT Southwestern researchers identify 170 potential therapeutic targets for lung cancer
April 20, 2018 - Finding the ‘keyhole’ to beat obesity at the cellular level
April 20, 2018 - Long-term exposure to cold temperatures reduces diabetes and obesity, shows study
April 20, 2018 - Metabolic Syndrome Common With Chronic Hep B Infection
April 20, 2018 - Tracking quality of life during prostate cancer treatment
April 20, 2018 - Study shows presence of beta-amyloid dimers in the brains of Alzheimer’s patients
April 20, 2018 - Researchers identify link between physical inactivity and increased risk of dying from cancer
April 20, 2018 - Breathtaking evolution amongst Indonesian tribe
April 20, 2018 - Study shows testosterone deficiency in men is associated with chronic diseases
April 20, 2018 - Simple one-page form helps improve satisfaction of patients with care
April 20, 2018 - Researchers evaluate accuracy of simple blood test to predict lung cancer
April 20, 2018 - Study looks at sperm producing ability in testicular cancer patients
April 20, 2018 - Exercise In, Vitamin D Out for Preventing Falls: U.S. Panel
April 20, 2018 - Skin cancers associated with decreased risk of developing AD
April 20, 2018 - Preserving fertility during chemotherapy
April 20, 2018 - Teva and Procter & Gamble Company terminate PGT Healthcare partnership
April 20, 2018 - People diagnosed with traumatic brain injury may have increased risk of Parkinson’s disease, shows study
April 20, 2018 - Researchers use smartphone to diagnose people infected with Loa loa worm
April 20, 2018 - College students with autism have high rate of suicidal thoughts
April 20, 2018 - Study sheds light on how the HSC niche is maintained
April 20, 2018 - Drug test spurs frank talk between hypertension patients and doctors
April 19, 2018 - Low-cost deworming drug improves female farmers’ physical fitness
April 19, 2018 - Genome editing identifies neural circuit behind leptin’s anti-obesity and anti-diabetes effects
April 19, 2018 - Many European countries lack comprehensive policy to eliminate viral hepatitis
April 19, 2018 - Young people with ADHD ‘more likely’ to come from deprived neighbourhoods
April 19, 2018 - SLU professor discovers new biomarkers for chlorine gas exposure
April 19, 2018 - Study proposes new mechanism that may contribute to gender differences in weight control
April 19, 2018 - Sleep restriction therapy does not interfere with insomnia patient’s driving ability, research shows
April 19, 2018 - Deep brain stimulation offers relief to UTHealth patient with treatment-resistant depression
April 19, 2018 - Study shows fatty fish and camelina oil boost HDL and IDL cholesterol
April 19, 2018 - FDA Alert: Euphoric Capsules by Epic Products: Recall
April 19, 2018 - Researchers identify peptide produced during cartilage deterioration as a potential source of osteoarthritis pain
April 19, 2018 - New breakthrough may allow scientists to orchestrate tissue regeneration in humans
April 19, 2018 - SYGNIS AG introduces new TruePrime apoptotic cell free DNA amplification kit
April 19, 2018 - Innovative device shows promise in capturing and releasing circulating tumor cells
April 19, 2018 - Researchers shed light on role of striosomal neurons in reinforcement learning
April 19, 2018 - Genetic make-up impacts long-term effectiveness of phobia treatment
April 19, 2018 - Novel biomarker can distinguish malignant lung nodules
April 19, 2018 - Study reports promising novel approach to treat therapy resistant pediatric brain tumors
April 19, 2018 - One-Hour Plasma Glucose Useful Predictor of Diabetic Retinopathy
April 19, 2018 - Hydroxychloroquine no more effective than placebo for relieving osteoarthritis hand pain
April 19, 2018 - Transplanted livers have a protective effect and reduce potential for organ rejection
April 19, 2018 - Researchers develop new method to study activity of inflammatory cells
April 19, 2018 - Researchers discover highly antibiotic resistant superbugs in Gulf States
April 19, 2018 - Smart-tooth technology shows promise in detecting certain diseases in high-risk patients
April 19, 2018 - Interaction between dioxin and HLA gene variant activates events associated with rheumatoid arthritis
April 19, 2018 - Eyes of adolescents could be associated with increased risk of cardiovascular disease in later life
April 19, 2018 - U.S. Women Less Likely Than Men to Get Statins After Heart Attack
April 19, 2018 - Health Canada grants cannabis cultivation license to High Park Farms
April 19, 2018 - Atypical brain development observed in preschoolers with ADHD symptoms
April 19, 2018 - SC Johnson releases annual Sustainability Report
April 19, 2018 - Positive attitudes about aging reduce risk of dementia in older adults
April 19, 2018 - Environmental pollutants found to worsen rheumatoid arthritis
April 19, 2018 - UT Southwestern scientists discover protein linked to metastatic breast cancer
April 19, 2018 - Study highlights need for further evidence to improve symptom management in end of life care
April 19, 2018 - Detecting diminished dopamine-firing cells inside brain could reveal earliest signs of Alzheimer’s
April 19, 2018 - Uniqsis offers high-power LED light unit for scalable flow photochemistry reactions
April 19, 2018 - Case study shows how intravascular ultrasound imaging helps detect acute aortic syndrome
April 19, 2018 - Research reveals new mechanism by which HIV evades the immune system
April 19, 2018 - Nanodisc-delivered cancer treatment helps eliminate tumors
April 19, 2018 - Functional connectivity MRI could help detect brain disorders and diseases
April 19, 2018 - Finding better way to quantify neuropathy symptoms and treatment efficacy
April 19, 2018 - Study examines effectiveness of caregiver education about sickle cell trait
April 19, 2018 - High-resolution images of tumor vasculature using new technology
April 19, 2018 - Lack of sleep may be linked to risk factor for Alzheimer’s disease
April 19, 2018 - Study finds neurotransmitter may play a role in alcohol relapse, addiction
April 19, 2018 - Researchers build molecular networks of calcific aortic valve disease
April 19, 2018 - Researchers develop highly specific apoptosis assay for pharmacodynamic analyses of tumor specimens
April 19, 2018 - Scientists decipher mechanism of chemotherapy induced female infertility
April 19, 2018 - New insight may allow researchers to design drugs that improve immune responses to vaccines
April 19, 2018 - FDA Approves Crysvita (burosumab-twza) for X-Linked Hypophosphatemia
April 19, 2018 - Researchers uncover origin of virus-fighting plasma B cells
April 19, 2018 - Study finds no evidence of lower intelligence in young children who had anesthesia
Santhera Reports Outcome of Exploratory Trial with Idebenone in PPMS Conducted at the NIH

Santhera Reports Outcome of Exploratory Trial with Idebenone in PPMS Conducted at the NIH

image_pdfDownload PDFimage_print

Pratteln, Switzerland, March 5, 2018 – Santhera Pharmaceuticals (SIX: SANN) announces that the exploratory Phase I/II clinical trial in primary progressive multiple sclerosis (PPMS) conducted as an investigator-initiated trial at the National Institutes of Health (NIH) confirms the safety profile of idebenone at a dose of 2,250 mg daily over a treatment period of two years. In assessing the efficacy of idebenone on disease progression, no difference between the active treatment group and placebo was observed.

The National Institute of Neurological Disorders and Stroke (NINDS), part of the US NIH, sponsored this investigator-initiated, double-blind, placebo-controlled Phase I/II trial (IPPoMS) investigating the safety and therapeutic efficacy of idebenone in PPMS. A total of 77 patients were randomized and 66 (idebenone: 33, placebo: 33) completed the trial which combined a one-year observational pre-treatment phase, followed by a two-year placebo-controlled intervention period. There was no difference in the occurrence and severity of adverse events between the treatment groups indicating that idebenone at a daily dose of 2,250 mg was well tolerated.

The primary outcome to explore the efficacy of idebenone was the change in the CombiWISE, a rating scale recently developed by Bibiana Bielekova, MD, and colleagues at the NINDS. Top-line analysis of the CombiWISE data and other clinical assessments and biomarkers (such as the disability progression scale EDSS-plus and changes in ventricular volume) indicate that there was no difference between treatment groups for measures of disease progression.

“The long-term study in patients with PPMS confirms the favorable safety profile of idebenone given at higher dose than the currently approved dose for Raxone® in Leber’s hereditary optic neuropathy,” said Thomas Meier, PhD, CEO of Santhera. “We thank the NIH team for conducting this long-term pilot study which will add to the knowledge of disease progression and data collection instruments. Clearly, the small sample size is a limitation when studying a therapeutic intervention in such a complex, relentlessly progressing neurological disease.”

About PPMS

Multiple sclerosis (MS) is an inflammatory and neurodegenerative disorder of the central nervous system that causes a wide range of physical symptoms, such as impaired movement, fatigue, numbness, and pins and needles, as well as problems with memory and understanding. In MS, the outer coating of nerve fibers (called myelin) is damaged, preventing the nerves from functioning properly. Primary progressive multiple sclerosis (PPMS) is the more aggressive of two main subtypes of MS and affects about 10-15% of all patients with MS. In remittent relapsing MS (RRMS), patients experience symptoms intermittently, with a slower accumulation of permanent disability than in PPMS. In PPMS, physical disability progressively worsens over time without symptom-free intervals.

About Santhera

Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative medicines for orphan and other diseases with high unmet medical needs. The portfolio comprises clinical stage and marketed treatments for neuro-ophthalmologic, neuromuscular and pulmonary diseases. The most advanced pipeline product, idebenone, is in clinical Phase III for the treatment of Duchenne muscular dystrophy (DMD). Santhera’s Raxone® (idebenone) is authorized in the European Union, Norway, Iceland, Liechtenstein and Israel for the treatment of Leber’s hereditary optic neuropathy (LHON) and currently commercialized in 20 countries. For further information, please visit www.santhera.com.

Disclaimer / Forward-looking statements

This communication does not constitute an offer or invitation to subscribe for or purchase any securities of Santhera Pharmaceuticals Holding AG. This publication may contain certain forward-looking statements concerning the Company and its business. Such statements involve certain risks, uncertainties and other factors which could cause the actual results, financial condition, performance or achievements of the Company to be materially different from those expressed or implied by such statements. Readers should therefore not place undue reliance on these statements, particularly not in connection with any contract or investment decision. The Company disclaims any obligation to update these forward-looking statements.

Source: Santhera Pharmaceuticals

Posted: March 2018

Tagged with:

About author

Related Articles