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July 19, 2017

 

STEALTH BIOTHERAPEUTICS SHARES PROMISING DATA
FROM MITOCHONDRIAL MYOPATHY TRIAL

 

Recently, at the 2017 UMDF Mitochondrial Medicine Symposium, Stealth BioTherapeutics shared very encouraging results from its second clinical trial for primary mitochondrial myopathy, the MMPOWER-2 study. Stealth is a Boston-based biopharmaceutical company developing therapeutics to treat mitochondrial dysfunction; its lead drug under investigation is elamipretide (previously known as Bendavia). Primary mitochondrial myopathy (PMM) is a genetically-acquired mitochondrial disease characterized by signs and symptoms of myopathy (debilitating muscle weakness, easy fatigability, exercise intolerance and pain). The Phase 2 MMPOWER-2 trial was conducted to evaluate safety, tolerability and efficacy of treatment using elamipretide in 30 adult (ages 16-65) patients with PMM. An overall assessment of the top-line MMPOWER-2 results showed benefit across multiple endpoints and is supportive of continuation toward a Phase 3 study in this patient population.

Patients enrolled in MMPOWER-2 previously participated in Stealth’s MMPOWER trial, designed to assess dosing, which demonstrated a dose-dependent improvement in distance walked in the 6-Minute walk test (6MWT) after 5 days of once daily intravenous administration of elamipretide or placebo. In MMPOWER-2, patients were randomized to once daily subcutaneous administration of elamipretide or placebo for a longer period of time (four weeks), and then, after a washout period, received the opposite treatment during a second four-week dosing period.  Patients, their doctors and investigators at Stealth were unaware of which group patients belonged to during the trial and the subsequent assessments.  


This type of study design, known as a randomized, double-blind, placebo-controlled crossover study, is considered a “gold standard” in clinical trials evaluating investigational drugs. Randomized controlled trials can be challenging in rare and orphan diseases because there simply are not as many patients available to participate, and there tend to be as many differences as there are similarities between patients who may have the same diagnosis (also known as heterogeneity). 

Multiple endpoints, or outcomes, were considered and evaluated during the MMPOWER-2 trial in addition to the 6MWT, including the Neuro-QoL (Quality of Life in Neurological Disorders) measurement system, and a new patient-reported outcome tool, the Primary Mitochondrial Myopathy Symptom Assessment (PMMSA), developed by Stealth specifically for this patient population. Other tools and measures for safety, tolerability and functional assessment were also applied. 

Chuck Mohan, Executive Director of the United Mitochondrial Disease Foundation, comments “Clinical trials for rare diseases are challenging and we are very supportive of Stealth for their efforts to evaluate potential therapeutics in a meaningful, rigorous and scientific manner. The progress of the MMPOWER-2 trial provides hope to our global mitochondrial disease community.”  

The primary objective of the study was to evaluate the effect of a single daily subcutaneous dose of elamipretide for four weeks on a PMM patient’s walking distance, as measured by the 6MWT. Patients receiving elamipretide walked an average of 20 meters more than those receiving placebo at the end of the 4-week dosing period. In addition, those patients who were the most impaired at baseline demonstrated the greatest improvement.  

Data gathered and analyzed for several other secondary endpoints is also informative and promising. Treatment with elamipretide resulted in statistically significant and clinically meaningful improvements in the Neuro-QOL Fatigue Short Form score and in the PMMSA Total Fatigue score. In other words, patients showed overall improvement in fatigue and in symptoms which impacted their daily quality of life and functional abilities.  Patients also reported a statistically significant improvement in the PMMSA identified symptom most bothersome to them individually, such as tiredness, muscle weakness, muscle pain, abdominal discomfort, vision problems, or balance problems. Data also continued to demonstrate safety and tolerability of elamipretide, with the most common side effect being redness or itching at the injection site. 

Kira Mann, CEO of MitoAction, is enthusiastic about these findings. “These results are very promising for patients struggling with fatigue, pain, and weakness due to mitochondrial myopathy. On behalf of patients and families with mitochondrial disease, we are excited about this data and continue to be supportive of future elamipretide studies.”

Stealth continues to be committed to developing mitochondrial therapeutics and engaging with the mitochondrial disease clinician and patient/family community. In March of 2017, Stealth initiated RePOWER, a prospective, observational study of patients with mitochondrial myopathy. RePOWER will assess approximately 300 PMM patients, ages 16-65, and will gather information about current symptoms, quality of life, functional abilities and medical history. Findings from MMPOWER, MMPOWER-2 and RePOWER will together help establish and inform a Phase 3 trial to continue to evaluate the potential efficacy, safety and tolerability of elamipretide. Stealth plans to launch its Phase 3 trial around the end of this year.

The executive director of the Foundation for Mitochondrial Medicine, Laura Stanley, has a child with mitochondrial disease. She states, “Results such as these demonstrate why it is so important for every patient and family with primary mitochondrial disease to be involved in these very important and groundbreaking studies. Together we are pioneering this field.” 

For more information, please visit MitoAction.org, mitochondrialdiseases.org and UMDF.org.

For more information about this and other trials being conducted by Stealth Biotherapeutics, please visit www.StealthBT.com or www.clinicaltrials.gov

 

 


 

July 24, 2017

 

Parents Decide to Withdraw Treatment Request
for Little Charlie Gard


The thoughts and prayers of the entire mitochondrial disease community are with Charlie Gard and his mother and father, Chris Gard and Connie Yates.  They have decided to withdraw their request to have little Charlie treated with an experimental therapy.  Medical experts have told the family it would not be effective for him.   This is a heartbreaking situation that no parent should ever have to face.  The path the Gard family has had to follow has been complicated and very difficult.


There are more than 200 different forms of mitochondrial disease ranging from mild to severe.  The disease is difficult to diagnose because it affects each patient differently.  No two patients are alike in their symptoms or care management.

Charlie’s path is not an uncommon path for those who battle mitochondrial disease and justifies the need for more awareness, and government and private support of faster diagnostic tools, potential treatments and ultimately a cure. 

To learn more about mitochondrial disease and dysfunction, its links to other more well-known diseases and our work towards developing treatments and potential cures, visit MitoAction, FMM, UMDF.


 

MitoAction, the Foundation for Mitochondrial Medicine and UMDF have jointly authored an editorial that was sent to major newspaper outlets across the United States. It is our hope that our collective voices will be heard and that the conversation will continue regarding the urgent need for further research and awareness of mitochondrial disease. Our thoughts are with the Gard family.

 

July 11, 2017

 

Dear Editor,

The plight of UK baby Charlie Gard has brought visibility of mitochondrial disease to the world. While many are unfamiliar with the disease, no one can live without mitochondria—tiny cellular powerhouses, essential for energy creation in organ systems, like brain, heart and muscles. Charlie's genetic mutation of mitochondrial depletion may be rare, but mitochondrial disease is not – approximately 1 in 2,500-5,000, adults, teens and children are affected, making mitochondrial disease more common than childhood cancers.

 

Mitochondrial dysfunction is proven to be at the root of many common diseases and conditions that affect young and old, from Autism to Parkinson's, Alzheimer's, some cancers, and it may even be responsible for aging itself. Almost everything related to human health can be traced back to the mitochondria. Research centered on the mitochondria has the potential to be the Holy Grail of medicine.

 

The recent events surrounding baby Charlie Gard intensify and justify the need for increased awareness and support of treatment-oriented research for the many aspects of mitochondrial disease. Due to the variable course of mitochondrial dysfunction and the fact that no two patients experience the same symptoms, treatments remain frustratingly basic, consisting mostly of supportive care that focuses on symptom management and the prevention of complications.

 

With mitochondrial dysfunction contributing to so many disparate diseases, collaboration across specialties, among clinicians, researchers and pharmaceutical companies, is vital. Non-profit organizations, such as ours, are forming partnerships to maximize our work. Treatments cannot come quickly enough to help Charlie and the many other adults and children across the globe struggling every day with mitochondrial disease. To learn more about mitochondrial dysfunction and disease, and the state of research and treatment, please visit: www.mitoaction.orgwww.hopeflies.orgwww.umdf.org

 


 


July 24, 2017

 

Courtagen Life Sciences Shifts Focus to its Medicinal Cannabis Genetics and Testing Business

Woburn, Mass.--(BUSINESS WIRE)—Courtagen Life Sciences Inc. announced today that the company will be shifting the core focus of its business to medicinal cannabis genetics and testing. As the company reorients its business to respond to the growing opportunities in the medicinal cannabis industry, the company has made the decision to wind down its diagnostic neurology testing division. The company’s decision to transition out of its diagnostic business was based in part on many challenging industry dynamics, such as a poor regulatory and reimbursement landscape for genetic testing, that have made the goal of achieving long term profitability in the diagnostic space extremely difficult. Despite significant scientific advancements, the current financial status and incentive structures of the U.S. healthcare system are not aligned with optimizing such scientific advancements to ensure patients are receiving the highest quality of care.

 

“We made a very difficult yet prudent business decision to exit the diagnostic business, andrefocus our business strategy on our cannabis science business called Medicinal Genomics Corp (MGC),” said Brian McKernan, Courtagen’s Chief Executive Officer. “We stand proud of our accomplishments and of the work we did to help thousands of sick children. Furthermore, we are deeply honored to have had the opportunity to work with so many amazing and talented individuals during our time building Courtagen’s diagnostic division. Of particular importance, we would like to recognize our employees and thank them for their great attitudes, friendship, dedication, perseverance and drive to change the world for the better.

 

The cannabis industry is currently the fastest growing industry in the U.S., with 30% compounded annual growth rates. The market is expected to expand significantly in the coming years as more states and countries legalize cannabis for medical and recreational purposes. The rapidly increasing applications of cannabis in mainstream medicine is poised to increase demand for high quality, consistent, and safe medicinal cannabis for patients.

 

MGC is a commercial stage company focused on helping growers and scientists to identify desirable genetic traits of interest and to detect harmful microbes; the end result of utilizing MGC’s technologies is greater crop yields, increased sustainable productivity and safer cannabis

for patients and consumers.

 

Medicinal Genomics is the industry leader in sequencing the cannabis genome and the genomes of the most destructive cannabis pests known to date, such as Powdery Mildew and Russet Mites. MGC’s proprietary platform has enabled the rapid development of field portable genomic tests, an innovative Cannabis genotyping notarization service utilizing the blockchain and a marker assisted selection service providing Intellectual property management through

MGC’s cannabis information portal Kannapedia.net.

 

Committed to advancing the cannabis industry, MGC has created an information sharing

platform to highlight these technologies and those of worldwide medical and scientific leaders, through the annual CannMed conference series. CannMed gathers the greatest minds in medical cannabis research, where they focus on the intersection of clinical application, medical research, Cannabis based therapeutics, cannabis safety and production.

 

About Medicinal Genomics Corporation

 

Medicinal Genomics Corporation applies state-of-the-art life science technology to cannabis plant genetics, and was the first company to sequence the cannabis genome. Our products, based on Next Generation DNA Sequencing technology, help growers, dispensaries, and safety testing laboratories characterize and understand the quality of medicinal cannabis. For more information, please visit http://www.medicinalgenomics.com/