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Stealth BT mitochondrial myopathy trial

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Friday, February 13 2015 12:30pm EST

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This is a joint teleconference between MitoAction & UMDF for the benefit of the mitochondrial disease community.

Patients, parents, and families -- please join us Friday, Feb. 13, 2015 at 12:30 p.m. EST (9:30 a.m. Pacific) by teleconference for an update from Stealth BioTherapeutics.

Join us with Stealth BioTherapeutics CEO Travis Wilson and others from the Stealth BT team for a live update from Stealth BT (click here to check out their website), information about Bendavia, and details about StealthBioTherapeutics' 2015 mitochondrial myopathy clinical trial.

A Q&A opportunity is included in this important discussion. Parents, patients and families - please join us!
This special teleconference/webinar is offered in collaboration by the UMDF and MitoAction on Friday, Feb. 13, 2015 from 12:30-1:30 p.m. Eastern (9:30-10:30 a.m.Pacific)

For more information about the Stealth Mitochondrial Myopathy Trial, please call the trial question hotline  1-877-227-5018

Or visit  (search Bendavia)


Stealth BioTherapeutics Mitochondrial Myopathy Trial

Presented by

Travis Wilson, CEO, Stealth BioTherapeutics

Introduction - Travis Wilson, the CEO of Stealth BioTherapeutics, has had a longstanding commitment to patients with mitochondrial disease. Stealth BioTherapeutics, formerly know as Stealth Peptides, is committed to broadening its scope to include therapeutic agents for mitochondrial disease and related myopathy. Today's discussion focused on the trials with the drug Bendavia, but there are also studies in the works for another drug, Acuvia, a topical eye drop developed for the treatment of mitochondrial optic neuropathy.

Bendavia is being studied in 15 clinical trials with over 500 patients and volunteers and is part of three ongoing cardiovascular studies conducted at both the Mayo Clinic and at Harvard. At the National Institute of Health's (NIH) Annual Kidney Meeting in February 2015, Stealth reported the positive data on patients with chronic kidney disease who undergo an acute injury as evidenced by a lowering of serum creatine and improvement in kidney function. The drug has been safe and well tolerated, demonstrating a biological benefit around the mechanism as it relates to mitochondria. Bendavia, given by mouth, injection, or intravenously, interacts with Cardiolipin, a lipid on the inner mitochondrial membrane, resulting in overall improvement in disease and a modification of disease progression by: 1) an overall restoration in ATP levels, and 2) a reduction in oxidative stress. Over 100 peer reviewed abstracts describe modifications in disease with the use of Bendavia.

This year Stealth plans to:

  1. explore multiple clinical areas, including mitochondrial myopathy;
  2. enroll the first patients in skeletal muscle studies at he University of Washington, studying mitochondria’s role in diseases of aging. Stealth is hoping to see improvement in skeletal muscle function (fatigue and exercise tolerance) with the use of Benedavia;
  3. study Acuvia in clinical trials of diabetic macular edema to see if ATP levels are restored and oxidative stress is reduced;
  4. study Acuvia in Leber's Hereditary Optic Neuropathy.

Mitochondrial Myopathy Clinical Trials

There are over 270 inherited mitochondrial disorders, most with some form of myopathy. Bendavia is believed to be able to fix or restore the electron transport chain and positively impact skeletal muscle myopathy. The rationale behind looking at mitochondrial myopathy is to address one of the major and most severe symptoms of  mitochondrial disease, namely skeletal muscle fatigue and exercise intolerance, which directly impacts quality of life for patients.

There are four sites for the trials in the USA:

  • Akron Children's Hospital (Ohio), with Dr. Bruce Cohen
  • University of California at San Diego, with Dr. Richard Haas
  • Pittsburg Children's Hospital, with Dr. Amy Goldstein and Dr. Gerard Vockley
  • Massachusetts General Hospital (Boston), with Dr. Amel Kara

Inclusion/exclusion criteria

  • Genetically confirmed mitochondrial disease with one component of the disease being myopathy. Confirmed genetic diagnosis, which can be confirmed by genetic sequencing of nuclear or mitochondrial DNA from muscle, blood, urine, or cheek swab samples. Future clinical trials may include those without genetic confirmation.
  • Aged 16 - 65 years
  • Skeletal muscle function impairment (symptoms of fatigue and exercise intolerance)
  • Must be able to complete a six-minute walk test (taking a break is allowed)
  • Laboratory test results
  • Initially seeking 36 participants in order to study Bendavia's safety and tolerance levels. Hopefully participants can be enrolled quickly, and, if the trial is successful, more and larger studies can be implemented.
  • Clinical trial will be held only in the four US sitesThe dosing schedule includes an overnight hospital stay at one of the four research sites for a total of seven days. The first day is registration followed by five days of intravenous dosing (2 hours daily for 5 days).

Outcome criteria

  • Exercise Capacity measured by a six-minute walk test. Cardio-pulmonary exercise testing (CPET) to assess overall cardiac function and the volume of air able to be inhaled and exhaled.
  • Clinical laboratory tests, looking for safety and for biomarkers.

Bendavia is not approved commercially in the US nor in other countries, but clinical studies are being conducted in Europe. Phase 2 studies are also being conducted at the Mayo Clinic to look for renal function benefits.

Future Studies Some earlier nonclinical trials have shown benefits to kidney function with Bendavia, which may be studied in future studies. As part of the current clinical trial a Newcastle Score will be looked at (looking for any neurological effects), but this will not be the focus of current trials. Future trials, however, will look more carefully at neurological benefits and other parameters. Participants in the current trial may be eligible for future trials. This current trial will not track long-term results. For the current small trial, compassionate use is not available, but may become an option in the future.

Hopeful conclusions from study

  • Bendavia is well tolerated and safe
  • Results lead to larger clinical studies in 2016 using other routes of delivery (oral or injection) and eventual commercial use by 2017-2018
  • Measurable increase in skeletal muscle exercise capacity across a number of inherited mitochondrial diseases

Summary In many ways, mitochondrial disease affects everyone as the aging process involves mitochondrial dysfunction. Bendavia may be able to help those with mitochondrial disease as well as others with other disease causing myopathy. Mito patients are encouraged to consider joining this trial to advance therapeutic treatment options for mitochondrial disease.

For additional information about Stealth Mitochondrial Myopathy Trial:  (search Bendavia)

Trial Question Hot Line: 1-877-227-5018

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