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The Mito Fund Announces Investment in Khondrion

UMDF’s venture philanthropy arm, The Mito Fund, recently announced an investment in Khondrion, a Netherlands-based clinical stage biopharmaceutical company discovering and developing therapies targeting primary mitochondrial disease (PMD).

Khondrion is currently preparing to initiate a Phase III study investigating the potential of its lead proprietary drug candidate, sonlicromanol, to treat adult patients with PMD due to the m.3243A>G mutation, the most common genetic defect causing multi-systemic PMD, which has an estimated prevalence of 4.4:100,000 impacting hundreds of thousands of patients worldwide.

One of the most advanced drug candidates in development for PMD, sonlicromanol is a disease-modifying, potentially first-in-class, brain-penetrant redox-modulator with anti-inflammatory properties that targets key metabolic and inflammatory pathways underlying PMD. Its ongoing development is supported by compelling clinical data from Khondrion’s Phase IIb program, published earlier this month in the peer-reviewed scientific journal, Brain, showing strong patient benefits from sonlicromanol in multiple outcome measures of global health, quality of life, mood, fatigue, pain and balance control.

“We believe sonlicromanol holds incredible potential for treating primary mitochondrial disease which impacts hundreds of thousands of people worldwide,” said Dr. Philip Yeske, UMDF’s Interim Managing Director and Science and Alliance Officer. “But it’s not just about numbers. The diseases associated with this mutation are devastating and remarkably progressive. The approval of this therapy would meet an urgent and unmet therapeutic need.”

Said Khondrion’s Chief Executive Officer, Prof. Dr. Jan Smeitink: “Patients with primary mitochondrial disease currently have no treatment options available to them. Through our clinical trials to date, we see the great potential sonlicromanol may have on these patients, particularly on their most burdensome symptoms of chronic fatigue and muscle weakness. With the support and investment by the UMDF via The Mito Fund, we look forward to further progressing our clinical work on sonlicromanol at pace in our upcoming Phase III trial in adult patients with PMD.”

This is the second official investment for The Mito Fund. In April 2024, the fund announced an investment in Pierrepont Therapeutics, Inc., which is working on a preclinical proprietary intracellular enzyme replacement therapy for Mitochondrial Neurogastrointestinal Encephalomyopathy (MNGIE).

mitoSHARE Assistive Tech Study Now Available

UMDF has launched a new mitoSHARE study focused on the use of assistive technology within the mitochondrial disease community. The Assistive Technology Survey assesses the needs of mito patients to help better understand what assistive technology products you use, what products you would like to use but don't, and why. This is a short survey that should take less than five minutes to complete. You can lend your voice to this by joining mitoSHARE today at umdf.org/mitoSHARE.

mitoSHARE, UMDF’s patient registry, is a worldwide research database for patients (both living and deceased) with any form of mitochondrial disease, as well as their caregivers. A confirmed diagnosis of mitochondrial disease is not required to join mitoSHARE. Patients at any stage of the diagnostic journey are encouraged to register. ”

Leaders Needed for Energy for Life Events in 2025

With UMDF celebrating another great year of Energy for Life events, volunteer leaders from across the country are already making plans for 2025. There are numerous ways to get involved now, ranging from walking, making plans for a team, or becoming a team captain. Get involved now by visiting energyforlifewalk.org or emailing events@5mr.c80.myftpupload.com.

Runners from “Team Activate” Join the November Powerhouse Podcast

In November's Powerhouse Podcast episode, listen in as UMDF’s Cassie Franklin and Angie Sommer talk with runners from the 2024 UMDF Team Activate who fundraised and ran in this year's Bank of America Chicago Marathon. Micah Fields, Lisa Clark, and Aaron Cohen share with us their training tips, race day peaks and valleys, and their experiences putting their miles toward the mission of UMDF. Listen wherever you get your podcasts or watch the episode below. Feeling inspired? Contact UMDF's Angie Sommer today to join UMDF Team Activate in 2025!

WATCH: Powerhouse Podcast #35: UMDF Team Activate 2024 & the Bank of America Chicago Marathon - YouTube

UMDF Ask the Mito Doc Webcast Series – Wrapping Up 2024 with the Mito Docs

Join us for an Ask the Mito Doc webinar wrapping up 2024 on December 11 at 7pm ET, featuring Amy Goldstein, MD of Children’s Hospital of Philadelphia, Hilary Vernon, MD, PhD of Johns Hopkins University, Matthew Snyder, MD of University of Virginia, and Russell Saneto, DO, PhD of Seattle Children’s Hospital. Click here to register or here to submit questions beforehand.

WATCH: Available for replay now is last month’s Ask the Mito Doc focused on Understanding POLG Disorders: A Dual Perspective on Research and Clinical Practice, featuring William Copeland, PhD of National Institute of Environmental Health Sciences, and Bruce Cohen, MD of Akron Children’s Hospital. Click here to watch.

WATCH: View our archive of Ask the Mito Doc webinars now on umdf.org.

All Welcome on Bench-to-Bedside Seminars in December & January Focused on Clinical Trial Updates

All in the mitochondrial disease community are invited to a special UMDF Bench-to-Bedside Clinical Trial Updates on December 9 at 12pm ET where you’ll get updates on clinical trials from representatives of some of the leading companies in the mitochondrial disease therapeutic pipeline, including BPGbio Inc., Minovia Therapeutics, OMEICOS, Saol Therapeutics, Stealth BioTherapeutics, Thiogenesis Therapeutics, and UCB. Click here to register.

Note that MELAS-related trial updates – including speakers from Abliva, Baylor College of Medicine, Khondrion, and Tisento Therapeutics – will take place on the Bench-to-Bedside on January 13, 2025 at 12pm ET. Click here to register.

Related Posts: UMDF’s Therapeutic Pipeline page

WATCH: View all previous Bench-to-Bedside Seminars at umdf.org/benchtobedside.

Studies: Adolescent Patients with MELAS Needed for Study

Adolescents living with the mitochondrial disease MELAS are needed to collect perspectives that will help shape how the effects of a drug in development will be assessed in an upcoming clinical trial.

The study is open to any English-speaking patients ages 12 – 17 who are clinically and genetically diagnosed with MELAS. Participants will receive $150 for completing two 45-minute interviews ($75 per interview) from the comfort of your home and at your convenience.

Click here for more information and details on how to enroll.

Related Posts: UMDF Surveys & Studies page

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If you were one of the hundreds of families who supported UMDF during Giving Tuesday, thank you for your commitment to the mitochondrial disease community. If you missed it, there are still plenty of ways to support UMDF before the end of the year!

Give Energy This Holiday Season
UMDF’s annual holiday giving campaign, Give Energy, is now underway. This year’s campaign tells the story of twin 4-year-old girls Kendall and Scarlett George, both of whom are affected by THG1L disorder, a mitochondrial disease that heavily disrupts energy production to the brain and muscles. Make your Give Energy gift today.

When you give the Gift of Energy this holiday season to UMDF, you could double that gift with a company match. Check with the HR staff at your place of employment or see for yourself on the matching gifts page on umdf.org.

Leave a Legacy with Planned Giving
There are many ways to support the great work of UMDF and our drive to help develop treatments and cures for mitochondrial disease. One of the most practical – and often overlooked – is including UMDF in your estate planning. Whether it’s through a Donor Advised Fund, Stock Donation, or a Bequest in your will, the Development Team at UMDF is here to help you achieve your goals and create your Legacy of Hope.

To learn more, download our free Legacy of Hope Planned Giving Brochure today or visit our Legacy of Hope Webpage. Already included UMDF in your estate plan? Please let us know.

Turn Your Old Vehicle into Research Funding
This holiday season, give the gift of a vehicle donation to UMDF and help drive our mission toward treatments and cures for mitochondrial disease. Click here to learn more.

Make a Tax-Friendly Donation to UMDF using your IRA
If you’re over 70.5 years old and are trying to maximize Qualified Charitable Distribution (QCD), you may be able to make a tax-friendly gift directly from your IRA to support UMDF. To learn more, contact your IRA administrator and provide UMDF’s details (United Mitochondrial Disease Foundation, 8085 Saltsburg Road, Suite 201, Pittsburgh, PA 15239; Federal Tax-ID 25-1767180). Remember, QDCs must be made by December 31, 2024 to qualify for the 2024 tax year. If you need more information, please reach out to UMDF’s Development team via phone 1-888-317-UMDF or email.

UMDF Swag Makes Perfect Stocking Stuffers
Cozy UMDF sweatshirts and soft tees make for the perfect holiday gift, all while helping fund services to the mito community! Find those and more at the UMDF Official Webstore at willpromo.com/umdf. Please allow two to three weeks for orders to process and arrive, depending upon location.

Mitochondrial Medicine 2025 Special Section

Save the Date for Mito Med

Make sure to mark your calendar for UMDF’s Mitochondrial Medicine Conference scheduled for June 18-21, 2025, at Hyatt Regency St. Louis at The Arch. Registration is expected to open in late January 2025.

Each year, UMDF’s Mitochondrial Medicine Conference brings together the best minds in mitochondrial medicine and the patients we serve. With over 700 attendees representing almost every state in the U.S. and more than 15 different countries, the Conference is recognized as the world’s preeminent event for mitochondrial disease.

Learn more at UMDFConference.org.

Mitochondrial Medicine 2025 to Host “Masterclass” for the First Time Ever

UMDF is proud to announce the Mitochondrial Medicine Conference in 2025 will feature a masterclass for the scientific and medical community.

The focus of the 2025 masterclass will be the mitochondrial disease Thymidine kinase 2 deficiency (TK2d). The class, scheduled for Saturday, June 21 at the Hyatt Regency St. Louis at The Arch, will feature a dual-track program to provide essential education for up-and-coming clinicians and early career investigators to increase their understanding of the diagnostic journey, care, and outcomes of patients living with mitochondrial disorders, specifically TK2d, and the families whose lives are impacted by these conditions. The early career track will provide insight into building a successful academic career, working with industry, and offering guidance in navigating job searches. Stay tuned for more details. Funded via an unrestricted medical education grant by UCB.

Attention Researchers -- Abstract Submissions Open Soon

As with previous years, UMDF will once again provide the research community the opportunity to share abstracts of their projects during the Mitochondrial Medicine Conference 2025 in St. Louis. Abstracts should highlight major aims of your research project so conference attendees can quickly understand the purpose and final conclusion of your work.

All submissions will be peer-reviewed by the Abstract Review Committee. Approximately 18-22 abstracts will be selected for oral presentations during select platforms. All approved submissions will have the opportunity to share their projects during the dedicated poster sessions. A Call for Abstracts for Mitochondrial Medicine 2025 in St. Louis will open in mid-December. NEW this year – UMDF will be offering a limited number of travel scholarships. You must submit an abstract to apply for this scholarship.

Stay tuned to umdfconference.org for more information.