Khondrion completes enrollment in KHENERGYZE Phase IIb trial evaluating sonlicromanol in adult patients with MELAS spectrum disorders
MEDIA RELEASE: Khondrion, a clinical stage biopharmaceutical company discovering and developing therapies targeting primary mitochondrial diseases, today announced that the last patient has been dosed with sonlicromanol in the KHENERGYZE Phase IIb clinical study. Sonlicromanol is Khondrion’s wholly-owned, lead asset being developed to treat a range of mitochondrial diseases in children and adults. Topline data from KHENERGYZE is expected in the third quarter of 2022.
The study’s primary objective is to evaluate the dose-effect of sonlicromanol on the attention domain score of cognitive functioning, as assessed by the computerised Cogstate visual identification test. Cognitive impairment is becoming increasingly recognised in mitochondrial disease patients and can have a significant and debilitating impact on many aspects of their lives. Sonlicromanol’s potential to counteract cognitive decline is supported by preclinical research and results from a completed Phase IIa study, which showed a significant improvement in attention and mood related outcomes in patients treated with sonlicromanol compared to placebo.
Prof. Dr. Jan Smeitink, Chief Executive Officer at Khondrion, said: "Completing enrollment in our Phase IIb study is an important milestone and brings us another step closer to providing an urgent and much needed disease-modifying therapy to patients with severe and debilitating mitochondrial diseases. I would like to express my gratitude to all the patients and their families for their participation in this trial, and to the investigators and wider team, whose dedication has been vital in advancing this important clinical programme. We look forward to receiving results from the trial later in the year.”
Sonlicromanol is a first-in-class, oral small molecule targeting key underlying mechanisms of mitochondrial disease based on its unique triple mode of action: redox modulation to help restore the cell’s metabolism, radical trapping preventing ferroptotic cell death, and mPGES-1 inhibition resulting in anti-inflammatory effects. In Phase I and Phase IIa studies, sonlicromanol showed a good safety and tolerability profile well beyond target therapeutic dosing levels.
KHENERGYZE is a double-blind, randomised, placebo-controlled, multi-centre, three-way cross-over study examining cognitive function in adult patients with a specific genetically confirmed DNA mutation in the mitochondrial transfer RNALeu(UUR) (MT-TL1m.3243A>G). This mutation is responsible for MELAS spectrum disorders, including MELAS (mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes), MIDD (maternally inherited diabetes and deafness) syndromes, and mixed phenotypes.
Sonlicromanol’s development programme also includes two other ongoing clinical studies: the KHENEREXT Phase IIb open label extension study, examining the long-term safety and efficacy of sonlicromanol in adult patients who have completed the KHENERGYZE study, and the KHENERGYC Phase II study in children.
Further details of the KHENERGYZE study are available on clinicaltrials.gov.
Mitochondrial Donation in Australia
Mitochondrial donation has taken a step further to become available to families in Australia.
Sixty eminent scientists, doctors and medical experts signed an open letter in support of Maeve's Law, urging the Australian Federal Parliamentarians to vote Yes. At the end of November, the House of Representatives voted yes for Maeve's Law and now the Bill sits with the Senate. IMP has provided support to the Mito Foundation and congratulations Sean Murray and his team on their dedication to changing legislation to giving families affected by mito the opportunity to use mito donation to have children free from mito.
Highlights from World Mitochondrial Disease Week 2021
Thank you to all our members who participated in World Mitochondrial Disease Week 2021 and raised awareness around the world. Working together we raised awareness, highlighted many of the issues facing people with mito, and celebrated our incredible mito warriors around the world. Our members hosted events including sponsored runs and walks, symposiums, support meetings, and workshops.
LHON Deutschland e.V. Joins IMP
Welcome to our newest member LHON Deutschland e.V. IMP is excited to work with them to support people living with LHON around the world. LHON Deutschland e.V. has played an important role in the last two LHON Awareness Day events by providing patients who have shared their experiences during the Facebook live events.
Tom Schuster commented, “We are a German-speaking self-help association for people with the very rare eye disease LHON. We want to make rare things visible and look with you strengthened, into the future! The focus of our young association is self-help and the creation of awareness for LHON. Therefore, we are committed to a broad education, so that LHON becomes more visible and better diagnosable. In the sense of the founding spirit we bring together patients, relatives as well as interested persons, create common networking and personal exchange. With the decision to join IMP, we take networking to a new, international level. We want to be an informative and helpful anchor for everyone and accompany and support you in your life with LHON.”
Paula Morandi, IMP Board Member commented, “As the LHON Patients Representative for Mitocon and IMP I’m extremely pleased to welcome LHON Deutschland e.V. to IMP as I have always expressed the importance to spread awareness on LHON particularly as it is often mistaken for an eye or retinal disorder which has nothing to do with the MTDNA. Today, since we have a drug which has been approved by EMA for the treatment of LHON, it is very important to join forces so as to reach a diagnosis as soon as possible and start treatment before it’s too late. IMP is the perfect network which brings patients and clinicians together towards finding a cure."
For more information visit LHON Deutschland e.V.'s website.
First ever United Nations Resolution to Increase Visibility for the 300 Million Persons Living with a Rare Disease
A global grassroots campaign led by persons living with a rare disease and their families has succeeded in securing the adoption of the first-ever UN Resolution on “Addressing the Challenges of Persons Living with a Rare Disease and their Families.”
On 16 December 2021, Rare Diseases International (RDI), the NGO Committee for Rare Diseases, and EURORDIS-Rare Diseases Europe announced that the UN General Assembly formally adopted a resolution recognising the over 300 million Persons Living with a Rare Disease (PLWRD) worldwide and their families.
The Resolution focuses on the importance of non-discrimination and advances key pillars of the UN Sustainable Development Goals (SDGs), including access to education and decent work, reducing poverty, tackling gender inequality, and supporting participation in society.
Adopted by consensus by the 193 UN Member States
The Resolution, proposed by Spain, Brazil, and Qatar and co-sponsored by 54 countries, was adopted by consensus with the support from all 193 UN Member States of the General Assembly. This follows the UN Third Committee’s adoption of a ground-breaking text as part of its agenda on Social Development and is a significant turning point that places the rare disease community firmly on the agenda of the UN.
“Addressing the specific challenges faced by Persons Living with a Rare Disease is a necessary step toward making this vulnerable population visible and contributing to the UN Agenda 2030 SDGs,” said Flaminia Macchia, Rare Diseases International Executive Director. “The consensus in support of the Resolution sends a clear message that Leaving No One Behind is a priority for the UN”.
Presenting the proposal to the UN Third Committee, Maria Bassols, Deputy Permanent Representative of Spain to the UN, affirmed on behalf of the Core Group of Member States that " Persons Living with a Rare Disease are at greater risk of stigmatisation, as well as intersecting forms of discrimination that are obstacles to their full participation in society”.
The global, landmark campaign for equity that achieved the Resolution was the outcome of coordinated and tireless advocacy led by civil society partners, including the NGO Committee for Rare Diseases, RDI, and EURORDIS, and engages national rare disease groups active in over 100 countries. The civil society partners worked to frame the needs of PLWRD beyond health and to illustrate the holistic impact of life with a rare disease on the whole family.
"This UN Resolution within the 2030 Agenda and its SDGs is a supportive global framework to encourage regional policy and action. In the European Union, it should translate into a Europe’s Action Plan for Rare Diseases," said EURORDIS-Rare Diseases Europe’s Chief Executive Officer, Yann Le Cam.
Anders Olauson, Chair of the NGO Committee, stated: “The UNGA Resolution illustrates the power of the global community. Individually, rare diseases are rare, but together PLWRD constitute a significant community deserving of UN support and recognition. Together, we are a powerful and inspirational voice.”
Civil society partners are now working to approach the World Health Organization (WHO) to continue the momentum from this historic achievement and call for a resolution that focuses on health equity and strengthening care systems for PLWRD
The impact of the Resolution
This first Resolution on PLWRD represents a major shift in the global policy landscape, promising greater integration of rare diseases in the agenda and priorities of the UN system.
"This UN Resolution within the 2030 Agenda and its SDGs is a supportive global framework to encourage regional policy and action. In the European Union, it should translate into a Europe’s Action Plan for Rare Diseases," said EURORDIS-Rare Diseases Europe’s Chief Executive Officer, Yann Le Cam.
Anders Olauson, Chair of the NGO Committee, stated: “The UNGA Resolution illustrates the power of the global community. Individually, rare diseases are rare, but together PLWRD constitute a significant community deserving of UN support and recognition. Together, we are a powerful and inspirational voice.”
Civil society partners are now working to approach the World Health Organization (WHO) to continue the momentum from this historic achievement and call for a resolution that focuses on health equity and strengthening care systems for PLWRD.
For information about how you can spread the word about this incredible milestone, visit Rare Diseases International.
© 2023 Int. Mito Patients