A Phase IIb Open-label, Multi-centre, Extension Study to Explore the Long-term Safety and Efficacy of KH176 in Subjects With a Genetically Confirmed Mitochondrial DNA tRNALeu(UUR) m.3243A>G Mutation Who Have Completed the KHENERGYZE Study KH176-202.

This is an open-label, multi-centre study in subjects with a genetically confirmed mitochondrial deoxyribonucleic acid (DNA) transfer ribonucleic acid (tRNA)Leu(UUR) m.3243A>G mutation who completed study KH176-202. In the KH176-203 study subjects will be receiving KH176 100 mg BID or KH176 50 mg bid in die (BID) (as determined by the investigator based on safety / tolerability considerations) for a year, thereby ensuring continued treatment with KH176 after study KH176-202. A final follow-up visit is scheduled 4 weeks after the intake of the last dose of study medication for patients not rolling over into the compassionate use program. Primary safety data and secondary efficacy (endpoint) data will be monitored and reviewed every three months by an independent Data Safety Monitoring Board (DSMB) to evaluate potential risks and benefits.

Start Date09 Aug 2021

Sponsor / Collaborator

Khondrion BV

[+2]

NCT04846036

/ SuspendedPhase 2

A Randomized Placebo Controlled, Double-blind Phase II Study to Explore the Safety, Efficacy and Pharmacokinetics of Sonlicromanol in Children With Genetically Confirmed Mitochondrial Disease

This a randomized placebo controlled, double-blind phase II study to explore the pharmacokinetics, safety and efficacy of sonlicromanol in children (from birth to 17 years) with genetically confirmed mitochondrial disease of which the gene defect is known to decrease one or more oxidative phosphorylation system enzymes and who suffer from motor symptoms ("KHENERGYC").

Start Date01 Feb 2021

Sponsor / Collaborator

Khondrion BV

[+3]

NCT04165239

/ CompletedPhase 2

A Phase IIb Double-blind, Randomised, Placebo-controlled, Multi-centre, Confirmative Three-way Cross-over Study on Cognitive Function With Two Doses of KH176 in Subjects With a Genetically Confirmed Mitochondrial DNA tRNALeu(UUR) m.3243A>G Mutation.

Mitochondrial diseases, estimated prevalence 1 in 4,300 adults, is caused by pathogenic mutations in genes finally encoding for mitochondrial proteins of the various enzyme complexes of the OXPHOS. Among these mutations, the 3243A>G nucleotide change in the mitochondrially encoded transfer RNALeu(UUR) leucine 1 gene (MT TL 1) is the most prevalent one. The OXPHOS dysfunction resulting from such mutations leads to increased production of reactive oxygen species (ROS), ultimately leading to irreversible oxidative damage of macromolecules, or to more selective and reversible redox modulation of cell signaling that may impact (adult) neurogenesis.
Despite advances in the understanding of mitochondrial disorders, treatment options are extremely limited and, to date, largely supportive. Therefore, there is an urgent need for novel treatments. KH176, a new active pharmaceutical ingredient (API), is an orally bio-available small molecule under development for the treatment of these disorders (see Section 1.4). The current study will further evaluate the effect of KH176 in various cognitive domains and evaluate the effect of different doses of KH176 (See Section 1.5).
In view of the growing recognition of the importance of mitochondrial function in maintaining cognitive processes in the brain, as well as the understanding of the safety profile and pharmacokinetics of KH176 following the two clinical studies described above, a more detailed study is indicated of the effects of KH176 in various cognitive domains, using the confirmed safe and well-tolerated KH176 dose of 100 mg bid, as well as a lower dose of 50 mg bid. The primary objective is an evaluation of KH176 in the attention domain of cognitive functioning, as assessed by the visual identification test score of the Cogstate computerised cognitive testing battery.

Start Date30 Oct 2019

Sponsor / Collaborator

Khondrion BV

[+1]

100 Clinical Results associated with Sonlicromanol Hydrochloride

100 Translational Medicine associated with Sonlicromanol Hydrochloride

100 Patents (Medical) associated with Sonlicromanol Hydrochloride

Literatures (Medical) associated with Sonlicromanol Hydrochloride

06 Mar 2025·BRAIN

Phase 2b program with sonlicromanol in patients with mitochondrial disease due to m.3243A>G mutation

Article

Author: Ruiterkamp, Gerrit ; Burgert, Lydia ; Klopstock, Thomas ; Renkema, Herma ; Bischoff, Almut ; Gorman, Grainne ; Edgar, Chris J ; van Maanen, Rob ; Abbink, Evertine J ; Kümmel, Anne ; van Es, Just ; Pogoryelova, Oksana ; Janssen, Mirian C H ; Vissing, John ; Stendel, Claudia ; Karin, Ivan ; Bosman, Brigitte ; Smeitink, Jan ; Munshi, Mahtab ; Verhaak, Christianne

Abstract:

Mitochondrial disease incorporates a group of rare conditions with no approved treatment to date, except for Leber hereditary optic neuropathy. Therapeutic options to alleviate the symptoms of mitochondrial disease are urgently needed. Sonlicromanol is a promising candidate, as it positively alters the key metabolic and inflammatory pathways associated with mitochondrial disease. Sonlicromanol is a reductive and oxidative distress modulator, selectively inhibiting microsomal prostaglandin E1 synthase activity. This phase 2b program, aimed at evaluating sonlicromanol in adults with m.3243A>G mutation and primary mitochondrial disease, consisted of a randomized controlled (RCT) study (dose-selection) followed by a 52-week open-label extension study (EXT, long-term tolerability, safety and efficacy of sonlicromanol).Patients were randomized (1:1:1) to receive 100 or 50 mg sonlicromanol or placebo twice daily (bid) for 28 days with a ≥2-week wash-out period between treatments. Patients who completed the RCT study entered the EXT study, wherein they received 100 mg sonlicromanol bid.Overall, 27 patients were randomized (24 RCT patients completed all periods). Fifteen patients entered the EXT, and 12 patients were included in the EXT analysis set. All patients reported good tolerability and favourable safety, with pharmacokinetic results comparable to the earlier phase 2a study.The RCT primary end point [change from placebo in the attentional domain of the cognition score (visual identification; Cogstate IDN)] did not reach statistical significance. Using a categorization of the subject's period baseline a treatment effect over placebo was observed if their baseline was more affected (P = 0.0338). Using this approach, there were signals of improvements over placebo in at least one dose in the Beck Depression Inventory (BDI, P = 0.0143), Cognitive Failure Questionnaire (P = 0.0113) and the depression subscale of the Hospital Anxiety and Depression Scale (P = 0.0256).Statistically and/or clinically meaningful improvements were observed in the patient- and clinician-reported outcome measures at the end of the EXT study [Test of Attentional Performance (TAP) with alarm, P = 0.0102; TAP without alarm, P = 0.0047; BDI somatic, P = 0.0261; BDI total, P = 0.0563; SF12 physical component score, P = 0.0008]. Seven of nine domains of RAND-Short Form-36-like SF-36 pain improved (P = 0.0105). Other promising results were observed in the Neuro-Quality of Life Short Form-Fatigue Scale (P = 0.0036), mini-Balance Evaluation Systems test (P = 0.0009), McGill Pain Questionnaire (P = 0.0105), EuroQol EQ-5D-5L-Visual Analog Scale (P = 0.0213) and EQ-5D-5L-Index (P = 0.0173). Most patients showed improvement in the Five Times Sit-To-Stand Test.Sonlicromanol was well-tolerated and demonstrated a favourable benefit/risk ratio for up to 1 year. Sonlicromanol was efficacious in patients when affected at baseline, as seen across a variety of clinically relevant domains. Long-term treatment showed more pronounced changes from baseline.

01 Dec 2022·BMC NeurologyQ4 · MEDICINE

A randomised placebo-controlled, double-blind phase II study to explore the safety, efficacy, and pharmacokinetics of sonlicromanol in children with genetically confirmed mitochondrial disease and motor symptoms (“KHENERGYC”)

Q4 · MEDICINE

ArticleOA

Author: Ruiterkamp, Gerrit ; de Boer, Lonneke ; Renkema, Herma ; van Maanen, Rob ; Smeitink, Jan

Abstract:

Background:

Methods:

The KHENERGYC trial will be a phase II, randomised, double-blinded, placebo-controlled (DBPC), parallel-group study in the paediatric population (birth up to and including 17 years). The study will be recruiting 24 patients suffering from motor symptoms due to genetically confirmed PMD. The trial will be divided into two phases. The first phase of the study will be an adaptive pharmacokinetic (PK) study with four days of treatment, while the second phase will include randomisation of the participants and evaluating the efficacy and safety of sonlicromanol over 6 months.

Discussion:

Effective novel therapies for treating PMDs in children are an unmet need. This study will assess the pharmacokinetics, efficacy, and safety of sonlicromanol in children with genetically confirmed PMDs, suffering from motor symptoms.

Trial registration:

clinicaltrials.gov: NCT04846036, registered April 15, 2021. European Union Clinical Trial Register (EUDRACT number: 2020–003124-16), registered October 20, 2020. CCMO registration: NL75221.091.20, registered on October 7, 2020.

01 Sep 2021·Stem cell reportsQ1 · MEDICINE

Sonlicromanol improves neuronal network dysfunction and transcriptome changes linked to m.3243A>G heteroplasmy in iPSC-derived neurons

Q1 · MEDICINE

ArticleOA

Author: Smeitink, Jan ; Verboven, Anouk H A ; Nadif Kasri, Nael ; Pelgrim, Iris ; Beyrath, Julien ; Hogeweg, Mark ; de Vries, Bert B A ; Hoen, Peter-Bram A C 't ; Klein Gunnewiek, Teun M ; Renkema, Herma ; Schoenmaker, Chantal ; Kozicz, Tamas

Mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes (MELAS) is often caused by an adenine to guanine variant at m.3243 (m.3243A>G) of the MT-TL1 gene. To understand how this pathogenic variant affects the nervous system, we differentiated human induced pluripotent stem cells (iPSCs) into excitatory neurons with normal (low heteroplasmy) and impaired (high heteroplasmy) mitochondrial function from MELAS patients with the m.3243A>G pathogenic variant. We combined micro-electrode array (MEA) measurements with RNA sequencing (MEA-seq) and found reduced expression of genes involved in mitochondrial respiration and presynaptic function, as well as non-cell autonomous processes in co-cultured astrocytes. Finally, we show that the clinical phase II drug sonlicromanol can improve neuronal network activity when treatment is initiated early in development. This was intricately linked with changes in the neuronal transcriptome. Overall, we provide insight in transcriptomic changes in iPSC-derived neurons with high m.3243A>G heteroplasmy, and show the pathology is partially reversible by sonlicromanol.

News (Medical) associated with Sonlicromanol Hydrochloride

18 Aug 2025

·www.khondrion.com

Khondrion and Medvisis Announce Exclusive Licensing Agreement to Commercialize Sonlicromanol for Primary Mitochondrial Diseases in Switzerland and Liechtenstein

NIJMEGEN, the Netherlands & Haegendorf, Switzerland – August 18, 2025 Khondrion B.V. and Medvisis Switzerland AG today announced an exclusive licensing agreement for the registration and commercialization of sonlicromanol in Switzerland and Liechtenstein for the treatment of Primary Mitochondrial Diseases (PMD). Under the agreement, Medvisis receives exclusive rights to commercialize sonlicromanol in its territory. Financial terms were not disclosed. Sonlicromanol, one of the most advanced drug candidates in development for PMD is a disease-modifying, potentially first-in-class, brain-penetrant redox-modulator with antiinflammatory properties, that targets key metabolic and inflammatory pathways underlying PMD. The investigational drug has been evaluated across multiple clinical studies, and a Phase 3 program is expected to enroll its first patient in Q4 2025. “We are very excited about our partnership with Medvisis as it marks an important new phase for Khondrion: preparing for the commercialization of sonlicromanol,” said Prof. Jan Smeitink, CEO of Khondrion. “As Khondrion’s first territorial licensing step, this agreement validates the commercial readiness of sonlicromanol and reinforces the company’s commercialization strategy.” “We are strongly committed to making sonlicromanol available to patients worldwide as soon as possible. This partnership represents an important steppingstone—potentially benefiting approximately 2,000 people living with primary mitochondrial disease in Switzerland and Liechtenstein—as well as a significant milestone for Khondrion.” “Medvisis is dedicated to bringing innovative therapies for rare diseases to patients in Switzerland and Liechtenstein,” said Manuel Ackermann, CEO of Medvisis. “Sonlicromanol addresses a clear unmet medical need in PMD. We look forward to collaborating closely with Khondrion to advance registration, secure market access and reimbursement, and enable rapid access for patients who are suffering from this debilitating disease.” Sonlicromanol: one of the most advanced drug candidates in development for PMD Sonlicromanol is an investigational, oral small-molecule redox modulator designed to restore mitochondrial redox homeostasis, normalize aberrant reactive species and prevent consequent cell damage and death. Sonlicromanol has been investigated in four clinical trials to date. Adult patients participating in Khondrion’s Phase 2 program have reported continued and progressive improvements on key symptoms (see Smeitink et al., Brain, 2025). In addition, patients who elected to participate in a named-patient program following completion of the Phase 2b study have been receiving sonlicromanol for more than three years, supporting the favorable safety profile observed to date. Beyond PMD, a proof-ofconcept clinical trial in post-COVID is expected to enroll its first patient in Q4 2025. About primary mitochondrial disease Primary mitochondrial diseases are devastating, progressive, multi-system disorders that affect approximately 1 in 5,000 people. These rare diseases arise from pathogenic variants in either nuclear DNA or mitochondrial DNA that disturb the mitochondrial oxidative phosphorylation (OXPHOS) system—the final biochemical pathway for cellular ATP production. OXPHOS dysfunction impairs ATP generation and triggers downstream cellular stress, disproportionately affecting energy-demanding tissues. Patients frequently suffer from multi-organ involvement, affecting organs and tissues with a high-energy demand like the brain, the eyes, the heart and skeletal muscle leading to cognitive decline, epilepsy, visual impairment, cardiomyopathy (a disease that changes the heart muscle, sometimes causing it to enlarge or stiffen, which can reduce its ability to pump blood), fatigue, exercise intolerance and muscle pain. There are currently no approved disease-modifying therapies for PMD. About Khondrion Khondrion is a Phase 3 clinical-stage Dutch biopharmaceutical company based in Nijmegen, pioneering disease-modifying therapies for primary mitochondrial diseases caused by defects in mitochondrial oxidative phosphorylation (OXPHOS). The company’s lead asset, sonlicromanol, is in late-stage development with a Phase 3 program in preparation. Sonlicromanol has received orphan drug designations for all inherited mitochondrial oxidative phosphorylation defects in Europe, and for all inherited mitochondrial respiratory chain disorders in the U.S., as well as U.S. rare pediatric disease designation for MELAS. For more information, visit www.khondrion.com. About Medvisis Switzerland AG Medvisis is a Swiss-based pharmaceutical company dedicated to bring transformative rare disease and specialty therapies with high unmet medical need to Swiss patients. A relatively young company, Medvisis unites senior commercial and medical executives from international pharmaceutical companies and Swiss university hospitals, securing unmatched access to national key opinion leaders and other critical healthcare system stakeholers. Medvisis is expanding its portfolio through in-licensing of high-potential therapies driving product registration, reimbursement and commercialization. To learn more, visit www.medvisis.com. Forward-looking statements This press release may contain certain forward-looking statements regarding, among other things, the results, conduct, progress and timing of the company’s clinical trials and presentation of data from clinical trials for sonlicromanol. Although the company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this press release about future events are subject to (i) change without notice and (ii) factors beyond the company’s control. These statements may include, without limitation, any statements preceded by, followed by or including words such as “target,” “believe,” “expect,” “aim,” “goal,” “intend,” “may,” “anticipate,” “foreseen,” “estimate,” “plan,” “project,” “will,” “can have,” “likely,” “potential,” “should,” “would,” “could” and other words and terms of similar meaning or the negative thereof. Forwardlooking statements are subject to inherent risks and uncertainties beyond the company’s control that could cause the company’s actual results, performance or achievements to be materially different from the expected results, performance or achievements expressed or implied by such forward-looking statements. Except as required by law, the company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. Contacts E-mail: info@khondrion.com Tel: +31 24 763 50 00 Website: www.khondrion.com E-mail: info@medvisis.com Tel: +41 79 422 14 92 Website: www.medvisis.com

Phase 2License out/inPhase 3Orphan DrugClinical Result

06 May 2025

·www.prnewswire.com

Khondrion Secures up to €5 Million Innovation Credit from Dutch Government plus Additional Funding from Current Investors to Advance Phase 3 Clinical Trial in m.3243A>G Primary Mitochondrial Disease

Government support, alongside private investments, funds first wave of pivotal Phase 3 trial of sonlicromanol for m.3243A>G Primary Mitochondrial Disease (PMD) NIJMEGEN, Netherlands, May 6, 2025 /PRNewswire/ -- Khondrion, a clinical-stage biopharmaceutical company pioneering therapies for primary mitochondrial disease (PMD), today announced it has been awarded an Innovation Credit of up to €5 million from the Netherlands Enterprise Agency, a government agency that supports entrepreneurs. This vital government funding, matched by a significant investment from Khondrion's committed private backers, will fund Wave 1 of the company's pivotal Phase 3 clinical trial of sonlicromanol. Sonlicromanol is a first-in-class small-molecule candidate therapy for m.3243A>G PMD, the most common genetic form of mitochondrial disease. The Phase 3 trial is expected to launch in H2-2025, marking a crucial milestone in Khondrion's mission to deliver the first approved treatment for these devastating, multisystem disorders. A Major Step Forward for the Mitochondrial Disease Community Primary mitochondrial diseases are a group of rare, progressive, and life-altering disorders that disrupt the body's ability to generate energy. This leads to a wide range of severe symptoms across multiple organ systems, including the brain, muscles, and digestive tract. For the over 250.000 of people living with PMD worldwide, there are currently no approved therapies that directly address the causes underlying the disease. "Receiving this support from the Dutch government, together with the continued commitment of our trusted investors, represents a powerful endorsement of Khondrion's work and the urgent need to bring innovative treatments to the PMD community," said Jan Smeitink, CEO of Khondrion. "With sonlicromanol, we are advancing a potentially transformative therapy designed not only to alleviate symptoms but also to modify the course of this devastating disease, offering new hope to patients and families who have long been waiting for effective solutions." With the Phase 3 trial on the horizon, Khondrion is taking a bold step towards changing the lives of patients suffering from mitochondrial disease — and creating meaningful opportunities for partners ready to join this journey. Sonlicromanol: one of the most advanced drug candidates in development for PMD Sonlicromanol has been investigated in four clinical trials. Adult patients participating in our extensive Phase 2 programme have reported continued and progressive improvements on key symptoms (see Smeitink et al., Brain 2025; ). Those patients that elected to participate in a named patient program following completion of the Phase 2b programme, have been receiving sonlicromanol for more than two and a half years now, thus confirming the drug's favorable safety profile. Notes to editors For press release with boilerplate click on this link: . Logo: SOURCE Khondrion WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 2Phase 3

21 Nov 2024

·www.globenewswire.com

UMDF and Khondrion Announce The Mito Fund’s Investment in Khondrion to Support Development of One of the Most Advanced Drug Candidates for Primary Mitochondrial Disease

21 November 2024 – The United Mitochondrial Disease Foundation’s (UMDF) venture philanthropy arm, The Mito Fund, has invested 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.” Like all investments made by The Mito Fund, the Khondrion consideration went through a rigorous review process by UMDF’s Venture Philanthropy Investment Committee, an advisory group consisting of world-class experts in the areas of mitochondrial disease, pharmaceutical development, and private equity investments. After receiving the committee’s recommendation, the investment was then approved by UMDF’s full Board of Trustees. “From an investment perspective, there were several really attractive elements to the investment. First, sonlicromanol represents an innovative treatment for an underserved population. It’s also a mature clinical stage asset, which shortens the runway to approval,” said Dr. Yeske. “UMDF looks forward to partnering with Khondrion in advancing this investigational therapeutic in a patient-centric manner.” Launched in the summer of 2023, The Mito Fund is UMDF’s most ambitious effort to date to ramp up the development of mitochondrial disease treatments and cures. The fund’s venture philanthropy model is designed not only to financially support the development of the next generation of mitochondrial disease treatments, but also to leverage UMDF’s experience and network to accelerate the development process and lower the risk associated with entry into the field for a wide range of investors and researchers. 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). Companies exploring the mitochondrial science space are encouraged to start a conversation about investment opportunities at www.themitofund.org. Proposals are accepted on a rolling basis. About UMDF For more than 25 years, The United Mitochondrial Disease Foundation (UMDF) has built a global network of patients, researchers, clinicians, institutions, and industry partners dedicated to fighting mitochondrial disease. Together with the mito community, UMDF is committed to a mission of funding the best science no matter where it is found in the world while at the same time providing critical education, advocacy, and support-focused programs and services to patient families. In the last year … Nearly 1,500 affected patients and families depended upon UMDF for support.More than 400 clinicians listened to UMDF’s Bench-to-Bedside seminars.UMDF advocates had more than 1,000 touchpoints with their congressional offices.UMDF provided hundreds of patients with no-cost genetic testing to help shorten the diagnostic journey. About Khondrion Khondrion is a clinical stage biopharmaceutical company developing therapies for patients with primary mitochondrial disease (PMD). The company’s lead asset, sonlicromanol, is a potentially first-in-class, brain-penetrant redox-modulator with anti-inflammatory properties, that targets key metabolic and inflammatory pathways underlying PMD. One of the most advanced, potentially disease-modifying drug candidates for mitochondrial disease in development, sonlicromanol has been tested in three clinical trials in patients with m.3243A>G PMD, as well as in the first wave of a 6-month Phase 2 study in children with genetically confirmed PMD and who suffer from motor symptoms. Sonlicromanol has been granted orphan drug designations for the treatment of MELAS syndrome (mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes), Leigh disease and patients with maternally inherited diabetes and deafness (MIDD) in Europe, and for all inherited mitochondrial respiratory chain disorders in the US. It has also been granted a rare pediatric disease designation in the US for the treatment of MELAS. Sonlicromanol and other compounds from Khondrion’s proprietary library have the potential to be developed for a wide range of diseases and conditions with the aim of benefiting patients whose daily lives are severely impacted by mitochondrial impairment. For more information visit www.khondrion.com. Contacts: Khondrion BVProf. Dr. Jan Smeitink, CEOE-mail: info@khondrion.comTel: +31-24-7635000www.khondrion.com ICR Healthcare David Daley, Kris Lam, Tom DanielEmail: khondrion@icrhealthcare.com UMDF / The Mito FundAndy Dearth, Director of Marketing & CommunicationsEmail: Andy.dearth@umdf.org

Phase 2Orphan DrugPhase 3

100 Deals associated with Sonlicromanol Hydrochloride

External Link

KEGG	Wiki	ATC	Drug Bank
-	-	-	DB16333

R&D Status

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Noninsulin-Dependent Diabetes Mellitus With Deafness	Phase 2	Denmark	Khondrion BV	09 Aug 2021
Noninsulin-Dependent Diabetes Mellitus With Deafness	Phase 2	Germany	Khondrion BV	09 Aug 2021
Noninsulin-Dependent Diabetes Mellitus With Deafness	Phase 2	Netherlands	Khondrion BV	09 Aug 2021
Noninsulin-Dependent Diabetes Mellitus With Deafness	Phase 2	United Kingdom	Khondrion BV	09 Aug 2021
Ophthalmoplegia, Chronic Progressive External	Phase 2	Denmark	Khondrion BV	09 Aug 2021
Ophthalmoplegia, Chronic Progressive External	Phase 2	Germany	Khondrion BV	09 Aug 2021
Ophthalmoplegia, Chronic Progressive External	Phase 2	Netherlands	Khondrion BV	09 Aug 2021
Ophthalmoplegia, Chronic Progressive External	Phase 2	United Kingdom	Khondrion BV	09 Aug 2021
Leigh Disease	Phase 2	Netherlands	Khondrion BV	01 Feb 2021
MELAS Syndrome	Phase 2	Netherlands	Khondrion BV	01 Sep 2016

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04165239 (KHENERGYZE, GlobeNewswire) Manual	Phase 2	MELAS Syndrome	27	Sonlicromanol	tydkedithk(cyljgtwtyf) = orjacduizs wyrkyotoam (pjauvyweqm )	Negative	22 Nov 2022
				Placebo	-

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

AI Agents Built for Biopharma Breakthroughs

Accelerate discovery. Empower decisions. Transform outcomes.

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis