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In this article we report a summary of the results of the Phase 2b clinical development program of Sonlicromanolthe therapy developed by the biopharmaceutical company Khondrion. The data, published in the journal Braindemonstrate the therapeutic potential of this drug for mitochondrial mutation m.3243A>Goften associated with MELAS (mitochondrial encephalomyopathy with lactic acidosis and stroke-like episodes) and a broad spectrum of phenotypes, including MIDD (maternally inherited diabetes and deafness), i mixed phenotypes (MP) and the CPEO (chronic progressive external ophthalmoplegia).
The results of the study
The study demonstrated that Sonlicromanol can have beneficial effects on cognitive functions, mood and motor control, contributing to a general improvement in the patient’s quality of life; it was well tolerated in clinical trials with a good safety profile up to the expected therapeutic doses. Sonlicromanol was also tested in the first cohort of a 6-month Phase 2 study in children with genetically confirmed PMD and motor symptoms.
It is currently being evaluated in several phase II studies, aimed at confirming its safety and effectiveness on specific symptoms of mitochondrial diseases, such as motor problems and depressive symptoms, with potential application also in broader inflammatory and neurological conditions.
Sonlicromanol is a redox modulator for the treatment of primary mitochondrial diseases, particularly those related to the m.3243A>G mutation, such as MELAS. It acts on oxidative stressa key factor in mitochondrial dysfunction, helping to protect cells from damage and stabilize energy production; it also has anti-inflammatory properties thanks to the inhibition of mPGES-1 (prostaglandin E synthetase), an enzyme involved in the inflammatory response.
The clinical study
The recent publication on Brain concerns the results of the phase 2b clinical study involving 27 adult patients with m.3243A>G in a 28-day, randomized, placebo-controlled study, accompanied by a 52-week open-label extension study. Selected indicators based on the most severe symptoms for patients, such as muscle weakness, chronic fatigue, pain and cognitive decline, were examined. In line with previous clinical studies, Sonlicromanol showed an excellent pharmacokinetic profile and a positive safety profile, being well tolerated for up to 52 weeks.
The experience gained from the Phase 2b program, together with data from two previous safety studies, provided valuable information to optimize the design – including the selection of primary and secondary endpoints – of the 52-week Phase 3 study, who will further examine the potential of Sonlicromanol in adult patients with the m.3243A>G mutation.
Orphan drug designation
Sonlicromanol has obtained the designation of orphan drug for the treatment of MELAS syndrome (mitochondrial encephalopathy, lactic acidosis and stroke-like episodes), Leigh disease and patients with diabetes and maternal hereditary deafness (MIDD) in Europe, and for all hereditary disorders of the mitochondrial respiratory chain in the United States United. It has received authorization from the FDA for the IND (Investigational New Drug) application which allows it to initiate the pivotal Phase 3 study.
The data from the phase 2b clinical study were presented by Jan Smeitink, CEO Khondrion, on the occasion of the 14th edition of the National Conference on Mitochondrial Diseases which was held in Padua from 25 to 27 October.
Statements
This content is taken from the press release issued by Khondrion Company and may contain certain forward-looking statements regarding, among other things, the results, conduct, progress and timing of the Company’s clinical trials and the presentation of clinical data for Sonlicromanol . Although the Company believes that its expectations are based on reasonable assumptions, all statements in this press release and in this news story regarding future events, other than statements of historical fact, are subject to (i) change without notice and ( ii) factors outside the company’s control.
Next phase of the clinical trials, aiming to further explore its efficacy in larger patient populations.
Great! Let’s get started. Can you introduce yourself and your organization briefly?
Guest 1: My name is Jane Smith, and I am the CEO of World Today News. Our website aims to provide the latest information on global events, breaking news, and advances in various industries.
Guest 2: My name is Dr. John Doe, and I represent Khondrion, a leading biopharmaceutical company specializing in the development of innovative therapies for rare diseases. Our primary focus is on developing treatments for mitochondrial disorders, a group of rare genetic diseases that affect energy production in the cells.
Can you explain Sonlicromanol and its potential for treating mitochondrial diseases like MELAS, MIDD, and CPEO?
Guest 2: Sure, Sonlicromanol is a highly promising drug that has shown potential in treating primary mitochondrial diseases like MELAS, MIDD, and CPEO. It’s a redox modulator with anti-inflammatory properties that helps stabilize energy production by targeting oxidative stress, a key factor in mitochondrial dysfunction. This drug acts on mutations like m.3243A>G, which are often associated with these conditions, and has the potential to improve symptoms like muscle weakness, fatigue, pain, and cognitive decline.
Guest 1: In your recent article, you discussed the results of a Phase 2b clinical trial involving adult patients with m.3243A>G mutation. Can you provide more details about the study design and its findings?
Guest 2: Absolutely. The study involved 27 adult patients with the m.3243A>G mutation and was randomized, placebo-controlled for 28 days and then open-label for an additional 52 weeks. We measured symptoms such as muscle weakness, fatigue, cognitive decline, and several others. The results showed that Sonlicromanol had excellent pharmacokinetic properties and was generally well-tolerated by patients for up to 52 weeks. Based on these findings, we’ve optimized our design for the
