MetrioPharm Starts Preclinical Experiments in Duchenne Muscular Dystrophy (DMD)
- MetrioPharm is testing the efficacy of its lead compound MP1032 in DMD model
- The company receives a grant of €125.000 from Duchenne UK
- DMD is the most common and severe form of muscular dystrophy; it is a designated orphan disease in Europe and the USA
Zurich, October 12, 2022. MetrioPharm AG, a pharmaceutical company developing drugs for inflammatory diseases, announced today that is has received a grant of €125.000 provided by UK's leading Duchenne muscular dystrophy charity, Duchenne UK. MetrioPharm is currently conducting preclinical experiments in order to evaluate the efficacy of MetrioPharm's lead compound MP1032 for the treatment of DMD.
DMD is a genetic disorder characterized by progressive muscle degeneration and weakness due to the alterations of a protein that is essential to maintaining integrity of muscle cells. It is the most severe of several conditions known as muscular dystrophies. DMD symptom onset is in early childhood, usually between ages 2 and 3. The disease primarily affects boys with severe muscle loss and heart failure, while girls can show milder symptoms as well. DMD shortens life expectancy significantly, current therapies can only slow disease progression.
DMD is classified as an orphan (rare) disease of high unmet medical need. New treatments for orphan diseases receive priority support during development and market authorization from the EMA and the US FDA.
MP1032 is currently tested in the well-established mdx mouse model for DMD. In this in vivo model the effects of MP1032 on early muscle inflammation and destruction are investigated and compared to the effects of two steroids. The results will provide MetrioPharm information about a potential clinical development of MP1032 in steroid-treated DMD patients.
MetrioPharm's Chief Scientific Officer (CSO) Dr. Wolfgang Brysch comments, I became aware of DMD through our work with our compound MP1032. An experienced researcher and physician in this field pointed out to us that our drug could influence the course of this devastating childhood disease. We are currently testing, if MP1032 can alleviate the loss of muscles, caused by the genetic defect that leads to inflammation and degeneration in the child's muscles. Currently, children suffering from DMD are undergoing lifelong treatment with highly dosed steroids that lead to strong side-effects. We are hoping to be able to develop a combined treatment consisting of our lead compound MP1032 and steroids that would reduce such side-effects and slow the muscle loss."
Dr Alessandra Gaeta, Director of Research at Duchenne UK: "Duchenne UK is driven to find new treatments for DMD, and in as short a time frame as possible. There is a desperate unmet need for effective DMD treatments and we are pleased to be partnering with Metriopharm to speed up this search by investigating a drug which is already shown to be safe and effective in other disease areas."
Alasdair Robertson, who supports Duchenne UK through the Family and Friends Fund, For Felix, explains, "My son Felix was diagnosed with Duchenne in December 2014. Since then, I have thrown myself into the race to find innovative, disease-mitigating treatments. I met Dr. Wolfgang Brysch and the MetrioPharm team during the course of this search and am very optimistic that their lead compound MP1032 could be part of a treatment that reduces the suffering of those children affected by DMD."
About Duchenne UK
As the leading charity for Duchenne muscular dystrophy in the UK, the organization funds groundbreaking scientific research, for the development of new therapies and technologies to treat this often still quite unfamiliar disease. Duchenne UK's stated goals are not only to improve the quality of life of patients, but to stop the disease altogether. The organization brings together leading researchers with the pharmaceutical industry, the NHS (National Health Service UK) and patients. The aim is to accelerate therapy development and provide treatments to those who so desperately need them.
About MetrioPharm AG
MetrioPharm AG is a private, clinical-stage biotech company focused on therapies for inflammatory and infectious diseases.
MetrioPharm targets the modulation of immune metabolism with first-in-class self-regulating drug candidates. Our platform has generated small-molecule metabolic modulators that have demonstrated pre-clinical and clinical efficacy in a wide range of inflammatory and infective diseases with an outstanding safety profile. Further opportunities of the technology lie in metabolic modulation of the tumor micro-environment which holds promise for enhancing the efficacy of immune-oncological therapies.
Based on this core technology, MetrioPharm has created a diversified pipeline of disease-specific drug combinations to target inflammatory, autoimmune and degenerative diseases. Pre-clinical and clinical efficacy data have been obtained in Multiple Sclerosis, Arthritis, Sepsis, Inflammatory Bowel Disease and Psoriasis. The company is currently also exploring the orphan indication Duchenne Muscular Dystrophy.
In a second line of development, the company is developing its technology to target infectious diseases like COVID-19. Supported by a grant from the European Health Emergency Preparedness and Response Authority (HERA), a phase II exploratory trial to evaluate the effect on hospitalized COVID-19 patients is currently ongoing (top line data in Q4 2022).
The company is headquartered in Zurich and has a subsidiary for R&D activities in Berlin.
This press release contains forward-looking statements that involve risks and uncertainties and are consistent with MetrioPharm AG's assessment as of the date of this release. Such forward-looking statements are neither promises nor guarantees but are subject to numerous risks and uncertainties. No liability or warranty, and no claim, if any, is made with respect to the timeliness, accuracy or completeness of such data and information, and no reliance should be placed on such data and information, either explicitly or impliedly.
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