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New food for special medical purposes to nutritionally manage Myotonic Dystrophy type 1

Periodic Reporting for period 1 - MYODM-FSMP (New food for special medical purposes to nutritionally manage Myotonic Dystrophy type 1)

Reporting period: 2019-10-01 to 2021-06-30

Myogem Health Company S.L. (www.myogemhealth.com) is a B2C life sciences company focused in R&D of nutritional products to manage neuromuscular rare and prevalent diseases. Through the project "New food for special medical purposes for the nutritional management of Myotonic Dystrophy type 1 (DM1)", funded by EU under SME Instrument Phase 1 in H2020 grant agreement No 87615, MYOGEM aimed to upgrade its product MYODM “food supplement” (a unique combination of caffeine and theobromine that restored atrophy, increased muscle area, recovered heart functions; increased mean survival and life expectancy in in vivo DM1 animal models) to MYODM-FSMP “food for special medical purposes”. FSMP are foods intended for the dietary management of individuals who suffer from certain diseases, and involve the supervision of the medical community. For certain rare diseases, this approach may provide a unique affordable option, since they are niche markets. The feasibility studies have included technical, economic and regulatory assessment for properly placing MYODM-FSMP product in the market.

DM1, or Steinert's disease, is a complex neuromuscular disease of genetic origin, without any cure and highly limiting of the quality of life. DM1 is a multisystemic autosomal dominant disorder, clinically characterized by progressive muscle atrophy and weakness, cardiomyopathy, insulin resistance and cataracts, among others. Overall, DM1 patients have a very limited quality of life (QoL), wherein QoL is a multidimensional concept that encompasses physical, psychological and social factors, whose impact on each patient depends on individual and social conditions

The main hypothesis guiding the present project is that the controlled and dosaged administration of the formulated composition containing caffeine and theobromine marketed as MYODM could be useful for the nutritional management of DM1 patients, potentially contributing to the reduction of daytime sleepiness and to the improvement of Health-Related Quality of Life of these patients.

The main goal of the present project was to evaluate the effect of MYODM on DM1’s patients quality of life and excessive daytime sleepiness through the pilot interventional clinical trial “Research project to evaluate the effect of the MYODM food supplement on quality of life, fatigue and hypersomnia in patients with Myotonic Dystrophy type 1” (clinicaltrials.gov nr NCT04634682), sponsored by MYOGEM, run at the Neurology Service of the Donostia University Hospital, led by Dr. Roberto Fernández Torrón and assisted by the CRO MIAKER Developments, S.L. The pilot clinical trial was conceived as a monocentric six month randomized pilot interventional clinical trial to test these hypothesis.
The present project took place in Spain during the COVID-19 pandemics. The study population was made up of 30 patients, both men and women, aged from 28 to 72 years old (mean age, 46.33±9.93) and diagnosed with DM1, who attended the outpatient demographic consultation of the Neuromuscular Unit of the Donostia University Hospital and who met the inclusion and exclusion criteria of this research project. Patients were selected in order to be representative of the DM1 patient community, by including subjects with low, moderate and high affectation, either considering their number of CTG repetitions or their clinical evaluation and muscular impairment rating scale (MIRS). The patients were randomly assigned to one of the two study arms: the active arm receiving MYODM treatment, and the control arm as a parallel group to follow the same evaluation program. Primary outcome measures were based on changes in QoL and ESS questionnaires’ scores from baseline visit to the follow-up visit. Female represented 37% whereas male represented 63%. Mean CTG repeats in both arms was 601.79±73.46 whereas muscular impairment rating scale (MIRS) was 3.03±1.10 corresponding to mild to moderate proximal weakness. All patients in both arms had a very complex history of comorbidities and comedications. It is worth mentioning that the treatment was well tolerated by the great majority of the patients.

Results on excessive daytime sleepiness suggest that the controlled and dosed administration of caffeine and theobromine in the formulated product MYODM could provide significant improvements in excessive daytime sleepiness, at least in adult male muscular impaired DM1 patients. Excessive daytime sleepiness is scored as one of the main aspects limiting the quality of life in DM1, due to its high impact in safety, sociability and employability of the patients suffering the disease.
Although the present pilot study encompassed a limited dataset, the obtained results in the Epworth sleepiness scale primary outcome encourage further clinical trials in larger cohorts, ideally to be performed beyond the era of COVID-19 pandemics. These results support the product’s upgrade to FSMP category and encourage future applications for the product's composition.
MYOGEM has opened a founding round for its expansion and internationalization.
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