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Mitochondrial-Based Therapeutics to Witness Significant Growth due to Positive Clinical Results in the Coming Years

According to a new research report Mitochondrial-Based Therapeutics - Pipeline Analysis 2018, Clinical Trials & Results, Patents, Designations, Collaborations, and Other Developments” published by Pharma Proff, mitochondrial-based therapeutics currently exhibits a strong pipeline with 16 therapeutic candidates.

Insights into Mitochondrial-Based Therapeutics Pipeline

Mitochondria are the organelles found in cytoplasm of most eukaryotic cells. These are also known as power-house of the cell. Although, the gene expression of mitochondria has been poorly understood; however, it is very important to understand the same in the diseased patients. Several chronic diseases, such as Leigh’s disease, and Alper’s syndrome, occur due to the mutation in mitochondrial DNA (mtDNA), which alters mtDNA expression. Currently, there are no approved drugs for the treatment of diseases caused due to mtDNA mutation.

Insights into Pipeline Segments

According to the research findings, majority of the drug candidates in the pipeline are being developed as a small molecule and synthetic peptides, to counteract the effect of mitochondrial-based diseases in body. These drug candidates have shown a positive-results in their Pre-Clinical and clinical studies.

Positive Clinical Trial Results are Expected to Drive the Mitochondrial-Based Therapeutics Pipeline

The companies who are developing mitochondrial-based therapeutics for the treatment of various chronic diseases have shown positive clinical results in the various phases of drug development. For instance, in November 2017, Khondrion BV announced results of its KHENERGY study, a Phase II exploratory trial with oral KH176 in the m.3243A>G multisystem mitochondrial MELAS (mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes) and maternally inherited diabetes and deafness (MIDD) syndromes and mixed phenotypes. With regard to clinical outcomes, significant improvement was observed in the total Beck Depression Inventory score (psychological test). Positive trends were observed in the HADS depression (psychological test) subsection and the RAND-36 SF affective symptoms. Self-reported outcomes revealed an improvement in migraine in all affected subjects.

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Various Designation Grants Play a Pivotal Role in the Mitochondrial-Based Therapeutics Pipeline Growth

It has been observed that many mitochondrial-based therapeutics are being granted various designation from the United States Food and Drug Administration (USFDA), and the European Commission. These milestones are helpful in accelerating the development of therapeutic molecular entity. For instance, in April 2018, Stealth BioTherapeutics Inc. announced that the USFDA granted Orphan Drug Designation to company’s frontrunner compound “Elamipretide”, to treat Barth syndrome and Leber’s hereditary optic neuropathy (LHON).

Some of the key players involved in the development of mitochondrial-based therapeutics include Khondrion BV, CohBar Inc., BioElectron Technology Corporation, Chondrial Therapeutics Inc., Immungenetics AG, Stealth BioTherapeutics Inc., Congenia S.r.l., Astellas Pharma Inc., and Novartis AG.

Mitochondrial-Based Therapeutics Pipeline Analysis

  • By Phase
  • By Molecule Type
  • By Route of Administration
  • By Company

The report comprises of detailed pipeline analysis of mitochondrial-based therapeutics, analyzing the emerging therapies and their progress status in different phases of development. Comprehensive insights into the pipeline phase products has been provided with special focus on strategic development activities inclusive of collaboration and licensing information, drug designations, financing, grants, technological advancements, and patent. The report also contains competitive analysis and extensive information on monotherapies, combination therapies, targets and mechanism of action, and drug origin with relevance to mitochondrial-based therapeutics.