RARE Daily

Epirium Raises $85 Million to Develop Treatments for Rare Neuromuscular Diseases

December 18, 2019

Epirium Bio has launched with $85 million in series A financing to advance a scientific platform focused on the restoration of tissue bioenergetics and function with an initial focus targeting rare progressive neuromuscular disorders associated with mitochondrial depletion.

Though the company’s initial focus is on Becker muscular dystrophy, it believes its technology may have application to slowing the process of aging.

Longitude Capital, ARCH Venture Partners, Bluebird Ventures, Adams Street Partners, Vertex Ventures, and The Longevity Fund provided the funding. Proceed from the financing will be used to further develop the company’s platform and advance its first clinical candidate in 2020.

Becker muscular dystrophy is an inherited condition that causes progressive weakness and wasting of the skeletal and cardiac muscles and primarily affects males, followed by drug development targeting other progressive neuromuscular disorders associated with mitochondrial depletion.

From orphan neuromuscular disorders to cancer, numerous diseases are characterized by either a failure to meet the bioenergetic requirements of tissues or an alteration in bioenergetic pathways caused by mitochondrial depletion or dysfunction. Despite their importance, the understanding of these fundamental mechanisms needed to keep cells alive and properly functioning has lagged, resulting in a dearth of pharmacologically tractable targets. Recently efforts have intensified to both dissect the role played by mitochondrial signaling pathways in the pathobiology of diseases and develop relevant mitochondrial-based therapeutics.

Epirium’s scientists have discovered a previously unknown hormonal pathway that appears to be an endogenous modulator of mitochondrial biogenesis – the formation of new mitochondria. Among the distinctive properties of this hormonal pathway is the generation of new, functional mitochondria, coupled with restoring the structure and function of previously atrophic tissue, particularly that of neuromuscular and central nervous systems. The coupling of these pathways mimics a physiological process that can be seen in response to physical exercise and that is essential for the proper functioning of organs. Modulating this response is expected to have meaningful clinical benefit.

Using this knowledge, Epirium is pursuing a novel and differentiated pharmacological approach for the treatment of diseases characterized by mitochondrial depletion or dysfunction leading to subsequent organ failure.

Epirium and its collaborators have carried out several proof-of-concept clinical studies in orphan diseases such as Becker and Duchenne muscular dystrophy and Friedreich’s ataxia. These studies have provided insights into the unique mechanism of action and the potential for clinical benefit while revealing a strong safety profile. Additionally, the data are very supportive of further developing the main clinical candidate in diseases of muscular dystrophy. Epirium has yet to publish any data on its findings but expects to do so in early 2020.

“Epirium has discovered a novel class of therapeutics addressing not only the loss of muscle structure, but also the underlying mitochondrial dysfunction, which represents another major progression factor in diseases of muscle failure,” said Craig McDonald, professor at the University of California at Davis and principal investigator of Epirium’s first company-sponsored study.

In addition to orphan neuromuscular diseases, the technology is also relevant to diseases of aging, which are believed to share a common mechanism of progressive mitochondrial loss.

The management team includes President and CEO Russell Cox, Chief Scientific Officer George Schreiner, and Chief Technology Officer Sundeep Dugar, all of whom have decades of experience in the biopharmaceutical industry.

Photo: Russell Cox, president and CEO of Epirium

Author: Rare Daily Staff

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