Muscular dystrophies and muscle-wasting disorders

Muscular dystrophy (“Dystrophies”) includes over 160 unique diseases that display progressive muscle degeneration, which often lead to weakness, loss of ambulation, and even loss of life. These diseases affect tens of thousands of people annually and are generally poorly served by current therapeutic approaches – none of which directly treat the regenerative deficits targeted by Satellos. Dystrophies arise as a result of mutations in genes that code for important muscle proteins, leading to the alteration or complete loss of their contribution toward ensuring a healthy, functional muscle tissue. Intriguingly, muscle wasting syndromes such as cachexia, although not genetic in nature can display a similar mechanism of action, resulting in muscle degeneration and loss of muscle function. Cachexia generally refers to muscle wasting that results alongside an underlying medical condition such as cancer or COPD . Despite considerable differences in their etiology, muscular dystrophy and wasting syndromes represent areas of extremely high unmet need for therapies that restore muscle loss and function.

Duchenne muscular dystrophy (“Duchenne”) is the most common childhood onset muscular dystrophy. It is caused by mutations in the gene that codes for dystrophin, a protein that contributes to the function of muscle cells. Duchenne affects approximately 1 in 3500 male births world-wide, irrespective of geography or socio-economic conditions, causing muscle stem cell dysfunction, progressive degeneration of muscle, functional decline, and ultimately, loss of life.

Satellos is developing first-in-class regenerative therapeutics that seek to reset proper muscle stem cell function, restore productive muscle regeneration, improve muscle strength and functionality, and ultimately, enhance the quality and duration of the lives of Duchenne patients. Satellos scientists were the first to discover that the loss of dystrophin in muscle stem cells underlies the classically observed, but previously not understood, impaired regeneration response in Duchenne; and also, we were first to discover an alternative signaling pathway that can be therapeutically targeted to restore regeneration – independent from and in the absence of the dystrophin protein. In both in vitro and in vivo preclinical studies, Satellos has shown that targeting of this pathway results in significantly improved muscle quantity, quality and function. We intend to expand into additional muscular dystrophies and wasting syndromes where we believe a similar mechanism may be at play and impaired regeneration has been observed.

Aging and trauma-induced muscle disorders

As humans age, we experience a loss in skeletal muscle mass and strength, a phenomena known as sarcopenia. Individuals that suffer from sarcopenia are at higher risk for slips, trips, and falls, reduced independent mobility, and mortality. Although the precise underlying cause of sarcopenia remains to be fully understood, Satellos scientists have identified a clear deficit in the muscle regeneration process during aging. Not only may these deficits contribute to the onset of sarcopenia, but likely hinder recovery in individuals who’ve undergone mobility related operations following injury or joint replacements. Onset of sarcopenia and impaired injury recovery are both cofactors that contribute to the potential for overall frailty in the elderly population and represent areas of high unmet need.

Though the use of MyoReGenX™, Satellos scientists have discovered a means to restore the regenerative deficit observed in aging. Satellos has identified multiple candidate pathways that can be targeted to improve muscle quality, quantity, and function in aged muscle tissue. Our plan is to develop regenerative therapeutics for the potential prevention or treatment of sarcopenia associated skeletal muscle loss, as well a locally delivered therapeutic designed to enhance post-surgical skeletal muscle recovery in the elderly.

Licensing and co-development opportunities

Oral-Trans™ Drug Formulation Technology

AmpB Technologies Inc. (“AMP-B”), a subsidiary of Satellos, holds exclusive commercialization rights to multiple granted patents and patent applications describing a proprietary drug formulation technology for delivering non-water soluble compounds orally, in a safe and effective manner (Oral-Trans™). AmpB is deploying Oral-Trans™ to clinically develop an oral formulation of amphotericin B (“Amp-009” formerly known as “iCo-019”) for treating a range of severe fungal infections in a less toxic manner. AMP-B is seeking licensing or co-development partners for Amp-009 or other formulation and clinical development applications of Oral-Trans™. To learn more about Amp-009 or Oral-Trans™ or explore partnering opportunities, please contact us for more information.

Bertilimumab (previously iCo-008)

Bertilimumab is a human monoclonal eotaxin antibody with potential to treat a range of allergic and inflammatory conditions mediated by eotaxin-1. Satellos, through the reverse takeover of iCo Therapeutics, holds development rights for ocular indications of Bertilimumab. Rights to the development of Bertilimumab in non-ocular indications have been out-licensed to and are held by Alexion (since acquired by Astra Zeneca). To learn more about Bertilimumab please click here to view or download the presentation or contact us.