Mitotech and its dry eye disease drug will be approaching their pivotal Phase 3 study with a certain amount of optimism: the dry eye therapies market is growing and is hugely underserved, they have a promising novel MoA on their side and efficacy signal across previous studies provides support for the upcoming Phase 3 trial.

Approximately 16 million Americans have been diagnosed with dry eye disease – the itchy, uncomfortable and inconvenient condition that remains the number one reason for ophthalmologist visits. It’s no surprise that Mitotech’s most advanced clinical program targets this underserved indication, but the company has more than just dry eye treatment in its clinical pipeline.

The company’s lead compound SkQ1 targets a biological process that has not been adequately addressed by existing ophthalmic therapies: mitochondria-specific oxidative stress. Since the introduction of the concept of free radical damage, there has been a plethora of scientific experiments and studies that point a finger at oxidative stress inside mitochondria as a factor in a number of disorders afflicting aging populations. And that includes a variety of eye disorders, including those where treatments are scarce.

Although there are molecules that can neutralize reactive oxygen species, protecting our mitochondria from damage by oxidative stress has traditionally been problematic because these molecules distribute throughout the body but don’t reach effective levels in the mitochondria. And that’s the problem that Mitotech is trying to solve with its novel mitochondria-targeted drug candidate formulated specifically for use in ophthalmology.

Outside dry eye mitochondria-specific oxidative stress has been implicated in several eye disorders connected to ageing such as dry AMD and glaucoma. In glaucoma oxidative stress may play a critical role in both the mechanism behind elevation of intraocular pressure (IOP) and the neurological damage that follows. When the former has a spectrum of available treatments, the latter remains unaddressed. Mitotech’s Phase 2 data demonstrated significant reduction of IOP with topical administration of its mitochondria-targeted drug, while its neuroprotective properties are still to be demonstrated in a clinical setting.

For Dr John Sheppard, president, Virginia Eye Consultant, Norfolk, the need for a new glaucoma treatment is obvious. “We have seen many new IOP-lowering interventions, both pharmacologic and surgical, emerge for our glaucoma patients, but in my opinion there is still ample room for versatile topical medications targeting both IOP and ocular surface disease.  Dry eye is ubiquitous in the aging glaucoma population, where efficacious prescription glaucoma drops nevertheless also create somewhat tolerable but truly undesirable dryness, redness and irritation.  A novel pharmaceutical intervention simultaneously effectively controlling both glaucoma and dry eye addresses a highly prevalent problem.  An even more essential and lofty target is neuroprotection of the glaucomatous optic nerve.  No proven treatment currently exists to prevent or repair damage to the optic nerve, the actual cause of permanent vision loss in glaucoma patients.  Such a treatment could transform the field entirely.”

While Mitotech pursues well-established ophthalmic therapy markets, it also has set its sights on orphan mitochondrial disorders with no adequate treatments such as Leber’s Hereditary Optic Neuropathy (LHON). Its data from an uncontrolled clinical study indicated vision improvement in patients suffering from this blinding disease after six months of treatment. The company hopes to confirm this finding in a controlled randomized study as the next step along its regulatory path for LHON indication in the U.S.

“We think that properly addressing oxidative stress in mitochondria is a critical missing piece in treatment of many eye disorders,” said Natalia Perekhvatova, CEO of Mitotech. “That includes indications with markets from stable to high growth rates, as well as orphan indications. We believe that mitochondria-targeted therapies offer a unique approach that has a potential to benefit millions of patients and we are working hard on our parallel development programs.”

Mitotech S.A. is a Luxembourg-based biotechnology company developing novel drugs for treatment of predominantly age-related disorders. The core technology behind Mitotech products is a novel class of small molecules – mitochondria targeted cardiolipin peroxidation inhibitors. Company’s lead compound SkQ1 is being developed in several drug formulations covering a variety of therapeutic areas with major focus on ophthalmology and neurodegenerative diseases. Mitotech successfully conducted two large clinical studies for Dry Eye indication in the U.S. with other indications at earlier stages of clinical development.