Many readers have been following closely the development of Squalamine Eye Drops for wet age-related macular degeneration, hoping that a self-administered at-home eye drop could reduce, or even eliminate, the need for monthly or as-needed eye injections. Unfortunately, a clinical trial designed to test this concept has produced disappointing results: Squalamine Eye Drops failed to reduce the average number of Lucentis injections required by the trial participants.
However, that clinical trial also revealed that squalamine produced positive changes in visual acuity – both overall in the Lucentis/squalamine treatment group, and, more significantly, in participants with a specific type of AMD lesion. As a result, a newer clinical trial, initiated in 2016 and continuing into 2019, will continue to study the effects of Squalamine Eye Drops, in combination with Lucentis injections, on gains in visual acuity in persons with wet macular degeneration.
What Is Squalamine?
Ohr Pharmaceutical, Inc. is dedicated to the clinical development of new drugs for underserved therapeutic needs. One of its lead drugs in development is Squalamine Eye Drops for the treatment of wet age-related macular degeneration (AMD).
Squalamine is a water-soluble molecule derived from the internal organs (primarily the liver) of the dogfish shark. It is believed to have great potential for treating some human viruses. Medically, squalamine has been shown to interrupt and reverse the process of angiogenesis.
About Angiogenesis and Anti-Angiogenic Drugs
Angiogenesis is a term that describes the growth of new blood vessels and plays a critical role in the normal development of body organs and tissue. Sometimes, however, excessive and abnormal blood vessel development can occur in diseases such as cancer (tumor growth) and AMD (retinal and macular bleeding).
Substances that stop the growth of these excessive blood vessels are called anti-angiogenic (anti=against; angio=vessel; genic=development), and anti-neovascular (anti=against; neo=new; vascular=blood vessels).
The focus of current anti-angiogenic drug treatments for wet macular degeneration is to reduce the level of a particular protein (vascular endothelial growth factor, or VEGF) that stimulates abnormal blood vessel growth in the retina and macula; thus, these drugs are classified as anti-VEGF treatments.
The squalamine researchers theorized that the ability to self-administer eye drops to treat wet AMD could be more effective (and less invasive) than the current standard of care, which involves regular injections of Lucentis, Avastin, or Eylea directly into the eye, via a very small needle. These injections are administered at the doctor’s office.
A History of Squalamine Eye Drop Research
Initially, the Genaera Corporation developed the squalamine project and administered squalamine as an intravenous formulation for wet AMD in Phase 1 and Phase 2 clinical trials. However, in 2007, Genaera discontinued their clinical trials, due to financial and subject-recruitment issues. The squalamine project was then acquired by Ohr Pharmaceutical, which continued the development of squalamine in a topical eye drop formulation.
In 2012, Ohr was awarded “Fast Track” designation by the United States Food and Drug Administration (FDA) to further develop its Squalamine Eye Drop product for the potential treatment of wet AMD. Fast Track is a process designed to facilitate the development, and expedite the review, of drugs to treat serious diseases and fill an unmet medical need. Filling an unmet medical need is defined as providing a therapy where none exists or providing a therapy that may be potentially superior to existing therapy.
The Phase 2 Clinical Trial
In August 2012, the FDA commenced a Phase 2 clinical trial to study the safety and effectiveness of Squalamine Eye Drops in combination with Lucentis injections. The researchers were seeking to determine (a) if squalamine was safe (safety) and (b) if Squalamine Eye Drops could reduce the number and frequency of Lucentis injections required to control the neovascularization and retinal bleeding associated with wet AMD (effectiveness).
The initial enrollment in the clinical trial was 142 “treatment-naïve” participants