Ohr Pharmaceutical, Inc., a company that is dedicated to the clinical development of new drugs for underserved therapeutic needs, has been awarded Fast Track designation by the U.S. Food and Drug Administration (FDA) for its Squalamine Eye Drops as a potential treatment for wet age-related macular degeneration (AMD).
In addition, Ohr has presented data from a squalamine Phase 1 clinical trial at the May 2012 Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO). The presentation highlighted the potential therapeutic value of the squalamine eye drop program in treating wet AMD. ARVO is an international organization that encourages and assists research, training, publication, and dissemination of knowledge in vision and ophthalmology, including low vision.
What is Squalamine?
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 (and most importantly for VisionAware readers), 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.
Squalamine Research and AMD
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 to treat wet AMD.
About Clinical Trials
Most clinical trials are designated as Phase I, II, or III, based on the questions the study is seeking to answer:
- In Phase I clinical trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe and effective dosage range, and identify possible side effects.
- In Phase II clinical trials, the study drug or treatment is given to a larger group of people (100-300) to determine if it is effective and to further evaluate its safety.
- In Phase III studies, the study drug or treatment is given to even larger groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
- In Phase IV studies, after the Food and Drug Administration (FDA) has approved the drug, continuing studies will determine additional information, such as the drug’s risks, side effects, benefits, and optimal use.
The Squalamine Eye Drop project is presently in Phase 1 clinical trials to evaluate its safety when used by human subjects. Ohr expects to commence a Phase 2 clinical trial in late summer 2012. Thus, the research is in its very earliest stages and its effectiveness for human subjects cannot be predicted.
About “Fast Track” Designation by the FDA
From The FDA for Consumers:
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. The purpose is to get important new drugs to the patient earlier. Filling an unmet medical need is defined as providing a therapy where none exists or providing a therapy which may be potentially superior to existing therapy.
Once a drug receives Fast Track designation, early and frequent communication between the FDA and a drug company is encouraged throughout the entire drug development and review process. The frequency of communication assures that questions and issues are resolved quickly, often leading to earlier drug approval and access by patients.
More about the Squalamine Eye Drop Research Project
The squalamine researchers hypothesize that the potential ability to self-administer eye drops to treat wet AMD may 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.
From The Wall Street Journal Market Watch:
“We believe the Fast Track designation underscores the importance of developing an eye drop for the treatment of wet AMD and its potential to address an unmet medical need for this large and growing patient population,” commented Dr. Irach B. Taraporewala, CEO of Ohr. “Our eye drop could provide tremendous benefit to these patients who currently need to take chronic intravitreal injections of … Lucentis or … Eylea directly into the eye. We are thrilled with the continued progress of the program and anticipate commencing a Squalamine Eye Drop phase II clinical trial in the third quarter of 2012.”
VisionAware will provide updates on this intriguing research as they become available.
Sources: ARVO May 2012 Annual Meeting; The Wall Street Journal Market Watch; Eye Doc News