Patients with neovascular age-related macular degeneration (wet-AMD) have some solid treatment options that can decrease the amount of visual acuity loss due to the disease. Roche's (OTCQX:RHHBY) Lucentis is the most commonly recognized of the treatments and was approved in 2006, bringing in $1.5 billion in sales for 2012. A newer therapeutic, Regeneron's (NASDAQ:REGN) Eylea, has gone on an explosive run, increasing sales in 1Q 2013 nearly 90% from 1Q2012, with sales of $837.9 million for 2012, the first year of FDA approval. However, both of these treatments, and others not mentioned, require patients to undergo an intravitreal injection. An intravitreal injection is literally an injection directly into the eye, targeting the vitreous humor, a clear gel substance between the lens and retina. This form of treatment is justified considering the deleterious impact of wet-AMD on the retina, but is surely a discomfort for patients. Moreover, the prescribing information for both Lucentis and Eylea list conjuctival hemorrhage and eye pain as the top two most common adverse events.
Eylea's sales run is most likely due to its dosing structure, which is once every two months versus once a month for Lucentis, especially considering the similar efficacy of the two drugs. This trend toward adopting less invasive therapies suggests that there is a strong opportunity for a treatment that does not require injections. Ohr Pharmaceutical (NASDAQ:OHRP) is working to make these injections a thing of the past. The company's eye drop-based treatment would definitely benefit patients, allowing comfortable administration of drug without the pain and complications of a needle. Ohr Pharmaceutical just announced that it has hit the 50% enrollment mark for a Phase II trial testing its eye drop treatment, and is expecting to release interim results in the second quarter of 2014. This company looks well capitalized, and its eye drop-based treatment would be a complete game changer.
Ohr Pharmaceutical's pipeline contains three products, although the main focus of the company is on its lead candidate squalamine, an eye drop formulation designed to treat wet-AMD. Ohr also has OH/AVR 118 under development to treat cancer associated wasting known as cancer cachexia, and will out-license this program to fund development of squalamine. Importantly, in addition to potential revenues from licensing agreements, Ohr has plenty of cash relative to operating expenses, and nearly no debt ($317,936 at the time of publication). Ohr should have no issues pushing the squalamine eye drop further in clinical trials in an effort to materialize its research efforts for investors.
Age-Related Macular Degeneration
AMD is a progressive condition that usually presents in older adults and can result in total loss of central vision. Although central vision only represents around 2% of the visual field, half of the brain's visual processing center is devoted to it, allowing for detailed vision. Consequently, central vision loss in AMD patients reduces the ability to perform daily tasks, read, drive or even recognize faces. Although peripheral vision remains intact, AMD patients are greatly impaired by their severely reduced visual function.
AMD can be grouped into two common types. The first, nonexudative (dry) AMD results from wasting of the retinal pigment layer and concomitant loss of photoreceptors from the central portion of the eye. As this happens, debris called drusen accumulates between two layers of the eye, the retina and the choroid, which can cause the retina to detach. Dry AMD results in gradual vision loss, comprises 90% of AMD cases, but is notably less severe than its counterpart wet AMD.
Wet AMD is the result of abnormal blood vessel growth that leads to bleeding and leaking of proteins that penetrate the membrane separating the choroid and the retina. Leakage of blood into the retina causes fluid build-up and eventually retina detachment. The wet mechanism of AMD is much more rapid and severe than dry AMD and although it only accounts for 10% of cases, it is the cause of nearly 90% of severe vision loss associated with AMD, impacting more than 1.75 million patients in the US alone. It is the leading cause of blindness in older adults. As mentioned above, wet AMD is treated with intravitreal injections that inhibit vascular endothelial growth factors, which subsequently inhibits angiogenesis.
Squalamine eye drop formulation to treat wet AMD
Ohr's squalamine eye drop formulation is also designed to inhibit inappropriate blood vessel growth associated with wet AMD. Similar to Lucentis and Eylea, squalamine blocks the growth factor VEGF. However, there are several important advantages of Ohr's topical formulation over the currently approved injections. Squalamine goes further than these previous drugs in that it also blocks other angiogenic factors including PDGF and bFGF, which could help reduce some of the fibrosis and morbidity associated with AMD. Of course, the main advantage is that these drops can be self administered and will eliminate the adverse events associated with injections.
A Phase II clinical trial commenced in September of 2012 to evaluate the safety and efficacy of squalamine eye drops in 120 wet AMD patients. The study examines the drops in combination with traditional intravitreal injection therapy, since depriving a patient of the standard of care would be a hard sell during enrollment. Patients will receive a Lucentis injection at the beginning of the trial, start squalamine treatment and be evaluated at the end of each month to determine if further Lucentis injections are necessary. The primary endpoint is the need for rescue intravitreal injections using Lucentis, and secondary endpoints include visual acuity and safety. Optimally, the drops would successfully stabilize patients and eliminate the requirement for further injections, but even a reduction of intravitreal injections would benefit patients. Ohr announced on July 10 that it has achieved 50% enrollment in the study and expects to release interim results in the second quarter of 2014.
If Eylea's successful launch says anything about the AMD market, it's that patients are not happy with intravitreal injections. Ohr Pharmaceutical is trying to take the next step and eliminate the injections entirely. Ohr has a good shot at this market for several reasons. First, it managed to keep cash reserves high relative to the burn rate. After raising over $5 million from the exercise of warrants in April 2013, cash reserves of $6.7 million should last for 2 years with operating expenses averaging around $3 million per year. Second, with multiple drugs in its clinical pipeline, Ohr has the opportunity to leverage the firm's other assets, namely OH/AVR 118, to fund ongoing clinical trials for its flagship squalamine eye drops.
The company has been very efficient in clinical and preclinical trials to date. There is little doubt that the experienced management team is capable of progressing through additional clinical trials. The remaining question for this company is whether squalamine drops have good efficacy for wet-AMD patients, and this will be answered in the near future. I think Ohr has a huge potential to improve the quality of life for wet-AMD patients and compete in this space. Investors should keep this Phase II trial in mind as they watch Eylea sales grow.
The question at hand is how successful will the squalamine eye drops be at treating wet AMD. Given the existing data, it seems likely that positive results will be presented in support of this therapy in early 2014. An investment in OHRP may, thus, be worth considering in the period of time leading up to this Q1/Q2 2014 catalyst.
Disclosure: I have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.