BLOG: First drug for ptosis fills gap
A few years back, our clinical research department was approached by Icon, a clinical research organization running what sounded like an interesting study.
The project was a human trial for RVL, a small company whose only asset, oxymetazoline, was seeking approval as the first non-surgical on-label treatment of acquired blepharoptosis or ptosis (droopy eyelid). In the absence of an approved on-label pharmaceutical indicated for adult-onset ptosis, surgery was the only option for these patients, making the agent particularly intriguing.
I recognized that a pharmaceutical for ptosis could potentially fill a big gap in our repertoire of treatments. As is often the case in such situations, a dearth of noninvasive treatment options means that ptosis can often be overlooked and underdiagnosed. Previously, eye care specialists had little choice other than to refer these patients to an oculoplastic surgeon. I looked into what was known about oxymetazoline, a direct-acting alpha-adrenergic receptor agonist, and found it to be shown safe and, in exploratory studies, had reasonable efficacy. My research department decided to partner with Icon, the CRO running the trial, and work again with medical monitor and oculoplastic surgeon Chuck Slonim at the University of Florida in Gainesville, who I knew from previous projects.
The product performed very well in the pivotal studies, meeting its primary endpoints. Specifically, oxymetazoline demonstrated statistically significant improvements compared with placebo in both superior visual field, as measured by the Leicester Peripheral Field Test, and eyelid lift, as measured by the marginal reflex distance test 1. The agent was well tolerated when administered once daily in the morning (to both eyes) over a 12-week period, and the majority of adverse events were mild and self-limited.
We — and more importantly, the subjects in the trial — were excited by what we saw. As it turned out, Osmotica Pharmaceuticals purchased the asset, making RVL a subsidiary. Executive vice president and chief operating officer James Schaub asked for a meeting, looking to get feedback from my team involved in the studies. My office and R&D staff showed incredible enthusiasm about the drug, sharing with him how happy the clinical trial patients were with their results and their excitement over seeing the benefit almost instantly. That is really unique for all of us to see — participants in clinical trials do not usually have that sort of reaction.
Our practice was invited to continue the relationship and to share our clinical experience with our colleagues; we were happy to stay involved. As the drug, now called Upneeq, became commercialized, Osmotica brought in a team that included good friends I knew from other companies, which helped the partnership to further blossom. Not only am I developing relationships with people in the company, but I have also gotten to work with new physicians.
Osmotica is not trying to circumvent the need for surgical correction with Upneeq, but rather provide another option for certain subgroups of patients, particularly those with mild to moderate ptosis. Many are not good candidates for surgery for a variety of reason. I have yet to find a patient concerned with their ptosis who has not wanted to try it.
Upneeq has also made me a better diagnostician. I did not always think about ptosis, primarily focusing my attention to the eye itself and not the lid during exams. It forced me to go back and re-review and re-understand the etiology and implications of ptosis. I have a more empathetic understanding for patients with the condition and have learned how to gently broach the topic without making people feel self-conscious.
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