Ocuphire Pharma is forging ahead with an FDA end-of-Phase II meeting, despite not achieving the primary endpoint with its oral diabetic retinopathy (DR) drug, APX3330. With unwavering determination, they are continuing to make strides towards a potential breakthrough in DR treatment.
The primary endpoint of this study was to assess whether APX3330 could improve patient scores on the Diabetic Retinopathy Severity Scale (DRSS), a categorical tool which evaluates the severity of retinopathy in the eyes of patients by looking at photographs.
The results of the data readout did not demonstrate a statistically significant improvement in reversing the vision-impacting effects of diabetic retinopathy with the use of APX3330.
The candidate’s treatment was a resounding success, with statistically significant results demonstrating the prevention of clinically meaningful progression of DR after 24 weeks of treatment in both eyes – a key pre-specified secondary endpoint.
The therapeutic proved to be highly successful in maintaining visual acuity, with fewer patients experiencing a loss of distance vision than those treated with a placebo. This demonstrated a significant stabilizing effect of the treatment.
APX3330 proved to be an effective and well-tolerated treatment, with mild side effects reported by patients. Most of the treatment-related adverse events were of mild severity, and uncommon overall. The safety and tolerability profile was generally favorable.
Phase III Objectives
If approved, APX3330 could offer a groundbreaking primary preventative therapeutic option for patients in the earlier stages of their disease. During a conference call Wednesday, the company highlighted the potential this new treatment could have in dramatically improving the health and wellbeing of those affected.
Charles Wykoff, M.D., Ph.D., a vitreoretinal specialist with the Retina Consultants of Texas, believes that a safe systemic drug could be the answer to preventing vision loss for patients suffering from diabetic retinopathy. During his presentation, Wykoff noted that this type of intervention is a “clear clinical intervention” for those affected by the condition. He stressed that this type of drug could be a major breakthrough in helping protect patients from losing their eyesight.
Ocuphire revealed in its presentation that a Phase III trial could be successful if it prevents 3-step binocular worsening in DRSS scores in both eyes. This endpoint could be a promising indicator of success.
Mitchell Brigell, Ph.D., head of clinical development and strategy, has identified a unique opportunity to gain approval for retinal drugs. By demonstrating an improvement or prevention of worsening of retinal disease, new treatments can now be accessed and utilized to help those suffering from vision-related issues.
A Phase III trial will demonstrate the necessity of measuring DRSS change in both eyes, as APX3330 is a systemic drug, unlike Anti-VEGF medications which are usually injected into one eye at a time. This endpoint is of utmost importance, as it will provide insight into the efficacy of the drug across both eyes.
The Oral Drug Difference
Although there are drugs that can effectively slow and reverse the effects of DR, they are not as widely used as they could be, according to Dr. Wykoff. These drugs have been approved and are capable of restoring vision and improving vision-related quality of life, yet many people living with DR still do not take advantage of them.
Delivering an intravitreal injection presents a daunting challenge, one that must be faced on a chronic basis. It is a challenge that requires a great deal of skill and expertise in order to be successful.
Intravitreal injections offer a critical opportunity to those diagnosed with eye-related conditions, as they are administered directly into the vitreous cavity in the back of the eye. By delivering the treatment directly to the affected area, patients can slow the disease’s progression and maintain their sight, even if they were already well-sighted at diagnosis.
Further exploration of the trial data could provide key insights for Ocuphire’s upcoming Phase III registration and trial design. With a more detailed analysis of the data, the team can gain a better understanding of the potential for success and make the necessary adjustments to ensure a successful outcome.
We are excited to announce that the development of APX3330, including its cGMP drug, NDA-enabling work, the first Phase III trial, and regional partnerships, is fully funded until 2025. This is an incredible opportunity for us to make a lasting impact in the healthcare industry.