PanOptica reported positive clinical data for PAN-90806, a once-daily topical formulation of a small-molecule anti-vascular endothelial growth factor (anti-VEGF) eye drop for the treatment of neovascular eye diseases, according to a company news release. At the Ophthalmology Innovation Summit at the American Academy of Ophthalmology annual meeting (OIS@AAO) in San Francisco, the company presented topline results from a phase 1/2 dose-ranging clinical trial in which PAN-90806 was the first topical anti-VEGF eye drop to demonstrate both safety and biological response as monotherapy in treatment-naïve patients with neovascular age-related macular degeneration (AMD).
More than half of participants receiving once-daily topical PAN-90806 ophthalmic suspension for 12 weeks completed the study without needing rescue with anti-VEGF intraocular injection medication. Of those patients, 88% experienced either clinical improvement or stability of their disease, as confirmed by a panel of independent retina experts, with no serious or severe adverse effects related to PAN-90806.
“The phase 1/2 trial results constitute the most robust set of data exploring a topical anti-VEGF eye drop as monotherapy, and the favorable safety profile and biological response to PAN-90806 support its continued clinical development in wet AMD and other neovascular eye diseases,” Paul G. Chaney, president and chief executive officer of PanOptica, said in the news release. “The results suggest that topical ocular PAN-90806 may provide clinical benefit and substantially reduce the injection burden for patients with wet AMD, while increasing their chances for improved vision outcomes though maintenance of anti-VEGF therapy during the chronic management of their disease. We are now engaging with potential strategic partners in an effort to support the rapid development, registration, and commercialization of PAN-90806.”
Based on the strength of these data, PanOptica is eager to begin pivotal trials and has retained JMP Securities to represent the company in merger and acquisition discussions and to manage ongoing and future dialogue with potential strategic partners.
Phase 1/2 Neovascular AMD Trial Data
The double-masked, dose-ranging phase 1/2 trial randomized 51 treatment-naïve patients to one of three once-daily doses of PAN-90806 monotherapy: 2 mg/mL (n = 16), 6 mg/mL (n = 18), or 10 mg/mL (n = 17). The primary endpoint was safety/tolerability; the secondary endpoint was anti-VEGF biological response. The study protocol allowed for rescue medication injections with ranibiziumab beginning Week 2 of the 12-week study; investigators followed patients through one month (Week 16) following PAN-90806 discontinuation.
The independent Safety Monitoring Committee characterized PAN-90806 as reasonably safe and well-tolerated, with no major or serious drug-related safety concerns or trends. PAN-90806 ophthalmic suspension also exhibited improved tolerability and significantly improved safety over the previous, abandoned clinical solution. Key safety results are summarized below:
- Nine patients (17.6%) reported at least one PAN-90806-related adverse event (AE); none were serious. Five patients (9.8%) reported six PAN-90806-related corneal AEs.
- Three patients discontinued eye drop therapy prior to their Week 12 visit; two of these discontinuations were considered related to PAN-90806.
- Reported ocular adverse events were consistent with those observed in an AMD population and following treatment with topical ocular medications in general.
- Reported non-ocular events were consistent with those observed in elderly populations.
“The phase 1/2 safety results and findings associated with anti-VEGF biological activity suggest that a topical eye drop may make anti-VEGF therapy safer, less burdensome, and more sustainable by helping patients avoid the frequent intravitreal injections that current regimens require,” Allen C. Ho, MD, FACS, Wills Eye Hospital Attending Surgeon and Director of Retina Research, Professor of Ophthalmology, Thomas Jefferson University, Mid Atlantic Retina, President, Retina Society, said in the news release. “Once-daily topical ocular PAN-90806 creates the opportunity to change the wet AMD treatment paradigm by potentially improving patient adherence, reducing losses to follow-up, and maintaining early patient benefits obtained with initial injection treatment throughout what may often be chronic, lifelong, anti-VEGF therapy.”