On May 7, 2021 Ocuphire Pharma, Inc. (Nasdaq: OCUP), a clinical-stage ophthalmic biopharmaceutical company focused on developing and commercializing therapies for the treatment of several eye disorders, reported financial results for the first quarter of 2021 and provided a corporate update (Press release, Rexahn, MAY 7, 2021, View Source [SID1234579465]).
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
"The highlight of the first quarter was our announcement of positive top-line results from the pivotal MIRA-2 Phase 3 trial investigating Nyxol eye drops for reversal of pharmacologically-induced mydriasis (pupil dilation). The highly statistically significant results provide the foundation to advance Nyxol towards NDA submission in an indication representing over 100 million annual eye exams with no commercial treatments currently available", said Mina Sooch, MBA, President and CEO of Ocuphire Pharma. "The recent Phase 3 data further validate Nyxol’s unique mechanism of action on the iris dilator muscle, its consistent therapeutic effect of pupil diameter reduction, and its favorable safety and tolerability profile for additional refractive indications including Presbyopia and Night Vision Disturbances. We are also making progress with our second product candidate, APX3330, and are currently enrolling patients in a Phase 2 trial primarily in Diabetic Retinopathy. Overall, 2021 is off to a strong start and we look forward to more updates on our programs throughout the year including the Phase 2 Presbyopia data readout expected at the end of June."
Cam Gallagher, MBA, Chair of Ocuphire’s Board of Directors commented, "Since our public listing via reverse merger just six months ago, the management team together with Ocuphire’s clinical and manufacturing partners have successfully launched an impressive four Phase 3 and 2 clinical trials for Nyxol and APX3330, clearly demonstrating their execution capabilities. Importantly, data from multiple trials at Ocuphire have been published in several peer-reviewed journals and presented at multiple ophthalmic and healthcare banking conferences, further establishing our presence within the industry. We look forward to continuing this momentum in 2021, and with our recent positive Phase 3 data, we are excited to move ahead with our commercialization strategies for Nyxol."
Key Anticipated Future Milestones
Presbyopia: Top-line data expected end of Q2 2021 for Phase 2 VEGA-1 trial investigating a kit combination of Nyxol and low-dose 0.4% pilocarpine
Reversal of Mydriasis: Presenting Phase 3 MIRA-2 results at the 2021 American Society of Cataract and Refractive Surgeons conference in Las Vegas in July
Night Vision Disturbances: Top-line data expected end of Q3 2021 for pivotal Phase 3 LYNX-1 trial investigating Nyxol
Reversal of Mydriasis: Planning to initiate second Phase 3 MIRA-3 registration trial in 2H 2021 investigating Nyxol with results expected in early 2022
Diabetic Retinopathy and Diabetic Macular Edema: Completion of enrollment in Phase 2 ZETA-1 trial investigating APX3330 with top-line data expected early 2022
First Quarter and Recent Business Highlights
Clinical Development
Reported positive results in pivotal Phase 3 MIRA-2 trial investigating Nyxol for Reversal of Mydriasis (within 4 months of initiation) which met primary and multiple secondary endpoints demonstrating a more rapid return to baseline pupil diameter after dilation across multiple commonly used dilating agents and iris colors
Initiated ZETA-1 Phase 2 trial investigating the first-in-class oral anti-VEGF and anti-inflammatory APX3330 in Diabetic Retinopathy and Diabetic Macular Edema
Initiated Phase 2 VEGA-1 trial evaluating a kit combination of Nyxol and low-dose pilocarpine in Presbyopia with a differentiated pharmacologic approach that moderately acts on both the iris dilator and iris sphincter muscles that control pupil diameter
Presentations and Publications
Presented positive pre-clinical data supporting oral APX3330’s efficacy and sufficient exposure to the retina at the Association for Research in Vision and Ophthalmology (ARVO) virtual Annual Meeting
Published positive results from the MIRA-1 Phase 2b clinical trial evaluating the safety and efficacy of Nyxol for Reversal of Mydriasis in Optometry and Visual Science journal
Presented highlights of Nyxol presbyopia program at the 2021 Ophthalmology Innovation Summit (OIS) Presbyopia Innovation Showcase
First Quarter 2021 Financial Highlights
As of March 31, 2021, the Company had cash and cash equivalents of approximately $10.6 million.
General and administrative expenses for the three months ended March 31, 2021 were $1.7 million compared to $0.4 million for the three months ended March 31, 2020. The $1.3 million increase was primarily attributable to an increase in administrative employee headcount, stock-based compensation, professional services, insurance, and legal costs associated with the operating as a public company in the current period subsequent to the merger.
Research and development expenses for the three months ended March 31, 2021 were $3.5 million compared to $0.2 million for the three months ended March 31, 2020. The $3.3 million increase as compared to the prior period was primarily attributable to four new clinical trials and manufacturing activities for Nyxol and APX3330 as well as regulatory, preclinical, and other development activities.
The total GAAP loss from operations for the three months ended March 31, 2021 was $5.2 million compared to $2.7 million for the three months ended March 31, 2020. This included non-cash stock-based compensation expense of $494,000 and $61,000, respectively.
The quarter ended March 31, 2021 included a non-cash charge of $33.8 million related to the fair value change in warrant liabilities included in other expense. The quarter ended March 31, 2020 included a non-cash charge of $0.6 million related to interest expense on convertible notes and a $0.2 million non-cash benefit due to the fair value change in premium conversion derivatives related to convertible notes, both included in other expense. As a result, GAAP net loss attributable to common stockholders for the quarter ended March 31, 2021 was $39.0 million compared to $3.1 million for the quarter ended March 31, 2020.
Effective February 3, 2021, each investor that invested in the Pre-Merger Financing entered into a Waiver Agreement with Ocuphire. The Waiver Agreements provide for the elimination of the full ratchet anti-dilution provisions contained in the Series A Warrants (as certain of the anti-dilution provisions had previously caused liability accounting treatment for the Series A Warrants). Upon the effective date of the Waiver Agreements, the Series A Warrants were reclassified to equity. In addition, the investors agreed to waive certain rights, finalize the exercise price and number of Series A Warrants and Series B Warrants, eliminate certain financing restrictions, extend the term of certain leak-out agreements, and, in the case of certain investors, grant certain registration rights for the shares underlying the Series A Warrants.
Non-GAAP adjusted net loss was $5.2 million or ($0.47) per share for the three months ended March 31, 2021, compared with a non-GAAP adjusted net loss of $3.3 million or ($0.93) per share for the three months ended March 31, 2020. Non-GAAP adjusted net loss for the three months ended March 31, 2021 and 2020 excludes expenses for the fair value change in warrant liabilities related to the Pre-Merger Financing and premium conversion derivatives related to convertible notes, respectively. See "Non-GAAP Financial Measures" and "Reconciliation of GAAP to Non-GAAP Financial Measures" below for a reconciliation of this GAAP and non-GAAP financial measure.
For further details on Ocuphire’s financial results, refer to the Company’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2021, to be filed with the Securities and Exchange Commission.