On May 15, 2023 Ocuphire Pharma, Inc. (Nasdaq: OCUP), a clinical-stage ophthalmic biopharmaceutical company focused on developing novel therapies for the treatment of unmet needs of patients with retinal and refractive eye disorders, reported financial results for the first quarter ended March 31, 2023, and provided a corporate update (Press release, Ocuphire Pharma, MAY 15, 2023, View Source [SID1234631745]).

"Ocuphire made significant progress across both our APX3330 and Nyxol programs throughout the first quarter," said Rick Rodgers, Interim Chief Executive Officer. "Based on the efficacy and safety data from the ZETA-1 Phase 2 trial of APX3330, we are preparing for an End-of-Phase 2 meeting with the FDA to confirm Phase 3 registration endpoints and study parameters. If approved, APX3330 has the potential to be a non-invasive, oral, early intervention treatment for millions of DR patients at risk of progressing to vision-threatening complications. For Nyxol, we look forward to the September 2023 PDUFA date in its first indication in RM. With a strong cash position and our partner Viatris funding the Nyxol program, we are well positioned to advance both APX3330 and Nyxol programs."

Key Anticipated Future Milestones

•
APX3330: End of Phase 2 meeting with FDA to confirm Phase 3 regulatory path for Oral APX3330 in DR (2H 2023)
•
Nyxol: PDUFA date for Nyxol in RM. FDA approval in RM would trigger a $10 million milestone payment to Ocuphire (September 28, 2023)
•
Nyxol: Topline results from VEGA-2 Phase 3 pivotal trial of Nyxol in Presbyopia (Late 2023)

Recent Business Highlights

Clinical and Regulatory Updates

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!

•
In January 2023, the Company announced topline results from the ZETA-1 Phase 2 trial of oral APX3330 for the treatment of diabetic retinopathy (DR). Oral APX3330 achieved statistical significance on a key pre-specified secondary endpoint of binocular ≥ 3-step worsening of DRSS and demonstrated favorable safety and tolerability. This binocular secondary endpoint is a potential Phase 3 registration endpoint. The Company plans an End-of-Phase 2 FDA meeting in the second half of 2023 to formally confirm this endpoint and other clinical trial parameters.

•
In February 2023, the Company announced that the FDA has accepted for review a New Drug Application (NDA) for Nyxol in RM and set a PDUFA date of September 28, 2023. FDA approval in RM would trigger a $10 million milestone payment to Ocuphire.

•
Topline results from the VEGA-2 Phase 3 pivotal trial of Nyxol in presbyopia are expected in late 2023. Nyxol clinical programs in presbyopia and dim light disturbances (DLD) continue to progress as planned.

Presentations, Publications, and Conferences

•
From January 2023 through May 2023, several papers, posters, and panel talks were presented at medical and industry conferences with updates on APX3330 in DR and Nyxol in RM, DLD and presbyopia. Highlights include:

•
Results from ZETA-1 Phase 2 trial of APX3330 in DR were presented for the first time to the medical community at the 20th Angiogenesis, Exudation, and Degeneration 2023 Meeting in February 2023.

•
Results from LYNX-1 Phase 3 trial of Nyxol in DLD were presented for the first time to the medical community at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting in April 2023, and at the American Society of Cataract and Refractive Surgery (ASCRS) Annual Meeting in May 2023.

•
Previously announced results from ZETA-1 Phase 2 trial of APX3330 in DR were presented to the medical community at the American Society of Cataract and Refractive Surgery (ASCRS) Annual Meeting in May 2023.

Corporate

In April, Ocuphire announced the appointment of Rick Rodgers as Interim President and Chief Executive Officer. Mr. Rodgers is a seasoned operating executive with 20 years of experience in biopharmaceutical management.

First Quarter ended March 31, 2023 Financial Highlights

As of March 31, 2023, Ocuphire had cash and cash equivalents of approximately $39.0 million. The Company has no debt. Based on current projections, management believes the present cash on hand will be sufficient to fund operations into 2025.

License and collaborations revenue was $1.7 million for the three months ended March 31, 2023. There was no revenue recorded in the three months ended March 31, 2022. Revenue during the first quarter of 2023 was derived from the reimbursement of research and development services under the Nyxol License Agreement.

General and administrative expenses for the three months ended March 31, 2023 were $2.3 million, compared to $1.7 million for the three months ended March 31, 2022. The increase was primarily attributable to an increase in stock-based compensation, professional services fees, legal support, and business development activities, offset in part by a decrease in payroll and insurance costs on a net basis. General and administrative expenses included $0.5 million and $0.3 million in stock-based compensation expense during the three months ended March 31, 2023, and 2022, respectively.

Research and development expenses for the three months ended March 31, 2023 were $5.6 million, compared to $4.8 million for the three months ended March 31, 2022. The increase was primarily attributable to increased manufacturing activities for Nyxol and APX3330 period over period as well as increased payroll and consulting costs during the current period. Research and development expenses also included $0.3 million and $0.1 million in stock-based compensation expense during the three months ended March 31, 2023, and 2022, respectively.

The loss from operations for the quarter ended March 31, 2023 was $6.1 million, compared to $6.5 million for the quarter ended March 31, 2022.

Net loss for the quarter ended March 31, 2023 was $5.8 million or ($0.28) per share, compared to $6.6 million or ($0.35) per share for the quarter ended March 31, 2022.

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, 2023 to be filed with the Securities and Exchange Commission.