On August 12, 2021 Ocuphire Pharma, Inc. (Nasdaq: OCUP), a clinical-stage ophthalmic biopharmaceutical company focused on developing and commercializing therapies for the treatment of refractive and retinal eye disorders, reported financial results for the second quarter of 2021 and provided a corporate update (Press release, Ocuphire Pharma, AUG 12, 2021, View Source [SID1234586458]).

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"The second quarter of 2021 proved to be highly productive for Ocuphire, with continued success in clinical trials, key pivotal data presentations featured at major medical meetings across both Nyxol and APX3330, and additional IP protection granted for our lead drug candidate Nyxol," said Mina Sooch, MBA, President and CEO of Ocuphire Pharma.

"Following our first quarter announcement of positive MIRA-2 Phase 3 results of Nyxol for the treatment of reversal of mydriasis (RM), we also announced positive top-line results from our VEGA-1 Phase 2 study, which evaluated Nyxol in combination with low-dose pilocarpine (LDP) for the treatment of presbyopia, the gradual loss of your eyes’ ability to focus on nearby objects. In our view, RM and presbyopia both represent attractive and large US and global market opportunities, with each indication having a significant unmet medical need with very few if any pharmaceutical treatment options. Following the recent ASCRS meeting, it is also clear that global pharmaceutical and consumer healthcare companies are aggressively pursuing innovative new medicines, further validating the significant commercial potential for the large presbyopia market. Based upon the success of our VEGA-1 trial, we are highly confident that Nyxol in combination with LDP presents a potentially best-in-class therapeutic solution that can address the needs of the presbyopia patient population."

"With our balance sheet recently strengthened in the second quarter combined with our capital efficient operations, Ocuphire is well positioned to deliver additional late-stage clinical milestones in 2022 for Nyxol and APX3330 while continuing our global business development efforts."

Key Anticipated Future Milestones

Reversal of Mydriasis (RM): Initiate second Phase 3 (MIRA-3) registration trial and a small pediatric trial (MIRA-4) in the second half of 2021 investigating Nyxol with results expected in early 2022; Planning to file NDA submission with FDA for Nyxol in RM indication in late 2022
Presbyopia: Initiate Phase 3 program (VEGA-2/3) in first half of 2022 investigating Nyxol and LDP
Night Vision Disturbances: Completion of enrollment expected by year-end 2021 and top-line data expected in early 2022 from Phase 3 (LYNX-1) registration trial investigating Nyxol
Diabetic Retinopathy and Diabetic Macular Edema: Completion of enrollment in Phase 2 (ZETA-1) trial investigating APX3330 and top-line data expected in 2022

Second Quarter and Recent Business Highlights

Clinical Development

In June, the Company announced successful results from the VEGA-1 Phase 2 trial of Nyxol plus low-dose pilocarpine (LDP) for the treatment of presbyopia; the trial met its primary endpoint of 3 lines of near vision improvement and multiple key secondary endpoints such as fast onset of action and durability with statistical significance and a favorable safety profile (including no headaches)
In April, the Company initiated the ZETA-1 Phase 2 clinical trial to evaluate oral APX3330 in non-proliferative diabetic retinopathy (NPDR) and mild proliferative diabetic retinopathy (mild PDR)

Presentations and Publications

In July, the Company announced publication in the Journal of Cellular Signaling featuring Ocuphire’s novel oral Ref-1 inhibitor APX3330 in Phase 2 trial for the treatment of retinal disease which highlighted the favorable safety profile of APX3330 and its unique anti-angiogenic and anti-inflammatory mechanism of action properties relevant to a broad range of retinal diseases
In July at the 2021 American Society of Cataract and Refractive Surgery (ASCRS) Annual Meeting, Dr. Jay S. Pepose, Medical Advisor and Board Director, presented two papers featuring the positive results Phase 2 Presbyopia (VEGA-1) and Phase 3 Reversal of Mydriasis (MIRA-2)
In July, Mina Sooch, CEO, participated in the presbyopia drug therapy panel at the Eyecelerator held on July 22nd and in the Eye on Innovation panel at the Virtual Salon Series held on July 28th
In late May, Ocuphire hosted a Key Opinion Leader Event on Nyxol as a potential new treatment option for reversing pharmacologically induced mydriasis, highlighting recent positive Phase 3 results from the MIRA-2 Phase 3 registrational study
In May at the 2021 Association for Research in Vision and Ophthalmology (ARVO) Virtual Annual Meeting, Ocuphire presented data for APX3330 on pre-clinical ocular data and predictive human retina and plasma model data

Corporate

Announced closing of a $15 million registered direct offering and just over $4 million from ATM, extending cash runway into late 2022
Appointed Jay S. Pepose, M.D., Ph.D. to the Company’s Board of Directors
Received two new U.S. patent grants covering Nyxol including Nyxol plus LDP claims for the treatment of presbyopia through 2039
Entered into a license agreement granting Processa Pharmaceuticals (Nasdaq: PCSA) an exclusive license to develop, manufacture and commercialize globally RX-3117 (Rexahn legacy oncology intellectual property), excluding China, Hong Kong, Macau, Republic of Singapore and Taiwan (already licensed to BioSense Global LLC ("BioSense"))
Ocuphire (Nasdaq: OCUP) added to the Russell Microcap Index

Second Quarter and Year to Date 2021 Financial Highlights

As of June 30, 2021, the Company had cash and cash equivalents of approximately $24.2 million. Net cash used in operating activities for the three and six months ended June 30, 2021, was $4.3 million and $10.1 million, respectively.

Collaborations revenue was $0.1 million for the three and six months ended June 30, 2021. Revenue during the periods was derived from the license agreement with BioSense related to certain technology transfers. There was no collaborations revenue recognized during the comparable prior year periods.

General and administrative expenses for the three and six months ended June 30, 2021, were $3.4 million and $5.1 million, respectively, compared to $0.6 million and $0.9 million, respectively, for the three and six months ended June 30, 2020. The increases from the comparable periods in 2020 were attributable to increased costs primarily in administrative employee headcount, stock-based compensation, insurance, legal and settlement costs, and costs associated with operating as a public company subsequent to the reverse merger.

Research and development expenses for the three and six months ended June 30, 2021, were $3.8 million and $7.3 million, respectively, compared to $0.7 million and $0.9 million, respectively, for the three and six months ended June 30, 2020. The increases from the comparable periods in 2020 were primarily attributable to four new clinical trials and manufacturing activities for Nyxol and APX3330 as well as regulatory, preclinical, and other development activities.

There were no acquired in-process research and development expenses in the current six-month period. In the prior year in connection with the sublicense agreement with Apexian for the continued research, development and potential commercialization of APX3330, the Company recorded acquired in-process research and development expenses of $2.1 million during the six-month period ended June 30, 2020.

The total loss from operations for the three and six months ended June 30, 2021 was $7.1 million and $12.3 million, respectively, compared to $1.3 million and $4.0 million for the three and six months ended June 30, 2020, respectively. This included non-cash stock-based compensation expense of $0.5 million and $1.0 million during the three and six months ended June 30, 2021, respectively, and $0.3 million and $0.4 million during the three and six months ended June 30, 2020, respectively.

For further details on Ocuphire’s financial results, including results for the three and six month periods ended June 30, 2021 refer to the Company’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2021, to be filed with the Securities and Exchange Commission.