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Ocuphire Announces Financial Results for the Fourth Quarter and Year Ended 2021 and Provides Corporate Update Building Momentum with Completion of Patient Enrollment in 4 Late-Stage Trials Multiple Late-Stage Clinical Tr

Key Takeaway: Ocuphire Announces Financial Results for the Fourth Quarter and Year Ended 2021 and Provides Corporate Update Building Momentum with Completion of Patient Enrollment in 4 Late-Stage Trials Multiple Late-Stage Clinical Trial Data Catalysts in 2022: -Nyxol MIRA-3 Phase 3 Results

Full Press Release Details

Ocuphire Announces Financial Results for the Fourth Quarter and Year Ended 2021 and Provides Corporate Update
Building Momentum with Completion of Patient Enrollment in 4 Late-Stage Trials
Multiple Late-Stage Clinical Trial Data Catalysts in 2022:
-Nyxol MIRA-3 Phase 3 Results Expected End of 1Q 2022
-MIRA-4 Pediatric, and LYNX-1 Phase 3 Results Expected to follow in 2Q 2022
-ZETA-1 Phase 2 Diabetic Retinopathy Results Expected in 2H 2022
NDA Filing for Nyxol in Reversal of Mydriasis Planned for Late 2022 with Potential Launch as First Dilation Reversal Drop in 2H 2023
Plans to Initiate VEGA Phase 3 FDA Registration Trials for Nyxol Alone and in Combination with Low-Dose Pilocarpine (LDP) in Presbyopia
FARMINGTON HILLS, Mich., March 24, 2022 - 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, today announced financial results for the fourth quarter and year ended December 31, 2021 and provided a corporate update.
"2021 proved to be a highly productive year for Ocuphire with 2022 setting up to be a more transformative year given our series of late-stage data read-outs in MIRA,
LYNX and ZETA trials throughout the year, ending with our first planned NDA filing. We have an ambitious vision in ophthalmology targeting highly prevalent refractive and diabetic retinal diseases with our 2 lead small molecule drug candidates,"
said Mina Sooch, MBA, Founder and CEO of Ocuphire Pharma. "We are pleased to rapidly exceed enrollment in and complete 4 clinical trials across Nyxol and APX3330 in the first months of 2022. At our R&D Day in January, we reported for the
first time positive Phase 2 results in presbyopia for Nyxol as a single-agent. With this new chronic opportunity for Nyxol alone as a pupil modulation agent, we can potentially realize synergies in presbyopia and NVD patients. We recently held a
FDA Type-C meeting and gained clear guidance for the VEGA Phase 3 presbyopia program, for which we plan to initiate in mid-2022. With the successful enrollment of the 24-week study for our retinal candidate APX3330 and the continued favorable
systemic and ocular safety profile that we shared at our recent R&D Day, we are also excited to lead the retinal landscape with an oral option for diabetic retinopathy patients and report our topline data from our placebo-controlled,
double-masked, Phase 2b ZETA-1 trial in the second half of 2022."
Jay Pepose, MD, PhD, Ocuphire's Medical Advisor and Board Member stated, "Ocuphire's product candidates, if approved, would give eye care practitioners the ability to
enhance their patients' vision and overall experience. As a refractive surgeon, I am particularly excited about Nyxol for RM because there is currently no FDA approved commercially available product to treat this major clinical need and patient
complaint. For presbyopia, I am impressed by Nyxol's favorable tolerability profile and durable near vision improvements for at least 12 hours across a broad age of patients (40 to 64 years old) in the recent VEGA-1 Phase 2 trial. Nyxol is
differentiated from the other miotics in the presbyopia landscape by its mechanism of action inhibiting the iris dilator muscle to achieve an optimal pupil size. Since Nyxol does not engage the iris sphincter or ciliary muscle, as a single drop,
this may become a viable treatment option for presbyopia patients with high myopia, for whom miotics are contraindicated because of the risk of retinal detachment."
Cam Gallagher, Chairman of the Board for Ocuphire added, "In the past year, Ocuphire has elevated its profile within the ophthalmology and optometry medical community
and we are delighted to have expanded our prestigious Medical Advisory Board to over 15 refractive and retinal KOLs. The team led by Mina has executed and delivered on several key clinical development milestones and set the momentum for a
catalyst-rich 2022 that has the potential to build significant value for our company and shareholders."
Key Anticipated Future Milestones
Fourth Quarter and Recent Business Highlights
Clinical Development
Presentations, Publications, and Conferences
Fourth Quarter and Year Ended December 31, 2021 Financial Highlights
As of December 31, 2021, the Company had cash and cash equivalents of approximately $24.5 million. Based on current projections, management believes the current cash on
hand will be sufficient to fund operations into the second quarter of 2023. Net cash used in operating activities for the quarter and year ended December 31, 2021 was $5.6 million and $19.4 million, respectively.
No collaboration revenue was recorded in the fourth quarter. Collaborative revenue was $0.6 million for the year ended December 31, 2021. Revenue was derived from the
collaboration and license agreements with Processa and Biosense related to certain Rexhan products and technology transfers. There was no collaboration revenue recognized during the comparable prior year periods.
General and administrative expenses for the quarter and year ended December 31, 2021 were $1.4 million and $8.1 million, respectively, compared to $1.3 million and $2.8
million for the quarter and year ended December 31, 2020, respectively. The $5.3 million increase for the year over year periods was primarily attributable to administrative employee headcount, stock-based compensation, insurance, legal and
settlement costs, costs associated with operating as a public company subsequent to the reverse merger, and professional services and other operating costs. General and administrative expenses included $0.3 million and $0.2 million in non-cash
stock-based compensation expense during the quarters ended December 31, 2021 and 2020, respectively, and $1.1 million and $0.7 million in non-cash stock-based compensation expense during the years ended December 31, 2021 and 2020, respectively.
Research and development expenses for the quarter and year ended December 31, 2021 were $4.7 million and $15.2 million, respectively, compared to $4.3 million and $6.6
million for the quarter and year ended December 31, 2020, respectively. The $8.5 million increase for the year over year periods was primarily attributable to clinical trial expense, manufacturing activities to support clinical advancement of
Nyxol and APX3330, consulting services as well as regulatory and other research and development efforts. Research and development expenses also included $0.2 million and $0.3 million in non-cash stock-based compensation expense during the
quarters ended December 31, 2021 and 2020, respectively, and $0.8 million in non-cash stock-based compensation expense during each of the years ended December 31, 2021 and 2020.
The loss from operations for the quarter ended December 31, 2021 was $6.2 million, compared to $14.0 million for the quarter ended December 31, 2020. The loss from
operations for the year ended December 31, 2021 was $22.7 million, compared to $20.0 million for the year ended December 31, 2020. Net loss for the quarter ended December 31, 2021 was $6.3 million, compared to $18.7 million for the quarter ended
December 31, 2020. Net loss for the year ended December 31, 2021 was $56.7 million, compared to $24.6 million for the year ended December 31, 2020. Net loss per share for the quarter ended December 31, 2021 was $0.35, compared to $2.46 for the
quarter ended December 31, 2020. Net loss per share for the year ended December 31, 2021 was $3.82, compared to $5.28 for the year ended December 31, 2020.
The fair value change in derivative and warrant liabilities was a non-cash expense of zero for the quarter ended December 31, 2021 compared to a non-cash expense of
$1.6 million for the quarter ended December 31, 2020. The fair value change in derivative and warrant liabilities was a non-cash expense of $33.8 million for the year ended December 31, 2021 compared to a non-cash expense of $1.5 million for the
year ended December 31, 2020.
For further details on Ocuphire's financial results, refer to the Company's Annual Report on Form 10-K for the year ended December 31, 2021 to be filed with the
Securities and Exchange Commission.
About Ocuphire Pharma
Ocuphire is a publicly-traded (NASDAQ: OCUP), clinical-stage ophthalmic biopharmaceutical company focused on developing and commercializing therapies for the treatment of
several eye disorders. Ocuphire's pipeline currently includes two small-molecule product candidates targeting refractive and retinal indications. The company's lead product candidate, Nyxol eye drops (0.75% phentolamine ophthalmic
solution) is a once-daily, preservative-free eye drop formulation of phentolamine mesylate, a non-selective alpha-1 and alpha-2 adrenergic antagonist designed to reduce pupil size, and is being developed for several indications, including reversal
of pharmacologically-induced mydriasis (RM), presbyopia and dim light or night vision disturbances (NVD), and has been studied in 9 completed clinical trials. Ocuphire reported positive top-line data for MIRA-2, the first Phase 3 registration trial
for the treatment of RM, and recently initiated and completed enrollment in the second Phase 3 registration trial (MIRA-3) and pediatric safety trial (MIRA-4) in RM. Ocuphire also reported positive top-line data from a Phase 2 trial of Nyxol for
treatment of presbyopia, both Nyxol as a single agent and Nyxol with low dose pilocarpine ("LDP") 0.4% as adjunctive therapy. The company recently completed enrollment in its Phase 3 study of Nyxol for NVD (LYNX-1). Ocuphire's second product
candidate, APX3330, is an oral tablet designed to inhibit angiogenesis and inflammation pathways relevant to retinal and choroidal vascular diseases, such as diabetic retinopathy (DR) and diabetic macular edema (DME) and has been studied in 11
Phase 1 and 2 trials. The company recently announced the completion of enrollment in a Phase 2b clinical trial of APX3330 to treat DR/DME (ZETA-1). Please visit www.clinicaltrials.gov to learn more about Ocuphire's recently enrolled second Phase 3
registration trial in RM (NCT05134974), MIRA-4 pediatric safety study in RM (NCT05223478), Phase 3 registration trial in NVD (NCT04638660), and Phase 2b trial in DR/DME (NCT04692688). Ocuphire previously completed the first Phase 3 registration
trial in RM (NCT04620213) and Phase 2 trial in presbyopia (NCT04675151). As part of its strategy, Ocuphire will continue to explore opportunities to acquire additional ophthalmic assets and to seek strategic partners for late-stage development,
regulatory preparation, and commercialization of drugs in key global markets. For more information, visit www.ocuphire.com.
Forward Looking Statements
Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities
Litigation Reform Act of 1995. Such statements include, but are not limited to, timing and results in RM, presbyopia, NVD and DR/DME future clinical trials as well as statements concerning the success and timing of planned regulatory filings and
commercialization. These forward-looking statements are based upon Ocuphire's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially
from those anticipated in such forward-looking statements as a result of various risks and uncertainties, including, without limitation: (i) the success and timing of regulatory submissions and pre-clinical and clinical trials, including
enrollment and data readouts; (ii) regulatory requirements or developments; (iii) changes to clinical trial designs and regulatory pathways; (iv) changes in capital resource requirements; (v) risks related to the inability of Ocuphire to obtain
sufficient additional capital to continue to advance its product candidates and its preclinical programs; (vi) legislative, regulatory, political and economic developments, (vii) changes in market opportunities, (viii) the effects of COVID-19 on
clinical programs and business operations, (ix) the success and timing of commercialization of any of Ocuphire's product candidates and (x) the maintenance of Ocuphire's intellectual property rights. The foregoing review of important factors that
could cause actual events to differ from expectations should not be construed as exhaustive and should be read in conjunction with statements that are included herein and elsewhere, including the risk factors detailed in documents that have been
and may be filed by Ocuphire from time to time with the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Ocuphire undertakes no obligation to update such statements to reflect
events that occur or circumstances that exist after the date on which they were made.
Mina Sooch, President & CEO
Ocuphire Pharma, Inc.
Ocuphire Pharma, Inc.
Condensed Consolidated Balance Sheets
(in thousands, except share amounts and par value)
As of December 31,
2021 2020
Assets
Current assets:
Cash and cash equivalents $ 24,534 $ 16,399
Prepaids and other assets 1,314 1,269
Short-term investments 219 -
Total current assets 26,067 17,668
Property and equipment, net 10 14
Total assets $ 26,077 $ 17,682
Liabilities and stockholders' equity (deficit)
Current liabilities:
Accounts payable $ 1,584 $ 1,214
Accrued expenses 1,733 1,971
Short-term loan 538 -
Total current liabilities 3,855 3,185
Warrant liabilities - 27,964
Total liabilities 3,855 31,149
Commitments and contingencies
Stockholders' equity (deficit)
Preferred stock, par value $0.0001; 10,000,000 shares authorized as of December 31, 2021 and 2020; no shares issued and outstanding at December 31, 2021 and 2020. - -
Common stock, par value $0.0001; 75,000,000 shares authorized as of December 31, 2021 and 2020; 18,845,828 and 10,882,495 shares issued and outstanding at December 31, 2021 and 2020, respectively. 2 1
Additional paid-in capital 111,588 19,207
Accumulated deficit (89,368 ) (32,675 )
Total stockholders' equity (deficit) 22,222 (13,467 )
Total liabilities and stockholders' equity (deficit) $ 26,077 $ 17,682
Ocuphire Pharma, Inc.
Condensed Consolidated Statements of Comprehensive Loss
(in thousands, except share and per share amounts)
For the Quarter Ended December 31, (unaudited) For the Year Ended December 31,
2021 2020 2021 2020
Collaborations revenue $ - $ - $ 589 $ -
Operating expenses:
General and administrative $ 1,414 $ 1,310 $ 8,121 $ 2,818
Research and development 4,736 4,337 15,173 6,648
Acquired in-process research and development - 8,376 - 10,502
Total operating expenses 6,150 14,023 23,294 19,968
Loss from operations (6,150 ) (14,023 ) (22,705 ) (19,968 )
Interest expense (2 ) (5,425 ) (2 ) (6,847 )
Fair value change in derivative and warrant liabilities - (1,644 ) (33,829 ) (1,486 )
Gain on note extinguishment - 2,412 - 3,672
Other (expense) income, net (161 ) - (157 ) 9
Loss before income taxes (6,313 ) (18,680 ) (56,693 ) (24,620 )
Benefit (provision) for income taxes - - - -
Net loss (6,313 ) (18,680 ) (56,693 ) (24,620 )
Other comprehensive loss, net of tax - - - -
Comprehensive loss $ (6,313 ) $ (18,680 ) $ (56,693 ) $ (24,620 )
Net loss per share:
Basic and diluted $ (0.35 ) $ (2.46 ) $ (3.82 ) $ (5.28 )
Number of shares used in per share calculations:
Basic and diluted 17,854,790 7,586,568 14,852,745 4,661,110
Last updated: Mar 24, 2022