Ocuphire Pharma Announces Financial Results for First Quarter 2024 and Provides Corporate Update
Ocuphire Pharma, a clinical-stage ophthalmic biopharmaceutical company, announced financial results for Q1 2024, highlighting progress with APX3330 and RYZUMVI™. The company is in dialogue with the FDA regarding APX3330 for diabetic retinopathy. RYZUMVI™, launched by partner Viatris, marks a significant milestone. Ocuphire also disclosed updates on APX3330 and Phentolamine Ophthalmic Solution. Financially, Ocuphire had $47.2 million in cash as of March 31, 2024, with positive revenue from collaborations and royalties but faced increased expenses.
Progress with APX3330 and RYZUMVI™ development
Dialogue with FDA for APX3330 in diabetic retinopathy
Successful commercial launch of RYZUMVI™ by Viatris
Positive clinical and regulatory updates for APX3330
Initiation of Phase 3 study for Phentolamine Ophthalmic Solution
Appointment of new CFO and Chief Scientific Officer
Healthy cash position of $47.2 million as of March 31, 2024
Positive revenue from collaborations and royalties
Increased general and administrative expenses
Net loss of $7.1 million for the quarter ended March 31, 2024
Decrease in research and development expenses compared to Q1 2023
Challenges in managing operating costs efficiently
Insights
Assessing Ocuphire Pharma's financial performance for the first quarter of 2024, the consistency in license and collaborations revenue year-over-year stands out. The reported
The net loss widening from
With royalty payments just beginning to trickle in, the company’s long-term revenue potential remains a key area to watch, particularly as Ocuphire's commercial agreements and clinical results mature.
The progress and dialogue with the U.S. FDA regarding APX3330's Special Protocol Assessment (SPA) suggests a strategic regulatory path is being carved out, a positive signal for investors interested in Ocuphire's commitment to bringing treatments to market. The focus on diabetic retinopathy (DR), an area with significant unmet medical needs, highlights the potential market opportunity for APX3330 if approval is granted. However, it's important to acknowledge that success in clinical trials is never guaranteed and the outcome will significantly affect the company's valuation.
Moreover, the commercial launch of RYZUMVI™ in partnership with Viatris is a critical development. This marks Ocuphire's transition into a revenue-generating phase, albeit at an early stage. The pursuit of additional indications for phentolamine ophthalmic solution could broaden the therapy’s market reach and drive future revenue growth.
The appointment of new executives with financial and scientific acumen suggests an organizational strengthening, necessary for navigating the complex landscape of drug development and commercialization.
Ocuphire Pharma's first royalty payment from sales of RYZUMVI™, despite being a modest
The engagement in Phase 2/3 registration studies and the submission of an SPA indicate the company is advancing in its clinical development pipeline. This is a vital phase for biopharmaceutical companies as it precedes potential commercialization. Additionally, the early enrollment in the LYNX-2 Phase 3 trial demonstrates ongoing momentum in their research activities.
Investors often view active and advancing pipelines as a positive sign, although the true impact on stock performance is inherently tied to the outcomes of these studies and eventual market penetration of these products.
FARMINGTON HILLS, Mich., May 10, 2024 (GLOBE NEWSWIRE) -- Ocuphire Pharma, Inc. (Nasdaq: OCUP), a clinical-stage ophthalmic biopharmaceutical company focused on developing small molecule therapies for the treatment of patients with retinal and refractive eye disorders, today announced financial results for the first quarter ended March 31, 2024, and provided a corporate update.
“Ocuphire has made significant progress in 2024, with important developments for both APX3330 and RYZUMVI™,” said George Magrath, M.D., M.B.A., M.S., CEO of Ocuphire. “We have been engaged in productive dialogue with the U.S. Food and Drug Administration (FDA) with respect to our submitted Special Protocol Assessment (SPA) to formalize the protocol and statistical analysis plan for future Phase 2/3 registrational trials of APX3330 in diabetic retinopathy (DR). If APX3330 is approved, we believe it could be a promising oral treatment option for slowing disease progression in patients with non-proliferative DR who otherwise are monitored and untreated until they progress to sight-threatening disease. The recent commercial launch of RYZUMVI™ by our partner Viatris, Inc. (Viatris) (NASDAQ: VTRS) was a major milestone, and an important validation of the clinical development work conducted by the Ocuphire team over the past several years to advance this product and secure FDA approval. Viatris now has the opportunity to create further value as it pursues additional indications for phentolamine ophthalmic solution, including the treatment of decreased low contrast visual acuity under low light conditions as well as presbyopia.”
Clinical and Regulatory Updates
APX3330
- In February 2024, Ocuphire submitted a Special Protocol Assessment (SPA) to the U.S. Food and Drug Administration (FDA) to seek agreement on the clinical trial protocol and statistical analysis plan for a Phase 2/3 registration study for APX3330 in DR. This request followed an End-of-Phase 2 meeting held with the FDA late in 2023, during which the company aligned on the registrational primary endpoint of 3-step or more worsening on a binocular Diabetic Retinopathy Severity Scale (DRSS) person-level scale. Dialogue with the FDA is ongoing, and specifics on the study design and the anticipated timing will be announced if and when an agreement is reached with the FDA.
- Earlier this month, a subset analysis from the ZETA-1 trial was presented at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting, which took place in Seattle, Washington. This analysis evaluated the efficacy of APX3330 in slowing DR progression on the target population of the planned Phase 2/3 study, and the FDA agreed upon registration endpoint of a 3-step change on a binocular DRSS person-level scale, which is considered clinically meaningful. The presented results in addition to further analyses demonstrated an enhanced treatment effect for APX3330 in this target population.
- In conjunction with this update, a subpopulation analysis of ZETA-1 data in NPDR will be released.
Phentolamine Ophthalmic Solution
- In April 2024, Ocuphire’s partner Viatris launched RYZUMVI™ (phentolamine ophthalmic solution) for the treatment of pharmacologically-induced mydriasis in the U.S. in adult and pediatric patients aged 3 and older. Ocuphire has a global license agreement with Viatris to co-develop and commercialize Phentolamine Ophthalmic Solution
0.75% (PS). Under the terms of this agreement, Ocuphire is now recognizing royalties on commercial sales and may be eligible to receive future commercial milestones. For more information on the commercial launch, refer to the announcement on Viatris’ corporate website here. - In April 2024, the first subject was enrolled in the LYNX-2 Phase 3 registration study evaluating PS for the treatment of decreased visual acuity under low (mesopic) light conditions following keratorefractive surgery. The LYNX-2 trial is being conducted under conditions of a SPA with the FDA. As previously announced, Ocuphire received written agreement from the FDA that the clinical trial protocol and planned statistical analysis of the LYNX-2 Phase 3 trial would adequately address objectives supporting regulatory submission and a potential future marketing application in this indication.
Corporate Updates
- In February 2024, Ocuphire appointed Nirav Jhaveri, C.F.A, M.B.A., as Chief Financial Officer and Ashwath (Ash) Jayagopal, Ph.D., M.B.A., as Chief Scientific and Development Officer.
Financial Highlights for the First Quarter Ended March 31, 2024
As of March 31, 2024, Ocuphire had cash and cash equivalents of
License and collaborations revenue was
General and administrative expenses for the first quarter ended March 31, 2024 were
Research and development expenses for the first quarter ended March 31, 2024 were
Net loss for the quarter ended March 31, 2024 was
For further details on Ocuphire’s financial results, refer to the Company’s Quarterly Report on Form 10-Q to be filed with the Securities and Exchange Commission.
About Ocuphire Pharma
Ocuphire is a clinical-stage ophthalmic biopharmaceutical company focused on developing novel therapies for the treatment of retinal and refractive eye disorders.
Ocuphire’s lead retinal product candidate, APX3330, is an oral small-molecule inhibitor of Ref-1 (reduction oxidation effector factor-1 protein) for the treatment of non-proliferative diabetic retinopathy (NPDR). Ref-1 is a regulator of the transcription factors HIF-1α and NF-κB. Inhibiting REF-1 reduces levels of vascular endothelial growth factor (VEGF) and inflammatory cytokines which are known to play key roles in ocular angiogenesis and inflammation. APX3330 is an oral tablet to be administered twice per day for the treatment of diabetic retinopathy (DR). A Phase 2 study in subjects with DR and an End-of-Phase 2 meeting have been completed, and a SPA was submitted to the U.S. Food and Drug Administration (FDA) in February 2024 and active discussions continue with the agency.
In addition, Ocuphire has a partnership with Viatris to develop and commercialize PS, a non-selective alpha-1 and alpha-2 adrenergic antagonist designed to reduce pupil size. PS was approved by the FDA for the treatment for pharmacologically-induced mydriasis under the brand name RYZUMVI™ in September 2023. PS is also in Phase 3 clinical development for the treatment of presbyopia and for the treatment of decreased visual acuity under low light (mesopic) conditions after keratorefractive surgery.
Ocuphire is also developing APX2009 and APX2014, second-generation analogs of APX3330. These programs are being evaluated for treating other retinal diseases such as age-related macular degeneration and geographic atrophy. For more information, please visit www.ocuphire.com.
Forward Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, statements concerning the research takeaways from the ZETA-1 trial, the efficacy of APX3330 in slowing the progression of diabetic retinopathy, the safety and tolerability of APX3330, applications of PS in ophthalmology, the registration program for PS, the LYNX-2 Phase 3 registration study, the benefits, uses, and side effects of PS treatment, ongoing discussions with the FDA regarding various of our drug products, continued drug development under our agreement with Viatris, and the sufficiency of cash on hand to meet future funding needs.
These forward-looking statements relate to us, our business prospects and our results of operations and are subject to certain risks and uncertainties posed by many factors and events that could cause our actual business, prospects and results of operations to differ materially from those anticipated by such forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to, those described under the heading “Risk Factors” included in our Annual Report on Form 10-K and in subsequent filing with the U.S. Securities and Exchange Commission (SEC). Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this report. In some cases, you can identify forward-looking statements by the following words: “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “ongoing,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. We undertake no obligation to revise any forward-looking statements in order to reflect events or circumstances that might subsequently arise.
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:
- The success and timing of regulatory submissions and pre-clinical and clinical trials, including enrollment and data readouts;
- Regulatory requirements or developments;
- Changes to or unanticipated events in connection with clinical trial designs and regulatory pathways;
- Delays or difficulties in the enrollment of patients in clinical trials;
- Substantial competition and rapid technological change;
- Our development of sales and marketing infrastructure;
- Future revenue losses and profitability;
- Our relatively short operating history;
- Changes in capital resource requirements;
- Risks related to the inability of Ocuphire to obtain sufficient additional capital to continue to advance its product candidates and its preclinical programs;
- Domestic and worldwide legislative, regulatory, political and economic developments;
- Employee misconduct;
- Changes in market opportunities and acceptance;
- Reliance on third-parties;
- Future, potential product liability and securities litigation;
- System failures, unplanned events, or cyber incidents;
- The substantial number of shares subject to potential issuance associated with our equity line of credit arrangement;
- Risks that our partnership with Viatris, or our other licensing arrangements, may not facilitate the commercialization or market acceptance of Ocuphire’s product candidates;
- Future fluctuations in the market price of our common stock;
- The success and timing of commercialization of any of Ocuphire’s product candidates; and
- Obtaining and maintaining 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. Readers are urged to carefully review and consider the various disclosures made by us in this report and in our other reports filed with the SEC that advise interested parties of the risks and factors that may affect our business. 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.
Contacts
| Corporate | Investor Relations |
| Nirav Jhaveri, M.B.A. CFO ir@ocuphire.com | Corey Davis, Ph.D. LifeSci Advisors cdavis@lifesciadvisors.com |
| Ocuphire Pharma, Inc. Condensed Balance Sheets (in thousands, except share amounts and par value) | ||||||||
| As of | ||||||||
| March 31, | December 31, | |||||||
| 2024 | 2023 | |||||||
| Assets | (unaudited) | |||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 47,161 | $ | 50,501 | ||||
| Accounts receivable | 1,924 | 926 | ||||||
| Contract assets and unbilled receivables | 1,194 | 1,407 | ||||||
| Prepaids and other assets | 1,560 | 1,099 | ||||||
| Short-term investments | 5 | 15 | ||||||
| Total current assets | 51,844 | 53,948 | ||||||
| Property and equipment, net | — | — | ||||||
| Total assets | $ | 51,844 | $ | 53,948 | ||||
| Liabilities and stockholders’ equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 2,064 | $ | 2,153 | ||||
| Accrued expenses | 3,649 | 1,815 | ||||||
| Derivative liability | 74 | 74 | ||||||
| Total current liabilities | 5,787 | 4,042 | ||||||
| Total liabilities | 5,787 | 4,042 | ||||||
| Commitments and contingencies | ||||||||
| Stockholders’ equity: | ||||||||
| Preferred stock, par value | — | — | ||||||
| Common stock, par value | 3 | 2 | ||||||
| Additional paid-in capital | 134,626 | 131,370 | ||||||
| Accumulated deficit | (88,572 | ) | (81,466 | ) | ||||
| Total stockholders’ equity | 46,057 | 49,906 | ||||||
| Total liabilities and stockholders’ equity | $ | 51,844 | $ | 53,948 | ||||
| Ocuphire Pharma, Inc. Condensed Statements of Comprehensive Loss (in thousands, except share and per share amounts) (Unaudited) | |||||||
| Three Months Ended | |||||||
| March 31, | |||||||
| 2024 | 2023 | ||||||
| License and collaborations revenue | $ | 1,711 | $ | 1,749 | |||
| Operating expenses: | |||||||
| General and administrative | 4,670 | 2,285 | |||||
| Research and development | 4,749 | 5,595 | |||||
| Total operating expenses | 9,419 | 7,880 | |||||
| Loss from operations | (7,708 | ) | (6,131 | ) | |||
| Fair value change in derivative liabilities | — | — | |||||
| Other income, net | 602 | 340 | |||||
| Loss before income taxes | (7,106 | ) | (5,791 | ) | |||
| Benefit (provision) for income taxes | — | — | |||||
| Net loss | (7,106 | ) | (5,791 | ) | |||
| Other comprehensive loss, net of tax | — | — | |||||
| Comprehensive loss | $ | (7,106 | ) | $ | (5,791 | ) | |
| Net loss per share: | |||||||
| Basic and diluted | $ | (0.29 | ) | $ | (0.28 | ) | |
| Number of shares used in per share calculations: | |||||||
| Basic and diluted | 24,520,475 | 20,939,607 | |||||
FAQ
<p>What important updates did Ocuphire Pharma provide?</p>
Ocuphire announced financial results for Q1 2024, with progress on APX3330 and RYZUMVI™ development. The company also discussed updates on clinical and regulatory matters.
<p>What is the financial status of Ocuphire Pharma as of March 31, 2024?</p>
As of March 31, 2024, Ocuphire had $47.2 million in cash and positive revenue from collaborations and royalties.
<p>What appointment was made by Ocuphire Pharma in February 2024?</p>
Ocuphire appointed Nirav Jhaveri as Chief Financial Officer and Ashwath Jayagopal as Chief Scientific and Development Officer in February 2024.
<p>What challenges did Ocuphire Pharma face in Q1 2024?</p>
Ocuphire experienced increased general and administrative expenses, resulting in a net loss of $7.1 million for the quarter ended March 31, 2024.
