Ocuphire Pharma Announces Financial Results for Third Quarter 2023 and Provides Corporate Update
- Successful End-of-Phase 2 meeting with FDA for oral APX3330 in diabetic retinopathy, leading to agreement on Phase 3 primary endpoint.
- FDA approval of RYZUMVI™ for the treatment of pharmacologically induced mydriasis, resulting in a $10 million milestone payment.
- VEGA-2 Phase 3 trial for presbyopia achieved its primary endpoint, with Viatris expected to continue Phase 3 development.
- George Magrath named as CEO, bringing in strong leadership to drive the company forward.
- Financial report for Q3 2023 shows $42.4 million cash and cash equivalents, with sufficient funds to support operations into 2025.
- None.
Successful End-of-Phase 2 Meeting with FDA for Oral APX3330; Agreement on Phase 3 Registration Endpoint
RYZUMVI™ Approved by FDA; Ocuphire Received
VEGA-2 Phase 3 Presbyopia Trial Met Primary Endpoint; Viatris Expected to Continue Phase 3 Development in 1H 2024
George Magrath, M.D., M.B.A., M.S. Named as CEO; Conference Call Scheduled for December 5th, 2023, at 10 AM ET
FARMINGTON HILLS, Mich., Nov. 13, 2023 (GLOBE NEWSWIRE) -- Ocuphire Pharma, Inc. (Nasdaq: OCUP), a clinical-stage ophthalmic biopharmaceutical company focused on developing and commercializing small-molecule therapies for the treatment of retinal and refractive eye disorders, today announced financial results for the third quarter ended September 30, 2023 and provided a corporate update.
“We achieved important regulatory and clinical milestones in recent months, particularly in advancing oral APX3330 towards a registrational Phase 3 program in diabetic retinopathy,” said Chief Executive Officer George Magrath, M.D., M.B.A., M.S., “We now have agreement with the FDA on the Phase 3 primary endpoint of 3-step worsening (measuring delay or prevention of progression) on a binocular Diabetic Retinopathy Severity Scale, and plan to finalize the protocol and statistical analysis plan with the FDA through a Special Protocol Assessment submission. If approved, APX3330 has the potential to be the first non-invasive, early treatment to delay or prevent vision-threatening complications in millions of patients with non-proliferative DR. We are very pleased to have recently received FDA approval of RYZUMVI™ (phentolamine ophthalmic solution)
Key Anticipated Future Milestones
- APX3330: Special Protocol Assessment (“SPA”) submission and agreement with U.S. Food and Drug Administration (“FDA”)
Recent Business Highlights
Clinical and Regulatory Updates
- In October 2023, Ocuphire had a successful End-of-Phase 2 (“EOP2”) meeting with the FDA for oral APX3330 in Diabetic Retinopathy and agreed on the Phase 3 primary endpoint of 3-step worsening (measuring delay or prevention of progression) on a binocular diabetic retinopathy severity scale. The Company plans to submit a SPA to the FDA to agree on the protocol and statistical analysis plan of the first Phase 3 trial.
- In September 2023, Ocuphire and Viatris announced FDA approval of RYZUMVI™ (phentolamine ophthalmic solution)
0.75% for the treatment of pharmacologically induced mydriasis produced by adrenergic agonists (e.g., phenylephrine) or parasympatholytic agents (e.g., tropicamide). Ocuphire received a$10 million milestone payment from Viatris upon the approval. RYZUMVI is expected to be commercially available in the U.S. in the first half of 2024. - The VEGA-2 Phase 3 study evaluating phentolamine ophthalmic solution
0.75% in presbyopia achieved its primary endpoint. Viatris is expected to continue Phase 3 development for this indication in the first half of 2024. - The Company has submitted a SPA to the FDA for night vision disturbances or dim light vision and Viatris is expected to continue Phase 3 development of phentolamine ophthalmic solution
0.75% for this indication in the first half of 2024.
Corporate
On August 10, 2023, Ocuphire entered into a common share purchase agreement with Lincoln Park Capital Fund, LLC (“LPC”). Subject to the terms and conditions of the purchase agreement, Ocuphire has the right to sell, and LPC is obligated to purchase, up to
Presentations, Publications, and Conferences
Beginning in the third quarter of 2023, several presentations at medical meetings featured previously announced data from the ZETA-1 Phase 2 trial of APX3330 in diabetic retinopathy. Highlights include:
- A presentation at the Eyecelerator American Academy of Ophthalmology 2023 Retina Showcase by Jay Pepose, M.D. Ph.D., Chief Medical Advisor, highlighting the agreement of Phase 3 registration endpoint at the EOP2 FDA meeting.
- A paper presentation delivered by Veeral Sheth, M.D., at the 23rd Euretina Congress in October 2023.
- An oral presentation delivered by Anat Lowenstein, M.D., at the 56th Annual Retina Society Scientific Meeting in October 2023.
- An oral presentation delivered by Priya Vakharia, M.D., at the Women in Ophthalmology Summer Symposium in August 2023. The abstract was rated as one of the top three scoring abstracts out of nearly 600 submissions and received the Joanne Angle Abstract of Distinction Award.
Third Quarter ended September 30, 2023, Financial Highlights
As of September 30, 2023, Ocuphire had cash and cash equivalents of approximately
License and collaborations revenue was
General and administrative expenses for the three and nine months ended September 30, 2023, were
Research and development expenses for the three and nine months ended September 30, 2023, were
Income (loss) from operations for the three and nine months ended September 30, 2023, was
Net income (loss) for the three and nine months ended September 30, 2023, was
For further details on Ocuphire’s financial results, refer to the Company’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2023, to be filed with the Securities and Exchange Commission.
Conference Call and Webcast Details:
Date: December 5th, 2023
Time: 10:00 AM ET
Dial-in information: 1-877-407-4018 (US); 1-201-689-8471 (International); Call me™
Passcode: 13742669
Webcast link
Participants can use Guest dial-in #s above and be answered by an operator OR click the Call me™ link for instant telephone access to the event. Call me™ link will be made active 15 minutes prior to scheduled start time.
About Ocuphire Pharma
Ocuphire Pharma, Inc. is a clinical-stage ophthalmic biopharmaceutical company focused on developing and commercializing small-molecule therapies for the treatment of retinal and refractive eye disorders.
Ocuphire’s lead retinal product candidate, APX3330, is a first-in-class small-molecule inhibitor of Ref-1 (reduction oxidation effector factor-1 protein). Ref-1 is a regulator of the transcription factors HIF-1a and NF-kB. 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. Through inhibition of Ref-1, APX3330 normalizes the levels of VEGF to physiologic levels, unlike biologics that deplete VEGF below the levels required for normal function. 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 recently been completed, and a Special Protocol Assessment is planned to be submitted with the U.S. Food and Drug Administration (FDA).
DR affects approximately 10 million people with diabetes and is projected to impact over 14 million Americans by 2050. DR is classified as Non-Proliferative Diabetic Retinopathy (“NPDR”), the early stage of the disease in which symptoms may be mild or non-existent or Proliferative Diabetic Retinopathy (“PDR”) which is the more advanced stage of diabetic eye disease that can be highly symptomatic with loss of vision. Approximately
Ocuphire has also in-licensed APX2009 and APX2014, which are second-generation analogs of APX3330. The unique dual mechanism of action of these Ref-1 inhibitors of reducing angiogenesis and inflammation could potentially be beneficial in treating other retinal diseases such as age-related macular degeneration, and geographic atrophy. Ocuphire is currently evaluating local delivery routes in addition to the systemic (oral) route as part of its pipeline expansion in retinal therapies.
Ocuphire has a partnership with Viatris, Inc. to develop and commercialize phentolamine ophthalmic solution
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, statements concerning the End-of-Phase 2 meeting with the FDA to confirm Phase 3 registration endpoints, study parameters for Phase 3 pivotal studies, Phase 3 development, FDA agreement on Special Protocol Assessment, the potential for APX330 to be the first non-invasive, early treatment to delay or prevent progression to vision-threatening complications, ability to fund operations into 2025, and the commercialization of RYZUMVI™. 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) risks that the phentolamine ophthalmic solution partnership may not facilitate the commercialization or market acceptance of Ocuphire’s product candidates; (x) the success and timing of commercialization of any of Ocuphire’s product candidates and (xi) 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.
Contacts
| Corporate | Investor Relations |
| George Magrath, M.D., M.B.A., M.S. CEO 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 | ||||||||
| September 30, 2023 (unaudited) | December 31, 2022 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 42,350 | $ | 42,634 | ||||
| Accounts receivable | 10,132 | 1,298 | ||||||
| Contract assets and unbilled receivables | 1,211 | 3,552 | ||||||
| Prepaids and other current assets | 484 | 1,453 | ||||||
| Short-term investments | 11 | 49 | ||||||
| Total current assets | 54,188 | 48,986 | ||||||
| Property and equipment, net | 3 | 6 | ||||||
| Total assets | $ | 54,191 | $ | 48,992 | ||||
| Liabilities and stockholders’ equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 1,890 | $ | 1,069 | ||||
| Accrued expenses | 1,926 | 1,684 | ||||||
| Derivative liability | 93 | — | ||||||
| Total current liabilities | 3,909 | 2,753 | ||||||
| Total liabilities | 3,909 | 2,753 | ||||||
| Commitments and contingencies | ||||||||
| Stockholders’ equity | ||||||||
| Preferred stock, par value | — | — | ||||||
| Common stock, par value | 2 | 2 | ||||||
| Additional paid-in capital | 126,951 | 117,717 | ||||||
| Accumulated deficit | (76,671 | ) | (71,480 | ) | ||||
| Total stockholders’ equity | 50,282 | 46,239 | ||||||
| Total liabilities and stockholders’ equity | $ | 54,191 | $ | 48,992 | ||||
| Ocuphire Pharma, Inc. Condensed Statements of Comprehensive Income (Loss) (in thousands, except share and per share amounts) (Unaudited) | |||||||||||||||||
| For the Three Months Ended September 30, | For the Nine Months Ended September 30, | ||||||||||||||||
| 2023 | 2022 | 2023 | 2022 | ||||||||||||||
| License and collaborations revenue | $ | 11,935 | $ | — | $ | 17,358 | $ | — | |||||||||
| Operating expenses: | |||||||||||||||||
| General and administrative | 2,055 | 1,703 | 8,680 | 5,215 | |||||||||||||
| Research and development | 3,494 | 2,835 | 13,812 | 10,769 | |||||||||||||
| Total operating expenses | 5,549 | 4,538 | 22,492 | 15,984 | |||||||||||||
| Income (loss) from operations | 6,386 | (4,538 | ) | (5,134 | ) | (15,984 | ) | ||||||||||
| Financing costs | (1,328 | ) | — | (1,328 | ) | — | |||||||||||
| Interest expense | — | — | — | (9 | ) | ||||||||||||
| Fair value change in derivative liability | 61 | — | 61 | — | |||||||||||||
| Other income (expense), net | 456 | 7 | 1,224 | (60 | ) | ||||||||||||
| Income (loss) before income taxes | 5,575 | (4,531 | ) | (5,177 | ) | (16,053 | ) | ||||||||||
| Provision for income taxes | (14 | ) | — | (14 | ) | — | |||||||||||
| Net income (loss) | 5,561 | (4,531 | ) | (5,191 | ) | (16,053 | ) | ||||||||||
| Other comprehensive income (loss), net of tax | — | — | — | — | |||||||||||||
| Comprehensive income (loss) | $ | 5,561 | $ | (4,531 | ) | $ | (5,191 | ) | $ | (16,053 | ) | ||||||
| Net income (loss) per share: | |||||||||||||||||
| Basic | $ | 0.26 | $ | (0.22 | ) | $ | (0.25 | ) | $ | (0.82 | ) | ||||||
| Diluted | $ | 0.25 | $ | (0.22 | ) | $ | (0.25 | ) | $ | (0.82 | ) | ||||||
| Number of shares used in per share calculations: | |||||||||||||||||
| Basic | 21,446,648 | 20,498,229 | 21,117,211 | 19,635,651 | |||||||||||||
| Diluted | 22,405,995 | 20,498,229 | 21,117,211 | 19,635,651 | |||||||||||||
FAQ
What are the recent FDA-related milestones achieved by Ocuphire Pharma, Inc. (OCUP)?
What is the significance of the VEGA-2 Phase 3 trial for presbyopia?
Who is the new CEO of Ocuphire Pharma, Inc.?
What are the financial highlights from the Q3 2023 report of Ocuphire Pharma, Inc.?
