Full Press Release Details
Opus Genetics Announces Financial Results for Third Quarter 2024 and Provides Corporate Update
In October, Ocuphire Pharma acquired Opus Genetics, creating a leading, clinical-stage company focused on the development of gene therapy
treatments for rare inherited retinal diseases (IRDs)
The pro forma cash balance of the combined company was approximately $37 million as of September 30, 2024 (preliminary and unaudited), expected
to extend runway into 2026
Four clinical data readouts expected in 2025
FARMINGTON HILLS, Mich., November 12, 2024 (GLOBE NEWSWIRE) - Opus Genetics, Inc. (Nasdaq: IRD), a clinical-stage ophthalmic biotechnology company developing gene
therapies for the treatment of inherited retinal diseases (IRDs) and other ophthalmologic disorders, today announced financial results for the third quarter ended September 30, 2024 and
provided a corporate update.
"In October 2024, we acquired Opus Genetics with the goal of creating a leading gene therapy franchise to treat inherited retinal diseases," said George Magrath, M.D.,
now CEO of Opus Genetics. "The transaction expanded our pipeline substantially, adding compelling gene therapy assets. The most advanced of these new candidates, LCA5, has generated positive six-month proof-of-concept data in patients with
advanced disease. A second candidate, OPGx-BEST1 targets one of the largest IRD populations, and we're excited to begin dosing patients
next year. We continue to develop Phentolamine Ophthalmic Solution 0.75% in new indications, and this franchise is expected to provide meaningful potential cashflow in the future, if approved for the new indications. Our expected cash runway has
been extended into 2026, through expected efficacy readouts from four clinical programs in 2025, including Phase 3 studies in two indications for Phentolamine Ophthalmic Solution 0.75% and two
studies for the new gene therapy clinical assets."
Acquisition of Opus Genetics
Phentolamine Ophthalmic Solution 0.75%
Financial Highlights for the Third Quarter Ended September 30, 2024
As of September 30, 2024, Ocuphire had cash and cash equivalents of $37 million. The
pro forma cash balance of the combined company was approximately $37 million as of September 30, 2024 (preliminary and unaudited). Based on current projections, management believes that the cash on hand
will be sufficient to fund operations into 2026.
License and collaborations revenue was $3.9 million and $11.9 million for the three months ended September 30, 2024 and 2023, respectively. Revenue during both
quarterly periods was derived from the License Agreement. Revenue for the three months ended September 30, 2024 was comprised largely of the reimbursement of research and development services. The decrease compared to the corresponding prior year
period was largely due to the one-time achievement of a $10.0 million milestone attributed to the FDA's approval of Phentolamine Ophthalmic Solution 0.75% for reversal of mydriasis in 2023.
Revenue for the three months ended September 30, 2024 also included an earned royalty payment in the amount of $14,000 from the sales of RYZUMVI, indicated for the
treatment of pharmacologically-induced mydriasis by our commercial partner. Until further notice, we will report earned RYZUMVI royalties as a component of revenue listed in the Income Statement.
General and administrative expenses for the three months ended September 30, 2024 were $2.9 million compared to $2.1 million for the three months ended September 30,
2023. The increase period over period was primarily attributable to personnel-related costs, stock-based compensation, legal support costs and business development costs. These were offset in
part by a reduction in non-legal professional service costs. General and administrative expenses included the following noncash items: $0.5 million and $0.3 million in stock-based compensation expense during both three months ended September 30,
2024 and 2023, respectively.
Research and development expenses for the three months ended September 30, 2024 were $9.0 million compared to $3.5 million for the three months ended September 30,
2023. The increase in the current period was primarily attributable to increased clinical costs related to the APX3330 development program and other research and development activities period over period, drug manufacturing costs and toxicology
service costs related to APX3330, increased payroll related costs and regulatory and operating related expenses. Pursuant to the License Agreement, our budgeted research and development expenses related to the development of Phentolamine
Ophthalmic Solution 0.75%, are fully reimbursed by our development partner. Research and development expenses also included $0.2 million in stock-based compensation expense during each of the three-month periods ended September 30, 2024 and 2023.
Net loss for the quarter ended September 30, 2024, was $7.5 million or $(0.29) per basic and diluted share as compared to net income of $5.6 million or $0.26 and $0.25 per basic and diluted share, respectively, for the third quarter of 2023.
For further details on our financial results, including results for the nine-month period ended September 30, 2024, please refer to our Quarterly Report on Form 10-Q to
be filed with the Securities and Exchange Commission.
Opus Genetics is a clinical-stage ophthalmic biotechnology company developing gene therapies to treat patients with inherited retinal diseases (IRDs) and therapies to
treat patients with other ophthalmologic disorders. The pipeline includes adeno-associated virus (AAV)-based gene therapies that address mutations in genes that cause different forms of bestrophinopathy, Leber congenital amaurosis (LCA) and
retinitis pigmentosa. The company's most advanced gene therapy program is designed to address mutations in the LCA5 gene, which encodes the lebercilin protein and is currently being evaluated in a Phase 1/2 open-label, dose-escalation trial, with
encouraging early data. BEST1 gene therapy is designed to address mutations in the BEST1 gene, which is associated with retinal degeneration; A Phase 1/2 study will be initiated in 2025. The pipeline also includes Phentolamine Ophthalmic Solution
0.75%, a non-selective alpha-1 and alpha-2 adrenergic antagonist to reduce pupil size, and APX3330, a novel small-molecule inhibitor of Ref-1 to slow the progression of non-proliferative diabetic retinopathy. Phentolamine Ophthalmic Solution 0.75%
is currently being evaluated in Phase 3 trials for presbyopia and dim (mesopic) light vision disturbances. For more information, please visit www.opusgtx.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 expectations
regarding our cash runway, data from and future enrollment for our clinical trials, our pipeline of additional indications, expectations of potential growth, and our expectations regarding integration following the acquisition of Opus Genetics,
including with respect to the combination of their portfolio of clinical assets into our existing portfolio and our combined focus on gene therapy treatment.
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 Ocuphire's Annual Report on Form 10-K. 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," "aim," "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 our 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 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 Securities and Exchange Commission 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. We undertake no obligation to update such statements to reflect events that occur
or circumstances that exist after the date on which they were made.
| Corporate | Investor Relations |
| Nirav Jhaveri, MBA CFO ir@opusgtx.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, | December 31, | |||||||
| 2024 | 2023 | |||||||
| Assets | (unaudited) | |||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 36,632 | $ | 50,501 | ||||
| Accounts receivable | 1,857 | 926 | ||||||
| Contract assets and unbilled receivables | 1,468 | 1,407 | ||||||
| Prepaids and other assets | 429 | 1,099 | ||||||
| Short-term investments | 3 | 15 | ||||||
| Total current assets | 40,389 | 53,948 | ||||||
| Property and equipment, net | - | - | ||||||
| Total assets | $ | 40,389 | $ | 53,948 | ||||
| Liabilities and stockholders' equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 844 | $ | 2,153 | ||||
| Accrued expenses | 5,171 | 1,815 | ||||||
| Derivative liability | 74 | 74 | ||||||
| Total current liabilities | 6,089 | 4,042 | ||||||
| Total liabilities | 6,089 | 4,042 | ||||||
| Commitments and contingencies | ||||||||
| Stockholders' equity: | ||||||||
| Preferred stock, par value $0.0001; 10,000,000 shares authorized as of September 30, 2024 and December 31, 2023; no shares issued and outstanding at September 30, 2024 and December 31, 2023. | - | - | ||||||
| Common stock, par value $0.0001; 125,000,000 and 75,000,000 shares authorized as of September 30, 2024 and December 31, 2023, respectively; 26,198,444 and 23,977,491 shares issued and outstanding at September 30, 2024 and December 31, 2023, respectively. | 3 | 2 | ||||||
| Additional paid-in capital | 138,160 | 131,370 | ||||||
| Accumulated deficit | (103,863 | ) | (81,466 | ) | ||||
| Total stockholders' equity | 34,300 | 49,906 | ||||||
| Total liabilities and stockholders' equity | $ | 40,389 | $ | 53,948 |
Ocuphire Pharma, Inc.
Condensed Statements of Comprehensive Loss
(in thousands, except share and per share amounts)
| For the Three Months Ended September 30, | For the Nine Months Ended September 30, | |||||||||||||||
| 2024 | 2023 | 2024 | 2023 | |||||||||||||
| License and collaborations revenue | $ | 3,867 | $ | 11,935 | $ | 6,690 | $ | 17,358 | ||||||||
| Operating expenses: | ||||||||||||||||
| General and administrative | 2,894 | 2,055 | 10,918 | 8,680 | ||||||||||||
| Research and development | 8,982 | 3,494 | 19,817 | 13,812 | ||||||||||||
| Total operating expenses | 11,876 | 5,549 | 30,735 | 22,492 | ||||||||||||
| (Loss) income from operations | (8,009 | ) | 6,386 | (24,045 | ) | (5,134 | ) | |||||||||
| Financing costs | - | (1,328 | ) | - | (1,328 | ) | ||||||||||
| Fair value change in derivative liability | - | 61 | - | 61 | ||||||||||||
| Other income, net | 483 | 456 | 1,648 | 1,224 | ||||||||||||
| (Loss) income before income taxes | (7,526 | ) | 5,575 | (22,397 | ) | (5,177 | ) | |||||||||
| Provision for income taxes | - | (14 | ) | - | (14 | ) | ||||||||||
| Net (loss) income | (7,526 | ) | 5,561 | (22,397 | ) | (5,191 | ) | |||||||||
| Other comprehensive (loss) income, net of tax | - | - | - | - | ||||||||||||
| Comprehensive (loss) income | $ | (7,526 | ) | $ | 5,561 | $ | (22,397 | ) | $ | (5,191 | ) | |||||
| Net (loss) income per share: | ||||||||||||||||
| Basic | $ | (0.29 | ) | $ | 0.26 | $ | (0.88 | ) | $ | (0.25 | ) | |||||
| Diluted | $ | (0.29 | ) | $ | 0.25 | $ | (0.88 | ) | $ | (0.25 | ) | |||||
| Number of shares used in per share calculations: | ||||||||||||||||
| Basic | 26,145,080 | 21,446,648 | 25,501,117 | 21,117,211 | ||||||||||||
| Diluted | 26,145,080 | 22,405,995 | 25,501,117 | 21,117,211 |