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| Delaware |
001-34079 |
11-3516358
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(State or other jurisdiction of incorporation)
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(Commission File Number)
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(IRS Employer Identification No.)
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8 Davis Drive
Durham, NC
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27709
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(Address of principal executive offices)
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(Zip Code)
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| ☐ |
Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
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Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
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Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
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Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))
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Title of each class
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Trading Symbol(s)
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Name of each exchange on which registered
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Common Stock, $0.0001 par value per share
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IRD |
The Nasdaq Stock Market LLC
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| Item 2.02 |
Results of Operations and Financial Condition.
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| Item 9.01 |
Financial Statements and Exhibits.
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Exhibit No.
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Description
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Press Release, dated May 15, 2025.
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104.1
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Cover Page Interactive Data File (embedded within Inline XBRL document).
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Dated: May 15, 2025
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OPUS GENETICS, INC.
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By:
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/s/ Dr. George Magrath
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Name:
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Dr. George Magrath
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Title:
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Chief Executive Officer
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| • |
Opus’s pipeline includes a portfolio of seven adeno-associated virus (AAV)-based gene therapy assets, each targeting a specific IRD, as well as Phentolamine Ophthalmic Solution 0.75%, which is
currently being evaluated in presbyopia and mesopic (dim) light vision disturbances (DLD) after keratorefractive surgery.
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Emerging clinical data on OPGx-LCA5 provide evidence of clinical proof of concept and support the potential of this novel gene therapy to restore meaningful vision for individuals with mutations in
the LCA5 gene.
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Another gene therapy candidate, OPGx-BEST1, which Opus is developing for treatment of bestrophin-1 (BEST1)-related IRD, is on track to enter the clinic by the fourth quarter of 2025, with
preliminary data expected in the first quarter of 2026.
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| • |
In March 2025, Opus completed a successful underwritten public offering and concurrent private placement anchored by leading healthcare investors, Perceptive Advisors and Nantahala Capital. The
transaction raised approximately $21.5 million in gross proceeds with the potential for up to $21.4 million in additional proceeds upon exercise of warrants that are tied to data release from the BEST1 program.
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Opus’ most advanced investigational gene therapy candidate, OPGx-LCA5, is being developed to treat patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene, an
early-onset, severe hereditary retinal degeneration.
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| • |
One-year open-label data on adult patients being treated in the ongoing Phase 1/2 clinical trial of OPGx-LCA5 were featured in an oral presentation by Dr. Tomas Aleman of the Scheie Eye Institute,
University of Pennsylvania, at the Association for Research in Vision and Ophthalmology (ARVO) annual meeting, in Salt Lake City. All treated patients had late-stage disease. Improvements in subjective and objective measures of efficacy that
had been observed at six months persisted for one year.
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| • |
Enrollment in a cohort of three pediatric patients in the Phase 1/2 trial began in February 2025. Preliminary data on the first patient showed an encouraging early safety profile and meaningful
improvement in visual function at one month. The pediatric cohort is expected to complete enrollment in the second quarter of 2025, with initial data from all three patients anticipated in the third quarter of 2025.
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The U.S. Food and Drug Administration (the “FDA”) granted a Regenerative Medicine Advanced Therapy (“RMAT”) designation to OPGx-LCA5 based on the early data from the first three patients treated.
The RMAT designation program offers the potential for expedited development and review of regenerative medicine therapies that demonstrate the potential to address serious or life-threatening diseases based on preliminary clinical evidence.
The designation provides sponsors with early interactions with the FDA, guidance on efficient development and manufacturing, and the opportunity to discuss surrogate endpoints to support accelerated approval.
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A Type D meeting was held with the FDA in March 2025 to discuss the potential regulatory path for OPGx-LCA5, including the design of a potential registrational study. Opus will continue to work with
the FDA on the most appropriate design, including the primary endpoint.
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OPGx-BEST1 is an investigational Phase 1/2-ready asset in development for IRDs associated with mutations in the BEST1 gene (sometimes referred to as “Best Disease”), which can lead to legal
blindness.
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In IND-enabling studies of OPGx-BEST1 provided safety and efficacy data in support of a first-in-human clinical trial.
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Opus plans to file an IND and begin a Phase 1/2 trial by the fourth quarter of 2025, with preliminary data expected in the first quarter of 2026.
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The LYNX-2 pivotal Phase 3 trial evaluating Phentolamine Ophthalmic Solution 0.75% for the treatment of visual loss in low light conditions associated with keratorefractive surgery completed
enrollment in the first quarter of 2025 with topline data expected mid-year 2025. The LYNX-2 trial is covered by a Special Protocol Assessment (“SPA”) agreement with the FDA, which ensures agreement with the FDA on the trial design,
endpoints, and study size (power).
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The FDA granted Fast Track designation for Phentolamine Ophthalmic Solution 0.75% for treatment of significant chronic night driving impairment with concomitant increased risk of motor vehicle
accidents and debilitating loss of best spectacle corrected mesopic vision in keratorefractive patients with photic phenomena (i.e., glare, halos, starburst).
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The VEGA-3 pivotal Phase 3 trial evaluating Phentolamine Ophthalmic Solution 0.75% for the treatment of presbyopia completed enrollment in the first quarter of 2025, with topline data expected in
the first half of 2025.
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The development portfolio related to Phentolamine Ophthalmic Solution 0.75% is being funded by the Company’s partner, Viatris Inc., in both indications (presbyopia and dim light vision
disturbances).
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Initial data from three pediatric patients treated with OPGx-LCA5 anticipated in Q3 2025.
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IND filing and initiation of a Phase 1/2 clinical trial for OPGx-BEST1 is planned for 2025, with preliminary data expected in Q1 2026.
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Topline data from the LYNX-2 pivotal Phase 3 trial evaluating Phentolamine Ophthalmic Solution 0.75% for visual loss in low light conditions associated with keratorefractive surgery are expected
mid-year 2025.
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Topline data from the VEGA-3 pivotal Phase 3 clinical trial evaluating Phentolamine Ophthalmic Solution 0.75% for the treatment of presbyopia are expected in the first half of 2025.
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Our clinical data related to gene therapies for the treatment of IRDs are preliminary and related to a relatively small group of patients, and, as a result, data that initially appear promising may
be revised, updated, or invalidated at a later data readout and/or may ultimately not be capable of duplication in additional patients;
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Failure to successfully integrate our businesses following our acquisition of former Opus Genetics Inc. (the “Opus Acquisition”) could have a
material adverse effect on our business, financial condition and results of operations;
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The Opus Acquisition significantly expanded our product pipeline and business operations and shifted our business strategies, which may not improve the value of our common stock;
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Our gene therapy product candidates are based on a novel technology that is difficult to develop and manufacture, which may result in delays and difficulties in obtaining regulatory approval;
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Our planned clinical trials may face substantial delays, result in failure, or provide inconclusive or adverse results that may not satisfy FDA requirements to further develop our therapeutic
products;
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Delays or difficulties associated with patient enrollment in clinical trials may affect our ability to conduct and complete those clinical trials and obtain necessary regulatory approvals;
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Changes in regulatory requirements could result in increased costs or delays in development timelines;
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We depend heavily on the success of our product pipeline; if we fail to find strategic partners or fail to adequately develop or commercialize our pipeline products, our business will be materially
harmed;
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Others may discover, develop, or commercialize products similar to those in our pipeline before or more successfully than we do or develop generic variants of our products even while our product
patents remain active, thereby reducing our market share and potential revenue from product sales;
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We do not currently have any sales or marketing infrastructure in place, and we have limited drug research and discovery capabilities;
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The future commercial success of our products could significantly depend upon several uncertain factors, including third-party reimbursement practices and the existence of competitors with similar
products;
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Product liability lawsuits against us or our suppliers or manufacturers could cause us to incur substantial liabilities and could limit commercialization of any product candidate that we may develop;
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Failure to comply with health and safety laws and regulations could lead to material fines;
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We have not generated significant revenue from sales of any products and expect to incur losses for the foreseeable future;
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Our future viability is difficult to assess due to our short operating history and our future need for substantial additional capital, access to which could be limited by any adverse developments
that affect the financial services market;
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Raising additional capital may cause our stockholders to be diluted, among other adverse effects;
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We operate in a highly regulated industry and face many challenges adapting to sudden changes in legislative reform or the regulatory environment, which affects our pipeline stability and could
impair our ability to compete in international markets;
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We may not receive regulatory approval to market our developed product candidates within or outside of the U.S.;
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With respect to any of our product candidates that receive marketing approval, we may be subject to substantial penalties if we fail to comply with applicable regulatory requirements;
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Our potential relationships with healthcare providers and third-party payors will be subject to certain healthcare laws and regulations, which could expose us to extensive potential liabilities;
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We rely on third parties for material aspects of our business, such as conducting our nonclinical and clinical trials and supplying and manufacturing bulk drug substances, which exposes us to certain
risks;
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We may be unsuccessful in entering into or maintaining licensing arrangements (such as the Viatris License Agreement) or establishing strategic alliances on favorable terms, which could harm our
business;
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Our current focus on the cash-pay utilization for future sales of RYZUMVI™ may limit our ability to increase sales or achieve profitability with this product;
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Inadequate patent protection for our product candidates may result in our competitors developing similar or identical products or technology, which would adversely affect our ability to successfully
commercialize;
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We may be unable to obtain full protection for our intellectual property rights under U.S. or foreign laws;
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We may become involved in lawsuits for a variety of reasons associated with our intellectual property rights, including alleged infringement suits initiated by third parties;
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We are dependent on our key personnel, and if we are not successful in attracting and retaining highly qualified personnel, we may not be able to successfully implement our business strategy;
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As we grow, we may not be able to operate internationally or adequately develop and expand our sales, marketing, distribution, and other corporate functions, which could disrupt our operations;
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The market price of our common stock is expected to be volatile;
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Our common stock may be subject to delisting from the Nasdaq Capital Market and delisting could adversely affect our ability to access capital markets;
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Factors out of our control related to our securities, such as securities litigation or actions of activist stockholders, could adversely affect our business and stock price and cause us to incur
significant expenses; and
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Impact from current or proposed tariffs on imported goods we purchase.
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Corporate
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Investor Relations
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Nirav Jhaveri, CFO
ir@ocuphire.com
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Corey Davis, Ph.D.
LifeSci Advisors
cdavis@lifesciadvisors.com
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As of
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March 31,
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December
31,
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2025
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2024
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Assets
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(Unaudited)
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Current assets:
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Cash and cash equivalents
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$
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41,792
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$
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30,321
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Accounts receivable
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3,080
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3,563
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Contract assets and unbilled receivables
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1,675
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2,209
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Prepaids and other current assets
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1,380
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515
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Short-term investments
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1
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2
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Total current assets
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47,928
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36,610
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Property and equipment, net
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239
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252
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Total assets
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$
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48,167
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$
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36,862
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Liabilities and stockholders’ equity
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Current liabilities:
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Accounts payable
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$
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2,430
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$
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3,148
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Accrued expenses and other liabilities
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9,106
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8,147
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Warrant liabilities
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12,715
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—
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Total current liabilities
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24,251
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11,295
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Total liabilities
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24,251
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11,295
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Commitments and contingencies
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Series A preferred stock, par value $0.0001; 14,146 shares were designated as of March 31, 2025 and December 31, 2024;
14,145.374 shares issued and outstanding at March 31, 2025 and December 31, 2024.
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18,843
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18,843
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Stockholders’ equity:
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Preferred stock, par value $0.0001; 9,985,854 shares authorized as of March 31, 2025 and December 31, 2024; no shares
issued and outstanding at March 31, 2025 and December 31, 2024.
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—
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—
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Common stock, par value $0.0001; 125,000,000 shares authorized as of March 31, 2025 and December 31, 2024; 45,483,823
and 31,574,657 shares issued and outstanding at March 31, 2025 and December 31, 2024, respectively.
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5
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3
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Additional paid-in capital
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152,260
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145,719
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Accumulated deficit
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(147,192
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)
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(138,998
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)
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Total stockholders’ equity
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5,073
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6,724
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Total liabilities, series A preferred stock, and stockholders’ equity
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$
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48,167
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$
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36,862
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Three Months Ended
March 31,
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2025
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2024
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License and collaborations revenue
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$
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4,370
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$
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1,711
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Operating expenses:
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General and administrative
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6,346
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4,670
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Research and development
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7,953
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4,749
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Total operating expenses
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14,299
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9,419
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Loss from operations
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(9,929
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)
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(7,708
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)
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Financing costs
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(1,372
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)
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—
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Fair value change in warrant liabilities
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2,805
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—
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Other income, net
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302
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602
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Loss before income taxes
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(8,194
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)
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(7,106
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)
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Benefit (provision) for income taxes
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—
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—
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Net loss
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(8,194
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)
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(7,106
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)
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Other comprehensive loss, net of tax
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—
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—
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Comprehensive loss
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$
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(8,194
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)
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$
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(7,106
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)
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Net loss per share:
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Basic and diluted
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$
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(0.24
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)
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$
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(0.29
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)
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Number of shares used in per share calculations:
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||||||||
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Basic and diluted
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33,884,920
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24,520,475
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