エドガーで原本を確認する
|
Delaware
|
001-34079
|
11-3516358
|
|
(State or other jurisdiction of incorporation)
|
(Commission File Number)
|
(IRS Employer Identification No.)
|
|
8 Davis Drive
Durham, NC
|
27709
|
|
|
(Address of principal executive offices)
|
(Zip Code)
|
|
| ☐ |
Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
|
| ☐ |
Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
|
| ☐ |
Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
|
| ☐ |
Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))
|
|
Title of each class
|
Trading Symbol(s)
|
Name of each exchange on which registered
|
|
Common Stock, $0.0001 par value per share
|
IRD
|
The Nasdaq Stock Market LLC
|
| Item 2.02 |
Results of Operations and Financial Condition.
|
| Item 9.01 |
Financial Statements and Exhibits.
|
|
(d) Exhibits
|
|
|
Exhibit No.
|
Description
|
|
Press Release, dated March 31, 2025.
|
|
|
104.1
|
Cover Page Interactive Data File (embedded within Inline XBRL document).
|
|
Dated: March 31, 2025
|
OPUS GENETICS, INC.
|
|
|
By:
|
/s/ Dr. George Magrath
|
|
|
Name:
|
Dr. George Magrath
|
|
|
Title:
|
Chief Executive Officer
|
|
| • |
On October 22, 2024, we acquired privately-held Opus Genetics Inc., a clinical-stage gene therapy company focused on IRDs, in an all-stock transaction. In connection with the acquisition, the
combined company adopted the name Opus Genetics, Inc., and began trading on Nasdaq under the ticker symbol “IRD,” effective as of October 24, 2024.
|
| • |
The Company’s IRD assets are supported by cutting-edge science developed by gene therapy pioneers at the University of Pennsylvania, Harvard Medical School, and University of Florida.
|
| • |
The combined company now has an expanded pipeline, which 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 (sometimes referred to as DLD) after keratorefractive surgery.
|
| • |
In March 2025, we completed an underwritten public offering, led by Perceptive Advisors and Nantahala Capital, with participation from other new institutional biotechnology investors, raising
gross proceeds of $20 million. This was accompanied by a concurrent private placement, generating gross proceeds of $1.5 million.
|
| • |
The financing increases the Company’s available cash resources to approximately $50.7 million, extending our runway and providing the resources needed to achieve key upcoming milestones for our
portfolio.
|
| • |
Additionally, there is potential for up to $21.4 million in further proceeds upon the exercise of warrants.
|
| • |
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 Leber congenital
amaurosis 5 (LCA5)-related LCA, an early-onset, severe hereditary retinal degeneration. The candidate is currently being evaluated in an open-label Phase 1/2 clinical trial which has shown clinical proof-of-concept. One-year data has
provided evidence that the therapy supported visual improvement in all three adult patients participating in the trial, each of whom has late-stage disease. Enrollment of the first pediatric patient occurred in the first quarter of 2025.
|
| • |
A Type D meeting was held with the FDA in March 2025 to discuss the 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 trial design, including the primary endpoint.
|
| • |
Six-month results from adult patients treated with OPGx-LCA5 were presented in a Key Opinion Leader webinar hosted by Opus on December 11, 2024. A replay of the webinar can be accessed here.
|
| • |
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.
|
| • |
In IND-enabling studies with OPGx-BEST1, we have observed compelling safety and efficacy data in support of a first-in-human clinical trial.
|
| • |
We anticipate commencement of a Phase 1/2 trial in 2025 and aim to obtain preliminary data by the first quarter of 2026.
|
| • |
The LYNX-2 pivotal Phase 3 trial evaluating Phentolamine Ophthalmic Solution 0.75% for the treatment of decreased vision under low light conditions following keratorefractive surgery completed
enrollment in the first quarter of 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).
|
| • |
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).
|
| • |
The VEGA-3 pivotal Phase 3 clinical 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.
|
| • |
The development portfolio related to Phentolamine Ophthalmic Solution 0.75% is being funded by our partner, Viatris Inc. (“Viatris”), in both indications (presbyopia and dim light vision
disturbances).
|
| • |
In December 2024, we reached agreement with the FDA under a SPA for a Phase 3 clinical trial evaluating APX3330, a novel, oral REF-1 inhibitor for the treatment of moderate to severe
non-proliferative diabetic retinopathy (NPDR). The SPA agreement reflects alignment on a proposed Phase 3 trial design, endpoints, and planned analyses to support submission of a New Drug Application for treatment of NPDR.
|
| • |
We intend to seek a strategic partner to advance late-stage development of APX3330.
|
| • |
Three abstracts on our investigational gene therapy candidates have been accepted for presentation at the ARVO 2025 Meeting, to take place from May 4 to 8, 2025 in Salt Lake City, UT. These
include a presentation on 12-month data from the first three adult patients in the ongoing Phase 1/2 trial of OPGx-LCA5.
|
| • |
Data on the first pediatric patient in the trial for OPGx-LCA5 data are anticipated in the third quarter of 2025.
|
| • |
Initiation of a Phase 1b/2a clinical trial for OPGx-BEST1 is planned for 2025. We aim to obtain preliminary data by the first quarter of 2026.
|
| • |
Topline date from the LYNX-2 pivotal Phase 3 trial evaluating Phentolamine Ophthalmic Solution 0.75% for decreased vision under low light conditions following keratorefractive surgery are expected
mid-year 2025.
|
| • |
Topline data from the VEGA-3 Phase 3 clinical trial evaluating Phentolamine Ophthalmic Solution 0.75% for the treatment of presbyopia are expected in the first half of 2025.
|
|
|
• |
Failure to successfully integrate our businesses with Former Opus could have a material adverse effect on our business, financial condition and results of operations;
|
|
|
• |
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;
|
|
|
• |
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;
|
|
|
• |
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;
|
|
|
• |
Changes in regulatory requirements could result in increased costs or delays in development timelines;
|
|
|
• |
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;
|
|
|
• |
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;
|
|
|
• |
We do not currently have any sales or marketing infrastructure in place and we have limited drug research and discovery capabilities;
|
|
|
• |
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;
|
|
|
• |
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;
|
|
|
• |
Failure to comply with health and safety laws and regulations could lead to material fines;
|
|
|
• |
We have not generated significant revenue from sales of any products and expect to incur losses for the foreseeable future;
|
|
|
• |
Our future viability is difficult to assess due to our short operating history and our future need for substantial additional capital, which could be limited by any adverse developments that affect
the financial services industry;
|
|
|
• |
Raising additional capital may cause our stockholders to be diluted, among other adverse effects;
|
|
|
• |
We operate in a highly regulated industry and face many challenges complying 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;
|
|
|
• |
We may not receive regulatory approval to market our developed product candidates within or outside of the U.S.;
|
|
|
• |
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;
|
|
|
• |
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;
|
|
|
• |
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;
|
|
|
• |
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;
|
|
|
• |
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;
|
|
|
• |
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;
|
|
|
• |
We may be unable to obtain full protection for our intellectual property rights under U.S. or foreign laws;
|
|
|
• |
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;
|
|
|
• |
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;
|
|
|
• |
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;
|
|
|
• |
The market price of our common stock is expected to be volatile and subject to certain dilutive risks associated with our Equity Line of Credit arrangement; and
|
|
|
• |
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.
|
|
Corporate
|
Investor Relations
|
|
Nirav Jhaveri, CFO
ir@opusgtx.com
|
Corey Davis, Ph.D.
LifeSci Advisors
cdavis@lifesciadvisors.com
|
|
As of December 31,
|
||||||||
|
2024
|
2023
|
|||||||
|
Assets
|
||||||||
|
Current assets:
|
||||||||
|
Cash and cash equivalents
|
$
|
30,321
|
$
|
50,501
|
||||
|
Accounts receivable
|
3,563
|
926
|
||||||
|
Contract assets and unbilled receivables
|
2,209
|
1,407
|
||||||
|
Prepaids and other current assets
|
515
|
1,099
|
||||||
|
Short-term investments
|
2
|
15
|
||||||
|
Total current assets
|
36,610
|
53,948
|
||||||
|
Property and equipment, net
|
252
|
—
|
||||||
|
Total assets
|
$
|
36,862
|
$
|
53,948
|
||||
|
Liabilities and stockholders’ equity
|
||||||||
|
Current liabilities:
|
||||||||
|
Accounts payable
|
$
|
3,148
|
$
|
2,153
|
||||
|
Accrued expenses
|
8,145
|
1,815
|
||||||
|
Derivative liability
|
2
|
74
|
||||||
|
Total current liabilities
|
11,295
|
4,042
|
||||||
|
Total liabilities
|
11,295
|
4,042
|
||||||
|
Commitments and contingencies
|
||||||||
|
Series A preferred stock, par value $0.0001; 14,146 shares and no shares were designated as of December 31, 2024 and
2023, respectively; 14,145.374 and no shares issued and outstanding at December 31, 2024 and 2023, respectively; no liquidation preference as of December 31, 2024 and 2023.
|
18,843
|
—
|
||||||
|
Stockholders’ equity:
|
||||||||
|
Preferred stock, par value $0.0001; 9,985,854 and 10,000,000 shares authorized as of December 31, 2024 and 2023,
respectively; no shares issued and outstanding at December 31, 2024 and 2023.
|
—
|
—
|
||||||
|
Common stock, par value $0.0001; 125,000,000 and 75,000,000 shares authorized as of December 31, 2024 and 2023,
respectively; 31,574,657 and 23,977,491 shares issued and outstanding at December 31, 2024 and 2023, respectively.
|
3
|
2
|
||||||
|
Additional paid-in capital
|
145,719
|
131,370
|
||||||
|
Accumulated deficit
|
(138,998
|
)
|
(81,466
|
)
|
||||
|
Total stockholders’ equity
|
6,724
|
49,906
|
||||||
|
Total liabilities, series A preferred stock and stockholders’ equity
|
$
|
36,862
|
$
|
53,948
|
||||
|
For the Year Ended
December 31,
|
||||||||
|
2024
|
2023
|
|||||||
|
License and collaborations revenue
|
$
|
10,992
|
$
|
19,049
|
||||
|
Operating expenses:
|
||||||||
|
General and administrative
|
18,215
|
11,959
|
||||||
|
Research and development
|
26,851
|
17,653
|
||||||
|
Acquired in-process research and development
|
28,000
|
—
|
||||||
|
Total operating expenses
|
73,066
|
29,612
|
||||||
|
Loss from operations
|
(62,074
|
)
|
(10,563
|
)
|
||||
|
Financing costs
|
—
|
(1,328
|
)
|
|||||
|
Fair value change in derivative liabilities
|
72
|
80
|
||||||
|
Other income, net
|
4,470
|
1,837
|
||||||
|
Loss before income taxes
|
(57,532
|
)
|
(9,974
|
)
|
||||
|
Provision for income taxes
|
—
|
(12
|
)
|
|||||
|
Net loss
|
(57,532
|
)
|
(9,986
|
)
|
||||
|
Other comprehensive loss, net of tax
|
—
|
—
|
||||||
|
Comprehensive loss
|
$
|
(57,532
|
)
|
$
|
(9,986
|
)
|
||
|
Net loss per share:
|
||||||||
|
Basic and diluted
|
$
|
(2.15
|
)
|
$
|
(0.46
|
)
|
||
|
Number of shares used in per share calculations:
|
||||||||
|
Basic and diluted
|
26,715,526
|
21,589,821
|
||||||