• |
On October 22, Ocuphire acquired Opus Genetics, Inc., a clinical-stage gene therapy company for inherited retinal diseases (IRDs), in an all-stock transaction. The merger creates a transformative
biotech company committed to being a leader in the development of gene therapies for the treatment of IRDs. In connection with the merger, the combined company was renamed Opus Genetics, Inc., and began trading on Nasdaq under the ticker
symbol “IRD,” effective as of October 24, 2024. As consideration for the Opus Acquisition, the Company issued 5,237,063 shares of its common stock and 14,145.374 shares of Series A Preferred Stock, each of which is convertible into 1,000
shares of common stock. As of November 7, 2024 there were 31,568,457 shares of the Company’s common stock outstanding. If the shares of Series A Preferred Stock were converted as of that date, there would be a total of 45,713,831 shares
of common stock outstanding.
|
• |
As a combined company, we have an expanded pipeline that includes seven assets from our adeno-associated virus (AAV)-based gene therapy portfolio, each of them being developed for a specific IRD,
as well as Phentolamine Ophthalmic Solution 0.75%, which is currently being evaluated in presbyopia and dim (mesopic) light vision disturbances (sometimes referred to as DLD) after keratorefractive surgery.
|
• |
Our most advanced gene therapy candidate, OPGx-LCA5, is being developed to treat LCA5, an early-onset, severe hereditary retinal diseases. An open-label, dose-escalation Phase 1/2 clinical trial is
ongoing. The trial has shown early clinical proof-of-concept, with new six-month data demonstrating visual improvement in three out of three adult patients participating in the trial, each of whom has late-stage disease. Enrollment of the
first pediatric patients is expected in the first quarter of 2025, with the first data anticipated in the third quarter of 2025.
|
• |
OPGx-BEST1 is in development for Bestrophin1-associated retinal disease. IND-enabling studies with OPGx-BEST1 have generated compelling proof-of-concept efficacy data and exhibited favorable
safety. We anticipate filing a Clinical Trial Authorization (CTA) application in Germany in 2025 for commencement of a Phase 1/2 clinical trial.
|
• |
Enrollment is ongoing in the LYNX-2 Phase 3 registration study evaluating Phentolamine Ophthalmic Solution 0.75% for the treatment of dim light disturbances under mesopic (low) light conditions
following keratorefractive surgery. Top-line data are expected in the first quarter of 2025. The LYNX-2 trial is being conducted under conditions of a Special Protocol Assessment (SPA) with the FDA.
|
• |
The VEGA-3 Phase 3 trial evaluating Phentolamine Ophthalmic Solution 0.75% for the treatment of presbyopia is also enrolling patients. Top-line results are expected in the first half of 2025.
|
• |
The Phentolamine Ophthalmic Solution 0.75% development portfolio is being funded by Ocuphire’s partner in both indications (presbyopia and dim light vision disturbances).
|
• |
The Company intends to seek a strategic partner to advance further late-stage development of APX330, its novel, oral REF-1 inhibitor for diabetic retinopathy (DR), given the development timelines
and capital requirements. In the meantime, discussions remain ongoing with the FDA regarding the SPA for a Phase 3 program in DR.
|
• |
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 our inability to obtain sufficient additional capital to continue to advance our product candidates and our preclinical programs;
|
• |
Domestic and worldwide legislative, regulatory, political and economic developments;
|
• |
Employee misconduct
|
• |
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 or other licensing arrangements, may not facilitate the commercialization or market acceptance of our product candidates;
|
• |
Future fluctuations in the market price of our common stock;
|
• |
Our ability to realize the expected benefits of the acquisition of Opus Genetics;
|
• |
Our ability to execute clinical programs for gene therapies successfully and changes in expected commercial value we predict from the development of gene therapies;
|
• |
The success and timing of commercialization of any of our product candidates; and
|
• |
Obtaining and maintaining our intellectual property rights.
|
Corporate
|
Investor Relations
|
Nirav Jhaveri, MBA
CFO
ir@opusgtx.com
|
Corey Davis, Ph.D.
LifeSci Advisors
cdavis@lifesciadvisors.com
|
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
|
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
|