Partner or
OCUP
Name
|
Study
|
Milestone
|
Anticipated
Indication
|
1H 2023
|
2H
2023
|
APX3330
|
ZETA-1
|
End of Phase 2 FDA meeting
|
Diabetic Retinopathy
|
•
|
|
MR-140
(Nyxol) |
MIRA program
|
PDUFA date Sept 28, 2023
|
Reversal of
Pharmacologically-
induced Mydriasis
|
•
|
|
MR-141
(Nyxol and Nyxol+LDP) |
VEGA-3
|
Initiate 2nd Phase 3 trial
|
Presbyopia
|
•
|
|
VEGA-2
|
Report 1st Phase 3 topline data
|
Presbyopia
|
•
|
||
MR-142 (Nyxol) |
LYNX-2
|
Initiate 2nd Phase 3 trial
|
Night Vision or Dim
Light Disturbances
(DLD)
|
•
|
|
MR-141 and MR-142
(Nyxol) |
LYRA-1
|
Initiate long-term safety trial
|
Presbyopia and DLD
|
•
|
• |
In February 2023, the Company announced that U.S. Food and Drug Administration (FDA) has accepted for review a New Drug Application (NDA) for Nyxol in RM and set a PDUFA date of September 28, 2023. The NDA was
submitted in November 2022 and was supported by positive results from the comprehensive MIRA clinical programs (MIRA-1, MIRA-2, MIRA-3 and MIRA-4) collectively involving over 600 subjects, including pediatric subjects over 3 years old.
|
• |
In January 2023, the Company announced topline results from the ZETA-1 Phase 2 trial of oral APX3330 for the treatment of diabetic retinopathy (DR). Oral APX3330 achieved statistical significance on a key
pre-specified secondary endpoint of binocular ≥ 3-step worsening of DRSS and demonstrated favorable safety and tolerability after 24 weeks of treatment. This binocular secondary endpoint is a potential Phase 3 registration endpoint which
the Company plans a meeting with the FDA to formally agree on this endpoint and Phase 3 development in an End-of-Phase 2 meeting in 2H 2023. The safety and tolerability of APX3330 were favorable in diabetic patients and consistent with
prior trials in non-ophthalmic indications. Prevention of progression is a clinically meaningful outcome that may change the treatment paradigm for a large number of diabetic retinopathy (DR) patients.
|
• |
In January 2023, the Company announced the initiation of the VEGA-2 Phase 3 pivotal trial, with the first patient enrolled in late December. VEGA-2 is evaluating treatment efficacy and safety for two labels for
presbyopia: Nyxol alone and Nyxol with adjunctive Low Dose Pilocarpine (LDP) therapy. The Company plans to initiate a second Phase 3 pivotal trial in presbyopia (VEGA-3), and a one-year safety study (LYRA-1) in 2023.
|
• |
From January 2022 through March 2023, more than 20 papers, posters, and panel talks were presented at over 30 medical and industry conferences with updates on Nyxol in RM by Justin Schweitzer, OD, Leslie O’Dell,
OD and Shane Foster, OD, presbyopia by Mitch Jackson, MD and Mitch Ibach, OD, and night vision disturbances by Shane Kannarr, OD and on APX3330 in DR by Douglas Devries, OD, Peter Kaiser, MD and Rishi Singh, MD.
|
• |
In November 2022, the Company announced publication of an earlier Phase 2 clinical trial in patients with severe night vision disturbances in the BMC Ophthalmology peer-reviewed journal. The publication can be
accessed here.
|
• |
In October 2022, the Company held a key opinion leader (KOL) webinar on oral APX3330 featuring presentations by KOLs Peter Kaiser, MD, from the Cleveland Clinic, Caroline Baumal, MD, from Tufts Medical Center, and
David Lally, MD, from New England Retina Consultants. A replay of the event, including slides, can be found on the corporate website here.
|
• |
For the Nyxol Program:
|
o |
The United States Patent & Trademark Office (USPTO) issued U.S. Patent No. 11,400,077 in August 2022 with claims directed to methods for treating mydriasis using phentolamine mesylate, which has a term
extending into year 2039.
|
o |
The USPTO issued U.S. Patent No. 11,566,005 in January 2023 with claims directed polymorphic forms of phentolamine mesylate and methods using and preparing the same, which has a term extending into year 2042.
|
• |
For the APX3330 Program:
|
o |
The USPTO issued U.S. Patent No. 11,351,130 in June 2022 with claims directed to methods of treating inflammation and chronic pain using APX3330 in combination with certain additional therapeutic agents, which has
a term extending into year 2038, not including any patent term extension.
|
o |
The European Patent Office issued European Patent No. 3725309 in November 2022 with claims directed to APX3330 for use in inhibiting advanced macular degeneration, which has a term extending to year 2028, not
including any supplementary protection certificate.
|
• |
In November, the Company entered into an exclusive license agreement with FamyGen Life Sciences, Inc. (Famy), which was subsequently acquired by Viatris Inc. (Nasdaq: VTRS), for the development and
commercialization of Nyxol across three indications in US, Europe, Japan, India, China and other global markets. Viatris, a leading global healthcare company will be responsible for the commercialization of Nyxol following each regulatory
approval. Under the terms of the agreement, Ocuphire received an upfront payment of $35 million. Ocuphire has the potential to receive a $10 million milestone payment upon FDA approval for the RM indication later in 2023 and thereafter to
receive additional regulatory milestone payments for presbyopia and night vision disturbances indications. Upon commercialization, Ocuphire will receive tiered double-digit royalties on worldwide net sales through 2040 and is eligible to
receive sales milestone payments upon achievement of certain annual sales thresholds.
|
Corporate
|
Investor Relations
|
|
Mina Sooch, MBA
CEO & Founder
|
Corey Davis, Ph.D.
LifeSci Advisors
|
Bret Shapiro
CoreIR
|
As of December 31,
|
||||||||
2022
|
2021
|
|||||||
Assets
|
||||||||
Current assets:
|
||||||||
Cash and cash equivalents
|
$
|
42,634
|
$
|
24,534
|
||||
Accounts receivable
|
1,298
|
—
|
||||||
Contract asset
|
3,552
|
—
|
||||||
Prepaids and other current assets
|
1,453
|
1,314
|
||||||
Short-term investments
|
49
|
219
|
||||||
Total current assets
|
48,986
|
26,067
|
||||||
Property and equipment, net
|
6
|
10
|
||||||
Total assets
|
$
|
48,992
|
$
|
26,077
|
||||
Liabilities and stockholders’ equity
|
||||||||
Current liabilities:
|
||||||||
Accounts payable
|
$
|
1,069
|
$
|
1,584
|
||||
Accrued expenses
|
1,684
|
1,733
|
||||||
Short-term loan
|
—
|
538
|
||||||
Total current liabilities
|
2,753
|
3,855
|
||||||
Warrant liabilities
|
—
|
—
|
||||||
Total liabilities
|
2,753
|
3,855
|
||||||
Commitments and contingencies
|
||||||||
Stockholders’ equity
|
||||||||
Preferred stock, par value $0.0001; 10,000,000 shares authorized as of December 31, 2022 and 2021; no shares issued and outstanding at
December 31, 2022 and 2021.
|
—
|
—
|
||||||
Common stock, par value $0.0001; 75,000,000 shares authorized as of December 31, 2022 and 2021; 20,861,315 and 18,845,828 shares
issued and outstanding at December 31, 2022 and 2021, respectively.
|
2
|
2
|
||||||
Additional paid-in capital
|
117,717
|
111,588
|
||||||
Accumulated deficit
|
(71,480
|
)
|
(89,368
|
)
|
||||
Total stockholders’ equity
|
46,239
|
22,222
|
||||||
Total liabilities and stockholders’ equity
|
$
|
48,992
|
$
|
26,077
|
For the Year Ended
December 31,
|
||||||||
2022
|
2021
|
|||||||
License and collaborations revenue
|
$
|
39,850
|
$
|
589
|
||||
Operating expenses:
|
||||||||
General and administrative
|
7,269
|
8,121
|
||||||
Research and development
|
14,355
|
15,173
|
||||||
Total operating expenses
|
21,624
|
23,294
|
||||||
Income (loss) from operations
|
18,226
|
(22,705
|
)
|
|||||
Interest expense
|
(9
|
)
|
(2
|
)
|
||||
Fair value change in warrant liabilities
|
—
|
(33,829
|
)
|
|||||
Other expense, net
|
(14
|
)
|
(157
|
)
|
||||
Income (loss) before income taxes
|
18,203
|
(56,693
|
)
|
|||||
Provision for income taxes
|
(315
|
)
|
—
|
|||||
Net income (loss)
|
17,888
|
(56,693
|
)
|
|||||
Other comprehensive income (loss), net of tax
|
—
|
—
|
||||||
Comprehensive income (loss)
|
$
|
17,888
|
$
|
(56,693
|
)
|
|||
Net income (loss) per share:
|
||||||||
Basic
|
$
|
0.90
|
$
|
(3.82
|
)
|
|||
Diluted
|
$
|
0.87
|
$
|
(3.82
|
)
|
|||
Number of shares used in per share calculations:
|
||||||||
Basic
|
19,931,080
|
14,852,745
|
||||||
Diluted
|
20,597,212
|
14,852,745
|
For the Quarter Ended
December 31,
|
||||||||
2022
|
2021
|
|||||||
License and collaborations revenue
|
$
|
39,850
|
$
|
—
|
||||
Operating expenses:
|
||||||||
General and administrative
|
2,054
|
1,414
|
||||||
Research and development
|
3,586
|
4,736
|
||||||
Total operating expenses
|
5,640
|
6,150
|
||||||
Income (loss) from operations
|
34,210
|
(6,150
|
)
|
|||||
Interest expense
|
—
|
(2
|
)
|
|||||
Fair value change in warrant liabilities
|
—
|
—
|
||||||
Other expense, net
|
46
|
(161
|
)
|
|||||
Income (loss) before income taxes
|
34,256
|
(6,313
|
)
|
|||||
Provision for income taxes
|
(315
|
)
|
—
|
|||||
Net income (loss)
|
33,941
|
(6,313
|
)
|
|||||
Other comprehensive income (loss), net of tax
|
—
|
—
|
||||||
Comprehensive income (loss)
|
$
|
33,941
|
$
|
(6,313
|
)
|
|||
Net income (loss) per share:
|
||||||||
Basic
|
$
|
1.63
|
$
|
(0.35
|
)
|
|||
Diluted
|
$
|
1.58
|
$
|
(0.35
|
)
|
|||
Number of shares used in per share calculations:
|
||||||||
Basic
|
20,807,734
|
17,854,790
|
||||||
Diluted
|
21,476,348
|
17,854,790
|