• |
Reversal of Mydriasis (RM): Planned New Drug Application (NDA) with the FDA for Nyxol in RM indication in late 2022, with potential launch as first dilation reversal drop in 2H 2023
|
• |
Presbyopia: Initiate
VEGA Phase 3 program in mid-2022 investigating Nyxol alone and Nyxol with 0.4% low-dose pilocarpine (LDP) as adjunctive therapy and, if successful, expect to file an NDA in 2023
|
• |
Night Vision Disturbances (NVD): Report top-line results from the Nyxol Phase 3 LYNX-1 trial in 2Q 2022
|
• |
Diabetic Retinopathy (DR) and Diabetic Macular Edema (DME): Report top-line results from the APX3330 Phase 2b ZETA-1 trial in 2H 2022
|
• |
In January, the Company announced new positive data from the VEGA-1 Phase 2 trial for Nyxol as a single agent in presbyopia, showing that one drop of Nyxol had statistically significant improvement in 3 lines of near vision efficacy at
12-hours compared to placebo.
|
• |
In January, the Company completed enrollment of the LYNX-1 Phase 3 Trial investigating Nyxol for the treatment of night vision disturbances in 145 patients (target of 140).
|
• |
In February, Ocuphire held a Type-C meeting with the FDA from which it obtained guidance regarding the design of pivotal studies for filing an NDA to seek approvals of Nyxol for the treatment of presbyopia, both as a single agent and
with LDP as adjunct therapy eye drops.
|
• |
In March, the Company announced successful results from the MIRA-3 Phase 3 registration trial of Nyxol for RM, demonstrating significant and rapid reversal of mydriasis. In addition, multiple key secondary endpoints met statistical
significance, including early onset of action, durable response over 24 hours, similar efficacy with one or two drops, and efficacy regardless of iris color or mydriatic agent used.
|
• |
In March, the Company completed enrollment of 103 (target of 90-100) diabetic retinopathy patients in the ZETA-1 Phase 2b trial of first-in-class oral APX3330. Masked safety data from the trial, first announced during the R&D Day
event in January 2022 and later presented through May at medical conferences, demonstrated a favorable safety profile, consistent with prior studies.
|
• |
In April, the Company completed the last clinical trial supporting a planned NDA submission with the announcement of positive results from the MIRA-4 Phase 3 pediatric study evaluating Nyxol for RM. The study met its primary safety
endpoint, demonstrating a favorable safety and tolerability profile with no adverse events reported.
|
• |
In January through May 2022, Ocuphire was represented at conferences by Mina Sooch and several prominent key thought leaders, including David Boyer, MD, David Lally, MD, Jay Pepose, MD, Inder Paul Singh, MD, Douglas Devries, OD, and
James Katz, MD, who presented updates on Nyxol in Presbyopia and RM, as well as masked safety data for APX3330 in DR. In total, 16 papers, posters, and panel talks were presented across 10 medical and industry conferences.
|
• |
In January, the Company held an Investor R&D Day webinar that featured six ophthalmic Key Opinion Leaders: Jay Pepose, MD, PhD, James Katz, MD and Mitchell Jackson, MD from refractive surgery, Paul Karpecki, OD from optometry, and
David Boyer, MD, and Peter Kaiser, MD, from retina practice areas who discussed the unmet needs in RM, presbyopia and DR addressed by Ocuphire’s two late-stage clinical drug assets, Nyxol and APX3300. A replay of the event can be found on
the Company’s corporate website here.
|
• |
In March, the Company appointed Jay Pepose, MD, PhD, as its Chief Medical Advisor.
|
As of
|
||||||||
March
31,
|
December
31,
|
|||||||
2022
|
2021
|
|||||||
(Unaudited)
|
||||||||
Assets
|
||||||||
Current assets:
|
||||||||
Cash and cash equivalents
|
$
|
19,246
|
$
|
24,534
|
||||
Prepaids and other current assets
|
1,095
|
1,314
|
||||||
Short-term investments
|
135
|
219
|
||||||
Total current assets
|
20,476
|
26,067
|
||||||
Property and equipment, net
|
9
|
10
|
||||||
Total assets
|
$
|
20,485
|
$
|
26,077
|
||||
|
||||||||
Liabilities and stockholders’ equity
|
||||||||
Current liabilities:
|
||||||||
Accounts payable
|
$
|
1,579
|
$
|
1,584
|
||||
Accrued expenses
|
1,419
|
1,733
|
||||||
Short-term loan
|
215
|
538
|
||||||
Total current liabilities
|
3,213
|
3,855
|
||||||
Warrant liabilities
|
—
|
—
|
||||||
Total liabilities
|
3,213
|
3,855
|
||||||
|
||||||||
Commitments and contingencies
|
||||||||
|
||||||||
Stockholders’ equity
|
||||||||
Preferred stock, par value $0.0001; 10,000,000 shares authorized as of March 31, 2022 and December 31, 2021; no shares issued and outstanding at March
31, 2022 and December 31, 2021.
|
—
|
—
|
||||||
Common stock, par value $0.0001; 75,000,000 shares authorized as of March 31, 2022 and December 31, 2021; 19,213,651 and 18,845,828 shares issued and
outstanding at March 31, 2022 and December 31, 2021, respectively.
|
2
|
2
|
||||||
Additional paid-in capital
|
113,233
|
111,588
|
||||||
Accumulated deficit
|
(95,963
|
)
|
(89,368
|
)
|
||||
Total stockholders’ equity
|
17,272
|
22,222
|
||||||
Total liabilities and stockholders’ equity
|
$
|
20,485
|
$
|
26,077
|
Three Months Ended
|
||||||||
March 31,
|
||||||||
2022
|
2021
|
|||||||
Operating expenses:
|
||||||||
General and administrative
|
$
|
1,736
|
$
|
1,704
|
||||
Research and development
|
4,772
|
3,482
|
||||||
Total operating expenses
|
6,508
|
5,186
|
||||||
Loss from operations
|
(6,508
|
)
|
(5,186
|
)
|
||||
Interest expense
|
(5
|
)
|
—
|
|||||
Fair value change in warrant liabilities
|
—
|
(33,829
|
)
|
|||||
Other (expense) income, net
|
(82
|
)
|
1
|
|||||
Loss before income taxes
|
(6,595
|
)
|
(39,014
|
)
|
||||
Benefit (provision) for income taxes
|
—
|
—
|
||||||
Net loss
|
(6,595
|
)
|
(39,014
|
)
|
||||
Other comprehensive loss, net of tax
|
—
|
—
|
||||||
Comprehensive loss
|
$
|
(6,595
|
)
|
$
|
(39,014
|
)
|
||
Net loss per share:
|
||||||||
Basic and diluted
|
$
|
(0.35
|
)
|
$
|
(3.57
|
)
|
||
Number of shares used in per share calculations:
|
||||||||
Basic and diluted
|
18,888,471
|
10,923,651
|