• |
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: Plan to initiate VEGA-2 trial, the first of
two Phase 3 registration trials intended to support the use of Nyxol alone and Nyxol with 0.4% low dose pilocarpine (LDP) in presbyopia, in 2H of 2022; if successful, the Company expects to file a supplemental NDA for Nyxol as a
single-agent for presbyopia and a new NDA for the combination thereafter
|
• |
Diabetic Retinopathy (DR) and Diabetic Macular Edema (DME):
Report top-line results from the APX3330 Phase 2b ZETA-1 trial in 2H 2022
|
• |
In April, the Company announced positive results from the MIRA-4 Phase 3 pediatric study in subjects aged 3-12 years, the final clinical trial supporting a planned NDA submission with for Nyxol
in RM. The study met its primary safety endpoint, demonstrating a favorable safety and tolerability profile with no adverse events reported with similar efficacy seen in adults.
|
• |
In May, the Company announced positive top-line results from the LYNX-1 Phase 3 trial evaluating Nyxol for Night Vision Disturbance (NVD). The study met the FDA-agreed upon primary endpoint with
statistically significantly more Nyxol-treated subjects having gained 15 or more letters of mesopic low contrast distance visual acuity compared to placebo. Key secondary efficacy endpoints were also met with statistical significance, and
Nyxol showed a favorable safety and tolerability profile with no serious adverse events reported. This is the first Phase 3 trial to study NVD and to show an improvement in distance and contrast vision at night with a pupil modulation
mechanism which has cross-over safety differentiation to presbyopia. The Phase 3 protocol for VEGA-2 was submitted to the FDA in July.
|
• |
In June, the Company held a Type B Pre-NDA meeting with the FDA at which it received guidance on the content of CMC, clinical and non-clinical modules of the planned NDA for Nyxol in RM. The FDA
confirmed that OraVerse and Regitine were appropriate to use as reference listed drugs to support a 505(b)(2) NDA.
|
• |
From April through August 2022, Ocuphire was represented at conferences by Mina Sooch and several prominent key thought leaders, including David Boyer, MD, Peter Kaiser, MD, David Lally, MD, and
Michael Allingham, MD, PhD who presented updates on APX3330 in DR, as well as Jay Pepose, MD, PhD, Marguerite McDonald, MD, Inder Paul Singh, MD, Ralph Chu, MD, James Katz, MD and Douglas Devries, OD who presented data for Nyxol in
Presbyopia and RM. Since the beginning of the year, 9 podium papers, 5 posters, and over 10 panel or company talks were presented across a total of 20 medical, scientific, industry and investment conferences.
|
• |
In June at the 45th Annual Macula Society Meeting in Germany and in July at the American Society of Retina Specialists (ASRS) 2022 in New York, David Boyer, MD and David Lally MD,
presented updated masked safety data from the ongoing ZETA-1 Phase 2b trial evaluating APX3330. The latest data from mid-June represented 100% of 103 subjects having completed 12 weeks or more and 70% having completed 24 weeks. These
safety data are consistent with favorable safety profile from the prior 11 clinical trials with total exposure experience of over 9,000 subject-days with a 600 mg daily dose of APX3330.
|
• |
In July, at the 40th Annual Meeting of ASRS, David Boyer, MD, presented for the first time to the retina medical community a late-breaking paper highlighting positive data from the MIRA-2 and
MIRA-3 Phase 3 registration trials for Nyxol eye drops to rapidly reverse mydriasis.
|
• |
In July, the Company announced the publication of a preclinical study supporting the novel mechanism of action inhibiting NFkB and the inflammatory pathway for APX3330 and APX pipeline
candidates. The publication, titled “RelA is an Essential Target for Enhancing Cellular Responses to the DNA Repair/Ref-1 Redox Signaling Protein and Restoring Perturbated Cellular Redox Homeostasis in Mouse PDAC Cells” was published in Frontiers in Oncology and can be accessed here.
|
• |
In April, Ocuphire appointed Jay Pepose, MD, PhD, as Chief Medical Advisor in addition to his Board of Directors role.
|
• |
Ocuphire was granted two new patents covering APX3330 and Nyxol:
|
o |
APX3330 for Use in Diabetics: U.S. Patent No. 11,351,130 with claims to methods of treating inflammation and chronic pain in subjects suffering from diabetes using APX3330 was issued on June 7,
2022 with expiry in 2038.
|
o |
Nyxol for Reversal of Mydriasis: U.S. Patent No. 11,400,077 with claims directed to methods for mydriasis treatment using phentolamine was issued on August 2, 2022 with expiry in 2039.
|
As of
|
||||||||
June 30,
2022
|
December 31,
2021
|
|||||||
(unaudited)
|
||||||||
Assets
|
||||||||
Current assets:
|
||||||||
Cash and cash equivalents
|
$
|
17,025
|
$
|
24,534
|
||||
Prepaids and other current assets
|
740
|
1,314
|
||||||
Short-term investments
|
126
|
219
|
||||||
Total current assets
|
17,891
|
26,067
|
||||||
Property and equipment, net
|
8
|
10
|
||||||
Total assets
|
$
|
17,899
|
$
|
26,077
|
||||
Liabilities and stockholders’ equity
|
||||||||
Current liabilities:
|
||||||||
Accounts payable
|
$
|
1,886
|
$
|
1,584
|
||||
Accrued expenses
|
1,418
|
1,733
|
||||||
Short-term loan
|
—
|
538
|
||||||
Total current liabilities
|
3,304
|
3,855
|
||||||
Warrant liabilities
|
—
|
—
|
||||||
Total liabilities
|
3,304
|
3,855
|
||||||
Commitments and contingencies
|
||||||||
Stockholders’ equity:
|
||||||||
Preferred stock, par value $0.0001; 10,000,000 shares authorized as of June 30, 2022 and December 31, 2021; no shares issued and
outstanding at June 30, 2022 and December 31, 2021.
|
—
|
—
|
||||||
Common stock, par value $0.0001; 75,000,000 shares authorized as of June 30, 2022 and December 31, 2021; 20,099,602 and 18,845,828 shares
issued and outstanding at June 30, 2022 and December 31, 2021, respectively.
|
2
|
2
|
||||||
Additional paid-in-capital
|
115,483
|
111,588
|
||||||
Accumulated deficit
|
(100,890
|
)
|
(89,368
|
)
|
||||
Total stockholders’ equity
|
14,595
|
22,222
|
||||||
Total liabilities and stockholders’ equity
|
$
|
17,899
|
$
|
26,077
|
For the Three Months Ended
June 30, |
For the Six Months Ended
June 30, |
|||||||||||||||
2022
|
2021
|
2022
|
2021
|
|||||||||||||
Collaborations revenue
|
$
|
—
|
$
|
100
|
$
|
—
|
$
|
100
|
||||||||
Operating expenses:
|
||||||||||||||||
General and administrative
|
1,776
|
3,408
|
3,512
|
5,112
|
||||||||||||
Research and development
|
3,162
|
3,829
|
7,934
|
7,311
|
||||||||||||
Total operating expenses
|
4,938
|
7,237
|
11,446
|
12,423
|
||||||||||||
Loss from operations
|
(4,938
|
)
|
(7,137
|
)
|
(11,446
|
)
|
(12,323
|
)
|
||||||||
Interest expense
|
(4
|
)
|
—
|
(9
|
)
|
—
|
||||||||||
Fair value change of warrant liabilities
|
—
|
—
|
—
|
(33,829
|
)
|
|||||||||||
Other income (expense), net
|
15
|
1
|
(67
|
)
|
2
|
|||||||||||
Loss before income taxes
|
(4,927
|
)
|
(7,136
|
)
|
(11,522
|
)
|
(46,150
|
)
|
||||||||
Benefit (provision) for income taxes
|
—
|
—
|
—
|
—
|
||||||||||||
Net loss
|
(4,927
|
)
|
(7,136
|
)
|
(11,522
|
)
|
(46,150
|
)
|
||||||||
Other comprehensive loss, net of tax
|
—
|
—
|
—
|
—
|
||||||||||||
Comprehensive loss
|
$
|
(4,927
|
)
|
$
|
(7,136
|
)
|
$
|
(11,522
|
)
|
$
|
(46,150
|
)
|
||||
Net loss per share:
|
||||||||||||||||
Basic and diluted
|
$
|
(0.25
|
)
|
$
|
(0.52
|
)
|
$
|
(0.60
|
)
|
$
|
(3.76
|
)
|
||||
Number of shares used in per share calculations:
|
||||||||||||||||
Basic and diluted
|
19,502,563
|
13,608,596
|
19,197,213
|
12,273,541
|