• |
Reversal of Mydriasis (RM): Initiate second
Phase 3 (MIRA-3) registration trial in subjects 12 and older and a small pediatric trial in subjects ages 3 to 11 (MIRA-4) in the fourth quarter of 2021 investigating Nyxol with results expected in early 2022; Planning to file NDA
submission with FDA for Nyxol in RM indication in late 2022
|
• |
Presbyopia: Initiate Phase 3 program (VEGA-2)
in first half of 2022 investigating Nyxol and Low-Dose Pilocarpine (LDP)
|
• |
Night Vision Disturbances (NVD): Top-line data expected in early 2022 from Phase 3 (LYNX-1) registration trial investigating Nyxol
|
• |
Diabetic Retinopathy (DR) and Diabetic Macular Edema
(DME): Top-line data expected in the second half of 2022 for the randomized, well-controlled Phase 2 (ZETA-1) trial investigating APX3330
|
• |
In November, clinical data on Nyxol® and APX3330 were accepted for presentation at poster sessions at the American Academy of Ophthalmology (AAO) 2021 annual meeting to take place in New Orleans, November 12 – 15. In addition, Ocuphire presented new data on improvement in intermediate vision and Snellen equivalent
near vision at the Eyecelerator@AAO 2021 conference on November 11. Ocuphire was one of two companies presenting clinical data
for presbyopia at this meeting.
|
• |
In October, the Company announced the publication of a review article
within the Special Issue “Advances in Molecular Activity of Potential Drugs” of the International Journal of
Molecular Sciences, focused on how novel inhibitors of APE1/Ref-1 such as APX3330 may have the potential to improve disease outcomes for
retinal disease patients. The article underscores the role of the APE1/Ref-1 protein in pro-angiogenic pathways associated with neovascular eye disease including diabetic retinal diseases and age-related macular degeneration. It can be accessed online
at the following link: Inhibition of APE1/Ref-1 for Neovascular Eye Disease: From Biology to Therapy.
|
• |
In October, the Company announced the publication of a review article in
Cells titled “Potential
Therapeutic Candidates for Age-Related Macular Degeneration” noting the potential of APX3330 (referred to as “E3330”) for the treatment of
age-related macular degeneration (AMD). Because APE1/Ref-1 has been shown to contribute to retinal angiogenesis, the authors conclude that APE1/Ref-1 inhibitors such as APX3330 could inhibit the abnormal blood vessel formation seen in AMD
by reducing retinal endothelial cell proliferation, migration, and tube formation. The article can be accessed online at the following link: Potential Therapeutic Candidates for Age-Related Macular Degeneration (AMD).
|
• |
In October, Michael J. Allingham, MD, PhD presented at the 39th
Annual Scientific Meeting of the American Society of Retina Specialists (ASRS) (Diabetic Retinopathy 1 Symposium), highlighting the favorable
safety and tolerability data for APX3330 in over 300 healthy volunteers and cancer/inflammation disease patients across 11 Phase 1 and Phase 2 studies. Also, Mina Sooch, CEO, presented APX3330 history and the design of the ongoing Phase 2
trial in DR at the OIS Retina Innovation Summit@ASRS.
|
• |
In July, the Company announced publication in the Journal of
Cellular Signaling featuring Ocuphire’s novel oral Ref-1 inhibitor APX3330 in Phase 2 trial for the treatment of retinal disease which
highlighted the favorable safety profile of APX3330 and its unique anti-angiogenic and anti-inflammatory mechanism of action properties relevant to a broad range of retinal diseases.
|
• |
In July, at the 2021 American Society of Cataract and Refractive Surgery (ASCRS) Annual Meeting, Dr. Jay
S. Pepose, Medical Advisor and Board Director, presented papers featuring positive results for Nyxol in two studies: Phase 2 Presbyopia (VEGA-1) and Phase 3 Reversal of Mydriasis (MIRA-2). The Phase 3 MIRA-2 data presentation at ASCRS won the Best Paper of the Session.
|
• |
In July, Mina Sooch, CEO, participated in the presbyopia drug therapy
panel at the Eyecelerator@ASCRS 2021 held on July 22nd and in the Eye on Innovation panel at the Virtual Salon Series held on July
28th.
|
• |
U.S. Patent and Trademark Office issued patent no. 11,160,770 “Compounds, compositions and methods for treating oxidative DNA damage disorders” which
provides protection for APX2009 and other APX pipeline candidates.
|
As of
|
||||||||
September 30,
|
December 31,
|
|||||||
2021
|
2020
|
|||||||
(unaudited)
|
||||||||
Assets
|
||||||||
Current assets:
|
||||||||
Cash and cash equivalents
|
$
|
22,250
|
$
|
16,399
|
||||
Short-term investments
|
383
|
—
|
||||||
Prepaids and other assets
|
560
|
1,269
|
||||||
Total current assets
|
23,193
|
17,668
|
||||||
Property and equipment, net
|
11
|
14
|
||||||
Total assets
|
$
|
23,204
|
$
|
17,682
|
||||
Liabilities and stockholders’ equity (deficit)
|
||||||||
Current liabilities:
|
||||||||
Accounts payable
|
$
|
1,434
|
$
|
1,214
|
||||
Accrued expenses
|
1,204
|
1,971
|
||||||
Total current liabilities
|
2,638
|
3,185
|
||||||
Warrant liabilities
|
—
|
27,964
|
||||||
Total liabilities
|
2,638
|
31,149
|
||||||
Commitments and contingencies
|
||||||||
Stockholders’ equity (deficit)
|
||||||||
Preferred stock, par value $0.0001; 10,000,000 shares authorized as of September 30, 2021 and December 31, 2020;
no shares issued and outstanding at September 30, 2021 and December 31, 2020.
|
—
|
—
|
||||||
Common stock, par value $0.0001; 75,000,000 shares authorized as of September 30, 2021 and December 31, 2020;
17,295,434 and 10,882,495 shares issued and outstanding at September 30, 2021 and December 31, 2020, respectively.
|
2
|
1
|
||||||
Additional paid-in-capital
|
103,619
|
19,207
|
||||||
Accumulated deficit
|
(83,055
|
)
|
(32,675
|
)
|
||||
Total stockholders’ equity (deficit)
|
20,566
|
(13,467
|
)
|
|||||
Total liabilities and stockholders’ equity (deficit)
|
$
|
23,204
|
$
|
17,682
|
For the Three Months Ended
|
For the Nine Months Ended
|
|||||||||||||||
September 30,
|
September 30,
|
|||||||||||||||
2021
|
2020
|
2021
|
2020
|
|||||||||||||
Collaborations revenue
|
$
|
489
|
$
|
—
|
$
|
589
|
$
|
—
|
||||||||
Operating expenses:
|
||||||||||||||||
General and administrative
|
1,595
|
565
|
6,707
|
1,508
|
||||||||||||
Research and development
|
3,126
|
1,383
|
10,437
|
2,311
|
||||||||||||
Acquired in-process research and development
|
—
|
—
|
—
|
2,126
|
||||||||||||
Total operating expenses
|
4,721
|
1,948
|
17,144
|
5,945
|
||||||||||||
Loss from operations
|
(4,232
|
)
|
(1,948
|
)
|
(16,555
|
)
|
(5,945
|
)
|
||||||||
Interest expense
|
—
|
(179
|
)
|
—
|
(1,422
|
)
|
||||||||||
Fair value change of warrant liability and premium conversion derivatives
|
—
|
879
|
(33,829
|
)
|
158
|
|||||||||||
Gain on note extinguishment
|
—
|
—
|
—
|
1,260
|
||||||||||||
Other income, net
|
2
|
—
|
4
|
9
|
||||||||||||
Loss before income taxes
|
(4,230
|
)
|
(1,248
|
)
|
(50,380
|
)
|
(5,940
|
)
|
||||||||
Benefit (provision) for income taxes
|
—
|
—
|
—
|
—
|
||||||||||||
Net loss
|
(4,230
|
)
|
(1,248
|
)
|
(50,380
|
)
|
(5,940
|
)
|
||||||||
Other comprehensive loss, net of tax
|
—
|
—
|
—
|
—
|
||||||||||||
Comprehensive loss
|
$
|
(4,230
|
)
|
$
|
(1,248
|
)
|
$
|
(50,380
|
)
|
$
|
(5,940
|
)
|
||||
Net loss per share:
|
||||||||||||||||
Basic and diluted
|
$
|
(0.25
|
)
|
$
|
(0.33
|
)
|
$
|
(3.64
|
)
|
$
|
(1.61
|
)
|
||||
Number of shares used in per share calculations:
|
||||||||||||||||
Basic and diluted
|
16,925,006
|
3,743,907
|
13,841,067
|
3,678,840
|