- Despite the ongoing COVID-19 pandemic, the Company’s laboratories have continued operations with limited impact on our research and development activities.
- The Company remains on track to initiate IND-enabling studies for Friedreich’s ataxia in the fourth quarter of this year.
- Confirmed overall response rate (ORR) of 21% in the dose-escalation stage across all doses, confirmed ORR 33% at the highest dose cohort and recommended Phase 2 dose.
- Target tumor shrinkage was observed in 37% of patients.
- Preliminary data show activity in patients with melanoma, Merkel cell carcinoma (MCC), and cutaneous squamous cell carcinoma (CSCC).
- Phase 2 arms in both MCC and CSCC are currently recruiting.
- In the second quarter of 2020, the Company began enrolling patients in the Phase 2 dose expansion phase of its Phase 1/2 clinical trial of intra-tumoral cavrotolimod (AST-008) in combination with approved checkpoint inhibitors pembrolizumab or cemiplimab, for the treatment of patients with advanced or metastatic MCC or CSCC.
- Currently, 14 clinical trial sites are open for enrollment and 7 additional sites are pending activation; the Company expects to open up to 25 sites for the Phase 2 stage of the clinical trial.
- We continue to monitor the impact that COVID-19 may be having on patient enrollment and safety, site initiation, and study integrity. We have put in place and continue to maintain a variety of measures to mitigate the effects of COVID-19 and our top priority is to maintain patient safety and clinical trial continuity. During the third quarter of 2020, we have observed delays in our enrollment plans for the Phase 2 dose expansion phase of this clinical trial. The effects of the COVID-19 pandemic or its impact may have contributed to such delays. As a result, we have taken additional measures to increase the enrollment of patients, including frequent interaction with our clinical trial sites currently open as well as increasing the number of clinical trial sites that potentially are activated for this clinical trial so that we may continue to enroll patients as planned. However, these delays have caused us to lengthen our clinical development timeline for cavrotolimod (AST-008) and we now expect to report ORR results in the first half of 2022 rather than by year-end 2021 as previously guided.
EXICURE, INC. UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS (in thousands, except share and per share data) |
|||||||
September 30, 2020 |
December 31, 2019 |
||||||
ASSETS |
|||||||
Current assets: |
|||||||
Cash and cash equivalents....................................................................................... |
$ |
31,459 |
$ |
48,460 |
|||
Short-term investments............................................................................................ |
62,621 |
62,326 |
|||||
Accounts receivable................................................................................................. |
59 |
16 |
|||||
Unbilled revenue receivable.................................................................................... |
6 |
19 |
|||||
Prepaid expenses and other assets........................................................................... |
2,684 |
1,955 |
|||||
Total current assets..................................................................................................... |
96,829 |
112,776 |
|||||
Property and equipment, net....................................................................................... |
4,269 |
2,099 |
|||||
Right-of-use asset....................................................................................................... |
8,768 |
356 |
|||||
Other noncurrent assets............................................................................................... |
1,414 |
32 |
|||||
Total assets................................................................................................... |
$ |
111,280 |
$ |
115,263 |
|||
LIABILITIES AND STOCKHOLDERS’ EQUITY |
|||||||
Current liabilities: |
|||||||
Current portion of long-term debt............................................................................ |
$ |
— |
$ |
4,965 |
|||
Accounts payable..................................................................................................... |
2,006 |
1,814 |
|||||
Accrued expenses and other current liabilities........................................................ |
2,124 |
2,435 |
|||||
Deferred revenue, current........................................................................................ |
8,479 |
21,873 |
|||||
Total current liabilities............................................................................................... |
12,609 |
31,087 |
|||||
Long-term debt, net..................................................................................................... |
16,500 |
— |
|||||
Common stock warrant liability, noncurrent.............................................................. |
— |
414 |
|||||
Deferred revenue, noncurrent..................................................................................... |
— |
2,956 |
|||||
Lease liability, noncurrent |
8,087 |
59 |
|||||
Other noncurrent liabilities......................................................................................... |
656 |
— |
|||||
Total liabilities............................................................................................. |
$ |
37,852 |
$ |
34,516 |
|||
Stockholders’ equity: |
|||||||
Common stock, $0.0001 par value per share; 200,000,000 shares authorized, |
|||||||
87,228,586 issued and outstanding, September 30, 2020; 86,069,263 |
|||||||
issued and outstanding, December 31, 2019................................................ |
9 |
9 |
|||||
Additional paid-in capital........................................................................................... |
166,499 |
162,062 |
|||||
Accumulated other comprehensive income (loss)...................................................... |
200 |
(27) |
|||||
Accumulated deficit.................................................................................................... |
(93,280) |
(81,297) |
|||||
Total stockholders' equity............................................................................. |
73,428 |
80,747 |
|||||
Total liabilities and stockholders’ equity..................................................... |
$ |
111,280 |
$ |
115,263 |
EXICURE, INC. UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (in thousands, except share and per share data) |
|||||||||||||||
Three Months Ended September 30, |
Nine Months Ended September 30, |
||||||||||||||
2020 |
2019 |
2020 |
2019 |
||||||||||||
Revenue: |
|||||||||||||||
Collaboration revenue................................................................................... |
$ |
2,443 |
$ |
527 |
$ |
16,473 |
$ |
986 |
|||||||
Total revenue.............................................................................. |
2,443 |
527 |
16,473 |
986 |
|||||||||||
Operating expenses: |
|||||||||||||||
Research and development expense................................................................................... |
9,139 |
4,245 |
22,222 |
11,073 |
|||||||||||
General and administrative expense................................................................................... |
2,424 |
2,228 |
7,227 |
6,421 |
|||||||||||
Total operating expenses.............................................................................. |
11,563 |
6,473 |
29,449 |
17,494 |
|||||||||||
Operating loss....................................................................................... |
(9,120) |
(5,946) |
(12,976) |
(16,508) |
|||||||||||
Other income (expense), net: |
|||||||||||||||
Dividend income................................................................................... |
2 |
228 |
45 |
415 |
|||||||||||
Interest income................................................................................... |
205 |
1 |
832 |
3 |
|||||||||||
Interest expense................................................................................... |
(27) |
(203) |
(155) |
(589) |
|||||||||||
Other income (expense), net................................................................................... |
118 |
104 |
271 |
357 |
|||||||||||
Total other income (expense), net.............................................................................. |
298 |
130 |
993 |
186 |
|||||||||||
Net loss....................................................................................... |
$ |
(8,822) |
$ |
(5,816) |
$ |
(11,983) |
$ |
(16,322) |
|||||||
Basic and diluted loss per common share....................................................................................... |
$ |
(0.10) |
$ |
(0.09) |
$ |
(0.14) |
$ |
(0.32) |
|||||||
Weighted-average basic and diluted common shares outstanding....................................................................................... |
87,227,136 |
64,651,040 |
87,160,520 |
51,200,072 |