Arcturus Therapeutics Announces Fourth Quarter and Full Year 2020 Financial Results and Positive Clinical Updates
ARCT-021 single-shot COVID-19 STARR™ mRNA vaccine to be advanced to Phase 3 clinical development – on track to initiate Phase 3 study in Q2
ARCT-021 single shot immunogenicity profile compares favorably with new data generated from recipients of a single dose of an approved conventional mRNA vaccine
ARCT-810 mRNA therapeutic for ornithine transcarbamylase deficiency to be advanced to Phase 2 clinical development – on track to file CTA for Phase 2 multiple dose study in Q2
ARCT-032 mRNA therapeutic for cystic fibrosis: completed successful pre-IND interaction with FDA – on track to file CTA in Q4
Investor conference call at
“Based on highly promising clinical data from our Phase 1/2 study and emerging mRNA vaccine immunological data, we are advancing ARCT-021 for further development in Phase 3. We are presently preparing to move forward a 5 µg single dose regimen, to be confirmed based on pending Phase 2 data. Our self-amplifying mRNA-based investigational vaccine may provide a differentiated clinical profile and characteristics that support widespread distribution across the globe. Our expectation is that successful protection from COVID-19 will require repeated vaccination of billions of individuals for years to come and that ARCT-021 will be re-dosable. We believe that a re-dosable, more easily distributable single shot mRNA vaccine would be a valuable option for many countries,” said
“In addition to advancing our vaccine franchise, we have made continued progress advancing our pipeline of promising liver and lung mRNA therapeutic candidates. After successfully beginning enrollment in the
Recent Corporate Highlights
ARCT-021, Vaccine Candidate for SARS-CoV-2
1) Ongoing Phase 2 clinical study and planning for Phase 3 development:
- More than 500 participants dosed across
USA andSingapore - Phase 3 study initiation remains on track for Q2
- Targeting application for Emergency Use Authorization in one or more jurisdictions in H2 2021
2) Encouraging new supportive data from
- Dr.
Eng Eong Ooi , Professor of Emerging Infectious Diseases atDuke-NUS Medical School , and colleagues, examined the adaptive immune responses of an approved conventional mRNA vaccine after a single administration - Data suggest that binding antibodies and cellular immunity are associated with COVID-19 protection at early timepoints after the first injection
- ARCT-021 single shot immunogenicity profile in Phase 1/2 study compares favorably, providing additional support for the potential efficacy of the ARCT-021 vaccine
|
|
Day |
BNT162b2 |
ARCT-021 |
|
IgG (≥ 4-fold rises) |
10 |
80% |
-- |
|
14 |
-- |
81%* |
|
|
PRNT (% Detectable) |
10 |
10% |
-- |
|
28 |
-- |
59% |
|
|
ELISpot (SFUs) |
10 |
28 |
-- |
|
15 |
-- |
211 |
|
|
21 |
13 |
-- |
|
| *Increases to 100% by Day 36 | |||
| IgG = Immunoglobulin G binding antibodies to full length spike protein | |||
| PRNT = plaque reduction neutralization test | |||
| SFUs = Spot Forming Units per million peripheral blood mononuclear cells | |||
3) Completed Phase 1/2 clinical study and supporting preclinical studies:
- Phase 1/2 study is complete and final data are under analysis; plan to submit results for publication in Q2
- ARCT-021 demonstrated robust protection with single dose in a primate challenge model and in a human ACE2 receptor transgenic mouse challenge model
- ARCT-021 demonstrated robust protection with a single dose in mice depleted of B cells, whereas depletion of T lymphocytes yielded no protection following virus challenge, emphasizing the importance of ARCT-021 induced cellular immunity
4) Manufacturing:
- Recently received
$46.6M from Singapore EDB to support ARCT-021 stockpiling - Manufacturing of lyophilized ARCT-021 to support Phase 3 and initial commercial supply well on track
- Stability studies for lyophilized ARCT-021 at -20°C, 2-8°C and room temperature storage conditions ongoing
5) Agreements:
- Strategic, government, and country supply agreement discussions continue to progress
ARCT-810, Therapeutic Candidate for Ornithine Transcarbamylase (OTC) Deficiency
- Received approval from
Health Canada to enroll subjects into Phase 1b study - CTA filing for Phase 2 multiple dose study on track for Q2
ARCT-032, Therapeutic Candidate for Cystic Fibrosis
- Completed successful pre-IND interaction with FDA
- CTA filing on track for Q4
Acquisition of Exclusive License to mRNA Manufacturing Technology from Alexion Pharmaceuticals
- The technology supports Arcturus’ highly efficient processes to manufacture high purity mRNA vaccine and therapeutic candidates at kilogram scale
- Extends the substantial intellectual property portfolio held by Arcturus
Financial results for the quarter and full year ended
Revenues in conjunction with strategic alliances and collaborations: Arcturus’ primary source of revenues is from license fees and collaboration payments received from research and development arrangements with our pharmaceutical and biotechnology partners.
On a quarterly basis, total revenue for the three months ended
On a yearly basis, reported revenues of
Operating expenses: On a quarterly basis, total operating expenses for the three months ended
On a yearly basis, total operating expenses were
Net loss: For the three months ended
For the year ended
Cash and Cash Equivalents: The Company’s cash balance was
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Domestic: |
877-407-0784 |
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International: |
201-689-8560 |
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Conference ID: |
13716298 |
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Webcast: |
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About
Founded in 2013 and based in
Forward Looking Statements
This press release contains forward-looking statements that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. Any statements, other than statements of historical fact included in this press release, are forward-looking statements, including those regarding strategy, future operations, collaborations, the likelihood of success, and the efficacy or safety, of the Company’s pipeline, including ARCT-021, ARCT-810 or ARCT-032, the planned initiation, design or completion of clinical trials, the likelihood that the Company will obtain clearance from regulatory authorities to proceed with planned clinical trials, the likelihood that preclinical or clinical data will be predictive of future clinical results, the likelihood that clinical data will be sufficient for regulatory approval or completed in time to submit an application for regulatory approval within a particular timeframe, the ability to enroll subjects in clinical trials, the Company’s efforts to develop a vaccine against COVID-19 and therapeutic potential thereof based on the Company’s mRNA therapeutics, the ability of the Company to scale up manufacturing of vaccine doses or to manufacture and scale up manufacturing of any other product or substance, the likelihood that a patent will issue from any patent application, the results of advancements in the Company’s manufacturing methods and technologies, including purification and lyophilization, its current cash position and expected cash burn and the impact of general business and economic conditions. Actual results and performance could differ materially from those projected in any forward-looking statements as a result of many factors including, without limitation, the ability to enroll subjects in clinical trials as a result of the COVID-19 pandemic, the impact of commercialization of third-party COVID-19 vaccines on the design, and ability to conduct, clinical trials, the availability of manufacturing capacity and raw materials, unexpected clinical results, and general market conditions that may prevent such achievements or performance. Arcturus may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in any forward-looking statements such as the foregoing and you should not place undue reliance on such forward-looking statements. Such statements are based on management’s current expectations and involve risks and uncertainties, including those discussed under the heading "Risk Factors" in Arcturus’ Annual Report on Form 10-K for the fiscal year ended
IR and Media Contacts
(858) 900-2682
IR@ArcturusRx.com
Kendall Investor Relations
(617) 914-0008
ctanzi@kendallir.com
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CONSOLIDATED BALANCE SHEETS (in thousands, except par value information) |
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|
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As of |
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|||||
|
|
|
2020 |
|
|
2019 |
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||
|
Assets |
|
|
|
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
462,895 |
|
|
$ |
71,353 |
|
|
Accounts receivable |
|
|
2,125 |
|
|
|
2,179 |
|
|
Prepaid expenses and other current assets |
|
|
2,769 |
|
|
|
758 |
|
|
Total current assets |
|
|
467,789 |
|
|
|
74,290 |
|
|
Property and equipment, net |
|
|
3,378 |
|
|
|
2,349 |
|
|
Operating lease right-of-use asset, net |
|
|
5,182 |
|
|
|
5,134 |
|
|
Equity-method investment |
|
|
— |
|
|
|
263 |
|
|
Non-current restricted cash |
|
|
107 |
|
|
|
107 |
|
|
Total assets |
|
$ |
476,456 |
|
|
$ |
82,143 |
|
|
Liabilities and stockholders’ equity |
|
|
|
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
|
|
|
Accounts payable |
|
$ |
10,774 |
|
|
$ |
5,793 |
|
|
Accrued liabilities |
|
|
20,639 |
|
|
|
7,134 |
|
|
Deferred revenue |
|
|
18,108 |
|
|
|
8,397 |
|
|
Total current liabilities |
|
|
49,521 |
|
|
|
21,324 |
|
|
Deferred revenue, net of current portion |
|
|
12,512 |
|
|
|
15,182 |
|
|
Long-term debt, net of current portion |
|
|
13,845 |
|
|
|
14,995 |
|
|
Operating lease liability, net of current portion |
|
|
4,025 |
|
|
|
4,850 |
|
|
Total liabilities |
|
|
79,903 |
|
|
|
56,351 |
|
|
Stockholders’ equity: |
|
|
|
|
|
|
|
|
|
Common stock: |
|
|
26 |
|
|
|
15 |
|
|
Additional paid-in capital |
|
|
540,343 |
|
|
|
97,445 |
|
|
Accumulated deficit |
|
|
(143,816 |
) |
|
|
(71,668 |
) |
|
Total stockholders’ equity |
|
|
396,553 |
|
|
|
25,792 |
|
|
Total liabilities and stockholders’ equity |
|
$ |
476,456 |
|
|
$ |
82,143 |
|
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CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (in thousands, except per share data) |
||||||||||||
|
|
|
Year Ended |
|
|||||||||
|
|
|
2020 |
|
|
2019 |
|
|
2018 |
|
|||
|
Collaboration revenue |
|
$ |
9,539 |
|
|
$ |
20,789 |
|
|
$ |
15,753 |
|
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and development, net |
|
|
57,846 |
|
|
|
33,640 |
|
|
|
16,982 |
|
|
General and administrative |
|
|
23,217 |
|
|
|
12,662 |
|
|
|
20,582 |
|
|
Total operating expenses |
|
|
81,063 |
|
|
|
46,302 |
|
|
|
37,564 |
|
|
Loss from operations |
|
|
(71,524 |
) |
|
|
(25,513 |
) |
|
|
(21,811 |
) |
|
Loss from equity-method investment |
|
|
(263 |
) |
|
|
(32 |
) |
|
|
(302 |
) |
|
Finance (expense) income, net |
|
|
(361 |
) |
|
|
(446 |
) |
|
|
328 |
|
|
Net loss |
|
|
(72,148 |
) |
|
|
(25,991 |
) |
|
|
(21,785 |
) |
|
Net loss per share, basic and diluted |
|
$ |
(3.55 |
) |
|
$ |
(2.15 |
) |
|
$ |
(2.16 |
) |
|
Weighted-average shares outstanding, basic and diluted |
|
|
20,305 |
|
|
|
12,069 |
|
|
|
10,069 |
|
|
Comprehensive loss: |
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss |
|
$ |
(72,148 |
) |
|
$ |
(25,991 |
) |
|
$ |
(21,785 |
) |
|
Unrealized gain on short-term investments |
|
|
— |
|
|
|
— |
|
|
|
3 |
|
|
Comprehensive loss |
|
$ |
(72,148 |
) |
|
$ |
(25,991 |
) |
|
$ |
(21,782 |
) |
|
|
|
Fourth Quarter 2019 (unaudited) |
|
|
First Quarter 2020 (unaudited) |
|
|
Second Quarter 2020 (unaudited) |
|
|
Third Quarter 2020 (unaudited) |
|
|
Fourth Quarter 2020 (unaudited) |
|
|||||
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Collaboration revenue |
|
$ |
2,968 |
|
|
$ |
2,646 |
|
|
$ |
2,322 |
|
|
$ |
2,333 |
|
|
$ |
2,238 |
|
|
Research and development expenses, net |
|
|
11,994 |
|
|
|
7,917 |
|
|
|
7,944 |
|
|
|
17,699 |
|
|
|
24,286 |
|
|
General and administrative expenses |
|
|
1,791 |
|
|
|
4,191 |
|
|
|
4,420 |
|
|
|
5,572 |
|
|
|
9,034 |
|
|
Loss from operations |
|
|
(10,817 |
) |
|
|
(9,462 |
) |
|
|
(10,042 |
) |
|
|
(20,938 |
) |
|
|
(31,082 |
) |
|
Net loss |
|
|
(10,989 |
) |
|
|
(9,777 |
) |
|
|
(10,263 |
) |
|
|
(21,004 |
) |
|
|
(31,104 |
) |
|
Net loss per share, basic and diluted |
|
$ |
(0.76 |
) |
|
$ |
(0.67 |
) |
|
$ |
(0.55 |
) |
|
$ |
(0.92 |
) |
|
$ |
(1.25 |
) |
|
Weighted average shares outstanding, basic and diluted |
|
|
14,505 |
|
|
|
14,521 |
|
|
|
18,794 |
|
|
|
22,938 |
|
|
|
24,886 |
|