Arcturus Therapeutics Announces Third Quarter 2024 Financial Update and Pipeline Progress
Cystic Fibrosis and OTC Deficiency Phase 2 studies on track for POC data in first half of 2025
KOSTAIVE® European CHMP opinion expected December
Positive results from multiple Phase 3 studies support KOSTAIVE®
Superior 12-month durability results from Phase 3 study of KOSTAIVE® published in The Lancet Infectious Diseases
Positive Phase 3 results showed KOSTAIVE® XBB.1.5. met all four primary study objectives and key secondary objectives
Investor conference call at
“I am thrilled about the approval of KOSTAIVE® for the COVID-19 JN.1 strain in
“I am happy to report our first commercial milestone achieved from our CSL partnership for the first commercial sale of KOSTAIVE in Japan,” said
Recent Corporate Highlights
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In September, Arcturus received clearance of an Investigational New Drug application from the
U.S. Food and Drug Administration (FDA), enabling the Company to initiate a Phase 2 multiple ascending dose study to evaluate the safety, tolerability and efficacy of ARCT-032 in people with cystic fibrosis (CF).- The Phase 2 study is screening individuals with CF who do not qualify for, or benefit from, CFTR modulator medicines due to dysfunctional or absent CFTR protein and/or drug intolerance.
- The Company remains on track to share ARCT-032 Phase 2 proof-of-concept (POC) interim data in 1H25.
-
In August, the Company announced the expansion of the Phase 2 clinical program of ARCT-810, an mRNA therapeutic to potentially treat ornithine transcarbamylase (OTC) deficiency, into
the United States .- This open-label multiple-dose study (NCT06488313) evaluating pharmacodynamics and safety is currently enrolling adults and adolescents requiring clinical management for OTC deficiency.
- The placebo-controlled Phase 2 European study has completed the dosing phase (N = 8; 0.3 mg/kg) in OTC deficient individuals.
-
The Company remains on track to share ARCT-810 Phase 2 POC interim data from both
U.S. and European studies in 1H25.
-
Meiji Seika Pharma , CSL’s exclusive partner inJapan , began KOSTAIVE commercial sales inSeptember 2024 . This event triggered a$25 million commercial milestone associated with the first sale of KOSTAIVE® inJapan . -
In September, the Company, along with partners CSL and Meiji, announced new 12-month post vaccination data for KOSTAIVE at OPTIONS XII for the Control of Influenza conference.
- The results of a head-to-head study demonstrated that KOSTAIVE maintained superior immunogenicity compared to the conventional mRNA vaccine COMIRNATY® for up to one year against Wuhan-Hu-1, Omicron BA.4/5 and certain other variants, and at one-sixth the dose of the comparator (5 μg vs 30 μg, respectively). The results were published in The Lancet Infectious Disease.
- Additional Phase 3 data presented by CSL, Meiji and Arcturus show that bivalent KOSTAIVE, ARCT-2301, induced superior immunogenicity over conventional bivalent mRNA vaccine COMIRNATY® that persists against key variants up to six months post vaccination.
-
Earlier this year, CSL Seqirus’s partner
Meiji Seika Pharma announced that it submitted a partial change application for an amendment to the manufacturing and marketing approval of KOSTAIVE® to include manufacturing sites inJapan , includingARCALIS, Inc. , Arcturus’ manufacturing joint venture inJapan . When approved,Meiji Seika Pharma will begin selling domestically produced KOSTAIVE® this season. -
The Company announced the results of a Phase 3 study which demonstrated the added value of an updated COVID-19 vaccine (ARCT-2303) containing the Omicron XBB.1.5 variant. The study supports co-administration of KOSTAIVE with licensed influenza vaccines.
- ARCT-2303 demonstrated superior immune response versus ARCT-154 as measured by neutralizing antibodies against Omicron XBB.1.5.6 in terms of GMT ratio and SCR difference.
- Co-administration of ARCT-2303 and cell-based quadrivalent influenza vaccine (QIV; FLUCELVAX®, CSL) showed noninferior immune response vs standalone QIV administration.
- Co-administration of ARCT-2303 and QIV showed noninferior immune response vs standalone ARCT-2303 administration.
- Co-administration of ARCT-2303 and adjuvanted QIV (FLUAD®, CSL) in older adults showed similar responses vs standalone administration of ARCT-2303 and adjuvanted QIV.
Financial Results for the three months ended
Revenues in conjunction with strategic alliances and collaborations:
Arcturus’ primary revenue streams include license fees, consulting and related technology transfer fees, reservation fees and collaborative payments received from research and development arrangements with pharmaceutical and biotechnology partners. For the three months ended
Revenue decreased by
Operating expenses:
Total operating expenses for the three months ended
Research and development expenses:
Research and development expenses consist primarily of external manufacturing costs, in vivo research studies and clinical trials performed by contract research organizations, clinical and regulatory consultants, personnel-related expenses, facility-related expenses and laboratory supplies related to conducting research and development activities. Research and development expenses were
General and Administrative Expenses:
General and administrative expenses primarily consist of salaries and related benefits for executive, administrative, legal and accounting functions and professional service fees for legal and accounting services as well as other general and administrative expenses. General and administrative expenses were
Net Loss:
For the three months ended
Cash Position and Balance Sheet:
Cash, cash equivalents and restricted cash were
Earnings Call:
- Domestic: 1-800-274-8461
- International: 1-203-518-9814
- Conference ID: ARCTURUS
- Webcast: Link
About Arcturus
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, the likelihood of success and continued advancement of the Company’s pipeline (including ARCT-032 and ARCT-810) and partnered programs (including the COVID-19 and flu programs partnered with CSL Seqirus), the likelihood and extent of commercialization of KOSTAIVE and the timing thereof, the continued clinical development of the rare disease programs, the interim Phase 2 proof-of-concept clinical data and the timing therefor, the likelihood and timing of European Marketing Authorization application approval for KOSTAIVE and of a milestone payment from CSL related thereto, the planned transfer of the CF manufacturing process to
Trademark Acknowledgements
The Arcturus logo and other trademarks of Arcturus appearing in this announcement, including LUNAR® and STARR®, are the property of Arcturus. All other trademarks, services marks, and trade names in this announcement are the property of their respective owners.
CONDENSED CONSOLIDATED BALANCE SHEETS |
||||||||
|
|
|
|
|
||||
(in thousands, except par value information) |
|
(unaudited) |
|
|
||||
Assets |
|
|
|
|
||||
Current assets: |
|
|
|
|
||||
Cash and cash equivalents |
|
$ |
237,178 |
|
|
$ |
292,005 |
|
Restricted cash |
|
|
55,000 |
|
|
|
55,000 |
|
Accounts receivable |
|
|
30,199 |
|
|
|
32,064 |
|
Prepaid expenses and other current assets |
|
|
8,444 |
|
|
|
7,521 |
|
Total current assets |
|
|
330,821 |
|
|
|
386,590 |
|
Property and equipment, net |
|
|
10,350 |
|
|
|
12,427 |
|
Operating lease right-of-use assets, net |
|
|
27,598 |
|
|
|
28,500 |
|
Non-current restricted cash |
|
|
1,885 |
|
|
|
1,885 |
|
Total assets |
|
$ |
370,654 |
|
|
$ |
429,402 |
|
Liabilities and stockholders’ equity |
|
|
|
|
||||
Current liabilities: |
|
|
|
|
||||
Accounts payable |
|
$ |
10,131 |
|
|
$ |
5,279 |
|
Accrued liabilities |
|
|
32,396 |
|
|
|
31,881 |
|
Deferred revenue |
|
|
26,936 |
|
|
|
44,829 |
|
Total current liabilities |
|
|
69,463 |
|
|
|
81,989 |
|
Deferred revenue, net of current portion |
|
|
13,338 |
|
|
|
42,496 |
|
Operating lease liability, net of current portion |
|
|
25,987 |
|
|
|
25,907 |
|
Other non-current liabilities |
|
|
— |
|
|
|
497 |
|
Total liabilities |
|
|
108,788 |
|
|
|
150,889 |
|
Stockholders’ equity |
|
|
|
|
||||
Common stock, |
|
|
27 |
|
|
|
27 |
|
Additional paid-in capital |
|
|
680,641 |
|
|
|
646,352 |
|
Accumulated deficit |
|
|
(418,802 |
) |
|
|
(367,866 |
) |
Total stockholders’ equity |
|
|
261,866 |
|
|
|
278,513 |
|
Total liabilities and stockholders’ equity |
|
$ |
370,654 |
|
|
$ |
429,402 |
|
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (unaudited) |
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|
|
Three Months Ended |
|
Nine Months Ended |
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|
|
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(in thousands, except per share data) |
|
2024 |
|
2023 |
|
2024 |
|
2023 |
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Revenue: |
|
|
|
|
|
|
|
|
||||||||
Collaboration revenue |
|
$ |
38,815 |
|
|
$ |
43,376 |
|
|
$ |
117,389 |
|
|
$ |
132,670 |
|
Grant revenue |
|
|
2,858 |
|
|
|
1,764 |
|
|
|
12,155 |
|
|
|
3,274 |
|
Total revenue |
|
|
41,673 |
|
|
|
45,140 |
|
|
|
129,544 |
|
|
|
135,944 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
||||||||
Research and development, net |
|
|
39,134 |
|
|
|
51,077 |
|
|
|
151,376 |
|
|
|
155,513 |
|
General and administrative |
|
|
13,276 |
|
|
|
13,377 |
|
|
|
40,443 |
|
|
|
40,364 |
|
Total operating expenses |
|
|
52,410 |
|
|
|
64,454 |
|
|
|
191,819 |
|
|
|
195,877 |
|
Loss from operations |
|
|
(10,737 |
) |
|
|
(19,314 |
) |
|
|
(62,275 |
) |
|
|
(59,933 |
) |
(Loss) gain from foreign currency |
|
|
(201 |
) |
|
|
4 |
|
|
|
(642 |
) |
|
|
(175 |
) |
Gain on debt extinguishment |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
33,953 |
|
Finance income, net |
|
|
3,818 |
|
|
|
3,981 |
|
|
|
11,981 |
|
|
|
9,710 |
|
Net loss before income taxes |
|
|
(7,120 |
) |
|
|
(15,329 |
) |
|
|
(50,936 |
) |
|
|
(16,445 |
) |
Provision for income taxes |
|
|
(217 |
) |
|
|
893 |
|
|
|
— |
|
|
|
1,573 |
|
Net loss |
|
$ |
(6,903 |
) |
|
$ |
(16,222 |
) |
|
$ |
(50,936 |
) |
|
$ |
(18,018 |
) |
Net loss per share, basic and diluted |
|
$ |
(0.26 |
) |
|
$ |
(0.61 |
) |
|
$ |
(1.89 |
) |
|
$ |
(0.68 |
) |
Weighted-average shares outstanding, basic and diluted |
|
|
27,062 |
|
|
|
26,574 |
|
|
|
26,970 |
|
|
|
26,559 |
|
Comprehensive loss: |
|
|
|
|
|
|
|
|
||||||||
Net loss |
|
$ |
(6,903 |
) |
|
$ |
(16,222 |
) |
|
$ |
(50,936 |
) |
|
$ |
(18,018 |
) |
Comprehensive loss |
|
$ |
(6,903 |
) |
|
$ |
(16,222 |
) |
|
$ |
(50,936 |
) |
|
$ |
(18,018 |
) |
View source version on businesswire.com: https://www.businesswire.com/news/home/20241107070661/en/
IR and Media Contacts
VP, Head of IR/PR/Marketing
(858) 900-2682
IR@ArcturusRx.com
Source: