5. Arcturus Therapeutics Holdings Inc. (NASDAQ:ARCT)
Short Interest as % of Shares Outstanding: 16.56%
Number of Hedge Fund Investors In Q3 2024: 17
Ark Invest’s Q3 2024 Stake: $48.7 million
Arcturus Therapeutics Holdings Inc. (NASDAQ:ARCT) is another biotechnology company in Cathie Wood’s portfolio that does not generate revenue by selling medicines. The firm makes money from collaborations; however, it is developing drugs to treat high blood ammonia and cystic fibrosis. As Arcturus Therapeutics Holdings Inc. (NASDAQ:ARCT) is a pre-revenue firm, the importance of its under-development drugs is paramount to its hypothesis. Two key drugs in its portfolio are ARCT-801 and ARCT-032 which target the aforementioned diseases. Both these treatments are in Phase 2 trials, and as of its third-quarter earnings update, Arcturus Therapeutics Holdings Inc. (NASDAQ:ARCT) plans to share their data in the first half of 2025. The firm is also developing a coronavirus treatment, and its Phase 3 data shows that the drug can protect against the virus and its variants for as long as six months post-vaccination.
Arcturus Therapeutics Holdings Inc. (NASDAQ:ARCT)’s management commented on its two key drugs during the Q3 2024 earnings call. Here is what they said:
“Now shifting our attention to our mRNA therapeutics franchise, let’s begin with an update on ARCT-032. ARCT-032 is an inhaled messenger RNA therapeutic for cystic fibrosis, and it’s formulated with Arcturus’ lunar delivery technology that differentiates us from our competitors. In September, we received clearance of an investigational new drug application to the U.S. Food and Drug Administration. The FDA clearance of the IND application enables Arcturus to initiate a Phase 2 multiple ascending dose study to evaluate the safety, tolerability, and efficacy of ARCT-032 in people with cystic fibrosis.
Our team is actively engaged in onboarding a substantial number of clinical sites to help us in this effort. We are fortunate to be able to be working closely with the CF Foundation in this process. The Phase 2 study is presently 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. This study will allow us to evaluate FEB lung function improvement in individuals with CF. And I’m very pleased to report that the company is on track to share in-term Phase 2 proof of concept data for our CF program in the first half of 2025. I’ll now move on to our ARCT-810 program. This is our messenger RNA therapeutic candidate for Ornithine Transcarbamylase or OTC deficiency.
Earlier this year, Arcturus announced the expansion of the Phase 2 clinical program of ARCT-810 into the United States. This open label multiple dose study evaluating pharmacodynamics and safety is currently enrolling adults and adolescents requiring clinical management for OTC deficiency. Our placebo-controlled Phase 2 European study has completed the dosing phase. So these concurrent Phase 2 studies in Europe and the U.S. will allow us to evaluate meaningful biomarker changes in individuals with OTC deficiency. And I’m happy to report that the company is on track to share in-term Phase 2 proof of concept data in the first half of 2025.”
