Arcturus: Potential Based On Rare Disease Therapy ARCT-810 (NASDAQ:ARCT)

Arcturus Therapeutics Holdings Inc. (NASDAQ:ARCT) is a great speculative biotech play to look into. That’s because it is gearing up to begin a phase 2 study using ARCT-810 for the treatment of patients with a rare disease known as ornithine transcarbamylase (OTC) deficiency. It is gearing up to dose the first patient in mid-2022 with interim results from the study expected in the 2nd half of 2022. Not only that, but it has shown that its two-dose vaccination of 5 mcg doses of ARCT-154 COVID-19 vaccine as effective in reducing severe (fatal) disease by 95%. The data generated from this has been submitted for Emergency Use Authorization (EUA) in Vietnam April 13 of 2022. If all goes accordingly, then it will receive EUA in Vietnam for ARCT-154. Not only that, but it released 3-month booster data for ARCT-154 in May of 2022. Based on all these factors, these are the reasons why I believe that Arcturus is a great speculative biotech play to look into.

EUA for ARCT-154 Submitted To Vietnam

Arcturus established proof of concept in its phase 1/2/3 registrational study for Vietnam. It recruited a total of 19,000 adults, including those who were at higher risk of having severe complications of COVID-19 disease. It was shown that two-dose vaccination series of 5 mcg doses of ARCT-154 established 55% efficacy protection against COVID-19, which was the primary endpoint. Not too bad, but the secondary endpoint was far more important in my opinion. The secondary endpoint was severe COVID-19 disease (including COVID-19 related deaths) that occurred. The bottom-line here is that ARCT-154 demonstrated vaccine efficacy of 95% against severe (fatal) COVID-19 disease. Why do I think this is more of an important endpoint? That’s because reducing hospitalization or death is far more important compared to just only possible prevention of getting COVID-19. Regardless, this data is very good and I believe it should get EUA in Vietnam. These positive results have been submitted to the Vietnam Ministry of Health on April 13, 2022. If all goes well, then Arcturus expects to potentially receive EUA for ARC-154 in Vietnam.

Not only that, but it soon reported some initial findings from its phase 1/2 booster study in the United States and Singapore using ARCT-154 as a booster vaccination. It was shown that the booster vaccination generated strong immune responses for at least 3 months post-boost. These measures occurred both with respect to magnitude of response and duration of response. About 12 patients first received two primary doses of Comirnaty. Then at least 5 months after receiving two primary Comirnaty doses, patients were given 5 mcg of ARCT-154. It was also observed after 3 months that the ARCT-154 booster helped patients achieve persistent neutralizing antibody activity against multiple variants of SARS-Cov-2. As such, Arcturus has initiated start-up process with an international CRO so that it can begin a phase 3 booster study, which will ultimately be used to support global registration.

ARCT-810 For Treatment Of Patients With Ornithine Transcarbamylase Deficiency

ARCT-810 is being developed for the treatment of patients with Ornithine Transcarbamylase Deficiency, which is a rare genetic disease. It is a rare disease that only affects around 10,000 people worldwide. Ornithine Transcarbamylase (OTC) Deficiency occurs when there is a lack of an enzyme (known as OTC) responsible for eliminating ammonia from the body. Ammonia comes about when protein is broken down by the body. It is okay to have low levels of ammonia because the liver constantly handles the breakdown of it. With these patients lacking OTC, ammonia levels go sky high, thus in turn causing several symptoms such as:

• Coma
• Death
• Mental status change
• Headaches
• Lack of energy/enthusiasm to do things

How exactly can Arcturus help these patients? Well, it is hoping to use its proprietary LUNAR delivery platform technology with mRNA ARCT-810 to deliver healthy functional OTC enzymes into their liver cells. The hope is that this drug will be able to help Ornithine Transcarbamylase Deficiency patients achieve normal levels of the urea cycle function (have normal level of ammonia in the blood) and possibly increase survival. The reason for starting this program is that preclinical studies have yielded some good outcomes in that LUNAR delivery with mRNA delivered to liver cells in a mouse model showed restoration of normal levels of OTC. If this can be replicated in humans, then it would really be a game changer. Why? That’s because the only known cure at the moment is a liver transplant.

As such, it had already completed a phase 1 safety study in humans which showed that ARCT-810 was safe to use. It expects to dose the first patient in a phase 2 randomized, double-blind, placebo-controlled study using ARCT-810 to treat these Ornithine Transcarbamylase Deficiency patients soon in mid-2022. This mid-stage study is expected to enroll up to 24 patients across two different dose levels. The primary endpoint for this study will be safety. However, the secondary endpoint is going to measure urea cycle function over a 12-week treatment period. That is, to see if ARCT-810 can help restore OTC enzymes necessary for clearing excess ammonia in the blood. It is expected that Arcturus will report interim results from this study in the 2nd half of 2022 which would be a very important catalyst for investors.

Financials

According to the 10-Q SEC Filing, Arcturus Therapeutics Holdings Inc. had cash and cash equivalents of $319.7 million as of March 31, 2022. The biotech is likely to burn through a significant portion of its cash in 2022. This will be due to the advancement of the COVID-19 booster trial and other flu programs it has in its pipeline. Not only that, but it must continue funding the phase 2 study using ARCT-810 for OTC deficiency and to submit a Clinical trial authorization (CTA) for its ARCT-032 program which is expected in Q3 of 2022. ARCT-032 will be used to treat patients with Cystic Fibrosis (CF). The biotech believes that it has enough cash on hand to fund its operations into late 2023.

Risks To Business

There are several risk factors that investors should be aware of. The first major risk deals with the release of results from the phase 2 study using ARCT-810 for the treatment of patients with OTC deficiency which is expected 2nd half of 2022. This mid-stage study will be initiated soon in mid-2022 first though. The second risk deals with the use of ARCT-154 both as a vaccine and a booster. There is no guarantee that the EUA for Vietnam for ARCT-154 will be approved. As I stated above, a phase 3 global registration trial for ARCT-154 as a booster vaccine will likely be initiated in 2022. Arcturus has already begun the start-up process with a CRO so that it can begin this late-stage booster study. The final risk deals with the financial position. Even though it is estimated that it has enough cash to fund its operations into late 2023, doesn’t mean it will wait until then to do so. As such, I expect a cash raise at least in the 1st half of 2023.

Conclusion

The final conclusion is that Arcturus Therapeutics Holdings Inc. is a great speculative biotech to look into. It has potential to receive EUA for ARCT-154 as a COVID-19 vaccine in Vietnam in 2022. Not only that, but the company is already in the process of initiating a pivotal phase 3 global registration study for ARCT-154 as a booster vaccine for COVID-19 as well. This is another catalyst that may ultimately play out for the pipeline. Lastly, it has several shots on goal with ARCT-032 for treatment of patients with Cystic Fibrosis and ARCT-810 for patients with OTC deficiency. With a few catalysts approaching, plus a pipeline full of several ongoing programs, these are the reasons why I believe that Arcturus Therapeutics is a great speculative buy.