Arcturus Therapeutics Provides Updates for Ornithine Transcarbamylase (OTC) Deficiency and Cystic Fibrosis (CF) Programs
Arcturus Therapeutics (ARCT), a company specializing in mRNA medicines, provided updates on its ARCT-810 and ARCT-032 programs. The ARCT-810 Phase 2 study for OTC deficiency completed enrollment in Europe, with data expected later this year. This program is expanding in the U.S. to include more severely affected patients. ARCT-032, aimed at treating cystic fibrosis (CF), will submit an IND for a Phase 2 multiple ascending dose study within 60 days, based on positive safety data from previous trials. Key updates include the completion of subject enrollment in Europe for ARCT-810, the expansion of this program in the U.S., and promising preliminary results for ARCT-032 in treating CF patients.
- ARCT-810 Phase 2 study in Europe completed enrollment; data expected later this year.
- ARCT-810 program expanding in the U.S. for more severely affected patients.
- ARCT-032 IND application for Phase 2 study to be submitted within 60 days.
- No serious adverse events observed in ARCT-032 trials to date.
- None.
Insights
Arcturus Therapeutics continues to make strides in their mRNA therapeutics development, particularly for rare diseases like OTC deficiency and cystic fibrosis. The ARCT-810 program's advancement to a Phase 2 study in Europe, with the expansion to include more severe cases in the U.S., indicates a robust commitment to addressing unmet medical needs.
For retail investors, it’s important to recognize the significance of completing enrollment in clinical trials. This milestone suggests the company is progressing as planned, reducing uncertainty about potential delays. Furthermore, Arcturus’s decision to include more severely affected patients in their U.S. trial could address a wider patient population, potentially increasing the market size for ARCT-810 if proven effective.
In the case of ARCT-032 for cystic fibrosis, the upcoming IND submission for a Phase 2 study is significant. The absence of serious adverse events thus far is promising for the safety profile of the drug, critical for obtaining regulatory approval. While early improvements in lung function in a patient with Class I mutations show potential, investors should be cautious as these results are preliminary. The success of the Phase 2 study will be pivotal for the drug’s future prospects.
Retail investors should note that while ongoing clinical trials are a positive sign, they are not without risk. The final outcome and regulatory approval are still uncertain and the results from these trials will provide more definitive data on efficacy and safety.
From a financial perspective, the updates on ARCT-810 and ARCT-032 offer a mixed bag of opportunities and uncertainties. The completion of enrollment for the Phase 2 study of ARCT-810 is a milestone that suggests that the company is adhering to its timeline, which is a positive signal for investors. Timely progression in clinical trials often correlates with increased investor confidence and potentially stock price appreciation.
The potential expansion of ARCT-810 to include more severe OTC deficiency cases in the U.S. could also expand its addressable market, thus increasing revenue potential if the drug is approved. However, investors should be cautious of the costs associated with expanding such programs, as they can significantly impact the company’s cash runway and profitability in the short term.
Regarding ARCT-032, the imminent IND submission for a Phase 2 study reflects the company’s proactive approach in advancing its pipeline. The safety data collected so far is encouraging and moving into Phase 2 could attract more investor interest. Nevertheless, the financial implications of further clinical trials will need to be monitored closely, as these studies are capital-intensive.
In summary, while the updates are promising and could lead to future revenue streams, the financial burdens and risks associated with ongoing and expanded clinical trials should be carefully considered by investors.
ARCT-810 Phase 2 study completed enrollment at 0.3 mg/kg dose level in
ARCT-810 to expand Phase 2 clinical program in the
ARCT-032 IND for Phase 2 multiple ascending dose study to be submitted in the next 60 days
“CFTR replacement using mRNA therapeutics is an area of significant medical interest, as it may benefit null patients and potentially many other CF patients,” said Dr. Juergen Froehlich, Chief Medical Officer of Arcturus Therapeutics. “The safety profile of ARCT-032 observed in Phase 1 and after two administrations in Phase 1b for patients dosed to date, including the trend of lung function improvement in the Class I participant, is encouraging. These data are supportive of expanding our clinical program to the
ARCT-810 (OTC Deficiency) Update
The double blind ARCT-810 Phase 2 study in the EU and
ARCT-810 is expanding the Company’s clinical program in the
ARCT-032 (CF) Update
The Company plans to submit an IND application in the next 60 days for an ARCT-032 Phase 2 multiple ascending dose study designed to identify a safe and effective dose in Class I (null) and other CF participants who do not benefit from CFTR modulators. This study is supported by safety and tolerability data collected in healthy volunteers (N = 32) and the ongoing two-administration Phase 1b study. No serious adverse events (SAEs) have been observed in any clinical trial participants to date. No febrile reactions have been observed within the target dose range of the planned Phase 2 study. The Phase 1b study is ongoing with the seventh and final participant scheduled to be dosed soon. Of the seven total CF participants in Phase 1b, six are receiving CFTR modulator treatment while one subject has Class I mutations and therefore does not benefit from modulator therapy. The Class I CF subject had low lung function at baseline (ppFEV1 below
About Ornithine Transcarbamylase Deficiency
Ornithine transcarbamylase (OTC) deficiency is the most common urea cycle disorder. Urea cycle disorders are a group of inherited metabolic disorders that make it difficult for affected patients to remove toxic waste products as proteins are digested. OTC deficiency is caused by mutations in the OTC gene which leads to a non-functional or deficient OTC enzyme. OTC is a critical enzyme in the urea cycle, which takes place in liver cells, and together with the other enzymes in the urea cycle converts ammonia to urea. This conversion does not occur properly in patients with OTC deficiency and ammonia accumulates in their blood, acting as a neurotoxin and liver toxin. A lack of the OTC enzyme in liver cells can results in high blood ammonia levels and cause seizures, coma, and death in untreated patients. OTC deficiency is an inherited disease that can cause developmental problems, seizures and death in newborn babies. It is an X-linked disorder, so is more common in males. Patients with less severe symptoms may present later in life, as adults. There is currently no cure for OTC deficiency, apart from liver transplant. However, this treatment comes with significant risk of complications such as organ rejection, and transplant recipients must take immunosuppressant drugs for the rest of their lives. Current standard of care for OTC deficiency patients is a well-controlled, but challenging to maintain, low-protein diet and treatment with ammonia scavengers to try and prevent patients from accumulating ammonia. These treatments do not address the underlying cause of disease. In
About ARCT-810
ARCT-810 is an intravenously administered investigational mRNA therapeutic designed to express normal functional OTC enzyme in the liver of individuals with OTC deficiency. ARCT-810 has received Orphan Medicinal Product Designation from the European Medicines Agency (EMA), including an approved pediatric investigation plan (PIP), and Orphan Drug Designation along with Rare Pediatric Disease Designation from the
About Cystic Fibrosis
Cystic fibrosis is a life-shortening disease with a worldwide prevalence. Mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene result in a reduction or absence of CFTR protein and/or function in the airways, causing insufficient chloride transport to maintain airway surface homeostasis. CF mucus is more difficult to clear, thus clogging the airways and leading to infection, inflammation, respiratory failure, or other life-threatening complications. Standard of care for many CF individuals include CFTR modulators. Nearly 40,000 people in the
About ARCT-032
ARCT-032 is an inhaled investigational mRNA therapeutic designed to express normal functional CFTR in the lungs of individuals with CF. ARCT-032 has received Orphan Medicinal Product Designation from the European Medicines Agency (EMA) and Orphan Drug Designation along with Rare Pediatric Disease Designation from the
About Arcturus Therapeutics
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 (including safety and efficacy) of ARCT-810 or ARCT-032, the likelihood that the trend of lung function changes in the Class I participant for ARCT-032 will continue or provide meaningful efficacy, the expected timing for the safety and biomarker data for ARCT-810, the planned expansion of the ARCT-810 clinical program and the Company’s ability and decision to enroll and conduct such expansion, the continued clinical development of ARCT-810, the potential for CFTR replacement to benefit null patients or other CF patients, the continued clinical development of ARCT-032 including the likelihood of and timing for submission of an IND for and plans to conduct a Phase 2 study, the likelihood that clinical results received to date (including data in the ARCT-032 Phase 1b study) will be predictive of future clinical results or sufficient to further advance the CF program, the likelihood of and timing for expanding the CF clinical program and the impact of general business and economic conditions. 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. These statements are only current predictions or expectations, and are subject to known and unknown risks, uncertainties, and other factors that may cause our or our industry’s actual results, levels of activity, performance or achievements to be materially different from those anticipated by the forward-looking statements, including those discussed under the heading "Risk Factors" in Arcturus’ most recent Annual Report on Form 10-K, and in subsequent filings with, or submissions to, the SEC, which are available on the SEC’s website at www.sec.gov. Except as otherwise required by law, Arcturus disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date they were made, whether as a result of new information, future events or circumstances or otherwise.
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.
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IR and Media Contacts
Arcturus Therapeutics
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Source: Arcturus Therapeutics Holdings Inc.
FAQ
What is the status of the ARCT-810 Phase 2 study?
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