RNA/mRNA Medicine Platforms in Rare Diseases and Vaccines

Platforms

Partnerships Maximize Platform
Program
Partner
Indication
Program:
LUNAR-HBV
Partner:
Indication:
Hepatitis B Virus (HBV)
Program:
LUNAR-NASH
Partner:
Indication:
Nonalcoholic Steatohepatitis (NASH)
Program:
LUNAR-GSD3
Partner:
Indication:
Glycogen Storage Disease Type III
Program:
LUNAR-RARE
Partner:
Indication:
Undisclosed Rare Disease
Program:
LUNAR-COV19
(ARCT-021)
Partner:
Undisclosed Global Entity
Indication:
COVID-19 Vaccine
Program:
LUNAR-COV19
(ARCT-154)
Partner:
Indication:
COVID-19 Vaccine
Greater than $1 Billion in Potential Milestones & Royalties
More than $1 Billion in Potential Milestones and Royalties
DiscoveryPreclinicalClinical
name

LUNAR-GSD3

indication
Glycogen Storage Disease Type III
api
mRNA

COLLABORATION PROGRAM

Ultragenyx

name

LUNAR-RARE

indication
Undisclosed Rare Disease
api
mRNA

COLLABORATION PROGRAM

Ultragenyx

name

LUNAR-HBV

indication
Hepatitis B
api
RNA

COLLABORATION PROGRAM

Janssen

name

LUNAR-NASH

indication
NASH
api
RNA

COLLABORATION PROGRAM

Takeda

name

LUNAR-COV19
(ARCT-021)

indication
COVID-19
Single Shot
api
STARR™ mRNA

COLLABORATION PROGRAM

Undisclosed
Global Entity

name

LUNAR-COV19
(ARCT-154)

indication
COVID-19
Targeting Variants of Concern
api
STARR™ mRNA

COLLABORATION PROGRAM

Vingroup

We have partnered with a number of industry leaders and have a robust and diverse preclinical and clinical drug development pipeline.

Our external development partners include Janssen Pharmaceuticals, Inc., Ultragenyx Pharmaceutical Inc., Takeda Pharmaceutical Company Limited, CureVac AG and Synthetic Genomics, Inc.



LUNAR-HBV

Arcturus has partnered with Janssen Pharmaceuticals, Inc., a Johnson & Johnson company, to develop nucleic acid medicines for the treatment of hepatitis B virus infection and other diseases.

Hepatitis B
Hepatitis B is caused by the Hepatitis B Virus (HBV) and affects more than 350 million people worldwide. It is a major cause of liver disease including cirrhosis and liver cancer. There is no cure for chronic HBV infection. In most cases of chronic hepatitis B, antiviral medication suppresses the virus, but does not clear it completely. Therefore, these medications must be taken for life. Unfortunately, many of these medications have common side effects, including fever, headaches, hair loss, and mental problems. As soon as a patient comes off the medication the virus can return.

Our LUNAR-HBV solution
Arcturus and Janssen are developing a nucleic acid medicine to fight HBV without the same side effect issues that plague existing therapies.

LUNAR-NASH

Arcturus, in partnership with Takeda Pharmaceutical Company Ltd., is developing RNA medicines for the treatment of nonalcoholic steatohepatitis (NASH) and other gastrointestinal disorders, utilizing our wholly-owned therapeutic delivery platform, LUNAR®.

Nonalcoholic steatohepatitis (NASH)
Nonalcoholic steatohepatitis (NASH) is the most serious form of nonalcoholic fatty liver disease (NAFLD) and affects 3.5-5% of Americans. NASH is predicted to become the leading cause of end-stage liver disease, liver transplant, and hepatocellular carcinoma in the next two decades. There are no FDA-approved drugs to treat NASH. Doctors currently recommend weight loss and increased exercise to control fat deposition, fibrosis and inflammation in the liver.

Our LUNAR-NASH solution
Takeda and Arcturus are collaborating to develop RNA-based therapeutic candidates to treat NASH. Current program activities include lead candidate identification and duration of action studies in various models of disease.

LUNAR-GSD3

Arcturus has partnered with Ultragenyx to develop messenger RNA (mRNA) therapeutic candidates for certain rare disease targets. The first disclosed indication is Glycogen Storage Disease type III.

Glycogen Storage Disease type III
Glycogen Storage Disease type III (GSD3) is a genetic disease affecting 1 in 100,000 Americans. GSD3 is caused by mutations in the AGL gene that allow glycogen to accumulate in cells. This can cause low blood sugar levels, muscle weakness and liver damage. There are no specific treatments for GSD3.

Our LUNAR-RARE solution
Arcturus and Ultragenyx are developing mRNA medicines to replace the defective AGL gene product and allow cells to breakdown glycogen using normal pathways.