RARE Daily

Arcturus Therapeutics Raises $150 Million in Public Offering

December 8, 2020

Rare Daily Staff

Arcturus Therapeutics, which is developing mRNA for infectious disease vaccines and treatments rare liver and respiratory diseases, raised $150 million in a public offering of 1.4 million shares of its common stock at $110.00 per share.

Arcturus also granted the underwriters a thirty-day option to purchase up to an additional 204,750 shares of common stock at the public offering price, less underwriting discounts and commissions.

The company intends to use proceeds of the offering to conduct clinical trials and to begin manufacturing its LUNAR-COV19 vaccine candidate for stockpiling purposes.

In rare diseases, the company recently began dosing in a phase 1b study of ARCT-810 in patients with Ornithine Transcarbamylase (OTC) deficiency, a serious urea cycle disorder with a prevalence of approximately 10,000 worldwide. A lack of the OTC enzyme in liver cells results in high blood ammonia levels and can cause seizures, coma, and death in untreated patients. There are no U.S. Food and Drug Administration approved medicines for OTC deficiency.

ARCT-810 utilizes Arcturus LUNAR lipid-mediated delivery platform to deliver OTC messenger RNA to liver cells. Expression of OTC enzyme in the liver of patients with OTC deficiency has the potential to restore normal urea cycle activity, preventing neurological damage and the need for liver transplantation. The ARCT-810 program is supported by preclinical data in OTC deficiency murine models demonstrating that dosing of LUNAR-OTC results in robust ornithine transcarbamylase protein expression and activity resulting in improvements in ureagenesis and plasma ammonia and in increased survival.

 

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