Arcturus Metabolic Disorder Candidate Gets Orphan Drug Status
Zacks Investment Research | Jun 30, 2019 09:26PM ET
Arcturus Therapeutics (NASDAQ:ARCT) announced that the FDA has granted an orphan drug designation to its pipeline candidate, ARCT-810, for the treatment of ornithine transcarbamylase deficiency (“OTCD”), an inherited metabolic disorder. OTCD is the most common urea cycle disorder, which impacts an individual’s ability to remove toxic waste products from the body.
ARCT-810 is a RNA-based pre-clinical candidate, which has been designed using the company’s proprietary LUNAR lipid-mediated delivery platform. The company is planning to submit an investigational new drug application to the FDA in early 2020 to initiate clinical study on ARCT-810.
Notably, the orphan drug designation is granted to drugs capable of treating rare diseases that affect less than 200,000 people in the United States. The designation also makes a company entitled to certain other benefits including tax credits related to clinical trial expenses and exemption from the FDA user fee.
The status makes ARCT-810 eligible for seven years of marketing exclusivity in the United States.
Apart from OTCD, the company has collaborated with Cystic Fibrosis Foundation for developing RNA-based therapy to treat cystic fibrosis.
Arcturus’ shares have rallied 108.4% so far this year compared with the industry ’s increase of 5.4%.
Multiple pharma/biotechs have signed collaborations with Arcturus to use its RNA-based technology to develop treatments that target different indications.
Last month, the company expanded its existing collaboration with Ultragenyx Pharmaceutical (NASDAQ:RARE) to discover and develop mRNA, DNA and siRNA therapeutics for up to 12 rare disease targets. Pursuant to the agreement, Ultragenx will pay $30 million of upfront payments to Arcturus, including $6 million cash for collaboration agreement amendment and $24 million equity investment at $10 per share. Arcturus is also entitled to preclinical, clinical, regulatory and sales milestone payments for each product developed under the collaboration. With the agreement, Ultragenyx is now Arcturus’ largest shareholder. Ultragenyx has an option to purchase an additional 600,000 shares of Arcturus’ common stock at $16 per share.
Arcturus has an agreement with Takeda Pharmaceutical (NYSE:TAK) for the development of therapies targeting nonalcoholic steatohepatitis and with J&J (NYSE:JNJ) for the treatment of hepatitis B.
The company completed the change of its domicile from Israel to Delaware last month.
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