Arrowhead Pharmaceuticals Inc (NASDAQ: ARWR) stock surged 24.03% on June 18th, 2018 and continued its bullish momentum on June 19th, 2018. The company announced that it has completed enrollment of a Phase 1 clinical study of ARO-AAT, the company’s second generation subcutaneously administered RNA interference (RNAi) therapeutic being developed as a treatment for a rare genetic liver disease associated with alpha-1 antitrypsin deficiency. ARO-AAT interferes with genetic messengers to curb production of the mutant protein, preventing it from accumulating in the liver. If successful, ARO-AAT would be the only treatment for liver disease caused by AATD aside from liver transplant.
Moreover, Cantor analyst Elemer Piros explained the bullish reaction on the stock. He believes that it is a strong signal that the company was able to complete Phase 1 enrollment in approximately three months, with potential plans to present data at the American Association for the Study of Liver Disease (AASLD) Conference taking place November 9-13. Late-breakers for the conference are due from September 10-17. The analyst also believe the data presented will be an important read across to the other clinical programs, which also utilize the TRiM platform technology.
Going Forward, Piros highlighted a number of upcoming catalysts for the company. As per the analyst, by 2019, ARWR could have three-four additional internal programs in development. In C4Q18, ARWR expects submitting CTAs for ARO-APOC3 (hypertriglyceridermia), ARO-ANG3 (hypertriglyceridermia), and ARO-ENaC (cystic fibrosis). In 2019, ARWR expects filing a CTA for AROHIF2 (renal cell carcinoma). Data from the Phase 1/2 study of ARO-HBV in patients with chronic hepatitis B virus (HBV) infection could potentially become available in 2018 at AASLD.
However, overall, Piros reiterates an Overweight rating on Arrowhead shares, with a price target of $13, which represents a slight downside potential from current levels.
Meanwhile, AROHBV1001 (NCT03365947) is a Phase 1/2 study evaluating the safety, tolerability, and pharmacokinetic effects of single-ascending doses (SAD) of ARO-HBV in healthy adult volunteers, and evaluating the safety, tolerability, and pharmacodynamic effects of multiple-ascending doses (MAD) of ARO-HBV in patients with chronic HBV.
The SAD portion included 5 cohorts of 6 subjects per cohort. Each SAD subject received a single-dose administration of either placebo or ARO-HBV at dose levels of 35, 100, 200, 300, or 400 mg. The MAD portion, which is ongoing, is designed to include up to 8 cohorts of 4 HBV patients per cohort. Each MAD patient will receive 3 doses of ARO-HBV at up to 4 dose levels (100, 200, 300, 400 mg). The first 2 MAD cohorts at doses of 100 mg and 200 mg have been fully enrolled and the company expects that the third cohort at a dose of 300 mg will be enrolled shortly