What happened

Shares of clinical-stage biotech company Arrowhead Pharmaceuticals (ARWR 0.45%) were up more than 17% Tuesday morning after the company released interim results from a phase 1/2 trial. Arrowhead's shares are down more than 13% this year but up more than 45% this month.

The company focuses on experimental RNA-based therapies that can block the production of proteins that can cause medical disorders. Arrowhead's therapies use the RNA interference mechanism to quickly and durably knock down certain target genes.

So what

Arrowhead released interim results from its ARO-RAGE phase 1/2 trial that showed 90% serum receptor for advanced glycation end products (RAGE) reduction and 80% RAGE reduction with mean maximum reduction after two doses. The protein RAGE is connected to several inflammatory diseases, including asthma.

The company said it plans to announce more details from the trial at the company's research and development day on June 1.

Now what

Arrowhead has had a big month.

  • On April 3, the company said it had earned a $30 million milestone payment from GSK regarding Arrowhead's former investigational RNA therapy, ARO-HSD, now called GSK4532990. It just entered a phase 2 trial to treat patients with non-alcoholic steatohepatitis (NASH).
  • One day later, the company said it was picking up a $40 million milestone payment from Takeda Pharmaceutical because fazirsiran, developed by Arrowhead, was entering a phase 3 trial to treat alpha-1 antitrypsin deficiency associated liver disease.

Those payments will add to momentum for the stock. The company reported first-quarter revenue of $62.5 million, up 128% year over year, and an earnings-per-share (EPS) loss of $0.39, compared to an EPS loss of $0.60 in the same period a year ago. Arrowhead is scheduled to report second-quarter financials on May 2.

Arrowhead also should soon benefit if cardiovascular therapy olpasiran is approved by the Food and Drug Administration. The company has the potential for $400 million in various milestone payments from Amgen regarding the therapy that Arrowhead developed. The drug is in a phase 3 trial.