Anthera Pharmaceuticals had been hoping to offer patients with cystic fibrosis a new option to help with some of the severe stomach problems they can face as part of their disease. But its experimental drug fell short in late-stage testing, sending shares of the Hayward, CA, company hurtling downward.
Cystic fibrosis is a genetic disease that affects about 70,000 people worldwide, according to the Cystic Fibrosis Foundation, and is characterized by a dangerous buildup of thick, sticky mucus in the lungs. But patients have digestion problems too. The thick mucus clogs ducts in the pancreas that would otherwise release digestive enzymes—a condition called exocrine pancreatic insufficiency—so most CF patients need to take enzyme capsules in order to properly digest their meals. But those drugs, derived from pig enzymes, can lead to other health problems, like viral infections or allergic reactions.
Anthera (NASDAQ: ANTH) developed a drug, liprotamase (Sollpura), meant to be a safer alternative. The drug, which Anthera licensed from Indianapolis-based Eli Lilly (NASDAQ: LLY) in 2014, is made from bioengineered digestive enzymes, so it shouldn’t carry the same safety risks. But liprotomase fell short of its main goal in a late-stage clinical trial Tuesday, failing to sufficiently show it helped with fat absorption. The company’s stock price fell by 66 percent to 70 cents per share late Tuesday.
In initial Phase 3 trial results released Tuesday, Anthera said liprotomase narrowly missed the goal of showing that it was statistically “non-inferior” to treatment with the pancrelipase (Pancreaze)—meaning it didn’t show that its therapy was roughly equal to the pig-derived enzyme. Anthera blamed the design of the clinical trial. The company said that time restrictions and limits on the amount of drug allowed in the trial limited the ability of patients in both groups—on Anthera’s drug or pancrelipase—to increase their doses during the study. Anthera added that liprotamase “may have been under dosed” versus pancrelipase.
While liprotamase failed its main goal, Anthera said that the study confirmed that the drug showed an “appropriate response” in a key measure of protein digestion and absorption. That measure is a key requirement of an FDA approval application, giving the company hope that a better designed trial might have better results. Anthera plans to start a new study that enables investigators to adjust the dose based on the patient’s response. That study should start in the first quarter of 2017.
Anthera said despite the setback, it expects only a modest delay in its previously announced plans to file for FDA approval in the first quarter of 2018.