 |
| Kamada CEO David Tsur |
Israeli biotech Kamada ($KMDA) watched its shares dive more than 30% on Friday after its treatment for a rare inherited disorder failed to beat out placebo in a pivotal trial.
The drug, AAT, is designed to quell a protein deficiency linked to an increased risk of lung and liver disease. In a Phase II/III trial on 168 patients lacking alpha-1 antitrypsin, the treatment performed just as well as placebo in delaying hospitalizations, the study's primary endpoint. In what Kamada called a "very important" secondary endpoint, the drug did lower the frequency of severe events by 50%, and the inhaled AAT "indicated positive trends" in lung function. The company said it will release a more detailed analysis of the results in the coming months.
Despite the miss on a primary goal, Kamada still believes its data can support a European regulatory application for AAT, and CEO David Tsur said in a statement that, "based on efficacy and ease of use, we shall have a competitive advantage in a $1 billion market."
Investors were less than convinced, however, sending Kamada's shares down roughly 33% to $9.21 on Friday. Kamada pulled off a $52 million IPO last year due in part to the potential of AAT, and the biotech's latest dive takes its share price below its $9.25 debut for the first time.
Kamada is in the midst of a Phase II trial on AAT, and the company plans to combine data from study with the Phase II/III to support an FDA application for the treatment. Italy's Chiesi Farmaceutici has signed up to commercialize the drug in Europe if and when it wins approval, a deal that could mean up to $60 million for Kamada.
Alpha-1 antitrypsin deficiency affects between 1 in every 1,600 people and 1 in every 5,000 people, according to the National Institutes of Health.
- read Kamada's filing