Shares of Insmed $INSM rocketed up 133% in premarket trading on Tuesday after the biotech reported that its Phase III study had hit the primary endpoint it believes will persuade the FDA to hand over a breakthrough approval. The data on effectiveness in improving patients’ ability to walk, though, is lacking and could cloud the upcoming shot at an OK.
Researchers in the study say that the inhaled drug Arikayce — now referred to as ALIS — added to standard of care eliminated evidence of a lung infection known as Nontuberculous Mycobacterial (NTM) disease in 29% of patients, compared to only 9% in the standard of care group.
That was a clear hit with investors this morning.
There was no difference between the drug and control groups in 6-minute walk test results, though, a key measure of efficacy. But if you mixed in all the patients from both arms that “achieved culture conversion,” then that group did achieve a faster 6-minute test result. That, however, is not the kind of clear drug efficacy the biotech was hoping for.
Still, investigators note that it’s the elimination of NTM that’s the key goal here, and they believe the data point to an accelerated approval. The FDA, though, will have to decide if the agency agrees with that hopeful assessment.
The drug arm also experienced a slightly higher rate of adverse events — 20.2% versus 17.9%. That didn’t escape the attention of Leerink’s Joseph Schwartz, who still saw more to celebrate than fear in the data. His note:
ALIS led to lower incidence of pt. death (2.7% ALIS vs. 4.5% GBT alone) in addition to cardiac disorders (0.4% ALIS vs. 4.5% GBT alone). Although p-values were not provided for these incidents, the absolute improvements are likely to support ALIS efficacy in conjunction with the primary endpoint of culture conversion. That said, we do see a generally high SAE (20.2% ALIS vs. 17.9% GBT alone), and infections and infestations (9% ALIS vs. 5.4% GBT alone) marked by higher pneumonia infections (3.6% ALIS vs. 1.8%). Since mgmt. had included genotyping in their protocol, we should have more insight on the frequency of re-infections among pts. in the CONVERT study.
This program ran into some serious issues with investors back in 2014, when Insmed disappointed its most enthusiastic backers who thought they would shoot for a quick OK at the time. Plans for the Phase III set that timeline back by several years. But the company believes it was worth it.
“We consider these compelling top-line data to be a remarkable accomplishment in a rare disease state with no currently approved therapies,” said Will Lewis, Insmed’s chief executive. “We are particularly encouraged by the consistency of these data when compared with our Phase 2 study results, and look forward to additional data as the CONVERT study continues over the next two years.”
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