INSM : NASDAQ : US$15.91
Insmed is focused on developing novel, targeted inhaled
therapies for the treatment of serious orphan lung diseases. Its
lead product candidate is Arikace, a liposomal formulation of FDA
approved antibiotic, amikacin.
All amounts in US$ unless otherwise noted.
Life Sciences — Biotechnology
DATA ANALYSIS ARBITRAGE:
TOTALITY OF NTM DATA SUPPORTS
ACTIVITY, SAFETY AND APPROVAL
Reiterate BUY, $30 PT on Arikace’s potential in nontuberculous
INSM’s lead drug Arikace is an inhaled liposomal form of FDA-approved amikacin. We view the Ph2 US NTM data as
clearly positive, and culture conversion/safety data as supportive of expedited approval. Our $30 target is based on a pNPV analysis.
We see a clear, and likely abbreviated path to approval strongly
supported by culture conversion and QoL data. We think six-month
extension culture conversion, conversion over time, and QoL data to
be presented May 20th at ATS (San Diego) will support a subpart H
filing, a positive AdComm and conditional approval, given what we
see as strong clinically meaningful data in an unmet need.
Primary endpoint statistics confounded by small numbers, unrelated
death. The sensitivity of the primary endpoint Wilcox rank sum
analysis is underscored by the change in p-value from p=0.148 to
p=0.02 when a repeated measures analysis is conducted excluding
the unrelated patient death. We think FDA’s previous strong focus
on cures/QoL (supported by our talks with KOLs) hasn’t changed.
QDIP/GAIN gives FDA the flexibility to focus on the drug’s activity:
we think breakthrough status is highly likely. While some investors
are focused on safety, we think the lack of renal and ototoxicity and
note FDA has approved antibiotics for unmet needs with much more
problematic safety profiles (e.g. Sirturo’s black box warning