From March 27th (reiteration of $30)) "breakthrough status"
Reiterate BUY, $30 PT on Arikace's potential in nontuberculous
mycobacteria (NTM). 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
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).
[ 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). ]
Sirturo is a brand name for ..... ?
The good news is that you won't have to go very far to find a clue to the answer.
The really depressing news is that the clue wasn't on the front page of the board during the three-day break :-(
Bedaquiline (trade name Sirturo, code names TMC207 and R207910) is a diarylquinoline anti-tuberculosis drug, which was discovered by a team led by Koen Andries at Janssen Pharmaceutica. It is the first new medicine to fight TB in more than forty years, and is specifically approved to treat multi-drug-resistant tuberculosis. WARNING:
•An increased risk of death was seen in the SIRTURO™ treatment group (9/79, 11.4%) compared to the placebo treatment group (2/81, 2.5%) in one placebo-controlled trial. Only use SIRTURO when an effective treatment regimen cannot otherwise be provided.
•QT prolongation can occur with SIRTURO. Use with drugs that prolong the QT interval may cause additive QT prolongation.
SIRTURO (bedaquiline) for oral administration is available as 100 mg strength tablets. Each tablet contains 120.89 mg of bedaquiline fumarate drug substance, which is equivalent to 100 mg of bedaquiline. Bedaquiline is a diarylquinoline antimycobacterial drug.