Potential to address unmet need: orphan status, accelerated approval for NTM could give Arikace fast path to US market. We estimate ~50k patients suffer from NTM infections in the US, making it a larger indication than CF. NTM causes significant morbidity and mortality, and there are no approved drugs to treat it. Arikace’s Ph2 NTM trial is currently enrolling (data expected Q4/13), as is a 25-patient compassionate use program. With GAIN Act QIDP status and good Phase 2 data in an orphan unmet need, INSM believes good data from the Ph2+extension could be enough to file an NDA in 2014.
~80% of adult CF patients have chronic P. aeruginosa infection; current Tx options are cumbersome and don’t prevent declines in lung function; if Arikace can stabilize FEV as Ph2 suggests, it’s a home run. TOBI (inhaled tobramycin, Novartis) is the current SOC for CF PA infections. Chemical properties of tobramycin prevent it from fully penetrating mucus present in CF lungs where bacteria reside, limiting its efficacy; its use is still associated with declines in lung function from infection-associated lung damage.
Arikace’s formulation allows it to penetrate into human macrophages (where NTM infections live) and into thick, CF mucus/biofilm to target bacteria. Arikace is a liposomal formulation of the approved and commonly used aminoglycoside antibiotic amikacin. A lipid bilayer (liposome) shell facilitates delivery to areas (inside cells, mucous/biofilm) where standard antibiotics don’t penetrate. Arikace has the potential to be the first effectiive Tx for NTM and an improvement over current inhaled antibiotics for CF.
From March 25 when INSM was $6+, and the commentary was in the context of a projected PPS target of $12. Posted by "jackbassteam" (?) without responsive comments. Guy made a pretty nice call.
Blog seems to be called "Apprentice Millionaire Portfolio", so it must be our temporary poster minorpdf.