Highlighting Cystic Fibrosis – No existing cure – patients taking 20 to 30 medications a day – Problem with medications now is growth of resistance to antibodies – Tobi is the leader in this space but they acknowledge 85% resistance growth – Tobi taken twice daily for 20 minutes each - The two leading drugs indicate downward trend line and lung function decline –
Arikace – Taken once daily [inhaled] for 12 minutes – Chemically charged Liposomes [high efficiency drug encapsulation] gets absorbed and penetrates the mucus/biofilm layer of the lung and gets directly to the site of the infection where Amikacin [the encapsulated drug] gets delivered – Statistically significant and exciting data indicates [from their previous phase II extension study] that Arikace has a sustained benefit, even when the patient is NOT receiving drug – It is very unique in this feature.
The phase III trial results coming mid 2013 head to head Arikace against Tobi – For European approval all that is needed is to demonstrate NON-INFERIORITY to Tobi drug. His comment about interest in Arikace.., this trial enrolled in 6 months when it took the other leaders 1 year to enroll before they receive approval
Highlighting NTM – No approved treatment – More common than CF – Growing 8% per year – Over age 65 you are 40% more likely to die when diagnosed with NTM – Off label drugs treating this disease, none penetrate the lung.
Arikace – Very good localized delivery to the lung – Delivers macrophage penetration – ‘Elite compound in this space’ – NTM is ‘ours to own’ – The phase II data’s primary endpoint is reduction of bacterial density by day 84 – Secondary endpoints as well – He believes with primary endpoint accomplishment, suitable for registration – potential for accelerated approval based on endpoint achievement, gain act, orphan and compassionate use launch after CF trial results – Will have dialogue with FDA as they met last September discussing endpoints .
Insmed – Is preparing to launch based on both getting approved – 44 employees and growing – 2 year stability data from the manufacturer – Market exclusivity to 2029 but likely into the 3030’s