"Update on Insmed And Their Focus On Getting Arikace Approved For Two Indications"
May 21 2013
"The treatment options for infections in patients with cystic fibrosis (CF) and for patients with infections caused by non-tuberculous mycobacterial lung infections (NTM) may soon improve. At least that is the hope of Insmed inc which have one orphan drug - Arikace (amikacin) - in develop for both indications. Yesterday, the President and CEO of Insmed, Will Lewis spoke at the UBS Global Healthcare Conference in New York City and provided the audience with an update on the clinical trials they have in developement to get Arikace approved - hopefully within the next couple of years.
Cystic Fibrosis and Arikace
There are approximately 30,000 U.S. patients and 35,000 European patients who have CF for a combined market of $500 million. Mr. Lewis noted that these patients have a life expectancy of 37 years and 70% of CF adults have chronic infection die to Pseudomonas aeruginosa. These infections lead to further deterioration in ling function of 1%-3% per year.
Arikace is an antibiotic in a liposomal formulation that along with Insmed’s proprietary nebulizer allows the drug to reach its intended target (lung) more effectively.
Mr. Lewis showed data from there open label phase 2 studies that were initially presented at the 24th Annual North American Cystic Fibrosis Conference in 2010. In that study, patients with CF were randomized to receive Arikace or placebo daily for 28 days followed by 28 days follow-up. Mr. Lewis showed that those patients taking Arikace (560 mg) had significantly higher lung function (FEV1) from baseline and that improvement continued up to 4 weeks following drug remmoval Data from that study can be seen here and here but to date, the data has not been published in a peer review journal.
This sustained FEV1 improvement is very promising in light of the fact that the two other drugs on the market for treating Pseudomonas aeruginosa....."
"...in CF patients are Cayston and TOBI, which according to Mr. Lewis, lose their effectiveness (as measured by FEV1) over a typical 24 week-3 cycle regimen due to drug resistance. Insmed is currently moving forward with a phase 3 trial to compare Arikace with TOBI in 300 patients with CF. The study involves three cycles of treatment (28 days on followed by 28 days off) with the primary end point being relative change in FEV1 . Mr. Lewis state the patient treatment period is now complete and they are currently analyzing the data with the help of key opinion leaders throughout the world. They hope to present the results from that Phase 3 study as soon as possible.
Non-TB Mycobateria Lung Infections and Arikace
The second orphan drug indication discussed by Mr. Lewis was Arikace for non-TB mycobateria lung infections. Currently there are no approved treatments for it and according to Mr. Lewis, most treatments are off label with significant toxicities and limitations (including a systemic formulation of amicacin). Non-TB mycobateria lung infections is a chronic, deliberating progressive infection that can lead to lengthy and repeated hospitalizations. Mr. Lewis also noted that with average hospital stays of 10 days, Insmed will examine the efficacy of Arikace in Non-TB mycobacteria lung infections with the premise that improved efficacy will also reduce hospitalizations and thereby reduce overall costs of care. A concept Insmed refers to as Orphan 2.0 – value for your money.
The company is currently conducting a phase 2 study comparing Arikace to placebo in patients also receiving background therapy. The primary efficacy endpoint will be reduction bacterial density at the end of the 12 week study. Mr. Lewis hopes to have results of the study by the end of the year.
"...that the study was designed in communication with both regulators and key opinion leaders to best create a study design that they hope will lead to regulatory approval. For example, the study also has numerous secondary endpoints (eg, time to sputum conversion, time to pulmonary exacerbation, change in PRO/QoL, 6 minute walk test, change in clinical signs/symptoms, safety) to hopefully encourage the drug to be approved if the drug proves to be effective. We look forward to see that data."