The treatment options for infections in patients with cystic fibrosis
(CF) and for patients with lung infections caused by non-tuberculous mycobacteria
may soon get a little better. At least that is the hope of the people at Insmed inc, who are developing Arikayce (amikacin) for both indications.
Recently, 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 development to get Arikayce approved – and hopefully within the next couple of years.
Cystic Fibrosis and Arikayce
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 due to Pseudomonas aeruginosa
. These infections lead to further deterioration in lung function of 1%-3% per year.
Arikayce (amikacin) 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.
In an open label phase 2 study initially presented
at the 24th
Annual North American Cystic Fibrosis Conference in 2010, patients with CF were randomized to receive Arikayce or placebo daily for 28 days followed by 28 days follow-up. Patients taking Arikace (560 mg) had significantly higher lung function (FEV1
) from baseline and that improvement continued up to 4 weeks following drug removal. Data from that study can be seen here and here although the study has not been published in a peer review journal yet.
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
, 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 stated 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 soon.
Non-Tuberculosis Mycobateria Lung Infections and Arikayce
The second orphan drug indication that Arikace is seeking is for non-TB mycobacteria lung infections, which current has no approved treatment. According to Mr. Lewis, most treatments are off-label with significant toxicities and limitations (including a systemic formulation of amikacin).
A non-TB mycobacteria lung infection is a chronic, deliberating progressive infection that can lead to lengthy and repeated hospitalizations. Mr. Lewis 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 Arikayce 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 stated thy hope to have results of the study by the end of the year.
Image of amikacin obtained from wikimedia commons