Pipeline updates are highly awaited events in the pharma/biotech sector as they play an important role in deciding whether or not to invest in a particular company. These updates provide information on experimental drugs and at times give an insight into the commercial potential of the candidate once it is successfully developed and commercialized.
Insmed Inc. (INSM) recently announced positive additional data from a phase II trial on pipeline candidate, Arikayce, at the American Thoracic Society’s Annual Meeting 2014.
Insmed is evaluating candidate Arikayce for the treatment of patients suffering from treatment resistant nontuberculous mycobacterial (:NTM) lung infections.
The randomized, double-blind phase II study (n=89) evaluated Arikayce plus standard of care treatment vis-à-vis standard of care treatment plus placebo in treatment resistant NTM lung infected adults for 84 days. These patients were on American Thoracic Society/Infectious Disease Society of America (ATS/IDSA) guideline therapy for a minimum period of six months before screening.
These patients also continued to have positive mycobacterial cultures.
Post randomization, the eligible patients had the option to receive Arikayce once daily for an additional 84 days in an open-label design.
It was observed that 21 of the 68 patients eligible were culture negative for NTM at the end of 168 days. In addition, the number of patients with negative cultures increased from the double blind phase of the trial.
The encouraging additional data should somewhat relieve the investors. Insmed suffered a setback in Mar 2014 when Arikayce failed to meet the primary objective in the double blind phase of the trial.
The primary objective of the phase II study was a semi-quantitative measurement of the difference in mycobacterial density on a seven-point scale from baseline to the end of the randomized portion of the study. However, Arikayce failed to meet the pre-specified level for statistical significance.
Consequently, Insmed now plans to provide the trial results to regulatory agencies in the U.S. and Europe in the coming months to determine the future development of Arikayce.
We note that Arikayce enjoys orphan drug designation for treating NTM both in the U.S. and EU. Insmed also applied for Breakthrough Therapy Designation for Arikaycee in the U.S in Apr 2014.
The candidate also enjoys Fast Track designation for NTM in the U.S.
Insmed is also developing Arikayce for the treatment of pseudomonas aeruginosa in patients suffering from cystic fibrosis.
Insmed currently does not have any approved drug in its portfolio. Hence, the successful development of the candidate is important for the company’s growth. We expect investor focus to remain on further updates on Arikayce.
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