Insmed to go ask for Alis accelerated approval

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Insmed’s anti-infective Alis scored a persuasive win in its phase III trial in non-tuberculous mycobacterial lung disease, prompting the group to seek accelerated approval from the US FDA.

Shares in the New Jersey-based group more than doubled as sales forecasts of $497m in 2022 became more realistic after the announcement that the inhaled amikacin on top of usual care outperformed usual care alone. With post-approval commercial concerns on executives’ minds and a jump in valuation putting Insmed over the $1bn threshold, a fundraising seems like the next step to build up cash reserves.

Alis, for amikacin liposome inhalation suspension and formerly known as Arikayce, plus guideline-based therapy beat guideline therapy alone in converting 29% of patients from positive to negative cultures at six months for non-tuberculous mycobacterial (NTM) disease caused by mycobacterium avium complex. In patients treated with guideline therapies the conversion rate was 9% – the trial was designed to detect a difference of at least 15 percentage points at a statistically significant level.

This focus on conversion from positive to negative mycobacterium avium complex grew from a mixed phase II trial in which Alis missed on the endpoint of reduced bacterial density in the broad NTM population (Upcoming events – Late-stage data due for Amicus and Insmed, August 18, 2017). This phase III trial included another endpoint, a six-minute walk test, which as the company notes is not an endpoint used in clinical practice and in any case missed showing any significant improvement.

Nonetheless, patient-centred outcomes are of increasing interest to the FDA and patients who did convert from positive to negative taking Alis also demonstrated an improvement in six-minute walk test, a pre-specified analysis.

Longer than six months

In the meantime, Insmed has an ongoing open-label extension that includes patients who did not achieve culture conversion in this phase III, data from which could be included in its new drug application. Leerink analyst Joseph Schwartz wrote recently that the commercial opportunity includes patients who will need treatment for longer than six months and those who experience re-infections.

An unanswered question is whether Insmed will persuade hospitals to buy Alis rather than formulating their own inhaled amikacin from IV formulations. Mr Schwartz wrote that only five or six hospitals specialise in NTM, so it may involve convincing only a few physicians on convenience grounds.

Even if that is a small audience that can be reached with a small sales force, it would not hurt Insmed to build up its cash supply to finance its post-approval commercial phase. It had $91m cash as of June 30, but opportunities this ripe for a fundraising do not happen every day.

With breakthrough therapy and qualified infectious disease product designations from the FDA, Alis should get a positive reception from agency staff. As long as no safety signals crop up, it seems odds-on to get approval – investors’ next focus needs to be commercial execution.

To contact the writer of this story email Jonathan Gardner in Virginia at jonathang-us@epvantage.com or follow @ByJonGardner on Twitter

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