Upcoming Events - Late-Stage Data Due For Amicus And Insmed

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Welcome to your weekly digest of approaching regulatory and clinical readouts. Amicus (NASDAQ:FOLD) is awaiting phase III data for SD-101, a potential topical epidermolysis bullosa treatment that carries a rare paediatric disease designation, meaning that the company could gain a valuable priority review voucher.

Also by September Insmed (NASDAQ:INSM) expects phase III data for the inhaled antibiotic amikacin, its lead product. In an earlier study in bacterial lung infections this missed its primary endpoint but hit on secondary measures, which comprise the primary analysis in the latest trial.

Rare skin disorder

Data from Amicus's registrational phase III Essence study, in 160 patients with epidermolysis bullosa aged one month and older, are due late in the third quarter.

This involves application of 6% SD-101 cream or placebo topically once a day to the entire body for 90 days, and measures time to wound closure within three months as first co-primary endpoint; the second, assessed sequentially, is the proportion of patients with complete closure of their target wound, as selected by the study investigator.

In phase II the 6% dose showed 67% complete target wound closure after one month, the trial's primary endpoint, but this fell short of statistical significance. It would have hit significance at two months had this been the endpoint, explaining the three-month cutoff in phase III.

SD-101, previously known as Zorblisa, has US orphan drug designation, and in May it received rare paediatric disease designation, making it eligible to receive an FDA priority review voucher if approved. When Amicus gained it through its Scioderm acquisition it said if a voucher was obtained and sold on Scioderm's sellers would receive 50% of the proceeds or $100m, whichever was lower (Bristol-Myers and Amicus affirm industry's love of orphans, September 1, 2015).

Sellside consensus forecasts for SD-101 sit at $400m by 2022, according to EvaluatePharma. The project has an NPV of $750m, or 36% of Amicus's market cap.

Study Trial ID
Essence NCT02384460

Convert trial

Insmed's amikacin liposome inhalation suspension, previously branded Arikayce, is an inhaled antibiotic for the treatment of non-tuberculosis mycobacterial (NTM) lung infections. In a 90-patient phase II study it failed to perform significantly better than the standard of care on its primary endpoint, a reduction in density of bacteria at 12 weeks (Insmed moves Arikayce goalpost and declares victory, March 26, 2014).

In that trial the prespecified secondary endpoint of negative culture conversion was more encouraging, with 11 amikacin patients converting from a positive to negative culture, compared with just three placebo recipients, with a p value of 0.01. At the time the company had hoped to file early based on the secondary measures, but ultimately decided against this.

The phase III study population is slightly different, comprising those with NTM lung infections caused by Mycobacterium avium complex; this subpopulation had shown the highest response rates in phase II.

The opel-label, 336-patient phase III trial, Convert, has the culture conversion measure at six months as primary endpoint, with change in baseline in six-minute walk test distance as a secondary measure. The study is said to be 90% powered to demonstrate a 15% difference between treatment and control.

In the phase II trial the dropout rate was 22%; inhaled antibiotics often cause upper airway irritation, and the company has noted that the rate in the phase III trial was slightly better than the expected 25%.

Amikacin's 2022 forecasts are $485m, according to EvaluatePharma consensus; the asset has an NPV of $497m, or 66% Insmed's market cap. If the trial is positive Insmed will file in the US on an accelerated basis.

The company has $91m in cash, with a second-quarter burn rate of $38m and debt of $55m, so a cash injection will be needed in the near future.

Study Trial ID
Convert NCT02344004
Open label extension NCT02628600

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EP Vantage is a forward-looking comment and analysis service tailored to the needs of pharma and finance professionals, focusing on the events that will define the future of companies, products and therapy areas. Written by experienced journalists, EP Vantage provides timely financial analysis of regulatory and patent decisions, marketing approvals, licensing deals, and M&A, giving fresh angles and insight to both current and future industry triggers. EP Vantage is powered by EvaluatePharma, the industry leader in consensus forecasts.

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