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Trial Tuesday: A Design Brief, A Heart Miss And A Restart

A melanoma advisory committee backs Replimune's RP-1 while pressing the field for better endpoints; Novo Nordisk's IL-6 inhibitor fails a 6,300-patient cardiovascular outcomes trial; and Rocket clears its first Danon disease patients under a revised protocol.

A hand holds up a book whose cover reads "design:" against a plain wall.
Design, as it happens. Photo by Felirbe on Unsplash.

Three developments over the past week spanned oncolytic immunotherapy, cardiovascular outcomes and rare-disease gene therapy.

Each one carries read-through past the individual program, so we dig into what happened and what the people closest to the data had to say for Trial Tuesday.

Replimune's RP-1 Review Offers A Design Brief For The Next Intratumoral Trials

The advisory committee that reviewed Replimune's RP-1 in melanoma spent less time on the product than on a question the field has left open for years: how to design and measure trials of intratumoral therapies in the first place.

In examining vusolimogene oderparepvec (RP-1) plus nivolumab in patients whose melanoma had progressed on a PD-1 inhibitor, members treated the IGNYTE study's flaws as a case study in what future sponsors should do differently. The clearest lesson concerned endpoints.