Prothena collapses on double trial failure

pharmafile | April 23, 2018 | News story | Manufacturing and Production, Research and Development Prothena Therapeutics, biotech, drugs, pharma, pharmaceutical 

Shares in Prothena have dropped by close to 70% on the news that two of its trials into its lead drug candidate, NEOD001, had both missed the mark.

The biotech has revealed that it will end development of the candidate, as they see no future for the investigational antibody in the treatment of AL amyloidosis.

The decision was reached based on the candidate missing its endpoints in a Phase 2b trial, and then a futility analysis revealing that there was no reason to pursue further investigation in its Phase 3 trial.

In the Phase 2b trial, placebo significantly outperformed NEOD001 by 47.6% to 39.4% based on the primary endpoint of cardiac best response through 12 months of treatment.

“We are deeply disappointed by this outcome, particularly for patients suffering from this devastating disease,” said Gene Kinney, President and Chief Executive Officer of Prothena. “We are surprised by the results from these two placebo-controlled studies and will continue to analyse the resulting data to share insights with our collaborators in the scientific, medical and advocacy communities. We thank all of the patients, their families, caregivers, investigators, study staff and our employees.”

The results mean it’s back to the drawing board for Prothena but not before its stock and investors took a hammering. In particular, Neil Woodford has been a big investor, owning 8.56% of the biotech through Woodford Investment Management, but now faces taking a huge hit on the investment.

Woodford Investment put out a blog piece shortly after the “extremely disappointing” news broke but claims it maintains its confidence in the biotech, writing, “Prothena has options. It still has an early and mid-stage clinical pipeline. It has a technology platform and a world-leading specialism in misfolding proteins, which are implicated in a number of different neurological disorders. This research platform has been validated by two major pharmaceutical companies – Roche (which is partnering Prothena in PRX002 in Parkinson’s disease, currently in Phase 2 trials) and Celgene (which has recently collaborated with Prothena on three earlier stage clinical assets).”

The latter deal is the one that will provide Prothena with some solace, alongside the $500 million it still holds in the bank, after Celgene agreed a deal potentially worth $2.2 billion for three candidates that will enter Phase 1 trials in the near-term.

After this big failure, it might be a little while before another large player comes in to partner on a drug candidate. A lot will depend on how successful the partnership with Roche proves to be and the readout from that Phase 2 trial.

Ben Hargreaves

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