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Prothena Corporation, a pharmaceutical company in neuroscience and orphan disease, has announced that it is discontinuing development of NEOD001, a drug being researched in clinical trials for the treatment of AL amyloidosis patients. The decision was taken because NEOD001 did not do as well as expected in its clinical development programme.

Myeloma Patients Europe (MPE) Chief Executive Officer Ananda Plate commented:

This is unexpected and extremely disappointing news for AL amyloidosis patients. As one of the first drugs to be developed specifically for AL amyloidosis, NEOD001 was very promising for patients.

Over the past years, MPE has developed a good working relationship with Prothena. We will now be engaging with them to fully understand the situation, lessons that can be learned and what will happen to some of their ongoing commitments and projects in the European AL amyloidosis community.

What is NEOD001?

NEOD001 is a drug being explored in clinical trials for the treatment AL amyloidosis. It is a monoclonal antibody targeting the circulating soluble and deposited aggregated amyloid that accumulate in patients with AL amyloidosis and can cause organ damage and failure.

Why are Prothena no longer developing NEOD001?

Prothena made the decision to stop development of NEOD001, as it did not do as well in clinical development as expected. Specifically, the decision was made due to the following:

  • the results of the ongoing Phase 2b PRONTO clinical trial which explored the safety and efficacy of NEO001 in previously treated AL amyloidosis patients with persistent cardiac dysfunction. The study did not meet its primary or secondary end-points (i.e. measurements taken during a clinical trial which determine whether a trial treatment has worked)
  • an independent futility analysis conducted on the Phase III VITAL clinical trial (following the PRONTO results) which was looking at the safety and efficacy of NEOD001 in treatment naive AL amyloidosis patients with cardiac dysfunction. A futility analysis is where a clinical trial is assessed for its ability to achieve its objectives. In the case of NEOD001, the analysis found that the VITAL trial was unlikely to achieve statistical significance for AL amyloidosis patients

Prothena has therefore ended their clinical development programme for NEOD001. This means it will not be studied further or brought to market for AL amyloidosis patients.

What happens to patients participating in a NEOD001 clinical trial?

The news means that there will be no further clinical trials held exploring NEOD001 in AL amyloidosis. All patients receiving the drug through the Phase III VITAL study will be discontinued on the treatment, following a discussion with their doctor.

If you are involved in a clinical trial looking at NEOD001, please speak to your doctor with any questions about next steps or concerns you have about the suspension of the clinical development programme for the drug.

Do they have any other products in amyloidosis?

This was the only drug that Prothena were exploring in AL amyloidosis.

Prothena have another pipeline drug for the treatment of ATTR amyloidosis, called PRX004 which is currently in the pre-clinical phases of development (laboratory testing). You can read more information about this here: https://www.prothena.com/pipeline/prx004/


Where can I find more information?

If you have any questions or comments about the decision, please email morgan@mpeurope.org

You can see the full Prothena press release on the situation here: http://ir.prothena.com/releasedetail.cfm?ReleaseID=1064584

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