Health and Healthcare

Ionis Stumbles on FDA Clinical Hold

Chris Lange

Published: April 7, 2016 12:10 pm

Last Updated: January 13, 2020 11:02 am

Ionis Pharmaceuticals Inc. (NASDAQ: IONS) made waves in the market on Thursday following a business update. The company provided an update on the IONIS-TTR program. This is currently being evaluated by IONIS in an ongoing Phase 3 study, NEURO-TTR, in patients with transthyretin (TTR) familial amyloid polyneuropathy.

For some background, this Phase 3 study (CARDIO-TTR) is also being planned by GlaxoSmithKline PLC (NYSE: GSK) to evaluate IONIS-TTR in patients with TTR amyloid cardiomyopathy.

GlaxoSmithKline is in the process of finalizing the protocol for the CARDIO-TTR study with the U.S. Food and Drug Administration (FDA). However, in that process, the FDA placed a clinical hold on the CARDIO-TTR study while this company provides answers to questions about the protocol stemming from Ionis’s ongoing NEURO-TTR study.

The NEURO-TTR study, which is regulated by a separate division at the FDA, is continuing as planned and is on track to complete in the first half of 2017.

Lynne Parshall, COO of Ionis, commented:

A clinical hold is a tool the FDA often uses while a sponsor answers questions the FDA may have about a clinical study. We are working closely with GSK, who have an option to exclusively license the IONIS-TTRRx program, to support their efforts to quickly answer the FDA questions and initiate the study.  We hope GSK will be in a position to advance the CARDIO-TTR study as quickly as possible to potentially bring IONIS-TTRRx to patients with TTR amyloid cardiomyopathy who are in serious need of treatment options that can address the root cause of their disease.

Shares of Ionis were trading down over 10% at $41.78 on Thursday, with a consensus analyst price target of $56.13 and a 52-week trading range of $30.93 to $71.50.

GlaxoSmithKline traded at $41.67, with a consensus price target of $47.33 and a 52-week range of $37.24 to $48.45.