US biotech company Intra-Cellular Therapies says it could file its lead drug lumateperone for schizophrenia as early as next year after discussions with the FDA.
Shares in the firm rocketed on the news, which comes after the US regulator had asked for more toxicology data on the drug, which Intra-Cellular licensed from Bristol-Myers Squibb (BMS) in 2005 as one of a clutch of central nervous system therapies.
The FDA agreed with the company’s position that toxicity seen in animal models did not apply to humans due to differences in physiology between species, clearing the path for Intra-Cellular to carry out a long-term safety study that should enable a marketing application in mid-2018.
Lumateperone is backed by two phase III trials in schizophrenia which showed that the drug can be administered at a fixed dose – in other words patients don’t need to be titrated to an effective dose – and controlled psychotic symptoms with a safety profile similar to placebo. While one of those trials was undermined by a high placebo response, the company says the FDA has agreed there is still enough evidence to move forward with the programme.
The drug is a dopamine receptor phosphoprotein modulator (DPPM) with selective action on dopamine D2 receptors, serotonin 5-HT2A receptors and dopamine D1 receptors. Intra-Cellular maintains this differentiates it from existing schizophrenia drugs, providing additional benefits such as improved psychosocial functioning.
It will need to convince prescribers of those benefits if it is to carve out market share for the drug in a crowded, highly genericised schizophrenia market, but is already looking beyond the first formulation with a new long-acting version of the drug, due to start testing next year in Europe.
That profile could unlock indications beyond schizophrenia, and the drug is also in phase III trials to treat bipolar depression and agitation associated with dementia, including Alzheimer’s disease. Prior to the failed trial and FDA toxicology concerns some analysts had suggested the drug could eventually become a $2bn-a-year blockbuster.
Intra-Cellular hit the headlines in the UK in 2013 after it emerged that the former chair of the National Institute for Health and Care Excellence (NICE), Sir Michael Rawlins, had joined the biotech’s board of directors after his retirement.