Autism

NPL-2008: Autism

The condition

Autism Spectrum Disorders, affect 1 in 150 people worldwide, according to the US Center for Disease Control.

It is a complex neurological disorder that typically impacts the development of children, persists into adult life, and embraces a range of conditions of varying degrees of severity.

Find out more about autism in our Patients and Carers section.

The program

Neuropharm’s program in autism, NPL-2008, is based on the generic compound fluoxetine, a selective serotonin reuptake inhibitor (SSRI) designed to increase serotonin levels in the brain.

Our rationale is that fluoxetine is able to address the low levels of serotonin observed in patients with autism, increasing brain activity and decreasing repetitive behaviour. This has been backed by peer-reviewed academic research and off-label prescribing of fluoxetine.

The market

It is estimated that there are more than 1.8 million cases of Autism Spectrum Disorders in the US and around 2.8 million cases in the EU. There is currently no approved drug for autism in the US.

Our market research in the US shows us that clinicians believe that up to 65% of patients would benefit from SSRI therapy.

There has been a positive reaction from key opinion leaders, child and adolescent psychiatrists and pediatric neurologists regarding the potential of NPL-2008 in treating autism.

Our progress

In 2006 Neuropharm acquired the rights to the NPL-2008 program from the Mount Sinai School of Medicine in New York, where Professor Eric Hollander was granted Orphan Drug Designation (ODD) in 1999 for the use of fluoxetine in autism.

Orphan drug status would give seven years marketing exclusivity for fluoxetine in autism and is key to the commercial viability of NPL-2008.

Professor Hollander has already completed a series of clinical trials of fluoxetine in children and adults in work funded by the Food & Drug Administration’s Office of Orphan Product Development.

In February 2007, Neuropharm formed an agreement with Catalent Pharma Solutions under which fluoxetine will be delivered through their patented Zydis® tablet. Zydis® is the fastest orally dissolving tablet in the world and designed to enhance compliance in both children and adults.

Catalent has now developed NPL-2008 in three dosage strengths in preparation for Neuropharm’s SOFIA (Study of Fluoxetine in Autism) trial at 16 centers of excellence in the US, being carried out as a collaboration with the US patient organisation Autism Speaks. SOFIA was granted Special Protocol Assessment status by the FDA.

The SOFIA trial will help us define titration and clinical use, and the results from the trial will form part of the New Drug Application (NDA) regulatory submission to FDA.

The earliest date for a product launch in the US is anticipated to be Q4 2009.

Key points

• Over 10 years’ work on the program by Professor Eric Hollander, now a consultant to Neuropharm
• The FDA Office of Orphan Drug Development has funded two clinical trials, showing a high level of confidence in the program
• US Orphan Drug status gives seven years marketing exclusivity
• Collaboration with Catalent for customised, low dose, fast melt Zydis® fomulation

Low risk

• Fluoxetine is one of the most widely prescribed CNS drugs in history
• Formulation and manufacturing program is on track
• SOFIA study in 16 leading centers of excellence commenced on schedule
• 20 compounds have already been successfully formulated and marketed in Zydis®

Regulation

• Special Protocol Assessment status granted for SOFIA
• On track for New Drug Application submission in US Q4 2008

Market opportunity

• Over 1.8 million US patients and 2.8 million patients in the EU with Autism Spectrum Disorders
• Neuropharm intends to market NPL-2008 in the US through its own specialist sales force team