Fragile X syndrome

NPL-2005 and NPL-2009: Fragile X syndrome

The condition

Fragile X syndrome is the largest single cause of inherited mental retardation in the US and Europe, affecting around 1 in every 3,000 male children and 1 in 8,000 female children.

Symptoms of Fragile X syndrome include intellectual handicap, hyperactivity, attention problems, autism, emotional liability and epilepsy. There is currently no approved treatment for Fragile X syndrome.

Find out more about Fragile X syndrome in our Patients and Carers section.

The programs

Neuropharm is pursuing two separate but potentially complimentary compounds to treat the symptoms of Fragile X syndrome.

NPL-2005 is an anticonvulsant drug currently used to treat patients with Fragile X syndrome off-label, while NPL-2009, a glutamate receptor antagonist, is a targeted treatment directed at the underlying cause of symptoms.

The market

Based on the conservative estimate of 1 in every 4,000 males and 1 in every 7,000 females being sufferers of Fragile X syndrome, there is a potential US market of around 60,000 people and a potential EU market of around 90,000 people.

Our progress

NPL-2005 is an existing prescription anticonvulsant drug identified by Neuropharm and key opinion leaders.

We have commenced Phase II clinical trials in children and adolescents at a major European university hospital. We plan to develop NPL-2005 into an oral granule for easy administration.

NPL-2009 is a compound which has been progressed through preclinical and clinical development to Phase II trials by an international pharmaceutical company for a different medical use, before being discontinued.

Neuropharm acquired the rights to data on NPL-2009 through a collaboration in the US with the Fragile X Research Foundation (FRAXA), a patient group seeking to advance treatment of Fragile X syndrome.

Neuropharm was granted a US Orphan Drug Designation for the use of NPL-2009 in the treatment of Fragile X syndrome in November 2006, and we are close to starting the first clinical trials with the compound in conjunction with FRAXA.

Key points

• Neuropharm is working on two compounds which could relieve the symptoms of Fragile X syndrome: NPL-2005 and NPL-2009
• Neuropharm has agreed commercial terms for the ‘right of reference’ to the previous IND with the original developer of NPL-2009
• Collaboration with FRAXA opens up a new network of contacts and expertise

Regulatory

• NPL-2009 granted Orphan Drug Status in the US

Commercialisation

• We intend to open our US office in 2008 and deploy a specialist sales and marketing team to promote our proposed fluoxetine in autism product NPL-2008. The same team could potentially market a Fragile X product in the future

Market Opportunity

• There is currently no licensed treatment for Fragile X syndrome in the US or EU
• The lack of approved treatments for Fragile X syndrome represents a significant unmet medical need
• Market opportunity of approximately 60,000 sufferers in the US and 90,000 in the EU