Biogen Idec gains ALS drug

Biogen Idec has obtained an exclusive worldwide license to a potential new drug for amyotrophic lateral sclerosis (ALS), or Lou Gehrig's disease, from Knopp Neurosciences in a deal that could be worth more than $345m.

The deal calls for Biogen Idec to develop and commercialise the drug, known as KNS-760704, for the treatment of ALS as well as potentially for other indications. The company said it expects to begin phase III clinical studies of the orally delivered drug in the first half of the next calendar year.

Phase II data from a clinical study conducted by Knopp showed that the neuroprotective therapy, which is a chirally pure form of dexpramipexole, was safe and generally well tolerated and produced favourable dose-related effects in preserving motor function and extending survival. Patients suffering from ALS, a rare neurodegenerative disease, generally live only three to five years after the onset of symptoms.

KNS-760704 has received both the orphan drug designation and fast-track status from the US Food and Drug Administration (FDA) for the ALS indication. The orphan status, which is reserved for drugs for conditions that affect fewer than 200,000 people in the US, will give Biogen Idec seven years of US marketing exclusivity if the drug is approved, as well as tax breaks and other benefits. The fast-track status, designed to speed up development of drugs for life-threatening diseases, will allow a rolling review for the drug, meaning that sections of the marketing application can be submitted and reviewed as they are completed. KNS-760704 has also received orphan status for ALS from the European Commission.

Biogen Idec will lead the development and commercialisation of KNS-760704 for ALS, with Knopp providing development support and conducting some US commercialisation activities under its partner's guidance.

In return for the rights to the drug, which is Knopp's lead product candidate, Biogen Idec will purchase $60m of Knopp stock and will make an upfront payment of $20m, as well as potential payments of up to $265m tied to developmental, regulatory, and sales milestones. Knopp will also receive double-digit royalties on global sales of KNS-760704.

Currently, sanofi aventis' Rilutek (riluzole) is the only ALS drug on the US market. However, there are other drugs in development for the disease, including CytRx's Arimoclomol, which is in late-stage clinical studies. In addition, under an FDA-approved programme, a limited number of people with ALS receive Insmed's Iplex, although the drug has not been granted marketing approval for the indication. Iplex was originally approved by the FDA as a paediatric growth failure treatment but is no longer marketed in the US.