The drug-device combination, named ALN-HTT, uses a Medtronic-developed implantable infusion system to deliver RNAi to the central nervous system (CNS). Alnylam and Medtronic developed ALN-HTT as part of a collaboration that began in 2005 and advanced in 2007.
CHDI Foundation, a not-for-profit Huntingdon’s Disease-focused virtual biotech, has now joined the collaboration and is funding up to 50 per cent of investigational new drug (IND) application-enabling activities.
“We are very pleased to add CHDI as a partner in our Huntington’s disease program; they bring a tremendous amount of disease area expertise that will contribute to advancing ALN-HTT towards the clinic with Medtronic”, said Dinah Sah, vice president, research, at Alnylam.
Medtronic and Alnylam remain 50-50 partners in the US. Commercialisation in the US will be handled by Medtronic. Alnylam can invest in development in return for a share of the profits. In Europe, Medtronic is responsible for ALN-HTT, with Alnylam eligible for milestones and royalties.
Delivery and data
Alnylam and Medtronic have generated preclinical data which they believe supports continued development of ALN-HTT. Data shows direct delivery to the CNS results in silencing of the huntingtin mRNA gene, even at substantial distances from the site of the infusion device.
Success in silencing huntingtin over these distances suggests the product can be effective in the larger human brain, according to the collaborators. Financial support from CHDI will now be used to help file an IND and advance ALN-HTT into Phase I.
Development of ALN-HTT has been supported by Medtronic’s knowledge of drug infusion systems. Medtronic currently markets implantable drug infusion systems for the treatment of severe spasticity and chronic pain.