Roche signs Huntington’s deal
pharmafile | April 10, 2013 | News story | Research and Development, Sales and Marketing | Huntington's, Isis, Roche
Roche is to pay California-based Isis Pharmaceuticals $30 million upfront to cement a new alliance in which the firms will develop treatments for Huntington’s disease.
The Swiss manufacturer’s total outlay could be as much as $362 million depending on the success of the project, including up to $80 million in potential milestone payments.
Huntington’s is an inherited genetic brain disorder, which takes hold between the ages of 30 and 50 and weakens sufferers’ mental and physical faculties, leading eventually to death from conditions such as pneumonia or heart failure.
The attraction for pharma companies is that Huntington’s has some affinity with conditions such as Alzheimer’s or Parkinson’s, making it a possible test of the effectiveness of a drug in those more lucrative therapy areas.
At present, Huntington’s treatments focus on reducing the severity of some symptoms but research in the new tie-up will focus on blocking production of all forms of the huntingtin (HTT) protein which is responsible for the disease.
Isis is responsible for the discovery and development of such a drug, which will be based on the firm’s antisense oligonucleotide (ASO) technology, combined with Roche’s proprietary ‘brain shuttle’ programme.
Isis will receive tiered royalties on sales of the resulting drugs, which Roche has the option to licence through the completion of the first Phase I trial.
The Basel-based group would then be responsible for global development plus regulatory and commercialisation activities for all products the collaboration comes up with.
“This dual track development programme ensures whichever candidate compound proves to be most promising – Isis’ lead target or Roche’s brain shuttle version – can be taken forward to pivotal clinical trials,” said Shafique Virani, global head neuroscience, cardiovascular & metabolism at Roche Partnering.
The deal is welcome news for Isis, which last December suffered a setback when the CHMP declined to recommend its lead product Kynamro, commercialised by Genzyme, as a novel treatment for homozygous familial hypercholesterolaemia.
“We believe that Roche’s expertise in developing CNS drugs, along with their clinical development experience, will greatly enhance our development efforts for this programme and allow us to move forward more rapidly,” said B. Lynne Parshall, Isis chief operating officer.
“By utilising Roche’s brain shuttle technology with our antisense drug discovery capabilities, we have the potential to significantly improve the therapeutic potential for this programme,” Parshall added.
Isis will use milestone payments to reimburse CHDI Foundation, a non-profit organisation dedicated to finding Huntington’s therapies, which gave the company financial and scientific support
CHDI is also to receive $1.5 million on the signing of the Roche agreement and will carry on providing advice to both firms.
Adam Hill
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