Shire joins orphan disease rush with $500 million Acceleron deal
This article was originally published in Scrip
Shire has joined the ranks of large pharmaceutical companies pursuing deals and acquisitions in the orphan disease market by acquiring the rights to Acceleron's Duchenne's muscular dystrophy (DMD) treatment ACE-031 outside of North America.
The deal follows a wave of recent activity in orphan disease deals. Pfizer acquired the private US company FoldRx Pharmaceuticals last week, while in June GSK extended its deal with Prosensa covering novel RNA-based treatments for DMD (scripintelligence.com, 2 September 2010 & scripintelligence.com, 23 June 2010). Sanofi-Aventis's pursuit of Genzyme, which has a health portfolio of rare disease products, coupled with Shire's deal with Acceleron adds further weight to speculation that the industry is finally realising the potential benefits offered by specialty and orphan disease research.
Shire is to pay Acceleron $45 million up front, as well as up to $165 million in regulatory and sales milestones for the commercialisation of ACE-031 and up to an additional $288 million if ACE-301 is commercialised in other indications. The deal has not impacted Shire's full-year non-GAAP EPS of $4.00.
ACE-031 is a human, recombinant fusion protein produced by joining a portion of the human activin receptor IIb (ActRIIB) receptor to a portion of a human antibody. Shire and Acceleron hope to advance the product into a global Phase II/III clinical programme designed to demonstrate disease modification in DMD patients.
The product is currently in a double-blind, placebo-controlled, dose-ranging Phase II trial in 76 patients with DMD receiving concurrent corticosteroid treatment, to assess its safety, tolerability and pharmacokinetics. Trial completion is expected in Feb 2012.
Although it is hard to predict when the product may reach commercialisation, it could be on the market by 2016, Shire told Scrip.
Shire's Human Genetics Therapies business already has a sizeable pipeline of orphan disease drugs including Vpriv (velaglucerase alfa) and Replagal (agalsidase alfa), which are in registration for the treatment of Gaucher's disease and Fabry disease, respectively.
However, through the acquisition of ACE-301 rights, Shire will be competing against the world's second largest pharma company, GSK, which acquired the rights to Prosensa's PRO-051, a 2'-O-methyl antisense oligonucleotide in October last year (scripintelligence.com, 14 October 2009). The product, which is due to enter pivotal Phase III trials this year, causes exon skipping of exon 51 during mRNA splicing.
Acceleron's CEO Dr John Knopf said: "The structure of this collaboration allows Acceleron to retain commercial rights in North America with the opportunity to build a highly valuable business while collaborating with an ideal partner for ACE-301."
Shire will use its manufacturing in Lexington, Massachusetts, to produce commercials supplies of the product for both itself and Acceleron, which will commercialise any product in North America.