Continuing one of the themes from last month, the gene therapy field saw the announcement of two major deals that underlined the growing commitment of large pharma companies to this platform. In late February, Roche announced its intent to acquire Spark Therapeutics in a deal worth $4.3 billion. Spark Therapeutics pioneered the first FDA-approved in vivo gene therapy Luxturna, which is part of a growing pipeline of gene therapies for inherited retinal disorders. The acquisition will also provide Roche access to several gene therapies in development for haemophilia, and Spark’s expertise with gene therapies based on the adeno-associated virus (AAV) platform.
Clinical-stage AAV gene therapy company Voyager Therapeutics, fresh from signing a CNS deal last month with Neurocrine Biosciences teamed up with AbbVie to sign another CNS-focused deal to develop and commercialize vectorized antibodies to treat Parkinson’s disease by targeting pathological species of alpha-synuclein. Voyager will receive a $69 million upfront payment and up to $245 million in preclinical and phase 1 option payments, as well as up to $728 million in development and regulatory milestone payments for each vectorized antibody compound, and $500 million in commercial milestones.
Also focusing on the area of CNS disorders—a field that has attracted a lot of recent early-stage investment — is Imbrium Therapeutics, Purdue Pharma’s newly created clinical research arm, which signed a licensing and development deal with TetraGenetics to develop non-opioid-based biologics to treat chronic pain based on its ion channel antibody discovery platform TetraExpress. Antibodies are increasingly being investigated to target ion channels as they may help address some of the limitations of small-molecule drugs. TetraGenetics will receive up to $25 million upfront and an additional $248 million in milestone payments to deliver a series of antibodies as potential pain treatments.
CNS-focused SK Biopharmaceuticals teamed up with Arvelle Therapeutics to develop and commercialize the antiepileptic drug cenobamate in Europe. A new drug application filed by SK Biopharmaceuticals for cenobamate for the treatment of partial-onset seizures in adult patients has recently been accepted for review by the US Food and Drug Administration (FDA). SK Biopharmaceuticals will receive an upfront payment of $100 million and up to $430 million in regulatory and commercial milestones, as well as net sale European royalties.
Bringing February to a close, a couple of further major acquisitions were announced. Merck and Co. acquired Seattle-based Immune Design for $300 million in cash. Immune Design—established in only 2014—is developing a mid-stage immunotherapy for non-Hodgkin lymphoma and is also developing vaccines for respiratory syncytial virus and peanut allergies. Shortly after, Ipsen announced its acquisition of Clementia Pharmaceuticals and its late-stage product palovarotene, a retinoic acid receptor gamma agonist that is close to filing for FDA approval for the rare bone disorder fibrodysplasia ossificans progressiva (FOP). Clementia gained worldwide rights to the product from Roche in 2013 after it performed poorly in phase 2 trials in patients with emphysema, and repurposed it for FOP as well as multiple osteochondromas (MO). Ipsen will pay $1 billion upfront to acquire the outstanding shares in Clementia, as well as a contingent value right of up to $263 million linked to the regulatory filing of palovarotene for MO.
