So far the only big pharma group to have pursued gene therapy to the point of regulatory success is GlaxoSmithKline. But Pfizer is redoubling its efforts, paying $150m up front for the 78% of Bamboo Therapeutics it did not already own.
This is despite the company, a spin-out from Asklepios BioPharmaceutical, not yet having any clinical-stage products. The deal is also notable for its rarity in this space. It appears that the Bamboo deal is the first time a big pharma has acquired a pure-play gene therapy company.
Pipeline
The focus of the deal is BMB-D001, Bamboo’s gene therapy for Duchenne muscular dystrophy, which is scheduled to enter a phase I/II trial by the end of the year. BMB-D001 is a recombinant adeno-associated viral (rhAAV) vector that delivers a shortened but functional copy of the dystrophin gene.
It also has pre-clinical gene therapies for Friedreich’s ataxia and the neurological disorder Canavan’s disease. The Friedreich’s programme uses an rhAAV vector to deliver a functional copy of the frataxin gene to patients with the movement disorder.
The Canavan’s therapy has been in development for some years, according to Bamboo’s website; an initial clinical trial of an AAV-delivered ASPA gene – which is mutated in Canavan’s patients – was begun in 2001. The product was directly injected into the brains of 13 patients between 2001 and 2005.
Five-year data showed decreased brain atrophy, improvement in several areas of neurological function, and decreased seizure frequency. Bamboo is now working on a next-generation version which is still pre-clinical.
The company also manufactured the AAV vector that is being used in an NIH-sponsored phase I/II study in giant axonal neuropathy.
Pfizer’s acquisition of this company is therefore a move to add gene therapies to its own pipeline, in contrast to its prior licensing-based efforts in this space. The group has a license to two clinical-stage gene therapies thanks to its tie-up with Spark Therapeutics. Recent results with the haemophilia B candidate SPK-9001 look good (New data Spark haemophilia B gene therapy battle, June 17, 2016).
First time
When it comes to gene therapy by far the most successful big pharma is GlaxoSmithKline. Its ex vivo cell-based gene therapy, the immunodeficiency treatment Strimvelis, in June became the second ever gene therapy to gain approval (Therapy focus – Strimvelis approval could rejuvenate gene therapy arena, June 2, 2016).
Glaxo has four other in-house clinical-stage gene therapies. Novartis and Merck & Co are the only other big pharmas with disclosed in-house gene therapy projects.
Licensing agreements are fairly typical of interactions between big pharma groups and gene therapy companies. Takeovers are rarer.
Selected big pharma gene therapy deals | ||||||
Company | Project | Status | Strategy | Deal source | Deal value ($m) | Deal date |
Novartis | TherAtoH and CGF166 | Both phase II | In-licensed | GenVec | 214 | Jan 2010 |
Pfizer | SPK-FIX and SPK-9001 | Both phase II | In-licensed | Spark Therapeutics | 280 | Dec 2014 |
Sanofi | UshStat and StarGen | Both phase II | In-licensed | Oxford BioMedica | 53 | Apr 2009 |
Johnson & Johnson | Ad-REIC | Phase II | Company acquisition | Crucell | 425 | Mar 2011 |
Sanofi | VY-AADC01 | Phase I | In-licensed | Voyager Therapeutics | 845 | Febr 2015 |
Amgen | AMT-090 | Phase I | Company acquisition | Synergen | 254 | Dec 1994 |
Bristol-Myers Squibb | AAV-S100A1 | Pre-clinical | In-licensed | uniQure | 603 | Apr 2015 |
Pfizer | BMB-D001 | Pre-clinical | Company acquisition | Bamboo Therapeutics | 645 | Aug 2016 |
More than 20 years ago Amgen bought Synergen, and one of the projects it picked up was a research-stage glial cell derived neurotrophic factor gene therapy. Now known as AMT-090, this has clawed its way into phase I trials in Parkinson’s disease – it is also in pre-clinical research for Huntingdon’s – but this is hardly speedy work.
The other big pharma buy that transferred a gene therapy was Johnson & Johnson’s acquisition of Crucell five years ago. But Crucell had two years earlier licensed rights to Ad-REIC, a prostate cancer gene therapy, to Japanese group Momotaro-Gene. J&J will simply collect royalties if the project makes it to market.
In any case neither of these acquisitions were focused on gene therapy. Amgen was taking out a rival biotech and J&J was more interested in Crucell’s vaccines than its gene therapy. It is therefore not an exaggeration to say that Pfizer buying Bamboo is the first time a big pharma has acquired a gene therapy company.
Pfizer has not staked the family fortune on Bamboo, however. The deal is back end-loaded, with the contract specifying $495m in milestones. Pfizer knows as well as anyone that despite two gene therapies having made it to market in Europe, this field still has a lot to prove.
To contact the writer of this story email Elizabeth Cairns in London at elizabethc@epvantage.com or follow @LizEPVantage on Twitter