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.
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).
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.