Novo Nordisk and Bluebird Bio., previously known as Genetix and purchased by Third Rock Ventures, will collaborate to develop genome editing treatments for genetic diseases, citing specifically the bleeding disease hemophilia as a target disorder, noted Jonathan Gardner on BioPharma Dive. Recently, Bluebird Bio. has been involved in a legal litigation for their alleged actions against Errant Gene Therapeutics (EGT), which include alleged “tortious interference, misappropriation of trade secrets civil conspiracy, [and] unjust enrichment.”
Found in the introduction of plaintiff EGT’s complaint against Bluebird Bio., the defendants “maliciously embarked upon a secret and unlawful scheme to misappropriate highly confidential intellectual property concerning a potentially life-saving gene therapy developed by EGT, a small biomedical company.”
Trialsite News has tracked the Bluebird Bio and EGT case with interest. The allegations have raised eyebrows across a wide spectrum of stakeholder in the clinical trials industry. We have received many private comments about the court case and will surely track it to its conclusion.
As reported by BioPharma Dive, recombinant factor replacement therapies have been used to treat hemophilia since the early 1990s. Newer products have been able to stretch the dosing frequency for bleeding prevention to at best once every four days. However, Roche disrupted the market with the introduction of Hemlibra (emicizumab) in 2017, which can be dosed as seldom as once every four weeks in hemophilia A patients. Further disruption awaits in the form of gene therapies from BioMarin Pharmaceutical, Spark Therapeutics, Uniqure and Sangamo Therapeutics, which should require only a single treatment.
Novo Nordisk has already felt the pressure from Hemlibra, as its 2018 sales of NovoSeven plummeted 14% to about $1.2 billion. NovoSeven is used to treat patients who develop inhibitors to factor VIII replacement therapies, a setting where Hemlibra was first approved. The deal with Bluebird will make use of a genome editing technology called MegaTAL, which the biotech says can be combined with viral or non-viral delivery methods.
Bluebird has already gained European approval of a gene therapy called Zynteglo, for the blood disease beta thalassemia. This treatment involves harvesting of stem cells and then modifying them outside the body using a virus encoding for production of a protein called beta-globin, before being re-infused into the patients.
Venture capital firm Third Rock Ventures LLC (TRV) is known for being an active investor—creating companies by bringing together experts and proactively building companies from the ground up. They acquired Genetix (EGT’s only competitor) in early 2010. If EGT’s published arguments are accurate, TRV sought to acquire the EGT Vector (used to treat thalassemia), claiming that it was superior to the Bluebird intellectual property, which they had just purchased for $35 million.
If that is successful, along with rivals from Spark or Sangamo and Pfizer, many eligible patients would likely have received gene therapy long before Novo and Bluebird can launch a product, leaving only new patients to be treated, wrote Cantor Fitzgerald analyst Elemer Piros.
“While the deal makes sense for both companies in our view, we believe other genetic therapies for hemophilia are progressing much faster, and could limit the market opportunity, should the program ever succeed,” he wrote in an October 9 note to clients.
Meantime, Madeleine Armstrong on Vantage noted that Novo Nordisk claims to be a leader in hemophilia but looks to have missed the boat with the first wave of gene therapies for the disorder. In hemophilia A, the initial focus of the Novo-Bluebird project, several gene therapies are approaching the market. The most advanced, BioMarin’s valoctocogene roxaparvovec (Valrox), is set to be filed by the end of this year and is expected to become the third-biggest seller in the disease by 2024, according to EvaluatePharma sell-side consensus.
Meanwhile, Novo’s next hemophilia hope, the tissue factor pathway inhibitor concizumab, is forecast to bring in just $47m that year. That asset is due to begin phase III development this year.
Daniel Timmerman, vice-president of Novo’s biopharm transformational research unit, admitted that were the company to start developing a traditional hemophilia gene therapy now, its chances of being competitive would be “relatively slim.” However, he stopped short of ruling out such a move. “Classic gene therapies are still attractive for Novo. We’re interested in new opportunities close to where we’re already working,” he told Vantage, citing rare genetic diseases as an area of interest for Novo and also a sector that “lends itself well to gene therapies”.
Given Novo’s reputation as a builder rather than a buyer, this seems unlikely to translate into a big acquisition. But perhaps this possibility should not be completely dismissed given the Danish group’s strategy shift last year, not long after it tried and failed to buy Ablynx.
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