Novo Nordisk's tie-up this week in haemophilia genome editing was undoubtedly interesting, but a better move would be an outright acquisition of one of the haemophilia gene therapy players – and Uniqure fits the bill.
Early-stage deals will not solve one of Novo's big problems. Unless it acts quickly the Danish company risks getting left behind in a core area while its diabetes business comes under increasing pressure.
And despite recent takeout speculation, Uniqure is not too pricey: with a market cap of around $1.8bn, Novo could expect to pay less than $3bn, even with a premium. The Danish company showed itself willing to spend this much with last year’s unsuccessful $3.1bn bid for Ablynx.
Best in haemophilia B?
Uniqure has what looks like the most promising haemophilia B gene therapy, AMT-061, with the caveat that this has only been tested in a small number of patients so far (Uniqure turns the screw on Spark and Pfizer, February 8, 2019).
What’s more, AMT-061 could become the first haemophilia B gene therapy to reach the market: the pivotal Hope-B trial reads out towards the end of next year. Meanwhile, the phase III study of Spark and Pfizer’s contender, fidanacogene elaparvovec, is not set to complete until 2021.
Haemophilia B is much rarer than haemophilia A, so is a smaller market. Nevertheless, EvaluatePharma sellside consensus puts AMT-061's net present value at $1.5bn, which would go a long way towards justifying Uniqure’s price tag.
And there is a more pragmatic reason for Novo to prioritise haemophilia B over haemophilia A in deal-making: most of the main contenders in the latter are out of the Danish company’s reach. Biomarin has a market cap of $12bn, while Spark is being acquired by Roche, and Sangamo’s SB-525 is partnered with Pfizer.
Uniqure does have a haemophilia A project, AMT-180, at the preclinical stage. Although this would not assuage any concerns at Novo about missing the haemophilia A gene therapy boat, it could be an intriguing asset given its different approach. Unlike other haemophilia A gene therapies AMT-180 might work in patients who have developed inhibitors; Uniqure plans to file an IND for it next year.
The rest of Uniqure’s pipeline would then be a bonus, although not all of it would make a good fit.
Assets in Fabry disease and other disorders with liver involvement could be attractive, but after AMT-061 Uniqure's next most advanced asset is the Huntington’s candidate AMT-130, an area well outside Novo’s wheelhouse. The company could just sell the project on, further boosting the premium-priced takeout thesis.
Novo has previously said it is willing to strike more external deals, but so far these efforts have been limited to early-stage tie-ups. It is time to take a bigger gamble.
|Annual sales ($m)|
|Project||Indication||Description||Status||2024e sales||Peak sales|
|AMT-061 (etranacogene dezaparvovec)||Haemophilia B||AAV5 FIX-Padua gene therapy||Phase III||744||1,229|
|AMT-130||Huntington's disease||AAV5-delivered micro-RNA||PhI/II to start end-2019||69||2,686|
|AMT-180||Haemophilia A||AAV5 FIX-Super9 gene therapy||Preclinical||-||-|
|AMT-190||Fabry disease||AAV5 NAGA gene therapy||Preclinical||-||-|
|AMT-150||Spinocerebellar ataxia type 3||AAV5-delivered micro-RNA||Preclinical||-||-|
|Undisclosed liver and CNS-directed projects||-||-||Preclinical||-||-|
|Source: EvaluatePharma, company presentation Oct 2019.|