- UniQure is exploring a potential sale, Bloomberg reported Sunday, as the gene therapy space has increasingly caught the attention of industry-leading drugmakers.
- The buyout buzz shot up UniQure's stock to an all-time high, trading at nearly $80 per share Monday morning, up about 8% on the day. The biotech's stock has increased 175% since the beginning of 2019 and carries a market value of roughly $3 billion.
- The report anonymously cited people with knowledge of the matter, adding that those sources said "no financial decisions have been made, and there's no certainty the deliberations will lead to a transaction." A UniQure spokesperson declined to comment to BioPharma Dive on the report.
Despite a bevy of regulatory, clinical and commercial challenges, the gene therapy space has validated itself over the past few years in a way that has gotten the attention of the entire industry.
|Partner||Gene therapy company||Date||Upfront amount||Deal type|
|Novartis||AveXis||April 2018||$8.7 billion||Buyout|
|Roche||Spark Therapeutics||Feb. 2019||$4.3 billion||Buyout|
|Biogen||Nightstar Therapeutics||March 2019||$800 million||Buyout|
|Neurocrine||Voyager Therapeutics||Jan. 2019||$115 million||Licensing|
|Novartis||Spark||Jan. 2018||$105 million||Licensing|
|J&J||MeiraGTx||Jan. 2019||$100 million||Licensing|
|AbbVie||Voyager Therapeutics||Feb. 2019||$65 million||Licensing|
SVB Leerink analyst Joseph Schwartz called UniQure "a logical pearl for a pharma company" looking to add gene therapies to its pipeline along with commercial-grade manufacturing capabilities. And Schwartz predicted such a deal may only get more expensive as the biotech progresses its pipeline.
"Rapid development progress on a number of fronts could provide some urgency for interested parties to act sooner rather than later," the analyst wrote in a June 17 note to investors.
UniQure's lead asset is a hemophilia B therapy called AMT-061. The company gave updated, interim data last month from a Phase 2b study, showing all patients had sustained factor levels for up to six months following treatment. A pivotal Phase 3 trial is expected to complete enrollment by year's end.
The biotech also has an experimental gene therapy for Huntington's disease, cleared by the Food and Drug Administration to begin clinical testing. A Phase 1/2 trial is expected to kick off in the latter half of 2019 with the first patient dosing, a company spokesperson said Monday.
Analysts have also pointed out UniQure's position in gene therapy manufacturing and its intellectual property as additional strengths. The company is in the process of expanding its commercial-grade production facility in Lexington, Massachusetts from 55,000 square feet to 80,000 square feet, according to UniQure's most recent company presentation.
On the patent front, the company said last month it has secured a pair of patents for the U.S. and Europe for its Huntington's therapy.
But as the gene therapy space has heated up, so has potential competition these treatments could face. In hemophilia B, for instance, Sangamo Therapeutics is clinically testing its own gene therapy. And in Huntington's, rival therapies are being developed by Ionis Pharmaceuticals and Roche, BioMarin and Wave Life Sciences among others.
Correction: A previous version said BioMarin Pharmaceutical and Sangamo Therapeutics are clinically testing gene therapies in hemophilia B. They are both testing gene therapies in hemophilia A, and Sangamo is also developing a therapy for hemophilia B. The story has been updated to reflect that distinction.