Date: 2017-07-31
Type of information: Termination of an agreement
Compound: Glybera®, gene therapy for hemophilia B (alipogene tiparvovec)
Company: Uniqure (The Netherlands) Chiesi Farmaceutici (Italy)
Therapeutic area: Rare diseases - Genetic diseases
Type agreement: co-developement, commercialisation
Action mechanism:
- gene therapy. Glybera® (alipogene tiparvovec) is an adeno-associated viral vector (AAV1) based gene therapy, administered intramuscularly (IM) at multiple-sites in a single session. AAV1 carrying the human variant LPLS447X gene is delivered to skeletal muscle, where it becomes active. The LPL protein is expressed and transported to the capillary endothelium where it binds to chylomicrons and VLDL. Alipogene tiparvovec is intended as a curative measure for patients with LPLD and, as well as enhancing chylomicron metabolism, may prevent episodes of pancreatitis.
Disease: lipoprotein lipase deficiency demonstrating hyperchylomicronaemia or having a history of acute pancreatitis, hemophilia B
Details:
- • On July 9, 2013, uniQure has announced it has signed collaboration agreements with Chiesi Farmaceutici SpA for the commercialization of Glybera®, the first gene therapy to receive regulatory approval in Europe, as well as the co-development of a gene therapy for hemophilia B. This agreement gives Chiesi exclusive rights to commercialize Glybera®, that has been approved in the European Union for the treatment of the orphan disease lipoprotein lipase deficiency (LPLD), as well as for uniQure’s pipeline product for hemophilia B, in Europe and selected other countries (Brazil, Mexico, Pakistan, Turkey, Russia, and the CIS countries, plus China for Glybera only). Commercial rights for the US, Japan, and parts of Latin America and Asia, and Australasia remain with uniQure. In exchange, uniQure stands to receive net royalties that range from 20 to 30 percent over time on sales of both products. Furthermore, Chiesi will fund half of the remaining development costs for uniQure’s hemophilia B program, as well as take an equity stake in uniQure.
- Uniqure has announced that in the next 12-18 months, the company anticipates clarifying the path toward an FDA filing for Glybera® in the US, reporting results from a Phase I/II study in acute intermittent porphyria, and starting at least two Phase I/II studies for additional pipeline programs.
Financial terms:
- In connection with this transaction, uniQure has received € 17 million ($ 21.8 million) in collaboration financing and € 14 million ($ 18 million) in equity financing from Chiesi. The investment by Chiesi will also result in the conversion into equity of the previously announced € 14.1 million ($ 18.1 million) investment led by Coller Capital (London, UK) with participation by existing investors.
Latest news:
- • On July 31, 2017, uniQure announced that it has entered into an agreement with Chiesi Group to reacquire the rights to co-develop and commercialize its hemophilia B gene therapy in Europe and other select territories and to terminate their co-development and license agreement. Under the terms of this agreement, uniQure will be responsible for all future development costs related to its hemophilia B program, including approximately $3 million of expenses in 2017 that would have otherwise been shared with Chiesi. The Company does not expect the transaction will impact its previous cash guidance, and continues to anticipate cash on hand will be sufficient to fund operations into 2019. As a result of the transaction, uniQure expects to recognize in the third quarter of 2017 the remaining deferred revenue of approximately $14 million from non-refundable payments received from Chiesi in 2013.
• On April 20, 2017, uniQure announced that it will not pursue the renewal of the Glybera® (alipogene tiparvovec) marketing authorization in Europe when it is scheduled to expire on October 25, 2017.
- Under the terms of the agreement between uniQure and Chiesi Group, which has exclusive rights for the commercialization of Glybera® in Europe and other selected countries, uniQure will continue to make product available to Chiesi to treat any patients that are approved for treatment prior to October 25, 2017, and will also be responsible for terminating the Phase IV post-approval study.
Is general: Yes
