Chiesi Group has ended its four-year-old collaboration with uniQure to co-develop and license a gene therapy for hemophilia B and has returned its development and commercialization rights to the gene therapy candidate in Europe and other areas, uniQure acknowledged.

In a statement yesterday, Chiesi CEO Ugo Di Francesco cited unspecified “recent changes in our strategic priorities” for his company’s decision to terminate its co-development and license agreement with uniQure for Glybera® (alipogene tiparvovec).

The termination gives uniQure full rights to AMT-060, its lead gene therapy candidate for patients with hemophilia B—but no partner to co-develop the gene therapy as it heads into pivotal trials.

“By regaining unencumbered, global rights to a late-stage program that has demonstrated significant clinical benefit for patients with hemophilia B, we believe uniQure is better positioned to accelerate the global clinical development plan, maximize shareholder return on our pipeline, and take advantage of new potential opportunities related to the program,” uniQure CEO Matthew Kapusta stated.

“We have made significant progress in preparing for a late-stage clinical program in hemophilia B, and look forward to providing additional updates this fall,” he added.

As for AMT-060, Chiesi’s termination capped a month in which uniQure reported two positive developments for the gene therapy candidate, while a rival hemophilia gene therapy developer announced strong early-to-mid clinical results.

On July 11, the company presented clinical data from an ongoing Phase I/II trial of AMT-060 that it said supported further expansion of the eligibility of adeno-associated virus 5 (AAV5) gene therapies to nearly all patients with hemophilia B. uniQure cited the observance of clinically meaningful Factor IX (FIX) activity levels from the trial at neutralizing antibody (NAB) titers up to 1:341, determined as corresponding up to the 90th percentile of a healthy control population.

A day earlier, Spark Therapeutics announced positive clinical news about its hemophilia B gene therapy candidate, saying that SPK-9001 treatment in 10 patients in an ongoing Phase I/II trial had their annualized infusion rate reduced approximately 99%, to a mean of 1.0 annual infusion as of the June 5 data cut-off date, compared with 67.5 annual infusions before SPK-9001 administration.

But on July 21, uniQure said it had successfully developed and optimized a reproducible and scaled-up manufacturing process for producing AMT-060 at its manufacturing facility in Lexington, MA.

2013 Agreement

Chiesi and uniQure agreed to collaborate on AMT-060, as well as uniQure’s marketed familial lipoprotein lipase deficiency (LPLD) gene therapy Glybera in 2013, though an agreement covering the European Union and selected other countries—Brazil, Mexico, Pakistan, Turkey, Russia, and the Commonwealth of Independent States (CIS) countries, as well as China for the Glybera portion of the agreement.

In April, uniQure confirmed that it will not renew European marketing approval of Glybera when the existing EC authorization expires later this year. By removing Glybera from the market, uniQure generated product-related savings of about $2 million a year net of any payments due to Chiesi.

Under the 2013 agreement, Chiesi and uniQure agreed to an equal sharing of all development related costs for the hemophilia B program, with Chiesi also taking an equity stake in uniQure. Going forward, uniQure will oversee 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. 

Uniqure added that it does not expect the additional expense to impact its previous cash guidance, and continues to anticipate cash on hand will be sufficient to fund operations into 2019.

In connection with the companies’ agreement, uniQure received €17 million ($20 million) in collaboration financing and €14 million ($16.6 million) in equity financing from Chiesi, and has converted into equity a previously-announced €14.1 million ($16.7 million) investment led by Coller Capital, with participation by existing investors. 

AMT-060 won the FDA’s Breakthrough Therapy designation in January.

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