UniQure - 2013 Fierce 15

UniQure targets one-time cures for chronic diseases

Based: Amsterdam
Founded: 2012
CEO: Jörn Aldag
Clinical focus: Gene therapy
Website: www.uniqure.com

The scoop: Whatever happens from here, uniQure can say its drug Glybera was the first gene therapy approved in the West. The Amsterdam-based company formed in April 2012 with the gene therapy assets of Amsterdam Molecular Therapeutics, and AMT backer Forbion Capital Partners led a €7 million financing to capitalize the new business. UniQure has been on the upswing since that transaction and the European Commission's November 2012 approval of Glybera for lipoprotein lipase deficiency, a rare genetic disease that leads to increased levels of fat in the blood.

What makes uniQure fierce: UniQure has emerged as one of the leading catalysts in the comeback for the gene therapy field, which suffered a near collapse after safety problems in early clinical development of the treatments years ago. Led by Glybera, the company has a pipeline of gene therapies that offer one-time treatments for a range of serious diseases of the liver and central nervous system.

CEO Jörn Aldag, who previously held the same job at AMT, aims to use his company's experience taking Glybera all the way through the regulatory process in Europe to advance other programs involving diseases such as hemophilia, Parkinson's disease and Sanfilippo syndrome. His company has also developed an industrial-scale approach to manufacturing modified adeno-associated virus (AAV) to deliver gene therapies at a facility in Amsterdam. And the NIH has granted the company an exclusive license to the AAV tech for applications in the brain and liver.

The AAV viruses are the vehicles the company uses to slip into cells and deliver healthy genes. To hear Aldag, his company has the ability to replicate its approach to deliver a wide variety of healthy genes in patients whose defective genes underlie their diseases.

"We are currently the only one that has evidence that we can get through the entire development process, including regulatory, and then initiate commercialization of gene therapy," Aldag says. "You don't find anyone that is as integrated as we are. On that basis, we are now building our pipeline."

In July, the Italian pharma group Chiesi paid uniQure $21.8 million (€17 million) in fees and invested $18 million (€14 million) in the company for its commercial rights to Glybera in Europe and to codevelop uniQure's gene therapy for hemophilia B. The Chiesi deal and the conversion of $18.1 million (€14.1 million) from Coller Capital of London and existing backers gave uniQure a stronger capital position.

Chiesi aims to begin sales of Glybera in Europe next year, Aldag says. Yet uniQure and Chiesi face the challenge of pricing a one-time treatment unlike any ever provided (or reimbursed). Media reports raised the question of whether payers would ever go for a drug with a $1 million price tag. However, Aldag says, uniQure has not released proposed pricing and has been looking at the possibility of charging annual fees long after patients get Glybera, basing the amount paid on the benefits to patients.

"We believe that gene therapy is delivering enormous value to patients," Aldag says. "Rather than taking continuous treatment for a chronic disease, [gene therapy] can do away with the disease for a long period of time, if not cure it, with a single intervention."

UniQure has held onto the North American rights to Glybera and seeks clarity from the FDA on a regulatory pathway for the pioneering therapy. The company hired Philip Astley-Sparke, the former CEO of BioVex, as its U.S. president. Amgen ($AMGN) bought Astley-Sparke's BioVex, a developer of cancer immunotherapy with an oncolytic virus component. And his former BioVex manufacturing ace, Lance Weed, has joined uniQure to lead an underway AAV production site in Lexington, MA, that is expected to begin operations in 2015.

Yet the company's comeback is a work in progress. It has yet to fetch a partnership with a drug giant with a global commercial and research infrastructure. Gene therapy player bluebird bio ($BLUE), on the other hand, this year landed a cancer gene therapy deal with biotech powerhouse Celgene ($CELG).

Aldag acknowledges that bluebird's lentivirus mode of delivering gene therapy has emerged as one of the validated technologies in the arena. Bluebird, though, lacks any approved therapies, unlike uniQure with the EC's nod for Glybera.  

Investors: Forbion Capital Partners, Coller Capital, Gilde Healthcare Partners, Chiesi, Lupus Alpha and Grupo Netco

For more:
UniQure snags $58M in collaboration cash plus equity
UniQure drug first gene therapy to get approval in West

-- Ryan McBride

UniQure - 2013 Fierce 15

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