Dutch drug developer ProQR Therapeutics is swinging for a $75 million Wall Street debut to fund its work on a treatment for cystic fibrosis, looking to compete with market leader Vertex Pharmaceuticals ($VRTX).
The company, co-founded by former Genzyme CEO Henri Termeer, is preparing a first clinical trial for its lead candidate, QR-010, an RNA-based therapy for CF. The drug is designed to correct an underlying cause of the disease by repairing defects in the CFTR gene, delivered through a handheld inhaler to improve lung function.
ProQR expects to file an IND in the fourth quarter and launch a Phase Ib trial thereafter, targeting CF patients with two copies of the F508del mutation in their CFTR genes--the same population treated by Vertex's in-development combination of lumacaftor and ivacaftor, a potential blockbuster.
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| ProQR CEO Daniel de Boer |
The company expects its IPO haul to pay QR-010's way through Phase IIa trials, all the while supporting the preclinical development of QR-110, an early-stage treatment for the rare Leber's congenital amaurosis, which leads to blindness. ProQR plans to take its lead candidate all the way to market on its own, figuring it can successfully commercialize the CF therapy with a small sales force in the U.S. and Europe.
ProQR began its work in 2012, founded by Termeer; Dinko Valerio, former CEO of the Johnson & Johnson ($JNJ)-acquired Crucell; current chief Daniel de Boer; and Gerard Platenburg, a veteran of Prosensa ($RNA) and ISA Pharmaceuticals. It has raised about €45.6 million ($61 million) in equity, plus received grants from the likes of the Cystic Fibrosis Foundation.
On its website, the Leiden-headquartered biotech said its roughly 50 employees work to "actively create a caring atmosphere filled with fun and joy," and among its staff is a "happiness manager."
- read the filing