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.
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."
Galapagos NV announced the achievement of a second partial milestone, thereby completing the nomination of a new pre-clinical candidate in the osteoarthritis alliance with Servier. On March 7, Galapagos announced the receipt of €2M as the first partial milestone. Today's payment remains undisclosed and contributes to Group revenues in the first half of 2014.
The FP7 Health project ENTENTE and EuropaBio event will gather 28 European early stage innovative companies active in the health sector and seeking investment opportunities and international exposure. The selected companies will have the opportunity to pitch their innovation, developments and needs to an international jury of about 40 business angels, venture capitalists, corporate investors and other industry experts. The deadline for applications is 29 August.
For his research on poisonous snakes Dr. Freek Vonk, researcher at Naturalis Biodiversity Center, received a VENI grant of a quarter million from the Dutch Organization for Scientific Research (NWO). Freek Vonk will use this amount to clarify the genomes of two species of poisonous snake species.
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MorphoSys and Galapagos advance Joint antibody program in Inflammatory diseases into preclinical developmentPublished : Wednesday, July 23, 2014
MorphoSys AG and Galapagos NV announced that the first program from their strategic alliance has advanced into preclinical development. MOR106 was realized based on a novel Galapagos target and MorphoSys' next-generation antibody library Ylanthia and will now be jointly developed in inflammatory diseases.
Pharming and Salix announce FDA approval of Ruconest® for the treatment of acute Angioedema Attacks in patients with Hereditary Angioedema (HAE)Published : Thursday, July 17, 2014
Pharming Group NV and Salix Pharmaceuticals, Ltd. announced that the Food and Drug Administration has approved RUCONEST® (C1 Esterase Inhibitor [Recombinant]) 50 IU/kg for the treatment of acute angioedema attacks in adult and adolescent patients with hereditary angioedema (HAE). Because of the limited number of patients with laryngeal attacks, effectiveness was not established in HAE patients with laryngeal attacks.
One of the riddles of mammal evolution explained: the strong conservation of the number of trunk vertebrae. Researchers of the Naturalis Biodiversity Center and the University of Utah show that this conservation is probably due to the essential role of speed and agility in survival of fast running mammals.
The first two courses of the Leiden Futurelab, a new international post-graduate learning center for biomedical professionals with an entrepreneurial spirit, took place in June and early July. Both courses were very successful: 'Clinical development & clinical trial application' (CD&CTA) and 'Intellectual Property in high tech start-ups' (IP). The courses offered a stimulating environment for the participants.
Leiden Bio Science Park and InnovationQuarter together with other regional organizations joined the Dutch Ministry and Holland Bio at the Holland Pavilion at the BIO International Convention 2014 in San Diego. In this joined effort The Netherlands was well represented and profiled as a biotech hub for international companies at the world’s largest and most prestigious biotechnology event.
Registration for the 5th edition of Science for Business-BioWin Day began on May, 12th. The event accounts already, six months prior, 31 exhibitors and 37 sponsors (including GSK, Baxter, IBA, Janssen Pharma, Novartis, Merck Belgium, Merck Millipore, Eurogentec Kaneka, etc.). Check out the program and join us on November 26th, 2014, at Aula Magna (in Louvain-la-Neuve, Belgium)!
On an excavation site in the Oegstgeest area of Leiden Bio Science Park, Leiden University archaeologists discovered a very rare silver bowl from the first half of the seventh century. The bowl is decorated with gold-plated representations of animals and plants and inlaid with semi-precious stones. The discovery suggests the existence of an elite with a wide international network in Oegstgeest.
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