EuroBiotech: More Articles of Note

> UniQure ($QURE) dosed the first patient in the high-dose cohort of its Phase I/II hemophilia B trial. The start of the dosing of patients with a higher dose of uniQure's AMT-060 comes two months after the Dutch biotech published preliminary top-line data from the first cohort of people to receive the gene therapy. UniQure saw increases in Factor IX expression levels of around 5% in the two patients who had received 12 weeks of low-dose AMT-060 as of early January. In the new cohort, uniQure is hoping to improve on this result, while also showing the drug to be safe at higher doses. Release

> Verona Pharma (LON:VRP) posted data from a dose-finding study of its nebulized formulation of RPL554. In the 7-way crossover Phase IIa trial, Verona gave four doses of the inhaled PDE3/PDE4 inhibitor, two doses of nebulized salbutamol or placebo to 29 mild to moderate persistent asthma patients. With RPL554 showing a dose-dependent effect on bronchodilator response, the trial met its primary endpoint. And, with the highest doses of RPL554 inducing comparable bronchodilator responses to salbutamol, without the side effects, Verona thinks its pipeline is on track. Release

> Bird Rock Bio, the U.S. biotech formerly known as RuiYi, committed to advancing a rheumatoid arthritis drug it picked up off arGEN-X (EBR:ARGX) into a pivotal trial. The plan is to file the paperwork for the study in the first half of the year. Bird Rock Bio made the commitment after going over data from a pair of clinical trials in healthy volunteers, in which the anti-IL-6 cytokine antibody displayed pharmacokinetics that have led the company to predict it can price the drug at $2,000 per patient, per year. If the arGEN-X-discovered drug can live up to this prediction, it will undercut the giants of the rheumatoid arthritis market. Release

> Quantum Genomics (EPA:ALQGC) raised €5.5 million ($6.1 million) through a private placement. The French biotech will use the funds to kick off a Phase IIa trial of its heart failure drug QGC101 in Europe. Quantum Genomics expects to start the trial in the middle of the year. In parallel to the start of the European trial, Quantum Genomics is working to get a U.S. study up and running. Quantum Genomics is in talks with FDA regarding the study. Release (French)

> Shares in BioBlast Pharma ($ORPN) jumped by as much as 100% on the back of data from a Phase II trial of its treatment for oculopharyngeal muscular dystrophy. The drug, a formulation of the sugar trehalose, was deemed safe and well tolerated in the 25-person study, resulting in the trial meeting its primary endpoint. Whether the drug is effective remains to be seen. BioBlast, which has its roots in Israel, is planning to put trehalose through the more rigorous test of a Phase IIb double-blind placebo-controlled study. Release

> Vectura (LON:VEC) and SkyePharma (LON:SKP) agreed to merge. The £441.3 million ($621 million) all-share acquisition of SkyePharma is being overseen by Vectura CEO James Ward-Lilley, who took up the post last year after previously working at AstraZeneca ($AZN). "We have today really got the opportunity to be an industry leader in terms of the device and technology focused on inhaled respiratory airway disease," Ward-Lilley told Reuters. FierceBiotech | Reuters

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