In October, Dr. Thaddeus (Ted) Dryja, a pioneer in the field of retinal genetic diseases, joined the Company’s Scientific Advisory Board. Dr. Dryja is a member of the faculty at Massachusetts Eye and Ear and Harvard Medical School, and previously served as the Global Head of Ophthalmology Research at the Novartis Institutes for BioMedical Research. He is expected to be a key advisor as the Company advances its growing ophthalmology pipeline.
In November, Chief Medical Officer, Noreen R. Henig, M.D., departed the Company to pursue a new opportunity and will serve as a special advisor to the Company going forward. David Rodman, M.D., Chief Development Strategy Officer, assumed leadership over clinical development. Dr. Rodman joined ProQR in March 2017 with extensive experience in rare disease drug development, translational medicine and RNA therapeutics, having previously served in leadership roles with Novartis Institute for Biomedical Research (NIBR), Vertex Pharmaceuticals, miRagen Therapeutics and Nivalis Therapeutics. The Company also announced the promotions of Peter Adamson to Senior Vice President Ophthalmology Franchise and Robert Friesen to Senior Vice President Science and Early Development.
In November, J. Stuart Elborn, Clinical Chair in Respiratory Medicine at Imperial College, Consultant at Royal Brompton Hospital, and immediate past-president of the European Cystic Fibrosis Society, presented data from the Phase 1b safety, tolerability and exploratory efficacy study of QR-010 during the North American Cystic Fibrosis Conference (NACFC) in Indianapolis, Indiana. During the meeting, the Company also held an investor and analyst event to discuss the recent Phase 1b data, new opportunities to target stop-codon (or Class I) mutations in the CFTR gene using its Axiomer® technology platform, and provide an update on other candidates in the pipeline.
In November, the Company presented its oligonucleotide therapeutics approach at the EuroTIDES meeting in Vienna, Austria.
In November, the first patient was dosed in the Phase 1/2 open-label trial (PQ-110-001) assessing the safety, tolerability, pharmacokinetics and efficacy of QR-110 in patients with LCA 10, the most common cause of blindness due to genetic disease in children. The trial will enroll six children (age 6 - 17 years) and six adults (= 18 years) who have LCA 10 due to one or two copies of the p.Cys998X mutation in the CEP290 gene. During the trial, patients will receive four intravitreal injections of QR-110 into one eye; once every three months. The QR-110 trial is expected to be conducted in three centers with significant expertise in genetic retinal disease in the US and Europe. The objectives of the trial will include safety, tolerability, pharmacokinetics and efficacy as measured by restoration or improvement of visual function and retinal structure through ophthalmic endpoints such as visual acuity, full field stimulus testing (FST), optical coherence tomography (OCT), pupillary light reflex (PLR), mobility course and fixation stability. Changes in quality of life in the trial subjects will also be evaluated. QR-110 is the Company’s lead program for genetic blindness and has received fast track designation by the FDA and been granted ODD in the US and EU. Interim safety and efficacy trial results from the majority of patients after six months of treatment are expected in 2018, with full 12-month treatment data from all patients expected in 2019.
On November 16, 2017, the Company consummated an underwritten public offering and concurrent registered direct offering of approximately 6 million ordinary shares at a price of $3.25 per share. ProQR granted the underwriters a 30-day option to purchase up to 745,471 additional ordinary shares at the public offering price, less underwriting discounts and commissions. Gross proceeds from both offerings were approximately $20 million, assuming no exercise of the underwriters’ option to purchase additional shares in the underwritten public offering. Investors in the offering included several new and existing shareholders, along with significant participation from the Management team and Supervisory Board in the underwritten public offering. Proceeds from these offerings along with existing cash on the balance sheet, are expected to fund operations into the second half of 2019.