| Tuesday | ||||
uniQure hosted a virtual… uniQure hosted a virtual investor event focused on AMT-260, an AAV gene therapy for refractory temporal lobe epilepsy, or rTLE, and other focal epilepsies in an event that featured a presentation from rTLE clinical expert Ellen Bubrick, MD, associate chair of Neurology at Harvard Medical School and director of the Epilepsy Surgery Program at Brigham and Women's Hospital in Boston, MA. "The presentation highlighted preclinical data that supports the safety and tolerability of AMT-260 and plans for its clinical development, as well as uniQure's miQURE and linQURE technology platforms that allow the Company to use miRNAs to safely reduce the expression of genes in the brain. The company also highlighted its progress in developing a commercial-scale AAV manufacturing platform," the company stated. "One week after the landmark FDA approval of the uniQure-developed world's first gene therapy for hemophilia B, we are pleased to share the progress that we have made in developing AMT-260, a first-in-class gene therapy for temporal lobe epilepsy. Today's event presented the preclinical data and the innovative technologies that we hope will allow us to develop AMT-260 and other gene therapies that can locally regulate neuronal activity for diseases of the central nervous system. We look forward to continuing to work toward submitting an investigational new drug application for AMT-260 and advancing it into the clinic in 2023," stated Ricardo Dolmetsch, Ph.D., president of research & development at uniQure. | ||||
Virtual Research &… Virtual Research & Development Event to be held on November 29 at 8:30 am. Webcast Link | ||||
Virtual Research &… Virtual Research & Development Event to be held on November 29 at 8:30 am. Webcast Link | ||||
| Monday | ||||
Virtual Research &… Virtual Research & Development Event to be held on November 29 at 8:30 am. Webcast Link | ||||
| Over a week ago | ||||
These names in the…
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Piper Sandler analyst… Piper Sandler analyst Christopher Raymond cautions that "reading FDA tea leaves can be a dangerous game," but with that said he thinks it is "reasonable to connect a couple of recent data points from FDA" that he thinks bode well for Roctavian's U.S. approval process, including yesterday's approval of uniQure's (QURE) hemophilia B gene therapy Hemgenix and today's announcement that the FDA no longer intends to conduct an AdCom to review BioMarin's (BMRN) Roctavian hemophilia A application. The analyst, who reminds investors that Roctavian's EU launch is currently underway and that his recent hematologist survey makes it appear there is "some degree of pent-up demand for the therapy," has an Overweight rating and $128 price target on BioMarin shares. | ||||
Truist analyst Robyn… Truist analyst Robyn Karnauskas raised the firm's price target on uniQure to $65 from $51 and keeps a Buy rating on the shares. The analyst cites the company's announcement yesterday regarding the FDA approval of Hemgenix with a "clean label". Karnauskas adds that she is updating her model to reflect the $3.5M price in U.S., modeling a peak $800M in royalties vs. $600M previously. | ||||
uniQure announced that… uniQure announced that its partner CSL has received approval from the U.S. Food and Drug Administration for HEMGENIX, a one-time gene therapy for the treatment of adults 18 years of age and older living with hemophilia B. The product is approved for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy or have current or historical life-threatening hemorrhage or have repeated, serious spontaneous bleeding episodes. CSL licensed the exclusive global rights to HEMGENIX from uniQure in May 2021 and is now solely responsible for the further development, registration, and commercialization of the therapy. "Today's landmark approval represents a major milestone in the field of genomic medicine and ushers in a new treatment paradigm for patients living with hemophilia B," said Matt Kapusta, chief executive officer of uniQure. "This is uniQure's second internally-developed gene therapy to achieve approval and the world's first gene therapy for hemophilia B, an historic achievement based on more than a decade of research and clinical development, as well as the tireless dedication of our employees, clinicians, patients and their families. For nearly 25 years, uniQure has been driven by a singular mission: to transform peoples' lives by harnessing the power of gene therapy. This groundbreaking approval delivers on this promise by providing people with hemophilia B the possibility of being liberated from burdensome infusions and an ability to unlock the potential in their daily lives. We offer our sincere gratitude to the hemophilia community, without whose years of dedication and invaluable support this medical breakthrough could not have been achieved." | ||||
| Over a month ago | ||||
Reports Q1 revenue $1.4M,… Reports Q1 revenue $1.4M, consensus $3.48M. "We are pleased that following a comprehensive review of all available safety, biomarker and imaging data, the Data Safety Monitoring Board has recommended that we resume patient enrollment at the higher dose in the ongoing Phase Ib/II study evaluating AMT-130 in Huntington's disease," stated Matt Kapusta, chief executive officer of uniQure. "Patient safety will always be our utmost priority, and we are grateful for the close collaboration between our clinical team, study investigators and most importantly, the patients, all of whom were instrumental in completing our comprehensive investigations. We believe that AMT-130 has the potential to provide a positive impact for patients with this devastating disease for which there is no currently approved treatment. We look forward to finishing patient enrollment in the higher-dose cohort of the European study in the first half of 2023 and remain on track to announce data from the U.S. Phase I/II study in the second quarter of 2023. In the third quarter, the U.S. Food and Drug Administration and the European Medicines Agency completed site inspections of our Lexington, Massachusetts gene therapy manufacturing facility, and we have since received good manufacturing practice certification from the European authorities. This is an important milestone as we seek to establish industry-leading commercial gene therapy manufacturing capabilities. Our partner, CSL Behring, continues to interact with U.S. and European health authorities as they complete their review of the marketing applications for etranacogene dezaparvovec and, assuming approval, we look forward to partnering with our colleagues at CSL Behring to bring this potentially transformational treatment option to people living with hemophilia B." | ||||
| Over a quarter ago | ||||
Wells Fargo analyst Jim… Wells Fargo analyst Jim Birchenough lowered the firm's price target on uniQure to $24 from $34 following the clinical update to the AMT-130 program. The analyst keeps an Equal Weight rating on the shares. | ||||
