AMT-180 / uniQure - LARVOL DELTA

Efficacy and Safety of Recombinant Factor IX-FIAV and Bypassing Agents in Thrombin Generation Analyses in Hemophilia A Patient Plasma: The FIVITAS Study (ASH 2021) - "This is suggestive of a minor synergistic procoagulant effect, which is consistent with the FIX(a)-FX(a) bridging capacity of emicizumab. In conclusion, FIX-FIAV could serve as a potential treatment for hemophilia A as it mitigates the hemophilia A phenotype in patient plasma, also in the presence of an inhibitory anti-FVIII antibody. While further safety assessment is warranted, no severe procoagulant effects were observed for the combination of FIX-FIAV with conventional hemophilia A therapeutics."

Clinical • Hematological Disorders • Hemophilia • Rare Diseases

Human Dose Prediction of a Novel Factor IX Variant Gene Therapy Candidate (AMT-180) Mediating Clotting Independently of Factor VIII (ASGCT 2020) - "These data were then used to predict theoretical FVIIIindependent clotting activity in the NHP. Based on the current data set, our model predicts a dose-dependent increase in FVIII-independent activity that can readily achieve meaningful hemostasis in hemophilia A patients with and without inhibitors."

F2

uniQure Presents New Preclinical Data in SCA3, Fabry Disease, and Hemophilia A at the ASGCT Annual Meeting (GlobeNewswire, uniQure Inc.) - "uniQure N.V....presented new preclinical data on its gene therapy candidates...AMT-180 for the treatment of Hemophilia A. The data are featured in presentations at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting. uniQure is delivering a total of 22 data presentations at the meeting, which is taking place virtually from May 12 to May 15....In addition to updates on our preclinical programs, an oral presentation provides important new data on AAV biology, showing that AAV5/FIX expression was maintained long-term, up to 18 months after dosing, in mice that were treated very early in life."

Preclinical • CNS Disorders • Hemophilia

"#ASGCT20 Human Dose Prediction of AMT-180 https://t.co/rFWZjKlpQk #HemophiliaA $QURE" (@floszcrxl)

Gene Therapies • Genetic Disorders • Hematological Disorders • Hemophilia • Rare Diseases

uniQure announces 2019 financial results and highlights recent company progress (Yahoo Finance) - "Nearly all of the eligible patients in the pivotal, Phase III HOPE-B study of etranacogene dezaparvovec have received their one-time administration...Approximately 60 patients were enrolled in the study, which is being conducted in approximately 40 sites in the United States and Europe. The Company is on track to provide top-line data...before the end of this year, and to submit a Biologics License Application (BLA) for marketing authorization in 2021....The Company is producing etranacogene dezaparvovec at commercial scale for product launch and plans to conduct manufacturing process validation throughout the year....The Company is advancing multiple preclinical and nonclinical studies of AMT-180 for the treatment of hemophilia A and expects to submit an Investigational New Drug (IND) application this year."

BLA • Commercial • IND • P3 data • Trial status

uniQure to Participate in Multiple Upcoming Industry Conferences in February (GlobeNewswire, uniQure Inc.) - "uniQure will deliver 7 presentations on the development of its gene therapy candidates etranacogene dezaparvovec for hemophilia B, AMT-180 for hemophilia A, and its technology platform. uniQure will deliver 5 presentations on the development of AMT-130, the company’s AAV-gene therapy candidate for the treatment of Huntington’s disease. Professor Wolfgang Miesbach, M.D., Ph.D., of the University Hospital Frankfurt in Germany, will present 12-month follow-up data on the company’s gene therapy candidate etranacogene dezaparvovec in hemophilia B, as well as four-year follow-up data on AMT-060, also in patients with hemophilia B."

Clinical

"Dank voor overzicht. Ten aanzien van uniQure zal de koers wellicht ook reageren op eerste toediening AMT-130 (vroeg in 2020) + safety data & toekenning IND voor AMT-180 door FDA. Maar meeste aandacht zal inderdaad uitgaan naar HOPE-B (fase III) data." (@superwoester)

Clinical

uniQure announces third quarter 2019 results and highlights recent company progress (GlobeNewswire) - "In early September 2019, the Company announced the achievement of patient enrollment in its HOPE-B pivotal study of etranacogene dezaparvovec for the treatment of patients with severe and moderately severe hemophilia B....The Company expects to provide top-line Factor IX (FIX) activity data on all patients before the end of 2020, and to submit a Biologics License Application (BLA) for marketing authorization of etranacogene dezaparvovec in 2021....Numerous preclinical and nonclinical studies of AMT-180 for the treatment of hemophilia A are underway to support the submission of a clinical trial application next year."

BLA • Enrollment closed • P3 data: top line • Preclinical • Regulatory

"As presented at #ASGCT19, a single dose of #genetherapy AMT-180 promotes clinically meaningful blood-clotting activity, independent of factor VIII levels, in mouse and primate models of hemophilia A. https://t.co/wnz2g33aHA" (@ASGCTherapy)

uniQure Announces New Preclinical Data in Hemophilia A and Fabry Disease in Oral Presentations at the 22nd ASGCT Annual Meeting (GlobeNewswire, uniQure Inc.) - "uniQure N.V....will present today new preclinical data on its gene therapy candidates AMT-180 for the treatment of hemophilia A...These data will be featured today in back-to-back oral presentations at the 22nd American Society for Gene and Cell Therapy (ASGCT) Annual Meeting in Washington D.C....The mechanistic proof-of-concept of AMT-180 was demonstrated in a hemophilia A mouse model, where a single intravenous administration of AMT-180 resulted in sustained, dose-dependent hemostatic effect as measured by one-stage clotting assay."

Preclinical

uniQure Announces 2018 Financial Results and Highlights Recent Company Progress (GlobeNewswire, uniQure Inc.) - "The Company announced earlier this month that the first patient has been treated in the global Phase III HOPE-B pivotal study of AMT-061 in hemophilia B. The Company expects to complete the enrollment of approximately 50 patients in the trial by year-end....At its Research and Development Day late last year, the Company unveiled a pipeline expansion of new AAV gene therapy...AMT-180 for hemophilia A....The Company expects to advance the preclinical development of all three programs during 2019, including the initiation of a GLP toxicology study of AMT-180 in the first quarter of 2019."

Enrollment status • Preclinical

"Where is AMT-180 for Hem A in the hemophilia sector?" (@Text2Meme)