Exhibit 99.1

 

 

uniQure Announces Second Quarter 2018 Financial Results and Highlights Company Progress

 

~ Patient Enrollment Underway in Global Phase III HOPE-B Pivotal Study of AMT-061

 

~ First Patient Successfully Screened into Phase IIb Dose-Confirmation Trial of AMT-061

 

~ Robert Gut, M.D., Ph.D., Named Chief Medical Officer and Other Appointments to Clinical Development Leadership

 

~ Ended Second Quarter with $259 Million in Cash and Cash Equivalents; Expected to Fund Operations into 2021

 

Lexington, MA and Amsterdam, the Netherlands, August 8, 2018 — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the second quarter of 2018 and highlighted recent progress across its business.

 

“We have made tremendous progress over the past several months advancing our gene therapy programs in hemophilia B and Huntington’s disease, as well as in expanding our intellectual property portfolio and executing on our manufacturing plan,” said Matt Kapusta, chief executive officer of uniQure. “Our Phase III pivotal trial of AMT-061 is actively enrolling patients, and we have consented and successfully screened the first of three patients in our Phase IIb dose-confirmation study.”

 

“The initiation of our registrational study provides the opportunity for uniQure to be first to market with a one-time treatment that has the potential to transform the standard of care for patients with hemophilia B,” Mr. Kapusta added. “We continue to work toward what we expect will be an equally productive second half of 2018 with the achievement of multiple value-creating catalysts across the company, including top-line data from our dose-confirmation study, the submission of an Investigational New Drug application for AMT-130 in Huntington’s disease, and hosting a Research and Development Day that will feature expansion of our early-stage pipeline.”

 

Recent Company Progress

 

·                 Advancing AMT-061 into late-stage clinical development for the treatment of hemophilia B

 

·                  The first patient in the Phase IIb dose-confirmation study of AMT-061 in hemophilia B has consented and successfully screened into the study and is expected to be treated shortly. The Phase IIb study is an open-label, single-arm, single-dose trial being conducted in multiple centers in the U.S. Three patients will receive a single intravenous (IV) infusion of 2x1013 vc/kg and be evaluated for a period of approximately six to eight weeks to assess Factor IX (FIX) activity and confirm the dose for the global Phase III HOPE-B study. Top-line data are expected to be made available by the end of this year.

 

·                  Patient enrollment has commenced for the global Phase III HOPE-B pivotal study. This registrational study includes a six-month lead-in phase to collect baseline data as patients will serve as their own control. The Company expects to begin dosing patients with AMT-061 in the first quarter of 2019.

 


The following information was filed by Uniqure N.V. (QURE) on Wednesday, August 8, 2018 as an 8K 2.02 statement, which is an earnings press release pertaining to results of operations and financial condition. It may be helpful to assess the quality of management by comparing the information in the press release to the information in the accompanying 10-Q Quarterly Report statement of earnings and operation as management may choose to highlight particular information in the press release.

View differences made from one quarter to another to evaluate Uniqure N.V.'s financial trajectory

Compare SEC Filings Year-over-Year (YoY) and Quarter-over-Quarter (QoQ)
Sample 10-K Year-over-Year (YoY) Comparison

Compare this 10-Q Quarterly Report to its predecessor by reading our highlights to see what text and tables were  removed  ,   added    and   changed   by Uniqure N.V..

Continue

Never Miss A New SEC Filing Again


Real-Time SEC Filing Notifications
Screenshot taken from Gmail for a new 10-K Annual Report
Last10K.com Member Feature

Receive an e-mail as soon as a company files an Annual Report, Quarterly Report or has new 8-K corporate news.

Continue

We Highlighted This SEC Filing For You


SEC Filing Sentiment Analysis - Bullish, Bearish, Neutral
Screenshot taken from Wynn's 2018 10-K Annual Report
Last10K.com Member Feature

Read positive and negative remarks made by management in their entirety without having to find them in a 10-K/Q.

Continue

Widen Your SEC Filing Reading Experience


Increased Reading Area for SEC Filings
Screenshot taken from Adobe Inc.'s 10-Q Quarterly Report
Last10K.com Member Feature

Remove data columns and navigations in order to see much more filing content and tables in one view

Continue

Uncover Actionable Information Inside SEC Filings


SEC Filing Disclosures
Screenshot taken from Lumber Liquidators 10-K Annual Report
Last10K.com Member Feature

Read both hidden opportunities and early signs of potential problems without having to find them in a 10-K/Q

Continue

Adobe PDF, Microsoft Word and Excel Downloads


Download Annual and Quarterly Reports as PDF, Word and Excel Documents
Screenshots of actual 10-K and 10-Q SEC Filings in PDF, Word and Excel formats
Last10K.com Member Feature

Export Annual and Quarterly Reports to Adobe PDF, Microsoft Word and Excel for offline viewing, annotations and analysis

Continue

FREE Financial Statements


Download Annual and Quarterly Reports as PDF, Word and Excel Documents
Screenshot of actual balance sheet from company 10-K Annual Report
Last10K.com Member Feature

Get one-click access to balance sheets, income, operations and cash flow statements without having to find them in Annual and Quarterly Reports

Continue for FREE

Intrinsic Value Calculator


Intrinsic Value Calculator
Screenshot of intrinsic value for AT&T (2019)
Last10K.com Member Feature

Our Intrinsic Value calculator estimates what an entire company is worth using up to 10 years of financial ratios to determine if a stock is overvalued or not

Continue

Financial Stability Report


Financial Stability Report
Screenshot of financial stability report for Coco-Cola (2019)
Last10K.com Member Feature

Our Financial Stability reports uses up to 10 years of financial ratios to determine the health of a company's EPS, Dividends, Book Value, Return on Equity, Current Ratio and Debt-to-Equity

Continue

Get a Better Picture of a Company's Performance


Financial Ratios
Available Financial Ratios
Last10K.com Member Feature

See how over 70 Growth, Profitability and Financial Ratios perform over 10 Years

Continue

Log in with your credentials

or    

Forgot your details?

Create Account