uniQure N : Announces Third Quarter 2021 Financial Results and Highlights Recent Company Progress - Form 8-K

Published: 2021-10-25 16:26:32 ET QURE

uniQure Announces Third Quarter 2021 Financial Results

and Highlights Recent Company Progress

~ Completed 78-week follow-up for hemophilia B pivotal study,

with top-line data expected by year-end 2021 ~

~ Enrolled 14 patients to date in Phase 1/2 clinical trial of AMT-130 in Huntington's Disease, with no significant safety concerns observed ~

~ Preliminary data on first four patients in ongoing Phase I/II clinical trial of AMT-130 and initiation of European Phase Ib/II clinical trial expected by year-end 2021 ~

~ Closed acquisition of Corlieve Therapeutics adding preclinical program in temporal lobe epilepsy to proprietary research pipeline ~

Lexington, MA and Amsterdam, the Netherlands, October 25, 2021 - uniQureN.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the third quarter of 2021 and highlighted recent progress across its business.

"We continue to make strong progress across our clinical trials and research programs, and CSL Behring is making significant strides towards a BLA submission for etranacogene dezaparvovec in hemophilia B, which is expected in the first half of 2022," stated Matt Kapusta, chief executive officer at uniQure."Before the end of this year, we look forward to announcing clinical data from our Huntington's disease gene therapy program, including preliminary data on the first four patients in our ongoing U.S. Phase I/II study of AMT-130. Additionally, top-line 78-week follow-up data on all HOPE-B pivotal study patients is expected to be announced by CSL Behring in late 4Q2021."

·Advancing late-stage development of etranacogene dezaparvovec (AMT-061) for the treatment of hemophilia B - licensed to CSL Behring

The Company announced initial 52-week follow-up data from the HOPE-B pivotal trial in June 2021. Data showed that after a single dose of etranacogene dezaparvovec, patients continued to demonstrate durable, sustained increases in FIX activity with a mean FIX activity of 41.5% of normal at 52 weeks of follow-up, compared to a mean of 39.0% of normal at 26-weeks of follow-up. During the 52-week period, patients reported an 80% reduction in total bleeding events requiring treatment, an 85% reduction in spontaneous bleeding events requiring treatment, and a 96% reduction in annualized usage of FIX replacement therapy.

Initial data from the first four enrolled patients in this first-in-human AAV-gene therapy trial are anticipated by year-end 2021. These initial data will focus primarily on safety up to 12 months following administration of AMT-130, as well as early imaging and biomarker data at one year. Additional clinical safety and efficacy data on longer patient follow-up in this clinical trial are expected throughout 2022.

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Upcoming Investor Events (each to be conducted virtually)

Financial Highlights

Cash Position: As of September 30, 2021, the Company held cash and cash equivalents of $578.5 million, compared to $244.9 million as of December 31, 2020. Upon the CSL Behring Agreement becoming fully effective on May 6, 2021, the Company received $462.4 million of payments. In January 2021, the Company and Hercules entered into an amended debt facility agreement, under which the Company drew down an additional $35.0 million for a total of $70.0 million outstanding under the facility as of September 30, 2021. In March and April 2021, the Company sold 921,730 ordinary shares for gross proceeds of approximately $29.6 million under an Open Market Sale Agreement with SVB Leerink LLC. In July 2021, the Company paid a net EUR 42.1 million ($49.6 million) related to its acquisition of Corlieve.

Revenues: Revenue for the three months ended September 30, 2021 was $2.0 million, compared to $1.8 million during the same period in 2020.

R&D Expenses: Research and development expenses were $36.4 million for the three months ended September 30, 2021, compared to $36.3 million during the same period in 2020. The change was primarily related to recruitment of personnel to support the development of product candidates, advancing the clinical development of the Company's Huntington's disease gene therapy program, and increased activities associated with preclinical product candidates.

SG&A Expenses: Selling, general and administrative expenses were $12.0 million for the three months ended September 30, 2021, compared to $10.8 million during the same period in 2020. The change was primarily related to recruitment of personnel and increased share-based compensation, offset partially by lower professional fees.

Other Income, net: Other income, net was $1.5 million for the three months ended September 30, 2021, compared to other income, net of $0.8 million during the same period in 2020. The increase in other income, net was primarily related to the receipt of employee retention credit under the U.S. CARES Act.

Other Non-operating Items, net: Other non-operating income, net was income of $8.6 million for the three months ended September 30, 2021, compared to other non-operating loss of $9.3 million for the same period in 2020. The increase in other non-operating income was primarily related to foreign currency gains in the current period compared to a net foreign currency loss in the same period in 2020.

Net Loss: The net loss for the three months ended September 30, 2021, was $36.5 million, or $0.79 basic and diluted net loss per share, compared to a loss of $53.8 million, or $1.21 basic and diluted loss per share during the same period in 2020.

About uniQure

uniQure is delivering on the promise of gene therapy - single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with hemophilia B, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases. www.uniQure.com

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uniQure Forward-Looking Statements

This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, the Company's expectations to announce clinical data from its gene therapy programs in hemophilia B and Huntington's disease , patient enrollment in the second cohort of AMT-130, the initiation of an open-label European study of AMT-130, the announcement of data on the first four patients in the Phase I/II study of AMT-130, the submission of the regulatory filing for marketing approval of etranacogene dezaparvovec, the Company's cash runway, or other updates on the Company's research pipeline, and its plans to advance or expand its pipeline, accelerate research, identify business development opportunities, invest in technology, or scale its manufacturing capabilities. The Company's actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with the impact of the ongoing COVID-19 pandemic on our Company and the wider economy and health care system, our Commercialization and License Agreement with CSL Behring, our clinical development activities, clinical results, collaboration arrangements, regulatory oversight, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading "Risk Factors" in the Company's periodic securities filings, including its Annual Report on Form 10-K filed March 1, 2021. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and the Company assumes no obligation to update these forward-looking statements, even if new information becomes available in the future.

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Disclaimer

uniQure NV published this content on 25 October 2021 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 25 October 2021 20:24:27 UTC.