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~ On Track to Begin Pivotal Study of AMT-061 in Hemophilia B in Q3 2018
~ Submitted IND Amendment for AMT-061 Dose Confirmation Study, with Topline Data Expected by End of 2018
~ Expects IND Submission for AMT-130 in Huntington's Disease in the Second Half of 2018
~ $159 million of cash and cash equivalents as of December 31, 2017
LEXINGTON, Mass. and AMSTERDAM, the Netherlands, March 14, 2018 (GLOBE NEWSWIRE) -- 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 2017 and highlighted recent progress across its business.
"We ended 2017 with significant momentum across all of our gene therapy programs, and a strong cash position to fund our business into 2020," stated Matthew Kapusta, chief executive officer of uniQure. "We remain highly focused on initiating our pivotal study for AMT-061 in hemophilia B and advancing AMT-130 into a Phase I/II study in Huntington's disease. We look forward to providing updates on our progress throughout the year, including the presentation of top-line clinical data on AMT-061 by year-end."
Recent Company Progress:
Upcoming Anticipated Milestones
Cash Position: As of December 31, 2017, the Company held cash and cash equivalents of $159.4 million, compared to $132.5 million as of December 31, 2016. In October 2017, the Company raised net proceeds of $85.3 million in an underwritten public offering of ordinary shares. The Company currently expects cash and cash equivalents will be sufficient to fund operations into early 2020.
Revenues: Revenue for the year ended December 31, 2017 was $13.1 million, compared to $25.1 million for the year ended December 31, 2016. Collaboration revenue for the year ended December 31, 2017 was $9.0 million, compared to $20.2 million for the year ended December 31, 2016. The decrease in collaboration revenue was primarily due to the termination of the Chiesi co-development agreement in July 2017, as well as nonrecurring revenue recognized in the prior year period associated with the production of AMT-126 product supplies.
R&D Expenses: Research and development expenses were $72.2 million for the year ended December 31, 2017, compared to $72.5 million for the year ended December 31, 2016. During the year ended December 31, 2017, we recorded expenses related to an increase in the fair market value of the contingent consideration owed to the sellers of the InoCard business, which was offset by cost savings associated with the consolidation of manufacturing activities into our Lexington, Massachusetts facility.
SG&A Expenses: Selling, general and administrative expenses were $24.6 million for the year ended December 31, 2017, compared to $26.0 million for the year ended December 31, 2016. The decrease in SG&A expenses was primarily due to certain one-time costs incurred in 2016 related to the settlement of our arbitration proceedings with Extera and the conversion of our financial reporting to U.S. GAAP. This reduction was offset, in part, by an increase in share-based compensation costs during the year ended December 31, 2017 compared to the prior year.
Other income: Other income was $15.4 million for the year ended December 31, 2017, compared to $1.5 million for the year ended December 31, 2016. The current year period includes the full amortization of the outstanding deferred revenue of $13.8 million following the termination of the Company's collaboration with Chiesi in July 2017.
Net Loss: The net loss was $79.3 million, or $2.94 per share, for the year ended December 31, compared to $73.4 million, or $2.93 per share, for the year ended December 31, 2016.
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 and partnered gene therapies to treat patients with liver/metabolic, central nervous system and cardiovascular diseases. www.uniQure.com
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,our upcoming anticipated milestones, the development of our gene therapy product candidates, the transition to our AMT-061 product candidate, the success of our collaborations and the risk of cessation, delay or lack of success of any of our ongoing or planned clinical studies and/or development of our product candidates. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our and our collaborators' clinical development activities, collaboration arrangements, corporate reorganizations and strategic shifts, regulatory oversight, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading "Risk Factors" in uniQure's Annual Report on Form 10-K filed on March 14, 2018. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future.
FOR INVESTORS: FOR MEDIA:
Maria E. Cantor Eva M. Mulder Tom Malone
Direct: 339-970-7536 Direct: +31 20 240 6103 Direct: 339-970-7558
Mobile: 617-680-9452 Mobile: +31 6 52 33 15 79 Mobile: 339-223-8541
m.cantor@uniQure.com e.mulder@uniQure.com t.malone@uniQure.com
UNAUDITED CONSOLIDATED BALANCE SHEETS
|December 31,||December 31,|
|in thousands, except share and per share amounts|
|Cash and cash equivalents||$||159,371||$||132,496|
|Accounts receivables and accrued income||1,586||9,180|
|Prepaid assets and other current assets||1,826||2,270|
|Total current assets||162,783||143,946|
|Property, plant and equipment, net||34,281||35,702|
|Intangible assets and goodwill||10,100||8,789|
|Other non-current assets||2,480||1,828|
|Total non-current assets||46,861||46,319|
|Accrued expenses and other current liabilities||8,838||9,766|
|Current portion of long-term debt||1,050||605|
|Current portion of deferred rent||737||684|
|Current portion of deferred revenue||4,613||6,142|
|Current portion of contingent consideration||1,084||-|
|Total current liabilities||19,230||22,721|
|Long-term debt, net of current portion||19,741||19,631|
|Deferred rent, net of current portion||9,114||6,781|
|Deferred revenue, net of current portion||67,408||75,612|
|Contingent consideration, net of current portion||2,880||1,838|
|Derivative financial instruments related party||1,298||51|
|Other non-current liabilities||614||-|
|Total non-current liabilities||101,055||103,913|
|Total shareholders' equity||89,359||63,631|
|Total liabilities and shareholders' equity||$||209,644||$||190,265|
UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS
|Years ended December 31, |
|in thousands, except share and per share amounts|
|Research and development expenses||(72,172||)||(72,510||)||(59,125||)|
|Selling, general and administrative expenses||(24,635||)||(25,999||)||(23,383||)|
|Total operating expenses||(96,807||)||(98,509||)||(82,508||)|
|Loss from operations||(71,343||)||(71,946||)||(71,151||)|
|Non operating items, net||(8,116||)||(283||)||(12,111||)|
|Loss before income tax expense||(79,459||)||(72,229||)||(83,262||)|
|Income tax benefit / (expense)||199||(1,145||)||1,179|
|Basic and diluted net loss per common share||$||(2.94||)||$||(2.93||)||$||(3.72||)|
|Weighted average shares used in computing basic and diluted net loss per common share||26,984,183||25,036,465||22,082,345|| |
Huntington's disease is a hereditary disease caused by a defect in a single gene on Chromosome 4 that is inherited in an autosomal dominant fashion. The defect causes a part of DNA, called a CAG repeat, to occur many more times than it is supposed to...
Gene therapy is the use of DNA as a pharmaceutical agent to treat disease. It derives its name from the idea that DNA can be used to supplement or alter genes within an individual's cells as a therapy to treat disease. The most common form of gene th...
Biological therapy involves the use of living organisms, substances derived from living organisms, or laboratory-produced versions of such substances to treat disease. Some biological therapies for cancer use vaccines or bacteria to stimulate the body&rs...