EX-99.1 2 a16-21899_1ex99d1.htm EX-99.1

Exhibit 99.1

 

 

uniQure Announces Financial Results For

Third Quarter 2016 and Provides Update on Company Progress

 

~ Strategic Review Completed to Refocus Pipeline, Reduce Operating Costs and Deliver

Long-Term Shareholder Value

 

~ Updated Clinical Data on AMT-060 in Hemophilia B at Upcoming ASH Annual Meeting

 

Lexington, MA and Amsterdam, the Netherlands, November 22, 2016 — uniQure N.V. (NASDAQ: QURE), a leader in human gene therapy, today announced its financial results for the third quarter ended September 30, 2016 and provided an update on corporate developments.

 

“With the completion of our company-wide strategic review, we have focused our pipeline, streamlined  operations and improved our financial position,” stated Matthew Kapusta, interim chief executive officer of uniQure. “Our near-term priorities are to aggressively prepare for a pivotal study in hemophilia B and to complete IND-enabling studies for our programs in Huntington’s disease and congestive heart failure.  We look forward to the presentation of updated Phase I/II data on AMT-060 in a couple of weeks at ASH, and discussing our late-stage development plans.”

 

Recent Highlights

 

·                  Completed strategic review to refocus pipeline, simplify organization and reduce operating expenses — On November 15, uniQure announced the completion of a company-wide strategic review aimed at focusing its pipeline, consolidating its manufacturing and enhancing overall execution to drive shareholder value.  As a result of this initiative, the Company will prioritize programs in hemophilia B, Huntington’s disease and those associated with its landmark collaboration with Bristol-Myers Squib (BMS) in cardiovascular disease.  Regarding the Company’s clinical product candidates in Sanfilippo B and Parkinsons’ disease, the Company has initiated discussions with its collaborators to explore options for these programs, including their potential transfer or partnership.  Additionally, the Company will restructure its research and development organization in the Netherlands and consolidate manufacturing in the United States.

 

·                  Updated Phase I/II data on AMT-060 at ASH; Preparations for pivotal study underway — Professor Frank W.G. Leebeek, a clinical investigator at the Erasmus Medical Center in Rotterdam, the Netherlands, will be presenting new and updated data from uniQure’s ongoing Phase I/II clinical trial of AMT-060 at the 58th American Society of Hematology (ASH) Annual Meeting, on Saturday, December 3, 2016. The data will include up to 52 weeks of follow-up on the first patient cohort receiving 5x1012 gc/kg and up to 26 weeks of follow-up on the second patient cohort receiving a higher dose of 2x1013 gc/kg.  In early 2017, uniQure expects to initiate interactions with regulatory authorities regarding a potential pivotal study of AMT-060 and will commence scaled-up production of clinical material in its Lexington, MA manufacturing facility.

 

·                  Progressing IND-enabling studies of AMT-130 — uniQure is conducting ongoing preclinical studies of AMT-130, its wholly owned AAV5 gene therapy product candidate for Huntington’s disease.  AMT-130 has demonstrated in preclinical studies up to 80 percent knockdown of the Huntingtin gene in the cortex and putamen in rat and humanized mouse models.  Additional preclinical data, presentations and publications are expected in 2017 that will feature ongoing progress in moving toward filing an investigational new drug (IND) application to begin clinical studies.

 

·                  Advancing S100A1 through multiple preclinical studies — Significant progress has been made over the past year in transferring S100A1 from a mammalian system to an insect cell preparation, as well as conducting preclinical dose-ranging analyses and comparability studies.  Various administration techniques have been analyzed and

 



 

further animal studies are expected in 2017 to support an IND filing in 2018.  uniQure also has begun early work on two additional cardiovascular targets, including vector development and other preclinical activities.

 

·                  Maintaining strong cash position sufficient to fund operations into 2019 — As of September 30, 2016, the Company held cash and cash equivalents of €140.3 million.  uniQure expects to realize significant cost savings as a result of the activities associated with its strategic restructuring. Specifically, uniQure expects to realize €5 million to €6 million of annualized cost savings in personnel and other related operating expenses as a result of the elimination of approximately 50 to 60 positions, or 20% to 25% of global headcount, by the end of 2017. Additionally, the Company expects to further reduce planned operating expenses by €11 million to €15 million over the next two years through the focusing of its pipeline. Based on its strong cash position and these cost savings, uniQure believes its existing cash resources will be sufficient to fund operations into 2019.

 

Financial Highlights for Q3 2016

 

Revenues for the three months ended September 30, 2016 were €6.5 million, compared with €3.1 million for the comparable period in 2015. The increase relates to increased research activity associated with S100A1 for heart failure, the expenses of which are fully reimbursed by BMS in accordance with the Company’s collaboration agreement.

 

Research and development expenses were €15.3 million for the three months ended September 30, 2016, compared with €11.9 million for the comparable period in 2015. The increase is mainly due to increased activity in support of our collaboration agreement with BMS, validation and scale-up activities related to AMT-060 in the Company’s Lexington facility, increased facility costs in Amsterdam and the continued progression of our clinical program for AMT-060 in hemophilia B and preclinical candidate AMT-130 in Huntington’s disease.

 

Selling, general and administrative expenses were €4.1 million for the three months ended September 30, 2016, compared with €4.8 million for the comparable period in 2015. The decrease results from a reduction in professional fees and a reversal of share based payment expenses for unvested equity grants to our former CEO upon his resignation in September 2016, offset in part by expenses associated with the Glybera Phase IV study and global registry, which in prior years were capitalized.

 

Losses related to foreign currency effects on U.S. dollar-denominated deposits were €0.6 million for the three months ended September 30, 2016, compared to €1.6 million for the comparable period in 2015. Net finance costs were €0.2 million for the three months ended September 30, 2016, compared to a gain of €1.2 million for the comparable period in 2015.

 

The net loss for the third quarter 2016 was €13.5 million, or €0.54 per share, compared with €25.8 million, or €1.08 per share, for the third quarter of 2015.

 

As of September 30, 2016, the Company held cash and cash equivalents of €140.3 million and had 25,143,760 ordinary shares outstanding.  Cash used in operating and investing activities in the third quarter of 2016 includes approximately €6.6 million of one-time capital expenditures related to the refurbishment of a new R&D facility in Amsterdam, the Netherlands. Excluding these one-time expenditures related to the new facility, cash used in operating and investing activities in the third quarter of 2016 would have been €18.0 million.

 

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 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,

 

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statements regarding the implementation and effects of the Company’s new strategic and organizational changes, the development of our gene therapy product candidates, 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 corporate reorganizations and strategic shifts, collaboration arrangements, our and our collaborators’ clinical development activities, 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 2015 Annual Report on Form 20-F filed with the Securities and Exchange Commission on April 4, 2016. 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.

 

uniQure Contacts:

 

Maria E. Cantor

Tom Malone

 

 

Direct: 339-970-7536

Direct: 339-970-7558

Mobile: 617-680-9452

Mobile: 339-223-8541

m.cantor@uniQure.com

t.malone@uniQure.com

 

 

Eva M. Mulder

 

 

 

Direct: +31 20 240 6103

 

Mobile: +31 6 52 33 15 79

 

e.mulder@uniQure.com

 

 

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UNIQURE N.V.

Unaudited Condensed Consolidated Statements of Financial Position

(€ in thousands)

 

 

 

December 31,

 

September 30,

 

 

 

2015

 

2016

 

Assets

 

 

 

 

 

Non-current assets

 

 

 

 

 

Goodwill

 

442

 

442

 

Intangible assets other than Goodwill

 

7,209

 

8,771

 

Property, plant and equipment

 

23,820

 

29,730

 

Other non-current assets

 

1,142

 

1,663

 

Total non-current assets

 

32,613

 

40,606

 

Current assets

 

 

 

 

 

Receivables from related parties

 

3,792

 

3,591

 

Trade and other receivables

 

1,730

 

6,142

 

Inventories

 

435

 

70

 

Cash and cash equivalents

 

203,532

 

140,331

 

Total current assets

 

209,489

 

150,134

 

Total assets

 

242,102

 

190,740

 

Equity

 

 

 

 

 

Share capital

 

1,216

 

1,257

 

Share premium

 

344,803

 

346,753

 

Other reserves

 

26,026

 

30,361

 

Accumulated deficit

 

(252,561

)

(305,989

)

Total equity

 

119,484

 

72,382

 

Liabilities

 

 

 

 

 

Non-current liabilities

 

 

 

 

 

Borrowings

 

13,434

 

18,246

 

Derivative financial instruments - related party

 

530

 

98

 

Deferred rent

 

5,737

 

5,922

 

Deferred revenue

 

75,852

 

72,335

 

Contingent consideration

 

2,687

 

1,286

 

Total non-current liabilities

 

98,240

 

97,887

 

Current liabilities

 

 

 

 

 

Trade and other payables

 

11,220

 

11,678

 

Derivative financial instruments - related parties

 

992

 

182

 

Borrowings

 

5,124

 

 

Borrowings - derivative

 

238

 

41

 

Deferred rent

 

579

 

597

 

Deferred revenue

 

6,225

 

5,573

 

Provisions

 

 

2,400

 

Total current liabilities

 

24,378

 

20,471

 

Total liabilities

 

122,618

 

118,358

 

Total equity and liabilities

 

242,102

 

190,740

 

 

4



 

UNIQURE N.V.

Unaudited Condensed Consolidated Statements of Operations and Comprehensive Loss

(€ in thousands except share and per share data)

 

 

 

Three months ended

 

Nine months ended

 

 

 

September 30,

 

September 30,

 

 

 

2015

 

2016

 

2015

 

2016

 

License revenues

 

1,041

 

1,100

 

1,754

 

3,302

 

Collaboration revenues

 

1,809

 

5,366

 

3,818

 

11,007

 

Product sales

 

300

 

 

300

 

 

Total revenues

 

3,150

 

6,466

 

5,872

 

14,309

 

Cost of goods sold

 

(428

)

(75

)

(428

)

(227

)

Other income

 

186

 

302

 

532

 

1,126

 

Research and development expenses

 

(11,933

)

(15,281

)

(32,652

)

(47,744

)

Selling, general and administrative expenses

 

(4,835

)

(4,140

)

(13,503

)

(16,248

)

Impairment of intangible assets

 

(11,640

)

 

(11,640

)

 

Other gains/(losses), net

 

(1,552

)

(623

)

(2,548

)

(5,002

)

Total operating costs

 

(30,202

)

(19,817

)

(60,239

)

(68,095

)

Operating loss

 

(27,052

)

(13,351

)

(54,367

)

(53,786

)

Finance income

 

5

 

280

 

70

 

1,864

 

Finance expense

 

1,173

 

(450

)

(3,102

)

(1,506

)

Finance income/(expense), net

 

1,178

 

(170

)

(3,032

)

358

 

Result before corporate income tax

 

(25,874

)

(13,521

)

(57,399

)

(53,428

)

Corporate income taxes

 

30

 

 

30

 

 

Net loss

 

(25,844

)

(13,521

)

(57,369

)

(53,428

)

Items that may be subsequently reclassified to profit or loss:

 

 

 

 

 

 

 

 

 

Currency translation differences on foreign operations

 

(159

)

(132

)

927

 

(433

)

Other comprehensive income/(loss)

 

(159

)

(132

)

927

 

(433

)

Total comprehensive loss

 

(26,003

)

(13,653

)

(56,442

)

(53,861

)

Loss per share attributable to the equity holders of the Company during the period:

 

 

 

 

 

 

 

 

 

Basic and diluted loss per share

 

(1.08

)

(0.54

)

(2.69

)

(2.14

)

 

5



 

UNIQURE N.V.

Unaudited Condensed Consolidated Statements of Changes in Equity/Deficit

(€ in thousands)

 

 

 

Share

 

Share

 

Other

 

Accumulated

 

Total

 

 

 

capital

 

premium

 

reserves

 

deficit

 

equity

 

Balance at January 1, 2015

 

905

 

206,111

 

17,149

 

(181,081

)

43,084

 

Loss for the period

 

 

 

 

(57,369

)

(57,369

)

Other comprehensive income/(loss)

 

 

 

927

 

 

927

 

Total comprehensive income/(loss)

 

 

 

927

 

(57,369

)

(56,442

)

Capital contributions

 

306

 

138,877

 

 

 

139,183

 

Share issuance costs

 

 

(612

)

 

 

(612

)

Share based payment expense

 

 

 

5,242

 

 

5,242

 

Balance at September 30, 2015

 

1,211

 

344,376

 

23,318

 

(238,450

)

130,455

 

Loss for the period

 

 

 

 

(14,111

)

(14,111

)

Other comprehensive income/(loss)

 

 

 

323

 

 

323

 

Total comprehensive income/(loss)

 

 

 

323

 

(14,111

)

(13,788

)

Capital contributions

 

5

 

427

 

 

 

432

 

Share based payment expense

 

 

 

2,385

 

 

2,385

 

Balance at December 31, 2015

 

1,216

 

344,803

 

26,026

 

(252,561

)

119,484

 

Loss for the period

 

 

 

 

(53,428

)

(53,428

)

Other comprehensive income/(loss)

 

 

 

(433

)

 

(433

)

Total comprehensive income/(loss)

 

 

 

(433

)

(53,428

)

(53,861

)

Capital contributions

 

41

 

1,950

 

 

 

1,991

 

Share based payment expense

 

 

 

4,768

 

 

4,768

 

Balance at September 30, 2016

 

1,257

 

346,753

 

30,361

 

(305,989

)

72,382

 

 

6



 

UNIQURE N.V.

Unaudited Condensed Consolidated Statements of Cash Flows

(€ in thousands)

 

 

 

Nine months ended

 

 

 

September 30,

 

 

 

2015

 

2016

 

Cash flow from operating activities

 

 

 

 

 

Net loss

 

(57,369

)

(53,428

)

Adjustments for:

 

 

 

 

 

Amortization on intangible assets

 

274

 

600

 

Impairment of intangible assets

 

11,640

 

 

Depreciation

 

2,872

 

3,718

 

Lease incentive

 

129

 

(607

)

Loss/(gain) on derivatives

 

164

 

(1,439

)

Loss/(gain) on foreign exchanges

 

3,160

 

1,829

 

Share-based payment expenses

 

5,242

 

4,768

 

Changes in provisions

 

 

2,400

 

Changes in trade and other receivables

 

(3,339

)

(4,211

)

Changes in inventories

 

(210

)

365

 

Changes in other current liabilities

 

1,933

 

429

 

Changes in deferred revenue

 

65,407

 

(4,169

)

Cash generated from/(used in) operations

 

29,903

 

(49,745

)

Interest paid

 

(1,404

)

(1,434

)

Net cash generated from/(used in) operating activities

 

28,499

 

(51,179

)

Cash used in investing activities

 

 

 

 

 

Purchases of property, plant and equipment

 

(3,289

)

(9,502

)

Purchases of intangible assets

 

(2,908

)

(1,716

)

Changes in restricted cash

 

 

(555

)

Interest received

 

70

 

61

 

Net cash used in investing activities

 

(6,127

)

(11,712

)

Cash flow from financing activities

 

 

 

 

 

Proceeds from shares issued

 

137,297

 

1,982

 

Payments of finance lease

 

(125

)

(134

)

Net cash generated from financing activities

 

137,172

 

1,848

 

Net increase/(decrease) in cash and cash equivalents

 

159,544

 

(61,043

)

Currency effect cash and cash equivalents

 

1,731

 

(2,158

)

Cash and cash equivalents at January 1

 

53,219

 

203,532

 

Cash and cash equivalents at September 30

 

214,494

 

140,331

 

 

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