EX-99.1 2 a16-7881_1ex99d1.htm EX-99.1

Exhibit 99.1

 

 

uniQure Announces Results for Fourth Quarter and Financial Year 2015

 

Amsterdam, the Netherlands, April 4, 2016 — uniQure N.V. (NASDAQ: QURE), a leader in human gene therapy, today announced audited results for the fourth quarter and year ending December 31, 2015, and provided a corporate overview on its pipeline programs and operations.

 

“Over the past several months, we announced proof-of-concept results from our Phase I/II clinical trials in Sanfilippo B and hemophilia B. Both studies demonstrated not only encouraging safety and efficacy data, but also validated our proprietary AAV5 technology and insect cell, baculovirus production system in both the brain and the liver,” said Dan Soland, CEO of uniQure. “In 2016, we will move closer to advancing these lead programs into pivotal studies and initiate Investigational New Drug (IND)-enabling studies on additional product candidates that are expected to enter human clinical trials in 2017.  We are particularly looking forward to sharing longer follow-up data from our ongoing Phase I/II clinical study of AMT-060 in hemophilia B and from the Sanfilippo B patients.  Since joining uniQure in December, I am increasingly convinced that our validated technology platform, commercial-scale manufacturing capabilities and broad clinical and preclinical programs will continue to drive our leadership in gene therapy.”

 

Corporate Highlights

 

Liver/Metabolism Therapeutic Focus Area

 

·                  Reported Encouraging Top-line Data from Low-Dose Cohort in Phase I/II Study of AMT-060 — On January 7, 2016, uniQure announced preliminary topline results from the first, low-dose cohort in the ongoing AMT-060-01 Phase I/II Hemophilia B study. Initial results showed that AMT-060 was generally well tolerated and the first two patients that completed at least 12 weeks of follow-up showed promising Factor IX expression levels of 5.5% and 4.5% of normal. Additionally, four out of five patients discontinued recombinant FIX prophylaxis as of January 6, 2016.  These early data validate successful transduction of the liver using uniQure’s proprietary AAV5 vector.  uniQure expects to report results on all five patients from the low-dose cohort at a scientific conference in the second quarter of 2016.

 

·                  Initiated Dosing of AMT-060 in High-Dose Cohort — On March 14, 2016, the Company announced that the first patient in the high-dose cohort of the AMT-060-01 trial had been treated.  As of today, 2 patients in the high-dose cohort have been treated. All 8 patients screened so far in the Phase I/II trial have tested negative for anti-AAV5 antibodies.

 

CNS Therapeutic Focus Area

 

·                  Positive Results from Phase I/II Study of AMT-110 in Sanfilippo B — On September 19, 2015, encouraging results from an academic-sponsored Phase I/II trial in Sanfilippo B using uniQure’s novel AAV5-based gene therapy were presented at the European Society of Gene and Cell Therapy (ESGCT) in Helsinki, Finland. In all four patients, researchers verified the restoration of catalytical activity of the NaGlu protein in the cerebrospinal fluid (CSF) from 0% at baseline up to 14-17% of normal at three months and maintained further at 12 months. The trial also demonstrated that incremental cognitive development was maintained in all four patients with no progression of brain atrophy detected via MRI scans. These data validate the effective transmission of the NaGlu gene into the brain with uniQure’s proprietary AAV5 viral vector.  uniQure expects to present 30-month follow-up data from the four patients in early 2017.

 

·                  Sponsorship of Extension Protocol for Phase I/II Study of AMT-110 to Transition to uniQure — In January 2016, uniQure and the academic consortium comprised of Institut Pasteur, the French Muscular Dystrophy Association, Vaincre les Maladies Lysosomales and Institut National de la Santé et de la Recherche Médicale (INSERM), executed a term sheet reflecting the license of certain data and intellectual property from the consortium-sponsored Phase I/II study of AMT-110 in Sanfilippo B. uniQure and the consortium are currently

 



 

negotiating the terms of a definitive agreement. Additionally, uniQure has assumed the sponsorship of the Phase I/II extension study, enabling the Company to continue the follow-up of the four patients treated to date.

 

·                  Phase I Study in Parkinson’s Disease to Complete Enrolling Second Cohort — A Phase I clinical study in Parkinson’s disease led by Krystof Bankiewicz, MD, PhD of the University of California at San Francisco together with John D. Heiss, MD, and colleagues from the National Institutes of Heath, using an AAV-glial cell line-derived neurotrophic factor (GDNF) product licensed from AMGEN for gene therapy applications, has completed enrollment of its first of four six-patient cohorts and is expected to complete the treatment of the second cohort later in the year.

 

·                  Preclinical Proof-of-Concept Achieved in Huntington’s Disease — On March 22, 2016, uniQure announced the publication of preclinical data in the March 2016 edition of Molecular Therapy-Nucleic Acids which showed that AMT-130, a novel AAV5-based gene therapy candidate for Huntington’s disease, achieved preclinical proof-of-concept.  The studies, which were conducted in vitro and in a humanized mouse model, demonstrated silencing of the mutated Huntingtin gene with therapeutic microRNAs delivered via an AAV5 vector.  uniQure has selected its lead candidate and has initiated IND-enabling studies.

 

Cardiovascular Therapeutic Focus Area

 

·                  Four Targets Designated by BMS; Advancing S100A1 Gene Therapy Towards IND Filing — On April 6, 2015, uniQure announced a collaboration with Bristol-Myers Squibb in which the two companies would develop gene therapies for cardiovascular diseases. In total, the parties may collaborate on up to ten targets, of which four have been designated.  uniQure and BMS are currently conducting safety and toxicology studies for the S100A1 gene therapy using uniQure’s proprietary insect-cell, baculovirus expression system and are advancing towards an IND filing.

 

Technology and Manufacturing

 

·                  Lexington Facility Operational and in Process of Ramping Up to Commercial-Scale Production — In 2015, uniQure completed the build-out of its 53,000 sq. ft. fully scalable gene therapy manufacturing plant in Lexington, Massachusetts. The facility is operational and currently producing research batches.  Scale-up of AMT-060 production is currently underway and expected to be completed in 2016 in order to supply clinical GMP material for a pivotal study in hemophilia B.

 

·                  Designation of AAV Capsid Variants with 4D; Initiating Preclinical Validation — In the fourth quarter of 2015, uniQure designated specific synthetic AAV capsid variants derived from 4D’s proprietary capsid library for further improvement of efficacy in both the CNS and Liver/Metabolism Therapeutic Focus Areas. uniQure will initiate preclinical validation of the designated capsid variants in non-human primates in 2016.

 

·                  Designation of Synthetic Liver-Specific Promoters from Synpromics Ltd; Initiating Preclinical Validation— As part of the cooperation and license agreement signed in January 2015, Synpromics delivered a series of synthetic liver-specific promoters derived from their rationally-designed promoter library technology. uniQure will use these promoters to further improve the therapeutic activity of AAV-vectors in the liver. uniQure will initiate preclinical testing of these promoters in non-human primates during 2016.

 

Corporate Finance

 

·                  Strong Financial Position — As of December 31, 2015, uniQure had €203.5 million of cash on hand, which is expected to fund operations into the second half of 2018.  To date, uniQure has received approximately $140 million in upfront and other consideration from BMS.  In addition, on April 7, 2015, uniQure closed a follow-on public offering of 3,000,000 ordinary shares at an offering price of $29.50 per share raising a total of $83.2 million.

 

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Human Resources

 

·                  Expanding Key Talent — Over the course of 2015 uniQure has strengthened its management team with experienced industry leaders in its two key locations in Lexington, Massachusetts and Amsterdam, including the appointment of Dan Soland as CEO in December 2015, Matt Kapusta as CFO in January 2015, Dr. Charlie Richard as SVP, R&D Neuroscience in July 2015 and Dr. Deya Corzo, SVP, R&D Liver/Metabolism in July 2015.

 

Financial Highlights

 

As of December 31, 2015, the Company held cash and cash equivalents of €203.5 million, compared with €53.2 million as of December 31, 2014. The increase was due to the consideration received from BMS during the period and the Company’s follow-on offering in April 2015, offset in part by cash used in research, development and general corporate activities.

 

Revenue for 2015 was €9.4 million, compared with €4.7 million in 2014. These revenues are primarily related to the Company’s collaboration agreements with Bristol-Myers Squibb and Chiesi, as well as Glybera product sales, which commenced in the third quarter of 2015.

 

Cost of goods sold in 2015 was €0.6 million, compared with zero in 2014.  Cost of goods sold includes the cost of Glybera product sales and amortization of specific Glybera-related license agreements, which were required to be expensed upon the initiation of commercialization in the third quarter of 2015.

 

Research and development expenses were €46.8 million in 2015, compared to €33.9 million in 2014. The increase is related to the continuation of uniQure’s Phase I/II clinical study of AMT-060 in hemophilia B, the continued progression of uniQure’s other product candidates and increased activity in the Company’s U.S. facility.

 

Selling, general and administrative expenses were €19.3 million in 2015, compared with €11.2 million in 2014. The increase was primarily due to expenses related to consultants and professional fees associated with business development, expenses related to the Company’s follow-on offering conducted in April 2015 and other general and administrative activities.

 

Other gains/losses were a loss of €0.2 million for 2015, compared to a gain of €5.8 million in 2014. The loss was primarily attributable to the impact of foreign currency exchange rates on the Company’s dollar-denominated deposits.

 

In the third quarter of 2015, the Company incurred a one-time, non-recurring impairment charge on certain Glybera-related intangible assets of €11.6 million related to the revision of the Company’s forecasted number of patients treated with Glybera and certain reimbursement-related developments in Europe.

 

The net loss for the fourth quarter of 2015 was €14.1 million, or €0.58 per share, compared with €11.2 million, or €0.62 per share, for the fourth quarter of 2014.  The net loss for the full years 2015 and 2014 was €71.5 million, or €3.24 per share and €37.0 million, or €2.16 per share, respectively.

 

For further financial information for the period ending December 31, 2015, please refer to the financial statements appearing at the end of this release.

 

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 CNS, liver/metabolic 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

 

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include, but are not limited to, statements regarding the development of our gene therapiesthe 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 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 2014 Annual Report on Form 20-F filed with the Securities and Exchange Commission on April 25, 2014 and its 2015 Annual Report on Form 20-F to be filed with the Securities and Exchange Commission on or about the date hereof. 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:

 

Eva Mulder

Direct: +31 20 240 6103

Main: +31 20 240 6000

e.mulder@uniQure.com

 

Media inquiries:

 

Gretchen Schweitzer

MacDougall Biomedical Communications

Direct: +49 172 861 8540

Main: +49 89 2424 3494 or +1 781 235 3060

gschweitzer@macbiocom.com

 

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uniQure N.V.

 

Consolidated Statements of Financial Position
(€ in thousands)

 

 

 

 

 

DECEMBER 31,

 

DECEMBER 31,

 

 

 

NOTE

 

2014

 

2015

 

Assets

 

 

 

 

 

 

 

Non-current assets

 

 

 

 

 

 

 

Goodwill

 

2, 5, 6

 

1,342

 

442

 

Intangible assets other than Goodwill

 

5, 6

 

16,368

 

7,209

 

Property, plant and equipment

 

7

 

19,667

 

23,820

 

Other non-current assets

 

8

 

1,022

 

1,142

 

Total non-current assets

 

 

 

38,399

 

32,613

 

Current assets

 

 

 

 

 

 

 

Receivables from related parties

 

9

 

2,426

 

3,792

 

Trade and other receivables

 

9

 

1,542

 

1,730

 

Inventories

 

10

 

200

 

435

 

Cash and cash equivalents

 

11

 

53,219

 

203,532

 

Total current assets

 

 

 

57,387

 

209,489

 

Total assets

 

 

 

95,786

 

242,102

 

Equity

 

 

 

 

 

 

 

Share capital

 

 

 

905

 

1,216

 

Share premium

 

 

 

206,111

 

344,803

 

Other reserves

 

 

 

17,149

 

26,026

 

Accumulated deficit

 

 

 

(181,081

)

(252,561

)

Total equity

 

12

 

43,084

 

119,484

 

Liabilities

 

 

 

 

 

 

 

Non-current liabilities

 

 

 

 

 

 

 

Borrowings

 

14

 

16,418

 

13,434

 

Derivative financial instruments- related parties

 

14

 

 

530

 

Financial lease liabilities

 

15

 

134

 

 

Deferred rent

 

27

 

5,658

 

5,737

 

Deferred revenue

 

17

 

15,387

 

75,852

 

Deferred tax liabilities

 

5, 23

 

1,379

 

 

Contingent considerations

 

5

 

1,454

 

2,687

 

Total non-current liabilities

 

 

 

40,430

 

98,240

 

Current liabilities

 

 

 

 

 

 

 

Trade and other payables

 

15, 16

 

9,617

 

11,220

 

Derivative financial instruments - related parties

 

14

 

645

 

992

 

Borrowings

 

14

 

 

5,124

 

Borrowings - derivative

 

14

 

207

 

238

 

Deferred rent

 

27

 

475

 

579

 

Deferred revenue

 

17

 

1,328

 

6,225

 

Total current liabilities

 

 

 

12,272

 

24,378

 

Total liabilities

 

 

 

52,702

 

122,618

 

Total equity and liabilities

 

 

 

95,786

 

242,102

 

 

5



 

uniQure N.V.

 

Consolidated Statements of Comprehensive Loss
(€ in thousands, except share and per share data)

 

 

 

 

 

TWELVE MONTHS ENDED

 

 

 

 

 

DECEMBER 31,

 

 

 

NOTE

 

2013

 

2014

 

2015

 

License revenues

 

17

 

440

 

883

 

2,854

 

Collaboration revenues

 

17

 

2,503

 

3,802

 

6,271

 

Product sales

 

17

 

 

 

300

 

Total revenues

 

 

 

2,943

 

4,685

 

9,425

 

Cost of goods sold

 

18

 

(800

)

 

(584

)

Other income

 

21

 

585

 

773

 

708

 

Research and development expenses

 

18, 19

 

(13,182

)

(33,932

)

(46,781

)

Selling, general and administrative expenses

 

18, 20

 

(11,628

)

(11,167

)

(19,317

)

Impairment of intangible assets

 

6

 

 

 

(11,640

)

Other gains / losses, net

 

 

 

(453

)

5,807

 

(248

)

Total operating costs

 

 

 

(25,478

)

(38,519

)

(77,862

)

Operating result

 

 

 

(22,535

)

(33,834

)

(68,437

)

Finance income

 

 

 

102

 

254

 

549

 

Finance expense

 

22

 

(4,387

)

(3,460

)

(4,023

)

Finance income/(expense)—net

 

 

 

(4,285

)

(3,206

)

(3,474

)

Result before corporate income tax

 

 

 

(26,820

)

(37,040

)

(71,911

)

Corporate income taxes

 

23

 

 

 

431

 

Net loss

 

 

 

(26,820

)

(37,040

)

(71,480

)

Items that may be subsequently reclassified to profit or loss

 

 

 

 

 

 

 

 

 

Currency translation differences on foreign operations

 

 

 

12

 

1,149

 

1,250

 

Other comprehensive income/(loss)

 

24

 

12

 

1,149

 

1,250

 

Total comprehensive loss

 

 

 

(26,808

)

(35,891

)

(70,230

)

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

 

 

 

 

 

 

 

 

 

Basic and diluted loss per share

 

25

 

(2.48

)

(2.16

)

(3.24

)

 

6



 

uniQure N.V.

 

Consolidated Statements of Changes in (Deficit)/Equity
(€ in thousands)

 

 

 

 

 

Total

 

 

 

 

 

 

 

 

 

 

 

 

 

Share

 

Share

 

Other

 

Accumulated

 

Total

 

 

 

Note

 

Capital

 

Premium

 

Reserves

 

Deficit

 

Equity/Deficit

 

Balance at January 1, 2013

 

 

 

483

 

114,795

 

1,508

 

(117,234

)

(448

)

Result for the period

 

 

 

 

 

 

(26,820

)

(26,820

)

Other comprehensive income/(loss)

 

12

 

 

 

 

12

 

12

 

Total comprehensive loss

 

 

 

 

 

 

(26,808

)

(26,808

)

Capital contributions

 

 

 

127

 

27,664

 

 

 

27,791

 

Result on conversion of loan

 

 

 

 

 

3,005

 

 

3,005

 

Share based payment/expense

 

13

 

 

 

2,023

 

 

2,023

 

Balance at December 31, 2013

 

 

 

610

 

142,459

 

6,536

 

(144,041

)

5,564

 

Result for the period

 

 

 

 

 

 

(37,040

)

(37,040

)

Other comprehensive income

 

 

 

 

 

1,149

 

 

1,149

 

Total comprehensive loss

 

 

 

 

 

1,149

 

(37,040

)

(35,891

)

Capital contributions

 

12

 

295

 

64,320

 

 

 

64,615

 

Share issuance costs

 

 

 

 

(668

)

 

 

(668

)

Share based payment/expense

 

13

 

 

 

9,464

 

 

9,464

 

Balance at December 31, 2014

 

12

 

905

 

206,111

 

17,149

 

(181,081

)

43,084

 

Result for the period

 

 

 

 

 

 

(71,480

)

(71,480

)

Other comprehensive income

 

24

 

 

 

1,250

 

 

1,250

 

Total comprehensive loss

 

 

 

 

 

1,250

 

(71,480

)

(70,230

)

Capital contributions

 

12

 

311

 

139,304

 

 

 

139,615

 

Share issuance costs

 

 

 

 

(612

)

 

 

(612

)

Share based payment/expense

 

13

 

 

 

7,627

 

 

7,627

 

Balance at December 31, 2015

 

12

 

1,216

 

344,803

 

26,026

 

(252,561

)

119,484

 

 

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uniQure N.V.

 

Consolidated Statements of Cash Flows
(€ in thousands)

 

 

 

 

 

YEARS ENDED DECEMBER 31,

 

 

 

NOTE

 

2013

 

2014

 

2015

 

Cash flow from operating activities

 

 

 

 

 

 

 

 

 

Net loss

 

 

 

(26,820

)

(37,040

)

(71,480

)

Adjustments for:

 

 

 

 

 

 

 

 

 

Depreciation

 

7

 

535

 

1,539

 

3,982

 

Amortization on intangibles assets

 

6

 

 

 

460

 

Impairment of intangible assets

 

6

 

 

 

11,640

 

Change in deferred tax liability

 

23

 

 

 

(479

)

Lease incentive

 

27

 

134

 

5,452

 

183

 

Loss/(gain) on derivatives

 

14

 

3,446

 

(87

)

(440

)

Loss/(gain) on foreign exchanges

 

14

 

49

 

(4,692

)

1,332

 

Changes in contingent consideration

 

 

 

 

153

 

1,232

 

Share-based expenses

 

13

 

2,023

 

9,464

 

7,627

 

Changes in other non-current assets

 

 

 

(923

)

 

 

Changes in trade and other receivables

 

 

 

(1,439

)

(952

)

(1,554

)

Movement in inventories

 

 

 

(865

)

664

 

(235

)

Changes in other current liabilities

 

 

 

3,655

 

1,540

 

(1,199

)

Changes in deferred revenue

 

17

 

16,958

 

(242

)

65,361

 

Intial recognition of warrants

 

3

 

 

 

2,622

 

Cash (used in) / generated by operations

 

 

 

(3,247

)

(24,201

)

19,052

 

Interest paid

 

 

 

(889

)

(1,224

)

(1,878

)

Net cash (used in) / generated by operating activities

 

 

 

(4,136

)

(25,425

)

17,174

 

Cash flow from investing activities

 

 

 

 

 

 

 

 

 

Purchases of property, plant and equipment

 

7

 

(1,336

)

(15,769

)

(5,671

)

Purchases of intangible assets

 

6

 

(4,652

)

(3,367

)

(2,940

)

Interest received

 

 

 

17

 

148

 

91

 

Acquisition of businesses

 

5

 

 

(1,463

)

 

Net cash used in investing activities

 

 

 

(5,971

)

(20,451

)

(8,520

)

Cash flow from financing activities

 

 

 

 

 

 

 

 

 

Proceeds from shares issued

 

12

 

14,294

 

63,097

 

138,342

 

Share issuance cost

 

12

 

 

(668

)

(612

)

Convertible loans drawn down

 

 

 

11,999

 

 

 

Proceeds from Borrowings

 

14

 

7,492

 

7,184

 

 

Payments of finance lease

 

16

 

(143

)

(156

)

(168

)

Net cash generated from financing activities

 

 

 

33,642

 

69,457

 

137,562

 

Net increase in cash, cash equivalents and bank overdrafts

 

 

 

23,535

 

23,581

 

146,216

 

Currency effect cash and cash equivalents

 

 

 

12

 

5,828

 

4,097

 

Cash, cash equivalents and bank overdrafts at beginning of the period

 

 

 

263

 

23,810

 

53,219

 

Cash, cash equivalents and bank overdrafts at end of the period

 

 

 

23,810

 

53,219

 

203,532

 

 

8