AMSTERDAM, the Netherlands, Nov. 30, 2015 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leader in human gene therapy, today announced financial results for the third quarter and nine months ending September 30, 2015, and provided an update on its development programs.
"In the last quarter uniQure and our collaborators, a consortium consisting of Institut Pasteur, INSERM, the French Muscular Dystrophy Association and Vaincre les Maladies Lysosomales, have clinically demonstrated proof-of-concept for safely treating a severe lysosomal storage disease, Sanfilippo B, with a gene therapy construct developed by uniQure. The study has shown encouraging signals of clinical benefit. By the second week of January 2016 we hope to announce preliminary top-line results in our Hemophilia B trial," said Jörn Aldag, Chief Executive Officer of uniQure. "To accelerate our pipeline development we have added two therapeutic area leaders, Charles Richard, Senior Vice President, Neuroscience and Deya Corzo, Senior Vice President, Liver/Metabolism, joining Patrick Most who leads our heart failure program partnered with BMS. Deya and Charlie will lead the preparation for two pivotal studies in 2016."
Pipeline and Products
Hemophilia B Program Update
- The first cohort of five hemophilia B patients has been dosed in the Phase 1/2 study of AMT-060.
- In early January 2016, the Company plans to release preliminary, top-line data for the two patients who will have completed at least 12 weeks of treatment follow-up. The data readout will include safety results and FIX expression levels. No additional data from the remaining cohort patients or other measures of the trial will be reported at that time.
- The Company expects to report on the full results of the first cohort at a scientific conference during 1H 2016.
Sanfilippo B Program Update
- Positive one-year follow-up results from four Sanfilippo B patients participating in a Phase 1/2 clinical trial were presented by Prof. Marc Tardieu representing Institut Pasteur, uniQure's collaborator, at the ESGCT and FSGT Collaborative Congress on September 19, 2015. The trial demonstrated durable increases in NAGLU expression in the cerebrospinal fluid (CSF) of 14% to 17% at 12-months post treatment. It also showed that incremental cognitive development was maintained in all four patients with no evidence of progressive brain atrophy. The trial provided the first clinical validation of the Company's proprietary AAV5 vector effectively delivering a target gene into the CNS leading to durable expression of NAGLU protein in the CNS.
- Subsequently, Prof. Tardieu presented a post-hoc biochemical analysis of glycosaminoglycans (GAG) levels in the CSF using samples from the same four patients. The results were presented at the International Conference on Sanfilippo Syndrome and Related Lysosomal Storage Diseases in Geneva on November 27, 2015. A decrease of Heparan sulfate, a brain-predominant GAG and a substrate for the NaGlu enzyme, could not be detected in the CSF. This result resembles a previously published study by Ausseil and colleagues using uniQure's AAV5 NAGLU gene therapy vector in a canine model of Sanfilippo B, where durable NAGLU expression in the dog brain resulted in significant reductions of brain tissue GAGs, although CSF GAGs were unchanged (previously unpublished). Given the encouraging positive clinical results previously reported, and the difficulty in interpreting CSF GAGs as a biomarker predictive of clinical benefit, uniQure is aggressively moving forward with its plans to complete the in-license of the data from the Company's collaborator, prepare the future pathway with regulatory authorities and complete close follow-up of the patients through the end of the 30-month study, expected around year-end 2016.
Glybera Program Update
- On October 28, 2015, the Charité University Clinic in Berlin, Germany, announced the treatment of the first patient with Glybera as a commercially-available gene therapy, enabled by uniQure's commercialization partner in the EU, Chiesi Farmaceutici.
- During the three month period ended September 30, 2015, the Company recorded a one-time, non-cash impairment charge of €11.6 million associated with certain intangible assets related to Glybera. The impairment was primarily due to a reduction in the estimated number of patients to be treated with Glybera.
- uniQure has decided not to pursue U.S. regulatory approval of Glybera in order to maintain the Company's focus on its three core therapeutic areas. The Company will continue to meet all of its obligations to its partner Chiesi and all EMA obligations including the execution of a post-approval phase IV clinical trial, and the continuation of the patient registry (GENIALL) to assess the long-term clinical benefit of Glybera.
Strategic Collaboration with Bristol-Myers Squibb
- On August 10, 2015, uniQure announced the receipt of an additional $53 million from Bristol-Myers Squibb in accordance with the companies' collaboration agreement. Included in the total was a $15 million target designation fee triggered by Bristol-Myers Squibb's selection of three new collaboration targets, in addition to S100A1 for congestive heart failure. Bristol-Myers Squibb also acquired an additional 1.3 million ordinary shares of uniQure priced at $29.67 per share, providing aggregate net proceeds to the Company of approximately $38 million. The purchase price represented an approximately 26% premium over uniQure's closing price per ordinary share on August 7, 2015. After this second equity closing, Bristol-Myers Squibb owns 9.9% of uniQure's outstanding ordinary shares. To date, uniQure has received a total of $140 million from Bristol-Myers Squibb in the context of the agreement.
Other Corporate Developments
- Infrastructure: uniQure's 50,000 sq. ft., fully scalable gene therapy manufacturing plant in Lexington, Massachusetts is now operational and research batches are being produced.
- Human Resources: In July, uniQure announced the appointment of Charles W. Richard, M.D., Ph.D., to the position of Senior Vice President, Research and Development, Neuroscience, to lead the Company's growing portfolio of gene therapies targeting neurological diseases, including current clinical trials for the treatment of Sanfilippo B syndrome and Parkinson's disease as well as preclinical programs in Huntington's disease and other rare CNS disorders.
- Also in July, Deya Corzo, M.D., who joined the Company in Spring 2014 as Vice President, Medical Affairs, was promoted to Senior Vice President, Therapeutic Area Head, Liver/Metabolism. She leads the efforts for the hemophilia B clinical trial program as well as research and development for hemophilia A.
- Hans Preusting, Chief Business Officer, has decided to pursue new entrepreneurial opportunities. He will remain available as a consultant following his departure in January 2016.
"Hans has been an outstanding operations and business development executive for us over the last 9 years and I have greatly enjoyed working with him as we crafted both large scale strategic partnerships and innovative agreements with our scientific collaborators. All of us at uniQure wish him the very best and thank him for all his contributions to the growth of the company," added Mr. Aldag.
As of September 30, 2015, the Company held cash and cash equivalents of €214.5 million, compared with €53.2 million as of December 31, 2014. The increase was due to the consideration received from BMS during the period, offset in part by cash used in research, development and general corporate activities. Revenue for the three months ended September 30, 2015 was €3.2 million, compared with €1.0 million for the comparable period in 2014. For the nine months ended September 30, 2015, licensing and collaboration revenues were €5.9 million compared with €3.2 million for the same period of 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.
Research and development expenses were €11.9 million for the three months ended September 30, 2015, compared to €9.5 million for the comparable period in 2014. For the nine months ended September 30, 2015, research and development costs were €32.7 million compared with €23.7 million for the same period of 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 €4.8 million for the three months ended September 30, 2015, compared with €3.2 million for the comparable period in 2014. Selling, general and administrative costs for the nine months ended September 30, 2015 were €13.5 million, compared with €8.0 million for the comparable period 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 €1.6 million for the three months ended September 30, 2015, compared to a gain of €3.6 million for the comparable period in 2014. For the nine months ended September 30, 2015, other gains/losses were a loss of €2.5 million compared with a gain of €3.7 million in the same period of 2014. The loss was primarily attributable to the impact of foreign currency exchange rates on the Company's dollar-denominated deposits and the periodic revaluation of outstanding warrants.
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 third quarter of 2015 was €25.8 million, or €1.08 per share, compared with €9.1 million, or €0.51 per share, for the third quarter of 2014. The net loss for the nine months ended September 30, 2015 and 2014 were €57.4 million, or €2.69 per share and €25.9 million, or €1.54 per share, respectively. Excluding the one-time impairment charge incurred in the third quarter of 2015, the net loss for the third quarter of 2015 was €14.2 million, or €0.67 per share, and the net loss for the nine months ended September 30, 2015 was €45.7 million, or €2.14 per share.
For further financial information for the period ending September 30, 2015, please refer to the financial statements appearing at the end of this release.
uniQure will host a webcast and conference call today at 8:30 am ET/14:30 CET to report on current developments. To access the conference call information and the webcast, log on to the "Events" section on the "Media" page of uniQure's website, at http://www.uniqure.com/news/calendar-of-events. A replay of the webcast will be available on uniQure's website for at least 72 days following the event.
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 Statement
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, statements regarding the future development of our programs in Sanfilippo B, hemophilia B and cardiovascular diseases, the success of our collaboration with Bristol-Myers Squibb, 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 7, 2015. 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.
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|Unaudited Condensed Consolidated Balance Sheets|
|(€ in thousands)|
|DECEMBER 31,||SEPTEMBER, 30|
|Intangible assets other than Goodwill||8,9||16,368||7,362|
|Property, plant and equipment||7||19,667||22,627|
|Other non-current assets||10||1,022||1,108|
|Total non-current assets||38,399||32,439|
|Receivables from related parties||11||2,426||5,142|
|Trade and other receivables||11||1,542||2,166|
|Cash and cash equivalents||13||53,219||214,494|
|Total current assets||57,387||222,213|
|Derivative financial instruments - related parties||16||--||740|
|Financial lease liabilities||16,26||134||--|
|Deferred tax liabilities||9||1,379||1,349|
|Total non-current liabilities||40,430||100,217|
|Trade and other payables||15||9,617||12,090|
|Derivative financial instruments- related parties||16||645||1,268|
|Borrowings - derivative||16||207||357|
|Total current liabilities||12,272||23,980|
|Total equity and liabilities||95,786||254,652|
|Unaudited Condensed Consolidated Statements of Comprehensive Loss|
| (€ in thousands, except share and per share data) |
|THREE MONTHS ENDED||NINE MONTHS ENDED|
|SEPTEMBER 30,||SEPTEMBER 30,|
|(€ in thousands)|
|Cost of goods sold||--||(428)||--||(428)|
|Research and development expenses||18||(9,514)||(11,933)||(23,740)||(32,652)|
|Selling, general and administrative expenses||19||(3,218)||(4,835)||(8,035)||(13,503)|
|Impairment of intangible assets||8||--||(11,640)||--||(11,640)|
|Other gains / losses, net||20||3,630||(1,552)||3,694||(2,548)|
|Total operating costs||(8,894)||(30,202)||(27,483)||(60,239)|
|Result before corporate income tax||(9,059)||(25,874)||(25,879)||(57,399)|
|Corporate income taxes||--||30||--||30|
|Items that may be subsequently reclassified to profit or loss|
|Currency translation differences on foreign operations||703||(159)||693||927|
|Other comprehensive income/(loss)||21||703||(159)||693||927|
|Total comprehensive loss||(8,356)||(26,003)||(25,186)||(56,442)|
|Loss per share attributable to the equity holders of the Company during the year:|
|Basic and diluted loss per share||24||(0.51)||(1.08)||(1.54)||(2.69)|
|Unaudited Condensed Consolidated Statement of Changes in Equity/Deficit|
|(€ in thousands)|
|Balance at January 1, 2014||610||142,459||6,536||(144,041)||5,564|
|Result for the period||--||--||--||(25,868)||(25,868)|
|Other comprehensive income/(loss)||--||--||693||(10)||683|
|Total comprehensive loss||--||--||693||(25,878)||(25,185)|
|Share issuance costs||--||(668)||--||--||(668)|
|Share based payment/expense||--||--||7,193||--||7,193|
|Balance at September 30, 2014||892||205,811||14,422||(169,920)||51,205|
|Result for the period||--||--||--||(11,170)||(11,170)|
|Other comprehensive income||--||--||456||9||465|
|Total comprehensive loss||--||--||456||(11,161)||(10,705)|
|Share based payment/expense||--||--||2,271||--||2,271|
|Balance at December 31, 2014||14||905||206,111||17,149||(181,081)||43,084|
|Result for the period||--||--||--||(57,369)||(57,369)|
|Other comprehensive income||--||--||927||--||927|
|Total comprehensive loss||--||--||927||(57,369)||(56,442)|
|Share issuance costs||--||(612)||--||--||(612)|
|Share based payment/expense||--||--||5,242||--||5,242|
|Balance at September 30, 2015||14||1,211||344,376||23,318||(238,450)||130,455|
|Unaudited Condensed Consolidated Statement of Cash Flows|
|(€ in thousands)|
|NINE MONTHS ENDED|
|Cash flow from operating activities|
|Amortization on intangibles assets||7||--||274|
|Impairment of intangible assets||8||--||11,640|
|Loss/(gain) on derivatives||16||(574)||164|
|Loss/(gain) on foreign exchanges||(2,545)||3,160|
|Other non-cash items||(1)||606|
|Changes in trade and other receivables||(142)||(3,339)|
|Movement in inventories||12||326||(210)|
|Changes in trade and other payables||15||(1,812)||(5,234)|
|Changes in deferred revenue and provisions||(635)||65,407|
|Initial recognition of warrants||3||--||2,622|
|Movement in other liabilities||1,209||2,435|
|Interest (income) / expense||1,035||1,504|
|Cash (used in) / generated by operations||(16,112)||29,903|
|Net cash (used in) / generated by operating activities||(16,919)||28,499|
|Cash flow from investing activities|
|Purchases of property, plant and equipment||7||(13,365)||(3,289)|
|Purchases of intangible assets||8||(2,129)||(2,908)|
|Acquisition of businesses||(1,463)||--|
|Net cash used in investing activities||(16,862)||(6,127)|
|Cash flow from financing activities|
|Proceeds from shares issued||14||62,786||137,909|
|Share issuance cost||14||(668)||(612)|
|Proceeds from Borrowings||16||7,184||--|
|Payments of finance lease||16||(116)||(125)|
|Net cash generated from financing activities||69,186||137,172|
|Net increase in cash, cash equivalents and bank overdrafts||35,405||159,544|
|Currency effect cash and cash equivalents||3,567||1,731|
|Cash, cash equivalents and bank overdrafts at beginning of the period||23,810||53,219|
|Cash, cash equivalents and bank overdrafts at end of the period||62,782||214,494|