QUREUniqure NV (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.

Corporate Updates

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.

Financial Highlights

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. (Original Source)

Shares of uniQure N.V. closed last Friday at $22.50, up $0.93 or 4.31%. QURE has a 1-year high of $36.38 and a 1-year low of $12.28. The stock’s 50-day moving average is $18.88 and its 200-day moving average is $24.94.

On the ratings front, uniQure has been the subject of a number of recent research reports. In a report issued on October 19, Piper Jaffray analyst Joshua Schimmer reiterated a Buy rating on QURE, with a price target of $40, which implies an upside of 77.8% from current levels. Separately, on October 13, Roth Capital’s Debjit Chattopadhyay maintained a Buy rating on the stock and has a price target of $38.

According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Joshua Schimmer and Debjit Chattopadhyay have a total average return of 2.3% and 19.2% respectively. Schimmer has a success rate of 51.2% and is ranked #945 out of 3649 analysts, while Chattopadhyay has a success rate of 57.5% and is ranked #41.

The street is mostly Bullish on QURE stock. Out of 6 analysts who cover the stock, 6 suggest a Buy rating . The 12-month average price target assigned to the stock is $43.20, which implies an upside of 92.0% from current levels.

uniQure NV is engaged in the field of gene therapy and has developed a gene therapy product to receive regulatory approval in the European Union.