qureUniqure NV (NASDAQ:QURE), a leader in human gene therapy, announced financial results for the first quarter ending March 31, 2016, and provided a corporate update.

“We are very pleased with our progress so far in 2016 as we continue to advance our clinical and preclinical programs and build out our gene therapy platform,” commented Daniel Soland, Chief Executive Officer of uniQure. “We achieved two important milestones related to our hemophilia B gene therapy AMT-060: announcing our first clinical data from the study’s low-dose cohort and completing the dosing of all 10 patients in the study, including the high-dose cohort. Additionally, we presented and published data from several preclinical studies, including proof-of-concept data for AMT-130 in Huntington’s disease and several new technologies designed to optimize gene therapy and expand its applicability to patients across the world. We look forward to presenting additional data on AMT-060 at EHA in June and remain on track to present new data from our high-dose cohort before the end of the year.”

Corporate Highlights

Pipeline and Technology Updates

Liver/Metabolic Diseases

  • Announced Upcoming Oral Presentation of Clinical Data from AMT-060 Low-Dose Cohort at EHA – On May 19, 2016 the Company announced that additional data from its Phase I/II trial of AMT-060 in hemophilia B will be presented on June 11, 2016 at the 21st Congress of the European Hematology Association (“EHA”) in Copenhagen, Denmark. The oral presentation by the clinical investigator will include new and updated data on five patients enrolled in the study’s low-dose cohort. uniQure will also host a webcast conference call to discuss the results at 02.00 pm CET/ 8:00 am EDT on Monday, June 13, 2016.
  • Completed Dosing of AMT-060 Phase I/II Clinical Trial – The Company announced today that it has successfully treated all five patients in its high-dose cohort, thus completing the enrollment of its 10 patient, Phase I/II study of AMT-060.  AAV5 continues to exhibit a very low screening failure rate, with all patients screened in the study testing negative for pre-existing, anti-AAV5 antibodies. The Company anticipates presenting data on the high-dose cohort before the end of the year.
  • Published Phase I Clinical Data from AAV5-Based Gene Therapy in AIP – On May 17, 2016, results from the AIPGENE Consortium-led Phase I clinical trial using uniQure’s AAV5-based gene therapy to treat acute intermittent porphyria (AIP) were published online in the Journal of Hepatology. In the trial, a total of eight patients in four dose cohorts received a single intravenous injection of uniQure’s AAV5 vector-PBGD transgene gene therapy in doses ranging from 2×1012gc/kg to 1.8×1013 gc/kg, without the use of prophylactic steroid therapy. The therapy was well-tolerated in all patients, with no cellular immune responses to either the vector or the transgene, and no transaminase elevations or serious adverse events related to the gene therapy. Including these patients, a total of 27 patients have been screened for pre-existing anti-AAV5 antibodies in three clinical programs to date, with only one AIP patient testing marginally positive for pre-existing antibodies. While the Company currently does not have plans to conduct further studies for AIP, these data support the broad safety and applicability of AAV5 as a differentiated vector serotype due to the low prevalence of pre-existing anti-AAV5 antibodies.

CNS Diseases

  • Initiated Extension Protocol for Phase I/II Study of AMT-110 in Sanfilippo B – On April 4, 2016, the Company announced that it has assumed the sponsorship of the Phase I/II extension study from 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).  The Company has initiated the 30-month follow-up of the four patients treated in the study and anticipates presenting additional data in early 2017.

Technology Programs

  • Presented Data from Multiple Preclinical Studies of New Gene Therapy Technologies at ASGCT – On May 9, 2016, the Company announced data from several preclinical research programs focused on the development and validation of optimized gene therapy delivery systems, AAV re-administration, regulated gene expression and total brain transduction. The results were presented in poster sessions at the American Society of Gene and Cell Therapy (ASGCT) 19th Annual Meeting in Washington D.C. and highlight the progress uniQure has made in developing new technologies designed to improve gene therapy and broaden its applicability to patients.

Human Resource and Organizational Updates

  • Added Key Talent – On May 3, 2016, the Company announced the appointment of Paul Firuta as Chief Commercial Officer.  Mr. Firuta, who held similar positions at NPS Pharmaceuticals and ViroPharma, will oversee global commercial and portfolio planning and program management, as well as will work closely with the Company’s cross-functional teams in building relationships with patient advocacy groups and key opinion leaders.
  • Entered Lease for New Amsterdam Facility – On March 1, 2016, the Company entered into a lease contract for a new 9,200 square meter facility in Amsterdam designed to meet the needs of its fully-integrated gene therapy operations and expanding product pipeline.  The Company has commenced the refurbishment of the space and intends to initiate the consolidation of its current three Amsterdam sites into the new facility by the end of the year.
  • Proposed Corporate Governance Changes – On May 9, 2016, the Company announced the nomination of Jack Kaye to the Board of Directors.  Mr. Kaye, a seasoned financial executive with over 40 years of diversified experience, will also chair the Company’s Audit Committee.  Additionally, the Company announced its intention to transition from the current two-tier Supervisory Board and Management Board structure to a single Board of Directors with executive and non-executive members. As part of the governance transition, Ferdinand Verdonck and Joseph Fezcko plan to retire from the Supervisory Board and Mr. Soland and Matthew Kapusta, Chief Financial Officer, will stand for election to the single Board as executive directors.

Corporate Finance and Other Updates

  • Lowered Cost and Expanded Flexibility of Venture Debt Agreement – On May 6, 2016, the Company executed a second amended and restated loan agreement with Hercules Technology Growth Capital, Inc. (Hercules).  The loan agreement includes a total commitment from Hercules of up to $40 million, of which $20 million is currently outstanding.  The Company has not drawn down any additional loan amounts.  The interest rate will be reduced from 10.25% to 8.25% per annum with a backend fee of 4.85% and facility fee of 0.75% of the outstanding loan amounts.  The interest-only payment period is initially set at 18 months from May 6, 2016, but can be extended to 36 months under certain conditions.  The maturity date of the loan facility was also extended from June 30, 2017 to March 1, 2020.
  • Established Stock Purchase Plan for Chief Executive Officer – On April 18, 2016, Mr. Soland established a pre-arranged personal stock purchase plan to acquire up to 100,000 ordinary shares of the Company’s stock. The plan is intended to comply with Rule 10b5-1 of the Securities Exchange Act of 1934, as amended. The transactions under this purchasing plan will commence no earlier than June 13, 2016.

Financial Highlights

As of March 31, 2016, the Company held cash and cash equivalents of €184.6 million, compared with of €203.5 million, as of December 31, 2015, a decrease of €18.9 million. The decrease includes €16.0 million of cash used in operating, investing and financing activities during the three months ended March 31, 2016, in addition to a €2.9 million loss related to foreign currency effects on U.S. dollar-denominated deposits.

Licensing and collaboration revenues for the three months ended March 31, 2016 were €3.9 million, compared with €1.1 million for the comparable period in 2015. The change was primarily due to the amortization of upfront payments and target designation fees received from BMS in the second and third quarters of 2015, and the increase of research activities associated with S100A1 for heart failure, which are fully reimbursed by BMS in accordance with our collaboration agreement.

Research and development expenses were €15.1 million for the three months ended March 31, 2016, compared with €10.1 million for the comparable period in 2015. The increase is mainly due to the initiation of our Phase I/II clinical study of AMT-060 in hemophilia B, an increase in activities in support of our collaboration agreement with BMS, which closed in May 2015, and the continued progression of our various research programs, including our collaboration with 4D and preclinical product candidates for hemophilia A and Huntington’s disease.

Selling, general and administrative expenses were €6.6 million for the three months ended March 31, 2016, compared with €4.2 million for the comparable period in 2015.  The change primarily related to increases in the numbers of employees, contractors and consultants, as well as increases in professional fees and expenses associated with the Glybera global registry and Phase IV study, which are currently classified as selling, general and administrative expenses, but were previously capitalized in the same period in 2015, prior to the commencement of Glybera commercialization.

The net loss for the first quarter 2016 was €20.6 million, or €0.83 per share, compared with €12.6 million, or €0.69 per share, for the first three months of 2015. (Original Source)

Shares of uniQure N.V. closed last Friday at $11.99, down $0.28 or -2.28%. QURE has a 1-year high of $36.38 and a 1-year low of $10.61. The stock’s 50-day moving average is $12.83 and its 200-day moving average is $15.00.

On the ratings front, uniQure has been the subject of a number of recent research reports. In a report issued on May 11, Chardan analyst Gbola Amusa MD CFA reiterated a Buy rating on QURE, with a price target of $35, which represents a potential upside of 191.9% from where the stock is currently trading. Separately, on April 12, Leerink Swann’s Michael Schmidt reiterated a Buy rating on the stock and has a price target of $27.

According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Gbola Amusa MD CFA and Michael Schmidt have a total average return of -7.7% and 5.4% respectively. CFA has a success rate of 40.0% and is ranked #3709 out of 3891 analysts, while Schmidt has a success rate of 59.8% and is ranked #629.

Overall, 8 research analysts have given a Buy rating to the stock. When considering if perhaps the stock is under or overvalued, the average price target is $25.00 which is 108.5% above where the stock closed last Friday.