~ Partner
etranacogene dezaparvovec in hemophilia B; MAA validated by the
and granted accelerated assessment ~
~ Received
of marketing and license applications ~
~ Completed patient enrollment of higher dose cohort of
~ One year safety and biomarker data from all 10 patients in lower dose cohort of
~ Multiple preclinical data presentations to be featured at
(ASGCT)
~ Appointed
of Corlieve as Chief Business Officer ~
“uniQure is off to a strong start in 2022, with important advancements across our key programs and earlier-stage research activities, as well as the strengthening of our executive leadership team,” stated
“We also made significant advancements enrolling patients in our Phase I/II studies for AMT-130 in Huntington’s disease,” he added. “To date, we have completed 32 patient procedures across both studies and look forward to presenting in the second quarter of 2022 one-year safety and biomarker data from the 10-patient lower dose cohort of the
Recent Key Accomplishments
- Advancing the late-stage development of etranacogene dezaparvovec (AMT-061) for the treatment of hemophilia B
- In
March 2022 , the Company’s global commercialization partner,CSL Behring , announced that the MAA for etranacogene dezaparvovec was validated by EMA and granted accelerated assessment. In accordance with the Company’s commercialization and license agreement,CSL Behring is solely responsible for all regulatory activities, including filings and agency interactions, associated with either etranacogene dezaparvovec or any required AAV5 neutralizing antibody companion diagnostic. - In March and
April 2022 , the Company received$55 million in milestone payments related to CSL Behring’s submissions of marketing and license applications for etranacogene dezaparvovec. - In
December 2021 , the Company andCSL Behring announced the achievement of primary and secondary endpoints from the HOPE-B pivotal trial of etranacogene dezaparvovec in severe and moderately severe hemophilia B patients. The primary endpoint of non-inferiority in annualized bleeding rate (ABR) 18-months following administration compared to baseline Factor IX (FIX) prophylactic therapy was achieved, as was a secondary superiority endpoint on ABR.
- In
- Advancing the clinical development of AMT-130 for the treatment of Huntington’s disease
- In
March 2022 , the Company announced the completion of enrollment of all 26 patients in the first two cohorts of its randomized, double-blinded, Phase I/II clinical trial of AMT-130 for the treatment of early-stage Huntington’s disease. The ongoing Phase I/II clinical trial of AMT-130 is a randomized, sham controlled, double-blinded study to explore the safety, tolerability, and proof of concept of AMT-130 in patients with early manifest Huntington’s disease. A third cohort exploring the use of an alternative stereotactic navigation system to simplify placement of infusion catheters in up to 18 additional randomized patients is expected to commence enrollment in the second half of 2022. - Also in
March 2022 , the Company completed enrollment of the lower-dose cohort of six patients in the European Phase Ib/II study of AMT-130. The open-label study will enroll 15 patients with early manifest Huntington’s disease across the same doses being explored in theU.S study. Together with the ongoingU.S. study, the European study is intended to establish safety, proof of concept, and the optimal dose of AMT-130 to take forward into Phase III development, or into an adaptive confirmatory study should an accelerated registration pathway be feasible. In total, 59 patients are expected to be enrolled in theU.S. and European Phase I/II trials of AMT-130.
- In
- In
December 2021 , initial observations were announced on the first four patients in the low-dose cohort of the double-blinded and randomizedU.S. Phase I/II study of AMT-130. The investigational gene therapy was generally well tolerated with no serious adverse events related to treatment. NfL increased as expected immediately following the AMT-130 surgical procedure and returned to baseline in the two treated patients. NfL remained relatively constant in the two untreated control patients. Structural magnetic resonance imaging did not reveal any clinically meaningful safety findings in either treated or control patients at one year of follow-up.- During the second quarter of 2022, the Company expects to provide a clinical update on all 10 patients followed for twelve months in the lower dose cohort, including safety, mHTT and NfL biomarker data. Volumetric MRI and functional data is expected to be available during the first half of 2023, after all patients in the two dose cohorts are unblinded.
- Advancing our research pipeline into the clinic and the Company’s proprietary technologies
- Progress towards 2023 INDs:
- Refractory Temporal Lobe Epilepsy (rTLE) – In
March 2022 , the Company initiated an IND-enabling dose-ranging study of AMT-260 and plans to initiate a GLP toxicity study in non-human primates in the second half of 2022 to support an IND submission in 2023. - Fabry disease – The Company expects to initiate a GLP toxicology study of AMT-191 in non-human primates in the second half of 2022, which is expected to support an IND submission in 2023.
- Refractory Temporal Lobe Epilepsy (rTLE) – In
- The Company today announced its significant scientific presence at the upcoming
American Society of Gene andCell Therapy (ASGCT) Hybrid Congress , to be heldMay 16-19, 2022 inWashington, DC . In total, six data presentations will be delivered by the Company’s researchers and scientists, including five posters, and one oral presentation. These presentations will highlight the Company’s research and technology capabilities and feature some new preclinical findings on the company’s gene therapy candidates forHuntington's disease, refractory temporal lobe epilepsy, Fabry disease, Parkinson’s disease, Amyotrophic lateral sclerosis (ALS) and Alzheimer’s disease.
- Progress towards 2023 INDs:
- Enhancements to the Leadership Team
- In
March 2022 ,Richard Porter was appointed Chief Business Officer, responsible for global business development, corporate strategic planning, market access and commercial planning.Dr. Porter joined the Company following the acquisition of Corlieve Therapeutics inJuly 2021 .Dr. Porter brings over 25 years of R&D experience to the Company, having previously served as the COO of Therachon, until its acquisition by Pfizer. Previously, he was at Roche in positions of increasing responsibility, most recently as the Global Head of Scientific Business Strategy and Operations for Neuroscience Ophthalmology and Rare Diseases.Dr. Porter holds a Ph.D. in neuropharmacology fromSouthampton University (UK ) and conducted his postdoctoral training at theStrong Memorial Hospital , University ofRochester, NY (USA) and theUniversity of Oxford (UK ). - In
April 2022 ,Erin Boyer joined the leadership team asChief People and Culture Officer, responsible for leading the global Human Resources team.Ms. Boyer brings more than 20 years of Human Resources experience, including extensive leadership experience at biotech companies. She joins from Epizyme inCambridge, Mass. , where she served asChief People and Culture Officer since 2020. Prior to Epizyme, she served as VP of Human Resources at Obsidian Therapeutics, and before that spent five years at Alnylam Pharmaceuticals in roles of increasing responsibility, including Global Head of Talent Strategy and Planning.
- In
- Strong cash position to advance the Company’s programs
- As of
March 31, 2022 , the Company had cash and cash equivalents of$524.9 million , which included the collection of$20 million of the$55 million in milestone payments owed byCSL Behring . The remaining$35 million in milestone payments were collected by the Company inApril 2022 . The Company expects that its cash and cash equivalents will fund operations into the first half of 2025 assuming the achievement of$175.0 million of first commercial sales milestones under the CSL Behring Agreement.
- As of
Upcoming Investor Events
RBC Global Healthcare Conference ,May 17-18, 2022 - uniQure Annual General Meeting,
June 14, 2022
Financial Highlights
Cash Position: As of
Revenues: Revenue for the three months ended
R&D Expenses: Research and development expenses were
SG&A Expenses: Selling, general and administrative expenses were
Other Non-operating Items, net:
Other non-operating income, net was income of
Net Loss:
The net loss for the three months ended
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 gene therapies to treat patients with hemophilia B,
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, whether we are able to enroll up to 18 additional patients or otherwise commence enrollment at any time for our third cohort exploring the use of an alternative stereotactic navigation system to simplify placement of infusion catheters, whether we are able to enroll up to 59 patients in the
uniQure Contacts:
FOR INVESTORS: | FOR MEDIA: | |
Direct: 339-970-7536 | Direct: 617-306-9137 | Direct: 339-970-7558 |
Mobile: 617-680-9452 | Mobile: 617-306-9137 | Mobile:339-223-8541 |
m.cantor@uniQure.com | c.russo@uniQure.com | t.malone@uniQure.com |
uniQure N.V.
UNAUDITED CONSOLIDATED BALANCE SHEETS
2022 | 2021 | |||||
(in thousands, except share and per share amounts) | ||||||
Current assets | ||||||
Cash and cash equivalents | $ | 524,886 | $ | 556,256 | ||
Accounts receivable and contract asset | 38,644 | 58,768 | ||||
Prepaid expenses | 12,422 | 10,540 | ||||
Other current assets and receivables | 2,260 | 2,675 | ||||
Total current assets | 578,212 | 628,239 | ||||
Non-current assets | ||||||
Property, plant and equipment, net | 44,918 | 43,505 | ||||
Operating lease right-of-use assets | 24,941 | 25,573 | ||||
Intangible assets, net | 61,135 | 62,686 | ||||
26,565 | 27,633 | |||||
Deferred tax assets, net | 15,442 | 15,647 | ||||
Other non-current assets | 5,937 | 5,897 | ||||
Total non-current assets | 178,938 | 180,941 | ||||
Total assets | $ | 757,150 | $ | 809,180 | ||
Current liabilities | ||||||
Accounts payable | $ | 9,920 | $ | 2,502 | ||
Accrued expenses and other current liabilities | 19,945 | 28,487 | ||||
Current portion of contingent consideration | 9,081 | - | ||||
Current portion of operating lease liabilities | 5,754 | 5,774 | ||||
Total current liabilities | 44,700 | 36,763 | ||||
Non-current liabilities | ||||||
Long-term debt | 101,421 | 100,963 | ||||
Operating lease liabilities, net of current portion | 28,232 | 28,987 | ||||
Contingent consideration, net of current portion | 22,059 | 29,542 | ||||
Deferred tax liability, net | 11,298 | 12,913 | ||||
Other non-current liabilities | 3,439 | 4,236 | ||||
Total non-current liabilities | 166,449 | 176,641 | ||||
Total liabilities | 211,149 | 213,404 | ||||
Shareholders' equity | ||||||
Total shareholders' equity | 546,001 | 595,776 | ||||
Total liabilities and shareholders' equity | $ | 757,150 | $ | 809,180 | ||
uniQure N.V.
UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS
Three months ended | |||||||
2022 | 2021 | ||||||
(in thousands, except share and per share amounts) | |||||||
Total revenues | $ | 1,792 | $ | 454 | |||
Operating expenses: | |||||||
Research and development expenses | (45,003 | ) | (32,656 | ) | |||
Selling, general and administrative expenses | (10,987 | ) | (12,375 | ) | |||
Total operating expenses | (55,990 | ) | (45,031 | ) | |||
Other income | 311 | 352 | |||||
Other expense | (193 | ) | (233 | ) | |||
Loss from operations | (54,080 | ) | (44,458 | ) | |||
Non-operating items, net | 6,786 | 3,115 | |||||
Loss before income tax benefit / (expense) | $ | (47,294 | ) | $ | (41,343 | ) | |
Income tax benefit / (expense) | 616 | (213 | ) | ||||
Net loss | $ | (46,678 | ) | $ | (41,556 | ) | |
Basic and diluted net loss per ordinary share | $ | (1.00 | ) | $ | (0.91 | ) | |
Weighted average shares used in computing basic and diluted net loss per ordinary share | 46,599,114 | 45,468,485 | |||||
Source: uniQure Inc.
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