uniQure N.V. — Q1 2023 Form 10-Q
Reporting period: Three months ended March 31, 2023; unaudited U.S. GAAP consolidated financial statements, reported in U.S. dollars. uniQure is a gene-therapy company. Its HEMGENIX gene therapy for hemophilia B was approved in the U.S. in November 2022 and the EU in February 2023; CSL Behring holds exclusive global rights and is responsible for commercialization.
Financial performance and position
| Metric | Q1 2023 | Q1 2022 / prior date |
|---|---|---|
| Total revenue | $5.3 million | $1.8 million |
| Contract manufacturing revenue | $4.9 million | None |
| Contract manufacturing cost | $2.4 million | None |
| Manufacturing gross profit / margin | $2.5 million / approximately 51% | Not applicable |
| Research and development expense | $60.8 million | $45.0 million |
| Selling, general and administrative expense | $17.8 million | $11.0 million |
| Net loss | $77.2 million | $46.7 million |
| Basic and diluted loss per share | $1.63 | $1.00 |
| Net cash used in operating activities | $78.3 million | $25.7 million |
Revenue increased by $3.5 million, primarily from HEMGENIX manufacturing for CSL Behring; collaboration revenue fell to $0.4 million from $1.8 million. R&D growth included $10.0 million expensed for the Apic Bio license, $3.1 million of HEMGENIX-related licensing costs following EU approval, and increased personnel and facility costs. SG&A rose mainly with staffing, share-based compensation and professional fees. Net loss also reflected a $2.4 million foreign-exchange loss, compared with an $8.6 million gain a year earlier, and higher interest expense; interest income increased to $1.7 million.
At March 31, cash and cash equivalents were $153.9 million and current investment securities were $161.5 million, totaling $315.3 million; including restricted cash, cash and investments totaled approximately $318.5 million. Cash, cash equivalents and restricted cash declined $74.1 million during the quarter. Total assets were $631.0 million, total liabilities $218.2 million, and shareholders’ equity $412.8 million. Accumulated deficit was $659.2 million.
Outstanding Hercules debt principal was $100.0 million, due in full in December 2025; the balance-sheet long-term debt carrying amount was $103.3 million. Future interest and financing fees were estimated at $42.8 million, including $15.4 million payable within 12 months. Fixed lease commitments were $58.8 million, including $8.1 million due within 12 months. The company reported compliance with debt covenants.
Material changes and business developments
- CSL Behring manufacturing generated $4.9 million in Q1 revenue for the first time in the comparable quarter. CSL Behring also owes a $100 million U.S. first-commercial-sale milestone, recorded as a contract asset and described as probable; $100.0 million of the company’s $104.8 million accounts receivable and contract asset balance related to this milestone.
- uniQure licensed APB-102/AMT-162, a SOD1-ALS gene-therapy candidate, from Apic Bio in January 2023. The $10 million initial payment was expensed; up to $43 million in additional milestones may be payable. The filing says the FDA had cleared the IND and granted Orphan Drug and Fast Track designations.
- The Bristol-Myers Squibb collaboration, research and license agreements were terminated on February 21, 2023.
- Shares issued and outstanding increased to 47.55 million at March 31, 2023, from 46.97 million at December 31, 2022.
Outlook, risks and contingencies
Management expects expenses to increase as it advances AMT-130 for Huntington’s disease toward Phase III, progresses programs for temporal lobe epilepsy, SOD1-ALS and Fabry disease, and invests in research and manufacturing. Management estimated existing cash resources would fund operations into 2025 assuming receipt of the $100 million U.S. first-sale milestone, and into the first half of 2025 if the $75 million European first-sale milestone is achieved in one of five specified countries by July 2, 2023. These are conditional estimates, not guaranteed funding outcomes. The filing warns that additional financing may be required and may be unavailable or dilutive or require giving up valuable rights.
- Corlieve acquisition consideration is contingent on milestones. The maximum nominal commitments disclosed were $43.5 million through Phase I/II and $174.0 million tied to Phase III and U.S./EU approvals of AMT-260. Fair value at quarter-end was $36.9 million, of which $27.3 million was current; potential payments were expected between 2023 and 2031. Timing and achievement are uncertain.
- Key risks include clinical-trial delays or failure, safety and efficacy uncertainty, manufacturing and regulatory compliance, reliance on collaborators and other third parties, intellectual-property disputes, competition, pricing and reimbursement, and the need for additional capital. The filing notes a prior voluntary postponement and safety investigation related to suspected serious adverse reactions in the AMT-130 Huntington’s program; it also cautions that early clinical results may not predict later outcomes.
- CSL Behring may not achieve the European first-sale milestone by the contractual deadline. Future revenue also depends on manufacturing supply, milestone receipts and royalties from CSL Behring.
- No legal proceedings or subsequent events were reported. Management concluded disclosure controls were effective as of March 31, 2023; no material change in internal control over financial reporting was reported.
Important facts for investors to verify
- Whether and when the $100 million U.S. milestone and $75 million European milestone are received, and the actual HEMGENIX supply and royalty contribution.
- AMT-130 clinical safety and efficacy updates and the timing, design and funding of any Phase III study.
- Quarterly operating cash burn and whether management’s conditional runway assumptions remain achievable.
- AMT-260 development progress and the probability, timing and potential cash or share settlement of Corlieve milestones.
- Ability to scale and maintain compliant manufacturing, and the availability and terms of any additional financing.