ProQR Therapeutics N.V 6-K, filed May 8, 2025
ProQR Therapeutics N.V. (PRQR)
About this 6-K
ProQR Therapeutics N.V filed this 6-K on May 8, 2025.
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 6-K
Report of Foreign Private Issuer
Pursuant to Rule 13a-16 or 15d-16 of
the Securities Exchange Act of 1934
May 8, 2025
Commission File Number: 001-36622
PROQR THERAPEUTICS N.V.
Zernikedreef 9
2333 CK Leiden
The Netherlands
Tel: +31 88 166 7000
(Address, Including ZIP Code, and Telephone Number,
Including Area Code, of Registrant’s Principal Executive Offices)
Indicate by check mark whether the registrant files or will file annual reports under cover of Form 20-F or Form 40-F.
Form 20-F ⌧ Form 40-F ◻
Indicate by check mark if the registrant is submitting the Form 6-K in paper as permitted by Regulation S-T Rule 101(b)(1): ◻
Indicate by check mark if the registrant is submitting the Form 6-K in paper as permitted by Regulation S-T Rule 101(b)(7): ◻
Furnished as Exhibit 99.1 to this Report on Form 6-K are the unaudited financial statements of ProQR Therapeutics N.V. (the “Company”) for the three-month period ended March 31, 2025, and furnished as Exhibit 99.2 to this Report on Form 6-K is a press release of ProQR Therapeutics N.V. dated May 8, 2025, announcing the Company’s results for the three-month period ended March 31, 2025.
On May 8, 2025, the Company issued a press release titled, “ProQR Announces First Quarter 2025 Operating and Financial Results,” announcing the Company’s results for the three-month period ended March 31, 2025 and providing a business update. A copy of this press release is attached hereto as Exhibit 99.2 and is incorporated herein by reference.
ProQR hereby incorporates by reference the information contained herein into ProQR’s registration statements on Form F-3 (File No. 333-282419, File No. 333-270943, File No. 333-263166 and File No. 333-285767).
INDEX TO EXHIBITS
Number |
| Description |
| |
|
99.1 | | |
| | |
99.2 | |
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized.
| PROQR THERAPEUTICS N.V. | |
| | |
| | |
Date: May 8, 2025 | By: | /s/ Jurriaan Dekkers |
| | Jurriaan Dekkers |
| | Chief Financial Officer |
PROQR THERAPEUTICS N.V.
Index to Unaudited Condensed Consolidated Financial Statements
| |
| PAGE |
1 | |
2 | |
3 | |
4 | |
Notes to Unaudited Condensed Consolidated Financial Statements | 5 |
PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Financial Position
| | | | March 31, | | December 31, |
| | | | 2025 | | 2024 |
| | | | €1,000 | | €1,000 |
Assets | | | |
| |
|
Property, plant and equipment | | 7 | | | ||
Investments in financial assets | | 17 | | — | | — |
Non-current assets | | | | | ||
| | | | | | |
Cash and cash equivalents | | 5 | | | ||
Prepayments and other receivables | | 6 | | | ||
Other taxes | | | | | ||
Current assets | | | | | ||
Total assets | | | | | ||
| | | | | | |
Equity and liabilities | | | |
| |
|
Equity | | | | | |
|
Equity attributable to owners of the Company | | | | | ||
Total equity | | 12 | | | ||
| | | | | | |
Liabilities | | | |
| |
|
Borrowings | | 9 | | — | | — |
Lease liabilities | | 10 | | | ||
Deferred income | | 11 | | | ||
Non-current liabilities | | | | | ||
| | | | | | |
Borrowings | | 9 | | | ||
Lease liabilities | | 10 | | | ||
Derivative financial instruments | | 9 | | | ||
Trade payables | | | | | ||
Social securities and other taxes | | | | | ||
Deferred income | | 11 | | | ||
Other current liabilities | | 8 | | | ||
Current liabilities | | | | | ||
Total liabilities | | | | | ||
| | | | | | |
Total equity and liabilities | | | | |
The accompanying notes are an integral part of these unaudited interim condensed consolidated financial statements.
PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Profit or Loss and Other Comprehensive Income
(€ in thousands, except share and per share data)
| | | | Three month period | ||
| | | | ended March 31, | ||
|
| | | 2025 | | 2024 |
| | | | €1,000 | | €1,000 |
Revenue | | 13 | | | ||
| | | | | | |
Other income | | 14 | | | ||
| | | | | | |
Research and development costs | | 15 | | ( | | ( |
General and administrative costs | | 16 | | ( | | ( |
Total operating costs | | | | ( | | ( |
| | | |
| |
|
Operating result | | | | ( | | ( |
Finance income and expense | | | | | ||
Results related to financial liabilities measured at fair value through profit or loss | | 9 | | | ( | |
| | | |
| |
|
Result before corporate income taxes | | | | ( | | ( |
Income taxes | | 18 | | — | | ( |
| | | |
| |
|
Result for the period | | | | ( | | ( |
Other comprehensive income (foreign exchange differences on foreign operation) | | | | ( | | |
| | | |
| |
|
Total comprehensive income | | | | ( | | ( |
| | | | | | |
Result attributable to | | | |
| |
|
Owners of the Company | | | | ( | | ( |
Non-controlling interests | | | | — | | — |
| | | | ( | | ( |
Total comprehensive income attributable to | | | | | | |
Owners of the Company | | | | ( | | ( |
Non-controlling interests | | | | — | | — |
| | | | ( | | ( |
| | | |
| |
|
Share information | | | |
| |
|
Weighted average number of shares outstanding1 | | | | | ||
| | | | | | |
Earnings per share attributable to owners of the Company (Euro per share) | | | | | | |
Basic loss per share1 | | | | ( | | ( |
Diluted loss per share1 | | | | ( | | ( |
The accompanying notes are an integral part of these unaudited interim condensed consolidated financial statements.
| 1. | For these periods the potential exercise of share options is not included in the diluted earnings per share as the Company was loss-making. Due to the anti-dilutive nature of the outstanding options, basic and diluted earnings per share are equal. |
PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Changes in Equity
| | | | | | | | | | | | | | | | | | |
| Attributable to owners of the Company | | | | | |||||||||||||
|
| Number |
| Share |
| Share |
| Equity settled |
| Translation |
| Accumulated |
| Total |
| Non- |
| Total |
|
|
| | €1,000 | | €1,000 | | €1,000 | | €1,000 | | €1,000 | | €1,000 | | €1,000 | | €1,000 |
Balance at January 1, 2024 |
| | | | | | ( | | | — | | |||||||
Result for the period |
| — | | — | | — | | — | | — | | ( | | ( | | — | | ( |
Other comprehensive income |
| — | | — | | — | | — | | | — | | | — | | |||
Recognition of share-based payments |
| — | | — | | — | | | — | | — | | | — | | |||
Treasury shares transferred | | ( | | — | | — | | — | | — | | — | | — | | — | | — |
Share options lapsed | | — | | — | | — | | ( | | — | | | — | | — | | — | |
Share options exercised / RSUs vested | | | — | | | ( | | — | | | | — | | |||||
| | | | | | | | | | | | | | | | | | |
Balance at March 31, 2024 |
| | | | | | ( | | | — | | |||||||
| | | | | | | | | | | | | | | | | | |
Balance at January 1, 2025 |
| | | | | | ( | | | — | | |||||||
Result for the period |
| — | | — | | — | | — | | — | | ( | | ( | | — | | ( |
Other comprehensive income |
| — | | — | | — | | — | | ( | | — | | ( | | — | | ( |
Recognition of share-based payments |
| — | | — | | — | | | — | | — | | | — | | |||
Treasury shares transferred | | ( | | — | | — | | — | | — | | — | | — | | — | | — |
Share options lapsed | | — | | — | | — | | ( | | — | | | — | | — | | — | |
Share options exercised / RSUs vested | | | — | | | ( | | — | | | | — | | |||||
|
|
| |
| |
| |
| |
| |
| |
| |
| |
|
Balance at March 31, 2025 |
| | | | | | ( | | | — | | |||||||
The accompanying notes are an integral part of these unaudited interim condensed consolidated financial statements.
PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Cash Flows
| | | | Three month period | ||
| | | | ended March 31, | ||
|
| | | 2025 | | 2024 |
| | | | €1.000 | | €1.000 |
Cash flows from operating activities | | | |
| |
|
Net result | | | | ( | | ( |
Adjustments for: | | | | | | |
— Other income | | | | ( | | — |
— Depreciation | | | | | ||
— Share-based compensation | | | | | ||
— Financial income and expenses | | | | ( | | ( |
— Results related to financial liabilities measured at fair value through profit or loss | | | | ( | | |
— Income tax expenses | | 18 | | — | | |
| | | | | | |
Changes in working capital | | | | ( | | ( |
Cash used in operations | | | | ( | ( | |
| | | |
| |
|
Corporate income tax paid | | | | — | | ( |
Interest received | | | | | ||
Interest paid | | | | ( | | ( |
| | | |
| |
|
Net cash used in operating activities | | | | ( | | ( |
| | | |
| |
|
Cash flow from investing activities | | | | | | |
Increase in financial asset - current | | | | — | | ( |
Purchases of property, plant and equipment | | | | ( | | ( |
| | | |
| |
|
Net cash used in investing activities | | | | ( | | ( |
| | | |
| |
|
Cash flow from financing activities | | | |
| |
|
Proceeds from exercise of share options | | 12 | | | ||
Repayment of lease liability | | 10 | | ( | | ( |
| | | |
| |
|
Net cash used in financing activities | | | | ( | | ( |
| | | |
| |
|
Net decrease in cash and cash equivalents | | | | ( | | ( |
| | | |
| |
|
Currency effect cash and cash equivalents | | | | ( | | |
Cash and cash equivalents at beginning of the period | | 5 | | | ||
| | | |
| |
|
Cash and cash equivalents at the end of the period | | | | | ||
The accompanying notes are an integral part of these unaudited interim condensed consolidated financial statements.
PROQR THERAPEUTICS N.V.
Notes to Unaudited Condensed Consolidated Financial Statements
1. General Information
ProQR Therapeutics N.V., or “ProQR” or the “Company”, is a biotechnology company domiciled in the Netherlands that primarily focuses on the discovery and development of novel therapeutic medicines.
Since September 18, 2014, the Company’s ordinary shares have been listed on Nasdaq. They are currently trading at Nasdaq Capital Market under ticker symbol PRQR.
The Company was incorporated in the Netherlands, on February 21, 2012 (Chamber of Commerce no. 54600790) and was reorganized from a private company with limited liability to a public company with limited liability on September 23, 2014. The Company has its statutory seat in Leiden, the Netherlands. The address of its headquarters and registered office is Zernikedreef 9, 2333 CK Leiden, the Netherlands.
ProQR Therapeutics N.V. is the ultimate parent company of the following entities:
| ● | ProQR Therapeutics Holding B.V. ( |
| ● | ProQR Therapeutics I B.V. ( |
| ● | ProQR Therapeutics II B.V. ( |
| ● | ProQR Therapeutics III B.V. ( |
| ● | ProQR Therapeutics IV B.V. ( |
| ● | ProQR Therapeutics V B.V. ( |
| ● | ProQR Therapeutics VI B.V. ( |
| ● | ProQR Therapeutics VII B.V. ( |
| ● | ProQR Therapeutics VIII B.V. ( |
| ● | ProQR Therapeutics IX B.V. ( |
| ● | ProQR Therapeutics I Inc. ( |
ProQR Therapeutics N.V. is also statutory director of Stichting Bewaarneming Aandelen ProQR (“ESOP Foundation”) and has full control over this entity.
As used in these condensed consolidated financial statements, unless the context indicates otherwise, all references to “ProQR” or the “Company” refer to ProQR Therapeutics N.V. including its subsidiaries and the ESOP Foundation.
2. Significant Accounting Policies
These interim condensed consolidated financial statements for the three month period ended March 31, 2025 have been prepared in accordance with IAS 34 Interim Financial Statements. They should be read in conjunction with the Company’s annual financial statements for the year ended December 31, 2024. These interim condensed consolidated financial statements do not include all information required for a complete set of financial statements prepared in accordance with IFRS Standards. However, selected explanatory notes are included to explain events and transactions that are significant to an understanding of the changes in the Company’s financial position and performance since the last annual financial statements. In the opinion of management, all events and transactions that are significant to an understanding of the changes in financial position and performance of the Company since the end of the last annual reporting period are disclosed in these interim condensed consolidated financial statements. The accounting policies adopted in the preparation of the interim condensed consolidated financial statements are consistent with those applied in the preparation of the Company’s annual financial statements for the year ended December 31, 2024.
During the quarter ended March 31, 2024, the Company invested in financial assets in the form of deposits with an original maturity of longer than three months but shorter than twelve months which are measured at amortized cost as they give rise on specified dates to cash flows that are solely payments of principal and interest on the principal amount outstanding.
The Company’s financial results have varied substantially, and are expected to continue to vary, from period to period. The Company believes that its ordinary activities are not linked to any particular seasonal factors.
The management of ProQR has, upon preparing and finalizing these interim condensed consolidated financial statements, assessed the Company’s ability to fund its operations for a period of at least one year after the date of signing these interim condensed consolidated financial statements. Management expects the Company to continue as a going concern based on its existing funding, taking into account the Company’s current cash position and the projected cash flows based on the activities under execution on the basis of ProQR’s business plan and budget. Thus, we continue to adopt the going concern basis of accounting in preparing the interim condensed consolidated financial statements.
The carrying amount of all financial assets and financial liabilities is a reasonable approximation of the fair value and therefore, information about the fair values of each class has not been disclosed.
The Company operates in
3. Adoption of New and Revised International Financial Reporting Standards
New Standards and Interpretations, which became effective as of January 1, 2025, did not have a material impact on our condensed consolidated financial statements.
4. Critical Accounting Estimates and Judgements
In the application of the Company’s accounting policies, management is required to make judgments, estimates and assumptions about the carrying amounts of assets and liabilities that are not readily apparent from other sources. The estimates and associated assumptions are based on historical experience and other factors that are considered to be relevant. Actual results may differ from these estimates.
The estimates and underlying assumptions are reviewed on an ongoing basis. Revisions to accounting estimates are recognized in the period in which the estimate is revised if the revision affects only that period or in the period of the revision and future periods if the revision affects both current and future periods.
The significant judgements made by management in applying the Company’s accounting policies and the key sources of estimation uncertainty were the same as those described in the Company’s annual financial statements for the year ended December 31, 2024.
(i) Revenue recognition for the Eli Lilly and Company research and collaboration agreement
a. Identification of the performance obligations
As further described in Note 13 the identification of the performance obligations for the Company’s original research and collaboration agreement with Eli Lilly and Company (“Lilly”), and the amended and restated research and collaboration agreement (collectively, the “Collaboration agreement”) involves significant judgement.
A key judgement was made in determining that the license granted to Lilly is not distinct from the associated research and development (“R&D”) services, due to the lack of stand-alone value of the license without the Company’s involvement and the significant interdependencies between the license and the R&D services to be provided by the Company. As a result, the license and the R&D services are accounted for together as a single combined performance obligation consisting of multiple activities that are not distinct.
b. Determining the timing of satisfaction of performance obligations
As further described in Note 13, before the handover of a compound to Lilly, the Company recognizes revenue over time, using an input method that estimates the satisfaction of the performance obligation as the percentage of labor hours incurred compared to the total estimated labor hours required to complete the promised services. As the Company’s estimate of the total labor hours required is dependent on the evolution of the research and development activities, it may be subject to change. If the progression and/or outcome of certain research and development activities would be different from the assumptions that were made during the preparation of these financial statements, this could lead to material adjustments to the total estimated labor hours, which might result in a reallocation of revenue between current and future periods. Our total deferred revenue balance related to this Lilly performance obligation amounts to €
c. Determining the transaction price
The Company applied judgement to determine whether the equity investments made by Lilly in ProQR are part of the transaction price for the Collaboration agreement. The Company concluded that the differences between the prices that Lilly paid for the shares and the ProQR stock closing prices on the days of entering into the equity investment agreements arose because of the Company’s existing obligations to deliver research and development services to Lilly under the terms of the Collaboration agreement. Therefore, the above differences between the closing share prices on the agreement effective dates and the equity investment prices paid by Lilly are considered to be part of the transaction price of the contract and are initially allocated to deferred revenue.
The contract also includes variable consideration, but no variable consideration was included in the initial transaction price at the inception, as it is not highly probable that a significant reversal in the amount of cumulative revenue recognized will not occur. The Company includes such variable consideration in the transaction price when the uncertainty associated with the variable consideration is resolved.
The Collaboration agreement includes variable consideration in the form of development milestones, commercial milestones, and sales-based royalties based on the level of sales. As further described in Note 13, during March 31, 2025 the Company achieved development milestones during the ProQR research program under the agreement, which were added to the transaction price and recognized partially as revenue during 2025 based on the status of completion (satisfied part) of the single combined performance obligation.
(ii) Research and development expenditures
Research expenditures are reflected in the income statement. Development expenses are currently also reflected in the income statement because the criteria for capitalization are not met. At each balance sheet date, the Company estimates the level of service performed by the vendors and the associated costs incurred for the services performed.
Although we do not expect the estimates to be materially different from amounts actually incurred, the understanding of the status and timing of services performed relative to the actual status and timing of services performed may vary and could result in reporting amounts that are too high or too low in any particular period.
5. Cash and Cash Equivalents
At March 31, 2025, the Company’s cash and cash equivalents were €
6. Prepayments and Other Receivables
| | March 31, | | December 31, |
| | 2025 | | 2024 |
| | €1,000 | | €1,000 |
Prepayments | | | ||
Other receivables | | | ||
Accrued income from Rett Syndrome Research Trust | | | ||
| | |
All receivables are considered short-term and due within one year. At March 31, 2025 and December 31, 2024 prepayments consisted principally of payments made by the Company for services not yet provided by vendors. At March 31, 2025 and December 31, 2024 other receivables consisted principally of accrued grant income and deposits. As at March 31, 2025 and December 31, 2024 the accrued grant income relating to Rett Syndrome Research Trust (“RSRT”) includes the initial fair value of the warrants issued to RSRT that was accounted for as a reduction of the transaction price. The RSRT agreement is described in Note 14. Other Income.
7. Property, Plant and Equipment
At March 31, 2025 and December 31, 2024, property plant and equipment consisted of buildings and leasehold improvements, laboratory equipment and other assets. Buildings and leasehold improvements include a right-of-use asset relating to the lease of our Leiden office and laboratory space, with a carrying amount of €
8. Other Current Liabilities
At March 31, 2025, other current liabilities amount to €
9. Borrowings
| | March 31, | | December 31, |
|
| 2025 | | 2024 |
| | €1,000 | | €1,000 |
Innovation credit | | | ||
Accrued interest on innovation credit | | | ||
| | | | |
Total borrowings | | | ||
Current portion | | | ||
Total non-current borrowings | | — | | — |
In December 2018, ProQR was awarded an Innovation credit for the sepofarsen program. Amounts were drawn under this facility from 2018 through 2022. The credit of €
In December 2023, ProQR received a waiver for the remaining balance of the Innovation credit including accrued interest. As a result, the repayment of the total loan of €
In September 2022, ProQR extinguished its debt with Pontifax and Kreos by repaying all outstanding principal amounts. Pontifax’ and Kreos’ warrants remain in place until their
10. Lease Liabilities
At March 31, 2025 and December 31, 2024, lease liabilities primarily consisted of the Company’s lease of office and laboratory facilities at Zernikedreef in Leiden, the Netherlands.
The Company leases office and laboratory facilities of
The initial lease agreement for the Leiden office and laboratory facilities was accounted for as of commencement date July 1, 2020. This period was extended by
The carrying amount of the right-of-use asset is disclosed in Note 7.
11. Deferred Income
The following table summarizes details of deferred income at March 31, 2025 and December 31, 2024. The nature of the deferred income is described in Note 13 and 14.
| | March 31, | | December 31, |
| | 2025 | | 2024 |
| | €1,000 | | €1,000 |
| | | | |
Payments from Eli Lilly and Company | | | ||
Payments from Rett Syndrome Research Trust | | | ||
Current deferred income | | | ||
| | | | |
Payments from Eli Lilly and Company | | | ||
Non-current deferred income | | | ||
| | | | |
Total deferred income | | |
c
12. Shareholders’ Equity
The authorized share capital of the Company amounting to €
In September 2024, the Company filed a shelf registration statement on Form F-3, which permitted: (a) the offering, issuance and sale by the Company of up to a maximum aggregate offering price of $
In October 2024, the Company consummated an underwritten public offering of
Concurrently with the Offering, the Company entered into a share purchase agreement with Lilly in a separately negotiated transaction (the “concurrent private placement”), pursuant to which the Company agreed to offer and sell, and Lilly agreed to purchase,
Translation reserve
The translation reserve comprises all foreign currency differences arising from the translation of the financial statements of foreign operations.
Share options
The Company operates an equity-settled share-based compensation plan, which was introduced in 2013. Options and Restricted Stock Units (“RSUs”) may be granted to employees, members of the Board and consultants. The compensation expenses included in operating costs for this plan in the three month period ended March 31, 2025 were €
13. Revenue
Eli Lilly and Company collaboration
In September 2021, the Company entered into a global licensing and research collaboration with Lilly focused on the discovery, development, and commercialization of potential new medicines for genetic disorders in the liver and nervous system. ProQR and Lilly will use ProQR’s proprietary Axiomer® RNA editing platform to progress new drug targets toward clinical development and commercialization.
Under the terms of the agreement, ProQR received an upfront payment and equity consideration, and is eligible to receive milestone payments and royalties on the net sales of any resulting products. In September 2021, the Company issued
In December 2022, the Company and Lilly amended their research and collaboration agreement described above, which expanded the collaboration. Under the amended and restated research and collaboration agreement, Lilly will gain access to additional targets in the central nervous system (“CNS”) and peripheral nervous system (“PNS”) with ProQR’s Axiomer platform.
As described under Note 12, pursuant to the amended and restated agreement, the Company issued
With regard to the original and amended and restated research and collaboration agreements with Lilly, the Company concluded as follows:
| ● | The amended and restated research and collaboration agreement is accounted for as a separate contract under IFRS 15 given the group of promises to be delivered are distinct and are priced commensurate with stand-alone selling prices. |
| ● | For each of the agreements, the company identified |
| ● | The transaction price of the agreement includes fixed components, consisting of an up-front fee and an equity component (premium or discount). The agreement also contains variable parts, notably milestones, which are included in the transaction price to the extent that it is highly probable that a significant reversal in the amount of cumulative revenue recognized will not occur when the uncertainty associated with the variable consideration is subsequently resolved. Development milestone payments to be reached during the ProQR research program will only be included to the extent that it is highly probable that a significant reversal in the amount of cumulative revenue recognized will not occur when the uncertainty associated with the milestones is subsequently resolved. Sales-based milestones and sales-based royalties will be included as the underlying sales occur. |
| ● | Initially, the Company recognizes revenue over time, using an input method that estimates the satisfaction of the performance obligation as the percentage of labor hours incurred compared to the total estimated labor hours required to complete the promised services. |
After the handover of a compound to Lilly:
| ● | The variable consideration for development milestones to be reached during the Lilly R&D activities is linked to a separable right to use the license which comes into existence for each successful compound transferred to Lilly. This license is a separate performance obligation and revenue will be recognized at a point in time when the development milestone for a license is achieved and the variable constraint is resolved. |
| ● | The variable consideration for commercial milestones is linked to a separable right to use the license which comes into existence for each successful compound transferred to Lilly. This license is a separate performance obligation and will be recognized at a point in time when the commercial milestone for a license is achieved and the variable constraint is resolved. |
| ● | For sales-based royalties, the license is the predominant item to which the royalty relates. The sales-based royalties will be recognized after the handover of the compound to Lilly (after completion of the initial performance obligation) and once the respective sale level occurs. |
During the three month period ended March 31, 2025, the Company reached milestones amounting to $
14. Other Income
| | Three month period | ||
| | ended March 31, | ||
|
| 2025 | | 2024 |
| | €1,000 | | €1,000 |
| | | | |
Grant income | | | ||
| | | ||
In January, 2024, the Company entered into an agreement with the RSRT that focuses on the design and development of editing oligonucleotides (“EONs”) using the Company’s Axiomer technology platform targeting the transcription factor Methyl CpG binding protein 2 (“MECP2”) and correcting mutations of interest. Under the agreement, RSRT awarded the Company up to €
Grants are recognized in other income in the same period in which the related R&D costs are recognized.
15. Research and Development Costs
Research and development costs amount to €
16. General and Administrative Costs
General and administrative costs amount to €
17. Investments in Financial Assets
Investment in financial assets consist of the Company’s investment in Phoenicis Therapeutics Inc. (“Phoenicis”) and Yarrow Biotechnology Inc. (“Yarrow”).
ProQR holds a
ProQR holds a
18. Income Taxes
The current income tax liability amounts to €
Tax losses may be carried forward indefinitely. However, the offset of losses will be limited in a given year against the first € 1 million of taxable profit. For taxable profit in excess of this amount, losses may only be offset up to 50% of this excess. In addition, unused non-deductible interest expenses may be carried forward indefinitely. However, the offset will be limited in a given year against the higher of 20% of adjusted taxable profit or € 1.0 million of interest income.
19. Related-Party Transactions
The Company does not have any transactions with related parties other than compensation to its Board members.
20. Events After Balance Sheet Date
None.
Exhibit 99.2
ProQR Announces First Quarter 2025 Operating and Financial Results
| ● | Axiomer™ ADAR-mediated RNA editing pipeline advancing across liver and CNS programs, with CTA filing on track for Q2 2025 for lead program AX-0810 targeting NTCP for Cholestatic diseases |
| ● | Strengthened leadership with appointments of Chief Financial Officer and Chief Medical Officer |
| ● | € 132.4 million cash and cash equivalents as of end Q1 providing runway into mid-2027, plus additional potential milestones from Lilly partnership |
LEIDEN, Netherlands & CAMBRIDGE, Mass., May 8, 2025 – ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer RNA editing technology platform, today reported its financial and operating results for the first quarter ended March 31, 2025, and provided a business update.
“ProQR entered 2025 in a position of strength, with solid fundamentals, a well-capitalized balance sheet, and a clear focus on execution,” said Daniel A. de Boer, Founder and Chief Executive Officer of ProQR. “We remain on track to submit the CTA for our lead RNA editing program, AX-0810 targeting NTCP for cholestatic diseases, in Q2 2025, with our first clinical data readout expected in Q4 – key milestones for our lead Axiomer pipeline program that we believe will drive significant value for stakeholders. I’m delighted that we further strengthened our leadership team with the recent appointments of Chief Financial Officer Dennis Hom and Chief Medical Officer Dr. Cristina Lopez Lopez, bringing deep financial and translational clinical development expertise to support ProQR’s next phase of growth.”
| ● | In April, ProQR announced the appointments of Dennis Hom as Chief Financial Officer and Cristina Lopez Lopez, MD, PhD as Chief Medical Officer. These key leadership appointments support the advancement of the Company’s Axiomer platform technology and pipeline of RNA editing programs as it enters the clinical stage. |
| ● | Advancing AX-0810 toward CTA filing, on track for Q2 2025. AX-0810 is ProQR’s lead Axiomer RNA editing pipeline program. By modulating NTCP, AX-0810 aims to reduce bile acid accumulation in hepatocytes and address the root cause of cholestatic liver |
| diseases. The program remains on track for a Clinical Trial Application (CTA) filing in Q2 2025, with the initial clinical assessment planned in healthy volunteers. |
| ● | In March, ProQR participated in the RNA Editing Gordon Research Conference in Lucca, Italy, presenting “Developing Axiomer™ RNA Editing Technology Towards Application in Liver and CNS Disease”. |
| ● | Accepted scientific abstracts underscore ProQR’s leadership in RNA editing. ProQR will present multiple abstracts at the upcoming American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, taking place May 13-17, 2025 in New Orleans. These presentations will highlight the Company’s proprietary Axiomer RNA editing platform and pipeline programs, including: |
| ● | Oral Presentation (333): “ADAR-Mediated RNA Editing of SLC10A1 (NTCP) as a Therapeutic Approach to Reduce Liver Bile Acid Re-Uptake in Cholestatic Diseases” |
| ● | Poster Presentation (AMA1324): “ADAR-Mediated RNA Editing-Based Correction of PNPLA3|148M Functionality to Address Hepatic Steatosis” |
| ● | Poster Presentation (AMA1427): “ADAR-Mediated RNA Editing of Premature Termination Codon Results in Functional Correction in MECP2 for Rett Syndrome |
Anticipated Upcoming Events
| ● | AX-0810 targeting NTCP for Cholestatic diseases |
| ● | Q2 2025 Clinical Trial Application (CTA) submission |
| ● | Q4 2025 first clinical target engagement and biomarker data in healthy volunteers |
| ● | AX-2402 targeting MECP2 (R270X) for Rett Syndrome |
| ● | 2025 clinical candidate selection |
| ● | 2026 clinical trial initiation and topline data readout |
| ● | AX-2911 targeting PNPLA3 for MASH |
| ● | 2025 clinical candidate selection |
| ● | 2026 clinical trial initiation and topline data readout |
| ● | AX-1412 targeting B4GALT1 for Cardiovascular diseases |
| ● | mid-2025 update on optimization for GalNAc delivery |
| ● | ProQR may selectively form new partnerships, which could include multi-target discovery alliances, or product alliances on specific programs. |
Financial Highlights
At March 31, 2025, ProQR held cash and cash equivalents of € 132.4 million, compared to
€ 149.4 million cash and cash equivalents at December 31, 2024. Net cash used in operating activities during the three-month period ended March 31, 2025 was € 15.8 million, compared to € 15.1 million for the same period last year. During the first quarter of 2025, the Company achieved a milestone in the collaboration agreement with Eli Lilly earning $1.0 million (€ 918,000).
Research and development (R&D) costs were € 12.3 million for the quarter ended March 31, 2025 compared to € 9.3 million for the same period last year.
General and administrative costs were € 3.2 million for the quarter ended March 31, 2025 compared to € 3.5 million for the same period last year.
Net loss for the three-month period ended March 31, 2025 was € 10.1 million, or € 0.10 per diluted share, compared to € 7.7 million, or € 0.09 per diluted share, for the same period last year. For further financial information for the period ended March 31, 2025, please refer to the Q1 financial report filing available on our website, www.proqr.com under Financials and Filings.
About Axiomer™
ProQR is pioneering a next-generation RNA base editing technology called Axiomer™, which could potentially yield a new class of medicines for diverse types of diseases. Axiomer™ “Editing Oligonucleotides”, or EONs, mediate single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human cells called ADAR (Adenosine Deaminase Acting on RNA). Axiomer™ EONs are designed to recruit and direct endogenously expressed ADARs to change an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G) – correcting an RNA with a disease-causing mutation back to a normal (wild type) RNA, modulating protein expression, or altering a protein so that it will have a new function that helps prevent or treat disease.
About ProQR
ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies. ProQR is pioneering a next-generation RNA technology called Axiomer™, which uses a cell’s own editing machinery called ADAR to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for both rare and prevalent diseases with unmet need.
Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.
Learn more about ProQR at www.proqr.com.
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 “continue,” "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Such forward-looking statements include, but are not limited to, statements regarding our business, technology, strategy, preclinical and clinical model data, our initial pipeline targets and the upcoming strategic priorities and milestones related thereto, the responsibilities, potential strengths and capabilities of our new leadership appointments, the continued advancement of our lead development pipeline programs, including ongoing and planned clinical trials, the anticipated timing of initial clinical data readouts across multiple programs in 2025 and 2026, and the filing of a CTA application for our lead program, AX-0810, in Q2 2025, our Axiomer™ platform, including the continued development and advancement of our Axiomer platform, the therapeutic potential of our Axiomer RNA editing oligonucleotides and product candidates, the timing, progress and results of our preclinical studies and other development activities, including the release of data related thereto, our patent estate, including our anticipated strength and our continued investment in it, as well as the timing of our clinical development, the potential of our technologies and product candidates, the collaboration with Lilly and the intended benefits thereof, including timing for data updates, potential milestones, exercise of an option to expand targets and the receipt of an opt-in payment, our ability to selectively form new partnerships and enter into future collaborations, and our financial position and cash-runway. 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. Our actual results could differ materially from those expressed or implied by these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our most recent annual report filed on Form 20-F. These risks and uncertainties include, among others, the cost, timing and results of preclinical studies and clinical trials and other development activities by us and our collaborative partners whose operations and activities may be slowed or halted shortage and pressure on supply and logistics on the global market, economic sanctions and international tariffs; the likelihood of our preclinical and clinical programs being initiated
and executed on timelines provided and reliance on our contract research organizations and predictability of timely enrollment of subjects and patients to advance our clinical trials and maintain their own operations; our reliance on contract manufacturers to supply materials for research and development and the risk of supply interruption from a contract manufacturer; the potential for future data to alter initial and preliminary results of early-stage clinical trials; the unpredictability of the duration and results of the regulatory review of applications or clearances that are necessary to initiate and continue to advance and progress our clinical programs; the ability to secure, maintain and realize the intended benefits of collaborations with partners, including the collaboration with Lilly; the possible impairment of, inability to obtain, and costs to obtain intellectual property rights; possible safety or efficacy concerns that could emerge as new data are generated in research and development; general business, operational, financial and accounting risks, and risks related to litigation and disputes with third parties; and risks related to macroeconomic conditions and market volatility resulting from global economic developments, geopolitical events and conflicts, high inflation, rising interest rates, tariffs and potential for significant changes in U.S. policies and regulatory environment. 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, except as required by law.
ProQR Therapeutics N.V.
Investor and media contact:
Sarah Kiely
ProQR Therapeutics N.V.
T: +1 617 599 6228
skiely@proqr.com
or
Investor contact:
Peter Kelleher
LifeSci Advisors
T: +1 617 430 7579
pkelleher@lifesciadvisors.com
Financial Tables
PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Financial Position
| | | | March 31, | | December 31, |
| | | | 2025 | | 2024 |
| | | | €1,000 | | €1,000 |
Assets | | | |
| |
|
Property, plant and equipment | | | | 13,608 | | 14,113 |
Investments in financial assets | | | | — | | — |
Non-current assets | | | | 13,608 | | 14,113 |
| | | | | | |
Cash and cash equivalents | | | | 132,414 | | 149,408 |
Prepayments and other receivables | | | | 5,137 | | 3,747 |
Other taxes | | | | 530 | | 690 |
Current assets | | | | 138,081 | | 153,845 |
Total assets | | | | 151,689 | | 167,958 |
| | | | | | |
Equity and liabilities | | | |
| |
|
Equity | | | | | |
|
Equity attributable to owners of the Company | | | | 78,935 | | 88,560 |
Total equity | | | | 78,935 | | 88,560 |
| | | | | | |
Liabilities | | | |
| |
|
Borrowings | | | | — | | — |
Lease liabilities | | | | 10,738 | | 11,067 |
Deferred income | | | | 28,299 | | 29,429 |
Non-current liabilities | | | | 39,037 | | 40,496 |
| | | | | | |
Borrowings | | | | 4,655 | | 4,582 |
Lease liabilities | | | | 1,460 | | 1,567 |
Derivative financial instruments | | | | 186 | | 468 |
Trade payables | | | | 414 | | 16 |
Social securities and other taxes | | | | 481 | | 1,478 |
Deferred income | | | | 19,250 | | 21,942 |
Other current liabilities | | | | 7,271 | | 8,849 |
Current liabilities | | | | 33,717 | | 38,902 |
Total liabilities | | | | 72,754 | | 79,398 |
| | | | | | |
Total equity and liabilities | | | | 151,689 | | 167,958 |
PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Profit or Loss and OCI
(€ in thousands, except share and per share data)
| | | | Three month period | ||
| | | | ended March 31, | ||
|
| | | 2025 | | 2024 |
| | | | €1,000 | | €1,000 |
Revenue | | | | 4,519 | | 4,450 |
| | | | | | |
Other income | | | | 222 | | 210 |
| | | | | | |
Research and development costs | | | | (12,323) | | (9,283) |
General and administrative costs | | | | (3,234) | | (3,452) |
Total operating costs | | | | (15,557) | | (12,735) |
| | | |
| |
|
Operating result | | | | (10,816) | | (8,075) |
Finance income and expense | | | | 455 | | 488 |
Results related to financial liabilities measured at fair value through profit or loss | | | | 282 | | (68) |
| | | |
| |
|
Result before corporate income taxes | | | | (10,079) | | (7,655) |
Income taxes | | | | — | | (3) |
| | | |
| |
|
Result for the period | | | | (10,079) | | (7,658) |
Other comprehensive income (foreign exchange differences on foreign operation) | | | | (371) | | 191 |
| | | |
| |
|
Total comprehensive income | | | | (10,450) | | (7,467) |
| | | | | | |
Result attributable to | | | |
| |
|
Owners of the Company | | | | (10,079) | | (7,658) |
Non-controlling interests | | | | — | | — |
| | | | (10,079) | | (7,658) |
Total comprehensive income attributable to | | | | | | |
Owners of the Company | | | | (10,450) | | (7,467) |
Non-controlling interests | | | | — | | — |
| | | | (10,450) | | (7,467) |
| | | |
| |
|
Share information | | | |
| |
|
Weighted average number of shares outstanding1 | | | | 105,296,833 | | 81,571,028 |
| | | | | | |
Earnings per share attributable to owners of the Company (Euro per share) | | | | | | |
Basic loss per share1 | | | | (0.10) | | (0.09) |
Diluted loss per share1 | | | | (0.10) | | (0.09) |
| 1. | For these periods the potential exercise of share options is not included in the diluted earnings per share as the Company was loss-making. Due to the anti-dilutive nature of the outstanding options, basic and diluted earnings per share are equal. |
PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Changes in Equity
| | Attributable to owners of the Company | | | | | ||||||||||||
|
| Number |
| Share |
| Share |
| Equity settled |
| Translation |
| Accumulated |
| Total |
| Non- |
| Total |
|
|
| | €1,000 | | €1,000 | | €1,000 | | €1,000 | | €1,000 | | €1,000 | | €1,000 | | €1,000 |
Balance at January 1, 2024 |
| 84,248,384 | | 3,370 | | 412,894 | | 25,159 | | 817 | | (400,850) | | 41,390 | | — | | 41,390 |
Result for the period |
| — | | — | | — | | — | | — | | (7,658) | | (7,658) | | — | | (7,658) |
Other comprehensive income |
| — | | — | | — | | — | | 191 | | — | | 191 | | — | | 191 |
Recognition of share-based payments |
| — | | — | | — | | 736 | | — | | — | | 736 | | — | | 736 |
Treasury shares transferred | | (307,627) | | — | | — | | — | | — | | — | | — | | — | | — |
Share options lapsed | | — | | — | | — | | (40) | | — | | 40 | | — | | — | | — |
Share options exercised / RSUs vested | | 307,627 | | — | | 162 | | (278) | | — | | 278 | | 162 | | — | | 162 |
| | | | | | | | | | | | | | | | | | |
Balance at March 31, 2024 |
| 84,248,384 | | 3,370 | | 413,056 | | 25,577 | | 1,008 | | (408,190) | | 34,821 | | — | | 34,821 |
| | | | | | | | | | | | | | | | | | |
Balance at January 1, 2025 |
| 107,710,916 | | 4,308 | | 483,812 | | 26,248 | | 1,350 | | (427,158) | | 88,560 | | — | | 88,560 |
Result for the period |
| — | | — | | — | | — | | — | | (10,079) | | (10,079) | | — | | (10,079) |
Other comprehensive income |
| — | | — | | — | | — | | (371) | | — | | (371) | | — | | (371) |
Recognition of share-based payments |
| — | | — | | — | | 758 | | — | | — | | 758 | | — | | 758 |
Treasury shares transferred | | (130,436) | | — | | — | | — | | — | | — | | — | | — | | — |
Share options lapsed | | — | | — | | — | | (826) | | — | | 826 | | — | | — | | — |
Share options exercised / RSUs vested | | 130,436 | | — | | 67 | | (180) | | — | | 180 | | 67 | | — | | 67 |
|
|
| |
| |
| |
| |
| |
| |
| |
| |
|
Balance at March 31, 2025 |
| 107,710,916 | | 4,308 | | 483,879 | | 26,000 | | 979 | | (436,231) | | 78,935 | | — | | 78,935 |
PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Cash Flows
| | | | Three month period | ||
| | | | ended March 31, | ||
|
| | | 2025 | | 2024 |
| | | | €1,000 | | €1,000 |
Cash flows from operating activities | | | |
| |
|
Net result | | | | (10,079) | | (7,658) |
Adjustments for: | | | | | | |
— Other income | | | | (222) | | — |
— Depreciation | | | | 678 | | 691 |
— Share-based compensation | | | | 758 | | 736 |
— Financial income and expenses | | | | (508) | | (488) |
— Results related to financial liabilities measured at fair value through profit or loss | | | | (282) | | 68 |
— Income tax expenses | | | | — | | 3 |
| | | | | | |
Changes in working capital | | | | (6,721) | | (9,224) |
Cash used in operations | | | | (16,376) | | (15,872) |
| | | |
| |
|
Corporate income tax paid | | | | — | | (3) |
Interest received | | | | 788 | | 932 |
Interest paid | | | | (210) | | (189) |
| | | |
| |
|
Net cash used in operating activities | | | | (15,798) | | (15,132) |
| | | |
| |
|
Cash flow from investing activities | | | | | | |
Increase in financial asset - current | | | | — | | (17,000) |
Purchases of property, plant and equipment | | | | (224) | | (732) |
| | | |
| |
|
Net cash used in investing activities | | | | (224) | | (17,732) |
| | | |
| |
|
Cash flow from financing activities | | | |
| |
|
Proceeds from exercise of share options | | | | 67 | | 162 |
Repayment of lease liability | | | | (567) | | (581) |
| | | |
| |
|
Net cash used in financing activities | | | | (500) | | (419) |
| | | |
| |
|
Net decrease in cash and cash equivalents | | | | (16,522) | | (33,283) |
| | | |
| |
|
Currency effect cash and cash equivalents | | | | (472) | | 71 |
Cash and cash equivalents at beginning of the period | | | | 149,408 | | 118,925 |
| | | |
| |
|
Cash and cash equivalents at the end of the period | | | | 132,414 | | 85,713 |