ProQR Therapeutics N.V. (PRQR) Business Model Canvas

ProQR Therapeutics N.V. (PRQR): Business Model Canvas [Dec-2025 Updated]

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You're looking at ProQR Therapeutics N.V. (PRQR) and wondering where the value is hiding, right? Honestly, after two decades analyzing biotech plays, I see this as a classic platform bet: the proprietary Axiomer™ RNA editing technology. The near-term story hinges on execution, especially advancing AX-0810 while managing the cash runway, which stood at about € 106.9 million in Q3 2025. That cash, coupled with the Eli Lilly collaboration-which brought in $2.0 million in milestone payments for the first nine months of 2025-is funding heavy Research and Development (R&D), which hit € 34.8 million over the same period. So, the model is simple: use platform validation and partnership cash to reach critical data readouts before the runway ends mid-2027. Dive into the full canvas below to see exactly how they are structuring this high-stakes race.

ProQR Therapeutics N.V. (PRQR) - Canvas Business Model: Key Partnerships

You're looking at the structure of ProQR Therapeutics N.V.'s alliances, which are critical for funding and advancing its Axiomer RNA editing platform. These partnerships de-risk the pipeline, especially for the Central Nervous System (CNS) programs.

Eli Lilly and Company for global licensing and research collaboration

The collaboration with Eli Lilly and Company is a massive strategic anchor for ProQR Therapeutics N.V. This is a global licensing and research deal valued up to $3.9 billion in total potential payments, which is a huge vote of confidence in the Axiomer technology. You need to track the milestone payments coming from this deal, as they directly impact cash runway.

For the first half of 2025, ProQR Therapeutics N.V. recognized milestone income of $2.0 million from Eli Lilly and Company. Specifically, a $1.0 million milestone payment was triggered in the first quarter of 2025. As of June 30, 2025, ProQR Therapeutics N.V. held cash and cash equivalents of approximately €119.8 million, with the Lilly milestones contributing to the runway extending into mid-2027.

Rett Syndrome Research Trust for AX-2402 development funding

The partnership with the Rett Syndrome Research Trust (RSRT) is specifically focused on advancing AX-2402, an RNA editing therapy targeting the R270X mutation in the MECP2 gene for Rett Syndrome. This funding is essential for moving a CNS program forward, which often requires external support to de-risk early stages.

The total funding secured from RSRT for AX-2402 development is reported as up to $9.2 million, building upon an initial $1 million grant from January 2024 with an additional $8.1 million. This support is aimed at accelerating the program toward clinical trials, with a Phase 1/2 trial planned for 2026. The accrued grant income related to RSRT, including the initial fair value of warrants issued to RSRT, was recorded as of June 30, 2025.

Here's a quick look at the RSRT funding progression:

Funding Event Amount Date Context
Initial Grant $1.0 million January 2024
Additional Funding $8.1 million December 2024
Total Funding Up to $9.2 million As of early 2025 reports

Clinical research organizations and academic institutions for trials

For ProQR Therapeutics N.V.'s lead program, AX-0810, clinical execution relies on specific site selection. The Phase 1 study for AX-0810 is authorized to begin in the Netherlands. This initial study is being conducted at a single site.

Key academic involvement is highlighted by the planned participation of Professor Henkjan J. Verkade, MD, PhD, as a Key Opinion Leader for the November 3, 2025, Analyst and Investor Event, focusing on the AX-0810 Phase 1 trial design.

  • Phase 1 AX-0810 site location: Netherlands
  • Trial to evaluate safety, PK, and target engagement biomarkers
  • Topline data for AX-0810 anticipated in Q4 2025

Potential future multi-target discovery alliances

The existing Eli Lilly and Company agreement has built-in expansion potential, which represents a clear path for future discovery alliances. The current deal includes an option for ProQR Therapeutics N.V. to expand the collaboration for an additional five targets, bringing the total to 15 targets. Exercising this option would trigger a significant $50 million opt-in payment to ProQR Therapeutics N.V.. This structure allows ProQR Therapeutics N.V. to secure future non-dilutive funding based on strategic expansion decisions by a major pharmaceutical partner.

ProQR Therapeutics N.V. (PRQR) - Canvas Business Model: Key Activities

ProQR Therapeutics N.V.'s key activities center on advancing its proprietary RNA editing technology and executing on its clinical pipeline, heavily supported by its strategic partnership.

Advancing the proprietary Axiomer™ RNA editing technology platform

The core activity involves the development and clinical application of the Axiomer™ RNA editing technology platform, which is described as a next-generation RNA base editing technology. This platform is being used across three key applications: modulate, correct, and protect.

The platform's first clinical evaluation is through its lead program, AX-0810. The company is focused on establishing proof of target engagement, safety, and pharmacokinetics (PK) in human subjects.

Key platform and technology metrics as of late 2025:

  • Cash and cash equivalents as of September 30, 2025: €106.9 million.
  • Projected cash runway into mid-2027.
  • Research and development (R&D) costs for the nine-month period ended September 30, 2025: €34.8 million.
  • Net cash used in operating activities for the nine-month period ended September 30, 2025: €39.4 million.

Conducting Phase 1 clinical trial for lead program AX-0810

The lead program, AX-0810, targets NTCP for the treatment of cholestatic diseases. A major activity in late 2025 is the initiation and execution of the first-in-human Phase 1 study.

The Clinical Trial Application (CTA) for AX-0810 was submitted in June 2025, and authorization was received in October 2025, allowing the study to begin dosing in healthy volunteers in the Netherlands.

Anticipated data readouts for AX-0810:

  • Initial safety, tolerability, and PK data from Cohort 1 expected by year-end 2025.
  • Target engagement data from all healthy volunteer cohorts expected in H1 2026.

Pre-clinical R&D for CNS and liver pipeline programs (e.g., AX-2402)

ProQR Therapeutics N.V. is actively managing its pipeline beyond the lead candidate, focusing on CNS and liver disorders using the Axiomer platform's ability to correct or modulate protein function.

The pipeline includes several programs in pre-clinical or early development stages:

Program Target Indication Axiomer Function Key Development Status/Funding
AX-0810 Cholestatic Diseases Modulate NTCP Phase 1 study initiating; Initial data by year-end 2025.
AX-2402 Rett Syndrome (R270X mutation) Correct MECP2 mutation Secured up to $9.2 million from Rett Syndrome Research Trust; Phase 1/2 trial planned for 2026.
AX-2911 MASH (I148M mutation) Modulate PNPLA3 Pre-clinical stage.
AX-1412 Cardiovascular Diseases Targeting B4GALT1 Mentioned in Q1 2025 update.

Managing the Eli Lilly collaboration and milestone execution

The collaboration with Eli Lilly and Company (Lilly) is a key financial and strategic activity, providing non-dilutive funding through milestone achievements.

Financial achievements from the Lilly collaboration for the nine-month period ending September 30, 2025:

  • Total milestone income achieved: $2.0 million (approximately €1.8 million).
  • Milestone payment received in Q2 2025: $2.0 million (approximately €1.8 million).
  • Milestone payment received in Q1 2025: $1.0 million (approximately €918,000).

The agreement structure includes a significant potential future payment:

Lilly has an option to expand the collaboration to a total of 15 targets, which would trigger an opt-in payment of $50 million to ProQR Therapeutics N.V.

ProQR Therapeutics N.V. (PRQR) - Canvas Business Model: Key Resources

When you look at the core assets ProQR Therapeutics N.V. is relying on right now, it's all about the platform and the capital to run the engine. These are the foundational elements that support everything else they are trying to achieve in RNA editing.

Proprietary Axiomer™ RNA editing technology platform

The Axiomer™ technology is ProQR Therapeutics N.V.'s next-generation RNA base editing platform. It's designed to make specific, single nucleotide changes to RNA inside human cells. It does this by recruiting the cell's own molecular machinery, specifically the ADAR (Adenosine Deaminase Acting on RNA) enzyme. This is achieved using what ProQR Therapeutics N.V. calls Editing Oligonucleotides, or EONs.

The platform is actively moving into human trials, which is a huge validation point for this key resource. The lead program, AX-0810, is the first clinical evaluation of an Axiomer editing oligonucleotide. This program targets NTCP for cholestatic diseases.

Here's a quick look at the platform's clinical progression as of late 2025:

  • Initiating Phase 1 study of AX-0810 in healthy volunteers.
  • CTA authorization received for the Phase 1 trial of AX-0810 in October 2025.
  • Initial safety, tolerability, and PK data from Cohort 1 expected by year-end 2025.
  • Target engagement data from all cohorts anticipated in the first half of 2026.

Strong intellectual property portfolio covering RNA editing

The intellectual property estate is critical because it protects the core mechanism: using an oligonucleotide to recruit endogenous ADAR in the cell. This is the fundamental protection for the Axiomer platform itself.

As of the reporting around the start of 2025, ProQR Therapeutics N.V. stated its global IP estate was extensive and growing. You need to know the scale of this protection.

IP Metric Value/Status as of Early 2025
Published Patent Families More than 20
Protection Focus Use of an oligonucleotide to recruit endogenous ADAR in the cell
Partnership Support Collaboration with Rett Syndrome Research Trust (RSRT) securing up to $9.2 million

This IP underpins the value of their strategic partnerships, like the one with Eli Lilly and Company, which is valued up to $3.9 billion.

Financial Capital and Operational Spending

You're looking at the runway, and that's where the balance sheet comes in. You need enough cash to get through the next set of critical clinical readouts. ProQR Therapeutics N.V. reported a solid cash position at the end of the third quarter of 2025, which they stated provides runway into mid-2027.

Here's the financial snapshot around that key resource:

Financial Metric (as of September 30, 2025) Amount
Cash and equivalents € 106.9 million
Cash and equivalents (as of June 30, 2025) € 119.8 million
Net cash used in operating activities (9 months ended Sept 30, 2025) € 39.4 million
Research and development (R&D) costs (9 months ended Sept 30, 2025) € 34.8 million
General and administrative (G&A) costs (9 months ended Sept 30, 2025) € 11.2 million
Net loss (9 months ended Sept 30, 2025) € 33.3 million
Milestone revenue from Eli Lilly (First 9 months of 2025) $2.0 million (approx. € 1.8 million)

The burn rate is definitely increasing; R&D costs for the nine-month period were € 34.8 million compared to € 25.7 million for the same period last year. Still, the cash on hand gives them time.

Specialized scientific and clinical development teams

The technology doesn't run itself, so the human capital is a definite key resource. You've got the specialized teams executing on the platform advancements and the clinical strategy. This includes the internal group managing the first-in-human study for AX-0810.

The structure of the team is supported by recent executive additions, which signals a focus on clinical execution:

  • Appointment of Chief Financial Officer Dennis Hom.
  • Appointment of Chief Medical Officer Dr. Cristina Lopez Lopez.
  • Engagement with Key Opinion Leaders, such as Professor Henkjan J. Verkade, for clinical insight.

These teams are focused on converting the Axiomer platform promise into human data. Finance: draft 13-week cash view by Friday.

ProQR Therapeutics N.V. (PRQR) - Canvas Business Model: Value Propositions

You're looking at the core of what ProQR Therapeutics N.V. is offering the market-it's a fundamental shift in how we treat genetic diseases. The value proposition centers on their proprietary Axiomer™ RNA editing technology platform, which is designed to be a next-generation approach compared to traditional gene therapies.

Pioneering next-generation RNA base editing for genetic diseases

ProQR Therapeutics N.V. is pioneering this next-generation RNA base editing technology called Axiomer™. This technology uses the cell's own editing machinery, specifically ADAR (Adenosine Deaminase Acting on RNA), to make precise, single nucleotide edits directly in the RNA molecule. This DNA-free, reversible editing approach is a key differentiator in the field. The company expects up to four clinical data readouts across its pipeline in 2025 and 2026, signaling an aggressive push to validate the platform.

Correcting disease-causing mutations directly at the RNA level

The core mechanism is the ability to make specific single nucleotide edits in RNA to either reverse a mutation or modulate protein expression. This capability falls under three key applications for the Axiomer pipeline: modulate, correct, and protect. The platform's clinical entry is marked by its lead program, AX-0810, which targets the NTCP protein. The Clinical Trial Application (CTA) for AX-0810 was submitted in Q2 2025, received authorization in October 2025, and initial safety, tolerability, and PK (pharmacokinetics) data from Cohort 1 of the Phase 1 study were expected toward the end of 2025.

Targeting high unmet need diseases like cholestatic diseases (AX-0810)

The immediate focus is on high unmet need areas. AX-0810 is specifically targeting NTCP for cholestatic liver diseases. This investigational RNA editing oligonucleotide is designed to selectively modulate NTCP function to reduce toxic bile acid accumulation in the liver, potentially stopping inflammation, fibrosis, and progression to liver failure. The strategy is grounded in human genetics, as naturally occurring variants like NTCP Q68R are associated with no clinical symptoms related to bile acid levels. The financial backing to pursue this is substantial; as of September 30, 2025, ProQR Therapeutics N.V. held € 106.9 million in cash and cash equivalents, providing a financial runway into mid-2027. This operational spending included Research and development (R&D) costs of € 34.8 million for the nine-month period ending September 30, 2025.

Potential to treat both rare and prevalent diseases with one platform

The platform's value extends beyond liver disease, positioning ProQR Therapeutics N.V. to address a broad spectrum of conditions. The Axiomer technology is intended to yield a new class of medicines for diverse types of diseases, covering both rare and prevalent conditions. The pipeline reflects this diversity, moving beyond the lead candidate. Here's a quick look at the pipeline programs that support this broad potential:

  • The lead program, AX-0810, targets NTCP for cholestatic diseases.
  • AX-2402 targets MECP2 (R270X) for Rett Syndrome, a rare CNS disorder, supported by a partnership that secured up to $9.2 million from the Rett Syndrome Research Trust.
  • AX-2911 is advancing toward clinical candidate selection, targeting MASH (a prevalent liver condition).
  • AX-1412 targets B4GALT1 for cardiovascular diseases.

The company's financial health is also supported by external validation, including a collaboration with Eli Lilly, which generated $2.0 million in milestone payments during the first nine months of 2025. The overall net loss for the nine-month period ending September 30, 2025, was € 33.3 million.

Program Target Indication Technology Application Key 2025/2026 Milestone
AX-0810 Cholestatic Liver Diseases (NTCP) Modulate Initial Safety/PK Data by year-end 2025
AX-2402 Rett Syndrome (MECP2) Correct/Modulate Phase 1/2 Trial planned for 2026
AX-2911 MASH Modulate Advancing toward clinical candidate selection in 2025
AX-1412 Cardiovascular Diseases (B4GALT1) Modulate Preclinical/Translational updates expected

The platform's ability to address both rare and prevalent diseases using a single, validated RNA editing approach is the central value proposition. If you're looking at the near-term catalysts, the first human data for AX-0810 in Q4 2025 is the key event that will validate the entire Axiomer technology in a clinical setting.

ProQR Therapeutics N.V. (PRQR) - Canvas Business Model: Customer Relationships

ProQR Therapeutics N.V. structures its external relationships around strategic alliances, patient-centric advocacy, and transparent communication with the financial community.

High-touch, collaborative relationships with major pharmaceutical partners

The relationship with Eli Lilly and Company (Lilly) is a cornerstone, representing a significant, long-term collaboration focused on the discovery, development, and commercialization of RNA editing medicines for genetic disorders. This partnership, which began in 2021 and was expanded in 2022, utilizes ProQR Therapeutics N.V.'s proprietary Axiomer™ RNA editing platform. The total potential value of this strategic collaboration is up to $3.9 billion. ProQR Therapeutics N.V. retains an option to expand the collaboration to a total of 15 targets, which would trigger a $50 million opt-in payment to ProQR Therapeutics N.V. The financial flow from this partnership directly supports operations, as evidenced by the $2.0 million (~€ 1.8 million) in milestone income earned by ProQR Therapeutics N.V. during the first nine months of 2025. Lilly also participated in the October 2024 financing, contributing to the $82.1 million in gross proceeds raised then.

Here's a snapshot of the financial interaction with the key pharmaceutical partner:

Metric Value/Detail
Collaboration Start Year 2021
Total Potential Collaboration Value $3.9 billion
Potential Opt-in Payment (Additional 5 Targets) $50 million
Milestone Income (First Nine Months of 2025) $2.0 million
Cash Reserves from Oct 2024 Financing (Lilly Participation) $82.1 million (Gross Proceeds)

Close engagement with patient advocacy groups like RSRT

Engagement with patient advocacy groups is critical, particularly for rare diseases like Rett Syndrome. ProQR Therapeutics N.V. has an active, expanded collaboration with the Rett Syndrome Research Trust (RSRT) to advance AX-2402, targeting the MECP2 R270X mutation. This relationship is structured as a co-investment model, where RSRT funding de-risks the program to incentivize full internal development by ProQR Therapeutics N.V. The total funding commitment from RSRT to date is $9.1 million. This total includes an initial $1 million research grant announced in January 2024, followed by an expansion in December 2024 with an additional $8.1 million in funding. ProQR Therapeutics N.V. is matching RSRT's investment in this program.

The RSRT partnership drives specific program milestones:

  • Total RSRT funding commitment: $9.1 million
  • Initial grant amount (Jan 2024): $1 million
  • Additional funding (Dec 2024): $8.1 million
  • Program focus: AX-2402 for Rett Syndrome

Investor relations and analyst events for transparency on data readouts

ProQR Therapeutics N.V. maintains active communication with investors and analysts to provide transparency, especially around clinical data milestones. The company hosted a virtual Analyst and Investor Event in November 2025, which detailed the Phase 1 trial design for AX-0810 and outlined expectations for 2025 data. Furthermore, management actively participated in several key industry and investor conferences in late 2025.

Investor engagement events in late 2025 included:

  • H.C. Wainwright Genetic Medicines Virtual Conference: October 14-15, 2025 (Presentation available on demand Oct 14 at 7:00 am ET)
  • H.C. Wainwright Liver Disease Virtual Conference: October 21-22, 2025 (Presentation available on demand Oct 21 at 7:00 am ET)
  • Chardan's 9th Annual Genetic Medicines Conference: October 21, 2025 (Included an RNA Editing panel at 1:00 p.m. ET)
  • Virtual Analyst and Investor Event (focused on AX-0810): November 2025
  • Evercore Healthcare Conference: December 01, 2025

Archived webcasts from these presentations are typically available for approximately 30 days following the presentation date on the ProQR Therapeutics N.V. website.

Specialized clinical support for trial participants

The relationship with trial participants is managed through rigorous, protocol-driven engagement, particularly as the lead program enters first-in-human testing. ProQR Therapeutics N.V. received Clinical Trial Application (CTA) authorization for AX-0810 and initiated the Phase 1 study in healthy volunteers. The trial is designed to assess specific parameters directly related to the therapeutic approach.

Key elements of the initial clinical engagement for AX-0810 include:

  • Trial Status (as of Q3 2025): Initiating Phase 1 study in healthy volunteers.
  • Assessment Focus: Safety, tolerability, pharmacokinetics (PK), and target engagement.
  • Data Timeline: Initial safety and PK data from Cohort 1 expected by year-end 2025.
  • Target Engagement Data: Expected in the first half of 2026 from all cohorts.

The company's cash position as of September 30, 2025, was € 106.9 million, providing a runway into mid-2027, which supports the ongoing operational commitment to these clinical programs.

ProQR Therapeutics N.V. (PRQR) - Canvas Business Model: Channels

You're looking at how ProQR Therapeutics N.V. gets its science and potential therapies in front of partners, regulators, and investors as of late 2025. It's a mix of formal agreements, clinical execution, and scientific dissemination. Honestly, the channels reflect a company deep in the clinical transition phase.

Direct licensing and collaboration agreements with pharmaceutical companies

The core of ProQR Therapeutics N.V.'s external commercial and development channel is through strategic alliances. The most significant is the $3.9 billion strategic collaboration with Eli Lilly and Company ("Lilly"), which started back in 2021 and saw expansion in 2022. This partnership is key for developing their Axiomer® RNA editing platform across multiple targets. ProQR Therapeutics N.V. is still executing on this, having recognized milestone income of $2.0 million through the first nine months of 2025 from Lilly.

Furthermore, the Lilly agreement includes an option for ProQR Therapeutics N.V. to expand to a total of 15 targets, which would trigger a substantial $50 million opt-in payment. On a smaller scale, the collaboration with the Rett Syndrome Research Trust ("RSRT") was expanded in December 2024, securing an additional funding for a total commitment of $9.2 million to support the advancement of AX-2402.

You should also note the historical licensing activity, such as the agreement signed in October 2018 with Ionis Pharmaceuticals for QR-1123, which involved upfront payments totaling $6,001,000 (made in shares) and includes future milestone payments plus royalties of 20% on net sales. To be fair, one older CNS license agreement was terminated as of July 2023.

Clinical trial sites in Europe and North America for drug testing

The primary channel for testing drug candidates like AX-0810 is through formal clinical trials. ProQR Therapeutics N.V. advanced its lead program by submitting a Clinical Trial Application (CTA) to the European Medicines Agency (EMA) in June 2025. Pending regulatory clearance, the first-in-human Phase 1 study for AX-0810 is expected to commence at a single site in the Netherlands. This is the first clinical development milestone for their Axiomer™ platform.

While the initial trial is in Europe, the target patient populations for their lead programs are significant across both regions. For instance, Primary Sclerosing Cholangitis (PSC) and Biliary Atresia (BA) are estimated to affect 80,000 and 20,000 individuals in North America and Europe, respectively. The company maintains a U.S. office in Cambridge, MA, which supports North American operations and engagement.

Here's a quick look at the financial context surrounding these clinical advancements as of late 2025:

Financial Metric (as of September 30, 2025) Amount (in millions) Context
Cash and Cash Equivalents € 106.9 million Provides runway into mid-2027.
Net Cash Used in Operating Activities (9 months 2025) € 39.4 million Reflects ongoing R&D and clinical spend.
Research & Development (R&D) Costs (9 months 2025) € 34.8 million Significant investment into pipeline advancement.

Scientific publications and conferences to showcase platform data

Disseminating platform data is a crucial channel for building scientific credibility and attracting future partners or investment. ProQR Therapeutics N.V. actively uses scientific venues to present its Axiomer technology. You can see evidence of this through their participation in key 2025 events, including the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting and the Oligonucleotide and Peptide Therapeutics Conference (TIDES USA) in May, and the RNA Editing Summit in July.

The company also uses dedicated investor events to detail trial designs and data expectations. They hosted a virtual Analyst and Investor Event in the fall of 2025, which focused on the AX-0810 Phase 1 trial design. The company is positioned to deliver initial safety and PK data from the first cohort of the AX-0810 trial by the end of 2025, with target engagement data expected in the first half of 2026. They anticipate up to four clinical data readouts across 2025 and 2026.

  • Presentations included data on the Axiomer ADAR-mediated RNA editing platform.
  • Specific abstracts presented at ASGCT 2025 related to NTCP and PNPLA3.
  • The company plans to provide an update on AX-1412 in mid-2025.

Investor communications for capital market access

Access to capital markets is a direct channel for funding operations, especially given the net loss reported. ProQR Therapeutics N.V. ended the third quarter of 2025 with € 106.9 million in cash and cash equivalents, which supports a financial runway extending well into mid-2027. This follows a successful underwritten public offering in October 2024 that generated gross proceeds of $82.1 million.

The company's financial reporting itself serves as a primary communication channel. They reported a net loss of € 33.3 million for the nine-month period ending September 30, 2025. For ongoing capital market access, ProQR Therapeutics N.V. has several effective registration statements on Form F-3 on file with the SEC, including File No. 333-282419. The company also uses press releases to announce key operational and financial milestones, such as the Q3 2025 results on November 6, 2025.

The key financial metrics related to investor confidence and capital structure as of late 2025 are summarized below:

Financial Event/Data Point Date/Period Value
Gross Proceeds from Oct 2024 Offering October 2024 $82.1 million
Cash & Equivalents September 30, 2025 € 106.9 million
Net Loss Nine Months Ended Sept 30, 2025 € 33.3 million
Lilly Collaboration Milestone Income First 9 Months of 2025 $2.0 million

Finance: draft 13-week cash view by Friday.

ProQR Therapeutics N.V. (PRQR) - Canvas Business Model: Customer Segments

You're looking at the core groups ProQR Therapeutics N.V. is targeting with its Axiomer RNA editing platform as of late 2025. These segments are defined by the specific, high unmet medical need diseases their pipeline programs address, and the strategic partners needed to fund and commercialize the technology.

Large pharmaceutical companies seeking novel RNA editing technology represent a major segment, primarily through strategic alliances that provide non-dilutive funding and future commercial reach. The collaboration with Eli Lilly is a prime example of this segment engagement.

The company's financial health, which supports engaging these segments, showed cash and cash equivalents of approximately €106.9 million at September 30, 2025, providing runway into mid-2027.

Partner/Segment Type Program Focus Financial Metric/Value Data Point (as of late 2025)
Large Pharmaceutical Partner (Eli Lilly) Axiomer Platform Technology Total Collaboration Value $3.9 billion (Source 11)
Large Pharmaceutical Partner (Eli Lilly) Milestone Achievement Income YTD Q3 2025 $2.0 million (~€ 1.8 million) (Source 1, 6)
Large Pharmaceutical Partner (Eli Lilly) Potential Future Value Opt-in Payment for 5 Additional Targets $50 million (Source 2)

Patients with severe cholestatic liver diseases (NTCP target) are targeted by the lead program, AX-0810. This segment is critical as it represents the first clinical validation of the Axiomer platform.

  • Program: AX-0810, modulating NTCP to reduce bile acid accumulation.
  • Clinical Status: CTA authorization received in October 2025 (Source 1, 6).
  • Trial Location: Phase 1 study initiating at a single site in the Netherlands (Source 8).
  • Near-Term Readout: Initial safety, tolerability, and PK data for Cohort 1 expected by year-end 2025 (Source 1, 6).
  • Follow-up Readout: Target engagement data across cohorts expected in H1 2026 (Source 1, 6).

Patients with rare neurodevelopmental disorders like Rett Syndrome (MECP2 target) are addressed by the CNS program, AX-2402. This segment is characterized by a severe, rare genetic condition with high unmet need.

  • Program: AX-2402, targeting the R270X mutation in the MECP2 gene.
  • Patient Population Size: Rett Syndrome affects approximately 350,000 people worldwide (Source 9).
  • Targeted Subset: Mutations addressable by this RNA editing approach account for 40% of all Rett cases (Source 10).
  • Development Status: Advancing toward clinical candidate selection as of Q2 2025 (Source 8).

Patient advocacy and research foundations funding drug development are essential early-stage customers/supporters, particularly for rare disease programs where patient advocacy drives research funding.

The Rett Syndrome Research Trust (RSRT) is a key example of this segment providing direct, non-dilutive funding to advance the AX-2402 program.

  • Foundation Partner: Rett Syndrome Research Trust (RSRT).
  • Total Funding Secured: Up to $9.2 million to support advancement into clinical trials (Source 2).
  • Funding Context: This funding supports IND-enabling studies, with ProQR Therapeutics N.V. matching the investment (Source 10).

ProQR Therapeutics N.V. (PRQR) - Canvas Business Model: Cost Structure

You're looking at the core expenditures driving ProQR Therapeutics N.V.'s operations as of late 2025. For a development-stage biotech, the cost structure is almost entirely weighted toward getting the science through the clinic and protecting the underlying technology.

Dominant Research and Development (R&D) expenses are the clear cost leader. For the nine-month period ended September 30, 2025, R&D costs hit € 34.8 million. This is a significant jump from the € 25.7 million reported for the same period in 2024, showing the increased investment as programs mature. Net cash used in operating activities for those nine months was € 39.4 million, meaning R&D accounts for the vast majority of cash burn.

The primary driver for this elevated R&D spend is the Clinical trial costs for AX-0810 and preclinical advancement. ProQR Therapeutics N.V. achieved a major milestone with the CTA authorization and is initiating the first-in-human Phase 1 study for AX-0810, which targets NTCP for cholestatic diseases.

Here's a quick look at how the main operating costs stacked up for the first nine months of 2025:

Cost Category Amount (9M 2025) Comparison Point
Research and Development (R&D) € 34.8 million Up from € 25.7 million (9M 2024)
General and Administrative (G&A) € 11.2 million Up from € 9.7 million (9M 2024)
Total Operating Costs (Implied from Net Loss & Revenue) Approx. € 45.6 million Net Loss of € 33.3M + Revenue of € 11.2M (9M 2025)

The R&D expenditure covers several critical, high-cost activities:

  • Costs associated with the Phase 1 trial initiation for AX-0810.
  • Manufacturing and supply chain setup for clinical material.
  • Ongoing work supporting preclinical advancement of other pipeline assets, like AX-2402 for Rett Syndrome.
  • Platform costs related to the Axiomer RNA editing technology.

General and Administrative (G&A) costs were € 11.2 million for the nine-month period ending September 30, 2025. This reflects the necessary overhead to run a public company advancing clinical trials. This category includes things like executive salaries, finance, and legal functions.

A key, though often bundled, component of the G&A and R&D spend is Intellectual property maintenance and patent prosecution fees. You have to spend money to keep the Axiomer platform protected globally. This is a non-negotiable cost of doing business in this space, ensuring exclusivity for their novel RNA editing approach.

To be fair, some of the cost pressure is offset by partnership income. During the first nine months of 2025, ProQR Therapeutics N.V. achieved milestones from its collaboration agreement with Eli Lilly amounting to $2.0 million, which is roughly € 1.8 million. That income helps temper the overall cash burn, but the underlying operational costs remain high as they push AX-0810 toward initial data readouts by year-end 2025.

Finance: draft 13-week cash view by Friday.

ProQR Therapeutics N.V. (PRQR) - Canvas Business Model: Revenue Streams

You're looking at the core ways ProQR Therapeutics N.V. brings in cash right now, which is heavily weighted toward partnerships and non-dilutive funding as they advance their Axiomer RNA editing platform.

The most immediate, concrete revenue source comes from the strategic collaboration with Eli Lilly and Company (Lilly). During the nine-month period ended September 30, 2025, ProQR Therapeutics N.V. achieved milestones under this agreement amounting to exactly $2.0 million. This is a key near-term income driver while the pipeline matures.

Another vital component of ProQR Therapeutics N.V.'s funding structure involves non-dilutive capital from non-profit organizations. Specifically, the collaboration with the Rett Syndrome Research Trust (RSRT) has been expanded. The total funding secured from RSRT to support the advancement of AX-2402 is $9.1 million, which includes an initial grant of $1.0 million and an additional $8.1 million announced in late 2024.

The potential for significant future revenue is tied directly to the success of these partnerships, particularly the Lilly agreement. This collaboration includes an option for Lilly to expand to a total of 15 targets, which would trigger a substantial future opt-in payment to ProQR Therapeutics N.V. of $50 million.

The long-term revenue vision for ProQR Therapeutics N.V. rests on successful commercialization, which translates into future royalties and product sales once any of their pipeline candidates, like AX-0810, achieve regulatory approval and market entry. Still, as of late 2025, the company's reported revenue for the nine-month period ended September 30, 2025, was €11.22 million.

Here's a quick look at the reported revenue and cash position as of the end of Q3 2025:

Financial Metric Amount (9M 2025) Amount (Q3 2025)
Total Sales/Revenue €11.22 million EUR 2.88 million
Lilly Milestone Payments $2.0 million Not Specified
Cash & Cash Equivalents (as of Sep 30) €106.9 million €106.9 million
Net Cash Used in Operating Activities €39.4 million Not Specified

The revenue streams can be categorized by their source and expected timing:

  • Milestone Payments from Eli Lilly and Company
  • Research Grants from Non-Profit Entities (e.g., RSRT)
  • Future Opt-In Payments (e.g., $50 million from Lilly expansion)
  • Long-Term Royalties and Product Sales

You can see the RSRT funding is a significant non-dilutive source supporting specific program advancement:

  • RSRT Total Funding Secured: $9.1 million
  • RSRT Additional Funding (Dec 2024): $8.1 million
  • Initial RSRT Grant (Jan 2024): $1.0 million

Finance: review the Q4 2025 cash burn rate against the mid-2027 runway projection by next Tuesday.


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