LEIDEN, Netherlands & CAMBRIDGE, Mass., March 25, 2026 (GLOBE NEWSWIRE) -- 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 announced that it will host a virtual Investor and Analyst Event highlighting its pipeline entitled: “Expanding the Axiomer™ RNA Editing Opportunity Beyond AX-0810” Ahead of the anticipated target engagement data for AX-0810 in the first hal ...

Question-and-Answer SessionDennis HomChief Financial Officer Sure. So the company has evolved over time, as you alluded to. But since 2022, we've been exclusively focused on developing our Axiomer technology, which is our version of RNA editing. So the strategy we're taking is fairly straightforward. We're developing our own, wholly-owned pipeline of programs based on Axiomer and we continue to innovate on that technology. I would mention that we are the pioneers of RNA editing. So ADAR-mediated RNA editing ...

Core Insights - Korro Bio, Inc. is focused on developing RNA editing-based genetic medicines for rare and prevalent diseases, reporting significant progress in its clinical development and financial position for the year ending December 31, 2025 [1][2]. Financial Performance - As of December 31, 2025, Korro reported cash, cash equivalents, and marketable securities of $85.2 million, a decrease from $163.1 million in 2024, but expects funding to last into the second half of 2028 due to a recent $85 million private placement [6][23]. - Collaboration revenue increased to $6.4 million in 2025 from $2.3 million in 2024, attributed to a full year of collaboration with Novo Nordisk [7]. - Research and Development (R&D) expenses rose to $65.6 million in 2025 from $63.6 million in 2024, driven by increased costs related to KRRO-121 [8]. - General and Administrative (G&A) expenses decreased to $28.2 million in 2025 from $30.5 million in 2024, mainly due to lower professional fees [9]. - The net loss for 2025 was $117.3 million, compared to $83.6 million in 2024, primarily due to non-cash impairment charges [13][21]. Product Development - Korro nominated KRRO-121 as a development candidate for treating hyperammonemia in patients with urea cycle disorders (UCDs) and hepatic encephalopathy (HE), targeting a market opportunity exceeding $1 billion [4][5]. - KRRO-121 aims to stabilize glutamine synthase (GS) RNA, enhancing ammonia clearance in the liver and offering a more convenient subcutaneous delivery method compared to current therapies [5][16]. - The company is on track to nominate a development candidate for its GalNAc-conjugated oligonucleotide for alpha-1 antitrypsin deficiency (AATD) in the second quarter of 2026 [4][11]. Strategic Initiatives - Korro hosted a virtual analyst day to discuss the unmet medical needs in hyperammonemia and the scientific rationale for KRRO-121 [11]. - The company is advancing its GalNAc-conjugated oligonucleotide program, achieving over 90% in vivo RNA editing, indicating high therapeutic potential [11]. - Upcoming milestones include regulatory filing for KRRO-121 in the second half of 2026 and the nomination of a third GalNAc-conjugated program [11].

Core Insights - ProQR Therapeutics reported significant clinical advancements in 2025, particularly with AX-0810 for cholestatic diseases entering Phase 1 trials, and aims to provide target engagement data in the first half of 2026 [2][3] - The company has strengthened its pipeline with new development candidates AX-2402 for Rett syndrome and AX-2911 for MASH, alongside a successful collaboration with Eli Lilly, achieving $4.5 million in milestones [2][8] Financial Highlights - As of December 31, 2025, ProQR held cash and cash equivalents of approximately €92.4 million, down from €149.4 million in 2024 [11] - The net cash used in operating activities for 2025 was €52.8 million, compared to €36.4 million in 2024 [11] - The net loss for 2025 was €42.2 million, or €0.40 per diluted share, an increase from €27.8 million, or €0.32 per diluted share, in 2024 [12] Clinical Development - AX-0810 is currently in a Phase 1 multiple-dose escalation study in healthy volunteers, with initial safety and pharmacokinetic data reported [6] - The company plans to include a PSC patient cohort in the ongoing Phase 1 trial after completing the healthy volunteer cohorts [6] - AX-2402 has shown promising preclinical results in a Rett syndrome mouse model, with plans to initiate first-in-human clinical testing in the first half of 2027 [6][12] Pipeline Expansion - AX-2402 targets the R270X mutation in MECP2 to restore normal protein function for Rett syndrome [4] - AX-2911 aims to address the I148M mutation in PNPLA3, a significant genetic risk factor for fatty liver disease, demonstrating over 80% reduction in hepatic fat content in preclinical studies [5][7] Strategic Collaborations - The collaboration with Eli Lilly is expected to continue in 2026, with potential data updates and additional milestone payments anticipated [8]

Core Viewpoint - Korro Bio, Inc. has announced a private investment in public equity (PIPE) financing expected to generate approximately $85 million in gross proceeds, aimed at advancing its RNA editing platform and extending its cash runway into the second half of 2028 [1][4][9] Financing Details - The PIPE financing is led by Venrock Healthcare Capital Partners, with participation from both new and existing investors including ADAR1 Capital Management and New Enterprise Associates [2] - Korro is selling 4,501,928 shares of common stock at $11.11 per share and pre-funded warrants for 3,148,836 shares at $11.109 each, with an exercise price of $0.001 per share [3] Use of Proceeds - The net proceeds will support clinical development milestones for key pipeline assets, including KRRO-121 and the GalNAc-conjugated AATD program, and will also be used for working capital and general corporate purposes [4][9] - The funding will enable advancements in Korro's longevity and liver health program targeting the AMPKγ1 pathway [4] Company Overview - Korro Bio is focused on developing a new class of genetic medicines through RNA editing for both rare and prevalent diseases, utilizing its proprietary OPERA platform [8][10] - The company aims to enhance the precision and tunability of genetic medicines, potentially improving specificity and long-term tolerability [10]

Summary of Wave Life Sciences FY Conference Call Company Overview - **Company**: Wave Life Sciences (NasdaqGM: WVE) - **Focus**: RNA medicine and oligonucleotide optimization, with a strong emphasis on human clinical genetics and innovative chemistry [2][4] Key Points from 4Q Results - **Pipeline Development**: Significant advancements in the pipeline, particularly in obesity treatment with Inhibin E and RNA editing for alpha-1 antitrypsin deficiency [3][4] - **Clinical Data**: Upcoming clinical data expected for Inhibin E, including 240 mg six-month data and 400 mg three-month data [3][15] Obesity Treatment Insights - **Targeted Mechanism**: Inhibin E aims to address obesity by reducing visceral fat without significant loss of lean muscle mass, which is crucial for metabolic health [6][10][14] - **Clinical Feedback**: Key opinion leaders (KOLs) are interested in the mechanism that focuses on body composition rather than just weight loss [5][12] - **Initial Results**: At three months, the 240 mg dose showed nearly 10% reduction in visceral fat and a 0.9% increase in lean mass, indicating a favorable profile compared to existing therapies [13][19] Future Expectations - **Data Anticipation**: The 400 mg cohort data is expected to provide insights into dose dependency and time dependency of fat loss [17][19] - **Regulatory Considerations**: The company is confident in meeting FDA requirements for obesity drugs, focusing on both weight loss and body composition improvements [45][49] RNA Editing Program - **Alpha-1 Antitrypsin Deficiency**: The program aims to correct the deficiency by producing endogenous proteins, with potential for accelerated approval based on existing data [66][75] - **Regulatory Engagement**: Discussions with regulators are ongoing to establish a clear path for market entry and confirmatory studies [75][76] Strategic Insights - **Combination Therapies**: There is potential for Inhibin E to be used in combination with GLP-1 therapies, enhancing treatment options for obesity [52][54] - **Market Potential**: The company sees a significant opportunity in addressing the obesity epidemic, with a scalable and accessible treatment model [58][60] Innovations and Future Pipeline - **Bifunctional Modality**: The company is exploring bifunctional modalities that can target multiple pathways, enhancing therapeutic efficacy [78][80] - **Targeting Multiple Cell Types**: Future developments may include targeting both adipocytes and hepatocytes, broadening the therapeutic applications in cardiometabolic diseases [80] Conclusion - Wave Life Sciences is positioned at a critical juncture with innovative approaches to RNA medicine, particularly in obesity and genetic disorders. The upcoming clinical data and regulatory discussions will be pivotal in shaping the future of its therapies and market presence.

Financial Data and Key Metrics Changes - Revenue for Q4 2025 was $17.2 million, down from $83.7 million in the prior year quarter, and full-year revenue was $42.7 million compared to $108.3 million in 2024 [27] - The decrease in revenue was primarily due to the termination of the Takeda collaboration in October 2024, partially offset by increased revenue from the GSK collaboration [28] - Net loss for Q4 2025 was $53.2 million, compared to a net income of $29.3 million in the prior year quarter, and the full-year net loss was $204.4 million compared to a net loss of $97 million in 2024 [30] Business Line Data and Key Metrics Changes - The company is focusing on two main programs: WVE-007 for obesity and WVE-006 for alpha-1 antitrypsin deficiency (AATD) [5][15] - WVE-007 aims to improve body composition by reducing fat while preserving muscle, with promising interim data showing fat loss similar to semaglutide without muscle loss [10][22] - WVE-006 is advancing with a goal to restore M-AAT protein levels and reduce mutant Z-AAT, with expectations for regulatory feedback on an accelerated approval pathway in mid-2026 [17][25] Market Data and Key Metrics Changes - The obesity treatment market is evolving, with a need for novel mechanisms beyond current therapies like GLP-1s, which have dosing and tolerability challenges [7][14] - The company is targeting a significant population with obesity and AATD, with approximately 200,000 individuals in the U.S. and Europe affected by AATD and a large number of individuals living with obesity [15][18] Company Strategy and Development Direction - The company is strategically focused on accelerating the development of WVE-007 and advancing its RNA editing portfolio, with plans to initiate new clinical trials in 2026 [31] - There is a recognition of the need for innovative obesity therapies that can provide better outcomes in body composition and overall health [14][64] - The company aims to leverage its proprietary technology to differentiate its products in the competitive landscape of RNA medicines [10][19] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential of WVE-007 to shift the obesity treatment landscape with a favorable safety profile and dosing regimen [14][64] - The company anticipates continued positive momentum in 2026, with upcoming data from clinical trials expected to support its therapeutic claims [31] Other Important Information - The company ended 2025 with $602.1 million in cash and cash equivalents, sufficient to fund operations into the third quarter of 2028 [30] - The collaboration with GSK is expected to yield up to $2.8 billion in milestone payments and royalties, contributing to future financial stability [19] Q&A Session Summary Question: How closely does your regulatory strategy mirror a competitor's recent alignment with the FDA? - Management indicated that their approach aligns well with existing therapies for AATD and anticipates positive discussions with the FDA regarding a biomarker-based pathway [34][35] Question: Are there any changes to the expected data release timelines for the INLIGHT study? - Management confirmed that data is on track and will be provided in upcoming updates, emphasizing the unique opportunity for data accumulation due to the study's design [40][41] Question: What are the expectations for confirmatory trial requirements if an accelerated approval pathway is achieved? - Management stated that discussions with the agency will focus on establishing the right pathway for both lung and liver indications, aiming for an efficient development process [46][48] Question: What outcomes data are planned to minimize potential payer pushbacks? - Management highlighted the importance of demonstrating significant fat reduction and lean mass preservation as key indicators for regulatory approval and market acceptance [52][55] Question: What treatment delta is expected for the upcoming obesity trial? - Management noted that they expect substantial reductions in liver fat and improvements in body composition, with ongoing assessments to establish the efficacy of their treatments [70][71]

Financial Data and Key Metrics Changes - Revenue for Q4 2025 was $17.2 million, down from $83.7 million in the prior year quarter, and full year revenue was $42.7 million compared to $108.3 million in 2024, primarily due to the termination of the Takeda collaboration [25][26] - Research and development expenses increased to $52.8 million in Q4 2025 from $44.6 million in Q4 2024, and for the full year, R&D expenses were $182.8 million compared to $159.7 million in 2024 [25][26] - The net loss for Q4 2025 was $53.2 million, compared to a net income of $29.3 million in the prior year quarter, and the full year net loss was $204.4 million compared to a net loss of $97 million in 2024 [27] Business Line Data and Key Metrics Changes - The company is focusing on two main programs: WVE-007 for obesity and WVE-006 for alpha-1 antitrypsin deficiency (AATD), with significant advancements in both areas [5][14] - WVE-007 showed promising interim data with a placebo-adjusted 4% reduction in total fat and a 9.2% reduction in visceral fat after three months at the lowest therapeutic dose [21] - WVE-006 demonstrated the ability to restore M-AAT protein levels to therapeutically meaningful levels, with over 11 micromolar protein and greater than 50% editing achieved [16] Market Data and Key Metrics Changes - The obesity treatment market is evolving, with a shift towards therapies that focus on body composition rather than just weight loss, highlighting the need for innovative mechanisms beyond current options like GLP-1s [7][13] - The company aims to address the unmet needs in the AATD market, where current treatment options are limited, and there is a significant patient population in need of effective therapies [14][15] Company Strategy and Development Direction - The company is strategically focused on accelerating the development of WVE-007 and advancing its RNA editing portfolio, with plans to initiate new clinical trials in 2026 [28] - There is a recognition of the need for novel obesity mechanisms and therapeutics, with WVE-007 positioned as a potential game-changer in the obesity landscape due to its unique dosing regimen and safety profile [13][28] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing clinical trials and the potential for WVE-007 and WVE-006 to meet significant unmet medical needs, with expectations for regulatory feedback on accelerated approval pathways by mid-2026 [32][42] - The company is optimistic about the future, aiming to unlock the full potential of its RNA medicines pipeline to transform human health [28] Other Important Information - The company ended 2025 with $602.1 million in cash and cash equivalents, which is expected to fund operations into the third quarter of 2028 [27] - The collaboration with GSK is progressing, with potential milestone payments expected in 2026 and beyond [18] Q&A Session Summary Question: How closely does your regulatory strategy mirror a competitor's recent alignment with the FDA? - Management indicated that their regulatory strategy is aligned with existing therapies for AATD, focusing on a biomarker approach and anticipates positive discussions with the FDA [31][32] Question: Will there be data updates for the 400 mg multi and 600 mg single doses? - Management confirmed that data is on track and will be included in the next update, emphasizing the ongoing data collection from the study [36][37] Question: What are the expectations for confirmatory trial requirements for AATD? - Management stated that they aim to establish an accelerated approval pathway for AATD patients and will align with the agency on the necessary requirements [42][43] Question: What outcomes data are planned to minimize potential payer pushbacks for obesity treatment? - Management highlighted the importance of demonstrating significant fat loss and lean mass preservation to support regulatory pathways and address payer concerns [48][50] Question: What treatment delta is expected for MRI-PDFF in the upcoming obesity trial? - Management noted that there is a substantial opportunity for liver fat reduction and emphasized the importance of monitoring these outcomes in the context of MASH [65][66]

Financial Data and Key Metrics Changes - Revenue for Q4 2025 was $17.2 million, down from $83.7 million in the prior year quarter, and full-year revenue was $42.7 million compared to $108.3 million in 2024, primarily due to the termination of the Takeda collaboration [26][28] - Research and development expenses increased to $52.8 million in Q4 2025 from $44.6 million in Q4 2024, and for the full year, they rose to $182.8 million from $159.7 million in 2024, driven by advancing programs and compensation-related expenses [27] - The net loss for Q4 2025 was $53.2 million, compared to a net income of $29.3 million in the prior year quarter, and the full-year net loss was $204.4 million compared to a loss of $97 million in 2024 [28] Business Line Data and Key Metrics Changes - The company is focusing on two main programs: WVE-007 for obesity and WVE-006 for alpha-1 antitrypsin deficiency (AATD), with significant clinical advancements reported for both [4][13] - WVE-007 demonstrated a placebo-adjusted 4% reduction in total fat and a 9.2% reduction in visceral fat in the lowest therapeutic dose cohort, while preserving muscle mass [20] - WVE-006 has shown the ability to restore M-AAT protein levels and reduce mutant Z-AAT, with a goal of achieving therapeutic thresholds for AATD patients [16][24] Market Data and Key Metrics Changes - The obesity treatment market is evolving, with a shift towards therapies that focus on body composition rather than just weight loss, highlighting the potential of WVE-007 to address this need [5][12] - The company is also targeting the liver disease market with WVE-008, aimed at correcting the PNPLA3 I148M variant, which affects a significant number of individuals in the U.S. and Europe [17][24] Company Strategy and Development Direction - The company aims to accelerate the development of WVE-007 and its RNA editing portfolio, with a focus on innovative treatment options for obesity and AATD [4][29] - There is a strategic emphasis on leveraging proprietary technologies to enhance the efficacy and safety profiles of its RNA medicines, differentiating from competitors [9][12] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the clinical data supporting WVE-007 and WVE-006, anticipating positive regulatory feedback and potential accelerated approval pathways [34][36] - The company is optimistic about the future, expecting to unlock the full potential of its RNA medicines pipeline to transform human health [29] Other Important Information - The company ended 2025 with $602.1 million in cash and cash equivalents, expected to fund operations into Q3 2028, excluding potential future milestone payments from collaborations [28] - The collaboration with GSK continues to progress, with the potential for significant milestone payments and royalties [18] Q&A Session Summary Question: How does the regulatory strategy for AATD align with competitors? - Management indicated alignment with FDA feedback on biomarker-driven pathways for AATD, emphasizing their unique approach to measuring therapeutic activity through MAAT protein levels [32][34] Question: Will data for the 400 mg multi-dose and 600 mg single-dose cohorts be available? - Management confirmed that data is on track and will be provided in upcoming updates, emphasizing the unique opportunity for data accumulation due to the study design [37][40] Question: What are the expectations for confirmatory trial requirements for AATD? - Management highlighted the importance of establishing a regulatory pathway for AATD patients, focusing on both lung and liver indications [44][45] Question: What outcomes data are planned to minimize payer pushbacks for obesity treatment? - Management discussed the importance of demonstrating fat loss and lean mass preservation, with a focus on overall body composition improvements as critical for regulatory approval [49][53] Question: What are the expectations for upcoming obesity and AATD readouts? - Management outlined expectations for fat loss and AAT levels that would indicate therapeutic success, emphasizing the importance of maintaining lean mass and achieving therapeutic thresholds [84][88]

Company Overview - Korro Bio, Inc. is a biopharmaceutical company focused on developing a new class of genetic medicines based on RNA editing for both rare and highly prevalent diseases [2] - The company aims to create a portfolio of differentiated programs that utilize the body's natural RNA editing process, allowing for precise and transient single base edits [2] - By editing RNA instead of DNA, Korro seeks to enhance the precision and tunability of genetic medicines, potentially leading to increased specificity and improved long-term tolerability [2] Technology and Approach - Korro employs an oligonucleotide-based approach to deliver its medicines, leveraging its proprietary platform along with established delivery modalities and manufacturing expertise [2] - The company plans to utilize established regulatory pathways of approved oligonucleotide drugs to bring its medicines to patients [2] Upcoming Events - Ram Aiyar, Ph.D., the CEO and President of Korro, will present at the TD Cowen 46 Annual Health Care Conference in Boston, MA, on March 3, 2026, at 11:10 a.m. ET [1]