Centessa Pharmaceuticals(CNTA) - 2025 Q2 - Quarterly Report
2025-08-12 11:13
Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q CENTESSA PHARMACEUTICALS PLC (Exact name of registrant as specified in its charter) England and Wales 98-1612294 (State or other jurisdiction of incorporation or organization) (Mark One) x QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2025 OR o TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1 ...
Zenas BioPharma, Inc.(ZBIO) - 2025 Q2 - Quarterly Results
2025-08-12 11:13
Exhibit 99.1 Zenas BioPharma Reports Second Quarter 2025 Financial Results and Provides Corporate Updates - Topline results from pivotal Phase 3 INDIGO trial in Immunoglobulin G4-Related Disease expected around year-end 2025 - - Completed enrollment of Phase 2 MoonStone trial in Relapsing Multiple Sclerosis; results expected early in the fourth quarter 2025 - - Enrollment of Phase 2 SunStone trial in Systemic Lupus Erythematosus expected to be completed by year-end 2025; topline results expected mid-2026 - ...
FREQUENCY THERAP(FREQ) - 2025 Q2 - Quarterly Report
2025-08-12 11:10
UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2025 ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from _____________________ to _____________________ Commission File Number: 001-39062 Korro Bio, Inc. (Exact name of registrant as specified in its charter) Delaware 4 ...
Korro Bio(KRRO) - 2025 Q2 - Quarterly Report
2025-08-12 11:10
[PART I. FINANCIAL INFORMATION](index=7&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) [Financial Statements (Unaudited)](index=7&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) This section presents the unaudited condensed consolidated financial statements for the periods ended June 30, 2025, including balance sheets, statements of operations, stockholders' equity, and cash flows, with accompanying notes [Condensed Consolidated Balance Sheets](index=7&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Total assets decreased to **$180.4 million** by June 30, 2025, from **$226.2 million** at year-end 2024, driven by reduced cash and securities, while the accumulated deficit grew to **$315.7 million** Condensed Consolidated Balance Sheet Highlights (in thousands) | Metric | June 30, 2025 | December 31, 2024 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $33,612 | $55,643 | | Total current assets | $102,634 | $131,532 | | Total assets | $180,425 | $226,240 | | **Liabilities & Stockholders' Equity** | | | | Total liabilities | $65,322 | $65,825 | | Accumulated deficit | $(315,743) | $(266,586) | | Total stockholders' equity | $115,103 | $160,415 | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=8&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) Collaboration revenue reached **$4.0 million** for the six months ended June 30, 2025, but increased research and development expenses led to a higher net loss of **$49.2 million** Statement of Operations Summary (Six Months Ended June 30, in thousands) | Metric | 2025 | 2024 | | :--- | :--- | :--- | | Collaboration revenue | $4,010 | $— | | Research and development | $40,770 | $30,710 | | General and administrative | $15,462 | $14,868 | | Loss from operations | $(52,222) | $(45,578) | | Net loss | $(49,157) | $(41,383) | | Net loss per share | $(5.24) | $(4.87) | [Condensed Consolidated Statements of Stockholders' Equity](index=9&type=section&id=Condensed%20Consolidated%20Statements%20of%20Stockholders'%20Equity) Total stockholders' equity decreased from **$160.4 million** at December 31, 2024, to **$115.1 million** at June 30, 2025, primarily due to the period's net loss - Total stockholders' equity decreased from **$160.4 million** at December 31, 2024, to **$115.1 million** at June 30, 2025, mainly due to the net loss incurred during the period[23](index=23&type=chunk) [Condensed Consolidated Statements of Cash Flows](index=10&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Net cash used in operating activities was **$43.7 million** for H1 2025, while investing activities provided **$21.5 million**, a significant shift from the **$108.0 million** used in H1 2024 Cash Flow Summary (Six Months Ended June 30, in thousands) | Activity | 2025 | 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | $(43,680) | $(36,484) | | Net cash provided by (used in) investing activities | $21,481 | $(108,004) | | Net cash provided by financing activities | $177 | $67,695 | [Notes to Unaudited Condensed Consolidated Financial Statements](index=11&type=section&id=Notes%20to%20Unaudited%20Condensed%20Consolidated%20Financial%20Statements) Notes confirm **$119.6 million** in cash and securities is sufficient for 12 months, detail **$4.0 million** in Novo Nordisk collaboration revenue, and disclose a May 2025 restructuring with a **19% workforce reduction** - The company believes its existing cash, cash equivalents, and marketable securities of **$119.6 million** as of June 30, 2025, are sufficient to fund planned operations for at least 12 months from the issuance date of the financial statements[31](index=31&type=chunk) - Under the collaboration agreement with Novo Nordisk, the company recognized **$4.0 million** in revenue for the six months ended June 30, 2025, with the total transaction price for the first program target estimated at **$39.9 million**[68](index=68&type=chunk) - On May 7, 2025, the company initiated a strategic plan that included a **19% workforce reduction** (21 positions), resulting in a one-time severance charge of **$1.2 million** during the second quarter[86](index=86&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=24&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's clinical-stage focus on RNA editing, with lead candidate KRRO-110 in Phase 1/2a trials, and highlights a **$10.1 million** increase in H1 2025 R&D expenses, confirming a cash runway into 2027 - The company's lead candidate, KRRO-110 for Alpha-1 Antitrypsin Deficiency (AATD), is in a Phase 1/2a clinical trial (REWRITE), with interim data from single ascending doses expected in the second half of 2025[92](index=92&type=chunk) Research and Development Expenses (Six Months Ended June 30, in thousands) | Expense Category | 2025 | 2024 | Change | | :--- | :--- | :--- | :--- | | KRRO-110 (AATD) external expenses | $12,557 | $9,222 | $3,335 | | Other research & pre-development | $9,695 | $6,272 | $3,423 | | Personnel expenses | $13,018 | $9,551 | $3,467 | | **Total R&D Expenses** | **$40,770** | **$30,710** | **$10,060** | - As of June 30, 2025, the company had **$119.6 million** in cash, cash equivalents, and marketable securities, which is expected to be sufficient to fund operating expenses and capital expenditure requirements into 2027[122](index=122&type=chunk) - Net cash used in operating activities increased to **$43.7 million** for the six months ended June 30, 2025, from **$36.5 million** in the prior-year period, primarily due to a higher net loss and changes in operating assets and liabilities[129](index=129&type=chunk)[130](index=130&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=38&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company's primary market risk is interest rate sensitivity on its **$119.6 million** cash and securities portfolio, which management deems immaterial, with foreign currency risk also considered minimal - The company's main market risk is interest rate sensitivity on its portfolio of cash, cash equivalents, and marketable securities, valued at **$119.6 million** as of June 30, 2025[139](index=139&type=chunk) - Management believes that an immediate **10% change** in market interest rates would not have a material effect on the fair market value of its cash and investments[139](index=139&type=chunk) - Exposure to foreign currency risk is considered minimal as operations are primarily in the U.S. and contracts in foreign currencies are of short duration[140](index=140&type=chunk) [Controls and Procedures](index=38&type=section&id=Item%204.%20Controls%20and%20Procedures) Management, including the CEO and CFO, confirmed the effectiveness of disclosure controls and procedures as of June 30, 2025, with no material changes to internal control over financial reporting during the quarter - Management, with the participation of the CEO and CFO, evaluated the company's disclosure controls and procedures and concluded they were effective at the reasonable assurance level as of June 30, 2025[142](index=142&type=chunk) - No changes in internal control over financial reporting occurred during the most recently completed fiscal quarter that have materially affected, or are reasonably likely to materially affect, these controls[143](index=143&type=chunk) [PART II. OTHER INFORMATION](index=39&type=section&id=PART%20II.%20OTHER%20INFORMATION) [Legal Proceedings](index=39&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently involved in any material legal proceedings expected to have a material adverse effect on its business - As of the report date, the company is not party to any claim or litigation that is expected to have a material adverse effect on its business[145](index=145&type=chunk) [Risk Factors](index=39&type=section&id=Item%201A.%20Risk%20Factors) Key risks include a history of significant losses, the unproven nature of its novel RNA editing technology, early-stage clinical development, and substantial risks related to regulatory approval, manufacturing, competition, and intellectual property - The company has a history of significant operating losses, with an accumulated deficit of **$315.7 million** as of June 30, 2025, and expects to incur losses for the foreseeable future[147](index=147&type=chunk) - RNA editing is a novel technology with limited clinical validation, and the company's approaches are unproven and may never lead to marketable products[174](index=174&type=chunk) - The company has only dosed a few participants in its first clinical trial for KRRO-110 and has not completed any clinical trials, with favorable preclinical results not predictive of clinical trial outcomes[156](index=156&type=chunk) - The company faces risks related to obtaining and protecting its intellectual property, including the possibility that patents may not be granted, may be challenged, or may not provide sufficient protection against competitors[300](index=300&type=chunk)[303](index=303&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=127&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) This item is not applicable for the reporting period - Not applicable[404](index=404&type=chunk) [Defaults Upon Senior Securities](index=127&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) This item is not applicable for the reporting period - Not applicable[405](index=405&type=chunk) [Mine Safety Disclosures](index=127&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This item is not applicable for the reporting period - Not applicable[406](index=406&type=chunk) [Other Information](index=127&type=section&id=Item%205.%20Other%20Information) Several executive officers adopted Rule 10b5-1 trading plans during Q2 2025 for the potential sale of shares issuable upon stock option exercise - During the three months ended June 30, 2025, CEO Ram Aiyar, COO Todd Chappell, CSO Loïc Vincent, and General Counsel Jeffrey Cerio each adopted a Rule 10b5-1 plan for the potential sale of common stock issuable upon the exercise of stock options[407](index=407&type=chunk) [Exhibits](index=128&type=section&id=Item%206.%20Exhibits) This section lists all exhibits filed with the Form 10-Q, including corporate governance documents and officer certifications required by the Sarbanes-Oxley Act - The report includes a list of filed exhibits, such as corporate governance documents and officer certifications required under the Sarbanes-Oxley Act[409](index=409&type=chunk)
Climb Bio, Inc(CLYM) - 2025 Q2 - Quarterly Results
2025-08-12 11:10
[Company Overview and Q2 2025 Highlights](index=1&type=section&id=Company%20Overview%20and%20Q2%202025%20Highlights) Climb Bio's Q2 2025 highlights include significant clinical trial advancements for budoprutug and CLYM116, alongside key leadership appointments, setting the stage for future data readouts [CEO Statement and Strategic Focus](index=1&type=section&id=CEO%20Statement) Climb Bio's CEO highlighted the company's focused execution, team building, and pipeline advancement, particularly noting patient dosing in ITP and SLE trials, regulatory clearance for pMN and subcutaneous budoprutug trials, and significant progress with CLYM116, setting the stage for a data-rich period - Patients have been dosed in both the ITP and SLE clinical trials of budoprutug, an anti-CD19 monoclonal antibody[3](index=3&type=chunk) - Regulatory clearance was achieved for the pMN Phase 2 trial and the Phase 1 trial of subcutaneous budoprutug in healthy volunteers, both expected to begin shortly[3](index=3&type=chunk) - CLYM116, a differentiated anti-APRIL antibody, is making exciting progress and is believed to be a best-in-class treatment for IgAN and other indications[3](index=3&type=chunk) - An investor event is planned for **September 2025** to showcase the CLYM116 program, share supportive preclinical data, and discuss IgAN development plans[3](index=3&type=chunk) [Second Quarter 2025 and Recent Highlights](index=1&type=section&id=Second%20Quarter%202025%20and%20Recent%20Highlights) The second quarter of 2025 saw significant advancements in Climb Bio's clinical pipeline, including the initiation of trials for budoprutug in ITP and SLE, progress towards a pMN trial, and the development of a subcutaneous formulation. The CLYM116 program also advanced towards clinical development, alongside a key corporate leadership appointment - Phase 1b/2a immune thrombocytopenia (ITP) trial and Phase 1b systemic lupus erythematosus (SLE) trial for budoprutug achieved First Patient In (FPI) and enrollment is ongoing[4](index=4&type=chunk) - Phase 2 primary membranous nephropathy (pMN) trial for budoprutug is on track to initiate in the coming weeks[4](index=4&type=chunk) - Budoprutug subcutaneous (SC) formulation demonstrated high bioavailability and favorable tolerability in non-clinical studies; a Phase 1 trial in healthy volunteers is expected to initiate soon, with initial results anticipated in the **first half of 2026**[5](index=5&type=chunk) - CLYM116 is advancing toward clinical development for IgA nephropathy (IgAN), with an IND or CTA submission expected in the **second half of 2025**[6](index=6&type=chunk) - Edgar D. Charles, M.D., MSc was appointed Chief Medical Officer in **June 2025**[7](index=7&type=chunk) [Pipeline Program Updates](index=1&type=section&id=Pipeline%20Program%20Updates) Climb Bio's pipeline is advancing with budoprutug trials in ITP, SLE, and pMN, a new subcutaneous formulation, and CLYM116 progressing towards clinical development for IgAN [Budoprutug Program Updates](index=1&type=section&id=Budoprutug%20Program%20Updates) Climb Bio provided updates on its budoprutug program, an anti-CD19 monoclonal antibody, detailing the ongoing clinical trials for ITP and SLE, the imminent initiation of a pMN trial, and the successful non-clinical development of a subcutaneous formulation [Immune Thrombocytopenia (ITP) Trial](index=1&type=section&id=Phase%201b%2F2a%20immune%20thrombocytopenia%20(ITP)%20trial) The Phase 1b/2a clinical trial for budoprutug in ITP patients is ongoing, evaluating safety, pharmacokinetics, and preliminary efficacy including B cell depletion and platelet counts - The Phase 1b/2a open-label, dose-escalation clinical trial of budoprutug in ITP patients has achieved FPI and enrollment is ongoing[4](index=4&type=chunk) - The trial evaluates safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy, including B cell depletion and platelet counts[4](index=4&type=chunk) [Systemic Lupus Erythematosus (SLE) Trial](index=1&type=section&id=Phase%201b%20systemic%20lupus%20erythematosus%20(SLE)%20trial) The Phase 1b clinical trial for budoprutug in SLE patients is enrolling, assessing safety, pharmacokinetics, and preliminary efficacy, including B cell depletion and autoantibody levels - The Phase 1b open-label, dose-escalation clinical trial of budoprutug in SLE patients has achieved FPI and enrollment is ongoing[4](index=4&type=chunk) - The trial evaluates safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy, including B cell depletion, autoantibody levels, and clinical activity[4](index=4&type=chunk) [Primary Membranous Nephropathy (pMN) Trial](index=1&type=section&id=Phase%202%20primary%20membranous%20nephropathy%20(pMN)%20trial) The Phase 2 pMN trial for budoprutug is set to begin soon, evaluating safety, pharmacokinetics, pharmacodynamics, and preliminary efficacy, including remission rates - The Phase 2 pMN trial, an open-label, dose-ranging study, is on track to initiate in the coming weeks[4](index=4&type=chunk) - The trial is designed to evaluate safety, pharmacokinetics, pharmacodynamics (including B cell depletion and anti-PLA2R antibody levels), and preliminary efficacy (including complete and partial remission)[4](index=4&type=chunk) [Subcutaneous (SC) Formulation Development](index=2&type=section&id=Continued%20development%20of%20subcutaneous%20(SC)%20formulation) Budoprutug's subcutaneous formulation shows high bioavailability and favorable tolerability in non-clinical data, with a Phase 1 trial in healthy volunteers expected to start soon - Supportive non-clinical data for budoprutug SC formulation demonstrated high bioavailability and favorable tolerability[5](index=5&type=chunk) - A Clinical Trial Application was cleared in Australia in August, and a Phase 1 trial in healthy volunteers is expected to initiate in the coming weeks[5](index=5&type=chunk) - Initial Phase 1 trial results for the budoprutug SC formulation are expected in the **first half of 2026**[5](index=5&type=chunk) [CLYM116 Program Updates](index=2&type=section&id=CLYM116%20Program%20Updates) Climb Bio's CLYM116 program, an anti-APRIL antibody for IgA nephropathy (IgAN), is progressing towards clinical development with an IND or CTA submission anticipated in the second half of 2025, complemented by an investor event in September 2025 to share preclinical data - CLYM116, an antibody targeting the APRIL pathway for IgA nephropathy (IgAN), is advancing toward clinical development[6](index=6&type=chunk) - Climb Bio remains on track to submit an Investigational New Drug (IND) or Clinical Trial Application (CTA) for CLYM116 in IgAN in the **second half of 2025**[6](index=6&type=chunk) - A CLYM116-focused investor event is planned for **September 2025** to share preclinical data, including head-to-head data in nonhuman primates with a first-generation anti-APRIL[6](index=6&type=chunk) [Corporate Developments](index=2&type=section&id=Corporate%20Developments) Climb Bio strengthened its leadership with the appointment of a new Chief Medical Officer, bringing extensive experience in immunology-focused pharmaceutical development [Chief Medical Officer Appointment](index=2&type=section&id=Appointed%20Edgar%20D.%20Charles%2C%20M.D.%2C%20MSc%20as%20Chief%20Medical%20Officer) Climb Bio strengthened its leadership team by appointing Edgar D. Charles, M.D., MSc as Chief Medical Officer in June 2025, leveraging his extensive experience in immunology-focused pharmaceutical development from previous roles at Bristol Myers Squibb and Merck & Co - Edgar D. Charles, M.D., MSc was appointed Chief Medical Officer in **June 2025**[7](index=7&type=chunk) - Dr. Charles brings over **20 years** of experience in immunology-focused pharmaceutical development, academic research, and clinical medicine[7](index=7&type=chunk) - He previously held significant clinical roles at Bristol Myers Squibb (BMS), leading immunology global program organization, and at Merck & Co., leading global clinical development programs[7](index=7&type=chunk) [Anticipated Milestones & Financial Summary](index=2&type=section&id=Anticipated%20Milestones%20and%20Financial%20Results%20Summary) Climb Bio anticipates key clinical and regulatory milestones for budoprutug and CLYM116 in H2 2025 and H1 2026, supported by a strong cash position extending through 2027 [Anticipated Milestones](index=2&type=section&id=Anticipated%20Milestones) Climb Bio outlined key upcoming milestones for its budoprutug and CLYM116 programs, including trial initiations, data readouts, and regulatory submissions, primarily concentrated in the second half of 2025 and first half of 2026 - Budoprutug (anti-CD19 monoclonal antibody) milestones[11](index=11&type=chunk) - pMN Phase 2 study – first patient in (**H2 2025**)[11](index=11&type=chunk) - Company to provide guidance on the anticipated timing of clinical readouts in SLE and ITP (**H2 2025**)[11](index=11&type=chunk) - Subcutaneous formulation Phase 1 clinical trial in healthy volunteers – first patient in (**H2 2025**) and initial results (**H1 2026**)[11](index=11&type=chunk) - CLYM116 (anti-APRIL monoclonal antibody) milestones[11](index=11&type=chunk) - Reporting preclinical data (**September 2025**) and submission of IND or CTA in IgAN (**H2 2025**)[11](index=11&type=chunk) - Investor event on CLYM116 to be held in **September 2025**[11](index=11&type=chunk) [Second Quarter 2025 Financial Highlights](index=2&type=section&id=Second%20Quarter%202025%20Financial%20Results) Climb Bio reported a strong cash position expected to fund operations through 2027, alongside increased R&D and G&A expenses for Q2 2025 compared to the prior year, while net loss significantly decreased due to the absence of acquired IPR&D expenses - Cash, cash equivalents and marketable securities were **$187.4 million** as of **June 30, 2025**, expected to fund operations through **2027**[11](index=11&type=chunk) Operating Expenses and Net Loss (Three Months Ended June 30) | Metric | Q2 2025 (in thousands) | Q2 2024 (in thousands) | Change (YoY) | | :-------------------------------- | :--------------------- | :--------------------- | :------------- | | Research and Development (R&D) | $6,575 | $1,046 | +$5,529 | | Acquired In-Process R&D | $0 | $51,659 | -$51,659 | | General and Administrative (G&A) | $4,102 | $3,667 | +$435 | | Other income, net | $2,011 | $1,483 | +$528 | | Net Loss | $(8,666) | $(54,889) | +$46,223 | | Net loss per share, basic and diluted | $(0.13) | $(1.81) | +$1.68 | [About Climb Bio and Product Information](index=2&type=section&id=About%20Climb%20Bio%2C%20Inc.) Climb Bio is a clinical-stage biotech company focused on immune-mediated diseases, developing budoprutug (anti-CD19) and CLYM116 (anti-APRIL) with distinct mechanisms of action [Company Profile](index=2&type=section&id=About%20Climb%20Bio%2C%20Inc.%20(Company%20Description)) Climb Bio, Inc. is a clinical-stage biotechnology company dedicated to developing therapeutics for patients with immune-mediated diseases, with a pipeline featuring budoprutug and CLYM116 - Climb Bio, Inc. is a clinical-stage biotechnology company developing therapeutics for patients with immune-mediated diseases[10](index=10&type=chunk) - The company's pipeline includes budoprutug, an anti-CD19 monoclonal antibody, and CLYM116, an anti-APRIL monoclonal antibody[10](index=10&type=chunk) [About Budoprutug](index=4&type=section&id=About%20Budoprutug) Budoprutug is a clinical-stage anti-CD19 monoclonal antibody designed to deplete B cells, targeting a broad range of B-cell mediated immune diseases, with ongoing trials in pMN, ITP, and SLE, and a subcutaneous formulation under development to enhance patient access - Budoprutug is a clinical-stage, anti-CD19 monoclonal antibody developed to address a broad range of B-cell mediated, immune-driven diseases[12](index=12&type=chunk) - It targets and depletes CD19-expressing B cells, including plasma blasts, which are key sources of pathogenic autoantibodies[12](index=12&type=chunk) - Climb Bio plans to evaluate budoprutug in multiple clinical trials across three lead indications: primary membranous nephropathy (pMN), immune thrombocytopenia (ITP), and systemic lupus erythematosus (SLE)[12](index=12&type=chunk) - Early clinical data suggest durable B-cell depletion, rapid reductions in autoantibodies, and clinical remission in pMN[12](index=12&type=chunk) - A subcutaneous formulation is in development to enable broader patient access and potential home-based dosing, and it has been granted orphan drug designation by the FDA for pMN[12](index=12&type=chunk) [About CLYM116](index=4&type=section&id=About%20CLYM116) CLYM116 is a preclinical-stage anti-APRIL monoclonal antibody with a novel pH-dependent mechanism, designed for potent, durable inhibition of APRIL with less frequent dosing, primarily being advanced for IgA nephropathy (IgAN) with potential for broader utility - CLYM116 is a preclinical-stage monoclonal antibody targeting APRIL (A Proliferation-Inducing Ligand), a key driver of pathogenic B-cell activity in autoimmune diseases[13](index=13&type=chunk) - It employs a novel pH-dependent bind-and-release mechanism to potently block APRIL signaling, promote lysosomal degradation of APRIL, and recycle the antibody to extend its half-life[13](index=13&type=chunk) - This differentiated design offers the potential for rapid, deep, and durable inhibition of APRIL with a favorable safety profile and less frequent dosing[13](index=13&type=chunk) - CLYM116 is being advanced for the treatment of IgA nephropathy (IgAN), with plans to initiate a Phase 1 clinical trial following IND-enabling studies and regulatory clearance[13](index=13&type=chunk) [Financial Statements](index=6&type=section&id=Financial%20Statements) Climb Bio's financial statements for Q2 2025 reflect a reduced net loss compared to the prior year, primarily due to the absence of acquired IPR&D expenses, alongside a decrease in cash and total assets [Condensed Consolidated Balance Sheets](index=6&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) The condensed consolidated balance sheets show Climb Bio's financial position as of June 30, 2025, reflecting a decrease in cash, cash equivalents, and marketable securities, leading to a reduction in total assets and stockholders' equity compared to December 31, 2024, while total liabilities slightly increased Condensed Consolidated Balance Sheet Highlights (in thousands) | Metric | June 30, 2025 | December 31, 2024 | Change | | :-------------------------------- | :------------ | :---------------- | :----- | | Cash, cash equivalents and marketable securities | $187,405 | $212,529 | -$25,124 | | Other assets | $4,981 | $4,658 | +$323 | | Total assets | $192,386 | $217,187 | -$24,801 | | Liabilities | $6,622 | $5,306 | +$1,316 | | Total stockholders' equity | $185,764 | $211,881 | -$26,117 | | Total liabilities and stockholders' equity | $192,386 | $217,187 | -$24,801 | [Condensed Consolidated Statements of Operations](index=6&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations) Climb Bio's statements of operations for Q2 2025 show a significant decrease in net loss compared to Q2 2024, primarily due to the absence of a large acquired in-process R&D expense from the prior year, despite an increase in ongoing R&D and G&A expenses Condensed Consolidated Statements of Operations (Three Months Ended June 30, in thousands, except per share amounts) | Metric | 2025 | 2024 | Change (YoY) | | :-------------------------------- | :--- | :--- | :----------- | | Research and development | $6,575 | $1,046 | +$5,529 | | Acquired in-process research and development | $0 | $51,659 | -$51,659 | | General and administrative | $4,102 | $3,667 | +$435 | | Total operating expenses | $10,677 | $56,372 | -$45,695 | | Loss from operations | $(10,677) | $(56,372) | +$45,695 | | Other income, net | $2,011 | $1,483 | +$528 | | Net loss | $(8,666) | $(54,889) | +$46,223 | | Net loss per share, basic and diluted | $(0.13) | $(1.81) | +$1.68 | Condensed Consolidated Statements of Operations (Six Months Ended June 30, in thousands, except per share amounts) | Metric | 2025 | 2024 | Change (YoY) | | :-------------------------------- | :--- | :--- | :----------- | | Research and development | $23,902 | $2,137 | +$21,765 | | Acquired in-process research and development | $0 | $51,659 | -$51,659 | | General and administrative | $9,793 | $5,581 | +$4,212 | | Total operating expenses | $33,695 | $59,377 | -$25,682 | | Loss from operations | $(33,695) | $(59,377) | +$25,682 | | Other income, net | $4,248 | $2,791 | +$1,457 | | Net loss | $(29,447) | $(56,586) | +$27,139 | | Net loss per share, basic and diluted | $(0.44) | $(1.95) | +$1.51 | [Legal and Contact Information](index=4&type=section&id=Legal%20and%20Contact%20Information) This section provides important disclaimers regarding forward-looking statements, highlighting inherent risks and uncertainties, and offers contact details for investor and media inquiries [Forward-Looking Statements](index=4&type=section&id=Forward-Looking%20Statements) This section serves as a cautionary note, indicating that the press release contains forward-looking statements subject to various risks and uncertainties that could cause actual results to differ materially from expectations, and Climb Bio disclaims any obligation to update these statements unless legally required - The press release contains "forward-looking statements" regarding future expectations, plans, and prospects for Climb Bio, including therapeutic benefits, clinical development, trial timelines, and financial resources[14](index=14&type=chunk) - These statements are subject to risks and uncertainties that could cause actual results to differ materially, including those related to acquisitions, regulatory changes, competition, intellectual property, expense management, and capital raising[14](index=14&type=chunk) - Climb Bio specifically disclaims any obligation to update these forward-looking statements, except as required by law[14](index=14&type=chunk) [Investors and Media Contact](index=4&type=section&id=Investors%20and%20Media) Contact information is provided for investors and media inquiries, directing them to Carlo Tanzi, Ph.D. at Kendall Investor Relations - For investor and media inquiries, contact Carlo Tanzi, Ph.D. at Kendall Investor Relations[15](index=15&type=chunk) - Email: ctanzi@kendallir.com[15](index=15&type=chunk)
Eliem Therapeutics(ELYM) - 2025 Q2 - Quarterly Results
2025-08-12 11:10
Exhibit 99.1 Climb Bio Reports Second Quarter 2025 Financial Results and Provides Business Updates Clinical Trials of Budoprutug in Immune Thrombocytopenia (ITP) and Systemic Lupus Erythematosus (SLE) Underway; Trial of Budoprutug in Primary Membranous Nephropathy (pMN) Expected to Initiate in the Coming Weeks Budoprutug Subcutaneous Formulation Demonstrated High Bioavailability and Favorable Tolerability in Non-clinical Studies; Phase 1 Trial Expected to Initiate in the Coming Weeks, with Initial Data Anti ...
Altimmune(ALT) - 2025 Q2 - Quarterly Results
2025-08-12 11:10
Exhibit 99.1 New data from the IMPACT trial demonstrates potentially class-leading improvements in corrected T1 (cT1), an important marker of liver inflammation and fibrosis End-of-Phase 2 Meeting with FDA expected in Q4 2025 Cash, cash equivalents and short-term investments of $183.1 million as of June 30, 2025 Webcast to be held today, August 12, 2025, at 8:30 a.m. ET Altimmune Announces Second Quarter 2025 Financial Results and Business Update Positive data from the IMPACT Phase 2b trial reinforces the p ...
Centessa Pharmaceuticals(CNTA) - 2025 Q2 - Quarterly Results
2025-08-12 11:07
[Company Overview and Strategic Vision](index=1&type=section&id=Company%20Overview%20and%20Strategic%20Vision) [CEO's Message and Strategic Focus](index=1&type=section&id=CEO%27s%20Message%20and%20Strategic%20Focus) Centessa Pharmaceuticals is advancing its OX2R agonist franchise, aiming to redefine care for sleep-wake disorders and related comorbidities - Centessa is well-positioned with a novel, potential best-in-class OX2R agonist pipeline targeting sleep-wake disorders and comorbidities like excessive daytime sleepiness, impaired attention, cognitive deficits, and fatigue across neurological, neurodegenerative, and neuropsychiatric conditions[3](index=3&type=chunk) - **ORX750** is progressing in an adaptive Phase 2a study, with data expected this year for NT1, NT2, and IH, aiming to be the first OX2R agonist for NT2 and IH[4](index=4&type=chunk) - **ORX142**, the second OX2R agonist candidate, recently initiated clinical development, with data in acutely sleep-deprived healthy volunteers also expected this year[4](index=4&type=chunk) [Orexin Receptor 2 (OX2R) Agonist Pipeline Highlights](index=1&type=section&id=Orexin%20Receptor%202%20%28OX2R%29%20Agonist%20Pipeline%20Highlights) The company's OX2R agonist pipeline includes ORX750 in Phase 2a, ORX142 in Phase 1, and ORX489 in IND-enabling studies, with key clinical data expected this year - **ORX750** Phase 2a CRYSTAL-1 study for NT1, NT2, and IH is on track, with data expected this year and first-in-class potential in NT2 and IH[5](index=5&type=chunk) - **ORX142** Phase 1 clinical trial for select neurological and neurodegenerative disorders is underway, with data in acutely sleep-deprived healthy volunteers expected this year[5](index=5&type=chunk) - **ORX489** is in IND-enabling studies for the treatment of neuropsychiatric disorders[5](index=5&type=chunk) [Recent Business Developments and Program Updates](index=2&type=section&id=Recent%20Business%20Developments%20and%20Program%20Updates) [ORX142 Clinical Development](index=2&type=section&id=ORX142%20Clinical%20Development) Following FDA IND clearance in June 2025, Centessa initiated a Phase 1 clinical trial for ORX142, with initial data anticipated by the end of 2025 - Initiated Phase 1 first-in-human clinical trial for **ORX142** in June 2025 after FDA IND clearance[9](index=9&type=chunk) - Data from **ORX142** Phase 1 study in acutely sleep-deprived healthy volunteers expected in 2025[9](index=9&type=chunk) [ORX750 Clinical Data and Presentations](index=2&type=section&id=ORX750%20Clinical%20Data%20and%20Presentations) Additional Phase 1 data for ORX750 showed sustained wakefulness and improved sleepiness scores, and an abstract on the Phase 2a CRYSTAL-1 study was accepted for World Sleep 2025 Congress - Additional Phase 1 data for **ORX750** presented at AAN Annual Meeting in April 2025, showing sustained effects (mean sleep latency >30 mins on MWT) and improved KSS scores at **5.0 mg dose** over 8 hours[9](index=9&type=chunk) - An abstract detailing the adaptive design of the **ORX750** Phase 2a CRYSTAL-1 study, including an open-label long-term extension, was accepted for poster presentation at World Sleep 2025 Congress (September 5-10, 2025)[9](index=9&type=chunk) - The Phase 2a CRYSTAL-1 study for **ORX750** is ongoing, with data in NT1, NT2, and IH expected in 2025[9](index=9&type=chunk) [ORX489 Preclinical Status](index=2&type=section&id=ORX489%20Preclinical%20Status) ORX489, another OX2R agonist candidate, is currently undergoing IND-enabling studies, advancing towards potential clinical development - **ORX489** is currently in IND-enabling studies[9](index=9&type=chunk) [Second Quarter 2025 Financial Results](index=2&type=section&id=Second%20Quarter%202025%20Financial%20Results) [Financial Performance Summary](index=2&type=section&id=Financial%20Performance%20Summary) For Q2 2025, R&D expenses increased to $42.7 million, G&A expenses rose to $11.9 million, leading to a net loss of $50.3 million, with cash reserves of $404.1 million providing runway into mid-2027 Q2 2025 Financial Highlights (in millions USD) | Metric | Q2 2025 | Q2 2024 | Change (YoY) | | :-------------------------- | :------ | :------ | :----------- | | Research & Development (R&D) Expenses | $42.7 | $32.8 | +$9.9 | | General & Administrative (G&A) Expenses | $11.9 | $11.2 | +$0.7 | | Net Loss | $(50.3) | $(43.8) | $(6.5) | | Cash, Cash Equivalents and Investments (as of June 30, 2025) | $404.1 | N/A | N/A | | Cash Runway | Into mid-2027 | N/A | N/A | - Cash, cash equivalents and investments totaled **$404.1 million** as of June 30, 2025, expected to fund operations into mid-2027[9](index=9&type=chunk) [Consolidated Statements of Operations and Comprehensive Loss](index=6&type=section&id=Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) The consolidated statement of operations shows a net loss of $50.3 million for Q2 2025, an increase from $43.8 million in Q2 2024, primarily due to higher R&D expenses Consolidated Statements of Operations and Comprehensive Loss (Three Months Ended June 30, in thousands USD) | Metric | June 30, 2025 | June 30, 2024 | | :------------------------------------ | :------------ | :------------ | | License and other revenue | $— | $— | | Research and development | 42,741 | 32,815 | | General and administrative | 11,912 | 11,165 | | Loss from operations | (54,653) | (43,980) | | Interest and investment income | 4,380 | 3,240 | | Interest expense | (2,884) | (2,525) | | Other non-operating income (expense), net | 3,592 | 154 | | Loss before income taxes | (49,565) | (43,111) | | Income tax expense | 778 | 705 | | Net loss | (50,343) | (43,816) | | Net loss per ordinary share - basic and diluted | $(0.38) | $(0.40) | | Weighted average ordinary shares outstanding | 133,677,405 | 109,489,184 | [Condensed Consolidated Balance Sheets](index=7&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) As of June 30, 2025, total assets decreased to $492.1 million from $576.8 million, primarily due to reduced cash and cash equivalents, while total shareholders' equity declined to $344.9 million Condensed Consolidated Balance Sheets (as of, in thousands USD) | Metric | June 30, 2025 | December 31, 2024 | | :-------------------------------- | :------------ | :---------------- | | Cash and cash equivalents | $44,242 | $383,221 | | Investments in marketable securities | 359,888 | 98,956 | | Other assets | 87,997 | 94,621 | | **Total assets** | **$492,127** | **$576,798** | | Other liabilities | $37,663 | $66,313 | | Long term debt | 109,545 | 108,940 | | **Total liabilities** | **$147,208** | **$175,253** | | **Total shareholders' equity** | **$344,919** | **$401,545** | [About Centessa Pharmaceuticals](index=3&type=section&id=About%20Centessa%20Pharmaceuticals) [About Centessa's Orexin Receptor 2 (OX2R) Agonist Program](index=3&type=section&id=About%20Centessa%27s%20Orexin%20Receptor%202%20%28OX2R%29%20Agonist%20Program) The OX2R agonist program targets the orexin pathway to regulate the sleep-wake cycle and address symptoms like excessive daytime sleepiness, with a pipeline including ORX750, ORX142, and ORX489 - Orexin is a neuropeptide regulating the sleep-wake cycle, and targeting the OX2R pathway is a promising approach for excessive daytime sleepiness, impaired attention, cognitive deficits, and fatigue[11](index=11&type=chunk) - Centessa's OX2R agonist pipeline includes **ORX750** (Phase 2a for NT1, NT2, IH), **ORX142** (for neurological/neurodegenerative disorders), and **ORX489** (for neuropsychiatric disorders)[11](index=11&type=chunk) [About Centessa Pharmaceuticals](index=3&type=section&id=About%20Centessa%20Pharmaceuticals) Centessa Pharmaceuticals is a clinical-stage company dedicated to discovering, developing, and delivering transformational medicines, primarily focusing on its pioneering OX2R agonist program - Centessa Pharmaceuticals is a clinical-stage company focused on discovering, developing, and delivering transformational medicines[12](index=12&type=chunk) - The company is pioneering a new class of potential therapies within its OX2R agonist program for EDS, impaired attention, cognitive deficits, and fatigue across neurological, neurodegenerative, and neuropsychiatric disorders[12](index=12&type=chunk) [Forward-Looking Statements](index=3&type=section&id=Forward-Looking%20Statements) This section outlines forward-looking statements regarding clinical trials, regulatory approvals, and financial projections, subject to various risks and uncertainties - Statements regarding future events, such as clinical trial timing, data readouts, regulatory approvals, and financial runway, are forward-looking and subject to risks and uncertainties[13](index=13&type=chunk)[14](index=14&type=chunk) - Risks include safety/tolerability profiles, patient recruitment, clinical trial success, intellectual property, financing, industry trends, and geopolitical factors[14](index=14&type=chunk) [Contact Information](index=5&type=section&id=Contact%20Information) This section provides contact details for investor relations inquiries - Investor Relations contact: Kristen K. Sheppard, Esq., SVP of Investor Relations, investors@centessa.com[15](index=15&type=chunk)
FREQUENCY THERAP(FREQ) - 2025 Q2 - Quarterly Results
2025-08-12 11:05
Exhibit 99.1 Korro Reports Second Quarter 2025 Financial Results and Provides Business Updates CAMBRIDGE, Mass., August 12, 2025 (GLOBE NEWSWIRE) -- Korro Bio, Inc. (Korro) (Nasdaq: KRRO), a clinical-stage biopharmaceutical company focused on developing a new class of genetic medicines based on editing RNA for both rare and highly prevalent diseases, today reported financial results for the second quarter of 2025 and provided a business update. Ram Aiyar, Ph.D., CEO and President of Korro, said, "Throughout ...
Korro Bio(KRRO) - 2025 Q2 - Quarterly Results
2025-08-12 11:05
[Business Overview and Highlights](index=1&type=section&id=Business%20Overview%20and%20Highlights) Korro reported significant progress in its Phase 1/2a REWRITE clinical trial for KRRO-110, achieving EMA Orphan Drug Designation and ending Q2 2025 with a strong cash position, with key milestones anticipated in H2 2025 [Second Quarter 2025 Highlights](index=1&type=section&id=Second%20Quarter%202025%20Highlights) Korro reported significant progress in its Phase 1/2a REWRITE clinical trial for KRRO-110, having dosed over 80% of healthy volunteers with no serious adverse events. The company received EMA Orphan Drug Designation for KRRO-110 and ended the second quarter of 2025 with a strong cash position of $119.6 million. Key milestones, including interim trial data and a new development candidate announcement, are expected in the second half of 2025 - The Phase 1/2a REWRITE clinical trial for KRRO-110 is progressing well, with an interim data readout expected in the second half of 2025[4](index=4&type=chunk)[6](index=6&type=chunk) - Dosing has been completed for over **80%** of planned healthy volunteers in the single ascending dose (SAD) cohorts, with no treatment-emergent serious adverse events (SAEs) or dose-limiting toxicities observed[6](index=6&type=chunk)[7](index=7&type=chunk) - The European Medicines Agency (EMA) granted Orphan Drug Designation to KRRO-110 for the treatment of Alpha-1 Antitrypsin Deficiency (AATD)[6](index=6&type=chunk)[7](index=7&type=chunk) - The company plans to announce its rare metabolic disorder development candidate by the end of 2025[4](index=4&type=chunk)[6](index=6&type=chunk) - Ended the second quarter of 2025 with **$119.6 million** in cash, cash equivalents, and marketable securities[6](index=6&type=chunk) [Pipeline and Strategic Updates](index=1&type=section&id=Pipeline%20and%20Strategic%20Updates) Korro's KRRO-110 program is advancing with positive clinical trial results and regulatory designations, while the company progresses towards key milestones including new development candidates and strategic collaborations through 2027 [KRRO-110 Program Update](index=1&type=section&id=KRRO-110%20Program%20Update) The Phase 1/2a REWRITE clinical trial for KRRO-110 in AATD is advancing well, with over 80% of healthy volunteers dosed across multiple single ascending dose (SAD) cohorts, showing a safe and well-tolerated profile. The drug also received Orphan Drug Designation from the European Medicines Agency (EMA), adding to its previous U.S. FDA designation, which provides significant development incentives - More than **80%** of planned healthy volunteers have received KRRO-110 across multiple SAD cohorts, including dose levels expected to be pharmacologically relevant[7](index=7&type=chunk) - As of August 12, 2025, KRRO-110 continues to be safe and well-tolerated, with no treatment-emergent SAEs or dose-limiting toxicities observed[7](index=7&type=chunk) - The EMA granted orphan designation status to KRRO-110, which provides development incentives such as protocol assistance, reduced regulatory fees, and market exclusivity upon approval[7](index=7&type=chunk)[8](index=8&type=chunk) - KRRO-110 also received orphan drug designation from the U.S. Food and Drug Administration in March 2025[8](index=8&type=chunk) [Anticipated Upcoming Milestones](index=2&type=section&id=Anticipated%20Upcoming%20Milestones) Korro anticipates several key milestones through 2027, guided by its 3-2-1 strategy. Near-term events include an interim data readout from the KRRO-110 trial in H2 2025, the announcement of a new rare metabolic disorder candidate by year-end 2025, and the full completion of the REWRITE trial in 2026. The company is also progressing its central nervous system programs and its collaboration with Novo Nordisk - Interim data from the Part 1 SAD portion of the Phase 1/2a REWRITE clinical trial is expected in the second half of 2025[14](index=14&type=chunk) - Completion of the full REWRITE trial, including the Part 2 multiple ascending dose (MAD) portion, is expected in 2026[14](index=14&type=chunk) - A development candidate for its rare metabolic disorder program will be announced by the end of 2025[14](index=14&type=chunk) - The company continues to execute its **3-2-1 strategy**, aiming for three clinical-stage programs and targeting two tissue types by the end of 2027[14](index=14&type=chunk) - Progress continues on the collaboration with Novo Nordisk to advance up to two preclinical programs for cardiometabolic diseases[14](index=14&type=chunk) [Financial Performance](index=2&type=section&id=Financial%20Performance) Korro's second quarter 2025 financial results show increased collaboration revenue and operating expenses, resulting in a net loss, while maintaining a strong cash position expected to fund operations into 2027 [Second Quarter 2025 Financial Results](index=2&type=section&id=Second%20Quarter%202025%20Financial%20Results) For the second quarter of 2025, Korro reported **$1.5 million** in collaboration revenue, a new stream compared to the prior year. Operating expenses increased to **$28.7 million**, driven primarily by higher R&D costs, resulting in a net loss of **$25.8 million**. The company maintained a solid financial position, ending the quarter with **$119.6 million** in cash and equivalents, which is expected to fund operations into 2027 Financial Metric Summary | Financial Metric | Q2 2025 | Q2 2024 | Change | | :--- | :--- | :--- | :--- | | Cash & Marketable Securities | $119.6M (as of Jun 30) | $163.1M (as of Dec 31, 2024) | -$43.5M | | Collaboration Revenue | $1.5M | $0.0M | +$1.5M | | R&D Expenses | $21.0M | $17.1M | +$3.9M | | G&A Expenses | $7.6M | $7.0M | +$0.6M | | Net Loss | $25.8M | $21.8M | +$4.0M | - Cash, cash equivalents, and marketable securities of **$119.6 million** as of June 30, 2025, are expected to fund operating expenses and capital expenditure requirements into 2027[10](index=10&type=chunk) - The increase in collaboration revenue was due to revenue earned from the collaboration with Novo Nordisk[11](index=11&type=chunk) - The rise in R&D expenses was primarily driven by increases in personnel expenses and other research and pre-development candidate costs[12](index=12&type=chunk) [Condensed Consolidated Statements of Operations](index=5&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations) This section provides the unaudited condensed consolidated statements of operations for the three and six months ended June 30, 2025, and June 30, 2024. It details revenue, operating expenses, other income, net loss, and comprehensive loss for these periods Condensed Consolidated Statements of Operations (in thousands) | (in thousands) | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :--- | :--- | :--- | :--- | :--- | | **Collaboration revenue** | **$1,460** | **$—** | **$4,010** | **$—** | | Research and development | $21,031 | $17,138 | $40,770 | $30,710 | | General and administrative | $7,631 | $6,987 | $15,462 | $14,868 | | **Total operating expenses** | **$28,662** | **$24,125** | **$56,232** | **$45,578** | | **Loss from operations** | **($27,202)** | **($24,125)** | **($52,222)** | **($45,578)** | | Other income, net | $1,433 | $2,329 | $3,066 | $4,242 | | **Net loss** | **($25,770)** | **($21,826)** | **($49,157)** | **($41,383)** | | **Net loss per share** | **($2.74)** | **($2.43)** | **($5.24)** | **($4.87)** | [Selected Condensed Consolidated Balance Sheet Data](index=6&type=section&id=Selected%20Condensed%20Consolidated%20Balance%20Sheet%20Data) This section presents selected unaudited condensed consolidated balance sheet data as of June 30, 2025, compared to December 31, 2024. It highlights key figures including cash, total assets, total liabilities, and total stockholders' equity Selected Condensed Consolidated Balance Sheet Data (in thousands) | (in thousands) | June 30, 2025 | December 31, 2024 | | :--- | :--- | :--- | | Cash, cash equivalents and marketable securities | $119,626 | $163,054 | | Working capital | $85,461 | $116,572 | | Total assets | $180,425 | $226,240 | | Total liabilities | $65,322 | $65,825 | | Total stockholders' equity | $115,103 | $160,415 | [Company and Program Information](index=3&type=section&id=Company%20and%20Program%20Information) This section details the REWRITE clinical trial design, the genetic basis of AATD, KRRO-110's mechanism of action, and Korro Bio's proprietary RNA editing platform for genetic medicines [About REWRITE Clinical Trial](index=3&type=section&id=About%20REWRITE%20Clinical%20Trial) The REWRITE trial is a two-part, single and multiple dose-escalating study designed to evaluate the safety and tolerability of KRRO-110 in up to 64 participants, including both healthy adults and AATD patients. The trial will also assess pharmacokinetic and pharmacodynamic parameters to guide future dose selection, with interim data from Part 1 expected in the second half of 2025 and full study completion anticipated in 2026 - REWRITE is a two-part study (Part 1: single ascending doses; Part 2: multiple ascending doses) that will enroll up to **64 participants**, including healthy volunteers and AATD patients with the PiZZ genotype[16](index=16&type=chunk) - The primary endpoints are safety and tolerability, while secondary endpoints include pharmacokinetic and pharmacodynamic parameters to guide optimal dose selection[16](index=16&type=chunk) - Interim data from Part 1 is expected in the second half of 2025, and the study is anticipated to be completed in 2026[16](index=16&type=chunk) [About Alpha-1 Antitrypsin Deficiency (AATD) and KRRO-110](index=3&type=section&id=About%20Alpha-1%20Antitrypsin%20Deficiency%20(AATD)%20and%20KRRO-110) Alpha-1 Antitrypsin Deficiency (AATD) is a genetic disorder caused by a mutation in the SERPINA1 gene, leading to severe lung and liver disease. Korro's lead candidate, KRRO-110, utilizes its proprietary OPERA® RNA editing platform to co-opt an endogenous enzyme (ADAR) to repair the mutated RNA. This process is designed to restore the secretion of normal AAT protein, potentially clearing harmful protein aggregates from the liver and protecting lung function - AATD is a genetic disorder most commonly caused by a single G-to-A missense mutation in the SERPINA1 gene, leading to pulmonary emphysema and/or hepatic cirrhosis[17](index=17&type=chunk) - KRRO-110 is an RNA editing oligonucleotide from the OPERA® platform designed to use the endogenous ADAR enzyme to edit the mutated SERPINA1 RNA[17](index=17&type=chunk) - The therapeutic goal is to repair an amino acid codon, restore normal AAT protein secretion, clear protein aggregates from the liver, and preserve lung function[17](index=17&type=chunk) [About Korro Bio](index=3&type=section&id=About%20Korro%20Bio) Korro Bio is a clinical-stage biopharmaceutical company focused on developing a new class of genetic medicines based on RNA editing for both rare and prevalent diseases. The company's proprietary OPERA® platform is designed to harness the body's natural RNA editing process to make precise, transient single-base edits. This approach aims to offer improved specificity and long-term tolerability compared to DNA-based genetic medicines - Korro is a clinical-stage biopharmaceutical company developing genetic medicines based on editing RNA[18](index=18&type=chunk) - The company's platform is designed to enable precise yet transient single base edits by harnessing the body's natural RNA editing process[18](index=18&type=chunk) - By editing RNA instead of DNA, Korro aims to expand the reach of genetic medicines with increased specificity and improved long-term tolerability[18](index=18&type=chunk) [Legal Disclosures](index=3&type=section&id=Legal%20Disclosures) This section provides cautionary language regarding forward-looking statements, highlighting inherent risks and uncertainties in future projections and directing readers to SEC filings for comprehensive risk factors [Forward-Looking Statements](index=3&type=section&id=Forward-Looking%20Statements) This section contains standard cautionary language regarding forward-looking statements within the press release. It advises investors that statements about future events, such as clinical trial timelines, data readouts, candidate announcements, and financial runway, are subject to significant risks and uncertainties and should not be unduly relied upon. Readers are directed to Korro's SEC filings for a more comprehensive discussion of potential risks - The press release includes forward-looking statements concerning the timing of clinical trial data, announcement of development candidates, KRRO-110's potential, and the company's cash runway[21](index=21&type=chunk) - Actual results may differ materially from current expectations due to various factors, including risks inherent in clinical trials, regulatory oversight, and general economic conditions[21](index=21&type=chunk) - Korro does not undertake any duty to publicly update or revise forward-looking statements and advises investors to consult its SEC filings for a full list of risk factors[21](index=21&type=chunk)