Market Opportunity - The estimated addressable population suffering from presbyopia in the U.S. is 128 million, representing a market opportunity exceeding $3 billion[85]. Clinical Trial Results - LNZ100 achieved a statistically significant three-lines or greater improvement in Best Corrected Distance Visual Acuity at near, with 71% of participants showing improvement at 3 hours in the CLARITY 2 trial[85]. - LNZ100 was well-tolerated in clinical trials, with no serious treatment-related adverse events reported in over 30,000 treatment days[93]. Financial Position - As of June 30, 2024, the company had $196.1 million in cash, cash equivalents, and marketable securities, sufficient to fund operations until positive operating cash flow post-commercial launch[88]. - The company incurred an accumulated deficit of $122.1 million as of June 30, 2024, primarily due to research and development costs[88]. - The company had $196.1 million in cash, cash equivalents, and marketable securities as of June 30, 2024, with an accumulated deficit of $122.1 million[110]. Financing Activities - The company completed a PIPE Financing in July 2024, raising $30.0 million by selling 1,578,947 shares at $19.00 per share[95]. - Cash provided by financing activities for the six months ended June 30, 2024, was $169.1 million, significantly higher than $83.2 million in the same period in 2023[117]. Research and Development Expenses - The company expects research and development costs to decrease in 2024 due to the completion of the Phase 3 CLARITY trials[92]. - Research and development expenses decreased by $5.7 million, or 45%, from $12.6 million in Q2 2023 to $6.9 million in Q2 2024, primarily due to a $9.2 million decrease in contract research expenses[102]. - For the six months ended June 30, 2024, research and development expenses totaled $17.5 million, down 24% from $22.9 million in the same period in 2023[106]. Selling, General, and Administrative Expenses - Selling, general, and administrative expenses are anticipated to increase in 2024 as the company prepares for a potential commercial launch of LNZ100[92]. - Selling, general and administrative expenses increased by $5.1 million, or 219%, from $2.3 million in Q2 2023 to $7.4 million in Q2 2024, driven by increased employee salaries and marketing expenses[103]. - Selling, general and administrative expenses for the six months ended June 30, 2024, rose to $13.0 million, an increase of 181% from $4.6 million in the same period in 2023[107]. Future Capital Requirements - Future capital requirements will depend on various factors, including manufacturing costs and regulatory review outcomes for LNZ100[111]. - The company expects to continue incurring significant expenses as it seeks approval and pursues the potential commercialization of LNZ100[110]. Licensing and Royalties - The Ji Xing License Agreement includes potential milestone payments of up to $95.0 million and tiered royalties ranging from 5% to 15% on net sales in Greater China[89]. Company Classification and Dividends - The company is classified as an emerging growth company under the JOBS Act and may remain so until December 31, 2026[125]. - The company has historically paid no dividends, except for a special dividend prior to the Merger, and does not anticipate paying dividends in the future[124]. Stock Valuation and Options - The fair value of the company's common stock prior to the Merger was determined by the board of directors based on various factors, including clinical milestones and capital market conditions[124]. - The company uses the Black-Scholes option pricing model to estimate the fair value of equity awards, which is influenced by assumptions such as risk-free interest rate and expected stock price volatility[123]. - The Series A Warrants were revalued at each balance sheet date, with changes recognized in other income (expense) until they became exercisable into common stock upon completion of the Merger[122]. - The company has limited historical stock price volatility data and derives expected volatility from comparable publicly traded companies[124]. - The company does not have sufficient historical exercise data to estimate the expected term for options granted to employees, using the simplified method instead[124]. - The company has not experienced material changes to its financial statements due to revisions in estimates historically[121].

Drug Development - Submitted New Drug Application (NDA) to the U.S. FDA for LNZ100 for the treatment of presbyopia, supported by positive data from the Phase 3 CLARITY study[2] - LNZ100 achieved statistically significant improvement in near vision, with 71% of participants achieving three-lines or greater improvement at 3 hours post-administration[4] - 84% of participants achieved at least 4 lines of near vision improvement with LNZ100, demonstrating its robust product profile[4] - The company is targeting a market of approximately 128 million individuals living with presbyopia in the United States[2] - The company plans to engage with key opinion leaders and eye care professionals for the commercialization of LNZ100, pending FDA approval[12] Financial Performance - Pro forma cash, cash equivalents, and marketable securities were $226.2 million as of June 30, 2024, following a $30 million private placement from Ridgeback Capital[1] - R&D expenses decreased to $6.9 million for Q2 2024, down from $12.6 million in Q2 2023, primarily due to reduced clinical trial-related expenses[7] - SG&A expenses increased to $7.4 million for Q2 2024, compared to $2.3 million in Q2 2023, driven by personnel-related expenses and pre-commercial planning[8] - Net loss for Q2 2024 was $10.3 million, or $0.40 per share, compared to a net loss of $14.7 million, or $7.53 per share in Q2 2023[9] - Total assets as of June 30, 2024, were $202.6 million, compared to $70.4 million as of December 31, 2023[13]

Product Development and Regulatory Approval - LENZ's lead product candidate LNZ100 achieved statistically significant results in Phase 3 trials, with 71% of participants showing a three-lines or greater improvement in near vision at 3 hours post-application [124]. - The company plans to submit a New Drug Application (NDA) for LNZ100 to the FDA in mid-2024, targeting a commercial launch in the second half of 2025 [126]. - LNZ100 has patent protection until at least 2039 in the United States, supported by a robust intellectual property portfolio [123]. - The company anticipates continued significant expenses and operating losses as it seeks regulatory approval and prepares for the potential commercial launch of LNZ100 [127]. - The company expects to incur significant expenses related to the regulatory approval process for LNZ100 and preparation for its potential commercial launch [150]. Financial Performance - Research and development expenses for the three months ended March 31, 2024, were $10.5 million, a slight increase of 2% compared to $10.3 million in the same period in 2023 [144]. - Selling, general and administrative expenses surged by 142% to $5.6 million in Q1 2024, up from $2.3 million in Q1 2023, reflecting increased operational activities [144]. - Net losses for Q1 2024 were $16.6 million, compared to $12.7 million in Q1 2023, reflecting ongoing costs related to research and development and administrative expenses [150]. - Total other income (expense), net for Q1 2024 was $(560,000), a significant increase in expense compared to $(54,000) in Q1 2023, reflecting a 937% change [144]. - Cash used in operating activities for Q1 2024 was $23.9 million, resulting from a net loss and changes in working capital [157]. - As of March 31, 2024, the company had $213.3 million in cash, cash equivalents, and marketable securities, with an accumulated deficit of $111.9 million [150]. Mergers and Financing - The merger with Graphite was completed on March 21, 2024, resulting in LENZ OpCo securityholders owning approximately 65% of the combined company's common stock [136]. - Cash provided by financing activities in Q1 2024 was $171.3 million, including $117.8 million from the Merger and $53.5 million from PIPE Financing [161]. - The company received $15 million in upfront payments from Ji Xing Pharmaceuticals as part of a licensing agreement for LNZ100 and LNZ101 in Greater China [131]. Future Outlook and Expenses - Future capital requirements will depend on clinical trial results, manufacturing costs, and the ability to generate positive operating cash flow from LNZ100 sales [153]. - The company anticipates increased rent expenses due to a new lease for office space in Solana Beach, California, starting July 1, 2024 [164]. Company Classification and Reporting - Graphite is classified as an emerging growth company and may remain so until December 31, 2026 [175]. - As of June 30, 2023, the market value of Graphite's stock held by non-affiliates was less than $700 million, qualifying it as a smaller reporting company [178]. - Graphite's annual revenue for the fiscal year ended December 31, 2023, was less than $100 million [178]. - The company can delay adopting new accounting standards until it meets certain revenue or market value thresholds [176]. - Graphite's total annual gross revenue must reach at least $1.235 billion to cease being an emerging growth company [177]. - The company has the option to present only the two most recent fiscal years of audited financial statements in its Annual Report on Form 10-K [178]. - Graphite is exempt from certain public company disclosure and reporting requirements under the JOBS Act [175]. - The company intends to rely on exemptions related to executive compensation disclosures as a smaller reporting company [178]. - Graphite has not issued more than $1.0 billion in non-convertible debt during the prior three-year period [177]. - The company is not required to provide certain market risk disclosures due to its smaller reporting company status [180].

Financial Performance - The net loss for Q1 2024 was $16.6 million, or $3.53 per share, compared to a net loss of $12.7 million, or $6.50 per share, in Q1 2023[16]. - Research and Development (R&D) expenses for Q1 2024 were $10.5 million, consistent with $10.3 million in the same period of 2023[14]. - Selling, General and Administrative (SG&A) expenses increased to $5.6 million in Q1 2024 from $2.3 million in Q1 2023, primarily due to pre-launch commercial expenses and increased personnel costs[15]. Mergers and Acquisitions - The company completed a merger with Graphite Bio and a concurrent private placement of $53.5 million, ending Q1 2024 with approximately $213.3 million in cash, cash equivalents, and marketable securities[9][12]. Product Development and Market Opportunity - LENZ Therapeutics reported positive topline data from the Phase 3 CLARITY study for presbyopia, with 84% of participants achieving at least four lines of near vision improvement[3]. - The Phase 3 CLARITY study achieved statistically significant improvements in near vision, with p<0.0001 for all primary and secondary endpoints[4]. - The estimated U.S. market opportunity for LNZ100 is over $3 billion, targeting the 128 million presbyopes in the United States[6][3]. - The company plans to submit a New Drug Application (NDA) for LNZ100 in mid-2024, with a potential commercial launch as early as the second half of 2025[7][3]. Commercial Strategy - The company is actively building its U.S. commercial capabilities in preparation for the projected launch following potential FDA approval[8]. - Capstone data from the Phase 3 CLARITY study will be presented at a Key Opinion Leader event on June 18, 2024[11].

Merger and Corporate Restructuring - The company announced a merger agreement with LENZ Therapeutics, Inc. on November 14, 2023, which is subject to stockholder approval and customary closing conditions[26]. - The merger with LENZ is critical for future operations, and there are no assurances that it will be successfully consummated[27]. - The merger agreement was unanimously approved by the company's board of directors, which resolved to recommend approval to stockholders[26]. - The company is restrained from soliciting other acquisition proposals during the merger's pendency, which may affect its strategic options[13]. - In February 2023, the company announced a corporate restructuring resulting in approximately 78.1% reduction in workforce[23]. Financial Performance and Expectations - The company has incurred significant losses since inception and expects to continue incurring losses for the foreseeable future, with no revenue generated from product sales to date[21]. - The company has never generated revenue from product sales and may never become profitable, indicating a need for substantial additional funding[5]. - The company is considered a "shell company" under federal securities laws due to the sale and write-off of certain operating assets, leading to more stringent reporting requirements[21]. Development Programs and Product Candidates - The company has no remaining ongoing development programs after transferring its pre-clinical non-genotoxic conditioning program to Maro Bio, Inc.[24]. - The company announced a voluntary pause of its Phase 1/2 CEDAR study for its lead product candidate nula-cel due to a serious adverse event in January 2023, leading to the decision to discontinue its development in February 2023[32]. - The company has terminated the development of its lead product candidate, nula-cel, and other related programs due to a serious adverse event and strategic review[32][34][35]. - GPH102 for beta-thalassemia and GPH201 for XSCID, both leveraging the same gene editing platform as nula-cel, have also been terminated due to similar technical limitations[33][34]. Intellectual Property and Licensing Agreements - The company continues to hold and maintain technology and intellectual property related to its nula-cel program, despite discontinuing its development[24]. - As of December 31, 2023, the company in-licensed two issued U.S. patents and two pending U.S. patent applications from Stanford, with expected expiration in 2036[40]. - The company has paid an upfront license fee of $50,000 to Stanford and issued approximately 0.6 million shares of common stock as part of its exclusive license agreement[45]. - The annual license maintenance fee to Stanford will increase from $5,000 to $50,000 over the first seven anniversaries, and $200,000 after the first commercial sale of a licensed product[46]. - The company is obligated to pay Stanford up to $12.8 million upon achieving certain development, regulatory, and commercial milestones for each licensed product[49]. - The company has granted Kamau an option to acquire certain technology and intellectual property related to the nula-cel program and related preclinical platform assets[42][53]. Regulatory Environment and Compliance - The FDA requires a Biologics License Application (BLA) for marketing biological products in the U.S., which includes extensive data from preclinical and clinical studies[83]. - An Investigational New Drug (IND) application must be submitted and becomes effective 30 days after receipt by the FDA unless safety concerns arise[83]. - Clinical trials are conducted in three phases, with Phase 3 requiring two adequate and well-controlled trials for BLA approval[94]. - The FDA aims to review standard BLAs within ten months and priority reviews within six months after acceptance for filing[97]. - Regulatory approval may come with specific indications and limitations on marketing, including the requirement for a Risk Evaluation and Mitigation Strategy (REMS)[100]. - The FDA may require post-marketing studies to monitor the safety and effectiveness of approved products[100]. - Compliance with Good Manufacturing Practices (cGMP) is mandatory for manufacturing facilities before BLA approval[98]. Market and Competitive Landscape - The company faces competition from several firms in gene editing and gene therapy, including Beam Therapeutics, bluebird bio, and CRISPR Therapeutics, among others[37]. - The competitive landscape includes several companies advancing gene editing and gene therapy programs, posing significant risks to the company's market position[37][38]. - The marketability of product candidates may suffer if government and third-party payors do not provide adequate coverage and reimbursement[144]. - In the U.S., third-party payors are increasingly challenging drug prices and may limit coverage to specific approved drug products[141]. Employment and Workforce - As of December 31, 2023, the company had six full-time employees, with no representation by labor unions[159]. - The company plans to attract and retain employees through stock-based compensation awards[160]. Legislative and Policy Environment - Legislative measures at the state level are increasingly aimed at controlling pharmaceutical pricing, which may impact the company's market strategies[158]. - The Biden administration has proposed measures to control drug costs, including a prescription drug pricing model to incentivize manufacturers[156].

Financial Performance - The net loss for the three months ended September 30, 2023, was $22.485 million, compared to a net loss of $24.682 million for the same period in 2022, showing an improvement of about 8.8%[17] - The net loss for the nine months ended September 30, 2023, was $101,733,000, compared to a net loss of $76,453,000 for the same period in 2022, representing an increase of approximately 33%[25] - Comprehensive loss for the nine months ended September 30, 2023, was $100.780 million, compared to $78.049 million for the same period in 2022, indicating a decline of approximately 28.9%[17] Cash and Cash Equivalents - As of September 30, 2023, cash and cash equivalents increased to $182.988 million from $47.730 million as of December 31, 2022, representing a significant growth[15] - Cash, cash equivalents, and restricted cash at the end of the period were $184,704,000, a significant increase from $58,107,000 at the end of the same period in 2022[25] - The company had cash, cash equivalents, and marketable securities totaling $234.0 million as of September 30, 2023, sufficient to fund operations for at least the next 12 months[32] Assets and Liabilities - Total current assets decreased to $238.783 million from $275.365 million as of December 31, 2022, indicating a reduction of approximately 13.3%[15] - Total liabilities increased to $62.209 million from $25.611 million as of December 31, 2022, reflecting a rise of approximately 143.5%[15] - The accumulated deficit increased to $344.136 million as of September 30, 2023, from $242.403 million as of December 31, 2022[15] Research and Development - Research and development expenses for the three months ended September 30, 2023, were $2.384 million, a decrease from $18.302 million in the same period of 2022[17] - The Company recognized $1.1 million in research and development expenses related to the LCGM MSA for the nine months ended September 30, 2023, compared to $4.5 million for the same period in 2022[68] - The Company has not recognized any research and development expenses in connection with the IDT License Agreement for the three and nine months ended September 30, 2023[73] Restructuring and Workforce Changes - The Company announced a restructuring plan in February 2023, resulting in a total reduction in force of 78.1% by August 2023[29] - The company incurred approximately $3.4 million in employee termination benefits expense related to the First Restructuring Plan, which eliminated about 50% of its workforce[126][127] - The Second Restructuring Plan, approved in August 2023, eliminated an additional 33.1% of the workforce, totaling 78.1% overall[128] Stock and Equity - The weighted-average shares used in computing net loss per share for the three months ended September 30, 2023, were 57,257,241, compared to 55,206,139 for the same period in 2022[17] - The company had a total of 57,971,910 shares outstanding as of September 30, 2023, compared to 58,149,317 shares as of September 30, 2022[22] - The company reserved a total of 17,465,405 shares for future issuance, up from 13,893,161 shares as of December 31, 2022, reflecting an increase of 25.0%[99] Impairment and Asset Sales - The company reported a significant impairment of assets amounting to $43,276,000 during the nine months ended September 30, 2023[25] - The company recorded impairment charges and loss on disposal of assets of $5.3 million and $6.8 million for the three and nine months ended, respectively[131] - The company sold certain assets related to its non-genotoxic conditioning technology for upfront consideration of $0.5 million, with potential additional payments of up to $1.0 million[133] Lease Agreements - The company entered into a lease agreement for 85,165 square feet of office and laboratory space with a total right-of-use asset of $32.0 million recognized upon lease commencement[88] - As of September 30, 2023, the Company had total operating lease liabilities of $53.1 million[90] - The Company entered into a sublease agreement for approximately 15,212 square feet of space in South San Francisco, California[141] Strategic Initiatives - The company paused its Phase 1/2 CEDAR study of its lead product candidate, nula-cel, in January 2023 due to a serious adverse event[27] - The Company continues to explore strategic alternatives following the discontinuation of the nula-cel development[30] - The Company has primarily relied on private equity and convertible debt financings to fund operations since inception[32]

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 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, 2023 OR ☐ 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: 001-40532 GRAPHITE BIO, INC. (Exact Name of Registrant as Specified in its Charter) Delaware 84-4867570 ( State or other ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 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 March 31, 2023 OR ☐ 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: 001-40532 GRAPHITE BIO, INC. (Exact Name of Registrant as Specified in its Charter) Delaware 84-4867570 ( State or other ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-K (Mark one) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2022 OR ☐ 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: 001-40532 GRAPHITE BIO, INC. (Exact name of Registrant as specified in its charter) Delaware 2836 84-4867570 (State or other jurisdiction ...