Friedreich Ataxia

SBT-589 showed mitochondrial protection across models of Friedreich's ataxia SBT-589 displayed cardioprotective effects in aggressive mouse model of FA cardiomyopathy Ongoing studies are expected to build on these insights as SBT-589 progresses through developmental stage-gates NEEDHAM, Mass., March 19, 2024 /PRNewswire/ -- Stealth BioTherapeutics Inc. (the "Company" or "Stealth"), a clinical-stage biotechnology company focused on the discovery, development, and commercialization of novel therapies for diseases involving mitochondrial dysfunction, announced today the presentation of new SBT-589 data demonstrating cardioprotective effects across pre-clinical models of Friedreich's ataxia (FA). The data were presented at the Wellcome Trust Conference on Mitochondrial Medicine – Therapeutic Development, held March 18-20, 2024, in Cambridge, England. FA is a chronic disease of metabolic disruption that is typically diagnosed in childhood or adolescence, is inexorably progressive and usually results in premature death from cardiomyopathy or heart failure. There are no approved therapies for the progressive cardiomyopathy, which is linked to severe mitochondrial dysfunction, and is the leading cause of early death in this devastating disease. SBT-589 is a promising novel molecule that acts on mitochondrial pathways essential for cellular health and energy production that are impaired in FA cardiomyopathy. To evaluate the potential of SBT-589 in FA, a series of studies were conducted in FA patient-derived cells, isolated heart mitochondria, and an aggressive mouse model of FA cardiomyopathy. SBT-589 improved bioenergetics in FA patient-derived cells and mitochondria. In an FA mouse model with prominent cardiac hypertrophy and aggressive mortality, mice treated with once-daily SBT-589 displayed significantly reduced cardiac hypertrophy and a delay in the onset of mortality compared to vehicle-treated mice. "Mitochondrial dysfunction is a central contributor to pathology in Friedreich's ataxia, a disease in which heart failure and sudden cardiac events are among the leading causes of mortality," said David A. Brown, Senior Vice President, Discovery. "Mitigating heart disease in FA is central to Stealth's ongoing efforts to improve the lives of individuals with FA. The new findings presented today support continued development of SBT-589 as a potentially disease-modifying therapy to address the unmet need in cardiomyopathy associated with Friedreich's ataxia." The data were presented by Dr. Laura E. Kropp, Senior Manager of Discovery Biology at Stealth, and recipient of the Keith Michael Andrus Cardiac Award from the Friedreich's Ataxia Research Alliance. About Friedreich's Ataxia Friedreich's ataxia is a rare genetic disease affecting an estimated 1 in 50,000 individuals in the United States. The disease is caused by a defect in the frataxin gene resulting in a relative deficiency of frataxin, leading to mitochondrial iron accumulation and oxidative stress. More than 90% of patients with Friedreich's ataxia experience progressive cardiomyopathy which usually becomes fatal by early adulthood and is the leading cause of early mortality. About Stealth BioTherapeutics Stealth BioTherapeutics is a clinical-stage biotechnology company focused on the discovery, development, and commercialization of novel therapies for age-related and rare genetic diseases involving mitochondrial dysfunction. The Company is initiating Phase 3 clinical trials of elamipretide, its lead investigational product candidate, in dry age-related macular degeneration. Elamipretide is also being tested in a fully enrolled Phase 3 clinical trial in primary mitochondrial myopathy, a rare skeletal myopathic disease caused by nuclear DNA mutations, and is the subject of a New Drug Application for Barth syndrome, an ultra-rare cardioskeletal disease. The Company is evaluating its second-generation clinical-stage candidate, SBT-272, for ophthalmic and neurological disease indications. The Company has a deep pipeline of novel mitochondria-targeted compounds under evaluation as therapeutic product candidates. Several of these lead compounds, including SBT-589, have progressed through pre-clinical development stage-gates and toward IND-enabling studies. Investor Contact Kendall Investor Relations Adam Bero, Ph.D. [email protected] [email protected] Media Contact Anna Stallman Communications Anna Stallman [email protected] SOURCE Stealth BioTherapeutics Inc.

Positive top-line data from Phase 2 dose exploration study of nomlabofusp, which was generally well-tolerated, with dose-dependent increases in tissue frataxin levels observedInitiated discussions with the Food and Drug Administration (FDA) on the potential use of tissue frataxin levels as a novel surrogate endpoint to support a Biologics License Application (“BLA”) submission for accelerated approval targeted for 2H 2025In January 2024, initiated open label extension (OLE) study with 25 mg daily dosing of nomlabofusp, with first patient dosed in March 2024; interim data expected in Q4 2024 Recent successful financing that raised $161.6 million extends expected operating runway into 2026. BALA CYNWYD, Pa., March 14, 2024 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (“Larimar”) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today reported its fourth quarter and full year 2023 operating and financial results. “This year we made tremendous progress across key clinical and regulatory milestones for our nomlabofusp program. We were thrilled to recently report positive top-line data and successful completion of our Phase 2 dose exploration study. Nomlabofusp was generally well-tolerated and demonstrated dose-dependent increases in skin and buccal cell frataxin levels. Importantly, skin frataxin levels as a percentage of levels in healthy volunteers more than doubled in all patients after 14 days of daily treatment with 50 mg. The clear dose-response and the magnitude of increase in tissue frataxin levels further reinforces the therapeutic potential of nomlabofusp to address frataxin deficiency, the known root cause of disease in patients with Friedreich’s ataxia (FA),” said Carole Ben-Maimon, MD, President, and Chief Executive Officer of Larimar. “Additionally, the first patient in our OLE study has been dosed. The OLE study will inform on the long-term safety profile and long-term tissue frataxin levels. We remain on track to report interim data from the OLE study in the fourth quarter of 2024.” Dr. Ben-Maimon continued, “On the regulatory front, we are continuing discussions with the FDA on the potential use of frataxin as a novel surrogate endpoint to support accelerated approval. We are also beginning to plan for a global double-blind placebo-controlled confirmatory study expected to be initiated prior to a potential BLA submission. The BLA submission is targeted for the second half of 2025. We believe our clinical datasets supporting nomlabofusp’s differentiated mechanism of action, coupled with our strengthened balance sheet and expected runway into 2026, strongly position us for execution of our upcoming pivotal milestones designed to help address the urgent unmet needs of the FA community.” Recent Highlights In February 2024, Larimar announced positive top-line data and successful completion of its four-week, placebo-controlled Phase 2 dose exploration study of nomlabofusp (CTI-1601) in patients with FA. Nomlabofusp was generally well-tolerated up to 4-weeks, had a predictable pharmacokinetic profile, and led to dose-dependent increases in frataxin in skin and buccal cells after daily dosing of 14 days followed by every other day dosing until day 28 in the 25 mg and 50 mg cohorts. Increases in frataxin levels in skin cells were seen in all treated patients, and in buccal cells for the majority of patients. At Day 14, all patients (with quantifiable levels at baseline and Day 14) treated with 50 mg of nomlabofusp achieved frataxin levels in skin cells greater than 33% of the average level found in healthy volunteers, with 3 patients achieving levels greater than 50% of the average healthy volunteer level.In February 2024, Larimar announced that it had initiated discussions with the FDA on use of tissue frataxin levels as a potential novel surrogate endpoint. Larimar received FDA acknowledgement that frataxin deficiency appears to be critical to the pathogenic mechanism of FA, and that there continues to be an unmet need for treatments that address the underlying disease pathophysiology. Discussions to support an accelerated approval are ongoing with a BLA submission targeted for 2H 2025.In February 2024, Larimar raised net proceeds of approximately $161.6 million through a public offering of common stock. In March 2024, the first patient in the OLE study evaluating daily subcutaneous injections of 25 mg of nomlabofusp self-administered or administered by a caregiver was dosed. Participants who completed treatment in the Phase 2 dose exploration trial, or who previously completed a prior clinical trial of nomlabofusp, are potentially eligible to screen for the OLE study. The OLE study will evaluate the safety and tolerability, pharmacokinetics, and frataxin levels in peripheral tissues as well as other exploratory pharmacodynamic markers (lipid profiles and gene expression data) following long-term subcutaneous administration of nomlabofusp. Clinical assessments collected during the trial will be compared to data from a matched control arm derived from participants in the Friedreich’s Ataxia Clinical Outcome Measures Study (FACOMS) database. Interim data is expected in Q4 2024. To potentially escalate dose in the OLE study, data from the 50 mg cohort of the Phase 2 study, as well as available data from the 25 mg dose in the OLE study will be submitted for FDA review due to continued partial clinical hold. Fourth Quarter and Full Year 2023 Financial Results As of December 31, 2023, the Company had cash, cash equivalents and marketable securities totaling $86.8 million. As noted above, in February 2024, we raised approximately $161.6 million in net proceeds through a public offering of common stock. The Company reported a net loss for the fourth quarter of 2023 of $13.0 million, or $0.30 per share, compared to a net loss of $9.4 million, or $0.21 per share, for the fourth quarter of 2022. Research and development expenses for the fourth quarter of 2023 were $10.6 million compared to $7.2 million for the fourth quarter of 2022. The increase in research and development expenses compared to the prior year period was primarily driven by an increase of $1.1 million in drug manufacturing costs, an increase of $0.8 million in consulting expenditures, an increase of $0.7 million in personnel expense, an increase of $0.4 million in clinical costs related to an increase in the OLE study partially offset by a decrease in clinical costs associated with the Phase 2 nomlabofusp trial discussed above. General and administrative expenses for the fourth quarter of 2023 were $3.5 million compared to $3.2 million for the fourth quarter of 2022. The increase in general and administrative expense was primarily driven by an increase of $0.2 million in stock-based compensation expense associated with stock option grants made in 2023, an increase of $0.1 million in personnel expense and an increase of $0.1 million in operational costs primarily related to technology, partially offset by a decrease of $0.2 million in legal fees. For the full year 2023, the Company reported a net loss of $36.9 million, or $0.84 per share, compared to a net loss of $35.4 million, or $1.37 per share for the same period in 2022. Research and development expenses for the full year 2023 were $27.7 million compared to $24.3 million for the same period in 2022. The increase in research and development expenses compared to the prior year period was primarily driven by an increase of $1.9 million in personnel expense, an increase of $1.3 million in clinical expense, an increase of $1.1 million in consulting expenditures, an increase of $0.5 million in nonclinical development costs, an increase of $0.5 million in internal lab costs, and an increase of $0.3 million in stock-based compensation expense associated with stock option grants made in 2022 and 2023, partially offset by a decrease of $2.0 million in drug manufacturing costs and a decrease of $0.2 million in royalty fees associated with the milestone achieved in 2022. General and administrative expenses for the full year 2023 were $14.1 million compared to $12.3 million for the same period in 2022. The increase in general and administrative expense was primarily driven by an increase of $0.7 million in stock-based compensation expense associated with stock option grants made in 2022 and 2023, an increase of $0.5 million in personnel expense, an increase of $0.5 million in operational costs primarily related to technology and an increase of $0.4 million in professional fees primarily related to legal, accounting and consulting services, partially offset by a decrease of $0.3 million in insurance expense. About Larimar TherapeuticsLarimar Therapeutics, Inc. (Nasdaq: LRMR), is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases. Larimar’s lead compound, nomlabofusp, is being developed as a potential treatment for Friedreich's ataxia. Larimar also plans to use its intracellular delivery platform to design other fusion proteins to target additional rare diseases characterized by deficiencies in intracellular bioactive compounds. For more information, please visit: https://larimartx.com. Forward-Looking StatementsThis press release contains forward-looking statements that are based on Larimar’s management’s beliefs and assumptions and on information currently available to management. All statements contained in this release other than statements of historical fact are forward-looking statements, including but not limited to statements regarding Larimar’s ability to develop and commercialize nomlabofusp (also known as CTI-1601) and other planned product candidates, Larimar’s planned research and development efforts, including the timing of its nomlabofusp clinical trials, interactions with the FDA and overall development plan and other matters regarding Larimar’s business strategies, ability to raise capital, use of capital, results of operations and financial position, and plans and objectives for future operations. In some cases, you can identify forward-looking statements by the words “may,” “will,” “could,” “would,” “should,” “expect,” “intend,” “plan,” “anticipate,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “ongoing” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. These statements involve risks, uncertainties and other factors that may cause actual results, performance, or achievements to be materially different from the information expressed or implied by these forward-looking statements. These risks, uncertainties and other factors include, among others, the success, cost and timing of Larimar’s product development activities, nonclinical studies and clinical trials, including nomlabofusp clinical milestones and continued interactions with the FDA; that preliminary clinical trial results may differ from final clinical trial results, that earlier non-clinical and clinical data and testing of nomlabofusp may not be predictive of the results or success of later clinical trials, and assessments; that the FDA may not ultimately agree with Larimar’s nomlabofusp development strategy; the potential impact of public health crises on Larimar’s future clinical trials, manufacturing, regulatory, nonclinical study timelines and operations, and general economic conditions; Larimar’s ability and the ability of third-party manufacturers Larimar engages, to optimize and scale nomlabofusp’s manufacturing process; Larimar’s ability to obtain regulatory approvals for nomlabofusp and future product candidates; Larimar’s ability to develop sales and marketing capabilities, whether alone or with potential future collaborators, and to successfully commercialize any approved product candidates; Larimar’s ability to raise the necessary capital to conduct its product development activities; and other risks described in the filings made by Larimar with the Securities and Exchange Commission (SEC), including but not limited to Larimar’s periodic reports, including the annual report on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K, filed with or furnished to the SEC and available at www.sec.gov. These forward-looking statements are based on a combination of facts and factors currently known by Larimar and its projections of the future, about which it cannot be certain. As a result, the forward-looking statements may not prove to be accurate. The forward-looking statements in this press release represent Larimar’s management’s views only as of the date hereof. Larimar undertakes no obligation to update any forward-looking statements for any reason, except as required by law. Investor Contact: Joyce Allaire LifeSci Advisors jallaire@lifesciadvisors.com (212) 915-2569 Company Contact:Michael Celano Chief Financial Officermcelano@larimartx.com(484) 414-2715 Larimar Therapeutics, Inc.Consolidated Balance Sheet(dollars in thousands except share and per share data) December 31, December 31, 2023 2022 Assets Current assets: Cash and cash equivalents $26,749 $26,825 Marketable securities 60,041 91,603 Prepaid expenses and other current assets 3,385 2,311 Total current assets 90,175 120,739 Property and equipment, net 684 831 Operating lease right-of-use assets 3,078 2,858 Restricted cash 1,339 1,339 Other assets 659 638 Total assets $95,935 $126,405 Liabilities and Stockholders’ Equity Current liabilities: Accounts payable $1,283 $1,686 Accrued expenses 7,386 8,408 Operating lease liabilities, current 837 611 Total current liabilities 9,506 10,705 Operating lease liabilities 4,709 4,797 Total liabilities 14,215 15,502 Commitments and contingencies (See Note 8) Stockholders’ equity: Preferred stock; $0.001 par value per share; 5,000,000 shares authorized as of December 31, 2023 and December 31, 2022; no shares issued and outstanding as of December 31, 2023 and December 31, 2022 — — Common stock, $0.001 par value per share; 115,000,000 shares authorized as of December 31, 2023 and December 31, 2022; 43,909,069 and 43,269,200 shares issued and outstanding as of December 31, 2023 and December 31, 2022, respectively 43 43 Additional paid-in capital 270,150 262,496 Accumulated deficit (188,554) (151,605) Accumulated other comprehensive loss 81 (31) Total stockholders’ equity 81,720 110,903 Total liabilities and stockholders’ equity $95,935 $126,405 Larimar Therapeutics, Inc.Consolidated Statements of Operations(In thousands, except share and per share data) Three Months Ended December 31, Year Ended December 31, 2023 2022 2023 2022 Operating expenses: Research and development $10,648 $7,218 $27,670 $24,250 General and administrative 3,514 3,221 14,088 12,276 Total operating expenses 14,162 10,439 41,758 36,526 Loss from operations (14,162) (10,439) (41,758) (36,526)Other income, net 1,169 1,014 4,809 1,171 Net loss $(12,993) $(9,425) $(36,949) $(35,355)Net loss per share, basic and diluted $(0.30) $(0.21) $(0.84) $(1.37)Weighted average common shares outstanding, basic and diluted 43,905,903 43,897,603 43,901,241 25,761,394

Lexeo announces $95.0M equity financing, which in addition to 2023 year-end cash and cash equivalents of $121.5M, extends runway to fund operations into 2027 Reports frataxin protein expression data from a subset of the second dose cohort of SUNRISE-FA, a Phase 1/2 clinical trial of LX2006 for the treatment of Friedreich’s ataxia (FA) cardiomyopathy, showing positive change in post-treatment frataxin levels Additional interim data readout from SUNRISE-FA expected in mid-2024, with follow-up out to one year from the low-dose and multiple time points of follow-up expected from at least three patients treated at the mid-dose Initiated SNAPSHOT-PKP2, a natural history study designed to evaluate PKP2-ACM disease progression up to two years retrospectively and over twelve months prospectively, in up to 20 patients in the U.S. All milestones for LX2020 remain on track NEW YORK, March 11, 2024 (GLOBE NEWSWIRE) -- Lexeo Therapeutics, Inc. (Nasdaq: LXEO), a clinical stage genetic medicine company dedicated to pioneering treatments for genetically defined cardiovascular diseases and APOE4-associated Alzheimer’s disease, today reported fourth quarter and full year 2023 financial results and provided operational highlights. “The past year has been transformative for Lexeo as we successfully completed our initial public offering and continued to advance our pipeline of genetic medicines,” stated R. Nolan Townsend, Chief Executive Officer of Lexeo Therapeutics. “Building on our success in 2023, we are pleased to report that we have observed a positive change in frataxin levels now in Cohort 2 of the SUNRISE-FA trial. The low baseline protein levels observed in these patients provide unique insights into the biology of FA and we believe the increases in post treatment protein expression mediated by LX2006 may be sufficient to restore mitochondrial function with physiological improvement. These data, combined with our recent financing and progress across our pipeline, reflect exciting developments and add to our track record of clinical execution.” SUNRISE-FA Liquid Chromatography Mass Spectrometry (LCMS) Assay Protein Expression Data SubjectPre-Treatment FXN Levels (ng/mg protein)Post-Treatment FXNLevels (ng/mg protein)Change fromBaseline (ng/mg protein)Cohort 1 Subject 1 (1.8x1011 vg/kg)0.750.970.22Cohort 2 Subject 1 (5.6x1011 vg/kg)0.972.771.81Cohort 2 Subject 2 (5.6x1011 vg/kg)1.832.230.40 Note: Only one patient in Cohort 1 underwent pre and post treatment cardiac biopsy. Average pre-treatment FXN level across three subjects was 1.18ng/mg. Compared to FXN levels observed in 29 cadaver tissue samples from 16 individuals without heart disease, we believe the average pre-treatment FXN level represents approximately two percent of healthy individual frataxin levels in the heart.To our knowledge, SUNRISE-FA is the first clinical trial to evaluate frataxin levels in the target organ of FA cardiomyopathy patients via cardiac biopsy. Data to date suggest that FA patients may have lower FXN levels in the heart versus peripheral tissues.Across all three cardiac biopsies, we observed an increase in FXN levels as measured by liquid chromatography mass spectrometry relative to pre-treatment baseline levels.We observed an approximately five-fold increase in protein on average in the second cohort (5.6x1011 vg/kg) to date, relative to the first cohort (1.8x1011 vg/kg). A dose-dependent response was also seen in our IND-enabling preclinical studies, where a non-linear relationship was observed between dose delivered and frataxin protein expression. Lexeo expects to present additional interim data from SUNRISE-FA Cohorts 1 and 2 in mid-2024, including measures of hypertrophy and other cardiac biomarkers at multiple timepoints(1). Ahead of this interim readout, Lexeo expects to provide an analysis of baseline characteristics from LX2006-treated patients to characterize the cardiovascular disease phenotype seen in FA as well as baseline levels of hypertrophy and other cardiac biomarkers that will be reported at mid-year. ____________(1) Trial participants receive immune suppression with prednisone beginning on the day prior to treatment/infusion through 14 weeks following LX2006 administration. Given the potential impact of corticosteroids on systemic status and cardiac biomarkers, efficacy data in the mid-2024 data readout will be presented for timepoints following cessation of corticosteroid administration. Business and Program Updates LX2006 for the Treatment of FA Cardiomyopathy: Announced preliminary frataxin protein expression data from the second dose cohort of SUNRISE-FA showing positive change in post-treatment frataxin levels three months following administration of LX2006. Lexeo expects to report additional interim data from the SUNRISE-FA Phase 1/2 clinical trial in mid-2024 with follow-up out to one year from the low-dose and multiple timepoints of follow-up expected from at least three patients treated at the mid-dose.LX2020 for the Treatment of PKP2-ACM: In December 2023, the FDA granted Fast Track designation (FTD) and Orphan Drug designation (ODD) to LX2020, which allows more frequent interactions with FDA to facilitate development and expedite review processes. The ODD also provides additional benefits to Lexeo, such as market exclusivity upon regulatory approval. Clinical trial start-up activities are underway and Lexeo expects to dose the first patient in the HEROIC-PKP2 Phase 1/2 clinical trial of LX2020 in 1H 2024, with an interim data readout from Cohort 1 expected in 2H 2024. Lexeo has also initiated SNAPSHOT-PKP2, a natural history study designed to evaluate PKP2-ACM disease progression up to two years retrospectively and over twelve months prospectively in up to 20 patients in the United States.APOE4-associated Alzheimer’s Disease Programs: Lexeo completed enrollment of all four cohorts in the LEAD Phase 1/2 clinical trial of LX1001 for the treatment of APOE4-associated Alzheimer’s disease in Q4 2023. The Company expects to provide an interim data readout of all cohorts in 2H 2024.Pricing of $95 Million Equity Financing: Lexeo today announced that it has entered into a common stock purchase agreement with certain new and existing investors to issue and sell an aggregate of 6,278,905 shares of its common stock at a price of $15.13 per share, through a private placement. Lexeo anticipates the gross proceeds from the private placement to be approximately $95.0 million, before deducting any offering related expenses. The financing is expected to close on or about March 13, 2024, subject to customary closing conditions. The proceeds from this financing, combined with current cash, cash equivalents and marketable securities, are expected to fund operating and capital expenditures into 2027.Completed Initial Public Offering: In November 2023, Lexeo completed its underwritten initial public offering of 10,139,656 shares of its common stock, including the exercise of the underwriter’s option to purchase 1,048,746 additional shares of its common stock. The aggregate gross proceeds from the offering were $111.5 million, before deducting underwriting discounts and commissions and offering expenses payable by Lexeo. Expected Upcoming Milestones LX2006 for the treatment of Friedreich’s ataxia cardiomyopathy Interim data readout in mid-2024 LX2020 for the treatment of PKP2-ACM First patient dosed in 1H 2024Interim data readout (Cohort 1) in 2H 2024 LX1001 for the treatment of APOE4-associated Alzheimer’s disease Interim Phase 1/2 data readout (all cohorts) in 2H 2024 LX2021 for the treatment of DSP cardiomyopathy Initiate IND-enabling studies in 2024 Fourth Quarter and Full Year Financial Results Cash Position: As of December 31, 2023, cash and cash equivalents were $121.5 million, excluding the $95.0 million gross proceeds expected to be raised in the private placement announced today. Lexeo expects the additional funds raised in the financing to extend its runway into 2027.R&D Expenses: R&D expenses were $8.2 million for the three months ended December 31, 2023, compared to $11.4 million for the three months ended December 31, 2022. Research and Development expenses were $53.1 million for the year ended December 31, 2023, compared to $49.2 million for the same period in 2022.G&A Expenses: G&A expenses were $6.8 million for the three months ended December 31, 2023, compared to $3.8 million for the three months ended December 31, 2022. G&A expenses were $15.4 million for the year ended December 31, 2023, compared to $12.0 million for the same period in 2022.Net Loss: Net loss was $14.2 million or $0.86 per share (basic and diluted) for the three months ended December 31, 2023, compared to $14.6 million or $8.86 per share (basic and diluted) for the three months ended December 31, 2022. Net loss was $66.4 million or $12.40 per share (basic and diluted) for the year ended December 31, 2023, compared to $59.3 million or $36.36 per share (basic and diluted) for the year ended December 31, 2022. About Lexeo Therapeutics Lexeo Therapeutics is a New York City-based, clinical stage genetic medicine company dedicated to transforming healthcare by applying pioneering science to fundamentally change how genetically defined cardiovascular diseases and APOE4-associated Alzheimer’s disease are treated. Using a stepwise development approach, Lexeo is leveraging early proof-of-concept functional and biomarker data to advance a pipeline of cardiovascular and APOE4-associated Alzheimer’s disease programs. Forward looking Statements Certain statements in this press release may constitute “forward-looking statements” within the meaning of the federal securities laws, including, but not limited to, our expectations and plans regarding our current product candidates and programs, including the anticipated timing of the initiation of and results from our clinical trials, expectations regarding the time period over which our capital resources will be sufficient to fund our anticipated operations and estimates regarding Lexeo’s financial condition. Words such as “may,” “might,” “will,” “objective,” “intend,” “should,” “could,” “can,” “would,” “expect,” “believe,” “design,” “estimate,” “predict,” “potential,” “develop,” “plan” or the negative of these terms, and similar expressions, or statements regarding intent, belief, or current expectations, are forward-looking statements. While Lexeo believes these forward-looking statements are reasonable, undue reliance should not be placed on any such forward-looking statements. These forward-looking statements are based upon current information available to the company as well as certain estimates and assumptions and are subject to various risks and uncertainties (including, without limitation, those set forth in Lexeo’s filings with the U.S. Securities and Exchange Commission (the SEC)), many of which are beyond the company’s control and subject to change. Actual results could be materially different from those indicated by such forward looking statements as a result of many factors, including but not limited to: risks and uncertainties related to global macroeconomic conditions and related volatility; expectations regarding the initiation, progress, and expected results of our preclinical studies, clinical trials and research and development programs; the unpredictable relationship between preclinical study results and clinical study results; delays in submission of regulatory filings or failure to receive regulatory approval; liquidity and capital resources, including the anticipated closing of the private placement and anticipated runway extension; and other risks and uncertainties identified in our Quarterly Report of 10-Q for the quarterly period ended September 30, 2023, filed with the SEC on December 11, 2023, and subsequent future filings we may make with the SEC. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. Lexeo claims the protection of the Safe Harbor contained in the Private Securities Litigation Reform Act of 1995 for forward-looking statements. Lexeo expressly disclaims any obligation to update or alter any statements whether as a result of new information, future events or otherwise, except as required by law. Media Response: Carolyn Hawley (619) 849-5382 Carolyn.hawley@inizioevoke.com Investor Response: Laurence Watts (619) 916-7620 laurence@gilmartinir.com Lexeo Therapeutics, Inc. Selected Condensed Financial Information (unaudited, in thousands, except share and per share amounts) Condensed Consolidated Statements of Operations Three Months Ended December 31, Year Ended December 31, 2023 2022 2023 2022 Revenue Grant revenue $- $- $- $654 Total revenue - - - 654 Operating expenses Research and development 8,210 11,437 53,130 49,162 General and administrative 6,764 3,760 15,383 12,001 Total operating expenses 14,974 15,197 68,513 61,163 Operating loss (14,974) (15,197) (68,513) (60,509)Other income and expense Loss on fair value adjustment to convertible SAFE Note (258) - (530) - Other income (expense), net (8) (1) (13) (2)Interest expense (51) (54) (205) (91)Interest income 1,103 675 2,867 1,325 Total other income and expense 786 620 2,119 1,232 Loss from operations before income taxes (14,188) (14,577) (66,394) (59,277)Income taxes - - - - Net loss and comprehensive loss $(14,188) $(14,577) $(66,394) $(59,277) Net loss per common share, basic and diluted $(0.86) $(8.86) $(12.40) $(36.36) Weighted average number of shares outstanding used in computation of net loss per common share, basic and diluted 16,438,237 1,644,403 5,354,368 1,630,348 Condensed Consolidated Balance Sheet Data December 31, December 31, 2023 2022 Cash, cash equivalents, and investments $121,466 $77,335 Total assets 139,807 97,076 Total liabilities 26,272 24,997 Total convertible preferred stock - 185,033 Total stockholders' equity (deficit) 113,535 (112,954)