Leonabio, Inc. · NASDAQ:LONA
Leonabio — Investor Presentation (March 2026)
41 slides from Leonabio's investor presentation, Mar 26, 2026
Slide 1: Corporate Presentation March 2026
Corporate Presentation March 2026 © LeonaBio, Inc.
Slide 2: Forward-looking statements
This presentation and any accompanying oral commentary contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements are based on our management’s beliefs and assumptions and on information currently available to our m
Slide 3: Management team with significant drug development and approval experience
Management team with significant drug development and approval experience LEADERSHIP Mark Litton, PhD President and CEO Mark Worthington, JD General Counsel Kevin Church, PhD CSO Javier San Martin, MD CMO Robert Renninger CFO Mark Kubik CBO Collectively involved in multiple developed products and ex
Slide 4: Expected key inflection points within ~2 years
Expected key inflection points within ~2 years Lasofoxifene Phase 3 registrational study ongoing with >50% enrolled ATH-1105 Phase 2 POC study in ALS planned to start in 2H2026 Topline results expected in 2H2027 Topline results expected 2027 © LeonaBio, Inc.
Slide 5: Late-stage program diversifies pipeline: opportunity in metastatic breast cancer
LATE-STAGE PROGRAM DIVERSIFIES PIPELINE Exciting opportunity in metastatic breast cancer Large Market Opportunity Differentiated Mechanism of Action Positive Phase 2 data Strategic Fit ER+/HER2- disease remains most common breast cancer subtype1 Represents approximately 70% of all breast cancers1 Gl
Slide 6: Lasofoxifene for the potential treatment of metastatic breast cancer
Small molecule selective estrogen receptor modulator (SERM) Lasofoxifene for the potential treatment of metastatic breast cancer © LeonaBio, Inc.
Slide 7: Lasofoxifene: Differentiated SERM for mESR1+ metastatic breast cancer
DESIGNED TO OVERCOME RESISTANCE, IMPROVE QOL, AND REDEFINE THE STANDARD OF CARE AS THE ENDOCRINE PARTNER OF CHOICE Key Differentiators: Potential best-in-class combination efficacy (13 months PFS)1 Recent outcomes (EMBER-3, EvERA) validate the combination approach to 2L+ mBC2,3 Active against WT and
Slide 8: Treatment-resistant ER+ breast cancer is a critical unmet need
Treatment-resistant ER+ breast cancer is a critical unmet need 1. Initial Tumor ER+ tumors depend on estrogen signaling via estrogen receptor (ESR1) to grow and survive 2. Therapy response (1L) 1L endocrine therapies (AIs, SERMs, SERDs) are highly effective at shrinking and stabilizing tumor cells 3
Slide 9: Lasofoxifene blocks ESR1 signaling even in the presence of mutations
Active ESR1 signaling drives tumor growth and metastasis in ER+ breast cancer1 Current endocrine therapies (SERDs, SERMs) aim to inhibit ESR1 signaling2 ESR1 mutations cause resistance to endocrine therapies3 Structural features of lasofoxifene inhibit mutant ESR1, overcoming resistance4,5 Lasofoxif
Slide 10: Outperforms SoC SERDs and SERMs across ESR1 mutations
OUTPERFORMS SOC SERDS AND SERMS ACROSS ESR1 MUTATIONS, SUSTAINING INHIBITORY POTENCY, AND DRIVING SUPERIOR ANTI-TUMOR ACTIVITY Lasofoxifene retains potency against ESR1 mutations, overcoming a key driver of endocrine resistance Affinity assay: Tested in luciferase reporter lines transfected with WT
Slide 11: Unique lasofoxifene MOA and pre-clinical data establish PoC in ESR1 mutations
Lasofoxifene reduces tumor growth in in vivo animal models and maintains potency in lines with mutated estrogen receptors1,2 Long half-life (6 days), excellent bioavailability, and volume of distribution optimizes receptor antagonism and tumor engagement2,3 No receptor degradation necessary and lack
Slide 12: ELAINE Program: Stepwise validation toward a new standard of care
ESTABLISHING LASOFOXIFENE AS THE NEXT-GENERATION ENDOCRINE BACKBONE FOR ER+ METASTATIC BREAST CANCER ELAINE Program: Stepwise validation toward a new standard of care Randomized Ph2 trial to demonstrate lasofoxifene’s targeted mechanism and establish its activity vs SoC fulvestrant in mutant ESR1+ m
Slide 13: ELAINE-1: Lasofoxifene clinical activity vs. fulvestrant
LASOFOXIFENE CLINICAL ACTIVITY VS. FULVESTRANT (SOC) IN ESR1-MUTANT BREAST CANCER IN A POC PHASE 2 STUDY ELAINE-1: Increased PFS and anti-tumor activity with lasofoxifene compared to SoC fulvestrant in mESR1 population HR, Hazard ratio Source: Goetz et. al, Annals of Oncology 2023. Clear efficacy ad
Slide 14: ELAINE-2: Lasofoxifene with abemaciclib in ESR1-mutant breast cancer
LASOFOXIFENE COMBINATION WITH CDK4/6I ABEMACICLIB IN ESR1-MUTANT BREAST CANCER, 100% PRIOR CDK4/6I (PHASE 2) ELAINE-2: Demonstrated potential best-in-class 13 months PFS with lasofoxifene combination with abemaciclib in a heavily pre-treated mESR1 population No head-to-head trials have been conducte
Slide 15: Emerging data support combination endocrine approaches
EMERGING DATA SUPPORT COMBINATION ENDOCRINE APPROACHES IN CDK4/6 INHIBITOR–PRETREATED ER+ BREAST CANCER In the 2L+ space, endocrine monotherapy approaches appear to reach a ceiling of ~6 months PFS Durable disease control is increasingly pursued through biomarker-driven combination strategies, rathe
Slide 16: ELAINE-3: FDA-aligned Phase 3 registrational trial
FDA-ALIGNED PH3 REGISTRATIONAL TRIAL TO ESTABLISH IMPROVED EFFICACY OF LASOFOXIFENE + ABEMACICLIB COMPARED TO CURRENT SOC COMBINATION ELAINE-3: Registrational trial of lasofoxifene in mESR1 population Adapted from Goetz et. al, Future Oncology, 2025. Stratification Factors: Visceral vs. non-visceral
Slide 17: Over 50% enrolled, expected readout in 2H2027
OVER 50% ENROLLED – EXPECTED TO READOUT IN 2H2027 Global study with >200 sites Europe, North America, and APAC (including China) Study is being executed by Medpace, a full-service global CRO with extensive experience running large, successful breast cancer trials Primary endpoint is determined by an
Slide 18: Well-tolerated with differentiated impact on quality of life
Lasofoxifene was generally well-tolerated and had a differentiated impact on women’s quality of life over standard of care in early trials VAS, vaginal assessment scale; VuAS, vulvar assessment scale Sources: 1. Goldfarb et al. ISSWSH Annual Meeting 2023 (Poster); 2. Sermonix Conf Presentation (Slid
Slide 19: Added clinical benefits over other endocrine therapies
Lasofoxifene exhibits added clinical benefits over other endocrine therapies important to breast cancer patients and clinicians No head-to-head trials have been conducted comparing combination lasofoxifene therapy to other endocrine therapies Sources: 1. McClung et al. American Society of Bone and M
Slide 20: Built on established biology, prior clinical signal, and SoC benchmarks
BUILT ON ESTABLISHED BIOLOGY, PRIOR CLINICAL SIGNAL, AND ESTABLISHED STANDARD-OF-CARE BENCHMARKS ELAINE-3 is a registrational Phase 3 program Comparisons are based on independently published study results. No head-to-head trials have been conducted comparing lasofoxifene combination regimens to SoC
Slide 21: Competitive advantages of Laso combo in 2L/3L ESR1-mutant BC
Competitive advantages of Laso combo in 2L/3L ESR1-mutant BC ER, estrogen receptor; SERD, selective estrogen receptor degrader; SERM, selective estrogen receptor modulator; ESR1 (gene), estrogen receptor 1; ORR, objective response rate; QoL, Quality of life; PFS, progression-free survival. Compariso
Slide 22: Lasofoxifene + abemaciclib vs. SERD + CDK4/6i regimens
LASOFOXIFENE + ABEMACICLIB vs. SERD + CDK4/6I REGIMENS Endocrine backbone choice drives long-term risk and differentiation Comparisons are based on independently published study results. No head-to-head trials have been conducted comparing combination lasofoxifene therapy to other combinations prese
Slide 23: Billion-dollar precision oncology opportunity in 2L/3L ER+ mBC
Lasofoxifene’s targeted activity in mutant ESR1 presents a billion-dollar precision oncology opportunity in 2L/3L ER+ mBC Sources: 1. Fortune Business Insights. Retrieved from https://www.fortunebusinessinsights.com/industry-reports/breast-cancer-therapeutics-market-100163; 2. LifeSci 1Q2023 Quantit
Slide 24: Orserdu sales illustrate the commercial opportunity
Orserdu sales illustrate the commercial opportunity for lasofoxifene’s precision oncology opportunity in 2L/3L ER+ mBC Sources: 1. IQVIA, RBC Capital Markets; 2. Bidard et al, J Clin Oncol 2022 Orserdu (elacestrant) achieved rapid commercial uptake, validating ESR1 mutation as a commercially viable
Slide 25: Potential backbone endocrine candidate with expansion potential
DRIVEN BY COMBINABILITY, TOLERABILITY, AND DIFFERENTIATED CLINICAL BENEFIT Lasofoxifene has the potential to be a backbone endocrine candidate with expansion potential beyond ESR1 mutant disease Treatment landscape schematic is illustrative and not intended to be comprehensive ESR1 and PIK3CA wild-t
Slide 26: A differentiated therapy to establish a new standard of care
A DIFFERENTIATED THERAPY DESIGNED TO OVERCOME RESISTANCE, IMPROVE QOL, AND ESTABLISH A NEW STANDARD OF CARE IN ER+ ESR1-MUTANT BREAST CANCER Lasofoxifene: Ongoing registrational Phase 3 trial with anticipated readout in 2027 Expansion potential as endocrine partner of choice Oral administration Larg
Slide 27: ATH-1105 for the potential treatment of ALS
Small molecule positive modulator of HGF ATH-1105 for the potential treatment of ALS © LeonaBio, Inc.
Slide 28: Slowing neurodegeneration in ALS is a critical unmet need
Slowing neurodegeneration in ALS is a critical unmet need Sources: 1. Packard Center. Retrieved from https://packardcenter.org/answer-als-partners-to-launch-the-end-als-challenge-digital-competition; 2. CDC. Retrieved from https://www.cdc.gov/als/php/abstracts-publications-reports/prevalence-2022-20
Slide 29: ATH-1105: A CNS-penetrant positive modulator of HGF
ATH-1105: A CNS-penetrant positive modulator of HGF as a potential treatment for ALS HGF, hepatocyte growth factor Sources: 1. Ishigaki et al., J Neuropathol Exp Neurol 2007; 2. Lee et al., Acta Neuropathol Commun 2019; 3. Vallarola et al., Int J Mol Sci 2020; 4. Berthiaume et al., Front. Neurosci 2
Slide 30: ATH-1105 enhanced MET activation and survival in ALS patient-derived motor neurons
CELLS DERIVED FROM FOUR PEOPLE WITH SPORADIC ALS ATH-1105 enhanced MET activation and survival in ALS-patient derived motor neurons MET activation: Data presented as mean ± SEM. Statistical significance was determined via paired t-test comparing MET activation (pMET/Total MET) in motor neurons treat
Slide 31: ATH-1105 preserved motor function and prolonged survival in ALS model
PRP-TDP43A315T TRANSGENIC MOUSE MODEL OF ALS ATH-1105 preserved motor function and prolonged survival in preclinical model of ALS Motor Function: Data presented as mean ± SEM. Statistics applied: 2-way ANOVA with the Dunnett’s test versus ALS + vehicle. ****p < 0.0001. n=10 mice per group Survival:
Slide 32: ATH-1105 preserved motor function and prolonged survival in ALS model
PRP-TDP43A315T TRANSGENIC MOUSE MODEL OF ALS ATH-1105 preserved motor function and prolonged survival in preclinical model of ALS Two months of treatment, Prp-TDP43A315T transgenic mice, “ALS” or “ALS mice.” WT = wild type (healthy control). Sciatic nerve sections stained with toluene blue to highli
Slide 33: Disease progression attenuated with early or delayed treatment
PRP-TDP43A315T TRANSGENIC MOUSE MODEL OF ALS Disease progression was attenuated in ALS mice following early or delayed treatment initiation with ATH-1105 Data presented as mean ± SEM. Statistics applied: 2-way ANOVA with the Dunnett’s test versus ALS + vehicle. **p < 0.01; ***p < 0.001; ****p < 0.00
Slide 34: Completed Phase 1 single and multiple dose studies in healthy volunteers
COMPLETED PHASE 1 SINGLE AND MULTIPLE DOSE STUDIES IN HEALTHY VOLUNTEERS Phase 1 results support continued development of ATH-1105 for the treatment of ALS Source: Ooi et al. NEALS Annual Meeting 2025 (Poster). All doses studied were generally well tolerated with no dose-limiting adverse events All
Slide 35: Exploratory analysis of neuronal-derived exosomes from Phase 1 studies
EXPLORATORY ANALYSIS OF NEURONAL-DERIVED EXOSOMES (NDE) IN THE PLASMA FROM PHASE 1 STUDIES Biomarker results indicated a biological effect of ATH-1105 in humans and suggest potential to improve protein pathology in ALS In vitro data: **p<0.01 vs. glutamate alone. One-way ANOVA with Fisher’s multiple
Slide 36: ATH-1105 is ready for a Phase 2 study in people living with ALS
ATH-1105 is ready for a Phase 2 study in people living with ALS Planning underway for a Phase 2 proof-of concept study to explore functional measures, biomarkers, and safety in people living with ALS Design considerations: ≥ 6 months treatment duration Biomarkers, including plasma NfL and NDEs Funct
Slide 37: A novel therapy designed to mitigate neurodegeneration in ALS
A NOVEL THERAPY DESIGNED TO ADDRESS THE COMPLEX PATHOLOGY AND MITIGATE NEURODEGENERATION IN ALS ATH-1105 is a Phase 2 ready asset for the treatment of ALS *in preclinical models Expansion potential Oral administration Neuroprotective* Favorable safety profile and generally well-tolerated Reduces pla
Slide 38: Transforming our future
Transforming our future Compelling clinical development programs in areas of profound need Multibillion-dollar market opportunities Key clinical readouts within 2 years Advancing 2 programs that have the potential to deliver life-changing therapies in breast cancer and ALS © LeonaBio, Inc.
Slide 39: PIPE financing up to $236 million
PIPE financing up to $236 million Financing up to $236M supports lasofoxifene development program through topline data with sufficient capital runway into 2028 $90M upfront priced at a greater than 50% premium to our December 17, 2025 closing price of $6.35/share If exercised, warrants provide up to
Slide 40: Expected key inflection points within ~2 years
Expected key inflection points within ~2 years Lasofoxifene Phase 3 registrational study ongoing with >50% enrolled ATH-1105 Phase 2 POC study in ALS planned to start in 2H2026 Topline results expected in 2H2027 Topline results expected 2027 © LeonaBio, Inc.
Slide 41: Thank You
Thank You ©LeonaBio, Inc. 41