Slides
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Unlocking the power of a patient’s own biology for the treatment of rare disease Investor Day October 2, 2025
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Welcome Dirk Thye, M.D. Chief Executive Officer & Chief Medical Officer
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3 Quince investor day agenda 10:00 a.m. ET Welcome, agenda, introductions Dirk Thye 10:05 a.m. ET Quince overview and key investment highlights Dirk Thye 10:20 a.m. ET Overview of innovative eDSP System Giovanni Mambrini 10:30 a.m. ET eDSP lead indication: A-T and Phase 3 NEAT study Dirk Thye 10:50 a.m. ET eDSP mechanism of action and RNA sequencing Caralee Schaefer 11:00 a.m. ET Regulatory overview and anticipated approval pathway Pamela Williamson 11:10 a.m. ET Commercial development and launch preparation Charles Ryan 11:20 a.m. ET Driving value creation Brendan Hannah 11:30 a.m. ET Question & answer session Leadership Team 12:00 p.m. ET Investor day concludes
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4 Seasoned leadership team Pamela Williamson, RAC, FRAPS, M.B.A. Head of Regulatory • 30+ years of regulatory affairs, quality assurance, pharmacovigilance, health authority compliance, and manufacturing operations experience • Alexion Pharmaceuticals, Genzyme Corporation, Serono/Ares-Serono Dirk Thye, M.D. CEO & CMO • 20+ years of experience in biotech executive leadership, company creation, R&D, and drug discovery • Agenovir, Cidara, Cerexa, Peninsula Charles Ryan J.D., Ph.D. President • 25+ years of experience in pharmaceuticals and biotech executive leadership, legal, IP, finance, and development • Forest Laboratories, Travecta, Neurotrope Brendan Hannah, M.B.A COO, CBO, & PFO • 15+ years of experience leading biotech business development, finance, and business operations • Led BD at Agenovir (acquired by Vir Biotech for up to $290 million) • Involved in $2+ billion in transactions Giovanni Mambrini, MSc CTO • 20+ years of medical device technology experience with cross-functional expertise in bringing complex programs to market • Co-founded EryDel with prior experience at Covidien, Dideco, Livanova Maureen Roden, M.S.N. VP, Clinical Development • 30+ years of drug development experience and executive leadership • Luna Consulting, BSPI, National Cancer Institute Gary Ward, Ph.D. VP, CMC • 30+ years managing chemistry, manufacturing, controls, and product development operations • Expert in broad range of NCE types/product dosage forms • Pfizer, Dura Pharma, Chugai Biopharma, 3M Company Stacy Roughan VP, Communications & IR • 25+ years leading comprehensive strategic communications and investor relations programs • Expert at crisis and activist situations, M&A transactions, and financings • NuVasive, Valeant Pharmaceuticals, Ribapharm Mary Ellen Sillivos VP, Human Resources • 30+ years of human resources experience in the biopharmaceutical and biotech industry • Dermira, Hyperion, Affymax Caralee Schaefer, Ph.D. VP, Nonclinical Development • 25+ years of nonclinical development experience, with particular focus on inflammation, autoimmunity, fibrosis, and rare disease research • 4DMT, Cortexyme, Indalo Tx, Blade Therapeutics, InterMune, Berlex Biosciences Brent Roeck VP, Program & Alliance Management • 25+ years of clinical operations, program, and alliance management experience with focus on oncology and rare disease • Reneo, Spectrum, Amgen, Salmedix, Agouron/Pfizer, Parexel
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Quince overview and key investment highlights Dirk Thye, M.D. Chief Executive Officer & Chief Medical Officer
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6 Forward-looking statements Statements in this presentation contain “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 as contained in Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, which are subject to the “safe harbor” created by those sections. All statements, other than statements of historical facts, may be forward-looking statements. Forward-looking statements contained in this presentation may be identified by the use of words such as “believe,” “may,” “should,” “expect,” “anticipate,” “plan,” “believe,” “estimated,” “potential,” “intend,” “will,” “can,” “seek,” or other similar words. Examples of forward-looking statements include, among others, statements relating to the timing, success, and reporting of results of the clinical trials and related data, including expected timing of Phase 3 NEAT topline results and submission of a related NDA; expected cash position and operating runway, including cash potentially receivable upon the exercise of warrants; current and future clinical development of eDSP, including for the potential treatment of Ataxia-Telangiectasia (A-T), Duchenne muscular dystrophy (DMD), and other potential indications; the strategic development path for eDSP, including the anticipated benefits of the strategic partnership with Option Care Health; planned regulatory agency submissions and clinical trials and timeline, prospects, and milestone expectations; and the potential benefits of eDSP and the company’s market opportunity. Forward-looking statements are based on Quince’s current expectations and are subject to inherent uncertainties, risks, and assumptions that are difficult to predict and could cause actual results to differ materially from what the company expects. Further, certain forward-looking statements are based on assumptions as to future events that may not prove to be accurate. Factors that could cause actual results to differ include, but are not limited to, the risks and uncertainties described in the section titled “Risk Factors” in the company’s Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC) on March 24, 2025, Quarterly Report on Form 10-Q filed with the SEC on May 13, 2025, Quarterly Report on Form 10-Q filed with the SEC on August 11, 2025, and other reports as filed with the SEC. Forward- looking statements contained in this news release are made as of this date, and Quince undertakes no duty to update such information except as required under applicable law.
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7 eDSP value proposition A Autologous I Intracellular D Drug E Encapsulation Red blood cell encapsulated dexamethasone sodium phosphate – eDSP – designed to chronically deliver corticosteroid efficacy without toxicity
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8 Quince Therapeutics key investment highlights Pivotal Phase 3 NEAT clinical trial in pediatric rare disease Ataxia-Telangiectasia (A-T) with no currently approved treatments and $1+ billion* commercial opportunity Significant pipeline expansion opportunity for eDSP with Duchenne muscular dystrophy (DMD) as targeted second indication with multiple other rare immunology and autoimmune focused targets NEAT enrollment complete with total of 105 participants – including 83 in six to nine year-old primary analysis population and 22 aged 10 years or older. Topline data in first quarter of 2026 Entered strategic partnership with Option Care Health – largest independent provider of home and outpatient infusion services in U.S. – to provide for administration of lead asset eDSP $34.7 million in cash provides funding through topline results in first quarter of 2026 and into the second quarter of 2026 – or second half of 2026 if warrants exercised *$1+ billion estimated global peak commercial opportunity is based on IQVIA Medical Claims (Dx), PharmetricsPlus (P+), and IQVIA Analytics in the U.S. and the company's internal estimates and assumptions outside the U.S.
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9 eDSP designed to deliver corticosteroid efficacy without toxicity One-touch, fully automated, and sterile Autologous Intracellular Drug Encapsulation (AIDE) device Corticosteroids encapsulated in autologous red blood cells designed to significantly alter drug characteristics eDSP designed to deliver corticosteroids in patient’s own red blood cells – distinct from standard cell or gene therapy Designed to deliver corticosteroid efficacy without toxicity and adrenal suppression associated with chronic corticosteroid use 20+ years of research & development and $100 million invested in AIDE technology
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10 eDSP encapsulates dexamethasone sodium phosphate (DSP) in patient’s red blood cells for once monthly treatment Approximately 90-minute procedure administered at point of care eDSP RBCs exposed to hypotonic solutions Blood removed from patient (50mL) Membranes become “leaky” DSP added - non diffusible through RBC membrane1 2 3 DSP enters RBC Membranes reseal “capturing” DSP to create eDSP 4 5 Administered to patient6 Intracellular enzymes remove phosphate7 DSP slowly released while blood circulates through tissues 8
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11 0 5 10 15 20 25 30 Plasma concentration (ng/mL) 1,000 100 10 1 0.1 0.01 0.001 Note: Information represented does not reflect a completed comparative study of eDSP versus oral/IV administration of dexamethasone but rather provides a comparison of published corticosteroid pharmacokinetic information relative to company data regarding eDSP. IC50 and SC50 refer to pharmacodynamic parameters of which IC50 reflects drug concentration eliciting 50% of the maximum inhibition and SC50 reflects drug concentration eliciting 50% of the maximum stimulation. References: Krzyzanski, Journal of Pharmacokinetics and Pharmacodynamics (2021) 48: 411-438; Aljebab, PLoS ONE 12(1):e0170259. doi:10.1371/journal.pone.0170259, January 26, 2017; Montanha et al, Frontiers in Pharmacology (2022) 13: 814134. 11 Glucose sensitivity parameter (SC50) – 17.9 ng/mL Lymphocyte sensitivity parameter (IC50) – 14 ng/ml Cortisol sensistivity parameter (IC50) – .05 ng/mL Pharmacokinetics of daily IV dexamethasone (6 mg) Corticosteroid therapy consistently exceeds toxicity thresholds Why are conventional corticosteroids toxic? • Low cortisol levels • Cushingoid appearance • Hirsutism • Loss of bone mineral density • Growth retardation in pediatrics • Delay in puberty Adrenal suppression Elevated glucose resulting in hyperglycemia and diabetes Immunosuppression resulting in infections Well-described toxicity of corticosteroids
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12 Followed by continuous exposure to maintain GC binding to GR How can potential efficacy be maintained without toxicity? Principles of corticosteroid delivery resulting in optimal efficacy Clinical efficacy depends on the magnitude and duration of exposure of glucocorticoid receptors (GRs) to glucocorticoid (GC) Optimal results achieved by initial bolus dose to achieve GR saturation Long tail of eDSP exposure avoids adrenal suppression Reference: Meduri GU, et al. Intensive Care Med. 2020;46(12):2284-2296.
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13 0 5 10 15 20 25 30 Days Average plasma concentration (ng/mL) 1,000 100 10 1 0.1 0.01 0.001 Average concentration eDSP monthly dose = 17.4 mg Sustained Efficacy Window Note: Pharmacokinetic (PK) curve from Population PK model (smoothed) based on company’s prior studies of eDSP. IC50 and SC50 refer to pharmacodynamic parameters of which IC50 reflects drug concentration eliciting 50% of the maximum inhibition and SC50 reflects drug concentration eliciting 50% of the maximum stimulation. References: Montanha et al, Frontiers in Pharmacology (2022) 13: 814134; Krzyzanski et al, Journal of Pharmacokinetics and Pharmacodynamics (2021) 48: 411-438. 13 Glucose sensitivity parameter (SC50) – 17.9 ng/mL Lymphocyte sensitivity parameter (IC50) – 14 ng/ml Cortisol sensistivity parameter (IC50) – .05 ng/mL eDSP designed to optimize dexamethasone delivery through once monthly dosing in patients with A-T Average concentration-time profile for plasma dexamethasone in eDSP
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1414 Clinical efficacy demonstrated in multiple indications Market value - $16.5B eDSP dose – avg 8.8mg EFFICACY 78% discontinued steroids, 44% endoscopic remission, pCDAI declined, reduced hospitalization SAFETY No serious AEs Crohn’s Disease Market value - $12.6B eDSP dose – avg 8.9mg EFFICACY eDSP: improvement in FEV1 (p=0.0002) and infection rate over placebo SAFETY No steroid AEs over 15 month Cystic Fibrosis Market value - $11.5B eDSP dose – avg 0.8-8.8mg EFFICACY Cessation of corticosteroids and β2-agonists, improved symptoms and physician- assessed signs SAFETY No steroid AEs Chronic Obstructive Pulmonary Disease Market value - $7.6B eDSP dose – avg 9.9mg EFFICACY eDSP: 68% off steroids Sham: 22% off steroids SAFETY eDSP: 36% with steroid symptoms at baseline, had residual AEs Sham: 100% had steroid AEs Ulcerative Colitis Sources: EvaluatePharma for market value estimates. Study data: Rossi et al., 2001. Rossi et al., 2004. Lucidi et al., 2006. Bossa et al. 2008. Castro et al., 2007. Bosa et al., 2013.
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15 Significant upside potential accompanies Phase 3 NEAT clinical trial success *$1+ billion estimated global peak commercial opportunity and estimated A-T patient population are based on IQVIA Medical Claims (Dx), PharmetricsPlus (P+), and IQVIA Analytics in the U.S. and the company’s internal estimates and assumptions outside the U.S. Ability to deliver corticosteroids without toxicities holds potential to transform treatment paradigms across multiple diseases Positions eDSP as potential first-to- market therapeutic in highly attractive $1+ billion* A-T opportunity Validates ability to deliver corticosteroids chronically without toxicities Potential to transform treatment paradigms across rare diseases where corticosteroids are already standard of care Significant pipeline expansion opportunities with DMD selected as second targeted eDSP indication Strong history of clinical efficacy across multiple indications and highly leverageable AIDE technology platform
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Innovative eDSP System Giovanni Mambrini, MSc Chief Technology Officer
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17 20+ years of R&D and $100 million invested in AIDE technology Dr. Mauro Magnani, (University of Urbino, Italy) files patent for compound encapsulation in RBCs EryDel S.p.A. founded to advance RBC encapsulation technology and start clinical development Red Cell Loader (RCL) obtains CE mark in Europe Initiated Phase 2 open label extension study in A-T patients Granted orphan drug designation in U.S. IND opened in U.S. and orphan drug designation granted in European Union Completed Phase 1 PK and RBC survival in circulation studies in healthy volunteers Initiated randomized- controlled Phase 3 ATTeST study in A-T patients Initiated Phase 3 ATTeST open label extension study Completed Human Factors program Secured Special Protocol Assessment (SPA) agreement with FDA for Phase 3 NEAT study in A-T patients Quince Therapeutics acquires EryDel S.p.A. Initiated Phase 3 NEAT study in A-T patients Entered strategic relationship with Option Care Health to support commercial development 1998 2008 First human use of RCL on COPD patients 2010 2011 2017 2018 2021 2022 2023 2024 Completed enrollment of Phase 3 NEAT study in A-T patients 2025 1Q26 Expect topline results for Phase 3 NEAT study in A-T patients 2012/2013 2015 More than 7,800 infusions administered to patients with A-T to date
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eDSP System demonstration
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19 Long history of eDSP administration eDSP Includes clinical study of eDSP across lead indication A-T, Crohn’s disease, ulcerative colitis, COPD, and cystic fibrosis >425 participants have received at least one dose >240 of those were patients with A-T >7,800 infusions administered to patients with A-T
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20 Innovative drug/device combination well-positioned given rigorous regulatory review to date Secured CE mark in Europe – covers RCL and treatment kit which are approved for clinical trial use in Europe Completed extractables and leachables testing of treatment kit – plastic bags/tubing changed to be compliant with European regulations eDSP System design ensures sterility throughout encapsulation process – automates 17-step process handling blood, drug, and processing solutions High quality of eDSP processed RBCs – similar to blood transfusion with mean in-vivo life span of 84.3 days Active Investigational New Drug application for 10+ years – successfully responded to FDA questions and requests throughout 20
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eDSP lead indication: Ataxia-Telangiectasia Dirk Thye, M.D. Chief Executive Officer & Chief Medical Officer
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22 No currently approved treatments for A-T patients A-T is an inherited rare neurodegenerative and immunodeficiency disorder caused by mutations in ATM gene Estimated prevalence of approximately 10,000 patients with A-T in U.S., U.K., and EU4 countries* Neurological symptoms worsen until patients are wheelchair dependent, usually by age 12 – with teenage years typically marked by repeated infections, pulmonary impairment, and malignancies. Median lifespan of approximately 25-30 years Patients with A-T cannot take standard steroids due to immunodeficiency and risk of lethal infections posed by immunosuppressive drug Currently no approved treatments for A-T and no currently known effective approaches to delay progression of disease *Patient population based estimated A-T patient population are based on IQVIA Medical Claims (Dx), PharmetricsPlus (P+), and IQVIA Analytics in the U.S. and the company’s internal estimates and assumptions outside the U.S.
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An A-T patient’s journey Meet Shane 2 3
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24 Gross motor symptoms stop progressing after ages 12-15 Neurological manifestations in classic A-T Marked by gradual loss of mobility 5 Walking begins at a normal age, but gait fails to improve Difficulty standing or sitting still, sway side- to-side or backwards Walking becomes more difficult, walls or doorways used as support Gait is quick, with a narrow stance 10 Progression to wheelchair use Increased difficulty reading due to impaired eye movement Increased risk of silent aspiration 15 At any age, patients with A-T develop increasing difficulty with: Involuntary movements | Chewing and swallowing | Articulation 101-2 References: 1. Rothblum-Oviatt C, et al. Orphanet J Rare Dis. 2016;11(1):159. doi:10.1186/s13023-016-0543-7. 2. Crawford TO. Semin Pediatr Neurol. 1998;5(4):287-294. Age
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25 Encouraging eDSP Phase 3 clinical trial results in prior ATTeST study 25 25 Completed largest global study of A-T patients in Phase 3 ATTeST clinical trial and OLE • Double-blind, placebo-controlled study (N=175) over 6-month treatment period with 12-month OLE (N=104) • Patients randomized 1:1:1 to eDSP low dose, high dose, or placebo • Primary endpoint: mICARS – Secondary endpoints: CGI-C • QOL • VABS eDSP ATTeST Trial Study observed eDSP slowed neurological deterioration in all ages of patients with A-T – with improvement observed in 6- to 9-year-old subgroup across multiple endpoints 12-month safety analysis - eDSP was well tolerated with no serious safety concerns typically associated with chronic corticosteroid administration mICARS = Modified International Cooperative Ataxia Rating Scale • CGI-C = Clinical Global Impression – Change • QOL = Quality of Life • VABS = Vineland Adaptive Behavior Scales • Company ATTeST clinical trial: ClinicalTrials.gov ID: NCT02770807
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26 ICARS primary endpoint measurement criteria Full ICARS 100 points 19 Items mICARS 54 points 11 items Rescored mICARS (FDA) 29 Points 9 items Posture and Gait Disturbance (34 points) Posture and Gait Disturbance (34 points) Posture and Gait Disturbance (23 points) 1. Walking capacities 0-8 1. Walking capacities 0-8 1. Walking capacities 0-4 2. Gait Speed 0-4 2. Gait Speed 0-4 2. Gait Speed 0-3 3. Standing Capacities eyes open 0-6 3. Standing Capacities eyes open 0-6 3. Standing Capacities eyes open 0-4 4. Spread of feet eyes open 0-4 4. Spread of feet eyes open 0-4 4. Spread of feet eyes open 0-3 5. Body sway feet together eyes open 0-4 5. Body sway feet together eyes open 0-4 5. Body sway feet together eyes open 0-3 6. Body sway feet together eyes closed 0-4 6. Body sway feet together eyes closed 0-4 6. Body sway feet together eyes closed 0-3 7. Quality of sitting position 0-4 7. Quality of sitting position 0-4 7. Quality of sitting position 0-3 Kinetic Function (52 points) test left & right except drawing Kinetic Function (12 points) test left & right except drawing Kinetic Function (2 points) test left & right except drawing 8. Knee tibia test 0-4 9. Action tremor 0-4 10. Finger to nose test (dysmetria) 0-4 11. Finger to nose test (intention tremor) 0-4 12. Finger finger test 0-4 13. Pronation supination 0-4 13. Pronation supination 0-4 14. Drawing 0-4 14. Drawing 0-4 14. Drawing 0-2 Speech Disorder (8 points) Speech Disorder (8 points) Speech Disorder (4 points) 15. Fluency of speech 0-4 15. Fluency of speech 0-4 16. Clarity of speech 0-4 16. Clarity of speech 0-4 16. Clarity of speech 0-4 Oculomotor Disorders (6 points) 17. Gave evoked nystagmus 0-3 18. Abnormalities of ocular pursuit 0-2 19. Dysmetria of the saccade 0-1 Total 0-100 Total 0-54 Total 0-29 ICARS = International Cooperative Ataxia Rating Scale • mICARS = Modified International Cooperative Ataxia Rating Scale • RmICARS = Rescored Modified International Cooperative Ataxia Rating Scale
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27 Rapid neurological progression in A-T *Scores based on the Crawford Quantitative Neurologic A-T Scale (100=normal). References: Rothblum-Oviatt C, et al. Orphanet J Rare Dis. 2016;11(1):159; Crawford TO, et al. Neurology. 2000;54(7):1505–1509. Symptoms progress quickly from 6 to 9 years of age Active decline Plateau
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28 Encouraging eDSP Phase 3 clinical trial results in prior ATTeST study eDSP ATTeST Trial ATTeST ICARS Values in ITT Population* Change vs. Placebo *Company ATTeST clinical trial data (ClinicalTrials.gov ID: NCT02770807) presented reflect nominal p-values for ICARS values in ITT populations. Values reflect Least Square Means (LSM) difference from placebo and the P value presented • ICARS = International Cooperative Ataxia Rating Scale • mICARS = Modified International Cooperative Ataxia Rating Scale • RmICARS = Rescored Modified International Cooperative Ataxia Rating Scale Improvement observed in 6 to 9 years old subgroup* At 12 months, eDSP well tolerated with no serious safety concerns 3+ years of ATTeST OLE observed no serious safety concerns Clinically important delay in progression at 6 months -7 -6 -5 -4 -3 -2 -1 0 ICARS mICARS RmICARS All Ages (n=58) 6-9 Years (n=33) P=0.020 P=0.002 P=0.009 Phase 3 NEAT Primary Endpoint
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29 Improvement observed in 6 to 9 year-old subgroup across multiple endpoints in prior ATTeST study eDSP ATTeST Trial ATTeST ICARS Mean % Change in 6 to 9 Year-Old ITT Population* Worsening of Symptoms *Company ATTeST clinical trial data (ClinicalTrials.gov ID: NCT02770807) presented reflect nominal p-values for ICARS values in ITT populations. Values reflect Least Square Means (LSM) difference from placebo and the P value presented • ICARS = International Cooperative Ataxia Rating Scale • mICARS = Modified International Cooperative Ataxia Rating Scale • RmICARS = Rescored Modified International Cooperative Ataxia Rating Scale eDSP slowed progression in 6 to 9 years old patients Magnitude of change in endpoints considered by investigators to be highly clinically significant 0% 0% -4% -13% -19% -28%-30% -20% -10% 0% 10% ICARS mICARS RmICARS High Dose (n=29) Placebo (n=29) P=0.020 P=0.002 P=0.009 Phase 3 NEAT Primary Endpoint
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30 ATTeST: Initial Treatment Period ATTeST: Through Month 12 Low Dose (N=59) High Dose (N=57) Placebo (N=59) Low Dose (N=59) High Dose (N=57) Non-switch Placebo (N=19) Patients With Any TEAE (%) 73% 82% 73% 76% 88% 79% Patients With Any Treatment–Related TEAE (%) 25% 37% 25% 32% 44% 26% Patients With Any Serious TEAE (%) 10% 12% 12% 14% 16% 21% Patients With Any Serious Treatment–Related TEAE (%) 0 2% 0 2% 2% 5% Patients With Any TEAE Leading to Discontinuation (%) 0 4% 0 2% 4% 0 Patients With Any TEAE Leading to Death (%) 0 0 0 0 0 0 Safety TEAEs of prior Phase 3 ATTeST study eDSP ATTeST Trial TEAE = Treatment Emergent Adverse Event • Company ATTeST clinical trial: ClinicalTrials.gov ID: NCT02770807 Three patients discontinued from the study, all unrelated to corticosteroid toxicity Two patients in high-dose group showed corticosteroid-related TEAEs of pyrexia and tachycardia/pain and pruritus
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31 September 2024 January 2025 Recent publications in scientific journals References: Zielen, Stefan et al. The Lancet Neurology. Volume 23, Issue 9, 871 – 882. Koenig MK, et al. Frontiers in Neurology. 15:1526914, DOI=10.3389/fneur.2024.1526914.
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32 Pivotal Phase 3 NEAT designed using valuable lessons learned Encouraging, consistent data in 6 to 9 year-old subgroup Encouraging positive data in the per-protocol population Challenges related to COVID Primary efficacy endpoint in 6 to 9 year-old subgroup (agreed with FDA within context of SPA agreement) Robust risk mitigation system developed based on lessons learned; India not included in study Treatment windows shortened and site training improved to enhance compliance ATTeST precedent NEAT optimized
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33 Pivotal Phase 3 NEAT study design Screening Baseline visit Six month treatment period Study completion visit Safety follow up visit Placebo eDSP Safety follow up Safety follow up ~ 86 Patients Primary efficacy endpoint – RmICARS • RmICARS measures primarily focused on posture and gait disturbance Completed enrollment in July 2025 with 105 total participants • 83 participants ages 6 to 9 years old comprise primary analysis population • 22 participants ages 10 years or older also included in study • Provides ~90% power to determine statistical significance • Participants eligible for open label extension (OLE) study – with all participants to date electing to transition to OLE Multicenter, randomized, double-blind, placebo-controlled clinical trial with six infusions scheduled once every 21 to 30 days • Topline data expected in first quarter of 202 6 RmICARS = Rescored Modified International Cooperative Ataxia Rating Scale • Company NEAT clinical trial: ClinicalTrials.gov ID: NCT06193200 Pivotal study being conducted under Special Protocol Assessment (SPA) agreement with FDA • NDA submission, assuming positive results, following this single global Phase 3 clinical trial
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34 NEAT* ATTeST Missed doses Discontinuation rate Screen failure rate Dosing within study window OLE rollover rate Missing data NEAT metrics of data integrity are high 1% 3.8% 14% 93% 21-30 days 100% 5% Excluding Covid 24% 26% 70% 21-38 days 96% 6% 26% *Data as of September 2025
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35 eDSP pediatric clinical trial initiated 35 Initiated pediatric investigational plan in Europe to evaluate the safety and pharmacokinetics of eDSP in smaller patients with A-T Eligible participants to weigh between 9 and 15 kilograms – estimated to be between 2 and 5 years- old – who will be administered dose of ~12.5mg DSP processed with 30mL of autologous blood Pediatric Encapsulated Dexamethasone Sodium Phosphate (PeD) Study Study agreed to with European Medicines Agency’s Paediatric Committee to support potentially broader indication in Europe and U.S. Could allow for treatment as soon as patients are diagnosed with A-T to hopefully mitigate progression of the disease at earliest onset possible
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36 Strong relationship with A-T patient advocacy groups 36 UNITED STATES UNITED KINGDOM ITALY FRANCEAUSTRALIA SPAIN
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37 NEAT trial design supports expectation of clear topline results Appropriate primary analysis population to demonstrate therapeutic effect – includes 6 to 9 year-olds when rate of neurological decline most apparent and potentially severe Well-defined primary endpoint – RmICARS as recommended by FDA to demonstrate clinical benefit, focusing largely on gait and posture Sufficiently powered at approximately 90% to determine statistical significance Inclusion of patients aged 10 years and older – recommended by FDA Pivotal trial being conducted under SPA agreement with FDA High level of data integrity – significantly better rates of compliance, fewer missed doses, discontinuations, missing data versus prior ATTeST study High level of compliance – further supports expectation of clear topline results 37
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eDSP mechanism of action and RNA sequencing Caralee Schaefer, Ph.D. Vice President, Nonclinical Development
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eDSP mechanism of action
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40 A-T is caused by mutations in the ATM gene leading to a deficiency in the ATM protein Characterized by progressive cerebellar degeneration, ataxia, telangiectasia, immunodeficiency, recurrent infections, and predisposition to cancer Progressive loss of neurons is a hallmark of A-T • Cerebellar Purkinje neurons Defective response to DNA double strand breaks and oxidative stress • Elevated ROS and altered cellular redox status Chronic activation of NF-κB, elevated pro-inflammatory cytokines, neuroinflammation, and immune dysregulation Pathogenesis of A-T: defects in DNA repair, redox/mitochondria, and immune regulation Dysfunction of ATM leads to genomic instability, chronic inflammation, neuroinflammation, and neurodegeneration Mutation in ATM Gene Loss of ATM protein function DNA Damage & Genomic Instability Impaired DNA double- strand break repair Oxidative Stress Increased free radicals and oxidative damage Neurodegeneration Mitochondria Dysfunction Inflammation & Neuroinflammation Chronic activation of inflammatory pathways Ataxia Telangiectasia References: Rothblum-Oviatt, 2016. Orphanet J Rare Dis. 11:159. Amirifar, 2019. Ped Allergy Immunol. 30(3):277-288. Ambrose, 2013. Blood. 25;121(20):4036–4045. Shiloh, 2003. Nature Rev Mol Cell Biol, 4(3): 177–187.
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41 Investigating eDSP mechanism of action and treatment responsive biomarkers by RNA sequencing ATTeST Phase 3 Trial eDSP Month 2 Month 3 Month 4 Month 6Month 1 eDSP eDSPeDSPeDSPeDSP Month 5 Baseline Whole blood for RNAseq Whole blood for RNAseq (TROUGH) Whole blood for RNAseq (TROUGH) Placebo (n=29) eDSP High Dose (n=33) ATTeST RNAseq (n=289) eDSP Low Dose (n=35) Baseline, Month 2, and Month 6 (TROUGH samples) Healthy adult controls (n=10) added for comparison to patients with A-T baseline samples
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42 RNA sequencing workflow for ATTeST samples 42 RNA extraction Library preparation Illumina stranded total RNA prep with Ribo- Zero Plus Sequencing Illumina Data Analysis and Bioinformatics Differentially Expressed Genes and Pathway Analysis Pax Gene Tubes ATTeST clinical samples collected at trough Whole blood collection Bioinformatics • Differential gene expression and pathway analysis RNA extraction • Great RNA quality in 96% of samples RNA sequencing successfully completed (n=289) 1 2 3 Whole blood collected at Baseline, Month 2, and Month 6 (TROUGH)
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43 Significant Dysregulation of Genes in A-T: DNA repair and redox response genes Humoral immune response, inflammation & neuroinflammation • Significantly lower expression of genes involved in V(D)J recombination and T cell development • Upregulation of pro-inflammatory genes Interferon-stimulated genes • Marked upregulation; potential biomarker of disease activity • Contributes to chronic inflammation Ion channel genes, neurotransmitters & mitochondrial dysfunction Identified differentially expressed genes (DEGs) in A-T samples compared to 10 healthy adult controls • More than 6,000 genes differentially expressed in A-T samples RNA sequencing generates comprehensive molecular characterization of patients with A-T DOWNREGULATED: UPREGULATED: >6,000 Differentially Expressed Genes A-T vs Healthy Controls Landmark RNA sequencing dataset from the largest clinical trial ever conducted in A-T
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44 RNA sequencing results reveal novel insights into eDSP mechanism of action & potential biomarkers RNA sequencing shows evidence of methylprednisolone gene signature and additional disease relevant gene modulation Interferon-stimulated genes • Significant downregulation; potential biomarkers of disease activity Immune response, inflammation, and oxidative stress genes • Enrichment of humoral immune response • Significant downregulation of pro-inflammatory genes Neuroprotective genes • Wnt activation has been shown to be neuroprotective in AD, PD, ALS, TBI • Activates anti-apoptotic genes, reduces oxidative stress, upregulates DNA repair and cell cycle checkpoint genes, promotes neuronal survival and neurogenesis post-injury Ion channel genes • Critical role in maintaining membrane potential, regulating calcium signaling, and modulating cell activation • ATM deficiency disrupts calcium homeostasis, making Purkinje neurons more vulnerable to excitotoxicity and oxidative stress • Purkinje neurons rely on voltage-gated ion channels (e.g., sodium, potassium, calcium) to generate and regulate action potential s Differentially Expressed Genes after eDSP High Dose Treatment eDSP downregulates IFN-alpha genes eDSP downregulates IFN-gamma genes
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45 eDSP demonstrates classic glucocorticoid gene signature and unique treatment responsive biomarkers Source: RNA sequencing from eDSP high-dose at 2 months (trough sample) Suppression of interferon-stimulated genes (ISGs) • Hallmark steroid activity Downregulation of inflammation • Reduction in NF-κB dependent pro-inflammatory cytokines Upregulation of mitochondrial genes • Mitochondrial dysfunction drives neurodegeneration in A-T • Restores energy metabolism and function, consistent with neuroprotective effects Upregulation of neurotransmitters & neuroprotective genes • Suppresses reactive oxygen species (ROS) and reduces oxidative damage • Promotes synaptic resilience and neuronal survival Modulation of ion channel genes • Excessive Ca²⁺ flux = mitochondrial stress, oxidative damage, excitotoxic neuronal death • Regulates neuronal excitability and calcium homeostasis • Stabilizes neuronal firing and protects against Purkinje cell dysfunction Landmark transcriptomic data generated from the largest A-T study completed to date
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46 GOALS RESULTS Expand knowledge of A-T biology • Treasure trove of transcriptome data generated from the largest A-T trial to date • A-T samples compared to healthy adult controls – more than 6,000 genes differentially expressed • Potential biomarkers of disease activity identified Demonstrate eDSP treatment has a biological effect • Novel insights into eDSP mechanism of action and potential treatment responsive biomarkers • Significant number of DEGs upregulated and downregulated by eDSP • Patterns of gene expression consistent between 2 and 6 month timepoints • Gene expression evaluated at trough (~30 days post infusion) – confirming sustained and durable biological activity of eDSP throughout treatment period Show magnitude of changes meaningful when compared to other steroids • Pathway analyses show evidence of a methylprednisolone gene signature RNA sequencing pathway analysis reveals novel insights into eDSP mechanism of action and potential biomarkers Next Steps • Potential disease-specific biomarkers and treatment responsive biomarkers (ongoing) • Correlation of gene expression with clinical outcomes and genetic mutations (ongoing) • RNA sequencing results confirmed by orthogonal method using long-read DNA sequencing (complete)
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47 eDSP eDSP has multiple synergistic mechanisms of action References: Crinelli, 2000. Blood Cells, Molecules, and Diseases; 26 (3): 211-222; doi: I 0.1006/bcmd.2000.0298. Meduri, 2020. Intensive Care Med; 46: 2284–2296; doi: 10.1007/s00134-020- 06289-8; Buttgereit, 2002; Ann Rheum Dis 2002; 61: 718±722; doi: 10.1136/ard.61.8.718. Wang, 2024. Neurobiol Stress; 28: 100593; doi: 10.1016/j.ynstr.2023.100593. eDSP Characteristic Mechanism of Action Clinical Impact Optimized GC Receptor Occupation Targeted Biodistribution to CNS Optimized Pharmacokinetics eDSP’s unique PK profile enables sustained and targeted GC activity, enhancing genomic and non-genomic effects Initial high exposure achieves near maximal GRα saturation followed by continuous release of DSP that maintains receptor occupancy throughout treatment period Avoids severe toxicities while maintaining anti-inflammatory effects (Improving safety and efficacy) Maximizes drug exposure at non-toxic levels (Improving efficacy) Improves DSP delivery and biodistribution (Improving efficacy) eDSP can migrate through small/tight capillaries in tissues, including blood-brain barrier Delayed response; GRα translocates to the nucleus, modulating gene expression via NFκB pathways Rapid (seconds to minutes) anti-inflammatory regulation, including inhibition of neutrophil degranulation and vasomotor regulation; ion channel modulation induces neuronal calcium homeostasis Genomic Glucocorticoid Effects Drives long-term, anti-inflammatory and immunomodulatory effects (Improving efficacy) Non-genomic Glucocorticoid Effects Immediate anti-inflammatory and immunomodulatory effects (Improving efficacy)
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Regulatory overview and approval pathway Pamela Williamson, RAC, FRAPS, M.B.A. Head of Regulatory Affairs
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49 Regulatory overview and anticipated approval pathway Orphan drug designations granted by U.S. FDA and European Union EMA for treatment of A-T Provides benefits such as waiver of certain application fees, marketing exclusivities eDSP System devices and single-use treatment kit are CE marked in European Union, derisking evaluation of device components Fast Track designation granted by U.S. FDA for treatment of A-T eDSP System regulated as drug/device combination product by U.S. FDA Center for Drug Evaluation and Research (CDER) primary NDA review division with consults from Center for Devices and Radiological Health (CDRH) and Center for Biologics Evaluation and Research (CBER)
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50 Regulatory overview and anticipated approval pathway (cont.) Phase 3 NEAT trial conducted under a U.S. FDA SPA agreement Includes primary endpoint of RmICARS and primary efficacy population 6 to 9 years-old European Union Pediatric Investigational Plan (PIP) PIP study #3 evaluates safety and PK in smaller patients with A-T who weigh between 9 and 15 kilograms as agreed with agency’s Paediatric Committee U.S. NDA submission expected in second half of 2026 Plan to request Priority Review U.S. 505(b)(2) regulatory pathway Allows for reliance in part on FDA’s prior findings of safety and efficacy for active pharmaceutical ingredient dexamethasone sodium phosphate
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Commercial development and launch preparedness Charles Ryan, J.D., Ph.D. President
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52 Attractive commercial opportunity for eDSP for A-T $1+ billion estimated global peak commercial opportunity for A-T indication alone Estimated prevalence of approximately 10,000 patients with A-T* in U.S., U.K., and EU4 countries with currently no approved therapies for A-T First-to-market potential with plans to commercialize in U.S. Orphan drug designation, strong patent protection, and technology barriers expected to provide long-term market exclusivity Attractive rare disease launch and pricing analogs with recently approved treatments Highly scalable manufacturing infrastructure in place with low direct cost of goods *$1+ billion estimated global peak commercial opportunity and estimated A-T patient population are based on IQVIA Medical Claims (Dx), PharmetricsPlus (P+), and IQVIA Analytics in the U.S. and the company’s internal estimates and assumptions outside the U.S. *
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53 U.S. Bioinformatics and claims data analysis estimates A-T market of 5,000+ patients – a strong commercial opportunity ~6,000 4,600 CLAIM DATA ANALYSIS Diagnosed and linked to HCP2 BIOINFORMATICS ANALYSIS Estimated US Prevalence1 Sources: 1. Bioinformatic Data 2. IQVIA Claims Data Analysis 3. Rothblum-Oviatt, et al Lederman, 2016 Under diagnosis and misdiagnosis Benefit from engaged, connected A-T patient community Standard of care creates opportunity for increased awareness and diagnosis EU4 + U.K. market size assumed to be similar given no population bias in A-T1,3 No currently approved A-T treatments, potential to expand patient number with market development
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54 Neurological symptoms and decline prevail throughout entire A-T patient journey • Unsteady gait when sitting or walking • Developmental delays • Difficulty with fine motor skills • Increased difficulty in walking and balance • Ocular telangiectasias • Speech difficulties • Frequent infections • Symptom management (physical and speech therapy) • Activities of daily living • Swallowing difficulties • Cancer monitoring • Respiratory issues • Wheelchair • Worsening ataxia and fine motor skills • Onset of involuntary movements (chorea) • Heightened risk of cancers Potentially addressed at launch LCM Opportunity Early Childhood (1-4 years) Preschool to School Age (4-10 years) Adulthood (18+) Adolescence (11-18)
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55 UNMET NEED OPPORTUNITY ADDRESSED BY eDSP A-T is underdiagnosed and time to diagnosis can be long Increase awareness and diagnosis to support early treatment No existing therapy to modify or slow progression of disease Effectively communicate value of eDSP in delaying disease progression Oral steroids cannot be used chronically due to severe side effects Leverage long-term safety data to show benefits of chronic use in patients with A-T Current treatment limited to physical therapy with the goal of supporting functions of daily living Communicate clinical evidence on delayed time to non-ambulation ✓ eDSP well-positioned to become standard of care in A-T ✓ ✓ ✓
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56 EasyKit eDSP EasyKit innovation Completed project to simplify end user experience Minimized packaging footprint for single-use treatment kit and ancillary materials Implementing robust Quince brand design Final prototype ready and commercial CDMO being selected Filed patent application Essential Pack Blood Draw Pack Drug Prep Pack Solvent Pack
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57 Ataxia-Telangiectasia product development landscape ASSET / COMPANY STAGE DRUG / MOA ROA AND DOSING COMMENTS eDSP Quince Tx Phase 3 (under SPA) Dexamethasone / Glucocorticoid Receptor Antagonist Once monthly autologous cell infusion • Positive Phase 3 efficacy signals • Well tolerated and good safety profile Levacetylleucine IntraBio Phase 3 N-acetyl-L-leucine / Modified Amino Acid Oral sachet 3x daily • Unknown efficacy in A-T; failed Phase 2 A-T study across all endpoints • Phase 3 A-T study includes patients ≥4 years old • Recently approved for NPC • Well tolerated and good safety profile MBM-01 Matrix BioMed Phase 2 (Last CT.gov update 2021) EPAS1/HIF1A Inhibitor Oral • Antioxidant. Also using for cancer treatment • 20 patient open-label study at UT completed 12/2022, no results posted Triheptanoin Univ of Queensland Phase 2 (open label IIT) Triheptanoin / Energy substrate replacement Oral ≥4x daily • Unknown efficacy in small Phase 2 IIT • Patents by Ultragenyx until 2029 (not funded) Nicotinamide Niagen Phase 2 (open label IIT) Nicotinamide / NF-κB modulatory Oral 3x daily • Positive efficacy signals in small Phase 2 IIT • Sold as nutraceutical Betamethasone Acasti Phase 1 complete (development stalled, attempted out-license) Betamethasone / Glucocorticoid Receptor Antagonist Oral spray for short-term use • No distinct advantages over common oral steroids. Higher Cmax than oral betamethasone, may lead to more AEs • Likely similar safety profile to standard steroids
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58 A-T pricing analogs Conducted qualitative payer research study with additional research planned Gross to net discount should be minimal given high rate of commercial pay Recent rare disease comparables: • Skyclarys (FA, 5K diagnosed patients in the U.S.) - $429K/year • Crenessity (CAH, 30K patients in the U.S.) - $460K/year • Daybue (Rett syndrome, 6-9K patients in the U.S.) - $585K/year • Vyjuvek (dystrophic epidermolysis bullosa, 3K diagnosed patients in the U.S.) - $631K/year • Duvyzat (DMD, 15K patients in the U.S.) - $700K/year • Mipplyfa (Neimann-Pick Type C, 300 diagnosed patients in the U.S.) - $1,020K/year
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59 Strategic partnership with nation’s leading infusion provider Option Care Health 59 Scalability to treat patients in additional targeted indications such as Duchenne muscular dystrophy Improved and standardized patient journey with higher control and consistency across eDSP administration sites Contracting with a single provider versus multiple individual academic centers of excellence Greater geographic flexibility to match patient locations and eDSP administration needs Leverage comprehensive suite of enhanced capabilities to handle key commercial services National footprint with more than 90 full-service pharmacies and 180-plus outpatient infusion suites located across the U.S.
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60 Substantial market exclusivity from generic competition Regulatory market exclusivities are available for both U.S. and European Union • Orphan drug designation provides 7 years protection in the U.S. and 10 years protection in Europe A broad portfolio of approved and filed patents covering the diagnosis, machine, process and methods of eDSP technologies • Existing patents will protect from generic competition at least up to 2036, (without PTE and PED extensions) • Secondary patent strategy to improve product characteristics, which can extend patent protection Technology barriers expected to create exclusivity beyond patent protection • eDSP integrates drug, device, process, and biologic (RBC) elements, creating high hurdles for therapeutic equivalence • Generics would require extensive in vivo and process validation far exceeding standard PK studies • Proprietary components and processes render replication clinically and commercially impractical Legal Technology Barriers T echnology eDSP
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Driving value creation Brendan Hannah, M.B.A. Chief Operating Officer & Chief Business Officer
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62 Quince well-positioned for driving value creation Strong competitive positioning Significant pipeline expansion opportunity Well-capitalized with sufficient cash runway into second quarter 2026 Preparing for strategic and financing activities post topline results Ability to expand quickly into late-stage clinical development in several high-value indications
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63 Recently approved first-to-market rare disease drug comparables: ~$400M annual run within 18 months of launch 63Source: Company internal competitive analysis $- $100 $200 $300 $400 $500 $600 $700 $800 Q1 Q2 Q3 Q4 Q5 Q6 Q7 Q8 Q9 Skyclarys Daybue Vyjuvek Daybue Rett syndrome 6-9K patients (estimated) $585K $1.0B Skyclarys Friedreich's ataxia 5K patients (diagnosed) $429K $1.1B Vyjuvek Dystrophic epidermolysis bullosa 3K patients (diagnosed) $631K $1.3B Product/ indication U.S. population size Pricing Annually Peak sales forecast Cumulative Sales of Rare Disease Products Since Launch
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64 Reata acquired by Biogen for $7.3B for recently approved FA therapy (Skyclarys) Case study: Reata/Friedreich Ataxia CHARACTERISTIC ATAXIA-TELANGIECTASIA FRIEDREICH ATAXIA US Prevalence (dx) 4,600 5,000 Global Sales No approved treatments $1.1B forecasted by peak Life Expectancy 25-30 years old 40 years old Symptoms & Severity Ataxia, gait ataxia, slurred speech, difficulty swallowing, infections, diabetes, cancer Non-ambulatory by teens Ataxia, gait ataxia, loss of reflexes, dysarthria, spasticity, scoliosis, hearing and vision loss Non-ambulatory 20-30 Pipeline Minimal competition (P1-P3 with unknown MOAs) Moderate+ competition (P1-NDA) Drug / LOE RBC encapsulated dexamethasone LOE – 2036 in US Difficult generic entry (potential perpetuity) Small molecule Nrf2 modulator LOE – 2033 Small molecule - quick generic entry Population Potentially 6+ at launch (PIP: 1-5) 16+ currently (PIP ongoing: 2-15) P3 Data -22% compared to placebo with RmICARS in 6-9 at 6 months Well tolerated -6% compared to placebo mFARS at 48 weeks 37% Liver tox, 33% nausea $- $300 $600 $900 $1,200 2023 2025 2027 2029 2031 2033 2035 Skyclarys Sales ($M) Source: Evaluate Pharma
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65 Assessment of additional indications for eDSP Beyond DMD, there are 10+ viable potential indications – how do we narrow it down and select what’s best? 1. Severe rare disease that allows A-T pricing 2. Chronic dosing required as opposed to acute/hospital dosing which would also create reimbursement issues 3. Pediatric indications ideal, but focus on orphan indications that do not trigger IRA negotiations 4. Focus on those with ≥5,000 patients (common cutoff for partners/investors) 5. Only proceed with those in which KOLs believe eDSP’s MOA/profile are viable Focus on:
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66 90 diseases Internal review of therapeutic categories to determine epidemiology, competitive landscape, and commercial opportunity 50 high potential TAs Secondary research to explore steroid utilization and level of clinical impact 20 TAs short listed Clinician/KOL interviews to elucidate clinical and commercial opportunities 12 targets ranked Clinical consultation to identify specific categories for clinical use and potential trial design Identifying best follow-on indications for eDSP
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67 Target eDSP to high potential immunology and autoimmune rare disease indications Potential to advance directly into Phase 2 studies for broad range of rare diseases Significant pipeline expansion opportunity Neurology/Neuromuscular Ataxia telangiectasia (1st indication) Duchenne muscular dystrophy (2nd indication) Becker muscular dystrophy Myasthenia gravis Limb-girdle muscular dystrophy Pediatric lupus Pulmonary sarcoidosis Juvenile idiopathic arthritis Rheumatology Inflammation & Immunology Autoimmune hepatitis Hashimoto's encephalopathy Pemphigus vulgaris Dermatomyositis Chronic inflammatory demyelinating polyradiculoneuropathy
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68 Selected DMD as second development program for eDSP eDSP For indications beyond A-T where chronic corticosteroid treatment is – or has the potential to become – standard of care Duchenne muscular dystrophy (DMD) ideal second indication for eDSP given well-described clinical benefits of corticosteroids in patients with DMD Finalized Phase 2 clinical trial study designs to evaluate eDSP for the potential treatment of patients with DMD Plan to prioritize capital efficient study approaches, including potential investigator-initiated trials (IITs) Plan to support dosing first patient in Phase 2 clinical study in 2026
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69 Favorable eDSP safety profile versus conventional corticosteroid administration Well-known toxicities of conventional corticosteroid administration Prednisone/ Deflazacort Agamree (Vamorolone) eDSP clinical safety profile Hirsutism Yes Yes No Delayed puberty Yes Yes No Hyperglycemia Yes Yes No Excessive weight gain Yes Yes No Acne Yes Yes No Growth suppression Yes No No Osteoporosis Yes Yes, but less than prednisone No References: Rossi L, et al. Biotechnol Appl Biochem. 2001;33(2):85-89. Koenig MK, et al. Front. Neurol., 22 January 2025 Sec. Pediatric Neurology. Volume 15 – 2024.
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70 Strong cash position to reach meaningful milestones with approximately $34.7 million in cash, cash equivalents, and short-term investments as of June 30, 2025 Evaluate potential strategic partnerships to out-license of ex-U.S. rights to provide additional operating runway Recent financing funds lean operating model: • eDSP through Phase 3 NEAT topline results and preparing for NDA and MAA submissions, assuming positive study results • ~$20 million NEAT study and ~$15 million OLE direct trial costs • Investigation of other potential indications for eDSP, including DMD Well-capitalized with sufficient cash runway into second quarter 2026 70
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Concluding remarks and Q&A Dirk Thye, M.D. Chief Executive Officer & Chief Medical Officer
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72 Key clinical and corporate milestones/goals 2025 2026 Prepare for potential NDA & MAA submission, assuming positive Phase 3 NEAT study results Prepare for the U.S. commercial launch of eDSP for A-T Readout Phase 3 NEAT topline results in first quarter of 2026 Secured opportunistic financing to extend cash runway into at least second quarter of 2026 Extended patent claims to 2036 in the U.S. with newly issued USPTO Notice of Allowance Completed Phase 3 NEAT study enrollment in July 2025 ATTeST long-term safety data published in Frontiers in Neurology in January 2025 Dose first patient in DMD Phase 2 clinical study Ongoing Phase 3 NEAT open label extension study Initiated EU pediatric investigational plan Ongoing EU pediatric investigational plan Selected Option Care Health as commercial partner
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Question & Answer Session Please type any questions you might have in the chat box on the screen
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Unlocking the power of a patient’s own biology for the treatment of rare disease Investor Day October 2, 2025