Abeona Therapeutics Inc. · NASDAQ:ABEO
Abeona Therapeutics — Technical Presentation (November 2026)
29 slides from Abeona Therapeutics's technical presentation, November 2026
Slide 1: APT-1011 Clinical Development History
APT-1011 Clinical Development History November 2026
Slide 2: Forward-Looking Statements
Forward-Looking Statements These materials contain certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. We have attempted
Slide 3: APT-1011 Clinical History 2011 – 2024
APT-1011 Clinical History 2011 – 2024 Core trials shown from first participant enrolled to last participant completed 2011 2013 2015 2017 2019 2021 2023 2024 Phase 1/2a PR-021 STUDY DESIGN / POPULATION KEY RESULTS / DEVELOPMENT IMPLICATION 2011 – 2012 Multicenter, randomized, double-blind, placebo-c
Slide 4: FLUTE
LAL Hi Abeona THERAPEUTICS
Slide 5: FLUTE | Study Design
FLUTE | Study Design Randomized, double-blind, placebo-controlled, multicenter dose-ranging design 1.5 mg HS 1.5 mg BID 3 mg HS 3 mg BID Placebo 1.5 mg/day 3 mg/day 3 mg/day 6 mg/day control Key eligibility Baseline disease characteristics Adults 18–75 years with confirmed EoE, ≥15 eos/HPF, prior PP
Slide 6: FLUTE | Prespecified Endpoint Framework
FLUTE | Prespecified Endpoint Framework Primary proof-of-concept was histologic; secondary measures spanned durability, endoscopy, symptoms and clinical events Primary endpoint Maintenance objective Histologic response at Week 12, defined as ≤6 eosinophils per Sustained histologic response through W
Slide 7: FLUTE | Safety and Regimen Selection
FLUTE | Safety and Regimen Selection Safety findings Dose-selection conclusion • Generally well tolerated • All active doses showed activity • No treatment-related serious adverse events reported • 6 mg/day did not add meaningful activity • No deaths reported • 3 mg/day was identified as the minimum
Slide 8: FLUTE | What the Study Established
FLUTE | What the Study Established Proof-of-concept, regimen selection, and long-term feasibility supported progression to Phase 3 1 Biologic activity Strong Week 12 histologic response across active doses 2 Endoscopic activity Significant improvement across EREFS responder definitions 3 Durability
Slide 9: FLUTE-2
LAL Hi Abeona THERAPEUTICS
Slide 10: FLUTE-2 | 12-Week Primary Endpoint
FLUTE-2 | 12-Week Primary Endpoint The pivotal induction comparison tested 3 mg HS against placebo, followed by longer-term phases Co-primary 1 | Histology Co-primary 2 | Symptoms Histologic response at Week 12, defined as ≤6 eosinophils per Symptomatic endpoint for FLUTE-2 was change in dysphagia h
Slide 11: FLUTE-2 | Adults Enrolled with Active, Symptomatic EoE
FLUTE-2 | Adults Enrolled with Active, Symptomatic EoE Baseline disease severity was defined by concurrent histologic inflammation and frequent dysphagia EoE had to be confirmed during screening by central pathology. At least 6 biopsies were Histologically active disease collected, with ≥3 from both
Slide 12: FLUTE-2 | Histologic Endpoint Met, Symptom Co-Primary Not Met
FLUTE-2 | Histologic Endpoint Met, Symptom Co-Primary Not Met Week 12 induction: APT-1011 3 mg HS (n=97) vs placebo (n=46) Phase 3 | n=143 CO-PRIMARY 1 | HISTOLOGIC RESPONSE CO-PRIMARY 2 | DYSPHAGIA EPISODES ≤6 peak eos/HPF at Week 12 Change from baseline at Week 12 APT-1011 66.0% APT-1011 15.3 → 6.
Slide 13: FLUTE-2 | Outcomes
FLUTE-2 | Outcomes Endoscopic effects were significant; symptom improvement favored active treatment but was less robust Endoscopic endpoints (post-hoc) EREFS responder 50.5% Overview of outcomes (p<0.001) 15.2% EREFS Inflammatory 37.1% FLUTE-2 demonstrated statistically significant responder (p<0.0
Slide 14: FLUTE-3
LAL Hi Abeona THERAPEUTICS
Slide 15: FLUTE-3 | 24-Week Phase 3 Test of Histologic and Complete Symptom Response
FLUTE-3 | 24-Week Phase 3 Test of Histologic and Complete Symptom Response SP-1011-005 | APT-1011 3 mg at bedtime in adults with active eosinophilic esophagitis n=218 CO-PRIMARY ENDPOINTS AT WEEK 24 Histologic response Complete symptomatic response 1 ≤6 peak eosinophils/HPF on esophageal mucosal bio
Slide 16: FLUTE-3 | Enrolled Adults with Histologic and Symptomatic EoE Activity
FLUTE-3 | Enrolled Adults with Histologic and Symptomatic EoE Activity HISTOLOGIC BURDEN CLINICAL BURDEN ANATOMIC BURDEN Active eosinophilic inflammation Recurrent dysphagia before Inflammatory and fibrostenotic randomization features assessed EoE confirmation required ≥15 peak eos/HPF. Patients ha
Slide 17: FLUTE-3 | Co-Primary Results (24 Weeks)
FLUTE-3 | Co-Primary Results (24 Weeks) Histologic response was statistically significant; complete symptomatic response was not different from placebo at week 24 Histology | MET 52.8% vs 4.5% 52.8% OR 22.63; p<0.001 Histologic response 4.5% 13.9% Symptoms | NOT MET Complete symptom 13.9% vs 12.7% r
Slide 18: FLUTE-3 | Other Outcomes
FLUTE-3 | Other Outcomes Multiple objective supportive measures favored APT-1011 Exploratory endoscopic endpoints Zero eosinophils OR 14.74; p<0.001 41.7% EREFS responder (p=0.009) 22.7% Clinicopathologic response Complete symptomatic response AND histological response at Week 24 27.8% OR 5.34; p=0.
Slide 19: FLUTE-3 | Summary of Safety and Study Conclusion
FLUTE-3 | Summary of Safety and Study Conclusion APT-1011 was observed to be well tolerated; efficacy conclusions were split by endpoint domain Safety Study conclusion • Safe and well tolerated through the 24-week induction period • Histologic response was statistically significant • No deaths; one
Slide 20: DSQ Mapping of APT-1011 Study Results
DSQ Mapping of APT-1011 Study Results 20
Slide 21: Rationale for Utilizing DSQ in Next APT-1011 Study
Rationale for Utilizing DSQ in Next APT-1011 Study • The DSQ was used with both FDA approved EoE products (Dupixent, Eohilia ) • Eohilia was approved based on FDA analysis of DSQ Q2=0 or Q3=0, i.e., subjects achieving no dysphagia or experiencing only dysphagia that “got better or cleared up on its
Slide 22: DSQ Endpoint for Eohilia Approval
DSQ Endpoint for Eohilia Approval DSQ Questionnaire Used By Eohilia for Approval DSQ Questionnaire Question DSQ Response DSQ Score 1. Since you woke up this morning, did you eat solid Yes – go to Q2 Not scored food? (1) 2. Since you woke up this morning, has food gone down No 0 slowly or been stuck
Slide 23: Methodology for Mapping DSQ Endpoint to PROSE NRS for Difficulty Swallowing
Methodology for Mapping DSQ Endpoint to PROSE NRS for Difficulty Swallowing (1) • Eligibility criteria in Ellodi and Takeda studies were similar • The lower end of the NRS (scale 0 – 10) for difficulty swallowing (NRS ≤3, ≤2, ≤1) was utilized as a representation of the DSQ Q2=0 or Q3=0 • Various NRS
Slide 24: Mapping DSQ Endpoint to PROSE NRS for Difficulty Swallowing
Mapping DSQ Endpoint to PROSE NRS for Difficulty Swallowing PROSE Difficulty DSQ Questionnaire Swallowing Question DSQ Response DSQ Score NRS Response (0 – 10) 1. Since you woke up this morning, did you eat Yes – go to Q2 Not scored Not asked in PROSE solid food? 2. Since you woke up this morning, h
Slide 25: Illustrative FLUTE-3 PROSE-to-DSQ Mapping Highlights Potential Statistically Significant Symptom-Based Endpoint
Illustrative FLUTE-3 PROSE-to-DSQ Mapping Highlights Potential Statistically Significant Symptom-Based Endpoint Threshold Selection of the PROSE NRS for FLUTE-3 Responder Exploratory Analysis Results Difficulty Swallowing to the DSQ Endpoint Using Mapping of PROSE to DSQ Placebo Responder Response R
Slide 26: Robustness Analysis of Responder Rates at Various Thresholds
Robustness Analysis of Responder Rates at Various Thresholds – All Subjects (1) Sample Size: Placebo = 110, APT-1011 = 108 50% 48% Placebo APT-1011 45% 40% 36% 34% 35% Responder Rate 30% 25% 21% 20% 18% 15% 10% 10% 5% 0% <=1 <=2 <=3 P-value=0.077 P-value=0.027 P-value=0.053 NRS Threshold (2) Notes:
Slide 27: Conclusion
Conclusion • When applied to the FLUTE-3 dataset, we believe use of the DSQ endpoint would have been more likely to yield a successful trial outcome • Mapping exercises provide confidence for a future study utilizing the DSQ 27
Slide 28: Expanded Access Program (EAP)
Expanded Access Program (EAP) Enabling access to APT-1011 following trial conclusion 28
Slide 29: Expanded Access Program | Purpose and Operating Period
Expanded Access Program | Purpose and Operating Period Goal Program linkage Provide continued access to APT-1011 for patients completing Under later SP-1011-005 protocol amendments, eligible subjects clinical trials who would otherwise lose therapy access while could enter the EAP rather than the st
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