Editas Medicine, Inc. · NASDAQ:EDIT
Editas Medicine — Technical Presentation (December 2023)
20 slides from Editas Medicine's technical presentation, Dec 11, 2023
Slide 1: Reni-cel Program Update: RUBY and EdiTHAL Trial Data Update
Dima, Tristan, & Stephanie LIVING WITH SICKLE CELL DISEASE Renizgamglogene autogedtemcel (reni-cel) Program Update RUBY and EdiTHAL Trial Data Update December 11, 2023
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995, including statements regarding the potential market for EDIT-301, if approved. The words "anticipate," "believe," "continue," "
Slide 3: Agenda and Speakers
Agenda and Speakers SPEAKERS │ Welcome and 18-Month Check-in │ Key Takeaways of reni-cel (EDIT-301) Program Gilmore O’Neill, MB, MMSc President and CEO, Editas Medicine │ Clinical Updates of reni-cel (EDIT-301): RUBY and EdiTHAL Trials │ Closing Remarks Baisong Mei, MD, PhD CMO, Editas Medicine │ Q&
Slide 4: 18-Month Check-in
18-Month Check-in Groundbreaking Science, New Management Team, Commercial Focus A world leading gene editing platform supported by foundational IP estate. Shareholder value driven team with a proven track record and strong domain expertise. Lead asset reni-cel (EDIT-301), a potentially differentiate
Slide 5: Desired Attributes of Reni-cel
Desired Attributes of Reni-cel • Clinical Outcomes: • Rapid correction of anemia to Normal Physiological Hemoglobin levels. • Fetal Hemoglobin levels ≥ 40%, well above anti-sickling threshold. • Safety profile consistent with myeloablative busulfan conditioning and autologous hematopoietic stem cell
Slide 6: Key Takeaways
Key Takeaways Reni-cel drives early, robust Reni-cel drives robust No VOEs seen to date in all correction of anemia to normal sustained increases in HbF dosed SCD patients physiological range of total Hb >40% for SCD Reni-cel safety profile consistent Initial Hb and HbF responses with myeloablative
Slide 7: RUBY Participant: In His Own Words
RUBY Participant: In His Own Words Before the study, I was not able to complete many things that I should have been doing every day. I felt that I would be at the finish line of completing something, then I would have a pain crisis and then that episode would knock me down and sometimes take me even
Slide 8: SCD is an Inherited Life-Threatening Hematological Disorder
SCD is an Inherited Life-Threatening Hematological Disorder Manifesting Shortly After Birth Lifelong complications, multi-organ damage, and comorbidities impact patient SCD is a genetic blood disorder caused quality of life1,2 by mutations in the HBB gene that cause sickling of RBCs; this leads to I
Slide 9: Increased Fetal Hemoglobin Correlates with Reduced SCD Symptoms
Increased Fetal Hemoglobin Correlates with Reduced SCD Symptoms Minimal or no symptoms when HbF >30% when SCD coinherited with Hereditary Persistence of Fetal Hemoglobin Patient with Patient with SCD and Sickle Cell Disease Hereditary Persistence of (SCD)1 Fetal Hemoglobin (HPFH)2, 4 Sickle cell dis
Slide 10: Reni-cel's Rational Design: Target Choices AND CRISPR Enzyme Matter
Reni-cel’s Rational Design: Target Choices AND CRISPR Enzyme Matter in Building a Medicine to Give Best Outcomes to Patients TARGET HBG1 and HBG2 BCL11A RBC Production Normal Reduced HBG1 and HBG2 promoters are Proliferative capacity Normal Reduced a more appropriate genomic target versus BCL11A for
Slide 11: RUBY Study of Reni-cel in Patients with Severe SCD
RUBY Study of Reni-cel in Patients with Severe SCD Design Key Inclusion Criteria Key Endpoints • Phase 1/2 • ~40 patients 18–50 years • Proportion of patients achieving complete resolution • International, multicenter • Diagnosis of severe SCD of severe VOEs • 24 months follow-up post- • Safety and
Slide 12: All Treated RUBY Patients Successfully Engrafted, Showed a Favorable Safety Profile
All Treated RUBY Patients Successfully Engrafted, Showed a Favorable Safety Profile DEMOGRAPHICS (N=11) Genotype, n(%) βS/βS 11 (100) • Safety profile is consistent Sex, n (%) with myeloablative busulfan conditioning and Female 6 (54.5) autologous HSCT Age, years, mean (SD) 27.6 (4.2) Severe VOEs, p
Slide 13: All Treated RUBY Patients are VOE-free Since Reni-cel Infusion
All Treated RUBY Patients are VOE-free Since Reni-cel Infusion VOEs Prior to Informed Consent VOE-free After Reni-cel Infusion Patient 1 9 Patient 2 6 Patient 3 11 Patient 4 11 Severe VOE Patient 5 7 Non-severe VOE Patient 6 7 Patient 7 7 Patient 8 9 Patient 9 4 Patient 10 12 -25 -20 -15 -10 -5 0* 0
Slide 14: RUBY Patients Show Total Hb Rapidly Returning to the Normal Range
RUBY Patients Show Total Hb Rapidly Returning to the Normal Range and Clinically Meaningful Improvements in HbF Levels of >40% 18 16.9 16.2 13.6–18.0 g/dL normal 16 Reni-cel 15.6 range in males* 15.1 infusion 14.3 14.1 14.5 14.6 Mean Total Hemoglobin (g/dL) 13.8 14 13.0 12.0–16.0 g/dL normal 12.3 ra
Slide 15: EdiTHAL Patients Successfully Engrafted, Similar Safety Profile to RUBY
EdiTHAL Patients Successfully Engrafted, Experienced Similar Engraftment and Similar Safety Profile to RUBY Patients DEMOGRAPHICS (N=6) Genotype, n(%) β0/β0 2 (33.3) • Safety profile is Non-β0/β0* 4 (66.7) consistent with Sex, n (%) myeloablative busulfan Female 4 (66.7) conditioning and autologous
Slide 16: EdiTHAL Patients Had Early and Robust Increase of Total Hb
EdiTHAL Patients Had Early and Robust Increase of Total Hb Above the Transfusion Independence Threshold 18 Reni-cel 13.6–18.0 g/dL normal 16 range in males* infusion Mean Total Hemoglobin (g/dL) 14 13.1 12.0–16.0 g/dL normal 12.9 12.8 12.4 range in females* 12 11.4 11.0 10.8 10.7 10 9.5 Transfusion
Slide 17: Key Takeaways
Key Takeaways Reni-cel drives early, robust Reni-cel drives robust No VOEs seen to date in all correction of anemia to normal sustained increases in HbF dosed SCD patients physiological range of total Hb >40% for SCD Reni-cel safety profile consistent Initial Hb and HbF responses with myeloablative
Slide 18: Acknowledgements
Acknowledgements Thank you to participating patients, their families, clinical investigators, and study site teams for support. 18
Slide 19: Closing Remarks
Closing Remarks Gilmore O’Neill, MB, MMSc President and CEO Editas Medicine 19
Slide 20: Questions & Discussion
Questions & Discussion Gilmore O’Neill, MB, MMSc Baisong Mei, MD, PhD Erick Lucera, MBA, MS President and CEO CMO CFO 20
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