Editas Medicine, Inc. · NASDAQ:EDIT
Editas Medicine — Technical Presentation (June 2023)
26 slides from Editas Medicine's technical presentation, Jun 12, 2023
Slide 1: EDIT-301 Program Update: RUBY and EdiTHAL Trial Data Update
Dima, Tristan, & Stephanie LIVING WITH SICKLE CELL DISEASE EDIT-301 Program Update RUBY and EdiTHAL Trial Data Update June 12, 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 │ Year-one Check-in │ Key Takeaways of EDIT-301 Program Gilmore O’Neill, MB, MMSc President and CEO, Editas Medicine │ Clinical Updates of EDIT-301: RUBY and EdiTHAL Trials │ Closing Remarks Baisong Mei, MD, PhD CMO, Editas Medicine │ Q&A 3
Slide 4: Year-one Check-in
Year-one Check-in Achieved two proof-of-concepts for in vivo and ex vivo editing platforms in 2022. Sharpened our focus on: Execution of EDIT-301 development toward regulatory approval. Discovery of in vivo edited therapeutics (including in vivo HSC). Decreased Cash Burn extending operational runway
Slide 5: Desired Attributes of EDIT-301
Desired Attributes of EDIT-301 • 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 OR CD34+
Slide 6: Key Takeaways
Key Takeaways* EDIT-301 drives early, robust EDIT-301 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% beginning as early as in as early as 4 months 4 months Initial clinical results are EDIT‐3
Slide 7: RUBY Trial Participant: Danielle Lee
RUBY Trial Participant: Danielle Lee editasmedicine.com © 2023 Editas Medicine 7
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: CRISPR Enzyme and Target Choices Matter in Building a Medicine
CRISPR Enzyme AND Target Choices Matter in Building a Medicine to Give Best Outcomes to Patients ENZYME 3’ 5’ 5’ 3’ AsCas12a is a differentiated AsCas12a Cas9 CRISPR nuclease with higher Specificity Higher Lower specificity and efficiency Editing Efficiency Higher Lower compared with Cas92,3 Images
Slide 11: RUBY Study of EDIT-301 in Patients with Severe SCD
RUBY Study of EDIT-301 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 and are VOE-free
All Treated RUBY Patients Successfully Engrafted, Showed a Favorable Safety Profile, and are VOE-free Since Infusion PATIENT 1 PATIENT 2 PATIENT 3 PATIENT 4 EDIT-301 Total CD34+ (106/kg) 10.0 4.0 4.1 3.7 Neutrophil Engraftment (day)* 23 29 23 24 Platelet Engraftment (day)† 19 37 23 28 Follow-Up Dura
Slide 13: RUBY Patient Maintained HbF >40% and is VOE-free
RUBY Patient 1 Maintained HbF >40% and is VOE-free Since Infusion; Total Hemoglobin Returned to the Normal Physiological Range by Month 5 PATIENT 1 (Male) 18 16.6 EDIT-301 16.0 16.3 16.4 16.4 16.4 Normal infusion 15.0 15.7 range in 16 males* Total Hemoglobin (g/dL) 14 12.9 11.9 12 10 8 6 45.4% 44.3%
Slide 14: RUBY Patients Follow Same Hemoglobin Trajectory as Patient 1
RUBY Patients Follow Same Total Hemoglobin and Fetal Hemoglobin Trajectory as Patient 1 PATIENT 1 (Male) PATIENT 2 (Female) 18 16 16.4 16.4 16.6 16.4 Normal EDIT-301 16.0 16.3 15.7 EDIT-301 Normal 16 infusion 15.0 range in 14 range in males* infusion 12.7 12.5 11.9 12.1 12.4 females* Total Hemoglobi
Slide 15: First EdiTHAL Patient Successfully Engrafted
First EdiTHAL Patient Successfully Engrafted, Experienced Similar Engraftment and Similar Safety Profile to RUBY Patients PATIENT 1 EDIT-301 Total CD34+ (106/kg) 6.1 Neutrophil Engraftment (day)* 23 Platelet Engraftment (day)† 26 Follow-Up Duration (months) 1.5 • The first EdiThal patient experience
Slide 16: First Treated EdiTHAL Patient Followed Similar Trajectory
First Treated EdiTHAL Patient Followed Similar Trajectory Expressing >30% HbF (>4 g/dL) at 1.5 Months Post-infusion PATIENT 1 (Male) 18 normal range 16 EDIT-301 in males* infusion 14 13.0 1.6% 12.6 Total Hemoglobin (g/dL) 1.8% 2.7% 0.0% 12 10.1 10 2.6% 2.3% 8 93.5% 81.2% 65.0% 6 4 2 34.9% 2.2% 13.9%
Slide 17: Consistent Levels of ex vivo Editing Correlated with Clinical Responses
Consistent Levels of ex vivo Editing Correlated with Similar Clinical Responses in Dosed Patients Similar editing levels achieved in cells from all patients, predicting similar and robust clinical responses; no detectable off target editing Editing Data from Representative EDIT-301 Clinical Producti
Slide 18: RUBY and EdiTHAL Study Site Activation and Patient Enrollment On-track
RUBY and EdiTHAL Study Site Activation and Patient Enrollment Remains On-track 22 RUBY sites activated 20 RUBY patients enrolled 7 EdiTHAL sites activated 5 EdiTHAL patients enrolled 18
Slide 19: Key Takeaways
Key Takeaways* EDIT-301 drives early, robust EDIT-301 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% beginning as early as in as early as 4 months 4 months Initial clinical results are EDIT‐3
Slide 20: Editas' Gene Edited HSC Manufacturing Capabilities
Editas’ Gene Edited HSC Manufacturing Capabilities ▪ In-house manufacturing capabilities allow Editas to expedite clinical development ▪ Editas fully controls its manufacturing process with capabilities to control and flex number of patients edited per month Waltham, MA Cambridge, MA cGMP Cell Manuf
Slide 21: Majority of Patients Still Awaiting Therapy at EDIT-301 Launch
Majority of Patients will Still be Awaiting Therapy at Anticipated Time of EDIT-301 Launch Prevalence of SCD in U.S. Prevalence of Beta-Thal in U.S. 100K 1K Transplant center and cost Payors are concerned about long- Patients will be hesitant, at first, to logistics may further limit initial term du
Slide 22: EDIT-301 Uses AsCas12a
EDIT-301 Uses AsCas12a • EDIT-301 uses AsCas12a • We have Exclusive Licenses to AsCas12a • We do not require Cas9 cross-licenses to commercialize EDIT-301 AsCas12a, CRISPR-associated protein 12a; Cas9, CRISPR-associated protein 9 22
Slide 23: Editas Controls Valuable Foundational Cas9 IP
Editas Controls Valuable Foundational Cas9 IP from Broad Institute and Harvard University Only a subset is subject to interference Harvard University Cas9 Patent Status Broad Institute Cas9 Cas9 patents Patent Interference (N=13) (Church, N=25) # Patent No. # Patent No. 1 11,535,863 1 9,840,713 2 11
Slide 24: Closing Remarks
Closing Remarks Gilmore O’Neill, MB, MMSc President and CEO Editas Medicine 24
Slide 25: Acknowledgements
Acknowledgements Thank you to participating patients, their families, clinical investigators, and study site teams for support. 25
Slide 26: Questions & Discussion
Questions & Discussion Gilmore O’Neill, MB, MMSc Baisong Mei, MD, PhD Erick Lucera, MBA, MS President and CEO CMO CFO 26