Editas Medicine, Inc. (EDIT) Q3 2023 Earnings Call Nov. 03, 2023
Here is a summary of the earnings call.
Gilmore O'Neill - CEO
EDIT's Strategic Focus:
Editas Medicine aims to deliver life-changing medicines for previously untreatable or undertreated diseases through gene editing.
Evolution of EDIT's Strategy:
In January, EDIT announced its strategy to position itself as a leader in programmable gene editing.
The strategy consists of three pillars:
Accelerate clinical development of EDIT-301 for severe sickle cell disease and beta-thalassemia.
Sharpen focus on in vivo editing therapies.
Expand business development activities to partner with complementary technologies.
2023 Objectives:
Clinical data updates for EDIT-301's RUBY trial for sickle cell disease and EdiTHAL trial for beta-thalassemia by the end of 2023.
Dose 20 patients in the RUBY trial by January 2024.
Hire a new Chief Scientific Officer for in vivo medicine development.
Leverage IP portfolio and business development for value creation.
Progress in Q3:
Enrolled 27 sickle cell and 8 beta-thalassemia patients in the RUBY and EdiTHAL studies.
Clinical data from 11 sickle cell patients and 6 beta-thalassemia patients to be shared in a webinar and ASH presentation in December.
RMAT designation granted by FDA for EDIT-301's severe sickle cell disease treatment.
Key hire: Caren Deardorf as Chief Commercial and Strategy Officer.
Expansion of clean room capacity for scaling EDIT-301 manufacturing.
In Vivo and Pipeline Development:
Discovery work on in vivo therapeutic targets in haematopoietic stem cells and other tissues initiated.
Linda Berkley hired as Chief Scientific Officer for leading these efforts.
Target selection criteria to differentiate from current standard of care.
Business Development:
Agreement with Vor Bio for a non-exclusive license for ex vivo Cas9 gene-edited HSC therapies for haematological malignancies.
Editas holds a substantial portfolio of foundational patents related to Cas9 use in developing human medicines.
Licensing deals, such as with Vor Bio, reinforce the value of EDIT's IP portfolio.
Conclusion:
Promising efficacy and safety data for EDIT-301 in June suggests it could be a one-time, durable medicine for patients with sickle cell disease and beta-thalassemia.
Focus on delivering differentiated editing medicines to patients with serious genetic diseases.
Strong strategic focus, dedicated scientists, and execution drive are building momentum toward EDIT's goals.
Baisong Mei - CMO
EDIT-301 Progress:
EDIT-301, a treatment for severe sickle cell disease and transfusion-dependent beta-thalassemia, is in development.
As of now, 27 patients have been enrolled in the RUBY trial, with dosing for the 20th patient expected in January 2024.
Eight patients have been enrolled in the EdiTHAL trial for beta-thalassemia.
Positive changes have been observed in patients dosed with EDIT-301.
Collaboration with the FDA planned for the second half of the year.
Exa-cel AdCom and Gene Editing Potential:
The recent exa-cel AdCom reaffirmed the potential of gene editing technology.
Gene editing has the potential to transform the treatment of serious diseases, improving patients' lives.
Anticipation for the approval of the first gene editing medicine, with Editas leading the way.
Upcoming Data Presentation:
Clinical data from both RUBY and EdiTHAL trials to be presented at ASH in December.
RUBY trial data to include efficacy and safety metrics for 11 patients.
EdiTHAL trial data to include efficacy and safety metrics for six patients.
Previous data presentations showed promising results, including early correction of anaemia and increased fetal haemoglobin.
CRISPR Technology and Differentiation:
EDIT-301 uses the proprietary AsCas12a enzyme for editing the HBG12 promoter.
AsCas12a is more efficient and has lower off-target editing compared to other CRISPR enzymes.
Editing the HBG12 promoter results in increased red blood cell production and improved red blood cell health.
The sustained normal total haemoglobin level is a potential point of differentiation for EDIT-301.
Correction of anaemia can significantly improve patients' quality of life and reduce end-organ damage.
Conclusion:
EDIT-301 continues to show promise in clinical trials, with positive changes observed in patients.
The presentation of additional clinical data next month at ASH will provide further insights into the potential of EDIT-301 and the power of CRISPR technology.
Erick Lucera - CFO
Financial Highlights:
Cash, cash equivalents, and marketable securities as of September 30: $446 million.
Expected funding to cover operating expenses and capital expenditures into the third quarter of 2025.
Third-quarter 2023 revenue: $5.3 million, primarily from the non-exclusive Cas9 license deal with Vor Bio.
R&D expenses in Q3 2023: $41 million, reflecting a focus on the EDIT-301 program.
G&A expenses in Q3 2023: $15 million, decreased from the same period in 2022.
Strong financial position supported by a sharpened discovery focus, capital raise, and recent out-licensing deal.
Cash runway into Q3 2025 provides ample resources for ongoing trials, manufacturing, and research.
Value of Investor Knowledge:
The CFO, a former buy-side investor, acknowledges the importance of both buy-side and sell-side knowledge.
Open to hearing from shareholders as Editas advances in the gene editing field.
Q&A followed.
Anne Chapman
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