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Editas (EDIT) Reports Upbeat New Data From Reni-Cel Studies
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Editas Medicine (EDIT - Free Report) reported new positive data from the phase I/II/III RUBY study of its lead candidate, reni-cel, in 18 patients with sickle cell disease (SCD) and the phase I/II EdiTHAL study of the same in seven patients with transfusion-dependent beta thalassemia (TDT) in separate press releases.
The company is focused on developing reni-cel as a potential one-time, durable gene editing medicine to treat these indications.
This data readout follows the December 2023 press release, when the company last reported positive safety and efficacy data in 17 patients treated with reni-cel in the RUBY and EdiTHAL studies (11 and six patients, respectively).
Per the latest data readout from the RUBY study, the 18 severe SCD patients have remained free of vaso-occlusive events for up to 22.8 months of follow-up, post-treatment with reni-cel. Notably, patients experienced early normalization of total hemoglobin(Hb) levels, averaging more than 14 g/dL, and significant and sustained increases in fetal hemoglobin (HbF) levels, exceeding 40%.
Moreover, efficacy results showed that SCD patients in the RUBY study experienced an early and sustained increase in the mean percentage of HbF-containing red cells (F-cells), remaining above 90% from month 4 onward for the 12 patients with at least four months of follow-up. Additionally, the mean corpuscular fetal hemoglobin of F-cells remained above the anti-sickling threshold of 10 pg/F-cell by month 3 post-reni-cel infusion for the 14 patients with at least three months of follow-up.
Markers of hemolysis showed normalization or improvement in patients treated with reni-cel, indicating effective management of the underlying disease pathology.
Editas stated that it has now dosed more than 20 SCD patients, completed adult cohort enrollment, and opened and enrolled patients in the adolescent cohort in the Ruby study, implicating significant progress.
Year to date, shares of EDIT have plunged 50% compared with the industry’s 6.3% decline.
Image Source: Zacks Investment Research
In the EdiTHAL study, it was observed that all TDT patients experienced early and robust increases in Hb and HbF levels, with total Hb rising above the transfusion independence threshold of 9.0 g/dL. Since the treatment with reni-cel, all seven patients remaintransfusion-free at last follow-up for a range of 4.1 to 12.8 months.
All patients in the RUBY, as well as the EdiTHAL study, demonstrated consistently high levels of genetic editing in the HBG1 and HBG2 promoter regions.
Safety data from both the studies showed that reni-cel was overall well-tolerated with a safety profileconsistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant by all evaluated RUBY and EdiTHAL study patients. Moreover, there were no reports of any serious treatment-related adverse events in either of the studies.
All patients in both studies demonstrated successful neutrophil and platelet engraftment after treatment with reni-cel.
Such safety and efficacy data from the 18 SCD patients in the RUBY study and seven TDT patients from the EdiTHAL study have been presented at a recent medical conference. The company plans to share further updates from both studies again toward the end of the year.
It is important to note that Vertex (VRTX - Free Report) and CRISPR Therapeutics’ (CRSP - Free Report) already market their one-time gene therapy for SCD and TDT, Casgevy(exa-cel). Casgevy was approved in multiple geographies in 2023 and early 2024, including the United States and the EU.
Vertex and CRISPR Therapeutics’ Casgevy is the first-ever CRISPR/Cas9-based therapy to be approved anywhere in the world. VRTX and CRSP believe Casgevy has the potential to be a one-time functional cure for SCD and TDT patients, with an estimated patient population of approximately 35,000 across the United States and the EU.
Vertex and CRISPR Therapeutics are also currently conducting two new phase III studies in pediatric patients with TDT and SCD.
In the past 30 days, the Zacks Consensus Estimate for ALX Oncology’s 2024 loss per share has remained constant at $2.89. During the same period, the consensus estimate for 2025 loss per share has remained constant at $2.73. Year to date, shares of ALXO have plunged 45.9%.
ALX Oncology beat estimates in two of the trailing four quarters and missed twice, delivering an average negative surprise of 8.83%.
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Editas (EDIT) Reports Upbeat New Data From Reni-Cel Studies
Editas Medicine (EDIT - Free Report) reported new positive data from the phase I/II/III RUBY study of its lead candidate, reni-cel, in 18 patients with sickle cell disease (SCD) and the phase I/II EdiTHAL study of the same in seven patients with transfusion-dependent beta thalassemia (TDT) in separate press releases.
The company is focused on developing reni-cel as a potential one-time, durable gene editing medicine to treat these indications.
This data readout follows the December 2023 press release, when the company last reported positive safety and efficacy data in 17 patients treated with reni-cel in the RUBY and EdiTHAL studies (11 and six patients, respectively).
Per the latest data readout from the RUBY study, the 18 severe SCD patients have remained free of vaso-occlusive events for up to 22.8 months of follow-up, post-treatment with reni-cel. Notably, patients experienced early normalization of total hemoglobin(Hb) levels, averaging more than 14 g/dL, and significant and sustained increases in fetal hemoglobin (HbF) levels, exceeding 40%.
Moreover, efficacy results showed that SCD patients in the RUBY study experienced an early and sustained increase in the mean percentage of HbF-containing red cells (F-cells), remaining above 90% from month 4 onward for the 12 patients with at least four months of follow-up. Additionally, the mean corpuscular fetal hemoglobin of F-cells remained above the anti-sickling threshold of 10 pg/F-cell by month 3 post-reni-cel infusion for the 14 patients with at least three months of follow-up.
Markers of hemolysis showed normalization or improvement in patients treated with reni-cel, indicating effective management of the underlying disease pathology.
Editas stated that it has now dosed more than 20 SCD patients, completed adult cohort enrollment, and opened and enrolled patients in the adolescent cohort in the Ruby study, implicating significant progress.
Year to date, shares of EDIT have plunged 50% compared with the industry’s 6.3% decline.
Image Source: Zacks Investment Research
In the EdiTHAL study, it was observed that all TDT patients experienced early and robust increases in Hb and HbF levels, with total Hb rising above the transfusion independence threshold of 9.0 g/dL. Since the treatment with reni-cel, all seven patients remaintransfusion-free at last follow-up for a range of 4.1 to 12.8 months.
All patients in the RUBY, as well as the EdiTHAL study, demonstrated consistently high levels of genetic editing in the HBG1 and HBG2 promoter regions.
Safety data from both the studies showed that reni-cel was overall well-tolerated with a safety profileconsistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant by all evaluated RUBY and EdiTHAL study patients. Moreover, there were no reports of any serious treatment-related adverse events in either of the studies.
All patients in both studies demonstrated successful neutrophil and platelet engraftment after treatment with reni-cel.
Such safety and efficacy data from the 18 SCD patients in the RUBY study and seven TDT patients from the EdiTHAL study have been presented at a recent medical conference. The company plans to share further updates from both studies again toward the end of the year.
It is important to note that Vertex (VRTX - Free Report) and CRISPR Therapeutics’ (CRSP - Free Report) already market their one-time gene therapy for SCD and TDT, Casgevy(exa-cel). Casgevy was approved in multiple geographies in 2023 and early 2024, including the United States and the EU.
Vertex and CRISPR Therapeutics’ Casgevy is the first-ever CRISPR/Cas9-based therapy to be approved anywhere in the world. VRTX and CRSP believe Casgevy has the potential to be a one-time functional cure for SCD and TDT patients, with an estimated patient population of approximately 35,000 across the United States and the EU.
Vertex and CRISPR Therapeutics are also currently conducting two new phase III studies in pediatric patients with TDT and SCD.
Editas Medicine, Inc. Price and Consensus
Editas Medicine, Inc. price-consensus-chart | Editas Medicine, Inc. Quote
Zacks Rank and Stock to Consider
Editas currently carries a Zacks Rank #3 (Hold).
A better-ranked stock from the drug/biotech industry is ALX Oncology Holdings (ALXO - Free Report) , carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
In the past 30 days, the Zacks Consensus Estimate for ALX Oncology’s 2024 loss per share has remained constant at $2.89. During the same period, the consensus estimate for 2025 loss per share has remained constant at $2.73. Year to date, shares of ALXO have plunged 45.9%.
ALX Oncology beat estimates in two of the trailing four quarters and missed twice, delivering an average negative surprise of 8.83%.