Shares of Editas Medicine EDIT gained 4.2% on Monday after the company announced updated safety and efficacy data from the severe sickle cell disease (SCD) study of its lead candidate, renizgamglogene autogedtemcel (reni-cel; formerly known as EDIT-301). The data readout is from 28 SCD patients who have been treated with reni-cel in the phase I/II/III RUBY study. Editas is scheduled to present detailed results at the upcoming American Society of Hematology Annual Meeting in San Diego, CA.
The company is focused on developing reni-cel as a potential one-time, durable gene editing medicine to treat SCD as well as transfusion-dependent beta thalassemia (TDT). This data readout follows the June 2024 press releases, when the company last reported new positive data from the phase I/II/III RUBY study of reni-cel in 18 patients with SCD and an early-to-mid-stage of the same in seven patients with TDT.
Per the latest data readout from the RUBY study, 27 out of 28 patients remained free of vaso-occlusive events. Notably, patients experienced early normalization of total hemoglobin (Hb) levels, averaging 13.8 g/dL, and significant and sustained increases in fetal hemoglobin (HbF) levels, exceeding 40%, at month six.
In the past three months, shares of Editas have plunged 41% compared with the industry’s 6.3% decline.
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More on EDIT’s SCD Study Data Readout
Efficacy results showed that SCD patients treated with reni-cel 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 four through the last follow-up. Additionally, the mean corpuscular fetal hemoglobin of F-cells increased early, with the mean value of 16.3 pg/F-cells at month four, and remained above the anti-sickling threshold of 10 pg/F-cell through the follow-up.
Per Editas, markers of hemolysis showed normalization or improvement in patients treated with reni-cel by month six and were generally maintained or improved as of the last follow-up, indicating effective management of the underlying disease pathology. Reni-cel therapy also demonstrated sustained clinically meaningful improvements in patient-reported outcome domains for pain, physical function, and social roles and activities.
Safety data from EDIT’s RUBY study showed that reni-cel was overall well-tolerated with a safety profile consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant by all evaluated patients. However, there were two serious adverse events reported in the RUBY study, possibly related to reni-cel treatment.
All evaluable patients in the RUBY study demonstrated successful neutrophil and platelet engraftment after treatment with reni-cel, which is important for limiting infection and bleeding risk.
EDIT’s TDT Study on Reni-Cel
Editas isalso evaluating reni-cel for the treatment of TDT. It has completed enrollment in the adult cohort and continues to dose patients with the candidate in the phase I/II EdiTHAL study for TDT. The company is also on track to report additional clinical data from the EdiTHAL study by the end of 2024.
Please note that in October 2024, Editas announced that it is seeking a global partner to advance reni-cel development or potentially out-license rights to the treatment entirely. As part of its strategic reprioritization efforts, Editas is set to focus resources on in vivo (within the living organism) pipeline development. The company reported that it has successfully demonstrated in vivo preclinical proof of concept for editing hematopoietic stem and progenitor cells and inducing fetal hemoglobin in humanized mice.
Based on such encouraging pre-clinical results, Editas believes that it has a clear path forward to develop a potentially first- and best-in-class in vivo gene-edited medicine for the treatment of sickle cell disease and beta thalassemia. An in vivo medicine could be more beneficial for SCD and TDT patients compared to the ex-vivo candidate, reni-cel, given administrative complexities.
Editas Medicine, Inc. Price and Consensus
Editas Medicine, Inc. price-consensus-chart | Editas Medicine, Inc. Quote
EDIT’s Zacks Rank & Stocks to Consider
Editas currently carries a Zacks Rank #3 (Hold).
Some better-ranked pharma stocks are Halozyme Therapeutics HALO, Pfizer PFE and Gilead Sciences GILD, each carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Over the past 60 days, Halozyme Therapeutics’ earnings estimates have increased from $3.95 to $4.05 per share for 2024, while that for 2025 has narrowed from $4.73 to $4.80. HALO shares have lost 17.4% in the past three months.
Halozyme Therapeutics’ earnings beat estimates in three of the trailing four quarters and matched once, delivering an average surprise of 14.86%.
In the past 60 days, Pfizer’s earnings estimates have risen from $2.62 to $2.91 per share for 2024, while that for 2025 has increased from $2.84 to $2.91. PFE shares have lost 12% in the past three months.
Pfizer’s earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 74.50%.
In the past 60 days, Gilead Sciences’ earnings estimates have risen from $3.79 to $4.32 per share for 2024, while that for 2025 has increased from $7.24 to $7.38. GILD shares have risen 11.9% in the past three months.
Gilead Sciences’ earnings beat estimates in three of the trailing four quarters and missed the same in one, delivering an average surprise of 15.46%.
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