Yahoo Finance Editas Medicine, Inc. (NASDAQ:EDIT) Q1 2024 Earnings Call Transcript
Editas Medicine, Inc. (NASDAQ:EDIT) Q1 2024 Earnings Call Transcript May 8, 2024
Editas Medicine, Inc. misses on earnings expectations. Reported EPS is $-0.76 EPS, expectations were $-0.63. Editas Medicine, Inc. isn’t one of the 30 most popular stocks among hedge funds at the end of the third quarter (see the details here).
Operator: Good morning, and welcome to the Editas Medicine's First Quarter 2024 Conference Call. All participants are now in a listen-only mode. There will be a question-and-answer session at the end of this call. Please be advised that this call is being recorded at the company's request. I would now like to turn the call over to Cristi Barnett, Corporate Communications and Investor Relations at Editas Medicine.
Cristi Barnett: Thank you. Good morning, everyone, and welcome to our first quarter 2024 conference call. Earlier this morning, we issued a press release providing our financial results and recent corporate updates. A replay of today's call will be available in the Investors section of our website approximately two hours after its completion. After our prepared remarks, we will open the call for Q&A. As a reminder, various remarks that we make during this call about the company's future expectations, plans and prospects constitute forward-looking statements for the purposes of the Safe Harbor provisions under the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including those discussed in the Risk Factors section of our most recent Annual Report on Form 10-K, which is on file with the SEC as updated by our subsequent filings.
In addition, any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. Except as required by law, we specifically disclaim any obligation to update or revise any forward-looking statements, even if our views change. Now, I will turn the call over to our CEO, Gilmore O'Neill.
Gilmore O'Neill: Thanks, Cristi, and good morning, everyone. Thank you for joining us today on Editas's first quarter 2024 earnings call. With me today are four members of the Editas executive team, our Chief Medical Officer, Baisong Mei; our Chief Scientific Officer, Linda Burkly; our Chief Financial Officer, Erick Lucera; and our Chief Commercial and Strategy Officer, Caren Deardorf. We are pleased with Editas' momentum and progress in the first quarter of 2024. Editas' goal is to deliver life-changing medicines to patients with previously untreatable or undertreated genetically determined diseases. And our vision and focus strategy is to position Editas as a leader in in vivo programmable gene editing. Three pillars underpin our strategy.
The first of those pillars is to drive reni-cel, an edited cell therapy for hemoglobinopathies, and formerly known as EDIT-301, toward BLA and commercialization. The second is to build an in vivo editing pipeline. And the third is to increase business development activities with a particular focus on monetizing our very strong intellectual property. At the start of 2024, we announced the following 2024 objectives. For reni-cel, we will provide a clinical update from the RUBY trial for severe sickle cell disease and the EdiTHAL trial for transfusion-dependent beta thalassemia in mid-2024 and by year-end 2024. We will complete adult cohort enrollment and initiate the adolescent cohort in RUBY and continue enrollment in EdiTHAL. For our in vivo pipeline, we will establish in vivo preclinical proof-of-concept for an undisclosed indication.
And for BD, we will leverage our robust IP portfolio and business developed to drive value and complement core gene editing technology capabilities. So how are we executed against this strategy and these objectives in the first quarter? Let us start with reni-cel. First, on enrollment. We've been very pleased with the growing patient and healthcare provider interest in reni-cel. Indeed, we are delighted to share that we have completed enrollment in the adult cohort of the RUBY clinical trial. Additionally, we have enrolled multiple patients and have more in screening in the adolescent cohort of a RUBY study, which was launched at the beginning of this year. And we continue to enroll beta thalassemia patients in our EdiTHAL study. Dosing continues in both the RUBY and EdiTHAL studies.
Second, on clinical data. We remain on track to present a substantive clinical data set of at least 18 sickle cell patients with 2 to 21 months of clinical follow up in the RUBY study in the middle of 2024. And we will share a further update by year-end. We are also on track to present clinical data from the EdiTHAL study of reni-cel in transfusion dependent beta thalassemia in the middle of 2024 and again by year end. Baisong Mei will share more reni-cel detail later on in this call. On the manufacturing front, I am pleased to share that we have promoted Greg Whitehead to the role of Chief Technology and Quality Officer, leading our technical development, technical operations and quality departments. Greg has more than 25 years of experience in the biotech industry and extensive cell and gene therapy clinical and commercial development expertise.
Now, let's turn to in vivo and our pipeline development, where we continue to strengthen our in vivo discovery capabilities and continue lead discovery work on in vivo therapeutic targets in hematopoietic stem cells and other tissues. Importantly, we remain on track to establish in vivo preclinical proof-of-concept for an undisclosed indication by the end of the year. Our internal development efforts are differentiated by leveraging the indel CRISPR technology we already use to upregulate gamma globin expression through direct editing of the HBG12 promoter site in our ex vivo reni-cel program. Our in vivo approach is aimed at functional upregulation of gene expression in genetically defined diseases with a preliminary focus on rare and orphan patient populations.
In the medium to long term, we intend to expand to more common genetically determined diseases. Linda Burkly, our CSO, will share more details on our in vivo strategy and progress towards building an in vivo pipeline later on in the call. Finally, what is happening in business development? In March, we signed a two year extension to the collaboration with Bristol-Myers Squibb to research, develop and commercialize autologous and allogeneic alpha beta T cell medicines for the treatment of cancer and autoimmune diseases. We also have options to extend that collaboration for an additional two years. To date, Bristol-Myers Squibb has opted into 13 different programs across 11 gene targets to date. Two programs are currently in IND enabling studies and four programs are in late stage discovery.
And in intellectual property, yesterday, oral arguments were held before the U.S. Court of Appeals for the Federal Circuit regarding an appeal of the Patent, Trial and Appeal Boards, or PTABs, previous decision favoring Broad Institute of the US Patent Interference involving specific patents for CRISPR-Cas9 Editing in human cells between the University of California, University of Siena and Emmanuelle Charpentier, or CDC, and the Broad. We expect a decision on the case in the second half of 2024. Eric will share more BD and IP details later on in the call. We are energized by our progress and execution this quarter. With our sharpened strategic focus, our world-class scientists and employees, our keen drive and execution and strong balance sheets, we continue to build momentum to progress our strategy to deliver differentiated editing medicines to patients with serious genetic disease.
Now, I will turn the call over to Baisong, our Chief Medical Officer.
Baisong Mei: Thank you, Gilmore. Good morning, everyone. Let's talk about reni-cel, which is on the clinical development for severe sickle cell disease and transfusion-dependent beta thalassemia. As Gilmore shared, we are pleased that we have completed enrollment in the adult cohort of the Phase 1, 2, 3 RUBY trial and the dosing continues. In the adolescent cohort of the RUBY study, we have enrolled multiple patients and several more patients in screening. The interest and demand are high. I'm very pleased about how quickly we have moved in screening and enrollment of the adolescent cohort. I'd like to thank colleagues and editors and our clinical trial partners for the collaboration and hard work. And more importantly, I would like to thank patients, their families, investigators, and the study side staff for their trust and support.
In the EdiTHAL trial for transfusion-dependent beta thalassemia, we continue to move forward with enrollment and dosing. We look forward to sharing clinical data in the middle of this year and also at the year-end. As I have shared, I visited and continue to visit our RUBY's EdiTHAL clinical trial site and speak with the investigators. I appreciate the enthusiasm and support from the investigators and study sites. I'm pleased with the momentum of reni-cel in patient recruitment, apheresis and dosing in both studies. I'm excited to hear from the investigators that patients those who use reni-cel have already seen positive changes in their lives. As we shared in our February earning call, we aligned with FDA that RUBY clinical trial is now considered a Phase 1, 2, 3 trial for BLA filing.
We also have alignment with the FDA on the study design, endpoints, and sample size. We look forward to future discussions with the FDA and continue the collaboration. Turning to clinical data, as Gilmore mentioned, we are on track to present a substantive clinical data set of sickle cell patients with considerable clinical follow-up in the RUBY study in the middle of 2024 and a further update by year-end 2024. What we will show, the RUBY data set will include clinical data from at least 18 sickle cell patients with a 2 to 21 month of follow-up. And the EdiTHAL data set will include clinical data from seven patients with 4 to 12 month follow-up. We will present efficacy data including total hemoglobin, fetal hemoglobin, and the vessel occlusive event or VOE for sickle cell patients in RUBY study.
And the red blood cell transfusion or transfusion-dependent beta thalassemia patients in EdiTHAL study. And safety data including neutrophil and platelet engraftment for both studies. As a reminder, in December 2023, we shared safety and efficacy data from 11 RUBY patients and six EdiTHAL patients. Once again, the data confirmed the observation from our prior clinical readouts, including reni-cel drove early robust correction of anemia to a normal physiological range of total hemoglobin in sickle cell patients. Reni-cel drove robust and sustained increase in fetal hemoglobin level in excess of 40%. All RUBY sickle cell patients remained free of vessel occlusive events following Reni-cel treatment. Reni-cel treated sickle cell patients and transfusion dependent beta thalassemia patients have shown successful engraftment, have stopped red blood cell transfusion.
And the safety profile of reni-cel observed today is consistent with myeloablative busulfan conditioning and autologous hemopoietic stem cell transplant. This data reinforce our belief that we have a competitive product and a product potentially differentiated from other treatments with a rapid correction of anemia. Thanks to the deliberate choice of our discovery group have made early in the program. The choice of CRISPR enzyme and the target to edit for increased fetal hemoglobin expression matters. Reni-cel uses our proprietary AsCas12a enzyme to upregulate HBG12 promoter. Based on the clinical data thus far, we believe that sustained normal levels of total hemoglobin could be a potential point of differentiation for reni-cel. Now I'll turn the call over to Linda, our Chief Scientific Officer.
Linda Burkly: Thanks, Baisong. And good morning, everyone. I'm happy to be talking to you this morning to share more details about our in vivo strategy and our progress towards building an in vivo pipeline. I would like to take a moment to remind you why we believe in vivo medicines will be a disruptive transformative development in medical history with the potential to address genetically determined diseases with durable and curative outcomes for patients. First, in vivo medicines may reduce the administration burden of delivering editing medicines to patients in need, which will provide for broader access to patients all around the world. Second, off the shelf administration may allow for scalable manufacturing and lower costs to produce.
Based on these two principles, we believe that in vivo gene editing will provide accessible cures for genetic diseases and therefore may be the most disruptive development in medical history. So how will Editas position itself? There are many monogenic diseases that can potentially be cured with gene editing approach. We have said that we will at first target the development of treatments that are clearly differentiated from current standard of care and that will leverage the aspects of CRISPR editing that give it a unique advantage over other therapeutic modalities. Our internal development efforts are differentiated by leveraging the indel CRISPR technology we use to upregulate gamma globin expression through direct editing of the HBG12 promoter site in our ex vivo reni-cel program.
Our in vivo approach is aimed at functional upregulation of gene expression in genetic diseases in rare and orphaned patient populations from which we intend to expand to more common diseases. I'm also pleased to share several progress updates as we advance our in vivo capabilities towards our long-term vision of being a leader in in vivo programmable gene editing. First and most importantly, as Gilmore mentioned, we remain on track to establish in vivo pre-clinical proof-of-concept for an undisclosed indication by the end of the year. Editas is well positioned with established capabilities in the four main components of in vivo gene editing medicine. One, guide RNA, two, editing enzyme, three, messenger RNA, and four, delivery technology. And we are currently evaluating lipid nanoparticles for delivery of gene editing cargo into multiple tissue types with multiple companies.
Additionally, we're evaluating next generation delivery technology. Second, our in vivo capabilities with the potential to be used in developing transformative in vivo gene editing medicines are demonstrated by the preclinical data we are presenting at the American Society of Gene and Cell Therapy or ASGCT Annual Meeting in three presentations taking place on Thursday and Friday of this week. On Friday, in an oral presentation, we will share in vivo preclinical data from mouse studies using lipid nanoparticle mediated delivery of an optimized guide RNA and engineered ASCAS12a messenger RNA. In post-presentations on Thursday and Friday, we will share preclinical data demonstrating ASCAS12a guide RNA modifications that enable high potency gene editing in multiple cell types, including in the liver, and improve gene editing outcomes in vivo, enabling the development of in vivo gene editing medicines.
And research on identifying potent large serine recombinases, LSRs, as a foundation to develop novel in vivo gene editing technologies for whole gene knock-in, expanding potential in vivo gene editing targets for developing medicine. Third, Editas CRISPR-based in vivo gene editing capability has been clinically validated. Notably, in 2020, Editas was the first company ever to treat a human with an in vivo delivered CRISPR-based gene editing medicine, EDIT-101. In fact, earlier this week, the New England Journal of Medicine published a manuscript entitled, Gene Editing for CEP290-Associated Retinal Degeneration, detailing our former lead development candidate, EDIT101, for the treatment of Leber's congenital amaurosis type 10, or LCA10. Editas established clear in vivo human proof-of-concept in 2022, and we are pleased that the results from this groundbreaking clinical trial were published by the New England Journal of Medicine.
These progress updates demonstrate Editas' execution on our in vivo strategy and our proven in vivo gene editing capabilities. And I look forward to sharing more details about our in vivo development strategy and our progress towards building an in vivo pipeline later this year. Now, I will turn the call over to Erick, our Chief Financial Officer.
Erick Lucera: Thank you, Linda, and good morning, everyone. I'm happy to be speaking to you, and I'm excited to provide updates on our business development achievements, intellectual property, and financial results for the first quarter of 2024. First, in regard to business development, as Gilmore mentioned, in March, we announced a two-year extension to the collaboration with Bristol-Myers Squibb to research, develop, and commercialize autologous and allogeneic alpha beta T cell medicines for the treatment of cancer and autoimmune diseases. We also have options to extend the collaboration for an additional two years. To date, Bristol-Myers Squibb has opted into 13 different programs across 11 gene targets to-date. Two programs are currently in IND enabling studies, and four programs are in late stage discovery.
As a reminder, for each new experimental medicine that Bristol-Myers Squibb develops and commercializes using opted-into genome editing tools, Bristol-Myers Squibb will pay Editas Medicine potential future milestone payments. Following the approval of any products resulting from the collaboration, Editas Medicine is also eligible to receive tiered royalties on net sales. We are pleased that our Bristol-Myers collaboration has proved to be a productive partnership, and we are committed to future collaborations and partnerships that will allow for the continued access and advancement of gene editing. And in IP, as Gilmore mentioned, yesterday the oral arguments were held before the U.S. Court of Appeals for the Federal Circuit regarding the CBC's appeal of the PTAB's decision involving patents for CRISPR-Cas9 editing in human cells.
As you know, the Broad Institute has previously prevailed three times against the CBC, twice with the PTAB, and once at the Federal Circuit. The Federal Circuit's review will determine whether the PTAB correctly applied the law. It is important to remember the Court will not hear new evidence. An appellate court decision in the Broad's favor would reaffirm Editas' position as the exclusive licensor of the patents covering Cas9 use in human medicines in the U.S. It is also important to remember that only a small fraction of the IP we licensed from the Broad are involved in the ongoing USPTO interference proceedings. We expect a decision on the case in the second half of 2024. We remain confident that the Broad will once again prevail. Our IP portfolio of foundational U.S. and international patents covering Cas9 and Cas12 use in human medicines are a source of meaningful value as we believe that globally there are more than 100 Cas9, Cas12a programs in development worldwide, with the majority of the programs being developed by 10 companies.
We believe these potential deals represent a potential material source of non-dilutive capital, as evidenced by our deal in the fourth quarter of 2023 that extended our cash runway by two quarters. We look forward to future discussions. And now I'd like to refer you to our press release issued earlier today for a summary of our financial results for the first quarter of 2024. I'll take this opportunity to briefly review a few items for the quarter. Our cash, cash equivalents, and marketable securities as of March 31, $377 million compared to $427 million as of December 31, 2023. We expect our existing cash, cash equivalents, and marketable securities, together with the near-term annual license fees and contingent upfront payment payable under our license agreement with Vertex, to fund our operating expenses and capital expenditures into 2026.
Revenue for the first quarter of 2024 was $1.1 million compared to $9.9 million for the same period in 2023. The decrease relates to the January 2023 one-time sale of the company's wholly owned oncology assets and related licenses. R&D expenses this quarter increased by $11 million to $49 million in the first quarter of 2023. This increase relates to additional clinical and manufacturing costs that support the continued progression of the company's reni-cel program. The increase is also attributable to one-time payments related to sub-license and license obligations. Editas will continue to incur these types of payments, as we and our collaboration partners advance certain license programs in the gene editing space. G&A expenses for the first quarter of 2024 were $19 million, which decreased from $23 million in the first quarter of 2023.
The decrease in expense is primarily attributable, to one-time professional service expenses related to the 2023 strategic initiatives, and business development activities, as well as reduced legal and patent costs. With our BD and IP activity and a cash runway into 2026, Editas remains in a strong financial position. We have ample resources to continue the advancement of our reni-cel program, support the progression of our in vivo capabilities to develop our pipeline, and leverage our strong IP position for additional business development and licensing opportunities. With that, I'll hand the call back to Gilmore.
Gilmore O'Neill: Thank you, Erick. We are proud of our progress in the first quarter of 2024, and we look forward to continue to accelerate the momentum in 2024. As we continue to evolve from a development stage technology platform company into a commercial stage gene editing company, we look forward to continuing our transformation and sharing our progress with you. As a reminder of our 2024 strategic objectives, for reni-cel, we will provide a clinical update from the reni-cel RUBY trial for severe sickle cell disease and the EdiTHAL trial for transfusion-dependent β-thalassemia in mid-2024 and year-end 2024. We have now completed the adult cohort enrollment, and have started enrolling patients in the adolescent cohort in RUBY.
We will also continue enrollment in EdiTHAL and dosing in both trials. For our in vivo pipeline, we will establish in vivo preclinical proof-of-concept for an undisclosed indication, and for BD, we will leverage our robust IP portfolio and business development capabilities to drive value and complement core gene editing technology capabilities. As we share today, we are making significant progress in all three pillars of our strategy this quarter, including reni-cel, in vivo, and business development, including intellectual property. We enter 2024 with great momentum, and I am proud of the Editas team's significant progress towards becoming a commercial-stage company, and on developing clinically differentiated transformational medicines for people living with serious previously untreatable diseases.
As always, we could not achieve our objectives without the support of our patients, caregivers, investigators, employees, corporate partners, and you. Thanks very much for your interest in Editas. And we're happy to answer questions. Thank you.
Operator: Thank you. [Operator Instructions] Our first question comes from Samantha Semenkow from Citi. Please proceed.
See also
10 Best Cookies and Crackers Stocks to Buy and
11 Best Delivery Stocks to Buy According to Hedge Funds.
To continue reading the Q&A session, please click here.
