Gamida Cell Ltd.
Q1 2019 Earnings Call Transcript

Published: 12 May 2019

[Operator:]

Good morning everyone. Welcome to today's call during which we will provide an update on the company and review our financial results for the First Quarter of 2019. With me here today are Julian Adams, Chief Executive Officer, Ronit Simantov, Chief Medical Officer, Shai Lankry, Chief Financial Officer and Tom Klima, Chief Commercial Officer. Following our remarks we will open up the call for Q&A. We also issued a press release this morning summarizing our financial results and progress across the company, which is available on our website at www.gamida-cell.com. During this call, we may make forward looking statements about our future expectations and plans, including clinical development objectives, the therapeutic potential of our product candidates, our operational plans and strategies and projected operating expenses and cash runway. Our actual results may differ materially from what we project today due to a number of important factors, including the considerations described in the risk factors section of our registration statements on an annual report Form 20F filed with the SEC on February 25, and other filings that we need to submit with the SEC from time to time. These forward looking statements represent our views only as of today, we caution you that we may not update them in the future, whether as a result of new information, future events, or otherwise. Now I'd like to turn the call over to Julian Adams.

[Julian Adams:]

Thank you, Jaron. And thanks to everyone for taking the time to join us this morning. At Gamida Cell we are leveraging our proprietary cell expansion technology or nanotechnology to bring new and potentially curative cell therapies to patients with hematologic malignancies and serious blood disorders. Our most advanced product candidate NiCord is in Phase 3 development and is designed to enhance the life-saving benefits of bone marrow transplantation. We are also developing NAM-NK, an investigational natural killer cell immunotherapy, which is in Phase 1 development. First, I want to highlight this name changes we announced today for our two programs. The United States Adopted Names or USAN Council recently selected omidubicel as the proprietary name for our investigational NAM-expanded cells. Going forward, we will refer to NiCord as omidubicel. The selection of a non-proprietary NAM marks a milestone and clinical development and highlights our progress toward bringing omidubicel to patients. We also changed our designation for NAM-NK GDA-201, this reflects our plans to build a multi-product company and allows us to standardize designations for future development candidates. A key priority this year is finishing enrollment in the Phase 3 study of omidubicel in patients with high risk hematologic malignancies. We expect to complete enrollment by the end of this year, leading to top line data in the first half of 2020. If the data are positive, we anticipate submitting the biologics license application or BLA for omidubicel in the U.S. in the second half 2020 with potential FDA approval and launch expected in 2021. We will use a common technical document for our regulatory filing which enables us to streamline the work required to file a marketing authorization application for MAA for omidubicel in Europe. We have started ramping up activities required for a successful product launch. Earlier this year Tom Klima joined Gamida Cell as the company's first ever Chief Commercial Officer. Tom brings nearly 20 years of global experience in the pharmaceutical industry with significant knowledge in cell therapy, hematology oncology and transplantation. He has led the launch of six products including novel first in class therapies in the field of oncology. Tom joined Gamida Cell at just the right time to begin establishing the team, the infrastructure and the support services needed to bring omidubicel to patients. We also nominated two Board members, Shawn Cline Tomasello and Stephen Wills who bring key commercial, operational and financial expertise to our Bboard of Directors. Bone marrow transplantation is one of the earliest forms of cell therapy, and we are pleased to be at the forefront of revolutionizing the field for the development of omidubicel. We are also witnessing the emergence of the next generation of cellular medicines, harnessing the innate immune system through natural killer cells or NK cells is garnering interest as a new approach in cancer immunotherapy. We are encouraged by the early data generated in our Phase 1 investigative sponsored study of GDA-201. Emboldened by the multiple complete responses reported earlier this year, we are scaling up our manufacturing process to enable the evaluation of a cryopreserved formulation of GDA-201 in a multi-center, multi-dose Phase 1/2 clinical study in patients with non-Hodgkin lymphoma next year. We are excited about the significant opportunity with our NK program because we have the potential to combine NK cells with a broad range of antibodies. This is allows us to evaluate solid tumors, in addition to hematologic malignancies and to further improve their effectiveness through genetic modification. Earlier this year, we established a research agreement with Editas medicine to support our efforts in developing even more effective NK cells. The agreement allows the two companies to explore whether gene editing could increase the persistence and potency of our NK cells. I will now turn the call over to Ronit Simantov. Our Chief Medical Officer to review our clinical studies in more detail.

[Ronit Simantov:]

Thanks, Julian. Good morning to everyone joining the call today I'm pleased to share an update on both the omidubicel and the GDA-201 program. Omidubicel is the first bone marrow transplant product to receive breakthrough therapy designation from the U.S. Food and Drug Administration and has also received orphan drug designation in the U.S. and Europe. While bone marrow transplant have curious potential. It is estimated that more than 40% of eligible patients do not receive a transplant for a number of reasons, including the inability to find a matched donor. Additionally, bone marrow transplants have risks, including the risk of infection and other complications such as graft versus host disease. We believe that omidubicel has the potential to address these limitations and to improve treatment outcomes for patients Our Phase 1/2 clinical study of omidubicel was recently published in the Journal of Clinical Oncology. This study evaluated the safety and activity of omidubicel in 36 patients with high risk hematologic malignancies who needed a bone marrow transplant but did not have a suitable match donor. The data demonstrated that patient transplanted with omidubicel had rapid and durable engraftment of neutrophils and platelets. The median time to neutrophil recovery was shortened by nearly 50% for patients who received omidubicel compared to a real world cohort of patients who received standard umbilical cord blood. The neutrophil recovery observant of omidubicel also resulted in fewer days spent in the hospital compared to the standard cohort. Furthermore, patients who received omidubicel have rapid and robust reconstitution of key immune cell, a critical factor in recovery from transplant. The strong clinical activity and safety profile observed in our Phase 1/2 study provided the basis for our Phase 3 trial in approximately 120 patients. Importantly, we thought to derisk our Phase 3 trial by keeping the enrollment criteria and the endpoint consistent with the previous study. The study is ongoing at approximately 40 centers with additional sites expected to open as we work towards completing enrollment by the end of this year. We're pleased with the level of engagement and enthusiasm from study investigators as historically randomized trial in bone marrow transplants have been difficult to enroll. It's gratifying to have the backing of the transplant community and we truly appreciate the efforts and participation of the investigators and patients as we seek to complete this registration of study. Positive Phase 3 data would be an important step forward toward making bone marrow transplant safer and more accessible to patients with life threatening blood cancers. In addition to the ongoing Phase 3 study, omidubicel is being evaluated in an investigator sponsored Phase 1/2 study and patients with severe, a classic anemia, a rare and life threatening blood disorder. Last February, we recorded initial data at the transplantation and cellular therapy or TCP meeting. The data showed that all three patients enrolled in the first cohort successfully underwent a bone marrow transplant consisting of omidubicel plus a hematopoietic stem cell graft following a reduced intensity conditioning regimen. The next step is to initiate a second cohort of approximately 20 patients who will receive omidubicel as a standalone graft. We expect enrollment into this cohort to begin soon, in addition to the potential for omidubicel to treat patients with non-malignant blood disorders this study supports future evaluation of omidubicel more broadly using a reduced intensity conditioning regimen which could make omidubicel a more feasible treatment option for elderly or failed patients and others unable to withstand a full conditioning regimen. I'll now turn to our second development program, GDA-201, an investigational NK cell immunotherapy. While NK cells have been recognized as a possible breakthrough approach to treating various cancers, a key limitation has been generating sufficient numbers of functional cells and culture. We are using our NAM technology to overcome this limitation. At the TCP meeting, we reported encouraging initial data from the first 14 patients and our Phase 1 study of GDA-201 in combination with an antibody and patients with relapse or refractory non-Hodgkins lymphoma or multiple myeloma. The primary focus of the study was to evaluate the safety of GDA-201 in combination with rituximab and patients with non-Hodgkins lymphoma, or in combination with elotuzumab in multiple myeloma. We were pleased to see that GDA-201 was generally well tolerated in these heavily pre-treated patients with advanced disease. Among the six patients with non-Hodgkins lymphoma, a valuable for activity, we were thrilled to see three complete responses and one partial response. Two of the patients who received -- who achieved a complete response subsequently received a bone marrow transplant with the intention of curing their disease. We also reported a complete response in a patient with multiple myeloma. This Phase 1 safety and dose finding study have exceeded our expectations in terms of the clinical observed in a patient population with very advanced disease. We expect additional data from the study to be presented in the second half of 2019. As Julian mentioned, these data compel us to move forward with our clinical development plans and we anticipate initiating a multicenter multi-dose Phase 1/2 clinical study of GDA-201 in patients with non-Hodgkin lymphoma next year. As a hematologist oncologist, I'm very excited about the potential to cure patients with omidubicel and to elicit complete responses with GDA-201 in patients who are facing a life threatening prognosis. Our clinical team is intensely focused on executing our plans to move these programs forward as we seek to bring the next generation of cell therapies to patient. I will now turn the call over to Shai to review a financial results.

[Shai Lankry:]

Thank you, Ronit. Good morning, everyone. If you read from Julian and Ronit's we are pleased with the advancements we have made so far this year and I continue to build on this progress over the course of the year. I will now summarize our financial results for the first quarter of 2019. As of March 31 2019, we had total cash, cash equivalents and available for sale securities of $50.3 million, compared to $60.7 million as of December 31, 2018. Research and Development expenses for the first quarter were $7.3 million, compared to $5.5 million for the same period last year. Difference was mainly related to clinical activities to advance the omidubicel and GDA-201 programs. General and administrative expenses were $3.8 million for the first quarter compared to $1.7 million for the same period last year. This increase was due mainly to the $1.5 million increase in cash and non-cash expenses related to Ireland and establishing the U.S. headquarters and 0.6 million increase in professional services, rent and other expenses. Finance expenses net were 4.4 million for the quarter compared to 0.7 million in the same period in 2018. The increase was primarily due to non-cash expenses resulting from revaluation of [indiscernible] related to grants received from the Israel Innovation Authority. Net loss for the first quarter of 2019 was 15.5 million, compared to a net loss of 7.4 million in the same period in 2018. We continue to anticipate cash used for ongoing operating activities in 2019 to range from $35 million to $40 million reflecting anticipated expenditure to advance our current clinical programs. We expect with our current total cash position will support our capital need through the data readout for the Phase 3 clinical study of omidubicel which is expected in the first half of 2020. This cash runway guidance is based on our current operation of [indiscernible] and excludes any additional funding or business development activities. With that I will turn back the call to Julian

[Julian Adams:]

Thanks, Shai. This is an exciting time in cell therapy and an important time for omidubicel. Omidubicel, our aspiration is to bring the first FDA approved cell therapy for bone marrow transplantation to patients. We look forward to completing patient enrollment in our Phase 2 study by the end of this year, and subsequently reporting the data that could enable regulatory filings in the U.S. and Europe. With GDA-201, our aspiration is to develop off-shelf cryopreserved cell therapy without many of the side effects currently commonly associated with CAR-T therapy. We are working on several activities including clinical study planning and manufacturing our cryopreserved products. This will enable the initiation of a multicenter multi-dose Phase 1/2 to study of GDA-201 next year. We have a strong team in place committed to moving these programs forward and look forward to provide an update on our progress throughout the year. Now we will open the call for questions.

[Operator:]

[Operator Instructions]. Our first response is from Gregory Renza of RBC Capital Markets. Please go ahead.

[Gregory Renza:]

I just want to start with the omidubicel pivotal. You've mentioned some good enrollment centers coming online. I think I heard 40 centers, just want to get your comments on, certainly your mention of historical challenges with enrollments in what you're doing to perhaps overcome those barriers to actually get good traction and good uptake.

[Ronit Simantov:]

Randomized trials, in general are difficult to enroll them, [indiscernible] and bone marrow transplantation have also been quite difficult. There have been a few in the past for years and it's struggled with enrollment. So we're aware of that. What we're doing is working very closely with the transplant community with one on one interaction and engagement with the transplanters and their sites and staff. And as we do that they recognize and our PI have gone out and actually said that this is a really important study to complete. They know that this is going to move the field forward and it's I think helpful to engage with the investigators and emphasize that. So we do anticipate completing the patient enrollment in the timelines that we stated

[Gregory Renza:]

And about how many centers would you expect to get up to?

[Ronit Simantov:]

We're looking to open another 5 to 10 centers by the end of the study.

[Gregory Renza:]

Great, thanks. And just one more if I may, just more broadly, Julian and team just on the cord blood market in general, the fact that the absence of perhaps standardization among research centers, among medical centers, I'm curious of your thoughts there, how you see NiCord perhaps fitting in to that logistical schematic and for solving for maybe the variability across practices in those settings? Thanks.

[Julian Adams:]

Well, it's actually a question, I think, with the FDA approval, there will be a standardized, quality controlled manufactured product that will come from our manufacturing sites. Hopefully, we will replace cord blood transplantation if this study is positive, and this will establish a new standard of care and it will also be the first ever FDA approved product in the field of bone marrow transplantation. So I think allowing for a standardized transplant graph really does move the field forward. This doesn't affect the way patients are conditioned, or the [indiscernible] transplant management of patients that does vary from site to site and we've allowed for that in the design of our trial.

[Operator:]

Thank you. Your next response is from Chad Messer of Needham & Company. Please go ahead.

[Unidentified Analyst:]

This is Galon [ph] for Chad. So a question about GDA-201 I know this is a little bit early considering this study is only starting next year, but could you discuss a little bit about what kind of patients you're looking to do the study and then maybe a potential festive market strategy? Thank you.

[Julian Adams:]

Let me begin. So that we're anticipating a cryopreserved product which would enable a multicentered, multi-dose approach. We haven't designed the final study yet and really make comment we will likely have an ad board. So I will let Ronit expand on that.

[Ronit Simantov:]

Yes, I think you know, our first study, as we expand will focus on patients with non-Hodgkins lymphoma and we will find the appropriate patient population to treat. We will engage with investigators in order to design the study more specifically. We'll have more news for you on that as we get closer to opening up that study.

[Julian Adams:]

I think importantly, we've seen a signal that's strong enough that compels us to start planning for this study. As you know, in cell therapies, there's a long lead time before being able to file the IND. But witnessing complete responses in this heavily pretreated patient population, for me is a very exciting sign.

[Operator:]

Thank you. Your next response is from Matthew Luchini of BMO Capital Markets. Please go ahead.

[Unidentified Analyst:]

This is Stephen on for Matthew. So previously, it was reported that omidubicel maybe looking for a partner for ex-US expansion. So now that there's been a number of steps forward in preparation for the commercialization of omidubicel could you confirm that that's still an option being looked at and looking for any potential partner?

[Julian Adams:]

So let me comment at a high level. We're constantly looking to optimize our business, which includes partnering. However, at this time, since we're in the middle of a Phase 3 study and prior to data, it's quite unlikely that we would secure such a partnership but I'd like to ask Tom, to comment on our commercial readiness, because we do intend to file both in the U.S. and Europe.

[Thomas Klima:]

Obviously, we're planning to file the MAA in Europe and about the same time as the BLA in the U.S. and so we're planning for simultaneous launch at the same time, as Julian said, we're evaluating all potential options which obviously could be to launch by ourselves but certainly also not going to rule out a partnership at this time. So we're simultaneously planning for either option and will be ready to launch if we go alone.

[Julian Adams:]

And I would just amplify on that. It only adds to the value of the program if we do the filing and secure licensure in Europe. It just makes it more attractive for any partner. So the more we do, the better. the economics will be downstream should we seek a partnership and it may turn out that we want to go it alone in Europe, so we're not ruling anything out.

[Operator:]

[Operator Instructions]. Your next response is from Mark Breidenbach of Oppenheimer. Please go ahead.

[Unidentified Analyst:]

This is Matt [ph] on for Mark. I know it's early, but are you able to provide us with some ideas to the extent of the GDA-201 update later this year, in terms of additional patients treated or extended follow up from the prior dataset?

[Ronit Simantov:]

We do intend to provide an update at the end of the year and we certainly will have the extended follow up on the patients who've already been reported on and enrolled in the study. We can't comment on the exact number of patients that will be included in that follow up but we can say that we do continue to enroll patients in the study.

[Unidentified Analyst:]

Okay, and then maybe another follow up on GDA-201. So we were wondering if you are starting to reach the upper limits of manufacturing capabilities now that you're at the third dose, and whether that may have played a role in the decision to stop this escalation and then as a related follow up, I know you're looking into cryopreservation but we're also wondering whether you're considering alternative sources such as cord blood derived NK cells for you from the future?

[Julian Adams:]

So, let me remind you that our current manufacturing process which has been scaled up allows us to manufacture 50 to 100 billion cells in a period of two weeks. So our protocol designed that to be the highest dose. As we consider the third dose as the NTV or the working NTV and we are preparing a cryopreserved product this will allow to us to do a multi-dose study. We haven't designed the schedule yet, but it'll allow us to explore a schedule where we're giving sufficient number of potent NK cells. Forgive me there was this last part of your question that I -- could you repeat it?

[Unidentified Analyst:]

Yes, I was just wondering whether you're considering alternative sources such as cord derived NK?

[Julian Adams:]

No, we're very pleased with the apheresis [ph] that is achieved from all normal healthy donors. We don't believe we need to access cord blood NK cells. We have a very robust process that we've been using throughout the year and the study in Minnesota.

[Operator:]

Thank you. There are no further questions at this time. I would like to turn the call back over to Julian Adams

[Julian Adams:]

Thank you everyone for joining us on the call today. We are continuing to work hard to bring advanced cell therapies to patients with limited treatment options. We look forward to providing additional updates as our programs progress.