Gamida Cell Ltd.
Q1 2021 Earnings Call Transcript

Published: 11 May 2021

[Josh Hamermesh:]

Thank you, Lodie, and 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 2021. Earlier this morning, we issued a press release summarizing our financial results and progress across the Company, which is available on our website at www.gamida-cell.com. Here with me on our call today are Julian Adams, Chief Executive Officer; Michele Korfin, our Chief Operating Officer and Chief Commercial Officer; and Shai Lankry, Chief Commercial Officer. Ronit Simantov, Chief Medical Officer; Tracey Lodie, our Chief Scientific Officer, are also on hand for the Q&A portion of the call following our prepared remarks.

[Julian Adams:]

Thank you, Josh. And thanks to everyone for joining us this morning. Those of you who've been following our progress know that Gamida Cell is approaching a major inflection point in the Company's history, as we approach a BLA submission later this year, and prepare for the potential product launch, what could be the first ever FDA approved bone marrow transplant graph product for blood cancer patients in need of a stem cell transplant. At Gamida Cell, we are dedicated to advancing two cell therapy programs that leverage our proprietary NAM cell expansion platform with the potential to redefine standards of care for patients with blood cancers and serious immunologic disorders. This platform continues to demonstrate compelling results in clinical trials based on critical features, such as cell expansion, functionality and safety, all leading to improve patient outcomes. 2021 has gotten off to a strong start marked by continued progress across our pipeline towards key milestones. We continue to be encouraged by the clinical profile of our product candidates, omidubicel and GDA-201. Omidubicel has the potential to transform medical practice for patients with hematologic malignancies and be the first FDA approved cell therapy for bone marrow transplant. Commercial preparations are ongoing as we work towards submitting the BLA in the fourth quarter of 2021. Our second candidate in clinical development is GDA-201, an advanced cell therapies that utilizes our NAM cell expansion technology to harness the power of the innate immune system and has demonstrated remarkable results in patients with non-Hodgkin's lymphoma specifically follicular lymphoma and diffuse large B cell lymphoma histologies.

[Michele Korfin:]

Thank you, Julian, and good morning, everyone. Today, I will review our progress on our manufacturing and launch readiness activities. As we continue to advance our breakthrough therapy, omidubicel for patients in need of an allogeneic stem cell transplant. During our Type B meeting with the FDA in December, 2020, we received clear feedback on what will be required for our commercial manufacturing facilities to be ready for BLA submission. This includes our Gamida Cell owned facility in Israel and a commercial facility for which we have a contractual relationship with Lonza. Given that neither of these commercial facilities were used for the Phase 3 omidubicel registration trial, we need to demonstrate comparability from these commercial facilities with the clinical manufacturing supply. We've made important progress preparing both of these facilities for commercial manufacturing readiness. Let me start with our state-of-the-art Israeli facility. The facility construction has been completed and our team has achieved each of our internal timeline milestones since construction was completed. We have hired, trained and qualified our initial production team, and during the first quarter of this year, our team has successfully completed the required engineering runs and aseptic process simulation for qualification. The methods validations are underway with a planned completion in the second quarter of 2021 and we anticipate finalizing our analytical compatibility runs and process performance qualification or PPQs in third quarter of '21. For the Lonza commercial facility, we are currently manufacturing clinical batches for our expanded access program and are progressing on the BLA requirements. We are currently on-track for the CMC requirements for our BLA, which we plan to submit in the fourth quarter of 2021. We believe that, there was a significant opportunity with omidubicel, specifically in the U.S. market alone as there are over 40,000 patients with hematological malignancies, who consider transplant each year. There were about 10,000 patients were actually transplanted, and then unfortunately, there are almost 9,000 patients were eligible, but not transplanted. The extensive market research we have conducted has enabled us to develop a strong launch strategy.

[Shai Lankry:]

Thank you, Michelle. Good morning everyone. Today, I will summarize our financial results for the first quarter of 2021. As of March 31, 2021, our total cash position was $174.8 million, compared to $127.3 billion as of December 31, 2020. The March 31st cash balance includes the $75 billion in gross proceeds to our recent financing with Highbridge Capital Management closed this quarter.

[Julian Adams:]

Thanks, Shai. We are dedicated to finding cures for patients with hematologic malignancies and blood disorders as well as solid tumors, and we're excited about the opportunities ahead. We could have not gotten this far without an accomplished team, and I think each of our employees for their unwavering dedication towards advancing potentially lifesaving therapies for cancer patients. With omidubicel, we expect to submit the BLA in the fourth quarter of this year and are committed to be launch ready at the time of approval. With GDA-201, we have very compelling data in lymphoma and are planning to initiate a Gamida Cell sponsored clinical study, which could potentially support registration, if the data are consistent with the Phase 1 results. 2021 is already off to a strong start and will prove to be a transformational year for Gamida Cell, and all of its stakeholders, most importantly, patients in need of better treatment options. In conclusion, we are very excited about the important achievements that Gamida Cell will make in the second half of this year including omidubicel BLA submission and initiating the Company-sponsored Phase 1/2 GDA-201 study in non-Hodgkin lymphoma. We understand the importance of our work for cancer patients, the healthcare system and society in general. As you've heard today, the Company is on a path to transform the way cellular therapies can treat patients with cancer. We look forward to updating you on our progress throughout the year, and now we will open the call for questions. Operator?

[Operator:]

And our first question comes from the line of Ted Tenthoff of Piper Sandler. Your line is open.

[Ted Tenthoff:]

Great, thank you very much, and congrats on all the progress. I'm curious what is sort of still being done for the BLA, as I'm assuming you guys with anticipated outcome, but just wanted to get a sense of your expectations around that? Thanks so much.

[Julian Adams:]

So, but let me begin and thank you, Ted, for your question. Obviously, right now, we're involved in two very distinct activities. One is making sure that our manufacturing of cell therapies is meeting all of the FDA requirements. And we're going through all of the validation and qualification runs of both our site in Israel and at Lonza. Our second supplier, and in addition, Michelle outlined, how she's building up the commercial team and Gamida Cell Assist, and services. Michelle, I don't know if you want to add anything to that.

[Michele Korfin:]

Thank you, Ted. So I'll take the BLA question and then I'll turn it back to either Julian or Ronit for the response to the outcome. So, in regards to the BLA, we are finalizing the CMC requirements for the BLA at both Kiryat Gat and Lonza. We are making very nice progress to date in both areas. So, at Lonza, we have completed a number of the initiatives required. At this point time we're focused on manufacturing commercial batches VAP, and then in the second, third, second or third quarter of this year, we will finalize the BLA requirements for Lonza. In regard to Kiryat Gat, the team has progressed very nicely in first quarter we completed engineering runs. We completed aseptic process simulation. We're in the process of completing the method validation runs, which we had discussed after our Type B meeting with FDA in December. And then we'll finalize and Kiryat Gat targeting by the roughly the end of third quarter the analytical variability and the PPQ runs. So to summarize, we have made very nice progress year-to-date at both Lonza and Kiryat Gat in terms of the CMC requirements for the BLA. So with that, let me turn it back to Julian or Ronit to answer your question in regards to the AdCom.

[Julian Adams:]

Yes, Ronit, please. Could you comment on any additional data we might -- any additional follow-up from the Phase 3 data and our plans for?

[Ronit Simantov:]

Yes, sure. Thanks. Thanks Ted. So, we will -- a very important part of the BLA, of course, is the clinical aspect of the submission and we are preparing all of the clinical sections for submission, we expect additional follow-up data on the patients in the Phase 3 study to include up to one year post transplant for every patient, or at least one year out follow back to handset for every patient with some patients having additional follow-up after that, and so all of those data will be included in the BLA submission. Now in terms of outcome, we don't know yet whether we will have one but we certainly will be prepared for one. We need to engage in advisory committee meetings.

[Ted Tenthoff:]

Make sense? Thank you so much. And I would just add, we're in extensive conversations, not only with key transplant centers, and KOLs in the field as well as regular conversations with FDA, as we enjoy break-through therapy designation. We have a kind of an open line to an FDA to continue to prepare for the BLA submission.

[Operator:]

And our next question comes from the line of Jonathan Miller of Evercore ISI. Your line is now open.

[Jonathan Miller:]

Hi guys. Thanks for taking the question. One in clarification, on manufacturing compensability in the CMC, I was under the impression that you would have to generate actual commercial product and dose patients with it in order to get that CMC compatibility. Is that true? And if so, what would we hear about those patients and when? That's one. Second, I'd love to hear your updated thoughts on potential partnerships for only ex-U.S. I think you had been talking about doing that. Do you have preferred timing for when we could expect a deal like that and what are you looking for in a potential partner ex-U.S.?

[Julian Adams:]

So, let me invite Ronit to talk about, the ongoing EAP study and the clinical compatibility from our manufacturing sites.

[Ronit Simantov:]

Sure. So, we have an open expanded access protocol, which is really a single arm protocol, where all patients receive omidubicel and we're using that to execute our clinical comparability for both of our manufacturing sites, and basically enrolling patients with product manufacturer, at the site that we need to and that it's available from. So, we don't intend to provide updates on an ongoing basis around those patients or their outcomes. We're going to treat patients from both sides. We will have data to submit to FDA for the BLA and we're on-track to do that. So, I wouldn't expect to hear anything about that. We have had conversations with FDA about the extensive data required and we're confident we'll be able to provide the level of data that they are asking for in terms of the patients treated with omidubicel.

[Jonathan Miller:]

And Josh, can you comment about omidubicel in other territories?

[Josh Hamermesh:]

Yes. So ,John, we continue to explore our strategic options for the optimal way to commercialize omidubicel outside of the United States. We've recently conducted some market research assessments to best understand the territories and the opportunities in those territories. We're not commenting or providing further guidance on timing or capabilities of potential partners. It's sort of the obvious stuff, we're looking for the best way to provide access and maximize the value of omidubicel for patients around the world.

[Operator:]

And your next question comes from the line of Jason Butler of JMP Securities. Your line is open.

[Jason Butler:]

Hi. Thanks for taking the question. First one, Michele, you talked about, the opportunity to increase the number of patients that might be eligible for a transplant. Could you maybe expand some numbers around how that opportunity compares to or differs from the population that today we know is eligible, but not matched? I guess that first bucket versus the third bucket of the opportunities you talked about before.

[Michele Korfin:]

Yes, thank you, Jason, good to hear from you. So, in regards to the three opportunities increase in eligibility was based on the physician feedback on the clinical profiles omidubicel. So, when you go back to the data that Ronit, Dr. Hurwitz and Professor Saunders has presented, we see the decrease in infection rate. That's generally the one that and also the graft versus host disease data. So, when physicians see those aspects of the clinical profile, the transplanters will say, there are some patients that I may not feel have the appropriate performance status to undergo transplant with current donor modalities, but based on the clinical profile of omidubicel, both in regards to the safety date around decreased infections decrease in graft versus host disease, but also the rapid time to nutrient graphs that they say there is a percentage of patients that I may consider now eligible omidubicel was available. So, we haven't guide on a guided on specific numbers from that portion. But what we have said is overall, once we reach a peak market share, we're probably at about 2500 patients, once we reach that peak market share, and we're excited by all three of those opportunities, both increasing eligibility, the increase or improving outcomes for patients from based on current donor modality, but also very importantly, that portion of the patients that are currently eligible but just can't find a match. That's also an incredibly important opportunity from omidubicel.

[Jason Butler:]

Right. And then thank you. And just as a follow-up on the data, you'll have a BLA. I think you mentioned you'll have one year follow-up data for most of the old patients. I guess to what extent you expect FDA to weigh one year survival rate? And is this data you think could be included in the label?

[Julian Adams:]

Thanks. Ronit, would you handle that, please.

[Ronit Simantov:]

Of course. So, we will have one year data on -- all patients will have been at least one year after transplant so that the data will include one year data on all patients. One year survival data are actually important in the transplant field. They're used as a marker for evaluation of transplant centers and transplanters are familiar with looking at one year transplant data. So, we expect those data to be important. As the study wasn't powered to detect a statistical difference in the two arms with respect to overall survival data, but we will show those data and certainly share them and include them in the BLA. And we expect to use those data to show the utility of omidubicel to whatever extent we're able to.

[Jason Butler:]

Okay, great. And then just one quick one for me. So, you mentioned before you're making progress on the client preserving cryo products, what are the next steps in terms of manufacturing and when can we hear an update on the progress towards a cryo preserved product we could use in a multicenter study?

[Julian Adams:]

Thank you for that. Tracy, could you elaborate?

[Tracey Lodie:]

Sure, I'd be happy to thank you, Jason and hope you're well. So, we continue to make fantastic progress and I can update since we spoke last that we have now finalized the cryopreservation formulation in the research setting. And we have successfully manufactured two engineering runs which are practice runs at our GMP facility, which is right out of University near there in Israel. So, the team has made significant progress now and not only finalizing the cryopreservation, but now made this to scale to clinical scale. So, the next step actually are just to continue manufacturing now for the clinical runs, so that we can store up enough inventory, and we're on-track to do that prior to the IND submission of the Phase 1/2, which we plan is on-track for the latter half of this year.

[Operator:]

Your next question comes from the line of Vernon Bernadino of H.C. Wainwright. Your line is open.

[Vernon Bernadino:]

Thanks for taking my question and congrats on the progress. Just perhaps this is a question for Michele. What reimbursement levels are currently available for bone marrow transplant or use of umbilical cord blood? And can it be initially apply for use of omidubicel established at the time of omidubicel approval?

[Ronit Simantov:]

Hi, Vernon, nice to hear from you. So, here we go to some reimbursement. Let me talk about the commercial payers in the U.S. and government. So, just taking a step back, we anticipate the majority of patients who would be receiving omidubicel would fall under private or commercial payers although Medicare certainly is an area of key focus for us too. So, let me start with the commercial payers. So, commercial payers in the transplant centers in the U.S. have confidential contracts that they negotiate in regards to their reimbursement. So, I would not be able to speak specifically on the details of that. But here's what I can tell you. We've had a number of conversations through either market research with U.S. payers or direct one-to-one communications. What payers were saying is? They would most likely reimburse omidubicel at time of FDA approval via their carve-out mechanism, which is part of the contract. So, what does that mean in practice? They reimburse the transplant center for the agreed to rates for hospitalization provider care and then they carve out the reimbursement from omidubicel, that's consistent with what they did with CAR-Ts in many cases upon FDA approval, and that's what they're saying they would do for omidubicel. We're encouraged by that, and also the transplant centers are comfortable with that approach. On the Medicare side, there are established diagnosis related groups or DRGs for allogeneic stem cell transplants. And there is also some always continual evolution of how to enhance DRGs. There was some update in the proposed in-patient perspective payment rule that just came out, but the key takeaway on the Medicare side is there are DRGs that a therapy like omidubicel could be mapped to. And then in addition, we are preparing to apply for the new technology add on payment or NTAP, which would then also support the additional reimbursement if approved. So, those are the two key avenues in terms of the commercial, and then on the Medicare side.

[Vernon Bernadino:]

I know someone asked you this before, but do you anticipate the established reimbursement to follow the pathway that was followed by CAR-T cell therapies?

[Ronit Simantov:]

So in terms of the commercial, what we're hearing from the payers is similar to what they did for in many cases, for CAR-T they would carve out the reimbursement with omidubicel. So that's something that, transplant centers and are used to getting encouraged by. So in that case, yes. In the case of Medicare, the difference between omidubicel and CAR-T were when CAR-T were approved, there was no appropriate or common DRG for them to be mapped to versus in the case of omidubicel, there are allogeneic STEM cell transplant DRGs that are already established. So, that is more difference with the CAR-T.

[Operator:]

Our next question comes from the line of Gregory Renza of RBC Capital Markets. Your line is open.

[Gregory Renza:]

Thanks for the update and thanks for taking my question. Well perhaps just one on each program, Julian just with as the omidubicel data circulated in the physician community and after a series of meetings across this year so far. What do you learn that is either reinforcing or incremental on the on the omidubicel profile that they think is worth highlighting? And then secondly, on GDA-201, just curious, what is your view on the various engineering and expansion strategies across the NK space and where perhaps, would 201 fit in there? Thank you very much.

[Julian Adams:]

So, let me direct that question to Ronit on the clinical side, a lot more contact with physicians.

[Ronit Simantov:]

Thank you and Greg, we have been in touch and reaching out to a number of transplanters across the U.S. especially many who were not investigators in our clinical study to talk to them about the results of a study and about omidubicel and to get their feedback on the results of the study. And we've been hearing an enormous amount of enthusiasm about the potential. They each have been able to think of patients that they would have been able to, or would have wanted to do the foreword available. Patients who wouldn't have fit the mold for other opportunities and transplants couldn't find a match or just weren't appropriate for some of the other modalities. And I think people are looking forward to having another option when they're looking for a grasp for their patients with illogic malignancies. So, that's what we've learned that folks who were not previously familiar with omidubicel, and are now learning about it are enthusiastic.

[Julian Adams:]

And for GDA-201, again, let me refer you to Tracy, to answer.

[Tracey Lodie:]

Sure, and thank you for the question, Greg. Good to hear from you. So as the NK field, is expanding and with many different approaches, and really information flowing very nicely. And we're really pleased to be, in this and at the forefront of this with our clinical data set. And so I'll say a few things about, GDA-201 which distinguishes us, and then where we're going in terms of the engineering. So, I think the key benefits of GDA-201 really is our quick manufacturing process, that we're able to isolate this perfect blood from normal donors have a product within 14-days. And now as I said, we're very excited to have a cryopreserved formulation of that, which has maintained the phenotype and the function in the lab of these cells. And so as a bore out in the clinical trial, we're really excited about the combined ability of our technology with other therapies and especially with other antibodies. So, I think this distinguishes us from heavily engineered CAR in case in other IPSC-derived CAR in case which required heavily on significant genetic engineering. Our cells do not persist past 7 to 10 days in the patients, what we've seen by fax analysis, at least in the IST Phase 1. But therefore, we think it's a different mechanism that's going on with GDA-201 and that their ability in part, because of our expanded with our NAM technology to maintain their function posting vivo-transplant as well as to creating our cytokine activating adaptive immune response, that we are working on still to provision some translational studies. And we think this is what's really leading to some of the durability that we've seen in the ongoing study. So, we're excited about that and to continue to explore the potential. And this is what distinguishes, our NAM technology because we believe this would not be possible without the NAM actually improving the fitness of the cells around the metabolism and really around the ability for them to be very active, when we're expanding them and not be exhausted. Where we're going in the future? The R&D team is making tremendous progress that we're really excited about and actually doing some gene editing in which we think we will improve the cells as we look to other tumor types, and especially in solid tumor types and how we can improve them, the persistence, which we think may be needed for this and also to tackle those difficult solid tumor types. So, we're making great progress and we think we have a product that is distinguished and we're looking forward to improve that as we expand out to different indications. So, thank you for the question.

[Operator:]

And I am showing no further questions. At this time, I would like to turn it back to Mr. Julian Adams for the closing remarks.

[Julian Adams:]

Thank you, everyone for joining us on today's call and we look forward to updating you in the future, and we really appreciate your continued support. And once again, I can't thank of the employees of Gamida Cell enough, particularly in this pandemic year where they just worked tirelessly and with such commitment to realize the potential of our programs for patients. Thank you all.