Gamida Cell Ltd (GMDA) 2020 Q1 法說會逐字稿

Ladies and gentlemen, thank you for standing by, and welcome to the Gamida Cell's conference call for the first-quarter 2020 results. My name is Chris, and I will be your operator for today's call. (Operator Instructions) Please be advised that this call is being recorded at Gamida Cell's request. (Operator Instructions)

Now I'd like to introduce your host for today's conference, Ms. Jaren Madden, Vice President of Investor Relations and Corporate Communications. Please go ahead.

Thank you, Chris, and good morning, everyone. Welcome to today's call where we'll provide an update on the company and review our financial results for the first quarter of 2020.

Earlier this morning, we issued a press release summarizing our financial results and progress across the company, which is available on our website, www.gamida-cell.com. You can find the press release related to today's call. Here with me on our call, today, is Julian Adams, Chief Executive Officer; Ronit Simantov, Chief Medical Officer; and Shai Lankry, Chief Financial Officer. Following our prepared remarks, we'll open the call for Q&A.

During this call, we may make forward-looking statements about our future expectations and plans, including clinical development and commercial objectives, the therapeutic potential of our product candidates, operational plans and strategy 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 Form 20-F and in other filings that Gamida Cell makes with the SEC from time to time. These forward-looking statements represent our views only as of today and we caution you that we may not update them in the future, whether as a result of new information, future events or otherwise.

And now I'd like to turn the call over to Julian Adams.

Thank you, Jaren, and thanks to everyone for joining us this morning. At Gamida Cell, we are committed to finding cures for patients with blood cancers and rare serious immunologic diseases through the development of next-generation cell therapies. Last week, we were thrilled to announce that the global randomized Phase 3 study of omidubicel, our most advanced product candidate met its primary endpoint with a high degree of statistical significance.

We are also making strong progress with GDA-201, our investigational expanded natural killer or NK cells therapy with the potential for both hematologic malignancies and solid tumors. Additionally, we announced a $60 million follow-on earlier this week, which is expected to close today. As we have experienced minimal impact due to COVID-19 today, we have made significant progress so far this year as we advance our programs.

Starting first with omidubicel. Last week, we reported positive, highly significant topline data from our global randomized Phase 3 clinical study of omidubicel in patients with high-risk hematological malignancies in need of a bone marrow transplant. The data demonstrated that omidubicel resulted in a significant reduction in time to neutrophil engraftment, a key milestone in transplant recovery. These data exceeded our expectations and were very consistent with our results from our Phase 1/2 clinical study.

These data underscore the potential for omidubicel to create a new standard of care. Based on the results of the Phase 3 study, we are confident that omidubicel could be served as a graft for any patient in need of a bone marrow transplant. By providing a readily available bone marrow transplant graft, we can reduce the time patients currently spend waiting for a donor match. We can also help relieve patients and their families of the anxiety they currently feel during the search process. Additionally, omidubicel can make transplant accessible to the 40% of patients who today are eligible for transplant, but unable to find a matched donor.

With these data in hand, we are focused on working towards initiating a BLA submission on a rolling basis in the fourth quarter of this year, which will position us for a potential launch in the second half of 2021. We are also advancing key activities to bring omidubicel to patients following potential FDA approval. Work is ongoing to build out our manufacturing infrastructure, both at Lonza and at our own facility to help ensure sufficient and reliable commercial supply. We are also working to develop comprehensive hospital services and patient assistance programs designed to seamlessly bring omidubicel patients.

Moving now to GDA-201. We are continuing to advance Phase 1 clinical study and are working hard to initiate a multi-center Phase 1/2 study in patients with lymphoma next year. We have previously reported striking early signs of efficacy with multiple complete responses in patients with advanced lymphoma. We expect to present updated Phase 1 data at a medical meeting in the second half of the year.

We are quite fortunate that the impact of COVID-19 on our business has been minimal and our hearts go out to all the families who have been affected by this tragic pandemic. At Gamida Cell, we have undertaken important steps to help ensure safety of employees and their families and to reduce the spread of COVID-19.

In early March, Gamida Cell established a work-from-home policy for all employees other than those performing or supporting business critical laboratory experiments and manufacturing-related activities. For those employees, the company has implemented stringent safety measures designed to comply with applicable government guidelines institute in response to the COVID-19 pandemic.

Today, we believe our guidance with respect to clinical development and regulatory milestones are unchanged. We will continue to closely monitor any possible impact from COVID-19, and we'll provide updates on any changes that occur.

I'll now turn the call over to Ronit Simantov, our Chief Medical Officer, to provide a further update on omidubicel and GDA-201. Ronit?

Thank you, Julian, and good morning, everyone. As Julian noted, last week, we reported positive topline data from our Phase 3 study of omidubicel. We are very proud of this rigorous, well-executed trial. And we truly appreciate the support of the transplant community, including the investigators and their team, the patients and their families who partnered with us to help move the [fields forward].

This Phase 3 study was designed to evaluate the safety and efficacy of omidubicel compared to standard umbilical cord blood [in the] allogeneic bone marrow transplant in patients with high-risk hematologic malignancies. Demographics and baseline characteristics were well-balanced across the two study groups and the primary endpoint results represented an intent-to-treat analysis of all 125 randomized patients.

The primary endpoint was time to neutrophil engraftment, the key milestone in recovery from bone marrow transplant, signifying how quickly the stem cells the patient received became established and began to make healthy new cells. We certainly define engraftment as achieving an absolute neutrophil count of greater than or equal to 500 cells per microliter on three consecutive measurements on different dates with subsequent donor chimerism.

Neutrophils are infection-fighting white blood cells and chimerism is genetic evidence that the donor cells have engrafted. In the intent-to-treat analysis, the median time to neutrophil engraftment was 12 days in patients randomized to omidubicel compared to 22 days for patients in the comparator group randomized to standard cord blood transplant. The p-value was less than 0.001. Importantly, this was a clinically significant difference because faster engraftment is associated with fewer infections and shorter hospitalizations, which is meaningful for patients, physicians, and the hospital.

These data were consistent with our Phase 1/2 study where we reported a median time to engraftment of 11.5 days for patients who were treated with omidubicel compared to 21 days for a historical cohort of 146 patients treated with standard cord blood. These data also compare favorably to the time to neutrophil engraftment that has previously been recorded in other study for other transplant modalities where we have seen data ranging from 16 to 21 days.

We remain blinded to individual patient outcomes and data on additional endpoints continues to be collected as they mature. Secondary endpoints include time to platelet engraftment, infections and hospitalizations and additional endpoints include graft-versus-host disease, immune reconstitution, and survival. We expect to present the data at a medical meeting at the end of the year.

We are also evaluating omidubicel in an investigator-sponsored Phase 1/2 study in patients with severe aplastic anemia, a rare and life-threatening blood disorder. Last year, we completed the first cohort of this study which showed that all three patients successfully underwent a bone marrow transplant consisting of omidubicel plus a haploidentical stem cell graft. Currently, patients are being enrolled into the second cohort, which is designed to evaluate omidubicel as a standalone. We expect to report additional data from this study in the second half of this year.

In addition to omidubicel, we are also advancing our second cell therapy program, GDA-201, a natural-killer-based therapy. The ongoing study in patients with non-Hodgkin lymphoma and multiple myeloma is designed to assess the safety of GDA-201 in combination with monoclonal antibodies and to determine the recommended Phase 2 dose. We have already achieved one objective. And as additional patients are enrolled, we continue to be very encouraged by the safety and activity observed.

In February, an abstract was published in conjunction with the European Society for Blood and Marrow Transplantation or EBMT Annual Meeting, which was to take place in March, but was subsequently postponed due to COVID-19. These data included 11 patients with non-Hodgkin lymphoma and 14 patients with myeloma. I'll briefly review the data in lymphoma.

Among the 11 patients with non-Hodgkin lymphoma, 7 achieved a complete response and 1 patient achieved a partial response. While this is a small dataset from a single site, the activity observed in these heavily pre-treated patients, including those with diffuse large cell lymphoma, compared favorably with responses observed in other studies, including early studies of CAR-T therapy. We also continue to be impressed with the safety profile of GDA-201. In 25 patients, there were no dose-limiting toxicities and no GvHD, and importantly, no neurotoxicity observed.

With the primary objectives of the study complete, we have an opportunity to evaluate the duration of response and to ask additional questions to inform future development plans. For example, we have successfully reached [re-treated] patients with GDA-201 without lymphodepletion. In multiple myeloma, we are evaluating the addition of pomalidomide to the regimen, which may provide better activity than elotuzumab with GDA-201 alone.

In summary, we are very excited about the potential of GDA-201. We expect to report additional data in the second half of 2020, and we are advancing the activities required to enable the initiation of a multi-center Phase 1/2 in patients with lymphoma next year.

With that, I will turn the call over to Shai to review our financial results.

Thank you, Ronit, and good morning, everyone. As Julian mentioned, we are pleased to close our $60 million follow-on offering today, which importantly, [strengthen] our runway into the second half of 2020.

Now I will review our 2020 first-quarter financial results. As of March 31, 2020, we had total cash, cash equivalents, and available-for-sale securities of $40.3 million compared to $55.4 million as of December 31, 2019. The $40.3 million exclude approximately $60 million gross proceeds from the follow-on offering, which we expect to close today.

Research and development expenses for the first quarter was $7.9 million compared to $7.3 million for the same period in 2019. The increase was primarily due to clinical activity related to the advances of GDA-201, offset by grants received from the Israel Innovation Authority. Commercial expenses were $1.5 million for the quarter compared to $1 million for the same period in 2019. The increase was mainly attributed to omidubicel's commercial readiness activities.

General and administrative expenses were $3 million for the quarter compared to $2.8 million for the same period in 2019. The increase was mainly due to expenses associated with being a publicly-traded company.

Net finance income was $1.7 million for the quarter compared to net finance expense of $4.4 million for the same period in 2019. The increase was primarily due to non-cash expenses resulting from revaluation of warrants. Net loss for the first quarter was $10.6 million compared to a net loss of $15.5 million for the same period in 2019.

At the beginning of the year, we provided guidance for the first six months of 2020. Today, we are updating our guidance to reflect full-year expectations. We expect cash used for ongoing operating activities this year to range from $60 million to $70 million. We anticipate that our current total cash position will support our ongoing operating activities into the second half of 2021. This cash runway guidance is based on our current operational plans and excludes any additional funding that may be received for business development activities that may be undertaken.

With that, I will turn the call back over to Julian.

Thanks, Shai. 2020 has proven to be a transformation year for the -- quarter for the company. The Phase 3 data further de-risk omidubicel and provide us with an opportunity, an important opportunity, to bring up a potential cure to patients in need of a bone marrow transplant. We look forward to reporting the data from the Phase 3 study, including secondary endpoints in the second half of the year. At the same time, we are focused on critical activities, including filing our BLA and ensuring commercial readiness as we look forward toward the potential launch of omidubicel in 2021.

With GDA-201, we have a highly active clinical development candidate in an area of science that is increasingly recognized as holding potential to further transform how certain cancers are treated. We have a strong team in place, committed to delivering the next generation of cell therapies to patients and who look forward to further updating you on our progress.

Now we will open the call for questions. Operator?

Thank you. (Operator Instructions)

Gregory Renza, RBC Capital Markets.

Good morning. This is [Ina Lu] on for Greg. Thank you for taking my questions and congrats on the progress.

Thank you.

I was wondering if you could share some feedback on the topline data now that's released in terms of what [could] KOLs and investors you're latching onto? And also what are the pushbacks and how that's shaping your focus for the awareness and alignment that you need to build as you go forward with the regulatory and also to establish the value proposition, increased visibility in the market and therapeutic space? Thank you.

Ronit?

Sure. I'll give you the impression that we have had in the transplant community. The physicians and investigators who participated in the study are very, very excited to see these data. They are really behind us in terms of developing omidubicel and are very, very gratified to see that the results of the study were so convincing and so clinically meaningful for their patients. They really see neutrophil engraftment as being critically important in the recovery of patients for transplant. And so, they believe that we really have the opportunity to be disruptive to the transplant community.

Having had the primary endpoint be complete and successful, we will have breakthrough therapy designations with the FDA. Until this time, we discussed the clinical development throughout the program with FDA. We expect to take these data and submit them in a BLA by the end of the year.

And I'll add to that. We're going to build out a medical affairs and medical education platform, engage KOLs and really broadly educate the transplant community as to the potential for omidubicel. And this will be done in conjunction with both the clinical department and the commercial group to prepare the market for omidubicel's launch -- potential launch next year.

Great. Thanks. That's very helpful. Maybe just another on the GDA-201 one program. How are you balancing the focus on that program and is that an increasing focus now? And would it make sense to accelerate the program? Would that be a possibility? How will we think about the strategy there? Thanks.

The GDA-201 is a very exciting program for us. It's based on the platform that brought us the omidubicel program and a lot of the experience, knowledge, and relationships that we've made are ones that we are leveraging for development of GDA-201. We continue to develop that program with the development of a cryopreserved product in our laboratories, and we will intend to bring that to a clinical study, a company-sponsored multi-center study, for patients next year. So, that program is quite important to us on the heels of omidubicel and we will continue.

Great. Thank you very much.

Chad Messer, Needham & Company.

Great. Thanks. Good morning and again, congrats on all the progress. Maybe a couple more (technical difficulty) on GDA-201. In terms of the data update in the second half, just wondering if you could maybe set expectations, in particular, on whether we can expect to see more data on redosing?

Absolutely. We incorporated redosing into the protocol last year -- at the end of last year in order to give patients the [opportunity] to have a second dose. And that second dose is done without lymphodepletion, so that gives us a lot of experience on eliminating lymphodepletion from the patient regimens. Additional data that will be presented by the end of the year will include multiple dosing in patients, including the lack of or the elimination of the lymphodepletion for those doses.

All right. That's very exciting. And maybe just to expand a little bit on a prior question. Do you think there's a fast-to-market strategy in NHL? And then, any further thoughts -- we've talked about this in the past -- on other indications besides NHL and multiple myeloma?

Absolutely. In non-Hodgkin lymphoma, in both patients with [sickle] cell disease as well as [follicular] lymphoma, we've seen very impressive responses, including a high percentage of complete responses. And we really feel that this is a population where patients had been treated with multiple therapies, have been refractory to standard of care and there's a need for the therapies in these patients. It's making a real clinical difference in the patients who've been treated on this study.

And so, we do feel that there's a potential fast-to-market strategy with the response rates at the level that we've already observed them in our Phase 1 study. We believe that a study that will be designed for look at response rates in a population of patients like this one could potentially lead to an approval in this patient population. So, absolutely.

In terms of other indications, we believe that the natural killer cell program is really a platform that can be used in conjunction with monoclonal antibodies to generate ADCC, antibody dependent cellular cytotoxicity, potentially for other tumors. And so, we and the laboratory have generated some data using other antibodies, including in solid tumors. And we hope to be able to bring that to patients at some point in the future to explore the capabilities in other tumors.

All right. Great. And you're obviously excited for omidubicel, but I'm looking forward to hearing more about this program as well. Thank you.

Thank you.

Thank you, Chad.

Thank you. (Operator Instructions) Jason Butler, JMP Securities.

Hi. Thanks for taking the questions. I had two. Just the first on, Julian, your comments about getting the medical affairs effort out and discussing the omidubicel data. Can you just talk to where you are in that process from a medical affairs leadership perspective? Or when do you think you'll be able to be out there talking about the topline data versus later in the year having both the secondary endpoints?

And then, on GDA-201, can you just talk about the progress you've made towards getting a manufacturing process applicable to a multi-center study? And if you're working or plan to work with a manufacturing partner at some point on that program also? Thanks.

Yes. We are actively recruiting for medical affairs talent and it was dependent on the successful financing so that we would have the wherewithal to continue to build out all of the infrastructure, both for medical affairs, commercial and manufacturing. So, this triggers our ability to go out and these data trigger our ability to go out and begin a real education process.

To your second question on manufacturing, we are undertaking to manufacture the NK GDA-201 product in our own facilities. And the key advantage is to -- now that we've learned how to cryopreserve and recover NK cell activity, it is to turn that into a GMP process. So there's still some process development going on, but we're quite confident that we will achieve (technical difficulty).

Great. Very helpful. Thanks and congrats on all the progress and the Phase 3 results last week.

Thank you.

Thank you. This does conclude today's question-and-answer session. I would now like to turn the call back to Julian Adams for any for the moment.

Thank you, everyone, for joining us on today's call. We are excited about the opportunities that lie ahead and look forward to sharing updates on our progress throughout the year. Chris?

Thank you. This concludes today's call. You may now disconnect.