Q1 2022 Gamida Cell Ltd Earnings Call

May 10, 2022

Pardon me, this is the operator. Welcome to today's program.

Pardon me this is neill.

Today's program is scheduled to begin shortly please continue to standby and thank you for your patience.

[music].

The.

Ladies and gentlemen, thank you for standing by welcome to give me the sales conference call for the first quarter 2022 financial results.

Ladies and gentlemen, thank you for standing by. Welcome to Gamita Sales Conference call for the first quarter 2022 financial.

My name is Carmen, and I'll be your operator for today's call. Please be advised that this call is being

Ms Carmen and I'll be your operator for todays call. Please be advised that this call is being recorded at gummy that sells request now I would like to introduce your host for today's conference Heather Demeter.

Now I would like to introduce your host for today's conference, Heather DeBekia, Camila's sales chief of staff. Please go ahead. I'm Heather DeBekia and I'll see you next time.

<unk> Chief of staff. Please go ahead.

Thank you, Carmen, 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 2022. 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.comedicel.com.

Thank you Carmen 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 2020 too.

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 Dot <unk> Dot com.

Please note that unfortunately, our Chief Executive Officer, Julian Adams, is unable to join us this morning due to contracting COVID-19.

Please note that unfortunately, our chief Executive Officer, Julian Adams is unable to join US. This morning due to contracting COVID-19 company operations are unaffected and Julian has advised us that his symptoms are improving and he expects to recover shortly.

Company operations are unaffected and Julian has advised us that his symptoms are improving and he expects to recover shortly.

With that here with me on our call today are Michelle Bachman, our Chief operating officer, and Chief Commercial Officer, Ernie <unk>, Our Chief Medical Officer, and Chief Scientific Officer, and Shai <unk>, our Chief Financial Officer.

With that, here with me on our call today are Michelle Corfin, our Chief Operating Officer and Chief Commercial Officer, Runeet Simantop, our Chief Medical Officer and Chief Scientific Officer, and Shai Lankri, our Chief Financial Officer.

During this call we may make forward looking statements about our future expectations and plans, including in respect of the timing of initiation and progress that and data reported from the clinical trials of our product candidates anticipated regulatory filings, including the submission of the BLA for <unk> to the FDA.

During this call, we may make forward-looking statements about our future expectations and plans, including in respect of the timing of initiation and progress of, and data reported from, the clinical trials of our product candidates, anticipated regulatory filings, including the submission of the BLA for amidupacil to the FDA.

commercialization planning efforts, the potential life-saving or curative therapeutic and commercial potential of Gametacel's product candidates, including GDA-201 and Amadoubicel, and our expectations regarding our projected cash to be used for operating activities and cash runway.

Commercialization planning efforts are potential life, saving or curative therapeutic and commercial potential of <unk> product candidates, including GDA 201, <unk> and our expectations regarding our projected cash to be used for operating activities and cash runway.

Our actual results may differ materially from what we project today due to a number of important factors.

Our actual results may differ materially from what we project today due to a number of important factors, including the impact of COVID-19 pandemic on our operations VESCO progress and expansion of our clinical trials and cost impacts thereof, clinical scientific regulatory and technical development and those inherent in the Prost.

including the impact of COVID-19 pandemic on our operations, the scope, progress, and expansion of our clinical trials, and cost impact thereof, clinical, scientific, regulatory, and technical development,

and those inherent in the process of developing and commercializing product candidates that are safe and effective for use as human therapeutics.

Costs of developing and commercializing product candidates that are safe and effective for use as human therapeutics and in the endeavor of building a business around such product candidates as well as those considerations described in the risk factors section of our most recent annual report on Form 10-K, and other filings that we make with the SEC.

and in the endeavor of building a business around such product candidates as well as those considerations described in the risk factor section of our most recent annual report on Form 10-K and other filings that we make with the SEC from time to time.

Time.

These forward looking statements represent.

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.

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. Now I'd like to turn the

Now I'd like to turn the call over to Michele.

Thank you Heather and thank you to everyone for joining us this morning.

Thank you, Heather, and thank you to everyone for joining us this morning. DomedaCell had a productive quarter as we made important progress across all areas of our company. We continue to build momentum as we head into several inflection points expected this year, most near term being the full BLA submission for AmadubaCell, which we are on track to complete in the second quarter of this year.

So had a productive quarter as we made important progress across all areas of our company. We continue to build momentum as we head into several inflection points expected. This year, most near term being the full BLA submission for <unk>, which we are on track to complete in the second quarter of this year.

Additionally, our GDA 201 program advanced marked by the recent clearance by FDA of our IND and removal of the clinical hold for the cryopreserved formulation.

Additionally, our GDA 201 program advanced marked by the recent clearance by FDA of our IND and removal of the clinical hold for the cryo preserved formulation.

We are excited to have reached this milestone and are now moving forward with our plans to initiate a company sponsored phase <unk> study in patients with Follicular and diffuse large b cell lymphomas, which Ronnie will provide additional detail on.

We are excited to have reached this milestone and are now moving forward with our plans to initiate a company-sponsored Phase I-II study in patients with follicular and diffuse large B-cell lymphomas, which Ronit will provide additional detail on.

Beyond GDA 2.0.1, which is our lead program in our expanding NAM-enabled NK cell pipeline, we are also looking forward to announcing a new product candidate from our genetically engineered NAM-enabled NK cell constructs later this year. We recently announced preclinical data at the ISCT conference supporting the development of GDA 3.0.1 and GDA 6.0.1.

Beyond GDA 201, which is our lead program and our expanding Nam enabled NK cell pipeline. We are also looking forward to announcing a new product candidate from our genetically engineered Nam enabled NK cell construct later this year.

We recently announced preclinical data at the IFC T Conference supporting the development of <unk> 301, and <unk> 601.

It's an exciting time in gametocell development, and we are focused on advancing our pipeline of potentially curative cell technology.

It's an exciting time in <unk> development, and we are focused on advancing our pipeline of potentially curative cell therapies. Our continued commitment as demonstrated by the new and recent data on the <unk> that we presented this quarter focused on the <unk> encouraging clinical data in our health economic efforts, which we will comment.

Our continued commitment is demonstrated by the new and recent data on amidobacil that we presented this quarter, focused on amidobacil's encouraging clinical data and our health economic efforts, which we will comment on later on in the call.

On later on in the call.

With all we have accomplished only this past quarter, but also in the last few months, we are poised to be a leader in the development of ma'am enabled cell therapies with two promising an important clinical programs and a robust pipeline of genetically modified NK cell product candidates in preclinical development.

With all we have accomplished only this past quarter, but also in the last few months, we are poised to be a leader in the development of NAM-enabled cell therapies with two promising and important clinical programs and a robust pipeline of genetically modified NK cell product candidates and preclinical development.

The progress we achieved this quarter continues to add to the important foundation that we have established. This strong foundation is due to our dedicated employees and their continued focus on patient care.

The progress we achieved this quarter continues to add to the important foundation that we have established the strong foundation is due to our dedicated employees and their continued focused on patients and our vision of advancing therapies to help address unmet needs.

and our vision of advancing therapies to help address unmet needs. With this, I will now.

With this I will now turn the call over to Ronnie.

Thanks, Michelle and good morning, everyone. Thank you for joining us on the call. This morning.

Thanks Michelle and good morning everyone. Thank you for joining us on the call this morning.

Let me start with our lead program, Omidubicel, which has breakthrough therapy designation as well as orphan drug status and the potential to be the first FDA-approved cell therapy for stem cell transplant.

So let me start with our lead program <unk>, which has breakthrough therapy designation as well as orphan drug status and the potential to be the first FDA approved cell therapy for stem cell transplant.

Earlier this year, we were excited to initiate the rolling DLA submission for Omidubicel, starting with our non-clinical module, followed by the submission of our clinical module.

Earlier this year, we were excited to initiate the rolling BLA submission for <unk>, starting with our non clinical module followed by the submission of our clinical module.

I'm pleased that we are currently on track to complete the full BLA submission in the second quarter of this year, moving us closer to bringing this important potential therapy to patients. Our BLA for Omidubicel is supported by strong phase three results, which met all primary and secondary endpoints.

I am pleased that we are currently on track to complete the full BLA submission in the second quarter of this year moving us closer to bringing this important potential therapy to patients.

BLA for <unk> supported by strong phase III results, which met all primary and secondary endpoints.

This past quarter, we announced the presentation of new data at the Transplantation and Cellular Therapy, or TCT, meeting, as we've continued to add to the body of evidence demonstrating the potential of Omoduva cell to address unmet needs in patients undergoing allogeneic stem cell transplantation.

This past quarter, we announced the presentation of new data at the transplantation and cellular therapy or TCT meeting as we've continued to add to the body of evidence demonstrating the potential of <unk> to address unmet needs in patients undergoing allogeneic stem cell transplantation.

Patients often have serious infections after transplant due to the slow recovery of their immune system following treatment.

In a presentation that received a TCT Best Abstract Award, Dr. Paul Zablosh presented data from our Phase III randomized trial of Omodubicel showing rapid recovery of a broad repertoire of immune cells, which was associated with reduced infection rates in Omodubicel-treated patients.

In our presentation that received a TCT best Abstract award Dr. <unk> presented data from our phase III randomized trials I'm going do the self showing rapid recovery of a broad repertoire of immune cells, which was associated with reduced infection rates would be with cell treated patients.

In another oral presentation at TCT, Dr. Mitch Horwitz presented the final results of our Phase 3 study, including patient follow-up of over one year after transplant.

And another oral presentation at TCT, Dr. Mitch Horowitz presented the final results of our phase III study, including patient follow up of over one year after transplant.

The results show that the early in <unk> and lower infection rates previously observed in patients treated with <unk> were accompanied by longer term clinical benefits, including a reduction in transplant related mortality and a trend towards an increase in overall survival with no increase in relapsed or graft versus host disease compared to transplantation with standard.

The results showed that the early engraftment and lower infection rates previously observed in patients treated with Omidube cell were accompanied by longer-term clinical benefits, including a reduction in transplant-related mortality and a trend towards an increase in overall survival with no increase in relapsed or graft versus host disease compared to transplantation with standard umbilical cord blood.

Medical cord blood.

In addition, among our six posters at TCP, we presented 10-year follow-up data of patients treated in the Omiduva Cell Clinical Development Program demonstrating long-term, sustainable hematopoiesis and immune competence, with one case of secondary graft failure and one case of severe chronic GVHD among the 22 patients in the cohort.

With one case of secondary graft failure in one case of severe chronic gvhd among the 22 patients in the cohort.

We also presented an analysis of resource utilization in the phase three study, demonstrating that faster hematopoietic recovery and lower rates of infections in patients transplanted with Omidupa cell were associated with significantly shorter length of hospital stay, reduced admissions to intensive care unit settings, and lower healthcare resource utilization compared to control.

We also presented an analysis of resource utilization and the phase III study.

Demonstrating that faster hematopoietic recovery and lower rates of infections in patients transplanted with <unk> cel, where associated with significantly shorter length of hospital stay reduced admissions to intensive care unit, setting and lower healthcare resource utilization compared to control.

Another poster outlined a quantitative analysis of well established measures of patient health related quality of life in a randomized phase III study.

Another poster outlined a quantitative analysis of well-established measures of patient health-related quality of life in a randomized phase 3 study.

The analysis demonstrated that omodubicil was associated with meaningfully greater preservation or improvement of quality of life, tying together clinical data in the phase three study to important outcomes from a patient-focused perspective.

The analysis demonstrated that <unk> was associated with meaningfully greater preservation or improvement of quality of life.

Hang together clinical data in the phase III study two important outcomes from a patient focused perspective.

Overall, we've continued to develop a robust set of clinical, scientific, translational, and quality-of-life data demonstrating the potential clinical benefit of transplantation with Omidubis.

Overall, we've continued to develop a robust set of clinical scientific translational and quality of life data demonstrating the potential clinical benefit of transplantation with only <unk>.

Now turning to our NK pipeline, I'll start with an update on GDA-201.

Now turning to our NK pipeline I'll start with an update on GDA 201.

We're proceeding with our plans to initiate a study of GDH01 in patients with non-Hodgkin lymphoma.

We are proceeding with our plans to initiate a study of GDA 201 in patients with non Hodgkin lymphoma.

Although there have been recent advances for patients with lymphoma, we recognize that there is still an unmet need for patients with relapse and refractory disease.

Although there have been recent advances for patients with lymphoma, we recognize that there is still an unmet need for patients with relapsed and refractory disease.

Given the responses observed in the investigator-led study at the University of Minnesota using the FRESH formulation of GDA-201, we've developed the acquired-preserved formulation, which allows us to perform a multi-center study.

Given the responses observed in the investigator led study at the University of Minnesota, using the fresh formulation of GDA 201, we've developed the Cryopreserved formulation, which allows us to perform a multicenter study.

We're very pleased to receive FDA clearance of our IND for GDA 201, removing the clinical hold and allowing us to move forward with the study.

We're very pleased to receive FDA clearance of our IMD for GVA-201, removing the clinical hold and allowing us to move forward with the study.

We are proceeding with the operational activities at several sites, and we anticipate conducting formal site initiation visits and opening sites for enrollment this quarter.

We are proceeding with the operational activities at several sites and we anticipate conducting formal site initiation visits and opening sites for enrollment this quarter.

Turning to our research and development activity, we're continuing to advance the preclinical data for our gene editing NK cell pipeline aimed at hematologic malignancies and solid tumors.

Turning to our research and development activities, we're continuing to advance the preclinical data for our gene-edited NK cell pipeline aimed at hematologic malignancies and solid tumors.

We recently presented two posters at the International Society for Cell and Gene Therapy, or ISGP, meeting in San Francisco.

We recently presented two posters at the International Society for cell and gene therapy, or <unk> meeting in San Francisco.

One poster presented new preclinical data on <unk> 301, our fish knockout membrane bound IL 15, expressing NK cell.

One poster presented new preclinical data on GDH301, our CISH knockout membrane-bound IL-15-expressing NK cell.

Our poster showed cytotoxicity and potency of GDA301 in multiple tumor cell lines.

Our poster showed cytotoxicity and potency of GDA 201 in multiple tumor cell line.

The second poster detailed preclinical data demonstrating cytotoxicity of <unk> 601, our CD 38 knockout anti CD 38 car expressing nam NK cells against multiple myeloma cell line.

The second poster detailed preclinical data demonstrating cytotoxicity of GDA601, our CD38 knockout anti-CD38 CAR expressing NAMNK cells against multiple myeloma cell lines.

We look forward to presenting more data at scientific meetings this year.

We plan to continue to conduct preclinical proof of concept studies for these genetically modified NK therapeutic candidate and by the end of 2022, we plan to select a pipeline candidate for IND, enabling studies.

We plan to continue to conduct preclinical proof of concept studies for these genetically modified NK therapeutic candidates. And by the end of 2022, we plan to select a pipeline candidate for IND-enabling studies.

With that I'll turn the call over to Michele to now talk more about <unk> and commercial preparation Michelle.

With that, I'll turn the call over to Michelle to now talk more about Omidubicel and commercial preparation. Michelle?

Thank you, Reni. I will now comment on our advancements with our CMC module and our launch readiness from Adobe itself.

Thank you Randy and I will now comment on our advancements with our CMC module and our launch readiness for <unk>.

Earlier this year, we initiated our rolling BLA submission for <unk> and we are nearing completion of the rolling BLA, which is on track for completion in the second quarter of this year.

Earlier this year, we initiated our rolling BLA submission for Omiduba cell, and we are nearing completion of the rolling BLA, which is on track for completion in the second quarter of this year.

In the first quarter of this year, we reached an important milestone with the FDA's acknowledgement of the analytical comparability from our planned gametocel-owned commercial facility in Israel as compared to the manufacturing facility used for the Phase III study.

In the first quarter of this year, we reached an important milestone with the Fda's acknowledgment of the analytical comparability from our plans can be to sell owned commercial facility in Israel as compare to the manufacturing facility used for the phase III study.

This allowed us to move forward with completion of the remaining production runs in our facility in Israel to support the BLA.

Our cross-functional team in Israel, including operations, quality, R&D, and regulatory are progressing well on the CMC module in preparation for completing that module and the BLA submission in the second quarter.

Our cross functional team in Israel, including operations quality, R&D and regulatory are progressing well on the CMC module in preparation for completing that module and the BLA submission in the second quarter.

We are not only preparing for the BLA submission from an operations perspective, but also preparing for launch readiness. Our facility in Israel is modular, and upon FDA approval of AmadubaCell, we will have the ability to add additional cores as demand grows for AmadubaCell. We are confident we could support the launch demand requirements from our facility, not only from a production, but also a supply chain standpoint.

We're not only preparing for the BLA submission from an operations perspective, but also preparing for launch readiness our facility in Israel is modular and upon FDA approval of <unk>. So we will have the ability to add additional cores as demand grows from <unk>. We are confident we could support the launch demand.

<unk> from our facility not only from a production, but also a supply chain standpoint.

The team is diligently working to ensure our manufacturing reliability, chain of identity and chain of custody for our commercial process to ensure a positive transplant center and positive patient experience.

The team is diligently working to ensure our manufacturing reliability chain of identity and chain of custody for our commercial process to ensure a positive transplant center and positive patient experience.

Transitioning to launch readiness the gamete of team recognizes the unmet need that <unk> may address for patients. Upon FDA approval. We are focused on our launch readiness to ensure upon FDA approval that patients could have access to <unk>.

Transitioning to launch readiness, the Gameta team recognizes the unmet need that OmiduvaCell may address for patients upon FDA approval. We are focused on our launch readiness to ensure upon FDA approval that patients could have access to OmiduvaCell.

For patients with hematologic malignancies that are deemed eligible for an allogeneic stem cell transplant. The procedure may be their best chance for a potential cure.

For patients with hematologic malignancies that are deemed eligible for an allogeneic stem cell transplant, the procedure may be their best chance for a potential cure.

There are two key opportunities that OmiduvaCell may address for patients upon FDA approval. The first being potentially improving outcomes as compared to other donor sources based on transplanter feedback, and the second, potentially increasing accuracy.

There are two key opportunities and I'm, a <unk> may address for patients upon FDA approval, the first being potentially improving outcomes as compared to other donor sources based on transplant or feedback and the second potentially increasing access.

In the United States. There are approximately 8000 patients above the age of 12 with hematologic malignancies, who undergo an allogeneic stem cell transplant each year.

In the United States, there are approximately 8,000 patients above the age of 12 with hematologic malignancies who undergo an allogeneic stem cell transplant each year.

<unk> potentially improving outcomes, we have conducted extensive market research over the last two years and the insights are consistent and clear.

For potentially improving outcomes, we have conducted extensive market research over the last two years, and the insights are consistent and clear. Transplanters see important opportunities for Amadoubicel to potentially improve outcomes based on their experience with other donor sources. This is due to the strength of our clinical data, the ability to provide patients with predefined number of cells, and the ability to provide Amadoubicel within approximately one month of patient identification.

Transplant or see important opportunities for <unk> to potentially improve outcomes based on their experience with other donor sources. This is due to the strength of our clinical data the ability to provide patients with pre defined number of cells and the ability to provide <unk> within approximately one month of patient identification.

The majority of the patients in the United States are still receiving their graft from an unrelated donor, and that can take on average at least two to three months to align the patient and the donor. This timing for unrelated donors unfortunately puts the patient at risk for relapse.

The majority of the patients in the United States are still receiving their graph from an unrelated donor and that can take on average at least two to three months to align the patient and the donor this timing for unrelated donors. Unfortunately puts the patient at risk for relapse.

Unfortunately, there are approximately 1200 patients each year, who are ages 12, and up with hematologic malignancies, who are deemed eligible for an allogeneic stem cell transplant, but cannot find an appropriate donor.

Unfortunately, there are approximately 1,200 patients each year who are ages 12 and up with hematologic malignancies who are deemed eligible for an allogeneic stem cell transplant but cannot find an appropriate donor.

In terms of potentially increasing access for patients, unfortunately there is racial disparity in the U.S. in regards to access to allogeneic stem cell transplant.

In terms of potentially increasing access for patients. Unfortunately, there is racial disparity in the U S. In regards to access to allogeneic stem cell transplants.

If you are non-Caucasian and do not have access to a family member donor, you have a very low likelihood of finding a match in the public data.

If you are non Caucasian and do not have access to a family member donor you have a very low likelihood of finding a match in the public database.

For example patients were African American may have less than a 20% chance of finding a match.

<unk> has a less stringent matching criteria and we demonstrated our ability to match racially and ethnically diverse patients in our phase III study approximately 40% of the patients in our study were non Caucasian.

In a study recently highlighted in a poster presentation at TCT, we leverage available registry data and population modeling to project the potential impact of <unk> on racial and ethnic disparities in the model population increases in <unk> and eligible patients were associated with.

Higher proportions of patients undergoing transplant and overall potentially improved outcomes with improvements being greater among racial minorities.

Taken together, we anticipate that these two opportunities combined pending FDA approval, both improving outcomes based on transplant or feedback and increasing access may result in <unk>, capturing approximately 20% to 25% of the addressable market once we reach peak market share.

Upon FDA approval this would equate to approximately 2000 to 2500 patients treated each year in the U S alone with them and do the film.

In regards to reaching transplant centers in the U S. The transplant centers that perform allogeneic stem cell transplants are extremely concentrated.

For reference in the U S. There are approximately 200 transplant centers that perform allogeneic stem cell transplants 70 of those centers conduct approximately 80% of the transplants, allowing for an optimized approach to commercialization by initially targeting those centers.

Another important aspect of our launch is engaging with payers to ensure that they understand the potential value of <unk> cel upon FDA approval.

[noise] payer conversations are underway with national and key regional payers and we continue to hear consistent encouraging feedback on the overall value proposition of <unk>, including the strength of the clinical data and health economic data, we have published to date.

We are also encouraged by their feedback on both the coverage and reimbursement approach they anticipate taking with them a deeper sell upon FDA approval.

We are excited to continue to hear positive feedback across all stakeholders and are extremely encouraged and driven by the potential of all the data so.

We look forward to continuing to provide updates on our commercial preparations.

I'll now turn the call over to Shai to review our financial results shy.

Thank you Michelle and good morning, everyone today, I will summarize our financial results for the first quarter of 2022.

As of March 31, 2022.

Cash position was approximately $70 million compared to $96 million.

December 31 2021.

Research and development expenses for the quarter grew $11 3 million compared to $11 4 million in the first quarter of last year.

Increase was primarily due to a $1 1 billion decrease in army do Brasil and GDA 201 clinical studies activities.

Also by an increase of $1 million and boarding our scientific capabilities and talent.

Commercial expenses for the quarter were $3 9 million compared to $4 $2 million in the first quarter of 2021. The decrease was mainly due to reducing our near term commercial readiness expenses as we are assessing alternatives for the commercialization of <unk>, including potential U S or global partnership.

General and administrative expenses were $4 $1 million in the first quarter of 'twenty, two compared to $3 $5 million for the same period in 'twenty. One the increase was mainly due to a zero point $5 million increase in headcount and related expenses.

Finance expenses net zero point $9 million for the first quarter of 2022 compared to zero point $1 million for the same period last year. The increase was primarily due to a zero point $6 million increase in interest expenses from our convertible notes.

Net loss for the first quarter of 2022 was $22 million compared to a net loss of $19 $2 million in the first quarter of 2021.

We expect cash used for ongoing operating activities for the entirety of this year to range from $65 million to $70 million, we anticipate the recurring total cash position will support our ongoing operating activities into mid 2023.

This cash runway guidance is based on our current operational plans and excludes any additional funding that may be received or business development activities that may be undertaken with that I will turn the call back over to Michelle.

Thank you Shai.

We look ahead to the rest of the year, we are uniquely poised to deliver on our mission of developing potentially curative cell therapies for patients with blood cancers and other serious diseases.

We look forward to a full BLA submission from <unk> expected in the second quarter of this year and the initiation of our phase one two multi center commuter sell sponsored study for the Cryopreserved formulation of GDA 201 in patients with Follicular and diffuse large b cell lymphoma for this year.

We are excited for the opportunity to continue to leverage our unique Nam enabled platform across a broad range of cell therapy candidates and we look forward to providing updates throughout the year.

Now we will open the call for questions Carmen.

Thank you and to ask a question simply press star one on your telephone to withdraw your question press the pound or hash key.

Your first question comes from Jon Miller with Evercore. Please go ahead.

Hi, This is erik calling in for Jonathan.

Two quick questions on GDA 201.

With trial sites opening in second quarter when can we expect the first patient in.

And what sort of dynamics do you see around enrollment do you expect COVID-19 or summer travel to impact that.

I have one follow up.

Excellent. Thank you Erik Thanks for joining the call I will turn to Rooney to address both of those thank you.

Thanks, Eric.

So in terms of opening sites or sites will open in this quarter and patients will need to be recruited and screened appropriately before the first patient is treated though it usually takes several weeks for that to happen until.

Until the patient is actually treated with GDA 201.

But there's a lot of interest among the investigators for the study there is an unmet need these are patients who are relapsed or refractory refractory and clearly need additional therapies and are engaged in enrolling in a clinical trial.

So with the with the high need for these patients and the fact that they are.

Ill with very serious cancer.

We believe that enrollment.

Will be robust for these patients I'll make one more point about this portion of the study to the phase one portion of the study and so.

This is a phase one where toxicities are being evaluated patient by patient we will be evaluating toxicity in each patient before proceeding with enrollment to the next patient. So if we can.

We can carefully gauge patient enrollment as we move forward in the next few months and evaluate dose limiting toxicity.

We evaluate that then we can enroll patients more concurrently.

Got it.

And just one more question.

Do you expect any differences in enrollment timeline between the Follicular.

Bcl cohorts.

Yeah.

At this point, we don't anticipate a difference between those two cohorts, but that's something that we can gauge is as we move along.

We certainly have the ability to analyze them separately if need be.

There is a discrepancy we can manage that within the confines of the clinical trial design.

But at this point, we don't anticipate that there'll be a difference because our investigators have expressed that patients with both histology are in need of new therapies.

Great. Thank you.

Thank you Eric.

Your next question comes from Tim Hoff with Piper Sandler. Please go ahead.

Great. Thank you.

Morning, everyone.

Curious what is sort of going on or what's the latest with respect to commercial prep for <unk>. I know you are considering potential strategic alternatives, but also really just probably get a sense for.

Finishing up the BLA.

Might be the most likely.

The scenario for marketing.

Excellent good morning, Todd and thank you for joining us I'll I'll go ahead and take that question. So in regards to commercial preparation.

We've accomplished some critical milestones to date. So first off was really honing the commercial insights to understand first off the overall opportunity, but second of all what could on the <unk> potential be for that opportunity. So we.

We've over the last couple of years and most recently, we've reiterated or.

We looked at some of these market insights.

We recognize the two key opportunities from <unk>, one is the ability to improve outcomes as compared to other donor sources and this is based on transplant or feedback and then the second is increasing access in terms of improving outcomes. Some of the key insights that.

Transplant or share with us is that the strength of the clinical data from our phase III study, both the efficacy and the safety that ROE need has presented also the ability of having pre defined predefined number of cells and then finally, the turnaround time in the United States. The majority of patients currently are still getting their donor source from in <unk>.

Unrelated donor and on average we are hearing that takes at least two to three months to align the donor and the patient and as you know with acute leukemias and aggressive lymphomas that puts the patient at risk for relapse. So with all the <unk> being approximately one month from time of patient identification in the phase III study to return on the <unk>. So that is a positive fee.

And then switching to the increasing access are improving access you know unfortunately in the United States. We do see many patients who are deemed eligible for transplant that cannot find the donor the latest data that we've assessed as approximately 1200 patients each year and that may end up growing.

Unfortunately, it's also a situation of racial disparity if you were non Caucasian in the U S and don't have access to a family member to incredibly difficult to find a match.

Summarize based on the encouragement of those market insights in regards to the opportunity from a <unk>. We have hired our commercial leadership team and we also have the operations and supply chain leadership team in place. So this way that the core group of leaders could continue to assess not only the opportunity, but most importantly.

The launch readiness and Thats moving in parallel to our assessment of potential strategic alternatives, we feel very very strongly that upon FDA approval, we do need to make sure patients have access to <unk>. So we've got our leadership team in place as we're also in parallel looking at potential strategic alternatives.

And would those alternatives include both the United States and overseas because it feels to me like a market that is appropriately sized for a biotech company to launch, especially since you guys have so much familiarity with the product with the treatment.

Treatment group, so I just wanted to give a little more color on that thanks.

Thank you Ted So we are assessing strategic opportunities.

Globally, including the U S. But also ex U S. We have conducted initial commercial assessments in Europe and in Asia, specifically in Japan and are very encouraged by the commercial opportunity from a <unk> in those areas. So.

We are as we discussed strategic alternatives for potential launch of <unk> looking globally.

Great. Thank you very much.

Thank you Tony.

Your next question comes from Joe <unk> with Needham <unk> Company. Please go ahead.

Good morning, everyone and thanks for taking my question.

Maybe a broader one considering recent result and donated.

NK cells so.

In the space.

I'm just curious how encouraged you are.

Seeing that a company sponsored study could lead to.

Some very interesting early results.

How do you think that translates for GDA 201. Thank you.

Excellent good morning, Jill and thank you for joining us for the question, let me turn to Rooney to address skills question.

Sure. Thanks Gil.

There have been some encouraging results that we've seen from other products.

NK cell type products recently and over all of those are actually really encouraging because it sets the stage for NK cells.

And in particular some of the results we've seen are related to.

Hi, doses delivered of NK cells with results seen with greater responses seen at the higher doses and that actually sets us up quite well because we're able to deliver with GDA 201 high doses of NK cells high numbers of NK cells that are extremely functional and retain their killing capacity.

Because the Nam.

Technology that we've used to expand and maintain the steadiness of stem cells actually expand and maintain the cadence or the killing potential of NK cells. So we.

We actually think it sets us up quite nicely for our.

Phase one two study.

Our study is.

Designed to evaluate the safety of the Cryopreserved formulation at first and then move right into a formal efficacy evaluation. The entire study is unblinded.

The open label study and we will be able to see as we go.

What's the potential for activity is with with this formulation. So we're excited to start enrolling patients have seen results.

Thank you. Thank you that was very helpful. Maybe also on the quick financial question.

So on the <unk>.

G&A spend there was a line that I mentioned.

There are some cost savings due to consideration of strategic options for commercialization.

Me understand does that mean that the company is cutting back on commercial preparedness launch or.

Maybe just help me understand that thank you.

Thank you Gail so I'll start with sort of the strategic aspect of all turn to Shanghai for any additions on the financial side. So.

We were fortunate last year to hire some incredibly strong commercial leaders Linda stamler to lead marketing and account management and you'll see them on ghani to lead market access.

Those two individuals and their teams were able to conduct a lot of the critical market insights and beginning of launch preparation last year. So then that allowed us to use this past quarter more to evaluate the insights and less opex associated with having to gather additional insights and also.

With hiring we were able to hire our core group of commercial and operations leaders last year. So that's sort of the.

High level strategic aspects of it let me turn to shine, if theres anything to add from the financial side.

Good morning, Thank you for the question.

The way we see it.

The beta cell as a company continue to diligently manage our cash position.

As such we announced back in January this year.

Assessing some strategic alternative for lunch you want me to be so as our vision is to bring duvelisib to patients.

Yes, we did prioritize key activities not only on the commercial side, but across all the business to make sure. We are not jeopardizing, our launch activities and as I mentioned and Joe mentioned before making sure each patients will have access to Amit to be slowing there are plenty of the approval.

Alright. Thank you both for taking our questions. This morning.

Thank you Bill.

Your next question comes from Jason Butler with JMP Securities. Please go ahead.

Hi, it's Roy in for Jason Thanks for taking my questions I wanted to follow up on the partnering.

Question for I guess can you give a little more detail maybe on the level of interest that youre seeing.

Do you think you could have a partnership in <unk>.

Place before.

A potential approval maybe around around year end and can you remind us what structure.

You prefer the U S may be a co promote thanks.

Thank you good morning, and thank you for joining US I'll go ahead and take both of the questions. So we won't comment on specifics in regards to our assessment at this point in time, but I do want to reiterate something very important that I had said earlier and thats. The fact that although we are assessing strategic alternatives.

As for launch we also do have the leadership team in place on both the commercial and medical affairs, the quality and the operations side to ensure that patients have access to <unk>. Upon FDA approval. So when you asked about sort of the latter part of the question in terms of timing.

We do have the strategy in place the plans in place to assure access to patients for them a <unk> upon FDA approval.

At this point in time as we indicated earlier this year, we are assessing strategic alternatives, but also continuing to do our work internally around assessing the opportunity in assessing what would be meter for launch planning.

Okay, Great. That's helpful. Thank you.

For the <unk>.

<unk> candidates beyond GDA 201 are you going to take only one into the IND, enabling studies or potentially multiple candidates and then what happens to the other candidates.

That don't go into IND, enabling studies. This year did they go on hold or you're going to continue to refine them pre.

Pre clinically.

Excellent. Thank you very much I'll turn to ROE need for <unk> to address that question.

Thanks, and thanks, Jason.

One at a time, so we will take the lead candidate at the by the end of this year and do the appropriate IND, enabling studies and continue to develop.

On data of course.

<unk>.

Additional candidates and.

As we move those forward as we are April .

Okay. Thank you very much.

Thank you.

Your next question comes from Mark <unk> with open Hymer. Please go ahead.

Hi, This is Jack on for Mark and thanks for taking our questions.

So can you please remind us what those levels will be tested in the dose escalation from the GDA 201, and if there are any differences in deflation.

Deflation condition, our two telecom support or we've talked to you Matt.

Dosing.

Thank you for joining us Jacqueline I'll turn to our need to address both the dosing question and the question around linked with depletion and Rituximab in the protocol.

Absolutely.

Sign of the study the design of the administration of the treatment.

<unk> is based on the.

Minnesota study using a fresh formulation and so we will we will be testing doses that are similar to the ones used in the Minnesota study.

And the administration of <unk>.

Lymphoid depleting chemotherapy Rituximab and <unk> are also very similar if not identical to what was done in the Minnesota study. So I don't think we've come out yet with the exact dose levels, but they are guided completely by the previous data in Minnesota.

When do you expect to commence manufacturing runs for GBS you all was for the phase one trial.

Yes, actually I'll ask Randy to if you could also take that of course I'm happy to take that so manufacturing we are building because in the phase one trial.

The <unk>.

<unk> 201.

Is produced head of time and not matched for patients that basically.

Italy available.

We are building our inventory already and expect to have them.

The product available for patients as soon as the first dosing is required.

Uh huh.

Great. Thanks for taking our questions.

Thank you. Thank you.

Your last question comes from Matthew Cross with Alliance Global Partners. Please go ahead.

Hi, all good morning, best wishes to Julian for a speedy recovery.

I had two quick questions related to the phase one phase two for <unk> hundred one.

And kind of following up on your comments there about the <unk>.

Scheduling for Olympic depleting chemotherapy antibody use.

Guess, considering internal study will be using the cryopreserved formulation not the fresh.

Just wanted to understand.

<unk>.

They are kind of special considerations here I guess for the study compared to the prior one.

It sounded like the schedule will be more or less the same or based off the prior study, but given that youre not baking in time for.

NIM expansion with the cryo preserved just wanted to understand.

Whether that's scheduling would still look similar and maybe to kind of recap any modifications that would have to be made to the study.

We're needing to remedy the fda's concerns around donor eligibility requirements and anything that may play into which sites are able to participate in this study given the combination of those.

What's your ability requirement changes and the cryopreservation.

<unk> versus some previously thanks.

Excellent. Thank you Matt. Thank you for joining us and thank you for the well wishes. We will we will pass them along thank you and I'll turn to Rami to answer those questions in regards to the phase one two.

Sure I'm happy to answer that and I'll take the donor eligibility piece first.

So the donor eligibility procedures in that assay qualification.

Just to make sure that those are compliant with FDA requirements and so we replaced our donor testing laboratory.

With the CLIA certified lab.

In the U S and we've now made sure that all appropriate tests, our license and cleared.

And that eligibility requirement has been met.

So there has been no change in the design of the study.

<unk>.

The hold itself was not did not require any changes in the design of the study and the protocol the sites or any or any anything that has to do with the clinical trial itself. It really was confined to the eligibility of the products. So that was actually.

Now in terms of Cryopreserved formulation so that.

It's great that you picked up on that so in the fresh formulation study patients had to have a donor available that the owner has to be recruited and had to be done and then the product has to be produced over time, so that that because we have a readily available product.

Now it will be at the site when the patient is enrolled and so that may save some time in terms of getting the patients up and traded because they will have product there. They don't have to wait for it then donor to get already get the donation.

And and do all that and then procedurally there will be a filing process.

The product will be there will be frozen and it'll be flat at the bedside to to provide the.

A patient with the product when they need it.

Perfect. Okay Super helpful. And then just had one quick follow up which was around the sizing of it looks like.

Or what you've drawn up on Clinicaltrials Gov that there is kind of a target enrollment of about 100 patients.

So I guess I know you are not commenting at this point on the actual dose levels that you may step through but just wanted to confirm whether that total sizing was based around kind of an expectation internally around the number of phase one cohort that you may look at if it will be ultimately still driven by the standard kind of safety evaluations as you step through doses or maybe just kind.

Or an expectation for the phase II population for that portion of the study I just wanted to get a little bit of an understanding around the assumptions for that so that sizing.

Absolutely so that number which was put into a clinical trial dot Gov, which is incorporated into the protocol itself.

There is some flexibility there because you don't know for sure how many patients you're going to end up enrolling in phase. One is a standard three by three design, where if there is a dose limiting toxicity observed in the <unk> numbers are expanded to include more patients and so this is the maximum size based on the possible expansion of.

Cohorts in the phase one portion and then it also includes the phase II portion, where we are going to be evaluating separately the patients with follicular lymphoma from the patients with the aggressive lymphomas.

And each of those.

The phase II portion has its own staff and analysis.

Surrounding it but.

Relatively small cohorts to allow us to evaluate rather quickly.

Efficacy results and then make decisions based on those early efficacy results that we see.

Understood. Okay. Thanks, again for the clarity I appreciate it.

Thanks, so much Matthew.

Thank you and this concludes our Q&A session I will turn the call back to Michelle coffee for her final remarks.

Thank you Carmen. Thank you very much for joining us for the call today, and we look forward to keeping you all updated on our future milestones have a nice day. Thank.

Thank you Carmen that'll conclude our call.

Thank you, ladies and gentlemen for participating and you may now disconnect.

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Ladies and gentlemen, thank you for standing by welcome to again meet our sales conference call for the first quarter 2022 financial results. My name is Carmen and I'll be your operator for todays call. Please be advised that this call is being recorded at <unk> request now I will.

Like to introduce your host for today's conference Heather <unk> <unk> Chief of staff. Please go ahead.

Thank you Carmen 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 2022.

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 Dot <unk> Dot com.

Please note that unfortunately, our chief Executive Officer, Julian Adams is unable to join US. This morning due to contracting COVID-19.

<unk> operations are unaffected and Julian has advised us that his symptoms are improving and he expects to recover shortly.

With that here with me on our call today are Michelle Bachman, our Chief operating officer, and Chief Commercial Officer, Tony <unk>, Our Chief Medical Officer, and Chief Scientific Officer, and Sean <unk>, Our Chief Financial Officer.

During this call we may make forward looking statements about our future expectations and plans, including in respect of the timing of initiation and progress and data reported from the clinical trials of our product candidates anticipated regulatory filings, including the submission of the BLA for <unk> to the FDA commercialization <unk>.

Planning efforts, the potential life, saving or curative therapeutic and commercial potential of <unk> product candidates, including GDA 201, and Amadou Lasalle.

Expectations regarding our projected cash to be used for operating activities and cash runway.

Assess of developing and commercializing product candidates that are safe and effective for use as human therapeutics and in the endeavor of building a business around such product candidates as well as those considerations described in the risk factors section of our most recent annual report on Form 10-K, and other filings that we make with the SEC.

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.

Now I'd like to turn the call over to Michele.

Thank you Heather and thank you to everyone for joining us. This morning commute so had a productive quarter as we made important progress across all areas of our company. We continue to build momentum as we head into several inflection points expected. This year, most near term being the full BLA submission for <unk>, which.

We are on track to complete in the second quarter of this year. Additionally.

Additionally, our GDA 201 program advanced marked by the recent clearance by FDA of our IND and removal of the clinical hold for the cryo preserved formulation.

Beyond GDA 201, which is our lead program in our expanding Nam enabled NK cell pipeline. We are also looking forward to announcing a new product candidate from our genetically engineered ma'am enabled NK cell constructs later this year.

We recently announced preclinical data at the IFC T Conference supporting the development of <unk> 301, and <unk> 601.

On later on in the call.

With all we have accomplished only this past quarter, but also in the last few months, we are poised to be a leader in the development of Nam enabled cell therapies with two promising an important clinical programs and a robust pipeline of genetically modified NK cell product candidates in preclinical development.

The progress we achieved this quarter continues to add to the important foundation that we have established a strong.

Foundation is due to our dedicated employees and their continued focused on patients and our vision of advancing therapies to help address unmet needs.

With this I will now turn the call over to Rohit.

Thanks, Michelle and good morning, everyone. Thank you for joining us on the call. This morning.

Let me start with our lead program <unk>, which has breakthrough therapy designation as well as orphan drug status and the potential to be the first FDA approved cell therapy for stem cell transplant.

Earlier this year, we were excited to initiate the rolling BLA submission for <unk>, starting with our non clinical module followed by the submission of our clinical module.

I'm pleased that we are currently on track to complete the full BLA submission in the second quarter of this year moving us closer to bringing this important potential therapy to patients.

<unk> is supported by strong phase III results, which met all primary and secondary endpoints.

This past quarter, we announced the presentation of new data at the transplantation and cellular therapy or TCT meeting as we've continued to add to the body of evidence demonstrating the potential <unk> to address unmet needs in patients undergoing allogeneic stem cell transplantation.

Patients Boston have serious infections after transplant due to the slow recovery of their immune system following treatment.

In our presentation that received a TCT best Abstract award Dr. <unk> presented data from our phase III randomized trials I'm going through the self showing rapid recovery of a broad repertoire of immune cells, which was associated with reduced infection rates and I will do the cell treated patients.

And another oral presentation at TCT, Dr. Mitch Hurwitz presented the final results of our phase III study, including patient follow up of over one year after transplant.

The results showed that the early in <unk> and lower infection rates previously observed in patients treated with <unk> were accompanied by longer term clinical benefits, including a reduction in transplant related mortality and a trend towards an increase in overall survival with no increase in relapsed or graft versus host disease compared to transplantation with standard umbilical.

Cord blood.

In addition, among our six posters at TCT, we presented 10 year follow up data of patients treated in the <unk> clinical development program, demonstrating long term sustainable natter polices and immune confidence with one case of secondary graft failure in one case of severe chronic gvhd among the 22 patients in the cohort.

We also presented an analysis of resource utilization in the phase III study, demonstrating that faster hematopoietic recovery and lower rates of infections in patients transplanted with <unk> were associated with significantly shorter length of hospital stay reduced admissions to intensive care unit setting and lower healthcare resource.

Utilization compared to control.

Another poster outlined a quantitative analysis of well established measures of patient health related quality of life in a randomized phase III study.

The analysis demonstrated that <unk> was associated with meaningfully greater preservation or improvement of quality of life.

Tying together clinical data in the phase III study two important outcomes from a patient focused perspective.

Overall, we continue to develop a robust set of clinical scientific translational and quality of life data demonstrating the potential clinical benefit of transplantation with Ahmedou Lasalle.

Now turning to our NK pipeline ill start with an update on GDA 201.

We are proceeding with our plans to initiate a study of GDA 201 in patients with non Hodgkin lymphoma.

Although there have been recent advances for patients with lymphoma, we recognize that there is still an unmet need for patients with relapsed and refractory disease.

We're very pleased to receive FDA clearance of our IND for GDA 201, removing the clinical hold and allowing us to move forward with the study.

We are proceeding with the operational activities at several sites and we anticipate conducting formal site initiation visits and opening sites for enrollment this quarter.

Turning to our research and development activity, we are continuing to advance the preclinical data for our gene edited NK cell pipeline aimed at hematologic malignancies and solid tumors.

We recently presented two posters at the International Society for cell and gene therapy, or <unk> meeting in San Francisco.

One poster presented new preclinical data on <unk> 301, our fish knockout membrane bound IL 15, expressing NK cell.

Our poster showed cytotoxicity and potency of GDA 201 in multiple tumor cell lines.

The second poster detailed preclinical data demonstrating cytotoxicity of <unk> 601, our CD 38 knockout antisense 38 car expressing nam NK cells against multiple myeloma cell line.

We look forward to presenting more data at scientific meetings this year.

With that I'll turn the call over to Michelle to now talk more about when we do the sell and commercial preparation Michelle.

Thank you Randy.

I will now comment on our advancements with our CMC module in our launch readiness for <unk>.

Earlier this year, we initiated our rolling BLA submission for <unk> and we are nearing completion of the rolling BLA, which is on track for completion in the second quarter of this year.

This allowed us to move forward with completion of the remaining production runs in our facility in Israel to support the BLA.

We are not only preparing for the BLA submission from an operations perspective, but also preparing for launch readiness our facility in Israel is modular and upon FDA approval of <unk>. So we will have the ability to add additional cores as demand grows from <unk>. We are confident we could support the launch demand.

<unk> from our facility not only from a production, but also a supply chain standpoint.

Transitioning to launch readiness the committed team recognizes the unmet need that <unk> may address for patients. Upon FDA approval. We are focused on our launch readiness to ensure upon FDA approval that patients can have access to <unk>.

For patients with hematologic malignancies that are deemed eligible for an allogeneic stem cell transplant. The procedure may be their best chance for a potential cure.

There are two key opportunities and on the <unk> may address for patients upon FDA approval, the first being potentially improving outcomes as compared to other donor sources based on transplant or feedback and the second potentially increasing access.

In the United States. There are approximately 8000 patients above the age of 12 with hematologic malignancies, who undergo an allogeneic stem cell transplant each year.

<unk> potentially improving outcomes, we have conducted extensive market research over the last two years and the insights are consistent and clear transplant or see important opportunities for <unk> to potentially improve outcomes based on their experience with other donor sources. This is due to the strength of our clinical data the ability to provide page.

<unk> with pre defined number of cells and the ability to provide <unk> within approximately one month of patient identification.

The majority of the patients in the United States are still receiving their graph from an unrelated donor and that can take on average at least two to three months to align the patient and the donor.

This timing for unrelated donors, Unfortunately puts the patient at risk for relapse.

In terms of potentially increasing access for patients. Unfortunately, there is racial disparity in the U S. In regards to access to allogeneic stem cell transplants.

If you are non Caucasian and do not have access to a family member donor you have a very low likelihood of finding a match in the public database.

For example, patients who are African American may have less than a 20% chance of finding a match.

<unk> has a less stringent matching criteria and we've demonstrated our ability to match racially and ethnically diverse patients in our phase III study approximately 40% of the patients in our study were non Caucasian.

Higher proportions of patients undergoing transplant and overall potentially improved outcomes with improvements being greater among racial minorities.

Taken together, we anticipate that these two opportunities combined pending FDA approval.

With improving outcomes based on transplant or feedback and increasing access may result in <unk>, capturing approximately 20% to 25% of the addressable market. Once we reach peak market share. So upon FDA approval. This would equate to approximately 2000 to 2500 patients treated each year in the U S alone with.

<unk>.

In regards to reaching transplant centers in the U S. The transplant centers that perform allogeneic stem cell transplants are extremely concentrated.

Another important aspect of our launch is engaging with payers to ensure that they understand the potential value of <unk> cel upon FDA approval.

We are also encouraged by their feedback on both the coverage and reimbursement approach they anticipate taking with them a deeper sell upon FDA approval.

We are excited to continue to hear positive feedback across all stakeholders and are extremely encouraged and driven by the potential of all the data so.

We look forward to continuing to provide updates on our commercial preparations.

I'll now turn the call over to Shai to review our financial results shy.

Okay.

Thank you Michelle and good morning, everyone today, I will summarize our financial results for the first quarter of 2022.

As of March 31, 2022, Alright, total cash position was approximately $70 million compared to $96 million as of December 31, 2021.

Research and development expenses for the quarter grew $11 3 million.

Compared to $11 $4 million in the first quarter of last year. The decrease was primarily due to a $1 1 billion decrease in army do Brasil and GDA 201, clinical studies activities offset by an increase of $1 million and broadening our scientific capabilities and talent.

Commercial expenses for the quarter were $3 9 million compared.

Compared to $4 2 million in the first quarter of 2021. The decrease was mainly due to reducing our near term commercial readiness expenses as we are assessing alternatives for the commercialization of <unk>, including potential U S or global partnership.

General and administrative expenses were $4 $1 million into first quarter of <unk> 22, compared to $3 $5 million for the same period in 2001. The increase was mainly due to a zero point $5 million increase in headcount and related expenses.

Net loss for the first quarter of 2022 was $22 million compared to a net loss of $19 $2 million since the first quarter of 2021.

Thank you Sean.

We look ahead to the rest of the year, we are uniquely poised to deliver on our mission of developing potentially curative cell therapies for patients with blood cancers and other serious diseases.

Now we will open the call for questions Carmen.

Thank you and to ask a question.

<unk> simply press Star one on your telephone to withdraw your question press, the pound or hash key.

Your first question comes from Jon Miller with Evercore. Please go ahead.

Hi, This is Erik calling in for Jonathan just had two quick questions on GDA 201 with.

With trial sites opening in second quarter. When can we expect the first patient in and what sort of dynamics around enrollment do you expect COVID-19 or summer travel to impact that.

Then I have one follow up.

Excellent. Thank you Erik Thanks for joining the call I will turn to Rooney to address both of those thank you.

Thanks, Eric.

In terms of opening sites or sites will open in this quarter and patients will need to be recruited and screened appropriately before the first patient is treated though it usually takes several weeks for that to happen.

Ah patient is actually treated with GDA 201.

Ill with very serious cancer.

We believe that enrollment will be robust for these patients I'll make one more point about this portion of the study to the phase one portion of the study and so.

As this is a phase one where toxicities are being evaluated patient by patient.

We'll be evaluating toxicity in each patient before proceeding with enrollment to the next patient. So if we can.

We can carefully gauge patient enrollment as we move forward in the next few months and evaluate dose limiting toxicity.

Once we evaluate that then we can enroll patients more concurrently.

Got it.

And just one more question.

Do you expect any differences in enrollment timeline between the Follicular <unk> cohort.

At this point, we don't anticipate a difference between those two cohorts, but that's something that we can gauges as we move along.

We certainly have the ability to analyze them separately if need be.

There is a discrepancy we can manage that within the confines of the clinical trial design.

But at this point, we don't anticipate that there'll be a difference because our investigators have expressed that patients with both histology are in need of new therapies.

Great. Thank you thank.

Thank you Eric.

Your next question comes from Tim Hoff with Piper Sandler. Please go ahead.

Great. Thank you and good morning, everyone.

Curious what is sort of going on or what's the latest with respect to commercial crop for <unk>. I know you are considering potential strategic alternatives, but also really just totally give us sense.

For sure.

Finishing up the BLA.

Might be the most likely.

The scenario for marketing.

Excellent good morning, Todd and thank you for joining US I'll go ahead and take that question. So in regards to commercial preparation.

We've over the last couple of years and most recently, we've reiterated or re looked at some of these market insights.

Transplant their share with us is that the strength of the clinical data from our phase III study, both the efficacy and the safety that <unk> has presented also the ability of having pre defined predefined number of cells and then finally, the turnaround time in the United States. The majority of patients currently are still getting their donor sourced from an <unk>.

Unrelated donor and on average we're hearing that takes at least two to three months to align the donor and the patient and as you know with acute leukemias and aggressive lymphomas that puts the patient at risk for relapse, So with <unk> being approximately one month from time of patient identification in the phase III study to return over on the <unk>. So that is a positive fee.

Unfortunately, it's also a situation of racial disparity if you are non Caucasian in the U S and don't have access to a family member incredibly difficult to find a match.

And with those alternatives include both the United States and overseas pre Christmas shelf totally like a market that is appropriately sized for a biotech company to launch, especially since you guys have so much familiarity with the product with the <unk>.

Treatment group, so I just wanted to get a little more color on that thanks.

Thank you Ted So we are assessing strategic opportunities.

Globally, including the U S. But also ex U S. We have conducted our initial commercial assessments in Europe and in Asia, specifically in Japan and are very encouraged by the commercial opportunity from the <unk> in those areas. So.

We are as we discussed strategic alternatives for potential launch of <unk> looking globally.

Great. Thank you very much.

Thank you Tony.

Your next question comes from Joe <unk> with Needham <unk> Company. Please go ahead.

Good morning, everyone and thanks for taking my question.

Maybe a broader one considering recent result and donated.

NK cells.

In the space.

I'm just curious how encouraged you are.

Seeing that a company sponsored study could lead to some very interesting early results.

How do you see that translates for GDA 201, Thank you.

Excellent good morning, Jill and thank you for joining us for the question, let me turn to Rooney to address skills question.

Sure. Thanks Gil.

There have been some encouraging results that we've seen from other products.

Uh huh.

NK cell type product recently and overall those are actually really encouraging because it sets the stage for NK cells.

And in particular some of the results we've seen are related to.

Hi, doses deliberative NK cells with the results seen with greater responses seen at the higher doses and that actually sets us up quite well because we're able to deliver with GDA 201 high doses of NK cells high numbers of NK cells that are extremely functional and retain their killing capacity.

The Nam.

Technology that we've used to expand and maintain the steadiness of stem cells actually expand and maintain the <unk> or the killing potential of NK cells. So.

We actually think it sets us up quite nicely for our.

Phase one two study.

Our study is.

Designed to evaluate the safety of the Cryopreserved formulation at first and then move right into a formal efficacy evaluation. The entire study is unblinded.

The open label study and we'll be able to see as we go what.

What the potential for activity is with with this formulation. So we're excited to start enrolling patients and seeing results.

Thank you. Thank you.

Very helpful. Maybe also on the quick financial question. So.

On the <unk>.

G&A spend.

I mentioned that.

There are some cost savings due to consideration of strategic options for commercialization.

Me understand does that mean that the company is cutting back on commercial preparedness launch or.

Just help me understand that thank you.

Thank you Gil so I'll start with sort of the strategic aspect I'll turn to Shai for any addition to almost financial side. So.

We were fortunate last year to hire some incredibly strong commercial leaders Linda stamler to lead marketing and account management Roseola ghani to lead market access.

With hiring we were able to hire our core group of commercial and operation leaders last year, So that's sort of the.

A high level strategic aspects of it let me turn to shine, if theres anything to add from the financial side.

Hi, Good morning, Thank you for the question.

The way we see it.

<unk> as a company continue to diligently manage our cash position.

As such we announced back in January this year.

Assessing some strategic alternative for lunch you want me to be so as our vision is to bring duvelisib to patients.

Yes, we did prioritize key activities not only on the commercial side, but across all the business to make sure. We are not jeopardizing, our launch activities and as I mentioned and Joe mentioned before making sure. Each patients will have access to me to be slowing there are plenty of the approval.

Alright. Thank you both for taking our questions. This morning.

Thank you Bill.

Your next question comes from Jason Butler with JMP Securities. Please go ahead.

Hi, it's Roy in for Jason Thanks for taking my questions I wanted to follow up on the partnering.

Questions I guess can you give a little more detail maybe on the level of interest that youre seeing.

Do you think you could have a partnership in place before.

A potential approval maybe around around year end and can you remind us what structure.

You prefer for the U S may be a co promote thanks.

Thank you good morning, and thank you for joining US I'll go ahead and take both of the questions. So.

Can't comment on specifics in regards to our assessment at this point in time, but I do want to reiterate something very important that I had said earlier and thats. The fact that although we are assessing strategic alternatives for launch. We also do have the leadership team in place on both the commercial medical Affairs.

The quality and the operation side to assure that patients have access to <unk>. Upon FDA approval. So when you asked about sort of the latter part of the question in terms of timing.

We do have the strategy in place the plans in place to assure access to patients for them a <unk> upon FDA approval.

Okay, Great. That's helpful. Thank you.

For the <unk>.

<unk> candidates beyond GDA 201 are you going to take only one into the IND, enabling studies or potentially multiple candidates and then what happens to the other candidates.

That don't go into IND, enabling studies. This year did they go on hold or you're going to continue to refine them pre.

Pre clinically.

Excellent. Thank you very much I'll turn to ROE need for <unk> to address that question.

Thanks, and thanks, Jason.

One at a time, so we will take the lead candidate at the by the end of this year and do the appropriate IND, enabling studies and continue to develop.

Based on data of course.

The additional candidates and.

As move those forward as we are April .

Okay. Thank you very much.

Thank you.

Your next question comes from Mark <unk> with open Hymer. Please go ahead.

Hi, This is Jack on for Mark and thanks for taking our questions.

So can you please remind us what those levels will be tested in the dose escalation from the GDA 201, and if there are any differences in deflation.

Deflation condition, our two telecom a core or we'll talk to you Matt.

Dosing.

Thank you for joining us Jacqueline I'll turn to our need to address both the dosing question and the question around them for depletion and Rituximab in the protocol.

Absolutely.

<unk> of the study over the design of the administration of the treatment.

<unk> is based on the.

Minnesota study using a fresh formulation and so we will we will be testing doses that are similar to the ones used in the Minnesota study.

And the administration of lymphoid depleting chemotherapy Rituximab and IL. Two are also very similar if not identical to what was done in the Minnesota study. So I don't think we've come out yet with the exact dose levels, but they are guided completely by the.

Previous data in Minnesota.

When do you expect to commence manufacturing runs for Gvhd, where it was for the phase one trial.

Yes, actually I'll ask Randy to if you could also take that please of course I'm happy to take that so manufacturing we are building because in the phase one trial.

The <unk>.

<unk> 201.

<unk> produced a ahead of time and not matched for patients but basically.

Italy available.

We are building our inventory already and expect to have.

The product available for patients as soon as the.

The first dosing is required.

Great. Thanks for taking our questions.

Thank you. Thank you.

Your last question comes from Matthew Cross with Alliance Global Partners. Please go ahead.

Hi, all good morning.

Wishes to Julian for a speedy recovery.

Had two quick questions related to the phase one phase two for <unk>.

And kind of following up on your comments there about the.

Scheduling for Liberal depleting chemotherapy antibody use I guess considering it.

<unk> study will be using the cryopreserved formulation about the fresh.

Just wanted to understand.

Any other kind of special considerations here I guess for the study compared to the prior one.

It sounded like the schedule will be more or less the same or based off the prior study.

You're not baking in time for.

NIM expansion with the cryo preserved just wanted to understand.

What does that schedule language would still look similar and maybe to kind of recap any modifications that would have to be made to the study.

We're needing to remedy the fda's concerns around donor eligibility requirements and anything that may play into which sites are able to participate in this study given the combination of dose donor eligibility requirement changes and.

The cryopreservation.

Here versus some previously thanks.

Sure I'm happy to answer that and I'll take the donor eligibility piece first.

No.

The donor eligibility procedures in that assay qualification.

We needed to make sure that those are compliant with FDA requirements and so we replaced our donor testing laboratory.

With the CLIA certified lab.

In the U S and we've now made sure that all appropriate test our license and cleared.

And that eligibility requirement has been met.

There has been no change in the design of the study.

Based on the quote.

The hold itself was not did not require any changes in the design of the study the protocol the sites or any or any anything that had to do with the clinical trial itself. It really was confined to the eligibility of the products. So that was actually.

Now in terms of Cryopreserved formulation so that.

It's great that you picked up on that so in the fresh formulation study patients had to have a donor available that the owner has to be recruited and if we can have to be done and then the product had to be.

Over time, so that that because we have a readily available product now it will be at the site. When the patient is enrolled and so that may save some time in terms of getting the patient up and traded because they will have product there. They don't have to wait for it then donor to get already get the donation and and and and do all that.

And then procedurally there will be a filing process.

The product will be there will be frozen and it'll be flat at the bedside to to provide the patient with the product when they need it.

Perfect. Okay Super helpful. And then just had one quick follow up which was around the sizing it looks like.

What you've drawn up on Clinicaltrials Gov that there is kind of a target enrollment of about 100 patients.

So I guess I know you are not commenting at this point on the actual dose levels that you may step through but just wanted to confirm whether that total sizing was based around kind of an expectation internally around the number of phase one cohort that you may look at it it will be ultimately still driven by the standard kind of safety evaluations as you step through doses or maybe just kind.

Or an expectation for the phase II population for that portion of the study just wanted to get a little bit of an understanding around the assumptions for that so that sizing yeah, absolutely. So so that number which was put in clinical trials dot Gov, which is incorporated into the protocol itself incorporates there is some flexibility there because you don't know for sure.

How many patients you're going to end up enrolling in phase one at the standard three by three design, where if there is a dose limiting toxicity observed in the <unk> numbers are expanded to include more patients and so this is the maximum size based on the possible expansion of.

And each of those.

Phase II portion has its own staff and analysis.

Surrounding it but they're relatively small cohorts to allow us to evaluate rather quickly.

<unk> results and then make decisions based on those early efficacy results that we see.

Understood. Okay. Thanks again for the clarity I appreciate it thank.

Thank you thanks, so much Matthew.

Thank you and this concludes our Q&A session I will turn the call back to Michelle coffee for her final remarks.

Thank you Carmen. Thank you very much for joining us for the call today, and we look forward to keeping you all updated on our future milestones have a nice day. Thank.

Thank you Carmen that'll conclude our call.

Thank you, ladies and gentlemen for participating and you may now disconnect.

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