Q2 2020 Geron Corp Earnings Call

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Operator: Thank you for your patience. [inaudible] BF-WATCH TV 2021, Ladies and gentlemen, thank you for standing by and welcome to the Geron second quarter 2020 earnings conference call. At this time, all participants are in a listen-only mode.

[noise].

Ladies and gentlemen, thank you for standing by and welcome to the Gerrard second quarter 20 Shiny earnings conference call.

Operator: After the speaker's remarks, there will be a question and answer session. To ask a question during the session, you will need to press star one on your telephone. If you would like to withdraw your question, press the power button.

This time, all participants ARNA listen only mode. After the speaker's remarks, there will be a question and answer session to ask a question. During this session. You want me to press Star one on your telephone if you'd like to draw. Your question press the pound Keith if you require further assistance. Please press star Zero I would now like to hand, the conference over to your Speaker today says Amazon.

Operator: If you require further assistance, please press Star-Z. I would now like to hand the conference over to your speaker today, Suzanne Massari, Head of Investor Relations. Thank you. You may begin. Thank you, Dorothy, and good afternoon, everyone.

Sorry head of Investor Relations. Thank you you may begin.

Thank you Dorothy and good afternoon, everyone. Thank you for joining us for today's conference call I'm joined today by Dr., John Scarlett Gerons, Chairman and Chief Executive Officer, Olivia Bloom, The company, Chief Financial Officer, and Alexandra Reserve, our Chief Medical Officer.

Unknown Attendee: Thank you for joining us for today's conference call. I am joined today by Dr. John Scarlett, Geron's Chairman and Chief Executive Officer, Olivia Bloom, the company's financial officer, and Alexander Rizzo, our Chief Medical Officer. After the market closed today, we announced our second quarter 2020 financial results and recent events in a press release. It is available on our website under www.geron.com slash investment.

After the market close today, we announced our second quarter 2020 financial results and reach an event by a press release. It is available on our website under www Dot Gerrard Dot com flash investors.

Unknown Attendee: In addition, a live webcast of this call is available on our website and will be archived for 30 days. Before we begin, please note that this presentation and question and answer session will contain forward-looking statements relating to Geron's plans, expectations, timelines, beliefs, statements of potentiality, and projections. These include, without limitation, those regarding the timelines for completion of enrollment in the ongoing phase 3 eMERGE trial and the results from the interim and final analyses from the planned phase 3 Refractory MF trial, that Imitelstat has potential disease-modifying activity, that Geron's existing financial resources will be sufficient to fund operations into the second half of 2022, and that Geron's 2020 operating expense burn will be in the range of $70 to $75 These and other forward-looking statements involve risks and uncertainty that can cause actual results to differ materially.

In addition, a live webcast of this call is available on our website and will be archived for 30 days.

Before we begin please note that this presentation and question and answer session will contain forward looking statements.

Relating to John's plan expectation timeline belief.

Mr Potentiality and projection.

These include without limitation those regarding.

The timeline for completion of enrollment of the ongoing phase three emerge and plan to refractory AML clinical trials. The topline results from the ongoing phase three emerged trial and the results from the interim and final analyses from the plan B three refractory Nf trial.

And Imetelstat has potential disease modifying activity.

Veterans existing financial resources will be sufficient to fund operations into the second half of Twentytwenty to.

That Gerons 2020 operating expense burn will be in the range of 70 to 75 million.

These and other forward looking statements involve risks and uncertainties that can cause actual results to differ material materially from notes in such forward looking statement.

Unknown Attendee: These risks and uncertainties include, without limitation, those regarding that the company may be unable to overcome all the clinical, safety, efficacy, technical, scientific, Operational, Manufacturing, and Regulatory Challenges to Enable Expected Timelines for eMERGE and the Planned Refractory MS Clinical Trial, or that regulatory authorities may not permit the further development of Imatelstat on a timely basis or at all. That the COVID-19 pandemic may significantly impact the timelines for both the enrollment and the results of the clinical trial and or drug supply, as well as expectations for operating expenses, and that there may be unexpected operating expenses or events or changes in Geron's plans that cause either the 70 to 75 million in 2020 financial guidance to be revised, or the existing financial resources to be insufficient to fund operations into the second half of 2022.

These risks and uncertainties include without limitation those regarding that the company maybe unable to overcome all the clinical safety efficacy technical scientific.

Operational manufacturing and regulatory challenges to enable expected timeline for emerge and the plan refractory unmet clinical trial.

That regulatory authorities may not for NASA further development of Imetelstat on a timely basis or at all.

That the cobot 19 pandemic may significantly impacted timelines for both the enrollment and the results of the clinical trial, Android drug supply as well as expectations of operating expenses.

And that there may be unexpected operating expenses or events or changes insurance plan and John plans. They cause I did the 70 to 75 million 2020 financial guidance to be revise.

Or the existing financial resources to be insufficient to fund operations into the second half of 2022.

Detailed information on the above risks and uncertainties and additional risks uncertainties and factors that could cause actual results to differ materially from those into forward looking statements.

Unknown Attendee: Detailed information on the above risks and uncertainties and additional risks, uncertainties, and factors that could cause actual results to differ materially from those in the forward-looking statements is explained under the heading Risk Factors in Endurance's Quarterly Report on Form 10-Q for the quarter ended June 30, 2020, filed with the SEC. Undue reliance should not be placed on forward-looking statements, which speak only as of the date they are made, and the facts and Dr. Rizzo will provide an update regarding the effects of the COVID-19 pandemic on enrollment in our Phase 3 trial in lower-risk MDS, discuss key takeaways from the KOL event we hosted last, and cover other recently announced clinical development activities in MDS. Dr. Scarlett will then finish the call with an EU regulatory update and closing remarks. I will now turn the call over to Dr. Scarlett, Geron's Chairman and CEO. Thanks, Suzanne.

Our explained under the heading risk factors injure on quarterly report on form 10-Q for the quarter ended June Thirtyth 2020 filed with the FCC undue reliance should not be placed on forward looking statements, which speak only as of the date they are made.

And the facts and assumptions underlying the forward looking statements may change.

On today's call Dr. Scarlett plans to make a few introductory comments after which mid Bloom will cover the recent financing second quarter financial results and 2020 guidance Dr. Rizzo will provide an update regarding the effects of the cobot 19 pandemic on the enrollment in our days.

Three trial in lower risk Mds and discuss key takeaways from the K well event, we hosted last month.

And cover other recently announced clinical development activities in Mds and then that.

Dr. Scarlet will then finish the call within E U regulatory update and closing remarks.

I will now turn the call over to Dr., Scarlett guarantees chairman and CEO chip.

Thanks to them like welcome everyone to our second quarter 2020 conference call.

John A. Scarlett: I'd like to welcome everyone to our second quarter 2020 conference call. Before we begin, I'd like to make a few comments on the COVID-19 pandemic and how we're managing it, in compliance with state and local rules and for the health and safety of our employees.

Before we begin I'd like to make a few comments on the could 19 pandemic and how we're managing.

[noise] in compliance with state and local Wootton for the health and trade people employees.

John A. Scarlett: We're restricting access to our data centers, Our employees are continuing to work from home, and we are limiting business travel to essential business needs only. Despite these challenges, employee productivity and efficiency continue to be very high. Later in the call, Alexander will comment on the effect of COVID on our clinical trials. So during the second quarter, we achieved several significant milestones that have helped to establish a very positive trajectory. First, after a successful meeting with the FDA, we announced plans to move forward with the Phase III clinical trial in refractory MF using a primary endpoint of overall survival. The OS data from MBARC suggest imitelstat treatment could potentially double the remaining life expectancy for patients who become refractory to JAK inhibitors, which today are the only approved therapies for intermediate to and high-risk MF.

Restricting access to our offices or employees are continuing to work from home and we are limiting business travel to central business needs only.

Despite these challenges employee productivity and efficiency continues to be very high.

Later in the call Alexander will come in on the effective coded phone or clinical activities.

So during the second quarter, we achieved several significant milestones that have helped to establish a very positive trajectory for the company.

First after a successful meeting with the FDA, we announced plans to move forward with the phase three clinical trial in refractory MF using a primary endpoint overall survival.

First data from embarked suggest imetelstat treatment could potentially double the remaining life expectancy for patients who become refractory to JAK inhibitors, which did they are the only approved therapies for intermediate two anhydrous chemists patients.

John A. Scarlett: Second, maturing data from the eMERGE Phase II trial in lower-risk MDS presented at EHA highlighted extraordinary durability of transfusion independence, including our first time reporting a one-year transfusion independence for a significant number of patients. Also at EHA, we reported new analyses from the MBARC Phase 2 clinical trial in myelofibrosis that correlated the median overall survival observed with other clinical endpoints from the trial, such as improvement in fibrosis. Overall, the EHA data and analyses continue to support the potential disease-modifying activity of Intel STAT treatment, as evidenced by clinically meaningful, durable transfusion independence in lower-risk MDFs and improvement in overall survival in relapsed refractory MS. A third major milestone was achieved when we completed our recent public offering, which netted approximately $140 million, giving us the financial resources to execute our current development plans and to potentially reach two significant value inflection points that are expected in the second half of 2022.

Second the touring data from the emerge phase two trial in lower risk and yes.

Turning to equal.

Well I Didnt extraordinary durability of transfusion independence, including our first time reporting a one year transfusion independence for a significant number of patients.

Ultimately, how we reported new analyses from the embark phase two clinical problem I will fibrosis. The correlated the median overall survival observed with other clinical and went to trial such as improvement in fibrosis overall, the Johan <unk> data and analysis. He has continued to support the potential disease modifying activity until.

Step treatment as evidenced by clinically meaningful durable transfusion independence, and lowers can be up and improvement in overall survival in relapsed refractory on that.

A third major milestone was achieved when we completed our recent public offering which netted approximately $140 million, giving us the financial resources to execute our current development plans and potentially reach two significant value inflection points that are expected in the second half or 2022 those are.

John A. Scarlett: Those are achieving top-line results from the Emerge Phase 3 in MDS and completion of enrollment for the Planned Phase 3 in Refractory MS. This financing also brought on board a number of very well respected biotech specialist investors, including Eagle R1 Capital, Great Point Partners, NEA, RA Capital Management, and Samsara Biocapital. We believe their investment shows a strong endorsement of both Immittelstadt and GMO. So I'd like to hand the call over to Olivia to discuss what the financing means for our cash position, as well as the second quarter financial results. Thank you, Tripp. And good afternoon, everyone. As of June 30, 2020, we had approximately $265 million in cash, cash equivalents, and current and non-current marketable securities.

TV topline results from the emerge phase three in Mds and completion of enrollment for the planned phase three refractory M.S.

This financing also brought on board and number of very well respected biotech specialist investors, including Eagle or one capital Great point partners and he already capital management intends horribly capital. We believe their investment shows a strong affirmation of both the Mattel scavenger.

So I'd like to him to call over to Libya to discuss with the financing needs for cash position as what was the second quarter financial results Olivia.

Thank you Chad.

Good afternoon, everyone.

As of June 30, 2020, we had approximately $265 million in cash cash equivalents and current and non current marketable securities.

Olivia Kyusuk Bloom: Our cash position reflects net proceeds of approximately $140 million from a public offering of securities in the second quarter. Based on current planning assumptions, we expect such funds to be sufficient for our operations into the second half of 2022. And, as Chip just mentioned, this is when we expect to have top-line results for the eMERGE Phase 3 clinical trial in low-risk MDF and completion of patient enrollment for the planned Phase 3 clinical trial in refractory MDF. Overall, the financial results for the second quarter and year-to-date periods were in line with our expectations.

Our cash position reflects net proceeds of approximately $140 million school of public offering of security in the second quarter.

Based on current planning assumptions, we expect such fun to be sufficient for operations into the second half of 2022.

And it's chip just mentioned this is when we expect to have top line results, where the emerge phase three clinical trial and lower risk Mds and completion of patient enrollment for the planned phase three clinical trial in refractory enough.

Overall, the financial results for the second quarter and year to date period were in line with our expectations.

Olivia Kyusuk Bloom: Operating expenses for the three- and six-month-ended June 30, 2020, were generally higher in comparison to the same period in 2019, due to headcount increases in 2019 across the company, increased activity for the eMERGE Phase III trial in lower-risk MDLs, and new costs associated with validating the Imitelstat manufacturing process with the contract manufacturer. We expect operating expenses to be higher in the second half of 2020, in comparison to the first half, as we begin to support two phase three clinical trials of imitel, the ongoing eMERGE Phase 3 trial and the planned Phase 3 trial and refractory. Regarding financial guidance for 2020, we are reiterating our expectations of operating expense burn rates to range from 70 to 75 million.

Property expense for the three and six month ended June 30, 2020 were generally higher in comparison to the same period in 2019 due to headcount increases in 29 team across the company.

Increased activity for their merged phase three trial in low risk Mds.

And you costs associated with validating the imetelstat manufacturing process without contract manufacturers.

We expect operating expenses to be higher in the second half of 2020 in comparison to the first half.

We begin to support two phase three clinical trials of Imetelstat <unk>.

The ongoing emerged phase three trial and the planned phase three trial in refractory in that.

Regarding financial guidance, where 2020.

We're reiterating our expectation operating expense burden to range from $70 million to $75 million.

Olivia Kyusuk Bloom: This guidance reflects cash conservation measures implemented in April due to the COVID-19 pandemic, such as suspending travel and postponing a planned Emmett Hill staff proof of concept study. It also includes new costs for startup activities associated with the planned phase three trial and refractory MS, and Additional Costs for the Expansion of Clinical Flight for the Emerge Phase III Trial. Financial guidance is based on a set of assumptions at a point in time, and if the company's plans change.

Guidance reflects cash conservation measures implemented in April due to the coordinating pandemic such as it's been in travel and post Tony a planned imetelstat proof of concept city.

It also includes new call for startup activity associated with the planned phase three trial in refractory Ana.

An additional call for the expansion of couldn't clinical fight for the emerged phase three trial.

Financial guidance is based on a set of assumptions at a point in time and if the company plans change called the assumptions to be revised didn't week to update gotten is at that time.

Olivia Kyusuk Bloom: Causes Assumptions to be Revised, then we expect to update guidance at that time. With that, I will now turn the call over to Alexandra to discuss our recent KOL event and to provide an update on our Phase 3 clinical development activities.

With that I will now turn the call over to Alexander to discuss our recent caone relevant and to provide an update on our phase three clinical development activity.

Sandra.

Thanks, So media and good afternoon, everyone.

Alexander Rizzo: Thanks, Olivia, and good afternoon, everyone. Before I describe the key outcomes from our CAREL event, I'd like to give a brief update on the enrollment status of the eMERGE Phase III trial in low-risk MDM. As of the end of July 2020, approximately 93% of the 90 clinical sites originally planned for the trial were open for screening and enrollment, compared to 68% reported in May. The momentum of patient enrollment has begun to improve as the effects of the COVID-19 pandemic begin to wane in the majority of the countries where eMERGE clinical sites are located. We continue to expect patient enrollment to be completed by the end of the first quarter of 2021, subject to potential future delays or interruptions associated with COVID-19.

Before I described that key outcome from our Carol event I'd like to give a brief update on the enormous status in the emerge phase three trials in lower risk Mds.

As of the end of July 2020, approximately 93% of the 90 clinical sites. Originally planned played a trial, we're open for screening and enrollment compared to 68% report to see me.

The momentum of patient enrollment has began to improve the effects of the koby 19 pandemic begin to waning majority of the country reemerge clinical sites are located.

We continue to expect patient enrollment to be completed by the end of the first quarter, Oh 2021 subject to potential future the laser interruptions associated with calling on team.

Under current enrollment assumptions, we continue to expect topline emirates resolved to be available in the second half 2022.

Alexander Rizzo: Under current enrollment assumptions, we continue to expect top-line eMERGE results to be available in the second half of 2022. To ensure achievement of these goals, we are expanding the trial from 90 to approximately 130 clinical sites, and we expect the majority of the 14 new sites to be open for enrollment by the end of the year. Next, I will highlight key takeaways from the very successful KOL event we hosted in June, where three key opinion leaders reprised a total of four presentations from this year's IHA Annual Congress.

And insurance cheap month of these goals, we're expanding the trial from 92, approximately 830 couldn't go sites and we expect the majority of the 14 new sites to be opened for enrollment by the end of the here.

Next I will highlight key takeaways from the very successful can't really bank. We had hosted in June or three key opinion leaders reprice until we know for presentations from these here he how annual Congress.

First of all area and Teeny Associate Professor Hematology other universities are floris.

Alexander Rizzo: First, Dr. Valeria Santini, Associate Professor of Hematology at the University of Florence, presented more mature data from 38 patients in the eMERGE Phase II trial in low-risk MDS. As previously reported, the patients in this trial had a very high median transfusion burden of 8 units for 8 weeks at baseline, and 16 of 38 patients, or 42%, achieved at least an 8-week period of transfusion independence. The most important observation reported with this more mature data set was longer durability of transfusion dependence, including 11 of 38 patients, or 29%, being transfusion free for more than one year, and a median duration of transfusion dependence of 20 months. Transfusion independence with such durability is unprecedented, especially given the high baseline transfusion rate, which indicates disease modifying activity from TELSTAT. Furthermore, similar T.I.

Resented more mature data from 38 patients in the marriage phase two trial in lower risk Mds.

As previously reported the patient can be strong had a very high medium transfusion burden that 80 units for eight weeks at baseline and 16 up 38 patients or 42%.

Chiefs at least an eight week period of transfusion dependent.

The most important observation reported to be more mature data that was longer if your ability to have transfusion independence, including 11, 38 patients or 29% being transfusion free for more than one here.

And a medium duration of transfusion independence X 22 months.

Transfusion independence with fetched your abilities and places like especially given the high being planned transfusion breathing.

Which indicates disease modifying <unk> TV different I'd tell stuck.

Furthermore.

Similar T. I M. HIV rates were observed in both I mean theater of less positive and negative patient subgroups.

Alexander Rizzo: and H.I.E. rates were observed in both ring-cedar blast positives and negative patient subtypes. These eMERGE Phase II data have been well received by the hemolygnous medical community, and we believe they will generate even more enthusiasm about our ongoing eMERGE Phase II trial as well as favorably impact patient enrollment. Our second presenter was Dr. John Mascarenas, Associate Professor of Medicine and Director of the Adult Leukemia Program at Mount Sinai. Dr. Mascarin has reviewed very exciting new analysis from the EMBARQ trial in relapsed refractory MS patients, which, as expected, show that Mattelstat achieved dose and exposure-dependent reduction of all of the previously known pharmacodynamic markers of telomerase inhibition, such as civil rights activity to a mere length and expression of H3, confirming the on target mechanism of action.

These emerge phase two data have been well received by the hemodialysis can make a community and we believe they will generate even more enthusiasm about our own going in March we see trial as well as favorably in fact impact patient enrollment.

Our second presenter was up to join US grass Associate Professor of Medicine director of the idled leukemia program I think on finite.

Oh from US kind has reviewed very exciting year now hit from the embark trial in relapsed refractory administration.

As expected show that my tells that achieved dose and exposure independent reductions.

Oh, the previously known Pharmacodynamic markers of telomerase inhibition.

I, just don't write activity telomere length and expression.

Oh H. dirt.

Confirming the on target mechanism of action from Intelsat.

Furthermore, it before supported by an arms is indicating better clinical outcomes in patients with short or two on yours or higher telomerase activity level.

Alexander Rizzo: Furthermore, this was supported by analysis indicating better clinical outcomes in patients with shorter telomeres or higher telomerase activity levels. And, as previously established, these patients are more amenable to a telomerase inhibitor compared to patients with longer telomeres and lower telomerase activity. In addition, other analyses presented by Dr. Ana Mascarena showed improvement in overall survival that was now correlated with clinical benefits after metelstat treatment, most notably improvement in bone marrow fibrosis. Significant dose-dependent reductions in JAK2, CAR-LAR, and metolalue of burden were also reported. Taken together, these new analyses highlight that the observed improvement of fibrosis, the reduction in allele burden, and the improvement in median OS indicate potential disease-modifying activity of hematopoietin by targeting the underlying malignant myofibrosis clone. The third key opinion leader at our event was Dr. Rami Khamrokshi, Vice Chair of the Malignant Hematology Department at the Moffitt Cancer Reviewed new analysis of the clinical outcomes from metelstat treatment in triple negative myelofibrosis patients in the environment.

As previously thought unleashed these patients are more amenable, Switzerland, rightsignature compared to patients with longer to in years animal weren't telomerase activities.

In addition, either and I just presented but apparently most current I showed improvement in overall survival that was no correlated with clinical benefits after imetelstat treatment, most notably with improvement in bone marrow fibrosis.

You can't dose dependent productions in Jack to call, our and people all your bread and we're also reports.

Taken together these new analysis highlight that you observed improvements in February.

Reduction you know you bring them.

And the improvement in media and awareness indicate potential disease modifying it can be different itself that by targeting the underlying malignant myelofibrosis clones.

The third key opinion leader at our event took to Rami come Robertson Vice chair of the malignant hematology departments and the most cancer Center.

We get more now I just have the clinical outcomes, whom they tell us that treatment in triple negative myelofibrosis patients anymore.

And next patients who do not have the Jack to car and you Commutations.

Alexander Rizzo: MS patients who do not have the JAK2, CAR-R, and MIPO mutations are called triple negative and represent 10 to 15% of the mild federalized population. This type of condition is associated with shorter survival compared to patients carrying these limitations. The impact data presented by Dr. Camarotti showed an improved overall survival of 35.9 months in patients with triple negative myofibrosis, compared to 24.6 months for non-triple negative patients.

Cool to triple negative and your present, 10% to 15% off the mile clinical and he's population.

These type of could be she is associated with short term survival compared to patients carrying these indications.

The England data presented kind of took a merchant showed improved overall survival of 35.9 months in patients with triple negative myelofibrosis.

Compared to 24.6 months for non triple negative patients.

Yeah and seems on response rates were also higher for the triple negative fishing comparison on people know that declines.

Alexander Rizzo: Screening and Symptom Response Rates were also higher for the triple negative patients compared to the non-triple negative. Finally, the triple negative patients enrolled in the study had short telomere lengths and high telomerase expression levels as baselines. These patients with poor outcomes to current treatment options represent a suitable patient population for Imatel-Vaxx, and such patients will be eligible to enroll in our planned Phase 3 trial in Refractory Myofibril. To summarize, these analyses further support our planned Phase III clinical trial in Jack and Hugh's Refractory MS, which will be led by two principal investigators, Dr. John Mascarinas from Mount Sinai and Dr. Serge Rasto We're honored to have them on board as collaborators.

Finally.

The triple negative patients and moving to study had sure accumulate and high telomerase expression levels of baseline.

These patients we poor outcome to current treatment options.

And a suitable patient cultivation cornerstone for.

Such patients will be Leachables Gainesville, you know a planned phase three trial, you know Soc three mile suburbs.

To summarize.

Deep analysis for their support our planned phase three clinical trial.

Jackie we didn't refractory among which was led by two principal investigators. So if you're joining us grants or Mount Sinai interest or search for still FSIC from MD Anderson cancer Center.

We're honored to have them onboard its collaborators.

The plan Phase three study is global randomized open label trial in approximately 320 patients, we intermediate two or high risk in that.

Alexander Rizzo: The Plant Phase III study is a global, randomized, opal-labeled trial in approximately 320 patients with Intermediate II or High-Risk MS who are refractory to JAK inhibitors. The study will compare metastatic treatment to best available therapy, such as hydroxyurea and anazole, while excluding JAK inhibitors. We expect to involve over 150 sites across North America, South America, Europe, and Asia.

Who are refractory to JAK inhibitor.

Let's start even compare imetelstat treatment to best available therapy that just hydroxyurea dynasil well, excluding JAK inhibitors.

We expect total each over 100 and he's been sites across North America, South America, Europe and Asia.

Alexander Rizzo: Startup activities are ongoing, such as identifying potential clinical sites for participation, as well as finalizing the protocol and obtaining clearance from institutional review boards or ethics committees and regulatory authorities. We plan to open the trial for screening enrollment by the end of the first quarter of 2021. The trial is designed to have a final analysis for overall survival after more than 50% of the patients planned to be enrolled have died. An interim analysis of overall survival is planned to be conducted after approximately 70% of the total projected number of death events for the final analysis have occurred. If the pre-specified statistically significant difference in OS between the two treatment R's is met at the interim analysis, we expect such data could support a registration file.

Startup activities are ongoing such us identifying potential clinical sites for participation, that's probably China I think the protocol NMT any clearance from institutional review boards or ethics committees and regulatory authority.

We plan to open in the Troffers community enrollment by the end of the first quarter of 2021.

The trial is designed to have a final analysis for overall survival after more than 50% of the patients glad to be you'll have to die.

I need to my knowledge musical real survival is planned to be come back to go after approximately 70% of the total projected number that event for the final analysis have occurred.

The pre specified statistically significant differences between the two treatment arms.

I'd be interested.

We expect such data could support a registration filing.

Our current expectations for this study are to complete patient enrollment and the second half of 2020 team.

Alexander Rizzo: Our current expectations for this study are to complete patient enrollment in the second half of 2022, to conduct the interim analysis in the first half of 2023, and to finalize the analysis in the first half of 2024. However, as you know, both the interim and final analysis are event-driven and could occur at different times than currently projected. If this plan for three trials in refractory MS is successful, we believe Shumetel's dad will be the first drug to demonstrate a survival benefit in this poor prognosis, high unmet need, MS patient population. Now, I'd like to hand the call back to Jim.

Going back to the interim analysis in the first talk of 20 to 20, who.

And final analysis, So my first top upswing 24.

However, as you know both the interim and final analysis, our eventual and could occur due from clients don't currently projected.

These plan because clearly trial the news talk to and make a successful we do lesion that tells that we'll get the first truck to demonstrate a survival benefit this quarter privileges high unmet need and mass patient population.

Now I'd like to hand, the call back to cheap.

[laughter].

John A. Scarlett: Thanks, Alexandra. I'd like to end with a late breaking positive milestone that was recently reached. At the end of July, the European Commission formally granted orphan drug designation for Imitelstat in MDS in the European Union. Metolstat has already been granted organ drug designation by the FDA as a potential treatment for MDS. So both of these designations provide for potential market exclusivity. In the case of the EU orphan drug designation, it allows for potential market exclusivity of 10 years from the date of first approval in the orphan indication, making this latest designation a very welcome milestone. So, in closing today,

[laughter], Thanks, Alexander I'd like to end with a late breaking positive milestone that was recently reached at the end of July The European Commission formally granted orphan drug designation for Imetelstat in Mds in the European Union.

Let me tell us that has already been granted orphan drug designation by the 58 as a potential treatment for Mds.

Both of these designations provide for potential market exclusivity in the case or the E. U orphan drug designation that allows for potential market exclusivity of 10 years from the data first approval in the orphan indication, making this latest designation of very welcome milestone to treat.

[noise] so in closing today.

I'd like to comment the drone is a fundamentally different companies and we were a year ago.

John A. Scarlett: I'd like to comment that Geron is a fundamentally different company than we were a year ago. We have data to show that very meaningful clinical activity and indications where they're significant. We have an ongoing phase 3 trial in lower-risk MDS and a planned phase 3 trial in refractory MF for which we expect to begin screening and enrollment activities by the end of the first quarter of 2021. And we have the financial resources to reach significant value inflection.

Data shows that are very meaningful clinical activity in indications, where there was significant unmet need.

We have an ongoing phase three trial and lowers come together in a planned phase two trial refractory AML for which we expect to begin screening and enrollment activities by the end of first quarter 2021, and we have the financial resources to meet significant value inflection points.

We believe these achievements along with her experience development team that strategically position Jerome to become a leader in the treatment of human a logic myeloid malignancies over the next several years.

Operator: We believe these achievements, along with our experienced development team, have strategically positioned Geron to become a leader in the treatment of hematologic myeloid malignancies over the next several years. So, with that, we'd now be happy to answer questions. I'll turn the call back over to our operator. At this time, if you would like to ask a question... If you are a star, then the number one on your telephone keypad is star number one to ask a question, from the line of Gil Blum, with Needham.

So with that we'd now be happy to answer questions I'll turn the call back over to our operator.

At this time, if he would like to ask the question is can I start any number one on one telephone keypad try to star one to ask a question.

Well first question comes from a line of Gill with Needham and company [noise].

[noise] Hello, everyone and thank you for your questions then Doug Congratulations on all the progress so maybe a maybe a quick one on the 40 additional sites that were added to the Mds study I'm assuming that covert spray.

Gil Joseph Blum: Hello everyone, and thank you for taking my questions and congratulations on all the progress. So maybe a maybe a quick one on the 40 additional sites that were added to the MDS study. I'm assuming the COVID spread was maybe a criterion here, if you can comment on that. Hi, I can take that question.

I had was it'd be a criteria here if you can comment on this.

Hi, how are we back and take that question, Yeah, I'm, absolutely I was a you know the cold ignore money that well actually are made us a change the guidance since you might remember that the woman who will be completed by the end of the first quarter 2021, and just to make sure that we need to the goal.

Alexander Rizzo: Yeah, absolutely. It was, you know, the COVID moment that actually made us change the guidance. As you might remember, enrollment will be completed by the end of the first quarter of 2021. And just to make sure that we meet the goal in front of us, we decided to go ahead and open additional sites for this study. Alright, thank you for the color.

And from survives we decided to go ahead and old friend additional oxides for this for the study.

Alright.

Thank you for the color.

Another question I have is about the of validation process you discuss with Cmos.

John A. Scarlett: Another question I have is about the validation process you discussed with CMOs. Is the company looking at production at this site with an eye on commercial production and also to support the two pivotal studies that are ongoing? Yes, Gil. This is Chip.

This.

The company looking at the production at the sites with an eye on commercial production and also to support the two pivotal studies that are ongoing.

Yes skilled this is chip I'll take that absolutely we are.

John A. Scarlett: I'll take that. Absolutely. We are involved in putting together a complete validation program for the commercial scale process.

Or involved in putting together a complete a validation program for the commercial scale process and of course materials Nathan that validation program, which will include.

John A. Scarlett: And of course, materials made in that validation program, which will include, which will include final validation runs, et cetera, will also be used for clinical trial activities as well. But the primary purpose is really to validate the commercial process. Right. And kind of a last one on the myelofibrosis of Pivotal. As Alessandra mentioned previously, there were some really interesting data at EHA showing different responses from different genetic makeup populations

Tooling and include a final validation runs it took her will also be used for clinical trial activities as well.

Primary purpose is really to nobody's commercial process.

Right I'm kind of a last one on them I live here versus if at all.

I guess as a a sound to mentioned previously there were some really interesting data showing cannot different responses from difference genetic makeup population is there any look prospectively into let's say triple negative patients.

Alexander Rizzo: Is there any prospective look into, let's say, you know, triple negative patients? I can take that question, Gil. Yes. We, of course, as you indicate, plan to enroll triple negative patients will be enrolled as well in the refractory myelofibrosis study, and we have planned, or we are planning to conduct a subgroup analysis on this patient population as well as other relevant populations. So absolutely, we'll be looking into this. All right, excellent. And congratulations on all the progress. Keep safe. Thanks, Gil. Your next question comes from the line Ellen. Hi, this is Ellen Sands on behalf of Steve Willey.

[noise] Grant, though I can take that question Gil, Yes, Oh, we of course, but as you indicated trend to enable our work that the patients the triple negative patients will be and goals as well in the refractories malice I supposed to study.

And we have client or we are planning to come back on subgroup analyses on these patient population as well as other relevant Apple collision. So absolutely we'll be looking came to me today.

Right excellent and congratulations on all the progress in the keepsakes.

Thank you yeah.

Our next question comes from a line of Allen with Stifel.

Hi, This is Alan Sands on for Steve Willey. Thank you for taking my questions to maybe just the first one is just a follow up related to the increase in trial sites for the Mds study are these trial sites, mostly U.S. space or the international or both.

Unknown Speaker: Thank you for taking my questions. So maybe just the first one is just a follow-up related to the increase in trial sites for the MDS study. Are these trial sites mostly US-based, or are they international, or both? Yes, they are; they're distributed globally.

They are there Ah.

All right distributed globally. So we're looking to Ah that's been nucor decided to be will be across and internationally.

Alexander Rizzo: So we are looking to the new 40 sites will be across an international network. Okay, great. Thank you. And then can you maybe just remind us what the key differences are in the patient populations between the population that was enrolled in the phase two EMBARQ study versus the patient population you'll be looking at for the phase three study and relapsed MF? I know they're quite similar, but just any of those key differences would be helpful.

Okay, great. Thank you and then can you maybe just remind us what the key differences are and the patient populations between the population that was enrolled in the phase to embark study versus the patient population you'll be looking out for the phase three say in relapsed M.S. I know there quite similar but I'm just need as key differences maybe helpful.

Well the patients in both of our image studies are are seen or a in our defined there'd be no one responses to Jack and suture, there our technical defensive CNB eligibility criteria between the two studies, but fundamentally obese patients are no response, you straight JAK inhibitor and as as as you might.

Alexander Rizzo: Well, the patients in both of our MS studies are similar and are defined as being non-responsive to JAK inhibitors. There are technical differences in the eligibility criteria between the two studies, but fundamentally, all these patients are not responsive to a JAK inhibitor. And as you might know, 50% of the patients will discontinue, for example, after three years; 75% will discontinue after five years. And eventually, all patients that discontinue treatment with a JAK inhibitor will become eligible for treatment with Matelstat in our new study. And they were the same ones that were eligible for treatment with Matelstat in the MBART study. Okay, guys, just very technical differences in eligibility criteria.

No I mean, 50% of the patients will just continue for example, a racks. After three years 75, Toby just continuing dr. After five years you know so eventually all patients discontinued treatment with the Jackie Victor will become the Leachable ore treatment was going to tell his thoughts on our new study and they were.

The same way said were eligible for treatment of you're going to tell sat on the.

Embark study.

Okay got it just very technical differences and eligibility criteria and so maybe just one last question about the phase three relapsed and that study. So theres a number of other assets in development for class a and that's I know patients are required had seen a prior JAK inhibitor a appeal.

Alexander Rizzo: And maybe just one last question about the phase three relapsed MF study. So there's a number of other assets in development for relapsed MF. I know patients are required to have seen a prior JAK inhibitor. But are patients allowed to have participated in a previous clinical trial for an investigational product that is not a JAK inhibitor, or is that prohibited from enrollment?

As a loud had participated in a previous the clinical trials for an investigational products that is not a JAK inhibitor or is that prohibited from enrollments.

[noise] as long as Preppy show had eat refractory to a JAK inhibitor that can be summed he's a log on to clinical trials irrespective of other prior treatments.

Alexander Rizzo: As long as the patient is refractory to a jacket signature, that patient is allowed in the clinical trials, irrespective of other priors. Okay, great. Thank you. Your next question comes from the line: Hey, thank you for taking my questions. Congratulations on the progress. And sorry, if you answered any of these; I was jumping between calls here. So I was just going through my notes.

Okay, great. Thank you.

Your next question comes from a line of Andrew Dsilva with B. Riley.

Thank you for taking my questions. Congrats on the progress and sorry, if you answered any of these I was jumping between calls here.

So I was just going through my notes and not not to delve too deep with already discussed topics, but I'm just trying to get a sense of how you see things playing out here given encouraged encouraging to date has been so I know a low risk is overwhelming majority of Mds patients and.

Unknown Speaker: Not to delve too deep into already discussed topics, but I'm just trying to get a sense of how you see things playing out here, given how encouraging the data has been. So I know low risk is the overwhelming majority of MDS patients and RS positive is around 10% of total MDS patients, but what percent of low risk MDS patients have 5Q syndrome and are refractory to ESAs but are also RS positive? And then I'll have just one follow-up question on that. Okay, I just have to clarify something. It's actually only 5% of the patients that are Zell 5Q positive, and approximately 70% of the patients are RS positive, which is why I was, I was, you know, I think that's, that's important to say, sorry, on the other way.

Rs positive is around 10% of.

Total mds patients, but like what what percent of low risk Mds patients have a five two syndrome and our refractory to you says.

But are also Rs positive and then I was just a follow up.

And to that.

Okay, I just have to clarify something.

It's actually only 5% of the patients that are Delphi Q positive and approximately 70% of the patients are our S positive, which is why hours. Our it you know I think that's that's important to Oh sorry.

The iwear so it it's.

Unknown Speaker: So it's, So it's 5% of the patients that are del 5q positive, whereas our study, right, let me start again. Our study is enrolling non-del 5q positive patients. I just wanted to clarify that. Also, our study and our current efficacy data show that we are able to induce transfusion independently, as well as HIEs in both RS-positive and RS-negative patients. So I would ask you to repeat the question.

So it's 5% of the patients that aren't Delphi Q positive, whereas our study right. Let me start again, our study isn't going to non-GAAP five coupons with these patients I just wanted to clarify that also our study and our current efficacy data.

Is that we are able to induce transfusion independence.

As well as HM East in both Rs positive and Rs negative stage.

So I would ask him to repeat the question Whats worth exactly that you were asking us.

Alexander Rizzo: What was exactly that you were asking us? I was just trying to get a sense of RS, a positive patient breakout within your specific patient population criteria. I know it's a small percent of the overall population for MDS, but what part of the population is it in your specific low-risk population that you're targeting for your phase 3 trial? We are going to enroll patients irrespective of their RS status. I think that's the key to understanding, right? So it doesn't matter whether you're RS positive or RS negative. You can be enrolled in the Imatel-Stat Phase III study. Olivia, I think you had a comment.

Great I was just trying to get a sense of ours a positive patient a breakout would then years specific patient population criteria.

I know, it's a small percent of the overall population.

For Mds, but before well part of the population. It is it in your specific below risk population that you're targeting for your phase three trial.

We are they are going to enroll patients irrespective of the Iris a status I think that's the key to understand right. So be it doesn't matter, whether you're Rs positive or Rs negative you can be a involved on that you might telesat a phase three study.

Okay.

Olivia Thank you have to come up.

Oh, I think Andy I think your question is from <unk>.

Olivia Kyusuk Bloom: I think, Andy, I think your question is from what percentage of low-risk MDS patients are RF-positive? Correct. Also within your specific, you know, criteria for enrollment.

What percentage of low risk Mds patients are Rs positive.

Correct.

So within your specific you know criteria for enrollment.

Unknown Speaker: Oh, well, I think that's what Alexander was trying to say is that there is no criteria to exclude or include based upon RS positive or negative, because we're allowing both to come in. So whether you are positive or negative, you're allowed to come into what you're eligible for, which, if you're trying to contract potentially with other trials or other agents, you know, is true. So, for example, the most recent drug that was approved for low-risk MDS was Patercept.

Oh, well I think that's what Alexander was trying to say that there isn't your criteria to exclude or include based upon our positive or negative tivity, because we're allowing both to come in so whether you're RF positive or negative you're allowed to come into your eligible for trial.

If you're trying to contract potentially to other trials or other agents. You know is true. So for example, the most recent drug that was approved in low risk Mds Patterson out.

John A. Scarlett: Their trial was focused solely on RS-positive Right, so the really thing I was trying to get at was, even though they've received approval, you did have amazing transfusion independence and HIE relative to them. When you look back at the phase two data that you presented recently, and I was just trying to figure out, did you really think that it would be a hindrance in being able to enroll RS positive patients because the RS positive population in the phase two study actually did better? And so, just the data that came out would lead me to think that you'd still be able to enroll a pretty hefty amount of RS-positive patients. Right? Let me take that, Andy.

Their trial was focused solely on Rs positive patients.

Right. So I'm really the thing I was trying to get out was even though they've received approval you did have amazing transfusion independence in H.I.E. relative to them. When you look back at the phase two data that you presented recently and I was just trying to figure out did.

Are you really think.

That it'll be a hindrance in being able to enroll Rs positive patients because the the ROI positive population in the.

Phase two study actually did I say Oh better.

And so.

Just the data they came out would lead me to think that you still be able to enroll at a pretty hefty amount of ours positive patients.

Right.

He picked up MB. So I think our view is that.

John A. Scarlett: So I think our view is that the RS positive patients make up a minority, but nevertheless, an important minority of patients who are, you know, who have lower risk MDS. As we've said numerous times now, we are not inhibited in enrolling either positive, RS positive, or negative patients. With regard to the availability of patients for the clinical trial, I think that we are very likely to see a distribution of patients, plenty of whom will have RS positivity, because we seem to have a better profile than some other products in high transfusion burden patients. And so I think because of that, it's very likely that some investigators will choose to put even RS positive patients who are obviously indicated to be treated with, for example, reblosal. I think that they will preferentially put some of those patients with very high transfusion burdens into the Imatelstat trial.

The Rs positive patients makeup a minority, but nevertheless, an important minorities patients who are.

You know who have low risk Mds as we've said numerous times now we are not.

Came together did in enrolling either I pods are supposed to reverse negative patients with regard to the availability of patients for the clinical trial I think that we are very likely.

To see a oh, a distribution of patients plenty of who will have onerous positivity, because we seem to have a better profile than some other products in hi, transfusion burden patients until I think because of that.

It's very likely that some investigators will choose to put even though as opposed to patients who are obviously indicated to be treated with for example were blows old Oh, I think that they will preferentially put some of those patients with very hard currencies.

Interviewing post like travel so I think we're not anticipating a major issue in that regard.

John A. Scarlett: So I think we're not anticipating a major issue in that regard. Okay, now that's great to hear. And my last question as it relates to the 40 new sites, since these are all post-COVID sites that are coming up, could you let me know what kind of differences are taking place as far as discussions are concerned or what they need to happen before they feel comfortable actually joining the trial? I'm not sure Alexandra is. She is suffering a little bit from IT challenges post-hurricane. She's on the East Coast.

Okay, that's great to hear perfect and my last question as it relates to the 40 new sites.

[laughter] since these are all post covidien sites that are coming up could you. Let me know what kind of differences are taking place as far as.

Discussions are what they need to happen before they feel comfortable.

Actually joining the trial.

Not sure Alexander is I was vendor suffering a little bit from a from Ah Ah Ah IP challenges post hurricane she's on the East Coast. Alex are you bought back on given their apparently so can you hear that did you hear Andy's question.

Unknown Speaker: Alex, are you back online there? I'm here. So did you hear Andy's question? Yes, I did.

Hi, good ideas I mean, the new sites that we are adding on the study on a [laughter], there's really approaching them. We've been you odd data that we that we have a you know showing that the durability and that and a high transfusion dependent trade show honestly I don't think that.

Alexander Rizzo: I mean, the new sites that we are adding to the study, we're really approaching them with the new data that we have, you know, showing the durability and the high transfusion dependence rate. So honestly, I don't think that, you know, there is any additional requirement. As I said, the new data has generated enthusiasm across the community. And we have added these sites, and we are expecting most of them, if not all, to be open for screening and enrollment by the end of the year.

I know, there's any additional rate requirement.

As I said, the new data has generated enthusiasm across that came community and we you know we've we've added decide when we are expecting most of them if not all to be opened for screening and enrollment by the end of a year.

Okay, Great Hey, Congrats again, all the progress year to date and best of luck.

Alexander Rizzo: Okay, great. Hey, congrats again on all the progress year to date and best of luck coming back after the year. Thank you so much. And as a reminder, if you would like to ask a question... star, then the number one. Pad.

Hi, there.

Oh, thank you so much.

As a reminder, if you would like to ask a question. Please press Star then the number one on your telephone keypad toward next question comes from a lineup Tom Shrader with.

<unk>.

Hi, good afternoon extra for holding the coal.

Operator: For next- and line up Tom Schrader with BTI. Unknown Speaker, Robert Driscoll, Anil Kapur, Aron Feingold, Gil Blum, Kalpit Patel, Unknown, Hard to Enroll. Given you've got OS as an endpoint and JAK inhibitors have a very modest OS benefit, do you think you can relax that? Partly relevant to the previous question about how many things there are competing for MF patients now. Just your thoughts. Does OS free you up a little bit there?

Just one question at each kind of well event, John MUSC arenas invariably goes on and on about how high your burden is for Jack failure and it makes heart patients hard to enroll given you got a west as an endpoint and JAK inhibitors.

Very modest or west benefit do you think you can relax up partly relevant to the previous quite about how many things there are competing for MF patients now just your thoughts that does all west free you up a little bit there.

[laughter].

Yeah, I wasn't yeah, I can take back I mean, I I didn't quite interesting rate could put their question Tom I I don't believe we you know.

Alexander Rizzo: Yeah, I can take that. I mean, I don't know, it's an interesting way to put the question. So I don't believe we are experiencing difficulty, or we will be experiencing difficulties, based on the stream criteria. Just one point to remember is that when the IMPARC study started, it was, you know, five years ago; there were no studies in relapsed refractory patients. It was kind of really paving the way for this definition of relapsed refractory patients, which has been evolving over time. I believe that nowadays, the MS community, the MS treating community, is used to these criteria; they understand that it is needed, you know, for the conduct of the trial. And therefore, you know, I don't think that would cause difficulties in enrollment.

We are experiencing because they only will be experiencing difficulties are based on the no on the street criteria. Just 1.2 remember that when Wendy embark study started with a you know five years ago.

There were no studies in relapsed refractory patients each what's kind of a really paving the way for this our definition of to relapse refractory patients, which has been evolving over time I believe that nowadays the a mask amazingly enough treaty community he used to.

To be criteria, they understand that he needed a you know for four it conduct off the trial and therefore, you know I don't think that caused difficulties in enrollment.

Alexander Rizzo: In addition to that, as you say, overall survival is the golden endpoint or the golden outcome that everybody would expect from clinical trials. So that certainly creates enthusiasm, and we believe it will be driving interest and enrollment in the study. I don't know, Chip, if you have anything to add to this. Yeah, actually, I do.

Turning to that as you say I mean overall survival east, though the goals at any point towards our goal of an outcome that everybody would expect something for trials. So that's certainly creates enthusiasts on them and we believe will will be driving a interest any movement on the study.

I don't know cheap if you have anything to add two days.

Yeah actually I do I'm, just sort of a technical nature, Tom I think that this field is moving in the direction that that we're we're leading in here.

John A. Scarlett: Just sort of of a technical nature, Tom. I think that the field is moving in the direction that we're leading in here. I think that if you think about it, we're really requiring a certain number of months of Rux-limited treatment, and then we require at least two months out of that to be at stably dosed Rux levels in order to make sure that patients have had an adequate trial of a JAK inhibitor. And if they then meet the criteria for being refractory, then they are qualified for study. I see this in some other protocols as well that are coming up, probably because it's a relatively small KOL group that promulgates very tight inclusion criteria and exclusion criteria for these types of studies.

[noise] [noise] I think that the the.

If you if you think about where we're really requiring.

Certainly you know a certain number of months of Ruxolitinib treatment and then we require at least two months or out of so out of that to be it's stable we dose trucks.

Levels in order to make sure that patients that had an adequate trial of JAK inhibitor and and if they didn't meet the criteria for being a refractory then they then they are qualified Chevy I see I see this in some other protocols as well there are coming up probably because it's a relatively small okay.

Well Greek that Oh promulgate.

Oh theory.

A very tight.

John A. Scarlett: So I don't think we're really at a disadvantage there. I think we will be at a big advantage when it comes time to look at the data, because I think we'll have patients who really did get into the study only if they were truly refractory. And that will obviously tend to heighten the differences, we hope, between active treatment and BAT treatment. Sounds like it's got to be a question or not. Yeah, no, no, absolutely not. It makes sense for all the color.

Oh, yeah inclusion criteria and exclusion criteria for these types of studies. So I don't think we're really at a disadvantage. There I think we will be at a big advantage. When it comes time to looking at the data consulting cool have patients who really did get into the study only if they were truly refractory nuts that will obviously a 10.

The heightened the differences, we hope between active treatment and BHP treatment.

[noise] sounds that got every question or not.

No absolutely since protocols.

Yeah.

There are no further questions at this time, well now turn the call back over to doctors Garland.

Unknown Speaker: There are no further questions at this time. Well, I'd like to thank once again all of our covering analysts, their colleagues, and their banks. And I'd also like to thank our recent investors in the company; we appreciate very deeply the show of support and enthusiasm, as mentioned before, and our very, very hardworking community of investigators, study site coordinators, and, of course, our own internal employees. COVID is a challenge. But interestingly, I think we're finding ways around many of these issues.

Well I'd like to think a once again all of our Oliver covering analysts their colleagues or other banks [laughter] and I'd also like to thank our recent industries and the company. We appreciate very pleased to show support and enthusiasm assumption before.

And are very very hard working community is a investigators study site coordinators and of course or an internal employees quoted is a challenge, but interestingly I think we're finding ways around many of these issues and we feel like.

John A. Scarlett: And we feel good, as commented on more directly in the previous comments about going forward. So I'd like to thank everybody for that effort. And thank everybody for joining the call; I will bring you all for that. Thank you, ladies and gentlemen.

We feel good news commented on.

More directly in the in the.

Previous comments about going forward, so I'd like to thank everybody for that I've heard and thank everybody for joining the call today.

Equals runoff with them thanks for that.

Thank you, ladies and gentlemen that does conclude todays conference call you may now disconnect.

[music].