Geron Corporation

Ladies and gentlemen, thank you for standing by and welcome to the fourth quarter 2020 Geron Earnings Conference Call. At this time, all participants are in the listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question during the session, you will need to press star 1 on your telephone. If you require any further assistance, please press star 0. I would now like to hand the conference over to Olivia Bloom, Jerome's Chief Financial Officer. Thank you. Please go ahead, ma'am.

Olivia Bloom Thank you, Erica, and good afternoon, everyone. Thank you for joining us for today's conference call. I'm joined today by Dr. John Scarlett, Jerome's Chairman and Chief Executive Officer, and Alexander Rizzo, our Chief Medical Officer. After the market closed today, we announced our fourth quarter and year end 2020 financial results and operational highlights via press release, which is available on our website under geron.com slash investors. In addition, a live webcast of this call is available on our website and an archive will be available for 30 days. Before we begin, please note that this presentation and question and answer session will contain forward-looking statements relating to Jerome's plans, expectations, timelines, beliefs, statements of potentiality, and projections. These include, without limitation, those regarding the expected timelines for completion of enrollment and the results from the eMERGE Phase III and IMPACT-MS clinical trials and submission of an MBA, the potential for positive outcomes from eMERGE Phase III and IMPACT-MS, potential approval of IMS HealthStats by regulatory authorities and commercialization of IMS HealthStats, the expectation that Geron's current financial resources will be sufficient to fund its operations until the end of 2022, and that Imatel's set has the potential to be disease-modifying and alter the course of MBS and MS. These and other forward-looking statements involve risks and uncertainty that can cause actual results to differ materially from those in such forward-looking statements. These risks and uncertainties include, without limitation, those regarding that the company may be unable to overcome all the enrollment, clinical, safety, efficacy, technical, scientific, operational, manufacturing, and regulatory challenges to meet the expected timeline for eMERGE Phase III and IMPACT-MF due to COVID or otherwise, that in the Phase III clinical trials, Imatelset may not prove to be as safe or efficacious as in the Phase II trials and may not demonstrate that it is safe, efficacious, and disease-modifying. that regulatory authorities may not permit the further development of IMITEL-STAT on a timely basis or at all, and may not approve it for commercialization, and that JIRA may need additional financial resources before the end of 2022 for the development and commercialization of IMITEL-STAT. Detailed information on the above risks and uncertainties and additional risks, uncertainties, and factors that could cause actual results to differ maturely from those in the forward-looking statements are explained under the heading Risk Factors in GERON's annual report on Form 10-K for the year ended December 31, 2020, filed with the Securities and Exchange Commission. Undue reliance should not be placed on forward-looking statements that speak only after the date they are made, and the facts and assumptions underlying the forward-looking statements may change. On today's call, Dr. Scarlett will make a few introductory comments, after which I will cover the fourth quarter and year-end financial results, as well as guidance for 2021. Dr. Rizzo will provide clinical development updates on the ongoing IMMERGE Phase III trial in our target patient population with low or intermediate one-risk myelodysplastic syndrome, which we call low-risk MDS, who are transfusion-dependent non-Delphi-Q and relapsed-actor or refractory tube prior treatment with an erythrocrease-stimulating agent, or ESA. She will also provide an update on our impact MS-based retrial, which is in a population of patients with intermediate to or high-risk myelofibrosis who are refracted to prior treatment with a JAK inhibitor, which we call refractory MF. Alexander will also discuss how the data and analyses we reported at the American Society of Hematology Annual Meeting in December 2020 have deepened our understanding on MS HealthSeth's mechanism of action and its effect on the underlying cause of the disease and the indications we're pursuing. Dr. Scarlett will then comment on the evolving low-risk MDS and MS markets and IMITEL staff positioning in those markets, given its potentially highly differentiated product profile. He'll finish the call with closing remarks on planned milestones for 2021. I'll now turn the call over to Dr. Scarlett, Geron's chairman and CEO. Chip?

Thanks, Olivia. I'd like to welcome everyone to our fourth quarter and year-end 2020 conference call. Let me start by sharing our vision for Geraldine, which is to become a leader in the treatment of hematologic malignancies by changing the course of these diseases, thereby improving and extending the lives of patients. As I look back on 2020, we made significant progress towards realizing this vision. To the end, we presented compelling and differentiating data from our eMERGE Phase II lower-risk MDS trial. These data showed high rates and exceptional durability of transfusion independence. Of any study in non-DEL5q lower-risk MDS patients who relapsed in a refractory to a EFA, the 20-month median duration of transfusion independence in eMERGE Phase II was the longest reported to date. In our eMERGE Phase III lower-risk MBS study, enrollment gains continued with over 50% enrollment achieved by the end of the year. We also presented exceptional overall survival data from our EMBARQ Phase II trial in JAK-I relapsed or refractory MF patients. In this trial, in the Telstat-treated patients had a median OS of 28 months, which is almost twice the median OS reported in medical literature. Based on these and other supporting data, and after conferring with FDA, we opened IMPACT-MF, our Phase III trial, and JAKI refractory MF patients. IMPACT-MF is the first and, to date, only Phase III trial with overall survival as the primary endpoint. We also presented strong results of disease-modifying activity in our patients with low-risk MDS and relapsed refractory MF. In both indications, we saw reductions in key driver mutations of the underlying disease. Furthermore, Imatel sets the only drug in development to establish a correlation of these and other measures of disease modification with key clinical outcomes, including durability of transfusion independence and low-risk MDS and improvement in MS. Such correlations have given us even greater confidence in the potential positive outcomes for our ongoing A3M. Okay. This doesn't stand out as important accomplishments in 2020. The first was that we successfully established our global Imatel stat supply chain, ensuring uninterrupted drug supply for clinical trials, and permitting inclusion of geron manufactured materials in our two Phase III trials. Second, we raised over $175 million in new capital from both an underwritten public offering and a new loan facility. As a result, we expect our current cash will be sufficient to fund our operations through the end of 2022. All of these accomplishments were made despite the challenges posed by the ongoing COVID pandemic. effect on clinical trial . And as of today, we've achieved 65% enrollment. As a result of the COVID pandemic, ongoing enrollment continues to be challenging. These enrollment challenges have been due primarily to the reluctance of patients to participate in clinical trials, as well as delays in opening new sites. These effects of the pandemic appear to be true for many oncology trials, not just ours. In addition, we believe enrollment in eMERGE Phase III may be starting to be impacted by luspatercept becoming more widely available as a treatment for patients who are RS-positive in low-risk MDS. From everything we can see, the enrollment constraints in this trial are not a result of eMERGE being inadequately prioritized by investigators. In our interactions with them, our investigators remain enthusiastic about our Immatelt stat data and are committed to enrolling their patients in this trial. Based on the information we currently have, we expect eMERGE Phase 3 to be fully enrolled in the second half of 2021. Depending on the exact timing when that full enrollment is achieved, we expect top-line results from Emerge Phase 3 to be available from the end of 2022 to the first half of 2023. Alexandra will discuss in more detail the initiatives we put in place to improve enrollment in the Phase 3 pandemic. She'll also update our activities in the conduct of Impact MF. We currently expect the interim analysis for IMPACT-MF to occur in 2024 and the final analysis to occur in 2025. Looking ahead, our plan strategic priorities for the next three years and commercially launching this highly differentiated drug in lower risk MDS. Now I'd like to hand the call. I'd like to hand the call over. Go ahead.