Geron Corporation

Good day and thank you for standing by and welcome to the Q1 2021 Chair and Earnings Conference call. At this time, all participants are in a listen-only mode. 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 1 on your telephone. If you require any further assistance, please press star 0. I would now like to hand the conference over to your speaker today. Olivia Bloom, please go ahead.

Great. Thank you, Celine. And good afternoon, everyone. Welcome to this conference call to discuss Geron's first quarter 2021 financial results and recent company events. I am joined today by Dr. John Scarlett, Geron's chairman and chief executive officer, and Dr. Alexander Rizzo, Geron's chief medical officer. After the market closed today, we announced our first quarter 2021 financial results via press release, which is available on our website. In addition, an archive of this webcast will be available on our website for 30 days. Before we begin, please note that this presentation and question and answer session will contain forward-looking statements relating to Gerald's plans, expectations, timelines, beliefs, statements of potentiality, and projections. These include, without limitations, those regarding the expected timelines for completion of enrollment of and the results from the eMERGE Phase III and IMPACT-MS clinical trials and submission of an NDA, the potential for positive outcomes from eMERGE Phase III and IMPACT-MS, potential approval of MMIC health stats by regulatory authorities and its commercialization, The expectation that Jaron's current financial resources will be sufficient to fund operations until the end of 2022, and that its operating expense burn in 2021 will be between $108 million and $112 million, and that Imatel's set has the potential to be these modifying and alter the course of MDS and MF. These and the other four looking statements involve risks and insurgencies that can cause actual results to differ materially from those in such four 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 timelines for the Emerge Phase III and IMPACT-MS clinical trials due to COVID or otherwise. that in the Phase III clinical trials, Imatel-STAT 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 Imatel-STAT on a timely basis or at all and may not approve it for commercialization, and that Geron may need additional financial resources before the end of 2022 for the development and commercialization of Imatel-STAT. 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 are explained under the heading Risk Factors in Jerome's quarterly report on Form 10-Q for the quarter ended March 31, 2021, filed with the Securities and Exchange Commission. 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 statement may change. And now I will turn the call over to Dr. Scarlett. Chip?

Thanks, Olivia, and good afternoon, everyone. During the first quarter of 2021, we continued to make progress in our two Immatolstat Phase III studies with registrational intent. First, enrollment continues to increase in our ongoing eMERGE Phase 3 trial. This trial is evaluating imotelstat and transfusion-dependent lower-risk MDS patients who are relapsed or refractory to erythropoiesis-stimulating agents, or ESAs. Last December, we had completed half of the planned enrollment of 170 patients in this trial. I'm happy to report that as of today, we have now achieved 75% of the planned enrollment. We continue to expect this trial to be fully enrolled in the second half of this year and for top-line results to be available as early as the end of 2022 or in the first half of 2023. Next, in IMPACT-MF, our second Phase III trial, that's evaluating imitelstat in patients with intermediate to or high-risk myelofibrosis who are refractory to prior treatment with the JAK inhibitor, we recently announced the first patient being dosed. This trial is the first trial of its kind in refractory MF patients with overall survival as the primary endpoint. Based on our current planning assumptions, we continue to expect the planned enrollment of 320 patients for this trial to be complete in 2024. Next, we are progressing on our preliminary activities for NDA and commercial readiness, which include long lead time manufacturing and quality activities, as well as developing a comprehensive organizational foundation to support a high growth and commercial stage company. Lastly, we look forward to presenting new data and analyses from our phase two Imatel-STAT trials at the upcoming European Hematology Association meeting, EHA, with the two abstracts that we submitted. The abstracts will be available online at ehoweb.org on May the 12th. Looking ahead over the next three years, we remain committed to achieving top-line results in eMERGE Phase 3, gaining regulatory approval of Imatelstat, and commercially launching this highly differentiated drug in low-risk MDFs. With that, I'll turn the call over to Alexandra. Alexandra?

Thank you, Chief, and good afternoon, everyone. As supported by our compelling Phase II data, we believe in a test that is clearly differentiated from the currently available treatment for lower risk MDS patients who are relapsed or refractory to ESA. Our numerous publications and presentations have reported meaningful and durable transfusion independence in high transfusion burden patients after treatment within a TELSAT, including a median duration of transfusion dependence of 20 months. In our eMERGE Phase III trial, as Chip mentioned previously, we have now achieved 75% of the planned enrollment. It appears patients are becoming more comfortable leaving their homes to participate in clinical trials, possibly due to the increasing vaccination rates and a decreasing number of severity of COVID cases in many of the countries where our sites are located. Moving on to our second phase three trial, Impact MS. Last month, we dosed the first patient in that trial. This was an important milestone in developing imetelstat for refractory myelophagrosis patients. Patients who fail or no longer respond to JAK inhibitor treatment have a median overall survival of only approximately 14 to 16 months. This is a clear indicator of the unmet needs in this patient population. As Chip mentioned, IMPACT-MS is the only study in refractory MS with overall survival as the primary endpoint. As the clinical results from this study is event-driven, the final analysis is planned to be conducted after more than 50% of the patients planned to be enrolled in the trial have died. An interim analysis is planned to be conducted after approximately 70% of the total projected number of death events for the final analysis have occurred. The number of events required to conduct the interim analysis could occur before enrollment is complete, as these events will accrue throughout the enrollment period. Based on current planning assumptions, our expected timeline for impact in that study remains the same as we've previously guided, with full enrollment and interim analysis projected to occur in 2024 and the final analysis in 2025. We're actively conducting site initiation activities around the world and recruiting patients. We currently plan to engage over 180 sites across five continents. We plan to employ similar enrollment boosting strategies for impact and less that we used for eMERGE phase three. Among the others, We will retain clinical sanitariums to interact with sites to alert patients and their physicians to the potential benefits of participating in IMPACT-MS. We will also utilize social media tactics to help drive patient awareness and recruitment. I look forward to the upcoming EHAD 2021 Virtual Congress in June, when our investigators present new clinical data and analysis from our phase two trials. The abstracts for these presentations will be available online later this week. These presentations will once again highlight IMETEL's potential to redefine the standard of care for MDS and MS patients. Now, I'd like to hand the call over to Olivia to discuss our first quarter financial results. Olivia? Thank you, Alexandra, and good afternoon again, everyone. As of March 31, 2021, the company had approximately $245 million in cash and marketable security, which we expect will be sufficient to fund our operations until the end of 2022. The increase in operating expenses for the first quarter of 2021 compared to the same period in 2020 was primarily driven by higher development expenses. This increase in R&D expenses include higher clinical development costs associated with our two ongoing phase three clinical trials, as well as the initiation of long lead time manufacturing and quality activities, such as manufacturing validation batches of Imatel Stats. These validation activities conducted in collaboration with our contract manufacturers are cornerstones for the planned MDA for Imatelstat and lower-risk MDF that we expect to file in 2023, assuming positive top-line results from eMERGE Phase 3. These validation batches will also provide the main data and information by which the commercial shelf life of Imatelstat will be set at the time of product launch. General and administrative expenses for the first quarter of 2021 increased slightly compared to the same period in 2020. This increase reflects our initial work to transform Geron into an enterprise fully prepared to rapidly ramp in both size and complexity in order to support potential commercialization efforts. Upon execution of our debt facility in September 2020, we drew down $25 million of the $75 million potential commitment. As such, interest expense for the first quarter of 2021 was $743,000. For net other income in the first quarter of 2021, we sold all of our holdings in an equity investment, resulting in a net realized gain of $1.2 million, including foreign currency translation adjustment. I'd like to conclude my comments by reaffirming our 2021 financial guidance We continue to expect operating expense burn to range from $108 million to $112 million. This burn includes cost of support to ongoing phase three clinical trials, produce validation batches of Imatel set of contract manufacturers, and to begin preparing regulatory filing for approval and commercial readiness. Financial guidance is based on a set of assumptions at a point in time, and if the company's plans change, causing assumptions to be revised, then we will update guidance at that time. With that, we have concluded our prepared remarks this afternoon. I will hand the call back to Chip and ask the operator to open the line for questions.

Thank you. At this time, I would like to remind everyone, in order to ask a question, press star, then the number one on your telephone keypad. Again, that is star, then the number one on your telephone keypad. We'll pause for just a moment to compile the Q&A roster. We have our first question coming from the line of Justin Walsh with B Reilly Securities. Your line is open.

Hi. Thanks for taking the question, and congratulations on the progress. Can you give us some color on what types of analyses we can expect at EHA and potential read through to the phase three trials?

Yeah, I think, unfortunately, Justin, the embargo rules prevent us from really commenting on the EHA abstracts or their content until they're published. And that happens on Wednesday. So we expect to comment at that point in time.

All right. Then maybe just, I don't know if you can comment on this. I'm wondering if the data cutoff for the abstract, do you expect that will be the same as what we see at the conference itself?

Alex, you want to comment?

I would refrain from commenting, Chip. I would just wait for the abstract to come online.

OK. Thank you, then. I'll have one more quick question here, then. I'm just curious. I'm correct in thinking that impact MS, you do not enroll patients who are receiving investigational therapies, correct? It's just current best available therapy? And then assuming that I'm right on that, Do you think that if you're having challenges in enrolling rapidly enough, that you'd be able to change the enrollment criteria, or would that potentially have some implications for your survival analysis? Go ahead, Alex.

Right. So Justin, you are correct. At the moment, we do not allow enrollment that are on other investigational therapies. We will just have to wait and see how things evolve. But typically, on clinical trials, you do not allow patients on investigational treatment to come on your trial as well.

Got it. Thank you for the questions. Thanks, Justin.

We have our next question coming from the line of Bonnie Quash with Stiefel. Your line is open.

Hi, this is Bonnie. I'm for Steve Wiley at Stiefel. I just have a few small questions about enrollment in the eMERGE trial and the impact of COVID. Do you have any thoughts on the effect of COVID on the type of patients enrolled? And for example, do you think that the patient population would skew more towards those with a higher transfusion burden since they're more willing to take the additional risks of going to a hospital and partaking in a clinical trial? And also, do you anticipate seeing more post-pattern set patients? Any color would be helpful. Thanks.

Alex?

Sure, I can take that shift. So, I mean, it's interesting. So I'll give first an overall kind of feeling of the enrollment and then maybe I'll answer to your two specific questions. So although we are seeing that the number of the COVID-19 cases is declining in certain regions, other regions are experiencing resurgence. And then we have these new variants that are coming up as well. So it's really unpredictable or uncertain about the pace at which the clinical trial operations may normalize. However, you know, at the moment, right, it is likely, like you are speculating, to say that maybe patients with a higher transfusion burden will be involved in a clinical trial. It's just very difficult to comment on that. The good side is that, as I said, it really works well in these patients. So, you know, if we have them on the trial, I wouldn't be worried about it. So I think that was one of your questions. And I'm sorry, I'm breaking on the other one. So one was the loose spider sets and the possible effects of loose spider sets. Lusparacept is doing great, right? The launch has been robust and that validates the unmet need and the market potential in this patient population. Remember that the label for Lusparacept is in RS negative, sorry, in RS positive patients only, whereas our trial, allows enrollment for all low risk MDS patients irrespective of presence of ring sideroblast. So while there might be some small impact on the patients that are RS positive, which let's remember it's not more than 20%, we do not expect to have any impact on the majority or on the bigger portion of the patient populations in lower risk MDS patients. Great. Thank you so much. Thanks, Bonnie.

Thank you. This concludes the Q&A portion of the call, and we'll hand it over to John Scarlett, Chief Executive Officer, for closing remarks.

Well, thanks very much for joining us today. It's been a busy earnings season, so we appreciate everyone who had the opportunity to dial in and participate. We're very excited about the progress we're making to bring this very important drug to patients. We're planning for Geron to become a commercial company in 2023 with the potential launch of Imetelstat and lower-risk MDS. The markets for both lower-risk MDS and refractory MS are highly attractive, and we look forward to sharing more updates with you as we progress through the year. Thanks, everyone, and have a good afternoon.

this concludes today's conference call thank you for participating you may now disconnect