Geron Corporation ($GERN)

Analyst working for Jason Zemansky. Welcome to the first day of the Annual Bank of America Vegas Healthcare Conference. We want to welcome Geron Corp. With me, I have -- sorry, I have Harout Semerjian, CEO; as well as Joe Eid, CMO. Welcome, guys.

Thank you very much, Jackie.

Great. So just to get us started, maybe to start broadly for those less familiar with Geron, can you give a quick overview of the story and your focus on blood cancers?

Sure, of course. And thank you for Bank of America for this invitation. Very happy to be here today sharing why we're excited at Geron as we progress with the company. So Geron is a company that has graduated to be a commercial stage company, really battling hematological diseases. We have launched our first asset, which is a telomerase inhibitor called imetelstat, RYTELO is the trade name, really trying to help more and more lower-risk MDS patients around the world. And now it's been about 18 months or so that we have launched, and we continue to grow and which is something we're really proud of. So as a company, we are very excited to be growing and helping more and more patients in lower-risk MDS as the first indication, but also we do have another indication, hopefully, that would have a readout in myelofibrosis as an interim in the second half of this year. So we can -- I'm sure we're going to talk about that as well.

Absolutely. Yes, that's a great intro. So maybe just to talk about the RYTELO launch. So as the launch evolves, what leading indicators do you view as most important RYTELO is gaining durable traction? And what should investors focus on over the next several quarters as proof points?

Well, I mean, it's -- our story is really one of focused execution at this point. So the most important metric, obviously, is our net revenue, which is an indicator of how we are able to advance and help more and more patients. And we're very happy to report that in Q1, we have made $51.8 million of net revenue. It's our biggest quarter ever. So that's something that we're very pleased with. But of course, the job will continue, and that's one of the things that we're going to be doing. At the same time, we've been sharing multiple different milestones with the investor community in terms of how many accounts are ordering RYTELO for the first time since launch, and that number is now 1,450 accounts across the U.S., which is really good. How much of our sales are coming from first-line, second-line patient population rather than the later-line patient population. That number has gone up to 33% as well in addition to our demand growth, which has grown 6% quarter-over-quarter and our revenue of 8% quarter-over-quarter as well. So these are all metrics that we continue to share with the market, and we plan to as well continue to share them over the next quarters to come that really would show our ability to get more and more patients to benefit from RYTELO in the low-risk MDS patient population.

Understood. And so you've been very clear that the second-line setting represents the core opportunity today. So how confident are you that RYTELO becomes foundational in this setting over time?

Look, we're very excited about RYTELO's opportunity in the second-line patient population in low-risk MDS. What we have communicated to the market is that in the U.S., there is more than 8,000 patients who can benefit from RYTELO. In the second-line patient population, we've refined our positioning so that all our messaging is focused on that second-line patient population. Our indication is in that second-line population. The NCCN guidelines support us in that second-line population. We also know that there are multiple more patients that can benefit from RYTELO in other lines of therapy, more advanced lines of therapy. But our focus is really in that second-line patient population, and we've been seeing growth quarter-over-quarter in that patient population. So we will continue to do our work from that side as a holistic company across commercial, medical and the idea is to grow that over time.

Sure. So part of this journey, cytopenias have come up quite a bit. And so you previously described them as predictable on target and potentially correlated with response. How are you driving this messaging and physician confidence?

Yes. Maybe that's a question. I'll turn to Joe. He's a hematologist. He's a trained hematologist and deals with these patients as well in the clinic, obviously, and he's our Chief Medical Officer. So Joe?

Yes. I mean, cytopenia for RYTELO is on target because we see a predictable pattern. Patients drop -- have a drop in their platelet and neutrophils in about 2 cycles on average. That's the majority of cases. And within 2 weeks after that drop, they recover in over 80% of the time. So this is what the IMerge data has shown. And we correlated and we presented the data at ASH that those patients that tend to have this level of cytopenia tend to have the most robust response overall. And the reason that, that happens is the drug does not work on the symptoms of the MDS being just improvement of anemia. It works on the disease itself. It's a mechanism of action that is directly correlating with that drop in counts and the recovery and eventually improvement in the symptoms and the anemia. The message that we have presented at ASH, the data is what is continuing in '26, making sure that we're bridging that gap in awareness and bridging the gap of how to manage patients because it's critical that physicians who are very familiar with the hem/onc space in terms of treatment, adverse events, et cetera, including cytopenia, they manage those patients on RYTELO properly so that they adhere to the treatment. And all they have to do is follow the label, how to dose manage, how to reduce, give a patient a break, resume treatment without dropping the patients at that point, which is critical. And now that we are telling them the cytopenia is correlating with response, it's better to maintain patient on drug. And there's an analog in MDS with lenalidomide, which has the same pattern. The difference is lenalidomide works in one specific group, which is the 5 deletion q group. Our drug has a broader spectrum beyond the 5 del q, and that's a good correlation between our drug and experience they have with lenalidomide. A lot of this messaging, Jackie, we've been pulling it through our engagement with the medical community as well. So it's not just about an ASH presentation. Now people know that the drug works in terms of durability of response, and that's a good thing. Now they also have a reason to believe of the why. Why is it important that you got to manage the cytopenias that are predictable. They happen in the first couple of cycles whenever they do happen. And what are you going to get in return in terms of efficacy for those patient populations.

Understood. So earlier today, you released a press release announcing you'll be presenting the first real-world evidence of RYTELO at EHA. What readouts should investors be focusing on?

Yes. Before I turn it to Joe, maybe just a kind of a framing around that. One of the areas that we've been heavily investing in over the last couple of years is really the real-world evidence and additional investigator-initiated trials. There has been a lot of interest from the medical community in terms of really seeing what else can we learn from RYTELO as we go with the launch. And that's been very good in terms of that engagement. And maybe today, Joe is in a situation where him and his team were able to get one of those very important trials as well move forward. So Joe?

Yes. A couple of things, Jacqueline. Our IMerge trial was mainly run in the European markets with very few sites in the U.S. So the hands-on experience was limited. The real-world evidence data that will be presented at ASCO and EHA is based at Moffitt Cancer Center and satellite setting. And that is reflecting on the real-world application versus what the IMerge trial, which is a controlled study. And what we have found in that study, which has 2 components, a retrospective component, which will be presented and then a prospective, which is accruing patients as it goes forward. The data is actually replicating the data from the IMerge, which is very comforting and providing confidence that if you manage patients appropriately, first line, second line, third line and beyond, the data that is observed is on par with IMerge. In some cases, actually, it's better, in particular, when we see a well-managed patient do better. And that's the message that I would convey from that real-world evidence. It's, again, a replication of the data in the real world in the clinic as opposed to a controlled setting.

Understood. So you mentioned on your Q1 earnings call, you would communicate your EU commercial strategy for RYTELO in LR-MDS by the end of the year. Can you discuss what strategies you're considering?

Sure. So as you know, RYTELO is a fully owned asset for Geron, and we have rights around the world, which is a very good situation to be in. And we actually have the EMA approval. So from a regulatory perspective, ex U.S. is also derisked. And that's important to know. Sometimes we forget that part. Now how do we bring the therapy to patients more and more. And that's something which is on our mind. Obviously, 90% of our focus and our team's focus is on the U.S. and making sure that, that happens. But we also believe that we have a global aspiration and duty to help more and more patients. We are seeing many other companies not necessarily wanting to move forward given -- and we understand why. I mean it is a volatile world. MFN is a real topic. It is something that everybody is thinking about as we progress. At the same time, we know that we have multiple different options. So as an example, partnership is always an option for us. A wholly-owned asset that we have full rights on an asset that's made $51.8 million in the last quarter and a company behind it that's saying it's $220 million to $240 million in terms of net revenue for this year. That's not too many of those assets are lying around. At the same time, we do know that what we don't want to do which is the old classical model in Europe, where you invest 50, 60 people in Germany or in France or other areas, those days are probably not the most beneficial in terms of cash allocation and resource allocation. At the same time, one of the things that we have said in our Q1 call is we are also seeing additional types of companies emerge in Europe where we can potentially partner with, but in very restricted manners, where they can be our extension in certain areas, be it contract sales organizations and others where some of the things that are important for us in terms of the pricing as an example, we're very close to it, and we're guarding it very carefully. But at the same time, we can partner. So what we have said is we're in the middle of looking at all these different options, and we plan to come back to the market with an update on our EU strategy before end of year.

Great. So in these last couple of minutes, maybe we can shift to your pipeline, specifically IMpactMF. So what gives you conviction that imetelstat can translate into meaningful benefit into myelofibrosis, particularly in the relapsed/ refractory post-JAK inhibitor setting?

Great question...

Yes. I mean the first foray of imetelstat in the clinic in the heme space was in essential thrombocytosis, which is caused by malignant megakaryocytes. That's the precursor of a platelet. They tend to secrete fibrin, which causes fibrosis in the marrow. That study showed almost 100% complete remission with half of that group of patients having the myelofibrosis mutation in the JAK load. And that was the basis for the IMbark study, which was 109 patients randomized to a high dose of imetelstat versus low dose. And in that study, there was improvement in the symptoms, but more importantly, almost tripling of the survival, also indicators of disease modification parameters such as reduction in the VAF or mutation load as well as reversal of myelofibrosis in the marrow, symptom relief, cytokine reduction. That was the ground to give the company confidence to go into registration stage, which is the IMpactMF trial. It's a 2:1 randomization with an overall survival endpoint, which is unique in this space. And this trial is comparing the high dose of imetelstat, RYTELO, which is higher, 8.9 milligram given more frequently every 3 weeks against best available therapy. So on that basis, we have confidence in the science, and we're hoping that this will read out to help patients.

Great. Well, it was great having you guys here. And yes, thanks for having you guys.

Thank you for the opportunity.

Thank you.