Q1 2024 Geron Corp Earnings Call
Thank you for standing by my name is Jillian I will be your conference operator today.
Jillian: This time I would like to welcome everyone to the Geron Q1, 2024 conference call.
Jillian: Lines have been placed on mute to prevent any background noise.
Jillian: After the Speakers' remarks, there will be a question and answer session.
Jillian: If you'd like to ask a question. During this time simply press star followed by the number one on your telephone keypad.
Jillian: If you would like to withdraw your question Press Star one again.
Speaker Change: I would now like to turn the conference over to Erin Fund Gold you may begin.
Speaker Change: Good morning, everyone. Welcome to the Geron Corporation first quarter 2024 earnings Conference call I Am Erin Feingold Jones, Vice President of Investor Relations and corporate Communications I'm joined today by several members of <unk> management team, Dr. John Scarlett, Chairman and Chief Executive Officer.
Speaker Change: And Youll Kapoor Executive Vice President of corporate strategy, and Chief Commercial officer, Dr. Pei seller Executive Vice President and Chief Medical Officer, Michelle Robertson Executive Vice President and Chief Financial Officer, and Dr. Andrew <unk> Executive Vice President and Chief operating officer before we begin.
Speaker Change: Please note that during the course of this presentation and question and answer session. We will be making forward looking statements regarding future events performance plans expectations and other projections, including those relating to the therapeutic potential and potential regulatory approval and that's helped that anticipated clinical and commercial events and really.
Speaker Change: <unk> timeline, the sufficiency of Geron financial resources and other statements that are not historical fact, actual events or results could differ materially.
Speaker Change: Therefore, I refer you to the discussion under the heading risk factors in <unk>. Most recent periodic report filed with the SEC, which identifies important factors that could cause actual results to differ materially from those contained in the forward looking statements Geron undertakes no duty or obligation to update our forward looking statements with that.
Speaker Change: I will turn the call over to chip.
Chip: Thanks, Aaron Good morning, everyone. Thanks for joining us.
We're poised for a successful U S launch of Intelsat for the treatment of transfusion dependent anemia in patients with lower risk Mds.
Chip: Its crude we're deeply excited for the opportunity to bring patients. What we believe is an important and differentiated medicine.
Chip: With our <unk> date of June 16, we continue to work closely with the FDA.
Chip: The review of our new drug application.
Chip: As part of the NDA review process on March 14th the Fda's oncology drugs Advisory Committee or <unk> voted 12 to two in favor of the clinical benefit risk profile with Intelsat for its intended indication in lower risk Mds.
Chip: The resounding support for <unk> reflected in the <unk>, which also echoed in comments from the lower risk Mds community during the public Forum.
Chip: Hematologists and patients alike spoke out about the burden of transfusion dependent anemia in this disease and the need for new treatments, particularly for patients with difficult to treat subtype, such as high transfusion burden and Rs negative patients.
Chip: <unk> is uniquely positioned to address these underserved transfusion dependent mds patient populations.
Chip: We are in the final stages of commercial readiness execution, which has included bringing on a sales force in April 2024.
Chip: We're also engaging in marketing commercial access payer and reimbursement preparatory efforts.
Chip: Just as we are completing the build out of our enterprise capabilities and systems to support our transition to a commercial stage company.
Chip: In short we are building on our momentum.
Chip: With urgency and fully covenant, our readiness for U S launch on potential approval.
Chip: We're also financially well resource to support the planned U S commercial launch with approximately $465 million on the balance sheet as at March the 30 <unk> of this year.
Chip: On the heels of the highly positive DAC outcome, we raised approximately $141 million in net proceeds from an underwritten public offering of common stock and pre funded warrants.
Chip: This offering included participation from our capital Fairmount <unk>.
Chip: Carolina outage boxer vivo deep track and multiple large investment management firms. In addition to other new and existing investors.
Chip: From an EU perspective, we also have an MAA or marketing authorization application under review in this indication is.
Chip: If <unk> is approved by the European Commission, we expected commercial launch in Europe would occur in 2025.
Chip: We're continuing to evaluate our strategic options for European commercialization, including self commercialization or partnering and expect to provide an update later this year.
Chip: Like to turn briefly to our ongoing clinical development efforts and Jack I relapsed and refractory MF patients.
Chip: As many of you recall in.
Chip: <unk> is the only phase III MF study with overall survival as the primary endpoint.
Chip: Last month, the data monitoring committee evaluated unblinded data and recommended the clinical trials continue.
Chip: In addition, the company reviewed enrollment rates and blinded death rates, which are lower than anticipated based on the initial planning assumptions. Accordingly, we are updating our guidance to extend the timelines by half a year with the interim analysis now expected in early 2026.
Chip: Final analysis expected in early 2027.
Chip: We in the trial investigators remain excited about this study and the potential of a treatment that could improve survival for these patients.
Chip: Currently have very few treatment options and dismal survival rates they.
Chip: Jay will provide more color on this trial later in this call.
Before I turn the call over to the team.
Speaker Change: To take a moment to reflect on how inspired identify all of our German people.
Speaker Change: Their passion for bringing our medicines to patients is palpable and drives our culture.
Speaker Change: From our longest tenured colleagues tell me <unk> I M D.
Speaker Change: Our sales force that joined US just a few weeks ago. There is a deeply shared sense of purpose that each one of us can meaningfully contribute to improving the lives of patients with hematologic malignancies.
Speaker Change: I believe that this unity around our mission drives our urgency and collaboration and is a critical factor in transforming our fast growing organization into a successful commercial company.
Speaker Change: With that I'm going to turn the call over to Aneel for a commercial update.
Aneel: And Neil.
Aneel: Thanks, Chip and good morning to everyone on the call.
Aneel: We believe that we are positioned very well for commercial and value creation.
Aneel: Well prepared to execute a successful U S launch upon potential approval.
Aneel: As Jeff mentioned in April we.
Speaker Change: We completed the build out of <unk>.
Speaker Change: <unk> commercial organization with the hiring of our sales force.
Speaker Change: Which has been now integrated and trained so that they will be prepared to be deployed in whiskey upon potential approval.
Speaker Change: I've had the privilege of getting to know this team really well.
Speaker Change: And I'm deeply impressed by their caliber experience and from a quick.
Speaker Change: We also have commercial supply arrangements in place and have finalized our specialty distribution network and third party logistics.
Speaker Change: From a market perspective, we have worked to identify the quantum graveyard noticed Mds prescriber base.
Speaker Change: And our patient access and affordability of distributions are on target for implementation at launch.
Speaker Change: To support our launch preparation and commercial strategy. We have conducted extensive market insights, which suggests that <unk> started hiring a differentiator in this transfusion dependent lower risk Mds market.
Speaker Change: Research has shown that medical affairs stakeholders are dissatisfied with the available options and the low risk Mds space, which we believe creates an opportunity for them it doesn't sag.
Speaker Change: Additionally, they expressed enthusiasm for the totality of clinical benefits team the dividend stocks, including delivery.
Speaker Change: Blood cell transfusion independence.
Speaker Change: Hemoglobin increases and reduction in transfusion burden.
Speaker Change: Balanced with a generally well characterize and manageable safety profile.
Speaker Change: Lastly from a pattern on regulatory exclusivity perspective in the U S. As shown at the end of the slide deck, we are using today.
Speaker Change: Orphan drug exclusivity through first half 'twenty <unk> MBS.
<unk> heard of us backing for Mds and Emma expires in March 2033.
But if the patent term extension is applied to this used backend we expect exclusivity would be extended through 2037, and we will apply to all users covered by the backend, including both Mds and MMS.
Speaker Change: We believe <unk> is uniquely positioned to address unmet needs in transfusion dependent lower risk Mds as captured on this slide which.
Speaker Change: Depicts the treatment landscape as well as the <unk>.
Speaker Change: Taking the current in CCM guidelines for MBS.
Speaker Change: As you may be aware the MTN guidelines, along with published results from there are no my style remain among the most important factors that influence clinical impaired pathways and significantly inform prescribing behavior.
Speaker Change: The NCC guidelines.
Speaker Change: And Esa.
Speaker Change: Treatment option for the largest segment of frontline Rs negative patients.
Speaker Change: It's important to remember that many of these patients will show a loss of response to Esa treatment in the frontline setting in approximately 18 to 24 months.
Speaker Change: That also continues to be limited treatment options for patients with serum <unk> levels greater than 500 ml per in Europe.
Speaker Change: And participation in clinical trials for the year.
Speaker Change: Ineligible Rs negative patients is encouraged by MCT and guidelines.
Speaker Change: From our perspective these guidelines reflect a lack of effective treatment options.
Speaker Change: In particular for those patients who are Esa ineligible.
Speaker Change: Transfusion burden patients and put Addis negative lower risk Mds patients, who constitute approximately 75% of the market.
Speaker Change: This is a need we believe <unk> can Paolo could you address as they potentially durable treatment.
Speaker Change: It can be used broadly across MBA setbacks.
Speaker Change: This next slide summarizes our latest market research from 'twenty to 'twenty call of 50 community and academic U S. Hematologist Vin.
Paolo: When survey about the product profile of <unk> and what factors made the right decisions.
Paolo: Neither snacks third priorities and the low risk Mds space.
Paolo: Our findings showed that these physicians note that achievement of RBC and rise in hemoglobin are two key factors that drive their decision making.
Paolo: They point to the greater than 24 week Ti and patient reported outcome data from emerge as essential considerations.
Paolo: These physicians view <unk> stock as the likely standard of care.
Paolo: Cross the relapsed refractory Rs negative patient population expressing enthusiasm for the significant efficacy improvement in EF.
Paolo: Population with limited current treatments.
Paolo: Physicians also note transfusion burden has strong impact from treatment decisions and believe <unk> started a compelling option for patients with high transfusion burden improved differentiated data in this patient population.
Paolo: The Amazon start profile also resonates with pad priorities and they recognize the unmet need in the transfusion dependent lower risk Mds space.
Paolo: The express back end CCM guideline inclusion and peer reviewed publications I've seen as important evidence for their consideration.
Paolo: Inclusion in <unk> guidelines requires an FDA label as well as the publication of the pivotal data, which is already available in the lines of it.
Paolo: Once potential FDA approval is secured or themes.
Paolo: I need to engage with end CCN to begin the process of updating the guidelines.
Paolo: Important to note that given the elderly patient population with transfusion dependent lower risk Mds. The majority of USD <unk> are expected to be treated.
Paolo: Under the Medicare part D. Setting we are confident patients will have broad access to them at that side of it.
Paolo: We expect to see those start uptake across Esa ineligible Esa fail Addis negative.
Paolo: Odyssey positive high transfusion burden patients.
Paolo: Based on our latest 2020 for market research of 50 U S based practicing hematologist across both community and academic settings.
Paolo: On the left hand side of this slide you can see that our market research suggests meaningful <unk> start to use and frontline Esa ineligible patients, especially those who are Rs negative.
Paolo: The <unk> side of the slide shows the estimated second line population.
Paolo: Ultimately, 90% of which I would expect it to be.
Paolo: Yesterday <unk> experienced.
Paolo: Our market research suggests a broad use of chemical start across the second line, regardless of frontline therapy, particularly put out its negative patients.
Paolo: As you can see on the figures on the right.
Paolo: Charles further segmented put out as positive <unk> negative patients within these subgroups.
Paolo: Our findings confirm the significant unmet need across the lower risk Mds patient population can be strongly believe that <unk> start to come through can play a meaningful role in the treatment paradigm of transfusion dependent motives MBS moving forward.
Paolo: We believe we are well positioned to capitalize on emmerdale start opportunity in transfusion dependent lower risk Mds by building on the unique product profile and executing on the launch critical success factors that are driving our commercial plan.
Paolo: From a prescriber perspective, we have a few important Ngos.
Paolo: That prescribers investor totality of clinical benefit achievable with <unk> stock and understand the efficacy profiles across MBA subgroups, including artist negative and high transfusion burden patients.
Paolo: It's also critical that we provide education and support positions on the <unk> safety profile and help them to contextualize and managed Cytopenia is so they can offer and optimize patient experience and duration.
Paolo: This support will also help prescribers had a good first experience with <unk>, which is another important growth.
Paolo: From a patient access perspective as reflected by our research.
Paolo: Need for new treatment options and the high unmet need.
Populations that <unk> can address.
Paolo: We expect it will be important considerations to drive access and reimbursement.
Speaker Change: We are honored and privileged to have the opportunity to launch a medicine.
Speaker Change: That could have such significant meeting for transfusion dependent lower risk Mds patients and their families.
Speaker Change: And we are deeply energize to embark on this journey.
Speaker Change: With that I'll turn the call over to <unk> for a medical and clinical update.
Speaker Change: Thanks Neil.
Speaker Change: Everyone for joining our call.
Speaker Change: As Chuck mentioned on March 14th.
Speaker Change: <unk> Advisory Council.
Speaker Change: <unk> in favor of the.
Speaker Change: Clinical benefit risk profile than the <unk>.
Speaker Change: Looked at in transfusion dependent lower risk can be absolutely correct in our MDI.
Speaker Change: <unk> results from our phase III emerge trial as long as the unmet need and limited available treatment options for patients with cancer.
Speaker Change: Needless to file.
Speaker Change: Very pleased with the community of propulsion to recognize the positive clinical benefit risk profile of an account that for the treatment of transfusion dependent anemia in adult patients with low aircrafts on block.
Speaker Change: As Ted mentioned, the public Forum provided a critical opportunity.
Speaker Change: To hear from members into lower risk Mds.
Speaker Change: Okay, let me apologize patient advocacy and patient.
Speaker Change: Rockies technologies, there are several time trucking until progressing.
Speaker Change: I think hematology experience and triple lower risk Mds patient.
Then also echo what we hear in our market research and that medical conferences.
Speaker Change: The profiling negative impact of <unk>.
Speaker Change: Fusion dependent on the quality of life for patients and their families are strikingly evident.
Speaker Change: <unk> are chronically fatigue and struggle to keep our protecting every activity, commonly resulting in like a social and emotional burden.
Speaker Change: Lower risk Mds is progressing quickly and by the time the patients progressed to transfusion dependent.
Speaker Change: Do not have any treatment options.
Speaker Change: In fact, Hematologists <unk> pointed out that they usually end up cycling through most or all of their options.
Speaker Change: In addition option in their armamentarium potentially practice changing.
Speaker Change: There was also a significant discussion around the deep meaningfulness of RBC transfusion independent for these patient.
One of whom testified at the air DAC that she believe transfusion independence, but give us more time to have a better quality of life.
Speaker Change: A nurse commented in the public hearing that time not spent any infusion chair time rich.
Speaker Change: Okay.
Speaker Change: From a clinical perspective Mafia infusions can provide short term relief, but can also cause long term consequences.
Speaker Change: <unk> Red blood cell transfusions can leak alanine ocean and difficulty in identifying a match donor to support the continuous transfusion need.
Speaker Change: Over time patients can develop and organ dysfunction due to iron overload.
Speaker Change: My question to the cycle of social and emotional toll that transfusion dependent patients.
Speaker Change: The hematology in person community has expressed that beyond durable RBC ti the hemoglobin increases and reduction in transfusion burden and observed within accounts that are important indicators of clinical benefit.
Speaker Change: Lastly, several hematologist spoke out about their comfort level managers had a clue given their familiarity with these clinical logic toxicity at the side effect of novel medicines regularly critical practice.
Speaker Change: This reinforces our belief that with the proper context and education. These cytopenia shouldn't be able to be well managed by hematologists in a real world setting.
Speaker Change: Overall, hematologists patient advocacy and patient expressed support for new treatment options.
Speaker Change: Fusion dependent lower risk Mds, especially for treatment with the durability of RPC Ti clean with Comstock.
Speaker Change: Turning next to our medical affairs readiness to support our expected launch.
Speaker Change: Our highly skilled medical affairs team in place.
Speaker Change: His team has been instrumental in conducting peer to peer scientific exchange of medical information, particularly medical Congresses and publication.
Speaker Change: Awareness that on the cost side of the hematology community has been heightened by tariff presents a congresses like <unk> and ash and particularly by the publication of phase III emerge data in Atlanta last year.
Speaker Change: Our medical affairs team will partner with our commercial colleagues on the important effort to have an account that including PCM guideline.
Speaker Change: It is approved.
Speaker Change: Lastly, our field medical colleagues are in place and are prepared to be a critical resource and how to support physicians in managing their lower risk Mds patients.
Speaker Change: Turning now to our phase III impact on that trial agenda talk that inject inhibitor relapsed refractory.
Speaker Change: Okay.
Speaker Change: We are proud to sponsor the first and only phase III trial in AMR overall survival as a primary endpoint.
Speaker Change: A total of 320 patients are planned to be enrolled.
Speaker Change: 301, randomization for Bina talked about arm versus the best available treatment arm.
Speaker Change: Protocol interim analysis had been clients and about 35% of the planned enrolled patients have died on the final analysis when approximately 50% of the planned enrolled patient.
Speaker Change: Got it.
Speaker Change: As an overall survival study the timeline for the interim and final analysis is the pending not only on the rate of enrollment, but also on the event rate for patient suffering from the trial.
Speaker Change: The data monitoring committee evaluated unblinded data last month and recommended the study continue.
Speaker Change: In addition, we review the enrollment rates and blindly gas rates, which are lower than anticipated in our initial planning assumptions.
Speaker Change: Accordingly, we are updating our guidance to extend the timelines are happier.
Speaker Change: Alex back the interim analysis in early 2026 and the <unk>.
Speaker Change: Final analysis in early 2027.
Speaker Change: The factors affecting these estimates are highly variable and difficult to predict.
Speaker Change: Actual interim and final analysis could occur sooner or later than we currently.
As the trial continues we'll continue to monitor enrollment and death rates and update items if appropriate.
Speaker Change: Of note, we have multiple ongoing work stream to increase trial enrollment our clinical operations team has been conducting on site visits to clinical trial sites around the globe.
Speaker Change: Additionally, we have been increasing our engagement with patient advocacy groups in the myelofibrosis disease space.
Speaker Change: I think it's important to note that we at Sharon as well as the impact of last trial investigators and also patient Africa continue to express excitement around the potential to extend survival and thus JAK inhibitor relapsed refractory population today.
Speaker Change: Today treatment in myelofibrosis is dominated by JAK inhibitors or therapies with other mechanisms of action in combination with JAK inhibitors.
Speaker Change: Once patients become unresponsive to JAK inhibitors, which leads to treatment discontinuation for approximately 75% of patients after five years.
Speaker Change: The assistance dismal overall survival of approximately 11 to 16 months.
Speaker Change: We believe that if our trial is successful and in the telecom is approved in this indication.
Speaker Change: Transform treatment for these patients with that I'll turn the call over to Michelle for a financial update Michelle.
Michelle J. Robertson: Thanks, Jay and.
Michelle J. Robertson: And good morning, everyone for details Q1, 2024 financial please refer to the press release, we issued this morning, which is available on our website.
Michelle J. Robertson: Now let me bring your attention to a few highlights from the quarter.
Michelle J. Robertson: As of March 31, 2024, the company had approximately $465 million in cash and marketable securities, including proceeds from an underwritten public offering of common stock in a pre funded warrant in March 2024 for net proceeds of approximately $141 million.
Michelle J. Robertson: Total operating expenses for the first quarter of 2024 was $56 4 million compared to $40 1 million for the same period in 2023.
Michelle J. Robertson: Research and development expenses for the first quarter of 2024 for $29 4 million compared to $27 2 million for the same period in 2023.
Michelle J. Robertson: The increase year over year, primarily reflects higher CMC costs due to the timing of <unk> commercial manufacturing batches and increased personnel related expenses for additional headcount.
Michelle J. Robertson: G&A expenses for the first quarter of 2024, or $27 1 million compared to $12 9 million for the same period in 2023.
Michelle J. Robertson: The increase primarily reflects our investment in commercial preparatory activities and higher personnel related expenses for additional head count as we have been preparing to transition from a clinical to commercial stage company.
Michelle J. Robertson: As of March 31, 2024, and prior to adding our Salesforce in April we had 162 full time employees.
Michelle J. Robertson: Object to approval of Intelsat in the U S. We plan to grow to a total of approximately 250 to 300 in place by year end 2024.
Michelle J. Robertson: Our projected full year 2024 operating expenses are expected to be between 270 and $280 million.
Michelle J. Robertson: Based on our current operating plans and our assumptions regarding the timing of a potential approval and commercial launch of Intelsat and TD LR Mds in the U S. We believe that our existing cash cash equivalents and current and non current marketable securities together with our projected revenues from U S sales within the pulse that if approved potential pro.
Michelle J. Robertson: From the exercise of our warrants and future drawdowns under our loan facility will be sufficient to support our operations into the second quarter of 2026.
Michelle J. Robertson: I will now turn the call back over to chip.
Chip: Thanks Michelle.
Chip: We believe <unk> has the potential to offer a life changing treatment option.
Chip: For patients with transfusion dependent lower risk Mds building.
Chip: Building on the momentum from the resoundingly positive low deck.
Chip: Deeply energized and well prepared to launch in the <unk> 70.
Chip: If approved.
Chip: Driven by our experienced leaders and expected excellent execution, we feel confident in our ability to capitalize on this robust opportunity.
Chip: And to transform <unk> into a successful commercial company.
Chip: We believe this transformation will create significant value for patients and shareholders.
Speaker Change: Let's now open line for questions operator.
Speaker Change: Thank you the floor is now open for questions. If you have dialed in and we'd like to ask a question. Please press star one on your telephone keypad to raise your hand and joined the queue.
Speaker Change: If you would like to withdraw your question simply press Star one again.
Speaker Change: Called upon to ask a question and are listening via loud speaker on your device. Please pickup your handset to ensure that your phone is not on mute when asking your question.
Speaker Change: Your first question comes from the line of.
Speaker Change: Tara Bancroft of TD Cowen Your line is open.
Tara Bancroft: Hi, good morning, and thanks, so much for taking the questions. So mine is regarding the myelofibrosis trial timeline can you comment on if it was more so due to slower enrollment or if there was a heavier impact from having fewer events than anticipated and if it's the latter can you remind us.
Tara Bancroft: Of the bar here and maybe if your expectations have changed for where OS could land for the control like how should we think about expectations here. Thanks.
Speaker Change: Sorry go ahead and take that please.
Speaker Change: Okay.
Speaker Change: Hey are you on mute.
Speaker Change: Oh I apologize issues.
Speaker Change: Part of my comments, the enrolment and death rates are both lower than anticipated.
Speaker Change: We are not able to provide additional detail into which ones.
Speaker Change: Okay.
Speaker Change: The driving force and as we noted that.
Speaker Change: Question is the timelines out about.
Speaker Change: Six months.
Speaker Change: We did see continued enthusiasm for the study and we will continue to monitor and update as appropriate.
Speaker Change: Okay. Thank you.
Speaker Change: Thanks Darren.
Speaker Change: Your next question comes from the line of Corinne Johnson of Goldman Sachs. Your line is open.
Corinne Johnson: Good morning. This is Craig on Kirker and <unk>. So first one from US have you all initiated labeling discussions with the FDA, the Mattel, Scott and lower risk Myelodysplastic syndrome.
Speaker Change: Yes, so we've received.
Corinne Johnson: Craig.
Corinne Johnson: We've received comments from the FDA on our on our draft label.
Corinne Johnson: However.
Corinne Johnson: As you would imagine the FDA hasnt yet completed its review.
Corinne Johnson: So we certainly look forward to continuing our conversations.
Corinne Johnson: With the agency as we approached producer, but we do not expect to publicly disclose or discuss any ongoing regulatory interactions with the division prior to private equity.
Speaker Change: Got it that makes a lot of sense and it seems like.
Speaker Change: Unlike the commercial standpoint, and clinical standpoint.
Speaker Change: And CCR and guidelines updated is a top priority. So how quickly do you think that could occur following the potential approval of Intelsat.
Speaker Change: Neil why don't you take that one.
Neil: Sure. So Craig we are able to provide an official submission to end CCN upon approval.
Neil: We are ready to do so this obviously includes the approved prescribing information or the label.
Neil: It includes the peer reviewed publication landscape, which exists and it includes our ACA related highlighting the patient populations.
Neil: Typically MCC in a bid to the guidelines within two to three months.
Neil: For new drugs that are received for low risk Mds within the NCC in low risk Mds community. So our expectation is.
Neil: If see submit in.
Neil: June upon.
Neil: Sure.
Neil: About two to three months later, we would expect to see the guidelines reflect images.
Speaker Change: Got it thank you so much.
Speaker Change: Your next question comes from the line of Gil Blum of Needham <unk> Company. Your line is open.
Gil Joseph Blum: Hi, good morning, everyone and thanks for taking my questions, maybe a slightly different tack on myelofibrosis.
Gil Joseph Blum: So.
Gil Joseph Blum: Were there changes to the standard of care over the last couple of years that may explain some of this slower.
Speaker Change: Accumulation and secondly.
Gil Joseph Blum: Can you comment on what the challenges are for enrollment there is competition from commercial treatments. Other studies whatever information you could provide thank you.
Gil Joseph Blum: Ill, let Fay take it first and.
Fay: I'll add.
Fay: Please go ahead Sir.
Fay: Thanks for the question Gil.
Fay: Putting a German indeed, we've had the approval of <unk>.
Speaker Change: A few.
Fay: A few more JAK inhibitors over the last couple of years.
Fay: I do believe that is impacted.
Fay: Enrollment rate.
Fay: [laughter].
Fay: And but it's multifactorial, including the new approvals of JAK inhibitors, the lack of JAK inhibitor treatment option in the <unk> arm and even still challenges of resourcing issues that site.
Fay: <unk>.
Fay: And staffing.
Fay: No.
Fay: All of these contribute you.
Fay: The lower enrollment rates.
Fay: That we predicted than.
Fay: And then pretty soon but we listen.
Fay: Not unanticipated and can be seen in minimum funding.
Fay: Okay.
Speaker Change: Alright, thanks for the color.
Fay: Okay.
Speaker Change: Thanks, Kevin.
Speaker Change: Again, if you would like to ask a question Press Star then followed by the number one on your telephone keypad. Your next question comes from the line of Stephen Willey of Stifel. Your line is open.
Stephen Douglas Willey: Hi, Good morning, guys. This is totally on for Steve. Thank you for taking my questions.
Stephen Douglas Willey: Congrats on the progress.
Stephen Douglas Willey: We have just two questions on our end the first one is.
Stephen Douglas Willey: Regarding the launch progression process for that coming.
Stephen Douglas Willey: <unk>.
Stephen Douglas Willey: So I mean like you definitely provided at least of things that you guys have accomplished so far so in terms of preparation what are the major steps that need to be done and maybe like additional color on that and the second question is related to.
Stephen Douglas Willey: Just to follow up on the prior question regarding MF trial.
Stephen Douglas Willey: <unk> is there so would it be possible to provide any like number or percentage.
Stephen Douglas Willey: Like percentage wise in terms of like our enrolment rate and.
Stephen Douglas Willey: And also like what are the debt that you guys are actually taking in order to expedite the enrollment and what is your confidence level that you guys will not needs to actually extend the extend the timelines for interim and final analysis. Thank you.
Stephen Douglas Willey: Let's take the first question on the launch preparations and Neil will we will.
Stephen Douglas Willey: That one and then <unk>.
Stephen Douglas Willey: Pivot over after that to your follow up question on the MF trial.
Neil: Go ahead sure sure. So thanks for the question.
Neil: A really important step for us is the completion of.
Neil: Onboarding of our full commercial team as we stated on our call.
Neil: Key account managers in the field are also now fully on boarded.
Neil: And we are working very hard towards.
Stephen Douglas Willey: Our potential approval in June.
Stephen Douglas Willey: Given our Peru project.
Stephen Douglas Willey: Our concentration is bolt on ensuring that we have for the commercial supply our distribution focus will be finalized.
Stephen Douglas Willey: Are preparing all the materials to inform stakeholders, including payers prescribers and.
Stephen Douglas Willey: Populations that we interact with we have identified the prescriber base, we are working towards our access and affordability solutions everything remains on track and our expectation is.
Stephen Douglas Willey: Well executed launch bottom or does it start in the U S market upon approval.
Stephen Douglas Willey: This block to Neil and.
Speaker Change: Hey, why don't you take the question on.
Neil: On the follow up question on the MF.
Neil: Thanks for the question and I'm going to try to remember all of the components.
Neil: Regarding the enrollment rate, we announced last year in November that the study was 50% enrolled and we plan to provide updates.
Stephen Douglas Willey: Hum.
Stephen Douglas Willey: Uh huh.
Stephen Douglas Willey: Ah.
Stephen Douglas Willey: And enrollment is continuing at.
Stephen Douglas Willey: I just added 30 P M.
Stephen Douglas Willey: As I mentioned before enthusiasm has not really diminished with the study.
Stephen Douglas Willey: So we continue to see movement Hollywood.
Speaker Change: Remind me the other question.
Speaker Change: I can take it.
Speaker Change: I think that.
Speaker Change: Yes, I think what she was.
Speaker Change: Getting at was sort of the.
Speaker Change: [noise] issue.
Speaker Change: How we would.
Speaker Change: Yeah.
Speaker Change: Follow all of this going forward and might it be necessary to to refine further.
Speaker Change: Look I think they said in the prepared comments that these estimates are really variable.
Speaker Change: Pardon me.
Speaker Change: And pretty difficult because you have two variables right you have an enrollment rate, which.
Speaker Change: I.
Speaker Change: Really cannot be seen as the only contributor to the timelines here and where.
Speaker Change: As you heard we're doing everything we can to keep that enrollment rate up and say commented in answer to a previous question.
Speaker Change: Whether there was that Gil would ask about <unk>.
Speaker Change: Commercial launches of other competing Jack products.
Speaker Change: You mentioned, the fact that the.
Speaker Change: But we don't have a JAK inhibitor in the in the arm.
Speaker Change: And so forth.
Speaker Change: But I think the other side is the pace at which we accrue.
Speaker Change: You bet right does events and Thats a loss harder to predict.
Speaker Change: Predict and I'll, just simply say, because we do not and have not.
Speaker Change: Unblinded the study of course, it's.
Speaker Change: Important to note all we can look at sort of the pace of those events and I will remind everybody that the study is a two for one randomization. So.
Speaker Change: I think we.
Speaker Change: We made the comment in the prepared remarks that we will have to follow along and yes things require further updates either we go slower.
Speaker Change: Towards the.
Speaker Change: The interim and final analysis or we go faster, we will certainly need to update but we don't make a specific commitment on exactly when we would do that.
Speaker Change: So I hope we.
Speaker Change: We will continue to monitor both enrollment and event rates and update guidance as required. So I hope that answers. Your question happy to take a follow on if needed.
Speaker Change: Thank you.
Speaker Change: I also believe there is a component to the question about what we're doing to them.
Speaker Change: Enhanced enrollment and I'll just speak quickly about that that we are.
Speaker Change: Putting a lot of efforts to meeting with investigators and.
Speaker Change: Country and thought leaders.
Speaker Change: Specially.
Speaker Change: On a one to one level with geron.
Speaker Change: Darren.
Speaker Change: Garen operations and clinical development staff as well as frequent virtual meetings.
Speaker Change: It virtually I guess, you can call them like investigator type meeting too.
Speaker Change: Remind investigators about the study and to hear.
Speaker Change: Hear any feedback.
Speaker Change: On enrollment we also.
Speaker Change: With our medical affairs team has been engaging more aggressively with.
Speaker Change: Myelofibrosis advocacy groups.
Speaker Change: In the U S and internationally.
Speaker Change: Thanks, that's all.
Speaker Change: Your next question comes from the line of Joel Beatty of Baird. Your line is open.
Joel Lawrence Beatty: Hi, Thanks for taking the questions first one is what percent of less fess up users currently.
Joel Lawrence Beatty: Positive compared to Rs negative and would you expect a similar split for <unk>.
Joel Lawrence Beatty: Neil.
Neil: Sure so we.
Neil: We don't have syndicated data to answer. This question. What we do have is public statements from the company's stating that an artist positive. They are very well penetrated in the numbers quoted are in excess of 65% from public statements from Bristol Myers Squibb.
Neil: Borgata is positive does that answer your question.
Speaker Change: Yeah. That's helpful. Thank you and then.
Speaker Change: And the last question.
Neil: Is there a kind of a specific definition of Esa failure that might prompt starting in <unk> or is there some clinical judgment involved.
Neil: Wonder if patients go through a period of time in which they are like partially responding to Esa.
Neil: I guess I'll take after the clinical trial, we had a specific definition.
Neil: And what will be on the label remains to be seen but it will be.
Neil: According to the investigator.
Speaker Change: Sorry physician.
Neil: That's happening.
Neil: Yeah.
Speaker Change: Great. Thank you Neil any further comments sorry go ahead.
Speaker Change: Yes.
Speaker Change: Joel I also will point you to.
Speaker Change: Hard deck that we're using for the call because we have recently completed.
Joel Lawrence Beatty: A survey of U S physicians 50 of them, both academic and community and if you look.
Joel Lawrence Beatty: Are there expectations for a metal start.
Joel Lawrence Beatty: We see ourselves being extremely well positioned across all of the patient segments that we serve in particular, what is highlighted from physicians and very favorably received by them.
Joel Lawrence Beatty: For them it does start as the population because Esa ineligible in the frontline setting.
Joel Lawrence Beatty: Patients who are Rs negative patients.
Joel Lawrence Beatty: Irrespective of either prior treatment with <unk> audio assays and I think these other places that we are extremely differentiator and are serving a very high level of unmet need.
Speaker Change: Your next question comes from the line of <unk> <unk> of B Riley Securities. Your line is open.
Speaker Change: Yeah, Hey, good morning, and thanks for taking the questions.
Speaker Change: I think chip you've previously said that you would make the patent lifecycle decision.
Speaker Change: For applying the PPE to the method of use patents around the time of FDA approval.
Speaker Change: The question is is.
Speaker Change: Is that guidance unchanged today or does that delay in the MF trial readout sort of impact the thinking behind the patent strategy.
Chip: Great question. Thanks Calvin.
Chip: Thank the.
Speaker Change: The specifics of how.
Speaker Change: Application for Pte works is a little bit above my pay grade, but I'll give you my understanding of it today.
Speaker Change: The best I can.
Speaker Change: After you have your first approval you can consider.
Speaker Change: Whether you have had a clear strategic idea of where you would like to apply PPE.
Speaker Change: And right now all of that is pointing towards the method of use patents. The reason for that is fairly straightforward as many people in the investment community have pointed out many and you have pointed out in your coverage of us.
Speaker Change: We're covered quite well with the with the orphan drug exclusivity.
Speaker Change: And you can apply and PPE.
Speaker Change: Comed patent wouldn't really Trump that and so there's every reason to imagine putting the pte onto the Mou we've done a lot of consideration and working on this and at the end of the day, we have to indicate to FTA win.
Speaker Change: At some point after approval, what we want to do and they will go and provide us with estimates of the exact timeframes and so forth, there's kind of a process there.
Speaker Change: Assuming all.
Speaker Change: Assuming all goes well there.
Speaker Change: I think it's important to say that.
Speaker Change: The X we believe from our analysis of this.
Speaker Change: Our outside firms analysis of this that exclusivity would be extended to 2037.
Speaker Change: And it would very importantly for.
Speaker Change: Method of use patents would apply the pte would apply to all use is covered by the patent including both of them to get to that math.
Speaker Change: So a lot of incentives to to apply it to the methods of use claim and.
Speaker Change: We'll take that up obviously the implications come much.
Speaker Change: Much later, but I don't know of any reason that we should not indicate that thats are our current expectations.
Speaker Change: Okay.
Speaker Change: Thank you and we've been getting questions on.
Speaker Change: The manufacturing in CMC front, given what's happening in the industry.
Speaker Change: I guess, if you can comment.
Speaker Change: It would be inspections and everything in that front go okay.
Speaker Change: And then I have a follow up.
Speaker Change: Yes.
Speaker Change: I think we're not going to provide specific.
Speaker Change: A follow up there but.
Speaker Change: I think that we are comfortable.
Speaker Change: As things.
Speaker Change: Have proceeded to date.
Speaker Change: Sure.
Speaker Change: Still looking forward to it could do to.
Speaker Change: Date with an approval and that's the best I can really say, we all know there are.
Speaker Change: Theres always some degree of uncertainty, but other than that I don't think were making specific comments about seats.
Speaker Change: Piece by piece March towards could do for you on any of the different fronts, including manufacturing inspections.
Speaker Change: Okay, Okay and then.
Speaker Change: Last one.
Speaker Change: Getting questions on how we should be modeling the U S launch.
Speaker Change: The initial ramp.
Speaker Change: Yes.
Speaker Change: Maybe one for Aneel are there any good examples are analogs.
Aneel: Mr Community can use to kind of projected at least for the initial ramp for the first year or two.
Aneel: <unk> was obviously approved a few years ago, but that market wasn't built at that time. So people are curious how they should model does now.
Speaker Change: Yeah. So.
Speaker Change: I think the.
Speaker Change: The best guidance I would give us to focus on unmet need of this patient population, what we have heard very clearly in the lower risk Mds market.
Speaker Change: There is dissatisfaction predominantly along three segments one is.
Speaker Change: The need for better more effective products for patients who are at high transfusion burden Richard the main part of your 50% of this marketplace.
Speaker Change: Auctions for Rs negative patients, who today do not have effective therapies and patients obviously, who are ineligible for Esa is because their outcomes of Purdue.
Speaker Change: So our expectation is that.
Speaker Change: Those are three patient populations with physicians.
Speaker Change: We'll use drugs such as ours, we also know that as <unk> said.
Speaker Change: <unk> has been in this marketplace for the last two to three years. So we would expect.
Speaker Change: Expect to see patients who have been treated with cytosorb and physicians are looking for more options.
Speaker Change: <unk> phase II study in RCC part.
Speaker Change: Part of this study covered the decider set pre treated patients and given uniqueness of our mechanism. We would expect to be effective in that population as well. So I think it's a mix of these patients it's hard to point to one effective analog because there are many characteristics rich make each and every one of these.
Speaker Change: Launches unique.
Speaker Change: But I do think the unmet need is very high our data remains highly differentiated and their dissatisfaction and the need for new treatments, we should see them or does it start well positioned.
Speaker Change: And we are doctor, but then dominant area and with the physicians as they feed their low risk Mds patients.
Speaker Change: Okay.
Speaker Change: Okay. Thank you very much for taking the questions.
Speaker Change: Thanks Kelvin.
Speaker Change: That concludes our Q&A session I will now turn the conference back over to Aaron <unk> for closing remarks.
Aaron: Thanks, so much to everyone for joining us today. We appreciate your interest in <unk> and look forward to keeping you updated during this very exciting time for our company halfway back why not break line.
Speaker Change: This concludes today's conference call you may now disconnect.
Speaker Change: Okay.
Speaker Change: Yeah.
Speaker Change:
Speaker Change: Yeah.