Q4 2022 Geron Corp Earnings Call
Good afternoon, My name is Emma and I will be your conference operator today.
At this time I would like to welcome everyone to Geron Corporation fourth quarter and full year 2022 conference call.
All lines have been placed on mute to prevent any background noise. After the speakers' remarks, there will be a question and answer session.
I'd like to ask a question. During this time simply press star followed by the number one on your telephone keypad.
If you would like to withdraw your question again press Star one thank you.
Aaron Fangled VP of Investor Relations and corporate Communications you.
You may begin your conference.
Good afternoon, everyone welcome to the Geron Corporation fourth quarter and year end 2022 earnings Conference call I Am Erin find God Jones, Vice President of Investor Relations and corporate communications.
And today by the following members of your management team.
Dr. John Scarlett, Chairman and Chief Executive Officer, Olivia Bloom Executive Vice President and Chief Financial Officer, Dr. Fey seller Executive Vice President and Chief Medical Officer, and a New Yorker Port Executive Vice President of corporate strategy and Chief commercial officer before.
We begin 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 up in the Tulsa anticipated clinical and commercial events.
And really the timelines the sufficiency of John's financial resources and other statements that are not historical fact actual events or results could differ materially. Therefore, I refer you to the discussion under the heading risk factors in <unk> annual report on Form 10-K for the year ended December 31 2022.
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.
Please refer to the press release and slide deck for today's call under events in the investors and media section of our website at Www Dot you're on Dot com slash investors for our fourth quarter and annual 2020 financial results as well as business updates and expected upcoming key.
Milestones the agenda for today's conference call will be as follows chip will provide introductory remarks.
He will highlight key data from the recent top line results from emerge phase III and provide a status update on our regulatory plans. She will also discuss our other ongoing development programs.
Neil will discuss this year's planned commercial preparedness activities.
Yeah, well review fourth quarter and year end 2022 financial results financial guidance for 2023 and current capital resources.
And Chuck will provide concluding remarks before going to the Q&A session.
With that I will turn the call over to chip chip.
Thanks, Aaron good afternoon, everyone. Thanks for joining us today.
With positive topline results from our mortgage phase III in hand, we now have a chartered path towards several potentially significant regulatory and commercial catalysts, which we expect will further enhance the value of <unk>.
Building on the momentum created by the lower risk Mds results. We are also advancing our pipeline to expand the potential applications for Intelsat and telomerase inhibition, which we envision will establish <unk> as a leader in the treatment of hematologic malignancies.
As a first step in.
In the commercially attractive lower risk Mds indication, we remain on track to submit the NDA in mid 2023.
And to complete the MAA submission in the second half of 2023.
In parallel we're preparing for a potential U S. Commercial launch in the first half of 2024 and a potential EU launch by the end of 2024.
We recently expanded our capabilities to support the potential commercialization of <unk> with the hiring of seasoned professionals with deep operational and launch experience.
These individuals who will serve on our growing senior commercial leadership team.
<unk> expertise in trade and channel relations market access marketing medical affairs and sales.
We believe that adding these colleagues to our already highly talented employee base provides the expertise and experience necessary to prepare for the anticipated commercial launch and then that helps that.
A key differentiator and quality of Intelsat and its novel Telomerase inhibition mechanism of action is the potential to modify the course of the underlying hematologic malignancy.
The compelling phase III readout in lower risk Mds has now added to our robust body of evidence of disease modification.
And we believe our other clinical and research programs will further corroborate the potential meaningful clinical benefits with Intelsat treatments.
In our second <unk> phase III trial impact MF, a potential positive overall survival outcome could set the stage for changing the treatment landscape in myelofibrosis.
Based on our current planning assumptions for enrollment and event rates, we expect an interim analysis for OS and impact MF in 2024.
To further assess the potential of Intelsat and other hematologic malignancies, we are advancing development programs, which are evaluating <unk> in additional indications and in combination regimens.
We expect significant readouts from these programs over the next several years.
We have a strong balance sheet to fuel, our regulatory commercial and retail staff development activities.
On the heels of positive topline results from our emerge phase III study in low risk Mds, we raised over $210 million.
And net proceeds from high quality strategic investors in early January of this year.
Combining our year end cash balance with the net proceeds from the successful financing in January .
The warrant exercise proceeds received in the first two months of this year.
We accumulated total cash holdings in excess of $445 million.
We believe that these capital resources will be sufficient to fund our projected operating requirements through the end of the third quarter of 2025 supporting the achievement of many of the value creating catalysts we're pursuing.
In parallel with our continuing preparations to commercialize Intelsat, we continue to consider relationships with potential partners, including exploration of regional and other deal structures with possible mutual interests, which could potentially maximize the value of <unk> for patients and your own shareholders.
In summary, the commercial preparedness and clinical development activities planned in 2023 as well as potential upcoming catalysts are expected to position <unk> to become a leader in the treatment of heme malignancies.
Our strong financial position and highly capable employee base provides the foundation for John to become fully integrated commercial biotech company over the next 15 months.
With that let me hand, the call over to say Tyler our Chief Medical Officer.
Right.
Thank you chip and good afternoon to everyone on the call.
We're thrilled and very proud to report positive topline results from our emerge phase III study I didn't help that in lower risk Mds and the beginning of January .
On our January conference call, we presented detailed efficacy and safety results, which are available on the IR section of our website under presentations.
Today, I will provide a high level summary of those results, which we believe confirms a positive overall benefit risk profile for Intelsat.
We saw statistically significant and clinically meaningful improvements in the <unk> treated patients compared to the placebo control arm.
Specifically the trial met its primary endpoint of eight week transfusion independence as.
As well as the secondary 24 week Ti endpoint.
We saw substantial increases in both hemoglobin level and reductions in transfusions.
We also saw efficacy across Mds subtypes, including both Rs positive and Rs negative patients and irrespective of baseline transfusion burden or Ips fresh category.
The initial clinical and molecular evidence, we reported support Intel's Scotch potential for MTS disease modification and the safety results were consistent with prior Intelsat clinical experience.
The most frequent adverse events were neutropenia and thrombocytopenia.
You were manageable and reversible with the majority being resolved in less than four weeks.
Importantly, any serious clinical consequences from the Cytopenia with them has helped that treatment were observed at low rates and were similar to placebo.
We are planning to present and publish additional data and analyses from my emerge throughout 2023, as we have been collaborating with key opinion leaders to prepare these abstracts and discussing the topline results with providers. We have received excellent feedback on the readout as well as acknowledgement on the potential for <unk> that represent a meaningful.
Treatment advance and lower risk Mds.
Yes.
Based on these positive topline results from my emerge phase III, we continue to execute on our plan to submit an NDA in mid 2023.
Is that a request for a rolling submission was granted we have submitted the non clinical module of the NDA this quarter.
We remain on track with our plan to submit the remaining modules, including clinical and CMC by mid year.
Also this quarter, we participated in a standard pre NDA meeting with the FDA. There were no showstoppers at that meeting nor comments that changed our expected timeline of our fundamental strategy for the NDA submission.
When we submit the NDA, we plan to request priority review.
Allowed under our fast track designation in lower risk Mds.
If granted we expect FDA approval of the NDA.
And the U S commercial launch them it helps that in lower risk Mds could occur as early as the first quarter of 2024.
In addition, the MAA submission is planned by the end of 2023.
Typically the review time for an MAA is up to 14 months Chesapeake.
Thus, we expect the timing of potential approval of the MAA by the end of 2024.
Please note going forward, we don't plan to provide any details relating to regulatory interactions unless they result in a change to our submission strategy or expected tightening.
Moving onto our second phase III clinical program, let me outline our plans in 2023 for relapsed refractory MF.
<unk> is the first and only phase III MF trial with overall survival as the primary endpoint.
To trigger the interim analysis for the study death events need to occur in more than 35% of the total planned enrollment of 320 patients.
Thus the timing of the interim analysis depends on the number of enrolled patients as well as the rate at which death events occur.
Potentially the number of events required to conduct the interim analysis for this study could occur before enrollment is complete as these death events will accrue throughout the enrollment period.
Using current planning assumptions around enrollment and median OS estimates for each treatment arm.
We expect the interim analysis for impact MF.
In 2024.
These analyses are event driven and it is uncertain, whether actual rates for enrollment and death events will reflect current assumptions.
This quarter, we initiated several strategies to increase the rate of site activation and patient enrollment.
Including recruitment initiatives for patient matching and more engagement with investigators through onsite visits or interactions at medical meetings.
We will consider the impact of these initiatives over the next six months.
As well as evaluate the overall rates of enrollment and death events occurring in the study.
We expect to provide an update on the projected timing of the interim analysis after that evaluation.
If the improvement that was observed in <unk> treated patients and our embark phase III trial.
Can be confirmed.
Phase III impact MF trial and.
And we believe in the cost that will be strongly differentiated from other treatments and MF currently approved or in development.
Will likely change the treatment paradigm for relapsed refractory MF patients.
As a hematologist I know from personal experience.
<unk> consider O F of the ultimate measure of benefit for the treatment of their cancer patients. We expect our west would be especially relevant in the case of impact MF patients, who no longer respond to JAK inhibitors, and who due to their current dismal prognosis and limited treatment options are in desperate need for novel third.
P.
Beyond our phase III trials, we have several additional programs to potentially expand the treatment applications for amatol stop in hematologic malignancies.
In May 2022, we started improve MF.
Phase one study designed to evaluate the safety and clinical activity of <unk>.
That in combination with rux lit nib and patients with frontline MF.
The study design was informed by data from preclinical studies, describing the sequential treatment of rustling, followed by Amytal stat, how those selective inhibitory effect on malignant MF stem cells, while sparing normal hematopoietic stem cells.
To improve our math, we aim to determine the safety profile of the combination regimen of <unk> and <unk> as.
As well as explore any potential for disease modifying activity in frontline MF disease setting similar to what was observed with <unk> treatment in the phase two embark trial in a relapsed refractory myelofibrosis patient population.
Two of the three trial sites for the study are open for patient enrollment.
The remaining site is expected to open for enrollment in 2023, we also expect to present preliminary data from this study by the end of 2023.
Based on preclinical models showing in the Tulsa prevented expansion of human AML leukemic stem cells.
And prolonged survival in stem cells.
We are also supporting an investigator led study in relapsed refractory AML and higher risk Mds.
For patients who are already treated with a hypo map leading agent.
As noted on the slide this study called impress is evaluating <unk> as a single agent treatment in this severe disease. The first site is planned to open in 2023.
If the initial impress data show promise from Intelsat and higher risk Mds and AML, we expect to support another investigator led study evaluating the combination of <unk> plus other drugs that are part of the standard of care for such patients.
Moving onto our preclinical and research programs.
Slide provides a snapshot of the status of our lymphoid malignancies and next generation Ti programs.
In November 2022 early data was published from the preclinical program and lymphoid malignancies being conducted at MD Anderson Cancer Center.
Based on these early results we are continuing the collaboration to assess the potential therapeutic effect of <unk>.
<unk> sat in lymphoid malignancies, and expect further data by the end of 2023.
The objective of our discovery program is to identify a lead compound as a potential next generation oral telomerase inhibitor.
We continue to investigate various chemical entities as potential scaffolds.
We expect completion of the current discovery effort in 2023, upon which we plan to potentially advance <unk> compounds into the next step of discovery research and.
If successful these efforts would permit initiation of an IMD, enabling non clinical studies in the future.
I hope you can see the positive top line results in lower risk Mds have provided an impetus for the next step in and of itself that's development we.
We believe continued progress in the other areas I described such as relapsed refractory MF frontline MF higher risk Mds and AML.
Lymphoid malignancies, and the discovery of potential next generation <unk> inhibitors.
We'll explore however top that in kilometers inhibition may provide potential benefit to many more patients.
With that I will turn the call over to Neil to describe the planned activities around potential commercial launch of Intelsat.
Neil.
Thank you Phil and good afternoon, everyone.
Lower risk Mds is a highly attractive market with a significant addressable patient population.
Given the positive topline results announced earlier this year, we expect them or does it start to be highly differentiated and we adopted widely in the treatment of lower risk Mds.
We are taking important steps to ensure commercial readiness at launch.
As Jeff mentioned, we have made multiple senior commercial leadership hires with extensive industry knowledge launch and operational executions.
Now with that leadership team on boarded we continue to build out the commercial organization in a phased manner with.
With the goal of hiring talent with deep oncology in the U S market experience.
Our commercial go to market strategy.
It's around the following fundamental pillars that are focused on successfully transitioning geron.
<unk> stage company on ensuring a successful launch of <unk>.
First on the product front. In addition to our regulatory activities. We are focused on building, a comprehensive and integrated clinical and economic value proposition.
That concludes <unk> outlines and it all starts with benefits to providers clinics.
Hospital systems and pairs.
We expect this messaging to highlight and it then starts and differentiating qualities.
Being in the top line results, which include.
The broad efficacy across Mds subtypes, including both Rs positive and <unk> negative patients.
The durability of continuous transfusion independence.
The totality of clinical benefit which includes a reduction in transfusion burden and significant increases in hemoglobin levels.
Strong evidence of potential disease modification and what we believe is that favorable benefit risk profile.
Second we are focused on the long lead times and supply chain activities, including state licensing and third party logistics efforts to facilitate efficient distribution of it and start and smooth flow through the U S health care system.
A key focus for us is on broad engagement with our diverse set of stakeholders.
Including providers payers and advocacy groups.
If favorable reimbursement for them against that.
To engage with and educate providers beams.
Beams have planned extensive presence globally.
Global National and local hematology scientific meetings to raise awareness of the challenges facing patients with lower risk Mds.
We had also extensively engaged with bears and planning submissions often with dial star data to major societies, including in CCM and <unk>.
Part of their consideration for inclusion in guidelines.
In addition, we remain heavily engaged with patient advocacy groups.
Ford our efforts are also focused on jet orange organizational evolution to a commercial entity.
This effort involves building and expanding the functions and capabilities across geron, including information technology human resources finance and legal.
A form <unk> future growth and our commercial ambitions.
I look forward to providing updates on commercial activities throughout the year.
Now I'll turn the call over to Olivia for the financial update Olivia.
Thanks, Danielle and thanks to everyone on the call for joining us today.
Please refer to the press release, we issued this afternoon, which is available on our web site for detailed financial results.
As expected overall operating expenses for the fourth quarter and full year 2022 were higher than the same period in 2021.
Total operating expenses for the three and 12 months ended December 31, 2022 were $42 1 million and $139 $1 million, respectively, compared to $32 million and $115 4 million for the same period in 2020.
One.
The increase in research and development expenses, primarily reflects higher personnel related expenses for additional head count.
And increased consulting costs.
Related to preparation for top line results and regulatory submissions in low risk Mds.
The increase in general and administrative expenses, primarily reflects increased cost for commercial preparatory activities.
Higher personnel related expenses for additional head count.
And our portion of settlement cost.
Related to the class action and derivative lawsuits.
For fiscal year 2023.
We expect non-GAAP total expenses in the range of approximately $200 million to $210 million.
For fiscal year 2023 guidance.
Cost to support planned regulatory submissions in 2023.
Ongoing clinical trials emerge phase III impact MF.
Prove MF and impressed.
As well as preclinical studies and lymphoid malignancies and.
And discovery research for a next generation telomerase inhibitor.
Manufacturing commercial inventory for <unk>.
Preparations for potential U S commercial launch of Intelsat in low risk Mds.
And projected increases in head count as well as interest payments on outstanding debt.
The fiscal year 2023 financial guidance is based on a set of assumptions.
If those assumptions are updated later in the year due to changes in our plans, including in response to potential revised timing of FDA approval and potential U S. Commercial launch of <unk> in low risk Mds.
And we plan to update guidance at that time.
As of December 31, 2022, we had approximately $173 1 million in.
Cash cash equivalents restricted cash and current and non current marketable securities.
On January 10, 2023, we closed our underwritten public offering for net cash proceeds of approximately $213 3 million.
After deducting the underwriting discount and other offering expenses.
In addition.
In January and February 2023.
We have received $59 $8 million in cash proceeds from the exercise of outstanding warrants.
As a result, we.
The accumulated total cash holding in excess of $445 million.
Based on our current operating plan and our expectations regarding the timing of the submission.
And potential acceptance and approval of our planned NDA by the FDA.
And the potential commercialization in the U S for the use of <unk> in adult patients with lower risk Mds.
We believe that our existing capital resources will be sufficient to fund our projected operating requirements through the end of the third quarter of 2025.
Which includes potential you add commercial launch of Intelsat in low risk Mds and the first half of 2024.
With that I will now turn the call over to chip for closing remarks.
Thanks, Olivia John has made remarkable progress over the last few years and especially in the past six months.
We look forward to completing the regulatory approval and commercial launch activities now underway.
And hopefully to seeing <unk> become part of the standard of care in lower risk Mds.
We also expect that <unk> will potentially be become part of the standard of care in relapsed refractory myelofibrosis.
The impact MF study has a positive readout either at the interim analysis expected in 2024 or at the final analysis expected in 2025.
As we transition from a clinical stage to a commercial stage company, we have never been more excited by our vision of becoming a leader in the treatment of hematologic malignancies.
And in doing so positively impacting the lives of patients with these diseases.
Thank you for your continued interest and support of Intelsat, and Joanne and we'll be glad to take your questions.
Thank you.
As a reminder, if you would like to ask a question press star followed by the number one on your telephone keypad.
Pause for just a moment to compile the Q&A roster.
Your first question today comes from the line of <unk> Patel with B Riley. Your line is now open.
Hey, good afternoon, thanks for taking our questions.
And maybe one on the phase III impact MF study in relapsed refractory myelofibrosis can.
Can you give us a little more color on how the enrollment is progressing in that trial, maybe what percentage of patients have actually been enrolled if you have that info available.
Okay.
Okay.
Okay.
Hey, it's chip.
I think they will take most of the clinical questions go ahead Jay.
Thanks Kyle.
As we mentioned in the prepared remarks, we.
We still expect the interim analysis in 2024 based on our current planning assumptions.
And we will announce when we have reached 50% enrollment.
Okay.
Okay.
And then maybe a couple on the.
Earlier stage improve MF trial.
Are you planning to maybe release all of the data from part one.
But by the end of 2023, I believe you mentioned.
We're currently focused on.
Continuing to opening price and accruing patients.
We will.
We will open the third site.
By the end of the year and our will prevent data readout.
Guidance later on at that time.
Okay, Okay, and maybe one more question on that trial I think the protocol requires in part one.
Use of Russell, let Ned for at least 12 weeks prior to enrollment.
Does that in any sense impact how you're viewing the efficacy bar for this trial is open label study I'm, just trying to get a sense of it.
If it's fair to make comparisons to historical efficacy with Brooks.
Thanks for the question really the purpose of the trial to assess safety.
<unk>.
The lead in period for the rug slipping abuse.
Supported by preclinical data, we have showing sequential use approximately a bad name account that has the potential for more efficacy, but it also allows us.
Two enrolled patients on a stable dose of <unk> that we have a better understanding of any additional taxes.
<unk>.
Add in the Tulsa and the safety profile, one thing to talk to us about it so it's really much more.
More for a safety first.
Perspective.
Okay. That's helpful. Thanks, very much for taking the questions.
Thanks, Tom.
Your next question comes from the line of Stephen Willey with Stifel. Your line is now open.
Yes, thanks for taking the questions.
Can you talk about the site overlap between impact MF and high emerge and just whether or not you've seen any pickup in enrollment following the positive I emerge data disclosure earlier this year.
Obviously, you're right.
Yes.
Yeah. Thanks for the question.
We have seen.
After the release of the topline results for emerging half full.
Interest and impact on that and then improve on math and just.
General positive feedback.
Regarding both of the studies and.
Major medical.
Center, there may be overlap in the sites, but there is both our global study.
There are plenty of sites for both.
Okay and then.
Can you maybe just provide some additional color around.
Cadence of incremental high emerge data that we should expect to see over the course of.
This year and I guess should we expect the patient reported outcomes data to be included in the next in the next medical conference presentation.
Sure we do plan to present additional data at medical meetings and also <unk>.
<unk>.
Data for publication.
So we do expect to report.
Patient reported outcomes in the near future.
Okay.
And then maybe just lastly for Olivia.
You provided cash runway guidance through the third quarter of $25 does.
Does this guidance assume that the second tranche of warrants are also exercised I think that these get triggered on the basis of FDA accepting the NDA filing in low risk Mds.
Oh.
Steve as Olivia Thanks for the question no.
Guidance does not include that so it would extend the runway further if those proceeds could be received so the guidance to the end of third quarter 2025 is just what is currently on the balance sheet. After the financing and after what's been received so far from exercise of warrants.
Alright, great. Thanks for taking the questions.
Your next question comes from the line of Joel Beatty with Baird. Your line is now open.
Great. Thanks for taking my questions.
Are you able to share any feedback from FDA on their openness to filing with priorities with you.
Sure I'll take that I'll take that really quickly and then they can add if she has anything.
Joel It's Chuck.
FCA generally.
Does not give any insights and generally we can carefully ask.
Regarding insights into priority versus standard review.
Answer would almost always be that's a review issue.
As you know they first have to look at the NDA submission accepted for filing and that generally takes 60 days and then at the time that they accepted for filing if they do then thats when they tell us whether we will have a priority or standard review.
Set about at that time.
That I recall.
That we.
I expect to request priority review, which is based on.
On our.
Yes.
On our current regulatory designations with fast track and so forth. So that's pretty much all I know that we can say hey did I leave out anything from your perspective.
Okay. Thanks, Chad that was pretty comprehensive I don't have anything perhaps.
Okay.
Great. That's helpful and then maybe a question.
The emerge phase III data with payers are there any drug that come up is with regards to the value that's provided to patients.
Neil you want to address that.
Sure.
So.
The question around the fan feedback is extremely positive.
The drug start continue to come up.
Obviously that lodestone.
And Covid, but then the Nordic MBS landscape, but.
Sure.
Entire discussions have focused on our value proposition of.
Both the efficacy profile, the totality of benefit and what it could mean for the payers within their value proposition. So.
As of right now these discussions are extremely favorable in this new does begin to speak to that.
Great. Thank you.
Your next question comes from the line of Gil Blum with need him. Your line is now open.
Hi, everyone. Good afternoon, so maybe one for Neil how important do you think long transfusion independence data will be.
For effective payer coverage.
Do you expect longer Ti to matter.
Laura It appears I E waiting on additional emerge data to really have a <unk>.
<unk> picture. Thank you.
Thanks for the question I think the durability of the EIA data is extremely important.
Bolton.
U S as well as in Europe .
This point is repeatedly come up from payers as well as clinicians too.
Showcase their durability of Ti.
And the most important metric being measured from their perspective is the 24 week data.
We have one <unk> on top of that which is pretty unprecedented in this landscape.
Is extremely favorable.
To us.
In addition, the totality of benefit.
In terms of both reductions in transfusion burdens as well as.
The fact that we go across both subgroups all of these points are being extremely well received.
And we expect to be in a very favorable position as they move forward towards launch.
Thank you very helpful and a quick one for Olivia.
It seems like Youre going to have quite a significant cash burn this year I mean.
In my own calculations that I had.
Some of your clinical study costs going away. So how do you think division is going to be is this mostly opex go into SG&A and sales force ramp up thank you.
So Gil banks. So it is about a $60 million increase from the 22 guidance to where we are in 'twenty three.
So and that is driven by a number of categories. So first it would be.
Head count.
And that's about 40% of the increase.
Regulatory submission work.
And everything that goes along with that for both the U S and the EU that's another 10%.
Commercial manufacturing and building up the inventory of Intelsat getting ready for potential commercial launch that's another 20% and then in general other actually is related to commercial readiness, which is another 30%.
So I do want to let you know, though that overall the clinical costs, though still are being maintained in the company. Because obviously, we have another phase III trial going on impact on that as well as the other studies that Jim mentioned.
Great.
Until then thank you for that.
I'll jump back in the queue.
Yeah.
This concludes our Q&A for today I now turn the call back to Erin Feingold for closing remarks.
Thanks, so much everyone for joining us today, we appreciate you taking the time to listen and participate.
We look forward to sharing the achievement of several milestones in the coming year stay healthy and safe everyone right.
This concludes today's conference call. Thank you for attending you may now disconnect.
Please wait the conference will begin shortly.
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