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spk01: quarter and full year 2023 financial results conference call. At this time, all participants are in a listen-only mode. This call is being webcast live on the Investors and Media section of Cirrus website at www.cirrus.com. Please be advised that today's call is being recorded. At this time, I would like to turn the call over to Karen Hunadi, Director of Investor Relations and Corporate Communications at Cirrus.
spk00: Thank you. This morning, we issued a press release announcing our fourth quarter and full year 2023 financial results. The full release is available on the investor and media section of the Cirrus's website at www.cirrus.com. We will begin the call with prepared remarks by Conley Chee, our Chief Executive Officer, Dr. David Roth, our Chief Medical Officer, and Jason Haas, our Chief Financial Officer. We will then open the call for questions. Kristen Stevens, our Chief Development Officer, is also here on the call and will be available for Q&A. Before we begin, I would like to remind everyone that the statements we make on this conference call will include forward-looking statements. Actual events or results could differ materially from those expressed or implied by any forward-looking statements as a result of various risks, uncertainties, and other factors. including those set forth in the risk factors section of our annual report on Form 10-K that we filed this morning, and any other filings that we may make with the SEC in the future. Any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. We specifically disclaim any obligation to update or revise any forward-looking statements. With that, I would now like to turn the call over to Conley. Conley?
spk05: Thank you, Karen. Good morning, everyone, and thank you for joining us. Late last year, we announced the initial data from our Select AML-1 trial, and since then, we've continued to make great progress in our effort to become a commercial-stage biotech company. Following our initial AML data, we completed an equity financing of approximately $45 million, reinforcing our cash position and providing us ample resources as we enter into 2024. This year will be a critical one in our evolution, with expected pivotal data from our Select MDS-1 trial, as well as additional randomized data from our Select AML-1 trial. And we're excited to share with you that we've completed enrollment of the 190 patients necessary for our primary endpoint analysis in our Select MDS-1 Phase 3 trial. And we remain on track to report pivotal CR data by the middle of Q4 this year. If successful, these data will allow us to file our first MDA and ultimately to deliver Tamiberitone to the approximately 50% of higher risk MDS patients with RARA overexpression in need of better options. Just as a reminder, our strategy is to launch Tamiberitone in the US with our own specialty sales force. And we continue to make great progress against our launch plan. We look forward to sharing more details on our commercial plan as we get closer to our pivotal data readout. As we've discussed previously, we believe Tamibaratine has the potential to bring about a transformational change for these patients by offering a unique and targeted new standard of care for the frontline treatment of hematologic malignancies. Our growing body of evidence indicates that Tamibaratine consistently produces impressive response rates with a rapid time to response, and it's generally well tolerated. We're excited to push forward with our program, and we'll be sure to keep you up to date as we progress throughout the year. With that, I now turn the call over to David to review our programs and upcoming milestones in more detail. David?
spk03: Thank you, Conley. We are very encouraged by the development of Tamibaratine and the potential for our targeted agent to improve the frontline treatment of higher risk MDS and AML patients with RARA overexpression. As Conley mentioned, in December of last year, we got our first look at the initial data from the ongoing randomized portion of the Select AML-1 Phase II study. The objective of this study is to evaluate the safety and efficacy of the triplet regimen of tamibaratine in combination with venetoclax and azacitidine compared to venetoclax and azacitidine in approximately 80 patients randomized one-to-one. The initial data included 23 patients with 19 response available and demonstrated a 100% CR-CRI rate in patients treated with the triplet regimen of tamibaratine, venetoclax, and azacitidine as compared to 70% among patients treated with venetoclax and azacitidine alone. Importantly, 78% of the responses among patients treated with the triplet were complete responses, or CRs, compared to only 30% among patients treated with the venasa combination alone. The time to response was rapid across both arms, with 100% of patients in the triplet arm responding by the end of cycle one compared with 60% in the doublet arm. Not only was the AML data encouraging from an efficacy standpoint, but the safety profile was compelling as well. Consistent with prior clinical experience, tamibaratine in combination with approved doses of venetoclax and azacitidine was generally well tolerated, and the overall safety profile demonstrated no additive toxicities or new safety signals. Importantly, We also saw no evidence of increased myelosuppression in the triplet arm compared to the doublet. We're very encouraged by these initial results, which strongly support the potential of tamibaratine in combination with standard of care in the frontline treatment of AML patients with RARA overexpression. And we look forward to sharing additional data later this year. We also believe the high CR rates in our AML study support the potential for tamibaratine to deliver complete responses in our ongoing SELECT-MDS-1 trial, which has a primary endpoint based on CR. Turning to MDS, our Phase III SELECT-MDS-1 trial is a randomized double-blind placebo-controlled trial evaluating the combination of tamibaratine and azacitidine versus placebo and azacitidine in newly diagnosed, higher-risk MDS patients with RARA overexpression. As Conley mentioned, We recently completed enrollment of the 190 patients necessary to support the complete response rate primary endpoint analysis. And we are on target to report pivotal data by mid-fourth quarter of this year. As a reminder, we're continuing to enroll patients in the trial to support the key secondary endpoint of overall survival. This approach allows us to potentially secure accelerated approval and subsequent conversion to full approval if needed. By integrating both primary and confirmatory endpoints into a single trial, we ensure we execute more efficiently and believe this increases the probability of success of the overall study. We believe Tamubarotene has the potential to be the first novel agent approved for the treatment of higher risk MDS in over a decade. In that time, the only approved therapies are hypomethylating agents, or HMAs, which provide limited efficacy of a 17% CR rate and a median overall survival of just 18.6 months, highlighting the critical need for new treatment options for this patient population. We look forward to delivering pivotal CR data by the mid-fourth quarter of this year and evaluating the potential of tamibaratine to meaningfully improve upon the standard of care and deliver improved treatment outcomes for patients with higher-risk MDS. I would now like to turn the call over to Jason to review our fourth quarter and full-year financial results. Jason?
spk06: Thank you, David. We continue to be well-capitalized to fund the ongoing development of Tamavera Team. In December 2023, we completed an equity financing which resulted in gross proceeds to Cirrus of approximately $45 million before underwriting discounts, commissions, and offering expenses. The financing included new and existing investors, including Bain Capital Life Sciences, Ciros co-founder and founding investor Flagship Pioneering, Adage Capital Partners, Invis, Samsara BioCapital, DeepTrack Capital, Blue Owl Healthcare Opportunities, Daphne Capital Management, as well as a life sciences-focused investment fund. We are grateful to our investors for their continued support. We believe our current cash position will be sufficient to fund our anticipated operating expenses and capital expenditure requirements into the second quarter of 2025 beyond our pivotal phase three data from the select MDS-1 trial and additional data from the randomized portion of the select AML-1 trial. Now turning to our fourth quarter and full year 2023 financial results. Revenues were $400,000 for the fourth quarter of 2023 and $9.9 million to the year ended December 31st, 2023, as compared to negative $800,000 in the fourth quarter of 2022 and $14.9 million to the year ended December 31, 2022. The increase for the fourth quarter of 2023 compared to the same period of 22 was driven primarily by the negative cumulative catch-up adjustments recognized in the fourth quarter of 2022. The decrease for the year reflects the termination of the collaboration agreement with Pfizer-GBT in October 2023. R&D expenses were $21.5 million for the fourth quarter of 2023 and $108.2 million for the full year 2023, as compared to $27.9 million for the fourth quarter of 22 and $111.9 million for the full year 2022. The decrease for the fourth quarter of 2023 compared to the same period in 2022 and the decrease for the year were primarily due to a reduction in employee-related expenses, consulting and professional fees, and other facilities-related costs. The decrease in these costs were driven by the restructuring of our operations to prioritize key development and pre-launch activities to advance Tamabera teams. G&A expenses were $5.9 million for the fourth quarter of 2023 and $28.3 million for the full year of 2023, as compared to $7.3 million for the fourth quarter of 2022 and $29.3 million for the full year of 2022. The decrease for the fourth quarter of 2023 compared to the same period in 2022 and the decrease for the year were primarily due to consulting and other professional fees and facility costs. we reported a net loss for the fourth quarter of $64.4 million or $2.18 per share compared to a net loss of $4.8 million or $0.17 per share for the same period in 2022. For the full year ended December 31st, 2023, Ciros reported a net loss of $164.6 million or $5.81 per share compared to a net loss of $94.7 million were $7.49 per share for the same period in 2022. Cash and cash equivalents as of December 31st, 2023 were $139.5 million as compared with cash, cash equivalents and marketable securities of $202.3 million at the end of 2022. With that, I will turn the call over to the operator for questions. Thank you.
spk01: Ladies and gentlemen, we will now begin the question and answer session. Should you have a question, please press star, followed by the number one on your touchtone phone. You will hear a three-tone prompt acknowledging your request. Should you wish to decline from the polling process, please press star, followed by the number two. If you are using a speakerphone, please lift your hands up before pressing any keys. Our first question comes from the line of Phil Nadeau from TD Callen. Please go ahead.
spk02: Good morning, congratulations on completion of enrollment, and thanks for taking our questions. A couple from us. First, in terms of the data that we're gonna see in Q4, can you discuss on the complete response endpoint, maybe in a bit more detail, your discussions with the regulatory agencies about how that could support an FDA filing, and how does duration of response factor into the regulatory authority's evaluation of the CR data?
spk03: Thanks, Phil, for that question. It's David here. And what I can say is that the complete response is a very important clinically meaningful endpoint because it's associated with hematologic improvement. And hematologic improvement is really the clinical outcome that reverses a lot of the side effects and complications of higher risk MDS. And that's why clinical complete responses are an important target clinical benefit, and a well-understood correlate to overall survival. So in that context, we chose the CR as the primary endpoint. We've had discussions with the FDA on more than one occasion where the nature of our primary endpoint was part of the dialogue. And we have received confirmation that the CR can support a regulatory decision as a primary endpoint for either a full approval or an accelerated approval And, of course, the durability of that remission is considered when they make that call. So, you know, we feel very confident that this is an important, you know, endpoint for us to focus on, and it enables us to deliver an approval in a meaningful timeframe. You know, so here we are. We've enrolled our 190 patients. We're continuing the study, as you all know, toward our key secondary endpoint, which requires 550. and we're well on our way to delivering the top line data in the middle of the fourth quarter.
spk02: In other trials in MDS or AML, the bar for duration of response seems to be somewhere in the five to six month range. Is that a reasonable expectation in select MDS1 as well, or are there other data points that we should be considering?
spk03: So, you're correct. The duration of response in some of the more recent approvals has been like in the five and a half month range over time that can increase with additional data and a longer term follow up. And I think really what determines a meaningful duration is one of those assessments that requires looking at the total data package. So, Obviously, a fleeting response that, you know, you can only measure once and then doesn't last, you know, would probably not be considered clinically important, whereas one that lasts over time, which provides the opportunity for benefit to the patient, would be. And so, I'm sure the agency will look at not only the duration, but, you know, the time to response, the quality of the response, you know, again, how durable it is, and then all of that in the context of the safety. So, you know, you need to really appreciate how much, you know, the safety signals could contribute or detract from that quality of the response. And that's where we also have a benefit, in our opinion, because tamibaratine has a generally well-tolerated safety profile. It's orally administered. And over many years, in many different studies, looking at it in different ways, we've really, you know, seen nothing of consequence with respect to the overall safety profile. And I believe that's where we hold a very special advantage for the opportunity for patients.
spk02: That's very helpful. And then one last question for Jason. Jason, based on your guidance for the cash runway into Q2 of 2025, it seems like the Q4 23 expenses in R&D and SG&A are a reasonable run rate for future quarters, at least through 2024. Is that a fair assessment or is there any lumpiness that I'm missing?
spk06: Yeah, I think it's fair. We've been, you know, kind of spending a little bit less on SG&A and R&D over the last couple of quarters as we've really prioritized and focused the programs on tannobarotene for MDS and AML. There's certainly some lumpiness, you know, depending upon, you know, some payables, you know, due to, you know, vendors along the way. But generally speaking, I think it's fair to say when you look at the Q4 numbers and you look at the cash that we spent, that allows us to get into the second quarter of 25.
spk02: That's very helpful. Thanks for taking our questions and congrats again on completion of enrollment.
spk01: Thank you. Ladies and gentlemen, just a reminder, should you have a question, please press star followed by the number one on your touchstone phone. We have our next question coming from the line of Jason Butler from Citizens JMP. Please go ahead.
spk04: Hi. Thanks for taking the question and congrats on the progress. I'm wondering if you could just talk about some of the medical affairs work and commercial prep that you're starting to do both for MDS and AML and just what your focus will be for the rest of 2024. Thanks.
spk03: Sure. Why don't I start off? Oh, okay. Yeah. Let me just start off by saying some of the medical affairs work that we're doing is really focused in the short term on key opinion leader engagement, meeting with various healthcare organizations, and making sure that the physicians who ultimately will be prescribing the drug truly understand how it works. They need to understand the thorough grounding in the biology and the data. So we're basically setting the stage for an appreciation of the importance of identifying patients who have RARA overexpression, and then being receptive to prescribing the drug upon success of the trial.
spk05: Jason, it's Conley here. I can add to that in terms of our launch prep. As David said, the first phase of this was really around education over expression and some awareness of county barotin, but also the launch team has done a great job in terms of laying the path, if you will, to launch, which includes investments in infrastructure and starting to look at the types of investments we'll need, most of which will be gated post-data. But I think we're in great shape in terms of envisioning what we're going to need to make it a really great launch for kind of our team.
spk04: Great. Thanks for taking the question.
spk01: Thank you. There are no further questions at this time. I'd now like to turn the call back over to Mr. Chee for final closing comments.
spk05: Thank you, Operator, and thank you, everyone, for joining us today. We're looking forward to an exciting and productive year ahead and appreciate your continued support of Cirrus. Please reach out to us if you have any further questions and have a great day.
spk01: Thank you, sir. Ladies and gentlemen, this concludes your conference call for today. We thank you for participating and ask that you please disconnect your lines. Have a lovely day.
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