Q4 2024 Day One Biopharmaceuticals Inc Earnings Call
Thanks for watching!
Speaker Change: Hello, ladies and gentlemen, and welcome to the Day 1 Biopharmaceuticals 4th Quarter and Full Year 2024 Financial and Operating Results Conference Call.
At this time, all participants are in a listen-only mode.
Later, we will conduct a question and answer session.
Please be advised that this conference call is being recorded.
Speaker Change: I would now like to turn the call over to Joey Perrone, Senior Vice President of Finance and Investor Relations.
Please go ahead.
Speaker Change: Thank you. Hello, everyone and good afternoon. Welcome to day 1, 4th quarter and full year 2024 financial and operating results conference call. Earlier today, we issued a press release, which outlines the topics we plan to discuss today.
Speaker Change: You can access the press release and the slides to accompany this conference call on the Investors & Media section of our website at www.dayonebio.com. An audio webcast with the corresponding slides is also available on the website.
Speaker Change: Before we get started, I'd like to remind everyone that some of the statements that we make on this call and information presented in the slide deck include forward looking statements as outlined on slide 2.
Speaker Change: Actual events and results could differ materially from those expressed or implied by any forward-looking statements. We encourage you to review the various risks, uncertainties, and other factors included in our most recent filings with the SEC and any other future filings that we may make with the SEC.
Speaker Change: These forward-looking statements are based on our current estimates and various assumptions and reflect management's intentions, beliefs, and expectations about future events, strategies, competition, products and product candidates, operating plans, and performance.
Speaker Change: Today I'm joined by Dr. Jeremy Bender, Chief Executive Officer, Lauren Merendino, Chief Commercial Officer, Charles York, Chief Operating and Financial Officer, and Ellie Berry, Chief Medical Officer.
I will now turn the call over to Jeremy.
Speaker Change: Thank you, Joey, and good afternoon. I'm pleased to share our fourth quarter earnings and full year financial results with you today following an exceptional 2024 for day one.
Speaker Change: I'll also provide an update on the significant progress we've made towards delivering on our mission of developing new medicines for people of all ages with life-threatening diseases.
Speaker Change: 2024 was a landmark year for our company and the patients we serve.
Speaker Change: Our most important achievement for the year was the FDA approval of our first new medicine, Ogemda, in relapsed refractory pediatric low-grade glioma.
Speaker Change: OGEMDA's approval and launch are an important step forward for PLGG patients and for day one.
We believe Ogemda commercialization will drive durable, long-term value creation.
Speaker Change: Our accomplishments in 2024 extend beyond the approval and commercial launch of OGEMDA.
Speaker Change: We also advanced enrollment in our frontline FireFly 2 clinical trial of tovirafenib in pediatric low-grade glioma. We established an ex-U.S. partnership for Ojemda with Ipsin, and we in-licensed day 301. All of this was achieved while also strengthening our financial position.
Speaker Change: Gemda's launch performance in 2024 put us in a strong position to further advance day one's goals.
Speaker Change: Throughout the year we observed consistent growth in quarterly net OGEMDA revenues.
Speaker Change: For the full year 2024, net product revenues totaled more than $57 million. This represents approximately eight months of sales following Agenda's April approval.
Speaker Change: Fourth quarter net product revenues for $29 million which represents 44% growth versus Q3.
Speaker Change: New patient starts exceeded our expectations and have set the foundation for Agenda's future growth.
Speaker Change: We also achieved modest improvements in our gross-to-net deductions in Q4, driven by CMS agreeing that Ojemda is approved exclusively for pediatric indications.
Speaker Change: We'll provide more detail on that topic later in this call.
Speaker Change: Importantly, in July, we entered into an exclusive licensing arrangement with Ipsen for ex-U.S. rights to OGMDA, which, if approved by regulatory authorities, will help patients who are living with relapsed or refractory PLGG outside of the United States to gain access to OGMDA.
Speaker Change: We also advanced our pipeline in 2024 through the Firefly 2 trial and the acquisition of Day 301.
Speaker Change: Firefly 2, our Phase 3 frontline PLGG trial, continues to enroll. It is now open at more than 110 sites in the United States, Canada, Europe, Australia, and Asia.
Speaker Change: In January, we announced that we expected to be fully enrolled in the first half of 2026.
Speaker Change: We also expanded our pipeline with the acquisition of Day 301 in June 2024. Day 301 is a clinical stage antibody drug conjugate targeting PTK7 and solid tumors in both adult and pediatric patients.
Speaker Change: In January of this year we announced that we cleared the first dose cohort in that trial and we're now enrolling patients in the second dose cohort.
Speaker Change: We also strengthened our financial position ending 2024 with over $500 million in cash. This strong cash position provides us with the ability to operate, to invest, and to advance our pipeline, all critical components of growing our company.
Speaker Change: We entered 2025 in a strong position, and we have three priorities this year to take advantage of and strengthen that position.
Speaker Change: The first is to continue driving OJMDA revenue growth. As Lauren will share with you shortly, the opportunity to drive growth through expansion of the prescriber and patient base for OJMDA is significant.
Speaker Change: Our second priority is to continue advancing our pipeline, specifically enrollment in the Firefly 2 trial and dose escalation in the Day 301 Phase 1 trial.
Speaker Change: Our third priority is to continue expanding our pipeline with programs that have the potential to become important new medicines for patients.
Speaker Change: We remain focused on acquiring and in licensing programs that have the potential to be first or best in class, and we are in a strong financial position to do so without requiring external financing.
Speaker Change: Two critical capabilities have driven our success last year and in the years leading up to 2024. The first is our team's expertise in the development, registration, and commercialization of new oncology medicines.
Speaker Change: The second is our ability to identify and acquire new programs such as Day 301.
Speaker Change: We have both the capability and the capital to identify high-value opportunities that will help lead us through the next stage of the company's growth.
Speaker Change: I'll now turn the call over to Lauren to discuss our commercial progress in greater detail.
Lauren: Thank you, Jeremy, and good afternoon. I'm excited to share strong results from the fourth quarter and full year of 2024.
Lauren: In Q4, Ogemda Net Product Revenues grew to $29 million, resulting in $57.2 million for the eight months since launch.
Lauren: This represents 88% compounded quarterly net revenue growth for this period.
Lauren: This performance was driven by over 1600 cumulative total prescriptions with Q4 nearly doubling the number of prescriptions in Q2 and Q3 combined.
Lauren: This momentum underscores the robustness of our growth trajectory and the continued demand for OGEMDA.
Lauren: Quarter over quarter in 2024, we continue to show substantial growth in net product revenues.
In Q4, we grew sales by 44% versus Q3.
We attribute these impressive results to three key factors.
First, continued growth in new patient starts.
Lauren: In each quarter since launch, we have delivered double-digit growth in the number of non-EAP new patient starts, driven by an expansion in both breadth and depth of prescribing.
Secondly, high on-label patient continuations.
Lauren: Each month, a high percentage of OGMDA PLGG patients continue to go on to receive additional months of therapy.
Lauren: This is consistent with our expectations based on our experience in the Firefly 1 trial, where we saw a median duration of treatment of about 24 months.
Lauren: While some patients have discontinued treatment, it's important to note that 30 to 40% of the Ogemda discontinuations since launch have been in patients receiving Ogemda for non-promoted off-label uses, generally in faster-moving tumor types.
Lauren: The median duration of therapy among these patients has been about 4 to 5 months.
Lauren: Overall, we ended the year in a strong position with over 280 active patients on drug as we head into 2025.
Lauren: The third factor in our net revenue growth this quarter is an improvement in our gross-to-net due to CMS granting OGEMDA an exclusively pediatric designation, which you'll hear more about from Charles in a moment.
Speaker Change: Now let's look at the progress we've been making on breadth and depth of prescribing.
Lauren: The data on this slide represents our on-label, non-EAP new patient starts.
Lauren: On the left, you can see that quarter over quarter, we have made significant progress in increasing the number of accounts utilizing Ogemda.
Lauren: Each priority category accounts for about a third of US PLGG patients and we're pleased to report that a hundred percent of our highest volume accounts are now utilizing OGEMDA.
Lauren: On the right, you can see the continued uptake of accounts utilizing Ogemda and the expansion of use into multiple new patients.
Lauren: Almost half of accounts using OGEMDA have now prescribed it for two or more patients, including over 80% of our Priority 1 accounts.
Lauren: While we are pleased with the progress we've made to date, most importantly, this slide highlights the tremendous potential we have moving forward.
Lauren: There are still many Priority 2 and 3 accounts that have not yet prescribed OGEMDA.
These may be prescribers whose habits are more embedded.
Lauren: We are focused on increasing their comfort with Ogemda to encourage them to prescribe for their first patient.
The opportunity to drive depth is even more compelling.
Lauren: There are many more PLGG patients who may benefit from OGEMDA.
Lauren: As physicians are gaining experience with our product, their confidence is growing and they are identifying new patients to put on Agenda.
Lauren: We continue to expand physicians' thinking on patient types and create urgency to use OGMDA in the second line.
Lauren: While our sales and marketing teams have been focused on driving breadth and depth, our access team has done an excellent job of establishing coverage for Ogemda.
Lauren: With over 75 percent of covered lives now having published policies and about 95 percent of on-label patients receiving coverage,
We can now state unequivocally that OGEMDA has broad coverage.
Lauren: This has been a significant driver in our launch year as payers have been covering patients at high percentages since early in launch. And it will continue to be a significant driver as prescribers have an easier path to starting a patient on OGMDA instead of going through multiple appeals to get approval for an off-label treatment.
Lauren: We're proud to report that in Q4, over 80% of PLGG patients received coverage approval for Rogemda on their initial request. And on average, their drug was shipped less than seven days after their script was written.
Lauren: 2024 has been an amazing year for Day One and for Ogemda. Off of this strong foundation, we will build our success in 2025 by focusing in three key areas.
Lauren: First, as previously mentioned, it's important that we continue our focus on increasing breadth and depth of prescribers.
Lauren: There continues to be significant opportunity for us to increase the number of prescribers with OGEMDA experience and expand their use to a greater number of patients.
Lauren: We expect to continue steady growth as prescribers gain more experience with Ogemda.
Lauren: Second, we continue to aim to establish OGENDA as the standard of care in the treatment of relapsed or refractory BRAF altered PLGG.
Lauren: While many physicians' first experience with our product may have been in later lines, we continue to differentiate Ogemda and establish the rationale to use it earlier in treatment when patients are likely to receive the most benefit.
Lauren: Finally, we want to equip prescribers and patients with the tools and support they need to stay on agenda for as long as they see benefit.
Lauren: This means providing prescribers with more education and support as they gain experience and ensuring that our patient support materials and services continue to meet the patient and family needs.
Lauren: I'll now pass it over to Charles for more details on our financials.
Charles: Hello, everyone. Earlier today, we reported detailed fourth quarter and full year 2024 financial results in our earnings release.
Charles: 2024 was an exciting year filled with remarkable achievements that create clear momentum as we enter 2025, all with a view to drive and value creation.
Charles: We finished the year strong, delivering our best quarter of net product revenue results since launch. Net product revenue from sales of Agenda was $29 million, representing 44% growth compared to the third quarter.
Charles: Total revenue for the fourth quarter of 2024 was $29.2 million.
Charles: which is comprised of $29 million in net product revenue and $200,000 in license revenue resulting from our ex-U.S. commercial licensing agreement with Ibsen.
Charles: For the full year, total revenue was $131.2 million, including $57.2 million in net product revenue from sales of Agenda and $73.9 million in license revenue.
Charles: For the full year, we would like to highlight a couple of changes in our business.
Charles: As is customary with newly approved medicines, day one provided access to Agenda for patients beyond U.S. commercial availability through a named patient and compassionate use program.
Charles: Beginning in 2025, Ipsen, our ex-US commercial partner, has assumed primary responsibility for delivering these programs to patients. As such, we will no longer book revenue for our named patient program.
Charles: For context, we booked approximately $3 million of revenue for the Named Patient Program in 2024, spread about evenly over the third and fourth quarter that will not be recurring in 2025.
Charles: As Lauren mentioned earlier, in the fourth quarter, we delivered tangible improvement in our Gristin Act after CMS approved our request to designate OJMDA exclusively for pediatric indications, which resulted in reducing OJMDA's Medicaid and 340B minimum rebate percentage 600 basis points from 23.1% to 17.1%.
Charles: This is a meaningful improvement for day one, and based on our product mix and standard discounts attributable to our distribution model, we believe our forward-looking gross net will be approximately 12 to 15% for the foreseeable future, absent any change in our payer mix.
Charles: Our operating expenses were $91.6 million and $343.2 million for the fourth quarter and full year 2024, respectively, as compared to $59.5 million and $206.1 million for the same periods in 2023.
Charles: This increase relative to the fourth quarter of 2023 was driven primarily by commercial investments.
Charles: that supported the U.S. launch of OGEMDA, and a one-time charge of $20 million in R&D expense associated with the in-license of Day 301, our PTK-7 targeted ADC program, which recently cleared the first dose cohort in the Phase 1a dose escalation trial.
Charles: We fundamentally believe that our ability to maintain our strong financial position is key to building long-term, durable growth.
Charles: Our cash flow from operations, equity financings, and corporate development transactions in 2024 resulted in a cash balance of $531.7 million.
Charles: We maintain a focused capital allocation plan that allows us to invest in opportunities that support growth, while also prioritizing our current development plan, as we believe these programs provide the opportunities for future value creation.
Charles: Earlier today, Jeremy highlighted our internal expertise in oncology development, registration, and commercialization that we believe is second to none, and those attributes set us on a clear path forward in 2025 and beyond.
Charles: In closing, we are poised to execute on our goals in 2025 with great momentum, a strong foundation for continued growth, and a durable financial position.
Charles: We are excited to continue making your GEMDA accessible to those who can benefit from our medicine, and we are marching down the path of generating long-term, sustainable value.
Charles: Now we will turn it over to the operators for Q&A.
Charles: Thank you. Well, now we conduct a question and answer session.
Charles: If you would like to ask a question, please press star 1 on your telephone keypad.
Thank you. Our first question.
unknown: This is from Anupam Rama with J.P. Morgan. Please proceed with your question.
Speaker Change: Hi guys, this is Priyanka An for Anupam. Our question is when might you be in position to give guidance for Agenda? Thanks.
Speaker Change: Hi Priyanka, this is Jeremy. Thanks for the question. At this stage, the focus for us on determining when guidance will be available is about when we see consistency in both the growth of the revenue we see with OGEMDA as well as the duration of treatment that will take some time for us to
Speaker Change: to really understand, just given that we are still quite proximal to launch.
Thanks so much.
Our next question is from Andrea Newkirk with Goldman Sachs.
I already won. Thanks so much.
Yeah, thank you for the question.
So from a depth perspective
Speaker Change: Really the focus is, number one, physicians gaining further experience with Ogemda. We do believe that the product has a really compelling efficacy and safety profile that once they experience, they'll have an interest in utilizing it in more patients.
Speaker Change: But additionally, it's also about helping them understand the full range of patients that can benefit from Ogemda.
Speaker Change: As I mentioned in the earnings comments, oftentimes physicians start with a later line, more complex patient.
a patient who's maybe utilized.
Speaker Change: all the other therapies that are available. And they do that to get experience with Ogemda, but over time, they tend to get more confidence and use it in a broader patient type. So our sales team and marketing efforts will be focused on
Speaker Change: educating them on the full range of patients that can benefit from OGEMDA.
Speaker Change: And then to your question about, you know, balancing breadth of getting to those P3 accounts and depth. I'll just say our team has been focused on priorities 1 through 3 from the start.
Speaker Change: The priority three accounts, keep in mind, although collectively they account for about a third of PLGG patients.
Speaker Change: It's spread over a much larger number of accounts. So the number of patients that are managed by each account
Speaker Change: is smaller. So for some of these physicians it can take time before they have an opportunity, even once they believe in Ogemda and want to try Ogemda, it can just take some time before they have a treatment decision because they may be managing a smaller population of patients.
Speaker Change: So in general, our sales team will continue to be focused on priorities one through three accounts. It's just that the work they'll be doing at each individual account will differ based on where that physician's experience is so far.
Okay, thank you.
Speaker Change: Our next question is from Tower Bancroft with TD Cowan. Please proceed with your question.
Speaker Change: Hi, good afternoon, and thanks for taking the questions. So mine is.
Speaker Change: really about new patients. So how should we be thinking about the cadence of new patient starts this year and what are you thinking could lead to inflection or acceleration there or should we really be thinking about this as more linear growth this year? Thanks.
Speaker Change: Tara, thanks for the question. Lauren, can you take that one?
Lauren: Yeah, so as we've discussed previously, the pace of this disease is a little bit different than what you see in other oncology tumor types.
Speaker Change: And so even when a physician believes in a product, it can take time for them to have treatment decisions in order to increase their usage.
Speaker Change: So we have seen, you know, steady growth in our new patient starts and we anticipate, you know, continuing to see that. I don't envision a singular inflection point or a dramatic change in that. It's just going to be, you know, continued kind of steady growth.
Okay, perfect. Thanks so much.
Speaker Change: Our next question is from Joe Catanzaro with Piper Sandler. Please proceed with your question.
Joe Catanzaro: Hey everybody, thanks for taking my question. Maybe first one for Lauren and your comments around creating urgency around second-line usage.
Joe Catanzaro: Wondering if you think about more second-line usage of just providing a near-term benefit and creating a larger addressable pool of patients who could be on on GEMDO or is it more of a
Joe Catanzaro: potential longer-term benefit with the opportunity for those second-line patients that have a longer duration of Treatment any thoughts there would be helpful. Thanks
Joe Catanzaro: Yeah, thank you for the question. I think it's really a little of both, right? So if you have an earlier patient, they tend to be healthier, they tend to be starting off in a better place.
Joe Catanzaro: which can lead to improved duration of treatment. It also means that there tends to be more patients who have reached that point, right? So the answer is really both.
Joe Catanzaro: Okay, thanks. And I guess if I could ask a follow-up maybe on day 301. I know it's still the early days, but any expectations around sort of what would be the gating factor to sharing any initial clinical data for that program? Thanks.
Joe, thanks for the question.
Speaker Change: you know, the external world investors, you all and the physician community to understand our development plan and the underlying rationale for it. And for that reason, I anticipate that certainly we'll need additional.
Speaker Change: time and and data from the phase 1a portion of the dose escalation portion of the trial but but potentially also
Speaker Change: some dose expansion or dose cohort expansion data as well. And we haven't yet guided specifically around the timing of when those two data sets could be available.
Okay, got it. Thanks for taking my question.
Alec Stranahan: Our next question is from Alec Stranahan with Bank of America
Alec Stranahan: Hey, guys. Thanks for taking our questions. First, just on the 1,600 scripts written since launch, which is a great number, it would be helpful if you could sort of break down how many of those scripts were seen in 4Q alone, since I believe you had, I think, 619 quarterly scripts in 3Q and some in 2Q, too. It's roughly, you know, 750 quarterly scripts in 4Q.
Alec Stranahan: the right ballpark here and then any sort of seasonal impact or stocking to note as you close the year. Thanks.
I'll ask Lauren to answer both of those.
Alec Stranahan: Yeah, so from a total prescriptions perspective there were about 800 that were brought on in Q4.
And I'm sorry, your second question was...
Alec Stranahan: about stock, just on any seasonal impacts or stocking as you close the 2024.
Alec Stranahan: So, Charles, if you'd like to answer the stocking. Yeah. Hey, Alec. How you doing? So, this is a good question. Actually, pertinent and relevant to this quarter. In addition, the important things to note that
Alec Stranahan: have changed for this quarter are two things. One, we did have a noted gross net change in regards to the reduction of the gross net associated pediatric exclusivity.
Alec Stranahan: which does drive an increase in our overall revenue. We also, as you would anticipate, with a growing demand of patient scripts
Alec Stranahan: on a dollar value basis did increase the volume associated with channel stock, which does drive up
Alec Stranahan: The revenue for this quarter associated with that. Keep in mind, though, what we've targeted from an operational perspective is a two to four weeks of inventory on hand. We met that during this quarter and we anticipate that going forward, but we would expect not only this quarter, but future quarters as we grow Scripps.
Alec Stranahan: to have the absolute value and volume of channel stock to increase.
Okay, very helpful. Thank you.
Speaker Change: Our next question is from Amy Fadia with Needham & Company. Please proceed with your question.
and how many sales introductions does it take?
with these physicians for them to prescribe Agenda.
And a second question, could you provide an overview of...
Speaker Change: Day 301 and how the product is differentiated from the other PPEs.
targeting ABCs. Thank you.
Thank you.
Speaker Change: Thanks for the questions. Let me start with the second question and then I'll ask Lauren to answer the first couple of questions you had. So day 301 is, as you know, the PTK7 targeted ADC.
Speaker Change: The linker payload in this case are a polysaccharosine linker with exotecan as a payload. The drug antibody ratio for this program is
is eight and
ADC in the clinic.
Speaker Change: There is another program out there that targets PTK7 that is currently in the clinic that has an orostatin payload.
Speaker Change: and there are a couple of others that we're aware of that may enter the clinic soon that contain TOPA1 inhibitors. But we have what we see based on preclinical profiling and based on all public information, what we believe is a first and best-in-class opportunity.
Lauren: Let me turn it to Lauren to talk about the commercial topics you asked.
Lauren: Yeah, so first let me just say for those accounts who have not yet used Ogemda, they really fall into two categories.
Lauren: The first category is that they're interested in trying Ogemda but they're waiting for the right patient to need a treatment decision, right?
Lauren: So, as I mentioned earlier, your Priority 2 and your Priority 3 accounts have less frequent treatment decisions than, say, a Priority 1 account that has a higher volume of patients, right?
Lauren: So we have a number of physicians who have told us they're interested in trying Ogemda, but the first patient they want to try it in, they're waiting for that opportunity to try it. So that's one category.
Lauren: The other category are those physicians who are a little more hesitant. They're late adopters. They're more hesitant to try something new. And for those physicians, they oftentimes want to hear from their peer physicians, from KOLs, their experience with a new product first.
Lauren: So there's no magic number of calls that gets an account on board. It really is a very individualized.
Lauren: customers thinking is on our product, how narrow they may be defining the patient type and whether we can help them see a broader picture and in some cases it's it's a bit of a waiting game for the right patient to need treatment.
Understood. Thank you so much.
Lauren: On the guidance for the Fly or Flight 2 enrollment, could you provide some color on what assumptions you took into account regarding the percentage of prescribers?
Lauren: that are potentially using Agenda in the front line to, and then follow.
Lauren: A quick one on 301, given that this ADC targets a very unique pseudokinase, PTK7,
Lauren: I guess what gives you the best conviction that using an ADC for this target is the best approach over different types of modality given the very unique signaling? Thank you very much.
Lauren: Sure, thanks for the questions. On the Firefly 2 trial, in our
Lauren: The sites we have open today and the enrollment rate we're seeing in real time, both in the U.S. and outside of the U.S., predominantly outside of the U.S. as expected.
Lauren: and is really not influenced or takes into account any potential off-label use in the frontline setting in the U.S., which I would note has been very modest to date.
Lauren: Regarding day 301, our conviction in PTK7 as a target comes from some work that was done for a program called Cofetuzumab Pellidotin.
Lauren: mid-stage clinical trials, and for which there was substantial efficacy activity, albeit with a fairly low therapeutic index that we believe led that program to be discontinued. However, it did establish some proof of principle for PTK7 as a target that could be
Lauren: its properties, and in particular, the antibody and the polysarcosine exotecan payload together will lead to a better therapeutic index. That's what we've observed in the preclinical modeling and studies.
Lauren: And that, of course, is what we're looking to replicate in the clinic. But that cofetuzumab precedence and proof of concept was quite important for our confidence in that as an ADC target overall.
Speaker Change: Thank you. There are no further questions at this time. This does conclude our question and answer session and our call today.