Q1 2024 Viridian Therapeutics Inc Earnings Call
Speaker Change: [music].
Welcome to the Viridian Therapeutics first quarter 2024 conference call.
Operator: Welcome to the Viridian Therapeutics first quarter 2024 conference call. At this time, all participants are in a listen-only mode. Later, we will conduct a question and answer session, and instructions will be given at that time. As a reminder, this conference call is being recorded. I would now like to hand the call over to Ms. Louisa Stone, Manager of Investor Relations at Viridian. Please go ahead.
At this time all participants are in a listen only mode.
Later, we will conduct a question and answer session and instructions will be given at that time.
As a reminder, this conference call is being recorded.
I would now like to hand, the call over to MS. Louisa Stone manager of Investor Relations at Meridian. Please go ahead.
Louisa Stone: Thank you and welcome everyone to our first quarter 2024 earnings conference call. The press release reporting our financial results and corporate updates is available on the investors page of our corporate website at www.viridiantherapeutics.com. Joining me on the call this morning are Steve Mahoney, our President and CEO, Tom Shula, our Chief Medical Officer, and Shan Wu, our Chief Business Officer. Before we begin, I would like to remind everyone that this conference call and webcast will contain forward-looking statements regarding our financial outlook, in addition to regulatory product development and commercialization plans and research activities.
Louisa Stone: Thank you and welcome everyone to her for first quarter 'twenty 'twenty four earnings conference call. The press release reporting our financial results and corporate update is available on the investors page of our corporate website at Www Dot Meridian Therapeutics dotcom.
Louisa Stone: Joining me on the call. This morning are Steve Mahoney, our president and CEO, Tom Ciulla, Our Chief Medical Officer, and Sean Nguyen, Our Chief business Officer.
Louisa Stone: Before we begin I would like to remind everyone that this conference call and webcast will contain forward looking statements regarding our financial outlook. In addition to regulatory product development and commercialization plans and research activities. These statements are subject to risks and uncertainties that could cause actual results to materially.
Louisa Stone: Differ from those forecasted a description of these risks can be found in our most recent Form 10-Q and 10-K on file with the S. E C.
Louisa Stone: These statements are subject to risks and uncertainties that could cause actual results to materially differ from those forecasted. A description of these risks can be found in our most recent Form 10-Q and 10-K on file with the SEC. I would now like to turn the call over to Steve Mahoney, our President and CEO.
Louisa Stone: I would now like to turn the call over to Steve Mahoney, our president and CEO.
Steve Mahoney: Thanks, Louisa and welcome everyone to our first quarter earnings call I'll start by giving a brief overview of Radian and then we'll get into more detail about our programs and recent progress. So those of you who are new to the viridian story, our strategy is to identify market opportunities, where there's a clear unmet need.
Steve Mahoney: Thanks, Louisa, and welcome everyone to our first quarter earnings call. I'll start by giving a brief overview of Veridian, and then we'll get into more detail about our programs and recent progress. For those of you who are new to the Veridian story, our strategy is to identify market opportunities where there is a clear unmet need and where there is potential for us to develop differentiated products. We then aim to engineer the best possible therapeutics and then move rapidly to advance our programs to patients.
Steve Mahoney: There is potential for us to develop differentiated products.
Steve Mahoney: We then aim to engineer the best possible Therapeutics, and then move rapidly to advance our programs to patients.
Steve Mahoney: Turning to slide four slide four shows our pipeline, which includes both a thyroid eye disease or Ted portfolio as well as in our CRM targeting autoimmune portfolio.
Steve Mahoney: Turning to slide four, slide four shows our pipeline, which includes both a thyroid eye disease or TED portfolio, as well as an FCRN targeting autoimmune portfolio. We have several exciting updates to provide across our pipeline today, which we'll get into next. We're really excited to highlight for you today the significant progress that we've made across the business so far this year.
Steve Mahoney: We have several exciting updates to provide across our pipeline today, which we'll get into next.
Steve Mahoney: We're really excited to highlight for you today the significant progress that we've made across the business. So far this year.
Steve Mahoney: Beginning with our O01 IV program, we are pleased to announce that THRIVE, our Phase III trial evaluating O01 in patients with ACTIV-10, completed enrollment in March. In fact, Not Only did Thrive reach the target enrollment of 90 patients in mid-March, but we exceeded enrollment for a total of 113 patients due to strong patient demand at our clinical trial site. We expect to share top-line results for Thrive in September 2024.
Steve Mahoney: Beginning with our old one IV program, we are pleased to announce that thrive our phase III trial evaluating <unk> in patients with active Ted completed enrollment in March.
Steve Mahoney: In fact, not only can thrive reached the target enrollment of 90 patients in March in mid March, but we exceeded enrollment for a total of 113 patients due to strong patient demand at our clinical trial sites.
Steve Mahoney: We expect to share top line results for thrive in September 2024.
Steve Mahoney: Tried to our phase III trial, initiating or one IV in patients with chronic Ted continues enrolling trial is on track for topline data at the end of this year.
Steve Mahoney: Thrive 2, our Phase 3 trial initiating O01 IV in patients with chronic TED, continues to enroll and is on track for top-line data at the end of this year. Lastly, for OO1, we are announcing that we anticipate filing a BLA for the OO1 program in the second half of 2025 for our subcutaneous 003 program, which we believe is substantially the best in class. We recently completed a positive Type C meeting with the FDA, and we are moving forward with our preparations for our Pivotal program in line with our previous guidance.
Steve Mahoney: Lastly for over one we are announcing that we anticipate filing a BLA for the old one program in the second half of 2025.
Steve Mahoney: Okay.
Steve Mahoney: For our subcutaneous <unk> three program, which we believe has substantially be best in class.
Steve Mahoney: We recently completed a positive type C meeting with the FDA and we are moving forward with our preparations for a pivotal program in line with our previous guidance.
Steve Mahoney: We will provide additional updates for <unk> III program before we start that pivotal program, which remains on track for midyear.
Steve Mahoney: We will provide additional updates for the 003 program before we start that pivotal program, which remains on track for mid-year. We are also progressing our FCRN portfolio as planned. We are aiming to file an IND for OO6 by the end of this year, and we plan to share OO8, non-human primate data in the second half of 2020. Lastly, we ended the quarter with $613 million in cash, cash equivalents, and short-term investments, and we maintain our cash runway into the second half of 2026, also as previously guided.
Steve Mahoney: We are also progressing our CRM portfolio as planned we are aiming to file an IND for all six by the end of this year and we plan to share Oh eight now.
Steve Mahoney: Non human primate data in the second half of 2024.
Steve Mahoney: Lastly, we ended the quarter with $613 million in cash cash equivalents and short term investments.
Steve Mahoney: Maintaining our cash runway into the second half of 2026 also as previously guided.
Steve Mahoney: Yeah.
Speaker Change: Now I would like to turn to our tech portfolio and talk more about the programs and our progress in more detail.
Steve Mahoney: Now I'd like to turn to our TED portfolio and talk more about the programs and our progress in a bit more detail. As a reminder, TED is an autoimmune condition characterized by inflammation and damage to the tissues around and behind the eyes. This leads to symptoms including proptosis or bulging of the eyes, redness, swelling, double vision, and retraction of the eyelids.
Speaker Change: As a reminder, Ted is an autoimmune condition characterized by inflammation and damage to the tissues around in behind the eyes.
Speaker Change: This leads to symptoms, including Proptosis or bulging of the eyes, redness swelling double vision and retraction to the islands.
Steve Mahoney: In severe cases, TED can be sight-threatening. With those symptoms as a backdrop, there is already a large market opportunity for TED that comes with global growth potential and an expansion that can come with better options for patients. There are an estimated 190,000 people in the U.S. alone who are living with moderate to severe TED. However, these patients are only served by one marketed IGF-1R IV therapy currently, which generated approximately $1.8 billion in sales in just the U.S. alone in 2023.
Speaker Change: In severe cases, Ted can be sight threatening.
Speaker Change: With those symptoms as a backdrop there is already a large market opportunity and Ted that comes with global growth potential and an expansion that can come with better options for patients.
Speaker Change: There isn't an estimated 190000 people in the U S alone who are living with moderate to severe Ted <unk>.
Speaker Change: These patients are only served by one marketed IGF one our IV therapy, currently which generated approximately $1 8 billion in sales in just the U S alone in 2023.
Steve Mahoney: This approved therapy requires eight infusions every three weeks, which can be a significant burden for patients. We see opportunity for us to provide differentiated options for TED patients with both our IV and sub-Q programs. Because TED is an autoimmune disease characterized by flaring symptoms, patients with moderate to severe symptoms struggle with quality of life issues that make it hard for them to drive, work, and even sleep because it is a flare-based disease.
Speaker Change: This approved therapy requires eight infusion every three weeks, which can be a significant burden for patients.
Speaker Change: Yeah.
Speaker Change: We see opportunity for us to provide differentiated options for Ted patients with both our IV and sub Q programs.
Speaker Change: Because <unk> is an autoimmune disease characterized by flaring symptoms patients with moderate to severe symptoms struggle with quality of life issues that make it hard for them to drive work and EBIT sleep.
Speaker Change: Because it is a swear based disease.
Steve Mahoney: It is considered a new startup market, which means that it doesn't matter when a patient was diagnosed because you need to treat the flare or the onset of those symptoms. This new startup market also means that all patients experiencing symptoms will have the opportunity to choose from available treatment options with no chronic treatment to replace. Once a flare is treated, patients do not remain on anti-IGF-1R therapy. Therefore, when a subsequent flare arises, physicians will have the opportunity to choose from available treatment options, including potential new options to manage these flares in their patients.
Speaker Change: It is considered a new start market, which means that it doesn't matter when a patient was diagnosed because you need to treat the flare or the onset of those symptoms. This new stock market also means that all patients experienced symptoms.
Speaker Change: We'll have the opportunity to choose the available treatment options with no chronic treatment to displace.
Speaker Change: Once a flare is treated patients do not remain under nanny anti IGF, one our therapy. So when a subsequent flare rises physicians will have the opportunity to choose from available treatment options, including potential new options to manage these flares and their patients.
Steve Mahoney: This is a great position for our IV and sub-Q programs in TED because we believe that we are developing potential best-in-class therapies in a drug class that is shown to be highly effective in inhibiting IGF-1R and in treating TED. Now, turning to the specifics of our product candidate.
Speaker Change: This is a great position for our IV and subcutaneous and Ted because we believe that we are developing potential best in class therapies and a drug class that steady is shown to be highly effective and inhibiting IGF one are and in treating Ted.
Speaker Change: Turning to the specifics of our product candidates Meridian is developing two anti anti IGF, one our antibodies for Ted or one which is delivered intravenously and over three which has delivered subcutaneously with the potential for self administration.
Steve Mahoney: Viridian is developing two anti-IGF-1R antibodies for TET. 001, which is delivered intravenously, and 003, which is delivered subcutaneously with the potential for self-administration. As you can see here, O03 and O1 have the same binding domain, and we expect them to bind IGF-1R similarly. However, they differ because 003 is engineered to have an extended half-life, which we have shown to be 40 to 50 days in healthy volunteers, which is four to five times that of 001, its parent molecule.
Speaker Change: As you can see here almost three and all want to have the same binding domain and we expect them to bind IGF one are similarly.
Speaker Change: They differ because our <unk> is engineered to have an extended half life, which we have shown to be 40% to 50 days in healthy volunteers, which is four to five times that of <unk> and its parent molecule.
Steve Mahoney: With 001, we hope to have a fast-to-market, differentiated IV therapy for patients with fewer doses and a shorter infusion time than the current standard of care. With 003, we hope to develop a convenient, less frequent, low-volume therapy that patients can self-administer at home. Let's review the OO1 program, our progress, and what makes us excited about the Phase 3 readout that we expect in September. On slide 11, this is a reminder that we've already shown robust clinical activity with O1 after just two infusions in a phase 2 clinical trial and active test. This robust activity is across all key areas of the disease, such as proptosis or the bulging of the eyes, clinical activity score, and diplopia or double vision.
Speaker Change: With over one we hope to have it a fast to market differentiated IV therapy for patients with fewer doses in a shorter infusion time than the current standard of care with <unk> three we hope to have we hope to develop a convenient less frequent low volume therapy that patients can self administer at home.
Speaker Change: Let's review the old one program, our progress and what makes US excited about the phase III readout Readouts that we expect in September.
Steve Mahoney: We have added data from the clinical trials after two doses on the slide to show the data side-by-side. While cross-trial comparisons are difficult, we are encouraged by the clinical responses observed after just two doses of OL1. On slide 12, you can see that 101 was well-tolerated and active TED patients with no serious adverse events, no infusion reactions, and no discontinuation. Similarly, in patients with chronic TED, just after just two infusions, O1 meaningfully reduced disease burden across each disease point as well. On slide 14, 001 was also well-tolerated in chronic TED patients.
Speaker Change: Slide 11. This is a reminder, that we have already shown robust clinical activity with all one after just two infusions in a phase II clinical trial in active Ted.
Speaker Change: This robust activity is across all key areas into disease, proptosis or bulging of the eyes clinical activity score and diplopia or double vision.
Speaker Change: We have added we have added data from the deposit clinical trials. After two doses on this slide to show the data side by side, while cross trial comparisons are difficult. We are encouraged by the clinical responses observed after just two doses of <unk> one.
Speaker Change: On slide 12, you can see that over one was well tolerated and active Ted patients with no serious adverse events no infusion reactions and no discontinuation.
Speaker Change: Similarly active active Ted in patients with chronic Ted just after just two infusions or meaningfully reduce disease burden across each disease point as well.
Speaker Change: On slide 14 O. One was also well tolerated in chronic Ted patients based on this phase II data, we believe that the clinical regimen of over one with fewer infusions shorter infusion time.
Steve Mahoney: Based on this phase 2 data, we believe that the clinical regimen of 001, with fewer infusions, shorter infusion time, and lower cumulative drug exposure, has the potential to be a better choice for moderate to severe patients with TED. Now, we turn to our phase three trials for O01. I would like to take a moment to thank all the patients and clinical trial site teams who have participated in our THRIVE trial. We're not done yet, and our achievements so far would not be possible without them.
Speaker Change: And lower cumulative drug exposure that has the potential to be a better choice for moderate to severe patients with Ted.
Speaker Change: Now turning to our phase III trials for <unk> I would like to take a moment to thank all of the patients in clinical trials site teams, who have participated in our thrive trial, we're not done yet and our achievements so far would not be possible without them.
Speaker Change: As we announced today, we completed enrollment for thrive in March with 113 patients, which exceeded our enrollment target of 90 patients due to strong demand and interest at our clinical sites.
Steve Mahoney: As we announced today, we completed enrollment for Thrive in March with 113 patients, which exceeded our enrollment target of 90 patients due to strong demand and interest at our clinical site. About half of Thrive's patients were from the U.S., and the other half came from Europe. We expect to provide top-line results for this study in September of this year. Thrive 2, our second pivotal study in TED, is ongoing and on track for top-line readout at the end of this year. In addition to THRIVE and THRIVE-2, we recently initiated STRIVE, which is a planned safety study.
Speaker Change: About half of drive patients were from the U S and the other half came from Europe.
Speaker Change: We expect to provide top line results for this study in September of this year.
Speaker Change: Drive to our second pivotal study and Ted is ongoing and on track for topline readout at the end of this year.
Speaker Change: In addition to thrive and thrive to weakness, we recently initiated strive which has a planned safety study stride is a study of over one in 10 patients to complete the sufficient safety database for BLA submission alongside the patient numbers from drive attractive.
Steve Mahoney: STRIVE is a study of O01 in TED patients to complete the sufficient safety database for BLA submission alongside the patient numbers from THRIVE and THRIVE-2. In conclusion, with O01, we are developing a potentially differentiated IV therapy for patients with fewer doses and shorter infusion time than the current standard of care, while inhibiting the same IGF-1R target, which has been shown clinically and commercially to be effective in treating tests We are very excited about bringing 4-0-1 as a potential option for patients, which could mean significantly less medication for patients, fewer visits to the infusion center, lower volumes, and less infusion chair time.
Speaker Change: In conclusion with the old one we are developing a potentially differentiated IV therapy for patients with fewer doses and shorter infusion time than the current standard of care, while inhibiting the same IGF, one our target, which has been shown clinically and commercially to be effective in treating that.
Speaker Change: We are very excited about bringing forward or one as a potential option for patients, which could mean significantly less drug for patients fewer visits to the infusion center lower volumes and less infusion chair time.
Speaker Change: Our next program subcutaneous <unk> III will take this differentiation, even further with the possibility of lower frequency.
Steve Mahoney: Our next program, Subcutaneous 003, will take this differentiation even further, with the possibility of lower frequency subcutaneous administration and the potential for at-home self-administration using auto-injection. We know from market examples that a later entrant subcutaneous therapy can convert meaningful portions of an existing IV market. We've included two of those examples here.
Speaker Change: Lower frequency lower frequency subcutaneous administrations and.
Speaker Change: And potential for at home self administration using auto injectors.
Speaker Change: We know from market examples at a later entrant sub acute therapy can convert meaningful portions of an existing IV market and we've included two of those examples here.
Steve Mahoney: In each of the cases on this slide, sub-Q offerings grew the overall market size of the class, in addition to quickly commanding a significant share of the IV market. And keep in mind that these sub-Q examples have the same or more frequent dosing than their IV counterparts. This would not be the case with 003, which is designed to have potentially a best-in-class dosing profile. Also, it is important to point out in both examples that neither of these are new startup markets.
Speaker Change: In each of the cases on this slide sub Q offerings grew the overall market size of the class. In addition to quickly commanding a significant share of the IV markets.
Speaker Change: And keep in mind that these sub Q examples have the same or more frequent dosing than their IV counterparts. This would not be the case with our three which is designed to have a potentially a best in class dosing profile.
Speaker Change: Also it is important to point out in both examples that neither of these are a new start market again, the Ted market is a new start market, where we need to treat flaring disease or onset of symptoms as opposed to trying to convert patients from a long term chronic therapy with over three we hope to provide patient.
Steve Mahoney: Again, the TED market is a new startup market where we need to treat flaring disease or the onset of symptoms as opposed to trying to convert patients from a long-term chronic therapy. With 003, we hope to provide patients with an anti-IGF-1R therapy that is a better option with respect to less overall drug exposure and more convenience. On slide 19, we show the complete data set from our Phase 1 Healthy Volunteer Study of 003 to assess PK and PD.
Speaker Change: With an anti IGF, one our therapy that has a better option with respect to west overall driving exposure and more convenience.
Speaker Change: On Slide 19, we show the complete data set from our phase one healthy volunteer study of our <unk> III to assess PK and PD.
Steve Mahoney: The update here is the inclusion of the last cohort, cohort five, where participants received two doses of OLA3 28 days apart. The data confirms the differentiated pKa and pD for O03 seen in the first four cohorts, with an extended half-life of 40 to 50 days and sustained increased levels of the pD biomarker IGF-1. On slide 20, you can see that subcutaneous O03 was well tolerated in the phase one study, including in the latest cohort five, with no serious adverse events or discontinuations related to treatment, and observed adverse events were generally grade one and mild.
Speaker Change: The update here is the inclusion of the last cohort cohort five we're participants receive two doses of <unk> 328 days apart.
Speaker Change: The data confirms the differentiate PK differentiated PK and PD for all three <unk> seen in the first folk four cohorts with an extended half life of 40% to 50 days at sustained increased levels of the PD biomarker IGF one.
Speaker Change: On slide 20, you can see that the subcutaneous <unk> three was well tolerated in the phase one study, including in the latest cohort five with no serious adverse events or discontinuation is related to treatment and observed adverse events were generally grade one and mile.
Speaker Change: As we shared previously our pharmacokinetic modeling for all three.
Steve Mahoney: As we shared previously, our pharmacokinetic modeling for O03 showed that or predicted that three potential dosing regimens are available to us. O03 every three, eight weeks, every four weeks, and every two weeks could achieve or exceed the exposure levels of O01 that we saw in the active and chronic studies that were correlated to robust clinical activity in our phase two clinical trials and tests. This gives us a lot of optionality as we move towards our pivotal studies for 003 and importantly gives us the potential to develop for patients a best-in-class, low-volume, sub-Q delivery option.
Speaker Change: It showed that our predicts that three potential dosing regimens that are available to us all three every three eight weeks every four weeks and every two weeks could achieve or exceed the exposure levels of old one that we saw in the active and chronic studies that were correlated to robust.
Speaker Change: On our call activity for our phase III clinical trials and Ted.
Speaker Change: This gives us a lot of Optionality as we move for move towards our pivotal studies for <unk> III and importantly gives us the potential to develop for patients a best in class low volume Saatchi delivery option.
Speaker Change: We are pleased to announce today that we have completed our type C meeting with the FDA and we are on track to initiate pivotal clinical trials for the <unk> three program, we will share more details on the pivotal trial design before we start those studies.
Steve Mahoney: We are pleased to announce today that we have completed our Type C meeting with the FDA, and we are on track to initiate pivotal clinical trials for the OO3 program. We will share more details on the pivotal trial design before we start those studies.
Speaker Change: Now turning to our <unk> portfolio.
Steve Mahoney: Now, turning to our FCRN portfolio. On slide 24, in addition to TED, and consistent with our development strategy, we are developing an exciting portfolio of potential best-in-class FCRN inhibitors to address the unmet needs of patients living with auto-antibody-mediated autoimmune disease. FCRN inhibitors represent a large market opportunity. The first FCRN inhibitor, FGART or VivGART, is approved for myasthenias gravis and is in registration for CIPD, and it's already annualizing to over $1 billion in annual sales. Myasthenia gravis alone is a large market, with projected sales of over $4 billion annually by 2028.
Speaker Change: On Slide 24 in addition to Ted and consistent with our development strategy. We are developing exciting portfolio of potential best in class F. Cri inhibitors to address the unmet needs of patients living with auto antibody mediated autoimmune disease.
Speaker Change: F CRM inhibitors represent a large market opportunity. The first SCR CRM inhibitor F guard or <unk> is approved for myasthenia gravis is in registration for Ci PD and it's already annualizing over over to over $1 billion in annual sales.
Myasthenia gravis alone is a large market with projected sales of over $4 billion annually by 2028. In addition to myasthenia gravis as you can see from the slide there are additional sizable autoimmune indications that would meaningfully add to the CRM.
Steve Mahoney: In addition to myasthenia scurvis, as you can see from the slide, there are additional sizable autoimmune indications that would meaningfully add to the FCRN opportunity. Our FCRN franchise includes two assets, 06 and 08. With those six, we are excited to have the only other FCRN targeting FC fragment in development other than Oscar Tigeman. Argenix has shown that its FC fragment achieves substantial efficacy while sparing an effect on albumin or LDL and shows better tolerability than the full-length antibody.
Speaker Change: Opportunity.
Speaker Change: Our <unk> franchise includes two assets or six in Norway.
Speaker Change: With over six we're excited to have the only other CRM targeting FC fragment in development other than <unk> or.
Speaker Change: <unk> has shown in its FC fragment achieved substantial efficacy, while sparing and effect on albumin or LDL and shows better tolerability than the full length antibodies.
Steve Mahoney: We're on track to submit an I.D. for O6 by the end of this year and look forward to sharing more about the program in the future. Next on the right is 08.
Speaker Change: We're on track to submit an IND for <unk> by the end of this year and look forward to sharing more about the program in the future.
Speaker Change: Next on the right is <unk> eight our protein engineering efforts identified a molecule derived from FC fragments that both extended the half life and generated meaningfully deeper IGT reductions in animal models.
Steve Mahoney: Our protein engineering efforts identified a molecule derived from FC fragments that both extended the half-life and generated meaningfully deeper IgG reductions in animal models. We believe OO8 is a potential best-in-class extended half-life molecule targeting FCRN with the potential to more durably suppress IgG. We are on track to provide 008 non-human primate data in the second half of this year as planned, and we are excited to bring forward this portfolio of next-generation FCRNs to potentially offer patients a more convenient dosing profile compared to current weekly IV or sub-Q infusions. In addition, by aiming to improve the duration and depth of IgG suppression with 008, we hope to offer a best-in-class option for patients.
Speaker Change: We believe OA is a potential best in class extended half life molecule targeting <unk> CRM with the potential to do more durably suppress.
Speaker Change: Agg.
Speaker Change: We are on track to provide OA nonhuman primate data in second half of this year as guided and we are excited to bring forward. This portfolio of next generation <unk> to potentially offer patients a more convenient dosing profile compared to current weekly IV are subdued fusions. In addition by Amy.
Speaker Change: To improve the duration and depth of IGT suppression with OE, we hope to offer a best in class option for patients.
Speaker Change: Our team is executing we have made excellent progress across the company in the first quarter of the year and we look forward to continuing that momentum through the rest of the year to deliver on our multiple upcoming catalysts.
Steve Mahoney: Our team is executing. We have made excellent progress across the company in the first quarter of the year. And we look forward to continuing that momentum through the rest of the year to deliver on our multiple upcoming catalysts. As I mentioned previously, we plan to report Thrive Topline in September, and Thrive 2 Topline is on track for the end of the year. The FCRN programs are also proceeding as expected. It has been my pleasure today to provide these exciting updates across our portfolio, and in particular for OO1 and OO3, reflecting the work that we've completed during this quarter.
Speaker Change: I mentioned previously we plan to report drive topline in September and thrived to topline is on track for the end of the year the.
Speaker Change: <unk> programs are also proceeding as expected.
Speaker Change: It has been my pleasure today to provide these exciting updates across our portfolio and in particular for <unk> III.
Speaker Change: III, reflecting the work that we've completed the call and during this quarter.
Steve Mahoney: This progress and our recent achievements reflect our team's ability to execute, and we are well positioned to continue the work and deliver on our exciting upcoming category. Last but not least, we remain well-capitalized, ending the quarter with $613 million, and the runway is into the second half of 2020. So with that, I'll ask the operator to open the call for questions. Operator?
Speaker Change: This progress on our recent achievements reflect our team's ability to execute and we are well positioned to continue to work and deliver on our exciting upcoming catalysts.
Speaker Change: Last but not least we remain well capitalized ending the quarter with $613 million and the runway is into the second half of 2026.
So with that I'll ask the operator to open the call for questions operator.
Speaker Change: Thank you we will now begin the question and answer session. 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.
Operator: Thank you. We will now begin the question and answer session. If you have dialed in and would like to ask a question, please press star 1 on your telephone keypad to raise your hand and join the queue. If you would like to withdraw your question, simply press star 1 again. If you're called upon to ask your question and are listening through a loudspeaker in your device, please pick up your handset and ensure that your phone is not in mute when asking your question. Again, press star one to join the conversation, and the first question comes from the line of Laura Chico with Wedbush. Please go ahead.
Speaker Change: If you would like to withdraw your question simply press Star one again.
Speaker Change: If you are called upon to ask your question and are listening by a loud speaker in your device. Please pickup your handset and ensure that your phone is not on mute when asking your questions.
Speaker Change: Again press star one to join the queue.
Speaker Change: And your first question comes from the line of Laura Chico with Wedbush.
Laura Kathryn Chico: Please go ahead.
Steve Mahoney: Good morning. Thanks very much for taking the time to answer that question. And congrats on the progress here. I guess I have two questions for you. First, with respect to the THRIVE data that's coming out in September, I'm wondering if you can kind of help us frame what success looks like on the efficacy side, but then also with respect to reduced drug exposure, how would you think this might impact the rate of hearing impairment events that you might see in THRIVE? And then, secondarily, just related to STRIVE, I'm wondering if you could talk a little Thanks.
Laura Kathryn Chico: Good morning, Thanks, very much for taking my question and congrats on the progress here I guess I have two questions for you.
Laura Kathryn Chico: First with respect to the thrive data that's coming in September I'm wondering if you can kind of help us frame what success looks like on the efficacy side, but then also with respect to reduce drug exposure. How would you think this might impact the rate of hearing impairment events that you might see in trials and then secondarily just related to strive I'm wondering if you could talk a little bit more about the inclusion of.
Laura Kathryn Chico: The active control arm.
Speaker Change: Yes sure.
Steve Mahoney: Yeah, sure. Thanks, Laura, for the question. In terms of what a good look like for Thrive Advocacy. As we've stated previously, we think a profile that looks like Tepeza is similar to Tepeza would be a really good place for us to land. With respect to hearing, yes, certainly. I think what we're looking for, in the same vein as efficacy for safety, getting a similar profile for safety, because the safety profile for Tepeza is good. It's benign. And you referenced hearing in particular.
Speaker Change: Laura for the question.
Speaker Change: In terms of what is good look like for thrive efficacy.
Laura Kathryn Chico: As we've as we've stated previously we think a profile that looks like <unk> is similar to <unk> would be a really good place for us to land.
So.
Laura Kathryn Chico: With respect to hearing.
Laura Kathryn Chico: Yes, certainly.
Laura Kathryn Chico: I think what we're looking for in the same vein on efficacy for safety getting a C.
Laura Kathryn Chico: More profile on safety, because the safety profile for <unk>.
Laura Kathryn Chico: Is good it's benign and.
Laura Kathryn Chico: You reference hearing in particular to the extent the lower exposures improve upon that that would be great to the extent that C. Max driven we obviously have a lower <unk> just by virtue of the volume.
Steve Mahoney: To the extent the lower exposures improve upon that, that would be great. To the extent it's CMAX driven, we obviously have a lower CMAX just by virtue of the volume that we deliver versus Tepeza. So that could possibly be helpful. We'll have to see. But I think in terms of what good looks like, we'd love to see a similar profile. With respect to your question on STRIVE, yeah, I mean, look, STRIVE is simply to complete the safety database, which is just normal blocking and tackling for a BLA submission. So nothing there is unusual.
Laura Kathryn Chico: That we deliver versus to pass so that could possibly be helpful. We will have to see but I think in terms of what good looks like we'd love to see a similar profile.
Laura Kathryn Chico: With respect to your question on strive.
Speaker Change: Yes, I mean look this drive is simply too.
Speaker Change: The safety database, which is just normal blocking and tackling for BLA submission. So.
Speaker Change: Nothing nothing there unusual on the active control arm. It's just you got to run a well controlled study.
Steve Mahoney: On the active control arm, it's just you've got to run a well-controlled study. And so we have the option of an active control arm of 3 megs per gig. It randomizes 3 to 1, so the numbers will be heavily weighted towards the 10 megs per gig. Again, it's all for the safety side of it. So I hope that answers the question.
Speaker Change: And so we had the option of a law.
Speaker Change: Of an active control arm of three makes the keg and randomized three to one so the numbers will be heavily weighted towards the 10 megs per keg again, its all for the safety side of it so I hope that answers the question.
Speaker Change: Yeah. It does thanks, Steve.
Speaker Change: Your next question comes from the line of Alex Thomson with Stifel. Please go ahead, great. Thanks for taking our questions I guess I wanted to drill down a little bit more.
Steve Mahoney: Your next question comes from the line of Alex Thompson with Stifel. Please go ahead. Great.
Steve Mahoney: Thanks for taking our questions. I guess I wanted to drill in a little bit more on safety, in particular. You know, I guess when you look back at the TEPEZA safety profile from the Phase 3s versus the more recent chronic TED study, like, do you feel like the chronic TED study might represent a better, you know, if you ran a TEPEZA study today with more, you know, more focused hearing measuring, if that's more of a priority for safety?
Alex Thompson: On safety in particular, I guess when you when you look back at the passive safety profile from the phase threes versus the more recent chronic Ted study.
Alex Thompson: Do you feel like the chronic Ted study might represent a better if you ran into peasant day today with more.
Alex Thompson: More focus hearing measuring if thats more of a bar for safety or how are you thinking about like what to present safety actually looks like today versus when those phase threes were started and then for the top line for <unk>. One in phase III do you expect to be able to share any data beyond 15 weeks as part of that either for safety.
Steve Mahoney: Or how are you thinking about, like, what TEPEZA safety actually looks like today versus when those Phase 3s were started? And then for the top line for O1 in Phase 3, do you expect to be able to share any data beyond 15 weeks as part of that, either for safety or efficacy? Thanks.
Alex Thompson: D or efficacy.
Speaker Change: Well I can take the second one first Alex.
Speaker Change: No its topline as topline so it'll be it'll be a readout at the 15 week end point.
Speaker Change: With respect to.
Speaker Change: Your question on what would it sounds like Youre, asking what is deposit post the clinical trials.
Speaker Change: What that real world experiences, yes that is all part of it we're trying to understand that as well I mean, I know Amgen is trying to understand that.
Speaker Change: Physician community the patient community, they're all trying to understand that.
Speaker Change: And.
Steve Mahoney: I'd like to talk to you. Do you want to just explain how we're approaching our reporting of AEs in the same way that that's about it? Sure. So Alex, as you know,
Speaker Change: Maybe talk to what do you want to just explain how we're approaching it.
Speaker Change: Our reporting of.
Speaker Change: Of of Aes in the same way that the president.
Speaker Change: Sure. So Alex as you know the field is evolving as you alluded to.
Steve Mahoney: Sure. So, Alex, as you know, the field is evolving, as you alluded to. There's updated guidance from the FDA, which led to a label change for TEPRO, as you know. And in current clinical practice, physicians are assessing patients' hearing at baseline, during, and after treatment. So, we're recording adverse events using MedDRA terms, and as you know, this is just a standard way of recording patient-reported changes in their health, including hearing. This is standard for any clinical trial, including TEPA and its pivotal trials. We're also assessing audiometry, as is done in clinical practice, at baseline and pre-specified points. So we're essentially doing what's done currently in evolving clinical practice. Great. Now, your next question.
Speaker Change: There is the updated guidance.
Alex: EMEA, which led to a label change for <unk> as you know and in current clinical practice physicians are assessing patients hearing at baseline during and after treatment.
Speaker Change: So we are recording adverse events using med returns and as you know this is just a standard way of recording patient reported changes in their health, including hearing.
Speaker Change: This is a standard for any clinical trial, including Teva in Nir.
Speaker Change: Heather in their pivotal trials.
Speaker Change: We're also assessing audiometry.
Speaker Change: <unk> is done in clinical practice.
Speaker Change: Baseline increased specified points. So we're essentially doing what's done currently in the evolving clinical practice.
Speaker Change: Great. Thanks.
Speaker Change: Your next question comes from the line of Michael Yee with Jefferies. Please go ahead.
Steve Mahoney: Your next question comes from the line of Michael Yee with Jefferies. Please go ahead.
Speaker Change: Okay.
Speaker Change: Okay.
Speaker Change: Okay.
Speaker Change: Hello.
Michael Yee: Okay, Great I guess, we can agree.
Steve Mahoney: Okay, great. I guess we can hear you.
Michael Yee: Two questions.
Michael Yee: First was on the ongoing thrive study can you talk a little bit about.
Steve Mahoney: So, two questions. The first was on the ongoing THRIVE study. Can you talk a little bit about how you can control for hearing impairment and hearing loss events? I know that if you actually look back at some of the TPEZA post-marketing studies, there's some commentary and analysis around how patients have some of this hearing loss already, and there's factors already going on with some of these TED patients in the background.
Michael Yee: How you can control for the hearing impairment and hearing loss.
Michael Yee: That's I know that if you actually go look back at some of the competitive push.
Michael Yee: Push marketing studies that there is some commentary and analysis around how patients have some of this hearing loss already in their factories already going on with somebody as Ted patients in the background and so I'm just trying to think about how you can screen and protect for that.
Steve Mahoney: And so just trying to think about how you can screen or protect for that and think about that as you go through your phase three. And then on the sub-Q plans for phase three, I think you said that you met with the FDA and you're planning to start phase three. Can you just talk a little bit about how that meeting went? And I know there were some uncertainties about going directly into phase three, so just talk a little bit about your confidence there or anything else that you need to do in order to start phase three for sub-Q. Thank you.
Michael Yee: Think about that as you go through your phase III.
Steve Mahoney: Yeah. Great. Thanks. Thanks, Mike. Let me take the second one first, and then I'll ask Tom Chula to weigh in on the baseline hearing question that you had first.
Michael Yee: And then on the sub Q plans for phase three I think you said that you met with the FDA and Youre planning to start phase III can you just talk a little bit about how that meeting went and I know there were some uncertainties about glenn directly into phase III and just talk a little bit about your confidence there or anything else that you need to do in order to.
Michael Yee: Start the phase III for sub Q. Thank you.
Speaker Change: Yeah, great. Thanks, Thanks, Mike Let me, let me take the second one first and then I'll and then I'll ask Tom Ciulla to weigh in on the.
Speaker Change: The baseline hearing question that you had first so with respect to the <unk> three program.
Steve Mahoney: So with respect to the 003 program, we did have a positive meeting. We have not received the minutes yet, but we had a positive meeting, and we are reiterating our guidance that we are going to start a pivotal program mid-year this year. So we will provide a lot more detail once we get on the other side of the minute, but before we start the study. So more to come, but to answer your question, positive meeting.
Speaker Change: Excuse me.
Speaker Change: We did have a positive meeting.
Speaker Change: We have not received the minutes yet so.
Tom Ciulla: But we had a positive meeting and we are reiterating our guidance that we are going to start a mid a pivotal program.
Tom Ciulla: Midyear 2000 this year.
Tom Ciulla: So we will provide a lot more detail once we get once we get it on the other side of minutes, but before we start the study.
Tom Ciulla: So more to come but.
Speaker Change: Answer your question positive meeting, we feel good about our reiterating our guidance on starting that pivotal program. So we're pretty excited there with.
Steve Mahoney: We feel good about reiterating our guidance on starting that pivotal program. So we're pretty excited about that. With respect to Thrive and the baseline hearing, I'll ask Tom Chula to jump in there. Thanks, Michael.
Speaker Change: With respect to the thrive in the baseline hearing I'll ask Tom Ciulla to jump in that please.
Steve Mahoney: Thanks, Michael. As we said in the previous answer, adverse events in the studies were reported via the measure of terms, which is a standard methodology for reporting patient changes in their health, including hearing. And this was done in the TETESA trials. As I mentioned, we're also using audiometry for monitoring, and that's consistent with the current clinical practice in FDA guidance. We do have an exclusion criteria for hearing loss at baseline, and you can see that exclusion on clinicaltrials.gov.
Thomas Jonathan Smith: Thanks, Michael So as we said in the previous answer adverse events in the studies reported via the merger terms.
Thomas Jonathan Smith: Standard methodology for reporting patient changes in their health, including hearing and this was done in the competitive trials as I mentioned, we're also using audiometry for monitoring and that's consistent with the current clinical practice and FDA guidance.
Thomas Jonathan Smith: We do have an exclusion criteria for hearing loss at baseline and you can see that exclusion on clinical trials dot Gov.
Thomas Jonathan Smith: Yes.
Thomas Jonathan Smith: Okay.
Speaker Change: Okay. Thank you.
Speaker Change: Your next question comes from the line of Gavin client scenario with Evercore. Please go ahead.
Steve Mahoney: Your next question comes from the line of Gavin Clark Gardner with Evercore. Please go ahead.
Gavin: Good morning, and thanks for taking my questions just had two.
Steve Mahoney: Good morning, and thanks for taking my questions. I just had two. First, at the type C meeting for 003, does the FDA want to see any dose-ranging work in TED patients as part of that pivotal? Or do you believe you can start dosing immediately in a blinded pivotal portion of the trial?
Gavin: First on the type C meeting for <unk> III does the FDA want to see any dose ranging work in Ted patients as part of that pivotal or do you believe you can start dosing immediately in like a blinded pivotal portion of the trial.
Speaker Change: Yeah. So thanks, Kevin like I said.
Steve Mahoney: Yeah, so thanks, Gavin. Like I said, give us give us a little bit more time. We'd like to see the minutes, but we can take comfort from the fact that we feel positive coming out of that meeting and that we are starting our Pivotal program, which is what we've been guiding to previously, but we've now had that meeting. So we feel good about where we're going, but give us a little bit more time, and we'll be able to break down those details for you. But, but that's kind of where we are, and we feel good.
Speaker Change: Give us give us a little bit more time, we'd like to see the minutes.
Speaker Change: But but Jack.
Speaker Change: But just take comfort from the fact that we are we.
Speaker Change: We feel positive coming out of that meeting and that we are starting our pivotal program, which is what we've been what we were guy.
Speaker Change: Guided to previously, but we've now had that meeting so we feel good about where we're going.
Speaker Change: But give us a little bit more time, and we'll be able to break down those details for you.
Speaker Change: But that's that's kind of where we are and we feel good.
Speaker Change: Sounds good we'll await more details and are you able to provide any details on how the thrive baseline characteristics comparator at the pedal phase III.
Steve Mahoney: Sounds good. We'll await more details. And are you able to provide any details on how the Thrive baseline characteristics compare to Tepeza's Phase 3?
Steve Mahoney: Yeah, that's another one, Gavin. I mean, it's a great question. I totally appreciate it. It's just that we're just not there yet. We don't have all that information for Baseline Thrive. We just completed enrollment, and so, you know, it's going to take us a bit to just get all that together. So more to come on that one as well.
Speaker Change: Yes, that's another one Gavin I mean, it's a great question and I totally appreciate it. It's just that we're just not there yet.
Speaker Change: We don't have all that information.
Speaker Change: For baseline thrive.
Speaker Change: We just completed enrollment.
Speaker Change: So it's going to take us a bit to just get all back together.
Speaker Change: More to come on that one as well.
Speaker Change: Makes sense. Thank you.
Steve Mahoney: Makes sense. Thank you. Your next question comes from the limelight, Rami Katkhuda with Lifesci Capital. Please go ahead. Hey guys.
<unk>: Your next question comes from the line of <unk> with lifestyle capital. Please go ahead.
Steve Mahoney: Your next question comes from the limelight, Rami Katkhuda with Lifesci Capital. Please go ahead. Hey guys.
Lifestyle Capital: Hey, guys. Congrats on all the progress and thanks for taking my questions as well.
Lifestyle Capital: You touched on the significant ex U S market opportunity and Ted I guess are you planning to file for approval of <unk> in the U S and Europe in parallel once the data is in hand, and how large of an opportunity could ultimately represent.
Ted: Yeah, I think the epidemiology in Europe is very similar so we know that to be the case similar to the U S that is with.
Steve Mahoney: Yeah, I think the epidemiology in Europe is very similar. So we know that to be the case, similar to the US, that is, with respect to our ex-US plans. Again, that's something we'll probably talk a bit more about later. We are, as you can imagine, absolutely thinking about all that and the best approaches in these different geographies, even beyond Europe. So that's all in the works. I will have more to say at a later time.
Speaker Change: With respect to our ex U S plans.
Speaker Change: That's something we'll probably talk a bit more about later.
Speaker Change: As you can imagine we are absolutely thinking about all of that and the best approach is.
Speaker Change: In these different geographies, even beyond Europe. So that's all in the works it will have more to say at a later time.
Speaker Change: Got it sounds good thanks.
Speaker Change: Your next question comes from the line of Derek <unk> with Wells Fargo. Please go ahead.
Steve Mahoney: Your next question comes from the line of Derek Archila with Wells Fargo. Please go ahead.
Speaker Change: Hey, this is Adam on for Derik, Thanks for taking our questions. Today I guess, just a couple of questions on the timeline really so given the tribe is still reading out like mid 2020 formation drive to leaning on end of year. What factors are driving second half 'twenty five BLA submission in tech.
Steve Mahoney: Hey, this is Adam on behalf of Derek. Thanks for taking our questions today. I guess just a couple questions on the timeline really. So given Thrive is still reading out like mid 2024-ish and Thrive 2 reading out end of year, what factors are driving a second half 25 BLA submission in TED? And is this related to the Strive study then? And in that sense, is this an interim cut; would that be sufficient from Strive for a BLA submission? Or does the whole Strive trial need to be completed to support the BLA submission?
Steve Mahoney: Great. Thanks, Adam.
Speaker Change: Ed.
Speaker Change: Is this related to this drive study than in and in that sense is an interim cut would that be sufficient from strive for a BLA submission or does the whole strive trial need to be completed to support the BLA submission.
Speaker Change: Great. Thanks, Adam I appreciate that question.
Steve Mahoney: I appreciate that question. Yeah, so What's driving the timeline is, remember, we talked about Thrive 2, that's a top-line readout at the end of the year. So by definition, it's not a completed study, right? So we have to let that, we have a follow-up period, there's a total of a 52-week follow-up period, but only 37 weeks post the last dose. So there's a follow-up period that has to be taken into account, and it's primarily Thrive 2 that's driving the timeline. It really doesn't, we're not expecting Strive to have an impact there.
Adam: Yes so.
Adam: What's driving the timeline is it remember so we've talked about drive to that a top line readout at the end of the year. So by definition, it's not a completed study right. So we have to let that we are we have a follow up period. If there is a.
Adam: There is a total of 52 week follow up period, but only 37 weeks post the last dose. So there is a follow up period that is has to be taken into account.
Adam: And it's primarily thrive to that's driving the timeline it really doesn't we're not expecting strive.
Adam: To have an impact there that strive should fit squarely within that timeline.
Steve Mahoney: That Strive should fit squarely within that timeline. And so we feel that that's probably the driver, and we don't need all of Strive. What you see here is that on ct.gov, you see 212 patients; that's the maximum number that we'll need. We can do a data cut as soon as we reach the requisite number for the safety database, and there are moving parts that go with that in terms of, you know, you can have dropout rates in your Thrive or Thrive 2.
Adam: And so we feel.
Adam: But that's probably the driver and we don't need all of strive what you see there on.
Adam: On the <unk> 212 patients.
Adam: That's the maximum number that we'll need we can do a data cut as soon as we reach.
Adam: The requisite number for the safety database.
Adam: And.
Adam: There's a little there moving parts that go with that in terms of you cannot dropout rates in Erith driver drive too so we kind of over.
Steve Mahoney: So we kind of over-engineer or over-set up the Strive study, but we can do a data cut when we hit that threshold. And again, all of this is really typical. You have to have a safety database that accompanies your BLA submission.
Adam: We over engineer are over set up the strive study, but we can do a we could do a data cut when we hit that threshold.
Adam: And again all of this is really typical you have to have.
Adam: Safety database that accompanies your BLA submission. So it all kind of is normal blocking and tackling from our perspective.
Steve Mahoney: So it all kind of is normal blocking and tackling from our perspective, but just kind of take into account that Thrive 2 is just a top-line readout, so there's more to do after a top-line readout, which drives the second half 25-5.
Adam: But just kind of take into account that that thrive too is just a top line readout. So theres more to do after topline readout, which drives the second half 'twenty five filings.
Speaker Change: Got it that makes sense.
Steve Mahoney: That makes sense. And I guess, can you provide any numbers on what that threshold is? And then, just kind of following up on the STRIVE study, is this the safety database you expect to leverage with any potential regulatory path for VRDN 003? Yeah, so all three.
Speaker Change: I guess can you provide any numbers on what that threshold is and then just kind of following up on the stride study. Two is this the safety database, you expect to leverage than with any potential regulatory path.
Speaker Change: For BRD and 003.
Speaker Change: Thank you.
Speaker Change: Yes, so all three as we've talked about previously as it is a.
Steve Mahoney: Yeah, so O3, as we've talked about previously, is a different molecular entity. So it will have its own path.
Speaker Change: Different molecular entity. So it will have its own path. So the answer that question was now on the.
Speaker Change: On the first question the threshold is.
Steve Mahoney: So the answer to that question is no. On the first question, the threshold is—the threshold, again, like I said, 212 is the max number. We're not—we'll see where we end up. The total number is 300, but that includes Thrive and Thrive II active patients on 10 migs per keg. So we're not anticipating needing the entire STRIVE study. And again, most importantly, STRIVE is not expected to drive the timeline for BLA submission. It's more like Thrive II.
Speaker Change: The threshold again like I said 212 is the Max number.
Speaker Change: We're not we will see where we end up the total number is 300, but that includes thrive and thrive to active patients on 10 Meg per kg. So we're not anticipating needing the entire strive study and again most importantly.
Speaker Change: Drive is not expected to drive the timeline for BLA submission, it's more that drive team.
Speaker Change: Got it thank you.
Speaker Change: Okay.
Steve Mahoney: Your next question comes from the line of Gregory Renza with RBC. Please go ahead.
Speaker Change: Your next question comes from the line of Gregory Renzo with RBC. Please go ahead.
Speaker Change: Yes.
Steve Mahoney: Hey Steve, congratulations on the progress, thanks for taking my question. Maybe Steve, for you and perhaps Tom, just wanted to get your latest views on the competition for patients and clinical trials, certainly as your trials are heating up, and appreciate all the progress you have made. There are others out there as well, TEPRO with the subcutaneous. What's the latest on driving, demand, and what levers are you pulling to really accelerate enrollment as well as the trial?
Gregory Renza: Hey, Steve Congrats on the progress. Thanks for taking my question, maybe for you and perhaps that Tom just wanted to get your latest views on the competition for patients and clinical trials certainly.
Gregory Renza: As your trials are heating up and I. Appreciate all of the progress you have made and there are others out there as well type ROE with a subcutaneous what's the latest on driving demand and what levers are you pulling to really accelerate the enrollment as well as the trial execution.
Steve Mahoney: Yeah, so I'll turn this over to Tom in a second. But yeah, I think if you could, you could see that we enrolled Thrive on time, we exceeded enrollment, and we had really strong patient demand to drive that all within the month, all within the month of March. I think that's a clear sign to the world that there are lots of patients out there with TED that want to access IGF-1R therapy. So that's a really good sign for us. Don't forget also that roughly half of the patients were enrolled in the U.S. I know that was a question mark for people.
Gregory Renza: Yes, so I'll turn this over to Tom.
Thomas Jonathan Smith: In a second but.
Thomas Jonathan Smith: Yes, I think if you could you could see that we enrolled not only did we enroll thrive on time, we exceeded enrollment we had really strong patient demand to drive that all within the mantra at were all within the month of March.
Thomas Jonathan Smith: That's a I think that should be a clear sign.
Thomas Jonathan Smith: To the world that there are lots of there are lots of patients out there with Ted.
Thomas Jonathan Smith: That water access IGF, one our therapy. So that's a really good sign for US don't forget also that we had roughly half of the patients were enrolled in the U S. I know that was a question Mark for people I think we've definitively answered that the U S.
Steve Mahoney: I think we've definitively answered that the U.S. is very, you know, we've got the opportunity within the U.S. and then the other half in Europe where, as I mentioned, the epidemiology is insane. So I think that might be just a general answer. Tom, Chula, do you want to talk about how competition for trials is shaking up? Sure.
Thomas Jonathan Smith: <unk>.
Thomas Jonathan Smith: We've got the opportunity within the U S. And then the other half in Europe, where they are as I mentioned the epidemiology is insane.
Thomas Jonathan Smith: So.
Thomas Jonathan Smith: It might be just a general answer Tom Ciulla do you want to talk about.
Thomas Jonathan Smith: How come.
Thomas Jonathan Smith: Competition for trials is shaking out sure yes. Thanks for your question Gregory.
Steve Mahoney: Thanks for your question, Gregory. I'm out in the field a lot talking to investigators and key informants both in the U.S. and ex-U.S., and I can tell you there's a lot of excitement about our entire portfolio. As you know, our Phase 2 trial showed really promising results, and that's driven a lot of interest. Hence, the over-enrollment that Steve referenced. Also, I can tell you that with respect to STRIVE, we have an active control, there is no placebo, and we think that's going to drive enrollment there.
Thomas Jonathan Smith: I'm out in the field a lot talking to investigators investigators and kols both in the U S and ex U S and I can tell you there's a lot of excitement about our entire portfolio.
Thomas Jonathan Smith: As you know our phase II trial showed really promising results and that's driven a lot of interest hence.
Thomas Jonathan Smith: The over enrollment that Steve referenced.
Thomas Jonathan Smith: Also I can tell you that.
Thomas Jonathan Smith: With respect to strive and we have an active control there is no placebo and we think that's going to drive enrollment there. So I think overall just lots of positivity around our Ted portfolio in our trials.
Steve Mahoney: So I think overall, just lots of positivity around our TED portfolio in our trials. I really can't comment on competitors, but what I can say is just lots of excitement and enthusiasm around our portfolio. Your next question comes from the line of Julian Harrison with BT.
Thomas Jonathan Smith: I really can't comp I really can't.
Thomas Jonathan Smith: Comment on.
Thomas Jonathan Smith: Competitors, but what I can say is just lots of excitement enthusiasm around our portfolio.
Speaker Change: Great. Thanks, guys.
Speaker Change: Your next question comes from the line of Julian Harrison with <unk>. Please go ahead.
Steve Mahoney: Your next question comes from the line of Jillian Harrison with BTIG. Please go ahead.
Julian Harrison: Hi, Good morning. Thank you for taking my question I understand that <unk> is kind of in the background. This year, but I'm wondering if there's maybe an idea of qunar to SCR in sequence and Ted that could be worthwhile to study in the future or are you mainly interested in F CRM opportunities beyond <unk> at this point.
Speaker Change: Yes, it's the latter Joe.
Steve Mahoney: Yeah, it's the latter, Julian. Yeah, we're, we'd, we just don't think, for TED patients. We firmly believe that IGF-1R is the key to that disease or the heart of that disease. That's where cell signaling is taking place. You've got to hit that receptor in order to disrupt it. And, you know, FCRNs, the IL-6s, the other modalities or not, or other mechanisms, we don't feel are on target for moderate to severe TED patients. So, for us, the IGF-1R is the key to TED. So, FCRN, we'll take that in, in different places, as we alluded to in the deck.
Julien: Julien yes.
Speaker Change: We would.
Speaker Change: We just don't take it for for CAD patients.
Speaker Change: We firmly believe that IGF, one or is the key to that disease or the heart of that disease Thats, where the cell signaling is taking place you've got to hit that receptor in order to disrupt that and.
Speaker Change: So CRM the IL six is the.
Speaker Change: The other modalities or not or other mechanisms.
Speaker Change: Feel are on target for moderate to severe Ted patients. So for us the IGF, one or is the key to pet So FCS Rob will take that in different places as we alluded to.
Unknown Executive: In the deck.
Unknown Executive: Thank you.
Trevor Reed: Your next question comes from the line of Trevor I'll read with Oppenheimer. Please go ahead.
Steve Mahoney: Your next question comes from the line of Trevor Allred with OpenEyedMirror. Please go ahead.
Steve Mahoney: Hey guys, good morning. Thanks for taking the question. So with deposit sales trending down slightly, can you give us some perspective on why you think the new start market there appears to be somewhat stagnant and how you see this as a potential opportunity for you?
Trevor Reed: Hey, guys. Good morning, Thanks for taking the question.
Trevor Reed: So it depends on sales trending down slightly can you give us some perspective on why you think the new certain market there appears to be somewhat stagnant and how you see this as a potential opportunity for you.
Trevor Reed: Yes, I mean, I think it's hard for us to comment on Amgen sales.
Steve Mahoney: Yeah, I mean, I think it's hard for us to comment on Amgen sales. I think they are, but they did report on their call last week that they had year over year growth, which is the first time they've done that since the announcement of the merger. So we see that as a really good sign.
Trevor Reed: I think.
Trevor Reed: They are they did report on their call last week, they did report year over year.
Trevor Reed: Growth, which is the first time, we've done that since the announcement of the merger. So we see that as a really good sign.
Steve Mahoney: Amgen was also really confident on their call that they believe that the market continues to be under penetrated, which we agree with. And they believe that it's going to continue to grow. Growth areas also include the other geographies, and the introduction of sub-Q.
Trevor Reed: Amgen was also really confident on their call the day.
Trevor Reed: Leave that the market continues to be Underpenetrated, which we agree with.
Trevor Reed: And they believe that it's going to continue to grow.
Trevor Reed: Growth areas don't forget growth areas also include the other geographies the introduction of sub Q.
Steve Mahoney: And so they've now filed in Japan and Europe, which is good if they're continuing to kind of blaze that trail for us. And, you know, don't forget, even in the backdrop of all of this, they did close to 1.8 billion or close to 2 billion in sales in 2023, in the backdrop of all of this as a first entry. So, again, we feel that there's plenty of room to run TED, not only in the US but elsewhere as well.
Steve Mahoney: And so they've now they filed in Japan, and Europe, which is good as they are continuing to cut our blaze that trail for us.
Trevor Reed: And don't forget even in the backdrop of all of this.
Trevor Reed: <unk>.
Steve Mahoney: They did close to $1 8 billion or close to $2 billion in sales in 2023.
Trevor Reed: In the backdrop of all of this is our first entry so.
Steve Mahoney: And then we think sub-Q, particularly our sub-Q, which we think is potentially going to be best in class where we can have patients that can access it just by delivery at home, and they can self-administer it at home. We think that's a game changer for TED patients. And I think the physician community agrees with us on that. So, yeah, we're not, we're not particularly worried about IGF-1R being the right place for TED patients. And I think Amgen is going to prove that as well.
Steve Mahoney: Again, we feel we feel that there is plenty of room to run in pad not only in the U S, but elsewhere as well and then we think sub Q.
Steve Mahoney: Particularly our sub Q, which we think is potentially going to be best in class.
Steve Mahoney: Where we can have patients that can access it just great delivery at home and they can self administer at home, we take that as a game changer for Ted patients and I think the physician community agrees with us on that so.
Steve Mahoney: Yeah, we're not we're not particularly worried about.
Steve Mahoney: <unk> the right place for for Ted patients and I think Amgen is going to prove that as well.
Speaker Change: Okay, Great. That's super helpful and I guess could you give us some perspective on when we might.
Steve Mahoney: Okay, great. That's super helpful. And I guess, could you give us some perspective on when we might expect to see initial SDRN data with the IND coming in at the end of the year, maybe second half, mid-year 2025?
Steve Mahoney: Checked to see initial let darin data with it with the A&D coming into the year, maybe second half mid year 2025.
Steve Mahoney: Yes in our deck you can see that we've got some we've got some healthy volunteer data that is pegged to the second half of.
Steve Mahoney: Yeah, in our deck, you can see that we've got some healthy volunteer data that's pegged to the second half of 25 for that OO6 program. You know, it's a little ways out, but we'll look to see if we can pull that timeline in.
Steve Mahoney: <unk> 25 for that our <unk> program.
Steve Mahoney: It's a little ways out, but so we will look to see if we can pull that timeline in but.
Steve Mahoney: We feel we're on track for that and then we'll get the healthy volunteer study going and so we'll get some data there I think don't forget the OE.
Steve Mahoney: Human primate data.
Steve Mahoney: That has in other CRM that has proven to be pretty translatable. So we're pretty excited to see that we saw great humanized mice data for OE, but obviously, that's mice data we want to see that in Hps, we will get that in the second half of this year. So we think thats actually a relatively important.
Steve Mahoney: Thing.
Steve Mahoney: For us to get so we're looking forward to F. CRM moving forward, we've got a lot to do there.
Steve Mahoney: Okay, great. Thanks, Steve.
Speaker Change: Great. Thank you.
Steve Mahoney: Alright at this time, we have reached the conclusion of the question and answer session I would now like to turn the call back to Vermilions, President and CEO, Steve Mahoney for closing remarks.
Steve Mahoney: But, you know, we feel we're on track for that IND, and then we'll get the healthy volunteer study going, and so we'll get some data there. I think, you know, don't forget the OO8 non-human primate data that has been in other FCRNs that has proven to be pretty translatable. So we're pretty excited to see that. We saw great humanized mice data for OO8, but obviously that's just mice data.
Steve Mahoney: We want to see the NHPs. We'll get that in the second half of this year. So we think that's actually a relatively important thing for us to get. So we're looking forward to FCRN moving forward. We have a lot to do there.
Speaker Change: Great. Thank you operator, and thanks, everyone for deferred joining the call. This morning.
Steve Mahoney: Okay, great. Thanks, Steve.
Operator: Great, thank you. Sorry, at this time, we have reached the conclusion of the question and answer session. I would now like to turn the call back to Viridian's President and CEO, Steve Mahoney, for closing remarks.
Steve Mahoney: Great. Thank you, Operator. And thanks to everyone for joining the call this morning. We've made a lot of progress, and we are executing, and we're delivering on what we said, and that's important. We know that's important to you as well. So thank you for your participation today, and we look forward to talking to you in the future.
Operator: We've made a lot of progress and we are executing we are delivering on what we've what we've said and thats important we know that's important to you as well so.
Steve Mahoney: Thank you for your participation today and look forward to talking to you in the future.
Steve Mahoney: Okay.
Operator: This concludes today's conference call. You may disconnect your lines. Thank you for participating.
Speaker Change: This concludes today's conference call you may disconnect your lines. Thank you for participating.
Operator: [music].