Q2 2025 Arrowhead Pharmaceuticals Inc Earnings Call
Ladies and gentlemen, welcome to the Arrowhead Pharmaceuticals Conference call.
Throughout today's recorded presentation, all participants will be in a listen-only mode.
Speaker Change: After the presentation, there will be an opportunity to ask questions. I will now hand the conference over to Vincent Loney, Vice President, Vector Relations for Arrowhead. Please go ahead, Vince.
Vince Anzalone: Thank you, and good afternoon everyone. Thank you for joining us today to discuss Arrowhead's results for its fiscal 2025 second quarter and its March 31, 2025.
Speaker Change: With us today for management, our president and CEO , Dr. Chris Anzalone, we will provide an overview. Dr. Bruce Given, in term chief medical scientist, who will provide an update on our Cardi Metabolic pipeline. Andy Davis, senior vice president and head of global Cardi Metabolic franchise.
will provide update on commercialization activities.
Speaker Change: Dr. James Hamilton, Chief Medical Officer, and Head of R&D, who will discuss our earlier stage development programs.
Speaker Change: and Ken Myszkowski, our outgoing Chief Financial Officer who is retiring this week, who will give a review of the financials. We also welcome Dan and El, our incoming CFO , who is also with us on the call today. Following management's prepared remarks, we will open the call to questions. We will open the call to questions.
Speaker Change: Before we begin, it would like to remind you that comments made during today's call contain certain forward-looking statements within the meeting of Section 27-8 of the Security's Act of 1933 and Section 21-E of the Security's Exchange Act of 1934.
Speaker Change: All statements other than statements of historical fact are forward-looking statements and are subject to numerous risks and uncertainties that could cause actual results differed materially from those expressed in any forward-looking statements. [inaudible]
Speaker Change: For further details concerning these risks, please refer to our SEC violence, including our most recent annual report on Form 10K and our quarterly reports on Form 10Q. I'd now like to turn the call over to Chris Anzalone President and CEO of the company, Chris.
Chris Anzalone: Thanks, thanks. Good afternoon, and one thank you for joining us today.
Chris Anzalone: Before we start, go and say thank you to Ken and wish him the best in his retirement.
Speaker Change: Kenneth has been a valuable member of the Arrowhead Team and he retired at a time of great financial strength, the company.
Speaker Change: The financial organization that can build over the years is very capable and provides strong support to our ambitious development and commercialization plans. From all of us at Arrowhead, thank you for all the important contributions over the last 15 years.
and others.
Speaker Change: I'm also excited that Dan and Bell will join us as our new CFO at a critical time for Arrowhead. We expect to make the transition from development stage to commercial stage with a planned launchable disaster end this year, pending regulatory review and approval.
Speaker Change: Dan from the countless pharmaceutical executive who can make immediate and important impact on our business.
Speaker Change: Let's talk about our business and the progress we've made toward our short, mid and long-term goals.
Speaker Change: Arrowhead is at an important point both in terms of capabilities and potential value as we drive our organization toward our first commercial launch which we anticipate this year. Following this, we expect multiple additional independent and partner launches over the next few years. This is the first commercial launch which we anticipate this year. This is the first commercial launch which we anticipate this year.
Speaker Change: The combination of commercial expansion are extraordinarily productive discovery engine, the increasingly validated nature of our platforms and already eye modality, our large pipeline of clinical stage assets.
Speaker Change: Our strong balance sheet and clear access to additional non-deleted capital together to provide us with a level of upside potential and stability that I believe is a priority in our industry.
Speaker Change: This is always attractive, but it's even more valuable at a time when biotech markets have been depressed for the past several years, and the near-term capital markets are uncertain at best.
Speaker Change: As the current biotech market weakness causes people to weigh the trade-off of stability versus the potential for its fullest value growth. I think we have the tools for both.
I view our value proposition in layers.
Layer 1 is Potassium.
Speaker Change: Constitutes our primary near-and-bitcher and value driver and provides a strong base for us.
Speaker Change: Well, that's a rant that is shown to be a potent triglyceride lowering agent across the multiple clinical studies in hundreds of patients.
Speaker Change: We believe there are three to four million people in the U.S. alone who suffer from severe hypertroglyceridemia or SHTG, as defined by fasting triglyceride levels above 500 milligrams per deciliter.
Speaker Change: We are very to launch into a small subgroup of this population. Patients with familial pilot Micronesia Syndrome, Lawrence, yes, and have a good day of November 18, 2025.
Speaker Change: We also complete the submission of the Marketing Authorization Application, or MAA, with the E&I, and are working through additional plan submissions in other select geographies.
Speaker Change: The Phase 3 data supporting our regulatory solutions will consistence and encourage you.
Speaker Change: Genetically fine and clinically defined, FCS patients responded similarly with reductions in triglycerides of about 80% from baseline [inaudible]
Speaker Change: Approximately 75% and 50% of patients with head diagnostics go below 80% and 500 milligrams [inaudible]
Speaker Change: with your discussing guidelines and the academic literature as important goals for minimizing pancreatitis risk.
Speaker Change: These are truly impressive levels to achieve in FCS patients as the main baseline to try to destroy a level in the study, was approximately 2500 milligrams per deciliter.
Speaker Change: Oda Soria was generally well-polaried, and Joe Trego, sorry, with options in 100% of patients treated at the primary end point of 10 months.
Speaker Change: Our hope of treating FCS patients is important. This is an historically underserved population and we believe the disaster and could be an important medicine for that. However, we do believe this is just beginning.
Speaker Change: Chesa 3, Chesa 4, and Bureau 3 are phase 3 studies designed to support a supplemental NDA and other applications on a global basis to enable us to treat the broader SHTG population.
Speaker Change: These studies are moving rapidly, and we believe they could be fully enrolled this summer. We are also in the process of initiating Chastified, which is an outcome study to specifically evaluate the risk reduction of acute pancreatitis in high risk patients with FHG. [inaudible]
Speaker Change: We think this is an innovative strategy to potentially demonstrate meaningful value for patients, physicians and parents.
Speaker Change: Our second layer of value may be our initial obesity candidates and initial CNS gains.
Speaker Change: Regarding the former, Arrow-I-N-H-B-E is currently docentino-beast patients and we expect Arrow out of seven to begin docentino-beast patients shortly.
Speaker Change: Both are designed to intervene in a biological pathway, regulating bad storage, airline HPE targets out of sites with the same trim platform, using several ongoing clinical studies, and has been thousands of patients [inaudible]
Speaker Change: The design to reduce the parasite expression of active and heat, which is a ligand for adipose health seven.
Speaker Change: Arrowout 7 is the first out of a different site targeted S.I.R. and Hague with a new trim platform that in animal models has shown good up taking out of post-issue and high levels of target team knocked down with a long direction above the main enable of Q4 month, Q6 month, or less frequent administration. [inaudible]
Speaker Change: Parallel ALK7 is designed to reduce the depression of the ALK7 receptor itself in adipose tissue.
Speaker Change: Both programs demonstrate the stance of reductions in visceral fat versus control while simultaneously preserving lead mass in animal models.
Speaker Change: Both targets are also supported by human genetics where lots of functioned carriers have favorable body composition and metabolic characteristics compared to non-carriers without any apparent safety costs.
Speaker Change: It's a very treating pathway that we believe that they fill some recording apps left by standard of care of EC treatments, directions filled with shortcomings, the GLT-1 GIP plus.
Speaker Change: The possibility of a long acting agent that are well tolerated, spare muscle mass, and enabled visceral fat loss without dependence on the floor of restriction. It's like arrowhead Nakae began dosing a phase 12 study in December 2024, and we need to stay having some initial data by the end of 2025.
Speaker Change: As I mentioned, we've picked Arrow out seven to begin dose shortly, and we should have some initial days soon after I've arrowed on HPE results that are available.
Speaker Change: Studies in both candidates include single dose and multiple dose, model therapy, R&B subjects. [inaudible]
Speaker Change: as well as multiple Joes' arms and clue combinations with your lemon nut. [inaudible]
Speaker Change: Arsenae Speedy Platform, has made great strides in recent years. We have a substantial amount of pre-medical data across multiple animal models that make us optimistically to deliver potent RNA-address to the brain to be a simple, self-attentious injection.
Speaker Change: Blurring large molecule drugs systemically and getting past the blood-braining barrier, has been only grailed virtually as long as complex biological drugs have been developed, and we expect to be in the clinic late this year.
Speaker Change: Operous Candid, Arrownaftee, Tarty The Tau Pro-To you for potential treatment of all times. David Lebowitz, David Lebowitz, David Lebowitz,
Speaker Change: We expect to follow that with AIR-HTT, Life and Cicerecta against Huntington's disease by the end of the year.
Speaker Change: In the first half of 2026, we expect to bring Arrow, SNCA to the clinic, which started out as a nucleine for potential treatment of Parkinson's.
Speaker Change: These are all well-validated targets against very important diseases for which effective agents have long been sought and we look forward to seeing how they translate from animals to humans.
Speaker Change: A third layer of value could come from our other Phase 3 drugs. We expect to begin and roll in a year-long Phase 3 study as our DASRAN for homo-vegus familial hyperclosteroleum cancer or H.O.F.H. shortly.
Speaker Change: The H.O.F. patients, excuse me, the H.O.F. patients treated, but stood after him in phase 1 and phase 2 studies, human covenants that they may have ate.
Hoden LVL
Speaker Change: I'm sorry that it may have a vote of LDLC lowering agent that only requires quarterly dosing in this important
Speaker Change: The sale of the structure will be your building for the disaster and could easily be leveraged post-pocketly.
Speaker Change: So this feels like straight forward, relatively rapid, low risk and low cost expansion of our commercial presence.
Thanks for ending our drug channel against A.T. Liberties.
Speaker Change: Our prior studies give us confidence that it could be an effective agent to reverse fibrosis in this largely uncertain patient population.
Speaker Change: Lisa Ren, is part of the Decade, and they have publicly guided the phase-free studies to a complete enrollment this year. They are two year studies to primary employment.
Speaker Change: While this is partner, our economics are substantial, with 50-50 profit share in the US, 20-25 percent of oil, the XUS, and up to $527 million per million in milestones.
Speaker Change: While we view these as our primary nearing midterm value drivers, there are substantial pieces of our business underneath them providing redundancy and additional outside potential.
Speaker Change: They couldn't fool wholly on the additional phase two ready clinical programs in ARSB-3, ARSB-FB, Arrow-Rage, and Arrow-P-U-P-L-A-3
Speaker Change: Two big two programs partnered with GSK, against chronic hepatitis B infection in match.
Speaker Change: Another phase three program, part of the engine in Old passer end. Four phase one, two clinical programs, part of the Serefta. Three designated pre-clinical programs, part of the Serefta, to one of which I already mentioned in HTTP. And six additional pre-clinical programs, to be named by Serefta. And six additional pre-clinical programs, part of the Serefta.
Speaker Change: And, of course, underlying all of this is the discovery engine that we believe is second to none in the S-I-RNA field. The expected to continue to drive value at the basis for many additional holy undrugs and through future partnerships.
Speaker Change: All these layers of what can really last counting of these would be required to create a large, productive, sustainable, pharmaceutical company. We indeed have many opportunities to create durable value.
Speaker Change: Importantly, we believe we have the capital to access this two substantial additional capital to support our work.
The threat to deal was a critical component of this.
Speaker Change: During the last quarter, we closed the global license and collaboration agreement with Srupta Therapeutics, materially strengthening our value.
Speaker Change: The transaction brought in $500 million as an upfront payments, and through either $25 million through the purchase by Sirepda, a very common stock at $27.25 per share.
Speaker Change: Arrowhead will also receive $250 million to be paid in annual installments of $50 million over five years.
Speaker Change: In short term, we have potential to receive an additional $300 million in milestone payments associated with the continued enrollment of the phase one, two, study of arrow, key, one, which we are on track to achieve during the next report
Speaker Change: Taken together, this adds up to $1.375 billion in cash payments.
Speaker Change: The total pinch of value of the steel including upfront payments, equity investments, and potential milestones exceeds $11 billion.
Speaker Change: We are all available to receive pure royalties on 26. Thank you.
This will be a transformational deal in any of our...
Speaker Change: As I mentioned with the state of biotech equity markets today, we feel very good about not having to raise equity capital at this time to fund our growth as we become a commercial comment.
Speaker Change: We are now funded in June 20, 2028 and through multiple important milestones that we think can drive substantial value for our shareholders. That overview, I now like to turn the color of your brusket at Bruce.
Thanks Chris, and good afternoon everyone.
Speaker Change: Carelhead has been working in R&I interference for nearly 20 years. [inaudible]
Speaker Change: During that time, we have made great strides creating a modality that is increasingly scalable, reliable, potent, and generally well tolerated.
Speaker Change: We've also made great strides bringing RNAi to where it is needed, in addition to delivering to the parasites, we are now able to address lung, CNS, muscle, adipose, and cardiomyocytes. [inaudible] to where it is needed, in addition to delivering RNAi to where it is needed
We have always been a great R&D platform company.
Speaker Change: and we are now taking the next step forward and as we seek our first marketing approval for Kuzasra and if you familiar kind of like beneath me you say you're more SPS.
Speaker Change: Most of you will be aware of the results of our Phase 3 policy study, which was published in the New England Journal of Medicine last year,
Speaker Change: Including a large reduction in the primary endpoint of triglyceride reduction in 10 months, as well as reduction in incidence of confirmed pancreatitis in the pro-qualified comparison placebo and the combined 25 and 50 milligram dose groups.
Speaker Change: Following pre-NDA discussions with the FDA, a marketing application for approval for using the SDS was submitted on November 16, 2024, and accepted for review by FDA with the due date of November 18, 2025.
Speaker Change: At this time, we are not anticipating being asked to participate in the advisory committee. [inaudible]
Speaker Change: We can frequently frequently ask whether the changes in Washington have impacted the review.
Speaker Change: While we have no special insights into the inner workings of the agency, our impression has been that the review is progressing as we would have anticipated, and we know of no FDA personnel or timing changes affecting our program at this time.
Speaker Change: We have also have routine pre-finement meetings with EMA and appointed CHMP Repetors that culminated in the submission of a marketing authorization application or MAA of February 28, 20, 2025, which was confirmed to be valid for review on March 20, 2025.
Speaker Change: Our plan is to seek approval in the UK following approval in either the U.S. four-year by leveraging the International Recognition Procedure.
Speaker Change: We've also had pretty filing meetings with the Canadian and Japanese regulatory authorities that have planned filing marketing applications in those and other jurisdictions as well.
Speaker Change: We're hopeful that these findings will lead to Arrowhead's first commercial watch, possibly beginning as early as late this year.
Speaker Change: As welcome as we believe that closed aspirin will be for STS patients, this is just the beginning of the story for this important drug. While we are studying closed aspirin and STS, we are also evaluating the drug in much larger patient populations, including severe hydropeglyceride immune or STG. This is just the beginning of the story for this important drug.
Speaker Change: C, to find this patient's capacity, that triglycerides above 500 milligrams per just one year, but without genetic estimates.
as well as the patients with mixed hyperlipidia. [inaudible]
Speaker Change: These important studies led to publications last year in the New England Journal of Medicine,
Speaker Change: After receiving end of phase two feedback from FDA and EMA, we have initiated a phase three program in severe end of the service for the EMF. This program is designed to meet key standards based on guidance documents.
from the International Council organization.
Speaker Change: Key requirements and droid design considerations include two pivotal placebo control trials in FHCG patients and a safety database of at least 1500 patients treated with aspirin, care of the placebo for 12 months.
Speaker Change: Hence, she has a three and four of her very similar studies designed to demonstrate to the police that you're going to improve with triglycerides with 25 milligrams of potassium, and the hair of the placebo over 12-month treatments.
Speaker Change: The two tribes hold the ground seven indigenous to a higher than time, given the results of two indigenous two-shots to teach that out, where the primary endpoint difference is that this one is at 2.4% of the baseline, at 2.5% of those indigenous was minus 53%, where the key value is less than two indigenous and less than that of that. To reach the necessary 15 indigenous species, safety distance, chances 3 and 4 are supplemented by a supportive year of three tribes mixed up the indigenous the indigenous and less than three tribes mixed up the indigenous
Speaker Change: Also landed at the Carey 25-month-olds, Quirin, Disassar, and Christopher Seable, for one year, with a planned enrollment of about 14 hundred patients. David Lebowitz, David Lebowitz, David Lebowitz, David Lebowitz,
Speaker Change: We have previously guided them to expect the last patients we randomized in the S&CG program this year. Based on enrollment today, we now anticipate the last patient to be enrolled sometimes in summer.
Speaker Change: We've been encouraged by the enthusiasm of our investigators and indicated by the rapid enrollment and also by a very low premature discontinuation rate for adverse events and other reasons.
Speaker Change: The last patient's enter will be treated for one year before the database can be locked, the data analyzed, and hopefully submissions made to regulatory authorities seeking approval for use in FHGG patients
Speaker Change: Thus, before the end of the summer, it should be possible to narrow the potential timing for FHTG Supplemental FDA submission.
Speaker Change: The SHTG program also features, in first this kind of study, named Shastified, to directly assess the ability of possessor and to reduce the risk of acute pancreatitis in SHTG patients with a purpose designed
Speaker Change: We are conducting this study to express the need to meet the needs of sophisticated payers, especially outside of the U.S. It is not gaining the number of CG filings, it is unlikely to be complete part of a submission.
Speaker Change: We do hope that as we complete this during the review, topic, and drill, that Elstert is the available price of the sessions that much disaster is supposed to occur there. We can that provide the site title of some cases. Not only because of uncertain timing, the enrollment can also become, as an outcome study, treatment will continue until required number of events are collected.
Speaker Change: Grabberville, Perstix, Jason, Screege, and Underway, and at center's hurtly open front.
The Trials and...
So the trial is underway.
Thank you.
Speaker Change: The R&D group is also playing its role in growing the awareness and nutrients of reaching the market for treating FCS.
Speaker Change: Our Medical Affairs team, reporting into R&D, continues to play a vital role in educating medical communities. Our medical side plays eyes are actively engaging healthcare professionals in scientific exchange, helping them better understand and raising awareness of familiar kind of equity with the syndrome. The significant unmet medical needs and the growing body of clinical data now available. [inaudible]
The team has been present at key medical conferences. [inaudible]
Speaker Change: In the during the last quarter, including the American College of Cardiology meeting, the European Health and Sclerosis Society, and British Health and Sclerosis Society joint meeting, and the European Society and the Penology meeting taking place presently.
Speaker Change: In parallel, our publications team continues to generate disseminated important data to support these scientific efforts and expand awareness within the clinical community.
Speaker Change: Close to recently NBAS, an abstract authored by experts in the field, Tyler Palisay, to as asher and decreases risk of acute pancreatitis and improves indices of quality of life and SDS will teach you.
Speaker Change: The authors concluded in the abstract that in patients with STS, was that strand markedly reduced triglycerides and risk-appropriate titus, with promising changes in indices for quality of life.
Speaker Change: Those of you who have watched this for a while, know that we have another agent in our curfew in their bottle of ice, like what was asked for at this very strong support from Team Genetic. [inaudible]
Speaker Change: I'm describing as a dance ran in the R&I drug design to reduce expression of Andrew Appointing protein like three or NHP two or three. This drug also produced strong results in two-based twos, including artist two in mixed hyperlipidemia, also published in the New England Journal of Medicine. This drug also produced strong results in two-based twos,
David, as well as the Gateway study in intro at age patient.
Speaker Change: with David presented last week at the European Alpha Sclerosis Society conference. David Lebowitz, David Lebowitz, David Lebowitz,
Speaker Change: However, as we've discussed previously, we saw as a dastro in this position in the crowded LDL space where the unmet medical need has largely been addressed by statins and PCS cadating computers.
Speaker Change: But there is an important population where we think so, Dastrad would make an important contribution without requiring an outsized commitment of resources by Arrowhead, just being prepared for patients from home designer's familial hypercluster in England.
Speaker Change: These patients have exceptionally high LDL cholesterol levels, and because of genetic abnormalities, usually result in very low or absent LDL receptor function, are usually not able to get to goal LDL levels, even with maxill statin and PCC
Speaker Change: Dr. Davis, Dr. Davis, Dr. Davis, Dr. Davis, Anybody who gets S-P-2-3 has already been proven effective in this patient.
Speaker Change: The required monthly intravenous infusions, it can lead to genealogy reactions. We conducted an explanatory phase to study in this population and saw a similar benefit with convenient quarterly subcutaneous dosing.
Speaker Change: Moreover, these patients are usually cared for by physicians which largely overlap the physicians that treat STS and SHTT, making the potential marketing of Zastrad and these patients efficient for us, going to be approved.
Speaker Change: We have designed a phasedry study of similar size to our phasedry palisate study compared to the S-ray at 200 milligrams quarter minutes of placebo and expect to be in lower patients this year, assuming successful demonstration of safety and efficacy and successful regulatory submission.
Speaker Change: So the aspirin could join, plus aspirin in the market as early as 2028 or 2029. [inaudible]
Speaker Change: As I said at the beginning of my remarks, we hope to see possessor and emerges our first commercially available drug to treat STS patients as humans this year.
Speaker Change: Based on our expected completion of enrollment in the SHTG phase 3, you know, studying the summer, we could see SHTG phase 3 completion in the summer of 2026, and an SAA, and an AND and an SAAEA pilot insurgent thereafter.
Speaker Change: Interestingly, also important completion of the decade, based on public guidance, progress from face-to-face program to program for a pass-round, and okay, relay, and physician-hand, and liver disease, associate with alcohol-related trips and disease, could result in improvements for these programs in a similar type of plan. David Lebowitz, David Lebowitz, David Lebowitz, David Lebowitz
Speaker Change: As well as with the possibility that some of our less-catured, partnered programs for important organ diseases might also fight approval at that time.
Speaker Change: It takes patience in this business to see an important new platform to arise requirements. [inaudible]
Speaker Change: We feel there are some good reason to have confidence in our name or fear to enjoy small molecule drugs and multiple antibodies as a foundation of technology and drug development, especially as the field, which we see Arrowhead still leading, continues to push into new cell types, opening up additional importance to these key interests. [inaudible]
I'll now turn the call over to Andy Davis.
Andy Davis: Thank you, Bruce. With the split of the day for disaster and sector in November 18, just six months away, I'm pleased to share that our commercialization efforts are advancing rapidly and with strong momentum.
Speaker Change: As discussed by Bruce, the medical affairs team is in the field helping educate the physician universe and facilitating publication of the results of Arrowhead's clinical trials.
Speaker Change: We're also making strong progress in building our commercial sales team.
Speaker Change: The National Sales Leader and a full complement of regional sales leaders are now on board and focused on hiring and onboarding top tier town with deep rare disease and therapeutic area expertise.
Speaker Change: Interest has been very encouraging with thousands of resumes in the queue, and we're on track to fully hire and train our sales force by late summer, ensuring ample time for part-evaliation and disease and education in advance launch.
Speaker Change: A market access team is executed effectively on a pre-approval information exchange or pie, strategy.
Speaker Change: Directed toward healthcare decision makers to help them plan for our potential approval. We've now engaged with payers representing a significant number of U.S. covered lives, delivering compelling content on the clinical value and anticipated profile of the disaster.
Speaker Change: We're encouraged by their interest in plasastering, especially regarding its potential to reduce dreadless rise and the cute pancreatitis risk
Speaker Change: Additionally, our analytics team is deploying innovative technologies to identify individuals potentially living with STS. We're connecting with these potential patients through disease and education efforts, including opportunities for them to opt in for continued communication and support.
Speaker Change: Across our research and stakeholder engagement, the clinical attributes of Plasasterin continue to resonate strong. Our market research suggests that Plasasterin's e-endurable triglyceride reduction is compelling to numerous stakeholders.
Speaker Change: The Palafate study show that was asked to reduce triglycerides by approximately 80% for baseline as early as month one.
Speaker Change: With this effect sustained over 12 months and with limited variability, while placebo subjects showed variable changes ranging from plus 10 to minus 18%
Speaker Change: As a reminder, the primary endpoint in policy was the median percent change from baseline and fasting triglyceride levels at month end. The plus assuring demonstrated the placebo-adjusted change of minus 59 percent with the planned commercial 25-minute grand dose.
Speaker Change: Our market research also suggests that health care providers, caregivers, and patients have a strong desire to see triglyceride levels fall below expert guideline threshold threshold, such as 880 milligrams per deciliter, and even 500 milligrams per deciliter
Speaker Change: In Palisade, approximately 75% of patients at the 25 milligram dose achieved levels below 880, and approximately 50% achieved levels below 500
Speaker Change: Numerous expert guideline emphasized the importance of maintaining triglyceride levels below 500 milligrams per deciliter as the aspirational goal to reduce acute pancreatitis risk.
Speaker Change: Finally, we receive feedback that patients are looking for a treatment option that minimizes disruption to their lives.
Speaker Change: Plasasserine shows a favorable docine safety profile, and as a reminder, Plasasserine is conveniently administered once every three months, potentially minimizing treatment burden and improving adherence.
Speaker Change: As our US launch preparations continue at full speed, we're equally pleased to report steady progress on our European commercial efforts as well.
Speaker Change: We've already established a field medical presence, interactively engaging in scientific exchange at sea European scientific meeting. Lay the strong foundation for a successful rollout.
Speaker Change: We remain on track and deeply motivated by the opportunity to bring investigational pizzazz turn to individuals living with FCS and their families in both the United States and priority countries outside the United States. We believe this potential first in class <unk> RNA therapy will mark a major advancement and we're fully committed to unlocking its patient impact.
Speaker Change: I'll now turn the call over to Jamie Henderson.
Jamie Henderson: Thank you Randy first I would like to provide a status update on our two early stage of BCE programs Aero activity and Aero et cetera.
Jamie Henderson: Aero inhibiting <unk> is designed to reduce expression of activities, which is a ligand adequate outset, well Aero Alex <unk> is designed to reduce expression at the outset.
Jamie Henderson: Receptor itself.
Jamie Henderson: Both of which are involved in regulating adipose storage effects.
Jamie Henderson: These programs have the potential to reduce visceral fat mass while simultaneously preserving remix, which we demonstrated in preclinical models.
Jamie Henderson: And are now evaluating and clinical studies.
Jamie Henderson: Irwin hip and knee began dosing in December of 2024 and is progressing on our planned timeline.
Jamie Henderson: As a reminder, this phase one two study will evaluate aero inhibitor monotherapy administered to obese otherwise healthy volunteers in both single and multiple dose escalation cohorts.
Jamie Henderson: This age cohort dosing is now complete and the multi dose monotherapy cohorts are actively enrolling.
Jamie Henderson: The study is also evaluating multiple doses of <unk> inhibitor <unk> in combination with <unk>. In these combination cohorts are actively enrolling now on our planned timeline.
Jamie Henderson: We anticipate the arrow out seven clinical program will be up and running shortly.
Jamie Henderson: The design of this study is very similar to the airline <unk> study with sad and Mad monotherapy cohorts, the mad cohorts in combination with <unk> appetite.
Jamie Henderson: This study will also enroll abuse otherwise healthy volunteers.
Jamie Henderson: Both studies are designed to assess safety tolerability pharmacokinetics, pharmacodynamics and multiple exploratory obesity efficacy endpoints.
Jamie Henderson: We anticipate that some initial data may be available for arrow in hip and knee around the end of 2025 and potentially for <unk>. Shortly thereafter.
Jamie Henderson: Our muscle targeted programs partnered with Sorretto Aero ducts floor for <unk> and <unk> one for my Atonic dystrophy type. One also continued to make good progress in the phase one two studies, which are ongoing.
Jamie Henderson: While the decision to release data will be made jointly with <unk>. It is our expectation that initial data release as possible in 2025.
Jamie Henderson: Lastly, I want to highlight some topline results from part two of the phase one two clinical study of <unk> three designed to reduce liver production of complement component three as a potential therapy for various complement mediated diseases.
Jamie Henderson: In patients with Iga nephropathy, or IGN aerospace three achieved deep and sustained reductions in alternative pathway of complement activity and protein area.
Jamie Henderson: The Max need reductions in Q3 was 89% serum age age 50, which is an alternative pathway hemolytic assay that was used by 85%.
Jamie Henderson: <unk> three also led to an important improvement in proteinuria.
Jamie Henderson: A mean reduction in spot PCR of 41% and a maximum individual reduction of 89% from baseline up through week 24.
Jamie Henderson: <unk> three was generally well tolerated and the observed duration of effect is supportive of once every three months or less frequent subcutaneous dosing.
Jamie Henderson: We are very pleased with these results and are planning to present Fuller dataset at the upcoming European Renal Association or <unk> Congress in June.
Jamie Henderson: I will now turn the call over to Ken Moskovsky.
Ken Moskovsky: Thank you James and good afternoon, everyone as.
Ken Moskovsky: As we reported today, our net income for the quarter ended March 31, 2025 was 370.
Ken Moskovsky: $4 million.
Ken Moskovsky: Or $2 75 per share based on a record $34 5 million fully diluted weighted average shares outstanding.
Ken Moskovsky: This compares to a net loss of $125 3 million or $1 two per share based on $123 3 million fully diluted weighted average shares outstanding at quarter ended March 31 2024.
Ken Moskovsky: Revenue for the quarter ended March 31 from 25 was $542 7 million.
Ken Moskovsky: No revenue was recorded in the quarter ended March 31 2024.
Ken Moskovsky: Revenue in the current period relates to our license and collaboration agreement what's directly.
Ken Moskovsky: Yes.
Ken Moskovsky: As you know the agreement with director with significant liquidity banking assets, including critical access preclinical assets and other assets to be developed.
Ken Moskovsky: Tommy guidance requires that we allocate consideration to several items, including the value of the licenses we try expert.
Ken Moskovsky: Ongoing work of certain clinical trials that we will oversee.
Ken Moskovsky: Well as expected obligations regarding future assets to be developed.
Ken Moskovsky: Initial fixed contract revenue to reallocate it included the upfront payment of 500 billion.
Ken Moskovsky: The premium paid on the stock purchase of $84 million and.
Ken Moskovsky: And $250 million relate.
Ken Moskovsky: Related to the five year annual milestone payments.
Ken Moskovsky: This totals to about $834 million majority of which was allocated to the license agreements are recognized immediately.
Ken Moskovsky: And the balance will be recorded as we fulfill our performance obligations.
Ken Moskovsky: We recognized revenue of $542 7 million during the quarter ended March 31, 2025, and we expect the barrel distribute recognized over the period that we satisfy these performance obligations.
Ken Moskovsky: These applications include overseeing certain clinical trials as well as performing R&D work related to future clinical candidates.
Ken Moskovsky: We expect $90 million to $125 million of revenue to be recognized over the next 12 months solely related to the revenue recognition of the initial fixed contract revenue.
Ken Moskovsky: The balance will be recognized over the next five years five or so years, most of which will be recognized in the first half of that time period.
Ken Moskovsky: We also expect future revenue related to cost reimbursement for certain discovery and manufacturing activities.
Ken Moskovsky: Future near term milestones are $300 million related to the <unk>. One program are expected to be earned in the next few quarters and will be recorded in their entirety as revenue at that point.
Ken Moskovsky: As with any other future milestone payments and royalties.
Ken Moskovsky: Total operating expenses for the quarter ended March 31, 2025, $461 5 million.
Ken Moskovsky: Compared to $126 2 million for the quarter ended March 31 2024.
Ken Moskovsky: Key drivers of this change for increased candidate costs at the company's pipeline clinical candidates.
Ken Moskovsky: Increase in advance into later stages of development.
Ken Moskovsky: Net cash provided by operating activities. During the quarter ended March 31, 2005 was $460 1 million.
Ken Moskovsky: Paired with net cash used in operating activities of $32 4 million for the quarter ended March 31 2024.
Ken Moskovsky: Increase in cash provided by operating activities was driven by the cash received for the <unk> agreement.
Ken Moskovsky: Turning to our balance sheet, our cash and investments totaled $1 1 billion at March 31 2025.
Ken Moskovsky: Our common shares outstanding at March 31, 2025.
Ken Moskovsky: Inventory was $38 1 million.
Chris Anzalone: With that brief overview ill turn the call back to Chris.
Chris Anzalone: Thanks, Ken.
Chris Anzalone: Arrowhead is in a strong and stable position of the business and we've made meaningful progress toward our long term goal of developing and ultimately commercialize new innovative medicines for millions of patients.
Chris Anzalone: We are on schedule to launch was accurate this year pending regulatory approval.
Chris Anzalone: With what we think is the best in class profile with meaningful differentiation from currently available therapies and Fcs.
Chris Anzalone: Yes.
Chris Anzalone: We are also well on our way to fully enrolling this summer our suite of Phase III studies designed to support regulatory submissions for the large <unk> patient population.
Chris Anzalone: We are funded into 2028 and potentially through multiple launches of wholly owned and partnered programs in late stage development.
Chris Anzalone: We believe our technology platform is the broadest investment yield given us many opportunities to receive additional capital inflows from business development in areas that are outside of our core commercial focus.
Chris Anzalone: Thank you for joining us today, and I would now like to open the call to your questions.
Chris Anzalone: Thank you to ask a question. Please press star one on your telephone keypad and wait for your name to be announced.
Chris Anzalone: Draw. Your question. Please press star one again in the interest of time, we ask that you. Please limit yourself to one question. Please standby, while we compile the Q&A roster.
Maury Raycroft: And our first question comes from Maury Raycroft of Jefferies. Your line is open.
Maury Raycroft: Hi, Congrats on the progress and thanks for taking my question and best wishes to Ken and now welcome Dan.
Maury Raycroft: <unk> four.
Speaker Change: <unk> inhibits <unk> and seven <unk>.
Speaker Change: You've noted that the goal there is not to compete with glick ones, but to be used in combination.
Speaker Change: What's the latest you're seeing and how you are setting expectations for initial monotherapy and potential combo data.
Speaker Change: What do you want to see in the initial update including changes on weight loss body composition and relevant biomarkers.
Speaker Change: Yeah. Thanks, Thanks Marty.
Speaker Change: Not giving any any.
Speaker Change: Sure.
Speaker Change: Any guidance on what we expect to see because this is the first in human study in the animal data were compelling we saw good weight loss.
Speaker Change: As a monotherapy, we saw good weight loss and in combination with <unk> appetite.
Speaker Change: As you know this is a new pathway that we think could fill some of the massive amounts of white space in the <unk>.
Speaker Change: Current standards.
Speaker Change: So look we're looking forward to see what we see.
Speaker Change: I think the REIT.
<unk> is here, we think are potentially not just weight loss, but quality weight loss are we seeing philosophies or all of that are we seeing.
Speaker Change: Retention of lean muscle mass, we saw that in animal models, we hope to see that.
Speaker Change: And that in humans as well also in animals we.
Speaker Change: We did not see this was not the result of caloric restriction. It was it was in the context of normal feeding that would also be that sort of thing would be helpful.
Speaker Change: Let's see what we see I think we'll have our first flooding data towards the end of this year and then should be data rich.
Speaker Change: Virtually every quarter for the next year or so after that.
Got it that's helpful. Okay. Thanks for taking my question.
Speaker Change: You're welcome.
Speaker Change: You.
Speaker Change: And our next question comes from Jason <unk> of Bank of America. Your line is open.
Speaker Change: Hey, guys. Thanks for taking my question.
Speaker Change: Ahead of the positives here and FDA review decision.
Speaker Change: Wondering how you think about the robustness of your pancreatitis data and how that might look or feel a little bit differently in the package insert.
Speaker Change: I noticed <unk>.
Speaker Change: Just as I mentioned of the numerical incidents reduction in the clinical section and I'm wondering if you guys feel like based on policy that you might get maybe a more robust upfront.
Speaker Change: Front page reference to the pancreatitis benefit.
Speaker Change: Anything you can just offer on the <unk>.
Speaker Change: Aspects of label differentiation would be much appreciated thanks.
Bruce Given: Yes, Hi. This is this is Bruce.
Speaker Change: Yes, we haven't had any labeling negotiations with the FDA at this point, which as you would expect.
Speaker Change: Still in the midst of the review process and labor negotiations come later so.
Speaker Change: It's really impossible for me to ask.
Speaker Change: The estimate.
Speaker Change: Ill.
Speaker Change: How do they maybe the states.
Speaker Change: We we obviously like our data quite a bit we did our study differently in that.
Speaker Change: We have looked at in young patients.
Speaker Change: Firmed.
Speaker Change: Equity tied to <unk>.
Speaker Change: Criteria.
Speaker Change: While the oldest units.
Speaker Change: As they develop the scale of that.
Speaker Change: Included possible or probable.
Speaker Change: Pancreatitis, which actually don't meet.
Speaker Change: Atlanta criteria as.
Speaker Change: Separately pancreatitis.
Speaker Change: So frankly, it's a little bit of an apples and oranges comparison.
Speaker Change: Don't know how that.
Speaker Change: How that might play out.
Speaker Change: Eric labeling discussions.
Speaker Change: Yes.
Speaker Change: Or even in the market, but it is.
Speaker Change: It's a little bit apples to oranges.
Speaker Change: I guess, what you might say is a more conservative route.
Speaker Change: So we think especially at Christmas with payers and in Europe, especially that might turn out to be important.
Speaker Change: But.
Speaker Change: It's clear that these drugs are extremely helpful. Just introducing triglycerides, but but also.
Speaker Change: Turning to debt reduction triglycerides into which is to download painting pancreatitis. So yes.
Speaker Change: Let's see.
Speaker Change: I can't give you any guidance for what Lee was going to look like.
Speaker Change: Yes.
Speaker Change: And our business.
Speaker Change: We do the studies we submitted.
Speaker Change: Where the FDA reviews of the FDA decides.
Speaker Change: Ultimately, it's going to be up to them.
Speaker Change: We will obviously discuss it with them.
Speaker Change: I can't speak for them.
Speaker Change: And let me just add here more broadly the biology here is clean as clear in these patients the higher the triglycerides the higher the risk of pancreatitis, the higher the risk of abdominal pain and so the goal here is to get triglyceride as well as in Canada.
Speaker Change: So while we are monitoring for pancreatitis and of course, <unk> and <unk>.
Speaker Change: And.
Speaker Change: And the shafts three and four studies, we are looking at it.
Speaker Change: Donald.
Speaker Change: The real thing to focus here on is how low can we get taken us triglyceride and as mentioned in the prepared response prepared remarks as well.
Speaker Change: In the past.
Speaker Change: We get something like 75% of patients in that phase III study below $88 million.
Speaker Change: And something like 50% below 500, if that's a real feat for these FCS patients who as we mentioned came into our study with a mean triglyceride level of 2500, I think that that's what we need to focus on here.
Speaker Change: Got it.
Speaker Change: Thanks, guys.
Speaker Change: Okay.
Speaker Change: Thank you.
Speaker Change: And our next question comes from Ellie Merle of UBS. Your line is open.
Speaker Change: Hi, This is Jasmine Zhang on for Elliot. Thanks, So much for taking my question. So Firstly Sachs Man and I think T. J just on thank you. Please go can.
Speaker Change: Can you talk about what the latest is that you're expecting for your baseline rate JP in your population Bruce Szostak reinforced and what do you think the magnitude of effect that you think that you can share with you Eric and then just quickly. Thank you Lindsay.
Speaker Change: Can you clarify your current expectations in terms of potentially expanding beyond HR. Thank you.
Speaker Change: Thanks.
Speaker Change: Let me take the second one first with such asset.
The expansion beyond the HOS H.
Speaker Change: Would be.
Speaker Change: Potentially substrate of high risk.
Speaker Change: Yes.
Speaker Change: H population.
Speaker Change: It's actually something we discussed.
Speaker Change: Discussed with <unk>.
Speaker Change: HFC.
Speaker Change: And proposed.
Speaker Change: Daryl.
Speaker Change: Aero approach.
Speaker Change: Looking only at those patients that.
Speaker Change: Despite.
Speaker Change: The export therapy, we're not able to get to the goal.
Speaker Change: LDL reduction.
Speaker Change: That would be associated with the greatest reduction of risk.
Speaker Change: Just weren't comfortable with that because they thought well I can't speak for why but.
Maybe because we thought it was going to set precedent, but yes.
Speaker Change: To get into <unk>.
Speaker Change: They wanted it for.
Speaker Change: Program.
Speaker Change: Yes.
Speaker Change: Multiple adequate well controlled trials.
Speaker Change: Good morning.
Speaker Change: 1500 patients or more safety et cetera.
Speaker Change: And it didn't make sense to us.
Speaker Change: To go after such a sliver of high risk patients.
Speaker Change: With a very expensive very long program. So we're going to stay focused on HOS age.
Speaker Change: And do not anticipate at this time that we would expand the desk.
Speaker Change: But beyond.
Speaker Change: H.
Speaker Change: It's just that it's just not feasible at <unk> could use of our resources.
Speaker Change: Back to this aspect if I understood your question.
Speaker Change: What we think the mean.
Speaker Change: Tree triglycerides might look like and what sort of response, we might see.
Speaker Change: You would expect the tests are pretty good that we will.
Speaker Change: Pretty much replicate what we saw in phase III.
Speaker Change: Phase II.
Speaker Change: The <unk> triglycerides, <unk> population and that is stable.
Speaker Change: Okay.
Speaker Change: And as I said, a placebo adjusted.
Speaker Change: Change was 53% reduction so substantial.
Speaker Change: Large reductions in <unk>.
Speaker Change: Triglycerides, which is why we said.
Speaker Change: <unk> really.
Speaker Change: Yes.
Speaker Change: These trials are really overpowered.
Speaker Change: Because even with <unk>.
Speaker Change: Smaller trial phase III.
Speaker Change: SSA.
Speaker Change: So yes it did.
Speaker Change: It will it should be an impressive.
Speaker Change: It will it should be an impressive, but we expect it to be an impressive reduction in triglycerides.
Speaker Change: No.
Speaker Change: Naeem.
Speaker Change: I mean the enrollment.
Speaker Change: Triglycerides Brexit needs.
Speaker Change: Okay.
Speaker Change: I think your question right did I understand your question.
Speaker Change: We also were just interested in the NIM.
Speaker Change: Do you expect for acute pancreatitis kind of a baseline.
Speaker Change: Good.
Oh in the pancreatitis study, okay, well I would expect that's going to be.
Speaker Change: Closer to the <unk>.
Speaker Change: Two the FCS to the palisade baseline so.
Speaker Change: Would expect that the pancreatitis based.
Speaker Change: Based on it.
Speaker Change: We won't be it won't surprise me.
Speaker Change: Our 2000.
Speaker Change: Could be a little bit lower because we're.
Speaker Change: For patients, especially when they've had a history of pancreatitis.
Speaker Change: They're more susceptible to repeat pancreatitis EBIT at lower values, but it wont shock me if it turns out to be somewhere north of about 2000.
Speaker Change: Okay. Thanks, so much very helpful.
Speaker Change: Walker.
Speaker Change: You.
Speaker Change: And our next question comes from Patrick <unk> of HC Wainwright <unk> Company. Your line is open.
Patrick: Thanks, Good afternoon, and congrats on all the progress I was actually curious about your <unk> III and <unk> programs.
Patrick: Our latest thinking regarding the potential positioning of these compounds in complement mediated diseases.
Patrick: How are you viewing these compounds with a core to your portfolio are there is potential here for partnering thanks.
Speaker Change: Sure I'll take the second part of that.
Patrick: First part on more for <unk>.
Patrick: James.
Patrick: Yes.
Patrick: We are.
We are open to discussing partnerships for <unk> and factor B those drugs at least in my mind those driving work.
Patrick: Do what they're designed to deal with a.
Patrick: <unk> Hello.
Patrick: Respectively.
Patrick: And we think there is number of places they can go.
Patrick: If push came to shop, we could certainly build out a commercial presence within a fairly narrow set of.
Patrick: Opportunities.
Patrick: <unk>.
Patrick: But if we can find the right partnerships I think that would be that would be more beneficial to us. We'll just see if we can.
Patrick: We can find the right REIT partnerships with the right Capex and right now it's too early to tell it seems and I talked about some of the opportunities yes.
Speaker Change: Yes, sure. So we act.
Speaker Change: The data that we talked about earlier today with aerospace three of course, but the Iga and data.
Speaker Change: We're still.
Speaker Change: Pending some data in the <unk> population with that molecule.
Speaker Change: <unk> Cfd is.
Speaker Change: Also applicable to be used in those renal disease populations potentially in some of the hematologic complement mediated indications as well.
Speaker Change: We think based on what we've seen so far with Aerosmith, three that it stacks up pretty well against the other.
Speaker Change: Complement mediated drugs approved or being studied in the renal indications in terms of proteinuria reduction we talked about the 41%.
Speaker Change: Production.
Speaker Change: With the aerospace three big bet.
Speaker Change: Relative to what else is out there is pretty competitive, particularly when you looked at.
Speaker Change: Yes.
Speaker Change: Infrequent dose administration that can be used.
Speaker Change: And our AI dosing every three or four months it could have some advantages.
Speaker Change: Thank you.
Speaker Change: And our next question comes from Andrew <unk> of Goldman Sachs. Your line is open good afternoon.
Speaker Change: Thanks for taking our question James maybe one for you as you think about entering the OTC space. How are you thinking about advancing both narrow Alex seven as well as arrow and heavy.
Speaker Change: Through clinical development and do you have interest your capacity you can spell with or would you like to make a decision of one versus the other following the initial datasets.
Speaker Change: Sure Yeah, so our thoughts with those.
Speaker Change: From a cost standpoint, it doesn't cost that much to do to preclinical and phase one.
Speaker Change: Relevant.
Speaker Change: We have <unk>, which targets <unk> expression uses the so called gallon that technology for delivery that technology is pretty well vetted, we know what the safety profile at least of the platform is.
We should be able to knock down the gene target in that pad site.
Speaker Change: The contrast, arrow, Alex said and users are completely different platform.
Speaker Change: The animal data with really compelling with that molecule platform has never been in humans before so.
Speaker Change: Presumably there's a little bit more risk that or just because the platform hasn't been de risked.
Speaker Change: <unk> was to take both of those through phase one and then look at the data.
Both the safety and the PD and efficacy data.
Speaker Change: Choose one to move forward and there may be opportunities for partnership either one or both of those potential but that was part of why we brought both of them into the clinic.
Speaker Change: Let's also be clear.
Speaker Change: I do not expect these to be our last few obesity assets with our ability to address the sites.
Speaker Change: Our ability to address potentially CNS.
Speaker Change: This blood brain barrier platform that we've got there are a ton of really compelling metabolic and obesity targets, but I think youll see coming down the pike.
Speaker Change: Near term as well.
Speaker Change: Okay. Thank you so much.
Speaker Change: Sure.
Speaker Change: Thank you.
Speaker Change: And our next question comes from Luca <unk> of RBC. Your line is open.
Luca: Oh, great. Thanks, so much for taking my question Congrats on the progress made.
Luca: Maybe Bruce circling back on a prior question can you just maybe remind us how you're thinking about <unk> five study design, how what patients will be enrolled and maybe most importantly, how many patients do you need to actually hit the stats. So if you can talk about powering assumptions Debbie much appreciate it and then maybe bigger picture on <unk>.
Luca: Chris.
Chris Anzalone: Are you thinking about commercialization of the molecule ex U S are you planning to do that by yourself or you're still looking for a partner.
Speaker Change: Very much appreciate it thanks, so much.
Speaker Change: Yes. So so again this trial will be we believe the first ever trial, where we are.
Speaker Change: Demonstrating a benefit.
Speaker Change: Scott your tightest as the primary endpoint.
Speaker Change: So it's similar to our base trial for.
Speaker Change: From the standpoint.
Speaker Change: Putting beds.
Speaker Change: We hit a certain number of events, we will analyze we havent yet the bulge what that number of events is.
Speaker Change: I think we probably will in the future, but so far we have it.
Speaker Change: But we have a pretty good idea obviously, what that is but we also haven't said how many patients.
Speaker Change: <unk>.
Speaker Change: We're targeting.
Speaker Change: But again my guess is that we may continue to.
Speaker Change: His role.
Speaker Change: Sort of what the rate pancreatitis is that we're seeing in the <unk>.
Speaker Change: Early days, but the trial itself is a trial enriched from the perspective that it requires.
Speaker Change: That patients have had a history of pancreatitis.
Speaker Change: Including history of pancreatitis in the recent past.
Speaker Change: Which those patients who are at higher risk of pancreatitis.
Speaker Change: Hyper triglyceride patients.
Speaker Change: Never had redundancy.
Speaker Change: But again no one's ever done a trial like this before.
Speaker Change: So understanding exactly.
Speaker Change: What the what.
Speaker Change: Chris will be is something that.
Speaker Change: It will get some understanding of course, all be blinded, but we will get some understanding as to trial gets underway in Vegas.
Speaker Change: And that will that will sort of contributor, but it's certainly not the size of.
Speaker Change: You'll see box or anything like that it's much smaller.
Speaker Change: Given the reduction of pancreatitis, we saw in palisade for instance.
Speaker Change: But thank.
Bill: Thank you Bill we haven't I.
Speaker Change: I think we will give more detail in the future, but we're not ready to give all that.
Bill: <unk>.
Bill: The deep detail at this point.
Bill: I am.
Bill: So regarding your question on <unk>. So as you know we're full speed ahead in terms of the U S for Fcs and potentially <unk>.
Bill: Rest of World, we are open to.
Bill: Two to find new partners.
Bill: We are preparing right now in Europe.
Bill: I discussed.
Bill: How we're approaching that right now, yes, Luca so we think the European markets in particular, the large four European markets and the UK lend themselves extremely well to commercialization for a rare disease like FCS. Many of these patients on their patient journey, our triage through a small number of centers of excellence and so with limited infrastructure and resource.
Bill: We believe we're able to to help these potential STS patients through those centers of excellence and so as far as priority market downside the X outside the U S. Those large markets in Europe, and the UK and also Canada are a priority for us.
Speaker Change: Got it thanks, so much guys.
Speaker Change: Thank you.
Speaker Change: Are you.
Speaker Change: And our next question comes from Edward <unk> of Piper Sandler Your line is open.
Speaker Change: Okay. Thank you very much appreciate it.
Speaker Change: So.
Speaker Change: I know that you guys have.
Speaker Change: Thank you.
Speaker Change: Steve are reviewed.
You anticipate promenade.
Speaker Change: And one other steps are you taking from rate.
Speaker Change: Or.
Speaker Change: Hopeful approval and will likely launch thanks.
Speaker Change: Well as far as that kind of goes.
Speaker Change: We have not been advised that there is an expectation for an ad com.
Speaker Change: So.
Speaker Change: At this time.
Speaker Change: We don't think that that cuts in our future.
Speaker Change: As you probably know.
Speaker Change: The agency could change there.
Speaker Change: The general opinion on that at any time.
Speaker Change: Data.
Speaker Change: <unk> develops and they're in their analysis that makes them want to have it add kind of for some reason, but at this point.
Speaker Change: There is no anticipation of head count so.
Speaker Change: The likelihood I suppose it's lower everyday we get closer to potential approval.
Speaker Change: We can't say with Wolfe handling because they.
Speaker Change: They can decide at any time.
Speaker Change: We don't see it though.
Speaker Change: And then the <unk>.
Speaker Change: Part of the cases.
Speaker Change: The second part was what actions are we taking to be ready for launch.
Speaker Change: Yes, so I guess, we talked a bit about that from a from the R&D perspective.
Speaker Change: Yes.
Speaker Change: We are.
Speaker Change: We have an educational initiatives.
Speaker Change: We are there.
Speaker Change: Under our medical affairs function.
Speaker Change: So that's very much just a matter of health.
Speaker Change: <unk>.
Speaker Change: The positions to be ready for a new.
Speaker Change: New drug coming into the SCS space with really there hasnt been a promotional effort.
Speaker Change: Up until the latest launch we've ever really.
Speaker Change: So there is huge.
Speaker Change: Sure.
Speaker Change: Okay.
Speaker Change: Hi, Amit.
Speaker Change: Following internally towards sort of priming the pump a little bit there and then hopefully you guys coming in with a.
Speaker Change: Better therapy after thank you.
Speaker Change: Well look I think any any orphan disease.
Speaker Change: Essentially no approved therapies has always benefited from as much educational effort as you can get it.
History tells us and I could quote many examples where we are the first approved drug really did not fully open that market often takes two or more companies.
Speaker Change: To be promoting into a space even in orphan space to really get those markets to <unk>.
Speaker Change: And up and grow.
Speaker Change: So it's paradoxical because people buy pick you're better off being low.
Speaker Change: In many ways you are actually better off when there are multiple competitors now yesterday fight it out who gets the most share in yet, but actually the more educational noise the better.
Speaker Change: Trying to open up a market like FCS in the exact same will be true for <unk> as well.
Speaker Change: Bore more noise is better than less.
Speaker Change: Yes, the best drug usually wins the share battle.
Speaker Change: Pie gets much bigger with more education.
Speaker Change: Thats really helpful.
Speaker Change: Thank you.
Speaker Change: Yeah.
Speaker Change: And our next question comes from Mike <unk> of Morgan Stanley. Your line is open.
Speaker Change: Good afternoon, thanks for taking the question.
Speaker Change: Maybe just another one on <unk> in the upcoming FCS launch it sounds like you guys are making good progress and launch prep, but maybe you could just talk about.
Speaker Change: Remind us the U S patient population.
Speaker Change: And then some of the progress youre, making identifying patients and if theres any way to try and accelerate that trend. Thanks.
Speaker Change: Yes, thanks for the call, Mike you'd be familiar with the epidemiological data, suggesting that the prevalence of FCS in the United States would be $1 13 per $1 million and so that spans anywhere from the one hundreds of patients in the U S to mid single digit thousands.
Speaker Change: And we think the variability around the single digit thousands is really around whether you think about genetic sts or even clinically diagnosed Fcs. So thats why there is some variability there.
Speaker Change: As a reminder of course in palisade, we studied <unk> in both the genetically confirmed and clinically diagnosed patients and showed similar.
Speaker Change: Show similar results.
Speaker Change: So that's how we think about the broader market for or FCS as far as patient identification I would say that our internal <unk> team here, our analytics team and our marketing team is comprised of.
Speaker Change: Seasoned professionals, who have cut their teeth and party metabolic and lipid specifically and are really using the latest technologies to two patient find.
Speaker Change: Similarly to support those individuals who may have FCS and don't know that they have FCS so patient finding will be.
Speaker Change: Absolutely critical as we move into this space as it is with any ultra rare condition and we feel like we have the people and the capabilities to summit.
Speaker Change: Alright, thank you.
Speaker Change: Thank you. Thank you.
Speaker Change: And our next question comes from Matthew <unk> of <unk>.
Speaker Change: <unk> Securities Your line is open.
Speaker Change: Yes, good afternoon, Dean thanks for taking our questions and congrats again.
Speaker Change: <unk> as a service.
Speaker Change: Maybe on the.
Speaker Change: A quick.
Speaker Change: Quick follow up on the SCB data is there anything that you are.
Speaker Change: You got it.
Speaker Change: Willing to share what may be the learnings there and maybe.
Speaker Change: <unk>, two which is the combination.
Speaker Change: Are those sequential or our ability are they happening in tandem and I just one follow on for Tim.
Speaker Change: Yes sure on the second part of the question.
Speaker Change: The combo studies and the Mad non combination.
Speaker Change: Studies those are in parallel so those are enrolling at the same time and then I think in terms of what we can share.
Speaker Change: Yes.
Chris Anzalone: As Chris said earlier.
Speaker Change: I'll have to wait and see later this year, what kind of data that we have available.
Speaker Change: Okay understood and again, what partnership related milestones that factored in your <unk> 28, <unk> guidance. If you could maybe just clarify that thanks for taking my question and congrats again.
Speaker Change: So we've included the near term milestones that we expect to get from <unk>.
Speaker Change: And beyond that there are other milestones if we think there.
Speaker Change: Probable of happening that we have included those as well.
Speaker Change: We're not providing specifics on that.
Speaker Change: Understood. Thank you.
Speaker Change: Thank you.
Speaker Change: And our next question comes from Manny.
Speaker Change: <unk> of Leerink. Your line is now open.
Speaker Change: Thank you guys you have Brian on for Bonnie. Thanks for taking our question maybe just a quick financial one I noticed you guys start to pay down that credit facility. Just wondering should we expect that to continue over the course of the year or was this more of just a onetime pay down to kind of bring that total down. Thanks.
Speaker Change: So the the onetime pay down was because of the <unk> agreement there will be future.
Speaker Change: Payments on that but it's only related to times when we have.
Speaker Change: Cash flow coming in from milestones as such.
Speaker Change: So it's only attributable to when we have some cash that's coming in.
Speaker Change: Yes.
Speaker Change: Got it thanks.
Speaker Change: Thank you.
Speaker Change: And our next question comes from Brendan Smith of TD Cowen Your line is open.
Speaker Change: Okay.
Speaker Change: Maybe just similar to a couple of earlier questions I wanted to ask a little bit more on your thinking.
Speaker Change: Thank you for the CNS assets.
Speaker Change: So is the plan as of today to carry those forward internally or are you already kind of thinking that this could potentially be partnering our license.
Speaker Change: And then maybe more specifically for map care are there any subset of the AIDS population, you'll look to target first just kind of wondering how we should think about the potential design, there and segmentation that market. Thanks guys.
Speaker Change: Sure.
Speaker Change: So when you take the MTF and I'll take the first question sure Victor So the second question on <unk> not at this point.
Speaker Change: For the phase one design, it's not entirely finalized yet, but suffice it to say that the.
Speaker Change: Main goal of the study will be to evaluate safety and measure PD.
Speaker Change: Harbors of knockdown.
Speaker Change: Tao.
Speaker Change: <unk> subtypes of Tau.
Speaker Change: In different populations.
Speaker Change: So we have not specifically.
Speaker Change: Pin down subtypes of ABB that we plan on study.
Speaker Change: And regarding your first question so as we talked about our first three candidates in the clinic.
Speaker Change: <unk> are expected to be mapped.
Speaker Change: Towards the end of this year.
Speaker Change: For Alzheimers HCP, Brian and Thats already licenses wrapped up that we expect that to you this year or two by the end of the year and then and then first part of next year I'll sneak in Parkinson's.
Speaker Change: So it's the HTS already outfit.
Speaker Change: Docket right now.
Speaker Change: Ultimately, we've always been clear to potential partners and that is off limits right now we view that as a.
Speaker Change: Very high value target.
Speaker Change: Look we don't know yet.
Speaker Change: This CNS BBB platform is going to translate from animals to humans, we will see some of it. So this is a bet that we're making but.
We think it's a good one given given the data we've seen in the preclinical models given that.
Speaker Change: The validated nature of map to your targets. So we're holding on to that for at least for right now, we'll see where that goes in the future.
Speaker Change: Snoop <unk>, we like that target a lot.
Speaker Change: And we if a partner comes in with the right partner comes in with the right kind of deal we would certainly consider a partner in that.
Speaker Change: So that's so that we don't feel quite as strongly at least in the near term as we do on that and then and then looking at those are the first three of we think we think many.
Speaker Change: Once we have this this platform.
Speaker Change: Related in humans, we're going to run there is a number of additional good targets that we are developing internally and my expectation is if we see that this translates as we hope it does you're going to see.
Speaker Change: A large number of additional CNS targets, some of which we will hold onto some of which were partner.
Speaker Change: Got it okay. Thanks, guys.
Speaker Change: Thank you.
Parker Agarwal: And our next question comes from Parker Agarwal of Cantor Fitzgerald. Your line is open.
Parker Agarwal: Alright, Thank you for taking my questions and congratulations on the quarter Iron This girl phase III Readouts coming soon.
Speaker Change: Awesome achieve statistical significance on frankly that as events in phase III, how does that impact the <unk>.
Speaker Change: But you do have the banker guidance reduction data and the labor around the time of launch was waiting for Roche ask if I have to read out and anything else you will be focusing on olive garden, Chris could you Travis Thank you.
Speaker Change: Well.
Speaker Change: It'll be interesting to see whether they can accrue enough events.
Speaker Change: To the group to actually achieve and there we will see.
Speaker Change: From our perspective.
Speaker Change: That was not a good debt to for us to rely on that.
Speaker Change: Especially in Europe.
Speaker Change: Two to do that.
Speaker Change: Our program is also designed to.
Speaker Change: Look at pancreatitis events, albeit we look at depth and in aggregate tightness.
Speaker Change: So we don't look at possible.
Speaker Change: There are cases, we just don't think that.
Speaker Change: We think there is a high chance that that's going to work.
Speaker Change: Health technology authorities in Europe.
Speaker Change: We really felt that we had.
Speaker Change: Definitely pancreatitis from our perspective.
Speaker Change: But we are we are adjudicating any pancreatitis that occurs and it shifts to three shifts in Florida, we are.
Speaker Change: And we haven't written those studies.
Speaker Change: And when we get the Orange.
Speaker Change: Two trials for the purpose specific.
Speaker Change: Okay.
Speaker Change: But ultimately.
Speaker Change: Yes, that's fair.
Speaker Change: <unk> feels like a high risk strategy.
Speaker Change: In all comers that's http.
Speaker Change: Okay.
Speaker Change: Again, I think that.
Speaker Change: Purpose built study.
Speaker Change: Rather than a secondary endpoint.
Speaker Change: Much more.
Speaker Change: Agreeable to the payers.
Speaker Change: Sophisticated difficult payers in the world.
Speaker Change: Most of the European Payors and some other places as well.
Speaker Change: Thank you.
Speaker Change: And our next question comes from David Leibowitz of Citi. Your line is open.
Speaker Change: Hi, everyone. This is Lee on for David Lebowitz. Thank you for taking our question.
Speaker Change: We have one on pricing or plus Sharon.
Speaker Change: How do you think about.
Speaker Change: Launching this in FCS and then waiting for the <unk>.
Speaker Change: <unk> data in the <unk> term.
Speaker Change: We expect something like trend goes.
Speaker Change: So getting a label expansion in <unk>, and therefore, having to move from an ultra rare pricing scheme into something that is a little bit cheaper is that something on your mind, yet as you look towards commercialization what are your thoughts on potentially adding to navigate the ultra rare.
Speaker Change: Versus much more prevalent disease pricing schemes.
Speaker Change: Sure.
Speaker Change: I think we can have and we have we are having transparent and open conversations with payers.
Speaker Change: Should we be lucky enough to have.
Speaker Change: So thats our unapproved in Fcs.
Speaker Change: It's a relatively narrow populations and so we would we would need.
Speaker Change: To get reimbursed at a high level that is commensurate with ultra orphan should we be lucky enough to have a shaft three and four are readout, well and get approved.
Speaker Change: <unk> or of course, we would bring the price down.
Speaker Change: Conceptually so so so that we know.
Speaker Change: A more granular level about what those two prices are we just don't know at this point its going to dependent bottom line. So.
Conceptually.
Speaker Change: That is our strategy.
Speaker Change: I cant get more granular than that at this point.
Speaker Change: Thank you I'm showing no further questions at this time I would like to turn it back to Chris Anzalone for closing remarks.
Speaker Change: I will thank everyone for joining us today. It is bittersweet to end. This call. This is the last time that we're going to have cannot buy less.
Speaker Change: During these calls and as we mentioned in the prepared remarks.
Speaker Change: We appreciate the great work for the last 16 years.
Speaker Change: I'm going to Miss having him here instead of course excited to have Dan onboard.
Speaker Change: Today's my right maybe to my left.
Speaker Change: Once again gone, but thank you all for joining us today.
Speaker Change: This concludes today's conference call. Thank you for participating and you may now disconnect.
Speaker Change: Yeah.
Speaker Change: [music].