Earnings call transcript: Ionis Pharmaceuticals Q2 2025 Surpasses Revenue Expectations

Ionis Pharmaceuticals Inc. (IONS) reported its second-quarter 2025 earnings, showcasing a robust financial performance that exceeded revenue expectations. The company posted a revenue of $452 million, significantly surpassing the forecast of $286 million, marking a 58.04% revenue surprise. The earnings per share (EPS) stood at $0.70, a substantial improvement from the anticipated -$0.24. This impressive financial outcome triggered a positive market response, with Ionis’ stock rising 8.44% in pre-market trading to $44.98. According to InvestingPro data, the company maintains strong liquidity with a current ratio of 9.66, though it has faced profitability challenges in recent quarters.

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Key Takeaways

• Ionis Pharmaceuticals’ Q2 2025 revenue doubled year-over-year, reaching $452 million.
• The company achieved a significant EPS of $0.70, outperforming the forecasted -$0.24.
• Ionis’ stock surged 8.44% in pre-market trading following the earnings release.
• Tringoza product sales tripled from the previous quarter, contributing to revenue growth.
• Ionis anticipates FDA approval for Donavolorsen in August, potentially boosting future revenue.

Company Performance

Ionis Pharmaceuticals demonstrated strong performance in Q2 2025, with revenue doubling compared to the same period last year. The company’s strategic focus on product launches and innovation has paid off, particularly with Tringoza, a treatment for familial chylomicronemia syndrome (FCS), which saw a threefold increase in sales from Q1. This growth aligns with broader industry trends emphasizing novel treatments for rare diseases. InvestingPro analysis shows the company operates with a moderate debt level, with a debt-to-capital ratio of 0.22, providing financial flexibility for continued innovation.

Financial Highlights

• Revenue: $452 million, 100% increase year-over-year.
• Earnings per share: $0.70, significantly above the forecast of -$0.24.
• Non-GAAP net income: $154 million.
• Tringoza sales: $19 million, tripling from Q1.
• Royalty revenues: $70 million, a 10% increase.

Earnings vs. Forecast

Ionis Pharmaceuticals exceeded expectations with an EPS of $0.70 against a forecast of -$0.24, resulting in a 391.67% surprise. This performance marks a notable deviation from previous quarters, where the company faced challenges in meeting earnings forecasts.

Market Reaction

Following the earnings announcement, Ionis’ stock experienced an 8.44% surge in pre-market trading, reaching $44.98. This positive movement reflects investor confidence in the company’s strategic direction and financial health. The stock’s performance is notable as it approaches its 52-week high of $51.62. InvestingPro data indicates the stock is currently trading above its Fair Value, suggesting investors should carefully consider entry points. The stock has shown strong momentum with a 24.49% return over the past six months.

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Outlook & Guidance

Ionis Pharmaceuticals has set a revenue guidance of $825-$850 million for 2025, reflecting optimism in its product pipeline and expected market launches. The company anticipates an operating loss of $300-$325 million but remains confident in achieving a year-end cash balance of approximately $2 billion. Key product launches, including Donavolorsen, are expected to drive future growth.

Executive Commentary

CEO Brett Monia emphasized the company’s strategic positioning, stating, "We are well positioned to deliver a steady cadence of independent and partnered launches over the next few years." Chief Global Product Strategy Officer Kyle Janae highlighted the execution of growth opportunities, while CFO Beth Haugen noted the potential for significant revenue growth driven by disciplined investment and a robust pipeline.

Risks and Challenges

• Regulatory hurdles: Potential delays in FDA approvals could impact projected timelines.
• Market competition: Emerging competitors in the rare disease treatment space.
• Operational costs: Rising sales and marketing expenses to support new product launches.
• Economic conditions: Macroeconomic factors that could affect healthcare spending.
• Supply chain issues: Potential disruptions impacting product availability.

Q&A

During the earnings call, analysts focused on the momentum of the Tringoza launch and the company’s strategy for identifying patients. Ionis expressed confidence in achieving significant triglyceride reduction in its SHTG studies and highlighted preparations for the Donavolorsen launch. Pricing strategy for future indications was also a topic of interest, with ongoing discussions aimed at maximizing market penetration.

Full transcript - Ionis Pharmaceuticals Inc (IONS) Q2 2025:

Conference Operator: Good morning, and welcome to Ionis second quarter twenty twenty five financial results conference call. As a reminder, this call is being recorded. At this time, I would like to turn the call over to Wade Walke, senior vice president of investor relations, to lead off the call. Please go ahead.

Wade Walke, Senior Vice President of Investor Relations, Ionis: Thanks, Steve. Before we begin, I encourage everyone to go to the investors section of the Ionis website to view the press release and related financial tables we will be discussing today, including a reconciliation of GAAP to non GAAP financials. We believe non GAAP financial results better represent the economics of our business and how we manage our business. We’ve also posted slides on our website that accompany today’s call. With me this morning are Brett Monia, Chief Executive Officer Kyle Janae, Chief Global Product Strategy Officer Richard Geary, Chief Development Officer and Beth Haugen, Chief Financial Officer Eugene Schneider, our Chief Clinical Development Officer, and Eric Swayze, Executive Vice President of Research, will also join us for the Q and A portion of the call.

I would like to draw your attention to slide three, which contains a forward looking language statement. During this call, we will be making forward looking statements that are based on our current expectations and beliefs. These statements are subject to certain risks and uncertainties, and our actual results may differ materially. I encourage you to consult the risk factors contained in our SEC filings for additional detail. And with that, I’ll turn the call over to Brett.

Brett Monia, Chief Executive Officer, Ionis: Thanks, Wade. Good morning, everyone, and thank you for joining us on today’s call. We have continued to build strong momentum across our business highlighted by the excellent early commercial performance of our first independent launch, Drongosa. The first and only FDA approved treatment for familial chylomicronemia syndrome. Trungolza exceeded revenue expectations during its second quarter on the market, underscoring its strong therapeutic profile, our well executed commercial strategy as well as the significant unmet need Trungolza is addressing.

The revenue and commercial performance of Trungolza to date along with our confidence in Trungolza’s continued commercial success supports the increase in our financial guidance for 2025, which Beth will cover in more detail shortly. We’re also pleased that Trungosa recently received a positive CHMP opinion paving the way to bring this transformative therapy to patients across Europe. Shifting gears, we anticipate Donavolorsen for hereditary angioedema or HAE will be our second independent launch by receiving FDA approval next month. Based on the progress of the review, we believe the approval and subsequent launch remains on track. Donvalorcin has the potential to become a preferred prophylactic therapy for many HAE patients with strong efficacy and favorable safety and tolerability in the pivotal studies.

A patient friendly auto injector and a convenient dosing schedule of monthly or every other month self administration. We’re well positioned to launch domdolorescent following approval. Additionally, our Phase three pipeline continues to advance well with data expected later this year from two wholly owned Ionis programs. If positive these results would support the continued steady cadence of independent launches next year bringing important new treatments to patients. These include a second indication for olazarsen in severe hypertriglyceridemia or SHTG, a condition with a large patient population in significant unmet need and zilgarnirsen for Alexander disease severe rare leukodystrophy with no approved disease modifying therapies.

Together, these programs in addition to two holes in FCS and Donvalorsen and HAE represent major breakthroughs for patients and represent multi billion dollar revenue potential for Ionis. Additionally, by the 2027, we anticipate four potential launches from our rich late stage partner pipeline, targeting serious life threatening conditions for both rare and highly prevalent diseases. These will further expand the impact and reach of Ionis discovered medicines and have the potential to meaningfully increase total revenue. With our strong momentum across the business including Trinvolta and our upcoming independent and partnered launches, Ionis is well positioned to bring transformative medicines to patients for years to come and in turn achieve sustained revenue growth and positive cash flow. And with that, I’ll turn the call over to Kyle.

Kyle Janae, Chief Global Product Strategy Officer, Ionis: Thank you, Brett. With our first independent launch now well underway and a second launch right around the corner, our commercial team is executing on our strategy to capitalize on these significant growth opportunities. Tringoza reported $19,000,000 in net product sales for the second quarter, reflecting a threefold increase in revenues quarter over quarter. In the second quarter, Tringoza continued to build launch momentum. This was a result of several factors, including effective patient identification efforts, a strong product profile, favorable payer dynamics, and overwhelmingly positive HCP reported experience.

Our patient identification initiatives are paying off. The breadth and depth of unique physicians prescribing Trangolza continues to grow, and many of these physicians have prescribed the therapy to two or more patients, underscoring the positive experience of both clinicians and patients. This demand also spans a broad mix of specialties, with cardiologists and endocrinologists representing roughly 5030% of prescribers respectively, and lipidologists and internal medicine providers making up the balance. Overall, physician feedback remains highly favorable, with significant benefits reported and awareness of Tringolsa continuing to gain traction. Coverage and reimbursement trends have remained favorable.

To date, the coverage mix for patients on Trangolza is approximately 60 commercial and 40% government. Importantly, patients, whether clinically diagnosed or genetically confirmed, have gained access. Patients have obtained coverage through a growing number of formal policies or via the medical exception process. This highlights both the urgent unmet need and payers’ willingness to support access even before formal policies are in place. Additionally, over ninety percent of patients have paid $0 out of pocket since launch, and timelines for patients obtaining the medicine are consistently beating our aggressive internal benchmarks.

Nearly all patients have opted into our Ionis Every Step Support Program, a testament to the value the program is providing. We established the Ionis Every Step Support Program to ensure a positive patient experience by providing disease and nutrition education, auto injector training, and reimbursement support among other offerings. For healthcare providers, the program provides helpful support from insurance authorizations and coverage coordination to reauthorizations and refills. We’re proud of Trangolta’s early momentum, but we know we’re still in the early innings. The vast majority of the estimated three thousand people living with FCS in The US remain unidentified.

To close that gap, we’re continuing to focus on our patient finding efforts and HCP education. Our customer facing team has reached over 3,000 physicians, and over 30,000 HCPs have been targeted through our omni channel capabilities, both intended to further increase awareness of FCS, expand patient identification, and educate on the potential benefits of Trangolsa treatment. Backed by an experienced and high performing team, we are well positioned to continue to take advantage of our first mover position, to bring Trincosa to patients in need, and keep them on treatment. Building on our early success in FCS, we are advancing toward a potential blockbuster opportunity in severe hypertriglyceridemia with olazarsen. SHTG represents a large patient population, many of whom struggle to manage their triglyceride levels with current treatments.

In The US alone, more than one million people have high risk SHTG, and includes individuals with triglyceride levels above eight eighty or above 500 with a history of acute pancreatitis. With a significant first mover advantage, we believe olanzarcen is well positioned to address the unmet needs of patients with severe hypertriglyceridemia. Our commercial team is making excellent progress as we prepare for a potential launch next year. Donadolorsen, our second independent launch, has the potential to transform the treatment paradigm for individuals with HAE, as the first and only RNA targeted prophylactic medicine. More than twenty thousand people in The US and in Europe are estimated to have HAE, with approximately seven thousand people in The US alone.

In The US, most patients are currently on prophylactic treatment. However, many HAE patients remain unsatisfied, with up to twenty percent of patients switching therapies each year in search of a more effective and convenient option, highlighting a need for new treatments with enhanced profiles. We believe Doloresyn is uniquely positioned to meet this demand. The Donnie Doloresyn clinical data have shown durable efficacy and favorable safety and tolerability profile, with a patient friendly monthly or every other month self administration with an auto injector. Importantly, as we prepare to bring Donnadolorsen to market, we are applying the same disciplined and innovative approach that has made the Trengorza launch a success.

We have deployed our field team who are educating and preparing the market for the anticipated approval of dynadolorsen and are energized to successfully execute the launch. Meanwhile, our market access team is actively engaging with payers, and our field medical team continues to lay the essential groundwork to ensure a smooth and successful launch. Our experienced and scalable commercial organization is already delivering excellent results, supported by the early success seen with the launch of Trangolza and FCS. As we build on this momentum, we remain focused on maximizing Trangolza’s full potential while preparing to successfully execute three additional launches by the end of next year, including Donnie Delorson in the coming weeks, enabling Ionis to reach more people in need with our medicines. With that, I’ll now turn it over to Richard.

Richard Geary, Chief Development Officer, Ionis: Thank you, Kyle. We are making excellent progress across our pipeline positioning Ionis to deliver on our mission of bringing transformational medicines to patients for years to come. Let me start with recent updates from our wholly owned pipeline. As a reminder, Donna Delorson is currently under regulatory review in both The US and EU, with submissions supported by its robust clinical data. We remain confident in the August 21 PDUFA date based on the engagement we’ve had with the FDA at this stage in the process.

Pending approval, we look forward to making this potential best in class prophylactic treatment available to people living with HAE. Beyond its approved use in FCS, olisarcen is also being evaluated for the treatment of severe hypertriglyceridemia, or SHTG, with three separate phase three studies supporting our planned sNDA filing, assuming positive data. In May, we reported top line results from the ESSENCE study, which will help satisfy the regulatory safety requirements for the broad SHTG population. ESSENCE primarily enrolled patients with triglyceride levels between one hundred fifty and five hundred milligrams per deciliter, a level of triglycerides that is generally not associated with high risk for acute pancreatitis. In contrast, the CORE and CORE-two studies exclusively enrolled SHTG patients.

Participants in these studies had triglycerides that were greater than 500 milligrams per deciliter, with approximately forty three percent of the participants across both studies with triglycerides greater than eight eighty milligram per deciliter at baseline. Triglycerides at this level put people at high risk for acute pancreatitis. The ESSENCE study met its primary endpoint, showing statistically significant placebo adjusted mean reductions in triglyceride levels of 6158% at six months, with the eighty and fifty milligram monthly dosing, respectively. The vast majority of participants achieved triglyceride levels below one hundred fifty milligram per deciliter, thus reaching the normal range. Olisarcin also met all key secondary endpoints, and demonstrated a favorable safety and tolerability profile.

The results from this study were recently accepted as a hotline session at the ESC Congress twenty twenty five. We look forward to sharing additional details from the study then. Now the phase three core and core two studies have enrolled approximately 1,100 participants, making this pivotal program the largest ever conducted in SHTG. These studies remain on track, with top line results expected in September. We believe this indication represents a significant opportunity, as many SHTG patients are unable to adequately manage their triglycerides with current therapies.

Physician feedback continues to highlight strong interest in more effective treatment options for managing triglyceride levels, underscoring the potential value of olosartan in this population. Importantly, this feedback indicates that physicians understand that lowering triglycerides will reduce the risk of acute pancreatitis, and that they would prescribe olicersin, if approved, based solely on its ability to substantially lower triglycerides. Although the CORE and CORE-two studies were not designed as AP outcome studies, we are seeing acute pancreatitis events on a blinded basis. As a result, we expect to have accumulated more combined pancreatitis events for COR and COR2 than we had in the FCS BALANCE study. This gives us confidence we could have sufficient data to observe at least a favorable trend in olesarsen’s impact on AP within the studies.

Turning our attention to zilgarnirsen, our medicine to treat Alexander disease, an ultra rare leukodystrophy that profoundly impacts patients and families who today have no approved disease modifying treatments. The phase three study remains on track, and we’re looking forward to sharing data later this year. Given the ultra rare nature of this disease, we implemented an innovative clinical development strategy for zilgarnsen, using a seamless phase one to phase three study design. This is the first study of a disease modifying therapy ever conducted in this patient group. And while this approach allows us to move efficiently toward a potential registration enabling study, it also means that the upcoming readout will represent the first clinical data in patients with Alexander disease.

As such, there is a higher degree of uncertainty associated with this readout compared to other medicines we’re advancing in late stage development. Assuming positive data and approval, zilganeursen would represent the first of what we expect to be many more independent launches from our leading neurology pipeline. ION five eighty two, an investigational medicine for the treatment of people living with Angelman syndrome, is the newest addition to our late stage pipeline. Angelman Syndrome is a serious and rare neurodevelopmental disorder that leads to significant and lifelong physical and cognitive impairments, and impacts tens of thousands of people living with this disorder. Supported by the phase onetwo open label HALO study results, we recently dosed the first patient in the global phase three REVEAL study.

We plan to have this study fully enrolled next year. Turning to our partnered programs. We are pleased that higher dose nusinersen is one step closer to market, with FDA and EMA regulatory submissions under review. With the well characterized profile of SPINRAZA established over the past ten plus years, and the positive data from higher dose SPINRAZA, we believe SPINRAZA is well positioned to continue to bring benefit to SMA patients around the world. Our partner Biogen also recently shared positive top line interim results from the phase one study of salinersen, an investigational antisense medicine being developed for the potential treatment of spinal muscular atrophy.

Leveraging the same mechanism of action as SPINRAZA, we designed salinersen with novel Ionis chemistry to achieve strong efficacy and once yearly dosing. In children previously treated with gene therapy, once yearly dosing with both the forty and eighty milligram doses were well tolerated and led to rapid and substantial slowing of neurodegeneration, as shown by reductions in neurofilament. Exploratory clinical outcome data also showed that children in the study achieved meaningful improvements in function and attained new milestones. Biogen is actively engaging with regulatory agency to align on the phase three study design. Notably, the initiation of a phase three study would trigger a $45,000,000 milestone payment to Ionis.

Additionally, our royalty rates for salinersen are significantly higher than for SPINRAZA. If approved, salinersen would also substantially extend the life cycle for the Ionis Biogen SMA franchise. The progress of this program underscores Ionis’ deep expertise in oligonucleotide medicinal chemistry, and reinforces the strength of our broader neurology pipeline. It also validates our approach as we advance new medicines with next generation chemistries, including follow on medicines for our wholly owned programs. With multiple data readouts and regulatory milestones expected this year and next, our advancing pipeline underscores the strength of our science and our commitment to addressing serious diseases.

With that, I’ll turn it over to Beth.

Beth Haugen, Chief Financial Officer, Ionis: Thank you, Richard. I’m pleased to report that for the second time this year, we are significantly raising our 2025 financial guidance. This time driven by strong revenue performance to date, including the early launch success of Trincolza, and an improved outlook for the year. In the second quarter, we earned revenue of $452,000,000 a twofold increase year over year, and $584,000,000 for the first June 2025, an increase of nearly 70% versus prior year. The strong second quarter revenue we earned also enabled to generate $154,000,000 in non GAAP net income for the quarter.

As you heard from Kyle, the early Tringulsa launch continues to perform well. We earned $19,000,000 in product sales, representing a threefold increase over the first quarter. Royalty revenues increased by approximately 10% to $70,000,000 in the second quarter, anchored by meaningful contributions from both SPINRAZA and WAYNUA. We also generated substantial revenue from our R and D collaborations, including the $280,000,000 upfront payment for the sapaglutzin license, a medicine outside of our core areas of focus. Nearly 100% of this revenue dropped directly to the bottom line, underscoring the important financial contributions of our partnered pipeline.

Total non GAAP operating expenses in the second quarter increased 8% year over year, highlighting our commitment to disciplined investment and driving operating leverage. As planned, our sales and marketing expenses increased year over year, driven by our investments in The US launch of Trincolza and preparations for the upcoming launch of donadolorsen. Our SG and A expenses also included our minority portion of Waynua’s sales and marketing costs. R and D expenses decreased year over year as several of our late stage studies have recently concluded. Importantly, we continue to strategically fund our advancing pipeline, with more than twothree of our total R expenses funding our late stage programs.

Based on our continued execution across the board, we have increased our revenue guidance range by $100,000,000 and now expect to generate between $8.25 and $850,000,000 in revenue this year. Importantly, our improved revenue guidance is driving our improved operating loss and cash guidance for the year. Our revenue guidance includes sizable commercial revenue supported by SPINRAZA and the continued strong TRINGOZA performance. We expect to generate between 75,000,000 and $80,000,000 in TRINGOZA product sales this year. We are also on track to add initial product revenue from our second launch with the FDA action date for donadolorsen set for August 21.

Given the timing of approval, we anticipate donadolorsen will modestly contribute to revenues this year, with a greater contribution next year. We continue to project our full year 2025 operating expenses to increase in the high single digit percentage range compared to last year, driven by investments to support the success of our multiple ongoing and planned launches. With the increase to our revenue guidance, we now project an operating loss between 300,000,000 and $325,000,000 and a year end cash balance of approximately $2,000,000,000 both substantially improved from our previous guidance as full year projected revenue is growing faster than projected operating expenses. The strength of our balance sheet reinforces our disciplined capital management, which enables us to continue to invest for growth. To maintain this strength, we plan to refinance our 2026 convertible debt ahead of the maturity date.

As always, we are rigorously evaluating a number of options against our objectives to minimize cost of capital, preserve our cash to support our products and pipeline, and maintain operational flexibility. We have historically used convertible debt and believe it remains an attractive option for us. With multiple product launches ahead, a rich pipeline, and continued disciplined investment, we are well positioned to achieve significant revenue growth resulting in sustained positive cash flow in the next few years, positioning Ionis for substantial value creation in both the near and longer term. And with that, I’ll turn the call back over to Brett.

Brett Monia, Chief Executive Officer, Ionis: Thank you, Beth. The second quarter marked another period of strong execution and momentum for Ionis. The continued excellent performance of TrengoZA highlights the strength of our commercial execution and the value of our innovative science in addressing serious unmet needs. As we prepare for the potential approval and launch of ondogalorcin and with a deep and advancing Phase three pipeline, we’re well positioned to deliver a steady cadence of independent and partnered launches over the next few years. These upcoming milestones reflect the significant progress we’re making toward delivering additional transformative medicines to people with serious diseases and building a sustainable high growth company.

Before I conclude, I’d like to provide two additional announcements. On October 7, we will be hosting an innovation day in New York City to highlight our pipeline, advancements in our drug discovery capabilities and will provide additional insights into our ongoing and upcoming independent launches. We’ll provide additional information as the day gets closer for what we believe will be a highly informative event. Additionally, we are eagerly awaiting results from the CORE and CORE two Phase three studies in SHTG. Given that we expect to report the results of both studies at one time in September, we will be initiating a quiet period starting tomorrow, July 31.

Quiet period will be lifted upon data announcement and with that we’ll now open it up for questions.

Conference Operator: Thank you. We will now begin the question and answer session. To ask a question, you may press star then one on your telephone keypad. If you are using a speakerphone, please pick up your handset before pressing the keys. If at any time your questions have been addressed and you would like to withdraw your question, please press star then 2.

At this time, we will pause momentarily to assemble our roster. First question comes from Gary Nachman with Raymond James. Please go ahead.

Gary Nachman, Analyst, Raymond James: Hi, guys, and congrats on the strong quarter.

: So first, just talk a

Gary Nachman, Analyst, Raymond James: bit more about how the TRINGOZA FCS launch is going in terms of finding these new patients and getting them through the reimbursement process and on drug. So what gives you confidence we’ll see that strong sequential growth in the back half of the year based on the fiscal year guidance of 75,000,000 to $80,000,000 So first a little bit more on that. And then on the upcoming SHTG readout in September, just what level of trig lowering are you expecting to see? What are you powered to see and what do you believe will be considered clinically meaningful in these patients that physicians want to see? And it sounds like you’re still confident there’ll be enough pancreatitis events across both studies to show a positive trend.

Do you think physicians need to see that to use it for severe high trigs? Maybe give us a sense of what you’re hearing in the physician community on that.

Brett Monia, Chief Executive Officer, Ionis: Thanks, Gary. Kyle, would you start with the FCS launch and what we’re expecting second half of the year?

Kyle Janae, Chief Global Product Strategy Officer, Ionis: Yeah, thanks, Gary. So as we communicated, we’re really off to an encouraging start, again, with the FCS launch in Trangosa for the first half of the year. The $19,000,000 in Q2 and a threefold increase over the previous quarter, really demonstrates the early momentum that we’re very proud of. The product profile, first of all, is playing through very effectively. The reductions APOC3, reductions in triglycerides, improvements in acute pancreatitis, reduction in risk of acute pancreatitis, and reductions in hospitalizations.

I mean, of these things stack up very, very favorably in terms of the receptivity for Trangosa and the FCS population. In addition to that, a very strong commercial strategy. As I referenced, our customer facing team has over 3,000 physicians that they’re interacting with currently. Our marketing and our omni channel capabilities, we’re reaching greater than 30,000 HCPs right now with that education. And then you also asked about reimbursement.

Access is going extremely well. Either clinically diagnosed or genetically confirmed patients are going through the process and getting reimbursement very quickly. Patient out of pocket expenses, you know, than 90% of those patients are paying $0 out of pocket. So overall, what we’re seeing is very strong execution, from a very strong program. In terms of the second half, the real focus is on additional patient identification and continuing to get those patients diagnosed.

And, through the activities of our commercial team, be it customer facing and or our omni channel and marketing capabilities, we expect that to continue. But to continue to identify these upwards of three thousand patients that potentially have FCS is going to take some work, and we expect that we’ll continue that growth throughout the rest of the year.

Brett Monia, Chief Executive Officer, Ionis: Thanks, Kyle. Gary, the triglyceride lowering that we’re expecting in SHTG is probably going to be similar to what we saw in Essence, where we saw a fifty eight percent to sixty two percent reduction based on the dose, fifty percent versus eighty milligrams. And what we know, based on extensive work we’ve done with the lipid specialists or the cardiologists or the endocrinologists that manage these patients, what they’re looking for are any substantial reductions in triglycerides on top of the treatments they’re already giving their patients, like omega-three fatty acids or fibrates. So they’ve already recognized that these patients need to be treated, and they’re not getting much triglyceride lowering with existing treatments, so they want something to produce a substantial lowering of triglycerides. Anything north of 50% is a big win in this patient population on top of standard of care, and that is what we expect.

With respect to AP events and what we’re hearing from physicians, we’re hearing exactly what I just said, is that they do not need to be convinced that these patients are in harm’s way for acute pancreatitis that can be fatal, and that we need to lower their triglycerides substantially to get them out of that way. They don’t need to see the AP advance. However, with that said, although the CORE and COR2 studies were not designed as AP outcome studies, we are seeing, as Richard mentioned in his formal remarks, we are seeing on a blinded basis acute pancreatitis events, and we expect to have more accumulated AP events in the combined COR and COR2 study than we had in the FCS BALANCE study. This gives us confidence we could have sufficient data to observe at least a favorable trend in olazarsen’s impact on AP within these studies, and we look forward to discussing more when we read out the data in September.

Gary Nachman, Analyst, Raymond James: Okay, great. And then just one quick one on Donnie with the PDUFA coming up on August 21. You’re in labeling discussions, so how you feel relative to your expectations, you know, if you think the switch data will make it on there, if it’ll be once every month and once every other month dosing, that option will be available for patients And sounds like you’re ready to launch basically right after approval, so just confirm everything is in place for that. Thanks.

Richard Geary, Chief Development Officer, Ionis: I’ll start. This is Richard. We’re definitely on track for the PDUFA date on August 21. Everything that we’ve been discussing with FDA indicates that that date is on track. And then I can’t speak to the launch, although everything looks to be in place, and we’re excited about it.

I think your other question in regard to what we get in the label is a matter of negotiation. It always is. And so we work with the agency. That’s certainly been our position. Let’s get that in there.

But we don’t need it in the label because it is published. And now that we have a publication and it’s related to the work that was done in the clinical trials, we have an opportunity to promote on that.

Brett Monia, Chief Executive Officer, Ionis: Kyle, maybe you could touch on the launch preparations.

Kyle Janae, Chief Global Product Strategy Officer, Ionis: In terms of launch readiness, we’re building on the synergies from our FCS capabilities. Our medical affairs teams have been out interacting with allergists and immunologists treating HAE. Our sales team is now in place, market access, marketing, patient support, our operations capabilities. So everything is ready to go in terms of launch readiness. As Richard was describing, the program from a regulatory standpoint, the overall totality of the data here for dinodolorsen is very, very strong.

From an efficacy standpoint, in terms of the durability and control of attacks, the tolerability using the easy to use self administered auto injector by the patient, and convenience. Here we have a program with the longest dosing interval available, potentially that will be available for patients living with HAE. The switch study is going to be helpful. This obviously is a switch market in The United States. The majority of patients are on a prophylactic treatment today.

And what we were able to demonstrate is that, one, patients are willing to switch. Two, they can do so safely and move over to Donnidolorsen. They can have an additional reduction of upwards of 62% improvement from baseline on an existing prophylactic treatment. And eighty four percent of the patients said that they preferred dinodolorsen over the existing treatment that they were on once they were switched. So the totality of the data combined with the timing of the launch here upcoming, hopefully on August 21, will be ready to go.

Brett Monia, Chief Executive Officer, Ionis: Thanks, Kyle.

Gary Nachman, Analyst, Raymond James: Great. Thanks for all that color.

Brett Monia, Chief Executive Officer, Ionis: Thanks, Gary.

Conference Operator: The next question is from the line of Yanan Zhu from Wells Fargo Securities. Please go ahead.

Yanan Zhu, Analyst, Wells Fargo Securities: Great. Thanks for taking our questions and congrats on a very strong quarter. Perhaps a first question about COR and COR2. So I think I heard in the prepared remarks that the cumulative AP event rate on a blinded basis is now above what you saw in FCS. So I just want to confirm, it seems like in the BALANCE study you had 13 events across 66 patients.

And just wanted to get a sense of the core and core two with across 1,000 plus patients, are we thinking about the rate above that? And could you also give us a sense that based on a core core two patient profile, what is the expected AP rate in untreated population like that? So that we can help us prepare for the data and the AP data. And I have a follow-up on the FCS and FHTG launch dynamic. Thanks.

Brett Monia, Chief Executive Officer, Ionis: Yanan, so you said the rate is higher. It’s not the rate, the rate is lower, of course, in SHCG. I think that’s why you knew that. What we’re talking about is the accumulated number of AP events in the CORE and CORE II studies combined. And I can confirm that there’ll be more events in CORE-two combined than in our FCS balanced study.

As far as what we think the AP I mean we don’t really know what the AP rate is. Study will be the first to actually really definitively provide information on what the AP event rate is in SHTJ, ain’t that right Eugene?

Eugene Schneider, Chief Clinical Development Officer, Ionis: Yeah absolutely right, there’s no outcome data that can inform us, so we’re eagerly awaiting the CORNCOR2 readout.

Yanan Zhu, Analyst, Wells Fargo Securities: Got it, got it, yeah, yeah, thanks. And on the SCS launch, and potentially the later launch of SHTG, congrats on a very strong second quarter of launch in FCS. It feels like opportunity in FCS is actually pretty sizable, given this momentum. You know, can you talk about, is this opportunity relative to your anticipation? Is it bigger or in line?

And more importantly, as we think about you going into SHTG with a much reduction in the pricing, presumably across the board for both indications, any strategy to protect the FCS opportunity in terms of timing of launch and also any way to manage the pricing change? That would be super helpful. Thank you.

Kyle Janae, Chief Global Product Strategy Officer, Ionis: Yeah, let me first talk about the FCS population that you described. I still believe that it’s representative up to 3,000 potential patients. It’s still early innings. The majority of these patients still are not diagnosed. Obviously, you know, several 100 patients have either participated in a clinical trial, or were waiting, for a medication because they had been previously diagnosed and needed a treatment.

First mover advantage here is very important, not only in FCS, but also in SHTG. So our work for the first half of this year was to convert our clinical trial patients, which we did effectively to help those patients that were previously identified with FCS to go on to treatment, and then to continue to expand our patient identification efforts. We’ve been spending a lot of time on that, as I referenced, in terms of reaching greater than 3,000 HCPs with our sales team, and reaching greater than 30,000 HCPs through our education, you know, related to our marketing and our omni channel efforts. So we’re continuing to educate. The time that we’re spending on FCS specifically is with physicians that are treating SHTG patients.

And so what we’re doing is having conversations around the implications of high triglycerides and how these patients present, and ultimately looking for a phenotype of an FCS patient in order to either clinically diagnose them or get a genetic confirmation of their disease. So that work is ongoing, and that’s what we’ll need to do through the second half of this year. You know, we’ve provided the guidance of 75 to $80,000,000 this year, which represents not only the patients that we’ve converted already in the first half, but the ongoing efforts that we believe will be successful with through the second half of this year. So I believe that the FCS population is in line with the up to 3,000 that we’ve represented up to this point. As it relates to SHTG, this is a much broader patient population obviously, and the treating physician population is much more significant.

Greater than a million patients have high risk SHTG, which is inclusive, obviously, of patients over 500 with a history of AP, or patients over, eight eighty milligrams per deciliter. So, there’s a lot of effort already around education in this population, and working to identify, you know, these potential patients, within these practices as we go out and have these interactions. As it relates to the pricing dynamic, we’re still working through that. There’s more work for us to understand in terms of the data, which we are still waiting for. And then we’ll work obviously with the payer community to understand, the budget impact of the patient population, their interpretation of the data, and obviously the value that that represents to patients.

So that’s the work that we will do, going into next year, and ultimately we will announce price upon the approval of the SHTG indication, pending positive outcome there.

Yanan Zhu, Analyst, Wells Fargo Securities: Great. Thanks for all the color. Congrats on the quarter again.

Brett Monia, Chief Executive Officer, Ionis: Thank you.

Conference Operator: Thank you. The next question is from the line of Steven from TD Securities. Please go ahead.

Wade Walke, Senior Vice President of Investor Relations, Ionis0: Hi, everyone. This is Steven Eynav on for Yaron Werber. Congrats again on a fantastic quarter. One more on DAWN A and on competition specifically. It looks like TAKHZYRO revenues are stable and Orlodayo seems to be growing.

Any visibility also as well on the Andembri launch, which was approved on June 16. Obviously, that drug has a couple of disadvantages, including monthly dosing versus versus Donnie’s two months and also requiring a loading dose, which Donnie does not. Any visibility on the competition and maybe insights from there on what the Donadolorsen launch could look like? Thanks.

Kyle Janae, Chief Global Product Strategy Officer, Ionis: Yeah, I’d be happy to talk about that. This is Kyle. I think what we know in this marketplace and what’s come through very clear is that patients on existing prophylactic treatments are not completely satisfied, and that they would be willing to switch to a new therapy. A recent Harris poll said that greater than ninety percent of patients actually would look to switch to an improved therapy. And I think you’re seeing that play out with a newly approved treatment already.

And we would expect to see the same from Donnievelorsen. The profile overall with Donnievelorsen is very strong, as mentioned. Not only the efficacy data, but also the ability for patients to self administer. As you pointed out, this therapy is gonna be potentially prescribed every four weeks or every eight weeks, depending upon how patients present, and then how stable they are with their disease. So that is, again, another key differentiator here.

And then finally, I’ll just mention the switch study. I mean, what we have demonstrated is that patients are willing to switch. When they do so, they can do so safely. They have, the potential to improve, the control of their disease. And, ultimately, when they’ve ended up on Donnie Dulorsen, they’ve preferred it over the treatment that they were on before.

So I believe from competitive standpoint, we’re in really good shape. And we’ve got a great team. We’ve hired a sales organization that has experience in the allergy and immunology space, many of whom have direct experience for many years. And, they know the treating physicians, and they’ve got, you know, not only existing relationships, but they understand how to competitively sell. So we’re really excited about the upcoming launch potentially with the approval on August 21.

Wade Walke, Senior Vice President of Investor Relations, Ionis0: Thank you very much.

Conference Operator: Thank you. The next question is from the line of Mike Holes with Morgan Stanley. Please go ahead.

Wade Walke, Senior Vice President of Investor Relations, Ionis1: Great. Good afternoon. Thanks for taking the question and congratulations on the strong quarter as well. Maybe just a question on the Phase III SHTG core data. Previously, you suggest data 3Q, you’re narrowing that a little bit to September.

I guess, first question, just any what’s the rationale there or anything behind that? And then secondly, last quarter, you suggested AP data might not be available in the top line release. I guess just maybe give us a sense of what level of AP data you plan to share in the top line release in September? Thanks.

Brett Monia, Chief Executive Officer, Ionis: Sure, Mike. Just refining the timeline to September from the second half of the year is just blocking and tackling, getting the studies completed, database locked, the data cleaned and all that. So we just felt it was appropriate to provide more specificity on the exact timing. So nothing significant behind there except operational in conducting studies. We will provide a statement on AP in our top line press release.

Our primary endpoint again is triglyceride lowering. We will provide top line information on triglycerides as well as safety, overall safety, and we’ll provide a statement on AP. What’s also I think even more important is that we will provide and present the full data set at a medical congress in the second half of this year and we look to publish the data in the second half of this year as well.

Wade Walke, Senior Vice President of Investor Relations, Ionis1: Very helpful. Thank you.

Conference Operator: The next question is from the line of Andy Chen from Wolfe Research. Please go ahead.

Wade Walke, Senior Vice President of Investor Relations, Ionis2: Hey. Brandon on for Andy. On whenua and polyneuropathy, where do you think those patients are coming from? Do you think that they are you’re winning new, winning new to brand share or stealing patients from Alnylam. If you can provide any quantitative splits on those details, that’d be great.

Thank you.

Kyle Janae, Chief Global Product Strategy Officer, Ionis: I’d be happy to talk about that, Brandon. Thanks. This is Kyle again. First, I’ll just say, WANUA continues to perform very well. Strong demand.

The polyneuropathy market is a growth market. The majority of these patients are not diagnosed yet, and that’s exactly where AstraZeneca is spending the time, is looking for and educating HCPs around hereditary polyneuropathy patients. Dollars 44,000,000 in Q2, which obviously was growth over Q1. The feedback that we’re getting from HCPs is continuing to be very positive around quality of life and efficacy and the control of the polyneuropathy symptoms on a consistent basis. The ability to self administer with an auto injector continues to be a differentiating factor.

And, physicians and patients very much appreciate the opportunity to manage their disease on their own without having to come into the HCP’s office in order to have a simple injection provided to that patient. So in a simple response here, these are new to brand patients. These are patients that are newly identified predominantly. We are seeing some switches, as you would expect, and we are seeing some combination use along with patients that are progressing on the stabilizers today. But really our focus is growing the polyneuropathy market and making sure that newly identified patients get prescribed whenua.

Richard Geary, Chief Development Officer, Ionis: Next question.

Conference Operator: The next question is from the line of Jenna Wang from Barclays. Please go ahead.

Wade Walke, Senior Vice President of Investor Relations, Ionis3: Thank you for taking my questions. Also, congrats on the very strong quarter. So I have two questions regarding Transgoza. When I look at the guidance for FCS this year, seventy five to eighty million, even if we’re using 80,000,000, seems every quarter is 3Q for only 5,000,000 growth. So maybe give us a little bit more color.

You know, are you is the guidance too conservative, or is there a reason that why, you know, the new patient add on was slowed down compared to 2Q over 1Q? So that’s the first question. And the second, I don’t know if you can comment. I think that the total accumulative events for core and core two will be more than 13. So giving, you know, certainly different disease and the prior history of AP in the two studies.

So maybe, like, how much more? Are we talking about the total AP event just in the teens, or could that be in the 20s?

Wade Walke, Senior Vice President of Investor Relations, Ionis2: Thanks, Gina. Beth, do you

Brett Monia, Chief Executive Officer, Ionis: want to touch on the 75,000,000 to $80,000,000 guidance?

Beth Haugen, Chief Financial Officer, Ionis: Yeah, actually, I think I’ll just toss that to Kyle because that’s in his shop. He’s responsible for that.

Kyle Janae, Chief Global Product Strategy Officer, Ionis: No, so I think what’s most important here is the way that we progress throughout the launch, right? As I referenced, we converted the clinical trial patients. We’ve been working very effectively with patients that were identified prior to the approval in order to get those patients formally diagnosed and put on to treatment. So you had a number of patients that were previously identified. So where we’re at right now with the launch is really looking for newly identified and newly diagnosed patients, right, in order to penetrate deeper into that up to three thousand FCS patient population that we believe exists in The US.

So that takes some time. It takes a lot of education, and it takes a lot of conversations. And then it also takes a little bit of time for physicians to get potential patients into the practice in order to be either clinically or genetically confirmed with their condition. So we’ve done a nice job throughout the first half of the year, and we expect to continue to identify these patients. But it just might take us a little bit of time because of the complexity of the rare disease that we’re dealing with.

And being first to market, and having a great product with positive physician and patient experience is helping us to increase that patient identification over time.

Wade Walke, Senior Vice President of Investor Relations, Ionis2: Yeah, thanks Kyle. And yeah, on your second question,

Brett Monia, Chief Executive Officer, Ionis: Gina, we’re not going to go more than that. I mean, in this call today we confirmed that we will see more AP events in the core and core two studies combined than we saw in FCS balance,

: we

Brett Monia, Chief Executive Officer, Ionis: confirmed that we will make a statement summarizing, know, to some extent, top line summary of AP findings in core and core two. We’re not gonna go further than that on how many more AP events we see. We look forward to sharing the date in September.

Conference Operator: Thank you. Next question is from the line of Luca Izzi from RBC. Please go ahead.

Wade Walke, Senior Vice President of Investor Relations, Ionis4: Well, great. Thanks so much for taking my question and congrats on a great quarter here. Maybe, Brett, if I can circle back on one statement you made earlier, you know, the physicians appreciate that triglycerides are the bad guys, so to speak, and you know, they don’t need to see a stat seg on acute pancreatitis. Is that a US comment or do you think that is applicable also to the rest of the world? I guess in a scenario where you don’t show stat seg or strong trend on AEP, how should we think about the uptake of this drug outside The United States?

And then I know I’m probably pushing my luck here, but you mentioned you’ll have a statement on AEP in the press release. Will that be a qualitative statement or quantitative statement? Thanks so much.

Brett Monia, Chief Executive Officer, Ionis: We’re not going to go more than that, Luca, on what we’re going to say. We’re going to we will speak to our findings in COR and COR2 on AP in our top line press release and share for the full data set at a medical congress this year, so we’re just gonna hold off on that one. Your first important question, because really what you’re getting at is payer dynamics outside of The US. Kyle will address that. But I can tell you that in The US and I have spent a lot of time with lipid specialists over the last two years who treat SHTG patients.

They are convinced that they need to get patients on the most effective triglyceride lowering agents possible if SHTG patients, because they are convinced, they know that their patients are in harm’s way for acute pancreatitis. In fact, they’re already treating them. They’re treating them with everything they have, whether it be fibrates or omega-3s, they’re just not effective, so they’re looking for something on top of standard of care. As a reminder, our core and core two studies are on top of standard of care to get their triglycerides down, and that’s true for payers in The United States too. Outside The US, let me maybe focus on Europe, it’s a little bit more complex, right?

Kyle Janae, Chief Global Product Strategy Officer, Ionis: It is. From a regulatory standpoint though, obviously triglyceride lowering is gonna be sufficient for a label in the EU, and that filing is there, and we’ve obviously positive CHMP opinion and optimistic about the outcome and where that goes in terms of the approval. So from a payer and reimbursement dynamic standpoint, you know, in the European countries, for example, it’s really about the breadth of the population that you’re ultimately going to be able to treat. You know, who to treat and why to treat those patients, right, is the key thing here in order to demonstrate outcomes in the patient population. One thing that’s very positive already is if we are able to secure an indication in Europe, is we’ve got FCS.

FCS already has AP data, it already has very strong hospitalization data, and so we’ve got information to be able to substantiate pricing and reimbursement from an FCS standpoint. We also have a very strong partner in Sobe, who has been in this market for many years, and they’ve navigated pricing and reimbursement very successfully in the FCS population, already across numerous countries, across Europe. And, I think we’re very optimistic that we’ll be able to continue that success, with a program like Trin Golza. So then the question becomes SHTG, if you have a potential label expansion, and what do you need to show there? Outcomes in AP are obviously going to be important for the broad population, But could you potentially look at a different population a little bit more narrow than anybody over 500?

For example, patients that are at high risk SHTG, such as patients over eight eighty, patients with 500 and a history of AP, or potentially patients with other comorbidities. So there are a lot of dynamics and things to think through there, and pending the data, obviously we’ll work through putting that body of evidence together and supporting Sobe in their pricing and reimbursement efforts across each country as they go to launch.

Wade Walke, Senior Vice President of Investor Relations, Ionis4: Got it. Thanks so much.

Brett Monia, Chief Executive Officer, Ionis: Thanks, Lucas.

Conference Operator: The next question is from the line of David Lebowitz from Citi. Please go ahead.

Wade Walke, Senior Vice President of Investor Relations, Ionis4: Hi. Thanks for taking my question. Given the strong early ramp of Trimgolsa and SCS, are you how are you thinking about the pricing change that would inevitably happen when it gets approved for severe hypertriglycerides? Is it just going to be a pretty prompt drop, or are you going to try to find some way to, observe the revenues while you’re dropping the price, and ramping up?

Kyle Janae, Chief Global Product Strategy Officer, Ionis: Yeah, thanks, David. So our pricing work is ongoing. We’ve got more work to do there. You know, what we’ve communicated up to this point is in a disease that has three to four million patients, typically The US payers are accepting of a price somewhere in the 10 to $20,000 price range. However, when we’re looking at SHTG specifically, and the data that olicersen can potentially have population.

We’d like to go back to the payers and do more testing and understand exactly where this price point could land based on the value to patients and the value to the payers. So there’s more work to do. In terms of the execution of that, you know, the FCS population ultimately will be consumed within the SHTG population. So it will become, you know, anybody over 500 would be within the SHTG population. So, there is a question around, you know, how do you execute that and the pricing dynamics, and do you bring the price down?

I think regardless of what the decision is there, if we do it immediately, or if it’s done over time, what we do know is that the SHTG population is quite substantial. And, I think what we believe is that we’ll be able to maintain enough patient population to be able to continue the revenues that we’re producing at the time of an SHTG potential approval.

Wade Walke, Senior Vice President of Investor Relations, Ionis4: Thanks for taking my question.

Conference Operator: The next question is from the line of Jay Olson from Oppenheimer. Please go ahead.

Wade Walke, Senior Vice President of Investor Relations, Ionis5: Oh, hey, congrats on the quarter and thank you for providing this update. Now that you have the early successful launch experience with Tringosa, you have several other launches that you’re planning in the near term. How does that impact your thinking about your earlier stage pipeline and deciding between out licensing earlier stage assets versus retaining full ownership and launching them independently? And then as a follow-up, can you discuss any plans to build out your ex US infrastructure for any future global product launches in the long term? Thank you.

Brett Monia, Chief Executive Officer, Ionis: Thank you Jay. We have an abundance of riches when it comes to our late stage pipeline, our wholly owned pipeline, our partnered pipeline, it’s a nice combination there. And our first independent launches off to a really good start, and we’re expecting Donahue Worsen to be equally successful as well as SHTG coming up next year. With that said, our research organization is incredibly innovative and prolific. We have to continue to bring in potentially transformational medicines continuously into the early stage pipeline and bring them to phase three development in due course.

Our focus remains, as we set out five years ago, five and a half years ago, to do is to prioritize the wholly owned pipeline. We’ll continue to do so and we will focus on cardiology and cardiometabolic diseases as well as neurology. We will always have room for exceptions like hereditary angioedema, we have such an excellent looking drug like Donipilorsen, but those will be our focus. Those areas will be our focus and it will be the priority. With that said, we still need to ensure that we live within our means.

We have limited, you know, our resources are limited. We are committed to achieving positive cash flow in the next few years. And although we will prioritize our wholly owned pipeline early stage through phase three development, there will always be assets that either are outside of our means or outside of our priority areas, which where we will make decisions to partner. A lot of interest in partnering with Ionis these days, our platform is delivering over and over again, a great example of that is the sapaplersin transaction that we did earlier this year for polycythemia vera with Ono, dollars $280,000,000 up front, and we did that because it’s outside of our areas of focus. Hematology is not a focused area for us.

So you’ll see us continue to partner, but that’s not our priority. Our priority is our wholly owned pipeline, early to late stage, and our launches out. With respect to outside The US, I’d like to just put that one on pause. We’re thrilled with the early launch of Trangolza and we’re looking forward to Donadolorsen and SHCG launches in The US. And we’ll know when it’s time to emerge from The US and maybe start building external or ex US commercial infrastructure, but now is not the time to be distracted with that.

We we need to get these launches right. We’re committed to get these launches right, and we’ll do so. With that said, we, of course, are having discussions internally on when when and what that asset might be that we emerge from The US market with, but it’s we’re in the early innings in those discussions.

Wade Walke, Senior Vice President of Investor Relations, Ionis5: Thank you. Super helpful. Congrats again on the launch.

Conference Operator: The next question is from the line of Akash Tewari from Jefferies. Please go ahead.

: Hi. This is Zaki on for Akash Tewari. Thanks so much for taking our question. So in the BALANCE study, looks like there was, like, one AP event that kinda leaked through towards the end of the study in the eighty milligram arm in a patient who did have triglycerides lowered on drug, whereas placebo events, they seem to cluster earlier on in the first half of the study. So I understand it’s really small end here, but now that you have ESSENCE data and, you know, some sense of how blinded events are tracking in Core one and two, how confident are you that this kind of variability won’t be a swing factor on AP powering in Core one and two?

Thank you.

Brett Monia, Chief Executive Officer, Ionis: I’ll start, and then Eugene, jump in. So we’re doing everything we can to power to increase the power for AP in our study if possible. AP analysis will be combined, COR and COR2, and will be at the twelve month time point. That’s correct, right, Eugene? So although the primary endpoint for triglycerides is at six months, AP secondary endpoint will be at twelve months with COR and COR2 combined.

So we’ll have the what we’re trying to do is maximize time and maximize patient numbers.

Eugene Schneider, Chief Clinical Development Officer, Ionis: How much do I have, Brett? The maximal power will be achieved by combining COR and COR2, and ensuring that the N is much larger than two separate elements.

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