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VIDEO LIBRARY
TRYNGOLZA Mechanism of Action Video
TRYNGOLZA MOA
See how TRYNGOLZA works for adult patients with sHTG.
Chapter 1: The validated technology behind TRYNGOLZA® (olezarsen)
Familial chylomicronemia syndrome, or FCS, is an underdiagnosed form of severe hypertriglyceridemia.
Historically, treating patients with FCS was a challenge due to a lack of effective pharmacologic interventions.
TRYNGOLZA is the first FDA-approved therapy for adults with FCS, indicated as an adjunct to diet to reduce triglycerides.
Today, we will explore the underlying causes of FCS and the unique and targeted mechanism of action of TRYNGOLZA.
Chapter 2: Familial chylomicronemia syndrome (FCS): from pathology to presentation
Our understanding of the pathology of FCS begins with triglyceride-rich lipoprotein, or TRL, metabolism.
FCS manifests as hypertriglyceridemia from the impaired metabolism of TRLs, such as chylomicrons and very low-density lipoproteins, or VLDL.
Normally, TRLs are hydrolyzed by lipoprotein lipase, or LPL, which breaks down dietary and endogenous triglycerides.
In FCS, loss-of-function mutations in the LPL gene or its cofactor genes lead to reduced or absent LPL activity. This impairs fat processing, resulting in plasma triglyceride levels persistently 10 to 100 times above normal.
Apolipoprotein C-III, or apoC-III, regulates TRL metabolism by inhibiting LPL and one or more LPL-independent pathways such as hepatic clearance.
The triglyceride buildup raises the risk of acute pancreatitis, pancreatic necrosis, organ failure, and type 3c diabetes, making effective and substantial triglyceride lowering imperative.
Chapter 3: TRYNGOLZA: a highly targeted therapy
TRYNGOLZA is a GaINAc-conjugated antisense oligonucleotide, or ASO, that inhibits the production of apoC-III, a key regulator of TRL metabolism and hepatic clearance.
The conjugation of GalNAc to the ASO enables targeted delivery to the liver, where apoC-III messenger RNA, or mRNA, is generated.
GalNAc binds to the asialoglycoprotein receptor and allows TRYNGOLZA to enter the hepatocyte.
In the hepatocyte, TRYNGOLZA directly and selectively binds to apoC-III mRNA and induces RNase cleavage of apoC-III mRNA, leading to its degradation.
This can result in a decrease in serum apoC-III protein levels and lower plasma triglyceride levels, which are known to impact the risk of acute pancreatitis.
Building on validated GalNAc-conjugated ASO technology, the targeted design of TRYNGOLZA represents a designated breakthrough therapy for adults with FCS as an adjunct to diet, finally giving patients and physicians a targeted and potentially effective solution for this critical and often underrecognized condition.
FCS=familial chylomicronemia syndrome; sHTG=severe hypertriglyceridemia.
TRYNGOLZA Injection Training Video
Injection Training
A resource for explaining the self-administration process to patients with severe hypertriglyceridemia (sHTG) or familial chylomicronemia syndrome (FCS).
Hi, I'm Maya, and I take TRYNGOLZA. When I first started using TRYNGOLZA, I had questions about the injection process. You might, too—that is completely normal. Once I understood the steps, the entire process became straightforward.
Now it's part of my routine, and today I'm going to walk you through exactly how to inject TRYNGOLZA using the autoinjector, so you can feel confident each time you inject.
We'll walk through this process one step at a time, so nothing feels rushed or overwhelming. Let's start with a few basics.
TRYNGOLZA comes in a convenient single-dose autoinjector that you can use at home or on the go. You should do your injection in a place where you feel comfortable.
It's available in 50- and 80-milligram doses, depending on what your healthcare provider prescribes. The label on your autoinjector may look slightly different depending on the dose, but the injection steps are exactly the same.
Each autoinjector contains 1 dose and is meant to be used only once. You'll inject it on the same day each month.
When I inject, I like to choose a space that feels familiar and I take the injection at roughly the same time of day. It helps make the process feel a little more predictable.
I also like to set a reminder on my phone so I don't forget.
Before your first injection, your healthcare provider will show you—and, if needed, your care partner—how to use the autoinjector.
Be sure to read the complete Instructions for Use and Patient Information that are available on TRYNGOLZA.com, and included in the packaging.
Now let's prepare for the injection.
Before I start, I make sure I have my autoinjector and a few other supplies I'll need so I'm not searching for anything while I'm injecting. That includes an alcohol wipe, gauze or a cotton ball, a small bandage, and a sharps container.
If you've been storing your autoinjector in the refrigerator, take it out and let it come to room temperature for 30 minutes before injecting. I usually take mine out while I'm getting ready in the morning. That way, it's ready when I am.
Let it warm up on its own. Don't try to speed things up with a microwave or hot water.
While it's coming to room temperature, let's take a quick look at the packaging.
You'll see the dose strength clearly listed on the front of the carton. Since I take the 80-milligram dose, the label here is purple.
But depending on what your healthcare provider prescribed, yours may look slightly different. Remember, no matter which dose you're prescribed, the injection process is exactly the same.
Now let's take the autoinjector out of the carton.
This clear cap fits over the orange needle shield and helps protect the needle. Under the orange needle shield is the needle itself. Here's the viewing window—you'll be able to see the medicine and the orange plunger rod through it.
It's important to inspect your autoinjector before use. Once it's warmed, check the expiration date on the label, and make sure the clear cap is securely attached.
If the expiration date has passed, or the clear cap isn't in place, don't use the autoinjector.
Next, look at the medicine through the viewing window. It should be clear and colorless to yellow, without particles. Small air bubbles are normal.
Now it's time to choose an injection site.
You can inject into your abdomen or the front of your thigh. If someone is helping you, the back of the upper arm is also an option. If you're injecting into your abdomen, choose a spot at least 2 inches away from your belly button.
No matter where you inject, avoid areas that are bruised, tender, red, hard, or scarred.
For today's demonstration, I'll be using the front of the thigh.
Before injecting, I always wash my hands with soap and water. After washing my hands, I like to sit somewhere comfortable, usually the same spot each month. It helps me feel settled.
Then I clean the injection site with an alcohol wipe, using a gentle circular motion. Let the skin air dry. It only takes a moment. Once it's dry, we're ready to move on.
First, hold the autoinjector in the middle, with the clear cap and needle facing away from you. Next, pull the clear cap straight off, making sure not to twist it. Go ahead and dispose the clear cap in your sharps container.
What you're seeing now is the orange needle shield. The needle stays protected underneath it. Avoid pushing the shield against your hand or finger.
Now we can begin the injection.
Place the orange needle shield flat against the skin at a 90-degree angle and make sure you can see the viewing window.
Push firmly and hold straight against the skin. You'll hear a click when the injection begins. You may hear a second click, too. Don't worry, that's normal. The injection isn't finished yet.
Hold it in place for 10 seconds to make sure the full dose is delivered. Keep the device steady the entire time. Steady pressure helps prevent what's sometimes called a "wet," or pre-activated, injection, where the medication leaks onto the skin.
After 10 seconds, check the viewing window. The orange plunger rod should have moved down to fill it completely.
If it hasn't, you may not have received the full dose. In that case, contact your healthcare provider. If it has, the dose is complete.
If everything looks complete, lift the autoinjector straight up. After removal, the orange needle shield automatically locks into place and covers the needle.
Because the autoinjector is for single use only, place it in a sharps container right away. Do not throw it away in your household trash.
You may notice a small amount of blood or liquid at the injection site. Don't be alarmed. If needed, you can gently press a cotton ball or gauze on the area and apply a small bandage.
And that's the full injection process. Once you've done it a couple of times, it becomes part of your routine, just like it did for me.
I hope walking through it together helps you feel more comfortable when it's your turn to inject TRYNGOLZA. If you ever have questions, speak with your healthcare provider.
Please read or listen to the Important Safety Information that follows.
FCS=familial chylomicronemia syndrome; sHTG=severe hypertriglyceridemia.
Launch Broadcast Video
sHTG Launch Broadcast
Leading clinicians in the field of lipidology discuss the approval of TRYNGOLZA for adults with severe hypertriglyceridemia (sHTG).
JEFF BACHMAN
Welcome, everybody. Please stand by as we give a few minutes to allow more attendees to join us. We will begin in just a few moments. Thank you.
Welcome! My name is Jeff Bachman, and I'm a Medical Director at Ionis Pharmaceuticals.
It is my distinct honor today to present this live broadcast on TRYNGOLZA—the first and only FDA-approved therapy indicated as an adjunct to diet to reduce triglycerides and the risk of acute pancreatitis in adults with severe hypertriglyceridemia, or sHTG, which is categorized as triglyceride levels greater than or equal to 500 mg/dL.
With me today, it is my pleasure to introduce our guests: Dr. Bajaj, Dr. Budoff, and Marianna Keegan.
Dr. Bajaj is an Associate Clinical Professor at the University of Pennsylvania.
Dr. Budoff is a Professor of Medicine at the David Geffen School of Medicine at UCLA.
Marianna Keegan is an Associate Director of Patient Services at Ionis.
Welcome, everybody, and thank you for being here with us for this exciting presentation.
Before we begin, I'd like to share a few important disclosures. This presentation is sponsored by, and the panelists are participating on behalf of, Ionis Pharmaceuticals. This presentation is not approved for continuing medical education credit.
Today's presentation, we will be discussing the burden of living with sHTG and the associated risks; the TRYNGOLZA mechanism of action; Ionis' pivotal trials of TRYNGOLZA in adults with sHTG, CORE and CORE2, including safety and efficacy; and the steps for getting your patients with sHTG started on TRYNGOLZA.
We will then close today's discussion with audience questions. To submit your questions, please utilize the question submission feature on the left side of your screen.
To download the TRYNGOLZA Prescribing Information, please go to the Important Safety Information section on the right side of your screen in the chat section, or visit TRYNGOLZAHCP.com.
To start us off, let's have Dr. Budoff and Dr. Bajaj take us through the unmet need faced by people living with sHTG. Dr. Budoff.
DR. MATTHEW BUDOFF
Thank you, Jeff.
It's my pleasure to join you here today. Let's start by grounding ourselves in what we mean when we talk about severe hypertriglyceridemia. sHTG is defined as fasting triglyceride levels of 500 mg/dL or greater, and it isn't driven by a single cause. There's a genetic component, ranging from a broad polygenic background to rare, biallelic pathogenic variants in genes such as LPL, APOC2, and APOA5. And metabolic factors—obesity, insulin resistance, diabetes, alcohol consumption—can further impair triglyceride clearance while also driving increased hepatic VLDL production and secretion. Regardless of the driver, the biological result is the same: impaired lipolytic activity leads to toxic accumulation of triglyceride-rich lipoproteins, increasing the risk of acute pancreatitis, or AP, and atherosclerotic cardiovascular disease, or ASCVD.
Tying all of this together is apoC-III, a pivotal regulator of triglyceride metabolism, inhibiting LPL activity and impairing hepatic clearance of these lipoproteins. We'll come back to apoC-III when we discuss how TRYNGOLZA works, but keep it in mind as we move through the sHTG landscape.
So we've established that severe hypertriglyceridemia is driven by a combination of genetic and metabolic factors, all converging on the same biological threat.
Now let's look at what that means clinically. When triglyceride levels cross the 500 mg/dL threshold, the risks become very real. As you can see on this slide, we're looking at a spectrum. Approximately 75% of the sHTG population falls in the 500 to 880 mg/dL range, with the remaining 25% at levels above 880. As the triglycerides rise across this spectrum, so do the risks. The risk of AP increases significantly at 500 and above, driven by the substantial rise in chylomicrons. This is exactly why expert guidelines highlight the urgency in lowering triglyceride levels that are 500 mg/dL or above to reduce both of these associated risks.
Before we go further, I want to spend a moment on something that's easy to overlook in clinical practice, and that's what happens to triglycerides after meals. While fasting triglyceride levels give us an important baseline, they don't tell the full story. Postprandial triglyceride increases can be substantial. Following a typical meal, dietary triglycerides peak over 3 to 5 hours, and because patients typically eat multiple times per day, those peaks accumulate.
As you can see in this graph, even in an average individual with normal fasting triglycerides, levels remain elevated above baseline for most of the day. Now consider what that looks like in a patient with sHTG, where fasting levels are already at or above 500—the postprandial surges on top of that baseline can be dramatic.
This is important because these postprandial elevations contribute directly to increased risk of both acute pancreatitis and ASCVD. It's a reminder that the risk our patients face isn't limited to what we capture on a fasting panel. It's happening throughout the day, after every meal. So we've established that triglyceride levels in patients with sHTG are elevated not just at fasting but throughout the day.
Now let's look at which lipoproteins are driving the clinical risks we've been discussing. At the lipoprotein level, it's chylomicrons and VLDL—and their remnants—that are the key culprits. Chylomicrons and VLDL contribute to acute pancreatitis risk, while VLDL and remnant cholesterol are the primary contributors to ASCVD. So we're dealing with 2 potentially life-threatening consequences in these patients, and both are driven by the accumulation of these triglyceride-rich lipoproteins.
JEFF BACHMAN
Thank you, Dr. Budoff. Now I'd like to turn it over to Dr. Bajaj to walk us through the risks of acute pancreatitis.
DR. ARCHNA BAJAJ
Thank you, Jeff. Let's put the acute pancreatitis risk into perspective, because the data here are striking. Adults with triglycerides between 500 and 879 mg/dL face more than a 5-fold higher risk of acute pancreatitis compared with those who have normal triglyceride levels. And at triglyceride levels of 880 or above, that risk jumps to more than 17-fold, driven by the accelerating accumulation of VLDL and chylomicrons at these levels. And the risk doesn't plateau. Acute pancreatitis risk continues to increase by approximately 3% with every 100 mg/dL rise in triglycerides once levels reach and exceed 1000. So, for patients at the higher end of the spectrum, the risk compounds quickly. And this isn't just a statistical observation.
The American Heart Association, the American College of Cardiology, the National Lipid Association, the American Diabetes Association, and the Endocrine Society all identify triglyceride levels below 500 as a critical threshold for reducing acute pancreatitis risk. That degree of consensus across organizations really underscores how seriously the medical community views this danger. And when acute pancreatitis does strike in these patients, the consequences can be devastating, which is what I'd like to turn to next.
When acute pancreatitis does occur in patients with severe hypertriglyceridemia, the consequences can be severe. We're talking about mortality rates as high as 8%, irreversible multisystem organ damage including pancreatic necrosis, prolonged hospitalizations averaging around 17 days, beta-cell dysfunction that can lead to pancreatogenic diabetes, and healthcare costs approaching $100,000 per episode.
But what I really want to emphasize is this: Even a single episode of acute pancreatitis can have significant consequences. Once a patient has had 1 episode, the chance of recurrence is as high as 24%. And after a second event, the risk of a third rises to 49%. That's why striving to prevent the first episode is so critical, because once that cycle begins, it becomes increasingly difficult to break.
So given the severity of what we just discussed—the escalating acute pancreatitis risk, the irreversible organ damage, the compounding recurrence—the question becomes: Are our current treatments getting the job done for most patients? In many cases, they're not. Lifestyle modifications, fibrates, statins, and omega-3 fatty acids can all play a role in lowering triglycerides. But in real-world practice, sustained adherence to lifestyle changes is difficult, and pharmacologic therapies often don't reduce triglyceride levels sufficiently on their own.
For omega-3 fatty acids specifically, a guideline from the American Association of Clinical Endocrinology found insufficient evidence that they prevent pancreatitis—in fact, there were no studies evaluating the effectiveness of omega-3 fatty acids in preventing acute pancreatitis in patients with severe HTG. And while GLP-1 receptor agonists offer glycemic and weight-loss benefits, their impact on triglyceride reduction is often modest and insufficient for patients with sHTG.
So while these tools have value—and maximizing standard of care remains important—there remains a clear unmet need for a therapy that can deliver robust, sustained triglyceride lowering and meaningful acute pancreatitis risk reduction—for patients who, despite our best efforts, aren't getting to goal.
JEFF BACHMAN
Thank you, both. We've just heard about the significant burden sHTG places on patients, from the risks of acute pancreatitis and ASCVD to the gaps in our current treatment approaches.
Now we'll hear from Dr. Bajaj on how TRYNGOLZA works in adults with sHTG.
DR. ARCHNA BAJAJ
Thank you, Jeff. So earlier, Dr. Budoff discussed how apoC-III acts as a pivotal regulator of triglyceride metabolism, inhibiting activity of lipoprotein lipase, or LPL, and impairing the hepatic clearance of triglyceride-rich lipoproteins across the full spectrum of severe hypertriglyceridemia. TRYNGOLZA targets apoC-III directly at its source.
As you can see on this slide, TRYNGOLZA is a GalNAc-conjugated antisense oligonucleotide. The GalNAc conjugation enables targeted delivery to the liver, where apoC-III mRNA is generated. Once there, TRYNGOLZA works through a three-step process: first, it targets hepatocytes; second, it selectively binds to apoC-III mRNA; and third, it degrades that mRNA, resulting in reduced production of the apoC-III protein and, ultimately, reduced triglyceride levels.
What's important to emphasize is that this mechanism works through both LPL-dependent and -independent pathways, which means it is relevant across the full spectrum of severe hypertriglyceridemia, including familial chylomicronemia syndrome, or FCS, the most severe genetic form of sHTG, for which TRYNGOLZA has been FDA approved since 2024. We've just seen how TRYNGOLZA is designed to work.
Now let's look at what that translates to pharmacodynamically, starting with data from the CORE and CORE2 trials, which were the pivotal phase 3 placebo-controlled trials of olezarsen in adults with severe hypertriglyceridemia. This chart shows the placebo-corrected percent change in fasting apoC-III from baseline in the CORE trial. ApoC-III, as we discussed, is a key regulator of triglyceride metabolism and serves as the primary biomarker of TRYNGOLZA activity. In CORE, the reductions were substantial across both doses. At the 80 mg dose, apoC-III was reduced by 77% at Month 6 and 72% at Month 12. The 50 mg dose showed similarly meaningful reductions: 68% at Month 6 and 67% at Month 12. And importantly, these reductions were sustained over the full year of treatment.
The CORE2 trial tells a consistent story. Again, looking at placebo-corrected percent change in fasting apoC-III from baseline, TRYNGOLZA demonstrated robust and durable reductions across both doses. At the 80 mg dose, apoC-III was reduced by 63% at Month 6 and 58% at Month 12. At the 50 mg dose, reductions were 57% at Month 6 and 53% at Month 12.
Once again, these reductions were maintained over the full year of follow-up. Taken together, the CORE and CORE2 data give us a consistent and compelling pharmacodynamic picture: sustained apoC-III suppression across 2 trials. And these consistent reductions in apoC-III are what set the stage for the triglyceride lowering and clinical outcomes we'll review next.
JEFF BACHMAN
Thank you, Dr. Bajaj. We've seen how TRYNGOLZA targets apoC-III at its source to reduce triglycerides through both LPL-dependent and -independent pathways. Now we'll hear again from Dr. Budoff on the pivotal CORE and CORE2 trials of TRYNGOLZA in adults with sHTG.
DR. MATTHEW BUDOFF
Thank you, Jeff. Let's walk through the pivotal trials that established the safety and efficacy of TRYNGOLZA in adults with sHTG. TRYNGOLZA was evaluated in the largest pivotal program in sHTG: 2 identically designed, randomized, double-blind, placebo-controlled, phase 3 studies—CORE and CORE2—which together enrolled a total of 1061 adults with severe hypertriglyceridemia. As you can see in the trial design here, patients in both studies were randomized to receive TRYNGOLZA 50 mg, TRYNGOLZA 80 mg, or placebo, administered once every 4 weeks. The primary analysis was conducted at 6 months, and patients continued treatment over a full 53-week period. Across the 2 trials, there were 354 participants in the 50 mg arm, 351 in the 80 mg arm, and 356 in the placebo arm.
Now let's look at who was enrolled in these trials and what we were measuring.
The primary endpoint—shown here at the top—was the mean percentage change in fasting triglyceride levels from baseline to Month 6 for both the TRYNGOLZA 50 mg and 80 mg doses compared with placebo. The key inclusion criteria for CORE and CORE2 included fasting triglycerides levels at or above 500 mg/dL, participation in a 4- to 8-week screening and qualification period that included at least 2 weeks of diet and lifestyle stabilization, and adherence to optimized, stable background lipid-lowering therapy prior to enrollment and maintained throughout the study.
The key exclusion criteria for CORE and CORE2 included genetically confirmed familial chylomicronemia syndrome, acute pancreatitis at the time of screening or in the 4 weeks before screening, and poorly controlled diabetes mellitus.
Before we get into the results, let's take a moment to review what was measured beyond the primary endpoint. As you can see here, the secondary endpoints included the mean percentage change in fasting triglycerides at Month 12, adjudicated pancreatitis events and the number of participants affected during the treatment period, as well as changes in apoC-III, remnant cholesterol, and non–HDL cholesterol. We also evaluated responder analysis—looking at the proportion of patients who achieved triglyceride levels below key clinical thresholds at 1 year. On the exploratory side, we assessed changes in total ApoB; ApoB-48, which is a reliable marker of intestinal chylomicrons; HDL cholesterol; and LDL cholesterol.
We'll be walking through the most clinically meaningful of these over the next several slides.
Let's look at who was enrolled in these trials. Patient demographics and baseline characteristics were generally similar across treatment groups in both CORE and CORE2, and I want to highlight a few points that are particularly relevant to clinical practice.
Sixty-three percent of patients had type 2 diabetes mellitus, and 99% were already on background triglyceride-lowering therapies, with approximately 74% on statins. Sixteen percent were also receiving a GLP-1, reflecting how commonly these agents are now part of the treatment landscape for this population. Fifteen percent had a history of acute pancreatitis within the prior 10 years. Despite all of this, mean fasting triglycerides at baseline were over 1100 mg/dL.
This is a population that I think reflects many of the patients you may see in your own practice—medically managed but still severely uncontrolled. And that's the context in which we should interpret the efficacy data that follow.
JEFF BACHMAN
Before you continue, Dr. Budoff, I'd like to let our audience know that you can submit your questions throughout this broadcast on the left side of the screen, and we will address them at the end of the presentation. Please continue, Dr. Budoff.
DR. MATTHEW BUDOFF
Now let's look at the primary endpoint results; this is really where the data speak for themselves. In CORE, TRYNGOLZA 80 mg demonstrated a placebo-corrected mean reduction of 72% in fasting triglycerides from baseline to Month 6, from a mean baseline of 1169. The 50 mg dose also showed a robust reduction of 63%.
These results were confirmed in CORE2, where the 80 mg dose achieved a 55% reduction and the 50 mg dose a 49% reduction, both from a mean baselines of approximately 1000 mg/dL. And remember, these results were achieved in patients who were largely already on background triglyceride-lowering therapies—statins, fibrates, omega-3s. So this is the additional benefit TRYNGOLZA delivered on top of existing management. We've just seen the primary endpoint results at Month 6.
Now let's look at how those reductions played out over time, starting with CORE. What you can see on this graph is that triglyceride reductions were rapid—evident within the first few weeks of treatment—and sustained throughout the full 12-month period. At Month 6, the 80 mg dose achieved approximately a 73% reduction from baseline and the 50 mg dose approximately 63%. What's particularly striking is the clear and consistent separation between both TRYNGOLZA arms and placebo across the entire treatment period. With once-monthly dosing, patients maintained this level of triglyceride control without any loss of effect over time.
Now let's look at whether CORE2 tells the same story. The short answer is: yes, it does. The CORE2 data tell a consistent story: rapid triglyceride reductions early in treatment, maintained over a full year. At Month 6, the 80 mg dose achieved approximately a 68% reduction from baseline and the 50 mg dose approximately 63%. And as you can see, the sustained separation between TRYNGOLZA and placebo holds across the entire treatment period, just as we saw in CORE.
So, across both pivotal trials, we're seeing the same pattern: rapid, robust, and durable triglyceride lowering with once-monthly TRYNGOLZA. The question then becomes—what does that mean for your patients clinically? And that's what we'll look at next.
One way to answer that is to look at how many patients crossed the thresholds that matter most in clinical practice. Across pooled CORE and CORE2 data at 1 year, the responder analysis here show that at 3 clinically relevant thresholds. Starting on the left, over 85% of patients receiving TRYNGOLZA achieved fasting triglyceride levels below 880 mg/dL, and that same pattern held at the 500 mg/dL threshold, with 86% of patients on either dose crossing that guideline-recommended target. Moving to the right, in an exploratory analysis, up to 54% of patients on the 80 mg dose achieved levels below 150 mg/dL, approaching the normal range.
As noted on the slide, these are exploratory analyses and should be interpreted with caution. As we discussed earlier, guidelines from the AHA, ACC, NLA, and Endocrine Society all identify fasting triglycerides below 500 mg/dL as a critical threshold for reducing acute pancreatitis risk. We've seen the triglyceride reductions and the proportion of patients crossing clinically meaningful thresholds. But ultimately, the question every clinician wants answered is: does this translate into fewer episodes of acute pancreatitis? And for the first time, we can say: yes, it does.
Looking at AP event reduction by dose, TRYNGOLZA 50 mg demonstrated up to 91% reduction in the adjudicated acute pancreatitis event rate compared with placebo, with a rate ratio of 0.09. At the 80 mg dose, there was a 76% reduction, with a rate ratio of 0.24. Both reductions are striking, and they reinforce that TRYNGOLZA is delivering meaningful clinical benefit at both doses across this population.
Let's look at the time-to-event data now. Now let's look at this same AP benefit through a different lens: the time to first event, in the pooled analysis across all 1061 patients. In this pooled analysis of CORE and CORE2, TRYNGOLZA demonstrated an 85% reduction in the adjudicated acute pancreatitis event rate compared with placebo, with a rate ratio of 0.15. And the time to first acute pancreatitis event was statistically significantly longer with TRYNGOLZA vs placebo, with a P value less than 0.0001. To put that in real numbers: in the placebo group, 17 patients experienced 22 adjudicated AP events over the treatment period. In the combined TRYNGOLZA group, just 5 patients experienced 7 events.
This makes TRYNGOLZA the first therapy indicated to reduce the risk of acute pancreatitis in adults with severe hypertriglyceridemia, a meaningful milestone for our patients. Number needed to treat, or NNT, is one of the most practical ways to think about treatment benefit. Put simply, NNT tells us how many patients would need to be treated with TRYNGOLZA for 1 year to prevent 1 episode of acute pancreatitis, compared with not using the therapy. The lower the number, the more effective the treatment.
Across the overall sHTG population—patients with triglycerides at or above 500 mg/dL—the NNT was 20. When we look at patients with triglycerides at or above 880 mg/dL, that drops to 9. And in the highest-risk patients, those with triglycerides at or above 880 and a prior history of acute pancreatitis, the NNT was just 4.
So the patients who need the most help are the ones who stand to benefit the most. I do want to note that the NNT data for all subgroups were exploratory analysis and should be interpreted with caution, but the trend across the spectrum is compelling and clinically meaningful.
JEFF BACHMAN
Thank you, Dr. Budoff. We've just seen some truly impressive results—robust triglyceride reductions, the majority of patients crossing critical thresholds, and a significant reduction in acute pancreatitis events.
Now I'd like to turn it over to Dr. Bajaj to walk us through the safety profile, dosing, and administration of TRYNGOLZA.
DR. ARCHNA BAJAJ
Thank you, Jeff. So now that we've reviewed the efficacy data, let's turn to the safety profile, because confidence in a therapy means understanding both its benefits and its risks. The safety of TRYNGOLZA was evaluated in 705 patients with severe hypertriglyceridemia across CORE and CORE2—there were 354 patients who received the 50 mg dose and 351 patients who received the 80 mg dose—along with 356 patients who received placebo. As you can see in the table, the adverse reactions occurring in 2% or more of patients treated with TRYNGOLZA—and more frequently than with placebo—were injection-site reactions and increased transaminases. Injection-site reactions occurred in 12% of patients at the 50 mg dose and 18% at 80 mg, compared with 2% on placebo. The majority of these were mild in severity, and none were severe. Increased transaminases were observed in 3% of patients at the 50 mg dose and 4% at 80 mg, compared with 1% on placebo.
Adverse reactions led to discontinuation of treatment in 5% of TRYNGOLZA-treated patients and 2% of placebo-treated patients, with injection-site reactions being the most common reason for discontinuation. So now let's talk about what dosing looks like in practice.
In patients with severe hypertriglyceridemia, the recommended dosage of TRYNGOLZA is 50 mg injected subcutaneously once monthly via a convenient autoinjector, along with a low-fat diet. From there, you assess triglycerides when clinically appropriate. The triglyceride-lowering effect of TRYNGOLZA may be measured within 3 months after initiation, so you have a clear early window to evaluate response. For patients who tolerate the 50 mg dosage and additional triglyceride reduction is clinically indicated, the dose may be increased to 80 mg once monthly. The once-monthly schedule and autoinjector delivery are designed to support individualized care for the patient in front of you.
TRYNGOLZA is self-administered via a convenient autoinjector, and the process is straightforward. Prior to initiation, patients and caregivers should be trained on proper preparation and administration, so please ensure your patients have received that training before they self-administer at home.
Now let me walk you through what that looks like. It's just 3 steps. First, remove the clear cap and place the orange needle shield at a 90-degree angle against the skin—making sure the viewing window is visible. Second, push the autoinjector firmly against the skin. Patients may or may not hear 2 clicks during this step, and that's completely normal—the procedure is not finished yet. Third, hold the autoinjector pressed against the skin for 10 seconds.
The key reminder for your patients is to not lift the autoinjector until the full 10 seconds have passed and the orange plunger rod has filled the entire viewing window. Then remove by lifting straight up and dispose of the autoinjector in a sharps container. An injection training video is also available at TRYNGOLZAHCP.com for additional support. One of the practical advantages of TRYNGOLZA is that it fits easily into your patients' lives, whether they're at home or on the go.
The autoinjector should be stored in the refrigerator between 36 and 46 °F in its original carton. But when removed from the refrigerator, it can be stored at room temperature—between 59 and 86 °F—in its original carton for up to 6 weeks. If not used within that 6-week window, it should be discarded.
This flexibility means that, if a patient is traveling or simply prefers not to inject at home, they have that option. It's a once-monthly dose with storage requirements that are designed to minimize disruption to their daily routine.
JEFF BACHMAN
Thank you, Dr. Bajaj. We've reviewed the robust efficacy data, the significant reduction in acute pancreatitis events, and the well-tolerated safety profile of TRYNGOLZA.
Now I'd like to remind our audience that you can submit your questions throughout this broadcast on the left side of the screen, and we will address them at the end of the presentation.
With that, I'd like to turn it over to Marianna Keegan, Associate Director of Patient Services at Ionis, to walk us through how to get your patients with sHTG started on TRYNGOLZA.
Marianna.
MARIANNA KEEGAN
Thank you, Jeff. I'm happy to be here today to walk you through how to get your patients with sHTG started on TRYNGOLZA—and the good news is, the process is straightforward.
There are 2 ways to prescribe. You can e-prescribe through your electronic health record system to an in-network pharmacy. Or you can fax a completed TRYNGOLZA Start Form to an in-network pharmacy, which is available for download from TRYNGOLZAHCP.com, or from the tear pad from your Ionis Specialist. To help support a smooth approval process, please include copies of the patient's insurance card, patient information such as age and diet, the documented diagnosis and ICD-10 code, and supporting clinical documentation, including fasting triglyceride levels, previous and current therapies, medical history with any prior pancreatitis events, and whether the patient has been prescribed TRYNGOLZA by or in consultation with a specialist. And please remember to encourage your patient to provide consent so they can access the full range of support from Ionis Every Step. So once you've submitted the prescription, where does it go?
TRYNGOLZA is available through a select network of specialty pharmacies—Accredo, BlinkRx, CVS Specialty, and Optum Specialty Pharmacy. It's important to confirm with the patient's insurance plan whether a specific pharmacy is required, as some plans do have preferences. BlinkRx is the digital pharmacy in our network—a streamlined platform designed to integrate into your office workflow.
The other in-network pharmacies—Accredo, CVS Specialty, and Optum Specialty Pharmacy—are specialty pharmacies that are each affiliated with specific payers or PBMs, and those details are shown here on the slide. All pharmacies in our network can accept all insurance plan types. One helpful tip for your patients: encourage them to save their pharmacy's phone number so they can easily recognize and answer calls related to setting up their TRYNGOLZA delivery. Let me take a minute to highlight what our in-network pharmacies specifically do to support your patients once a prescription has been submitted.
We just reviewed the pharmacies in our network, including BlinkRx as the digital option. Regardless of which pharmacy your patient uses, all pharmacies in our network will contact patients to coordinate delivery details and provide injection training for their first dose, ship prescriptions through free home delivery, help connect eligible patients with coverage and affordability programs, and initiate prior authorization requests through CoverMyMeds or fax and provide support with appeals. So regardless of which pharmacy your patient ends up with, they'll have a consistent level of support from day one.
I've mentioned Ionis Every Step a few times, so let me give you a fuller picture of what that support looks like. Ionis Every Step offers a broad range of support designed to help your patients at every step of their treatment journey, across 4 key areas: access support from our pharmacy partners to help you navigate prior authorizations and appeals; coverage and affordability programs to help eligible patients access TRYNGOLZA regardless of insurance type; ongoing support to help patients start and stay on treatment, including injection training and regular check-ins; and patient education and engagement through digital resources and additional training.
To enroll, patients can visit TRYNGOLZA.com/Enroll or by signing the TRYNGOLZA Start Form in your office.
You can call the Ionis Every Step TRYNGOLZA Support Center at 1-844-789-8744, select option 2, Monday to Friday, 8 AM to 8 PM Eastern, for more support.
Finally, let's talk about cost, because we know that can be a real concern for patients.
There are 3 coverage affordability programs available through Ionis Every Step. First, Copay Assistance—your commercially insured patients may pay as little as $0 per fill through the Ionis Every Step Copay Program. Second, our Quick Start Program—if insurance coverage is delayed, Ionis Every Step can work with the pharmacy to send the first dose quickly and at no cost. And third, the Patient Assistance Program—for patients whose insurance doesn't cover TRYNGOLZA or who don't have insurance, they may be eligible to receive it at no cost through a PAP application.
All programs are subject to terms, conditions, and limits.
The Copay Program is not available to individuals using state or federal government-funded healthcare programs such as Medicare or Medicaid.
So now that you know how to prescribe and where to send the prescription, let me share a few important takeaways about the access process. First, encourage your patients to visit TRYNGOLZA.com/Enroll to sign up for Ionis Every Step. Second, after the pharmacy initiates the PA request through CoverMyMeds or fax, ensure you complete the PA form and confirm your patient meets all the plan-specific PA criteria. Third, if the plan denies your coverage request, initiate an appeal in a timely manner to ask them to reconsider their decision.
Finally, helpful resources for the insurance approval process are available for download at TRYNGOLZAHCP.com.
JEFF BACHMAN
Thank you, Marianna. And thank you to our audience for your interest in TRYNGOLZA.
As a reminder, you may download the TRYNGOLZA Prescribing Information by going to the Important Safety Information section on the right side of your screen in the chat section. If you'd like to learn more or have a conversation about TRYNGOLZA for your patients, we encourage you to request a visit from an Ionis Specialist by scanning the QR code on your screen.
OK, we're ready to start answering your questions that you have come in during the presentation.
Our first question is about GLP-1s: 'GLP-1s have been great for A1C and weight in my patients. When those improve, what are you seeing happen with triglycerides in patients who start at or above 500 mg/dL?'
Dr. Bajaj, I'll turn that one over to you.
DR. ARCHNA BAJAJ
Thanks, Jeff. That's a great question and one we hear a lot. GLP-1s have shown real effectiveness in glycemic and weight control, but patients with triglycerides at or above 500 may still need additional therapy even when their A1C and weight improve. Studies have shown GLP-1s have some reduction in triglyceride levels, but they are not indicated for lowering triglycerides for patients. It is worth noting that 16% of patients in the CORE and CORE2 studies were already on GLP-1s, and TRYNGOLZA 80 mg still demonstrated up to a 72% placebo-corrected reduction in triglycerides at Month 6 in addition to the background lipid-lowering therapy. So, when you have a patient doing well metabolically on a GLP-1 but their triglycerides remain elevated, TRYNGOLZA can be a meaningful, additional option.
JEFF BACHMAN
Thank you, Dr. Bajaj. Now we have a question about LDL: 'How should I be managing LDL-C alongside triglycerides in patients on TRYNGOLZA?'
Dr. Budoff, could you speak to that please?
DR. MATTHEW BUDOFF
Sure. So LDL-C is certainly an important consideration. With apoC-III inhibition, what we're seeing is a shift in lipid metabolism as triglyceride-rich particles are cleared, and that can lead to some increases in LDL-C. At the same time, in patients with severe hypertriglyceridemia, TRYNGOLZA produces meaningful reductions in other atherogenic markers that are particularly relevant in this population—specifically, non–HDL-C and apoB. Current guidelines actually note that non–HDL-C and apoB may be more informative than LDL-C alone in patients with elevated triglycerides.
So the LDL-C changes we see with TRYNGOLZA should be considered in that broader context of overall lipid remodeling. And for patients already on a statin therapy, which was roughly 74% of the trial population, that LDL-C piece is often already being addressed.
JEFF BACHMAN
Thank you, Dr. Budoff. Let's shift now to a question regarding access: ‘Are the 2 strengths of TRYNGOLZA priced differently?’ Marianna, I think you can respond to this one.
MARIANNA KEEGAN
Yeah, sure, great question. Yes, TRYNGOLZA is priced the same for both 50 mg and 80 mg.
Ionis actually reduced the price of TRYNGOLZA on April 1, 2026, to better address the needs of the patient population. And for your eligible commercially insured patients, the out-of-pocket cost can be even lower: through the Ionis Every Step Copay Program, eligible patients may pay as little as $0 per fill. So while the list price is one data point, the real-world cost for many of your patients will look very different. And if you have patients with coverage challenges, we have additional support programs in place.
JEFF BACHMAN
Thank you very much. Our next question that just came in is about clinical trials. Our audience wants to know: ‘Why were 2 separate trials, CORE and CORE2, conducted and why is some of the data presented pooled?’ Dr. Bajaj, do you think you can address that?
DR. ARCHNA BAJAJ
Sure. Thanks, Jeff. So in order to meet the FDA requirements, CORE and CORE2 were identically designed as parallel studies. The goal was to confirm the findings and build a more robust safety and efficacy dataset in the severe hypertriglyceridemia population. So each study independently demonstrated statistically significant triglyceride reductions for certain endpoints, such as acute pancreatitis events and the proportion of patients achieving specific triglyceride thresholds. Pooled analyses were used to provide a more complete picture across a broader patient population.
JEFF BACHMAN
Thank you for that clarity, Dr. Bajaj. Our next question is: ‘What should I be mindful of around hepatic fat fraction?’ Dr. Budoff, would you like to take that one on?
DR. MATTHEW BUDOFF
Yeah, I can answer that. So hepatic fat fraction was measured in a substudy of CORE and CORE2 using MRI. Patients at baseline had elevated hepatic fat fraction, which is typical for this patient population. These increases were seen but not associated with the increases in liver enzymes, and the clinical relevance of this is yet to be determined.
JEFF BACHMAN
Some great context to put on that, Dr. Budoff. Thank you. Next is a question about genetic testing: ‘Do I need to order a genetic test and submit results every time I prescribe TRYNGOLZA for an sHTG patient?’ Mariana, do you think you can provide some clarity on that?
MARIANNA KEEGAN
Yes, good question. This is a question we hear often, and it’s an important one to clarify. A genetic test is not required for the diagnosis of sHTG. If a payer is asking for genetic test results, that may be a carryover from their coverage credit criteria for FCS, which does have a genetic component. For sHTG, proof of diagnosis is typically established by documenting fasting triglyceride levels at or above 500. A payer may ask for additional documentation to support medical necessity, things like ICD-10 code, prior therapy history, and acute pancreatitis history. But a genetic test should not be a standard requirement. If you do encounter that, our pharmacy partners and the Ionis Every Step team can help you navigate it.
JEFF BACHMAN
Thank you, Mariana. Next question is: ‘Does TRYNGOLZA reduce ASCVD risk and why didn't the trial study ASCVD outcomes?’ Dr. Budoff, I'll punt that to you.
DR. MATTHEW BUDOFF
Yeah, that's an important question and one that comes up frequently in practice. TRYNGOLZA is indicated to reduce triglycerides and the risk of acute pancreatitis in adults with severe hypertriglyceridemia. The CORE and CORE2 trials were specifically designed to evaluate triglyceride lowering as the primary endpoint, with acute pancreatitis events as a prespecified secondary endpoint. These trials weren’t powered for ASCVD outcomes.
So for patients where ASCVD risk reduction is the primary clinical concern, evidence-based ASCVD-lowering therapies remain the standard of care. That said, TRYNGOLZA does produce reductions in other atherogenic lipid parameters, specifically non–HDL cholesterol and apolipoprotein B, through its impact on ASCVD reduction is unconfirmed.
JEFF BACHMAN
Thank you very much, Dr. Budoff. Would you recommend stopping other triglyceride-lowering agents altogether once achieving normal triglycerides with TRYNGOLZA? Dr. Bajaj, walk me through your thought process on that.
DR. ARCHNA BAJAJ
Yeah, so I think this is where you have to take it on a patient-by-patient basis and see how they respond to TRYNGOLZA first. We know that in many patients with severe hypertriglyceridemia that the traditional triglyceride-lowering therapies are clearly not producing enough of a response. And that’s why we’re using TRYNGOLZA in these patients. But I wouldn’t be so quick to pull those medications off. I would first track triglyceride levels and see how they respond to TRYNGOLZA, and then decide based on the response and by tracking those triglyceride levels if you can back off on some of the other medications. Although, as we just talked about ASCVD, I do want to point out you do want to continue the statin and other ASCVD risk reduction medications in these patients, and TRYNGOLZA is certainly not a replacement for those.
JEFF BACHMAN
Fantastic. Important question, very complex patients. So yeah, thank you very much for that clarity. Now a question about coverage: ‘How do I know if prior authorization is required for TRYNGOLZA and what information do I need to submit to get my patient covered?’ Mariana, can you answer this?
MARIANNA KEEGAN
Yes, thanks Jeff. So most insurance plans will require a prior authorization to approve TRYNGOLZA coverage. The good news is our in-network pharmacies—Accredo, BlinkRx, CVS Specialty, and Optum Specialty Pharmacy—can initiate that PA request through CoverMyMeds or fax so you’re not starting from scratch. In terms of what to have ready, common PA criteria for sHTG typically include patient age and dietary information; the ICD-10 diagnosis code; fasting triglyceride levels at or above 500; medical history including any prior acute pancreatitis events; response to previous or current lipid-lowering therapies; and documentation that TRYNGOLZA was prescribed by or in consultation with a specialist.
Having that clinical documentation prepared up front can really help streamline that approval process. And if a denial does come back, our pharmacy partners can help support the appeals process.
JEFF BACHMAN
Thank you very much for that. Let’s see, our next question is a question about treatment priorities. Specifically: ‘Why should high triglycerides be a treatment priority for my patients?’ Dr. Budoff, I'm going to let you take that one.
DR. MATTHEW BUDOFF
Absolutely. Well, severe hypertriglyceridemia, defined again as triglycerides at or above 500, puts patients at risk of potentially life-threatening acute pancreatitis and is a risk-enhancing factor for ASCVD. In fact, patients with severe hypertriglyceridemia have more than 5 times the risk of acute pancreatitis compared with those with normal triglyceride levels.
And unlike the management conditions, such as type 2 diabetes or hypercholesterolemia, which tend to focus on long-term outcomes, severe hypertriglyceridemia composed an acute and immediate risk of pancreatitis at very high triglyceride levels. So for patients sitting at or above 500 mg/dL, the urgency to act is real.
JEFF BACHMAN
Thank you so much for that, Dr. Budoff. Next question: ‘Can you please comment on severity of acute pancreatitis episodes in placebo vs treatment?’ Dr. Bajaj, would you like to take that one on?
DR. ARCHNA BAJAJ
Yeah. So, I mean, we know from the studies that there were obviously a greater number of acute pancreatitis episodes on placebo compared to those on treatment. I don’t think we necessarily have the information for the specific acute pancreatitis events themselves, but we know an episode of acute pancreatitis is severe. So these are usually,—obviously, they’ve been adjudicated for the trial—but acute pancreatitis in patients with sHTG can frequently lead to hospitalizations that can be prolonged, sometimes requiring ICU stays. It can be very difficult to manage and treat.
I also like to add patients, we talk about acute pancreatitis as an endpoint for these patients, but sometimes these patients are not necessarily diagnosed with acute pancreatitis because they don’t show up to the hospital in time. Sometimes they’ve been at home for a couple of days with severe abdominal pain, nausea, vomiting, and maybe have stopped eating for a couple days before they show up to the hospital. And so there can be a little bit of misdiagnosis there and a missed opportunity to diagnose with acute pancreatitis.
JEFF BACHMAN
Great answer. I think we can all agree any pancreatitis is bad pancreatitis.
DR. ARCHNA BAJAJ
Exactly.
JEFF BACHMAN
And that, you know, once you have one, you’re at a higher risk of a second one.
DR. ARCHNA BAJAJ
Yeah.
JEFF BACHMAN
So next question is: ‘I work as a medical director in a pancreas clinic.’ Sorry about that. ‘What are the doses of standard triglyceride-lowering medication used in these studies?’ Dr. Budoff, would you like to take this?
DR. MATTHEW BUDOFF
Yeah. So, I mean, when we think about the background therapy that were engaged in these trials, a lot of it were omega-3s. We try to get patients up to 4 grams per day of omega-3s. Typically, statins were used in 74% of patients. I personally like to use atorvastatin in patients with high triglycerides because it has a tendency to have a little better triglyceride-lowering effect than rosuvastatin or some of the other statins. But of course, there was a mix in the clinical trials. And then, of course, fenofibrates were used quite a bit. They are probably one of our more potent background therapies for triglyceride reduction. And then, of course, layered on top of that was either TRYNGOLZA 50 mg or 80 mg or placebo, and that’s how we did CORE and CORE2.
JEFF BACHMAN
Awesome. Dr. Bajaj, I'm going to give you our last question of the day. Now, TRYNGOLZA, as you know, is indicated for two things, FCS and severe hypertriglyceridemia. Our most recent indication is for adults with severe hypertriglyceridemia to reduce triglycerides and the risk of acute pancreatitis. So as a clinician, what excites you most about this indication for your patients?
DR. ARCHNA BAJAJ
I think it’s really exciting to see that both pieces of information were included in that indication for sHTG, right? Not just the triglyceride lowering, but also for acute pancreatitis reduction, right? So speaking directly to the outcome of interest and outcome of concern in these patients, which is, it’s really exciting to see that included.
JEFF BACHMAN
That’s awesome. Dr. Budoff, anything to add?
DR. MATTHEW BUDOFF
I would just say that the number needed to treat is quite impressive, especially for those patients with severe hypertriglyceridemia and prior acute pancreatitis—number needed to treat of 4 is dramatic. I mean, we all know how horrific pancreatitis can be. And if you only have to treat 4 or 9 or even 20 patients for a year to prevent an episode, that's a very effective therapy.
JEFF BACHMAN
Yeah. Efficacy and benefits we have not seen in this disease state so far, so…
Well, that’s all the time we have for today. I’d like to thank you all for being here with me, with us. You really had some great information to share on TRYNGOLZA. Thank you to everyone tuning in from across the country. We hope you have thoroughly enjoyed this broadcast. And from Ionis Pharmaceuticals, I am Jeff Bachman. Take care.
FCS=familial chylomicronemia syndrome; sHTG=severe hypertriglyceridemia.
Genevie: Running from Adversity
Genevie: Running from Adversity
Genevie was told there would never be a medication for FCS, which left her worried for the future. That was until her doctor told her about a promising clinical trial in adults with FCS.
Genevie, living with FCS and a paid speaker for Ionis.
I've always had this tendency to run away from my problems.
FCS has been such an unknown for me. I was told there was never going to be a medication that was going to work for me. I just didn't want to face the scariness of the future.
I was diagnosed with FCS when I was a baby. I had a real limited but safe diet. We certainly weren't experimenting eaters. My mom found out angel food cake was fat-free, so that's the one I had for every occasion.
When I was in middle school my friends were saying well just have a french fry. And trying it for the first time it's like wow that tastes really good, but I would get a good stomach ache.
The first time that I really suffered is when I was pregnant with my daughter and I got pancreatitis attacks. I was hospitalized multiple times. I was in a lot of pain. That was a really, really difficult time. But I thought, I've got a daughter now. And I was resolved to double down on all my efforts to be as healthy as I can for somebody else.
As the years progressed, no matter how hard I tried, my triglyceride levels would just rise higher and higher. It was frustrating. I would do all that I could and there was this thing that was happening that was out of my control. The thing that gets me the most is, I really, really want to show up for people. But I wasn't myself anymore. I stopped making plans. I stopped being reliable. I just kind of wanted to run away.
Fast forward, my doctor told me that there was this promising trial. I never even had it in my head that that was a possibility. In the trial, they put me on TRYNGOLZA, which is a prescription medicine for adults with FCS that you take along with your diet to lower your triglycerides. In the clinical trial, for people taking TRYNGOLZA, their triglycerides were lowered by 43%.
My doctor talked to me about the potential side effects of taking TRYNGOLZA. While I didn't experience any side effects, others may be different. They should talk to their doctor about side effects that may occur with treatment.
I'm thrilled to be able to show up for my people. My family and my friends have all been on this journey with me and they've been so incredibly supportive. It just feels really powerful that I can reliably give back.
I just give myself an injection once a month and now that I have a lower triglyceride level, I don't worry about getting pancreatitis. I feel like I'm just really running confidently towards my future.
With TRYNGOLZA, I can be in control.
FCS=familial chylomicronemia syndrome; sHTG=severe hypertriglyceridemia.
Aaron: A New Lens on Life
Aaron: A New Lens on Life
Aaron shares how his FCS diagnosis gave him a new perspective on life and how TRYNGOLZA is helping him manage his disorder.
Aaron, living with FCS and a paid speaker for Ionis.
Katy: I came home one day and he's doing chores. I literally said, who is this person and what have you done with my husband? That was the moment for me when I realized that something was very different.
Aaron: Growing up, I kind of knew something was wrong. If you can imagine waking up with flu symptoms, but it's every day on top of every day, it's exhausting. We went to a bunch of different doctors. I went to specialists and it never got better. I'd had pancreatitis over 34 times, and the last time I was having some major body shutdown. And it really scared me. I'd seen 14 doctors by this time, and I was really, really close to just giving up.
Theron being born with autism put in really sharp focus what I wanted to do and who I wanted to be—and that was the best dad and husband possible. But I just didn't have the energy to do it. Katy looked at me straight in the eyes and said, please see one more doctor. He was a lipid expert and he came in and said, I want to look at a few things. Let me see your palms. Let me see behind your ears. And he looked into my eyes, do you ever get these red bumps? And he said, you know, your body doesn't process fat and that's what's causing the stomach problems, the body ache, the fatigue. And he goes, congratulations, you have FCS. It was like my life coming back to me, but through the lens of this diagnosis. I was told, probably not in our lifetime is there going to be a drug for this.
Eventually, TRYNGOLZA became available. TRYNGOLZA is a prescription medicine for adults with FCS that you take along with your diet to lower your triglycerides.
I was worried I wouldn't be able to go on TRYNGOLZA because my genetic test wasn't positive, but my doctor told me that it didn't matter because I was clinically diagnosed. After 3 months of treatment, my doctor rechecked my triglycerides and they had lowered by more than 50%. I pinched myself. I think Katy and I both kind of were like, I think they need to recheck them. And I feel better knowing that I might have fewer pancreatitis events. In the clinical trial, people taking TRYNGOLZA saw their triglycerides lowered by 43% after 6 months. They also had fewer attacks of acute pancreatitis. My doctor talked to me about the potential side effects of taking TRYNGOLZA. While I didn't experience any side effects, others may be different. They should talk to their doctor about side effects that may occur with treatment.
I take TRYNGOLZA once a month. I can keep it at room temperature for up to 6 weeks. I'm looking forward to doing a bunch of travel, going to concerts and wrestling events and all kinds of stupid things. Especially with you. It's one less thing on my plate. So my plate has opened up to do other things…being a better husband, being a better dad, being a better friend to people. I've kind of prioritized relationships over almost anything else. Man, nothing makes life more crystal clear than having a kid with special needs. I want to make up for 48 lost years, and I want to live my best years with Katy moving forward.
Katy: He's pretty special.
Aaron: TRYNGOLZA worked for me. Ask your doctor about FCS and if TRYNGOLZA might be right for you.
FCS=familial chylomicronemia syndrome; sHTG=severe hypertriglyceridemia.
Roxanne: Driven by Hope
Roxanne: Driven by Hope
Roxanne shares her journey from growing up in a family familiar with FCS to learning how to manage it on her own. Discovering TRYNGOLZA not only helped her manage her condition but gave her hope for the future.
Roxanne, living with FCS and a paid speaker for Ionis.
This is a picture of me and my dad. I call him Papi, my hero.
This picture actually just reminds me of how much he was there for us, making sure that we were always okay.
FCS impacted my family a lot. They tested me when I was born because my sister, Emily, already had it. And I came out with the same condition. You go to doctors, and they basically tell you we don't have answers. There's no medication out there. We weren't the normal, typical kids where we can have birthday cakes, or we can have specific foods. But my dad did everything in his power to try to have somewhat a normal life.
So, fast food. He would get us the burger with no meat, no cheese, no mayo, and he would create it in the car. The ketchup, the turkey, the fat-free cheese from home.
He did that for me.
My dad used to say, "No hagas trampa." But in English it would be, "Don't cheat off your diet." It was like a constant reminder.
But when I moved away from home, it was hard. Going out, even if you ask, like, "Try not to, like, put oil or butter," you'll still get the plate of that food. So, I would just eat it. And it ended up getting me sick.
Actually, there was one month where I had 3 pancreatitis, back to back. And that was, that was awful. The pain is so severe. Then I would go to the hospital and they would ask me like, "How did you drive here with the pain?" And I'm like, "I just did it. You know, I just, I drove, there's no other option." So, I would drive myself. That was hard.
I decided to move back to Florida to be near family again. I felt like there has to be more answers out there. There has to be doctors. And my dad's like, "You need to look for a lipid specialist."
The office told me about a trial for adults that have FCS. And I was excited. I was like, what trial, what clinical, you know, what's going on? I didn't, I never heard of this.
The trial was for TRYNGOLZA, a prescription medicine for adults with FCS that you take along with your diet to lower your triglycerides.
In the clinical trial, people taking TRYNGOLZA saw their triglycerides lowered by 43% after 6 months. They also had fewer attacks of acute pancreatitis.
I take TRYNGOLZA with an autoinjector once a month. I get the prescriptions sent to the house, so it's super easy.
Ionis Every Step has been super helpful in answering any questions that I may have.
My doctor talked to me about the potential side effects of taking TRYNGOLZA. I've had a little soreness around the injection, but that's it. Everyone's experience may be different. They should talk to their doctor about side effects that may occur with treatment.
Since taking TRYNGOLZA, my triglycerides have lowered more than half.
I have a lot of hope now. I don't get sick as often. I'm not missing as much work. I'm looking forward to traveling, have a family, and settle down.
My dad, I would be lost without him. He was always there for me, but now I can be there for him. So, it made it possible for me. Thank you.
With TRYNGOLZA, I finally feel in control. Ask your doctor about FCS and if TRYNGOLZA could be right for you.
FCS=familial chylomicronemia syndrome; sHTG=severe hypertriglyceridemia.
Downloadable Resources
Resources to help you and your office support your patients
Getting Started Guide
Use this guide to understand how to prescribe TRYNGOLZA, get patients started with Ionis Every Step, and become familiar with available support.
Download PDF
Getting Started Guide
Navigating Coverage for TRYNGOLZA
This resource can get you familiar with the exceptions, prior authorization, and appeals processes for TRYNGOLZA.
Download PDF
Navigating Coverage for TRYNGOLZA
sHTG Sample Letter of Medical Necessity
A letter template stating why TRYNGOLZA is medically necessary for your patient.
Download PDF
sHTG Sample Letter of Medical Necessity
sHTG Sample Letter of Appeal
A letter template requesting that the insurance plan overturn a coverage denial for TRYNGOLZA.
Download PDF
sHTG Sample Letter of Appeal
TRYNGOLZA sHTG Brochure
A comprehensive overview of the CORE and CORE2 trials, including the safety and efficacy of TRYNGOLZA.
Download PDF
TRYNGOLZA sHTG Brochure
TRYNGOLZA FAQs
Evidence-based answers to common clinical questions about TRYNGOLZA, covering mechanism of action, efficacy, safety, dosing, and patient access.
Download PDF
TRYNGOLZA FAQs
TRYNGOLZA Patient Profiles
Hypothetical patient profiles that show how TRYNGOLZA may fit into existing treatment plans and help lower triglycerides in adult patients with sHTG.
Download PDF
TRYNGOLZA Patient Profiles
TRYNGOLZA Dosing Card
A quick-reference guide to recommended dosing for TRYNGOLZA in adults with sHTG and FCS, plus how to prescribe and where to send prescriptions.
Download PDF
TRYNGOLZA Dosing Card
Resources for your patients
Ionis Every Step™ Brochure
Overview for your patients about the support offered by Ionis Every Step.
Download PDF
Ionis Every Step™ Brochure
Getting Started with TRYNGOLZA
Step-by-step guide for your patients after being prescribed TRYNGOLZA.
Download PDF
Getting Started with TRYNGOLZA
TRYNGOLZA Fact Sheet
Overview of TRYNGOLZA, including what it is, how it works, possible side effects, and how to get started on treatment.
Download PDF
TRYNGOLZA Fact Sheet