Adding to that, we know that there's many of our patients who end up getting the delayed diagnosis before they get to us and get the diagnosis. Can you comment on what the problems are when patients with lupus have a delayed diagnosis in terms of long-term outcomes?

: There are a couple of things that lend itself to the delayed diagnosis. One is that there's not one clear laboratory or radiographic finding that definitively makes the diagnosis. Providers who are sending patients to rheumatologists for the diagnosis of lupus or potential lupus are not as comfortable and may not have their antenna up as much in thinking about that as a possible diagnosis. Sometimes there's a delay because patients are seeing other sub-specialists but not rheumatologists because there's a whole picture that's there. And the focus may be on one organ system manifestation instead of looking at the systemic manifestations in total to help think about is there an overarching diagnosis that might explain multiple clinical manifestations. That part of it is that it's a hard diagnosis to make, that the referrers may not always have it at the forefront of their minds. And then also sometimes people don't check that sort of ANA, they just may say, they're just fatigued. Some of the vagueness of the symptoms that may go along with lupus may lend itself to making it harder to make the diagnosis as well. And then once patients get to the rheumatologist it's not always a clear-cut diagnosis either. Then we have to go through our process of doing both the science part of the diagnosis but the art part of the diagnosed with history exam to put it all together to come up with that diagnosis.

I also think part of the problem with the delay in diagnosis is that lot of us have not really realized until we looked at cohort studies. Is that 10 to 30% of patients within one year of diagnosis already have irreversible damage. And we don't think about that. And if we think about then at 5 years somewhere between 30 to 50% of patients based on cohort data also have permanent irreversible organ damage. And that's with the average age of patients entering the cohort studies of 30 and entering with lower 70% on moderately high doses of prednisone, 70 and a half mg or higher. When you think about that, maybe they were seeing someone else, was maybe on a lot of steroids, and finally gets into treatment that we need to be doing besides steroids. They may already have some irreversible damage.

: It speaks to the urgency of the lupus diagnosis because that's where it all begins. That's how we get to the place where we can help patients and begin treatments to try to prevent the irreversible damage that you brought up. And sometimes there's a lack of that urgency or lack of an understanding of how quickly damage can occur. And like you said, in the first 5 years of disease if they haven't had a diagnosis, we could have potentially prevented some organ damage, even in that short period of time. And often times when patients do have particularly lupus nephritis, they're going to have the lupus nephritis start within the first 5 years of their lupus diagnosis. And we have to have our antenna up or our clinical suspicion very high for patients who come in for evaluation and to be assertive and aggressive in evaluating patients. And that is somewhat on the rheumatologist as well in that we must see the urgency in it. It should be just as urgent in a new lupus evaluation as it is to see someone with rheumatoid arthritis with multiple joints that are inflamed and impacting their life such that they can't do their activities of daily living. We must see it as just as urgent. And when patients come in say for an example, with rheumatoid arthritis it's very severe. You see them present and say, I'm feeling horrible, I'm feeling terrible. They don't look good, but a lupus patient may not in your clinical examination look as bad but could have nearly as much of a risk of permanent damage as that patient who has hot swollen joints that are, that has rheumatoid arthritis. We have to have a greater understanding of the urgency of diagnosing these patients and being assertive and slash aggressive with treatment so that we prevent the damage. Because once that scope occurred, we cannot reverse it.

 I was just thinking the same thing is we take our RA patients but we don't always take our lupus patients seriously. So great point.

Drs. Anne E. Winkler and Kristi V. Mizelle discuss the negative long term disease outcomes noted in patients who experience a delay in their lupus diagnosis. 

Impact of Diagnosis Delay on Long Term Lupus Outcomes

Anne E. Winkler, MD, PhD, MACP

Mukkamala discussed other priorities the AMA’s Substance Use and Pain Care Task Force is tackling with OUD.

The Shifting Field of Care of Opioid Use Disorder, with Bobby Mukkamala, MD

Last month, the FDA approved the prior supplement submission for Indivior’s 

buprenorphine extended-release injection 

(SUBLOCADE) to treat opioid use disorder (OUD) almost 1 year after its initial approval for treating treat moderate to severe OUD.

The new labeling expands the approved subcutaneous injection site of the abdomen to the thigh, buttock, and back of the upper arm for induction and maintenance.

In another shift, the current 7-day minimum on transmucosal buprenorphine will be amended to a single dose of transmucosal buprenorphine with a 1-hour observation period to confirm tolerability and patients will be allowed to receive a second 300 mg dose 1 week after the initial 300 mg injections.

spoke with otolaryngologist Bobby Mukkamala, MD, who is serving as president-elect of the American Medical Association (AMA) and chair of the AMA’s Substance Use and Pain Care Task Force, to learn more about the labeling change. He also shared other priorities in the shifting world of care for OUD.

“We would really encourage folks that are trying to take care of their communities and trying to take care of their patients to look and see what comes from our task force. It's a very useful, but one of many sources of knowledge so we can take better care of our communities and our patients,” Mukkamala said.

Conversations about supporting treatment for OUD are especially pertinent as Congress just a couple of days ago failed to address payment cuts imposed in the CY25 Medicare Physician Fee Schedule (MPFS), comprising care for vulnerable patient populations.

Mukkamala has no relevant disclosures to report.

Indivior Announces FDA Approval of Label Changes for SUBLOCADE® (buprenorphine extended-release) Injection. PR Newswire. February 24, 2025. https://www.prnewswire.com/news-releases/indivior-announces-fda-approval-of-label-changes-for-sublocade-buprenorphine-extended-release-injection-302382920.html. Accessed March 17, 2025.

Braeburn's BRIXADI™ (buprenorphine) Extended-Release Subcutaneous Injection (CIII) Receives FDA Approval for Moderate to Severe Opioid Use Disorder. PR Newswire. May 23, 2023. 

https://www.prnewswire.com/news-releases/braeburns-brixadi-buprenorphine-extended-release-subcutaneous-injection-ciii-receives-fda-approval-for-moderate-to-severe-opioid-use-disorder-301832521.html 

Congress Fails to Address Medicare Fee Schedule in Continuing Resolution. News release. American Physical Therapy Association. March 17, 2025. https://www.apta.org/news/2025/03/17/congress-fails-to-address-fee-schedule

Bobby Mukkamala, MD

Closing Remarks

Video content above is prompted by the following:

Can each of you provide closing remarks on how you treat your own patients to achieve remission?

Panelists discuss how their approaches to treating patients with rheumatoid arthritis focus on personalized strategies that prioritize achieving remission while carefully monitoring safety and efficacy outcomes.

Catherine Bakewell, MD

Key Learnings From the CATALYST Study on Hypercortisolism Prevalence

What are some of the challenges of identifying and diagnosing hypercortisolism?

The diagnosis of hypercortisolism is often delayed by several years. Why do you think that the diagnosis of hypercortisolism is often delayed or missed?

Hypercortisolism presents with a wide spectrum of clinical signs and symptoms. How does this affect clinicians’ ability to diagnose this condition?

The prevalence of hypercortisolism in certain at-risk patients is higher than previously thought. Can you discuss some of the recent data on hypercortisolism prevalence?

Prevalence of hypercortisolism in patients with difficult-to-control diabetes was 24%.

Prevalence of hypercortisolism in patients with difficult-to-control diabetes and difficult-to-control hypertension was 40%.

Patients with cardiovascular disease had a higher prevalence of hypercortisolism than those without (33.3% vs 20.9%).

Experts discuss the challenges of identifying and diagnosing hypercortisolism, the reasons behind its often-delayed diagnosis, the impact of a wide spectrum of clinical signs and symptoms on clinicians’ ability to diagnose the condition, and recent data on the prevalence of hypercortisolism in at-risk populations, including those with difficult-to-control diabetes, hypertension, and cardiovascular disease.

Davida Kruger, MSN, APN-C, BC-ADM 

Henry A. Nasrallah, MD. This article was developed and sponsored in collaboration with Neurocrine Biosciences, Inc.

The last question that we should address is the following: How would you introduce the patient to the fact that they will be or could be taking a biosimilar? What type of discussion would you have with the patient?

 This is a great question, where we’ll have to come up with our ways of explaining a very complicated aspect to our patients in ways that are understandable and reassuring. I’ll be telling most of my patients that this is good news, that these products have been carefully tested and studied at a number of levels and their experience should basically be the same. The benefit of having these biosimilars available is that it should make the therapies more accessible to our patients because they’re so expensive.

It’s going to be challenging, though. With my own practice, we have so many options for psoriasis. I’ll often say, “We have to try biologic A,” and we agree on it. I send them the information on it and then the insurance company says, “No, it’s going to be biologic B.” Even when we agree it’s biologic A, they may be told it’s going to be biologic B and say, “But it isn’t the drug you told me.” It’s now this other thing with a different name they don’t recognize, and we have to go back and have that conversation. That’s likely to be a big source of anxiety for our patients. We know a lot of our patients are reluctant to go on biologics sometimes, so adding these additional complexities and barriers for our patients may cause harm in individuals who tend to be pretty reluctant or anxious about this process from the get-go.

It’s all about patient education, though. When I see a new patient with rheumatoid arthritis—it’s the easiest one—I can persuade 99% of patients to go on methotrexate for the first drug. It isn’t a big problem. Then when 70% of patients respond to methotrexate, and then we have all those other molecules available. I talk about all those molecules. You give the patient informed consent, which has gotten much more complex as time has gone on because there are many more drugs. Then I ask, “Which would you prefer first? Which would you prefer second?” In the end, I say, “Your insurance company is going to make the decision. I want to know what you’d do if the insurance company doesn’t pick your first choice.” They pick one. “What do we do if that one doesn’t work?” That’s how I handle that, and it usually works.

In speaking to a patient, you have to simplify, just as you did, that the biosimilars have shown that they’re very similar to the reference product and that almost all patients who will respond to a reference product will respond to a biosimilar. It’s that switch patient, the ones who do well, where I think there’s a little more angst. Even there, I’d say, “The literature shows that 95% of patients do well,” but I like to have the availability to switch back to the bio-original if I can.

 The critical thing for clinicians to know is the importance of instilling confidence, because the data give us the confidence that’s well earned. In my practice, when I choose mechanism of action A and then the insurance company says, “No, you have to use B,” as long as I think B is a very good option, I’ll go back to the patient and say, “The company wants you to use this biologic. It’s an outstanding option for you. It’s very likely to clear your skin, and I recommend it.” Almost all my patients in those settings are OK with that situation and are willing to make that switch. It should be an even easier thing to instill confidence when we’re staying within the same mechanism of action and a similar biologic. They should expect the same experience, whether we call it adalimumab or whatever the biosimilar name is going to be.

Thank you. To summarize, the prescribing physician has to have confidence in the biosimilar, and then the physician, the nurse practitioner, and all the ancillary personnel have to have that same degree of confidence. I can imagine a physician telling the patient, “There’s almost no difference between these drugs,” and then the infusion nurse or some laboratory person says, “I wouldn’t take a biosimilar.” Who’s the patient going to listen to? A well-educated physician or the laboratory tech? It’s going to be the lab tech.

 Or it might be social media. It might be what someone googles online or reads in a chatroom, where someone says, “I was doing OK on this, and I went on that, and now I’m having a problem.” As clinicians, we have to expect that there will be some uncertainty, and we’ll have to be there for our patients, empathize with them, and help them make the best decisions possible and move forward in a way that’s of best interest to their health.

presentation. We hope you found this presentation to be rich and informative.

Drs Roy M. Fleischmann and Joel M. Gelfand discuss addressing patient concerns to biosimilars and provide take-home messages to clinicians utilizing biosimilars.

Addressing Patients' Concerns With Biosimilars

Joel M. Gelfand, MD, MSCE, FAAD

Greiwe discussed findings from the phase 3 ADORING 1 and 2 trials in people as young as 2 years of age.

Confirming Tapinarof’s Efficacy and Safety Across Atopic Dermatitis Severities, with Justin Greiwe, MD

Tapinarof was well-tolerated and demonstrated consistent efficacy across body regions in people as young as 2 years old with atopic dermatitis, according to data from the phase 3 ADORING 1 and 2 trials presented by Justin Greiwe, MD, Bernstein Allergy Group, and Clinical Assistant Professor of Medicine, Division of Immunology, Allergy, and Rheumatology, University of Cincinnati, at the 2025 American Academy of Allergy, Asthma, and Immunology (AAAAI)/World Allergy Organization (WAO) Joint Congress, February 28-March 3, in San Diego, California.

Greiwe and colleagues found that in ADORING 1, least squares (LS) mean overall EASI score improvements were –8.4 vs –4.1 (P <.0001) at week 8 and in ADORING 2, LS mean overall EASI score improvements were –10.1 vs –5.2 (P <.0001). By body region, LS mean changes in EASI scores by body region at Week 8 were: head/neck –8.7 vs –4.4 (ADORING 1) and –8.8 vs –4.7 (ADORING 2); trunk –5.9 vs –2.6 (ADORING 1) and –7.2 vs –3.8 (ADORING 2); upper extremities –12.0 vs –5.2 (ADORING 1) and –15.0 vs –6.6 (ADORING 2); and lower extremities –8.6 vs –4.4 (ADORING 1) and –9.9 vs –5.5 (ADORING 2; all P <.0001). Tapinarof was well tolerated across sensitive areas, including the face/neck.

 spoke with Greiwe to learn more about tapinarof, which is a topical aryl hydrocarbon receptor agonist, the efficacy and safety findings from the ADORING 1 and 2 trials, and how it could potentially benefit patients with atopic dermatitis under the age of 2 years.

“I think with some of these new advancements and new indications, we're slowly moving into the pediatric realm, which, to be honest, is really important, because this is a group that typically suffers a lot and tends to have pretty refractory severe disease,” Greiwe said.

Tapinarof is currently FDA-approved under the name Vtama for the topical treatment of atopic dermatitis in adults and pediatric patients 2 years of age and older.

Greiwe J, Tallman A, Sublett JW, et al. Tapinarof Cream 1% Once Daily: Consistent Efficacy Across All Body Regions, Including Head And Neck, In Adults And Children Down To 2 Years Of Age With Atopic Dermatitis. Presented at: 2025 AAAAI/WAO Joint Congress, February 28-March 3. Abstract 595

FDA Approves VTAMA® (tapinarof) cream, 1% for the Treatment of Atopic Dermatitis in Adults and Children 2 Years of Age and Older. News release. Organon. December 16, 2024. Accessed March 5, 2025. https://www.organon.com/news/fda-approves-vtama-tapinarof-cream-1-for-the-treatment-of-atopic-dermatitis-in-adults-and-children-2-years-of-age-and-older/

Justin Greiwe, MD

Michelle Huffaker, MD, is the director of Clinical Translational Medicine in the Allergy Portfolio at the Immune Tolerance Network and a practicing allergist.

Edwin H. Kim, MD

Sayantani Sindher, MD

Michelle Huffaker, MD

 is a phenomenon that touches the lives of almost everyone in some way, whether one is a patient or has a relationship with a patient.

Allergies to food are a significant concern, given the potential for anaphylaxis among those with Type 1 hypersensitivities. In fact, close to 200,000 individuals require emergency medical care for allergic reactions to food allergens and more than 50% of the 33 million adults in the US with food allergies report experiencing severe reactions.

Parents had become concerned over the years about their children reacting to food allergies, so protocols for avoidance of allergens became the norm by the outset of the 21st century. The 2000 recommendations by the American Academy of Pediatrics (AAP) urged avoidance of major allergens such as peanuts, milk, and eggs.

Allergies to these food products appeared to rise among children in the US between 2000 - 2009.

 It was only recently that the allergy research space made a dramatic shift away from avoidance and toward early exposure and immunotherapy, resulting in the landmark 

US Food and Drug Administration (FDA) approval

 in 2020 of AR101 (Palforzia) as the first food allergy-related immunotherapy.

Since 2020, this dramatic shift in the field of food allergy research has given way to a variety of advancements. 2024 represented another major leap forward, with 2 significant FDA approvals: 

Omalizumab was the first treatment designed to prevent anaphylaxis resulting from exposure to 1 or more food allergens in adults and childrens aged 1 year and older with immunoglobulin E (IgE)-mediated food allergies. Epinephrine nasal spray, also known as ‘neffy,’ is the first alternative option to injectable epinephrine (in such products as the EpiPen).

 editorial team reached out to 3 experts in the field of allergy research to discuss these notable advancements, their significance as ‘firsts’ in the allergy space, and what they mean for patients now that they have been approved.

The team spoke with Edwin H. Kim, MD, associate professor of pediatrics at the University of North Carolina’s School of Medicine. We also spoke with Sayantani Sindher, MD, a clinical associate professor of allergy and immunology at Stanford Medicine. Our third expert was Michelle Huffaker, MD, the director of clinical translational medicine in the Allergy Portfolio at the Immune Tolerance Network.

Each of these speakers are leading experts in the field of allergy and immunology. Below are several notable quotes from 3 interviews on the topic of the aforementioned developments in the allergy space in 2024.

On Evolving Allergy Research and New Developments:

 I would absolutely agree that this has been the biggest year I could think of when it comes to food allergy therapeutics and treatment options. Really thinking back over many years, it's really just been about diagnosing the allergy and giving guidance and avoidance. Several years ago, we had the approval of peanut oral immunotherapy as well as clinical use of food products for this type of thing.

But these were very sort of narrow types of treatments that were only available through specialized clinics. So omalizumab being approved by the FDA is a dramatic change when it comes to food allergy. One of the biggest reasons, in my mind, is because pretty much every allergy office in the United States at this point is likely offering omalizumab for asthma or for hives and urticaria. So now to be able to offer that same medication that they have so much experience with for food allergy, inevitably, is going to increase access.

 It felt like a very momentous year for food allergy, with omalizumab, with neffy, and in fact with another product, Palforzia. The age range went down even lower, as it was previously 4 and up and now it's been shifted to 1 year olds and up. That's extending the age group for when we can offer therapies.

I think having all these treatment options early on changes the mindset when we think of allergy care, instead of what we used to do forever where we diagnose and we say ‘Okay great, now you just avoid it, hope for the best, and we'll see what comes around down the line.’ Whereas now, when I'm diagnosing a 5-month-old or 6-month-old, I can provide them with ‘You know, when you turn 1, you have these options.’ There are these other treatment options in the pipeline and there's just a lot more opportunities for patients to decide what's the right track for them.

The patients are very grateful to have options now. For years, it was avoidance and epinephrine auto injectors. And now they are very grateful that the field is moving forward and that there are more treatment options available, even if they don't just choose to elect those treatment options at this time. There are a variety of reasons that they might choose not to, but just having choices and options has really changed things for those patients.

On Omalizumab and Its Impact on Patients:

I think the earliest patients are on the therapy at this point. So it will take a little bit of time to really get an assessment of how this truly affects those patients. We know what to expect from the clinical trials, but real life is not always the same. I do want to bring up the point that I started with previously, that it increases access and one of the biggest things that it does is (function as) a treatment that can be given for any and all food allergies.

So it really can potentially address food allergies for almost every patient that's out there. The prior treatments, the oral immunotherapy, really could not. It will be really interesting to see the effect that this treatment has on patients, as well as whether there are ‘ideal patients’ for this or patients with certain food allergies do better than those with other food allergies. (There are) lots of these types of questions, so we can fine tune how we use this in the clinic.

I still remember when it got approved. I was away internationally, and I started getting texts from all my colleagues, along with emails. Patients were reaching out when the FDA first approved it, so my colleagues and I have been very excited about this development…One of the reasons I'm super excited is that it gives patients and families choices, because that's one of the things I am hoping for, with more and more advancements coming through…It is great for anyone who doesn't want the hassle of daily oral immunotherapy, which we can address later.

This has been a very exciting year for the world of food allergy and for patients and families dealing with food allergies. And one word you said there really hits on how omalizumab changes the landscape, and that's that's the word ‘options.’ Traditionally, food allergies have been managed completely with avoidance and managing reactions with epinephrine.

It can be frightening for patients. It can be frightening for families. It can be very challenging to avoid all the food allergens and what omalizumab does is it's an extra safety net against those accidental exposures. So decreases the likelihood that anaphylaxis will occur if you were to have an accidental exposure. And so it really opens up options for patients, it potentially liberalizes their diet. It also really changes the level of anxiety that there may be around food allergy.

On the Impact of Epinephrine Nasal Spray:

 I think this has the potential to have a dramatic effect, because every patient out there who has food allergy should have an epinephrine auto-injector to be used as treatment if they were to have an allergic reaction. So a change to that has the potential to affect pretty much every patient that's out there. We know that this is separate from the treatments, because the treatments are there to prevent allergic reactions, but when allergic reactions happen, we know that epinephrine is the best medicine.

That first line medicine that will treat all the symptoms and ideally prevent symptoms from becoming more significant or more severe. At the same time, what we have understood anecdotally, but also now more from actual data on usage, is that a lot of folks that could and should be using that epinephrine auto-injector just haven't been for a multitude of reasons. One of the biggest ones being the injection route itself. So now to have a medication that I hope people are going to be more willing to use, it'll be fantastic to see what kind of effect this will have from the usage of the medication and, hopefully, prevent more significant allergic reactions.

 I think a lot of our adolescent patients are very excited. They love the idea of just being able to spray in their nose and not the injection. We've had so many. We do a lot in our research unit and we do a lot of food challenges. As part of participation in a clinical trial, we have to show that you are truly allergic to a food, and the gold standard of diagnosis is giving them the food that they're allergic to and waiting for a reaction and then treating that reaction.

So we see kids and adults just more terrified about the injection than about the reaction itself. We've had patients who have sat through anaphylaxis on their own because they didn't want to inject themselves. There's also user error. Some parents may have injected themselves instead of the child in the process. So there's lots of kinds of barriers to getting optimal care when it comes to the injectables.

It is another exciting new option for patients in the world of food allergy. It's still kind of coming into the market, so we're still seeing how patients are adopting it. I think one thing that I'm hopeful about with it is that it will lower the bar for use of epinephrine. Giving yourself an injection often feels like a very big deal to patients, even though that really should be the first thing they turn to with systemic allergic reactions. Previously, we only had that through injection.

What I'm hoping is that patients that may be hesitant to give themselves an epinephrine injection would potentially give themselves a nasal spray of epinephrine, if the treatment is just as effective as the injectable form. As a clinician, I don't care which one gets into them, but I want one of them to get into them for patients having a reaction. So if the nasal spray can lower that bar for use, its has potential to save lives.

The quotes contained in this article were edited for the purposes of clarity. For further information, view the interviews posted above.

Kim has reported consulting fees from ALK-Abelló, Ukko Inc., Cellergy Pharma, DBV Technologies, Genentech, Hanimune Therapeutics, Novartis, Nutricia, Phyalxis BioScience, and Revolo Biotherapeutics; safety review committee membership with Allergy Therapeutics Ltd.; royalties from UpToDate; and has stock options for Cellergy Pharma.

Sindher has reported receiving research grant support from NIH, FARE, CoFAR, DBV, AIMMUNE, and Regeneron and has worked as an advisor for Genentech.

Facts and Statistics. Food Allergy Research & Education. https://www.foodallergy.org/resources/facts-and-statistics?gad_source=1&gclid=Cj0KCQiAm4WsBhCiARIsAEJIEzXD8KiNDPvqo8HvRtRBBTjbmyyWhktLJrOiNjLUEqTNRSyo_6_4Z3caAl0JEALw_wcB. Date accessed: December 16, 2024.

Committee on N. Hypoallergenic infant formulas. Pediatrics. (2000) 106(2):346–9. 10.1542/peds.106.2.346. Date accessed: December 16, 2024.

Branum AM, Lukacs SL. Food allergy among children in the United States. Pediatrics. (2009) 124(6):1549–55. 10.1542/peds.2009-1210. Date accessed: December 16, 2024.

FDA approves Xolair® (omalizumab) as first and only medicine for children and adults with one or more food allergies. Novartis. February 16, 2024. https://www.novartis.com/us-en/news/media-releases/fda-approves-xolair-omalizumab-first-and-only-medicine-children-and-adults-one-or-more-food-allergies#:~:text=EAST%20HANOVER%2C%20N.J.%2C%20Feb.,patients%20aged%201%20year%20and. Date accessed: December 16, 2024.

Office of the Commissioner. FDA approves first nasal spray for treatment of anaphylaxis. U.S. Food and Drug Administration. August 9, 2024. Accessed August 9, 2024. https://www.fda.gov/news-events/press-announcements/fda-approves-first-nasal-spray-treatment-anaphylaxis. Date accessed: December 16, 2024.

Kim, Sindher, Huffaker spoke with HCPLive about some of the year’s most important developments in the food allergy space, namely the approvals of omalizumab and neffy.

Hope on the Horizon: 2 Food Allergy Breakthroughs in 2024

An expert in dermatology reviews the phase 3 SUNSHINE and SUNRISE trials evaluating the use of secukinumab given in 2-week intervals in patients with HS and shares clinical pearls for managing  the condition.

SUNSHINE and SUNRISE Trials in HS

Renata Block, MMS, PA-C

Professor, Transfusion Medicine, Nagoya University Hospital

Matsushita discussed findings from the OLE FRONTIER4 study of Mim8.

Tadashi Matsushita, MD, PhD: Reducing Treatment Burden in Hemophilia A With Mim8

Mim8 prophylaxis twice weekly was well tolerated in people with hemophilia A with no discontinuations, a few treated bleeds, and 84% of participants had zero treated bleeds.

These findings, from the open-label extension FRONTIER4 study, were presented at the

 66th American Society of Hematology (ASH) Annual Meeting and Exposition

, held December 7-10, 2024, in San Diego, California, by Tadashi Matsushita, MD, PhD, Professor, Transfusion Medicine, Nagoya University Hospital.

“Mim8 has a small injection volume, and it is an subcutaneous injection. You don't have to do some intravenous injection every day, twice a day, so now it's not going to be necessary to inject intravenous injection,” Matsushita told 

The study included 37 of 39 patients that completed the FRONTIER1 phase 2 extension. All adverse events (AEs) were non-serious, except 3 (in 2 patients), which were considered unlikely to be related to Mim8 and resolved.Participants had an estimated mean annualized bleeding rate of 0.4 (95% CI, 0.17-0.82) for treated bleeding episodes with Mim8 Q2W dosing.

Mim8 (denecimig) is a next-generation, activated factor VIII mimetic, fully human bispecific IgG4 antibody. In this interview, Matsushita discussed the safety findings in FRONTIER4 and overviewed advantages of Mim8 over current therapies for hemophilia A, including its subcutaneous, twice weekly dosing.

“I think this portability of the devices should be very welcomed by the patient,” Matsushita said.

Matsushita T, Chowdary P, Banchev AM, et al. Safety and Efficacy of Mim8 Prophylaxis Administered Once Every Two Weeks for Patients with Hemophilia A with or without Inhibitors: Interim Analysis of the FRONTIER4 Open-Label Extension Study. Presented at: ASH Annual meeting; December 7-10; San Diego, California. Abstract 718

Authors