Aleena Banerji, MD, professor at Harvard Medical School and director of clinical care at the Center for Drug and Vaccine Allergy at Massachusetts General Hospital.

HAELO Phase 3: Lonvo-z Secondary Endpoints, QoL Data Detailed at EAACI

Lonvoguran ziclumeran (lonvo-z; Intellia Therapeutics), a one-time in vivo CRISPR gene-editing therapy for

, continued to demonstrate compelling efficacy across secondary endpoints in the phase 3 HAELO trial. The team presented new HAELO data in a late-breaking oral session at the European Academy of Allergy and Clinical Immunology (EAACI) Annual Congress 2026 in Istanbul and simultaneously published it in the 

 following the meeting, lead investigator Aleena Banerji, MD, professor at Harvard Medical School and director of clinical care at the Center for Drug and Vaccine Allergy at Massachusetts General Hospital, said the secondary endpoint data exceeded her expectations despite entering phase 3 with cautious optimism.

“Going into any phase 3 study [is] unpredictable [in terms of] what the efficacy is going to be,” Banerji said. “It was very exciting to see that not only did the data match what was being seen in the prior phase 1 and phase 2 study, but it really exceeded expectations."

Secondary Endpoints and HAE Attack Reductions

As previously reported, HAELO met its primary endpoint with an 87% reduction in mean monthly attacks in the lonvo-z arm versus placebo during the efficacy evaluation period from weeks 5 to 28.

 The newly disclosed secondary data built further on that result.

The monthly rate of moderate and severe attacks was reduced by 91% in patients receiving lonvo-z compared with placebo (0.11 vs. 1.23; 

 <.0001), and the monthly rate of attacks requiring on-demand treatment fell by 89% (0.19 vs. 1.79; 

Banerji called the absence of non-responders a particularly meaningful finding. Patient-level data demonstrated that all patients in the lonvo-z arm experienced attack-rate reductions from baseline during weeks 5 to 28.

"It's rare that we see that when you look across a lot of other trials or even outside of hereditary angioedema," Banerji said.

Quality of Life Improvements With Lonvo-Z

Patients receiving lonvo-z experienced a mean improvement of 17.04 points in the Angioedema Quality of Life (AE-QoL) total score compared with approximately 6.5 points in the placebo arm, a difference that exceeded the 6-point threshold considered clinically meaningful.

Banerji said that threshold context matters when interpreting the magnitude of that gain. “A 17-point change far exceeds what we believe is clinically meaningful," she noted. Patients in the trial reported being able to think differently about work, school, and travel—including activities like taking a cruise—without organizing their lives around the fear of an impending attack.

"It's the constant fear of that next attack and when it's going to occur," Banerji said. "Being able to change that and seeing this significant improvement in quality-of-life matching what we're seeing with efficacy is really promising for the real world."

The HAELO population included patients across a range of prior treatment histories, from those naive to long-term prophylaxis (LTP) to those who were attack-free on existing regimens. Notably, 20% of enrolled patients reported having complete disease control as their best response to prior long-term prophylaxis therapies and yet still chose to enroll.

Banerji addressed why patients already well-controlled on LTP would pursue an investigational gene editing therapy: the burden of treatment itself, including insurance approvals, supply management, and injection schedules.

“Just because they're doing well on long-term prophylaxis in terms of not having attacks or feeling that they are attack-free isn't the only aspect of hereditary angioedema,” she said.

All treatment-emergent adverse events (TEAEs) reported in the lonvo-z arm were mild or moderate, with no serious adverse events observed. The most common TEAEs exceeding placebo rates were infusion-related reactions, headache, fatigue, back pain, and upper respiratory tract infection.

Banerji said the safety monitoring focus centered on liver function, given the hepatic targeting mechanism of an in vivo CRISPR therapy. No transient or persistent elevations in liver function tests were observed, and infusion reactions resolved without interrupting dosing in any patient.

Plasma kallikrein levels decreased substantially by the first measurement at day 15, reached a steady state by week 5, and remained stable through the data cutoff of February 10, 2026, with all patients remaining LTP-free as of that date. 

Banerji said durability questions will require continued follow-up but drew confidence from the longer-term phase 1 and phase 2 data.

A rolling BLA submission for lonvo-z was initiated in April, and the company continues to anticipate regulatory approval and a US launch in the first half of 2027.

 When asked which patients she would prioritize, Banerji said her view has shifted toward broad candidacy.

"I think that every patient is eligible," she said. "The concerns are going to be around insurance and navigating some of those challenges, but in terms of talking to patients… we are going to help them understand that this is a newer treatment that's coming up on the market...and then go through all of the data, the efficacy, the safety concerns, the potential benefit, [and] risks. [We] help them think through this in a shared decision-making process so that they themselves can be the person that decides [if] is this the right treatment for them.”

Editor’s note: Reported disclosures for Banerji include CSL Behring and BioCryst Pharmaceuticals.

Intellia Therapeutics. Intellia Therapeutics Reports Additional Positive Phase 3 Results for Lonvoguran Ziclumeran (lonvo-z) in Patients with Hereditary Angioedema. June 13, 2026. 

https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-reports-additional-positive-phase-3

Derman C. Single-Dose Lonvo-z Cuts HAE Attacks by 87% in Phase 3 Trial, With Aleena Banerji, MD. HCPLive. Published May 11, 2026. 

https://www.hcplive.com/view/single-dose-lonvo-z-cuts-hae-attacks-87-phase-3-trial-aleena-banerji-md

Aleena Banerji, MD, reviews HAELO secondary data: 91% reduction in moderate/severe attacks, 17-point QoL gain, and zero non-responders for lonvo-z.

Aleena Banerji, MD

On March 16, Eli Lilly and Company released 

 from the phase 3 ADorable-1 study regarding lebrikizumab (Ebglyss) and its success in treating those with moderate to severe atopic dermatitis, including infants and children as young as 6 months.

These data suggest both of the ADorable-1 study’s co-primary endpoints were met while using this interleukin (IL)-13–targeting monoclonal antibody, currently 

-approved for moderate to severe atopic dermatitis in adults and adolescents.

 Lilly has also indicated its intention to submit the data to US and international regulators for a label expansion in younger pediatric patients.

Amy Paller, MD, chair of the department of dermatology at Northwestern University and an investigator in the study, spoke with 

 following the release of these findings. The following is a Q&A discussion with Paller about the biggest highlights:

What would you say stood out most among these findings from ADorable-1 as an investigator on this trial?

 The biggest takeaway is that we've been living with one advanced systemic only, for such a long time now, yet we have such a large part of our population, the pediatric population under 12, who have moderate to severe atopic dermatitis. They are just not responding well enough to topicals, or parents are putting on so much steroid that I'm sure that it's having some adverse effects over time. Not to mention the burden of disease and spending so much time.

The fact that we now have a second one that, hopefully, will move forward for FDA clearance is really thrilling to us as pediatric dermatologists. We've been waiting for this for a long time. We don't have any options other than what we've had for decades: methotrexate and cyclosporine. These are the options for kids who don't respond well to dupilumab or have an issue with it.

ADorable-1 enrolled patients as young as 6 months, a population where we have very few approved targeted options. How meaningful are these topline results to you as an investigator, and what does a 63% EASI-75 response rate tell us about the potential role of selective IL-13 inhibition in this age group?

 What it tells us is probably going to be on par with the type of changes that we've seen with dupilumab, and that's great. You know, we've all now had some experience with those who are 12 and above. We know that they're very different, these approaches, and we can't take a response to one and just apply it to the other. They're quite different, and again, it gives us an option. But it tells us that it's an option that looks to be pretty promising, and the majority, that is two-thirds of children within as short a period of 16 weeks, will have a tremendously meaningful improvement in their disease.

The trial used weight-based dosing alongside background topical corticosteroids. Can you walk us through the rationale for that design, and how confident are you that the efficacy signal here reflects lebrikizumab's contribution specifically, rather than the combination with topical therapy?

This has also been done with these other pediatric trials, including with dupilumab. It's just really not considered ethical in children to not provide some modicum of relief. Of course, these are children in whom the topicals weren't enough anyway, so putting them on a topical steroid on both arms is really an ethical move to try to assuage some of the pain, itch, and terrible aspects of this disease. That's where that comes from. The weight-based dosing, of course, reflects the fact that this is pediatrics.

We can't just give them adult doses, but it is tiered. So, the smallest children in the study would be getting something like the two loading doses, and then going to 125 every 4 weeks. Whereas the older children in the study would be, depending on where they were, if they were 40 kilograms and above, on the standard 250 milligram for 0, 2 weeks, and then every 2 weeks after that. If they were in the next tiered group, that is under 40, but I think it was over 15 milligrams, they would be getting the 250 after that, 2 weeks on an every-4-week basis.

Atopic dermatitis in infants and toddlers carries a significant burden not just for the patient but for the whole family. Beyond the clinical endpoints, what did you observe in terms of the real-world impact on patients and caregivers throughout the trial?

I don't know that that data is out there in the public domain yet, but I will just say, from my own experience, when you get something that works great, you get a lot of smiles on everybody's face. This is a disease that has a terrible burden, not just for the young children, but for the entire family. The parents don't sleep. When the kids don't sleep, the parents spend more time at the doctors and out of work. Kids are homesick, and it's a tremendous burden for, not just the parents but, of course, the entire family. Being able, in a relatively short period of time in this study, to turn around the burden of this disease really helps the physical and emotional burden for the entire family.

With Lilly planning regulatory submissions based on these data, what would a label expansion down to 6 months mean for how pediatric dermatologists approach treatment sequencing?

As I mentioned, it gives us options. I think we're very used to using dupilumab. I don't think that's going to go away, but I think that we now have an option for one or the other. There is going to potentially be a difference. We'll see what the FDA does. I really don't know, but the cutoff for going every 2-week-dosing with dupilumab was set at 30 milligrams. You get that 200-milligram every 2 weeks. Here, it's set at 40. So that means under 40, they'd be every 4 weeks based on the FDA. I don't know if that'll make a difference, but it gives a certain group of kids more of an on-label every 4 weeks.

For dupilumab, I often use it every 4 weeks for the kids in that group. That's 30 and above. This is just because that's the on-label for Europe, and it works just fine. The answer to your question is that we have options now for these kids of this age. We can have first-line dupilumab, we can have first line lebrikizumab. We certainly have options if they don't do well with whatever our first choice is.

What remaining questions would you most like answered in the next phase of research for lebrikizumab in this younger patient population?

I think it's always important to see a larger group beyond what's in the study. We want to see what the real-world experience is with first-line lebrikizumab, with changing from dupilumab to lebrikizumab. We certainly didn't see much in terms of any kind of adverse events during this trial, no new signals, and nothing of concern. But we'll keep an eye on whether there seems to be any difference with instance of ocular surface disease, although it's so low with dupilumab as well in this young population.

I think we're also going to be looking at the long term. One of the big questions: We talk now about the potential that dupilumab, which we know is on-label for asthma, and we know it is helpful for a variety of allergic and new mediated disorders. [The question is] whether there is going to be a role in reducing the development of other atopic diseases, in addition to reducing the severity of those diseases while on the drug; we just don't know how much of that is.

Specifically, the fact that the IL-4 receptor inhibitor of dopamine also inhibits IL-4 signaling directly…We may not get that with the IL-13-specific targeting. I think that will be an interesting question to look at further, looking at the development or the occurrence of these other allergic disorders with IL-13. That's going to be something. Then obviously, other questions that we have as well, are why vaccines? Is there any concern with giving a live vaccine? Theoretically, there would not be, but obviously that remains to be answered with real data. I think those are the 2 big questions that are out there.

The quotes used in this interview were edited for the purposes of clarity.

Disclosures for Paller include the following: AbbVie, AnaptysBio, Eli Lilly, Galderma, Incyte, Janssen, LEO Pharma, Novartis, Regeneron Pharmaceuticals, Inc., Sanofi – investigator; AbbVie, Amgen, Asana, Dermavant, Dermira, Eli Lilly, Forte, Galderma, LEO Pharma, Matrisys Bioscience, Menlo Therapeutics, MorphoSys/Galapagos, Novartis, Pfizer, Regeneron Pharmaceuticals. Inc., Sanofi – consultant.

Campbell P. Phase 3 Trial Supports Lebrikizumab in Pediatric Atopic Dermatitis. HCPLive. March 16, 2026. Accessed March 17, 2026. 

https://www.hcplive.com/view/phase-3-trial-supports-lebrikizumab-in-pediatric-atopic-dermatitis

Eli Lilly and Company. Lilly’s EBGLYSS (lebrikizumab-lbkz) is the first and only selective IL-13 inhibitor to deliver positive Phase 3 outcomes in patients aged six months to 18 years with moderate-to-severe atopic dermatitis | Eli Lilly and Company. Eli Lilly and Company. Published March 16, 2026. Accessed March 17, 2026. 

https://investor.lilly.com/news-releases/news-release-details/lillys-ebglyss-lebrikizumab-lbkz-first-and-only-selective-il-13

Phase 3 ADorable-1 topline data support lebrikizumab, a selective IL‑13 monoclonal antibody, in moderate-to-severe atopic dermatitis for patients aged 6 months to 18 years, meeting both co-primary endpoints and achieving ~63% EASI‑75 at 16 weeks with background topical corticosteroids. Weight-tiered dosing aligned with pediatric pharmacology and mirrored ethical trial norms for symptom control. A potential label expansion would add a second advanced systemic option for younger children, enabling alternative first-line sequencing and switch strategies beyond dupilumab and legacy immunosuppressants.

Following the release of new results from the phase 3 ADorable-1 trial on lebrikizumab in pediatric patients, Amy Paller, MD, speaks in a new Q&A interview.

Significance of ADorable-1 Data on Lebrikizumab in Children with Atopic Dermatitis, With Amy Paller, MD

Amy S. Paller, MD

Benjamin Natelson, MD

But maybe we could finish up this discussion. And Dr Seminerio, you could start us off. Maybe a piece of advice or one take home message from the discussion today and we could go around.

: The take home is that there is a lot that's coming and that it is going to be very, very important for us to have discussions with our patients, come up with a plan together, and just be very cognizant that that plan may need some adjustments. If we put that out there that it's not a failure if we have to make an adjustment or one treatment doesn't work but that we're on top of it as quickly as possible. That is the best thing we can do for our patients is to show that we're never going to give up on them.

I'd say a couple of things, one listen to your patient. Obviously, the shared decision, set the goals with the patient. And then number 2, like we've talked about is if you know that Crohn's disease, 80% of Crohn's disease, from the start will require an advanced therapy. Starting that earlier, Jen said that several times, but also then, even within that group, what are the ones with the highest risk factors? Fistula and deep ulcers, multiple segments involved versus which are the ones that are moderate to severe single segments. I know we have great options for both.

: Not much more to add other than we are not exactly at precision medicine and practice of precision medicine in IBD but we are certainly getting there. And again, identifying- initiate early appropriate therapy and recognize that we could still practice individualized care.

: I would echo everyone's comments here. But the key takeaway I would say is start our patients early. Don't delay their care. We can limit all the complications and poor outcomes if you start that patient early. Then personalize it based on all their risk, their severity, comorbidities. Choose appropriately. And then once you have done that, follow them closely over time. This is not a disease that ends after one year. This is a chronic lifetime disease. You cannot go wrong by starting early, choosing a therapy that you think may be the best for that patient. It may or may not work but if it doesn't then you know that you're following them closely and then follow them throughout their time. And of course, you can adjust how frequently you follow them once you get them into complete remission.

I agree. And just one last thing to add is personalizing the target. We've been hearing about treat to target so much. But the target for each patient is different. And as we started the discussion, it's different for each patient. Starting early and personalizing that target. But I want to thank everyone for this rich and informative discussion and thank you for watching this 

® Peer Exchange. If you enjoy the content, please subscribe to our e-newsletters to receive upcoming Peer Exchanges and other great content right in your inbox.

Experts in gastroenterology share advice for the treatment of patients of Crohn’s disease.

Take-Home Messages for the Management of Crohn’s Disease

Jennifer Seminerio, MD

John Sonnenberg, PhD, clinical psychologist, founder of Uptown Research Institute, and faculty at Northwestern University Feinberg School of Medicine.

Emerge Phase 3: DT120 Shows Rapid, Durable Relief in MDD

Emerge, a phase 3 trial evaluating a single dose of DT120 (lysergide) ODT 100 µg in adults with 

, met its primary endpoint and all key secondary efficacy endpoints, according to topline results announced by Definium Therapeutics on June 22, 2026.

“I was agnostic about whether psychedelics had true efficacy potential, and now I am convinced as a clinician that they do," said Emerge investigator John Sonnenberg, PhD, clinical psychologist, founder of Uptown Research Institute, and faculty at Northwestern University Feinberg School of Medicine, in an interview with 

Emerge, a multicenter, randomized, double-blind, placebo-controlled phase 3 study, enrolled 149 adults (18 – 74 years) with DSM-5-confirmed MDD across 20 sites. Participants received a single dose of DT120 ODT 100 µg or placebo. The trial met its primary endpoint, with a placebo-adjusted MADRS reduction of 8.1 points at week 6 (

 <.0001). The placebo-adjusted reduction at week 1 was 14.2 points (

 <.0001), and a 7.3-point difference was sustained at week 12 (

Sonnenberg noted that early responses are common in effective antidepressant interventions. The week 1 signal was less notable to him than its persistence.

"The fact that it stood up [for] over 6 weeks and the fact that it persisted to 12 weeks is, from a clinician-researcher standpoint, very impressive," he said.

Sonnenberg said patients experienced fewer symptoms and reported being less troubled by the ones that remained, an observation that contextualizes the remission data. The 24% remission rate at week 6, compared with 3% for placebo, was statistically significant, though roughly 3 quarters of patients on active drug did not reach remission.

Sonnenberg characterized that gap as consistent with a first-generation result in a new treatment area. He noted that Part B open-label extension data is already informing questions about which patients may benefit from a second dose.

Safety Profile and the Dosing Environment

DT120 ODT was generally well tolerated in Emerge. Of treatment-emergent adverse events, 99% were mild to moderate in severity. No serious adverse events or suicidality signals were identified. Sonnenberg noted that adverse events occurring within the supervised dosing session, rather than at home, allow clinical staff to respond in real time.

The mean time to meeting end-of-session checklist criteria was 5.8 hours. Sonnenberg said most patients at his site were coherent and able to return home independently by hour 6, with the final 2 hours used for observation.

3Sonnenberg addressed Ascend, Definium's second Phase 3 MDD study, which includes a 50 µg low-dose arm designed to address functional unblinding concerns. Sonnenberg addressed Ascend, Definium's second Phase 3 MDD study, which includes a 50 µg low-dose arm designed to address functional unblinding concerns. He called the design important, pointing to Phase 2b GAD data suggesting that correctly identifying one's dose condition alone does not account for clinical outcomes, and said Ascend is intended to confirm that finding.

"I've never worked on a drug that could create this type of profound change in such a short period of time with the ability to maintain that change for 12 weeks," Sonnenberg said. "This is at a different level."

Definium Therapeutics. Definium Therapeutics. Published June 22, 2026. Accessed June 23, 2026. 

https://ir.definiumtx.com/news-events/press-releases/detail/232/definium-therapeutics-announces-positive-topline-results-from-phase-3-emerge-study-of-dt120-orally-disintegrating-tablet-odt-in-major-depressive-disorder

John Sonnenberg, PhD, reflects on Emerge Phase 3 topline data for DT120 in MDD, including MADRS outcomes, remission rates, and what comes next.

John Sonnenberg, PhD

Experts discuss the recent use of biosimilars like inolimomab as treatment for ulcerative colitis (UC).

Biosimilar Treatment Options for Patients With UC

Maia Kayal, MD

Pulmonary critical care physician, Cone Health

Monoclonal Antibodies and the Future of Lung Preservation in COPD

, Joseph Khabbaza, MD, moderates an expert discussion with Nikita Desai, MD, and Eric Costanzo, MD, on the impact of and recent developments with monoclonal antibodies in chronic obstructive pulmonary disease (COPD).

In this clip, Desai and Costanzo reflected on the future direction of monoclonal antibody use in COPD, including sequencing, combination therapy, and long-term impact on disease progression. Desai highlighted that, while current use is largely shaped by payer approvals and clinical trial inclusion criteria, she typically assesses efficacy after 6 to 12 months before switching agents, acknowledging the need for head-to-head trials to inform sequencing decisions. Simultaneous biologic therapy, while mechanistically plausible, remains unlikely in the near future due to cost and logistical barriers.

Turning to disease modification, Costanzo emphasized the importance of early, upstream intervention to limit inflammatory cascades that drive irreversible lung remodeling. He underscored the clinical value of preserving lung function, noting that while COPD cannot be cured, biologics may arrest progression and mitigate long-term damage. Both clinicians agreed that unchecked type 2 or neutrophilic inflammation leads to structural changes and that the evolving use of targeted therapies offers an opportunity to protect lung health over time. As the field advances, they anticipate growing physiologic and imaging data will reinforce biologics’ role in modifying COPD's natural history.

is a pulmonary and critical care physician at the Cleveland Clinic and a director of the non-cystic fibrosis bronchiectasis program., who serves as the moderator for this panel discussion.

is a pulmonary critical care physician at Cone Health in Greensboro, North Carolina. She is triple board certified in critical care medicine, internal medicine, and pulmonary medicine.

is a pulmonary critical care and neurocritical care physician and program director of the Pulmonary and Critical Care fellowship at Jersey Shore University Medical Center with Hackensack Meridian Health System and Shore Pulmonary Associates

Relevant disclosures for Khabbaza include Insmed, Regeneron, Sanofi, and Baxter Healthcare Corporation. Desai and Costanzo have no relevant disclosures to report.

Nucala (mepolizumab) approved by US FDA for use in adults with chronic obstructive pulmonary disease (COPD). News release. GSK. May 22, 2025. https://www.gsk.com/en-gb/media/press-releases/nucala-mepolizumab-approved-by-us-fda/

In this video, the last in a 6-part series, panelists discuss the potential future impact of biologics in the COPD field.

Nikita S. Desai, MD

Oksana Hamidi, DO, is an associate professor in the division of endocrinology and metabolism at the University of Texas Southwestern Medical Center.

Crinecerfont Enables Sustained Glucocorticoid Dose Reductions Through 2 Years in Adults With CAH

2-year data from the CAHtalyst Adult Study suggest treatment with crinecerfont (Crenessity) enables sustained reductions in glucocorticoid (GC) exposure among adults with classic congenital adrenal hyperplasia (CAH), with nearly 70% of participants achieving physiologic replacement dosing after 24 months.

Presented at the Endocrine Society (ENDO) Annual Meeting 2026, the analysis showed participants maintained substantial reductions in GC doses through 2 years of treatment while generally preserving androgen control, addressing a longstanding challenge in CAH management. Historically, many patients require chronic supraphysiologic GC doses to suppress excess adrenal androgen production, exposing them to increased risks of obesity, hypertension, insulin resistance, osteoporosis, and other complications.

"At 24 months, nearly 70% of patients were able to achieve physiological corticoid dose replacement, defined as hydrocortisone equivalent less than 11 milligram per meter square per day," Oksana Hamidi, DO, associate professor in the division of endocrinology and metabolism at UT Southwestern Medical Center, told 

. "That was definitely a significant and very meaningful finding."

Sustained Reductions in Glucocorticoid Exposure

The phase 3 CAHtalyst Adult Study (NCT04490915) enrolled 182 adults with classic CAH. Participants completed a 24-week double-blind, placebo-controlled phase followed by a 6-month open-label period and an ongoing open-label extension.

At baseline, participants were receiving a mean total daily GC dose of 17.6 mg/m²/day, equivalent to 32.3 mg/day hydrocortisone.

Investigators evaluated GC dose reductions using 2 approaches. The primary analysis assessed dose reductions while maintaining or improving androstenedione (A4) levels relative to baseline. Under this method, any GC reduction associated with worsening A4 was imputed to zero.

Using this more conservative analysis, mean GC dose reductions were 26% at Month 12, 28% at Month 18, and 25% at Month 24. At Month 24, 45% of participants achieved physiologic GC dosing of ≤11 mg/m²/day while maintaining or improving A4 control.

Investigators also examined observed GC doses at Months 18 and 24, reflecting real-world clinical decision-making after protocol-mandated titration requirements ended.

The observed analysis demonstrated larger reductions, with mean GC doses decreasing by 38% from baseline at both Month 18 and Month 24. Mean observed GC dose fell to 10.6 mg/m²/day (19.4 mg/day), and 69% of participants at Month 24 achieved physiologic replacement dosing.

"When we are representing the data, it's important to know the methods behind it," Hamidi said. "More real-world data would be the observed glucocorticoid dose reduction."

She explained that the observed-dose analysis reflects clinician-guided dose adjustments based on laboratory values, tolerability, and individual patient circumstances rather than protocol-driven requirements.

According to Hamidi, the significance of the findings extends beyond reductions in GC dose alone.

Patients entered the study receiving a mean hydrocortisone-equivalent dose of approximately 32 mg/day, substantially above physiologic replacement levels. Despite these reductions, investigators reported relatively stable A4 levels through Month 24, with mean changes of -11.5 ng/dL at Month 18 and +56.6 ng/dL at Month 24.

Hamidi noted that reducing chronic GC exposure may ultimately translate into improvements in metabolic health and quality of life.

"I want to see improvements in patients' weight, blood pressure measurements, as well as body composition changes that are favorable," she said. "I hope that we are able to improve quality of life of patients who have low quality of life at the beginning."

Crinecerfont was generally well tolerated through 24 months, with no new safety signals identified.

Investigators reported several cases of adrenal insufficiency and adrenal crisis, underscoring the importance of patient education regarding stress dosing and crisis prevention during GC dose reduction. One fatal event occurred in a participant with cholecystitis and bacterial infection during the open-label extension; investigators considered the event unlikely to be treatment-related.

As clinicians begin incorporating crinecerfont into routine practice, Hamidi recommended a gradual approach.

"I always advocate for making one change at a time," she said. "Brand new medication, start with that first, observe that patient, check their levels, see how they're doing, then potentially consider glucocorticoid dose reduction, if that's applicable."

Editor’s Note: Hamidi reports relevant disclosures with Corcept Therapeutics, Neurocrine Biosciences, Xeris Pharma, Camurus, Crinetics Pharmaceuticals, and Recordati Rare Diseases.

Bancos I, Hamidi O, Kiefer FW, et al. Adults with classic congenital adrenal hyperplasia taking crinecerfont demonstrated sustained decreases in glucocorticoid doses: 2-year results from the CAHtalyst Adult Study. Poster presented at: ENDO 2026; June 14, 2026; Chicago, IL.

Hillenbrand A. Crinecerfont sustains glucocorticoid reduction, improves cardiometabolic outcomes in classic CAH. 

. Published June 15, 2026. Accessed June 18, 2026. 

https://www.hcplive.com/view/crinecerfont-sustains-glucocorticoid-reduction-improves-cardiometabolic-outcomes-in-classic-cah

Crinecerfont enabled sustained GC dose reductions over 2 years, with nearly 70% of adults with classic CAH reaching physiologic dosing.

Nearly 70% of Adults With Classic CAH Reach Physiologic GC Dosing With Crinecerfont

Oksana Hamidi, DO

Biliary complications remain a common source of morbidity following liver transplantation. The most common post-transplant biliary complications are strictures and leaks. Endoscopic retrograde cholangiopancreatography (ERCP) has become the first line of therapy for post-transplant biliary complications. However, outcomes of ERCP in early as compared to late liver transplant period are not known. We aimed to study the safety and outcomes of late (> 30 days) versus early (≤ 30 days) ERCP in liver transplant recipients with biliary complications.  

After IRB approval, we conducted a retrospective analysis of patients who underwent ERCP after liver transplant at Cleveland Clinic. Variables including transplant indication; MELD score at the time of transplant; baseline labs prior to ERCP; and procedure factors such as antibiotic use, prior medication use, and post-procedure outcomes were collected. Patients were then divided into 2 groups: ERCP ≤ 30 and ERCP > 30 days after liver transplant. The outcomes were technical success, clinical success, and adverse events post-ERCP.  

A total of 174 patients were included in our analysis. Mean age, gender, race, BMI, etiology of liver disease, type of liver graft, and type of anastomosis were similar in both groups. Patients who had ERCP in ≤ 30 days after liver transplant had significantly lower hemoglobin, alkaline phosphatase, bilirubin, and albumin as well as higher white cell count compared to patients who underwent ERCP > 30 days post-transplant. More patients in the ERCP ≤ 30 days group were on anticoagulation (10.6% vs 3.2%; 

= .049) with no difference in antiplatelet drugs.

ERCP ≤ 30 days after transplant was performed more commonly for indication of bile leak (36.2% vs 4.7%), whereas ERCP > 30 days post-transplant was more often performed for biliary stricture (90.6% vs 57.4%; 

 <.001). Patients who underwent ERCP ≤ 30 days at significantly high rates of post-procedure bleeding (12.8% vs 3.9%; 

In terms of procedural details, there were no significant differences in patients with prior ERCP and sphincterotomy done, history of Roux-en-Y anatomy, antibiotic use during the procedure, or advanced cannulation technique. However, there were significantly high rates of balloon dilation in patients who underwent ERCP > 30 days post-liver transplant.

Technical success, late postprocedural bleeding, post-ERCP pancreatitis, stent deployment, dislodgment, and cardiopulmonary complications were similar in both groups. The need for future ERCP, interventional radiology, or surgical interventions was also similar in both groups.  In this study, we report high rates of early post-procedure bleeding in patients who underwent ERCP ≤ 30 days after liver transplant as compared to ERCP > 30 days with overall similar rates of technical and clinical success. These data will help risk stratify patients prior to ERCP and raise awareness of the higher risk of bleeding in the early liver transplant period. 

Patients who underwent ERCP ≤ 30 days post-transplant had greater rates of early post-procedure bleeding but similar rates of technical and clinical success compared to late ERCP.

Safety, Outcomes of Early Versus Late ERCP in Liver Transplant Recipients

Rajat Garg, MD

 I’d love for you to share with the viewers what strategies you have implemented based on your experiences to educate patients about their journey. Because that’s so important, as John has said, about making this a patient-centered diagnosis. What’s a day in the life of Dr Maron and Dr Ommen in how you approach these issues?

 I’ll go first. Like I’m sure Steve does, we spend a lot of time with patients when they come and do our own education with them, along with our health care team and nurse practitioners to educate patients about the disease. We spend a lot of time on education, reassurance, putting these issues that we’ve talked about in perspective, and doing a lot of shared decision-making, going through treatment options. At the end of the day, we try to deliver the message that hypertrophic cardiomyopathy [HCM] is a highly treatable disease. It’s going to probably be more so in the future, but even today it’s very treatable and a disease that’s compatible with normal life expectancy and good quality of life for the vast majority of patients. That’s the messaging that we give to patients the best that we can.

 I’d echo the same thing. Patients come to us after having done their own education attempts on the internet, freaked out with wide eyes because they’ve read all the dramatic stories that are available, and we have to remind them that people who have normal lives don’t usually blog about it.

I’d like to reiterate the same words about HCM in the modern era. We’ve gone from an era where it was 100% fatal in 1958 when Donald Teare described it to a 6% annual mortality in the 1980s to an annual mortality that’s the same as the general population. It’s a highly treatable disease for most patients. There are patients in whom there are issues. This discussion wasn’t about sudden cardiac arrest, which would be another 2 hours. Then there are quality-of-life issues that some patients have. But it’s a highly treatable disease and we’re getting more tools in our toolbox for the future, which is exciting.

 That’s super. Javed, any final thoughts you want to share?

 My final thought is that we have these therapies and we want to do anything and everything that we can for the patients to not unnecessarily suffer. The question is how do we educate the clinical community at large to refer the patients to the appropriate centers and to diagnose and screen these patients? While the specialists are very interested in and have a lot of these tools, more mass education about this disease would also be needed.

 From an administrator’s point of view, let me ask you to put that hat on for a second. When you’re looking at making recommendations for clinicians who are interested in getting involved in this, what things would you say are needed in terms of understanding things like catheter-based approaches, surgical approaches, and volume, which you talked a bit about? What do you think about setting certain expectations?

 You can modify behavior quite a lot by creating appropriate incentive structures in terms of education, screening, and quality metrics. You can also set up tools. We live in this digital age and everybody is on an electronic health record. A lot of the education and best practice advisories can be had for our patients in this era. Education and reminder systems are easier in the current day and age.

 Super. John, I’m going to turn to you last. You always keep the patient in the center. I’m curious to hear your final thoughts.

 First, I’m humbled by the expertise at this panel and not being an HCM expert. I feel a tremendous need to think about the infrastructure for elevating the quality of care throughout the nation. What does that entail? Does it entail thinking about algorithms, building shared decision-making tools, integrating routine assessments of health status with the KCCQ [Kansas City Cardiomyopathy Questionnaire], and coming up with very practical management strategies so that you don’t have to be in Boston or Rochester, Minnesota, to get high-quality care?

 Or Kansas City. That’s what I’m left with. It’s a complicated disease, but it’s an exciting disease. As more treatment options become available, there’s a greater call for thinking about the infrastructure of delivering high-quality patient-centered care. That’s what I’d like to see us move toward so that we can all benefit from the advances being made.

 Super. That’s a great way to conclude. I want to thank all of you for this rich and informative discussion. I want to thank you, our viewers, for watching this 

. If you enjoyed the content, please subscribe for our e-newsletters to receive upcoming 

 and other great content right in your inbox. I’m Jim Januzzi from the Massachusetts General Hospital. Thanks very much for joining.

Expert cardiologists share strategies for educating patients on the treatment and management of hypertrophic cardiomyopathy. 

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