New findings presented at ENDO 2026 reported sustained clinical benefits across classic CAH, hypoparathyroidism, primary aldosteronism, osteoporosis and X-linked hypophosphatemia.
New data presented at ENDO 2026 highlighted the continued evolution of endocrine care, with studies spanning rare disease management, long-term hormone replacement, artificial intelligence-supported screening and real-world treatment outcomes. Across several presentations, investigators reported sustained improvements in biochemical, metabolic, skeletal and patient-reported outcomes, while also emphasising the importance of long-term follow-up and careful interpretation of emerging data.
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Palopegteriparatide sustained long-term disease control in adults with hypoparathyroidism
Hypoparathyroidism is caused by insufficient parathyroid hormone (PTH), but conventional treatment with active vitamin D and calcium primarily addresses hypocalcaemia rather than replacing the missing hormone signal.1 Final results from the phase 3 PaTHway trial evaluated whether once-daily palopegteriparatide, a PTH(1-34) prodrug, could provide durable disease control over more than 3 years.
The trial enrolled 82 adults with hypoparathyroidism across European and North American sites, with 73 completing the open-label extension. At Week 182, most participants had achieved key biochemical treatment goals while substantially reducing reliance on conventional therapy. Overall, 96% were independent from therapeutic calcium doses, none required active vitamin D and 89% maintained normocalcaemia. Longer-term follow-up also showed stabilisation of bone turnover markers within the normal range, sustained patient-reported benefits, improved and stabilised eGFR, and normalisation of previously elevated 24-hour urine calcium. Treatment was generally well tolerated, with most treatment-emergent adverse events reported as mild or moderate.
These final PaTHway data support palopegteriparatide as a durable long-term treatment option for adults with hypoparathyroidism, with sustained effects across calcium control, conventional therapy independence, renal measures and patient-reported outcomes.
Further data on palopegteriparatide were presented in a related ENDO 2026 poster showing sustained improvements in work functioning and productivity among employed adults with chronic hypoparathyroidism through Week 156.
AI model may support more targeted screening for primary aldosteronism
Primary aldosteronism (PA) is an important and potentially under-recognised cause of hypertension. Although recent Endocrine Society guidance supports broader PA screening in patients with hypertension, implementation at scale may be challenging. An ENDO 2026 study evaluated whether an artificial intelligence (AI)-based model using routinely collected electronic health record data could help identify patients at increased risk before diagnosis.2
The XGBoost-based model was developed and validated using de-identified Mayo Clinic data from adults with PA and controls with negative renin/aldosterone screening. It achieved an AUROC of 0.71 for 30-day prediction and 0.67 for 1-year prediction. In a large holdout cohort of patients with hypertension, threshold selection had a substantial effect on how many patients would be flagged for screening. A lower threshold of 0.3 prioritised sensitivity and identified 68.0% of patients for screening, while a higher threshold of 0.6 prioritised specificity and identified 5.9%.
These findings suggest that AI-supported risk stratification could help make PA screening more targeted and operationally feasible, particularly where universal screening is difficult to implement. However, further validation and prospective evaluation are needed to determine whether this approach improves diagnostic yield, workflow efficiency or patient outcomes.
Crinecerfont linked with sustained metabolic improvements in adults with classic CAH
Long-term exposure to supraphysiologic glucocorticoid doses is an important contributor to obesity, insulin resistance and cardiometabolic risk in adults with classic congenital adrenal hyperplasia (CAH). At ENDO 2026, Dr Oksana Hamidi presented 2-year findings from the phase 3 CAHtalyst Adult study, which assessed weight-related and metabolic outcomes in adults receiving crinecerfont, a corticotropin-releasing factor type 1 receptor antagonist.3
The analysis included 182 adults with substantial baseline metabolic risk: 71% were overweight or obese and 42% had insulin resistance. Over 2 years, crinecerfont treatment was associated with sustained reductions in glucocorticoid exposure, alongside maintained improvements in weight, BMI, body composition and HOMA-IR. Mean glucocorticoid dose decreased by 37% at Month 12 and by 38% at Month 24. Among participants who were overweight or obese at baseline, 38% achieved more than 5% weight reduction at Month 24. Reductions in fat mass exceeded reductions in lean mass, suggesting a favourable effect on body composition. No new safety signals were observed during long-term treatment.
These findings suggest that crinecerfont may help reduce cardiometabolic risk in adults with classic CAH by enabling sustained reductions in glucocorticoid exposure while maintaining improvements in weight-related and insulin-resistance measures.
Further 2-year data for crinecerfont in classic CAH were also presented at ENDO 2026, including analyses of paediatric bone-age and predicted-height outcomes, adult glucocorticoid dose reduction, paediatric glucocorticoid exposure and metabolic outcomes, paediatric ACTH, 17-hydroxyprogesterone and androgen-related outcomes, and adult bone health outcomes.
We asked Dr Oksana Hamidi (Associate Professor, Division of Endocrinology, UT Southwestern Medical Center) about the key clinical takeaway from this research:
“Clinicians should recognize that targeting upstream ACTH signaling with crinecerfont enables meaningful glucocorticoid dose reduction while improving key cardiometabolic outcomes. Over two years, sustained benefits in weight and insulin resistance suggest a shift toward more physiologic disease control in CAH. These findings support integrating adjunctive therapies to minimize GC burden and may redefine long-term management toward reducing metabolic complications and improving overall patient health.”
Real-world romosozumab use associated with improved BMD in high-risk osteoporosis
Romosozumab is approved for postmenopausal osteoporosis, but real-world use may extend to other high-risk osteoporosis populations where treatment options are limited. A retrospective cohort study from a tertiary care centre in Saudi Arabia, presented by Dr Atheer Ismail, assessed romosozumab effectiveness, safety and treatment adherence across a broader osteoporosis population, including patients with cardiovascular disease or a history of malignancy.4
Among 154 patients prescribed romosozumab between January 2023 and December 2025, postmenopausal osteoporosis was the most common indication, followed by steroid-induced osteoporosis. In patients who completed treatment and post-treatment assessment, romosozumab was associated with significant improvements in lumbar spine and right femoral neck BMD and T-scores. Lumbar spine BMD increased from 0.817 to 0.900 mg/cm², while right femoral neck BMD increased from 0.706 to 0.729 mg/cm². Improvements at the left femoral neck were numerically favourable but did not reach statistical significance. No meaningful efficacy signal was reported among non-adherent patients.
These real-world findings support the effectiveness of romosozumab in improving BMD among patients who complete treatment, while reinforcing the importance of adherence. Safety findings were broadly acceptable in this cohort, with major adverse cardiovascular events reported in 2.8% of patients and one malignancy relapse. However, given the retrospective design and the need for careful cardiovascular risk assessment with romosozumab, larger studies with longer follow-up are needed to better define safety in patients with cardiovascular disease or prior malignancy.
Burosumab associated with real-world improvements in pain, stiffness and physical function in adults with XLH
Adults with X-linked hypophosphatemia (XLH) can experience pain, stiffness, fatigue and muscle weakness, which may contribute to progressive functional limitation and reduced quality of life. An item-level analysis from the XLH Disease Monitoring Program evaluated whether burosumab, an anti-FGF23 antibody, was associated with real-world improvements in patient-reported disease burden.5
The analysis included 109 burosumab-naïve adults with XLH, including 54 who initiated burosumab and 55 who received oral phosphate/active vitamin D. Patient-reported outcomes were assessed using the Western Ontario and McMaster Universities Osteoarthritis Index across pain, physical function and stiffness domains. Over 3 years, burosumab was associated with statistically significant changes in 17 of 24 WOMAC items, spanning pain, physical function and stiffness. These changes reflected improvements across several activities of daily living, including mobility, transfers, self-care and household tasks. By contrast, no statistically significant item-level changes were observed in the oral phosphate/active vitamin D cohort.
These findings suggest that burosumab may provide meaningful real-world benefits for adults with XLH across multiple aspects of pain, stiffness and physical function. The authors noted that future work should define clinically meaningful item-level WOMAC thresholds in adults with XLH to better benchmark treatment response.
Additional data presented on burosumab at ENDO 2026 also supported improvements in physical function and reductions in pain in adults with X-linked hypophosphatemia.
These ENDO 2026 presentations highlight important progress across several areas of endocrine practice, from treatments that address underlying hormonal or metabolic drivers of disease to tools that may help identify underdiagnosed conditions more efficiently. While several findings require longer follow-up, prospective validation or further real-world confirmation, the data point to an increasingly outcomes-focused approach in endocrinology, with sustained disease control, patient functioning, metabolic risk and quality of life emerging as key measures of treatment value.
References:
- Clarke B. Palopegteriparatide Treatment in Adults with Hypoparathyroidism: Final Results of the Phase 3 PaTHway Trial. ORF16-07. Presented at ENDO 2026, Chicgo, Il, USA, June 13-16, 2026.
- Lee A, et al. AI-Based Approach to Screening Primary Aldosteronism. SAT-830. Presented at ENDO 2026, Chicgo, Il, USA, June 13-16, 2026.
- Hamidi O. Crinecerfont Improves Weight-Related Outcomes and Insulin Resistance in Adults with Classic Congenital Adrenal Hyperplasia: 2-Year Results from the CAHtalyst™ Adult Study. ORF32-07. Presented at ENDO 2026, Chicgo, Il, USA, June 13-16, 2026.
- Ismail A. Real-World Effectiveness, Safety, and Compliance With Romosozumab in Osteoporosis: A Retrospective Cohort Study From a Tertiary Care Center. MON-822. Presented at ENDO 2026, Chicgo, Il, USA, June 13-16, 2026.
- Imel EA. Real-World Effectiveness of Burosumab in Adults with X-Linked Hypophosphatemia (XLH): An Item-Level Analysis of the Western Ontario and McMaster Universities Osteoarthritis Index. ORF08-05. Presented at ENDO 2026, Chicgo, Il, USA, June 13-16, 2026.
Cite: ENDO 2026: 5 data highlights across CAH, hypoparathyroidism, primary aldosteronism, osteoporosis and XLH. touchENDOCRINOLOGY. 30 June 2026.
Editor: Nicola Cartridge, Director of Content
Acknowledgment: This content has been developed independently by Touch Medical Media for touchENDOCRINOLOGY. It is not affiliated with ENDO. No funding was received in the publication of this short article. This article was created by the touchENDOCRINOLOGY team utilizing AI as an editorial tool (ChatGPT (GPT-5.4) [Large language model]. https://chat.openai.com/chat.) The content was developed and edited by human editors. No funding was received in the publication of this article. Thank you to the presenting authors for sharing their perspectives.
