Oral abstract session 2
The fourth session of the scientific track at the HAEi Regional Conference APAC returned to the oral abstracts and chair Dr Michihiro Hide. This time, 2 research studies were presented:
- Identification of candidate biomarkers for diagnosing type-2 hereditary angioedema and assessing disease severity via plasma N-glycomics
- Long-term safety and efficacy of oral deucrictibant for prophylactic and on-demand treatment of hereditary angioedema attacks: Results of the ongoing CHAPTER-1 and RAPIDe-2 extension trials.
Identification of candidate biomarkers for diagnosing type-2 hereditary angioedema and assessing disease severity via plasma N-glycomics
Dr Xiangyi Cui from China gave her presentation via video. She told the audience that the study’s background is that the current HAE test can be limited due to instability and challenges with sample handling, storage, and transport. She also noted that there are currently no biomarkers to assess disease severity in HAE Type 2, limiting prophylactic treatment optimization.
Early work, highlighted by Dr Cui, indicated that N-glycans could diagnose and assess the severity of HAE Type 1. This study aimed to explore the potential of plasma and glycan biomarkers in HAE Type 2.
Samples were collected from patients with HAE Type 2, mast-cell angioedema, and healthy controls. These were analyzed, leading to 109 N-glycans being identified in total and 63 passing quality control procedures. Several N-glycans were identified that were significantly different in HAE Type 2 patients and healthy controls. Of these, 2 N-glycans were significantly associated with disease severity.
Dr Cui concluded that these novel biomarkers show significant potential for diagnosing HAE Type 2, predicting edema size, and assessing disease severity. She told the audience that the team working on this research is excited about the potential.
Long-term safety and efficacy of oral deucrictibant for prophylactic and on-demand treatment of hereditary angioedema attacks: Results of the ongoing CHAPTER-1 and RAPIDe-2 extension trials
Despite being from Bulgaria, Associate Professor Anna Valerieva was a welcome addition to the Asia-Pacific region. She presented data on this potential new treatment on behalf of her co-authors.
Associate Professor Valerieva stressed that deucrictibant remains an investigational agent that is not currently licensed for use. It is being trialed for both on-demand and prophylaxis of HAE attacks. Associate Professor Valerieva characterized the current prophylactic therapies are having some drawbacks, and there is a need for a treatment that is easily administered with efficacy as good as injectable therapies. Deucrictibant is an oral agent. An immediate-release capsule is given as on-demand therapy, while an extended-release tablet is being tested as a prophylactic agent.
In both on-demand and prophylaxis studies, Professor Valerieva characterized the safety data as very encouraging. There were very few adverse events considered to be related to treatment and no treatment discontinuation.
In terms of efficacy, Associate Professor Valerieva suggested they are promising. The studies showed reductions in attacks. For prophylaxis, this reduction happens within 1 to 2 weeks of drug initiation. The reduction translated to a 14-month attack-free period for patients in the study. In the on-demand trial, there is a rapid reduction in the severity of attack symptoms, typically 2.6 hours (median time). 98.5% of patients experienced the start of symptom relief within 12 hours.
In conclusion, Associate Professor Valerieva said the studies provide evidence for the long-term safety and efficacy of deucrictibant. It is also well tolerated, Associate Professor Valerieva told the audience.
Some asked Professor Valerieva of the audience about the oral nature of the medication and its use in abdominal attacks where patients may be vomiting. She suggested that the absorption of the medicine is very rapid, about 5 to 10 minutes. Her view was that if the patient can keep the pill down for that long, it would work. There were also quite a lot of abdominal attacks in the study. The size of the pill was also asked. Professor Valerieva suggested it is no larger than a regular pill, such as an antibiotic. The availability of the medicine, or a liquid formulation, for children was also discussed, and trials of the drug in children are planned.