Ionis Pharmaceuticals' Donidalorsen Shows Promise in HAE Treatment: Phase 3 OASISplus Study Results

Ionis Pharmaceuticals announced promising results from its Phase 3 OASISplus study on donidalorsen, a potential treatment for hereditary angioedema (HAE). The study showed a 62% reduction in HAE attack rates from baseline and 84% of patients preferred donidalorsen over their prior medications. The FDA is reviewing the New Drug Application for donidalorsen, targeting a decision date of August 21, 2025. If approved, donidalorsen could become the preferred prophylactic choice for HAE patients.

KalVista Pharmaceuticals (KALV, Financial) has received a significant milestone with the marketing authorization from the UK's Medicines and Healthcare products Regulatory Agency (MHRA) for EKTERLY, a new plasma kallikrein inhibitor. This breakthrough drug is now the first oral on-demand treatment available for hereditary angioedema (HAE) in the UK, intended for patients 12 years and older. The approval follows the company's recent FDA approval and underscores its global mission to provide innovative HAE therapies [1].

EKTERLY's development is a notable achievement for KalVista, originating from their Salisbury, UK laboratories. The authorization is supported by findings from the extensive phase 3 KONFIDENT clinical trial, which involved 136 participants across 66 sites in 20 countries. The study, published in the New England Journal of Medicine in May 2024, demonstrated that EKTERLY delivers faster symptom relief, reduces attack severity, and resolves attacks more effectively than a placebo, while maintaining a similar safety profile [1].

Wall Street analysts forecast an average target price for KalVista Pharmaceuticals Inc (KALV, Financial) of $30.89, with a high estimate of $40.00 and a low estimate of $20.00. The average target implies an upside of 103.62% from the current price of $15.17. The consensus recommendation from 10 brokerage firms is a "1.6" rating, indicating an "Outperform" status [1].

In parallel, KalVista has submitted additional Marketing Authorization Applications (MAAs) for sebetralstat, another oral plasma kallikrein inhibitor, to regulators in the UK, Switzerland, Australia, and Singapore. If approved, sebetralstat will be the first oral on-demand treatment for HAE in these regions. The submissions are supported by data from the KONFIDENT phase 3 clinical trial and ongoing KONFIDENT-S open label extension trial [2].

KalVista's KONFIDENT-KID clinical trial, designed to evaluate the safety and efficacy of sebetralstat in a pediatric population aged 2-11 years, began dosing patients ahead of schedule in June 2024. If approved, sebetralstat would be the first oral on-demand therapy for this age group, and only the second FDA-approved on-demand therapy of any type for this population [2].

KalVista's focus on developing innovative HAE therapies highlights its commitment to addressing the significant unmet need for people living with HAE worldwide. The company's efforts to expedite the global availability of these therapies underscore its dedication to improving the lives of patients suffering from this debilitating disease.

References:
[1] https://www.gurufocus.com/news/2979288/kalvista-kalv-gains-uk-authorization-for-ekterly-as-hae-treatment-kalv-stock-news
[2] https://www.businesswire.com/news/home/20240930529468/en/KalVista-Announces-the-Submission-of-Additional-Marketing-Authorization-Applications-for-Sebetralstat-for-the-Oral-On-Demand-Treatment-of-Hereditary-Angioedema