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2021-03-31| R&DTrials & Approvals

Ionis’ Once-Monthly Treatment for Rare Hereditary Disease Achieves Trial Endpoints

by Daniel Ojeda
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On March 29th, Ionis reported their investigational antisense medicine, IONIS-PKK-LRx, significantly reduced the number of severe swelling attacks in patients of hereditary angioedema. The once-monthly treatment met all its endpoints.

Hereditary angioedema (HAE) is a genetic disease characterized by severe swelling of the skin, gastrointestinal tract, and upper airway. Some of these attacks can be life-threatening. There are several types of this disease, and two of them are caused by either dysfunction or lack of the C1 inhibitor protein.

There are numerous medications that reduce the number of attacks. However, they do not completely eliminate attacks, and several of them require multiple injections per week making it burdensome for the patients.

 

Antisense Therapy

IONIS-PKK-LRx developed using Ionis’ proprietary ligand-conjugated antisense (LICA) technology is an antisense therapy design to stop the production of a protein known as prekallikrein (PKK). PKK plays a crucial role in the inflammatory response associated with acute HAE attacks.

The safety and efficacy of IONIS-PKK-LRx were tested on a placebo-controlled Phase 2 clinical trial. The results showed that once-monthly injection of IONIS-PKK-LRx reduced by 90% the number of monthly HAE attacks in weeks 1 to 17. Additionally, 92% of patients had no attacks in weeks 5 to 17. By comparison, all patients in the placebo groups suffer from one or more attacks over the same period. The full results will be presented at a scientific conference later this year.

“These topline Phase 2 study results support a profile for IONIS-PKK-LRx as a potential best-in-class prophylactic treatment for patients with HAE, with excellent efficacy, safety, and tolerability along with the convenience of once per month low volume subcutaneous injections,” said Kenneth Newman, M.D., M.B.A., Ionis’ Vice President of Clinical Development and leader of the pulmonology and immunology franchise.

“These results highlight the potential benefits and advantages of IONIS-PKK-LRx for the treatment of hereditary angioedema and more broadly underscore the power of Ionis’ antisense technology to target the root causes of rare diseases like HAE.”

 

Current Competitors

The HAE market is getting packed with at least three FDA-approved therapies targeting the C1 protein pathway, and at least three others targeting the kallikrein pathway. Although, not all of these therapies would directly compete with IONIS-PKK-LRx.

Currently, there are two main types of treatments against HAE. One is used to prevent attacks, the second is used to stop or reduce the duration of the attacks. IONIS-PKK-LRx would be part of the first type.

Its main competitors would be CSL Behring’s Haegarda and Takeda’s Cinryze, and Takhzyro. All of them are FDA-approved treatments with a high efficacy rate at reducing and preventing HA attacks. Ionis’s drug is administered only once a month. That’s one advantage it has over other medications that require a more frequent dosage. If IONIS-PKK-LRx shows a high efficacy, it could help it stand out in the HAE market.

Related Article: Orladeyo’s FDA approval Heralds New Funding for BioCryst

 

 

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