USA – KalVista Pharmaceuticals’ experimental pill has succeeded in Phase 3 Trial, paving the way for first oral “On-Demand” treatment for rare swelling disorder hereditary angioedema 

In a statement Tuesday, KalVista said the drug, sebetralstat, met the study’s main and secondary goals, reducing the severity of swelling attacks significantly faster than did a placebo.  

Participants on sebetralstat first felt relief from symptoms a median of 1.6 hours after receiving a low dose and 1.8 hours after a high dose, within range of available, injectable medicines. 

KalVista also said, without offering specifics, that the drug had a safety profile “similar to placebo.”  

No serious treatment-related adverse events occurred, and no patients left the trial because of side effects. The company plans to file for U.S. approval in the first half of 2024 and later this year in Europe and Japan. 

Though hereditary angioedema, or HAE, is a rare condition estimated to affect one in 50,000 people globally, the disease has become a crowded area of drug research. 

Multiple medicines exist to either treat or prevent the disease’s hallmark swelling attacks, which can last for days if untreated and affect a variety of organs. All are limited in some way, however.  

Acute treatments such as Takeda’s Firazyr and Pharming Group’s Ruconest can require several injections each episode. Preventive options, like BioCryst Pharmaceuticals’ Orladeyo, reduce the rates of attacks, but don’t ward them off entirely. 

Those drawbacks have left room for improvement, and several drugmakers have stepped in to try to fill the gap. 

 CSL, Ionis Pharmaceuticals and Intellia Therapeutics are developing longer-lasting preventive therapies, while Kalvista and rival Pharvaris are advancing pills to treat swelling episodes as effectively as injectable drugs. 

Mid-stage study results reported in 2021 and published in The Lancet last year indicated Kalvista’s drug might fulfill that promise, with symptom relief occurring between 1.5 to 2 hours.  

That finding made the company’s Phase 3 study, a larger trial, closely watched by analysts and investors. Shares climbed by about one-third this year in anticipation. 

KalVista’s preliminary findings meet analysts’ expectations 

While KalVista only disclosed summary findings in its press release Tuesday, they suggest the drug has met analysts’ expectations.  

The median time to symptom relief observed is close to what was reported in testing of Ruconest and Firazyr, and within a “bull case” previously outlined by Leerink Partners analyst Joseph Schwartz. 

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In a recent survey of 44 HAE specialists, analysts at the investment bank Jefferies reported that specialists want an oral drug that could relieve symptoms sooner than 2.5 hours. A quarter of those surveyed said they’d switch their patients to Kalvista’s drug if it proved as effective as Firazyr. 

Still, the company didn’t share details that could help determine its competitive positioning versus injectable drugs as well as Pharvaris’ rival treatment. Among them are more granular details on the size and speed of its effects. 

About 40% of the patients treated in the study required a second dose of Kalvista’s drug, while about 10% needed rescue medication within 12 hours. That finding could have implications for sebetralstat’s standing versus Pharvaris’ drug. 

There are also questions about the size of the potential market for KalVista’s medicine, though experts polled by Stifel and Jefferies have suggested that such a drug, because of its convenience, could generate additional demand.  

Detailed results will be presented at a medical meeting later this month. 

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