KalVista Pharmaceuticals said Tuesday that its oral, on-demand treatment for the swelling “attacks” commonly experienced by patients with a genetic condition called hereditary angioedema achieved the goals of a Phase 3 clinical trial.
Based on the study results, the company plans to submit a marketing application to the Food and Drug Administration by the middle of the year. Regulatory filings in Europe and Japan will be completed later in the year.
In the Phase 3 study involving 136 participants, a low and high dose of Kalvista’s drug, called sebetralstat, showed a median time to the beginning of symptom relief of 1.6 hours and 1.8 hours, respectively, compared to 6.7 hours for a placebo. The result was statistically significant and achieved the study’s main efficacy goal.
All of the study’s secondary efficacy outcomes were also met. There were no serious side effects reported in the study, and no patients withdrew from the study due to side effects. KalVista released the study results via a press release but will present data at a medical meeting on Feb. 25.
Hereditary angioedema, or HAE, is a rare, genetic disease that is debilitating for patients, and can be fatal. It is caused by a genetic defect that controls a specific blood protein. The mutation, in turn, triggers episodes of biochemical imbalances that cause swelling in different parts of the body, including the hands, feet, face, and airway. Airway swelling can cause asphyxiation, which is why HAE can be deadly. HAE attacks can also occur in the intestinal wall, which causes patients to experience severe bouts of abdominal pain, nausea, and vomiting.
Today, the most commonly prescribed treatment for an HAE swelling episode is administered by a self-injection just under the skin. If approved, KalVista’s sebetralstat would be the first treatment for HAE attacks that patients can take more conveniently by swallowing a single pill. The outcome from a mid-stage study was reported in 2021.
“We’re thrilled with the study results,” said KalVista CEO Andrew Crockett. “Our goal from the start was to provide patients with an innovative oral option to better manage their disease. We couldn’t be happier.”
KalVista’s study did not directly compare sebetralstat to the currently approved self-injected treatment, a generic medicine called icatibant. In its pivotal study conducted in 2011, icatibant showed a median time to initial symptom relief of just under one hour, and symptom severity was cut in half by a median of two hours. Cross-trial comparisons are difficult, however, because the study designs and baseline characteristics of participants in the sebetralstat and icatibant studies were different.
KalVista believes its pill will be preferred over icatibant because the injections are painful, causing patients to sometimes wait hours before treating themselves, said Crockett. But if approved, sebetralstat is likely to cost significantly more than generic icatibant.
The market for HAE treatments is relatively small, given that a majority of patients with HAE also take injectable or oral medicines to prevent or reduce the frequency of swelling attacks before they happen. Still, some of these patients carry an acute HAE drug as a “just in case” treatment. The target audience for sebetralstat will be those patients, as well as patients with HAE who choose to treat attacks acutely rather than with preventative drugs.
Peak sales of sebetralstat could reach $600 million in the U.S. and Europe, according to a forecast published by Leerink analyst Joseph Schwartz. He has a buy rating on the stock.
KalVista shares are up 31% this year, as investors anticipated the sebetralstat study readout. The company carries a $555 million market value.
Pharvaris, a Swiss drugmaker, is also developing a treatment for HAE swelling attacks that will start a Phase 3 study later this year. Intellia Therapeutics is developing a CRISPR-based treatment for HAE with the potential to be curative.
STAT+ artikel van Adam Feuerstein.
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