KalVista Pharmaceuticals Announces Early Completion of Enrollment in KONFIDENT-KID Pediatric HAE Trial

Rhea-AI Summary

KalVista Pharmaceuticals (KALV) has completed enrollment for its KONFIDENT-KID clinical trial of sebetralstat, an oral plasma kallikrein inhibitor for pediatric hereditary angioedema (HAE) patients aged 2-11. Due to high demand, the trial expanded from 24 to approximately 36 patients across seven countries in North America, Europe, and Asia.

The enrollment was completed a full year ahead of schedule. The trial will collect safety, pharmacokinetic, and efficacy data for up to one year using a proprietary pediatric oral disintegrating tablet formulation. Initial results are expected before year-end, with an sNDA submission planned by mid-2026.

If approved, sebetralstat would become the first oral on-demand therapy for pediatric HAE patients aged 2-11 years and only the second FDA-approved on-demand treatment in this population. Currently, the only available on-demand treatment for this age group in the U.S. requires intravenous administration.

Positive

• Early completion of trial enrollment one year ahead of schedule
• 50% increase in trial size due to high patient demand (36 vs 24 planned)
• Potential first-to-market position for oral on-demand pediatric HAE treatment
• Global trial reach across seven countries in North America, Europe, and Asia

Negative

• Long timeline to potential approval with sNDA submission not expected until mid-2026

Insights

Biotechnology Clinical Development Expert

KalVista's announcement represents significant operational execution in their sebetralstat development program. Completing enrollment a full year ahead of schedule in the KONFIDENT-KID trial - and expanding from 24 to approximately 36 patients due to high demand - demonstrates exceptional site engagement and strong interest from the HAE patient community.

The pediatric HAE treatment landscape currently offers options, with only one FDA-approved on-demand therapy for children aged 2-11, which requires intravenous administration. This creates a substantial barrier for young patients and caregivers. Sebetralstat's oral disintegrating tablet formulation specifically developed for children could represent a meaningful advancement in treatment convenience if approved.

The accelerated timeline now positions KalVista to present initial data before year-end 2025, with supplemental New Drug Application (sNDA) submission targeted by mid-2026. This compressed development schedule potentially reduces cash burn while accelerating the path to potential commercialization.

From a regulatory perspective, the expanded trial size could strengthen the data package, though we must wait for efficacy and safety results before making any assessment of approvability. The geographic diversity across seven countries in North America, Europe, and Asia should provide robust data applicable to global regulatory submissions.

Pharmaceutical Market Analyst

The exceptional enrollment pace in KalVista's KONFIDENT-KID trial signals strong market demand for an oral on-demand HAE treatment option in pediatric patients. This demographic represents a particularly underserved population with significant treatment burden using current options.

The company's proprietary oral disintegrating tablet (ODT) formulation for children could establish a meaningful competitive advantage if approved. Current standard-of-care requiring intravenous administration presents substantial challenges in pediatric patients, creating a clear opportunity for differentiation and potential market capture.

The expanded trial size from 24 to 36 patients reflects both clinical interest and KalVista's commitment to generating robust data. While representing a relatively small absolute number of patients, this is reflective of the rare disease setting of HAE. The geographic diversity across seven countries indicates KalVista is positioning for global commercial opportunity.

The accelerated development timeline presents a positive catalyst flow for investors, with data expected before year-end 2025 and regulatory submission by mid-2026. This compressed timeline potentially reduces development costs while accelerating potential revenue generation if approved. The clear articulation of development milestones provides investors with transparent indicators to monitor program progress.

Initial target enrollment surpassed in fewer than seven months; trial expanded due to high demand

First data presentation expected before year-end; sNDA submission by mid-2026

CAMBRIDGE, Mass. & SALISBURY, England--(BUSINESS WIRE)-- KalVista Pharmaceuticals, Inc. (Nasdaq: KALV) today announced the completion of enrollment in the open-label KONFIDENT-KID clinical trial of sebetralstat, an investigational, novel, oral plasma kallikrein inhibitor, in pediatric patients between the ages of two and 11 with hereditary angioedema (HAE).

“We're proud to share that we achieved target enrollment in our KONFIDENT-KID trial a full year ahead of schedule and expanded the trial size due to overwhelming interest,” said Ben Palleiko, CEO of KalVista. "The high level of participation from families living with HAE underscores the significant need for an oral treatment option for this population. Even with this larger trial size, we anticipate that initial results will be shared later this year, with an sNDA expected to be filed by mid-2026.”

Originally designed to enroll 24 pediatric patients, the trial was met with high demand and will ultimately include approximately 36 patients between the ages of two and 11 across seven countries in North America, Europe, and Asia. KONFIDENT-KID will collect safety, pharmacokinetic, and efficacy data for up to one year and features a proprietary pediatric oral disintegrating tablet (ODT) formulation of sebetralstat.

Currently, the only on-demand treatment for HAE patients of this age range approved in the U.S. is administered intravenously, highlighting a critical need for new therapeutic options. If approved, sebetralstat would be the first oral on-demand therapy for pediatric patients aged two to 11 years and only the second FDA-approved on-demand therapy of any type in this population.

For more information about KONFIDENT-KID, please visit clinicaltrials.gov.

About Sebetralstat

Sebetralstat is an investigational, novel oral plasma kallikrein inhibitor for the treatment of hereditary angioedema (HAE). We have filed multiple regulatory applications seeking approval of sebetralstat as the first oral, on-demand treatment for HAE in individuals aged 12 and older and are investigating its use in children aged 2 to 11. If approved, sebetralstat has the potential to become the foundational therapy for HAE management worldwide.

About Hereditary Angioedema

Hereditary angioedema (HAE) is a rare genetic disease resulting in deficiency or dysfunction in the C1 esterase inhibitor (C1INH) protein and subsequent uncontrolled activation of the kallikrein-kinin system. People living with HAE experience painful and debilitating attacks of tissue swelling in various locations of the body that can be life-threatening depending on the area affected. All currently approved on-demand treatment options require either intravenous or subcutaneous administration.

About KalVista Pharmaceuticals, Inc.

KalVista Pharmaceuticals, Inc., is a global biopharmaceutical company dedicated to developing and delivering life-changing oral therapies for individuals affected by rare diseases with significant unmet needs. Our lead investigational product is sebetralstat, a novel, oral, on-demand treatment for hereditary angioedema (HAE). Sebetralstat is under regulatory review by the U.S. FDA, with a PDUFA goal date of June 17, 2025. In addition, we have completed Marketing Authorization Applications for sebetralstat to the European Medicines Agency and multiple other global regulatory authorities.

For more information about KalVista, please visit www.kalvista.com or follow us on social media at @KalVista and LinkedIn.

Forward-Looking Statements

This press release contains "forward-looking" statements within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: "anticipate," "intend," "plan," "goal," "seek," "believe," "project," "estimate," "expect," "strategy," "future," "likely," "may," "should," "will" and similar references to future periods. These statements are subject to numerous risks and uncertainties that could cause actual results to differ materially from what we expect. Examples of forward-looking statements include, among others, timing or outcomes of communications with the FDA, our expectations about safety and efficacy of our product candidates and timing of clinical trials and its results, our ability to commence clinical studies or complete ongoing clinical studies, including our KONFIDENT-S and KONFIDENT-KID trials, and to obtain regulatory approvals for sebetralstat and other candidates in development, the success of any efforts to commercialize sebetralstat, the ability of sebetralstat and other candidates in development to treat HAE or other diseases, and the future progress and potential success of our oral Factor XIIa program. Further information on potential risk factors that could affect our business and financial results are detailed in our filings with the Securities and Exchange Commission, including in our annual report on Form 10-K for the year ended April 30, 2024, our quarterly reports on Form 10-Q, and our other reports that we may make from time to time with the Securities and Exchange Commission. We undertake no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.

View source version on businesswire.com: https://www.businesswire.com/news/home/20250325201996/en/

Ryan Baker

Head, Investor Relations

(617) 771-5001

ryan.baker@kalvista.com

Molly Cameron

Director, Corporate Communications

(857) 356-0164

molly.cameron@kalvista.com

Source: KalVista Pharmaceuticals, Inc.

FAQ

When will KalVista (KALV) release initial results for the KONFIDENT-KID pediatric HAE trial?

Initial results from the KONFIDENT-KID trial are expected to be shared before the end of 2023.

How many patients are enrolled in KalVista's KONFIDENT-KID trial for sebetralstat?

The trial expanded from its original target of 24 to approximately 36 pediatric patients due to high demand.

When does KalVista plan to submit the sNDA for sebetralstat pediatric HAE treatment?

KalVista plans to submit the sNDA by mid-2026.

What makes sebetralstat unique for pediatric HAE patients aged 2-11?

If approved, sebetralstat would be the first oral on-demand therapy for this age group, as current U.S. treatment options are to intravenous administration.