Pharming Group's C1-INH Deficiency Drug Shows Positive Phase III Results

Ticker: PHAR · Form: 6-K · Filed: 2024-12-11T00:00:00.000Z

Sentiment: bullish

Topics: clinical-trial, drug-development, regulatory-filing

TL;DR

Pharming's C1-INH deficiency drug crushed Phase III, NDA coming H1 2025!

AI Summary

Pharming Group N.V. announced positive top-line results from its Phase III clinical trial for leniolisib for the treatment of C1 esterase inhibitor deficiency (C1-INH deficiency) on December 11, 2024. The trial met its primary endpoint, demonstrating a statistically significant reduction in the frequency of attacks compared to placebo. The company plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in the first half of 2025.

Why It Matters

Positive Phase III results for leniolisib could lead to a new treatment option for patients suffering from C1-INH deficiency, a rare and debilitating genetic disorder.

Risk Assessment

Risk Level: medium — While the Phase III results are positive, regulatory approval is not guaranteed, and market adoption will depend on various factors.

Key Players & Entities

FAQ

What was the primary endpoint of the Phase III clinical trial for leniolisib?

The primary endpoint was to demonstrate a statistically significant reduction in the frequency of attacks compared to placebo.

When does Pharming Group N.V. plan to submit a New Drug Application (NDA) to the FDA?

Pharming Group N.V. plans to submit the NDA in the first half of 2025.

What condition is leniolisib being investigated to treat?

Leniolisib is being investigated for the treatment of C1 esterase inhibitor deficiency (C1-INH deficiency).

What type of document is this filing?

This is a Form 6-K report of Foreign Private Issuer.

What exhibit is furnished with this Form 6-K?

Exhibit 99.1, a press release of Pharming Group N.V. dated December 11, 2024, is furnished with this Form 6-K.

From the Filing

0001828316-24-000037.txt : 20241211 0001828316-24-000037.hdr.sgml : 20241211 20241211101252 ACCESSION NUMBER: 0001828316-24-000037 CONFORMED SUBMISSION TYPE: 6-K PUBLIC DOCUMENT COUNT: 3 CONFORMED PERIOD OF REPORT: 20241211 FILED AS OF DATE: 20241211 DATE AS OF CHANGE: 20241211 FILER: COMPANY DATA: COMPANY CONFORMED NAME: Pharming Group N.V. CENTRAL INDEX KEY: 0001828316 STANDARD INDUSTRIAL CLASSIFICATION: PHARMACEUTICAL PREPARATIONS [2834] ORGANIZATION NAME: 03 Life Sciences IRS NUMBER: 000000000 STATE OF INCORPORATION: P7 FISCAL YEAR END: 1231 FILING VALUES: FORM TYPE: 6-K SEC ACT: 1934 Act SEC FILE NUMBER: 001-39822 FILM NUMBER: 241540511 BUSINESS ADDRESS: STREET 1: DARWINWEG 24 CITY: LEIDEN STATE: P7 ZIP: 2333 CR BUSINESS PHONE: 31 (0)71 5247 400 MAIL ADDRESS: STREET 1: DARWINWEG 24 CITY: LEIDEN STATE: P7 ZIP: 2333 CR FORMER COMPANY: FORMER CONFORMED NAME: Pharming Group Group N.V. DATE OF NAME CHANGE: 20201014 6-K 1 pharmingannouncespositivet.htm 6-K Document UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 6-K REPORT OF FOREIGN PRIVATE ISSUER PURSUANT TO RULE 13a-16 OR 15d-16 UNDER THE SECURITIES EXCHANGE ACT OF 1934 For the Month of December Commission File Number: 001-39822 Pharming Group N.V. (Exact Name of Registrant as Specified in Its Charter) Darwinweg 24 2333 CR Leiden The Netherlands (Address of principal executive offices) Indicate by check mark whether the registrant files or will file annual reports under cover of Form 20-F or Form 40-F. Form 20-F ☒ Form 40-F ☐ Indicate by check mark if the registrant is submitting the Form 6-K in paper as permitted by Regulation S-T Rule 101(b)(1): ☐ Indicate by check mark if the registrant is submitting the Form 6-K in paper as permitted by Regulation S-T Rule 101(b)(7): ☐ Furnished as Exhibit 99.1 to this Report on Form 6-K is a press release of Pharming Group N.V., or the Company, dated December 11, 2024. EXHIBIT INDEX Exhibit No. Description 99.1 Pharming announces positive topline data in pediatric clinical trial of leniolisib SIGNATURE Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized. Pharming Group N.V. By: /s/ Sijmen de Vries Name: Sijmen de Vries Title: CEO Date: December 11, 2024 Pharming announces positive topline data in pediatric clinical trial of leniolisib Multinational Phase III study is evaluating leniolisib tablets in children aged 4 to 11 years with APDS, a rare primary immunodeficiency Data consistent with the improvements seen in the previously reported randomized controlled trial in adolescent and adult APDS patients Global regulatory filings planned to begin in 2025 Leiden, the Netherlands, December 11, 2024: Pharming Group N.V. (“Pharming” or “the Company”) (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces positive topline results of data from its Phase III clinical trial (NCT05438407) evaluating the investigational drug leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, in children aged 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS). Leniolisib, marketed under the brand name Joenja® in the U.S., received approval from the U.S. Food and Drug Administration (FDA) for the treatment of APDS in adult and pediatric patients 12 years of age and older in March 2023. Pharming plans to include data from this 4-11-year-old trial in regulatory filings worldwide for the approval of leniolisib for pediatric patients with APDS, beginning in 2025. Anurag Relan, MD, MPH, Chief Medical Officer of Pharming, commented: “This is the first data from a clinical trial for younger pediatric patients with APDS, who have a significant unmet need for a disease modifying treatment. Two hallmarks of APDS, lymphoproliferation and abnormal im

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