PHARMING GROUP NV

Pharming Group Initiates Phase II Trial of Leniolisib for CVID with Immune Dysregulation

2 months ago

• Pharming Group has dosed the first patient in a Phase II clinical trial evaluating leniolisib for common variable immunodeficiency (CVID) with immune dysregulation, representing a significant expansion beyond its approved APDS indication. • The multi-center trial will enroll approximately 20 patients aged 12 and older across sites in the US, UK, and EU, targeting a condition with an 11-fold higher mortality rate compared to CVID patients with infectious manifestations alone. • CVID with immune dysregulation affects approximately 39 patients per million globally and currently has no approved therapies, making this trial particularly significant for addressing a substantial unmet medical need.

PharmaMar Advances Lurbi Eadine Development with Japanese Licensing Talks and European Submission Plans

3 months ago

• PharmaMar is in advanced licensing discussions for Lurbi Eadine in Japan, with negotiations expected to conclude in 2025, indicating expansion into a key Asian market. • The company plans to submit Lurbi Eadine's first-line therapy dossier to EMA in first half of 2024, with potential approval timeline of 7-12 months depending on accelerated review status. • PharmaMar's partner Rua Pharma received approval in China in December 2024, with product launch expected in first half of 2025, marking significant market expansion.

Major Advances in Angioedema Pipeline: Intellia's Gene Therapy Enters Phase 3 as Multiple Companies Race for Novel Treatments

3 months ago

• Intellia Therapeutics has initiated Phase 3 trials for NTLA-2002, a groundbreaking CRISPR-based gene therapy for hereditary angioedema, with potential U.S. launch targeted for 2027. • KalVista Pharmaceuticals' sebetralstat receives Orphan Drug Designation in Japan, marking progress toward the first oral on-demand treatment for HAE attacks. • The global angioedema pipeline includes 20+ companies developing innovative therapies, with several promising candidates in late-stage development from companies like ADARx, BioMarin, and Astria Therapeutics.

Pharming Group to Acquire Abliva in $66.1 Million Deal, Bolstering Mitochondrial Disease Pipeline

5 months ago

• Pharming Group N.V. has announced a public cash offer to acquire Abliva AB for approximately US$66.1 million, strengthening its pipeline. • Abliva's lead product, KL1333, is in a pivotal clinical trial for primary mitochondrial disease (PMD) driven by mitochondrial DNA mutations. • KL1333 has shown positive clinical effects and has received Fast Track and Orphan Drug designations, targeting over 30,000 patients. • The acquisition, funded by Pharming's existing cash, aims for a U.S. launch of KL1333 in 2028, pending regulatory approvals.

MHRA Approves Joenja (leniolisib) as First Treatment for Rare Immune Disease APDS

8 months ago

• The MHRA has approved Joenja (leniolisib) as the first medicine for Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS) in the UK. • APDS is a rare, inherited immune disorder affecting approximately 30-40 people in the UK, characterized by recurrent infections and abnormal immune function. • Leniolisib demonstrated a statistically significant reduction in lymph node size and normalization of immunophenotype in a 12-week placebo-controlled trial. • The approval was expedited through the International Recognition Procedure (IRP), leveraging prior FDA approval to provide quicker access to UK patients.