Kallidinogenase

FDA Approves Five Groundbreaking Treatments for Ultra-Rare Diseases in Recent Months

3 months ago

• The FDA has approved novel therapies for Duchenne Muscular Dystrophy, including ITF Therapeutics' Duvyzat (givinostat), which targets histone deacetylases to potentially slow muscle deterioration. • Breakthrough gene therapy Lenmeldy received approval for Metachromatic Leukodystrophy, offering the first targeted treatment option with significant survival benefits for pediatric patients. • Multiple rare diseases saw first-ever treatments, including Xolremdi for WHIM syndrome, dual approvals for Niemann-Pick Disease Type C, and Ctexli for Cerebrotendinous Xanthomatosis.

Astria Therapeutics Advances Navenibart for HAE with Phase 3 Trial

4 months ago

• Astria Therapeutics is initiating the ALPHA-ORBIT Phase 3 trial to evaluate navenibart for hereditary angioedema (HAE) with flexible dosing. • The trial will assess the efficacy and safety of navenibart administered every 3 or 6 months over a 6-month period in HAE patients. • Positive Phase 1b/2 results showed a 90-95% reduction in mean monthly attack rate and up to 67% attack-free rate with navenibart. • ALPHA-ORBIT's design incorporates feedback from regulators and aims for global registration, potentially making navenibart a first-choice HAE therapy.

Major Breakthroughs in HAE Treatment: 2024 Highlights Gene Editing and Novel Therapeutics

5 months ago

2024 marked significant advances in hereditary angioedema (HAE) treatment, with Intellia's gene-editing therapy NTLA-2002 showing a remarkable 95% reduction in monthly attacks. The year also saw important developments in multiple therapeutic candidates, including donidalorsen and garadacimab, while established treatments like Takhzyro demonstrated continued efficacy in adolescent populations.

CSL's Andembry (Garadacimab) Approved in Europe and UK for Hereditary Angioedema

5 months ago

• The European Commission and the MHRA have approved CSL's Andembry (garadacimab) for preventing hereditary angioedema (HAE) attacks in patients aged 12 and older. • Andembry, a Factor XIIa-inhibitory monoclonal antibody, offers a novel approach by targeting the initiation of the angioedema cascade with a once-monthly subcutaneous injection. • Clinical trials, including the VANGUARD trial, demonstrated that Andembry significantly reduced HAE attack rates and achieved attack-free status in a substantial portion of patients. • The approvals mark a significant advancement in HAE management, providing patients with a convenient, long-term control option, and are based on positive Phase 3 trial results.

Navenibart Shows Strong Efficacy in Hereditary Angioedema Trial

5 months ago

• Astria Therapeutics' navenibart demonstrated a 90-95% reduction in mean monthly attack rates in HAE patients over six months. • The ALPHA-STAR Phase 1b/2 trial supports quarterly or bi-annual dosing for navenibart, with a favorable safety profile observed. • A 67% attack-free rate was achieved over six months in two cohorts, highlighting the drug's potential as a preventative treatment. • Astria plans to initiate Phase 3 development in Q1 2025, pending regulatory feedback, with topline results expected by year-end 2026.

Astria Therapeutics Plans Phase 3 Trial of Navenibart for Hereditary Angioedema

6 months ago

• Astria Therapeutics is set to launch a Phase 3 clinical trial for navenibart in early 2025, evaluating different dosing schedules for hereditary angioedema (HAE). • Navenibart, a kallikrein inhibitor, aims to reduce bradykinin production and prevent swelling attacks in HAE patients, with dosing options every three or six months. • Positive interim results from the Phase 1b/2 ALPHA-STAR trial showed over 90% reduction in swelling attacks, with final data expected by the end of 2024. • Navenibart has received orphan drug status from the FDA and orphan medicinal product designation in Europe, providing market exclusivity incentives.

NTLA-2002 Shows Promise as Functional Cure for Hereditary Angioedema in Phase 2 Trial

7 months ago

• Phase 2 trial results show NTLA-2002 significantly reduces swelling attacks in hereditary angioedema (HAE) patients, with up to 80% attack reduction at the 50 mg dose. • A substantial portion of patients receiving NTLA-2002 experienced complete attack-free periods, with some remaining attack-free through the latest assessments. • NTLA-2002 was generally well-tolerated, with no serious side effects reported, suggesting a favorable safety profile for this gene-editing therapy. • Intellia Therapeutics has initiated a Phase 3 trial based on these positive results, potentially redefining the treatment paradigm for HAE.

CRISPR Gene Editing Shows Promise as Single-Dose Treatment for Hereditary Angioedema

7 months ago

• A single dose of CRISPR-Cas9 gene editing therapy, NTLA-2002, significantly reduces angioedema attacks in patients with hereditary angioedema (HAE). • The Phase 2 study demonstrated a substantial and sustained reduction in kallikrein levels, a key mediator of swelling in HAE patients. • The international trial involved multiple national excellence centers and showed promising results, potentially offering a functional cure for HAE. • NTLA-2002 is advancing to Phase 3 clinical trials, building on the positive outcomes from Phase 1 and 2 studies.

Intellia Therapeutics Initiates Phase 3 Trial of NTLA-2002 for Hereditary Angioedema

8 months ago

• Intellia Therapeutics has dosed the first patient in its Phase 3 HAELO trial evaluating NTLA-2002 for hereditary angioedema (HAE). • The HAELO trial is a global, randomized, double-blind, placebo-controlled study involving 60 adults with Type I or Type II HAE. • NTLA-2002, a single-dose CRISPR-based therapy, targets the _KLKB1_ gene to reduce plasma kallikrein activity and prevent HAE attacks. • Intellia anticipates completing enrollment in the second half of 2025 and plans for a potential U.S. launch in 2027, pending regulatory approval.

Kallidinogenase

Drug Development Updates