Clinical Trial News

Intellia's CRISPR Gene Therapy Shows Potential as Functional Cure for Hereditary Angioedema in Long-Term Study

Rare Disease Gene Editing Orphan Drug

a year ago

Intellia Therapeutics' NTLA-2002, an in vivo CRISPR-based gene editing therapy, demonstrated a 98% mean reduction in monthly hereditary angioedema attack rates across all patients, with follow-up extending beyond two years.
Eight of ten patients remained completely attack-free following the initial 16-week observation period, with the longest attack-free duration reaching over 26 months and continuing.
The single-dose treatment showed a favorable safety profile across all dose levels, with no serious adverse events reported, positioning NTLA-2002 as a potential functional cure for this rare genetic disease.

NTLA-2002 in Adults With Hereditary Angioedema (HAE)

NCT05120830Active, Not RecruitingPhase 1

Intellia Therapeutics

Posted 12/10/2021

ImPact Biotech Reports 77% Complete Response Rate for Padeliporfin VTP in Low-Grade Upper Tract Urothelial Cancer

a year ago

ImPact Biotech's Phase 3 ENLIGHTED trial demonstrated a 77% complete response rate (10/13 patients) for Padeliporfin VTP therapy in low-grade upper tract urothelial cancer patients who completed the induction treatment phase.
The vascular targeted photodynamic therapy showed a favorable safety profile with primarily Grade 1-2 adverse events that resolved within two to seven days, and no Grade 4 or 5 serious adverse events.
The minimally invasive treatment offers a potential organ-sparing alternative to surgery for UTUC patients, with enrollment completion expected by end of 2024 and additional interim analysis planned for second half 2024.

Bio-Thera's BAT8006 Shows Promising 37% Response Rate in Platinum-Resistant Ovarian Cancer Phase I Trial

FDA Approval

a year ago

Bio-Thera Solutions reported that BAT8006, a folate receptor-α antibody drug conjugate, achieved a 37% overall response rate in 54 patients with platinum-resistant ovarian cancer regardless of FR-α expression levels.
The Phase I study demonstrated favorable safety with manageable toxicity, no interstitial lung disease or notable ocular toxicity, and median progression-free survival of 7.47 months.
BAT8006 utilizes a unique bystander effect mechanism that allows it to eliminate neighboring cancer cells, potentially addressing tumor heterogeneity challenges.
The FDA has approved Bio-Thera to proceed with a Phase II clinical trial for BAT8006, expanding development beyond the ongoing Chinese Phase I study.

Caribou Biosciences Reports Positive Clinical Data from CB-010 ANTLER Phase 1 Trial

a year ago

Caribou Biosciences has presented encouraging clinical data from its ANTLER Phase 1 trial of CB-010, an allogeneic CAR-T cell therapy, showing potential to match the efficacy and safety of approved autologous CAR-T cell therapies. The data, presented at the 2024 ASCO Annual Meeting, highlights improved progression-free survival in patients with partial HLA matching and outlines plans for further trials.

CRISPR-Edited Allogeneic Anti-CD19 CAR-T Cell Therapy for Relapsed/Refractory B Cell Non-Hodgkin Lymphoma (ANTLER)

NCT04637763RecruitingPhase 1

Caribou Biosciences, Inc.

Posted 5/26/2021

Sandoz Launches First Ustekinumab Biosimilar Autoinjector in Europe with Pyzchiva

Monoclonal Antibody

a year ago

Sandoz has launched Pyzchiva (ustekinumab) as the first ustekinumab biosimilar autoinjector commercially available in Europe, developed by Samsung Bioepis.
The biosimilar is approved to treat multiple chronic inflammatory conditions including plaque psoriasis, psoriatic arthritis, Crohn's disease, and pediatric plaque psoriasis in patients aged six and older.
The autoinjector features automatic dosing, reduced injection pain, compact design, and flexible storage options to potentially improve treatment adherence for chronic inflammatory diseases.
Pyzchiva has launched in 23 European markets with the autoinjector now available in Spain, representing a strategic milestone for Sandoz's immunology biosimilar portfolio.

Camrelizumab Plus Rivoceranib Shows Sustained Survival Benefit in Unresectable HCC

a year ago

Final analysis of the phase 3 CARES-310 trial reveals that first-line treatment with camrelizumab plus rivoceranib maintains a clinically meaningful survival improvement in patients with advanced, unresectable hepatocellular carcinoma (HCC).
The combination therapy demonstrated a median overall survival (OS) of 23.8 months compared to 15.2 months with sorafenib alone (HR, 0.64; 95% CI, 0.52-0.79; 1-sided P <.0001) at a median follow-up of 22.1 months.
Camrelizumab plus rivoceranib also showed improved progression-free survival (PFS) with a median of 5.6 months versus 3.7 months for sorafenib (HR, 0.54; 95% CI, 0.44-0.67; P <.0001).
The ORR for camrelizumab plus rivoceranib was 26.8% (95% CI, 21.7%-32.5%) compared with 5.9% (95% CI, 3.4%-9.4%) for sorafenib, with a manageable safety profile.

A Study to Evaluate SHR-1210 in Combination With Apatinib as First-Line Therapy in Patients With Advanced HCC

NCT03764293CompletedPhase 3

Jiangsu HengRui Medicine Co., Ltd.

Posted 6/10/2019

Chime & Mabgeek Achieve Phase III Milestone with MG-K10

a year ago

Chime Biologics and Mabgeek have reached a significant milestone in their collaboration by completing the Process Performance Qualification (PPQ) for MG-K10, a humanised anti-IL-4Rα monoclonal antibody, paving the way for Phase III clinical trials targeting Th2-mediated inflammatory conditions.

Ultra-Sensitive Blood Test Predicts Breast Cancer Recurrence Up to 41 Months Before Clinical Symptoms

a year ago

A new ultra-sensitive liquid biopsy using whole genome sequencing can detect breast cancer recurrence an average of 15 months before clinical symptoms appear, with the longest lead time reaching 41 months.
The test successfully identified all 11 patients who relapsed during a five-year trial by detecting circulating tumor DNA (ctDNA) in blood samples, with no false negatives among 60 patients who remained cancer-free.
This breakthrough technology analyzes up to 1,800 mutations compared to conventional tests that examine only 16-50 mutations, making it significantly more sensitive for early detection.
The findings could enable earlier intervention strategies for high-risk breast cancer patients, potentially improving treatment outcomes before cancer spreads to other parts of the body.

Subcutaneous Amivantamab Demonstrates Superior Outcomes Over IV Administration in EGFR-Mutated NSCLC

a year ago

The phase 3 PALOMA-3 trial showed subcutaneous amivantamab was noninferior to intravenous administration based on pharmacokinetics and objective response rates in patients with refractory EGFR-mutated advanced NSCLC.
Subcutaneous delivery significantly improved overall survival with a hazard ratio of 0.62 and reduced infusion-related reactions five-fold compared to IV administration.
Treatment administration time was dramatically reduced from 5 hours to less than 5 minutes with subcutaneous delivery, with 85% of patients rating it as convenient or very convenient.
Prophylactic anticoagulation reduced venous thromboembolism rates from 21% to 10% in patients receiving amivantamab plus lazertinib combination therapy.