Clinical Trial News
FDA Approves First Gene Therapy, Vijuvek, for Rare Skin Disease Epidermolysis Bullosa
- The FDA has approved Vijuvek, a topical gene therapy developed by Krystal Biotech, as the first treatment of its kind for epidermolysis bullosa (EB).
- Clinical trials demonstrated Vijuvek's effectiveness in healing wounds and preventing new blisters in patients with EB, offering a significant improvement in their quality of life.
- The therapy, while promising, comes with a high annual cost of up to $500,000, raising concerns about accessibility and affordability for patients.
- Vijuvek offers hope for increased mobility and independence for individuals with EB, potentially transforming the lives of those affected by this debilitating condition.
Tvardi Therapeutics to Present Phase 1 Trial Data of STAT3 Inhibitor TTI-101 at ASCO 2023
Tvardi Therapeutics, Inc. is set to present clinical results from the Phase 1 study of TTI-101, a STAT3 inhibitor, at the ASCO 2023 annual meeting. The study focuses on the safety, tolerability, and clinical activity of TTI-101 monotherapy in patients with advanced solid tumors.
Highlighted Clinical Trials:
Tvardi Therapeutics, Incorporated
Posted 3/23/2023
Tvardi Therapeutics, Incorporated
Posted 1/9/2023
Tvardi Therapeutics, Incorporated
Posted 5/15/2023
BIAL R&D Doses First Patient in Phase 2 Trial of BIA 28-6156 for Parkinson's Disease with GBA1 Mutation
- BIAL R&D has dosed the first patient in its Phase 2 ACTIVATE study, evaluating BIA 28-6156 for Parkinson's disease patients with a GBA1 gene mutation.
- The ACTIVATE study is a multicenter, randomized, double-blind, placebo-controlled trial assessing the efficacy, safety, and tolerability of BIA 28-6156 at two fixed dose levels.
- BIA 28-6156 is a novel allosteric activator of beta-glucocerebrosidase (GCase), designed to enhance enzyme activity and potentially delay motor progression in GBA-PD patients.
- The trial will enroll approximately 237 genetically confirmed GBA-PD subjects across sites in North America and Europe, with results expected to inform future treatment strategies.
Highlighted Clinical Trials:
Bial R&D Investments, S.A.
Posted 3/31/2023
Vega Therapeutics' VGA039 Receives FDA Orphan Drug Designation for Von Willebrand Disease
- Vega Therapeutics' VGA039, a first-in-class antibody, has been granted Orphan Drug Designation by the FDA for treating von Willebrand disease (VWD).
- VGA039 modulates Protein S to enhance thrombin generation, addressing a core mechanism of clot formation in VWD.
- A Phase 1 clinical study (NCT05776069) is underway in the EU and US to assess VGA039's safety, tolerability, and pharmacokinetics in healthy volunteers and VWD patients.
- VGA039, designed for subcutaneous self-administration, aims to reduce the treatment burden associated with current VWD therapies.
Highlighted Clinical Trials:
Vega Therapeutics, Inc
Posted 3/16/2023
Valbiotis Announces Success of REVERSE-IT Clinical Study on TOTUM-63
Valbiotis SA has reported significant success in the REVERSE-IT Phase II/III clinical study on TOTUM-63, showing its efficacy in reducing fasting blood glucose in prediabetic and untreated type 2 diabetic patients. This achievement has triggered a CHF 4 million payment from Nestlé Health Science as part of their global strategic partnership.
Microbiome Modulation with SIM01 Shows Promise for Long COVID Treatment in Randomized Trial
- A randomized, triple-blind, placebo-controlled clinical trial by Chinese University of Hong Kong demonstrated that SIM01, an oral microencapsulated live bacteria formula, significantly improved long COVID symptoms compared to placebo.
- The RECOVERY study of 463 patients showed SIM01 effectively addressed digestive problems, fatigue, difficulty concentrating, memory loss, and general unwellness through restoration of gut microbiome diversity.
- Researchers identified this as the first clinical trial demonstrating gut microbiome modulation can improve neurological symptoms of long COVID, highlighting the importance of the gut-brain axis in treatment approaches.
EBR Systems' Wireless Cardiac Pacing System Shows Positive Clinical Trial Results, Paving Way for FDA Approval
- EBR Systems announced positive clinical trial results for its wireless cardiac pacing system, Wireless Stimulation Endocardial (WiSE) CRT System, marking a significant milestone.
- The trial results pave the way for potential FDA approval, offering a new treatment option for heart failure patients who do not respond to traditional pacemakers.
- The WiSE CRT system is designed to stimulate the heart's left ventricle wirelessly, addressing limitations of conventional cardiac resynchronization therapy (CRT) devices.
- Investors responded positively to the news, with shares of the ASX-listed medical technology firm surging following the announcement.
Study Highlights Delays in Confirmatory Studies for Accelerated FDA Approvals
A cross-sectional study reveals significant delays in the initiation and completion of confirmatory studies for drugs granted accelerated approval by the FDA, with noncancer products experiencing longer delays than cancer treatments.
Nexcella's NXC-201 CAR-T Therapy Shows 100% Response in Darzalex-Relapsed AL Amyloidosis
- Nexcella's NXC-201, a BCMA-targeted CAR-T cell therapy, demonstrated a 100% overall response rate in AL amyloidosis patients relapsed or refractory to Darzalex-based regimens.
- The Phase 1b/2a NEXICART-1 study showed a 63% complete response rate and a 75% organ response rate in treated patients with AL amyloidosis.
- The CAR-T therapy was well-tolerated, with no grade 4 cytokine release syndrome events reported, suggesting a favorable safety profile in this patient population.
- The best responder in the study had a duration of response of 16.5 months, with the response ongoing at the data cutoff, indicating sustained efficacy.
Highlighted Clinical Trials:
Hadassah Medical Organization
Posted 1/1/2021
FDA Approves Krystal Biotech's Vyjuvek, a Topical Gene Therapy for Dystrophic Epidermolysis Bullosa
- The FDA has approved Vyjuvek, Krystal Biotech's topical gene therapy, marking the first treatment of its kind for dystrophic epidermolysis bullosa.
- Vyjuvek is approved for patients aged six months and older with either recessive or dominant forms of dystrophic epidermolysis bullosa.
- Clinical trials showed Vyjuvek completely healed wounds in approximately 65% of patients, compared to 21.6% in the placebo group.
- Krystal Biotech anticipates Vyjuvek will be available in the U.S. in Q3 2023, with plans to provide access to all U.S. patients.