Clinical Trial News
CRISPR Gene Editing Shows Promise in Treating Rare Form of Blindness
- A CRISPR-based gene editing therapy, EDIT-101, has shown promising results in treating Leber Congenital Amaurosis (LCA) Type 10, a rare genetic form of blindness.
- In the BRILLIANCE trial, 79% of participants experienced measurable improvements in vision after receiving the experimental treatment.
- The study demonstrated that EDIT-101 was safe and well-tolerated, with no dose-limiting toxicities reported among the 14 participants.
- These findings offer hope for individuals with LCA Type 10, for which there is currently no FDA-approved treatment.
Novartis Acquires Mariana Oncology for $1 Billion to Strengthen Radioligand Therapy Pipeline
- Novartis announced a $1 billion acquisition of Mariana Oncology with up to $750 million in milestone payments to expand its radioligand therapy capabilities.
- The deal provides access to Mariana's lead candidate MC-339, an actinium-based radioligand therapy targeting small cell lung cancer currently in preclinical development.
- Mariana brings specialized radiopharmaceutical manufacturing capabilities and an oncology radio-conjugation platform using both alpha- and beta-emitting radionuclides.
- The acquisition strengthens Novartis' position in the competitive radioligand therapy space, following similar billion-dollar deals by AstraZeneca, Bristol Myers Squibb, and Eli Lilly.
Repare Therapeutics Advances Oncology Pipeline with Multiple Clinical Trials
- Repare Therapeutics' MYTHIC trial shows reduced Grade 3 anemia with lunresertib and camonsertib combination, with FDA agreement on RP2D.
- Phase 2 TRESR expansion initiated to evaluate camonsertib monotherapy in NSCLC patients with ATM mutations, with initial data expected in 2025.
- First patient dosed in Phase 1 LIONS trial for PLK4 inhibitor RP-1664, expanding Repare's portfolio of precision oncology therapeutics.
- A Phase 1 trial of RP-3467, a Polθ ATPase inhibitor, is expected to begin in the second half of 2024, further diversifying Repare's pipeline.
D3S-001 Shows Superior KRAS G12C Inhibition with Rapid Target Engagement and Promising Clinical Activity
- D3S-001 demonstrates substantially improved covalent potency with a kinact/KI of 1.43 × 10⁶ mol/L⁻¹ second⁻¹, representing one to two orders of magnitude improvement over approved KRAS G12C inhibitors sotorasib and adagrasib.
- The compound achieves rapid target engagement with a t1/2 of 5.8 minutes compared to 44 and 34 minutes for sotorasib and adagrasib respectively, and uniquely maintains efficacy even in the presence of growth factor stimulation.
- Early clinical data from a first-in-human phase 1 trial shows durable RECIST responses across all dose cohorts from 50-900 mg daily, with two NSCLC patients achieving sustained partial responses lasting over 8-16 months.
- D3S-001 exhibits favorable CNS penetration properties and demonstrates robust antitumor activity in brain metastasis models and acquired resistance xenografts where sotorasib and adagrasib showed limited efficacy.
Highlighted Clinical Trials:
Amgen
Posted 8/27/2018
Mirati Therapeutics Inc.
Posted 1/15/2019
Amgen
Posted 4/19/2022
Eli Lilly and Company
Posted 7/19/2021
Novartis Pharmaceuticals
Posted 2/24/2021
Mirati Therapeutics Inc.
Posted 12/2/2022
Deciphera Pharmaceuticals, LLC
Posted 6/15/2021
Ikena Oncology
Posted 1/7/2022
Mirati Therapeutics Inc.
Posted 6/24/2021
Vivace Therapeutics, Inc
Posted 3/24/2021
NYU Langone Health
Posted 8/1/2023
Mirati Therapeutics Inc.
Posted 8/7/2023
D3 Bio (Wuxi) Co., Ltd
Posted 7/10/2023
Mirati Therapeutics Inc.
Posted 12/2/2020
Erasca, Inc.
Posted 9/2/2021
ROCKIF Trial: Re-sensitization of Carboplatin-resistant Ovarian Cancer With Kinase Inhibition of FAK
Michael McHale
Posted 2/6/2018
Merck Sharp & Dohme LLC
Posted 12/17/2021
Novartis Pharmaceuticals
Posted 10/24/2022
Verastem, Inc.
Posted 4/12/2022
M.D. Anderson Cancer Center
Posted 12/6/2023
The University of Texas Health Science Center at San Antonio
Posted 6/4/2019
Mirati Therapeutics Inc.
Posted 3/6/2023
Kura Oncology, Inc.
Posted 10/18/2023
Jiangsu HengRui Medicine Co., Ltd.
Posted 9/15/2022
Amgen
Posted 12/17/2019
Revolution Medicines, Inc.
Posted 11/14/2023
Genentech, Inc.
Posted 7/29/2020
Innovent Biologics (Suzhou) Co. Ltd.
Posted 8/18/2022
M.D. Anderson Cancer Center
Posted 1/30/2024
Astellas Pharma Inc
Posted 6/8/2022
Allist Pharmaceuticals, Inc.
Posted 2/17/2022
Revolution Medicines, Inc.
Posted 9/7/2023
Boehringer Ingelheim
Posted 9/9/2021
Mirati Therapeutics Inc.
Posted 7/28/2021
Mirati Therapeutics Inc.
Posted 1/20/2022
D3 Bio (Wuxi) Co., Ltd
Posted 8/3/2022
Verastem, Inc.
Posted 12/31/2020
InventisBio Co., Ltd
Posted 10/2/2020
Denali Therapeutics Announces Positive Clinical Updates and Financial Results for Q1 2024
- Denali Therapeutics reported positive two-year clinical data for tividenofusp alfa in MPS II, showing sustained normalization of heparan sulfate in CSF and improvements in clinical outcomes.
- The company initiated a Phase 1/2 clinical trial for DNL126, an enzyme replacement therapy for children with MPS IIIA, with biomarker and safety data expected by the end of 2024.
- Enrollment is complete in the Phase 2/3 HEALEY ALS Platform Trial for DNL343, an eIF2B activator being developed for the treatment of amyotrophic lateral sclerosis (ALS).
- Denali completed a private investment in public equity (PIPE) financing with gross proceeds of $500 million and divested its preclinical small molecule portfolio.
Recce Pharmaceuticals Secures Final China Patent for Synthetic Anti-Infectives, Completing Global Protection Until 2035
- Recce Pharmaceuticals received patent approval from China's National Intellectual Property Administration for its "Copolymer and Method for Treatment of Bacterial Infection" covering RECCE 327 and RECCE 529 anti-infectives.
- The China patent represents the final piece of Recce's global patent portfolio, providing comprehensive protection across all major pharmaceutical markets until 2035.
- This milestone positions Recce to access China's $140 billion pharmaceutical market, the world's second-largest, which continues growing at approximately 3.2% annually.
- The patent covers synthetic polymer anti-infectives designed to combat antibiotic-resistant superbugs and viral pathogens through novel multi-layered mechanisms of action.
Cybin's CYB003 Receives FDA Breakthrough Therapy Designation for Major Depressive Disorder
- Cybin Inc. has obtained FDA Breakthrough Therapy Designation for CYB003, a psychedelic-based therapy targeting Major Depressive Disorder.
- The company secured $150 million in funding to support the advancement of its psychedelic-based therapies for mental health.
- Cybin plans to initiate a Phase 3 study for CYB003 in mid-2024 and expects Phase 2 results for CYB004 in late 2024, which targets Generalized Anxiety Disorder.
Lyra Therapeutics' Phase 3 ENLIGHTEN 1 Trial for Chronic Rhinosinusitis Treatment Fails to Meet Primary Endpoint
- Lyra Therapeutics announced that its Phase 3 ENLIGHTEN 1 trial evaluating LYR-210, a bioabsorbable sinonasal implant for chronic rhinosinusitis, failed to meet its primary endpoint of demonstrating statistically significant improvement in cardinal symptoms compared to sham control at 24 weeks.
- The trial enrolled 190 patients with chronic rhinosinusitis who had failed medical management and had not undergone prior ethmoid sinus surgery, with LYR-210 showing a mean improvement of 2.13 points in the composite symptom score versus 2.06 points for sham control.
- Despite the setback, LYR-210 was generally well tolerated with no product-related serious adverse events, and the company continues its second Phase 3 trial ENLIGHTEN 2 while planning workforce reductions to preserve cash.
- The failed trial represents a significant disappointment for addressing the estimated four million chronic rhinosinusitis patients in the United States who fail medical management annually and require further intervention.
Mediar Therapeutics Advances Fibrosis Treatment with Phase 1 Trial of MTX-463
Mediar Therapeutics has initiated a Phase 1 clinical trial for MTX-463, a first-in-class antibody targeting WISP-1-mediated fibrotic signaling, following FDA clearance. The trial aims to assess the safety, tolerability, and pharmacokinetics of MTX-463 in healthy participants. Additionally, Mediar has formed a Clinical Advisory Board of global fibrosis experts to guide the development of its fibrosis treatment portfolio, including a second antibody program, MTX-474, expected to enter Phase 1 trials in Q3 2024.
Highlighted Clinical Trials:
Mediar Therapeutics
Posted 4/15/2024
Systematic Review of Phase II and III Clinical Trials for Relapsing Multiple Sclerosis
A comprehensive systematic review of phase II and III clinical trials for relapsing multiple sclerosis (RMS) has been conducted, focusing on trial designs and primary endpoints. The review, which analyzed 60 trials from three major trial registration systems, highlights the dominance of randomized controlled parallel-arms trials and discusses the most common primary endpoints across different trial phases and patient groups. The findings suggest a need for revisions in regulatory guidelines and expert recommendations to better align with current clinical trial practices.
Highlighted Clinical Trials:
Oslo University Hospital
Posted 10/16/2019
Beijing InnoCare Pharma Tech Co., Ltd.
Posted 3/1/2021
Cinnagen
Posted 12/20/2017
EMD Serono Research & Development Institute, Inc.
Posted 3/7/2017
Haukeland University Hospital
Posted 11/2/2020
Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany
Posted 7/2/2020
Immunic AG
Posted 1/28/2019
GeNeuro Innovation SAS
Posted 6/17/2020
Sanofi
Posted 3/29/2019
Immunic AG
Posted 11/18/2021
Polpharma Biologics S.A.
Posted 10/1/2019
GeNeuro Innovation SAS
Posted 8/27/2021
Imcyse SA
Posted 4/12/2022
Hoffmann-La Roche
Posted 3/17/2021
EMD Serono Research & Development Institute, Inc.
Posted 9/10/2019
Biogen
Posted 11/15/2017
Novartis Pharmaceuticals
Posted 10/5/2021
Haukeland University Hospital
Posted 3/21/2018
Sanofi
Posted 6/11/2020
Sanofi
Posted 6/30/2020
Biogen
Posted 11/27/2018
TG Therapeutics, Inc.
Posted 5/27/2016
AbbVie
Posted 12/11/2018
Biogen
Posted 6/30/2021
Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany
Posted 6/12/2020
GeNeuro SA
Posted 6/6/2017
Cinnagen
Posted 8/19/2019
Rigshospitalet, Denmark
Posted 4/28/2021
Novartis Pharmaceuticals
Posted 3/15/2018
EMD Serono Research & Development Institute, Inc.
Posted 8/26/2019
Hoffmann-La Roche
Posted 3/24/2021
TG Therapeutics, Inc.
Posted 8/25/2017
Novartis Pharmaceuticals
Posted 12/16/2021
Hoffmann-La Roche
Posted 2/4/2022
Sanofi
Posted 6/7/2021
Actelion
Posted 6/4/2015
Karolinska Institutet
Posted 7/4/2018
Immunic AG
Posted 1/12/2022
Novartis Pharmaceuticals
Posted 12/13/2021
Hoffmann-La Roche
Posted 11/26/2020
Bosnalijek D.D
Posted 3/26/2019
Sanofi
Posted 9/23/2019
GeNeuro SA
Posted 4/1/2016
TG Therapeutics, Inc.
Posted 6/1/2017
Mapi Pharma Ltd.
Posted 9/19/2019
Northwestern University
Posted 11/8/2017
Biocad
Posted 6/7/2019
TG Therapeutics, Inc.
Posted 9/19/2017
National Institute of Allergy and Infectious Diseases (NIAID)
Posted 12/19/2019