Drug Discovery World

MAY 15, 2024

Dr Catherine Beech , CEO at Exonate and Dr Pete Adamson , CSO at Breye Therapeutics discuss the need for better therapies to treat diabetic retinal disease and current progress in the field. Despite affecting such a large subset of diabetic patients, treatment options are limited to repeated, invasive intravitreal injections to the eye.

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Drug Discovery World

DECEMBER 11, 2023

MC2 Therapeutics has completed enrolment in its Phase II MC2-25 Ph2 chronic kidney disease-associated pruritus trial, with topline results expected in Q2 2024. MC2-25 CKD is a first-in-class drug candidate and could represent a potential breakthrough in the understanding and treatment of urea associated skin diseases.

Disease 130

Disease Treatment Trials Drugs 130

Drug Discovery World

JANUARY 5, 2024

Kyverna Therapeutics has revealed plans to progress to Phase II trials of KYV-101 for multiple sclerosis (MS) following a green light from the US Food and Drug Administration (FDA). KYV-101 is an autologous, fully human CD19 chimeric antigen receptor (CAR) T-cell product candidate for use in B cell-driven autoimmune diseases.

Therapies 312

Therapies Treatment Protein Expression Trials 312

Drug Discovery World

SEPTEMBER 4, 2024

The first patient has been treated in a Phase II trial of EVO756 in adults with chronic inducible urticaria (CIndU). Evommune’s pre-clinical data demonstrates that by blocking activation of MRGPRX2 via all disease relevant ligand categories, EVO756 can prevent the degranulation of mast cells.

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Clinical Trials Trials Small Molecule Clinical Development 130

Drug Discovery World

MARCH 15, 2024

Biotechnology company Allyx Therapeutics has reported positive Phase Ib clinical data for its lead compound, ALX-001, a first-in-class, synapse-targeted, disease-modifying oral therapy in development for neurodegenerative diseases. The findings were presented at the AD/PD 2024 Conference in Lisbon.

Disease 130

Disease Trials Small Molecule Pharmacokinetics 130

Drug Discovery World

DECEMBER 21, 2023

Insilico Medicine has initiated the first-in-human study for ISM5411, a poly hydroxylase domain (PHD) 1/2 inhibitor for the treatment of inflammatory bowel disease (IBD) designed by its generative AI platform. We believe this new treatment – which is potentially first-in-class – could offer a promising alternative.”

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Drug Discovery World

NOVEMBER 29, 2023

At sites in the US, Japan, and the EU, the clinical trial will evaluate the safety, tolerability, and efficacy of leniolisib in 15 children, one to six years of age, who have a confirmed APDS diagnosis. The post Trials investigate targeted APDS treatment in a paediatric setting appeared first on Drug Discovery World (DDW).

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Drug Discovery World

AUGUST 20, 2024

The US Food and Drug Administration (FDA) has approved axatilimab-csfr (Niktimvo), a colony stimulating factor-1 receptor-blocking antibody, for the treatment of chronic graft-versus-host disease (cGVHD).

FDA Approval 147

FDA Approval FDA Disease Therapies 147

Drug Discovery World

OCTOBER 11, 2023

Collaborators Boehringer Ingelheim and CDR-Life have commenced a Phase I evaluation of BI 771716, their antibody fragment-based treatment developed to preserve the vision of people with geographic atrophy (GA). GA is an irreversible retinal disease that occurs in people with late-stage dry age-related macular degeneration (AMD).

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Clinical Trials Treatment Trials FDA Approval 130

Drug Discovery World

JUNE 23, 2023

PTC Therapeutics has shared interim data from the 12-week portion of the PIVOT-HD Phase II study of PTC518 in Huntington’s disease (HD) patients. PTC518 treatment was also demonstrated to be well tolerated, with no treatment-related serious adverse events, no reports of peripheral neuropathy or dose-limiting toxicities.

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Disease Trials Treatment Drugs 130

Drug Discovery World

JUNE 21, 2023

Citius Pharmaceuticals has revealed positive results from its Phase IIb Study of Halo-Lido (CITI-002) for the treatment of haemorrhoids. Following the 21 st Century Cures Act, higher emphasis is placed on using Patient Reported Outcome (PRO) instruments in clinical trials.

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Treatment Trials Clinical Trials Clinical Development 130

Drug Discovery World

AUGUST 8, 2024

The first cohort of participants has been dosed in a Phase I trial MTX-474, a human IgG1 antibody designed to neutralise EphrinB2 signalling to treat fibrosis. WISP1 is a secreted matricellular protein shown to have a relevant role in fibrosis progression, measurable in human blood, and correlates with disease severity.

Treatment 147

Treatment Pharmacokinetics Trials Disease 147

Drug Discovery World

JULY 5, 2024

Amgen has reported positive topline results from its randomised, double-blind, multicentre, placebo-controlled Phase III clinical trial evaluating the efficacy and safety of its treatment for Immunoglobulin G4-related disease (IgG4-RD).

Treatment 147

Treatment Medical Schools Trials Clinical Trials 147

Drug Discovery World

JULY 26, 2023

ARMGO Pharma has enrolled the first patient in a Phase II proof-of-concept clinical trial using its Rycal ARM210 (also known as S48168), for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT). CPVT is a rare genetic heart disease causing arrhythmia.

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Disease Trials Clinical Trials Drugs 130

Drug Discovery World

JANUARY 30, 2023

The data from MOVE have helped us to understand the potential for treatments that reduce HO progression to be used in the management of FOP,” said Dr Genevieve Baujat, Clinical Geneticist Consultant at Necker-Enfants Malades Hospital, Paris, France.

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Treatment Disease Clinical Trials Trials 130

Drug Discovery World

FEBRUARY 16, 2023

Disease progression X-ALD male patients (paediatric and adult) develop cerebral lesions that can evolve to progressive cerebral ALD (cALD), which is devastating and leads to death within two to four years. Treatment with leriglitazone also significantly reduced plasma levels of MMP-9, a marker of blood-brain barrier integrity.

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Treatment Disease Clinical Trials International 130

Drug Discovery World

JUNE 28, 2024

A new Phase II trial, REGENERATE-PD, will study AB-1005, an adeno-associated virus 2 (AAV2) glial cell line-derived neurotrophic factor (GDNF) gene therapy for the treatment of moderate-stage Parkinson’s disease. The trial will include an estimated 87 subjects with study sites located in the US, UK and Europe.

Therapies 147

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Drug Discovery World

AUGUST 28, 2024

It is approved for the first-line treatment of adult patients with unresectable or metastatic cancer, who are eligible for platinum-containing chemotherapy. I look forward to seeing the treatment combination implemented as a first-line regimen in the clinical setting.” months compared to 16.1 The median PFS of 12.5

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Drug Discovery World

OCTOBER 6, 2022

A new Alzheimer’s disease (AD) treatment has been shown to reduce clinical decline in mild cognitive impairment (MCI) due to AD and mild AD with confirmed presence of amyloid pathology in the brain. . The recruitment strategy for the Clarity AD clinical trial ensured greater inclusion of ethnic and racial populations in the US.

Trials 130

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Drug Discovery World

JULY 19, 2023

Eli Lilly has announced positive results of the TRAILBLAZER-ALZ 2 Phase III study showing that donanemab significantly slowed cognitive and functional decline in people with early symptomatic Alzheimer’s disease.

Disease 130

Disease Trials Clinical Trials Therapies 130

Drug Discovery World

SEPTEMBER 11, 2024

A gene therapy developed by University of Florida scientists restored useful vision to most patients with rare condition Leber congenital amaurosis type I (LCA1) in a small trial. The trial also tested the safety profile of the treatment. Side effects were largely limited to minor surgical complications.

Therapies 147

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Drug Discovery World

JULY 12, 2024

Scientists at the University of Oxford in the UK have launched a new clinical trial to test a vaccine to protect against “devastating” Marburg virus. This is the first-in-human trial of the ChAdOx1 Marburg vaccine, which has been developed and manufactured by researchers at the University of Oxford.

Virus 147

Virus Vaccine Trials Clinical Trials 147

Drug Discovery World

FEBRUARY 15, 2023

With this financing, we expect to reach several key clinical milestones across multiple disease areas with unmet needs.” Cerevance has a growing pipeline of clinical and preclinical programs developed using the company’s NETSseq platform to discover and validate novel therapeutic targets in some of the most challenging neurological diseases.

Disease 130

Disease Trials Clinical Trials Therapies 130

Drug Discovery World

JULY 30, 2024

A new partnership between NHS Scotland, AstraZeneca and the Universities of Dundee and Glasgow hopes to accelerate research into treating chronic kidney disease. The partners will work together on new medicines to slow the progression of chronic kidney disease, reducing the risk that patients might need dialysis or kidney transplantation.

Disease 130

Disease Research Government Clinical Trials 130

Drug Discovery World

JULY 6, 2023

The first patient has been dosed in the clinical Phase I evaluation of the Sema3A monoclonal antibody, part of the Evotec-Bayer multi-target research collaboration in kidney diseases. Sema3A is upregulated in injured human kidneys and implicated in the development and progression of acute and chronic kidney diseases.

Disease 130

Disease Trials Clinical Development Regulations 130

Drug Discovery World

JULY 4, 2023

New data from Boehringer Ingelheim and Zealand Pharma has demonstrated superior efficacy with survodutide (also known as BI 456906) versus placebo in people with overweight or obesity without type 2 diabetes after 46 weeks of treatment. Current treatments for obesity mainly focus on weight loss by reducing energy intake.

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Drug Trials Trials Drugs Treatment 235

Drug Discovery World

JANUARY 11, 2024

Verge Genomics has initiated a Phase Ib proof-of-concept study for the treatment of sporadic and familial forms of amyotrophic lateral sclerosis (ALS) with VRG50635. ALS is a progressive neurodegenerative disease that affects motor neuron cells in the brain and spinal cord causing progressive paralysis of voluntary muscles.

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Drug Trials Trials Small Molecule Clinical Trials 246

Drug Discovery World

JUNE 25, 2024

A new study demonstrates that AI-powered digital twin solutions can potentially replace the standard-of-care (SOC) control arm of a clinical trial. Phesi created a digital twin for chronic graft versus host disease (cGvHD) in the primary treatment setting.

Clinical Trials 147

Clinical Trials Trials Hospitals Clinical Research 147

Drug Discovery World

MAY 8, 2024

Calliditas Therapeutics has announced data from the proof-of-concept Phase II trial evaluating setanaxib, its lead NOX enzyme inhibitor, in combination with pembrolizumab, in patients with squamous cell carcinoma of the head and neck (SCCHN).

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Trials Clinical Trials Treatment Disease 173

Drug Discovery World

OCTOBER 10, 2022

A new clinical trial, led by the University of Glasgow and NHS Greater Glasgow and Clyde (NHSGGC), hopes to improve treatment options for patients with Crohn’s disease. . Unfortunately, no currently available treatment provides an effective solution for all patients. Official comments.

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Disease Treatment Hospitals Therapies 130

Drug Target Review

OCTOBER 11, 2023

Almost every one of us will know someone with a neurodegenerative disease such as Alzheimer’s or Parkinson’s. It is less common to know someone with a rare genetic disease; but while individually rare, collectively they have an enormous impact. Most of these conditions are genetic in origin and the majority have no effective treatment.

Disease 113

Disease Treatment Small Molecule Therapies 113

Drug Discovery World

JULY 5, 2024

AviadoBio has launched its Phase I/II ASPIRE-FTD clinical trial at the Ohio State University in the US. People with FTD who have disease-causing GRN mutations produce a reduced amount of progranulin protein.

Therapies 147

Therapies Trials Clinical Trials Clinical Development 147

Drug Discovery World

SEPTEMBER 15, 2022

A new combination therapy could represent a significant advancement in the treatment of metastatic colorectal cancer. Findings from the SUNLIGHT trial have revealed that the combination of trifluridine/tipiracil plus bevacizumab showed a statistically significant improvement in overall survival compared to trifluridine/tipiracil alone.

Trials 130

Trials Treatment Clinical Trials Hospitals 130

Drug Discovery World

AUGUST 14, 2024

The US Food and Drug Administration (FDA) has approved a Phase II clinical trial for Biosyngen’s BRG01, an EBV-specific CAR-T cell therapy. This marks the first cell therapy to enter Phase lI trials in both the US and China for the treatment of relapsed/metastatic EBV-positive nasopharyngeal carcinoma.

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Therapies Trials Clinical Trials FDA 130

Drug Discovery World

JULY 2, 2024

The National Institutes of Health (NIH) is sponsoring a clinical trial to evaluate the safety of an investigational monoclonal antibody to treat enterovirus D68 (EV-D68), which can cause severe respiratory and neurological diseases such as acute flaccid myelitis (AFM). Credit: NIAID and CDC.

Virus 147

Virus Trials Clinical Trials Laboratories 147

Drug Discovery World

APRIL 30, 2024

Four new LifeArc Translational Centres for Rare Diseases have been created to give people living with rare medical conditions access to improved tests and treatments. Globally, there are more than 300 million people living with rare diseases. However, rare disease research can be fragmented. The £9.4 ($11.7)

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Drug Discovery World

JUNE 21, 2024

Allogene Therapeutics and Foresight Diagnostics have announced the initiation of a Phase II trial evaluating the use of cemacabtagene ansegedleucel (cema-cel) as part of the first line treatment regimen for newly diagnosed large B-cell lymphoma (LBCL) patients who are likely to relapse.

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