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. The post Positive interim data from Huntington’s disease trial appeared first on Drug Discovery World (DDW).

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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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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.

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Drug Discovery World

DECEMBER 19, 2023

The first patient has been dosed with an engineered B cell investigational therapy in a Phase I trial in Mucopolysaccharidosis type I (MPS I). Developer Immusoft has received FDA Orphan Drug Designation and Rare Pediatric Disease Designation for the therapy, designated ISP-001, in this indication.

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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).

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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.

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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.

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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.

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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.

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Conversations in Drug Development Trends

AUGUST 29, 2024

By: Nathan Chadwick, Therapeutic Strategy Lead, Rare Disease Over the last few years in clinical trials, particularly within the rare disease community, a notable shift is underway, where patients and caregivers are taking the lead in reaching out to clinical trial sites rather than the other way around.

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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.

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Drug Discovery World

AUGUST 12, 2022

Pfizer has launched a Phase III clinical trial of a vaccine to protect people against Lyme disease, in collaboration with pharmaceutical company Valneva. . The trial will enrol up to 6,000 participants aged five years and older and will be conducted across 50 sites where Lyme disease is highly endemic. ” . “We

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BioPharma Drive: Drug Pricing

JUNE 20, 2024

The agency has lifted a partial trial suspension based on one-year data showing PTC’s pill suppressed a key protein associated with the disorder.

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Conversations in Drug Development Trends

JANUARY 24, 2024

Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment. One diagnostic example that I discussed in my presentation is autism.

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Drug Discovery World

JULY 24, 2024

Not only do they have the capabilities to identify and develop candidate drugs, they can then help us take them right through clinical trials and beyond. They are instrumental in supporting us as we prepare for those clinical trials.”

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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.

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Drug Discovery World

JULY 14, 2022

Biotech company Actigen has moved closer to clinical trials for rare disease treatment for Hunter syndrome, following a pre-Investigational New Drug (IND) meeting with the US Food and Drug Administration (FDA). ? . Hunter syndrome .

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Cytel

MARCH 11, 2024

In 2023, rare diseases accounted for 30% of product pipeline under development, about half of which comprising non-oncology rare diseases. Clinical development in rare diseases has specific challenges. Effective clinical development strategy for rare diseases requires agility to adapt to accumulating learning.

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Drug Discovery World

JANUARY 11, 2024

VRG50635 is a small molecule PIKfyve inhibitor and is reportedly one of the first drugs to enter clinical trials that was entirely discovered and developed using an AI-enabled platform. ” The post AI-discovered ALS drug trial uses unique remote monitoring appeared first on Drug Discovery World (DDW).

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Drug Discovery World

NOVEMBER 24, 2023

Project leader, Associate Professor Keith Chappell, said the preliminary clinical trial results were an ‘exciting reward’ for the team’s continued dedication and the community’s widespread support for the project. “We The Clamp2 trial group showed a 2.5-fold fold boost in neutralising ‘titres’ and it was 2.1 in the Novavax group.

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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.

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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. They aim to tackle barriers that ordinarily prevent new tests and treatments reaching patients with rare diseases and speed up the delivery of rare disease treatment trials.

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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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Drug Discovery World

JULY 24, 2024

Enterprise Therapeutics announced it has dosed the first person with cystic fibrosis (pwCF) in its Phase IIa trial of ETD001. The Phase IIa trial aims to deliver clinical proof-of-concept and to assess the safety profile of ETD001 in the 10% of pwCF with the highest unmet medical need.

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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.

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Drug Discovery World

JULY 4, 2023

Given the prevalence of obesity and its many disease-related complications, there is a dire need for treatments that can help treat the disease of obesity effectively,” said Carel le Roux, Professor at University College in Dublin, Ireland, and Principal Investigator of the trial.

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Drug Discovery World

MAY 30, 2024

Swedish company IRLAB Therapeutics has initiated a Phase I study of IRL757, its drug candidate for apathy – a condition associated with Parkinson’s disease and other CNS diseases. IRL757 has shown positive effects in several preclinical models of cognitive function, including improved motivation.

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Drug Discovery World

MAY 17, 2024

This could open new opportunities for a precision medicine approach to heart disease. The target is now being evaluated as part of PlaqueTec’s ongoing BIOPATTERN trial and, if validated, further development will be pursued. This data formed the basis for the current CIA-funded project.

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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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Drug Discovery World

MAY 14, 2024

The studies are based on the Semaglutide and Cardiovascular Outcomes (SELECT) trial, the largest and longest clinical trial of the effects of semaglutide on weight in over 17,000 adults. of their bodyweight.

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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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Drug Discovery World

NOVEMBER 28, 2023

An experimental drug has shown potential as a disease-modifying therapy for Parkinson’s disease, according to a new study published in Nature Communications. Parkinson’s disease is highly associated with mitochondrial dysfunction. Mission is planning to initiate a MTX325 Phase I trial in humans in early 2024.

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Drug Discovery World

JUNE 8, 2023

Axol Bioscience has signed an exclusive agreement with StrataStem to access and commercialise its collection of Alzheimer’s Disease (AD) patient samples. Grown in vitro, these cells can provide patient specific human brain models from a large cohort of AD patients – to create a ‘clinical trial in a dish’.

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Drug Discovery World

MARCH 14, 2024

NeuroSense Therapeutics has reported additional positive data from its six-month double-blind Phase IIb PARADIGM trial of its lead drug candidate PrimeC for the treatment of amyotrophic lateral sclerosis (ALS). The PARADIGM trial’s secondary clinical efficacy outcome measure endpoints included Quality of Life and Survival.

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Drug Discovery World

APRIL 8, 2024

Their research signals a transformative leap in liver disease management, addressing both inflammation and fibrosis. It is progressing to Phase I clinical trials with JD Bioscience. The post Potent drug candidate could transform liver disease treatment appeared first on Drug Discovery World (DDW).

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Drug Discovery World

APRIL 19, 2024

Askbio’s AB-1005 (also known as AAV2-GDNF), an investigational gene therapy for the treatment of Parkinson’s disease, was well tolerated with no serious adverse events in a Phase Ib clinical trial. The data were presented at the American Academy of Neurology 2024 Annual Meeting in Denver, Colorado, USA.

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Drug Discovery World

OCTOBER 31, 2023

Professor Andy Whiting and Dr Tony Lockett discuss how specific levels of a protein could be used to advance drug development in neurodegenerative diseases. These parameters aim to measure the progression of disease over time by looking at the functional or observable change in a patient.

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