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. Analysts GlobalData predict that the total sales of IBD drugs will reach approximately $10.2 billion in the US in 2023.

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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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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. Currently, there are no drugs on the market to treat apathy.

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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. In addition, PTC518 exposure in the cerebrospinal fluid (CSF) was consistent with or higher than plasma unbound drug levels.

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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

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

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. This fantastic drug really is a gamechanger,” study lead Professor John Deanfield from University College London, UK, told The Guardian.

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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

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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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. Lilly will work with the FDA and other global regulators to achieve the fastest path to traditional approvals.

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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

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

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

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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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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Drug Discovery World

APRIL 16, 2024

Axoltis Pharma has received authorisation from ANSM, the French agency for the safety of health products, to launch the SEALS study for Amyotrophic Lateral Sclerosis (ALS). This Phase II clinical trial of drug candidate NX210c in patients with ALS is the first to target the integrity of the blood brain barrier (BBB).

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Drug Discovery World

NOVEMBER 30, 2023

Biotechnology company Certa Therapeutics has revealed positive Phase II clinical trial data for FT011 in patients with scleroderma. This suggests that FT011 may have a positive effect for scleroderma patients by reducing the inflammation and fibrosis associated with the disease.

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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

JANUARY 31, 2024

Vera Therapeutics has announced positive 72-week data from the open label extension (OLE) period of its Phase IIb ORIGIN clinical trial of atacicept in participants with IgA nephropathy (IgAN). In aggregate, the 72-week data with atacicept are consistent with a profile of true disease modification in IgAN. PLOS ONE 2015). “We

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Drug Discovery World

JULY 24, 2024

SynaptixBio and Evotec have announced they are extending their partnership to find further candidate drugs to treat H-ABC, the most severe form of TUBB4A leukodystrophy. 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.

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Drug Discovery World

MARCH 6, 2024

UK-based biotech SynaptixBio has secured a second Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA). This second ODD allows research and development of a therapy for another form of the disease, Isolated Hypomyelination.

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Drug Discovery World

OCTOBER 20, 2023

A new free service will offer insights in under ten days to sponsors conducting clinical trials, identifying potential ‘zombie trials’ and providing actionable recommendations for failing trials. The Health Check and Trial Rescue Service is an initiative of data software and services company Phesi.

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Drug Discovery World

MAY 17, 2024

The collaborators, led by Dr Heidi Welch, tested the pro-inflammatory protein in a human cell culture model, demonstrating its potential to be targeted by small molecule drugs. The post Partners explore precision medicine approach to heart disease appeared first on Drug Discovery World (DDW).

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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

SEPTEMBER 4, 2024

The European Commission has granted orphan medicinal product designation (OMPD) to Cereno Scientific’s lead drug candidate CS1 for pulmonary arterial hypertension (PAH). The EU OMPD complements the orphan drug designation (ODD) granted by the US FDA in 2020, enhancing the protection and value of CS1 in these major markets.

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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

JANUARY 9, 2024

The company intends to use the proceeds to initiate and complete the first-in-human Phase I clinical trial and reach Phase II readiness for its lead program, RES-010, in obesity. The post €10M financing to support disease-modifying obesity drug appeared first on Drug Discovery World (DDW).

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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.

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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

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

JULY 5, 2023

A new project by researchers in Edinburgh aims to identify combinations of existing drugs that could be used together to treat motor neurone disease (MND). There is currently only one drug approved to treat MND in the UK, riluzole, which has only a modest effect. Led by Prof Siddharthan Chandran, the £3.3

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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

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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Quanticate

SEPTEMBER 13, 2024

With the implementation of technology rapidly increasing, stricter regulatory standards, and growing demand for innovative treatments, the trends within the industry continue to evolve, impacting the future of clinical trials both positively and negatively.

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Drug Discovery World

JUNE 9, 2023

Bellerophon Therapeutics has announced it will close its Phase III study of INOpulse, its treatment for fibrotic Interstitial Lung Disease (fILD), after the trail failed to meet its primary endpoint. The post Phase III pulmonary fibrosis drug trial fails 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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