Drug Discovery World

APRIL 4, 2024

The US Food and Drug Administration (FDA) has approved Vafseo (vadadustat) Tablets for the treatment of anaemia due to chronic kidney disease (CKD) in adults who have been receiving dialysis for at least three months. Vafseo is now approved in 37 countries.

FDA Approval 130

FDA Approval FDA Disease Treatment 130

Drug Discovery World

NOVEMBER 8, 2023

The UK lags comparable countries, including France and Germany, on the degree of availability of treatments for rare diseases, according to a new report. In response, the BioIndustry Association (BIA)’s Rare Disease Industry Group (RDIG) is calling for the UK to improve access to new treatments for patients.

Disease 130

Disease Treatment International Pharmaceuticals 130

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

MARCH 14, 2023

Teitur Trophics, a biotech company developing new treatments for patients suffering from neurodegenerative diseases, has complete a €28M Series A financing. The financing was co-led by Sunstone Life Science Ventures and Sound Bioventures, with participation from new investors, Industrifonden, Innovestor’s Life Science Fund and P53 Invest.

Disease 130

Disease Treatment Science Clinical Development 130

Drug Discovery World

AUGUST 23, 2024

A new study from Korea University suggests that treatment with direct-acting antivirals (DAAs) improves liver disease-related clinical outcomes in patients with chronic hepatitis C virus (HCV) infection and reduces liver fibrosis-based disease burden. The differences were most prominent in age groups 40–60 years.

Treatment 147

Treatment Disease Virus Drugs 147

Drug Discovery World

NOVEMBER 23, 2022

Scientists have found a new drug treatment that can slow the progression of neurodegenerative disease in mice. The breakthrough research may offer fresh hope in tackling currently untreatable conditions such as Alzheimer’s disease (AD). .

Disease 243

Disease Drugs Treatment Science 243

Drug Discovery World

MARCH 24, 2023

Israeli researchers have developed a drug that is effective in treating a rare genetic muscle disease. The team demonstrated that the disease is caused by a mutation in the gene encoding HMG CoA reductase, inhibiting the enzyme’s activity. This is the first study to link muscle pain and weakness to statin use.

Disease 130

Disease Treatment Research Licensing 130

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

AUGUST 28, 2024

Under the collaboration, the companies will analyse somatic mutations that occur in diseased patient tissue to inform the discovery and development of therapies for cardiovascular and renal diseases. The collaboration was the first initiated under Flagship’s Pioneering Medicines strategic partnership with Pfizer.

Disease 147

Disease Therapies International Drug Development 147

Drug Discovery World

SEPTEMBER 22, 2022

Nefecon has become the first and only approved treatment in the EU for primary immunoglobulin A nephropathy (IgAN), a rare, progressive kidney disease. The drug was developed in collaboration with Calliditas Therapeutics, and will be marketed in Europe by STADA Arzneimittel, with the first launch in Germany.

Disease 130

Disease Treatment Biosimilars Marketing 130

Drug Discovery World

JULY 24, 2024

The trial was carried out at the Queen Elizabeth Hospital, Birmingham, UK, by the University of Birmingham’s Drugs, Devices, Diagnostics and Biomarkers (D3B) team. The delivery of fast functional benefit alongside long-term disease stabilisation would make ILB a unique treatment option for this devastating disease.

Trials 130

Trials Disease Hospitals Drugs 130

Drug Discovery World

JANUARY 30, 2023

Cell and gene therapies (CGT) are transforming the way diseases are treated. The event will cover aspects affecting growth of the CGT market, including how manufacturing technologies are enabling drug development, what these therapies mean for the healthcare market, and the opportunities for developers.

Disease 130

Disease Treatment Therapies Drug Development 130

Drug Discovery World

AUGUST 20, 2024

Positive results from multiple in vivo studies have shown the impact of sphingosine kinase-2 (SPHK2) inhibition in various models of metabolic disease. “Opaganib, which acts as a sphingosine competitor, is the first clinical drug to target three key enzymes in this pathway.”

Treatment 147

Treatment Virus RNA Therapies 147

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

FEBRUARY 16, 2023

X-linked adrenoleukodystrophy (X-ALD) is an orphan inherited neurodegenerative disease. 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 130

Treatment Disease Clinical Trials International 130

Drug Discovery World

JULY 9, 2024

The Scottish Medicines Consortium (SMC) has published final advice on two rare disease therapies. Elfabrio (pegunigalsidase alfa), a novel enzyme replacement therapy (ERT), has been approved as a treatment option for Fabry disease in adults. Birch bark extract gel EB is the name for a group of rare, genetic skin disorders.

Disease 147

Disease Therapies Government Treatment 147

Drug Discovery World

AUGUST 19, 2024

Researchers have uncovered how Flaviviruses like Dengue and Zika propagate, suggesting the possibility of new treatment strategies. With the global risk of such virus-borne diseases increasing, DENV alone is known to cause approximately 390 million infections and 100 million symptomatic cases annually, and JEV around 70,000 cases each year.

Treatment 147

Treatment Virus Vaccine Disease 147

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

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. . Crohn’s disease is a chronic condition which currently affects more than 115,000 people in the UK and causes inflammation of the lining of the digestive system.

Disease 130

Disease Treatment Hospitals Therapies 130

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.

Drugs 264

Drugs FDA Marketing Treatment 264

Drug Discovery World

JANUARY 30, 2023

The primary objectives of MOVE were to evaluate the efficacy of palovarotene in reducing new HO volume, as assessed using whole-body computed tomography. “There is a significant need for a treatment specifically developed to help manage the progression of this disease.

Treatment 130

Treatment Disease Clinical Trials Trials 130

Drug Discovery World

JUNE 26, 2024

Scientists at the University of Bristol have uncovered a new disease driving mechanism which they are aiming to target to help treat the development of atherosclerotic plaques. This unique award will allow us to develop a therapeutic to target this novel non-coding RNA, and validate its use in patients with heart disease.”

Treatment 147

Treatment Research Medical Schools RNA 147

Drug Discovery World

JANUARY 8, 2024

Milan-based Newron Pharmaceuticals has reported “exceptional” one-year results from study 014/15 into evenamide as an add-on to antipsychotics for the management of treatment-resistant schizophrenia (TRS). More than 70% of the patients experienced clinically important reduction in disease severity.

Treatment 147

Treatment Drugs Laboratories Pharmaceuticals 147

Drug Discovery World

APRIL 8, 2024

Class B GPCRs are recognised as important targets for new medicines, particularly in chronic endocrine and metabolic diseases. We are grateful to VLAIO for its continued support, which will bolster our capabilities in Class B GPCR drug discovery and development.” Confo’s platform includes its ConfoBody and ConfoChimer technologies.

Disease 130

Disease Small Molecule Drugs Treatment 130

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.

FDA 130

FDA Disease Therapies Drugs 130

Drug Discovery World

AUGUST 28, 2024

The European Commission has granted marketing authorisation for antibody-drug conjugate (ADC) Padcev (enfortumab vedotin) in combination with PD-1 inhibitor Keytruda (pembrolizumab) for urothelial cancer. I look forward to seeing the treatment combination implemented as a first-line regimen in the clinical setting.”

Treatment 130

Treatment Clinical Trials Trials FDA 130

Drug Discovery World

MAY 14, 2024

In 2023, the SELECT trial reported that adults with overweight or obesity but not diabetes taking semaglutide for more than three years had a 20% lower risk of heart attack , stroke, or death due to cardiovascular disease, and lost an average 9.4% of their bodyweight.

Disease 147

Disease Drugs Clinical Trials Trials 147

Drug Discovery World

AUGUST 8, 2024

Additionally, MTX-463 was granted orphan drug and fast track designations by the FDA, underscoring the potential of WISP1 to address significant unmet needs in treating rare fibrotic diseases, including idiopathic pulmonary fibrosis (IPF).

Treatment 147

Treatment Pharmacokinetics Trials Disease 147

Drug Discovery World

JANUARY 31, 2024

In aggregate, the 72-week data with atacicept are consistent with a profile of true disease modification in IgAN. These cytokines are members of the tumour necrosis factor family that promote B-cell survival and autoantibody production associated with certain autoimmune diseases, including IgAN and lupus nephritis. PLOS ONE 2015). “We

Disease 130

Disease Clinical Trials Drugs Trials 130

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 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

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

SEPTEMBER 13, 2024

This week, DDW has opened registrations for its webinar ‘ The latest advancements driving neuroscience drug discovery research ’, so our news highlights focus on developments in neuroscience, including a novel drug that has been shown to reduce neuronal death, and a potential disease-modifying treatment for dementia.

Drugs 147

Drugs Disease Hospitals Treatment 147

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.

Drug Development 147

Drug Development Disease Drugs Clinical Trials 147

Drug Discovery World

JUNE 11, 2024

Researchers have discovered a new biological pathway in inflammatory bowel disease (IBD) and related conditions that can be targeted using existing drugs. About 5% of the world’s population are currently affected by an autoimmune disease, such as IBD, the umbrella term for Crohn’s disease and ulcerative colitis.

Disease 147

Disease Research DNA Laboratories 147

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). ? . Official comments .

Clinical Trials 130

Clinical Trials Treatment Trials Disease 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

Therapies Disease Medical Schools Trials 147