Drug Discovery World

APRIL 26, 2024

A clinical trial of a personalised mRNA cancer vaccine for melanoma patients has been launched in the UK. This is a really finely honed tool”. “This is very much an individualised therapy and it’s far cleverer in some senses than a vaccine,” said Shaw. “It in the combination arm and 62.2%

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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). ” Amgen is now planning to file for approval in the US following results on the MITIGATE study. .”

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Drug Discovery World

DECEMBER 11, 2023

The US Food and Drug Administration (FDA) has approved the first cell-based gene therapies for the treatment of sickle cell disease (SCD), Casgevy and Lyfgenia. Casgevy Casgevy, a cell-based gene therapy, is approved for the treatment of SCD in patients 12 years of age and older with recurrent vaso-occlusive crises (VOCs).

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Drug Discovery World

APRIL 16, 2023

Adjuvant therapy with atezolizumab (Tecentriq) and bevacizumab (Avastin) increased recurrence-free survival of patients with hepatocellular carcinoma (HCC) following surgical resection or ablation, according to the results from a Phase III trial. According to the results of an interim analysis conducted after a median follow-up of 17.4

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Drug Discovery World

MARCH 6, 2024

This second ODD allows research and development of a therapy for another form of the disease, Isolated Hypomyelination. Earlier last year the biotech successfully led a second round of investment, taking the total up to £13.2m ($16.7m), which will take it up to the start of in-human clinical trials later this year.

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Drug Discovery World

SEPTEMBER 20, 2022

SKYSONA has become the first FDA approved therapy to slow the progression of neurologic dysfunction in boys with cerebral adrenoleukodystrophy (CALD). This will probably include data from the ongoing long-term, follow-up study LTF-304, which follows patients treated in clinical trials for 15 years, and from commercially treated patients. “As

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Drug Discovery World

FEBRUARY 24, 2023

A Phase I safety and pharmacodynamic activity trial shows IMC-M113V, the first soluble T cell receptor (TCR) therapy for people living with HIV, is well tolerated. Although people living with HIV can control their disease with antiretroviral therapies, lifelong treatment is needed as reservoirs of HIV infected cells persist.

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Drug Discovery World

FEBRUARY 27, 2023

An investigational gene therapy for Sanfilippo syndrome – which leads to a form of childhood dementia – has shown promising early results in a proof-of-concept study. The post Gene therapy for ‘childhood dementia’ shows promise appeared first on Drug Discovery World (DDW).

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Drug Discovery World

APRIL 5, 2023

CureSearch for Children’s Cancer, a national non-profit with a mission to end childhood cancer by driving targeted and innovative research in an accelerated time frame, will fund a Phase I clinical trial to bring new therapies to paediatric brain cancer patients, including those with limited or no treatment options.

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Drug Discovery World

NOVEMBER 14, 2023

A Phase Ib trial has shown low-density lipoprotein cholesterol (LDL-C) reductions up to 55% and blood PCSK9 protein reductions up to 84% after a single infusion of Verve Therapeutics’ Verve-101. The post Single-course in vivo base editing therapy proven to lower cholesterol appeared first on Drug Discovery World (DDW).

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Drug Discovery World

APRIL 10, 2023

RESP-X, Infex Therapeutics’ anti-virulence therapy to treat Pseudomonas aeruginosa (Pa) infections in non-cystic fibrosis bronchiectasis (NCFB) patients, has shown a promising interim safety and tolerability profile in its ongoing first-in-human Phase I clinical trial.

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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.

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Drug Discovery World

DECEMBER 16, 2022

Jannsen has revealed clinical trial results for its pipeline of products to treat multiple myeloma and leukaemia. . Along with positive Phase II trial data for talquetamab , the company presented promising findings on the long-term results of daratumumab in newly diagnosed, transplant-ineligible multiple myeloma patients. .

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Drug Discovery World

FEBRUARY 28, 2023

The combination therapy of niraparib and abiraterone acetate (AA) could become the first dual action tablet (DAT) available in the European Union for metastatic castration-resistant prostate cancer (mCRPC) with BRCA1/2 mutations.

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Drug Discovery World

JULY 12, 2023

Diuretic-resistance is widespread with nearly half of these patients leaving hospital with persistent congestion, and one in four readmitted within 30 days of discharge. The post Sequana announces first patient enrolled in heart failure trial appeared first on Drug Discovery World (DDW).

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Drug Discovery World

SEPTEMBER 5, 2022

A pathway to better therapies. The findings suggest a pathway to better therapies for one of the least treatable forms of cancer. Phase I trial on the horizon. We’re talking to a number of other groups about starting a Phase I clinical trial as soon as possible,” Mosammaparast said. This has not changed for 30 years.

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Drug Discovery World

JUNE 6, 2024

The combination therapy was also shown to reduce the risk of distant metastasis or death by 62% compared to Keytruda alone in these patients. The global Phase III clinical trial is being led by University College London Hospitals NHS Foundation Trust in the UK and will include around 1,100 people.

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KIF1A

APRIL 25, 2024

She is scheduled to be the 2nd patient in the world to receive ASO therapy for KAND. March 2023 – We met with n-Lorem and found out Susannah’s ASO (1st KAND patient to receive ASO therapy for KAND) would work for Sloane. Sloane is an adorable 3 year old with KAND. Hello KIF1A Community! We are also paying for our housing.

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Drug Discovery World

AUGUST 2, 2023

Renaissance Pharma has announced its first development programme focused on Hu14.18, a humanised anti-GD2 monoclonal antibody (mAb), licensed from St Jude Children’s Research Hospital for the treatment of newly diagnosed high-risk neuroblastoma. A novel Phase II trial incorporating Hu14.18 and three-year event-free (EFS) of 73.7%.

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Drug Discovery World

MAY 31, 2024

The selected news highlights this week report breakthrough findings from early phase research and development, mainly preclinical studies and Phase I clinical trials, including a drug that can prevent sepsis deaths, a microbiome based immuno-oncology candidate, and a plant-based therapy for diarrhoea.

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Drug Discovery World

JANUARY 18, 2024

The results, together with the data from the recent LPS human challenge trial, further support the strategic expansion of the Phase II ready POLB 001 to treat and prevent cancer immunotherapy-induced CRS. Reductions were seen in all serum proinflammatory cytokines tested.

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Drug Discovery World

MARCH 29, 2024

targeted therapy approved by any regulatory agency in the world. Both trials met their primary endpoint, progression-free survival (PFS), as well as a key secondary endpoint, overall survival (OS), showing statistical significance in patients treated with Vyloy plus chemotherapy compared to placebo plus chemotherapy.

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Drug Target Review

FEBRUARY 21, 2024

Stem cell transplants have saved patients’ lives time and time again, which led us to launch our own Stem Cell Transplant and Cellular Therapy Program. Regarding CAR-T cell therapy, could you highlight the promising findings that have been identified in the early stages of drug discovery, as well as potential challenges?

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PPD

JUNE 7, 2023

The number of pediatric patients diagnosed with nonalcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis (NASH) is on the rise, yet there are currently no approved therapies to treat NAFLD and NASH in adult or pediatric populations. As therapies for the treatment of NASH in adult patients go this year to the U.S.

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Drug Discovery World

JANUARY 31, 2024

Breye Therapeutics, a clinical-stage biopharmaceutical company developing novel oral therapies for retinal vascular diseases within ophthalmology, has started a Phase Ib/IIa clinical trial to investigate danegaptide, following oral administration, in patients suffering from diabetic macular oedema (DMO).

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Drug Discovery World

JUNE 28, 2023

Takeda has revealed favourable interim results from its Phase III crossover trial evaluating the safety and efficacy of TAK-755 (recombinant ADAMTS13) replacement therapy for the prophylactic treatment of congenital thrombotic thrombocytopenic purpura (cTTP). Acute TTP events have a mortality rate of >90%, if left untreated.

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Drug Target Review

FEBRUARY 1, 2024

This can present challenges when attempting to recruit an enriched patient population for clinical trials. Historically, what has limited investor interest in funding neurological and psychiatric therapies in development? The lack of heterogeneity can introduce significant risk into mid- to late-stage studies.

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Drug Discovery World

DECEMBER 28, 2023

When I was a second-year resident training as a paediatric oncologist at Nicklaus Children’s Hospital, I met a teenage boy diagnosed with Hodgkin’s Lymphoma. He was admitted to the hospital for weekly chemotherapy. When I made afternoon patient rounds, I would find him sitting up in his hospital bed doing homework.

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Drug Discovery World

JULY 31, 2023

DDW’s Reece Armstrong speaks to Hendrik Streefkerk , Chief Medical Officer and Drug Safety Officer of CellProthera about the company’s cell therapy for acute myocardial infarction (AMI) and why regenerative medicine could be key to treating cardiac diseases. million people every year.

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Drug Discovery World

DECEMBER 22, 2023

In other news, the FDA approved a promising antibody-drug conjugate for bladder cancer and trial results demonstrated that psilocybin could reduce depression symptoms in cancer patients. In the study, trial participants treated with psilocybin not only experienced a lessening of depressive symptoms but also spoke highly of the therapy.

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Drug Discovery World

APRIL 22, 2024

The treatment becomes only the second CAR-T cell therapy to secure routine funding for use in the NHS, and the only one for treating ALL in children and young adults under 26 years old. It offers a chance of durable remissions and prolonged overall survival for people who often have no other option.

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Drug Discovery World

JUNE 12, 2023

Immutep revealed positive final data from Part C of the TACTI-002 Phase II trial at the ASCO 2023 Annual Meeting in Chicago, US. The combination treatment was investigated in patients unselected for PD-L1 expression (N=37), with disease progression on, or after, platinum-based therapy (± cetuximab).

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Drug Discovery World

MAY 8, 2024

The ORCA-V1 trial was led by Achieve Life Sciences, and co-led by an investigator from Massachusetts General Hospital. In their previous clinical trial, the research team found that cytisinicline helped people to quit smoking traditional cigarettes. They hypothesised that it might also help people to stop vaping nicotine.

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The Pharma Data

OCTOBER 20, 2020

20, 2020 — It may seem counterintuitive, but when someone with the eating disorder anorexia nervosa is hospitalized, treatment often begins by cutting calories. Now, new research suggests that those eating restrictions can be safely relaxed in the hospital. That led to shorter hospital stays, the study found. TUESDAY, Oct.

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The Pharma Data

DECEMBER 29, 2020

29, 2020 /PRNewswire/ — Phase 3 program in hospitalized patients to continue based on passing futility analysis on ability to reduce incidence of death or mechanical ventilation. As in earlier outpatient trial, immune status when patients entered the trial was a strong predictor of viral load and clinical outcomes.

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Drug Discovery World

SEPTEMBER 7, 2023

1 There are a number of challenges faced in paediatric R&D, including the fact that the overall group includes five different age groups, the need for specific biomarkers, the extra length of time needed for trials, and the ethical concerns of conducting clinical trials in children.

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Drug Discovery World

MAY 31, 2024

Cell and gene therapy delivery is another area where current digital tracking solutions are needed to replace the paper-based documentation often used in clinical trials. Especially, as they require removing cells from a patient or donor, modifying them, and then reintroducing to a patient. “Our

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