Drug Discovery World

JUNE 23, 2023

Sarepta Therapeutics’ Elevidys has become the first gene therapy for Duchenne muscular dystrophy (DMD) to gain marketing authorisation in the US. The Food and Drug Administration (FDA) has granted accelerated approval to Elevidys (delandistrogene moxeparvovec-rokl), an adeno-associated virus (AAV) based gene therapy for the treatment of DMD.

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Drug Discovery World

SEPTEMBER 25, 2024

Mylotarg was cleared by the FDA’s accelerated approval process, allowing it to be marketed on the basis of early data in the expectation that a larger trial would confirm its benefits. And, last but not least, therapy with ADCs offers physicians the decisive advantage that it can be administered on site at the hospital.

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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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Treatment Clinical Trials Immune Response Therapies 162

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

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

AUGUST 30, 2022

The Phacilitate Advanced Therapies Europe 2022 conference will take place from 30 August to 1 September at the Royal Lancaster in London, UK. The conference is the European twin to Phacilitate Advanced Therapies Week, an event held in January in Florida, US. Improving patient experience within clinical trials and long term follow up.

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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

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

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, from Vertex Pharmaceuticals and CRISPR Therapeutics, is also the first FDA-approved treatment to use CRISPR gene editing technology.

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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). Nearly half of patients who do not receive treatment die within five years of symptom onset. Today, parents whose boys receive a CALD diagnosis can have renewed hope for the future.”.

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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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Therapies Treatment FDA Approval Vaccine 64

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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Clinical Trials Trials Therapies Treatment 130

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

SEPTEMBER 16, 2024

A new study has demonstrated that the frequency of second primary malignancies (SPMs) is no higher with CAR-T cell therapies than with previous standard-of-care strategies. in the three years after therapy. They identified 326 SPMs across 5,517 patients from 18 clinical trials and seven real-world studies.

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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

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. Since the mutation only affects a single gene, it makes it a prime candidate for genome editing treatments.

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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. Three additional patients are currently within the normal cognitive development range at nine to 18 months post-treatment, but require longer follow-up to assess outcomes.

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Therapies Trials Virus Disease 130

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

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). ORR was 75% in the 79 patients treated with the recommended dosage. The median time to first response was 1.5

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FDA Approval FDA Disease Therapies 147

Drug Target Review

AUGUST 28, 2024

What are the main challenges currently faced in the treatment of chronic liver diseases, and how does Resolution Therapeutics aim to address these challenges? Once a patient develops advanced cirrhosis/end-stage liver disease there are no specific therapies to significantly avoid major decompensations and death in the next few years.

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Therapies Disease Engineering Medical Schools 114

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. A lethal disease Prostate cancer is the most common cancer in men in Europe.

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Therapies Hospitals Disease Treatment 130

Drug Discovery World

SEPTEMBER 13, 2022

Ayala Pharmaceuticals has revealed positive interim results from its ongoing RINGSIDE Pivotal Phase II and III clinical trial into gamma-secretase inhibitor AL102 in desmoid tumours. The post New treatment hope for patients with rare tumours appeared first on Drug Discovery World (DDW).

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Treatment FDA Approval Clinical Trials Hospitals 130

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.

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Drug Discovery World

JULY 11, 2022

Pharmaceutical company Novo Nordisk has announced positive results for its prophylactic treatment targeting people living with haemophilia A or B with inhibitors. . The company’s concizumab treatment is an anti-tissue factor pathway inhibitor (TFPI)? The treatment of haemophilia is complex and no one treatment fits all, ” ?

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Drug Discovery World

OCTOBER 2, 2024

ETD001 ETD001 is a novel, first in class blocker of the epithelial sodium channel (ENaC) aimed at treating people with cystic fibrosis (pwCF) without current effective therapies. The drug entered Phase II clinical trials in July 2024, which are expected to complete in 2025.

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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. Following four weeks of treatment, there is a three-month safety follow-up period.

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Trials Disease Therapies Treatment 130

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. The problem is that these tumours respond to treatment initially, but then they come back. Phase I trial on the horizon. Hindering this repair process makes the drug much more effective. .

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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 s submission.

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Drug Target Review

JANUARY 23, 2024

What innovative approaches and technologies are anticipated to reshape immuno-oncology treatments in 2024? Immuno-oncology, notably through checkpoint inhibitors, has significantly reshaped cancer treatment over the past decade. Espen integrates clinical and research expertise as Director of Medical Affairs.

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Treatment Immune Response Vaccine Therapies 122

KIF1A

MAY 29, 2024

Fortunately, Sloane does not currently have epilepsy, and Megan hopes this treatment will prevent it’s onset as her disease progresses. On the day of the treatment, they checked into the hospital where Sloane was administered sedation medication. The post Sloane’s 1st ASO treatment appeared first on KIF1A.

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Treatment Doctors Hospitals Therapies 52

Drug Discovery World

MARCH 29, 2024

targeted therapy approved by any regulatory agency in the world. These results support Vyloy as a new treatment option for the CLDN18.2-positive Vyloy is the first and only CLDN18.2-targeted Gastric cancer is the third deadliest cancer in Japan, with 126,724 cases diagnosed in 2022. positive population in Japan.”

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Drug Discovery World

SEPTEMBER 26, 2024

Islet allotransplantation – the transplantation of isolated pancreatic islets from a deceased donor to a living recipient with type 1 diabetes – has emerged as a promising therapy for patients with type 1 diabetes, particularly those experiencing hypoglycemia unawareness. Rita Bottino leads Imagine Pharma’s Islet Programs.

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Clinical Trials Pharma Discussion Therapies Trials 147

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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Trials Clinical Trials Drugs Therapies 130

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. months after initial treatment. 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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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. “The For cytisinicline treatment compared to placebo, 31.8%

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Clinical Trials Trials Compliance Drugs 182

Drug Discovery World

DECEMBER 22, 2023

There have been some ground-breaking advances in women’s health this week, with new treatments for hot flushes and pregnancy sickness, and a discovery that could aid in development of treatments for secondary breast cancer. The post This week in drug discovery (18-22 December) appeared first on Drug Discovery World (DDW).

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