Drug Discovery World

JULY 25, 2023

The efficacy of quizartinib with chemotherapy was evaluated in the QuANTUM-First trial of 539 patients with newly diagnosed FLT3-ITD positive AML. FLT3-ITD status was determined prospectively with a clinical trial assay and verified retrospectively with the companion diagnostic LeukoStrat CDx FLT3 Mutation Assay.

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Drug Discovery World

JUNE 12, 2023

The US Food and Drug Administration (FDA) has approved Minoryx Therapeutics’ Phase III clinical trial (CALYX) of leriglitazone, to treat adult male X-linked Adrenoleukodystrophy (X-ALD) patients with cerebral Adrenoleukodystrophy (cALD).

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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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Drug Discovery World

APRIL 3, 2023

They think the drug has the potential to delay or even reduce the need for joint replacement surgery, the only existing FDA-approved therapy for osteoarthritis. The post Potentially ‘regenerative’ osteoarthritis drug moves to clinical trial appeared first on Drug Discovery World (DDW).

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Drug Discovery World

FEBRUARY 14, 2024

AlphaMedix is a targeted alpha therapy currently in Phase II clinical development, which consists of an SSTR-targeting peptide complex radiolabeled with lead-212 (212Pb) that serves as an in vivo generator of alpha particles. In the Phase I study, treatment was well-tolerated, with a response rate of 62.5%

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Drug Discovery World

SEPTEMBER 13, 2022

The US Food and Drug Administration (FDA) has approved Bristol Myers Squibb’s allosteric tyrosine kinase 2 inhibitor Sotyktu (deucravacitinib) for the treatment of adults with moderate-to-severe plaque psoriasis. The post FDA approves new first-line treatment for plaque psoriasis appeared first on Drug Discovery World (DDW).

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Drug Discovery World

OCTOBER 12, 2023

Dr Samulski’s discoveries in AAV played a profound role in shaping the evolution of the gene therapy field. His work remains a key component of every FDA-approved AAV therapeutic and will remain central to future advances. The post The evolution and future of gene therapy appeared first on Drug Discovery World (DDW).

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Drug Discovery World

OCTOBER 4, 2022

The approval is the result of a long advocacy campaign to convince the FDA to approve the treatment prior to completion of an ongoing Phase III trial. . “We This is a victory for the entire ALS community, which came together to advocate for early approval of AMX0035.

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Drug Discovery World

OCTOBER 12, 2023

Dr Samulski’s discoveries in AAV played a profound role in shaping the evolution of the gene therapy field. His work remains a key component of every FDA-approved AAV therapeutic and will remain central to future advances. The post Podcast: The evolution and future of gene therapy appeared first on Drug Discovery World (DDW).

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BioPharma Drive: Drug Pricing

JUNE 4, 2024

A group of independent experts wasn't convinced by clinical trial data from company Lykos Therapeutics, which is seeking FDA approval of MDMA-assisted treatment for post-traumatic stress disorder.

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Drug Discovery World

SEPTEMBER 12, 2024

Childhood cancers often have a different biology than those in adults, yet targeted treatment options are mostly based on adult therapies. In addition, small patient populations make clinical trials difficult and reduce incentives for investment.

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Drug Discovery World

NOVEMBER 14, 2023

Takeda’s Adzynma has become the first recombinant protein product approved in the US for prophylactic or on-demand enzyme replacement therapy (ERT) for congenital thrombotic thrombocytopenic purpura (cTTP). The post First therapy for very rare blood clotting disorder gets US greenlight appeared first on Drug Discovery World (DDW).

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Drug Discovery World

DECEMBER 8, 2023

Andrew Busey , Co-Founder, Form Bio discusses why vertical AI is set to reshape the cell and gene therapy sector. The cell and gene therapy industry is no exception to this digital transformation. General AI tools such as ChatGPT have acted as a catalyst, igniting a wave of AI innovation akin to the early days of the internet era.

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Drug Discovery World

APRIL 23, 2024

Given the increased awareness of concussion and the associated long-term sequela from concussive injury it is imperative that we accelerate drug development efforts to find an effective therapy. Tremendous strides have been made with other diseases, yet there are still no FDA-approved drug therapies for TBI.

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PLOS: DNA Science

MARCH 28, 2024

FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. The New Drug Duvyzat (givinostat), a type of drug called an HDAC inhibitor, has been in clinical trials to treat cancers and other disorders of the blood, Crohn’s disease, and a form of juvenile arthritis.

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Drug Discovery World

JUNE 16, 2023

Regulatory approvals and industry guidelines are key to the continued development and discovery of drugs, which is a common theme throughout this week’s news highlights, including an FDA approval for Pfizer, a NICE recommendation for AstraZeneca UK, and an update to the HER2 Breast Cancer Testing Guidelines.

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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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Chemical Biology and Drug Design

OCTOBER 10, 2023

Later, a new type of non-nucleoside reverse transcriptase inhibitors (NNRTIs) were approved as anti-HIV drugs. Zidovudine, didanosine, and stavudine are FDA-approved NRTIs, while nevirapine, efavirenz, and delavirdine are FDA-approved NNRTIs.

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Drug Discovery World

MARCH 1, 2024

Pepaxto associated with increased risk of death The FDA is alerting patients and health care professionals that a clinical trial (OCEAN, Study OP-103) evaluating Pepaxto with dexamethasone showed an increased risk of death. The FDA has now suspended enrolment in all ongoing Pepaxto clinical trials.

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Drug Discovery World

DECEMBER 21, 2023

In a record year for gene therapy approvals, it isn’t surprising that cell & gene therapy (CGT) has been a hot topic. Regulatory issues and diversity in clinical trials were also important in 2023, as well as the role that artificial intelligence tools like ChatGPT might play.

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Drug Discovery World

JUNE 10, 2024

The US Food and Drug Administration (FDA) has approved mRESVIA (mRNA-1345), an mRNA respiratory syncytial virus (RSV) vaccine, to protect adults aged 60 years and older from lower respiratory tract disease caused by RSV infection. This approval is also the first time an mRNA vaccine has been approved for a disease other than Covid-19.”

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Drug Discovery World

MARCH 12, 2024

Adoptive T-cell therapy (ACT), particularly T-cell-based transplantation, is attracting increasing attention and is advancing rapidly since the FDA approval of CAR-T therapy for the treatment of relapsed/refractory acute lymphoblastic leukaemia and B-cell malignancies.

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Drug Discovery World

MAY 1, 2024

Many of the best-selling drugs in 2023 were monoclonal antibody (mAB) therapies, including Keytruda (pembrolizumab), Humira (adalimumab), and Dupixent (dupilumab) 1. Hammond says: “Many of the most popular drugs in 2023 are mAb-based therapies, which we predict will continue in 2024.

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Drug Discovery World

APRIL 4, 2023

Orserdu (elacestrant), Stemline Therapeutics The first and only treatment specifically indicated for patients with ESR1 mutations in ER+, HER2- advanced or metastatic breast cancer was approved in the US in February. This made Jesduvroq is the first oral hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI) to be approved in the US.

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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.

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The Pharma Data

JULY 8, 2021

The update includes an addition to the Indications and Usage section of the label (Section 1) to emphasize the disease stages studied in the clinical trials, as seen below ( italics to note updated language). The update clarifies the indication by emphasizing information about the disease stages studied in the ADUHELM clinical trials.

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The Pharma Data

APRIL 19, 2022

Kite’s Global CAR T-Cell Therapy Manufacturing Network Increasing Capacity by 50% to Meet Patient Demand for New Cancer Therapies. — Scalable and Adaptable Facility Provides Flexibility for Current and Future Cell Therapy Innovation. The site will produce Kite’s FDA approved CAR T-cell therapy used to treat blood cancer.

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Drug Discovery World

JULY 31, 2023

President and Chief Executive Officer Christopher Viehbacher said: “In the second quarter, Biogen continued to advance ground-breaking science with the FDA approval of two first-in-class therapies for Alzheimer’s disease and ALS, while also delivering on our base business expectations. Biogen’s business is in transition.

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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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Advarra

JANUARY 11, 2024

The applications of mRNA-based therapies in cancer research represent one of the next groundbreaking steps toward improved cancer treatments. While mRNA usage has played several roles in clinical research , oncology researchers in particular are eager to explore the possibilities of mRNA-based cancer vaccines.

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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

MARCH 29, 2024

The top stories: Novo Nordisk acquires cardiovascular therapies developer Novo Nordisk has acquired Cardior Pharmaeuticals for up to €1.025 billion, including an upfront payment and additional payments if certain milestones are achieved. News round-up for 25-29 March by DDW Digital Content Editor Diana Spencer.

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Drug Discovery World

SEPTEMBER 16, 2022

First biologic therapy for young children with severe eczema . A biologic therapy for very young children with moderate to severe eczema (or atopic dermatitis) has been shown to be safe and effective in an international trial. . Trial sheds light on combined treatment for colorectal cancer .

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The Pharma Data

DECEMBER 17, 2020

Food and Drug Administration (FDA) has approved belimumab (Benlysta®), the first-ever treatment for adults with lupus nephritis (LN) who are currently receiving standard therapy. We at the LRA are thrilled to share this news about belimumab and look forward to future approvals of other lupus nephritis treatment options.”.

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The Pharma Data

JANUARY 21, 2021

Food and Drug Administration (FDA) gave a greenlight for ViiV Healthcare ’s Cabenuva. Today’s FDA approval of Cabenuva represents a shift in the way HIV is treated, offering people living with HIV a completely new approach to care,” said Lynn Baxter, Head of North America, ViiV Healthcare. clinical practices. “Not

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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.

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The Pharma Data

JANUARY 17, 2021

Food and Drug Administration (FDA) approved Janssen Pharmaceuticals ’ (a Johnson and Johnson company) Darzalex Faspro for adults with newly diagnosed light chain amyloidosis. It was approved in combination with bortezomib, cyclophosphamide and dexamethasone (D-VCd). Michael Vi/Shutterstock. It was developed with Genmab.

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