Drug Discovery World

SEPTEMBER 2, 2024

The European Commission (EC) has approved Braftovi (encorafenib) in combination with Mektovi (binimetinib) for the treatment of adult patients with advanced non-small cell lung cancer (NSCLC) with a BRAFV600E mutation. In the treatment-naïve population, the ORR was 75%, including 15% complete responses (CRs) and 59% partial responses (PRs).

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Drug Discovery World

AUGUST 23, 2022

A Phase I/II clinical trial targeting a treatment for Huntington’s Disease (HD) using a gene therapy has been given the clearance to go ahead in France. . There are currently no approved disease modifying therapies for Huntington’s Disease. In 2019, BV-101 was granted orphan drug designation in the European Union. .

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Drug Discovery World

SEPTEMBER 2, 2024

DDW’s Megan Thomas discusses the future of the cell and gene therapy (CGT) sector with industry experts, who predict what to expect in 2024 and beyond. Setting a standard For CGTs to find success in the journey from clinic to bedside, everything from clinical trial design through to quality control requires standardisation.

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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). “As we continue to execute on our clinical development plans for EVO756, we are commencing this Phase II trial in 15 study sites across the United States.

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Drug Discovery World

FEBRUARY 21, 2024

DDW Editor Reece Armstrong speaks to Dr Paul Song , MD, NKGen and Dr Vince DeOrchis , clinician, to find out more about the investigational natural killer therapy SNK01 and the benefit of using a patient’s own cells to treat Alzheimer’s. RA: What are the clinical benefits of using the patient’s own cells as a treatment?

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Drug Discovery World

FEBRUARY 6, 2024

The first UK patients have received an mRNA cancer therapy – mRNA-4359 – as part of a Phase I/II clinical trial investigating its potential for treating melanoma, lung cancer and other solid tumour cancers. The study will assess the therapy administered alone or in combination with immune checkpoint inhibitor pembrolizumab.

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Drug Discovery World

APRIL 12, 2024

The company plans to initiate a Phase I study of the investigational allogeneic off-the-shelf Tr1 Treg therapy for this indication in the second quarter of 2024. ” “Allogeneic stem cell transplantation is the only curative treatment for many advanced blood cancers and genetic and acquired diseases.

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Drug Discovery World

NOVEMBER 16, 2023

The Medicines and Healthcare products Regulatory Agency (MHRA) has authorised the world’s first gene therapy for sickle-cell disease (SCD) and transfusion-dependent β-thalassemia (TDT). Trial results for Casgevy The authorisation is based on positive clinical trial data.

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Drug Discovery World

JUNE 19, 2024

KYV-101, a fully human anti-CD19 chimeric antigen receptor (CAR) T-cell product candidate, has been used to treat a 69-year-old patient suffering from treatment-refractory stiff-person syndrome (SPS). The therapy was given as part of a named-patient use in Germany for critically ill individuals who fail conventional therapies.

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Drug Discovery World

MARCH 4, 2024

In an early phase clinical trial, a combination of antibody-based medications targeting the immune system generated promising safety data and anti-tumour activity in individuals with advanced cancer. Both medications tested in the trial support immune responses against tumour cells. Various dosing schedules of CS1002 (0.3,

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Drug Discovery World

NOVEMBER 20, 2023

“We are delighted to announce the commencement of our ASCEND study in individuals who have recently been diagnosed with Parkinson’s disease and not yet received levodopa treatment,” said Craig Thompson, Chief Executive Officer of Cerevance.

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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

NOVEMBER 28, 2023

An international team of scientists has shown that a stem cell therapy for multiple sclerosis (MS) could prevent further damage to the brain. The early-stage clinical trial was the result of a collaboration between researchers at University of Colorado Anschutz Campus, the University of Cambridge and the University of Milano-Bicocca. “We

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Drug Discovery World

OCTOBER 9, 2023

Positive Phase I trial data indicates that Reqorsa (quaratusugene ozeplasmid) has shown early efficacy for the treatment of non-small cell lung cancer (NSCLC). Developed by gene therapy company Genprex, Reqorsa is a non-viral gene therapy that leads to expression of the TUSC2 tumour suppressor gene in cancers.

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Drug Discovery World

OCTOBER 31, 2023

UK regulatory authorities have approved the first trial of a gene therapy for young children with Hunter syndrome. The drug was developed over eight years by Brian Bigger, Professor of Cell and Gene Therapy at The University of Manchester. The modified HSCs are then infused back into the patient to engraft in the bone marrow.

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Drug Discovery World

NOVEMBER 29, 2023

At sites in the US, Japan, and the EU, the clinical trial will evaluate the safety, tolerability, and efficacy of leniolisib in 15 children, one to six years of age, who have a confirmed APDS diagnosis. The post Trials investigate targeted APDS treatment in a paediatric setting appeared first on Drug Discovery World (DDW).

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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).

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Drug Discovery World

JULY 17, 2024

The US Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to Kyverna Therapeutic’s CAR-T candidate KYV-101 for refractory stiff-person syndrome (SPS). The RMAT designation was based on positive clinical outcomes in patients treated in Germany under a named-patient treatment option.

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Drug Discovery World

OCTOBER 27, 2023

The US Food and Drug Administration (FDA) has granted clearance for Endogena’s Investigational New Drug (IND) application for EA 2351, a potential treatment for geographic atrophy (GA). The post FDA gives green light for AMD treatment trials appeared first on Drug Discovery World (DDW).

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Drug Discovery World

JUNE 25, 2024

A new study demonstrates that AI-powered digital twin solutions can potentially replace the standard-of-care (SOC) control arm of a clinical trial. Phesi created a digital twin for chronic graft versus host disease (cGvHD) in the primary treatment setting.

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Drug Discovery World

AUGUST 5, 2024

The FDA’s decision to grant rare paediatric drug designation to VCN-01 highlights the urgent need for new treatment options for paediatric patients with retinoblastoma,” said Steven Shallcross, Chief Executive Officer of Theriva Biologics. “We

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Drug Discovery World

APRIL 16, 2023

Patients with treatment-naïve resectable non-small cell lung cancer (NSCLC) who received neoadjuvant durvalumab (Imfinzi) plus chemotherapy and adjuvant durvalumab monotherapy had improved event-free survival (EFS) and pathological complete response (pCR) compared with those who received neoadjuvant chemotherapy alone. Overall, 77.6%

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Drug Discovery World

JULY 18, 2024

CatalYm has completed a $150 million Series D financing to support the Phase II development of its cancer therapy visugromab. The proceeds will fund the expansion of the company’s broad Phase IIb development of visugromab into randomised Phase IIb studies in select checkpoint naïve frontline and second-line treatment settings.

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Drug Discovery World

JULY 12, 2024

AB-1005 has also been awarded the innovative medicine designation, the Innovation Passport by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) for the treatment of Parkinson’s disease.

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Drug Discovery World

JUNE 15, 2023

A Phase I clinical trial testing a CAR-T cell therapy treatment in children with acute myeloid leukaemia (AML) has been paused due to a patient’s death. The PLAT-08 clinical trial is a Phase I study of 2seventy bio’s cell therapy SC-DARIC33 in patients with AML.

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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

DECEMBER 18, 2023

University College London (UCL) researchers have developed a new gene therapy to cure focal cortical dysplasia, which a new study shows can significantly reduce seizures in mice. The researchers plan to conduct a first-in-human clinical trial in the next five years. The team then monitored the mice’s brain activity for another 15 days.

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Drug Discovery World

DECEMBER 4, 2023

Qnovia has announced positive results from its first in-human study of QN-01, an inhaled smoking cessation therapy. QN-01 is designed to meet the urgent need for new safe and effective pharmacotherapies that address the shortcomings of currently available nicotine replacement therapies (NRTs).

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Drug Discovery World

AUGUST 7, 2024

The US Food and Drug Administration (FDA) has cleared GigaGen’s application to initiate a Phase I trial of its polyclonal drug for the treatment of hepatitis B virus (HBV) infection, GIGA-2339. With over 1,000 different HBV-targeted antibodies in the mixture, GIGA-2339 is unlike any therapy currently in development.

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Drug Discovery World

AUGUST 9, 2024

The first patient has been dosed in a Phase I clinical trial of AGX101, a novel TM4SF1-directed antibody-drug conjugate being developed for the treatment of solid cancers. The dose expansion portion of the study will evaluate treatment at the recommended Phase II dose in multiple indications.

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Drug Discovery World

AUGUST 8, 2024

Two veteran advocacy organisations, Reason for Hope and the Veteran Mental Health Leadership Coalition, have issued a joint statement urging the US Food and Drug Administration (FDA) to approve MDMA-assisted therapy for post-traumatic stress disorder (PTSD).

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Drug Discovery World

AUGUST 22, 2024

The US Food and Drug Administration (FDA) has approved lazertinib (Lazcluze) in combination with amivantamab-vmjw (Rybrevant) for the first-line treatment of locally advanced or metastatic non-small cell lung cancer (NSCLC). The median PFS was 23.7 months in the lazertinib with amivantamab arm and 16.6 months in the osimertinib arm.

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Drug Discovery World

JUNE 26, 2024

The gene therapy EG110A is a non-replicating HSV-1 vector that has been designed to selectively silence the signals of key bladder sensory neurons responsible for the bladder muscle overactivity, whilst preserving motor neuron and retaining normal bladder function. “NDO

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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

JULY 8, 2024

Biopharmaceutical company Syncromune has had its lead-candidate for the treatment of metastatic castrate-resistant prostate cancer (mCRPC) granted Fast Track designation by the FDA. We look forward to initiating trials at multiple US sites later this year to expand our efforts to develop the SYNC-T SV-102 Therapy.”

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Drug Discovery World

JANUARY 30, 2024

Viral vector manufacturing At the Advanced Therapies Awards ceremony, Dr Luigi Naldini, Director of the San Raffaele Telethon Institute for Gene Therapy, was awarded a Lifetime Achievement Award in recognition of ‘extraordinary contributions to gene therapy’, a field he has helped shape through his pioneering work on lentiviral vectors.

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Drug Discovery World

JULY 22, 2024

Keir Loiacono , CEO of BlueSphere Bio, discusses the goals of the company’s lead programme, the opportunities and potential of T-cell receptor (TCR) T cell therapies, and how to make oncology therapies tailored to individuals a reality. The goals of TCX-101 BlueSphere’s lead programme, TCX-101, includes two goals.

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