Drug Target Review

NOVEMBER 17, 2023

Up until 2023 around 600 NK cell clinical trials have been reported and some 200 are clinically active and still recruiting patients. The vast majority (65/74; 88%) of those trials involved NK cells without CARs; while only 12 percent were CAR-NK studies. Only three of the CAR-NK studies were for the treatment of solid tumours.

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Drug Target Review

OCTOBER 24, 2023

Live Bacterial Products (LBPs) will provide patients with a desirable, credible, safe and effective treatment option. The human microbiome, a complex ecosystem of microorganisms residing within our bodies, has emerged as a promising frontier in the quest for innovative medical treatments.

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Treatment Pharmacokinetics Pharmaceuticals Trials 122

Broad Institute

OCTOBER 4, 2023

A new small-molecule drug candidate being tested in an early-stage clinical trial aims to improve patient responses to immunotherapy. AbbVie and Calico discovered the molecule, called ABBV-CLS-484 , after TIDE researchers at Broad identified the PTPN2 gene as a promising cancer immunotherapy target in 2017.

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Small Molecule Clinical Trials Engineering Trials 128

Codon

JANUARY 21, 2024

yellow fever, malaria, and dengue), evaluating new drugs or treatments, and studying pathogenesis, the process by which a disease develops. Human challenge trials were an indispensable part of the development of the malaria vaccine, R21/Matrix-M, endorsed by the World Health Organization last October.

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The Pharma Data

DECEMBER 30, 2020

Long-term safety profile consistent with previously completed controlled trials. Treatment with AXS-07, rapidly, substantially, and durably relieved migraine pain and associated symptoms in this trial. Axsome remains on track to submit an NDA for AXS-07 in the acute treatment of migraine in the first quarter of 2021.

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The Pharma Data

AUGUST 5, 2020

Following an interim analysis of the phase 2 ELARA trial, researchers found that Kymriah met the primary endpoint of complete response rate (CRR) – a standard measure of response to therapy in FL patients. The therapy is a one-time treatment created individually for each patient using their own T cells. Source link.

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The Pharma Data

JANUARY 3, 2021

United Kingdom Medicines and Healthcare Products Regulatory Agency authorized Clinical Trial Application. With these important regulatory clearances for our first-in-human clinical trial for INZ-701 in subjects with ENPP1 deficiency, we have transitioned from a research-stage to a clinical-stage company. “We

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Clinical Trials Trials Treatment FDA 52

Drug Target Review

NOVEMBER 17, 2023

Up until 2023 around 600 NK cell clinical trials have been reported and some 200 are clinically active and still recruiting patients. The vast majority (65/74; 88%) of those trials involved NK cells without CARs; while only 12 percent were CAR-NK studies. Only three of the CAR-NK studies were for the treatment of solid tumours.

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Therapies Clinical Trials Immune Response Trials 103

Vial

JUNE 12, 2024

PROs in clinical trials are important as they capture the patient’s perspective and ensure that the impact of an intervention is comprehensively evaluated. What are PROs in clinical trials? From a regulatory standpoint, agencies like the U.S. These aspects influence a patient’s perceived health-related quality of life (QoL).

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Clinical Trials Trials FDA Clinical Research 52

The Pharma Data

DECEMBER 17, 2020

18, 2020 /PRNewswire/ — Camurus AB (NASDAQ STO: CAMX) announced today that the Swiss agency for therapeutic products, Swissmedic, has approved weekly and monthly Buvidal ® prolonged release buprenorphine for the treatment of opioid dependence in adults and adolescents from 16 years of age. LUND, Sweden , Dec. About Buvidal.

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Treatment Clinical Trials International Pharmaceutical Companies 52

Vial

DECEMBER 5, 2023

In 2017, the Food & Drug Administration (FDA) approved the first gene therapy for cancer and for inherited diseases, the first multiplex NGS panel for companion diagnostics (CDx), and the first drug targeting a genetic signature though not a disease. Research through WGS and WES has provided novel insights into biological processes.

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Clinical Trials Trials RNA Bioinformatics 52

Alta Sciences

APRIL 17, 2024

Five Promising Treatment Areas in Early-Phase Drug Development in 2024 aasimakopoulos Wed, 04/17/2024 - 15:52 Early-phase drug development is an ever-changing landscape, as emerging science leads to new promising areas of research for the treatment of human health issues.

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The Pharma Data

NOVEMBER 12, 2020

The DETOUR1 trial is the first-in-man trial of the FDA-designated Breakthrough Device, The Detour System, evaluating the safety and effectiveness of the novel procedure and device system for treating long, complex lesions in the superficial femoral artery (SFA). This press release features multimedia. About the Company: PQ Bypass, Inc.

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The Pharma Data

APRIL 7, 2022

It may also limit coverage for any future approved treatment in the class. When additional data from this new class of treatments become available, Biogen urges CMS to reconsider today’s decision for all FDA-approved amyloid-beta targeting therapies. ADUHELM is indicated for the treatment of Alzheimer’s disease.

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Treatment Therapies Disease FDA Approval 52

The Pharma Data

NOVEMBER 24, 2020

25, 2020 /CNW/ – On August 21, 2020 , Health Canada approved Lynparza® (olaparib), for the treatment of adult patients with deleterious or suspected deleterious germline and/or somatic BRCA or ATM- mutated metastatic castration-resistant prostate cancer (mCRPC) who have progressed following prior treatment with a new hormonal agent (NHA).

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Treatment DNA Trials Therapies 52

Drug Target Review

OCTOBER 2, 2024

Cell and gene therapies (CGTs) have made significant advancements in treating oncological diseases, with therapies like CAR-T cell treatments transforming cancer care. Early-phase clinical trials have shown the safety and efficacy of using stem cells for cardiac repair, encouraging further research and development.

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Therapies Disease RNA Engineering 98

The Pharma Data

AUGUST 22, 2021

The trial accrued 25 cases of symptomatic COVID-19 at the primary analysis.There were no cases of severe COVID-19 or COVID-19-related deaths in those treated with AZD7442. The trial included 5,197 participants in a 2:1 randomisation AZD7442 to placebo. The trial was conducted in 87 sites in the US, UK, Spain, France and Belgium.

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Trials Vaccine Virus Pharmacokinetics 52

The Pharma Data

FEBRUARY 8, 2021

Janssen Announces Treatment with ERLEADA ® (apalutamide) Significantly Improved Overall Survival in Patients with Metastatic Castration-Sensitive Prostate Cancer. The recruitment period for the study spanned from December 2015 to July 2017. 6] ERLEADA ® is being studied in five Phase 3 registrational clinical trials. . ‡

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Drug Target Review

JUNE 21, 2023

Strikingly, a strong genetic link between target and disease biology is emerging as a predictor for success in clinical trials. Strikingly, a strong genetic link between target and disease biology is emerging as a predictor for success in clinical trials. 2017 Mar 29;9(383):eaag1166. Sci Transl Med. medRxiv; 2023.

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The Pharma Data

JULY 21, 2021

(NYSE: PFE), today announced that they have completed recruitment for the Phase 2 trial, VLA15-221, of Lyme disease vaccine candidate, VLA15. The trial builds on previous positive Phase 2 trials and includes both adult and pediatric participants with the aim to support acceleration of the vaccine candidate’s pediatric program.

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Vaccine Disease Trials Protein Expression 52

The Pharma Data

JANUARY 11, 2021

The trial plans to enroll 99 boys aged 4 to 7 years across 55 sites in 15 countries. The agency previously granted the gene therapy Orphan Drug and Rare Pediatric Disease designations in 2017. Meanwhile, Sarepta Therapeutics reported disappointing top-line results from a phase 2 trial evaluating SRP-9001, its gene therapy for DMD.

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Therapies Trials FDA Treatment 52

New Drug Approvals

OCTOBER 12, 2024

Years later, a subgroup analysis of the trial data indicated a potential positive effect in participants who carried two copies of ApoE4 ( Abushakra et al., ALZ-801 is an advanced and markedly improved candidate for the treatment of alzheimer’s disease. 2007 ; Kocis et al., 2016 ; Abushakra et al., 2018; 32(9): 849–861. [2].

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Pharmacokinetics Small Molecule Disease Licensing 62

Drug Target Review

DECEMBER 16, 2024

Since their first approvals in 2017, autologous CAR T-cell therapies have revolutionised the treatment of blood cancers, offering hope where previous options had failed. Treatment outcomes for large B-cell lymphoma were dismal before CAR T-cell therapies.

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Therapies Clinical Trials Treatment Trials 52

New Drug Approvals

NOVEMBER 13, 2023

WO 2017/208267A1, WO 2020/250133 Al and WO 2022/024072A1, which are hereby incorporated by reference, disclose methods of use of the compound of Formula I for the treatment of Parkinson’s disease, synucleinopathies and Alzheimer’s disease (AD) respectively. amcrasto@amcrasto /////////// Ref [1].

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Treatment Disease Therapies Production 62

Drug Target Review

OCTOBER 26, 2023

1 Adeno-associated virus (AAV) vectors are the leading platform for in vivo gene delivery for the treatment of various human diseases. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial. Wang D, Tai PW, Gao G.

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Engineering Virus Therapies Immune Response 105

New Drug Approvals

SEPTEMBER 13, 2024

2] As of July 2022, it is in phase 3 clinical trials for major depressive disorder. [2] 2] Like other kappa opioid antagonists currently under clinical investigation for the treatment of major depression, its efficacy may be compromised by the countervailing activation of pro-inflammatory cytokines in microglia within the CNS. [7]

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Treatment Clinical Trials Pharmacokinetics Pharmaceuticals 62

Drug Target Review

JUNE 19, 2024

Our goal is to stand on the shoulders of giants and advance this exciting and new field of medicine, so that it can deliver valuable treatments to patients. That would provide wonderful information as we think about then going into a first-in-human clinical trial. SVRA) in April 2017. from May 2017 until December 2022.

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Therapies Small Molecule Pharmaceutical Companies Molecular Biology 84

The Pharma Data

JANUARY 3, 2021

Anecdotal feedback from Foralumab-treated patients was positive and suggests that the treatment was well-tolerated. This trial is the first to evaluate nasally administered Foralumab to improve the immune system’s fight against coronaviruses. NEW YORK and LONDON, Jan.

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Drug Target Review

APRIL 3, 2024

This multi-layered mechanism contributes to the development of immunoresistance against current standard-of-care treatments such as checkpoint inhibitors, chemotherapy, or antibody-drug conjugates (ADCs). Visugromab is a monoclonal antibody – a treatment modality that has been extensively studied and de-risked.

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Science Clinical Development Treatment DNA 105

The Pharma Data

DECEMBER 3, 2020

The TAK-620-303 (SOLSTICE) trial (NCT02931539) is a multicenter, randomized, open-label, active-controlled trial comparing eight weeks of treatment with either maribavir or investigator assigned treatment (IAT) in transplant recipients with CMV infection refractory or resistant to existing antiviral treatments (i.e.,

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Treatment Pharmaceutical Companies Therapies Virus 52

Drug Target Review

AUGUST 24, 2023

We will explore how GDF-15 impacts the tumour microenvironment and hinders the infiltration of T cells into the tumour, as well as the implications of neutralising GDF-15 to reverse its inhibitory effects and sensitise tumours to anti-PD-1 treatment. T-cell infiltration is a prerequisite for responses to checkpoint inhibitors.

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Treatment Therapies Clinical Development Cell Biology 98

The Pharma Data

JANUARY 25, 2021

agree to place handheld Ceribell Rapid Response EEG systems in hospitals in connection with its Phase 3 RSE clinical trial. Within the RAISE trial, it will allow clinicians to rapidly establish a diagnosis of nonconvulsive SE in the absence of immediate availability of conventional EEG. About the RAISE Trial. RADNOR, Pa.–(

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Clinical Trials Trials Hospitals Pharmaceutical Companies 40

Drug Target Review

MAY 18, 2023

Tedesco Distinguished Chair of Paediatric Haematology and Oncology, are collaborating as co-principal investigators on a new clinical trial, which has been awarded a $2.3 The trial represents an early-stage effort to determine the viability of this approach. million grant from the National Heart, Lung and Blood Institute.

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The Pharma Data

MARCH 4, 2021

Together we generated positive Phase 2 ELEKTRA study data, and as a result, soticlestat is poised to enter two pivotal trials,” said Andy Plump, M.D., Our goal is to one day bring new treatment options that provide greater seizure control, tolerability and function to DS and LGS patients around the world.”.

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Treatment Clinical Trials Pharmaceutical Companies Trials 52

The Pharma Data

AUGUST 9, 2020

In the HIBISCUS I induction study, in people without prior anti-tumour necrosis factor (anti-TNF) treatment, etrolizumab met the primary endpoint. In contrast, the HIBISCUS II induction study, which also included people without prior anti-TNF treatment, did not meet its primary endpoint.

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Clinical Trials Disease Treatment Trials 52

The Pharma Data

AUGUST 5, 2020

Ofatumumab is a targeted B-cell therapy that delivers superior efficacy with a similar safety profile when compared with teriflunomide, a commonly prescribed oral treatment for multiple sclerosis 1 . 10% of patients 1,3. ” These data were published in the August 6, 2020 issue of The New England Journal of Medicine.

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Trials Treatment Disease Therapies 52

The Pharma Data

MARCH 16, 2022

(Nasdaq: BIIB) announced that The Journal of Prevention of Alzheimer’s Disease (JPAD) today published a peer-reviewed manuscript detailing data from the pivotal Phase 3 EMERGE and ENGAGE trials for ADUHELM ® (aducanumab-avwa) 100 mg/mL injection for intravenous use in early Alzheimer’s disease. In June 2021, the U.S.

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