Disease, Events, Treatment and Trials

MHRA approves semaglutide to reduce risk of cardiovascular events

Drug Discovery World

JULY 31, 2024

The GLP-1 receptor agonist is already approved for use in the treatment of obesity and for weight management, to be used alongside diet, physical activity and behavioural support. In a trial of over 17,600 participants, Wegovy significantly reduced the risk of MACE by 20%, which occurred in 6.5%

Disease

Disease Production Trials Treatment

Early trial data supports neurodegenerative disease-modifying drug

Drug Discovery World

MARCH 15, 2024

Biotechnology company Allyx Therapeutics has reported positive Phase Ib clinical data for its lead compound, ALX-001, a first-in-class, synapse-targeted, disease-modifying oral therapy in development for neurodegenerative diseases. All doses were well-tolerated and there were no serious adverse events.

Disease

Disease Trials Small Molecule Pharmacokinetics

Positive interim data from Huntington’s disease trial

Drug Discovery World

JUNE 23, 2023

PTC Therapeutics has shared interim data from the 12-week portion of the PIVOT-HD Phase II study of PTC518 in Huntington’s disease (HD) patients. PTC518 treatment was also demonstrated to be well tolerated, with no treatment-related serious adverse events, no reports of peripheral neuropathy or dose-limiting toxicities.

Disease

Disease Trials Treatment Drugs

Diabetic eye disease: The urgent need for early, non-invasive treatments

Drug Discovery World

MAY 15, 2024

Dr Catherine Beech , CEO at Exonate and Dr Pete Adamson , CSO at Breye Therapeutics discuss the need for better therapies to treat diabetic retinal disease and current progress in the field. Despite affecting such a large subset of diabetic patients, treatment options are limited to repeated, invasive intravitreal injections to the eye.

Disease

Disease Treatment Therapies Pharmaceuticals

Semaglutide reduces major cardiovascular events by 20%

Drug Discovery World

NOVEMBER 14, 2023

Semaglutide 2.4mg (Wegovy), a drug approved for weight loss, can reduce the risk of major adverse cardiovascular events (MACE) by 20%, according to new data released by Novo Nordisk. Participants in the trial who received semaglutide lost an average of 9.4% of total body weight which was sustained throughout the trial.

Trials

Trials Disease Treatment Research

Patients report improvement in haemorrhoid treatment trial

Drug Discovery World

JUNE 21, 2023

Citius Pharmaceuticals has revealed positive results from its Phase IIb Study of Halo-Lido (CITI-002) for the treatment of haemorrhoids. Moreover, there were no reported significant adverse events and CITI-002 was well tolerated by patients in the study.

Trials

Trials Treatment Clinical Trials Clinical Development

Do JAK inhibitors increase the risk of serious adverse events?

Drug Discovery World

JANUARY 2, 2024

South Korean researchers have found that the use of Janus kinase (JAK) inhibitors as a treatment for atopic dermatitis (AD) is not associated with a greater risk of serious adverse events. However, the FDA also issued a boxed warning to include the risk of major adverse events. appeared first on Drug Discovery World (DDW).

FDA

FDA Treatment Disease Trials

Gene therapy for Parkinson’s disease progresses to Phase II

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.

Therapies

Therapies Disease Medical Schools Trials

Donanemab significantly slowed decline in early Alzheimer’s disease trial

Drug Discovery World

JULY 19, 2023

Eli Lilly has announced positive results of the TRAILBLAZER-ALZ 2 Phase III study showing that donanemab significantly slowed cognitive and functional decline in people with early symptomatic Alzheimer’s disease.

Disease

Disease Trials Clinical Trials Therapies

Phase II trial investigates allogeneic CAR-T for large B-cell lymphoma

Drug Discovery World

JUNE 21, 2024

Allogene Therapeutics and Foresight Diagnostics have announced the initiation of a Phase II trial evaluating the use of cemacabtagene ansegedleucel (cema-cel) as part of the first line treatment regimen for newly diagnosed large B-cell lymphoma (LBCL) patients who are likely to relapse.

Trials

Trials Compliance Clinical Trials Doctors

Phase Ib/IIa trial in diabetic macular oedema successfully completed

Drug Discovery World

DECEMBER 12, 2023

Exonate, an mRNA therapy company focused on treatments for diabetic complications, has announced that its lead ophthalmology asset, EXN407, has achieved its prespecified endpoints in a Phase Ib/IIa study. During the trial, EXN407 met all safety and pharmacokinetic parameters and displayed encouraging signals of biological activity.

Small Molecule

Small Molecule Trials Pharmacokinetics Treatment

Investigating potential for an oral treatment in diabetic retinopathy

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).

Treatment

Treatment Clinical Trials Pharmacokinetics Trials

Gene therapy proved to be safe in patients with Parkinson’s disease

Drug Discovery World

APRIL 19, 2024

Askbio’s AB-1005 (also known as AAV2-GDNF), an investigational gene therapy for the treatment of Parkinson’s disease, was well tolerated with no serious adverse events in a Phase Ib clinical trial. The data were presented at the American Academy of Neurology 2024 Annual Meeting in Denver, Colorado, USA.

Therapies

Therapies Disease Clinical Trials Trials

Optimizing Patient-Driven Site Engagement in Rare Disease Clinical Trials

Conversations in Drug Development Trends

AUGUST 29, 2024

By: Nathan Chadwick, Therapeutic Strategy Lead, Rare Disease Over the last few years in clinical trials, particularly within the rare disease community, a notable shift is underway, where patients and caregivers are taking the lead in reaching out to clinical trial sites rather than the other way around.

Clinical Trials

Clinical Trials Trials Disease Drug Development

J&J presents successful data from plaque psoriasis trial

Drug Discovery World

MARCH 13, 2024

Johnson & Johnson has announced the first data from FRONTIER 2, the long-term extension of the Phase IIb FRONTIER 1 clinical trial evaluating JNJ-2113, the first and only investigational targeted oral peptide designed to block the IL-23 receptor. Across JNJ-2113 treatment groups, 58.6% at 16 weeks and 76.2% at 16 weeks and 76.2%

Trials

Trials Clinical Trials Treatment Licensing

Early trial success for Dravet syndrome and ALS therapeutic

Drug Discovery World

AUGUST 22, 2023

Bloom Science has announced positive topline data from a Phase I clinical trial of BL-001 , a potential first-in-class therapeutic the company is developing for Dravet syndrome and amyotrophic lateral sclerosis (ALS). BL-001 shows promise as a novel treatment option that can change lives.

Trials

Trials Clinical Trials Science Clinical Development

Daily statin reduces the risk of cardiovascular disease in HIV

Drug Discovery World

APRIL 13, 2023

A National Institutes of Health (NIH) clinical trial was stopped early because a daily statin medication was found to reduce the increased risk of cardiovascular disease among people living with HIV. Advances in HIV treatment have drastically reduced AIDS-related complications and deaths.

Disease

Disease Clinical Trials Trials Virus

Breaking clinical trial news from ESMO 2023

Drug Discovery World

NOVEMBER 1, 2023

However, it was associated with higher rates of grade ≥3 cytokine release syndrome and neurological events. Datopotamab deruxtecan, Daiichi Sankyo and AstraZeneca The companies shared results from multiple trials of their TROP2 directed DXd antibody drug conjugate (ADC) datopotamab deruxtecan (Dato-DXd). months compared to 4.9

Clinical Trials

Clinical Trials Trials Disease Treatment

First base editing therapeutic for T-ALL/T-LL enters trials

Drug Discovery World

SEPTEMBER 8, 2023

The first quadruplex-edited, allogeneic CAR-T cell therapy candidate has entered clinical trials in the US. The first patient has been treated with Beam Therapeutics’ BEAM-201 as part of a Phase I/II clinical study for the treatment of relapsed/refractory T-cell acute lymphoblastic leukaemia/T-cell lymphoblastic lymphoma (T-ALL/T-LL). “As

Trials

Trials Clinical Trials Engineering Therapies

Therapy represents ‘new treatment paradigm’ for non-small cell lung cancer

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%

Treatment

Treatment Therapies Clinical Trials Trials

Semaglutide could also benefit patients with heart failure

Drug Discovery World

AUGUST 23, 2024

A new study indicates that anti-obesity drug semaglutide helps prevent cardiac events among overweight people who have cardiovascular disease, even if they also have heart failure. whose hearts did not pump blood around the body properly) by a clinician at the start of the trial. Read the full paper in The Lancet.

Disease

Disease Trials Clinical Trials Regulations

Exclusive event: An overview of single-cell RNA sequencing as a relevant tool for drug discovery

Drug Discovery World

DECEMBER 1, 2022

Extensive manipulation, mechanical force and labelling methods used to enrich cells activate transcriptional programs that mask molecular profiles truly significant to any underlying disease. Director, Application Development, LevitasBio will present the event. The event will take place on December 14, 2022 at 4PM GMT, 8AM PT.

RNA

RNA Clinical Trials Drugs Trials

FDA approves first gene therapies for sickle cell disease (with a warning)

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.

FDA Approval

FDA Approval Therapies FDA Disease

Initially positive findings from AML treatment study

Drug Discovery World

DECEMBER 23, 2022

A study by Ellipses Pharma, a global drug development company focused on accelerating the development of new oncology treatments, has presented preliminary data from the first in human Phase I/II trial of EP0042, a dual FLT-3 an Aurora kinase inhibitor, in patients with acute myeloid leukaemia (AML). . The data . Official comments

Treatment

Treatment Clinical Trials Drug Development Trials

The killer instinct: Using investigational natural killer therapy to treat Alzheimer’s

Drug Discovery World

FEBRUARY 21, 2024

In October 2023, pharmaceutical company NKGen Biotech released data from its Phase I clinical trial on the use of its investigational natural killer therapy, SNK01, to treat patients with Alzheimer’s disease. RA: What are the clinical benefits of using the patient’s own cells as a treatment?

Therapies

Therapies Trials Treatment Disease

Sequana announces first patient enrolled in heart failure trial

Drug Discovery World

JULY 12, 2023

Sequana Medical, a company targeting fluid overload in liver disease, heart failure and cancer, has enrolled the first patient in its MOJAVE study. Following four weeks of treatment, there is a three-month safety follow-up period.

Trials

Trials Disease Therapies Treatment

Positive results announced for COPD therapy

Drug Discovery World

APRIL 11, 2024

Biopharmaceutical company EpiEndo has completed a Phase IIA clinical trial for its therapy addressing chronic obstructive pulmonary disease (COPD). Damage of this barrier is implicated in several chronic inflammatory diseases, including COPD. ” 1: The Global Economic Burden of Noncommunicable Diseases.

Therapies

Therapies Clinical Trials Disease Trials

Encouraging results for RESP-X in ongoing Phase I clinical trial

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.

Clinical Trials

Clinical Trials Trials Laboratories Clinical Research

Vaccine successfully lowers cholesterol in preclinical studies

Drug Discovery World

FEBRUARY 27, 2024

The results support the continued clinical development of VXX-401 as a candidate for the treatment of hypercholesterolemia and prevention of atherosclerotic cardiovascular disease. High LDL-C is a major risk factor for coronary heart disease, heart attack, and stroke, and atherosclerosis is the leading cause of disease burden globally.

Vaccine

Vaccine Clinical Trials Disease Clinical Development

GSK announces positive data for gonorrhoea antibiotic

Drug Discovery World

APRIL 18, 2024

First results from the EAGLE-1 Phase III trial for gepotidacin show that two oral doses of the antibiotic displayed superior success rates (92.6%) when compared to the combination treatment of ceftriaxone and oral azithromycin, which saw a 91.2% The imperative for innovative treatments has never been clearer. success rate.

Trials

Trials DNA Treatment Regulations

Trial success for first new approach to atrial fibrillation for 20 years

Drug Discovery World

MARCH 15, 2023

The primary endpoint has been achieved in a Phase II trial of AP30663, a first-in-class SK ion channel inhibitor for conversion of atrial fibrillation (AF) to normal sinus rhythm. Acesion Pharma enrolled 63 patients with a current episode of AF in the trial and tested two intravenous doses of AP30663, 3 and 5mg/kg, compared to placebo.

Trials

Trials Small Molecule Clinical Trials Treatment

First-line AZ treatment for adults with biliary tract cancer approved

Drug Discovery World

FEBRUARY 9, 2023

The Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorisation to AstraZeneca for Imfinzi (durvalumab) in combination with gemcitabine and cisplatin in Great Britain for use as a first-line treatment of adults with locally advanced, unresectable or metastatic biliary tract cancer (BTC). versus 11.5%).

Treatment

Treatment Clinical Research International FDA

Therapeutic implications of crocin in Parkinson's disease: A review of preclinical research

Chemical Biology and Drug Design

MAY 11, 2023

The figure depicts the neuroprotective role of crocin in Parkinson's disease. Abstract Parkinson's disease is among the most common forms of neurodegenerative illness, with present treatment being primarily symptomatic and frequently coming with substantial adverse effects.

Disease

Disease Research Clinical Trials Treatment

This week in drug discovery (15-19 April)

Drug Discovery World

APRIL 19, 2024

The headline news this week all concerns clinical trials for new drugs that could have a significant impact on future patient treatment, including trials testing therapeutics for ALS, cystic fibrosis, gonorrhoea, TB and Parkinson’s.

Clinical Trials

Clinical Trials Vaccine Trials Drugs

The Next Generation of Vaccine Mega Trials

PPD

JUNE 24, 2024

In the realm of vaccine development, mega trials — studies enrolling 5,000 subjects or more — have been instrumental in the fight against many pathogens, including influenza, rotavirus, malaria, RSV and most recently in the rapid development of vaccines against COVID-19.

Vaccine

Vaccine Trials Clinical Trials Laboratories

Promising new drug for chronic blood clotting disorder

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.

Trials

Trials Clinical Trials Drugs Therapies

Before Clinical Trials: The Long Game of Rare Disease Research

Conversations in Drug Development Trends

FEBRUARY 28, 2023

95% of rare diseases don’t have a treatment approved and won’t anytime soon. Miraculously, there was an approved treatment. Over the next few decades, that drug made its way through clinical trials, securing approval in 2007—just 36 months before Rob was diagnosed in 2010. It saved my life.”

Clinical Trials

Clinical Trials Disease Trials Research

Weight loss drug reduces sleep apnoea severity by 62%

Drug Discovery World

JUNE 26, 2024

on the apnoea-hypopnea index (AHI) compared to placebo in the SURMOUNT-OSA Phase III clinical trials. The drug was tested for the treatment of moderate-to-severe obstructive sleep apnoea (OSA) in adults with obesity, with and without positive airway pressure (PAP) therapy. “OSA is a very common, under recognised disease. .

Drugs

Drugs Clinical Trials Disease Treatment

This week in drug discovery (29 July – 2 August)

Drug Discovery World

AUGUST 2, 2024

GLP-1 receptor agonists have dominated the headlines this week, with data revealed on new potential beneficial effects of treatment with semaglutide and dual receptor agonists like pemvidutide. of subjects achieving LFC normalisation after 12 weeks of treatment. of subjects achieving LFC normalisation after 12 weeks of treatment.

Clinical Trials

Clinical Trials Drugs Treatment Disease

Renaissance Pharma licenses Hu14.18 for neuroblastoma

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%.

Licensing

Licensing Licensing Hospitals Therapies

The Value of Patient-Designed Clinical Trial Concierge Service

PPD

OCTOBER 9, 2023

“Patient-centric” is used frequently to describe today’s clinical trial and health care landscape. The industry applauds that point of view and strives to include a patient-centric approach in all aspects of clinical trial designs. Education on the disease state and regular reinforcement.

Clinical Trials

Clinical Trials Trials Compliance International

Closing the translation gap with patient-derived organoids

Drug Discovery World

OCTOBER 2, 2024

This advertorial appeared in DDW Volume 25 – Issue 4, Fall 2024 Read the digital issue Earlier this year, DDW hosted a webinar, ‘From patient to petri dish: Replicating disease with patient-derived organoids’, supported by HUB Organoids.

Assay Development

Assay Development Clinical Trials Laboratories Disease

QL Biopharm announces new obesity drug results

Drug Discovery World

SEPTEMBER 13, 2024

QL Biopharm, a clinical stage biopharmaceutical company developing biologic drugs for the treatment of metabolic diseases, has shared Phase Ic clinical data for its lead drug candidate ZT002. The majority of adverse events (AEs) were mild or moderate in severity, no serious AEs were reported in the ZT002 treatment groups.

Pharmacokinetics

Pharmacokinetics Drugs Treatment FDA

A Reflection on Shared Moments of Strength and Progress in the Rare Disease Communities

Conversations in Drug Development Trends

APRIL 5, 2024

For others, this rare day is aptly the day we honor rare disease communities. Observed annually on the last day of February since 2008, Rare Disease Day has grown into a global movement for raising awareness, promoting research, and advocating for improved access to rare disease treatments and support services.

Disease

Disease Clinical Trials Trials Treatment

MHRA approves semaglutide to reduce risk of cardiovascular events

Early trial data supports neurodegenerative disease-modifying drug

Trending Sources

Positive interim data from Huntington’s disease trial

Diabetic eye disease: The urgent need for early, non-invasive treatments

Semaglutide reduces major cardiovascular events by 20%

Patients report improvement in haemorrhoid treatment trial

Do JAK inhibitors increase the risk of serious adverse events?

Gene therapy for Parkinson’s disease progresses to Phase II

Donanemab significantly slowed decline in early Alzheimer’s disease trial

Phase II trial investigates allogeneic CAR-T for large B-cell lymphoma

Phase Ib/IIa trial in diabetic macular oedema successfully completed

Investigating potential for an oral treatment in diabetic retinopathy

Gene therapy proved to be safe in patients with Parkinson’s disease

Optimizing Patient-Driven Site Engagement in Rare Disease Clinical Trials

J&J presents successful data from plaque psoriasis trial

Early trial success for Dravet syndrome and ALS therapeutic

Daily statin reduces the risk of cardiovascular disease in HIV

Breaking clinical trial news from ESMO 2023

First base editing therapeutic for T-ALL/T-LL enters trials

Therapy represents ‘new treatment paradigm’ for non-small cell lung cancer

Semaglutide could also benefit patients with heart failure

Exclusive event: An overview of single-cell RNA sequencing as a relevant tool for drug discovery

FDA approves first gene therapies for sickle cell disease (with a warning)

Initially positive findings from AML treatment study

The killer instinct: Using investigational natural killer therapy to treat Alzheimer’s

Sequana announces first patient enrolled in heart failure trial

Positive results announced for COPD therapy

Encouraging results for RESP-X in ongoing Phase I clinical trial

Vaccine successfully lowers cholesterol in preclinical studies

GSK announces positive data for gonorrhoea antibiotic

Trial success for first new approach to atrial fibrillation for 20 years

First-line AZ treatment for adults with biliary tract cancer approved

Therapeutic implications of crocin in Parkinson's disease: A review of preclinical research

This week in drug discovery (15-19 April)

The Next Generation of Vaccine Mega Trials

Promising new drug for chronic blood clotting disorder

Before Clinical Trials: The Long Game of Rare Disease Research

Weight loss drug reduces sleep apnoea severity by 62%

This week in drug discovery (29 July – 2 August)

Renaissance Pharma licenses Hu14.18 for neuroblastoma

The Value of Patient-Designed Clinical Trial Concierge Service

Closing the translation gap with patient-derived organoids

QL Biopharm announces new obesity drug results

A Reflection on Shared Moments of Strength and Progress in the Rare Disease Communities

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