Disease, Events and Trials - Drug Discovery Digest

MHRA approves semaglutide to reduce risk of cardiovascular events

Drug Discovery World

JULY 31, 2024

The approval means that semaglutide is the first weight loss drug to be prescribed to prevent cardiovascular events in people with established cardiovascular disease and a Body Mass Index (BMI) higher or equal to 27kg/m2. of participants who received Wegovy compared to 8% of participants who received placebo.

Disease

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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

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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

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

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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

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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

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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

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

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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

Phase Ib/IIa trial in diabetic macular oedema successfully completed

Drug Discovery World

DECEMBER 12, 2023

During the trial, EXN407 met all safety and pharmacokinetic parameters and displayed encouraging signals of biological activity. We are excited to progress to the Phase II trial next year and welcome enquiries by potential partners for the programme.”

Small Molecule

Small Molecule Trials Pharmacokinetics Treatment

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. Adverse drug events observed in the study were like those in the general population taking statin therapy.

Disease

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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).

Trials

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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

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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

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Ozempic Lowers Odds for Death, Illness in People With Kidney Disease and Type 2 Diabetes

Drugs.com

MAY 24, 2024

FRIDAY, May 24, 2024 -- Ozempic provides a wide variety of health benefits for people with kidney disease and type 2 diabetes, a major clinical trial has found. The drug significantly reduces the risk of severe kidney events, heart problems and.

Disease

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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. at 16 weeks and 76.2% Across JNJ-2113 treatment groups, 58.6%

Trials

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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

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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. Specific numerical values (steps) denote the severity of NPDR, with 47-53 describing the moderately severe disease.

Disease

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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 primary objectives of the Phase I portion of the trial are the assessment of safety, tolerability, and identification of the recommended Phase II dose and lymphodepletion regimen.

Trials

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Patients report improvement in haemorrhoid treatment trial

Drug Discovery World

JUNE 21, 2023

Moreover, there were no reported significant adverse events and CITI-002 was well tolerated by patients in the study. Following the 21 st Century Cures Act, higher emphasis is placed on using Patient Reported Outcome (PRO) instruments in clinical trials.

Trials

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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. Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited.”

FDA Approval

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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

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Semaglutide reduces risk of all-cause death

Drug Discovery World

SEPTEMBER 3, 2024

Semaglutide has been found to reduce the rates of all-cause death, including as a result of Covid-19, in those who are overweight or have obesity and established cardiovascular disease without diabetes. Of the 833 deaths in trial participants, 58% were CV related, and 42% were non-CV related. on semaglutide vs 3.1% on placebo).

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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. The post Sequana announces first patient enrolled in heart failure trial appeared first on Drug Discovery World (DDW).

Trials

Trials Disease Therapies Treatment

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

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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

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

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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

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. 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. Uplifting Athletes (UA) is on a mission to change that. But what was truly remarkable?

Clinical Trials

Clinical Trials Disease Trials Research

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

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

Everything You Need to Know About Virtual Clinical Trials

Quanticate

APRIL 12, 2024

The landscape of clinical research is undergoing major changes with virtual clinical trials driving these transformations. These innovative trials promise to revolutionise the way medical research is conducted, making it more accessible, efficient, and patient-centric than ever before.

Clinical Trials

Clinical Trials Trials Clinical Research Disease

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. Why is this significant, and why are there currently no approved disease-modifying therapies for these patients?

Therapies

Therapies Treatment Trials Disease

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. The post This week in drug discovery (15-19 April) appeared first on Drug Discovery World (DDW).

Clinical Trials

Clinical Trials Vaccine Drugs Trials

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

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% success rate.

Trials

Trials DNA Treatment Regulations

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

Using Predictive Intelligence to Uncover the Rare Disease Patient Journey

Fierce BioTech

SEPTEMBER 20, 2023

In this podcast interview, Dan Fisher, managing director and practice lead for IPM.ai, explains the use of artificial intelligence for stitching together key medical events that may happen over a r | IPM.ai helps brands assess a unique patient population, accelerate clinical trial recruitment, and optimize commercial outcomes

Clinical Trials

Clinical Trials Disease Trials

Data confirms that semaglutide lowers blood pressure

Drug Discovery World

JUNE 24, 2024

A weekly dose of semaglutide 2.4mg significantly reduced the risk of major adverse cardiovascular events (MACEs) in people with overweight or obesity and cardiovascular disease but not diabetes, regardless of blood sugar level, according to a clinical trial. Semaglutide is a glucagon-like peptide-1 (GLP-1) receptor agonist.

Clinical Trials

Clinical Trials Trials International FDA

ALXN1840 FoCus Phase III trial in Wilson disease met primary.

The Pharma Data

AUGUST 30, 2021

Positive high-level results from the FoCus Phase III trial in Wilson disease showed ALXN1840 met the primary endpoint with a statistically significant improvement in daily mean copper mobilisation from tissues, demonstrating superiority compared with standard-of-care (SoC) treatments.

Disease

Disease Trials Therapies Treatment

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

Relieve Caregiver Burden, Improve Neurodegenerative Clinical Trials

PPD

MAY 18, 2023

For neurodegenerative disease research, the care partner, or caregiver, is equally important as the person with dementia. Even with stronger awareness of studies, drug developers must contend with the inherent lack of trust between the dementia community and clinical trials. There’s little time to lose.

Clinical Trials

Clinical Trials Trials Clinical Research Doctors

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

Drug Discovery World

AUGUST 2, 2024

MHRA approves semaglutide to reduce risk of cardiovascular events The UK Medicines and Healthcare products Regulatory Agency (MHRA) has approved a new indication for semaglutide (Wegovy) to reduce the risk of overweight and obese adults suffering from heart attacks or stroke. relative reduction in liver fat content (LFC), with up to 55.6%

Clinical Trials

Clinical Trials Drugs Treatment Disease

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

Semaglutide reduces major cardiovascular events by 20%

Do JAK inhibitors increase the risk of serious adverse events?

Donanemab significantly slowed decline in early Alzheimer’s disease trial

Gene therapy for Parkinson’s disease progresses to Phase II

Optimizing Patient-Driven Site Engagement in Rare Disease Clinical Trials

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

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

Phase Ib/IIa trial in diabetic macular oedema successfully completed

Daily statin reduces the risk of cardiovascular disease in HIV

Early trial success for Dravet syndrome and ALS therapeutic

Breaking clinical trial news from ESMO 2023

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

Ozempic Lowers Odds for Death, Illness in People With Kidney Disease and Type 2 Diabetes

J&J presents successful data from plaque psoriasis trial

Semaglutide could also benefit patients with heart failure

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

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

Patients report improvement in haemorrhoid treatment trial

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

The Next Generation of Vaccine Mega Trials

Semaglutide reduces risk of all-cause death

Sequana announces first patient enrolled in heart failure trial

Vaccine successfully lowers cholesterol in preclinical studies

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

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

Positive results announced for COPD therapy

Before Clinical Trials: The Long Game of Rare Disease Research

The Value of Patient-Designed Clinical Trial Concierge Service

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

Everything You Need to Know About Virtual Clinical Trials

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

This week in drug discovery (15-19 April)

Promising new drug for chronic blood clotting disorder

GSK announces positive data for gonorrhoea antibiotic

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

Using Predictive Intelligence to Uncover the Rare Disease Patient Journey

Data confirms that semaglutide lowers blood pressure

ALXN1840 FoCus Phase III trial in Wilson disease met primary.

Closing the translation gap with patient-derived organoids

Relieve Caregiver Burden, Improve Neurodegenerative Clinical Trials

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

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