Drug Discovery World

SEPTEMBER 16, 2024

A new study has demonstrated that the frequency of second primary malignancies (SPMs) is no higher with CAR-T cell therapies than with previous standard-of-care strategies. The authors found that T cell malignancies represented a small minority of adverse events at just 1.5%. in the three years after therapy.

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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).

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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.

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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.

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Drug Discovery World

MAY 24, 2023

Moderna has reported encouraging interim data from its Phase I/II trial of mRNA-3927, an mRNA therapy for rare metabolic disorder propionic acidemia (PA). The data was presented at the 2023 American Society of Gene & Cell Therapy (ASGCT) Annual Meeting.

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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.

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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.

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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.

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Drug Discovery World

DECEMBER 19, 2022

Researchers have found that a topical gene therapy gel helps heal wounds in patients with life-threatening blistering skin disease epidermolysis bullosa (EB). . It’s an intensely painful condition, and medical treatment for it has been limited largely to palliative care. . One trial participant was Vincenzo Mascoli, aged 22.

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Drug Discovery World

NOVEMBER 1, 2023

Axicabtagene ciloleucel, Kite Two indirect comparative analyses of CAR T-cell therapy axicabtagene ciloleucel vs. bispecific antibodies epcoritamab and glofitamab, for relapsed/refractory diffuse large B-Cell lymphoma, showed improved progression-free survival (PFS). Median PFS was 13.8 months and median duration of response (DoR) was 15.5

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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.

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Drug Discovery World

NOVEMBER 14, 2023

A Phase Ib trial has shown low-density lipoprotein cholesterol (LDL-C) reductions up to 55% and blood PCSK9 protein reductions up to 84% after a single infusion of Verve Therapeutics’ Verve-101. Since the mutation only affects a single gene, it makes it a prime candidate for genome editing treatments.

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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.

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Drug Discovery World

NOVEMBER 30, 2023

The reports were received from clinical trials and postmarketing adverse event (AE) data sources. The FDA has cautioned that patients and clinical trial participants receiving treatment with these products should be monitored life-long for new malignancies.

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Drug Discovery World

JULY 19, 2023

The primary endpoint of iADRS measures cognition and activities of daily living such as managing finances, driving, engaging in hobbies, and conversing about current events. 52% of participants completed their course of treatment by one year and 72% completed by 18 months as a result of achieving plaque clearance.

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Drug Discovery World

SEPTEMBER 6, 2022

In recent years the advanced therapy industry has been the most significant disruptor in medicine. Cell and gene therapies are not only transforming the way diseases are treated but are also aiming to cure entirely new patient populations. ViaCyte, Beam, and Verve are all due to begin gene editing trials this year.

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Drug Discovery World

OCTOBER 1, 2024

As further news comes out about the benefits that the diabetes and weight-loss drug Ozempic offers patients, it’s hard not to get caught up in the hype surrounding a therapy many are calling a ‘wonder drug’ Of course, it’s not the first time the pharmaceutical industry has produced a therapy that entered into the public discourse.

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Drug Discovery World

NOVEMBER 24, 2023

AstraZeneca has announced that the National Institute for Health and Care Excellence (NICE) has recommended Imfinzi (durvalumab) in combination with gemcitabine and cisplatin (chemotherapy) for NHS use in England and Wales for the first-line treatment of adults with locally advanced, unresectable, or metastatic biliary tract cancer (BTC).

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Drug Discovery World

MAY 8, 2024

The ORCA-V1 trial was led by Achieve Life Sciences, and co-led by an investigator from Massachusetts General Hospital. In their previous clinical trial, the research team found that cytisinicline helped people to quit smoking traditional cigarettes. “The For cytisinicline treatment compared to placebo, 31.8% versus 15.1%

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Drug Discovery World

AUGUST 2, 2024

This translates to a placebo-adjusted weight loss from baseline of 7.4%, 9.2%, 11.3%, and 13.10% in the treatment groups. In a Phase I trial of an oral tablet formulation of VK2735, up to 3.3% The company plans to initiate a Phase II trial with the oral formulation of VK2735 in obesity later this year.

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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.

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Drug Discovery World

JULY 12, 2023

There is an urgent need for new therapies that safely and effectively eliminate congestion and improve clinical outcomes, and we believe DSR has the potential to be a disease-modifying heart failure therapy for these patients.” MOJAVE study design The non-randomised cohort consists of three eligible patients who will be treated with DSR 2.0,

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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.

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Drug Discovery World

APRIL 3, 2024

million from the Spanish Research Agency (AEI), under the call for grants for Public-Private Collaboration Projects, to accelerate the development of an innovative treatment aimed at controlling disabling and lethal haemorrhage. Our initial focus will be on the US FDA.

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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%.

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Drug Discovery World

MAY 10, 2024

291 patients were randomised to treatment either with evenamide or placebo as add-on to their current antipsychotic therapy. There was no difference in the incidence of CNS, psychiatric, gastrointestinal or other adverse events between evenamide and placebo. points, compared to 7.6

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PPD

AUGUST 7, 2024

Clinical trials are the backbone of medical research, enabling the development of new treatments and therapies that can improve patient outcomes. However, conducting successful clinical trials requires efficient communication and coordination among various stakeholders.

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Drug Discovery World

JUNE 12, 2024

Immune-checkpoint inhibitor (ICI) therapy has transformed treatment outcomes for RCC. However, ICIs can also trigger immune-related adverse events and may not benefit all patients. If successful, the data we generate could help us learn more about the biology of renal cell carcinoma and its treatments.”

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Drug Discovery World

SEPTEMBER 6, 2022

DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. billion, compared to $19.9 Cancer is king .

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Conversations in Drug Development Trends

JULY 8, 2024

By: Lisle Kingery, PhD, Therapeutic Strategy Lead, Neuroscience With depression rates at an all-time high and limited new treatments available, depression clinical trials are more important than ever. Treatment Treatment can take various forms, including therapy, medication, a combination of both, or a clinical trial.

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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. relative reduction in liver fat content (LFC), with up to 55.6%

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Vial

DECEMBER 8, 2023

With technological innovation and scientific research propelling the development of new therapies, anticipation for the future is high. As we look forward to 2024, several groundbreaking therapies are poised to radically change the landscape of ophthalmology, providing hope and improved treatment options for patients with ocular conditions.

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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

APRIL 21, 2023

The event showcased the very latest innovations in cancer therapies, from personalised mRNA vaccines to bispecific antibodies. This is according to results of a Phase I/II clinical trial presented at the AACR Annual Meeting 2023 in Florida.

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Drug Discovery World

APRIL 25, 2023

University of California researchers have dosed the second participant in their clinical trial to identify a potential cure for HIV utilising CAR-T cell therapy. The trial is the first-in-human clinical study investigating the duo CAR-T cell therapy for the treatment of HIV. “We In 2021 there were an estimated 1.5

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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.

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Drug Discovery World

JULY 8, 2024

argenx shared trial and real-world data on Vyvgart (efgartigimod alfa) in patients with generalised myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive at the European Academy of Neurology (EAN) Annual Meeting. It is also approved for IV use in adults with primary immune thrombocytopenia (ITP) in Japan.

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