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.

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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.

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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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Chemical Biology and Drug Design

FEBRUARY 13, 2024

Alzheimer's disease (AD) is a common age-related neurodegenerative brain disorder characterized by the impairment in memory and other cognitive functions. Abstract Alzheimer's disease (AD) is a common age-related neurodegenerative brain disorder characterized by the impairment in memory and other cognitive functions.

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Drug Discovery World

MARCH 6, 2023

The post Rhythm Pharmaceuticals buys biotech focused on rare disease appeared first on Drug Discovery World (DDW). Biopharmaceutical subsidiary Rhythm Pharmaceuticals Netherlands has acquired Xinvento, a Netherlands-based biotech company focused on developing therapies for congenital hyperinsulinism (CHI).

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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.

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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

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.

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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.

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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 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.

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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 .

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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.

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Drug Discovery World

JUNE 26, 2024

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. In the primary endpoint, tirzepatide reduced moderate-to-severe OSA severity by up to 62.8% (about 30 fewer events per hour).

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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.

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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%).

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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?

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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. COPD currently has limited treatment options despite being a leading cause of death globally.

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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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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.

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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.

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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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PLOS: DNA Science

MARCH 28, 2024

A new drug has entered the arsenal against Duchenne muscular dystrophy (DMD), a genetic disease that affects boys and is challenging to treat. FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. In 2023, two gene-based treatments became available.

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Drug Discovery World

JANUARY 23, 2023

Over the three days of Advanced Therapies Week (ATW) 2023 in Miami, Florida, US, from 18 to 20 January, DDW’s Megan Thomas heard from attendees about what they were most looking forward to learning from the event, as well as why they think the potential of cell and gene therapies outweighs the obstacles that must be overcome to achieve it.

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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. News round-up for 15-19 April by DDW Digital Content Editor Diana Spencer.

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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

JUNE 20, 2023

One analysis from AstraZeneca’s ZORA study showed that discontinuation of RAASi treatment following a HK episode in cardiorenal patients with chronic kidney disease (CKD) and/or heart failure (HF) was still common in clinical practice in the US and Japan despite guidelines recommending maintained treatment.

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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. and three-year event-free (EFS) of 73.7%. and three-year event-free (EFS) of 73.7%.

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Drug Discovery World

DECEMBER 14, 2022

A new study has demonstrated the ability of a novel peptide to selectively detect and neutralise an early molecular trigger of Alzheimer’s and other neurodegenerative diseases. . Early diagnosis and treatment for Alzheimer’s disease and other amyloid diseases have been elusive,” said Valerie Daggett, Founder and CEO of AltPep. “We

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Drug Discovery World

JANUARY 31, 2024

By Megan Thomas, DDW Multimedia Editor The last time the DDW team were in Boston for a SLAS event two years ago, the world was very much in the early recovery period of Covid-19. For many, it was their first in-person event – a welcome return.

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Drug Discovery World

SEPTEMBER 27, 2023

Genetic variations inherited from our parents shape our response to drugs, disease susceptibility, physiology and metabolism. This genetic variability forms the basis for personalised approaches to disease prevention, treatment and overall well-being.

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Drug Discovery World

JUNE 3, 2024

Additionally, there were no Grade 3 or higher CRS events and no reported immune effector cell-associated neurotoxicity syndrome (ICANS). Follicular lymphoma is considered incurable and patients at all stages of disease need innovative treatment options.

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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. This treatment only exists because a small charity in the ’70s donated the funds needed for the researcher to discover it. Uplifting Athletes (UA) is on a mission to change that. It saved my life.”

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Drug Discovery World

JUNE 6, 2024

Two other studies presented at the event suggested semaglutide, which is approved for diabetes and weight loss, could reduce the risk of breast cancer recurrence. The drug is also being investigated as a treatment for alcohol use disorder, Alzheimer’s disease and polycystic ovary syndrome.

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Drug Discovery World

MARCH 22, 2024

The US Food and Drug Administration (FDA) has approved Tryvio (aprocitentan) for the treatment of hypertension in combination with other antihypertensive drugs, to lower blood pressure in adult patients who are not adequately controlled on other drugs. Now we are bringing significant medical progress for patients with systemic hypertension.

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Drug Discovery World

NOVEMBER 13, 2023

A number of drugs can slow its progression but currently no treatment exists that can reverse it. We discovered in this latest study that too much of 4-hydroxynonenal switches off a vital event for the cell: processing of microRNAs.

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The Pharma Data

JANUARY 4, 2021

Investors can pre-register for the event here. Eduardo Schiffrin, Medical Director, will participate in a panel discussion at the LifeSci Partners 10 th Annual Healthcare Corporate Access Event on Friday, January 8 from 11am Eastern Standard Time. Investors can pre-register for the event here. LAUSANNE, Switzerland, Jan.

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