PharmaFocus Today – Page 135 – media

LifeArc provides £750,000 fund for clinical trial to treat neuroferritinopathy

The progressive and incurable brain disorder currently affects approximately 100 patients worldwide LifeArc has announced that it has funded a pivotal clinical trial at the University of Cambridge for £750,000 to re-purpose a UK-licensed medicine to treat the ultra-rare genetic disease neuroferritinopathy. The recently launched DefINe trial aims to stop the progression of the disease with an existing drug known as Ferriprox (deferiprone), manufactured by Chiesi Global Rare Diseases. Affecting approximately 100 patients worldwide, neuroferritinopathy is a progressive and incurable brain disorder caused by changes in a gene that produces the ferritin light chain protein. This change can lead to a build-up of iron in the brain, which can cause severe symptoms such as loss of speech and swallowing. With currently no effective treatments available to treat the condition, the new randomised, placebo-controlled trial aims to reduce the iron accumulation in the brain using Ferriprox, an affordable oral tablet licensed ...
- Source: drugdu
- 122
- March 29, 2024
PhaSER Biomedical and the Sanders TDI partner for clinical drug discovery research

PhaSER will provide 8HUM mouse models for the TDI to accelerate a pipeline of novel therapeutic targets PhaSER Biomedical has announced its long-term partnership with the Sanders Tri-Institutional Therapeutic Discovery Institute (TDI) to advance the institute’s pre-clinical drug discovery research. Based in the US, the TDI comprises the Memorial Sloan Kettering Cancer Center, Rockefeller University and Weill Cornell Medicine. The TDI, first formed in 2013, aims to provide academic investigators with access to industrial-scale tools and techniques, to more effectively accelerate drug discovery and bring new cures to patients with the greatest medical need. As part of the ten-year agreement, PhaSER will provide 8HUM mouse models for TDI to use to accelerate and improve drug discovery on a pipeline of novel targets in a variety of therapeutic areas. PhaSER’s 8HUM unique mouse models aim to transform the ways drugs are metabolised in humans and use multiple applications in drug discovery ...
- Source: drugdu
- 94
- March 29, 2024
Merck Drug for Heart and Lung Disorder Wins a First-in-Class FDA Approval

Merck drug Winrevair is the first in a new class of pulmonary arterial hypertension therapies that address a key signaling pathway behind the disease. The drug comes from Merck’s $11.5 billion acquisition of Acceleron Pharma. By FRANK VINLUANPulmonary arterial hypertension, a disease that leads to worsening lung and heart problems that eventually become life-threatening, is treated with drugs that alleviate symptoms. FDA approval of a new Merck drug marks the first for a therapy addressing an underlying cause of the disease. The Tuesday approval of the Merck drug covers the treatment of adults whose pulmonary arterial hypertension (PAH) is at intermediate or high risk of progression. The drug, known in development as sotatercept, will be marketed under the brand name Winrevair. Hypertension—high blood pressure—is common. PAH is a rare form of hypertension that specifically affects the arteries carrying blood from the right side of the heart and into the lungs. ...
- Source: drugdu
- 90
- March 28, 2024
Viking Therapeutics Reveals Positive Results from Oral Obesity Drug

Don Tracy, Associate Editor Reportedly, the oral weight loss pill VK2735 showed promising signs of effectiveness and a tolerable safety profile. Today, Viking Therapeutics announced promising results from a Phase I trial of VK2735, an investigational oral tablet targeting both GLP-1 and GIP receptors for the treatment of metabolic disorders, such as obesity. Results from the 28-day trial show that patients administered VK2735 experienced up to 5.3% weight loss, Additionally, 57% achieved more than 5% in weight loss resulting from treatment with VK2735, a major difference compared to those treated with a placebo. The tablet’s safety profile was reported to be well tolerated, with most adverse effects (AEs) being mild and no serious AEs reported.1 “These Phase I results highlight VK2735’s promising early weight loss and tolerability profile when dosed as an oral tablet,” said Brian Lian, PhD, CEO, Viking, in a press release. “We believe these data indicate that ...
- Source: drugdu
- 130
- March 28, 2024
New Genomic Method Helps Diagnose Patients with Unexplained Kidney Disease

Kidney failure poses a significant threat to life if not adequately treated, yet precise diagnosis often eludes patients, leaving them uncertain about the best treatment approach. Now, a groundbreaking advancement by scientists has led to the discovery of a new technique for examining genomic data that could offer an accurate diagnosis for those suffering from unexplained kidney failure. Researchers from Newcastle University (Newcastle upon Tyne, UK) used data from the Genomics England 100,000 Genomes Project to establish a diagnosis for patients facing unexplained kidney failure. They discovered that certain segments of these patients’ genomes were absent, as a result of which their conditions went undiagnosed with standard genetic analysis techniques. The researchers have now identified this missing gene and found the mutations within it, allowing them to classify this as NPHP1-related -related kidney failure. For their study, the team reviewed the genetic sequencing data of 959 individuals with advanced kidney ...
- Source: drugdu
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- March 28, 2024
Fluid Biomarker Test Detects Neurodegenerative Diseases Before Symptoms Appear

Amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s disease) and frontotemporal dementia (FTD) are two progressively neurodegenerative conditions that damage essential nerve cells. ALS compromises nerve cells in the brain and spinal cord, leading to movement impairment, while FTD damages brain areas responsible for personality, behavior, and language. Studies indicate that in ALS or FTD patients, TAR DNA-binding protein 43 (TDP-43) malfunctions. This disruption prevents the correct splicing of ribonucleic acid (RNA), essential for generating proteins necessary for nerve growth and function. Instead, RNA strands become riddled with erroneous code sequences, known as “cryptic exons,” which are linked to an elevated risk of developing ALS and FTD. The timing of this anomaly in the disease progression of ALS and FTD was previously unknown. Now, researchers from Johns Hopkins Medicine (Baltimore, MD, USA) have developed a method for identifying a specific protein, hepatoma-derived growth factor-like 2 [HDGFL2], linked to TDP-43 ...
- Source: drugdu
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- March 28, 2024
$770 Million! AriBio authorizes Marketing Rights for a Drug to Treat Alzheimer’s Disease in China

It’s reported according to businesswire, AriBio recently announced that it has signed a licensing agreement for the exclusive marketing rights of AR1001, an investigational drug for the treatment of early Alzheimer’s disease, in China at a price of up to US$770 million. Considering the market competition and sales strategy of Alzheimer’s disease drugs in China, the licensee requested that no disclosure be made until the agreed time. The agreement includes an upfront non-refundable payment of 120 billion won (approximately US$90 million), with a total transaction value of up to 5.59 billion yuan (approximately US$770 million), including milestone payments and additional royalties. Upfront payments will begin in mid-2024. AR1001 is a phosphodiesterase type 5 (PDE5) inhibitor in development and an investigational oral drug for the treatment of Alzheimer’s disease. Pre-clinical studies have confirmed that AR1001 can inhibit neuronal apoptosis and restore synaptic plasticity, thus having neuroprotective effects. AR1001 also demonstrated a ...
- Source: drugdu
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- March 28, 2024
EU recommendations for 2024/2025 seasonal flu vaccine composition

EMA has issued recommendations for the influenza virus strains that vaccine manufacturers should include in vaccines for the prevention of seasonal influenza from autumn 2024. Every year, EMA issues EU recommendations for the composition of seasonal influenza vaccines on the basis of observations by the World Health Organization (WHO) which are informed by regular monitoring activities on the prevalence and characteristics of different influenza viruses worldwide. Based on this data EMA’s Emergency Task Force (ETF) has issued a statement recommending a transition from quadrivalent to trivalent vaccines that do not include the B/Yamagata component. Currently, most authorised influenza vaccines are quadrivalent, which means that they are formulated to protect against the four main strains of influenza responsible for seasonal flu, A(H1N1)pdm09 and A(H3N2), B/Victoria and B/Yamagata. However, the B/Yamagata strain of the influenza B virus has not been detected in circulation since March 2020. This is thought to be due ...
- Source: drugdu
- 123
- March 28, 2024
Another Chiatai Tianqing’s contrast agent, iopromide injection, was approved for marketing

Recently, Chiatai Tianqing received the Certificate of Drug Registration approved and issued by the State Drug Administration (NMPA), in which the company’s product Iopromide Injection in the field of medical imaging was approved to be listed and deemed to have passed the consistency evaluation as the second domestic one. Iopromide injection is a diagnostic drug, which can be used for intravascular and intracorporeal imaging, and is currently widely used in clinical applications and has great potential. Iopromide is a tri-iodinated non-ionic water-soluble X-ray contrast agent with high iodine content and good contrast effect, which is used for computed tomography (CT) enhancement, arteriography and venography, arterial/venous digital subtraction angiography (DSA), cardiovascular angiography, venous urography, endoscopic retrograde cholangiopancreatography (ERCP), arthrocentesis, and other body cavity examinations. Iopromide has the advantages of stable nature, easy to use, lower toxic side effects than ionic iodine contrast agent, good systemic tolerance, etc., and has become a ...
- Source: drugdu
- 135
- March 28, 2024
BIO-THERA’s Phase II Clinical Trial Application for BAT8006 (ADC- FRα) for Injection Approved by US FDA

BIO-THERA Biopharmaceutical Co., Ltd (SSE: 688177), a global science-based and innovative biopharmaceutical company based in Guangzhou, China, hereinafter referred to as “BIO-THERA” or the “Company”, today announced that its Phase II clinical trial application (IND) for BAT8006 (ADC- FRα) for injection has been approved by the U.S. Federal Drug Administration (FDA). The Company today announced that it has recently received approval from the U.S. Food and Drug Administration (FDA) for a Phase II clinical trial (IND) of its investigational drug, BAT8006 for injection, to evaluate its efficacy and safety in patients with platinum-resistant epithelial ovarian, fallopian tube or primary peritoneal cancer. Previously, BAT8006 has initiated a Phase I dose-escalation and dose-expansion study in China with phase I data. BAT8006 is an antibody drug coupling (ADC) developed by BIO-THERA targeting folate receptor alpha (FRα), which is intended to be developed for the treatment of solid tumors.FRα is a folate-binding protein located ...
- Source: drugdu
- 126
- March 28, 2024

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