BioPharma Drive: Drug Pricing

MARCH 12, 2024

Physicians are preparing for tough conversations with patients on Amylyx Pharmaceuticals’ drug, Relyvrio, while holding out hope it still may help some with the disease.

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Drug Target Review

DECEMBER 20, 2024

However, recent breakthroughs in AI, such as predictive modelling, clinical trial optimisation, and personalised medicine, have demonstrated its potential. We spoke with Aaron Smith, a mathematician-turned-machine learning scientist and the founder of Unlearn , a company leading the charge in applying AI to optimise clinical trial efficiency.

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Science Daily: Pharmacology News

APRIL 8, 2025

Food and Drug Administration (FDA) for clinical trials for the treatment of amyotrophic lateral sclerosis (ALS) -- improves neuron health in animal models of Alzheimer's disease, according to a new study. Experimental drug NU-9 -- a small molecule compound approved by the U.S.

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Science Daily: Pharmacology News

DECEMBER 10, 2023

Leukaemia trial shows 'exceptional' results. Personalized treatment for the most common form of adult leukemia helps patients survive for longer and stay in remission, a phase III trial has found.

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FDA Law Blog: Drug Discovery

JANUARY 30, 2025

However, as we note in that post, the design, timing of initiation, and timely conduct of confirmatory trials are also important considerations in FDAs determination of whether accelerated approval is appropriate. This blog post focuses on interpreting these new authorities with respect to timely conduct of confirmatory trials.

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FDA Law Blog: Drug Discovery

NOVEMBER 10, 2024

Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.

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BioPharma Drive: Drug Pricing

OCTOBER 23, 2023

The trial, which is ongoing in the U.K. since late last year as the FDA sought more details on Verve’s in vivo treatment for heart disease. and New Zealand, has been on hold in the U.S.

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Conversations in Drug Development Trends

OCTOBER 29, 2024

By: Juliane Mills, Senior Director, Therapeutic Strategy Lead, Rare Disease The rise of patient-led clinical research, particularly in rare disease, represents a significant shift in the clinical trial landscape. Why Is There an Increase in Patient-Led Rare Disease Research?

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BioPharma Drive: Drug Pricing

OCTOBER 7, 2024

The study, of Intellia’s treatment for hereditary angioedema, is the second Phase 3 trial the biotech has begun testing its “in vivo” gene editing medicines.

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Drug Target Review

OCTOBER 11, 2023

Almost every one of us will know someone with a neurodegenerative disease such as Alzheimer’s or Parkinson’s. It is less common to know someone with a rare genetic disease; but while individually rare, collectively they have an enormous impact. Most of these conditions are genetic in origin and the majority have no effective treatment.

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BioPharma Drive: Drug Pricing

JUNE 3, 2024

Liquid biopsy has emerged as a revolutionary tool for clinical trials, offering cellular and cell-free methods that provide valuable insights into disease progression and treatment response.

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Science Daily: Pharmacology News

MARCH 19, 2025

An experimental drug appears to reduce the risk of Alzheimer's-related dementia in people destined to develop the disease in their 30s, 40s or 50s, according to the results of a new study.

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Drug Patent Watch

JUNE 13, 2025

According to our analysis of clinical trial listings, the following industries are leading the charge in seeking legal counsel: Biotech: With a 25% increase in clinical trial listings over the past year, biotech companies are racing to secure patents and protect their intellectual property.

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Drugs.com

JUNE 18, 2024

TUESDAY, June 18, 2024 -- There's more good news in the battle against sickle cell disease, with another trial finding CRISPR gene-editing therapy delivering impressive results for patients.“It’s It’s encouraging that this gene-editing treatment con.

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PPD

JUNE 20, 2025

Patients diagnosed with idiopathic pulmonary fibrosis (IPF) — a progressive, rare lung disease marked by scarring and worsening lung function — often face a poor prognosis. While two approved treatments, pirfenidone and nintedanib, slow disease progression, there currently is no treatment that reverses the effects or offers a cure for IPF.

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BioPharma Drive: Drug Pricing

AUGUST 28, 2023

The treatment, developed by a biotech Bayer acquired in 2019, appeared safe in a small trial and showed signs of an effect on disease progression.

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The Pharma Data

JUNE 25, 2025

Biogen Reports Promising Interim Phase 1 Results for Salanersen in Spinal Muscular Atrophy, Prepares for Registrational Trials Biogen has announced encouraging topline results from its Phase 1 clinical trial evaluating salanersen (BIIB115/ION306) , an investigational antisense oligonucleotide (ASO) therapy for spinal muscular atrophy (SMA).

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Drug Target Review

APRIL 7, 2025

As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. A surrogate endpoint is a marker used in clinical trials as a substitute for a direct clinical outcome.

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ProRelix Research

SEPTEMBER 7, 2023

Autoimmune disorders comprise several diseases in which the immune system mistakenly attacks and destroys the body’s own healthy cells instead of destroying bacteria and viruses to keep the body healthy. […] The post Autoimmune disorders and current developments of novel treatments under clinical trials appeared first on ProRelix Research.

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BioPharma Drive: Drug Pricing

OCTOBER 9, 2024

The Series B round will fund a Phase 1/2 trial of the company’s experimental treatment for IgA nephropathy.

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The Pharma Data

JUNE 23, 2025

These findings represent a pivotal advancement in the treatment of this rare, progressive, and life-threatening disease. Participants were classified as WHO Group 1 PAH, functional class (FC) II or III, indicating moderate to severe disease at either intermediate or high risk of clinical deterioration.

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Conversations in Drug Development Trends

JANUARY 6, 2025

With 20-25% of the global population affected by metabolic problems, finding effective treatments is crucial for cardiovascular and metabolic (CVM) conditions. Traditional study designs feature predetermined sample sizes, assessments, and analyses that must remain in place over long treatment durations.

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The Pharma Data

JUNE 18, 2025

The IDeate-Prostate01 trial marks a significant advancement in the B7-H3 ADC development program and underscores the shared commitment of both companies to addressing difficult-to-treat cancers. This has created an urgent need for more effective and tolerable treatments for this population. There is a critical need for new treatments.

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Science Daily: Pharmacology News

JULY 15, 2024

Importantly the research team isolated a molecule produced by gut bacteria that in the future could potentially be trialed as a simple treatment, in the form of a dietary supplement, for children at risk of asthma to prevent them developing the disease.

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ProRelix Research

SEPTEMBER 17, 2023

The rising prevalence of ophthalmology disorders such as dry eye disease (DED) and vision-related issues is largely related to the increasing use of mobile phones, tablet screens, and laptops.

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Science Daily: Pharmacology News

FEBRUARY 22, 2024

A large-scale clinical trial of treatment strategies for Crohn's disease has shown that offering early advanced therapy to all patients straight after diagnosis can drastically improve outcomes, including by reducing the number of people requiring urgent abdominal surgery for treatment of their disease by ten-fold.

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Drug Target Review

OCTOBER 30, 2024

Imagine being able to create an in vitro replica of a diseased organ to study the molecular mechanism underlying the illness. Now take a step further: envision testing drugs in these organoids to identify the ones that can treat disease safely and effectively without needing to run expensive clinical trials first.

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The Pharma Data

JULY 2, 2025

Biogen Launches Global Phase 3 PROMINENT Trial Evaluating Felzartamab in Primary Membranous Nephropathy Biogen Inc. The trial is expected to complete in 2029. By selectively depleting these cells, felzartamab offers a promising, mechanism-driven approach to potentially halting disease progression.

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The Pharma Data

JUNE 12, 2025

The results come from a open-label, proof-of-concept, Phase 2 study (NCT04520451) and highlight rilzabrutinib’s potential as a disease-changing treatment option for a condition that currently has limited and non-specific treatment options and involves substantial patient suffering due to its chronic and progressive course.

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Quanticate

DECEMBER 16, 2024

A randomised controlled clinical trial is the most prevalent design used in Phase II and Phase III clinical trials, whereby participants are randomly assigned to a control or experimental group with a fixed probability, normally 50% for each treatment.

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Chemical Biology and Drug Design

DECEMBER 11, 2024

The study also explores and compiles the latest patents and clinical trial data into a single platform to help develop selective Mtb -DHFR inhibitors for effective tuberculosis treatment. ABSTRACT Tuberculosis (TB) is an obstinate and infectious disease requiring a relatively longer treatment duration than other bacterial infections.

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SCIENMAG: Medicine & Health

SEPTEMBER 7, 2023

7, 2023 – Alzheimer’s disease is the most common cause of dementia that affects more than 6.5 To find effective treatments and slow the progression of this debilitating disease, researchers have made much progress in developing new drugs that target beta-amyloid plaques, one of […] WINSTON-SALEM, N.C.

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Drug Target Review

MARCH 3, 2025

The search for effective treatments for neurodegenerative diseases like Parkinson’s disease has long been hindered by the brain’s complexity and the absence of adequate models for drug discovery. “For diseases like Parkinson’s, it’s more than sufficient,” he explains.

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The Pharma Data

JUNE 11, 2025

Dapirolizumab Pegol Demonstrates Promise in Phase 3 SLE Trial, Showing Improvements in Fatigue and Disease Activity at EULAR 2025 UCB (Euronext Brussels: UCB) and Biogen Inc. The latest data from the PHOENYCS GO trial reinforce the potential of dapirolizumab pegol to improve disease outcomes in people with moderate-to-severe SLE.

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BioPharma Drive: Drug Pricing

SEPTEMBER 22, 2023

The drug, which is already cleared as a neoadjuvant lung cancer treatment, could further compete with Merck’s Keytruda if it gains approval in the adjuvant setting for patients with the non-small cell form of the disease.

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Drug Hunter

AUGUST 15, 2023

LRRK2 has been a hotly pursued drug target for Parkinson’s Disease based on human genetics. On Sunday, it was disclosed that additional, likely on-target adverse findings were observed in a primate GLP study on long-term treatment with Merck’s second-generation LRRK2 inhibitors. Get ahead now by requesting a trial.

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The Pharma Data

JUNE 11, 2025

Ionis Begins Pivotal Phase 3 REVEAL Study of ION582 in Angelman Syndrome, Dosing First Patient in Global Trial Ionis Pharmaceuticals , Inc. Currently, treatment options are limited to supportive care aimed at managing symptoms rather than targeting the underlying cause.

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