Biogen, Sage tremor drug fails key trial
BioPharma Drive: Drug Pricing
JULY 24, 2024
Known as SAGE-324, the drug was one of the key assets Biogen gained rights to through a billion-dollar research deal inked back in 2020.
2020 Drugs Trials 
Understanding the Regulatory Environment in Japan for Generic Drug Development
Drug Patent Watch
DECEMBER 30, 2024
The regulatory environment in Japan for generic drug development is complex and has undergone significant changes in recent years. Regulatory Authority: Pharmaceuticals and Medical Devices Agency (PMDA) The PMDA is the primary regulatory authority responsible for overseeing the drug approval process in Japan.
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The rising impact of biomarkers in early clinical development
Drug Target Review
APRIL 7, 2025
A surrogate endpoint is a marker used in clinical trials as a substitute for a direct clinical outcome. Diagnostic biomarkers typically confirm or establish a diagnosis and are often used in selecting patient populations for clinical trials.
New tRNA tech aims to rewrite rare disease treatment
Drug Target Review
JUNE 9, 2025
A personal journey to Alltrna Michelle Werner’s career has spanned over 20 years in the pharmaceutical industry, where she developed her expertise in oncology drug development at leading companies such as Bristol Myers Squibb, AstraZeneca, and Novartis.
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Time for change: non-human primates in drug research
Drug Target Review
JULY 7, 2025
Non-human primates (NHPs), such as macaques and cynomolgus monkeys, have long served as a cornerstone in preclinical drug development due to their close genetic, anatomical and physiological resemblance to humans. Conventional models often fail to predict cardiac side effects, especially with biologics.
Emerging Trends in Clinical Trial Design
PPD
AUGUST 16, 2023
The COVID-19 pandemic rapidly accelerated the adoption of hybrid and decentralized clinical trial (DCT) models. However, as the world settles into its post-pandemic state and returns to pre-pandemic paradigms in many areas, the pharmaceutical industry remains dedicated to moving beyond traditional, centralized clinical trial constructs.
Antibody Drug Conjugates: windows of opportunity
Drug Target Review
JULY 4, 2023
Over the last two decades, an increasing number of Antibody Drug Conjugate (ADC) therapeutics have been approved for oncology indications. These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. 3D rendering of Antibody Drug Conjugate Molecules.
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The Real Cost of Complexity: What Every Drug Developer Needs to Know
PPD
DECEMBER 16, 2024
Our annual look at the state of the drug development industry highlights a dual set of challenges complicating progress. Pressure and requirements to engage diverse patient populations in trials have become more challenging and expensive, requiring tailored strategies that can stretch both resources and budgets.
How to Get Drugs Into the Brain
Drug Hunter
AUGUST 21, 2023
Obtaining adequate drug exposure in the brain is key to treating CNS diseases effectively. Recently, Dennis Koester gave us a crash course in CNS drug discovery in a Drug Hunter Flash Talk. Why Kp,uu is the Most Important Parameter in CNS Drug Discovery What Influences the Kp,uu of Drugs?
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Organoids: the versatile platform for discovery and regeneration
Drug Target Review
OCTOBER 30, 2024
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. 12 Testing drugs in vitro in organoid cultures A clear validation of the utility of organoids is in drug discovery and development.
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Educating Patients about Generic Drugs: Strategies for Success
Drug Patent Watch
JANUARY 7, 2025
Generic drugs play a crucial role in providing affordable medication options to patients. As healthcare professionals, it’s our responsibility to educate patients about generic drugs and empower them to make informed decisions about their treatment options. What Are Generic Drugs?
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Advancing reduction of drug use as an endpoint in addiction treatment trials
National Institute on Drug Abuse: Nora's Blog
MARCH 6, 2025
Advancing reduction of drug use as an endpoint in addiction treatment trials astewart Thu, 03/06/2025 - 09:59 Nora's Blog March 18, 2025 Image Getty Images/ SolStock This blog was also published in the American Society of Addiction Medicine (ASAM) Weekly on March 18, 2025.&
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Advancements in hit identification for membrane protein drug discovery
Drug Target Review
APRIL 7, 2025
The challenge of GPCR drug discovery G protein-coupled receptors (GPCRs) are one of the most desirable and challenging target classes in drug discovery, as their mutation can lead to a wide range of diseases such as cancer, cardiovascular disorders and neurological conditions.
CDER regulatory science division touts 2020 accomplishments
The Pharma Data
MAY 3, 2021
In its 2020 annual report, the division of the US Food and Drug Administration (FDA) tasked with addressing emerging regulatory issues and developing new tools and approaches for regulatory reviews said it was able to expand its efforts despite the COVID-19 pandemic impacting most of the year. . 2020 was a challenging year.
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PRITELIVIR MESYLATE
New Drug Approvals
JULY 8, 2025
1428321-10-1 Pritelivir mesylate is an antiviral drug currently under development, specifically targeting herpes simplex virus types 1 and 2 (HSV-1 and HSV-2). Clinical Trials: Pritelivir is currently in phase II clinical trials, with ongoing research into its effectiveness and safety.
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Possible adulticidal drugs for Onchocerca volvulus
Drug Target Review
SEPTEMBER 21, 2023
The findings supported the possibility put forward by Cotton et al (2016) concerning the chance of repurposing these drugs for adulticidal treatment. Need for adulticidal drugs More than 99 percent of countries worldwide has been affected by this disease. They provided a big stepping stone, needed for the development of drugs.
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New cell therapy model accelerates cancer treatment development
Drug Target Review
FEBRUARY 10, 2025
In just two years, CTMC has advanced eight therapies into clinical trials, harnessing genetic engineering to enhance T-cell effectiveness in the fight against cancer. Around 2020, Bock assumed the challenge of creating a new division at MD Anderson focused on advancing cell therapies by bridging the gap between research labs and the clinic.
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How 3D human tissue models are transforming drug discovery
Drug Target Review
FEBRUARY 2, 2024
The standard animal model paradigm for drug discovery does not translate well to effects in patients for a number of reasons. In summary, all these factors can affect the response to drugs and drug target manipulation.
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Clinical Trials Join the Remote Work Revolution: FDA’s New Draft Guidance on Decentralized Clinical Trials
FDA Law Blog: Drug Discovery
MAY 4, 2023
Cato — On May 2nd, FDA released a new draft guidance with recommendations for decentralized clinical trials (DCTs) for drugs, biologics, and devices. In a DCT, trial-related activities may occur in trial participants’ homes, at local health care providers’ offices, or in local clinical laboratories.
Unlocking the potential of natural products in drug discovery
Drug Target Review
MARCH 28, 2024
Between 2000 and 2020, approximately 30 percent of the newly introduced small molecule drugs were derived from natural products. In recent decades there has been a decline in interest in natural products for drug discovery, with the industry gravitating towards screening libraries of synthetic molecules with predefined chemistries.
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UK Pharma Company Scancell Selects COVID-19 Vaccine for Clinical Trials | 2020-12-21
The Pharma Data
DECEMBER 20, 2020
UK drug developer Scancell said it has chosen a COVID-19 vaccine candidate, SN14, from more than a dozen potential products to advance to a clinical trial. . In October, Scancell signed a manufacturing deal for SN14 with Cobra Biologics, a Cognate BioServices subsidiary, ahead of a planned phase 1 trial in 2021.
Colchicine Added to UK RECOVERY Trial Evaluating COVID-19 Treatments | 2020-12-01
The Pharma Data
NOVEMBER 30, 2020
The UK-based RECOVERY trial, which is evaluating multiple treatments for COVID-19, will add the inexpensive anti-inflammatory drug colchicine to the list of therapies being tested in hospitalized patients.
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Altasciences At CPHI Americas 2025
Alta Sciences
JULY 3, 2025
Altasciences At CPHI Americas 2025 pmjackson Thu, 07/03/2025 - 13:41 In the ever-changing drug development world, staying agile is essential to enabling more informed decisions, faster. It’s a challenge that affects a large percentage of new drug candidates—one we’re passionate about helping to solve.
Zilucoplan
New Drug Approvals
JUNE 15, 2025
Retrieved 15 September 2024. ^ “Notice: Multiple additions to the Prescription Drug List (PDL) [2024-08-13]” Health Canada. Retrieved 15 August 2024. ^ “Regulatory Decision Summary for Zilbrysq” Drug and Health Products Portal. Food and Drug Administration (FDA). Food and Drug Administration (FDA).
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Anti-Inflammatory Drug Colchicine Investigated for COVID-19 in UK RECOVERY Trial | 2020-11-30
The Pharma Data
NOVEMBER 30, 2020
Drug Industry Daily (DID) the premier online resource for savvy pharmaceutical professionals whose jobs depend on accurate knowledge about the daily activities of Congress, the FDA, other key regulators … and what their competitors are up to. Become a subscriber to Drug Industry Daily today. No questions asked.
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New Rare Disease Dataset Just Dropped
DrugBank
JULY 20, 2023
Rare diseases, therefore, present compelling opportunities for Drug Development. In the United States, the Orphan Drug Act has provided a framework for this support since 1983 by issuing orphan designations to drugs being investigated for treating a rare disease. At any given time, millions of people ( 3.5–5.9%
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New molecular insights on medical cannabis
Drug Target Review
NOVEMBER 1, 2023
2 Implication of ATX in a large range of human diseases have been highlighted by both fundamental research and clinical trials. 1-5 Implication of ATX in a large range of human diseases have been highlighted by both fundamental research and clinical trials. He is a researcher at University of Oldenburg, Germany. Frontiers in Medicine.
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Elacestrant
New Drug Approvals
MAY 9, 2025
1] History The efficacy of elacestrant was evaluated in the EMERALD trial, which was a randomized, open-label, active-controlled, multicenter study involving 478 postmenopausal women and men with ER-positive, HER2-negative advanced or metastatic breast cancer. Food and Drug Administration (FDA). 1] [4] It is taken by mouth. [1]
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Reaction of Investigational Drugs to Acid-Reducing Agents Should Be Studied, Guidance Says | 2020-11-30
The Pharma Data
NOVEMBER 30, 2020
Sponsors of drug trials should determine what effect the use of acid-reducing agents (ARA), such as antacids, proton pump inhibitors and histamine blockers, could have on the solubility of an orally administered drug, according to a new FDA draft guidance released Monday.
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AstraZeneca COVID-19 Vaccine Shows Good Results in Late Trials
The Pharma Data
DECEMBER 8, 2020
8, 2020 — AstraZeneca’s COVID-19 vaccine is safe and effective, new data from late-stage trials shows. The interim findings are from phase 3 trials in the United Kingdom and Brazil that included more than 11,600 participants. Food and Drug Administration’s website in advance of a Dec. © 2020 HealthDay.
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Optimizing Your Drug Patent Strategy: A Comprehensive Guide for Pharmaceutical Companies
Drug Patent Watch
JANUARY 8, 2025
This comprehensive guide will walk you through the intricacies of optimizing your drug patent strategy, ensuring that your innovations are safeguarded and your market position is strengthened. Understanding the Basics of Drug Patents Before diving into the nuances of patent strategy, it’s crucial to grasp the fundamentals.
Resolutions at the Extraordinary General Meeting in BioInvent on November 27, 2020
The Pharma Data
NOVEMBER 26, 2020
27, 2020 /PRNewswire/ — BioInvent International AB ‘s (publ) (“BioInvent”) (OMXS: BINV) Extraordinary General Meeting (the “EGM”) today resolved to approve the Board of Directors’ resolution on a directed issue of 29,395,311 new shares and 14,697,655 new warrants to CASI Pharmaceuticals Inc.
Human neuronal cells: possibilities in drug safety testing
Drug Target Review
SEPTEMBER 26, 2023
These cells demonstrate considerable promise for uncovering drug-induced perturbations to neuronal function such as seizure, and their use extends further to sedation, anti-epileptic drug discovery and modelling of neurological diseases.
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How Many Clinical Trials Are Run by CROs?
Vial
FEBRUARY 29, 2024
Contract research organizations (CROs) are an integral partner of the drug development process, as they play a pivotal role supporting clinical trial conduct for pharmaceutical, biotechnology, and medical device sponsor companies. That is, how many clinical trials are actually managed by these organizations?
Syros Presents New Data From Phase 2 Clinical Trial of SY-1425 and Announces Plans to Initiate Registration-Enabling Trial in MDS and Randomized Phase 2 Trial in AML
The Pharma Data
DECEMBER 5, 2020
–(BUSINESS WIRE) December 5, 2020 — Syros Pharmaceuticals (NASDAQ:SYRS), a leader in the development of medicines that control the expression of genes, today announced new clinical data from its Phase 2 trial evaluating SY-1425, its first-in-class selective retinoic acid receptor alpha (RAR?) CAMBRIDGE, Mass.–(BUSINESS
Stem cell therapy to slow progression of multiple sclerosis (MS)
Drug Target Review
OCTOBER 5, 2023
This study sought to shed light on the safety and effectiveness of aHSCT in routine healthcare settings, moving beyond the confines of clinical trials. The research team identified 231 individuals diagnosed with relapsing-remitting MS, with 174 of them having undergone aHSCT treatment before the year 2020.
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Pivotal phase III clinical trial IND submission of the original innovative anticancer drug Chiauranib for the treatment of small cell lung cancer was accepted by the Center of Drug Evaluation of the NMPA.
The Pharma Data
JANUARY 10, 2021
SH ) submitted its pivotal phase III clinical trial application (IND) of national class I innovative drug Chiauranib to NMPA, for the treatment of Small Cell Lung Cancer (SCLC) as a single agent for the patients after 2nd-line systemic chemotherapy and recurrence afterwards. There is an urgent need for innovative drugs to treat SCLC.
Cancer immunotherapy candidate provokes powerful dual response in cancer and immune cells
Broad Institute
OCTOBER 4, 2023
Cancer immunotherapy drugs called PD-1 inhibitors are widely used to stimulate the immune system to fight cancer, but many patients either don’t respond or develop resistance to them. A new small-molecule drug candidate being tested in an early-stage clinical trial aims to improve patient responses to immunotherapy.
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Spectrum Pharmaceuticals Announces Two Oral Presentations at Upcoming IASLC 2020 World Conference on Lung Cancer
The Pharma Data
JANUARY 21, 2021
.–( BUSINESS WIRE )– Spectrum Pharmaceuticals (NasdaqGS: SPPI), a biopharmaceutical company focused on novel and targeted oncology therapies, today announced an oral presentation on updated efficacy, safety, and dosing management of poziotinib from Cohorts 1 and 2 of the ZENITH20 clinical trial.
Vertex’s and CRISPR Therapeutics’ Gene Therapy Shows Positive Efficacy | 2020-12-08
The Pharma Data
DECEMBER 8, 2020
Blood disease trial participants given Vertex’s and CRISPR Therapeutics’ CRISPR/Cas9-based gene-editing therapy CTX001 demonstrated a “consistent and sustained response” to the treatment, according to new phase 1/2 trial data the companies have announced. To date, a total of 19 trial participants have been dosed with the gene therapy.
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AbbVie Reports Full-Year and Fourth-Quarter 2020 Financial Results
The Pharma Data
FEBRUARY 4, 2021
3, 2021 /PRNewswire/ — AbbVie (NYSE: ABBV) announced financial results for the fourth quarter and full year ended December 31, 2020. In both Phase 3 trials, significantly more patients treated with Skyrizi achieved the primary endpoint of ACR20 response at week 24 versus placebo. NORTH CHICAGO, Ill., Recorded a $4.7
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Building Korro Bio: A CEO’s Perspective on Innovation and Risk Management
LifeSciVC
JUNE 12, 2025
Building medicines by activating biological pathways As I joined the Company in Q4 2020 as its CEO, I had a vision of creating protein variants to activate biological pathways. This meant focusing on clinical benefit for patients with AATD and build a drug candidate profile that was meaningful for patients.
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Microsoft Warns of Cyberattacks Against COVID-19 Vaccine, Drug Developers | 2020-12-11
The Pharma Data
DECEMBER 11, 2020
Among the targets, the majority are vaccine makers that have COVID-19 vaccines in various stages of clinical trials. One is a clinical research organization involved in trials, and one has developed a COVID-19 test,” Microsoft said.
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Therapeutics for ‘COVID-19 Long-Haulers’ Exploding onto the Scene as 2020 Ends
The Pharma Data
DECEMBER 23, 2020
On December 3, the PureTech initiated a global, Phase II trial of LYT-100 (deupirfenidone) in Long COVID respiratory complications and related sequelae. The trial is ongoing in the U.S. PureTech Health Plc., and Europe, and PureTech Head of Innovation, Dr. Michael C. Organicell Regenerative Medicine, Inc. , said in a statement.
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