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.
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.
Join 15,000+
Insiders
Sign Up for our Newsletter
This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.
Understanding the Regulatory Environment in Japan for Generic Drug Development
Drug Patent Watch
DECEMBER 30, 2024
Types of Drug Applications The PMDA accepts three main types of drug applications: Investigational New Drug (IND) : Required for conducting clinical trials in Japan. Challenges and Opportunities Ethnic Bridging : Japan has historically required clinical trials to be conducted within the country to account for ethnic differences.
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.
Affibody AB to commence trial of its the novel bispecific IL-17A inhibitor ABY-035 in patients with psoriatic arthritis
Drug Discovery Today
NOVEMBER 10, 2020
Solna, Sweden, November 10, 2020. Affibody AB (“Affibody”), a clinical stage biopharmaceutical company, today announced the initiation of its 52-week trial investigating the novel bispecific IL-17A inhibitor ABY-035 in patients with psoriatic arthritis (PsA).
Trials 
100
100 Trials 100
100 
David Liu receives Breakthrough Prize in Life Sciences
Broad Institute
APRIL 5, 2025
The organization has honored Liu for the development of the gene editing platforms base editing and prime editing, which can correct the vast majority of known disease-causing genetic variations and have already been used in at least 15 clinical trials, with life-saving results.
Science 139
139 
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.
Science 52
52 
Wrapping Up 2020: What We’ve Accomplished This Year
Olympian Clinical Research
DECEMBER 30, 2020
2020 has been an incredibly difficult year, and we’ve seen firsthand the physical, emotional, and financial struggles that COVID has caused for our patients, our loved ones, and the healthcare industry as a whole over the past several months. 2020 was a year of obstacles, but it was also a year of incredible growth.
China’s CanSino Enrolls More Than 20,000 in Late-Stage COVID-19 Vaccine Trial | 2020-12-22
The Pharma Data
DECEMBER 22, 2020
The trial is being conducted in Argentina, Pakistan, Russia, Mexico and Chile. The adenovirus-based vaccine is one of five that China has progressed to phase 3 trials so far. CanSino begun enrollment in September with plans to enroll up to 40,000 adults aged 18 and over, according to ClinicalTrials.gov.
Vaccine 52
52 
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.
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.
Therapies 65
65 
The Real Cost of Complexity: What Every Drug Developer Needs to Know
PPD
DECEMBER 16, 2024
Cost and complexity go hand-in-hand The rising costs and growing complexity in clinical trials are deeply linked, with patient recruitment, extended timelines and meeting regulatory demands emerging as some of the key drivers. Nearly half (49%) of clinical trial sponsors surveyed identified rising costs as their foremost concern in 2024.
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.&
Treatment 69
69 
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.
Clinical Trials 134
134 
Inotrem’s COVID-19 Phase IIa Clinical Trial Declared “National Priority Research” by the French Government’s Clinical Trial Council
The Pharma Data
DECEMBER 23, 2020
24, 2020 07:00 UTC. In the context of a substantial amount of COVID-19-related research and the launch of numerous clinical trials, the French government created this steering committee to prioritize and accelerate high potential clinical trials. This Phase IIa trial follows a preliminary study conducted by Prof.
Antibody Drug Conjugates: windows of opportunity
Drug Target Review
JULY 4, 2023
The drivers behind the demand for ADCs Providing more targeted therapeutic options in the oncology space has underpinned the rapid growth of the ADC market; between 2016 to 2020, the US ADC market grew by 45 percent to a value of nearly $1.5 3D rendering of Antibody Drug Conjugate Molecules.
Small Molecule 145
145 
Sanofi and Translate Bio initiate Phase 1 clinical trial of mRNA influenza vaccine
The Pharma Data
JUNE 26, 2021
Sanofi and Translate Bio initiate Phase 1 clinical trial of mRNA influenza vaccine. The trial will evaluate the safety and immunogenicity of a monovalent flu vaccine candidate coding for the hemagglutinin protein of the A/H3N2 strain of the influenza virus. JUNE 22 , 2021.
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?
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. MedComm (2020). Further still, think about implanting these mini organs into the patient to restore lost function. 2009;459(7244):262-5.
Treatment 106
106 
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
Production 52
52 
Pfizer and BioNTech to Provide COVID-19 Vaccine Doses for Olympic Athletes at the 2020 Tokyo Games
The Pharma Data
MAY 6, 2021
This donation of the vaccine is another tool in our toolbox of measures to help make the Olympic and Paralympic Games Tokyo 2020 safe and secure for all participants and to show solidarity with our gracious Japanese hosts,” said IOC President Thomas Bach. “We This press release features multimedia. View the full release here: [link].
Vaccine 52
52 
Sanofi and Translate Bio initiate Phase 1/2 clinical trial of mRNA COVID-19 vaccine candidate
The Pharma Data
MARCH 11, 2021
Clinical trial to assess safety, immune response and reactogenicity, after preclinical data showed high neutralizing antibody levels. The Companies expect interim results from this trial in the third quarter of 2021. About the Phase 1/2 clinical trial. Expected to enroll 415 participants; interim results expected in Q3 2021.
New Rare Disease Dataset Just Dropped
DrugBank
JULY 20, 2023
We are excited to bring FDA Orphan data into DrugBank because we know that at the earliest stages of drug discovery, curating reliable scientific data, identifying relevant previous trials, and designing protocols for success are all important, challenging, and time-consuming tasks. of trials stopped due to recruitment issues.
Disease 98
98 
Calliditas Announces Full Enrollment of the Phase 3 NefIgArd Trial
The Pharma Data
JANUARY 20, 2021
21, 2021 /PRNewswire/ — Calliditas Therapeutics AB (publ) (“Calliditas”) (Nasdaq OMX: CALTX) (NASDAQ: CALT) today announced that all 360 patients have been enrolled for the global Phase 3 clinical trial NefIgArd, which investigates the effect of Nefecon® versus placebo in patients with primary IgA nephropathy (IgAN).
Trials 52
52 
CureVac provides update on Phase 2b/3 trial of first-generation COVID-19 vaccine candidate, CVnCoV
The Pharma Data
JUNE 25, 2021
To identify strains causing COVID-19 infections within the trial, sequencing of virus variants has so far been performed on 474 COVID-19 cases, of which 124 fulfilled adjudication criteria and were included in the present efficacy analysis. CureVac began development of mRNA-based COVID-19 vaccine candidates in January 2020.
Vaccine 52
52 
Ultimovacs ASA Initiates FOCUS Phase II Trial for Universal Cancer Vaccine, UV1, in Head and Neck Cancer Patients Receiving Pembrolizumab
The Pharma Data
DECEMBER 21, 2020
22, 2020 07:13 UTC. The trial will be conducted at 10 sites across Germany and led by principal investigator Prof. The FOCUS trial is Ultimovacs’ fourth Phase II clinical trial with UV1 and comes in addition to the collaboration Phase II trial announced in May 2020. “We Mascha Binder, M.D.,
Vaccine 52
52 
Topline Results from NIH-Sponsored Clinical Trial of Investigational COVID-19 Hyperimmune Globulin Medicine
The Pharma Data
APRIL 3, 2021
The CoVIg-19 Plasma Alliance today announced that the Phase 3 Inpatient Treatment with Anti-Coronavirus Immunoglobulin (ITAC) clinical trial sponsored and funded by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), did not meet its endpoints. About the ITAC Trial.
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.
Lilly and Banner Alzheimer’s Institute collaborate on planned Phase 3 prevention trial of donanemab
The Pharma Data
JULY 15, 2021
Trial will include participants at risk for cognitive and functional decline related to Alzheimer’s disease. TRAILBLAZER-ALZ 3 will evaluate whether treatment with donanemab can slow the clinical progression of Alzheimer’s disease in trial participants. vice president of pain and neurodegeneration, Lilly.
Trials 52
52 
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. Leronlimab is currently undergoing a Phase IIb/III trial to evaluate its efficacy and safety for patients with severe-to-critical COVID-19.
Johnson & Johnson Expands Phase 2a Clinical Trial of COVID-19 Vaccine Candidate to Include Adolescents
The Pharma Data
APRIL 3, 2021
Johnson & Johnson (the Company) has begun vaccinating adolescent participants in the ongoing Phase 2a clinical trial for its COVID-19 vaccine candidate, developed by the Janssen Pharmaceutical Companies of Johnson & Johnson. Vice Chairman of the Executive Committee and Chief Scientific Officer at Johnson & Johnson. “It
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.
Therapies 116
116 
ASLAN Pharmaceuticals Opens Expansion Cohort in ASLAN004 Atopic Dermatitis Clinical Trial
The Pharma Data
JANUARY 10, 2021
001- 38475), including the Company’s Annual Report on Form 20-F filed with the SEC on April 16, 2020. Other factors that may cause actual results to differ from those expressed or implied in such forward-looking statements are described in the Company’s Securities and Exchange Commission (“SEC”) filings and reports (Commission File No.
Imfinzi plus chemotherapy significantly improved overall survival in 1st-line advanced biliary tract cancer in TOPAZ-1 Phase III trial at interim analysis
The Pharma Data
OCTOBER 25, 2021
First immunotherapy combination to demonstrate superior clinical outcomes over standard of care in a global, randomised trial in this setting. 4 In December 2020, Imfinzi was granted Orphan Drug Designation in the US for the treatment of BTC.
Trials 52
52 
The Most Engaging Decision You’ll Read All Year – Five Stars
FDA Law Blog: Biosimilars
JANUARY 15, 2024
FDA complied with that order, later extended the deadline because of COVID, and eventually settled on a PMTA deadline of September 9, 2020. FDA said randomized clinical trials could be used, but so could observational studies with respect to cessation data. FDA also directed manufacturers to produce detailed marketing plans.
FDA 115
115 
Sentiment & Themes Emerging From JPM 2024
LifeSciVC
JANUARY 16, 2024
Whether trial design, execution, or otherwise, drug development even where there is precedent is a challenging road and should not be taken for granted. Additional trials (e.g., The incretins as a class also look interesting across a number of follow-on indications, as evidenced by clinical trials in these areas (below).
Small Molecule 140
140 
What FDA’s Newest Gene Therapy Approval Tells Us About Durability: How Long is Long Enough?
FDA Law Blog: Drug Discovery
DECEMBER 7, 2022
The gene therapy demonstrated that it increased Factor IX (“FIX”) plasma levels at 6 months, the original primary endpoint of the Phase 3 trial. Again, this was despite the fact that subjects in the trial had demonstrated durable FIX activity to this point, with a mean of 41.5%
Therapies 119
119 
Protalix BioTherapeutics and Chiesi Global Rare Diseases Announce Final Results of BRIDGE Phase III Open-Label, Switch-Over Clinical Trial Evaluating Pegunigalsidase Alfa for the Treatment of Fabry Disease
The Pharma Data
DECEMBER 29, 2020
Phase III BRIDGE open-label, switch-over clinical trial met key objectives for safety and efficacy. 30, 2020 /PRNewswire/ — Protalix BioTherapeutics, Inc. . 30, 2020 /PRNewswire/ — Protalix BioTherapeutics, Inc. Topline results from the BRIDGE study were announced in May 2020.
Axsome Therapeutics Announces Positive Efficacy and Safety Results from the Phase 3 MOVEMENT Long-Term Trial of AXS-07 in the Acute Treatment of Migraine
The Pharma Data
DECEMBER 30, 2020
Long-term safety profile consistent with previously completed controlled trials. 31, 2020 (GLOBE NEWSWIRE) — Axsome Therapeutics, Inc. Treatment with AXS-07, rapidly, substantially, and durably relieved migraine pain and associated symptoms in this trial. NDA on track for submission in 1Q 2021.
Trials 52
52 
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. SHENZHEN, China , Jan. Chipscreen Biosciences, Stock Symbol: 688321.SH
FOLLOW-UP DATA FROM PHASE 3 TRIAL OF PFIZER-BIONTECH COVID-19 VACCINE SUPPORT SAFETY AND HIGH EFFICACY IN ADOLESCENTS 12 THROUGH 15 YEARS OF AGE
The Pharma Data
NOVEMBER 22, 2021
The streamlined findings from the companies’ vital Phase 3 trial show that a two- cure series of the Pfizer-BioNTech COVID-19 Vaccine (30-µg per cure) was 100 effective against COVID-19, measured seven days through over four months after the alternate cure. These data were collected from November 2020 to September 2021.
Vaccine 52
52 
Cancer immunotherapy candidate provokes powerful dual response in cancer and immune cells
Broad Institute
OCTOBER 4, 2023
A new small-molecule drug candidate being tested in an early-stage clinical trial aims to improve patient responses to immunotherapy. AbbVie and Calico are currently testing the molecule and another related molecule , also developed by AbbVie and Calico, in phase 1 clinical trials. We were really impressed by that.”
Small Molecule 128
128 
Sanofi and GSK COVID-19 vaccine candidate demonstrates strong immune responses across all adult age groups in Phase 2 trial
The Pharma Data
MAY 16, 2021
The Phase 3 trial is expected to enrol more than 35,000 adult participants from a broad range of countries and will assess the efficacy of two vaccine formulations including the D614 (Wuhan) and B.1.351 1.351 (South African) variants. An earlier stage collaboration with SK Bioscience is also ongoing. Source link.
Article EMA Thank You New guidance provides definition for orphan device, offers alternative trial designs
Agency IQ
JUNE 28, 2024
New guidance provides definition for orphan device, offers alternative trial designs New guidance from the European Commission outlines alternatives for full pre-market clinical trials for orphan devices, defined by the Commission for the first time. MDCG 2020-10 provides guidance on safety reporting during clinical investigations.
Trials 40
40 
Let's personalize your content