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2019 Therapies Trials 
Drug Target Review
NOVEMBER 17, 2023
They do not induce ‘graft versus host’ disease when transplanted for allogeneic therapy and there appears little sign of immune rejection. Peripheral blood NK cells have been used in allogeneic cancer therapies and shown to be safe. CAR-NK cell trials only commenced in 2017 and most are still recruiting patients.
118 
PLOS: DNA Science
OCTOBER 19, 2023
Last week DNA Science covered a setback in a clinical trial of a gene therapy for Duchenne muscular dystrophy (DMD). Also recently, FDA’s Cellular, Tissue, and Gene Therapies Advisory Committe turned down a stem cell treatment for amyotrophic lateral sclerosis, aka ALS, Lou Gehrig’s disease, or motor neuron disease.
111 
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.
98 
Drug Target Review
FEBRUARY 28, 2024
Over the past 25 years, T-cell therapies have gained significant ground in the treatment of cancer. Preclinical research on γδ T cells has made great strides since the cells were first identified in the 1980s, with γδ T-cell therapies from several companies, including IN8bio, now in or nearing clinical trials for various cancers.
105 
Antidote
JULY 29, 2022
billion to bring a new therapy to market. In 2019 alone, research and development spending totaled $186 billion for the global pharmaceutical industry. Between 2009 and 2018, U.S.
Advarra
AUGUST 8, 2023
Gene therapy research is booming in the clinical setting. In this blog, we summarize the growth, risks, and regulatory requirements for gene therapy research. Defining the Boom in Gene Therapy Research The gene therapy field is experiencing explosive growth in today’s competitive research environment.
52 
PLOS: DNA Science
OCTOBER 12, 2023
In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It , I predicted that the technology would soon expand well beyond the rare disease world. Gene therapy clearly hasn’t had a major impact on health care, offering extremely expensive treatments for a few individuals with rare diseases.
69 
The Pharma Data
OCTOBER 27, 2020
Dry AMD is one of the leading causes of vision loss globally and gene therapies hold tremendous promise for this disease. Delivering gene therapy directly to the site of disease through a subretinal injection is a proven approach; however, the current method requires a vitrectomy. at Ophthalmic Consultants of Boston. About FOCUS.
40 
The Pharma Data
MARCH 25, 2021
this week announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) approved Breyanzi (lisocabtagene maraleucel: liso-cel), a CD19-directed chimeric antigen receptor (CAR) T cell therapy for the treatment of patients with relapsed or refractory (R/R) large B-cell lymphoma 1 and R/R follicular lymphoma. Bristol Myers Squibb K.K.
52 
The Pharma Data
MAY 31, 2022
the completion of enrollment of its Phase 1 (Ph1), open-label trial of pluripotent stem cell-derived dopaminergic neurons in patients with Parkinson’s disease (PD). Ph1 clinical trial is to evaluate the safety, tolerability, and?preliminary?efficacy?of AG, announced?the The purpose of the?Ph1 preliminary?efficacy?of F.C.A.H.S. (PI)
52 
The Pharma Data
AUGUST 28, 2020
The Phase 1 trial will be conducted by MindMed later this year and will be the first to investigate what happens when you mix the two drugs to work as a therapy. Researchers are set to investigate a combined dose of LSD and MDMA to treat patients undergoing psychotherapy.
The Pharma Data
AUGUST 7, 2020
As the pharma industry stands firm in its commitment to advance the sector to fight Covid-19, news has emerged from the European Commission who intend to streamline the development of therapies using genetically modified organisms to treat Covid-19. Regenerative medicine and advanced therapies thriving despite Covid-19 disruption.
The Pharma Data
APRIL 19, 2022
Kite’s Global CAR T-Cell Therapy Manufacturing Network Increasing Capacity by 50% to Meet Patient Demand for New Cancer Therapies. — Scalable and Adaptable Facility Provides Flexibility for Current and Future Cell Therapy Innovation. The site will produce Kite’s FDA approved CAR T-cell therapy used to treat blood cancer.
52 
Broad Institute
NOVEMBER 6, 2024
The results also highlight possible therapeutic targets that bolster the rationale behind an ongoing clinical trial recently launched at MGH that is testing a drug for this kind of heart inflammation. This work provides a biological foundation for testing more targeted therapies for myocarditis due to an immune checkpoint inhibitor.
118 
The Pharma Data
AUGUST 4, 2020
Therapy has been approved to treat advanced NTRK fusion-positive solid tumours. The approval is based on results from the pivotal phase 2 STARTRK-2, phase 1 STARTRK-1 and phase 1 ALKA-372-001 trials, as well as data from the phase 2/3 STARTRK-NG study. Source link.
52 
Agency IQ
APRIL 19, 2024
Meet C3TI, FDA’s new clinical trial innovation hub On April 14, FDA announced that its Center for Drug Evaluation and Research (CDER) is launching a new clinical trial innovation hub, the CDER Center for Clinical Trial Innovation (C3TI). set specific standards and expectations for discrete components of clinical trials (e.g.,
The Pharma Data
JANUARY 17, 2021
18, 2021 /PRNewswire/ — Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, and Lonza announced today their agreement in support of ARU-1801, Aruvant’s one-time investigational gene therapy for sickle cell disease (SCD). NEW YORK , Jan.
52 
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.
Drug Target Review
SEPTEMBER 25, 2024
Most targeted cancer therapies used today operate by inhibiting targets along well-known oncogenic signalling cascades. The reactivation of oncogenic signalling upstream or downstream of the driving oncogene is a well-studied source of resistance to targeted cancer therapies.
105 
The Pharma Data
SEPTEMBER 12, 2020
Food and Drug Administration to expand the enrollment of their Phase 3 pivotal COVID-19 vaccine trial to up to approximately 44,000 participants which also allows for the enrollment of new populations. The pivotal trial is event-based and there are many variables that will ultimately impact read-out timing.
52 
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?)
The Pharma Data
AUGUST 11, 2020
.–( BUSINESS WIRE )– Bristol Myers Squibb (NYSE: BMY) today announced the Phase 3 CheckMate -577 trial evaluating Opdivo (nivolumab) as an adjuvant therapy for patients with resected esophageal or gastroesophageal junction (GEJ) cancer met its primary endpoint of disease-free survival (DFS) at a pre-specified interim analysis.
52 
The Pharma Data
NOVEMBER 12, 2020
12 November 2020 — The CALAVI Phase II trials for Calquence (acalabrutinib) in patients hospitalised with respiratory symptoms of COVID-19 did not meet the primary efficacy endpoint. No new safety signal for Calquence was observed in the trials. No new safety signal for Calquence was observed in the trials.
52 
Drug Target Review
OCTOBER 26, 2023
Genome engineering and gene therapies that manipulate DNA sequences in cells have driven a biotechnological revolution over the past decade. Endgame: glybera finally recommended for approval as the first gene therapy drug in the European union. Molecular Therapy 20 , 1831-1832 (2012). Russell S, et al. Wang D, Tai PW, Gao G.
105 
The Pharma Data
SEPTEMBER 8, 2020
Phase III clinical trial programme initiated for investigational medicine fenebrutinib, designed to be a highly selective and reversible Bruton’s tyrosine kinase (BTK) inhibitor, in relapsing multiple sclerosis (RMS) and primary progressive MS (PPMS).
The Pharma Data
DECEMBER 2, 2020
Based on the Phase 2 study findings to date, marstacimab may have the potential to offer improved bleed control via subcutaneous injection and potentially eliminate the need for prophylactic factor replacement, providing an enhanced treatment option compared to factor replacement therapy.”. In September 2019, the U.S.
52 
The Pharma Data
DECEMBER 29, 2020
As in earlier outpatient trial, immune status when patients entered the trial was a strong predictor of viral load and clinical outcomes. First antibody therapy to demonstrate anti-viral effect in patients hospitalized with COVID-19. Regeneron Pharmaceuticals, Inc. futility analysis). Yancopoulos , M.D.,
52 
The Pharma Data
NOVEMBER 20, 2020
NASDAQ: REGN) today announced that the antibody cocktail casirivimab and imdevimab administered together (formerly known as REGN-COV2 or REGEN-COV2), a therapy currently being investigated for use in COVID-19 , has received Emergency Use Authorization (EUA) from the U.S. TARRYTOWN, N.Y., November 21, 2020 – Regeneron Pharmaceuticals, Inc.
52 
The Pharma Data
DECEMBER 1, 2020
NASDAQ: AXSM), a biopharmaceutical company developing novel therapies for the management of central nervous system (CNS) disorders, today announced positive results from the open-label Phase 2 COMET-TRD trial of AXS-05 in patients with treatment resistant depression (TRD). AXS-05 was well tolerated in the COMET trial.
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The Pharma Data
JANUARY 21, 2021
For one, the patients were not part of a clinical trial that directly tested stem cell transplants against standard MS medications. They all underwent transplants at various Italian medical centers between 1997 and 2019. At the same time, there are important caveats, said Bebo, who was not involved in the study.
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DrugBank
OCTOBER 4, 2024
The FDA's accelerated approval pathways, such as the Breakthrough Therapy designation and the Priority Review program, have shortened development timelines and reduced costs for certain drugs, making them more attractive acquisition targets. However, such acquisitions also entail significant risks for biotech firms.
Drug Target Review
OCTOBER 25, 2024
We’re investigating powerful tools that may allow for an earlier – and biologically based – prediction of Alzheimer’s disease, which are being evaluated to help identify patients for clinical trials. 8 These may also help in identifying or validating novel targets for therapy beyond A and T. 2019, June 11). Meneses, A.,
59 
The Pharma Data
NOVEMBER 18, 2020
This Phase 1 trial will evaluate the safety, tolerability, and pharmacokinetics of SEFA-1024 in 96 otherwise healthy volunteers with elevated plasma triglyceride levels. A Phase 2b study was initiated in July 2019 (ICONA) to study the efficacy of icosabutate in NASH. 19, 2020 09:00 UTC. SEFA-1024 is one of NST’s three SEFA programs.
40 
The Pharma Data
JANUARY 17, 2021
The clinical trial to evaluate the safety and efficacy of the plasma-derived medicine will have doctors Oriol Mitjà and Bonaventura Clotet as the principal researchers and is expected to begin in early 2021. In 2019, Grifols’ economic impact in its core countries of operation was 8.5 BARCELONA, Spain , Jan.
Alta Sciences
APRIL 17, 2024
Psychedelics Psychedelic therapy (or psychedelic-assisted therapy) refers to the use of psychedelic drugs, such as psilocybin, MDMA, LSD, ketamine, and ayahuasca, to treat mental disorders, especially those that have no effective treatments available or are treatment resistant.
The Pharma Data
NOVEMBER 2, 2020
(NASDAQ:ADIL; ADILW) , a clinical-stage biopharmaceutical company focused on the development of treatments for addictions, today provided an update on its landmark ONWARD Phase 3 pivotal trial. 40% of expected Trial patient screenings have been completed and the Trial is more than 25% enrolled relative to planned enrollment.
52 
Drug Target Review
MAY 18, 2023
Tedesco Distinguished Chair of Paediatric Haematology and Oncology, are collaborating as co-principal investigators on a new clinical trial, which has been awarded a $2.3 The trial represents an early-stage effort to determine the viability of this approach. Kutlar says the possibilities of this additional therapy are exciting.
80 
Vial
MAY 15, 2024
Vial | Pros and Cons The unprecedented consequences of the COVID-19 pandemic gave rise to the advent of decentralized clinical trials (DCTs), which are studies that use telemedicine and remote or local healthcare professionals to enable participants to join from different locations. billion and now functions as a subsidiary.
Eye on FDA
JULY 5, 2022
Most notably in 2012 the Food and Drug Administration Safety and Innovation Act (FDASIA) was signed into law, bring to bear among other things, breakthrough therapy designation. The most obvious impact is that of COVID-19 to recruiting and maintaining clinical trials. Since that time, there has been a healthy output of new treatments.
81 
The Pharma Data
APRIL 25, 2021
Nirsevimab demonstrate d protect ion against respiratory syncytial virus disease in healthy infants in Phase 3 trial. The overall safety profile of nirsevimab in the trial remains consistent with previously reported results. Lurie Children’s Hospital of Chicago, Illinois, US and primary investigator of the MELODY Phase III trial.
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The Pharma Data
JANUARY 31, 2021
Clover plans to initiate a global Phase 2/3 efficacy trial of its protein-based S-Trimer COVID-19 vaccine candidate adjuvanted with Dynavax’s CpG 1018 plus alum in the first half of 2021 with an interim analysis for vaccine efficacy potentially in the middle of 2021. EMERYVILLE, Calif. and CHENGDU, China , Feb.
52 
The Pharma Data
OCTOBER 22, 2020
. INTERLINK-1 represents first Phase 3 study examining IO approach in R/M SCCHN patients who have been treatedwith a platinum-based therapy and PD-(L)1 inhibitor. Dosing of the first patient in this trial has triggered a $50 million milestone payment from AstraZeneca to Innate. “W MARSEILLE, France, Oct.
Advarra
AUGUST 16, 2022
Orphan drugs have historically faced a number of barriers, such as limited research and development (R&D) investment due to an expected lack of profitability as well as challenges in clinical trial design and recruitment. Before 1983, only 38 orphan drugs had received U.S. Food and Drug Administration (FDA) approval. A Lasting Impact.
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The Pharma Data
JANUARY 12, 2021
Preclinical data presented at the 2020 Society for Immunotherapy of Cancer’s Annual Meeting and the 2019 American Association for Cancer Research Annual Meeting support the development of JTX-8064 as a novel immunotherapy to reprogram immune-suppressive macrophages and enhance anti-tumor immunity.
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