Drug Target Review

NOVEMBER 7, 2023

Although PROTACs are currently in clinical trials and have shown efficacy in treating cancer , they can only target a protein if it is inside the cell, which is 60 percent of the time. To test which patients are more likely to respond to LYTAC therapy, the level of neddylated CUL3 could be measured. This study was published in Science.

Therapies 59

Therapies Science Disease Treatment 59

Eye on FDA

AUGUST 4, 2021

Below is a chart that looks at 3 numbers – the number of AdComms; the number of new molecular entities approved each year, and the number of drugs that had breakthrough therapy designation. Fewer meetings – more approvals.

FDA 122

FDA Clinical Pharmacology FDA Approval Therapies 122

The Pharma Data

DECEMBER 22, 2020

Tagrisso Approved as Adjuvant Therapy for NSCLC With EGFR Mutations. Tagrisso was approved in 2018 for first-line treatment of patients with metastatic non-small cell lung cancer with EGFR exon 19 deletions or exon 21 L858R mutations. Professional. TUESDAY, Dec. Food and Drug Administration announced Friday.

Therapies 52

Therapies Treatment FDA Disease 52

Codon

JULY 14, 2024

While this departs from the reductionist, single-component therapies that have dominated transfusion medicine since World War II, emerging data underscores that whole blood transfusions—blood with all its parts—yield better outcomes following severe blood loss than transfusions involving discrete blood components. Always free.

Production 141

Production Clinical Trials Trials Therapies 141

Advarra

OCTOBER 19, 2023

The clinical trial startup process has seen significant shifts over the past five years: growth in decentralized trials, improved technology, and an increased demand for accelerated timelines in getting therapies to market. Sponsor Technology and Training The role of technology in clinical trials has never been more pronounced.

Clinical Trials 52

Clinical Trials Trials Clinical Research Research 52

Drug Target Review

NOVEMBER 6, 2023

2 It is this complexity that necessitates powerful, targeted combination therapies. 4, 5 More recently, new combination regimens have emerged that incorporate targeted therapies to treat a variety of blood cancers, including multiple myeloma (MM), chronic lymphocytic leukaemia (CLL) and acute myelogenous leukaemia (AML).

Small Molecule 111

Small Molecule Therapies Treatment Disease 111

Drug Target Review

OCTOBER 11, 2023

1,2 Many, however, do have dysfunctional proteins at their core, and for these diseases, a new option is being explored: autophagy-boosting therapies. Symptoms were reversed in mouse models and a clinical trial is planned for later this year. Gene therapy, of course, also holds great promise. 2018 [cited 2023 Sep 5].

Disease 113

Disease Treatment Small Molecule Therapies 113

The Pharma Data

NOVEMBER 9, 2020

Talaris Chief Executive Officer Scott Requadt sees FCR001, the company’s investigational cell therapy, as a potential pipeline in a product (an experimental treatment that could have multiple uses across a number of indications). The trial is expected to enroll 120 adult patients who will receive kidney donations from living donors.

Therapies 52

Therapies Production Disease Treatment 52

PLOS: DNA Science

JUNE 1, 2023

The newest FDA-approved gene therapy treats the severe, skin-peeling condition dystrophic epidermolysis bullosa (DEB). The gene treatment has been a long time coming, but it differs from the handful of other approved gene therapies: it isn’t a one-and-done. DEB has been a candidate for a gene therapy since 2002.

FDA Approval 52

FDA Approval Therapies FDA Disease 52

Drug Target Review

JANUARY 25, 2024

The peptide in development is based on a 2018 Cleveland Clinic discovery and serves as a proof-of-concept for this type of drug for triple-negative breast cancer. Currently, they are exploring the drug’s potential for clinical trials. This study was published in Molecular Cancer Therapeutics. References 1 Marqus S, Pirogova E, Piva TJ.

Treatment 105

Treatment Clinical Trials Science Research 105

Conversations in Drug Development Trends

FEBRUARY 28, 2023

Over the next few decades, that drug made its way through clinical trials, securing approval in 2007—just 36 months before Rob was diagnosed in 2010. If we don’t fund this research, we never have the chance for treatments, cures, or innovative therapies. But what was truly remarkable? It really has to start from square one,” says Rob.

Clinical Trials 52

Clinical Trials Disease Trials Research 52

Drug Target Review

MARCH 3, 2025

In this interview, Professor Schwamborn discusses his research, the promise of brain organoids, and his vision for the future of stem cell-based therapies. ” By using personalised models, researchers can identify subgroups of patients with similar molecular characteristics, potentially leading to the development of targeted therapies.

Disease 52

Disease Treatment Therapies Cell Biology 52

PPD

SEPTEMBER 6, 2023

Approaches to outsourcing clinical trials have changed significantly in recent years. Mixing of service models — a strategy that drug developers are leveraging now more than ever — can bring life-changing therapies to market faster. Growth of the FSP market is steadily increasing. Overseen by an insourced project manager.

Drug Development 98

Drug Development Clinical Trials Drugs Clinical Research 98

The Pharma Data

JANUARY 13, 2021

PITTSBURGH–( BUSINESS WIRE )– Knopp Biosciences LLC today announced positive top-line results in a Phase 2 dose-ranging trial of the novel oral drug dexpramipexole in patients with moderate-to-severe eosinophilic asthma. The trial was conducted at 28 U.S. 14, 2021 11:00 UTC. study centers. ABOUT THE EXHALE STUDY.

Trials 52

Trials Small Molecule Clinical Trials Treatment 52

Codon

JANUARY 21, 2024

Human challenge trials were an indispensable part of the development of the malaria vaccine, R21/Matrix-M, endorsed by the World Health Organization last October. But for all of their benefits, human challenge trials have their drawbacks. Jake himself has participated in both Zika and Shigella challenge trials.

Vaccine 126

Vaccine Trials Disease Treatment 126

The Pharma Data

NOVEMBER 9, 2020

Trial Also Met the Primary Endpoint in Patients With Low Levels of Eosinophils. In the subgroup of patients with baseline eosinophil counts less than 300 cells per microliter, the trial met the primary endpoint with tezepelumab demonstrating a statistically significant and clinically meaningful reduction in AAER.

Trials 52

Trials Production Clinical Trials Government 52

The Pharma Data

JANUARY 31, 2021

Adjuvanted S-Trimer COVID-19 vaccine candidates demonstrated favorable safety and tolerability profiles and strong neutralizing immune responses in a phase 1 trial. Clover plans to initiate a global phase 2/3 trial in the first half of 2021 with an interim analysis for vaccine efficacy potentially in the middle of 2021.

Clinical Trials 52

Clinical Trials Vaccine Trials Virus 52

Drug Target Review

JUNE 21, 2023

Enabling the systematic discovery of these largely uncharted targets can be a valuable opportunity for development of novel therapies. Genetic-based therapeutics hold significant promise in the clinic Despite the growing number of genes linked to human disease, the proportion of genes targeted by approved therapies remains low.

Disease 111

Disease Clinical Trials Therapies Science 111

The Pharma Data

OCTOBER 20, 2020

In hopes to advance the best and brightest, MD Anderson, in partnership with The Focus Fund GP, has launched the Cancer Focus Fund to propel compelling investigational cancer therapies from preclinical development through Phase II clinical trials. But now they’ll be choosing the therapies themselves. . Cancer is still the No.

Clinical Trials 52

Clinical Trials Therapies Pharma Companies Trials 52

The Pharma Data

JANUARY 14, 2021

(NASDAQ: FPRX) today announced clinical results from the global, randomized, double-blind placebo-controlled Phase 2 FIGHT trial evaluating first-in-class targeted therapy bemarituzumab in advanced gastric or gastroesophageal junction (GEJ) cancer. Phase 2 FIGHT Trial: Summary of Efficacy*. Wainberg, M.D.,

Trials 52

Trials Treatment Therapies Clinical Trials 52

The Pharma Data

MARCH 15, 2021

Trial enrolled patients with advanced cervical cancer regardless of PD-L1 status. The trial will be stopped early based on a unanimous recommendation by the Independent Data Monitoring Committee (IDMC), and the data will form the basis of regulatory submissions in 2021. This is reflected in the trial where the average age was 51.”.

Trials 52

Trials Disease FDA Treatment 52

Drug Target Review

AUGUST 24, 2023

Neutralising GDF-15 with CatalYm’s anti-GDF-15 antibody visugromab was shown to reverse its inhibitory effects and to re-sensitise tumours to anti-PD-1 treatment, achieving commensurate survival benefit of anti-GDF-15-anti-PD-1 combination therapy in vivo. in North America) in late 2017.

Treatment 98

Treatment Therapies Clinical Development Cell Biology 98

DrugBank

OCTOBER 4, 2024

For example, Takeda Pharmaceutical's $62 billion acquisition of Shire plc in 2018 expanded its global footprint and tapped into Shire's strong presence in emerging markets. These deals usually provide a much-needed infusion of capital, enabling the acceleration of clinical trials and expansion of manufacturing capabilities.

Pharmaceuticals 98

Pharmaceuticals Pharmaceutical Companies Therapies Marketing 98

The Pharma Data

DECEMBER 9, 2020

9, 2020 — More women with early-stage breast cancer may be able to safely skip chemotherapy after having surgery, according to initial results from a major clinical trial. The trial, conducted in nine countries, found that adding chemotherapy to hormone-blocking drugs brought no added benefit to a particular group of patients.

Trials 52

Trials Therapies Clinical Trials Treatment 52

New Drug Approvals

SEPTEMBER 8, 2024

2222514-07-8 ] Palopegteriparatide , sold under the brand name Yorvipath , is a hormone replacement therapy used for the treatment of hypoparathyroidism. [1] 5] History The effectiveness of palopegteriparatide was evaluated in a 26-week, randomized, double-blind, placebo-controlled trial that enrolled 82 adults with hypoparathyroidism. [5]

FDA 62

FDA International Trials FDA Approval 62

The Pharma Data

JULY 8, 2021

8) There were no clinically meaningful differences in safety results between the tezepelumab and placebo groups in the NAVIGATOR trial.(8) 8) There were no clinically meaningful differences in safety results between the tezepelumab and placebo groups in the NAVIGATOR trial.(8) About Severe Asthma. Globally, there are approximately 2.5

FDA 52

FDA Trials Therapies Treatment 52

The Pharma Data

DECEMBER 21, 2020

21, 2020 /PRNewswire/ — Amgen (NASDAQ:AMGN) and AstraZeneca today announced the SOURCE trial did not meet the primary endpoint of a statistically significant reduction in the daily oral corticosteroid (OCS) dose, without loss of asthma control, with tezepelumab compared to placebo. THOUSAND OAKS, Calif. ,

Trials 40

Trials Production Clinical Trials Therapies 40

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.

Clinical Trials 40

Clinical Trials Trials Cell Biology Production 40

Drug Target Review

SEPTEMBER 26, 2023

Early detection of neurotoxicity induced by potential new therapies is a major challenge, and hiPSC-neuronal cells may provide a solution. Dr Rockley previously completed an MSc in Cancer Pharmacology and have experience in clinical research from an oncology clinical trial coordinator role. 2018 Mar;17(3):167–81. 2016;81:37–46.

Drugs 111

Drugs FDA Disease Animal Testing 111

The Pharma Data

NOVEMBER 1, 2020

The trial is expected to enroll 160 patients. billion in 2018, and it is expected to be up to US$48.9 I am very delighted to learn the swift IND clearance by the US FDA, and we are initiating the phase II clinical trial immediately,” said Dr. Jasmine Cui, the co-founder, chairman, and Chief Executive Officer of InnoCare.

Clinical Trials 40

Clinical Trials Trials FDA Treatment 40

The Pharma Data

JANUARY 14, 2021

The approval was based on data from Study ADVL0912, a multicenter, single-arm, open-label trial in 121 patients ranging in ages from one to 21, and included 26 patients with r/r/, systemic ALK-positive ALCL after at least one systemic treatment. For the full year of 2019, Xalkoria brought in $530 million, an increase of 1% from 2018. “We

FDA Approval 52

FDA Approval FDA Therapies Treatment 52

The Pharma Data

JULY 8, 2021

Tezepelumab is the first and only biologic to consistently and significantly reduce asthma exacerbations in a broad population across Phase II and III clinical trials. The BLA was based on results from the PATHFINDER clinical trials programme, including results from the pivotal NAVIGATOR Phase III trial.

Treatment 52

Treatment FDA Clinical Trials Trials 52

New Drug Approvals

SEPTEMBER 10, 2024

2] [3] It is the first approval by the US Food and Drug Administration (FDA) of a systemic therapy for people with grade 2 astrocytoma or oligodendroglioma with a susceptible isocitrate dehydrogenase-1 or isocitrate dehydrogenase-2 mutation. [2] April 2018). mol), (R)-1,1,1-trifluoropropan-2-amine hydrochloride (0.39 6 August 2024.

FDA Approval 62

FDA Approval FDA Clinical Trials Trials 62

The Pharma Data

JANUARY 17, 2021

Based in Seattle, Washington, Sana focuses on in vivo and ex vivo cell engineering platforms to develop therapies for cancer, diabetes, cardiovascular disease, CNS disorders, and genetic diseases. When the company launched an IPO in late 2018, its valuation was $7 billion, which at the time was a record biotech IPO.

Marketing 52

Marketing Engineering Therapies Disease 52

FDA Law Blog: Drug Discovery

DECEMBER 13, 2023

An Opportunity to Convene this New Advisory Committee for All Rare Disease Therapies In 2018, HP&M attorneys Frank Sasinowski and James Valentine proposed a Rare Disease Center of Excellence (which we blogged about here ). Note that FDA is currently soliciting applications to staff this committee.

Disease 59

Disease Production FDA Drug Development 59

DrugBank

FEBRUARY 19, 2025

The application of Artificial Intelligence (AI) in drug discovery is rapidly transforming the pharmaceutical industry, offering opportunities to accelerate the identification of novel therapeutic targets, optimize molecule design, and enhance clinical trial efficiency. Adaptive trials allow researchers to modify specific parameters (e.g.,

Clinical Trials 52

Clinical Trials Drugs Trials Treatment 52

The Pharma Data

MAY 5, 2022

1 Clinical trials for new therapies in AML are becoming increasingly lengthy to complete based on the traditional clinical endpoint of survival. “Measuring MRD as a surrogate endpoint may help forge a path to bring new AML therapies to patients quickly.”

Disease 52

Disease Clinical Trials Drug Development Pharmaceutical Companies 52