Drug Target Review

JUNE 12, 2023

TEAD proteins are known to be very important in cancer progression TEAD proteins are known to be very important in cancer progression, and there are a number of therapies in development. What are the preclinical characteristics of ISM6331, including its efficacy, safety profile, and drug metabolism and pharmacokinetics (DMPK) properties?

Metabolic Stability 92

Metabolic Stability Clinical Trials Small Molecule Therapies 92

Conversations in Drug Development Trends

DECEMBER 20, 2023

Single-Arm Trial Design Single-arm trials have also become an increasingly common development strategy to support regulatory approval and allow patients expedited access to novel therapies, particularly in the accelerated access setting.

Clinical Trials 83

Clinical Trials Trials Pharmacokinetics FDA 83

LifeSciVC

APRIL 24, 2024

If adverse events are anticipated, it is important to understand gene dosage for such an effect (e.g., with gene editing or gene therapy, enzyme replacement therapy), agonism (e.g., adverse profile seen in homozygous null individuals but not in heterozygous carriers?) in liver, in CNS)? with antibodies), or correction (e.g.,

Production 117

Production Small Molecule Regulations Trials 117

DrugBank

APRIL 3, 2025

This increases costs and delays the introduction of potentially life-saving therapies. These studies typically include in vitro assays to evaluate cytotoxicity and in vivo models to study pharmacokinetics, pharmacodynamics, and toxicological profiles. Preclinical studies form the backbone of early safety assessment.

Drug Development 52

Drug Development Drugs Clinical Trials Pharmacokinetics 52

The Pharma Data

JULY 12, 2021

These data include results from a late-breaking presentation from a Phase 2a study evaluating the safety and pharmacokinetics (PK) of once-monthly (QM) oral islatravir for pre-exposure prophylaxis (PrEP) through 24 weeks. Safety and Pharmacokinetics of Islatravir in Study Participants with Severe Renal Insufficiency. Abstract 2361.

Pharmacokinetics 52

Pharmacokinetics Clinical Trials Trials Treatment 52

The Pharma Data

JANUARY 31, 2021

Traumatic brain injury and other tauopathies can have devastating effects on cognitive function and quality of life, yet there are currently no therapies that can halt or slow the rate of decline in these patients,” said Martin Jefson, Ph.D., Pinteon’s chief executive officer. “We

Pharmacokinetics 52

Pharmacokinetics Disease Treatment Therapies 52

Advarra

JUNE 20, 2023

cell and gene therapies), with other therapeutic areas then pushing it further. Because of this, oncology researchers may only have access to minimal existing scientific literature related to cell-based data and animal testing to predict an investigational therapy’s effect on human factors.

Trials 52

Trials Clinical Trials FDA Approval Therapies 52

The Pharma Data

JUNE 28, 2021

Current guidelines are limited and recommend treating pediatric patients with or at risk for reoccurrence of blood clots with standard anticoagulation therapy which requires injections, dietary restrictions, and regular laboratory monitoring. Study The randomized, open-label phase III EINSTEIN-Jr. UNIVERSE was conducted in two parts.

FDA 52

FDA Clinical Trials Treatment Therapies 52

The Pharma Data

MARCH 13, 2021

Takeda Pharmaceutical Company Limited ( TSE:4502/NYSE:TAK ) (“Takeda”) today announced that it has submitted a New Drug Application (NDA) to the Ministry of Health, Labour and Welfare (MHLW) in Japan for lanadelumab subcutaneous injection, a monoclonal antibody therapy for prophylaxis against attacks of hereditary angioedema (HAE).

Treatment 52

Treatment Pharmacokinetics Drugs Therapies 52

Drug Target Review

JULY 1, 2024

Could you elaborate on the potential market size for SRP-001 and how it fits into the broader landscape of pain therapies? SRP-001 is a novel non-opioid pain relief candidate that works centrally in the brain, offering robust pharmacokinetics without the adverse effects of current medications. South Rampart Pharma, Inc.

Treatment 59

Treatment Clinical Trials Therapies Molecular Biology 59

The Pharma Data

APRIL 11, 2021

In this cohort, the most common treatment-emergent adverse events of any grade (?20%) No patients in this cohort discontinued treatment due to treatment-related adverse events. Initiate or adjust anti-hypertensive therapy as appropriate. Serious, including fatal, hemorrhagic events can occur with Retevmo.

Research 52

Research Treatment Therapies Trials 52

The Pharma Data

DECEMBER 9, 2020

The first trial will be conducted as a Phase 1, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, and pharmacokinetics of the intravenous liposomal formulation of ibrexafungerp in healthy subjects. The study will be conducted in South Africa. For more information, visit www.scynexis.com.

Hospitals 52

Hospitals Pharmacokinetics Treatment Trials 52

The Pharma Data

AUGUST 19, 2021

3] While MET inhibitors have recently received accelerated approval in this setting in some regions, the vast majority of patients eventually acquire resistance to these therapies, thus underscoring the need for new treatment options. [4] The majority of treatment-related adverse events (AEs) were Grade 1-2. All events were Grade 1-2.

Treatment 52

Treatment FDA Approval Pharmacokinetics FDA 52

The Pharma Data

JANUARY 3, 2021

The Phase 1/2 clinical trial will primarily investigate the safety and tolerability of INZ-701 and characterize its pharmacokinetic and pharmacodynamic profile, including plasma pyrophosphate (PPi) and other biomarker levels, to establish a recommended dosing regimen for further clinical development.

Clinical Trials 52

Clinical Trials Trials Treatment FDA 52

The Pharma Data

AUGUST 29, 2020

Oral, investigational complement factor B inhibitor LNP023 substantially improved hematological response as add-on therapy to eculizumab. To date, after at least six months of stable LNP023 add-on therapy, and at the investigators’ discretion, seven patients (70%) have discontinued eculizumab and remained on LNP023 as monotherapy.

Treatment 52

Treatment Disease Small Molecule Production 52

The Pharma Data

DECEMBER 29, 2020

PRX-102 was well-tolerated in the study, with all adverse events being transient in nature without sequelae. The most common moderate treatment emergent adverse events were nasopharyngitis, headache and dyspnea. The most common moderate treatment emergent adverse events were nasopharyngitis, headache and dyspnea. nM and 13.81

Clinical Trials 52

Clinical Trials Disease Treatment Trials 52

The Pharma Data

JANUARY 17, 2021

18, 2021 /PRNewswire/ — Genkyotex SA , a subsidiary of Calliditas Therapeutics AB (publ) (“Calliditas”) (Nasdaq OMX – CALTX; NASDAQ – CALT), today announced positive Phase 1 data demonstrating a favorable safety and pharmacokinetic profile of high-dose setanaxib, Genkyotex’s lead asset. STOCKHOLM , Jan.

Trials 40

Trials Clinical Trials Pharmacokinetics Small Molecule 40

The Pharma Data

OCTOBER 18, 2020

Several clinical sites are screening patients for the Phase 1 a/b multicenter, open-label, dose escalation study of safety, pharmacodynamics, and pharmacokinetics of CG-806 in ascending cohorts (3+3 design) to determine the maximum tolerated dose or recommended dose in patients with relapsed or refractory AML. About CG-806.

Clinical Trials 40

Clinical Trials Small Molecule Trials Therapies 40

The Pharma Data

SEPTEMBER 19, 2020

2.80] in the PD-L1 positive population, based on 21% of patients with an event; updated HR=1.12 [95% CI: 0.76-1.65]), 1.65]), updated analysis based on 41% of patients with an event). Secondary endpoints include OS, event-free survival, disease-free survival and quality of life measures.

Treatment 52

Treatment Disease Therapies Pharmaceuticals 52

The Pharma Data

APRIL 8, 2021

FDA and global approvals for our groundbreaking therapies in SMA and NMOSD, Roche’s data at AAN reflect our continued commitment to meaningful therapeutic progress for people living with neurological disorders,” said Levi Garraway, M.D., Following U.S. Roche’s Chief Medical Officer and Head of Global Product Development. Claims Database.

Clinical Trials 52

Clinical Trials Trials Disease Therapies 52

The Pharma Data

JULY 22, 2021

No new risks were identified, with no serious adverse events related to rVWF reported. 1 The pharmacokinetics (PK) of VWF:ristocetin cofactor (VWF:RCo) and FVIII pharmacodynamics (PD) [PB0917] were also studied and presented at the congress. The sABR was reduced by 91.5% About von Willebrand Disease (VWD). About Takeda Hematology.

Treatment 52

Treatment Laboratories Production Disease 52

The Pharma Data

OCTOBER 25, 2020

Treatment choices are limited, and commonly used off-label therapies are not always effective or may result in prolonged, deep sedation. A therapy that quickly and effectively reduces agitation, without causing excessive sedation, is needed to speed up recovery time and improve patient outcomes. NEW HAVEN, Conn.,

Treatment 40

Treatment Trials FDA Hospitals 40

The Pharma Data

DECEMBER 9, 2020

The open-label Phase 2a ‘AMBITION’ study is designed to assess safety, tolerability, pharmacokinetics and biomarker analyses for early assessments of efficacy of 75 mg and 225 mg CRV431, administered orally to F2 and F3 NASH patients (n=18/dosing group), once daily for 28 days. ” About Hepion Pharmaceuticals.

Clinical Trials 40

Clinical Trials Trials Pharmaceuticals Pharmacokinetics 40

The Pharma Data

DECEMBER 13, 2020

The Phase 2b trial will also evaluate efficacy on other histology endpoints (fibrosis), assessment of metabolic and non-metabolic parameters, pharmacokinetic assessment as well as safety and tolerability. NASH Investor Event Information. A live webcast of the event will be available on Poxel’s website at [link] under Events.

Trials 40

Trials Disease Treatment Pharmacokinetics 40

The Pharma Data

JULY 11, 2021

For more than a decade, we’ve listened to the HAE community to further understand the need for long-term, preventive targeted therapies and have committed our resources to developing treatment options,” said Neil Inhaber, M.D., percent of patients (n=116) and the most common being injection-site pain. Interactions. Adverse Reactions.

Treatment 52

Treatment Pharmacokinetics Disease Production 52

The Pharma Data

JULY 27, 2021

Each of these three investigational therapies uses a different approach to treat a highly prevalent disease for which new treatment options are very much needed. Our stock price is volatile and may be affected by a number of events. ” In June 2021, AbbVie Inc. exercised its right to acquire TeneoOne, Inc.

Production 52

Production Clinical Trials Government Disease 52

The Pharma Data

DECEMBER 5, 2020

” In the trial, the 49 patients evaluated had a median of five prior lines of therapy (range: 2-17) with 100% being triple-refractory and 57% being penta-refractory; all patients were refractory to anti-CD38 therapy. With a median follow up of 2.6 74% of responders (n=14) remain on treatment. About Regeneron Pharmaceuticals, Inc.

Production 40

Production Trials Treatment Disease 40

The Pharma Data

JANUARY 10, 2021

TransCon PTH: TransCon PTH is an investigational long-acting prodrug of parathyroid hormone (PTH) in development as a potential once-daily replacement therapy for adult hypoparathyroidism (HP): . . •. ?. . ?. . . •.

Clinical Trials 40

Clinical Trials Clinical Development Trials Small Molecule 40

The Pharma Data

NOVEMBER 22, 2020

Preclinical data support MP0420’s potential efficacy as both a prophylactic and as an acute therapy. MP0420 is designed to bind the receptor-binding domain (RBD) of the SARS-CoV-2 spike protein at three distinct locations to prevent viral entry into cells. Chief Medical Officer of Molecular Partners.

Trials 40

Trials Clinical Trials Licensing Licensing 40

Agency IQ

AUGUST 16, 2024

However, the agency can describe a concomitant therapy in labeling for a product it has authority to regulate if it is essential for the therapeutic effect. While the ability to describe and specify aspects of the therapy is limited, the agency can specify the number and licensure of therapists who would participate in the MDMA treatment.

FDA 40

FDA Trials Therapies Regulations 40

The Pharma Data

AUGUST 30, 2021

For the 16-week treatment period, overall rates of adverse events (AEs) were 64% for Dupixent and 74% for placebo. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for Dupixent for the treatment of severe atopic dermatitis (in children aged 6 months to 11 years of age). In 2016, the U.S.

Trials 52

Trials Disease Treatment Clinical Trials 52

Agency IQ

FEBRUARY 23, 2024

Additionally, this approach is generally no longer applicable to new treatments that are more targeted in their effects, such as immunotherapies and targeted small molecule therapies. This has spawned a movement focused on optimizing dose selection in oncology drug development. We need to think [now] that not always MTD is the approach.”

Science 40

Science FDA Trials Clinical Pharmacology 40

The Pharma Data

NOVEMBER 4, 2020

Food and Drug Administration (FDA) Breakthrough Therapy and Fast Track Designations, is now in pivotal testing, and CTP-692 for schizophrenia is currently on track for topline data readout in the first quarter of 2021,” said Roger Tung, Ph.D., CTP-543 for moderate to severe alopecia areata, which received U.S.

Pharmaceuticals 52

Pharmaceuticals Clinical Trials Trials Treatment 52

The Pharma Data

OCTOBER 27, 2020

Significant improvements were also observed in each of the study’s secondary endpoints, including reduction in the cardiac biomarker NT-proBNP, time to first clinical worsening event, change in peak 6MWD at Week 12, and change in trough 6MWD at week 15. Autologous cell therapy for PAH — SAPPHIRE. ADVANCE OUTCOMES.

FDA 40

FDA Production Licensing Licensing 40

Agency IQ

SEPTEMBER 15, 2023

The committee also made recommendations regarding pharmacokinetic and safety assessments. When looking at several pharmacokinetic studies, the FDA found that the 10 mg oral dose of phenylephrine has a very low bioavailability of less than 1% and, subsequently, low systemic alpha-1 adrenergic activity. “We

Science 40

Science FDA Clinical Trials Clinical Pharmacology 40

The Pharma Data

JULY 21, 2021

Most adverse events (AEs) were mild and there were no serious drug-related AEs in people who received islatravir. The company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise.

Clinical Trials 52

Clinical Trials Pharmacokinetics Trials International 52

DrugBank

FEBRUARY 26, 2025

This stagnation has created a perfect storm, threatening the effectiveness of current therapies and limiting the pipeline of new drugs. Advances in pharmacology and drug formulation have enabled researchers to refine these therapies and extend their utility.

Therapies 52

Therapies Pharmaceutical Companies Pharmacokinetics Treatment 52