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

MARCH 9, 2022

Sanofi and Swedish Orphan Biovitrum AB (publ) (Sobi ® ) (STO:SOBI) today announced positive topline results from the pivotal XTEND-1 Phase 3 study evaluating the safety, efficacy and pharmacokinetics of efanesoctocog alfa (BIVV001) in previously treated patients ? 12 years of age with severe hemophilia A.

Treatment 52

Treatment Pharmacokinetics Therapies FDA 52

The Pharma Data

MARCH 9, 2022

PAXLOVID™ (nirmatrelvir [PF-07321332] tablets and ritonavir tablets) is the first oral therapy specifically designed to combat COVID-19 to be evaluated in a pediatric clinical study PAXLOVID is currently authorized under U.S. Pfizer Inc. The safety and effectiveness of PAXLOVID have not yet been directly established in pediatric patients.

Treatment 52

Treatment Pharmacokinetics Hospitals Trials 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

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 Pharmacokinetics 92

The Pharma Data

MAY 5, 2021

It also provided supporting pharmacokinetic data demonstrating the opioid antagonist’s safety and efficacy. . A total of 197 patients in the study arm had at least one of the composite endpoint events, compared with 312 patients in the placebo arm. controlled trial had not received previous systemic therapy for metastatic disease.

FDA Approval 52

FDA Approval FDA Therapies Pharmacokinetics 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

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 Production 52

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 114

Production Small Molecule Regulations Trials 114

The Pharma Data

APRIL 14, 2021

government for its neutralizing antibody therapies authorized for emergency use as a treatment for COVID-19. who require oxygen therapy due to COVID-19, OR. who require an increase in baseline oxygen flow rate due to COVID-19 in those on chronic oxygen therapy due to underlying non-COVID-19 related comorbidity. Adverse Events.

Government 52

Government Long-Term Care Facilities Hospitals Treatment 52

The Pharma Data

JANUARY 26, 2021

Across 1,035 patients, there were 11 events (2.1 percent) in patients taking therapy and 36 events (7.0 Bamlanivimab and etesevimab together also demonstrated statistically significant improvements on all key secondary endpoints, providing strong evidence that the therapy reduced viral load and accelerated symptom resolution.

Hospitals 52

Hospitals Treatment Trials Long-Term Care Facilities 52

The Pharma Data

DECEMBER 21, 2020

As UNITY reported in its press release, this “Phase I, first-in-human, open-label, single-ascending dose study being conducted by UNITY is designed to evaluate the safety, tolerability, and pharmacokinetics of UBX1325 in patients with advanced DME. SUZHOU, China and ROCKVILLE, Md. , 22, 2020 /PRNewswire/ — Ascentage Pharma (6855.HK),

Clinical Development 40

Clinical Development Disease Treatment Licensing 40

The Pharma Data

NOVEMBER 17, 2021

The EC’s blessing of VUMERITY is grounded on data from pharmacokinetic bridging studies comparing VUMERITY and TECFIDERA to establish bioequivalent exposure of monomethyl fumarate, the active metabolite, and reckoned in part on the well- established long- term efficacity and safety profile of TECFIDERA. Encyclopedically, an estimated2.8

Treatment 52

Treatment Marketing Clinical Trials Biosimilars 52

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 Therapies Treatment Trials 52

The Pharma Data

APRIL 7, 2021

The new route of administration offers comparable efficacy and safety to the TYSABRI intravenous (IV) formulation building on the therapy’s long-term data, established clinical benefits and well-characterized safety profile. TYSABRI is a trusted high-efficacy therapy with a well characterized safety profile for patients living with MS.

Clinical Trials 52

Clinical Trials Treatment Virus Pharmacokinetics 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

MARCH 17, 2021

Verzenio (abemaciclib) : Abstract # P008 : Patients’ quality of life and side effect perceptions in monarchE, a study of abemaciclib plus endocrine therapy in adjuvant treatment of HR+, HER2-, node-positive, high risk, early breast cancer (Sara M. Accepted as ePoster – Adjuvant Systemic Therapy. Selected for one of the three St.

Trials 52

Trials Therapies Treatment Pharmacokinetics 52

The Pharma Data

MARCH 9, 2021

The primary endpoint was the maximum change in plasma HIV-1 RNA during parts one and two while secondary endpoints measured safety, tolerability, and pharmacokinetic (PK) parameters. There were no adverse events (AEs) leading to discontinuation and no deaths reported. log 10 and -2.0 About ViiV Healthcare.

Treatment 52

Treatment RNA Pharmacokinetics Virus 52

The Pharma Data

APRIL 16, 2021

who require oxygen therapy due to COVID-19, OR. who require an increase in baseline oxygen flow rate due to COVID-19 in those on chronic oxygen therapy due to underlying non-COVID-19 related comorbidity. Serious and unexpected adverse events may occur that have not been previously reported with bamlanivimab and etesevimab together.

Treatment 52

Treatment Hospitals Long-Term Care Facilities Virus 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

Codon

APRIL 2, 2023

Read A one-time gene therapy injection for spinal muscular atrophy. Mice and human cells that were given the therapy had normal levels of the survival motor neuron protein, and no symptoms. Gene Therapy. Read A really simple way to isolate AAVs, which are a type of virus often used to deliver gene therapies into cells.

DNA 52

DNA RNA Engineering Licensing 52

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.

Small Molecule 40

Small Molecule Clinical Trials Trials Therapies 40

The Pharma Data

SEPTEMBER 26, 2021

The positive CHMP opinion was supported data from pharmacokinetic bridging studies comparing VUMERITY and TECFIDERA® (dimethyl fumarate) to determine bioequivalent exposure of monomethyl fumarate, the active metabolite, and relied partially on the well-established long-term safety and efficacy profile of TECFIDERA. for TECFIDERA.

Treatment 40

Treatment Clinical Trials Virus Pharmacokinetics 40

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

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

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 52

Treatment Trials FDA Hospitals 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

FEBRUARY 8, 2021

Grade 3/4 treatment emergent adverse events (TEAEs) were reported in 63.3 1 Quality of life was comparable between treatment arms per Functional Assessment of Cancer Therapy–Prostate (FACT-P Total). Grade 3/4 treatment emergent adverse events (TEAEs) were reported in 63.3 percent of patients in the combination arm versus 56.2

Pharmaceutical Companies 52

Pharmaceutical Companies Treatment Clinical Trials Therapies 52

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

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

SEPTEMBER 15, 2020

Of note, The Phase 1b study is a double-blind, placebo-controlled clinical trial evaluating the safety, tolerability and pharmacokinetics of PF-07304814, a phosphate prodrug that when administered intravenously is metabolized to the active compound PF-00835321, shown to be a very potent inhibitor of the SARS-Cov2 3CL protease in preclinical studies.

Vaccine 52

Vaccine Therapies Trials Clinical Trials 52

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

Agency IQ

OCTOBER 20, 2023

FDA’s newest draft guidance lays out considerations for developing treatments for diabetic foot infections In a new draft guidance document released within weeks of a related clinical practice guideline, the FDA provides considerations for developing therapies to treat diabetic foot infections, focusing on Phase 3 efficacy trials.

FDA 40

FDA Treatment Trials Therapies 40

The Pharma Data

JANUARY 3, 2021

Dr. Kimble Matos, the lead coordinating physician of the study, commented: “The observations made during the Clinical study did not show any adverse events.”. The pharmacokinetics of nasally administered Foralumab will also be evaluated.

Clinical Trials 40

Clinical Trials Science Treatment Trials 40

The Pharma Data

FEBRUARY 8, 2021

IBRANCE is indicated for the treatment of adult patients with HR+, HER2- advanced or metastatic breast cancer in combination with an aromatase inhibitor as initial endocrine based therapy in postmenopausal women or in men; or with fulvestrant in patients with disease progression following endocrine therapy. 2,3 In the U.S.,

Clinical Trials 52

Clinical Trials Treatment Therapies Production 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