The Premier Consulting Blog

MAY 31, 2023

While the type, number, and design of these studies vary based on product-specific characteristics, IND-enabling packages submitted to the FDA generally include key information about the pharmacology, pharmacokinetics, and toxicology of the product. All these studies need to be performed under GLP.

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LifeSciVC

APRIL 24, 2024

In the absence of a clinical trial result or FDA label to point to, how does one create the case and target product profile (TPP) around a new target? and whether a molecule’s pharmacology can help to mitigate safety risk. Finally, as part of a broader pathway analysis, consider potential implications for selectivity.

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Practical Cheminformatics

JANUARY 8, 2024

In November and December, several large pharmas held “AI Day” presentations featuring LLM applications for clinical trial data analysis. Many of these groups demonstrated the ability of LLMs to ingest large bodies of unstructured clinical data and subsequently generate tables and reports based on natural language queries.

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The Pharma Data

DECEMBER 15, 2020

SetPoint Medical received FDA Investigational Device Exemption (IDE) approval for a multicenter, double-blind, randomized, sham-controlled pivotal trial that will enroll up to 250 patients at 40 clinical trial sites in the U.S. Small Molecule Inhibitors. The secondary outcome involves pharmacokinetic endpoints.

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The Pharma Data

JANUARY 19, 2021

This small molecule therapy is presently in Phase 1 clinical trial for mild to moderate Alzheimer’s disease (AD), which is supported by a NIA R01 grant in healthy aged volunteers. CLARKSVILLE, Md., 20, 2021 (GLOBE NEWSWIRE) — Neuronascent Inc. , About Neuronascent, Inc.

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The Premier Consulting Blog

JANUARY 31, 2023

Of note, this guidance is specific to small molecule pharmaceuticals; similar guidance for biologics can be found in the ICH’s S6(R1) Preclinical Safety Evaluation for Biotechnology-Derived Pharmaceuticals. Pharmacokinetic and systemic exposure data is also important to consider.

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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.

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The Pharma Data

APRIL 11, 2023

Bayer will present the first clinical Phase 1 results on aryl hydrocarbon receptor (AhR) inhibitor BAY 2416964, the company’s most advanced Immuno-Oncology program. Based on these data, a first-in-human trial with BAY 2965501 in patients with advanced solid tumors was initiated and is currently enrolling patients.

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The Pharma Data

NOVEMBER 4, 2020

Initiated CTP-543 THRIVE-AA1 Phase 3 Trial in November 2020. The THRIVE-AA1 Phase 3 trial is a randomized, double-blind, placebo-controlled clinical trial of CTP-543 to evaluate hair regrowth using the Severity of Alopecia Tool (SALT) after 24 weeks of dosing in approximately 700 adult patients with moderate to severe alopecia areata.

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The Pharma Data

OCTOBER 27, 2020

An expanded Series A will support Orbus’ ongoing STELLAR study, a Phase III clinical trial studying eflornithine in patients with anaplastic astrocytoma, a type of brain tumor, whose cancer has recurred following radiation and adjuvant chemotherapy. Orbus Therapeutics . The $71 million funding includes an initial financing of $32.5

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The Pharma Data

OCTOBER 18, 2020

The 450mg starting dose in AML patients was selected because that dose, when administered to CLL patients being treated in a separate Phase 1 a/b trial, appeared safe, well tolerated and achieved plasma exposure levels that effectively inhibited phospho-FLT3 activity, which is a key driver of AML.”. Forward Looking Statements.

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Molecular Design

FEBRUARY 7, 2023

However, the CYP51 inhibitor posaconazole (an antifungal medication) showed poor efficacy in a clinical trials for chronic Chagas disease. Does this mean that CYP51 is a bad target? As is the case for chemical probes intended for in vivo use, you’ll want to be in a position to invoke the FDH.

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New Drug Approvals

APRIL 18, 2025

“Crinecerfont, a CRF1 Receptor Antagonist, Lowers Adrenal Androgens in Adolescents With Congenital Adrenal Hyperplasia” The Journal of Clinical Endocrinology & Metabolism. 108 (11): 28712878. doi : 10.1210/clinem/dgad270. PMC 10583973. PMID 37216921. External links “Crinecerfont (Code C174708)” NCI Thesaurus.

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New Drug Approvals

SEPTEMBER 13, 2024

2] As of July 2022, it is in phase 3 clinical trials for major depressive disorder. [2] 2] Like other kappa opioid antagonists currently under clinical investigation for the treatment of major depression, its efficacy may be compromised by the countervailing activation of pro-inflammatory cytokines in microglia within the CNS. [7]

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The Pharma Data

JANUARY 10, 2021

. During the second quarter of 2021, Ascendis expects to provide a 12-month OLE update and plans to submit a Clinical Trial Notification for a clinical trial evaluating TransCon PTH for adult HP in Japan. Ascendis reported pre-clinical data for TransCon IL-2 ?/? .

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Alta Sciences

APRIL 17, 2024

Our experts align the appropriate bioanalytical approach with the goals of the oligonucleotide drug development program, based on its size/type, the sensitivity and specificity required, as well as throughput needed based on the preclinical or clinical phase of the drug in development.

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The Pharma Data

AUGUST 29, 2020

About the Study NCT03439839 is a Phase II, multicenter, open-label, sequential 2-cohort trial to assess the safety, efficacy, tolerability and pharmacokinetics/pharmacodynamics of LNP023 in PNH patients (cohort 1: n=10) with active hemolysis despite treatment with eculizumab. Blood 2019;134(Suppl 1):3517. 3. Schubart A, et al.

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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.

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Broad Institute

OCTOBER 29, 2024

This small molecule reactivates the apoptosis cascade in tumor cells while sparing healthy cells in animal models. Previous clinical and laboratory studies on other MCL1 inhibitors have suggested that these molecules can impair heart cells, likely because of prolonged exposure to the compounds.

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PPD

APRIL 14, 2025

This expansion is creating opportunities for clinical trials related to a range of new therapy areas and their subpopulations. Participant fatigue in GLP-1 clinical trials The success of GLP-1 drugs in SELECT and other trials has biotech and biopharma companies racing to find which indications respond well to similar treatment.

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Sygnature Discovery

NOVEMBER 29, 2023

A new treatment for G4-targeted solid tumors including pancreatic cancer has been administered to patients for the first time in a Phase 1a clinical trial. This latest development follows the US Food and Drug Administration (FDA) giving clearance for QN-302 in July 2023 to proceed to this initial clinical trial stage.

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Sygnature Discovery

NOVEMBER 29, 2023

A new treatment for G4-targeted solid tumors including pancreatic cancer has been administered to patients for the first time in a Phase 1a clinical trial. This latest development follows the US Food and Drug Administration (FDA) giving clearance for QN-302 in July 2023 to proceed to this initial clinical trial stage.

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Agency IQ

JUNE 9, 2023

However, nonclinical studies are not conducted exclusively before human trials begin; they usually continue throughout the early phases of clinical trials to further assess a product’s performance on different metabolic pathways and at different doses.

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The Pharma Data

OCTOBER 14, 2020

The designation of lanifibranor as a Breakthrough Therapy for the treatment of NASH follows the publication in June 2020 of positive topline results from Inventiva’s NATIVE Phase IIb clinical trial with lanifibranor in NASH patients. The trial plans to evaluate safety, tolerability, cytokine profile and efficacy parameters.

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DrugBank

FEBRUARY 26, 2025

Clinical trials are currently evaluating bacteriophage therapy for MDR infections, including MRSA and Pseudomonas aeruginosa. Developing Antimicrobial Peptides and Biomolecules In addition to traditional small-molecule antibiotics, researchers are exploring the potential of antimicrobial peptides (AMPs) and other biomolecules.

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Agency IQ

DECEMBER 11, 2023

So far in 2023, the Office has co-authored fifteen guidance documents—the majority of which were drafts—offering insight into the agency’s thinking on several key policy issues, such as clinical trial diversity and the accelerated approval pathway. These issues are especially apparent in treatments that are intended for chronic use.

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Codon

APRIL 2, 2023

Read Safety and pharmacokinetics of escalating doses of neutralising monoclonal antibody CAP256V2LS administered with and without VRC07-523LS in HIV-negative women in South Africa (CAPRISA 012B): a phase 1, dose-escalation, randomised controlled trial. ” Clinical trials are underway. Ricciardi M.J. Cell Systems.

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Alta Sciences

SEPTEMBER 27, 2024

Featuring two scenarios that explore the complexities of bioanalysis for immunomodulators, The Altascientist offers practical considerations for ensuring accurate bioanalysis, as well as pharmacokinetic, pharmacodynamic , and safety data in clinical trials.

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DrugBank

FEBRUARY 5, 2025

Conversely, conjugation involves chemically attaching the drug to the nanoparticle surface, allowing for targeted delivery and improved pharmacokinetics. These ligands can be antibodies, peptides, aptamers, or small molecules.

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The Pharma Data

SEPTEMBER 15, 2020

The company announced the initiation of its Phase 1b clinical trial to evaluate the safety of a novel investigational therapeutic for COVID-19, PF-07304814. Phase 1 data and development plans for danuglipron (PF-06882961), which has the potential to be the first-ever small molecule oral GLP-1RA for treating obesity and type 2 diabetes.

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Metabolite Tales Blog

JANUARY 22, 2025

Metabolism of 2024 FDA approved small molecules part 1 By Julia Shanu-Wilson Involvement of active metabolites The number of small molecules approved by the FDA in 2024 totaled 31 out of 50 NMEs with 56% (28) of these comprising small molecule therapies. Clin Pharmacokinet. 2015 May;54(5):457-71.

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