Events, Pharmacokinetics and Small Molecule

Looking beyond traditional oncogenic pathways to break cancer resistance

Drug Target Review

SEPTEMBER 25, 2024

At TOLREMO, we have developed a proprietary modular phenotypic screening platform that leverages transcriptional reprogramming events associated with non-genetic resistance to deliver new chemical scaffolds and identify novel resistance regulators. Inhibiting KDM5A, a regulator of cell proliferation, poses another interesting approach.

Small Molecule

Small Molecule Therapies Regulations Treatment

The Data-Driven Future of Drug Development

DrugBank

OCTOBER 17, 2024

Data Science in Lead Optimization and Drug Design Data science accelerates the lead optimization and drug design process, transforming how molecules are engineered for therapeutic applications. Furthermore, data-driven approaches can optimize clinical trial design, leading to smaller sample sizes and reduced overall costs.

Drug Development

Drug Development Metabolic Stability Clinical Trials Drugs

PureTech’s LYT-100 (Deupirfenidone) Demonstrates Tolerability and Pharmacokinetic Proof-of-Concept in Phase 1 Multiple Ascending Dose and Food Effect Study

The Pharma Data

NOVEMBER 17, 2020

The study demonstrated favorable proof-of-concept for LYT-100’s tolerability and pharmacokinetic (PK) profile, which will also enable twice-a-day (BID) dosing of LYT-100 in future studies. All adverse events (AEs) that were possibly or probably related to LYT-100 were mild and transient and there were no discontinuations. 1 Cottin, V.,

Pharmacokinetics

Pharmacokinetics Treatment Trials Production

Deconstructing the Diligence Process: An Approach to Vetting New Product Theses

LifeSciVC

APRIL 24, 2024

If adverse events are anticipated, it is important to understand gene dosage for such an effect (e.g., and whether a molecule’s pharmacology can help to mitigate safety risk. and whether a molecule’s pharmacology can help to mitigate safety risk. loss of function” (LoF) effect, but can also be quite nuanced (e.g.,

Production

Production Small Molecule Regulations Trials

Game-changing pan-TEAD inhibitor for solid tumours

Drug Target Review

JUNE 12, 2023

The current version of Chemistry42 uses over 40 generative models, including generative autoencoders and generative adversarial networks as well as both structure-based and ligand-based drug design to generate and optimise de novo small molecules.

Metabolic Stability

Metabolic Stability Clinical Trials Small Molecule Pharmacokinetics

ATUZAGINSTAT

New Drug Approvals

SEPTEMBER 29, 2024

link] 01 Aug 2022 Cortexyme is now called Quince Therapeutics You need to be a logged in or subscribed to view this content This small molecule is an orally available protease inhibitor targeting the lysine proteases of the periodontal pathogen Porphyromonas gingivalis. All volunteers with AD had P. 2019 Jan;5(1):eaau3333.

Small Molecule

Small Molecule DNA Disease Trials

Positive Phase 1 results in high-dose setanaxib trial

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

Trials Clinical Trials Pharmacokinetics Small Molecule

Aptose Initiates Dosing of CG-806 in Patients with Acute Myeloid LeukemiaPhase 1 a/b Study of CG-806 in AML Initiates with Starting Dose of 450mg

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. . .

Small Molecule

Small Molecule Clinical Trials Trials Therapies

Novartis announces positive results from Phase II study of LNP023 in patients with paroxysmal nocturnal hemoglobinuria (PNH)

The Pharma Data

AUGUST 29, 2020

LNP023 also demonstrated a favorable safety and tolerability profile with no serious treatment-related infections or thromboembolic events. Following the cut-off date for the presented data, one participant, who had severe lymphopenia at study entry, discontinued treatment due to a serious adverse event (AE) of lymphoproliferative disorder.

Treatment

Treatment Disease Small Molecule Production

Analysis Life Sciences Thank You FDA finalizes new guidance on nonclinical assessment of potential immunotoxicity

Agency IQ

JUNE 9, 2023

However, this guideline is primarily intended to provide recommendations on evaluating the immune system’s response to small molecule drugs. The guidance contains recommendations for small molecule drugs, as well as any biological products that fall within CDER’s purview.

FDA

FDA Small Molecule Science Immune Response

Evotec Expands Oxfordshire Site into a Fully-Integrated R&D Centre Named ‘Dorothy Crowfoot Hodgkin Campus’

The Pharma Data

NOVEMBER 11, 2020

Adding a new building, which will house up to 100 biologists by the end of the year, will bring together all the key functions for cutting-edge and high-performance small molecule discovery and up to commercial development. 0)40.56081-255, gabriele.hansen@evotec.com.

Small Molecule

Small Molecule Science Pharmacokinetics Disease

Ascendis Pharma A/S Provides Vision 3×3 Update at 39th Annual J.P. Morgan Healthcare Conference

The Pharma Data

JANUARY 10, 2021

Independently optimized receptor bias and potency as well as pharmacokinetics to create a potentially best-in-class IL-2 product. Morgan presentation will be available on the Events & Presentations section of the investor relations webpage at [link]. . . . . .

Clinical Trials

Clinical Trials Clinical Development Small Molecule Trials

Codon Digest: Injected Gene Editors

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. Read Small-molecule aptamer for regulating RNA functions in mammalian cells and animals.

DNA

DNA RNA Engineering Licensing

Pfizer Investor Day Features Significant Number of Pipeline Advances for COVID-19 Programs and Across Numerous Therapeutic Areas

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

Vaccine Therapies Trials Clinical Trials

Analysis Life Sciences Thank You Workshop addresses oncology dose optimization across full span of development

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. So if you have a small number of patients, and as you mentioned, you’ve collected patient reported outcomes, that can just add in.

Science

Science FDA Trials Clinical Pharmacology

Aptorum Group Announces Submission of Clinical Trial Application for ALS-4, an Orally Administered Small Molecule Drug for the Treatment of Infections caused by Staphylococcus aureus including Methicillin-resistant Staphylococcus aureus (MRSA)

The Pharma Data

DECEMBER 20, 2020

Pending Health Canada’s approval, the Phase 1 trial is designed to test the safety, tolerability and pharmacokinetics of ALS-4 in healthy volunteers. ALS-4 is a novel small molecule adopting an anti-virulence (non-antibiotic) approach to address the growing unmet medical needs of infections caused by Staphylococcus aureus.

Small Molecule

Small Molecule Clinical Trials Trials Treatment

Concert Pharmaceuticals Reports Third Quarter 2020 Financial Results and Provides Company Update

The Pharma Data

NOVEMBER 4, 2020

The 8 mg and 12 mg twice-daily doses continued to be generally well tolerated and adverse events were consistent with those reported in the Phase 2 trials. Published Findings on Alopecia Areata Burden of Disease in Journal of Investigative Dermatology.

Pharmaceuticals

Pharmaceuticals Clinical Trials Trials Treatment

Aptorum Group Receives Clearance from Health Canada to Initiate a Phase 1 Clinical Trial for ALS-4, a Small Molecule Drug for Infections Caused by Staphylococcus aureus including Methicillin-resistant Staphylococcus aureus (MRSA)

The Pharma Data

JANUARY 19, 2021

The secondary objective is to assess the pharmacokinetic profile of SAD and MAD of ALS-4 administered orally to healthy subjects. Aptorum Group assumes no obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise. About ALS-4.

Small Molecule

Small Molecule Clinical Trials Trials Pharmacokinetics

Drug Discovery Digest

Looking beyond traditional oncogenic pathways to break cancer resistance

The Data-Driven Future of Drug Development

Trending Sources

PureTech’s LYT-100 (Deupirfenidone) Demonstrates Tolerability and Pharmacokinetic Proof-of-Concept in Phase 1 Multiple Ascending Dose and Food Effect Study

Deconstructing the Diligence Process: An Approach to Vetting New Product Theses

Game-changing pan-TEAD inhibitor for solid tumours

ATUZAGINSTAT

Positive Phase 1 results in high-dose setanaxib trial

Aptose Initiates Dosing of CG-806 in Patients with Acute Myeloid LeukemiaPhase 1 a/b Study of CG-806 in AML Initiates with Starting Dose of 450mg

Novartis announces positive results from Phase II study of LNP023 in patients with paroxysmal nocturnal hemoglobinuria (PNH)

Analysis Life Sciences Thank You FDA finalizes new guidance on nonclinical assessment of potential immunotoxicity

Evotec Expands Oxfordshire Site into a Fully-Integrated R&D Centre Named ‘Dorothy Crowfoot Hodgkin Campus’

Ascendis Pharma A/S Provides Vision 3×3 Update at 39th Annual J.P. Morgan Healthcare Conference

Codon Digest: Injected Gene Editors

Pfizer Investor Day Features Significant Number of Pipeline Advances for COVID-19 Programs and Across Numerous Therapeutic Areas

Analysis Life Sciences Thank You Workshop addresses oncology dose optimization across full span of development

Aptorum Group Announces Submission of Clinical Trial Application for ALS-4, an Orally Administered Small Molecule Drug for the Treatment of Infections caused by Staphylococcus aureus including Methicillin-resistant Staphylococcus aureus (MRSA)

Concert Pharmaceuticals Reports Third Quarter 2020 Financial Results and Provides Company Update

Aptorum Group Receives Clearance from Health Canada to Initiate a Phase 1 Clinical Trial for ALS-4, a Small Molecule Drug for Infections Caused by Staphylococcus aureus including Methicillin-resistant Staphylococcus aureus (MRSA)

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