Clinical Development, Pharmacokinetics and Small Molecule

Early trial data supports neurodegenerative disease-modifying drug

Drug Discovery World

MARCH 15, 2024

Biotechnology company Allyx Therapeutics has reported positive Phase Ib clinical data for its lead compound, ALX-001, a first-in-class, synapse-targeted, disease-modifying oral therapy in development for neurodegenerative diseases. The findings were presented at the AD/PD 2024 Conference in Lisbon.

Disease

Disease Trials Small Molecule Pharmacokinetics

Immunotherapy for solid tumours progresses to human trials

Drug Discovery World

AUGUST 14, 2023

Research partners in the UK are progressing their orally available small molecule cancer immunotherapy drug into clinical development as a treatment for advanced solid tumours.

Trials

Trials Small Molecule Pharmacokinetics Clinical Trials

Positive Phase I results for first-in-kind small molecule complement inhibitor

Drug Discovery World

JANUARY 4, 2024

ANX1502 is a first-in-kind oral, selective small molecule inhibitor that targets the active form of C1s responsible for propagating classical pathway activation in association with C1q. No serious adverse events were reported, and there were no significant clinical or lab findings.

Small Molecule

Small Molecule Pharmacokinetics Clinical Development Disease

First patients dosed in Phase 1a clinical trial of UCL-backed G-Quadruplex (G4) targeting treatment for solid tumors, including pancreatic cancer

Sygnature Discovery

NOVEMBER 29, 2023

It was conceived, invented and developed by Professor Stephen Neidle and his team in the School of Pharmacy at University College London with the involvement of Sygnature Discovery scientists. The Phase 1a clinical trial involves a small group of patients who will be given carefully monitored doses of the drug.

Clinical Trials

Clinical Trials Treatment Trials Small Molecule

Metabolism of 2023 FDA Approved Small Molecules – PART 1

Metabolite Tales Blog

JANUARY 23, 2024

Metabolism of 2023 FDA Approved Small Molecules – PART 1 By Julia Shanu-Wilson 2023 was a fruitful year for drug approvals by the FDA, with a crop of 34 small molecules out of a total of 55 new drugs [1]. References [1] 2023 Novel Small Molecule FDA Drug Approvals. link] [9] Patent EP1242061B1.

Small Molecule

Small Molecule FDA Approval FDA Pharmacokinetics

First patients dosed in Phase 1a clinical trial of UCL-backed G-Quadruplex (G4) targeting treatment for solid tumors, including pancreatic cancer

Sygnature Discovery

NOVEMBER 29, 2023

It was conceived, invented and developed by Professor Stephen Neidle and his team in the School of Pharmacy at University College London with the involvement of Sygnature Discovery scientists. The Phase 1a clinical trial involves a small group of patients who will be given carefully monitored doses of the drug.

Clinical Trials

Clinical Trials Treatment Trials Small Molecule

Biopharma Money on the Move: October 21-27

The Pharma Data

OCTOBER 27, 2020

Sirnaomics is the only biopharma conducting R&D and clinical development in the field of RNAi therapeutics in both the U.S. Primmune aims to advance the development of their novel orally-administered, small molecule toll-like receptor 7 agonists as therapeutic-adjuvants for acute viral diseases and cancer.

Small Molecule

Small Molecule Immune Response Pharmacokinetics Clinical Development

Bayer to present latest research across its advancing oncology portfolio at AACR 2023 Annual Meeting

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. The company has the passion and determination to develop innovative medicines that help improve and extend the lives of people living with cancer.

Research

Research Clinical Trials Small Molecule Pharmacokinetics

Metabolism of 2022 FDA approved small molecule drugs PART 2

Metabolite Tales Blog

APRIL 4, 2023

Metabolism of 2022 FDA approved small molecule drugs part 2 Mixing it Up By Julia Shanu-Wilson In Part 1 of this topic we looked at metabolism of the small molecule drugs approved by the FDA in 2022 that were mediated by CYP3A4. We hope it was a useful two-parter! We’re looking forward to the next crop! Acta Pharm Sin B.

Small Molecule

Small Molecule FDA Approval FDA Drugs

Drug-Fc conjugates exhibit robust anti-tumour activity

Drug Discovery World

DECEMBER 13, 2023

It demonstrated a differentiated in vitro activity profile across several assays compared with small molecule and antibody inhibitors of CD73 in clinical development. The company also presented data for CBO421, an AMP-competitive inhibitor of CD73.

Small Molecule

Small Molecule Biosimilars Pharmacokinetics Drugs

Informatics helps virtual biotech bring compound to clinical trial

Drug Discovery World

FEBRUARY 22, 2024

Libra Therapeutics is a San Diego-based biotech focused on developing novel disease-modifying therapeutics, which restore the cellular balance that is disrupted in neurodegenerative diseases. Libra’s pipeline targets mechanisms that can mitigate protein pathology associated with CNS degenerative disorders.

Clinical Trials

Clinical Trials Trials Small Molecule Compound Screening

AI-driven biotech initiates Phase I trial for ALS drug

Drug Discovery World

NOVEMBER 3, 2022

Verge Genomics, a clinical-stage, tech-enabled biotechnology company using artificial intelligence (AI) and human data to transform drug discovery, has announced today that the first subject has been dosed in a Phase I clinical trial of VRG50635 for amyotrophic lateral sclerosis (ALS). . Through the evaluation of more than 11.4

Trials

Trials Small Molecule Clinical Trials Pharmacokinetics

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

The Pharma Data

AUGUST 29, 2020

3,4 LNP023 is currently in clinical development for PNH and a number of renal conditions with complement system involvement where significant unmet needs exist, including IgA nephropathy, complement 3 glomerulopathy (C3G), atypical hemolytic uremic syndrome and membranous nephropathy. Blood 2019;134(Suppl 1):3517.

Treatment

Treatment Disease Small Molecule Production

Optimized 505(b)(1) and 505(b)(2) Clinical Pharmacology Programs to Accelerate Drug Development

The Premier Consulting Blog

JULY 31, 2024

In this blog, we explain the role of clinical pharmacology in drug development and demonstrate how the right strategy can accelerate development under the US Food and Drug Administration (FDA) 505(b)(1) and 505(b)(2) New Drug Application (NDA) pathways. Author: Jayesh Patel , Principal Scientist, Pharmacokinetics

Clinical Pharmacology

Clinical Pharmacology Drug Development Pharmacokinetics Drugs

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

The Pharma Data

NOVEMBER 4, 2020

Concert Pharmaceuticals is a clinical stage biopharmaceutical company that is developing small molecule drugs that it discovered through the application of its DCE Platform® (deuterated chemical entity platform).

Pharmaceuticals

Pharmaceuticals Clinical Trials Trials Small Molecule

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

LifeSciVC

APRIL 24, 2024

There is a continuum of evidence for a given target – at one end are novel targets with some evidence of importance in disease, and at the other end are “de-risked” targets where the biology is precedented with an approved product or late-stage clinical asset(s). and whether a molecule’s pharmacology can help to mitigate safety risk.

Production

Production Small Molecule Regulations Trials

Fortis Life Sciences leads the way in custom antibody discovery

Drug Discovery World

DECEMBER 13, 2023

Custom antibodies can be developed for pharmacokinetic assays and biodistribution assays, supporting therapeutic development by giving researchers data about the behaviour of their novel therapeutic in vivo. Moreover, custom antibodies are a key reagent in companion diagnostics for these novel therapeutics.

Science

Science Small Molecule Laboratories Engineering

Selva Therapeutics Announces First Dosing in Phase 1 Clinical Study of SLV213, a Potential Oral Treatment for COVID-19

The Pharma Data

NOVEMBER 11, 2020

“These data provide further evidence of efficacy and high potency of SLV213 against SARS-CoV-2 and support the clinical development of SLV213 as a potential oral treatment for COVID-19,” said Ted Daley, President and CEO, Selva Therapeutics. “As Cofounder and Chief Scientific Officer of Selva Therapeutics. About SLV213.

Treatment

Treatment Small Molecule Virus Pharmacokinetics

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

Agency IQ

JUNE 9, 2023

For this reason, animal models are still used both during drug discovery and the more intensive nonclinical studies conducted during clinical development. However, this guideline is primarily intended to provide recommendations on evaluating the immune system’s response to small molecule drugs.

FDA

FDA Science Small Molecule Immune Response

Five Promising Treatment Areas in Early-Phase Drug Development in 2024

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. Asia, and Europe.

Drug Development

Drug Development Treatment FDA Approval Drugs

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

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

The Pharma Data

JANUARY 10, 2021

“We expect 2021 will mark a number of key clinical and commercial milestones. By the end of the year, we expect to have five independent TransCon product candidates in clinical development leveraging TransCon technologies through our algorithm for product innovation. Pipeline Updates. •. .

Clinical Trials

Clinical Trials Clinical Development Small Molecule Trials

Analysis Life Sciences Thank You FDA’s new guidance on postapproval manufacturing changes for biosimilars focuses on current practice, new dosage forms

Agency IQ

JULY 26, 2024

Postapproval manufacturing changes for biological products During clinical development or following a product’s approval , sponsors and/or manufacturers may identify the need for a change in the product, production process, quality controls, equipment, facilities, responsible personnel, or labeling. Any CMC changes (e.g.,

Biosimilars

Biosimilars Science FDA Licensing

Advances in the Battle Against Autoimmune Disease

The Pharma Data

DECEMBER 15, 2020

Anokion is responsible for preclinical activities and Phase I clinical development of partnered programs, and Bristol Myers Squibb will fund subsequent trials and commercial activities at clinical proof-of-concept. Small Molecule Inhibitors. The secondary outcome involves pharmacokinetic endpoints.

Disease

Disease Clinical Trials Small Molecule Trials

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

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. In addition to the COVID-19 vaccine program, Pfizer aims to deliver five innovative vaccines by 2025, subject to clinical success and regulatory approval.

Vaccine

Vaccine Therapies Trials Clinical Trials

Analysis Life Sciences Thank You The FDA and oncology: 2023 year in review

Agency IQ

DECEMBER 11, 2023

In 2015, the FDA, along with the American Association for Cancer Research (AACR), held a workshop on dose optimization for small molecules. This proposal is an exciting example of how new approaches at the forefront of clinical pharmacology can be leveraged to inform product use in particularly vulnerable patient populations.

FDA

FDA Science Clinical Trials Trials

BioSpace Global Roundup, Oct. 15

The Pharma Data

OCTOBER 14, 2020

Priothera will use the funds to progress the clinical development of mocravimod, a modulator of sphingosine 1 phosphate (S1P) receptors, to enhance the curative potential of allogeneic hematopoietic stem cell transplantation (HSCT) for treating AML. Dolmatics – U.K.-based based Dotmatics Ltd.

Clinical Trials

Clinical Trials Trials Pharmaceuticals Pharmaceutical Companies

Drug Discovery Digest

Early trial data supports neurodegenerative disease-modifying drug

Immunotherapy for solid tumours progresses to human trials

Trending Sources

Positive Phase I results for first-in-kind small molecule complement inhibitor

First patients dosed in Phase 1a clinical trial of UCL-backed G-Quadruplex (G4) targeting treatment for solid tumors, including pancreatic cancer

Metabolism of 2023 FDA Approved Small Molecules – PART 1

First patients dosed in Phase 1a clinical trial of UCL-backed G-Quadruplex (G4) targeting treatment for solid tumors, including pancreatic cancer

Biopharma Money on the Move: October 21-27

Bayer to present latest research across its advancing oncology portfolio at AACR 2023 Annual Meeting

Metabolism of 2022 FDA approved small molecule drugs PART 2

Drug-Fc conjugates exhibit robust anti-tumour activity

Informatics helps virtual biotech bring compound to clinical trial

AI-driven biotech initiates Phase I trial for ALS drug

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

Optimized 505(b)(1) and 505(b)(2) Clinical Pharmacology Programs to Accelerate Drug Development

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

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

Fortis Life Sciences leads the way in custom antibody discovery

Selva Therapeutics Announces First Dosing in Phase 1 Clinical Study of SLV213, a Potential Oral Treatment for COVID-19

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

Five Promising Treatment Areas in Early-Phase Drug Development in 2024

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

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

Analysis Life Sciences Thank You FDA’s new guidance on postapproval manufacturing changes for biosimilars focuses on current practice, new dosage forms

Advances in the Battle Against Autoimmune Disease

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

Analysis Life Sciences Thank You The FDA and oncology: 2023 year in review

BioSpace Global Roundup, Oct. 15

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