The Pharma Data

NOVEMBER 11, 2020

As an orally bioavailable small molecule with broad antiviral activity, SLV213 could be a valuable treatment to meet today’s urgent need to fight COVID-19 as well other life-threatening infectious diseases, such as Chagas disease, Ebola virus disease, and Nipah virus infection.”. About SLV213.

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The Premier Consulting Blog

JULY 31, 2024

Regulatory Pathway 505(b)(2) versus 505(b)(1) In the US, novel new small molecule drug products, including some peptides, are regulated, and approved by the FDA under the Federal Food, Drug, and Cosmetic Act (the “Federal FD&C Act”) under two key regulatory pathways: Section 505(b)(1) and Section 505(b)(2) NDAs.

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Practical Cheminformatics

JANUARY 8, 2024

The authors provided examples where GPT-4 could provide a SMILES string, IUPAC name, or descriptive text for a marketed drug. pBRICS: A Novel Fragmentation Method for Explainable PropertyPredictionof Drug-Like Small Molecules [link] Perspective: I can’t say I saw a lot of progress in explainable AI for molecules this year.

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The Premier Consulting Blog

SEPTEMBER 11, 2024

The 505(b)(2) new drug application (NDA) pathway offers a unique opportunity for small molecule developers to bring innovative products to market more efficiently by leveraging existing data they do not own or have right of reference to. Human factors. The new formulation resulted in a drug product that was very viscous.

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The Pharma Data

DECEMBER 15, 2020

Small Molecule Inhibitors. NMD670 is a first-in-class small molecule inhibitor of the muscle specific chloride ion channel, the ClC-1 ion channel. The secondary outcome involves pharmacokinetic endpoints. Oral, Small Molecules. The primary outcome includes safety and tolerability endpoints.

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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

A focus of the presentation will be on the target engagement results and pharmacokinetics from its ongoing monotherapy dose-escalation study to explore a potential optimal dose and schedule to effectively inhibit AhR.

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Alta Sciences

APRIL 17, 2024

The FDA approved the first gene therapy in 2017 for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL), and as of December 2023, there are over 30 approved cell and gene therapies on the market in the U.S.A. Several bioanalytical strategies for the quantitation of ASOs are available at Altasciences.

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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.

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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. 0)40.56081-775, volker.braun@evotec.com. [1]

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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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The Pharma Data

JANUARY 10, 2021

The company submitted its Marketing Authorisation Application (MAA) to the EMA in September 2020 and anticipates receiving MAA approval for lonapegsomatropin for use in pediatric GHD in the fourth quarter of 2021. If approved on the PDUFA date, Ascendis anticipates commercial launch in the third quarter of 2021. ?.

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The Pharma Data

NOVEMBER 4, 2020

Unrealized gain (loss) on marketable equity securities. 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). . . .

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Agency IQ

JULY 26, 2024

BY LAURA DIANGELO, MPH , RACHEL COE, MSC | JUL 23, 2024 9:54 PM CDT Biosimilarity and interchangeability: A quick recap The Biologics Price Competition and Innovation (BPCI) Act of 2009 intended to increase the number of biologic products on the market by creating two types of approvals for biosimilar products. Any CMC changes (e.g.,

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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.

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DrugBank

FEBRUARY 26, 2025

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

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The Premier Consulting Blog

APRIL 25, 2023

In the US, new small molecule drugs are developed under the requirements of Sections 505(b)(1) and 505(b)(2) of the FFDCA. Synthroid® oral tablets) were marketed as unapproved drugs for the treatment of hypothyroidism in the US. This strategy can reduce the overall study requirements for approval. [1]

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The Pharma Data

FEBRUARY 25, 2022

The study met its primary goal by demonstrating pharmacokinetic equivalence in patients who switched multiple times between treatment with the two medicines. They have the ability to help increase patient access to essential medicines and provide value to the healthcare system by driving market competition that can lower the cost of care.

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The Pharma Data

OCTOBER 14, 2020

has expanded its product portfolio to include Blueprint, a self-service, scientific visualization and analytics application for small molecule discovery. The funds will be used to bring Ori’s innovative manufacturing platform to the market. Dolmatics – U.K.-based based Dotmatics Ltd. Source link.

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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. In addition, it may also be unethical to operate a control arm in some cases, such as when there is no currently marketed treatment option for a life-threatening disease.

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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.

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Metabolite Tales Blog

NOVEMBER 13, 2024

Several marketed drugs contain this ring system, for which oxadiazole ring reductive cleavage products have been reported [3]. References [1] Pharmacokinetics, disposition, and biotransformation of the cardiac myosin inhibitor aficamten in humans. Drug Metab Pharmacokinet. Xu D, Divanji P, Griffith A, et al. 2024; 12:e70006.

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New Drug Approvals

MAY 11, 2025

2] [5] In December 2023, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Skyclarys, intended for the treatment of Friedreich’s ataxia. [3] 2] [5] It is taken by mouth. [2]

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