Drug Target Review

JULY 18, 2025

Whether we are working with small molecules or larger biologics, our platform enables a precise design of innovative drug candidates with highly differentiated profiles.” This knowledge will guide the strategic design and derisk its transition into Phase I/II trials to maximise success.”

Small Molecule

BioPharma Drive: Drug Pricing

JULY 8, 2025

Multiple deals have been struck by large companies such as Roche and Novartis, suggesting pharmaceutical firms believe degraders have only begun to show their promise. Specially designed small molecule drugs can bring together target proteins with an enzyme that tags them with ubiquitin, thereby marking them for destruction.

Targeted Protein Degradation

New Drug Approvals

JULY 13, 2025

(CH2O)n, NaBH3CN, MeOH, 20 °C, 16 h; f) LiOH.H2O, MeOH, 40 °C, 12 h; g) MeNH3Cl, HOBT, EDCI, TEA, DCM, RT, 16 h; h) Chiral SFC separation PATENTS WO2023016434 36% WO2021061643 US20230024905 WO2023016434 Jacobio Pharmaceuticals Co., WO2023225477 PMV Pharmaceuticals, Inc. US20230024905 PMV Pharmaceuticals, Inc. Li Sujing, et al.

Pharmaceuticals

The Pharma Data

JUNE 24, 2025

Bayer and Tsinghua University Deepen Strategic Alliance to Advance Pharmaceutical Innovation in China Bayer and Tsinghua University (THU), two powerhouses in global pharmaceutical innovation and academic research, have announced a renewed commitment to their long-standing partnership.

Pharma R&D

New Drug Approvals

JULY 2, 2025

Orforglipron ( LY-3502970 ) is an oral, non-peptide, small-molecule GLP-1 receptor agonist developed as a weight loss drug by Eli Lilly and Company. [1] 1] It was discovered by Chugai Pharmaceutical Co. 1] It was discovered by Chugai Pharmaceutical Co. 1] It was discovered by Chugai Pharmaceutical Co.

Small Molecule

New Drug Approvals

APRIL 2, 2025

1] [2] It is a non- opioid , small-molecule analgesic that works as a selective inhibitor of Na v 1.8 1] [2] It was developed by Vertex Pharmaceuticals , [5] and was approved for medical use in the United States in January 2025. [2] The FDA granted approval of Journavx to Vertex Pharmaceuticals Incorporated.

FDA

The Pharma Data

JUNE 9, 2025

Pimicotinib: A Promising CSF-1R Inhibitor Pimicotinib is an investigational small molecule developed by Abbisko Therapeutics Co., In this context, the potential approval of pimicotinib could address a large and unmet medical need , especially in China where treatment options remain limited.

Treatment

New Drug Approvals

JULY 10, 2025

Retrieved 9 July 2025. ^ “KalVista Pharmaceuticals Announces FDA Approval of Ekterly (sebetralstat), First and Only Oral On-demand Treatment for Hereditary Angioedema” (Press release). “International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 87” WHO Drug Information.

FDA

New Drug Approvals

APRIL 18, 2025

potassium hydroxide), and water to prepare 4-(2-chloro-4-methoxy-5-methylphenyl)-N-[(1S)-2-cyclopropyl-1-(3-fluoro-4-methylphenyl)ethyl]-5-methyl-N-prop-2-ynyl-1,3-thiazol-2-amine (Compound 1) or a pharmaceutically acceptable salt thereof is provided below in Scheme VII. Br), in the presence of an alkylating-step solvent (e.g.,

FDA

New Drug Approvals

JULY 6, 2025

It is a small molecule delivered orally. [1] mmol) was added tert-butyl piperidin-4-ylcarbamate (841 mg, 98% Wt, 1.2 mmol) and DIPEA (1.19 mL, 3 Eq, 10.3

Small Molecule

PPD

APRIL 14, 2025

Medical researchers and practitioners see in their own patients how these ailments interconnect and overlap, as do the pharmaceutical and biotech companies working to develop GLP-1 therapeutics. This expansion is creating opportunities for clinical trials related to a range of new therapy areas and their subpopulations.

Clinical Trials

BioPharma Drive: Drug Pricing

JULY 14, 2025

PCI helps biopharmaceutical companies manufacture and package drug products used in clinical trials as well as commercially. into a “ center of excellence ” that will produce a wide range of small molecules and biologic drugs, including protein-degrading “PROTACs” and antibody-drug conjugates. You can unsubscribe at anytime.

Production

Codon

JULY 17, 2025

Perkins’ observation drew little attention for a decade or so, until a group of Stanford doctors dug it up and ran a clinical trial to test the effects of DNP on overweight participants. The results of this trial, and its subsequent coverage in the press, kicked off a surge in demand for DNP.

Drugs

Drug Hunter

JUNE 11, 2023

Small molecule drugs make up most of the drugs we take conveniently as pills, including painkillers like ibuprofen (Advil), antibiotics like penicillin and amoxicillin, or cholesterol-lowering drugs like atorvastatin (Lipitor). The small molecules drugs of today look nothing like the molecules of the 1970s.

Small Molecule

New Drug Approvals

JULY 21, 2025

49 Akilo-scale, chromatography-freesynthesisofhetrombopag has been reported by researchers at Jiangsu Hengrui Pharmaceutical in the Chinese-language patent literature (Scheme 12).50,51 A multicenter, randomized phase III trial of hetrombopag: a novel thrombopoietin receptor agonist for the treatment of immune thrombocytopenia.

Small Molecule

New Drug Approvals

JULY 28, 2025

2] Dorzagliatin is under investigation in clinical trial NCT03173391 (Long-term Efficacy and Safety of HMS5552 in T2DM). 133 This first-in-class, small molecule,oral,glucokinaseactivator(GKA)wasfirst approved in ChinainSeptember2022foradultpatientswithT2DMasa monotherapy and in combination with metformin (an antidiabetic medication).134

Small Molecule

Drug Target Review

JULY 4, 2023

These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. 2 However, when dosed at the MTD, ADCs display improved efficacy over small molecules in oncology trials. 3D rendering of Antibody Drug Conjugate Molecules.

Small Molecule

Broad Institute

OCTOBER 4, 2023

By Allessandra DiCorato October 4, 2023 Credit: AbbVie The new small molecule inhibitor (green) sits inside the PTPN2 protein, where acidic sites are marked in red and basic sites are marked in blue. A new small-molecule drug candidate being tested in an early-stage clinical trial aims to improve patient responses to immunotherapy.

Small Molecule

Drug Target Review

JULY 6, 2023

1 TPD is expected to challenge undruggable proteins, which are highly difficult to target by conventional small molecules. 1 The concept of TPD was first demonstrated with a heterobifunctional small molecule to degrader a protein of interest in the early 2000s.

Targeted Protein Degradation

The Pharma Data

JANUARY 19, 2021

The Phase 1 clinical trial is planned to be conducted in Canada and targeted to recruit up to 48 and 24 healthy volunteers for the single-ascending dose (SAD) and multiple- ascending dose (MAD) cohorts, respectively. About ALS-4. About Aptorum Group.

Small Molecule

DrugBank

OCTOBER 17, 2024

By harnessing the vast amounts of data generated throughout the development pipeline, pharmaceutical companies can accelerate the discovery of novel therapies, optimize clinical trial design, enhance drug safety monitoring, and deliver personalized medicine, ultimately improving patient outcomes and transforming the future of healthcare.

Drug Development

Drug Target Review

OCTOBER 11, 2023

Symptoms were reversed in mouse models and a clinical trial is planned for later this year. 5 This drug candidate is progressing towards the clinic, with phase 1 trials due to start within 18 months. Autophagy boosters, in contrast, are conventional small molecule drugs. References England Rare Diseases Action Plan 2022.

Disease

Broad Institute

NOVEMBER 2, 2023

Our joint goal is to bring innovation to cancer patients by building a robust, sustainable pipeline in oncology,” said Dominik Ruettinger, Global Head of Research and Early Development for Oncology, Pharmaceuticals Division, Bayer AG.

Therapies

The Pharma Data

NOVEMBER 30, 2020

Biohaven acquires exclusive global rights to a portfolio of novel, small-molecule CGRP antagonists. The lead candidate, HTL0022562, has advanced through preclinical development demonstrating promising and differentiated properties for further investigation in human trials.

Small Molecule

Conversations in Drug Development Trends

APRIL 4, 2024

Developing pharmaceuticals is a complex process, requiring stringent bioanalytical method validation guidelines to ensure drug safety and efficacy. To help integrate these various regulatory standards, the International Council for Harmonization (ICH) of Technical Requirements for Pharmaceuticals for Human Use was founded in 2016.

Small Molecule

The Pharma Data

NOVEMBER 19, 2020

The Company is also aiming to leverage the highly conserved structure of the SARS-CoV-2 M pro protease as a basis for the design of novel oral small molecules against predicted future variants of SARS-CoV-2 and other related human viruses.

Small Molecule

Drug Target Review

NOVEMBER 1, 2023

The mission of the CCDD is to discover novel small-molecule therapeutics for the treatment of cancer and progress them to hypothesis testing phase 1 clinical trials. We have several exciting projects in various stages of discovery, but a small-molecule drug that is causing a lot of excitement at the moment is Capivasertib.

Fragment Screening

Drug Target Review

AUGUST 25, 2023

However, in tissues where the 3 Hs are not present, the small molecule has anti-toxicity activity through 1) direct inhibition of the NLRP3 inflammasome, an inflammation starter and perpetuator, and 2) upregulation of the master antioxidant transcription factor, Nrf2. References Oronsky B, Scribner C, Aggarwal R, Cabrales P.

Small Molecule

The Pharma Data

OCTOBER 15, 2020

Vertex Pharmaceuticals has decided to give up on its experimental VX-814, a small molecule drug for the rare genetic disease Alpha-1 antitrypsin deficiency (AATD), canning the drug’s development after seeing lackluster results from an early phase 2 trial.

Disease

The Pharma Data

DECEMBER 5, 2020

–(BUSINESS WIRE) December 5, 2020 — Syros Pharmaceuticals (NASDAQ:SYRS), a leader in the development of medicines that control the expression of genes, today announced new clinical data from its Phase 2 trial evaluating SY-1425, its first-in-class selective retinoic acid receptor alpha (RAR?) CAMBRIDGE, Mass.–(BUSINESS

Clinical Trials

The Premier Consulting Blog

NOVEMBER 20, 2024

Process validation is a critical regulatory requirement in pharmaceutical manufacturing. In the case of urgent or immediate public interest, process validation may be conducted concurrently with manufacturing the commercial small molecule or biologic product to expedite product availability for patients.

Small Molecule

Drug Target Review

MARCH 28, 2024

Between 2000 and 2020, approximately 30 percent of the newly introduced small molecule drugs were derived from natural products. In short, this hypothesis underscores the potential benefits of aligning pharmaceutical development more closely with nature.

Production

The Pharma Data

OCTOBER 25, 2020

NASDAQ: PTCT), today announced the initiation of a registration-directed Phase 2/3 clinical trial to evaluate vatiquinone (PTC743) in patients with mitochondrial epilepsy, the highly morbid condition of refractory seizures in children with inherited mitochondrial disease. . SOUTH PLAINFIELD, N.J. , Peltz , Ph.D.,

Clinical Trials

The Pharma Data

OCTOBER 14, 2020

15, 2020 (GLOBE NEWSWIRE) — Eloxx Pharmaceuticals, Inc. About Eloxx Pharmaceuticals. Eloxx Pharmaceuticals, Inc. Eloxx’s lead investigational product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. SOURCE: Eloxx Pharmaceuticals, Inc.

Pharmaceuticals

The Pharma Data

SEPTEMBER 12, 2020

Food and Drug Administration to expand the enrollment of their Phase 3 pivotal COVID-19 vaccine trial to up to approximately 44,000 participants which also allows for the enrollment of new populations. The pivotal trial is event-based and there are many variables that will ultimately impact read-out timing.

Vaccine

The Pharma Data

DECEMBER 10, 2020

11, 2020 (GLOBE NEWSWIRE) — Lexicon Pharmaceuticals, Inc. Lexicon is currently enrolling patients with diabetic peripheral neuropathic pain in a Phase 2 proof-of-concept study of LX9211 and is preparing to initiate a second Phase 2 clinical trial of LX9211 in post-herpetic neuralgia. About Lexicon Pharmaceuticals.

FDA

The Pharma Data

NOVEMBER 4, 2020

–( BUSINESS WIRE )– Concert Pharmaceuticals, Inc. President and Chief Executive Officer of Concert Pharmaceuticals. Initiated CTP-543 THRIVE-AA1 Phase 3 Trial in November 2020. The trial will evaluate 8 mg and 12 mg twice-daily doses of CTP-543 compared to placebo at sites in the U.S., 5, 2020 11:00 UTC.

Pharmaceuticals