Drug Target Review

JUNE 11, 2025

Drug development faces significant challenges: long timelines, high costs , complex processes and low probabilities of success (PoS), exacerbated by the shift towards more complex molecules, biologics and cell and gene therapies, hindering patient access to vital treatments.

Drugs

BioPharma Drive: Drug Pricing

JULY 8, 2025

Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. Drugmakers are working to coopt this process with targeted therapies. You can unsubscribe at anytime.

Targeted Protein Degradation

The Pharma Data

JUNE 9, 2025

The acceptance, which follows the CDE’s grant of Priority Review status in May, represents an accelerated regulatory pathway for what may become the first approved systemic therapy for TGCT in China. Pimicotinib: A Promising CSF-1R Inhibitor Pimicotinib is an investigational small molecule developed by Abbisko Therapeutics Co.,

Treatment

Codon

JULY 28, 2025

The gene therapy is designed to protect this infant from the neurological disorders that otherwise would have resulted from this rare, and often fatal, condition. And while matching a gene therapy to its targeted mutation is an intricate and nuanced process, much boils down to two core elements: a vector and its cargo.

Immune Response

New Drug Approvals

JUNE 25, 2025

While this therapy can decrease blood glucose level, it has limited efficacy and tolerability, causes weight gain and often induces hypoglycemia. (B) WO 2010103437 [link] Scheme I outlines the general procedures one could use to provide compounds of the present invention having Formula (I). B) Biguanides (e.g., PATENT Pfizer Inc.

Small Molecule

Drug Target Review

NOVEMBER 7, 2024

In these conditions, the goal of therapy is typically to suppress or mitigate immune activity. Understanding the intricate role of PSGL-1 in immune-cell recruitment and activation is essential for developing targeted therapies aimed at modulating immune responses in various inflammatory and autoimmune disorders.

Disease

Alta Sciences

APRIL 24, 2025

Case Study: How Altasciences Overcame Pharmacological Challenges in a GLP-1 IND-Enabling Study pmjackson Thu, 04/24/2025 - 21:43 For emerging therapies with pronounced pharmacological effects, thoughtful study design is critical to ensuring reliable and interpretable data. Download a copy of this GLP-1 RA case study!

Small Molecule

New Drug Approvals

APRIL 18, 2025

2] The US Food and Drug Administration (FDA) granted the application for crinecerfont fast track , breakthrough therapy , orphan drug , and priority review designations. [2] New Drug Therapy Approvals 2024 (PDF). 1,1-carbonyldiimidazole), a coupling-step base (e.g., triethylamine), and a coupling-step solvent (e.g., 1 December 2024.

FDA

Drug Patent Watch

JANUARY 13, 2025

Oncology: Expanding Access to Life-Saving Treatments In the field of oncology, biosimilars have made significant inroads, particularly in supportive care and targeted therapies. We’re now able to offer biologic therapies to patients earlier in their disease course, potentially altering the long-term trajectory of their condition.

Biosimilars

DrugBaron

JULY 19, 2022

Despite the current hype around so called “advanced therapies”, which range from gene editing to cell therapies, and the inexorable advance of biologic therapeutics such as monoclonal antibodies, even in 2022 the majority of drugs in development and reaching patients are still small organic molecules.

Small Molecule

ASPET

OCTOBER 12, 2023

The self-assembled structures can be small molecules, polymers, peptides, proteins, which can be utilized and functionalized to achieve tailored release and target specific cells, tissues, or organs. Different SNs approaches and recent literature reviews on peptide delivery are also presented to the readers.

Therapies

DrugBank

JULY 24, 2024

These multifunctional small molecules are like tiny spies, hijacking the body’s natural protein degradation system to remove unwanted proteins. For example, PROTACs targeting STAT3, a protein crucial for tumor growth, have shown promise where conventional therapies have failed.

Small Molecule

Drug Target Review

SEPTEMBER 25, 2024

Most targeted cancer therapies used today operate by inhibiting targets along well-known oncogenic signalling cascades. The reactivation of oncogenic signalling upstream or downstream of the driving oncogene is a well-studied source of resistance to targeted cancer therapies.

Small Molecule

Drug Target Review

JUNE 15, 2023

In this article Drug Target Review’s Izzy Wood spoke to Sam Hasson, Director of Target Biology at Rgenta Therapeutics, a biotech firm in Massachusetts, US, that aims to develop small molecule therapeutics to target RNA processing. It is somewhat like how PROTACs have changed the perspective on what is a tractable therapeutic target.

RNA

The Pharma Data

JANUARY 5, 2021

(Nasdaq: RLAY) is scheduled to present at the 39th Annual J.P. The presentation will be webcast live and may be accessed from the Investor page of Relay’s website under Events and Presentations. An archived replay of the webcast will be available for at least 30 days following the presentation. About Relay Therapeutics.

Small Molecule

The Pharma Data

NOVEMBER 18, 2020

Presentations to highlight the OBI-833 Phase 1 clinical study results in non-small cell lung cancer (NSCLC) and the dose escalation cohort. These results will be presented by the lead investigators of OBI Pharma’s novel anti-Globo H therapeutic cancer vaccine, OBI-833. ” Presentation number: 397P / Poster: ID 680.

Vaccine

The Pharma Data

SEPTEMBER 2, 2020

From the success of our first-in-class B-cell MS therapy OCREVUS, we are poised to continue advancing the science in MS with our new investigational BTK inhibitor fenebrutinib, and in NMOSD with the recent FDA approval of ENSPRYNG.”. A full list of Roche presentations can be found at: [link]. Abstract Title.

Clinical Trials

Broad Institute

JUNE 30, 2023

The researchers also looked at the tool’s ability to detect changes in cellular phenotypes after exposure to drugs and small molecule compounds. Typically, studying drug therapies is an expensive and time-consuming undertaking.

Small Molecule

The Pharma Data

APRIL 11, 2023

Bayer is advancing its oncology R&D efforts in three scientific areas that have the potential to address unmet needs in cancer patients: next-generation Immuno-Oncology, Targeted Radionuclide Therapies and Precision Molecular Oncology. Data from all three areas of scientific focus will be showcased during this year’s meeting.

Research

The Pharma Data

OCTOBER 26, 2020

Nasdaq: ARDS), a biopharmaceutical company focused on the discovery and development of novel anti-infective therapies to treat life-threatening infections, today announced the Company will present at the ROTH Capital Partners 2020 MedTech Innovation Forum on Wednesday, October 28, 2020. SAN JOSE, Calif. ,

Pharmaceuticals

Creative Biolabs

JUNE 29, 2023

At present, a large number of G protein-coupled receptor-targeted drugs have been marketed, and a large number of drugs are under development. While the development of small molecule drugs targeting GPCRs, the development of monoclonal antibody drugs has also received great attention.

Therapies

The Pharma Data

APRIL 6, 2021

Bayer will present new research across its oncology portfolio at the virtual American Association for Cancer Research (AACR) Annual Meeting 2021 , taking place over two weeks on April 10-15 and May 17-21, 2021. The compound has also received a breakthrough therapy designation in the U.S. Israel and Taiwan under the brand name Aliqopa.

Research

The Pharma Data

DECEMBER 5, 2020

The data is being presented today in oral presentations at the 62nd American Society of Hematology (ASH) Annual Meeting. The new data presented today strengthen our conviction that SY-1425 has the potential to become the foundation of care for all RARA-positive patients,” said David A.

Clinical Trials

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.”. “As About SLV213.

Treatment

The Pharma Data

JANUARY 10, 2021

IDE397 program update to be presented at the 39th Annual J.P. “The IDE397 IND submission is an important milestone for IDEAYA as we advance our broader synthetic lethality pipeline of potential first-in-class therapies. IDEAYA’s presentation for the 39 th Annual J.P. SOUTH SAN FRANCISCO, Calif. ,

FDA

The Pharma Data

JANUARY 13, 2021

Knopp expects to present results of the trial at a forthcoming medical meeting. Knopp’s clinical-stage oral small molecule, dexpramipexole, is in Phase 2 clinical trials in moderate-to-severe eosinophilic asthma. The trial was conducted at 28 U.S. study centers. Please visit www.knoppbio.com. ABOUT ORAL DEXPRAMIPEXOLE.

Trials

The Pharma Data

OCTOBER 27, 2021

Small molecule candidate Anle138b targets disease modification for multiple system atrophy and other neurological disorders. With the introduction of small-molecule medication, we open a new chapter in the fight against neurodegenerative diseases and have the chance to improve the lives of millions of patients drastically.”.

Licensing

LifeSciVC

APRIL 2, 2024

The science presents an opportunity and a challenge for us at Sionna. We are confident that there is room for improvement over existing cystic fibrosis (CF) therapies because we have not yet achieved fully normalized function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein.

Science

The Pharma Data

JANUARY 31, 2021

Convergent has exclusive rights to technology related to next generation radiopharmaceutical therapy for prostate cancer that is covered by multiple issued U.S. The key component of Convergent’s PRRT prostate cancer therapy is its proprietary drug, CONV 01-?, and foreign patents.

Small Molecule

The Pharma Data

MAY 16, 2022

AbbVie (NYSE: ABBV) today announced that 27 abstracts across its gastroenterology portfolio will be presented at the Digestive Disease Week (DDW) Annual Meeting, May 21-24, 2022, in San Diego and virtually. “We are proud to present data on a range of digestive conditions across our portfolio and pipeline. .

Disease

Drug Target Review

JULY 12, 2023

We also leverage existing clinical gene expression data from different subsets of molecularly profiled cancer patients, who differ in a certain aspect of disease or response to a particular therapy. With this drop off, the goal is to reach a near-zero drug trough before the 24-hour mark.

Biochemical Assays

The Pharma Data

MARCH 23, 2022

“These positive results demonstrate that etrasimod, if approved, could be a potential breakthrough option for patients with ulcerative colitis who aren’t able to experience improvement on current therapies,” said Michael Corbo, Chief Development Officer, Inflammation & Immunology, Pfizer Global Product Development.

Trials

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. , There is a great need for a disease-modifying treatment for the 5.3

Disease

The Pharma Data

JANUARY 10, 2021

Chiauranib as a single agent therapy inhibits the overactive Aurora B pathway existed in and related to molecular mechanism of SCLC, which is an important feature of Chiauranib distinct to other antiangiogenic drugs. At present, Chiauranib has globally applied for more than 40 patents of invention. About CHIPSCREEN BIOSCIENCES.

Clinical Trials

Broad Institute

JUNE 21, 2023

The researchers also looked at the tool’s ability to detect changes in cell morphology after exposure to drugs and small molecule compounds. Typically, studying drug therapies is an expensive and time-consuming undertaking.

Small Molecule

LifeSciVC

DECEMBER 6, 2022

Darnell’s 2002 paper Transcription Factors as Targets for Cancer Therapy , in which he wrote: “A limited list of transcription factors are overactive in most human cancer cells, which makes them targets for the development of anticancer drugs. We are rapidly advancing the chemistry required modulate its activity.

Small Molecule

LifeSciVC

MARCH 1, 2023

Importantly, they also pointed to something that we at HotSpot Therapeutics and a small group of other researchers and companies recognized several years ago – that scientific advances have made this attractive, but once considered ‘undruggable,’ target druggable.

Small Molecule