The Pharma Data

AUGUST 1, 2025

This regulatory privilege is reserved for investigational therapies that demonstrate the potential to offer a major therapeutic advantage over existing treatments or benefit patients without treatment options. There remains a high unmet need for more effective and durable therapies, especially in the post-BTK inhibitor setting.

Targeted Protein Degradation

Drug Target Review

JULY 18, 2025

Tackling resistance from a different angle: PAR2 While CCR8 is drawing attention, Domain is also exploring less conventional GPCRs like PAR2 – long known in inflammation and pain, but only recently linked to cancer therapy resistance.

Medical Chemistry

Alta Sciences

JULY 3, 2025

Attendees were particularly drawn to our integrated solutions, which encompass everything from lead candidates advancing to IND-enabling programs to early-phase clinical trials to full-scale commercial manufacturing. Beyond the science, what’s exciting is how this technology opens doors. The response?

Small Molecule

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

Broad Institute

OCTOBER 29, 2024

This small molecule reactivates the apoptosis cascade in tumor cells while sparing healthy cells in animal models. Apoptosis, or programmed cell death, is a natural end process for all cells. This rapid clearance minimizes the drug’s potential impact on healthy cells.

Drugs

The Pharma Data

JUNE 24, 2025

Oncology remains a core priority, reflecting the rising global burden of cancer and the need for more effective, targeted therapies. The goal is to transform laboratory discoveries into clinically relevant therapies that can reach patients faster and more efficiently. Source link

Pharma R&D

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 The application received Breakthrough Therapy , Fast Track and Priority Review designations by the FDA. 11] The FDA granted the application for suzetrigine priority review , fast track , and breakthrough therapy designations. [2]

FDA

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

clinical candidate currently in Phase 2 development. Several glucokinase activators have advanced to clinical studies and demonstrated promising efficacy; however, many of these early candidates also revealed hypoglycemia as a key risk. glipizide, glimepiride, glyburide) and meglitinides (e.g., B) Biguanides (e.g., Aggarwal, N.;

Small Molecule

New Drug Approvals

JULY 7, 2025

1] Pimicotinib is under investigation in clinical trial NCT05804045 (Study of Pimicotinib (ABSK021) for Tenosynovial Giant Cell Tumor (MANEUVER)). 1] Pimicotinib is under investigation in clinical trial NCT05804045 (Study of Pimicotinib (ABSK021) for Tenosynovial Giant Cell Tumor (MANEUVER)). 6] The U.S. 6] The U.S.

Small Molecule

New Drug Approvals

APRIL 18, 2025

2] History Crinecerfont’s approval is based on two randomized, double-blind, placebo-controlled trials in 182 adults and 103 children with classic congenital adrenal hyperplasia. [2] 2] In the first trial, 122 adults received crinecerfont twice daily and 60 received placebo twice daily for 24 weeks. [2] 1 December 2024.

FDA

PPD

APRIL 14, 2025

This expansion is creating opportunities for clinical trials related to a range of new therapy areas and their subpopulations. Participant fatigue in GLP-1 clinical trials The success of GLP-1 drugs in SELECT and other trials has biotech and biopharma companies racing to find which indications respond well to similar treatment.

Clinical Trials

New Drug Approvals

JUNE 9, 2025

Clinical trials: Clinical trials have demonstrated the safety and potential efficacy of hexasodium phytate in reducing hydroxyapatite crystallization in patients undergoing hemodialysis, providing a basis for its use in treating calciphylaxis.

Clinical Trials

BioPharma Drive: Drug Pricing

JULY 14, 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. PCI helps biopharmaceutical companies manufacture and package drug products used in clinical trials as well as commercially.

Production

Drug Target Review

JULY 4, 2023

These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. Patients and caregivers also assess the benefits offered by different therapies, weighing the progression-free survival with their off-target effects.

Small Molecule

Broad Institute

NOVEMBER 2, 2023

Bayer and Broad Institute extend cancer therapy research collaboration By Corie Lok November 2, 2023 Breadcrumb Home Bayer and Broad Institute extend cancer therapy research collaboration Long-standing industry-academia collaboration has already resulted in three clinical oncology candidates.

Therapies

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

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

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

JUNE 19, 2024

The mission of Lineage Cell Therapeutics is to deliver on some of the early promises of cell therapy. Cell therapy as a concept is a wonderful idea, but many of the early efforts never generated the kind of clinical data that gets people excited and leads to new medicines. Hearing aids also have all sorts of deficits.

Therapies

Drug Target Review

OCTOBER 11, 2023

1,2 Many, however, do have dysfunctional proteins at their core, and for these diseases, a new option is being explored: autophagy-boosting therapies. Symptoms were reversed in mouse models and a clinical trial is planned for later this year. Gene therapy, of course, also holds great promise.

Disease

LifeSciVC

JANUARY 16, 2024

These are incredible forecasts and CAGRs, which if we assume directionally correct, rely not only on steady growth for approved therapies but also a substantial success rate of, and continued investment in, the development pipeline. Small molecule GLP1s?

Small Molecule

Drug Target Review

NOVEMBER 6, 2023

2 It is this complexity that necessitates powerful, targeted combination therapies. 4, 5 More recently, new combination regimens have emerged that incorporate targeted therapies to treat a variety of blood cancers, including multiple myeloma (MM), chronic lymphocytic leukaemia (CLL) and acute myelogenous leukaemia (AML).

Small Molecule

Advarra

FEBRUARY 24, 2023

How do you know your experimental therapy is working? In most cases, the results you are really looking for and trying to prove in a trial can be complex. In most cases, the results you are really looking for and trying to prove in a trial can be complex.

Clinical Trials

The Premier Consulting Blog

APRIL 18, 2024

Development of cell and gene therapies is growing rapidly, given the major advances in genomic technologies and increasing scientific understanding of genetic regulation and immunology. This blog will outline three characteristics of a successful nonclinical program to support entry into clinical trials for a cell or gene therapy product.

Therapies

The Pharma Data

JANUARY 10, 2021

SH ) submitted its pivotal phase III clinical trial application (IND) of national class I innovative drug Chiauranib to NMPA, for the treatment of Small Cell Lung Cancer (SCLC) as a single agent for the patients after 2nd-line systemic chemotherapy and recurrence afterwards. SHENZHEN, China , Jan.

Clinical Trials

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. Vlad Coric , M.D.,

Small Molecule

The Pharma Data

SEPTEMBER 8, 2020

Phase III clinical trial programme initiated for investigational medicine fenebrutinib, designed to be a highly selective and reversible Bruton’s tyrosine kinase (BTK) inhibitor, in relapsing multiple sclerosis (RMS) and primary progressive MS (PPMS).

Clinical Trials

Conversations in Drug Development Trends

APRIL 4, 2024

Stability Tests: For small molecule, only -20°C stability testing is required, while large molecule need both -20°C and -70°C tests if samples are intended for storage at these temperatures. The post Demystifying the ICH M10 Bioanalytical Method Validation Guidelines appeared first on Worldwide Clinical Trials.

Small Molecule

DrugBank

JANUARY 9, 2025

Targeted Therapies vs. Traditional Treatments Traditional cancer treatments, such as chemotherapy and radiotherapy, are inherently cytotoxic, targeting both cancerous and healthy cells. The development of imatinib to treat chronic myeloid leukemia (CML) serves as a hallmark for targeted therapies.

Therapies

Drug Target Review

SEPTEMBER 11, 2024

How does morADC technology combine the properties of small molecules and monoclonal antibodies to enhance anti-aggregation effects for CNS applications? The morADC are able to cross the blood-brain barrier more efficiently and offer higher potency than individual parent molecules.

Small Molecule

The Premier Consulting Blog

APRIL 18, 2024

Designing an adequate nonclinical program to support the safety of cell or gene therapy products is not always straightforward. The cell or gene therapy should be pharmacologically active in the animal model. Note that there is no strict requirement of toxicity studies in two species, which is often needed for small molecule programs.

Therapies

Drug Target Review

JUNE 12, 2023

With 13 preclinical candidates and three AI-designed drugs currently undergoing clinical trials, Insilico is spearheading a revolution in cancer treatment and beyond. 2021 – and a robust pipeline that includes 3 AI-designed drugs (for IPF, COVID-19 and cancer) currently in clinical trials.

Metabolic Stability

Drug Target Review

MAY 6, 2024

However, it turned out that the stunning initial clinical results – dramatic tumour shrinkage within just a couple of weeks – were unfortunately short-lived. The clinical efficacy of BRAF inhibitors, like most other targeted cancer therapies, is inevitably limited by resistance development.

Therapies

The Premier Consulting Blog

NOVEMBER 20, 2024

For drugs with FDA Orphan Drug Designation (ODD), Breakthrough Therapy Designation (BTD), and EMA PRIority MEdicines designation (PRIME), all clinical, non-clinical, and CMC requirements are still required for regulatory approval, despite being on pathways for an expedited approval process.

Small Molecule

LifeSciVC

APRIL 24, 2024

In the absence of a clinical trial result or FDA label to point to, how does one create the case and target product profile (TPP) around a new target? and whether a molecule’s pharmacology can help to mitigate safety risk. with gene editing or gene therapy, enzyme replacement therapy), agonism (e.g.,

Production

Agency IQ

APRIL 19, 2024

Meet C3TI, FDA’s new clinical trial innovation hub On April 14, FDA announced that its Center for Drug Evaluation and Research (CDER) is launching a new clinical trial innovation hub, the CDER Center for Clinical Trial Innovation (C3TI). In particular, regulations in both the U.S.

Clinical Trials