2024, Clinical Development and Small Molecule

Sentiment & Themes Emerging From JPM 2024

LifeSciVC

JANUARY 16, 2024

The market for each target class likely asymptotes with the number of Pharma or large biotech who can clinically develop and commercialize such assets; thus, there is intense focus on the first handful of assets to market. Small molecule GLP1s? ALT) continue to build momentum in the indication.

Small Molecule

Small Molecule Trials Therapies Disease

Redefining Acceleration of the Drug Development Journey

PPD

NOVEMBER 11, 2024

According to the United Nation’s 2024 World Population Prospects data, the global population of those 65 years old and over will grow from 0.8 From an aging population increasing health care demand to urgent, unmet medical needs, there are clear drivers to advance safe and effective medicines to patients faster than ever. billion in 2035.

Drug Development

Drug Development Clinical Trials Drugs Small Molecule

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

Alta Sciences

APRIL 17, 2024

Five Promising Treatment Areas in Early-Phase Drug Development in 2024 aasimakopoulos Wed, 04/17/2024 - 15:52 Early-phase drug development is an ever-changing landscape, as emerging science leads to new promising areas of research for the treatment of human health issues. Asia, and Europe.

Drug Development

Drug Development Treatment FDA Approval Drugs

Lessons From A Private Funding Round: Science, Relationships, And Experience

LifeSciVC

APRIL 2, 2024

For years, NBD1 was considered “undruggable”, but Sionna could be on the verge of a breakthrough with four programs in or entering the clinic this year leveraging years of experience and knowledge to crack the code on NBD1. We experienced strong enthusiasm for small molecule research in the investor community.

Science

Science Small Molecule International Clinical Development

Sanofi continues on path to industry leadership in Immunology with Dupixent® (dupilumab) as key driver

The Pharma Data

MARCH 28, 2022

Dupixent peak sales ambition raised to more than €13 billion Chronic obstructive pulmonary disease 2023 pivotal readouts provide potential for additional Dupixent sales ambition upgrade 13 potential new medicines currently in the clinic to treat chronic inflammatory diseases, with 17 readouts expected by the end of 2024.

Small Molecule

Small Molecule Clinical Development Disease Science

Liquid biopsy helps advance oncology drug development

Drug Target Review

JULY 18, 2024

How is leveraging liquid biopsy leading to innovative trial designs in oncology and ultimately our ability to develop new drugs in the adjuvant setting? Nat Med 30, 531–542 (2024). This helped us to identify as many patients as possible who already had microscopic tumour spread.

Drug Development

Drug Development Clinical Trials Drugs Vaccine

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

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.,

Biosimilars

Biosimilars Science FDA Production

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. What to look for in 2024: This year, OCE amassed a significant volume of feedback from its advisory committee members and from stakeholders via the rulemaking process on trial design.

FDA

FDA Science Clinical Trials Trials

Present Treatments & Hopeful Future Directions for ALS Pharmacotherapies

Conversations in Drug Development Trends

MAY 30, 2024

Differences in regulatory sentiments and industry sponsors’ subsequent clinical development strategies historically restricted access based on geography. However, work is needed before CRISPR-mediated treatments become possible, including a better understanding of the genes involved in ALS.

Treatment

Treatment Clinical Trials Small Molecule Therapies

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

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

Using clinical genomics and AI in drug development to elevate success

Drug Target Review

FEBRUARY 11, 2025

Addressing the long timelines and high costs of drug development is therefore critical for the advancement of medical innovation. Clinical development failure may be due to an inability to demonstrate efficacy or sufficient safety. Drugs that were developed and commercialised 30+ years ago were relatively simple small molecules.

Drug Development

Drug Development Bioinformatics Drugs Small Molecule

Major biotransformation routes of aficamten

Metabolite Tales Blog

NOVEMBER 13, 2024

Aficamten (CK-3773274) is a cardiac myosin inhibitor currently in phase 3 clinical development to treat this inherited disorder. 2024; 12:e70006. link] [8] In vitro and in vivo preclinical pharmacokinetic characterization of aficamten, a small molecule cardiac myosin inhibitor. Xu D, Divanji P, Griffith A, et al.

Pharmacokinetics

Pharmacokinetics Small Molecule Production Drugs

A Tale of Two Sentiments: JPM 2025

LifeSciVC

JANUARY 21, 2025

In contrast, the mood is more apprehensive for those companies with data or timing setbacks, especially on top of one of the highest rates of RIFs in 2024. Stifel recently reported that ~1/3 of Pharma licensing deals in 2024 were sourced from China, an incredible statistic and one that points to the shifting landscape for asset-centric deals.

Trials

Trials Small Molecule Marketing Disease

Drug Discovery Digest

Sentiment & Themes Emerging From JPM 2024

Redefining Acceleration of the Drug Development Journey

Trending Sources

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

Lessons From A Private Funding Round: Science, Relationships, And Experience

Sanofi continues on path to industry leadership in Immunology with Dupixent® (dupilumab) as key driver

Liquid biopsy helps advance oncology drug development

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

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

Present Treatments & Hopeful Future Directions for ALS Pharmacotherapies

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

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

Using clinical genomics and AI in drug development to elevate success

Major biotransformation routes of aficamten

A Tale of Two Sentiments: JPM 2025

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