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Early trial data supports neurodegenerative disease-modifying drug

Drug Discovery World

Biotechnology company Allyx Therapeutics has reported positive Phase Ib clinical data for its lead compound, ALX-001, a first-in-class, synapse-targeted, disease-modifying oral therapy in development for neurodegenerative diseases. All doses were well-tolerated and there were no serious adverse events.

Disease 130
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Sosei Heptares and Biohaven Enter Global Collaboration and License Agreement to Advance Novel Small-Molecule CGRP Antagonist Portfolio

The Pharma Data

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.,

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Sentiment & Themes Emerging From JPM 2024

LifeSciVC

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?

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Comment: LSX World Congress 2023 

Drug Discovery World

DDW’s Megan Thomas comments on the event. New approaches to partnering In this event in the Biotech track, Clive Cookson, Science Editor at the Financial Times, interviewed John McDonald, Corporate Vice President, Global R&D Business Development, Novo Nordisk.

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Multiple Dynamic Endpoints and the Role of Endpoint Adjudication Committees in Clinical Trials

Advarra

How do you know your experimental therapy is working? But what does “working” really mean when efficacy means something subjective, like quality of life, reduced psychiatric events, or less symptoms? All key questions to consider and address before the therapy even leaves the lab.

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Where is the drug discovery expertise happening in the UK?

Drug Discovery World

A company within this ecosystem is STORM Therapeutics, a clinical stage biotechnology company creating novel small molecule therapies that inhibit RNA modifying enzymes (RME) for use in oncology and other diseases. billion in funding. Northern Ireland “Northern Ireland is a medtech super-hub,” says Saleko.

Drugs 189
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Knopp Biosciences Announces Positive Top-Line Results from the Phase 2 EXHALE Trial of Oral Dexpramipexole in Moderate-to-Severe Eosinophilic Asthma

The Pharma Data

Preliminary analyses showed no clinically meaningful differences in safety results between the dexpramipexole and placebo groups, with no serious adverse events and 96% of subjects completing the primary assessment phase. 2 Dexpramipexole has been well tolerated in clinical trials comprising approximately 1,200 patients.

Trials 52