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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.

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Positive Phase I results for first-in-kind small molecule complement inhibitor

Drug Discovery World

ANX1502 is a first-in-kind oral, selective small molecule inhibitor that targets the active form of C1s responsible for propagating classical pathway activation in association with C1q. Across all doses evaluated, ANX1502 was generally well tolerated with mild to moderate treatment-emergent adverse events (TEAEs).

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Novartis announces positive results from Phase II study of LNP023 in patients with paroxysmal nocturnal hemoglobinuria (PNH)

The Pharma Data

LNP023 also demonstrated a favorable safety and tolerability profile with no serious treatment-related infections or thromboembolic events. Following the cut-off date for the presented data, one participant, who had severe lymphopenia at study entry, discontinued treatment due to a serious adverse event (AE) of lymphoproliferative disorder.

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Concert Pharmaceuticals Reports Third Quarter 2020 Financial Results and Provides Company Update

The Pharma Data

The 8 mg and 12 mg twice-daily doses continued to be generally well tolerated and adverse events were consistent with those reported in the Phase 2 trials. Published Findings on Alopecia Areata Burden of Disease in Journal of Investigative Dermatology.

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Deconstructing the Diligence Process: An Approach to Vetting New Product Theses

LifeSciVC

There is a continuum of evidence for a given target – at one end are novel targets with some evidence of importance in disease, and at the other end are “de-risked” targets where the biology is precedented with an approved product or late-stage clinical asset(s). and whether a molecule’s pharmacology can help to mitigate safety risk.

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Analysis Life Sciences Thank You FDA finalizes new guidance on nonclinical assessment of potential immunotoxicity

Agency IQ

For this reason, animal models are still used both during drug discovery and the more intensive nonclinical studies conducted during clinical development. However, this guideline is primarily intended to provide recommendations on evaluating the immune system’s response to small molecule drugs.

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Positive Phase 1 results in high-dose setanaxib trial

The Pharma Data

18, 2021 /PRNewswire/ — Genkyotex SA , a subsidiary of Calliditas Therapeutics AB (publ) (“Calliditas”) (Nasdaq OMX – CALTX; NASDAQ – CALT), today announced positive Phase 1 data demonstrating a favorable safety and pharmacokinetic profile of high-dose setanaxib, Genkyotex’s lead asset. STOCKHOLM , Jan.

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