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Nine for 2023, part two: healthcare’s hard problem, the prognosis for diagnosis, and key new pharmacotherapy platforms

pharmaphorum

There were developments in the past 12 months, true – Biogen/Eisai’s lecanemab (now branded Leqembi) showed clear success in the Clarity AD trial and received approval by FDA on 6th January 2023. This is linked to wider questions about the role damaged proteins and their build up plays in ageing across different tissues and organs.

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The Biotech Effect

Pharmaceutical Technology

“The inherent advantages of biopharma are that it allows you to pursue therapies against targets that are not small molecule therapies, for example by targeting protein-protein interactions and targeting GPCRs (G-protein coupled receptors),” he says. “Biotechnology has exploded across the industry. .

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Sosei Heptares to Explore Structure-based Drug Discovery (SBDD) Approaches to Ion Channels through Strategic Technology Collaboration with Metrion Biosciences

The Pharma Data

Ion channels represent a large but under-exploited class of drug targets beyond G protein-coupled proteins (GPCRs). Ion channels are a class of integral membrane proteins that regulate the flow of ions across the cell membrane as a means of conducting signals between cells and their environment.

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AbbVie Snatches Up Antibody from Harbour BioMed to Fight COVID-19 and Related Mutations

The Pharma Data

AbbVie has initiated a Phase I clinical trial of the antibody, and it will conduct the initial clinical program in the U.S. The antibody targets a specific conserved region of the SARS-CoV-2 spike protein. ” Under the license agreement, the development of ABBV-47D11 will be greatly advanced.

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Roche’s Evrysdi (risdiplam) show long-term improvements in survival and motor milestones in babies with Type 1 spinal muscular atrophy (SMA)

The Pharma Data

Among the infants with an available assessment (n=48) treated with Evrysdi, 32 infants maintained and four gained the ability to sit without support for at least five seconds since month 24, as assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III).

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FDA approves Roche’s Evrysdi for use in babies under two months with spinal muscular atrophy (SMA)

The Pharma Data

More than 5,000 patients have now been treated worldwide with Evrysdi in clinical trials, compassionate use or real-world settings. Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics. Jude Children’s Research Hospital.

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NRG announces £16m Series A for IND for Parkinson’s and ALS

pharmaphorum

A pre-clinical pipeline of potential first-in-class brain-penetrant small molecule inhibitors of the mitochondrial permeability pore (mPTP) are to be developed. Inhibition of the mPTP has been shown, however, to protect neurons, reduce neuroinflammation, and extend survival in pre-clinical disease models.