Santhera flunks DMD phase 3; Scribe emerges with $20 M; Merck sells off Osteoarthritis drug to Novartis; Research updates on flu vaccine and glioblastoma

Posted on Oct 8, 2020 by Delveinsight

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Designing a universal flu vaccine by focusing on the immune system 

A universal flu vaccine that can foster protection to people against any influenza strain is considered a goal in flu research as it could spare scientists from the often erroneous process of predicting the circulating strains each year and redesigning the vaccine to match them.

A group of researchers at the Massachusetts Institute of Technology, the Ragon Institute, and Bristol Myers Squibb has thrown light on a possible strategy for developing a universal flu vaccine by aiming at a more stable region of the influenza virus is normally not targeted by the immune system.

In mice, a vaccine that uses nanoparticles to carry flu proteins stimulated an antibody response to the desired segment of the virus, suggesting the vaccine could be broadly effective against any flu strain. The team showed the findings in the journal Cell Systems.

The influenza virus coats itself with a protein called hemagglutinin, which is vital for infecting human cells. Hemagglutinin consists of two parts:- a globular head region and a stem or stalk region. The head region often mutates and varies across different strains of influenza viruses. In contrast, the stem or stalk region rarely mutates, making it a great target for a universal vaccine.

Santhera flunks DMD phase 3, eliciting restructuring and withdrawal of approval application

Santhera Pharmaceuticals has ceased a phase 3 trial of Duchenne Muscular Dystrophy (DMD) drug idebenone after it flunked an interim analysis. The setback induced Santhera to outline plans to pull a filing for approval and restructure around a second asset that it nabbed global rights to last month.

Last year, Santhera refiled for the European Medicines Agency (EMA) approval of idebenone on the strength of data from a phase 3 trial that linked the synthetic short-chain benzoquinone to a slowdown in the loss of respiratory function. The application followed an earlier, drawn-out effort to get idebenone to market in DMD that ended with an EMA rejection in 2017 and a failed appeal early the following year.

Santhera started a second phase 3 trial, SIDEROS, to support a filing for approval in the U.S. That trial has brought the whole idebenone program crashing down and forced Santhera to shift its focus.

SIDEROS randomized 255 DMD patients were taking glucocorticoid steroids to receive idebenone tablets or placebo. The phase 3 trial was designed to display whether idebenone delays the loss of respiratory function. 

New immuno-oncology combo attacks glioblastoma in mice 

The brain cancer glioblastoma does not respond to immune-boosting therapies because of immune-suppressing molecules in its environment.

A team led by University Hospital Zurich and the University of Zurich engineered a protein-based drug that fuses immune-stimulating cytokines with antibodies that target glioblastoma. The drug suppressed tumor growth in mouse models of the disease and boosted the ability of immune cells to reach patients’ brain tumors in a small, ongoing clinical trial, as reported in Science Translational Medicine.

The Swiss scientists tested three different cytokines combined with antibodies targeting glioblastoma: IL-2, IL-12, and TNF. They found that antibodies fused to IL-12 or TNF and given to mice intravenously accumulated in glioblastoma tumors, decreasing their growth. Two out of five of the animals tested were cured, as reported.

Germany’s Merck sells off Osteoarthritis drug to Novartis

Germany’s Merck has decided to dismiss an unwanted bone drug to Novartis. For just USD 58 million upfront and USD 470 million backloaded in biobucks, Novartis is securing the phase 2-ready asset M6495 that has been in early tests for Osteoarthritis.

Novartis now chooses the program and will push on with the Osteoarthritis work as it seeks future approval.

The experimental drug works as an anti-ADAMTS5 Nanobody, focusing on self-administered via subcutaneous injections to maintain the structural integrity of the knee joint and suppress pain.

Osteoarthritis can range from mild to severe and can cause significant pain and disruption to everyday life. It typically affects older people, and more women are also affected than men, with common treatments focused on diminishing pain rather than treating the condition itself.

There have been efforts lately to search for new treatments, but some have fallen short, including Regeneron’s anti-NGF antibody fasinumab, which was culled from a phase 3 in 2018 after it flunked to find a favorable risk-benefit ratio.

Scribe Therapeutics emerges with USD 20 Million 

Scribe Therapeutics launches with USD 20 million in series A funding to develop that platform and a deal with Biogen to apply its technology. Biogen is handing over USD 15 million upfront to work on gene-editing treatments for Amyotrophic Lateral Sclerosis, with the option to go after another neurological disease aim. The Biotech is on the hook for another USD 400 million in development and commercial milestones between two Amyotrophic Lateral Sclerosis targets.  

CRISPR molecules include a guide RNA that identifies the target DNA sequence and an enzyme, usually Cas9, that nicks it. The scientists, including Doudna’s team at the University of California, have recognized more enzymes over the years, but this method of discovering them in nature, rather than designing new, purpose-built ones, curbs the reach of CRISPR.

Building CRISPR molecules from base allows Scribe to create treatments that can safely and effectively edit the genome and are small enough to fit inside adeno-associated viruses, or AAVs, which are one of the best ways for delivering CRISPR treatments to the body.

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