Regeneron’s I-O Drug; Gene Therapy for Parkinson’s Disease; Yumanity and Merck Inks $500M Deal; Simcha Raises $25M

Posted on Jun 25, 2020 by Anuj

Pharma-news

Regeneron recognizes new combos to boost I-O drug Libtayo’s cancer response

PD-1/L1 inhibitors work by enhancing the immune response to cancer, have benefitted patients combat several types of tumors. However, many people either do not respond to these checkpoint inhibitors, or they eventually develop resistance to them.

The scientists at Regeneron were searching for ways to enhance responses to PD-1 inhibitors, so they turned to bispecific antibodies that link the T cells of the immune system and tumour cells by binding to molecules present on cell surfaces.

Regeneron showed that adding bispecific antibodies aiming a T cell protein called CD28 to its Sanofi-partnered PD-1 inhibitor Libtayo led to improved anti-tumour activity in multiple animal models and long-term T-cell memory against the tumors as published in Science Translational Medicine.

Gene therapy eradicates Parkinson’s symptoms in mouse models

A researcher at the University of San Diego, California (USCD) made an accidental discovery several years back after he silenced a gene called PTB in mouse fibroblasts, cells in connective tissue. Within weeks, the majority of all of the fibroblasts were wiped out, and the rest had transformed into neurons.

Now, researchers are using the discovery to Parkinson’s disease in the anticipations of creating a one-time gene therapy to restore the dopamine-producing neurons, which are lost to the disease.

The UCSD team created a gene therapy technique, which impairs the ability of the PTB gene to produce a functioning protein. In mouse models of Parkinson’s, the gene therapy turned into supportive cells, also known as, astrocytes into dopamine-producing neurons, removing symptoms of the disease as reported by the researchers in the journal Nature.

Merck inks USD 500 Million Yumanity agreement 

Merck is penning USD 500 million-plus on a pact that looks it garner access to a neurodegenerative disease drugs of Yumanity.

The U.S. Big Pharma grabs the exclusive rights for two preclinical assets aiming amyotrophic lateral sclerosis (ALS) and frontotemporal lobar dementia (FTLD).

FTLD is a group of uncommon brain disorders, which primarily impair the frontal and temporal lobes of the brain. These areas of the brain are, usually, associated with personality, behaviour and language.

Whereas Amyotrophic Lateral Sclerosis causes a gradual deterioration and death of motor neurons, and will usually prove fatal.

There are, presently, four drugs approved by the U.S. FDA for treatment of ALS are Radicava, Rilutek, Tiglutik and Nuedexta although none is curative.

Simcha debuts with USD 25 Million to progress custom-built IL-18 for cancer

Cytokines, like interleukin-2, emerged as a promising treatment for cancer. They were the first modern drugs that proved the immune system could be aimed for cancer treatment.

Now, the problem is that the cytokines evolved as signalling molecules and not as cancer drugs. Some functions can be beneficial, and others can be deadly and toxic, or even oppose the function. 

Native interleukins required to be given at high doses and can cause side effects such as immunosuppression and vascular leak, where fluids and proteins leak from blood vessels into surrounding tissues, leading to organ damage. 

Simcha intends to solve those problems, and others, by engineering cytokines with a deliberate therapeutic purpose and it has raised USD 25 million from WuXi AppTec, Sequoia Capital and Connecticut Innovations to do so. Its lead program is a custom-built IL-18 that overcomes hurdles faced by the native protein.

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