Remdesivir accelerates for COVID-19 recovery; Merck pact with Almac; Pfizer, BioNTech finish dosing in; Taysha Gene Therapies for trials

Posted on Apr 30, 2020 by Delveinsight

pharma-news

Remdesivir of Gilead accelerates COVID-19 recovery

Gilead Sciences’ Remdesivir shows the first data from a placebo-controlled study. Remdesivir decreases recovery time for hospitalized COVID-19 patients by four days or 31% as published in a National Institutes of Health-sponsored study setting the drug against placebo in more than thousands of patients.

The Food and Drug Administration (FDA) could give the nod to remdesivir for emergency use according to the results.

The study had carried out at 68 sites around the world, recovery as being healthy to return normal activity level. The trial was not reserved for the sickest of patients, not like other trials that only comprised of patients with severe disease, but the patients still had to be hospitalized with confirmed COVID-19 infection along with confirmation of lung involvement involving the need for supplemental oxygen or mechanical ventilation. The study did not cover the patients with milder, cold-like symptoms or those who were asymptomatic.

Merck pact with Almac for neurodegenerative diseases 

Merck has the latest deal which is an R&D pact with Almac Discovery. The deal is centred on deubiquitinases, which is a family of enzymes that play many roles in cellular regulatory processes.

The duo will recognize and create small molecule inhibitors of deubiquitinases, or DUBs, for the treatment of the neurodegenerative diseases. Merck will pick up lead optimization, preclinical and clinical development and commercialization. They did not reveal the financial terms or specific disease areas of interest.

There are some clear, neural pathological processors that both partners are very engrossed in and contemplate an essential role for certain [DUB-targeting] molecules. They will explore those pathological processes. At their core, DUBs eliminate ubiquitin from proteins and other molecules. Ubiquitin is a protein, cells utilize to tag damaged, or unneeded proteins for degradation in a protein complex called a proteasome.

Pfizer, BioNTech finish dosing in COVID-19 vax test

Pfizer and partner BioNTech are keeping pace with the speed of the pandemic as they complete dosing in the first cohort of their COVID-19 inoculation test.  

The biotech-pharma partners informed that the first cohort of BioNTech’s phase 1/2 clinical trial has been dosed in BioNTech’s native Germany and comes after a week when they were given the clear run trials in the country. It is a small study with just 12 participants, but all have been dosed with BioNTech’s vaccine candidate BNT162 six days after the trial commenced.

The full dose-escalation portion of the Phase 1/2 trial will run around 200 healthy subjects between the ages from 18 to 55 and will aim a dose range of 1 µg to 100 µg that targets to determine the optimal dose for further studies as well as to access the safety and immunogenicity of the vaccine. The study will also evaluate the effects of repeated vaccination following a prime injection for the three vaccine candidates that comprise of uridine-containing mRNA or nucleoside-modified mRNA.

Taysha Gene Therapies plans to run for clinical trails  

The team that developed Zolgensma is now for round two. A group of former AveXis executives and investors revealed a new gene therapy company. With 15 programs, USD 30 million in seed funding and an unparalleled collaboration with UT Southwestern Medical Center, Taysha Gene Therapies slates to be in the clinic year-end.

It is working on adeno-associated vector (AAV) gene therapies for monogenic diseases—diseases that are caused by a defect in a single gene of the central nervous system. Taysha’s lead program aims GM2 gangliosidosis, a very rare disorder that progressively destroys nerve cells in the brain and spinal cord; however, the company’s portfolio comprises of more prevalent conditions.
The company schedules to commence clinical trials for three more programs by the end of the 2021 year- treatments for Rett syndrome, a neurodevelopmental disorder; SURF1 deficiency, the most frequent cause of Leigh syndrome; and SLC6A1 genetic epilepsy that is similar to Dravet syndrome.

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