This week's Philadelphia-area life sciences news includes updates on NRx Pharmaceuticals' Covid-19 therapy, ExpressCells' gene editing study, and Madrigal Pharmaceuticals' stock sale.
Here's the roundup:
NRx Pharmaceuticals
The Radnor biopharmaceutical company has filed a provisional composition of matter patent application with the U.S. Patent and Trademark Office tied to its Covid-19 therapy Zyesami.
The provisional application is titled "Stable, Buffer-free Compositions of Vasoactive Intestinal Peptide (VIP)." It describes compositions of vasoactive intestinal peptide, the synthetic form of which is aviptadil, that are both shelf stable and biologically active when used to treat Covid-19 and other diseases. NRx Pharmaceuticals (NASDAQ: NRXP) is developing a new drug candidate, Zyesami, that contains aviptadil, initially as a treatment for patients with critical Covid-19 and respiratory failure.
In its previous guidance to investors, the company advised that there were no filed patents protecting the manufacture of Zyesami. With the filing of the patent, NRx CEO Dr. Jonathan Javitt said the company believes it has a "path to a long-term commercial life" for a stable and pharmaceutically active form of VIP as an innovative drug.
"Although, for obvious reasons, our initial focus has been the use of VIP in lung disease caused by Covid-19, we are now considering potential use of Zyesami in non Covid-related lung disease, liver disease, eye disease, and organ transplantation," he said.
NRx filed an emergency use application for Zyesami with the Food and Drug Administration earlier this week.
ExpressCells
The Philadelphia biotechnology company spun out of Temple University said The CRISPR Journal has published the first study that validates the company’s Fast-HDR gene-editing technology.
The primary author of the article is ExpressCells' Co-founder Dr. Oscar Perez-Leal of the Temple University School of Pharmacy, who invented the Fast-HDR system.
The system involves the use of an empty vector to rapidly create donor templates for inserting DNA into the genome of mammalian cells. The donor templates are used in combination with the gene editing technique clustered regularly interspaced short palindromic repeats, or CRISPR, to insert new genetic information into living cells by promoting a natural process called homology donor repair (HDR). The process allows for the precise insertion of genes that can tailor cells to a scientist's experiment.
In the article, Perez-Leal and his co-authors describe the benefits of using Fast-HDR to create genetically modified cell lines that allowed the scientists to study three different proteins by tagging them with genes that code for fluorescent proteins. The process makes it easier to detect proteins being studied because the technology makes them glow.
“As a research scientist, I grew frustrated with the long lead times and inflexibility of older methods to detect multiple proteins by microscopy that needed cell fixing and antibodies,” Perez-Leal said. “I invented the Fast-HDR technology and realized it would benefit other scientists. That is why we established ExpressCells.”
Perez said the system leverages the cell’s own repair mechanism to insert DNA-encoded labeling tags into specific genes of the cell’s genome.
Baudax Bio
The Malvern pharmaceutical company raised $4.2 million in a private stock sale.
Baudax (NASDAQ: BXRX) sold 42,289 shares of convertible preferred stock and warrants to purchase up to nearly 12.7 million shares of common stock to a group of unnamed institutional investors.
The shares of preferred stock were valued at $100 per share, and are convertible into an aggregate of 16.9 million shares of common stock at a conversion price of $0.25 per share. The warrants have an exercise price of $0.32 per share.
Baudax intends to use the proceeds from the offering primarily for the continued commercialization efforts of Anjeso, the company's non-opioid pain medicine that was approved by the FDA in February 2020 for moderate to severe pain relief for patients in hospitals or other acute care settings.
H.C. Wainwright & Co. was the exclusive placement agent for the offering.
Madrigal Pharmaceuticals
The Conshohocken biopharmaceutical company pushed back its timeline for reporting late-stage clinical trial results for its lead drug candidate, resmetirom, to later this month from the end of last year.
Resmetirom is under development as a treatment for NASH, a non-alcoholic fatty liver disease.
Dr. Paul Friedman, CEO of Madrigal, said although blinded study data was delivered to the vendor conducting the statistical analyses on time, unexpected staffing issues at the vendor delayed its analyses.
Madrigal (NASDAQ: MDGL) is currently conducting two phase 3 clinical trials of resmetirom involving more than 2,000 patients for the treatment of NASH.
Quick hits
The United States Adopted Names Council has assigned the unique name Buntanetap for Berywn-based Annovis Bio's (NYSE: ANVS) lead drug candidate ANVS-401. The new drug candidate is under development as a potential treatment for Alzheimer's disease, Parkinson's disease, and dementia in Down syndrome. … The Wistar Institute in Philadelphia initiated a Phase 2 clinical study of VK-2019 in patients with advanced Epstein-Barr Virus-positive nasopharyngeal carcinoma, a rare type of head and neck cancer, and lymphoma. The study, led by researchers at the Stanford University School of Medicine, will enroll patients whose condition has recurred or progressed following standard therapy or have not responded to prior therapy. Funding for the mid-stage clinical trial is being provided by a grant from the National Cancer Institute.
