September - November 2020
Serum Institute of India Initiates Manufacturing of Codagenix’s Intranasal Live-Attenuated COVID-19 Vaccine Candidate
The Serum Institute of India, Ltd., the world's largest vaccine maker by the number of doses produced, has begun manufacturing CDX-005, the company's intranasal, live-attenuated vaccine candidate for SARS-CoV-2, the virus that causes COVID-19. Having received the necessary regulatory approval from the Review Committee on Genetic Manipulation (RCGM) of India's Department of Biotechnology (DBT), Serum Institute will initiate manufacturing for large-scale safety and efficacy studies. Codagenix is collaborating with the Serum Institute of India to develop CDX-005. Preclinical animal studies have been successfully completed, and Codagenix expects to initiate a Phase 1 first-in-human clinical trial in the UK by the end of 2020.
Blueprint’s Bet on Itself Pays Off With Rare Disease Drug Data
A cancer drug developed by Blueprint Medicines could help treat a rare and debilitating disease called systemic mastocytosis, based on Blueprint’s results from two trials. Treatment with Ayvakit, which was approved by the FDA in January 2020 for an uncommon type of gastrointestinal tumor, significantly reduced the proliferation of mast cells throughout the body, addressing the cellular cause of the chronic and unpredictable symptoms that people with systemic mastocytosis experience. Blueprint plans to ask the FDA to expand Ayvakit's use to allow for the treatment of systemic mastocytosis, a step that would further boost the company's drug pipeline.
Gene Therapy Company Taysha Completes Sprint From First Funding to IPO
Taysha Gene Therapies announced it would sell its first publicly traded shares at $20 apiece in an upsized initial public offering in a year that has featured dozens of lucrative biotech listings. The offering was priced at the top end of its estimated range and should raise $157 million. The company's focus is on rare, mutation-driven neurodegenerative disorders, and prior to its IPO, it received $126 million in venture financing to develop a pipeline of 18 announced programs, including a therapy it bought from Abeona Therapeutics. By developing a large arsenal of experimental gene therapies, Taysha is attempting to build a sustainable company that won't be tied to the market potential of a potentially one-time treatment for a disease with few affected individuals.
White House Approves Tougher Rules for COVID-19 Vaccine Development
The White House approved tough new rules for coronavirus vaccine developers that will make it unlikely that a vaccine will be approved before the 2020 Election Day. The approval came only after the U.S. FDA published the updated guidelines on its website as part of briefing materials for outside vaccine advisers. The guidelines recommend that participants in late-stage vaccine clinical trials be followed for a median of at least two months, starting after they receive a second shot. The standards, which would be applied to an emergency-use authorization for a vaccine, are similar to the standards for a traditional approval, but the criteria are likely to delay authorization of a vaccine.
FDA Approves Regeneron Antibody Drug as First Ebola Virus Treatment
The FDA approved the first treatment for the Ebola virus, clearing an antibody drug cocktail that was shown in a landmark 2019 study to reduce the risk of dying from the lethal and feared infectious disease. The drug, now dubbed Inmazeb, was developed by a New York biotech, Regeneron, in response to a different Ebola outbreak that spread virulently throughout several West African nations between 2014 and 2016. U.S. government researchers, working together with international aid groups and investigators from the Democratic Republic of Congo, tested the drug alongside three other antiviral treatments in the trial, which was conducted last year during the recent Ebola virus outbreak in the central African country.
Lily Wagers $135M on a Startup’s Plan to Stop Neurodegeneration
Eli Lilly has reached a deal to acquire Disarm Therapeutics, a biotech startup developing treatments for neurological diseases. Lilly will pay $135 million upfront and could shell out another $1.2 billion in milestone payments, although that additional cash isn't guaranteed. The acquisition gives Lilly rights to an early portfolio of experimental medicines meant to prevent the degeneration of axons, the thin tails of nerve cells that send out electrical impulses. None of the drugs are in human testing yet. The deal is a bet by Lilly on a startup trying to prove that devastating neurological conditions like multiple sclerosis can be treated by protecting axons.
Gloomy COVID Drug Data Shows Why Big, Randomized Trials Matter
According to the results of the world’s largest randomized clinical trial of potential COVID-19 drugs run by the WHO, Gilead’s Remdesivir—an antiviral originally developed to treat Ebola—showed no benefit on survival rates. A striking factor of the results was the apparent difference to the company’s own study. It showed that the treatment cut recovery time by five days compared with patients who got a placebo. In response to the data, Gilead questioned the accuracy of the WHO study, spurring a debate about what makes a trial more accurate.