Weekly Rundown: Report of first in vivo base editing

Welcome to the Weekly Rundown! In this new weekly column, the DDN editors will break down the most important biotech and pharma news from the past week. With short summaries of the latest industry news, we will keep you up to date on everything you need to know in drug discovery, development, and beyond.

This is a brand new column, so if there’s anything in particular you’d like to see, we welcome your thoughts and feedback at: editor@drugdiscoverynews.com

Report of first in vivo base editing

By making precise, single-base edits to the genome without needing to make double-stranded breaks, base editing is considered a safer gene editing technique than its older sibling CRISPR. Researchers at the biotechnology company Beam Therapeutics announced on Monday that they had demonstrated the first in vivo base editing in patients with alpha-1 antitrypsin deficiency (AATD), a genetic lung and liver disease. In Phase 1/2 results released so far, their drug, BEAM-203, resulted in no serious adverse events and led to higher levels of the functional alpha-1 antitrypsin protein in nine AATD patients with lung disease. The company plans to release more data from this trial in the second half of the year. Just one dose of BEAM-203 could be a potential cure for AATD. –Stephanie DeMarco

Long-acting PrEP injections could be given once per year

Individuals at risk of HIV can use pre-exposure prophylaxis (PrEP) medications to prevent infection, but they have to take oral pill options daily or receive an injectable option every two months. Gilead Sciences has been testing their drug lenacapavir, approved to treat HIV, as a long-acting injectable PrEP option. In new data from their Phase 1 trial, they showed an improvement in the dosage of lenacapivir — down from requiring two doses per year to just one (1). The trial demonstrated that two different lenacapavir formulations given intramuscularly resulted in higher drug concentrations in the blood compared to twice-yearly subcutaneous lenacapavir from Phase 3 trial data (2,3). Gilead Sciences has announced that they will begin testing once-yearly lenacapavir in a Phase 3 trial later this year. – Allison Whitten

Microplastics may spark antibiotic resistance

Microplastics — tiny pieces of plastic less than five millimeters in size — are increasingly associated with harms to the human body and the environment. Now, researchers at Boston University published results revealing that microplastics could even be driving antibiotic resistance (4). The scientists exposed Escherichia coli to different types of microplastics and found that the bacteria became more resistant to four antibiotics, and remained resistant even when the microplastics were removed. – Allison Whitten

Stem cell therapy shows promise for mild Alzheimer’s disease

A new therapy for mild, early-stage Alzheimer’s disease (AD) may be on the horizon. In a recent Phase 2a trial, researchers at Longeveron found that laromestrocel — a living cell product derived from mesenchymal stem cells isolated from fresh, young adult bone marrow — was safe, well-tolerated, and led to improvements in cognitive function and brain volume in patients with mild AD (5). These results could support the need for larger-scale clinical trials to test the new therapy, paving the way for potential regulatory approval and commercialization. That's good news for the nearly 13 million Americans projected to be living with AD by 2050. – Dika Ojiakor

High-dose vitamin D for treating multiple sclerosis

While there is no cure for multiple sclerosis (MS), a new therapy offers a promising new approach to managing the disease. In a randomized clinical trial with 303 patients, researchers found that a high dose of oral cholecalciferol — a supplement used to treat vitamin D deficiency — reduced disease activity in both clinically isolated syndrome (the initial neurological episode suggesting potential MS) and early-relapsing MS (6). The discovery raises hope for the use of vitamin D as an add-on therapy for managing MS. – Dika Ojiakor

A big week for obesity drugs

This week saw a few exciting updates in the drugs for obesity and type 2 diabetes space:

Roche is paying $1.65 billion to collaborate with Zealand Pharma to develop Zealand Pharma’s drug petrelintide — an amylin analog, planned to be taken weekly. The body normally secretes amylin from the pancreas where it works with insulin to slow gastric emptying and make people feel full faster.
Novo Nordisk released Phase 3 trial results of their weekly drug CagriSema, a combination of cagrilintide (an amylin analog) and semaglutide (a GLP-1 receptor agonist). While the drug led to 13.7 percent weight loss in patients in the study, that amount was less than investors were expecting. Novo Nordisk has plans to test CagriSema at higher doses.
Viking Therapeutics announced a $150 million manufacturing deal that they will pay to produce their obesity pill VK2735, a dual GLP-1 and GIP receptor agonist.
Amgen is getting ready for two Phase 3 trials for its monthly obesity medication, Maridebart Cafraglutide (MariTide): MARITIME-1 for obesity and MARITIME-2 for type 2 diabetes and obesity. – Stephanie DeMarco

Fifth anniversary of the start of the COVID-19 pandemic

March 11^th marked five years since the World Health Organization declared the COVID-19 outbreak a global pandemic. In just the few months before, scientists rallied to sequence the novel coronavirus, which would aid the development of diagnostic tests and eventually the life-saving mRNA vaccines. Those COVID-19 vaccines, which were built off of decades of mRNA vaccine research that would eventually lead to a Nobel Prize, saved countless lives. However, since 2023 the percentage of people who say they received a COVID-19 vaccine or booster shot within the past three months has decreased, according to a recent Axios-Ipsos poll. With SARS-CoV-2 still circulating in communities, the effects of the pandemic will resonate for some time. – Stephanie DeMarco

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