Pharma’s memorable moments of 2024

More uses of GLP-1 drugs emerged

Since the glucagon-like peptide 1 (GLP-1) receptor agonists semaglutide and tirzepatide entered the market, they have become blockbusters and earned billions in sales. Even more remarkable are new results suggesting that the drugs might have more uses than first imagined.

Semaglutide is sold as Ozempic for diabetes and Wegovy for weight loss, while tirzepatide is sold as Mounjaro and Zepbound. But in March this year, the US Food and Drug Administration also approved semaglutide to reduce the risk of cardiovascular disease in people with a body mass index of 25 or higher. Other analyses have found that the drugs can help treat kidney disease, anxiety, depression, and substance use disorder.

Olivier Rascol, a neurologist at Toulouse University Hospital, told C&EN earlier this year that if these drugs can help those with mental illnesses or neurodegenerative diseases, he “would compare it to some of the major breakthroughs of the history of medicine.”

CRISPR drugs made their way into the world

This year, Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy for the treatment of sickle cell disease and β-thalassemia became the first drug using CRISPR gene editing to enter the market. Drug regulators approved the drug for those indications in late 2023 and early 2024. Time magazine even listed the drug as one of the best inventions of 2024. But uptake has been slow. The drug requires patients to undergo chemotherapy to prepare their cells for extraction. Then, the harvested cells are sent away to be edited. Doctors receive the edited cells after weeks or months and then infuse them back into the patient. On Vertex’s third-quarter earnings call, executives said around 40 people had started the process of cell harvesting, and just 1 patient outside clinical trials had received their edited cells back.

Protein-folding algorithms win Nobel Prizes

On Oct. 9, David Baker of the University of Washington and Demis Hassabis and John M. Jumper of Google DeepMind got calls from Stockholm telling them that they had won the Nobel Prize in Chemistry for 2024. The protein structure prediction and design tools built by these Nobel laureates have revolutionized biochemical research, and they have also found their way into pharmaceutical research. Big Pharma firms have partnered with Isomorphic Labs, a drug discovery spin-off founded by Hassabis, and Baker’s lab continues to create start-ups that aim to turn de novo designed proteins into medicines.

Firms built up ADC capacity

Antibody-drug conjugates (ADCs) received major investment this year. Syneos Health’s Dealmakers’ Intentions Survey 2024, published in January, listed the modality as number 1 on investors’ radar.

ADCs are what they sound like: compounds in which a small-molecule drug is attached to a monoclonal antibody with some linker chemistry. A handful of the drugs are already approved, and more are in the pipeline. Multiple firms have also invested in new ADC manufacturing facilities this year.

Researchers have also delved more into the role of the linkers themselves. While many firms prefer to stick to well-characterized linkers that are used in approved drugs, like Daiichi Sankyo and AstraZeneca’s Enhertu, others have started to explore new chemistries in an attempt to give themselves an edge. “Right now, we’re at the height of the ADC hype,” ADC expert Dowdy Jackson told C&EN in September. “I hope there will be some novel innovations in the field that can move it forward.”

Amylyx responded to bad news

In March, an approved drug for treating amyotrophic lateral sclerosis failed a confirmatory trial. It was a blow for ALS patient groups but Amylyx Pharmaceuticals made good on its preapproval promises and pulled the drug from shelves the following month. Amylyx is continuing to explore the drug for other uses, including Wolfram syndrome and progressive supranuclear palsy.

A call to recognize research in Africa

In September, Kelly Chibale argued in C&EN that there is untapped scientific potential and talent in Africa. “The time for African drug discovery,” he concluded, “is now.”

Virologists were kept busy

H5N1, mpox, Marburg. The virology and public health communities were kept busy this year monitoring multiple viral outbreaks.

North American scientists and public health officials have closely watched bird flu outbreaks in dairy cows that researchers suspect started at the tail end of last year. The US Centers for Disease Control and Prevention started tracking the virus in wastewater in May. In November, the agency recommended that all farmworkers be tested for the diseaseif they had come into contact with infected cattle, regardless of whether they were experiencing symptoms. Most cases of the disease in humans appear mild, and when C&EN went to press, human-to-human transmission had yet to be identified. But researchers are monitoring mutations that would make the virus better suited to human receptors, such as those found in a teenager from Canada.

Beyond North American shores, the deadly Marburg virus caused an outbreak in Rwanda this year, the country’s first. While there are no approved vaccines or therapeutics for the disease, the Rwandan government vaccinated health-care providers and other high-risk adults with an experimental vaccine from the nonprofit Sabin Vaccine Institute as part of a Phase 2 trial. A study of an experimental monoclonal antibody is also underway in the country.

In August, the World Health Organization declared mpox a global health emergency after outbreaks in the Democratic Republic of the Congo and other countries in Africa. Drug regulators in Congo had already approved the emergency use of two vaccines, Bavarian Nordic’s Jynneos and KM Biologics’ LC16m8, to try to control the outbreak. But despite donations from companies and countries, the total doses available remained a fraction of the number needed.

Radiopharma stayed hot

Radiopharmaceuticals—drugs containing a radioactive isotope, a chelating agent, a linker, and a targeting molecule—became hot properties this year. After Bristol Myers Squibb’s purchase of RayzeBio for $4.1 billion, announced at the very end of 2023, this year saw more big-ticket deals in the space. Novartis bought Mariana Oncology for $1 billion, while AstraZeneca nabbed Fusion Pharmaceuticals for $2 billion. Other radiopharma firms, like Ratio Therapeutics and Aktis Oncology, scored major partnerships with pharmaceutical companies.

That rising interest also drove increased investment in radiopharma manufacturing. A report by SNS Insider analysts estimates that the radiopharmaceuticals market will grow by 10% from 2024 to 2032. The number of options will also increase as companies expand the list of isotopes they use beyond actinium-225.

Evidence that vaccines protect against dementia grew

Studies published this year added to a growing body of evidence that the shingles vaccine may protect people from developing dementia in later years. Prior vaccination against tetanus, diphtheria, and pneumococcus is also protective, according to research. Other vaccinations, such as the flu jab, also appear to reduce the risk of developing the neurodegenerative disease. The unanswered question is how these vaccines are providing that protection.