What to expect from biotech clinical trials in the coming year 

clinical trends 2024

There’s an air of cautious optimism about 2024, after a difficult few years following the end of the pandemic when investment in pharma and biotech research dropped substantially. After this significant reset, there’s now hope that in 2024, in line with emerging trends, the funds will be there for the vital clinical research and cutting-edge technology that could redefine care for diseases and conditions for which treatment options are limited or non-existent. 

Investors are still likely to be risk-averse, however, and the consensus is that the bonanza seen during the pandemic is unlikely to return. But there are some ‘hot’ areas that are attracting investment because of recent successes such as obesity and neurology.

Roche finished 2023 with a $2.7 billion buyout of obesity biotech Carmot Therapeutics, which has a dual glucagon-like peptide-1 (GLP-1) and gastric inhibitory polypeptide (GIP) agonist, ready for phase 2 development.

It’s aimed for treatment of obesity in patients with and without type 2 diabetes, and could be a direct competitor to Eli Lilly’s U.S. Food and Drug Administration (FDA)-approved Zepbound, (tirzepatide) which has the same mechanism of action, and Novo Nordisk’s GLP-1 Wegovy (semaglutide).

After around a decade of clinical trial failures, there has finally been progress in recent years with a new generation of Alzheimer’s drug – first with the FDA approval in 2021 of Biogen’s Aduhelm (aducanumab), and of Eisai’s Leqembi (lecanemab) in 2023.

Dr. Jörg-Thomas Dierks, chief executive officer (CEO) of specialist neurology pharmaceutical company Neuraxpharm, pointed out that these successes are just the tip of the iceberg as both drugs can only slow progression of the disease.

He said: “Trying to address the many unmet medical needs that still persist in this therapeutic area with new disruptive therapies like AI and cell and gene therapies require high investment and consequently high risk. Nonetheless, it is great to see that pre-pandemic investment flows have now recovered.”

In medtech, Anne Osdoit, partner at Sofinnova Partners, said that neurology is likely to attract investment in 2024, in line with the trend in pharmaceutical R&D.

She said: “Neurotechnology, particularly neurostimulation, is poised to reach record-high investments, driven by deals focusing on pivotal clinical trials. Orthopedics and cardiovascular (interventional heart failure specifically) investments have also seen robust activity.”

Osdoit was cautious over the prospects for investment in digital health and medtech.

“The tough investment environment we’ve been experiencing over the past 12 months means the deals that do happen involve the best scientific ideas, great engineering and resilient, experienced teams. The medtech sector experienced a 35% drop in investment, to $3.7 billion across 276 deals in the first half of 2023, a significant decline from the previous year. This contraction is attributed to a decrease in new investor-led financings, and a notable dip in large financings over $200 million,” she said.

Renée Aguiar-Lucander, CEO of rare disease biotech Calliditas Therapeutics, said capital markets will “remain difficult to navigate” for public biotechs in 2024.

She added: “For good data and significant catalysts, there is certainly an ability to raise capital.  The private market has been somewhat more resilient, but even there, it has been challenging for many companies over the last couple of years. Hopefully, as rates stabilize, there is more certainty around cost of capital, facilitating funding.”

Innovations in gene editing and rare diseases

But there has been progress with closely-watched technologies such as gene editing after the U.K. became the first country in the world to approve the first CRISPR treatment for a disease.

The country’s Medicines and Healthcare products Regulatory Agency (MHRA) approved Vertex/CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel), for patients aged 12 and over for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDBT).

This preceded a decision from the U.S. FDA, which approved Casgevy in December 2023, based on data from an ongoing single-arm trial in patients with SCD.

In 2024, clinical research into CRISPR-derived technologies, base editing and prime editing, is expted to gather pace. In late 2023, there were the first clinical results from Verve Therapeutics’ VERVE-101, a base editing therapy which permanently deactivates the gene PCSK9, and was the first therapy of this kind in the clinic in 2022.

This controls the level of low-density lipoprotein (LDL) “bad” cholesterol associated with heart disease – a readout from the company’s phase 1b trial in patients with high-risk heterozygous familial hypercholesterolemia (HeFH), established atherosclerotic cardiovascular disease and uncontrolled LDL levels on standard oral therapy.

Three patients out of 10 saw their LDL cholesterol levels cut but one patient experienced a myocardial infarction the day after receiving the treatment, considered potentially related to the dosing.

Another patient suffered a fatal cardiac arrest around five weeks after treatment, although this was determined to be caused by underlying ischaemic heart disease rather than the treatment.

The safety data were of concern to investors but the efficacy data has been seen as a proof-of-concept for this iteration of CRISPR gene editing.

Clinical trials of another CRISPR variant, prime editing, are also expected to begin in 2024, in the quest for gene editing therapies that are more accurate and with fewer or no off-target editing events.

Jean-Philippe Combal, CEO of gene therapy biotech Vivet Therapeutics, added that approved treatments for hemophilia A and B will generate “significant commercial momentum” for proven adeno-associated virus (AAV) gene therapy technology. 

AAVs are now well established as a gene therapy vector, and have proven to be safe and effective in the clinic, and have been on the market since 2017.

Their major drawback is they can only carry smaller genes (around 2,000 bases), although there are hopes this could be improved as the technology develops.

Combal added: “In Duchenne muscular dystrophy, Sarepta, Pfizer, Solid and RGNXBIO will lead the way for new clinical data, and in Wilson’s disease our own Gateway clinical trial is ongoing.”

Data for ocular, CNS and hearing loss disorder, AAV gene therapies could drive financing rounds, while refinements to the technology could improve manufacturing yield and reduce costs.

Overall, rare diseases will continue to attract clinical research in 2024, continuing a long-term trend, according to Calliditas’ Aguiar-Lucander, including genetic and pediatric diseases such as Duchenne muscular dystrophy, Alport syndrome and amyotrophic lateral sclerosis.

She said: “Rare disease research has grown significantly over the last 15 years, and there seems to be an unabated interest and need for new medications for patients with rare diseases. Less than 10% of rare diseases have so far been addressed with medications, so there is a substantial unmet medical need.”

Investment and progress in cancer research: a key clinical trend in 2024

Oncology is an area that has always attracted research investment, with some arguing in the past that this came at the expense of other disease areas such as infectious diseases.

According to a 2023 analysis by contract research giant IQVIA, oncology trial starts are at historically high levels, and global spending on cancer medicines has increased annually by an average of 5% over the past five years. This is expected to continue in the coming years, according to IQVIA.

Mai-Britt Zocca, founder and CEO of IO Biotech, a specialist in cancer vaccines, added: “There’s no doubt the funding environment continues to be challenging, but companies with strong clinical data will be able to attract what they need to progress clinical trials and make regulatory submissions. However, I think it’s going to be harder for companies with early pipeline assets.”

Dr John Maher, chief scientific officer at cancer cell therapy biotech, Leucid Bio, said there was a surprising lack of funding for research into the area, despite a high profile success in the clinic from a group led by Franco Locatelli, in the Ospedale Pediatrico Bambino Gesù in Rome, Italy.

Maher said: “It surprises me that funding remains somewhat tight in the current climate. The transformational data achieved by the Locatelli group in solid tumor patients really should have translated into a large bounce for the field, but the message doesn’t seem to have quite permeated as yet.”

Leucid’s Dr Maher added that in the field of CAR-T Immunotherapy, where a patient’s T-cells are genetically manipulated to fight cancer, further progress will be made in solid tumors following the findings from the Italian study.

“I remain firmly optimistic that the field will continue to advance steadily, and we will see more impressive developments in 2024,” Maher said.

Elsewhere in cancer, Catherine Pickering, CEO of iOnctura noted progress in the first-line setting for many cancers, noting that some have a five year survival rate of 95% thanks to the latest therapies.

But she added: “A key trend in clinical research for 2024 will be a shifting focus to acquired and adaptive resistance.  More and more companies, iOnctura included, are focusing on characterizing the mechanisms behind this resistance using validated multi-omic approaches at the intersection of biology / immunology and computer science. As resistance mechanisms are uncovered, companies will be looking to intelligently combine different drugs to overcome them.”

IO Biotech’s Mai-Britt Zocca added that cancer vaccines could play a role in improving response rates to checkpoint inhibitors by modulating the tumor micro-environment and making it less immunosuppressive.

In 2024, IO Biotech expects an interim analysis of a phase 3 trial for its immune-modulating vaccine IO102-IO103, combination with U.S. based Merck & Co’s anti-PD-1 checkpoint inhibitor, Keytruda (pembrolizumab).

While new modalities are making the headlines, there has been a resurgence in interest in small molecules as cancer drugs.

They are often available as patient-friendly oral therapies that don’t require hospital visits to administer. 

There are other technical advantages as they can pass through cell membranes to reach targets within the cell. They tend to be cheaper to manufacture and can be easy to store without need for refrigeration.

Jerry McMahon, CEO of STORM Therapeutics, said: “Small molecules are back at the forefront, especially with novel chemistries that do not rely upon catalytic inhibition of the target. Inhibition or modulation of RNA represents a new frontier for small molecules.”

Recruitment challenges in clinical trials in 2024

After the continuing geopolitical turmoil in 2023, there are challenges with research regions affected by war and like many industries, pharma and biotech will be hoping for stability in 2024, as they search for places to recruit patients for clinical trials.

The ongoing issue of clinical trial recruitment will continue, according to industry experts.

Paul Little, CEO of Vesper Bio, a company which specializes in sortilin receptor biology and has its lead programme focused on frontotemporal dementia, outlined the challenges of trial recruitment for rare diseases.

He said: “Site visits can be challenging and upsetting for patients, so allowing patients to self-dose at home and minimize the need to interrupt their, and their caregiver’s day is important. To overcome this, it is critical to build a network of contract research organizations, key opinion leaders and patient groups, with whom you can work closely with, to optimize your clinical trial design, while always keeping the patient in mind.”

Meanwhile, iOnctura’s Catherine Pickering welcomed U.S. legislation contained in spending legislation (Consolidated Appropriations Act, 2023), aimed at increasing representation of groups on clinical trials who have been historically omitted from clinical research, such as women and ethnic minority patients.

The U.S. National Institutes of Health (NIH) and numerous other healthcare figures have raised concerns that decades of testing drugs primarily on white men, means there are gaps in knowledge about how drugs work in large parts of the population.

Pickering said: “[The] FDA will require diversity plans for all phase 3 clinical trials conducted in the U.S.. This will start to shape the clinical trial landscape in 2024. Historically, women and racial and ethnic minorities have been underrepresented in clinical trials.  It is a positive move that companies are being encouraged to address this.”

The rise of AI: a top trend in clinical research in 2024

Edward Kliphuis, partner at Sofinnova Partners, said artificial intelligence will continue to shape clinical research in 2024 and beyond, helping to interpret the vast amounts of health data that is emerging from various technologies.

At the same time, use of wearable devices will allow health monitoring to grow substantially, creating a far more personalized healthcare experience.

A key development has been the rise of generative artificial intelligence (GenAI), which is capable of generating text, images or other media, Kliphuis said. This can be used to create a whole ecosystem of sophisticated, patient-centric health management tools, he added.

He said: “We expect the collaboration between tech companies and healthcare providers to deepen, leveraging GenAI to create more sophisticated, patient-centric health-management tools. This collaboration is expected to bridge the gap between data collection and practical healthcare delivery, making digital health solutions more integral to everyday healthcare practices.”

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24 top innovations for 2024

24 scientific discoveries from academic teams around the world selected by the global R&D community  

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