From gene therapies to psychedelics and cancer vaccines to HIV pre-exposure prophylaxis (PrEP) drugs, 2024 has been a busy year in the clinical trial space.
While many drugs have hailed positive data, sending stock prices soaring, others have failed to show benefit, with companies terminating the development of some highly anticipated therapies.
According to GlobalData’s Pharmaceutical Intelligence Centre, most trials completed in 2024 were Phase I studies (40.92%), with the second highest completion in Phase II (36.28%). During 2024, 15.62% of trial completions were Phase III studies. In 2024, there were 7,089 completions, more than the 6,234 trials that completed in 2023.
GlobalData is the parent company of Clinical Trials Arena.
Twice yearly HIV PReP boasts near-perfect efficacy
A Gilead representative received a standing ovation at AIDS24, held in July in Munich, Germany, after unveiling data from the PURPOSE 1 trial (NCT04994509) of lenacapavir for HIV PReP.
How well do you really know your competitors?
Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.
Thank you!
Your download email will arrive shortly
Not ready to buy yet? Download a free sample
We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form
By GlobalDataIn a Phase III trial in cisgender women, the twice-yearly injectable PrEP boasted 100% efficacy, with no infections in the treatment arm compared to daily PrEP.
A second Phase III, PURPOSE 2 trial (NCT04925752), read out in September 2024 and enrolled cisgender men, transgender men, transgender women, and gender non-binary patients who have sex with partners assigned male at birth. Not as perfect as the PURPOSE 1 study, in PURPOSE 2, Lenacapavir proved to be 89% more effective than Truvada, and it reduced HIV infections by 96% compared to background HIV incidence (bHIV).
Gilead said that due to the high efficacy and potential benefit in sub-Saharan African countries in reducing HIV infection, it will be offering generic licences for lenacapavir PReP to be developed at a lower cost to make it more accessible.
Fiona Chisholm, senior infectious diseases analyst for GlobalData said: “Low or inconsistent adherence to daily oral pre-exposure prophylaxis (PrEP) represents a major challenge in HIV prevention. Following the results of the PURPOSE 1 and 2 trials, lenacapavir is on track to become the first twice-yearly injectable PrEP agent in the global HIV market, providing an important alternative for people who struggle to adhere to daily treatment. The results of the studies were truly groundbreaking and validated the drug’s potential as PrEP among diverse populations. Yet despite the excitement surrounding these trial results, there are notable concerns surrounding the anticipated high cost of lenacapavir for PrEP compared to oral PrEP, which would limit accessibility for many at-risk populations.”
Scholar Rock SMA drug scores at Phase III
Scholar Rock’s spinal muscular atrophy (SMA) Phase III trial success caused the company’s stock price to increase by 362%. The Phase III SAPPHIRE trial (NCT05156320) evaluating Scholar Rock’s lead pipeline candidate apitegromab, met its primary endpoint of statistically significant and clinically meaningful improvement in motor function, as measured by the Hammersmith Functional Motor Scale Expanded (HFMSE).
Apitegromab is a monoclonal antibody that inhibits myostatin activation by selectively binding the pro- and latent forms of myostatin in the skeletal muscle. It is the first muscle-targeted treatment candidate to demonstrate clinical proof-of-concept in SMA.
There are very few treatment options for patients with SMA, highlighting the importance of this successful study. The company said it will look to file a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) and a marketing authorisation application (MAA) to the European Union in Q1 2025.
Obesity therapies
There have been several developments in glucagon-like peptide-1 receptor agonists (GLP-1RAs), with both Novo Nordisk and Eli Lilly reporting advancements with their therapies, Wegovy/Ozempic (semaglutide) and Zepbound.
A recent head-to-head battle saw Lilly crowned victorious as the superior weight loss drug. The open-label Phase IIIb SURMOUNT-5 trial (NCT05822830) enrolled 751 obese or overweight participants with at least one of the comorbidities such as hypertension, dyslipidemia, obstructive sleep apnea, or cardiovascular disease. By week 72, patients given Zepbound lost 20.2% of their body weight on average, compared to 13.7% for Wegovy – a 47% greater relative weight loss.
Pharmaceutical analyst for GlobalData, Costanza Alciati, said: “This outcome was expected from previous trial results and experience in the clinic, but head-to-head trials are important to remove bias from trial design and compare two drugs on the same grounds. Following these results and the negotiations that Eli Lilly is working on with different National Health authorities in multiple countries, we can expect Zepbound to take the lead in the obesity space soon. The price of weight loss medications is very high (a burden for patients and insurance companies) and if Lilly's drug not only is more effective for weight loss but also offers better pricing options, it will certainly steal a big patient share.”
The companies have also been investigating their therapies this year in indications beyond metabolic diseases, including cardiovascular and respiratory.
The Phase III SELECT study (NCT03574597) showed that Novo Nordisk’s semaglutide significantly reduced hospital admissions and overall length of hospital stay for adults with obesity or overweight with established cardiovascular disease (CVD) and without diabetes.
Meanwhile, Lilly’s Phase III SUMMIT trial (NCT04847557) found that tirzepatide reduced the risk of heart failure outcomes in patients with heart failure with preserved ejection fraction (HFpEF) and obesity.
“Semaglutide is expected to be approved for chronic kidney disease by next year and tirzepatide for obstructive sleep apnea and cardiovascular risk factors. These medications, and maybe the following generation which will be coming through in the next few years will impact the whole cardiometabolic sector, possibly revolutionising it, as they did with obesity,” Alciati added.
Other major pharma companies have seen trial read outs of their GLP-1RA obesity candidates including Amgen which provided data for its monthly drug MariTide (maridebart cafraglutide, formerly AMG 133). In a Phase II trial (NCT05669599), there was an average of 20% weight loss at 52 weeks for patients who are overweight or obese without type 2 diabetes.
Roche also had a trial read out for its $2.7bn weight loss bet on Carmot Therapeutics’s oral GLP-1RA CT-996, with patients reporting a 7.3% decrease in body weight after four weeks.
Amylyx scrapped ALS drug after Phase III flop
Despite previously bagging accelerated approval for its amyloid lateral sclerosis (ALS) drug Relyvrio (AMX0035), Amylyx Pharmaceuticals was forced to remove the drug from the market after a Phase III flop.
Relyvrio bagged approvals for the ALS drug from Health Canada in June 2022, and the FDA in September 2022, based on results from the Phase II CENTAUR clinical trial (NCT03127514). However, the European Medicines Agency (EMA) rejected Amylyx Pharmaceuticals’ marketing authorisation in October 2023, citing concerns surrounding results from the same study.
In the Phase III study (NCT05021536), which read out in March, researchers concluded that the drug didn’t have better results than the placebo after the trial failed to meet its endpoints. The news decimated the company’s stock by more than 85%.
Pfizer’s DMD drug despair
Pfizer has dropped the development of its Duchenne muscular dystrophy (DMD) candidate fordadistrogene movaparvovec after a Phase III failure (NCT04281485) and Phase II patient death.
The Phase III CIFFREO study didn’t cause improvement in motor function among DMD patients aged 4 to 7 years old. The global, multi-centre, randomised, double-blind, placebo-controlled trial aimed to evaluate the safety and efficacy of the gene therapy in participants who were on a stable daily regimen of glucocorticoids. The primary endpoint was the change from baseline in the North Star Ambulatory Assessment (NSAA) total score after one year. Meanwhile, the Phase II DAYLIGHT trial (NCT05429372) enrolled boys aged 2 to 4 years old.
Cassava Sciences Alzheimer’s drug failure
Cassava Sciences ended the development programme of its Alzheimer’s disease drug simufilam after it failed to show benefit in a Phase III ReThink-ALZ study (NCT04994483). The company’s stock dropped dramatically following the announcement.
Cassava was due to reveal data from the ReFocus-ALZ trial (NCT05026177) in January but stated that due to the ReThink-ALZ data, it Is also dropping the ReFocus-ALZ study and the open-label study (NCT05575076) – effectively ending all development of the drug.
This year the company was accused of data manipulation and presenting misleading data from its Phase IIb trial (NCT04079803). Without admitting or denying the allegations, Cassava agreed to enter a settlement with the Securities and Exchange Commission (SEC), paying $40 million.
“Given the large unmet need in the Alzheimer’s disease market for more effective DMTs, the failure of another potential DMT is disappointing and highlights how challenging drug development is for Alzheimer’s disease,” Phillipa Salter, neurology analyst at GlobalData said.
“However, when I spoke to key opinion leaders before the negative trial results were announced, there was a lot of negative sentiment surrounding the drug and the company. This type of controversy can have a lasting negative impact on the drug and company image.”
Eisai reports Alzheimer's disease success
Amid Cassava's controversies, Eisai reported positive data from a Phase III open-label extension study of its Alzheimer's disease drug Leqembi (lecanemab).
In the Phase III CLARITY AD study (NCT03887455), Leqembi reduced cognitive decline on the Clinical Dementia Rating scale Sum of Boxes (CDR-SB) by 0.95 points compared to the expected decline as seen in the Alzheimer’s Disease Neuroimaging Initiative (ADNI) study (3.09 vs 4.04). The data was shared at the 2024 Alzheimer’s Association International Conference (AAIC), being held 28 July – 1 August in Philadelphia, Pennsylvania.
The drug was first approved by the FDA in Alzheimer's disease in July 2023.
