Crucial 2024 Trial Results on the Horizon
In 2024, many trials await anticipated results. These studies represent the forefront of medical research, and their outcomes will influence the future of clinical development and treatment options for years to come.
1. NCT05669599
Amgen, not typically known for metabolic disease research, has sparked investor excitement with its entry into the obesity drug market. In May, 2024, the company announced promising results for its experimental weight loss drug, maridebart cafraglutide (MariTide), which led to a significant stock surge and a boost in market value. MariTide is an injectable therapy that targets gut hormones GLP-1 and GIP. Unlike Lilly’s Zepbound, which stimulates GIP, MariTide blocks it and may offer longer-lasting effects with just monthly injections. In a Phase 1 trial, participants on the highest dose lost 15% of their body weight over 85 days, raising hopes that MariTide could rival Zepbound and Wegovy. Amgen's executives expressed confidence in MariTide’s potential following an internal review of Phase 2 results, noting fewer study dropouts due to side effects—a common issue for obesity drugs. However, specific details were not revealed, building anticipation for full study data expected later this year. Even with a successful trial, Amgen faces challenges in a market dominated by Novo and Lilly. MariTide would likely require a Phase 3 trial over a year, plus a longer study to assess heart health benefits. Novo has already completed such a trial, securing Medicare coverage for certain patients with obesity and heart disease.
2. RELIEVE UCCD
Antibodies targeting the regulatory protein TL1A have attracted significant interest from major drugmakers as potential treatments for inflammatory conditions. Merck’s nearly $11 billion acquisition of Prometheus Biosciences and Roche’s $7.1 billion purchase of Telavant were driven by promising Phase 2 trial results showing their TL1A blockers could effectively treat hard-to-manage inflammatory bowel disease. These drugs may prove more effective and longer-lasting than current therapies. Teva, primarily known for generics, is focusing on branded drugs and has positioned a TL1A blocker at the center of its growth strategy. Teva claims its candidate may outperform others, prompting Sanofi to invest $500 million for shared rights. This drug could become a leading option for serious gastrointestinal diseases. Teva’s study parallels those of Prometheus and Telavant, which evaluated TL1A therapies in moderate-to-severe ulcerative colitis or Crohn’s disease. The results will also impact AbbVie and Spyre Therapeutics, both of which have preclinical TL1A inhibitors..
3. REDEFINE programs
With billions in potential profits at stake, leaders in obesity drug research like Novo Nordisk and Eli Lilly are not becoming complacent. Both companies are heavily investing in new medicines and combinations to strengthen their market positions. Novo’s experimental treatment, CagriSema, will yield Phase 3 results later this year. This once-weekly injection combines the active ingredient from Wegovy with an amylin analogue, aiming for greater weight loss than Wegovy alone. CagriSema is being tested in two large trials, Redefine 1 and Redefine 2, involving obese participants with and without diabetes. Redefine 1 includes 3,400 non-diabetic participants, measuring total weight loss over 68 weeks and the percentage of participants losing at least 5% of their body weight. Redefine 2 involves 1,200 participants with the same objectives. In earlier trials, CagriSema resulted in a 17% weight loss over 20 weeks, comparable to Wegovy’s performance over 68 weeks. Novo is hoping for improved results in the longer trials, while Lilly’s Zepbound has set a benchmark of around 21% weight loss.
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4. EMPOWER programs
AbbVie is investing nearly $9 billion in Cerevel Therapeutics, hoping an experimental schizophrenia treatment will prove profitable. Results from two clinical trials, set to be released by year’s end, will reveal if this investment pays off. The placebo-controlled trials, involving around 750 participants, are testing three doses of Cerevel’s drug, emraclidine. The primary aim is to determine if it can alleviate schizophrenia symptoms—both “positive” (like hallucinations) and “negative” (like emotional withdrawal)—within six weeks. Initial data is expected in November. AbbVie needs favorable results to compete with Bristol Myers Squibb, which recently invested $14 billion in a rival therapy, KarXT, developed by Karuna Therapeutics. Unlike older antipsychotics, emraclidine and KarXT target specific muscarinic receptors critical for brain function. Bristol Myers is ahead, having already achieved success in two late-stage trials for KarXT, which is under FDA review with a decision expected by late September.
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5. KOASTAL-1
Neumora Therapeutics was founded to tackle the challenges of drug development in neuroscience. Since its inception in late 2019, the biotech has secured over $600 million in private funding and executed one of the largest IPOs in the industry last year, focusing on developing more precise treatments and optimizing clinical trials. Its leading candidate is navacaprant, a depression drug that targets kappa opioid receptors (KORs), seen as a promising new approach for treating depression. Competing versions are also being developed by Johnson & Johnson and Cerevel. Neumora anticipates results from its first of three Phase 3 trials of navacaprant in depression by the fourth quarter. This will be a crucial test of the drug's potential, though the other two trials may provide flexibility if initial data fall short of high expectations.
6. GA43191
In the past year, many pharmaceutical and biotech companies have invested in cell therapies for autoimmune diseases, aiming to replicate academic findings that suggest these treatments can induce long-lasting remission for certain inflammatory conditions that other drugs can't achieve. Drugmakers are also exploring dual-targeting antibody drugs. Roche's mosunetuzumab, currently approved for lymphoma under the brand name Lunsumio, is among the first to provide early clinical data. Like other T cell engagers targeting CD20 and CD3, it eliminates B cells that produce protective antibodies and are linked to various autoimmune diseases. Early research suggests that B cell depletion may lead to drug-free remission in conditions like lupus. T-cell engagers could offer a more convenient and safer alternative to cell therapies, though their effectiveness and ability to deplete B cells thoroughly remain uncertain. The results of Roche's Phase 1 trial of mosunetuzumab in patients with the most common form of lupus could be significant. This study has been ongoing for two years, with a primary completion date set for September. By then, several other T cell engagers targeting different proteins may also be in or nearing human trials.
7. BROADWAY
CETP inhibitors, once seen as potential breakthroughs in heart medicine, have largely fallen out of favor due to multiple failures and serious side effects, leading companies like Pfizer, Roche, and Eli Lilly to abandon research. However, one CETP inhibitor, obicetrapib, developed by NewAmsterdam Pharma and licensed from Amgen in 2020, may change the narrative. After Amgen halted its development following a 2015 acquisition of its original maker, Dezima, NewAmsterdam restarted testing, believing obicetrapib could be more effective and safer than previous drugs. Early studies show that obicetrapib raises HDL while lowering “bad” cholesterol and other heart disease markers without the safety concerns seen in other CETP inhibitors. Three ongoing Phase 3 trials will provide more insight, with results expected soon. Despite skepticism from investors due to the troubled history of CETP drugs and existing cholesterol treatments, several analysts are optimistic about obicetrapib's potential, predicting it could generate billions in annual sales as an add-on therapy.
8. CMVictory
Moderna has been seeking its next major revenue source since COVID-19 vaccine sales began to decline last year. A potential opportunity could arise by the end of 2024 when the company expects results from a pivotal study of its cytomegalovirus (CMV) vaccine. Currently, there are no vaccines for CMV, the most common congenital infection globally. While it is asymptomatic for most people, it can cause serious health issues for those with weakened immune systems, including hearing loss and developmental delays in newborns. Historically, awareness of CMV has been low, with expectant mothers and newborns not typically screened due to the lack of preventive options. However, this may change as antiviral valacyclovir has shown promise, and several vaccine candidates are in development. Moderna’s vaccine is at the forefront, and if successful, it could significantly raise awareness of the virus and potentially generate over $3 billion in annual sales at peak.
