It’s not every day that drug companies need to worry about a treatment working too well, but in a recent trial for a new Eli Lilly weight-loss treatment, some slimmer patients had to drop out for fear of shedding too many pounds.
The findings helped solidify why the triple-hormone receptor agonist drug retatrutide has an estimated value of nearly $30 billion, and why it’s become one of the most closely watched next-generation options in obesity.
Lilly’s success is among a number of recent clinical trial wins in pharma, with some fueling fresh optimism for tough diseases. Revolution Medicines notched a high-profile victory with its RAS(ON) inhibitor, daraxonrasib, which nearly doubled survival in previously treated pancreatic cancer patients with metastatic disease.
While these may be two of the most dramatic success stories, other companies have moved the needle with trial results in psoriasis, hepatitis B and melanoma. Here are three notable hits.
A transformative approach in chronic hepatitis B
An antisense oligonucleotide drug, bepirovirsen, could offer a functional cure for patients with chronic hepatitis B infections, freeing them from the need for lifelong treatments with antiviral medications like Gilead Sciences' Vemlidy. While antivirals can reduce viral load and prevent complications from the infection, they typically don’t eliminate the virus.
GSK and Ionis Pharmaceuticals published results in May from a phase 3 trial showing that the treatment was able to clear the virus in 19% of participants, potentially positioning the drug for a rapid FDA approval and paving the way for more than $2 billion in peak sales for GSK.
“As the first medicine to deliver clinically meaningful functional cure rates, bepirovirsen is uniquely positioned to effectively treat CHB based on its potential to reduce the replication of hepatitis B virus, suppress hepatitis B surface antigen and stimulate the immune system,” Brett Monia, CEO of Ionis, which developed the drug and licensed it to GSK, said in a written release.
The FDA is already reviewing the drug, which was granted fast track and breakthrough therapy designations, with a PDUFA date slated for Oct. 26.
A potential new contender in psoriasis
While the psoriasis market is already competitive, there could still be room for new options to elbow their way in. Takeda Pharmaceuticals is making that bet with zasocitinib, an oral TYK2 inhibitor, in severe plaque psoriasis. Two global phase 3 trials found that the drug completely cleared skin plaques in 30% of patients, and 50% saw the lesions clear or almost clear after 16 weeks, Takeda said. The treatment improved outcomes compared to placebo as early as four weeks into the trial.
Zasocitinib appears poised to outperform Bristol Myers Squibb’s oral option, Sotyktu, which was performing below market expectations last year. But the drug will face competition from drugs such as Johnson & Johnson's FDA approved IL-23 receptor antagonist Icotyde and Alumis’ investigational TYK2 blocker, envudeucitinib, which may have an efficacy edge over zasocitinib.
While envudeucitinib hasn’t been tested against zasocitinib, in phase 3 trials, about 65% of patients had clear or almost clear skin after 24 weeks, and 40% had complete clearance. Alumis stated it may apply for FDA approval for envudeucitinib in the second half of 2026.
Many plaque psoriasis patients have historically opted against systemic treatments because they required office visits or injections, which is leading to this heated new battle to rule the market for oral options.
Personalized mRNA vaccines gain traction for deadly cancer
Moderna and Merck & Co. recently demonstrated the long-term promise of personalized mRNA cancer vaccines with intismeran autogene, which significantly reduced disease recurrence in high-risk stage 3 and 4 melanoma patients after five years, according to data from a phase 2 follow-up.
Results presented at ASCO showed that the customized investigational mRNA therapy, used in combination with Keytruda, increased progression-free survival by 49% compared with Keytruda alone. The drug is designed to spur the immune system to attack tumor cells. According to one of the study’s investigators, the results show the treatment could be an important addition to cancer treatment options, particularly for people with heavily mutated cancers.
The findings for intismeran autogene also hint at the highly anticipated potential of personalized mRNA vaccines, but additional studies are needed to solidify their place in the cancer treatment landscape.
