#clinical trials

#clinical trials

Incyte tops this list due to its rare combination of commercial scale, cash generation, and pipeline depth. The company posted FY2025 revenue of $5.14 billion, up 21.2% YoY, anchored by Jakafi generating $828.2 million in Q4 2025 alone (+7% YoY) and Opzelura delivering $207.3 million (+28% YoY). With $3.58 billion in cash and 14 pivotal clinical trials underway, Incyte offers an acquirer immediate revenue, margin expansion potential, and a deep oncology pipeline spanning KRASG12D, CDK2 inhibition, and mutCALR.

Lilly's fourth quarter revenue reached $19.29 billion, up 42.6% year-over-year, powered almost entirely by two products. Mounjaro generated $7.41 billion, up 110%, while Zepbound added $4.26 billion, up 123%.

Keytruda, first approved in 2014, belongs to a class of immunotherapy drugs that enable the immune system to attack cancer cells, extending survival for millions and transforming fatal diagnoses into manageable conditions.

The study published in the Lancet analyzed data from 381 patients with platinum-resistant ovarian cancer, revealing a 35% reduction in the risk of death among those treated with relacorilant compared to usual care.

On that sunny March morning, in a small health center in Lobamba, a rural area of Eswatini, this 32-year-old sex worker has just become one of the first people in the world to receive lenacapavir, a drug that, administered twice a year, offers nearly 100% protection against HIV.

Some 1 million patients in the U.S. live with a type of heart disease called heart failure with preserved ejection fraction, or HFpEF, caused by a stiffening of a chamber of the heart that makes it much more challenging to distribute blood throughout the body. The condition has few approved therapies and high mortality rates.

The INLIGHT interim readout reported a 14.3% placebo-adjusted reduction in visceral fat six months after a single dose, alongside preservation of lean muscle mass - a differentiated profile compared to GLP-1 therapies that often reduce muscle alongside fat.

The condition, called recessive RNU2-2-related neurodevelopmental disorder, is associated with seizures and severe developmental delay in children less than a year-old, in areas such as speech and walking.

Public health consultant Dr Ross Keat said supporting people earlier to make small preventative changes would make "a big difference later on". Some 3,500 people in the north of the island within that age bracket are eligible for the checks. The checks will be carried out by two pre-existing nurses that support GP staff and would not replace GP appointments, Keat explained, adding that the cost would be minimal and absorbed by Ramsey Group Practice.

Emma Dyer remembers the moment she clicked "buy now" on a set of weightloss jabs she found online. She had no medical consultation, no ID checks, and no questions about her history of anorexia and bulimia. "It was just so easy - too easy," she says. "They never asked for my medical history or what medication I was taking. It was like buying groceries."

Before treatment began, participants underwent neuroimaging. Instead of relying on a single modality, the researchers fused structural connectivity (how regions are physically wired) with functional connectivity (how regions co-activate at rest). The goal was not to throw every possible feature at a black box, but to learn a constrained pattern-what the authors call structure-function "covariation"-that carries the most predictive signal for outcome. In other words, the model tries to find the smallest set of connections that meaningfully forecasts symptom change.

A report last year found unnecessary surgeries were carried out, cancers were missed and poor standards of care were delivered at the University Hospital of North Durham and Darlington Memorial Hospital. CDDTF said it wanted to support the patients it had let down, including by offering access to psychological support, and to ensure they knew how to make a claim or raise concerns with police.

The cancer was fastacting, and if I'd left it even six months, the outcome could have been much worse,

Mucopolysaccharidosis type II (MPS II), or Hunter syndrome, is a rare genetic disorder primarily affecting boys, caused by a deficiency in the enzyme needed to break down sugar molecules. This harmful buildup in cells and tissues impacts multiple body systems, causing frequent infections, organ enlargement and developmental disabilities. Management involves supportive care and enzyme replacement therapy, as there is currently no cure,

The Food and Drug Administration commissioner's effort to drastically shorten the review of drugs favored by President Donald Trump's administration is causing alarm across the agency, stoking worries that the plan may run afoul of legal, ethical, and scientific standards long used to vet the safety and effectiveness of new medicines. Marty Makary's program is causing new anxiety and confusion among staff already rocked by layoffs, buyouts, and leadership upheavals, according to seven current or recently departed staffers.

Eduardo Vilar-Sanchez has spent more than 10 years pursuing a goal that seemed very distant, but which he now sees as a little closer: to develop a preventive vaccine against cancer. The physician and researcher is leading a study that presented the first promising results of a colon cancer vaccine in a small group of patients suffering from a rare disease that makes them 17 times more likely to develop colon cancer than the general population.

He has been living with atrial fibrillation (AF), a common heart rhythm problem, affecting 1.4m people in the UK, that can cause your heart to beat irregularly and often too fast. "It's very debilitating. On my worst day I feel very tired, my heart rate increases rapidly - I could walk for 2 or 3 miles and be okay, I could walk for 100 yards and it would hit me."

As for side effects, the companies reported that little had changed from previous analyses; adverse events were similar between the two groups. The top side effects linked to the vaccine were fatigue, injection site pain, and chills. The results "highlight the potential of a prolonged benefit" of the vaccine combined with Keytruda in patients with high-risk melanoma," Kyle Holen, a senior vice president at Moderna, said. They also "illustrate mRNA's potential in cancer care," he said, noting that the company has eight more Phase 2 and Phase 3 trials going for mRNA vaccines against a variety of other cancers, including lung, bladder, and kidney cancers.

EMPAVELI is the first and only approved treatment for C3G and IC-MPGN across pediatric patients 12+, adults, and post-transplant recurrence. That's roughly 5,000 patients in the U.S., with EMPAVELI holding exclusive approval for about two-thirds. Add the European CHMP positive opinion in December 2025, and you have a rare disease franchise with global expansion potential and pricing power that typically commands gross margins north of 90%.