Novartis (NOVN.S), opens new tab said on Thursday an experimental drug, which it acquired ​as part of its $12 billion takeover of ‌Avidity, showed promise in an early-to-mid-stage study in patients with a type ​of genetic muscle disorder characterized ​by slowly progressive muscle weakness.

• The Swiss ⁠drugmaker said the drug, known as ​del-brax, lowered two blood markers linked ​to the disease and showed reduced signs of muscle damage in patients with facioscapulohumeral ​muscular dystrophy.
• Novartis said the drug ​shows potential to become the first disease-modifying ‌treatment ⁠for FSHD, which can cause weakness in the face, shoulders, arms and other muscles.
• The company estimates it affects ​about ​45,000 to ⁠87,000 people in the U.S. and EU.
• The drug’s safety ​profile was consistent with ​previous ⁠results, the company said.
• Novartis plans to discuss the data with health regulators ⁠around ​the world, while a ​late-stage study of the drug is currently enrolling ​patients.

 The Thomson Reuters

Boehringer Ingelheim said on Sunday ​its experimental obesity drug cut visceral and liver fat while minimizing loss of lean mass in ‌a late-stage study, data showed, bolstering its case for benefits beyond weight loss as competition in obesity drugs intensifies.

The drug, survodutide, was licensed from Denmark’s Zealand Pharma (ZELA.CO), opens new tab. An injectable that mimics the proteins GLP-1 and glucagon to create a feeling of fullness, its weight-loss trial results were ​announced in April, showing patients lost an average of 16.6% over 76 weeks.

Analysis of a group of ​patients who had MRI measurements at the start and end of a 76-week trial showed that survodutide reduced harmful ⁠abdominal fat by up to 34% and liver fat by up to 63.1% from the baseline, Boehringer said.

Analysts have ​said the weight-loss numbers were broadly comparable to existing GLP-1 injections from Novo Nordisk (NOVOb.CO), opens new tab and Eli Lilly (LLY.N), opens new tab and below newer ​rivals in development, and that the company needed to differentiate the drug’s benefits.

Lean mass accounted for no more than 10.8% of the change in body composition at the highest dose of 6 milligrams, suggesting the weight loss was driven mainly by fat reduction.

The drug’s effect ​on liver-fat reduction and preservation of lean mass are central to whether it will be able to stand out ​commercially, alongside tolerability and how long patients stay on the drug. Detailed data from the study could help Boehringer make a ‌stronger case ⁠that survodutide should be judged not only by pounds lost but by where weight is lost.

“We believe survodutide will become an important new option at the intersection of obesity and liver disease, two conditions that are deeply connected but rarely addressed together,” said Boehringer executive Shashank Deshpande, who leads the company’s human medicines business.

Boehringer acquired the rights in ​2011 to solely develop and ​commercialise survodutide from Zealand, which is ⁠entitled to royalty payments on global revenue.

PATIENTS WITH LIVER DISEASE BENEFIT

In a separate late-stage study of overweight or obese patients with a fatty liver disease called MASLD, survodutide ​met both its main goals.

After 48 weeks, up to 84.2% of patients on the ​drug showed ⁠a liver fat reduction of at least 30%, compared with 24.3% for those on placebo. Patients on survodutide also lost up to 12.2% of their body weight, versus 1% for placebo.

In 61% of the patients the drug helped achieve liver fat ⁠normalization, or ​a liver fat content below 5%, compared with 5.7% on placebo.

U.S. ​biotech Altimmune (ALT.O), opens new tab is also developing a drug that targets both the appetite-suppressing gut hormone, GLP-1, and glucagon.

Survodutide is also being tested in other late-stage ​studies, including for patients with fatty liver disease and fibrosis.

 REUTERS

Millions of people with breast cancer could safely avoid chemotherapy as scientists have developed a DNA test that can distinguish between patients who are likely to benefit from the treatment and those who are not, according to trial results.

The international study found that more than two-thirds of its participants could be spared the side of effects of chemotherapy and treated with hormone therapy alone.

Chemotherapy can cause fatigue, nausea, hair loss, a weakened immune system and fertility issues.

The study, led by University College London (UCL), involved more than 4,000 newly diagnosed patients over the age of 40 in the UK, Norway, Sweden, Australia, New Zealand and Thailand.

Scientists used a gene test called Prosigna to measure the activity of 50 genes involved in breast cancer growth and calculate a patient’s risk of the disease returning.

Those who received a low score – two-thirds of the group – were not treated through chemotherapy. The five-year survival rate of their group was 93.7%, compared with a 94.9% rate among patients who received chemotherapy as part of their care.

The primary treatment for breast cancer is usually surgery to remove tumours. Chemotherapy is often recommended afterwards to diminish the risk of return.

It is also regularly offered to people with early-stage breast cancer that has spread to the nearby lymph nodes.

Clinicians are concerned the treatment provides little benefit to those with the most common type of breast cancer, UCL said.

The university said more than 5,000 NHS patients a year could avoid chemotherapy as a result of the trial.

Karen Bonham, from Cardiff, took part in the trial and said the results are an “immense relief” and feel “like Christmas”.

The 64-year-old avoided chemotherapy thanks to the Prosigna test and has instead received radiotherapy and hormone therapy over eight years.

“Cancer diagnosis and treatment can be shocking,” she said.

“It certainly propels you into a world of uncertainty. Life priorities realign – you simply want to survive.”

The findings of the study will be presented at the world’s largest cancer conference, the American Society of Clinical Oncology’s annual meeting, in Chicago, United States, on Saturday.

Professor David Miles, a leading cancer specialist, described the findings as “practice-changing”.

“We can now confidently predict many patients will get no benefit at all, and therefore there’s no need for them to have the chemotherapy,” he told BBC’s Newshour.

He added that the test would allow doctors to “confidently define a large population of women who simply aren’t going to benefit and don’t need to go through all that unpleasantness for no benefit at all.”

“We used to give chemotherapy to 100 women to benefit 10, knowing that 90 didn’t need it,” he said.

Tanya Hutson, who was diagnosed with breast cancer in 2022 and had chemotherapy as part of her treatment, called the new DNA test “absolutely amazing”.

“It just proves what happens when money is put into research,” she said, adding that chemotherapy had been “brutal”.

“For all these people out there who don’t need it but are still getting it – it’s an absolute game changer.”

It is not known whether the findings apply to people under the age of 40, with a result still several years away, according to UCL.

BBC