PharmaFocus Today – Page 386 – media

Precision eye therapy for dogs ready for human clinical development

A successful gene therapy trialed at Michigan State University in dogs with an inherited eye disease is ready to be developed for clinical use in human patients with a rare condition called retinitis pigmentosa. Simon Petersen-Jones, professor and Donald R. Meyers and William E. Dunlap Endowed Chair in Canine Health at the Michigan State College of Veterinary Medicine, and his collaborators have published “Development of a translatable gene augmentation therapy for CNGB1-Retinitis Pigmentosa” in Molecular Therapy. Retinitis pigmentosa encompasses a group of rare genetic diseases that cause vision loss due to death of the light-sensing cells in the retina. Vision loss begins at a young age, and progresses throughout the lifespan. “There is currently an unmet need for treatment to save the vision of patients with CNGB1-retinitis pigmentosa,” Petersen-Jones said. “This promising therapy that works so well in dogs is now sufficiently developed that the next step is to take it forward for ...
- Source: drugdu
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Immunotherapy strongly improves prognosis of babies with leukemia

Immunotherapy with blinatumomab leads to a strongly improved survival rate—from 66% to 93%—for children with an aggressive form of acute lymphoblastic leukemia (ALL). They also had fewer side effects from the treatment. Based on the results, blinatumomab will now become standard treatment worldwide for babies with this aggressive form of leukemia. Three quarters of babies with acute lymphoblastic leukemia (ALL)—three babies a year in the Netherlands—have a particular fault in the DNA of their leukemia cells. This so-called KMT2A rearrangement leads to an aggressive form of ALL with a poor prognosis. Despite intensifying chemotherapy, the prognosis for these babies has not improved in recent decades. Intensive chemotherapy works very well for half of the babies. But in the other half of the children, the disease returned within two years, or children died from the disease or sometimes from the side effects of the therapy. 90% of recurrences—when the cancer comes ...
- Source: drugdu
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Sanofi, Regeneron unveil ‘blow-out’ smoker’s lung drug data

March 23 (Reuters) – Sanofi’s (SASY.PA) asthma drug Dupixent met all targets in a trial to treat “smoker’s lung”, potentially adding billions to the French drugmaker’s growth prospects, but also underscoring a heavy reliance on its bestseller. In a late stage trial Dupixent, jointly developed with Regeneron (REGN.O), led to a 30% reduction in moderate or severe acute exacerbations of chronic obstructive pulmonary disease (COPD), a potentially deadly disease marked by progressive lung function decline. Shares in Sanofi and Regeneron surged after they said in a joint statement on Thursday a Phase III trial involving 939 current or former smokers also showed improvements in lung function, quality of life and respiratory symptoms. The French group’s stock was up 5.2% at 1205 GMT, reaching a seven-month high and far outperforming the STOXX Europe 600 Health Care index (.SXDP), which was little changed. Regeneron jumped 8.6% in U.S. ...
- Source: drugdu
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Eli Lilly to sell low blood sugar drug Baqsimi to Amphastar in deal worth over $1bn

The drug is available in 27 markets, with worldwide sales totalling over $139m in 2022. Eli Lilly will sell its low blood sugar drug Baqsimi to Amphastar Pharmaceuticals in a deal worth over $1bn, the two companies announced. Under the terms of the definitive agreement, Amphastar will pay Lilly $500m at closing and an additional $125m after one year, with Lilly also eligible to receive sales-based milestone payments of up to $450m. Baqsimi is the first and only nasally administered glucagon to treat severe hypoglycaemia – very low blood sugar – in diabetes patients aged four years and above. Classed as a diabetic emergency, severe hypoglycaemia is a complication that can occur in diabetes patients who take insulin and certain anti-diabetic tablets. The condition is characterised by altered mental or physical functioning, including confusion and disorientation, convulsions, loss of consciousness and coma. Lilly launched Baqsimi ...
- Source: drugdu
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Leisure-time physical activity may improve mental health in teens

A recent study led by the University of Eastern Finland suggests that regular leisure-time physical activity, even in small doses, is associated with lower odds of depression, anxiety, chronic stress, and school psychologist visits among Finnish adolescents. However, the relationship between active school transport, i.e., walking or cycling to school, and mental health remains inconclusive. The findings were published in the prestigious Scandinavian Journal of Medicine & Science in Sports. Regular physical activity can improve mental health through various pathways, such as increasing the production of “feel-good” hormones, enhancing sleep quality, and boosting self-confidence. While mental health problems are common, few large population-based studies have examined the relationship between physical activity and adolescent mental health. Furthermore, it is currently unknown whether active school transport can have beneficial effects on mental health. The researchers observed that leisure-time moderate-to-vigorous physical activity was associated with better mental health among nearly 33,000 15-to-16-year-old ...
- Source: drugdu
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Orbital raises $270 million in biotech’s largest Series A round this year

Gwendolyn Wu Reporter Biotechnology venture firms have become more selective about where to invest in recent months, wary of the headwinds currently blowing against the industry. But that newfound caution hasn’t hurt Orbital Therapeutics, which said Wednesday it raised $270 million in biotech’s largest Series A round this year. The funds will help Orbital, which emerged from stealth last year partnered with gene editing pioneer Beam Therapeutics, to build its team and select a lead program. It claims its technology could be applied in many different directions, from use in next-generation RNA vaccines to protein replacement therapies or drugs for autoimmune diseases and cancer. Based in Cambridge, Massachusetts, Orbital has not yet named specific disease areas it’s prioritizing. However, the financing positions the company to submit its first regulatory filing, like an application to begin human testing, in the next two to four years, CEO Giuseppe Ciaramella said. “The ...
- Source: drugdu
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New research suggests that french fries may be linked to depression

French fries — they’re greasy, starchy and a comfort food for many. But reaching for fried foods may have a negative impact on mental health. A research team in Hangzhou, China, found that frequent consumption of fried foods, especially fried potatoes, was linked with a 12% higher risk of anxiety and 7% higher risk of depression than in people who didn’t eat fried foods. The link was more pronounced among young men and younger consumers. Fried foods are known risk factors for obesity, high blood pressure and other health effects. These results “open an avenue in the significance of reducing fried food consumption for mental health,” according to the paper published Monday in the journal PNAS. However, experts who study nutrition said the results are preliminary, and it’s not necessarily clear whether the fried foods were driving mental health issues, or people experiencing symptoms of ...
- Source: drugdu
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UK monkeypox vaccine rollout to wind down

The UK Health Security Agency (UKHSA) has announced its vaccination programme against mpox will end this summer. Cases of the disease have fallen from a peak of 350 per week in July to just six new cases so far in 2023, three of which were caught overseas. First doses of the vaccine will remain available to those at highest risk until 16 June, with second doses available until the end of July. The UKHSA says it will monitor cases and restart the programme if needed. While the mpox, formerly known as monkeypox, virus can infect anyone, most UK cases were among men who have sex with men. Anyone eligible who has not yet received a vaccine is being encouraged to book an appointment to protect themselves before summer. Greg Owen, from sexual health and Human Immunodeficiency Virus (HIV) charity the Terrence Higgins Trust, said: ...
- Source: drugdu
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BrainStorm files ALS drug application over FDA protest

Ned Pagliarulo Lead Editor A sign for the Food And Drug Administration is seen outside of the headquarters on July 20, 2020 in White Oak, Maryland. Sarah Silbiger via Getty Images A yearslong disagreement between the Food and Drug Administration and a developer of an experimental medicine for ALS will get a public hearing sometime in the future, setting up another high-profile debate of what amount of evidence merits an approval for the progressive and fatal disease. On Monday, BrainStorm Cell Therapeutics said the agency will convene a panel of outside experts to review its medicine, a personalized stem cell treatment called NurOwn. The planned meeting, which has not yet been scheduled, is the result of BrainStorm taking the rare step of requesting its approval application be filed over the FDA’s protest. Last November, the regulator refused to review BrainStorm’s application, which was based on a late-stage clinical trial that failed to meet its main goal. ...
- Source: drugdu
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Pfizer, BioNTech enlist Marvel’s Avengers in latest COVID-19 vaccine booster push

Just like how the Avengers have repeatedly kept the world safe from Ultron, people need to protect themselves by updating their COVID-19 vaccination with the latest booster. That’s the message Pfizer and BioNTech are trying to get across in a new custom comic book partnered with Marvel. The companies unveiled the project Tuesday. “We are proud to work with Marvel, which is so firmly entrenched in global culture and entertainment, to help remind people of the actions they can each take to help protect themselves, similarly to how the Avengers protect their community,” Pfizer said in a statement shared with Fierce Pharma Marketing. The new comic, titled “Everyday Heroes,” represents Pfizer flexing its marketing muscle. COVID vaccines are slated to switch to the private commercial market after the U.S. government failed to secure additional funding from Congress. As Pfizer CEO Albert Bourla, Ph.D., has said, Pfizer can be “even more competitive” and ...
- Source: drugdu
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