Is Pharma finally looking 'Beyond the pill'?

• London, UK scientists have conducted the first-ever trial of a new drug for Alzheimer's disease that is able to safely and successfully lower levels of the harmful tau protein known to cause the disease. The trial, led by a team at the University College London, represents the first time that a "gene silencing" approach has been taken in dementia and Alzheimer's disease. The approach uses a drug called BIIB080 (IONIS-MAPTRx) to "silence" the gene coding for the tau protein -- known as the microtubule-associated protein tau (MAPT) gene. This prevents the gene from being translated into the protein in a doseable and reversible way. It will also lower the production of that protein and alter the course of disease. They found a greater than 50 per cent reduction in levels of total tau and phosphor tau concentration in the CNS after 24 weeks in the two treatment groups that received the highest dose of the drug. There are currently no treatments targeting tau. The drugs aducanumab and lecanemab -- recently approved for use in some situations by the US Food and Drug Administration -- target a separate disease mechanism in AD, the accumulation of amyloid plaques. (ETHealthWorld)
• GlaxoSmithKline (GSK) Pharma has announced the launch of Shingrix vaccine in India for the prevention of shingles (herpes zoster) and post-herpetic neuralgia in adults aged 50 years and above. Shingrix is the world’s first non-live, recombinant subunit vaccine to be given intramuscularly in two doses.?Shingles is caused by the reactivation of the varicella zoster virus (VZV), the same virus that causes chickenpox. A seroprevalence study in Indian subjects showed that by the age of 40 years, more than 90% had this virus in their body and were vulnerable to shingles, a company statement said. Shingles causes a painful rash. In most cases, the rash disappears, but in some cases the pain persists for months or years. This pain is known as post-herpetic neuralgia (PHN). The vaccine is being imported from its plant in Belgium and will be available at healthcare practitioners and certain specialists like dermatologists, across the country, Bhushan Akshikar, Managing Director with GSK Pharmaceuticals told businessline. The vaccine adds to GSK’s adult vaccine portfolio and is priced at a “significantly lower price” than in the United States, he said. Two doses of the vaccine in the US is priced at $450. (TOI, Businessline)
• Boehringer Ingelheim announced that the U.S. FDA granted breakthrough therapy designation for spesolimab as an investigational treatment for the prevention of flares in adults with generalized pustular psoriasis (GPP). GPP is a rare and potentially life-threatening chronic neutrophilic skin disease characterized by flares of widespread eruptions of painful, sterile pustules across the body. Flares greatly affect a person's quality of life and can lead to hospitalization with serious complications, including heart failure, renal failure, sepsis, and death. The FDA's Breakthrough Therapy designation is intended to expedite the development and review of a medicine that is intended to treat a serious or life-threatening disease and preliminary clinical evidence indicates the drug may demonstrate substantial improvement over available treatments. Spesolimab is marketed as SPEVIGO (spesolimab-sbzo) injection, for intravenous use by Boehringer Ingelheim. (Contract Pharma)
• The Food and Drug Administration granted accelerated approval for Biogen's drug tofersen, which treats a rare and aggressive form of the disease known as ALS. Accelerated approval is an FDA designation that clears drugs faster if they fill an unmet medical need for serious conditions. The approval requires Biogen and its co-developer Ionis to further study tofersen and verify its clinical benefits. ALS, commonly known as Lou Gehrig's disease, is a progressive and fatal neuromuscular disease that causes nerve cells in the brain and spinal cord to break down over time. Tofersen specifically targets a form of ALS in people with mutations in a specific gene, which are passed down through generations within families. (CNBC)
• AstraZeneca India announced plans to launch five Centers of Excellence across New Delhi, Ahmedabad, Goa and Cochin to provide a systematic approach to standardised and uniform management of patients with severe asthma. According to the company, the centers will serve as the exemplar hub for treatment and management of severe asthma patients, and they will, in turn guide and support evolving severe asthma centres for a systematic and guidelines-based approach to identify and treat severe asthma patients. “AstraZeneca is committed to reducing the strain that non-communicable diseases (NCDs), including severe asthma, place on healthcare systems through our innovative and focused therapies. We are deeply invested in developing ‘Beyond the Pill’ solutions to accelerate, improve and scale up holistic respiratory care across the healthcare ecosystem. In India, severe asthma is largely managed by oral glucocorticoids that have side effects such as osteoporosis, hypertension, ulcers, weight gain, etc., despite availability of newer treatment options such as biologicals that have shown to improve outcomes in patients. Our goal is to establish more such centers of excellence throughout the country and tackle complications by continually monitoring patients, educating HCPs, and improving respiratory health for all,” Dr. Anil Kukreja, Vice President – Medical Affairs & Regulatory, AstraZeneca India, said in a statement. Non-Communicable Diseases (NCDs) such as severe asthma can become a socio-economic burden for the patients, their families, and the healthcare system due to the various treatment costs involved. As a result, effective disease management necessitates specialized care from a multidisciplinary team of HCPs and caregivers. Early identification of severe asthma enables prompt diagnosis and treatment, resulting in improved clinical outcomes. The establishment of severe asthma clinics serves as a cornerstone of technological advancement and comprehensive management of the disease, it added. (Financial express)

The above is based on my personal research and does not represent my organisation’s views.