PharmaFocus Today – Page 246 – media

Interest in RNA Editing Accelerates as Therapies Approach the Clinic

By Gerard Platenburg Pictured: RNA/iStock, Artur Plawgo Based on the significant progress made over the last few decades with RNA therapeutics, RNA editing is widely considered the next generation of promising medicines in this field. RNA therapies have made significant progress over the last few years, with an increasing number of FDA approvals beginning in 1998 with Vitravene for CMV retinitis, followed by Macugen for macular degeneration in 2004 and Spinraza for spinal muscular atrophy in 2016. There have also been multiple siRNA-based drugs, including Onpattro for polyneuropathy of hereditary transthyretin-mediated amyloidosis in 2018. And finally, in 2020, perhaps the most well-known products in the RNA space were introduced: the mRNA-based COVID-19 vaccines. All of these demonstrate the strength of RNA therapies and their potential impact on diseases with high unmet need. RNA therapeutics are indeed elegant approaches to altering RNA and thus protein expression, opening the potential to target ...
- Source: drugdu
- 135
- October 17, 2023
FDA Approves Pfizer’s Ulcerative Colitis Drug, $6.7B Arena Buy Pays Off

By Connor Lynch Pictured: Pfizer’s office in Belgium/iStock, Alexandros Michailidis The FDA has greenlit Pfizer’s ulcerative colitis drug etrasimod, marketed as Velsipity, based on Phase III results published in The Lancet, the company announced Friday. Pfizer’s Elevate UC Phase III registrational program, consisting of the Elevate UC 52 and Elevate UC 12 clinical trials, examined the safety and efficacy of a 2-mg daily dose of the oral medication for patients intolerant to or who had failed on trials of a conventional, biological, or Janus kinase inhibitor therapy. Both studies “achieved all primary and key secondary efficacy endpoints, with a favorable safety profile consistent with previous studies of Velsipity,” Pfizer said in Friday’s announcement. In the Elevate 52 UC trial, 27% of patients achieved clinical remission, compared to 7% of patients receiving a placebo at week 12, and by week 52 it was 32% versus 7%. In the Elevate 12 UC ...
- Source: drugdu
- 105
- October 17, 2023
FDA Clears Pfizer’s Braftovi + Mektovi for Non-Small Cell Lung Cancer Treatment

Treatment indicated for patients with BRAF V600E-mutant metastatic form of disease. Image Credit: Adobe Stock Images/Tex vector Pfizer revealed that the FDA has approved Braftoni (encorafenib) + Mektovi (binimetinib) for the treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) with a BRAF V600E mutation. Previously, the medication was approved for the treatment of adult patients with metastatic colorectal cancer (CRC) with a BRAF V600E mutation. “Today’s approval builds on our long-standing commitment to deliver innovative, personalized medicines to patients with lung cancer. By pursuing precision medicines that target a patient’s specific type of cancer, we are leveraging our deep understanding of tumor biology to help address the underlying cause of disease,” said Chris Boshoff, MD, PhD, chief oncology research and development officer, EVP, Pfizer. “Since its initial FDA approval in 2018, Braftovi + Mektovi combination therapy has helped thousands of people living with BRAF V600E- or V600K-mutant ...
- Source: drugdu
- 108
- October 17, 2023
Financial Pressures Drove Nearly 40% of Hospitals M&A Deals in Q3

Hospital M&A activity has been returning to pre-pandemic levels this year, according to a new report. There were 18 M&A transactions in Q3, compared to seven in Q3 2021 and 10 in Q3 of last year. The reason why M&A activity is regaining its momentum is because hospitals are seeking partnerships to grow and protect their long-term financial sustainability — the report found financial distress was the driving factor behind nearly 40% of deals announced during Q3. By KATIE ADAMS Hospitals and health systems have undergone extreme financial pressure in the past couple years, and this issue is being reflected in the sector’s M&A activity, according to a new report from Kaufman Hall. The report pointed out that hospitals’ median operating margins were in the red for all of 2022. Things have gotten better this year, and 2023 will likely end as a much more stable year for hospital finance ...
- Source: drugdu
- 112
- October 17, 2023
Akero’s Three-Step Plan to Win Back Investors

By Jia Jie Chen Pictured: Stocks trending down/iStock, lerbank Shares of Akero Therapeutics crashed this week after the biotech released data from a late-stage trial of its lead candidate, efruxifermin, in nonalcoholic steatohepatitis. Although the Phase IIb study met multiple secondary endpoints, the effect on the primary endpoint was not considered statistically significant. The SYMMETRY trial compared 182 patients with compensated cirrhosis—also known as F4 fibrosis—who were treated with efruxifermin (EFX) with a placebo control group. The data showed that 22% of patients treated weekly with 28 mg of efruxifermin (EFX) saw at least a one-stage improvement in liver fibrosis with no non-alcoholic steatohepatitis (NASH) worsening, while 24% of those treated with 50 mgs of EFX achieved this result; this compared to 14% of patients in the placebo arm who experienced similar improvement. The differences between the placebo group and both experimental arms were not statistically significant, with p-values at ...
- Source: drugdu
- 116
- October 16, 2023
Ozempic’s Pros, Cons and Correct Use Cases

By George Kramb Pictured: Man preparing Ozempic injection/iStock, imyskin Thanks to TikTok, Ozempic has emerged as this year’s go-to weight loss drug. The current debate revolves around a need to understand Ozempic’s true benefits, unmarred by the overshadowing emphasis on weight loss. Ozempic is not a weight loss drug. It is an injectable prescription medicine used “along with diet and exercise to improve blood sugar (glucose) in adults with type 2 diabetes,” according to its label. As such, it is crucial to recognize its multifaceted nature and the responsible approach it demands. Ozempic’s role in the evolving world of healthcare—and inadvertently weight loss—involves both medical benefits and societal perceptions. The Authentic Benefits of Ozempic Amidst the clamor surrounding Ozempic’s media coverage, it’s imperative to redirect the focus to its true advantages when employed for its designated purposes. Ozempic’s primary function lies in regulating blood sugar levels, offering crucial assistance to ...
- Source: drugdu
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- October 16, 2023
Newly discovered synthetic adjuvant materials could make vaccines more accessible, sustainable

New raw vaccine materials that could make vaccines more accessible, sustainable, and ethical have been discovered. Adjuvants are vaccine ingredients that boost a person’s immune response to a vaccine, providing greater protection against disease. One of the most prevalent adjuvant materials used in vaccines is squalene, which is typically sourced from shark livers. Researchers at the University of Nottingham collaborated with the Access to Advanced Health Institute (AAHI) to identify synthetic alternatives to squalene that ensure sustainable, reliable, and ethical sourcing of adjuvant raw materials for vaccines moving forward. New synthetic adjuvant materials were developed from commercially available methacrylate monomers, ensuring that a reliable supply of the material is continually available. The combination of these adjuvant materials are scalable through catalytic chain transfer polymerization, a process that allows high levels of control over the molecular weight of the product polymer. Controlling the molecular weight is key to the use of ...
- Source: drugdu
- 113
- October 16, 2023
How Nemours Children’s Is Working to Solve the ‘Horrendous’ Youth Mental Health Crisis

If you think the state of young people’s mental health in the U.S. hasn’t reached a crisis point yet, you are sorely mistaken, Lawrence Moss said Monday during an interview at HLTH. He is the CEO of Nemours Children’s Health, a pediatric health system with more than 95 locations in four states. Moss gave some sobering statistics on the youth mental health crisis, saying that nearly a quarter of children in the U.S. have a diagnosable mental health disease and that youth suicide rates have quadrupled over the past few years. “When you talk about child health right now, it’s more about behavioral health than physical health. We don’t typically like to think of it that way, but that’s the reality. If you can only do one thing for the health of children in this country, it would be in the behavioral health sphere,” he declared. One of the most ...
- Source: drugdu
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- October 16, 2023
WHO announces €1.1bn collaboration to eradicate polio

The European Commission, the European Investment Bank and the Bill and Melinda Gates Foundation have announced a €1.1bn ($1.17bn) financing collaboration to tackle polio and make healthcare systems more accessible in low-income countries. The funding partnership is expected to include €500m ($531m) for the Global Polio Eradication Initiative (GPEI), including World Health Organization (WHO) and UNICEF, €500m ($531m) in investments and grants to strengthen health systems and €80m ($85m) grants for technical assistance. Polio is a serious viral infection that mainly affects children in low-income countries. The virus attacks the nervous system, leading to paralysis and sometimes death. According to WHO, wild poliovirus cases have decreased by over 99% since 1988. However, wild poliovirus remains an endemic in Pakistan and Afghanistan, with most of the cases being found in just four subnational regions. President of the European Commission Ursula von der Leyen said: “With 1 billion euros supported by our ...
- Source: drugdu
- 104
- October 16, 2023
Birmingham researchers develop nanobodies to understand platelet disorders

Researchers at the University of Birmingham have developed antibody fragments, called nanobodies, in a bid to understand platelet disorders such as thrombosis. The team at the Institute of Cardiovascular Sciences has been able to produce the first binding molecules of defined composition to make platelets clump together in a predictable way. The institute aims to use these nanobodies to develop validated clinical assays for testing patients with platelet disorders such as bleeding or thrombosis, as well as use them as a research tool to study platelet activation. The institute’s work is set to be published in the Journal of Thrombosis and Haemostasis later this week (12 October.) In previous studies carried out by the Birmingham Platelet Group, Professor Steve Watson and Dr Natalie Poulter used novel nanobodies the team had raised to bind to the GPVI (Glycoprotein VI) receptor. This receptor plays a role in thrombosis but only has minor ...
- Source: drugdu
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- October 16, 2023

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