PharmaFocus Today – Page 263 – media

Landscape review reveals £1.1bn of UK funding for rare diseases

The National Institute for Health and Care Research (NIHR) and the Medical Research Council (MRC) have published a landscape review report, revealing that £1.1bn has been invested in UK research for rare diseases. The report findings aim to help focus areas for future funding and better target the needs of people with rare diseases. The review found that the NIHR and MRC accounted for almost £627m in funding across 698 studies between 2016 and 2021 for rare disease research. Comprised of 7,000 different types, rare diseases affect around 3.5 million people in the UK. The report revealed that motor neuron disease received the most funding in the portfolio, accounting for 8%, followed by Huntington’s disease (5%) and then preeclampsia (5%). Other diseases that received significant funding included cystic fibrosis (4%), frontotemporal dementia (4%) and idiopathic pulmonary (3%). Over five years, approximately £99.7m out of the nearly £627m of investment was ...
- Source: drugdu
- 211
- October 1, 2023
NHS Confederation research supports investment in NHS services

Report shows that if health service invests in community services hospital admissions will reduce Research commissioned by the NHS Confederation shows that the more the health service budget is invested in priming community services the more it decreases hospital admissions – bringing further savings across the wider NHS. Key figures show that for every £100 spent in the NHS community care sector, delivering care to patients closer to and in their own homes, there is a £131 return on investment in acute sector savings. This money would typically have been spent providing care to patients in hospitals and across acute services. The findings show how those areas of the country that spent less on community care in terms of population need have also seen – on average – higher levels of hospital and emergency activity, compared to those spending more. Indeed, those of the 42 integrated care systems that invested ...
- Source: drugdu
- 104
- October 1, 2023
Roche scores—again—in hemophilia drug patent case against Takeda

In a patent case centered on two rival hemophilia medicines, Roche’s Genentech unit has scored its second straight win. After a U.S. district court last year ruled in favor of Roche and invalidated a patent held by Takeda’s Baxalta unit, the U.S. Court of Appeals for the Federal Circuit reaffirmed the ruling. In the decision, U.S. Circuit Judge Timothy B. Dyk wrote that in order to be valid, patents must describe inventions “in such full, clear, concise and exact terms” so that any skilled person can make and use the same product “without undue experimentation.” In this case, Baxalta’s patent describes an antibody that binds to a protein that’s key to enable blood clotting. In its appeal, Baxalta argued that skilled practitioners can use a screening process that doesn’t amount to undue experimentation. But the circuit court shot down this argument, supporting the decision that the patent should be invalidated. ...
- Source: drugdu
- 188
- October 1, 2023
analysts

Six months after Travere Therapeutics’ Filspari won an accelerated nod to treat patients with a rare kidney disease, the first-of-its-kind drug narrowly failed its confirmatory trial. While the results bode poorly for a full FDA approval anytime soon, Travere may take some solace in the fact that the drug’s removal from market looks unlikely, at least according to two groups of analysts. The readout comes from Travere’s phase 3 PROTECT study pitting Filspari (sparsentan) against the popular angiotensin II receptor blocker irbesartan in patients with primary immunoglobulin A nephropathy (IgAN). In the study, Filspari failed to reach statistical significance over irbesartan when it came to kidney function outcomes as measured by the estimated glomerular filtration rate (eGFR). It wasn’t all bad news for Filspari, which demonstrated long-term kidney function preservation and achieved a clinically meaningful difference on the eGFR slope versus irbesartan. The drug also hit statistical significance on its ...
- Source: drugdu
- 156
- October 1, 2023
Parkinson’s UK calls on government to mark World Patient Safety Day

The report outlines ways the government can help patients with Parkinson’s disease Parkinson’s UK has called on the government to take action to help people with Parkinson’s disease (PD) get their medication on time in hospital, as part of its relaunched ‘Get It On Time’ campaign to mark World Patient Safety Day on 17 September. The charity, alongside Diabetes UK, Epilepsy Action, National Aids, Rethink Mental Illness and the Richmond Group, supported by the Royal College of Emergency Medicine and the Royal Pharmaceutical Society, outlined in a joint statement how the government can take action to help patients with PD. PD is a progressive neurological condition which affects an estimated 128,000 people in England. People living with PD can experience severe harm to their health, such as trauma, as a result of not receiving their medication on time while in hospital. The issue also affects patients living with diabetes, epilepsy ...
- Source: drugdu
- 133
- October 1, 2023
Cancer Research UK invests £123m in Scotland

The investment will support research into cancer treatments and diagnosis Cancer Research UK (CRUK) has announced its largest ever investment of £123m in Scotland as part of a seven-year commitment to the CRUK Scotland Institute. The institute, which is facilitated in partnership with the University of Glasgow, will use the investment to support research into new and improved cancer treatments and diagnosis, as well as bolster Scotland as a major global hub for cancer research. It will also aid in attracting research talent to the Scotland Institute and will support major studies into specific types of cancer that largely affect Scotland’s population, including liver, pancreatic, bowel and lung. Despite mortality rates falling by 8% in the past decade, more than 34,000 people are diagnosed with cancer every year in Scotland. Previously known as the CRUK Beatson Institute, the CRUK Scotland Institute studies the roots of how cancer begins, the genetics ...
- Source: drugdu
- 111
- October 1, 2023
Merck announces partnerships worth $1.3bn with BenevolentAI and Exscientia

German group Merck has announced two new drug discovery collaborations with BenevolentAI and Exscientia to harness artificial intelligence (AI)-driven design and discovery capabilities to accelerate drug discovery. The partnerships are expected to generate several new clinical development drug candidates in key therapeutic areas of oncology, neurology and immunology. Under the terms of the agreements, three potential targets have been selected to initiate each partnership, with the potential of identifying and nominating additional targets in the future. The collaborations will focus on advancing small-molecule development candidates, which Merck will select for further preclinical and clinical development. As part of the agreements, both UK-based companies will receive upfront payments from Merck and will be eligible for future milestone payments plus tiered royalties based on net sales. Exscientia will receive an upfront payment of $20m and up to $674m in milestone payments if all three projects meet their objectives. Meanwhile, BenevolentAI is set ...
- Source: drugdu
- 106
- October 1, 2023
MIT study reveals why certain immunotherapies do not always work

A new Massachusetts Institute of Technology (MIT) study has revealed a potential explanation as to why certain immunotherapies for cancerous tumours do not always work as predicted. If validated in clinical trials, the findings could help doctors identify cancer patients who would benefit the most from drugs known as checkpoint blockade inhibitors. Checkpoint blockade inhibitors work by stimulating immune cells to destroy tumours in the body’s T cell response. In previous studies, findings have shown that these drugs work effectively in patients whose tumours have a large number of mutated proteins. However, 50% of patients who received the US Food and Drug Administration-approved checkpoint blockade inhibitor, pembrolizumab, did not respond well or only showed short-lived responses, despite their tumours showing high mutational burden. In a study of mice, the researchers revealed that measuring the diversity of mutations within a tumour generated a more accurate prediction as to whether immunotherapy treatment ...
- Source: drugdu
- 109
- October 1, 2023
Kezar and Everest enter lupus nephritis therapy development deal

Kezar Life Sciences and Everest Medicines have signed a partnership and licence agreement for the development and commercialisation of zetomipzomib for lupus nephritis (LN) in select territories. The territories covered under the agreement include Greater China, South Korea and South East Asia. As per the deal terms, Kezar is eligible to receive up to $132.5m in initial upfront and milestone payments contingent on meeting development, regulatory and commercialisation goals in the future. Kezar will also receive tiered royalty payments on net product sales from Everest. Everest will obtain sole rights for developing and marketing zetomipzomib in regions such as Greater China, Indonesia, Malaysia, the Philippines, Singapore, South Korea, Thailand and Vietnam. Everest will also collaborate with Kezar for the Phase IIb PALIZADE clinical trial of zetomipzomib, an immunoproteasome inhibitor of Kezar, in active LN patients in Greater China, South Korea and South East Asia. In the licensed territories, Everest will ...
- Source: drugdu
- 157
- October 1, 2023
Ozempic, Wegovy drug prescriptions hit 9 million, surge 300% in under three years

U.S. health care providers wrote more than nine million prescriptions for Ozempic, Wegovy and similar diabetes and obesity drugs during the last three months of 2022, according to a new analysis released Wednesday. The report, from analytics firm Trilliant Health, shows that quarterly prescriptions for those drugs increased 300% between early 2020 and the end of last year. Novo Nordisk ’s weekly diabetes injection Ozempic accounted for more than 65% of total prescriptions as of the end of 2022, and was primarily prescribed off-label for its ability to help patients lose weight. The data further confirms the rise in demand for that group of drugs, which have fueled a frenzy among Americans and on Wall Street for their ability to cause significant weight loss. Those treatments, known as GLP-1s, mimic a hormone in the gut to suppress a person’s appetite. But the rate of future prescription volumes will largely depend ...
- Source: drugdu
- 111
- September 30, 2023

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