Latest news with #IonisPharmaceuticals
Yahoo
23-05-2025
- Health
- Yahoo
This experimental drug could help young people with a rare and aggressive form of ALS
An experimental drug could help young people with a rare form of ALS, researchers at New York's Columbia University Irving Medical Center announced on Thursday. Known as 'ulefnersen,' the therapy showed promise in treating patients with FUS-ALS, which is caused by a genetic mutation in a gene called FUS. The gene provides instructions for making a protein and plays a crucial role in cellular processes. While the mutations are only responsible for between one and two percent of ALS cases, they cause some of the most aggressive forms of ALS that begin in adolescents and young adults. Fewer than 30,000 people in the U.S. are living with ALS, which is also known as amyotrophic lateral sclerosis or Lou Gehrig's disease, while 5,000 people are diagnosed each year. The nervous system disease affects nerve cells in the brain and spinal cord, leading to a loss of muscle control that gets progressively worse. The exact cause of the disease remains unknown, but a small number of cases are genetic. There is no cure and it is, eventually, fatal. 'When testing new drugs for ALS, we do not expect to see clinical improvement,' neurologist and scientist Neil Shneider said in a statement. 'What we've seen in one patient is really unprecedented functional recovery. It's surprising and deeply motivating for us, the ALS research community, but also the community of ALS patients.' Shneider, who developed the drug in collaboration with the biotech company Ionis Pharmaceuticals, published his findings in the peer-reviewed journal The Lancet. Starting as an effort to help a single patient, the case series was small, including just 12 patients who were treated with the drug. Two of the patients showed a remarkable response to the drug. A young woman who had received injections of ulefnersen since late 2020 regained the ability to walk unaided and to breathe without the use of a ventilator, both previously lost to ALS. The medical center said she has lived longer with this disease than any other known patient with this juvenile-onset form of FUS ALS. A man in his mid-thirties was asymptomatic when he began treatment, but tests of electrical activity in his muscles indicated that symptoms would likely emerge imminently. However, after three years of treatment, he has yet to develop FUS-ALS symptoms and the abnormal electrical activity in his muscles has improved. Lastly, the researchers found patients experienced an up to 83 percent decrease in a protein called neurofilament light — a biomarker of nerve damage — following six months on the drug. 'These responses show that if we intervene early enough and go after the right target at the right time in the course of disease, it's possible to not only slow disease progression, but actually reverse some of the functional losses,' said Shneider. 'It's also a wonderful example of precision medicine and therapeutic development based on science and an understanding of the biology of disease.' While most of the other symptomatic patients did not survive their disease, Shneider said that 'several apparently benefited from the treatment. The progression of their disease slowed, and they lived a longer life as a consequence.' There were no serious adverse events related to the drug and a global clinical trial is now in progress. At least 25 patients have been treated with the drug around the world, including the dozen in Shneider's case studies. It was first tested six years ago in an Iowa patient named Jaci Hermstad. The drug was originally named for her. 'Now we are eagerly awaiting those results, which we hope will lead to the approval of ulefnersen,' Shneider said.
Hans India
23-05-2025
- Health
- Hans India
Experimental drug shows promise of treatment for young patients with rare form of ALS
Treatment with an experimental drug has shown significant improvements in young patients with a rare form of Amyotrophic Lateral Sclerosis (ALS) -- a progressive neurodegenerative disorder, said a team of US researchers on Friday. ALS, also known as Lou Gehrig's disease, is a rare disorder that affects nerve cells in the brain and spinal cord, leading to the loss of motor neurons causing difficulty with movement, balance, coordination, and potentially even breathing. While experimental therapies have so far slowed down the disease or halted its progression, the new treatment using ulefnersen (previously known as jacifusen) -- showed that functional losses in young patients can be reversed. "When testing new drugs for ALS, we do not expect to see clinical improvement," said neurologist and scientist Neil Shneider at Columbia University. But, "what we've seen in one patient is really unprecedented functional recovery. It's surprising and deeply motivating for us, the ALS research community, but also the community of ALS patients," he added. Data from 12 patients -- all treated with the novel therapy for a rare form of ALS caused by a genetic mutation in a gene called FUS -- were presented in a case series published by Shneider online in The Lancet. Though these gene mutations are responsible for only 1-2 per cent of ALS cases, they cause some of the most aggressive forms of ALS that begin in adolescents and young adults. In patients with these mutations, toxic FUS proteins accumulate in the motor neurons that control the patient's muscles, eventually killing the neurons. Two of the patients in the published case series showed a remarkable response to the experimental therapy, ulefnersen developed by Shneider in collaboration with California-based Ionis Pharmaceuticals. One young woman, who has received injections of the therapy since late 2020, recovered the ability to walk unaided and to breathe without the use of a ventilator, both previously lost to ALS. She has lived longer with this disease than any other known patient with this juvenile-onset form of FUS ALS. The second patient, a man in his mid-30s, was asymptomatic when he began treatment, but tests of electrical activity in his muscles indicated that symptoms would likely emerge soon. In three years of continuous treatment with the experimental drug, the man has yet to develop any symptoms of FUS-ALS and the abnormal electrical activity in his muscles has improved. Overall, after six months of treatment, patients in the series experienced up to an 83 per cent decrease in a protein called neurofilament light, a biomarker of nerve damage. "These responses show that if we intervene early enough and go after the right target at the right time in the course of the disease, it's possible to not only slow disease progression but actually reverse some of the functional losses," Shneider said. Though most of the other symptomatic patients in the series did not survive their aggressive disease, Shneider said "Several apparently benefited from the treatment. The progression of their disease slowed, and they lived a longer life as a consequence." The case series also showed that the drug is safe and well tolerated, with no serious adverse events related to the drug. Following the results from the first of these patients, a global clinical trial of the drug is now in progress. "Now we are eagerly awaiting those results, which we hope will lead to the approval of ulefnersen," Shneider said.
The Independent
22-05-2025
- Health
- The Independent
This experimental drug could help young people with a rare and aggressive form of ALS
An experimental drug could help young people with a rare form of ALS, researchers at New York's Columbia University Irving Medical Center announced on Thursday. Known as 'ulefnersen,' the therapy showed promise in treating patients with FUS-ALS, which is caused by a genetic mutation in a gene called FUS. The gene provides instructions for making a protein and plays a crucial role in cellular processes. While the mutations are only responsible for between one and two percent of ALS cases, they cause some of the most aggressive forms of ALS that begin in adolescents and young adults. Fewer than 30,000 people in the U.S. are living with ALS, which is also known as amyotrophic lateral sclerosis or Lou Gehrig's disease, while 5,000 people are diagnosed each year. The nervous system disease affects nerve cells in the brain and spinal cord, leading to a loss of muscle control that gets progressively worse. The exact cause of the disease remains unknown, but a small number of cases are genetic. There is no cure and it is, eventually, fatal. 'When testing new drugs for ALS, we do not expect to see clinical improvement,' neurologist and scientist Neil Shneider said in a statement. 'What we've seen in one patient is really unprecedented functional recovery. It's surprising and deeply motivating for us, the ALS research community, but also the community of ALS patients.' Shneider, who developed the drug in collaboration with the biotech company Ionis Pharmaceuticals, published his findings in the peer-reviewed journal The Lancet. Starting as an effort to help a single patient, the case series was small, including just 12 patients who were treated with the drug. Two of the patients showed a remarkable response to the drug. A young woman who had received injections of ulefnersen since late 2020 regained the ability to walk unaided and to breathe without the use of a ventilator, both previously lost to ALS. The medical center said she has lived longer with this disease than any other known patient with this juvenile-onset form of FUS ALS. A man in his mid-thirties was asymptomatic when he began treatment, but tests of electrical activity in his muscles indicated that symptoms would likely emerge imminently. However, after three years of treatment, he has yet to develop FUS-ALS symptoms and the abnormal electrical activity in his muscles has improved. Lastly, the researchers found patients experienced an up to 83 percent decrease in a protein called neurofilament light — a biomarker of nerve damage — following six months on the drug. 'These responses show that if we intervene early enough and go after the right target at the right time in the course of disease, it's possible to not only slow disease progression, but actually reverse some of the functional losses,' said Shneider. 'It's also a wonderful example of precision medicine and therapeutic development based on science and an understanding of the biology of disease.' While most of the other symptomatic patients did not survive their disease, Shneider said that 'several apparently benefited from the treatment. The progression of their disease slowed, and they lived a longer life as a consequence.' There were no serious adverse events related to the drug and a global clinical trial is now in progress. At least 25 patients have been treated with the drug around the world, including the dozen in Shneider's case studies. It was first tested six years ago in an Iowa patient named Jaci Hermstad. The drug was originally named for her. 'Now we are eagerly awaiting those results, which we hope will lead to the approval of ulefnersen,' Shneider said.
Yahoo
22-05-2025
- Health
- Yahoo
Ionis reports success in olezarsen trial for high triglycerides
Ionis Pharmaceuticals has reported positive topline results from the ESSENCE study evaluating olezarsen in individuals with moderate hypertriglyceridemia who are at risk of atherosclerotic cardiovascular disease (ASCVD). The study met its primary endpoint, demonstrating a statistically significant, placebo-adjusted reduction in triglyceride levels of 61% and 58% at six months with 80mg and 50mg monthly doses respectively (p < 0.0001). Olezarsen also met all key secondary endpoints, with the majority of participants achieving triglyceride levels below 150mg/dL, bringing them into the normal range. Olezarsen was well tolerated, with a favourable safety profile consistent with previous studies. Olezarsen is an antisense oligonucleotide (ASO) designed to selectively inhibit the expression of the APOC3 gene. By decreasing the production of apolipoprotein C-III (ApoC-III) — a protein that hinders triglyceride metabolism — it promotes the efficient breakdown and clearance of triglyceride-rich lipoproteins. This leads to a marked reduction in plasma triglyceride levels, positioning olezarsen as a promising treatment for severe hypertriglyceridemia, familial chylomicronemia syndrome (FCS) and other dyslipidemia. Key opinion leaders (KOLs) interviewed by GlobalData emphasised a significant unmet need for effective treatments targeting rare genetic lipid disorders, particularly FCS and familial hypercholesterolemia. Notably, there are currently no FDA-approved therapies for FCS, underscoring the urgency for new options that can lower triglyceride levels and mitigate associated complications. With its targeted mechanism and encouraging data, olezarsen has the potential to address this therapeutic gap. According to GlobalData, the drug is projected to achieve global sales of around $849 million by 2032. Pivotal Phase III results from the ongoing CORE and CORE2 trials, which are evaluating olezarsen for the treatment of severe hypertriglyceridemia (sHTG), are expected in the third quarter of 2025. This data is highly anticipated and will be crucial in determining the drug's efficacy and safety profile. Positive results could pave the way for regulatory approval and establish olezarsen as a first-in-class treatment option for patients with limited alternatives. "Ionis reports success in olezarsen trial for high triglycerides" was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
20-05-2025
- Health
- Yahoo
Ionis reports positive data from study of olezarsen for hypertriglyceridemia
Ionis Pharmaceuticals has reported positive topline outcomes from the randomised, global Phase III Essence study of Tryngolza (olezarsen) in individuals with moderate hypertriglyceridemia and, or at risk for, atherosclerotic cardiovascular disease (ASCVD). This condition is characterised by fasting triglyceride (TG) levels between ≥150mg/dL and <500mg/dL. The multicentre, double-blind, placebo-controlled Essence study was carried out with the TIMI Study Group, the company's research partner. It enrolled 1,478 subjects aged 18 years and above diagnosed with or at risk for ASCVD, and 9% of them had fasting triglycerides of ≥500mg/dL at baseline. Subjects received either 50mg or 80mg of olezarsen or a placebo every four weeks via subcutaneous injection for 12 months. The primary endpoint focused on the percentage change from baseline in fasting TG levels at the six-month mark, compared to the placebo. Key secondary endpoints were changes in TG levels at 12 months, the proportion of subjects achieving fasting TG levels of <150mg/dL, and the percentage variations in other lipid parameters against the placebo over the treatment period. The trial, which included subjects already on standard of care, lipid-lowering medications, achieved its primary endpoint. A significant decrease in TG levels was observed, with a placebo-adjusted 61% decrease at the 80mg monthly dose and a 58% decrease at the 50mg dose over a six-month period, compared to the placebo. In addition, the therapy met all key secondary endpoints, with the majority of subjects achieving normal TG levels of <150mg/dL. Olezarsen also demonstrated a favourable tolerability and safety profile in the trial. Ionis Pharmaceuticals global cardiovascular development senior vice-president Sam Tsimikas said: 'Following the Food and Drug Administration (FDA) approval and encouraging launch of Tryngolza (olezarsen) for people living with familial chylomicronaemia syndrome (FCS), a rare, genetic form of severely elevated TGs, these data support olezarsen's potential tobenefit the much broader population of people living with severe hypertriglyceridemia (sHTG).' The investigational RNA-targeted medicine is designed to decrease the body's apolipoprotein C III (apoC-III) production, a protein for regulating the metabolism of triglyceride in the blood. Last November, Ionis announced the design for a pivotal Phase III trial of ION582, an investigational treatment for Angelman syndrome (AS), following discussions with the US FDA. "Ionis reports positive data from study of olezarsen for hypertriglyceridemia" was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site.
