Resultat från en fas III-studie för behandling av agitation hos patienter med Alzheimers demens visade att patienter behandlade med brexpiprazole hade signifikant större minskning jämfört med placebo.
Läs hela pressmeddelandet på engelska här
Minskad agitation visad med brexpiprazole i fas III vid Alzheimers demens
Namninsamling för jämlik vård överlämnas till Socialutskottets ordförande
Tisdagen den 21 juni överlämnade Nätverket för Spinal muskelatrofi (NSMA) en namninsamling till Socialutskottets ordförande Acko Ankarberg Johansson för att uppmärksamma att patienter med spinal muskelatrofi (SMA) nekas livsavgörande läkemedel i Sverige. Insamlingen har nått cirka 9500 underskrifter.
Under våren har en namninsamling med anledning av att patienter med spinal muskelatrofi (SMA) nekas effektiva och godkända läkemedel i Sverige ägt rum.
Sedan läkemedel som bromsar upp sjukdomsförloppet för patienter med spinal muskelatrofi (SMA) introducerades år 2017, har medicinen distribuerats till patienter i majoriteten av de europiska länder utan åldersbegränsningar. Men i Sverige får endast de tillgång till medicin som var under 18 år när behandlingarna introducerades, eftersom det annars skulle bli för dyrt.
Därmed nekas svenska SMA-patienter effektiva och godkända läkemedel trots att de förlänger liv och ökar livskvaliteten. Främsta argumentet från regeringen och regionernas arbetsgrupp NT-rådet är att behandlingarna anses kosta för mycket.
– Att som 24-åring nekas medicin som skulle förenkla, förbättra och förlänga mitt liv tvingar verkligen fram frågan om vad ett liv egentligen är värt. Att behöva fråga sig det är inte värdigt eller etiskt försvarbart i ett välfärdsland som Sverige, säger Linnéa Svensson, som själv har SMA och har tagit initiativ till namninsamlingen.
– Så länge som tillgång till behandling grundas på ålder i stället för behov kan Sverige knappast anses vara ett land som eftersträvar jämlik vård. Socialutskottet kan se till att dagens orättvisa upphör. De kan se till att sätta press på regeringen och att människor som behöver läkemedel också får läkemedel som förlänger liv. Därför är det mycket glädjande att så många skrivit under insamlingen och att socialutskottets ordförande tagit emot den, säger Pernilla Becker, ordförande för Nätverket för spinal muskelatrofi.
Insamlingen lämnades till Socialutskottets ordförande Acko Ankarberg Johansson av Daniella Ås, en av initiativtagarna till namninsamlingen som själv lever med SMA.
För ytterligare information kontakta:
Pernilla Becker, ordförande NSMA; tel: 073 848 14 43.
Rapport från internationell MG-konferens
Anna Rostedt Punga, professor och överläkare vid Uppsala Universitet och Akademiska sjukhuset har för Neuros medlemmar skrivit ett nyhetsbrev om myastenia gravis (MG). Hon summerar vad som presenterades på den internationella MG-konferensen i Miami 10-12 maj 2022.
Konferensen anordnas av Myasthenia Gravis Foundation of America och Anna Rostedt Punga var med och organiserade den. Hittills har den anordnats var femte år. På konferensen beslutades att detta är lite för långt tidsintervall så nästa konferens kommer att anordnas redan om tre år.
Crenezumab stoppade eller bromsade inte kognitiv försämring hos patienter med en specifik gen som orsakar tidig Alzheimer
Roche tillsammans med Banner Alzheimer’s Institute, meddelade idag resultaten från Alzheimer’s Prevention Initiative (API) Autosomal Dominant Alzheimer’s Disease (ADAD) Colombia Trial. Studien utvärderad potentialen för Crenezumab möjligheter att stoppa eller bromsa utvecklingen av tidig Alzheimer hos de som har en specifik genmutation för just detta.
Small numerical differences favouring crenezumab were observed across the co-primary and multiple secondary and exploratory endpoints, but these were not statistically significant. No new safety issues were identified with crenezumab during the study. Further analyses of data are ongoing. Initial data will be presented at the Alzheimer’s Association International Conference (AAIC) on August 2, 2022.
“We’re disappointed that the treatment did not demonstrate a statistically significant clinical benefit,” said Eric M. Reiman, M.D., Banner Alzheimer’s Institute executive director and one of the study leaders. “At the same time, we’re proud of the impact that this precedent-setting trial has had in shaping a new era in Alzheimer’s prevention research and we’re extremely grateful to our research participants and their families. This trial, the data, samples and findings that we’ll share with the research community, and the related work that we and others are doing promise to further accelerate the evaluation and approval of future prevention therapies.”
The trial enrolled 252 people who are members of the world’s largest extended family with ADAD in Colombia, with 94% of participants completing the study. Two-thirds of participants carried the Presenilin 1 E280A mutation which typically causes cognitive impairment due to Alzheimer’s disease around age 44. Participants were randomised to receive crenezumab, an investigational treatment discovered by AC Immune SA, or placebo over five to eight years. During the trial, the dose of crenezumab was increased as knowledge about potential treatment approaches for Alzheimer’s disease evolved.
“While this is a disappointing result, we would like to thank the participants and their families – they have made an enormous contribution to advancing both understanding and the search for new treatments for familial Alzheimer’s disease,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. “We remain committed to contributing further scientific evidence to advance how Alzheimer’s disease is understood, diagnosed and treated.”
The study, which was supported by the National Institute on Aging, generous philanthropic contributions to Banner Alzheimer’s Foundation, and Roche, has generated a wealth of data that will advance the early detection, tracking and study of Alzheimer’s disease and inform the design of future Alzheimer’s prevention trials.
Within its Alzheimer’s pipeline, Roche is also evaluating the potential of gantenerumab in autosomal dominant Alzheimer’s disease, as well as for the prevention of sporadic Alzheimer’s and treatment of early Alzheimer’s in late stage clinical trials. Results from the phase III GRADUATE studies of gantenerumab in early Alzheimer’s are expected in Q4, 2022.
About Banner Alzheimer’s Institute
Since its inception in 2006, Banner Alzheimer’s Institute (BAI) has sought to find effective Alzheimer’s disease prevention therapies without losing another generation, establish a new model of dementia care for patients and family caregivers, and forge new models of collaboration in biomedical research. It has made groundbreaking contributions to the unusually early detection, tracking, diagnosis and study of Alzheimer’s, and aims to find an effective prevention therapy by 2025. It includes the pioneering Alzheimer’s Prevention Initiative (API), an extensive profile of research studies and clinical trials, comprehensive clinical, family and community service programs, a leading brain imaging research program, and strategic partnerships with numerous public and private research organisations around the world.
About the Alzheimer’s Prevention Initiative (API) and the API ADAD (Colombia) Trial
The Alzheimer’s Prevention Initiative (API) is an international collaborative formed in 2009 to launch a new era of Alzheimer’s prevention research. Led by the Banner Alzheimer’s Institute, the API conducts prevention trials in cognitively healthy people at increased risk for Alzheimer’s disease. API continues to establish brain imaging, fluid biomarker and cognitive endpoints needed to rapidly test promising prevention therapies. It also leads participant recruitment registries to accelerate enrollment into Alzheimer’s-focused studies. API is intended to provide the scientific means, accelerated approval pathway and enrollment resources needed to evaluate the range of promising Alzheimer’s prevention therapies and find ones that work without losing another generation.
First proposed by investigators from BAI, the API ADAD trial (NCT01998841) was a prospective, randomised, double-blind, placebo-controlled, parallel-group label enabling Phase II study of the efficacy of crenezumab versus placebo in cognitively unimpaired individuals who have no clinical symptoms of Alzheimer’s disease and carry the PSEN1 E280A autosomal dominant mutation. Participants who are mutation carriers were randomised in a 1:1 ratio to receive either crenezumab or placebo for at least 260 weeks. Crenezumab was initially administered subcutaneously 300 mg every two weeks. Dosing was amended in 2015 to 720 mg subcutaneously every two weeks and in 2019 the option to increase the dose to 60 mg/kg, delivered intravenously every four weeks, was offered to participants. A cohort of participants (non-mutation carriers) were also enrolled and dosed solely on placebo.
The trial, which was supported by National Institute on Aging (NIA) generous philanthropic contributions to Banner Alzheimer’s Foundation and Roche, was the first NIH-supported prevention trial of an experimental prevention therapy in cognitively unimpaired persons at known risk for the disease.
For more information, go to https://alzheimerspreventioninitiative.com/.
Dosering av natalizumab var sjätte vecka istället för var fjärde för personer med skovvis förlöpande multipel skleros
Lancet Neurology publicerar resultat från fas 3b NOVA-studien som utvärderar effekten av dosering av natalizumab var sjätte vecka för personer med skovvis förlöpande multipel skleros.
- Resultaten av NOVA-studien visar att natalizumab IV som administreras var sjätte vecka kontrollerar sjukdomsaktiviteten hos MS-patienter som bytte från den godkända doseringsregimen var fjärde vecka
- Säkerhetsresultaten överensstämde med den redan kända säkerhetsprofilen för natalizumab IV
- Europeiska läkemedelsmyndigheten har nyligen lagt till effekt- och säkerhetsdata för IV administrering var sjätte vecka i produktresumén för TYSABRI™ (natalizumab) i EU
Nyheten på engelska:
Biogen Inc. (Nasdaq: BIIB) can announce that data from the NOVA Phase 3b study evaluating the efficacy of every six-week (Q6W) dosing with 300 mg natalizumab IV were published in The Lancet Neurology. The data show that Q6W dosing with natalizumab controls multiple sclerosis (MS) disease activity in patients with relapsing-remitting MS who switched to Q6W after at least one year of disease stability on the approved every four-week (Q4W) IV dosing schedule.
“The NOVA trial is the first prospective study evaluating every six-week dosing of natalizumab in patients with relapsing-remitting MS, providing important information about the consistent effect of every four- or six-week dosing,” said John Foley, M.D., Rocky Mountain MS Clinic and lead author of The Lancet Neurologypublication. “These results build upon the retrospective safety analyses of the TOUCH Prescribing Program, which found that extended interval dosing of approximately every six weeks with IV natalizumab is associated with significantly lower risk of progressive multifocal leukoencephalopathy (PML) than the every four-week dosing schedule, to provide a more comprehensive understanding of the every six-week dosing regimen for natalizumab.”
Topline results from the NOVA study were first announcedin August 2021, and new data published in The Lancet Neurology include additional information on baseline characteristics of the trial, as well as safety outcomes.
“The publication of peer-reviewed NOVA data in The Lancet Neurology highlights Biogen’s commitment to pursue scientific research and innovation on behalf of the MS community, affirming the real-world evidence reports from the clinical community about every six-week dosing of natalizumab1,2,3,” said Christina Gip, medical director, Biogen in Sweden. “Biogen will continue to evaluate the data generated by the NOVA study to further advance our understanding of natalizumab, an MS therapy with 15 years of market experience resulting in more than one million patient years of experience5.”
The NOVA study (NCT03689972) was designed to assess the efficacy of Q6W dosing with natalizumab IV administration following analyses from the TOUCH (Tysabri Outreach: Unified Commitment to Health) Prescribing Program, which showed that extended interval dosing (approximately every six weeks) was associated with a significant 87 percent reduction (hazard ratio 0.127; P<0.0001) in the probability of PML in comparison to the approved Q4W dose.4 NOVA was a randomized, controlled, prospective, open-label, multinational trial including participants diagnosed with MS aged 18-60 years who had been treated with the approved dose of natalizumab IV (300 mg every four weeks) with no relapses for at least 12 months. Study participants (n=499) were randomly assigned across 89 study sites in 11 countries to either receive Q6W dosing (n=251) or continue treatment with Q4W dosing (n=248). The primary endpoint was the mean number of new or newly enlarging T2 hyperintense lesions at week 72. Secondary study clinical endpoints were assessment of the time to first relapse, annualized relapse rate (ARR) at week 72 and time to 24-week confirmed disability worsening (CDW) (defined as an increase of ≥1 point from a baseline EDSS score of ≥1 or an increase of ≥1.5 points from a baseline EDSS score of 0, confirmed ≥24 weeks after the initial increase).
There was a numerical but not statistically significant difference in the primary efficacy endpoint between the Q4W and Q6W treatment arms, the mean number of new or newly enlarging T2 hyperintense lesions at week 72 of 0.05 (Q4W) and 0.20 (Q6W) (p=0.0755). There were no statistically significant or clinically meaningful differences in secondary endpoints at week 72 between the Q4W and Q6W treatment arms, and disease activity was well-controlled in both arms: annualized relapse rates were low at 0.00010 (Q4W) and 0.00013 (Q6W), with 97.9 percent of patients in the Q4W arm remaining relapse-free compared to 97.2 percent of patients in the Q6W arm.
The safety findings in the NOVA study were consistent with the known safety profile of IV natalizumab, and the incidence of serious adverse events (SAEs) and adverse events (AEs) was similar between the two treatment arms. AEs leading to discontinuation of study treatment were reported by four of 250 (2%) Q6W participants and one of 247 (0.4%) Q4W participants. NOVA was not powered to assess differences in the probability of PML for Q6W and Q4W dosing. The NOVA study protocol includes an open-label extension (OLE) study (NOVA Part 2) assessing the subcutaneous and IV routes of administration of natalizumab Q6W and exploring the long-term efficacy, safety, and tolerability of natalizumab Q6W.
The full manuscript, “Comparison of switching to 6-week dosing of natalizumab versus continuing with 4-week dosing in patients with relapsing-remitting multiple sclerosis (NOVA): a controlled, open-label, phase 3b trial,” is available onlineand will appear in the July print issue of The Lancet Neurology.
In March 2022, the TYSABRITM (natalizumab) Summary of Product Characteristics (SmPC) was updated by the European Medicines Agency (EMA) to include data on the efficacy of Q6W IV dosing from NOVA while maintaining Q4W as the only approved dosing regimen.
