Latest news with #RichardFinkel
Associated Press
27-06-2025
- Health
- Associated Press
New Data for Nusinersen Underscore Biogen's Commitment to Advancing Clinical Research to Improve Outcomes in SMA
CAMBRIDGE, Mass., June 27, 2025 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) today announced new data that reinforce the clinical impact of nusinersen across a broad spectrum of individuals affected by spinal muscular atrophy (SMA). These latest findings from Part C of the DEVOTE trial evaluating a higher dose regimen of nusinersen and the NURTURE trial which evaluated the approved 12 mg regimen (SPINRAZA®) in clinically presymptomatic SMA were presented at the SMA Research & Clinical Care Meeting hosted by Cure SMA in Anaheim, Calif. Biogen's applications for the higher dose regimen of nusinersen are currently under review in the U.S., Europe, Japan and other global markets. The higher dose regimen of nusinersen comprises a more rapid loading regimen – two 50 mg doses 14 days apart – and a higher maintenance regimen – 28 mg every four months. 'As the SMA treatment landscape continues to evolve, we remain steadfast in our commitment to address the unmet needs of the community. The findings from Part C of the DEVOTE study further strengthen the growing body of evidence supporting the potential benefits of the higher dose regimen of nusinersen,' said Stephanie Fradette, Pharm.D., Head of the Neuromuscular Development Unit at Biogen. Improvements Observed With Higher Dose Regimen of Nusinersen in Previously Treated Patient Population Detailed results from Part C of the DEVOTE study highlight the potential clinical benefits of an investigational higher dose of nusinersen in a broad range of individuals (n=38) who had been previously treated with nusinersen at the approved 12 mg dose for approximately four years (median: 3.9 years). Participants were 4 to 65 years of age and half (n=19) were ambulatory. Participants in Part C received one loading dose (50 mg) and two maintenance doses (28 mg each) of open-label higher dose nusinersen during the study period. Most participants experienced improvements on the Hammersmith Functional Motor Scale – Expanded (HFMSE), Revised Upper Limb Module (RULM), and/or Clinical Global Impression of Change (CGI-C; assessed by investigator or caregiver) after transitioning to the higher dose regimen. These improvements were observed across phenotypes, functional status and age. For example, non-ambulatory participants improved by +2.5 (95% CI: 0.49, 4.56) on average on the HFMSE, and ambulatory participants improved by +1.1 (95% CI: -0.68, 2.89). 'These emerging data indicate that additional gains in function might be possible even in those with established disease who have been on therapy for years,' said Richard Finkel, M.D., director, Center for Experimental Neurotherapeutics (CENT) at St. Jude Children's Research Hospital. 'This effort to optimize the dosing of SPINRAZA is very exciting for the field and could fundamentally change how we treat our patients.' The safety profile of the higher dose regimen of nusinersen is broadly consistent with the known safety profile of 12 mg SPINRAZA. In the DEVOTE study overall, reported adverse events (AEs) included pneumonia, respiratory failure, pyrexia, COVID, upper respiratory tract infection, procedural pain and procedural headache. In Part C (n=40), AEs were reported in 37/40 participants, the majority of which were mild or moderate in severity. Serious AEs were reported by six participants (15%), none of which were considered by the investigator to be related to study treatment or administration. Final NURTURE Data Redefine Expectations for Early Treatment Final data from the eight-year, open-label NURTURE study highlight the impact of early intervention with 12 mg SPINRAZA in clinically presymptomatic infants with SMA. At the study conclusion, all children who participated in NURTURE (n=25) were alive and experienced continued clinical benefits over the course of the study. No participants required permanent ventilation, and the majority (20 of 25 participants) went without any ventilatory support throughout the study. Ninety-two percent of participants achieved the ability to walk independently, many within normal developmental timeframes. Participants with elevated levels of neurofilament light chain (NfL) at baseline experienced rapid and sustained reductions in NfL after initiation of nusinersen, reinforcing the potential utility of NfL as an objective biomarker of disease activity and treatment response in SMA. Nusinersen was generally well tolerated with no new safety concerns identified with eight years of follow-up. All participants had at least one AE, the majority of which were mild to moderate in severity; no AEs led to treatment discontinuation or study withdrawal. About SPINRAZA SPINRAZA (nusinersen) 12 mg/5 mL injection is approved in more than 71 countries to treat infants, children and adults with spinal muscular atrophy (SMA). As a foundation of care in SMA, more than 14,000 individuals have been treated with SPINRAZA worldwide.1 The currently approved 12 mg regimen for SPINRAZA is comprised of four loading doses administered over approximately 60 days, followed by maintenance dosing every four months thereafter. SPINRAZA is an antisense oligonucleotide (ASO) that targets the underlying cause of motor neuron loss by continuously increasing the amount of full-length survival motor neuron (SMN) protein produced in the body.2 It is administered directly into the central nervous system, where motor neurons reside, to deliver treatment where the disease starts.2 SPINRAZA has shown sustained efficacy across ages and SMA types with a well-established safety profile based on data in patients treated up to 10 years,3,4 combined with unsurpassed real-world experience. The nusinersen clinical development program encompasses more than 10 clinical studies, which have included more than 460 individuals across a broad spectrum of patient populations, including two randomized controlled studies (ENDEAR and CHERISH). The most common adverse events observed in clinical studies were respiratory infection, fever, constipation, headache, vomiting and back pain. Laboratory tests can monitor for renal toxicity and coagulation abnormalities, including acute severe low platelet counts, which have been observed after administration of some ASOs. Biogen licensed the global rights to develop, manufacture and commercialize SPINRAZA from Ionis Pharmaceuticals, Inc. (Nasdaq: IONS). Please click here for Important Safety Information and full Prescribing Information for SPINRAZA in the U.S., or visit your respective country's product website. About Biogen Founded in 1978, Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patients' lives and to create value for shareholders and our communities. We apply deep understanding of human biology and leverage different modalities to advance first-in-class treatments or therapies that deliver superior outcomes. Our approach is to take bold risks, balanced with return on investment to deliver long-term growth. We routinely post information that may be important to investors on our website at Follow us on social media - Facebook, LinkedIn, X, YouTube. Biogen Safe Harbor This news release contains forward-looking statements, including related to the potential clinical effects of a higher dose regimen of nusinersen; the potential benefits, safety and efficacy of higher dose regimen of nusinersen; the clinical development program for higher dose regimen of nusinersen; the identification and treatment of SMA; our research and development program for the treatment of SMA; the potential of our commercial business and pipeline programs, including SPINRAZA; and risks and uncertainties associated with drug development and commercialization. These forward-looking statements may be accompanied by words such as 'aim,' 'anticipate,' 'believe,' 'could,' 'estimate,' 'expect,' 'forecast,' 'intend,' 'may,' 'plan,' 'potential,' 'possible,' 'will,' 'would' and other words and terms of similar meaning. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Results in early-stage clinical trials may not be indicative of full results or results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on our forward-looking statements. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including without limitation, uncertainty of success in the development and potential commercialization of higher dose regimen of nusinersen; the risk that we may not fully enroll our clinical trials or enrollment will take longer than expected; unexpected concerns may arise from additional data, analysis or results obtained during our clinical trials; regulatory authorities may require additional information or further studies, or may fail or refuse to approve or may delay approval of our drug candidates, including SPINRAZA; the occurrence of adverse safety events; the risks of unexpected hurdles, costs or delays; failure to protect and enforce our data, intellectual property and other proprietary rights and uncertainties relating to intellectual property claims and challenges; product liability claims; results of operations and financial condition. The foregoing sets forth many, but not all, of the factors that could cause actual results to differ from our expectations in any forward-looking statement. Investors should consider this cautionary statement, as well as the risk factors identified in our most recent annual or quarterly report and in other reports we have filed with the U.S. Securities and Exchange Commission. These statements speak only as of the date of this news release. We do not undertake any obligation to publicly update any forward-looking statements. References:
Los Angeles Times
12-06-2025
- Business
- Los Angeles Times
Commission's denial of home ownership project reveals cracks in Costa Mesa's zoning
In Costa Mesa, where 60% of the populace are renters and home prices make purchases prohibitive, city leaders have repeatedly expressed a desire to see more home ownership projects. But at what cost? That issue came to a head this week as the city's planning commission considered a proposal for Victoria Place, a 40-unit condominium complex at 220 Victoria St., a 1.77-acre commercial lot situated just west of Newport Boulevard. A hearing Monday followed an August 2024 screening by the City Council, during which officials pressed for more common areas, open space and better landscaping but had no qualms about the project's density or location. Put forth by Newport Beach-based WMC, LLC., the proposal features 18 duplexes and four detached residences, each comprising roughly 2,700 square feet in three stories with a ground-floor workspace, two-car garage, balcony and rooftop deck at a maximum height of 39 feet, 6 inches. The property lies close enough to Newport Boulevard to qualify for rezoning under Measure K, a 2022 voter-backed initiative that makes allowances for high-density residences near certain commercial and industrial corridors. Because the requisite rezoning has not been completed, the developers of Victoria Place, with help from city planners, have had to get creative. The project was pitched to commissioners for inclusion in a residential incentive overlay district created in 2016 to allow for the redevelopment of 14 identified commercial properties along Newport and Harbor boulevards for high-density residential uses. None of the property owners took advantage of the designation and, two years later, city leaders reduced eligibility to just four sites. Victoria Place seeks designation as a fifth parcel and, if built, would be the first site developed under the overlay's more permissive building standards. However, applicants came to Monday's hearing asking for even more concessions than the overlay allows, namely a reduction in side and rear setbacks, garages and on-site parking stalls narrower by 6 inches, less off-street parking and a reduction in required common open space. 'We feel that Costa Mesa's need for this type of housing is pretty clear. And [this] offers a thoughtful and well-designed response to that need,' Tony Weeda, managing partner of WMC, LLC. told commissioners. He explained architects incorporated a barbecue and play area into the site plans, along with a flexible-use space that can be used for events and as a fire lane. The gated complex would allow children to play safely on the property. Architect Richard Finkel said the city encouraged the applicants to fit as much housing as the overlay allowed. Commissioners, however, were not convinced the multiplicity of concessions were appropriate for the space and expressed concerns about the traffic vehicles and pedestrians would face so close to Newport Boulevard. They further lamented the city's delay in rezoning Measure K properties. 'We still don't have a cohesive plan for what we expect from our Measure K sites,' said Commissioner David Martinez. 'Now, we get stuck in this weird situation where we're applying something for the very first time and trying to figure out what any of it means — it just sucks.' Commissioner Rob Dickson said it would be unfair to hold an applicant hostage because the city couldn't figure out how to zone a project, and pointed out the council already ostensibly approved the concept last August. 'If this was the first impression of the project before us, I would be 100% absolutely not; it's just too outside the scope,' he said. 'However, this has been going on for a while, and it seems to be something the council asked for.' Commissioners opposed recommending the project in a 5-2 vote, with Dickson and Commissioner Angely Andrade Vallarta opposed. It will move on to the City Council for its consideration.
Yahoo
21-02-2025
- Health
- Yahoo
This rare genetic disorder was just treated in the womb for the first time ever
Following the first-ever treatment for spinal muscular atrophy in the womb, physicians say a 2-year-old girl shows no signs of the rare genetic disorder. Spinal muscular atrophy is a genetic condition set in motion before birth that causes worsening muscle weakness. There are four types of the disorder, each with varying degrees of severity. However, for those with the most common and severe form, children typically do not live past age 2. There is no cure, but treatment can help to manage symptoms and prevent complications. The child's mother was given the drug risdiplam, which is the first oral medication approved to treat the progressive neurodegenerative disorder by the Food and Drug Administration. It is manufactured by Swiss biotech firm Roche. 'Our primary objectives were feasibility, safety and tolerability, so we're very pleased to see that the parent and child are doing well,' Dr. Richard Finkel, the director of the St. Jude Center for Experimental Neurotherapeutics, said in a statement. Scientists at St. Jude Children's Research Hospital led the first in utero treatment. 'The results suggest it would be worthwhile to continue investigating the use of prenatal intervention for SMA.' Finkel was the corresponding author of the research which was published Wednesday in a letter to the New England Journal of Medicine. Until now, treatments were given after birth. 'There was still room for improvement,' Finkel told Nature on Thursday. This progress came about by understanding the cause of spinal muscular atrophy. Approximately one in every 6,000 babies is born with the disorder. The research hospital noted in a release that it is caused by a lack of survival motor neuron protein. The protein is essential because it enables muscles to receive signals from the nerves, according to Johns Hopkins Medicine. The absence of the protein occurs in around one in every 11,000 births in the U.S. The drug works by helping produce more of the protein. Because the protein is most needed in the third trimester of fetal development and the first three months of life after birth, St. Jude's says that symptom severity is closely linked with intervention time. So, they launched the clinical protocol to study risdiplam in a single patient. The Food and Drug Administration approved the study. The parents of the patients had a prior infant born with Type 1 and were known carriers of genetic variants. Testing confirmed that their child would likely be born with Type 1. The idea of giving the drug in utero came from the parents, and doctors gave the mother the drug within the final six weeks of her pregnancy. After her birth, the baby was diagnosed with several abnormalities that are considered to have occurred before exposure to the drug. She started taking it at a week old and will likely continue to take it for the rest of her life. They are continuing to monitor her periodically at the research center. 'During the course of the assessment, we really have seen no indication of any signs of SMA,' Finkel said.
.jpeg%3Ftrim%3D0%2C0%2C0%2C0%26width%3D1200%26height%3D800%26crop%3D1200%3A800&w=3840&q=100)
The Independent
20-02-2025
- Health
- The Independent
This rare genetic disorder was just treated in the womb for the first time ever
Following the first-ever treatment for spinal muscular atrophy in the womb, physicians say a 2-year-old girl shows no signs of the rare genetic disorder. Spinal muscular atrophy is a genetic condition set in motion before birth that causes worsening muscle weakness. There are four types of the disorder, each with varying degrees of severity. However, for those with the most common and severe form, children typically do not live past age 2. There is no cure, but treatment can help to manage symptoms and prevent complications. The child's mother was given the drug risdiplam, which is the first oral medication approved to treat the progressive neurodegenerative disorder by the Food and Drug Administration. It is manufactured by Swiss biotech firm Roche. 'Our primary objectives were feasibility, safety and tolerability, so we're very pleased to see that the parent and child are doing well,' Dr. Richard Finkel, the director of the St. Jude Center for Experimental Neurotherapeutics, said in a statement. Scientists at St. Jude Children's Research Hospital led the first in utero treatment. 'The results suggest it would be worthwhile to continue investigating the use of prenatal intervention for SMA.' Finkel was the corresponding author of the research which was published Wednesday in a letter to the New England Journal of Medicine. Until now, treatments were given after birth. 'There was still room for improvement,' Finkel told Nature on Thursday. This progress came about by understanding the cause of spinal muscular atrophy. Approximately one in every 6,000 babies is born with the disorder. The research hospital noted in a release that it is caused by a lack of survival motor neuron protein. The protein is essential because it enables muscles to receive signals from the nerves, according to Johns Hopkins Medicine. The absence of the protein occurs in around one in every 11,000 births in the U.S. The drug works by helping produce more of the protein. Because the protein is most needed in the third trimester of fetal development and the first three months of life after birth, St. Jude's says that symptom severity is closely linked with intervention time. So, they launched the clinical protocol to study risdiplam in a single patient. The Food and Drug Administration approved the study. The parents of the patients had a prior infant born with Type 1 and were known carriers of genetic variants. Testing confirmed that their child would likely be born with Type 1. The idea of giving the drug in utero came from the parents, and doctors gave the mother the drug within the final six weeks of her pregnancy. After her birth, the baby was diagnosed with several abnormalities that are considered to have occurred before exposure to the drug. She started taking it at a week old and will likely continue to take it for the rest of her life. They are continuing to monitor her periodically at the research center. 'During the course of the assessment, we really have seen no indication of any signs of SMA,' Finkel said.
