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Biogen Reports Promising Interim Phase 1 Results for Salanersen in Spinal Muscular Atrophy, Prepares for Registrational Trials Biogen has announced encouraging topline results from its Phase 1 clinical trial evaluating salanersen (BIIB115/ION306) , an investigational antisense oligonucleotide (ASO) therapy for spinal muscular atrophy (SMA).
As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. Safety biomarkers account for adverse effects of a therapy under study. There are several types of biomarkers to consider.
The study marks a significant step forward in the treatment landscape for haemophilia A, demonstrating not only clinical safety and pharmacokinetic stability, but also strong patient preference for the Mim8 pen-injector delivery system. Throughout the 26-week study period, the investigational therapy was well-tolerated.
MENU logo-altasciences EN - Main navigation Proactive Drug Development Solutions Compare Us Facilities About Us Resource Center Careers News & Events 30th Anniversary Celebration Contact Us Participate in a study Français Home Current: Salt Lake City Salt Lake City Share this page Submitted by pmjackson on Thu, 06/26/2025 - 15:11 Image Downtown (..)
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Prof Rory Johnson, Associate Professor, University College Dublin, and Dr Shalini Andersson, Vice President Nucleic Acid Therapeutics, AstraZeneca will lead this years event focussed on drugging the undruggable.
By harnessing the vast amounts of data generated throughout the development pipeline, pharmaceutical companies can accelerate the discovery of novel therapies, optimize clinical trial design, enhance drug safety monitoring, and deliver personalized medicine, ultimately improving patient outcomes and transforming the future of healthcare.
These studies demonstrated that the BRUKINSA tablet formulation is bioequivalent to the existing capsule form, confirming that patients switching between formulations can expect comparable pharmacokinetics and clinical outcomes. In the United States, BRUKINSA has continued to demonstrate its clinical and commercial strength. Source link
The GEMZ study is a randomized, double-blind, placebo-controlled, fixed-dose, multi-center clinical trial designed to assess the safety, efficacy, and pharmacokinetics of fenfluramine in children and adults aged 1 to 35 years diagnosed with CDD who experience uncontrolled seizures.
These pivotal findings were unveiled at the 85th Scientific Sessions of the American Diabetes Association (ADA) and were concurrently published in The New England Journal of Medicine , underscoring the significant promise of MariTide in the growing field of anti-obesity therapies.
The therapy is administered via subcutaneous injection and is being studied for its ability to produce and sustain significant weight loss over extended periods. The findings are expected to inform healthcare systems and providers about adherence gaps and optimization strategies in statin and non-statin therapy use. to 3:00 p.m.
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However, a deeper understanding of brain function particularly the role of synaptic plasticity is now opening the door to innovative therapies. Event-driven pharmacology Donello highlights the growing recognition of synaptic plasticity’s crucial role in the biology of depression.
Most targeted cancer therapies used today operate by inhibiting targets along well-known oncogenic signalling cascades. The reactivation of oncogenic signalling upstream or downstream of the driving oncogene is a well-studied source of resistance to targeted cancer therapies.
The study demonstrated favorable proof-of-concept for LYT-100’s tolerability and pharmacokinetic (PK) profile, which will also enable twice-a-day (BID) dosing of LYT-100 in future studies. All adverse events (AEs) that were possibly or probably related to LYT-100 were mild and transient and there were no discontinuations. 1 Cottin, V.,
The results, which demonstrate that patients treated with Biogen’s portfolio of MS therapies mount an effective antibody response to COVID-19 vaccination, are being presented at the 37th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) virtual meeting, October 13-15, 2021.
TEAD proteins are known to be very important in cancer progression TEAD proteins are known to be very important in cancer progression, and there are a number of therapies in development. What are the preclinical characteristics of ISM6331, including its efficacy, safety profile, and drug metabolism and pharmacokinetics (DMPK) properties?
Single-Arm Trial Design Single-arm trials have also become an increasingly common development strategy to support regulatory approval and allow patients expedited access to novel therapies, particularly in the accelerated access setting.
If adverse events are anticipated, it is important to understand gene dosage for such an effect (e.g., with gene editing or gene therapy, enzyme replacement therapy), agonism (e.g., adverse profile seen in homozygous null individuals but not in heterozygous carriers?) in liver, in CNS)? with antibodies), or correction (e.g.,
These therapies aim to reverse abnormal epigenetic patterns, restore normal gene function, and offer hope for previously uncurable diseases. Understanding these processes is critical for developing effective epigenetic therapies. These mechanisms form a complex, reversible regulatory network governing cellular identity and behavior.
These data include results from a late-breaking presentation from a Phase 2a study evaluating the safety and pharmacokinetics (PK) of once-monthly (QM) oral islatravir for pre-exposure prophylaxis (PrEP) through 24 weeks. Safety and Pharmacokinetics of Islatravir in Study Participants with Severe Renal Insufficiency. Abstract 2361.
Traumatic brain injury and other tauopathies can have devastating effects on cognitive function and quality of life, yet there are currently no therapies that can halt or slow the rate of decline in these patients,” said Martin Jefson, Ph.D., Pinteon’s chief executive officer. “We
cell and gene therapies), with other therapeutic areas then pushing it further. Because of this, oncology researchers may only have access to minimal existing scientific literature related to cell-based data and animal testing to predict an investigational therapy’s effect on human factors.
Current guidelines are limited and recommend treating pediatric patients with or at risk for reoccurrence of blood clots with standard anticoagulation therapy which requires injections, dietary restrictions, and regular laboratory monitoring. Study The randomized, open-label phase III EINSTEIN-Jr. UNIVERSE was conducted in two parts.
Takeda Pharmaceutical Company Limited ( TSE:4502/NYSE:TAK ) (“Takeda”) today announced that it has submitted a New Drug Application (NDA) to the Ministry of Health, Labour and Welfare (MHLW) in Japan for lanadelumab subcutaneous injection, a monoclonal antibody therapy for prophylaxis against attacks of hereditary angioedema (HAE).
This increases costs and delays the introduction of potentially life-saving therapies. These studies typically include in vitro assays to evaluate cytotoxicity and in vivo models to study pharmacokinetics, pharmacodynamics, and toxicological profiles. Preclinical studies form the backbone of early safety assessment.
In this cohort, the most common treatment-emergent adverse events of any grade (?20%) No patients in this cohort discontinued treatment due to treatment-related adverse events. Initiate or adjust anti-hypertensive therapy as appropriate. Serious, including fatal, hemorrhagic events can occur with Retevmo.
Could you elaborate on the potential market size for SRP-001 and how it fits into the broader landscape of pain therapies? SRP-001 is a novel non-opioid pain relief candidate that works centrally in the brain, offering robust pharmacokinetics without the adverse effects of current medications. South Rampart Pharma, Inc.
The first trial will be conducted as a Phase 1, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, and pharmacokinetics of the intravenous liposomal formulation of ibrexafungerp in healthy subjects. The study will be conducted in South Africa. For more information, visit www.scynexis.com.
3] While MET inhibitors have recently received accelerated approval in this setting in some regions, the vast majority of patients eventually acquire resistance to these therapies, thus underscoring the need for new treatment options. [4] The majority of treatment-related adverse events (AEs) were Grade 1-2. All events were Grade 1-2.
The Phase 1/2 clinical trial will primarily investigate the safety and tolerability of INZ-701 and characterize its pharmacokinetic and pharmacodynamic profile, including plasma pyrophosphate (PPi) and other biomarker levels, to establish a recommended dosing regimen for further clinical development.
Oral, investigational complement factor B inhibitor LNP023 substantially improved hematological response as add-on therapy to eculizumab. To date, after at least six months of stable LNP023 add-on therapy, and at the investigators’ discretion, seven patients (70%) have discontinued eculizumab and remained on LNP023 as monotherapy.
PRX-102 was well-tolerated in the study, with all adverse events being transient in nature without sequelae. The most common moderate treatment emergent adverse events were nasopharyngitis, headache and dyspnea. The most common moderate treatment emergent adverse events were nasopharyngitis, headache and dyspnea. nM and 13.81
18, 2021 /PRNewswire/ — Genkyotex SA , a subsidiary of Calliditas Therapeutics AB (publ) (“Calliditas”) (Nasdaq OMX – CALTX; NASDAQ – CALT), today announced positive Phase 1 data demonstrating a favorable safety and pharmacokinetic profile of high-dose setanaxib, Genkyotex’s lead asset. STOCKHOLM , Jan.
Several clinical sites are screening patients for the Phase 1 a/b multicenter, open-label, dose escalation study of safety, pharmacodynamics, and pharmacokinetics of CG-806 in ascending cohorts (3+3 design) to determine the maximum tolerated dose or recommended dose in patients with relapsed or refractory AML. About CG-806.
2.80] in the PD-L1 positive population, based on 21% of patients with an event; updated HR=1.12 [95% CI: 0.76-1.65]), 1.65]), updated analysis based on 41% of patients with an event). Secondary endpoints include OS, event-free survival, disease-free survival and quality of life measures.
FDA and global approvals for our groundbreaking therapies in SMA and NMOSD, Roche’s data at AAN reflect our continued commitment to meaningful therapeutic progress for people living with neurological disorders,” said Levi Garraway, M.D., Following U.S. Roche’s Chief Medical Officer and Head of Global Product Development. Claims Database.
No new risks were identified, with no serious adverse events related to rVWF reported. 1 The pharmacokinetics (PK) of VWF:ristocetin cofactor (VWF:RCo) and FVIII pharmacodynamics (PD) [PB0917] were also studied and presented at the congress. The sABR was reduced by 91.5% About von Willebrand Disease (VWD). About Takeda Hematology.
Treatment choices are limited, and commonly used off-label therapies are not always effective or may result in prolonged, deep sedation. A therapy that quickly and effectively reduces agitation, without causing excessive sedation, is needed to speed up recovery time and improve patient outcomes. NEW HAVEN, Conn.,
The fast turnover of landiolol will diminish most adverse events due to self-limiting administration. Landiolol prophylaxis is associated with reduced incidence of postoperative atrial fibrillation without triggering adverse events related to a beta-blockade. New Drug Therapy Approvals 2024 (PDF). 32 (2): 828. 2015.09.002.
The open-label Phase 2a ‘AMBITION’ study is designed to assess safety, tolerability, pharmacokinetics and biomarker analyses for early assessments of efficacy of 75 mg and 225 mg CRV431, administered orally to F2 and F3 NASH patients (n=18/dosing group), once daily for 28 days. ” About Hepion Pharmaceuticals.
The Phase 2b trial will also evaluate efficacy on other histology endpoints (fibrosis), assessment of metabolic and non-metabolic parameters, pharmacokinetic assessment as well as safety and tolerability. NASH Investor Event Information.
A live webcast of the event will be available on Poxel’s website at [link] under Events.
For more than a decade, we’ve listened to the HAE community to further understand the need for long-term, preventive targeted therapies and have committed our resources to developing treatment options,” said Neil Inhaber, M.D., percent of patients (n=116) and the most common being injection-site pain. Interactions. Adverse Reactions.
Each of these three investigational therapies uses a different approach to treat a highly prevalent disease for which new treatment options are very much needed. Our stock price is volatile and may be affected by a number of events. ” In June 2021, AbbVie Inc. exercised its right to acquire TeneoOne, Inc.
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