Neurona Therapeutics’ Post

In a trial led by Ali Rezai, MD, from West Virginia University, a new method combining Aducanumab (Aduhelm) infusions with focused ultrasound showed promise in lowering amyloid-beta levels in Alzheimer's disease. The treatment involves using MRI-guided focused ultrasound to open the blood-brain barrier, enhancing drug delivery. Three patients who received Aducanumab infusions showed greater reduction in amyloid in brain regions targeted with focused ultrasound compared to regions without exposure to it. PET scans indicated a drop in amyloid levels in these targeted areas over 26 weeks, showcasing a potential 32% reduction in standardized uptake value ratio. However, there were some side effects reported. Headaches were the most common, mostly mild, except for one moderate case. Additionally, discomfort during ultrasound treatment led to two severe adverse events for one participant, quickly resolved after the procedure. No amyloid-related imaging abnormalities were observed. This method, which temporarily opens the blood-brain barrier, has shown success in various neurological conditions, and previous studies hinted at reduced amyloid-beta levels without therapeutic agents like Aducanumab. Though the trial's scale was limited to three patients and one side of the brain, larger trials exploring both brain sides are deemed crucial for further understanding and efficacy determination. Despite this, the findings fuel optimism that this approach combined with amyloid-beta removal agents could potentially slow Alzheimer's progression. #Aducanumab #AlzheimersResearch #FocusedUltrasound #endalz https://lnkd.in/gKuHNaK3

A recent #advisoryboard focused on feedback for Phase 2 programs in #gastroenterology sparked insightful discussions on key development strategies. Here are some of the questions we discussed: - What benchmarks should a clinical candidate hit in terms of absolute lymphocyte count (ALC) and placebo-adjusted clinical remission rate to become a commercially viable product? - Assuming safety is confirmed, should dose escalation be pursued to demonstrate stronger efficacy and remission rates? - For “me too” or 2nd generation candidates, how much improvement over existing commercial products is necessary to stand out and succeed in the market? It's always fascinating to watch the clinical development of new candidates unfold over the years. One of the most rewarding aspects is seeing how the insights of our advisors and KOLs evolve. Their perspectives continually shape and refine strategies, and being part of that journey is both exciting and inspiring. #clinicaldevelopment #KOLengagement #biopharma #drugdevelopment #medicalaffairs #lifesciences #clinicaltrials #Phase2

#ICYMI our EVP, Chief Medical Officer Jorge A. Quiroz MD, MBA, was recently interviewed by NeurologyLive to discuss our Phase 2 study assessing NMD670, a neuromuscular transmission enhancer, for patients with spinal muscular atrophy (#SMA). The Phase 2 clinical trial (NCT05794139) is a randomized, double-blind, placebo-controlled, 2-way crossover study to evaluate the efficacy, safety, and tolerability of twice-a-day oral dosing of NMD670 for 21 days in ambulatory adult patients with SMA Type 3. The study is an international multicenter study including sites in North America and Europe. Watch the full interview here: https://lnkd.in/e_kjgcHN #NMDPharma #Biotech #Innovation #NeuromuscularDisease #SMA #SpinalMuscularAtrophy #ClinicalResearch

The buildup of amyloid beta (Aβ) peptides in the brain leads to cognitive decline and memory loss associated with Alzheimer's disease (AD). It's been suggested that the smaller soluble Aβ monomers are more detrimental to healthy brain function than larger Aβ plaques. However, recent breakthroughs suggest that disrupting plaque formation may hinder the progression of Alzheimer’s. IGC Pharma has developed a patent-pending molecule, TGR-63, which disrupts intermolecular interactions between Aβ peptides. This destabilize assembly and prevents the formation of plaques. In preclinical studies, TGR-63 reduced amyloid load in transgenic AD mice. Last year, the FDA approved the use of Lecanemab, an anti-Aβ drug that reduces aggregated Aβ, for the treatment of Alzheimer’s with mild cognitive impairment. It will be interesting to see how TGR-63 performs in clinical trials. The impact of Aβ removing compounds on soluble monomers is a question that remains to be answered. Stay tuned as we continue to follow the latest developments in Alzheimer's research. #AlzheimersDisease #MedicalResearch #Dementia #AlzheimersResearch #BreakThroughStudy #Aβ #AmyloidBeta

Ultrasound Blood–Brain Barrier Opening and Aducanumab in Alzheimer’s Disease ▪️Exciting successful proof of concept study - 3 patients enrolled: 77 yo man, 59 yo man, and 64 yo woman. - Diagnozed with Alzheimer's within 1 year of enrollment. - None received prior treatment of Aducanumab (amyloid beta-directed monoclonal antibody) - 32% average reduction of amyloid beta after 26 weeks of focused ultrasound to open blood brain barrier + direct administration of Aducanumab. - No serious adverse neurologic, cognitive, behavioral, or imaging–related adverse events were observed during the 6-month combination-treatment phase. #pharma #biotech #medicalaffairs #medicine #msl #nuerology #alzheimers https://lnkd.in/g_G8DUnF

Are you considering a cell therapy project? Clinical grade iPSCs are the beginning of many regenerative medicine development projects. In our recent webinar by Dr Mahendra Rao, we discussed the factors to consider when considering a GMP iPSC-based therapeutic project.  We covered the complete GMP iPSC generation process including the choice of reprogramming method, important factors to consider for QC, sourcing the starting cells, and other significant considerations. Watch the webinar: https://hubs.li/Q02hRm0h0

Factors to consider for your clinical grade iPSCs. In our recent webinar by Dr Mahendra Rao, we discussed the factors to consider when considering a GMP iPSC-based therapeutic project. 

With no drugs yet approved despite considerable effort and investment, the stage seems to be setting right for the start of the golden age of muscarinic acetylcholine receptor modulation in neurological diseases. Driven by advances in structure-based drug design and an appreciation of the optimal pharmacological properties necessary to deliver clinical efficacy while minimizing adverse effects, a new generation of M1-receptor and M4-receptor orthosteric agonists and positive allosteric modulators are now entering the clinic. These agents offer the prospect of novel therapeutic solutions for‘hard to treat’ neurological diseases, heralding a new era of muscarinic drug discovery. https://lnkd.in/g3b59ncj

✨A case report published in EMBO Molecular Medicine details the use of IASO Bio's Equecabtagene Autoleucel (Eque-cel, CT103A), a fully human BCMA-targeted autologous CAR-T cell therapy, in treating refractory myasthenia gravis (MG). ✨The report highlights that the therapeutic effectiveness of this CAR-T cell treatment in patients with treatment-resistant MG is based on the reconstitution of B cell lineage. ✨Two patients with advanced-stage, treatment-resistant myasthenia gravis—one with AChR-IgG and the other with MuSK-IgG—BCMA was administered, resulting in favorable safety profiles and sustained clinical enhancements for a duration of 18 months. https://lnkd.in/gw2K76Q2

Improving outcomes after post–cardiac arrest brain injury, ILCOR scientific statement 🧠 post CA brain injury mechanisms ⛔️ challenges in translating preclinical research to clinical practice & therapeutic interventions which failed to improved outcomes in clinical trials 🚧 prioritization of barriers to discovery/translation of neuroprotective therapies for post CA BI #FOAMcc 🔓 https://bit.ly/3VMioOU