Augustine Therapeutics’ Post

We're proud sponsors and active participants in the 2023 SLC6A1 Connect International Symposium in Orlando, Florida. Delighted to have Dr. Nianwei Lin representing iXCells as one of the presenters, shedding light on how disease-relevant cell models contribute to the progress in rare neurogenetic disorder research. Missed the event? No worries! Gain exclusive access to Dr. Lin's insightful presentation, exploring practical solutions for rare neurogenetic disorders and potential therapeutic strategies, and stay updated on the latest in neurogenetic research. At iXCells, our commitment extends to rare disease advocacies, researchers, doctors, and the scientific community. 🤝 Our goal to contribute to the future of patient care through personalized drug discovery and translational research. #Biotechnology #Innovation #DrugDiscovery #PersonalizedMedicine #RareDiseases #ResearchandDevelopment #SCL6A1 #GeneEditing #EngineeredCells #CRISPRCas9 #CellModels #STEMCells #LifeSciences #ResearchandDevelopment #rarediseaseawareness #aso

On August 22nd, Alkermes announced the initiation of the Vibrance-2 study, a phase 2 clinical trial evaluating the safety and efficacy of ALKS 2680 compared to placebo in adults with narcolepsy type 2 (NT2). "We are pleased to initiate Vibrance-2, a phase 2 clinical study for adults with narcolepsy type 2, based on the data from our phase 1, proof-of-concept study in this patient population. ALKS 2680 is the most advanced investigational orexin 2 receptor agonist currently in development for narcolepsy type 2," said Craig Hopkinson, M.D., Chief Medical Officer and Executive Vice President, Research & Development at Alkermes. "Across narcolepsy type 1 and narcolepsy type 2, significant unmet need remains, and we look forward to further characterizing the efficacy and safety profile of ALKS 2680 in the Vibrance studies in both of these important patient populations." Read the full article at: https://lnkd.in/eCZBfH_x #narcolepsy #sleepdisorder #phase2 #clinicaltrial #Alkermes

#Biotechnology firm Tiziana Life Sciences Ltd (NASDAQ:TLSA) said it had successfully enrolled and dosed four new patients with non-active secondary progressive multiple sclerosis (na-SPMS) in the Brigham and Women’s Hospital, a key member of the Mass General Brigham #Healthcare System Expanded Access (EA) Program. The news brings the total number of patients being followed in the EA Program to 10. Notably, two patients have been dosed for over a year, and an additional four for six months, all without experiencing serious side effects. These findings, combining imaging, immune-biomarkers, and clinical measures, constitute the first comprehensive dataset on patients receiving long-term intranasal foralumab. More at #Proactive #ProactiveInvestors http://ow.ly/lntr1054hoA #NASDAQ #TLSA

Great to hear about a really positive story coming out from a type of funding under the #EJPRD (European Joint Programme for #RareDiseases ) This afternoon Bert Smeets presented very nicely on the multistakeholder collaborations set in motion for #LAMA2 Muscular Dystrophy, enabled by the 'EJPRD Networking Scheme'. Joanne Lee and I have also managed to secure funding through this valuable scheme in the past, which we used to advance our efforts to spread a powerful model for better clinical trials into more rare diseases. The model was created in the #Neuromuscular community under TREAT-NMD , many years ago, and our work in EJP-RD has been to try to expand its use outside of this area. We were able to complement our toolkit (https://shorturl.at/9HXbu) with a 2023 workshop aimed at ERN RARE-LIVER ERN RARE-LIVER and ERN GUARD-Heart in particular, to encourage them to think about how such a model could improve clinical research in their areas. Meanwhile, with support from Joanne Lee and colleagues in the John Walton Muscular Dystrophy Research Centre, there is now an ACT (Advisory Committee for Therapeutics) in the #ataxia field operated by the Ataxia Global Initiative (find out more here - https://lnkd.in/eDWwbMap and here https://shorturl.at/JYQ1k )! Great to see things which work in one rare community being transplanted into others :-) The Networking Scheme -https://shorturl.at/2iOyn- will continue under the European Rare Disease Partnership, #ERDERA so look out for opportunities to bring diverse stakeholders together to drive forwards RD research!

Our CSO Martin Gosling was recently interviewed by Labiotech for an article discussing how new treatments for #cysticfibrosis (CF) are boosting life expectancy. Just three decades ago, the average life expectancy of people with CF was around 30 years of age. This has now improved to 50, with some patients living into their 80s. Recent advancements in CF research have played a key role in contributing to this. One section of the article discusses how Enterprise Therapeutic’s EDT001 has the potential to meet the needs of people with CF. In preclinical studies, ETD001 boosted the clearance of mucus for longer than 12 hours after a single dose, and Phase 1 studies showed a pharmacokinetic profile consistent with long lung retention. Phase 2 trials are due to begin this month. ETD001 has the potential to treat people with cystic fibrosis not currently eligible or receiving benefit from CFTR modulators, and further improve lung function when administered in combination with CFTR modulators. Read more about new treatments for people with #CF here: https://rb.gy/1jdi5x

Zevra Therapeutics announced top-line data from its placebo-controlled, double-blind Phase 2 clinical trial evaluating the safety and tolerability of KP1077 in patients with idiopathic hypersomnia (IH). This proof-of-concept study was not powered to demonstrate statistical significance.  The data gathered for several secondary and exploratory endpoints, including the Epworth Sleepiness Scale (ESS), Idiopathic Hypersomnia Severity Scale (IHSS) and Sleep Inertia Visual Analog Scale (SIVAS) will inform the Phase 3 study design. “Zevra’s Phase 2 trial evaluating KP1077 as a treatment for IH demonstrated clinically meaningful impact and encouraging outcomes on both clinical safety and efficacy,” stated Christopher Drake, PhD, FAASM, DBSM, Principal Investigator of the study. Read more here - https://lnkd.in/gPSzkbdG

🔬📢 𝗘𝘅𝗰𝗶𝘁𝗶𝗻𝗴 𝗡𝗲𝘄𝘀 🔬📢 Proud to share that Parc Taulí Research and Innovation Institute (I3PT), CONNECTA Therapeutics, Centre for Genomic Regulation (CRG) Hospital del Mar Research Institute, have been awarded a €2.7 million grant to drive forward the Phase IIa trials for #CTH120 in #FragileXsyndrome 🧬 The Phase IIa study will assess the efficacy of CTH120 as an innovative #therapy in adults with #FXS, the most common form of inherited intellectual disability without a specific treatment 🤝 This milestone underscores our commitment to advancing #research and improving outcomes for those affected by FXS https://shorturl.at/imvFN

AlzeCure reports anti-inflammatory effects with NeuroRestore ACD856 with relevance to Alzheimer's leading to new patent application AlzeCure  has received preclinical data for NeuroRestore in inflammation, with relevance to Alzheimer's disease, and submitted a new patent application for NeuroRestore ACD856, which is now being prepared for phase II clinical studies. “We continue to strengthen the patent portfolio for our NeuroRestore program, and already have approved patents in all major markets such as the USA, Europe, Japan and China. The new results for our clinical drug candidate ACD856, which we are preparing for phase II against Alzheimer's, provide further evidence for a disease-modifying effect as well as general use against neurodegenerative diseases, where neuroinflammation is central. This further improves our commercial opportunities for this promising substance,” said Martin Jönsson, CEO. Read more here: https://lnkd.in/dTrg-DHy About the share issue: https://lnkd.in/d6PHyuRe #biostock #finwire #alzecure #alzheimers #alzheimerfonden #redeye #avanza #nordnet #seb #swedbank #nordea #aktiespararna #privataaffarer #vatorsecurities #erikpenserbank #carlsquare

Insilico Medicine announced that following the initiation of Phase 1 clinical trials in Australia in November 2023, the company has achieved a key milestone by completing dosing for the first participant in Phase 1 clinical trials for its candidate, ISM5411, in China. ISM5411 is an oral, PHD 1/2-specific inhibitor that facilitates the treatment of IBD by inducing the expression of gut barrier protective genes. Dr. Feng Ren, Co-CEO and CSO of Insilico Medicine, said, "ISM5411 marks another candidate to advance into clinical trials both domestically and internationally, following our IPF project. With its unique mechanism targeting intestinal mucosal repair, ISM5411 demonstrates considerable innovation potential. Preclinical studies have shown its potential to reduce systemic side effects. Additionally, ISM5411 boasts favorable safety profiles and significant efficacy against colitis. We are enthusiastic about the prospect of advancing ISM5411, discovered through the synergy of artificial and human intelligence, to address the growing incidence of IBD." #BiotechInnovation #InflammatoryBowelDisease #QimingPortfolio #QimingHealthcare

That’s some serious computing, virtual screening of almost half a billion compounds! Will be exciting to see how much future virtual screening will successfully identify new lead scaffolds and structures in all kinds of disease areas as computer power continues to increase. Excerpt from original post: “We virtually screened 450 million compounds against the EP4 prostaglandin receptor and identified a novel, potent scaffold with in vivo efficacy. Current first-line anti-inflammatory pain medication such as aspirin and ibuprofen treat pain by inhibiting prostaglandin synthesis which can lead to severe side effects. Targeting the prostaglandin receptor may offer safer therapeutics. A particular challenge for our docking calculations was the membrane-exposed lipid-binding pocket of EP4R, which wasn't well characterized by classical scoring functions. Using molecular dynamics simulations, we embedded the receptor in a lipid bilayer and improved the scoring.”