LifeSci Events’ Post

Breakthrough in ALS Treatment at EAN 2024 On June 30, 2024, at the 10th Congress of the European Academy of Neurology (EAN), Prilenia Therapeutics unveiled exciting results from the Phase II Healey ALS platform trial. The trial focused on pridopidine, an oral sigma-1 receptor activator, as a potential treatment for ALS. Despite the historical challenges faced in ALS clinical trials, the emergence of 12 drugs in mid to late-stage development reflects a promising trend in the ALS pipeline. The Healey ALS platform trial aims to revolutionize the process of drug approval by concurrently testing multiple drugs, thus expediting the identification of effective therapies with a reduced participation requirement. Notably, the trial revealed significant advancements in ALS patients, particularly in the deceleration of disease progression and respiratory decline. Furthermore, a survival analysis suggests that pridopidine could potentially double the survival time for certain ALS patients. # Thank you Benedetta Rossi for your submission!

Cartesian Therapeutics Therapeutics (NASDAQ:RNAC) announced updated efficacy and safety data from its Phase 2b trial of Descartes-08 in myasthenia gravis (MG), showing deep, durable responses without the need for chemotherapy.

Finally, our paper about hepatocyte spheroids is out! We characterize the spheroids and the extracellular vesicles they release, and combine hepatocytes with tumoral cells to challenge them with antitumoral drugs. Please, check it out here.

Regeneron pharmaceutical <at the start> •Founded in 1988 by Len Schelifer, MD, PhD, Professor of Neurology, Cornell Medical School •Four original employees •Initially focused on neuroscience •Specialized in neurotropins (CNTF, BDNF, NGF, NT-3) •Initial therapeutic target: neurodegenerative diseases like ALS <Current Status> •Broad clinical development portfolio across various stages •Therapeutic areas expanded to include: CAPS / Cancer / Hematology / Metabolic disease/ Eye diseases /Arthritis •Utilization of neurotrophic factors, cytokine traps, monoclonal antibodies, and fusion proteins •9 approved drugs with over 30 candidates in pipeline (including 12 programs are under Phase 3) ◇ DUPIXENT: eosinophilic esophagitis (first and only) ◇ EVKEEZA: homozygous familial hypercholesterolemia (HoFH) (first approval) ◇ EYLEA : wetAMD (first approval) ◇ EYLEA HD (8mg) : wetAMD ◇ INMAZEB : Ebola ◇ KEVZARA : polymyalgia rheumatica (first and only) ◇ LIBTAYO : Cancer ◇ PRALUENT : hypercholesterolemia ◇ Veopoz : CHAPLE Disease (first approval) <Keys to Success> •Commitment to cutting-edge science and enabling technologies •Strategically changing their pipeline and development program •Focused on achieving the first and only approval •Distinguished Board of Directors, including Nobel laureates and pharmaceutical professionals •Ability to attract investment and form favorable corporate collaborations

Eli Lilly and Company and Novo Nordisk are facing competition from smaller biotech companies. The challengers are developing innovative drugs targeting various diseases, including diabetes, cancer, and neurodegenerative disorders. The stock performance of these smaller companies reflects growing investor interest and potential threats to the market dominance of established players like Lilly and Novo Nordisk. The underdogs are looking to shake things up... watch this space! #biotech #pharma #Neurology #Endocrinology #Oncology

The FDA has approved Spinogenix, Inc.'s Investigational New Drug (IND) application for their compound, SPG601, which is now ready for Phase 2a trials as a potential treatment for Fragile X syndrome (FXS). Dr. Craig Erickson remarks, "We look forward to the first study of a BK channel modulator in humans with Fragile X and taking the first step to evaluate this important drug mechanism in Fragile X Syndrome." BK channel openers were proposed to treat Fragile X years ago by a French team of FRAXA-funded researchers. Spinogenix is a new company founded by Dr. Peter Vanderklish, Chief Science Officer, and Dr. Erickson, Chief Medical Advisor, and both FRAXA investigators. Their compound was tested in Fragile X mice with excellent results at the FRAXA Drug Validation Initiative (FRAXA-DVI) in Chile, paving the way for clinical trials. #FragileX #FXResearch #ClinicalTrials #SynapticRestoration #Neurodevelopment #InnovativeTherapeutics #TreatmentAdvances #BKChannelModulator #Neuroscience

In early April of this year, PureTech Health launched Seaport Therapeutics with $100m Series A funding to advance a pipeline of neuropsychiatric drugs with improved oral bioavailability for the treatment of depression, anxiety and other mood disorders. Behind the company are pharma executives from neuro-focused Karuna Therapeutics, which was acquired by Bristol Myers Squibb for $14Bn back in December 2023. As part of its early-stage pipeline, Seaport disclosed “SPT-348” - a prodrug of a non-hallucinogenic neuroplastogen - which is currently in preclinical studies and positioned to treat various mood and neuropsychiatric disorders. Neuroplastogens are novel compounds that harness the rapid-acting efficacy of first-generation psychedelics without inducing hallucinogenic and other associated adverse effects. These compounds may show clinically meaningful benefit in the treatment of various psychiatric and non-psychiatric disorders. We are thrilled to see other companies explore the therapeutic potential of neuroplastogens as part of their drug discovery and development efforts, and we expect this field of research to grow rapidly in the near future. #neuroplastogens #neuroscience #neuropsychiatry https://lnkd.in/eUDcGt7u

𝐈𝐦𝐛𝐫𝐢𝐮𝐦 𝐓𝐡𝐞𝐫𝐚𝐩𝐞𝐮𝐭𝐢𝐜𝐬 𝐀𝐜𝐡𝐢𝐞𝐯𝐞𝐬 𝐋𝐚𝐬𝐭 𝐏𝐚𝐭𝐢𝐞𝐧𝐭 𝐋𝐚𝐬𝐭 𝐕𝐢𝐬𝐢𝐭 𝐢𝐧 𝐏𝐡𝐚𝐬𝐞 𝟏𝐛 𝐒𝐭𝐮𝐝𝐲 𝐨𝐟 𝐒𝐮𝐧𝐨𝐛𝐢𝐧𝐨𝐩 𝐟𝐨𝐫 𝐎𝐯𝐞𝐫𝐚𝐜𝐭𝐢𝐯𝐞 𝐁𝐥𝐚𝐝𝐝𝐞𝐫 𝐒𝐲𝐧𝐝𝐫𝐨𝐦𝐞 Imbrium Therapeutics L.P. Imbrium Therapeutics, a subsidiary of Purdue Pharma L.P., announced the completion of the last patient visit in its Phase 1b study of sunobinop, an investigational treatment for overactive bladder syndrome (OAB). Sunobinop, a novel oral compound, is designed to bind to and activate the nociceptin/orphanin-FQ peptide receptor (NOP), a protein involved in bladder function. The multicenter, randomized, double-blind, placebo-controlled crossover study enrolled 51 female patients to assess the effects of sunobinop on urinary urgency, frequency, incontinence, and nocturia compared to placebo. "Millions of patients with overactive bladder are in need of additional treatment options," said Dr. Julie Ducharme Julie Ducharme , Vice President and Chief Scientific Officer. "This study will help us learn more about sunobinop’s potential to be a new treatment option.” “We are pleased to achieve last patient last visit in the sunobinop Phase 1b study,” said Craig Landau, MD Craig Landau, President and CEO of Purdue. “Our continued progress demonstrates our commitment to innovation through research and development.” In addition to OAB, Imbrium is also evaluating sunobinop as a potential treatment for interstitial cystitis/bladder pain syndrome (IC/BPS) and alcohol use disorder. #ImbriumTherapeutics #PurduePharma #Sunobinop #OveractiveBladder #ClinicalTrials #Innovation #PharmaceuticalResearch #Urology

📢 The Complement-Based Drug Development Community is Shifting Gear...📢 With more companies than ever targeting alternative complement proteins for their therapies, indication expansion in becoming a trend in the field with more assets than ever before, spanning: 🆎 Haematology 🔬 Nephrology 👀 Opthalmology 💊 Oncology 🧠 Neurology The complement-based therapeutics field has everyone asking the same question: which inhibitor will be the next to harness the complement system for the development of groundbreaking therapies? From the discovery of new pathways to target to post-approval indication expansion, the immunology landscape is primed to capitalize on the huge strides being taken in achieving efficacy and approving the safety profile of complement-based therapeutics.  Join 100+ complement industry leaders, scientists, and clinicians at the premier, end-to-end 8th Complement-Based Drug Development Summit to shape the future of complement-targeted therapies. Secure your ticket here: https://ter.li/jj8ur0

Extensive evidence of preclinical anti-NASH activity has spawned the clinical testing of different #PPAR-modulating drugs as NASH therapeutics. Whereas several single and dual PPAR agonists have demonstrated some efficacy on individual histological parameters, pan-PPAR agonism appears required to achieve significant results on histological endpoints. Intra- and extrahepatic vascular dysfunction in NASH likely contributes to hepatic and cardiovascular morbidity and is modulated by PPARs. All PPAR agonists are chemically distinct and hence display different pharmacokinetic and pharmacodynamic properties which result in drug-specific responses. This selective receptor interaction implies that each PPAR agonist requires #individual efficacy and safety assessment.