Pikralida ’s Post

Avidity Biosciences, Inc. which received early funding from CureDuchenne, announced positive initial data from their EXPLORE44 Trial for individuals with Duchenne amenable to skipping exon 44. Their experimental exon-skipping agent, AOC 1044 (del-zota), at 5 mg/kg increased exon 44 skipping by an average of 37% (from 7% at baseline to 44%) and increased average dystrophin levels by 25% (from 7% of normal at baseline to 32%) after 3 doses received once every 6 weeks. Creatine kinases levels were reduced to near normal levels. Register for the webinar on August 16, 2024, at 9 AM PST: https://lnkd.in/gEEhp-kc Learn more and read the letter to the community: https://lnkd.in/gq562NU7 #DMD #duchenne #exonskipping #CureDuchenne #AvidityBioscience #FDA #clinicaltrials

#CRAB #POLIMIXIN #CLSI "A Susceptible* category for polymyxins cannot be established because there is no MIC for which likely clinical efficacy can be predicted. When used, polymyxins should be administered at maximally tolerated doses and in combination with a second agent.”24 Work with your antimicrobial stewardship program to determine the appropriate lab testing and reporting strategy for your facility. If you are going to use a polymyxin as part of your treatment regimen, polymyxin B is preferred over colistin given its more favorable pharmacokinetic profile." Dr. Tamma interview 2024. (CLSI Newdoc).

Physiogenex will be presenting the effects of resmetirom (the first drug to be FDA approved for MASH) in its 3-week MASH mouse model at APASL 2024 Kyoto in Japan this week: moderated poster presentation on March 28th at 5:10-5:16pm Check out our model data here: https://lnkd.in/e4s5nMwz As a CRO running #MASH animal models for more than a decade, Physiogenex is very happy to see Resmetirom FDA approved for MASH patients without cirrhosis. This major event also opens new paths for other therapies to be approved for treatment of MASH and liver fibrosis. If you are developing these kinds of therapies and want to gain time and money to reach the clinics asap, we have the right preclinical model for you 🙂 ! Let's meet at APASL 2024 this week or contact us on our website: https://lnkd.in/eR8rpD7H We have a >20-year expertise in drug development and a 97.5% customer satisfaction rate over the last 3 years. Our expertise – your success   #MASH #drugdiscovery #preclinicalresearch

Today we announced positive topline results from our Phase 1 study of DARE-PDM1, our investigational product designed to deliver diclofenac, a nonsteroidal anti-inflammatory drug (NSAID), vaginally via our proprietary hydrogel to treat primary dysmenorrhea, or painful menstruation. At Daré, we’re committed to advancing a portfolio of differentiated product candidates that address meaningful unmet needs in women’s health. The data from this Phase 1 study, which evaluated the pharmacokinetics, safety and exploratory efficacy of DARE-PDM1, support its continued clinical development as well as its potential as a first-in-category product, delivering diclofenac in a convenient vaginal format that may extend the duration of pain relief and reduce the risks associated with the oral delivery of NSAIDs. Learn more about DARE-PDM1: https://lnkd.in/ea6h2ZiH

I am gratified to share my #RSCPoster on the LinkedIn platform about my first publication titled "Polymorphs and isostructural cocrystals of dexamethasone: towards the improvement of aqueous solubility". #RSCInorg #RSCOrg #RSCMat #RSCChemBio In this work, we have reported the crystal structure of a polymorph and two cocrystals of dexamethasone for the first time. Dexamethasone a BCS Class-II drug, has low solubility used to treat various inflammatory diseases. It was also recommended to treat COVID-19 patients with severe respiratory symptoms as it reduces the mortality rate. The crystal engineering approach is employed to alter the properties of the drug. Cocrystals of DEX were synthesized using phenolic coformers, Resorcinol and Catechol which significantly enhanced the solubility and dissolution profile. By improving the solubility of low-soluble drugs, we can thereby increase their bioavailability to provide better clinical efficacy. I thank my research supervisor Palash Sanphui and our collaborator Vladimir Chernyshev for their support and contribution throughout. For more details find the CrystEngComm journal https://lnkd.in/gz7CuKP8.

I’m excited to share these positive topline results from our Phase 1 study of DARE-PDM1, a potential first-in-category treatment for primary dysmenorrhea, otherwise known as painful menstruation. The most common interventions for primary dysmenorrhea include oral nonsteroidal anti-inflammatory drugs (NSAIDs) and hormonal contraceptives, which frequently create undesirable side effects. These topline Phase 1 data indicate that a vaginal route of administration may provide a more convenient and accessible treatment option for women that addresses both the pain-related symptoms of dysmenorrhea and minimizes the side effects commonly associated with the use of oral medications. #womenshealth Learn more: https://lnkd.in/gBMsfVqU

Pliant announced positive safety and exploratory efficacy data from the INTEGRIS-PSC Phase 2a trial of Bexotegrast (PLN-74809) 320 mg in patients with primary sclerosing cholangitis and suspected liver ibrosis. PLN-74809 is an oral small-molecule dual-selective inhibitor of αvβ6 and αvβ1 integrins for the treatment of IPF and PSC. While present at very low levels in healthy tissues, these integrins are upregulated in the lungs of IPF patients and the bile ducts of PSC patients where they activate TGF-β, a key driver of the fibrotic process. Blocking these integrins is designed to stop TGF-β activation, potentially halting the growth of scar tissue. Chemenu team is always committed to developing more compounds for drug discovery. Here come the related building blocks we can provide: https://lnkd.in/gdkMaz8v 👇 #Chemenu #Pliant #INTEGRIS #Bexotegrast #inhibitor #αvβ6 #αvβ1 #integrins #IPF #PSC #TGF-β #drugdiscovery #buildingblocks

A Superagonist, A Novel Anti-obesity Agent from a Zebrafish Screen, and More News Highlights From January 2024 | https://lnkd.in/gkBDxXgk Among the small molecule highlights in January’s news were a $100M+ Series A for a PARP1 + PI3Kα focused company, clinical data with an SLC inhibitor for PKU (Jnana Therapeutics), a 5-HT2C superagonist for seizures (Longboard Pharmaceuticals), and a NK1,3 dual antagonist for women’s health (Bayer | Pharmaceuticals, GSK). A novel anti-obesity agent from phenotypic screening also made headlines, and the close of a major acquisition may bring relief to the industry. You can read about these notable scientific highlights and more in our January news roundup. Full Article: https://lnkd.in/gkBDxXgk

On 16 Sep in 1928, Alexander Fleming discovered penicillin, the first true antibiotic. How has drug discovery evolved since then? Share your views in Comments #didyouknow #todayinhistory #medicalhistory #vigilare #vigilarebp #vigilarebiopharma #history #medicine

Catch a concise 15 min update (or three) on key topics for the Pharamceutical Industry #methodmodernisation #nitrosamines #oligonucleotide analysis Link below ! #waters