Juliette Borri’s Post

Heading to the World Orphan Drug Congress in #Boston this week? On Wednesday, April 24 at 3:20 pm, Michael Fusakio and Samantha Z. will lead a fireside chat on "Rare but not Forgotten: Challenges and Opportunities for Rare Disease Drugs." You'll hear stories and lessons learned on the path to getting rare disease drugs on the market. To help further accelerate the development of novel drug and biological products for rare diseases, the #FDA launched START. This is a pilot program allowing for more communication with the agency to address development challenges. Read Mike Fusakio's review of the program and its benefits. https://hubs.la/Q02tRwPf0 Stop by the fireside chat to learn more about opportunities to advance your rare disease drug development. #WODC #OrphanDrugDesignation #RareDisease

Sarcoidosis Treatment Market Size in the 7MM was ~USD 828 million in 2023, estimates DelveInsight: Sarcoidosis Treatment Market is projected to witness substantial growth over the next few years, driven by advancements in treatment modalities and increasing awareness of the condition. The pipeline for Sarcoidosis Therapeutics includes promising candidates that aim to not only treat … Continue reading → #PharmaceuticalsBiotech

I wanted to point out Immunovant's investor call at 8:00am ET this morning to provide an update on the Graves’ disease development program. Some key points to note from the release: High dose batoclimab achieved 76% response rate in patients uncontrolled on antithyroid drugs (ATDs) at week 12 High dose batoclimab achieved 56% ATD-free response rate in patients uncontrolled on ATDs at week 12 Strong correlation observed between degree of IgG lowering and clinical outcomes yields potential best-in-class and first-in-class opportunity for IMVT-1402 in Graves’ Disease (GD) Real world claims data indicates 25-30% of Graves’ Disease patients per year are uncontrolled on ATDs with minimal to no existing therapeutic options representing an attractive commercial opportunity with limited competition IND cleared with initiation of IMVT-1402 pivotal trial in GD expected by calendar year end See below PR link for additional details and webcast info Roivant Sciences #gravesdisease #innovation #antifcrn #biotech https://lnkd.in/eSW2gMdG

Thoughts on this? >> AN2 Therapeutics undergoes makeover after late-stage lung disease trial underwhelms >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #pharmaceutical #productmarketing #healthcare #biotech #pharma

We are pleased to share that our CPa9-HNE blood-based biomarker assay has received a Letter of Support from the FDA. CPa9-HNE, also known as the Nordic Serum Calprotectin biomarker, measures activated neutrophils and is now recommended for inclusion in studies on inflammatory bowel disease (IBD). CPa9-HNE may enhance clinical trials by identifying patients with moderate to severe active ulcerative colitis or Crohn’s disease, potentially reducing the need for unnecessary endoscopies. The FDA encourages formal qualification of CPa9-HNE as an enrichment biomarker through analytical validation and data sharing across clinical trials to advance its development as a drug development tool. ➡️ Read the full news and the Letter of Support on the link below: https://lnkd.in/e_7TSxr9 #FDA #IBD #LetterOfSupport #DrugDevelopment #Neutrophils #CPa9HNE

Moderna's investigational therapeutic for methylmalonic acidemia (MMA), mRNA-3705, has been selected by the U.S. Food and Drug Administration (FDA) for the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program 🎉 3 Key takeaways: 🔴 FDA’s START Pilot Program: mRNA-3705 will receive enhanced support and guidance from the FDA under the START pilot program, which aims to accelerate the development of novel treatments for rare diseases by providing rapid, ad-hoc interactions with the FDA. 🔴 Methylmalonic Acidemia (MMA): MMA is a rare, life-threatening genetic liver condition caused by a deficiency in the enzyme MUT, leading to a toxic buildup of acids in the body. There are currently no approved treatments for MMA, and the disease is associated with significant mortality and morbidity. 🔴 Moderna’s mRNA-3705: This investigational therapeutic encodes the MUT enzyme, restoring acid metabolism in patients with MMA. It has received orphan-drug, fast-track, and rare pediatric disease designations from the FDA and is currently being tested in a phase 1/2 clinical trial. Credit to the full article Fierce Pharma - https://lnkd.in/ecdAqHd3 #moderna #FDA #Clinical #trials #contractresearch #lifescience #USA #Clinicaltrial #rarediseases

Noxopharm Limited (ASX:NOX, OTC:NOXOF) is preparing to launch a first-in-human Phase 1 trial assessing SOF-SKN™ drug candidate and Sofra™ platform for #AutoimmuneDiseases in its HERACLES or ‘Harnessing Endogenous Regulators Against CLE Study’. NOX sees the Australia-based trial as the first step in leveraging the Sofra™ platform’s potential to tackle the autoimmune disease market in areas such as #RheumatoidArthritis. Holding the trials within Australia will offer access to R&D tax incentives from the Federal Government and Australian expertise in #lupus research and early-phase clinical trials. The trial is planned to start in early 2025 and is designed to provide proof of concept for the skin disease that is caused by cutaneous lupus erythematosus (CLE). More at #Proactive #ProactiveInvestors #Biotech #Biopharmaceutcials #ASX #NOX http://ow.ly/4ENI105FPZa

As per the estimate, there are over 7,000 rare diseases, collectively affecting over 300 million people worldwide. Rare diseases, by their very nature, present unique challenges. Often affecting a minuscule fraction of the population, these disorders can be exceedingly complex, with symptoms that vary widely from person to person. As we commemorate Rare Disease Awareness Day, let us not only celebrate the remarkable progress that has been made, but also rededicate ourselves to the ongoing pursuit of solutions that will improve the lives of individuals affected by rare diseases. Central to this endeavor is the indispensable role of pharma and biotech companies in rare disease therapeutics development, whose unwavering commitment, ingenuity, and compassion continue to drive transformative change, bringing hope to those who need it most. Pharma giants such as Novartis, Roche, Sanofi, Biogen, Pfizer, Shire (now part of Takeda), Vertex Pharmaceuticals, Regeneron, Alexion Pharmaceuticals, Inc. (now part of AstraZeneca), BioMarin Pharmaceutical Inc., CSL, Ultragenyx, Sarepta Therapeutics, Agios Pharmaceuticals, Ionis Pharmaceuticals, Inc., Horizon, Amicus Therapeutics, Spark Therapeutics, Inc., bluebird bio, Alnylam Pharmaceuticals, Celgene (now part of Bristol Myers Squibb), Astellas Pharma, Ultragenyx, among others, are contributing significantly to the development of therapies and driving transformative change in the rare disease treatment outlook. At DelveInsight Business Research LLP, we provide essential insights to pharmaceutical companies embarking on the development of innovative therapies for rare diseases. Our analysis of disease landscapes, identification of market trends, and assessment of competition aids companies in navigating challenges and capitalizing on opportunities. Through patient engagement and regulatory monitoring, we facilitate informed decision-making, ultimately supporting the efficient development and successful commercialization of transformative treatments. #raredisease #rarediseaseawareness #rarediseaseday #rarediseaseresearch #rarediseaseday2024 #showyourrare #invisibleillness #uncommonconditions #rarebutnotalone #careaboutrare #rarediseasesupport #rarediseaseaware #raiseawareness #globalgenes #findacure #patientvoice #oneinamillion #nevergiveup

In a special #MemberSpotlight by EUCOPE, Enrico Piccinini, Head of Europe & International Rare Diseases, discusses our work to deliver innovative therapies and solutions for people living with rare diseases and shares insights on key challenges impacting the #lifesciences industry today. Read more: https://lnkd.in/dnGnadYK EUCOPE - European Confederation of Pharmaceutical Entrepreneurs #RareDiseases #ChiesiGlobalRareDiseases

OK getting closer! Aldeyra claims their latest results are the "first positive Phase 3 clinical trial in a dry eye chamber with a symptom as a primary endpoint". I hope they're right about efficacy, because our dry eye community's greatest need is tools for improving chronic ocular surface pain (aka DED symptoms) and hence quality of life. Dry eye, or rather the ever growing bucket of things we are calling dry eye today, is a symptom-driven condition - yet the industry is continuing to pour the majority of its effort and investment into ways to modify a disease state, hoping that symptoms will come along for the ride despite decades of evidence that it really doesn't work like that. We need industry squarely focused on producing relief for COSP and we need our regulators supporting the pathway to produce it.