Benjamin Stecher’s Post

Did you know that Worldwide partners with over 115 rare disease organizations around the globe to help leverage shared expertise so we can improve patient outcomes and accelerate breakthroughs in drug development? Learn more about these partnerships and how these organizations are helping make an impact on rare disease clinical research in our blog: https://bit.ly/3V5djBD

Adam Feuerstein spent four crazy years writing about Cassava Sciences. The story ended exactly as he predicted and many knew it would — with the company’s experimental drug for #Alzheimers disease, simufilam, failing a clinical trial. He explains why in a new column: https://trib.al/73mD9Do

Oral drug regimes, delivered in community settings, have the ability to reach wider sections of society. The FDA continue to take diversity and inclusion seriously. Latest guidance: https://lnkd.in/eT5AhNTP CellCentric is developing a new oral treatment for multiple myeloma. While cell and antibody-based therapies have brought great progress for patients, they can be complex to administer, and not suitable for many. There is a growing need for complementary approaches, able to be administered in community settings. Despite significant gains in recent times, 75% of people diagnosed with multiple myeloma still die of the disease. There remains a real need for continued progress. #multiplemyeloma Will West Debbie Haynes Tomasz Knurowski Kris Frese Karen Clegg

At Putnam, we have a proven track record of delivering superior strategic support to pharma leaders, especially in the evolving landscape of autoimmune disease treatments. With the increasing understanding of disease pathogenesis, the clinical focus on drugs targeting the complement pathway has accelerated. In our latest blog by Rohit Katoch, Senior Director, get an in-depth overview of the clinical development landscape, spotlighting key targets and emerging trends. Read more to see how these advancements are shaping treatment strategies: https://lnkd.in/et-DHCrd #PharmaStrategy #AutoimmuneDiseases #ComplementPathway #ClinicalDevelopment

Thanks to Gary Rafaloff and co-authors, including others living with Parkinson's and researchers like Brian Fiske, for creating a digestible summary of where the Parkinson's clinical trial pipeline currently stands. I appreciate that the pipeline includes nearly 60% symptomatic (easing the motor and non-motor symptoms) but more than 40% disease-modifying (impacting the underlying disease to slow or stop progression over time) therapies. And that there is diversity in treatment approaches (inflammation, mitochondria, cell pathways, etc.), especially in the disease-modifying portfolio. Check out the full paper for details: https://shorturl.at/NJyQG

ALS, or Lou Gehrig’s Disease, is a disease that causes progressive degeneration of nerve cells in the spinal cord and brain. It affects 30,000 people in the US every year and treatment for it focuses primarily on reducing discomfort and slowing disease progression. US-based company Spinogenix, Inc. is developing a novel drug therapy for ALS that focuses on restoring synapses for patients affected with this disease. The treatment developed is a once-a-day pill that targets synapse loss, which is central to ALS. The company has just received FDA clearance for Investigational New Drug Application for its clinical trials in Australia. Studies so far in healthy volunteers have shown excellent tolerability and plasma levels aligned with efficacy in animal models. Read more about this drug here: https://lnkd.in/g3XiHc6d . . . #research #SimplerScience #als #healthcare #treatment #futureofhealthcare

Here’s a simulation of my own beating heart, developed by ELEM Biotech. ❤️ As the World Heart Federation highlights, cardiovascular disease (CVD) is the leading cause of deaths worldwide, with more than 20.5 million every year. Replicating the complex environment of CVD for drug testing is a major challenge due to its heterogeneity and its progressive nature, which makes it difficult to predict long-term responses based on short-term trial data. ELEM Biotech’s #VirtualHearts provide virtual patient populations for in-silico drug testing tailored to each particular testing scenario, enabling Pharma companies to develop SMART trials that account for a necessary population variability, accelerating the drug development. #CardiovascularHealth #UseHeart #VirtualHumanTwins #SMARTtrials #UseVirtualHearts

2025 promises to represent another rich year of progress. https://ow.ly/5JxC50TKMAf A wealth of new therapies are set to successfully graduate from the pipeline. Within this cohort are a mixture of therapeutic areas, drug classes, first-time approvals, label expansions, and, most importantly, treatments that will meaningfully change how diseases are treated. This article provides a snapshot of these advancements and the potential new approvals to add to your vocabulary, from apitegromab to Zegalogue. Click the link in this post to read the article.

2025 promises to represent another rich year of progress. https://ow.ly/Ez2M30sHTw7 A wealth of new therapies are set to successfully graduate from the pipeline. Within this cohort are a mixture of therapeutic areas, drug classes, first-time approvals, label expansions, and, most importantly, treatments that will meaningfully change how diseases are treated. This article provides a snapshot of these advancements and the potential new approvals to add to your vocabulary, from apitegromab to Zegalogue. Click the link in this post to read the article.