SynaptixBio Ltd.’s Post

Important article in Pharmafile.com by our client Dr Dan Williams at SynaptixBio Ltd. Dan considers the ongoing challenges facing rare disease patients, and discusses developments in the search for therapies. Many thanks to Betsy Goodfellow. The full article is here: https://lnkd.in/e_58twVs.

Developing rare disease therapies is complex and challenging. Read this insightful report by Rare Revolution Magazine that includes perspectives from all stakeholders.  https://lnkd.in/gtA-GZYR #lifesciences #raredisease #clinicialtrials #patientcentricity

Yet another example of when #realworldevidence #RWE is powerful: when clinical trials are not feasible. We analyzed the effectiveness of the antiviral sotrovimab, when Omicron BA.2 and BA.4/5 variants were most prevalent in 2022, a fast-moving period when setting up a clinical trial was not feasible. We published 2 papers from this work, one on the descriptive characteristics of the patients and another showing our use of inverse probability of treatment weighting to compare outcomes for patients treated with sotrovimab versus untreated patients, in which we show significant reduction of risk of COVID-19 hospitalization for sotrovimab-treated patients aged >65 years and with renal disease. Have a read! 👇 Descriptive data ➡ : https://lnkd.in/dD-Wu9fb Comparative effectiveness ➡ : https://lnkd.in/dWC8CYRf

Developing a product to prevent or treat a rare disease involves unique challenges from hard-to-identify and dispersed patient populations for clinical trials through to undefined endpoints and regulatory requirements. While each condition individually has a small population there are over 30 million people affected by rare diseases in the US alone. It’s therefore encouraging to read today that the FDA is establishing a Rare Disease Innovation Hub to connect with the rare disease patient and caregiver community, to enhance collaboration across FDA centres and to advance regulatory science. We look forward to seeing how this new initiative can accelerate treatment and prevention options across rare disease populations #raredisease #fda #patientcentricity

Some great insights from rare disease patients that highlight key considerations for sponsors in clinical trials. Discover what matters most to these individuals and how sponsors can enhance the clinical trial experience. Valuable perspectives shaping the future of rare disease research! #ClinicalTrials #RareDiseases #PatientPerspective

This Phase 2 study evaluated GRANITE as maintenance therapy in front-line metastatic MSS-CRC, with 80% of patients having liver metastases. GRANITE showed a 21% relative risk reduction in PFS overall, with greater benefit in low disease burden patients (38% reduction). Safety profile was favorable, with no treatment discontinuations due to adverse events. https://lnkd.in/gxWta8Cu

Conducting clinical trials for rare disease presents unique challenges and opportunities. Innovative approaches are emerging that promise to revolutionize how we study and development treatments. This article explores fresh ideas that are being implemented to enhance the efficiency and effectiveness of rare disease trials, ultimately bringing hope to patients and their families. https://lnkd.in/ef7hc938

A Phase 3 trial evaluated the use of bulevirtide in patients with chronic hepatitis D. The primary combined end-point response at week 48, defined as an undetectable HDV RNA level or a level that decreased by at least 2 log10 IU per milliliter from baseline, and normalization of the ALT level, occurred in a significantly greater percentage of patients in the bulevirtide treatment groups compared to the control group. Bulevirtide treatment led to reductions in HDV RNA and ALT levels, with a dose-dependent effect observed. Adverse events associated with bulevirtide treatment were generally mild to moderate in severity and did not lead to drug discontinuation. Further studies are ongoing to assess the long-term efficacy and safety of bulevirtide therapy. #KeyTakeaways => Significantly more patients in the bulevirtide groups showed a combined virologic and biochemical response at week 48 compared to the control group. => HDV RNA levels and liver enzyme levels were reduced in patients receiving bulevirtide. => The treatment was well-tolerated, with mild to moderate side effects reported. #JoinTheDiscussion: What do you think this breakthrough in the treatment of chronic hepatitis D means for the future of liver disease management and patient care? Share your thoughts below! #Comment, #Like, #Share, & #Follow @Dr. David Mwin #Source: https://lnkd.in/gcjDHqkE #HepatitisResearch #MedicalAdvancements #LiverDiseaseTreatment #LiverHealth

Despite increasing access to new therapies to treat IBD, a paper presented at #ECCO24 tells a story of continued clinical reliance on steroids to control #IBD disease activity. Explore through this explanation of the study presented, the impact increased steroid usage is having on the #IBDCommunity: https://hubs.li/Q02FV0S20 European Federation of Crohn's and Ulcerative Colitis Associations #EFCCA #SteroidToxicity #SteritasGTI

Looking forward to having specific effective therapy for Sjogren:s Disease. It is over 90 years since it was first clearly defined as a systemic autoimmune rheumatic condition. by Dr. Henrik Sjogren...and yet we struggle for effective treatment.