Sciensus - Rare Disease Services’ Post

On July 3rd, we’ll join CureDuchenne for a webinar about the efficacy and safety data from the DELIVER trial of DYNE-251, an investigational therapeutic for people with #DMD amenable to exon 51 skipping: https://bit.ly/3zvfpTf #clinicaltrial #drugdevelopment

💡 𝐁𝐢𝐠 𝐍𝐞𝐰𝐬 𝐢𝐧 𝐭𝐡𝐞 𝐑𝐚𝐫𝐞 𝐃𝐢𝐬𝐞𝐚𝐬𝐞 𝐒𝐩𝐚𝐜𝐞! 💡 Immedica Pharma AB has announced its agreement to acquire Marinus Pharma, marking a transformative milestone in the fight against rare seizure disorders. This acquisition brings ZTALMY® (ganaxolone) oral suspension, an FDA-approved breakthrough for treating CDKL5 deficiency disorder (CDD) seizures, to Immedica's portfolio. This move expands Immedica's rare disease focus into North America and strengthens its mission to deliver life-changing treatments for underserved patients. 💬 What does this mean for patients? More access to innovative therapies and hope for improved outcomes. 👉 𝐋𝐞𝐚𝐫𝐧 𝐦𝐨𝐫𝐞 𝐚𝐛𝐨𝐮𝐭 𝐭𝐡𝐢𝐬 𝐢𝐦𝐩𝐚𝐜𝐭𝐟𝐮𝐥 𝐜𝐨𝐥𝐥𝐚𝐛𝐨𝐫𝐚𝐭𝐢𝐨𝐧 𝐡𝐞𝐫𝐞: https://lnkd.in/gdY7HyFm #rarediseases #pharmainnovation #seizuredisorders #pharmaceuticalnews #healthcareinnovation #immedicapharma #marinuspharma #medicalbreakthroughs #fdaapproved #rarediseasetreatment #globalhealth #healthcareleadership

More than 95% of all #rarediseases lack any FDA-approved treatment options, and bringing a drug to market for a #raredisease is an inherently complicated and risky endeavor. That is where pilot programs like the Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program come in, promising to potentially shave off years of the #drugdevelopment pipeline. #FDA announced the first pilot program participants earlier this week, offering much-needed hope to the 7 associated rare disease patient communities - and ultimately to everyone in the rare space who will be able to benefit from the shared learnings. Read the full article here: https://lnkd.in/eeJYSsMA

Thanks for sharing Targeted Oncology and Sabrina S.! Obviously a significant milestone for MRD (and likely a sign of things to come for other tumor types) . . . More to come later but for for now, here's a link to the meeting information and materials: https://lnkd.in/gMeHzXzN Some interesting tidbits from the article: [FDA representatives] "confirmed that . . . there was a strong individual-level association for PFS and OS, but trial-level associations were weak to moderate in disease subpopulations for PFS and were generally weaker for OS." And "a strong trial-level association could become a validated surrogate end point and support regular approval; however, very few oncology end points have met this standard, and most end points that support accelerated approval have not been assessed for trial-level surrogacy or have weak trial-level associations." #fda #mrd #acceleratedapproval #multiplemyeloma

Despite significant changes in treatment options over the past decade, MM patients still face inevitable relapse. Confirmative trials typically last 5+ years, focusing on PFS (primary endpoint) and OS (key 2nd endpoint). With MRD as an early endpoint, new treatments could reach patients sooner. MRD testing is well-established for MM and correlates directly with disease burden. We eagerly await detailed FDA guidance, especially regarding single-arm studies and another early endpoint, objective response rates. Additionally, FDA expanded approval for two CAR T-cell therapies as earlier treatment options for Relapsed/Refractory MM. Despite concerns from CBER about early deaths, the ODAC committee views these risks as manageable. Personally, I remain skeptical about the overall survival benefit of BMS/Celgene therapy due to high crossover rates. Overall, this April brings new hope for MM patients and oncology professionals.

Cardiol Therapeutics Inc. (Nasdaq: $CRDL) is advancing its late-stage Phase III MAVERIC trial for CardiolRx™, targeting the $1B recurrent pericarditis market. #CRDL aims to redefine heart disease care with a safer, more effective oral option. Check out the latest Q&A with CEO David Elsley to learn more. #StocksInFocus https://lnkd.in/dGQ8dSHz

The field of complement therapeutics is rapidly advancing, offering new potential in treating autoimmune and inflammatory diseases. Our latest insight by Rohit Katoch, Senior Director, dives into the evolving landscape, spotlighting key trends and innovations driving this space forward. As biopharma continues to push boundaries, complement therapeutics is set to reshape treatment paradigms. Read more about these emerging trends here: https://lnkd.in/dmHv3B8A #PharmaStrategy #AutoimmuneDiseases #ComplementPathway #ClinicalDevelopment

In my new article, I talk about the incredible potential of a unique new drug, SPG302, developed by Spinogenix, Inc., for treating amyotrophic lateral sclerosis (ALS) and how the FDA has approved it for ongoing clinical trials. The drug regenerates the synapses damaged by the fatal disease. In Phase 1 trials conducted in Australia, the drug has proven safe and is now being trialled in people with ALS. It could be a watershed moment in ALS treatment. #ALS #synapse #smallmolecule https://lnkd.in/gbJhSmbx

We are proud to announce our new partnership with AFYX Therapeutics A/S; covering the Mediterranean and CEE regions. Our partnership with AFYX will encompass two treatments across 22 countries in these two regions. We look forward to collaborating with AFYX and other healthcare stakeholders to bring these essential treatment options to local communities. Read the press release here: https://lnkd.in/d6s8UATK

Vascular Ehlers Danlos Syndrome is a life-threatening #RareDisease, which is why Zevra Therapeutics is committed to bringing a new treatment to the #VEDS community.  Through our Decentralized Study Evaluating Celiprolol on VEDS Related Events (DiSCOVER) clinical trial, we are investigating celiprolol as a potential therapy for VEDS.  Want to know if you’re eligible? Learn more about our DiSCOVER trial and enroll here: https://bit.ly/3C305i8  #RareDisease #ClinicalTrial