Denali Therapeutics

We're pleased to share important progress in our efforts to address the urgent needs of individuals and families with MPS II (Hunter syndrome). Following a recent successful meeting with the FDA's CDER division, we now have a path to accelerated approval and conversion to full approval for tividenofusp alfa (DNL310). This progress, including CDER's recognition of cerebrospinal fluid heparan sulfate (CSF HS) as a surrogate biomarker, reflects a collective effort?across the patient community, academia and industry?to?communicate the science and advocate for faster paths to effective treatments?for MPS II.?We thank CDER for a positive and collaborative discussion. Additionally, at?SSIEM2024?this week, we're presenting new interim data from the Phase 1/2 study of tividenofusp alfa, showing robust and durable biomarker responses and positive effects on clinical endpoints. These results reinforce our commitment to delivering effective therapies as quickly as possible. Read more in our press release here: https://lnkd.in/giQmhav6 View our poster presentations here: https://lnkd.in/gYbRTRtQ

We look forward to joining the community at the SSIEM 2024 Annual Symposium next week and to presenting new interim data from the Phase 1/2 study of tividenofusp alfa (DNL310), demonstrating the potential to address both body and brain manifestations of MPS II (Hunter syndrome). We hope to see you there!?

What an incredible day?coming together?with ALS Network to rally support and raise awareness for those living with ALS!?A huge thank you to?Cytokinetics and all our colleagues who joined us for the third consecutive year at the South San Francisco SPLASH to Cure ALS on August 14th. Coming together,?taking the plunge, and standing with the local ALS community was truly inspiring. This month marks the 10th anniversary of the #ALSIceBucketChallenge, making our efforts this year even more impactful—together, we’re making a difference!

We are grateful to Jennifer Thompson and two remarkable young adults for joining us in an important discussion on the impact of ALS on families and the resilience of youth caregivers. Thank you for sharing your experiences with?us and for everything Hope Loves Company, Inc. does to support families and children affected by ALS.? #ALS #Caregivers #CommunitySupport

We are pleased to share that?Scientific Translational Medicine?has published our research in an article titled,?"Targeting the transferrin receptor to?transport antisense oligonucleotides (ASOs) across the mammalian blood-brain barrier".?This is the first publication of data showing that ASOs can be delivered uniformly throughout the primate CNS and to muscle via a less invasive route of intravenous administration enabled by Denali's Transport Vehicle (TV) platform. Read the article here: https://lnkd.in/gdW9fjMJ

We reported our second quarter financial results and business highlights. Read our press release here: https://lnkd.in/gsaNH-DP

We were thrilled to join the The ALS Association's first-ever NEXUS Summit this week! It was a powerful few days of connecting, listening, and learning from hundreds of dedicated participants who attended to contribute to the ongoing fight against ALS. We were also delighted to celebrate with the community, commemorating the 10th anniversary of the Ice Bucket Challenge, a global initiative that has significantly raised awareness and funds for ALS. #ALSNEXUS #IceBucketChallenge #CommunityImpact #FightALS

We are pleased to share?the?Reagan-Udall Foundation for the FDA's summary of the?public workshop?they hosted?titled,?"Qualifying Biomarkers to Support Rare Disease Regulatory Pathways." This impactful meeting, held on February?21, 2024, convened experts in MPS to discuss the scientific evidence supporting CSF heparan sulfate levels as a?biomarker to support accelerated approval of new treatments for neuronopathic MPS.?Thank you to the Reagan-Udall Foundation for hosting this workshop and capturing the outcomes of this meeting. Read the?full summary: https://bit.ly/4fjfMB9

We are pleased to share the recent article titled, "Community Consensus for Heparan Sulfate as a Biomarker to Support Accelerated Approval in Neuronopathic Mucopolysaccharidoses" in?Molecular Genetics and Metabolism.?This article highlights the 2020 FDA guidance supporting accelerated approval based on substrate reduction for diseases like neuronopathic MPS, characterized by heparan sulfate (HS) in the brain.?The article reflects the incredibly?collaborative efforts across academia, advocacy, and industry, which culminated in the?Reagan-Udall Foundation for the FDA-hosted workshop in February 2024. Appreciation to the authors and countless others for their unwavering commitment to advocating for science-driven approaches to accelerate the approval path for new treatment options for individuals and families living with MPS diseases. Read the full article here: https://lnkd.in/dNA49ThV Congratulations to the authors of the article:?Joseph Muenzer, UNC at Chapel Hill; Carole Ho, Denali; Heather Lau MD MS, Ultragenyx; Mark Dant, The Ryan Foundation; Maria Fuller,?University of Adelaide; Nidal Boulos, PhD, CCRP, Regenxbio; Patti Dickson, Washington University School of Medicine; Matthew Ellinwood, National MPS Society;?simon jones,?Manchester University NHS Foundation Trust; Eric Zanelli, Allievex Corp.; Cara O'Neill,?Cure Sanfilippo Foundation. #MPS #defeatdegeneration

On June 2nd, Denali team members joined?the San Francisco Giants and Major League Baseball (MLB) to celebrate the fourth annual Lou Gehrig Day, honoring the legendary player and raising awareness for?Amyotrophic?Lateral?Sclerosis?(ALS).? It's inspiring to see the MLB community unite to honor Lou Gehrig and support those affected by?ALS. The camaraderie among fans, players, and organizations demonstrates the power of collective efforts in raising awareness and advancing?ALS?research. ?#LouGehrigDay #EndALS #DenaliTherapeutics #SanFranciscoGiants #MLB