Denali Therapeutics

We are pleased to share that dosing has begun in the Denali Phase 2a "BEACON" study of the investigational therapeutic BIIB122 in individuals with LRRK2-associated Parkinson's disease. Our hope is that targeting LRRK2, in both the BEACON study and the ongoing Phase 2b LUMA study, will lead to an effective treatment for all people living with Parkinson’s disease. For more information: https://lnkd.in/gm6gS2z8   Learn more about the BEACON study here: https://lnkd.in/gWqeAmVD Learn more about the LUMA study here: https://lnkd.in/gyxwRaK3   #parkinsonsdisease

We reported our third quarter financial results and business highlights. Read our press release here: https://lnkd.in/gFC_RC8Y

Thank you to Cure Sanfilippo Foundation for bringing everyone together at ADVANCE 2024! We appreciate the opportunity to share our work in MPS IIIA and learn from the day-to-day experiences of the inspiring families and experts in this community.

We had the pleasure of attending Project Alive's Family Fair this past weekend to connect with individuals, families and advocates. Hearing from those who live with Hunter syndrome every day helps guide our work and is truly motivating. A huge thank you to Project Alive for hosting this event and providing a special opportunity to meaningfully connect with the community.

#TeamDenali participated in the Alzheimer's Association Utah Chapter and the Alzheimer's Association of Northern California & Northern Nevada Walk to End Alzheimer’s events. As a company dedicated to advancing research in Alzheimer’s disease, we are genuinely inspired by the opportunity to come together with local communities for these annual events, raising awareness and support in the fight to end Alzheimer’s.

Advancing Rare Disease Therapies Through an FDA Rare Disease Innovation Hub was the topic of a public meeting convened today by the Reagan-Udall Foundation for the FDA, in collaboration with FDA Rare Disease Innovation Hub. Our Chief Medical Officer Carole Ho had the honor of speaking about key challenges in drug development for rare diseases and opportunities to collaborate with patient advocates, academic researchers, industry colleagues, and FDA to forge a more efficient path forward for the benefit of patients. See the meeting agenda and slides here: https://lnkd.in/epxcdyZk #raredisease #MPS #defeatdegeneration

Members of our team were thrilled to participate in The Michael J. Fox Foundation for Parkinson's Research Tour de Fox Wine Country! It was inspiring to join so many passionate individuals riding to support research and raise awareness for Parkinson’s disease. #TourDeFox #ParkinsonsAwareness

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!