AZAFAROS B.V.

This weekend is full of events to support the GM1 and GM2 gangliosidoses community, and AZAFAROS B.V. is looking forward to taking part. In Germany, the Hand in Hand gegen (against) Tay-Sachs and Sandhoff patient organisation is holding its annual family conference in 🚩Würzburg. Chris Freitag, Chief Medical Officer at Azafaros, will join patient families at the conference while our investigator, Dr Andreas Hahn, will give an update on the clinical development plan for our lead asset, #nizubaglustat. Simultaneously, in Spain, MPS Lisosomales, an umbrella organization campaigning for many lysosomal diseases including GM1 and GM2 gangliosidoses, is also holding its family conference in 🚩Barcelona. Laura López de Frutos, our Medical Director, will discuss new therapeutic options in a roundtable discussion. The Azafaros team is grateful for the invitations to attend, thankful for the collaborations, and is looking forward to meeting with all these communities in person. #GM1, #GM2, #NPC, #collaboration, #rarediseases, #patientengagement

Today marks the start of an important month for the Niemann-Pick disease community, as October is #NiemannPickAwarenessMonth. Raising awareness about a disease is important because it promotes prevention, early diagnosis and intervention, and provides support to patients and their families. When the focus is on #rarediseases however there is a particular significance; lack of awareness is partially responsible for delays in diagnosis and the uncertainty that families suffer. Discussing NP diseases and raising awareness is of great importance to the community in order to improve diagnosis, disease management and treatment. The ultimate goal is to improve the lives of patients and their family members. AZAFAROS B.V. supports the Niemann-Pick community, and we are proud to collaborate with all NPC patient groups around the world. #collaboration #awareness #NPC, #NNPDF, #niemannpickawareness, #raredisease

AZAFAROS B.V. is proud to be one of the silver sponsors of the Latin American Course on Lysosomal Storage Diseases (LAC-LSD). The course, organized by the institute “Genética para Todos” takes place all this week in 🚩Foz do Iguaçu (Brazil). This training is aimed at healthcare providers, particularly those new to the LSD field, and includes sections on biochemistry, genetics, the clinical symptoms of several lysosomal diseases, and information on current treatment options. The training is chaired by Professor Roberto Giugliani, and its scientific committee includes experts working across South and North America. #collaboration, #rarediseases, #awareness, #training, #LSDs, #sponsorship

The Cure GM1 Foundation is today holding its International GM1 Virtual Community Conference, bringing families, researchers, healthcare providers, and biotech companies together to share knowledge and discuss advances in #GM1 research. AZAFAROS B.V. is proud to be part of the GM1 community and to provide support by attending this meeting. Our CMO, Chris Freitag, will present new data gathered from our #PRONTO study, an international prospective natural history study aimed at improving the knowledge of the neurological progression of late-infantile/juvenile forms of GM1 and GM2 Gangliosidoses. We look forward to a mutually productive and successful collaboration, and to seeing all members of this special community. #curegm1, #collaboration, #rarediseases, #research, #PRONTO, #GM1, #GM2 #clinicaltrials

🚀We are delighted to announce encouraging early efficacy trends in the extension phase of our 🌈RAINBOW Phase 2 safety/PK trial for GM2 gangliosidosis and Niemann-Pick disease type C, ⏩ paving the way to start our Phase 3 trial. The results were presented at the #SSIEM2024 conference last week.   💈 Our 🌈RAINBOW trial was designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of nizubaglustat in patients with GM2 and NPC 💈 Data showed that our compound had a positive safety profile and was well tolerated in the 13 study participants 💈The extension phase of 🌈RAINBOW and initial, unblinded data from 10 patients showed 8 had improved or stabilized SARA (Scale for the Assessment and Rating of Ataxia) scores. Additionally, a reduction in seizure frequency (daily at baseline) was reported 💈Data from the 🌈 RAINBOW study will be used to inform the target dose for our planned Phase 3 pivotal studies, 🎯 due to be initiated in 2025 💈At SSIEM, we also presented our 12-month follow-up data from our PRONTO trial – the largest prospective natural history study in late-infantile/juvenile GM1 and GM2 gangliosides   We are immensely thankful to everyone who contributed to both our 🌈 RAINBOW and PRONTO trials.   📢 To see the full announcement, go to our website: https://lnkd.in/e4Bbcud4   💬Azafaros CEO, Stefano Portolano, MD, said: “The RAINBOW trial results support the positive safety profile of nizubaglustat and provide us with encouraging early efficacy trends for the compound. We want to thank all the physicians, patients, patient families, and patient organisations who contributed to this important study.” #Biotech, #clinicaltrials, #rarediseases, #Ataxia, #GM1, #GM2, #NPC, #nizubaglustat, #azasugar

Today at #SSIEM2024, Dr. Roberto Giugliani, Principal Investigator in Brazil for our PRONTO and RAINBOW studies, and AZAFAROS B.V. Regional Medical Director, Laura López de Frutos, will present 👉 2 posters. One on 12-month follow-up data from our prospective natural history study of late-infantile and juvenile GM1 and GM2 gangliosidoses 📜(PRONTO), and the other onthe results of our Phase 2 study, 🌈RAINBOW.   Dr. Giugliani and Dr. López de Frutos will be happy to answer any questions during this inspiring and informative poster session, which will be held between ⌚12:10 and 13:40 in the 📍Poster Room.

The AZAFAROS B.V. Team is attending the #SSIEM2024 Annual Symposium, a gathering of professionals from all disciplines with an interest in inherited metabolic diseases. As a company developing treatment options for patients with #GM1, #GM2, and Niemann-Pick disease type C (#NPC), we look forward to discussing our program with experts from around the world. Our CEO, Stefano Portolano, MD, will be joined at the conference with members of our medical and patient advocacy team. 👉 Visit our booth at 📍B01 on the ground floor to meet our team and hear about our ongoing and future clinical trials. ✴️The following information about our Azafaros program will be available during the poster sessions: 🔈Blinded safety data of nizubaglustat Phase 2 study for GM2 gangliosidosis and NPC disease ➡️ Poster PO-200 🔈One year of follow-up data from the natural history study of late-infantile/juvenile GM1 and GM2 gangliosidoses ➡️ Poster PO-220 #collaboration, #rarediseases, #teamwork, #LSDs

The #SLEIMPN meeting is one of the largest conferences on metabolic diseases and neonatal screening in South America. This year it will be held in Punta del Este in Uruguay on 22-25 October, and members of the clinical team at AZAFAROS B.V. will be there to present the results of two clinical trials. Our #PRONTO study, a prospective natural history study of the disease progression of patients with late-infantile/juvenile onset #GM1 and #GM2 gangliosides, will be presented as a poster with an update on patient follow-up. Our #RAINBOW study, a Phase 2 trial carried out in Brazil to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and explore the efficacy of our lead asset, #nizubaglustat, will also be presented as a poster. #SLEIMPN2024 (XIII Congreso de la Sociedad Latinoamericana de Errores Innatos del Metabolismo y Pesquisa Neonatal), will be a great opportunity for the Azafaros team to impart knowledge of the natural history of GMx diseases, and to share our progress with nizubaglustat, a small molecule being investigated in lysosomal neurodegenerative diseases such as NPC, GM1, and GM2. At the same time, it is a fantastic opportunity to engage with and strengthen our bond with the Central and South American rare disease community. See you there! #Azafaros, #SLEIMPN, #conferencing, #collaboration, #rarediseases, #GM1, #GM2, #NPC, #clinicaltrials, #biotech

At the beginning of 2024, the State of Florida officially recognised 10 August as Tay-Sachs Disease Awareness Day. AZAFAROS B.V. marked this important event by participating in the podcast “Rare 5, Rare Spotlights: Explorando las Enfermedades raras en 5 minutos,” with our Medical Director, Laura López de Frutos, answering questions on the disease. The @Rare5potlights is an initiative of Dr José Elías García Ortiz, senior researcher and chief of the genetics laboratory at the Centro de Investigación Biomédica de Occidente (CIBO) in México. The objective of the initiative is to spread awareness of rare diseases across the Spanish-speaking community. You can listen to the podcast here: https://lnkd.in/e-JEnCwd

We look forward to sharing important data from PRONTO, the largest prospective study of the natural history of gangliosidoses, and the exciting results of RAINBOW, a phase 2 study in GM2 and Niemann Pick type C patients. See you in beautiful #Porto!