AviadoBio Ltd.

Astellas Pharma and AviadoBio have entered into an exclusive option and licence agreement worth over $2bn for an investigational dementia gene therapy. AviadoBio’s candidate, AVB-101, is currently in phase 1/2 development for patients with frontotemporal dementia (FTD) with progranulin mutations. Astellas will be given the option to receive a worldwide exclusive licence for the development and commercialisation rights to the asset in FTD with progranulin mutations and other potential indications. In exchange, Astellas will make a $20m equity investment in AviadoBio and up to $30m in upfront payments, with AviadoBio also eligible to receive up to $2.18bn in licence fees and milestone payments, plus royalties, if Astellas exercises its option. Accounting for less than one in 30 dementia cases, FTD is a form of early-onset dementia that typically leads to death within three to 13 years from diagnosis. Symptoms typically begin between the ages of 45 and 64 years, and patients commonly experience personality changes, uncharacteristic behaviours, loss of language, apathy and reduced mobility. AVB-101 is delivered as a one-time treatment using a minimally invasive procedure directly to the part of the brain called the thalamus, which has extensive connections throughout the brain, to restore progranulin levels in the frontal and temporal cortex. Astellas’ chief strategy officer, Adam Pearson, said: “We look forward to collaborating with the team at AviadoBio as we expand our gene therapy pipeline to help a broader range of people living with debilitating, neurodegenerative diseases. “AVB-101 represents a truly innovative approach to the treatment of FTD with progranulin mutations and has the potential to be part of the next generation of gene therapy products through the creation of this agreement.” Also commenting on the agreement, AviadoBio’s chief executive officer, Lisa Deschamps, said: “This strategic collaboration will combine our promising gene therapy candidate for FTD with progranulin mutations and delivery expertise with Astellas’ global capabilities in development and commercialisation of gene therapies. “Together, we can further accelerate delivering this investigational medicine to families around the world who so desperately need treatment options for FTD with progranulin mutations and other neurological diseases.”

Astellas receives an exclusive option to license AVB-101, an investigational, AAV-based gene therapy in Phase 1/2 development AviadoBio receives $20 million equity investment, up to $30 million in upfront payments and is eligible to receive up to $2.18 billion in license fees and milestone payments plus royalties if Astellas exercises its option LONDON, UK and TOKYO, Japan I October 8, 2024 I AviadoBio Ltd. (“AviadoBio”) and Astellas Pharma Inc. (TSE: 4503, President and CEO: Naoki Okamura, “Astellas”) today announced an exclusive option and license agreement for AVB-101, an investigational, AAV-based gene therapy in Phase 1/2 development for patients with frontotemporal dementia with progranulin mutations (FTD-GRN). FTD is a devastating form of early-onset dementia that typically leads to death within three to 13 years from diagnosis.  People with FTD commonly experience a rapid decline in executive function (attention control, working memory, problem-solving etc.), uncharacteristic behaviors, loss of language, apathy, and reduced mobility. It is an important cause of dementia in those under the age of 65 and is often underrecognized, and misdiagnosed. 1,2,3,4 Under the terms of the agreement, Astellas will have the option to receive a worldwide exclusive license for the development and commercialization rights to AVB-101 in FTD-GRN and other potential indications. Astellas will make a $20 million equity investment and up to $30 million in upfront payments for the option to license AVB-101. AviadoBio is also eligible to receive up to $2.18 billion in license fees and milestone payments plus royalties if Astellas exercises its option. Lisa Deschamps, CEO, AviadoBio “As we complete dosing of the first cohort of patients in our Phase 1/2 ASPIRE-FTD trial of AVB-101, we are excited about the potential of this collaboration to help address the unmet need that exists today in frontotemporal dementia. This strategic collaboration will combine our promising gene therapy candidate for FTD-GRN and delivery expertise with Astellas’ global capabilities in development and commercialization of gene therapies. Together, we can further accelerate delivering this investigational medicine to families around the world who so desperately need treatment options for FTD-GRN and other neurological diseases.” Adam Pearson, Chief Strategy Officer, Astellas “We look forward to collaborating with the team at AviadoBio as we expand our gene therapy pipeline to help a broader range of people living with debilitating, neurodegenerative diseases. AVB-101 represents a truly innovative approach to the treatment of FTD-GRN and has the potential to be part of the next generation of gene therapy products through the creation of this agreement. Genetic regulation remains a cornerstone of our primary focus strategy at Astellas and this agreement helps us to continue to provide potential solutions for patients in need.” About AviadoBio At AviadoBio, we are relentlessly chasing cures by translating groundbreaking science and precision delivery into life-changing medicines for people living with neurological conditions. With our deep understanding of the brain and suite of proprietary gene therapy platforms and delivery technologies, AviadoBio is working to overcome the challenges of delivering the right drug to the right place. Its innovative, neuroanatomy-led approach is designed to maximize the therapeutic potential of gene therapy to halt or potentially reverse neurodegenerative diseases. AviadoBio was founded on pioneering research from King’s College London and the UK Dementia Research Institute and has a leadership team with extensive gene therapy development, delivery, and commercialization experience which uniquely positions the company for success in bringing transformative medicines to patients. AviadoBio’s investors include New Enterprise Associates (NEA), Monograph Capital, F-Prime Capital, Johnson & Johnson Innovation – JJDC, Inc. (JJDC), SV Health Investor’s Dementia Discovery Fund (DDF), Advent Life Sciences, EQT Life Sciences (Dementia Fund), and LifeArc Ventures. For more information, please visit www.aviadobio.com and follow us on X @AviadoBio and LinkedIn at AviadoBio . About Astellas Astellas Pharma Inc. is a pharmaceutical company conducting business in more than 70 countries around the world. We are promoting the Focus Area Approach that is designed to identify opportunities for the continuous creation of new drugs to address diseases with high unmet medical needs by focusing on Biology and Modality. Furthermore, we are also looking beyond our foundational Rx focus to create Rx+ ® healthcare solutions that combine our expertise and knowledge with cutting-edge technology in different fields of external partners. Through these efforts, Astellas stands on the forefront of healthcare change to turn innovative science into VALUE for patients. For more information, please visit our website at https://www.astellas.com/en . About Astellas Gene Therapies Astellas Gene Therapies is an Astellas Center of Excellence developing genetic medicines with the potential to deliver transformative value for patients. Our gene therapy drug discovery engine is built around innovative science, a validated AAV platform, and industry leading internal manufacturing capability with a particular focus on rare diseases of the eye, CNS and neuromuscular system. Astellas Gene Therapies will also be advancing additional Astellas gene therapy programs toward clinical investigation. Astellas Gene Therapies is based in South San Francisco, with manufacturing and laboratory facilities in South San Francisco, Calif., Sanford, N.C. and Tsukuba, Japan. SOURCE: Astellas Pharma

Today, a brief rundown of news from The Royal Swedish Academy of Sciences and Alnylam Pharmaceuticals, as well as updates from Astellas, Johnson & Johnson and AstraZeneca that you may have missed.David Baker, Demis Hassabis and John Jumper won this years Nobel Prize in chemistry for their respective work on computational protein design and protein structure prediction. A serial entrepreneur, Baker spent decades studying proteins, knowledge that helped him and his team to design unique protein structures. Hassabis and Jumper, both employees of Googles DeepMind group, developed an artificial intelligence model, dubbed AlphaFold, that could be used to predict the structure of virtually all the 200 million or so proteins known to science. Together, their work has helped solve the protein folding problem, which could allow researchers to design better drugs. Ned PagliaruloAlnylam Pharmaceuticals has submitted its medicine vutrisiran for a second approval from the Food and Drug Administration. Vutrisiran is already available in the U.S., sold under the brand name Amvuttra, as a treatment for nerve damage associated with the inherited form of a rare disease called transthyretin amyloidosis. Alnylam is now trying to get the medicine cleared for use in another form of the disease hallmarked by serious, potentially fatal heart problems. The company is using a regulatory fast pass to speed the FDAs review. If approved, vutrisiran would directly compete against a blockbuster Pfizer drug and, potentially, an under-review therapy from BridgeBio Pharma. Jacob BellJohnson & Johnson said Monday it is terminating a trial of its drug delivery system TAR-200 in bladder cancer because treatment didnt outperform a combination of chemotherapy and radiation. The Sunrise-2 trial tested TAR-200, a small device that secretes the chemotherapy gemcitabine, and chemoradiation in people whose bladder cancer had spread into surrounding muscle tissue and who arent having their cancerous tissue removed with surgery. J&J has multiple other studies of TAR-200 underway and plans to ask the Food and Drug Administration for approval next year based on the results of one completed study. Jonathan GardnerGemma Biotherapeutics, a biotechnology company founded by gene therapy pioneer Jim Wilson, is partnering with a public health research institution in Brazils Ministry of Health. Gemma says the institution, known as Fiocruz, has extensive experience manufacturing drugs, and is the main supplier of immunobiologics, biopharmaceuticals and diagnostics to the ministry. The agreement has Fiocruz providing up to $100 million, which will be used for clinical research and manufacturing, with the ultimate goal of more widely distributing rare disease gene therapies in Brazil at a more affordable cost. Jacob BellAstraZeneca said Monday it is paying Hong Kong-based drugmaker CPSC Pharmaceutical Group $100 million for a preclinical oral cholesterol-lowering drug. The drug, codenamed YS2302018, blocks a lipoprotein called Lp(a) that is implicated in cardiovascular disease and is slightly raised by statins. Lp(a) is targeted by other drugs in development such as Amgens olpasiran, Eli Lillys lepodisiran and Ionis and Novartis pelacarsen. Per deal terms, CPSC is eligible for up to $1.9 billion in additional payments based on achievement of development and commercial milestones. Jonathan GardnerJapan-based drugmaker Astellas has bought rights to an experimental gene therapy through a heavily backloaded deal announced Tuesday. Upfront, Astellas is investing $20 million in the therapys developer, AviadoBio, and providing a payment of up to $30 million. But if the program hits certain goals, AviadoBio could take home up to $2.18 billion more through milestone payments and additional licensing fees. The therapy is currently being evaluated in a small clinical trial, as a potential treatment for certain patients with frontotemporal dementia. Jacob Bell '