Tuberous Sclerosis

Events will raise funds for tuberous sclerosis complex (TSC) research and community support programs SILVER SPRING, Md., April 1, 2024 /PRNewswire/ -- As part of its 50th anniversary activities, the TSC Alliance® will present its 23rd Annual Step Forward to Cure TSC® event series, which will take place between April 13 and May 19. Nobelpharma is the 2024 Title Sponsor. Registrants can participate virtually from anywhere in the world or at 13 in-person locations across the United States. Proceeds will benefit the TSC Alliance's research efforts to find new treatments for tuberous sclerosis complex (TSC) and to offer an array of support programs and services. Tuberous sclerosis complex is a rare genetic disease that affects people at all stages of life. TSC causes tumors to grow in different organs and can impair their function, primarily the brain, heart, kidneys, skin, eyes and lungs. Changes in the brain caused by TSC have the biggest impact on quality of life, from seizures and developmental delays to intellectual disabilities, behavioral challenges and autism. In fact, TSC is the leading genetic cause of epilepsy. A strong correlation also exists between TSC and autism—an estimated 40-50% of individuals with TSC have autism spectrum disorder. "Each year, our Step Forward to Cure TSC events offer easy opportunities to support our cause," explained Kari Luther Rosbeck, TSC Alliance President & CEO. "While the events raise much-needed funding, they also provide a platform for thousands of families, individuals and supporters to build camaraderie, learn from each other and educate their local communities about the disease." This year's in-person locations include Fishers, IN (April 13); Nashville, TN (April 27); Salt Lake City, UT (April 27); Renton, WA (April 28); Rock Hill, SC (May 4); Shepherdstown, WV (May 11); Northglenn, CO (May 11); Irwindale, CA (May 18); Chicago, IL (May 18); Tulsa, OK (May 18); West Chester, PA (May 18); Rockville, MD (May 19); and Carrollton, TX (May 19). "Bringing widespread awareness to a rare disease such as TSC requires a strong commitment to growing and sustaining a vibrant community of people who understand—or wish to learn about—the condition and who strive to meet the needs of those affected," said Yoshiki Kida, President & CEO of Nobelpharma America. "The TSC Alliance's Step Forward to Cure TSC campaign offers a fun and healthy way for anyone to have a positive impact on achieving that goal. Nobelpharma America is proud to support the TSC Alliance in this very important effort." To register online and learn more, visit StepFowardtoCureTSC.org. About the TSC Alliance The TSC Alliance is an internationally recognized nonprofit that improves quality of life for everyone affected by tuberous sclerosis complex by catalyzing new treatments, driving research toward a cure and expanding access to lifelong support. For more information, visit tscalliance.org. Contact Jaye Isham Chief Outreach Officer TSC Alliance (301) 562-9890 [email protected] SOURCE TSC Alliance

Aeovian Pharmaceuticals raised $50 million to fuel clinical trials of its lead candidate, AV078, to treat a rare, genetic form of epilepsy. The round, which was led by Hevolution, comes as Aeovian dosed the first participant in a Phase I trial assessing the safety of AV078 in healthy volunteers. The compound is a central nervous system-penetrant selective mTORC1 inhibitor in development for tuberous sclerosis complex (TSC) epilepsy. "TSC is a genetic disorder of mTORC1 hyperactivation,” CEO Allison Hulme said. “AV078 has the potential to be transformative for patients with TSC refractory epilepsy, a patient population in need of therapeutic options with better efficacy and tolerability.”The financing – which saw participation from existing investors Apollo Health Ventures, Sofinnova Investments, venBio, Evotec and b2venture – is expected to provide Aeovian enough runway to finish the Phase I study and prepare for a Phase II trial in adult and paediatric patients with TSC refractory epilepsy.Aeovian also welcomed new faces to its executive team on Thursday. Hevolution’s William Greene joined the board along with Justin Gover, former CEO of GW Pharmaceuticals. Additionally, Micah Zajic was named as chief financial officer; he previously served as chief business officer of Lexeo Therapeutics.

First Cohort of Participants Dosed in Phase 1 Clinical Trial with Lead Development Candidate AV078 Intended for the Treatment of Seizures Associated with Tuberous Sclerosis Complex (TSC) Leadership Team Strengthened with the Appointment of William Greene, MD, CIO at Hevolution, and Justin Gover, Former CEO at GW Pharmaceuticals to the Board of Directors, and Micah Zajic Appointed as CFO $50M Financing Led by Hevolution BERKELEY, Calif., March 28, 2024 /PRNewswire/ -- Aeovian Pharmaceuticals, a clinical-stage biopharmaceutical company developing novel and highly selective therapeutics that potently inhibit the mTORC1 pathway, today announced that it has dosed the first cohort of participants in a Phase 1 clinical trial for its lead development candidate AV078, a first-in-class CNS penetrant selective mTORC1 inhibitor. Supporting this transition into the clinic, the company has also expanded its leadership team with the appointment of seasoned industry veterans William Greene, M.D., Chief Investment Officer at Hevolution, and Justin Gover, former Chief Executive Officer of GW Pharmaceuticals, to the company's Board of Directors, and appointed Micah Zajic as Chief Financial Officer. The company also recently completed a $50 million financing, led by Hevolution. "We are delighted to partner with Aeovian's management team and current investors, who have proven track records of success in drug discovery, development, and commercialization. Aeovian has assembled a compelling platform for the development of selective mTORC1 inhibitors," said Dr. Greene, CIO at Hevolution. "By developing novel small molecules to restore cellular metabolic quality control, Aeovian has several opportunities to address major unmet medical needs for indications spanning orphan neurological diseases to highly prevalent diseases of aging." In the $50M financing, Hevolution was joined by existing investors Apollo Health Ventures, Sofinnova Investments, venBio, Evotec and b2venture. This financing will support the completion of the Phase 1 clinical trial in healthy volunteers and prepare for the start of a subsequent Phase 2 clinical trial in adult and pediatric patients with TSC refractory epilepsy. "TSC is a genetic disorder of mTORC1 hyperactivation. We believe that our first-in-class CNS penetrant selective mTORC1 inhibitor, AV078, has the potential to be transformative for patients with TSC refractory epilepsy, a patient population in need of therapeutic options with better efficacy and tolerability. Beyond AV078, we believe that there is vast potential for our library of proprietary compounds in treating mTORC1-mediated rare and age-related diseases," said Allison Hulme, Ph.D., President and Chief Executive Officer, Aeovian Pharmaceuticals. "The entire Aeovian team welcomes Hevolution, and is thankful for the continued support and commitment of our existing investors as we move to this next exciting stage of the company's development." Dr. William Greene brings over 25 years of experience in leadership positions, which have included founder, biotechnology executive, investor, and clinician. As CEO, he built and led Iconic Therapeutics through discovery, clinical development, and venture financing, which culminated in a successful sale of the company. Dr. Greene spent 12 years at MPM Capital where he was a Managing Director and member of its Investment Committee, responsible for biotechnology and medical technology investments worldwide. He was also founding Chairman and head of the Investment Committee at the Global Health Investment Fund, a groundbreaking impact-oriented venture fund in collaboration with the Gates Foundation, which successfully scaled both investment returns and health impact simultaneously. Dr. Greene earned his BA from Wesleyan University and his MD from UCSF. He was a Robert Wood Johnson Clinical Scholar at Yale and a Howard Hughes Medical Institute Research Scholar at the US National Institutes of Health (NIH). Justin Gover brings 25 years of experience in leadership positions in the biotechnology industry. Mr. Gover served as founding CEO and as a director of GW Pharmaceuticals plc from the company's inception in 1999 until its sale to Jazz Pharmaceuticals plc for $7.2 billion in 2021. From 2018 to 2021, Mr. Gover served on the Board of Directors of the Biotechnology Innovation Organization (BIO). Mr. Gover currently serves on the Board of Directors for Xenon Pharmaceuticals, CURE Epilepsy and Rady Children's Institute for Genomic Medicine. Mr. Gover holds an M.B.A. from the INSEAD business school in France and a B.Sc. (Hons) from Bristol University, UK. "I am delighted to be joining the Aeovian Board at this exciting time as the company enters the clinic. I have a deep commitment to the epilepsy community and to patients with TSC and believe that AV078 has the potential to be a valuable new treatment option for this high need population," said Justin Gover. "I am impressed by the breadth of potential applications for Aeovian's science and by the caliber of the Aeovian team and look forward to contributing to the company's future." "On behalf of our Board of Directors, I am delighted to welcome William and Justin to our Board as they bring diverse and relevant experience from various leadership roles within the biotechnology industry and we look forward to their contributions as we navigate the company's next phase of growth," said Richard Gaster, M.D., Ph.D., Chairman of the Aeovian Board of Directors. Micah Zajic has been appointed Chief Financial Officer at Aeovian. Mr. Zajic brings nearly 20 years of experience in biotech corporate development, healthcare capital markets and investment banking to the company. Mr. Zajic most recently served as Chief Business Officer and Chief Financial Officer at LEXEO Therapeutics (NASDAQ). Prior to this he served as Chief Financial Officer of SQZ Biotechnologies (NYSE). He began his career at Morgan Stanley in the Global Capital Markets and Healthcare Investment Banking Groups, raising capital and executing strategic transactions for companies across the healthcare industry. In addition, Mr. Zajic worked in corporate development roles at Alexion Pharmaceuticals and MeiraGTx, focusing on the evaluation and execution of external partnerships, license agreements and acquisitions. About Aeovian Aeovian Pharmaceuticals is a clinical-stage biopharmaceutical company developing targeted and highly selective small molecules to restore cellular metabolic quality control, thereby addressing the dysregulated growth and hyperactive signaling found in certain rare genetic and age-related diseases. Our lead development candidate, AV078, is a first-in-class CNS penetrant selective mTORC1 inhibitor being developed for the treatment of TSC refractory epilepsy and is currently being evaluated in a Phase 1 trial. TSC is a rare genetic disorder caused by the hyperactive signaling of mTORC1. Beyond AV078, we have developed a proprietary library of small molecules, which are potent and selective inhibitors of either mTORC1 or CD38 and have the potential to precisely target the underlying biology of multiple rare and age-related diseases. For more information, please visit our website at . Contact: Micah Zajic Chief Financial Officer ir@aeovian.com