Axonis Therapeutics, Inc.

Boston biotech Axonis Therapeutics has raised a $115 million Series A to test whether its oral drug AXN-027 can help patients with focal epilepsy and diabetic neuropathic pain. The financing, disclosed Wednesday morning, will get the biotech into the clinic around the end of this year, or about the same time Ovid Therapeutics expects to begin human trials with a similar approach by way of an AstraZeneca platform it licensed. Both companies are looking at KCC2, a CNS chloride transporter that is key to electrical signaling in the brain. KCC2 allows for neuronal GABA signaling. The GABA pathway has been targeted by multiple drugmakers , including Sage’s postpartum depression medicine Zurzuvae , but biotechs have yet to enter the clinic with a focus on KCC2, CEO Joanna Stanicka told Endpoints News . “Often GABA treatments can induce global inhibition, which leads to this cognitive impairment and sedation often reported by patients,” Stanicka said, adding that focused inhibition should avoid those side effects. The 13-employee biotech also said Wednesday that it added Donald Manning as chief medical officer and Jeff Imbaro as operating chief. Manning previously held the same post at Travecta, Pacira BioSciences and Adynxx. Imbaro was in the same role at stroke-focused Elevian, and before that he was a founding member of Vertex-acquired Semma Therapeutics. Axonis was founded by spinal cord injury patient advocate and board observer Bob Yant about 15 years ago, but it didn’t become “operational” until 2019, Stanicka said. Prior to the Series A, Axonis had raised about $34 million in seed funding and about $9 million in government and foundation money, Stanicka said. Its investors include Cormorant Asset Management, venBio, Sofinnova Investments, Merck’s corporate venture arm and others. The startup anticipates the Series A will fund AXN-027 through Phase 2 proof-of-concept studies, she said.

SHORT HILLS, N.J., June 10, 2024 /PRNewswire/ -- Maggie Goldberg, President and CEO, Christopher & Dana Reeve Foundation, discusses the launch of SCI Ventures, the world's first philanthropic venture fund and groundbreaking global initiative dedicated to catalyzing the funding and commercialization of innovative treatments for spinal cord injury (SCI). Christopher & Dana Reeve Foundation Executive Memo Continue Reading Maggie Goldberg Life's defining moments are often strung together on invisible thread, the path between them illuminated only after the world shifts. A family facing a child's devastating injury hatches an ambitious plan to pursue a cure for paralysis from a living room in New Jersey. A celebrated actor embarks on a quest to transform spinal cord injury research. Lives intersect. Missions merge. A movement quietly grows. Mothers and veterans and grandchildren join the effort, entering marathons and hosting fundraisers and fighting to give loved ones the hope they deserve. Year after year, they raise the money to support the labs, to sustain the science, to defy the skeptics until, one by one, the breakthroughs deemed impossible arrive. Sometimes we're lucky enough to recognize these milestones in real time, glimpsing the spark before it ignites. Today, the Christopher & Dana Reeve Foundation is thrilled to announce the launch of SCI Ventures, the world's first philanthropic venture fund dedicated to catalyzing the funding and commercialization of innovative treatments for spinal cord injury (SCI). This groundbreaking global initiative — kicking off with $27 million and an anticipated $40 million by year's end — is a direct response to the momentum currently transforming the field of spinal cord injury research. After decades of incremental gains, long awaited therapies are finally within reach. The notion that paralysis is treatable — that blood pressure and cardiovascular function might be regulated by epidural stimulation, that new pathways can bridge scar tissue — is no longer simply a dream of determined families. But moving these life-changing treatments beyond the lab demands a new kind of collaboration. SCI Ventures was co-founded in a unique partnership between the Reeve Foundation and leading spinal cord injury organizations including Wings for Life, the International Spinal Research Trust ("Spinal Research"), the Promobilia Foundation and Shepherd Center. Its mission is to bridge the gap from bench to bedside, supercharging innovative lab breakthroughs, boosting early-stage companies, and building financial incentives to bring treatments to market. Modeled on the Cystic Fibrosis Foundation and the JDRF T1D Fund for Type 1 diabetes, SCI Ventures grew out of the Reeve Foundation's pioneering investments in ONWARD Medical and AXONIS Therapeutics. The fund will build on the success of these investments, targeting therapies that restore function alongside emerging technologies with the potential to fully repair the spinal cord. Though Reeve and the co-founding organizations will provide knowledge gathered across decades of experience with spinal cord injury, SCI Ventures is an independently managed fund. Founding Managing Director Adrien Cohen, a tech entrepreneur and investor who co-founded Tractable and Lazada — two 'unicorn' startups whose worth climbed to $1 billion — will direct investments in consultation with neuroscientists from around the world. All profits from investment gains will be channeled back into the fund, accelerating the pace of discovery to meet the potential of this moment. For so many of us, the launch of SCI Ventures is an extraordinary milestone that's decades in the making. When 17-year-old Henry Stifel sustained a spinal cord injury in a car accident in 1982, there was nowhere for his parents to turn for help, no organization with its gaze trained on a cure. So, Hank and Charlotte Stifel decided to build their own. The Stifel Paralysis Research Foundation set up shop at a bridge table in a spare bedroom later that year. Hank, Charlotte, and friends dedicated to helping Henry and all those impacted by spinal cord injury immersed themselves in academic journals and began scouring the world for researchers able — and willing — to study spinal cord injury. The plan was simple. Fund new labs. Recruit promising scientists. Prove the naysayers wrong. And they did. By the time Christopher Reeve was paralyzed in a horseback riding accident in 1995, the Stifels' pursuit of progress had laid a foundation for transformative change; Reeve's embrace of the mission and unyielding quest to find a cure for paralysis would provide the spark that launched a new era in research. The newly named Christopher Reeve Foundation rocketed into the public consciousness with Chris at its center. He increased the Foundation's reach as he traveled the country spotlighting innovative labs, pushing for increased federal funding, and challenging Congress to support stem cell research. With his voice rising above the whoosh of his ventilator — and the unmistakable smile curling his lips — Reeve captured the public's imagination, his vision for spinal cord injury research redefining what was deemed possible. Over four decades, the Reeve Foundation seeded the field with nearly $145 million. Our investments critically boosted the work of early pioneers, including UCLA scientist Reggie Edgerton whose lab demonstrated that damaged spinal cords could adapt and be retrained with epidural stimulation. As the scope of research expanded, Reeve fueled efforts to restore long sought functional gains, including hand dexterity, bladder function and blood pressure regulation; helped launch The Big Idea, the first comprehensive clinical trial studying epidural stimulation in patients with paralysis; and advanced an understanding of spinal cord biology that underpins the breakthroughs of recent years. And we're not finished yet. Today, 20 years since Christopher's passing, the launch of SCI Ventures represents a critical next step in his and the Reeve family's determination to identify cures for this injury and address the subsequent medical challenges that severely impact quality of life for those living with paralysis. The progress in spinal cord injury research — the very success that inspired SCI Ventures — stems from the determination of all the families since the Stifels and the Reeves who refused to give up hope. But until transformational treatments are in hand, there is still much to be done. As SCI Ventures bridges the critical gap between bench and bedside, the Reeve Foundation will continue the work that brought us to this moment. In the coming year, we will tap the expertise of our Spinal Cord Injury Advisory Board, comprised of leading scientists from across the country, to help identify results-driven research that will yield the greatest impact for community members. We will seek out innovative new scientists and fund labs gaining speed. This summer, we will cohost the second Reeve Foundation Symposium, linking scientists and academics with industry peers and investors — sparking new conversations and watching for flickers of light. Like the Stifel and Reeve families — like so many of you — we will imagine a brighter future and work tirelessly until it arrives. To learn more about SCI Ventures, visit SCIVentures.com and follow SCI Ventures on LinkedIn here. About the Christopher & Dana Reeve Foundation The Christopher & Dana Reeve Foundation is dedicated to curing spinal cord injury by funding innovative research and improving the quality of life for individuals and families impacted by paralysis. Additionally, through a cooperative agreement with the Administration for Community Living, the Reeve Foundation's National Paralysis Resource Center (NPRC) promotes the health, well-being, and independence of people living with paralysis, providing comprehensive information, resources, and referral services assisting over 128,000 individuals and families since its launch in 2002. The Reeve Foundation is committed to elevating our community's voices and needs to achieve greater representation and independence. We meet all 20 of the Better Business Bureau's standards for charity accountability and hold the BBB's Charity Seal. For more information, please visit ChristopherReeve.org or call 800-225-0292. Media Contact: Julia Leonard, Media Specialist 973-933-7222 [email protected] SOURCE Christopher & Dana Reeve Foundation

KENNEDY SPACE CENTER, Fla., Dec. 22, 2023 /PRNewswire/ -- Dozens of science and technology development payloads returned to Earth from the International Space Station (ISS) on December 22 as SpaceX's 29th Commercial Resupply Services (CRS) mission, contracted by NASA, successfully concluded. More than 30 payloads sponsored by the ISS National Laboratory® returned on this mission, many of which were life science investigations aimed at benefiting humanity by improving care for patients on Earth. Here is a quick look at some of the payloads that returned on SpaceX CRS-29: Researchers from the Naval Research Laboratory sent microbes to the orbiting laboratory to better understand how microgravity and space radiation affect their ability to produce melanin. Results could enable the production of new melanin variants and ultimately new biomaterials with valuable applications in drug delivery and protective coatings for spacecraft and spacesuits. An investigation from the University of California, San Francisco (UCSF) that was funded by the U.S. National Science Foundation (NSF) focused on aging and liver regeneration. Inspired by previous research, the UCSF team utilized 3D immune-liver tissue chips in space to better understand the relationship between immune system aging and the ability of liver cells to regenerate, which could lead to novel therapies on Earth. An investigation from the University of California, San Diego (UCSD), which also builds on previous space station research, studied microgravity's effects on stem-cell derived brain organoids. The project leveraged accelerated aging in space to better understand how cognitive decline happens in the human brain. Results could lead to new ways to treat dementia, Alzheimer's, and other neurodegenerative conditions. Earlier this summer, a team from Emory University continued research on station examining how microgravity affects the growth and function of heart muscle cells as they mature into tissue-like structures. Findings will help advance heart disease modeling and could contribute to the development of new therapies for patients on Earth. A project from biotech startup Axonis, which also launched earlier this summer, examined how microgravity affects the maturation of human brain cells as they form three dimensional spheroids that mimic certain aspects of the human brain. Findings will help improve disease modeling and aid in the development of new treatments for neurological disorders. The ISS National Lab enables access and opportunity for researchers to leverage this unique orbiting outpost for the benefit of humanity and to enable commerce in space. To learn more about ISS National Lab-sponsored investigations that flew on SpaceX CRS-29, please visit our launch page. To download a high-resolution photo for this release, click here. About the International Space Station (ISS) National Laboratory: The International Space Station (ISS) is a one-of-a-kind laboratory that enables research and technology development not possible on Earth. As a public service enterprise, the ISS National Laboratory® allows researchers to leverage this multiuser facility to improve quality of life on Earth, mature space-based business models, advance science literacy in the future workforce, and expand a sustainable and scalable market in low Earth orbit. Through this orbiting national laboratory, research resources on the ISS are available to support non-NASA science, technology, and education initiatives from U.S. government agencies, academic institutions, and the private sector. The Center for the Advancement of Science in Space™ (CASIS™) manages the ISS National Lab, under Cooperative Agreement with NASA, facilitating access to its permanent microgravity research environment, a powerful vantage point in low Earth orbit, and the extreme and varied conditions of space. To learn more about the ISS National Lab, visit our website. As a 501(c)(3) nonprofit organization, CASIS accepts corporate and individual donations to help advance science in space for the benefit of humanity. For more information, visit our donations page. SOURCE International Space Station National Lab