Orsobio, Inc.

Six months after launching with a mega-round of financing, Seaport Therapeutics has raised $225 million more to advance a lead drug candidate that could overcome some limitations of currently available treatments for depression. Seaport’s platform technology, called Glyph, designs oral drugs that leverage the lymphatic system to deliver the medicine in a way that makes more of the active pharmaceutical ingredient available in the body to confer its therapeutic effect. The Boston-based company also says its drugs reduce complications such as elevated liver enzyme levels, a sign of drug toxicity that can limit the use of some molecules. Lead Seaport program SPT-300 is a prodrug, a compound that converts inside the body into the active drug allopregnanolone. This neurosteroid modulates the GABA-A receptor to spark effects on the central nervous system. Seaport is advancing the drug into Phase 2b testing as a potential treatment for major depressive disorder with or without anxious distress. Seaport says the new capital will go toward advancing its pipeline through important clinical milestones. The company also plans to further advance the capabilities of its Glyph technology platform. presented by Health IT Accelerating Claim Processing: Strategies to Shorten the Life of a Claim These strategies and practices can significantly shorten the life cycle of claims, leading to quicker resolutions and improved financial outcomes. By Greenway Health Seaport was spun out of PureTech Health, a company that forms startups around platform technologies. In April, Seaport launched, revealing its science, its lead drug candidate, and $100 million in funding. That Series A round was led by Arch Venture Partners and Sofinnova Investments. General Atlantic led Seaport’s new Series B financing. Other participants in the latest round included T. Rowe Price Associates, Foresite Capital, Invus, Goldman Sachs Alternatives, CPP Investments, and other unnamed new investors. Founding investors Arch and Sofinnova also joined the round, as did Third Rock Ventures and PureTech Health. Seaport is part of a bevy of life science companies that have secured new capital. Here’s a recap of recent biotech financing news: —Radiopharmaceuticals developer Alpha-9 Oncology closed a $175 million Series C round to support its pipeline, including A9-3202, which began a Phase 1 test earlier this year in advanced melanoma. Lightspeed Venture Partners and Ascenta Capital led Alpha-9’s new financing. —AvenCell Therapeutics has $112 million in Series B funding to continue Phase 1/2 testing of AVC-101, a CAR T-therapy that can be switched “on” or “off” to address safety and efficacy challenges of currently available cell therapies, which are always “on.” AvenCell was formed in 2021 by Intellia Therapeutics and Cellex Cell Professionals. The company’s latest financing was led by Novo Holdings. Sponsored Post The Future of Hospitals and Pharma Companies Will Depend on Strength of Healthcare Analytics Insights PurpleLab® stands out from others in this sector by providing its data analytics services to several different groups of users across healthcare and pharma companies. By Stephanie Baum —Tolerance Bio launched with $17.2 million to support development of allogeneic stem cell-based cell therapies. These therapies are intended to preserve the thymus, the organ that trains T cells to defend against threats and preserve autoimmunity. The Philadelphia-based biotech’s seed financing, led by Columbus Venture Partners, will be used to advance toward the clinic with potential therapies for immune diseases. —March Biosciences, spun out of the Center for Cell and Gene Therapy, unveiled $28.4 million in Series A financing. Lead program MB-105 is a CAR T-therapy designed to target CD5, a protein expressed on cancerous T cells. A Phase 1 test is underway enrolling patients with refractory T cell lymphoma and leukemia. Mission BioCapital and 4BIO Capital led March Bio’s financing. —Be Biopharma closed $82 million in financing as the cell therapy developer moves into the clinic with BE-101, a potential treatment for hemophilia B. The biotech’s therapies are made by engineering B cells to produce therapeutic proteins. Be Bio last raised money in 2022, a Series B financing that totaled $130 million. —Terray Therapeutics, a biotech that uses generative AI to discover and develop small molecule drugs, unveiled a $120 million Series B financing. Los Angeles-based Terray will use the capital to advance internal programs into clinical trials for indications that were not disclosed. The financing was led by new investor Bedford Ridge Capital and earlier investor NVenture, the investment arm of NVIDIA. Here’s more about Terray and its technology platform, tNova, which it unveiled in 2022. —Genetic medicines have been difficult to deliver to the kidney. Judo Bio is developing oligonucleotide drugs that target a particular receptor to reach the organ, and the Cambridge-based startup unveiled $100 million in seed and Series A financing to advance its research toward the clinic. The Series A round was co-led by Atlas Venture, TCG, and Droia Venture. —Purespring Therapeutics, a developer of gene therapies for kidney disease, raised £80 million (about $105 million). The London-based company’s therapies target the podocyte, a special cell implicated in 60% of renal diseases. Proceeds will be used to advance lead program PS-002 into a Phase 1/2 clinical trial for immunoglobulin A nephropathy, a rare disease currently treated with chronically administered therapies. Sofinnova Partners led Purespring’s Series B financing. —Artificial intelligence biotech company Basecamp Research revealed a $60 million Series B financing to scale its collection of data and strengthen its AI capabilities. The London-based startup also announced a multi-year collaboration with David Liu of the Broad Institute of MIT and Harvard. Basecamp said this partnership will research new approaches to programmable genetic medicines with potential applications in a range of diseases. —City Therapeutics launched with $135 million to develop next-generation RNA interference (RNAi) drugs. The company says its engineering platform can design therapies in smaller sizes that have novel cellular mechanisms and improved potency, efficiency, and specificity. The Cambridge-based biotech, co-founded by scientists involved in the first generation of RNAi therapies, expects to enter the clinic by the end of 2025. Arch Venture Partners led City’s Series A round. —Resolution Therapeutics raised £63.5 million to advance to Phase 1/2 testing with RTX001, an engineered autologous macrophage cell therapy that could offer anti-fibrotic and anti-inflammatory effects in patients with end-stage liver disease. The company, which maintains operations in London and Edinburgh, Scotland, said this trial will be conducted in the United Kingdom and Spain. Syncona led the Series B round, which will also support research in other inflammatory fibrotic conditions such as graft-versus-host disease and lung fibrosis. —Triveni Bio raised $152 million to support a pipeline whose most advanced program, TRIV-573, is a bispecific antibody that blocks two targets associated with atopic dermatitis. The Watertown, Massachusetts-based biotech aims to file an investigational new drug application for this program in the first quarter of 2025. Triveni launched last year supported by a $92 million Series A financing. Goldman Sachs Alternatives led the company’s Series B. —Kailera Therapeutics launched with $400 million to support its GLP-1 and GIP receptor agonist drugs for metabolic indications, including obesity. Kailera’s four programs were licensed from China-based Jiangsu Hengrui Pharmaceuticals. The Series A financing was co-led by Atlas Venture, Bain Capital Life Sciences, and RTW Investments. —Radiopharmaceutical developer Aktis Oncology unveiled $175 million in financing to support development of a pipeline led by AKY-1189, an alpha radiotherapy that targets Nectin-4, a tumor-associated antigen found in a range of solid tumors. The Series B round was led by RA Capital Management and co-led by RTW Investments and Janus Henderson Investors. Boston-based Aktis emerged from stealth in 2021 backed by $72 million in Series A financing. —Genespire raised €46.6 million (about $52 million) to advance to Phase 1/2 testing with GENE202, an in vivo gene therapy in development for methylmalonic acidemia, a rare inherited disorder that impairs the metabolism of certain amino acids and fats. The Milan, Italy-based startup’s Series B round was co-led by Sofinnova Partners, XGEN Venture, and CDP Venture Capital. —Vaccines developer Vicebio unveiled $100 million in financing as it begins a Phase 1 test of VXB-241, a bivalent vaccine designed to address both respiratory syncytial virus (RSV) )and human metapneumovirus (hMPV). Data are expected in mid-2025. The next Vicebio program is VXB-251, a trivalent vaccine for RSV, hMPV, and parainfluenza virus 3. The London-based company’s Series B round was led by TCGX. —GC Therapeutics launched with $65 million in financing. The biotech’s next-generation cell therapies come from a platform technology called TFome, which programs induced pluripotent stem cells. GC’s initial therapeutic areas of focus include gastrointestinal, neurological, and immunological diseases. Cormorant Asset Management led Cambridge, Massachusetts-based biotech’s Series A financing. —Neuroscience startup Nura Bio extended its Series A financing by $68 million to proceed to Phase 2 testing with an oral small molecule designed to block SARM1, an enzyme associated with axonal damage. It faces potential competition from Eli Lilly, which is in early clinical development with a drug that goes after the same target. Nura Bio’s founding investor The Column Group led the startup’s new financing. —F2G raised $100 million for olorofim, which is in late-stage development to treat aspergillosis and other invasive fungal infections. Olorofim is from a novel class of antifungal agents that work by inhibiting the pyrimidine synthesis pathway. The cash will enable the Manchester, U.K.-based company to complete late-stage clinical development, submit regulatory applications, and prepare for U.S. commercialization of the drug. The new financing was led by new investor AMR Action Fund and co-led by ICG. —T cell engagers first reached patients as cancer treatments. Candid Therapeutics aims to bring such drugs to autoimmune disorders, and its efforts are backed by more than $370 million in financing. Candid’s drug candidates came from acquiring and merging two biotechs, Vignette Bio and TRC 2004. The Candid pipeline includes two T cell engagers for cancer now being repositioned for development in autoimmune diseases. These therapies are designed to selectively deplete B cells that drive disease. —Startup creator Flagship Pioneering unveiled its newest company, Mirai Bio. Mirai’s platform technology is applied to the development of genetic medicines for the company’s partners working across a range of therapeutic areas and therapeutic modalities. It says its platform technology unlocks delivery of a drug to any tissue and cell type, optimize design of the drug cargo, and facilitates manufacturing. Like other startups that come from Flagship, Mirai is backed by the customary $50 million in financing. —Superluminal Medicines, a company developing small molecule drugs that target elusive G protein-coupled receptors (GPCRs), closed $120 million in financing. The Boston-based startup says its drugs target membrane receptors that mediate cell signaling and response processes fundamental to human physiology. These small molecule come from a generative biology and chemistry platform technology. The new capital will be used to advance toward the clinic in undisclosed indications. RA Capital Management led the Series A financing. —Riding strong investor interest in metabolic disorder drugs, OrsoBio closed $67 million to advance a pipeline that includes programs for type 2 diabetes, obesity, and the fatty liver disease MASH. The Menlo Park, California-based startup’s drugs address pathways associated with energy metabolism. The Series B round, led by Ascenta Capital and Woodline Partners, comes less than a year after the company unveiled $60 million in Series A financing. Photo by Flickr user Massachusetts Office of Travel & Tourism via a Creative Commons license

ACMSD inhibition offers potential benefit across a broad range of liver and kidney diseases MENLO PARK, Calif.--(BUSINESS WIRE)-- #OrsoBio --OrsoBio, Inc. (“OrsoBio” or “the Company”), a clinical-stage biopharmaceutical company developing treatments for obesity and obesity-associated disorders, together with a team of academic researchers from Europe, Japan, and the United States, published promising preclinical data regarding the potential of ACMSD inhibition in animal and cellular models of metabolic dysfunction-associated steatotic liver disease/steatohepatitis (MASLD/MASH). In addition to enhancing synthesis of NAD + , a key co-factor in diverse bodily processes, the researchers discovered that ACMSD inhibition prevents DNA damage and cellular redox stress, leading to significantly reduced inflammation and fibrosis due to MASH. “We see tremendous therapeutic potential in ACMSD inhibitors across a spectrum of metabolic and inflammatory hepatic and renal diseases,” said Mani Subramanian, MD, PhD, Chief Executive Officer of OrsoBio. “The mechanism of action of ACMSD inhibition is highly complementary to our broader portfolio, including mitochondrial protonophores and an ACC2 inhibitor, as they target causal drivers of inflammation and fibrosis. Their potential therapeutic impact extends well beyond the obesity-associated diseases that are the focus of the rest of our pipeline.” The study, published in the Journal of Hepatology , investigates the mechanistic role and therapeutic implications of inhibiting a key enzyme involved in cellular energy metabolism for the potential treatment of MASLD/MASH. Researchers found that inhibition of ACMSD (aminocarboxymuconate semialdehyde decarboxylase), an enzyme restricted to liver and kidney, enhances de novo NAD + synthesis and significantly reduces DNA damage responses in liver, thereby preventing MASLD/MASH progression. 1 These findings were reproduced in mouse models and in a human liver organoid (HLO) model of steatohepatitis. “Enhancing NAD + levels in hepatocytes represents an attractive yet underexplored area for preventing redox stress. We observed that inhibiting ACMSD in human hepatocytes, both ex vivo and in vivo, increases NAD + levels, enhances mitochondrial respiration, and helps maintain genomic stability,” said Takanori Takebe, MD, PhD, Associate Professor at Cincinnati Children’s Hospital Medical Center and a scientific advisor to OrsoBio. “Here, we have reproduced our earlier findings from a study published in Cell that demonstrated the benefits of alternative approaches to cellular NAD + /redox/mitochondrial modulation in a MASH organoid model from human induced pluripotent stem cells (iPSCs). 2 Importantly, ACMSD inhibition alleviated phenotypes found in MASH organoids even from iPSCs of genetically at-risk individuals.” “Prevention and delayed progression of organ injury in the setting of acute and chronic liver and kidney diseases is a major unmet medical need,” said Johan Auwerx, MD, PhD, Professor at the École Polytechnique Fédérale de Lausanne (EFPL), Switzerland, and a scientific advisor to OrsoBio. “This research suggests that pharmacologic ACMSD inhibition could have a role in advanced disease where there is both metabolic and mitochondrial dysfunction leading to cellular redox mediated exacerbation of the DNA damage response, which sustains progressive inflammation and fibrosis. We look forward to continued collaboration with the OrsoBio team to evaluate the benefits of ACMSD inhibition as monotherapy and in combination with other mechanisms to better understand its therapeutic potential.” A range of proprietary compounds within OrsoBio’s ACMSD inhibitor portfolio are currently being evaluated for their potential to replenish NAD + and improve mitochondrial function in metabolic and inflammatory liver and kidney disorders. The company plans to complete lead optimization and IND-enabling activities in 2025. About ACMSD ACMSD (aminocarboxymuconate semialdehyde decarboxylase) is a key enzyme involved in the de novo synthesis of NAD + from tryptophan. Enhancing NAD + synthesis by silencing or inhibiting ACMSD improves mitochondrial function, increases the lifespan of C. elegans , and protects against liver and kidney injury in mice. OrsoBio’s preclinical portfolio of ACMSD inhibitors is being advanced for the treatment of a range of metabolic and inflammatory liver and kidney disorders. About OrsoBio, Inc. OrsoBio, Inc. is a privately held, clinical-stage biopharmaceutical company dedicated to developing therapies to treat obesity and obesity-associated disorders, including type 2 diabetes, MASH, and severe dyslipidemias. OrsoBio currently has four programs in clinical and preclinical development with first-in-class compounds that address central pathways in energy metabolism. For more information, please visit . 1 Liu YJ, et al. J Hepatol 2024 Aug 22:S0168-8278(24)02484-X. doi: 10.1016/j.jhep.2024.08.009. Available: (24)02484-X/fulltext . 2 Kimura M, et al. Cell 2022;185(22):4216-4232.e16. doi: 10.1016/j.cell.2022.09.031. Available: (22)01250-8 . Contacts Media Contact Gwen Gordon Gwen@gwengordonpr.com

In a year when metabolic diseases have drawn major investor interest, the newest player has big-name backing, a sizable war chest and veteran leadership.Kailera Therapeutics, details of which first emerged in May under the name Hercules CM NewCo, emerged from stealth Tuesday with plans to develop a portfolio of GLP-1 drugs for obesity and other metabolic conditions. The drugs were acquired from Jiangsu Hengrui Pharmaceuticals Co., which had already completed early clinical trials in China for what is now Kailera's lead program, an injectable medicine dubbed KAI-9531.We have an incredible opportunity to develop next-generation treatments for chronic weight management, helping people reclaim their health and live their lives to the fullest, said Ron Renaud, a well-known biotech executive whos become Kaileras CEO.KAI-9531, which targets the GLP-1 and GIP hormone receptors, is being tested in people with obesity and Type 2 diabetes. A Jiangsu Hengrui subsidiary was expected to complete a Phase 2 trial of the drug in Type 2 diabetes earlier this year, and is running two ongoing trials in obesity.Kailera has three other GLP-1 agonists in development, including KAI-7535, which is in clinical testing, as well as two preclinical assets.Though the GLP-1 field is dominated by Novo Nordisk and Eli Lilly, rival pharmas and biotechs alike are working quickly to develop competitors. For instance, Roche and Viking Therapeutics are each developing medicines that act on GLP-1 and GIP, the hormones that are targeted by Lilly's Zepbound.And over the past two years, startups like OrsoBio, SixPeaks Bio and BioAge Labshave raised notable private financings and attracted the attention of pharma partners. The recent IPO performance of BioAge, which pivoted from age-related diseases to obesity drug research, has also suggested investor confidence in the promise of this new wave of metabolic drugs.Renaud, a long-time fixture of the biotech industry, sold his previous three companies: Idenix Pharmaceuticals, bought in a 2014 deal with Merck & Co.; Translate Bio, purchased by Sanofi in 2021; and Cerevel Therapeutics, acquired in 2023 by AbbVie.Kaileras early investors include Atlas Venture, Bain Capital, RTW Investments and Lyra Capital. In May, the venture firms incorporated the company and licensed the drug candidates from Hengrui for $110 million in upfront and near-term payments as well as a roughly 20% equity stake, according to a filing. Bain Capital, where Renaud is a partner, held the largest stake in Kailera as of May at 39%.Joining Renaud on Kaileras leadership team is John Milligan, the former CEO of Gilead Sciences, as chair of the board of directors. '