Latest news with #MolecularTherapy
The Hindu
22-05-2025
- Health
- The Hindu
CAR-T therapy can be safely manufactured at hospital, finds ICMR-funded trial led by CMC Vellore
An ICMR-funded trial led by CMC Vellore demonstrated that CAR-T therapy, which uses a patient's own T cells to fight cancer, can be safely manufactured at the hospital and infused to treat patients in India at a low cost. For the first time, these CAR-T cells were produced and infused in a hospital in India. CMC Vellore director and principal author of the study Vikram Mathews explained that Chimeric antigen receptor T cells (CAR-T cells) are normal T-cells that are part of the patient's own immune system. In CART-T cell therapy, these cells are engineered to recognise and target the specific cancer cell, thus using the immune system to fight the disease. CAR-T cell therapy has been proven to be very effective even in patients who have failed all other therapies, he said. This process usually involves inserting into the normal T cells, the required genetic information to produce an antibody receptor that will recognise the antigen/substance on the surface of the cancer cell. "This process is usually done in large centralised commercial corporations, which contributes to logistic challenges, increased costs, and decreased efficacy," Dr. Mathews said. One of many strategies to reduce the cost of this therapy, is to produce the CAR-T cells at the hospital site itself, this strategy is called decentralized or point-of-care manufacturing (PoC). In this study, the authors provided evidence that this is feasible in India. What did the study find? Early data from this study establishes its safety and also shows promising results, said Dr Mathews said. A total of 10 patients, aged 6-59 years, six of them with acute leukemia and four with lymphoma, who had failed all earlier treatments, were treated with CAR-T cells manufactured at CMC Vellore under this PoC strategy. The study found that the therapy brought about 100 per cent remission in acute lymphoblastic leukaemia patients, 50 per cent remission in large B-cell lymphoma patients. Overall, eight of the ten patients remained cancer-free at a median follow-up of 15 months since starting the therapy. The doctors further reported that this was safe, well-tolerated, and had minimal side effects. The study, 'Safety, efficacy and total cost of point-of-care manufactured anti-CD19 CAR-T cell therapy in India: VELCART trial' has been published in the journal Molecular Therapy. The study also stated that when CART-T cells are manufactured in this PoC model, the cost of the therapy is nearly 90 per cent less than the global average. The production time for the CART-T cells was nine days in in-hospital settings. Dr Mathews said that the use of a fresh, unfrozen product also contributed to better outcomes. "This trial redefines how cancer therapy can be delivered - efficiently, affordably, and close to patients. India is leading the way in developing next-generation, in-house biotherapies with global relevance," he said. "This model could be easily replicated in most tertiary healthcare facilities in the country," Dr Mathews said.
Yahoo
15-05-2025
- Business
- Yahoo
Voyager Demonstrates ALPL Receptor-Mediated Blood-Brain Barrier Transport of Novel AAV Capsids in Molecular Therapy Publication
LEXINGTON, Mass., May 15, 2025 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to leveraging genetics to treat neurological diseases, today announced the first peer-reviewed publication of data demonstrating the ability of alkaline phosphatase (ALPL) to transport a novel AAV capsid across the blood-brain barrier (BBB). The article, titled 'Highly conserved brain vascular receptor ALPL mediates transport of engineered AAV vectors across the blood-brain barrier,' was published in Molecular Therapy and can be accessed here. 'Understanding ALPL and its ability to mediate transport across the blood-brain barrier has been foundational to the evolution of our gene therapy programs, two of which are advancing towards IND filings this year with a partner,' said Mathieu Nonnenmacher, Ph.D., Vice President of Gene Therapy at Voyager. 'Building on our first-generation capsids, such as VCAP-102, which is featured in this paper, we have evolved next-generation capsids with even stronger brain transduction and liver de-targeting, as well as stealth capsids with immune-evading capabilities.' The Molecular Therapy paper outlines the generation of novel, cross-species AAV capsid VCAP-102, which demonstrates 20- to 400-fold increased gene transfer across multiple brain regions relative to AAV9 in both rodents and non-human primates (NHP), and the identification of ALPL as the primary receptor used by VCAP-102 to cross the BBB. In addition, the confirmation that the ALPL capsid family binds and demonstrates transcytosis with human ALPL in a cell barrier in vitro model suggests clinical translatability. As previously announced, Voyager presented next-generation and stealth-capsid data at the American Society of Gene & Cell Therapy's (ASGCT) 28th annual meeting. In multiple NHP studies utilizing a variety of payloads, a single intravenous 3e13 vg/kg dose of Voyager's second-generation CNS capsids transduced up to 98% of dopaminergic neurons in substantia nigra, up to 94% of motor neurons in the spinal cord, up to 66% of neurons in the thalamus, up to 43% of neurons in the motor cortex, and 87-99% of astrocytes broadly across brain regions. 'In addition to speeding the evolution of novel capsid families, we are leveraging our work with ALPL and other receptors to deliver diverse classes of non-viral candidates into the CNS,' said Todd Carter, Ph.D., Chief Scientific Officer of Voyager Therapeutics. 'We believe this multi-modality approach, encompassing both viral and non-viral CNS delivery, is critical to addressing unmet needs in neurological disease.' About the TRACER™ Capsid Discovery PlatformVoyager's TRACER™ (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) capsid discovery platform is a broadly applicable, RNA-based screening platform that enables rapid discovery of novel AAV capsids to enable gene therapy. Voyager has leveraged TRACER to create multiple families of novel capsids that, following intravenous delivery in preclinical studies, harness the extensive vasculature of the central nervous system (CNS) to cross the blood-brain barrier and transduce a broad range of CNS regions and cell types. In cross-species preclinical studies (rodents and multiple non-human primate species), intravenous delivery of TRACER-generated capsids resulted in widespread payload expression across the CNS at relatively low doses, enabling selection of multiple development candidates in Voyager's wholly-owned and partnered gene therapy programs for neurologic diseases. About Voyager TherapeuticsVoyager Therapeutics, Inc. (Nasdaq: VYGR) is a biotechnology company dedicated to leveraging the power of human genetics to modify the course of – and ultimately cure – neurological diseases. Our pipeline includes programs for Alzheimer's disease, Friedreich's ataxia, Parkinson's disease, amyotrophic lateral sclerosis (ALS), and multiple other diseases of the central nervous system. Many of our programs are derived from our TRACER™ AAV capsid discovery platform, which we have used to generate novel capsids and identify associated receptors to potentially enable high brain penetration with genetic medicines following intravenous dosing. Some of our programs are wholly owned, and some are advancing with partners including Alexion, AstraZeneca Rare Disease; Novartis Pharma AG; and Neurocrine Biosciences, Inc. For more information, visit Voyager Therapeutics® is a registered trademark, and TRACER™ is a trademark, of Voyager Therapeutics, Inc. Forward-Looking StatementsThis press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as 'will,' 'anticipated,' 'expect,' 'believe,' 'anticipate,' 'potential,' 'may,' or 'continue,' and other similar expressions are intended to identify forward-looking statements. For example, all statements Voyager makes regarding Voyager's ability to advance its AAV-based gene therapy programs and non-viral CNS delivery programs, including the potential for Voyager's novel TRACER capsids to achieve desired results in humans, including neuronal and glial transduction across multiple brain regions, and ALPL-mediated transcytosis similar to the results demonstrated in rodents and NHPs; potential clinical translatability in humans; increased patient eligibility to receive AAV gene therapies; and expectations for advancement of gene therapy product candidates under the collaboration programs, including anticipated submission of IND filings and initiation of clinical trials in two partnered programs are forward looking. All forward-looking statements are based on estimates and assumptions by Voyager's management that, although Voyager believes such forward-looking statements to be reasonable, are inherently uncertain and subject to risks and uncertainties that may cause actual results to differ materially from those that Voyager expected. Such risks and uncertainties include, among others, the continued development of Voyager's technology platforms, including Voyager's TRACER platform and its non-viral discovery platform; Voyager's scientific approach and program development progress, and the restricted supply and increased costs of critical research components; the development by third parties of capsid identification platforms that may be competitive to Voyager's TRACER capsid discovery platform; Voyager's ability to create and protect intellectual property rights associated with the TRACER capsid discovery platform, the capsids identified by the platform, and development candidates for Voyager's pipeline programs; the timing, initiation, conduct and outcomes of Voyager's preclinical and clinical studies; the availability of data from clinical trials; the expectations and decisions of regulatory authorities; the availability or commercial potential of product candidates under collaborations; the success of Voyager's product candidates; the willingness and ability of Voyager's collaboration partners to meet obligations under collaboration agreements with Voyager; the possibility or the timing of Voyager's receipt of program reimbursement, development or commercialization milestones, option exercise, and other payments under Voyager's existing licensing or collaboration agreements; the ability of Voyager to negotiate and complete licensing or collaboration agreements with other parties on terms acceptable to Voyager and the third parties; the success of programs controlled by third-party collaboration partners in which Voyager retains a financial interest; the ability to attract and retain talented directors, employees, and contractors; and the sufficiency of Voyager's cash resources to fund its operations and pursue its corporate objectives. These statements are also subject to a number of material risks and uncertainties that are described in Voyager's most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission. All information in the press release is as of the date of this press release, and any forward-looking statement speaks only as of the date on which it was made. Voyager undertakes no obligation to publicly update or revise this information or any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law. ContactsTrista Morrison, tmorrison@ Investors: Sarah McCabe, smccabe@ Brooke Shenkin, brooke@ in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Daily Mail
08-05-2025
- Health
- Daily Mail
Jab duo could extend life by up to 16 years and de-age the brain - but are you brave enough to take it?
Scientists are a step closer to creating a 'fountain of youth' treatment that could help humans live into their nineties—and beyond. The injections involve the 'anti-ageing molecule' klotho, a type of protein that is naturally produced by the body. Levels of klotho, named after the mythological Greek figure Clotho, who spun the thread of human life, naturally decline as we get age. This process occurs at the same time we start experiencing age-related maladies like weaker bones, loss of muscle mass and declining cognitive abilities. Now, Spanish scientists have developed a klotho-based treatment that both increased levels of the protein in mice and boosted the lifespan of the rodents by a fifth. The experts said this is the equivalent of adding an extra 16 years to an 80 year-old human's lifespan. Not only did these mice live longer they showed improved muscle strength, bone density and brain function. While further research is needed before the treatment can be trialled on humans, the authors of the new study claim it could make a massive contribution to improving the ageing process. In the study, published in the journal Molecular Therapy, experts from the University of Barcelona used a specially modified virus to deliver klotho into the mice's cells. This harmless virus carried the biological blueprints for cells to produce the protein, enabling the mice to boost their klotho levels over a long period. Mice were given the virus via two injections—one into a vein in the body and the other directly into the brain. This enabled it to bypass the natural barrier that normally protects the vital organ and allowed scientists to examine klotho's effect on the brain. In total, three groups of mice were tested. One received the treatment at six months of age, another got the dose at 12 months, and a final group of six month old mice got a placebo to act as a control. Male mice injected at 12 months were found to live the longest (31.5 months on average), a fifth longer than the control group who only lasted 26.3 months. The average mouse lives for roughly 12 to 18 months. The researchers noted that klotho levels were the highest among the animals injected at one year old. Researchers also tested the mice on their physical fitness and found those in the treatment group performed better in experiments measuring coordination and muscle strength. Tissue analysis showed mice on klotho had less internal scarring on their muscles and were generally more muscular. Female mice on the treatment, however, did not see the same lifespan extension due to severe health complications that the scientists said were not related to the treatment. However they did develop stronger bones. Analysis of brain tissue also showed mice of both sexes showed signs they were developing new neurons in the hippocampus, an area of the brain closely linked to learning. This suggests the treatment could combat age-related dementia. Currently the implications for using the treatment in people remain limited. Mice aren't humans, and many drugs and treatments that have shown promise on rodents have failed to replicate this success when trialled on people. Also of note is that many of the tissue samples used in the analysis only came from a limited number of rodents, between three and four, which could limit the results. However, Joan Roig-Soriano, an expert in neuroscience and author of the new study, said techniques that can provide klotho to humans are already available. 'We now have viral vectors that can reach the brain after being administered intravenously, which would make it easier to safely transfer this therapy to humans,' he said. 'Another option would be to administer the protein directly as a drug instead of using viral vectors, but we still need to find an efficient way to deliver it and ensure it reaches the target organs,' he said. Previous research has shown klotho can improve brain function in old primates and 'de-age' the brains of mice by decades. Klotho was originally discovered by Japanese researchers who found that the amount produced by mice could affect how long the rodents lived.
Associated Press
11-04-2025
- Business
- Associated Press
Investing in the $532B Oncology Boom: Key Stocks Shaping the Future of Cancer Treatment
The global oncology drug market, valued at over $200 billion today, is on pace to reach $532 billion by 2031—a growth story driven not just by rising demand but by genuine innovation. After years of incremental progress, new therapies like antibody-drug conjugates (ADCs) and immunotherapies are making strides against some of oncology's toughest challenges: rare pediatric cancers, relapsed tumors, and diseases like osteosarcoma, where survival rates have barely improved in decades. Regulators are helping accelerate this progress with tools like accelerated approvals and breakthrough designations that are shortening development timelines. At the same time, approaches like comparative oncology—using naturally occurring canine cancers as research models—are providing faster, more clinically relevant data than traditional preclinical studies. This convergence of scientific advancement and commercial opportunity is creating a market that's evolving faster than ever before. Within this expanding landscape, several companies are at the forefront of pioneering new treatments. Let's take a closer look at how some of the most innovative players in this space are tackling these pressing challenges. OS Therapies (NYSE-A: OSTX) is focused on transforming the treatment landscape for osteosarcoma, a rare and aggressive bone cancer that primarily affects children and young adults. The company's lead drug, OST-HER2, is a novel off-the-shelf immunotherapy that uses a modified form of Listeria bacteria to stimulate the immune system to target and destroy cancer cells that express the HER2 protein. Recent data has further validated the potential of OST-HER2 in treating osteosarcoma. New unpublished research shows that when combined with palliative radiation, OST-HER2 has had a significant impact on dogs with unresected, primary osteosarcoma. Out of 15 dogs treated, 5 experienced survival times exceeding 500 days, with clinical and radiographic arrest of the primary tumor and delayed pulmonary metastases. These findings could have profound implications for the potential use of OST-HER2 as a frontline therapy in humans, potentially before chemotherapy is even considered. This approach could reduce or even eliminate the need for surgery and chemotherapy, offering a more effective and less invasive treatment alternative for patients. This data complements previous research published in the journal Molecular Therapy, which demonstrated how OST-HER2 induces strong immune responses from the very first dose. These responses were shown to correlate with both the prevention of metastasis and long-term survival in dogs that had undergone surgery to remove their primary osteosarcoma. Additionally, the study showed that dogs who initially had weaker immune responses showed significant improvement after the second and third doses, supporting the use of repeated dosing as a potential strategy for treating the disease. The combination of these results marks a critical milestone in OS Therapies' development of OST-HER2. The company is now preparing to submit this new data to the USDA, along with information on their improved manufacturing process, aiming for conditional approval in the United States by 2025. Following this, OS Therapies plans to conduct a pivotal clinical study with the goal of gaining full approval for the treatment by 2026. The company is also on track to secure FDA Accelerated Approval for OST-HER2 in human osteosarcoma, with plans to submit an application by the end of 2025. If approved, OST-HER2 could be one of the first treatments to offer a meaningful improvement in survival for patients with this rare and difficult-to-treat cancer. Moreover, a successful approval would make OS Therapies eligible for a Priority Review Voucher (PRV), which could be sold for a significant financial gain, providing the company with the resources needed to fund future projects. As OS Therapies continues to advance in both human and veterinary applications, its approach to Comparative Oncology is proving to be a game-changer. With a 96% genetic similarity between human and canine osteosarcoma, research in dogs with osteosarcoma offers valuable insights that could accelerate the development of new therapies for humans. OS Therapies is leveraging this unique advantage to not only improve treatments for dogs but also to push the boundaries of cancer treatment in humans. Financially, OS Therapies remains well-positioned for the future. The company raised $12 million in 2024 through an IPO and private placement, and it expects its cash reserves to last through mid-2026. With clinical costs now tapering off as the company moves forward in its regulatory journey, OS Therapies is in a solid position to continue advancing its pipeline without needing to raise additional capital in the near term. The company's growth isn't limited to just one drug. Beyond OST-HER2, OS Therapies is also working on an innovative antibody-drug conjugate (ADC) platform, which could allow for custom-designed cancer treatments tailored to various cancers. This growing pipeline positions OS Therapies as a company to watch in the biotech space, offering not only a potential breakthrough in osteosarcoma treatment but also future opportunities in oncology. As the company works toward Accelerated Approval for OST-HER2 by the end of 2025, the potential for significant regulatory milestones, a potential PRV sale, and an expanding clinical pipeline make OS Therapies a standout in the emerging biotech field. Investors, clinicians, and patients alike should keep a close eye on this company as it continues to push forward in the fight against osteosarcoma and other forms of cancer. Day One Biopharmaceuticals (Nasdaq: DAWN) is gaining traction in the pediatric oncology world with OJEMDA (tovorafenib), its lead treatment for children with low-grade glioma (pLGG), a rare brain cancer. OJEMDA is a Type II RAF kinase inhibitor that targets BRAF alterations, which are often found in pLGG patients. It received FDA approval under the accelerated approval pathway, and the early numbers suggest strong adoption—more than 1,600 prescriptions were written in the eight months following its April 2024 launch. Full-year net product revenue came in at $57.2 million, with $29 million in the fourth quarter alone. In late 2024, OJEMDA also earned the 'Exclusively Pediatric' designation from CMS, lowering its Medicaid and 340B rebate obligations, which could help margins moving forward. The drug is currently at the center of Day One's pipeline, with the Phase 3 FIREFLY-2 study ongoing. The company expects to complete enrollment by mid-2026. Beyond OJEMDA, Day One is working to expand its reach in pediatric cancer. DAY301, an antibody-drug conjugate (ADC) targeting PTK7, has cleared its first dosing cohort in a Phase 1a/b trial. If development goes well, it could become a valuable second asset alongside OJEMDA. From a financial standpoint, Day One ended 2024 with $531.7 million in cash and equivalents, giving the company plenty of runway. While the full-year net loss totaled $95.5 million—largely due to R&D and launch costs—the company continues to invest in growth. R&D expenses jumped to $227.7 million in 2024, up from $130.5 million in 2023, driven by the advancement of DAY301 and other pipeline efforts. Even with the losses, Day One is in a strong position: OJEMDA is gaining traction, the pipeline is moving, and the balance sheet is healthy. For anyone watching the space, Day One stands out as a biotech laser-focused on filling a serious treatment gap in pediatric cancer. GSK plc (NYSE: GSK) is making real moves in oncology, especially in tough-to-treat cancers like osteosarcoma. In January, the FDA gave Breakthrough Therapy Designation to one of GSK's experimental antibody-drug conjugates (ADCs) that targets B7-H3—a protein linked to tumor growth. The drug showed early promise in a mid-stage trial for patients with relapsed or refractory osteosarcoma who've already gone through two lines of treatment. That's a big deal in a space with no currently approved therapies for patients at that stage. Osteosarcoma mostly affects children and young adults, and once it comes back after initial treatment, the outlook gets bleak. GSK's drug could help fill that gap. The company is now running a global trial aimed at eventually getting the treatment approved more broadly. On the business side, GSK is firing on all cylinders. In February the company launched a $2.5 billion stock buyback after a strong Q4 and raised its long-term revenue forecast. Oncology is now a major focus for GSK's pipeline, along with respiratory diseases, HIV, and other specialty areas. With five product approvals expected this year—including a relaunch of its blood cancer drug Blenrep—the company looks well-positioned to keep growing in high-need treatment areas. ADC Therapeutics (NYSE: ADCT) stands out as a promising player in the antibody drug conjugate (ADC) space, focusing on the treatment of hematologic malignancies and solid tumors. With a proprietary ADC technology platform, the company is positioning itself to make a significant impact in oncology. Investors looking for growth potential in this innovative field should take note of ADC Therapeutics, particularly with its lead product, ZYNLONTA (loncastuximab tesirine). Recent clinical trial results further solidify the company's growth trajectory. In December 2024, ADC Therapeutics published updated data from a Phase 2 clinical trial evaluating ZYNLONTA in combination with rituximab for treating relapsed or refractory follicular lymphoma (FL). The results showed a robust 97.4% overall response rate and 76.9% complete response rate, positioning ZYNLONTA as a strong treatment option for high-risk FL patients. These results were published in The Lancet Haematology and presented at the prestigious American Society of Hematology (ASH) Annual Meeting, raising the company's profile in the oncology field. With progression-free survival remaining strong at 94.6% at 12 months, the long-term potential for ZYNLONTA in treating indolent B-cell lymphomas is clear. Additionally, ADC Therapeutics is making strides with the LOTIS-7 trial, which is evaluating ZYNLONTA in combination with glofitamab for relapsed or refractory diffuse large B-cell lymphoma (DLBCL). The initial data showed impressive results, with a 94% overall response rate and 72% complete response rate, alongside a manageable safety profile. This combination therapy could provide a competitive edge in the highly saturated DLBCL market, demonstrating the potential for ZYNLONTA beyond its initial indication. From a financial perspective, ADC Therapeutics reported stable revenues in Q4 2024, generating $16.4 million in product sales. Despite the flat revenue growth, the company is focused on reducing operating expenses, achieving a 13% year-over-year reduction. With $251 million in cash reserves at the end of 2024, the company is well-positioned to fund operations into the second half of 2026, allowing for continued investment in its clinical pipeline and commercial efforts. Disclaimers: RazorPitch Inc. 'RazorPitch' is not operated by a licensed broker, a dealer, or a registered investment adviser. This content is for informational purposes only and is not intended to be investment advice. The Private Securities Litigation Reform Act of 1995 provides investors a safe harbor in regard to forward-looking statements. Any statements that express or involve discussions with respect to predictions, expectations, beliefs, plans, projections, objectives, goals, assumptions, or future events or performances are not statements of historical fact and may be forward-looking statements. Forward-looking statements are based on expectations, estimates, and projections at the time the statements are made that involve a number of risks and uncertainties that could cause actual results or events to differ materially from those presently anticipated. Forward-looking statements in this action may be identified through the use of words such as projects, foresee, expects, will, anticipates, estimates, believes, understands, or that by statements indicating certain actions & quote; may, could, or might occur. Understand there is no guarantee past performance will be indicative of future results. Investing in micro-cap and growth securities is highly speculative and carries an extremely high degree of risk. It is possible that an investor's investment may be lost or impaired due to the speculative nature of the companies profiled. RazorPitch has been retained and compensated by O S Therapies Inc to assist in the production and distribution of content related to OSTX. RazorPitch is responsible for the production and distribution of this content. It should be expressly understood that under no circumstances does any information published herein represent a recommendation to buy or sell a security. This content is for informational purposes only; you should not construe any such information or other material as legal, tax, investment, financial, or other advice. Nothing contained in this article constitutes a solicitation, recommendation, endorsement, or offer by RazorPitch or any third-party service provider to buy or sell any securities or other financial instruments. All content in this article is information of a general nature and does not address the circumstances of any particular individual or entity. Nothing in this article constitutes professional and/or financial advice, nor does any information in the article constitute a comprehensive or complete statement of the matters discussed or the law relating thereto. RazorPitch is not a fiduciary by virtue of any persons use of or access to this content. City: NAPLES State: Florida Country: United States
Yahoo
10-04-2025
- Business
- Yahoo
OS Therapies Announces Positive Data for OST-HER2 in the Treatment of Unresected Osteosarcoma in Dogs Opening the Potential for Use as Front-Line Therapy in Humans
Yet to be published data from clinical study combining OST-HER2 with palliative radiation in dogs with unresected, primary osteosarcoma shows clinical and radiographic arrest of the primary tumor, delayed pulmonary metastases and prolonged overall survival of greater than 500 days in 5 out of 15 dogs. Data from new publication in the journal "Molecular Therapy" show OST-HER2 induces strong innate and cytotoxic immune responses beginning at the 1st dose of 3 dose regimen, that correlate with prevention of metastasis and long-term survival in dogs with resected primary osteosarcoma. Data from the same study show that short-term survivors have defective immune responses to OST-HER2 that improve with the second and third administration, supporting a proposed regimen of recurrent dosing to potentially alter clinical course and repeated use after treatment failure. NEW YORK, April 10, 2025--(BUSINESS WIRE)--OS Therapies (NYSE-A: OSTX) ("OS Therapies" or "the Company"), a clinical-stage immunotherapy and Antibody Drug Conjugate (ADC) biopharmaceutical company, today announced positive data in the prevention or delay of amputation during the treatment of primary osteosarcoma for OST-HER2 combined palliative radiation in dogs with unresected appendicular osteosarcoma. The treatment led to clinical and radiographic arrest of the primary tumor and prolonged time to metastasis in dogs without surgery or chemotherapy. The data opens the potential for OST-HER2 to be used in frontline therapy in human osteosarcoma prior to initiation of chemotherapy, and potentially reduce the need for chemotherapy altogether, for the purposes of preventing or delaying limb amputation or primary tumor resection surgeries, in addition to the prevention, delay and/or control of lung metastasis. Additionally, the Company announced the publication of positive data in the journal "Molecular Therapy" entitled "Immunological responses and clinical outcomes in dogs with osteosarcoma receiving standard therapy and a Listeria vaccine expressing HER2" demonstrated the correlation of innate and adaptive immune responses to OST-HER2, with prevention of metastasis and long-term survival benefit when used in the adjuvant setting, following standard of care amputation and chemotherapy. Treatment with OST-HER2 was found to be safe and well tolerated in both studies. Taken together, the data support the potential of OST-HER2 to achieve progression free survival (PFS) of primary osteosarcoma, prevent or delay or metastatic disease, prolong progression free survival (PFS) in metastatic disease, and significantly improve long term survival in patients with osteosarcoma. The data on the use of OST-HER2 in unresected primary osteosarcoma in dogs is being prepared for peer-reviewed publication. "We first published strong clinical data on the benefit of OST-HER2 in canine osteosarcoma in 2016, and that led to an initial conditional approval based on a prior manufacturing process that was suboptimal for widespread veterinary use," said Dr. Nicola Mason, the Paul A. James and Charles A. Gilmore Endowed Professor at the University of Pennsylvania, School of Veterinary Medical. "Based upon this initial research, OS Therapies conducted a successful translational Phase 2b clinical trial in the rare pediatric indication of prevention of recurrence of fully resected, osteosarcoma lung metastases. Our newly published canine data and unpublished radiation combination canine data expands the potential use of OST-HER2 into delay/prevention of metastasis and PFS following resected primary osteosarcoma and raises the intriguing possibility of combination radiation therapy and OST-HER2 in the treatment of unresectable osteosarcoma." The Company is preparing to submit this data to USDA, along with new data generated on its newly patented, commercially superior manufacturing process, with the aim of gaining conditional approval for the new manufacturing process for OST-HER2 in the United States to begin sales in 2025. Thereafter, the Company intends to conduct a pivotal clinical study with the aim of gaining full approval in 2026. A link to the publication is available here: "It has been my dream since founding the Company that OST-HER2 could potentially change the standard of care in osteosarcoma, potentially limiting the need for amputation or surgical resection of the primary tumor," said Paul Romness, CEO of OS Therapies. "With today's data, we believe we are taking the first steps towards this given that our Comparative Oncology approach, as a result of the 96% genetic homology between human and canine osteosarcoma, leads us to believe there is significant potential for this canine data to translate into humans in the treatment of frontline and primary metastatic osteosarcoma, similarly to how it has in recurrent, fully-resected, lung metastatic osteosarcoma." Mr. Romness continued, "We are laser focused on getting an Accelerated Approval for OST-HER2 in recurrent, fully resected, lung metastatic human osteosarcoma via Accelerated Approval by year-end 2025 and then using funds obtained from the sale of our pending Priority Review Voucher to expand the potential clinical uses of OST-HER2 throughout the human osteosarcoma treatment paradigm. In parallel, we believe the data from these two studies support the use of OST-HER2 in all phases of canine osteosarcoma treatment and we are hopeful to obtain conditional approval with our newly improved, patented manufacturing for OST-HER2 later this year so that we can launch the product at specialized cancer centers and our four-legged patients can begin getting treatment very soon. About OS Therapies OS Therapies is a clinical stage oncology company focused on the identification, development, and commercialization of treatments for osteosarcoma and other solid tumors. OST-HER2, the Company's lead asset, is an immunotherapy leveraging the immune-stimulatory effects of Listeria bacteria to initiate a strong immune response targeting the HER2 protein. OST-HER2 has received Rare Pediatric Disease Designation (RPDD) from the US Food & Drug Administration and Fast-Track and Orphan Drug designations from the US FDA and European Medicines Agency. The Company has demonstrated positive data in its Phase 2b clinical trial of OST-HER2 in recurrent, fully resected, lung metastatic osteosarcoma demonstrating statistically significant benefit in the 12-month event free survival (EFS) primary endpoint of the study. The Company anticipates submitting a BLA to the US FDA for OST-HER2 in osteosarcoma in 2025 and, if approved, would become eligible to receive a Priority Review Voucher that it could then sell. OST-HER2 has completed a Phase 1 clinical study primarily in breast cancer patients, in addition to showing preclinical efficacy data in various models of breast cancer. OST-HER2 has been conditionally approved by the U.S. Department of Agriculture for the treatment of canines with osteosarcoma. In addition, OS Therapies is advancing its next-generation Antibody Drug Conjugate (ADC) and Drug Conjugates (DC), known as tunable ADC (tADC), which features tunable, tailored antibody-linker-payload candidates. This platform leverages the Company's proprietary silicone Si-Linker and Conditionally Active Payload (CAP) technology, enabling the delivery of multiple payloads per linker. For more information, please visit Forward-Looking Statements Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute forward-looking statements within the meaning of the federal securities laws. These forward-looking statements and terms such as "anticipate," "expect," "intend," "may," "will," "should" or other comparable terms involve risks and uncertainties because they relate to events and depend on circumstances that will occur in the future. Those statements include statements regarding the intent, belief or current expectations of OS Therapies and members of its management, as well as the assumptions on which such statements are based. OS Therapies cautions readers that forward-looking statements are based on management's expectations and assumptions as of the date of this news release and are subject to certain risks and uncertainties that could cause actual results to differ materially, including, but not limited to the approval of OST-HER2 by the US FDA and grant of a priority review voucher and other risks and uncertainties described in "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" in the Company's registration statement on Form S-1 filed with the Securities and Exchange Commission (the "SEC") on November 12, 2024, as amended on November 27, 2024, and other subsequent documents we file with the SEC, including but not limited to our Quarterly Reports on Form 10-Q. Any forward-looking statements contained in this press release speak only as of the date hereof, and, except as required by the federal securities laws, OS Therapies specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise. View source version on Contacts OS Therapies Contact Information: Jack Doll410-297-7793Irpr@ Sign in to access your portfolio
