Bone Diseases, Developmental

NEW YORK--( BUSINESS WIRE )--OS Therapies, Inc. (NYSE-A: OSTX), a clinical-stage biotechnology company advancing immunotherapies and targeted drug conjugates for cancer treatment, today announced positive data from a Phase 2b clinical trial ( NCT04974008 ) of OST-HER2 (OST31-164) - the Company’s HER2-targeted immunotherapy candidate in the rare pediatric-designated indication of prevention of recurrent, fully resected, lung metastatic osteosarcoma. The data demonstrate statistically significant results in the primary endpoint of the study, 12-month event free survival (‘EFS’), where an event is defined as the recurrence of metastatic osteosarcoma, in OST-HER2-treated patients when compared with the leading published historical comparable control group. Further as of the most recent follow up, the data show a strong trend in favor of OST-HER2-treated patients in overall survival (‘OS’, remaining alive) at the 1-year and 2-year interim timepoints of the ongoing 3-year overall survival secondary endpoint. Notably, all patients who achieved the primary 12-month EFS endpoint remain alive. “We are extremely pleased with these results of our Phase 2b clinical trial because they show that OST-HER2-treated patients achieved the primary endpoint of 12-month EFS in a statistically significantly higher ratio than comparable historical controls, in addition to increasing the likelihood of 1-year and 2-year survival as compared with comparable historical controls,” commented Dr. Robert Petit, Chief Medical & Scientific Officer of OS Therapies. “The strong safety profile shown in this study also supports the use of OST-HER2 in this incredibly difficult-to-treat population that has no currently approved therapies.” “The achievement of the primary endpoint in the OST-HER2 phase 2b is a tremendous success that opens the possibility, for the first time, of meaningful therapy for patients suffering from osteosarcoma with lung metastases after resection. This is a leap forward in the development of OST-HER2 and we are pleased that our regulatory strategy is consistent with the FDA’s recent draft guidance update for accelerated approvals. With these positive data in hand, we are preparing to engage with U.S. FDA on an accelerated pathway for approval in this extremely challenging indication,” said Paul Romness, MHP, Chair & CEO of OS Therapies. “We do not expect to have to treat additional patients as part of this process with FDA.” Phase 2b Clinical Trial Data Enrollment Criteria: 12-39 years old Recurred, fully resected lung only metastatic (METS) Osteosarcoma 39 evaluable patients at 21 centers, single treatment arm 52 Weeks on Study: Dosed 16 times every 3 weeks for 48 weeks with 4-week follow-up final visit Primary Endpoint of 12-month EFS: 33% for OST-HER2 vs. 20% for historical control 1 (p = 0.0158) Interim Analysis of Ongoing Secondary Endpoint, OS: 1-year OS: 91% for OST-HER2 vs. 80% for historical control 2 (p = 0.0700) 2-year OS: 61% for OST-HER2 vs. 40% for historical control 2 (p = 0.0576) Post-Hoc Analyses 12-months EFS Subgroup Analysis in the OST-HER2 Treatment Group: Gender (Males vs Females), n = 39: Females (n=19) = 47% Males (n= 20) = 20% (p= 0.0604) Number of lung resections (1 Prior Resection vs. 2+ Prior Resections), n= 39: 1 Prior Resection (n= 28) = 25% 2+ Prior Resections (n= 11) = 55% (p = 0.1366) 12-month EFS Responders using a Non-Concurrent Control Group from the only existing US osteosarcoma database with patients qualified for disease-free status following a fully-resected lung-only metastasis of osteosarcoma origin: OST-HER2, n=39 12-month EFS Responders = 13/39 (33%) NCCG, n=9 12-month EFS Responders = 1/9 (11%) (p = 0.1848) Time to recurrence in patients who did not achieve 12-month EFS: OST-HER2, n=26 = 5.9 months NCCG, n=8 = 4.7 months (p = 0.1454) About OST-HER2 OST-HER2 is an innovative immunotherapy using a HER2 bioengineered form of the bacteria Listeria monocytogenes (Lm) to trigger a strong immune response against cancer cells expressing HER2. This off-the-shelf immunotherapy treatment is designed to prevent metastasis, delay recurrence, kill primary tumors expressing HER2 alone and potentially in combination with existing approved therapies, and increase overall survival. OST-HER2 has received Rare Pediatric Disease Designation (RPDD) from the FDA and Fast Track and Orphan Drug Designations from the FDA and European Medicines Agency (EMA). The US FDA granted OST-HER2 rare pediatric disease designation for osteosarcoma in 2021. The US FDA rare pediatric disease PRV program aims to incentivize drug development for rare pediatric diseases. Under this voucher program, a sponsor who receives an approval for a drug or biological product for a rare pediatric disease qualifies for a voucher that can be redeemed to receive priority review for a different product. The sponsor may also transfer or sell the voucher to another sponsor. OS Therapies intends to sell the PRV it would earn upon receiving an approval of OST-HER2 for recurrent, fully resected, lung metastatic osteosarcoma. The most recent publicly disclosed sale price of a PRV was on November 27 th , 2024 when PTC Therapeutics announced selling its PRV to Kebilidi for $150M. With emerging scarcity in the PRV market, the Company expects the value of PRVs to increase going forward. The maximum sale price of a PRV was in 2015 when AbbVie bought a priority review voucher from United Therapeutics for $350 million. The most recent continuing resolution (CR) negotiations in the US House of Representatives failed to reauthorize the PRV program for pediatric cancers such as osteosarcoma. Despite this, as a result of OS Therapies’ having been granted OST-HER2’s rare pediatric disease designation prior to December 20, 2024 in addition to the Company’s aim to receive an approval for OST-HER2 in the rare pediatric disease osteosarcoma in 2025, prior to the September 30, 2026 deadline, OS Therapies remains eligible to receive the PRV upon approval of OST-HER2 in recurrent, resected metastatic osteosarcoma. The osteosarcoma treatment market was estimated at $1.2 billion in 2022 according to Data Bridge Market Research . Approximately 50% of patients are diagnosed with a lung metastasis at some point following surgical resection and chemotherapy. 3-year survival rates in patients who were not diagnosed with a metastasis are 59% . 3-year survival rates in patients who were diagnosed with pulmonary metastasis were 30% . The Company believes the market opportunity for OST-HER2 in the prevention of lung metastases is over $500 million. About OS Therapies OS Therapies is a clinical stage oncology company focused on the identification, development, and commercialization of treatments for Osteosarcoma (OS) 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. The Company has completed enrollment for a 41-patient Phase 2b clinical trial of OST-HER2 in resected, recurrent osteosarcoma, with results expected in the fourth quarter of 2024. 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) platform, known as tunable ADC (tADC), which features tunable, tailored antibody-linker-payload candidates. This platform leverages the Company's proprietary silicone linker technology, enabling the delivery of multiple payloads per linker. For more information, please visit www.ostherapies.com . 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. 1 Lagmay JP, Krailo MD, Dang H, et al: Outcome of patients with recurrent osteosarcoma enrolled in seven Phase II trials through Children's Cancer Group, Pediatric Oncology Group, and Children's Oncology Group: learning from the past to move forward. J Clin Oncol. 2016;34:3031-8 2 A.H. Aljubran, A. Griffin, M. Pintilie, M. Blackstein; Osteosarcoma in adolescents and adults: survival analysis with and without lung metastases; Annals of Oncology; Volume 20, Issue 6; 2009;1136-41

Every Cure will leverage Google Cloud's infrastructure and AI technologies, including Gemini 2.0, to accelerate discoveries and improve patient outcomes PHILADELPHIA and SUNNYVALE, Calif., Jan. 13, 2025 /PRNewswire/ -- Every Cure, a leading nonprofit dedicated to identifying and validating new uses for existing drugs, today announced an expanded collaboration with Google Cloud to advance AI-driven drug repurposing. By leveraging Google Cloud's AI tools, including Gemini 2.0 large language models to power Every Cure's MATRIX platform, Every Cure aims to accelerate the discovery, validation, and global dissemination of life-saving treatments for diseases lacking effective therapies. More than 300 million people globally suffer from diseases without available treatments, while many face barriers to accessing existing therapies. Drug repurposing utilizes existing medicines, which are often affordable, widely accessible generics with established safety profiles and extensive data, to address this unmet need. By combining Every Cure's mission-driven expertise with Google Cloud's advanced AI capabilities, the collaboration seeks to transform healthcare delivery and patient outcomes worldwide. Every Cure researchers use Google Cloud's infrastructure to stay at the cutting edge of drug repurposing, an approach that can bypass the prolonged timelines and high costs associated with traditional drug development. The Every Cure Leadership Team has already advanced over a dozen repurposed treatments for diseases they weren't initially intended for, including Castleman disease, DADA2 syndrome, POEMS syndrome, and angiosarcoma. By leveraging iterative learning cycles, Every Cure will not only advance computational biology but also deliver immediate benefits to patients with Google Cloud. "We are so excited about the potential for this collaboration with Google Cloud to rapidly scale the impact that Every Cure can make on patients' lives. We created Every Cure to treat patients with existing drugs as quickly as possible and this collaboration supercharges our ability to do this," said Every Cure Co-Founder & President David Fajgenbaum, MD, MBA, MSc. This collaboration focuses on three key use cases: Improving accuracy of AI-driven drug repurposing predictions Every Cure's MATRIX platform will use Google Cloud's AI technology, including Gemini 2.0, to analyze complex biomedical knowledge and integrate diverse data sources, including omics datasets, clinical data, real-world evidence and biomedical knowledge graphs. This synergy will enhance prediction accuracy, identifying overlooked therapeutic opportunities, and accelerating hypothesis generation. Validating predictions through accelerated preclinical testing and optimized clinical trials By leveraging Gemini 2.0-driven trial design and predictive analytics, Every Cure will streamline in vitro and in vivo preclinical testing and clinical trial workflows to confirm efficacy and safety of repurposed drugs. Every Cure will also use Google Cloud's AI-powered tools to select optimal trial designs, reducing resource use and ensuring more efficient evaluation of treatments. Ensuring global adoption of validated treatments to save lives With the help of Google Cloud's scale and infrastructure, Every Cure will ensure that validated repurposed drugs are rapidly adopted into clinical practice, particularly in underserved regions. A few examples of Google Cloud and Workspace tools that are used as part of this collaboration are Google Kubernetes Engine (including GPUs), Ray on VertexAI, BigQuery and Cloud Storage to facilitate the secure dissemination of clinical guidelines. Meanwhile Data Studio provides visual dashboards for global stakeholders. "Every Cure is doing incredible work with the power of cloud and AI technologies by repurposing medicine to save lives," said Scott Penberthy, Senior Director of Applied AI, Office of the CTO, Google Cloud. "We're delighted customers are tapping into the power of GPUs, TPUs, search and AI models that have been purpose built for Google systems, now available on Google Cloud. We are proud to collaborate with them on this journey." This collaboration builds on a successful collaboration between Every Cure and Google Cloud. Google Cloud has supported Every Cure with infrastructure and services to manage biomedical datasets and execute complex computations. With the help of Gemini 2.0's advanced capabilities, Every Cure aims to transform healthcare by accelerating the pace of innovation while ensuring that breakthroughs reach patients worldwide. Every Cure has already identified a number of promising drug repurposing opportunities through its AI-powered pipeline for diseases with major unmet need that are undergoing further investigation, including a neurodevelopmental disorder involving folate receptor auto-antibodies which can be helped with a generic drug that repletes the folate deficiency and can address neurodevelopmental challenges in these patients. About Every Cure Every Cure is a nonprofit biotech organization committed to unlocking the potential of existing medicines to treat as many diseases as possible. By harnessing AI-driven platforms, rigorous preclinical and clinical testing, and global dissemination strategies, Every Cure accelerates the delivery of life-saving treatments to patients worldwide. Every Cure has been profiled on Good Morning America, USA Today, and the Wall Street Journal and selected as both an Audacious Project grantee from TED and a Founder's Prize recipient from Elevate Prize Foundation. Learn more at . Media Contact: [email protected] SOURCE Every Cure WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

- Remarkable early Attruby demand: 430 scripts written by 248 unique HCPs since FDA approval with broad uptake across academic centers and community centers in all patient types - Fully enrolled three major market Phase 3 clinical trials: FORTIFY (BBP-418 for LGMD2I/R9); CALIBRATE (encaleret for ADH1); and PROPEL 3 (infigratinib for Achondroplasia) - Well-financed to launch Attruby and read out major market Phase 3 trials: $406M in cash as of last quarter, received $500M upon acoramidis FDA approval from royalty facility, and anticipate $105M in regulatory milestones in 1H 2025 from acoramidis Europe and Japan approvals PALO ALTO, Calif., Jan. 13, 2025 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a new type of biopharmaceutical company focused on genetic diseases, today provided updates on its commercial progress for Attruby (acoramidis), status of late-stage pipeline programs, and anticipated 2025 milestones. “With the FDA’s approval of Attruby, we marked an important moment for both our organization and the broader ATTR-CM patient community in need of new treatment options. We’re grateful for the enthusiasm surrounding the product and the associated initial commercial momentum, with 430 prescriptions written by 248 unique physicians, and we look forward to continued progress,” said Neil Kumar, Ph.D., Founder and CEO of BridgeBio. “Additionally, we are excited to share that we have completed enrollment of all three of our major market Phase 3 clinical trials. I look forward to continuing to work with this stellar team to serve patients with genetic disease in 2025.” Business UpdateOn November 22, 2024, the U.S. Food and Drug Administration (FDA) approved Attruby (acoramidis), a near-complete TTR stabilizer (≥90%), to reduce cardiovascular death and cardiovascular-related hospitalization in adult patients with ATTR-CM, a progressive fatal disease presenting as an infiltrative, restrictive cardiomyopathy resulting in heart failure. Since the approval, BridgeBio has seen remarkable momentum with 430 patient prescriptions written by 248 physicians. Pipeline Updates BBP-418 – Glycosylation substrate for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9): FORTIFY is a Phase 3 clinical trial of BBP-418 in LGMD2I/R9, a rare genetic disorder caused by variants in the fukutin‑related protein (FKRP) gene that result in progressive muscle degeneration and damage, and eventual loss of functional independence. The trial is fully enrolled with 112 patients.The Company expects Last Patient – Last Visit (LPLV) and topline readout of the interim analysis cohort in second half 2025.If successful, BBP-418 would be the first approved therapy for individuals living with LGMD2I/R9. Encaleret – Calcium-sensing receptor (CaSR) antagonist for autosomal dominant hypocalcemia type 1 (ADH1): CALIBRATE, the Phase 3 clinical trial of encaleret in ADH1, a rare, genetic form of hypoparathyroidism, is fully enrolled with 70 patients. The trial is designed to evaluate the efficacy and safety of encaleret compared to standard of care in adult patients with ADH1.The Company expects Last Patient – Last Visit and topline readout in second half 2025.If successful, encaleret would be the first approved therapy for individuals living with ADH1. Infigratinib – FGFR1-3 inhibitor for achondroplasia and hypochondroplasia: PROPEL 3, the Phase 3 clinical trial of infigratinib in achondroplasia, the most common form of disproportionate short stature, is fully enrolled with 114 participants.The Company expects Last Participant – Last Visit in second half 2025.If successful, infigratinib would be the first approved oral therapy for children living with achondroplasia. 2025 Milestones ProgramStatusAnticipated 2025 MilestoneAcoramidis for ATTR-CMUS FDA approval on November 22, 2024EU and Japan approvals in 1H 2024BBP-418 for LGMD2I/R9FORTIFY, Phase 3 study enrollment completedLast Patient – Last Visit and Topline readout in 2H 2025Encaleret for ADH1CALIBRATE, Phase 3 study enrollment completedLast Patient – Last Visit and Topline readout in 2H 2025Infigratinib for achondroplasiaPROPEL 3, Phase 3 study enrollment completedLast Participant – Last Visit in 2H 2025 About Attruby™ (acoramidis) INDICATIONAttruby is a transthyretin stabilizer indicated for the treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization. IMPORTANT SAFETY INFORMATIONAdverse ReactionsDiarrhea (11.6% vs 7.6%) and upper abdominal pain (5.5% vs 1.4%) were reported in patients treated with Attruby versus placebo, respectively. The majority of these adverse reactions were mild and resolved without drug discontinuation. Discontinuation rates due to adverse events were similar between patients treated with Attruby versus placebo (9.3% and 8.5%, respectively). About BridgeBio Pharma, Inc.BridgeBio Pharma, Inc. (BridgeBio) is a new type of biopharmaceutical company founded to discover, create, test, and deliver transformative medicines to treat patients who suffer from genetic diseases. BridgeBio’s pipeline of development programs ranges from early science to advanced clinical trials. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn, Twitter and Facebook. BridgeBio Pharma, Inc. Forward-Looking StatementsThis press release contains forward-looking statements. Statements in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act), which are usually identified by the use of words such as “anticipates,” “believes,” “continues,” “estimates,” “expects,” “hopes,” “intends,” “may,” “plans,” “projects,” “remains,” “seeks,” “should,” “will,” and variations of such words or similar expressions. BridgeBio intends these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act. These forward-looking statements, including express and implied statements relating to the Company’s expectations regarding the commercial success of Attruby; the Company’s clinical trials, including the timing of the last patient-last visit and topline data readouts for each of FORTIFY, CALIBRATE and PROPEL 3; the potential for encaleret to become a new treatment for ADH1; the potential for BBP-418 to become a new treatment for LGMD2I/R9; the potential for infigratinib to become a new treatment for achondroplasia; timing of approval of Attruby for ATTR-CM in the European Union and Japan; and the Company’s preliminary and unaudited estimate of cash and the Company’s anticipated funding of its current operations and related timelines; and the Company’s expectations regarding reaching regulatory milestones and receipt of milestone payments, among others, reflect the Company’s current views about the Company’s plans, intentions, expectations and strategies, which are based on the information currently available to us and on assumptions the Company has made. Although the Company believes that its plans, intentions, expectations and strategies as reflected in or suggested by those forward-looking statements are reasonable, the Company can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, initial and ongoing data from the Company’s preclinical studies and clinical trials not being indicative of final data, the potential size of the target patient populations the Company’s product candidates are designed to treat not being as large as anticipated, the design and success of ongoing and planned clinical trials, future regulatory filings, approvals and/or sales, despite having ongoing and future interactions with the FDA or other regulatory agencies to discuss potential paths to registration for the Company’s product candidates, the FDA or such other regulatory agencies not agreeing with the Company’s regulatory approval strategies, components of the Company’s filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted, the continuing success of the Company’s collaborations, the Company’s ability to obtain additional funding, including through less dilutive sources of capital than equity financings, potential volatility in the Company’s share price, the impacts of current macroeconomic and geopolitical events, including changing conditions from hostilities in Ukraine and in Israel and the Gaza Strip, increasing rates of inflation and changing interest rates, on business operations and expectations, as well as those risks set forth in the Risk Factors section of the Company’s most recent Quarterly Report on Form 10-Q and Annual Report on Form 10-K and the Company’s other filings with the U.S. Securities and Exchange Commission. Moreover, the Company operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of the Company’s management as of the date of this press release, and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, BridgeBio assumes no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise. BridgeBio Media Contact:Bubba Murarka, EVP Communicationscontact@bridgebio.com(650)-789-8220