Opitz GBBB Syndrome, X-Linked

ROCKVILLE, Md.--(BUSINESS WIRE)-- OS Therapies, a research and clinical stage biopharmaceutical company, is extremely excited to announce Full Enrollment for its lead OST-HER2 (OST31-164) program in its ongoing clinical trial AOST-2121 (NCT04974008). The trial is being conducted at 21 Children’s Oncology Group (COG) sites across the country for patients with Osteosarcoma that had metastasized to the lungs requiring surgical removal. Historically, 80-90% of these patients would develop additional metastasis within the period of the trial. The planned number of patients have now received treatment and the few remaining slots will be allocated to patients that have already been identified. Patients continue OST-HER2 treatment for 48 weeks and then are followed for survival 2 additional years. The primary endpoints of the trial are Event Free Survival and Overall Survival. “Several patients have completed the treatment phase of the trial and many more are undergoing treatment with OST-HER2. We hope that this treatment will reduce additional recurrences and extend the lives of all osteosarcoma patients and look forward to the outcome once all patients have completed treatment,” said Dr. Robert Petit, CMO/CSO of OS Therapies. Osteosarcoma is a rare but challenging disease for patients and their physicians, occurring primarily in teens and young adults. “Reaching thirty-nine patients enrolled is a major milestone in this trial, occurring more quickly than projected. A sincere thank you to the patients, families and healthcare providers - the Children’s Oncology Group, and the outstanding leadership of Principle Investigators Damon Reed, MD, and Kelly Bailey, MD,“ said Paul Romness, CEO of OS Therapies. About Osteosarcoma Osteosarcoma is a solid tumor of the bone that predominantly occurs in adolescents and young adults (AYA). Standard treatment includes surgery and chemotherapy. For patients with metastatic osteosarcoma or have recurrence after chemotherapy, the prognosis is poor. About OS Therapies OS Therapies Incorporated is a research and clinical-stage therapeutic company focused on the identification, development, and commercialization of treatments for Osteosarcoma (OS) and other deadly cancers in kids and adults. OS Therapies has two platform technologies: OST-HER2 and OST-TDC being developed for therapies to treat Osteosarcoma (OS) and other solid tumors including ovarian. About OST-HER2 The OST-HER2 Lm vector platform technology has been administered to over 450 cancer patients in ongoing and completed clinical trials. AOST-2121 is a Phase IIB clinical trial intended to prevent or delay metastasis and improve Overall Survival (OS) in Osteosarcoma. OST-HER2 has already received Fast-Track, Orphan, and Rare Disease Designation (RDD). OST hopes to seek a Break-Through Designation (BTD) based on data from this Phase IIb clinical trial. OST31-164 has previously received USDA provisional approval for treatment of Osteosarcoma in canines. In a completed Phase III study in canines (n=180), early data demonstrated a clear separation of treated and untreated canine patients (p=.0007) in Overall Survival (OS) and Disease Progression. For more information, please see the Company's website at

- Decipher Prostate test improves risk-stratification to help reduce under- and over-treatment - SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- Veracyte, Inc. (Nasdaq: VCYT) today announced that data published in the International Journal of Radiation Oncology, Biology, Physics (aka, “The Red Journal”) demonstrate that the company’s Decipher Prostate Genomic Classifier can improve risk stratification among men with clinically high-risk prostate cancer. The data, from a pre-specified analysis of three phase 3 trials, represent the first validation of any gene-expression biomarker on pre-treatment biopsy samples from prospective randomized trials, and suggest the Decipher test provides clinically useful prognostic information to help personalize shared treatment decision-making. Approximately 30% of men with newly diagnosed localized prostate cancer present with high-risk disease, meaning they have an increased risk of disease recurrence, metastasis and death compared to men with lower-risk disease. However, previous studies have shown that more than 70% of men with high-risk prostate cancer who receive standard-of-care definitive radiotherapy and long-term androgen deprivation therapy (ADT) will never develop metastatic disease. “High-risk prostate cancer is heterogenous and standard prognostic tools do a poor job of discriminating which men are likely to develop distant metastases and may benefit from more aggressive therapy, and who will do well when treated with standard radiotherapy and ADT,” said Paul L. Nguyen, M.D., vice-chair for Clinical Research in the Department of Radiation Oncology at The Dana Farber/Brigham Cancer Center and professor at Harvard Medical School, and lead author of the published manuscript. “Because of this, clinicians are forced to use a one-size-fits-all approach that inherently leads to under- and over-treatment of many men. The data published today validate the role of the Decipher Prostate test in more accurately prognosticating patient outcomes, even within a high-risk group, to better personalize care. This utility is being further examined prospectively in ‘PREDICT-RT’, two parallel, randomized phase 3 clinical trials conducted by NRG Oncology and sponsored by the National Cancer Institute.” The Decipher Prostate Genomic Classifier is a 22-gene prognostic biomarker that provides a low, intermediate or high score indicating the aggressiveness of an individual patient’s cancer, to help healthcare professionals more accurately categorize risk and select appropriate treatment. For the current analysis, Dr. Nguyen and colleagues obtained Decipher test scores on 265 biopsy samples from men with prostate cancer that was designated “high-risk” by National Comprehensive Cancer Network (NCCN) classification and who participated in one of three NRG Oncology randomized Phase 3, definitive radiotherapy trials (RTOG 9202, RTOG 9413 and RTOG 9902). With a median follow-up of 11 years, the researchers evaluated the association between Decipher scores and participants’ time to distant metastases (DM), prostate cancer-specific mortality (PCSM) and overall survival (OS). Results demonstrate the Decipher Prostate test was independently prognostic for DM, PCSM and OS, with Decipher high-risk patients experiencing worse outcomes than Decipher low-risk patients on all three endpoints. After adjusting for age, PSA, Gleason score, cT-stage, trial, and randomized treatment arm, the analysis found that the Decipher test scores were independently associated with DM (HR 1.22, 95%CI 1.09-1.36), PCSM (sHR 1.23, 95%CI 1.09-1.39), and OS (HR 1.12, 95%CI 1.05-1.20). Notably, the Decipher test had similar prognostic ability in patients receiving short-term (4 months) or long-term (>24 months) ADT, though the absolute improvement in outcomes varied by Decipher risk. For example, at 10 years, the DM estimates were 31% for short-term vs 20% for long-term ADT in those with higher Decipher Prostate risk scores (an absolute benefit of 11%) but were 7% vs 4% in those with lower Decipher Prostate risk scores (an absolute benefit of 3%). “This comprehensive analysis of three high-quality, randomized Phase 3 studies demonstrates that the Decipher Prostate Genomic Classifier provides information that can meaningfully impact clinical care for men with prostate cancer,” said Elai Davicioni, Ph.D., Veracyte’s medical director for Urology. “Rigorous analyses and data like these are why we believe the Decipher Prostate test was given ‘Level 1’ evidence status in the NCCN’s most recent prostate cancer guidelines - the only gene-expression test to receive such designation. We believe the new data generated by Dr. Nguyen and colleagues could help physicians considering various treatment options for men with high-risk disease and who desire to tailor the duration and intensity of hormonal therapy for their patients on an individual basis.” About Veracyte Veracyte (Nasdaq: VCYT) is a global diagnostics company whose vision is to transform care for patients all over the world. We empower clinicians with the high-value insights they need to guide and assure patients at pivotal moments in the race to diagnose and treat cancer. Our high-performing tests enable clinicians to make more confident diagnostic, prognostic and treatment decisions for some of the most challenging diseases such as thyroid, prostate, breast bladder and lung cancers, as well as interstitial lung diseases. We help patients avoid unnecessary procedures and speed time to diagnosis and appropriate treatment. In addition to making our tests available in the U.S. through our central laboratories, we also aim to deliver our tests to patients worldwide through a distributed model to laboratories that can perform them locally. Veracyte is based in South San Francisco, California. For more information, please visit and follow the company on Twitter (@veracyte). Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements, including, but not limited to our statements related to our plans, objectives, expectations (financial and otherwise) or intentions with respect to our clinical tests in and outside of the United States. Forward-looking statements can be identified by words such as: “appears,” "anticipate," "intend," "plan," "expect," "believe," "should," "may," "will," “positioned,” “designed” and similar references to future periods. Examples of forward-looking statements include, among others, that the Decipher Prostate test can help to more accurately prognosticate patient outcomes, even within a high-risk group, to better personalize care; that the Decipher Prostate Genomic Classifier can help physicians consider treatment options for men with high-risk disease and help tailor duration and intensity of hormonal therapies for patients on an individual basis; and that the Decipher Prostate Genomic Classifier can improve risk stratification among men with clinically high-risk prostate cancer. Additional factors that may impact these forward-looking statements can be found under the caption “Risk Factors” in our Annual Report on Form 10-K filed on February 28, 2022, and our Quarterly Report on Form 10-Q filed for the three months ended September 30, 2022. Copies of these documents, when available, may be found in the Investors section of our website at . These forward-looking statements speak only as of the date hereof and, except as required by law, we specifically disclaim any obligation to update these forward-looking statements or reasons why actual results might differ, whether as a result of new information, future events or otherwise. Veracyte, the Veracyte logo and Decipher are registered trademarks of Veracyte, Inc. and its subsidiaries in the U.S. and selected countries. nCounter is the registered trademark of NanoString Technologies used by Veracyte under license. Veracyte delivers the Decipher Prostate Genomic Classifier from its CLIA laboratories. Those tests are not CE-IVD marked and have not been cleared or approved by the FDA; their performance characteristics were determined by Veracyte and they might be considered for Research Use Only in some markets. Please contact Veracyte for confirmation. This piece is distributed purely for educational purposes and is not intended to promote or encourage any off-label use of Veracyte products.

ROCKVILLE, Md. and OREFIELD, Pa., Sept. 22, 2022 /PRNewswire/ -- OS Therapies, a research and clinical-stage biopharmaceutical company and Team Arnav Foundation, a non-profit 501c3 organization focused on finding and funding new research to diagnose and treat pediatric bone and soft tissue tumors including sarcoma and osteosarcoma - announced today their partnership to support the on-going AOST-2121 COG PhIIb clinical trial in Osteosarcoma. "We are excited to partner with OS Therapies to develop this very promising new treatment for Osteosarcoma," said Dhruv Krishna, CEO and Founder of Team Arnav Foundation, named in memory of Dhruv's twin brother who is an Osteo Angel. The ultimate goal of Team Arnav is to support research to develop better therapies, improve cure rates and minimize treatment toxicities for patients with malignant bone and soft tissue tumors. "We are honored to have Team Arnav join our battle against Osteosarcoma, along with six other foundations and numerous investors. As we have said all along, it will take all of us doing everything we can to beat this deadly bone cancer in kids," said Paul Romness, CEO and Founder of OS Therapies. OS Therapies' OST-HER2 is a Lm vector-based off-the-shelf Immunotherapy intended to prevent metastasis, delay recurrence, and increase overall survival. Twenty-three (23) of 39 to 45 patients have enrolled in AOST-2121 PhIIb Clinical Trial in Recurred, Resected Osteosarcoma testing OST-HER2 (Listeria monocytogenes). Twenty (20) Clinical Trial sites are open across the U.S.: Trial Sites About Osteosarcoma Osteosarcoma is a solid tumor of the bone that predominantly occurs in adolescent and young adults (AYA). Standard treatment includes surgery and chemotherapy. For patients with initially metastatic or recurrence after chemotherapy, there is a significantly poorer prognosis. About OS Therapies OS Therapies Inc. (OST) is a research and clinical-stage therapeutic company focused on the identification, development and commercialization of treatments for Osteosarcoma (OS) and other deadly cancers in kids and adults. OS Therapies has two platform technologies being developed for therapies to treat and cure Osteosarcoma (OS) and other solid tumors including ovarian, esophageal, endometrial and lung cancers. About Team Arnav Foundation Team Arnav Foundation's mission is to find cure for Pediatric Osteosarcoma Cancer which claims majority of its victim's life. Foundation plans to do so through raising awareness and much required donations so it can fund research to eradicate this disease for more information visit About OST-HER2 The OST-HER2 Lm vector platform technology has been administered to over 450 cancer patients in ongoing and completed clinical trials. AOST-2121 is a Phase IIB clinical trial intended to prevent metastasis and improve Overall Survival (OS) in Osteosarcoma. OST-HER2 has received Rare Disease Designation (RDD), Fast-Track and Orphan Designations by the FDA and EMA. OST hopes to seek a Break-Through Designation (BTD) based on data from this Phase IIb clinical trial. For more information, please see the Company's website at