Kyphosis

- In Cohort 5 of PROPEL 2, daily oral treatment of infigratinib at 0.25mg/kg resulted in statistically significant and sustained increases in annualized height velocity (AHV), with a mean change from baseline of +2.50cm/year at Month 18 (P=0.001) - Data also presented at European Society of Paediatric Endocrinology on November 18th at 10 am GMT - To date, infigratinib has received Breakthrough Designation from the U.S. Food and Drug Administration for achondroplasia, as well as Orphan Drug Designation, Fast Track Designation, and Rare Pediatric Disease Designation - PROPEL 3, the global Phase 3 registrational study of infigratinib in achondroplasia, continues to enroll on schedule, with enrollment completion anticipated by end of 2024 PALO ALTO, Calif., Nov. 18, 2024 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a new type of biopharmaceutical company focused on genetic diseases, announced that positive 18-month results from PROPEL 2, a Phase 2 trial of the investigational therapy infigratinib in children with achondroplasia, were published as an original research article in the New England Journal of Medicine (NEJM) today. Infigratinib is an investigational oral small molecule designed to inhibit FGFR3 signaling and target achondroplasia at its source. These data, which were also presented today at the 62nd Annual European Society for Paediatric Endocrinology (ESPE) Meeting in Liverpool, demonstrate continued potential best-in-class efficacy and an encouraging safety profile as a first-in-class oral treatment option. “Publication of results from PROPEL 2 in the New England Journal of Medicine and Breakthrough Therapy Designation of infigratinib by the FDA underscore the significance and importance of these data and the potential of this oral therapy to not only increase height, but more importantly, enhance functionality for children with achondroplasia,” said Ravi Savarirayan, M.D., Ph.D., of Murdoch Children’s Research Institute in Melbourne, Australia, the global lead investigator for PROPEL 2 and lead author of the NEJM publication. Key results from the PROPEL 2 dataset were presented today at ESPE in a talk titled “Oral infigratinib for children with achondroplasia: Month 18 results from the PROPEL 2 study demonstrate safety and durability of treatment effect on linear growth with improved body proportionality” by Melita Irving, M.D., a clinical geneticist at Guy’s and St Thomas’ NHS Foundation Trust, London, UK and investigator for the infigratinib clinical program at the Evelina London Children’s Hospital. The key data that were shared included: Statistically significant increase in annualized height velocity (AHV) was observed in children in Cohort 5 who received a daily dose of 0.25 mg/kg/ day of infigratinib), with a mean change from baseline in AHV of +2.50 cm/year (P=0.001) at Month 18Mean change from baseline in height Z-score was +0.54 (P<0.001) relative to an untreated achondroplasia population at Month 18Statistically significant improvement in body proportionality (mean upper to lower body segment ratio) was -0.12 (P=0.001) at Month 18Oral infigratinib was well tolerated at Month 18, with no serious adverse events (SAE) or treatment-emergent adverse events (TEAEs) leading to treatment discontinuation Additionally, there was no accelerated progression of bone age, negative changes in bone mineral density, or other bone-related adverse events observed “We are encouraged to see no safety signals and no adverse changes in bone age or bone mineral density,” said Dr. Irving. “These results point to the potential of infigratinib for children living with skeletal dysplasia, and we look forward to further evaluation of infigratinib in PROPEL 3, the ongoing Phase 3 trial, as well as in the Phase 2 portion of the ACCEL program in children with hypochondroplasia.” Infigratinib has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) based on the shared results from the PROPEL 2 clinical trial, which meet the FDA’s requirement of potentially demonstrating substantial improvement in efficacy over available therapies on clinically significant endpoints. In addition to receipt of Breakthrough Therapy Designation, infigratinib has also received Orphan Drug Designation, Fast Track Designation, and Rare Pediatric Disease Designation for achondroplasia from the FDA. If infigratinib is approved, BridgeBio may qualify for a Priority Review Voucher. Information about PROPEL 3 (NCT06164951), which is currently enrolling children with achondroplasia, can be found here on clinicaltrials.gov. Information about PROPEL (NCT04035811), BridgeBio’s observational lead-in study in achondroplasia for PROPEL 3 and other studies, can be found here on clinicaltrials.gov. BridgeBio is committed to exploring the potential of infigratinib on wider medical and functional impacts of achondroplasia, hypochondroplasia and other skeletal dysplasias, which hold significant unmet needs for families. About AchondroplasiaAchondroplasia is the most common cause of disproportionate short stature, affecting approximately 55,000 people in the United States (U.S.) and European Union (EU), including up to 10,000 children and adolescents with open growth plates. Achondroplasia impacts overall health and quality of life, leading to medical complications such as obstructive sleep apnea, middle ear dysfunction, kyphosis, and spinal stenosis. The condition is uniformly caused by an activating variant in FGFR3. About BridgeBio Pharma, Inc.BridgeBio Pharma (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. We intend 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 statements relating to the clinical, therapeutic and market potential of our programs and product candidates, the progress of our ongoing and planned clinical trials of infigratinib in achondroplasia, hypochondroplasia and other skeletal dysplasias, infigratinib’s potential best-in-class efficacy, infigratinib’s potential for obtaining a Priority Review Voucher, our planned interactions with regulatory authorities and the statements regarding the potential clinical benefits of infigratinib for patients in the quotes of Dr. Savarirayan and Dr. Irving, reflect our current views about our plans, intentions, expectations and strategies, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations and strategies as reflected in or suggested by those forward-looking statements are reasonable, we 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 our clinical trials not being indicative of final data, the design and success of ongoing and planned clinical trials, difficulties with enrollment in our clinical trials, adverse events that may be encountered in our clinical trials, the FDA or other regulatory agencies not agreeing with our regulatory approval strategies, components of our filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted, the ability of infigratinib to retain Breakthrough Therapy Designation, Fast Track Designation, Rare Pediatric Disease Designation, and Orphan Drug Designation from the U.S. Food and Drug Administration and potential adverse impacts due to global health emergencies, including delays in regulatory review, manufacturing and supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy, 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 rising interest rates, on our business operations and expectations, as well as those risks set forth in the Risk Factors section of our most recent Annual Report on Form 10-K and our other filings with the U.S. Securities and Exchange Commission. Moreover, we operate 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 our 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, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise. BridgeBio Contact:Vikram Balicontact@bridgebio.com(650)-789-8220

IRVINE, Calif., Aug. 6, 2024 /PRNewswire/ -- Aspen Medical Products (Aspen), the industry leader in spine solutions for pain and mobility management, has launched the Align Orthosis. In collaboration with spine surgeon Han Jo Kim, MD of the Hospital for Special Surgery, the all-new Align Orthosis is the first prefabricated TLSO designed to help mitigate post-surgical incidence of proximal junctional kyphosis (PJK). Continue Reading Align Orthosis "Isolation of a single strategy is yet to be successful in PJK prevention. When used post-operatively, the Align Orthosis is a non-invasive addition to mitigation strategies, so we truly have a multifactorial approach to prevention," said Dr. Kim. According to a published study in Spine1, spine deformity surgeries are increasing at a rapid rate, up 141 percent between 2004 and 2014. Additional research2 suggests that proximal junctional kyphosis (PJK) occurs in approximately up to 30 percent of adult deformity surgery patients, with most cases occurring within two years of surgery. Symptoms include pain, neurologic deficit and ambulatory difficulties – which can often lead to revision surgeries that are costly and painful. The lack of a singular solution for surgeons to mitigate the incidence of PJK underscores the need for a multifactorial strategy. Dr. Kim recognized this issue and sought to find a solution. "Some of my patients had altered body mechanics, so I thought that using a brace for flexion control would be helpful in teaching them a better way to move their spine after an operation and ultimately reduce risk of developing PJK," said Dr. Kim. This led to a retrospective analysis, comparing outcomes of a cohort of patients that were either braced or non-braced post-surgery. This research showed that post-surgical bracing with a hyperextension brace made a patient three times less likely to develop PJK.3 Despite the positive outcomes of the study, post-operative compliance varied due to flexion control problems and discomfort, such as the brace riding up when patients sat down and irritation at the surgical site. "We thought we could make improvements to the design to make it more patient-friendly, therefore increasing compliance and potentially mitigating the risk for developing PJK," said Dr. Kim. The Align Orthosis is an evolution of current hyperextension braces with better flexion control, comfort and patient compliance, helping to reduce readmission and revision surgery rates. This is achieved by a three-point pressure system that contacts key points at the chest, lower abdomen and mid-back. The design of the brace also helps to restore symmetrical spinal loads and guide patients through correct spinal biomechanics to help patients safely move after surgery and improve balance. "The Align Orthosis embodies our commitment to advancing treatment and improving outcomes for spinal deformity patients, made possible through collaboration with spine surgeons," said Jim Cloar, Aspen's Chief Executive Officer. "As a leader in spinal care, we are dedicated to supporting surgeons with new strategies to help mitigate the increasing post-surgical PJK incidence rate." More information on the Align Orthosis is available at alignpjk.aspenmp.com. About Aspen Medical Products Founded in 1994, Aspen Medical Products, LLC is the industry leader in the development and manufacturing of spinal orthopedics and pain therapy solutions. Designed to restore function and alleviate pain, Aspen's products address a variety of patient needs across the continuum of care to help them resume activities of daily living. Headquartered in Irvine, California, Aspen has local sales representation throughout the U.S. and internationally. Beschloss, A., Dicindio, C., Lombardi, J., Varthi, A., Ozturk, A., Lehman, R., Lenke, L., & Saifi, C. (2021). Marked increase in spinal deformity surgery throughout the United States. Spine. Advance online publication. Kim, H. J., Lenke, L. G., Shaffrey, C. I., Van Alstyne, E. M., & Skelly, A. C. (2012). Proximal junctional kyphosis as a distinct form of adjacent segment pathology after spinal deformity surgery: A systematic review. Spine, 37(22 Suppl), S144–S164. Shahi, P., Merrill, R. K., Pajak, A., Samuel, J. T., Akosman, I., Clohisy, J. C., Du, J., Zhang, B., Elysee, J., Kim, D. N., Jordan, Y., Knopp, R. L., Lovecchio, F. C., & Kim, H. J. (2024). Post-operative hyperextension bracing has the potential to reduce proximal junctional kyphosis: A propensity matched analysis of braced versus non-braced cohorts. Global Spine Journal. SOURCE Aspen Medical Products

KING OF PRUSSIA, Pa.--( BUSINESS WIRE )-- Camber Spine , a leading innovator in spine and medical technologies, announced today that it has received U.S. Food and Drug Administration (FDA) 510(k) clearance for its SPIRA-A Integrated technology. Part of the SPIRA product platform, the SPIRA-A Integrated Fixation System is an anterior lumbar interbody fusion device that has an open matrix design to permit packing with autogenous and/or allogenous graft material to facilitate fusion, as well as additional fixation options to secure the implant in the disc space. “The FDA’s 510(k) clearance of our SPIRA-A Integrated technology ushers in the next evolution in our innovative SPIRA platform and marks another major step forward in our company’s development,” said Camber Spine CEO Brooks McAdam. “It also helps bring more innovation-based solutions and options to the surgical community and a solution for surgeons engaging or wanting to engage in the ALIF market. We believe SPIRA Technology, which encompasses the structure, surface, and science behind our 3D-printing process, is the archetypal design for modern spine implants and exemplifies the true benefits of additive manufacturing.” SPIRA-A Integrated offers a complete solution to the ALIF procedure, with integrated fixation deployed in a traditional ALIF cage and approach, as well as a windswept cage geometry for accessing L5-S1 with difficult vascular anatomy, and each implant offers up to 40 points of endplate contact. The superior and inferior surfaces of the SPIRA-A Integrated device have a rough surface to help prevent movement of the device while fusion takes place, and structural arches to help distribute load across the joint space. The device contains three holes to insert bone screws or anchors for integrated fixation, as well as blocking screws to prevent fixation back-out. SPIRA-A’s screws and anchors have been designed to complement the performance of the cage, increase fixation in the cortical endplate, and provide first-of-their-kind 3D-printed anchors with a SPIRA Surface, designed to increase potential for osseointegration and resist pull-out. The robust inserter attachment, multiple technique possibilities, and a wide variety of screw prep options are designed to facilitate clear visualization of, and easy access to, the surgical site once the approach is complete. The FDA’s action means that SPIRA-A Integrated is now indicated for use at one or more levels, from L1-S1 as an adjunct to fusion in skeletally mature patients with degenerative disc disease (DDD), disc herniation (with myelopathy and/or radiculopathy) spondylolisthesis, deformity (degenerative scoliosis or kyphosis), spinal stenosis and failed previous fusion (pseudarthrosis). Camber’s SPIRA implants are 3D printed. This specialized manufacturing technology allows Camber to create unique patented structures featuring open arched matrices and proprietary surfaces designed to enhance fusion and promote bone growth. All of Camber Spine’s products are developed and manufactured in the United States. Innovative spine and medical technology company Camber Spine Technologies is dedicated to creating surgeon-designed solutions in MIS and minimally disruptive access for the treatment of complex spinal pathology. Incorporating state-of-the-art manufacturing, 3-D printing, and an acute sensitivity to patient anatomy, Camber Spine is making quantum leaps in the spinal fusion market. Learn more at CamberMedtech.com .