Brain Damage, Chronic

A C-section was not performed for over two hours after being ordered, leading to the child's asphyxiation and severe, lifelong disability, the plaintiffs said. The nonprofit health system intends to appeal the decision. A Michigan jury has found Detroit-based Henry Ford Health and some of its practitioners to be negligent in a case involving a delayed caesarean section and resulting severe disability. Jurors handed down the verdict March 28 and awarded $120 million in damages to a mother, Kristen Drake, and her 13-year-old son, K’Jon Drake, according to court documents. The minor has severe disabilities, plaintiffs said, and jurors agreed, which are the result of a Henry Ford obstetrician and hospital nurses’ failure to perform a C-section procedure for more than two hours. In a statement, the nonprofit health system told Fierce Healthcare it intends to appeal the decision. The case was filed in Michigan’s Wayne County Circuit Court in 2020. Plaintiffs were joined by the Michigan Department of Health and Human Services, which sought to recover money paid to support K’Jon Drake’s medical care. Kristen Drake had presented to Henry Ford Hospital in 2010, where fetal monitoring “began to show decelerations due to cord compression and decreased variability” after arrival, according to a court filing from the plaintiffs. The attending obstetrician called for a C-section that was “unnecessarily delayed” for two hours and 19 minutes, they wrote. “Obviously time is of the essence in delivering a baby who is showing non reassuring fetal heart tones. There is simply nothing to be gained by waiting,” plaintiffs wrote in a filing. “On the other hand delay can result in a baby becoming progressively more hypoxic to the point where he develops metabolic acidosis and can suffer hypoxic ischemic brain damage and other sequelae of intrapartum asphyxia. Tragically … that is exactly what happened in this case.” K’Jon Drake, who is now 13, lives with cerebral palsy, blindness and permanent brain damage due to that asphyxiation, plaintiffs said. He requires lifelong care, which until now was largely delivered by his mother and grandmother. The four-week medical malpractice trial culminated in the jury awarding about $120 million in damages for the family, part of which will be paid to the state’s health department. It represents a combination of past and future economic damages for needed care, future loss of wage earning capacity, and past and future noneconomic damages broadly related to pain and suffering. Henry Ford Health is an integrated care provider with more than 250 locations and five acute hospitals in Michigan. In an emailed statement, the nonprofit said it’s been “deeply saddened for the Drake family since the birth of their son more than a decade ago. “At the same time, we do not believe the verdict is consistent with the facts of this case and plan to vigorously appeal the jury’s verdict. Given the ongoing nature of the case, we are not able to provide any additional comment at this time,” the system wrote.

WASHINGTON, March 28, 2024 /PRNewswire/ -- Building on the legacy of 15 years as a growing and impactful foundation serving the rare disease community, the EveryLife Foundation for Rare Diseases announces the appointment of Michael Pearlmutter into the new role of Chief Executive Officer of the foundation. Michael is a distinguished executive with more than 13 years of leadership experience in mission-driven organizations, most recently as the Executive Director of the Cerebral Palsy Alliance Research Foundation (CPARF). While there, he was instrumental in significantly expanding research initiatives, boosting fundraising efforts, and initiating both the Remarkable US disability technology accelerator program and the strategic partnership with the Cerebral Palsy Research Network (CPRN). During his tenure, CPARF saw a remarkable 475% increase in grant applications and 140% rise in fundraising, including securing three of the organization's largest donations. Michael's appointment is an instrumental part of EveryLife Foundation's vision to expand the foundation's donor community and activities as it embarks on its next era. The Foundation seeks to double its budget and reach over the next 5-7 years, to support a bold new vision that not only builds on its highly successful Community Congress and Rare Disease Week but expands its ability to change rare disease outcomes. New initiatives will address science policies, including novel therapy platforms, such as gene and cell therapies; regulatory science strategies to address new clinical trial approaches; and the use of AI and big data to design and develop methods to shorten the diagnostic odyssey and reduce disease impact on patients and caregivers living with rare disease. Building on our groundbreaking studies in both areas, these programs will allow for the expansion of the EveryLife community to further illustrate the case for rare disease as a public health imperative. A graduate of the United States Naval Academy, Michael brings a passion and dedication to service. Michael also has firsthand experience with a rare disease, furthering his dedication and personal connection to the mission of the EveryLife Foundation. His own experiences bring a deep understanding and empathy for challenges faced by rare disease patients, including the often-prolonged diagnostic odyssey that many endure. Vicki Seyfert-Margolis, Chair of the Board of Directors at the EveryLife Foundation for Rare Diseases, expressed excitement about Pearlmutter's appointment, stating, "Michael's dynamic leadership and personal connection to the rare disease community make him the ideal choice to lead our Foundation. His visionary approach and unwavering commitment are exactly what we need to grow the foundation and drive awareness that rare disease is a public health crisis that impacts everyone, and that innovations in rare disease almost lead the way in development of new treatments for diseases affecting many." Upon his appointment, Pearlmutter shared his enthusiasm, "I am excited to be stepping into the role of CEO at the EveryLife Foundation for Rare Diseases. As someone who has personally navigated the challenges of a rare blood disorder, I am deeply motivated to harness our collective power to make a significant impact. Every day, I am grateful that my diagnosis comes with a treatment and a positive prognosis, but I know that the journey of every individual who has a rare disease is unique. Together, we'll foster innovation, strengthen partnerships, and accelerate progress to transform the lives of the rare disease community. I am filled with energy, optimism, and determination as we embark on this journey together." Michael's extensive background in nonprofit management and fundraising, coupled with his innovative leadership, positions him perfectly to lead the Foundation in broadening its vision to advance treatments and cures for rare diseases. About: EveryLife Foundation for Rare Diseases is a nonprofit, nonpartisan organization dedicated to advancing the development of treatment and diagnostic opportunities for rare disease patients through science-driven public policy. The Foundation works to improve the lives of the millions of Americans suffering from rare diseases by advocating for policies that foster innovation and remove barriers to life-saving treatments. Founded in 2009 and headquartered in Washington, DC, the EveryLife Foundation for Rare Diseases has programs throughout the United States. To learn more, visit EveryLifeFoundation.org. For additional information visit everylifefoundation.org/news/. Follow us on Facebook, Twitter, Instagram, and LinkedIn. SOURCE EveryLife Foundation for Rare Diseases

Enrollment in CARPO, Phase 2b trial of Auxora™ in acute pancreatitis (AP), on track with topline data expected in 2Q 2024 Auxora granted Investigational New Drug clearance by the FDA for Phase 2 trial in severe acute kidney injury (AKI); KOURAGE initiating in 2Q 2024 CRSPA Phase 1/2 trial of Auxora in asparaginase-induced pancreatic toxicity (AIPT) expanded and progressing to Phase 2 following initial patient cohort results presented at ASH 2023 Following a private placement financing in January, Company's cash position is expected to fund operations into 2H 2025 LA JOLLA, Calif., March 28, 2024 /PRNewswire/ -- CalciMedica Inc. ("CalciMedica") (Nasdaq: CALC), a clinical-stage biopharmaceutical company focused on developing novel calcium release-activated calcium (CRAC) channel inhibition therapies for acute and chronic inflammatory and immunologic illnesses, today reported financial results for the year ended December 31, 2023. "Over the course of 2023 and into this year, we have continued to build upon CalciMedica's clinical and financial strength, which has culminated in several recent achievements and has positioned us to execute on key milestones in 2024 including presenting topline data from our ongoing CARPO trial in acute pancreatitis in the second quarter," said Rachel Leheny, Ph.D., Chief Executive Officer of CalciMedica. "Importantly, the support from leading life science investors in our recent private placement financing allows us to initiate KOURAGE, our Phase 2 trial of Auxora in severe AKI patients, and to continue to progress other key programs in our pipeline." Recent Clinical and Preclinical Updates and Anticipated Milestones: Phase 2b CARPO enrollment on track and topline data expected in 2Q: Enrollment in CARPO, CalciMedica's randomized, double-blind, placebo-controlled Phase 2b trial of Auxora™ in AP patients, is expected to be complete and topline data from the trial to be announced in the second quarter of 2024. IND application for Auxora in AKI approved and Phase 2 KOURAGE trial initiation underway: In February 2024, CalciMedica received clearance of its Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) for Auxora to be evaluated in a Phase 2 trial in AKI with associated acute hypoxemic respiratory failure (AHRF) which has been named KOURAGE. CalciMedica expects to enroll the first patient in KOURAGE in the second quarter of 2024, with data expected in 2025. Data from initial cohort of Phase 1/2 CRSPA study with St. Jude presented at ASH: In December 2023, CalciMedica's collaborators at St. Jude Children's Research Hospital presented data from the initial cohort of the Phase 1/2 CRSPA study of Auxora in AIPT at the 65th Annual American Society of Hematology Meeting & Exposition (ASH) in San Diego, CA. The data from the initial cohort compared to a historical matched control group showed fewer hospital and ICU days for treated patients. No patients in the CRSPA study required total parenteral nutrition, compared to 68.8% in the historical matched control group, who required 27 days of nutritional support on average. 27% of patients in the matched control group had greater than 30% pancreatic necrosis at 30 days compared to none of the Auxora-treated patients. CRSPA study expanded and continuing to enroll in Phase 2 portion of trial: The CRSPA study has been expanded to additional sites, the dose used in the initial cohort has been established as the recommended Phase 2 dose and a target total trial enrollment has been set at 24. Data is expected in 2025. Preclinical data presented at AKI & CRRT: In March 2024, CalciMedica presented data from preclinical studies of Auxora in AKI at the 29th International Acute Kidney Injury and Continuous Renal Replacement Therapy Conference (AKI & CRRT) in San Diego, CA. The results of the studies indicate that Auxora has the ability to hasten the recovery of kidney function and improve survival in rat models of AKI through inhibiting the Orai1 channels on Th17 and endothelial cells. 2023 Financial Results and Corporate Updates: As of December 31, 2023, CalciMedica had $11.2 million in cash, cash equivalents and short-term investments. In January 2024, CalciMedica completed a private placement of securities to new and existing investors for up to approximately $54 million in gross proceeds that includes initial upfront funding of $20.4 million and up to an additional approximately $33.1 million upon exercise of accompanying warrants. CalciMedica intends to use the upfront net proceeds from the private placement to fund the Company's ongoing Phase 2 clinical trials for Auxora in AP and AKI. As of the end of the first quarter of 2024, the Company expects its cash, cash equivalents and short-term investments to be approximately $25.5 million, which, based on its current operating plan, it expects to be sufficient to fund its operations into the second half of 2025. Total operating expenses were $38.1 million for the year ended December 31, 2023, which included $16.2 million of one-time charges of which $10.5 million was non-cash, related to accelerated vesting of stock options and severance for employees of Graybug Vision, Inc. at the time of the reverse merger with Graybug. Net loss was $34.4 million, or $7.66 per share (basic and diluted) for the year ended December 31, 2023. About Auxora™ CalciMedica's lead clinical compound, Auxora™, is a potent and selective small molecule inhibitor of Orai1-containing CRAC channels that is being developed for use in patients with acute inflammatory and immunologic illnesses. CRAC channels are found on many cell types, including immune system cells, endothelium cells and pancreatic acinar cells, where aberrant activation of these channels may play a key role in the pathobiology of acute and chronic inflammatory syndromes. Auxora is currently being evaluated in: (i) a Phase 2b trial for acute pancreatitis (AP) with accompanying systemic inflammatory response syndrome (SIRS), called CARPO, (ii) a Phase 2 trial in acute kidney injury (AKI) with associated acute hypoxemic respiratory failure (AHRF), called KOURAGE, expected to initiate in the second quarter of 2024 and (iii) an investigator-sponsored Phase 1/2 trial, called CRSPA, being conducted in pediatric patients with asparaginase-induced pancreatic toxicity (AIPT) as a side effect of pediatric acute lymphoblastic leukemia treatment with asparaginase. There are currently no approved therapies to treat either AP, AKI or AIPT. In previous trials, patients responded well to Auxora regardless of severity or cause of disease. CalciMedica is also exploring the potential of Auxora treatment for other acute indications including acute respiratory distress syndrome. About CARPO and AP CARPO is an international, randomized, double-blind, placebo-controlled, dose-ranging trial intended to establish Auxora's dose-response and efficacy in AP with accompanying SIRS. It is expected to enroll 216 patients. AP can be a life-threatening condition where the pancreas becomes inflamed, sometimes leading to pancreatic cell death or necrosis, systemic inflammation, organ failure and death. There are an estimated 275,000 hospitalizations for AP annually in the United States, of which approximately 40% present with SIRS, which can compromise the function of other tissues or organs, especially the lungs. Organ failure is responsible for much of the mortality seen in AP. There is currently no approved therapy for AP. Details of the CARPO trial are available on clinicaltrials.gov (NCT04681066). About KOURAGE and AKI KOURAGE is a randomized, double-blind, placebo-controlled study that will evaluate 150 patients with Stage 2 and 3 AKI who have AHRF and are receiving oxygen by non-invasive mechanical ventilation, high flow nasal cannula or intermittent mandatory ventilation (IMV). AKI denotes a sudden reduction in kidney function, or the organ's ability to clean and filter the blood. AKI can result as a complication of other serious illnesses such as sepsis, respiratory infections and failure, acute pancreatitis, trauma, surgery and burns. There are approximately 3.7 million hospitalized with AKI in the United States each year with approximately 1.1 million of these patients advancing to Stage 2 and Stage 3 AKI, over half of whom have associated AHRF. The risk of serious morbidities and mortality is significant for advanced Stage 2 and Stage 3 AKI patients. There are currently no approved therapies for AKI. About CRSPA and AIPT CRSPA is an investigator-sponsored Phase 1/2 trial being conducted in pediatric acute lymphoblastic leukemia (ALL) patients with AIPT, which is acute pancreatitis toxicity caused by the administration of asparaginase (such as Oncaspar and Rylaze). CalciMedica believes that the CRSPA trial has defined an optimal pediatric dose for Auxora in this setting and the trial is currently being expanded to additional sites and is expected to enroll 24 patients. Approximately 3,000 pediatric patients are treated for ALL in the United States each year and treatment with asparaginase triggers the development of AIPT in 7-10% of these patients, with approximately half developing pancreatic necrosis and/or pseudocysts. There are currently no approved therapies for AIPT. Details of the CRSPA trial are available on clinicaltrials.gov (NCT04195347). About CalciMedica CalciMedica is a clinical-stage biopharmaceutical company focused on developing novel CRAC channel inhibition therapies for inflammatory and immunologic diseases. CalciMedica's proprietary technology targets the inhibition of CRAC channels to modulate the immune response and protect against tissue cell injury, with the potential to provide therapeutic benefits in life-threatening inflammatory and immunologic diseases for which there are currently no approved therapies. CalciMedica's lead product candidate Auxora™, a proprietary, intravenous-formulated CRAC channel inhibitor, has demonstrated positive and consistent clinical results in multiple completed efficacy clinical trials. CalciMedica is currently conducting a Phase 2b trial (called CARPO – NCT04681066) for AP with SIRS, with topline data expected in the second quarter of 2024, as well as supporting the ongoing Phase 1/2 AIPT study (called CRSPA – NCT04195347), with data expected in 2025. CalciMedica plans to initiate its Phase 2 study (called KOURAGE) in AKI with associated AHRF in the second quarter of 2024 with data expected in 2025. CalciMedica was founded by scientists from Torrey Pines Therapeutics and the Harvard CBR Institute for Biomedical Research, and is headquartered in La Jolla, CA. For more information, please visit . Forward-Looking Statements This communication contains forward-looking statements which include, but are not limited to, statements regarding CalciMedica's expected cash, cash equivalents and short-term investments as of the end of the first quarter of 2024; CalciMedica's expected use of proceeds from the private placement; CalciMedica's expected cash runway; CalciMedica's business strategy; CalciMedica's planned and ongoing clinical trials and the timing, design, expected patient enrollment thereof and the expected timing for the release of data from those trials, including its planned Phase 2 KOURAGE trial of Auxora in AKI with associated AHRF, its ongoing Phase 2b CARPO trial of Auxora for AP with accompanying SIRS and its ongoing Phase 1/2 CRSPA trial of Auxora in pediatric patients with AIPT; the potential benefits of Auxora for the treatment of AKI, AP and AIPT; the estimated patient populations in the United States for AKI, AIPT and AP; the potential of Auxora for the treatment of other acute indications including acute respiratory distress syndrome; and the potential of CalciMedica's proprietary technology to provide therapeutic benefits in life-threatening inflammatory and immunologic diseases. These forward-looking statements are subject to the safe harbor provisions under the Private Securities Litigation Reform Act of 1995. CalciMedica's expectations and beliefs regarding these matters may not materialize. Actual outcomes and results may differ materially from those contemplated by these forward-looking statements as a result of uncertainties, risks, and changes in circumstances, including but not limited to risks and uncertainties related to: the impact of fluctuations in global financial markets on CalciMedica's business and the actions it may take in response thereto; CalciMedica's ability to execute its plans and strategies; the ability to obtain and maintain regulatory approval for Auxora; results from clinical trials or preclinical studies may not be indicative of results that may be observed in the future; potential safety and other complications from Auxora; the scope, progress and expansion of developing and commercializing Auxora; the size and growth of the market therefor and the rate and degree of market acceptance thereof; economic, business, competitive, and/or regulatory factors affecting the business of CalciMedica generally; CalciMedica's ability to protect its intellectual property position; and the impact of government laws and regulations. Additional risks and uncertainties that could cause actual outcomes and results to differ materially from those contemplated by the forward-looking statements are included under the caption "Risk Factors" in CalciMedica's Annual Report on Form 10-K for the year ended December 31, 2023 being filed with the Securities and Exchange Commission (SEC) later today and elsewhere in CalciMedica's subsequent reports on Form 10-K, Form 10-Q or Form 8-K filed with the SEC from time to time and available at . These documents can be accessed on CalciMedica's web page at ir.calcimedica.com/financials-filings/sec-filings. The forward-looking statements contained herein are made as of the date hereof, and CalciMedica undertakes no obligation to update them after this date, except as required by law. CalciMedica Contact: Investors and Media Argot Partners Sarah Sutton/Kevin Murphy calcimedica@argotpartners.com (212) 600-1902 Company Codes: NASDAQ-NMS:CALC