Disorders of Sex Development

WALTHAM, Mass.--( BUSINESS WIRE )-- Revvity, Inc. (NYSE: RVTY), today announced that it received 510(k) clearance from the U.S. Food and Drug Administration (FDA) for EUROIMMUN’s automated chemiluminescence-based immunoassay (ChLIA) test for free testosterone. This innovative test is the first of its kind to receive FDA clearance for direct quantitative measurement of free testosterone levels, marking a significant advancement in diagnostic capabilities for androgen disorders. Key features of the new test include: The only FDA-cleared ChLIA assay for direct quantitative measurement of free testosterone in human serum or plasma. Rapid results on EUROIMMUN’s ChLIA platforms with the first result available in just 48 minutes and an estimated throughput of nearly 60 tests per hour. Incorporation of monoclonal antibodies to ensure specificity and consistent performance across test batches. The state-of-the-art assay is processed on the Company’s random-access iSYS TM or i10 TM instruments to deliver quick turnaround times and high-throughput testing with minimal technician training and expertise, while maintaining superior accuracy and reliability. The assay provides direct measurement of free testosterone levels in a single test, enhancing diagnostic capabilities for conditions such as hypogonadism, impotence, polycystic ovarian syndrome (PCOS), and other androgenital syndromes. "Laboratory customers have been asking for a user-friendly FDA-cleared test, on a random-access platform, for direct measurement of free testosterone," said Jonathan Friend, general manager at Revvity’s EUROIMMUN US. "This clearance reinforces our commitment to expanding the FDA-cleared menu for the EUROIMMUN family of ChLIA automation solutions with assays that serve diverse patient populations across all demographics.” For more information about EUROIMMUN and its diagnostic solutions, please visit this webpage . About Revvity At Revvity, “impossible” is inspiration, and “can’t be done” is a call to action. Revvity provides health science solutions, technologies, expertise, and services that deliver complete workflows from discovery to development, and diagnosis to cure. Revvity is revolutionizing what’s possible in healthcare, with specialized focus areas in translational multi-omics technologies, biomarker identification, imaging, prediction, screening, detection and diagnosis, informatics and more. With 2023 revenue of more than $2.7 billion and over 11,000 employees, Revvity serves customers across pharmaceutical and biotech, diagnostic labs, academia and governments. It is part of the S&P 500 index and has customers in more than 190 countries. Stay updated by following our Newsroom , LinkedIn , X , YouTube , Facebook and Instagram .

Substantial, Rapid and Sustained Statistically Significant Reductions of Key Biomarkers Achieved Across Doses, Including up to 80% Mean Reduction of Androstenedione Meaningful Improvements Demonstrated in Multiple Clinical Signs and Symptoms of CAH Affecting Patient Health Safety and Efficacy Data Support Initiation of Phase 3 Clinical Trial Management to Host Investor Conference Call Today at 8:30 AM ET SAN DIEGO, Jan. 10, 2025 (GLOBE NEWSWIRE) -- Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX) today announced positive topline results from an open-label, Phase 2 congenital adrenal hyperplasia (CAH) study of investigational atumelnant, a novel, once-daily oral adrenocorticotropic hormone (ACTH) receptor antagonist candidate being developed for the treatment of classic CAH and ACTH-dependent Cushing’s syndrome. “These exciting results show atumelnant not only lowered key biomarkers, but also had a significant impact on the signs and symptoms of CAH that are important to the overall health of people living with this condition,” said Scott Struthers, Ph.D., founder and chief executive officer of Crinetics. “We are eager to move forward with a global Phase 3 pivotal trial for adults in CAH, as we simultaneously prepare to start a Phase 2b/3 trial in pediatric patients this year. Our internally discovered pipeline now has two drug candidates with positive later stage data, and we look forward to submitting INDs for four additional candidates now in first-in-human enabling studies, as we continue our strategy for building the premier global endocrine company.” “There has been a long-standing interest in using a potent, selective antagonist of the ACTH receptor for the treatment of CAH and other diseases of ACTH excess, leading to the design of atumelnant by Crinetics scientists,” said Dr. Alan Krasner, M.D., chief endocrinologist of Crinetics. “This Phase 2 study demonstrated that atumelnant was well tolerated and resulted in a reduction of adrenal androgen levels so rapid and robust that it allowed patients to realize meaningful improvements in long-term, pre-existing medical challenges, even within the short 12-week treatment period of this study.” Highlights from the Phase 2 TouCAHn TrialThe TouCAHn trial is an open-label, global, Phase 2 study designed to evaluate the efficacy, safety, and pharmacokinetics of atumelnant when administered for 12 weeks in people with CAH caused by 21-hydroxylase deficiency. The study enrolled 28 patients across 3 dose cohorts with classic CAH on a stable dose of glucocorticoid replacement. Primary endpoints included change from baseline in morning serum androstenedione (A4) levels and incidence of treatment-emergent adverse events. Change from baseline in morning serum 17-hydroxyprogesterone (17-OHP) was also evaluated as a secondary endpoint. Results For all doses, treatment with atumelnant resulted in rapid, substantial and sustained statistically significant reduction in A4 levels, the key biomarker for disease control (results in chart below). Primary Endpoint Atumelnant, Dosed Once DailyMean A4 Baseline*(ng/dL)A4 Change from Baseline at Week 12 (ng/dL)**40 mg (n=11) 1,213-619 (p=0.0003)80 mg (n=11)1,231-774 (p<0.0001)120 mg (n=6)1,064-954 (p<0.0001) *Morning serum levels **Least square mean change Additionally, rapid, substantial and sustained statistically significant reductions in 17-OHP, a confirmatory secondary biomarker of disease control, were achieved across doses. Treatment with atumelnant also had a significant impact on CAH signs and symptoms, including: Substantial reduction and normalization of testosterone in the majority of female participants (8/13),1 with 6 of the 11 impacted participants resuming mensesConsistent reduction in total adrenal volume observed across dose cohortsResolution of androgen mediated polycythemia in 5 of the 6 impacted participants Atumelnant has been generally well tolerated with no treatment-related severe or serious adverse events to date, irrespective of disease severity or dose level. No participants required dose reduction or discontinued from the trial. All adverse events to-date have been mild to moderate and generally transient. No consistent clinically important trends were observed across key safety parameters, including clinical safety laboratory values, physical examination, electrocardiogram or vital signs. The most common treatment-emergent adverse events included headache (7/28) and fatigue (5/28). Conference Call and WebcastCrinetics will host an investor conference call on January 10, 2025, at 8:30 am Eastern Time to discuss the topline results from this study. Following the live event, a replay will be available on the Investors section of the Company’s website. Dial-in Details:Domestic: 1-800-445-7795 International: 1-785-424-1699 Conference ID: CRNXQ4 Webcast: https://viavid.webcasts.com/starthere.jsp?ei=1703675&tp_key=d409cb44ff ABOUT ATUMELNANT Atumelnant, Crinetics’ second investigational compound, is the first once-daily, oral adrenocorticotropic hormone (ACTH) receptor antagonist that acts selectively at the melanocortin type 2 receptor (MC2R) on the adrenal gland. Diseases associated with excess ACTH can have significant impact on physical and mental health. Atumelnant has exhibited strong binding affinity for MC2R in preclinical models and has demonstrated suppression of adrenally derived glucocorticoids and androgens that are under the control of ACTH. Data from a 12-week Phase 2 study demonstrated compelling treatment benefits of atumelnant, evidenced by the rapid, substantial and sustained statistically significant reductions in key CAH disease related biomarkers, including androstenedione and 17-hydroxyprogesterone, in a diverse population. Atumelnant is currently in development for congenital adrenal hyperplasia and ACTH-dependent Cushing’s syndrome. For more information about the Phase 2 TouCAHn study in classic CAH, please visit clinicaltrials.gov (NCT05907291). ABOUT CRINETICS PHARMACEUTICALS Crinetics Pharmaceuticals is a clinical stage pharmaceutical company focused on the discovery, development, and commercialization of novel therapeutics for endocrine diseases and endocrine-related tumors. Crinetics’ lead development candidate, paltusotine, is the first investigational once-daily, oral, selective somatostatin receptor type 2 (SST2) nonpeptide agonist that is in clinical development for acromegaly and carcinoid syndrome associated with neuroendocrine tumors. Atumelnant is currently in development for congenital adrenal hyperplasia and ACTH-dependent Cushing’s syndrome. All of the company’s drug candidates are orally delivered, small molecule, new chemical entities resulting from in-house drug discovery efforts, including additional discovery programs addressing a variety of endocrine conditions such as hyperparathyroidism, polycystic kidney disease, Graves’ disease (including thyroid eye disease), diabetes, obesity and GPCR-targeted oncology indications. Forward-Looking Statements This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. All statements other than statements of historical facts contained in this press release are forward-looking statements, including statements regarding the plans and timelines for the clinical development of atumelnant, including the therapeutic potential and clinical benefits or safety profile thereof; the expected timing of additional data and topline results from studies of atumelnant in CAH and ACTH-dependent Cushing’s syndrome; the expected timing and initiation of Phase 3 studies of atumelnant in CAH; the therapeutic potential and clinical benefits or safety profile of paltusotine; and the future development and focus of Crinetics. In some cases, you can identify forward-looking statements by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplates,” “believes,” “estimates,” “predicts,” “potential,” “upcoming” or “continue” or the negative of these terms or other similar expressions. These forward-looking statements speak only as of the date of this press release and are subject to a number of risks, uncertainties and assumptions, including, without limitation, initial or topline data that we report may change following completion or a more comprehensive review of the data related to the clinical studies and such data may not accurately reflect the complete results of a clinical study, and the FDA and other regulatory authorities may not agree with our interpretation of such results; unexpected adverse side effects or inadequate efficacy of the company’s product candidates that may limit their development, regulatory approval and/or commercialization; clinical studies and preclinical studies may not proceed at the time or in the manner expected, or at all; the timing and outcome of research, development and regulatory review is uncertain, and Crinetics’ drug candidates may not advance in development or be approved for marketing; and the other risks and uncertainties described in the company’s periodic filings with the Securities and Exchange Commission (SEC). The events and circumstances reflected in the company’s forward-looking statements may not be achieved or occur and actual results could differ materially from those projected in the forward-looking statements. Additional information on risks facing Crinetics can be found under the heading “Risk Factors” in Crinetics’ periodic filings with the SEC, including its annual report on Form 10-K for the year ended December 31, 2023 and its Quarterly report on Form 10-Q for the quarter ended March 31, 2024, June 30, 2024, and September 30, 2024. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. Except as required by applicable law, Crinetics does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Contact: Investors: Gayathri Diwakar Head of Investor Relations gdiwakar@crinetics.com (858) 345-6340 Media: Natalie Badillo Head of Corporate Communications nbadillo@crinetics.com (858) 345-6075  1 Sample size includes female participants not on hormonal contraceptives with intact uterus. Those with restored menses following atumelnant treatment included three previously amenorrheic participants and three with previously irregular menses.

First patient enrolled in the Phase 3 CHONQUER clinical trial in patients with IDH1-mutant conventional chondrosarcoma Phase 3 PyramIDH clinical trial in patients with IDH1-mutant myelodysplastic syndromes has been initiated with sites actively recruiting across the world BOSTON, Jan. 9, 2025 /PRNewswire/ -- Servier today announced updates to two of its Phase 3 programs evaluating TIBSOVO (ivosidenib tablets) in isocitrate dehydrogenase 1 (IDH1)-mutated cancers. The first patient has been enrolled in the CHONQUER study, a pivotal Phase 3 clinical trial evaluating the efficacy and safety of TIBSOVO versus placebo in patients with IDH1-mutated conventional chondrosarcoma. In addition, the Phase 3 PyramIDH clinical trial has been initiated, and sites are actively recruiting across the globe. The PyramIDH study is evaluating TIBSOVO® (ivosidenib tablets) monotherapy and azacitidine monotherapy in the treatment of patients with IDH1-mutated myelodysplastic syndromes (MDS) who have not previously been treated with a hypomethylating agent. "As the leader in IDH-mutated cancers, Servier aspires to provide new treatment options for those impacted by newly diagnosed IDH1-mutated conventional chondrosarcoma and IDH-1 mutated MDS. The advancement of two of our Phase 3 registration enabling studies is an incredibly important milestone for our TIBSOVO clinical development program and underscores our commitment to understanding the application of IDH1m inhibition in a broad range of malignancies to meet the needs of patients," said Susan Pandya, MD, Vice President, Clinical Development and Global Head of Oncology LS/LCM, Servier Pharmaceuticals. "We are deeply grateful to the patients, families and healthcare providers who are participating in this important research." The use of TIBSOVO in patients with IDH1-mutated conventional chondrosarcoma, as well as, patients with IDH1-mutated myelodysplastic syndromes who have not previously been treated with a hypomethylating agent are investigational, and its safety and efficacy in these cases have not been evaluated by any regulatory authority. About the PyramIDH Trial (NCT06465953) PyramIDH is a pivotal Phase 3, multicenter, open label, randomized clinical trial of TIBSOVO (ivosidenib tablets) monotherapy and azacitidine monotherapy in adult patients with myelodysplastic syndromes (MDS) with an isocitrate dehydrogenase protein, 1 (IDH1) mutation, who have not received treatment with a hypomethylating agent previously. The primary endpoint of PyramIDH is complete remission (CR) or partial remission (PR) as per International Working Group (IWG) 2006 criteria at 4 months. Secondary outcome measures include overall response (OR) rate, event-free survival (EFS) and overall survival (OS). About the CHONQUER Trial (NCT06127407) CHONQUER is a pivotal Phase 3, multicenter, double-blind, randomized, placebo-controlled crossover clinical trial of TIBSOVO (ivosidenib tablets) in adult patients with locally advanced or metastatic conventional chondrosarcoma with an isocitrate dehydrogenase protein, 1 (IDH1) mutation who are untreated or were previously treated with one systemic treatment regimen in the advanced/metastatic setting for conventional chondrosarcoma. The primary endpoint of CHONQUER is progression-free survival (PFS) in Grades 1 and 2 participants. Key secondary outcome measures include endpoints such as PFS in all randomized participants, overall survival (OS) in Grades 1 and 2 participants, and OS in all randomized participants. The study also will evaluate the impact of TIBSOVO on health-related quality of life (HRQoL) and health economic outcomes. About TIBSOVO® (ivosidenib tablets) TIBSOVO is a precision medicine that targets a specific type of mutation known as isocitrate dehydrogenase 1 (IDH1). TIBSOVO is approved in five indications globally, including approvals in the U.S., European Union, Australia and China. In the U.S., TIBSOVO is approved for the treatment of adults with IDH1-mutant relapsed or refractory AML and in monotherapy or in combination with azacitidine for adults with newly diagnosed IDH1-mutant AML who are ≥75 years old or who have comorbidities that preclude the use of intensive induction chemotherapy, as monotherapy for the treatment of adult patients with IDH1-mutant relapsed or refractory MDS, and for patients with previously treated IDH1-mutated cholangiocarcinoma. For more information about TIBSOVO in the U.S., please visit . About Myelodysplastic Syndromes Myelodysplastic Syndromes (MDS) are disorders in which progenitor (stem) cells do not mature into healthy blood cells.1 In the U.S., approximately 16,000 new cases of MDS are reported each year.2 Approximately 3.6% of MDS patients have an IDH1 mutation,3 which is considered an early "driver" mutation.4 For MDS patients with an IDH1 mutation, the prognosis has often been associated with worse overall outcomes and in an increased risk of transformation to AML.3 Prior to the approval of TIBSOVO, there were no approved targeted therapies for this molecularly defined subset. About Chondrosarcoma Chondrosarcoma is a group of bone tumors that are made up of cells that make too much cartilage.1 Chondrosarcoma, the second most common sarcoma of the bones following osteosarcoma,5 accounts for 20-30% of all skeletal sarcomas and has an estimated incidence of 1 in 200,000 per year in the U.S.6 Conventional primary chondrosarcoma is the most common variant and makes up 85% of all cases.2 IDH mutations are found in 50-70% of chondrosarcomas.7 Surgery currently remains the mainstay of treatment for conventional chondrosarcoma as both radiation and chemotherapy have been shown to be ineffective.8 About Servier in Oncology Servier is a global leader in oncology, governed by a non-profit foundation. Servier approaches innovation with a long-term vision, free of influence from fiduciary responsibilities. Servier is the leader in IDH-mutant targeted therapies and devotes more than 65% of its research and development budget to Oncology. Servier aspires to advance more targeted therapies by identifying mutations and understanding how these mutations impact cancer and its progression. Servier believes we can serve more people by helping the right patients find the right treatment, at the right time. Servier takes a One Innovation Engine approach to R&D and is actively seeking alliances, partnerships and acquisitions at various stages of the portfolio. For more information about working with Servier to bring the promise of tomorrow to the patients it serves, visit Servier.us. Media Contact Sara Noonan [email protected] TIBSOVO IMPORTANT SAFETY INFORMATION AND INDICATION FOR U.S. PATIENTS INDICATIONS TIBSOVO is indicated for patients with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test with: Newly Diagnosed Acute Myeloid Leukemia (AML) In combination with azacitidine or as monotherapy for the treatment of newly diagnosed AML in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy Relapsed or Refractory AML For the treatment of adult patients with relapsed or refractory AML Relapsed or Refractory Myelodysplastic Syndromes (MDS) For the treatment of adult patients with relapsed or refractory myelodysplastic syndromes Locally Advanced or Metastatic Cholangiocarcinoma For the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma who have been previously treated IMPORTANT SAFETY INFORMATION WARNINGS AND PRECAUTIONS Differentiation Syndrome in AML and MDS: Differentiation syndrome is associated with rapid proliferation and differentiation of myeloid cells and may be life-threatening or fatal. Symptoms of differentiation syndrome in patients treated with TIBSOVO included noninfectious leukocytosis, peripheral edema, pyrexia, dyspnea, pleural effusion, hypotension, hypoxia, pulmonary edema, pneumonitis, pericardial effusion, rash, fluid overload, tumor lysis syndrome and creatinine increased. If differentiation syndrome is suspected, initiate dexamethasone 10 mg IV every 12 hours (or an equivalent dose of an alternative oral or IV corticosteroid) and hemodynamic monitoring until improvement. If concomitant noninfectious leukocytosis is observed, initiate treatment with hydroxyurea or leukapheresis, as clinically indicated. Taper corticosteroids and hydroxyurea after resolution of symptoms and administer corticosteroids for a minimum of 3 days. Symptoms of differentiation syndrome may recur with premature discontinuation of corticosteroid and/or hydroxyurea treatment. If severe signs and/or symptoms persist for more than 48 hours after initiation of corticosteroids, interrupt TIBSOVO until signs and symptoms are no longer severe. QTc Interval Prolongation: Patients treated with TIBSOVO can develop QT (QTc) prolongation and ventricular arrhythmias. Concomitant use of TIBSOVO with drugs known to prolong the QTc interval (e.g., anti-arrhythmic medicines, fluoroquinolones, triazole anti-fungals, 5-HT3 receptor antagonists) and CYP3A4 inhibitors may increase the risk of QTc interval prolongation. Conduct monitoring of electrocardiograms (ECGs) and electrolytes. In patients with congenital long QTc syndrome, congestive heart failure, electrolyte abnormalities, or those who are taking medications known to prolong the QTc interval, more frequent monitoring may be necessary. Interrupt TIBSOVO if QTc increases to greater than 480 msec and less than 500 msec. Interrupt and reduce TIBSOVO if QTc increases to greater than 500 msec. Permanently discontinue TIBSOVO in patients who develop QTc interval prolongation with signs or symptoms of life-threatening arrhythmia. Guillain-Barré Syndrome: Guillain-Barré syndrome can develop in patients treated with TIBSOVO. Monitor patients taking TIBSOVO for onset of new signs or symptoms of motor and/or sensory neuropathy such as unilateral or bilateral weakness, sensory alterations, paresthesias, or difficulty breathing. Permanently discontinue TIBSOVO in patients who are diagnosed with Guillain-Barré syndrome. ADVERSE REACTIONS In patients with AML, the most common adverse reactions including laboratory abnormalities (≥25%) are leukocytes decreased, diarrhea, hemoglobin decreased, platelets decreased, glucose increased, fatigue, alkaline phosphatase increased, edema, potassium decreased, nausea, vomiting, phosphate decreased, decreased appetite, sodium decreased, leukocytosis, magnesium decreased, aspartate aminotransferase increased, arthralgia, dyspnea, uric acid increased, abdominal pain, creatinine increased, mucositis, rash, electrocardiogram QT prolonged, differentiation syndrome, calcium decreased, neutrophils decreased, and myalgia In patients with MDS, the most common adverse reactions including laboratory abnormalities (≥25%) are creatinine increased, hemoglobin decreased, arthralgia, albumin decreased, aspartate aminotransferase increased, fatigue, diarrhea, cough, sodium decreased, mucositis, decreased appetite, myalgia, phosphate decreased, pruritus, and rash In patients with cholangiocarcinoma, the most common adverse reactions (≥15%) in patients with cholangiocarcinoma are fatigue, nausea, abdominal pain, diarrhea, cough, decreased appetite, ascites, vomiting, anemia, and rash. The most common laboratory abnormalities (≥10%) in patients with cholangiocarcinoma are hemoglobin decreased, aspartate aminotransferase increased, and bilirubin increased DRUG INTERACTIONS Strong or Moderate CYP3A4 Inhibitors: Reduce TIBSOVO dose with strong CYP3A4 inhibitors. Monitor patients for increased risk of QTc interval prolongation. Strong CYP3A4 Inducers: Avoid concomitant use with TIBSOVO. Sensitive CYP3A4 substrates: Avoid concomitant use with TIBSOVO. QTc Prolonging Drugs: Avoid concomitant use with TIBSOVO. If co-administration is unavoidable, monitor patients for increased risk of QTc interval prolongation. LACTATION: Advise women not to breastfeed. Please see Full Prescribing Information, including BOXED WARNING for AML and MDS patients 1 Pagliuca S, Gurnari C, Visconte V. Molecular Targeted Therapy in Myelodysplastic Syndromes: New Options for Tailored Treatments. Cancers. 2021;13(4):784. . 2 SEER MDS Incidence Rates. Available at: ; U.S. Census Bureau Population Estimates 2017 National Population Projections Tables: Main Series (census.gov). . Accessed December 5, 2024. 3 Thol F, Weissinger EM, Krauter J, et al. IDH1 mutations in patients with myelodysplastic syndromes are associated with an unfavorable prognosis. Haematologica. 2010;95(10)1668-1674. . Accessed December 5, 2024. 4 DiNardo CD, Jabbour E, Ravandi F, et al. IDH1 and IDH2 mutations in myelodysplastic syndromes and role in disease progression. Leukemia. 2016;30(4):980-4. . Accessed December 5, 2024. 5 Anfinsen KP, Devesa SS, Bray F, et al. Age-Period-Cohort Analysis of Primary Bone Cancer Incidence Rates in the United States (1976–2005) Cancer Epidemiol. Prev. Biomark. 2011;20:1770-1777. . Accessed December 5, 2024. 6 Giuffrida AY, Burgueno JE, Koniaris LG, et al. Chondrosarcoma in the United States (1973 to 2003): An Analysis of 2890 Cases from the SEER Database. JBJS. 2009;91:1063-1072. . Accessed December 5, 2024. 7 Amary MF, Bacsi K, Maggiani F, et al. IDH1 and IDH2 Mutations Are Frequent Events in Central Chondrosarcoma and Central and Periosteal Chondromas but Not in Other Mesenchymal Tumours. J. Pathol. 2011;224:334-343. . Accessed December 5, 2024. 8 Polychronidou G, Karavasilis V, Pollack SM, et al. Novel Therapeutic Approaches in Chondrosarcoma. Future Oncol. 2017;13:637-648. . Accessed December 5, 2024. SOURCE Servier Pharmaceuticals WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED