ENDO

Rhythm Pharmaceuticals Presents Patient-reported Experiences with Hyperphagia in Hypothalamic Obesity at ENDO 2024

Retrieved on: 
月曜日, 6月 3, 2024
ENDO, Metabolism, Polyphagia, BMI, TBX3, Patient, University, Translation, Caregiver, NIH, Setmelanotide, University of Michigan, Sleep, LEPR, Body mass index, MEND, Associate, Hypothalamic disease, BBS, Bardet–Biedl syndrome, Division, Quality of life, POMC, Insulin resistance, University of Washington, Proopiomelanocortin, MC4R, WHO, Obesity, Chronic, SIM1, Pharmaceutical industry

These results were among six Rhythm presentations at the Endocrine Society Annual Meeting & Expo (ENDO 2024) being held June 1-4 in Boston.

Key Points: 
  • These results were among six Rhythm presentations at the Endocrine Society Annual Meeting & Expo (ENDO 2024) being held June 1-4 in Boston.
  • After one year of treatment with setmelanotide, improvements of at least one obesity class were observed across all cohorts (HO: 92%; POMC deficiency: 89%; LEPR deficiency: 36%; BBS: 42%).
  • Treatment was well-tolerated and stable weight and food intake reductions and improved insulin sensitivity were observed after three weeks.
  • Dr. Roth presented the design of Rhythm’s first-in-human Phase 1 trial of once-weekly RM-718 in individuals with obesity or patients with HO.

Crinetics Presents New Data at ENDO 2024 that Increases Body of Evidence Positioning Once-Daily, Oral Paltusotine as Potential First-Choice Treatment Option for Acromegaly

Retrieved on: 
月曜日, 6月 3, 2024
ENDO, Metabolism, Acromegaly, Safety, Patient, Endocrine system, Manuscript, ULN, PRO, Prior, Journal, Abstract, Pharmaceutical industry

SAN DIEGO, June 03, 2024 (GLOBE NEWSWIRE) -- Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX) today presented findings from its clinical development program evaluating oral, once-daily investigational paltusotine in acromegaly. Data presented included results of the Phase 3 PATHFNDR-2 trial, a new analysis of patient reported outcome (PRO) data from the Phase 3 PATHFNDR-1 trial, and interim long-term efficacy and safety results at 42 months from the open-label ACROBAT Advance extension study. The data were presented today at the Endocrine Society’s Annual Meeting (ENDO2024), with findings from PATHFNDR-1 published as a manuscript in The Journal of Clinical Endocrinology & Metabolism.

Key Points: 
  • Among those treated with paltusotine, a reduction in IGF-1 levels occurred in 92.6% of patients (n=50/54) by the end of treatment.
  • The frequency of participants with at least one treatment emergent adverse event (TEAE) was comparable in the paltusotine treatment arm and placebo arm.
  • Findings clearly demonstrated that once-daily, oral paltusotine maintained biochemical and symptom control in patients with acromegaly switched from SRL.
  • Once-daily oral paltusotine treatment was well-tolerated, with stable biochemical and symptom control, comparable to that observed with prior injected SRLs.

Crinetics Announces Positive Initial Findings at ENDO 2024 for Atumelnant in Two Ongoing, Open-Label Studies for the Treatment of Congenital Adrenal Hyperplasia (CAH) and ACTH-Dependent Cushing’s Syndrome (ADCS)

Retrieved on: 
月曜日, 6月 3, 2024
ENDO, Aes, Headache, A4, Fatigue, Diabetes, Endocrine system, CAH, ULN, Glucocorticoid, Safety, SAN, UFC, ACTH, Cohort, Upper respiratory tract infection, Single Center, Abstract, Food, Human, ADCS, University College Hospital, Congenital adrenal hyperplasia, Endocrinology, Bangladesh Technical Education Board, 24 Hours, Pharmaceutical industry

SAN DIEGO, June 03, 2024 (GLOBE NEWSWIRE) -- Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX) today announced initial findings from the development program of its second clinical product candidate, atumelnant* (CRN04894), a novel, once-daily oral adrenocorticotropic hormone (ACTH) receptor antagonist. The results, presented at the Endocrine Society’s annual meeting (ENDO2024), include initial data from the Phase 1b/2a, open-label study in participants with ACTH-dependent Cushing’s syndrome (ADCS) conducted in collaboration with the National Institutes of Health, and the Phase 2 open-label TouCAHn study in participants with congenital adrenal hyperplasia (CAH).

Key Points: 
  • The trial continues to enroll three treatment cohorts: 80 mg once daily (n=9), 40 mg once daily (n=9) and 120 mg once daily (n=6).
  • Available data for 80 mg includes: n=4 at 12 weeks of treatment, with two additional participant’s data up to six weeks of treatment.
  • The TouCAHn study is ongoing, with topline results from the complete study expected in the second half of 2024.
  • Crinetics will host an investor conference call on June 3, 2024, at 4:30 pm Eastern Time to discuss the initial findings from these two studies.

Entera Bio Reports Phase 1 Clinical Data of First-in-Class, Oral PTH(1-34) Peptide Candidate (EB612) for Patients with Hypoparathyroidism at ENDO 2024

Retrieved on: 
月曜日, 6月 3, 2024
ENDO, Therapy, QID, Entera, Injection, Patient, Annual general meeting, Serum, Trial of the century, Safety, Ish-Shalom, Teriparatide, Hypoparathyroidism, Life, Hypercalcaemia, Blood, PD, PTH, Calcium, Vital signs, BID, Endocrine Society, Tablet

Entera’s EB612 program aims to provide the first oral PTH daily tablet hormone replacement therapy for patients suffering from hypoparathyroidism.

Key Points: 
  • Entera’s EB612 program aims to provide the first oral PTH daily tablet hormone replacement therapy for patients suffering from hypoparathyroidism.
  • Late stage investigational PTH replacement treatments include TransCon PTH (palopegteriparatide) by Ascendis Pharma A/S and eneboparatide (AZP-3601) by Amolyt Pharma.
  • Entera previously published positive Phase 2a study results in hypoparathyroid patients with four times a day (QID) regimen (Ish-Shalom, JBMR 2021) of EB612.
  • “The encouraging findings from this Phase 1 study reaffirm the ability of our N-Tab™ platform to develop simple oral tablet treatments of important peptide therapeutics.

Corcept Therapeutics Announces Presentations of Results of Pivotal Phase 3 GRACE Trial Evaluating Relacorilant in Patients with Hypercortisolism (Cushing’s Syndrome)

Retrieved on: 
月曜日, 6月 3, 2024
ENDO, Syndrome, Patient, HID, Oncology, Heart, GRACE, Doctor of Philosophy, Endocrine Society, Saint Andrew's Society, Conference

MENLO PARK, Calif., June 03, 2024 (GLOBE NEWSWIRE) -- Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of medications to treat severe endocrinologic, oncologic, metabolic and neurologic disorders by modulating the effects of the hormone cortisol, today announced that the results from GRACE, the Phase 3 trial of its proprietary selective cortisol modulator relacorilant in patients with hypercortisolism (Cushing’s syndrome), will be presented at the Endocrine Society (ENDO) annual meeting in Boston (June 3) and the Heart in Diabetes (HiD) conference in Philadelphia (June 7).

Key Points: 
  • MENLO PARK, Calif., June 03, 2024 (GLOBE NEWSWIRE) -- Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of medications to treat severe endocrinologic, oncologic, metabolic and neurologic disorders by modulating the effects of the hormone cortisol, today announced that the results from GRACE, the Phase 3 trial of its proprietary selective cortisol modulator relacorilant in patients with hypercortisolism (Cushing’s syndrome), will be presented at the Endocrine Society (ENDO) annual meeting in Boston (June 3) and the Heart in Diabetes (HiD) conference in Philadelphia (June 7).
  • Presenter: Rosario Pivonello, MD, PhD, Principal Investigator of the GRACE study and Professor of Endocrinology at Università Federico II di Napoli, Italy
    Session: P108 - Late-Breaking Poster Presentations: Day 3
    8th Heart in Diabetes Conference, Hilton Penn’s Landing
    Oral Presentation: Medical Treatment of Hypercortisolism with Relacorilant: Final Results of the Phase 3 GRACE Study
    Presenter: Rosario Pivonello, MD, PhD, Principal Investigator of the GRACE study and Professor of Endocrinology at Università Federico II di Napoli, Italy
    Each of the above presentations will be available on Corcept.com following the event.

Spruce Biosciences Presented Phase 2 POWER Study Results of Tildacerfont for the Treatment of Polycystic Ovary Syndrome at ENDO 2024

Retrieved on: 
月曜日, 6月 3, 2024
Biotechnology, Pharmaceutical, Health, Clinical Trials, ENDO, POWER, DHEAS, Patient, PCOS, FACOG, Gynaecology, CRF1, Polycystic ovary syndrome, Reproductive Sciences, Demography, Woman, Polycystic disease, Diagnosis, MBBS, FDA, Endocrine Society, Yale School of Medicine, Participation, Pharmaceutical industry

(Nasdaq: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for endocrine disorders with significant unmet medical need, presented results from its Phase 2 POWER study of tildacerfont, a second-generation CRF1 receptor antagonist, for the treatment of polycystic ovary syndrome (PCOS) at the 2024 Annual Meeting of the Endocrine Society (ENDO 2024).

Key Points: 
  • (Nasdaq: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for endocrine disorders with significant unmet medical need, presented results from its Phase 2 POWER study of tildacerfont, a second-generation CRF1 receptor antagonist, for the treatment of polycystic ovary syndrome (PCOS) at the 2024 Annual Meeting of the Endocrine Society (ENDO 2024).
  • Further studies of tildacerfont for the treatment of PCOS are warranted, and we continue to evaluate strategic collaboration opportunities to advance this potentially disease-modifying treatment option forward.”
    The POWER study enrolled 27 women with a confirmed diagnosis of PCOS.
  • Participant demographics and baseline hormone levels are detailed in Table 1 below.
  • Summary of Demographics and Baseline Hormones; Intent to Treat Analysis Population

Xeris Presents New Post Hoc Analysis on Effects of Levoketoconazole (Recorlev®) in Cushing’s Syndrome Patients at ENDO 2024

Retrieved on: 
月曜日, 6月 3, 2024
Health, Clinical Trials, Research, Pharmaceutical, Science, Biotechnology, ENDO, ITT, Aes, Group 2, Quantile, Patient, ULN, Doctor of Pharmacy, Publication, UFC, Female, Group 3, Incidence, Disease, Group 1, ALT, GGT

This exploratory analysis brings further perspective to the importance of individualizing and tailoring medical management,” said James Meyer, PharmD, Xeris’ Senior Director, Publications and Medical Communications.

Key Points: 
  • This exploratory analysis brings further perspective to the importance of individualizing and tailoring medical management,” said James Meyer, PharmD, Xeris’ Senior Director, Publications and Medical Communications.
  • Daily doses of levoketoconazole were related to baseline mUFC.
  • Group 3 had a higher incidence of liver test (ALT, AST, GGT) abnormalities compared to Group 1 and Group 2.
  • This post hoc analysis demonstrated:
    Normalization of mUFC with levoketoconazole in Cushing’s syndrome patients maintained on levoketoconazole in the SONICS study for up to 6 months appeared to vary inversely with baseline mUFC.

Veracyte Announces that New Afirma GRID Data Suggest Prognostic Ability of Molecular Testing for Thyroid Tumors

Retrieved on: 
月曜日, 6月 3, 2024
Science, Other Science, Biotechnology, Research, Pharmaceutical, Oncology, Health, Genetics, ENDO, Endocrinology, GSC, Trial of the century, Aggression, Patient, Thyroid, Signature, Cancer, Intelligence, Thyroid cancer, Thyroid nodule, Prostate cancer, Pathology, Metastasis, B.A, Endocrine Society, Risk, Gene expression, FOLH1, Progression-free survival, Fibroblast, Vanderbilt University Medical Center, Correlation, FNA

“Clinicians generally want to avoid overtreatment of patients with non-aggressive thyroid tumors while targeting more-intensive treatment to those patients with aggressive disease.

Key Points: 
  • “Clinicians generally want to avoid overtreatment of patients with non-aggressive thyroid tumors while targeting more-intensive treatment to those patients with aggressive disease.
  • The challenge is distinguishing between them, especially given the heterogeneity of thyroid tumors,” said Joshua Klopper, M.D., Veracyte’s medical director for Endocrinology.
  • “The studies presented at ENDO 2024 today show how our Research-Use-Only Afirma GRID tool, which leverages our whole-transcriptome-derived testing approach, is helping scientists better understand important nuances in the molecular underpinnings of thyroid tumors.
  • Researchers leveraged the Afirma GRID database to characterize the expression of PSMA (FOLH1) in a cohort of nearly 50,000 thyroid nodules sent for Afirma GSC molecular testing.

Septerna Presents Preclinical Data at ENDO 2024 Highlighting Therapeutic Potential of its GPCR Drug Discovery Platform

Retrieved on: 
月曜日, 6月 3, 2024
Health, Genetics, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, ENDO, Molecule, Injection (medicine), Thyroid, Endocrine system, Patient, Hyperthyroidism, TED, Concept, Peptide, Autoantibody, Kidney, Hypoparathyroidism, PTH1R, Graves' ophthalmopathy, Gene, PTH, Tissue, G protein-coupled receptor, Poster, Drug discovery

The data were presented in two poster presentations and a rapid-fire oral presentation during the Endocrine Society’s Annual Meeting (ENDO 2024) held June 1-4, 2024, in Boston, MA.

Key Points: 
  • The data were presented in two poster presentations and a rapid-fire oral presentation during the Endocrine Society’s Annual Meeting (ENDO 2024) held June 1-4, 2024, in Boston, MA.
  • These results suggest that oral small molecule PTH1R agonists may be suitable alternatives to injectable PTH peptides for the treatment of hypoparathyroidism.
  • The company plans to initiate a Phase 1 trial with its lead PTH1R agonist candidate in healthy volunteers in late 2024.
  • The data also demonstrated sustained control of serum calcium and phosphate levels over 28 days with daily oral administration.

Ascendis to Spotlight Latest Updates for TransCon™ Rare Endocrinology Disease Portfolio at ENDO 2024

Retrieved on: 
木曜日, 5月 30, 2024
ENDO, Achondroplasia, Endocrine Society, Drug, Health, GHD, Saint Andrew's Society, Hypoparathyroidism, Turner syndrome

“In addition to physician and caregiver presentations in pediatric GHD, we look forward to our first in-person opportunity to share data from foresiGHt, our pivotal trial of TransCon hGH in adults with GHD,” said Aimee Shu, M.D., Ascendis Pharma’s Senior Vice President of Clinical Development, Endocrine Medical Sciences.

Key Points: 
  • “In addition to physician and caregiver presentations in pediatric GHD, we look forward to our first in-person opportunity to share data from foresiGHt, our pivotal trial of TransCon hGH in adults with GHD,” said Aimee Shu, M.D., Ascendis Pharma’s Senior Vice President of Clinical Development, Endocrine Medical Sciences.
  • “And with World Hypoparathyroidism Day coinciding with the first day of ENDO, we are pleased to host an informational booth focused on deepening physician awareness of the significant health and quality-of-life impacts of hypoparathyroidism.”
    Ascendis presentations during ENDO 2024 include:
    June 3, 2:45 – 3:00 p.m ET.
  • Baseline Demographics and Design of the SkybriGHt Registry Study.
  • A US Multi-Center, Prospective, Non-Interventional, Long-Term Effectiveness and Safety Study of Children and Adolescents with Growth Hormone Deficiency Treated with Lonapegsomatropin