Pediatric endocrinology

Rhythm Pharmaceuticals Announces Updates on MC4R Pathway Programs at R&D Event

Retrieved on: 
Wednesday, December 6, 2023
Bardet–Biedl syndrome, Quality of life, Genotype, Food, Stage 2, Hyperpigmentation, Life, Shanda, Polyphagia, FDA, Type, Weight loss, POMC, BMI, European, Disease, MC1R, University of Florida, MD, Pediatric endocrinology, New Drug Application, University, SIM1, Obesity, TBX3, Endocrinology, PCSK1, Human, Patient, Proopiomelanocortin, Hunger, BBS, LEPR, Pharmaceutical industry

-- Company to host R&D webcast today at 8:00 a.m. ET--

Key Points: 
  • “Today, we are excited to showcase our commitment to understanding the MC4R pathway and developing the next generation of therapies,” said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm.
  • The hyperphagia and severe obesity of rare genetically-caused MC4R pathway diseases can present early in life, and these data show potential efficacy in patients younger than 6.
  • A live webcast of the R&D call will be available under "Events and Presentations" in the Investor Relations section of the Rhythm Pharmaceuticals website at https://ir.rhythmtx.com/ .
  • The archived webcast will be available on Rhythm Pharmaceuticals’ website approximately two hours after the conference call and will be available for 30 days following the call.

Lumos Pharma to Host Virtual Key Opinion Leader Webinar to Discuss Phase 2 OraGrowtH Trial Data and Treatment Potential of Oral LUM-201 for Pediatric Growth Hormone Deficiency

Retrieved on: 
Tuesday, November 21, 2023
Mother, Child, University of Chile, MD, Pediatric endocrinology, Research, University, LUMO, KOL, Pharmaceutical industry

AUSTIN, Texas, Nov. 21, 2023 (GLOBE NEWSWIRE) -- Lumos Pharma, Inc. (NASDAQ:LUMO), a biopharmaceutical company advancing an oral therapeutic candidate for moderate Pediatric Growth Hormone Deficiency (PGHD), announced today that the Company will host a key opinion leader (KOL) webinar to discuss topline results from its Phase 2 OraGrowtH210 and OraGrowtH212 Trials in greater detail.

Key Points: 
  • AUSTIN, Texas, Nov. 21, 2023 (GLOBE NEWSWIRE) -- Lumos Pharma, Inc. (NASDAQ:LUMO), a biopharmaceutical company advancing an oral therapeutic candidate for moderate Pediatric Growth Hormone Deficiency (PGHD), announced today that the Company will host a key opinion leader (KOL) webinar to discuss topline results from its Phase 2 OraGrowtH210 and OraGrowtH212 Trials in greater detail.
  • Management will be joined by three esteemed thought leaders in the field of endocrinology:
    Andrew Dauber, MD, Chief of Endocrinology at Children's National Medical Center, Washington, D.C.
    Fernando Cassorla, MD, Chief of Pediatric Endocrinology at the Institute of Maternal and Child Research, University of Chile
    Virtual KOL Event: Review of Phase 2 OraGrowtH Trial Data and Treatment Potential of Oral LUM-201 for Pediatric Growth Hormone Deficiency Wednesday, December 6, 2023 | 1:30 PM EST
    To register for the virtual KOL Event, please click through the link here .

Tonix Pharmaceuticals Announces Enrollment Initiated in Mass General Brigham Phase 2 Investigator-Initiated Study of TNX-1900 (Intranasal Potentiated Oxytocin) for Bone Health in Children with Autism Spectrum Disorder

Retrieved on: 
Monday, November 13, 2023
Vitamin D, ASD, Tonix Pharmaceuticals, Osteoporosis, Woman, Adolescence, Safety, MGH, Calcium, Prevalence, Endocrinology, Bone density, IND, BOX, Spectrum, Division, Oxytocin, MPH, Massachusetts General Hospital, Pediatric endocrinology, Neurology, Autism spectrum, Biology, Hospital, Dietary supplement, Pediatrics

CHATHAM, N.J., Nov. 13, 2023 (GLOBE NEWSWIRE) -- Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a biopharmaceutical company with marketed products and a pipeline of development candidates, today announced that the first participant was enrolled in an investigator-initiated Phase 2 study of TNX-1900 (intranasal potentiated oxytocin) for improving bone health in children with autism spectrum disorder (ASD), named the BOX study, at Massachusetts General Hospital (MGH). The aim of this Department of Defense-funded study is to investigate the efficacy and safety of TNX-1900 as a novel therapeutic agent to increase bone density and improve bone structure and strength in children with ASD. Tonix is providing active drug and placebo for the BOX study as part of a drug donation agreement with MGH. MGH is the sponsor of the trial, which is being conducted under an investigator-initiated investigational new drug (IND) application.

Key Points: 
  • Tonix is providing active drug and placebo for the BOX study as part of a drug donation agreement with MGH.
  • MGH is the sponsor of the trial, which is being conducted under an investigator-initiated investigational new drug (IND) application.
  • “Low bone density in ASD is a serious problem,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals.
  • A Z-score compares one’s bone density to the average bone density of age and gender matched controls.

Lumos Pharma Announces Topline Data from Phase 2 OraGrowtH210 and OraGrowtH212 Trials of LUM-201 in PGHD Met All Primary and Secondary Endpoints

Retrieved on: 
Tuesday, November 7, 2023
Research, ECG, PD, Injection, Injection (medicine), Comparable, Classification, AHV, Child, Transgender hormone therapy, PEM, KOL, University, Safety, Patient, LUMO, Unique, MD, Growth, Standard error, GH, Growth hormone, Coronavirus Scientific Advisory Board, FDA, Șaeș, Aes, Pediatric endocrinology, City of Hope National Medical Center, Mother, LSM, SDS, University of Chile, Political moderate, Silviculture, Vaccine, Pharmaceutical industry, Birth control, Wood drying, Online shopping, Endocrinology

AUSTIN, Texas, Nov. 07, 2023 (GLOBE NEWSWIRE) -- Lumos Pharma, Inc. (NASDAQ:LUMO) today announced that topline results from its Phase 2 OraGrowtH210 dose-finding trial and its Phase 2 OraGrowtH212 Pharmacokinetic/Pharmacodynamic (PK/PD) trial met all primary and secondary endpoints. Data from the OraGrowtH210 Trial demonstrated annualized height velocity (AHV) on the 1.6 mg/kg dose of orally administered LUM-201 of 8.2 cm/yr at six months and 8.0 cm/yr at 12 months on treatment,* in line with historical data in moderate pediatric growth hormone deficiency (PGHD) patients and within the targeted 2 cm/yr margin of the comparator injectable recombinant growth hormone (rhGH) arm. Data also provided preliminary validation of the predictive enrichment marker (PEM) strategy, with prespecified primary and secondary outcomes met, de-risking our patient selection for our Phase 3 program. Data from the OraGrowtH212 Trial confirmed that LUM-201’s unique pulsatile mechanism produces an increase in growth rates while restoring growth hormone secretion and IGF-1 to within normal ranges †, with levels substantially below those produced by exogenous injectable rhGH.†† Additionally, data from a small subset of 10 subjects combined 1.6 and 3.2 mg/kg dosage of LUM-201 in both OraGrowtH210 and OraGrowtH212 trials demonstrated the sustained effectiveness of AHV up to 24 months. Furthermore, the safety profile for LUM-201 remained clean throughout both Phase 2 studies, with no safety concerns identified in either of our Phase 2 trials conducted thus far.

Key Points: 
  • Data also provided preliminary validation of the predictive enrichment marker (PEM) strategy, with prespecified primary and secondary outcomes met, de-risking our patient selection for our Phase 3 program.
  • The OraGrowtH210 trial met its primary objective, with 6-month AHV data of 8.2 cm/yr supporting the 1.6 mg/kg as the optimal dose for a Phase 3 clinical trial.
  • Eighteen and 24-month growth data were available for 10 subjects from the OraGrowtH210 and OraGrowtH212 Trials who met AHV criteria per protocol at 12 months.
  • Every participant in the OraGrowtH212 Trial met the criteria for Patient PEM positivity, ensuring their potential responsiveness to LUM-201.

Lumos Pharma Reports Third Quarter 2023 Financial Results and Clinical Development Updates

Retrieved on: 
Tuesday, November 7, 2023
Research, Transgender hormone therapy, American Association of Clinical Endocrinologists, Event, Child, LUMO, Pediatric endocrinology, Therapy, City of Hope National Medical Center, Rare, IGFBP3, Mother, MD, Quarter, SDS, Endocrine Society, Clinical trial, PEM, KOL, Lumos, University, Serum, University of Chile, Political moderate, European Society of Gynaecological Oncology, Growth hormone, Investment, Consistency, AHV, Silviculture, Pharmaceutical industry, Birth control, Endocrinology

AUSTIN, Texas, Nov. 07, 2023 (GLOBE NEWSWIRE) -- Lumos Pharma, Inc. (NASDAQ:LUMO), a clinical-stage biopharmaceutical company focused on therapeutics for rare diseases, today announced that topline results met primary and secondary endpoints for its Phase 2 Dose-Finding OraGrowtH210 Trial and Phase 2 Pharmacokinetic/Pharmacodynamic (PK/PD) OraGrowtH212 Trial evaluating oral LUM-201 for subjects with moderate pediatric growth hormone deficiency (PGHD) who screened PEM-positive utilizing Lumos Pharma’s predictive enrichment marker (PEM) strategy. Lumos also announced its financial results for the quarter ended September 30, 2023.

Key Points: 
  • Lumos also announced its financial results for the quarter ended September 30, 2023.
  • The Company expects cash use of approximately $9.0 to $10.0 million in the fourth quarter of 2023.
  • Net Loss – The net loss for the quarter ended September 30, 2023 was $8.3 million compared to a net loss of $7.3 million for the same period in 2022.
  • Lumos Pharma ended the third quarter 2023 with 7,914,582 shares outstanding.

Rhythm Pharmaceuticals Announces Six Abstracts to be Presented at The Obesity Society’s Annual Meeting at ObesityWeek ®

Retrieved on: 
Tuesday, October 10, 2023
University, Marshfield Clinic, Kay Bailey Hutchison Convention Center, Division, Pediatric endocrinology, Obesity, Abstract, Research, POMC, Metabolic syndrome, LEPR, Bardet–Biedl syndrome, Columbia University, MC4R, Syndrome, Risk, University of Ulm, Adolescent medicine, Hypothalamic disease, Endocrinology, Eunice Kennedy Shriver National Institute of Child Health and Human Development, NIH, Polyphagia, Pharmaceutical industry, Medical device, Pediatrics, MD, Setmelanotide, Doctor of Philosophy, Rhythm, Patient

The lead author on this research is Wendy K. Chung, M.D., Ph.D., Division of Molecular Genetics, Department of Pediatrics, Columbia University, New York, NY.

Key Points: 
  • The lead author on this research is Wendy K. Chung, M.D., Ph.D., Division of Molecular Genetics, Department of Pediatrics, Columbia University, New York, NY.
  • Rhythm will post these data presentations on the Company’s website on the “Publications and Presentations” page following the conference.
  • This conference call will be accessible under “Events & Presentations” in the Investor Relations section of the Company’s website at www.rhythmtx.com .
  • A replay will be available on the Rhythm website for 30 days following the presentation.

Rhythm Pharmaceuticals Presents New Data at ESPE 2023

Retrieved on: 
Friday, September 22, 2023
Polyphagia, LEPR, Adolescent medicine, University of Alberta, Risk, Obesity, University, Pediatrics, Metabolic syndrome, Doctor of Philosophy, Genetics, Pediatric endocrinology, Bardet–Biedl syndrome, ESPE, Endocrine Society, POMC, ROAD, BBS, Early-onset Alzheimer's disease, Patient, Division, Endocrinology, University of Ulm, Medicine, Body mass index, MC4R, European Society for Paediatric Research, Imperial College London, Proopiomelanocortin, Vaccine, Nursing, Rhythm, The Hague

BOSTON, Sept. 22, 2023 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases, today announced four oral presentations at the 61st Annual European Society for Paediatric Endocrinology (ESPE) Meeting being held September 21-23, 2023 in The Hague, Netherlands.

Key Points: 
  • BOSTON, Sept. 22, 2023 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases, today announced four oral presentations at the 61st Annual European Society for Paediatric Endocrinology (ESPE) Meeting being held September 21-23, 2023 in The Hague, Netherlands.
  • “We are excited to deliver multiple presentations at ESPE 2023 including statistics that deepen the understanding of the genetics of obesity from our Rare Obesity Advanced Diagnosis™ (ROAD) testing program as we learn more about the importance of genetics in patients with early-onset, severe obesity,” said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm.
  • “Frequency of Obesity-Related Gene Variants in a European Population with Early-Onset, Severe Obesity,” as presented orally by Jesús Argente M.D., Ph.D., Department of Pediatrics and Pediatric Endocrinology, Universidad Autónoma de Madrid in Spain, shows that among individuals with early-onset, severe obesity, approximately 31.4% carried variants believed to be associated with obesity.
  • In addition, Rhythm is hosting a satellite symposium at ESPE 2023, titled, “Hyperphagia and early-onset, severe obesity: The role of precision medicine in the treatment of leptin melanocortin-4 receptor (MC4R) pathway diseases.”

Abata Therapeutics Announces Second Development Candidate, ABA-201, a Novel Treg Cell Therapy for the Treatment of Type 1 Diabetes

Retrieved on: 
Thursday, August 24, 2023
Biotechnology, Diabetes, Health, Pharmaceutical, Clinical Trials, Linda, Partnership, University, Pediatrics, Therapy, NOD, Outcomes research, Pediatric endocrinology, Immune tolerance, Pancreas, University of California, Research, Multiple sclerosis, Inflammation, Autoimmunity, Assistant, TCR, Harvard Medical School, ITN, Behavior, Indiana University School of Medicine, Patient, Division, Lymph, Insulitis, Benaroya Research Institute, Endocrinology, Biology, Joslin Diabetes Center, Pharmaceutical industry, Nursing, T1D, Regulatory T cell, Medicine

“We are rapidly developing ABA-201 for T1D and expect to begin clinical studies in 2025.

Key Points: 
  • “We are rapidly developing ABA-201 for T1D and expect to begin clinical studies in 2025.
  • Additional non-clinical studies have established the role of Tregs in suppressing β-cell injury, a further potential benefit of the approach.
  • “This second program expands the scope of our pioneering Treg cell-based approach to patients with T1D.
  • This group brings deep expertise in T1D pathogenesis and the clinical development of novel therapeutics.

Independent Panel of Renowned Pediatric Endocrinologists to Discuss Pediatric Growth Hormone Deficiency and the Therapeutic Landscape

Retrieved on: 
Tuesday, August 22, 2023
LUMO, KOL, Pediatrics, University, Orange County, Pediatric endocrinology, Indiana University School of Medicine, Section, Chlorine, CHKD, Associate, Harvard Medical School, Division, Mass, Hospital, Walter L. Miller (endocrinologist), Research, MPH, Massachusetts General Hospital, CHOC, Nursing, MD, Endocrinology

Alejandro Diaz, MD, Chief of the Division of Pediatric Endocrinology at Niclaus Children's Pediatric Specialists in Miami, FL.

Key Points: 
  • Alejandro Diaz, MD, Chief of the Division of Pediatric Endocrinology at Niclaus Children's Pediatric Specialists in Miami, FL.
  • Erica A. Eugster, MD, Professor of Pediatrics and Division Chief in Pediatric Endocrinology/Diabetology and Co-Director of the Pediatric Endocrine Fellowship Program at the Indiana University School of Medicine.
  • Kent Reifschneider MD, Associate Professor of Pediatrics in the Division of Endocrinology and Children’s Specialty Group at the Children's Hospital of The King's Daughters (CHKD), Norfolk, VA.
  • The moderated panel discussion will be followed by a live question and answer session.

SAB Biotherapeutics Provides Company Update for Q2 2023 Financial Results

Retrieved on: 
Monday, August 21, 2023
SAB, Post-exposure prophylaxis, GLP, JDRF, Serum sickness, New Animal Drug Application, LLP, BIO, Omicron, Higgs, KOL, University, FDA Center for Devices and Radiological Health, Partnership, Diabetes, Blas, T1D, Type A and Type B personality theory, Safety, Pediatric endocrinology, COVID-19, Center for Biologics Evaluation and Research, BTD, G&A, Immunotherapy, Antibody, Breakthrough therapy, Professor, Federation, HIV disease progression rates, Patient, NADA, Administration, International Society, ATG, Endocrinology, Entrepreneurship, Research and development, Blaine Higgs, Federation of Clinical Immunology Societies, FDA, Private law, R, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, US Foods, NIH, Treatment, Type 1 diabetes, Food, Vaccine, Pharmaceutical industry, Medical device, Cryptocurrency, Research

SIOUX FALLS, S.D., Aug. 21, 2023 (GLOBE NEWSWIRE) -- SAB Biotherapeutics ( Nasdaq: SABS ), (SAB), a clinical-stage biopharmaceutical company with a novel immunotherapy platform that produces specifically targeted, high-potency, fully-human immunoglobulins (hIgG), also known as fully-human polyclonal antibodies, without the need for human donors, today reported financial results for the second quarter ended June 30, 2023, and provided a company update.

Key Points: 
  • SIOUX FALLS, S.D., Aug. 21, 2023 (GLOBE NEWSWIRE) -- SAB Biotherapeutics ( Nasdaq: SABS ), (SAB), a clinical-stage biopharmaceutical company with a novel immunotherapy platform that produces specifically targeted, high-potency, fully-human immunoglobulins (hIgG), also known as fully-human polyclonal antibodies, without the need for human donors, today reported financial results for the second quarter ended June 30, 2023, and provided a company update.
  • "We are pleased to share the significant progress achieved during the second quarter of 2023.
  • On August 21, 2023, we announced that EisnerAmper LLP was appointed as our independent registered public accounting firm for the fiscal year ending December 31, 2023, effective August 22, 2023.
  • The decrease was primarily due to discretionary cost reduction measures and increased efficiencies as we continue to mature as a publicly traded company.