ESPE

Lumos Pharma Announces New Data and Analysis of 15 Subjects from OraGrowtH212 Trial Presented at the 2023 ESPE Annual Meeting

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Tuesday, September 26, 2023
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“The presented results add to the growing body of evidence supporting the physiologic mechanism of action for LUM-201, as well as its potential as a treatment for moderate PGHD.

Key Points: 
  • “The presented results add to the growing body of evidence supporting the physiologic mechanism of action for LUM-201, as well as its potential as a treatment for moderate PGHD.
  • The slides presented at ESPE are available on the Posters & Publications page under the Investors & Media section of Lumos Pharma’s website.
  • At baseline, subjects (10M:5F) were (mean ± SD) aged 7.9±1.4 years, with IGF-1 SDS -0.82±0.9, and peak GH 7.2±2.2 ng/mL (clonidine stimulation), consistent with moderate PGHD.
  • Deconvolution analysis was performed on serum GH measured every 10 minutes (0800 h to 2000 h).

Ascendis Pharma Presents Results from Long-Term enliGHten Trial of TransCon™ hGH in Pediatric Growth Hormone Deficiency

Retrieved on: 
Saturday, September 23, 2023
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COPENHAGEN, Denmark, Sept. 23, 2023 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced results from enliGHten, the Company’s open-label extension trial evaluating the long-term safety and efficacy of TransCon hGH as a once-weekly treatment for children and adolescents with growth hormone deficiency (GHD). Results were shared today in Poster LB-17 at ESPE 2023, the annual meeting of the European Society for Paediatric Endocrinology being held in The Hague, September 21-23.

Key Points: 
  • COPENHAGEN, Denmark, Sept. 23, 2023 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced results from enliGHten, the Company’s open-label extension trial evaluating the long-term safety and efficacy of TransCon hGH as a once-weekly treatment for children and adolescents with growth hormone deficiency (GHD).
  • The enliGHten trial enrolled 298 participants (mean age 10.3 years) from the Phase 3 heiGHt Trial of treatment-naïve pediatric GHD patients and the Phase 3 fliGHt Trial of pediatric GHD patients switching from daily somatropin treatment.
  • Of these treatment completers, 59% met or exceeded their average parental height SDS, with mean TransCon hGH treatment duration of 3.2 years.
  • “Additionally, these results demonstrate that long-term treatment goals can be safely reached with TransCon hGH administered once weekly.”
    TransCon hGH was generally safe and well-tolerated.

Rhythm Pharmaceuticals Presents New Data at ESPE 2023

Retrieved on: 
Friday, September 22, 2023
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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.”

BioMarin to Present Data Showing Long-Term Benefit of VOXZOGO® (vosoritide) on Growth in Children with Achondroplasia at 2023 European Society for Paediatric Endocrinology (ESPE) Meeting

Retrieved on: 
Thursday, September 21, 2023
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SAN RAFAEL, Calif., Sept. 21, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, today announced that new data on VOXZOGO® (vosoritide) in children with achondroplasia will be presented at the 61st Annual European Society for Paediatric Endocrinology (ESPE) Meeting in The Hague, Netherlands.

Key Points: 
  • In an open-label, long-term Phase 2 extension study, VOXZOGO demonstrated consistent and durable growth benefits in young children with achondroplasia when initiated before the age of 5.
  • Both groups of treated children demonstrated substantial restoration of height when compared to untreated children with achondroplasia.
  • VOXZOGO is currently approved in Europe in children with achondroplasia who are 2 years of age and older with open growth plates.
  • VOXZOGO is also currently approved in the United States in children with achondroplasia who are 5 years of age and older with open growth plates.

Neurocrine Biosciences Presents Post Hoc Data Analysis in Congenital Adrenal Hyperplasia at ESPE 2023

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Thursday, September 21, 2023
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SAN DIEGO, Sept. 21, 2023 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX), today announced that it will present a new post hoc analysis of Phase 2 data of the investigational drug crinecerfont in adolescent patients with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency (21-OHD).

Key Points: 
  • SAN DIEGO, Sept. 21, 2023 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX), today announced that it will present a new post hoc analysis of Phase 2 data of the investigational drug crinecerfont in adolescent patients with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency (21-OHD).
  • These new data will be presented at the 61st Annual European Society for Pediatric Endocrinology ( ESPE ) Meeting in The Hague, Netherlands from Sept. 21–23, 2023.
  • Post hoc analysis of these data released today assessed whether baseline hormone concentration and glucocorticoid (GC) doses correlated with response to treatment.
  • We plan to announce top-line data from our Phase 3 CAHtalyst™ Pediatric Study in early Q4 2023."

Lumos Pharma Reports Second Quarter 2023 Financial Results, Provides Clinical Update

Retrieved on: 
Wednesday, August 9, 2023
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Encouraging Interim Data from OraGrowtH Trials Presented at ENDO 2023, Highlighted in KOL Webinar

Key Points: 
  • Encouraging Interim Data from OraGrowtH Trials Presented at ENDO 2023, Highlighted in KOL Webinar
    AUSTIN, Texas, Aug. 09, 2023 (GLOBE NEWSWIRE) -- Lumos Pharma, Inc .
  • (NASDAQ:LUMO), a biopharmaceutical company advancing an oral therapeutic candidate for Pediatric Growth Hormone Deficiency (PGHD) through Phase 2 clinical trials, today announced financial results for the second quarter ended June 30, 2023.
  • Cash on hand as of June 30, 2023 is expected to support operations for at least 12 months following the date of the filing of our second quarter 2023 financial statements.
  • ET today to discuss its financial results and to give an update on clinical programs.

KronosMD Inc’s CEO Files Provisional Patent Application for an Advanced Ultrasound Dental Digital Impression System

Retrieved on: 
Thursday, March 23, 2023
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KronosMD believes this technology is the first of its kind and will improve the digital workflow of clear orthodontic aligner manufacturing.

Key Points: 
  • KronosMD believes this technology is the first of its kind and will improve the digital workflow of clear orthodontic aligner manufacturing.
  • The filing of this provisional patent application directly relates to KronosMD's intellectual property development goals to manufacture and market an advanced and innovative dental ultrasound imaging product line.
  • If approved, KronosMD believes this patent could significantly enhance manufacturing processes and workflows for the multi-billion clear orthodontic aligners market.
  • The issued patents and the provisional patents are in two main fields, remote at-home digital virtual imaging devices, and office-based dental ultrasound imaging platforms and sensors, covering KronosMD disruptive 3D medical ultrasound products and software.

Rezolute Reports First Quarter Fiscal 2023 Results and Highlights Company Progress

Retrieved on: 
Wednesday, November 9, 2022
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REDWOOD CITY, Calif., Nov. 09, 2022 (GLOBE NEWSWIRE) -- Rezolute, Inc. (Nasdaq: RZLT), a clinical-stage biopharmaceutical company dedicated to developing transformative therapies with the potential to shift the treatment paradigms of devastating metabolic diseases, today announced its financial results for the first quarter of fiscal 2023 ended September 30, 2022.

Key Points: 
  • Research and development (R&D) expenses were $7.7 million for the first quarter of fiscal 2023, compared to $5.8 million for the same period in fiscal 2022.
  • General and administrative (G&A) expenses were $2.5 million for the first quarter of fiscal 2023, compared to $1.9 million for the same period in fiscal 2022.
  • Net loss was $9.8 million for the first quarter of fiscal 2023, compared to $7.8 million for the same period in fiscal 2022.
  • Rezolute is also developing RZ402, an orally available plasma kallikrein inhibitor, for the treatment of diabetic macular edema.

Rhythm Pharmaceuticals Presents New Disease Burden Findings and Analyses of Setmelanotide Data in Children and Adolescents with Rare MC4R Pathway Diseases at ESPE 2022

Retrieved on: 
Monday, September 19, 2022
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BOSTON, Sept. 19, 2022 (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, announced the presentation of new findings on the burden of hyperphagia and obesity on patients with Bardet-Biedl syndrome (BBS) and their caregivers during the 60th Annual Meeting of the European Society for Paediatric Endocrinology (ESPE 2022) held in Rome on Sept. 15 to 17.

Key Points: 
  • Rhythm and its collaborators delivered one poster and three oral presentations during ESPE 2022, including data from new analyses that showed setmelanotide achieved substantial weight loss benefit in adolescent and pediatric patients with rare MC4R pathway diseases across three separate pivotal trials.
  • CARE-BBS participants at the time of the survey were residents of the United States (n=60), Canada (n=62), United Kingdom (n=59) or Germany (n=61).
  • In addition, the findings illustrate how the negative impact of hyperphagia on caregivers affects family dynamics, emotional well-being, and caregiver employment.
  • Rhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases.

Zealand Pharma Presents Data from Phase 3 Trial of Dasiglucagon in Congenital Hyperinsulinism at the 60th Annual ESPE Meeting

Retrieved on: 
Monday, September 19, 2022
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Though congenital hyperinsulinism is an incredibly challenging disease to manage, the efficacy and safety of dasiglucagon observed in this Phase 3 trial, support its potential as a new treatment option for children with CHI.

Key Points: 
  • Though congenital hyperinsulinism is an incredibly challenging disease to manage, the efficacy and safety of dasiglucagon observed in this Phase 3 trial, support its potential as a new treatment option for children with CHI.
  • We are very pleased to present the compelling results from the Phase 3 study of dasiglucagon in newborns and infants up to 12 months of age at ESPE 2022.
  • This study deepens our understanding of dasiglucagons potential as an innovative treatment for CHI patients, said David Kendall, M.D., Chief Medical Officer of Zealand Pharma.
  • Zealand Pharma A/S (Nasdaq: ZEAL) ("Zealand") is a biotechnology company focused on the discovery and development of peptide-based medicines.