BBS

Rhythm Pharmaceuticals Announces Commercial Launch of IMCIVREE® (setmelanotide) in Germany for the Treatment of Obesity and Control of Hunger in Bardet-Biedl Syndrome

Retrieved on: 
Monday, April 24, 2023

The German Federal Joint Committee (G-BA) ruled that IMCIVREE is eligible for reimbursement by Statutory Health Insurances for BBS.

Key Points: 
  • The German Federal Joint Committee (G-BA) ruled that IMCIVREE is eligible for reimbursement by Statutory Health Insurances for BBS.
  • People living with BBS may experience insatiable hunger, also known as hyperphagia, and severe obesity beginning early in life.
  • In 2022, Rhythm was granted marketing authorization for IMCIVREE by the EC for BBS and early-access authorization for setmelanotide in France.
  • Rhythm expects to launch IMCIVREE for the treatment of patients with BBS in the Netherlands later this year and in Italy, Spain, and the UK in 2024.

HKSTP and AstraZeneca signing MOU on Strategic Collaboration

Retrieved on: 
Tuesday, April 4, 2023

Hong Kong Science and Technology Parks (HKSTP) continues to strengthen the strategic partnership with AstraZeneca, one of the world's leading pharmaceutical companies.

Key Points: 
  • Hong Kong Science and Technology Parks (HKSTP) continues to strengthen the strategic partnership with AstraZeneca, one of the world's leading pharmaceutical companies.
  • Since 2021, AstraZeneca and HKSTP have been collaborating, and this partnership will not only continue but also be upgraded.
  • The extended collaboration involves AstraZeneca and HKSTP supporting and providing services to startup companies under the HKSTP co-incubation programme.
  • Dr Sunny Chai, Chairman of HKSTP, expressed his delight in witnessing the collaboration between HKSTP and AstraZeneca in building a healthcare innovation ecosystem in Hong Kong.

Rhythm Pharmaceuticals Announces Publication of Bardet-Biedl Syndrome Patient and Caregiver Perspectives of Hunger and Quality of Life with Setmelanotide

Retrieved on: 
Monday, March 27, 2023

BOSTON, March 27, 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 the publication of interview-based patient and caregiver reported experiences of hunger and quality of life with setmelanotide treatment in Bardet-Biedl syndrome (BBS). The research is published in the peer-reviewed journal Advances in Therapy.

Key Points: 
  • A total of 19 interviews were conducted with patients (n=8) and caregivers (n=11) to explore patient experience and caregiver observations of hyperphagia before and during setmelanotide treatment.
  • Before setmelanotide treatment, most patients (n=7; 87.5%) and caregivers (n=10; 90.9%) experienced negative effects directly related to hyperphagia.
  • “Until now, the impact of hyperphagia on the lives of these patients and their families has not been fully understood.
  • These data can be incorporated into cost-utility models conducted to assess the value of treatments for rare MC4R pathway diseases.

Complete Genomics Drops Genome Sequencing Price to Sub $100 at AGBT General Meeting

Retrieved on: 
Tuesday, February 7, 2023

HOLLYWOOD, Fla., Feb. 7, 2023 /PRNewswire/ -- Complete Genomics Inc., a global life science tools leader and subsidiary of MGI Tech Co., Ltd. ("MGI"), today launched new revolutionary products and prices at Advances in Genome Biology and Technology (AGBT) General Meeting.

Key Points: 
  • Complete Genomics offers T Series, G Series, and E Series genetic sequencers covering wide range from low- medium- high- ultra high throughput sequencing applications.
  • During the conference, Complete Genomics also announced a stunning new US reagent promotional price of $1.5/Gb for its flagship sequencer DNBSEQ-T7 that is already on the market.
  • Complete Genomics, an innovative sequencing systems company, is now offering six sequencers to US sequencing labs including portable sequencer DNBSEQ-E25*, and high throughput sequencer DNBSEQ-T10×4, and medium throughput benchtop sequencer DNBSEQ-G400*.
  • Complete Genomics also provides sample and library prep automation and downstream bioinformatics solutions.

Rhythm Pharmaceuticals Announces Publication of Analysis of Quality of Life Improvements from Phase 3 Clinical Trial in Bardet-Biedl Syndrome

Retrieved on: 
Monday, January 23, 2023

BOSTON, Jan. 23, 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 that patients with Bardet-Biedl syndrome (BBS) and obesity who participated in the company’s global Phase 3 clinical trial of setmelanotide reported clinically meaningful improvements across multiple health-related quality of life (HRQOL) measures based on an analysis published in the Orphanet Journal of Rare Diseases.

Key Points: 
  • “Living with hyperphagia and severe obesity associated with BBS presents a clinically significant burden on patients and families,” said corresponding author Prof. Andrea M. Haqq, M.D., Department of Pediatrics, Faculty of Medicine & Dentistry, University of Alberta.
  • For the 52-week analysis, 9 patients younger than 18 years, or their caregiver, reported a significant and clinically meaningful change in their quality of life with a mean change from baseline of +11.2 points on the Pediatric Quality of Life Inventory (PedsQL) measure.
  • In addition, 11 adult patients who completed the Impact of Weight on Quality of Life Questionnaire-Lite (IWQOL-Lite) achieved a significant and clinically meaningful improvement in their weight related quality of life with a mean change from baseline of +12.0 points.
  • IWQOL-Lite score was significantly correlated with changes in percent body weight (P=0.0037) and body mass index (P=0.0098).

OcuSciences Announces Publication of Study Showing Novel Flavoprotein Fluorescence Measure Is Increased in Inherited Retinal Dystrophies

Retrieved on: 
Thursday, January 19, 2023

OcuSciences, Inc., a medical device company developing retinal imagers to detect early disease by assessing retinal metabolic activity, today announced the publication of a rare inherited mitochondrial diseases study in Ophthalmology Genetics journal .

Key Points: 
  • OcuSciences, Inc., a medical device company developing retinal imagers to detect early disease by assessing retinal metabolic activity, today announced the publication of a rare inherited mitochondrial diseases study in Ophthalmology Genetics journal .
  • These findings suggest that the novel FPF measure can differentiate between patients diagnosed with inherited retinal dystrophies and their age-matched controls.
  • Mean FPF intensity and heterogeneity was significantly higher in patients with inherited retinal dystrophies than age-matched controls.
  • “These findings suggest that flavoprotein fluorescence could be used as an additional tool in the assessment and diagnosis of patients with inherited retinal dystrophies,” said Dr. Traboulsi.

Rhythm Pharmaceuticals Announces New Employment Inducement Grants

Retrieved on: 
Wednesday, December 7, 2022

About Rhythm PharmaceuticalsRhythm 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. Rhythm’s precision medicine, setmelanotide, is approved by the U.S. Food and Drug Administration (FDA) for chronic weight management in adult and pediatric patients 6 years of age and older with monogenic or syndromic obesity due to pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency confirmed by genetic testing, or patients with a clinical diagnosis of Bardet-Biedl syndrome (BBS). The European Commission (EC) and Great Britain’s Medicines & Healthcare Products Regulatory Agency (MHRA) have authorized setmelanotide for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 6 years of age and above. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare genetic diseases of obesity and is leveraging the Rhythm Engine and the largest known obesity DNA database -- now with approximately 45,000 sequencing samples -- to improve the understanding, diagnosis and care of people living with severe obesity due to certain genetic deficiencies. Rhythm’s headquarters is in Boston, MA.

Key Points: 

    Rhythm Pharmaceuticals to Present at BofA Securities 2022 Virtual Biotech SMID Cap Conference

    Retrieved on: 
    Thursday, December 1, 2022

    About Rhythm PharmaceuticalsRhythm 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. Rhythm’s precision medicine, setmelanotide, is approved by the U.S. Food and Drug Administration (FDA) for chronic weight management in adult and pediatric patients 6 years of age and older with monogenic or syndromic obesity due to pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency confirmed by genetic testing, or patients with a clinical diagnosis of Bardet-Biedl syndrome (BBS). The European Commission (EC) and Great Britain’s Medicines & Healthcare Products Regulatory Agency (MHRA) have authorized setmelanotide for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 6 years of age and above. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare genetic diseases of obesity and is leveraging the Rhythm Engine and the largest known obesity DNA database -- now with approximately 45,000 sequencing samples -- to improve the understanding, diagnosis and care of people living with severe obesity due to certain genetic deficiencies. Rhythm’s headquarters is in Boston, MA.

    Key Points: 
    • A replay of the webcast will be available on the Rhythm website for 30 days following the presentation.
    • 2022 Rhythm Pharmaceuticals, Inc. All Rights Reserved.
    • Inthe United StatesandEurope, setmelanotide should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology.
    • This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995.

    Vietnam - Hong Kong New Vision . New Opportunities - Smart Cities' Leaders Forum Closes with Great Success

    Retrieved on: 
    Tuesday, November 22, 2022

    HONG KONG, Nov 22, 2022 - (ACN Newswire) - Supported by the Consulate General of Vietnam in Hong Kong, hosted by CT Group, and organized by New Media Foundation and Aliquantum Group, "Vietnam - Hong Kong: New Vision .

    Key Points: 
    • HONG KONG, Nov 22, 2022 - (ACN Newswire) - Supported by the Consulate General of Vietnam in Hong Kong, hosted by CT Group, and organized by New Media Foundation and Aliquantum Group, "Vietnam - Hong Kong: New Vision .
    • New Opportunities - Smart Cities' Leaders" forum-cum-luncheon (the "forum") was successfully held at Island Shangri-La, Hong Kong this afternoon.
    • The gathering of ministerial officials, business leaders and professionals from diverse industries at the forum discussed the booming business opportunities in and between Vietnam and Hong Kong.
    • I believe the forum has given us a guiding vision on achieving interconnection between Vietnam and Hong Kong and seeing that interconnection become yet more diverse and in-depth in the future."

    Social Enterprise Summit Gathers Leaders from Civil, Business, Government and Academic Sectors  to“Bounce Forward Together”

    Retrieved on: 
    Sunday, November 27, 2022

    HONG KONG SAR - Media OutReach - 24 November 2022 - The 15th Social Enterprise Summit (SES 2022) International Symposium kicks off today at Hong Kong Convention and Exhibition Centre.

    Key Points: 
    • HONG KONG SAR - Media OutReach - 24 November 2022 - The 15th Social Enterprise Summit (SES 2022) International Symposium kicks off today at Hong Kong Convention and Exhibition Centre.
    • Mrs Rebecca CHOY YUNG, Chair of Social Enterprise Summit Organising Committee, said: Since its inception, SES has made a significant impact in driving social innovation.
    • I am very proud to see how SES grew from a local conference to a prominent movement and platform over the last 15 years.
    • It has also engaged over 1,200 thought leaders from different sectors as speakers and developed a community of 70,000 participants around the world.