Propionic acidemia

Burjeel Holdings to Launch Rare Disease Research & Development Project ‘NADER’ in Partnership with US-based BridgeBio Pharma

Retrieved on: 
Wednesday, July 12, 2023

ABU DHABI, United Arab Emirates and PALO ALTO, Calif., July 12, 2023 (GLOBE NEWSWIRE) -- Burjeel Holdings, one of the largest healthcare providers in the MENA region, and BridgeBio Pharma, Inc., a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, have announced a new project to revolutionize the field of early diagnosis and treatment of rare diseases or disorders in the UAE and the region. The two entities signed a preliminary, non-binding Collaboration Agreement establishing a mutual intention to work together on project ‘NADER’ (Needs Assessment and Therapeutics Development for Rare Diseases – ‘nader’ meaning ‘rare’ in Arabic). Genetic in origin, these often life-threatening or chronically debilitating diseases affect a small percentage of the population and are present throughout the person's entire life, even if symptoms do not immediately appear. The partnership will launch operations in Abu Dhabi to conduct clinical trials and research, leveraging the Emirate’s advanced infrastructure for innovation and life science.

Key Points: 
  • The partnership will launch operations in Abu Dhabi to conduct clinical trials and research, leveraging the Emirate’s advanced infrastructure for innovation and life science.
  • Project ‘NADER’ aims to revolutionize the field of early diagnosis and treatment in order to potentially improve patient outcomes.
  • Dr. Khaled Musallam, Group Chief Research Officer of Burjeel Holdings and project lead said, “Many rare diseases are highly clustered in our region due to our unique genetic pool.
  • As the collaboration unfolds, both Burjeel Holdings and BridgeBio anticipate significant progress in the field of rare diseases in the region.

Ultragenyx Announces Upcoming Data Presentations at American Society of Gene & Cell Therapy (ASGCT) 2023 Annual Meeting

Retrieved on: 
Monday, May 8, 2023

The company will present additional data supporting its gene therapy portfolio and discuss critical topics during the clinical trials spotlight symposium and at pre-meeting workshops.

Key Points: 
  • The company will present additional data supporting its gene therapy portfolio and discuss critical topics during the clinical trials spotlight symposium and at pre-meeting workshops.
  • 1424)
    Poster presentation: Development of UX055 AAV Gene Therapy for Cyclin-dependent Kinase-like 5 (CDKL5) Deficiency Disorder (CDD), a Rare Neurological Disorder (Board No.
  • 1340)
    Poster presentation: Rare Disease Patient Advocacy Perspectives on the Promise and Challenges of Gene Therapy (Board No.
  • 1578)
    In addition to the data presentations, Ultragenyx will present at one pre-meeting workshop on Tuesday, May 16:

VectivBio Reports Full Year 2022 Financial Results and Provides Business Update

Retrieved on: 
Wednesday, April 19, 2023

ET

Key Points: 
  • ET
    BASEL, Switzerland, April 19, 2023 (GLOBE NEWSWIRE) -- VectivBio Holding AG (“VectivBio”) (Nasdaq: VECT), a clinical-stage biopharmaceutical company pioneering novel transformational treatments for severe rare conditions, today reported financial results and provided a business update for the full year ended December 31, 2022.
  • “2022 was a very good year for VectivBio as we made significant progress against our R&D and corporate objectives, despite the challenging biotech environment.
  • In November 2022, the Company announced the completion of enrollment of the Colon-in-Continuity (CIC) stratum of the Phase 3 STARS study.
  • The Company will discuss these results on a call today, April 19, 2023, at 8:00 a.m.

VectivBio Announces Results of Extraordinary General Meeting

Retrieved on: 
Friday, December 9, 2022

BASEL, Switzerland, Dec. 09, 2022 (GLOBE NEWSWIRE) -- VectivBio Holding AG (“VectivBio”) (Nasdaq: VECT), a clinical-stage biopharmaceutical company pioneering novel transformational treatments for severe rare conditions, announced today that Wouter Joustra was elected as a new member of the Board of Directors at the Extraordinary General Meeting held on December 9, 2022.

Key Points: 
  • BASEL, Switzerland, Dec. 09, 2022 (GLOBE NEWSWIRE) -- VectivBio Holding AG (“VectivBio”) (Nasdaq: VECT), a clinical-stage biopharmaceutical company pioneering novel transformational treatments for severe rare conditions, announced today that Wouter Joustra was elected as a new member of the Board of Directors at the Extraordinary General Meeting held on December 9, 2022.
  • Mr. Wouter Joustra brings a breadth of capital markets and investment experience in the life sciences investment industry to VectivBio.
  • He joined Forbion, a leading European life sciences venture capital firm, in October 2019, where he is currently General Partner.
  • His deep experience in life-science capital markets will benefit VectivBio as we enter our next stage of growth,” said Tom Woiwode, VectivBio Board Chairman.

VectivBio Publishes Invitation to the Extraordinary General Meeting of Shareholders

Retrieved on: 
Wednesday, November 16, 2022

The sole agenda item of the Extraordinary General Meeting will be the election of Wouter Joustra as a new member of the Board of Directors.

Key Points: 
  • The sole agenda item of the Extraordinary General Meeting will be the election of Wouter Joustra as a new member of the Board of Directors.
  • Shareholders shall be represented at the Extraordinary General Meeting exclusively by the independent proxy.
  • For information on how to exercise rights and issue voting instructions to the independent proxy, shareholders can access the invitation to the Extraordinary General Meeting at www.edocumentview.com/VECT.
  • These statements include, but are not limited to, statements concerning the timing of the Extraordinary General Meeting.

VectivBio to Present at the 2022 Jefferies London Healthcare Conference

Retrieved on: 
Wednesday, November 9, 2022

BASEL, Switzerland, Nov. 09, 2022 (GLOBE NEWSWIRE) -- VectivBio Holding AG (VectivBio) (Nasdaq: VECT), a clinical-stage biopharmaceutical company pioneering novel transformational treatments for severe rare conditions, today announced that Luca Santarelli, M.D., Ph.D., Founder and Chief Executive Officer of VectivBio, will be presenting at the Jefferies London Healthcare Conference on Thursday, November 17, 2022.

Key Points: 
  • BASEL, Switzerland, Nov. 09, 2022 (GLOBE NEWSWIRE) -- VectivBio Holding AG (VectivBio) (Nasdaq: VECT), a clinical-stage biopharmaceutical company pioneering novel transformational treatments for severe rare conditions, today announced that Luca Santarelli, M.D., Ph.D., Founder and Chief Executive Officer of VectivBio, will be presenting at the Jefferies London Healthcare Conference on Thursday, November 17, 2022.
  • VectivBio is a global clinical-stage biotechnology company focused on transforming and improving the lives of patients with severe rare conditions.
  • VectivBio is also advancing its modular, small molecule CoMET platform to address a broad range of previously undruggable Inherited Metabolic Diseases (IMDs).
  • Candidates from the CoMET platform are initially being evaluated in methylmalonic acidemia (MMA), propionic acidemia (PA), and other organic acidemias.

VectivBio Announces Completion of Enrollment for Colon-In-Continuity (CIC) Cohort of STARS Pivotal Phase 3 Study Investigating Apraglutide in Short Bowel Syndrome with Intestinal Failure (SBS-IF)

Retrieved on: 
Thursday, November 3, 2022

BASEL, Switzerland, Nov. 03, 2022 (GLOBE NEWSWIRE) -- VectivBio Holding AG (“VectivBio”) (Nasdaq: VECT), a clinical-stage biopharmaceutical company pioneering novel transformational treatments for severe rare conditions, today announced the completion of enrollment of the Colon-in-Continuity (CIC) cohort for the company’s Phase 3 STARS (STudy of ApRglutide in SBS) pivotal study investigating the long-acting synthetic GLP-2 agonist apraglutide in Short Bowel Syndrome with Intestinal Failure (SBS-IF).

Key Points: 
  • This result is a testament to a successful operational effort based on our strong partnership with patients and investigators involved in this study.
  • STARS is a pivotal, global Phase 3 study enrolling approximately 144 patients with SBS-IF stratified 50/50 for CIC and stoma, with patients being evaluated over 48 weeks and 24 weeks respectively.
  • The study includes an improved PS weaning algorithm tailored to remnant bowel anatomy to enhance signal detection.
  • STARS is designed to establish a new standard of care for all patients with SBS-IF and to tailor apraglutide treatment to patients with distinct remnant bowel anatomy.

VectivBio Announces Closing of $125 Million Underwritten Offering of Ordinary Shares

Retrieved on: 
Tuesday, October 18, 2022

BASEL, Switzerland, Oct. 18, 2022 (GLOBE NEWSWIRE) -- VectivBio Holding AG (VectivBio) (Nasdaq: VECT), a clinical-stage biopharmaceutical company pioneering novel transformational treatments for severe rare conditions, today announced the closing of its previously announced underwritten offering of 16,700,000 ordinary shares at an offering price of $7.50 per share.

Key Points: 
  • BASEL, Switzerland, Oct. 18, 2022 (GLOBE NEWSWIRE) -- VectivBio Holding AG (VectivBio) (Nasdaq: VECT), a clinical-stage biopharmaceutical company pioneering novel transformational treatments for severe rare conditions, today announced the closing of its previously announced underwritten offering of 16,700,000 ordinary shares at an offering price of $7.50 per share.
  • Before deducting the underwriting discounts and commissions and offering expenses, VectivBio received total gross proceeds of approximately $125 million.
  • Jefferies, SVB Securities and Piper Sandler acted as the joint book-running managers for the offering.
  • VectivBio is a global clinical-stage biotechnology company focused on transforming and improving the lives of patients with severe rare conditions.

VectivBio Announces Pricing of $125 Million Underwritten Offering of Ordinary Shares

Retrieved on: 
Thursday, October 13, 2022

BASEL, Switzerland, Oct. 13, 2022 (GLOBE NEWSWIRE) -- VectivBio Holding AG (VectivBio) (Nasdaq: VECT), a clinical-stage biopharmaceutical company pioneering novel transformational treatments for severe rare conditions, today announced the pricing of an underwritten offering of 16,700,000 ordinary shares at an offering price of $7.50 per share.

Key Points: 
  • BASEL, Switzerland, Oct. 13, 2022 (GLOBE NEWSWIRE) -- VectivBio Holding AG (VectivBio) (Nasdaq: VECT), a clinical-stage biopharmaceutical company pioneering novel transformational treatments for severe rare conditions, today announced the pricing of an underwritten offering of 16,700,000 ordinary shares at an offering price of $7.50 per share.
  • Before deducting the underwriting discounts and commissions and offering expenses, VectivBio expects to receive total gross proceeds of approximately $125 million.
  • The offering is expected to close on or about October 17, 2022, subject to satisfaction of customary closing conditions.
  • These statements include, but are not limited to, whether VectivBio will offer the ordinary shares or consummate the offering, and the timing, size and use of proceeds of the offering described herein.

VectivBio Reports Positive Interim Clinical Data from STARS Nutrition, a Phase 2 Study Investigating Apraglutide in Short Bowel Syndrome with Intestinal Failure Patients (SBS-IF) with Colon-In-Continuity (CIC)

Retrieved on: 
Thursday, October 13, 2022

BASEL, Switzerland, Oct. 13, 2022 (GLOBE NEWSWIRE) -- VectivBio Holding AG (“VectivBio”) (Nasdaq: VECT), a clinical-stage biopharmaceutical company pioneering novel transformational treatments for severe rare conditions, today announced positive interim data from the company’s ongoing Phase 2 STARS Nutrition study evaluating the safety, pharmacokinetics and efficacy of apraglutide, an investigational new drug that is a next-generation, long-acting synthetic GLP-2 agonist, in adult patients with Short Bowel Syndrome with Intestinal Failure (SBS-IF) and Colon-in-Continuity (CIC). The STARS Nutrition clinical program is the first-ever study prospectively evaluating the clinical benefit of a GLP-2 agonist specifically in a CIC patient population. Patients with CIC anatomy represent over half of the total SBS-IF patient population and are underserved by current treatment options.

Key Points: 
  • The STARS Nutrition clinical program is the first-ever study prospectively evaluating the clinical benefit of a GLP-2 agonist specifically in a CIC patient population.
  • Patients with CIC anatomy represent over half of the total SBS-IF patient population and are underserved by current treatment options.
  • Interim data showed that six-month treatment with weekly apraglutide resulted in an average 50% reduction in PS volume and a 47% reduction in parenteral energy content.
  • In the nine patients who reached at least three months of treatment, the average PS reduction was 31% after three months of treatment.