Rare diseases

Exicure, Inc. to Present at Ladenburg Thalmann 2021 Healthcare Conference

Retrieved on: 
Tuesday, June 29, 2021
Biotechnology, Health, Genetics, Pharmaceutical, Clinical trials, Spherical nucleic acid, Rare diseases, Friedreich's ataxia, Thalmann, Clinical medicine, Branches of biology, Health

Exicure, Inc. (NASDAQ: XCUR), a pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA) technology, today announced that CEO David Giljohann will present a company update as well as participate in a series of 1x1 meetings at the Ladenburg Thalmann 2021 Healthcare Conference occurring July 13-14, 2021.

Key Points: 
  • Exicure, Inc. (NASDAQ: XCUR), a pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA) technology, today announced that CEO David Giljohann will present a company update as well as participate in a series of 1x1 meetings at the Ladenburg Thalmann 2021 Healthcare Conference occurring July 13-14, 2021.
  • Replays of the presentation will be available on Exicures website for 30 days following the presentation.
  • Exicure is in preclinical development of XCUR-FXN a lipid-nanoparticle SNAbased therapeutic candidate, for the intrathecal treatment of Friedreichs ataxia (FA).
  • Exicures therapeutic candidate cavrotolimod (AST-008) is in a Phase 1b/2 clinical trial in patients with advanced solid tumors.

US FDA grants VICO Therapeutics Orphan-Drug Designation for VO659, an Investigational Therapy for Spinocerebellar Ataxia

Retrieved on: 
Tuesday, June 29, 2021
Health, Autosomal dominant disorders, Rare diseases, Life sciences, Pharmaceuticals policy, Spinocerebellar ataxia, Orphan drug, Food and Drug Administration

In February, VICO already received orphan drug designation for VO659 in SCA from the European Commission.

Key Points: 
  • In February, VICO already received orphan drug designation for VO659 in SCA from the European Commission.
  • Our investigational RNA modulating therapy is designed to lower the mutant protein levels causing these neurodegenerative diseases."
  • "We're excited to obtain orphan-drug designation from also the FDA, following the EC grant earlier this year.
  • The designation allows VICO to qualify for a number of incentives, including seven years of market exclusivity upon regulatory approval; exemption from FDA application fees for spinocerebellar ataxia; and tax credits for qualified clinical trials.

US FDA grants VICO Therapeutics Orphan-Drug Designation for VO659, an Investigational Therapy for Spinocerebellar Ataxia

Retrieved on: 
Tuesday, June 29, 2021
Health, Autosomal dominant disorders, Rare diseases, Life sciences, Pharmaceuticals policy, Spinocerebellar ataxia, Orphan drug, Food and Drug Administration

In February, VICO already received orphan drug designation for VO659 in SCA from the European Commission.

Key Points: 
  • In February, VICO already received orphan drug designation for VO659 in SCA from the European Commission.
  • Our investigational RNA modulating therapy is designed to lower the mutant protein levels causing these neurodegenerative diseases."
  • "We're excited to obtain orphan-drug designation from also the FDA, following the EC grant earlier this year.
  • The designation allows VICO to qualify for a number of incentives, including seven years of market exclusivity upon regulatory approval; exemption from FDA application fees for spinocerebellar ataxia; and tax credits for qualified clinical trials.

Cabaletta Bio Appoints Biopharmaceutical Leader Scott Brun, M.D. to Board of Directors

Retrieved on: 
Monday, June 28, 2021
Articles, Business, Rare diseases, Pemphigus vulgaris, Desmoglein-3, Pemphigus, Advisory board, Board of directors, Cabaletta, Vulgaris, Shareholder value

His expertise will be particularly valuable to Cabaletta as we advance our lead program, DSG3-CAART, in mucosal pemphigus vulgaris, said Steven Nichtberger, M.D., Chief Executive Officer and Co-founder of Cabaletta.

Key Points: 
  • His expertise will be particularly valuable to Cabaletta as we advance our lead program, DSG3-CAART, in mucosal pemphigus vulgaris, said Steven Nichtberger, M.D., Chief Executive Officer and Co-founder of Cabaletta.
  • We welcome him to the Board of Directors and look forward to his contributions to our strategic and operational objectives as we seek to increase shareholder value.
  • Earlier in his career, he held positions of increasing leadership responsibility in drug development within the R&D organization at Abbott Laboratories.
  • Dr. Brun will succeed Brian Daniels, M.D., who resigned from the Board of Directors effective June 24, 2021, and subsequently joined the Scientific Advisory Board.

Chugai’s Enspryng (Satralizumab) Approved by European Commission as First At-home Subcutaneous Treatment for Neuromyelitis Optica Spectrum Disorder (NMOSD)

Retrieved on: 
Monday, June 28, 2021
Biotechnology, Health, Pharmaceutical, Optical, Clinical trials, Monoclonal antibodies, Orphan drugs, Autoimmune diseases, Rare diseases, Organ systems, Health, Neuromyelitis optica spectrum disorder, Satralizumab, Clinical medicine, T helper 17 cell, Inebilizumab

Enspryng is the first treatment approved in the EU for adolescents from 12 years of age with NMOSD.

Key Points: 
  • Enspryng is the first treatment approved in the EU for adolescents from 12 years of age with NMOSD.
  • We are confident that Enspryng will meaningfully contribute to improving the treatment of people with NMOSD, by fitting into their day-to-day lives.
  • Enspryng is designed to prevent NMOSD relapses by inhibiting IL-6 signal signaling, which is a key driver in NMOSD.
  • Lin J, Li X, Xia J. Th17 cells in neuromyelitis optica spectrum disorder: a review.

Orphazyme presents 36-month data supporting durable response to arimoclomol during Parseghian Scientific Conference for NPC Research

Retrieved on: 
Monday, June 28, 2021
Lipid storage disorders, Rare diseases, Health, Medicine, Pharmaceutical industry, Niemann–Pick disease, type C, Food and Drug Administration, Niemann–Pick disease, NPC, New Drug Application

The data are featured in a presentation as part of the Parseghian Scientific Conference for Niemann-Pick disease type C Research.

Key Points: 
  • The data are featured in a presentation as part of the Parseghian Scientific Conference for Niemann-Pick disease type C Research.
  • The results demonstrate that arimoclomol provided a sustained benefit to study participants by reducing NPC progression as measured by the 5-domain NPC Clinical Severity Scale (5D-NPCCSS).
  • Orphazyme continue to pursue regulatory approval in Europe and evaluate a path forward for arimoclomol in NPC in the US.
  • On June 17, 2021, Orphazyme received a Complete Response Letter from the FDA regarding its New Drug Application for arimoclomol for the treatment of NPC.

Orphazyme announces restructuring to focus resources on supporting a path forward for arimoclomol in NPC

Retrieved on: 
Monday, June 28, 2021
Lipid storage disorders, Rare diseases, Health, Clinical medicine, Medicine, Niemann–Pick disease, type C, Niemann–Pick disease, Gaucher's disease, Niemann, Food and Drug Administration, Arimoclomol

I thank each of them for their strong commitment to Orphazyme and dedication to showing up for patients in need.

Key Points: 
  • I thank each of them for their strong commitment to Orphazyme and dedication to showing up for patients in need.
  • This includes pursuing regulatory approval in Europe, assessing the path forward in partnership with the FDA in the U.S., and supporting the existing global Expanded Access Program (EAP).
  • In Denmark, Orphazyme will immediately initiate negotiations under the Danish Act on Collective redundancies and the Act on Information and Consultation.
  • Arimoclomol, the companys lead candidate, is in clinical development for rare diseases including Niemann-Pick disease type C (NPC) and Gaucher disease.

BioMarin Receives Positive CHMP Opinion in Europe for Vosoritide for the Treatment of Children with Achondroplasia from Age 2 Until Growth Plates Close

Retrieved on: 
Friday, June 25, 2021
Drugs, Rare diseases, Clinical medicine, Achondroplasia, Medical specialties, Immunosuppressants, Monoclonal antibodies

Vosoritide is potentially the first medicine to be approved to treat children with achondroplasia in Europe and would be marketed under the brand name VOXZOGO (vosoritide).

Key Points: 
  • Vosoritide is potentially the first medicine to be approved to treat children with achondroplasia in Europe and would be marketed under the brand name VOXZOGO (vosoritide).
  • "The positive opinion from the CHMP represents a significant step towards making vosoritide available as a treatment choice for families.
  • The CHMP opinion reinforces the strength of the data and the clinical benefit to children as young as two years old.
  • "This positive CHMP opinion represents a significant step toward delivering on the promise of the first pharmacological treatment option for children and families affected by achondroplasia.

Major shareholder announcement

Retrieved on: 
Thursday, June 24, 2021
Branches of biology, Biology, Lipid storage disorders, Rare diseases, Protein folding, Neurological disorders, Neurodegenerative disorders, Heat shock response, Niemann–Pick disease, type C, Heat shock protein, Niemann–Pick disease, Arimoclomol

Orphazyme is a late-stage biopharmaceutical company pioneering the heat shock protein response for the treatment of rare diseases.

Key Points: 
  • Orphazyme is a late-stage biopharmaceutical company pioneering the heat shock protein response for the treatment of rare diseases.
  • The company is harnessing amplification of heat shock proteins (or HSPs) in order to develop and commercialize novel therapeutics for diseases caused by protein misfolding, protein aggregation, and lysosomal dysfunction.
  • Arimoclomol, the companys lead candidate, is in clinical development for rare diseases including Niemann-Pick disease type C (NPC) and Gaucher disease.
  • Orphazyme is headquartered in Denmark and has operations in the U.S. and Switzerland.

Fulcrum Therapeutics Announces Results from ReDUX4 Trial with Losmapimod in Facioscapulohumeral Muscular Dystrophy (FSHD) Demonstrating Slowed Disease Progression and Improved Function

Retrieved on: 
Thursday, June 24, 2021
Health, Rare diseases, Facioscapulohumeral muscular dystrophy, Muscular dystrophy, Losmapimod, Medical terminology, Medicine, Quality of life

I am enthusiastic about the potential for losmapimod to offer meaningful improvements in preserving muscle function and patient quality of life.

Key Points: 
  • I am enthusiastic about the potential for losmapimod to offer meaningful improvements in preserving muscle function and patient quality of life.
  • The ReDUX4 trial with losmapimod is the first trial to demonstrate a positive benefit in several measures of FSHD, including slower disease progression and patient reported improvement over time.
  • Disease progression results in accumulation of disability, with many patients ultimately becoming dependent upon the use of a wheelchair for daily mobility.
  • Fulcrum will present detailed results from the ReDUX4 trial during the FSHD International Research Congress today at 1:33pm ET.