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Details:
The acquisition of arimoclomol aligns perfectly with the strategy to build KemPharm’s value via the advancement and commercialization of novel treatments that address rare CNS conditions, including the lead clinical candidate, KP1077 in idiopathic hypersomnia.
Lead Product(s): Arimoclomol
Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed
Highest Development Status: Phase II/ Phase IIIProduct Type: Small molecule
Partner/Sponsor/Collaborator: KemPharm
Deal Size: Undisclosed Upfront Cash: $12.8 million
Deal Type: Acquisition May 15, 2022
Details:
Decision to withdraw the European Marketing Authorisation Application for BRX-345 (arimoclomol) for the treatment of Niemann-Pick disease Type C (NPC) ahead of a final vote and opinion by the CHMP on the MAA scheduled for later this month.
Lead Product(s): Arimoclomol
Therapeutic Area: Genetic Disease Product Name: BRX-345
Highest Development Status: Phase II/ Phase IIIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 22, 2022
Details:
Orphazyme has made progress towards resubmission of the NDA for BRX-345 (arimoclomol citrate), amplifies the production of heat shock proteins for the treatment of NPC to the FDA and plans to request a Type C Meeting in Q2 2022.
Lead Product(s): Arimoclomol
Therapeutic Area: Genetic Disease Product Name: BRX-345
Highest Development Status: Phase II/ Phase IIIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 11, 2022
Details:
Arimoclomol, is an investigational drug candidate that amplifies the production of heat shock proteins (HSPs). Arimoclomol is administered orally, and has now been studied in Phase 1, Phase 2, and pivotal Phase 2/3 trials.
Lead Product(s): Arimoclomol
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase II/ Phase IIIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 07, 2022
Details:
arimoclomol, a heat shock protein co-inducer that promotes nascent protein folding, in patients with rapidly progressive SOD1 amyotrophic lateral sclerosis (ALS) and Niemann-Pick disease type C.
Lead Product(s): Arimoclomol
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase II/ Phase IIIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 31, 2021
Details:
NPC patients receiving routine clinical care was estimated to be a mean increase of 5.2 points after three years, based on a statistical model combining placebo data from the NPC-002 double-blind study and prospective data from the observational NPC-001 study.
Lead Product(s): Arimoclomol
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase II/ Phase IIIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 28, 2021
Details:
Orphazyme announced that ORARIALS-01 pivotal trial of arimoclomol in amyotrophic lateral sclerosis (ALS) did not meet its primary and secondary endpoints to show benefit in people living with ALS. No important safety signals were reported in the trial.
Lead Product(s): Arimoclomol
Therapeutic Area: Neurology Product Name: Miplyffa
Highest Development Status: Phase IIIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 07, 2021
Details:
Orphazyme's phase 2/3 trial evaluating arimoclomol for the treatment of inclusion body myositis (IBM), a progressively debilitating muscle-wasting disease, did not meet its primary and secondary endpoints.
Lead Product(s): Arimoclomol
Therapeutic Area: Immunology Product Name: Undisclosed
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 29, 2021
Details:
Orphazyme plans to present a series of data on its investigational drug arimoclomol during the 17th Annual WORLDSymposium Scientific Meeting, to be held virtually on February 8-12, 2021.
Lead Product(s): Arimoclomol,Miglustat
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase II/ Phase IIIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 04, 2021
Details:
The long-term data demonstrate a continued positive impact on disease progression over two years. Furthermore, separate data from a post-hoc genetic sub-group analysis of patients provides more evidence of the efficacy of arimoclomol.
Lead Product(s): Arimoclomol,Miglustat
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase II/ Phase IIIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 22, 2021
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