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Details:
BRX-345 (arimoclomol) is a first in class investigational HSF1 activator, which is being evaluated in phase 2/3 clinical trials for the treatment of Niemann-Pick disease type C.
Lead Product(s): Arimoclomol
Therapeutic Area: Rare Diseases and Disorders 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 04, 2024
Details:
Under the agreement, XOMA has acquired the royalty and milestone rights associated with two assets from LadRx Corporation, BRX-345 (arimoclomol), an oral therapeutic for Niemann-Pick disease type C, and aldoxorubicin, an albumin-linked formulation of doxorubicin.
Lead Product(s): Arimoclomol
Therapeutic Area: Rare Diseases and Disorders Product Name: BRX-345
Highest Development Status: Phase II/ Phase IIIProduct Type: Small molecule
Partner/Sponsor/Collaborator: XOMA
Deal Size: $58.6 million Upfront Cash: $5.0 million
Deal Type: Agreement January 11, 2024
Details:
Arimoclomol is a first in class investigational HSF1 activator, which is under phase 2/3 clinical development for the treatment of Niemann-Pick disease type C.
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: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 08, 2024
Details:
Arimoclomol is a first in class investigational HSF1 activator, which is under phase 2/3 clinical development for the treatment of Niemann-Pick disease type C.
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: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable December 27, 2023
Details:
Arimoclomol is believed to function by stimulating a normal cellular protein repair pathway through the activation of molecular chaperones. It is being developed as a treatment for Niemann-Pick disease type C (NPC).
Lead Product(s): Arimoclomol
Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed
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 February 23, 2023
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:
CytRx’s drug candidate, arimoclomol, was sold to Orphazyme A/S in exchange for milestone payments and royalties. Orphazyme is developing arimoclomol in two indications, including Niemann-Pick disease Type C (NPC), and Gaucher disease.
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 14, 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:
At 12-months, a significant treatment effect in favor of arimoclomol of −1.40 points was observed, corresponding to a 65% relative reduction in annual disease progression.
Lead Product(s): Arimoclomol
Therapeutic Area: Genetic Disease Product Name: Miplyffa
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 August 24, 2021
Details:
Orphazyme stated arimoclomol demonstrated a consistent safety profile throughout the 36-month treatment period. Arimoclomol is an investigational drug candidate that amplifies the production of heat-shock proteins (HSPs).
Lead Product(s): Arimoclomol
Therapeutic Area: Genetic Disease Product Name: Miplyffa
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 29, 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:
CytRx has received a Complete Response Letter from the U.S. Food and Drug Administration following its review of the new drug application for arimoclomol, a heat shock protein amplifier intended for the treatment of Niemann-Pick disease type C.
Lead Product(s): Arimoclomol
Therapeutic Area: Genetic Disease Product Name: Miplyffa
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 18, 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
Details:
U.S. Food and Drug Administration (FDA) has extended the review period of the New Drug Application (NDA) for arimoclomol for the treatment of Niemann-Pick Disease Type C (NPC) by a standard extension period of three months.
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 December 27, 2020
Details:
There are currently no approved products for NPC in the US and arimoclomol has the potential to make a significant difference to patients with this devastating disease.
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 23, 2020
Details:
CytRx disclosed receipt of a filing communication from the U.S. Food and Drug Administration (“FDA”) relating to the agency’s ordinary course review of its new drug application for arimoclomol in the treatment of Niemann-Pick disease Type C.
Lead Product(s): Arimoclomol
Therapeutic Area: Genetic Disease Product Name: Miplyffa
Highest Development Status: Phase IIIProduct Type: Small molecule
Partner/Sponsor/Collaborator: CytRx Corporation
Deal Size: $83.7 million Upfront Cash: Undisclosed
Deal Type: Public Offering September 30, 2020
Details:
If approved, arimoclomol would become the first approved therapy in the U.S. for people with Niemann-Pick disease Type C (NPC).
Lead Product(s): Arimoclomol
Therapeutic Area: Genetic Disease Product Name: Undisclosed
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 September 16, 2020
Details:
Orphazyme has completed the rolling submission of its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for its investigational therapy arimoclomol for the treatment of Niemann-Pick disease Type C (NPC).
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 July 20, 2020
Details:
Orphazyme’s drug candidate arimoclomol has potential across a range of neurodegenerative diseases, including GCase-deficient Parkinson’s disease (PD).
Lead Product(s): Arimoclomol
Therapeutic Area: Neurology Product Name: Undisclosed
Highest Development Status: PreclinicalProduct Type: Small molecule
Partner/Sponsor/Collaborator: Michael J. Fox Foundation
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Collaboration July 17, 2020
Details:
Arimoclomol demonstrated a marked and clinically meaningful dose-dependent reduction in liver and spleen size. Data supports Orphazyme's intention to proceed with pivotal stage clinical development in Gaucher disease .
Lead Product(s): Arimoclomol
Therapeutic Area: Genetic Disease 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 June 29, 2020
Details:
The data show a dose-dependent effect of arimoclomol on certain disease-relevant clinical secondary endpoints, such as liver and spleen size.
Lead Product(s): Arimoclomol
Therapeutic Area: Genetic Disease 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 June 24, 2020
Details:
Orphazyme has initiated the submission of its New Drug Application (NDA) for a rolling review by the US Food and Drug Administration (FDA) for arimoclomol, based on the promising results reported from clinical trials. Trial conducted in collaboration with CytRx Corporation.
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 01, 2020
Details:
Orphazyme has received Fast Track Designation from the US Food and Drug Administration (FDA) for the development of arimoclomol for the treatment of Amyotrophic Lateral Sclerosis (ALS).
Lead Product(s): Arimoclomol
Therapeutic Area: Neurology Product Name: Undisclosed
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 22, 2020
Details:
Orphazyme A/S provided updated clinical and regulatory guidance on arimoclomol milestones in its four indications: ALS, sIBM, Niemann-Pick disease Type C and Gaucher Disease.
Lead Product(s): Arimoclomol
Therapeutic Area: Neurology Product Name: Undisclosed
Highest Development Status: Phase IIIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Orphazyme
Deal Size: $120.0 million Upfront Cash: Undisclosed
Deal Type: Licensing Agreement February 04, 2020
Details:
Orphazyme expects to receive approval for NPC in H1 2021 in the US through the fast track approval process, with the aim to bring the product to patients shortly thereafter.
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 January 03, 2020
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