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                            01

                            Zevra Therapeutics

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                            Lead Product(s) : Arimoclomol,Miglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II/ Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Zevra Launches MIPLYFFA™ in US for Niemann-Pick Disease Type C Treatment

                            Details : Miplyffa (arimoclomol) is a first in class oral HSF1 activator approved for Niemann-Pick disease type C in combination with miglustat.

                            Brand Name : Miplyffa

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            November 21, 2024

                            Lead Product(s) : Arimoclomol,Miglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            02

                            Zevra Therapeutics

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                            Lead Product(s) : Arimoclomol,Miglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            FDA Approves First Treatment for Niemann-Pick Disease, Type C

                            Details : Miplyffa (Arimoclomol) is indicated for use in combination with miglustat for the treatment of Niemann-Pick disease type C in adult and pediatric patients 2 years of age and older.

                            Brand Name : Miplyffa

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            September 20, 2024

                            Lead Product(s) : Arimoclomol,Miglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            03

                            Zevra Therapeutics

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                            Lead Product(s) : Arimoclomol

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II/ Phase III

                            Sponsor : Cantor

                            Deal Size : Undisclosed

                            Deal Type : Public Offering

                            DEALS_DEV arrow-down

                            Zevra Therapeutics Announces Proposed Public Offering of Common Stock

                            Details : The net proceeds from this offering will used to support the pre-commercial launch activities for BRX-345 (arimoclomol) for the treatment of Niemann-Pick Disease Type C.

                            Brand Name : BRX-345

                            Molecule Type : Small molecule

                            Upfront Cash : Undisclosed

                            August 08, 2024

                            Lead Product(s) : Arimoclomol

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Cantor

                            Deal Size : Undisclosed

                            Deal Type : Public Offering

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                            04

                            Zevra Therapeutics

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                            Zevra Therapeutics

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                            Lead Product(s) : Arimoclomol

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II/ Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            FDA Advisory Committee Supports Arimoclomol as Effective for Niemann-Pick Disease Type C

                            Details : BRX-345 (arimoclomol) is a first in class oral HSF1 activator being investigated for Niemann-Pick disease type C. The FDA has accepted the resubmitted NDA and set a PDUFA date of September 21, 2024.

                            Brand Name : BRX-345

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            August 02, 2024

                            Lead Product(s) : Arimoclomol

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            05

                            Zevra Therapeutics

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                            Zevra Therapeutics

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                            Lead Product(s) : Arimoclomol

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II/ Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Zevra Announces FDA Advisory Committee Meeting for Arimoclomol in Niemann-Pick Disease

                            Details : BRX-345 (arimoclomol) is a first in class oral HSF1 activator being investigated for Niemann-Pick disease type C. The FDA has accepted the resubmitted NDA and set a PDUFA date of September 21, 2024.

                            Brand Name : BRX-345

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            July 09, 2024

                            Lead Product(s) : Arimoclomol

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            06

                            Zevra Therapeutics

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                            Zevra Therapeutics

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                            Lead Product(s) : Serdexmethylphenidate

                            Therapeutic Area : Sleep

                            Study Phase : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Zevra Announces Positive Phase 2 Results of KP1077 for Hypersomnia at SLEEP 2024

                            Details : KP1077 (serdexmethylphenidate) is a prodrug of d-methylphenidate, which acts as dopamine & norepinephrine transporter antagonists. It is being evaluated for the treatment of idiopathic hypersomnia.

                            Brand Name : KP1077

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            June 03, 2024

                            Lead Product(s) : Serdexmethylphenidate

                            Therapeutic Area : Sleep

                            Highest Development Status : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            07

                            Zevra Therapeutics

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                            Zevra Therapeutics

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                            Lead Product(s) : Sodium Phenylbutyrate

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved

                            Sponsor : Perceptive Advisors

                            Deal Size : Undisclosed

                            Deal Type : Financing

                            DEALS_DEV arrow-down

                            Zevra Refinances Debt with Up to $100M in Committed Capital Under New Credit Facility

                            Details : The net proceeds will be used for launching Olpruva, a new formulation of sodium phenylbutyrate approved as an oral suspension by the FDA, to treat patients with urea cycle disorders.

                            Brand Name : Olpruva

                            Molecule Type : Small molecule

                            Upfront Cash : Undisclosed

                            April 10, 2024

                            Lead Product(s) : Sodium Phenylbutyrate

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved

                            Sponsor : Perceptive Advisors

                            Deal Size : Undisclosed

                            Deal Type : Financing

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                            08

                            Zevra Therapeutics

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                            Lead Product(s) : Serdexmethylphenidate

                            Therapeutic Area : Sleep

                            Study Phase : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Zevra Announces Top-Line Data From Phase 2 Trial Of KP1077 For Idiopathic Hypersomnia

                            Details : KP1077 (serdexmethylphenidate) is a prodrug of d-methylphenidate, which acts as dopamine & norepinephrine transporter antagonists. It is being evaluated for the treatment of idiopathic hypersomnia.

                            Brand Name : KP1077

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            March 26, 2024

                            Lead Product(s) : Serdexmethylphenidate

                            Therapeutic Area : Sleep

                            Highest Development Status : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            09

                            Zevra Therapeutics

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                            Lead Product(s) : Arimoclomol

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II/ Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Zevra Therapeutics Provides FDA Update on PDUFA Action Date for Arimoclomol Treatment

                            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.

                            Brand Name : BRX-345

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            March 04, 2024

                            Lead Product(s) : Arimoclomol

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            10

                            Zevra Therapeutics

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                            Lead Product(s) : Arimoclomol

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II/ Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Zevra Therapeutics Receives FDA Acceptance of NDA Resubmission for Arimoclomol Treatment

                            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.

                            Brand Name : Undisclosed

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            January 08, 2024

                            Lead Product(s) : Arimoclomol

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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