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II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Jaguar Health \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Jaguar Health \/ Inapplicable"},{"orgOrder":0,"company":"Azafaros","sponsor":"Jeito Capital","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"NETHERLANDS","productType":"Other Small Molecule","year":"2025","type":"Series B Financing","leadProduct":"Nizubaglustat","moa":"GBA2 protein","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Azafaros","amount2":0.14999999999999999,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.14999999999999999,"dosageForm":"Oral Capsule","sponsorNew":"Azafaros \/ Jeito Capital","highestDevelopmentStatusID":"8","companyTruncated":"Azafaros \/ Jeito Capital"},{"orgOrder":0,"company":"NovelMed Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Antibody","year":"2025","type":"Inapplicable","leadProduct":"Ruxoprubart","moa":"CFB","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"NovelMed Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"NovelMed Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"NovelMed Therapeutics \/ Inapplicable"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            NovelMed Therapeutics

                            U.S.A arrow
                            IFT FIRST
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                            NovelMed Therapeutics

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                            Lead Product(s) : Ruxoprubart

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            NovelMed’s Ruxoprubart Shows Best-in-Class Results for PNH in Phase II Trial

                            Details : NM8074 (ruxoprubart), a human immunoglobulin, exhibits dual specificity, it blocks only the Alternative Pathway, dysregulated by the disease, and it binds Bb which has no binding to Factor B.

                            Product Name : NM8074

                            Product Type : Antibody

                            Upfront Cash : Inapplicable

                            May 19, 2025

                            Lead Product(s) : Ruxoprubart

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Azafaros

                            Netherlands arrow
                            IFT FIRST
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                            Azafaros

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                            Not Confirmed
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                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : Jeito Capital

                            Deal Size : $147.0 million

                            Deal Type : Series B Financing

                            DEALS_DEV arrow-down

                            Azafaros Raises €132M Series B for Phase 3 Lysosomal Disorder Programs

                            Details : The financing aims to advance the clinical development of company's mid-stage product AZ-3102 (nizubaglustat), which is being evaluated for Niemann-Pick disease.

                            Product Name : AZ-3102

                            Product Type : Other Small Molecule

                            Upfront Cash : Undisclosed

                            May 13, 2025

                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : Jeito Capital

                            Deal Size : $147.0 million

                            Deal Type : Series B Financing

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                            03

                            Jaguar Health

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                            IFT FIRST
                            Not Confirmed

                            Jaguar Health

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                            Lead Product(s) : Crofelemer

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Crofelemer Shows TPN Reduction in MVID and SBS-IF Patients by 27% and 12.5%

                            Details : Mytesi (crofelemer) is an inhibitor of both the CFTR Clˉ and CaCC, currently being investigated in pediatric patients with microvillus inclusion disease and short bowel syndrome.

                            Product Name : Mytesi

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            April 30, 2025

                            Lead Product(s) : Crofelemer

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            NS Pharma

                            U.S.A arrow
                            IFT FIRST
                            Not Confirmed

                            NS Pharma

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                            IFT FIRST
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                            Lead Product(s) : NS-229

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            FDA Grants Orphan Drug Status to NS-229 for Treating EGPA

                            Details : NS-229 is being investigated as a selective Janus kinase 1 (JAK1) inhibitor to help regulate immune cell function and prevent the immune system from causing tissue damage.

                            Product Name : NS-229

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            April 21, 2025

                            Lead Product(s) : NS-229

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            MoonLake Immunotherapeutics

                            Switzerland arrow
                            IFT FIRST
                            Not Confirmed

                            MoonLake Immunotherapeutics

                            Switzerland arrow
                            IFT FIRST
                            Not Confirmed
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                            Lead Product(s) : Sonelokimab

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : Hercules Capital

                            Deal Size : $500.0 million

                            Deal Type : Financing

                            DEALS_DEV arrow-down

                            MoonLake Secures up to $500 Million in Non-Dilutive Financing from Hercules

                            Details : Proceeds from the financing will be used to rapidly advance sonelokimab, a novel investigational nanobody, for the treatment of palmoplantar pustulosis (PPP).

                            Product Name : M1095

                            Product Type : Antibody

                            Upfront Cash : Undisclosed

                            April 03, 2025

                            Lead Product(s) : Sonelokimab

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : Hercules Capital

                            Deal Size : $500.0 million

                            Deal Type : Financing

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                            06

                            Omeros

                            U.S.A arrow
                            IFT FIRST
                            Not Confirmed

                            Omeros

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                            IFT FIRST
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                            Lead Product(s) : Zaltenibart

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Omeros Updates Ongoing Zaltenibart Phase 3 PNH Clinical Trial Program

                            Details : OMS906 (zaltenibart) is a MASP-3 inhibitor, antibody candidate, which is currently being evaluated for the treatment of paroxysmal nocturnal hemoglobinuria.

                            Product Name : OMS906

                            Product Type : Antibody

                            Upfront Cash : Inapplicable

                            March 21, 2025

                            Lead Product(s) : Zaltenibart

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Corvus Pharmaceuticals

                            U.S.A arrow
                            IFT FIRST
                            Not Confirmed

                            Corvus Pharmaceuticals

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                            IFT FIRST
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                            Lead Product(s) : Soquelitinib

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Corvus Starts Phase 2 Soquelitinib Trial in Autoimmune Lymphoproliferative Syndrome

                            Details : CPI-818 (soquelitinib) is an investigational small molecule drug given orally designed to selectively inhibit ITK, which is being evaluated for treatment of autoimmune lymphoproliferative syndrome.

                            Product Name : CPI-818

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            March 12, 2025

                            Lead Product(s) : Soquelitinib

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Jaguar Health

                            U.S.A arrow
                            IFT FIRST
                            Not Confirmed

                            Jaguar Health

                            U.S.A arrow
                            IFT FIRST
                            Not Confirmed
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                            Lead Product(s) : Crofelemer

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : Napo Therapeutics

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Jaguar Doses First Patient in IIT Evaluating Crofelemer for Microvillus Inclusion Disease

                            Details : Mytesi (crofelemer) is a plant-based drug extracted & purified from the red bark sap. It is being developed for MVID in pediatric patients.

                            Product Name : Mytesi

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            January 22, 2025

                            Lead Product(s) : Crofelemer

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : Napo Therapeutics

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Arctic Therapeutics

                            Iceland arrow
                            IFT FIRST
                            Not Confirmed

                            Arctic Therapeutics

                            Iceland arrow
                            IFT FIRST
                            Not Confirmed
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                            Lead Product(s) : Grapiprant

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : EIC fund

                            Deal Size : $27.7 million

                            Deal Type : Series A Financing

                            DEALS_DEV arrow-down

                            Arctic Therapeutics Announces €26.5M Oversubscribed Series A Financing

                            Details : The financing aims to fund the development of AT-001 an oral treatment designed to prevent the aggregation of harmful amyloid proteins in the brain. It is being evaluated for treating HCCAA.

                            Product Name : AT-001

                            Product Type : Other Small Molecule

                            Upfront Cash : Undisclosed

                            January 22, 2025

                            Lead Product(s) : Grapiprant

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : EIC fund

                            Deal Size : $27.7 million

                            Deal Type : Series A Financing

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                            10

                            Bio-Thera Solutions

                            China arrow
                            IFT FIRST
                            Not Confirmed

                            Bio-Thera Solutions

                            China arrow
                            IFT FIRST
                            Not Confirmed
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                            Lead Product(s) : BAT4406F

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Bio-Thera Solutions Publishes Phase I Results for BAT4406F in Neuromyelitis Optica

                            Details : BAT4406F is an ADCC-enhanced fully humanized anti-CD20 monoclonal antibody in Chinese patients with neuromyelitis optica spectrum disorders (NMOSD).

                            Product Name : BAT4406F

                            Product Type : Antibody

                            Upfront Cash : Inapplicable

                            January 14, 2025

                            Lead Product(s) : BAT4406F

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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