Company profile: Minoryx Therapeutics
1.1 - Company Overview
Company description
- Provider of pharmacological chaperone small-molecule drugs for genetic, neurometabolic, and rare pediatric diseases. Develops leriglitazone, a selective PPAR gamma agonist for X-linked adrenoleukodystrophy and other CNS diseases, supported by phase 2/3 results. Programs include ADVANCE (adult X-ALD), NEXUS (pediatric cALD), CALYX (adult progressive cALD), and a Friedreich's ataxia proof-of-concept.
Products and services
- Leriglitazone: A selective PPARγ agonist small molecule targeting X-linked adrenoleukodystrophy and other CNS diseases, demonstrating significant clinical benefit in phase 2/3 studies
- ADVANCE study: A phase 2/3 adult X-ALD clinical study of leriglitazone that showed significant reduction in cerebral lesion progression and improvements in myelopathy symptoms
- NEXUS study: A pediatric-focused cALD assessment of leriglitazone reporting clinical stability and cerebral lesion growth stabilization after 24 weeks of treatment
Key contacts
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Financial details
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1.2 - Competitors and similar companies to Minoryx Therapeutics
Clementia Pharmaceuticals
HQ: Canada
Website
- Description: Provider of clinical-stage, disease-modifying treatments for ultra-rare bone disorders and other debilitating diseases with high unmet medical need, including development of its lead product candidate, palovarotene, an oral small molecule.
- Rationale: This competitor operates in a highly similar market segment with overlapping features.
- Company type: Private company
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- Total funding raised: $●●●m
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Imara
HQ: United States
Website
- Description: Provider of novel therapeutics for people living with sickle cell disease and hemoglobinopathies.
- Rationale: This competitor operates in a highly similar market segment with overlapping features.
- Company type: Private company
- Employees: ●●●●●
- Total funding raised: $●●●m
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Cogent Biosciences
HQ: United States
Website
- Description: Provider of therapies for solid cancer tumors as a biotechnology company.
- Rationale: This competitor operates in a highly similar market segment with overlapping features.
- Company type: Private company
- Employees: ●●●●●
- Total funding raised: $●●●m
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Orphazyme
HQ: Denmark
Website
- Description: Provider of biopharmaceutical R&D for novel therapeutics targeting lysosomal storage diseases and other genetic disorders; conducts clinical trials, offers early access programs for investigational drugs, and delivers safety reporting/pharmacovigilance. Developed arimoclomol for neurodegenerative diseases, including Niemann Pick disease type C (sold to KemPharm Denmark A/S).
- Rationale: This competitor operates in a highly similar market segment with overlapping features.
- Company type: Private company
- Employees: ●●●●●
- Total funding raised: $●●●m
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XORTX Therapeutics
HQ: Canada
Website
- Description: Provider of biopharmaceutical therapies for progressive kidney disease: XRx-008 for ADPKD to reduce cyst growth and improve kidney function; XRx-101 to lower uric acid for acute kidney injury and issues tied to serious viral infections, including COVID-19; and XRx-225, a pre-clinical treatment for Type 2 Diabetic Nephropathy targeting chronic kidney failure and end-stage kidney disease.
- Rationale: This competitor operates in a highly similar market segment with overlapping features.
- Company type: Private company
- Employees: ●●●●●
- Total funding raised: $●●●m
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View full XORTX Therapeutics company profile →
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2.M&A buyers
2.1 Potential strategic acquirers in the sector
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2.2 - Strategic buyer groups for Minoryx Therapeutics
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3. Investors and private equity firms
3.1 - Buyout funds investing in similar companies to Minoryx Therapeutics
2.2 - Growth funds investing in similar companies to Minoryx Therapeutics
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4 - Top valuation comps for Minoryx Therapeutics
4.2 - Public trading comparable groups for Minoryx Therapeutics
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