Company profile: PTC Therapeutics
1.1 - Company Overview
Company description
- Provider of biopharmaceutical therapies. Products include EMFLAZA for Duchenne muscular dystrophy; TRANSLARNA for Duchenne muscular dystrophy with a nonsense mutation; EVRYSDI for spinal muscular atrophy; TEGSEDI for hereditary transthyretin amyloidosis polyneuropathy; WAYLIVRA for familial chylomicronemia syndrome; and UPSTAZA gene therapy for aromatic L-amino acid decarboxylase deficiency.
Products and services
- TRANSLARNA (ataluren): Mutation-specific therapy approved in EU and Brazil for ambulatory Duchenne muscular dystrophy in patients aged 2+, targeting nonsense mutations in the dystrophin gene
- UPSTAZA (eladocagene exuparvovec): Gene therapy approved in the EU, Great Britain, and Israel for aromatic L-amino acid decarboxylase deficiency in patients 18 months and older
- EMFLAZA (deflazacort): Deflazacort-based medication approved in the US to treat Duchenne muscular dystrophy in patients aged 2 years and older
Key contacts
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Financial details
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1.2 - Competitors and similar companies to PTC Therapeutics
Audentes Therapeutics
HQ: United States
Website
- Description: Provider of gene therapy research and development for rare muscle diseases, serving as the Gene Therapy Center of Excellence with divisions in technical operations, medical and development, and future commercialization of gene therapy programs.
- 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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Rallybio
HQ: United States
Website
- Description: Provider of biotechnological R&D services developing investigational therapies, including RLYB212 to prevent HPA-1a alloimmunization in expectant mothers at higher risk of FNAIT; RLYB116, a self-delivered C5-targeting antibody mimetic; RLYB114, a C5-targeted Affibody for ophthalmic diseases; RLYB331, a monoclonal antibody targeting MTP-2 for iron overload; and an ENPP1 inhibitor program for hypophosphatasia.
- 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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Lumos Pharma
HQ: United States
Website
- Description: Provider of biopharmaceutical programs developing a novel treatment for Creatine Transporter Deficiency and LUM-201 for Pediatric Growth Hormone Deficiency, an oral small molecule that stimulates growth hormone secretion from the pituitary; supporting the OraGrowtH210 Phase 2 and OraGrowtH212 PK/PD studies, a Predictive Enrichment Markers strategy to identify responders, and an Expanded Access/Compassionate Use program.
- 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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Harmony Biosciences
HQ: United States
Website
- Description: Provider of novel therapies for rare neurological disorders, including U.S. commercialization of pitolisant, a histamine-3 receptor antagonist/inverse agonist approved for narcolepsy; developing HBS-102, a melanin-concentrating hormone receptor 1 antagonist; and evaluating pitolisant for idiopathic hypersomnia, Prader-Willi syndrome, and myotonic dystrophy.
- Rationale: This competitor operates in a highly similar market segment with overlapping features.
- Company type: Private company
- Employees: ●●●●●
- Total funding raised: $●●●m
- Backers: ●●●●●●●●●●
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Lumena Pharma
HQ: United States
Website
- Description: Provider of oral therapeutics for rare liver diseases, with clinical-stage candidates that selectively target a transporter in the intestine to improve liver function and relieve symptoms. Offers GMP-compliant nutraceutical manufacturing (capsules, tablets, powders, coatings), laboratory/testing and consulting, private label solutions, quality assurance, and plans distribution of generic pharmaceutical brands.
- Rationale: This competitor operates in a highly similar market segment with overlapping features.
- Company type: Private company
- Employees: ●●●●●
- Total funding raised: $●●●m
- Backers: ●●●●●●●●●●
- Acquisitions: ●●
View full Lumena Pharma 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 PTC Therapeutics
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3. Investors and private equity firms
3.1 - Buyout funds investing in similar companies to PTC Therapeutics
2.2 - Growth funds investing in similar companies to PTC Therapeutics
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4 - Top valuation comps for PTC Therapeutics
4.2 - Public trading comparable groups for PTC Therapeutics
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