The interesting part was chief business officer Gerry mentioned the interested party offer. How much was the offer?
here’s the list of acquisitions for phase 3 stage indications in rare diseases areas:
Acquisition Deals with Potential Market Size for Each Indication
• Biogen acquired Nightstar Therapeutics (2019) for $800 million
• Drug: NSR-REP1
• Indication: Choroideremia (ultra-rare genetic retinal disease)
• Potential Market Size: $1 billion to $5 billion
• Choroideremia affects approximately 1 in 50,000 to 100,000 people, translating to 79,000 to 158,000 individuals globally. With gene therapy treatments potentially costing $300,000 to $2 million per patient, the market could reach billions, depending on diagnosis rates and accessibility.
• Ipsen acquired Clementia Pharmaceuticals (2019) for $1.3 billion
• Drug: Palovarotene
• Indication: Fibrodysplasia ossificans progressiva (FOP, ultra-rare musculoskeletal disorder)
• Potential Market Size: $361.2 million in 2023, projected to reach $503.8 million by 2034
• FOP has a prevalence of about 1 in 2 million, equating to roughly 3,950 patients worldwide. According to an IMARC report, the market was valued at $361.2 million in 2023 and is expected to grow at a compound annual growth rate (CAGR) of 3.07% through 2034.
• Sanofi acquired Principia Biopharma (2020) for $3.7 billion
• Drug: Rilzabrutinib
• Indication: Pemphigus (rare autoimmune skin disorder)
• Potential Market Size: $1 billion to $2 billion
• Pemphigus affects approximately 0.5 to 3.2 per 100,000 people, resulting in around 146,150 cases globally. Treatments such as biologics, costing $20,000 to $50,000 per year, suggest a market size in the $1 billion to $2 billion range.
• UCB acquired Ra
Pharmaceuticals (2019) for $2.5 billion
• Drug: Zilucoplan
• Indication: Myasthenia gravis (rare neuromuscular disorder)
• Potential Market Size: $3 billion to $5 billion
• Myasthenia gravis has a prevalence of about 14 to 20 per 100,000, with an estimated 1.58 million cases worldwide. High-cost treatments like eculizumab (over $500,000 annually) and others averaging $50,000 to $100,000 per year contribute to a market size of several billion dollars.
• Pfizer acquired Biohaven Pharmaceuticals (2022) for $11.6 billion
• Drug: Troriluzole
• Indication: Spinocerebellar ataxia (rare neurodegenerative disorder)
• Potential Market Size: $5 billion to $10 billion
• Spinocerebellar ataxia affects 1 to 5 per 100,000 people, or approximately 237,000 cases globally. If a therapy is developed with pricing similar to other rare disease treatments ($100,000 to $300,000 per year), the market could range from $5 billion to $10 billion.
• Pfizer acquired Global Blood Therapeutics (2022) for $5.4 billion
• Drug: Inclacumab
• Indication: Sickle cell disease (rare genetic blood disorder)
• Potential Market Size: $1 billion to $2 billion (current), with potential for growth
• Sickle cell disease affects about 100,000 people in the U.S. and millions globally, especially in sub-Saharan Africa. Current treatments generate $1 billion to $2 billion annually, with significant growth potential as gene therapies and other novel treatments emerge.
• Merck acquired Acceleron Pharma (2021) for $11.5 billion
• Drug: Sotatercept
• Indication: Pulmonary arterial hypertension (PAH, rare cardiovascular disorder)
• Potential Market Size: $5.8 billion in 2020, projected to reach $7.3 billion by 2026
• PAH has a prevalence of 15 to 50 per million, with approximately 256,750 cases worldwide. The global PAH market was valued at $5.8 billion in 2020 and is forecasted to grow to $7.3 billion by 2026, driven by new therapies.
• Novo Nordisk acquired Forma Therapeutics (2022) for $1.1 billion
• Drug: Etavopivat
• Indication: Sickle cell disease
• Potential Market Size: $1 billion to $2 billion (current), with potential for growth
• Similar to the Pfizer acquisition, the sickle cell disease market currently stands at $1 billion to $2 billion, with room for expansion as new therapies targeting different mechanisms are introduced.
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