The interesting part was chief business officer Gerry mentioned...

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.