Clinical and Commercial Stage Assets for Rare Indications

A mid-cap publicly traded US Pharma company specializing in rare diseases with a highly efficient global commercial team is seeking assets for a wide range of rare disease indications that have reached clinical development. Our client’s pipeline consists of 3 products approved in several indications and 2 products in Phase 1-3. They are interested in expanding their portfolio with clinical-stage and commercial assets. 

Approaches of Interest: 

• Open to a wide range of therapeutic areas. A non-exhaustive list with some examples of diseases of interest can be found here
• Indications should have a patient population of 300 – 200,000 in the US
• Open to a wide range of modalities, including small molecules, recombinant proteins, antibodies, and antisense oligonucleotides. Non-gene-therapy nucleic acid-based therapeutics are of particular interest 
• Repurposed drugs will be considered only if they have been reformulated into a new product for the rare indication 

Out of Scope: 

• Cell therapy, gene therapy, and medical devices 
• Assets for oncology indications and psychiatric disorders 
• Assets that are under clinical development or marketed for non-rare indications 
• Generics, biosimilars, and natural products that are not IP-protected 
• Diagnostics and drug discovery platforms 

Developmental Stages of Interest: 

• Opportunities from Phase 1 onwards (including commercial) are in scope 
• Opportunities that are Phase 2 onward are of highest interest 

Submission Information 

Submission of one-page, 200–300-word briefs is encouraged, along with any optional supplementary information e.g. slide decks, relevant publications. Submissions that contain information on the therapeutic area, mechanism of action, modality, route of administration, patient population, and development stage will be prioritized. In submitting to this campaign, you confirm that your submission contains only non-confidential information. 

Opportunity for Collaboration 

Our client is open to a range of collaboration opportunities, with the main focus on licensing of assets.