Biotechnology/Chemical Practice Committee Educational Session

AIPLA 2012 Annual MeetingOct. 25, 2012

Hypothetical Scenario• Recent Publications:– Certain cancer stem cells have dopamine receptors.– Only in CNS in healthy individuals.– In cancer patients, on blood stem cells or breast cancer

stem cells.• Invention (Dr. Gold and Post-Doc):– Proprietary stem cell assay.– Identify known psychiatric drugs (dopamine receptor

antagonists) that make breast cancer stem cells differentiate into harmless cell types.

– Want to develop compounds further and ID novel compounds based on modifying the known compounds.

Hypothetical Scenario• IP:– Dr. Gold assigns all the IP to University. Patent app filed. – University unsuccessfully tries to license to established

company (too early stage).• Early Stage Research Collaborations:– University/Dr. Gold use NIH’s National Center for Advancing

Translational Sciences (NCATS) to help develop their known compounds and test additional compounds available to be ‘repurposed’ from the NCATS.• Pharmaceutical companies have made known compounds available

to NIH for preclinical, clinical feasibility studies for new indications. – Dr. Gold develops known and new lead compounds.

• All may be eligible for orphan drug regulatory exclusivity for a rare breast cancer subtype having the dopamine receptor.

Hypothetical Scenario

• Corporate and Funding Structures:– University/Dr. Gold start up “Goldcell”– Shareholders: Univ, Gold, post-doc (also employee).– Virtual company working out of Univ space.

• Funding– $150K of seed funding (angel investment)– $100K non-conventional funding - crowd sourcing -

Jobs Act– Goldcell spends all the money on pre-clinical testing

and needs to raise more money.

Hypothetical Scenario

• Pharma-Industry Collaboration:– Collaboration with University of the Atlantic– Develop companion diagnostic to ID patients with

dopamine receptor cancer stem cell.

• Consortium:– U of A is a member of consortium of universities

working with a big pharmaceutical company.– Screens patients to identify sub population that

may be treated with a particular drug.

Hypothetical Scenario

• Venture Capital:– Competition between conventional VC firm and

Pharma Corporate VC firm to be lead investor in Goldcell.

– Competition could be resolved by a scenario where conventional VC takes lead investment position and Pharma VC is drawn in b/c of expertise in designing critical studies.