2. GRANTSEEKERS & GRANTMAKERS
Academic teams
Scientists
Start-ups
Later-stage biotechs
need funding
Patients associations
Foundations
Charities
provide support, and
funding
3. GRANTSEEKERS: INDUSTRY
Financing a rare or ultra-rare disease drug project is a harsh task. Despite incentives
provided by FDA and EMA orphan drugs regulations (as well as other countries
regulatory bodies), developing an orphan drug still requires significant funding.
Most frequently, financing comes from founders, friends and family, business
angels, venture capital, corporate venture, and tax-payer money (“public funding”).
Almost everybody in the orphan drug industry has dealt with the above-mentioned
stakeholders. With more or less success. Financing an orphan disorder project is not
easy at all: it is like looking for a needle in a haystack.
Despite success stories, the financing industry is still not comfortable with orphan
drugs (there are exceptions): poor understanding of diseases, niche markets,
attrition rates similar to non-orphan, increasing payers’ pressure on pricing, etc.
Furthermore, their money is highly-dilutive.
4. GRANTSEEKERS: ACADEMY
In light of the scarcity of public funding, research laboratories and clinicians are
increasingly looking for alternative sources of funding to finance their projects.
Industry has been a source for years, but, when the projects focus on orphan diseases,
only a few established or well-funded early-stage companies partner, provided they have
an interest.
Since a couple of years, patients organizations and dedicated foundations provided
funding to academic- and industry-led early-stage orphan diseases projects, everywhere
in the world.
This is a growing trend, and we believe that, in the case of orphan diseases, these
organizations are the best partners for academic projects and investigators-led clinical
trials, as they are primarily seeking a therapeutic return, instead of a financial return.
5. GRANTMAKERS: PATIENTS ORGANIZATIONS
Among the different missions of a patients association, contributing to the financing
of research and development of therapeutics or diagnostics addressing the disease
is of utmost importance. In fact, academic and industry R&D teams often lack the
financial resources that are needed to progress a project to an available treatment.
Diminishing public research budgets, poor understanding of the rare diseases
ecosystem, and frequent negative risk assessment by venture capital lead to a
funding bottleneck, particularly at the preclinical development stage.
For decades now, small and large patients associations filled the gap, mainly
through grants to academic teams. More recently, patients associations started to
fund start-ups and late-stage biotech companies. Large associations process grants
through yearly requests for proposal. Midsize and small associations examine
industry grants request on a case by case basis.
Recent actuality proved that grants and equity investments made by patients
associations can trigger either the targeted “therapeutic return”- a therapy -, and/or
a significant financial return (eg. Kalydeco in Cystic Fibrosis).
6. FUNDING MODALITIES
Patients organizations fund academic teams, investigators, biotechs and industry with:
Non-refundable grants
Refundable grants
Royalty-bearing grants
Convertible loans
Equity
7. HOW WE WORK
Our experience in industry, our understanding of the orphan drugs ecosystem and our
connections with patients organizations, academic teams and biotech companies are our
most valuable assets. We may assist start-ups and established companies, as well as
patients associations.
In a nutshell, we:
evaluate orphan drugs projects suitability to patients organizations funding
review projects’ presentation material and customize it if necessary
identify relevant patients associations (we have an international proprietary database of
several thousand organizations)
establish preliminary contacts, and validate their level of interest and financing capacity
introduce our customer to interested organizations
assist our customers in their presentations
advise on collaboration and financing terms
help our clients in negotiations and closing