Patents in Life Sciences

Regenerative medicine Diagnostics Natalia Giovannini, EPFL – TTO (September 28th 2015) Teaching Project Design course for Master students organised by S Maerkl & M Lutolf

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I - CONTEXT OF REGENERATIVE MEDICINE CHALLENGES OF THE HEALTHCARE INDUSTRY

II - INTELLECTUAL PROPERTY – PATENTS

DEFINITION USEFULNESS

PATENTABILITY

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I - CONTEXT OF REGENERATIVE MEDICINE CHALLENGES OF THE HEALTHCARE INDUSTRY

II - INTELLECTUAL PROPERTY – PATENTS

DEFINITION USEFULNESS

PATENTABILITY

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Drug development, drug delivery

Medtech, microfluidics, Diagnostics, Medical imaging, Implants .. Personalised medicine Biomarkers, genomics, proteomic, epigenetics… ..

STEM CELL THERAPY

DISEASE MODELING

REGENERATIVE MEDECINE CELL BASED THERAPY

GENE THERAPY

TISSUE ENGINEERING AND BIOMATERIALS, SYNTHETIC MATERIALS

SMALL MOLECULE AND BIOLOGICS

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“Our working definition of regenerative medicine includes a broad range of products that leverage the body’s intrinsic abilities to heal itself.” “Regenerative medicine is the use of cells or entities that stimulate cells to repair or replace damaged tissues.”

“We define regenerative medicine broadly. We include all technologies that are regenerative including cells, antibodies, gene therapies, small molecules, biologics, biomaterials, etc. Our company also considers stem cells for drug screening and safety toxicology testing as regenerative medicine. Immunotherapy is not positioned within our regenerative medicine group.”

“Our team views ‘cell-based immunotherapy’as regenerative medicine with a large focus on oncology.” “Regenerative medicine means any therapy that will repair or restore cells and physiology leading to improved function.

“Within our venture group we don’t have a specific definition, but from our understanding it can include a range of technologies including small molecules, biomaterials, cell-based therapies and stem cells. We would also include gene therapy.”

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Drug Discovery Phase 4: Post Marketing Surveillance

Preclinical Clinical Trials

250

5 PHASE 1

PHASE 3

NUMBER OF VOLUNTEERS 20 - 100

3 – 6 Years

PHASE 2

100 - 500

NDA SUBMITTED

5, 000 - 10, 000 Compounds

IND SUBMITTED

PRE- DISCOVERY

Drug Discovery

One drug approved (FDA /EMA)

1,000 - 5,000

Years 6 6-8 – 7½ Years

average 10-15 years / 0.5 - 5 BIO 6

MEDICAL DEVICES

Average 2-6 years 7

Short comparison medtech time to market Europe/USA

USA

mix of sponsor time, FDA, insurance reimbursement status rapid EUROPE CE marking very rapid, insurance reimbursement status slower

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SHORT COMPARISON MEDTECH / PHARMA

MEDTECH time to market 2-6, more patent coverage in time Scope of protection usually smaller than pharmaceuticals,

example the stent market (2009): 24 BIO, >10’000 patents

PHARMA time to market 8-12, less patent coverage in time Potential for scope of patent protection more important (average number of patents per drug: 2-3)

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CELL THERAPIES Until 2013, the FDA had approved only one stem cell product, Hemacord, a cord blood-derived product manufactured by the New York Blood Center and used for specified indications in patients with disorders affecting the body’s blood-forming system. 2014, First stem-cell therapy recommended for approval in EU of Holoclar (New treatment for rare condition caused by burns to the eye, the first advanced therapy medicinal product (ATMP) containing stem cells, as treatment for moderate to severe limbal stem cell deficiency (LSCD) due to physical or chemical burns to the eye(s) in adults. Estimate 2012 from the Reg. Medicine Annual Report 2012-2013): 60’000 stem cell transplants annually performed (oncology, blood disorders) for USD900Mio / 160K patients 10

CELL THERAPIES APPROVALS (through out the world) 2002-2008

none

2008-2011

5 approvals

2012

7 approvals

NB only one stem cell therapy approved (2014, EMA, for Chiesi F, Holoclar) Source: Alliance for Regenerative Medicine, Annual report 2012-2013

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Healthcare industry has low advantage to invest in product development costs without a patent protection

because 12

Healthcare companies bear the high costs of obtaining approval (FDA, EMA,..) only because they can charge high prices for PATENTED drugs

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I - CONTEXT OF REGENERATIVE MEDICINE CHALLENGES OF THE HEALTHCARE INDUSTRY

II - INTELLECTUAL PROPERTY – PATENTS

DEFINITION USEFULNESS

PATENTABILITY

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WHAT IS INTELLECTUAL PROPERTY ?

Intellectual Property (IP) is the protection granted by the State for creations of the mind (in the artistic or technical fields) and for symbols or names used in commerce.

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Patents

new inventions

application, examination (use)

Copyright

creative work/ authorship

exists automatically upon creation

Trademarks

Distinctive identification of products or services

use and register

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WHAT IS A PATENT ? A patent is a set of EXCLUSIVE RIGHTS granted by a SOVEREIGN STATE to an inventor or assignee for a LIMITED PERIOD OF TIME in exchange for detailed public disclosure of an INVENTION .

The Venetian Patent Statute issued by the Senate of Venice in 1474 and one of the earliest statutory patent systems in the world 17

Assignee is protected by the priority date, as a counterpart he must disclose its invention (18 months) Assignee is deciding about the exploitation Leverage for company investment Research exemption

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Commercialisation and exploitation of licence revenues (revenue source)

Asset value (negotiation tool) Licensing or cross-licensing (exchange of value, collaborations)

Blocking / Defensive uses (dissuasion weapon) Innovative image (marketing of a product, a company)

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A PATENT IS NOT

Not a guarantee for «freedom to operate» A patent is not an authorization to use or market a product/an invention from law Not a market authorization by an authority

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10 years - external patent filing, maintenance costs (including translations) 140 120 100 80

60 40 20 0 2 COUNTRIES

7 COUNTRIES

15 COUNTRIES 21

WHAT ARE PATENTS USEFUL FOR Product income

PROFITABLE CNIES, PHARMA, BIOTECH

Public, gvnt (no/light strings attached) Private funding (possible conditions)

UNIVERSITIES

Private investor, ecosystem support, angels, VC, ..

START UPS

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WHAT ARE PATENTS USEFUL FOR Public, gvnt (no/light strings attached) Private funding (possible conditions)

UNIVERSITIES

Promote, impact on (national) economy by giving companies (as licensees) a competitive advantage In Switzerland an obligation by law: "The role of the Swiss Federal Institutes if Technology (ETHs) and the Research Institutes shall be [...] to exploit their research findings"

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WHAT CAN YOU PATENT ?

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Invent create, generate a new and useful ideawhich may be realised, can be a process, machine, apparatus, device, substance, system, assembly of hardware…) Discover find, explain, demonstrate, make something known which already existed but which was not known before an invention is potentially patentable ! a discovery is not patentable ! 25

«AN INVENTION THAT PROVIDES A NEW

TECHNICAL SOLUTION TO A TECHNICAL PROBLEM»

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PATENTABILITY CRITERIA An invention must be NEW never described before («prior art») INVENTIVE (Europe) or NON-OBVIOUS (USA) ENABLE Described sufficiently to enable the skilled artisan to reproduce the invention without undue burden USEFUL (US) or have an INDUSTRIAL APPLICATION (Europe) 27

PATENTABILITY CRITERIA An invention must be STATUTORY SUBJECT MATTER, NOT EXCLUDED by law (territory) Excluded subject matter is independent of novelty or inventiveness “The categories of exclusions and exceptions may, depending on one’s moral, social or other point of view, appear acceptable or unacceptable, quixotic or outdated, liberal or conservative…” (Cf Board of Appeal of the European Patent Office ). 28

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CRITERIA OF PATENTABILTY MAY VARY ACCORDING TO -

Territorial law Case law Examiner ..

DO NOT HESITATE TO CONTACT THE TTO IN CASE OF PATENTABILITY QUESTIONS, EVALUATION OF AN INVENTION

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Contact Office de transfert de technologies (TTO)

EPFL-TTO EPFL Innovation Park J CH-1015 Lausanne Suisse Tel: +41 (0) 21 693 70 23 Fax: +41 (0) 21 693 70 40

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ARE METHODS OF DIAGNOSTIC PATENTABLE ? USA - In principle yes, but in practice difficult (cf case law Prometheus versus Mayo) Europe - no, but The European Patent Convention (EPC) excludes "methods for the treatment of the human or animal body by surgery or therapy and diagnostic methods practiced on the human or animal body" from patentability. .. However, this exclusion does not extend to known compounds or compositions for use in the treatment methods.

Others - Case law (2014) Methods of medical treatment confirmed patentable in Australia but not in New Zealand 32

ARE NEW USES PATENTABLE ? USA patentable

Europe patentable for compositions / substances only New use of a known medical device not patentable in Europe

NOT TO FORGET that new use must be new non obvious/ inventive, etc) Example EP / Case law (2015) – Board of appeal / the patent EP1852136, had an independent claim to a dialysis membrane for the treatment of multiple myeloma. The membrane itself was known in the prior art, yet the use of the membrane was novel.

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THANK YOU FOR YOUR ATTENTION

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