Exhibit 10.1

 

CONFIDENTIAL TREATMENT REQUESTED

 

EXECUTION VERSION

 

 

LICENSE AGREEMENT

 

 

 

By and between

 

Généthon

 

and

 

AveXis, Inc.

 

 

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TABLE OF CONTENTS

 

 

 

 

 

 

 

    

 

    

Page

BACKGROUND

 

3

ARTICLE 1.

 

DEFINITIONS AND INTERPRETATION

 

4

ARTICLE 2.

 

GRANT OF RIGHTS

 

8

ARTICLE 3.

 

SUBLICENSING

 

9

ARTICLE 4.

 

LICENSEE UNDERTAKINGS AND PERFORMANCE

 

9

ARTICLE 5.

 

FINANCIAL CONSIDERATION

 

10

ARTICLE 6.

 

INTELLECTUAL PROPERTY

 

13

ARTICLE 7.

 

CONFIDENTIALITY

 

14

ARTICLE 8.

 

RECORD KEEPING – AUDIT

 

15

ARTICLE 9.

 

REPORTS ON PROGRESS, SALES AND PAYMENTS

 

16

ARTICLE 10.

 

PROSECUTION AND MAINTENANCE

 

16

ARTICLE 11.

 

INFRINGEMENT AND ENFORCEMENT

 

17

ARTICLE 12.

 

TERM, TERMINATION AND MODIFICATION OF RIGHTS

 

18

ARTICLE 13.

 

REPRESENTATION AND WARRANTIES

 

20

ARTICLE 14.

 

INDEMNIFICATION, INSURANCE AND LIABILITY

 

21

ARTICLE 15.

 

PUBLICATION AND PUBLICITY

 

22

ARTICLE 16.

 

DISPUTE RESOLUTION

 

23

ARTICLE 17.

 

MISCELLANEOUS

 

23

Exhibit A

 

 

 

28

Exhibit B

 

 

 

29

 

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This license agreement (“Agreement”) is effective as of March 9, 2018
(“Effective Date”) made by and between AveXis, Inc., a corporation organized
under the laws of the State of Delaware, having its principal place of business
at 2275 Half Day Road, Suite 200, Bannockburn, IL 60015 (“AveXis” or “Licensee”)
and Genethon, a French non-profit organization organized under the French law of
July 1, 1901, having its principal place of business at 1 bis rue de
l’Internationale, 91002 Evry Cedex, France (“Genethon”), acting in its own name
and for its own behalf as well as in the name and on behalf of Centre National
de la Recherche Scientifique (Scientific and Technological Public Institute),
having its principal place of business at 3 rue Michel-Ange, 75794 Paris Cedex
16 France, and for business identification (SIRET) number: 180089013 04033
(“CNRS”).

 

Each of Genethon and CNRS may be referred to in this Agreement as “Licensor”.
All rights and obligations set forth in this Agreement shall be performed by
Genethon, acting in its own name and for its own behalf as well as in the name
and on behalf of CNRS.

 

Each of Licensor and Licensee may be referred to in this Agreement individually
as a “Party” or together as the “Parties.”

 

BACKGROUND

 

WHEREAS, Genethon is a leading center for the development of gene therapies for
rare diseases.

 

WHEREAS, AveXis is a clinical-stage gene therapy company focused on bringing
gene therapy out of the lab and into the clinical setting for patients and
families suffering from rare and orphan neurological genetic diseases.

 

WHEREAS, AveXis is currently developing a treatment for spinal muscular atrophy
(SMA).

 

WHEREAS, Genethon is co-owner of the Licensed Patents as attached in Exhibit A
that are necessary for the administration of such treatment.

 

WHEREAS, Licensor desires to grant rights under the Licensed Patents to Licensee
through a license to enable the commercial Development of gene therapies in the
Field.

 

WHEREAS, Licensee desires to obtain a license under the Licensed Patents, in
order to Develop gene therapies in the Field and, if such efforts are
successful, to Develop and Commercialize resulting gene therapy products.

 

 

NOW, THEREFORE, the Parties hereby agree as follows:

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ARTICLE 1.     DEFINITIONS AND INTERPRETATION

 

1.1.     Definitions

 

Unless the context otherwise requires, the terms in this Agreement, when used
with initial capital letters, shall have the meanings set forth below or at
their first use in this Agreement:

 

“AAV Vector” means the recombinant adeno-associated virus vector as defined in
the Licensed Patents.

 

“AAV9” means (a) the recombinant adeno-associated virus serotype 9 vector with
the specified sequence set forth in GenBank (protein id AAS99264.1) and (b) any
recombinant adeno-associated virus derivatives of such serotype 9 vector covered
by a Licensed Patent.

 

“Abandon” (or “Abandonment”) means, with respect to a Licensed Product and a
country in the Territory:

 

(a)

a failure from Licensee and/or its Affiliates and/or sublicensees to initiate or
conduct material Development activities or Commercial activities with respect to
particular Licensed Product in any country of the  Territory during any
consecutive twelve (12) month period;  provided, that such twelve (12) month
period shall automatically be extended if any failure to start or conduct
material Development activities is a result of any Force Majeure event or any
clinical or regulatory hold imposed by the applicable Regulatory Authority or is
otherwise outside the reasonable control of Licensee and/or its Affiliates
and/or sublicensees; or

 

(b)

an affirmative decision by Licensee and/or its Affiliates and/or sublicensees
(which shall be provided in writing to Licensor) to permanently discontinue all
Commercialization activities with respect to a particular Licensed Product in
such country; provided, that such decision shall not constitute notice of
Abandonment if any decision to discontinue Commercialization activities is a
result of any Force Majeure event or any clinical or regulatory hold imposed by
the applicable Regulatory Authority or is otherwise outside the reasonable
control of Licensee and/or its Affiliates and/or sublicensees.

 

“Abandonment Cure Period” means the hundred twenty (120) days period after the
date of the written notice of Abandon, as provided under paragraph (a) above,
from Licensor to Licensee.

 

“Affiliate” means, with respect to a Party, any person, corporation, firm, joint
venture or other entity which directly or indirectly through one or more
intermediaries, controls, is controlled by or is under common control with such
Party. As used in this definition, “control” means the possession of the
majority of the ownership, or the power to direct or cause the direction of the
management of policies, of an entity, whether through the ownership of the
outstanding voting securities thereof, by contract or otherwise.

 

“AFM-Téléthon” means the French Association against Myopathies (Association
Française contre les Myopathies) which is Licensor’s founder and principal
funder.

“Background IP” means, with respect to one Party, all Patents, Know-How and
other intellectual property rights (a) owned or possessed though a license
agreement by such Party as of Effective Date or (b) owned or possessed through a
license agreement by such Party on or after Effective Date. Background IP does
not include Licensed Patents.

 

“BLA” means a Biologics License Application, as defined in the U.S Public Health
Service Act and the regulations promulgated thereunder.

 

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“Clinical Trial” means any study of a product in human subjects.

 

“Commercialization” means activities directed to marketing, promoting,
distributing or selling a Licensed Product, including all activities directed to
obtaining pricing approval in the Territory; and excluding Development,
Manufacturing and supply of such product. “Commercialize” and “Commercialized”
shall have correlative meanings.

 

“Confidential Information” means any confidential or proprietary information
disclosed or made available in any form whatsoever by one Party (the “Disclosing
Party”) to the other Party (the “Receiving Party”), including this Agreement,
the content of the transactions contemplated herein, all technology belonging to
the Disclosing Party and any improvements thereto, any information relating to a
Party’s interests, business, finances, products, operations, sales, marketing,
customers, suppliers and suppliers’ bills of materials, trade secrets, know-how,
data, processes, methods, protocols, techniques, specifications, formulas, test
data, calibration information, presentations, analyses, studies, regulatory
communications, patent applications (as long as unpublished and/or undisclosed),
financial data, product development, assays, strategic and market research
information, other relevant marketing information, clinical data and any other
information, whether developed in connection with this Agreement or not and
whether marked as confidential or not.

 

“Development” means, with respect to a product, research and any and all
processes and activities conducted to obtain and maintain Marketing
Authorization for a product, including pre- and post-marketing approval clinical
studies and activities relating to development or preparation of such product
for Commercialization. Development includes performance of Clinical Trials.
“Develop” shall have the correlative meaning.

 

“Effective Date” shall have the meaning in the third page.

 

“Expert” has the definition set forth in Section 5.3.3.

 

“Field” means the treatment based on in vivo gene replacement therapy using an
AAV9 Vector to deliver the SMN gene addressing and restricted to Spinal Muscular
Atrophy (“SMA”) in humans.

 

“First Commercial Sale” means, with respect to a Licensed Product and a country,
the first sale for monetary value for use or consumption by the end user, within
a country of the Territory, of such Licensed Product while covered by at least
one Valid Claim of a Licensed Patent in such country after regulatory approval
for commercialization (including pricing and reimbursement if applicable) has
been obtained for such Licensed Product in such country. Treatment IND sales and
compassionate use sales shall not be construed as First Commercial Sale. First
Commercial Sale may occur directly from Licensee or through any of its
Affiliates or sublicensees.

 

“Force Majeure” has the definition set forth in Section 17.5.

 

“Invention” means any new and useful process, article of manufacture, compound,
composition of matter, formulation, or apparatus, or any improvement thereof,
discovery or finding, whether or not patentable created, generated or Developed
by or on behalf of one Party using the Licensed Patents, during the Term.
Invention does not include Background IP and Licensed Patents.

 

“Know-How” means any tangible and intangible information, data, results
(including pharmacological, research and development data and reports), and
trade secrets, rights in data, materials, discoveries,

 

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improvements, compositions of matter, cell lines, assays, sequences, processes,
methods, knowledge, protocols, formulas, utility, formulations, Inventions, and
all other scientific, pre-clinical information or data.

 

“Licensed Patents” mean the Patents that are listed in Exhibit A. For the
avoidance of doubt, the Licensed Patents’ status as stated in Exhibit A will
include any rights listed in the definition of Patents derived from the Licensed
Patents.

 

“Licensed Product(s)” means any human therapeutic product in the Field using or
having used at least one Valid Claim of the Licensed Patents whether this
product is Developed, Manufactured or Commercialized by AveXis, AveXis’
Affiliates and/or AveXis’ sublicensees.

 

“Licensee Publication” means any public announcement, public presentation,
medical communication related to the Licensed Product, where Licensee believes
it is appropriate and reasonable to do so and in all cases where Licensee
includes the names of its other licensing partners.

 

“Losses” means any liability or expense (including reasonable legal expenses,
costs of litigation and attorneys’ fees), damages, or judgments, whether for
money or equitable relief.

 

“Manufacture” means activities directed to the manufacture, receipt, incoming
inspections, storage and handling of raw materials and the manufacture,
processing, formulation, packaging, labeling, warehousing, quality control
testing (including in-process release and stability testing), supplying,
shipping and release of a product, as the case may be and to the extent
applicable, including manufacturing process development, scale-up and
validation. “Manufacturing” and “Manufactured” shall have the correlative
meaning.

 

“Marketing Authorization” means the technical, medical and scientific licenses,
registrations, authorizations and approvals (including supplements and
amendments, pre and post-approvals, pricing approvals and labeling approvals) of
any Regulatory Authority necessary for the Commercialization of a product in the
Field in such Regulatory Authority’s jurisdiction in the Territory.

 

“Marketing Authorization Application” or “MAA” means an application for
regulatory approval in order to market and/or sell a Licensed Product in any
country other than the U.S.

 

“Net Sales”: means the total gross receipts from sales or other dispositions of
Licensed Products by or on behalf of Licensee or its Affiliates or any
sublicensee to unaffiliated Third Parties for end use, less the following
deductions that are documented as attributable to the Licensed Products: (a)
costs actually incurred for transportation, including packing costs, freight out
and insurance costs; (b) rebates, trade, quantity and wholesaler and cash
discounts in amounts customary in the trade to the extent actually granted; (c)
returns, chargebacks, credits and allowances actually granted; and (d) sales,
taxes or excise duties actually paid. No deductions shall be made for
commissions paid to individuals, whether they are with independent sales
agencies or regularly employed by Licensee, or sublicensees and on its payroll,
or for the cost of collections. Sales between or among Licensee and its
Affiliates or any sublicensees shall be excluded from the computation of Net
Sales, except where such Affiliates or sublicenses are end users, but Net Sales
shall include the subsequent final sales to unaffiliated Third Parties by such
Affiliates or sublicensees. In the event consideration other than cash is paid
to Licensee, its Affiliates or any sublicensees, for purposes of determining Net
Sales, the Parties shall use the cash consideration that Licensee, its
Affiliates or any sublicensees would realize from an unrelated buyer in an arm’s
length sale of an identical item sold in the same quantity and at the time and
place of transaction, as determined jointly by the Parties based on transactions
of a similar type and standard industry practice, if any. Notwithstanding
anything to the contrary herein, the computation of Net Sales shall not include
sales of

 

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Licensed Products for early access programs, named patient programs,
compassionate use programs or similar uses.

 

“Patent” means (a) any patent, re-examination, reissue, renewal, extension,
supplementary protection certificate and term restoration, any confirmation
patent or registration patent or patent of addition based on any such patent,
(b) any pending application for patents, including provisional, converted
provisional, continuations, continuations-in-part, divisional and substitute
applications, and inventors’ certificates, (c) all foreign counterparts of any
of the foregoing, and (d) all applications claiming priority to any of the
foregoing.

 

“Pivotal Clinical Trial” means: (a) a Clinical Trial that would satisfy the
requirements for a Phase 3 study as defined in 21 CFR § 312.21(c) (or successor
regulation); or (b) any other Clinical Trial that the applicable Regulatory
Authority has agreed is sufficient to form the primary basis of an efficacy
claim in an MAA submission, including any such study that is determined to have
become pivotal after its commencement.

 

“Reasonable Efforts” means the efforts and resources normally used by a company
in the biopharmaceutical industry of similar size and resources as Licensee for
a product that is of similar market potential at a similar stage in its
development (before Market Authorization) or product life (after Market
Authorization), taking into account all relevant factors, including the
potential profitability of the Licensed Products, the costs and risks of
Developing, Manufacturing and Commercializing the Licensed Product, scientific,
safety and regulatory concerns, product profile, the competitiveness of the
marketplace and the proprietary position of the Licensed Products.

 

“Regulatory Authority” means, in a particular country or jurisdiction in the
Territory, any applicable governmental authority involved in granting (a)
approval to initiate or conduct clinical testing in humans,

(b) the authorizations, approvals, licenses, permits, consents, registrations
and filings necessary for the Commercialization of a product in a country in the
Territory including Marketing Authorizations and manufacturing licenses, or (c)
to the extent applicable in such country or jurisdiction, pricing approval for a
product in such country or jurisdiction.

 

“Term” has the definition set forth in the Section 12.1.

 

“Territory” means worldwide.

 

“Third Party” means any party other than a Party.

 

“Treatment IND” means an Investigational New Drug treatment used for Clinical
Trial before the drug has been approved.

 

“Valid Claim” means, with respect to any country, a claim of (a) an
issued/granted and unexpired patent (as may be extended through supplementary
protection certificate or patent term extension or the like); and/or (b) a
pending patent application that has not been pending for more than five (5)
years from the Effective Date, in each case of (a) and (b) to the extent such
claim has not been revoked, held invalid or unenforceable by a patent office,
court or other governmental agency of competent jurisdiction in a final and
non-appealable judgment (or judgment from which no appeal was taken within the
allowable time period) and which claim has not been disclaimed, denied or
admitted to be invalid or unenforceable through reissue, re-examination or
disclaimer or otherwise.

 

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1.2.     Interpretation

 

The captions and headings to this Agreement are for convenience only, and are to
be of no force or effect in construing or interpreting any of the provisions of
this Agreement. Unless specified to the contrary, references to Sections or
Exhibits shall refer to the particular Sections or Exhibits of or to this
Agreement and references to this Agreement include all Exhibits hereto. Unless
context otherwise clearly requires, whenever used in this Agreement:

 

a.

the words “include” or “including” shall be construed as incorporating, also,
“but not limited to” or “without limitation;”

 

b.

the word “day,” “quarter” or “year” (and derivatives thereof, e.g., “quarterly”)
shall mean a calendar day, calendar quarter or calendar year unless otherwise
specified (and “annual” or “annually” refer to a calendar year);

 

c.

the word “notice” shall mean notice in writing (whether or not specifically
stated) and shall include notices, consents, approvals and other written
communications contemplated under this Agreement;

 

d.

the word “hereof,” “herein,” “hereby” and derivative or similar word refers to
this Agreement (including any Exhibits);

 

e.

the word “or” shall have its inclusive meaning identified with the phrase
“and/or;”

 

f.

the words “will” and “shall” shall have the same obligatory meaning;

 

g.

provisions that require that a party or the parties hereunder “agree,” “consent”
or “approve” or the like shall require that such agreement, consent or approval
be specific and in writing, whether by written agreement, letter, approved
minutes or otherwise;

 

h.

words using the singular or plural number also include the plural or singular
number, respectively.

 

ARTICLE 2.       GRANT OF RIGHTS

 

2.1.     Licensor hereby grants to Licensee, subject to the terms and conditions
of this Agreement, an exclusive, sub-licensable, non-transferable (except in
accordance with Section 17.3), royalty and milestone bearing license under the
Licensed Patents to Develop, to Manufacture, to Commercialize or to import any
Licensed Products for such purpose in the Territory, solely in the Field.

 

2.2.     Nothing in this Agreement shall be interpreted as a transfer of
Licensor ownership rights in the Licensed Patents to Licensee or any of
Licensee’s Affiliates or Licensee’s sublicensees. Licensor shall remain the sole
owner of the Licensed Patents.

 

2.3.     Notwithstanding anything to the contrary in this License Agreement,
Licensor may use and permit any Third Party to use the Licensed Patents for (a)
noncommercial-research or teaching purposes in the Field limited to a
collaboration with another academic or not-for-profit research organization or
on its own; and (b) any purposes outside of the Field, particularly but not
restricted to other therapeutic indications and commercial purposes.

 

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ARTICLE 3.       SUBLICENSING

 

3.1.     Licensee may enter into non-sublicensable sublicensing agreements under
the Licensed Patents with a Third Party, subject to prior information of
Licensor of the identity of said Third Party and of the business terms of such
sublicensing agreement.

 

3.2.     Licensee agrees that any sublicenses granted by it under the Licensed
Patents shall contain terms and conditions in compliance with the terms of this
Agreement, and any obligations of this Agreement applicable to sublicensees. In
particular, each sublicense agreement shall have a provision providing that, in
the event of termination of this Agreement for any reason, Licensor and
sublicensee shall enter into good faith negotiations to conclude a direct
license under the terms of this Agreement so long as such termination did not
arise directly or indirectly as a result of such sublicensee.

 

Licensee guarantees performance and acceptance of the obligations and
commitments set forth in this Agreement and in the sublicense agreement of each
and any of its sub-licensees.

 

3.3.     Licensee agrees to forward to Licensor (and to obtain agreement of
sub-licensee to do so) a complete copy of each fully executed sublicense
agreement postmarked within thirty (30) days of the execution of the sublicense
agreement. To the extent permitted by law, Licensor agrees to maintain such
sublicense agreement in confidence.

 

ARTICLE 4.       LICENSEE UNDERTAKINGS AND PERFORMANCE

 

4.1.     Diligence. Licensee shall use its Reasonable Efforts to Develop and
Commercialize, in the European Union (including the UK), in particular in
France, and the USA, a Licensed Product in the Field.

 

4.2.     Manufacture and Commercialization of Licensed Products. Licensee shall
be responsible for the Manufacture and Commercialization of the Licensed
Products.

 

4.3.     Clinical Trials. Licensee agrees to make in France Clinical Trials on
patients suffering from Spinal Muscle Atrophy and to retain “Association
Institut De Myologie” (whose founders are the AFM-Téléthon and Genethon) as an
investigative site to the extent it is regulatory possible. The clinical trial
agreement shall be negotiated on commercially reasonable terms, directly by and
between the “Association Institut De Myologie” and Licensee, and the parties
shall execute such agreement before December 31th, 2019.

 

4.4.     Approval from Regulatory Authorities. Licensee shall have the
responsibility to prepare and file all requested documentation to obtain and
maintain approval from the Regulatory Authorities (including Marketing
Authorizations) necessary for the Development and Commercialization of the
Licensed Products in the Field and the Territory, and otherwise interact with
the relevant authorities as appropriate with respect to the Licensed Products.
This includes in particular (i) the drafting of a protocol, application for
clinical trial approval, interviewing and identifying clinical centers,
appointing a CRO or recruiting patients ; (ii) the analysis of data generated
from any trial or manufacturing; (iii) the preparation of regulatory filings for
the Licensed Product or preparing briefings for such regulatory filings; and
(iv) meetings with regulatory authorities.

 

4.5.     French  Patient  Access.  Following  the  appropriate  regulatory  approvals,  Licensee  will  use

Reasonable Efforts to make available within France all the Licensed Products
indicated for SMA at prices that would allow appropriate reimbursement scheme
and that would not constitute an obstacle for patients

 

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to have access to the therapy. Licensee shall be solely responsible for
designing and conducting all Commercialization activities necessary to fulfill
its obligations under this Section 4.5.

 

4.6.     Process Development. Upon Licensor’s request, Licensee agrees to
discuss in good faith with Licensor regarding a collaboration concerning the
Development of cells in suspension process, in or outside the Field.

 

4.7.     License with ReGenX Biosciences. Upon Licensor’s request, Licensee will
provide either to Licensor or to its Affiliates a worldwide, royalty-bearing,
non-exclusive license under the AAV9 Patents controlled by Licensee for the
SMArd indication under its current license with ReGenX Biosciences, LLC
(“ReGenX”; such agreement, the “ReGenX Agreement”). This license agreement shall
be subject to the terms of the existing license with ReGenX, including but not
limited to the royalty obligations and milestone obligations to ReGenX and the
prohibition on further sublicensing.

 

4.8.     Licensee  agree  to provide Licensor with with five hundred thousand US
dollars ($500,000) on an annual basis, for three (3) years, to support the
conduct of novel gene therapy research by Licensor in the field of SMA. Such
payment shall be made on the signing date of this Agreement and thereafter upon
each anniversary date of the Agreement. Licensor will provide annual report on
the R&D work performed. In consideration of such compensation, should the
conduct of such novel gene therapy research by Licensor in the field of SMA
results in a Patent, Licensor shall first propose to Licensee a license on such
patent, provided that Licensee is in compliance with the terms of this
Agreement. Licensee shall have a time limit of thirty (30) calendar days from
receiving the request from Licensor, giving details of the proposed license, to
notify its acceptance or refusal of the license. If Licensee declines such
license or does not respond to Licensor in due time, Licensor shall be free to
propose and conclude such license with any third party of its choice, without
any further notification to be given to Licensee.

 

ARTICLE 5.       FINANCIAL CONSIDERATION

 

In consideration for the exclusive license granted to Licensee and Licensor’s
commitments pursuant to this Agreement, Licensee shall pay Licensor the
following:

 

5.1.     License Fees

 

In consideration for the license rights granted to Licensee under this
Agreement, Licensee shall pay Licensor:

 

(i)

a one-time, non-refundable, non-creditable amount of four million US dollars
($4,000,000 USD), which shall be paid to Licensor at signing of this Agreement.

 

(ii)

a  nonrefundable, ongoing annual fee of twenty-five thousand US dollars ($25,000
USD) upon each anniversary date of the Agreement to cover Licensor’s management
fees of this Agreement.

 

5.2.     Milestone Payments

 

Licensee agrees to pay to Licensor during the Term, the one-time,
non-refundable, non-creditable Milestone Payments set forth in the table below,
upon first achievement, by Licensee alone or together with its Affiliates and/or
a sublicensees of the applicable milestones with respect to the Licensed Product
which first achieves such applicable milestone:

 

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* 1st Milestone: 1st treatment in EU Pivotal Clinical Trial

Two million US dollars ($2,000,000 USD)

* 2nd Milestone: 1st US BLA or EU MAA submission

Two million US dollars ($2,000,000 USD)

* 3rd Milestone: 1st USA BLA or EU MAA approval

Two million US dollars ($2,000,000 USD)

* 4th Milestone: annual world-wide net sales equal

five hundred million US dollars ($500,000,000 USD)

Two million US dollars ($2,000,000 USD)

* 5th Milestone: annual world-wide net sales equal

one billion US dollars ($1 000 000 000 USD)

Three million US dollars ($3,000,000 USD)

Total

Eleven million US dollars ($11,000,000 USD)

 

For purpose of the above the terms “net sales” shall have similar meaning as the
definition of “Net Sales” but shall include the sales of Licensed Products and
the sales of identical products not covered by a Valid Claim in a given country
of the Territory (hereinafter referred to as “Identical Products”).

 

5.3.     Royalties on Net Sales

 

5.3.1.  As of the  First Commercial Sale, a five percent (5%) Royalty shall be
paid on Net Sales on a country-by-country and Licensed Product-by-Licensed
Product basis. For each country and each Licensed Product, such Royalties shall
start as of the First Commercial Sale of the Licensed Product in the country and
shall end at the later of: (a) expiration of the last Valid Claim of an issued
Licensed Patent covering such Licensed Product inside the Field in in such
country or (b) ten years from First Commercial Sale of such Licensed Product in
the Country (the “Royalty Term”).

 

Notwithstanding the foregoing:

 

·

Royalties shall be reduced by 50%, on a Licensed Product-by-Licensed Product and
country- by-country basis, should a Licensed Product not be covered anymore by a
Valid Claim in the concerned country of the Territory during the Royalty Term
provided above. Such reduction shall only apply to the sales of such Licensed
Product concluded after the expiration of the last Valid Claim of an issued
Licensed Patent covering such Licensed Product inside the Field in such country.

 

·

In addition, Royalties shall not be payable in the event that a Licensed Product
is not covered by a Valid Claim in the country of its sale at the time of First
Commercial Sale of such Licensed Product in such country of the Territory, and
for so long as a Valid Claim continues not to exist.

 

·

In addition and notwithstanding the foregoing, should the Licensed Patent
No15/713,347 filed on September 22, 2017 (the US continuation) referred in the
Exhibit A as being still “pending” at the Effective Date, be refused by the US
patent office before the first commercial sale of a product using Licensor
intellectual property rights that were intended to be protected by Licensed
Patent application No15/713,347, Licensee shall pay to Licensor a fee amounting
to two and a half percent (2.5%) of the net sales of such products in the USA
for a duration of ten years from such first commercial sale of such product in
the USA. For the purpose of the above, the terms “first commercial sale” and
“net sales” shall have similar meaning as the definition of these terms when
applied to Licensed Products.

 

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5.3.2.  Royalty Monetization ROFR. In the event Licensor seeks to monetize a
portion or the entirety of its royalty obligation under this Agreement, Licensor
shall inform Licensee to allow the Licensee to make an offer. Licensor shall
give Licensee a thirty (30) days period to submit an offer prior to make a
decision with respect to such royalty monetization.

 

5.3.3.  The Parties will seek to negotiate any dispute between them about Net
Sales in good faith. Any dispute between the Parties about Net Sales in any
instance, which has not been resolved by the Parties with twenty (20) Business
Days, may, at the request of either Party, be referred to an expert. The expert
will be a single, independent chartered accountant to be agreed between the
Parties, or in default of agreement between the Parties within five (5) Business
Days, to be selected at the request of either of them by the Brussels (Belgium)
Court (“Expert”). Any dispute to be referred to the Expert will be decided upon
in a final and binding manner by the Expert acting as a technical expert and not
as an arbitrator. Any actions, decisions, awards or payments to be made or taken
pursuant to the determination of the Expert will be made or taken within thirty
(30) Business Days of notification of the same to the relevant Parties. The
costs of the Expert will be borne by the Parties as determined by the Expert.

 

5.4.     Sublicense Fees

 

5.4.1.  In addition to milestones that Licensee shall pay to Licensor as per
Section 5.2 in case of achievement of such milestones by Licensee (together with
its Affiliates and its sublicensees) and in addition to the royalties that
Licensee shall pay to the Licensor as per Section 5.3, Licensee shall pay to
Licensor a percentage of any non-royalty sublicense fee (including upfront
payments, milestone payments and profit share payments) received by Licensee
from any sublicensee following execution of a sublicense agreement for a
Licensed Patents, after deducting any non-royalty fee mentioned above from
milestone payments that Licensee owes Licensor pursuant to Section 5.2 for the
same milestone event.

 

5.4.2.  The applicable percentage due to Licensor for each sublicense shall be
fifteen percent (15%).

 

5.5.     Royalty Stacking

 

5.5.1.  If, in connection with the Manufacture, use, or Commercialization of a
Licensed Product, Licensee is obligated to pay royalties to Licensor and any
Third Parties that, in the aggregate, exceed fifteen percent (15%) of Net Sales
for any Licensed Product, then the royalty owed to Licensor for that Licensed
Product will be reduced by an amount calculated as follows:

 

R = (C*(A/(A+B)))

 

Where:

R = Reduction of Licensor royalty, A = Unreduced Licensor royalty,

B = sum (in percentage) of all Third Party royalties, and C = increment of
projected total royalty above 15%.

 

5.5.2.     Notwithstanding the foregoing, Licensee will pay to Licensor no less
than 75% of the royalties that Licensee would otherwise pay to Licensor if there
were no royalties due to Third Parties.

 

5.6.  Payment Method

 

Payments under this Agreement shall be paid in US Dollar by wire transfer, or
electronic funds transfer (EFT) of immediately available funds to:

 

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Account owner:

Généthon

Bank name & address:

BNP Paribas, 37-39 rue d’Anjou, 75008 Paris, France

Bank Code:

30004

Agency Code:

02837

Account No.:

00000006928

Key:

56

IBAN:

FR76 3000 4028 3700 0000 0692 856

BIC:

BNPAFRPPXXX

 

Any value, taxes, or other expenses incurred in the transfer shall be paid
entirely by Licensee.

 

All sums due under this Agreement are exclusive of any value added tax which
will be payable in addition by Licensee on the issuing by Licensor of an
appropriate value added tax invoice.

 

5.7.     Late payment

 

Should Licensee fail to make any royalties and other payments, including patent
expense reimbursements, required to be paid by Licensee pursuant to this
Agreement, on the due date, Licensor reserves the right, without any further
notification being given by Licensor:

 

(a)

to suspend and/or cancel any of its obligations; and/or

 

(b)

to charge interest calculated on a day by day basis, as from the due date of
payment, and equal to higher of (i) three (3) times the French legal interest
and (ii) the last European Central Bank rate plus ten (10) points; and/or

 

(c)

to terminate the Agreement by right in accordance with Section 12.2.1.

 

The interest payment shall be due from the day the original payment (or portion
thereof) was due until the day that such payment was received by Licensor. The
payment of such interest shall not restrict Licensor from exercising any other
rights it may have because any payment is overdue.

 

ARTICLE 6.     INTELLECTUAL PROPERTY

 

6.1.     Background IP

 

As between the Parties, Licensor and Licensee shall remain sole owners of their
respective Background IP. For the avoidance of doubt, this Agreement does not
confer any rights, titles or interests of a Party’s Background IP to the other
Party.

 

6.2.     Inventions

 

As between the Parties, Licensor and Licensee shall remain sole owners of their
respective Inventions. For the avoidance of doubt, this Agreement does not
confer any rights, titles or interests of a Party’s Invention to the other
Party.

 

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ARTICLE 7.     CONFIDENTIALITY

 

7.1.     Confidentiality; Exceptions

 

Except to the extent expressly authorized by this Agreement or otherwise agreed
by the Parties in writing, during the term of this Agreement and for ten (10)
years thereafter (except that with respect to Confidential Information that
constitutes a trade secret, the Recipient’s obligations under this Agreement
will continue with respect to such trade secret for as long as such information
remains a trade secret), the Parties agree that the Receiving Party shall keep
confidential and shall not publish or otherwise disclose or use for any purpose
other than as provided for in this Agreement any Confidential Information
furnished to it by the other Party pursuant to this Agreement, regardless of
whether such information is specifically designated as confidential and
regardless of whether such information is in oral, written, electronic or other
form.

 

Notwithstanding the foregoing, Confidential Information shall not be deemed to
include information or materials to the extent that it can be established by
written documentation by the Receiving Party that such information or material:

 

(a)

was already known to or possessed by the Receiving Party or any of its
Affiliates, other than under an obligation of confidentiality (except to the
extent such obligation has expired or an exception is applicable under the
relevant agreement pursuant to which such obligation is established), at the
time of disclosure;

 

(b)

was generally available to the public or otherwise part of the public domain at
the time of its first disclosure to the Receiving Party or any of its
Affiliates, except by breach of this Agreement;

 

(c)

became generally available to the public or otherwise part of the public domain
after its disclosure and other than through any act or omission of the Receiving
Party or any of its Affiliates in breach of this Agreement;

 

(d)

was independently developed by the Receiving Party or any of its Affiliates as
demonstrated by documented evidence prepared contemporaneously with such
independent development; or

 

(e)

was disclosed to the Receiving Party or any of its Affiliates, other than under
an obligation of confidentiality, by a Third Party who had no obligation to the
Disclosing Party not to disclose such information to others.

 

7.2.     Authorized Use and Disclosure

 

The Receiving Party shall receive, maintain, and hold the Confidential
Information in strict confidence; and exercise the same degree of care as it
shall exercises to safeguard its own information but in no event less than
reasonable care. Each Party may use and disclose Confidential Information of the
other Party as follows:

 

(a)

under appropriate confidentiality provisions substantially equivalent to those
in this Agreement, in connection with the performance of its obligations or
exercise of rights granted to such Party in this Agreement; however, the
Receiving Party will be responsible for any disclosure or use of Confidential
Information of the Disclosing Party made by any person to

 

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whom the Receiving Party disclosed such Confidential Information as though such
disclosure or use was conducted by the Receiving Party; and

 

(b)

to the extent such disclosure is reasonably necessary in prosecuting and
maintaining Patents (including applications therefor) in accordance with this
Agreement, prosecuting or defending litigation, complying with applicable
governmental regulations, conducting Development or Commercialization hereunder,
obtaining and maintaining Marketing Authorizations, or otherwise required by law
applicable to such Party or a Party’s disclosure under regulations promulgated
by applicable security exchanges; provided however, that if a Party is required
by applicable law or under security exchange rules it will, except where
prohibited by applicable law or impracticable, give reasonable advance notice to
other Party of such disclosure requirement and, where practicable, will use its
reasonable efforts to secure confidential treatment of such Confidential
Information required to be disclosed.

 

Either Party may disclose to bona fide potential investors, lenders and
acquirors/acquirees, and to such Party’s consultants and advisors, the existence
and terms of this Agreement to the extent necessary in connection with a
proposed equity or debt financing of such Party, or a proposed acquisition or
business combination, or to bona fide potential sublicensees, so long as such
recipients are bound in writing to maintain the confidentiality of such
information in accordance with the terms of this Agreement.

 

Notwithstanding the forgoing, Licensor may disclose any Licensee Confidential
Information to the CNRS as co-owner of the Licensed Patents, subject to
obligations of confidentiality and non-use at least as restrictive as those set
forth in this Section 7.

 

ARTICLE 8.     RECORD KEEPING – AUDIT

 

8.1.     Licensee agrees to keep, and will ensure that each of its Affiliates
and sub-licensees keeps, accurate and correct records of Licensed Products
Developed, Manufactured and Commercialized under this Agreement appropriate to
determine the amount of royalties due to Licensor. These records shall be
retained for at least five (5) years following a given reporting period.

 

8.2.     Records shall be made available by Licensee, its Affiliates and
sub-licensees during normal business hours for inspection, by an accountant or
other designated auditor selected by Licensor for the sole purpose of verifying
reports, milestones achievements and royalty payments hereunder. The accountant
or auditor shall only disclose to Licensor information relating to the accuracy
of reports, milestones achievements and royalty payments made under this
Agreement or under the agreement concluded between Licensee and its Affiliates
or sub-licensees for the purpose of the Development, Manufacture or
Commercialization of the Licensed Product.

 

8.3.     Subject to Section 8.1 and 8.2, Licensor can demand at any time to
access the elements of the special accounting allowing the evaluation of the
commercial transactions made under the present Agreement.

 

8.4.     Audit shall be made at the expense of Licensor, except when an
inspection shows an underreporting or underpayment in  excess of three percent
(3%) for any twelve (12) month period. In such case, Licensee shall reimburse
Licensor for the cost of the audit at the time Licensee pays the unreported
milestones or the unreported royalties, including any additional payment and
interest as required by Section 5.7.

 

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8.5.     All payments required under this ARTICLE 8 shall be due within forty
five (45) days of the date Licensor provides Licensee notice of the payment due.

 

ARTICLE 9.     REPORTS ON PROGRESS, SALES AND PAYMENTS

 

9.1.     Licensee shall provide written annual reports on its Licensed Products
Development progress or efforts to Manufacture or Commercialize for the Field
within sixty (60) days after December 31 of each calendar year. These progress
reports shall include, but not be limited to: progress on research and
Development, status of applications for regulatory approvals, Manufacture and
status of sublicensing, marketing, importing, and sales during the preceding
calendar year, as well as, plans for the present calendar year.

 

9.2.     If reported progress differs from that projected between the Parties
and in Milestones, Licensee shall explain the reasons for such differences.
Licensee agrees to provide any additional information reasonably required by
Licensor to evaluate Licensee’s performance under this Agreement.

 

9.3.     Licensee shall report to Licensor the dates for achieving milestones,
the First Commercial Sale of each Licensed Product and the first commercial sale
of each Identical Product in each country of the Territory within thirty (30)
days of such occurrences.

 

9.4.     Licensee shall submit to Licensor, within forty (40) days after each
calendar half year ending June 30 and December 31, a royalty report setting
forth for the preceding half year period the amount of the Licensed Products and
Identical Products sold by or on behalf of Licensee in each country within the
Territory, the Net Sales and net sales of Identical Products, and the amount of
royalty accordingly due.

 

9.5.     With each royalty report, Licensee shall submit payment of earned
royalties due, in conformity with Section 5.3 and Section 5.5. If no earned
royalties are due to Licensor for any reporting period, the written report shall
so state. The royalty report shall be certified as correct by an authorized
officer of Licensee and shall include a detailed listing of all deductions made
to determine Net Sales made under ARTICLE 5 to determine royalties due.

 

9.6.     Licensee agrees to forward semi-annually to Licensor a copy of these
reports received by Licensee from its sublicensees during the preceding half
year period as shall be relevant to a royalty accounting to Licensor by Licensee
for activities under the sublicense.

 

ARTICLE 10.     PROSECUTION AND MAINTENANCE

 

10.1.     Each Party shall have the right, but not the obligation, at its sole
expense, to prosecute and maintain Patents solely owned by such Party, including
its own Background IP and its own Invention. Licensor shall notify Licensee in
writing of material aspects of the prosecution of Licensed Patents related to
the Field, and shall reasonably consider Licensee’s comments with respect
thereto in good faith.

 

10.2.     Subject to Section 10.3, Licensee shall reimburse Licensor for all
Third Party patent costs incurred for each Patent of the Licensed Patents as
from the Effective Date and until termination of the Agreement or expiry of the
last Valid Claim of the Licensed Patents. The sum reimbursed shall be creditable
on an annual basis against the annual fee provided in Section 5.1(ii).

 

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10.3.     If the Licensed Patents are licensed by Licensor to Third Parties,
outside of the Field, the Third Party Patent costs incurred for the Licensed
Patents as from the effective date of such additional licenses will be divided
by the number of licensees, including Licensee.

 

10.4.     Patent costs shall include all Third Party costs relating to the
Licensed Patents drafting, filing, prosecution, and maintenance in all
countries, including ex parte re-examination, grant, re-issuance and validation
in all countries.

 

ARTICLE 11.     INFRINGEMENT AND ENFORCEMENT

 

11.1.     Validity challenge against Licensed Patent

 

11.1.1.  In the event a Third Party initiate an action challenging the validity
of any of the Licensed Patents (“Third Party Challenge”), Licensor agrees to
notify Licensee of such an action. Third Party Challenge shall include but not
be limited to declaratory judgment action, opposition, post-grant proceedings,
interferences, inter-partes review, inter-partes re-examination and patent
validity challenges against national or regional courts.

 

11.1.2.  Unless the Parties otherwise agree in writing, Licensor shall have the
right, but not the obligation, to defend any Third Party challenge related to
the Licensed Patents, and Licensee shall reasonably assist Licensor and
cooperate in any such litigation at Licensor’s request and at Licensee’s
expenses. Licensor shall keep Licensee reasonably informed with respect to the
progress of any such litigation.

 

11.1.3.  If Licensor does not exercise its right to defend any Third Party
challenge related to the Licensed Patents within the Field within a commercially
reasonable period of time, then Licensee shall have such right to defend such
Third Party challenge that would limit Licensee’s rights in the Field, and
Licensee agrees to keep Licensor reasonably informed of all material
developments in connection with such Third Party challenge. Licensee shall not
settle or otherwise make any admissions in connection with such Third Party
challenge that would materially adversely affect Licensor’s rights or interests,
without Licensor’s prior written consent.

 

11.1.4.  Licensee shall reimburse Licensor for all Third Party costs incurred
due to Third Party Challenges related to the Field.

 

11.2.     Infringement of Licensed Patents by Third Parties

 

11.2.1.  Licensor and Licensee agree to notify each other promptly of each
infringement or possible infringement of the Licensed Patents, as well as of any
facts which may affect the validity, scope, or enforceability of the Licensed
Patents of which either Party becomes aware.

 

11.2.2.  Licensor may enforce any Licensed Patents with respect to Third Party
infringements, at its sole discretion and at its sole expense. Licensor shall
inform Licensee of its decision regarding such enforcement.

 

·

In case of such enforcement action brought by Licensor, Licensee shall be
entitled to join such action at its own costs and expenses. Licensor shall
remain free to withdraw its action at any time during the procedure.

 

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·

In case Licensor does not bring such enforcement action, Licensee may enforce
any Licensed Patents with respect to Third Party infringements at its own costs
and expenses only after having obtained Licensor’s written approval which shall
not be unreasonably withheld with regards to Licensor own interests. Licensor
shall be entitled to join such action at its own costs and expenses.

 

11.2.3.  Licensee shall reimburse Licensor for all costs incurred due to such
action if the infringement related to the Licensed Patents by Third Parties
relates to the Field.

 

11.2.4.  Any damages or other compensation, monetary or not, shall be collected
by Licensor. In case Licensee joins any action to enforce Licensed Patents
initiated by Licensor, it may request and collect damages or other compensation
for its own prejudice.

 

ARTICLE 12.     TERM, TERMINATION AND MODIFICATION OF RIGHTS

 

12.1.     Term

 

This Agreement commences on the Effective Date and shall continue on a Licensed
Product-by-Licensed Product and country-by-country basis until the expiration of
the Royalty Term with respect to such Licensed Product in such country (the
“Term”).

 

12.2.     Termination

 

12.2.1.  Termination for cause

 

12.2.1.1     Either Party may terminate this Agreement entirely, effective upon
written notice to the other Party, if the other Party materially breaches this
Agreement and fails to cure such breach within ninety

(90) days after receiving written notice thereof.

 

12.2.1.2  While the following list cannot be construed to limit Licensor’s right
to terminate the Agreement entirely in case of a material breach by Licensee of
the Agreement under Sections 12.2, Licensor shall in particular have the right
to terminate this Agreement on a country-by-country basis and Licensed Product-
by-Licensed Product basis or in its entirety, effective upon written notice to
Licensee, if Licensee fails to cure such breach within ninety (90) days after
receiving written notice thereof:

 

(a)

if Licensor determines that Licensee has willfully made a false statement of, or
omitted, a material fact in any report required by this Agreement, including
under Article 9;

 

(b)

if Licensor determines that Licensee is not keeping Licensed Products reasonably
available to the public after commercial use commences in application of Section
4.5;

 

(c)

if Licensor determines that Licensee cannot reasonably justify a failure to
comply with undertakings provided for in Section 4.5;

 

(d)

if Licensee does not enter into the clinical trial agreement mentioned in
Section 4.3;

 

(e)

if the Clinical Trial contemplated in such clinical trial agreement mentioned in
Section 4.3 is not entirely performed for reason attributable to Licensee;

 

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(f)

if Licensee fails to make any royalties and other payments, including patent
expense reimbursements, required to be paid by Licensee on the due date pursuant
to ARTICLE 5.

 

12.2.1.3  Notwithstanding the foregoing, in the event that a breach by Licensee
pursuant to Section 12.2.1.1 or 12.2.1.2 would reasonably require more than
ninety (90) days to cure, and Licensee provides Licensor with a reasonable plan
for diligently curing such breach, such ninety (90)-day cure period shall be
extended for the amount of time set forth in the plan not to exceed an
additional ninety (90) days.

 

12.2.2.     Abandonment

 

12.2.2.1   On a Licensed Product-by-Licensed Product and country-by-country
basis, Licensor may terminate the license under Section 2.1 with respect to a
Licensed Product in a given country in the event of Abandonment by Licensee with
respect to such Licensed Product in such country by giving written notice to
Licensee. If such Abandonment has been cured by Licensee within the Abandonment
Cure Period, such termination shall not occur. If such Abandonment has not been
cured by Licensee within the Abandonment Cure Period then Licensor shall be
entitled at its own discretion to (i) terminate the license under Section 2.1
entirely with respect to such Licensed Product in such country, with immediate
effect upon delivery to Licensee of a written notice, or to (ii) modify the
license grant in removing the exclusivity granted by Licensor to Licensee under
the Licensed Patents in the Field with respect to such Licensed Product in such
country, with immediate effect upon delivery to Licensee of a written notice.

 

12.2.2.2   Notwithstanding anything in this Section 12.2, in the event Licensee
fails to achieve a First Commercial Sale in EU or in the USA before December
31th, 2020, Licensor may (i) terminate this Agreement in its entirety with
immediate effect upon delivery to Licensee of a written notice, or (ii) modify
the license under Section 2.1, in removing the exclusivity granted by Licensor
to Licensee under the Licensed Patents in the Field with respect to any or all
Licensed Products in any or all countries, with immediate effect upon delivery
to Licensee of a written notice.

 

12.2.3.     Action against the Licensed Patents

 

By giving notification to Licensee, Licensor may terminate immediately the
Agreement without any further financial or other obligations to Licensee, in the
event that Licensee, its Affiliates or its sublicensees brings an action against
Licensor or against the Licensed Patents, in any country of the Territory, in
each case, challenging the validity, enforceability or scope of any Licensed
Patent, in particular if Licensee, its Affiliates or its sublicensees, directly
or indirectly, files an opposition to the Licensed Patents before any patent
office in the Territory.

 

12.2.4.     Commercialization of a Licensed Product outside the Field

 

By giving written notification to Licensee, Licensor may terminate immediately
the Agreement without any further financial or other obligations to Licensee, in
the event that Licensee, on its own or on its behalf, Commercializes a Licensed
Product outside the Field.

 

12.2.5.     Insolvency / Bankruptcy

 

Either Party may terminate the Agreement in the event that the other Party
becomes insolvent, files a petition in bankruptcy, has such a petition filed
against it, determines to file a petition in bankruptcy, or receives notice of a
Third Party’s intention to file an involuntary petition in bankruptcy, and such
proceeding or action remains un-dismissed or un-stayed for a period of sixty
(60) days, to the extent permitted by applicable law.

 

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Each Party shall inform the other Party within thirty (30) days of occurrence of
any such event.

 

12.3.     Consequences of Termination

 

12.3.1.  Accrued Obligations

 

Expiration or termination of this Agreement for any reason shall not release any
Party of any obligation or liability which, at the time of such expiration or
termination, has already accrued or which is attributable to a period prior to
such expiration or termination.

 

12.3.2.  Consequence in Case of Termination of the Agreement in its Entirety

 

In the event of termination of this Agreement in its entirety by Licensor under
Section 12.2, all license rights granted to Licensee pursuant to Article 2 shall
terminate and any Development and Commercialization of the Licensed Product by
Licensee should cease in all the Territory.

 

12.3.3.  Within ninety (90) days of expiration or termination of this Agreement
under this Article 12, a final report shall be submitted by Licensee to
Licensor. Any royalty payments, including those incurred but not yet paid, and
those related to patent expense, due to Licensor shall become immediately due
and payable upon termination or expiration. Unless otherwise specifically
provided for under this Agreement, upon termination of this Agreement, Licensee
shall provide Licensor with written certification of the destruction of the
Licensed Products (i.e. destruction of the products integrating patented vectors
as detailed in Licensed Patents).

 

12.4.     Non-Exclusive Remedy

 

Termination of this Agreement by a Party shall be without prejudice to other
remedies such Party may have at law or equity.

 

12.5.     Survival

 

Notwithstanding the expiry of the term or prior termination of this Agreement
the provisions of Articles 1 (as applicable), 6 (Intellectual Property), 7
(Confidentiality), 8 (Record Keeping - Audit), 14 (Indemnification, Insurance
and Liability), 15 (Publication and Publicity), 16 (Dispute Resolution) and 17
(Miscellaneous) and Sections 5.3.3, 5.6 (Payment Method), 5.7 (Late Payment),
12.3 (Consequences of Termination), 12.4 (Non-Exclusive Remedy) and 12.5
(Survival), and any other provisions that by their nature or intent are intended
to survive post expiration or termination, shall remain in full force and
effect.

 

ARTICLE 13.     REPRESENTATION AND WARRANTIES

 

13.1.     Licensor represents and warrants to Licensee, as of the Effective
Date, that: (a) the information on the Licensed Patents listed in Exhibit A is
accurate; (b) the Licensed Patents constitute all of the Patents owned or
controlled by Licensor that are related to the construction of a
self-complementary AAV9-SMN and AAV9-SMN systemic administration; (c) Licensor
has the right to grant to Licensee the rights and licenses in and to the
Licensed Patents set forth in this Agreement; (d) Licensor has not received any
notice of infringement of any Licensed Patents other those described in Exhibit
A at the Effective Date, and to Licensor’s knowledge, no third party is
infringing, or challenging the inventorship, ownership or enforceability of, any
Licensed Patents in the Field; and (e) Licensor has not entered, and

 

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shall not enter, into any agreement with any Third Party (including any related
party agreements) that is in conflict with the rights granted to Licensee under
this Agreement.

 

13.2.     Except as expressly set forth in Section 13.1 of this Agreement,
Licensor does not warrant the validity of the Licensed Patents and makes no
representations whatsoever with regard to the scope of the Licensed Patents, or
that the Licensed Patents may be exploited without infringing other patents or
other intellectual property rights of Third Parties. Licensee acknowledges that
the status of the Licensed Patents are available to the public and that Licensee
has been provided with all reasonable information regarding the Licensed Patents
available at the Effective Date and that Licensee is full aware of the filing
procedures and the status of the Licensed Patents.

 

13.3.     Except as expressly set forth in Section 13.1 of this Agreement,
Licensor makes no warranties, expressed or implied, of merchantability or
fitness for a particular purpose of any subject matter defined by the claims of
the Licensed Patents or tangible materials related thereto.

 

13.4.     Subject to Section 14.1.1, Licensee shall indemnify and hold Licensor,
its directors, employees, students, agents, and consultants harmless from and
against all liability, demands, damages, expenses, and losses, including but not
limited to death, personal injury, illness, or property damage in connection
with or arising out of the Development, Manufacture, Commercialization, or use
of any Licensed Patents or Licensed Products by Licensee, its Affiliates or
sublicensees.

 

13.5.     If data passed on for the purpose of use of one Party (the “receiving
Party”) contain personal data, the other Party guarantees to the receiving Party
that he complied with all the obligations imposed under the term of the January
6th, 1978 law "Computing and Liberties" and of the Directive 95/46/EC of the
European Parliament and of the Council of 24 October 1995 “on the protection of
individuals with regard to the processing of personal data and on the free
movement of such data” that will be repealed, as from the May 25, 2018, by the
regulation (EU) 2016/679 of the European Parliament and of the Council of 27
April 2016 “on the protection of natural persons with regard to the processing
of personal data and on the free movement of such data”, and that he informed
the concerned physical persons of use which is made of the aforementioned
personal data. As such, each Party guarantees the other Party against any
appeal, complaint or demand from a person whose personal data would be
reproduced and host via Licensee.

 

ARTICLE 14.     INDEMNIFICATION, INSURANCE AND LIABILITY

 

14.1.     Indemnification

 

14.1.1.  Indemnity.

 

a.

Subject to the Section 14.1.1.b) below, the defaulting Party (the “Indemnifying
Party”) will indemnify, defend, and hold harmless the other Party (the
“Indemnified Party”) and its Affiliates, its sublicensees and its directors,
officers, employees, agents (collectively the “Indemnitees”), against any Losses
incurred by or imposed upon any of the Indemnitees in connection with any Third
Party claims, suits, investigations, actions, judgments or demands arising out
of or related to :

 

i.

any  breach  of  this  Agreement  by  the  Indemnifying  Party,  its
Indemnitees, Affiliates or sublicensees; or

ii.

the wrongful intentional acts or omissions of the Indemnifying Party, its
Indemnitees, Affiliates or sub-licensees, in connection with the performance of
its obligations or exercise of its rights under this Agreement;

 

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b.

Except, in each case, to the extent that the respective Losses are caused by the
negligence or willful misconduct of, or breach of this Agreement or violation of
law by, the Indemnified Party, Indemnitees, its Affiliates, sublicensees.

 

14.1.2.  Settlement. Notwithstanding anything to the contrary in this Agreement,
neither Party will enter into any settlement, consent judgment, or other
voluntary final disposition of any claim that has an adverse effect on the
rights of the other Party, or admits any wrongdoing or fault by the other Party,
or imposes on the other Party any payment or other liability, without the prior
written consent of such other Party, except when Licensor defends the Licensed
Patents in application of Article 11.

 

14.2.     Insurance. The Licensee agrees to maintain a liability insurance
program consistent with sound business practice.

 

14.3.     Liability. Notwithstanding anything in this Agreement or otherwise,
neither Party, their directors, employees, students, agents, and consultants as
applicable, will be liable to the other with respect to any subject matter of
this Agreement for any indirect, punitive, special or consequential damages,
including incidental, or lost profits, even if such Party has been informed,
should have known or in fact knew of the possibility of such damages.

 

ARTICLE 15.     PUBLICATION AND PUBLICITY

 

15.1.     Scientific Publication

 

Licensee and its Affiliates shall have the right to publish or publicly
disclose, as part of scientific publications or scientific presentations, the
results generated in the course of performing by Licensee any research related
to the Licensed Products. Licensor may publish or publicly disclose any
information or results generated in the course of performing any research
related to the Licensed Patents without the prior written consent of Licensee.

 

15.2.     Press Release and Public Communication

 

15.2.1   The Parties shall issue the initial press release set forth on Exhibit
B hereto following the Effective Date.

 

15.2.2     Except as required by law, neither Licensor nor Licensee (or its
Affiliates or sublicensees) shall issue or cause the publication of any other
press release or public announcement (orally or in writing) regarding the
existence or terms of this Agreement without the express prior written approval
of the other Party, which approval shall not be unreasonably withheld,
conditioned or delayed. The Licensor or the Licensee (or its Affiliates or
sublicensees) shall reasonably consider, timely comments from the other Party on
such publication or press release.

 

15.2.3   Unless prohibited by law and unless otherwise requested by Licensor,
Licensee shall include the words “Developed in partnership with Genethon” in any
press release, public presentation, or medical communication related to the
Licensed Product where Licensee believes it is appropriate and reasonable to do
so and in all cases where Licensee includes the names of its other licensing
partners. In addition, Licensee would include the words “Developed in
partnership with Genethon” in significant global press releases announcing
initiation of a Clinical Trial, results of a Clinical Trial, submission for
regulatory approval and the receipt of regulatory approvals. In France, Licensee
would help promote Licensor’s

 

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branding by including the wording when announcing any joint advocacy
communication, local advisory board and local conference/presentation.

 

15.2.4   The Parties acknowledge that the French Association against Myopathies
(AFM-Téléthon, Association Française contre les Myopathies), which is Genethon’s
founder and principal funder, in view of accomplishing its recognized role of
working in the public interest, that is, by curing rare diseases and reducing
the disabilities to which they give rise, has an obligation to provide the
general public with information on the research programs and work to which it
provides a financial contribution, in order to facilitate, directly or
indirectly, an understanding of these diseases, the development of treatments,
and the prevention of disabilities.

 

Subject to Licensee’s prior approval which should not be unreasonably withheld,
each Party agrees that AFM-Telethon may use its name and report thereon, orally
and/or in writing, to the general public, notably during fundraising campaigns
and during the Telethon and annual general meeting, without releasing details
which may be detrimental to such Party’s intellectual property rights or
Confidential Information.

 

ARTICLE 16.     DISPUTE RESOLUTION

 

16.1.     In the event of any dispute arising out of or relating to this
Agreement, the Parties will use all reasonable efforts to arrive at a mutually
acceptable resolution.

 

16.2.     If a dispute is not resolved within sixty (60) days from the date that
the other Party receives notice of the dispute, the matter will be referred to
the CEOs of both Parties (or their designees) for resolution within thirty (30)
days after such escalation to such officers.

 

16.3.     If an agreement is not reached within such thirty (30) day period,
then the dispute will be resolved consistent with Section 17.2.

 

16.4.     The provisions of this Article 16 shall survive for five (5) years
from the date of termination or expiration of this Agreement.

 

16.5.     Subject to the non-terminating Party’s right to seek an injunction or
protective order, the provisions of this Article 16 shall not affect the right
of either Party to terminate this Agreement pursuant to Section 12.2 of this
Agreement.

 

ARTICLE 17.     MISCELLANEOUS

 

17.1.     Governing Law

 

This Agreement and all disputes arising out of or related to this Agreement, or
the performance, enforcement, breach or termination hereof, and any remedies
relating thereto, will be construed, governed, interpreted and applied in
accordance with the laws of Belgium, without regard to conflict of laws
principles, except that questions affecting the construction and effect of any
patent will be determined by the law of the country in which the patent will
have been granted.

 

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17.2.     Attribution of Jurisdiction

 

Any dispute arising out of or relating to this Agreement, any terms of this
Agreement or any breach of this Agreement (in particular with regard to the
negotiation, performance or termination of the Agreement) that is not resolved
pursuant to Article 16 will be resolved by the courts of Brussels (Belgium)
which will have exclusive jurisdiction, without regard to conflict of
jurisdiction principles thereof. The Parties hereby expressly exclude the
application of the United National Convention on Contracts for the International
Sales of Goods.

 

17.3.     Assignment of Rights and Obligations

 

This Agreement and its rights or obligations may not be assigned or otherwise
transferred by Licensee without the prior written consent of Licensor; provided
that, Licensee may make such an assignment or transfer without Licensor’s
consent, but with notice promptly following such assignment or transfer, to its
Affiliates or to a Third Party successor to all or substantially all of the
business of Licensee to which this Agreement relates, whether in a merger, sale
of stock, sale of assets or other transaction.

 

17.4.     Further Actions

 

Each Party agrees to execute, acknowledge and deliver such further instruments,
and to do all such other acts, as may be necessary or appropriate in order to
carry out the purposes and intent of the Agreement.

 

17.5.     Force Majeure

 

Except with respect to payment of money, no Party shall be liable to the other
Party for failure or delay in the performance of any of its obligations under
this Agreement for the time and to the extent such failure or delay is caused by
earthquake, riot, civil commotion, war, terrorist acts, strike, flood, or
governmental acts or restriction, or other cause that is beyond the reasonable
control of the respective Party (“Force Majeure”). The Party affected by such
Force Majeure will provide the other Party with full particulars thereof as soon
as it becomes aware of the same (including its best estimate of the likely
extent and duration of the interference with its activities), and will use
reasonable efforts to overcome the difficulties created thereby and to resume
performance of its obligations as soon as practicable. If the performance of any
such obligation under this Agreement is delayed owing to an event of Force
Majeure for any continuous period of more than ninety (90) days, the Parties
will consult with respect to an equitable solution, including the possibility of
the termination of this Agreement in accordance with Section 12.2.

 

17.6.     Representation by Legal Counsel

 

Each Party hereto represents that it has been represented by legal counsel in
connection with this Agreement and acknowledges that it has participated in the
drafting hereof. In interpreting and applying the terms and provisions of this
Agreement, the Parties agree that no presumption shall exist or be implied
against the Party which drafted such terms and provisions.

 

17.7.     Notices

 

Any notice, request, delivery, approval or consent required or permitted to be
given under this Agreement shall be in writing and shall be deemed to have been
sufficiently given if delivered in person, transmitted by facsimile (receipt
verified) or by express courier service (signature required) or five (5) days
after it was sent by registered letter, return receipt requested (or its
equivalent), provided that no postal strike or

 

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other disruption is then in effect or comes into effect within two (2) days
after such mailing, to the Party to which it is directed at its address or
facsimile number shown below or such other address or facsimile number as such
Party will have last given by notice to the other Party.

 

If to Licensor:

1bis, Rue de l’Internationale - 91000 EVRY, FRANCE

Attention: Alexandre LEMOALLE

Tel:

+33 1 69 47 25 87

Mail:

alemoalle@genethon.fr

 

If to Licensee:

2275 Half Day Road, Suite 200

Bannockburn, IL 60015

Attention: General Counsel

Fax:

847-510-0775

Mail:

mjohannesen521@avexis.com

 

17.8.     Entire Agreement

 

This Agreement, together with the exhibits A to B attached hereto, set forth the
entire agreement and understanding of the Parties hereto as to the subject
matter hereof, and supersedes all prior and contemporaneous discussions,
agreements and writings in respect.

 

17.9.     Amendment

 

No amendment, modification or supplement of any provision of this Agreement
shall be valid or effective unless made in writing and signed by a duly
authorized officer of each Party.

 

17.10.     Waiver

 

No provision of the Agreement shall be waived by any act, omission or knowledge
of a Party or its agents or employees except by an instrument in writing
expressly waiving such provision and signed by a duly authorized officer of the
waiving Party. The waiver by any of the Parties of any breach of any provision
hereof by another Party shall not be construed to be a waiver of any succeeding
breach of such provision or a waiver of the provision itself.

 

17.11.     Severability

 

If any clause or portion thereof in this Agreement is for any reason held to be
invalid, illegal or unenforceable, the same shall not affect any other portion
of this Agreement, as it is the intent of the Parties that this Agreement shall
be construed in such fashion as to maintain its existence, validity and
enforceability to the greatest extent possible. In any such event, this
Agreement shall be construed as if such clause of portion thereof had never been
contained in this Agreement, and there shall be deemed substituted therefor such
provision as will most nearly carry out the intent of the Parties as expressed
in this Agreement to the fullest extent permitted by Law.

 

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17.12.     Relationship of the Parties

 

Nothing herein will be deemed to establish a relationship of principal and agent
between Licensor and Licensee, nor any of their agents or employees, nor will
this Agreement be construed as creating any form of legal association or
arrangement which would impose liability upon one Party for the act or failure
to act of the other Party. Nothing in this Agreement, express or implied, is
intended to confer on any person other than the Parties or their permitted
assigns any benefits, rights or remedies.

 

17.13.     Third Party Beneficiaries

 

All rights, benefits and remedies under this Agreement are solely intended for
the benefit of the Parties (including any successor in interest or permitted
assigns), and no Third Party shall have any rights whatsoever to (a) enforce any
obligation contained in this Agreement, (b) seek a benefit or remedy for any
breach of this Agreement, or (c) take any other action relating to this
Agreement under any legal theory, including actions in contract, tort (including
negligence, gross negligence and strict liability), or as a defense, setoff or
counterclaim to any action or claim brought or made by the Parties.

 

17.14.     Counterparts

 

This Agreement may be executed in any number of counterparts, each of which need
not contain the signature of more than one Party but all such counterparts taken
together shall constitute one and the same agreement.

 

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Executed in two originals, one for each Party

 

 

For AVEXISFor GENETHON

at Bannockburn, ILat Evry

the 9th of March, 2018the 9th of March, 2018

 

 

/s/ RA Session II/s/ Frederic REVAH

 

Name: RA Session II       Name : Frederic REVAH

Title: Senior Vice President Corporate Title : Chief Executive Officer
Strategy and Business Development

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Exhibit A

Licensed Patents at the Effective Date

 

Country

Filing date

Administration

Product*

Filing number

Publication number

 

 

 

 

 

 

 

 

 

 

 

 

 

 

“CNS gene delivery using peripheral administration of AAV vectors”  GENETHON
[B00701 MB],  CNRS [DI 02300-01]

 

 

 

Europe

23/07/2007

N/A (withdrawn)

N/A (withdrawn)

EP 07301263.5

EP 2019143

 

 

 

PCT

22/07/2008

N/A (expired)

N/A (expired)

PCT/EP2008/059595

WO 2009/013290

 

 

 

Europe

22/07/2008

All

scAAV9-SMN

EP 08786331.2

EP2185712

 

 

 

Europe

22/07/2008

N/A (other serotype)**

N/A            (other serotype**

EP 12 172 848.9

EP2514827

 

 

 

USA

22/07/2008

Intravascular or

Intraperitoneal

scAAV9-SMN

12/452,789

US 2010/0130594

 

 

 

Canada

22/07/2008

Intravascular, intraperitoneal, intramuscular

scAAV9-SMN

CA20082694241

CA 2694241

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

“Widespread Gene Delivery to Motor Neurons Using Peripheral Injection of AAV
Vectors”  GENETHON [B00703 MB],  CNRS [DI 02300- 02]

 

 

 

Europe

05/10/2007

N/A (withdrawn)

N/A (withdrawn)

EP 07301435.9

EP2058401

 

 

 

PCT

03/10/2008

N/A (expired)

N/A (expired)

PCT/EP2008/063297

W02009/043936

 

 

 

Canada

03/10/2008

Intravascular, intraperitoneal, intramuscular

scAAV9-SMN

CA20082701561

CA 2701561

 

 

 

China

03/10/2008

N/A (rejected)

N/A (rejected)

CN20088117413

CN 101883859

 

 

 

China

03/10/2008

Peripheral

scAAV9-SMN

CN201510438197.1

CN 105087650

 

 

 

Europe

03/10/2008

Intravenous, Intraperitoneal  or

intramuscular

scAAV9-SMN

EP 08836776.8

EP 2212424

 

 

 

Europe

03/10/2008

Peripheral

ssAAV9-SMN

EP 12 180 951.1

EP 2527457

 

 

 

USA

03/10/2008

Intravenous        or intra-arterial

scAAV9-SMN

12/734,016

US 2010/0240739

 

 

 

USA

22/09/2017

All

scAAV9-SMN

15/713,347

 

 

 

 

Japan

03/10/2008

Peripheral

scAAV9-SMN

JP20100527467T

JP 2010540598

 

 

 

Japan

01/07/2014

Peripheral

ssAAV9-SMN

2014-136031

JP2014221789

 

 

 

 

 

 

 

 

 

*: By product we mean the Licensed Product constructions covered by the Licensed
Patents in the Field of the license granted under section 2.1, i.e. restricted
to AAV9.

 

**: The European divisional with the publication number EP2514827 covers a
serotype that is not included in the Field of the License.

 

 

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Exhibit B
Press Release

 

Picture 1 [avxs20180331ex10140af41001.jpg]

Picture 3 [avxs20180331ex10140af41002.jpg]

 

Media Inquiries:

 

Lauren Barbiero W2O Group 646-564-2156

lbarbiero@w2ogroup.com

 

Investor Inquiries:

Jim Goff AveXis, Inc. 650-862-4134

jgoff@avexis.com

 

AFM-Telethon Press Contact:

Stéphanie Bardon

+330169471278

presse@afm.genethon.fr

 

AveXis Enters into Licensing Agreement with Genethon

Includes exclusive worldwide rights to AAV9-SMN product and route of
administration

 

Chicago, Ill. and Evry, France (March XX, 2018) – AveXis, Inc. (NASDAQ:AVXS) and
Genethon today announced they have entered into an exclusive, worldwide license
agreement for in vivo gene therapy delivery of AAV9 vector into the central
nervous system (CNS) for the treatment of spinal muscular atrophy (SMA).

 

“Adding to our robust intellectual property estate, this agreement further
strengthens our position by providing freedom to operate when using intravenous
or intrathecal routes of administration to deliver the AAV9 vector into the CNS
for the treatment of SMA,” said Sean Nolan, President and Chief Executive
Officer of AveXis. “With our proprietary gene therapy, AVXS-101, currently being
evaluated in patients with SMA in ongoing clinical trials in the U.S., and soon
in Europe, we are pleased to have this exclusive worldwide agreement in place.”

 

Under the terms of the agreement, Genethon granted AveXis a license to patents
in the U.S., Europe and Japan, for the AAV9 SMN product and in vivo gene therapy
delivery of AAV9 vector into the CNS using intrathecal or intravenous routes of
administration for the treatment of SMA.

 

“Genethon is pleased to enter into this agreement with AveXis and to contribute
to the efforts for the development of treatments for SMA patients who have
urgent medical needs,” said Frédéric Revah, Chief Executive Officer of Genethon.
“It demonstrates Genethon’s capability

 

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to develop effective first-in-class technologies and the excellence of our
translational research driven by the commitment to treat patients living with
rare diseases.”

 

About SMA

SMA is a severe neuromuscular disease characterized by the loss of motor neurons
leading to progressive muscle weakness and paralysis. SMA is caused by a genetic
defect in the SMN1 gene that codes SMN, a protein necessary for survival of
motor neurons. The incidence of SMA is approximately one in 10,000 live births
and is the leading genetic cause of infant mortality.

 

The most severe form of SMA is Type 1, a lethal genetic disorder characterized
by motor neuron loss and associated muscle deterioration, which results in
mortality or the need for permanent ventilation support before the age of two
for greater than 90 percent of patients. SMA Type 2 typically presents between
six and 18 months of age, and those affected will never walk without support and
most will never stand without support. SMA Type 2 results in mortality in more
than 30 percent of patients by the age of 25.

 

About AVXS-101

AveXis’ initial product candidate, AVXS-101, is its proprietary gene therapy
currently in development for the one-time treatment of SMA Types 1 and 2,
designed to address the monogenic root cause of SMA and prevent further muscle
degeneration by addressing the defective and/or loss of the primary SMN gene.
AVXS-101 also targets motor neurons, providing rapid onset of effect and
crossing the blood brain barrier to allow effective targeting of both central
and systemic features.

 

About AveXis, Inc.

AveXis, Inc. is a clinical-stage gene therapy company, dedicated to developing
and commercializing novel treatments for patients suffering from rare and
life-threatening neurological genetic diseases. Our initial product candidate,
AVXS-101, is our proprietary gene therapy currently in development for the
treatment of spinal muscular atrophy, or SMA, Type 1, the leading genetic cause
of infant mortality, and SMA Type 2. The U.S. Food and Drug Administration, or
FDA, has granted AVXS-101 Orphan Drug Designation for the treatment of all types
of SMA and Breakthrough Therapy Designation, as well as Fast Track Designation
for the treatment of SMA Type 1. In addition to developing AVXS-101 to treat SMA
Type 1 and Type 2, we also plan to develop other novel treatments for rare
neurological diseases, including Rett syndrome and a genetic form of amyotrophic
lateral sclerosis caused by mutations in the superoxide dismutase 1 (SOD1) gene.

 

About Genethon

Created by the AFM-Telethon, the French Muscular Dystrophy Association (AFM),
Genethon, located in Evry, France, is a non-profit R&D organization dedicated to
the development of biotherapies for orphan genetic diseases, from the research
to clinical validation. Genethon is specialized in the discovery and development
of gene therapy drugs and has multiple ongoing programs at clinical, preclinical
and research stage for neuromuscular, blood, immune system, and liver diseases.

 

AveXis Forward-Looking Statements

This press release contains "forward-looking statements," within the meaning of
the Private Securities Litigation Reform Act of 1995, regarding, among other
things, AveXis’ freedom to operate afforded by the license agreement with
Genethon and AveXis’ research, development and regulatory plans for AVXS-101.
Such forward-looking statements are based on current

 

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expectations and involve inherent risks and uncertainties, including factors
that could delay, divert or change any of them, and could cause actual results
to differ materially from those projected in its forward-looking statements.
Meaningful factors which could cause actual results to differ include, but are
not limited to, the scope, progress, expansion, and costs of developing and
commercializing AveXis’ product candidates; regulatory developments in the U.S.
and EU, as well as other factors discussed in the "Risk Factors" and the
"Management's Discussion and Analysis of Financial Condition and Results of
Operations" sections of AveXis’ Annual Report on Form 10-K for the year ended
December 31, 2017, filed with the SEC on February 28, 2018. In addition to the
risks described above and in the Annual Reports on Form 10-K, Quarterly Reports
on Form 10-Q, Current Reports on Form 8-K and other filings with the SEC, other
unknown or unpredictable factors also could affect AveXis’ results. There can be
no assurance that the actual results or developments anticipated by AveXis will
be realized or, even if substantially realized, that they will have the expected
consequences to, or effects on, AveXis. Therefore, no assurance can be given
that the outcomes stated in such forward- looking statements and estimates will
be achieved.

 

# # #

 

 

 

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