Exhibit 10.3

***Text Omitted and Filed Separately

with the Securities and Exchange Commission.

Confidential Treatment Requested

Under 17 C.F.R. Sections 200.80(c) and 240.24b-2

EXECUTION VERSION

CONFIDENTIAL

AMENDMENT NO. 1 TO

AMENDED AND RESTATED LICENSE AGREEMENT

This Amendment No. 1 to the Amended and Restated License Agreement, (the
“Amendment”) is entered into as of September 11, 2018 (the “Amendment Effective
Date”), by and between:

THE REGENTS OF THE UNIVERSITY OF CALIFORNIA, a California corporation having its
statewide administrative offices at 1111 Franklin Street, Oakland, California
94607-5200 (“University”), represented by its San Diego campus having an address
at University of California, San Diego, Office of Innovation and
Commercialization, Mail Code 0910, 9500 Gilman Drive, La Jolla, California
92093-0910 (“UCSD”); and

HORIZON ORPHAN LLC, a Delaware limited liability company having an address at
150 South Saunders Road, Lake Forest, Illinois 60045 (“Licensee”),

each a “Party” and collectively the “Parties”.

All capitalized terms not herein defined shall have the meaning ascribed to them
in the Agreement (as defined below).

Background

WHEREAS, UCSD and Licensee, as successor-in-interest to Raptor Pharmaceuticals,
Inc. (f/k/a Encode Pharmaceuticals, Inc.), are parties to that certain License
Agreement, as amended and restated on May 31, 2017 and effective as of
October 31, 2007 (the “Agreement”);

WHEREAS, on July 2, 2018 and pursuant to Paragraph 7.4 of the Agreement, the
Parties mutually terminated the license granted under the Agreement solely with
respect to the NASH Indication (the “NASH Termination”);

WHEREAS, on September 11, 2018 and pursuant to Paragraph 7.4 of the Agreement,
the Parties mutually terminated the license granted under the Agreement solely
with respect to the HD Indication (the “HD Termination”);

WHEREAS, Licensee has corrected inventorship with respect to the Patent Rights
under SD2017-236 “Delayed release cysteamine bead formulation” to add Dr. Ranjan
Dohil as an inventor (the “Inventorship Correction”); and

WHEREAS, the Parties wish to amend the Agreement to reflect the NASH
Termination, the HD Termination and the Inventorship Correction, on the terms
and conditions set forth below.

NOW THEREFORE, in consideration of the mutual promises and covenants hereinafter
set forth, the Parties agree to amend the Agreement as follows:

Agreement

 

1.

Exhibit A. Exhibit A to the Agreement shall be and hereby is amended and
restated in its entirety by the Exhibit A-1 attached hereto.

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CONFIDENTIAL

 

2.

Full Force and Effect. Except as set forth in this Amendment, in all other
respects the Agreement remains in full force and effect.

 

3.

Counterparts. This Amendment may be executed in two or more counterparts, each
of which will be deemed an original and all of which will together be deemed to
constitute one agreement. The Parties agree that the execution of this Amendment
by industry standard electronic signature software and/or by exchanging PDF
signatures shall have the same legal force and effect as the exchange of
original signatures.

[Signature page follows]

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The Parties have executed this Amendment to be effective as of the Amendment
Effective Date.

 

HORIZON ORPHAN LLC By:   /s/ Jeffrey Sherman Name: Jeffrey Sherman Title: Chief
Medical Officer

 

THE REGENTS OF THE UNIVERSITY
OF CALIFORNIA By:   /s/ Ruben Flores Name: Ruben Flores Title: Director of
Commercialization

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Exhibit A-1

Patent Rights

[See Attached]

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CONFIDENTIAL

EXHIBIT A-1

[…***…]

***Confidential Treatment Requested