Exhibit 10.1

CONFIDENTIAL TREATMENT REQUESTED

Redacted portions are indicated by [****]1

AMENDMENT NO. 1 TO LICENSE AGREEMENT

This AMENDMENT NO. 1 TO LICENSE AGREEMENT (herein referred to as “Amendment
No. 1”) is made and entered effective as of the 8th day of April, 2014 (the
“Effective Date”), by and between BioMarin Pharmaceutical Inc., a Delaware
corporation (hereinafter, “BioMarin”) and Catalyst Pharmaceutical Partners,
Inc., a Delaware corporation (hereinafter, “Catalyst”), each herein referred to
individually as “Party” and collectively as “Parties”.

WHEREAS, the Parties desire to amend that certain License Agreement, dated as of
October 26, 2012 (the “Agreement”), between BioMarin and Catalyst, to capture
certain sales by BioMarin to Catalyst under the Agreement of API, Firdapse
Tablets, and placebo prior to the Effective Date of this Amendment No. 1, and to
provide for an additional sale of Firdapse Tablets pursuant to this Amendment
No. 1; and

WHEREAS, the Parties desire to make certain other changes to the Agreement, as
more particularly set forth herein.

NOW, THEREFORE, in consideration of the premises and mutual covenants herein
contained, and for other good and valuable consideration, the receipt and
sufficiently of which are hereby acknowledged, the Parties hereto agree as
follows:

 

1. This Amendment No. 1 amends the Agreement in the manner set forth herein.
Except as expressly modified hereby, the Agreement shall continue in full force
and effect in accordance with its terms. Capitalized terms not otherwise defined
in this Amendment No. 1 shall have the meaning ascribed to such term in the
Agreement.

 

2. Section 5.1 of the Agreement is hereby deleted in its entirety and replaced
with the following:

“5.1 Clinical Supply of Firdapse. BioMarin shall deliver (or cause to be
delivered) to Catalyst, free of charge, BioMarin’s clinical inventory of
Firdapse and placebo reserved for the BioMarin Ongoing Study, as set forth in
Exhibit E, to be used by Catalyst as its clinical supply for the BioMarin
Ongoing Study. In addition to the quantities set forth in Exhibit E, Catalyst
may place orders for, and BioMarin shall sell to Catalyst during the term of the
Agreement: (a) up to a maximum amount of [****] kilograms of the active
pharmaceutical ingredient of Firdapse (“API”), at a per kilogram cost of
€[****]; (b) up to a maximum of [****] active tablets containing API (“Firdapse
Tablets”) packaged in blister cards, each containing 10mg tablets, at a per
tablet cost of €[****]; and (c) up to a maximum of [****] tablets of placebo, at
a per tablet cost of €[****]. Notwithstanding the foregoing, Catalyst
acknowledges and understands that the usable portion of a manufacturing run of
Firdapse Tablets may be less than the quantity ordered and agrees: (i) to
reimburse BioMarin based on the quantity ordered and not based on the usable
portion; (ii) that BioMarin shall not be responsible for replacing any unusable
portion of an order of Firdapse Tablets; and (iii) that the agreed upon per
tablet cost of €[****] into account the unusable portion of a manufacturing run.
The Parties agree that the final order pursuant to this Section 5.1 shall be an
order of [****] Firdapse Tablets to be manufactured by BioMarin’s contract
manufacturer, [****], for completion in approximately March 2014 (“Final
Order”); that Catalyst will arrange for and be fully responsible and liable for
pick up of the Final Order; and that Catalyst shall make a payment to BioMarin
of €[****] within 30 days of the Effective Date in full consideration for the
Final Order.”

 

1  [****] Certain confidential information contained in this document, marked by
brackets, has been omitted and filed separately with the Securities and Exchange
Commission pursuant to Rule 24b-2 of the Securities Exchange Act of 1934.

 

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3. Section 4.3 of the Agreement is hereby deleted in its entirety and replaced
with the following:

4.3 Regulatory Data. In addition to BioMarin’s technology transfer obligations
under Section 3.2, each Party shall provide the other Party on a timely basis
with copies of all material pre-clinical and clinical data generated or compiled
in connection with its Development or Commercialization of Licensed Products
(via electronic copies of such data in a form that may be analyzed and
manipulated by the other Party). For clarity, this shall include all analytical
data obtained with respect to Licensed Products, descriptions of the
manufacturing processes for Licensed Compounds and Licensed Products (and any
material changes thereto), case report forms and patient medical records
generated during Clinical Trials, and any data generated during post-marketing
studies. Further, it shall include all other clinical trial and available
post-approval patient safety and registry study data, including historical
regulatory data contained in patient registry filings and files. Catalyst shall
provide development reports required under Section 3.7 on an annual basis, and
both parties shall provide the other party with other information described
above within 30 days of any request by the other Party for such information that
is required to support Regulatory Filings in the ROW or in the Territory, as
applicable. Except for delivery of the development reports required under
Section 3.7, which shall be delivered at Catalyst’s cost, the party requesting
information pursuant to this Section 4.3 shall pay the producing party’s
reasonable external and internal costs (including without limitation internal
FTE costs) associated with providing the requested information, within 30 days
of invoice from the producing party.

 

4. Section 5.3 of the Agreement is hereby deleted in its entirety.

 

5. The following is hereby added to the end of Section 7.2(a) of the Agreement:

“No later than forty-five (45) days after the exchange of the Conducting Party’s
Development Cost expenditure information, the Parties shall reconcile all Joint
Development Cost expenditure amounts through a net payment to the Party
incurring greater Joint Development Cost expenditures in such Calendar Quarter.”

 

6. Sections 7.2 (b) and (c) of the Agreement are hereby deleted in their
entirety.

 

7. Catalyst and BioMarin hereby agree that, as of the Effective Date, the
Agreement is in full force and effect and both parties are in full compliance
with the Agreement. The Parties further agree that to the extent that, prior to
the Effective Date, there have been any violations of the Agreement by either
Party, then any and all such violations are hereby waived.

 

8. This Amendment No. 1 may be executed in any number of counterparts, each of
which shall be deemed an original, but all of which together shall constitute
one and the same instrument.

 

9. This Amendment No. 1 shall inure to the benefit of and be binding upon the
Parties hereto and their respective heirs, successors, trustees, transferees and
assigns

[Signatures on Next Page]

 

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IN WITNESS WHEREOF, the Parties hereto, intending to be legally bound hereby,
have caused this Amendment No. 1 to be executed and delivered by their proper
and duly authorized officers effective as of the Effective Date.

 

CATALYST PHARMACEUTICAL PARTNERS, INC.     BIOMARIN PHARMACEUTICAL INC.

/s/ Patrick J. McEnany

   

/s/ Joshua Grass

By:  

Patrick J. McEnany

    By:  

Joshua Grass

Its:  

President and CEO

    Its:  

SVP, Business and Corporate Development

 

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