<SEC-DOCUMENT>0001070081-18-000071.txt : 20181005
<SEC-HEADER>0001070081-18-000071.hdr.sgml : 20181005
<ACCEPTANCE-DATETIME>20181005172736
ACCESSION NUMBER:		0001070081-18-000071
CONFORMED SUBMISSION TYPE:	8-K
PUBLIC DOCUMENT COUNT:		3
CONFORMED PERIOD OF REPORT:	20181003
ITEM INFORMATION:		Other Events
ITEM INFORMATION:		Financial Statements and Exhibits
FILED AS OF DATE:		20181005
DATE AS OF CHANGE:		20181005

FILER:

	COMPANY DATA:	
		COMPANY CONFORMED NAME:			PTC THERAPEUTICS, INC.
		CENTRAL INDEX KEY:			0001070081
		STANDARD INDUSTRIAL CLASSIFICATION:	PHARMACEUTICAL PREPARATIONS [2834]
		IRS NUMBER:				043416587
		STATE OF INCORPORATION:			DE
		FISCAL YEAR END:			1231

	FILING VALUES:
		FORM TYPE:		8-K
		SEC ACT:		1934 Act
		SEC FILE NUMBER:	001-35969
		FILM NUMBER:		181111410

	BUSINESS ADDRESS:	
		STREET 1:		100 CORPORATE COURT
		CITY:			SOUTH PLAINFIELD
		STATE:			NJ
		ZIP:			07080-2449
		BUSINESS PHONE:		9082227000

	MAIL ADDRESS:	
		STREET 1:		100 CORPORATE COURT
		CITY:			SOUTH PLAINFIELD
		STATE:			NJ
		ZIP:			07080-2449

	FORMER COMPANY:	
		FORMER CONFORMED NAME:	PTC THERAPEUTICS INC
		DATE OF NAME CHANGE:	19980909
</SEC-HEADER>
<DOCUMENT>
<TYPE>8-K
<SEQUENCE>1
<FILENAME>a8-k10518.htm
<DESCRIPTION>8-K
<TEXT>
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<div><a name="sd0e7aa1f2db040689e024be9e622b05c"></a></div><div><div style="line-height:120%;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;"><br></font></div></div><div><br></div><div style="line-height:174%;text-align:center;font-size:18pt;"><font style="font-family:inherit;font-size:18pt;font-weight:bold;">UNITED STATES</font></div><div style="line-height:174%;text-align:center;font-size:18pt;"><font style="font-family:inherit;font-size:18pt;font-weight:bold;">SECURITIES AND EXCHANGE COMMISSION</font></div><div style="line-height:174%;text-align:center;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;font-weight:bold;">WASHINGTON, D.C. 20549</font></div><div style="line-height:174%;text-align:center;font-size:18pt;"><font style="font-family:inherit;font-size:18pt;font-weight:bold;">FORM 8-K</font></div><div style="line-height:174%;text-align:center;font-size:12pt;"><font style="font-family:inherit;font-size:12pt;font-weight:bold;">CURRENT REPORT</font></div><div style="line-height:174%;text-align:center;font-size:12pt;"><font style="font-family:inherit;font-size:12pt;font-weight:bold;">Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934</font></div><div style="line-height:174%;text-align:center;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">Date of Report (Date of earliest event reported):  </font><font style="font-family:inherit;font-size:10pt;font-weight:bold;">October 3, 2018</font></div><div style="line-height:174%;text-align:center;font-size:18pt;"><font style="font-family:inherit;font-size:18pt;font-weight:bold;">PTC THERAPEUTICS, INC.</font></div><div style="line-height:174%;text-align:center;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">(Exact Name of Company as Specified in Charter)</font></div><div style="line-height:120%;padding-bottom:9px;text-align:left;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;"><br></font></div><div style="line-height:120%;text-align:center;font-size:10pt;"><div style="padding-left:0px;text-indent:0px;line-height:normal;padding-top:10px;"><table cellpadding="0" cellspacing="0" style="font-family:Times New Roman;font-size:10pt;margin-left:auto;margin-right:auto;width:677px;border-collapse:collapse;text-align:left;"><tr><td colspan="5"></td></tr><tr><td style="width:214px;"></td><td style="width:16px;"></td><td style="width:214px;"></td><td style="width:16px;"></td><td style="width:214px;"></td></tr><tr><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="text-align:center;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;font-weight:bold;">Delaware</font></div></td><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="overflow:hidden;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">&#160;</font></div></td><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="text-align:center;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;font-weight:bold;">001-35969</font></div></td><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="overflow:hidden;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">&#160;</font></div></td><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="text-align:center;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;font-weight:bold;">04-3416587</font></div></td></tr><tr><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="padding-bottom:2px;padding-top:2px;text-align:center;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">(State or Other Jurisdiction</font></div><div style="padding-bottom:2px;padding-top:2px;text-align:center;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">of Incorporation)</font></div></td><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="overflow:hidden;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">&#160;</font></div></td><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="padding-bottom:2px;padding-top:2px;text-align:center;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">(Commission</font></div><div style="padding-bottom:2px;padding-top:2px;text-align:center;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">File Number)</font></div></td><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="overflow:hidden;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">&#160;</font></div></td><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="padding-bottom:2px;padding-top:2px;text-align:center;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">(IRS Employer</font></div><div style="padding-bottom:2px;padding-top:2px;text-align:center;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">Identification No.)</font></div></td></tr></table></div></div><div style="line-height:120%;text-align:center;font-size:10pt;"><div style="padding-left:0px;text-indent:0px;line-height:normal;padding-top:10px;"><table cellpadding="0" cellspacing="0" style="font-family:Times New Roman;font-size:10pt;margin-left:auto;margin-right:auto;width:682px;border-collapse:collapse;text-align:left;"><tr><td colspan="3"></td></tr><tr><td style="width:333px;"></td><td style="width:16px;"></td><td style="width:333px;"></td></tr><tr><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="text-align:center;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;font-weight:bold;">100 Corporate Court</font></div></td><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="overflow:hidden;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">&#160;</font></div></td><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="overflow:hidden;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">&#160;</font></div></td></tr><tr><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="text-align:center;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;font-weight:bold;">South Plainfield, NJ</font></div></td><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="overflow:hidden;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">&#160;</font></div></td><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="text-align:center;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;font-weight:bold;">07080</font></div></td></tr><tr><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="text-align:center;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">(Address of Principal Executive Offices)</font></div></td><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="overflow:hidden;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">&#160;</font></div></td><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="text-align:center;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">(Zip Code)</font></div></td></tr></table></div></div><div style="line-height:174%;text-align:center;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">Company&#8217;s telephone number, including area code: </font><font style="font-family:inherit;font-size:10pt;font-weight:bold;">(908) 222-7000</font></div><div style="line-height:174%;text-align:center;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;font-weight:bold;">Not applicable</font></div><div style="line-height:174%;text-align:center;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">(Former Name or Former Address, if Changed Since Last Report)</font></div><div style="line-height:174%;text-align:center;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;"><br></font></div><div style="line-height:174%;text-align:left;text-indent:48px;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (</font><font style="font-family:inherit;font-size:10pt;font-style:italic;">see</font><font style="font-family:inherit;font-size:10pt;">&#32;General Instruction A.2. below):</font></div><div style="line-height:174%;text-align:left;text-indent:48px;font-size:10pt;"><font style="font-family:Wingdings;font-size:10pt;">o</font><font style="font-family:inherit;font-size:10pt;">&#32;</font><font style="font-family:inherit;font-size:5pt;">&#32;&#32;&#32;&#32;</font><font style="font-family:inherit;font-size:10pt;">&#32;Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)</font></div><div style="line-height:174%;text-align:left;text-indent:48px;font-size:10pt;"><font style="font-family:Wingdings;font-size:10pt;">o</font><font style="font-family:inherit;font-size:10pt;">&#32;</font><font style="font-family:inherit;font-size:5pt;">&#32;&#32;&#32;&#32;</font><font style="font-family:inherit;font-size:10pt;">&#32;Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)</font></div><div style="line-height:174%;text-align:left;text-indent:48px;font-size:10pt;"><font style="font-family:Wingdings;font-size:10pt;">o</font><font style="font-family:inherit;font-size:10pt;">&#32;</font><font style="font-family:inherit;font-size:5pt;">&#32;&#32;&#32;&#32;</font><font style="font-family:inherit;font-size:10pt;">&#32;Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))</font></div><div style="line-height:174%;text-align:left;text-indent:48px;font-size:10pt;"><font style="font-family:Wingdings;font-size:10pt;">o</font><font style="font-family:inherit;font-size:10pt;">&#32;</font><font style="font-family:inherit;font-size:5pt;">&#32;&#32;&#32;&#32;</font><font style="font-family:inherit;font-size:10pt;">&#32;Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))</font></div><div style="line-height:174%;text-align:left;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (&#167;230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (&#167;240.12b-2 of this chapter).</font></div><div style="line-height:174%;padding-bottom:16px;text-align:left;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">Emerging growth company&#160;&#160;&#160;&#160;</font><font style="font-family:Wingdings;font-size:10pt;">o</font></div><div style="line-height:174%;text-align:left;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act.&#160;&#160;&#160;&#160;  </font><font style="font-family:Wingdings;font-size:10pt;">o</font></div><div style="line-height:120%;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;"><br></font></div><div><br></div><div><div style="line-height:120%;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;"><br></font></div></div><hr style="page-break-after:always"><div><a name="s7396fb17cb414e0097bd62d581b64038"></a></div><div><div style="line-height:120%;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;"><br></font></div></div><div><br></div><div style="line-height:120%;padding-bottom:10px;padding-top:10px;text-align:left;-sec-extract:summary;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;font-weight:bold;">Item 8.01.     Other Events.</font></div><div style="line-height:120%;text-align:left;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">On October 5, 2018, supplemental information on the FIREFISH study that was the subject of the press release issued by PTC Therapeutics, Inc. on October 3, 2018, was presented by a principal investigator in the trial. As disclosed in the original press release, interim data showed a total of three babies sitting unassisted. On October 5, 2018, the principal investigator showed a video of an additional SMA type 1 baby sitting unassisted. The video also showed SMA type 1 babies from the FIREFISH trial demonstrating head control and rolling.</font></div><div style="line-height:120%;padding-bottom:10px;padding-top:10px;text-align:left;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">A copy of the press release issued on October 3, 2018, is attached to this Current Report on Form 8-K as Exhibit 99.1.</font></div><table cellpadding="0" cellspacing="0" style="font-family:Times New Roman; font-size:10pt;"><tr><td style="width:72px;"></td><td></td></tr><tr><td style="vertical-align:top"><div style="line-height:120%;font-size:10pt;padding-left:0px;"><font style="font-family:inherit;font-size:10pt;font-weight:bold;">Item 9.01. </font></div></td><td style="vertical-align:top;"><div style="line-height:120%;text-align:left;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;font-weight:bold;">Financial Statements and Exhibits.</font></div></td></tr></table><div style="line-height:120%;text-align:left;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;"><br></font></div><div style="line-height:120%;text-align:left;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">(d) Exhibits</font></div><div style="line-height:120%;text-align:left;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;"><br></font></div><div style="line-height:120%;text-align:center;font-size:10pt;"><div style="padding-left:0px;text-indent:0px;line-height:normal;padding-top:10px;"><table cellpadding="0" cellspacing="0" style="font-family:Times New Roman;font-size:10pt;margin-left:auto;margin-right:auto;width:95.8984375%;border-collapse:collapse;text-align:left;"><tr><td colspan="3"></td></tr><tr><td style="width:17%;"></td><td style="width:6%;"></td><td style="width:77%;"></td></tr><tr><td style="vertical-align:bottom;border-bottom:1px solid #000000;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="text-align:left;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;font-weight:bold;">Exhibit No.</font></div></td><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="text-align:left;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">&#160;</font></div></td><td style="vertical-align:bottom;border-bottom:1px solid #000000;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="text-align:center;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;font-weight:bold;">Description</font></div></td></tr><tr><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-top:1px solid #000000;"><div style="text-align:center;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">99.1</font></div></td><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="overflow:hidden;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">&#160;</font></div></td><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-top:1px solid #000000;"><div style="text-align:left;font-size:10pt;"><a style="text-decoration:underline;color:#0000FF;-sec-extract:exhibit;" href="a2018-10x03ptct2018wmssmax.htm"><font style="font-family:inherit;font-size:10pt;">Press Release, dated October 3, 2018 issued by PTC Therapeutics, Inc.</font></a></div></td></tr></table></div></div><div style="line-height:120%;text-align:center;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;"><br></font></div><div style="line-height:120%;text-align:center;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;font-weight:bold;">Signature</font></div><div style="line-height:120%;text-align:center;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;"><br></font></div><div style="line-height:120%;text-align:left;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this Report to be signed on its behalf by the undersigned hereunto duly authorized.</font></div><div style="line-height:120%;text-align:left;text-indent:33px;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;"><br></font></div><div style="line-height:120%;text-align:left;text-indent:33px;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;"><br></font></div><div style="line-height:120%;text-align:center;font-size:10pt;"><div style="padding-left:0px;text-indent:0px;line-height:normal;padding-top:10px;"><table cellpadding="0" cellspacing="0" style="font-family:Times New Roman;font-size:10pt;margin-left:auto;margin-right:auto;width:100%;border-collapse:collapse;text-align:left;"><tr><td colspan="3"></td></tr><tr><td style="width:56%;"></td><td style="width:6%;"></td><td style="width:38%;"></td></tr><tr><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="overflow:hidden;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">&#160;</font></div></td><td colspan="2" style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="text-align:left;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;font-weight:bold;">PTC Therapeutics, Inc.</font></div></td></tr><tr><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="overflow:hidden;height:18px;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">&#160;</font></div></td><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="overflow:hidden;height:18px;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">&#160;</font></div></td><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="overflow:hidden;height:18px;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">&#160;</font></div></td></tr><tr><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="text-align:left;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">Date: October 5, 2018</font></div></td><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="text-align:left;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">By:</font></div></td><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="text-align:left;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">/s/ Mark E. Boulding</font></div></td></tr><tr><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="overflow:hidden;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">&#160;</font></div></td><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="text-align:left;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">Name:</font></div></td><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-top:1px solid #000000;"><div style="text-align:left;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">Mark E. Boulding</font></div></td></tr><tr><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="overflow:hidden;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">&#160;</font></div></td><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="text-align:left;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">Title:</font></div></td><td style="vertical-align:bottom;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;"><div style="text-align:left;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">Executive Vice President and Chief Legal Officer</font></div></td></tr></table></div></div><div style="line-height:120%;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;"><br></font></div><div><br></div><div><div style="line-height:120%;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;"><br></font></div></div>	</body>
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<DOCUMENT>
<TYPE>EX-99.1
<SEQUENCE>2
<FILENAME>a2018-10x03ptct2018wmssmax.htm
<DESCRIPTION>EXHIBIT 99.1
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<div><a name="s1D2968500DC313760E8E46111F64C1A8"></a></div><div></div><div><br></div><div style="line-height:138%;text-align:left;"><img src="ptclogoimage.jpg" alt="ptclogoimage.jpg" style="height:86px;width:152px;"></div><div style="line-height:138%;text-align:center;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;"><br></font></div><div style="line-height:138%;text-align:center;font-size:14pt;"><font style="font-family:inherit;font-size:14pt;font-weight:bold;">Risdiplam Demonstrates Preliminary Evidence of Clinical Benefit in </font><font style="font-family:inherit;font-size:10pt;">&#32;<br></font><font style="font-family:inherit;font-size:14pt;font-weight:bold;">Type 1, 2, &amp; 3 Spinal Muscular Atrophy Patients</font></div><div style="line-height:138%;text-align:center;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;font-style:italic;">&#8211; Babies from FIREFISH Part 1 study showed increased survival &amp; gains in developmental milestones &#8211;</font></div><div style="line-height:138%;text-align:center;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;font-style:italic;">&#8211; SUNFISH Part 1 study demonstrated a 3.1-point increase in motor function as measured by MFM32 &#8211;</font></div><div style="line-height:138%;text-align:center;padding-left:24px;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;font-style:italic;">&#8211; Pivotal portion of SUNFISH Part 2 study in Type 2 &amp; 3 patients has completed enrollment &#8211; </font></div><div style="line-height:138%;text-align:center;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;"><br></font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;font-weight:bold;">SOUTH PLAINFIELD, N.J., Oct. 3, 2018 &#8211; </font><font style="font-family:inherit;font-size:11pt;">PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced interim clinical data from the Part 1, open-label studies of FIREFISH and SUNFISH demonstrating the benefit of risdiplam (RG7916) for the treatment of Type 1, 2 and 3 spinal muscular atrophy (SMA). The results showed that patients across all SMA types benefited from an oral systemic therapy indicated by increases in developmental motor milestones. Risdiplam was well tolerated at all doses across studies to date and no participants have withdrawn due to drug-related safety findings. The pivotal portions of both studies are ongoing. The data were presented at the 23rd International Annual Congress of the World Muscle Society in Argentina. The SMA program is a collaboration between PTC, the SMA Foundation, and Roche.</font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;"><br></font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">"The emerging results from FIREFISH and SUNFISH trials support the broad clinical benefit of a systemic oral treatment for SMA patients," said Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics. "Patients with Type 2 and 3 SMA typically decline over the course of a year and the increase in motor function by over 3 points in SUNFISH when compared to natural history is exceptionally encouraging. We are excited by the gains in developmental motor milestones exhibited by Type 1 babies in FIREFISH. The observation of six babies sitting to date in a dose finding study is remarkable. SMA is a systemic disease, and risdiplam which is an oral treatment that reaches all affected organs has the potential to be a best-in-class therapy.&#8221;</font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;"><br></font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">In Part 1 of the FIREFISH study, at Day 245 of treatment, 43% (6/14) of infants were able to sit (with or without support), including three who achieved unassisted sitting. Natural history indicates that Type 1 SMA babies never achieve this milestone. Ninety percent of babies remain alive with two having discontinued due to the fatal progression of their disease. In Part 1 of the SUNFISH study in Type 2 and 3 SMA patients, 63% (19/30) of patients treated with risdiplam for at least one year achieved a median increase in motor function (as measured by MFM32) of 3.13 points versus baseline. Typically patients with Type 2 or 3 SMA decline by 0.85 to 0.67 point per year. In addition, SMN protein median increases of greater than 2-fold were sustained over 12 months.  </font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;"><br></font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">The pivotal portion of the SUNFISH clinical study has completed enrollment. Part 2 of SUNFISH is a randomized, double-blinded, multi-center, placebo-controlled study which enrolled 180 Type 2 and Type </font></div><div><br></div><div></div><hr style="page-break-after:always"><div></div><div><br></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">3 SMA patients 2-25 years of age for 24 months, followed by an open-label extension. Patients enrolled in the SUNFISH trial have a broad age range (2-24 years; median age 8 years) and with broad functional characteristics. The primary endpoint is change from baseline in the total Motor Function Measure 32 (MFM-32) score at Month 12.</font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;"><br></font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">Risdiplam is an investigational small molecule SMN2 splicing modifier targeting the survival motor neuron 2 (SMN2) RNA, restoring a functional transcript. In preclinical studies, risdiplam, which was given orally, crossed the blood brain barrier, and showed systemic distribution to the organs that are affected by low levels of SMN protein. </font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">The table below depicts improvements across the FIREFISH and SUNFISH dose-finding studies: </font></div><div style="line-height:120%;font-size:11pt;"><div style="padding-left:0px;text-indent:0px;line-height:normal;padding-top:10px;"><table cellpadding="0" cellspacing="0" style="font-family:Times New Roman;font-size:10pt;width:98.07692307692307%;border-collapse:collapse;text-align:left;"><tr><td colspan="2"></td></tr><tr><td style="width:24%;"></td><td style="width:76%;"></td></tr><tr><td colspan="2" style="vertical-align:top;border-bottom:1px solid #00000a;background-color:#e7e6e6;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-left:1px solid #00000a;border-right:1px solid #00000a;border-top:1px solid #00000a;"><div style="text-align:center;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;font-weight:bold;">FIREFISH</font></div></td></tr><tr><td style="vertical-align:top;border-bottom:1px solid #00000a;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-left:1px solid #00000a;border-right:1px solid #00000a;border-top:1px solid #00000a;"><div style="text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">Outcome</font></div></td><td style="vertical-align:top;border-bottom:1px solid #00000a;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-left:1px solid #00000a;border-right:1px solid #00000a;border-top:1px solid #00000a;"><div style="text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">Result</font></div></td></tr><tr><td style="vertical-align:top;border-bottom:1px solid #00000a;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-left:1px solid #00000a;border-right:1px solid #00000a;border-top:1px solid #00000a;"><div style="text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">Sitting after 8 months of treatment (n=14)</font></div></td><td style="vertical-align:top;border-bottom:1px solid #00000a;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-left:1px solid #00000a;border-right:1px solid #00000a;border-top:1px solid #00000a;"><div style="padding-left:10px;text-indent:-11px;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">&#8226;</font><font style="font-family:inherit;font-size:10pt;">&#160;&#160;&#160;&#160;</font><font style="font-family:inherit;font-size:11pt;">6 infants (43%) sit with or without support including 3 infants (21%) who sit without assistance</font></div></td></tr><tr><td style="vertical-align:top;border-bottom:1px solid #00000a;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-left:1px solid #00000a;border-right:1px solid #00000a;border-top:1px solid #00000a;"><div style="text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">Motor milestone achievement after 8 months of treatment (n=14)</font></div></td><td style="vertical-align:top;border-bottom:1px solid #00000a;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-left:1px solid #00000a;border-right:1px solid #00000a;border-top:1px solid #00000a;"><div style="padding-left:10px;text-indent:-11px;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">&#8226;</font><font style="font-family:inherit;font-size:10pt;">&#160;&#160;&#160;&#160;</font><font style="font-family:inherit;font-size:11pt;">10 infants (71%) achieved full or partial head control including 6 (43%) babies with upright head control</font></div><div style="padding-left:10px;text-indent:-11px;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">&#8226;</font><font style="font-family:inherit;font-size:10pt;">&#160;&#160;&#160;&#160;</font><font style="font-family:inherit;font-size:11pt;">7 infants (50%) kicking</font></div><div style="padding-left:10px;text-indent:-11px;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">&#8226;</font><font style="font-family:inherit;font-size:10pt;">&#160;&#160;&#160;&#160;</font><font style="font-family:inherit;font-size:11pt;">4 infants (29%) demonstrated rolling to the side</font></div></td></tr><tr><td style="vertical-align:top;border-bottom:1px solid #00000a;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-left:1px solid #00000a;border-right:1px solid #00000a;border-top:1px solid #00000a;"><div style="text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">Survival</font></div></td><td style="vertical-align:top;border-bottom:1px solid #00000a;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-left:1px solid #00000a;border-right:1px solid #00000a;border-top:1px solid #00000a;"><div style="padding-left:10px;text-indent:-11px;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">&#8226;</font><font style="font-family:inherit;font-size:10pt;">&#160;&#160;&#160;&#160;</font><font style="font-family:inherit;font-size:11pt;">90% (19/21) babies were event-free vs. 50% of age-matched SMA Type 1 babies in natural history studies</font></div></td></tr><tr><td style="vertical-align:top;border-bottom:1px solid #00000a;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-left:1px solid #00000a;border-right:1px solid #00000a;border-top:1px solid #00000a;"><div style="text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">Pulmonary function and swallowing</font></div></td><td style="vertical-align:top;border-bottom:1px solid #00000a;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-left:1px solid #00000a;border-right:1px solid #00000a;border-top:1px solid #00000a;"><div style="padding-left:10px;text-indent:-11px;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">&#8226;</font><font style="font-family:inherit;font-size:10pt;">&#160;&#160;&#160;&#160;</font><font style="font-family:inherit;font-size:11pt;">No baby has required tracheostomy, reached permanent ventilation or lost the ability to swallow (Latest visit day - 546 days)</font></div></td></tr><tr><td style="vertical-align:top;border-bottom:1px solid #00000a;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-left:1px solid #00000a;border-right:1px solid #00000a;border-top:1px solid #00000a;"><div style="text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">CHOP-INTEND</font></div></td><td style="vertical-align:top;border-bottom:1px solid #00000a;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-left:1px solid #00000a;border-right:1px solid #00000a;border-top:1px solid #00000a;"><div style="padding-left:10px;text-indent:-11px;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">&#8226;</font><font style="font-family:inherit;font-size:10pt;">&#160;&#160;&#160;&#160;</font><font style="font-family:inherit;font-size:11pt;">Increases over time &#8211; from 5.5 points median at Day 56 (n=20) to 16 points median at Day 245 (n=14)</font></div><div style="padding-left:10px;text-indent:-11px;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">&#8226;</font><font style="font-family:inherit;font-size:10pt;">&#160;&#160;&#160;&#160;</font><font style="font-family:inherit;font-size:11pt;">Increases in younger and older patients &#8211; 18.5 points median for babies 3-5 months old (n=4) and 14.5 points median for babies 5-7 months old (n=10) at Day 245</font></div><div style="padding-left:10px;text-indent:-11px;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">&#8226;</font><font style="font-family:inherit;font-size:10pt;">&#160;&#160;&#160;&#160;</font><font style="font-family:inherit;font-size:11pt;">13 infants (93%) achieved &#8805;4-point increase (n=14) at Day 245</font></div><div style="padding-left:10px;text-indent:-11px;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">&#8226;</font><font style="font-family:inherit;font-size:10pt;">&#160;&#160;&#160;&#160;</font><font style="font-family:inherit;font-size:11pt;">8 infants (57%) achieved a score of 40 or above (n=14) at Day 245</font></div></td></tr></table></div></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">The most common adverse events were fever (pyrexia: 52.4%,) diarrhea (26.8%), upper respiratory tract infections (19%), ear infections (14.3%), pneumonia (14.3%), constipation (14.3%), vomiting (14.3%), cough (14.3%) and upper respiratory inflammation (14.3%). Data cutoff: 7-Sept 2018</font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;"><br></font></div><div style="line-height:120%;font-size:11pt;"><div style="padding-left:0px;text-indent:0px;line-height:normal;padding-top:10px;"><table cellpadding="0" cellspacing="0" style="font-family:Times New Roman;font-size:10pt;width:99.78632478632478%;border-collapse:collapse;text-align:left;"><tr><td colspan="4"></td></tr><tr><td style="width:38%;"></td><td style="width:20%;"></td><td style="width:21%;"></td><td style="width:21%;"></td></tr><tr><td colspan="4" style="vertical-align:top;border-bottom:1px solid #00000a;background-color:#e7e6e6;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-left:1px solid #00000a;border-right:1px solid #00000a;border-top:1px solid #00000a;"><div style="text-align:center;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;font-weight:bold;">SUNFISH</font></div></td></tr><tr><td style="vertical-align:top;border-bottom:1px solid #00000a;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-left:1px solid #00000a;border-right:1px solid #00000a;border-top:1px solid #00000a;"><div style="text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">Endpoint:  <br>(at 12 months of treatment)</font></div></td><td colspan="3" style="vertical-align:top;border-bottom:1px solid #00000a;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-left:1px solid #00000a;border-right:1px solid #00000a;border-top:1px solid #00000a;"><div style="text-align:center;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">&gt;12 months Treatment</font></div></td></tr><tr><td style="vertical-align:top;border-bottom:1px solid #00000a;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-left:1px solid #00000a;border-right:1px solid #00000a;border-top:1px solid #00000a;"><div style="text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">MFM</font></div></td><td style="vertical-align:top;border-bottom:1px solid #00000a;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-left:1px solid #00000a;border-right:1px solid #00000a;border-top:1px solid #00000a;"><div style="text-align:center;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">All patients <br>(n=30) *</font></div></td><td style="vertical-align:top;border-bottom:1px solid #00000a;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-left:1px solid #00000a;border-right:1px solid #00000a;border-top:1px solid #00000a;"><div style="text-align:center;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">Aged 2-11 <br>(n=17)</font></div></td><td style="vertical-align:top;border-bottom:1px solid #00000a;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-left:1px solid #00000a;border-right:1px solid #00000a;border-top:1px solid #00000a;"><div style="text-align:center;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">Aged 12-24 <br>(n=13)</font></div></td></tr><tr><td style="vertical-align:top;border-bottom:1px solid #00000a;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-left:1px solid #00000a;border-right:1px solid #00000a;border-top:1px solid #00000a;"><div style="text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">Total MFM change from baseline, mean (SD)</font></div></td><td style="vertical-align:top;border-bottom:1px solid #00000a;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-left:1px solid #00000a;border-right:1px solid #00000a;border-top:1px solid #00000a;"><div style="text-align:center;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">2.47 (4.17)</font></div></td><td style="vertical-align:top;border-bottom:1px solid #00000a;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-left:1px solid #00000a;border-right:1px solid #00000a;border-top:1px solid #00000a;"><div style="text-align:center;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">3.31 (4.5)</font></div></td><td style="vertical-align:top;border-bottom:1px solid #00000a;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-left:1px solid #00000a;border-right:1px solid #00000a;border-top:1px solid #00000a;"><div style="text-align:center;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">1.36 (3.57)</font></div></td></tr><tr><td style="vertical-align:top;border-bottom:1px solid #00000a;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-left:1px solid #00000a;border-right:1px solid #00000a;border-top:1px solid #00000a;"><div style="text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">Total MFM change from baseline, median (range)</font></div></td><td style="vertical-align:top;border-bottom:1px solid #00000a;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-left:1px solid #00000a;border-right:1px solid #00000a;border-top:1px solid #00000a;"><div style="text-align:center;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">3.13 (-7.3-11.5)</font></div></td><td style="vertical-align:top;border-bottom:1px solid #00000a;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-left:1px solid #00000a;border-right:1px solid #00000a;border-top:1px solid #00000a;"><div style="text-align:center;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">4.17 (-6.3-11.5)</font></div></td><td style="vertical-align:top;border-bottom:1px solid #00000a;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-left:1px solid #00000a;border-right:1px solid #00000a;border-top:1px solid #00000a;"><div style="text-align:center;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">2.08 (-7.3-5.2)</font></div></td></tr><tr><td style="vertical-align:top;border-bottom:1px solid #00000a;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-left:1px solid #00000a;border-right:1px solid #00000a;border-top:1px solid #00000a;"><div style="text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">Proportion of patients who achieve improvement (i.e. a change from baseline in MFM score &#8805;3), % (n)</font></div></td><td style="vertical-align:top;border-bottom:1px solid #00000a;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-left:1px solid #00000a;border-right:1px solid #00000a;border-top:1px solid #00000a;"><div style="text-align:center;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">63.3 (19/30)</font></div></td><td style="vertical-align:top;border-bottom:1px solid #00000a;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-left:1px solid #00000a;border-right:1px solid #00000a;border-top:1px solid #00000a;"><div style="text-align:center;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">76.5 (13/17)</font></div></td><td style="vertical-align:top;border-bottom:1px solid #00000a;padding-left:2px;padding-top:2px;padding-bottom:2px;padding-right:2px;border-left:1px solid #00000a;border-right:1px solid #00000a;border-top:1px solid #00000a;"><div style="text-align:center;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">46.2 (6/13)</font></div></td></tr></table></div></div><div><br></div><div></div><hr style="page-break-after:always"><div></div><div><br></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">*Excludes 4 patients who performed the MFM20 assessment (only patients who performed the full MFM32 assessment are included in the analysis) and one patient who had dropped out of the study prior to the Month 12 visit. Serious adverse events that occurred in two or more of the 51 patients were nausea (4%), upper respiratory tract infection (4%), and vomiting (4%). Data cut-off 6</font><font style="font-family:inherit;font-size:11pt;"><sup style="vertical-align:top;line-height:120%;font-size:7pt">th</sup></font><font style="font-family:inherit;font-size:11pt;">&#32;July 2018</font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;"><br></font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;"><br></font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">For more information on the Congress, visit </font><font style="font-family:inherit;font-size:11pt;color:#0000ff;text-decoration:underline;">http://www.wms2018.com/</font><font style="font-family:inherit;font-size:11pt;">&#32;</font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;"><br></font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;font-weight:bold;">About Spinal Muscular Atrophy (SMA)</font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">Spinal muscular atrophy (SMA) is a genetic neuromuscular disorder that is the leading genetic cause of mortality in infants and toddlers caused by a missing or defective survival of motor neuron 1 (SMN1) gene, which results in reduced levels of SMN protein. The homologous SMN2 pre-mRNA is predominantly spliced to a truncated mRNA, and only produces small amounts of functional SMN protein. Insufficient levels of SMN protein are responsible for the loss of motor neurons within the spinal cord leading to muscle atrophy and death in its most severe form. It is estimated that this devastating disease affects 1 in every 11,000 children born.</font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;"><br></font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;font-weight:bold;">About the SMA Clinical Trials</font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;font-weight:bold;">FIREFISH: </font><font style="font-family:inherit;font-size:11pt;">An open-label, two-part clinical trial. Part 1 was a dose escalation study in 21 infants. The primary objective of Part 1 was to assess the safety profile of risdiplam in infants and determine the dose for Part 2. Part 2 is a single-arm study with the dose selected in Part 1 in approximately 40 infants with Type 1 SMA for 24 months, followed by an open-label extension. This study is recruiting globally.</font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;"><br></font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;font-weight:bold;">SUNFISH: </font><font style="font-family:inherit;font-size:11pt;">A double&#8208;blind, two&#8208;part, placebo&#8208;controlled trial. Part 1 enrolled patients with Type 2 or 3 SMA to evaluate the safety, tolerability, and PK/PD of several risdiplam dose levels. The pivotal SUNFISH Part 2, in non&#8208;ambulant patients with Type 2 or 3 SMA, is evaluating safety and efficacy of the risdiplam dose level selected from Part 1 for 24 months, followed by an open label extension. This study has finished recruiting globally.</font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;"><br></font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;font-weight:bold;">JEWELFISH: </font><font style="font-family:inherit;font-size:11pt;">An ongoing, exploratory, open-label study to establish the safety and tolerability of risdiplam in people with SMA who have previously participated in a study with another therapy targeting SMN2 splicing.</font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;"><br></font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;font-weight:bold;">About the SMA collaboration</font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">The SMA program was initiated by PTC Therapeutics in partnership with the SMA Foundation in 2006. In November 2011, Roche gained an exclusive worldwide license to the PTC/SMA Foundation SMN2 alternative splicing program. The development of risdiplam RG7916 is being executed globally by Roche, including in the US through Genentech, a member of the Roche group. The SMA program is overseen by a Joint Steering Committee with members from PTC, Roche, and the SMA Foundation.</font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;"><br></font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;background-color:#ffffff;font-weight:bold;">About PTC Therapeutics, Inc.</font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">PTC is a science-led, global biopharmaceutical company focused on the discovery, development and commercialization of clinically-differentiated medicines that provide benefits to patients with rare </font></div><div><br></div><div></div><hr style="page-break-after:always"><div></div><div><br></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">disorders. Founded 20 years ago, PTC Therapeutics has successfully launched two rare disorder products and has a global commercial footprint. This success is the foundation that drives investment in a robust pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need.</font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;"><br></font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;background-color:#ffffff;font-weight:bold;">For More Information: </font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;"><br></font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;background-color:#ffffff;font-weight:bold;">Investors: </font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;background-color:#ffffff;">Emily Hill</font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;background-color:#ffffff;">+1 (908) 912-9327</font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;background-color:#ffffff;text-decoration:underline;">ehill@ptcbio.com</font><font style="font-family:inherit;font-size:11pt;background-color:#ffffff;">&#32;</font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;"><br></font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;background-color:#ffffff;font-weight:bold;">Media: </font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;background-color:#ffffff;">Jane Baj</font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;background-color:#ffffff;">+1 (908) 912-9167</font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;background-color:#ffffff;text-decoration:underline;">jbaj@ptcbio.com</font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;"><br></font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;font-weight:bold;">Forward-Looking Statements:</font></div><div style="line-height:138%;padding-bottom:13px;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">All statements, other than those of historical fact, contained in this press release, are forward-looking statements, including statements regarding: any advancement of the joint development program in SMA with PTC, Roche, and SMAF, in particular as related to the timing of enrollment, completion and evaluation of the Phase 2 clinical studies of risdiplam in SMA patients and the period during which the results of the studies will become available; the clinical utility and potential advantages of risdiplam, including its potential to impact every aspect of the disease; the timing and outcome of PTC's regulatory strategy and process; PTC's strategy, future expectations, plans and prospects, future operations, future financial position, future revenues or projected costs; and the objectives of management. Other forward-looking statements may be identified by the words "potential," "will," "promise," "expect," "plan," "target," "anticipate," "believe," "estimate," "intend," "may," "project," "possible," "would," "could," "should," "continue," and similar expressions.</font></div><div style="line-height:138%;padding-bottom:13px;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the initiation, enrollment, conduct and availability of data from either the SUNFISH or FIREFISH studies and the outcome of such studies; events during, or as a result of, these studies that could delay or prevent further development of risdiplam, including future actions or activities under the SMA joint development program; our expectations for regulatory approvals; PTC's scientific approach and general development progress; and the factors discussed in the "Risk Factors" sections of PTC's most recent Quarterly Report on Form 10-Q and Annual Report on Form 10-K as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors.</font></div><div style="line-height:138%;padding-bottom:13px;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products, including with respect to PTC's joint development </font></div><div><br></div><div></div><hr style="page-break-after:always"><div></div><div><br></div><div style="line-height:138%;padding-bottom:13px;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">program in SMA with Roche and the SMAF. There are no guarantees that any product candidate under the joint development program will receive regulatory approval in any territory or prove to be commercially successful.</font></div><div style="line-height:138%;padding-bottom:13px;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;">The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law.</font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;"><br></font></div><div style="line-height:138%;text-align:left;font-size:11pt;"><font style="font-family:inherit;font-size:11pt;"><br></font></div><div><br></div><div></div>	</body>
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end
</TEXT>
</DOCUMENT>
</SEC-DOCUMENT>
