Document


UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934
Date of Report (Date of earliest event reported): August 1, 2018
PTC THERAPEUTICS, INC.
(Exact Name of Company as Specified in Charter)

Delaware
 
001-35969
 
04-3416587
(State or Other Jurisdiction
of Incorporation)
 
(Commission
File Number)
 
(IRS Employer
Identification No.)
100 Corporate Court
 
 
South Plainfield, NJ
 
07080
(Address of Principal Executive Offices)
 
(Zip Code)
Company’s telephone number, including area code: (908) 222-7000
Not applicable
(Former Name or Former Address, if Changed Since Last Report)

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 (see General Instruction A.2. below):
o Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
o Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
o Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
o Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))
Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).
Emerging growth company    o
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.     o






Item 1.01. Entry into a Material Definitive Agreement.

On August 1, 2018 (the “Effective Date”), PTC Therapeutics International Limited (“PTC”), a subsidiary of PTC Therapeutics, Inc. (the “Company”), entered into a Collaboration and License Agreement (the “Agreement”) with Akcea Therapeutics, Inc. (“Akcea”), for the commercialization by PTC of TEGSEDITM (inotersen), WAYLIVRA™ (volanesorsen) and products containing those compounds (collectively, the “Products”) in countries in Latin America and the Caribbean (the “PTC Territory”). TEGSEDI is an antisense oligonucleotide inhibitor of human transthyretin production, or TTR protein, for the treatment of patients with hereditary transthyretin amyloidosis (hATTR amyloidosis), a severe, rare and fatal genetic disease. WAYLIVRA is an antisense drug candidate in development for two rare metabolic disorders: familial chylomicronemia syndrome (FCS) and familial partial lipodystrophy (FPL). Neither TEGSEDI nor WAYLIVRA is currently approved for marketing in the PTC Territory. In addition, Akcea has granted to PTC a right of first negotiation (“ROFN”) to commercialize AKCEA-TTR-Lrx, a follow-on product candidate to inotersen, on an exclusive basis in the PTC Territory.

Under the terms of the Agreement, Akcea has granted to PTC an exclusive right and license, with the right to grant certain sublicenses, under Akcea’s product-specific intellectual property to develop, manufacture and commercialize the Products in the PTC Territory. In addition, Akcea has granted to PTC a non-exclusive right and license, with the right to grant certain sublicenses, under Akcea’s core intellectual property and manufacturing intellectual property to develop, manufacture and commercialize the Products in the PTC Territory and to manufacture the Products worldwide in accordance with a supply agreement with Akcea. Akcea has in-licensed certain of the Akcea intellectual property from its affiliate, Ionis Pharmaceuticals, Inc. (“Ionis”). Each party has agreed not to, independently or with any third party, commercialize any competing oligonucleotide product in the PTC Territory for the same gene target as inotersen.

Within 30 days after the effective date, Akcea has agreed to assign and transfer to PTC the ownership and sponsorship of applicable regulatory approvals in countries in the PTC Territory, after which PTC has agreed to prepare, file and maintain regulatory filings and approvals for the applicable Products in such countries. After the Effective Date, PTC is responsible for all meetings, communications and other interactions with regulatory authorities in the PTC Territory.

PTC has agreed to pay to Akcea an upfront licensing fee of $18 million, consisting of a payment of $12 million within ten business days after the Effective Date, and $6 million within 30 days after receipt of regulatory approval of WAYLIVRA from the United States Food and Drug Administration or the European Medicines Agency, whichever occurs earlier. In addition, Akcea is eligible to receive milestone payments, on a Product-by-Product basis, of $4 million upon receipt of regulatory approval for a Product from ANVISA, the Brazilian Health Regulatory Authority, subject to a maximum aggregate amount of $8 million for all such Products. Akcea is also entitled to receive royalty payments in the mid-twenty percent range of net sales on a country-by-country and Product-by-Product basis, commencing on the earlier to occur of (1) 12 months after the first commercial sale of such Product in Brazil or (2) the date when PTC, its affiliates or sublicensees have recognized revenue of $10 million or more in cumulative net sales for such Product in the PTC Territory. The royalty payments are subject to reduction in certain circumstances as set forth in the Agreement.

Akcea has granted to PTC a ROFN to commercialize AKCEA-TTR-Lrx in the PTC Territory, subject to negotiation of the terms of a definitive agreement and certain other terms and conditions. Such a definitive agreement would provide for a royalty rate to be paid by PTC for AKCEA-TTR-Lrx equal to the royalty rate PTC has agreed to pay for TEGSEDI under the Agreement, or in the mid-twenty percent range of net sales, and the term of such royalty payments would be the same as the term of the TEGSEDI royalty payments. During a specified period in the Agreement, neither Akcea nor Ionis may enter into an agreement or grant any license to AKCEA-TTR-Lrx that is inconsistent with PTC’s ROFN.
 
The activities of the parties pursuant to the Agreement will be overseen by a Joint Steering Committee, to be composed of an equal number of representatives appointed by each of PTC and Akcea.

The Agreement continues until the expiration of the last to expire royalty term with respect to all Products in all countries in the PTC Territory. Either party may terminate the Agreement on written notice to the other party if such other party is in material breach of its obligations thereunder and has not cured such breach within 30 days after notice in the case of a payment breach or 60 days after notice in the case of any other breach.

The foregoing description of the Agreement is a summary only and is qualified in its entirety by reference to the terms of the Agreement, a copy of which will be filed with the Company’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2018.








Item 7.01.    Regulation FD Disclosure.
 
On August 2, 2018, the Company issued a press release in which it announced that it entered into the Agreement. A copy of the press release is attached to this Report as Exhibit 99.1 and is incorporated by reference into this Item 7.01.
 
The information set forth in or incorporated by reference into this Item 7.01, including Exhibit 99.1, shall not be deemed to be “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liability of that section, and shall not be incorporated by reference into any registration statement or other document filed under the Securities Act or the Exchange Act, except as shall be expressly set forth by specific reference in such filing.

Item 9.01.
Financial Statements and Exhibits.

(d) Exhibits

Exhibit No.
 
Description
99.1
 

Cautionary Statement Concerning Forward Looking Statements
 
This Report contains forward-looking statements addressing the Collaboration and Licensing Agreement and any other statements about future expectations, prospects, estimates and other matters that are dependent upon future events or developments. All statements, other than those of historical fact, contained in this Report are forward-looking statements, including statements related to the Company’s expectations with respect to the Collaboration and Licensing Agreement; the potential financial impact and benefits to the Company of the Collaboration and Licensing Agreement, including with respect to the timing of regulatory approval of TEGSEDI and WAYLIVRA in countries in the PTC Territory, the commercialization of TEGSEDI and WAYLIVRA, and the Company’s expectations with respect to contingent payments to Akcea based on net sales and the potential achievement of regulatory milestones; the future expectations, plans and prospects for the Company; the Company’s strategy, 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 “look forward”, “plan,” “anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,” “target,” “potential,” “will,” “would,” “could,” “should,” “continue,” and similar expressions.  The Company’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 the factors discussed in the “Risk Factors” section of the Company’s most recent Quarterly Report on Form 10-Q or Annual Report on Form 10-K as well as any updates to these risk factors filed from time to time in the Company’s other filings with the SEC.   As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product candidate will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including TEGSEDI or WAYLIVRA, or any other product candidates.  The forward-looking statements contained herein represent the Company’s views only as of the date of this Report and the Company 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 Report except as required by law. All website addresses given in this Report or incorporated herein by reference are for information only and are not intended to be an active link or to incorporate any website information into this Report.

Signature

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.


 
PTC Therapeutics, Inc.
 
 
 
Date: August 2, 2018
By:
/s/ Mark E. Boulding
 
Name:
Mark E. Boulding
 
Title:
Executive Vice President and Chief Legal Officer


Exhibit


http://api.tenkwizard.com/cgi/image?quest=1&rid=23&ipage=12384833&doc=3
 
http://api.tenkwizard.com/cgi/image?quest=1&rid=23&ipage=12384833&doc=4
                        
Akcea Therapeutics and PTC Therapeutics Collaborate to Commercialize
Two Rare Disease Drugs in Latin America
- PTC in-licenses regional rights to TEGSEDI™ and WAYLIVRA™ from Akcea -

South Plainfield, N.J. and Cambridge, M.A., August 2, 2018 – PTC Therapeutics, Inc. (NASDAQ: PTCT), and Akcea Therapeutics, Inc. (NASDAQ: AKCA), an affiliate of Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), today announced a collaboration under which PTC will commercialize two of Akcea’s rare disease drugs in Latin America (LATAM): TEGSEDI™ (inotersen) and WAYLIVRA™ (volanesorsen).

TEGSEDI has received marketing authorization approval from the European Commission (EC) for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR amyloidosis). TEGSEDI is also the subject of a pending new drug application in the U.S. and Canada. TEGSEDI has a PDUFA date of October 6, 2018. WAYLIVRA is under regulatory review in the U.S., Europe and Canada for the treatment of people with familial chylomicronemia syndrome (FCS). WAYLIVRA recently received a positive vote from the FDA's Division of Metabolism and Endocrinology Products Advisory Committee and has a PDUFA date of August 30, 2018. WAYLIVRA is also in clinical development for Familial Partial Lipodystrophy, or FPL.

“This collaboration reflects our strategic initiative to leverage PTC's global infrastructure and reflects our successes in bringing innovative drugs to patients in Latin America,” said Stuart W. Peltz, Ph.D., chief executive officer, PTC Therapeutics. “We are excited to work with Akcea as part of the global launch and commercialization of TEGSEDI and WAYLIVRA.”

“One of the key mutations that leads to hATTR amyloidosis occurs more frequently in individuals of Portuguese descent,” stated Marcio Souza, chief operating officer, PTC Therapeutics, Inc. “There are approximately 6,000 patients with polyneuropathic hATTR amyloidosis in Latin America, making it a strategically important region for TEGSEDI. We are well positioned to file for registration in key Latin American countries in the short term.”

“Our decision to partner with PTC to accelerate commercial access for patients in Latin America reflects our commitment to bringing TEGSEDI and WAYLIVRA to patients as rapidly as possible,” said Paula Soteropoulos, chief executive officer, Akcea Therapeutics. “PTC’s established rare disease team in Latin America has a proven record of success in patient identification, in physician and patient education and support programs and in efficiently obtaining market access. PTC’s patient-focused approach for rare diseases aligns with ours and will be important as we bring TEGSEDI and WAYLIVRA to patients in this region.”


 
 
 




Key Transaction Details
Under the agreement, PTC will gain exclusive rights to TEGSEDI and WAYLIVRA in Latin America and certain Caribbean countries. PTC will pay Akcea an upfront licensing fee of $18 million, $12 million which is due on signing and $6 million which will be paid on the earlier of FDA or EMA approval of WAYLIVRA. In addition, PTC will pay Akcea regulatory milestones in the PTC territory up to a total of $8 million. Akcea is also eligible to receive royalties from PTC in the mid-twenty percent range on net sales of each drug in the PTC territory. PTC’s obligation to pay Akcea royalties begins on the earlier of 12 months after the first commercial sale of a product in Brazil or the date that PTC recognizes revenue of at least $10 million in LATAM. The collaboration will be governed by a joint steering committee with representation from both parties. Akcea gained the global rights to TEGSEDI and WAYLIVRA through licenses from Ionis. Milestone payments and royalties that Akcea receives from PTC for TEGSEDI will be split 60% to Ionis and 40% to Akcea. All WAYLIVRA milestone payments and royalties that Akcea receives from PTC will be split 50/50 with Ionis.

About TEGSEDITM (inotersen)
TEGSEDITM (inotersen), a product of Ionis’ proprietary antisense technology, is an antisense oligonucleotide (ASO) inhibitor of human transthyretin (TTR) production and was developed by Ionis. TEGSEDI is the world’s first RNA-targeted therapeutic to treat patients with hATTR amyloidosis. TEGSEDI is approved in the E.U. for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR) and is currently under regulatory review in the U.S. and Canada. For important safety information for TEGSEDI, including method of administration, special warnings, drug interactions and adverse drug reactions, please see the European Summary of Product Characteristics (SmPC), available from the EMA website at www.ema.europa.eu.

About hereditary transthyretin (hATTR) amyloidosis
hATTR amyloidosis is a progressive, systemic and fatal inherited disease caused by the abnormal formation of the TTR protein and aggregation of TTR amyloid deposits in various tissues and organs throughout the body, including in peripheral nerves, heart, intestinal tract, eyes, kidneys, central nervous system, thyroid and bone marrow. The progressive accumulation of TTR amyloid deposits in these tissues and organs leads to sensory, motor and autonomic dysfunction often having debilitating effects on multiple aspects of a patient's life. Patients with hATTR amyloidosis often present with a mixed phenotype and experience overlapping symptoms of polyneuropathy and cardiomyopathy.

Ultimately, hATTR amyloidosis results in death within three to fifteen years of symptom onset. Therapeutic options for the treatment of patients with hATTR amyloidosis are limited and there are currently no disease-modifying drugs approved for the disease. There are an estimated 50,000 patients with hATTR amyloidosis worldwide. Additional information on hATTR amyloidosis, including a full list of organizations supporting the hATTR amyloidosis community worldwide, is available at www.hattrchangethecourse.com.
 
About WAYLIVRATM (volanesorsen)
WAYLIVRATM, a product of Ionis’ proprietary antisense technology, is under regulatory review

 
 
 




in the U.S., E.U. and Canada as a treatment for familial chylomicronemia syndrome (FCS). The U.S. and E.U. regulatory agencies have granted Orphan Drug Designation to WAYLIVRA for the treatment of FCS. If approved, WAYLIVRA would be the first and only therapy indicated for people with FCS.

About Familial Chylomicronemia Syndrome (FCS)
FCS is an ultra-rare disease caused by impaired function of the enzyme lipoprotein lipase (LPL) and characterized by severe hypertriglyceridemia (>880mg/dL) and a risk of unpredictable and potentially fatal acute pancreatitis. Because of limited LPL function, people with FCS cannot breakdown chylomicrons, lipoprotein particles that are 90% triglycerides. In addition to pancreatitis, FCS patients are at risk of chronic complications due to permanent organ damage. They can experience daily symptoms including abdominal pain, generalized fatigue and impaired cognitions that affect their ability to work. People with FCS also report major emotional and psychosocial effects including anxiety, social withdrawal, depression and brain fog. There is no effective therapy for FCS currently available. Additional information on FCS is available at www.fcsfocus.com, and through the FCS Foundation at http://www.livingwithfcs.org and the LPLD Alliance at www.lpldalliance.org. For a full list of organizations supporting the FCS community worldwide, please click here.

About Akcea Therapeutics, Inc.
Akcea Therapeutics, Inc., an affiliate of Ionis Pharmaceuticals, Inc., is a biopharmaceutical company focused on developing and commercializing drugs to treat patients with serious and rare diseases. Akcea is advancing a mature pipeline of six novel drugs, including TEGSEDITM (inotersen), WAYLIVRATM (volanesorsen), AKCEA-APO(a)-LRx, AKCEA-ANGPTL3-LRx, AKCEA-APOCIII-LRx, and AKCEA-TTR-LRx, all with the potential to treat multiple diseases. All six drugs were discovered by and are being co-developed with Ionis, a leader in antisense therapeutics, and are based on Ionis’ proprietary antisense technology. TEGSEDI is approved in the E.U. for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR) and is currently under regulatory review in the U.S. and Canada. WAYLIVRA is under regulatory review in the U.S., E.U. and Canada for the treatment of familial chylomicronemia syndrome, or FCS, and is currently in Phase 3 clinical development for the treatment of people with familial partial lipodystrophy, or FPL. Akcea is building the infrastructure to commercialize its drugs globally. Akcea is a global company headquartered in Cambridge, Massachusetts. Additional information about Akcea is available at www.akceatx.com.

About PTC Therapeutics
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 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 PTC's mission to provide access to best-in-class treatments for patients who have an unmet medical need.

PTC’S and AKCEA’S Forward-Looking Statement

 
 
 




This press release includes forward-looking statements regarding the business of PTC Therapeutics, Inc. and Akcea Therapeutics, Inc. and the therapeutic and commercial potential of TEGSEDITM (inotersen) and WAYLIVRATM (volanesorsen) and other products in development. Any statement describing PTC’s or Akcea’s goals, expectations, financial or other projections, intentions or beliefs, including the commercial potential of TEGSEDI, WAYLIVRA or other of PTC’s or Akcea’s drugs in development is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs, including receiving any necessary regulatory approval, that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. PTC’s and Akcea’s forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although PTC’s and Akcea’s forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by PTC and Akcea, and PTC and Akcea do not undertake or plan to update or revise any such statements to reflect actual results or changes in goals, expectations, financial or other projections, intentions or beliefs occurring after the date of this press release except as required by law. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning PTC’s and Akcea’s programs are described in additional detail in PTC’s and Akcea’s quarterly reports on Form 10-Q and annual reports on Form 10-K, which are on file with the SEC, as well as in other filings with the SEC by PTC and Akcea. Copies of these and other documents are available from each company.

In this press release, unless the context requires otherwise, “PTC,” “Akcea,” “Company,” “Companies,” “we,” “our,” and “us” refers to PTC Therapeutics and/or Akcea Therapeutics.

Ionis Pharmaceuticals™ is a trademark of Ionis Pharmaceuticals, Inc. Akcea Therapeutics™, TEGSEDITM and WAYLIVRATM are trademarks of Akcea Therapeutics, Inc.

For More Information:

PTC Therapeutics, Inc.
Investors:
Emily Hill
+ 1 (908) 912-9327
ehill@ptcbio.com

Media:
Jane Baj
+1 (908) 912-9167
jbaj@ptcbio.com

Akcea Therapeutics, Inc.
Investors:

 
 
 




Kathleen Gallagher
Vice President of Communications and Investor Relations
617-207-8509
kgallagher@akceatx.com

Media:
Lynn Granito
Berry & Company
T: 212 253-8881
lgranito@berrypr.com