UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM 8-K/A
Amendment No. 1
CURRENT REPORT
Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934
Date of Report (Date of earliest event reported): June 29, 2017
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
EXPLANATORY NOTE
This Amendment No. 1 on Form 8-K/A (this “Amendment”) is being filed solely to include Exhibit 99.1 to the Current Report on Form 8-K filed by the Company with the Securities and Exchange Commission on June 29, 2017 (the “Original 8-K”), which exhibit was inadvertently omitted from the original filing due to a technical error.
Except as provided herein, the disclosures contained in this Amendment have not been updated to reflect events, results or developments that have occurred since the filing of the Original 8-K.
Item 7.01. Regulation FD Disclosure.
On June 29, 2017, the Company presented at the PPMD 2017 Connect Conference. The accompanying slide deck from the presentation has been posted on the Events and Presentations page under the Investors section of the Company’s website. A copy of the slide deck is also attached to this Current Report on Form 8-K (this “Report”) as Exhibit 99.1 and is incorporated by reference into this Item 7.01.
Please refer to page 2 of the presentation attached hereto as Exhibit 99.1 for a discussion of certain forward-looking statements included therein and the risks and uncertainties related thereto.
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
See Exhibit Index attached hereto.
Signature
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this Amendment No. 1 on Form 8-K/A to be signed on its behalf by the undersigned hereunto duly authorized.
PTC Therapeutics, Inc. | |||||
Date: June 30, 2017 | By: | /s/ Christine Utter | |||
Name: | Christine Utter | ||||
Title: | Principal Financial Officer | ||||
EXHIBIT INDEX
Exhibit No. | Description | |
99.1 | Presentation - PPMD 2017 Connect Conference | |
Accessing EMFLAZA™ (deflazacort) for Duchenne muscular Dystrophy
PPMD 2017 Connect Conference
June 29, 2017; 4:00pm
Stuart Peltz, PhD
PTC Therapeutics, South Plainfield, NJ
Forward looking statements within the meaning of
The Private Securities Litigation Reform Act of 1995
All statements, other than those of historical fact, contained in this presentation, are forward-looking statements, including statements
regarding: the future expectations, plans and prospects for PTC; the PDUFA date for the new drug application (NDA) for Translarna for the
treatment of nonsense Duchenne muscular dystrophy (nmDMD) as well as the date of the advisory committee meeting; PTC’s ability to
maintain the current label under the marketing authorization in the European Economic Area (EEA) for TranslarnaTM (ataluren) for
nmDMD; the clinical utility and potential advantages of Translarna (ataluren) and Emflaza™ (deflazacort); PTC’s expectations with respect
to the future commercial availability of, and access to, Emflaza; PTC'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 "plan,"
"anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," and similar
expressions.
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: PTC's ability to resolve the matters set forth in the
Refuse to File letter it received from the FDA in connection with its NDA for Translarna for the treatment of nmDMD, including whether
PTC's filing of the NDA over protest with the FDA will result in a timely or successful review of the NDA, and whether PTC will be required
to perform additional clinical and non-clinical trials or analyses at significant cost, which, if successful, could potentially support the
approval of the NDA filed over protest or a new NDA submission; PTC's ability to maintain its marketing authorization of Translarna for the
treatment of nmDMD in the EEA, including whether the European Medicines Agency (EMA) determines in future annual renewal cycles
that the benefit-risk balance of Translarna authorization supports renewal of such authorization; PTC's ability to enroll, fund, complete and
timely submit to the EMA the results of Study 041, which is a specific obligation to continued marketing authorization in the EEA; PTC's
scientific approach and general development progress; matters related to PTC’s commercial launch of Emflaza in the United States; PTC’s
ability to realize the anticipated benefits of its acquisition of Emflaza, including the possibility that the expected benefits from the
acquisition will not be realized or will not be realized within the expected time period; the sufficiency of PTC's cash resources and its ability
to obtain adequate financing in the future for its foreseeable and unforeseeable operating expenses and capital expenditures; and the
factors discussed in the "Risk Factors" section of PTC’s Quarterly Report on Form 10-Q 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.
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 will receive or maintain regulatory approval in any territory, or prove to be
commercially successful, including Translarna or Emflaza.
The forward-looking statements contained herein represent PTC's views only as of the date of this presentation 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 presentation except as required by law.
PTC Therapeutics, Inc.
Founded 20 years ago
Now has a global footprint
with US headquarters
located in NJ
Focused on discovery,
development and
commercialization of oral
therapies for rare diseases
Multiple programs across
genetic disorders and
oncology
Our Mission
To leverage our knowledge
of RNA biology to bring
novel therapeutics to patients affected
by rare and neglected disorders
Commitment to patients with rare and
neglected diseases
PTC– A history steeped in commitment to DMD
FDA PDUFA IN
OCT
AdCom Sept. 28
Translarna™
discovery
Phase 2a (004)
38 patients
Phase 1
62 healthy volunteers
Phase 2b (007)
174 patients
Initial Natural
History Publications
19 years of research and development
1,000+ healthy volunteers / patients exposed / treated
Safety profile: generally well tolerated
2004 2005 2006 2007 2008 2009 2010 2011 2012 2013 2014 98 – 2003 2015 2016
EMA DMD Draft
Guidelines
Phase 3 (020)
228 patients
EU Initial Approval &
Renewal for
Translarna™
* Approval is subject to annual review and renewal by the European Commission, following reassement by the EMA of the risk/benefit balance
FDA DMD Draft
Guidelines
2017
Acquired Emflaza™
T
R
A
N
S
L
A
R
N
A
DMD Milestones are Irreversible; drugs that slow disease
progression change the course of the disease
0 5 10 15 20 25 30 Years
Loss of
Ambulation
Death
Loss of Stair
Climb
Loss of Stair
Descend
Early Physical Manifestations
Late Physical Manifestations
Ventilation
Loss of Upper
Limb
Impaired ability to
Hop, Run, Jump, Rise
Targeting the inflammatory component of DMD
DUCHENNE MUSCULAR DYSTROPHY
Immune-mediated pathology in Duchenne muscular dystrophy
Amy S. Rosenberg,1* Montserrat Puig,1 Kanneboyina Nagaraju,2 Eric P. Hoffman,2
S. Armando Villalta,3 V. Ashutosh Rao,1 Lalage M. Wakefield,4 Janet Woodcock1
Immunological and inflammatory processes downstream of dystrophin deficiency as well asmetabolic abnormalities,
defective autophagy, and loss of regenerative capacity all contribute to muscle pathology in Duchenne muscular dystrophy
(DMD). These downstream cascades offer potential avenues for pharmacological intervention. Modulating the
inflammatory response and inducing immunological tolerance to de novo dystrophin expression will be critical to the
success of dystrophin-replacement therapies. This Review focuses on the role of the inflammatory response in DMD
pathogenesis and opportunities for clinical intervention.
Science Translation Medicine; Volume 7; 5 Aug. 2015; Page 1-12
Acquired in April, 2017
Acquisition based on our understanding
of the results from the placebo arm from
our Randomized Control Trials
EMFLAZA™ indication is regardless of
genetic mutation
Received FDA approval on February 9,
2017
EMFLAZA™ Improved muscle strength
and slowed disease progression
We believe that EMFLAZA™ should be
available for Duchenne patients as part
of their totality of care
EMFLAZA™ is the first FDA approved corticosteroid
for treatment of Duchenne muscular dystrophy
EMFLAZA™ (deflazacort)
EMFLAZA is indicated for the treatment of
Duchenne muscular dystrophy in patients 5 years of age and older.
Contraindication: Do not use if you are allergic to deflazacort
or any of the inactive ingredients in EMFLAZA.
Please see Indication & Important Safety Information
for EMFLAZA in this deck
The first
FDA-approved
corticosteroid
indicated for
the treatment
of Duchenne
Once-a-day
dosing regimen
taken with or
without food
Improved
muscle strength
and slowed disease
progression
EMFLAZA™ strategic rationale
Aligns with mission to bring important therapies to patients with rare
diseases
Continues PTC’s commitment to DMD patients and families
Establishes U.S. commercial footprint for potential ataluren launch if
FDA approved
A second commercial DMD therapy that is complementary to
ataluren
Treatment guidelines recommend corticosteroids
(CS) as part of standard of care for Duchenne
CS are the only medication currently
available that slows the decline in muscle
strength and function in Duchenne
muscular dystrophy regardless of mutation
CS help reduce the risk of scoliosis and
stabilize pulmonary function; cardiac
function may also improve
CS should be offered for improving
strength and pulmonary function in
Duchenne patients
CS improve timed motor function, reduce the
need for scoliosis surgery, delay
cardiomyopathy onset and loss of ambulation
and increase survival
The Centers for Disease Control 2010 Guidelines urge consideration of
corticosteroid therapy for Duchenne patients1
The American Academy of Neurology 2016 Guidelines urge consideration of
corticosteroid therapy for Duchenne patients2
1. Bushby K, et al. Lancet Neurol. 2010: 9(1); 77-93. 2. Gloss D, et al. Neurology. 2016; 86(5); 465-472. 3. Data on file.
A service program providing personalized support
and resources to help you gain access to EMFLAZA
Enroll in EMFLAZACares™
Healthcare provider completes the
Prescription Start Form, which serves
as the prescription
Patient/guardian signs the consent
section of the Prescription Start form
The form is faxed to EMFLAZACares™
with copies of both sides of insurance
and prescription benefit cards
The Prescription Start Form can be
found at Emflaza.com
Work with an EMFLAZACares™ Case Manager
EmflazaCares
Case Manage
Out-of-pocket Assistance
program
Bridging program
Patient Assistance
program
Patient foundation support
(e.g., Assistance Fund,
NORD)
Patient
financial
assistance*
*to eligible participants
Prescription Assistance Program
• For patients with commercial insurance and out-of-pocket costs
associated with EMFLAZA who qualify, EMFLAZACares™ offers a copay
assistance program
• For patients who need additional assistance or are recipients of state or
federal insurance and qualify for assistance, EMFLAZACares™ will direct
them to alternative funding, such as independent charitable patient
assistance foundations that may be able to help with out-of-pocket costs,
as determined solely by the independent charitable foundation
• For patients who have no insurance, EMFLAZACares™ offers a Free Drug
Program for those who qualify
• If insurance decision is delayed, EMFLAZACares™offers a Bridge
Program so that no qualified patient has a gap in treatment
Successfully delivering EMFLAZA to patients since May
Growing number of prescriptions
~700 patients have received EMFLAZA
via either commercial or bridge supply
Out-of-pocket Assistance
program
Bridging program
PAP program
Patient foundation support
(e.g., Assistance Fund,
NORD)
Patients in 40 states receiving drug
Medicaid coverage in ~45 States
Current deflazacort patients eligible to bridge
Average commercial out-of-pocket close to $0
Fill out the Prescription Start Form with signatures from both the
healthcare provider and patient/guardian and fax to EMFLAZACares™
Case manager at EMFLAZACares™ works with insurers and patient
assistance programs to get coverage with little to no out-of-pocket cost
EMFLAZA is delivered to the patient’s home by a specialty pharmacy
Refills of EMFLAZA are automatic. EMFLAZACares™
will contact the patient/guardian to schedule a delivery
When EMFLAZA™ treatment is appropriate…
Prescribing and access takes 4 steps
1
2
3
4
A service program providing personalized support
and resources to help you gain access to EMFLAZA
Contact EMFLAZACares™
Phone: 1-844-EMFLAZA (1-844-363-5292)
Fax: 1-844-322-9980
EMFLAZA.com
Contraindication: Do not use if you are allergic to deflazacort or any of the inactive ingredients in
EMFLAZA.
Do not stop taking EMFLAZA, or change the amount you are taking, without first checking with your
healthcare provider, as there may be a need for gradual dose reduction to decrease the risk of
adrenal insufficiency and steroid “withdrawal syndrome”. Acute adrenal insufficiency can occur if
corticosteroids are withdrawn abruptly, and can be fatal. A steroid “withdrawal syndrome,” seemingly
unrelated to adrenocortical insufficiency, may also occur following abrupt discontinuance of
corticosteroids. For patients already taking corticosteroids during times of stress, the dosage may
need to be increased.
Hyperglycemia: Corticosteroids can increase blood glucose, worsen pre-existing diabetes,
predispose those on long-term treatment to diabetes mellitus, and may reduce the effect of anti-
diabetic drugs. Monitor blood glucose at regular intervals. For patients with hyperglycemia, anti-
diabetic treatment should be initiated or adjusted accordingly.
Increased Risk of Infection: Tell your healthcare provider if you have had recent or ongoing
infections or if you have recently received a vaccine or are scheduled for a vaccination. Seek
medical advice at once should you develop fever or other signs of infection, as some infections can
potentially be severe and fatal. Avoid exposure to chickenpox or measles, but if you are exposed,
medical advice should be sought without delay.
Alterations in Cardiovascular/Kidney Function: EMFLAZA can cause an increase in blood pressure,
salt and water retention, or a decrease in your potassium and calcium levels. If this occurs, dietary
salt restriction and potassium supplementation may be needed.
Behavioral and Mood Disturbances: There is a potential for severe behavioral and mood changes
with EMFLAZA and you should seek medical attention if psychiatric symptoms develop.
EMFLAZA™ important safety information
Effects on Bones: There is a risk of osteoporosis or decrease in bone mineral density with prolonged
use of EMFLAZA, which can potentially lead to vertebral and long bone fractures.
Effects on Growth and Development: Long-term use of corticosteroids, including EMFLAZA may
slow growth and development in children.
Ophthalmic Effects: EMFLAZA may cause cataracts or glaucoma and you should be monitored if
corticosteroid therapy is continued for more than 6 weeks.
Vaccination: The administration of live or live attenuated vaccines is not recommended. Killed or
inactivated vaccines may be administered, but the responses cannot be predicted.
Serious Skin Rashes: Seek medical attention at the first sign of a rash.
Drug Interactions: Certain medications can cause an interaction with EMFLAZA. Tell your
healthcare provider of all the medicines you are taking, including over-the-counter medicines (such
as insulin, aspirin or other NSAIDS), dietary supplements, and herbal products. Alternate treatment,
dosage adjustment, and/or special test(s) may be needed during the treatment.
Common side effects that could occur with EMFLAZA include: Facial puffiness or Cushingoid
appearance, weight increased, increased appetite, upper respiratory tract infection, cough, frequent
daytime urination, unwanted hair growth, central obesity, and colds.
Please see the accompanying full Prescribing Information
For medical information, product complaints, or to report an adverse event, please call 1-866-562-
4620.
You may report adverse events to FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
EMFLAZA™ important safety information
EMFLAZA™ and EMFALZACares ™ are registered trademarks of PTC Therapeutics, Inc.
A service program providing personalized support
and resources to help you gain access to EMFLAZA
Contact EMFLAZACares™
Phone: 1-844-EMFLAZA (1-844-363-5292)
Fax: 1-844-322-9980
EMFLAZA.com