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): January 9, 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))
Item 2.02. Results of Operations and Financial Condition.
On January 9, 2017, PTC Therapeutics, Inc. (the “Company”) issued a press release (the “press release”) announcing certain preliminary (unaudited) financial information for its fourth quarter and fiscal year ending December 31, 2016, including that the Company expects to report (i) TranslarnaTM (ataluren) net sales for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) of approximately $81 million and (ii) ending cash and cash equivalents of approximately $230 million. Final results are subject to completion of the Company’s year-end audit.
Item 7.01. Regulation FD Disclosure.
The Company also announced financial guidance for its fiscal year ending December 31, 2017 in the press release, including that the Company expects (i) ex-U.S. Translarna nmDMD net sales of between $105 and $125 million, assuming current exchange rates; (ii) non-GAAP operating expenses $190 and $200 million, excluding estimated non-cash stock-based compensation expense of approximately $35 million, for total GAAP operating expenses of approximately $225 to $235 million; and (iii) ending cash and cash equivalents of approximately $160 million.
The Company announced that on Wednesday, January 11th at 7:30 am PT at the 35th Annual J.P. Morgan Healthcare Conference (the "Conference"), the Company will present its 2017 strategic priorities, preliminary 2016 financial results, and 2017 financial guidance. The presentation will be webcast live and the accompanying slide deck has been posted on the Events and Presentations page under the Investors section of the Company’s website. A copy of the slide deck, which the Company intends to utilize in various meetings at the Conference, is also attached as Exhibit 99.2.
This Current Report on Form 8-K and Exhibits 99.1 and 99.2 include a forward-looking financial measure that was not prepared in accordance with accounting principles generally accepted in the United States (GAAP), non-GAAP operating expenses (which excludes stock-based compensation expense). Management uses this measure to assess its operations and, in management’s opinion, this non-GAAP measure is useful to investors and other users of its financial statements by providing greater transparency into the expected operating performance at PTC and the Company’s future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP.
The information in this Current Report on Form 8-K, including Exhibits 99.1 and 99.2, shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such a filing.
Forward Looking Statements: All statements, other than those of historical fact, contained in this Current Report on Form 8-K, are forward-looking statements, including preliminary (unaudited) financial information for 2016 and financial guidance for 2017. 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 those related to the preliminary nature of the Company's 2016 financial information, which is subject to completion of the Company's year-end audit; the assumptions underlying the Company's financial guidance for 2017; and the factors discussed in the “Risk Factors” section of the Company's most recent Quarterly Report on Form 10-Q as well as any updates to these risk factors filed from time to time in the Company's other filings with the SEC. You are urged to carefully consider all such factors. The forward-looking statements contained herein represent the Company's views only as of the date of this Current Report on Form 8-K 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 Current Report on Form 8-K except as required by law.
Item 9.01. Financial Statements and Exhibits.
(d) Exhibits:
99.1 Press Release dated January 9, 2017
99.2 Corporate Presentation - 35th Annual J.P. Morgan Healthcare Conference
SIGNATURES
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: January 9, 2017 | By: | /s/ Shane Kovacs |
Shane Kovacs | ||
Chief Financial Officer | ||
EXHIBIT INDEX
Exhibit No. | Description | |
99.1 | Press Release dated January 9, 2017 | |
99.2 | Corporate Presentation - 35th Annual J.P. Morgan Healthcare Conference | |
Exhibit 99.1PTC Therapeutics Provides Corporate Update and Outlines 2017 Strategic Priorities
to Maximize the Global Value of Translarna™ and Advance its Innovative Pipeline
to Maximize the Global Value of Translarna™ and Advance its Innovative Pipeline
– Preliminary 2016 Translarna unaudited net sales of approximately $81M, a 140% increase vs. 2015 –
– Strategic update for Translarna regulatory path in the U.S. –
– Topline ACT CF data anticipated late first quarter 2017 –
– SMA program actively enrolling patients across SUNFISH and FIREFISH trials –
– RG7916 granted orphan-drug designation for the treatment of SMA –
– Translarna 2017 net sales guidance of $105M to $125M –
SOUTH PLAINFIELD, N.J., Jan. 9, 2017 - PTC Therapeutics, Inc. (NASDAQ: PTCT) today provided a corporate update, which will be detailed as part of the company presentation at the 35th Annual
J.P. Morgan Healthcare Conference on Wednesday, January 11th at 7:30 am PT. Stuart W. Peltz, Ph.D., PTC’s Chief Executive Officer, will present the company’s 2017 strategic priorities, preliminary 2016 financial results and 2017 financial guidance. The presentation will be webcast live and available with the related slide deck on the Events and Presentations page under the investors section of PTC Therapeutics' website at www.ptcbio.com.
J.P. Morgan Healthcare Conference on Wednesday, January 11th at 7:30 am PT. Stuart W. Peltz, Ph.D., PTC’s Chief Executive Officer, will present the company’s 2017 strategic priorities, preliminary 2016 financial results and 2017 financial guidance. The presentation will be webcast live and available with the related slide deck on the Events and Presentations page under the investors section of PTC Therapeutics' website at www.ptcbio.com.
Commercial Highlights, Preliminary 2016 Unaudited Financial Results, and 2017 Guidance
• | PTC expects to report Translarna (ataluren) net sales for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) of approximately $81 million for 2016, an increase of 140% over the prior year and achieving the upper-end of guidance. This strong performance reflects rapid uptake, sustainable pricing, and high (>90%) compliance to treatment. |
• | PTC expects to report year-end 2016 cash and cash equivalents of approximately $230 million. |
• | For 2017, PTC expects to achieve ex-U.S. Translarna nmDMD net sales of between $105 and $125 million, assuming current exchange rates, representing continued strong growth year-over-year of its sustainable DMD business. This is driven by both increased penetration into the over 25 countries where Translarna is currently available as well as continued geographic expansion into new territories. |
• | Non-GAAP operating expenses for 2017 are expected to be between $190 and $200 million excluding estimated non-cash stock-based compensation expense of approximately $35 million, for total operating expenses of approximately $225 to $235 million. |
• | PTC expects to finish 2017 with approximately $160 million of cash and cash equivalents. |
Clinical and Regulatory Highlights
• | Following multiple interactions with U.S. FDA officials and PTC’s advisors, PTC plans to file the Translarna New Drug Application (NDA) for nmDMD over protest with the U.S. FDA in the first quarter of 2017. Feedback indicated this process, rather than continued appeal, is the best path forward for the current Translarna NDA to receive a full and fair review. Filing over protest is a procedural path permitted by U.S. FDA regulations that allows a company to have its NDA filed and reviewed when there is a disagreement with regulators over the acceptability of the NDA submission. PTC plans to supplement the current NDA with additional efficacy analyses utilized by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) in their recent renewal recommendation. |
• | The EMA’s CHMP recommended the renewal of the conditional marketing authorization for Translarna (ataluren) for the treatment of nmDMD based on a continued positive benefit-risk assessment. As a specific obligation of the renewal, PTC will conduct an additional trial of Translarna in nmDMD. |
• | Top-line results of ACT CF are anticipated late in the first quarter of 2017. ACT CF is a Phase 3, international, multicenter, randomized, double-blind, placebo-controlled trial that is evaluating the absolute change in percent predicted forced expiratory volume in one second (FEV1) in patients with nonsense mutation cystic fibrosis (nmCF). |
• | The spinal muscular atrophy (SMA) program, a joint collaboration with Roche and the SMA Foundation, is expected to advance into two pivotal studies in 2017. SUNFISH and FIREFISH are both two part studies in childhood onset (Type 2/3) and infant onset (Type 1) SMA patients, respectively. Both studies are enrolling the initial dose escalation part of the study which will then transition to the pivotal part of the study evaluating efficacy. Commencement of the pivotal portion of either study will trigger a $20 million milestone payment to PTC from Roche. RG7916 was recently granted orphan-drug designation by the U.S. FDA. |
Pipeline Highlights:
• | Phase 2 proof-of-concept studies of Translarna in additional rare disease indications, including aniridia, MPS I, and Dravet/CDKL5, continue to progress. Proof-of-concept from these studies would further validate Translarna’s potential as a precision medicine for a number of rare genetic disorders caused by a nonsense mutation. |
• | Clinical development of PTC596 is expected to progress into additional clinical studies in 2017. PTC596 is a novel, oral investigational drug that reduces the levels of BMI1, a protein required for cancer stem cell survival. An ongoing Phase 1 dose escalating study confirms that PTC596 is generally well tolerated at doses that achieved or exceed plasma concentrations in preclinical models. |
• | PTC’s genetic disorders research organization is actively advancing lead optimization programs from its splicing platform focused on Huntington’s disease and Familial Dysautonomia. |
About PTC Therapeutics, Inc.
PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of orally administered, proprietary small molecule drugs targeting an area of RNA biology we refer to as post-transcriptional control. Post-transcriptional control processes are the regulatory events that occur in cells during and after a messenger RNA, or mRNA, molecule is copied from DNA through the transcription process. PTC's internally discovered pipeline addresses multiple therapeutic areas, including rare disorders and oncology. PTC has discovered all of its compounds currently under development using its proprietary technologies. PTC plans to continue to develop these compounds both on its own and through selective collaboration arrangements with leading pharmaceutical and biotechnology companies. For more information on the company, please visit our website www.ptcbio.com.
For More Information:
Investors:
Emily Hill
+1 (908) 912-9327
ehill@ptcbio.com
Media:
Jane Baj
+1 (908) 912-9167
jbaj@ptcbio.com
Forward Looking Statements:
All statements, other than those of historical fact, contained in this press release, are forward-looking statements, including statements regarding: the future expectations, plans and prospects for PTC; PTC’s preliminary 2016 unaudited results, including (i) 2016 net sales of Translarna for the treatment of nmDMD and (ii) year-end 2016 cash and cash equivalents; PTC’s financial guidance for 2017, including (i) net sales, (ii) non-GAAP and GAAP operating expenses, and (iii) ending cash and cash equivalents; the timing and outcome of PTC’s regulatory process including, (i) PTC’s ability to resolve the matters set forth in the Refuse to File letter from the FDA or otherwise advance Translarna for the treatment of nmDMD in the U.S., including its ability to supplement the NDA with additional efficacy analyses and submit its NDA for nmDMD with the FDA via the file over protest process in the first quarter of 2017 and (ii) the final determination by the European Commission with respect to renewal of the marketing authorization in the European Economic Area (EEA) for Translarna for the treatment of nmDMD and PTC’s plan to conduct an additional Phase 3 randomized trial of Translarna in nmDMD; the clinical utility and potential advantages of Translarna; when top-line results of ACT CF will be available and reported; the timing, results and conduct of PTC’s clinical studies of PTC596 and Translarna for the treatment of other indications, including statements regarding the timing of initiation, evaluation, enrollment and completion of the studies; any further advancement of either or both of the FIREFISH and SUNFISH studies under the joint SMA collaboration, including transition into the pivotal part of either study; the timing of a milestone payment, if any, to PTC from Roche; PTC’s ability to continue to supply Translarna to patients, increase commercial penetration in countries where Translarna is currently available, and continue commercial expansion into new territories; 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 “preliminary,” “guidance,” “will,” “plan,” “expect,” “target,” “anticipate,” “believe,” “estimate,” “intend,” “may,” “potential,” “project,” “possible,” “potential,” “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: the preliminary nature of PTC’s 2016 financial results, which are subject to completion of its year-end audit; the assumptions underlying PTC’s financial guidance for 2017; PTC’s ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in the EEA, including whether the European Commission determines to approve the renewal of such authorization and whether the EMA determines in future annual renewal cycles that the benefit-risk balance of Translarna authorization supports renewal of such authorization; the final design of the new nmDMD trial that PTC will undertake pursuant to the specific obligation associated with the marketing authorization (if renewed) and PTC’s ability to enroll, fund and conduct such trial; the timing and outcome of future interactions PTC has with the FDA with respect to Translarna for the treatment of nmDMD, including PTC's ability to resolve the matters set forth in the Refuse to File letter from the FDA or otherwise advance Translarna for the treatment of nmDMD in the United States (whether pursuant to the file over protest process or otherwise), including whether PTC is required to perform additional clinical and non-clinical trials at significant cost and whether such trials, if successful, may enable FDA review of a NDA; the outcome of ongoing or future clinical trials or studies in Translarna, in particular ACT CF; events during, or as a result of, the SUNFISH or FIREFISH studies that could delay or prevent further advancement of the SMA program, including future actions or activities under the SMA joint development program; the eligible patient base and commercial potential of Translarna and PTC’s other product candidates; the EMA’s determinations with respect to PTC’s variation submission which seeks to add Translarna for the treatment of nmCF to PTC’s marketing authorization in the EEA; the scope of regulatory
approvals or authorizations for Translarna (if any), including labeling and other matters that could affect the availability or commercial potential of Translarna; PTC’s ability to commercialize and commercially manufacture Translarna in general and specifically as a treatment for nmDMD, including its ability to establish and maintain arrangements with manufacturers, suppliers, distributors and production and collaboration partners on favorable terms; the outcome of pricing and reimbursement negotiations in those territories in which PTC is authorized to sell Translarna; expectations for regulatory approvals, including PTC’s ability to make regulatory submissions in a timely manner (or at all), the period during which the outcome of regulatory reviews will become available, adverse decisions by regulatory authorities (or other delay or deceleration of the regulatory process), and PTC’s ability to meet existing or future regulatory standards with respect to Translarna; PTC’s scientific approach and general development progress; 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 most recent 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 Translarna will receive full regulatory approval in any territory or maintain its current marketing authorization in the EEA, or prove to be commercially successful in general, or specifically with respect to the treatment of nmDMD.
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.
PageJan-17
PTC Therapeutics:
A Precision Medicine Platform
35th Annual JP Morgan Healthcare Conference
1
Page
Forward looking statements within the meaning of
The Private Securities Litigation Reform Act of 1995
Jan-17
All statements, other than those of historical fact, contained in this presentation, are forward-looking statements, including the information appearing under the
headings “2016 Preliminary Results” and “2017 Forecast” as well as statements regarding: the future expectations, plans and prospects for PTC; the timing and
outcome of PTC’s regulatory process in the U.S., including as related to a planned file over protest of the new drug application for TranslarnaTM for the treatment of
nmDMD, and in the European Economic Area (EEA), including as related to the European Commission’s determination as to renewal of the marketing authorization
for Translarna for the treatment of nmDMD and PTC’s plan to conduct an additional Phase 3 randomized trial of Translarna in nmDMD; the clinical utility and
potential advantages of Translarna; when top-line results of ACT CF will be available and reported and any statements related to the potential results of such trial;
PTC's estimates regarding the potential market opportunity for Translarna, including the size of eligible patient populations and PTC's ability to identify such
patients; PTC’s ability to maintain the current label under the marketing authorization in the EEA; the timing of, and PTC’s ability to, expand the approved product
label of Translarna for the treatment of nmDMD in the EEA, whether pursuant to its ongoing Phase 2 study of Translarna for nmDMD in pediatric patients, or
otherwise; the timing of, and PTC’s ability to, obtain additional marketing authorizations for Translarna in other territories, including the U.S., or for additional
indications, including nmCF; the timing, results and conduct of PTC’s clinical studies of PTC596 and Translarna for the treatment of other indications; further
advancement of the FIREFISH and SUNFISH studies under the joint SMA collaboration, including transition into the pivotal part of either study; the timing of a
milestone payment, if any, to PTC from Roche; PTC’s ability to increase commercial presence in countries where Translarna is currently available and commercially
expand into new territories; 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 “estimate,” “outlook,” “will,” “plan,” “expect,” “anticipate,” “believe,” “estimate,” “intend,” “may,” “potential,”
“possible,” “potential,” “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: the completion of PTC’s year-end audit; PTC’s ability to maintain its marketing authorization of
Translarna for the treatment of nmDMD in the EEA, including whether the European Commission determines to approve the renewal of such authorization and
whether the EMA determines in future annual renewal cycles that the benefit-risk balance of Translarna authorization supports renewal of such authorization; the
final design of the new nmDMD trial that PTC will undertake pursuant to the specific obligation associated with the marketing authorization (if renewed) and PTC’s
ability to enroll, fund and conduct such trial; the timing and outcome of future interactions PTC has with the FDA with respect to Translarna for the treatment of
nmDMD, including PTC's ability to resolve the matters set forth in the Refuse to File letter from the FDA or otherwise advance Translarna for the treatment of
nmDMD in the United States (whether pursuant to the file over protest process or otherwise), including whether PTC is required to perform additional clinical and
non-clinical trials at significant cost; the outcome of ongoing or future clinical trials or studies in Translarna, in particular ACT CF and the Translarna nmDMD
pediatric study; events during, or as a result of, the SUNFISH or FIREFISH studies that could delay or prevent further advancement of the SMA program, including
future actions or activities under the SMA joint development program; the eligible patient base and commercial potential of Translarna and PTC’s other product
candidates; the scope of regulatory approvals or authorizations for Translarna (if any), including labeling and other matters that could affect the availability or
commercial potential of Translarna; PTC’s ability to commercialize and commercially manufacture Translarna in general and specifically as a treatment for nmDMD;
the outcome of pricing and reimbursement negotiations in those territories in which PTC is authorized to sell Translarna; PTC’s scientific approach and general
development progress; 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 most recent 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 Translarna will receive full regulatory approval in any territory or maintain its current marketing authorization in the EEA, or prove to be
commercially successful in general, or specifically with respect to the treatment of nmDMD. The forward-looking statements contained herein represent PTC’s views
only as of the date of this presentation and PTC does not undertake to update or revise any such forward-looking statements occurring after the date of this
presentation except as required by law.
2
Page
Our mission
To leverage our knowledge
of RNA biology to bring
novel therapeutics to patients
affected by rare and neglected
disorders
Jan-17 3
Page
Sustainable, growing DMD business enables innovative
research & development engine
Translarna™: Strong commercial growth for DMD ex-US
Development and regulatory milestones for Translarna
CHMP recommended renewal of approval of DMD*
File NDA over protest for DMD with the U.S. FDA in Q1 2017
ACT CF Phase 3 clinical trial results in Q1 2017
Proof-of-concept trials advance: MPS I, Aniridia & Dravet / CDKL5
Expanding pipeline
SMA: advancing into two pivotal studies
BMI1, Huntington’s and FD programs
Strong financial position with healthy balance sheet
Jan-17 3
*Annual renewal subject to approval by the European Commission
PageJan-17
Translarna™
Precision medicine platform: Realizing Translarna’s full value
through multiple indications
5
Page
Translarna™ : Sustainable, growing DMD
business supporting long term success
Jan-17 6
25+ Countries worldwide with Translarnacommercial therapy*
Preliminary 2016 Translarna unaudited net sales of ~$81M*
Established footprint in 47 countries worldwide
EMA renewal establishes sustainability of ex-US business
Experienced commercial team in orphan disease
*Commercial sales through commercial or early access programs
Page
$1
$34
$81
$105
$0
$20
$40
$60
$80
$100
$120
$140
2014 2015 2016 2017
Strong demand drives substantial year-over-year net
sales growth
Jan-17 7
2017 Translarna net sales guidance of $105 - $125M
Translarna™ ex-US DMD Net Sales
($ in millions)
$125
Page
Key Drivers of Continued Market Growth
Jan-17 8
Increase penetration in current countries
New geographies
>90% patient compliance to Translarna
Opportunities for label expansion
PageJan-17 9
Europe
~2,000 pts.
Asia Pac:
~600 pts.
U.S. &
Canada:
~1,000 pts.
>7,000 addressable nmDMD patients worldwide
Lat Am/ Mex:
~2,000 pts.
~85% of patients outside the U.S.
MENA
~1,400 pts.
Internal estimates of potential patient population based on data derived from a variety of sources
and are subject to change
Page
Developing insight on patient prevalence
Jan-17 10
1:4,000 male
births are DMD
Birth rate varies
country to country
Global nmDMD prevalence drivers
Current diagnosed population >5yrs =
>7,000* patients
Typically patients not diagnosed <5 years of age
~10-15%
nonsense
mutation
Under current
label 5+yrs and
ambulatory~35%
~65%
Non-ambulatory
*Internal estimates of potential patient population based on data derived from a
variety of sources and are subject to change
Page
Current efforts to grow addressable DMD population
11
Earlier diagnosis Increase disease awareness
Genotyping Improving standards of
care
Jan-17
Identifying 2-4 year olds increases potential addressable
population by ~20%
?
Page
Potential market opportunity for Translarna™ in DMD
Jan-17 12
Ex-US,
5+ yrs and
ambulatory
U.S.
all patients
ambulatory and
non-ambulatory
Ex-US 2-4 yrs
Potential peds
label in 2018
~$50M peak
NDA
submission
in 2017
Ex-U.S.,
5+ yrs and
non-ambulatory
Gathering
long-term
data (FVC)
Current
Label ~$250M
peak
Page
Translarna™ regulatory update: CHMP recommends
renewal of approval, NDA to be reviewed by FDA
EMA
Renewal of Translarna DMD approval recommended by CHMP
– Commitment to conduct post marketing DMD trial
FDA
Multiple interactions with FDA officials and advisors over the last several
months
Feedback indicated best path forward for full and fair review of the current
Translarna NDA is to file over protest rather than continue appeal of RTF
NDA to be supplemented with additional efficacy data utilized by CHMP in
renewal recommendation
Jan-17 13
Page
Potential Market opportunity for Translarna™ in nmCF:
>9,000 patients worldwide
Jan-17 14
Cystic Fibrosis
nmCF represents a significant commercial opportunity
DMD CF US CF ex-US Potential peak for
nmDMD & nmCF
Global CF
trial data
Q1:17
DMD
~3,000 pts
estimated
~6,000 pts
estimated
Internal estimates of potential patient population based on data derived from a
variety of sources and are subject to change
>7,000 pts
estimated
~16,000 pts
estimated
Page
Cystic fibrosis is caused by a malfunction of the
CFTR protein due to different mutations
Jan-17
CFTR channel
through
cell membrane
CFTR protein
cftr gene in
cell nucleus
V
Reduced
SynthesisClass Normal
IV
Conductance
I
No Synthesis
III
Gating
II
Processing
Block
% CF Patients 2%10% ~5%70%
Compounds Translarna Kalydeco®Orkambi®
CFTR
CFTR
CFTR
CFTR
CFTR
CFTR
CFTR
CFTR
CFTR
CFTR
CFTR
CFTR
CFTR
15
Translarna™ is only compound in development targeting Class I CF
Page
Slowing disease progression provides clinically meaningful benefits
to CF patients over the long-term
Translarna™ CF data is comparable to other
approved CF correctors***
Jan-17
0%
2%
4%
6%
8%
10%
12%
14%
A
bs
ol
ut
e
Δ
pp
FE
V
1
Class III
Class I
Class II
* Post hoc analysis; relative Δ 5.7% ** Post hoc analysis; relative Δ 8.4% *** Tested in separate trials
abs. Δ
3.5% *
abs. Δ
5.4% **
Translarna
non TOBI®
subgroup
Translarna
non TOBI® <18
subgroup
abs. Δ
12.5%
ENVISION
(Kalydeco®)
abs. Δ
10.7%
KONNECTION
(Kalydeco®)
abs. Δ
2.1%
KONDUCT
(Kalydeco®)
abs. Δ
3.0%
TRAFFIC and
TRANSPORT
(Orkambi®)
CFTR
CFTR
CFTR
CFTR
CFTR
CFTR
16
Page
Translarna™ reduced exacerbations
Jan-17 17
62%†
Overall population
(n=232)
Non-TOBI®
(n=146)
Non-TOBI® (age 6-18 years)
(n=49)
*p=.006 Nominal post-hoc data.
†p=.024 Nominal post-hoc data
De Boeck K, et al. 39th European Cystic Fibrosis Conference, Basel, Switzerland, June 8-11, 2016.
Abstract WS13.1.
.
41%*
23%
Page
Length of Trial TranslarnaTM (n) Placebo (n)
48 weeks 140 140
Primary outcome measure:
% predicted FEV1
Secondary outcome measure:
Pulmonary exacerbation rate
CFQ-R
BMI
ACT CF (Ataluren Confirmatory Trial) study:
Topline data expected late Q1 2017
Eligibility Criteria Stratification
Nonsense mutation CF
≥6 years
FEV1 ≥40% and ≤90% predicted
No chronic inhaled TOBI
Age: ˂18 vs ≥18 years
Inhaled antibiotic use (yes vs no)
Baseline FEV1: ≥65% vs <65% of
predicted
Trial powered with 90% confidence for 3% absolute Δ FEV1
Jan-17 18
Page
Ion channel
disorders
Publications: 1
Genetically defined
epilepsy
Metabolic
disorders
Publications: 10
Muscle
disorders
Publications: 6
Eye
disorders
Publications: 3
Skin
disorders
Publications: 2
Neurological
disorders
Publications: 8
Pulmonary
disorders
Publications: 10
Translarna™: Realizing a new paradigm for the treatment
of rare diseases, progressing proof of concept studies
Jan-17 19
~ 40 publications in many disease models
Page
Translarna™ restores morphology and sight in
Aniridia mouse model
20-40% nonsense mutation
– 12 month placebo controlled trial
– Primary endpoint PAX6 levels, eye form and function
– Enrolling well, targeting up to 40 patients
Jan-17 20
Untreated
Wild type
Treated
with
Translarna
Sey+/-
Sey+/-
Scotopic
response
Photopic
response
Oscillatory
potentials
12 Hz
flicker
WT
P60
Gregory-Evans 2014 JCI
Page
Progressing proof of concept studies:
MPS I and Dravet / CDKL5
MPS I: Metabolic enzyme disorder
– 3 month open label trial currently
enrolling naïve patients
– Primary endpoint change in urinary
& CNS GAG levels
– Challenges in enrolling, targeting 8
patients
Dravet / CDKL5: Genetic epilepsy
– 32 weeks placebo controlled trials
– Primary endpoint number of
monthly seizures
– Targeting up to 16 patients (8 for
Dravet and 8 for CDKL5)
Jan-17 21
60-80%
MPS I
nonsense
mutation
50% Dravet
10% CDKL5
nonsense mutation
PageJan-17
Small molecule splicing technology platform
Multiple on-going programs
22
Page
The spinal muscular atrophy program validates PTC’s
small molecule splicing platform
Jan-17 23
Target splicing event to restore or
reduce proteinSplicing
Tumor Resistance – MCL1
SMA - SMN2
FD – IKBKAP
HD – HTT
Page
Pivotal portion of both Sunfish & Firefish trials
expected to begin in 2017
SUNFISH
Clinical study in SMA type 2/3 patients initiated in November
– Enrolling 36 patients for dose escalation phase, placebo controlled 2:1
– Pivotal phase will be 150 patients, placebo controlled 1:1, endpoint of total motor
function measure (MFM-32) at 12 months
FIREFISH
Clinical study in SMA type 1 patients <7 months old
– Enrolling 8 patients for dose escalation phase
– Pivotal phase will enroll 40 babies, open label
endpoint of sitting as measured by Bayley infant scale
Jan-17 24
Two pivotal studies expected to begin in 2017
Page
HD
Huntington’s disorder is a progressive, inherited
neurodegenerative disorder
HD is caused by expression of mutant Huntingtin (HTT) protein
Causes selective and devastating neuronal loss
– Predominantly in the striatum and cerebral cortex
Adult onset at ~30-50 years
Unmet medical need
– Patients: 30,000 US, 100,000 WW
25
Healthy
Jan-17
Page
Multiple orally bioavailable, brain penetrant compounds being
optimized
– Lowers HTT by altering splicing
Opportunity to provide first-in-class disease modifying, HTT lowering
small molecule therapy
– Potential to circumvent delivery and distribution challenges seen with the
current modalities
HD program in Lead Optimization – showcases
PTC’s alternative splicing platform
Jan-17 26Jan-17 3
V e h ic le 1 0
0
2 0
4 0
6 0
8 0
1 0 0
S tr ia tu m
%
H
T
T
l
o
w
e
ri
n
g
P 3 0 1 9 0 5 (m g /k g )
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0
2 0
4 0
6 0
8 0
1 0 0
C o r te x
%
H
T
T
l
o
w
e
ri
n
g
P 3 0 1 9 0 5 (m g /k g )
V e h ic le 1 0
0
2 0
4 0
6 0
8 0
1 0 0
S k in
%
H
T
T
l
o
w
e
ri
n
g
P 3 0 1 9 0 5 (m g /k g )
Page
Expanding pipeline through in-house innovation
Jan-17 27
Product /
Platform
Discovery Preclinical Phase 1 Phase 2 Phase 3
DMD
CF
BMI1
SMA
HD
TranslarnaTM
(ataluren)
nonsense readthrough
SMA
alternative splicing
PTC596
tumor stem cell targeting
Huntington’s
alternative splicing
Commercial
Aniridia
Oncology
Orphan genetic disorders
MPS I
Next Generation
nonsense readthrough
Dravet / CDKL5
* Marketing authorization (MA) recommended by CHMP in the European Economic Area, which is subject to approval by the European Commission; MA requires
annual renewal following reassessment by the European Medicines Agency (EMA)
*
FDFamilial
Dysautonomia
Page
Strong financial position with sustainable,
growing DMD business
2016 Preliminary Results
Preliminary 2016 Translarna™ unaudited net sales of ~$81 million
Commercial business cash flow positive 2yrs post launch
12/31/16 year-end cash position of ~$230 million
2017 Guidance
2017 Translarna net sales guidance of $105 – $125 million*
$20 million SMA milestone payment expected mid-2017
2017 non-GAAP operating expenses of $190 - $200 million (excludes
~$35M in non-cash stock based compensation)
12/31/17 year-end cash position anticipated to be ~$160 million
Jan-17 28
• ex-US DMD sales
• Based on current exchange rates.
Page
PTC Near-term milestones
Jan-17 29
Phase 3
ACT CF data
in late Q1:17
SMA – 2
pivotal
studies
Continued
Translarna™
net sales
growth
ex-US
Additional
pipeline
progress
US FDA
regulatory
outcome for
Translarna™
in DMD