8-K
PTC THERAPEUTICS, INC. (PTCT)
8-K
2022-09-07
For: 2022-09-07
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April 07, 2026
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): September 7, 2022
PTC THERAPEUTICS, INC.
(Exact Name of Company as Specified in Charter)
| | | | | |
|---|---|---|---|---|
| Delaware | 001-35969 | 04-3416587 | ||
| (State or Other Jurisdiction | | (Commission | | (IRS Employer |
| of Incorporation) | | File Number) | | Identification No.) |
| | | |
|---|---|---|
| 100 Corporate Court | | |
| South Plainfield , NJ | | 07080 |
| (Address of Principal Executive Offices) | | (Zip Code) |
Registrant’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):
☐Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
☐Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
☐Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
☐Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))
Securities registered pursuant to Section 12(b) of the Act:
| | | | | |
|---|---|---|---|---|
| Title of each class | **** | Trading Symbol(s) | **** | Name of each exchange on which registered |
| Common Stock, $0.001 par value per share | | PTCT | | Nasdaq Global Select Market |
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 ☐
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. ☐
Item 7.01. Regulation FD Disclosure.
On September 7, 2022, PTC Therapeutics, Inc. (the “Company”) updated its corporate presentation in anticipation of upcoming investor conferences. The Company’s corporate presentation will be posted in the Events and Presentations page under the Investors section of the Company’s website. A copy of the corporate presentation 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.
The information set forth in or incorporated by reference into this Report, including Exhibit 99.1, 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. 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.
Item 9.01. Financial Statements and Exhibits.
(d) Exhibits
| | | |
|---|---|---|
| Exhibit No. | Description | |
| 99.1 | | PTC Therapeutics, Inc. Corporate Presentation |
| 104 | | The cover page from this Current Report on Form 8-K, formatted in Inline XBRL |
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: September 7, 2022 | By: | /s/ Emily Hill |
| | Name: | Emily Hill |
| | Title: | Chief Financial Officer |
Exhibit 99.1
| PTC 2022<br>Corporate Presentation<br>September 2022 | ||||||||
|---|---|---|---|---|---|---|---|---|
| Forward<br>-<br>Looking Statements<br>This presentation contains forward<br>-<br>looking statements within the meaning of The Private Securities Litigation Reform Act of 1995<br>.. All statements contained in this presentation, other than statements of<br>historic fact, are forward<br>-<br>looking statements,<br>including statements with respect to guidance relating to 2022 total<br>revenue,<br>2022 DMD franchise net product revenue<br>, 2022 operating expenditure guidance<br>and future revenue guidance and statements regarding: the future expectations, plans and prospects for PTC, including with re<br>spe<br>ct to the expected timing of clinical trials and studies, availability of data,<br>regulatory submissions and responses and other matters; expectations with respect to<br>Upstaza<br>and other programs within PTC's gene therapy platform, including any regulatory submissions,<br>commercialization and manufacturing capabilities; advancement of PTC's joint collaboration program in SMA, including any regu<br>lat<br>ory submissions, commercialization or royalty or milestone payments;<br>PTC's expectations with respect to the licensing, regulatory submissions and commercialization of its products and product ca<br>ndi<br>dates;<br>PTC's strategy, future operations, future financial position, future<br>revenues, projected costs; and the objectives of management. Other forward<br>-<br>looking statements may be identified by the<br>words<br>"guidance", "plan," "anticipate," "believe," "estimate," "expect," "intend,"<br>"may," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions.<br>PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward<br>-<br>looking sta<br>tements it makes as a result of a variety of risks and uncertainties, including<br>those related to: expectations with respect to the COVID<br>-<br>19 pandemic and related response measures and their effects on PTC's bu<br>siness, operations, clinical trials, regulatory submissions and approvals,<br>and PTC's collaborators, contract research organizations, suppliers and manufacturers; the outcome of pricing, coverage and r<br>eim<br>bursement negotiations with third party payors for PTC's products or<br>product candidates that PTC commercializes or may commercialize in the future; expectations with respect to<br>Upstaza<br>and other programs within PTC's gene therapy platform, including any regulatory<br>submissions and potential approvals, commercialization,<br>manufacturing capabilities and the potential financial impact and benef<br>its of its leased biologics manufacturing facility and the potential achievement<br>of development, regulatory and sales milestones and contingent payments that PTC may be obligated to make; expectations with<br>res<br>pect to the commercialization of<br>Evrysdi<br>under our SMA collaboration;<br>PTC's ability to maintain its marketing authorization of<br>Translarna<br>for the treatment of<br>nmDMD<br>in Brazil, Russia, the European Economic Area (EEA) and other regions, including whether the European<br>Medicines Agency (EMA) determines in future annual renewal cycles that the benefit<br>-<br>risk balance of<br>Translarna<br>authorization supports renewal of such authorization; PTC's ability to complete Study 041,<br>which is a specific obligation to continued marketing authorization in the EEA; PTC's ability to utilize results from Study 0<br>41,<br>a randomized, 18<br>-<br>month, placebo<br>-<br>controlled clinical trial of<br>Translarna<br>for the<br>treatment of<br>nmDMD<br>followed by an 18<br>-<br>month open<br>-<br>label extension, to support a marketing approval for<br>Translarna<br>for the treatment of<br>nmDMD<br>in the United States; expectations with respect to the<br>commercialization of<br>Tegsedi<br>and<br>Waylivra<br>; the results of PTC's clinical trial for<br>emvododstat<br>for COVID<br>-<br>19; significant business effects, including the effects of industry, market, economic, political or<br>regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commerci<br>al<br>potential of PTC's products and product candidates; PTC's scientific approach<br>and general development progress; PTC's ability to satisfy its obligations under the terms of its lease agreements, including<br>fo<br>r its leased biologics manufacturing facility; the sufficiency of PTC's cash<br>resources and its ability to obtain adequate financing in the future for its foreseeable and unforeseeable operating expenses<br>an<br>d capital expenditures; and the factors discussed in the "Risk Factors" section<br>of PTC's most recent Annual Report on Form 10<br>-<br>K, as well as any updates to these risk factors filed from time to time in PTC's o<br>ther filings with the SEC. You are urged to carefully consider all such<br>factors.<br>As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commerc<br>ial<br>ization of new products. There are no guarantees that any product will<br>receive or maintain regulatory approval in any territory, or prove to be commercially successful, including<br>Translarna<br>,<br>Emflaza<br>,<br>Upstaza<br>,<br>Evrysdi<br>,<br>Tegsedi<br>or<br>Waylivra<br>..<br>The forward<br>-<br>looking statements contained herein represent PTC's views only as of the date of this presentation and PTC does not<br>undertake or plan to update or revise any such forward<br>-<br>looking statements<br>to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occur<br>rin<br>g after the date of this presentation except as required by law.<br>2 | ||||||||
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| Commercialize<br>Providing patients with access<br>to transformative treatments<br>Develop<br>Enduring<br>innovation engine<br>PTC Continually Innovates to Bring New<br>Therapies to Patients<br>3<br>Discover<br>Proven<br>groundbreaking science | ||||||||
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| 4<br>Building a Pipeline to Produce a Therapy<br>Every 2<br>-<br>3 Years<br>Discover<br>Develop<br>2<br>to<br>3<br>years<br>New product every<br>Commercialize | ||||||||
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| PTC Has a Growing Global Footprint<br>5<br>1,300+<br>employees<br>20<br>offices<br>worldwide<br>Footprint<br>in<br>>50<br>countries<br>Founded in 1998 | ||||||||
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| Diversified Platform Drives Strong Portfolio<br>6<br>Clinical<br>Commercial<br>Research<br>Nonsense<br>Mutation<br>LatAm<br>Commercial<br>Deflazacort<br>Metabolic<br>Oncology<br>US<br>A<br>taluren<br>Emvododstat<br>AML<br>Unesbulin<br>DIPG<br>Unesbulin<br>LMS<br>PTC923 PKU<br>Emvododstat<br>COVID<br>-<br>19<br>Virology<br>SCIENTIFIC PLATFORMS and RESEARCH<br>SCA<br>-<br>3<br>MAP<br>-<br>Tau<br>PTC518 HD<br>Splicing<br>Bio<br>-<br>e<br>V<br>atiquinone<br>MDAS<br>V<br>atiquinone<br>FA<br>PTC857<br>ALS<br>3 Undisclosed<br>Gene<br>Therapy<br>Angelman<br>IRDs<br>Cog Disorders<br>FA<br>8 Undisclosed<br>AADC, aromatic L<br>-<br>amino acid decarboxylase deficiency; AML; acute myeloid leukemia; COVID<br>-<br>19, coronavirus disease 2019; DIPG, dif<br>fuse intrinsic pontine glioma; FA, Friedreich’s ataxia; ALS, amyotrophic lateral<br>sclerosis;<br>HD, Huntington‘s disease; IRD, inherited retinal dystrophy; LMS, leiomyosarcoma; MDAS, mitochondrial disease associated seizu<br>res<br>; PKU, phenylketonuria; SCA<br>-<br>3, spinocerebellar ataxia type 3.<br>Potential registrational studies<br>Early<br>-<br>stage programs<br>3 Undisclosed<br>2 Undisclosed | ||||||||
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| Multiple Platforms Provide Opportunity to<br>Target Over 700,000 Patients by 2030<br>7<br>ALS<br>+ ~1<br>50K<br>DMD/SMA<br>/<br>AADC<br>+ ~<br>50K<br>Mito<br>Sz<br>+ ~20<br>K<br>PKU<br>+ ~<br>58K<br>FA<br>+ ~<br>25K<br>HD<br>+ ~<br>135K<br>2021<br>2030<br>=10,000 pts<br>GBA<br>-<br>PD<br>+ ~<br>190K<br>AS<br>+ ~75K<br>~<br>50K<br>~<br>70K<br>~<br>128K<br>~<br>153K<br>~<br>288K<br>~<br>438K<br>~<br>628K<br>~<br>703K<br>Estimated Global Prevalence | ||||||||
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| 8<br>Enduring Innovation Drives Value Creation<br>2026<br>Key Drivers:<br>Evrysdi<br>®<br>,<br>PTC<br>-<br>AADC<br>,<br>Tegsedi<br>®<br>,<br>Waylivra<br>®<br>,<br>Translarna<br>™<br>,<br>Vatiquinone<br>,<br>PTC923,<br>Unesbulin<br>$539M<br>2021<br>Vatiquinone<br> | <br>PTC923<br> | <br>PTC518<br> | <br>PTC857<br> | <br>Unesbulin<br> | <br>Emvododstat<br> | <br>GT<br>-<br>FA<br> | <br>GT<br>-<br>AS<br> | <br>Research<br>2030<br>Key Drivers:<br>Evrysdi<br>®<br>,<br>PTC<br>-<br>AADC<br>,<br>Tegsedi<br>®<br>,<br>Waylivra<br>®<br>,<br>Vatiquinone<br>, PTC923,<br>Unesbulin<br>, PTC518, PTC857<br>~$3B<br>~$8B<br>Commercial<br>Clinical<br>Total revenue<br>Potential future revenues |
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| Revenue Contribution of Our Pipeline Grows<br>9<br>Potential future revenues<br>Commercial Products + Royalties<br>Current Pipeline<br>57<br>%<br>43<br>%<br>2026<br>2025<br>64<br>%<br>36<br>%<br>2024<br>2023<br>88<br>%<br>12<br>%<br>100<br>% | ||||||||
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| Strong Financial Performance Supports Innovation<br>10<br>Total<br>Revenue<br>$539<br>M<br>DMD<br>Franchise<br>Net Product<br>Revenue<br>$423<br>M<br>*Non<br>-<br>GAAP measure which excludes<br>estimated non<br>-<br>cash, stock<br>-<br>based compensation expense of approximately $115 million.<br>GAAP R&D and<br>SG&A expense for the full year<br>2022 is anticipated to be between $915 and $965 million.<br>Total<br>Revenue<br>Guidance<br>$475<br>-<br>495<br>M<br>OPEX<br>Guidance*<br>$800<br>-<br>850<br>M<br>$700<br>-<br>750<br>M<br>DMD Franchise<br>Net Product<br>Revenue Guidance | ||||||||
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| Diversified Platform Drives Strong Portfolio<br>11<br>Clinical<br>Commercial<br>Research<br>Nonsense<br>Mutation<br>LatAm<br>Commercial<br>Deflazacort<br>Metabolic<br>Oncology<br>US<br>A<br>taluren<br>Emvododstat<br>AML<br>Unesbulin<br>DIPG<br>Unesbulin<br>LMS<br>PTC923 PKU<br>Emvododstat<br>COVID<br>-<br>19<br>Virology<br>SCIENTIFIC PLATFORMS and RESEARCH<br>SCA<br>-<br>3<br>MAP<br>-<br>Tau<br>PTC518 HD<br>Splicing<br>Bio<br>-<br>e<br>V<br>atiquinone<br>MDAS<br>V<br>atiquinone<br>FA<br>PTC857<br>ALS<br>3 Undisclosed<br>Gene<br>Therapy<br>Angelman<br>IRDs<br>Cog Disorders<br>FA<br>8 Undisclosed<br>AADC, aromatic L<br>-<br>amino acid decarboxylase deficiency; AML; acute myeloid leukemia; COVID<br>-<br>19, coronavirus disease 2019; DIPG, dif<br>fuse intrinsic pontine glioma; FA, Friedreich’s ataxia; ALS, amyotrophic lateral<br>sclerosis;<br>HD, Huntington‘s disease; IRD, inherited retinal dystrophy; LMS, leiomyosarcoma; MDAS, mitochondrial disease associated seizu<br>res<br>; PKU, phenylketonuria; SCA<br>-<br>3, spinocerebellar ataxia type 3.<br>Potential registrational studies<br>Early<br>-<br>stage programs<br>3 Undisclosed<br>2 Undisclosed | ||||||||
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| Success Across Our Commercial Portfolio<br>12<br>•<br>Treatment for<br>nonsense<br>mutation DMD<br>for ages 2<br>and older<br>•<br>Distributed in<br>50+ countries<br>•<br>New patients<br>in existing<br>geographies<br>and geographic<br>expansion<br>•<br>First and only<br>corticosteroid<br>approved for DMD;<br>approved for all<br>US DMD patients<br>>2yrs<br>•<br>Data show clinical<br>benefit over<br>prednisone<br>•<br>New patient starts,<br>favorable access,<br>high compliance,<br>and appropriate<br>weight<br>-<br>based<br>dosing<br>•<br>Evrysdi<br>now<br>approved in<br>85 countries<br>•<br>Continued strong<br>uptake in the<br>U.S. >20%<br>market share<br>•<br>Potential for<br>$300M in sales<br>-<br>based milestones<br>•<br>Innovative<br>treatment for<br>hATTR<br>amyloidosis<br>patients<br>•<br>Disease awareness<br>and patient ID<br>continuing<br>•<br>LATAM patients<br>benefiting through<br>early<br>-<br>access<br>programs<br>•<br>Fulfilled first group<br>purchase order<br>in Q2<br>•<br>For treatment of<br>familial<br>chylomicronemia<br>syndrome (FCS)<br>•<br>LATAM patients<br>benefiting through<br>early<br>-<br>access<br>programs<br>•<br>Regulatory<br>decision on<br>FPL indication<br>expected in 2H22<br>•<br>First approved<br>disease<br>-<br>modifying<br>treatment<br>for<br>AADC<br>deficiency for<br>patients 18<br>months and older<br>•<br>First marketed<br>gene therapy<br>directly infused<br>into the brain<br>•<br>Approved by<br>EMA in July | ||||||||
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| 13<br>Continued Strong DMD Franchise Growth<br>Net Product Revenue (USD Millions)<br>34<br>81<br>29<br>92<br>101<br>139<br>188<br>145<br>171<br>190<br>192<br>236<br>2018<br>2020<br>2015<br>2016<br>2017<br>2019<br>291<br>2021<br>174<br>263<br>331<br>423<br>+52%<br>Emflaza<br>Translarna | ||||||||
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| Disease<br>Aromatic L<br>-<br>amino acid decarboxylase deficiency<br>(AADC<br>-<br>d) is a rare, highly morbid, and fatal childhood<br>disease. Children with severe AADC deficiency never<br>achieve motor development milestones.<br>Current Treatments<br>Upstaza is the first and only approved<br>disease<br>-<br>modifying therapy for AADC<br>-<br>d<br>and will become the standard of care.<br>Mechanism of Action<br>Upstaza is the first marketed gene therapy directly<br>infused into the brain.<br>14<br>~5,000<br>Global<br>Prevalence<br>Upstaza<br>™<br>Has the Potential to<br>Provide Significant Benefit to<br>AADC Deficiency Patients | ||||||||
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| 15<br>Upstaza<br>Gene<br>Therapy for AADC Deficiency<br>Approved by European Commission<br>•<br>Potential over $1B in<br>cumulative revenue<br>•<br>Successful patient<br>finding is ongoing<br>Approved by<br>EMA in July<br>•<br>PTC<br>-<br>AADC BLA<br>submission expected<br>in 4Q22<br>•<br>Development of virtual<br>education: disease<br>-<br>specific webinars and<br>congress symposia<br>•<br>Engaging with patient<br>advocacy groups<br>and payers<br>•<br>Identification and<br>preparation of expert<br>pediatric neurosurgical<br>centers<br>•<br>Continued KOL<br>engagement<br>Treatment Centers<br>Disease Education<br>Regulatory<br>Market Opportunity | ||||||||
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| Diversified Platform Drives Strong Portfolio<br>16<br>Clinical<br>Commercial<br>Research<br>Nonsense<br>Mutation<br>LatAm<br>Commercial<br>Deflazacort<br>Metabolic<br>Oncology<br>US<br>A<br>taluren<br>Emvododstat<br>AML<br>Unesbulin<br>DIPG<br>Unesbulin<br>LMS<br>PTC923 PKU<br>Emvododstat<br>COVID<br>-<br>19<br>Virology<br>SCIENTIFIC PLATFORMS and RESEARCH<br>SCA<br>-<br>3<br>MAP<br>-<br>Tau<br>PTC518 HD<br>Splicing<br>Bio<br>-<br>e<br>V<br>atiquinone<br>MDAS<br>V<br>atiquinone<br>FA<br>PTC857<br>ALS<br>3 Undisclosed<br>Gene<br>Therapy<br>Angelman<br>IRDs<br>Cog Disorders<br>FA<br>8 Undisclosed<br>AADC, aromatic L<br>-<br>amino acid decarboxylase deficiency; AML; acute myeloid leukemia; COVID<br>-<br>19, coronavirus disease 2019; DIPG, dif<br>fuse intrinsic pontine glioma; FA, Friedreich’s ataxia; ALS, amyotrophic lateral<br>sclerosis;<br>HD, Huntington‘s disease; IRD, inherited retinal dystrophy; LMS, leiomyosarcoma; MDAS, mitochondrial disease associated seizu<br>res<br>; PKU, phenylketonuria; SCA<br>-<br>3, spinocerebellar ataxia type 3.<br>Potential registrational studies<br>Early<br>-<br>stage programs<br>3 Undisclosed<br>2 Undisclosed | ||||||||
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| Substantial Pipeline Progress Planned<br>17<br>Study Results<br>Study Initiations<br>1Q 2023<br>3Q 2022<br>4Q 2022<br>1H 2022<br>2Q 2023<br>GT<br>-<br>FA<br>Ph1<br>Initiation<br>DIPG<br>Ph2<br>Initiation<br>Study 041<br>12<br>-<br>week data | ||||||||
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| 18<br>Data Expected<br>1<br>Q 2023<br>Vatiquinone<br>Mitochondrial Disease<br>Associated Seizures<br>Data Expected<br>2Q<br>2023<br>Vatiquinone<br>Friedreich Ataxia<br>Data Expected<br>YE 2022<br>PTC923<br>PKU<br>Three Registration<br>-<br>Directed Clinical Trials<br>Drive Near<br>-<br>Term Value | ||||||||
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| SCA<br>-<br>3<br>MAP<br>-<br>Tau<br>PTC518 HD<br>Splicing<br>Clinical<br>Commercial<br>Research<br>Nonsense<br>Mutation<br>LatAm<br>Commercial<br>Deflazacort<br>Metabolic<br>Oncology<br>US<br>A<br>taluren<br>Emvododstat<br>AML<br>Unesbulin<br>DIPG<br>Unesbulin<br>LMS<br>PTC923 PKU<br>Emvododstat<br>COVID<br>-<br>19<br>Virology<br>AADC, aromatic L<br>-<br>amino acid decarboxylase deficiency; AML; acute myeloid leukemia; COVID<br>-<br>19, coronavirus disease 2019; DIPG, dif<br>fuse intrinsic pontine glioma; FA, Friedreich’s ataxia; GBA, glucocerebrosidase;<br>HD, Huntington‘s disease; IRD, inherited retinal dystrophy; LMS, leiomyosarcoma; MDAS, mitochondrial disease associated seizu<br>res<br>; PD, Parkinson’s disease; PKU, phenylketonuria; SCA<br>-<br>3, spinocerebellar ataxia type 3.<br>SCIENTIFIC PLATFORMS and RESEARCH<br>Potential registrational studies<br>Early<br>-<br>stage programs<br>Gene<br>Therapy<br>Angelman<br>IRDs<br>Cog Disorders<br>FA<br>PTC<br>-<br>AADC<br>3 Undisclosed<br>2 Undisclosed<br>8 Undisclosed<br>Diversified Platform Drives Strong Portfolio<br>19<br>Reducing Oxidative Stress<br>Inhibition of 15 lipoxygenase<br>(15<br>-<br>LO) blocks inflammation<br>and oxidative stress response<br>Bio<br>-<br>e<br>V<br>atiquinone<br>MDAS<br>V<br>atiquinone<br>FA<br>PTC857<br>ALS<br>Science of Bio<br>-<br>e<br>•<br>Differentiated therapy for<br>neurological indications<br>•<br>High levels of oxidative stress and<br>inflammation underpin multiple<br>CNS disease pathologies<br>•<br>Bio<br>-<br>e platform targets the key<br>enzymatic hubs that regulate<br>oxidative stress and inflammatory<br>pathways to modulate disease<br>progression<br>3 Undisclosed | ||||||||
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| 20<br>MIT<br>-<br>E:<br>Registration<br>-<br>directed trial<br>of<br>vatiquinone<br>for<br>Mitochondrial<br>Disease Associated<br>Seizures | ||||||||
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| Disease<br>Mitochondrial disease associated seizures (MDAS)<br>is<br>the highly morbid condition of refractory seizures in<br>patients with inherited mitochondrial disease<br>Current Treatments<br>No approved disease<br>-<br>modifying treatments<br>Mechanism of Action<br>Vatiquinone<br>targets 15<br>-<br>lipoxygenase, a regulator<br>of the key energetic and oxidative stress pathways<br>that underpin seizures in these patients<br>21<br>~20,000<br>Global<br>Prevalence<br>Vatiquinone<br>Has the Potential to<br>Show Clinically Differentiated<br>Improvement for MDAS Patients | ||||||||
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| Vatiquinone<br>Has the Potential to<br>Show Clinically Differentiated<br>Improvement for MDAS Patients<br>22<br>Primary Endpoint<br>Cha<br>nge<br>from baseline<br>in frequency of observable<br>motor seizures<br>PTC743 (<br>vatiquinone<br>)<br>Placebo<br>N = 30<br>24 weeks<br>R<br>N = 30<br>48 weeks<br>PTC743 (<br>vatiquinone<br>)<br>N = 60<br>Open<br>-<br>Label Extension<br>Placebo<br>-<br>Controlled<br>Primary Endpoint<br>Trial Status<br>•<br>Enrolling<br>•<br>Data expected<br>1Q<br>2023 | ||||||||
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| 23<br>MOVE<br>-<br>FA:<br>Registration<br>-<br>directed<br>trial<br>of<br>vatiquinone<br>for<br>Friedreich Ataxia | ||||||||
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| Disease<br>Friedreich ataxia (FA)<br>is a rare,<br>inherited,<br>progressive disease resulting from<br>mitochondrial dysfunction<br>Current Treatments<br>No approved disease<br>-<br>modifying therapies<br>Opportunity<br>Vatiquinone targets 15<br>-<br>lipoxygenase, a regulator of<br>key energetic and oxidative stress pathways that<br>are disrupted in FA<br>24<br>~25,000<br>Global<br>Prevalence<br>Vatiquinone<br>Has the Potential<br>to Provide Improvement<br>in Neurological Function | ||||||||
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| Vatiquinone<br>Has the Potential<br>to Provide Improvement<br>in Neurological Function<br>25<br>Primary Endpoint<br>Change in<br>mFARS<br>Key Secondary Endpoint<br>Change in FA<br>-<br>ADL<br>Open<br>-<br>Label Extension<br>Placebo<br>-<br>Controlled<br>PTC743 (<br>vatiquinone<br>)<br>Placebo<br>N = ~<br>60<br>72 weeks<br>R<br>N = ~<br>60<br>24 weeks<br>PTC743 (<br>vatiquinone<br>)<br>N = ~120<br>Primary Endpoint<br>Trial Status<br>•<br>Data expected in 2Q 2023<br>Enrollment complete | ||||||||
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| 26<br>CardinALS<br>:<br>Phase 2<br>trial of PTC857<br>for<br>Amyotrophic Lateral<br>Sclerosis | ||||||||
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| 27<br>Disease<br>Amyotrophic lateral sclerosis (ALS) is a rapidly<br>progressing neurodegenerative disease caused by<br>oxidative damage which leads to neuronal cell<br>death and muscular atrophy<br>Current Treatments<br>No approved disease<br>-<br>modifying therapies<br>Mechanism of Action<br>PTC857 inhibits pathways leading to oxidative<br>damage and ferroptosis, resulting in protection of<br>motor neurons<br>~<br>150,000<br>Global<br>Prevalence<br>PTC857 Has the Potential<br>to Slow Disease Progression<br>in ALS | ||||||||
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| Primary Endpoints<br>Change in ALSFRS<br>-<br>R<br>Secondary Endpoints<br>Safety and PK<br>28<br>Treatment Period<br>PTC857 250 mg BID<br>Placebo<br>24<br>weeks<br>Primary Endpoint<br>R<br>Long<br>-<br>Term<br>Treatment Extension<br>28 weeks<br>PTC857 250 mg BID<br>Trial Status<br>•<br>Initiated in<br>1Q 2022<br>Screening<br>8<br>weeks<br>PTC857 Has the Potential<br>to Slow Disease Progression<br>in ALS<br>N =<br>~85<br>N =<br>~170 | ||||||||
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| Clinical<br>Commercial<br>Research<br>Nonsense<br>Mutation<br>LatAm<br>Commercial<br>Deflazacort<br>Metabolic<br>Oncology<br>US<br>A<br>taluren<br>Emvododstat<br>AML<br>Unesbulin<br>DIPG<br>Unesbulin<br>LMS<br>PTC923 PKU<br>Emvododstat<br>COVID<br>-<br>19<br>Virology<br>AADC, aromatic L<br>-<br>amino acid decarboxylase deficiency; AML; acute myeloid leukemia; COVID<br>-<br>19, coronavirus disease 2019; DIPG, dif<br>fuse intrinsic pontine glioma; FA, Friedreich’s ataxia; GBA, glucocerebrosidase;<br>HD, Huntington‘s disease; IRD, inherited retinal dystrophy; LMS, leiomyosarcoma; MDAS, mitochondrial disease associated seizu<br>res<br>; PD, Parkinson’s disease; PKU, phenylketonuria; SCA<br>-<br>3, spinocerebellar ataxia type 3.<br>SCIENTIFIC PLATFORMS and RESEARCH<br>Potential registrational studies<br>Early<br>-<br>stage programs<br>Bio<br>-<br>e<br>V<br>atiquinone<br>MDAS<br>V<br>atiquinone<br>FA<br>PTC857<br>ALS<br>Gene<br>Therapy<br>Angelman<br>IRDs<br>Cog Disorders<br>FA<br>PTC<br>-<br>AADC<br>Diversified Platform Drives Strong Portfolio<br>3 Undisclosed<br>29<br>3 Undisclosed<br>2 Undisclosed<br>8 Undisclosed<br>0<br>SCA<br>-<br>3<br>MAP<br>-<br>Tau<br>PTC518 HD<br>Splicing<br>Splicing<br>•<br>Pioneers in splicing<br>•<br>Small molecule regulation<br>of splicing events<br>•<br>Chemistry optimized<br>for uniform distribution,<br>blood brain barrier<br>penetration and<br>limited efflux<br>5’ Splice Site<br>3’ Splice Site<br>Isoform plex<br>HTSpliceseq<br>Databases of<br>Splicing Targets<br>Isoform plex<br>HTSpliceseq<br>Databases of<br>Splicing Targets<br>Leaders in splicing technology | ||||||||
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| 30<br>PIVOT HD:<br>PTC518 for<br>Huntington's<br>Disease | ||||||||
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| 31<br>Disease<br>Huntington’s disease (HD) is a progressive<br>brain disorder that causes uncontrolled<br>movements and cognitive loss<br>Current Treatments<br>No approved disease<br>-<br>modifying therapies<br>Mechanism of Action<br>PTC518 modulates splicing to induce<br>degradation of HTT mRNA, reducing<br>expression of the toxic HTT protein<br>~135,000<br>Global<br>Prevalence<br>PTC518 Reduces HTT mRNA<br>and Protein to Target the<br>Proximal Cause of HD | ||||||||
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| Crosses blood brain<br>barrier and is not<br>effluxed<br>Dose<br>-<br>dependent<br>reduction of<br>HTT mRNA and protein<br>Consistent<br>pharmacology<br>Generally<br>well tolerated<br>32<br>Results from Phase 1 Healthy Volunteer Study<br>PTC518 Reduces HTT mRNA and Protein to<br>Target the Proximal Cause of HD | ||||||||
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| 33<br>PTC518 Has the Potential to Reduce HTT<br>Protein in Huntington Patients<br>Trial Status<br>•<br>Initiated in 1Q 2022<br>3 months<br>9 months<br>5 mg PTC518<br>10 mg PTC518<br>Alternative Dose PTC518<br>R<br>Primary Endpoint<br>N = ~30<br>N = ~<br>30<br>N = ~<br>30<br>Part A<br>Part B<br>•<br>Safety and tolerability of<br>PTC518 in Huntington<br>disease patients<br>•<br>Percent reduction in HTT<br>mRNA and protein in blood<br>Primary endpoints<br>Placebo<br>5 mg PTC518<br>10 mg PTC518<br>Alternative Dose PTC518 | ||||||||
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| 34<br>•<br>Safety and tolerability of<br>PTC518 in Huntington’s<br>disease patients<br>•<br>Percent reduction in HTT<br>mRNA and protein in blood<br>Primary Endpoints<br>•<br>Percent reduction in HTT<br>protein in CSF<br>•<br>Changes in neurofilament light<br>chain (<br>NfL<br>) in plasma and CSF<br>•<br>Change in caudate,<br>putamenal<br>,<br>ventricular volume on<br>volumetric MRI imaging<br>•<br>Changes in clinical<br>scales of motor and<br>cognitive function<br>Secondary Endpoints<br>•<br>Ambulatory Huntington’s<br>patients ages 25 and older<br>•<br>CAG repeats 42<br>-<br>50 inclusive<br>•<br>Motor and Cognitive Function:<br>•<br>UHDRS<br>-<br>IS score of 100<br>•<br>UHDRS TFC score of 13<br>•<br>PIN<br>HD<br>score 0.18<br>-<br>4.93<br>•<br>Multivariate calculation<br>including SDMT, TMS, age,<br>CAG<br>Inclusion Criteria<br>PIVOT HD Trial Target Population and<br>Endpoints | ||||||||
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| 35<br>APHENITY:<br>Registration<br>-<br>directed<br>trial<br>of PTC923 for<br>PKU | ||||||||
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| 36<br>Disease<br>Phenylketonuria (PKU) is a metabolic condition caused<br>by mutations to phenylalanine hydroxylase that<br>can lead to cognitive disabilities and seizures<br>Current Treatments<br>Majority of patients do not initially respond or are<br>not well controlled by standard of care<br>Mechanism of Action<br>PTC923 is a more bioavailable precursor<br>than exogenously administered synthetic<br>BH4 and has the potential to treat a broader<br>range of PKU patients<br>~58,000<br>Global<br>Prevalence<br>APHENITY<br>Is a Global<br>Registration<br>-<br>Directed Trial<br>of PTC923 for<br>PKU | ||||||||
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| APHENITY<br>Is a Global<br>Registration<br>-<br>Directed Trial<br>of PTC923 for<br>PKU<br>37<br>Primary Endpoint<br>Reduction in<br>blood<br>phenylalanine<br>levels<br>Primary Endpoint<br>Trial Status<br>•<br>Data expected YE 2022<br>R<br>PTC923<br>Placebo<br>42 Days<br>Open<br>-<br>Label Extension<br>Placebo<br>-<br>Controlled<br>PTC923<br>12 Months<br>PTC923<br>14<br>Days<br>Run<br>-<br>in | ||||||||
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| Clinical<br>Commercial<br>Research<br>Nonsense<br>Mutation<br>LatAm<br>Commercial<br>Deflazacort<br>Metabolic<br>US<br>A<br>taluren<br>PTC923 PKU<br>Emvododstat<br>COVID<br>-<br>19<br>Virology<br>AADC, aromatic L<br>-<br>amino acid decarboxylase deficiency; AML; acute myeloid leukemia; COVID<br>-<br>19, coronavirus disease 2019; DIPG, dif<br>fuse intrinsic pontine glioma; FA, Friedreich’s ataxia; GBA, glucocerebrosidase;<br>HD, Huntington‘s disease; IRD, inherited retinal dystrophy; LMS, leiomyosarcoma; MDAS, mitochondrial disease associated seizu<br>res<br>; PD, Parkinson’s disease; PKU, phenylketonuria; SCA<br>-<br>3, spinocerebellar ataxia type 3.<br>SCIENTIFIC PLATFORMS and RESEARCH<br>Potential registrational studies<br>Early<br>-<br>stage programs<br>SCA<br>-<br>3<br>MAP<br>-<br>Tau<br>PTC518 HD<br>Splicing<br>Bio<br>-<br>e<br>V<br>atiquinone<br>MDAS<br>V<br>atiquinone<br>FA<br>PTC857<br>ALS<br>Diversified Platform Drives Strong Portfolio<br>38<br>3 Undisclosed<br>8 Undisclosed<br>Gene<br>Therapy<br>Angelman<br>IRDs<br>Cog Disorders<br>FA<br>PTC<br>-<br>AADC<br>2 Undisclosed<br>Oncology<br>Emvododstat<br>AML<br>Unesbulin<br>DIPG<br>Unesbulin<br>LMS<br>•<br>Niche oncology indications<br>•<br>Small molecule inhibitors of<br>cell proliferation:<br>•<br>Unesbulin<br>disrupts tubulin<br>function<br>•<br>Emvododstat<br>inhibits<br>pyrimidine biosynthesis<br>•<br>Targeting rare and difficult to<br>treat cancers with poor<br>prognosis<br>Oncology<br>3 Undisclosed | ||||||||
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| 39<br>Disease<br>Leiomyosarcoma (LMS) is a rare and aggressive<br>cancer with tumors found in smooth muscle<br>Current Treatments<br>Several chemotherapeutics are utilized but offer<br>minimal meaningful clinical benefit<br>Mechanism of Action<br>Unesbulin is an oral small molecule tubulin inhibitor<br>that arrests tumor cells in G2/M phase, including<br>cancer stem cells by inhibiting tubulin<br>polymerization<br>~<br>4,000<br>Diagnosed<br>annually<br>in US<br>Unesbulin<br>Has the Opportunity to<br>Provide Additional Progression<br>-<br>Free Survival in LMS<br>Not an actual LMS patient. | ||||||||
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| 40<br>Phase 1b study results<br>300 mg was selected as RP2D<br>Unesbulin<br>was well tolerated<br>21<br>Day Treatment Cycles<br>Phase 1b Study Design<br>Ascending doses 200, 300 and 400 mg<br>u<br>nesbulin<br>+ 1000mg/m<br>2<br>dacarbazine<br>N = 29<br>•<br>Patients with locally advanced<br>or metastatic LMS intolerant or<br>refractory to standard therapy<br>Inclusion Criteria<br>•<br>Any number of previous lines<br>of treatments allowed<br>Unesbulin<br>Has the Opportunity to<br>Provide Additional Progression<br>-<br>Free Survival in LMS<br>SLD = sum of largest diameter<br>Percentage Change for<br>SLD<br>from Baseline | ||||||||
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| Unesbulin<br>Has the Opportunity to<br>Provide Additional Progression<br>-<br>Free Survival in LMS<br>R<br>300 mg<br>U<br>nesbulin<br>+ 1000mg/m<br>2<br>dacarbazine<br>Placebo + 1000 mg/<br>m<br>2<br>dacarbazine<br>Primary Endpoint<br>PFS as determined by RECIST<br>Secondary Endpoints<br>OS, ORR, DCR, DOR<br>21 Day Treatment Cycle<br>24<br>Months<br>Interim Analysis<br>N<br>≈<br>115<br>N<br>≈<br>230<br>41<br>Trial Status<br>•<br>Initiated in<br>1Q<br>2022 | ||||||||
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| 42<br>Enduring Innovation Drives Value Creation<br>2026<br>Key Drivers:<br>Evrysdi<br>®<br>,<br>Upstaza<br>™<br>,<br>Tegsedi<br>®<br>,<br>Waylivra<br>®<br>,<br>Translarna<br>™<br>,<br>Vatiquinone<br>,<br>PTC923,<br>Unesbulin<br>$539M<br>2021<br>Vatiquinone<br> | <br>PTC923<br> | <br>PTC518<br> | <br>PTC857<br> | <br>Unesbulin<br> | <br>Emvododstat<br> | <br>GT<br>-<br>FA<br> | <br>GT<br>-<br>AS<br> | <br>Research<br>2030<br>Key Drivers:<br>Evrysdi<br>®<br>,<br>Upstaza<br>™<br>,<br>Tegsedi<br>®<br>,<br>Waylivra<br>®<br>,<br>Vatiquinone<br>, PTC923,<br>Unesbulin<br>, PTC518, PTC857<br>~$3B<br>~$8B<br>Commercial<br>Clinical<br>Total revenue<br>Potential future revenues |
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