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PTC Therapeutics Provides an Update on Commercial Progress and R&D Pipeline at 40th Annual J.P. Morgan Healthcare Conference
PTC Therapeutics Provides an Update on Commercial Progress and R&D Pipeline at 40th Annual J.P. Morgan Healthcare Conference
January 10, 2022
Key 2021 Corporate Highlights:
- Unaudited net product revenue of
$429 million in 2021 representing 29% year-over-year growth. - Strong year-over-year growth for the Duchenne muscular dystrophy (DMD) franchise, with unaudited net product revenue of
$424 million for Translarna™ (ataluren) and Emflaza® (deflazacort) in 2021. Cumulative net product revenue for Translarna exceeds$1 billion and Emflaza exceeds$500 million , since respective launches. - Translarna growth was driven by new patients and high compliance in existing geographies and continued geographic expansion.
- Emflaza growth was due to continued new prescriptions, high compliance, less patient discontinuations and more favorable access.
- Evrysdi® (risdiplam) has shown continued rapid uptake in
the United States and is now approved in all major markets including theEuropean Union andJapan . Evrysdi is a product of the Spinal Muscular Atrophy (SMA) collaboration between PTC, theSMA Foundation and Roche. - Waylivra® (volanesorsen), the only treatment for familial chylomicronemia syndrome was approved by
Brazilian Health Regulatory Agency , Agência Nacional de Vigilância Sanitária (ANVISA), and both Waylivra and Tegsedi® (inotersen) received Category 1 classification from Câmara de Regulação doMercado de Medicamentos - CMED (Drug Market Regulation Chamber) inBrazil . Category 1 classification is given to innovative treatments that provide greater efficacy than the standard of care and allows for pricing in line with international markets. - PTC successfully advanced its clinical pipeline in 2021:
- APHENITY, a Phase 3 registration-directed trial of PTC923 in phenylketonuria (PKU) was initiated.
- Phase 1 healthy volunteer trials of PTC518 and PTC857 were completed.
- Advanced the oncology platform with the completion of Phase 1b studies of unesbulin in leiomyosarcoma and diffuse intrinsic pontine glioma (DIPG).
- Completed enrollment for the vatiquinone MOVE-FA registration-directed trial with results expected in the second quarter of 2023.
2022 Potential Key Value-Creating Milestones:
- Results from Study 041 for Translarna are expected mid-year 2022 and if positive could support
re-submission of a New Drug Application (NDA) to theFood and Drug Administration (FDA). - Results for MIT-E, the registration-directed study of vatiquinone in mitochondrial disease associated seizures, are expected in the fourth quarter of 2022.
- Results are expected by year end 2022 for the Phase 3 registration-directed study, APHENITY, for PTC923 in patients with PKU.
- From the splicing platform, the PIVOT-HD Phase 2 study of PTC518 in Huntington's disease patients is planned to initiate in the first quarter of 2022.
- From the Bio-e platform, the registration-directed CardinALS study of PTC857 in amyotrophic lateral sclerosis (ALS) patients is expected to be initiated in the second quarter of 2022.
- Progress in the gene therapy platform is anticipated in 2022:
- PTC expects an opinion from the
European Medicine Agency's (EMA)Committee for Medicinal Products for Human Use (CHMP) for PTC-AADC, the first gene therapy for AADC deficiency, inApril 2022 . - Submission of a Biologics License Application (BLA) to the FDA for PTC-AADC is expected in the second quarter of 2022.
Preliminary Unaudited 2021 Financial Results:
- Total unaudited net revenue for full year 2021 was approximately
$536 million . - Total unaudited net product revenue for full year 2021 was approximately
$429 million . - DMD franchise revenue for year end 2021 included net product revenue for Translarna of approximately
$236 million , with$70 million in revenue in the fourth quarter, and Emflaza of approximately$188 million , with$48 million in revenue in the fourth quarter. - PTC expects to report approximately
$107 million in 2021 collaboration and royalty revenue associated with Evrysdi. - PTC expects to report 2021 year-end cash, cash equivalents and marketable securities of approximately
$773 million .
PTC is currently in the process of finalizing its financial results for the 2021 fiscal year. The above information is based on preliminary unaudited information and management estimates for the full year 2021, subject to the completion of PTC's financial closing procedures.
2022 Financial Guidance:
- PTC anticipates total revenues for the full year 2022 to be between
$700 and$750 million . - PTC anticipates net product revenues for the DMD franchise for the full year 2022 to be between
$475 and$495 million . - PTC anticipates GAAP R&D and SG&A expense for the full year 2022 to be between
$915 and$965 million . - PTC anticipates Non-GAAP R&D and SG&A expense for the full year 2022 to be between
$800 and$850 million , excluding estimated non-cash, stock-based compensation expense of$115 million .
Non-GAAP Financial Measures:
In this press release, the financial results and financial guidance of PTC are provided in accordance with GAAP and using certain non-GAAP financial measures. In particular, the non-GAAP financial measures exclude non-cash, stock-based compensation expense. These non-GAAP financial measures are provided as a complement to financial measures reported in GAAP because management uses these non-GAAP financial measures when assessing and identifying operational trends. In management's opinion, these non-GAAP financial measures are useful to investors and other users of PTC's financial statements by providing greater transparency into the historical and projected operating performance of PTC and the company's future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. Quantitative reconciliations of the non-GAAP financial measures to their respective closest equivalent GAAP financial measures are included in the table below.
|
|
||||||||
|
Low End of Range |
High End of Range |
|||||||
|
Projected GAAP R&D and SG&A Expense |
$ |
915,000 |
$ |
965,000 |
||||
|
Less: projected non-cash, stock-based compensation expense |
115,000 |
115,000 |
||||||
|
Projected non-GAAP R&D and SG&A expense |
$ |
800,000 |
$ |
850,000 |
||||
About
PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to innovate to identify new therapies and to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines. PTC's mission is to provide access to best-in-class treatments for patients who have little to no treatment options. PTC's strategy is to leverage its strong scientific and clinical expertise and global commercial infrastructure to bring therapies to patients. PTC believes this allows it to maximize value for all its stakeholders. To learn more about PTC, please visit us at www.ptcbio.com and follow us on Instagram, Facebook, Twitter, and LinkedIn.
For More Information:
Investors
+1 (908) 300-0691
kokeefe@ptcbio.com
Media
+1 (908) 912-9406
jclemente@ptcbio.com
Forward Looking Statements:
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historic fact, are forward-looking statements, including the information provided under the heading "2022 Financial Guidance", including with respect to (i) 2022 total revenue guidance, (ii) 2022 net product revenue guidance for the DMD franchise and (iii) 2022 GAAP and non-GAAP R&D and SG&A expense guidance, and statements regarding: the future expectations, plans and prospects for PTC, including with respect to the expected timing of clinical trials and studies, availability of data, regulatory submissions and responses and other matters; expectations with respect to PTC's gene therapy platform, including any regulatory submissions and manufacturing capabilities; advancement of PTC's joint collaboration program in SMA, including any regulatory submissions, commercialization or royalty or milestone payments; PTC's expectations with respect to the licensing, regulatory submissions and commercialization of its products and product candidates; PTC's strategy, future operations, future financial position, future revenues, projected costs; and the objectives of management. Other forward-looking statements may be identified by the words, "guidance", "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: expectations with respect to the COVID-19 pandemic and related response measures and their effects on PTC's business, operations, clinical trials, regulatory submissions and approvals, and PTC's collaborators, contract research organizations, suppliers and manufacturers; the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; expectations with respect to PTC's gene therapy platform, including any regulatory submissions and potential approvals, manufacturing capabilities and the potential financial impact and benefits of its leased biologics manufacturing facility and the potential achievement of development, regulatory and sales milestones and contingent payments that PTC may be obligated to make; the enrollment, conduct, and results of ongoing studies under the SMA collaboration and events during, or as a result of, the studies that could delay or prevent further development under the program, including any regulatory submissions and commercialization with respect to Evrysdi; PTC's ability to utilize results from Study 041, a randomized, 18-month, placebo-controlled clinical trial of Translarna for the treatment of nmDMD followed by an 18-month open-label extension, to support a marketing approval for Translarna for the treatment of nmDMD in
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, Emflaza, Evrysdi, Tegsedi, Waylivra or PTC-AADC.
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.
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