PTC Therapeutics Provides an Update on Commercial Progress and R&D Pipeline at 42nd Annual J.P. Morgan Healthcare Conference
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Insights
PTC Therapeutics' report of a 35% year-over-year growth in total revenue to $946 million indicates a robust financial performance, which is significant for stakeholders and potential investors. This performance is particularly notable given the broader biopharmaceutical industry's volatile market, where R&D costs and regulatory challenges often erode profitability. The company's strategic focus on its Duchenne muscular dystrophy (DMD) franchise, which saw a 23% increase in net product revenue, demonstrates an effective market penetration and product lifecycle management. The financial agility of PTC Therapeutics is further underscored by its ability to retire debt obligations following a lucrative royalty agreement with Royalty Pharma.
The projected revenue for 2024, however, indicates a potential contraction to between $600 million and $850 million, which may raise questions about market saturation, pricing pressures, or pipeline progression. The anticipated GAAP R&D and SG&A expenses ranging from $740 to $835 million suggest a continued investment in R&D, which is critical for sustaining long-term growth but may impact short-term profitability. The reconciliation of GAAP to non-GAAP measures, excluding non-cash stock-based compensation, provides a clearer picture of operational expenses and could be indicative of management's confidence in maintaining operational efficiency.
The clinical pipeline progress of PTC Therapeutics, especially the regulatory filings in the EU and US for sepiapterin in PKU, represents a significant milestone with a potential $1 billion commercial opportunity. The success in the Phase 3 APHENITY trial for sepiapterin in PKU patients demonstrates the company's capability in advancing its pipeline towards market approval. The strategic prioritization of the portfolio, with a focus on core therapies like PTC518 for Huntington's Disease (HD), is a calculated move to optimize R&D expenditure and focus on areas with high unmet medical needs.
Moreover, the upcoming data readouts for the PIVOT-HD study and the potential submissions for a Biologics License Application (BLA) for Upstaza in AADC deficiency are critical events that could significantly impact the company's valuation and future revenue streams. The outcomes of these events will likely be pivotal in determining the company's trajectory and the potential expansion of its market presence in the neurology space.
The positive financial results and promising pipeline updates from PTC Therapeutics are likely to influence investor sentiment positively. The company's strategic decisions, such as expanding geographically and optimizing its product portfolio, cater to the ever-evolving demands of the biopharmaceutical market. The growth in the DMD franchise and the anticipation of new treatments for PKU and HD could open up new market segments for the company.
Additionally, the strategic financial moves, including the royalty agreement with Royalty Pharma, demonstrate a forward-thinking approach to capital management. The emphasis on non-GAAP financial measures, excluding stock-based compensation, suggests a focus on operational efficiency that may appeal to investors who are concerned with the underlying performance of the company. The upcoming regulatory events and clinical trial readouts will be critical in shaping the market's perception of the company's growth potential and its ability to capitalize on its R&D investments.
– Unaudited 2023 total revenue of $946 million, representing
– Regulatory filings in the EU and US for sepiapterin in PKU, a potential
– Multiple study readouts planned for 2024, including 12-month interim data from the PIVOT-HD study of PTC518 in HD patients –
"2023 was a transformational year for PTC as we made necessary and important changes to become a leaner, more focused and financially stronger company positioned for future success," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "We look forward to a successful 2024, with anticipated key global regulatory submissions for our PKU program and multiple data readouts, including from our PTC518 HD program."
Key 2023 Corporate Highlights:
- Unaudited net product revenue of
$661 million in 2023 representing23% year-over-year growth.- Strong year-over-year growth for the Duchenne muscular dystrophy (DMD) franchise, with unaudited net product revenue of
$355 million for Translarna™ (ataluren) and$255 million for Emflaza® (deflazacort) in 2023.- Translarna growth was driven by new patients in existing geographies and continued geographic expansion.
- Emflaza growth was due to continued new prescriptions, high compliance, and more favorable access.
- Strong year-over-year growth for the Duchenne muscular dystrophy (DMD) franchise, with unaudited net product revenue of
- PTC announced positive readouts from key programs in its clinical pipeline in 2023:
- Highly statistically significant and clinically meaningful results in the Phase 3 registration-directed APHENITY clinical trial of sepiapterin in adult and pediatric patients with PKU.
- Achievement of all key objectives of the readout of 12-week interim data in the PIVOT-HD study of PTC518 in HD.
- PTC announced strategic portfolio prioritizations, which resulted in reductions in both operating expenses and headcount of approximately
25% and30% , respectively. - In October, PTC finalized a royalty agreement, in which Royalty Pharma acquired additional royalties of Evrysdi for
$1.0 billion $500 million $250 million $250 million
2024 Potential Key Clinical and Regulatory Events:
- Submission of a MAA to the EMA for sepiapterin for the treatment of PKU is expected in the first quarter.
- Submission of an NDA to the FDA for sepiapterin for the treatment of PKU is expected no later than the third quarter.
- Results from 12-month interim data from the PIVOT-HD trial of PTC518 in HD patients are expected in the second quarter.
- Submission of a BLA to the FDA for Upstaza for the treatment of AADC deficiency is expected in the first quarter.
- Topline results from the CardinALS trial of utreloxastat in ALS are expected in the fourth quarter.
- PTC expects the CHMP opinion from the re-examination procedure of the negative opinion on the Translarna conditional marketing authorization renewal in late January 2024, with ratification of that opinion by the European Commission 67 days later.
- FDA meeting for Translarna to align on the specific contents of a potential NDA resubmission is scheduled for the first quarter.
- FDA meeting for vatiquinone to discuss how the MOVE-FA data along with additional clinical and preclinical data could support an NDA submission in FA is scheduled for the first quarter.
- Scientific advice feedback from the EMA on a potential submission of vatiquinone for conditional marketing authorization for Friedreich ataxia is expected in the first quarter.
Unaudited 2023 Financial Results:
- Total unaudited net revenue for full-year 2023 was approximately
$946 million . - Total unaudited net product revenue for full-year 2023 was approximately
$661 million . - DMD franchise unaudited revenue for full-year 2023 was approximately
$610 million , including unaudited net product revenue for Translarna of approximately$355 million and for Emflaza of approximately$255 million . - PTC expects to report approximately
$278 million for full-year 2023 collaboration and royalty revenue associated with Evrysdi.
PTC is currently in the process of finalizing its financial results for the 2023 fiscal year. The above information is based on preliminary unaudited information and management estimates for the full year 2023, subject to the completion of PTC's financial closing procedures. Evrysdi royalty revenue estimates are based on internal estimates.
2024 Financial Guidance:
- PTC anticipates total revenues for the full-year 2024 to be between
$600 million and$850 million . - PTC anticipates GAAP R&D and SG&A expense for the full-year 2024 to be between
$740 and$835 million . - PTC anticipates Non-GAAP R&D and SG&A expense for the full year 2024 to be between
$660 and$755 million , including expected R&D expense milestone payments of up to$65 million $80 million . - PTC anticipates up to
$90 million of payments for the full year 2024 upon achievement of potential regulatory success-based milestones from previous acquisitions, of which up to$65 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.
PTC Therapeutics, Inc. (In millions) | ||
Low End of Range | High End of Range | |
Projected GAAP R&D and SG&A Expense | ||
Less: projected non-cash, stock-based compensation expense | 80 | 80 |
Projected non-GAAP R&D and SG&A expense | ||
PTC Therapeutics, Inc. (in millions) | |
Projected GAAP R&D Expense Related Milestone Payments | |
Projected GAAP Contingent Consideration Payable Related Milestone Payments | 25 |
Total Projected GAAP Milestone Payments | |
Acronyms:
AADC: Aromatic I-Amino Acid Decarboxylase
ALS: Amyotrophic Lateral Sclerosis
BLA: Biologics License Application
CHMP: Committee for Medicinal Products for Human Use
DMD: Duchenne Muscular Dystrophy
EMA: European Medicines Agency
FDA: Food and Drug Administration
MAA: Marketing Authorization Application
NDA: New Drug Application
PKU: Phenylketonuria
R&D: Research and Development
SG&A: Selling, General and Administrative
SMA: Spinal Muscular Atrophy
About PTC Therapeutics, Inc.
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
Kylie O'Keefe
+1 (908) 300-0691
kokeefe@ptcbio.com
Media
Jeanine Clemente
+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 "2024 Financial Guidance", including with respect to (i) 2024 total revenue guidance, (ii) 2024 GAAP and non-GAAP R&D and SG&A expense guidance and (iii) 2024 acquisition related milestone payment 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, commercialization and other matters with respect to its products and product candidates; PTC's strategy, future operations, future financial position, future revenues, projected costs; the extent, timing and financial aspects of our strategic pipeline prioritization and reductions in workforce; 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: 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; PTC's ability to maintain its marketing authorization of 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, Upstaza, Evrysdi, Tegsedi or Waylivra.
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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SOURCE PTC Therapeutics, Inc.
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