– Unaudited 2023 total revenue of
$946 million, representing 35% year-over-year growth
–
– Regulatory filings in the EU and
US for sepiapterin in PKU, a potential $1
billion global commercial opportunity, remain on track for
2024 –
– Multiple study readouts planned
for 2024, including 12-month interim data from the PIVOT-HD
study of PTC518 in HD patients –
SOUTH
PLAINFIELD, N.J., Jan. 8, 2024
/PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) will present
an update on its commercial progress and R&D pipeline at the
42nd Annual J.P. Morgan Healthcare Conference today, Monday, January 8, at 10:30am EST/7:30am
PST. Matthew B. Klein, M.D.,
Chief Executive Officer of PTC Therapeutics, will provide an
update on 2023 accomplishments and highlight potential 2024
value-creating milestones. Preliminary 2023 unaudited financial
results and 2024 financial guidance will also be provided. The
presentation is being webcast live on the Events and Presentations
page of the Investors section of PTC Therapeutics website at
www.ptcbio.com.
"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
representing 23% 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.
- 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% and 30%, respectively.
- In October, PTC finalized a royalty agreement, in which Royalty
Pharma acquired additional royalties of Evrysdi for $1.0 billion upfront. The agreement included
options for PTC to sell the remainder of its royalties of Evrysdi
for up to $500 million or for Royalty
Pharma to acquire half of such retained royalties for up to
$250 million at a later date, less
royalties received by PTC. PTC maintains all economics associated
with up to $250 million in the
remaining commercial sales milestones associated with Evrysdi
global net sales. The proceeds from the financing were used to
retire all outstanding debt obligations with Blackstone Life
Sciences.
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 and excluding estimated
non-cash, stock-based compensation expense of $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 will be recorded as
R&D operating expense.
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.
Reconciliation of GAAP to Non-GAAP Projected Full Year 2024
R&D and SG&A Expense
(In
millions)
|
|
|
Low End of
Range
|
High End of
Range
|
Projected GAAP R&D
and SG&A Expense
|
$ 740
|
$ 835
|
Less: projected
non-cash, stock-based compensation expense
|
80
|
80
|
Projected non-GAAP R&D and SG&A
expense
|
$ 660
|
$ 755
|
|
|
|
PTC Therapeutics, Inc.
Reconciliation of GAAP Milestone
Payments Full Year 2024
(in
millions)
|
|
Projected GAAP R&D
Expense Related Milestone Payments
|
$ 65
|
Projected GAAP
Contingent Consideration Payable Related Milestone
Payments
|
25
|
Total Projected GAAP Milestone
Payments
|
$ 90
|
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
Brazil, Russia, the European Economic Area (EEA) and
other regions, including whether the European Medicines Agency
(EMA) determines in the re-examination process that the
benefit-risk balance of Translarna authorization supports renewal
of such authorization; PTC's ability to use the results of 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, which was a specific obligation to continued
marketing authorization in the EEA, to support a renewal of the
conditional marketing authorization for Translarna for the
treatment of nmDMD in the EEA; PTC's ability to utilize results
from Study 041 to support a marketing approval for Translarna for
the treatment of nmDMD in the United
States; whether investigators agree with PTC's
interpretation of the results of clinical trials and the totality
of clinical data from our trials in Translarna; expectations with
respect to Upstaza, including any regulatory submissions and
potential approvals, commercialization, manufacturing capabilities,
the potential achievement of development, regulatory and sales
milestones and contingent payments that PTC may be obligated to
make; expectations with respect to the commercialization of Evrysdi
under our SMA collaboration; expectations with respect to the
commercialization of Tegsedi and Waylivra; the timing of and actual
expenses incurred in connection with the discontinuation of PTC's
preclinical and early research programs in gene therapy and
reductions in workforce, which may be in different periods and may
be materially higher than estimated; the savings that may result
from the discontinuation of PTC's strategic pipeline prioritization
and reductions in workforce, which may be materially less than
expected; significant business effects, including the effects of
industry, market, economic, political or regulatory conditions;
changes in tax and other laws, regulations, rates and policies; the
eligible patient base and commercial potential of PTC's products
and product candidates; PTC's scientific approach and general
development progress; the potential financial impact and benefits
of PTC's leased biologics manufacturing facility; PTC's ability to
satisfy its obligations under the terms of its lease agreements,
including for its leased biologics manufacturing facility; 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 and Annual Report on Form
10-K, as well as any updates to these risk factors filed from time
to time in PTC's other filings with the SEC. You are urged to
carefully consider all such factors.
As with any pharmaceutical under development, there are
significant risks in the development, regulatory approval and
commercialization of new products. There are no guarantees that any
product will receive or maintain regulatory approval in any
territory, or prove to be commercially successful, including
Translarna, 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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