- Preclinical and early research gene therapy
programs discontinued -
- Expected reductions of
approximately 15% in residual 2023 OPEX -
SOUTH
PLAINFIELD, N.J., May 23, 2023
/PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT)
announced today the discontinuation of preclinical and early
research programs in gene therapy as part of a strategic portfolio
prioritization. PTC will continue its development and global
commercialization of Upstaza, the first-ever approved gene therapy
directly administered to the brain. As a result of the
prioritization, estimated reductions of approximately fifteen
percent in residual 2023 operating expenses (OPEX) are expected.
Additional 2023 OPEX guidance will be provided as part of second
quarter earnings.
"We are incredibly proud of PTC's accomplishments in the field
of gene therapy, including the pioneering work associated with the
approval of Upstaza. We believe that the decision to discontinue
our pipeline gene therapy programs enables PTC to focus R&D
efforts on our other innovative and differentiated scientific
platforms and strongly positions us for long-term growth and
success," said Chief Executive Officer Dr. Matthew Klein. "Where possible, we will work to
ensure that the discontinued gene therapy programs can be developed
by other parties so that the therapies have the potential to
benefit patients."
The decision to deprioritize early-stage gene therapy programs
reflects the company's desire to focus resources on areas of the
pipeline likely to deliver significant return on investment and
transformative therapies for patients with high unmet medical need.
The discontinuation of the gene therapy programs will also result
in a reduction in workforce which will be conducted in accordance
with appropriate labor regulations.
The discontinued gene therapy programs include preclinical stage
programs in Friedreich ataxia and Angelman syndrome as well as
several other programs targeting rare CNS and ophthalmological
disorders of high unmet medical need at various stages of
preclinical development. The prioritization decision will allow for
additional focus on PTC's proprietary splicing platform as well as
additional CNS and metabolic disorders that leverage its
differentiated and innovative scientific expertise.
In addition, PTC Therapeutics also announced today that PTC's
Chief Financial Officer Emily Hill
has been relieved of her responsibilities and will be leaving the
organization.
Conference Call and Webcast Details:
PTC will hold a
conference call at 5:00 pm EDT today
to discuss this news. To access the call by phone, please click
here to register and you will be provided with dial-in details. To
avoid delays, we recommend participants dial in to the conference
call 15 minutes prior to the start of the call. The webcast
conference call can be accessed on the Investor section of the PTC
website at https://ir.ptcbio.com/events-presentations. A replay of
the call will be available approximately two hours after completion
of the call and will be archived on the company's website for 30
days following the call.
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 Facebook, Instagram, LinkedIn and Twitter at
@PTCBio.
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 Statement:
This press release contains forward-looking statements within
the meaning of The Private Securities Litigation Reform Act of
1995. All statements contained in this press release, other than
statements of historic fact, are forward-looking statements,
including statements regarding the extent, timing and financial
aspects of the discontinuation of our preclinical and early
research programs in gene therapy and reduction in workforce; 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, future 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: 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; the timing of and actual expenses
incurred in connection with the discontinuation of our preclinical
and early research programs in gene therapy and reduction in
workforce, which may be in different periods and may be materially
higher than we estimate; the savings that may result from the
discontinuation of our preclinical and early research programs in
gene therapy and reduction in workforce, which may be materially
less than we expect; 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; 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
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 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.
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.