- PTC to receive $1.0B in cash at closing -
- PTC is
eligible to receive up to $1.9B in
development, regulatory and sales milestones -
- PTC to
share profits in the U.S. and tiered double-digit royalties on
ex-U.S. net sales -
- Novartis will assume global
development, manufacturing and commercial responsibilities
following completion of placebo-controlled portion of ongoing
PIVOT-HD study -
- PTC will host a conference call on
Dec. 2, 2024, at 8:30 am EST-
WARREN,
N.J., Dec. 2, 2024 /PRNewswire/ -- PTC
Therapeutics, Inc. (NASDAQ: PTCT) announced today the signing of an
exclusive global license and collaboration agreement with Novartis
Pharmaceuticals Corporation, a subsidiary of Novartis AG (NYSE:
NVS), for its PTC518 Huntington's disease program, which includes
related molecules. Under the agreement, PTC will receive an upfront
payment of $1.0 billion, up to
$1.9 billion in development,
regulatory and sales milestones, a profit share in the U.S., and
double-digit tiered royalties on ex-U.S. sales.
"PTC518 is the leading oral disease-modifying therapy in
development for Huntington's disease and the economics of this
agreement are consistent with the promise of this treatment," said
Matthew B. Klein, M.D., Chief
Executive Officer, PTC Therapeutics. "This collaboration combines
PTC's expertise in developing small molecule splicing therapies
with Novartis's expertise in global development and
commercialization of neuroscience therapies. We are excited to
collaborate with Novartis to accelerate the potential of PTC518 for
the hundreds of thousands of HD patients worldwide in need of a
therapy designed to be well-tolerated and an effective
disease-modifying therapy. PTC will use the proceeds of this
transaction to expand our splicing platform as well as to support
commercial and development portfolio activities."
"Huntington's Disease is a devastating, fatal, familial disease.
This agreement with PTC is intended to bolster our neuroscience
pipeline and reflects our strategic focus and commitment to explore
new and potentially transformative approaches for neurodegenerative
diseases with high unmet needs," said Vas Narasimhan, CEO of
Novartis. "We look forward to building on our expertise in
neurodegenerative diseases and experience in HD with the intention
to advance this potential first in class oral therapy for the HD
community."
PTC518 was discovered from PTC's validated splicing platform and
is currently being studied in the ongoing Phase 2 PIVOT-HD trial.
Interim results reported in June 2024
demonstrated that PTC518 treatment resulted in durable,
dose-dependent reduction in blood and cerebrospinal fluid (CSF)
mutant Huntingtin protein (HTT) levels as well as early signals of
dose-dependent benefit on key clinical measurements at 12
months.1 Importantly, PTC518 continues to demonstrate a
favorable safety and tolerability profile.1
Novartis will assume responsibility for PTC518's development,
manufacturing and commercialization, following the completion of
the on-going placebo-controlled portion of PIVOT-HD, which is
expected to occur in H1 2025.
The companies will share U.S. profits and losses, on a 40/60
basis (40% PTC and 60% Novartis).
The closing of the transaction is subject to customary closing
conditions, including regulatory clearance. The parties anticipate
that the agreement will close in the first quarter of 2025.
Conference Call and Webcast Details:
PTC will hold a
conference call at 8:30 am EST 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 Huntington's Disease
Huntington's disease (HD)
is a fatal, hereditary, genetic disorder of the central nervous
system.2 It is caused by a defective gene. This gene
produces a protein, called Huntingtin, which is involved in the
functioning of the nerve cells in the brain (neurons). When the
gene is defective, it produces an abnormal (or mutated) Huntingtin
protein that is toxic and causes neuron damage and neuron
death.3 HD usually presents in people who are in their
30s or 40s. Symptoms can present earlier in life, and this is
called the Juvenile HD.3,4 There are also cases of
infantile HD, when symptoms develop in children who are younger
than 10 years old.3 While symptoms vary from person to
person, the disease primarily affects the brain and results in
abnormal movements, difficulties with speech, swallowing and
walking, as well as a number of other symptoms including
behavioral, cognitive and motor symptoms.5,6 While there
are therapies approved for specific disease symptoms, currently,
there is no cure for HD and there are no approved drugs that delay
the onset or slow disease progression.
About PTC's Splicing Platform
PTC has pioneered
the use of advanced alternative splicing technology to identify
small molecules that affect mRNA splicing for the
treatment of disease of high unmet need. PTC's
validated splicing platform identified the first-ever
approved small molecule splicing modifier -
Evrysdi® (risdiplam), and PTC
has leveraged the extensive learnings from the SMA
program to broaden the platform to support discovery programs
across numerous therapeutic areas including
neurodegenerative disease, oncology and metabolism.
PTC has also developed a powerful high-throughput drug
discovery platform (PTSeek™) that identifies small
molecules that modulate pre-mRNA splicing to
upregulate or down regulate targeted protein production,
accelerating the discovery and early preclinical development
process for candidate small molecule splicing agents.
About PTC Therapeutics, Inc.
PTC is a global
biopharmaceutical company focused on the discovery, development and
commercialization of clinically differentiated medicines that
provide benefits to children and adults living 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 X.
For More Information:
Investors:
Ellen Cavaleri
+1 (615) 618-6228
ecavaleri@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 with
respect to the future expectations, plans and prospects for
PTC, including with respect to PTC's right to
receive any upfront payment from Novartis;
PTC's right to receive development, regulatory and sales
milestones, profit sharing and royalty payments from
Novartis; the continued development of PTC518; 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 expected benefits and
opportunities related to the licensing agreement may not be
realized or may take longer to realize than expected due to a
variety of reasons, including any inability of the parties to
perform their commitments and obligations under the agreement,
challenges and uncertainties inherent in development; success in
early clinical trials, especially if based on a small patient
sample, does not ensure that later clinical trials will be
successful, and early results from a clinical trial do not
necessarily predict final results; data for PTC518 may not be
sufficient for obtaining regulatory approval; 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; 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.
References:
- PTC Therapeutics, "Interim PIVOT-HD Results
Demonstrate Evidence of Favorable CNS Biomarker and Clinical
Effects at Month 12 in Huntington's Disease Patients," news
release, June 20, 2024,
https://ir.ptcbio.com/news-releases/news-release-details/interim-pivot-hd-results-demonstrate-evidence-favorable-cns
- World Health Organization, 2020. 8A01.10 Huntington disease.
Available at:
https://icd.who.int/browse11/l-m/en#/http://id.who.int/icd/entity/2132180242 Accessed October
2021.
- Gatto EM, González Rojas N, Persi G, et al. Clin Parkinsonism
Rel Disord 2020;3:100056.
- Tabrizi SJ, Flower MD, Ross CA, et al. Nat Rev Neurol
2020;16(10):529–546.
- Roos RAC. Orphanet J Rare Dis 2010;5:40.
- Kirkwood SC, Su JL, Conneally P, et al. Arch Neurol
2001;58(2):273–278.
View original
content:https://www.prnewswire.com/news-releases/ptc-therapeutics-enters-into-a-global-license-and-collaboration-agreement-with-novartis-for-ptc518-huntingtons-disease-program-302319374.html
SOURCE PTC Therapeutics, Inc.