Rapid advancement of PureTech’s Wholly Owned
Pipeline, with three clinical trials now underway and five
completed this year that demonstrated compelling safety and
tolerability data for LYT-100 (deupirfenidone) and proof of
principle, oral bioavailability and tolerability for LYT-300 (oral
allopregnanolone). Robust dose escalation and safety data also
announced from completed monotherapy portion of Phase 1 program for
LYT-200 (anti-galectin-9 mAb)
Catalyst-rich 2023 anticipated, with results
from late-stage trial of LYT-100 in idiopathic pulmonary fibrosis
and Phase 1b trial of LYT-200 in leukemia. Initiation of clinical
trials planned with LYT-300 and LYT-310 (oral cannabidiol)
targeting neurological conditions as well as the combination
portion of the Phase 1 trial of LYT-200 in solid tumors
Continued momentum across Founded Entities,
including Karuna’s positive Phase 3 results for KarXT in
schizophrenia, commercial progress for Gelesis’ Plenity® and
Akili’s EndeavorRx® and Vor’s promising initial data in acute
myeloid leukemia
Board composition and committee changes also
noted
PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) (“PureTech” or the
“Company”), a clinical-stage biotherapeutics company dedicated to
changing the treatment paradigm for devastating diseases, today
reported on the key progress made across its Wholly Owned Programs1
and Founded Entities2 in 2022.
Daphne Zohar, Founder and Chief Executive Officer of PureTech,
commented: “2022 has been a particularly successful and productive
year in both crystallizing value and shaping the future of
PureTech. Our primary focus is to progress our Wholly Owned
Pipeline to commercialization and deliver new classes of medicines
for patients with serious, debilitating conditions. We are
advancing toward this goal by deepening our commitment to programs
with compelling clinical data that are moving into late-stage
studies and that we believe have the potential for broad
impact.
“Over the next 12 months, we anticipate multiple important
catalysts that will further guide how we prioritize our pipeline,
informing our decisions regarding which programs we will drive to
commercial launches ourselves and which programs could be most
successfully advanced through other avenues such as a partnership,
sale, or spinout into another entity.”
PureTech’s Wholly Owned Programs are centered on developing
breakthrough medicines for patients with underserved and serious
diseases. The Company’s therapeutic candidates are being advanced
for the potential treatment of devastating conditions including
idiopathic pulmonary fibrosis (IPF), metastatic solid tumors,
leukemia and certain neurological and neuropsychological
indications.
Additionally, PureTech’s Founded Entities are advancing 20
therapeutics and therapeutic candidates, of which two (Plenity® for
weight management3 and EndeavorRx® for treating inattention in ADHD
ages 8-124) have received both U.S. FDA clearance and European
marketing authorization and a third (KarXT for schizophrenia) will
soon be filed for FDA approval, as of the most recent update by the
company. Key highlights include the following:
Key Wholly Owned Program
Updates
- LYT-100 (deupirfenidone) is in development for the
potential treatment of conditions involving inflammation and
fibrosis, including IPF, for which current standards of care are
associated with significant tolerability issues, resulting in
approximately three out of four patients in the U.S. foregoing
treatment with these otherwise efficacious medicines.5 LYT-100 is a
deuterated form of one of these treatments, pirfenidone, which has
proven efficacy and has been shown to improve survival in these
patients by approximately three years, but its side effects cause
patients to discontinue or dose reduce, thereby limiting its
effectiveness.6
- Announced results from a randomized, double-blind crossover
study in healthy older adults demonstrating that approximately 50%
fewer subjects treated with PureTech’s LYT-100 experienced
gastrointestinal-related adverse events compared to subjects
treated with pirfenidone.
- Initiated a global, randomized double blind placebo-controlled
trial of LYT-100 in patients with IPF. This trial is expected to
serve as the first of two registration-enabling trials, and topline
results are expected by the end of 2023.
- Initiated a preclinical study of LYT-100 for the prevention and
treatment of radiation induced fibrosis, an indication for which
the United States government stockpiles medical countermeasures.
This program is subject to the Animal Rule, which allows for the
approval of drugs based on validated animal models when human
efficacy studies are not feasible.7
- Completed a Phase 2a trial of LYT-100 in patients with breast
cancer-related, upper limb secondary lymphedema, which met the
primary endpoint of safety and tolerability, adding to the growing
body of data demonstrating a favorable tolerability profile for
LYT-100. Secondary endpoints assessed exploratory biomarkers
related to lymphedema, and the data did not provide support for a
clear development path for this indication. Given the Company’s
current priorities in IPF, where the regulatory path is
well-defined and there is a wealth of strong efficacy data with
pirfenidone, the Company has deprioritized lymphedema as an
indication.
- PureTech is also exploring the potential evaluation of LYT-100
in other inflammatory and fibrotic conditions, such as progressive
fibrosing interstitial lung diseases, myocardial and other organ
system fibrosis, based on the strength of existing clinical data
around the use of pirfenidone in these indications.
- LYT-200 (anti-galectin-9 mAb) is in development for the
potential treatment of metastatic solid tumors that have poor
survival rates as well as hematological malignancies, such as acute
myeloid leukemia (AML), where more than 50% of patients either
don’t respond to initial treatment or experience relapse after
responding to initial treatment.8
- Completed the bi-monthly and weekly monotherapy dose escalation
portion of the Phase 1 program assessing the safety and
tolerability of escalating doses of LYT-200 as a potential
treatment for metastatic solid tumors. No dose-limiting toxicities
were reported, and the full results will be presented in an
upcoming scientific forum. Evaluation of LYT-200 in combination
with tislelizumab is expected to begin in the first quarter of
2023.
- Shared new preclinical data supporting the clinical potential
of LYT-200 as a therapeutic agent for the treatment of leukemia in
a scientific poster at the American Society of Hematology (ASH)
64th Annual Meeting. The data presented support the role of
galectin-9 in multiple types of leukemia and the ability of
anti-galectin-9 antibodies to provide effective anti-tumor activity
in these cancers. Based on these and other compelling preclinical
data generated with LYT-200 in blood cancers, PureTech initiated a
clinical trial to evaluate LYT-200 as a single agent for the
treatment of AML, with results expected in 2023.
- Given the expansion of the clinical evaluation of LYT-200, the
Company has deprioritized preclinical development of
LYT-210.
- LYT-300 (oral allopregnanolone) is in development for
the potential treatment of neurological and neuropsychological
conditions where there is a need for more effective treatments that
work quickly, have more favorable tolerability and can be
administered conveniently.
- Completed the multi-part Phase 1 trial of LYT-300, which
demonstrated oral bioavailability in healthy adults, achieving
blood levels of allopregnanolone at or above those associated with
therapeutic effect and ninefold greater than orally administered
allopregnanolone, based on third-party published data.9 The data
also demonstrated target engagement with GABAA receptors, which are
known to regulate mood and other neurological conditions. LYT-300
was generally well-tolerated with no treatment-related severe or
serious adverse events observed. A Phase 1b/2a trial is expected to
begin in the first half of 2023.
- LYT-310 (oral cannabidiol [CBD]) is in development to
expand the therapeutic application of CBD across a range of
neurological disorders.
- Announced nomination of a new therapeutic candidate, LYT-310,
which is expected to enter the clinic in Q4 of 2023. As with
LYT-300, PureTech scientists applied the Glyph™
Platform to enable advantages such as oral capsule dosing and
the potential for improved safety and tolerability. These
differentiated features of LYT-310 could expand CBD use across a
broad range of patient populations (such as adolescents and adults)
and indications, including rare and common forms of epilepsy as
well as other central nervous system disorders. LYT-310 has the
potential to be a readily scalable and consistent product that can
be produced in a cost-effective manner.
- LYT-503/IMB-150 (partnered non-opioid pain program) is
being advanced through a collaboration for the potential treatment
of interstitial cystitis/bladder pain syndrome, a chronic bladder
condition that consists of discomfort or pain in the bladder or
surrounding pelvic region that is not adequately controlled for
many patients.
- First patient, first visit was achieved in the Phase 1 clinical
trial evaluating LYT-503/IMB -150 as a potential non-opioid
treatment for female patients with Interstitial Cystitis/Bladder
Pain Syndrome.
- LYT-510 (oral inflammation-targeting formulation of
tacrolimus) is in development for the potential treatment of
chronic pouchitis and inflammatory bowel disease (IBD), a condition
for which current treatments require multiple injections over time
and are associated with several limitations, including
dose-limiting adverse events, loss of efficacy over time, a lack of
efficacy entirely and the potential for opportunistic infections or
malignancies.
- LYT-510 is the lead candidate generated from the
Alivio™ Platform, with two others, LYT-503/IMB-150
and LYT-500, also developed using PureTech’s proprietary
inflammation-targeting technology. PureTech will prioritize the
development of LYT-510 ahead of LYT-500.
- PureTech’s technology platforms, which include the
Glyph, Alivio and Orasome™ platforms, contribute to
the Company’s advancement by generating new wholly-owned candidates
(Glyph: LYT-300 and LYT-310) and enabling strategic
partnerships (Alivio: LYT-503/IMB-150). As part of its
pipeline prioritization, the Company may opportunistically enter
into additional strategic partnerships or spinout programs into new
entities around one or several of its platforms or related
candidates, especially as resources are increasingly dedicated to
late-stage clinical development.
Key Founded Entity Updates:
- Karuna (Nasdaq: KRTX)
- Announced positive topline Phase 3 data evaluating the
efficacy, safety and tolerability of KarXT in adults with
schizophrenia, meeting its primary endpoint and key secondary
endpoints. Karuna plans to submit a New Drug Application (NDA) for
KarXT in schizophrenia with the U.S. FDA in mid-2023, as of the
most recent update by the company.
- Announced the initiation of its Phase 3 ADEPT program, which is
evaluating KarXT for the treatment of psychosis related to
Alzheimer’s Disease (AD.)
- Gelesis (NYSE: GLS)
- Generated $36.8M in revenue and obtained 185,000 customers
since the launch of Plenity.3
- Announced that based on the extensive safety data and
differentiation due to affordability, broad label and safety
profile, it is preparing an application to the FDA to broaden the
classification of Plenity in the U.S. from prescription-only to
over the counter. Gelesis plans to submit the application to the
FDA during the first quarter of 2023 and could receive market
clearance by the third quarter of 2023.
- Vor (Nasdaq: VOR)
- Announced initial clinical data from VBP101, Vor’s Phase 1/2a
multicenter, open-label, first-in-human study of tremtelectogene
empogeditemcel or "trem-cel" (formerly VOR33) in patients with AML.
The data observed from the first treated patient support the
potential of a trem-cel transplant to be successfully manufactured,
to engraft normally and to maintain blood counts following
treatment with the CD33-targeted therapy Mylotarg. The clinical
trial continues to enroll patients and additional data are expected
in 2023.
- Akili (Nasdaq: AKLI)
- Deployed first wave of its EndeavorRx4 go-to-market sales force
in 14 priority territories across the U.S. with a focus on
Integrated Behavioral Health Centers and pediatric providers.
- Announced that Highmark, the fourth largest Blue Cross Blue
Shield organization, included EndeavorRx in its medical policy for
the reimbursement of prescription digital therapeutics.
- Announced that its partner, Shionogi, started a pivotal Phase 3
randomized, controlled study of SDT-001 in children with
attention-deficit hyperactivity disorder (ADHD).
- Vedanta
- Opened a new facility designed to manufacture clinical and
commercial supply for its therapeutic portfolio, including for the
planned Phase 3 study (expected to begin in the first half of 2023)
and potential commercial launch of lead candidate, VE303, in
Clostridioides difficile infection. Vedanta believes it was the
first company to manufacture CGMP-grade defined bacterial consortia
in powdered form, which enables stable, consistent oral
formulations.
Board Composition & Committee
Changes:
- As noted in PureTech’s 2022 Half-Year Report, Dame Marjorie
Scardino, Senior Independent Director, chair of the Nomination
Committee and member of the Audit Committee, will retire as of the
close of business on December 31, 2022. Raju Kucherlapati, Ph.D.,
will assume the role of PureTech’s Senior Independent Director as
well as the chair of its Nomination Committee, effective as of Dame
Scardino’s retirement.
- Christopher Viehbacher, Chair of PureTech’s Board, was recently
appointed President, Chief Executive Officer and a member of the
Board of Biogen Inc. (Nasdaq: BIIB). Given the time commitment
required by this new role, Mr. Viehbacher will not stand for
re-election at the Company’s 2023 Annual General Meeting. The
Nomination Committee will initiate a process to identify a new
Chair.
Mr. Viehbacher noted: “It has been truly rewarding to work with
such a talented group of people who share a genuine passion for
helping patients. Together, we have developed a pharmaceutical
pioneer delivering innovative medicines with a proven track record
that is truly differentiated across the industry. The foundation
for these successes is supported by a strong cash position and
unique business model to support continued growth. I’m proud of
these collective accomplishments and I look forward to continuing
to interact with my colleagues at PureTech in this next exciting
phase for the company.”
Ms. Zohar commented: “For nearly a decade, Chris has brought a
balance of enthusiasm and skepticism to the PureTech Board that is
emblematic of our approach. His commercial expertise, deal-making
ethos and patient-centered focus have helped to shape our mission
and laid the foundation for our future growth. I am grateful that
Chris will remain a trusted advisor, and we wish him much success
as he takes the helm at Biogen.”
About PureTech Health
PureTech is a biotherapeutics company dedicated to changing the
treatment paradigm for devastating diseases. The Company has
created a broad and deep pipeline through the expertise of its
experienced research and development team and its extensive network
of scientists, clinicians and industry leaders. This pipeline,
which is being advanced both internally and through PureTech's
Founded Entities, is comprised of 26 therapeutics and therapeutic
candidates, including two (Plenity® and EndeavorRx®) that have
received both U.S. FDA clearance and European marketing
authorization and a third (KarXT) that will soon be filed for FDA
approval, as of the most recent update by the Company. All of the
underlying programs and platforms that resulted in this pipeline of
therapeutic candidates were initially identified or discovered and
then advanced by the PureTech team through key validation points
based on unique insights in immunology and drug development.
For more information, visit www.puretechhealth.com or connect
with us on Twitter @puretechh.
Cautionary Note Regarding Forward-Looking Statements
This press release contains statements that are or may be
forward-looking statements within the meaning of the Private
Securities Litigation Reform Act of 1995. All statements contained
in this press release that do not relate to matters of historical
fact should be considered forward-looking statements, including
without limitation those related to the Company’s LYT-100
development program and the timing for results from ongoing
clinical trials of LYT-100, the LYT-200 development program and the
timing for results from ongoing clinical trials of LYT-200, the
planned initiation of clinical trials for LYT-300 and LYT-310, the
potential therapeutic benefits of the product candidates within
Company’s Wholly Owned Programs, the Company’s plan related to the
prioritization of programs and activities associated with its
pipeline, the Company’s approach to potential partnerships or
spinouts of its platforms or candidates and our future prospects,
developments and strategies. The forward-looking statements are
based on current expectations and are subject to known and unknown
risks, uncertainties and other important factors that could cause
actual results, performance and achievements to differ materially
from current expectations, including, but not limited to, those
risks, uncertainties and other important factors described under
the caption "Risk Factors" in our Annual Report on Form 20-F for
the year ended December 31, 2021 filed with the SEC and in our
other regulatory filings. These forward-looking statements are
based on assumptions regarding the present and future business
strategies of the Company and the environment in which it will
operate in the future. Each forward-looking statement speaks only
as at the date of this press release. Except as required by law and
regulatory requirements, we disclaim any obligation to update or
revise these forward-looking statements, whether as a result of new
information, future events or otherwise.
1 References to “Wholly Owned Programs” refer to the Company’s
six therapeutic candidates (LYT-100, LYT-200, LYT-300, LYT-310,
LYT-510 and LYT-503/IMB-150), lymphatic and inflammation technology
platforms and potential future therapeutic candidates and platforms
that the Company may develop or obtain. References to “Wholly Owned
Pipeline” refer to LYT-100, LYT-200, LYT-300, LYT-310, LYT-510 and
LYT-503/IMB-150. On July 23, 2021, Imbrium Therapeutics exercised
its option to license LYT-503/IMB-150 pursuant to which it is
responsible for all future development activities and funding for
LYT-503/IMB-150. 2 Founded Entities represent companies founded by
PureTech in which PureTech maintains ownership of an equity
interest and, in certain cases, is eligible to receive sublicense
income and royalties on product sales. As of the date of this
release, PureTech maintained control over Follica Incorporated,
Vedanta Biosciences, Inc., and Entrega, Inc. by virtue of (a)
majority voting control or (b) the right to elect representation to
the entity’s Board of Directors. As of the date of this release,
PureTech did not have a controlling interest in Gelesis Holdings,
Inc., Karuna Therapeutics, Inc., Akili, Inc., Sonde Health, Inc.
and Vor Biopharma Inc. 3 Important Safety Information about
Plenity: Patients who are pregnant or are allergic to cellulose,
citric acid, sodium stearyl fumarate, gelatin, or titanium dioxide
should not take Plenity. To avoid impact on the absorption of
medications: For all medications that should be taken with food,
take them after starting a meal. For all medications that should be
taken without food (on an empty stomach), continue taking on an
empty stomach or as recommended by your physician. The overall
incidence of side effects with Plenity was no different than
placebo. The most common side effects were diarrhea, distended
abdomen, infrequent bowel movements, and flatulence. Contact a
doctor right away if problems occur. If you have a severe allergic
reaction, severe stomach pain, or severe diarrhea, stop using
Plenity until you can speak to your doctor. Rx Only. For the safe
and proper use of Plenity or more information, talk to a healthcare
professional, read the Patient Instructions for Use, or call
1-844-PLENITY. 4 EndeavorRx is the first-and-only FDA-authorized
treatment delivered through a video game experience. EndeavorRx is
indicated to improve attention function as measured by
computer-based testing in children ages 8 to 12 years old with
primarily inattentive or combined-type ADHD, who have a
demonstrated attention issue. Patients who engage with EndeavorRx
demonstrate improvements in a digitally assessed measure Test of
Variables of Attention (TOVA®) of sustained and selective attention
and may not display benefits in typical behavioral symptoms, such
as hyperactivity. EndeavorRx should be considered for use as part
of a therapeutic program that may include clinician-directed
therapy, medication, and/or educational programs, which further
address symptoms of the disorder. EndeavorRx is available by
prescription only. It is not intended to be used as a stand-alone
therapeutic and is not a substitution for a child’s medication. The
most common side effect observed in children in EndeavorRx’s
clinical trials was a feeling of frustration, as the game can be
quite challenging at times. No serious adverse events were
associated with its use. EndeavorRx is recommended to be used for
approximately 25 minutes a day, 5 days a week, over initially at
least 4 consecutive weeks, or as recommended by your child’s health
care provider. To learn more about EndeavorRx, please visit
EndeavorRx.com. 5 Dempsey, T. M., Payne, S., Sangaralingham, L.,
Yao, X., Shah, N. D., & Limper, A. H. (2021). Adoption of the
Antifibrotic Medications Pirfenidone and Nintedanib for Patients
with Idiopathic Pulmonary Fibrosis. Annals of the American Thoracic
Society, 18(7), 1121–1128.
https://doi.org/10.1513/AnnalsATS.202007-901OC 6 Margaritopoulos,
G. A., Trachalaki, A., Wells, A. U., Vasarmidi, E., Bibaki, E.,
Papastratigakis, G., Detorakis, S., Tzanakis, N., & Antoniou,
K. M. (2018). Pirfenidone improves survival in IPF: results from a
real-life study. BMC pulmonary medicine, 18(1), 177.
https://doi.org/10.1186/s12890-018-0736-z 7 The use of the Animal
Rule is intended for drugs and biological products developed to
reduce or prevent serious or life-threatening conditions caused by
exposure to lethal or permanently disabling toxic chemical,
biological, radiological or nuclear substances 8 Walter, R. B.,
Othus, M., Burnett, A. K., L�wenberg, B., Kantarjian, H. M.,
Ossenkoppele, G. J., Hills, R. K., Ravandi, F., Pabst, T., Evans,
A., Pierce, S. R., Vekemans, M. C., Appelbaum, F. R., & Estey,
E. H. (2015). Resistance prediction in AML: analysis of 4601
patients from MRC/NCRI, HOVON/SAKK, SWOG and MD Anderson Cancer
Center. Leukemia, 29(2), 312–320.
https://doi.org/10.1038/leu.2014.242 9 Brexanolone NDA 211371
Multi-disciplinary Review and Evaluation, FDA CDER, 2018.
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PureTech Public Relations
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Nichole Sarkis +1 774 278 8273
nichole@tenbridgecommunications.com
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