TIDMPRTC
RNS Number : 5452K
PureTech Health PLC
22 December 2022
22 December 2022
PureTech Health plc
PureTech Provides End of Year Report on Key Progress
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(R) and Akili's EndeavorRx(R) 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 Programs
[1] and Founded Entities [2] 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(R)
for weight management [3] and EndeavorRx(R) for treating
inattention in ADHD ages 8-12 [4] ) 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]
o 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.
o 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.
o 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]
o 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.
o 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]
o 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.
o 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)
64(th) 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.
o 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.
o 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 GABA(A) 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.
o 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 (TM) 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.
o 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.
o LYT-510 is the lead candidate generated from the Alivio(TM)
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- (TM) 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)
o 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.
o 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)
o Generated $36.8M in revenue and obtained 185,000 customers
since the launch of Plenity.(3)
o 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)
o 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)
o Deployed first wave of its EndeavorRx(4) go-to-market sales
force in 14 priority territories across the U.S. with a focus on
Integrated Behavioral Health Centers and pediatric providers.
o Announced that Highmark, the fourth largest Blue Cross Blue
Shield organization, included EndeavorRx in its medical policy for
the reimbursement of prescription digital therapeutics.
o 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
o 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(R) and EndeavorRx(R)) 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.
Contact:
PureTech
Public Relations
publicrelations@puretechhealth.com
Investor Relations
IR@puretechhealth.com
EU Media
Ben Atwell, Rob Winder
+44 (0) 20 3727 1000
ben.atwell@FTIconsulting.com
U.S. Media
Nichole Sarkis
+1 774 278 8273
nichole@tenbridgecommunications.com
# # #
[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(R))
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., Löwenberg, 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.
This information is provided by RNS, the news service of the
London Stock Exchange. RNS is approved by the Financial Conduct
Authority to act as a Primary Information Provider in the United
Kingdom. Terms and conditions relating to the use and distribution
of this information may apply. For further information, please
contact rns@lseg.com or visit www.rns.com.
RNS may use your IP address to confirm compliance with the terms
and conditions, to analyse how you engage with the information
contained in this communication, and to share such analysis on an
anonymised basis with others as part of our commercial services.
For further information about how RNS and the London Stock Exchange
use the personal data you provide us, please see our Privacy
Policy.
END
BOADDBDDRUDDGDB
(END) Dow Jones Newswires
December 22, 2022 02:00 ET (07:00 GMT)
Puretech Health (LSE:PRTC)
Historical Stock Chart
From Jun 2024 to Jul 2024
Puretech Health (LSE:PRTC)
Historical Stock Chart
From Jul 2023 to Jul 2024