VE303 receives Fast Track designation for
prevention of recurrent C. difficile infection ahead of global
pivotal Phase 3 study
Exploratory analyses from the VE303 Phase 2
CONSORTIUM study identify statistically significant relationships
between VE303 bacterial species and multiple parameters that
provide further mechanistic support for VE303’s observed
efficacy
PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) (“PureTech” or the
“Company”), a clinical-stage biotherapeutics company, noted today
that its Founded Entity, Vedanta Biosciences, a clinical-stage
company that is developing a potential new category of oral
therapies based on defined bacterial consortia, announced that the
U.S. Food and Drug Administration (FDA) granted Fast Track
designation to Vedanta’s defined bacterial consortium candidate,
VE303, for the prevention of recurrent Clostridioides difficile
infection (rCDI). Fast Track designation is a process designed to
facilitate the development and expedite the review of drugs that
treat serious conditions and fill an unmet medical need.
Vedanta also announced the details of a podium presentation of
research informed by the VE303 Phase 2 CONSORTIUM study at
Digestive Disease Week (DDW) 2023, held in Chicago, Illinois from
May 6-9. The analyses review multiple aspects of the
pharmacodynamic response to VE303 in patients at high risk for
rCDI, including how safety and efficacy correlate with dosing
regimen, consortium strain colonization, metabolic changes, and
restoration of a patient’s gut microbial community. The strong
correlation between the presence of VE303 strains and both
beneficial metabolic changes and nonrecurrence of CDI provides
mechanistic support for the study’s observed efficacy results.
The full text of the announcement from Vedanta is as
follows:
Vedanta Biosciences Receives Fast Track
Designation for VE303 and Presents Phase 2 Data at Digestive
Disease Week
VE303 receives Fast Track designation for
prevention of recurrent C. difficile infection ahead of global
pivotal Phase 3 study
Exploratory analyses from the VE303 Phase 2
CONSORTIUM study identify statistically significant relationships
between VE303 bacterial species and multiple parameters that
provide further mechanistic support for VE303’s observed
efficacy
CAMBRIDGE, MA, May 8, 2023 – Vedanta Biosciences, a
clinical-stage company that is developing a potential new category
of oral therapies based on defined bacterial consortia, today
announced that the U.S. Food and Drug Administration (FDA) granted
Fast Track designation to Vedanta’s defined bacterial consortium
candidate, VE303, for the prevention of recurrent Clostridioides
difficile infection (rCDI). Fast Track designation is a process
designed to facilitate the development and expedite the review of
drugs that treat serious conditions and fill an unmet medical need.
Vedanta also announced the details of a podium presentation of
research informed by the VE303 Phase 2 CONSORTIUM study at
Digestive Disease Week (DDW) 2023, held in Chicago, Illinois from
May 6-9.
The presented analyses review multiple aspects of the
pharmacodynamic response to VE303 in patients at high risk for
rCDI, including how safety and efficacy correlate with dosing
regimen, consortium strain colonization, metabolic changes, and
restoration of a patient’s gut microbial community. The strong
correlation between the presence of VE303 strains and both
beneficial metabolic changes and nonrecurrence of CDI provides
mechanistic support for the study’s observed efficacy results.
A disrupted intestinal microbiome from recent antibiotic
exposure can lead to overgrowth of C. difficile. In the United
States, approximately 500,000 people develop CDI each year, with
symptoms that can include diarrhea, fever, abdominal pain, and
cramping. Antibiotics are used to treat CDI and are generally
successful, but they can further perturb the microbiome. As a
result, an estimated 20% or more of these patients go on to have
recurrent CDI episodes, which can leave them increasingly
debilitated. Building on the positive Phase 2 data that were
recently published in JAMA, the analyses presented at DDW provide
additional evidence that VE303 strains colonize robustly and work
to more quickly restore a disrupted microbiome to a normal, healthy
state.
“The FDA’s decision to grant Fast Track designation to VE303
underscores the continuing need for medical innovation for this
condition. Over 150,000 people experience recurrent CDI annually in
the U.S. alone, and this requires scalable, effective treatment
solutions,” said Jeffrey L. Silber, M.D., Chief Medical Officer of
Vedanta Biosciences. “Fecal microbiota transplants and other
donor-derived treatments are often prescribed—they can be
effective, but they can vary greatly in composition and potency,
and they may carry a risk of transmitting infectious agents.
Vedanta’s approach of growing the strains for our defined bacterial
consortia from pure clonal cell banks is a highly scalable process
that obviates the need for fecal donors and leads to a consistently
manufactured candidate. We look forward to initiating our global
Phase 3 pivotal study for VE303, RestoratiVE303, later in
2023.”
About the VE303 DDW Presentation Title:
Pharmacodynamic response to a defined bacterial consortium, VE303,
in patients with Clostridioides difficile infection (CDI): Results
of the Phase 2 CONSORTIUM study
Presenter: Rajita Menon, Ph.D.
Overview: The research reviews various aspects of the
Phase 2 CONSORTIUM study of VE303. The study was a randomized,
double-blind, placebo-controlled, dose-finding trial in individuals
at high risk of rCDI. After completing a course of antibiotics for
a laboratory-confirmed qualifying CDI episode, subjects were
randomized in a 1:1:1 ratio to receive low-dose VE303, high-dose
VE303, or placebo orally once daily for 14 days. In the Phase 2
study, the VE303 high dose arm had an acceptable safety profile and
compared with placebo, reduced the odds of developing rCDI through
Week 8 by over 80%. The VE303 high dose also led to superior VE303
strain colonization at 14 days, achieving long-term engraftment,
and promoted early restoration of the microbiota and beneficial
metabolites.
To aid in the understanding of the mechanisms underlying the
observed clinical efficacy, fecal samples were collected and
analyzed at multiple timepoints. Among the 378 microbial species
identified in these samples, the only ones significantly associated
with non-recurrence were three of the strains in VE303. Several of
the VE303 strains were associated with beneficial metabolic changes
within the first 14 days. These observations suggest that VE303 may
protect against rCDI via multiple mechanisms—both directly, through
growth and preferential establishment of the VE303 strains in the
gut relative to more pathogenic species; and indirectly, through
synthesis of beneficial metabolites and enhanced recovery of the
broader gut microbiome.
This research presentation was developed by Vedanta in
collaboration with researchers at the University of Massachusetts
Medical School and Mt. Sinai School of Medicine.
About VE303 VE303 is a defined bacterial consortium
therapeutic candidate designed for the prevention of recurrent
Clostridioides difficile infection (rCDI). It consists of eight
strains that were rationally selected using Vedanta’s discovery
engine. VE303 is produced from pure, clonal bacterial cell banks,
which yield a standardized drug product in powdered form and bypass
the need to rely on direct sourcing of donor fecal material of
inconsistent composition. Vedanta reported positive topline results
in October 2021 from the Phase 2 CONSORTIUM trial, in which VE303
was associated with a 31.7% absolute risk reduction in the rate of
recurrence when compared with placebo, representing a greater than
80% reduction in the odds of a CDI recurrence. Vedanta believes
VE303 has the potential to become a first-in-class therapeutic
based on a defined bacterial consortium. Vedanta Biosciences
received a $5.4 million research grant from the Combating
Antibiotic Resistant Bacteria Biopharmaceutical Accelerator
(CARB-X) in 2017. Additionally, the positive results of the Phase 2
study, first reported in October 2021, triggered a $23.8 million
contract option from the Biomedical Advanced Research and
Development Authority (BARDA) to support a Phase 3 clinical study
of VE303. This project has been funded in part with federal funds
from the Department of Health and Human Services; Administration
for Strategic Preparedness and Response; BARDA, under contract
number 75A5012C00177 for a contract value up to $81.9 million.
VE303 was granted Orphan Drug Designation in 2017 and Fast Track
Designation in 2023 by the U.S. Food and Drug Administration (FDA)
for the prevention of recurrent CDI.
About Vedanta Biosciences Vedanta Biosciences is leading
the development of a potential new category of oral therapies based
on defined consortia of bacteria isolated from the human microbiome
and grown from pure clonal cell banks. The company’s clinical-stage
pipeline includes product candidates being evaluated for the
prevention of recurrent C. difficile infection, inflammatory bowel
diseases, food allergy, and liver disease. These investigational
therapies are grounded in our team’s pioneering research –
published in leading journals including Science, Nature, Cell, and
JAMA – to identify beneficial bacteria that live symbiotically
within the healthy human gut, fight pathogens, and induce a range
of potent immune responses. Vedanta Biosciences controls a
foundational portfolio of more than 70 patents and has built what
it believes is the industry-leading platform for development of
defined bacterial consortia drugs. This platform includes one of
the largest libraries of bacteria derived from the human
microbiome, vast datasets from human interventional studies,
proprietary capabilities in consortium design, and end-to-end
capabilities for CGMP-compliant manufacturing of oral drug
candidates spanning cell banking, fermentation, lyophilization, and
fill finish.
About Digestive Disease Week® Digestive Disease Week®
(DDW) is the largest international gathering of physicians,
researchers and academics in the fields of gastroenterology,
hepatology, endoscopy and gastrointestinal surgery. Jointly
sponsored by the American Association for the Study of Liver
Diseases (AASLD), the American Gastroenterological Association
(AGA) Institute, the American Society for Gastrointestinal
Endoscopy (ASGE) and the Society for Surgery of the Alimentary
Tract (SSAT), DDW is an in-person and virtual meeting from May 6-9,
2023. The meeting showcases more than 3,100 abstracts and hundreds
of lectures on the latest advances in GI research, medicine and
technology. More information can be found at www.ddw.org.
About PureTech Health PureTech is a clinical-stage
biotherapeutics company dedicated to giving life to new classes of
medicine to change the lives of patients with devastating diseases.
The Company has created a broad and deep pipeline through its
experienced research and development team and its extensive network
of scientists, clinicians and industry leaders that is being
advanced both internally and through its Founded Entities.
PureTech's R&D engine has resulted in the development of 27
therapeutics and therapeutic candidates, including two (Plenity®
and EndeavorRx®) that have received both US FDA clearance and
European marketing authorization and a third (KarXT) that is
expected to be filed soon for FDA approval. A number of these
programs are being advanced by PureTech or its Founded Entities in
various indications and stages of clinical development, including
registration enabling studies. 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.
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 statements that relate to the VE303’s Fast
Track Designation by the FDA, Vedanta’s presentation of research
informed by the VE303 Phase 2 CONSORTIUM study at Digestive Disease
Week (DDW) 2023, and Vedanta’s and PureTech's future prospects,
development plans, 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, the following: our history of incurring significant operating
losses since our inception; our need for additional funding to
achieve our business goals, which may not be available and which
may force us to delay, limit or terminate certain of our
therapeutic development efforts; our limited information about and
limited control or influence over our Non-Controlled Founded
Entities; the lengthy and expensive process of preclinical and
clinical drug development, which has an uncertain outcome and
potential for substantial delays; potential difficulties with
enrolling patients in clinical trials, which could delay our
clinical development activities; side effects, adverse events or
other safety risks which could be associated with our therapeutic
candidates and delay or halt their clinical development; our
ability to obtain regulatory approval for and commercialize our
therapeutic candidates; our ability to realize the benefits of our
collaborations, licenses and other arrangements; our ability to
maintain and protect our intellectual property rights; our reliance
on third parties, including clinical research organizations,
clinical investigators and manufacturers; our vulnerability to
natural disasters, global economic factors, geo-political actions
and unexpected events; and those additional important factors
described under the caption "Risk Factors" in our Annual Report on
Form 20-F for the year ended December 31, 2022 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.
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