BridgeBio Pharma Affiliate Venthera Presents Preliminary Results from Clinical Trial of VT30 (BBP-681) for Venous, Lymphatic, and Venolymphatic Malformations
PALO ALTO, CA – June 10, 2022 — BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio) and its affiliate company Venthera, Inc. (Venthera), today announced preliminary data from the Phase 1b trial of VT30 topical gel (BBP-681) in patients with venous, lymphatic and mixed venolymphatic lesions of the skin (VM, LM and VLM, respectively), which are rare genetic vascular anomalies driven by dysregulated activation of intracellular PI3K. The data are being shared in a virtual presentation to the International Society for the Study of Vascular Anomalies (ISSVA).
“People living with cutaneous VMs and LMs can experience clinical complications including pain, bleeding, and impaired functioning of the affected area. To date, their treatment options are limited to interventional procedures and surgeries. We are pleased that the trial has achieved its goal of characterizing the tolerability of a range of dosage strengths in affected patients and we believe it has provided us with the data we need to make decisions on the future direction of the program,” said Thomas Rossi, Ph.D., Chief Executive Officer of Venthera.
The data presented are preliminary results from an open-label, dose-finding study of VT30 topical gel in patients with cutaneous VM/LM/VLM lesions associated with mutations that result in dysregulated PI3K activation. VT30 topical gel is designed to potently inhibit PI3K in the treated tissue without systemic side effects. Early findings include:
- Across 15 subjects treated in escalating-dose cohorts, VT30 topical gel has been generally well-tolerated; local self-limited rash was the only adverse experience of note, occurring at an increased incidence with the highest gel concentration. All reported adverse experiences were characterized as mild to moderate.
- Pharmacokinetic and pharmacodynamic skin punch biopsies were taken from within the lesion. These data demonstrate that high concentrations of drug (in the micromolar range) are present in the lesion at day 28 and show a reduction of pS6 in the lesion from baseline to day 28.
- No circulating drug was detected in plasma with any of the gel strengths studied.
Based on the findings shared during ISSVA 2022, Venthera has tentatively selected the 0.6% gel for further study. Venthera is currently seeking partners to support development of VT30 topical gel in Phase 2. To learn more, please contact [email protected].
For more information about study specifics, see ClinicalTrials.gov (NCT04409145).
About Venthera
Venthera is dedicated to
improving the lives of patients living with rare vascular
anomalies by suppressing the underlying pathways that drive
vascular anomaly growth. By targeting the molecular root cause
of disease(s), Venthera is seeking to address unmet needs and
create a new option for treating VMs, LMs, and VLMs with the
hope of reducing the need for invasive and systemic treatments.
About BridgeBio Pharma, Inc.
BridgeBio Pharma, Inc. (BridgeBio) is a commercial-stage biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBio’s pipeline of development programs ranges from early science to advanced clinical trials. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebiodev.wpengine.com and follow us on LinkedIn and Twitter.
BridgeBio Pharma Forward-Looking Statements
This press release contains forward-looking statements.
Statements we make in this press release may include statements
that are not historical facts and are considered forward-looking
within the meaning of Section 27A of the Securities Act of 1933,
as amended (the “Securities Act”), and Section 21E of the
Securities Exchange Act of 1934, as amended (the “Exchange
Act”), which are usually identified by the use of words such as
“anticipates,” “believes,” “estimates,” “expects,” “intends,”
“may,” “plans,” “projects,” “seeks,” “should,” “will,” and
variations of such words or similar expressions. We intend
these forward-looking statements to be covered by the safe
harbor provisions for forward-looking statements contained in
Section 27A of the Securities Act and Section 21E of the
Exchange Act and are making this statement for purposes of
complying with those safe harbor provisions. These
forward-looking statements, including statements relating to the
timing and success of Venthera’s Phase 1/2 clinical trial of
VT30/BBP-681 for the treatment of venous malformations,
lymphatic malformations, and venolymphatic malformations
associated with PIK3CA or TEK mutations, expectations, plans and
prospects regarding Venthera’s regulatory approval process for
VT30/BBP681, the ability of VT30/BBP-681 to treat venous
malformations, lymphatic malformations, and venolymphatic
malformations associated with PIK3CA or TEK mutations in humans,
and the timing and success of initial top-line Phase 1/2 data of
VT30/BBP-681, reflect our current views about our plans,
intentions, expectations, strategies and prospects, which are
based on the information currently available to us and on
assumptions we have made. Although we believe that our plans,
intentions, expectations, strategies and prospects as reflected
in or suggested by those forward-looking statements are
reasonable, we can give no assurance that the plans, intentions,
expectations or strategies will be attained or achieved.
Furthermore, actual results may differ materially from those
described in the forward-looking statements and will be affected
by a number of risks, uncertainties and assumptions, including,
but not limited to, Venthera’s ability to continue and complete
its Phase 1/2 clinical trial of VT30/BBP-681 for the treatment
of venous malformations, lymphatic malformations, and
venolymphatic malformations associated with PIK3CA or TEK
mutations, past data from preclinical studies not being
indicative of future data from clinical trials, Venthera’s
ability to advance VT30/BBP-681 in clinical development
according to its plans, the ability of VT30/BBP-681 to treat
venous malformations, lymphatic malformations, and venolymphatic
malformations associated with PIK3CA or TEK mutations, the
extent to which Venthera identifies collaborators to support
development of VT30 topical gel in Phase 2, as well as those
risks set forth in the Risk Factors section of BridgeBio’s most
recent Annual Report on Form 10-K and BridgeBio’s other SEC
filings. Moreover, BridgeBio and Venthera operate in a very
competitive and rapidly changing environment in which new risks
emerge from time to time. Except as required by applicable law,
we assume no obligation to update publicly any forward-looking
statements, whether as a result of new information, future
events or otherwise.
BridgeBio Contact:
Grace Rauh
(917) 232-5478