PALO ALTO, Calif., June 10, 2022 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. ( 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).
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 bridgebio.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.