PhaseBio Announces European Regulatory Update for PB2452
PB2452 granted PRIME Designation by the
PhaseBio receives written scientific advice confirming PB2452 clinical development plan
In Phase 1 and Phase 2a clinical trials, PB2452 provided immediate and sustained reversal of the antiplatelet effects of ticagrelor
PB2452 Granted PRIME Designation
PRIME designation is granted by the EMA to enhance support for the development of medicines that demonstrate the potential to address substantial unmet medical need based on early clinical data. The EMA prioritizes PRIME designated drugs for special support, including enhanced interactions and dialogue with EMA during development, as well as a pathway for accelerated evaluation and review for marketing authorization. The program is intended to optimize development plans and potentially expedite the review and approval process so that these medicines may reach patients as early as possible.
In its review comments for PRIME designation, EMA recognized the unmet need for an effective reversal agent for ticagrelor-induced platelet inhibition. EMA acknowledged that, while infrequent, ticagrelor-related major bleeding events are associated with relevant morbidity and mortality. Urgent surgical interventions (such as coronary artery bypass grafting) that cannot be delayed to allow full washout of ticagrelor are associated with an increased risk for intra- and post-procedural bleeding events. EMA also acknowledged that there are no established effective treatment options to reverse ticagrelor-induced platelet inhibition.
EMA further agreed that the preliminary clinical data provided in the application suggested that PB2452 is likely to be able to address, to a significant extent, the targeted unmet medical need. As the basis of the application, EMA reviewed PB2452 clinical trial results from the recently completed Phase 2a study, which demonstrated immediate and sustained reversal of the antiplatelet effects of ticagrelor in older and elderly patients treated with dual antiplatelet therapy (low-dose aspirin and ticagrelor) and in healthy subjects treated with supratherapeutic doses of ticagrelor. The clinical data corroborated non-clinical findings that were also reviewed, which demonstrated selective, high-affinity binding of PB2452 to ticagrelor and its active metabolite and its potential to provide rapid reversal of ticagrelor. Additionally, EMA cited literature evidence indicating that the platelet-function assays utilized in the PB2452 development program are likely to be predictive of clinical outcomes in cardiovascular patients.
Scientific Advice Regarding the PB2452 Development Program
Separately, PhaseBio met with the
With respect to the clinical development program, CHMP generally agreed with PhaseBio’s plan to conduct a single-arm, open-label, pivotal clinical study of PB2452 in the target population of patients on ticagrelor who experience a major bleeding event or are in need of an urgent surgical procedure. Regarding the overall number of patients targeted to participate in the Phase 3 trial, CHMP agreed with PhaseBio’s proposal to conduct the trial in a total of 200 patients, with results from the first 100 patients expected to be included in the MAA package seeking conditional marketing authorization (CMA). CHMP also agreed with PhaseBio’s proposed use of the VerifyNow PRUTest® biomarker as the primary endpoint for the Phase 3 trial. PhaseBio has used VerifyNow PRUTest in its Phase 1 and Phase 2a clinical trials, where it demonstrated a high degree of correlation with other biomarkers used to measure platelet function.
PB2452 is a novel, recombinant, human monoclonal antibody antigen-binding fragment designed to reverse the antiplatelet activity of ticagrelor in major bleeding and urgent surgery situations. In a Phase 1 clinical trial, PB2452 demonstrated the potential to bring life-saving therapeutic benefit through immediate and sustained reversal of ticagrelor’s antiplatelet activity, mitigating concerns regarding bleeding risks associated with the use of antiplatelet drugs. The Phase 1 clinical trial of PB2452 in healthy volunteers was published in the
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Forward-looking statements include statements concerning or implying the conduct or timing of our clinical trials and our research, development and regulatory plans for PB2452, the potential for PB2452 to receive regulatory approval from the
Risks regarding our business are described in detail in our Securities and Exchange Commission filings, including in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2019. These forward-looking statements speak only as of the date hereof, and PhaseBio Pharmaceuticals, Inc. disclaims any obligation to update these statements except as may be required by law.
Chief Financial Officer
1 Bhatt DL, Pollack CV, Weitz JI, et al. Antibody-Based Ticagrelor Reversal Agent in
Source: PhaseBio Pharmaceuticals, Inc.