Press Release
PhaseBio Reports Fourth-Quarter and Full-Year 2020 Financial Results and Recent Business Highlights
Update on Phase 3 REVERSE-IT trial - enrollment ahead of schedule
Announced commercial supply agreement with
Expanded pivotal Phase 3 REVERSE-IT trial of bentracimab into
“Throughout 2020, PhaseBio made significant progress advancing our pipeline of therapies for serious cardiopulmonary diseases,” said
Mow continued, “Looking ahead into 2021, we are excited to continue advancing this pivotal program through key planned milestones including publishing Phase 2a data, announcing the interim analysis of the first 100 patients from the REVERSE-IT Phase 3 trial, initiating additional trial sites abroad, and working with key collaborators on our bentracimab commercialization plans. We also remain focused on advancing pemziviptadil for pulmonary arterial hypertension and are targeting to report Phase 2b data in the second half of 2021.”
Bentracimab (PB2452) Highlights
-
Accelerated Enrollment in Ongoing REVERSE-IT Phase 3 Clinical Trial: As of
March 2021 , the REVERSE-IT Phase 3 clinical trial had enrolled 60 of the first approximately 100 patients needed to support a Biologics License Application (BLA), nearly all of whom to date have required urgent surgery or an invasive procedure. PhaseBio is attempting to accelerate enrollment of patients with uncontrolled major or life-threatening bleeding, including by working to increase the number of enrolling clinical trial sites inthe United States ,Canada , and theEuropean Union as it is believed that a broader site footprint will increase the probability of enrolling these patients. All of the first approximately 100 patients enrolled in the REVERSE-IT trial will be measured against the same VerifyNow® PRUTest biomarker that is the primary endpoint for all patients enrolled in the REVERSE-IT trial. The trial is enrolling faster than PhaseBio originally projected, and PhaseBio now expects to complete enrollment of the first 100 patients in mid-2021 and is targeting to submit a BLA for bentracimab in mid-2022, although those timelines could be impacted by the continued scope and duration of the COVID-19 pandemic. Bentracimab is a novel, human monoclonal antibody fragment that in earlier clinical trials has shown immediate and sustained reversal of the antiplatelet effects of Brilinta® (ticagrelor).
-
Announced Supply Agreement with
BioVectra for Bentracimab toSupport Development and Commercialization: InMarch 2021 , PhaseBio announced a commercial scale supply agreement withBioVectra , an innovative global contract development and manufacturing organization (CDMO), for the production of bentracimab. Under the terms of the agreement,BioVectra will provide its integrated CDMO services for the manufacturing of the active pharmaceutical ingredient (API) of bentracimab for use in PhaseBio’s ongoing Phase 2b and Phase 3 clinical trials and for global commercial use if bentracimab receives regulatory approval.
-
Expanded REVERSE-IT Trial into the
European Union and Dosed First Patients: InJanuary 2021 , PhaseBio announced that, working with its financing and co-development partnerSFJ Pharmaceuticals , PhaseBio had expanded the REVERSE-IT trial into theEuropean Union , having opened trial sites for enrollment and began dosing its first patients.
-
Expanded REVERSE-IT Trial into
Canada and Dosed First Patients: InOctober 2020 , PhaseBio announced that it had expanded the Phase 3 trial of bentracimab intoCanada , where the first patients outside ofthe United States were enrolled and dosed. Cardiovascular disease remains a leading cause of mortality inthe United States ,Canada and globally. The company also announced that the global Phase 3 trial had been named REVERSE-IT (Rapid and SustainEd ReVERSal of TicagrElor – Intervention Trial).
-
Initiated Phase 3 Clinical Trial for Bentracimab: In
March 2020 , PhaseBio commenced the pivotal Phase 3 clinical trial to evaluate reversal of the antiplatelet effects of ticagrelor in patients with uncontrolled major or life-threatening bleeding or requiring urgent surgery or an invasive procedure.
-
Received PRIME Designation for Bentracimab from
European Medicines Agency : InFebruary 2020 , bentracimab was granted PRIority MEdicines (PRIME) designation by theEuropean Medicines Agency (EMA) for the reversal of the antiplatelet effects of ticagrelor in patients with uncontrolled major or life-threatening bleeding or requiring urgent surgery or an invasive procedure. The EMA prioritizes PRIME designated drugs for special support, including enhanced interactions and dialogue with the EMA during development, as well as a pathway for accelerated evaluation and review for marketing authorization.
-
Received Written Scientific Advice from EMA Confirming the Bentracimab Clinical Development Plan: In
February 2020 , PhaseBio received written guidance from the Committee for Medicinal Products for Human Use (CHMP) of the EMA that generally agreed with PhaseBio’s proposed development plan for bentracimab. After reviewing the Scientific Advice from CHMP, PhaseBio believes that the development plan for bentracimab has been designed to support regulatory filings in theEuropean Union . PhaseBio similarly believes that the development plan for bentracimab has been designed to support regulatory filings inthe United States based on feedback from theU.S. Food and Drug Administration .
-
Entered Into Financing and Co-Development Collaboration with SFJ Pharmaceuticals®: In
January 2020 , PhaseBio announced a financing and co-development collaboration withSFJ Pharmaceuticals (SFJ) to support the development of bentracimab. Under the terms of the agreement, SFJ agreed to fund up to$120 million to support the clinical development of bentracimab and to assume a central role in global clinical development and regulatory activities for bentracimab outside ofthe United States .
From execution of the co-development agreement throughDecember 31, 2020 ,SFJ Pharmaceuticals has funded or reimbursed$47.1 million of clinical trial costs and other expenses of the initial$90 million commitment under the agreement, leaving$42.9 million of funding remaining available to support the Phase 3 program through the end of 2021. PhaseBio is eligible to receive up to an additional$30 million of funding if specific, pre-defined clinical development milestones for bentracimab are met.
Pemziviptadil (PB1046) and Other Pipeline Highlights
-
Presented Data from Phase 1b/2a Trial of Pemziviptadil for the Treatment of Pulmonary Arterial Hypertension at
Pulmonary Vascular Research Institute Virtual World Congress : InJanuary 2021 , PhaseBio announced presentation of data from a Phase 1b/2a pilot study highlighting three patients who received pemziviptadil (PB1046), the company’s first-in-class, sustained-release vasoactive intestinal peptide (VIP) analogue for the treatment of pulmonary arterial hypertension (PAH). The data, which were presented virtually at the 15thPulmonary Vascular Research Institute (PVRI) WorldCongress onJanuary 27, 2021 , continue to highlight the favorable safety and tolerability profile of pemziviptadil, as well as clinically-meaningful, long-term improvement of six-minute walk test (6MWT) distance for one patient after 18 months of treatment. Additionally, the data demonstrate stability in functional status with no clinically-meaningful deterioration for two patients at two and six months after treatment. All three patients completed the study with no drug-related serious adverse events associated with study drug discontinuation and pemziviptadil appeared to be well tolerated.
-
Resumed Enrollment in Ongoing Phase 2b Trial of Pemziviptadil in PAH: In
October 2020 , PhaseBio announced that the ongoing Phase 2b trial of pemziviptadil in patients with PAH, named the VIP trial (Vasoactive Intestinal Peptide in adult patients with pulmonary arterial hypertension), resumed enrollment after a pause related to the impacts of the COVID-19 pandemic and re-prioritization of drug supply to the VANGARD trial. At the time of announcement, approximately one-third of the patients targeted for enrollment had completed the initial 16-week protocol, with approximately 90% of these patients electing to enroll in VIP EXTEND (Vasoactive Intestinal Peptide extension trial in adult patients with pulmonary arterial hypertension), the open label extension of the Phase 2b trial. PhaseBio is targeting to report results from the VIP in the second half of 2021, although that timeline could be impacted by the continued scope and duration of the COVID-19 pandemic.
-
Bolstered Pipeline with Acquisition of Novel Oral Aldosterone Synthase Inhibitor for Treatment-Resistant Hypertension: In
January 2020 , PhaseBio signed an agreement withViamet Pharmaceuticals Holdings, LLC and its wholly-owned subsidiary,Selenity Pharmaceuticals (Bermuda), Ltd. , under which PhaseBio acquired all of the assets and intellectual property rights related to certain novel aldosterone synthase inhibitors, including the company’s lead development compound, now called PB6440, being developed for treatment-resistant hypertension. In preclinical studies completed to date, PB6440 was observed to be a highly potent and selective inhibitor of aldosterone synthase (CYP11B2) versus the closely related steroid 11β-hydroxylase enzyme (CYP11B1). PB6440 demonstrated dose-dependent aldosterone reduction without a significant increase in 11-deoxycorticosterone or deoxycortisol in both rodent and primate models.
Operational Updates
-
Appointed New Member to the Board of Directors: In
February 2020 ,Alex C. Sapir was appointed to the company’s board of directors.
Fourth-Quarter and Full-Year 2020 Financial Results
Cash Position
-
Cash and cash equivalents at
December 31, 2020 were$28.1 million , compared to$74.0 million atDecember 31, 2019 . The decrease reflects cash used in operating activities.
Quarter Ending
-
Net loss for the quarter was
$30.4 million , compared to a net loss of$11.3 million for the prior-year period.
-
Research and development expense increased to
$22.4 million , as compared to$8.4 million for the same period in 2019, driven by an increase in manufacturing, clinical and nonclinical development activities related to bentracimab and pemziviptadil.
-
General and administrative expense decreased to
$3.6 million , compared to$3.7 million for prior-year period.
Year Ending
-
Net loss for the year was
$98.6 million , compared to a net loss of$39.2 million for the prior-year period.
-
Research and development expense increased to
$72.1 million , as compared to$30.9 million for the same period in 2019, driven by an increase in manufacturing, clinical and nonclinical development activities related to bentracimab and pemziviptadil.
-
General and administrative expense increased to
$13.1 million , compared to$11.2 million for prior-year period, primarily due to increases in professional services, personnel, and insurance-related expenses.
About PhaseBio
PhaseBio is located in
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “anticipates,” “believes,” “expects,” “intends,” “projects,” and “future” or similar expressions are intended to identify forward-looking statements.
Forward-looking statements include statements concerning or implying the conduct or timing of our clinical trials and our research, development and regulatory plans for our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed, as well as the success of our partnerships with
Risks regarding our business are described in detail in our
|
||||||||
Condensed Balance Sheets |
||||||||
(in thousands) |
||||||||
|
|
|||||||
Assets: |
||||||||
Cash and cash equivalents |
$ |
28,122 |
|
$ |
74,025 |
|||
Other receivables, prepaid expenses and other current assets |
|
12,027 |
|
|
4,798 |
|||
Property and equipment, net |
|
8,224 |
|
|
1,924 |
|||
Operating lease right-of-use assets |
|
1,927 |
|
|
1,715 |
|||
Other non-current assets |
|
57 |
|
|
32 |
|||
Total assets |
$ |
50,357 |
|
$ |
82,494 |
|||
Liabilities and stockholders' (deficit) equity: |
||||||||
Current portion of long-term debt |
$ |
5,355 |
|
$ |
2,378 |
|||
Accounts payable, accrued expenses and other current liabilities |
|
9,605 |
|
|
6,101 |
|||
Long-term debt, net |
|
6,773 |
|
|
12,326 |
|||
Operating lease liabilities, net |
|
1,548 |
|
|
1,508 |
|||
Development derivative liability |
|
51,719 |
|
|
— |
|||
Other long-term liabilities |
|
559 |
|
|
203 |
|||
Stockholders’ (deficit) equity |
|
(25,202 |
) |
|
59,978 |
|||
Total liabilities and stockholders' (deficit) equity |
$ |
50,357 |
|
$ |
82,494 |
|||
|
|||||||||||||||||
Statements of Operations |
|||||||||||||||||
(in thousands, except share and per share amounts) |
|||||||||||||||||
Quarter Ended |
Year Ended |
||||||||||||||||
|
2020 |
|
|
2019 |
|
|
2020 |
|
|
2019 |
|
||||||
Revenue: |
|||||||||||||||||
Grant revenue |
$ |
— |
|
$ |
689 |
|
$ |
320 |
|
$ |
1,786 |
|
|||||
Revenue under collaborative agreement |
|
— |
|
|
75 |
|
|
— |
|
|
575 |
|
|||||
Total revenue |
|
— |
|
|
764 |
|
|
320 |
|
|
2,361 |
|
|||||
Operating expenses: |
|||||||||||||||||
Research and development |
|
22,367 |
|
|
8,381 |
|
|
72,088 |
|
|
30,911 |
|
|||||
General and administrative |
|
3,611 |
|
|
3,663 |
|
|
13,088 |
|
|
11,186 |
|
|||||
Total operating expenses |
|
25,978 |
|
|
12,044 |
|
|
85,176 |
|
|
42,097 |
|
|||||
Loss from operations |
|
(25,978 |
) |
|
(11,280 |
) |
|
(84,856 |
) |
|
(39,736 |
) |
|||||
Other (expense) income |
|
(4,397 |
) |
|
(51 |
) |
|
(13,709 |
) |
|
489 |
|
|||||
Net loss |
$ |
(30,375 |
) |
$ |
(11,331 |
) |
$ |
(98,565 |
) |
$ |
(39,247 |
) |
|||||
Net loss per common share, basic and diluted |
$ |
(1.03 |
) |
$ |
(0.39 |
) |
$ |
(3.39 |
) |
$ |
(1.43 |
) |
|||||
Weighted average common shares outstanding, basic and diluted |
|
29,397,718 |
|
|
28,762,962 |
|
|
29,056,304 |
|
|
27,493,558 |
|
View source version on businesswire.com: https://www.businesswire.com/news/home/20210315005223/en/
Investor Contact:
Chief Financial Officer
(610) 981-6506
john.sharp@phasebio.com
Media Contact:
will.zasadny@canalecomm.com
(619) 961-8848
Source: