Prothena Reports First Quarter 2020 Financial Results and Provides R&D Update and Updated Financial Guidance
- Net cash used in operating and investing activities was
$23.2 millionin the first quarter; quarter-end cash and restricted cash position of $355.4 millionprovides funding to advance a broad pipeline
“While the COVID-19 pandemic has brought significant challenges to people around the world and to our industry,
First Quarter 2020 and Recent Developments
- The Company continues to monitor the potential impacts of the COVID-19 pandemic to its business. Adaptations to the work environment have been implemented and the Company’s laboratories have remained productive. The known and potential impact to active clinical studies is described in more detail below.
Prothenahas drug supply for its ongoing studies and currently does not expect delays to its programs due to manufacturing or supply chain issues.
- As part of Roche’s first quarter earnings announcement, an update was provided on Part 1 of the Phase 2 PASADENA study of prasinezumab in patients with early Parkinson’s disease. As updated by Roche, the study did not meet the primary objective, but showed signals of efficacy. These signals were observed on multiple prespecified secondary and exploratory clinical endpoints. Roche has begun further clinical development planning activities and is evaluating the data from Part 1 of the PASADENA study to determine next steps. Based on ongoing evaluation of the data, including potential discussions with health authorities, a further update on prasinezumab is expected later this year.
- In a virtual oral presentation at the Advances in Alzheimer’s and Parkinson’s Therapies AAT-AD/PD Focus Meeting (AAT-AD/PD), Roche presented baseline data from the Phase 2 PASADENA study of prasinezumab in patients with early Parkinson's disease. The presentation, titled A Phase 2 study to evaluate the safety and efficacy of prasinezumab in early Parkinson's disease (PASADENA): rationale, design and baseline data is posted on www.prothena.com here. The presentation noted that the PASADENA study population can be considered representative of a wider Parkinson’s disease population, such as the one studied in the Parkinson's Progression Markers Initiative (PPMI) and is therefore suitable for testing the potential beneficial effect of drugs acting on disease progression, such as prasinezumab.
- The Company appointed
Brandon Smithas Chief Business Officer to lead Prothena’s business development initiatives, portfolio strategic planning and alliance management activities. Mr. Smithhas extensive corporate, business development and operational expertise, and has held a number of executive roles at biotechnology companies.
Research and Development Updates and Upcoming Milestones
Prasinezumab (PRX002/RG7935), a potential treatment for Parkinson’s disease, is a monoclonal antibody designed to target alpha-synuclein and is the focus of the worldwide collaboration with Roche
- Part 1 of the Phase 2 PASADENA study in patients with early Parkinson’s disease (N=316) being conducted by Roche is complete. Based on ongoing evaluation of Part 1 PASADENA study data, including potential discussions with health authorities, a further update on prasinezumab is expected later this year.
- The 52-week blinded extension of the study (Part 2 of the Phase 2 PASADENA Study) is ongoing. Due to the COVID-19 pandemic, patients have missed assessments in Part 2 of the study. The full extent of the COVID-19 disruption to Part 2 is not yet known.
PRX004, a potential treatment for ATTR amyloidosis, is a monoclonal antibody designed to deplete the pathogenic, non-native forms of the TTR protein
- The Phase 1 study of PRX004 is fully enrolled and as of early March patients in all 6 cohorts had received the three infusions and assessments that comprise the dose-escalation portion of the study. Interim data from cohorts 1 through 5 was reported in December.
- At the conclusion of the dose-escalation portion of the study, patients meeting eligibility requirements were provided the option to enroll in the long-term-extension (LTE) portion of the study, which allows for up to 15 additional infusions per patient and is designed to further assess safety, tolerability, pharmacokinetics and pharmacodynamics of PRX004. The LTE also includes certain clinical outcome measures for patients in cohorts 4 through 6 at the month 10 assessment (prior to infusion) and at the end of study (month 19 assessment). As of early April, patients in cohorts 1 through 5 in the LTE had completed at least their month 12 assessment and one patient in cohort 6 had completed their month 10 assessment.
- Because of the disruptions caused by the COVID-19 pandemic, some patients have had to discontinue from the study and most patients in the LTE portion of the study have missed at least one infusion and/or assessment. The Company continues to monitor the impact that COVID-19 will have on the LTE portion of the study based on the fluid circumstances at each of the sites in
the United Statesand Europe. If the COVID-19 pandemic results in missed visits over a prolonged period, it is possible that more patients may not be able to complete the LTE portion of the study.
- The Company believes the study has advanced sufficiently to determine next steps for the program and has begun further clinical development planning activities. The Company currently expects additional data from the dose-escalation and LTE portions of the study, as well as an update on next steps for clinical development, to be reported later this year. This timing, however, is dependent on any additional impacts of COVID-19.
Discovery and Preclinical Development:
- The Company continues to expect to advance IND-enabling activities in 2020 for our preclinical tau program, part of a global neuroscience collaboration with Bristol-Myers Squibb
- The Company continues to expect to initiate IND-enabling activities in 2020 for our preclinical Aβ program
First Quarter 2020 Financial Results and Updated 2020 Financial Guidance
For the first quarter of 2020,
Research and development (R&D) expenses totaled
General and administrative (G&A) expenses totaled
Total non-cash share-based compensation expense was
The Company is updating its projected full year 2020 net cash burn from operating and investing activities, and expects it to be
This press release contains forward-looking statements. These statements relate to, among other things, the sufficiency of our cash position to fund advancement of a broad pipeline; the treatment potential and proposed mechanisms of action of prasinezumab and PRX004; plans for the ongoing Phase 2 clinical study of prasinezumab and the ongoing Phase 1 clinical study of PRX004; the expected timing of reporting data from the Phase 1 clinical study of PRX004 and from the Phase 2 clinical study of prasinezumab; the continued advancement of our discovery and preclinical pipeline; the timing of IND-enabling activities from our tau and Aβ programs; our anticipated net cash burn from operating and investing activities for 2020 and expected cash balance at the end of 2020; and our estimated net loss and non-cash share-based compensation expense for 2020. These statements are based on estimates, projections and assumptions that may prove not to be accurate, and actual results could differ materially from those anticipated due to known and unknown risks, uncertainties and other factors, including but not limited to the effects on our business of the worldwide COVID-19 pandemic and the risks, uncertainties and other factors described in the “Risk Factors” sections of our Annual Report on Form 10-K filed with the
CONSOLIDATED STATEMENTS OF OPERATIONS
(unaudited - amounts in thousands except per share data)
|Three Months Ended
|Research and development||15,248||13,296|
|General and administrative||9,741||9,905|
|Total operating expenses||24,989||23,140|
|Loss from operations||(24,848||)||(22,954||)|
|Other income, net||1,113||2,287|
|Loss before income taxes||(23,735||)||(20,667||)|
|Provision for (benefit from) income taxes||(166||)||198|
|Basic and diluted net loss per share||$||(0.59||)||$||(0.52||)|
|Shares used to compute basic and diluted net loss per share||39,909||39,864|
CONSOLIDATED BALANCE SHEETS
(unaudited - amounts in thousands)
|Cash and cash equivalents||$||352,685||$||375,723|
|Prepaid expenses and other current assets||6,832||2,584|
|Total current assets||359,691||378,375|
|Property and equipment, net||3,531||3,874|
|Operating lease right-of-use assets||21,929||23,274|
|Restricted cash, non-current||2,704||2,704|
|Other non-current assets||11,566||11,041|
|Total non-current assets||39,730||40,893|
|Liabilities and Shareholders’ Equity|
|Accrued research and development||$||5,223||$||5,826|
|Lease liability, current||5,202||5,101|
|Other current liabilities||6,661||6,787|
|Total current liabilities||17,086||17,714|
|Lease liability, non-current||16,501||17,838|
|Other non-current liabilities||553||553|
|Total non-current liabilities||127,296||128,633|
|Total shareholders’ equity||255,039||272,921|
|Total liabilities and shareholders’ equity||$||399,421||$||419,268|
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Source: Prothena Corporation plc