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May 7, 2019
Prothena Reports First Quarter 2019 Financial Results and Provides R&D Update
Prothena Reports First Quarter 2019 Financial Results and Provides R&D Update
- Net cash used in operating and investing activities was
$17.6 million in the first quarter; quarter-end cash and restricted cash position of$414.2 million provides funding to advance neuroscience pipeline - Initiated cell line development of a lead candidate for preclinical tau program
“We continue to advance our neuroscience pipeline of investigational therapies, and are progressing several programs towards key milestones,” said
First Quarter 2019 and
NEOD001 (birtamimab) is an investigational humanized immunoglobulin G1 designed to directly neutralize soluble toxic aggregates and promote clearance of amyloid via phagocytosis to remove organ-deposited amyloid thought to cause organ dysfunction in patients with AL amyloidosis.
- Reported results from the Phase 3 VITAL Amyloidosis Study of NEOD001 (birtamimab) in newly diagnosed, treatment naïve patients with AL amyloidosis and cardiac dysfunction (N=260), which was discontinued in 2018. The final hazard ratio (HR) for the composite primary endpoint (time to all-cause mortality or time to cardiac hospitalization more than 90 days after first infusion of study drug) of 0.835 (95% CI: 0.5799, 1.2011; p=0.330) was consistent with the futility analysis reported in
April 2018 . Post hoc analyses of all-cause mortality revealed a potential survival benefit favoring NEOD001 in the category of patients at highest risk for early mortality (Mayo Stage IV , n=77) with a HR of 0.544 (95% CI: 0.2738, 1.0826; p=0.0787). This potential survival benefit was more pronounced inMayo Stage IV patients during the initial 12 months of treatment, with a HR of 0.498 (95% CI: 0.2404, 1.0304; p=0.0556).
Prasinezumab (PRX002/RG7935), a potential treatment for Parkinson’s disease, is a monoclonal antibody designed to target alpha-synuclein and is the focus of a worldwide collaboration with
- The Phase 2 PASADENA study in patients with early Parkinson’s disease (N=316) is being conducted by
Roche and data from Part 1 of this study are expected in 2020
PRX004, a potential treatment for ATTR amyloidosis, is a monoclonal antibody designed to target and clear the pathogenic, misfolded forms of the TTR protein.
- The Phase 1 study of PRX004 continues to enroll patients with hATTR amyloidosis, and preliminary data from the first four of six dose level cohorts (0.1mg/kg, 0.3 mg/kg,1.0 mg/kg, 3.0 mg/kg, 10.0 mg/kg and 30.0 mg/kg), including safety, tolerability and pharmacodynamics, are expected in the fourth quarter of 2019
Tau is a protein implicated in diseases including Alzheimer's disease (AD), progressive supranuclear palsy (PSP), frontotemporal dementia (
- The preclinical tau program, part of a worldwide collaboration with
Celgene , initiated cell line development of a lead candidate
First Quarter 2019 Financial Results and Updated 2019 Guidance
Research and development (R&D) expenses totaled
General and administrative (G&A) expenses totaled
Total non-cash share-based compensation expense was
As of
As of
The Company is updating its full year 2019 net cash burn from operating and investing activities, and expects it to be
About
Forward-looking Statements
This press release contains forward-looking statements. These statements relate to, among other things: the sufficiency of our funding to advance our neuroscience pipeline; the progress of our neuroscience pipeline of investigational therapies towards key milestones; our expected timing of reporting data from the Phase 2 PASADENA study of prasinezumab; our expected timing of reporting data from the Phase 1 study of PRX004 and the expected scope of that data; our exploration of business development opportunities that could result in further clinical investigation of NEOD001; the design and proposed mechanisms of action of NEOD001 and its potential as a treatment for AL amyloidosis; the results of post hoc analyses that reveal a potential survival benefit favoring NEOD001; the design of prasinezumab and its potential as a treatment for Parkinson’s disease; the design of PRX004 and its potential as a treatment for ATTR amyloidosis; our goal of advancing a research pipeline of novel targets for a number of potential indications; our expected net cash burn from operating and investing activities in 2019 and cash balance at the end of 2019; and our estimated net loss and non-cash share-based compensation expense for 2019. 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 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 March 31, |
||||||||
| 2019 | 2018 | |||||||
| Collaboration revenue | $ | 186 | $ | 227 | ||||
| Total revenue | 186 | 227 | ||||||
| Operating expenses: | ||||||||
| Research and development | 13,296 | 34,706 | ||||||
| General and administrative | 9,905 | 14,229 | ||||||
| Restructuring charges (credits) | (61 | ) | — | |||||
| Total operating expenses | 23,140 | 48,935 | ||||||
| Loss from operations | (22,954 | ) | (48,708 | ) | ||||
| Other income (expense), net | 2,287 | (72 | ) | |||||
| Loss before income taxes | (20,667 | ) | (48,780 | ) | ||||
| Provision for (benefit from) income taxes | 198 | (37 | ) | |||||
| Net loss | $ | (20,865 | ) | $ | (48,743 | ) | ||
| Basic and diluted net loss per share | $ | (0.52 | ) | $ | (1.26 | ) | ||
| Shares used to compute basic and diluted net loss per share | 39,864 | 38,684 | ||||||
CONSOLIDATED BALANCE SHEETS
(unaudited - amounts in thousands)
| March 31, | December 31, | ||||||
| 2019 | 2018 | ||||||
| Assets | |||||||
| Cash and cash equivalents | $ | 410,106 | $ | 427,659 | |||
| Restricted cash, current | 1,352 | — | |||||
| Prepaid expenses and other current assets | 3,735 | 3,731 | |||||
| Total current assets | 415,193 | 431,390 | |||||
| Property and equipment, net | 4,632 | 52,835 | |||||
| Operating lease right-of-use assets | 27,234 | — | |||||
| Restricted cash, non-current | 2,704 | 4,056 | |||||
| Other assets | 9,771 | 10,515 | |||||
| Total non-current assets | 44,341 | 67,406 | |||||
| Total assets | $ | 459,534 | $ | 498,796 | |||
| Liabilities and Shareholders’ Equity | |||||||
| Accrued research and development | $ | 4,738 | $ | 5,370 | |||
| Restructuring liability | — | 461 | |||||
| Operating lease liability, current | 4,810 | — | |||||
| Other current liabilities | 5,358 | 9,095 | |||||
| Total current liabilities | 14,906 | 14,926 | |||||
| Deferred revenue | 110,242 | 110,242 | |||||
| Operating lease liability, non-current | 21,703 | — | |||||
| Other non-current liabilities | 553 | 50,630 | |||||
| Total non-current liabilities | 132,498 | 160,872 | |||||
| Total liabilities | 147,404 | 175,798 | |||||
| Total shareholders’ equity | 312,130 | 322,998 | |||||
| Total liabilities and shareholders’ equity | $ | 459,534 | $ | 498,796 | |||
Media and Investor Contact:
650-922-2405, ellen.rose@prothena.com
Source: Prothena Corporation plc