<< Back
Feb 11, 2021
Prothena Reports Fourth Quarter and Full Year 2020 Financial Results, and Provides Financial Guidance and R&D Update
Prothena Reports Fourth Quarter and Full Year 2020 Financial Results, and Provides Financial Guidance and R&D Update
- Net cash used in operating and investing activities was
$19.1 million in the fourth quarter and$80.6 million for the full year 2020; quarter-end cash and restricted cash position of$298.1 million provides funding to advance the R&D pipeline
- Announced confirmatory Phase 3 AFFIRM-AL study of birtamimab in Mayo Stage IV patients with AL amyloidosis under SPA agreement with FDA at p≤0.10
- Reported significantly reduced decline in motor function in Phase 2 study of prasinezumab in Parkinson’s disease
- Reported improvements in neuropathy and cardiac function in Phase 1 study of PRX004 in ATTR amyloidosis
“We continue to see positive momentum in our pipeline with the recent announcement of the confirmatory, registration-enabling Phase 3 AFFIRM-AL study of birtamimab in AL amyloidosis and positive clinical findings reported in 2020 from both the Phase 2 study of prasinezumab in Parkinson’s disease and Phase 1 study of PRX004 in ATTR amyloidosis,” said
Full Year 2020 and Recent Developments:
Birtamimab, a potential treatment for AL amyloidosis, is a humanized monoclonal antibody designed to directly neutralize soluble toxic aggregates and promote clearance of amyloid that causes organ dysfunction and failure
- Based on significant survival benefit observed in the previous VITAL study in Mayo Stage IV patients (HR=0.413, p=0.025, over 9 months) and multiple in-depth discussions with the
U.S. Food and Drug Administration (FDA),Prothena announced plans inFebruary 2021 to advance birtamimab into the confirmatory Phase 3 AFFIRM-AL study in Mayo Stage IV patients with AL amyloidosis. AFFIRM-AL is a global, registration-enabling Phase 3 study that will be conducted with a primary endpoint of all-cause mortality at p≤0.10 under a Special Protocol Assessment (SPA) agreement with FDA.
Prasinezumab, a potential treatment for Parkinson’s disease, is a humanized monoclonal antibody designed to target key epitopes within the C-terminus of alpha-synuclein and is the focus of the worldwide collaboration with Roche
- Presented results from Phase 2 PASADENA study showing prasinezumab significantly slows progression on pre-specified clinical measures of Parkinson’s disease in
September 2020 at the International Parkinson and Movement Disorder Society’s 2020Congress . Prasinezumab is the first potentially disease-modifying, anti-alpha-synuclein antibody to demonstrate signals of efficacy on multiple pre-specified secondary and exploratory clinical endpoints in patients with early Parkinson’s disease. - Announced that Roche and
Prothena will advance prasinezumab into a late-stage Phase 2b study in patients with early Parkinson’s disease. The study will be designed to further assess the efficacy of prasinezumab by expanding upon the patient population enrolled in PASADENA to include patients with early Parkinson’s disease on stable levodopa therapy. Prasinezumab is the first anti-alpha synuclein antibody to advance into late-stage development.
PRX004, a potential treatment for ATTR amyloidosis, is a humanized monoclonal antibody designed to deplete the pathogenic, non-native forms of the TTR protein
- Reported results from the Phase 1 study of PRX004, the first anti-amyloid immunotherapy designed to deplete amyloid to demonstrate efficacy in ATTR amyloidosis. In the first report of clinical results with this depleter mechanism of action, PRX004 showed favorable results as demonstrated by slowing of neuropathy progression for all 7 evaluable patients at 9 months, including improvement in neuropathy in 3 of the 7 patients, and improved cardiac systolic function for all 7 patients. In this Phase 1 study, PRX004 was found to be generally safe and well tolerated across all dose levels.
PRX012, a potential treatment for Alzheimer’s disease, is monoclonal antibody targeting key epitopes within the N-terminus of Aβ
- Presented data from next generation anti-Aβ antibodies for subcutaneous administration to improve patient access at the 13th Clinical Trials on Alzheimer’s Disease (CTAD) Conference in
November 2020
Multi-immunogen vaccine, for the potential prevention and treatment of Alzheimer’s disease, is a multi-immunogen Aβ-tau vaccine
- Presented data on a multi-immunogen vaccine that targets key Aβ and tau epitopes, the two main pathological proteins involved in the cause and progression of Alzheimer’s disease, at the
CTAD Conference inNovember 2020
Corporate
- Appointed
Brandon Smith as Chief Business Officer, responsible for leading Prothena’s business development activities, portfolio strategic planning and alliance management activities.Mr. Smith joinedProthena after serving as Chief Operating Officer at Iconic Therapeutics
Upcoming Milestones:
Birtamimab
- Phase 3 AFFIRM-AL study initiation expected mid-2021
- VITAL study 9-month results expected to be presented at medical conference in 2021
Prasinezumab
- New pre-specified exploratory subgroup analyses from Part 1 of the Phase 2 PASADENA study to be presented at the 15th International Conference for Alzheimer’s and Parkinson’s Diseases in
March 2021 (ADPD 2021) $60 million clinical milestone payment to be achieved upon first patient dosed in late-stage Phase 2b study in patients with early Parkinson’s disease; further details expected in 2Q 2021- Results from Part 2 of the PASADENA study expected to be presented at an upcoming medical conference
PRX004
- Phase 1 study results expected to be presented at medical conference in 2021
- Phase 2/3 study in patients with ATTR-cardiomyopathy expected to initiate 4Q 2021
PRX005
- Preclinical data to be presented in March at ADPD 2021
- IND filing expected 3Q 2021
$80 million potential payment from Bristol-Myers Squibb upon exercising their US license option in 2021
PRX012
- IND filing expected in 1Q 2022
Upcoming Investor Conferences
Members of the senior management team will present and participate in investor meetings at the following upcoming investor conferences:
H.C. Wainwright Global Life Sciences Conference on Tuesday and Wednesday,March 9-10, 2021 , virtual presentations will be available on demand both days- Oppenheimer 31st Annual Healthcare Conference on
Wednesday March 17, 2021 at10:00 AM ET - Stifel’s 3rd Annual CNS Day on
Thursday April 1, 2021 at8:00 AM ET
Fourth Quarter and Full Year of 2020 Financial Results and 2021 Financial Guidance
For the fourth quarter and full year of 2020,
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 expects the full year 2021 net cash used in operating and investing activities to be
Conference Call Details
To access the call via dial-in, please dial (877) 887-5215 (
About
Forward-looking Statements
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; our goal of building a protein dysregulation platform; the treatment potential and proposed mechanisms of action of birtamimab, prasinezumab, PRX004, PRX005, PRX012, and multi-immunogen Aβ-tau vaccine; plans for future clinical studies of birtamimab, prasinezumab, PRX004, PRX005, and PRX012; amounts we might receive under our collaborations with Roche and Bristol-Myers Squibb; the expected timing of reporting data from prior clinical studies of birtamimab, the Phase 2 clinical study of prasinezumab, and preclinical studies of PRX005; our anticipated net cash burn from operating and investing activities for 2021 and expected cash balance at the end of 2021; and our estimated net loss and non-cash share-based compensation expense for 2021. 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 |
Twelve Months Ended |
|||||||||||||||
| 2020 | 2019 | 2020 | 2019 | |||||||||||||
| Collaboration revenue | $ | 121 | $ | 256 | $ | 564 | $ | 814 | ||||||||
| License revenue | 239 | — | 289 | — | ||||||||||||
| Total revenue | 360 | 256 | 853 | 814 | ||||||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | 20,760 | 15,471 | 74,884 | 50,836 | ||||||||||||
| General and administrative | 9,908 | 8,059 | 38,703 | 35,736 | ||||||||||||
| Restructuring (credits) | — | — | — | (61 | ) | |||||||||||
| Total operating expenses | 30,668 | 23,530 | 113,587 | 86,511 | ||||||||||||
| Loss from operations | (30,308 | ) | (23,274 | ) | (112,734 | ) | (85,697 | ) | ||||||||
| Other income (expense), net | (55 | ) | 1,589 | 1,307 | 8,399 | |||||||||||
| Loss before income taxes | (30,363 | ) | (21,685 | ) | (111,427 | ) | (77,298 | ) | ||||||||
| Provision for (benefit from) income taxes | 353 | (131 | ) | (283 | ) | 379 | ||||||||||
| Net loss | $ | (30,716 | ) | $ | (21,554 | ) | $ | (111,144 | ) | $ | (77,677 | ) | ||||
| Basic and diluted net loss per share | $ | (0.77 | ) | $ | (0.54 | ) | $ | (2.78 | ) | $ | (1.95 | ) | ||||
| Shares used to compute basic and diluted net loss per share | 39,921 | 39,897 | 39,915 | 39,882 | ||||||||||||
CONSOLIDATED BALANCE SHEETS
(unaudited - amounts in thousands)
| 2020 | 2019 | |||||||
| Assets | ||||||||
| Cash and cash equivalents | $ | 295,380 | $ | 375,723 | ||||
| Accounts receivable | 15 | 68 | ||||||
| Prepaid expenses and other current assets | 2,537 | 2,584 | ||||||
| Restricted cash, current | 1,352 | — | ||||||
| Total current assets | 299,284 | 378,375 | ||||||
| Property and equipment, net | 2,551 | 3,874 | ||||||
| Operating lease right-of-use assets | 17,811 | 23,274 | ||||||
| Restricted cash, non-current | 1,352 | 2,704 | ||||||
| Other non-current assets | 11,977 | 11,041 | ||||||
| Total non-current assets | 33,691 | 40,893 | ||||||
| Total assets | $ | 332,975 | $ | 419,268 | ||||
| Liabilities and Shareholders’ Equity | ||||||||
| Accrued research and development | 9,044 | 5,826 | ||||||
| Lease liability, current | 5,512 | 5,101 | ||||||
| Other current liabilities | 11,292 | 6,787 | ||||||
| Total current liabilities | 25,848 | 17,714 | ||||||
| Deferred revenue, non current | 110,242 | 110,242 | ||||||
| Lease liability, non-current | 12,326 | 17,838 | ||||||
| Other non-current liabilities | 553 | 553 | ||||||
| Total non-current liabilities | 123,121 | 128,633 | ||||||
| Total liabilities | 148,969 | 146,347 | ||||||
| Total shareholders’ equity | 184,006 | 272,921 | ||||||
| Total liabilities and shareholders’ equity | $ | 332,975 | $ | 419,268 | ||||
Contacts:
Media
650-922-2405, ellen.rose@prothena.com
Investors
650-837-8535, jennifer.zibuda@prothena.com
Source: Prothena Corporation plc