On February 11, 2021 Prothena Corporation plc (NASDAQ:PRTA), a late-stage clinical company with expertise in protein dysregulation and a pipeline of investigational therapeutics for rare peripheral amyloid and neurodegenerative diseases, reported financial results for the fourth quarter and full year 2020 (Press release, Prothena, FEB 11, 2021, View Source [SID1234574889]). In addition, the Company provided 2021 financial guidance and an update on its R&D programs.

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"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 Gene Kinney, Ph.D., President and Chief Executive Officer of Prothena. "We are particularly pleased that our unique protein dysregulation platform has now resulted in the translation of our preclinical findings into clinical benefit for patients across multiple programs in our portfolio. We look forward to a number of significant milestones in 2021, with the planned initiation of 3 late-stage clinical studies, including the birtamimab AFFIRM-AL study, the prasinezumab Phase 2b study and the PRX004 Phase 2/3 study. In addition to these late-stage programs, we have also advanced the three targets under our global neuroscience collaboration with Bristol-Myers Squibb and expect to file an IND for PRX005 this year. Our strong cash position and potential partner payments provide a foundational capital position to fund the company through value-creating milestones as we transition to a fully integrated commercial company."

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 in February 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 2020 Congress. 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 in November 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 joined Prothena 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 at 10:00 AM ET
Stifel’s 3rd Annual CNS Day on Thursday April 1, 2021 at 8:00 AM ET
Fourth Quarter and Full Year of 2020 Financial Results and 2021 Financial Guidance

For the fourth quarter and full year of 2020, Prothena reported a net loss of $30.7 million and $111.1 million, respectively, as compared to a net loss of $21.6 million and $77.7 million for the fourth quarter and full year of 2019, respectively. Net loss per share for the fourth quarter and full year of 2020 was $0.77 and $2.78, respectively, as compared to a net loss per share of $0.54 and $1.95 for the fourth quarter and full year of 2019, respectively.

Prothena reported total revenue of $0.4 million and $0.9 million for the fourth quarter and full year of 2020, respectively, primarily due to license revenue in the fourth quarter and collaboration revenue for the full year as compared to total revenue of $0.3 million and $0.8 million for the fourth quarter and full year of 2019, respectively, primarily due to Roche collaboration revenue.

Research and development (R&D) expenses totaled $20.8 million and $74.9 million for the fourth quarter and full year of 2020, respectively, as compared to $15.5 million and $50.8 million for the fourth quarter and full year of 2019, respectively. The increase in R&D expense for the fourth quarter and full year of 2020 compared to the same periods in the prior year was primarily due to higher manufacturing costs primarily related to our PRX005, birtamimab and PRX012 programs and to a lesser extent PRX004, higher collaboration expense with Roche related to the prasinezumab program and higher R&D consulting expense. R&D expenses included non-cash share-based compensation expense of $2.1 million and $8.2 million for the fourth quarter and full year of 2020, respectively, as compared to $2.0 million and $8.1 million for the fourth quarter and full year of 2019, respectively.

General and administrative (G&A) expenses totaled $9.9 million and $38.7 million for the fourth quarter and full year of 2020, respectively, as compared to $8.1 million and $35.7 million for the fourth quarter and full year of 2019, respectively. The increase in G&A expenses for the fourth quarter and full year of 2020 compared to the same periods in the prior year was primarily related to higher costs for our director and officer insurance premiums. G&A expenses included non-cash share-based compensation expense of $3.2 million and $13.8 million for the fourth quarter and full year of 2020, respectively, as compared to $3.3 million and $15.5 million for the fourth quarter and full year of 2019, respectively.

Total non-cash share-based compensation expense was $5.2 million and $22.0 million for the fourth quarter and full year of 2020, respectively, as compared to $5.3 million and $23.6 million for the fourth quarter and full year of 2019, respectively.

As of December 31, 2020, Prothena had $298.1 million in cash, cash equivalents and restricted cash and no debt.

As of February 5, 2021, Prothena had approximately 39.9 million ordinary shares outstanding.

The Company expects the full year 2021 net cash used in operating and investing activities to be $51 to $74 million, which includes an expected $60 million milestone payment from Roche upon first patient dosed in the late-stage Phase 2b study of prasinezumab and expects to end the year with approximately $235 million in cash, cash equivalents and restricted cash (midpoint). The estimated full year 2021 net cash used in operating and investing activities is primarily driven by an estimated net loss of $79 to $111 million, which includes an estimated $20 million of non-cash share-based compensation expense.

Conference Call Details

Prothena management will discuss these results and its 2021 financial guidance during a live audio conference call today, Thursday, February 11, 2021, at 8:30 AM ET. The conference call will be made available on the Company’s website at www.prothena.com under the Investors tab in the Events and Presentations section. Following the live audio webcast, a replay will be available on the Company’s website for at least 90 days.

To access the call via dial-in, please dial (877) 887-5215 (U.S. and Canada toll free) or (315) 625-3069 (international) five minutes prior to the start time and refer to conference ID number 5677514. A replay of the call will be available until February 25, 2021 via dial-in at (855) 859-2056 (U.S. toll free) or (404) 537-3406 (international), Conference ID Number 5677514.