Prothena Reports Second Quarter 2024 Financial Results and Business Highlights
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Net cash provided by operating and investing activities was
$15.8 million in the second quarter and net cash used by operating and investing activities was$57.3 million for the first six months of 2024; quarter-end cash and restricted cash position was$565.0 million -
Revised year-end cash guidance to be approximately
$468 million in cash, cash equivalents and restricted cash, representing an increase of$63 million from prior guidance of$405 million -
Bristol Myers Squibb obtained the exclusive global license for PRX019 for$80 million , a potential treatment of neurodegenerative disease with an undisclosed target;Prothena to initiate a Phase 1 clinical trial by YE 2024 -
Partner
Bristol Myers Squibb presented a poster for BMS-986446 at AAIC 2024 of the clinical trial design for the ongoing Phase 2 TargetTau-1 clinical trial to treat Alzheimer’s disease - Presented poster for PRX012 at AAIC 2024 of the clinical trial design for the ongoing Phase 1 ASCENT clinical trial to treat Alzheimer’s disease; program update expected in 2024
- Highlighted poster for birtamimab at ISA 2024 of additional efficacy analysis from the VITAL Phase 3 clinical trial to treat AL amyloidosis; ongoing global confirmatory Phase 3 AFFIRM-AL clinical trial with topline results expected between 4Q 2024 and 2Q 2025
“We continue to meaningfully advance our programs and move towards becoming a fully integrated commercial company, which will enable
Second Quarter, Recent Business Highlights and Upcoming Milestones
Neurodegenerative Diseases Portfolio
Alzheimer’s Disease
PRX012, a wholly-owned potential best-in-class, next-generation antibody delivered subcutaneously for the treatment of Alzheimer’s disease that targets a key epitope at the N-terminus of amyloid beta (Aβ) with high binding potency. The
- Poster presentation at AAIC 2024 highlighted the clinical trial design of the ongoing Phase 1 ASCENT program for PRX012
- Initial Phase 1 single ascending dose and multiple dose data supports once-monthly subcutaneous administration and ongoing evaluation in multiple dose cohorts
- Phase 1 clinical trial continues as planned and expect to update in 2024
BMS-986446 (formerly PRX005), a potential best-in-class antibody for the treatment of Alzheimer’s disease that specifically targets a key epitope within the microtubule binding region (MTBR) of tau, a protein implicated in the causal pathophysiology of Alzheimer’s disease.
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Poster presentation by partner
Bristol Myers Squibb at AAIC 2024 highlighted the design of the ongoing Phase 2 TargetTau-1 clinical trial for BMS-986446 -
Bristol Myers Squibb continues to enroll the ongoing Phase 2 clinical trial in approximately 475 patients with early Alzheimer’s disease for BMS-986446 (NCT06268886) -
Bristol Myers Squibb is responsible for all development, manufacturing, and commercialization of BMS-986446
PRX123, a wholly-owned potential first-in-class dual Aβ/tau vaccine designed for the treatment and prevention of Alzheimer’s disease, is a dual-target vaccine targeting key epitopes within the N-terminus of Aβ and MTBR-tau designed to promote amyloid clearance and block the transmission of pathogenic tau. The FDA cleared the investigational new drug (IND) application and granted Fast Track designation for PRX123 for the treatment of Alzheimer’s disease.
- Phase 1 timeline update expected in 2024
Parkinson’s Disease
Prasinezumab, a potential first-in-class antibody for the treatment of Parkinson’s disease that is designed to target key epitopes within the C-terminus of alpha-synuclein, and is the focus of a worldwide collaboration with Roche.
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Topline results from Phase 2b
PADOVA clinical trial in patients with early Parkinson’s disease, which has completed enrollment of 586 patients, expected in 2H 2024 (NCT04777331)
Neurodegenerative Diseases
PRX019, a potential treatment of neurodegenerative diseases with an undisclosed target.
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Bristol Myers Squibb obtained the exclusive global license for PRX019 for$80 million -
As part of the PRX019 global license with
Bristol Myers Squibb ,Prothena will be eligible to receive additional development, regulatory, and sales milestone payments of up to$617.5 million and tiered royalties on net sales -
Prothena will initiate a Phase 1 clinical trial for PRX019 by year-end 2024
Rare Peripheral Amyloid Diseases Portfolio
AL Amyloidosis
Birtamimab, a wholly-owned potential best-in-class anti-amyloid antibody for the treatment of AL amyloidosis designed to directly neutralize soluble toxic light chain aggregates and promote clearance of amyloid that causes organ dysfunction and failure. Among patients with AL amyloidosis, a rare, progressive, and fatal disease, newly diagnosed individuals with advanced disease (e.g., Mayo Stage IV) are at the highest risk for early death. Birtamimab has been granted Fast Track designation by the FDA for the treatment of patients with Mayo Stage
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Longitudinal Health-Related Quality of Life data (SF-36v2) across domains from the VITAL Phase 3 clinical trial for birtamimab was presented at the
International Society of Amyloidosis (ISA) 2024 meeting -
The ongoing confirmatory Phase 3 AFFIRM-AL clinical trial in patients with Mayo Stage
IV AL amyloidosis is being conducted under a Special Protocol Assessment (SPA) agreement with the FDA with a primary endpoint of all-cause mortality (time-to-event) at a significance level of 0.10 - Topline results from confirmatory AFFIRM-AL Phase 3 clinical trial expected between 4Q 2024 and 2Q 2025 (NCT04973137)
ATTR Amyloidosis
Coramitug (formerly PRX004), a potential first-in-class amyloid depleter antibody for the treatment of ATTR cardiomyopathy designed to deplete the pathogenic, non-native forms of the transthyretin (TTR) protein and is being developed by
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Ongoing Phase 2 clinical trial in patients with ATTR cardiomyopathy is being conducted by
Novo Nordisk - Phase 2 clinical trial has completed enrollment of approximately 99 patients with topline data expected in 1H 2025 (NCT05442047)
Second Quarter and First Six Months of 2024 Financial Results
For the second quarter and first six months of 2024,
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
2024 Financial Guidance
The Company is updating it projected full year 2024 net cash used in operating and investing actives, 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 cash position to fund advancement of a broad pipeline and completion of our ongoing clinical trials; the continued advancement of our discovery, preclinical, and clinical pipeline, and expected milestones in 2024, 2025, and beyond; the treatment potential, designs, proposed mechanisms of action, and potential administration of PRX012, BMS-986446/PRX005, PRX123, prasinezumab, PRX019, birtamimab, and coramitug/PRX004; plans for ongoing and future clinical studies of PRX012, BMS-986446/PRX005, PRX123, prasinezumab, PRX019, birtamimab, and coramitug/PRX004; the expected timing of reporting data from clinical studies, including any updates regarding our ongoing Phase 1 clinical trial evaluating PRX012 in 2024 and topline study results for our Phase 3 AFFIRM-AL clinical trial between 4Q 2024 and 2Q 2025; amounts we might receive under our collaboration with BMS; our anticipated net cash burn from operating and investing activities for 2024 and expected cash balance at the end of 2024; and our estimated net loss and non-cash share-based compensation expense for 2024. 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 those described in the “Risk Factors” sections of our Quarterly Report on Form 10-Q filed with the
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CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS |
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(unaudited - amounts in thousands except per share data) |
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Three Months Ended
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Six Months Ended
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|
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2024 |
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|
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2023 |
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|
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2024 |
|
|
|
2023 |
|
Collaboration revenue |
|
$ |
132,014 |
|
|
$ |
4,019 |
|
|
$ |
132,014 |
|
|
$ |
6,138 |
|
Revenue from license and intellectual property |
|
|
— |
|
|
|
— |
|
|
|
50 |
|
|
|
50 |
|
Total revenue |
|
|
132,014 |
|
|
|
4,019 |
|
|
|
132,064 |
|
|
|
6,188 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
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Research and development |
|
|
57,510 |
|
|
|
56,011 |
|
|
|
121,624 |
|
|
|
100,767 |
|
General and administrative |
|
|
16,127 |
|
|
|
14,512 |
|
|
|
33,591 |
|
|
|
28,250 |
|
Total operating expenses |
|
|
73,637 |
|
|
|
70,523 |
|
|
|
155,215 |
|
|
|
129,017 |
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Income (loss) from operations |
|
|
58,377 |
|
|
|
(66,504 |
) |
|
|
(23,151 |
) |
|
|
(122,829 |
) |
Total other income, net |
|
|
6,470 |
|
|
|
7,603 |
|
|
|
13,558 |
|
|
|
14,152 |
|
Income (loss) before income taxes |
|
|
64,847 |
|
|
|
(58,901 |
) |
|
|
(9,593 |
) |
|
|
(108,677 |
) |
Benefit from income taxes |
|
|
(2,039 |
) |
|
|
(4,306 |
) |
|
|
(4,240 |
) |
|
|
(7,218 |
) |
Net income (loss) |
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$ |
66,886 |
|
|
$ |
(54,595 |
) |
|
$ |
(5,353 |
) |
|
$ |
(101,459 |
) |
Basic net income (loss) per ordinary share |
|
$ |
1.24 |
|
|
$ |
(1.03 |
) |
|
$ |
(0.10 |
) |
|
$ |
(1.92 |
) |
Diluted net income (loss) per ordinary share |
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$ |
1.22 |
|
|
$ |
(1.03 |
) |
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$ |
(0.10 |
) |
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$ |
(1.92 |
) |
Shares used to compute basic net income (loss) per share |
|
|
53,767 |
|
|
|
53,121 |
|
|
|
53,740 |
|
|
|
52,812 |
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Shares used to compute diluted net income (loss) per share |
|
|
55,043 |
|
|
|
53,121 |
|
|
|
53,740 |
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|
|
52,812 |
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CONDENSED CONSOLIDATED BALANCE SHEETS |
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(unaudited - amounts in thousands) |
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2024 |
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2023 |
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Assets |
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|
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Cash and cash equivalents |
$ |
564,124 |
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$ |
618,830 |
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Restricted cash, current |
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— |
|
|
1,352 |
|
Prepaid expenses and other current assets |
|
21,854 |
|
|
19,100 |
|
Total current assets |
|
585,978 |
|
|
639,282 |
|
Property and equipment, net |
|
3,486 |
|
|
3,836 |
|
Operating lease right-of-use assets |
|
12,066 |
|
|
12,162 |
|
Restricted cash, non-current |
|
860 |
|
|
860 |
|
Other non-current assets |
|
43,175 |
|
|
40,242 |
|
Total non-current assets |
|
59,587 |
|
|
57,100 |
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Total assets |
$ |
645,565 |
|
$ |
696,382 |
|
Liabilities and Shareholders’ Equity |
|
|
|
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Accrued research and development |
|
15,927 |
|
|
14,724 |
|
Deferred revenue, current |
|
8,025 |
|
|
— |
|
Lease liability, current |
|
2,579 |
|
|
1,114 |
|
Other current liabilities |
|
20,213 |
|
|
41,053 |
|
Total current liabilities |
|
46,744 |
|
|
56,891 |
|
Deferred revenue, non-current |
|
7,366 |
|
|
67,405 |
|
Lease liability, non-current |
|
9,538 |
|
|
10,721 |
|
Total non-current liabilities |
|
16,904 |
|
|
78,126 |
|
Total liabilities |
|
63,648 |
|
|
135,017 |
|
Total shareholders’ equity |
|
581,917 |
|
|
561,365 |
|
Total liabilities and shareholders’ equity |
$ |
645,565 |
|
$ |
696,382 |
View source version on businesswire.com: https://www.businesswire.com/news/home/20240808568805/en/
Investors
650-417-1974, mark.johnson@prothena.com
Media
609-664-7308, michael.bachner@prothena.com
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