Prothena Reports Fourth Quarter and Full Year 2023 Financial Results, and Provides Financial Guidance and Business Highlights
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Net cash used in operating and investing activities was
$52.6 million in the fourth quarter and$136.7 million for the full year of 2023; quarter-end cash and restricted cash position was$621.0 million -
The company expects cash guidance for the full year 2024 net cash used in operating and investing activities to be
$208 to$225 million and expects to end the year with approximately$405 million in cash (midpoint) -
Advanced potential best-in-class Alzheimer’s disease portfolio in 2023: initial data supportive of ongoing Phase 1 clinical trial for PRX012, an anti-amyloid beta antibody; received FDA clearance for IND application and Fast Track designation for PRX123, a dual amyloid beta/tau vaccine; reported Phase 1 data for BMS-986446 (formerly PRX005), an anti-tau antibody, data supports moving into a Phase 2 clinical trial by partner
Bristol Myers Squibb -
Strengthened leadership position in the amyloidosis community with ongoing enrollment of the confirmatory Phase 3 AFFIRM-AL clinical trial of birtamimab in patients with Mayo Stage
IV AL amyloidosis; published Phase 3 VITAL clinical trial data in Blood, the peer-reviewed journal of ASH -
Received
$55 million milestone payment fromBristol Myers Squibb inJuly 2023 for exclusive worldwide license to BMS-986446 (formerly PRX005)
“2023 was a year of strong progress for
2023 Business Highlights and Upcoming Milestones
Neurodegenerative Diseases Portfolio
Alzheimer’s Disease
PRX012, a wholly-owned potential best-in-class, next-generation subcutaneous antibody 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
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Presented two preclinical studies at AD/PD in
March 2023 and AAIC inJuly 2023 showing superior binding characteristics of PRX012 -
Partnered with Walgreens in
April 2023 to accelerate patient identification and recruitment for ongoing ASCENT-2 clinical trial - Initial Phase 1 single ascending dose (SAD) and multiple ascending dose (MAD) data supports once-monthly subcutaneous administration and ongoing evaluation in MAD cohorts
- Ongoing 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. BMS-986446 is part of a
-
Presented Phase 1 clinical trial SAD results in a poster presentation at AAIC in
July 2023 showing that all three tested dose levels (low, medium, high) of PRX005 were considered generally safe and well tolerated, meeting the primary objective of this part of the clinical trial and supporting evaluation of doses in the ongoing MAD portion of this two-part clinical trial -
Bristol Myers Squibb paid$55 million for exclusive worldwide rights for PRX005 inJuly 2023 under theGlobal Neuroscience Research and Development Collaboration -
Bristol Myers Squibb will be responsible for future development, manufacturing, and commercialization of BMS-986446 -
Bristol Myers Squibb reported that Phase 1 data supports moving BMS-986446 into a Phase 2 clinical trial in 1H 2024
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
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Presented preclinical results in a late breaker poster presentation at AAIC in
July 2023 showing a PRX123 vaccine surrogate elicited robust antibody responses that bound with high avidity to Aβ plaques in Alzheimer’s disease brain tissue ex vivo and significantly reduced Aβ brain plaques - Investigational new drug (IND) application cleared by FDA
- Fast Track designation granted by FDA
- 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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Roche completed enrollment for the Phase 2b
PADOVA clinical trial in patients with early Parkinson’s disease in the first quarter of 2023 -
Poster and oral presentations at AD/PD in March/
April 2023 highlighted aspects of the Phase 2 PASADENA clinical trial of prasinezumab for the treatment of Parkinson’s disease -
Roche presented data at the
International Congress of Parkinson’s Disease and Movement Disorders (MDS) from the open-label extension of the PASADENA clinical trial which shows that prasinezumab slowed the progression of motor deficits (MDS-UPDRS Part III OFF state score) in early-stage Parkinson’s disease -
Topline results from Phase 2b
PADOVA clinical trial expected in 2024 (NCT04777331)
Neurodegenerative Diseases
PRX019, a potential treatment of neurodegenerative diseases with an undisclosed target, is part of a
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IND application cleared by FDA in
December 2023 - Phase 1 clinical trial timeline update expected in 2024
Rare Peripheral Amyloid Diseases Portfolio
AL Amyloidosis
Birtamimab, a wholly-owned potential best-in-class amyloid depleter 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
-
Published Phase 3 VITAL clinical trial data in
June 2023 in Blood, the peer-reviewed journal ofAmerican Society of Hematology (ASH) -
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
NNC6019 (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 Novo Nordisk as part of their up to
- Ongoing Phase 2 clinical trial in patients with ATTR cardiomyopathy is being conducted by Novo Nordisk
- The Phase 2 clinical trial has fully recruited patients with topline data expected in 1H 2025 (NCT05442047)
2023 Organizational and Corporate Highlights
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Announced the appointment of
Billy Dunn , M.D., founding, former Director of theFDA CDER Office of Neuroscience , to its Board of Directors
Fourth Quarter and Full Year of 2023 Financial Results
For the fourth quarter and full year of 2023,
Research and development (R&D) expenses totaled
General and administrative (G&A) expenses totaled
Total non-cash share-based compensation expense was
As of
2024 Financial Guidance
The Company expects the full year 2024 net cash used in operating and investing activities to be
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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, birtamimab, and NNC6019/PRX004; plans for ongoing and future clinical trials of PRX012, BMS-986446/PRX005, PRX123, prasinezumab, PRX019, birtamimab, and NNC6019/PRX004; the expected timing of reporting data from clinical trials, 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; and 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 uncertainties related to the completion of operational and financial closing procedures, audit adjustments and other developments that may arise that would require adjustments to the preliminary financial results included in this press release, as well as those described in the “Risk Factors” sections of our Quarterly Report on Form 10-Q filed with the
CONSOLIDATED STATEMENTS OF OPERATIONS (unaudited - amounts in thousands except per share data) |
||||||||||||||||
|
|
Three Months Ended
|
|
Year Ended |
||||||||||||
|
|
|
2023 |
|
|
|
2022 |
|
|
|
2023 |
|
|
|
2022 |
|
Collaboration revenue |
|
$ |
316 |
|
|
$ |
9,923 |
|
|
$ |
91,320 |
|
|
$ |
13,855 |
|
Revenue from license and intellectual property |
|
|
— |
|
|
|
40,000 |
|
|
|
50 |
|
|
|
40,050 |
|
Total revenue |
|
|
316 |
|
|
|
49,923 |
|
|
|
91,370 |
|
|
|
53,905 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
||||||||
Research and development |
|
|
61,891 |
|
|
|
36,871 |
|
|
|
220,571 |
|
|
|
135,562 |
|
General and administrative |
|
|
16,940 |
|
|
|
13,124 |
|
|
|
61,835 |
|
|
|
49,900 |
|
Total operating expenses |
|
|
78,831 |
|
|
|
49,995 |
|
|
|
282,406 |
|
|
|
185,462 |
|
Income (loss) from operations |
|
|
(78,515 |
) |
|
|
(72 |
) |
|
|
(191,036 |
) |
|
|
(131,557 |
) |
Other income (expense), net |
|
|
7,897 |
|
|
|
3,417 |
|
|
|
30,556 |
|
|
|
5,952 |
|
Income (loss) before income taxes |
|
|
(70,618 |
) |
|
|
3,345 |
|
|
|
(160,480 |
) |
|
|
(125,605 |
) |
Provision for (benefit from) income taxes |
|
|
(3,142 |
) |
|
|
(3,004 |
) |
|
|
(13,452 |
) |
|
|
(8,656 |
) |
Net income (loss) |
|
$ |
(67,476 |
) |
|
$ |
6,349 |
|
|
$ |
(147,028 |
) |
|
$ |
(116,949 |
) |
Basic net income (loss) per ordinary share |
|
$ |
(1.26 |
) |
|
$ |
0.13 |
|
|
$ |
(2.76 |
) |
|
$ |
(2.47 |
) |
Diluted net income (loss) per ordinary share |
|
$ |
(1.26 |
) |
|
$ |
0.12 |
|
|
$ |
(2.76 |
) |
|
$ |
(2.47 |
) |
Shares used to compute basic net income (loss) per share |
|
|
53,668 |
|
|
|
48,960 |
|
|
|
53,216 |
|
|
|
47,369 |
|
Shares used to compute diluted net income (loss) per share |
|
|
53,668 |
|
|
|
53,979 |
|
|
|
53,216 |
|
|
|
47,369 |
|
CONSOLIDATED BALANCE SHEETS (unaudited - amounts in thousands) |
|||||
|
|
||||
|
2023 |
|
2022 |
||
Assets |
|
|
|
||
Cash and cash equivalents |
$ |
618,830 |
|
$ |
710,406 |
Restricted cash, current |
|
1,352 |
|
|
— |
Prepaid expenses and other current assets |
|
19,100 |
|
|
8,692 |
Total current assets |
|
639,282 |
|
|
719,098 |
Property and equipment, net |
|
3,836 |
|
|
1,731 |
Operating lease right-of-use assets |
|
12,162 |
|
|
6,277 |
Restricted cash, non-current |
|
860 |
|
|
2,212 |
Other non-current assets |
|
40,242 |
|
|
28,717 |
Total non-current assets |
|
57,100 |
|
|
38,937 |
Total assets |
$ |
696,382 |
|
$ |
758,035 |
Liabilities and Shareholders’ Equity |
|
|
|
||
Accrued research and development |
|
14,724 |
|
|
10,794 |
Deferred revenue, current |
|
— |
|
|
11,442 |
Lease liability, current |
|
1,114 |
|
|
6,473 |
Other current liabilities |
|
41,053 |
|
|
21,438 |
Total current liabilities |
|
56,891 |
|
|
50,147 |
Deferred revenue, non current |
|
67,405 |
|
|
85,293 |
Lease liability, non-current |
|
10,721 |
|
|
— |
Other non-current liabilities |
|
— |
|
|
553 |
Total non-current liabilities |
|
78,126 |
|
|
85,846 |
Total liabilities |
|
135,017 |
|
|
135,993 |
Total shareholders’ equity |
|
561,365 |
|
|
622,042 |
Total liabilities and shareholders’ equity |
$ |
696,382 |
|
$ |
758,035 |
View source version on businesswire.com: https://www.businesswire.com/news/home/20240215091501/en/
Investors
650-417-1974, mark.johnson@prothena.com
Media
609-664-7308, michael.bachner@prothena.com
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