Prothena Reports Fourth Quarter and Full Year 2023 Financial Results, and Provides Financial Guidance and Business Highlights
- Positive financial results for Q4 2023 and full year with a quarter-end cash position of $621.0 million
- Strong progress in the Alzheimer's disease portfolio with promising Phase 1 data for PRX012
- FDA clearances and Fast Track designation for PRX123 and BMS-986446
- Received a $55 million milestone payment from Bristol Myers Squibb
- Appointment of Dr. Billy Dunn to the board of directors brings regulatory expertise
- Sufficient capital to fund activities beyond ongoing clinical trials
- Net loss of $67.5 million reported for Q4 2023
- Increase in R&D and G&A expenses compared to the previous year
- Estimated net cash used in operating and investing activities for 2024 is $208 to $225 million
- Non-cash share-based compensation expenses impacting financials
Insights
The financial results reported by Prothena Corporation plc indicate a substantial net loss for both the fourth quarter and the full year of 2023, which is a significant shift from the net income reported in the fourth quarter of the previous year. This change reflects increased R&D expenses, which may be attributed to the company's investment in advancing its clinical trials, particularly in the neurodegenerative and rare peripheral amyloid diseases portfolios. The reported increase in G&A expenses due to higher personnel and consulting costs suggests an expansion in operational capacity, potentially in preparation for commercialization activities.
Prothena's partnership with Bristol Myers Squibb, evidenced by the $55 million milestone payment, demonstrates a strategic collaboration that could provide future development and commercialization support. However, the projected net cash used in operating and investing activities for 2024 and the anticipated end-of-year cash position underscore the importance of these clinical developments reaching commercial success to ensure long-term financial sustainability.
The advancement of Prothena's Alzheimer's disease portfolio, especially the progression of PRX012 into ongoing Phase 1 clinical trials and the FDA's Fast Track designation for PRX123, is noteworthy. These developments are crucial as Alzheimer's disease remains a significant unmet medical need with a substantial market potential. The Fast Track designation could expedite the review process, potentially bringing the treatment to market sooner. Furthermore, the confirmatory Phase 3 AFFIRM-AL clinical trial for birtamimab in patients with Mayo Stage IV AL amyloidosis, a condition with high unmet need and limited treatment options, could, if successful, position Prothena as a leader in this therapeutic area.
However, the results of these trials and their subsequent regulatory approvals will be critical in determining the company's future, given the high costs associated with the development of biotechnology therapeutics. The clinical success of these assets will be a determining factor for the company's valuation and its ability to attract further investment or partnerships.
Prothena's strategic initiatives, such as the collaboration with Walgreens to accelerate patient identification and recruitment for clinical trials, demonstrate the company's efforts to streamline its clinical development process. This could potentially reduce time-to-market for its pipeline products. The company's focus on neurodegenerative diseases, specifically Alzheimer's and Parkinson's, aligns with a growing market segment driven by an aging population and increasing prevalence of these conditions.
However, the biotechnology sector is characterized by high volatility and significant risks, especially for companies like Prothena that are in the late-stage clinical phase without a product currently on the market. The outcome of the ongoing and upcoming clinical trials will be closely monitored by investors as they will significantly influence the company's stock performance and market position. Moreover, the anticipated cash runway into the completion of ongoing clinical trials provides a finite window for the company to achieve milestones that could secure additional funding or revenue streams.
-
Net cash used in operating and investing activities was
$52.6 million $136.7 million $621.0 million -
The company expects cash guidance for the full year 2024 net cash used in operating and investing activities to be
$208 $225 million $405 million - 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
“2023 was a year of strong progress for Prothena as we advanced our protein dysregulation portfolio and moved closer to becoming a fully integrated commercial company. The next 12 to 18 months have the potential to transform Prothena with multiple upcoming clinical readouts across our robust portfolio,” said Gene Kinney, Ph.D., President and Chief Executive Officer, Prothena. “We continue to advance our confirmatory Phase 3 AFFIRM-AL clinical trial for birtamimab and for PRX012 are evaluating multiple dose level cohorts in our ongoing Phase 1 clinical trial. In addition, we ended the year with IND clearances by the FDA for both PRX123 and PRX019, including Fast Track designation for PRX123. As we continue to grow our leadership at Prothena, we also appointed the founding, former Director of the FDA CDER Office of Neuroscience, Dr. Billy Dunn, to our board of directors. Dr. Dunn brings immeasurable regulatory and clinical development expertise, combined with a passion for helping patients, which will greatly benefit the millions of people affected by diseases caused by protein dysregulation. Lastly, Prothena remains well financed with sufficient capital to ensure funding of activities beyond the completion of ongoing clinical trials.”
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
- Presented two preclinical studies at AD/PD in March 2023 and AAIC in July 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 Global Neuroscience Research and Development Collaboration with Bristol Myers Squibb.
- 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 - 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
- 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
-
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 Global Neuroscience Research and Development Collaboration with Bristol Myers Squibb.
- 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 IV AL amyloidosis to reduce the risk of mortality and has been granted Orphan Drug Designation by both the FDA and European Medicines Agency. A significant survival benefit was observed in the post hoc analysis of birtamimab-treated patients categorized as Mayo Stage IV at baseline in the previous Phase 3 VITAL clinical trial (Blood 2023).
- Published Phase 3 VITAL clinical trial data in June 2023 in Blood, the peer-reviewed journal of American 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
- Announced the appointment of Billy Dunn, M.D., founding, former Director of the FDA 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, Prothena reported a net loss of
Prothena reported total revenue of
Research and development (R&D) expenses totaled
General and administrative (G&A) expenses totaled
Total non-cash share-based compensation expense was
As of February 9, 2024, Prothena had approximately 53.7 million ordinary shares outstanding.
2024 Financial Guidance
The Company expects the full year 2024 net cash used in operating and investing activities to be
Conference Call Details
Prothena management will discuss these results and its 2024 financial guidance during a live audio conference call today, Thursday, February 15, 2024, at 4:30 PM 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 +1 (800) 715-9871 (
About Prothena
Prothena Corporation plc is a late-stage clinical biotechnology company with expertise in protein dysregulation and a pipeline of investigational therapeutics with the potential to change the course of devastating neurodegenerative and rare peripheral amyloid diseases. Fueled by its deep scientific expertise built over decades of research, Prothena is advancing a pipeline of therapeutic candidates for a number of indications and novel targets for which its ability to integrate scientific insights around neurological dysfunction and the biology of misfolded proteins can be leveraged. Prothena’s pipeline includes both wholly-owned and partnered programs being developed for the potential treatment of diseases including AL amyloidosis, ATTR amyloidosis, Alzheimer’s disease, Parkinson’s disease and a number of other neurodegenerative diseases. For more information, please visit the Company’s website at www.prothena.com and follow the Company on Twitter @ProthenaCorp.
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 Securities and Exchange Commission (SEC) on November 2, 2023, discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the SEC, and our Annual Report on Form 10-K to be filed with the SEC for our fiscal year 2023. We undertake no obligation to update publicly any forward-looking statements contained in this press release as a result of new information, future events, or changes in our expectations.
PROTHENA CORPORATION PLC CONSOLIDATED STATEMENTS OF OPERATIONS (unaudited - amounts in thousands except per share data) |
||||||||||||||||
|
|
Three Months Ended
|
|
Year Ended December 31, |
||||||||||||
|
|
|
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 |
|
PROTHENA CORPORATION PLC CONSOLIDATED BALANCE SHEETS (unaudited - amounts in thousands) |
|||||
|
December 31, |
||||
|
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
Mark Johnson, CFA, Vice President, Investor Relations
650-417-1974, mark.johnson@prothena.com
Media
Michael Bachner, Senior Director, Corporate Communications
609-664-7308, michael.bachner@prothena.com
Source: Prothena Corporation plc
FAQ
What was Prothena's net loss for the fourth quarter of 2023?
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