- Net cash used in operating and investing activities was $45.7
million in the second quarter and $93.2 million for the first six
months of 2023; quarter-end cash and restricted cash position was
$661.3 million
- Revised year-end cash guidance to be approximately $600 million
in cash, cash equivalents and restricted cash, representing an
increase of $88 million from prior guidance of $512 million
- Presented research across multiple Alzheimer’s disease programs
targeting amyloid-beta and tau at AAIC 2023, including data on
PRX012, PRX005 and PRX123
- Bristol Myers Squibb (BMS) obtained the $55 million exclusive
worldwide license to PRX005 in July, expanding on the exclusive
U.S. license from July 2021
- Published Phase 3 VITAL clinical trial results in Blood, the
peer-reviewed journal of ASH; data showed a significant survival
benefit for birtamimab in patients with Mayo Stage IV AL
amyloidosis, as well as significant improvements across key
secondary endpoints
- Billy Dunn, M.D., founding and former Director of the Office of
Neuroscience, CDER, at the FDA, joined Prothena’s Board of
Directors
Prothena Corporation plc (NASDAQ:PRTA), a late-stage clinical
biotechnology company with a robust pipeline of investigational
therapeutics built on protein dysregulation expertise, today
reported financial results for the second quarter and first six
months of 2023 and provided business highlights.
“We continue to make significant progress across our pipeline,
further enabling Prothena to deliver transformative medicines for
people living with devastating diseases caused by protein
dysregulation. From our Alzheimer’s disease portfolio, we presented
at the AD/PD and AAIC 2023 conferences new data that showcases the
differentiating profiles of PRX012, PRX005 and PRX123. From our
peripheral diseases pipeline, we published peer-reviewed results
from the Phase 3 VITAL amyloidosis trial that highlights evidence
of the important role that birtamimab may have in improving
outcomes for patients with Mayo Stage IV AL amyloidosis. Our
collaborations also continue to advance and add value as Bristol
Myers Squibb recently exercised their option to acquire exclusive
worldwide rights to PRX005, Roche completed enrollment for the
Phase 2b PADOVA study evaluating prasinezumab in Parkinson’s
disease, and Novo Nordisk continues to enroll patients in their
Phase 2 study evaluating NNC6019 in ATTR amyloidosis,” said Gene
Kinney, Ph.D., President and Chief Executive Officer, Prothena.
“Looking ahead to the rest of the year, we are excited to file our
IND for PRX123, a dual amyloid beta/tau vaccine, and to provide
initial topline results from our ongoing Phase 1 SAD and MAD
clinical trials of PRX012, an anti-amyloid beta antibody.
“We also expanded our Board of Directors during the quarter with
the addition of Dr. Billy Dunn, a world-renowned neuroscience
leader whose dedicated career as a public servant at the FDA
directly led to the approval of multiple innovative new products
that address unmet medical needs for patients. Dr. Dunn’s expertise
and passion for patient-directed scientific advancements will help
guide key strategic decisions for our pipeline of potentially
best-in-class therapies to address the significant unmet needs for
neurodegenerative and rare peripheral amyloid diseases,” added
Kinney.
Second Quarter, Recent Business Highlights and Upcoming
Milestones
Neurodegenerative Diseases
Portfolio
Alzheimer’s Disease (AD)
PRX012, a wholly-owned potential best-in-class,
next-generation subcutaneous antibody for the treatment of AD,
targets a key epitope at the N-terminus of amyloid beta (Aβ) with
high binding potency. The U.S. Food and Drug Administration (FDA)
has granted Fast Track Designation for PRX012 for the treatment of
AD.
- Encore poster presentation at AAIC 2023 of two preclinical
studies showing superior binding characteristics of PRX012,
demonstrating a 20-fold higher affinity to Aβ soluble protofibrils
when compared to lecanemab and cleared pyroglutamate-modified Aβ at
lower concentrations when compared to donanemab
- Phase 1 single ascending dose (SAD) and multiple ascending dose
(MAD) clinical trials are ongoing; initial topline data expected by
year end 2023
PRX005, a potential best-in-class antibody for the
treatment of AD, specifically targets a key epitope within the
microtubule binding region (MTBR) of tau, a protein implicated in
diseases including AD, frontotemporal dementia (FTD), progressive
supranuclear palsy (PSP), chronic traumatic encephalopathy (CTE),
and other tauopathies. PRX005 is part of a Global Neuroscience
Research and Development Collaboration with Bristol Myers
Squibb.
- Bristol Myers Squibb obtained the $55 million exclusive
worldwide rights for PRX005 in July under the Global Neuroscience
Research and Development Collaboration, expanding on the $80
million exclusive U.S. license from July 2021; Bristol Myers Squibb
will be responsible for future development, manufacturing, and
commercialization of PRX005
- Phase 1 clinical trial SAD results presented in a poster
presentation at AAIC 2023 showed 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; as planned, cerebral
spinal fluid (CSF) drug levels were measured in the high
single-dose cohort and reached sufficient CSF concentrations to
predict pharmacological targeting of MTBR tau in the central
nervous system (CNS) (day 29 CSF:plasma ratio=0.2%)
- Phase 1 clinical trial MAD portion ongoing; going forward, all
program updates, including results from ongoing and any future
PRX005 clinical studies, will be reported by Bristol Myers
Squibb
PRX123, a wholly-owned potential first-in-class dual
Aβ/tau vaccine designed for the treatment and prevention of AD, 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.
- Preclinical results presented in a late breaker poster
presentation at AAIC 2023; results showed that a PRX123 vaccine
surrogate elicited robust antibody responses that bound with high
avidity to Aβ plaques in AD brain ex vivo and significantly reduced
Aβ brain plaques representing the first time that a dual target
vaccine for Alzheimer’s disease has been shown to reduce pathology
in a transgenic mouse model of AD pathology
- Investigational new drug (IND) application filing expected by
year end 2023
Parkinson’s Disease (PD)
Prasinezumab, a potential first-in-class antibody for the
treatment of PD, is designed to target a key epitope 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 PD (NCT04777331); topline results
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, is
designed to directly neutralize soluble toxic 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.
- Phase 3 VITAL clinical trial data published in Blood, the
peer-reviewed journal of American Society of Hematology (ASH),
demonstrated that in a post hoc analysis of patients with Mayo
Stage IV AL amyloidosis, a statistically significant survival
benefit of 74 percent was observed for those treated with
birtamimab plus standard of care (SOC) versus 49 percent in
patients on placebo plus SOC at 9 months (HR 0.413, p=0.021)
- Confirmatory Phase 3 AFFIRM-AL clinical trial in patients with
Mayo Stage IV AL amyloidosis, under a Special Protocol Assessment
(SPA) with the FDA with a primary endpoint of all-cause mortality
at a significance level of 0.10, is ongoing (NCT04973137); topline
results expected in 2024
ATTR Amyloidosis
NNC6019 (formerly PRX004), a potential first-in-class
amyloid depleter antibody for the treatment of ATTR cardiomyopathy,
is 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 $1.2 billion acquisition of Prothena’s ATTR
amyloidosis business and pipeline.
- Phase 2 study in patients with ATTR cardiomyopathy is being
conducted by Novo Nordisk (NCT05442047); topline results expected
in 2024
Second Quarter and First Six Months of 2023 Financial
Results
For the second quarter and first six months of 2023, Prothena
reported net loss of $54.6 million and $101.5 million, as compared
to a net loss of $41.2 million and $77.5 million for the second
quarter and first six months of 2022. Net loss per share for the
second quarter and first six months of 2023 was $1.03 and $1.92,
respectively, as compared to net loss per share of $0.88 and $1.66
for the second quarter and first six months of 2022,
respectively.
Prothena reported total revenue of $4.0 million and $6.2 million
for the second quarter and first six months of 2023, respectively,
as compared to total revenue of $1.3 million and $2.5 million for
the second quarter and first six months of 2022, primarily from
collaboration revenue from Bristol Myers Squibb.
Research and development (R&D) expenses totaled $56.0
million and $100.8 million for the second quarter and first six
months of 2023, as compared to $31.6 million and $58.8 million for
the second quarter and first six months of 2022, respectively. The
increase in R&D expense for the second quarter and first six
months of 2023 compared to the same periods in the prior year was
primarily due to higher clinical trial expenses, higher personnel
related expenses, higher consulting and manufacturing expenses.
R&D expenses included non-cash share-based compensation expense
of $4.9 million and $9.2 million for the second quarter and first
six months of 2023, as compared to $3.8 million and $7.1 million
for the second quarter and first six months of 2022,
respectively.
General and administrative (G&A) expenses totaled $14.5
million and $28.3 million for the second quarter and first six
months of 2023, as compared to $13.0 million and $24.8 million for
the second quarter and first six months of 2022, respectively. The
increase in G&A expenses for the second quarter and first six
months of 2023 compared to the same periods in the prior year was
primarily related to higher personnel related expenses. G&A
expenses included non-cash share-based compensation expense of $5.2
million and $9.7 million for the second quarter and first six
months of 2023, as compared to $4.5 million and $8.8 million for
the second quarter and first six months of 2022, respectively.
Total non-cash share-based compensation expense was $10.1
million and $18.9 million for the second quarter and first six
months of 2023, as compared to $8.3 million and $15.9 million for
the second quarter and first six months of 2022.
As of June 30, 2023, Prothena had $661.3 million in cash, cash
equivalents and restricted cash, and no debt.
As of July 28, 2023, Prothena had approximately 53.5 million
ordinary shares outstanding.
2023 Financial Guidance
The Company is updating its projected full year 2023 net cash
burn from operating and investing activities, and expects it to be
$148 to $161 million (versus prior guidance of $213 to $229
million), and expects to end the year with approximately $600
million (midpoint) in cash, cash equivalents and restricted cash,
representing an increase of $88 million from prior guidance of $512
million (midpoint). This increase in cash position is primarily
driven by Bristol Myers Squibb obtaining the $55 million exclusive
worldwide rights for PRX005, and to a lesser extent financing
proceeds received in the first half of 2023 and higher interest
income. The updated estimated full year 2023 net cash burn from
operating and investing activities is primarily driven by an
updated estimated net loss of $153 to $171 million (versus prior
guidance of $250 to $275 million), which includes an estimated $42
million of non-cash share-based compensation expense.
About the Global Neuroscience Research and Development
Collaboration with Bristol Myers Squibb
This global neuroscience research and development collaboration
is focused on three proteins implicated in the pathogenesis of
several neurodegenerative diseases, including tau, TDP-43 and an
undisclosed target. PRX005 is designed to be a best-in-class
anti-tau, MTBR-specific antibody for the potential treatment of
Alzheimer’s disease and is the first program to advance to the
clinic from this collaboration. Prothena is eligible to receive up
to an additional $160 million for U.S. rights, up to $110 million
for global rights, and up to $1.7 billion for regulatory and
commercial milestone payments for a total of up to $2.2 billion,
which also includes amounts received to date, plus potential tiered
commercial sales royalties across multiple programs.
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; the
continued advancement of our discovery, preclinical, and clinical
pipeline, and expected milestones in 2023, 2024, and beyond; the
treatment potential, designs, proposed mechanisms of action, and
potential administration of birtamimab, prasinezumab,
NNC6019/PRX004, PRX005, PRX012, and PRX123; plans for ongoing and
future clinical studies of birtamimab, prasinezumab,
NNC6019/PRX004, PRX005, PRX012, and PRX123 (including the filing of
an IND application); the expected timing of reporting data from
clinical studies of birtamimab, prasinezumab, PRX005, and PRX012;
amounts we might receive under our collaboration with BMS and Novo
Nordisk; our anticipated net cash burn from operating and investing
activities for 2023 and expected cash balance at the end of 2023;
and our estimated net loss and non-cash share-based compensation
expense for 2023. 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
Securities and Exchange Commission (SEC) on August 3, 2023, and
discussions of potential risks, uncertainties, and other important
factors in our subsequent filings with the SEC. 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
CONDENSED CONSOLIDATED STATEMENTS OF
OPERATIONS
(unaudited - amounts in thousands
except per share data)
Three Months Ended
June 30,
Six Months Ended
June 30,
2023
2022
2023
2022
Collaboration revenue
$
4,019
$
1,312
$
6,138
$
2,415
Revenue from license and intellectual
property
—
—
50
50
Total revenue
4,019
1,312
6,188
2,465
Operating expenses:
Research and development
56,011
31,569
100,767
58,831
General and administrative
14,512
12,952
28,250
24,787
Total operating expenses
70,523
44,521
129,017
83,618
Loss from operations
(66,504
)
(43,209
)
(122,829
)
(81,153
)
Other income, net
7,603
637
14,152
620
Loss before income taxes
(58,901
)
(42,572
)
(108,677
)
(80,533
)
Benefit from income taxes
(4,306
)
(1,328
)
(7,218
)
(2,999
)
Net loss
$
(54,595
)
$
(41,244
)
$
(101,459
)
$
(77,534
)
Basic net loss per ordinary share
$
(1.03
)
$
(0.88
)
$
(1.92
)
$
(1.66
)
Diluted net loss per ordinary share
$
(1.03
)
$
(0.88
)
$
(1.92
)
$
(1.66
)
Shares used to compute basic net loss per
share
53,121
46,805
52,812
46,755
Shares used to compute diluted net loss
per share
53,121
46,805
52,812
46,755
PROTHENA CORPORATION PLC
CONDENSED CONSOLIDATED BALANCE
SHEETS
(unaudited - amounts in
thousands)
June 30,
December 31,
2023
2022
Assets
Cash and cash equivalents
$
659,111
$
710,406
Restricted cash, current
1,352
—
Prepaid expenses and other current
assets
16,640
8,692
Total current assets
677,103
719,098
Property and equipment, net
2,170
1,731
Operating lease right-of-use assets
3,307
6,277
Restricted cash, non-current
860
2,212
Other non-current assets
37,209
28,717
Total non-current assets
43,546
38,937
Total assets
$
720,649
$
758,035
Liabilities and Shareholders’
Equity
Accrued research and development
18,927
10,794
Deferred revenue, current
25,123
11,442
Lease liability, current
3,448
6,473
Other current liabilities
24,067
21,438
Total current liabilities
71,565
50,147
Deferred revenue, non-current
67,405
85,293
Other non-current liabilities
—
553
Total non-current liabilities
67,405
85,846
Total liabilities
138,970
135,993
Total shareholders’ equity
581,679
622,042
Total liabilities and shareholders’
equity
$
720,649
$
758,035
View source
version on businesswire.com: https://www.businesswire.com/news/home/20230803583843/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
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