- Cash, cash equivalents, and marketable securities totaled
$349.0 million as of June 30, 2023
- Announced positive interim data from BHV-7000 EEG biomarker
study demonstrating evidence of central nervous system (CNS) target
engagement, confirming previously reported preclinical and clinical
data, and highlighting the paradigm-changing potential of BHV-7000
in the treatment of epilepsy and mood disorders
- Brain penetrant TYK2/JAK1 inhibitor, BHV-8000, advanced into
Phase 1 with projected therapeutic concentrations achieved and
well-tolerated profile observed to date
-
- Biohaven anticipates initiating a Phase 2 clinical trial in
Parkinson's disease and potentially other neuroinflammatory
diseases in 2024
- Advanced targeted extracellular protein degradation platform
with potential to support numerous clinical candidates across a
broad range of high unmet need indications
-
- Investigational New Drug (IND) submission for pan-IgG degrader,
BHV-1300, on track for the second half of 2023 with potential to
treat multiple immune-mediated diseases
- IND submission with IgA degrader for IgA nephropathy expected
in the first half of 2024
- Orphan drug designation (ODD) granted by the European
Commission for taldefgrobep alfa a novel anti-myostatin
adnectin, for the treatment of spinal muscular atrophy (SMA)
-
- taldefgrobep previously received Fast-Track and ODD from the
FDA
- taldefgrobep Phase 3 SMA trial expected to complete enrollment
in the second half of 2023
- Type A meeting planned with FDA regarding troriluzole program
in Spinocerebellar Ataxia Type 3 (SCA3) and enrollment in Phase 3
obsessive compulsive disorder (OCD) trial expected to complete at
end of 2023
NEW
HAVEN, Conn., July 31,
2023 /PRNewswire/ -- Biohaven Ltd. (NYSE: BHVN)
(Biohaven or the Company), a global clinical-stage
biopharmaceutical company focused on the discovery, development and
commercialization of life-changing therapies to treat a broad range
of rare and common diseases, today reported financial results for
the second quarter ended June 30,
2023, and provided a review of recent accomplishments and
anticipated upcoming milestones.
Vlad Coric, M.D., Chairman and
Chief Executive Officer of Biohaven, commented, "In less than a
year since our spin-off, the Biohaven team has driven strong and
consistent progress across multiple drug development platforms. We
were excited to recently report positive interim data from the
ongoing EEG biomarker study of our lead Kv7 activator,
BHV-7000. As expected, BHV-7000 demonstrated CNS activity
consistent with what has been reported in EEG studies with other
Kv7 channel activators in development that are clinically effective
in treating epilepsy and with other approved anti-seizure
medications. Importantly, BHV-7000 demonstrates CNS target
engagement but is not associated with the typical CNS adverse
events associated with other ASMs, like somnolence and fatigue. We
look forward to completing the higher dose groups in this EEG study
and initiating Phase 2/3 studies with our once daily,
extended-release oral formulation later this year."
Dr. Coric continued, "We also shared a number of equally
exciting announcements across our broader pipeline this quarter. We
successfully administered the first three doses in the SAD portion
of our ongoing SAD/MAD study evaluating BHV-8000, an oral,
brain-penetrant, dual TYK2/JAK1 inhibitor. Potential
therapeutic concentrations of BHV-8000 were achieved and BHV-8000
was well tolerated with only mild adverse events reported,
reinforcing our plans to start a Phase 2 clinical trial with
BHV-8000 in Parkinson's disease and potentially other
neuroinflammatory diseases in 2024. In addition, we have advanced
IND enabling preclinical studies and remain on track to submit an
IND with our IgG degrader, BHV-1300, later this year. We also
anticipate submitting an IND with our IgA degrader in 2024 as we
continue to advance multiple clinical degrader candidates across a
wide range of indications. We also shared key updates for our
taldefgrobep alfa program (an anti-myostatin) with enrollment
completion in our pivotal study in SMA and a planned Phase 2 trial
initiation in obesity."
"Finally, I want to reaffirm Biohaven's commitment to the SCA3
patient community in spite of the recent disappointing regulatory
decision by the FDA not to review our submitted NDA. Given the
seriousness of SCA3 and consistent treatment benefits observed
across multiple prespecified outcome measures, including an 80%
delay in disease progression over the one-year study period with
substantial risk reduction in falls, we believe that full
consideration of all available data is warranted for this
ultra-rare disease. Biohaven has built its foundation around our
patient mission and we plan to continue to work with regulatory
agencies to try to advance troriluzole for individuals suffering
from SCA3."
Second Quarter 2023 and Recent Business Highlights
- Announced positive interim data from EEG biomarker study
with BHV-7000 - In July 2023, the
Company provided a preliminary data update from the ongoing
electroencephalogram (EEG) biomarker study for the Kv7 Channel
Activator platform. Preliminary Phase 1 data confirmed evidence of
target engagement in the central nervous system for subjects with
projected therapeutic concentrations of BHV-7000 (based on the EC50
from preclinical models), measured by changes from baseline in EEG
spectral power that occurred after dosing. These pharmacodynamic
(PD) effects were similar to those reported in the literature for
anti-seizure medicines (ASMs), including Kv7 activators in
development that are clinically effective in treating epilepsy.
BHV-7000's PD effects were also differentiated from those reported
for other Kv7 activators including, specifically, the absence of
increases in EEG spectral power in frequency bands associated with
drowsiness and somnolence. Results from the low-dose group validate
the preclinical hypothesis, confirm the Phase 1 SAD/MAD clinical
data, and provide strong support for Biohaven's plans to initiate
pivotal studies with BHV-7000 in focal epilepsy and bipolar
disorder in the second half of 2023. The preliminary data highlight
BHV-7000's differentiation and potentially favorable clinical
profile compared to other ASMs, and the Company expects to present
the complete EEG results by the end of the year. Additionally, new
pharmacokinetic data from multiple clinical formulations being
studied has now confirmed a once daily extended-release oral
formulation that will be used in the Phase 2/3 clinical
programs.
- Successfully dosed three cohorts with single ascending doses
of oral, brain penetrant, dual TYK2/JAK1 agent, BHV-8000 - In
July 2023, the Company announced that
it successfully dosed three cohorts in the SAD portion of an
ongoing SAD/MAD Phase 1 study evaluating brain penetrant TYK2/JAK1
agent, BHV-8000 in healthy volunteers. The ongoing Phase 1 study is
designed to evaluate the safety, tolerability, pharmacokinetics
(PK) and PD of single and multiple ascending doses of BHV-8000 in
healthy volunteers. Based on the preliminary data available,
projected therapeutic concentrations of BHV-8000 were achieved, and
BHV-8000 was well tolerated with only mild adverse events reported.
These data provide support for further development of BHV-8000, and
the Company anticipates beginning a Phase 2 clinical trial with
BHV-8000 in Parkinson's disease and potentially other
neuroinflammatory diseases in 2024. The Company had previously
reported initiation of dosing with BHV-8000 in the Phase 1 study at
Biohaven's R&D Day in May
2023.
- Reported on continued progress observed with Biohaven's
first-in-class bispecific IgG degrader, BHV-1300, as compound
advances to IND filing in 2H2023 - In July 2023, the Company shared an update on
BHV-1300's highly competitive safety, manufacturable and PD
profile. The Company is assembling a pipeline of partially
de-risked, follow-on IgG degraders as well as antigen-specific
degraders providing both optionality and a sustainable output of
drug candidates for several years. An IND application for BHV-1300
is on track for submission in 2023 and an IND application for
Biohaven's IgA degrader on track for submission in 2024
- Type A meeting planned to comprehensively address FDA's
concerns in connection with Troriluzole program in SCA3
- In July 2023, the Company
announced that the FDA informed Biohaven it would not review the
recently submitted New Drug Application (NDA) for troriluzole for
the treatment of spinocerebellar ataxia type 3 (SCA3), an
ultra-rare, genetically-defined, neurodegenerative disease
associated with progressive disability, frequent falls, loss of
ambulation, speech and swallowing impairment, and premature death
that is the most common SCA genotype worldwide. The FDA informed
Biohaven it would not review the application given that the study's
primary endpoint was not met and thus, would not permit a
substantive review. Biohaven is committed to working closely with
the FDA to bring troriluzole to people with SCA3 as quickly as
possible given no therapy is currently approved for this ultra-rare
genetic disorder. Biohaven plans to request a Type A meeting to
comprehensively address FDA's concerns cited in the refusal to file
letter.
- EMA orphan drug designation granted for taldefgrobep alfa
for the treatment of SMA: - In July
2023, the Company announced that taldefgrobep alfa received
orphan drug designation (ODD) from the European Commission for
taldefgrobep for the treatment of SMA. Taldefgrobep previously
received Fast-Track and ODD from the FDA. At Biohaven's R&D
Day, the Company announced plans to complete randomization of
approximately 180 patients in global SMA trial.
Upcoming Milestones:
Biohaven is progressing its product candidates through clinical
programs in a number of common and rare disorders. The Company
plans to reach significant pipeline milestones in the coming
periods. Biohaven expects to:
- Announce Phase 1 EEG study results by year-end 2023: The
Company expects to present complete results from its ongoing EEG
study with BHV-7000 in healthy volunteers by the end of the
year.
- Initiate Phase 2/3 studies with BHV-7000 in the second half
of 2023: Biohaven expects to initiate pivotal trials in
patients with focal epilepsy and bipolar disorder in the second
half of 2023.
- Submit IND with BHV-1300, the Company's lead extracellular
degrader: The Company expects to submit an IND with
pan-IgG degrader BHV-1300 in the second half of 2023 and expects to
initiate Phase 2 studies in 2024.
- Submit IND with selective Gd-IgA1 degrader: The
Company expects to submit an IND with a Gd-IgA1 degrader indicated
for IgA nephropathy in the first half of 2024.
- Initiate Phase 2 study with BHV-8000: The Company
commenced Phase 1 studies with BHV-8000, an oral, brain-penetrant,
dual TYK2/JAK1 inhibitor for neuroinflammatory disorders, in the
first half of 2023 and expects to initiate a Phase 2 study in
Parkinson's disease in 2024.
- Submit IND with BHV-2100 in chronic pain:
The Company expects to submit an IND with BHV-2100, a selective
TRPM3 antagonist in the Company's ion channel platform, in the
second half of 2023.
- Complete enrollment in Phase 3 study of troriluzole in OCD
in 2023: Two Phase 3 randomized, double-blind,
placebo-controlled studies of troriluzole in OCD are expected to
enroll up to 700 patients (in each trial) across nearly 200 global
study sites. The Company anticipates completing enrollment in at
least one Phase 3 trial by year-end 2023.
- Complete enrollment in Phase 3 clinical study of
taldefgrobep alfa in SMA: The Company expects to complete
enrollment in the study of taldefgrobep in SMA in the second half
of 2023.
- Continue advancements across multiple neuroscience and
immunoscience indications: The Company's preclinical pipeline
includes a platform of bispecific degraders of extracellular
proteins directed against IgG, IgA and other targets, TRPM3 and Kv7
family of ion channels, and other undisclosed targets, including
those with disease-modifying potential.
Capital Position:
Cash, cash equivalents and marketable securities as of
June 30, 2023 was $349.0 million, including $13.9 million of restricted cash, and excluding
$40.4 million of cash payable
to Biohaven Pharmaceutical Holding Company Ltd. (the Former
Parent), compared to $467.9 million,
including $2.5 million of restricted
cash, and excluding $35.2 million of
cash payable to the Former Parent, as of December 31, 20221.
Second Quarter 2023 Financial Highlights:
Research and Development (R&D) Expenses: R&D
expenses, including non-cash share-based compensation costs, were
$79.5 million for the three months
ended June 30, 2023, compared to
$177.1 million for the three months
ended June 30, 2022. Non-cash
share-based compensation expense was $2.5
million for the three months ended June 30, 2023, a decrease of $10.3 million as compared to the same period in
2022. Non-cash share-based compensation expense was higher in the
second quarter of 2022 primarily because expense allocated from the
Former Parent equity plan, prior to the spin-off, was based on
equity awards with higher grant date fair values.
General and Administrative (G&A) Expenses: General
and administrative expenses were $14.5
million for the three months ended June 30, 2023, compared to $20.0 million for the three months ended
June 30, 2022. The decrease of
$5.5 million was primarily due to
decreased non-cash share-based compensation costs. Non-cash
share-based compensation expense was $2.2
million for the three months ended June 30, 2023, a decrease of $5.8 million as compared to the same period in
2022. Non-cash share-based compensation expense was higher in the
second quarter of 2022 primarily because expense allocated from the
Former Parent equity plan, prior to the spin-off, was based on
equity awards with higher grant date fair values.
Other Income (Expense), Net: Other income (expense), net
was a net income of $5.8 million for
the three months ended June 30, 2023,
compared to net expense of $0.1
million for the three months ended June 30, 2022. The increase of $5.9 million was primarily due to an increase in
net investment income and an increase of $1.7 million in other income related to our
transition services provided to the Former Parent, which is largely
non-recurring.
Net Loss: Biohaven reported a net loss for the three
months ended June 30, 2023, of
$90.3 million, or $1.32 per share, compared to $203.3 million, or $5.16 per share, for the same period in 2022.
Non-GAAP adjusted net loss for the three months ended June 30, 2023 was $85.7
million, or $1.25 per share,
compared to $182.5 million, or
$4.63 per share for the same period
in 2022. These non-GAAP adjusted net loss and non-GAAP adjusted net
loss per share measures, more fully described below under "Non-GAAP
Financial Measures," exclude non-cash share-based compensation
charges. A reconciliation of the GAAP financial results to non-GAAP
financial results is included in the tables below. For periods
prior to Biohaven's spin-off from the Former Parent on October 3, 2022 (the "Spin-Off"), net loss per
share and non-GAAP adjusted net loss per share were calculated
based on the 39,375,944 common shares of Biohaven distributed to
the Former Parent shareholders at the time of the distribution,
including common shares issued in connection with the Former Parent
share options that were settled on October
3, 2022 and common shares issued in connection with the
Former Parent restricted share units that vested on October 3, 2022. The same number of shares
is being utilized for the calculation of basic and diluted earnings
per share for all periods presented prior to the Spin-Off.
Non-GAAP Financial Measures
This press release
includes financial results prepared in accordance with accounting
principles generally accepted in the
United States (GAAP), and also certain non-GAAP financial
measures. In particular, Biohaven has provided non-GAAP adjusted
net loss and adjusted net loss per share, which are adjusted to
exclude non-cash share-based compensation, which is substantially
dependent on changes in the market price of common shares. Non-GAAP
financial measures are not an alternative for financial measures
prepared in accordance with GAAP. However, Biohaven believes the
presentation of non-GAAP adjusted net loss and adjusted net loss
per share, when viewed in conjunction with GAAP results, provides
investors with a more meaningful understanding of ongoing operating
performance and can assist investors in comparing Biohaven's
performance between periods.
In addition, these non-GAAP financial measures are among those
indicators Biohaven uses as a basis for evaluating performance, and
planning and forecasting future periods. These non-GAAP financial
measures are not intended to be considered in isolation or as a
substitute for GAAP financial measures. A reconciliation between
these non-GAAP measures and the most directly comparable GAAP
measures is provided later in this news release.
About Biohaven
Biohaven is a global clinical-stage
biopharmaceutical company focused on the discovery, development and
commercialization of life-changing therapies to treat a broad range
of rare and common diseases. Biohaven's experienced management team
brings with it a track record of delivering new drug approvals for
products for diseases such as migraine, depression, bipolar and
schizophrenia. The company is advancing a pipeline of therapies for
diseases, many of which have limited or no treatment options,
leveraging its proven drug development capabilities and proprietary
platforms, including Kv7 ion channel modulation for epilepsy and
neuronal hyperexcitability, glutamate modulation for
obsessive-compulsive disorder and spinocerebellar ataxia, myostatin
inhibition for neuromuscular diseases and metabolic disorders, and
brain-penetrant TYK2/JAK1 inhibition for neuroinflammatory
disorders. Biohaven's portfolio of early- and late-stage product
candidates also includes discovery research programs focused on
TRPM3 channel activation for neuropathic pain, CD-38 antibody
recruiting, bispecific molecules for multiple myeloma, antibody
drug conjugates (ADCs), and targeted extracellular protein
degradation platform technology (MoDE™) with potential application
in neurological disorders, cancer, and autoimmune diseases.
Forward-looking Statements
This news release includes
forward-looking statements within the meaning of the Private
Securities Litigation Reform Act of 1995. The use of certain words,
including "continue", "plan", "will", "believe", "may", "expect",
"anticipate" and similar expressions, is intended to identify
forward-looking statements. Investors are cautioned that any
forward-looking statements, including statements regarding the
future development, timing and potential marketing approval and
commercialization of development candidates, are not guarantees of
future performance or results and involve substantial risks and
uncertainties. Actual results, developments and events may differ
materially from those in the forward-looking statements as a result
of various factors including: the expected timing, commencement and
outcomes of Biohaven's planned and ongoing clinical trials; the
timing of planned interactions and filings with the FDA; the timing
and outcome of expected regulatory filings; complying with
applicable U.S. regulatory requirements; the potential
commercialization of Biohaven's product candidates; the potential
for Biohaven's product candidates to be first in class therapies;
and the effectiveness and safety of Biohaven's product candidates.
Additional important factors to be considered in connection with
forward-looking statements are described in Biohaven's filings with
the Securities and Exchange Commission, including within the
sections titled "Risk Factors" and "Management's Discussion and
Analysis of Financial Condition and Results of Operations". The
forward-looking statements are made as of the date of this new
release, and Biohaven does not undertake any obligation to update
any forward-looking statements, whether as a result of new
information, future events or otherwise, except as required by
law.
|
|
|
|
|
1 On May 9,
2022, the Board of Directors of the Former Parent approved and
directed the Former Parent's management to effect the spin-off of
the Kv7 ion channel activators, glutamate modulation
and myostatin inhibition platforms, preclinical product
candidates, and certain corporate infrastructure then owned by the
Former Parent in connection with the sale of the Former Parent to
Pfizer. On October 3, 2022, the purchase of the Former Parent by
Pfizer was consummated and the Former Parent completed the
distribution to holders of its common shares of all of the
outstanding common shares of Biohaven Ltd.
|
BIOHAVEN LTD.
|
CONDENSED
CONSOLIDATED STATEMENTS OF OPERATIONS
|
(Amounts in
thousands, except share and per share amounts)
|
(Unaudited)
|
|
|
|
Three Months Ended
June 30,
|
|
Six Months Ended
June 30,
|
|
|
2023
|
|
2022
|
|
2023
|
|
2022
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
Research and
development
|
|
$
79,490
|
|
$
177,087
|
|
$
142,951
|
|
$
247,183
|
General and
administrative
|
|
14,521
|
|
20,023
|
|
28,842
|
|
39,700
|
Total operating
expenses
|
|
94,011
|
|
197,110
|
|
171,793
|
|
286,883
|
Loss from
operations
|
|
(94,011)
|
|
(197,110)
|
|
(171,793)
|
|
(286,883)
|
Other income
(expense):
|
|
|
|
|
|
|
|
|
Other income
(expense), net
|
|
5,842
|
|
(67)
|
|
14,071
|
|
(71)
|
Total other income
(expense), net
|
|
5,842
|
|
(67)
|
|
14,071
|
|
(71)
|
Loss before provision
for income taxes
|
|
(88,169)
|
|
(197,177)
|
|
(157,722)
|
|
(286,954)
|
Provision for income
taxes
|
|
2,177
|
|
6,110
|
|
3,116
|
|
13,365
|
Net loss
|
|
$
(90,346)
|
|
$ (203,287)
|
|
$ (160,838)
|
|
$ (300,319)
|
Net loss per share —
basic and diluted
|
|
$
(1.32)
|
|
$
(5.16)
|
|
$
(2.36)
|
|
$
(7.63)
|
Weighted average
common shares outstanding— basic and diluted
|
|
68,248,023
|
|
39,375,944
|
|
68,227,564
|
|
39,375,944
|
BIOHAVEN LTD.
|
CONDENSED
CONSOLIDATED BALANCE SHEETS
|
(Amounts in
thousands)
|
|
|
|
June 30,
2023
|
|
December 31,
2022
|
|
|
(Unaudited)
|
|
|
Assets
|
|
|
|
|
Current
assets:
|
|
|
|
|
Cash and cash
equivalents
|
|
$
147,612
|
|
$
204,877
|
Marketable
securities
|
|
187,503
|
|
260,464
|
Prepaid
expenses
|
|
27,654
|
|
20,945
|
Income tax
receivable
|
|
8,656
|
|
46,139
|
Restricted cash held
on behalf of Former Parent
|
|
40,415
|
|
35,212
|
Other current
assets
|
|
22,278
|
|
19,331
|
Total current
assets
|
|
434,118
|
|
586,968
|
Property and
equipment, net
|
|
17,277
|
|
17,512
|
Intangible
assets
|
|
18,400
|
|
18,400
|
Goodwill
|
|
1,390
|
|
1,390
|
Other non-current
assets
|
|
35,551
|
|
37,513
|
Total assets
|
|
$
506,736
|
|
$
661,783
|
Liabilities and
Equity
|
|
|
|
|
Current
liabilities:
|
|
|
|
|
Accounts
payable
|
|
$
12,039
|
|
$
10,703
|
Due to Former
Parent
|
|
40,415
|
|
35,212
|
Accrued expenses and
other current liabilities
|
|
35,393
|
|
44,106
|
Total current
liabilities
|
|
87,847
|
|
90,021
|
Long-term operating
lease liability
|
|
29,115
|
|
30,581
|
Other non-current
liabilities
|
|
2,519
|
|
2,410
|
Total
liabilities
|
|
119,481
|
|
123,012
|
Shareholders'
Equity:
|
|
|
|
|
Preferred shares, no
par value; 10,000,000 shares authorized, no shares issued and
outstanding as of June 30, 2023 and December 31, 2022
|
|
—
|
|
—
|
Common shares, no par
value; 200,000,000 shares authorized as of June 30, 2023 and
December 31, 2022; 68,316,953 and 68,190,479 shares issued and
outstanding as of June 30, 2023 and December 31, 2022,
respectively
|
|
617,510
|
|
615,742
|
Additional paid-in
capital
|
|
21,687
|
|
13,869
|
Accumulated
deficit
|
|
(251,962)
|
|
(91,124)
|
Accumulated other
comprehensive income
|
|
20
|
|
284
|
Total shareholders'
equity
|
|
387,255
|
|
538,771
|
Total liabilities and
shareholders' equity
|
|
$
506,736
|
|
$
661,783
|
BIOHAVEN LTD.
|
RECONCILIATION OF
GAAP TO NON-GAAP FINANCIAL MEASURES
|
(Amounts in
thousands, except share and per share amounts)
|
(Unaudited)
|
|
|
|
Three Months Ended
June 30,
|
|
Six Months Ended
June 30,
|
|
|
2023
|
|
2022
|
|
2023
|
|
2022
|
Reconciliation of
GAAP to Non-GAAP adjusted net loss:
|
|
|
|
|
|
|
|
|
GAAP net
loss
|
|
$ (90,346)
|
|
$
(203,287)
|
|
$
(160,838)
|
|
$
(300,319)
|
Add: non-cash
share-based compensation expense
|
|
4,695
|
|
20,810
|
|
8,460
|
|
60,930
|
Non-GAAP adjusted net
loss
|
|
$ (85,651)
|
|
$
(182,477)
|
|
$
(152,378)
|
|
$
(239,389)
|
|
|
|
|
|
|
|
|
|
Reconciliation of
GAAP to Non-GAAP adjusted net loss per share — basic and
diluted:
|
|
|
|
|
GAAP net loss per
share — basic and diluted
|
|
$
(1.32)
|
|
$
(5.16)
|
|
$
(2.36)
|
|
$
(7.63)
|
Add: non-cash
share-based compensation expense
|
|
0.07
|
|
0.53
|
|
0.12
|
|
1.55
|
Non-GAAP adjusted net
loss per share — basic and diluted
|
|
$
(1.25)
|
|
$
(4.63)
|
|
$
(2.23)
|
|
$
(6.08)
|
MoDEs is a trademark of Biohaven Therapeutics Ltd.
Investor Contact:
Jennifer Porcelli
Vice President, Investor Relations
jennifer.porcelli@biohavenpharma.com
+1 (201) 248-0741
Media Contact:
Mike Beyer
Sam Brown Inc.
mikebeyer@sambrown.com
+1 (312) 961-2502
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SOURCE Biohaven Ltd.