- Acquired exclusive license for oral, brain-penetrant, dual
TYK2/JAK1 inhibitor for immune-mediated brain disorders in
March 2023 covering global rights
(excluding China)
- Appointed Nick Kozauer, M.D. as SVP of Clinical Development and
Regulatory Strategy following his tenure as Director of the
Division of Neurology 2 in the Office of New Drugs of the U.S. Food
and Drug Administration
- Taldefgrobep alfa granted Fast Track Designation in SMA
- Driving strong and consistent progress across six robust drug
development platforms in 2023:
-
- in Kv7 activation, targeting Phase 2/3 study start in focal
epilepsy and bipolar disorder in the second half of 2023;
- Phase 1 study initiation planned with potentially
first-in-class TYK2/JAK1 brain-penetrant inhibitor in
immune-mediated brain disorders;
- three Phase 3 studies gaining momentum with regulatory
engagement and enrollment completion expected in programs spanning
glutamate modulation in SCA and OCD, respectively, and enrollment
ongoing in myostatin inhibition program in SMA;
- three IND submissions anticipated in 2023 spanning Kv7
activation, TRPM3 antagonism, and IgG degradation offer potential
to drive long-term value creation beyond 2023
NEW
HAVEN, Conn., May 12, 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 for people with debilitating neurological
and neuropsychiatric diseases, including ultra-rare disorders,
today reported financial results for the first quarter ended
March 31, 2023, and provided a review
of recent accomplishments and anticipated upcoming milestones.
Vlad Coric, M.D., Chairman and
Chief Executive Officer of Biohaven, commented, "We have set forth
ambitious goals for the balance of 2023, as we continue advancing
what we believe is one of the most innovative and exciting
neuroscience platform in development. We were energized by the
preliminary safety, tolerability and pharmacokinetic data we
reported from our SAD/MAD Phase 1 study of Kv7.2/Kv7.3 activator
BHV-7000 earlier in the quarter, supported by the preponderance of
preclinical efficacy data generated to date with BHV-7000
demonstrating potent anti-seizure efficacy, concentration-dependent
hyperpolarization of the resting membrane potential, meaningful
increases in action potential threshold, and encouragingly low
GABAA activity. We also look forward to unlocking Kv7
platform expansion opportunities across a spectrum of indications
with high unmet medical need."
Dr Coric continued, "With our glutamate platform, we look to
gain clarity on the regulatory path forward for SCA and expect to
complete enrollment in our Phase 3 OCD program in 2023, which we
believe are two critically important milestones given the dearth of
approved therapies for these distinct patient populations.
Regarding our myostatin platform, we continue to activate sites and
drive enrollment in our ongoing Phase 3 SMA study. With our
recently acquired TYK2/JAK1 platform, we remain focused on starting
Phase 1 studies in 2023 and exploring BHV-8000's potential to
address an array of immune-mediated brain disorders. And finally,
we look forward to submitting IND applications across three
separate programs, including BHV-7010, a next-gen Kv7 activator
targeting epilepsy and mood disorders, BHV-2011, a TRPM3 inhibitor
targeting chronic pain, and BHV-1300, our IgG degrader, following
the report of robust preclinical data earlier in the year. We
remain as committed as ever to driving data-driven, efficient
results for patients and the shareholders who support our continued
work, and look forward to executing and delivering continued value
in the year ahead."
First Quarter 2023 and Recent Business Highlights
- Acquired BHV-8000, a brain-penetrant inhibitor of TYK2/JAK1,
from Highlightll - As previously reported, in March 2023, the Company acquired exclusive rights
(excluding China) to a novel,
oral, first-in-class, brain-penetrant, dual inhibitor of TYK2/JAK1
offering wide therapeutic index with TYK2 inhibition and high
selectivity for JAK1 inhibition without the severely limiting
adverse class effects of JAK2/JAK3 inhibitors. The Company expects
to commence Phase 1 development in 2023, exploring its potential to
address immune-mediated brain disorders.
- Delivered oral and poster presentations demonstrating
preclinical efficacy and initial Phase 1 safety and tolerability of
BHV-7000 on ASENT 2023 virtual platform - In March 2023, the Company presented data at the
2023 American Society for Experimental Neurotherapeutics Annual
Meeting (ASENT 2023) demonstrating that activating Kv7.2/7.3 with
BHV-7000, a structurally and pharmacologically distinct compound
from ezogabine, produced concentration-dependent hyperpolarization
of the resting membrane potential, potentiated meaningful increases
in action potential threshold, exhibited significantly lower
GABAA activity than ezogabine, and delivered potent
anti-seizure efficacy in the maximal electroshock seizure (MES)
model, without negatively impacting neurobehavior. The Company also
presented data from the Phase 1 SAD/MAD clinical trial,
demonstrating BHV-7000 was well-tolerated at single doses up to 100
mg and multiple doses up to 40 mg per day for 15 days. Most adverse
events were mild and resolved spontaneously, and there were no
serious or severe adverse events or dose-limiting toxicities
reported. In BHV-7000 treated subjects in the MAD study,
CNS-related adverse events typically associated with other
anti-seizure medications were not reported.
- Taldefgrobep Alfa Granted Fast Track Designation and Orphan
Drug Designation - As previously reported, Taldefgrobep alfa,
an anti-myostatin adnectin in development for SMA, was granted Fast
Track designation and Orphan Drug designation by the U.S. Food and
Drug Administration (FDA) in February
2023, and December 2022,
respectively. Phase 3 studies are ongoing; the Company expects to
enroll approximately 180 patients in this randomized, double-blind,
placebo-controlled global trial.
- Reported preclinical data with extracellular target degrader
platform technology (MoDE™), a pan-IgG degrader - As previously
reported, in January 2023, the
Company evaluated the effect of a single dose of IgG degrader,
BHV-1300, in cynomolgus monkeys. The Company reported 75% reduction
of IgG levels from baseline and noted the observation occurred in
three days; the data in this pre-clinical study compares favorably
to standard of care therapy efgartigimod, where reduction of IgG
levels with efgartigimod was observed to be 50% and had taken 5-7
days. The Company expects BHV-1300 will be ready for IND submission
in the second half of 2023.
- Reported preclinical data with second MoDE in bispecific
platform targeting IgA Nephropathy - As previously reported, in
January 2023, the Company reported
preclinical data with a second MoDE targeting galactose deficient
IgA (Gd-IgA), which is believed to play a pathogenic role in IgA
Nephropathy. Specific removal of pathogenic Gd-IgA with
preservation of normal IgA potentially permits disease remission
without incurring an infection risk. The Company shared preliminary
data demonstrating the chimeric antibody-ASGPR ligand conjugate
specifically mediated endocytosis of Gd-IgA, as opposed to normal
IgA, in an endocytosis assay with HepG2 cells.
- Leadership team expanded with key appointment - In
April 2023, Biohaven announced the
appointment of Nick Kozauer, M.D. as SVP of Clinical Development
and Regulatory Strategy following his tenure as Director of the
Division of Neurology 2 in the Office of New Drugs of the FDA. Dr.
Kozauer had supervised and reviewed the approval of over 15
marketed drugs during his tenure at the FDA.
Upcoming Expected Milestones:
Biohaven is progressing its product candidates through clinical
programs in a number of common and rare disorders. The Company
expects to reach significant pipeline milestones in the coming
periods. Biohaven expects to:
- Initiate EEG study with BHV-7000 in the first half of
2023: Following Phase 1 study completion, Biohaven expects to
initiate pivotal trials in patients with epilepsy and patients with
bipolar disorder in the second half of 2023.
- Submit IND with BHV-7010 in epilepsy and mood disorders:
The Company expects to submit an IND with next-generation Kv7
activator BHV-7010 in epilepsy in the second half of 2023.
- Submit IND with BHV-2100 in chronic pain: The
Company expects to submit an IND with BHV-2100, a TRPM3 antagonist
in the Company's ion channel platform targeting a pain disorder in
the second half of 2023.
- Submit IND with BHV-1300: The Company expects to submit
an IND with pan-IgG degrader BHV-1300 in the second half of
2023.
- Commence Phase 1 studies with BHV-8000: The Company
expects to commence Phase 1 studies with BHV-8000, an oral,
brain-penetrant, dual TYK2/JAK1 inhibitor for immune-mediated brain
disorders in 2023.
- Complete enrollment in Phase 3 study of troriluzole in OCD
in 2023: Two Phase 3 randomized, double-blind,
placebo-controlled studies are expected to enroll up to 700
patients (in each trial) across nearly 200 global study sites. The
Company anticipates completing enrollment in year-end 2023.
- Provide an update on troriluzole in SCA: The Company
intends to interact with the FDA and/or European Medicines Agency
in the first half of 2023 on next steps.
- Continue advancing Phase 3 clinical studies of taldefgrobep
alfa in SMA: The Company expects to enroll approximately 180
patients in the study through patient enrollment in up to 60
clinical sites.
- Continue advancements across multiple neuroscience and
immunoscience indications: The Company's preclinical pipeline
includes molecular degraders of extracellular proteins, CD38
targeting antibody recruiting molecules (ARMs), TRP channels, and
other undisclosed targets, including those with disease-modifying
potential.
Capital Position:
Cash, cash equivalents and marketable securities as of
March 31, 2023 was $392.0 million, including $4.0 million of restricted cash, and excluding
$61.5 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.
First Quarter 2023 Financial Highlights:
Research and Development (R&D) Expenses: R&D
expenses, including non-cash share-based compensation costs, were
$63.5 million for the three months
ended March 31, 2023, compared to
$70.1 million for the three months
ended March 31, 2022. The decrease of
$6.6 million was primarily due to a
decrease of $14.7 million in
personnel-related costs including non-cash share-based compensation
costs, and a decrease in expenses for verdiperstat, BHV-2100 and
BHV-1200, partially offset by an increase in expenses for our
clinical programs for Kv7 (BHV-7000 and 7010), troriluzole and
BHV-2000. The decrease in personnel-related costs is due to
non-cash share-based compensation expense for the first quarter of
2022 being allocated from the Former Parent equity plan based on
equity awards with higher grant date fair values, which was
partially offset by increased personnel costs related to an
increase in headcount for our discovery operations. Non-cash
share-based compensation expense was $2.2
million for the three months ended March 31, 2023, a decrease of $22.3 million as compared to the same period in
2022.
General and Administrative (G&A) Expenses: G&A
expenses, including non-cash share-based compensation costs, were
$14.3 million for the three months
ended March 31, 2023, compared to
$19.7 million for the three months
ended March 31, 2022. The decrease of
$5.4 million was primarily due to
decreased non-cash share-based compensation costs. This was
partially offset by increased personnel costs in the first quarter
of 2023 compared to the same period in 2022, due to a majority of
the personnel costs in the first quarter of 2022 being allocated to
the Former Parent. Non-cash share-based compensation expense was
$1.5 million for the three months
ended March 31, 2023, a decrease of
$14.1 million as compared to the same
period in 2022. The decrease in non-cash share-based compensation
expense is due to the first quarter of 2022 expense being allocated
from the Former Parent equity plan based on equity awards with
higher grant date fair values.
Other Income (Expense), Net: Other income (expense),
net was a net income of $8.2 million
for the three months ended March 31,
2023, compared to net expense of $4.0
thousand for the three months ended March 31, 2022. The increase of $8.2 million was primarily due to an increase in
net investment income and an increase of $3.9 million in other income related to our
transition services provided to our Former Parent, which is largely
non-recurring.
Net Loss: Biohaven reported a net loss for the three
months ended March 31, 2023, of
$70.5 million, or $1.03 per share, compared to $97.0 million, or $2.46 per share, for the same period in 2022.
Non-GAAP adjusted net loss for the three months ended March 31, 2023 was $66.7
million, or $0.98 per share,
compared to $56.9 million, or
$1.45 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 for people with debilitating neurological
and neuropsychiatric diseases, including rare disorders. 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 with little 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
brain-penetrant TYK2/JAK1 inhibition for immune-mediated brain
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 extracellular target degrader 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
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 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 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
March 31,
|
|
|
2023
|
|
2022
|
Operating
expenses:
|
|
|
|
|
Research and
development
|
|
$
63,461
|
|
$
70,096
|
General and
administrative
|
|
14,321
|
|
19,677
|
Total operating
expenses
|
|
77,782
|
|
89,773
|
Loss from
operations
|
|
(77,782)
|
|
(89,773)
|
Other income
(expense):
|
|
|
|
|
Other income
(expense), net
|
|
8,229
|
|
(4)
|
Total other income
(expense), net
|
|
8,229
|
|
(4)
|
Loss before provision
for income taxes
|
|
(69,553)
|
|
(89,777)
|
Provision for income
taxes
|
|
939
|
|
7,255
|
Net loss
|
|
$
(70,492)
|
|
$
(97,032)
|
Net loss per share —
basic and diluted
|
|
$
(1.03)
|
|
$
(2.46)
|
Weighted average
common shares outstanding— basic and diluted
|
|
68,206,879
|
|
39,375,944
|
BIOHAVEN
LTD.
CONDENSED
CONSOLIDATED BALANCE SHEETS
(Amounts in
thousands)
|
|
|
|
March 31,
2023
|
|
December 31,
2022
|
|
|
(Unaudited)
|
|
|
Assets
|
|
|
|
|
Current
assets:
|
|
|
|
|
Cash and cash
equivalents
|
|
$
125,031
|
|
$
204,877
|
Marketable
securities
|
|
262,998
|
|
260,464
|
Prepaid
expenses
|
|
26,400
|
|
20,945
|
Income tax
receivable
|
|
43,830
|
|
46,139
|
Restricted cash held
on behalf of Former Parent
|
|
61,548
|
|
35,212
|
Other current
assets
|
|
24,669
|
|
19,331
|
Total current
assets
|
|
544,476
|
|
586,968
|
Property and
equipment, net
|
|
17,494
|
|
17,512
|
Intangible
assets
|
|
18,400
|
|
18,400
|
Goodwill
|
|
1,390
|
|
1,390
|
Other non-current
assets
|
|
36,761
|
|
37,513
|
Total assets
|
|
$
618,521
|
|
$
661,783
|
Liabilities and
Equity
|
|
|
|
|
Current
liabilities:
|
|
|
|
|
Accounts
payable
|
|
$
14,456
|
|
$
10,703
|
Due to Former
Parent
|
|
61,548
|
|
35,212
|
Accrued expenses and
other current liabilities
|
|
38,002
|
|
44,106
|
Total current
liabilities
|
|
114,006
|
|
90,021
|
Long-term operating
lease liability
|
|
29,760
|
|
30,581
|
Other non-current
liabilities
|
|
2,497
|
|
2,410
|
Total
liabilities
|
|
146,263
|
|
123,012
|
Shareholders'
Equity:
|
|
|
|
|
Preferred shares, no
par value; 10,000,000 shares authorized, no shares issued
and outstanding as of March 31, 2023 and December 31,
2022
|
|
—
|
|
—
|
Common shares, no par
value; 200,000,000 shares authorized as of March 31, 2023
and December 31, 2022; 68,212,479 and 68,190,479 shares issued and
outstanding
as of March 31, 2023 and December 31, 2022, respectively
|
|
616,246
|
|
615,742
|
Additional paid-in
capital
|
|
17,462
|
|
13,869
|
Accumulated
deficit
|
|
(161,616)
|
|
(91,124)
|
Accumulated other
comprehensive income
|
|
166
|
|
284
|
Total shareholders'
equity
|
|
472,258
|
|
538,771
|
Total liabilities and
shareholders' equity
|
|
$
618,521
|
|
$
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
March 31,
|
|
|
2023
|
|
2022
|
Reconciliation of
GAAP to Non-GAAP adjusted net loss:
|
|
|
|
|
GAAP net
loss
|
|
$ (70,492)
|
|
$ (97,032)
|
Add: non-cash
share-based compensation expense
|
|
3,765
|
|
40,120
|
Non-GAAP adjusted net
loss
|
|
$ (66,727)
|
|
$ (56,912)
|
|
|
|
|
|
Reconciliation of
GAAP to Non-GAAP adjusted net loss per share — basic and
diluted:
|
GAAP net loss per
share — basic and diluted
|
|
$
(1.03)
|
|
$
(2.46)
|
Add: non-cash
share-based compensation expense
|
|
0.05
|
|
1.02
|
Non-GAAP adjusted net
loss per share — basic and diluted
|
|
$
(0.98)
|
|
$
(1.45)
|
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.