NEW HAVEN, Conn., May 7, 2019 /PRNewswire/ -- Biohaven
Pharmaceutical Holding Company Ltd. (NYSE: BHVN), a biotechnology
company focused on advancing innovative therapies for neurological
and neuropsychiatric diseases, reported financial results and
business updates for the quarter ended March
31, 2019.
Vlad Coric, M.D., CEO of Biohaven
commented, "In the first quarter of 2019, we took key strategic
steps to accelerate the regulatory review and advance the
commercialization of rimegepant Zydis® Orally Dissolving Tablet
(ODT). Our efforts to secure a priority review voucher will enable
an expedited 6-month review of our NDA. Additionally, we made
important progress in our broader NOJECTION™ migraine platform
including: commenced enrollment in our Phase 2/3 clinical trial of
BHV-3500, the first CGRP receptor antagonist designed to be
administered in an intranasal formulation, for the acute treatment
of migraine, continued enrollment in our Phase 3 rimegepant
migraine preventive treatment clinical trial and the acceptance of
our rimegepant IND in China
through our wholly-owned subsidiary BioShin."
"Beyond migraine, we were pleased to continue enrollment in four
Phase 3 clinical trials evaluating troriluzole in Alzheimer's
Disease, Generalized Anxiety Disorder, Obsessive Compulsive Disorder and
Spinocerebellar Ataxia. We also completed important preclinical
workstreams to further enable the clinical development of BHV-5000,
our oral, low-trapping NMDA antagonist. In our MPO platform, we
announced receipt of FDA orphan drug designation for verdiperstat
for the treatment of multiple system atrophy. Our world-class team
of clinical and commercial professionals remain dedicated and
focused on advancing our drug candidates to patients as efficiently
as possible."
First Quarter and Recent Business Highlights:
- Late-Breaking Oral Presentation Accepted at American Academy
of Neurology (AAN) 2019 Annual Meeting – In May 2019, Biohaven announced that results from
the pivotal rimegepant Zydis® ODT Phase 3 clinical trial will be
presented as a late-breaking oral presentation at the AAN 2019
Annual Meeting in Philadelphia,
May 7, 2019. This presentation is 1
of only 11 abstracts accepted as part of the Emerging Science
(Late-Breaking) program, and the only CGRP-targeting migraine data
accepted for the Late-Breaking session. Rimegepant is an oral,
single-dose, selective and potent small molecule calcitonin
gene-related peptide (CGRP) receptor antagonist in development for
the acute and preventive treatment of migraine.
- Appointed William "BJ" Jones as Chief Commercial Officer, Migraine
and Common Diseases – In April
2019, Biohaven announced the appointment of BJ Jones as
Chief Commercial Officer, Migraine and Common Diseases. Mr.
Jones will oversee the commercial
development of the Company's CGRP receptor antagonist platform
portfolio and glutamate portfolio in Alzheimer's Disease (AD),
obsessive compulsive disorder (OCD), and generalized anxiety
disorder (GAD).
Mr. Jones is a seasoned
pharmaceutical executive with two decades of commercial and
neuroscience expertise in large pharmaceutical companies and small
biotech firms. His commercial experience includes mass market
product launches for notable brands like Excedrin Migraine®,
Farxiga®, Pradaxa®, BiDil®, and Abilify®. He joins Biohaven
from Takeda Pharmaceuticals, Inc., where he served as Vice
President, Sales and Commercial Operations. In the ten years prior
to joining Takeda, he held leadership roles in marketing,
operations and business development at AstraZeneca, Bristol-Myers
Squibb, Boehringer-Ingelheim and NitroMed. He has led large
diverse teams and has significant neuroscience (schizophrenia,
bipolar, depression) and migraine experience in large primary care
markets.
Mr. Jones is a graduate of the
U.S. Air Force Academy and attained the
rank of Major through his active duty to reserve service. He has a
Master of Business Administration from Stanford Graduate School of
Business and a Master of Science degree in Industrial Engineering
from Texas A&M University.
- Commenced Enrollment in Phase 2/3 Clinical Trial of BHV-3500
for the Acute Treatment of Migraine – In
April 2019, the Company announced the
commencement of enrollment in a Phase 2/3 clinical trial assessing
the efficacy of BHV-3500, the first CGRP receptor antagonist drug
candidate to be administered in an intranasal formulation and
Biohaven's second drug candidate from its NOJECTION™ migraine
platform. The trial will randomize approximately 1,600 patients
across four treatment arms, 5, 10, and 20 mg of BHV-3500 versus
placebo, and topline results are anticipated in the fourth quarter
of 2019.
In February 2019, the Company
reported that administration of intranasal BHV-3500 in a Phase 1
clinical trial had achieved targeted therapeutic exposures and
demonstrated significantly earlier time to maximal concentration
(Tmax) than those observed with other small molecule CGRP receptor
antagonists, suggesting the potential for an ultra-rapid onset of
action without the need for an injection. BHV-3500 is a novel,
third generation CGRP receptor antagonist being developed by
Biohaven. Intranasal BHV-3500 utilizes the Aptar Pharma Unit
Dose System, which is designed to enable systemic delivery of drugs
without the need for injection or administration by a healthcare
professional.
- Secured Priority Review Voucher to Expedite Regulatory
Review of Rimegepant Zydis ODT NDA – In March 2019, the Company announced the purchase of
a U.S. Food and Drug Administration (FDA) priority review
voucher (PRV) to use with the New Drug Application (NDA) submission
for rimegepant Zydis® ODT. The PRV
entitles the holder to designate an NDA for priority review and
provides for an expedited 6-month review.
- Announced $125 million
transaction with Royalty Pharma – The purchase of the PRV, and
additional funding for general corporate uses, was funded by a sale
of $125 million preferred shares to
Royalty Pharma.
- Announced Completion of Pre-NDA meeting with FDA for Oral
CGRP Receptor Antagonist Rimegepant – In March 2019, the Company announced that it
concluded its pre-NDA meetings with the FDA for rimegepant
Zydis® ODT and tablet formulations for the acute
treatment of migraine. Biohaven submitted a pre-NDA briefing
document to the FDA that outlined the Company's preliminary data
package being prepared for the NDA submission, including clinical
safety and efficacy, non-clinical results, CMC and other regulatory
elements. Based on the feedback from the FDA, the Company believes
its regulatory data package will be sufficient for submission, with
acceptance of the final NDA subject to the FDA's review
of the complete filing.
- Commenced Enrollment in Phase 3 Clinical Trial of
Troriluzole in SCA – In March 2019, the Company announced enrollment in a
Phase 3 clinical trial assessing the efficacy and safety of
troriluzole in Spinocerebellar Ataxia (SCA). The trial will
randomize approximately 230 patients across two treatment arms, 200
mg of troriluzole versus placebo, and enrollment completion is
anticipated in the first quarter of 2020. Based on findings from
the previous Phase 2b/3 clinical
trial, Biohaven enriched the trial with specific genotypes,
extended the treatment period of the trial to one year, implemented
the use of a modified SARA scale and increased the dose of
troriluzole from 140 to 200 mg.
The Company also announced results from a post-hoc analysis of
patients enrolled in the short-term randomization and long-term
extension phase of Study BHV4157-201, an initial Phase 2b/3 randomized controlled trial of troriluzole
in patients with SCA, compared to patients selected from a natural
history cohort of SCA patients who were matched on multiple
eligibility criteria. Based on analysis of covariance (ANCOVA)
least square mean changes after one year were -0.34 points
(representing numerical improvement with a 95% confidence interval
of -0.94 to 0.26) for 81 troriluzole-treated patients versus +1.07
points (representing numerical decline with a 95% confidence
interval of 0.56 to 1.58) for 112 natural history cohort patients
(increasing score indicates worsening disease status). The LS Mean
difference between cohorts was -1.41 points (95% confidence
interval of -2.22 to -0.60) suggesting therapeutic benefits of
troriluzole (p=0.0007). Patients in Study BHV4157-201 were treated
with 140 mg of troriluzole administered daily for one year.
- Commenced Enrollment in Phase 3 Clinical Trial of
Troriluzole in GAD – In February 2019, the Company announced enrollment
in a Phase 3 clinical trial assessing the efficacy and safety of
troriluzole in GAD. The trial will randomize approximately 372
patients across two treatment arms, 100 mg of troriluzole versus
placebo, and enrollment completion is anticipated in the fourth
quarter of 2019. The primary outcome measure is the change in a
patient's score on the Hamilton Anxiety Rating Scale, a scale
designed to assess the severity and type of symptoms in patients
with GAD. The trial will also assess the safety, tolerability and
pharmacokinetics of troriluzole. Troriluzole is a
third-generation tripeptide prodrug and new chemical entity that
modulates glutamate, the most abundant excitatory neurotransmitter
in the human body. The therapeutic potential of troriluzole in GAD
is supported by clinical and translational research studies
suggesting that glutamatergic dysfunction is implicated in the
pathophysiology of anxiety disorders.
- Received Orphan Drug Designation from FDA for Verdiperstat
for Multiple System Atrophy – In February 2019, the Company announced receipt of
orphan drug designation from the FDA for its product candidate
verdiperstat (previously BHV-3241), a novel myeloperoxidase (MPO)
inhibitor, for the treatment of multiple system atrophy (MSA).
Verdiperstat completed Phase 1 clinical trials at doses up to 900mg
twice a day and preliminary results from a Phase 2a trial in
patients with MSA showed numerical improvements on the change from
baseline Unified MSA Rating Scale.
The Company had received notification from the FDA that it may
proceed with its clinical investigation of verdiperstat in January
of 2019. The FDA May Proceed Letter was received following
Biohaven's reactivation of the IND application initially filed by
AstraZeneca prior to licensing the compound to Biohaven.
Verdiperstat is a potential first-in-class, oral, brain-penetrant,
irreversible inhibitor of MPO, an enzyme that acts as a key driver
of pathological oxidative stress and inflammation in the brain.
- Announced Acceptance of IND Filing for Rimegepant in
China – In January 2019, the Company, together with its
wholly-owned Asia-Pacific
subsidiary company BioShin, announced that the National Medical
Products Administration (NMPA) had accepted its IND application for
rimegepant, Biohaven's lead oral CGRP receptor antagonist product
candidate, for the treatment of migraine. Rimegepant is the
first small molecule, orally administered, CGRP receptor antagonist
potentially being developed for both the acute and preventive
treatment of migraine in China. Biohaven's wholly-owned
Asia-Pacific subsidiary, BioShin,
is developing and potentially commercializing Biohaven's late-stage
product portfolio in China.
Upcoming Milestones:
Biohaven is progressing drug
candidates through clinical programs in a number of common and rare
disorders. The Company expects to reach significant pipeline
milestones with its CGRP receptor antagonists, glutamate modulators
and myeloperoxidase inhibitor in the coming quarters.
The Company expects to:
- Continue to advance the rimegepant Zydis® ODT (orally
dissolving tablet) and tablet formulation development programs
towards commercialization for the
acute treatment of migraine.
- Report Phase 3 topline data for rimegepant in preventive
treatment of migraine in 4Q2019.
- Report Phase 2/3 topline efficacy and safety results for
intranasal BHV-3500 in acute treatment of migraine in 4Q2019.
- Initiate a Phase 2 proof of concept trial in 2019 to evaluate
the safety and efficacy of rimegepant in patients with treatment
refractory trigeminal neuralgia.
- Hold an Investor and Key Opinion Leader Event at AAN.
- Announce PDUFA decision on Nurtec™ NDA for treatment of
amyotrophic lateral sclerosis via Section 505(b)(2) pathway in
3Q2019.
- Complete enrollment in Phase 2/3 trial of troriluzole in
Alzheimer's disease in 4Q2019.
- Complete enrollment in Phase 2/3 trial with troriluzole in
Obsessive-compulsive Disorder by the end of 2019.
- Complete enrollment in Phase 2/3 trial of troriluzole in
Generalized Anxiety Disorder by the end of 2019.
- Complete enrollment in Phase 3 trial of troriluzole in
Spinocerebellar Ataxia in the first quarter of 2020.
- Initiate a Phase 3 clinical trial for the treatment of MSA, a
rare, rapidly progressive and fatal neuroinflammatory disease with
no cure or effective treatments, in 3Q2019.
First Quarter 2019 Financial Results
Cash
Position: Cash as of March 31,
2019 was $217.4 million, compared to $264.2
million as of December 31,
2018.
R&D Expenses: Research and development (R&D)
expenses were $41.0 million for the three months ended
March 31, 2019, compared
to $75.6 million for the three months ended March 31, 2018. The decrease of $34.6 million was primarily due to the upfront
payment to BMS in the three months ended March 31, 2018 of $50.0
million, partially offset by increases in direct costs of
$6.3 million for the BHV-3500
program, $3.4 million in personnel
costs, including non-cash share-based compensation, $3.4 million in direct costs for the troriluzole
program, and $2.0 million in direct
costs for the rimegepant program. The increases in direct costs for
the BHV-3500 and troriluzole programs were primarily due to an
increase in the number of clinical trials, and the cost of
operating later-stage trials, for the three months ended
March 31, 2019 as compared to the
same period in 2018. The increase in personnel-related costs,
including non-cash share-based compensation, was a result of hiring
additional research and development personnel. Headcount in R&D
increased to 43 as of March 31, 2019,
compared to 29 as of March 31,
2018. Non-cash share-based compensation expense, included in
personnel-related costs, was $3.7
million for the three months ended March 31, 2019, an increase of $2.3 million as compared to the same period in
2018.
G&A Expenses: General and administrative
(G&A) expenses were $13.5 million for the three
months ended March 31, 2019, compared
to $7.9 million for the three months ended March 31, 2018. The increase of $5.6 million was primarily due to increases in
personnel-related costs, including non-cash share-based
compensation, due to the hiring of additional personnel in G&A
functions, preparation for commercialization activities,
professional fees supporting ongoing business operations, and
additional fees to comply with being a public company. Headcount,
outside of R&D, increased to 30 as of March 31, 2019, compared to 20 as of March 31, 2018. Non-cash share-based
compensation expense, included in personnel-related costs, was
$3.6 million for the three months
ended March 31, 2019, an increase of
$2.0 million as compared to the same
period in 2018.
Net Loss: The Company reported a net loss
attributable to common shareholders of $62.3
million or $1.41 per share for the three months
ended March 31, 2019, compared to $85.5 million,
or $2.32 per share, for the three months ended
March 31, 2018.
About Biohaven
Biohaven is a clinical-stage
biopharmaceutical company with a portfolio of innovative,
late-stage product candidates targeting neurological diseases,
including rare disorders. Biohaven has combined internal
development and research with intellectual property licensed from
companies and institutions including Bristol-Myers Squibb Company,
AstraZeneca AB, Yale University,
Catalent, ALS Biopharma LLC and Massachusetts General Hospital.
Currently, Biohaven's lead development programs include multiple
compounds across its CGRP receptor antagonist and glutamate
modulator platforms. The Company's common shares are listed on the
New York Stock Exchange and traded under the ticker symbol BHVN.
More information about Biohaven is available
at www.biohavenpharma.com.
About Catalent
Catalent is the leading global provider
of advanced delivery technologies and development solutions for
drugs, biologics and consumer health products. With over 85 years
serving the industry, Catalent has proven expertise in bringing
more customer products to market faster, enhancing product
performance and ensuring reliable clinical and commercial product
supply. Catalent employs over 11,000 people, including over 1,800
scientists, at more than 30 facilities across five continents, and
in fiscal 2018 generated approximately $2.5 billion in
annual revenue. Zydis® is a registered trademark of Catalent.
Catalent is headquartered in Somerset, New Jersey. For more information,
visit www.catalent.com.
Forward-Looking Statements
This news release includes
forward-looking statements within the meaning of the Private
Securities Litigation Reform Act of 1995. These forward-looking
statements involve substantial risks and uncertainties, including
statements that are based on the current expectations and
assumptions of the Company's management. All statements, other than
statements of historical facts, included in this press release
regarding the Company's business and product candidate plans and
objectives are forward-looking statements. Forward-looking
statements include those related to: the expected commencement and
completion of clinical trials, the anticipated timing of
availability of data from those trials, the timing of expected
regulatory submissions and approvals, the efficacy and safety
profiles of the Company's product candidates and their expected
benefits compared to other treatment options, and other statements
regarding the Company's plans and objectives, expectations and
assumptions of management. The use of certain words, including the
words "expect," "anticipate," "will," "potential," "plan," "might"
and similar expressions are intended to identify forward-looking
statements. The Company may not actually achieve the plans,
intentions or expectations disclosed in the forward-looking
statements and you should not place undue reliance on the Company's
forward-looking statements. Various important factors could cause
actual results or events to differ materially from those that may
be expressed or implied by the forward-looking statements including
risks and uncertainties related to the timing of initiating,
enrolling and completing clinical trials; the commencement or
completion of enrollment in any clinical trial does not guarantee
the continuation or successful outcome of the trial, or the
acceptance by the FDA of a regulatory package for the drug
candidate being tested; the submission of an IND does not guarantee
that the FDA will permit clinical trials to begin; and those
factors described in the "Risk Factors" section of the Company's
Annual Report on Form 10-K filed with the Securities and Exchange
Commission on February 28, 2019 and
the Company's other filings with the Securities and Exchange
Commission. The forward-looking statements are made as of this date
and the Company 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.
BIOHAVEN
PHARMACEUTICAL HOLDING COMPANY LTD.
|
CONDENSED
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE
LOSS
|
(Amounts in
thousands, except share and per share amounts)
|
(Unaudited)
|
|
|
|
Three Months Ended
March 31,
|
|
|
2019
|
2018
|
Operating
expenses:
|
|
|
|
Research and
development
|
|
$
|
41,003
|
$
|
75,579
|
General and
administrative
|
|
13,462
|
7,857
|
Total operating
expenses
|
|
54,465
|
83,436
|
Loss from
operations
|
|
(54,465)
|
(83,436)
|
Other income
(expense):
|
|
|
|
Non-cash interest
expense on liability related to sale of future royalties
|
|
(6,813)
|
—
|
Change in fair value of
warrant liability
|
|
—
|
(1,182)
|
Loss from equity method
investment
|
|
(900)
|
(728)
|
Other
|
|
(17)
|
(29)
|
Total other expense,
net
|
|
(7,730)
|
(1,939)
|
Loss before provision
for income taxes
|
|
$
|
(62,195)
|
$
|
(85,375)
|
Provision for income
taxes
|
|
109
|
87
|
Net loss and
comprehensive loss
|
|
(62,304)
|
(85,462)
|
Net loss per share —
basic and diluted
|
|
$
|
(1.41)
|
$
|
(2.32)
|
Weighted average
common shares outstanding—basic and diluted
|
|
44,242,070
|
36,793,090
|
BIOHAVEN
PHARMACEUTICAL HOLDING COMPANY LTD.
|
CONDENSED
CONSOLIDATED BALANCE SHEETS
|
(Amounts in
thousands)
|
|
|
March 31,
2019
|
December 31,
2018
|
|
(Unaudited)
|
|
Assets
|
|
|
Current
assets:
|
|
|
Cash
|
$
|
217,407
|
$
|
264,249
|
Prepaid expenses and
other current assets
|
9,966
|
8,090
|
Total current
assets
|
227,373
|
272,339
|
Property and
equipment, net
|
7,157
|
6,248
|
Equity method
investment
|
10,514
|
11,414
|
Other
assets
|
36
|
11
|
Total
assets
|
$
|
245,080
|
$
|
290,012
|
Liabilities and
Shareholders' Equity
|
|
|
Current
liabilities:
|
|
|
Accounts
payable
|
$
|
8,210
|
$
|
10,752
|
Accrued
expenses
|
15,483
|
8,782
|
Total current
liabilities
|
23,693
|
19,534
|
Liability related to
sale of future royalties, net
|
124,332
|
117515
|
Other long-term
liabilities
|
44
|
2,043
|
Total
liabilities
|
$
|
148,069
|
$
|
139,092
|
Total shareholders'
equity
|
$
|
97,011
|
$
|
150,920
|
Total liabilities and
shareholders' equity
|
$
|
245,080
|
$
|
290,012
|
For further information, contact Dr. Vlad Coric, Chief Executive Officer,
at Vlad.Coric@biohavenpharma.com
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SOURCE Biohaven Pharmaceutical Holding Company Ltd.