NEW HAVEN, Conn., Aug. 8,
2019 /PRNewswire/ -- Biohaven Pharmaceutical Holding Company
Ltd. (NYSE: BHVN, the "Company"), a clinical-stage
biopharmaceutical company with a portfolio of innovative,
late-stage product candidates targeting neurological and
neuropsychiatric diseases, today reported financial results for the
quarter ended June 30, 2019, and
provided a review of recent accomplishments and anticipated
milestones.
Vlad Coric, M.D., CEO of Biohaven
commented, "The submission of our NDAs to the FDA for rimegepant
represents a significant achievement for Biohaven and advances the
development of what we believe will be an important next generation
oral therapy for the acute treatment of migraine." Dr. Coric
continued, "The strength of our clinical data is reflected in the
recent publications in the New England Journal of Medicine and the
Lancet, highlighting rimegepant's early onset of action and
sustained pain freedom and relief out to 48 hours after taking a
single dose. We are excited about advancing this drug candidate for
migraine patients that want or need another treatment option."
Biohaven also applied a Priority Review Voucher (PRV) to its
rimegepant Zydis ODT NDA in the second quarter of 2019. A PRV
entitles the holder to designate an NDA for priority review and
provides for an expedited 6-month review of the application.
Jim Engelhart, Biohaven's CFO
added, "We have recently completed a common shares public offering
with gross proceeds of $322.6
million that we believe puts the company in a strong financial
position to continue to advance the Company's goals including, (1)
advancing the rimegepant Zydis® ODT and tablet formulation
development programs towards commercialization for the
acute treatment of migraine, (2) reporting Phase 3 topline data for
rimegepant in the preventive treatment of migraine, (3) reporting
Phase 2/3 topline efficacy for intranasal BHV-3500 in acute
treatment of migraine in the fourth quarter of 2019, (4) continuing
to enroll patients in a Phase 2 proof of concept trial evaluating
the safety and efficacy of rimegepant in patients with treatment
refractory trigeminal neuralgia, (5) completing enrollment in three
Phase 2/3 clinical trials of troriluzole in Alzheimer's disease,
Obsessive-Compulsive Disorder, and Generalized Anxiety Disorder by
the end of 2019, and (6) initiating a Phase 3 clinical trial for
the treatment of MSA in the third quarter of 2019."
Second Quarter and Recent Business Highlights
Oral CGRP-Antagonist Programs:
- Submitted New Drug Applications for rimegepant and applied
PRV – In the second quarter of 2019, the Company submitted New
Drug Applications (NDAs) with the U.S. Food and Drug Administration
(FDA) for the Zydis ODT and tablet formulations of rimegepant. The
Company also applied its PRV to the Zydis ODT NDA.
- Published Phase 3 data in leading medical journals and
presented expanded data at the AHS Annual Scientific
Meeting – In July, the Company presented sixteen
scientific presentations (oral and poster) at the American Headache
Society (AHS) Annual Scientific Meeting highlighting new efficacy
and safety data for rimegepant; notably, Biohaven presented the
following posters:
-
- Poster #P235LB: Interim results from the
long-term, open-label safety study of rimegepant 75 mg in over
1,780 patients with a combined exposure of over 105,000 doses were
presented for the first time at a scientific meeting. Patients
were dosed up to one year and were allowed to take rimegepant up to
once daily. The drug was well tolerated in adults with migraine,
only 2.7% of subjects discontinued due to an adverse event, and
there were no signs of hepatoxicity. A cohort with high-frequency,
scheduled dosing of rimegepant 75 mg at least every other day
demonstrated consistent results with the overall population.
- Poster #P239LB: A pooled analysis of results
from the three rimegepant Phase 3 trials looked at the efficacy of
rimegepant in patients taking concurrent preventive medication and
found rimegepant to be more effective than placebo for the acute
treatment of migraine. Among subjects on preventive medication
(547 subjects, rimegepant n=272, placebo n=275), pooled two-hour
pain freedom rates for rimegepant and placebo were 20.6% and 10.2%,
respectively (p=0.0007). Additionally, the two-hour rates for
freedom from most bothersome symptom were 37.1% for rimegepant and
20.4% for placebo (p<0.0001).
- Poster #P236LB: A pooled analysis of results
from the three rimegepant Phase 3 trials looked at the efficacy of
rimegepant in patients with high migraine attack frequency (at
least four attacks per month), a patient population that has
previously been shown to be more difficult to treat, and found
rimegepant was an effective acute treatment of migraine. Among
subjects with 4 or more moderate or severe attacks per month (2426
subjects, rimegepant n=1217, placebo n=1209), two-hour pain freedom
rates for rimegepant and placebo among these patients were 20.6%
and 12.6%, respectively (p<0.0001). For two-hour freedom from
the most-bothersome symptom, the rates were 35.8% and 26.9% for
rimegepant and placebo, respectively (p<0.0001).
- Poster #P238LB: The first clinical report
evaluating the efficacy and safety of rimegepant for the acute
treatment of migraine for breakthrough attacks in two patients
concurrently receiving erenumab, a CGRP receptor binding monoclonal
antibody, for preventive treatment of migraine was presented for
the first time in a scientific meeting. The two patients were
enrolled in the rimegepant long-term open-label safety study. With
rimegepant used as an acute treatment, both patients were able to
eliminate use of additional rescue medications and successfully
treat their attacks with rimegepant alone. After starting erenumab
for preventive treatment, both patients were able to successfully
treat breakthrough attacks with oral rimegepant that occurred
despite the use of the injectable monoclonal antibody preventive
therapy.
- Announced expanded safety and preliminary preventive
efficacy data from ongoing long-term safety study and presented
case reports of using rimegepant to successfully treat breakthrough
migraine attacks in patients taking preventive CGRP-targeting
monoclonal antibodies – In May, the Company announced expanded
safety & efficacy data from an ongoing long-term safety study
with rimegepant at the American Academy of Neurology Annual
Meeting.
- Based upon the interim analysis performed at the database
cutoff of February 20, 2019 of Study BHV3000-201 (NCT03266588 or
"Study 201"), the Company reported that rimegepant was well
tolerated with long-term dosing up to one year in patients with
migraine. At that point in time, 105,192 doses of rimegepant 75 mg
were administered up to once daily to over 1,780 patients with
migraine. Interim hepatic data were reviewed by an external
independent panel of liver experts who concluded that there was no
liver safety signal and compared to placebo arms of other migraine
treatments, there was a very low incidence of overall elevations of
liver laboratory abnormalities (1% incidence of serum ALT or AST
> 3x the upper limit of normal (ULN) through the data analysis
cut-off date).
For the first time, the Company also reported Study 201 safety
& preliminary efficacy data from the 286 cohort of patients
with a history of 4 to 14 moderate to severe migraine attacks per
month, treated with rimegepant 75 mg every other day for up to 12
consecutive weeks. During the on-treatment period, no
rimegepant-treated patients (N=281) experienced ALT or AST levels
>3x the ULN. There were also no rimegepant-treated patients who
experienced alkaline phosphatase or bilirubin >2x the ULN. With
regard to efficacy, 48.4% of subjects in the scheduled dosing
cohort experienced a ≥50% reduction in the frequency of monthly
migraine days with moderate-to-severe pain intensity during the
third month of treatment.
- The Company also presented the first clinical reports
suggesting that oral rimegepant has the potential to be safe and
consistently effective for the treatment of breakthrough migraine
attacks in patients taking injectable CGRP-targeting monoclonal
antibody preventive therapy.
- Enrolled first patient in Phase 2 trial of rimegepant for
trigeminal neuralgia – In July, the Company announced it
enrolled the first patient in a Phase 2 clinical trial of
rimegepant for the treatment of refractory trigeminal neuralgia.
This trial will enroll patients in a double-blind,
placebo-controlled, crossover design study at Johns Hopkins Medical
Center.
- Announced completion of enrollment in pivotal Phase 3
migraine prevention trial – In August, the Company announced it
completed enrollment in its Phase 3 trial of rimegepant as a
preventive therapy for migraine. The Phase 3 trial examines the
efficacy and safety of rimegepant in adult subjects who have
suffered from migraine for at least one year and who have a
frequency of 4 to 14 migraine attacks of moderate to severe
intensity per month over the three months prior to enrollment. Top
line data is expected in the fourth quarter of 2019.
- Advanced enrollment in the intranasal BHV-3500, Biohaven's
third generation CGRP-antagonist, Phase 2 dose finding study.
- Launched the "Demand More" migraine disease awareness campaign
at the 2019 American Headache Society Annual Scientific Meeting
highlighting patient needs in the acute treatment of migraine. The
"Demand More" campaign is based on research that suggests patients
are not getting the relief they want and need from their current
acute treatments. In surveys of patients with migraine, 56 percent
of patients reported they still experienced migraine pain two hours
post-treatment, and 54 percent of patients taking existing acute
treatments reported inadequate pain relief at 24 hours.
Approximately 48 percent of patients weren't satisfied with their
ability to function after taking their current acute treatment.
Approximately 67 percent of patients reported they delayed or
avoided acute medications due to concerns about side effects. More
information about the campaign can be found at:
www.realrelieffrommigraine.com
Glutamate Modulating Program:
- Provided an update on Phase 2/3 Alzheimer's Disease clinical
trial – In July, the Company announced that enrollment in the
T2 Protect AD Study, a Phase 2/3 clinical trial of troriluzole in
Alzheimer's disease, is progressing on schedule with more than 400
subjects enrolled (for screening) and over 180 subjects randomized
to study treatment (troriluzole or placebo). The trial is expected
to be fully enrolled before the end of the fourth quarter of 2019
and the Company, in collaboration with the Alzheimer's Disease
Cooperative Study (ADCS), anticipates performing the pre-planned
interim futility analysis by the end of 2019.
- Provided an update on Nurtec (riluzole) 505(b)2
application – In July, the Company announced that it had
received a Complete Response Letter (CRL) from the FDA for its
505(b)2 application seeking approval for NURTEC™ (riluzole) for the
treatment of Amyotrophic Lateral Sclerosis (ALS). The sole issue
identified in the CRL relates to an FDA concern regarding the use
of an active pharmaceutical ingredient (API) manufactured by Apotex
Pharmachem India Private Limited (Apotex) and used in the drug
product supplies for the bioequivalence study in 2017. In the CRL,
the FDA stated that it provided recommendations to Apotex regarding
the information needed to qualify previous API batches manufactured
at Apotex during the time period in question. Biohaven has been
subsequently informed by the manufacturer that the manufacturer had
an exemption from the FDA to supply riluzole to the U.S. market
during that time period. Biohaven has been in contact with the
FDA's Chemical Manufacturing Controls (CMC) group and Apotex to
resolve the matter and Biohaven has already submitted additional
information to the FDA regarding this issue. Biohaven notes that
the API for commercial supply of Nurtec is currently sourced from
another supplier, with whom no CMC issues have been identified. The
FDA did not cite any other concerns in their CRL regarding
Nurtec.
- Completed a $322.6 million
Public Offering of Common Shares - In June 2019, the Company announced the pricing of
an underwritten public offering of 6,976,745 of its common shares
at a price to the public of $43.00
per share for net proceeds of $281.1
million after deducting underwriting discounts and
commissions of $18.0 million and
other offering expenses of approximately $0.9 million. Subsequently, in July 2019, the underwriters of the follow-on
offering partially exercised their option to purchase additional
shares, and Biohaven issued and sold 525,000 common shares for
additional net proceeds of $21.2
million after deducting underwriting discounts and
commissions of $1.4 million. Thus,
the aggregate net proceeds from the follow-on offering, after
deducting underwriting discounts and commissions and other offering
costs, were $302.3 million. The
Company plans to use the net proceeds from the offering for
advancing and expanding the development and commercialization
activities of Biohaven's ongoing oral CGRP antagonist platform,
advancing and expanding the development of glutamate modulation
platform product candidates and the MPO platform and for working
capital and other corporate purposes. We believe that the aggregate
proceeds received through this offering, in addition to current
cash on hand, provides Biohaven with the resources to support and
expand the Company's rimegepant development program through
commercialization, advance the glutamate modulator platform, and
further develop the Company's earlier-stage pipeline, clinical
infrastructure and commercialization activities beyond the next
twelve months.
Upcoming Milestones
Biohaven is progressing drug
candidates for a number of common and rare disorders through
clinical programs. 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 the fourth quarter of 2019.
- Report Phase 2/3 topline efficacy and safety results for
intranasal BHV-3500 in acute treatment of migraine in fourth
quarter of 2019.
- Complete enrollment in Phase 2/3 trial of troriluzole in
Alzheimer's disease and conduct futility analysis in the fourth
quarter of 2019.
- 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.
- Continue enrolling patients in Phase 2 proof of concept trial
evaluating the safety and efficacy of rimegepant in patients with
treatment refractory trigeminal neuralgia.
- Initiate a Phase 3 clinical trial for the treatment of multiple
system atrophy (MSA), a rare, rapidly progressive and fatal
neuroinflammatory disease with no cure or effective treatments, in
the third quarter of 2019.
- Conduct ongoing non-clinical studies defined under the
scientific research agreement with University
of Connecticut to support the advancement of UC1MT, a
therapeutic antibody targeting extracellular metallothionein.
Second Quarter and First Half 2019 Financial
Results
Cash Position: Cash as of June 30,
2019, was $465.7 million, compared to $217.4
million as of March 31, 2019. This increase was
primarily due to net proceeds of approximately $281.1 million
from the Company's June common share offering. Subsequent to
June 30, 2019, we received additional
net proceeds of $21.2 million from
the underwriters of the follow-on offering who partially exercised
their option to purchase additional shares.
Research and Development (R&D) Expenses: R&D
expenses, including one-time regulatory and non-cash share-based
compensation costs, were $176.0
million for the three months ended June 30, 2019, compared to $29.1 million for the three months ended
June 30, 2018. The increase of
$146.9 million was primarily due
to:
- the purchase of a PRV for $105.0
million to expedite the regulatory review of the ODT version
of rimegepant;
- filing fees of $7.6 million
related to the Company's submission of NDAs with the FDA for the
Zydis ODT and tablet formulations of rimegepant;
- accrual for CGRP development milestones payable to
Bristol-Myers Squibb Company in the amount of $11.5 million;
- one-time issuance of common shares to Fox Chase Chemical
Diversity Center Inc. which resulted in an increase of $5.6 million in direct costs for our preclinical
research program; and
- an increase of $7.5 million in
non-cash share-based compensation.
The issuance of $5.6 million of
common shares to Fox Chase Chemical Diversity Center Inc. was for
preclinical development for eventual clinical evaluation for the
treatment of one or more TDP-43 proteinopathies.
The increase in personnel related costs, including non-cash
share-based compensation, was a result of additional options issued
and hiring additional research and development personnel. Our
headcount, in research and development, increased to 47 as of
June 30, 2019, compared to 30 as of
June 30, 2018. Non-cash
share-based compensation expense was $10.3
million for the three months ended June 30, 2019, an increase of $7.5 million as compared to the same period in
2018.
General and Administrative (G&A)
Expenses: G&A expenses, including non-cash
share-based compensation costs, were $23.2
million for the three months ended June 30, 2019, compared to $9.1 million for the three months ended
June 30, 2018. The increase of
$14.2 million was primarily due to
increases in personnel-related costs, including non-cash
share-based compensation, due to the hiring of additional personnel
in our general and administrative functions primarily to prepare
for commercialization of our product candidates, and increases in
professional fees supporting ongoing business operations, and
additional fees to comply with the requirements of being a public
company. Our headcount, in general and administrative activities,
increased to 43 as of June 30, 2019,
compared to 23 as of June 30,
2018. Non-cash share-based compensation expense, included in
personnel-related costs, was $7.2 million for the three months ended
June 30, 2019, an increase of
$4.4 million as compared to the same
period in 2018.
Net Loss: The Company reported a net loss
attributable to common shareholders for the three months ended
June 30, 2019 of $211.1 million, or $4.67 per share, compared to $39.3 million,
or $1.01 per share for the same period in 2018. Net
loss per share, adjusted to remove the effects of the $105.0 million one-time payment for the PRV in
April 2019 was $2.35 per share for the three months ended
June 30, 2019.
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, and ALS Biopharma LLC. Currently, Biohaven's lead
development programs include multiple compounds across its CGRP
receptor antagonist, glutamate modulation and myeloperoxidase
inhibition platforms. More information about Biohaven is available
at www.biohavenpharma.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 timing,
commencement and outcomes of the Company's planned and ongoing
clinical trials, the timing of planned interactions and filings
with the FDA, the timing and outcome of expected regulatory
filings, the potential commercialization of the Company's
product candidates, the potential for the Company's product
candidates to be first in class or best in class therapies and the
effectiveness and safety of the Company's product candidates. The
use of certain words, including "believe", "continue", "may", "on
track", "expects" and "will" and similar expressions, are intended
to identify forward-looking statements. Various important factors
could cause actual results or events to differ materially from
those that may be expressed or implied by our forward-looking
statements. Additional important factors to be considered in
connection with forward-looking statements are 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 Quarterly Report on Form
10-Q for the quarter ended March 31,
2019, filed with the Securities and Exchange Commission on
May 8, 2019. 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
June 30,
|
|
|
2019
|
|
2018
|
Operating
expenses:
|
|
|
|
|
Research and
development
|
|
$
|
175,977
|
|
$
|
29,052
|
General and
administrative
|
|
23,235
|
|
9,064
|
Total operating
expenses
|
|
199,212
|
|
38,116
|
Loss from
operations
|
|
(199,212)
|
|
(38,116)
|
Other income
(expense):
|
|
|
|
|
Non-cash interest
expense on mandatorily redeemable preferred shares
|
|
(3,955)
|
|
—
|
Non-cash interest
expense on liability related to sale of future royalties
|
|
(5,151)
|
|
(501)
|
Change in fair value
of derivative liability
|
|
(1,263)
|
|
—
|
Loss from equity
method investment
|
|
(1,415)
|
|
(641)
|
Other
|
|
(16)
|
|
14
|
Total other expense,
net
|
|
(11,800)
|
|
(1,128)
|
Loss before provision
for income taxes
|
|
$
|
(211,012)
|
|
$
|
(39,244)
|
Provision for income
taxes
|
|
58
|
|
25
|
Net loss and
comprehensive loss
|
|
(211,070)
|
|
(39,269)
|
Net loss per share —
basic and diluted
|
|
$
|
(4.67)
|
|
$
|
(1.01)
|
Weighted average
common shares outstanding—basic and diluted
|
|
45,226,434
|
|
38,942,545
|
BIOHAVEN
PHARMACEUTICAL HOLDING COMPANY LTD.
CONDENSED
CONSOLIDATED BALANCE SHEETS
(Amounts in
thousands)
|
|
|
|
|
June 30,
2019
|
|
December 31,
2018
|
|
|
(Unaudited)
|
|
|
Assets
|
|
|
|
|
Current
assets:
|
|
|
|
|
Cash
|
|
$
|
465,739
|
|
$
|
264,249
|
Prepaid expenses and
other current assets
|
|
8,613
|
|
8,090
|
Total current
assets
|
|
474,352
|
|
272,339
|
Property and
equipment, net
|
|
7,433
|
|
6,248
|
Equity method
investment
|
|
9,099
|
|
11,414
|
Other
assets
|
|
24
|
|
11
|
Total
assets
|
|
$
|
490,908
|
|
$
|
290,012
|
Liabilities and
Shareholders' Equity
|
|
|
|
|
Current
liabilities:
|
|
|
|
|
Accounts
payable
|
|
$
|
13,502
|
|
$
|
10,752
|
Accrued
expenses
|
|
31,167
|
|
8,782
|
Total current
liabilities
|
|
44,669
|
|
19,534
|
Liability related to
sale of future royalties, net
|
|
129,487
|
|
117515
|
Mandatorily
redeemable preferred shares, net
|
|
94,859
|
|
—
|
Derivative
liability
|
|
23,815
|
|
—
|
Other long-term
liabilities
|
|
43
|
|
2,043
|
Total
liabilities
|
|
$
|
304,167
|
|
$
|
139,092
|
Total shareholders'
equity
|
|
$
|
186,741
|
|
$
|
150,920
|
Total liabilities and
shareholders' equity
|
|
$
|
490,908
|
|
$
|
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.