Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company),
a rare disease therapeutics company, today provided corporate
updates and reported its financial results for the second quarter
ended June 30, 2024.
“During the second quarter, we made steady
progress executing on our strategic objectives,” said Neil
F. McFarlane, President and Chief Executive Officer of
Zevra. “We are encouraged by favorable vote from the FDA’s
Genetic Metabolic Diseases Advisory Committee, that data presented
support that arimoclomol is effective in the treatment in patients
with Niemann Pick disease type C. While the vote is non-binding, we
believe it is an important factor as the FDA completes its
consideration for approval. Additionally, we continue
the launch of OLPRUVA with a keen focus on driving patient
awareness and intend to use the same commercial team to launch
arimoclomol, if approved. Additionally, we expect to meet with the
FDA to discuss the design of a pivotal Phase 3 trial to study
KP1077 in idiopathic hypersomnia at the end of third quarter.”
“We continue to be prudent in our capital
allocation as we focus on creating long-term value for
stockholders,” said R. LaDuane Clifton, Zevra’s Chief
Financial Officer and Treasurer. “Our recent underwritten
offering provided net proceeds to the Company of $64.5 million,
along with attracting a cadre of institutional investors well known
as long term supporters of innovation and building momentum as we
lean into our near-term catalysts while also extending our cash
runway.”
Q2 2024 Corporate
Highlights:
- Arimoclomol
- On August 2, 2024, the U.S. Food
and Drug Administration’s (FDA) newly formed Genetic Metabolic
Diseases Advisory Committee (the “GeMDAC”) convened for the first
time and discussed the benefits and risks of arimoclomol.
- The GeMDAC reviewed comments
received from independent experts, NPC patients, and patient
advocacy group representatives, and voted favorably that
arimoclomol is effective in the treatment of NPC.
- On Friday, August 9, 2024, the
Company received the first round of labeling comments, and is
working closely with the FDA.
- OLPRUVA
- On June 18, 2024, announced
transition of specialty pharmacy partner to Orsini.
- Increased OLPRUVA reimbursement
coverage to 75% of covered lives and improved preferred status on
formulary plans.
- Added nine (9) new patient
enrollments during Q2 2024, which is defined as a prescription for
a patient on the Quick Start program or receiving a paid dispense
of OLPRUVA.
- KP1077
- On June 3, 2024, presented positive
topline data from its Phase 2 study in patients with idiopathic
hypersomnia (IH) at the SLEEP 2024 Annual Meeting.
- The Company submitted a briefing
book to the FDA for an end-of-Phase 2 meeting to be held at the end
of Q3 2024.
- Celiprolol
- Restarted recruitment for the Phase
3 DiSCOVER trial of celiprolol in patients with Vascular
Ehler-Danlos Syndrome (VEDS).
- Corporate
- On April 5, 2024, the Company
entered into a $100 million credit facility with leading biotech
investors, Perceptive Advisors and Healthcare Royalty
Partners.
- On June 25, 2024, the Company
announced the appointment of Rahsaan Thompson as Chief Legal
Officer, Secretary and Compliance Officer, and Alison Peters as
Chief People Officer, bringing expertise that will support the next
phase of the Company’s growth.
Overview of Q2 2024 Financial
Results:
Net revenue for Q2 2024 was $4.4 million,
compared to net revenue of $8.5 million in Q2 2023. The components
of revenue during the second quarter included $3.1 million in net
reimbursements from the French EAP for arimoclomol, $1.3 million of
royalties and other reimbursements under the AZSTARYS® License
Agreement, and de minimis OLPRUVA revenue via sales to the new
specialty pharmacy, Orsini, were offset by returns from the prior
pharmacy in Q2 2024. In addition, cost of goods sold
was inflated during the quarter due to recognition of a $3.2
million obsolescence reserve against OLPRUVA inventory which is
nearing expiration. This excess inventory was ordered prior to our
acquisition of Acer, and the delayed launch impacted the rate of
usage, leading to the need for this reserve to be recognized in the
quarter.
Research and development (R&D) expenses were
$10.5 million for Q2 2024, compared to $7.4 million in Q2 2023. The
increase in R&D expenses was primarily driven by an increase in
spending for the KP1077 Phase 2 clinical trial and an increase in
personnel-related costs, partially offset by a decrease in
third-party costs related to arimoclomol.
Selling, general and administrative (SG&A)
expenses were $12.6 million for Q2 2024, compared to $6.6 million
in Q2 2023. The period-over-period increase reflects the commercial
team in place for the entire quarter and actively engaged in
activities to build awareness and provide patient services related
to OLPRUVA, leading to an increase in personnel costs due to the
additional headcount and an increase in other expenses related
primarily to the launch of OLPRUVA.
Net loss for Q2 2024 was ($19.9) million, or
($0.48) per basic and diluted share, compared to a net loss of
($2.6) million, or ($0.08) per basic and diluted share for Q2
2023.
As of June 30, 2024, total cash, cash
equivalents, and investments were $49.3 million, a decrease of $3.4
million compared to $52.7 million as of March 31, 2024. The
decrease was driven, in part, by increased third-party R&D
costs related to the KP1077 clinical development program and
increased SG&A expenses during the period as the Company
invested in its commercial infrastructure.
As of June 30, 2024, total shares of common
stock outstanding were 41,991,464, and fully diluted common shares
were 57,324,496, which included 5,483,537 shares issuable upon
exercise of warrants.
On April 5, 2024, the Company announced the
refinancing of its existing debt with up to $100 million in
committed capital, which strengthened its balance sheet, simplified
its debt structure, and provided non-dilutive capital flexibility.
The refinancing was led by Perceptive Advisors and HealthCare
Royalty Partners, premier biotech investors. From the initial draw
of $60 million at closing, the Company refinanced its existing debt
of $43 million and added an incremental $14 million in net cash
proceeds to the cash balance after fees and discounts. A second
tranche of up to $20 million is available at the Company’s
discretion until October 5, 2025, and a third tranche of up to $20
million will become available upon approval of arimoclomol, in each
case subject to certain terms and conditions.
On August 12, 2024, the Company completed an
underwritten public offering, further strengthening its balance
sheet. The underwriters fully exercised their overallotment option,
resulting in the issuance of approximately 10.6 million shares at a
price of $6.50 per share, raising net proceeds of $64.5 million
after fees and expenses. Combining the net proceeds from this
offering with our existing resources, pro forma June 30, 2024 total
cash, cash equivalents and investments was $113.8 million.
Following the closing of the underwritten public
offering on August 12, 2024, pro forma June 30, 2024, common shares
and fully diluted shares outstanding were 52,606,849 and
67,939,881, respectively. No warrants were issued as part of the
underwritten public offering.
Based on our current operating plan, available
cash, cash equivalents, and investments, including proceeds from
the underwritten offering closed in August, are expected to extend
our cash runway into the first quarter of 2027, subject to
continuing compliance with our debt covenants.
- Cash runway forecast includes:
revenue from the expected sales of OLPRUVA, ongoing reimbursements
from the French EAP for arimoclomol, ongoing royalties under the
AZSTARYS license agreement, and investments into the incremental
commercial activities needed to support the launch of arimoclomol,
if approved.
- Cash runway forecast does not
include commercial revenue from arimoclomol which could follow a
potential FDA approval or the potential sale of the Priority Review
Voucher which would be received upon approval.
Conference Call Information
Zevra will host a conference call and audio
webcast today at 4:30 p.m. ET, to discuss its corporate and
financial results for Q2 2024.
The audio webcast will be accessible via the
Investor Relations section of the Company’s website,
http://investors.zevra.com/. An archive of the audio webcast will
be available for ninety (90) days beginning at approximately 5:30
p.m. ET, on August 13, 2024.
Additionally, interested participants and investors may access
the conference call by dialing either:
- (800) 225-9448 (United States)
- +1 (203) 518- 9708 (International)
- Conference ID: ZVRAQ224
About Urea Cycle Disorders
UCDs are a group of rare, genetic disorders that
can cause harmful ammonia to build up in the blood, potentially
resulting in brain damage and neurocognitive impairments if ammonia
levels are not controlled.i Any increase in ammonia over time is
serious. Therefore, it is important to adhere to any dietary
protein restrictions and have alternative medication options to
help control ammonia levels.
About OLPRUVA®
OLPRUVA (sodium phenylbutyrate) was approved for
the treatment of certain UCDs in December 2022 and has recently
been marketed under the brand name, OLPRUVA®. OLPRUVA (sodium
phenylbutyrate) for oral suspension is a prescription medicine used
along with certain therapies, including changes in diet, for the
long-term management of adults and children weighing 44 pounds (20
kg) or greater and with a body surface area (BSA) of 1.2 m2 or
greater, with UCDs, involving deficiencies of carbamylphosphate
synthetase (CPS), ornithine transcarbamylase (OTC), or
argininosuccinic acid synthetase (AS). OLPRUVA is not used to treat
rapid increase of ammonia in the blood (acute hyperammonemia),
which can be life-threatening and requires emergency medical
treatment. For more information, please visit www.OLPRUVA.com.
Important Safety
Information
Certain medicines may increase the level of
ammonia in your blood or cause serious side effects when taken
during treatment with OLPRUVA. Tell your doctor about all the
medicines you or your child take, especially if you or your child
take corticosteroids, valproic acid, haloperidol, and/or
probenecid.
OLPRUVA can cause serious side effects,
including: 1) nervous system problems (neurotoxicity). Symptoms
include sleepiness, tiredness, lightheadedness, vomiting, nausea,
headache, confusion, 2) low potassium levels in your blood
(hypokalemia) and 3) conditions related to swelling (edema).
OLPRUVA contains salt (sodium), which can cause swelling from salt
and water retention. Tell your doctor right away if you or your
child get any of these symptoms. Your doctor may do certain blood
tests to check for side effects during treatment with OLPRUVA. If
you have certain medical conditions such as heart, liver or kidney
problems, are pregnant/planning to get pregnant or breast-feeding,
your doctor will decide if OLPRUVA is right for you.
The most common side effects of OLPRUVA include
absent or irregular menstrual periods, decreased appetite, body
odor, bad taste or avoiding foods you ate prior to getting sick
(taste aversion). These are not all of the possible side effects of
OLPRUVA. Call your doctor for medical advice about side effects.
You may report side effects to FDA at 1-800-FDA-1088.
About Niemann-Pick Disease Type C
(NPC)
Niemann-Pick disease type C (NPC) is an
ultra-rare, progressive, and neurodegenerative lysosomal storage
disorder characterized by an inability of the body to transport
cholesterol and other lipids within the cell, leading to an
accumulation of these substances in various tissue areas, including
brain tissue. The disease is caused by mutations in the NPC1 or
NPC2 genes, which are responsible for making lysosomal proteins.
Both children and adults can be affected by NPC with varying
clinical presentations. Those living with NPC lose independence due
to physical and cognitive limitations, with key neurological
impairments presenting in speech, cognition, swallowing,
ambulation, and fine motor skills. Disease progression is
irreversible and can be fatal within months or take years to be
diagnosed and advance in severity.
About Arimoclomol
Arimoclomol, Zevra’s orally-delivered,
investigational drug product candidate for the treatment of NPC,
has been granted Orphan Drug designation, Fast Track designation,
Breakthrough Therapy designation, and Rare Pediatric Disease
designation by the FDA, and Orphan Medicinal Product designation
for the treatment of NPC by the European Medicines Agency (EMA).
The FDA has accepted the resubmission of the NDA for arimoclomol
and has set a user fee action date (PDUFA date) of September 21,
2024.
About Idiopathic Hypersomnia
(IH)
Idiopathic hypersomnia (IH) is a rare sleep
disorder characterized by excessive daytime sleepiness (EDS).
Patients with IH experience daytime lapses into sleep, or an
irrepressible need to sleep that persists even with adequate or
prolonged nighttime sleep. Additionally, those with IH have extreme
difficulty waking, otherwise known as sleep inertia, severe brain
fog, and often fall asleep unintentionally or at inappropriate
times. These symptoms of IH often lead to further, even more
debilitating problems such as memory lapses, difficulty maintaining
focus, and depression.
It is estimated, based on claims data, that
approximately 37,000 patients in the United States are currently
diagnosed with IH, although the total patient population may be
much larger due to some patients who have not yet been diagnosed,
have been misdiagnosed, or are not currently seeking treatment.
About KP1077
KP1077 (serdexmethylphenidate, or SDX) is
Zevra’s proprietary prodrug of d-methylphenidate (d-MPH) and its
sole active pharmaceutical ingredient (API). KP1077 has been
granted Orphan Drug Designation by the FDA, and by the
European Commission, for the treatment of IH. The U.S. Drug
Enforcement Agency (DEA) has classified SDX, the sole API in
KP1077, as a Schedule IV controlled substance based on evidence
suggesting SDX has a lower potential for abuse when compared to
d-MPH, a Schedule II controlled substance. In addition, KP1077 has
intellectual property protection though 2037 and potentially
beyond.
About Celiprolol
Celiprolol is an investigational clinical
candidate for the treatment of Vascular Ehlers-Danlos Syndrome
(VEDS). Celiprolol has been granted Orphan Drug and
Breakthrough Therapy Designations by the FDA. Zevra recently
restarted enrollment in the Phase 3 trial, known as the DiSCOVER
trial being conducted under a Special Protocol Assessment (SPA)
agreement with the FDA. Celiprolol’s mechanism of action is
designed to reduce the mechanical stress on collagen fibers within
the arterial wall through vascular dilation and smooth muscle
relaxation.
About Vascular Ehlers-Danlos
Syndrome
Vascular Ehlers-Danlos syndrome is a rare
genetic cardiovascular disorder which impairs collagen 3 rich
connective tissue and leads to vascular and hollow organ
ruptures.
It is estimated that approximately 7,500
patients in the United States are currently diagnosed patients with
VEDS. There remains an unmet need with no approved treatment in the
United States. Celiprolol is currently the standard of care in
Europe.
About Zevra Therapeutics
Zevra Therapeutics is a rare disease company
combining science, data, and patient needs to create
transformational therapies for diseases with limited or no
treatment options. Our mission is to bring life-changing
therapeutics to people living with rare diseases. With unique,
data-driven development and commercialization strategies, the
Company is overcoming complex drug development challenges to make
new therapies available to the rare disease community.
For more information, please visit www.zevra.com
or follow us on X and LinkedIn.
Cautionary Note Concerning
Forward-Looking Statements
This press release may contain forward-looking
statements within the meaning of the Private Securities Litigation
Reform Act of 1995. Forward-looking statements include all
statements that do not relate solely to historical or current
facts, including without limitation statements regarding the
promise and potential impact of our preclinical or clinical trial
data; the initiation, timing and results of any clinical trials or
readouts, the content, information used for, timing or results of
any NDA submissions or resubmissions for arimoclomol or any other
product candidates for any specific disease indication or at any
dosage; the potential benefits of any of our products or product
candidates for any specific disease or at any dosage; the impact of
meetings or communications with the FDA or any advisory committee;
decisions by the FDA or any other entity for arimoclomol or any
other product candidates; our strategic and product development
objectives, including with respect to becoming a leading,
commercially focused rare disease company; the potential benefits
of our debt facility;, our our financial position, including our
cash balance: our corporate governance objectives; potential
revenues from our arimoclomol expanded access program; the
potential for royalty and milestone contributions, the presentation
of data at conferences; and the timing of any of the foregoing.
Forward-looking statements are based on information currently
available to Zevra and its current plans or expectations. They are
subject to several known and unknown uncertainties, risks, and
other important factors that may cause our actual results,
performance, or achievements to be materially different from any
future results, performance, or achievements expressed or implied
by the forward-looking statements. These and other important
factors are described in detail in the “Risk Factors” section of
Zevra’s Annual Report on Form 10-K for the year ended December
31, 2023, Zevra’s Quarterly Report on Form 10-Q for the three
months ended June 30, 2024, and Zevra’s other filings with
the Securities and Exchange Commission. While we may elect to
update such forward-looking statements at some point in the future,
except as required by law, we disclaim any obligation to do so,
even if subsequent events cause our views to change. Although we
believe the expectations reflected in such forward-looking
statements are reasonable, we cannot assure that such expectations
will prove correct. These forward-looking statements should not be
relied upon as representing our views as of any date after the date
of this press release.
________________________i Ah Mew N, et al. Urea
cycle disorders overview [updated June 22, 2017]. In: Adam MP,
Ardinger HH, Pagon RA, et al, eds. GeneReviews® [Internet].
University of Washington; 1993-2022. Accessed March 20, 2022.
Zevra Contact
Nichol Ochsner+1 (732) 754-2545nochsner@zevra.com
Russo Partners Contacts
Adanna G. Alexander, Ph.D.+1 (646)
942-5603adanna.alexander@russopartnersllc.com
|
ZEVRA THERAPEUTICS, INC. UNAUDITED
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
(in thousands, except share and per share
amounts) |
|
|
|
Three months endedJune 30, |
|
Six months endedJune 30, |
|
|
2024 |
|
2023 |
|
2024 |
|
2023 |
Revenue, net |
|
$ |
4,449 |
|
|
$ |
8,470 |
|
|
$ |
7,874 |
|
|
$ |
11,646 |
|
Cost of product revenue
(excluding $1,546 and $3,074 in intangible asset amortization for
the three and six months ended June 30, 2024, respectively shown
separately below) |
|
|
3,573 |
|
|
|
677 |
|
|
|
3,748 |
|
|
|
802 |
|
Intangible asset
amortization |
|
|
1,546 |
|
|
|
— |
|
|
|
3,074 |
|
|
|
— |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
|
10,521 |
|
|
|
7,433 |
|
|
|
22,798 |
|
|
|
16,088 |
|
Selling, general and administrative |
|
|
12,604 |
|
|
|
6,612 |
|
|
|
22,535 |
|
|
|
13,839 |
|
Total operating expenses |
|
|
23,125 |
|
|
|
14,045 |
|
|
|
45,333 |
|
|
|
29,927 |
|
Loss from operations |
|
|
(23,795 |
) |
|
|
(6,252 |
) |
|
|
(44,281 |
) |
|
|
(19,083 |
) |
Other income (expense): |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Interest expense |
|
|
(2,110 |
) |
|
|
(197 |
) |
|
|
(2,845 |
) |
|
|
(379 |
) |
Fair value adjustment related
to warrant and CVR liability |
|
|
5,779 |
|
|
|
2,118 |
|
|
|
9,406 |
|
|
|
575 |
|
Fair value adjustment related
to investments |
|
|
1 |
|
|
|
131 |
|
|
|
(26 |
) |
|
|
327 |
|
Interest and other income
(expense), net |
|
|
270 |
|
|
|
1,553 |
|
|
|
1,199 |
|
|
|
2,593 |
|
Total other income |
|
|
3,940 |
|
|
|
3,605 |
|
|
|
7,734 |
|
|
|
3,116 |
|
Loss before income taxes |
|
|
(19,855 |
) |
|
|
(2,647 |
) |
|
|
(36,547 |
) |
|
|
(15,967 |
) |
Income tax benefit |
|
|
(70 |
) |
|
|
74 |
|
|
|
— |
|
|
|
177 |
|
Net loss |
|
$ |
(19,925 |
) |
|
$ |
(2,573 |
) |
|
$ |
(36,547 |
) |
|
$ |
(15,790 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Basic and diluted net loss per
share of common stock: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss |
|
$ |
(0.48 |
) |
|
$ |
(0.08 |
) |
|
$ |
(0.87 |
) |
|
$ |
(0.46 |
) |
Weighted average number of
shares of common stock outstanding: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Basic and diluted |
|
|
41,899,087 |
|
|
|
33,898,233 |
|
|
|
41,839,582 |
|
|
|
34,180,818 |
|
|
ZEVRA THERAPEUTICS, INC. UNAUDITED
CONDENSED CONSOLIDATED BALANCE SHEETS (in
thousands, except share and par value amounts) |
|
|
|
June 30, |
|
December 31, |
|
|
2024 |
|
2023 |
Assets |
|
|
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
39,260 |
|
|
$ |
43,049 |
|
Securities at fair value |
|
|
9,998 |
|
|
|
24,688 |
|
Accounts and other receivables |
|
|
8,947 |
|
|
|
17,377 |
|
Prepaid expenses and other current assets |
|
|
2,686 |
|
|
|
1,824 |
|
Total current assets |
|
|
60,891 |
|
|
|
86,938 |
|
Inventories |
|
|
10,198 |
|
|
|
9,841 |
|
Property and equipment,
net |
|
|
678 |
|
|
|
736 |
|
Operating lease right-of-use
assets |
|
|
911 |
|
|
|
790 |
|
Goodwill |
|
|
4,701 |
|
|
|
4,701 |
|
Intangible assets, net |
|
|
66,154 |
|
|
|
69,227 |
|
Other long-term assets |
|
|
875 |
|
|
|
94 |
|
Total assets |
|
$ |
144,408 |
|
|
$ |
172,327 |
|
|
|
|
|
|
|
|
|
|
Liabilities and
stockholders' equity |
|
|
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
|
|
Accounts payable and accrued expenses |
|
$ |
20,452 |
|
|
$ |
28,403 |
|
Line of credit payable |
|
|
— |
|
|
|
37,700 |
|
Current portion of operating lease liabilities |
|
|
596 |
|
|
|
543 |
|
Current portion of discount and rebate liabilities |
|
|
6,768 |
|
|
|
4,550 |
|
Other current liabilities |
|
|
2,633 |
|
|
|
2,524 |
|
Total current liabilities |
|
|
30,449 |
|
|
|
73,720 |
|
Long-term debt |
|
|
58,328 |
|
|
|
5,066 |
|
Warrant liability |
|
|
7,856 |
|
|
|
16,100 |
|
Operating lease liabilities,
less current portion |
|
|
544 |
|
|
|
456 |
|
Discount and rebate
liabilities, less current portion |
|
|
8,115 |
|
|
|
7,663 |
|
Other long-term
liabilities |
|
|
6,638 |
|
|
|
7,458 |
|
Total liabilities |
|
|
111,930 |
|
|
|
110,463 |
|
|
|
|
|
|
|
|
|
|
Commitments and
contingencies |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Stockholders’ equity: |
|
|
|
|
|
|
|
|
Preferred stock: |
|
|
|
|
|
|
|
|
Undesignated preferred stock, $0.0001 par value, 10,000,000 shares
authorized, no shares issued or outstanding as of June 30, 2024, or
December 31, 2023 |
|
|
— |
|
|
|
— |
|
Common stock, $0.0001 par
value, 250,000,000 shares authorized, 43,567,156 shares issued and
41,991,464 shares outstanding as of June 30, 2024; 43,110,360
shares issued and 41,534,668 shares outstanding as of December 31,
2023 |
|
|
4 |
|
|
|
4 |
|
Additional paid-in
capital |
|
|
479,361 |
|
|
|
472,664 |
|
Treasury stock, at cost |
|
|
(10,983 |
) |
|
|
(10,983 |
) |
Accumulated deficit |
|
|
(436,325 |
) |
|
|
(399,778 |
) |
Accumulated other
comprehensive income (loss) |
|
|
421 |
|
|
|
(43 |
) |
Total stockholders' equity |
|
|
32,478 |
|
|
|
61,864 |
|
Total liabilities and
stockholders' equity |
|
$ |
144,408 |
|
|
$ |
172,327 |
|
Grafico Azioni Zevra Therapeutics (NASDAQ:ZVRA)
Storico
Da Nov 2024 a Dic 2024
Grafico Azioni Zevra Therapeutics (NASDAQ:ZVRA)
Storico
Da Dic 2023 a Dic 2024