Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company),
a commercial-stage company focused on addressing unmet needs for
the treatment of rare diseases, today reported its financial
results for the third quarter ended Sept. 30, 2024, and provided
corporate updates.
“The third quarter was one of the most exciting
and transformational periods in Zevra’s journey,” said
Neil F. McFarlane, Zevra’s President and Chief Executive
Officer. “After years of tireless effort, our team has
achieved a major milestone with FDA approval of
MIPLYFFA™ (arimoclomol), and we’re celebrating with the
Niemann-Pick disease type C (NPC) community. We intend to achieve
our goals with a high-performing team committed to execute, focus
and innovate to drive our continued growth and long-term
transformation.”
Program Highlights
- On Sept. 20, the U.S. Food and Drug
Administration (FDA) approved MIPLYFFA capsules as an orally
delivered treatment for NPC. MIPLYFFA, the first NPC drug approved
by the FDA, is indicated for use in combination with miglustat for
the treatment of neurological manifestations of NPC in adult and
pediatric patients 2 years of age and older. With the approval of
MIPLYFFA, Zevra received a rare pediatric disease Priority Review
Voucher (PRV) which the Company could monetize to help fund its
growth.
- As of Oct. 31, Zevra has received
90 prescription enrollment forms for MIPLYFFA of which 30% are
approved for reimbursement and are ready for fulfillment upon drug
availability, consistent with the Company’s prior guidance of eight
to 12 weeks post launch.
- Zevra has launched AmplifyAssist™,
a comprehensive support program for caregivers and those eligible
living with NPC or UCDs.
- OLPRUVA® had three new patient
enrollments in the third quarter, and reimbursement coverage is at
approximately 76% of covered lives. Improving OLPRUVA’s formulary
position is a primary objective to make it the preferred treatment
for UCDs.
- Zevra is refining its commercial
strategy for OLPRUVA to focus on specific patient segments where
there are fewer access barriers, and, together with The National
Urea Cycle Disorders Foundation, launched a targeted patient
education campaign highlighting the importance of prompt and
accurate testing of blood ammonia levels, which, when elevated, may
be a sign of a UCD. Campaign materials are available at
www.checkammonia.com.
- The Company recently completed an
End-of-Phase 2 meeting with the FDA for KP1077. The Agency
indicated that a single pivotal study with appropriate confirmatory
evidence will be sufficient to submit a new drug application (NDA).
Zevra is evaluating strategic alternatives to advance clinical
development and later, commercialization.
- The Phase 3 DiSCOVER trial of
celiprolol for the treatment of Vascular Ehler-Danlos Syndrome
(VEDS) is underway, and the Company has achieved its objective to
restart enrollment and to begin dosing new patients, reporting that
a total of 19 patients are now in the trial as of the end of the
third quarter. Zevra is continuing its efforts to expand its
partnerships with patient advocacy groups, treatment centers of
excellence, and medical key opinion leaders.
Corporate Highlights and Upcoming
Milestones
- In August, Zevra completed an
underwritten public offering of 10,615,385 shares of its common
stock at an offering price of $6.50 per share for total net
proceeds of approximately $64.5 million, after deducting
underwriting discounts and commissions and offering expenses.
Cantor and William Blair were joint book-running managers for the
offering. Citizens JMP and H.C. Wainwright & Co. were
co-managers.
- The Company completed a thorough
portfolio assessment and developed a strategic plan for 2025 that
builds from its 2024 accomplishments and furthers Zevra’s
transformation towards becoming a leading rare disease company with
a five-year vision to create value for patients and shareholders by
organizing our priorities on four key pillars: Commercial
Excellence, Pipeline and Innovation,
Talent and Culture, and Corporate
Foundation.
Now focused on late-stage clinical development and commercial
opportunities, Zevra has discontinued its in-house drug discovery
activities and is closing its laboratory facilities in Iowa and
Virginia. Future early research and development activities will be
outsourced.
Q3 2024 Financial
Highlights
- Revenue, Net: $3.7
million, comprised of $2.6 million in net reimbursements from the
French expanded access program (EAP) for arimoclomol, $1.1 million
of royalties and other reimbursements under the AZSTARYS® license
agreement, and de minimis OLPRUVA revenue.
- Cost of Goods
Sold: $2.3 million, which includes recognition of $2.0
million in inventory obsolescence reserve expense related to
OLPRUVA inventory that is nearing expiration.
- R&D Expenses:
$10.9 million, which represents a decrease of $1.4 million compared
to Q3 2023 due to the completion of the KP1077 phase 2 trial.
- SG&A Expenses:
$16.2 million for Q3 2024, compared to $5.8 million in Q3 2023,
which reflects the commercial team fully in place and actively
engaged in commercial launch activities and patient services
initiatives. This includes non-cash stock compensation of $6.1
million during the quarter, of which $2.5 million was
performance-based upon the approval of MIPLYFFA.
- Net Loss: ($33.2)
million, or ($0.69) per basic and diluted share for Q3 2024,
compared to a net loss of ($10.4) million, or ($0.30) per basic and
diluted share in Q3 2023.
- Cash Position:
Cash, cash equivalents and investments were $95.5 million as of
Sept. 30, 2024.
- Cash Runway
Forecast: Based on the Company’s current operating plan,
available cash, cash equivalents and investments are expected to
extend Zevra’s cash runway into 2027, subject to continuing
compliance with debt covenants. Cash runway forecast includes
anticipated revenue from MIPLYFFA sales, reimbursements from the
French EAP for arimoclomol, royalties under the AZSTARYS license
agreement, and continued investments into our development pipeline
programs. It does not include potential proceeds from a PRV
sale.
- Common and Fully Diluted
Shares O/S: As of Sept. 30, 2024, total shares of common
stock outstanding were 53,227,364, and fully diluted common shares
were 67,698,898, which included 5,483,537 shares issuable upon
exercise of warrants.
Upcoming Events
- Guggenheim Securities Healthcare
Innovation Conference, with fireside chat on Wednesday, Nov. 13,
2024, at 4:00 p.m. ET. Management will be available for one-on-one
meetings with registered attendees.
Conference Call Information -
UPDATED
Zevra will host a conference call and audio
webcast today at 4:30 p.m. ET to discuss its corporate update and
financial results for the third quarter of 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 Nov. 12, 2024.
Additionally, interested participants and
investors may access the conference call by dialing either:
- (785) 838-9345 (United States) --
updated dial-in
- +1 (203) 518-9814
(International)
- Conference ID: ZVRAQ324
About MIPLYFFA
MIPLYFFA (arimoclomol) increases the activation
of the transcription factors EB (TFEB) and E3 (TFE3) resulting in
the upregulation of coordinated lysosomal expression and regulation
(CLEAR) genes. MIPLYFFA has also been shown to reduce unesterified
cholesterol in the lysosomes of human NPC fibroblasts. The clinical
significance of these findings is not fully understood. MIPLYFFA
was granted Breakthrough Therapy designation, Rare Pediatric
Disease designation, Orphan Drug designation, and Fast Track
designation by the FDA for the treatment of NPC. MIPLYFFA was
further granted Orphan Medicinal Product designation by the
European Medicines Agency (EMA) for the treatment of NPC.
INDICATIONS AND USAGE
MIPLYFFA is indicated for use in combination
with miglustat for the treatment of neurological manifestations of
Niemann-Pick disease type C (NPC) in adult and pediatric patients 2
years of age and older.
IMPORTANT SAFETY
INFORMATION
Hypersensitivity Reactions:
Hypersensitivity reactions such as urticaria and
angioedema have been reported in patients treated with MIPLYFFA
during Trial 1: two patients reported both urticaria and angioedema
(6%) and one patient (3%) experienced urticaria alone within the
first two months of treatment. Discontinue MIPLYFFA in patients who
develop severe hypersensitivity reactions. If a mild or moderate
hypersensitivity reaction occurs, stop MIPLYFFA and treat promptly.
Monitor the patient until signs and symptoms resolve.
Embryofetal Toxicity:
MIPLYFFA may cause embryofetal harm when
administered during pregnancy based on findings from animal
reproduction studies. Advise pregnant females of the potential risk
to the fetus and consider pregnancy planning and prevention for
females of reproductive potential.
Increased Creatinine without Affecting
Glomerular Function:
Across clinical trials of MIPLYFFA, mean
increases in serum creatinine of 10% to 20% compared to baseline
were reported. These increases occurred mostly in the first month
of MIPLYFFA treatment and were not associated with changes in
glomerular function.
During MIPLYFFA treatment, use alternative
measures that are not based on creatinine to assess renal function.
Increases in creatinine reversed upon MIPLYFFA discontinuation.
The most common adverse
reactions in Trial 1 (≥15%) in MIPLYFFA-treated patients
who also received miglustat were upper respiratory tract infection,
diarrhea, and decreased weight.
Three (6%) of the MIPLYFFA-treated patients had
the following adverse reactions that led to withdrawal from Trial
1: increased serum creatinine (one patient), and progressive
urticaria and angioedema (two patients). Serious adverse reactions
reported in MIPLYFFA-treated patients were hypersensitivity
reactions including urticaria and angioedema.
To report SUSPECTED ADVERSE REACTIONS,
contact Zevra Therapeutics, Inc. at toll-free phone 1-844-600-2237
or FDA at 1 800-FDA-1088 or
www.fda.gov/medwatch.
Before prescribing MIPLYFFA,
please read the full Prescribing
Information, including Instructions for
Use.
Drug Interaction(s):
Arimoclomol is an inhibitor of the organic
cationic transporter 2 (OCT2) transporter and may increase the
exposure of drugs that are OCT2 substrates. When MIPLYFFA is used
concomitantly with OCT2 substrates, monitor for adverse reactions
and reduce the dosage of the OCT2 substrate.
Use in Females and Males of Reproductive
Potential:
Based on animal findings, MIPLYFFA may impair
fertility and may increase post-implantation loss and reduce
maternal, placental, and fetal weights.
Renal Impairment:
The recommended dosage of MIPLYFFA, in
combination with miglustat, in patients with an eGFR ≥15 mL/minute
to <50 mL/minute is lower than the recommended dosage (less
frequent dosing) in patients with normal renal function.
MIPLYFFA capsules for oral use are available in
the following strengths: 47 mg, 62 mg, 93 mg, and 124 mg.
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 KP1077
Serdexmethylphenidate (SDX) is Zevra’s
proprietary prodrug of d-methylphenidate (d-MPH) and is the sole
active pharmaceutical ingredient (API) of KP1077. KP1077 has been
granted Orphan Drug designation by the FDA, and by the
European Commission, for the treatment of Idiopathic Hypersomnia,
or IH. The U.S. Drug Enforcement Agency (DEA) has
classified SDX 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 through 2037.
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 Zevra Therapeutics,
Inc.
Zevra Therapeutics, Inc. is a commercial-stage
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.
Expanded access programs are made available by
Zevra Therapeutics, Inc. and its affiliates and are subject to the
Company's Expanded Access Program (EAP) policy, as published on its
website. Participation in these programs is subject to the laws and
regulations of each jurisdiction under which each respective
program is operated. Eligibility for participation in any such
program is at the treating physician's discretion.
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 any products or 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;
statements or decisions by the FDA or any other entity for any
products or product candidates; our ability to monetize any PRV;
future research and development activities; 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 financial position, including
our cash balance; our corporate governance objectives; potential
revenues from our arimoclomol expanded access program in France;
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 Dec. 31,
2023, Zevra’s Quarterly Report on Form 10-Q for the three months
ended Sept. 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.
Zevra Contact
Nichol Ochsner+1 (732) 754-2545nochsner@zevra.com
Russo Partners Contact
David Schull+1 (858)
717-2310david.schull@russopartnersllc.com
ZEVRA THERAPEUTICS, INC. UNAUDITED
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS(in
thousands, except share and per share amounts) |
|
|
|
Three months ended September 30, |
|
|
Nine months ended September 30, |
|
|
|
2024 |
|
|
2023 |
|
|
2024 |
|
|
2023 |
|
Revenue, net |
|
$ |
3,695 |
|
|
$ |
2,895 |
|
|
$ |
11,569 |
|
|
$ |
14,541 |
|
Cost of product revenue
(excluding $1,545 and $4,619 in intangible asset amortization for
the three and nine months ended September 30, 2024, respectively
shown separately below) |
|
|
2,303 |
|
|
|
144 |
|
|
|
6,051 |
|
|
|
946 |
|
Intangible asset
amortization |
|
|
1,545 |
|
|
|
— |
|
|
|
4,619 |
|
|
|
— |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
|
10,945 |
|
|
|
12,297 |
|
|
|
33,743 |
|
|
|
28,385 |
|
Selling, general and administrative |
|
|
16,208 |
|
|
|
5,818 |
|
|
|
38,743 |
|
|
|
19,657 |
|
Total operating expenses |
|
|
27,153 |
|
|
|
18,115 |
|
|
|
72,486 |
|
|
|
48,042 |
|
Loss from operations |
|
|
(27,306 |
) |
|
|
(15,364 |
) |
|
|
(71,587 |
) |
|
|
(34,447 |
) |
Other (expense) income: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Interest expense |
|
|
(2,312 |
) |
|
|
(366 |
) |
|
|
(5,157 |
) |
|
|
(745 |
) |
Fair value adjustment related to warrant and CVR liability |
|
|
(4,746 |
) |
|
|
3,678 |
|
|
|
4,660 |
|
|
|
4,253 |
|
Fair value adjustment related to investments |
|
|
90 |
|
|
|
124 |
|
|
|
64 |
|
|
|
451 |
|
Interest and other income (expense), net |
|
|
1,049 |
|
|
|
1,738 |
|
|
|
2,248 |
|
|
|
4,331 |
|
Total other (expense) income |
|
|
(5,919 |
) |
|
|
5,174 |
|
|
|
1,815 |
|
|
|
8,290 |
|
Loss before income taxes |
|
|
(33,225 |
) |
|
|
(10,190 |
) |
|
|
(69,772 |
) |
|
|
(26,157 |
) |
Income tax expense |
|
|
— |
|
|
|
(177 |
) |
|
|
— |
|
|
|
— |
|
Net loss |
|
$ |
(33,225 |
) |
|
$ |
(10,367 |
) |
|
$ |
(69,772 |
) |
|
$ |
(26,157 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Basic and diluted net loss per
share of common stock: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss |
|
$ |
(0.69 |
) |
|
$ |
(0.30 |
) |
|
$ |
(1.59 |
) |
|
$ |
(0.76 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Weighted average number of
shares of common stock outstanding: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Basic and diluted |
|
|
47,808,817 |
|
|
|
34,724,614 |
|
|
|
43,843,851 |
|
|
|
34,364,075 |
|
ZEVRA THERAPEUTICS, INC. UNAUDITED
CONDENSED CONSOLIDATED BALANCE SHEETS (in
thousands, except share and par value amounts) |
|
|
|
September 30, |
|
|
December 31, |
|
|
|
2024 |
|
|
2023 |
|
Assets |
|
|
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
54,039 |
|
|
$ |
43,049 |
|
Securities at fair value, current |
|
|
35,337 |
|
|
|
24,688 |
|
Accounts and other receivables |
|
|
7,790 |
|
|
|
17,377 |
|
Prepaid expenses and other current assets |
|
|
2,276 |
|
|
|
1,824 |
|
Total current assets |
|
|
99,442 |
|
|
|
86,938 |
|
Inventories |
|
|
8,756 |
|
|
|
9,841 |
|
Securities at fair value,
long-term |
|
|
6,105 |
|
|
|
— |
|
Property and equipment,
net |
|
|
607 |
|
|
|
736 |
|
Operating lease right-of-use
assets |
|
|
820 |
|
|
|
790 |
|
Goodwill |
|
|
4,701 |
|
|
|
4,701 |
|
Intangible assets, net |
|
|
70,608 |
|
|
|
69,227 |
|
Other long-term assets |
|
|
512 |
|
|
|
94 |
|
Total assets |
|
$ |
191,551 |
|
|
$ |
172,327 |
|
|
|
|
|
|
|
|
|
|
Liabilities and
stockholders' equity |
|
|
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
|
|
Accounts payable and accrued expenses |
|
$ |
24,464 |
|
|
$ |
28,403 |
|
Line of credit payable |
|
|
— |
|
|
|
37,700 |
|
Current portion of operating lease liabilities |
|
|
540 |
|
|
|
543 |
|
Current portion of discount and rebate liabilities |
|
|
8,547 |
|
|
|
4,550 |
|
Other current liabilities |
|
|
930 |
|
|
|
2,524 |
|
Total current liabilities |
|
|
34,481 |
|
|
|
73,720 |
|
Long-term debt |
|
|
58,904 |
|
|
|
5,066 |
|
Warrant liability |
|
|
13,902 |
|
|
|
16,100 |
|
Operating lease liabilities,
less current portion |
|
|
483 |
|
|
|
456 |
|
Discount and rebate
liabilities, less current portion |
|
|
8,490 |
|
|
|
7,663 |
|
Other long-term
liabilities |
|
|
5,521 |
|
|
|
7,458 |
|
Total liabilities |
|
|
121,781 |
|
|
|
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 September 30,
2024, or December 31, 2023 |
|
|
— |
|
|
|
— |
|
Common stock, $0.0001 par
value, 250,000,000 shares authorized, 54,803,056 shares issued and
53,227,364 shares outstanding as of September 30, 2024; 43,110,360
shares issued and 41,534,668 shares outstanding as of December 31,
2023 |
|
|
5 |
|
|
|
4 |
|
Additional paid-in
capital |
|
|
550,413 |
|
|
|
472,664 |
|
Treasury stock, at cost |
|
|
(10,983 |
) |
|
|
(10,983 |
) |
Accumulated deficit |
|
|
(469,550 |
) |
|
|
(399,778 |
) |
Accumulated other
comprehensive income (loss) |
|
|
(115 |
) |
|
|
(43 |
) |
Total stockholders' equity |
|
|
69,770 |
|
|
|
61,864 |
|
Total liabilities and
stockholders' equity |
|
$ |
191,551 |
|
|
$ |
172,327 |
|
Zevra Therapeutics (NASDAQ:ZVRA)
Gráfica de Acción Histórica
De Nov 2024 a Dic 2024
Zevra Therapeutics (NASDAQ:ZVRA)
Gráfica de Acción Histórica
De Dic 2023 a Dic 2024