Zevra Therapeutics Reports Corporate Updates and First Quarter 2023 Financial Results
Zevra Therapeutics Reports Corporate Updates and First Quarter 2023 Financial Results
May 15, 2023 at 4:05 PM EDT
Conference call and live audio webcast with slide presentation scheduled for today,
“Zevra is moving at full speed as a company strategically focused on developing and commercializing novel therapeutics designed to address the unmet needs of people with rare diseases,” said
Recent Business and Corporate Highlights:
- Rebranded Zevra Therapeutics, reinforcing the Company's strategic transformation into a commercially focused rare disease company.
- Continued advancement of arimoclomol toward resubmission of its New Drug Application (NDA) to the
U.S. Food and Drug Administration (FDA) anticipated as early as Q3 2023:- Safety and efficacy data from the four-year open-label extension of the Phase 2/3 clinical trial of arimoclomol in NPC were publicly announced and will be included as part of the updated arimoclomol NDA.
- Safety and efficacy data from the four-year open-label extension of the Phase 2/3 clinical trial of arimoclomol in NPC were publicly announced and will be included as part of the updated arimoclomol NDA.
- Continued advancement of a Phase 2 clinical trial evaluating KP1077 as a treatment for IH:
- Interim efficacy and safety data are expected as early as Q3 2023, with the potential for topline Phase 2 data as early as year-end 2023.
- Data gathered from the Phase 2 trial in IH will support the advancement of KP1077 into a pivotal Phase 3 trial in patients with IH, as well as inform the decision of whether to initiate a future Phase 3 trial in patients with narcolepsy.
- Expanded the clinical program for KP1077 by opening an IND for narcolepsy, extending its potential to address multiple rare sleep disorders.
- Strong balance sheet, with
$95.3 million in cash, cash equivalents, and investments as ofMarch 31, 2023 , which supports our operating cash runway into 2026. - Announced Board and Chief Executive Officer (CEO) changes:
John B. Bode ,Douglas E. Calder ,Wendy Dixon andCorey Watton joined the Board of Directors at the 2023 Annual Meeting of Stockholders.Tamara A. Favorito was unanimously appointed Chair of the Board of Directors.Richard W. Pascoe resigned from his role as CEO, effectiveJune 1, 2023 .Christal Mickle , Co-Founder and Chief Development Officer, appointed to serve as interim CEO and President effectiveJune 1, 2023 .- The Board will begin a search for both a new CEO and replacement Board members for
Matthew Plooster andJoseph B. Saluri who have announced their intention to not stand for re-election at the Company’s 2024 Annual Meeting of Stockholders, and to retire from the Board as soon as replacements are found.
Overview of Q1 2023 Financial Results:
Net revenue for Q1 2023 was
Research and development (R&D) expenses were
General and administrative (G&A) expenses were
Net loss attributable to common stockholders for Q1 2023 was
During Q1 2023, the Company repurchased 665,739 shares of the Company’s common stock for approximately
As of
Based on the Company’s current operating forecast, existing cash, cash equivalents, and investments are expected to be sufficient to continue operations into 2026. The operating forecast includes the remaining activities required to resubmit the arimoclomol NDA, funding of the commercial preparation for the potential launch of arimoclomol in the
As of
Conference Call Information:
Zevra will host a conference call and live audio webcast with a slide presentation today at
The audio webcast with a slide presentation will be accessible via the Investor Relations section of the Company’s website, http://investors.zevra.com/. An archive of the webcast and presentation will be available for 90 days beginning at approximately
Additionally, interested participants and investors may access the conference call by dialing either:
- (800) 267-6316 (
U.S. ) - +1 (203) 518-9783 (International)
- Conference ID: ZVRAQ123
About Zevra:
Zevra Therapeutics is a rare disease company melding science, data, and patient need to create transformational therapies for diseases with limited or no treatment options. With unique, data-driven clinical, regulatory, and commercialization strategies, the Company is overcoming complex drug development challenges to bring much-needed therapies to patients.
Arimoclomol, Zevra’s orally-delivered, first-in-class investigational product candidate for the treatment of Niemann-Pick disease type C (NPC), has been granted orphan drug designation, Fast Track designation, Breakthrough Therapy designation and rare pediatric disease designation for NPC by the
KP1077 is Zevra’s lead clinical candidate being developed to treat idiopathic hypersomnia (IH) and narcolepsy. KP1077 is comprised solely of serdexmethylphenidate (SDX), Zevra’s proprietary prodrug of d-methylphenidate (“d-MPH”). The FDA has granted KP1077 orphan drug designation for the treatment of IH, and the
Early access programs are made available by
Caution 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, and which can be identified by the use of words such as "may," "will," "expect," "project," "estimate," "anticipate," "plan," "believe," "potential," "should," "continue," "could," "intend," "target," "predict," or the negative versions of those words or other comparable words or expressions, although not all forward-looking statements contain these identifying words or expressions. Forward-looking statements are not guarantees of future actions or performance. These forward-looking statements include without limitation statements regarding senior leadership and board member transitions and refreshment, or the timing thereof, our strategic and product development objectives, the promise and potential impact of our preclinical or clinical trial data, including without limitation the initiation, timing and results of any clinical trials or readouts, the content, timing or results of any Investigational New Drug (“IND”) applications and New Drug Application (“NDA”) submissions or resubmissions for arimoclomol, KP1077, or any other product candidates for any specific disease indication or at any dosage, the potential achievement of commercial sales or revenue milestones for AZSTARYS and the timing thereof, the sufficiency of our cash, cash equivalents and investments to fund our operating activities for any specific period of time, expected net revenue from the French EAP program, and our strategic and product development objectives, including with respect to becoming a leading, commercially-focused rare disease company. 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 (formerly
Contacts:
Nichol Ochsner
+1 (732) 754-2545
nochsner@zevra.com
Jennifer Arcure
+1 (917) 603-0681
Jennifer.arcure@evokegroup.com
| ZEVRA THERAPEUTICS, INC. | ||||||||
| UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS | ||||||||
| (in thousands, except share and per share amounts) | ||||||||
| Three Months Ended |
||||||||
| 2023 | 2022 | |||||||
| Revenue, net | $ | 2,879 | $ | 3,965 | ||||
| Operating expenses: | ||||||||
| Cost of revenue | 125 | 8 | ||||||
| Research and development | 8,844 | 3,082 | ||||||
| General and administrative | 6,834 | 2,734 | ||||||
| Total operating expenses: | 15,803 | 5,824 | ||||||
| (Loss) income from operations | (12,924 | ) | (1,859 | ) | ||||
| Other (expense) income: | ||||||||
| Interest expense | (182 | ) | (5 | ) | ||||
| Fair value adjustment related to derivative and warrant liability | (2 | ) | 241 | |||||
| Fair value adjustment related to investments | 196 | (352 | ) | |||||
| Interest and other income, net | 1,042 | 107 | ||||||
| Total other income (expense): | 1,054 | (9 | ) | |||||
| Loss before income taxes: | (11,870 | ) | (1,868 | ) | ||||
| Income tax benefit | 103 | 4 | ||||||
| Net loss: | (11,767 | ) | (1,864 | ) | ||||
| Net loss attributable to common stockholders: | $ | (11,767 | ) | $ | (1,864 | ) | ||
| Basic and diluted net loss per share of common stock: | ||||||||
| Net loss attributable to common stockholders | $ | (0.34 | ) | $ | (0.05 | ) | ||
| Weighted average number of shares of common stock outstanding: | ||||||||
| Basic and diluted | 34,466,542 | 34,506,597 | ||||||
| ZEVRA THERAPEUTICS, INC. | ||||||||
| UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS | ||||||||
| (in thousands, except share and par value amounts) | ||||||||
| 2023 | 2022 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 40,181 | $ | 65,466 | ||||
| Securities at fair value | 34,403 | 16,900 | ||||||
| Short-term investments - other | 20,700 | 481 | ||||||
| Accounts and other receivables | 7,822 | 8,299 | ||||||
| Prepaid expenses and other current assets | 1,174 | 1,877 | ||||||
| Total current assets: | 104,280 | 93,023 | ||||||
| Inventories | 620 | 671 | ||||||
| Property and equipment, net | 744 | 794 | ||||||
| Operating lease right-of-use assets | 898 | 988 | ||||||
| Long-term investments - other | — | 20,000 | ||||||
| Other long-term assets | 53 | 53 | ||||||
| Total assets: | $ | 106,595 | $ | 115,529 | ||||
| Liabilities and stockholders' equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable and accrued expenses | $ | 10,098 | $ | 6,169 | ||||
| Current portion of operating lease liabilities | 470 | 480 | ||||||
| Current portion of discount and rebate liabilities | 4,746 | 4,655 | ||||||
| Other current liabilities | 302 | 422 | ||||||
| Total current liabilities: | 15,616 | 11,726 | ||||||
| Line of credit payable | 12,914 | 12,800 | ||||||
| Derivative and warrant liability | 3 | 1 | ||||||
| Operating lease liabilities, less current portion | 736 | 843 | ||||||
| Discount and rebate liabilities, less current portion | 5,764 | 4,327 | ||||||
| Other long-term liabilities | 158 | 25 | ||||||
| Total liabilities: | 35,191 | 29,722 | ||||||
| Commitments and contingencies | ||||||||
| Stockholders’ equity: | ||||||||
| Preferred stock: | ||||||||
| Undesignated preferred stock, |
— | — | ||||||
| Common stock, |
3 | 3 | ||||||
| Additional paid-in capital | 402,786 | 401,799 | ||||||
| (10,983 | ) | (7,536 | ) | |||||
| Accumulated deficit | (320,339 | ) | (308,572 | ) | ||||
| Accumulated other comprehensive (loss) income | (63 | ) | 113 | |||||
| Total stockholders' equity: | 71,404 | 85,807 | ||||||
| Total liabilities and stockholders' equity: | $ | 106,595 | $ | 115,529 | ||||
