FDA interactions on Barth syndrome and Leber’s hereditary optic neuropathy expected by mid-year
- Completed initial public offering with gross proceeds of $85.1 million
- Dosed first patient in Phase 2b study of elamipretide in patients with dry AMD with geographic atrophy
- FDA interactions on Barth syndrome and Leber’s hereditary optic neuropathy expected by mid-year
- On track for top-line Phase 3 primary mitochondrial myopathy read-out by year end
BOSTON, April 4, 2019 /PRNewswire/ -- Stealth BioTherapeutics Corp (NASDAQ: MITO), a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today reported financial results for the year ended December 31, 2018 and announced recent business highlights.
“In 2018, we executed across multiple clinical, preclinical and regulatory milestones, augmenting our leading role in the development of mitochondrial medicine. We made significant strides toward enrolling our pivotal study in primary mitochondrial myopathy; announced data from our trials in dry AMD, Barth syndrome and Leber’s hereditary optic neuropathy; initiated IND-enabling studies for the first of our new pipeline compounds; and secured Orphan Drug designation for elamipretide for the treatment of Barth syndrome and Leber’s hereditary optic neuropathy and Fast Track designation for elamipretide for the treatment of dry AMD with geographic atrophy,” said Reenie McCarthy, Chief Executive Officer at Stealth. “We’re continuing the momentum into 2019, with the first patient now enrolled in our Phase 2b study in dry AMD with geographic atrophy, key regulatory interactions for Barth syndrome and Leber’s hereditary optic neuropathy on our near-term horizon, and anticipated read-out of our pivotal primary mitochondrial myopathy trial by year end. The completion of our initial public offering earlier this year provides us with the financial strength to execute on our key clinical trials, data read-outs and regulatory interactions in the months ahead.”
Fourth Quarter 2018 and Recent Highlights
- Dosed first patient in Phase 2b study of elamipretide in patients with dry age-related macular degeneration (dry AMD) with geographic atrophy. ReCLAIM-2, a Phase 2b, randomized, double-masked, placebo-controlled clinical study, will evaluate the safety and efficacy of subcutaneous injections of elamipretide in 180 patients with dry AMD with geographic atrophy. Full enrollment is targeted by early 2020.
- Completed initial public offering (IPO). We completed our IPO of 7,088,232 American Depositary Shares (ADSs) at a public offering price of $12.00 per ADS, which includes a partial exercise of the underwriters’ over-allotment option. The total gross proceeds, before deducting underwriting discounts and commissions and other offering expenses, were approximately $85.1 million.
- Granted Fast Track designation for elamipretide for the treatment of dry AMD with geographic atrophy in November 2018. The FDA’s Fast Track program facilitates the development and review of drugs to treat serious conditions with unmet medical needs. Elamipretide has also been granted Fast Track designation for the treatment of primary mitochondrial myopathy, Barth syndrome and Leber’s hereditary optic neuropathy, three rare primary mitochondrial diseases.
Key Upcoming Milestones
- Meet with FDA in the first half of 2019 to discuss regulatory path for elamipretide in Barth’s syndrome
- Mid-year meeting with FDA to discuss regulatory path for elamipretide in Leber’s hereditary optic neuropathy
- Report top-line data from Phase 3 clinical trial in primary mitochondrial myopathy by the end of 2019
- Initiate Phase 1 clinical trial of new pipeline candidate, SBT-272, by the end of 2019
2018 Financial Results
Research and Development (R&D) Expenses: R&D expenses were $53.1 million for fiscal year 2018, compared to $63.2 million for fiscal year 2017. This decrease was primarily due to a $10.4 million decrease in clinical trial costs, as our cardiovascular clinical trials ended during early 2018, offset by an increase in costs of approximately $1.3 million for our primary mitochondrial myopathy studies, for which we incurred expenses for a full year in 2018.
General and Administrative (G&A) Expenses: G&A expenses were $22.2 million for fiscal year 2018, compared to $16.5 million for fiscal year 2017. The increase in administrative expenses was primarily attributable to an increase of $5.1 million in financing costs and an increase in legal intellectual property costs of $0.6 million.
Net Loss: Net loss for the year ended December 31, 2018 was $96.7 million, or $1.41 basic and diluted net loss per ordinary share, as compared to $82.9 million for the year ended December 31, 2017, or $1.21 basic and diluted net loss per ordinary share.
Cash Position: Cash and cash equivalents were $10.9 million at December 31, 2018, compared to $4.1 million at December 31, 2017. In February and March 2019, Stealth raised $85.1 million of gross proceeds from its initial public offering.
About Stealth
We are a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction. Mitochondria, found in nearly every cell in the body, are the body’s main source of energy production and are critical for normal organ function. Dysfunctional mitochondria characterize a number of rare genetic diseases, collectively known as primary mitochondrial diseases, and are also involved in many common age-related diseases. We believe our lead product candidate, elamipretide, has the potential to treat both rare genetic and common age-related mitochondrial diseases. We are studying elamipretide in the following primary mitochondrial diseases: primary mitochondrial myopathy, Barth syndrome and Leber’s hereditary optic neuropathy. We are also studying elamipretide in dry AMD. Our other pipeline candidates include SBT-272, which we are evaluating for rare neurodegenerative disease indications, and SBT-20, which we are evaluating for rare peripheral neuropathies. We have optimized our discovery platform to identify novel mitochondrial-targeted compounds, which may be nominated as therapeutic product candidates or utilized as scaffolds to deliver other compounds to mitochondria. We have assembled a highly experienced management team, board of directors and group of scientific advisors to help us achieve our mission of leading mitochondrial medicine.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding Stealth BioTherapeutics’ plans, strategies and expectations for its preclinical and clinical advancement of its drug development programs, including its ongoing clinical trials of elamipretide and planned clinical trial of SBT-272; the potential benefits of Stealth BioTherapeutics’ product candidates; its key milestones for 2019; its plans regarding future data presentations; and its financial guidance regarding the period in which it will have capital available to fund its operations. Statements that are not historical facts, including statements about Stealth BioTherapeutics’ beliefs, plans and expectations, are forward-looking statements. The words “anticipate,” “expect,” “hope,” “plan,” “potential,” “possible,” “will,” “believe,” “estimate,” “intend,” “may,” “predict,” “project,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Stealth BioTherapeutics may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements as a result of known and unknown risks, uncertainties and other important factors, including: Stealth BioTherapeutics’ ability to obtain additional funding; the ability to successfully demonstrate the efficacy and safety of Stealth BioTherapeutics’ product candidates and future product candidates; the preclinical and clinical results for Stealth BioTherapeutics’ product candidates, which may not support further development and marketing approval; the potential advantages of Stealth BioTherapeutics’ product candidates; the content and timing of decisions made by the U.S. FDA, the EMA or other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies, which may affect the initiation, timing and progress of preclinical studies and clinical trials of Stealth BioTherapeutics product candidates; Stealth BioTherapeutics’ ability to obtain and maintain requisite regulatory approvals and to enroll patients in its planned clinical trials; unplanned cash requirements and expenditures; competitive factors; Stealth BioTherapeutics’ ability to obtain, maintain and enforce patent and other intellectual property protection for any product candidates it is developing; and general economic and market conditions. These and other risks are described in greater detail under the caption “Risk Factors” included in Stealth BioTherapeutics’ most recent Annual Report on Form 20-F filed with the Securities and Exchange Commission (“SEC”), as well as in any future filings with the SEC. Forward-looking statements represent management’s current expectations and are inherently uncertain. Except as required by law, Stealth BioTherapeutics does not undertake any obligation to update forward-looking statements made by us to reflect subsequent events or circumstances.
Media Relations
dna Communications
Kate Contreras, 617-520-7088
Media@StealthBT.com
Investor Relations
Stern Investor Relations
Rachel Frank, 212-362-1200
IR@StealthBT.com
| STEALTH BIOTHERAPEUTICS CORP | ||||
| Consolidated Statements of Operations | ||||
| (in thousands, except share and per share data) | ||||
| Year Ended December 31, | ||||
| 2018 | 2017 | |||
| Operating expenses: | ||||
| Research and development | $ 53,062 | $ 63,220 | ||
| General and administrative | 22,217 | 16,500 | ||
| Total operating expenses | 75,279 | 79,720 | ||
| Loss from operations | (75,279) | (79,720) | ||
| Other income (expense), net | (21,433) | (3,190) | ||
| Net loss attributable to ordinary shareholders | $ (96,712) | $ (82,910) | ||
| Net loss per share attributable to ordinary shareholders | $ (1.41) | $ (1.21) | ||
| Weighted average ordinary shares used in net loss per | 68,476,149 | 68,472,262 | ||
| STEALTH BIOTHERAPEUTICS CORP | ||||
| Consolidated Balance Sheets | ||||
| (in thousands) | ||||
| December 31, | ||||
| 2018 | 2017 | |||
| Assets | ||||
| Current assets: | ||||
| Cash and cash equivalents | $ 10,855 | $ 4,119 | ||
| Prepaid expenses and other current assets | 2,438 | 1,801 | ||
| Total current assets | 13,293 | 5,920 | ||
| Property and equipment, net | 499 | 796 | ||
| Deferred offering costs | 1,325 | — | ||
| Other non-current assets | 406 | 439 | ||
| Total assets | $ 15,523 | $ 7,155 | ||
| Liabilities, convertible preferred shares and shareholders’ | ||||
| Current liabilities: | ||||
| Accounts payable | $ 11,023 | $ 7,455 | ||
| Accrued expenses and other current liabilities | 13,826 | 12,520 | ||
| Accrued interest payable | 7,297 | 2,526 | ||
| Current portion of long-term debt | 8,465 | 2,094 | ||
| Total current liabilities | 40,611 | 24,595 | ||
| Convertible notes payable | 103,257 | 50,000 | ||
| Long-term debt, less current portion | 10,317 | 11,957 | ||
| Derivative liability | 36,568 | — | ||
| Warrant liability | 100 | 512 | ||
| Total liabilities | 190,853 | 87,064 | ||
| Series A convertible preferred shares | 211,377 | 211,377 | ||
| Total shareholders’ deficit | (386,707) | (291,286) | ||
| Total liabilities, convertible preferred shares and | $ 15,523 | $ 7,155 | ||
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SOURCE Stealth BioTherapeutics
Company Codes: NASDAQ-NMS:MITO
