Madrigal Pharmaceuticals Reports 2017 First Quarter Financial Results
- Phase 2 clinical studies of MGL-3196, a liver-directed thyroid hormone receptor (THR) beta selective agonist, are underway in patients with non-alcoholic steatohepatitis (NASH) and familial hypercholesterolemia (HeFH)
- Top-line results from these two trials, expected by year-end, have the potential to support the design and initiation of Phase 3 registration trials in 2018 -
“Enrollment is continuing as planned in our two Phase 2 proof-of-concept clinical trials of MGL-3196, for patients with NASH and HeFH,” said
“Because MGL-3196 selectively agonizes THR-β, it has the potential to safely address key pathological mechanisms responsible for the progression of liver injury and address the underlying causes of NASH,” said
Clinical Program Summaries for MGL-3196
NASH
Non-alcoholic Steatohepatitis (NASH) is a common liver disease in
In
In this trial, patients are randomized 2:1 to receive either MGL-3196 or placebo. The primary endpoint of the trial is the reduction of liver fat, assessed by MRI-PDFF at 12 weeks. Recent published data show a high correlation of reduction of liver fat measured by MRI-PDFF to NASH scoring on liver biopsy.
Efficacy will be confirmed at the end of the trial (36 weeks) by repeat MRI-PDFF and conventional liver biopsy to examine histological evidence for the resolution of NASH. Additional secondary endpoints include changes in clinically relevant biomarkers at 12 and 36 weeks, improvement in fibrosis by at least one stage, improvement of NASH, and safety and tolerability. Top-line results for the primary endpoint of the trial, the reduction of liver fat, assessed by MRI-PDFF at 12 weeks, are expected by year-end.
HeFH
Heterozygous familial hypercholesterolemia (HeFH) is a severe genetic dyslipidemia, typically caused by an inactivating mutation in one copy of the LDL receptor gene that leads to early onset cardiovascular disease. With conventional therapy, including statins and ezetimibe, the majority of HeFH and virtually all HoFH patients fail to reach their cholesterol (LDL-C) reduction goals. Based on evidence of impressive LDL cholesterol lowering in Phase 1, and data suggesting that MGL-3196 has a mechanism of action that is different from and complementary to statins, Madrigal initiated a Phase 2 proof-of-concept trial in HeFH. Top-line results of this trial are also expected by year-end.
The 12-week, randomized, double-blind, placebo-controlled, multi-center study will enroll up to 105 patients with HeFH randomized in a 2:1 ratio to receive either MGL-3196 or placebo, in addition to their current drug regimen (including high dose statins and ezetimibe). The primary endpoint of the study is reduction of LDL cholesterol, with secondary endpoints including reductions in triglycerides, Lp(a), and ApoB, as well as safety. Lp(a) is a severely atherogenic lipid particle, commonly elevated in familial hypercholesterolemia patients and poorly controlled by existing lipid lowering therapies. THR-β agonism is one of the few therapeutic approaches that can substantially lower Lp(a). As previously announced, the first patient in this study was dosed in
HoFH
Homozygous familial hypercholesterolemia (HoFH) is a much rarer form of severe genetic dyslipidemia, which results from inactivating mutations in both copies of the LDL receptor gene, and can produce cardiovascular disease before age 20. The protocol for a Phase 2, open-label study of MGL-3196 in HoFH is in development. The 12-week trial will have endpoints similar to the HeFH study and is expected to begin enrolling patients by the end of 2017.
Financial Results for the Three Months Ended
As of
Operating expenses were
Research and development expenses for the three month period ended
General and administrative expenses for the three month period ended
Interest income (expense), net, for the three month period ended
About
Forward-Looking Statements
This communication contains “forward-looking statements” made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements reflect management's current knowledge, assumptions, judgment and expectations regarding future performance or events. Although management believes that the expectations reflected in such statements are reasonable, they give no assurance that such expectations will prove to be correct and you should be aware that actual results could differ materially from those contained in the forward-looking statements. Forward-looking statements are subject to a number of risks and uncertainties including, but not limited to, the company's clinical development of MGL-3196, the timing and outcomes of clinical studies of MGL-3196, and the uncertainties inherent in clinical testing. Undue reliance should not be placed on forward-looking statements, which speak only as of the date they are made. Madrigal undertakes no obligation to update any forward looking statements to reflect new information, events or circumstances after the date they are made, or to reflect the occurrence of unanticipated events. Please refer to Madrigal's filings with the
(Tables Follow)
Madrigal Pharmaceuticals, Inc. | |||||||
Condensed Consolidated Statements of Operations | |||||||
(in thousands, except share and per share amounts) | |||||||
(unaudited) | |||||||
Three Months Ended | |||||||
March 31, | |||||||
2017 | 2016 | ||||||
Revenues: | |||||||
Total revenues | $ | - | $ | - | |||
Operating expenses: | |||||||
Research and development | 4,380 | 516 | |||||
General and administrative | 1,695 | 222 | |||||
Total operating expenses | 6,075 | 738 | |||||
Loss from operations | (6,075) | (738) | |||||
Interest income (expense), net | 76 | (975) | |||||
Net loss | $ | (5,999) | $ | (1,713) | |||
Basic and diluted net loss per common share | $ | (0.50) | $ | (9.72) | |||
Basic and diluted weighted average number of common shares outstanding | 11,955,739 | 176,158 | |||||
Madrigal Pharmaceuticals, Inc. | ||||||
Condensed Consolidated Balance Sheets | ||||||
(in thousands) | ||||||
(unaudited) | ||||||
March 31, | December 31, |
|||||
2017 | 2016 | |||||
Assets | ||||||
Cash, cash equivalents and marketable securities | $ | 40,127 | $ | 40,499 | ||
Other current assets | 225 | 708 | ||||
Other non-current assets | 5 | 3 | ||||
Total assets | $ | 40,357 | $ | 41,210 | ||
Liabilities and Equity | ||||||
Current liabilities | $ | 5,906 | $ | 4,800 | ||
Long-term liabilities | - | - | ||||
Stockholders’ equity | 34,451 | 36,410 | ||||
Total liabilities and stockholders’ equity | $ | 40,357 | $ | 41,210 | ||
Investor Contact:Marc Schneebaum ,Madrigal Pharmaceuticals, Inc. IR@madrigalpharma.com Media Contact:Mike Beyer ,Sam Brown Inc. mikebeyer@sambrown.com 312-961-2502