Madrigal Pharmaceuticals Reports 2017 Second Quarter Financial Results
- Completed patient enrollment in Phase 2 clinical study of MGL-3196, a liver-directed thyroid hormone receptor (THR) beta selective agonist, for treatment of NASH -
- Raised
“Completion of enrollment in our Phase 2 NASH clinical study is an important milestone, and enrollment is continuing as planned in our Phase 2 clinical study of MGL-3196 for patients with HeFH,” said
“Both NASH and HeFH are conditions with major unmet patient needs that we believe can be safely and effectively addressed by MGL-3196. We look forward to data readouts from these studies which, if positive, should enable us to initiate Phase 3 registration trials in 2018,” said
Clinical Program Summaries for MGL-3196
NASH
Non-alcoholic Steatohepatitis (NASH) is a common liver disease in
In
In this trial, patients were 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 homozygous familial hypercholesterolemia (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 or in early 2018.
The 12-week, randomized, double-blind, placebo-controlled, multi-center study is expected to 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.
Financial Results for the Three Months and Six Months Ended
As of
Operating expenses were
Research and development expenses for the three month and six month periods ended
General and administrative expenses for the three month and six month periods ended
Interest income (expense), net, for the three month and six month periods 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. Such statements contain words such as “expect,” “could,” “may,” “will,” “believe,” “estimate,” "continue," "future,”or the negative thereof or comparable terminology and the use of future dates. 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
Madrigal Pharmaceuticals, Inc. | |||||||||||||||
Condensed Consolidated Statements of Operations | |||||||||||||||
(in thousands, except share and per share amounts) | |||||||||||||||
(unaudited) | |||||||||||||||
Three Months Ended | Six Months Ended | ||||||||||||||
March 31, | June 30, | ||||||||||||||
2017 | 2016 | 2017 | 2016 | ||||||||||||
Revenues: | |||||||||||||||
Total revenues | $ | - | $ | - | $ | - | $ | - | |||||||
Operating expenses: | |||||||||||||||
Research and development | 6,816 | 2,088 | 11,196 | 2,604 | |||||||||||
General and administrative | 1,623 | 551 | 3,318 | 773 | |||||||||||
Total operating expenses | 8,439 | 2,639 | 14,514 | 3,377 | |||||||||||
Loss from operations | (8,439 | ) | (2,639 | ) | (14,514 | ) | (3,377 | ) | |||||||
Interest income (expense), net | 92 | (238 | ) | 168 | (1,213 | ) | |||||||||
Net loss | $ | (8,347 | ) | $ | (2,877 | ) | $ | (14,346 | ) | $ | (4,590 | ) | |||
Basic and diluted net loss per common share | $ | (0.69 | ) | $ | (16.33 | ) | $ | (1.20 | ) | $ | (26.06 | ) | |||
Basic and diluted weighted average number of common shares outstanding | 12,039,005 | 176,158 | 11,997,602 | 176,158 | |||||||||||
Madrigal Pharmaceuticals, Inc. | |||||||||||||||
Condensed Consolidated Balance Sheets | |||||||||||||||
(in thousands) | |||||||||||||||
(unaudited) | |||||||||||||||
June 30, | December 31, | ||||||||||||||
2017 | 2016 | ||||||||||||||
Assets | |||||||||||||||
Cash, cash equivalents and marketable securities | $ | 67,166 | $ | 40,500 | |||||||||||
Other current assets | 781 | 707 | |||||||||||||
Other non-current assets | 120 | 3 | |||||||||||||
Total assets | $ | 68,067 | $ | 41,210 | |||||||||||
Liabilities and Equity | |||||||||||||||
Current liabilities | $ | 6,436 | $ | 4,800 | |||||||||||
Long-term liabilities | - | - | |||||||||||||
Stockholders’ equity | 61,631 | 36,410 | |||||||||||||
Total liabilities and stockholders’ equity | $ | 68,067 | $ | 41,210 | |||||||||||
Investor Contact:Marc Schneebaum ,Madrigal Pharmaceuticals, Inc. [email protected] Media Contact:Kristin Paulina ,Sam Brown Inc. [email protected] 610-524-2959