Gemcabene reduced LDL-C by 20% and hsCRP
by 53% when added to moderate intensity statin therapy
Gemphire Therapeutics Inc. (NASDAQ:GEMP), a clinical-stage
biopharmaceutical company focused on developing and commercializing
therapies for cardiometabolic disorders, including dyslipidemia and
NASH, today announced the presentation of pre-clinical and clinical
study results, its financial results for the three and nine month
periods ended September 30, 2017, and provided a corporate update.
The study results were featured in poster presentations Sunday
November 12th and today at the American Heart Association® (AHA) in
Anaheim, California.
“The third quarter of 2017 was marked by great progress in
advancing gemcabene in dyslipidemia and NASH,” said Steven Gullans,
Ph.D., interim CEO of Gemphire. “ROYAL-1 was the second of two
recent Phase 2b studies to successfully meet its primary endpoint,
and new data from ROYAL-1 presented at the American Heart
Association meeting have given us further insights into gemcabene’s
benefits to patients. We are now preparing for end of Phase 2
meetings for the hypercholesterolemia indications with the FDA and
EMA and anticipate that these will take place early in 2018, with
the goal of reaching agreement on the overall design of the planned
Phase 3 development program. We are also moving forward in NASH,
and plan to begin a Phase 2 trial before year end 2017.”
American Heart Association Scientific Sessions
2017
- Final results for ROYAL-1 Clinical Data: Gemcabene
Add-on Therapy to High- and Moderate-Intensity Statin Stratums in
Hypercholesterolemic Patients • Greater
effects were observed in a cardiometabolic population, patients
with mixed dyslipidemia, who have a particularly high atherogenic
particle burden. In the mixed dyslipidemia group of patients,
gemcabene 600 mg demonstrated a placebo adjusted LDL-C reduction of
23% (p < 0.05). • Consistent with the
gemcabene’s mechanism of action, patients with mixed dyslipidemia
showed greater placebo adjusted reductions in non-HDL-C by 19%,
ApoB by 26%, ApoE by 34% and triglycerides (TGs) by 33%.
• Gemcabene treatment was associated with a significant
median reduction in high-sensitivity C-reactive protein (hsCRP) of
40%, compared to 6% for those treated by placebo. The reductions in
hsCRP were 53% and 33% for those on moderate- and high-intensity
statins, respectively, vs 6% for placebo. hsCRP is a plasma protein
that serves as a biomarker for inflammation and there is growing
acceptance that reducing hsCRP as a marker of reduced inflammation
is correlated with reductions in major adverse cardiovascular
events (MACE). • Patients with NASH have many
overlapping co-morbidities with cardiometabolic patients.
Gemcabene lowers high levels of LDL-C, triglyceride, and hsCRP
levels that are associated with NASH. NASH patients have
significant liver fat deposits which produce a plethora of CVD risk
factors, leaving NASH patients with a much higher risk of
cardiovascular events which are the number one cause of death in
this population. Gemcabene’s ability to significantly clear VLDL
and greatly reduce inflammation (hsCRP), may play a key role in
easing the cardiometabolic burden on both the liver and heart.
- Poster Session on MOA at the Heart Meeting:
• Titled “An Orally Administered Small Molecule
that Inhibits Hepatic Sulfatase-2 Expression In Vivo: A Novel
Strategy to Correct Diabetic Dyslipoproteinemia with Implications
for Residual Atherosclerotic Cardiovascular Disease (ASCVD) Risk,”
was presented by Charles L. Bisgaier, Daniela C. Oniciu and Kevin
J. Williams. • Sulfatase-2 enzyme (SULF2) has been
identified as a key biologic target in ASCVD. Obesity and
type 2 diabetes (T2DM) cause hepatic overexpression of SULF2.
SULF2 inhibits hepatic disposal of cholesterol- and
triglyceride-rich remnant ApoB-lipoproteins (C-TRLs) also known as
atherogenic remnant lipoproteins in human T2DM.
• Treatment with gemcabene lowered hepatic SULF2 and
ApoC-III expression. These reductions in SULF2 and ApoC-III mRNA
levels correlated with reductions in plasma triglycerides.
• Gemcabene may enhance clearance of C-TRLs via
downregulation of hepatic SULF2, independent of LDLR mRNA.
To view the posters, please refer to the Events and
Presentations section of the company website.
Third Quarter and Recent Corporate
Highlights
- August: Announced topline ROYAL-1 Phase 2b
data, • Gemcabene achieved the primary
endpoint for a significant LDL-C reduction in hypercholesterolemic
patients on stable high/moderate intensity statin and/or ezetimibe
therapy.
- September: Presented new COBALT-1 Clinical Data at the
2017 FH Global Summit, • COBALT-1
achieved its primary endpoint, showing a statistically significant
reduction in LDL-C compared to baseline at 12 weeks
(p=0.0035). • Gemcabene is efficacious in both
Homozygous Familial Hypercholesterolemia (HoFH) and Heterozygous
Familial Hypercholesterolemia (HeFH) patients. LDL-C decreased by
30% overall and 38% in HeFH patients with gemcabene 600 mg.
• Validated MOA by lowering LDL-C in HoFH patients with
null/null LDL receptors (no LDL receptor activity).
• Gemcabene 600 mg was well tolerated by statin
intolerant patients and reductions in LDL-C of up to 51% were
observed.
- October: Presented poster at The Liver Meeting®, the
annual meeting of the Association for the Study of Liver Diseases
(AASLD), • Data supported clinical
evaluation of gemcabene as a potential treatment for
NAFLD/NASH. • Clinical trial in NAFLD/NASH is
planned to begin in the fourth quarter of 2017.
Upcoming 2017 and 2018 Clinical Milestones
- Gemphire is preparing for end of Phase 2 meetings with both
the FDA and EMA, anticipated to take place in early
2018. The primary focus of these meetings will be to reach
agreement on the design of the Phase 3 development programs for
gemcabene in hypercholesterolemia indications.
- Top-line results from the INDIGO-1 Phase 2b trial in severe
hypertriglyceridemia (SHTG) are targeted for the second quarter of
2018 based on the current pace of enrollment and the unexpected
impact of hurricanes Harvey, Irma and Maria on many of the clinical
sites.
- A Phase 2 clinical development program in NASH is targeted to
begin in the fourth quarter of 2017 with top-line results targeted
for second half of 2018.
Third Quarter 2017 Financial Update and
Guidance
The primary drivers of the increase in G&A expenses over the
prior year third quarter and nine-month periods were increased
infrastructure costs to support the ongoing clinical trials and
public company requirements, focused primarily in personnel costs,
including non-cash equity compensation expense, professional
services and separation expenses related to our previous CEO
recorded in the second quarter of 2017.
The increase in R&D expense was primarily attributable to
increased clinical trial activities encompassing two separate Phase
2b and four separate Phase 1 clinical trials ongoing in the current
year period versus minimal expenses related to the initiation of
one clinical study in the prior year period.
Cash used in operations in the nine months ended September 30,
2017 was $20.1 million compared to $7.4 million for the nine months
ended September 30, 2016.
In July, the Company entered into a term loan agreement for up
to $15.0 million with Silicon Valley Bank, subject to funding in
several tranches, and immediately drew $10.0 million to extend the
cash runway for the ongoing development program for gemcabene. The
remaining $5.0 million under the term loan may be drawn, at the
Company’s option, subject to the achievement of certain
pre-clinical and clinical milestones, through July 31, 2018.
Management expects operating expenses and cash used in operating
activities to continue to trend above 2016 levels, primarily in
research and development, as the Company funds its ongoing clinical
trials and initiates the NASH clinical program. Based on the
Company’s current operating plans, management believes existing
cash, along with anticipated future proceeds from the July 2017
term loan agreement, is sufficient to fund operations through
completion of our remaining dyslipidemia trial as well as
completion of the planned NASH clinical trial anticipated to be
completed in the second half of 2018 and will support the work to
develop our Phase 3 study plans.
Gemcabene’s mechanism of action and safety profile are
highly differentiated from other clinical candidates
Gemphire’s product candidate gemcabene is a first-in-class,
once-daily, oral therapy that may be suitable for patients who are
unable to achieve normal levels of LDL-C or triglycerides with
currently approved therapies, primarily statins. Gemcabene's
mechanism of action (MOA) is designed to enhance the clearance of
very low-density lipoproteins (VLDLs) in the plasma and inhibition
of the production of cholesterol and triglycerides in the
liver. The combined effect of these mechanisms has been
clinically observed to result in a reduction of plasma non-HDL-C,
VLDL-C, LDL-C, apolipoprotein B and triglycerides. In
addition, gemcabene has been shown to markedly lower C-reactive
protein in humans and improve insulin sensitization.
Gemcabene’s MOA is liver-directed involving downregulation of
hepatic apolipoprotein C-III (apoC-III) mRNA expression and
decrease of plasma apoC-III levels. Gemcabene also reduces
acetyl-CoA carboxylase (ACC1) and CCR2/CCR5 receptor mRNA levels,
markers involved in the progression of non-alcoholic
steatohepatitis (NASH)/non-alcoholic fatty liver disease
(NAFLD). Gemcabene has demonstrated proof of concept efficacy
for NASH in the rodent STAM™ model developed at SMC Laboratories in
Tokyo, Japan. Gemcabene has been tested as monotherapy and in
combination with statins and other drugs in 956 subjects across 20
Phase 1 and Phase 2 clinical trials. Given this profile of efficacy
across multiple pathological pathways, as well as evidence of
safety and tolerability, particularly when used as an add-on to
many other therapeutic drugs, gemcabene has attributes that support
studies in humans for NASH.
About Gemphire
Gemphire is a clinical-stage biopharmaceutical company that is
committed to helping patients with cardiometabolic disorders,
including dyslipidemia and NASH. The Company is focused on
providing new treatment options for cardiometabolic diseases
through its complementary, convenient, cost-effective product
candidate gemcabene as add-on to the standard of care, especially
statins, that will benefit patients, physicians, and payors.
Gemphire has initiated 3 clinical trials for homozygous familial
hypercholesterolemia (HoFH), heterozygous familial
hypercholesterolemia (HeFH)/atherosclerotic cardiovascular disease
(ASCVD), and severe hypertriglyceridemia (SHTG) under NCT02722408,
NCT02634151, and NCT02944383, respectively, with a fourth planned
trial in NASH to initiate in the fourth quarter of 2017.
Please visit www.gemphire.com for more information.
Forward Looking Statements
Any statements in this press release about Gemphire’s future
expectations, plans and prospects, including statements about
Gemphire’s financial prospects, future operations and sufficiency
of funds for future operations, clinical development of Gemphire’s
product candidate, expectations regarding future clinical trials,
regulatory submissions and meetings and future expectations and
plans and prospects for Gemphire, expectations regarding operating
expenses and cash used in operations, and other statements
containing the words "believes," "anticipates," "estimates,"
"expects," "intends," "plans," "predicts," "projects," "targets,"
"may," "potential," "will," "would," "could," "should," "continue,"
“scheduled” and similar expressions, constitute forward-looking
statements within the meaning of The Private Securities Litigation
Reform Act of 1995. Actual results may differ materially from
those indicated by such forward-looking statements as a result of
various important factors, including: the success and timing of
Gemphire’s regulatory submissions and pre-clinical and clinical
trials; regulatory requirements or developments; changes to
Gemphire’s clinical trial designs and regulatory pathways; changes
in Gemphire’s capital resource requirements; Gemphire’s ability to
obtain additional financing; Gemphire’s ability to successfully
market and distribute its product candidate, if approved;
Gemphire’s ability to obtain and maintain its intellectual property
protection; and other factors discussed in the "Risk Factors"
section of Gemphire’s Annual Report on Form 10-K for the year ended
December 31, 2016, Gemphire’s Quarterly Report on Form 10-Q for the
quarter ended September 30, 2017 and in other filings Gemphire
makes with the SEC from time to time. In addition, the
forward-looking statements included in this press release represent
Gemphire’s views as of the date hereof. Gemphire anticipates
that subsequent events and developments will cause Gemphire’s views
to change. However, while Gemphire may elect to update these
forward-looking statements at some point in the future, Gemphire
specifically disclaims any obligation to do so. These
forward-looking statements should not be relied upon as
representing Gemphire’s views as of any date subsequent to the date
hereof.
Contact:Andrew McDonald, Ph.D.LifeSci Advisors,
LLC(646) 597-6987
Jeff Mathiesen, CFOGemphire Therapeutics Inc.(734)-245-1700
|
Gemphire Therapeutics Inc. |
Balance Sheet Data |
(in thousands) |
|
|
|
|
|
|
|
|
|
September 30, |
|
|
December 31, |
|
|
2017 |
|
|
2016 |
|
|
|
|
|
|
|
|
|
(unaudited) |
|
|
|
|
|
|
|
|
|
|
|
Cash and cash
equivalents |
|
$ |
25,340 |
|
|
$ |
24,033 |
|
Total assets |
|
|
26,211 |
|
|
|
24,754 |
|
Accounts payable and
accrued liabilities |
|
|
6,295 |
|
|
|
4,121 |
|
Term loan |
|
|
9,964 |
|
|
|
— |
|
Total liabilities |
|
|
16,262 |
|
|
|
4,122 |
|
Common stock |
|
|
18 |
|
|
|
17 |
|
Additional paid–in
capital |
|
|
63,659 |
|
|
|
47,674 |
|
Accumulated
deficit |
|
|
(53,728) |
|
|
|
(27,059) |
|
Total stockholders’
equity |
|
|
9,949 |
|
|
|
20,632 |
|
|
|
|
|
|
|
|
|
|
Condensed Statements of Comprehensive
Loss |
(in thousands, except per share
amounts) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
For the Three MonthsEnded |
|
For the Nine MonthsEnded |
|
|
September 30, |
|
September 30, |
|
|
2017 |
|
2016 |
|
2017 |
|
2016 |
|
|
(unaudited) |
|
(unaudited) |
Operating
expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
General and
administrative |
|
$ |
2,050 |
|
$ |
1,466 |
|
$ |
8,951 |
|
$ |
3,567 |
Research and
development |
|
|
6,489 |
|
|
1,936 |
|
|
17,606 |
|
|
3,901 |
Total operating
expenses |
|
|
8,539 |
|
|
3,402 |
|
|
26,557 |
|
|
7,468 |
Loss from
operations |
|
|
(8,539) |
|
|
(3,402) |
|
|
(26,557) |
|
|
(7,468) |
Interest and other
income (expense), net |
|
|
(132) |
|
|
(476) |
|
|
(112) |
|
|
96 |
Net loss |
|
|
(8,671) |
|
|
(3,878) |
|
|
(26,669) |
|
|
(7,372) |
Adjustment to
redemption value on Series A convertible preferred stock |
|
|
— |
|
|
(67) |
|
|
— |
|
|
(366) |
Net loss attributable
to common stockholders |
|
$ |
(8,671) |
|
$ |
(3,945) |
|
$ |
(26,669) |
|
$ |
(7,738) |
Net loss per
share: |
|
|
|
|
|
|
|
|
|
|
|
|
Basic and diluted |
|
$ |
(0.82) |
|
$ |
(0.56) |
|
$ |
(2.60) |
|
$ |
(1.65) |
Number of shares used
in per share calculations: |
|
|
|
|
|
|
|
|
|
|
|
|
Basic and diluted |
|
|
10,624 |
|
|
6,984 |
|
|
10,253 |
|
|
4,704 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
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