Mirna Therapeutics, Inc. (Nasdaq:MIRN), a clinical stage
biopharmaceutical company developing a pipeline of microRNA-based
oncology therapeutics, today reported financial results for the
second quarter of 2016 and provided an update on recent
developments.
MRX34 CLINICAL PROGRAM AND SECOND QUARTER UPDATE
- Presented clinical data of MRX34
at ASCO 2016. In an oral presentation, investigators
reported on the final dose-escalation results from the
first-in-human Phase 1 trial of MRX34, highlighting the safety
profile, pharmacodynamic evidence of activity, and multiple
clinical responses in cancer patients with a variety of advanced
solid tumors.
Data highlights included four confirmed partial responses for up
to 50+ weeks in duration in patients with late-stage, metastatic
hepatocellular carcinoma cancer or HCC (liver cancer), renal cell
carcinoma or RCC (kidney cancer) and acral melanoma (a rare and
difficult-to-treat form of skin cancer). Stable disease was also
observed in an additional 15 patients for more than four cycles
(approximately three months) of therapy, including a small cell
lung cancer (SCLC) patient who showed stable disease for more than
one year on MRX34 as fourth line therapy.
As of the ASCO presentation, three patients had experienced
possible immune-mediated serious adverse events (SAEs) after
receiving MRX34. These previously reported events included
enterocolitis, systemic inflammatory response syndrome, and
pneumonitis/colitis. The first two patients recovered; the patient
experiencing pneumonitis/colitis subsequently died.
- Clinical Update. In early
August, a recently enrolled acral melanoma patient experienced an
SAE of increased ALT and AST liver function tests, determined
likely to be due to acute hepatitis, with subsequent liver failure
leading to death. The event was deemed possibly related to MRX34
and reported to the FDA and Korean regulatory authority.
The timing and pattern of response to treatment with MRX34 and
the associated safety profile suggest a potential immune component
to MRX34 activity.
- Plan to continue the expansion phase
of the ongoing Phase 1 study. Enrollment efforts are focused on
patients with cancer types where confirmed responses have been
observed as well as others where miR-34 has shown biological
relevance. The Company had planned to initiate Phase 2 trials in
RCC and melanoma by the end of 2016. However, it is now planned to
let the results from the Phase 1 expansion cohorts and the
translational medicine study guide the next steps in development of
MRX34.
- Preparing to launch translational
medicine trial in late 2016. This study is intended to develop
deeper insights into the mechanism of action of MRX34 in metastatic
melanoma patients and identify potential biomarkers of drug
activity and treatment response. The study is planned to include
serial tumor biopsies as well as liquid biopsies for cell-free DNA
and exosomal RNA analysis.
- Preclinical studies underway,
evaluating potential of combination regimens to enhance
effectiveness of standard cancer therapies. Mirna researchers
presented in vitro findings at the American Association for Cancer
Research (AACR) 2016 annual meeting demonstrating the synergistic
anticancer effects between MRX34 and platinum and other commonly
used cytotoxic chemotherapy drugs across a range of non-small cell
lung cancer (NSCLC) cell lines. Synergistic anticancer effects were
also shown between MRX34 and tyrosine kinase inhibitors, suggesting
the potential for combination therapies that include MRX34 with
other standard of care cancer therapeutics.
CORPORATE UPDATES
- Appointed Dr. Vincent J. O'Neill as
the Company’s Chief Medical Officer. Dr. O'Neill, a medical
oncologist, joined Mirna with 15 years of therapeutic and
diagnostic product development experience, most recently serving as
Chief Medical Officer at Exosome Diagnostics. Previous roles
included senior leadership positions at Sanofi, Genentech and
GlaxoSmithKline.
- Appointed Dr. Perry Nisen to the
Board of Directors. Dr. Nisen is currently the Chief Executive
Officer and Donald Bren Chief Executive Chair of the Sanford
Burnham Prebys Medical Discovery Institute. Previously, he served
as Senior Vice President of Science and Innovation at
GlaxoSmithKline (GSK), where he was integral to the discovery,
development, and commercialization of a vast portfolio of oncology
drugs. Earlier roles included Senior Vice President and Oncology
Therapy Area Head at GSK and Divisional Vice President of Cancer
Research and Oncology Development at Abbott Laboratories.
SECOND QUARTER 2016 FINANCIAL RESULTS
- Cash Position and Guidance:
Cash, cash equivalents, and marketable securities
totaled $72.6 million as of June 30, 2016, compared
to $89.7 million as of December 31, 2015. The
Company has no debt. Based on the current operating plan, the
Company expects that current cash resources will be sufficient to
meet operating requirements into 2018.
- Research and development
expenses: Research and development expenses in the second
quarter of 2016 were $3.7 million and $8.2 million, respectively,
for the three and six months ended June 30, 2016, compared to $4.5
million and $7.9 million during the comparable periods in 2015. The
decrease for the three months ended June 30, 2016 compared to the
same period in 2015 was primarily attributable to higher costs
associated with our Phase 1 clinical trial for our lead product
candidate MRX34, specifically adding additional sites and upfront
drug costs which were incurred in 2015. This decrease in 2016 was
partially offset by an increase in employee compensation, benefits
and stock compensation.
The increase for the six months ended June 30, 2016 is primarily
attributable to higher compensation, benefits and stock
compensation expense due to a higher headcount. The increase was
largely offset by higher costs associated with our Phase 1 trial in
the prior year.
- General and Administrative
Expenses: General and administrative expenses in the second
quarter of 2016 were $2.0 million and $4.2 million, respectively,
for the three and six months ended June 30, 2016, compared to $1.2
million and $2.1 million during the comparable periods in 2015. The
increase in general and administrative expenses was primarily
attributable to increased employee compensation expense due to a
higher headcount and higher outside professional and consulting
costs, the majority of which were costs to comply with public
company operating and reporting requirements.
- Net Loss: Net loss was
approximately $5.6 million for the second quarter of 2016 and $12.2
million for the six months ended June 30, 2016, compared to a net
loss of $5.7 million and $10.0 million for the comparable periods
in 2015. The results included non-cash, stock-based related
compensation charges of $243,000 and $689,000 for the second
quarter and six months ended June 30, 2016 and $217,000 and
$351,000 for the comparable periods in 2015.
About Mirna Therapeutics, Inc.
Mirna is a clinical stage biopharmaceutical company developing a
pipeline of microRNA-based oncology therapeutics and is the first
to bring a synthetic microRNA mimic into clinical development for
the treatment of cancer. Mirna's lead product candidate, MRX34, a
mimic of naturally occurring microRNA-34 (miR-34), is being studied
in a Phase 1 clinical trial which has included patients with
primary liver cancer, advanced solid tumors and hematological
malignancies. miR-34 is one of the most widely published microRNAs
and is considered a key regulator of multiple oncogenes across key
oncogenic pathways, with the capacity to regulate more than 30
different oncogenes and repress immune checkpoint signaling
molecules, including PD-L1. The potential capacity to
simultaneously affect multiple pathways and processes that are
critical to cancer cell viability may make mimics of tumor
suppressor microRNAs an important new class of anti-cancer agents.
Mirna plans to develop MRX34 as a monotherapy and in combination
with other therapies. The Company was founded in 2007 and is
located in Austin, Texas.
For more information, visit www.mirnarx.com.
Forward-Looking Statements
To the extent that statements contained in this press release
are not descriptions of historical facts regarding Mirna, they are
forward-looking statements reflecting the current beliefs and
expectations of management made pursuant to the safe harbor
provisions of the Private Securities Litigation Reform Act of 1995,
including statements regarding our plans for MRX34 development; our
plans to let the results from the Phase 1 expansion cohorts and the
translational medicine study guide the next steps in development of
MRX34; our plans to continue the expansion phase of the ongoing
Phase 1 study; our plans to launch a translational medicine trial
in late 2016; and our expectation that current cash resources will
be sufficient to meet operating requirements into 2018. Such
forward-looking statements involve substantial risks and
uncertainties that could cause our clinical development program,
future results, performance or achievements to differ significantly
from those expressed or implied by the forward-looking statements.
Such risks and uncertainties include, among others, the
uncertainties inherent in the clinical drug development process,
including the outcomes of clinical trials, the regulatory approval
process, our substantial dependence on MRX34, our commercialization
plans and efforts and other matters that could affect the
availability or commercial potential of our product candidates and
the risk that our product candidates may cause undesirable side
effects or have other properties that could delay or prevent their
regulatory approval. We undertake no obligation to update or revise
any forward-looking statements. For a further description of the
risks and uncertainties that could cause actual results to differ
from those expressed in these forward-looking statements, as well
as risks relating to our business in general, see our Annual Report
on Form 10-K filed with the U.S. Securities and Exchange Commission
(SEC) on March 30, 2016 and our Quarterly Report on Form 10-Q,
expected to be filed with the SEC on or about August 15, 2016.
Mirna Therapeutics, Inc.
Condensed Balance Sheets
(in thousands, except share
data)
June 30, December 31,
2016 2015 (unaudited) Assets Current
Assets: Cash and cash equivalents $ 30,796 $ 89,713 Marketable
securities 41,756 — Grant reimbursement and other receivables — —
Prepaid expenses and other current assets 947 829
Total current assets 73,499 90,542 Property and equipment, net
1,237 375 Restricted cash 2,430 — Total assets $
77,166 $ 90,917
Liabilities and Stockholders’ Equity
(Deficit) Current Liabilities: Accounts payable $ 1,462 $ 3,687
Accrued expenses 2,193 2,214 Total liabilities 3,655
5,901 Commitments and contingencies
Stockholders’ Equity
(Deficit): Preferred stock, $0.001 par value, 5,000,000 shares
authorized at June 30, 2016 and December 31, 2015; 0 shares
outstanding at June 30, 2016 and December 31, 2015 — — Common
stock, $0.001 par value; 250,000,000 shares authorized at June 30,
2016 and December 31, 2015; 20,835,868 and 20,830,555 shares issued
and outstanding at June 30, 2016 and December 31, 2015,
respectively 21 21 Additional paid in capital 162,216 161,518
Accumulated other comprehensive income 6 — Accumulated deficit
(88,732) (76,523) Total stockholders’ equity
73,511 85,016 Total liabilities and stockholders’ equity $
77,166 $ 90,917
Mirna Therapeutics, Inc.
Condensed Statements of Operations and
Comprehensive Loss (unaudited)
(in thousands)
Three Months Ended Six Months Ended June
30, June 30, 2016 2015 2016
2015 Operating expenses: Research and development $
3,682 $ 4,499 $ 8,205 $ 7,901 General and administrative
2,049 1,185 4,179 2,062 Total operating
expenses 5,731 5,684 12,384 9,963 Other income: Interest income
93 — 175 — Total other income 93
— 175 — Net loss $ (5,638) $ (5,684) $
(12,209) $ (9,963) Less: Accretion and dividends on convertible
preferred stock — (1,544) — (2,662) Net
loss attributable to common stockholders $ (5,638) $ (7,228) $
(12,209) $ (12,625)
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InvestorsThe Trout GroupPete Rahmer,
646-378-2973prahmer@troutgroup.comorMediaBMC
CommunicationsAmy Bonanno,
646-513-3117abonanno@bmccommunications.com
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