Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN)
today reported financial results for the three months ended March
31, 2017. For the first quarter of 2017, the Company reported a net
loss of $20.2 million or $0.15 per share, compared with a net loss
of $18.1 million or $0.13 per share for the first quarter of 2016.
Cash, cash equivalents, marketable securities and interest
receivable as of March 31, 2017 were $386.6 million.
“During the first quarter of 2017, we advanced our
first, orally administered factor D inhibitor, ACH-4471, into Phase
2 development for PNH. As we continue to enroll patients into this
study, the first two PNH patients have now completed 28 days of
dosing. After assessment of safety and efficacy by the
investigators both patients continue to receive up to two
additional months of therapy. Our plan is to submit interim results
from this study for presentation at a major medical conference this
quarter,” commented Milind Deshpande, Ph.D., President and CEO of
Achillion.
Dr. Deshpande further stated, “With Achillion’s
robust balance sheet, the ongoing worldwide HCV collaboration with
Janssen, who has fully enrolled patients into their Phase 2b
OMEGA-1 clinical trial, and the emerging results from our
complement factor D inhibitor program, I believe Achillion is well
positioned to achieve its goal of advancing novel therapies that
can address significant unmet needs for patients around the
globe.”
First Quarter 2017 Results
For the first quarter of 2017, the Company reported
a net loss of $20.2 million, or $0.15 per share, compared with a
net loss of $18.1 million, or $0.13 per share for the first quarter
of 2016. Cash, cash equivalents, marketable securities, and
interest receivable as of March 31, 2017 were $386.6 million.
Research and development expenses were $15.5
million in the first quarter of 2017, compared with $13.3 million
for the same period of 2016. The increase was primarily due to
increased clinical trial and consulting costs related to
ACH-4471combined with increased preclinical and manufacturing costs
related to the Company’s next generation factor D inhibitor
compounds.
For the three months ended March 31, 2017, general
and administrative expenses totaled $5.7 million, compared to $5.4
million for the same period in 2016, with the increase primarily
due to increased corporate legal fees and consulting fees.
Non-cash stock compensation expense totaled $3.2
million for the first quarter of 2017 as compared to $3.0 million
for the first quarter of 2016, and is included in research and
development and general and administrative expenses.
Status of Complement Factor D Inhibitor
Program: Developing ACH-4471 for Rare Diseases
Dr. Deshpande further commented, “As our complement
factor D inhibitor platform continues to grow, with nearly 2,000
small molecule compounds synthesized to date, and our broad IP
strategy, we have continued to make significant advancements in
complement biology and research, all of which supports our ongoing
clinical development program evaluating factor D inhibition by
ACH-4471 for the potential treatment of PNH and C3G.”
Developing ACH-4471 Complement Factor D
Inhibitor for Rare Diseases
- PNH (Paroxysmal Nocturnal Hemoglobinuria)In
April 2017, Achillion announced the initiation of a phase 2 trial
with ACH-4471 for patients with untreated PNH. The primary
objective of the study is to assess the change-from-baseline in
serum lactate dehydrogenase (LDH) levels, a sensitive biomarker for
intravascular hemolysis, during 28 days of dosing. The protocol
allows for intra-patient dose-escalation with patients initially
receiving 100 mg three times daily of ACH-4471 with the ability to
increase dosage during the treatment period. Secondary endpoints
being assessed include changes in hemoglobin and red blood cell
levels, complement pathway biomarkers, such as Bb and factor D,
levels, pharmacokinetics, and safety.Enrollment of patients into
this trial in ongoing. Two patients have completed 28 days of
dosing and each continues to receive longer term treatment, for up
to an additional two months with ACH-4471, under the protocol.
Interim results are anticipated during the second quarter.PNH is a
rare, acquired, life-threatening disease characterized by
destruction of red blood cells (hemolytic anemia), blood clots
(thrombosis), impaired bone marrow function, and a risk of
developing leukemia. Preclinical studies suggest ACH-4471 has a
distinct mechanism of action inhibiting factor D within the
alternative pathway of the complement cascade leading to blockade
of C3 convertase production. Furthermore, unlike C5 inhibitors,
ACH-4471 is also thought to prevent C3 fragment deposition on PNH
cells and may confer a pharmacological advantage by protecting PNH
cells from both intravascular and extravascular hemolysis.
- C3G (C3 Glomerulopathy)In February 2017,
Achillion announced that it has entered into an agreement with
Imperial College London to conduct a natural history study of C3
glomerulopathy (C3G), a rare renal disorder which includes dense
deposit disease (DDD) and C3 glomerulonephritis (C3GN). This study,
conducted by a team of researchers led by Dr. Matthew Pickering and
Dr. H. Terry Cook, both of Imperial College, tracks the course of a
disease over time. The aim of such studies is to collect data on
disease progression which can inform and support product
development and approval.C3G is a rare renal disease which is
believed to be the result of over-activity of the alternative
pathway. As ACH-4471 has been shown in vitro to inhibit alternative
pathway activity, potentially decreasing the formation of C3
protein fragments, the company plans to initiate a phase 2 study of
ACH-4471 in C3G patients during the second half of 2017.There is
currently no cure available for C3G, no approved treatment to
prevent disease progression and a poor prognosis for patients, of
whom approximately 30-50% require dialysis or transplant 10 years
after diagnosis.
Update on world-wide collaboration with
Janssen for HCV
In May 2015, Achillion announced an exclusive
worldwide collaboration with Janssen Pharmaceuticals, Inc.
(Janssen), one of the Janssen Pharmaceutical Companies of Johnson
& Johnson, for the treatment of chronic hepatitis C viral
infection (HCV).
Update of HCV Clinical Program
- ‘604 Study’ Phase 2 randomized, open-label study to
evaluate the safety, pharmacokinetics and efficacy of the
combination of AL-335, odalasvir (ACH-3102), and simeprevir in
treatment-naïve subjects with genotype 1 chronic hepatitis
CThe Janssen-sponsored phase 2a clinical trial evaluating
all-oral regimens for durations of eight weeks and less remains
ongoing, with additional results presented in April 2017. This
trial supports the evaluation of a triplet regimen, JNJ-4178,
consisting of odalasvir, AL-335, and simeprevir, for durations of
eight and six weeks Initiated in October 2015, this phase 2a
clinical trial demonstrated JNJ-4178 was highly effective in
treatment-naïve patients with HCV genotype 1 infection, achieving
100% sustained viral response 24 weeks after the completion of
treatment, or SVR24, for treatment durations of both 6 and 8 weeks.
Janssen is continuing to evaluate the short-duration triple regimen
for the treatment of HCV genotypes 1, 2, 4, 5, or 6.
- OMEGA-1 Phase 2b, multicenter, randomized, open-label
study to investigate the efficacy, safety and pharmacokinetics of
different treatment regimens of AL-335, odalasvir, and simeprevir
in treatment-naive and treatment-experienced subjects with chronic
hepatitis C virus genotype 1, 2, 4, 5, and 6 infection, with and
without cirrhosisIn April 2017, we reported that Janssen’s
OMEGA-1 global phase 2b clinical trial was fully enrolled with a
total of 365 subjects. Results from this trial are anticipated
during the second half of 2017.
About Achillion’s Complement Alternative
Pathway (AP) Factor D Inhibitor PlatformAchillion has
leveraged its internal discovery capabilities and a novel
complement-related platform to develop small molecule factor D
inhibitor compounds that target the complement AP. Factor D is an
essential serine protease involved in the AP, a part of the innate
immune system. Achillion's complement platform is focused on
seeking to advance small molecule compounds that inhibit factor D
and can potentially be used in the treatment of immune-related
diseases in which the AP plays a critical role. Potential
indications currently being evaluated for these compounds include
paroxysmal nocturnal hemoglobinuria (PNH), C3 glomerulopathy (C3G),
and geographic atrophy (GA), an advanced form of age-related
macular degeneration (AMD).
About Paroxysmal Nocturnal Hemoglobinuria
(PNH)
PNH is thought to be caused by a mutation resulting
in the absence of receptors normally present on red blood cells
(RBCs) that interact with the AP. The AP of the complement system
typically functions normally in these patients but due to the lack
of key receptors, known as CD55 and CD59, on the surface of PNH
RBCs, the AP treats these cells as foreign and destroys them via
hemolysis in the circulatory system (intravascular) and in the
liver or spleen (extravascular). Complement factor D is a critical
protein within the amplification loop of the AP and it is believed
that inhibiting it could control the AP response. Furthermore, this
mechanism of action represents a potentially distinct and unique
therapeutic approach for controlling intravascular and
extravascular hemolysis associated with PNH.
About Chronic Hepatitis C Viral
Infection
The hepatitis C virus (HCV) is one of the most
common causes of viral hepatitis, which is an inflammation of the
liver. It is currently estimated that more than 150 million people
are infected with HCV worldwide including more than 5 million
people in the United States. Three-quarters of the HCV patient
population is undiagnosed; it is a silent epidemic and a major
global health threat. Chronic hepatitis, if left untreated, can
lead to permanent liver damage that can result in the development
of liver cancer, liver failure or death. Few therapeutic options
currently exist for the treatment of HCV infection.
About Achillion
Pharmaceuticals
Achillion Pharmaceuticals, Inc. (NASDAQ:ACHN) is a
science-driven, patient-focused company seeking to leverage its
strengths across the continuum from discovery to commercialization
in its goal of providing better treatments for people with serious
diseases. The company employs a highly-disciplined discovery and
development approach that has allowed it to pursue best-in-class
oral antiviral therapy for chronic hepatitis C (HCV) and build a
platform of potent and specific complement factor D inhibitors for
AP-mediated diseases. Achillion is rapidly advancing its efforts to
become a fully-integrated pharmaceutical company with a goal of
bringing life-saving medicines to patients with rare diseases. More
information is available at http://www.achillion.com.
Cautionary Note Regarding Forward-Looking
Statements
This press release includes forward-looking
statements within the meaning of the Private Securities Litigation
Reform Act of 1995 that are subject to risks, uncertainties and
other important factors that could cause actual results to differ
materially from those indicated by such forward-looking statements.
Achillion may use words such as “expect,” “anticipate,” “project,”
“target,” “intend,” “plan,” “aim,” “believe,” “seek,” “estimate,”
“can,” “could,” “focus,” “will,” “look forward,” “goal,” “may,”
“potential,” and similar expressions to identify such
forward-looking statements. These forward-looking statements also
include statements about: the potential benefits of, and potential
indications for, Achillion’s compounds that inhibit factor D,
including the potential for its compounds to treat PNH,C3G and
other diseases; the timing for interim results from the Phase 2
study of ACH-4771 in PNH; and statements concerning Achillion’s
ability to achieve its strategic goals, and statements concerning
its plans, and prospects, including those relating to ACH-4771 and
its complement factor D inhibitor program. Among the important
factors that could cause actual results to differ materially from
those indicated by such forward-looking statements are risks
relating to, among other things, Achillion’s ability to: advance
the preclinical and clinical development of its complement factor D
inhibitors under the timelines it projects in current and future
preclinical studies and clinical trials; obtain and maintain patent
protection for its technologies and drug candidates and the freedom
to operate under third party intellectual property; demonstrate in
any current and future clinical trials the requisite safety,
efficacy and combinability of its drug candidates; obtain and
maintain necessary regulatory approvals; establish commercial
manufacturing arrangements; identify, enter into and maintain
collaboration agreements with third-parties, including the current
collaboration with Janssen; compete successfully in the markets in
which it seeks to develop and commercialize its product candidates
and future products; manage expenses; manage litigation; raise the
substantial additional capital needed to achieve its business
objectives; and successfully execute on its business strategies.
Furthermore, because Janssen is solely responsible for the
development and commercialization of Achillion's HCV assets under
the exclusive worldwide license Achillion granted to it and has the
deciding vote on all collaboration matters, Janssen generally has
full discretion over all development plans and strategies and may
not advance the HCV programs in the time frames Achillion or
Janssen projects, or at all, including with regard to the current
and planned phase 2b combination trials that include Achillion's
licensed drug candidates. Moreover, Janssen may not demonstrate in
any current and future clinical trials the requisite safety,
efficacy and combinability of drug candidates that incorporate
Achillion's HCV assets, or obtain and maintain necessary regulatory
approvals for such programs. These and other risks are described in
the reports filed by Achillion with the U.S. Securities and
Exchange Commission, including its Annual Report on Form 10-K for
the fiscal year ended December 31, 2016, and any subsequent SEC
filings.
In addition, any forward-looking statement in this
press release represents Achillion's views only as of the date of
this press release and should not be relied upon as representing
its views as of any subsequent date. Achillion disclaims any duty
to update any forward-looking statement, except as required by
applicable law.
-- Financial Tables Attached
--
ACHILLION PHARMACEUTICALS INC. (ACHN) |
|
|
|
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Statements of
Operations |
|
|
|
|
(Unaudited, in thousands, except per share
amounts) |
|
|
|
|
|
|
|
|
|
|
Three Months
Ended |
|
|
March
31, |
|
|
|
|
|
|
|
|
2017 |
|
|
|
2016 |
|
|
|
|
|
|
|
Revenue |
$ |
- |
|
|
$ |
- |
|
|
|
|
|
|
|
Operating
expenses: |
|
|
|
|
Research
and development |
|
15,495 |
|
|
|
13,278 |
|
|
General
and administrative |
|
5,648 |
|
|
|
5,440 |
|
|
|
|
|
|
|
Total
operating expenses |
|
21,143 |
|
|
|
18,718 |
|
|
|
|
|
|
|
Loss from
operations |
|
(21,143 |
) |
|
|
(18,718 |
) |
|
|
|
|
|
|
Other income
(expense): |
|
|
|
|
Interest
income |
|
1,008 |
|
|
|
679 |
|
|
Interest
expense |
|
(17 |
) |
|
|
(15 |
) |
|
|
|
|
|
|
Net loss |
$ |
(20,152 |
) |
|
$ |
(18,054 |
) |
|
|
|
|
|
|
Net loss per share -
basic and diluted |
$ |
(0.15 |
) |
|
$ |
(0.13 |
) |
|
|
|
|
|
|
Weighted average shares
outstanding - basic and diluted |
|
136,722 |
|
|
|
136,640 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance Sheets |
|
|
|
|
(Unaudited, in thousands) |
|
|
|
|
|
|
|
|
|
|
March
31, |
|
December
31, |
|
|
|
2017 |
|
|
|
2016 |
|
|
|
|
|
|
|
Cash, cash equivalents,
marketable securities and interest receivable |
$ |
386,565 |
|
|
$ |
392,486 |
|
|
Working capital |
|
372,795 |
|
|
|
368,564 |
|
|
Total assets |
|
393,353 |
|
|
|
413,875 |
|
|
Long-term
liabilities |
|
376 |
|
|
|
450 |
|
|
Total liabilities |
|
11,007 |
|
|
|
14,421 |
|
|
Total stockholders'
(deficit) equity |
|
382,346 |
|
|
|
399,454 |
|
|
|
|
|
|
|
Investors & Media:
Glenn Schulman, PharmD, MPH
Executive Director, Investor Relations
Achillion Pharmaceuticals, Inc.
Tel. (203) 752-5510
gschulman@achillion.com
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