Ra Pharmaceuticals, Inc. (NASDAQ:RARX), a clinical stage
biopharmaceutical company focused on the development of
next-generation therapeutics for the treatment of
complement-mediated diseases, today announced financial results for
the fourth quarter and year ended December 31, 2017 and provided an
update on recent corporate and clinical developments.
“We made tremendous progress in 2017, advancing the development
of our lead candidate, RA101495, a convenient, subcutaneous (SC),
self-administered inhibitor of complement component 5 (C5). In
addition to advancing our global clinical program for the treatment
of paroxysmal nocturnal hemoglobinuria (PNH), we initiated a Phase
2 study in generalized myasthenia gravis (gMG), the first of
several additional C5-mediated indications for RA101495 SC,” said
Doug Treco, PhD, President and Chief Executive Officer of Ra
Pharma.
The top-line data from the recently completed Phase 2 PNH
studies showed that RA101495 SC was well-tolerated and achieved
consistent, sustained, and near-complete inhibition of C5. “These
data provide the foundation for RA101495 SC to offer meaningful
therapeutic value and, importantly, based on convenience and
anticipated pricing flexibility, support expanded patient access
and broad utility as a potential first line treatment option for
complement-mediated diseases. We look forward to our upcoming
end-of-phase 2 discussions with regulators, with the goal of
initiating a registrational program in the second half of this
year,” added Dr. Treco.
Recent Developments
- Reported topline data in the global
Phase 2 clinical program evaluating RA101495 SC for the treatment
of PNH. The program evaluated RA101495 SC in three cohorts:
eculizumab-naïve patients (n=10), eculizumab-switch patients
(n=16), and eculizumab-switch patients that had evidence of an
inadequate response while on eculizumab (eculizumab inadequate
responders; n=3). A total of 21 patients completed the initial
12-week dosing period, and 16 of those patients (76%) are
continuing treatment with RA101495 SC in the Company's long-term
extension study (8 naïve patients and 8 switch patients, including
all 3 inadequate responders).
- In eculizumab-naïve patients, RA101495
SC met the primary endpoint, demonstrating a rapid, robust, and
sustained reduction in lactate dehydrogenase (LDH) levels from
baseline to the mean of weeks 6-12 (p=0.002) and near-complete
suppression of complement activity.
- In eculizumab-switch patients,
transfusion-independent patients (n=5) switching from eculizumab to
RA101495 SC maintained an overall stable mean LDH level, with one
patient withdrawing early due to breakthrough hemolysis and
reverting to eculizumab without complications. Among switch
patients who were transfusion-dependent at baseline (n=11),
breakthrough hemolysis occurred after switching in seven patients,
who all reverted to eculizumab treatment without complications.
Persistent transfusion-dependence in patients treated with
eculizumab is not adequately addressed by C5 inhibition, as it is
most commonly attributable to extravascular hemolysis which is
promoted by the action of complement factors upstream of C5.
- In patients who were inadequate
responders to eculizumab and have a history of elevated LDH levels,
all three patients (two transfusion-independent, one
transfusion-dependent) completed 12 weeks of dosing and elected to
enter the long-term extension study with stable mean LDH
levels.
Collectively, the Phase 2 data support the selection of 0.3
mg/kg RA101495 SC daily as the recommended dose in our Phase 3
studies in PNH, which the Company plans to advance in
treatment-naïve patients and transfusion-independent
eculizumab-switch patients. These studies are anticipated to
commence in the second half of 2018.
- Initiated dosing in the Phase 2
clinical trial evaluating RA101495 SC for the treatment of gMG. gMG
is a rare, complement-mediated, autoimmune disease that causes
weakness in skeletal muscles. This Phase 2 trial is designed to
evaluate the safety, tolerability, and preliminary efficacy of
RA101495 SC, and topline data is expected in the first half of
2019.
- Initiated dosing in the Phase 1b
clinical trial evaluating RA101495 SC in patients with renal
impairment. This trial is designed to characterize the
pharmacokinetics (PK) of RA101495 SC in these patients, thereby
enabling the potential evaluation of RA101495 SC in
complement-mediated renal diseases, such as atypical hemolytic
uremic syndrome (aHUS), and lupus nephritis (LN).
- Strengthened balance sheet with a
follow-on offering raising approximately $58.0 million in gross
proceeds, which included the full exercise by the underwriters of
their over-allotment option.
- Announced the acceptance of a platform
presentation at the American Academy of Neurology 70th Annual
Meeting on April 25, 2018, in Los Angeles. The presentation will
highlight Phase 1 healthy volunteer data for RA101495 SC, and
provide an overview of the Phase 2 clinical trial design evaluating
RA101495 SC for the treatment of gMG.
Fourth Quarter and Full Year 2017 Financial Results
For the fourth quarter of 2017, the Company reported a net loss
attributable to common shareholders of $15.1, or a net loss of
$0.67 per share (basic and diluted), compared to net loss of $10.9
million, or a net loss of $0.73 per share, for the same period in
2016. For the full year 2017, Ra Pharma reported a net loss
attributable to common shareholders of $54.4 million, or a net loss
of $2.41 per share (basic and diluted), compared to a net loss of
$28.9 million, or a net loss of $6.98 per share, for the full year
2016.
Research and development expenses for the fourth quarter of 2017
were $12.6 million compared to $9.4 million for
the same period in 2016. Research and development expenses for the
full year 2017 were $45.3 million compared to $27.9
million for the full year 2016. The increase in R&D
expenses for both the fourth quarter and full year were primarily
due to primarily due to clinical development costs associated with
our lead program, RA101495 SC, for the treatment of PNH.
General and administrative expenses for the fourth quarter of
2017 were $2.7 million, compared to $1.6 million for
the same period in 2016. General and administrative expenses for
the full year 2017 were $9.8 million, compared to $5.0
million for the full year 2016. The increase in G&A
expenses for both the fourth quarter and the full year were
primarily due to employee-related costs, including salary,
benefits, and non-cash stock-based compensation due to the increase
in G&A headcount to support the growth of the Company.
There was no revenue earned in the three months
ended December 31, 2017 or the three months
ended December 31, 2016. Total revenue for the full year 2017
was $0 million compared to $4.9 million for the full
year 2016. The decrease was due to the expiration of the research
term of the Merck Agreement in April 2016.
As of December 31, 2017, Ra Pharma reported total cash and
cash equivalents of $70.4 million. The Company expects that
its cash and cash equivalents at December 31, 2017 and the net
proceeds of $54.1 million from its follow-on equity offering in
February 2018, will be sufficient to fund operations through the
end of 2019.
About RA101495 SC
Ra Pharma is developing RA101495 SC for paroxysmal
nocturnal hemoglobinuria (PNH), generalized myasthenia gravis
(gMG), atypical hemolytic uremic syndrome (aHUS), and lupus
nephritis (LN). The product is designed for convenient, once-daily
subcutaneous self-administration. RA101495 SC is a synthetic,
macrocyclic peptide discovered using Ra Pharma's powerful
proprietary drug discovery technology. The peptide binds complement
component 5 (C5) with sub-nanomolar affinity and allosterically
inhibits its cleavage into C5a and C5b upon activation of the
classical, alternative, or lectin pathways. By binding to a region
of C5 corresponding to C5b, RA101495 SC is designed to disrupt the
interaction between C5b and C6 and prevent assembly of the membrane
attack complex (MAC). This activity may define an additional, novel
mechanism for the inhibition of C5 function.
About RA101495 SC Phase 2 PNH Clinical Program
The global, dose-finding Phase 2 program was designed to
evaluate the safety, tolerability, preliminary efficacy,
pharmacokinetics, and pharmacodynamics of RA101495 SC in patients
with PNH. The study evaluated RA101495 SC in three cohorts. The
first cohort included eculizumab-naïve patients, the second cohort
included patients switching from eculizumab to RA101495 SC, and the
third cohort included patients who were currently treated with
eculizumab, but had evidence of an inadequate response. Patients in
all three cohorts were eligible for entry into a long-term
extension study following the completion of the initial 12-week
studies. The primary efficacy endpoint was the change in LDH from
baseline to the mean level from week 6 to week 12.
About RA101495 SC Phase 2 gMG Clinical Program
The Phase 2, multicenter, randomized, double-blind,
placebo-controlled trial is designed to evaluate the safety,
tolerability, and preliminary efficacy of RA101495 SC in patients
with gMG. The trial will enroll approximately 36 patients and will
include a screening period of up to four weeks. At the outset of
the 12-week treatment period, patients will be randomized in a
1:1:1 ratio and will receive daily, subcutaneous doses of 0.1 mg/kg
of RA101495 SC, 0.3 mg/kg of RA101495 SC, or matching placebo. The
primary efficacy endpoint is change in Quantitative Myasthenia
Gravis (QMG) score from baseline to week 12. All patients will have
the opportunity to receive RA101495 SC in a long-term extension
study.
About Ra Pharmaceuticals
Ra Pharmaceuticals is a clinical stage biopharmaceutical
company focusing on the development of next-generation therapeutics
for complement-mediated diseases. The Company discovers and
develops peptides and small molecules to target key components of
the complement cascade. For more information, please visit:
www.rapharma.com.
Forward-Looking Statement
This press release contains "forward-looking statements" within
the meaning of the Private Securities Litigation Reform Act of
1995, including, but not limited to, statements regarding the
safety, efficacy and regulatory and clinical progress of our
product candidates, including RA101495, and statements regarding
trial design, timeline and enrollment of our ongoing and planned
clinical programs. All such forward-looking statements are based on
management's current expectations of future events and are subject
to a number of risks and uncertainties that could cause actual
results to differ materially and adversely from those set forth in
or implied by such forward-looking statements. These risks and
uncertainties include the risks that Ra Pharma's product
candidates, including RA101495, will not successfully be developed
or commercialized; the risk that topline results as
of February 7, 2017 from the Company's global Phase 2
clinical program evaluating RA101495 for the treatment of PNH may
not be indicative of final study results; as well as the other
factors discussed in the "Risk Factors" section in Ra Pharma's most
recently filed Annual Report on Form 10-K, as well as other risks
detailed in Ra Pharma's subsequent filings with the Securities
and Exchange Commission . There can be no assurance that the
actual results or developments anticipated by Ra Pharma will be
realized or, even if substantially realized, that they will have
the expected consequences to, or effects on, Ra Pharma. All
information in this press release is as of the date of the release,
and Ra Pharma undertakes no duty to update this information unless
required by law.
Ra Pharmaceuticals, Inc. Condensed Consolidated
Balance Sheets (Unaudited) (In thousands)
December 31, 2017 December
31, 2016 Assets Cash and cash equivalents $
70,381 $ 117,812 Prepaid expenses and other current assets 2,496
1,690 Property and equipment, net 5,606 5,537 Other noncurrent
assets 1,714 1,779 Total assets $ 80,197 $ 126,818
Liabilities and Stockholders’ Equity Accounts payable
and accrued expenses $ 8,285 $ 6,434 Deferred rent 329 303
Noncurrent liabilities 2,399 2,859 Stockholders' equity
69,184 117,222 Total liabilities and stockholders’ equity $
80,197 $ 126,818
Ra Pharmaceuticals, Inc.
Condensed Consolidated Statements of Operations
(Unaudited) (in thousands, except per share data)
Three Months Ended December
31
Twelve Months Ended December
31
2017 2016 2017 2016 Revenue $ - $ - $ -
$ 4,928 Operating expenses: Research and development 12,645 9,387
45,251 27,928 General and administrative 2,677
1,606 9,778 5,024 Total
operating expenses 15,322 10,993
55,029 32,952 Loss from operations (15,322 )
(10,993 ) (55,029 ) (28,024 ) Other income (expense), net 162 87
571 (858 ) Benefit from income taxes (19 ) (18 )
(19 ) (18 ) Net loss $ (15,141 ) $ (10,888 ) $
(54,439 ) $ (28,864 ) Net loss per common share – basic and
diluted $ (0.67 ) $ (0.73 ) $ (2.41 ) $ (6.98 ) Weighted average
number of common shares outstanding – basic and diluted 22,626
14,816 22,591 4,135
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Argot PartnersInvestors:Natalie Wildenradt,
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212-600-1902david.rosen@argotpartners.com
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