Robust Enrollment Continues in Phase 2 PNH
Study; On Track for Data Update Around Year-end
Ra Pharmaceuticals, Inc. (NASDAQ:RARX), a clinical stage
biopharmaceutical company focusing on the development of
next-generation therapeutics for the treatment of
complement-mediated diseases, today announced financial results for
the third quarter ended September 30, 2017, and provided an update
on recent corporate and clinical developments, including enrollment
in the Phase 2 program for RA101495 SC in paroxysmal nocturnal
hemoglobinuria (PNH) and progress toward the initiation of a Phase
2 study in generalized myasthenia gravis (gMG).
“Enthusiasm for a convenient, subcutaneous (SC),
self-administered C5 inhibitor continues to be reflected in the
rapid recruitment of our Phase 2 PNH study, with 28 patients
enrolled to date,” said Doug Treco, PhD, President and Chief
Executive Officer of Ra Pharma. “We look forward to reporting data
from this program around the end of the year, initiating our Phase
2 study in gMG, and expanding our RA101495 SC clinical program by
initiating a Phase 1b renal impairment study to support clinical
trials in atypical hemolytic uremic syndrome and lupus nephritis.
We believe RA101495 SC holds the potential to become an important
and convenient treatment option accessible to a broad spectrum of
patients across a number of complement-mediated diseases.”
Recent Developments
- Progressed enrollment in Phase 2
clinical program evaluating RA101495 SC in PNH, with 28 patients
enrolled to date. A total of 27 patients have been dosed with
RA101495 SC, including 10 patients in Cohort A (eculizumab-naïve
patients), 16 patients in Cohort B (patients switching from
eculizumab to RA101495 SC), and one patient in the third cohort
(inadequate responders to eculizumab). The Company remains on track
to report additional data around the end of the year.
- Opened an Investigational New Drug
(IND) application with the US Food and Drug Administration and
activated sites in preparation for the Company’s Phase 2 trial
evaluating RA101495 SC for the treatment of gMG. gMG is a
complement-mediated autoimmune disease that causes muscle weakness
and reduced mobility. Convenient, self-administered RA101495 SC has
the potential to address a broader gMG patient population than is
practical with intravenous infusions. This target population
includes, but is not limited to, patients who have failed multiple
immunosuppressants. Ra Pharma anticipates dosing the first patient
in this Phase 2 trial in the fourth quarter of this year.
- Strengthened senior management team
with the addition of Steve Caffé, MD, Senior Vice President,
Regulatory Affairs and Quality. Dr. Caffé brings significant
expertise in the areas of pharmacovigilance and regulatory affairs,
having served in senior leadership roles at Sucampo
Pharmaceuticals, MedImmune LLC (the global biologics arm of
AstraZeneca), Baxter International, Aventis/Sanofi, and Merck &
Co. Dr. Caffé obtained his doctorate medical degree from Université
Pierre et Marie Curie (Paris 6), Faculté de Médecine
Saint-Antoine.
- Presented data at medical conferences,
including the European Meeting on Complement in Human Diseases, the
Muscle Study Group, the Macrocyclic and Constrained Peptides
Conference, and the Boulder Peptide Symposium. Data presented
includes Phase 1 and initial Phase 2 data demonstrating RA101495
SC’s complement C5 inhibition.
Third Quarter 2017 Financial Results
For the third quarter of 2017, the Company reported a net loss
of $15.3 million, or a net loss of $0.68 per share (basic and
diluted), compared to a net loss of $8.1 million, or a net loss of
$14.22 per share for the same period in 2016.
Research and development expenses for the third quarter of 2017
were $13.1 million, compared to $7.1 million for the same period in
2016. The increase in R&D expenses for the third quarter 2017
was primarily due to clinical development costs associated with our
lead program, RA101495 SC, for the treatment of PNH.
General and administrative expenses for the third quarter of
2017 were $2.3 million, compared to $1.0 million for the same
period in 2016. The increase in G&A expenses for the third
quarter 2017 was due primarily 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 September
30, 2017 or the three months ended September 30, 2016.
As of September 30, 2017, Ra Pharma reported total cash and
equivalents of $84.1 million. The Company expects that its cash and
cash equivalents will be sufficient to fund operations through the
end of 2018.
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 (SC)
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 also disrupts the interaction
between C5b and C6 and prevents assembly of the membrane attack
complex (MAC). This activity defines an additional, novel mechanism
for the inhibition of C5 function. In Phase 1 studies in healthy
volunteers and as noted in the initial data reported in June 2017
on two eculizumab-naïve patients in Ra Pharma’s Phase 2 program,
dosing of RA101495 SC was well tolerated and demonstrated sustained
and near complete suppression of hemolysis and complement activity.
To learn more about RA101495 SC, please visit:
http://rapharma.com/pipeline/ra101495/.
About RA101495 SC Phase 2 PNH Clinical Program
The global, dose-finding Phase 2 program is designed to evaluate
the safety, tolerability, preliminary efficacy, pharmacokinetics,
and pharmacodynamics of RA101495 SC in patients with PNH. The study
will evaluate RA101495 SC in three cohorts. Cohort A includes
eculizumab-naïve patients, Cohort B includes patients switching
from eculizumab to RA101495 SC, and a third cohort includes
patients who are currently treated with eculizumab, but have
evidence of an inadequate response. Patients in all three cohorts
will be eligible for a long-term extension study following the
completion of the initial 12-week studies. The primary efficacy
endpoint is change in lactate dehydrogenase (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 SC, including the timing,
designs, plans and announcement of results regarding our ongoing
and future studies and programs described in this press release,
and our expected cash runway from existing cash and cash
equivalents. 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 initial data from the Company’s
global Phase 2 clinical program evaluating RA101495 for the
treatment of PNH may not be indicative of final study results; the
risk that initial data from a limited number of patients may not be
indicative of results from the fully patient enrollment planned for
such study; 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) September 30,
2017 December 31, 2016 Assets Cash and
cash equivalents $ 84,091 $ 117,812 Prepaid expenses and other
current assets 1,123 1,690 Property and equipment, net 5,967 5,537
Other noncurrent assets 1,730 1,779 Total assets $
92,911 $ 126,818
Liabilities and Stockholders’ Equity
Accounts payable and accrued expenses $ 7,371 $ 6,434 Deferred rent
432 303 Noncurrent liabilities 2,531 2,859 Stockholders' equity
82,577 117,222 Total liabilities and stockholders’
equity $ 92,911 $ 126,818
Ra
Pharmaceuticals, Inc. Condensed Consolidated Statements of
Operations (Unaudited) (in thousands, except per
share data)
Three Months EndedSeptember 30
Nine Months EndedSeptember 30
2017 2016 2017 2016 Revenue $ - $ - $ -
$ 4,928 Operating expenses: Research and development 13,130 7,079
32,606 18,541 General and administrative 2,284
1,042 7,101 3,418 Total
operating expenses 15,414 8,121
39,707 21,959 Loss from operations (15,414 )
(8,121 ) (39,707 ) (17,031 ) Other income (expense), net 139
7 409 (945 ) Net loss $
(15,275 ) $ (8,114 ) $ (39,298 ) $ (17,976 ) Net loss
per common share – basic and diluted $ (0.68 ) $ (14.22 ) $ (1.74 )
$ (32.73 ) Weighted average number of common shares outstanding –
basic and diluted 22,614 571 22,579 549
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version on businesswire.com: http://www.businesswire.com/news/home/20171109006622/en/
Investors:Ra Pharmaceuticals, Inc.Jennifer Robinson,
617-674-9873jrobinson@rapharma.comorMedia:Argot PartnersEliza
Schleifstein, 917-763-8106eliza@argotpartners.com
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