Ra Pharmaceuticals Initiates Dosing in Phase 1b Pharmacokinetic Study Evaluating RA101495 SC in Patients with Renal Impairment
January 08 2018 - 7:12AM
Business Wire
Ra Pharmaceuticals, Inc. (NASDAQ:RARX) today announced
that it has initiated dosing in the Company’s 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 evaluation of RA101495 SC in complement-mediated renal
diseases, such as atypical hemolytic uremic disorder (aHUS) and
lupus nephritis (LN). Ra Pharma is a clinical stage
biopharmaceutical company focusing on the development of
next-generation therapeutics for the treatment of
complement-mediated diseases and is developing RA101495 as a novel,
subcutaneously-administered (SC) inhibitor of complement component
5 (C5). RA101495 SC is currently in Phase 2 clinical development
for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and
generalized myasthenia gravis (gMG).
“Building on the clinical data collected to date from which we
observed the favorable PK and pharmacodynamic (PD) profile of
RA101495, this Phase 1b trial is designed to evaluate RA101495 in a
subset of patients with renal impairment, with the aim of expanding
the range of indications for RA101495 as a potential therapy for
patients with complement-mediated disorders,” said Ramin
Farzaneh-Far, MD, Chief Medical Officer of Ra Pharma. “C5 is a
clinically-validated target for multiple complement-mediated renal
disorders, including aHUS, and this trial is designed to provide
the necessary PK data to support the future evaluation of RA101495
in renal disorders.”
The Phase 1b, multi-center, open-label trial is designed to
evaluate the PK profile of RA101495 SC in patients with renal
impairment. The trial is planned to enroll approximately 16
subjects, including eight patients with severe renal impairment
matched with eight healthy control subjects with normal renal
function. Each patient will receive a single, SC dose of 0.3 mg/kg
of RA101495. The trial will compare the PK profile in patients with
renal impairment with subjects with normal renal function.
“The initiation of dosing in this Phase 1b trial is a meaningful
step for advancing RA101495 in multiple complement-mediated renal
indications,” said Doug Treco, PhD, President and Chief Executive
Officer. “The convenient, once-daily, SC, self-administration
dosing profile of RA101495 provides a compelling alternative to
currently available, intravenously-administered (IV) therapies, and
we continue to work toward providing a differentiated treatment
option for these patients and addressing this significant unmet
need. Following our recently reported positive interim data from
the RA101495 Phase 2 program in PNH, we look forward to expanding
the scope of RA101495 with the goal of maximizing its potential as
a safe, well-tolerated, convenient, and efficacious C5 inhibitor
for patients with complement-mediated disorders.”
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. To learn more about RA101495 SC,
please visit: http://rapharma.com/pipeline/ra101495/.
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
therapeutic potential and regulatory and clinical progress of our
product candidates, including RA101495, the potential for RA101495
to address additional indications, and the Phase 1b PK clinical
trial evaluating RA101495 in patients with renal impairment,
including statements regarding trial design and enrollment. 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 of
delay in enrollment or release of topline results from that
expected, including with respect to the Phase 1b PK clinical trial;
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 full 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.
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version on businesswire.com: http://www.businesswire.com/news/home/20180108005661/en/
Investors:Ra Pharmaceuticals, Inc.Jennifer Robinson,
617-674-9873jrobinson@rapharma.comorMedia:Argot PartnersEliza
Schleifstein, 917-763-8106eliza@argotpartners.com
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