Ra Pharma Announces Positive Results from Phase 1b Pharmacokinetic Study of Zilucoplan (RA101495 SC) in Patients with Renal I...
26 September 2018 - 9:30PM
Business Wire
Study enables clinical development in
complement-mediated renal disorders
Ra Pharmaceuticals, Inc. (Nasdaq:RARX) today announced positive
results from the Company’s Phase 1b pharmacokinetic (PK) study
evaluating zilucoplan (RA101495 SC) in patients with renal
impairment. The Company has received International
Nonproprietary Names (INN) clearance for use of the
name zilucoplan from the World Health Organization.
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Figure 1. Plasma Concentration-Time
Profile of Zilucoplan after Single Dose (Graphic: Business
Wire)
The Phase 1b, multi-center, open-label trial was designed to
evaluate the PK profile of zilucoplan in patients with severe renal
impairment as a lead-in to studying zilucoplan in
complement-mediated renal disorders. The trial enrolled 16
subjects, including eight patients with severe renal impairment
matched with eight healthy control subjects with normal renal
function. Each patient received a single, subcutaneous dose of 0.3
mg/kg of zilucoplan. The PK profile of zilucoplan was consistent
across both groups, with exposures similar in renally-impaired
patients and healthy volunteers (Figure 1). There were no adverse
events reported. Overall, the data indicate that zilucoplan can be
used in clinical studies of patients with renal impairment without
any need for dose adjustment.
Zilucoplan is currently being studied in a Phase 2 study in
generalized myasthenia gravis (gMG) with a top-line data read-out
expected around year-end 2018. The Company also recently completed
interactions with regulators, including the U.S. Food and Drug
Administration, for its global Phase 3 program of zilucoplan for
the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Based
on these interactions, Ra Pharma plans to initiate a global,
pivotal, single-arm Phase 3 trial in treatment-naïve PNH patients
during the first half of 2019.
“These results allow Ra Pharma to expand into
complement-mediated renal disorders, adding to a now broad
portfolio of clinical programs that includes our Phase 3-ready
program in PNH and an ongoing Phase 2 program in gMG,” said Doug
Treco, PhD, Chief Executive Officer of Ra Pharma. “In each of these
indications, we believe zilucoplan, as a once-daily, subcutaneously
self-administered therapy, offers the potential to address broader
patient populations with complement-mediated diseases by offering
greater access and convenience. We look forward to a number of
important milestones ahead, including top-line data in gMG around
year-end 2018.”
About Zilucoplan (RA101495 SC)
Ra Pharma is developing zilucoplan for paroxysmal nocturnal
hemoglobinuria (PNH), generalized myasthenia gravis (gMG), and
other complement-mediated disorders. The product is designed for
convenient, once-daily subcutaneous self-administration. Zilucoplan
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, zilucoplan is
designed to disrupt the interaction between C5b and C6 and prevent
assembly of the membrane attack complex. This activity may define
an additional, novel mechanism for the inhibition of C5
function.
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
potential safety, efficacy and regulatory and clinical progress of
our product candidates, including without limitation zilucoplan,
beliefs regarding clinical trial data, statements regarding trial
design, timeline and enrollment of our ongoing and planned clinical
programs, including without limitation our Phase 3 trial of
zilucoplan for the treatment of PNH, upcoming milestones, including
without limitation the release of top-line data in gMG around
year-end 2018, and expectations surrounding USAN approval of the
name zilucoplan. 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 zilucoplan, will not successfully be
developed or commercialized, in the timeframe we expect or at all;
the risk that topline results as of February 7, 2017 from
the Company's global Phase 2 clinical program evaluating zilucoplan
for the treatment of PNH may not be indicative of final study
results; the risk that USAN does not approve the name zilucoplan;
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: https://www.businesswire.com/news/home/20180926005245/en/
Argot PartnersInvestors:Natalie Wildenradt,
212-600-1902natalie@argotpartners.comorMedia:David Rosen,
212-600-1902david.rosen@argotpartners.com
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