Initial Data from Global Phase 2 Program Expected Mid-2017
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Apr. 26, 2017--
Ra Pharmaceuticals, Inc. (NASDAQ:RARX) today announced that it has
initiated dosing in the Company’s global Phase 2 clinical program
evaluating RA101495 for the treatment of paroxysmal nocturnal
hemoglobinuria (PNH). 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 and potent subcutaneously-administered (SC) inhibitor of
complement component 5 (C5). Inhibition of C5 is a clinically validated
approach for the control of complement-induced hemolysis, the process
responsible for the rupture and destruction of red blood cells (RBCs) in
patients with PNH.
“Commencement of dosing in our Phase 2 clinical program for RA101495 in
PNH is an important step in bringing a potential new therapy to patients
with this rare and life-threatening blood disorder,” said Doug Treco,
PhD, President and Chief Executive Officer of Ra Pharma. “Building on
our promising Phase 1 data, our Phase 2 program is designed to
demonstrate RA101495’s safety and preliminary efficacy in PNH patients.
RA101495 is a convenient, once daily, self-administered subcutaneous
dose and represents a significant advancement in the treatment of PNH.
We look forward to sharing initial data from our Phase 2 program in
mid-2017 and additional data by year-end.”
The global Phase 2 program is designed to evaluate the safety,
tolerability, preliminary efficacy, pharmacokinetics, and
pharmacodynamics of RA101495 in patients with PNH. The program is
comprised of two open-label Phase 2 studies, one conducted outside the
U.S. and one conducted in the U.S.
Outside of the U.S., the study will evaluate RA101495 in two cohorts.
Cohort A is expected to include 8-12 eculizumab-naïve patients and
Cohort B is expected to include 6-8 patients switching from eculizumab
The U.S. study will evaluate RA101495 in approximately 6-8 patients
who are currently treated with eculizumab but have evidence of an
inadequate response. An inadequate response is defined as a lactate
dehydrogenase (LDH) level equal to or greater than 1.5-fold the upper
limit of normal, indicating ongoing hemolysis. LDH is a measure of red
blood cell hemolysis and a key disease biomarker.
All patients enrolled in the Phase 2 studies will receive a single, 0.3
mg/kg SC loading dose of RA101495, followed by 0.1 mg/kg SC daily
thereafter. Following two weeks of treatment and based on a review of
safety and efficacy data, patients will continue with 0.1 mg/kg daily
or, if needed, the dose can be increased to 0.3 mg/kg daily to achieve
adequate control of hemolysis. 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 LDH
Pharma is developing RA101495 for paroxysmal
nocturnal hemoglobinuria (PNH), refractory
generalized myasthenia gravis (rMG), and lupus
nephritis (LN). The product is designed for convenient, once daily
SC self-administration. RA101495 is a synthetic, macrocyclic peptide
discovered using Ra Pharma’s powerful proprietary drug discovery
technology. The peptide binds complement 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 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, dosing
of RA101495 was well tolerated in healthy volunteers and demonstrated
sustained and near complete suppression of hemolysis and complement
activity. To learn more about RA101495, please visit: http://rapharma.com/pipeline/ra101495/.
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.
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. 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; 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.
View source version on businesswire.com: http://www.businesswire.com/news/home/20170426005369/en/
Source: Ra Pharmaceuticals, Inc.
Ra Pharmaceuticals, Inc.
Eliza Schleifstein, 917-763-8106