- Rapid Declines in LDH and Near-Complete
Inhibition of Hemolytic Activity
- Review of Initial Safety and Efficacy Data
Enables Opening of Eculizumab Switch Cohort
- Company to Host Conference Call Today, June
27, at 8:00am ET to Discuss Initial Data
Ra Pharmaceuticals, Inc. (NASDAQ:RARX) today announced initial
data from 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, subcutaneously-administered (SC) inhibitor of
complement component 5 (C5).
The dose-finding Phase 2 clinical program is designed to
evaluate the safety, tolerability, preliminary efficacy,
pharmacokinetics (PK) and pharmacodynamics (PD) of RA101495 in
patients with PNH (see About RA101495 Phase 2 Clinical Program,
below). Currently, the study has three eculizumab-naïve patients
enrolled, of which two have completed seven weeks of follow-up and
are included in the Company’s initial data set. Review of these
initial data enables the opening of Cohort B, which will enroll
patients switching from eculizumab, the current standard of care,
for PNH.
Notable findings include:
- No safety or tolerability concerns
identified; no injection site reactions
- Near-complete inhibition of hemolytic
activity
- Rapid declines in lactate dehydrogenase
(LDH); mean LDH at Week 7 = 1.6X upper limit of normal (ULN)
- 100% compliance with once daily,
subcutaneous self-administration
At the week 6 visit, one patient reported recurrence of PNH
symptoms associated with hemoglobinuria and an elevated LDH level.
This episode of breakthrough hemolysis occurred in the setting of
an intercurrent illness, judged by the investigator to be most
likely due to a viral infection. The episode was transient and
resolved rapidly with LDH levels returning to 1.3X ULN by the week
7 visit with resolution of hemolytic symptoms.
“Currently, the only approved treatment for PNH is a monoclonal
antibody which must be administered intravenously every two weeks
by healthcare professionals,” said Principal Investigator
Anita Hill, M.D., PhD, MRCP, FRCPath, Consultant Haematologist
for Leeds Teaching Hospitals NHS Trust, UK, and Lead
Clinician for the National PNH Service in England. “As a
once daily, self-administered, subcutaneous therapy, RA101495
has the potential to offer a convenient option for patients with
PNH. These initial data on eculizumab-naïve patients are
encouraging, and I look forward to seeing data from patients
switching from eculizumab to RA101495.”
“We are pleased by the initial results from this study, our
first in PNH patients, whose primary purpose is to gain important
safety, preliminary efficacy, PK and PD data in the target patient
population,” said Doug Treco, PhD, President and Chief Executive
Officer of Ra Pharma. “These data will help inform dose selection
for pivotal trials and will help refine our clinical development
strategy. We look forward to giving you an update on our progress
around year-end.”
Conference Call & Webcast
Date:
Tuesday, June 27, 2017
Time:
8:00 a.m. ET
Telephone Access:
Domestic callers, dial 844-419-1655,
International callers, dial 216-562-0467, Reference the Ra
Pharmaceuticals conference call;
Telephone Access:
Go to the Investor Relations section of
the Ra Pharma website
(http://ir.rapharma.com/phoenix.zhtml?c=254447&p=irol-calendar)
and follow instructions for accessing the live webcast. Please
connect to the website at least 15 minutes prior to the start of
the conference call to ensure adequate time for any software
download that may be necessary.
About RA101495
Ra 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 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 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/.
About RA101495 Phase 2 Clinical Program
The global, dose-finding Phase 2 program is designed to evaluate
the safety, tolerability, preliminary efficacy, pharmacokinetics
and pharmacodynamics of RA101495 in patients with PNH. The study
will evaluate RA101495 in three cohorts. Cohort A includes
eculizumab-naïve patients, Cohort B includes patients switching
from eculizumab to RA101495 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 from baseline to the mean level from Week 6
to week 12.
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. 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.
View source
version on businesswire.com: http://www.businesswire.com/news/home/20170627005550/en/
Investors:Ra Pharmaceuticals, Inc.Jennifer Robinson,
617-674-9873jrobinson@rapharma.comorMedia:Argot PartnersEliza
Schleifstein, 917-763-8106eliza@argotpartners.com
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