- Dosing Complete, Primary Endpoint Met in Eculizumab Naïve
Cohort (p=0.002 for LDH reduction)- Switch Cohort: Interim Data
Demonstrate Successful Switching from Eculizumab- Phase 2
Enrollment Complete in All Cohorts- Company to Host Conference Call
Today, December 4, at 8:00am ET to Discuss Interim Data
Ra Pharmaceuticals, Inc. (NASDAQ: RARX) today announced positive
interim results from the Company’s ongoing, global Phase 2 clinical
program evaluating RA101495 SC 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).
This press release features multimedia. View
the full release here:
http://www.businesswire.com/news/home/20171204005307/en/
Eculizumab Naïve Cohort Data (Graphic:
Business Wire)
The global, dose-finding Phase 2 clinical program is designed to
evaluate the safety, tolerability, preliminary efficacy,
pharmacokinetics (PK), and pharmacodynamics (PD) of RA101495 SC in
patients with PNH. As of the data cut-off date of November 30,
2017, a total of 29 patients have been dosed with RA101495 SC in
three cohorts:
- Eculizumab naïve: 10 patients enrolled,
dosing completed
- Eculizumab switch: 16 patients
enrolled, dosing ongoing
- Eculizumab inadequate responders: 3
patients enrolled, dosing ongoing
Eculizumab Naïve Cohort: RA101495 SC met the primary
endpoint in eculizumab naïve patients (n=10). In these patients, a
rapid, robust, and sustained reduction in lactate dehydrogenase
(LDH) levels from baseline to the mean of Weeks 6-12 (p=0.002) and
near-complete suppression of complement activity were observed
(See figure). Fifty percent of eculizumab naïve patients who
were transfusion-dependent prior to enrollment have been
transfusion-free since commencing RA101495 SC. Based on preliminary
data, meaningful improvements in standard measures of quality of
life, as shown by the Functional Assessment of Chronic Illness
Therapy (FACIT) fatigue score, have been observed, as well as a
high level of patient satisfaction with subcutaneous
self-administration based on patient surveys completed to date.
Furthermore, 8 of 10 subjects enrolled in the study have elected to
continue treatment with RA101495 SC in a long-term extension
study.
Eculizumab Switch Cohort: Interim results from the
ongoing switch cohort demonstrate near complete, sustained, and
uninterrupted inhibition of complement activity during and after
eculizumab washout. The LDH response observed in switch patients is
bimodal based on prior transfusion requirements on eculizumab. In
transfusion-independent patients from this cohort (n=5), a
population segment representing approximately 80% of patients on
long-term eculizumab therapy, switching to RA101495 SC has been
successful as indicated by stable LDH levels and no episodes of
breakthrough hemolysis. Among difficult to treat eculizumab
patients who were transfusion-dependent at baseline (n=11) from
this cohort, breakthrough hemolysis occurred after switching in 7
patients, who all reverted to eculizumab without complications.
Inadequate Responder Cohort: In the U.S.-based cohort of
inadequate responders to eculizumab, who have a history of elevated
LDH, 3 patients have been enrolled. LDH stabilization and relief of
side effects associated with eculizumab intolerance have been
observed in the first patient enrolled in this cohort.
Across all cohorts, no meaningful safety or tolerability
concerns have been identified after more than 300 patient weeks of
cumulative exposure. The majority of adverse events were deemed
unrelated to the study drug and the most frequent study
drug-related adverse event to date was headache. No meningococcal
infections or thromboembolic events have been observed. Out of more
than 2,000 doses administered, only 7 mild (grade 1) injection site
reactions have occurred among 3 patients. Full compliance with once
daily subcutaneous self-administration of RA101495 SC has been
observed thus far.
“These results support the potential for
RA101495 to be a safe, effective, and convenient subcutaneous,
self-administered therapeutic option that patients may
readily adopt,” 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. “This is evident from the
meaningful and sustained LDH reduction seen in treatment
naïve patients, and by maintenance of LDH control in
transfusion-independent switch patients, who make up the majority
of the eculizumab-treated population.”
“RA101495 has now demonstrated the potential to serve as
first-line therapy for newly diagnosed patients with PNH, as well
as an attractive alternative for transfusion-free patients
switching from eculizumab,” said Doug Treco, PhD, President and
Chief Executive Officer of Ra Pharma. “We believe the vast majority
of PNH patients could benefit from greater access to therapy and
the convenience of subcutaneous self-administration. We look
forward to engaging with regulators around the world to advance
into a registrational program for RA101495 in PNH. We believe the
data announced today support the potential utility of RA101495
across the entire spectrum of C5-mediated diseases, including our
clinical development programs in gMG, aHUS, and LN.”
Conference Call & Webcast
Date:
Monday, December 4, 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 SCRa 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 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 is designed to disrupt the
interaction between C5b and C6 and prevent assembly of the membrane
attack complex (MAC). This activity may define an additional, novel
mechanism for the inhibition of C5 function.
About RA101495 SC Phase 2 Clinical ProgramThe 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 is
evaluating 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 LDH from baseline to the mean level from week
6 to week 12.
About Ra PharmaceuticalsRa 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.
Forward-Looking StatementThis 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 interim results as of November 30,
2017 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 the interim results from the patient
enrollment and dosing to date may not be indicative of the data
from the full patient enrollment and dosing 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/20171204005307/en/
Investors:Ra Pharmaceuticals, Inc.Jennifer Robinson,
617-674-9873jrobinson@rapharma.comorMedia:Argot PartnersEliza
Schleifstein, 917-763-8106eliza@argotpartners.com
Ra Pharmaceuticals (NASDAQ:RARX)
Historical Stock Chart
From Mar 2024 to Apr 2024
Ra Pharmaceuticals (NASDAQ:RARX)
Historical Stock Chart
From Apr 2023 to Apr 2024