Market Overview

Ra Pharma Announces Completion of End-of-Phase 2 Interactions with FDA and Design of Phase 3 PNH Program


Plans to initiate a global, pivotal, single-arm Phase 3 trial in
treatment-naïve PNH patients

Ra Pharmaceuticals, Inc. (NASDAQ:RARX) today announced the completion of
End-of-Phase 2 interactions with the U.S. Food and Drug Administration
(FDA) for its global Phase 3 program of RA101495 SC for the treatment of
paroxysmal nocturnal hemoglobinuria (PNH).

Based on feedback provided by FDA during End-of-Phase 2 discussions
regarding the Company's planned global Phase 3 PNH program, as well as
advice previously provided by the Medicines and Healthcare products
Regulatory Agency in the United Kingdom (MHRA) and Health Canada, Ra
Pharma plans to initiate a global, pivotal, single-arm Phase 3 trial to
evaluate the safety and efficacy of RA101495 SC in approximately 40
treatment-naïve PNH patients. The co-primary endpoints will be
hemoglobin stabilization and reduction in lactate dehydrogenase (LDH)
levels from baseline.

Ra Pharma anticipates the data from the planned global Phase 3
registrational trials, if positive, will serve as the basis to support
submission of a New Drug Application (NDA) for RA101495 SC for the
treatment of PNH. Ra Pharma also plans to meet with the European
Medicines Agency (EMA) in the fourth quarter of 2018.

In addition, Ra Pharma expects to initiate a supportive trial in
approximately 40 transfusion-independent patients switching from
eculizumab to RA101495 SC. The primary endpoint for this study will be
maintenance of transfusion-independence after switching.

Furthermore, in a separate CMC Type C meeting, Ra Pharma has also
reached agreement with the FDA on the pharmaceutical development plans
for RA101495 SC required to support the Phase 3 program.

"With feedback from FDA, MHRA, and Health Canada now incorporated into
the design of our global Phase 3 PNH program, we're looking forward to
meeting with the EMA in the fourth quarter of 2018 and, pending the
successful outcome of those discussions, anticipate initiating our Phase
3 clinical trials during the first half of 2019," said Doug Treco, PhD,
Chief Executive Officer of Ra Pharma. "Our recent regulatory progress
brings us one step closer to being able to provide a more convenient and
readily accessible C5 inhibitor to patients with PNH, generalized
myasthenia gravis (gMG), and other complement-mediated disorders."

About RA101495

Ra Pharma is developing RA101495 SC for paroxysmal
nocturnal hemoglobinuria (PNH)
myasthenia gravis (gMG)
, and other complement-mediated disorders.
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 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:

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 RA101495 SC, planned meetings
with regulatory authorities, statements regarding trial design, timeline
and enrollment of our ongoing and planned clinical programs, including
without limitation our Phase 3 studies of RA101495 SC for the treatment
of PNH, and our expectation that Phase 3 data, if positive, will serve
as the basis to support submission of an NDA. 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 SC, 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 RA101495 SC for the treatment of PNH may not
be indicative of final study results; 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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