ADDING and REPLACING Ra Pharmaceuticals Reports Third Quarter 2018 Financial Results and Provides Corporate Update

Dosing completed in Phase 2 trial of zilucoplan in generalized
myasthenia gravis

On track for topline data read-out around year-end 2018

Announces issuance of U.S. patent covering zilucoplan composition of
matter and methods of use

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Add after last paragraph of release: two financial tables.

The updated release reads:


Dosing completed in Phase 2 trial of zilucoplan in generalized
myasthenia gravis

On track for topline data read-out around year-end 2018

Announces issuance of U.S. patent covering zilucoplan composition of
matter and methods of use

Ra Pharmaceuticals, Inc. (Nasdaq:RARX) today announced financial results
for the third quarter ended September 30, 2018 and highlighted recent
progress in advancing its pipeline programs, including zilucoplan for
the treatment of generalized myasthenia gravis (gMG), paroxysmal
nocturnal hemoglobinuria (PNH), and complement-mediated renal disorders.

We are pleased to report that dosing in our Phase 2 trial of zilucoplan
for gMG is now complete and we look forward to reporting top-line data
around year-end,” said Doug Treco, PhD, Chief Executive Officer of Ra
Pharma. “The rapid over-enrollment of this study underscores both
patient and physician enthusiasm for an accessible and convenient
self-administered, subcutaneous, complement inhibitor. Notably, to date,
over 12,000 doses of zilucoplan have been self-administered and were
well-tolerated by patients across our clinical programs.”

Third Quarter 2018 Highlights and Recent Developments

  • In August, Ra Pharma completed enrollment in the Phase 2 trial
    evaluating zilucoplan for the treatment of gMG. The multi-center,
    randomized, double-blind, placebo-controlled trial enrolled a total of
    44 patients. At the outset of the 12-week treatment period, patients
    were randomized in a 1:1:1 ratio and received daily SC doses of 0.1
    mg/kg of zilucoplan, 0.3 mg/kg of zilucoplan, or matching placebo.
    Dosing was completed at the end of October 2018 and the Company plans
    to report top-line data around year-end 2018. All 44 patients
    completed the 12-week study and, of these, 43 (98%) elected to enter a
    long-term extension to receive active study drug.
  • In September, Ra Pharma 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 zilucoplan for the treatment of PNH. Based
    on FDA feedback and advice provided by the Medicines and Healthcare
    products Regulatory Agency in the UK (MHRA) and Health Canada, and
    pending discussions with the European Medicines Agency (EMA) in the
    fourth quarter of 2018, Ra Pharma plans to initiate a global, pivotal,
    single-arm Phase 3 trial to evaluate the safety and efficacy of
    zilucoplan in approximately 40 treatment-naïve PNH patients in the
    first half of 2019. In addition, the Company expects to initiate a
    supportive trial in approximately 40 transfusion-independent patients
    switching from eculizumab to zilucoplan. In a separate CMC Type C
    meeting, Ra Pharma has also reached agreement with the FDA on the
    pharmaceutical development program required to support the Phase 3
    clinical program.
  • In September, Ra Pharma reported positive results from the Phase 1b,
    multi-center, open-label, pharmacokinetic (PK) study of zilucoplan in
    patients with renal disease. The trial enrolled 16 patients, eight
    with severe renal impairment matched with eight healthy control
    subjects. The PK profile of zilucoplan was consistent and similar
    across both groups. These results support the expansion of zilucoplan
    into complement-mediated renal disorders without the need for dose
  • In September, Ra Pharma elected Aoife M. Brennan, M.B., B.Ch., to its
    Board of Directors. Dr. Brennan serves as President, Chief Executive
    Officer, and Chief Medical Officer of Synlogic, Inc. (Nasdaq:SYBX).
  • Ra Pharma announced today that the U.S. Patent and Trademark Office
    (USPTO) has issued U.S. Patent No. 10,106,579 which covers a family of
    molecules, including zilucoplan. The patent provides composition of
    matter protection of zilucoplan and methods of use in the treatment of
    complement-mediated disorders. This patent is the first in a series of
    composition of matter and treatment filings that is designed to
    protect zilucoplan through at least 2035.

Third Quarter 2018 Financial Results

For the third quarter of 2018, the Company reported a net loss of $16.5
million, or a net loss of $0.51 per share (basic and diluted), compared
to a net loss of $15.3 million, or a net loss of $0.68 per share for the
same period in 2017.

Research and development expenses for the third quarter of 2018 were
$13.4 million, compared to $13.1 million for the same period in 2017.
The increase in R&D expenses for the third quarter of 2018 was primarily
due to clinical development costs associated with our lead program,

General and administrative expenses for the third quarter of 2018 were
$3.5 million, compared to $2.3 million for the same period in 2017. The
increase in G&A expenses for the third quarter of 2018 was primarily due
to employee-related costs due to the increase in headcount to support
the growth of the Company and cost associated with pre-commercialization

There was no revenue earned in the three months ended September 30, 2018
or the three months ended September 30, 2017.

As of September 30, 2018, Ra Pharma reported total cash and cash
equivalents of $81.1 million. The Company expects that its cash and cash
equivalents will be sufficient to fund our operating expenses and
capital expenditure through the end of 2019.

About Zilucoplan
(formerly RA101495 SC)

Ra Pharma is developing zilucoplan for generalized
myasthenia gravis (gMG)
, paroxysmal
nocturnal hemoglobinuria (PNH)
, and other complement-mediated
disorders. The product candidate 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 additionally 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, pending discussions with the EMA in the fourth quarter of 2018, and
upcoming milestones, including without limitation the release of
top-line data in gMG around year-end 2018. 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.

Ra Pharmaceuticals, Inc.
Condensed Consolidated Statements of Operations
(in thousands, except per share data)

Three Months Ended
September 30,

Nine Months Ended
September 30,

2018 2017 2018 2017
Operating expenses:
Research and development $ 13,375 $ 13,130 $ 39,092 $ 32,606
General and administrative   3,504     2,284     10,637     7,101  
Total operating expenses   16,879     15,414     49,729     39,707  
Loss from operations (16,879 ) (15,414 ) (49,729 ) (39,707 )
Other income (expense), net   375     139     981     409  
Net loss $ (16,504 ) $ (15,275 ) $ (48,748 ) $ (39,298 )
Net loss per common share – basic and diluted $ (0.51 ) $ (0.68 ) $ (1.60 ) $ (1.74 )
Weighted average number of common shares outstanding – basic and
32,349 22,614 30,652 22,579
Ra Pharmaceuticals, Inc.
Condensed Consolidated Balance Sheets
(In thousands)

September 30,

December 31,

Cash and cash equivalents $ 81,059 $ 70,381
Prepaid expenses and other current assets 2,502 2,496
Property and equipment, net 5,394 5,606
Other noncurrent assets   1,664   1,714
Total assets $ 90,619 $ 80,197
Liabilities and Stockholders’ Equity
Accounts payable and accrued expenses $ 7,648 $ 8,285
Deferred rent 469 329
Noncurrent liabilities 2,043 2,399
Stockholders’ equity   80,459   69,184
Total liabilities and stockholders’ equity $ 90,619 $ 80,197


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