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Ra Pharmaceuticals Reports Second Quarter 2018 Financial Results and Announces Early Completion of Enrollment in gMG Phase 2 Program

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Surpassed target enrollment in Phase 2 trial of RA101495 SC for gMG

Top-line data now expected around year-end 2018

Regulatory progress on Phase 3 PNH program

Ra Pharmaceuticals, Inc. (NASDAQ:RARX) today announced financial results
for the second quarter ended June 30, 2018 and highlighted recent
progress in advancing its pipeline programs, including RA101495 SC for
the treatment of generalized myasthenia gravis (gMG), paroxysmal
nocturnal hemoglobinuria (PNH), and complement-mediated renal diseases
such as atypical hemolytic uremic disorder (aHUS), and its
first-in-class, orally bioavailable, small molecule C5 inhibitor program.

"Enthusiasm from neurologists and their patients for a convenient,
self-administered, subcutaneous (SC) C5 inhibitor has resulted in the
rapid recruitment of 44 patients in our Phase 2 clinical trial of
RA101495 SC for the treatment of gMG, not only allowing us to complete
enrollment ahead of schedule, but also to surpass our original
enrollment target of 36 patients. We now expect to report top-line data
around year-end 2018. The acceleration of our gMG Phase 2 program
highlights RA101495 SC's potential as a convenient and accessible
subcutaneous treatment option designed to deliver everyday complement
control to more patients with gMG," said Doug Treco, PhD, Chief
Executive Officer of Ra Pharma.

Dr. Treco continued, "We are also pleased to report that we have made
significant progress in discussions with several regulatory authorities
regarding our planned global Phase 3 studies of RA101495 SC for the
treatment of PNH and, to date, have met with the Medicines and
Healthcare products Regulatory Agency in the United Kingdom (MHRA),
Health Canada, and the US Food and Drug Administration (FDA) to discuss
the design of our Phase 3 registrational studies. The MHRA and Health
Canada have indicated that a single arm Phase 3 design in both naïve and
switch patients is acceptable, and discussions with the FDA are ongoing.
We plan to meet with the European Medicines Agency (EMA) in the fourth
quarter of 2018 to discuss our global Phase 3 program and, pending the
successful outcome of these discussions, anticipate initiating our Phase
3 clinical trials during the first half of 2019."

"We also completed dosing in our Phase 1b study designed to evaluate the
pharmacokinetic (PK) profile of RA101495 SC in patients with renal
impairment and expect to report top-line data by the end of the third
quarter of 2018. We believe these data will allow us to proceed with the
development of RA101495 SC in complement-mediated renal diseases, such
as aHUS," said Ramin Farzaneh-Far, MD, Chief Medical Officer of Ra
Pharma.

Second Quarter 2018 Highlights and Recent Developments

  • In August, Ra Pharma completed enrollment in the Phase 2 trial
    evaluating RA101495 SC 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 RA101495, 0.3 mg/kg of RA101495, or matching placebo. The
    Company plans to report top-line data around year-end 2018.
  • Dialogue advanced with global regulatory agencies regarding the design
    of the Phase 3 registrational program for RA101495 SC in both naïve
    and switch PNH patients.
    • Ra Pharma has met with the MHRA and Health Canada. Both agencies
      have indicated that a single arm Phase 3 design in both naïve and
      switch patients is acceptable.
    • Ra Pharma has met with the FDA, and discussions are ongoing.
    • Ra Pharma plans to meet with the EMA in the fourth quarter of 2018.

Pending the successful outcome of these discussions, Ra Pharma
anticipates initiating the Phase 3 clinical trials during the first half
of 2019.

  • Dosing has been completed in the Phase 1b PK study evaluating RA101495
    SC in patients with renal impairment, which is designed to enable
    development in complement-mediated renal diseases such as aHUS. The
    Company remains on track to report top-line data by the end of the
    third quarter 2018.
  • Data was presented on two programs at the 23rd Congress of
    the European Hematology Association, June 14-17, 2018 in Stockholm.
    • Oral, small molecule complement inhibitor program: This
      presentation described pre-clinical PK data demonstrating that
      compounds in the series display dose-dependent oral exposure and
      low clearance values, ex vivo and in vitro assays
      demonstrating selective engagement of complement C5, and a PK/PD
      relationship informing the level of exposure required to achieve
      complete complement inhibition. Collectively, these data
      confirmed, for the first time in an in vivo setting, the
      feasibility of oral, small molecule inhibition of complement C5.
    • Phase 2 trial of RA101495 SC in PNH: This presentation
      described the results of the Phase 2, global, multi-center,
      open-label, dose-finding study. These data demonstrated that
      RA101495 SC appears safe and well-tolerated in patients with PNH,
      with the ability to rapidly and robustly reduce lactate
      dehydrogenase (LDH) to the levels seen in patients receiving
      eculizumab, and which are associated with improved long-term
      outcomes.
  • An overview of the Phase 2 clinical trial design evaluating RA101495
    SC for the treatment of gMG was presented at the 4th
    Congress of the European Academy of Neurology, June 16-19, 2018, in
    Lisbon. The Phase 2, multi-center, randomized, double-blind,
    placebo-controlled trial is designed to evaluate the safety,
    tolerability, and preliminary efficacy of RA101495 SC in patients with
    gMG. The trial enrolled 44 patients. The presentation also highlighted
    previously reported Phase 1 healthy volunteer data for RA101495 SC.

Second Quarter 2018 Financial Results

For the second quarter of 2018, the Company reported a net loss of $15.7
million, or a net loss of $0.49 per share (basic and diluted), compared
to a net loss of $12.7 million, or a net loss of $0.56 per share for the
same period in 2017.

Research and development (R&D) expenses for the second quarter of 2018
were $12.3 million, compared to $10.5 million for the same period in
2017. The increase in R&D expenses for the second quarter of 2018 was
primarily due to clinical development costs associated with our lead
program, RA101495 SC.

General and administrative (G&A) expenses for the second quarter of 2018
were $3.8 million, compared to $2.3 million for the same period in 2017.
The increase in G&A expenses for the second quarter of 2018 was
primarily due to employee-related costs due to the increase in G&A
headcount to support the growth of the Company.

As of June 30, 2018, Ra Pharma reported total cash and cash equivalents
of $95.1 million. The Company expects that its cash and cash equivalents
will be sufficient to fund operations through the end of 2019.

About RA101495
SC

Ra 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 gMG Clinical Program

The Phase 2, multi-center, randomized, double-blind, placebo-controlled
trial is designed to evaluate the safety, tolerability, and preliminary
efficacy of RA101495 SC in patients with gMG. The trial has enrolled
[44] patients and will include a screening period of up to four weeks.
At the outset of the 12-week treatment period, patients were randomized
in a 1:1:1 ratio and will receive daily, subcutaneous doses of 0.1 mg/kg
of RA101495 SC, 0.3 mg/kg of RA101495 SC, or matching placebo. The
primary efficacy endpoint is change in Quantitative Myasthenia Gravis
(QMG) score from baseline to week 12. All patients will have the
opportunity to receive RA101495 SC in a long-term extension study.

About RA101495 SC Phase 2 PNH Clinical Program

The global, dose-finding Phase 2 program was designed to evaluate the
safety, tolerability, preliminary efficacy, pharmacokinetics, and
pharmacodynamics of RA101495 SC in patients with PNH. The study
evaluated RA101495 SC in three cohorts. The first cohort included
eculizumab-naïve patients, the second cohort included patients switching
from eculizumab to RA101495 SC, and the third cohort included patients
who were currently treated with eculizumab but had evidence of an
inadequate response. Patients in all three cohorts were eligible for
entry into a long-term extension study following the completion of the
initial 12-week studies. The primary efficacy endpoint was the change in
LDH 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 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 anticipated timelines for the release of clinical data . 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.

Ra Pharmaceuticals, Inc.

Condensed Consolidated Statements of Operations

(Unaudited)

(in thousands, except per share data)

     
 
 
 
 
Three Months Ended

June 30,

Six Months Ended

June 30,

2018 2017 2018 2017
Operating expenses:
Research and development $ 12,305 $ 10,464 $ 25,717 $ 19,476
General and administrative 3,821 2,348 7,133 4,817
Total operating expenses 16,126 12,812 32,850 24,293
Loss from operations (16,126) (12,812) (32,850) (24,293)
Other income (expense), net 380 149 606 270
Net loss $ (15,746) $ (12,663) $ (32,244) $ (24,023)
 
 
Net loss per common share – basic and diluted $ (0.49) $ (0.56) $ (1.08) $ (1.06)

Weighted average number of

common shares outstanding – basic and diluted

32,307 22,575 29,789 22,562
Ra Pharmaceuticals, Inc.  
Condensed Consolidated Balance Sheets
(Unaudited)
(In thousands)
 
June 30, 2018 December 31, 2017
 
Assets
Cash and cash equivalents $ 95,057 $ 70,381
Prepaid expenses and other current assets 3,092 2,496
Property and equipment, net 5,709 5,606
Other noncurrent assets 1,681 1,714
Total assets $ 105,539 $ 80,197
 
Liabilities and Stockholders' Equity
Accounts payable and accrued expenses $ 7,993 $ 8,285
Deferred rent 460 329
Noncurrent liabilities 2,166 2,399
Stockholders' equity 94,920 69,184
Total liabilities and stockholders' equity $ 105,539 $ 80,197

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