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
Add after last paragraph of release: two financial tables.
The updated release reads:
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
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
adjustment.
- 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, zilucoplan.
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 planning.
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 (Unaudited) (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 diluted 32,349 22,614 30,652 22,579
Ra Pharmaceuticals, Inc. Condensed Consolidated
Balance Sheets (Unaudited) (In thousands)
September 30,2018
December 31, 2017
Assets 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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Investors:Argot PartnersNatalie
Wildenradt212-600-1902natalie@argotpartners.comorMedia:Argot
PartnersDavid Rosen212-600-1902david.rosen@argotpartners.com
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