Enrollment ongoing in Phase 3 clinical trial of
zilucoplan in gMG, with top-line results expected in early 2021
First patient dosed in Phase 2 clinical trial
of zilucoplan in IMNM, with top-line results expected in the second
half of 2020
IND cleared by FDA for the HEALEY ALS Platform
Trial at Mass General; Zilucoplan selected as one of the first
clinical candidates to be evaluated
UCB acquisition of Ra Pharma expected to close
by the end of the first quarter of 2020
Ra Pharmaceuticals, Inc. (Nasdaq:RARX) today announced financial
results for the fourth quarter and year ended December 31, 2019,
and provided an update on recent corporate and clinical
developments.
“2019 was a transformational year for Ra Pharma in our efforts
to discover, develop, and expand access to important therapies for
patients with rare diseases, including the strengthening of our
balance sheet with a $150 million financing and the advancement of
key clinical and pre-clinical programs. With the initiation of our
global, pivotal, Phase 3 RAISE study of zilucoplan in generalized
myasthenia gravis (gMG) and our Phase 2 clinical trial of
zilucoplan in immune-mediated necrotizing myopathy (IMNM), as well
as the selection of zilucoplan for a pioneering platform trial for
amyotrophic lateral sclerosis (ALS) sponsored by the Sean M. Healey
& AMG Center for ALS at Mass General, we’ve significantly
expanded zilucoplan’s pipeline of neurologic, tissue-based,
complement-mediated diseases,” said Doug Treco, Ph.D., President
and Chief Executive Officer of Ra Pharma.
Dr. Treco added: “Ra Pharma’s pending acquisition by UCB remains
on track, and we continue to anticipate the transaction to close by
the end of the first quarter of 2020. Sharing our commitment to the
rare disease patient community and our goal of developing novel and
accessible therapies, UCB is the optimal partner to further advance
zilucoplan, our C5 inhibitor life-cycle extension program, and our
early-stage discovery pipeline and platform research efforts.”
Fourth Quarter 2019 Highlights and Recent
Developments
- In October 2019, Ra Pharma announced the entry into a merger
agreement pursuant to which UCB has agreed to acquire Ra Pharma.
Under the terms of the agreement, Ra Pharma shareholders will
receive $48 in cash for each Ra Pharma share at closing (equity
value of approximately $2.5 billion). The Boards of Directors of
both companies unanimously approved the transaction, and a majority
of Ra Pharma shareholders have voted to approve the transaction,
which remains subject to obtaining antitrust clearance and other
customary closing conditions. The transaction is expected to close
by the end of the first quarter of 2020.
- In February 2020, Ra Pharma announced that full results from
its Phase 2 clinical trial of zilucoplan in patients with gMG were
published online in JAMA Neurology. The publication in JAMA
Neurology recognizes the impact and significance of the trial’s
findings demonstrating zilucoplan’s potential for the treatment of
gMG. The RAISE study, a pivotal Phase 3 clinical trial evaluating
zilucoplan for the treatment of gMG is ongoing, with top-line
results expected in early 2021.
- In December 2019, Ra Pharma announced the initiation of dosing
in a randomized, double-blind, placebo-controlled, multi-center,
Phase 2 clinical trial of zilucoplan for the treatment of IMNM. The
trial is designed to evaluate the safety, tolerability, and
efficacy of a once-daily, subcutaneously (SC) self-administered
dose of 0.3 mg/kg of zilucoplan versus placebo. Top-line results
are expected in the second half of 2020.
- In January 2020, Ra Pharma announced the U.S. Food and Drug
Administration’s (FDA) clearance of the Investigational New Drug
(IND) application for the HEALEY ALS Platform Trial for the
treatment of amyotrophic lateral sclerosis (ALS). Zilucoplan was
selected as one of the first clinical candidates to be evaluated in
this platform trial for ALS led by the Sean M. Healey & AMG
Center for ALS at Mass General. The platform trial is designed to
accelerate the development of effective treatments for patients
with the disease.
Fourth Quarter and Full Year 2019 Financial Results
For the fourth quarter of 2019, the Company reported a net loss
of $36.8 million, or a net loss of $0.79 per share (basic and
diluted), compared to a net loss of $16.2 million, or a net loss of
$0.47 per share, for the same period in 2018. For the full year
2019, Ra Pharma reported a net loss of $102.7 million, or a net
loss of $2.31 per share (basic and diluted), compared to a net loss
of $64.9 million, or a net loss of $2.06 per share, for the full
year 2018.
Research and development (R&D) expenses for the fourth
quarter of 2019 were $26.2 million, compared to $15.4 million for
the same period in 2018. Research and development expenses for the
full year 2019 were $82.6 million, compared to $54.5 million for
the full year 2018. The increase in R&D expenses for both the
fourth quarter and full year was primarily due to expenses relating
to our lead product candidate, zilucoplan, and employee-related
costs to support our increased research and development
activities.
General and administrative (G&A) expenses for the fourth
quarter of 2019 were $11.6 million, compared to $3.8 million for
the same period in 2018. G&A expenses for the full year 2019
were $27.3 million, compared to $14.4 million for the full year
2018. The increase in G&A expenses for both the fourth quarter
and full year was primarily due to an increase in third-party
professional fees related to the merger agreement with UCB,
employee-related costs due to the increase in headcount to support
the growth of the Company, and increased costs related to
pre-commercialization activities.
There was no revenue earned in the fourth quarter of 2019. Total
revenue for the year ended December 31, 2019, was $3.0 million,
related to the achievement of a clinical development milestone
under the Company’s collaboration with Merck. Total revenue earned
in the fourth quarter and full year ended December 31, 2018, was
$2.5 million, related to the achievement of a pre-clinical
development milestone under the Company’s collaboration with
Merck.
As of December 31, 2019, Ra Pharma reported total cash and cash
equivalents of $265.0 million. The Company expects that its cash
and cash equivalents will be sufficient to fund operating expenses
and capital expenditures through at least the end of 2021.
About Zilucoplan
Ra Pharma is developing zilucoplan and zilucoplan extended
release (XR) for generalized myasthenia gravis (gMG),
immune-mediated necrotizing myopathy (IMNM), amyotrophic lateral
sclerosis (ALS), and other tissue-based complement-mediated
disorders with high unmet medical need. The product candidate is
designed for convenient subcutaneous (SC) self-administration.
Zilucoplan is an investigational, synthetic, macrocyclic peptide
discovered using Ra Pharma's powerful proprietary drug discovery
technology. The peptide is designed to bind complement component 5
(C5) with sub-nanomolar affinity and allosterically inhibit its
cleavage into C5a and C5b upon activation of the classical,
alternative, or lectin pathways. The U.S. Food and Drug
Administration (FDA) has granted Orphan Drug Designation to
zilucoplan for the treatment of MG.
About Ra Pharmaceuticals
Ra Pharmaceuticals is a clinical-stage biopharmaceutical company
focused on leading the field of complement biology to bring
innovative and accessible therapies to patients with rare 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 Statements
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 Ra
Pharma’s ability to expand patient access to important therapies,
the safety, efficacy, regulatory and clinical progress, and
therapeutic potential of Ra Pharma’s product candidates, including
without limitation zilucoplan and zilucoplan XR, statements
regarding trial design, timeline, and enrollment of Ra Pharma’s
ongoing and planned clinical programs, including without limitation
the Phase 3 trial of zilucoplan for the treatment of gMG, the Phase
2 trial of zilucoplan for the treatment of IMNM and the HEALEY ALS
Platform Trial, plans and timing for the presentation of and
beliefs regarding clinical trial data, statements regarding the
merger with UCB, including the expected timing of closing, and the
expectation that Ra Pharma’s cash and cash equivalents will be
sufficient to fund operating expenses and capital expenditures
through at least the end of 2021. 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 and zilucoplan XR, will
not successfully be developed or commercialized, in the timeframe
we expect or at all; the risk that Ra Pharma may fail to enroll
patients in its clinical trials, which may cause delays or other
adverse effects; the risk that Ra Pharma may fail to obtain
additional financing on favorable terms or at all; risks relating
to the Merger, including: that Ra Pharma may be unable to obtain
stockholder approval as required for the Merger; conditions to the
closing of the Merger may not be satisfied and required regulatory
approvals may be delayed or not be obtained; the Merger may involve
unexpected costs, liabilities or delays; the business of Ra Pharma
may suffer as a result of uncertainty surrounding the Merger; the
outcome of any legal proceedings related to the Merger; Ra Pharma
may be adversely affected by other economic, business, and/or
competitive factors; the occurrence of any event, change or other
circumstances that could give rise to the termination of the Merger
Agreement; and other risks to the consummation of the Merger,
including the risk that the Merger will not be consummated within
the expected time period or at all. If the Merger is consummated,
Ra Pharma stockholders will cease to have any equity interest in Ra
Pharma and will have no right to participate in its earnings and
future growth. Additional factors that may affect the future
results of Ra Pharma are 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
Twelve Months Ended
December 31
December 31
2019
2018
2019
2018
Revenue
$0
$2,500
$3,000
$2,500
Operating expenses: Research and development
(26,202)
(15,357)
(82,642)
(54,449)
General and administrative
(11,625)
(3,801)
(27,337)
(14,439)
Total operating expenses
(37,827)
(19,158)
(109,979)
(68,888)
Loss from operations
(37,827)
(16,658)
(106,979)
(66,388)
Other income (expense), net
1,051
463
4,291
1,445
Net Loss
($36,776)
($16,195)
($102,688)
($64,943)
Net loss per common share - basic and diluted
($0.79)
($0.47)
($2.31)
($2.06)
Weighted average number of common shares outstanding - basic and
diluted
47,158
34,185
44,363
31,542
Ra Pharmaceuticals, Inc. Condensed Consolidated
Balance Sheets (In thousands) December 31,
2019 December 31, 2018 Assets Cash and
cash equivalents
$264,992
$209,822
Prepaid expenses and other current assets
9,702
2,585
Property and equipment, net
4,732
5,165
Operating lease right-of-use assets
3,109
-
Other noncurrent assets
1,617
1,648
Total assets
$284,152
$219,220
Liabilities and Stockholders' Equity Accounts payable
and accrued expenses
$16,995
$9,722
Operating lease liabilities
1,584
-
Deferred rent
-
479
Noncurrent liabilities
3,459
1,901
Stockholders' equity
262,114
207,118
Total liabilities and stockholders' equity
$284,152
$219,220
View source
version on businesswire.com: https://www.businesswire.com/news/home/20200227005242/en/
Investors: Ra Pharmaceuticals, Inc. Natalie Wildenradt,
617-674-9874 nwildenradt@rapharma.com
Media: Argot Partners David Rosen, 212-600-1902
david.rosen@argotpartners.com
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