Ra Pharmaceuticals, Inc., (NASDAQ: RARX), a clinical stage
biopharmaceutical company focusing on the development of
next-generation therapeutics for the treatment of
complement-mediated diseases, today announced financial results for
the fourth quarter and year ended December 31, 2016 and provided an
update on recent corporate and clinical developments.
“Following our successful initial public offering
in October 2016, we are well positioned to advance our lead
complement C5 inhibitor, RA101495, and other pipeline programs in
2017,” said Doug Treco, PhD, President and Chief Executive Officer
of Ra Pharma. “Having secured approval from the FDA and other
ex-U.S. regulatory agencies to initiate our Phase 2 program in PNH
patients, we look forward to starting these studies by the end of
the first quarter. RA101495 is designed as a convenient
self-administered subcutaneous injection, with highly predictable
pharmacokinetics and robust pharmacodynamic effects. We believe
that it represents an attractive alternative to the current
standard of care, which requires regular intravenous infusions and
does not adequately address the needs of all PNH patients. We
expect to share data from these studies in the second half of this
year.”
Dr. Treco added: “In addition to our PNH program, we plan to
initiate a Phase 2 clinical trial of RA101495 in myasthenia gravis
and a Phase 1b clinical trial supporting development in lupus
nephritis in the second half of 2017. We will also advance our
once-weekly formulation of RA101495, our orally-available small
molecule C5 inhibitors and other pipeline programs. We look forward
to reporting on our continued progress.”
Recent Developments
- Successfully completed our initial
public offering which raised approximately $105.4 million in gross
proceeds, including the full exercise by the underwriters of their
over-allotment option. Net proceeds to Ra Pharma, after deducting
underwriting discounts and commissions and offering expenses, were
approximately $95.7 million.
- Granted Orphan Drug Designation by the
European Commission for RA101495 for the treatment of PNH. This
designation provides certain regulatory and financial incentives
for companies to develop and market therapies that treat a
life-threatening or chronically debilitating condition affecting no
more than five in 10,000 persons in the European Union, and for
which no satisfactory treatment is available, or for which the
product under consideration is expected to provide significant
benefits to patients versus existing therapies.
- Presented data from the Company’s
RA101495 and Oral C5 Inhibitor programs at the International PNH
Interest Group Annual Scientific Assembly on December 2, 2016 in
San Diego. The presentation highlighted a completed Phase 1 study
of RA101495 in healthy volunteers, which demonstrated that it is a
well-tolerated, potent, subcutaneously-administered inhibitor of C5
capable of achieving rapid, complete and sustained inhibition of
hemolysis. The presentation also included a description of the
planned Phase 2 study for RA101495 in PNH, and highlighted new
molecules discovered through the Company’s oral small molecule C5
inhibitor program.
Upcoming Milestones
- In the first quarter of 2017, the
Company expects to initiate a global Phase 2 development program of
RA101495 in PNH. The program is comprised of two open-label
studies, one in the U.S. and one ex-U.S. Data is expected in the
second half of 2017.
- In the second half of 2017, the Company
plans to initiate a Phase 2 clinical trial of RA101495 in
refractory generalized myasthenia gravis (rMG) and a Phase 1b
clinical trial to support development in lupus nephritis (LN).
Fourth Quarter and Full Year 2016 Financial Results
As of December 31, 2016, Ra Pharma reported total cash and
equivalents of $117.8 million.
For the fourth quarter of 2016, the Company reported a net loss
attributable to common shareholders of $10.9 million, or a net loss
of $0.74 per share (basic and diluted), compared to net loss of
$4.6 million, or a net loss of $8.63 per share, for the same period
in 2015. For the full year 2016, Ra Pharma reported a net loss
attributable to common shareholders of $28.9 million, or a net loss
of $6.98 per share (basic and diluted), compared to a net loss of
$12.3 million, or a net loss of $24.68 per share, for the full year
2015.
Research and development expenses for the fourth quarter of 2016
were $9.4 million compared to $4.6 million for the same period in
2015. Research and development expenses for the full year 2016 were
$27.9 million compared to $15.2 million for the full year 2015. The
increase in R&D expenses for both the fourth quarter and full
year were primarily due to pre-clinical and clinical development
costs associated with our lead program, RA101495, for the treatment
of PNH.
General and administrative expenses for the fourth quarter of
2016 were $1.6 million, compared to $0.7 million for the same
period in 2015. General and administrative expenses for the full
year 2016 were $5.0 million, compared to $2.2 million for the full
year 2015. The increase in G&A expenses for both the fourth
quarter and the full year were due to employee-related costs,
including salary, benefits and stock-based compensation due to the
increase in G&A headcount to support the growth of the
Company.
The Company’s revenue is derived from its collaboration and
licensing agreement with Merck. Revenue for the three months ended
December 31, 2016 was $0 compared to $0.8 million for the same
period in 2015. The decrease was due to the expiration of the
research term of the Merck Agreement in April 2016. Total revenue
for the full year 2016 was $4.9 million compared to $4.1 million
for the full year 2015, and included a $3.0 million milestone
payment for the delivery and transfer of novel, orally-available
cyclic peptides for a non-complement cardiovascular target with a
large market opportunity, to Merck for further development.
About RA101495
Ra Pharma is developing RA101495 for paroxysmal nocturnal
hemoglobinuria (PNH), refractory generalized myasthenia gravis
(rMG), and lupus nephritis (LN). The product is designed for
convenient, subcutaneous self-administration. RA101495 is a
synthetic, macrocyclic peptide discovered using Ra Pharma’s
powerful proprietary drug discovery technology. The peptide binds
complement C5 with subnanomolar 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 also disrupts the interaction
between C5b and C6 and prevents assembly of the membrane attack
complex (MAC). This activity defines an additional, novel mechanism
for the inhibition of C5 function. In Phase 1 studies, dosing of
RA101495 was well tolerated in healthy volunteers and demonstrated
sustained and near complete suppression of hemolysis and complement
activity.
About the Extreme Diversity™ Platform
Ra Pharma’s proprietary Extreme Diversity™ mRNA display platform
allows the Company to produce synthetic macrocyclic peptides that
combine the diversity and specificity of antibodies with the
pharmacological properties of small molecules. The platform
generates highly specific and stable peptide-like molecules with
the potential for greatly increased bioavailability, improved cell
permeability, and the opportunity to address protein-protein
interactions including previously undruggable targets. It can
produce libraries of 10 to 100 trillion members, allowing for the
rapid discovery of highly potent candidate molecules. The platform
is being leveraged for the Company’s emerging pipeline projects in
Factor D and C1.
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
safety, efficacy and regulatory and clinical progress of our
product candidates, including RA101495. 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, will not successfully be
developed or commercialized; 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 Balance Sheets (unaudited)
(In thousands) December 31, December
31, 2016 2015 Assets Cash and cash
equivalents $ 117,812 $ 19,386 Prepaid expenses and other current
assets 1,690 800 Property and equipment, net 5,537 2,143 Other
assets, noncurrent 1,779 2,013 Total assets $
126,818 $ 24,342
Liabilities and stockholders’
equity (deficit) Accounts payable and accrued expenses $ 6,434
$ 2,593 Deferred revenue - 1,621 Other liabilities, current 303
2,724 Other liabilities, noncurrent 2,859 928 Redeemable
convertible preferred stock - 53,675 Stockholders' equity (deficit)
117,222
(37,199
)
Total liabilities, redeemable convertible preferred
stock and stockholders’ equity (deficit) $ 126,818 $ 24,342
Ra
Pharmaceuticals, Inc. Condensed Consolidated Statements of
Operations (Unaudited) (In thousands, except share and per
share data) Three Months Ended
Twelve Months Ended
December 31, December 31, 2016 2015
2016 2015 Revenue $ - $ 821 $
4,928 $ 4,094 Operating expenses Research and
development 9,387 4,624 27,928 15,217 General and administrative
1,605 684 5,024
2,233 Total operating expenses 10,992
5,308 32,952 17,450 Loss from
operations (10,992 ) (4,487 ) (28,024 ) (13,356 ) Other income
(expense), net and benefit from income taxes 104
(76 ) (840 ) (587 ) Net loss $ (10,888 ) $
(4,563 ) $ (28,864 ) $ (13,943 ) Gain on extinguishment of
redeemable convertible preferred shares - -
- 1,673 Net loss attributable to
common shareholders $ (10,888 ) $ (4,563 ) $ (28,864 ) $ (12,270 )
Net loss per share attributable to common shareholders-basic
and diluted $ (0.73 ) $ (8.63 ) $ (6.98 ) $ (24.68 )
Weighted-average number of common shares oustanding-basic and
diluted 14,815,949 528,511 4,135,331 497,073
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version on businesswire.com: http://www.businesswire.com/news/home/20170306006360/en/
Investors:Ra Pharmaceuticals, Inc.Jennifer Robinson,
617-674-9873jrobinson@rapharma.comorMedia:Argot PartnersEliza
Schleifstein, 917-763-8106eliza@argotpartners.com
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