- Rapid Recruitment into PNH Phase 2 Study with
17 Patients Currently Enrolled
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 second quarter ended June 30, 2017, and provided an update on
recent corporate and clinical developments, including enrollment in
the Phase 2 program for RA101495 in paroxysmal nocturnal
hemoglobinuria (PNH).
“We are pleased with the initial data from the first two
eculizumab-naïve patients enrolled in our Phase 2 program
evaluating RA101495 in PNH, which showed rapid declines in lactate
dehydrogenase (LDH), near-complete inhibition of hemolytic
activity, and no safety or tolerability concerns. The strength of
these data enabled the opening of the eculizumab-switch cohort and
generated strong interest from investigators and patients,”
said Ramin Farzaneh-Far, MD, Chief Medical Officer of Ra
Pharma. “Recruitment has since progressed rapidly, with a total of
17 patients currently enrolled, 14 of whom are already receiving
study drug. Enrollment targets in both the eculizumab-naïve and
eculizumab-switch cohorts have been met, and the first two patients
receiving RA101495 have transitioned to a long-term extension
study.”
Doug Treco, PhD, President and Chief Executive Officer of Ra
Pharma, added: “The initial Phase 2 data with this
self-administered, subcutaneous product candidate support our goal
of improving treatment options for patients suffering from PNH, as
well as other complement-mediated diseases, including myasthenia
gravis (MG) and lupus nephritis (LN). We plan to initiate a Phase 2
trial evaluating RA101495 in MG and a Phase 1b trial supporting
development in LN in the second half of 2017. We look forward to
providing updates on these and other pipeline programs later this
year.”
Recent Developments
- Announced initial data from Ra Pharma’s
global Phase 2 clinical program evaluating the safety,
tolerability, preliminary efficacy, pharmacokinetics (PK), and
pharmacodynamics (PD) of RA101495 in patients with PNH. The initial
data on two eculizumab-naïve patients with seven weeks of follow-up
showed no safety or tolerability concerns, no injection site
reactions, near-complete inhibition of hemolytic activity, rapid
declines in LDH, and 100% compliance with the once daily,
subcutaneous self-administration dosing regimen.
- Commenced enrollment in Cohort B of Ra
Pharma’s Phase 2 clinical program evaluating RA101495 in PNH,
comprised of patients switching from eculizumab, the current
standard of care. Cohort B was opened following review of the
initial data reported in Cohort A, which consists of
eculizumab-naïve patients. To date, 17 patients have been enrolled,
including 9 patients in Cohort A representing an expanded range of
elevated baseline LDH levels, and 8 patients in Cohort B, in whom
LDH was well-controlled at baseline. A total of 14 patients have
received RA101495, 8 patients in Cohort A and 6 patients in Cohort
B. The first two patients in Cohort A successfully completed the
Phase 2 study and have elected to continue treatment with RA101495
in a long-term extension study. The Company remains on track to
report additional data around year-end.
- Received Orphan Drug Designation from
the US Food and Drug Administration (FDA) for RA101495 for the
treatment of PNH. This designation provides various development
incentives, including tax credits for qualified clinical testing,
for drugs that treat a rare disease or condition, defined as
affecting fewer than 200,000 people in the US.
- Presented data on the Company’s oral
small molecule complement inhibitor program at the 22nd Congress of
the European Hematology Association on June 24, 2017, in Madrid.
The presentation highlighted this novel class of orally
bioavailable small molecules that bind to C5 with high affinity and
inhibit its cleavage into C5a and C5b, demonstrating the
feasibility of an orally-administered therapy for
complement-mediated disorders.
Second Quarter 2017 Financial Results
For the second quarter of 2017, the Company reported a net loss
of $12.7 million, or a net loss of $0.56 per share (basic and
diluted), compared to a net loss of $4.8 million, or a net loss of
$8.90 per share for the same period in 2016.
Research and development expenses for the second quarter of 2017
were $10.5 million, compared to $6.5 million for the same period in
2016. The increase in R&D expenses for the second quarter 2017
was primarily due to clinical development costs associated with our
lead program, RA101495, for the treatment of PNH.
General and administrative expenses for the second quarter of
2017 were $2.3 million, compared to $1.1 million for the same
period in 2016. The increase in G&A expenses for the second
quarter 2017 was due primarily 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.
There was no revenue recorded in the three months ended June 30,
2017, compared to $3.0 million for the same period in 2016. The
Company’s 2016 revenue was derived from its collaboration and
licensing agreement with Merck, which expired in April 2016.
As of June 30, 2017, Ra Pharma reported total cash and
equivalents of $96.6 million. The Company expects that its cash and
cash equivalents will be sufficient to fund operations through the
end of 2018.
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, once daily subcutaneous (SC) self-administration.
RA101495 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 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. To learn more about RA101495,
please visit: http://rapharma.com/pipeline/ra101495/.
About RA101495 Phase 2 Clinical Program
The global, dose-finding Phase 2 program is designed to evaluate
the safety, tolerability, preliminary efficacy, pharmacokinetics,
and pharmacodynamics of RA101495 in patients with PNH. The study
will evaluate RA101495 in three cohorts. Cohort A includes
eculizumab-naïve patients, Cohort B includes patients switching
from eculizumab to RA101495, and a third cohort includes patients
who are currently treated with eculizumab, but have evidence of an
inadequate response. Patients in all three cohorts will be eligible
for a long-term extension study following the completion of the
initial 12-week studies. The primary efficacy endpoint is 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
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; the risk that initial data from the
Company’s global Phase 2 clinical program evaluating RA101495 for
the treatment of PNH may not be indicative of final study results;
the risk that initial data from a limited number of patients may
not be indicative of results from the fully patient enrollment
planned for such study; 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) June 30, 2017 December 31,
2016 Assets Cash and cash equivalents $ 96,605 $
117,812 Prepaid expenses and other current assets 1,231 1,690
Property and equipment, net 6,239 5,537 Other noncurrent assets
1,746 1,779 Total assets $ 105,821 $ 126,818
Liabilities and Stockholders’ Equity Accounts payable and
accrued expenses $ 6,578 $ 6,434 Deferred rent 422 303 Noncurrent
liabilities 2,645 2,859 Stockholders' equity 96,176
117,222 Total liabilities and stockholders’ equity $ 105,821 $
126,818
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 2017 2016 2017 2016 Revenue $ -
$ 3,044 $ - $ 4,928 Operating expenses: Research and development
10,464 6,505 19,476 11,462 General and administrative 2,348
1,085 4,817 2,376
Total operating expenses 12,812 7,590
24,293 13,838 Loss from operations
(12,812 ) (4,546 ) (24,293 ) (8,910 ) Other income (expense), net
149 (260 ) 270 (952 ) Net
loss $ (12,663 ) $ (4,806 ) $ (24,023 ) $ (9,862 )
Net loss per common share – basic and diluted $ (0.56 ) $ (8.90 ) $
(1.06 ) $ (18.32 ) Weighted average number of common shares
outstanding – basic and diluted 22,575 540 22,562 538
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version on businesswire.com: http://www.businesswire.com/news/home/20170809006142/en/
Investors:Ra Pharmaceuticals, Inc.Jennifer Robinson,
617-674-9873jrobinson@rapharma.comorMedia:Argot PartnersEliza
Schleifstein, 917-763-8106eliza@argotpartners.com
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