-- 100 mg Results Demonstrated a Mean Reduction
of Greater than 93% in Free C5 with Low Volume Once-a-Week
Subcutaneous Dosing --
-- Data Supports the Study of RLYB116 as a
Differentiated Therapeutic for the Treatment of Generalized
Myasthenia Gravis --
-- Company Announces Extension of Runway to 3Q
2025 As Part of Portfolio Prioritization --
-- Conference Call and Webcast Today at 8:30 AM
Eastern Time --
Rallybio Corporation (Nasdaq: RLYB) today announced preliminary
Phase 1 multiple ascending dose (MAD) data for RLYB116, an
innovative, long-acting, low volume subcutaneously injected
inhibitor of complement component 5 (C5), in development for
patients with complement-mediated diseases.
The Phase 1 MAD study for RLYB116 evaluated the safety,
tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of
subcutaneous RLYB116 in healthy participants with multiple dose
administration. The MAD study utilized an adaptive design and
included four cohorts of 12 participants receiving doses of up to
200 mg per week of RLYB116 or placebo, with a four-week treatment
duration and a 10-week follow-up period.
The preliminary results showed:
- A 100 mg low volume (1 mL) once-a-week dose of subcutaneously
administered RLYB116 achieved sustained mean reductions in free C5
of greater than 93%, including at Day 29 with measurement prior to
the last dose. The reduction in free C5 at 24 hours after the first
dose of 100 mg was greater than 99%. These data and additional work
we have conducted with RLYB116 reinforce our confidence that
RLYB116 has the potential to be an effective treatment for patients
with certain complement-mediated diseases, including generalized
myasthenia gravis (gMG).
- RLYB116 also demonstrated low inter-subject variability and
consistent increases in exposure relative to dose. The mean
estimated elimination half-life for RLYB116 was >300 hours.
- In comparison to 100 mg weekly administration, higher
concentrations of RLYB116 were observed in a cohort with 100 mg
administered twice per week and were associated with a greater than
97% mean reduction in free C5.
- RLYB116 administered as a 100 mg once-a-week dose was observed
to be generally well tolerated. The most common adverse event (AE)
in the cohort was injection site reaction (ISR), which occurred in
60% of the participants in the cohort. All AEs during subcutaneous
administration with the 100 mg weekly dose were mild in
severity.
- The ISR rate for all participants in the 4 cohorts was 59% and
all were mild in severity. There were no serious AEs reported for
participants receiving study treatment.
- A participant with a history of hepatitis A receiving the 150
mg dose experienced liver enzyme test elevation that resulted in
discontinuation and a reduction in the dose for the 3rd cohort from
150 mg to 125 mg.
- The measurement of anti-drug antibody (ADA) formation in the
study did not demonstrate an effect on PK or PD parameters and did
not appear to be associated with an effect on AE incidence or
severity.
“The preliminary results from this Phase 1 multiple ascending
dose study of RLYB116 support continued development in patients
with gMG,” said Eric Watsky, M.D., RLYB116 Program Lead for
Rallybio. “We are encouraged by the free C5 reductions demonstrated
by RLYB116 as well as the exposures achieved with subcutaneous
administration. Through enhancements in the manufacturing process,
we believe we have the opportunity to increase the dose of RLYB116
and improve the tolerability thereby opening up the opportunity to
treat a wider range of complement mediated diseases. Our market
research is consistent with our belief that an effective,
once-a-week, well-tolerated therapy that can be rapidly
self-administered with an autoinjector would be an attractive
alternative for patients.”
RLYB116 Near-Term Development Plans and Cash Runway
The preliminary data from the Phase 1 MAD study confirm
improvements made to date in the manufacturing process will enable
the Company to advance RLYB116 into studies in patients. Rather
than immediately proceed to a Phase 2 study in gMG, the Company
intends to prioritize near-term investments in RLYB116 in the
manufacturing process. The Company expects that the additional
manufacturing work will improve tolerability at higher doses with a
low injection volume and infrequent subcutaneous administration.
The Company believes such enhancements will enable higher exposure
to RLYB116 and potentially increase C5 reduction, which can result
in treating a broader range of complement-mediated diseases,
including paroxysmal nocturnal hemoglobinuria and antiphospholipid
syndrome. In addition, this will allow the Company to direct
available cash resources to advance RLYB212, its product candidate
for the prevention of fetal and neonatal alloimmune
thrombocytopenia (FNAIT).
The Company is also updating its cash runway guidance and now
expects its current cash, cash equivalents and marketable
securities to extend the runway into the third quarter of 2025.
“We are pleased to see substantial reductions in free C5 with
once weekly subcutaneous dosing of RLYB116,” said Stephen Uden,
M.D., Chief Executive Officer of Rallybio. “The Phase 1 MAD data
show us that RLYB116 can be a potential therapeutic to treat gMG
and other complement mediated diseases. In the spirit of managing
our cash runway to realize the most value from our portfolio, we
have decided to focus our RLYB116 investments on the manufacturing
process with a goal of expanding the scope of therapeutic
indications and addressing unmet medical need. In parallel, we
continue to advance our lead program, RLYB212, and have extended
our runway into the third quarter of 2025.”
Conference Call Information
Rallybio will host a conference call and webcast today, December
20, 2023 at 8:30 a.m. Eastern Time to discuss the RLYB116 Phase 1
multiple ascending dose (MAD) study. The live webcast and replay
may be accessed by visiting Rallybio’s website at
http://investors.rallybio.com. In addition, key slides from the
RLYB116 Phase 1 MAD study will be discussed on the conference call
and are posted to the “Events and Presentations” section of the
Rallybio website. A replay of the webcast will be available on the
Rallybio website for 30 days following the event.
About RLYB116 Phase 1 Study
RLYB116 is an innovative, long-acting, subcutaneously injected
inhibitor of C5 in development for the treatment of patients with
complement-mediated diseases. Phase 1 in healthy participants
included the study of RLYB116 as a single ascending dose and
multiple ascending dose. The multiple ascending dose (MAD) study of
RLYB116 included an adaptive single-blind design initiated in the
first quarter of 2023 with a 4-week treatment duration to evaluate
the safety, tolerability, pharmacokinetics, and pharmacodynamics of
subcutaneous RLYB116 in healthy participants with multiple dose
administration.
The MAD study of RLYB116 included 4 cohorts: Cohort 1 (weekly
dosing of 100 mg), Cohort 2 (3 doses of 100 mg the first week
followed by weekly dosing), Cohort 3 (150 mg weekly dosing reduced
to 125 mg weekly dosing) and Cohort 4 (75 mg twice the first week
followed by 100 mg twice per week).
Post-treatment / study follow-up continued for 10 weeks.
About Rallybio
Rallybio (NASDAQ: RLYB) is a clinical-stage biotechnology
company with a mission to develop and commercialize
life-transforming therapies for patients with severe and rare
diseases. Rallybio has built a broad pipeline of promising product
candidates aimed at addressing diseases with unmet medical need in
areas of maternal fetal health, complement dysregulation,
hematology, and metabolic disorders. The Company has two clinical
stage programs: RLYB212, an anti-HPA-1a antibody for the prevention
of fetal and neonatal alloimmune thrombocytopenia (FNAIT) and
RLYB116, an inhibitor of complement component 5 (C5), with the
potential to treat several diseases of complement dysregulation, as
well as additional programs in preclinical development.
Rallybio is headquartered in New Haven, Connecticut with an
additional facility at the University of Connecticut’s Technology
Incubation Program in Farmington, Connecticut. For more
information, please visit www.rallybio.com and follow us on
LinkedIn and Twitter.
Forward-Looking Statements
This press release contains forward-looking statements that are
based on our management’s beliefs and assumptions and on currently
available information. All statements, other than statements of
historical facts contained in this press release are
forward-looking statements. In some cases, forward-looking
statements can be identified by terms such as “may,” “will,”
“should,” “expect,” “plan,” “anticipate,” “could,” “intend,”
“target,” “project,” “contemplate,” “believe,” “estimate,”
“predict,” “potential” or “continue” or the negative of these terms
or other similar expressions, although not all forward-looking
statements contain these words. Forward-looking statements in this
press release include, but are not limited to, statements
concerning results from the Phase 1 MAD study of RLYB116; potential
clinical effects and benefits of RLYB116, including for the
treatment of gMG; the timing and initiation of future clinical
studies for RLBY116; the success cost and timing of our clinical
development of our product candidates, including RLYB212 and
RLYB116; and statements concerning the Company’s anticipated use of
cash and cash runway. The forward-looking statements in this press
release are only predictions and are based largely on management’s
current expectations and projections about future events and
financial trends that management believes may affect Rallybio’s
business, financial condition and results of operations. These
forward-looking statements speak only as of the date of this press
release and are subject to a number of known and unknown risks,
uncertainties and assumptions, including, but not limited to, our
ability to successfully initiate and conduct our planned clinical
studies, and complete such clinical studies and obtain results on
our expected timelines, or at all, whether our cash resources will
be sufficient to fund our operating expenses and capital
expenditure requirements and whether we will be successful raising
additional capital, our ability to enter into strategic
partnerships or other arrangements, competition from other
biotechnology and pharmaceutical companies, and those risks and
uncertainties described in Rallybio’s filings with the U.S.
Securities and Exchange Commission (SEC), including Rallybio’s
Quarterly Report on Form 10-Q for the period ended September 30,
2023, and subsequent filings with the SEC. The events and
circumstances reflected in our forward-looking statements may not
be achieved or occur and actual future results, levels of activity,
performance and events and circumstances could differ materially
from those projected in the forward-looking statements. Except as
required by applicable law, we are not obligated to publicly update
or revise any forward-looking statements contained in this press
release, whether as a result of any new information, future events,
changed circumstances or otherwise.
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version on businesswire.com: https://www.businesswire.com/news/home/20231219094324/en/
Investors Ami Bavishi Head of Investor Relations and
Communications 475-47-RALLY (Ext. 282) abavishi@rallybio.com
Hannah Deresiewicz Stern Investor Relations, Inc. 212-362-1200
hannah.deresiewicz@sternir.com
Media Jorge Gaeta Mission North (516) 430-7659
Rallybio@missionnorth.com
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