Catabasis Pharmaceuticals Provides Edasalonexent and Rare Disease Pipeline Updates at Investor Day
November 17 2016 - 8:30AM
Business Wire
-- Upcoming Phase 2 Results for Edasalonexent,
a Potential Disease-Modifying Treatment for Duchenne Muscular
Dystrophy (DMD) --
-- Rare Disease Pipeline Now Includes CAT-5571,
a Potential Treatment for Cystic Fibrosis --
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage
biopharmaceutical company, today is holding its first Investor Day
and will provide an in-depth review of the Company’s strategy and
pipeline in rare diseases, including edasalonexent (CAT-1004) and
other programs. Guest speakers will include Craig McDonald, M.D.,
UC Davis NeuroNEXT Program Director, University of California, and
H. Lee Sweeney, Ph.D., Myology Institute Director, University of
Florida.
“At Catabasis, we have been executing relentlessly on our
strategic plan and mission to bring hope and life-changing
therapies to patients and their families suffering from rare
diseases,” said Jill C. Milne, Chief Executive Officer of
Catabasis. “We expect to report top-line safety and efficacy
results from the Phase 2 portion of the MoveDMD® clinical trial in
the first half of Q1 2017 after the JP Morgan conference. Assuming
positive Phase 2 results, we anticipate initiating two additional
clinical trials in DMD next year, including a pivotal Phase 3
trial. In addition to our work in DMD, we are also progressing the
Catabasis rare disease pipeline. We look forward to initiating
clinical work in an additional rare disease for edasalonexent where
NF-kB plays an important role. Further, Catabasis recently expanded
its rare disease pipeline by adding CAT-5571, an activator of
autophagy, as a potential treatment of cystic fibrosis.”
Program Updates and Highlights:
Edasalonexent (CAT-1004) for the Potential Treatment of
DMD
- Top-line safety and efficacy results
from the placebo-controlled portion of the MoveDMD Phase 2 trial
are expected in the first half of Q1 2017.
- MoveDMD Phase 2 trial design: The
primary efficacy end point is change in magnetic resonance imaging
(MRI) T2 for the composite of five lower leg muscles for the pooled
edasalonexent dose groups compared to placebo. Safety and
tolerability will also be evaluated. Additional assessments are
being measured, however the trial is not powered for statistical
significance for these assessments. The additional assessments
include timed function tests (10-meter walk/run, 4-stair climb and
time to stand), muscle strength measures, the North Star Ambulatory
Assessment (NSAA) and the pediatric outcomes data collection
instrument (PODCI).
- The open-label extension for the
MoveDMD trial, in which patients will continue on open-label
edasalonexent for 36 weeks following completion of the 12-week,
placebo-controlled portion of the trial, is ongoing. During the
open-label extension, safety will be monitored and assessments
including MRI, timed function tests, muscle strength measures, the
NSAA and the PODCI, will be performed. Catabasis expects to report
periodic results from the MoveDMD open-label extension in
2017.
- Assuming positive results from the
Phase 2 MoveDMD clinical trial and supportive discussions with
regulatory authorities, Catabasis intends to initiate a Phase 3
placebo-controlled pivotal clinical trial of edasalonexent in
ambulatory boys with DMD aged 4 to 7 in H2 2017. The primary end
point is expected to be one of the age-appropriate timed function
tests included in the Phase 2 trial. The final design of the Phase
3 trial is expected to be informed by the results of the Phase 2
MoveDMD trial as well as the open-label extension data available
prior to the initiation of the Phase 3 trial.
- Assuming positive results from the
Phase 2 MoveDMD clinical trial, Catabasis also intends to initiate
a clinical trial in non-ambulatory patients with DMD in H2
2017.
- There are additional diseases in which
inhibiting NF-kB with edasalonexent may be beneficial. Catabasis
expects to initiate a Phase 2 trial for an additional rare disease
indication for edasalonexent in Q4 2017 or Q1 2018.
Additional Rare Disease Programs
- Preclinical research with CAT-4001 has
continued in diseases such as amyotrophic lateral sclerosis (ALS)
and Friedreich’s ataxia. Ongoing preclinical research for CAT-4001
is expected in 2017.
- CAT-5571 is a potential therapy to
treat cystic fibrosis (CF). CAT-5571, an activator of autophagy, in
combination with lumacaftor/ivacaftor, enhanced cell-surface
trafficking and function of cystic fibrosis transmembrane
conductance regulator (CFTR) in bronchial epithelial cells from CF
patients with the F508del mutation. Catabasis also demonstrated
that CAT-5571 enhanced the clearance of Pseudomonas aeruginosa
infection in preclinical models of CF, irrespective of CFTR
mutation status. Catabasis expects to initiate a Phase 1 trial with
CAT-5571 for the potential treatment of CF in Q4 2017 or Q1
2018.
A live webcast of the presentations is available via the
investor section of the Catabasis website, www.catabasis.com.
Following the live webcast, an archived version will be available
for 90 days.
About Edasalonexent (CAT-1004)Edasalonexent (CAT-1004) is
an oral small molecule that has the potential to be a
disease-modifying therapy for all patients affected by Duchenne
muscular dystrophy (DMD or Duchenne), regardless of their
underlying mutation. Edasalonexent inhibits NF-kB, a protein that
is activated in Duchenne and drives inflammation and fibrosis,
muscle degeneration and suppresses muscle regeneration. In animal
models of DMD, edasalonexent produced beneficial effects in
skeletal, diaphragm and cardiac muscle and improved function. The
FDA has granted orphan drug, fast track and rare pediatric disease
designations and the European Commission has granted orphan
medicinal product designation to edasalonexent for the treatment of
DMD. We have previously reported safety, tolerability and reduction
in NF-kB activity in Phase 1 trials in adults. We are currently
conducting the MoveDMD® trial of edasalonexent in 4-7 year-old boys
affected by Duchenne. From Part A of the MoveDMD trial, we have
reported that edasalonexent was generally well tolerated with no
safety signals observed and we observed NF-kB target
engagement. Pharmacokinetic results demonstrated edasalonexent
plasma exposure levels consistent with those previously observed in
adults, at which inhibition of NF-kB was observed.
About CAT-4001Catabasis is developing CAT-4001 as a
potential treatment for neurodegenerative diseases such as
Friedreich’s ataxia (FA) and amyotrophic lateral sclerosis (ALS).
CAT-4001 is a small molecule that activates Nrf2 and inhibits
NF-kB, two pathways that have been implicated in FA and ALS.
Catabasis has shown that CAT-4001 modulates the Nrf2 and NF-kB
pathways in both cellular assays and animal models.
About CAT-5571Catabasis is developing CAT-5571 as a
potential oral treatment for cystic fibrosis (CF) with potential
effects on both the cystic fibrosis transmembrane conductance
regulator (CFTR) and on the clearance of Pseudomonas aeruginosa.
CAT-5571 is a small molecule that activates autophagy, a process
that maintains cellular homeostasis and host defense mechanisms,
and is known to be impaired in CF. Catabasis has shown in
preclinical studies that CAT-5571, in combination with
lumacaftor/ivacaftor, enhances cell-surface trafficking and
function of CFTR with the F508del mutation. Catabasis has also
shown that CAT-5571 enhances the clearance of P. aeruginosa
infection in preclinical models of CF, irrespective of CFTR
mutation status.
About CatabasisAt Catabasis Pharmaceuticals, our mission
is to bring hope and life-changing therapies to patients and their
families. Our SMART (Safely Metabolized And Rationally Targeted)
linker drug discovery platform enables us to engineer molecules
that simultaneously modulate multiple targets in a disease. We are
applying our SMART linker platform to build an internal pipeline of
product candidates for rare diseases and plan to pursue
partnerships to develop additional product candidates. For more
information on the Company's drug discovery platform and pipeline
of drug candidates, please visit www.catabasis.com.
Forward Looking StatementsAny statements in this press
release about future expectations, plans and prospects for the
Company, including statements about future clinical trial plans and
other statements containing the words “believes,” “anticipates,”
“plans,” “expects,” “may” and similar expressions, constitute
forward-looking statements within the meaning of the Private
Securities Litigation Reform Act of 1995. Actual results may differ
materially from those indicated by such forward-looking statements
as a result of various important factors, including: uncertainties
inherent in the initiation and completion of preclinical studies
and clinical trials and clinical development of the Company’s
product candidates; availability and timing of results from
preclinical studies and clinical trials; whether interim results
from a clinical trial will be predictive of the final results of
the trial or the results of future trials; expectations for
regulatory approvals to conduct trials or to market products;
availability of funding sufficient for the Company’s foreseeable
and unforeseeable operating expenses and capital expenditure
requirements; other matters that could affect the availability or
commercial potential of the Company’s product candidates; and
general economic and market conditions and other factors discussed
in the “Risk Factors” section of the Company’s Quarterly Report on
Form 10-Q for the period ended September 30, 2016, which is on file
with the Securities and Exchange Commission, and in other filings
that the Company may make with the Securities and Exchange
Commission in the future. In addition, the forward-looking
statements included in this press release represent the Company’s
views as of the date of this press release. The Company anticipates
that subsequent events and developments will cause the Company’s
views to change. However, while the Company may elect to update
these forward-looking statements at some point in the future, the
Company specifically disclaims any obligation to do so. These
forward-looking statements should not be relied upon as
representing the Company’s views as of any date subsequent to the
date of this release.
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version on businesswire.com: http://www.businesswire.com/news/home/20161117005455/en/
Catabasis Pharmaceuticals, Inc.Andrea Matthews,
617-349-1971amatthews@catabasis.com
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