Catabasis Pharmaceuticals Reports Third Quarter 2017 Financial Results and Reviews Business Progress
-- Edasalonexent Substantially Slowed Duchenne Muscular Dystrophy Disease Progression Through 36 Weeks; Plan to Initiate Phase 3 Trial in the First Half of 2018 --
-- Additional Preclinical Data Demonstrate Potential of CAT-5571 as a Treatment for Cystic Fibrosis; Plan to Initiate Phase 1 Trial in the Second Half of 2018 --
“We have achieved an important milestone for the edasalonexent program,”
said
Dr. Milne continued, “Our team also advanced our research of CAT-5571, a potential oral treatment for cystic fibrosis-associated respiratory infections, which are the leading cause of morbidity and mortality for patients with cystic fibrosis. We expect to initiate a Phase 1 trial for CAT-5571 in the second half of 2018.”
Recent and Upcoming Corporate Highlights
Edasalonexent (CAT-1004) for the Treatment of Duchenne Muscular Dystrophy (DMD)
-
In the MoveDMD Phase 2 trial and open-label extension, sustained
disease-modifying effects were seen through 36 weeks of treatment with
edasalonexent. Across all assessments of muscle function, prespecified
analyses showed improvements in the rate of decline after 24 and 36
weeks of oral 100 mg/kg/day edasalonexent treatment in the open-label
extension compared to the rate of change in the control period for
boys prior to receiving edasalonexent treatment. The totality of these
data provides clinically meaningful evidence that edasalonexent
substantially slowed the progression of DMD. Additionally,
statistically significant changes in supportive measures of muscle
health were seen in muscle enzymes and the magnetic resonance imaging
(MRI) T2 composite measure of lower leg muscles. Edasalonexent
continued to be well tolerated with no safety signals observed in the
trial. These data were presented at the
World Muscle Society Conference in October. - New biomarker results from the MoveDMD Phase 2 trial and open-label extension showed that C-reactive protein (CRP) was significantly decreased with edasalonexent at 12 and 24 weeks compared to baseline in the 100 mg/kg/day treatment group. CRP is a well-characterized blood test marker that provides a global assessment of inflammation. CRP is elevated in boys affected by DMD. The significant decrease observed in CRP supports the biological activity of NF-kB inhibition by edasalonexent treatment decreasing inflammation.
-
Based on the consistency of the MoveDMD results and supportive
regulatory input from
FDA , Catabasis plans to initiate a single global Phase 3 trial with edasalonexent in patients with DMD regardless of mutation type in the first half of 2018 with top-line results expected in 2020.
CAT-5571 for the Treatment of Cystic Fibrosis (CF)
-
In preclinical models of CF, CAT-5571 improved cellular clearance of
the opportunistic and often fatal pathogen Burkholderia cenocepacia
as reported at the
North American Cystic Fibrosis Conference in November. This activity has the potential to address cystic fibrosis-associated respiratory infections by enhancing the clearance of pathogens, including Pseudomonas aeruginosa and Burkholderia cenocepacia, which are the leading cause of morbidity and mortality for patients with CF. CAT-5571 is designed to restore host defense by activating autophagy, which is known to be depressed in CF. - Catabasis expects to initiate a Phase 1 trial for CAT-5571 in the second half of 2018 and report top-line results in 2019.
Third Quarter 2017 Financial Results
Cash Position: As of
R&D Expenses: Research and development expenses were
G&A Expenses: General and administrative expenses were
Operating Loss: Loss from operations was
Net Loss: Net loss was
Conference Call and Webcast
Catabasis will host a conference
call and webcast at 4:30pm ET today to provide an update on corporate
developments and to discuss third quarter 2017 financial results.
Participant Toll-Free Dial-In Number: | (877) 388-2733 | ||
Participant International Dial-In Number: | (541) 797-2984 | ||
Pass Code: | 99108456 | ||
Please specify to the operator that you would like to join the “Catabasis Third Quarter 2017 Results Call.”
Interested parties may access a live audio webcast of the conference call via the investor section of the Catabasis website, www.catabasis.com. Please connect to the Catabasis website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary. The webcast will be archived for 90 days.
About Edasalonexent (CAT-1004)
Edasalonexent (CAT-1004) is
an investigational oral small molecule that is being developed as a
potential disease-modifying therapy for all patients affected by DMD,
regardless of their underlying mutation. Edasalonexent inhibits NF-kB, a
protein that is activated in DMD and drives inflammation and fibrosis,
muscle degeneration and suppresses muscle regeneration. In the Phase 2
and open-label extension of the MoveDMD trial investigating the safety
and efficacy of edasalonexent in boys enrolled at ages 4 to 7 affected
with DMD (any confirmed mutation), edasalonexent substantially slowed
DMD disease progression through 36 weeks of treatment. Across all key
assessments of muscle function, consistent improvements were observed in
the rate of decline after 24 and 36 weeks of oral 100 mg/kg/day
edasalonexent treatment compared to the rate of change in the control
period for boys prior to receiving edasalonexent treatment. Improvements
were also seen across measures of muscle health. Edasalonexent continued
to be well tolerated with no safety signals observed in the trial.
Catabasis plans to initiate a single global Phase 3 trial to evaluate
the efficacy and safety of edasalonexent for registration purposes in
the first half of 2018. The
About CAT-5571
CAT-5571 is an investigational oral small
molecule that is being developed as a potential host-directed therapy
for cystic fibrosis. CAT-5571 is designed to restore host defense by
activating autophagy, a mechanism for recycling cellular components and
digesting pathogens. Autophagy is depressed in CF, and by restoring
autophagy, CAT-5571 reestablishes host defense to enhance the clearance
of pathogens, including Pseudomonas aeruginosa and Burkholderia
cenocepacia, in preclinical models of CF. People with CF suffer from
persistent lung infections with opportunistic pathogens such as P.
aeruginosa and B. cenocepacia, causing chronic
infections that are difficult to eradicate and lead to respiratory
failure. CAT-5571 has the potential to augment the efficacy of
antibiotics and could also be used with other CF therapies, including
transmembrane conductance receptor (CFTR) targeted agents.
About Catabasis
At
Forward Looking Statements
Any statements in this press
release about future expectations, plans and prospects for the Company,
including statements about future clinical trial plans including, among
other things, statements about the Company’s plans to commence a single
global Phase 3 trial in DMD in the first half of 2018 to evaluate the
efficacy and safety of edasalonexent for registration purposes, the
Company’s plans to report top-line results from this trial in 2020, the
Company’s plans to initiate a Phase 1 trial for CAT-5571 in the second
half of 2018 and report top-line results in 2019 and the Company’s
expectation that its current operating plan provides for cash to fund
operations through
Catabasis Pharmaceuticals, Inc. | ||||||||||||||||
Condensed Consolidated Statements of Operations | ||||||||||||||||
(In thousands, except share and per share data) |
||||||||||||||||
(Unaudited) |
||||||||||||||||
Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||
2017 | 2016 | 2017 | 2016 | |||||||||||||
Revenue | $ | 250 | $ | - | $ | 250 | $ | - | ||||||||
Operating expenses: | ||||||||||||||||
Research and development | 4,776 | 5,936 | 14,693 | 19,190 | ||||||||||||
General and administrative | 2,426 | 2,347 | 7,189 | 7,695 | ||||||||||||
Total operating expenses | 7,202 | 8,283 | 21,882 | 26,885 | ||||||||||||
Loss from operations | (6,952) | (8,283) | (21,632) | (26,885) | ||||||||||||
Other (expense) income: | ||||||||||||||||
Interest expense | (105) | (199) | (381) | (662) | ||||||||||||
Interest and investment income | 45 | 50 | 128 | 183 | ||||||||||||
Other (expense) income, net | (5) | 13 | 18 | 82 | ||||||||||||
Total other expense, net | (65) | (136) | (235) | (397) | ||||||||||||
Net loss | $ | (7,017) | $ | (8,419) | $ | (21,867) | $ | (27,282) | ||||||||
Net loss per share - basic and diluted | $ | (0.31) | $ | (0.54) | $ | (1.03) | $ | (1.77) | ||||||||
Weighted-average common shares outstanding used in net loss per share - basic and diluted | 22,563,174 | 15,512,608 | 21,163,591 | 15,407,747 | ||||||||||||
Catabasis Pharmaceuticals, Inc. | ||||||||
Condensed Consolidated Balance Sheets | ||||||||
(In thousands) |
||||||||
(Unaudited) |
||||||||
September 30, | December 31, | |||||||
2017 | 2016 | |||||||
Assets | ||||||||
Cash and cash equivalents | $ | 21,713 | $ | 23,596 | ||||
Available-for-sale securities | - | 14,931 | ||||||
Total assets | 23,313 | 40,209 | ||||||
Liabilities and stockholders’ equity | ||||||||
Current portion of notes payable, net of discount | 3,296 | 3,243 | ||||||
Notes payable, net of current portion and discount | - | 2,479 | ||||||
Total liabilities | 7,656 | 11,123 | ||||||
Total stockholders’ equity | $ | 15,657 | $ | 29,086 | ||||
Catabasis Pharmaceuticals, Inc. | ||||||||||
Condensed Consolidated Statements of Cash Flows | ||||||||||
(In thousands) |
||||||||||
(Unaudited) |
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Nine Months Ended September 30, | ||||||||||
2017 | 2016 | |||||||||
Net cash used in operating activities | $ | (21,199 | ) | $ | (24,874 | ) | ||||
Net cash provided by (used in) investing activities | 14,883 | (21,300 | ) | |||||||
Net cash provided by financing activities | 4,433 | 9,858 | ||||||||
Net decrease in cash and cash equivalents | $ | (1,883 | ) | $ | (36,316 | ) | ||||
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Source:
Investor and Media Contact
Catabasis
Pharmaceuticals, Inc.
Andrea Matthews, 617-349-1971
amatthews@catabasis.com