Catabasis Pharmaceuticals Reports Second Quarter 2017 Financial Results and Reviews Business Progress
-- Favorable Results Seen in 12-week Edasalonexent Phase 2 MoveDMD® Trial in Duchenne Muscular Dystrophy; Phase 3 Clinical Trial Plan Expected Second Half of 2017 --
-- Preclinical Data Support CAT-5571 as a Potential Treatment to Enhance Host Defenses by Restoring Autophagy in Cystic Fibrosis --
“In the second quarter, we presented an important prespecified crossover
analysis of data from boys with Duchenne in our Phase 2 edasalonexent
trial. We are very excited to see improvements in the rate of decline of
muscle function across multiple assessments in boys treated with
edasalonexent for 12 weeks. The results are very consistent with and
support our earlier analysis of functional assessments in boys treated
with edasalonexent for 12 weeks as compared to placebo and further
strengthen our confidence in the potential of edasalonexent as a novel
treatment for DMD. We continue to advance the open-label extension and
expect to share 24-week edasalonexent results in the third quarter as
well as announce our Phase 3 clinical trial plan for edasalonexent in
the second half of 2017,” said
Dr. Milne continued, “We are also encouraged by the results of our preclinical studies of CAT-5571, supporting its potential as an oral treatment for cystic fibrosis, and with our progress across our research programs driven by our SMART Linker℠ drug discovery technology.”
Recent and Upcoming Corporate Highlights
Edasalonexent (CAT-1004) for the Treatment of Duchenne Muscular Dystrophy (DMD)
In the MoveDMD Phase 2 edasalonexent trial, a crossover analysis of
the rates of change across five assessments of muscle function in
patients after 12 weeks of treatment compared to off-treatment prior
to Phase 2 dosing showed clinically meaningful numerical improvements
in rates of decline. The analysis was presented at the
American Academy of Neurology69th Annual Meeting in April and was the second of two prespecified analyses of the 12-week data. The first analysis was presented in March and showed meaningful improvements in assessments of muscle function in boys treated with edasalonexent compared to placebo. Functional assessments have precedence as endpoints in pivotal trials in DMD. The MoveDMD 12-week Phase 2 results are consistent with the therapeutic goal of treatment, to delay the predictable, sequential loss of function in DMD. There were no safety signals and edasalonexent was well tolerated in this study.
- The open-label extension of the MoveDMD trial is progressing as planned and results from 24 weeks of edasalonexent treatment are expected to be announced in the third quarter of 2017. All boys participating in the open-label extension have now moved to the higher 100 mg/kg/day edasalonexent treatment group. Pending IRB approval, the open-label extension will be extended for an additional 52 weeks so that participating boys can continue to receive edasalonexent.
- Catabasis expects to announce the Phase 3 clinical trial plan for edasalonexent in DMD in the second half of 2017.
- The first boy in the MoveDMD trial who is amenable to exon 51 skipping treatment has started EXONDYS 51™ treatment along with edasalonexent in the open-label extension. The Catabasis and Sarepta joint research collaboration previously showed increased dystrophin expression in the mdx mouse with edasalonexent in combination with an exon-skip modality. Edasalonexent may have the potential to increase dystrophin levels in combination with dystrophin-targeted therapies.
CAT-5571 for the Treatment of Cystic Fibrosis (CF)
CAT-5571 demonstrated in preclinical studies improved intracellular
clearance of bacterial pathogens that are clinically important in CF,
as reported at the
European Cystic Fibrosis Society Conferencein June. This activity has the potential to improve lung function by reducing the intracellular load of multiple types of bacteria, including the pathogens, Pseudomonas aeruginosa and Burkholderia cenocepacia, which are the leading cause of morbidity and mortality for patients with CF. CAT-5571 restores autophagy, a host defense mechanism, which is known to be impaired in CF. Catabasis expects to initiate a Phase 1 trial for CAT-5571 in 2018.
Second 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 second quarter 2017 financial results.
|Participant Toll-Free Dial-In Number:||(877) 388-2733|
|Participant International Dial-In Number:||(541) 797-2984|
Please specify to the operator that you would like to join the “Catabasis Second 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. We are currently conducting the MoveDMD trial, a three-part clinical trial investigating the safety and efficacy of edasalonexent in boys enrolled at ages 4 – 7 affected with DMD (any confirmed mutation). The third part of the trial, an open-label extension with edasalonexent, is ongoing. The
Catabasis is developing CAT-5571 as a potential oral treatment for CF with potential effects on both the cystic fibrosis transmembrane conductance regulator (CFTR) and on the clearance of multiple types of bacteria including 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 observed 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 observed that CAT-5571 enhances the clearance of P. aeruginosa infection in preclinical models of CF.
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 our plans to identify, develop and commercialize novel therapeutics based on our SMART Linker drug discovery platform, our plans to continue to evaluate data from the open-label extension of our MoveDMD® clinical trial of edasalonexent for the treatment of DMD, our plans for ongoing and planned clinical trials for edasalonexent and other product candidates, whether conducted by us or by any future collaborators, including the timing of initiation of these trials and of the anticipated results, 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
Catabasis Pharmaceuticals, Inc.
|Three Months Ended June 30,||Six Months Ended June 30,|
|Research and development||$||4,519||$||6,818||$||9,917||$||13,254|
|General and administrative||2,400||2,578||4,763||5,348|
|Total operating expenses||6,919||9,396||14,680||18,602|
|Loss from operations||(6,919||)||(9,396||)||(14,680||)||(18,602||)|
|Other (expense) income:|
|Interest and investment income||44||80||83||133|
|Other income, net||28||91||23||69|
|Total other expense, net||(55||)||(49||)||(170||)||(261||)|
|Net loss per share - basic and diluted||$||(0.32||)||$||(0.61||)||$||(0.73||)||$||(1.23||)|
Weighted-average common shares outstanding
Catabasis Pharmaceuticals, Inc.
|Cash and cash equivalents||$||29,369||$||23,596|
|Liabilities and stockholders’ equity|
|Current portion of notes payable, net of discount||3,278||3,243|
|Notes payable, net of current portion and discount||831||2,479|
|Total stockholders’ equity||$||21,550||$||29,086|
Catabasis Pharmaceuticals, Inc.
|Six Months Ended June 30,|
|Net cash used in operating activities||$||(13,785||)||$||(17,976||)|
|Net cash provided by (used in) investing activities||14,901||(18,971||)|
|Net cash provided by (used in) financing activities||4,657||(1,555||)|
|Net increase (decrease) in cash and cash equivalents||$||5,773||$||(38,502||)|
Investor and Media Contact
Catabasis Pharmaceuticals, Inc.
Andrea Matthews, 617-349-1971