-- 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 --
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today reported financial results for the second quarter ended June 30, 2017, and reviewed recent business progress.
“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 Jill C. Milne, Ph.D., Chief Executive Officer of Catabasis.
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 Neurology 69th 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 Conference in 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 June 30, 2017, Catabasis had cash and cash equivalents of $29.4 million, compared to $31.8 million in cash, cash equivalents and available-for-sale securities as of March 31, 2017. Catabasis expects that its current operating plan provides for cash to fund operations through August, 2018. Net cash used in operating activities for the three months ended June 30, 2017 was $5.7 million, compared to $8.8 million for the three months ended June 30, 2016. Net cash used in operating activities for the six months ended June 30, 2017 was $13.8 million, compared to $18.0 million for the six months ended June 30, 2016.
R&D Expenses: Research and development expenses were $4.5 million for the three months ended June 30, 2017, compared to $6.8 million for the three months ended June 30, 2016 and $9.9 million for the six months ended June 30, 2017, compared to $13.3 million for the six months ended June 30, 2016. The decrease in research and development expenses was primarily attributable to the completion of certain clinical activities.
G&A Expenses: General and administrative expenses were $2.4 million for the three months ended June 30, 2017, compared to $2.6 million for the three months ended June 30, 2016 and $4.8 million for the six months ended June 30, 2017, compared to $5.3 million for the six months ended June 30, 2016. The decrease in general and administrative expenses was primarily attributable to headcount reductions.
Operating Loss: Loss from operations was $6.9 million for the three months ended June 30, 2017, compared to $9.4 million for the three months ended June 30, 2016, and $14.7 million for the six months ended June 30, 2017, compared to $18.6 million for the six months ended June 30, 2016.
Net Loss: Net loss was $7.0 million, or $0.32 per share, for the three months ended June 30, 2017, compared to a net loss of $9.4 million, or $0.61 per share, for the three months ended June 30, 2016. Net loss for the six months ended June 30, 2017 was $14.9 million, compared to $18.9 million for the six months ended June 30, 2016.
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.
