Catabasis Pharmaceuticals, Inc. reported financial results for the fourth quarter and full year ended December 31, 2017, and reviewed recent business progress.
“We achieved several important clinical milestones in 2017 as we work toward our vision of improving the lives of those affected by rare diseases,” said Jill C. Milne, Ph.D., Chief Executive Officer of Catabasis. “Edasalonexent demonstrated consistent and sustained disease-modifying effects in boys with Duchenne muscular dystrophy through more than a year of treatment compared to control without evidence of adverse effects associated with current standard of care. We also made progress in the evaluation of CAT-5571, a potential oral treatment for cystic fibrosis. In 2018, we are focused on advancing our edasalonexent program into Phase 3 and bringing our cystic fibrosis program into the clinic.”
Recent and Upcoming Corporate Highlights
Edasalonexent (CAT-1004) for the Treatment of Duchenne Muscular Dystrophy (DMD)
Consistent and sustained disease-modifying effects of edasalonexent following 48 and 60 weeks of treatment in boys with DMD were reported at the XVI International Conference on Duchenne and Becker Muscular Dystrophy in February 2018.
- Preservation of muscle function and substantially slowed DMD disease progression compared to the pre-specified control period was observed through more than a year of edasalonexent treatment. Consistent improvements in all assessments of muscle function were observed following 48 and 60 weeks of oral 100 mg/kg/day edasalonexent treatment compared to the rates of change in the control period for boys prior to receiving edasalonexent treatment.
- Statistically significant changes from baseline in multiple non-effort based biomarkers of muscle health were seen.
- Edasalonexent continued to be well tolerated with no safety signals observed.
Additional data on edasalonexent were presented this week at the 2018 Muscular Dystrophy Association Clinical Conference.
- Height and weight growth following 48 and 60 weeks of edasalonexent treatment was age-appropriate and on track with standard growth curves for unaffected boys in the same age range. Body mass index (BMI) trended towards a decrease. This profile is favorably differentiated from the typical profile associated with the corticosteroid standard of care in DMD which includes weight gain and curtailed growth.
- Heart rate data from boys treated with edasalonexent decreased toward age-normative values through 48 weeks of treatment. Cardiac failure is a leading cause of mortality in DMD. In the 4-7 year old age range, boys typically have resting tachycardia, a heart rate that exceeds the normal resting rate, which is the first cardiac manifestation in DMD.
Also presented at the 2018 Muscular Dystrophy Association Clinical Conference were new data from the ImagingDMD natural history study, a collaboration led by the University of Florida and independent of the MoveDMD trial.
- The ImagingDMD study annually assessed timed function tests, and a subset analysis was presented of 28 boys initially aged 5 to 8.5 years old at times when they were not taking corticosteroids. The timed function tests performed in the study were the same as those assessed in the MoveDMD trial, which was conducted at ImagingDMD sites.
- The observations in the ImagingDMD natural history study were generally consistent with the absolute functional abilities as well as declines in abilities experienced by boys in the off-treatment control period of the Catabasis MoveDMD trial. These data provide important corroboration that the MoveDMD off-treatment control period observations are characteristic of the expected natural history and provide added confidence in the slowing of disease progression treatment effects observed with edasalonexent.
Catabasis plans to initiate a 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, dependent on raising capital.
CAT-5571 for the Treatment of Cystic Fibrosis (CF)
Data demonstrating CAT-5571 improved cellular clearance of bacteria in preclinical models of CF were presented at the North American Cystic Fibrosis Conference in November 2017. CAT-5571 enhanced the clearance of the opportunistic and often fatal pathogen Burkholderia cenocepacia.
Catabasis expects to initiate a Phase 1 trial for CAT-5571 in the second half of 2018 and report top-line results in 2019, based on our current operating plan. This is supported by preclinical data showing CAT-5571 enhances the clearance of multiple types of pathogens.
Fourth Quarter and Full Year 2017 Financial Results
Cash Position: As of December 31, 2017, Catabasis had cash and cash equivalents of $16.4 million, compared to $21.7 million as of September 30, 2017 and $38.5 million in cash, cash equivalents and available-for-sale securities as of December 31, 2016. Following December 31, 2017, Catabasis raised an additional $8.3 million in net proceeds under an at-the-market offering program. Catabasis’ current operating plan provides for cash to fund operations through September 2018. To advance edasalonexent in the Phase 3 trial and CAT-5571 in the Phase 1 trial, Catabasis expects to seek additional funds through equity or debt financings and/or through partnering or licensing transactions. Net cash used in operating activities for the three months ended December 31, 2017 was $5.6 million, compared to $8.0 million for the three months ended December 31, 2016. Net cash used in operating activities for the full year 2017 was $26.8 million, compared to $32.9 million for the full year 2016. Recognized revenue for the three months ended December 31, 2017 was $0.3 million and $0.5 million for the full year 2017 from an option agreement with an unaffiliated party.
R&D Expenses: Research and development expenses were $4.0 million for the three months ended December 31, 2017, compared to $6.3 million for the three months ended December 31, 2016 and $18.7 million for the full year 2017, compared to $25.5 million for the full year 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 $1.7 million for the three months ended December 31, 2017, compared to $2.4 million for the three months ended December 31, 2016 and $8.9 million for the full year 2017, compared to $10.1 million for the full year 2016.
Operating Loss: Loss from operations was $5.5 million for the three months ended December 31, 2017, compared to $8.7 million for the three months ended December 31, 2016, and $27.1 million for the full year 2017, compared to $35.6 million for the full year 2016.
Net Loss: Net loss was $5.5 million, or $0.24 per share, for the three months ended December 31, 2017, compared to a net loss of $8.8 million, or $0.47 per share, for the three months ended December 31, 2016. Net loss for the full year 2017 was $27.4 million, or $1.26 per share, compared to $36.1 million for the full year 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 fourth quarter and full year 2017 financial results.
| Participant Toll-Free Dial-In Number: | (877) 388-2733 | |||
| Participant International Dial-In Number: | (541) 797-2984 | |||
| Pass Code: | 7693708 | |||
Please specify to the operator that you would like to join the “Catabasis Fourth Quarter and Full Year 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. Edasalonexent continues to be dosed in the open-label extension of the MoveDMD Phase 2 clinical trial and 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, dependent on raising capital. 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. For a summary of clinical results reported to-date, please visit www.catabasis.com.
About CAT-5571
CAT-5571 is an investigational oral small molecule designed to restore host defense by activating autophagy that is being developed for the treatment of cystic fibrosis (CF). Autophagy is a mechanism for recycling cellular components and digesting pathogens, which is depressed in CF. People with CF suffer from persistent lung infections with opportunistic pathogens such as Pseudomonas aeruginosa and Burkholderia cenocepacia, causing chronic infections that are difficult to eradicate and lead to respiratory failure. CAT-5571 has been shown to restore autophagy, reestablish host defense and enhance the clearance of pathogens, including P. aeruginosa and B. cenocepacia, in preclinical models of CF. 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.
