Study Results

• In the MoveDMD trial, a considerable slowing of disease progression of DMD was noted in boys treated with Edasalonexent versus the rates of change during the control period.

• In the 100-mg/kg/day-treatment group, 16 boys started Edasalonexent either at the beginning of Phase-2 or at the beginning of the open-label extension. In the open-label extension data analysis, all 14 boys continuing to participate had received 100 mg/kg/day for 24 weeks and 11 had completed 36 weeks of 100 mg/kg/day Edasalonexent treatment. Results are exhaustive for the 100-mg/kg/day-treatment group as all boys are now taking this dose in the open-label extension.

• Moreover, additional supportive measures of muscle health were also in-line with a positive Edasalonexent treatment effect. After Edasalonexent treatment for 12 weeks, the four muscle enzymes – i.e. creatine kinase, alanine aminotransferase, aspartate aminotransferase, and lactate dehydrogenase - were considerably reduced compared to baseline. This was coupled with the ability to slow muscle degeneration and improve muscle integrity. Furthermore, rate of change in lower leg MRI T2 considerably improved at 12 weeks and at last observation on treatment compared to control period (p≤0.05). This was also matched with a reduction of muscle inflammation.

No Significant Adverse Effects

In the MoveDMD trial, Edasalonexent continued to be well tolerated with no safety signals observed so far.

• Most of the Adverse Effects (AE) have been mild in nature while there have been no dose reductions and no drug-related discontinuations.

• In fact, the height, weight, and BMI growth patterns were in-line with the standard growth curves for unaffected boys in the age range of MoveDMD subjects.

• The most common AEs include gastrointestinal, primarily mild and transient diarrhea.

• Boys suffering with DMD in this age group mostly have resting tachycardia, i.e. their heart rate that exceeds the normal resting rate. Providentially, the heart rate of the boys treated with Edasalonexent decreased toward age-normative values during treatment. These clinical heart rate observations are intriguing and need follow-up.

Affirmative Remarks for Positive Results of MoveDMD Trials

Jill C. Milne, Ph.D., Chief Executive Officer of Catabasis expressed his excitement on seeing Edasalonexent change the course of disease in the MoveDMD trial with considerably slowed disease progression. He shared that, boys who were treated with Edasalonexent showed significant improvements in muscle function as compared to the rates of change observed during the control period. Other supportive positive measures of muscle health were also noted. He mentioned that Catabasis looks forward to developing Edasalonexent as a disease-modifying therapy in a single Phase-3 pivotal trial as soon as possible. The Company aims to deliver noteworthy impact on disease progression for all boys affected by Duchenne.

Richard Finkel, M.D., Chief, Division of Neurology, Department of Pediatrics at Nemours Children's Health System, shared his opinion about the positive results of Phase-2 Trial. He is serving as the Principal Investigator for the study. He stated that their goal in treating boys with Duchenne is to slow the progression of the disease. It is extremely reassuring to see Edasalonexent have a stabilizing effect on boys. Those treated with Edasalonexent demonstrated improvements in functional abilities and MRI T2 measures along with its continued safety profile. He plans to continue investigating Edasalonexent as a potential therapy for all boys suffering from Duchenne.

Pat Furlong, Founding President and Chief Executive Officer of Parent Project Muscular Dystrophy (PPMD), also expressed his excitement on noting positive effects of Edasalonexent on muscle function. Research suggests that effects on muscle function are the most important aspect of a therapy for Duchenne for the affected patients as well as their families. Pat Furlong is eager to know more about Edasalonexent as Catabasis is about to commence its Phase-3 trial. He believes given its disease-modifying effects and safety and tolerability profile, Edasalonexent has the potential to become the foundational therapy for all people affected by Duchenne.

Plan for a Single Global Phase-3 Trial

On the basis of the positive MoveDMD results and supportive regulatory input from FDA, Catabasis intends to initiate a single global Phase-3 trial with Edasalonexent in patients with DMD irrespective of mutation type in the first half of 2018 with top-line results expected in 2020.

The Phase-3 trial would help evaluate the efficacy and safety of Edasalonexent for registration purposes. Catabasis has already discussed and planned the design for the randomized, double-blind, placebo-controlled trial with the FDA. The Phase-3 trial would have many elements in common with the Phase-2 trial including the patient population and endpoints. It is expected that approximately 125 patients aged 4 to 7 years, who have not been on steroids for at least 6 months, would be enrolled for the trial. The primary endpoint would be a change in the North Star Ambulatory Assessment score after 12 months of Edasalonexent treatment compared to placebo. Key secondary endpoints would include age-appropriate timed function tests.

Last Close Stock Review

On Thursday, October 05, 2017, the stock closed the trading session at $2.84, declining 6.27% from its previous closing price of $3.03. A total volume of 7.52 million shares have exchanged hands, which was higher than the 3-month average volume of 837.51 thousand shares. Catabasis Pharma's stock price skyrocketed 89.33% in the last one month, 113.53% in the past three months, and 110.37% in the previous six months. The stock currently has a market cap of $67.11 million.

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