May is ALS Awareness Month. Learn more about what you can do to end ALS with MDA.

About Us

Our mission is to empower people living with neuromuscular diseases to live longer, more independent lives.

Catabasis Pharmaceuticals Presents Positive CAT-1004 Data from Part A of the MoveDMDSM Trial at the 2016 Muscular Dystrophy Association Clinical Conference

Full Release:

- Trial Demonstrated Favorable Safety, Tolerability and Pharmacokinetics in Patients –

- Results Support Initiation of Part B of Trial, Expected in First Half of 2016 –

CAMBRIDGE, MA, March 21, 2016 – Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced positive data from Part A of the MoveDMD trial of CAT-1004 for Duchenne muscular dystrophy (DMD). The objectives for Part A were to establish safety, tolerability and pharmacokinetics in boys with DMD age 4 to 7 to support initiation of Part B of the MoveDMD trial, a 12-week trial to assess efficacy. All three doses of CAT-1004 tested were generally well tolerated with no safety signals observed. The majority of adverse events were mild in nature, and the adverse events observed were diarrhea (4 events),soft feces (3 events) and upper abdominal pain (2 events). There were no serious adverse events and no drug discontinuations. Six of the total of 9 adverse events were observed in the highestdose group. There were no trends or safety issues in labs, physical exams, EKG or vital signs.

From the pharmacokinetics data, we conclude that doses of 33 mg/kg given 2 or 3 times daily are predicted to achieve systemic exposures at which NF-kB inhibition was observed in adults. Pharmacokinetics demonstrated dose-dependent increases in exposure and meal composition had a modest effect. With single doses of 33 mg/kg, there were minimal differences in either area under the curve (AUC) or maximum serum concentration (Cmax) when CAT-1004 was administered either with a high-fat or a low-fat meal. Based on these results, the daily doses selected for Part B will be 67 or 100 mg/kg administered with food at 33 mg/kg either 2 or 3 times daily. From these results, Catabasis plans to initiate Part B of the MoveDMD trial in the first half of 2016, subject to regulatory approval of the proposed protocol. The Parent Project Muscular Dystrophy and the Muscular Dystrophy Association are providing funding to support participant travel for the MoveDMD trial.

“The favorable safety, tolerability and pharmacokinetic results in boys affected by DMD are encouraging and we look forward to starting Part B of the MoveDMD trial. CAT-1004 is a potential disease-modifying oral DMD therapy regardless of dystrophin mutation type,” said Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Catabasis. “We have received significant support and enthusiasm for our MoveDMD trial and CAT-1004 from parents, advocacy groups, investigators and study staff, and we thank them for their participation and support.”

“The unmet medical need in DMD is profound and potential therapies that could make a meaningful difference are needed” said Richard Finkel, M.D., Division Chief, Division of Neurology, Department of Pediatrics at Nemours Children’s Health System. “Showing positive safety, tolerability and pharmacokinetics results is an important milestone in the development of CAT-1004. I look forward to the advancement of this novel potential therapy.”

CAT-1004 is an oral small-molecule that the Company believes has the potential to be a disease-modifying therapy for the treatment of DMD, regardless of the underlying dystrophin mutation. CAT-1004 is an inhibitor of NF-kB, a protein that is chronically activated in DMD as well as multiple other skeletal muscle disorders and rare diseases. In animal models of DMD, CAT-1004 inhibited NF-kB, reduced muscle degeneration and increased muscle regeneration. The MoveDMD trial is being conducted in two sequential parts, Part A and Part B. In Part A of the MoveDMD trial, 17 ambulatory boys between ages 4 and 7 with a genetically confirmed diagnosis of DMD across a range of dystrophin mutations received CAT-1004. The boys were steroid naïve or had not used steroids for at least six months prior to the trial. Part A of the trial was conducted at three sites in the U.S., and assessed the safety, tolerability and pharmacokinetics of CAT-1004 in patients at three dosing levels (33 mg/kg/day, 67 mg/kg/day and 100 mg/kg/day) during seven days of dosing. Part B will be a randomized, double-blind, placebo-controlled trial to evaluate the safety and efficacy of CAT-1004 in DMD over a 12-week period at 5 clinical trial sites in the U.S. at two dosing levels, 67 mg/kg/day and 100 mg/kg/day, subject to regulatory approval. The boys that participated in Part A of the MoveDMD trial will be asked to participate in Part B, and additional participants will also be enrolled for up to approximately 30 boys. We are currently identifying additional patients who are interested in participating in Part B of the trial. Entry criteria are expected to be similar to those in Part A.

More information about the MoveDMD trial can be found on the clinical trials page of the Catabasis website and on under trial identifier NCT02439216.

About CAT-1004
CAT-1004 is an oral small molecule that has the potential to be a disease-modifying therapy for all patients affected by Duchenne muscular dystrophy (DMD or Duchenne), regardless of the underlying mutation. CAT-1004 inhibits NF-kB, a protein that is activated in Duchenne and drives inflammation and fibrosis, muscle degeneration and suppresses muscle regeneration. In animal models of DMD, CAT-1004 inhibited NF-kB, reduced muscle degeneration and improved muscle regeneration and function, and beneficial effects were observed in skeletal, diaphragm and cardiac muscle. The FDA has granted orphan drug, fast track and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to CAT-1004 for the treatment of DMD. We have previously reported safety, tolerability and reduction in NF-kB activity in Phase 1 trials in adults. We are currently conducting the MoveDMDSM trial of CAT-1004 in 4-7 year-old boys affected by Duchenne. From Part A of the MoveDMD trial, we have reported that all three doses of CAT-1004 tested were generally well tolerated with no safety signals observed. Pharmacokinetic results demonstrated CAT-1004 average plasma exposure levels consistent with those previously observed in adults at which inhibition of NF-kB was observed.

About MoveDMDSM
MoveDMD is a Phase 1 / 2 clinical trial of CAT-1004 in boys ages 4-7 affected with DMD (any confirmed mutation). The MoveDMD trial is a two-part clinical trial investigating the safety and efficacy of CAT-1004 in DMD. Part A of the MoveDMD trial evaluated the safety, tolerability and pharmacokinetics of CAT-1004 with positive top-line results. In addition, the Company collected data at baseline on the muscles of the lower and upper legs using MRI, physical function (including timed function tests), and muscle strength. The boys in Part A of the trial will be asked to participate, if eligible, in Part B of the trial. Part B of the trial will be planned to evaluate the safety and efficacy of CAT-1004 in DMD over a 12-week treatment period. Preparations are underway for Part B of the trial.

About MRI
Magnetic resonance imaging (MRI) is a non-invasive imaging technique that can visualize muscle structure and composition and measure disease status in children with DMD. Two MRI measures used in Duchenne to indicate muscle degeneration are T2 and fat fraction. MRI is sensitive to changes in muscle structure and composition induced by disease processes such as the inflammation, edema, muscle damage and fat infiltration that occur in Duchenne. Changes in T2 may be seen in less than 12 weeks while changes in fat fraction may take longer. Changes in these MRI measures have been correlated with longer-term changes in clinically meaningful measures of functional activity. Changes in MRI can show the effects of an investigational therapy on disease progression in Duchenne in an objective and quantifiable manner.

About Catabasis
At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. We have product candidates in both rare diseases and serious lipid disorders. Our SMART (Safely Metabolized And Rationally Targeted) linker drug discovery platform enables us to engineer molecules that simultaneously modulate multiple targets in a disease. We are applying our SMART linker platform to build an internal pipeline of product candidates for rare diseases and plan to pursue partnerships to develop additional product candidates. For more information on the Company's drug discovery platform and pipeline of drug candidates, please visit

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 and other statements containing the words “believes,” “anticipates,” “plans,” “expects,” “may” and similar expressions, constitute forward-looking statements. 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 Annual Report on Form 10-K for the year ended December 31, 2015, and in other filings that the Company may make with the Securities and Exchange Commission in the future. The forward-looking statements contained in this presentation reflect our current views with respect to future events, and we do not undertake, and specifically disclaim, any obligation to update any forward-looking statements.


Corporate and Media Contact
Andrea Matthews
Catabasis Pharmaceuticals, Inc.
T: (617) 349-1971