Catabasis Pharmaceuticals, Edasalonexent for the Treatment of Duchenne Muscular Dystrophy Showing Early Clinical Success

Catabasis Pharmaceuticals, Inc. (NASDAQ: CATB), is a clinical-stage biopharmaceutical company. The company’ SMART (Safely Metabolized and Rationally Targeted) Linker, drug discovery platform, enables it to engineer molecules that simultaneously modulate multiple targets in disease. The company’ lead program in development is edasalonexent for the treatment of Duchenne muscular dystrophy.


The company had an incredibly strong start to 2018, and its Lead molecule (edasalonexent) posted stellar early data results to position it as a highly effective molecule for patients affected by Duchenne muscular dystrophy (“DMD”). The market believes that Edasalonexent has an extremely strong likelihood of delivering robust phase 3 clinical results.


Key Investment highlights:


Other recent announcements:

  • On June 20th, the company announced the pricing of an underwritten offering of an aggregate of 42,000,000 units. Each unit is comprised of one share of common stock and one common warrant to purchase one share of common stock, at a price of $1.00 per unit. The gross proceeds of the offering are expected to be approximately $42 million, and Catabasis intends to use the net proceeds from the offering for the planned Phase 3 clinical trial of edasalonexent for the treatment of Duchenne muscular dystrophy, as well as for working capital and general corporate purposes.


  • On May 10th, the company reported financial results for the first quarter ended March 31, 2018 and reviewed recent business progress. As of March 31, 2018, Catabasis had cash and cash equivalents of $17.0 million, compared to $16.4 million as of December 31, 2017. Based on the Company’s current operating plan, Catabasis believes that it has sufficient cash to fund operations through December 2018. 


  • During the quarter, the company reported additional positive clinical data from its MoveDMD trial early in 2018 that continue to demonstrate the potential of edasalonexent as a disease-modifying therapy for all patients affected by Duchenne, regardless of mutation. As evidenced by sustained improvements in all assessments of physical function and in biomarkers of muscle health and inflammation, edasalonexent has slowed the progression of Duchenne in the MoveDMD trial. The company believes that these effects ultimately will translate to boys with Duchenne maintaining functional abilities longer and as per management company resources have been aligned to focus on its lead program edasalonexent for the treatment of DMD. Catabasis is preparing for a global Phase 3 trial to evaluate the safety and efficacy of edasalonexent for registration purposes.



Upcoming presentation: The company will present data from the MoveDMD® trial of edasalonexent (CAT-1004) at the New Directions in Biology and Disease of Skeletal Muscle Conference, the 2018 Parent Project Muscular Dystrophy (PPMD) Annual Connect Conference and the 15thInternational Congress on Neuromuscular Diseases. New Directions in Biology and Disease of Skeletal Muscle Conference to be held between June 25 – June 28, 2018, in New Orleans, LA, at The Westin New Orleans Canal Place.


Present Pipeline:

Key and Unique differentiating factor of Edasalonexent

‣ Disease-modifying non-steroid oral therapy

  • Intended for all patients, regardless of mutation type
  • Inhibit muscle degeneration, enhance regeneration
  • Benefits in skeletal muscle, diaphragm, and heart

‣ Preparing for single Phase 3 trial for registration

  • In MoveDMD® trial, edasalonexent preserved muscle function and slowed disease progression

‣ Potential foundational therapy

  • Initiate upon diagnosis
  • Potential as monotherapy and may enhance the efficacy of dystrophin upregulation approaches

‣ Favorably differentiated tolerability profile from the standard of care

‣ Strong IP position and wholly owned

Q1 Financial position: 

  • R&D Expenses: Research and development expenses were $5.2 million for the three months ended March 31, 2018, compared to $5.4 million for the three months ended March 31, 2017.
  • Net Loss: Net loss was $7.7 million, or $0.29 per share, for the three months ended March 31, 2018, compared to a net loss of $7.9 million, or $0.41 per share, for the three months ended March 31, 2017.


Liquidity and financial flexibility:

  • As of March 31, 2018, Catabasis had cash and cash equivalents of $17.0 million, compared to $16.4 million as of December 31, 2017. Based on the Company’s current operating plan, Catabasis believes that it has sufficient cash to fund operations through December 2018.


Key risk factors and potential stock drivers:

  • Company business profile is significantly dependent on its lead program edasalonexent for the treatment of DMD. Catabasis is preparing for a global Phase 3 trial to evaluate the safety and efficacy of edasalonexent for registration purposes.
  • Any adversities related to these upcoming milestones might adversely impact the overall investor sentiments.
  • Results of upcoming catalyst would drive the near to medium term performance of the company. Edasalonexent would continue to remain one of the most critical factors for Catabasis business risk profile over the near to medium term.
  • The company is still at a pre-commercialization stage and its ability to maintain its liquidity and financial flexibility to fund its incremental capital requirements.


Stock Chart:


  • On Friday, June 22th, 2018, CATB closed at $0.19, on an above average volume of 1.0M shares exchanging hands. Market capitalization is $26.4 million. The current RSI is 18.79.
  • In the past 52 weeks, shares of CATB have traded as low as $0.86 and as high as $3.78
  • At $0.91, shares of CATB are trading below its 50-day moving average (MA) at $1.55 and below its 200-day moving average (MA) at $1.74
  • The present support and resistance levels for the stock are at $0.82 & $0.96 respectively.


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