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Catabasis Pharmaceuticals poised be a potential game changer in the DMD Therapeutic Industry

15 Jan Catabasis Pharmaceuticals poised be a potential game changer in the DMD Therapeutic Industry

Catabasis Pharmaceuticals (NASDAQ: CATB), a clinical-stage Company leveraging its proprietary SMART (Safely Metabolized and Rationally Targeted) Linker drug discovery platform to treat multiple biological targets announced a partnership with Duchenne UK, a London-based charity that funds treatments for Duchenne Muscular Dystrophy (DMD). The partnership will see Duchenne UK fund Catabasis’s pivotal phase II study of edasalonexent, an NF-kB inhibitor and its lead drug candidate for the treatment of non-ambulatory Duchenne muscular dystrophy (DMD).

The Study will be exploratory in nature and evaluate the safety, pharmacokinetics and efficacy including cardiac, skeletal muscle and pulmonary function, in an estimated 16 non-ambulatory patients aged 10 years and above. The patients have to be off steroids for at least six months and can participate regardless of the mutation type. Once a patient completes the trial, he will have the option of enrolling in Catabasis’s new open-label extension named study GalaxyDMD to receive edasalonexent.

Edasalonexent is currently being evaluated in an ongoing phase III clinical trial, namely PolarisDMD study, the topline data from which is expected to be announced in Q4, 2020. In addition, the Company has also initiated GalaxyDMD in March 2019, with the intention of providing long-term safety data to fulfil the requirements of registration filings.

The candidate is also being evaluated for the treatment of other forms of muscular dystrophy namely becker muscular dystrophy (BMD) and those caused by dysferlin protein deficiency. In August 2019, Catabasis entered into preclinical research collaboration with the Jain Foundation for DMD caused by dysferlin protein deficiency, which includes Limb-girdle muscular dystrophy type 2B and Miyoshi myopathy.

Catabasis Pharmaceuticals (NASDAQ: CATB)

Market Cap: $78.21M; Current Share Price: 6.63 USDChart
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The Muscular Dystrophy Association describes Duchenne Muscular Dystrophy (DMD) as a fatal, X-linked genetic neuromuscular disorder that leads to progressive, irreversible loss of muscle function, including the heart and lungs. It has an X-linked recessive inheritance pattern and is acquired by a “Carrier” mother. Globally, 1 in 3500 male births is affected by this rare neuromuscular disease. There are over 250,000 people in the United States alone who are afflicted by this disorder.

The growing awareness about this disease and increasing research efforts to find a cure, along with improved insurance coverage and favorable government regulations will drive the growth in the market. According to a report by Grand View Research, Inc the market of DMD therapeutics will grow at a CAGR of 41.3% and reach USD 4.11 billion by 2023.

Researchers have identified the lack of dystrophin, a protein that helps regulate the functioning of membrane (sarcolemma) of muscle cells as the cause of this disorder. Exon skipping is gaining credence as a potential treatment approach to correct for specific genetic mutations and restore production of dystrophin protein, with an increasing number of companies coming up with first-in-class therapeutics based on this approach.

However the emergence of new mechanisms of action such as NF-κB inhibition, Gene therapy, and Exosome technology can redefine the therapeutic industry.

Catabasis Pharmaceuticals lead candidate for the treatment of Duchenne Muscular Dystrophy (DMD) is Edasalonexent that can inhibit NF-κ, and has shown to preserve muscle function, improve skeletal health and slowdown the disease progression in DMD. It is currently conducting Phase 3 clinical trials and has received an Orphan drug designation, a fast track designation and rare paediatric disease designation from the FDA, in addition to receiving the Orphan Medicinal Product designation by the EMA.

The chronic activation of NF-kB in DMD, due to a lack of dystrophin, causes skeletal and cardiac muscle disease progression. Edasalonexent has the potential to mitigate muscle damage and promote muscle regeneration due to its ability to inhibit NF-kB.

Image Source: Company

It’s SMART (Safely Metabolized And Rationally Targeted) Linker drug discovery platform enables it to re-engineer molecules in the disease pathways, and thus increase the efficiency of bioactives, by allowing them to combine two separate bioactives whose pharmacological activity is already known.

Image Source: Company

In the pipeline is CAT-5571 an oral small molecule for the treatment of cystic fibrosis (CF) which has the potential to restore host defence by activating autophagy, a crucial mechanism that recycles cellular components and digesting pathogens, which is dysfunctional in patients with cystic fibrosis. In addition CAT-5571 has the ability to improve the clearance of pathogens such as P. aeruginosa and B. cenocepacia, which are responsible for causing chronic infections and respiratory failure in people with CF.

As of 30 September 2019, Catabasis had cash, cash equivalents and short-term investments of $40.6 million, compared to $46.1 million as of June 30, 2019, as per its Third quarter 2019 financial statement.

Risk Assessment 

  • The Duchenne Muscular Dystrophy space is seeing the adaptation of varied approaches to treatment ranging from gene therapy, utrophin modulators, myostatin inhibition, exon skipping, CRISPR, to name a few. The DMD market will see as many as 7 new drugs hitting the market by 2021. Some of the competitors include names like Sarepta and PTC Therapeutics, which are likely to give a tough competition to the Company.


  • Clinical trials are fraught with risk, the DMD space has seen a few failures in the recent past, In December 2019, Wave Life Sciences (NASDAQ: WVE) announced the decision to discontinue the development of suvodirsen for patients with Duchenne muscular dystrophy (DMD), who have mutations amenable to exon 51 skipping, based on the interim results from a phase 1 open-label extension (OLE) study. Catabasis is using a differentiated approach to treatment of DMD, which may hold the key for a potential treatment.


  • The Company has announced cash and cash equivalents of $40.6 million as on September 30, 2019, which it believes is sufficient to fund its ongoing clinical trials as well as take care of its research and development and operational expenses.


  • Catabasis has been able to find strategic partners to fund the development of its pipeline, such as Duchenne UK and Jain Foundation, which are imperative for clinical-stage firms. This augurs well for its finances and serves as a validation of its technology. The Company has also entered a pre-clinical joint research collaboration agreement with Sarepta Therapeutics (NASDAQ: SRPT), for working on a treatment approach for DMD. Catabasis is also collaborating with the University of Texas Southwestern to evaluate the potential benefits of edasalonexent treatment.


  • Besides its primary focus on DMD, it is also targeting the cystic fibrosis market, which is estimated to be worth over USD 13.9 billion by 2025, according to a reportby Grand View Research, Inc. This will help diversify its pipeline and mitigate risks associated with focussing only on one drug candidate.


Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.

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