Mark Chapman Shares an Investor’s Guide for The Race To Cure Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy [DMD] is a deadly genetic disorder that currently does not have a cure. The only current treatment options are corticosteroids, such as prednisone or deflazacort, which have been found effective in slowing the progression of DMD. With a couple of New Drug Applications [NDA] pending with the FDA and several IPOs for company’s attempting to develop new cures recently completed, relief may finally be on the way for DMD patients. This article serves as a summary of potential investment opportunities for those looking to invest in a cure for DMD.

Overview of DMD

DMD is one of nine types of muscular dystrophy, which is characterized by muscle degeneration and weakness. It is caused by an absence of dystrophin, which is an essential protein in muscle cells. Symptoms generally start to manifest in young boys as early as three years old, typically through difficulty standing up or maintaining balance. Although greater understanding of DMD has led to some patients being able to live into their 30’s or 40’s, many often die in their 20’s or even earlier, which just highlights the need to find a cure.

Given that strong muscles are required to help us breathe and pump blood throughout the body, it’s not surprising that breathing complications and cardiomyopathy are common causes of death in DMD patients. Therefore, some of the treatments currently being developed specifically target the heart or respiratory system, while others are looking to alter the specific genetic defects.

What this ultimately means is that one specific treatment is unlikely to work for every patient (or possibly even a majority), and a combination of products will likely be needed. So despite an advancement or “breakthrough” of one company, each of the following will need continued funding to advance the development of their potentially lifesaving drug.

Investment Opportunities

The following companies are developing a treatment for DMD, listed from furthest along to the beginning stages of development. Since there are currently no FDA approved drugs for DMD, each treatment has been given fast track and orphan designation. It is estimated that DMD occurs in approximately 1 in 3,500 live male births, which equates to a total population of about 15,000 in the United States and 19,000 in the European Union.

For any readers that are familiar with DMD, please let me know if there are any additional treatments in development that I have missed.

Sarepta (NASDAQ:SRPT)

• Lead candidate is eteplirsen, which is an intravenous infusion designed to skip the exon 51 in the dystrophin gene. By skipping exon 51, the drug should correct the specific genetic mutation and restore the gene’s ability to make a functional, though shorter, form of the dystrophin protein.
• Clinical trials are complete, and despite some setbacks during clinical trials, the rolling NDA was completed on June 26, 2015.
• About 13% of DMD patients may be candidates for exon 51 skipping therapy, although the company believes that up to 80% of DMD patients may be amenable to its other exon skipping therapies in development.

BioMarin (NASDAQ:BMRN)

• Lead candidate is drisapersen, which similar to Sarepta’s eteplirsen, is an intravenous infusion designed to skip exon 51. BioMarin acquired drisapersen through its acquisition of Prosensa.
• Clinical trials are complete, and despite some setbacks during clinical trials, the NDA was accepted by the FDA and a PDUFA date of December 27, 2015 has been set.
• About 13% of DMD patients may be candidates for exon 51 skipping therapy.

Santhera (OTCPK:SPHDF)

• Lead candidate is idebenone, which is a synthetic derivative of the antioxidant CoQ10. The drug is not designed to be disease modifying in DMD, but instead delay the loss of respiratory function.
• Successfully completed the “DELOS” phase 3 trial, with an NDA filing pending.

PTC Therapeutics (NASDAQ:PTCT)

• Lead candidate is ataluren, which was conditionally approved in the EU in August 2014 under the trade name Translarna. Ataluren is a small molecule compound for the treatment of DMD caused by a nonsense mutation (nmDMD). Nonsense mutations create a premature stop signal in the translation of the genetic code contained in mRNA, which prevents the production of full-length, functional proteins.
• Despite previous setbacks in clinical trials, a Phase 3 trial is currently ongoing with the data readout expected Q4 2015. A rolling NDA was commenced in December 2014.
• About 13% of DMD patients are estimated to have the nonsense mutation causing DMD.

Pfizer (NYSE:PFE)

• Lead candidate is PF-06252616, which is an infused anti-myostatin monoclonal antibody. Myostatin is a naturally occurring protein in muscles that helps control growth. Pfizer hopes that inhibiting myostatin will cause more initial muscle mass, which would help offset the loss associated with DMD and allow boys to retain their ability to function for a longer period of time.
• Phase 2 trial was started in December 2014, with an estimated primary completion date of January 2017.

Akashi (Privately Held)

• Lead candidate is HT-100, which is a delayed release halofuginone small molecule designed to reduce fibrosis (scarring) and inflammation, along with promoting regeneration. Halofuginone is found in the root of a hydrangea plant called “chang shan”, and blocks TH17 cells which play a role in the pathogenesis of inflammatory diseases.
• Phase 1b/2a trial is expected to be complete in January 2016, with an open label extension until February 2017.
• Expected to help all DMD patients, regardless of the underlying dystrophin mutation.

Catabasis (NASDAQ:CATB)

• Lead candidate is CAT-1004, which uses the company’s SMART linker technology to create a conjugate of salicylate [NSAID] and the omega- 3 fatty acid docosahexaenoic acid [DHA]. DHA is a naturally occurring anti-inflammatory, and when combined with salicylate, is designed to inhibit NF-kb. Activated NF-kb is believed to drive muscle degeneration and suppress muscle regeneration.
• Phase 1/2 clinical trial was initiated in June 2015, with top-line phase 2 data expected in late 2016.
• Expected to help all DMD patients, regardless of the underlying dystrophin mutation.

Summit (NASDAQ:SMMT)

• Lead candidate is SMT C1100, which is an utrophin modulator. Utrophin is similar to dystrophin, but is switched off in maturing muscle.
• Top-line data from a second Phase 1b trial is expected mid-2015.

Capricor (NASDAQ:CAPR)

• Lead candidate is CAP 1002, which is a cardiac cell therapy from donor heart tissue infused directly into the coronary artery, and is designed to reduce cardiomyopathy in DMD patients.
• The IND was approved on June 8, 2015.
• Collaboration with Janssen biotech, a subsidiary of Johnson & Johnson (NYSE:JNJ).