What This New Therapeutic Drug Trial Means for Cystic Fibrosis

If you have cystic fibrosis, chances are that at some point you have had difficulty breathing due to mucus build-up in your lungs. That thick mucus can make it more than just hard to breathe, as it can cause inflammation and increase your chance of injury. Clear airways may be just around the corner thanks to a new drug, moving forward into a new trial.

The therapeutic drug is called SPX-101. Research began in 2015, when Phase 1 of the study started. The study is currently in Phase 1b. The bio-pharmaceutical firm responsible has just secured funding to properly move on to the next phase, Phase 2, which they are also calling HOPE-1.

The People Responsible

Spyryx Biosciences, Inc., is a Canadian private pharmaceutical form dedicated to creating a drug to keep lungs and airways clear. They have received funding from the Cystic Fibrosis Foundation Therapeutics to continue their research. Up to five million dollars, in fact.

Cystic Fibrosis Foundation Therapeutics is the somewhat-awkwardly named drug research and development affiliate of the Cystic Fibrosis Foundation. They initially awarded some funding to Spyryx in 2015, but have just announced the massive increase in money awarded.

There are other investors who are helping Spyryx research SPX-101. Canaan Partners, 5AM Ventures, and Hatteras Venture Partners have teamed up with the pharmaceutical firm to research their drug.

The people behind this research are obviously passionate about the drug they are studying. Spyryx is solely focused on this one medicine, and the researchers are all passionate in the reports they have spread.

The Drug

The drug is called SPX-101. It is an inhaled peptide which affects the sodium pathways in the lungs. A peptide is an amino acid chain, similar to a protein, except smaller. In fact, SPX-101 is derived from a naturally occurring protein, called SPLUNC1.

For most people, SPLUNC1 is a good thing. It acts on the epithelial sodium channel, also known as ENaC. ENaC is responsible for bringing sodium into cells. However, for people with cystic fibrosis, ENaC becomes hyperactive. This causes the cells to become unbalanced when it comes to sodium and water. Thicker mucus is a result.

The peptide in SPX-101, derived from SPLUNC1, is taken into the body as if it were SPLUNC1. Since it is not the same as the protein, the sodium pathways will not respond “properly,” and will be suppressed.

For most people, this would be a bad event. For people with cystic fibrosis who have a hyperactive sodium channel, this is a good thing. By blocking the absorption of sodium into the lung cells, SPX-101 causes more water to enter the cells.

More water movement means that mucus stays more hydrated. Hydrated mucus is thinner and easier to move by the body. With less mucus in the lungs and airways, oxygen can flow more freely. Less sodium in the lung cells, more oxygen can be used by the lungs.

Part of why this drug is wonderful is that it affects unlike other inhaled drugs, which try to directly attack the mucus, it affects the biological process at the core of the problem. It brings the hyperactivity of ENaC down to normal activity levels. This also means that the drug will be good for everyone with cystic fibrosis, regardless of the cause of the disease.

In Animals

The first Phase of the study tested the drug in mice with cystic fibrosis. This disease often leads to a high rate of death among affected mice, as they cannot deal with difficulty breathing as well as humans.

SPX-101 was successful in lowering the mortality rate of the mice. Just a single dose per day raised the survival rate astronomically. By the end of the trial, almost every one of the mice receiving the medicine survived!

Cats and dogs were also tested upon. Though they were dosed for 28 days, no side effects were discovered. Not even an increase in the level of potassium, which was considered a possibility by the researchers. The balance between potassium and sodium in your body is important, so the potassium levels staying the same despite the sodium being blocked is a good thing.

In People

Phase 1 of the study also tested SPX-101 on people. Being preliminary testing, the test subjects were not people with cystic fibrosis. They were healthy adults with no history of lung disease. The intent was to discover if the drug had any negative side effects. Basically, is it safe?

There were 32 adults tested. They were dosed twice a day for 14 days, at varying levels of dosing. Of course, some of the test subjects were given a placebo. SPX-101 was administered using a nebulizer system called eFlow.

None of the participants had any change to their lung function. Potassium levels stayed the same. Airways remained unrestricted. It was found to be safe. They concluded that it doesn’t have a heavy effect upon the human body, and was cleared rapidly after dosing.

Phase 1b is in progress and so there have been no reports as of yet. It involves a small amount of people with cystic fibrosis.

Phase 2

The next phase of the study is called HOPE-1, an auspicious name. As it has not yet started, hard information of what the next trial in the study is not yet available. It will be a much larger study, however. It will also have multiple objectives.

Phase 2 will be a multinational trial covering a larger amount of people of different ethnicities and different causes of cystic fibrosis. Spyryx will also use Phase 2 to research how to make the drug as efficacious as possible. Different administration methods will be explored as well as different dosing levels.

What Does This Mean?

Unfortunately, it does not mean an awful lot at the moment for most people. The successful funding merely means that the scientists have the resources they need to prepare for a hard job ahead of them. A large amount of work goes into a successful pharmaceutical trial.

There will be three basic sections to the trial. First, the researchers will have to prepare for the trial. This involves writing the plan for the trial, seeking people with cystic fibrosis to join as test subjects, arranging for the manufacture of large amounts of the drug, and meeting safety and research regulations.

The trial itself will probably be the easiest part for the next few years. They will meet with the test subjects, observe them, administer the medicine, and record everything that happens.

After that part of the trial has concluded, then comes the grueling job of compiling the data and digging out something useful from the sheer amount of information acquired. Sometimes, writing the report at the end of the experiment can take longer than the experiment itself took.

If the conclusions are positive, and things are looking that way, then Spyryx will team up with pharmaceutical production and distribution firms. After governments okay the drug, only then will it hit the market.

This will take a lot of time. Phase 1 and 1b took two years from start to finish, and they were on a much smaller scale than what Phase 2 will be.

However, a few years from now, this will mean good things for cystic fibrosis. All people with cystic fibrosis, regardless of the source of the disease, may be able acquire a medicine that is easily administered, safe, and efficacious. It may even be inexpensive, as it is derived from a natural protein rather than synthesized artificially.

The Bottom Line

SPX-101 is an exciting new drug with lots of potential. It has so far shown itself to be safe and to increase the survival rate of animals with cystic fibrosis. With the five million dollars in funding from Cystic Fibrosis Foundation Therapeutics, Spyryx should be able to bring this hopeful new drug to the people who need it. 

If you are interested in taking part of the new trial, visit ClinicalTrials.gov and search for “Spyryx” in the “Other Terms” field. When Spyryx Biosciences starts recruiting for Phase 2, it will be available on that website.