First, manufacturing is one of the greatest challenges in gene therapy. Many drug developers have had great difficulty delivering cancer-fighting genes into cancer cells safely and effectively. Most gene therapies use viral delivery systems, which have unwanted side effects. Ours is non-viral, and after treating more than 50 patients, it has a favorable safety profile and demonstrated ability to deliver genes into tumors. Some therapies require the drug to be manufactured specifically for each individual patient, which is very expensive and limiting in practical application. We have shown that we can manufacture our drug at large scale, and the drug can be stored, shipped, and delivered to distant locations for use as needed.
Our manufacturing is done in several steps. DNA plasmid is manufactured, and this plasmid is formulated with DOTAP cholesterol molecules, which encapsulate the plasmid and carry it through the bloodstream into cells. (DOTAP is a type of cationic lipid that's used as a surfactant and also used in vitro and in vivo applications.) Our DNA plasmid has been made at a commercial plasmid manufacturer for quite some time. However, we were recently able to significantly increase efficiency in production which will result in a much lower plasmid cost per dose.
Until now, the second step of combining the DOTAP molecules with DNA plasmid has been done at (the University of Texas') MD Anderson's manufacturing facility at small scale. Each dose was formulated as it was needed for a patient and delivered to the clinic where it was injected. It was sufficient for our earlier trials, but not for larger, multi-site trials, and certainly not for commercialization. We believe that we have successfully transferred the formulation of DOTAP with plasmid DNA from the research lab setting to commercial manufacturers and scaled up the process to allow production at much larger scale. This is a major accomplishment.
... I would add ... the required shipping and storage temperature in only two to eight degrees Celsius, not minus 50 to minus 80 degrees Celsius required by many biologics.
When do you anticipate enrollment beginning? Will Acclaim-1 and Acclaim-2 start at the same time?
We anticipate enrollment beginning in both trials in the first half of 2021. We are working on both now and are not yet announcing which will start first.
If you have success with both trials, would you prioritize one combination over another? Would success in one have any bearing on a future in the other?
No, if both trials are successful, we believe each would be a fantastic program. (The non-small cell lung cancer drug) Tagrisso's 2019 sales were around $3 billion, and (cancer drug) Keytruda's are much larger than that. There isn't much overlap between patients who receive Tagrisso and those who receive Keytruda. We believe that success in either of these trials would bring great value to our company. If we are successful in combining with both, then we believe we could potentially address a majority of late-stage non-small cell lung cancer patients.
Overall, I like seeing a big buyer step in. I'm intrigued as to where the $12 million might be deployed. Genprex has begun pulling synergistic technologies for cancer treatment and cancer prevention into its mix. It's possible, though one cannot say for sure, that this $12 million is a forest situation. The view isn't as simple as what's in front of you.
While the recent news may seem ho-hum, manufacturing is incredibly important. These updates also show Genprex continues to remain exactly on track with what I wrote a few months ago in regards to timelines for 2020 and 2021.
This name falls into the speculative category of investments, but still remains one of my favorite names for 2021 and beyond.