Genocea Biosciences

Genocea Biosciences [$GNCA, market cap $131M] is a company that researches immunotherapies, focusing on the T-Cell¬†component of the immune system. They claim to use a proprietary research platform called ATLAS, but there’s not a lot of info available on how ATLAS works or compares to other platforms.

Their chief candidate (in phase 2b trials) right now is a drug called GEN-003, a vaccine aimed at decreasing the severity and viral transmission rate of genital herpes. They are working on some cancer stuff as well, but it’s all smoke at the moment. Two things are exciting about GEN-003, should it be a viable therapy:

1) Their drug has so far shown comparable results to daily oral medication (i.e. valtrex), but GEN-003 is an annual injection.

2) If GEN-003 is successful, it will be one of the first-ever T-cell vaccines for an infectious disease. Some T-cell therapies are used to treat leukemia/lymphoma, but as far as I know, this would be one of the first commercially available T-Cell therapies for something other than cancer.

The latter point especially makes GNCA a prime buyout target if GEN-003 is successful. Remember the ATLAS thing? If they’ve successfully used it to create a widely-applicable T-cell vaccine, a large company will want that technology.

Gen-003

Here’s where we’re at so far.

In 2012, Phase 1 trials established that the concept would probably work and was safe.

In 2014 they started the first part of Phase 2 trials, using 6 different formulations of the vaccine to see which one worked the best.

In 2015 they started Phase 2b, a closer look at the 2 best-performing formulations.

The Good

In March this year, GNCA announced that they had positive 12-month efficacy results for their drug. The presentation is here. This is big, though — positive 12-month efficacy will probably be their phase 3 endpoint and determinant of drug approval. Hitting it here means that the NDA is pretty likely to be approved.

The next step for Gen-003 is the phase 2b trial (ongoing), which is comparing the two most promising formulations of the drug. GNCA estimates that they will meet with the FDA in Q1 of 2017 to discuss the end of the phase 2 period. Given the strong results in Phase 2, if the drug passes phase 2, a phase 3 go seems extremely likely.

The Bad

Their research seems pretty solid, on the whole, but there are two problems so far. First, they rolled the placebo group into a testing group after the drug administration and first datapoint (on day 28 of the trial). Basically they confirmed that there was a significant difference with the drug and no change for the placebo group, then rolled the placebo group. Given that the rest of the trial was just following up to make sure the results stuck around, this isn’tterrible, but it’s not great. I don’t know why they rolled the placebo group. Potential explanations include a need for more test participants and a desire to give the patients access to the real thing after the first datapoints showed a statistically significant difference between the drug and the placebo.

Second, in every trial far, GEN-003 has outperformed the placebo in viral shedding, but matched the placebo in actual lesion outbreak [results album] Potential explanations: a) viral shedding is determined in a lab, but lesion outbreak is patient-reported. It’s possible that there are some reporting issues there. b) placebo effect can decrease lesion outbreak (external symptom and immune mediated) but not viral shedding (asymptomatic, less immune mediated).

Summary

Although I’m not thrilled about the lesion thing, even if the drug only decreases viral load it could still be considered a success, as viral shedding is necessary for herpes transmission. The company itself seems to have a very positive outlook on GEN-003’s chances, and if you’re looking for a biotech that might deliver some spikes in the near term (expect a spike in Q1 2017 if Phase 2 is completed successfully) with good potential for longer term (6mos – 2yrs), GNCA is a good target.

Prediction: Phase 3 Approval

Disclosure: I own a stake in GNCA.