March has been a month associated with bad news. Remember Julius Caesar and the ides of March? At least Julius had the opportunity to enjoy a couple of good weeks before disaster struck.
The CAR-T pipeline keeps rolling for Juno
Juno's discontinuation of JCAR015 was nearly anticlimactic. The company slipped the news that it wouldn't advance the chimeric antigen receptor T-cell (CAR-T) drug in its fourth-quarter financial update. It was bad news for Juno, but not surprising news.
The biotech had placed the phase 2 ROCKET clinical trial evaluating JCAR015 on hold in November. Two patients in the study taking the experimental drug died after suffering from swelling in the brain.
However, losing JCAR015 isn't a death blow for Juno. The biotech still has another CAR-T candidate in phase 2 with JCAR014. Juno's pipeline also includes six early stage candidates.
Juno's relationship with Celgene (NASDAQ:CELG) is another plus. In April of last year, the big biotech exercised its option for marketing rights to Juno's CD19 program outside of North American and China. This program at the time included JCAR014, JCAR015, and early stage candidate JCAR017. Celgene was involved in the decision to throw in the towel on JCAR015.
The partnership with Celgene gives Juno stability that it probably wouldn't have otherwise. Juno is in good shape financially thanks in large part to money received from Celgene. At the end of 2016, Juno had cash, cash equivalents, and marketable securities totaling $922.3 million. Celgene also owns a stake in Juno and therefore has an even greater interest in Juno's success.
Although it hurt Juno to discontinue JCAR015, the biotech's pipeline and financial position allow it to keep rolling on. However, the biotech is still years away from potential commercialization of any of its products.
Cystic fibrosis opportunity for PTC evaporates
Just a few days ago, I wrote about five small-cap biotech stocks that adventurous investors could buy in March. I mentioned in the article that the stocks were risky, but good news from upcoming events could serve as positive catalysts. One of those small-cap stocks was PTC Therapeutics.
As luck would have it, the article published the same morning that PTC announced that its late-stage study of Translarna in treating nonsense mutation cystic fibrosis (nmCF) didn't meet the primary endpoint. The biotech said that it was shutting down its cystic fibrosis program. PTC's stock plunged around 20% on the news.
This illustrates just how risky investing in biotech stocks can be. Had PTC reported positive results from the cystic fibrosis study, the stock would have soared. However, good earlier-stage results don't always translate to success in late-stage studies.
So what's next for PTC Therapeutics? Translarna won approval in Europe to treat Duchenne muscular dystrophy (DMD), but the U.S. Food and Drug Administration (FDA) wouldn't accept the biotech's regulatory submission last year. PTC plans to move forward with submitting to the FDA under protest. As you might expect, the odds aren't too great for success taking this approach.
Translarna is also being evaluated in three other phase 2 studies for treating rare diseases. Results won't be available for any of these studies until next year at the earliest.
Roche and PTC are collaborating on development of an alternative splicing therapy for treating spinal muscular atrophy (SMA). This experimental treatment is in phase 2 testing. Two pivotal studies are expected to begin this year.
PTC Therapeutics will likely report cash, cash equivalents, and marketable securities of around $220 million at the end of 2016 when the company announces its fourth-quarter results. That should be enough to keep PTC going well into the future.
For some small biotechs, abandoning major programs like Juno and PTC Therapeutics recently had to do would be devastating. Juno and PTC, though, are in better shape than many of their peers.
The pipelines, cash positions, and relationships with bigger companies that these two small biotechs have will allow them to survive for a long time to come. Juno and PTC will move past their March disasters, although what happens in the future is anyone's guess.