The view from Endpoints
In the world of drug development, mouse data is a little like a broken twig on a confusing trail. Itmight tell you you’ve started down the right path and it might lead absolutely nowhere. In the vastmajority of cases, you wind up in the wilderness — completely lost and with nothing to show foryour effort.
Unless you put out the right kind of press release heralding your broken twig as a clear sign of major progress. And if you’re a public company, you can also get an instantaneous boost in your ailing stock price.
Just ask Israel’s Pluristem.
This morning Pluristem ($PSTI) put out a release citing results for its cell therapy in a mouse model for muscular dystrophy. The biotech handed over its cell therapy to a local Duchenne association, which did the mouse study and spotlighted a biomarker outcome.
“These preclinical data suggest that PLX-PAD cells could possibly be a breakthrough therapy to help treat symptoms of Duchenne muscular dystrophy,” says Hila Krupsky, CEO of ADI, the Association Duchenne Israel, in a statement for Pluristem. “We are thankful for Pluristem’s donation of PLX-PAD and are eager to continue studying the cells since new therapeutic approaches are needed to manage this disease, save children’s lives, and give them hope and a chance for the future.”
The key words here are “breakthrough therapy,” “children’s lives,” “hope” and “future.”
Treating Duchenne, of course, has been a Holy Grail for a lineup of biopharma companies. GSK ran late-stage studies on drisapersen and failed. BioMarin bought the drug and failed. PTC has failed, twice, with its treatment, including in Phase III. And Sarepta’s 12-patient study has become famous for falling flat with critical regulators, only to find that a chorus of patients and advocates can keep hope alive in the hunt for some positive biomarker evidence of its own.
When it comes to an ugly disease like Duchenne, hope is a key sales point. And all positive animal studies — which happen every day — spur the hope that fatal illnesses can be successfully treated some day.
For one type of investor, that’s the kind of hype that’s worth a buy-in, and Pluristem’s stock leaped more than 10% this morning.
With this kind of Pavlovian response mechanism in place, Pluristem wins the hype award of the week from BioRegnum for tarting up animal data from an advocacy group that has its own vested interest in demonstrating its involvement with patients.
— John Carroll
C’mon John. This is breakthrough news. $PSTI could be keeping tiny DMD mice from a life in tiny mice wheelchairs.
— Adam Feuerstein (@adamfeuerstein) June 22, 2016
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