For most biotechs, a successful Phase I dose-escalation study with good safety data and positive pharmacodynamics would barely win a nod of approval. For the RNAi drug developer Alnylam, ($ALNY) it sparked an unusual market frenzy yesterday that drove its share price up more than 50%, breaking the $19 mark.
The data revolved around a Phase I study of ALN-TTR02, an RNAi drug for rare cases of TTR-mediated cases of amyloidosis. By knocking down the TTR protein, Alnylam logically believes it can treat the disease. And in 17 healthy Phase I subjects recruited for the study, the drug effectively slashed TTR by 94%, indicating that it could be a winner in the patient population--especially as they believe that a 50% reduction could be enough to stabilize or improve patients.
"I am impressed with the almost complete knockdown of TTR after just a single dose of drug, which is important since TTR protein reduction in patients with ATTR has the potential to delay or even reverse disease progression with associated clinical benefits," said Teresa Coelho, the director of the Unidade Clinica de Paramiloidose.
Some analysts could barely contain themselves.
"With orphan drug pricing, we forecast blockbuster global sales of $2 billion by 2020," wrote Piper Jaffray analyst Edward Tenthoff, according to a Reuters report.
"It's Phase I but you should think of it as Phase II," Rodman's Michael King told Bloomberg. And Marko Kozul, an analyst with Leerink Swann, enthused about the prospects of a clinical development program that only required a relative handful of patients to achieve clear efficacy data.
That's true, of course, but there have been plenty of other biotechs engaged in early-stage development of new drugs for rare diseases which have never seen anything like this kind of response on the stock side. What's particularly striking about the bullish spike is that RNAi has been operating under a cloud for some time now, with troubling questions about drug delivery and time of development which has hampered the field.
Today, Alnylam's biggest issue seems to be curbing the enthusiasm of its analysts.