Replies to post #221170 on Biotech Values

[semi_infinite]

How long does it take to rehab hip fracture or surgery? Doesn't that matter to how long the drug is used?

[RockRat]

RE VK5211: The main issue, from an investment standpoint, is whether or not they can partner this based on existing data, since that is their stated strategy. I agree LBM isn't an approvable endpoint. They'd likely require direct fracture data for approval, at least in the first indication. Perhaps subsequent studies can employ BMD. As such, we have to look at the preclinical data wrt effects on bone. Here it is:

LGD-4033 builds muscle and bone with reduced prostate activity and may be beneficial in age-related frailty

Not sure why you're focused on durability of anabolic response. Nobody expected that.

As for the walk test, the tested population was pretty heterogeneous, so in a small study the variance is going to be too much to extrapolate benefit with any degree of certainty. I don't know what a "PRO" is in this context. Surrogate endpoint? REMS?

Completely agree more data would be helpful. Isn't it always? Safety looks fairly clean, and dosing to sustain the anabolic effect looks possible at this stage, with prophylaxis still on the table. So I think it is too soon to read the tea leaves and call 5211 a definitive bust. It's definitely got plenty of risk of good money after bad, based on what we know now, so I'd be interested in having them partner the program, even if they don't get a big upfront. If they post more data before then, we can revisit.

Anyhow, I think the risk of the program just got priced in, and then some, so I've sold a couple of rounds of puts.

Regards, RockRat

[biomaven0]

>>VK5211

I really don't understand why you see this as a fail. It's incomplete data from a small trial, but nothing they disclosed to date gives cause for concern. You ask about the dogs that didn't bark (like lipids) - that is indeed a legitimate concern, but we have to wait for more data before deciding one way or the other.

This is designed to be a more selective anabolic drug, so there is no reason at all to believe it would do better than a conventional steroid on efficacy. So the fact that the durability wasn't 24 weeks is to be expected based on oxandrolone also not being durable at 24 weeks. Why you conflate that lack of durability with androgen induced hypogonadism I'm not at all sure.

What endpoint the FDA will want is unclear to me. 6-minute walk would be great - very likely they could get efficacy there if the trial was bigger. Fractures would be harder - need a way bigger trial. But FDA going to want reassurance on safety (prostate cancer for one), so this will require a fairly big study anyhow.

[biomaven0]

No lipid AEs:

http://s1.q4cdn.com/460208960/files/pictures/2018/10032018_VKTX_6.png