So how does a small cap biotech go toe-to-toe with the likes of Pfizer, when the pharma giant is lining up for one of the most carefully watched FDA decisions in biopharma?
Eidos Therapeutics $EIDX thinks it has the answer as they play David to Pfizer’s Goliath.
The biotech today outlined its pivotal strategy for AG10, an oral small molecule designed to treat transthyretin amyloid cardiomyopathy, ATTR-CM. Working with the FDA, the biotech came up with a Phase III trial design that the execs in charge say gives them two potential pivotal deadlines. The first arrives after 12 months of treatment, as patients are given a 6-minute walk test. The next comes after 30 months, as researchers evaluate patient data on all-cause mortality and frequency of cardiovascular-related hospitalizations.
That trial starts today. And Pfizer gets its FDA decision — a likely yes — in July. That puts them right up against Alnylam, which got a pioneering OK for its RNAi therapy last year.
Trial launches by and large don’t get a lot of coverage. In this heated development era we’re in, it’s hard to gain much attention for your pivotal timeline — especially if you’re playing catch-up. But Eidos hopes to prepare a casebook example of how to carve out a timeline that can give you a shot at competing relatively quickly with the heavyweights of the world. And it’s worth a look as we begin to track their late-stage progress.
“If you are coming from behind with a better drug, you should get it to the marketplace,” says Neil Kumar, who runs BridgeBio, the umbrella organization that controls Eidos and a slate of other startups.
“It’s a pretty cool path,” says Kumar, “a 12-month placebo-controlled trial, which is a great option for patients with no access to any small molecule stabilizer.”
Kumar doesn’t want to talk price right now — no one does in this business ahead of an FDA approval. But you can pretty much bet that if it can get out early with 12-month data, pricing will be key if it wants to carve away at the franchise Pfizer will have already built.
The pharma giant has been under the gun on pricing, and just about everyone expects they’ll come up with an aggressive 6-figure price tag on their drug, once it’s approved — particularly if they can’t keep raising the price after it hits the market.
In addition, a lot of these patients will be on Medicare, and could be facing a big out-of-pocket expense as long as the donut hole remains. If Eidos comes up with a much better price, they stand a better chance at winning over market share. And they can move in after Pfizer helps identify the patient population — which Kumar says is in the hundreds of thousands — through improved diagnosis.
“It’s almost like Alzheimer’s of the heart, one of the biggest genetic markets out there,” he adds.
Eidos shares got a 10% boost on the plan, but the biotech is playing a tough game, against tough odds.
Pfizer’s marketing machine is one of the biggest and best in the business. Any new launch like this will be given a top priority. And they’ll have an early mover advantage, which in this marketplace comes with a ton of built-in advantages.
Eidos also is hoping to score early with 12-month data against the more substantial 30-month outcome data that Pfizer already has on hand. Kumar counters, though, that his team will be able to demonstrate improved efficacy where it counts with the specialists who matter the most. Then they can follow up with rival 30-month data to seal the deal.
There are, of course, no guarantees that he’s right.
We’ll see how this plays out.
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