The biopharmaceutical industry's summer doldrums will unofficially come to a close this week thanks to steady gusts of wind from the Food and Drug Administration, plus a slate of presentations in front of scientists and investors.
Some events are more important than others. If you only have time to focus on a few of the stories about to unfold this week, make sure you don't miss upcoming catalysts for Alnylam (NASDAQ:ALNY), Moderna (NASDAQ:MRNA), and Aimmune Therapeutics (NASDAQ:AIMT). Here's what to look out for.
Tuesday: Alnylam -- how limited?
Alnylam's first drug to earn approval, Onpattro, is aimed at a very limited population. Although it carries a six-figure price tag, sales of the transthyretin (TTR) reducing RNA-interference drug reached just $38 million in the second quarter.
A $152 million annualized run rate for a company's first drug launch is commendable, but it didn't come cheap. Launching one new drug while developing a pipeline bursting with potential new drugs has driven operating expenses up to an annualized $1.1 billion.
Alnylam clearly needs to bump up sales, but investors are worried that its next approved drug might not have what it takes, commercially speaking. In April, Alnylam showed us pivotal results that say givosiran could lower the annualized rate of painful attacks of hepatic porphyria (AHP) by 74% versus a placebo. Unfortunately, around half of the patients treated still experienced at least one attack.
AHP is a blanket term that describes hemoglobin formation issues that can be caused by a handful of different gene mutations that result in the buildup of aminolevulinic acid (ALA) and porphobilinogen (PBG). Givosiran inhibits ALA production, but that might not be enough to help people similar to the 50% of patients who experienced an attack during the pivotal Envision study.
Alnylam has already rushed an application for the treatment of AHP to the FDA for givosiran, but there's a chance the agency might limit an already small audience even further. An estimated 50,000 Americans are affected by AHP, and on Tuesday, Sept. 10, the company will present further results that tell us if givosiran will have a chance to treat the entire AHP population or just a sliver.
Moderna: What's next?
Moderna pulled off an impressive initial public offering earlier this year, but the stock has fallen around 40% since a peak reached in May. Even with that, it sports a $5.3 billion market cap, but still doesn't have a lot of convincing evidence that its messenger RNA (mRNA) drugs work as hoped.
A first look at Moderna's cancer vaccine, mRNA-4157, wasn't necessarily bad, but it wasn't the rousing success that investors were expecting. In a nutshell, it's hard to say whether mRNA-4157 helped two different groups of cancer patients.
Moderna lost $268 million in the first half of the year and started the ball rolling on several clinical-stage proof-of-concept trials. Now, there are 13 drugs in clinical-stage development, which means losses are going to accelerate.
The company's balance sheet looks solid with $1.4 billion in cash, but that will only last another year and a half before Moderna needs to visit the equity tap again. That makes it awfully important to reassure shareholders that the rest of the company's mRNA drugs are viable at this year's research and development day on Sept. 12.
Aimmune: Net benefit?
On Friday, Sept. 13, the FDA will assemble a panel of independent physicians to discuss a new drug application from Aimmune. The agency doesn't have to follow the panel's recommendation, but it usually does.
Aimmune's candidate, AR101, could become the first protective therapy for peanut allergy, but approval is far from guaranteed. That's because AR101 is just peanut allergen powder delivered in low, measured doses that have been shown to desensitize patients to the allergen.
While there's no question about AR101's ability to desensitize people to the peanut allergen, the agency could hold up the application by asking for evidence that desensitization with AR101 leads to fewer dangerous reactions related to accidental peanut exposure in the real world.
We'll find out just how concerned the FDA is about the overall risk-to-benefit ratio regarding AR101 when the agency releases official briefing documents at least two days ahead of the meeting. If the regulator and independent experts agree there's a net health benefit to desensitization with AR101, the stock will soar.
Most likely to move
Alnylam has a large enough late-stage pipeline that great (or lousy) data for givosiran might not move the stock very far. With so many active programs, it's hard to guess what Moderna will reveal at its annual R&D day, but we know it will involve early clinical-stage development at best.
Aimmune is the stock here that's most likely to make a big move this week. So far, the only thing we know about the FDA's attitude toward AR101 has been conveyed to us through Aimmune. The briefing documents provided by the FDA to the independent advisory committee and the general public will be the first time we get to read about the regulator's concerns unfiltered.