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15 For ’18: Key Clinical Data to Watch For Next Year (Part 2)

Xconomy National — 

[Corrected, 12/11/17, 1 p.m. ET. See below.] On Monday we posted the first part of our look at what should be some of 2018’s most important clinical data, including trials for lung cancer, heart disease, melanoma, Alzheimer’s disease, rare blood disorders, and more. With part two, we’re previewing studies for migraine, brain cancer, lymphoma, peanut allergy, schizophrenia, the rare Hunter syndrome, and the flu.

[Ben Fidler, Corie Lok, and Frank Vinluan contributed to this report.]

Disease area: Migraine

Company: Alder Biopharmaceuticals

Trial: PROMISE 2

Data expected: First half of 2018

Why we’re watching: Alder (NASDAQ: ALDR) is among the companies developing drugs to prevent migraine headaches before they start. These drugs, called calcitonin gene-related peptide (CGRP) inhibitors, block a protein thought to play a role in the transmission of pain.

Amgen (NASDAQ: AMGN) and partner Novartis (NYSE: NVS), Eli Lilly (NYSE: LLY), and Teva Pharmaceutical (NYSE: TEVA) have already filed for FDA approval for their respective CGRP migraine drugs. Like Alder’s drug, eptinezumab, they are all biological drugs. But Bothell, WA-based Alder hopes to stand apart with eptinezumab by requiring an infusion just once every three months, instead of the monthly dosing of the Amgen and Lilly drugs. Teva, however, has also reported success testing its drug for quarterly dosing.

Alder, by far the smallest of the competitors, does not yet have any FDA-approved therapies. CEO Randy Schatzman said recently that eptinezumab is its main focus. All four companies have tested subcutaneous dosing of their respective drugs, though Schatzman said Alder first plans to win approval of the infused version before pursuing the subcutaneous version, which would offer patients another, potentially more convenient, option. [Corrected to clarify that all four companies have tested subcutaneous forms of their migraine drugs.]

Alder has already reported positive results from the PROMISE 1 study, which tested eptinezumab in patients with episodic migraine—five to 14 migraine headaches per month. PROMISE 2, also a Phase 3 study enrolling more than 1,000 patients, is for chronic migraine patients, defined as having more than 15 headaches per month. The goal is to ask for FDA approval in the second half of 2018 to treat both episodic and chronic migraine patients. That would line with the plans that Amgen, Lilly, and Teva, have already submitted.

Disease area: Peanut allergy

Company: Aimmune Therapeutics

Trial: PALISADE

Data expected: First quarter of 2018

Why we’re watching: An estimated 30 million people in the U.S. and Europe have a food allergy, and peanut allergy is among the most common. Aimmune (NASDAQ: AIMT) is taking an immunotherapy approach. The Brisbane, CA, biotech’s lead drug, AR101, is a pill formulation of peanut protein meant to desensitize patients to the allergen. Last year, Aimmune completed enrollment of 554 patients in its Phase 3 study, dubbed PALISADE. In Aimmune’s randomized controlled Phase 2 study enrolling 55 patients, six dropped out of the treatment group due to intestinal side effects. The company said those problems resolved after patients stopped taking the drug.

While there are no FDA-approved therapies that work the way Aimmune’s drug does, Paris-based DBV Technologies (NASDAQ: DBVT has developed an immunotherapy approach to peanut allergy with a skin patch called Viaskin. DBV last month reported early positive results from a study with more than 700 patients.

Meanwhile, Aimmune’s work has caught the attention of big players in the food industry. The company last year entered a two-year collaboration with Nestlé Health Science, which gained a three-month exclusive negotiating window and also bought $145 million in Aimmune stock. The Nestlé portfolio already includes a nutritional product for people with allergies to cow milk proteins. Aimmune expects to file for approval of its therapy in the U.S. and Europe by late 2018.

Disease area: Influenza

Company: Moderna Therapeutics

Trials: Phase 1

Data expected: TBA, 2018

Why we’re watching: Moderna Therapeutics, the high-flying, secretive, privately-held company reportedly worth some $5 billion—at least a few months ago—aims to prove the worth of an entirely new way of making drugs. Moderna injects patients with synthetic messenger RNAs, which slide into cells with instructions for making proteins that the patients need. In other words, Moderna wants to turn a patient into his or her own drug factory.

That’s the idea, anyway. Moderna has attracted envy and criticism for its ability to raise an exorbitant amount of cash with little evidence that its technology works. But its first publicly disclosed clinical data trickled in this year, and more substantial results are due in 2018.

This year, Moderna reported that its two experimental mRNA flu vaccines were well tolerated. Next year, Moderna plans to publish complete findings from the phase 1 studies of mRNA-1440 and mRNA-1851, meant to protect against the H10N8 and H7N9 flu strains, respectively. Both strains are thought to have pandemic potential. H7N9, a bird flu that can spread to humans, has infected more than 1,500 people globally, killing about 40 percent of them. H10N8 is another bird virus and has sickened only a few people. Neither flu strain has an approved vaccine.

Moderna has early-stage studies in cancer and infectious disease that may also produce results in 2018, but the flu results will be the most detailed picture yet of Moderna’s mRNA technology and a much better stress test of what, for now, seems like an impossibly high valuation.

Disease area: Schizophrenia

Company: Alkermes

Trial: ENLIGHTEN-2

Data expected: Fall 2018

Why we’re watching
: Weight gain is a common side effect of psychiatric medications, and it’s one that Alkermes (NASDAQ: ALKS) hopes to avoid with its drug, ALKS 3831. The drug has already shown … Next Page »

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