15 For ’19: Key Clinical Data to Watch For Next Year (Part 2)

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another experimental drug: inclisiran, from The Medicines Co. (NASDAQ: MDCO) and partner Alnylam Pharmaceuticals (NASDAQ: ALNY).

The proposed benefit is convenience. Alirocumab and evolocumab have to be administered once or twice a month, while inclisiran could potentially be injected just two or three times a year, which may help with patient compliance and lead to better control their cholesterol. MedCo has made a huge bet on that thesis: it jettisoned other programs, chopped its workforce, and raised $150 million in convertible notes to pay for the pricey late-stage studies underway.

In ORION-9, ORION-10 and ORION-11, patients with heart disease are getting either a placebo or four total doses of inclisiran over the course of 18 months. Data are expected in the second half of 2019.

Will the results matter to payers and physicians, or will they wait for the results of MedCo’s big “outcomes” study, akin to the ones Amgen and Regeneron have already run? And if inclisiran makes it to market, how will MedCo—which has seen the commercial struggles of Regeneron and Amgen—price the drug? Those are some of the questions ahead for MedCo, which expects to file for FDA approval by the end of 2019 if the ORION results are positive.

Like alirocumab and evolocumab, inclisiran aims to lower the levels of the protein PCSK9; lower levels correlate with better clearance of LDL from the blood. Inclisiran uses RNA interference—a method of stopping genes from producing disease-causing proteins—to prevent the liver from making PCSK9 in the first place. RNAi is a drug-making method that has just come of age after years of ups and downs. In August, Alnylam’s patisiran (Onpattro), for a rare genetic disease, became the first-ever FDA-approved RNAi medicine.

The ORION studies are the first big test of RNAi in a broader population and could signal that this new way of attacking disease could have a wider field of play.

Disease Area: Spinal muscular atrophy

Companies: Novartis, Roche

Trials: STRONG/SPR1NT (Novartis), FIREFISH/SUNFISH (Roche)

Data Expected: 1H 2019 (Novartis), 2019 (Roche)

Why We’re Watching: Things have begun to change for patients with the rare genetic disease spinal muscular atrophy. And that change is about to accelerate.

SMA robs people of their ability to walk and function independently. Some types of SMA can be lethal. There were no drugs available until late 2016, when the FDA approved nusinersen (Spinraza) from Biogen (NASDAQ: BIIB), an RNA-based treatment infused a few times a year. In clinical studies nusinersen showed the ability to slow the disease’s progression for people with multiple forms of the disease. A gene therapy and a pill may soon follow, setting the stage for multiple treatment options and a high-stakes commercial battle between Biogen, Novartis, and Roche.

Likely on its way first is a gene therapy, AVXS-101 (Zolgensma). Novartis paid $8.7 billion to buy AveXis and get its hands on the treatment, which offers long-lasting effects from a single infusion and is under regulatory review in the U.S., Europe, and Japan. But the data are only for SMA Type 1, the most severe form that afflicts infants.

Next year, Novartis aims to prove the gene therapy is effective in other groups, as Biogen has shown with nusinersen. Watch for two key studies: STRONG, for Type 2 patients; and SPR1NT, for patients with Type 1, 2, or 3 who have yet to show symptoms. Both are expected to produce data no later than the American Academy of Neurology’s big meeting in May 2019.

Then there’s Roche. Through an alliance with PTC Therapeutics (NASDAQ: PTCT), the company is developing a drug called risdiplam. Like nusinersen and AVXS-101, risdiplam works by boosting levels of the key protein, survival motor neuron, that SMA patients lack. But risdiplam is a liquid taken by mouth, or via a gastronomy tube for people who can’t swallow. It’s thus more familiar to doctors and payers than a one-time gene therapy (AVXS-101) that could cost millions of dollars—Novartis has said a $4 million price tag would be justified, given its benefits—or a complex spinal injection (nusinersen) that costs $750,000 for the first year and $375,000 each one thereafter.

Roche, of course, has to prove that risdiplam can hang with its two rivals. Nusinersen has by far the most extensive body of data, followed by AVXS-101. Risdiplam has produced encouraging early data from the trials FIREFISH, in Type 1 patients, and SUNFISH, in Type 2 and 3 patients. The same trials will produce more substantial information next year, and strong data could prompt Roche to ask for FDA approval in 2020. Two more studies—JEWELFISH (patients who have been treated with other SMA drugs) and RAINBOWFISH (pre-symptomatic patients)—have started, or soon will.

Disease Area: Sickle cell disease

Company: Global Blood Therapeutics

Trial: HOPE (Part A)

Data Expected: 2019

Why We’re Watching: Genome-altering medicines, like the one noted above and this one, could one day fix the mutated gene that produces sickle cell disease. But complicated biology, health economics, and other factors make that prospect highly speculative. Even in a best-case scenario, gene therapies are years away.

Meanwhile, roughly 100,000 people in the U.S., predominantly African-American, and millions more around the world with the disease need relief now. Sickle cell’s telltale malformed red blood cells clog vessels and can’t carry enough oxygen to muscles and other tissues. Patients suffer bouts of excruciating pain that often force hospital visits, as well as strokes, organ damage, and early death, even with the best treatment.

The new drug closest to market is voxelotor from Global Blood Therapeutics (NASDAQ: GBT), a once-a-day pill that targets a key blood protein, hemoglobin, and aims to prevent red blood cells from bending into the malformed sickle shape. GBT has reported … Next Page »

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