It's almost time for the American Academy of Opthalmology's (AAO) annual meeting again, and this year's event will contain some high-stakes presentations. This year, all three of these biopharmaceutical companies will present clinical trial results that could send their stock prices soaring -- or tumbling.
| Company | New Drug Candidate | Year-to-Date Gain/(Loss) | Market Cap |
|---|---|---|---|
| Kodiak Sciences (KOD +11.11%) | KSI-301 | 95% | $513 million |
| Adverum Biotechnologies (ADVM +0.00%) | ADVM-022 | 79% | $359 million |
| Regenexbio (RGNX +3.26%) | RGX-314 | (9%) | $1.42 billion |
Data source: Company Filings, Yahoo! Finance.
Which stocks have the best chance to climb this week? Here's what to look out for when investigators from each company present their results at AAO.
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Kodiak Sciences: Durability Data
Ophthalmologists have been treating age-related macular degeneration (AMD) and similar causes of blindness with vascular endothelial growth factor (VEGF) inhibitors like Eylea from Regeneron for a long time. Sadly, many patients continue losing their sight despite VEGF treatment and Kodiak has an elegant solution to the problem.
Ophthalmologists would like to apply a VEGF treatment to the retina every few days but nobody wants to visit the doctor to get a needle in the eye more often than necessary. Four- and eight-week intervals are the norm and toward the end of each interval, there are days where treatments have already been depleted. That allows new blood vessels to begin invading the retina until the next generation.
On Friday, Oct. 11, 2019, Kodiak will present durability data from an ongoing study with patients that are losing their vision to AMD or a related disorder. The company's lead candidate, KSI-301 is a VEGF antibody with long, heavy hair extensions called phosphorylcholine. That's the same biopolymer used in drug-eluting stents.
Kodiak's KSI-301 appeared to work well after at a 12-week checkup, but that's not very long. If durability data presented on Friday continues to impress, this stock's terrific run-up in 2019 could go much further.
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Adverum Biotechnologies: Gene Therapy
In September, Adverum Biotechnologies stock sank after the company reported 24-week data for the first six patients treated with a single injection of ADVM-022, its experimental gene therapy for many of the same AMD patients that Kodiak's aiming at. A one-time treatment that keeps invading blood vessels at bay for the rest of a patient's life would be a welcome improvement, but patients experienced an average loss of two letters when their best-corrected visual acuity (BCVA) scores were assessed.
While Adverum hailed the results as a success because none of the patients received rescue injections with a regular VEGF treatment, some observers wondered why rescue injections weren't provided. According to Adverum, AMD wasn't responsible for the observed vision losses, and the gene therapy actually worked as expected.
On Friday, Oct. 11, Adverum will present more details from the disappointing 24-week data that hammered the stock a month earlier. If the company can convince investors that the results they saw earlier contained strong signs of encouragement, the stock could regain some of last month's losses.
Unfortunately, ADVM-022 doesn't appear to work any better than AVA-101, another experimental gene therapy that Adverum quit developing years ago when the company was still named Avalanche Biotechnologies. Investors should know that the company ended up settling a class action lawsuit for $13 million rather than try to defend itself against allegations of false statements and omissions of important information that would have sent investors running for the hills much earlier than they did in 2015.
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Regenexbio: Ascending doses
At last year's AAO conference, Regenexbio stock soared after the company presented early clinical trial results for RGX-314, an experimental gene therapy for AMD patients. In May, the company completed dosing all 42 patients in the trial with five escalating dosages of RGX-314. On Friday, Oct. 11, Regenexbio will present 18-month data for cohort three and interim data from cohorts four and five.
Only three out of six patients in group three were still VEGF injection-free 18 months after a single dose of RX-314, so success with a larger dosage is a must for this program. If interim data from cohorts four and five lead to a stronger response that keeps invasive blood vessel formation from robbing patients of their vision.
So far, we've seen dosage-dependent responses in cohorts one through three, and a continuation of this trend in cohorts four and five could send this biotech stock through the roof.
Image source: Getty Images.
Further ahead
While Adverum's gene therapy seems headed for further trouble, Regenexbio's gene therapy has a pretty good shot at impressing investors later this week. Further down the line, however, Kodiak Sciences probably has the best chance to successfully launch a new drug for the treatment of AMD and related causes of blindness.
There's a lot to like about single-shot solutions from Adverum and Regenexbio, but they could end up solving a problem that isn't really a big problem anymore. That's because Kodiak's candidate might be able to outperform standard care with just a couple of injections per year instead of every four to 12 weeks as is the norm today.
