How This Biotech IPO Is Facing Off Vs. BioMarin In Metabolic Disease


Rubius Therapeutics (RUBY) could rival BioMarin Pharmaceutical (BMRN) in treating a metabolic disorder, but first the duo will face a dark horse candidate in the form of Synlogic (SYBX).


Leerink analyst Jonathan Chang recently initiated coverage of Rubius stock with an outperform rating and a 30 price target. On Wednesday, he imagined Rubius could carve itself a chunk of the market in treating phenylketonuria, rivaling two approved drugs from BioMarin.

It's possible Rubius' treatment — derived from red blood cells — could have fewer side effects than BioMarin's recently approved drug called Palynziq, Chang said in a note to clients. It could also be stronger than Kuvan, a drug from BioMarin that U.S. officials approved in 2007.

Chang said one noted physician believes that "Rubius' red cell approach is 'intriguing' for phenylketonuria and that there could be a place for Rubius' treatment if it is (effective)."

But Rubius stock plunged 6.3%, to 23.10, on the stock market today. Investors weighed the results of a study from Synlogic for a phenylketonuria drug. Synlogic stock, on the other hand, popped 18.9%, to 12.76. BioMarin stock lifted 0.9%, to 96.60.

Treating Phenylketonuria

Phenylketonuria is a rare genetic disorder in which an amino acid called phenylalanine builds up in the body. In healthy people, an enzyme breaks down phenylalanine. But in phenylketonuria patients, there's a defect in the gene responsible for creating that enzyme.

Physicians have identified patients via newborn screening since the 1960s. Patients often have a protein-restrictive diet to help control the cognitive side effects of the disease. Older patients, who didn't begin dietary restrictions early enough, often have psychiatric symptoms.

BioMarin's Kuvan works with varying degrees in milder patients, a physician told Chang. The drug has an "outstanding safety profile with virtually no significant side effects."

Palynziq works in more severe patients. Specifically, Palynziq injects a substance that exists in bacteria, but isn't common in mammals. That substance breaks down dangerous phenylalanine in a unique way. But because it's a foreign material, the immune system can react to it.

Some patients may need to carry an EpiPen to combat related side effects, Chang said.

"Overall, it's a very complicated protein and early trials took time to figure out the appropriate dosing schedule," Chang said.

Using Red Blood Cells

Here's where Rubius' therapy is interesting. It uses the same substance delivered via a red blood cell product. This could help to protect patients from an immune system attack, Chang said.

"Presumably, the patients wouldn't have to do frequent dosing if the red (blood) cells have good staying power in circulation," he said.

So far, testing in mice has had good results. Although the physician noted it's easy to prompt enzyme breakdown in mice. It's much more difficult in larger humans.

"But if comparable data are seen in humans that would be great with the data showing nice lowering of phenylalanine levels," he said.

Synlogic, on the other hand, said Tuesday it tested its treatment in healthy volunteers. The drug had no severe side effects. Moderately severe side effects included nausea and vomiting. Next, Synlogic plans to test its drug in patients with phenylketonuria.


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