This Gene Therapy Player Duchenne Data Is Impressive, Analyst Upgrades REGENXBIO

RBC Capital Markets upgraded REGENXBIO Inc RGNX, a company focused on developing gene therapies.

Recently, REGENXBIO reported additional interim safety and efficacy data in the Phase 1/2 AFFINITY DUCHENNE trial of RGX-202 in patients with Duchenne muscular dystrophy (Duchenne) ages 4 to 11.

In new data from the first patient, aged 12.1 years, who received RGX-202 at dose level 2, RGX-202 microdystrophin expression was measured to be 75.7% compared to control at three months. 

Related: Gene Therapy Player REGENXBIO’s RGX-121 Marks Success In Trial, Yet Faces Challenges In A Crowded Market: Analyst.

A reduction in serum creatinine kinase (CK) levels of 77% was observed at ten weeks from baseline.

Elevated CK levels are associated with muscle injury and are uniformly elevated in Duchenne patients. 

RBC Capital remains cautious about the company’s ocular programs, though the analyst notes that the recent execution of DMD has impressed and should drive the stock higher from here.

While the data is still early, the RBC analyst highlights that the crucial question of whether retaining the CT domain can result in better outcomes is yet to be determined. 

Nevertheless, the expression data is impressive, and the upcoming PDUFA for Sarepta Therapeutics Inc SRPT can be a significant catalyst. 

RBC upgraded REGENXBIO stock to Outperform from Sector Perform, with a price target of $35, up from $20.

The analyst underwrites that 15-20% of patients are not eligible for Sarepta/Pfizer Inc PFE due to neutralizing antibodies. 

The analyst notes a potential upside if REGENXBIO shows better benefits or captures a broader population. 

Read Next: Median Drug Prices For Rare Diseases Hit $300K In 2023, Report Highlights Lack of Clear Rationale Behind Escalating Drug Prices.

Price Action: RGNX shares are down 0.95% at $23.01 on the last check Friday.

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