Rocket Pharmaceuticals Receives FDA Drug Clearance

Genetics Investing

The company’s drug, PR-L201, is in development to treat Leukocyte Adhesion Deficiency-I, a rare pediatric disease.

Rocket Pharmaceuticals (NASDAQ:RCKT) announced Monday (November 19) its gene therapy drug has been cleared by the US Food and Drug Administration (FDA) ahead of schedule.

The company’s drug, PR-L201, is in development to treat Leukocyte Adhesion Deficiency-I (LAD-I), which is a rare pediatric disease.

With this Investigational New Drug (IND) application clearance, the company’s drug is now ready to enter the first stage of clinic development in a Phase 1/2 clinical trial.

“This IND enables Rocket to initiate a Phase 1/2 clinical trial to assess the safety, tolerability, and efficacy of severe LAD-I in children,” Dr. Gaurav Shah, CEO of Rocket, said in the press release.

According to the announcement a Phase 1, open-label, single-arm study will be initiated in the US with two patients. Shah confirmed the trial will begin next year. The Phase 2 study for the drug will be a global study in the US, UK and Spain.

The drug is a lentiviral vector (LVV)-based gene therapy drug, which was in-licensed by the Centre for Energy, Environment and Technology (CIEMAT), which is a public research body in Spain.

LAD-I is caused by a mutation of the ITGB2 gene, which encodes the Beta-2 integrin component CD18. This leads to impaired CD18 expression and white blood cell migration. From birth, patients have severe and recurrent, life-threatening infections that don’t respond well to antibiotics.

About two-thirds of these patients die before the age of two. Current treatments rarely address the need as stem cell transplants are also associated with graft vs host disease. Infections include: pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. About 25-50 patients could be treated per year if this treatment is approved, according to the Rocket’s company presentation.

The presentation also says some potential clinical endpoints for the trials could be modest correction of the CD18 expression and patient survival. Recruitment is underway globally and the US principal investigator for the trial has been identified.

For product manufacturing optimization, GMP-quality vector production is ongoing and its transduction optimization.

Rocket is a clinical-stage company focused on gene-therapy treatments for rare diseases. The company’s multi-platform development addresses adeno-associated viral vector (AAV) aside from the LVV gene therapy platforms. The company’s RP-L102 drug is its most advanced drug, which is in clinical development for a monogenic DNA disorder, fanconi anemia (FA).

Investor takeaway

On Monday, Rocket’s share price decreased under four percent to US$13.70 as of market close.

According to TipRanks, the most recent note to investors was from Raju Prasad with William Blair. Prasad issued a “Buy” position for the company without a price target.

Including the IND for this drug, Rocket expects to have four programs in the clinic by the end of 2019. Investors can also look forward to additional FA clinical data from, which should be released in the next 12-18 months.

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Securities Disclosure: I, Gabrielle Lakusta, hold no direct investment interest in any company mentioned in this article.

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