FDA grants orphan drug status to Precision BioSciences’ DMD treatment

DURHAM, N.C. - Precision BioSciences, Inc. (NASDAQ:DTIL), a biotechnology company currently trading at $4.86 and showing a notable 27.5% gain year-to-date, announced Wednesday that the U.S. Food and Drug Administration has granted Orphan Drug Designation to its PBGENE-DMD treatment for Duchenne muscular dystrophy (DMD). According to InvestingPro analysis, the company maintains a strong liquidity position with a current ratio of 6.62.

The designation provides certain benefits including financial incentives to support clinical development and the potential for up to seven years of market exclusivity in the U.S. if the drug is ultimately approved for the designated indication.

PBGENE-DMD is designed to use two complementary ARCUS nucleases delivered via a one-time administration to excise exons 45-55 of the dystrophin gene. According to the company, this approach aims to restore near full-length dystrophin protein to improve functional outcomes and could potentially address up to 60% of the DMD patient population.

"Receipt of Orphan Drug Designation from the FDA for PBGENE-DMD underscores the tremendous unmet need and urgency to deliver safe treatments that significantly improve muscle function over time for boys living with Duchenne muscular dystrophy," said Cindy Atwell, Chief Development and Business Officer at Precision BioSciences.

The FDA grants Orphan Drug Designation to treatments intended for rare diseases affecting fewer than 200,000 people in the United States.

This regulatory milestone follows the company’s recent receipt of Rare Pediatric Disease designation for the same treatment. Precision BioSciences stated it is advancing final IND-enabling toxicology studies and working with DMD clinicians to design initial human trials, with clinical data expected in 2026. InvestingPro data reveals the company holds more cash than debt on its balance sheet, though analysts note rapid cash utilization - crucial factors for investors monitoring the company’s ability to fund its clinical development pipeline. Get access to 8 more exclusive InvestingPro Tips and comprehensive financial analysis with a subscription.

In preclinical studies, the company reported that PBGENE-DMD demonstrated the ability to target key muscle types involved in DMD progression and produced functional improvements in a humanized DMD mouse model.

The information in this article is based on a press release statement from Precision BioSciences.

In other recent news, Precision BioSciences has made significant strides in its efforts to treat Duchenne muscular dystrophy (DMD). The company announced that its gene editing therapy, PBGENE-DMD, received Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA), emphasizing its potential impact on the 15,000 Americans affected by this rare genetic disorder. Additionally, Precision BioSciences was granted FDA Orphan Drug Designations for its DMD19-20L.431 and DMD35-36L.457 nucleases, further supporting their development for DMD treatment. Promising preclinical data revealed a substantial increase in dystrophin-positive muscle cells in a DMD mouse model, showcasing up to an 85% positivity rate in the calf muscle. This development highlights the potential efficacy of their PBGENE-DMD candidate across multiple muscle types. In corporate governance news, the company held its annual shareholder meeting, where Kevin J. Buehler and Shari Lisa Piré were elected as Class III directors. The meeting saw participation from approximately 71% of the company’s outstanding common stock. These developments reflect Precision BioSciences’ ongoing commitment to advancing its gene editing technologies and strengthening its leadership team.

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