Stifel reiterates Buy rating on Neurogene stock, backs pivotal trial design

Published 01/07/2025, 14:12
Stifel reiterates Buy rating on Neurogene stock, backs pivotal trial design

Investing.com - Stifel maintained its Buy rating and $46.00 price target on Neurogene (NASDAQ:NGNE), representing over 200% upside from current levels, following the company’s regulatory and pivotal trial update for its Rett Syndrome treatment NGN-401. According to InvestingPro data, the stock appears undervalued based on its Fair Value analysis.

The investment firm expressed confidence in Neurogene’s agreed-upon pivotal study design, which will utilize a small, single-arm approach with a primary outcome composite that Stifel believes looks "favorable/doable" based on existing NGN-401 data. With a strong current ratio of 21.03 and more cash than debt on its balance sheet, the company appears well-positioned to execute its clinical programs. (InvestingPro subscribers can access 8 additional key insights about NGNE’s financial health.)

Stifel noted that the study design provides "affirmation of clinical meaningfulness" and should help address investor concerns that emerged after a patient death in the NGN-401 high dose trial last fall raised questions about potential FDA flexibility.

Two key differences distinguish Neurogene’s study from competitor TSHA’s pivotal trial: Neurogene can enroll patients as young as three years old compared to TSHA’s minimum age of six, and Neurogene’s primary outcome leverages a gain of milestone responder with CGI-I in a composite.

The favorable regulatory update supports Stifel’s continued bullish stance on the Rett Syndrome treatment candidate, which represents a significant program in Neurogene’s development pipeline.

In other recent news, Neurogene Inc. has received approval from the U.S. Food and Drug Administration on the design of its registrational trial for NGN-401, a gene therapy targeting Rett syndrome in females aged 3 years and older. This trial, named Embolden, will evaluate the therapy’s impact on clinical improvement and developmental milestones over a 12-month period. Craig-Hallum initiated coverage on Neurogene with a buy rating, highlighting NGN-401’s potential as a leading treatment for Rett syndrome. The firm cited Neurogene’s regulatory technology and manufacturing capabilities as competitive advantages. Additionally, BMO Capital raised its price target for Neurogene to $26, noting the effectiveness of the updated monitoring protocol for the NGN-401 treatment. Stifel maintained a buy rating with a $46 price target, acknowledging the competitive landscape and recent updates from competitors. The company’s progress, including the management of adverse events and regulatory updates, continues to be a focal point for investors. Neurogene’s expected data release later this year is anticipated to provide further insights into the potential of NGN-401.

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