Phase III Failure of AAV Gene Therapy Bota-vec Triggers 11% Stock Drop for MeiraGTx: What’s Next for AAV-Based Therapeutics?

May 06, 2025 18:31 CST Updated 18:31

MeiraGTx

Developer of Innovative Gene Therapy Products

Beacon Therapeutics

Ophthalmology Gene Therapy Technology Researcher

Recently,StrongLife Announces ItsGene Therapy Purchased for $415 Millionbota-vec, used inXLRP（X-linkedRetinitis Pigmentosa)Failed in Phase III clinical trials.

Or affected by this news, the original research pharmaceutical companiesMeiraGTxAs of the closing on May 5th,Stock price plummeted by 11.03%.By the end of 2023,Johnson & JohnsonIn$65 millionDown payment,$415 million total transaction price,FromAcquired by MeiraGTxGlobal rights of bota-vec.

According to reports,Bota-vec isA product based on adeno-associatedViral Vector（AAV）Gene therapy for the treatment ofByRPGR（Retinitis PigmentosaGTPase Regulator）Caused by gene mutationsXLRP。

Image Source: Johnson & Johnson Official Website

XLRP isRP（Retinitis Pigmentosa) ofA GeneticClassType, namelyX-linkedRecessive Inheritance。According toChinese Journal of Medical Genetics, published in 2020, "Clinical Practice Guidelines for Retinitis Pigmentosa》, this genetic typeAboutAccounting for a total of RP5%～15%。

In the United States and Europe,RPThe incidence rate is 1/4000 to 1/3000, and about 1/3500 in China, thereforeXLRP is undoubtedly rarer. Currently, there are no targeted therapies approved in China or abroad.

bota-vecBefore Johnson & Johnson introduced it, it had beenFDA grantedFast Track Designation andOrphan Drug Designation. This setbackPhase III Clinical Trial NamedLumeos, recruiting 95 patients,The primary endpoint was set as the improvement in patients' ability to visually navigate through a virtual maze.

So-calledVirtual Maze,Refers to a three-dimensional maze environment generated by a computer, where subjects need to complete navigation tasks in the virtual space using visual cues (such as wall textures, path markers, target locations). This method is commonly used forEvaluate the visual perception, spatial cognitive ability, or navigation skills of the subjects.

The results showed,The trial did not meet the primary endpoint of improving visual navigation ability.But Johnson & Johnson did not give up and plans to conduct follow-up research.

Image Source:clinicaltrials.gov

In terms of safety,All enrolled patients were at leastAppearIn a single treatmentAdverse events, 86% of which were moderate or severe in severity, and 53% of patients experienced at least one adverse event related to bota-vec.

In March this year, withSarepta'sGene TherapyElevidysFirst Treatment-Related Death Case Reported,Safety issues related to AAV vectors have once again come into the industry's view.Bota-vec, as an ophthalmic medication, has an adverse reaction rate that requires attention.

Currently, accompanied byThe clinical failure of bota-vec,The first tier in the XLRP treatment field has only MeiraGTx Limited and Beacon Therapeutics, Ltd. left.Beacon TherapeuticsAGTC-501, which is currently under investigationPhase 2/3 clinical trial.

Compared tobota-vec，AGTC-501 can express the full-length RPGR protein, thus treating all photoreceptor damage caused by XLRP, including the loss of rod and cone cells.Bota-Vec usesTruncated RPGR protein,SeemsOnly affects peripheral vision, without affecting central vision.

Whether Beacon Therapeutics can overtake in the curve remains to be seen.

Announced by Johnson & JohnsonNot long after the clinical failure of bota-vec,Vertex announced the termination of itsResearch on AAV Gene Therapy.

In February this year,VertexAnnouncementReturn the rights of the liver disease gene editing project toVerve Therapeutics, this transaction can be traced back to 2022,VertexAn upfront payment of $60 million and up to $400 million in milestone payments. In March this year,Vertex Announces TerminationDevelopment of Islet Cell Therapy VX-264.

Image Source:Vertex Official Website

It can be saidVertex's exit has leftThe overseas market enthusiasm for AAV gene therapy is about to hit rock bottom. Previously, with...Pfizer,Takeda,RocheAndTop pharmaceutical companies like BiogenReducedAAV Gene Therapy R&D or Termination of Related Clinical Projects

However,The cold wind from overseas has not blown to China.. According to incomplete statistics,In China, there is already50 AAV ProductsGene therapy IND applications approved, with 8 entering Phase III clinical trials and 1 approved for marketing.

Among them, individual companies inHas achieved a leading position in localized gene therapy,The world's first inner ear gene therapy trial and dual AAV vector clinical trial to achieve clinical efficacy have been completed in China, ushering in a truly curative era for deafness.

For marketed products,Novartis for SMAAAVThe gene therapy Zolgensma achieved approximately $1.351 billion in sales revenue in 2021 and has maintained annual sales exceeding $1 billion in subsequent years, becoming the first blockbuster drug in its category.

AndElevidys，Total net revenue in 2024 was US$821 million, including theSales exceeding $200 million have also reached $1 billion.

In summary, inIn the field of AAV gene therapy, there is a significant divide between China and foreign countries, as well as between products under research and those already on the market.

Image Source: 123rf

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