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Latest Up and Coming Gene Therapies

Posted on: 8 May 2018, source: GEN
Gene therapy’s numbers look promising. In a year full of biopharma mergers and acquisitions, one of the biggest deals has been Novartis’ planned $8.7 billion purchase of AveXis, whose lead candidate AVXS-101 has advanced into a pivotal trial following positive Phase I data published in November in The New England Journal of Medicine. Additional figures furnished to GEN by Informa show the extent of gene-therapy development: Informa Pharma Intelligence’s Trialtrove database records 729 gene therapies as having been developed, of which nearly two-thirds (461) were preclinical. Those therapies have been assessed in 1,855 clinical trials, most in early phases: 657 in Phase I, 509 in Phase I/II, and 455 in Phase II. As for later development, 89 have reached Phase III, 32 are in Phase II/III, and 28 in Phase IV.


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By far the most crowded therapeutic area is oncology, accounting for two-thirds or 1,254 of the 1,855 trials. The next-largest indication is cardiovascular with 214 trials (11.5%), followed by infectious disease (6.5%).

Clinical activity is expected to increase, in part due to the FDA’s Regenerative Medicine Advanced Therapy (RMAT) designation, created through the 21st Century Cures Act. “There are a lot of incentives for sponsors who get RMAT, including early and frequent interactions with the FDA, as well as the ability to discuss early on any potential surrogate or intermediate endpoints in their clinical trials,” Amanda Micklus, principal analyst with Informa Pharma Intelligence, told GEN. “The RMAT designation potentially could really advance the progress of these therapies through the pipeline.”

Patricia Reilly, Informa Pharma Intelligence vice president, Intelligence Alliances and Product Unification, highlighted another sign of growth: The number of gene-therapy developers has ballooned from the 69 counted in 2014 by the Alliance for Regenerative Medicine (ARM), to 255 today.

Below is a list of 25 “up and coming” gene-therapy candidates that had reached Phase III and/or registrational trials as of the first quarter of 2018, based on figures from ARM, as well as ClinicalTrials.gov and/or company announcements. Each candidate is listed by name, sponsor(s), indication(s), mechanism, late-stage trials, and updates.

Two gene-therapy candidates on ARM’s list not included here are TransVax™, whose co-developer Vical eliminated more than half its staff and halted development after it failed the Phase III HELIOS trial. The other is Amgen’s Imlygic® (talimogene laherparepvec), which is in trials for additional melanoma indications (NCT02263508 and NCT02211131) following initial approval in 2015.

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Posted on: 8 May 2018, source: GEN
Gene therapy’s numbers look promising. In a year full of biopharma mergers and acquisitions, one of the biggest deals has been Novartis’ planned $8.7 billion purchase of AveXis, whose lead candidate AVXS-101 has advanced into a pivotal trial following positive Phase I data published in November in The New England Journal of Medicine. Additional figures furnished to GEN by Informa show the extent of gene-therapy development: Informa Pharma Intelligence’s Trialtrove database records 729 gene therapies as having been developed, of which nearly two-thirds (461) were preclinical. Those therapies have been assessed in 1,855 clinical trials, most in early phases: 657 in Phase I, 509 in Phase I/II, and 455 in Phase II. As for later development, 89 have reached Phase III, 32 are in Phase II/III, and 28 in Phase IV.
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