Amicus Therapeutics will address clinical and commercial supply needs for its intrathecal adeno-associated virus (AAV) Batten disease gene therapy programs by partnering with Thermo Fisher Scientific’s recently-acquired Brammer Bio, the companies said Monday.

The companies’ strategic manufacturing collaboration—whose value was not disclosed—comes over nine months after Amicus acquired worldwide development and commercial rights for lead programs in ceroid lipofuscinosis, neuronal, subtypes 6, 3, and 8 (CLN6, CLN3, and CLN8) Batten disease among 10 gene therapy programs developed at The Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital and The Ohio State University.

Amicus acquired the 10 programs—including the potentially first-to-market curative treatments for the subtypes of Batten disease—when it bought Nationwide Children’s Hospital spinout Celenex for up to $452 million, a deal that expanded the rare disease drug developer into gene therapy. The 10 also included programs to develop gene therapies for Niemann-Pick disease type C (NPC), Wolman disease, Tay Sachs disease, and lysosomal storage disorders for other unspecified CNS disorders.

“These ten programs have the potential to cure thousands of children worldwide and to create a remarkably valuable and important piece of our business—one with the potential to generate up to $1 billion annually,” Amicus stated in its 2018 Annual Report.

Under the new agreement with Thermo Fisher, Amicus will transfer research and development production technologies and capabilities related to Amicus’ preclinical and clinical-stage gene therapy programs for CLN6, CLN3, and other potential Batten disease programs, for development at Thermo Fisher’s viral vector services business.

Amicus said it also plans to transfer to Thermo Fisher the existing process for the CLN6 Batten disease gene therapy, for development of a similar GMP process for clinical and commercial-scale manufacturing and supply.

In addition, the companies have agreed to partner on developing platform manufacturing capabilities to support Amicus’ broader portfolio of AAV gene therapy programs.

“Significant next step”

“As we advance one of the industry’s leading gene therapy pipelines, our partnership with Brammer Bio, now part of Thermo Fisher, is a significant next step in fulfilling our manufacturing strategy so that we can deliver novel gene therapies to more people living with rare genetic diseases as quickly as possible, especially in devastating diseases like Batten’s, where time is of the essence,” Amicus chairman and CEO John F. Crowley said in a statement.

Added Chris Murphy, vice president and general manager, viral vector services at Thermo Fisher Scientific: “We see this as a strong strategic relationship to enable manufacturing of a deep gene therapy pipeline across various stages of development for people with devastating genetic diseases. These therapies have the potential to transform patients’ lives and, as an organization, we are delighted to support partners such as Amicus as they advance tomorrow’s potential medicines.”

Amicus’ agreement comes two months after Thermo Fisher completed its $1.7 billion acquisition of Brammer Bio, a viral vector contract development and manufacturing organization (CDMO) headquartered in Cambridge, with primary locations in Massachusetts and Florida.

Last month during the 2019 Biotechnology Innovation Organization (BIO) International Convention, held in Philadelphia, Murphy and Paul Jorjorian, head of bioprocess sciences in Thermo Fisher’s Pharma Services Group, discussed Brammer’s $100 million in expansion plans, designed in part to enhance its gene therapy manufacturing capabilities.

Amicus’ CLN6 gene therapy candidate is being evaluated in a Phase I/II trial (NCT02725580) that has completed target enrollment, with 12 patients receiving a single administration of AAV serotype 9 (AAV9)-CLN6 gene therapy. Amicus expects to report additional two-year data from the study later this year, the company stated in its Form 10-Q quarterly report for the first quarter of this year, filed May 9.

The CLN3 gene therapy candidate is the subject of another Phase I/II trial (NCT03770572), in which three patients had been dosed in the low-dose group, “with no serious adverse events after up to 5 months following a single administration of AAV9-CLN3 gene therapy,” Amicus said in the Form 10-Q,

Amicus’ gene therapy pipeline includes two additional Nationwide/Ohio State-originated Batten disease gene therapy candidates, both in preclinical phases: One for CLN8 Batten disease, and one for CLN1 Batten.

“We look forward to embedding our Amicus team and other strategic partners in the tech transfer process to Thermo Fisher, and to further augmenting our manufacturing capabilities and partnerships across our entire gene therapy portfolio,” Crowley added.

Two gene therapy cornerstones

The acquisition of Celenex and Nationwide/Ohio gene therapy programs was one of two cornerstones of Amicus’ gene therapy portfolio. The other is Amicus’ ongoing collaboration with the Perelman School of Medicine at the University of Pennsylvania (Penn).

On May 29, Amicus and Penn Perelman announced a major expansion of the collaboration they launched in October 2018. The partners expanded their rare genetic disease programs from three to six, adding programs in NPC as well as Pompe disease, Fabry disease, CDKL5 deficiency disorder (CDD), Mucopolysaccharidosis Type IIIB (MPS IIIB), as well as a next generation program in Mucopolysaccharidosis Type IIIA (MPS IIIA), both also known as part of Sanfillipo Syndrome.

Through a discovery research agreement, Amicus also obtained exclusive disease-specific access to rights to collaborate with Penn’s Gene Therapy Program (GTP), whose director is pioneer researcher James M, Wilson, MD, PhD, to develop potentially disruptive new gene therapy platform technologies and programs for the majority of lysosomal disorders and 12 additional rare diseases.

The additional rare diseases include Rett Syndrome, Angelman Syndrome, Myotonic Dystrophy and select other muscular dystrophies. Terms of the expansion agreement include a $10 million annual investment from Amicus, each year for five years, into GTP’s discovery research program, with the ability to extend.

Wilson is clinical development editor of Human Gene Therapy, a journal published by GEN publisher Mary Ann Liebert, Inc.

Amicus is also expanding its gene therapy development through the Global Research and Gene Therapy Center of Excellence the company plans to open later this year within Philadelphia’s uCity Square. Up to 200 employees are eventually expected to be based at the new Center, which will occupy 75,000 square feet of office and lab space over three floors at 3675 Market Street.

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