Amicus Therapeutics said it has acquired Nationwide Children’s Hospital spinout Celenex for up to $452 million, in a deal that expands the rare disease drug developer into gene therapy with a portfolio of 10 clinical and preclinical programs in neurologic lysosomal storage disorders (LSDs).

The 10 adeno-associated virus 9 (AAV9) gene therapy programs—developed at the hospital’s Center for Gene Therapy and The Ohio State University, both based in Columbus, OH—include lead programs in CLN6, CLN3, and CLN8 Batten disease that are potential first-to-market curative treatments, Amicus said.

The other programs include efforts to develop gene therapies for Niemann Pick C, Wolman disease, Tay Sachs disease, and LSDs for other unspecified CNS disorders, according to a presentation for investors by Amicus.

“The groundbreaking work of [principal investigators] Brian Kaspar, Ph.D. and Kathrin Meyer, Ph.D., at Nationwide Children’s Hospital, along with collaborator, Arthur Burghes, Ph.D., professor at The Ohio State University, on these programs has led to remarkably strong and consistent preclinical results and now, in CLN6 Batten disease, encouraging early results in children. This is science and biotechnology at its best,” Amicus Chairman and CEO John F. Crowley said in a statement.

Recruiting of patients has begun for a Phase I/II clinical trial of the CLN6 therapy (NCT02725580), while the first patient is expected to be dosed in a Phase I/II trial of the CLN3 program “in coming months,” Amicus stated in its presentation.

According to Amicus, a single administration of the CLN6 gene therapy generated “encouraging” preliminary efficacy data in the first two patients treated: Two years post-treatment, Hamburg motor and language scores indicated no disease progression in the younger sibling, while disease progression in older sibling showed evidence of stabilization. Additional data for CLN6 will be presented in 2019.

The CLN6, CLN3, and CLN8 programs gave all generated promising preclinical data in mouse models: “The preclinical proof-of-concept we have seen to date in CLN6, CLN3, and CLN8 further support the applicability of the AAV vector we developed at Nationwide Children’s in genetic disease of the CNS,” Dr. Meyer adds.


Billion-Dollar Vision

Amicus said the deal would more than achieve its vision of growing into a rare disease developer whose therapies treat more than 5,000 patients, and generate more than $1 billion in worldwide sales by 2023. Amicus generated $38.005 million in the first half of this year and $36.93 million for all of 2017 in net product sales, all from its sole marketed drug, the Fabry disease treatment Galafold™ (migalastat), authorized in Europe since 2016 and only approved in the U.S. last month.

Of the Celenex programs, Amicus said in the presentation, the largest estimated addressable patient population is the 5,000 projected for the CLN3 treatment, followed by the 3,500 for the Niemann Pick C, Wolman disease, Tay Sachs disease, and other CNS LSD programs. Another 1,000 patients are estimated for the CLN6 treatment, and 750 for the CLN8 therapy.

Amicus has agreed to pay Celenex shareholders $100 million cash upfront, $15 million in payments tied to achieving development milestones, and $262 million in regulatory submission and approval milestones across multiple programs. Amicus said it expected to shell out no more than $75 million over the next four years in milestone payments, and would not pay out any royalties.

Celenex shareholders are also eligible for up to $75 million in payments tied to tiered sales milestones (tiers of $500 million/$750 million). The acquisition and several years of related development costs for all of the gene therapy programs will be financed through a new $150 million debt facility to be provided by BioPharma Credit, an investment fund managed by Pharmakon Advisors. 

The transaction was closed immediately following approval by the boards of Amicus and Celenex.

Columbus-based Celenex was established by Gordon Gray, producer of movies that include Secretariat, and his wife, Kristen Gray, whose daughters Charlotte and Gwenyth were both diagnosed with Batten’s disease in 2015, at ages 4 and 2, respectively. The Grays established the Charlotte and Gwenyth Gray Foundation, created to fund research and clinical studies toward Batten disease treatments.

The Celenex acquisition comes after Amicus on August 10 won FDA accelerated approval for Galafold 123 mg capsules, an oral pharmacological chaperone of alpha-Galactosidase A (alpha-Gal A) taken every other day. Galafold was approved based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate. As a condition of accelerated approval, Amicus agreed to continue studying Galafold in a confirmatory Phase IV program.

Amicus is also conducting a global Phase I/II study (NCT02675465) for another clinical candidate, AT-GAA for Pompe disease. AT-GAA consists of ATB200, a recombinant human acid alpha-glucosidase enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate, co-administered with AT2221, a pharmacological chaperone. Clinical results are expected to be shared at the 23rd International Congress of the World Muscle Society, set for October 2-6 in Mendoza, Argentina.







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