PTC Therapeutics Gets Funding from Blackstone for Drug Development

03.11.2022 - US biopharma PTC Therapeutics has entered into a financing collaboration with private equity group Blackstone so that it can achieve its goal of delivering at least one therapy every two-to-three years.

Funds managed by Blackstone Life Sciences and Blackstone Credit will initially provide $500 million. Another $500 million could be provided as a potential credit facility or other investment capital to support business development opportunities, subject to mutual agreement between Blackstone and PTC.

"This strategic financing will support the acceleration of PTC's robust and diversified pipeline, business development opportunities and general corporate purposes," said Emily Hill, PTC’s chief financial officer. "Our strong internal drug development and commercialization efforts, record of business development and value creation, combined with Blackstone's network and life sciences expertise, puts PTC in a strong position to continue to execute our mission."

In July, PTC announced that it had received marketing authorization from the European Commission for Upstaza, a gene-therapy for treating aromatic L-amino acid decarboxylase (AADC) deficiency. PTC said Upstaza is the first approved disease-modifying treatment for AADC deficiency and the first marketed gene therapy directly infused into the brain.

AADC deficiency is a fatal and rare genetic disorder that typically causes disability and suffering from the first months of life. Upstaza is indicated for patients aged 18 months and older.

The New Jersey-based company is also conducting several clinical trials for indications such as relapsed/refractory acute leukemias, diffuse intrinsic pontine glioma and high-grade glioma, leiomyosarcoma, ovarian cancer, COVID-19 and mitochondrial epilepsy.

The company’s existing portfolio includes Emflaza and Translarna for treating Duchenne muscular dystrophy, Evrysdi for treating spinal muscular atrophy and Tegsedi for the treatment of stage 1 or 2 polyneuropathy in adults with hereditary transthyretin amyloidosis.

PTC also has commercialization rights in Latin America for Waylivra, which is approved in the US for treating familial chylomicronemia syndrome. Ionis Pharmaceuticals is commercializing Waylivra outside of Latin America.

Author: Elaine Burridge, Freelance Journalist