The first potential gene therapy for hemophilia B has moved one step closer to patients as the FDA has accepted CSL Behring’s BLA for its unique treatment in partnership with uniQure. The therapy, called etranacogen dezaparvovec, has been accepted under “accelerated review” by the European Medicines Agency in March.
A priority review will be done for gene therapy for patients with the inherited bleeding disorder, CSL said Tuesday morning. The Pennsylvania biotech did not provide a date for the regulator’s decision, but priority review usually means the treatment will be decided within six months instead of 10.
Regulators will base their decision on a Phase III study that found the treatment is not inferior to baseline factor IX prophylactic therapy on annualized bleeding rate (ABR) after 18 months. The companies said the treatment was also statistically superior in reducing ABR after 18 months in the HOPE-B study.
This is good news for patients and companies after a five-month suspension last year due to a safety issue that revealed a patient in the study had developed liver cancer. UniQure concluded that its therapy was “highly unlikely” to have led to the cancer.
SVB Securities analysts said they expect a decision from the EMA in the third quarter and from the FDA in the following quarter. Analysts said the gene therapy has “compelling endorsement potential that could rekindle interest in the broader gene therapy space,” noting that another key decision will be in hemophilia gene therapy. A from BioMarin, which is under review in Europe and provided for a new submission in June in the United States.
Meanwhile, Cowen analysts noted that in their survey of estimated use of hemophilia B gene therapy, doctors said 13% of patients would use such treatment three years after launch, while payers expected 31% to do so.
Analysts have estimated peak worldwide sales at $1.2 billion, with uniQure potentially earning $485 million in peak royalties after selling exclusive worldwide rights to CSL in a $2 billion pact. — Kyle LaHucik
Fosun Pharma Reaches Agreement with VerImmune for License and Options Agreement
Fosun Pharma is looking to continue making deals as they reach a new cancer deal.
Fosun Pharma USA, the U.S. subsidiary of Shanghai-based Fosun Pharmaceutical, has signed an agreement with Washington, DC-based biotech VerImmune Inc for an exclusive license and option agreement effective May 18, 2022.
VerImmune is developing a new Virus-Inspired Particle (ViP) therapeutic modality that redirects the body’s pre-existing immunity against viral pathogens to attack cancer.
Under the terms of the agreement, VerImmune has granted Fosun Pharma an exclusive license to VerImmune’s lead anti-tumor immune redirection, or AIR, candidate, called VERI-101, with an option to license certain potential future AIR-VIP products. from the development of VerImmune’s VERI-101 pipeline. This will include licenses for mainland China, Hong Kong, Macao and Taiwan. Fosun Pharma will be responsible for all clinical development and commercialization in these territories.
VerImmune is eligible to receive up to $125 million in upfront and milestone payments, plus tiered royalties up to double digits on net sales of VERI-101 in specified territories. Subject to Fosun Pharma exercising its option to select future AIR-VIP product candidates, VerImmune is eligible to receive additional milestone payments and net sales royalties for each additional product candidate.
“Cancer is a global unmet medical need and so we are very pleased to work with Fosun Pharma companies to bring our new AIR-ViP immunotherapeutic products to China. Fosun Pharma has both the expertise and the access to reach these fast-growing markets and we hope to benefit more Chinese patients and the region through our cooperation,” said Joshua Wang, Founder and CEO of VerImmune.
This is not the only deal Fosun has signed this year as they reached an agreement with Insilico, which received $13 million upfront from Fosun for a collaborative R&D project, which includes co-development of Insilico’s QPCTL program. — Tyler Patchen