Gene therapy specialist Uniqure N.V. said Tuesday that, with confidence inspired by new 52-week data on its investigational hemophilia B therapy, etranacogene dezaparvovec, it plans to submit a BLA for the program with partner CSL Behring LLC in first quarter of 2022. Uniqure meanwhile has moved to acquire Corlieve Therapeutics SAS and its lead program to treat temporal lobe epilepsy, the most common form of focal epilepsy. The acquisition, worth up to €250 million (US$297.3 million) for Corlieve, includes €46.3 million cash up front.
As debate continues to rage over the approval and pricing of the Alzheimer’s disease therapy Aduhelm (aducanumab), Biogen Inc. CEO Michel Vounatsos said his firm is “taking a position that is very responsible” but has “yet to be understood out there, and we have to do a better job.”
LONDON – Newco Amyl Therapeutics Ltd. has raised €18.3 million (US$22.3 million) in a series A, to pick up the baton on a technology for neutralizing toxic protein aggregates by binding to their amyloid folds.
Biogen Inc. has announced contrasting results from phase III trials of therapies for a rare eye disease and depression, following last week’s controversial FDA approval of Alzheimer’s drug Aduhelm. The Cambridge, Mass.-based firm said a phase III gene therapy study in the rare retinal disease choroideremia missed its primary and secondary endpoints, although the news was better from a potential therapy for major depressive disorder.
LONDON – The pioneers of gene therapy in Europe now aim to take the field to the next level, moving into the development of virally delivered antibodies in the treatment of central nervous system and muscular diseases, with Alzheimer’s disease one of the first targets.
Biogen Inc.’s pricing of its newly approved Alzheimer’s drug, Aduhelm (aducanumab), has made it the latest bull’s eye for lawmakers and advocacy groups targeting U.S. drug prices, especially given the controversy surrounding the drug’s approval, which has resulted in the resignation of three of the 11 members of the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee.
Parkinson’s disease (PD) is one of the most common neurodegenerative diseases, and the role of ?-synuclein accumulation and the subsequent death of dopaminergic neurons in the midbrain have long been recognized as key steps in the disease. Progress in understanding genetic risk factors, meanwhile, has uncovered multiple genetic risk factors. Even though aging is the single biggest risk factor for PD, there are versions of the disorder that affect children.
DUBLIN – Aelis Farma SAS is banking $30 million up front as part of a strategic collaboration and option license deal with Indivior plc involving AEF-0117, its candidate drug for treating cannabis-related disorders, including addiction and psychosis. It could receive another $100 million, should Invidior trigger the option on completion of a phase IIb trial in the U.S.
During a conference call with investors, Biogen Inc. officials defended the price of Aduhelm (aducanumab) for Alzheimer’s disease (AD), with CEO Michel Vounatsos saying the $56,000 per year price tag was based on “the value it is expected to bring to patients, caregivers, and society.”
By granting accelerated approval for Biogen Inc.’s Aduhelm (aducanumab) for Alzheimer’s disease (AD), the FDA is “essentially confirming that the beta-amyloid hypothesis has been validated,” Mizuho analyst Salim Syed said in an alert to investors. Shares of Cambridge, Mass.-based Biogen (NASDAQ:BIIB) closed $395.85, up $109.71, or 38.3%, as Wall Street hailed the first new AD therapy to reach the market since 2003.
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