Taysha Gene Therapies Inc., a new Dallas-based company reuniting former executives of Avexis Inc. and its funders, has launched with a $30 million seed financing intended to advance a pipeline of 15 new AAV-based candidates. Its team expects to file four INDs by the end of 2021, starting with one for GM2-gangliosidosis that could move to the clinic later this year.
A once-daily add-on therapy for Parkinson's disease (PD) used in Europe for years has now gained clearance in the U.S. with FDA approval of Ongentys (opicapone). The drug, an improvement upon generics in its class, will be sold by Neurocrine Biosciences Inc. The medicine, a catechol-O-methyltransferase (COMT) inhibitor first approved by the EMA in 2016, extends the half-life of levodopa, increasing doses of which are required to achieve motor control as PD progresses. Portugal-based Bial-Portela & Ca SA, from which Neurocrine licensed North American rights to the drug, will receive a $20 million award from its partner. Shares of San Diego-based Neurocrine (NASDAQ:NBIX), a neuroscience company focused on treating movement disorders, rose 3.5% following the approval, closing at $101.67 on April 27.
Privately held Swanbio Therapeutics Inc., of Bala Cynwyd, Pa., completed a $52 million expanded series A financing designed to boost the company’s R&D of adeno-associated virus (AAV)-based gene therapies to treat adrenomyeloneuropathy (AMN) and other rare, monogenic neurological diseases.
The acceptance by the FDA of Prevail Therapeutics Inc.’s IND for the one-time, fast-tracked gene therapy PR-006 provided hope for 50,000 to 60,000 people in the U.S beset by frontotemporal dementia with the GRN mutation (FTD-GRN), and the New York-based company is moving ahead with a phase I/II experiment called Proclaim.
Supernus Pharmaceuticals Inc. has struck an agreement with privately held Navitor Pharmaceuticals Inc. to run a joint phase II program for Navitor's mTORC1 activator, NV-5138, in treatment-resistant depression (TRD).
Biogen Inc. handily beat earnings expectations in its first-quarter 2020 earnings report to investors but leavened the good news by adding that it now plans to submit its BLA for beta-amyloid-targeting aducanumab for treating Alzheimer’s disease in the third quarter of 2020.
Although the product pipeline for vaccines and therapeutics targeting COVID-19 is top of mind right now, investors are also keeping a close eye on companies involved in the development of medicines targeting cancer and the central nervous system. According to financings tracked by BioWorld and deals and grants logged in Cortellis, the therapeutic areas of cancer, neurology and psychiatric attracted the highest amounts of investments last year with a collective $101.9 billion and $27.5 billion raised, respectively.
Positive top-line results from the first of two pivotal phase III studies of daridorexant, a dual orexin receptor antagonist (DORA) for treating insomnia from Idorsia Ltd., of Allschwil, Switzerland, showed significantly improved sleep onset, sleep maintenance and improved subjective total sleep time in 930 adult and elderly patients.
Chinese scientists have shown for the first time that the down-regulation of a single RNA-binding protein, polypyrimidine tract-binding protein 1 (Ptbp1), locally converted glial cells to neurons and showed promise for treating the symptoms of neurodegenerative diseases in mice.