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.
Privately held Cerevance Inc., of Boston, raised $45 million in a series B designed to propel the discovery and development of therapies for treating CNS diseases such as Alzheimer’s disease.
It has been more than 200 years since British doctor James Parkinson first identified the symptoms of a condition that he termed shaking palsy; unfortunately, there is still no cure to the disease that carries his name.
Sage Therapeutics Inc.’s chief business officer, Michael Cloonan, said the firm is “not going to give details around the geographies and the number of sites” that will continue to use Zulresso (brexanolone) for postpartum depression (PPD) after the restructuring of the Cambridge, Mass.-based firm.
With positive top-line data reported for Axsome Therapeutics Inc.’s oral migraine candidate, AXS-07, in its INTERCEPT trial, the company and the candidate have notched two successful phase III trials in little more than three months.
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