Karuna Therapeutics Inc. remains on track to launch a phase III study by the end of this year with Karxt against acute psychosis in schizophrenia by way of the broad-based program called Emergent, and the company continues planning for a phase II study testing the combo drug as adjunctive therapy with standard of care in the same indication.
The FDA’s go-ahead for Roche Holding AG’s Enspryng (satralizumab-mwge) in anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder had watchers of the space weighing its market odds against two therapies approved earlier: Soliris (eculizumab) from Boston-based Alexion Pharmaceuticals Inc., and the more recently cleared Uplizna (inebilizumab-cdon) from Viela Bio Inc., of Gaithersburg, Md.
Triggering receptor expressed on myeloid cells 2 (TREM2) was first discovered because variants affect the risk of developing late-onset Alzheimer’s disease (AD).
Knocked back by a complete response letter (CRL) in late 2018, Trevena Inc. dusted itself off, resubmitted its NDA for oliceridine and found satisfaction as the FDA has approved the opioid agonist.
Investigators have developed a new approach to classifying neurodegenerative disorders that used the overall patterns of protein aggregation, rather than specific proteins, to define six clusters of patients that crossed traditional diagnostic categories.
More than two weeks ahead of its expected PDUFA date, PTC Therapeutics Inc.’s spinal muscular atrophy (SM) drug, risdiplam, gained FDA approval, making it the first at-home, oral treatment intended for use in adults and children 2 months and older.
News from Biogen Inc. and partner Eisai Co. Ltd. that U.S. regulators accepted the BLA related to aducanumab for Alzheimer’s disease (AD) – and assigned it priority review, no less – set Wall Street abuzz, especially as the companies noted in their press release that the FDA “if possible, plans to act early” on the anti-amyloid beta (a-beta) monoclonal antibody. Regulators’ decision came about 30 days after they took receipt of the submission; they could have waited 60.
Biogen Inc., one the world's top neurological disease specialists, is committing to invest more than $1 billion in cash and equity in Denali Therapeutics Inc. for rights to co-develop and co-commercialize a potential therapy for Parkinson's disease (PD) and options to secure up to four more neurodegenerative disease programs from its new partner.
Bold up-fronts and even bigger milestones defined ambitious neurology deals Abbvie Inc. struck with Voyager Therapeutics Inc. in 2018 and 2019. With vectorized antibodies, they planned to target multiple indications tied to excess aggregations of tau and tragic synucleinopathies. Considerable progress was made, said Omar Khwaja, Voyager's chief medical officer. But despite millions of dollars invested in the programs, Abbvie has now decided to quit the venture, leaving Voyager to either go it alone or find a new partner in its work on the challenging indications.
Sangamo Therapeutics Inc. continued its collaboration spree, signing up its sixth big pharma/biotech partner: Novartis AG. The three-target deal will use Sangamo's zinc finger protein transcription factors to up-regulate undisclosed genes to treat autism spectrum disorder and other neurodevelopmental disorders.
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