SAN DIEGO – Smaller companies looking to move their Alzheimer’s disease drugs into late-stage testing as quickly as possible are eschewing cognitive endpoints that can take years to readout for biomarkers and functional assays of brain activity.

ORLANDO, Fla. – Not long past the opening of the American Society of Hematology’s (ASH) massive annual conference, Janssen Biotech Inc. released upbeat follow-up survivability data on Imbruvica (ibrutinib) from two studies and an integrated analysis evaluating Imbruvica in previously untreated patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).

ORLANDO, Fla. – New phase I/II data from Autolus Therapeutics plc announced at the American Society of Hematology’s (ASH) annual conference show that AUTO-3, the first bicistronic CAR T targeting CD19 and CD22 followed by an anti-PD1, was well-tolerated in a phase I/II study.

ORLANDO, Fla. – Kura Oncology Inc., encouraged by new data on tipifarnib and positive feedback from the FDA, said Sunday that next year it will initiate a single-arm, phase II registration-directed trial of the candidate in angioimmunoblastic T-cell lymphoma (AITL), a rare and often aggressive form of peripheral T-cell lymphoma (PTCL).  

ORLANDO, Fla. - Chimeric antigen receptor T (CAR T) cells have proved to be a major advance in treating patients with refractory B-cell malignancies so far. But, often, in approaching those issues "we solve one and we create another," Stephen Schuster, a doctor at the Abramson Cancer Center at the University of Pennsylvania in Philadelphia, told reporters at the 61st American Society of Hematology (ASH) annual meeting Saturday.

The “remarkably appealing” route of administration and every-three-month dosing put Ferring Pharmaceuticals SA’s nadofaragene firadenovec (rAd-IFN/Syn3, also known as Instiladrin) in strong position for approval in high-grade Bacillus Calmette-Guérin (BCG) unresponsive non-muscle invasive bladder cancer (NMIBC).  

News of a partial FDA clinical hold for pediatric patients after safety reports from an ongoing trial testing palovarotene in fibrodysplasia ossificans progressiva (FOP) identified cases of early growth plate closure sent shares of Ipsen SA (Paris:IPN) falling 13.6% Friday.

Despite some encouraging top-line data from Sage Therapeutics Inc.’s phase III Mountain study of SAGE-217 on depressive symptoms in adults with major depressive disorder (MDD), the company, the market and analysts were taken aback at the trial’s failure to hit its primary endpoint.