The driving interest in Bellicum revolves around its safety switch for use in hematologic stem cell transplants. It’s product, BPX-501, is a T-cell therapy containing a caspase-9 gene that can.
Loxo Oncology presented highly anticipated results from its ongoing Phase 1 study of TRK inhibitor LOXO-101 yesterday November 8, in a latebreaker at AACR-NCI-EORTC; they did not disappoint. Disclosure of.
The Bull/Bear tussle in Sarepta has been ongoing for years now. Long term investors have ridden a rollercoaster as excitement has inevitably been followed by disappointment. Its Exon 51 skipping.
Global Blood Therapeutics (GBT) is a recent graduate of Third Rock Ventures, a highly regarded firm with a list of innovative portfolio companies including Bluebird Bio, Foundation Medicine, Agios, and.
Raptor Pharmaceuticals is a small Novato, CA. company that markets Procysbi for the treatment of nephropathic cystinosis. It has active clinical trials in Huntington’s Disease and Leigh Syndrome. A recent.
This is a brief analysis of recent entrectinib data presented by Ignyta. Entrectinib is a multi-kinase inhibitor with potent activity against TrkA/B/C, ROS1, and ALK. It was licensed from Nerviano.
Incyte Pharmaceuticals’ lead drug Jakafi (Ruxolitinib) is approved for the treatment of the bone marrow disorders Myelofibrosis and Polycythemia Vera. It is expected to reach blockbuster sales despite these relatively.
Loxo Oncology (LOXO) is a biotech company built around the idea that cancer is best treated with highly targeted drugs paired to companion diagnostics. CEO Josh Bilenker, a partner at.
Trevena Inc. (TRVN) presented positive Phase 2b results Monday August 31 from its TRV130 pain study. The primary endpoint of superior pain reduction compared to placebo was met, as well as.
Esperion shares have fallen over 8% today as The Medicines Co and Alnylam released early data on their RNAi candidate, ALN-PCSsc, targeting PCSK9. The drug was shown to efficiently and.
Embargoed Data was released yesterday in an announcement from AAN regarding GW Pharmaceuticals’ Epidiolex study: https://www.aan.com/PressRoom/Home/PressRelease/1364 This paragraph summarizes the Key Takeaway: “For the 137 people who completed the 12-week.
Cynapsus is developing a sublingual film formulation of apomorphine for the treatment of acute “Off” episodes in patients with Parkinson’s Disease resulting from long term use of Levodopa. While Levodopa.
Marinus Pharmaceuticals is developing the allopregnanolone analog Ganaxolone as a treatment for epilepsy and other seizure disorders. Its most advanced program is a multinational P3 trial of patients with refractory.
San Diego’s Pfenex Inc. is riding the market’s enthusiasm for biosimilars, molecules that although not considered identical, can be automatically substituted for biologic drugs. Previously considered immune to generic competition,.
The year is nearly over, though it feels like scientific conferences never end. December 6th marks the last big Cancer meeting of the year- then we turn the page to.
TG Therapeutics has two programs, the CD20 targeting antibody TG-1101, and PI3K delta inhibitor TGR-1202. The focus of this post is on TGR-1202. Earlier coverage can be found here.
GW Pharma’s Tuesday morning R&D meeting looked pretty good. Efficacy data were released for 58 patients with full 12-weeks of epidiolex treatment- 40 of whom have been treated for 16-weeks..
The only new data provided by Sarepta were various measures of lung function, including, including %predicted MIP, MEP, and FVC; all of which demonstrated stabilization through 144 weeks for the.
Nice 36-month Natural history study (or “Pane Study”) in Duchenne published essentially confirming what’s pretty much already known- that boys age 7 and over decline more rapidly than those under.