Phase I

Hutchison China MediTech Limited has initiated a Phase I study of HMPL-523, its novel spleen tyrosine kinase (“Syk”) inhibitor, in combination with azacitidine, an approved nucleoside metabolic inhibitor, in elderly patients with acute myeloid leukemia (“AML”) in China.
Bioavailability significantly higher than with oral administration
Proteostasis Therapeutics announced positive preliminary data from its ongoing Phase I clinical trial of PTI-808 and PTI-801 in cystic fibrosis (CF). Despite it being an early-stage trial and preliminary data, company shares exploded upward by 301 percent, with more than 44 million shares trading hands.
KB103 is Krystal’s first “off-the-shelf”, topical, gene therapy candidate to treat patients suffering from Dystrophic Epidermolysis Bullosa.
MaxCyte announced today that the first patient has been dosed in its Phase I dose-escalation clinical trial in the United States with the Company’s lead wholly-owned chimeric antigen receptor (CAR) therapeutic candidate, MCY-M11.
There are plenty of great scientific research stories out this week. Here’s a look at just a few of them.
Ionis Pharmaceuticals is on a roll. Days after securing regulatory approval from the FDA for Tegsedi, the company inked a deal worth more than $700 million with pharma giant Roche to develop IONIS-FB-L for the treatment of complement-mediated diseases.
In May the FDA placed a hold on the trial and IND for CTX001, an investigational gene editing treatment, citing concerns over questions that had not been addressed in the IND.
Affimed, based in Heidelberg, Germany, placed a clinical hold on its two AFM11 Phase I clinical trials. AFM11 is being evaluated in the trials for relapsed or refractory CD19 positive B-cell non-Hodgkin lymphoma and acute lymphoblastic leukemia.
Data Supports Akari’s Ongoing Clinical Expansion into a Range of Orphan Autoinflammatory Diseases That Are Either Mediated by Complement C5 or Have Synchronous C5 and Leukotriene B4 (LTB4) Activity
PRESS RELEASES