With a just-begun phase III trial, Malvern, Pa.-based Galera Therapeutics Inc. is testing its small-molecule mimicker of superoxide dismutase, avasopasem manganese (GC-4419), against severe oral mucositis (SOM) in patients with head and neck cancer.
Matinas Biopharma Holdings Inc. CEO Jerome Jabbour told BioWorld that, with the company's omega-3 fatty acid-based MAT-9001 for high triglycerides, "we've come full circle," thanks to cardiovascular (CV) outcomes data disclosed last month by Amarin Corp. plc with omega-3 drug Vascepa (icosapent ethyl). "Now we find ourselves sitting on a potential best-in-class asset," which beat Dublin-based Amarin's compound in a head-to-head trial several years ago.
Sarepta Therapeutics Inc.'s potential licensing deal with Paris-based Lysogene SA drew more attention to the already hot space of gene therapy for lysosomal storage disorders (LSDs), the news coming just a week after Amicus Therapeutics Inc. – eager to guard its franchises in Pompe and Fabry disease – signed a pact with Perelman School of Medicine at the University of Pennsylvania (Penn) to pursue gene therapy treatments for those two conditions as well as CDKL5 deficiency and an additional rare metabolic disorder that was not disclosed.
"What we've all been waiting for" is how H.C. Wainwright analyst Joseph Pantginis characterized details about the successful end-of-phase-II meeting with the FDA disclosed by Iovance Biotherapeutics Inc., news that helped the San Carlos, Calif.-based company raise $219.3 million through a stock sale.
In June 2015, when officials of Matinas Biopharma Holdings Inc. saw the results of the 42-patient trial testing its omega-3 fatty acid-based MAT-9001 for high triglycerides against the approved Vascepa (icosapent ethyl) from Dublin-based Amarin Corp. plc, they knew they had something. But, at the time, "there was not a lot of wind in the sails" of cardiovascular (CV) research, Matinas CEO Jerome Jabbour told BioWorld. "Everyone knew it would be another three years before Amarin was ready to talk about its outcomes study."
As if to highlight the need for speed in the midstage race in alopecia areata (AA, or hair loss in patches), Wayne, Pa.-based Aclaris Therapeutics Inc. in July gained fast track status from the FDA for the phase II topical JAK1/3 inhibitor ATI-502.
Saying "now is the time to be a shareholder" in the company, Ziopharm Oncology Inc. CEO Laurence Cooper outlined changes to the company's relationship with Precigen Inc., a wholly owned subsidiary of Intrexon Corp., that gives his Boston-based firm "full autonomy" to execute on its research efforts.
Now that Ionis Pharmaceuticals Inc. and affiliate Akcea Therapeutics Inc. have gained FDA clearance – albeit delayed and with a black box warning – of antisense oligonucleotide Tegsedi (inotersen) for polyneuropathy of hereditary transthyretin (ATTR)-mediated amyloidosis in adults, speculation has begun about the drug's chances in the marketplace given the double-stranded small interfering RNA drug Onpattro (patisiran) from Alnylam Pharmaceuticals Inc., which won the FDA's go-ahead in August for the same indication.
San Diego-based Viking Therapeutics Inc. enjoyed an 87 percent stock hike in September when the company disclosed that, relative to placebo, its thyroid receptor beta agonist VK-2809 reduced low-density lipoprotein cholesterol and lowered liver fat in people with elevated LDL-C and nonalcoholic fatty liver disease (NAFLD). The top-line phase II data suggest the drug may be effective in battling the progressive form of NAFLD, nonalcoholic steatohepatitis (NASH).
Sarepta Therapeutics Inc.'s new data in Duchenne muscular dystrophy (DMD) ought to have provided relief for investors worried about gene therapy competition from Pfizer Inc., but the stock hardly moved, possibly because Wall Street did not give enough weight to the first functional outcomes data from the North Star Ambulatory Assessment (NSAA) composite endpoint or put too much on the possible toxicity of super-high microdystrophin expression levels in the fourth patient tested.
