"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.
The release of abstracts related to the upcoming Society for Immunotherapy in Cancer (SITC) meeting in Washington whetted investor appetite in many precincts of oncology, and an area of particular focus relates to a pair of sizzling therapeutic modalities: CRISPR/Cas9 and chimeric antigen receptor (CAR) T-cell therapies.
Dragonfly Therapeutics Inc. CEO Bill Haney told BioWorld that his firm's deal with Merck & Co. Inc. provides "a pretty considerable up-front payment" and gives Merck the option to license exclusive worldwide intellectual property rights to products developed using the TriNKET natural killer (NK) cell technology platform against solid tumors for an undisclosed number of programs.
Hopes were high for tipifarnib a few years ago, when Kura Oncology Inc., of La Jolla, Calif., completed a reverse merger and closed a $60 million private placement with a syndicate of top venture funds around the same time it executed an agreement with Johnson & Johnson (J&J) unit Janssen Pharmaceutica NV, of Beerse, Belgium, for an exclusive license to develop and commercialize the oral farnesyltransferase inhibitor in multiple oncology indications.
