Verrica Pharmaceuticals Inc.'s positive phase III data from its pivotal Cantharidin Application in Molluscum Patients-1 (CAMP-1) and CAMP-2 trials with lead candidate VP-102 at the American Academy of Dermatology meeting in Washington, followed encouraging top-line results made known in early January and put the firm in position to submit an NDA in the second half of this year.
CEO Jan Møller Mikkelsen said Ascendis Pharma A/S is "still analyzing a lot of the data" from its phase III trial with its Transcon human growth hormone (hGH), a weekly therapy that beat daily growth hormone in the experiment called Height, but researchers are able to affirm that the better-than-expected outcome was "driven by the molecule itself, not by a difference in adherence between the two arms" of the study.
Last month, Hayward, Calif.-based Aradigm Corp. filed for protection under Chapter 11 of the U.S. Bankruptcy Code in the Alameda County Court District to sell its assets, and about a week later the company made known the bad news in notes from the type B meeting held with the FDA.
The dataset that emerged from Dublin-based Horizon Pharma plc's phase III trial with teprotumumab for active thyroid eye disease (TED) is "not just OK – it's amazing," said Raymond Douglas, co-principal investigator in the phase II and phase III programs with the compound.
Sarepta Therapeutics Inc. CEO Doug Ingram told Wall Street that the firm "has couple of things we've got to get done very soon and report back to you on," given early results in the first three-patient cohort of the MYO-101 study in patients with limb-girdle muscular dystrophy (LGMD) known as LGMD2E – strongly positive results that triggered an early exercise of the option to acquire Myonexus Therapeutics Inc. for $165 million.
Douglas Kahn, CEO of Tetragenetics Inc., told BioWorld that his firm "in pretty short order went from introducing the technology to discussing the terms of a potential relationship" for the purpose of developing non-opioid pain drugs with Imbrium Therapeutics LP, a subsidiary of Purdue Pharma LP, the embattled maker of Oxycontin (oxycodone).
Spark Therapeutics Inc. told BioWorld there was "not much more we can say beyond the press release" and the company wasn't offering executive interviews, but the buyout by Roche Holding AG generated vigorous buzz on Wall Street as pundits tried to guess which gene therapy firm might be next for a takeover in the space.
During 2017 and 2018, the FDA put life aplenty into the AML space by taking action to clear or reapprove no fewer than eight drugs for acute myeloid leukemia (AML), including Agios Pharmaceuticals Inc.'s Tibsovo (ivosidenib) to treat relapsed or refractory disease with an isocitrate dehydrogenase 1 (IDH1) mutation who are not eligible for standard therapy and Idhifa (enasidenib), cleared for patients with IDH2 mutations. And the indication only looks to grow hotter.
When a woman in Cork, Ireland, cutting off her own right index finger to relieve her pain made headlines recently, the mainstream media brought to popular knowledge the condition with which she had been diagnosed: chronic regional pain syndrome (CRPS), a devilish affliction being pursued by the likes of New York-based Axsome Therapeutics Inc. and the Grunenthal GmbH, of Aachen, Germany.
After excitement on Wall Street early in the day about pivotal phase III data with obeticholic acid (OCA) in patients with liver fibrosis due to nonalcoholic steatohepatitis (NASH), shares of New York-based Intercept Pharmaceuticals Inc. (NASDAQ:ICPT) settled to close at $117.57, up $6.75.
