Beam Therapeutics Inc. CEO John Evans told BioWorld that the company's $135 million series B influx – following sooner than some expected the $87 million series A raise last May – is meant to "make sure that resources aren't the limiting factor" in pushing along the company's next-generation CRISPR technologies, expanding the pipeline of base editing programs, and adding to the firm's scientific and technical leadership.
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.
