During its recent fourth-quarter and full-year earnings report, Foster City, Calif.-based Mirum Pharmaceuticals Inc. provided an update on the apical sodium dependent bile acid transporter (ASBT) inhibitor maralixibat for Alagille syndrome (ALGS), which will be the subject of a rolling NDA starting in the third quarter of 2020.
The allure of gene therapy was proved yet again as Waltham, Mass.-based Affinia Therapeutics Inc. bagged an oversubscribed $60 million series A financing to boost the push for drugs to benefit people affected by muscle and central nervous system conditions.
Akero Therapeutics Inc. rolled out stellar non-COVID-19 (for a change) clinical news with long-acting fibroblast growth factor 21 (FGF21) analogue AKR-001 in biopsy-confirmed nonalcoholic steatohepatitis (NASH), but even in liver disease the pandemic became part of the discussion.
Cambridge, Mass.-based Elevatebio LLC has triple-pronged plans for its $170 million series B round, which the company said will be directed toward manufacturing cell and gene therapies, enabling new technology platforms, and pursuing therapeutics.
During the conference call on earnings in March, CEO Dror Harats told investors that “the most important thing” about VBL Therapeutics Ltd.’s then-upcoming analysis of interim phase III data with gene therapy VB-111 (ofranergene obadenovec) is that it was “designed in a way that will enable us to tell the market if we are at least as good as what we've seen” in the phase II experiment.
Castle Creek Biosciences Inc. CEO John Maslowski told BioWorld the firm’s $75 million financing will propel phase III research with FCX-007 in recessive dystrophic epidermolysis bullosa (RDEB), with top-line data due in the first quarter of 2021 – “a big inflection point” for the Exton, Pa.-based company, which plans a BLA filing for that year as well.
Cytomx Therapeutics Inc. CEO Sean McCarthy told BioWorld that the longish hiatus between deals broken by the tie-up with Astellas Pharma Inc. was “very deliberate. Over that period of time, we were generating our first clinical data to get experience with how the technology works in cancer patients with our first two lead programs,” both of which have reached the phase II stage. A year ago in January, he said, the South San Francisco-based firm brought aboard Chief Business Officer Nick Galli to “hit the gas pedal a bit more in business development,” which led to the signing with Astellas, of Tokyo. Shares of Cytomx (NASDAQ:CTMX) closed March 24 at $6.53, up $1.60, or 32%.
Cytomx Therapeutics Inc. CEO Sean McCarthy told BioWorld that the longish hiatus between deals broken by the tie-up with Astellas Pharma Inc. was “very deliberate. Over that period of time, we were generating our first clinical data to get experience with how the technology works in cancer patients with our first two lead programs,” both of which have reached the phase II stage.
Stoke Therapeutics Inc. is marching ahead in the second half of this year with its phase I/IIa study with STK-001 in Dravet syndrome (DS), one of the more abysmal forms of epilepsy, although the FDA has temporarily hobbled part B of the test, pending preclinical data that will more fully characterize the safety profile of the antisense oligonucleotide (ASO).
