Aclaris Therapeutics Inc. CEO Neal Walker said “a lot of room within rheumatoid arthritis [RA]” remains for new drugs, such as his firm’s orally delivered ATI-450. “We see opportunities given the profile, the relative efficacy and safety that we've already demonstrated, to look at not only potentially monotherapy or earlier treatment of disease but also combo treatment, particularly given the safety profile. I think it's pretty well-known that polypharmacy is the rule in this indication.”
With the table set for phase III data due next month from a trial testing Travere Therapeutics Inc.’s sparsentan against focal segmental glomerulosclerosis (FSGS), investor appetite grows ever sharper for prospects in kidney disease.
Moncef Slaoui, chief advisor to Operation Warp Speed, the public-private partnership to hurry along a vaccine for COVID-19, cited tough going in the early days of development, as researchers met “a real challenge to engage the population.” During a panel discussion at the virtual 39th J.P. Morgan Healthcare Conference, he pointed to “a double-edged sword, to talk about what a vaccine can do, when we don’t know – and then once you know, you’re going to have to change your message.”
KSQ Therapeutics Inc.’s chief scientific officer, Frank Stegmeier, said that the CRISPRomics technology that drew Takeda Pharmaceutical Co. Ltd. to the table allows, “for the first time, genome-scale functional screening [in vivo as well as in vitro] across multiple disease settings. It really takes the guessing game out of your drug target selection.” Working with “an encyclopedia of gene function,” he said, KSQ aims to identify prospects that can have monotherapy activity in PD-1-refractory solid tumors.
Biond Biologics Ltd. co-founder and CEO Tehila Ben-Moshe told BioWorld that “a relatively small group of scientists who are very motivated started with a very basic scientific idea, which we were able to take all the way into clinical trials in four years,” and draw the interest of Paris-based Sanofi SA in a checkpoint inhibitor with multi-cell effects. In its second major deal of the week, Sanofi is pledging $125 million up front and more than $1 billion more in potential development, regulatory and sales-related milestone payments to Biond, of Misgav, Israel.
Bluebird Bio Inc. CEO Nick Leschly conceded that it’s “hard for folks on the outside looking in” to understand why the firm would cleave its severe genetic disease (SGD) and oncology efforts into two independently traded public companies, but said the Cambridge, Mass.-based firm is making the change by the end of this year in order “to make sure we can basically fall down and get back up and learn everything we can in the most disciplined manner,” he said.
Sarepta Therapeutics Inc.’s miss on a key phase II ambulatory endpoint in its Duchenne muscular dystrophy (DMD) trial may have been caused by a dramatic disparity in functional ability at baseline among older vs. younger patients afflicted with the progressively worsening disorder. In any case, Wall Street had knives out, carving 51% of the value away from shares (NASDAQ:SRPT) of the Cambridge, Mass.-based firm, which closed at $82.29, a loss of $86.66, or 51%.
Alnylam Pharmaceuticals Inc. said the 164-patient Helios-A phase III study with next-generation RNAi drug vutrisiran hit its primary endpoint as well as both secondary goals in the treatment of hereditary transthyretin-mediated amyloidosis with polyneuropathy. The primary endpoint was change from baseline in the modified Neuropathy Impairment Score at nine months as compared to historical placebo data from the Apollo phase III study of Alnylam’s Onpattro, cleared by the FDA for ATTR polyneuropathy in August 2018.
Pointing to his company’s work on “the next frontier in small molecules,” Ribometrix Inc. CEO Mike Solomon said the potential $1 billion-plus deal with Roche Holding AG offers evidence of big pharma’s mounting interest. Expertise in RNA folds helped Ribometrix nail down an agreement with Basel, Switzerland-based Roche’s Genentech arm that brings $25 million up front and potential milestone payments beyond $1 billion, as the pair discovers and develops RNA-targeted small-molecule therapeutics. Genentech gains exclusive rights to several predefined targets, including an exclusive global license for the development and commercialization of molecules.
COVID-19 vaccines have taken most of the limelight lately, but therapies are making progress, too, with San Diego-based Atyr Pharma Inc. and Cerecor Inc., of Rockville, Md., separately offering favorable phase II news.
