Although Geron Corp.’s imetelstat met its primary and key secondary endpoints in a phase III study, the U.S. FDA is questioning the magnitude and durability of the effect of the first-in-class telomerase inhibitor as a second-line treatment of transfusion-dependent anemia in adults with low- to intermediate-1 risk myelodysplastic syndromes. The agency’s concerns resulted in more than an 12% stock tumble March 12 after the FDA released its briefing document two days ahead of an Oncology Drugs Advisory Committee meeting, in which the panel will be asked to vote on whether imetelstat’s benefits outweigh its risks.
Dimerix Ltd. announced a AU$20 million ($US13.22 million) capital raise following the news that its lead candidate, DMX-200, was successful in a prespecified interim analysis of the efficacy endpoint in its pivotal phase III trial in focal segmental glomerulosclerosis, a rare kidney disease.
Sosei Group Corp. is getting €25 million (US$27.3 million) up front in a global collaboration and option-to-license deal with Boehringer Ingelheim GmbH aimed at developing GPR52 agonists, a new target for schizophrenia designed to potentially address positive, negative and cognitive symptoms at the same time.
Prism Biolab Co. Ltd. has added Genentech Inc., a unit of Roche Holding AG, to the list of users for its peptide mimetic small-molecules library after signing a multitarget research collaboration and licensing agreement. Under the agreement, Tokyo-based Prism Biolab will open its Pepmetics Library, a library of peptide mimetic small molecules, to screen against targets selected by Roche and Genentech.
In June, Morphosys AG acquired Constellation Pharmaceuticals Inc. in a deal worth $1.7 billion in which the German biotech monetizes a royalty from a big-selling antibody, spending the lump sum on research into small-molecule BET inhibitors. It’s a bold move and one that perhaps demonstrates a renewed interest in small-molecule drugs after a period when antibody-based molecules have been the dominant force in the market for branded drugs.
Boston-based Nirogy Therapeutics Inc.’s $16.5 million series A round is meant to enable a pipeline of small-molecule drugs targeting the solute carrier family of transporter proteins (SLCTs) embedded in the cell membrane, and let the firm bring its front oncology runner to the clinic in 2022.
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.
Skyhawk Therapeutics Inc., one of the early leaders in developing small-molecule drugs that target RNA, closed out the year with yet another big pharma partnering deal, an alliance with Vertex Pharmaceuticals Inc., in which it is getting $40 million up front plus up to $2.2 billion more in milestones and royalties on product sales.
LONDON – DNA damage repair (DDR) specialist Artios Pharma Ltd. has sealed a $860 million per target deal with Merck KGaA, around a series of small-molecule DNA nuclease inhibitors. The deal, for up to eight targets, will see Artios get $30 million in up-front and near-term milestones to take programs to the point at which Merck will decide on its option to take them in-house. For any products that make it to market, the $860 million in milestones along the way will be followed by double-digit royalties on sales. Subject to certain conditions, Cambridge-based Artios has rights to do joint development with Darmstadt, Germany-based Merck.
Kronos Bio Inc. closed a $105 million series A preferred stock financing, shored up by a board composed of longtime pharma executives that includes the company CEO and president, Norbert Bischofberger, the former Gilead Sciences Inc. R&D executive vice president and chief science officer.
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