Kiniksa Pharmaceuticals Inc. and Scholar Rock Holding Corp. completed their journeys to the public markets, as Aptinyx Inc. – fresh off completion of its option deal with Allergan plc for a candidate to treat major depressive disorder – sought to follow in their footsteps.
Shares of Cara Therapeutics Inc. (NASDAQ:CARA) rocketed 42.8 percent Wednesday on word of the Stamford, Conn.-based company's global licensing deal with Vifor Fresenius Medical Care Renal Pharma Ltd. (VFMCRP), a chronic kidney disease (CKD)-focused joint venture (JV) formed by Vifor Pharma Group and Fresenius Medical Care. The arrangement covers the injectable formulation of difelikefalin (CR-845), conditionally branded Korsuva, to treat CKD-associated pruritus (CKD-aP), which has advanced into a pivotal phase III program.
Allergan plc exercised its option to acquire AGN-241751, an oral small-molecule N-methyl-D-aspartate (NMDA) receptor modulator that was discovered by Aptinyx Inc. and advanced under an ongoing research collaboration between the companies. Allergan gained option rights to certain small molecules from the Aptinyx discovery platform under a research collaboration initiated in conjunction with its 2015 acquisition of Naurex Inc., which simultaneously spun out Aptinyx and its platform. As part of that transaction, Allergan also acquired rapastinel, an intravenously administered NMDA receptor modulating tetrapeptide that subsequently received breakthrough therapy designation from the FDA and has advanced to phase III development in major depressive disorder (MDD). (See BioWorld Today, Sept. 16, 2015.)
Like David against Goliath, Roswell Park Cancer Institute (RPCI) spinout Mimivax LLC is seeking to take down one of the most common and aggressive forms of primary brain cancer, and interim findings from its U.S. multicenter phase II study suggest the Buffalo, N.Y.-based company may have a shot. Key Interim results of Survaxm, its survivin peptide mimic immunotherapeutic vaccine, showed that 91 percent of patients with newly diagnosed glioblastoma multiforme (nGBM) who received the treatment in combination with standard of care achieved 12-month overall survival (OS) compared to 61 percent historical standard of care. In addition, 96 percent achieved six-month progression-free survival (PFS) on the Survaxm regimen compared to 54 percent historical standard of care.
Accent Therapeutics Inc.formally launched with a $40 million series A and a platform built around epitranscriptomics – the field referring to RNA-modifying proteins (RMPs) that control many aspects of RNA biology – to pursue precision cancer therapies. The investment syndicate included the Column Group, Atlas Venture and Ecor1 Capital.
The celebrated annual release of abstracts from the American Society of Clinical Oncology (ASCO) meeting claimed an early victim Thursday as shares of Jounce Therapeutics Inc. (NASDAQ:JNCE) fell 35.2 percent, closing at $11.45 for a loss of $6.22, after analysts began sifting through the data dump and found the company's update wanting.
Prolific researcher, serial entrepreneur and investor Gregory Verdine nailed another nameplate to his wall with the formal introduction of 2016 startup Fog Pharmaceuticals Inc. (Fogpharma) in conjunction with the close of a $66 million series B. The round was led by 6 Dimensions Capital with participation from additional new investors GV (formerly Google Ventures), Blue Pool Capital, Horizons Ventures, Nan Fung Group and Leerink Partners. Existing investors Deerfield Management, Boyu Capital, Wuxi Apptec Corporate Ventures and undisclosed international noninstitutional investors joined the round.
Celsius Therapeutics became the latest biopharma spin-out from Third Rock Ventures, which also led the Cambridge, Mass.-based company's $65 million series A financing. GV (formerly Google Ventures), Heritage Provider Network, Casdin Capital, Alexandria Venture Investments and other undisclosed investors participated in the round for Celsius, which is seeking to develop precision medicines in autoimmune diseases and cancer by drilling genomic insights to the single cell level to understand which cells drive disease and identify the genes that trigger their malfunction.
The DNA base editing technology developed in the lab of David Liu, professor of chemistry and chemical biology, emerged as a core technology platform at Beam Therapeutics, which officially launched with up to $87 million in a cumulative series A financing. The multi-stage round will help Beam "go broad" across multiple years of drug discovery, according to CEO John Evans, to develop precision genetic medicines by making edits to individual base pairs in the genetic code. The funding also will support a cryptically described "affiliated entity."
In weighing presentations from Akcea Therapeutics Inc. and the FDA on the risk/benefit profile of the antisense oligonucleotide (ASO) volanesorsen (Waylivra) in familial chylomicronemia syndrome (FCS), members of the Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) came down on the side of the drug in a 12-8 vote, with no abstentions. In comments following the vote late Thursday, several EMDAC members said they gave more weight to the unmet need in FCS, an orphan disease with no approved treatments, than to concerns about adequate characterization of the patient population and the number and severity of adverse events (AEs). A few openly acknowledged they were swayed by comments during the open public hearing, when 13 individuals – patients, family caregivers and physicians – all spoke in favor of approval.
