Prime Medicine Inc. (PM) likely has “a lock on prime editing technology for therapeutic uses,” CEO Keith Gottesdiener told BioWorld, though research labs are continuing to refine the approach. Cambridge, Mass.-based PM has $315 million in the bank that will help advance the platform, which behaves like a DNA word processor to search and replace disease-causing genetic sequences at their exact location in the genome.
Riding high on dramatic phase Ib results with KIT receptor-binding monoclonal antibody CDX-0159 in urticaria (hives), Celldex Therapeutics Inc. plans to begin phase II work in the first half of next year. Shares of Hampton, N.J.-based Celldex (NASDAQ:CLDX) closed July 12 at $40.67, up $8.26, or 25%, as Wall Street reacted to favorable data from the trial in antihistamine-refractory cold urticaria and symptomatic dermographism, the two most common forms of chronic inducible urticaria (CindU).
Officials at Sigilon Therapeutics Inc. declined to comment beyond a press release on the FDA’s clinical hold for the phase I/II study with encapsulated cell therapy SIG-001 for severe or moderately severe hemophilia A. Shares of Cambridge, Mass.-based Sigilon (NASDAQ:SGTX) closed at $6.90 on July 9, down $2.34, or 25% after Wall Street learned of the regulatory move, which came because one of three patients treated has developed inhibitors to factor VIII (FVIII).
As rumblings continued about possible skulduggery between Biogen Inc. and the FDA related to Aduhelm (aducanumab), an already precedent-setting case took yet another turn: Acting FDA Commissioner Janet Woodcock asked acting U.S. Inspector General Christi Grimm’s office to conduct an independent review of goings-on that led to approval of the amyloid-beta-targeting drug in Alzheimer’s disease.
Following a priority review, the FDA approved Bayer AG’s Kerendia (finerenone) for chronic kidney disease (CKD) associated with type 2 diabetes (T2D). A non-steroidal mineralocorticoid receptor antagonist, Kerendia targets a key driver in CKD and has proven positive as well with regard to cardiovascular outcomes – risked especially by patients with loss of kidney function.
Recent findings with the sodium glucose transporter-2 inhibitor Jardiance (empagliflozin) from Boehringer Ingelheim International GmbH and Eli Lilly and Co. drew more attention to the cardiovascular space.
The whopper June buyout of by Morphosys AG of Constellation Pharmaceuticals Inc. brought renewed attention to myelofibrosis (MF) and other players in the space, such as Sierra Oncology Inc.
Advaxis Inc.’s long journey is ending happily by way of a merger with Biosight Ltd. that leaves the new firm with $50 million to help advance aspacytarabine (BST-236), a prodrug of cytarabine for acute myeloid leukemia and myelodysplastic syndromes.
Alector Inc. Chief Operating Officer Shehnaaz Suliman said her company’s deal with Glaxosmithkline plc (GSK) – worth as much as $2.2 billion – “allows us to expand into indications that we have been quite thoughtful about doing, but in a more expeditious manner. This is really an opportune time to continue to explore the biology of these programs across multiple indications.”
Diamedica Therapeutics Inc.’s chief medical officer, Harry Alcorn, said that “due to the complexity [of diabetic kidney disease (DKD)], there's not a clear answer” as to why such patients did less well in the company’s Redux phase II trial with DM-199 (recombinant human tissue kallikrein 1 [KLK1]). “But I wouldn't say that there wasn't a response in the DKD group,” he said, citing upside in 30% of subjects.
