While an “unexpected placebo effect” marred its primary endpoint, the pivotal phase II/III study testing AMO-02 (tideglusib) showed clinically significant benefits across a range of functional and objective assessments, according to developer Amo Pharma Ltd., which is prepping to meet with regulators to discuss potential approval for use in children and adolescents with congenital myotonic dystrophy type 1 (CDM1), an ultra-rare subtype of myotonic dystrophy type 1 for which no treatment options are available.
Star Therapeutics Inc. brought on board six new investors in its oversubscribed $90 million series C round, raising the company’s total funding to more than $190 million since its founding in 2018 to advance in-house drug discovery efforts via formation of portfolio companies. Since emerging from stealth in early 2022, Star has launched two aptly named companies, Electra Therapeutics Inc. and Vega Therapeutics Inc., with plans to unveil additional ventures arising from its antibody discovery efforts.
As its name suggests, Superluminal Medicines Inc. is aiming for speed. The startup, which closed a $33 million seed round led by RA Capital Management, is combining a biology-focused approach with a generative AI platform it says has the potential to create candidate-ready compounds in a matter of months, with its initial sights set on G protein-coupled receptor targets.
As its name suggests, Superluminal Medicines Inc. is aiming for speed. The startup, which closed a $33 million seed round led by RA Capital Management, is combining a biology-focused approach with a generative AI platform it says has the potential to create candidate-ready compounds in a matter of months, with its initial sights set on G protein-coupled receptor targets.
The narrative of TIGIT-targeting immunotherapy development, beset by negative news in recent months, has found a positive plotline again, thanks to what Roche Holding AG referred to as “an inadvertent disclosure” of a second interim analysis from the phase III Skyscraper-01 study testing anti-TIGIT candidate tiragolumab with anti-PD-L1 antibody Tecentriq (atezolizumab) in non-small-cell lung cancer.
The impact of a black box warning on Neurocrine Biosciences Inc.’s expanded U.S. label for Ingrezza (valbenazine) to treat chorea associated with Huntington’s disease (HD) appears up for debate, following FDA approval late Aug. 18. But analysts agree that it is unlikely to affect sales of the VMAT2 inhibitor in tardive dyskinesia, for which the company recently raised 2023 guidance as high as $1.82 billion.
Six years of collaboration is ending in a buyout, as Decibel Therapeutics Inc. agreed to be acquired by Regeneron Pharmaceuticals Inc. in a deal valued at up to $213 million, including $109 million in equity, with additional payments via contingent value rights linked to clinical and regulatory milestones for DB-OTO, the lead gene therapy program targeting hearing loss.
When Nektar Therapeutics Inc. decided to push ahead with development of Treg stimulator rezpegaldesleukin (rezpeg) in atopic dermatitis despite what appeared to be middling early stage data, investors weren’t exactly jumping up and down. But it turns out those data are more promising than originally thought.
When looking for a name, the founders of startup Nexo Therapeutics Inc., which recently emerged from stealth mode with a $60 million series A financing and a research collaboration with the University of Texas MD Anderson Cancer Center, sought a moniker that truly captured its approach to drug development.
Citius Pharmaceuticals Inc.’s retooled version of withdrawn cancer drug Ontak (denileukin diftitox) is not quite ready for prime time, according to the U.S. FDA, which issued a complete response letter (CRL) for the interleukin-2-diphtheria toxin fusion protein known as Lymphir. The agency is asking for Citius to incorporate enhanced product testing and additional controls but did not raise any concerns regarding safety and efficacy, the company said.
