At first glance, the number of drugs that received accelerated approval from the U.S. FDA’s Center for Drug Evaluation and Research (CDER) in 2023 was nothing to write home about. Yes, CDER granted nine accelerated approvals last year, up from six in 2022. But the proportion of novel drugs with accelerated approval was 16% both years. And when compared with the 12 drugs in 2020 and the 14 that received accelerated approval in 2021, last year’s crop was a little lackluster. However, a deeper look at the 2023 accelerated approvals shows a historic milestone. For the first time since the path was created in 1992, the number of novel biologics getting accelerated approval at CDER outpaced the number of small-molecule drugs.
Biotheus, Hansoh ink potential ¥5B deal for new bispecific ADCs
Chinese biotechs Biotheus Inc. and Hansoh Pharmaceutical Group Co. Ltd. partnered again under a new potential ¥5 billion (US$698.98 million) deal to develop bispecific antibody-drug conjugates (ADCs), using Biotheus’ EGFR-cMet bispecific antibody. Zhuhai, South Guangdong province-based Biotheus said it would grant Hansoh a worldwide license to use its EGFR-cMet bispecific antibody called PM-1080 (HS-20117) to develop, produce and commercialize bispecific ADCs for cancer.
Norgine nabs ex-US rights to Pedmarqsi in deal worth up to $273M
Fennec Pharmaceuticals Inc. has out-licensed its chemoprotective formulation of sodium thiosulfate, Pedmarqsi, to Norgine BV for €40 million (US$44 million) up front and up to an additional €210 million (US$229 million) in commercial and regulatory milestones. The injectable drug reduces the risk of cisplatin-induced ototoxicity in pediatric patients 1 month and older with localized, nonmetastatic solid tumors. The formulation was approved in the U.S. on Sep. 21, 2022, where it is branded as Pedmark. It later won approval in the EU and the U.K. for the same indication.
PYC’s AU$74M advancing RNA therapy for retinitis pigmentosa
PYC Therapeutics raised AU$40 million of an anticipated AU$74 million (US$48.6 million) capital raise to advance three candidates, including lead candidate VP-001, which could potentially be the first treatment for retinitis pigmentosa type 11 (RP11), which causes blindness that begins in childhood and ultimately leads to legal blindness by middle age. The company’s CEO Rohan Hockings told BioWorld that VP-001 is poised to enter a registrational study in 2025. There are currently no treatment options available for patients with RP11, which represents an estimated market worth more than $1 billion per year.
Relation raises additional $35M for ‘lab in the loop’ system
Techbio specialist Relation Therapeutics Ltd. has raised $35 million in new seed funding, bringing total seed money to $60 million, as it advances development of its in silico/wet lab platform for identifying drug targets in the non-coding parts of the genome. The company is building a “lab in the loop” system where in depth ‘omics profiles of single cells from fresh patient tissues are analyzed by its machine learning engine to uncover the genetic basis of clinical phenotypes and identify novel targets. In the year since it raised the initial seed funding, Relation has validated the technology in its lead indication of osteoporosis, identifying a number of targets it is now preparing to drug.
Aignostics, Bayer deal connects tumor profiles to patient outcomes
Aignostics GmbH has embarked on raising a €20-€30 million (US$21.74-$32.61 million) series B round after validating its artificial intelligence-driven precision oncology platform in a co-development deal with Bayer AG. This is the first pharma partnership for the Berlin, Germany-based company. Financial terms were not disclosed, but Aignostics will receive an up-front payment and is eligible to receive success-based milestone payments and royalties on any commercialized therapies that result from the collaboration. Aignostics and Bayer will now work together to build a target identification platform and also to connect tumor profiles to patient outcomes, enabling the identification of likely responders in clinical trials.
US appeals court: States can fill the silence on 340B discounts
The U.S. Court of Appeals for the Third Circuit may have ruled last year that the Department of Health and Human Services doesn’t get to fill in the gaps in the law that created the 340B prescription drug discount program, but some states and lawmakers are coming up with their own workarounds to force the discounts to contract pharmacies. The Eighth Circuit last week upheld one of those workarounds, saying states can fill in that gap, as “the practice of pharmacy is an area traditionally left to state regulation.” In so ruling, the appeals court found that an Arkansas law requiring drug manufacturers to give the discounts to contract pharmacies in the state is not pre-empted by federal law.
Also in the news
Adaptive Phage, Aquestive, Audentes, Auxilius, Beigene, Biomx, Bluebird, Bristol Myers Squibb, Cantargia, Cartesian, Checkpoint, Clearside, Contineum, Cybin, Elicio, Fulcrum, Gilgamesh, Idorsia, Immvira, Jasper, Karuna, Lantern, Madrigal, Merck, Nuvation, Ocugen, Oragenics, Puretech, Recce, Scilex, Senisca, Spyre, Viatris, Virpax