Becoming the first and only fully FDA-approved treatment for focal segmental glomerulosclerosis (FSGS), Travere Therapeutics Inc.’s Filspari (sparsentan) has gained access to a second lucrative market with a regulatory package that validates proteinuria as a surrogate endpoint. The approval, disclosed during after-market hours on the April 13 PDUFA date, drove company shares (NASDAQ:TVTX) up by 34% to $41.19 midday on April 14. Filspari won accelerated approval in 2023 and full approval in 2024 for IgA nephropathy (IgAN). The approval for FSGS is specifically to reduce proteinuria for children and adults 8 and older who do not have nephrotic syndrome (NS) – with the exclusion of NS a surprise to many analysts but one with low impact as the expanded label suggests peak sales in both FSGS and IgAN could reach $3 billion.

More of everything as Amazon moves into AI-driven drug R&D

Amazon is extending the reach of its “everything store” into drug R&D with the launch of an artificial intelligence-powered Bio Discovery business. The company has compiled a catalogue of 40-plus foundation models that have been trained on extensive biology datasets and are able to generate and evaluate drug molecules in silico. For now, this covers antibodies only, but it is intended to move into other modalities. To extend the utility of this resource beyond the coding skills of computational biologists, there is a chatbot/assistant that automates complex tasks, selecting the appropriate model for a particular project, optimizing the outputs and homing in on which molecules should be synthesized and tested in the lab.

Cortec prepares to implant BCI tech into third stroke patient

Cortec GmbH is preparing to implant its brain-computer interface (BCI) technology into a third stroke patient, with early clinical data showing that the device leads to significant improvement in hand and arm function. The company believes its Brain Interchange system could transform the lives of millions of patients. The technology recently secured breakthrough device designation, which CEO Frank Desiere told BioWorld is confirmation of the therapeutic use case and the technology by the FDA.

‘Immune reset’ is ambition of Tr1x’s allogenic Tr1 cell therapy

Tr1x Inc. was founded with a simple but ambitious goal: to change how autoimmune and inflammatory diseases are treated, moving away from chronic treatment and toward durable cures. “We are trying to flip the script on regulatory T cells (Tregs),” Tr1x CEO David de Vries told BioWorld. “The goal is to reset the immune system rather than continuously suppress it, and we believe we have a unique technology to do that.” Tr1x uses proprietary technology to take healthy donor-derived CD4+ T cells and reprogram them into allogenic, in vivo type 1 regulatory T cells (Tr1 cells) at commercial scale.

Abbvie nabs two Nav1.8 pain projects from Haisco in $745M deal

Abbvie Inc. is buying exclusive rights to develop, manufacture and commercialize two Nav1.8 inhibitors for pain – HSK-55718 and HSK-51155 – from Haisco Pharmaceutical Group Co. Ltd. for $30 million up front and up to $715 million in milestone payments, plus royalties. Under the terms announced April 13, Haisco will grant Abbvie rights to develop the two compounds for pain-related indications globally excluding mainland China, Hong Kong and Macau. HSK-55718 and HSK-51155 are designed to inhibit abnormal discharges of pain nerves and reduce pain signal transmission by selectively blocking specific sodium ion channels, according to Haisco.

Avanos Medical to go private in $1.27B acquisition by AIP

Avanos Medical Inc. has agreed to be acquired by affiliates of American Industrial Partners (AIP) in an all-cash deal valuing the company at approximately $1.272 billion. Under the terms of the definitive agreement, Avanos stockholders will receive $25 per share in cash for each share of common stock they hold.

Super enhancers drive super selective tumor killing

Researchers at the University of Edinburgh are pioneering a cancer therapy that destroys tumors from within while reawakening the immune system, using synthetic super-enhancers to drive targeted killing and durable protection against recurrence. The work builds on a decade of research focused on how glioblastoma stem cells sustain their aggressive cancer identity. The key insight was that these cancer cells are governed by a central transcriptional network, a regulatory system controlled by master factors such as SOX2 and SOX9 that determine cell identity and direct their behavior.

Nearly 30% of trials not up to date on results disclosures

The U.S. FDA sent out a friendly reminder to more than 2,200 sponsors and researchers, associated with more than 3,000 clinical trials, who may be delinquent in disclosing trial results – both positive and negative – on clinicaltrials.gov. That number represents nearly 30% of the drug and device studies that likely fall under the mandatory reporting requirements. If the data aren’t disclosed soon, the next step may be pre-notices or notices of noncompliance, the agency said.

US FDA cites first dispenser for violating Rx supply chain act

If a recent warning letter is anything to go by, the U.S. FDA could be clamping down on dispensers that purchase drug products from unauthorized sources in violation of the 2013 Drug Supply Chain Security Act (DSCSA). The first warning to a dispenser under the DSCSA, the letter reminded Pure Indulgence Aesthetics, of Southlake, Texas, that it qualifies as a dispenser under the act and, thus, is subject to its requirements pertaining to product tracing, product identification, verification and use of authorized trading partners.

Also in the news

Abbvie, Allogene, Alx Oncology, Apotex, Beckman Coulter Diagnostics, Biontech, Caliway, Collplant, Corcept, Daiichi, Dariohealth, Duality, Eli Lilly, Epitopea, Frontier, Genmab, Geron, GSK, Hemab, Ideaya, Imagenebio, Impact, Kymera, Leal, Lineage, Lixte, Lobe, Merck, Neomorph, Nrx, Ocular, OS, Palvella, Pelthos, Pharmasens, Prosomnus, Prothena, Quiver, Regeneron, Replimune, Revolution, Roche, Sanofi, Seaport, Servier, Sharp, Spyre, Synox