https://www.bioworld.com/rss en-us Scarlet Therapeutics Ltd. has demonstrated its manufactured red blood cells (RBCs) act in the same way as their natural counterparts in vivo, opening the way for the cells to be used as drug delivery vehicles and raising the possibility they could replace conventional blood transfusions. To build on this, Scarlet has closed a $4 million seed round to work on the first clinical application, in which RBCs loaded with therapeutic proteins will be used to treat rare metabolic diseases. http://www.bioworld.com/articles/730981 Fri, 08 May 2026 12:00:00 -0400 https://www.bioworld.com/articles/730981-scarlet-raises-4m-to-take-manufactured-red-blood-cells-into-clinic Red blood cells The U.S. FDA approved Corcept Therapeutics Inc.’s oral, selective glucocorticoid receptor antagonist, Lifyorli (relacorilant), nearly four months ahead of schedule for adults with platinum-resistant epithelial ovarian, fallopian tube or primary peritoneal cancer. A short time after the agency approved Lifyorli, it cleared Denali Therapeutics Inc.’s Avlayah (tividenofusp alfa) under the accelerated approval pathway for mucopolysaccharidosis II, also called Hunter syndrome, ahead of the April 5 PDUFA date. http://www.bioworld.com/articles/729838 Wed, 25 Mar 2026 12:00:00 -0400 https://www.bioworld.com/articles/729838-early-fda-wins-corcepts-lifyorli-denalis-avlayah Lifyorli Credit: Corcept Therapeutics Inc. A new study has reinforced that GLP-1 receptor agonists are unlikely to produce durable weight loss, but indicates that rather than returning to the starting weight, individual weight gain will plateau at 75.5% of the weight lost. http://www.bioworld.com/articles/729342 Thu, 05 Mar 2026 12:00:00 -0500 https://www.bioworld.com/articles/729342-study-weight-regain-after-glp-1s-plateaus-below-starting-weight Bathroom scale and injector pens Targeting an ultrarare disease affecting about 250 U.S. residents, Immedica Pharma AB’s Loargys (pegzilarginase) won accelerated FDA approval to treat hyperargininemia in those ages 2 and older with arginase 1 deficiency (ARG1-D). http://www.bioworld.com/articles/729191 Tue, 24 Feb 2026 12:00:00 -0500 https://www.bioworld.com/articles/729191-immedica-wins-us-fda-nod-for-loargys-to-treat-arg1-d Green approved stamp After expanding development of DM-199 (rinvecalinase alfa) into preeclampsia last year, Diamedica Inc. rolled out early phase II results showing the recombinant form of human tissue kallikrein-1 reduced the mother’s blood pressure, did not cross the placental barrier, and potentially improves blood flow to the fetus. http://www.bioworld.com/articles/722267 Fri, 18 Jul 2025 11:00:00 -0400 https://www.bioworld.com/articles/722267-diamedicas-dm-199-shows-promise-for-serious-pregnancy-disorder Pregnancy Alteogen Inc. completed a merger between two subsidiaries – Altos Biologics Inc. and Alteogen Healthcare Inc. – branding the new entity as Alteogen Biologics Inc. http://www.bioworld.com/articles/720695 Tue, 27 May 2025 11:00:00 -0400 https://www.bioworld.com/articles/720695-alteogen-merges-two-subsidiaries-forming-alteogen-biologics Alteogen headquarters in Daejeon, South Korea Alteogen headquarters in Daejeon, South Korea. Credit: Alteogen Inc. Alteogen Inc. completed a merger between two subsidiaries – Altos Biologics Inc. and Alteogen Healthcare Inc. – branding the new entity as Alteogen Biologics Inc. http://www.bioworld.com/articles/720469 Fri, 23 May 2025 11:00:00 -0400 https://www.bioworld.com/articles/720469-alteogen-merges-two-subsidiaries-forming-alteogen-biologics Alteogen headquarters in Daejeon, South Korea Alteogen headquarters in Daejeon, South Korea. Credit: Alteogen Inc. It’s been a big week for Inozyme Pharma Inc. On the heels of a promising interim readout for phase III-stage enzyme replacement therapy (ERT) candidate INZ-701 in ENPP1 deficiency, the firm agreed to be acquired by Biomarin Pharmaceuticals Inc. in a deal valued at about $270 million, putting the rare disease ERT in the hands of an experienced commercial team. http://www.bioworld.com/articles/720190 Fri, 16 May 2025 11:00:00 -0400 https://www.bioworld.com/articles/720190-biomarin-snags-potential-first-in-disease-ert-in-270m-inozyme-buy Acquisition puzzle Just two months after Inozyme Pharma Inc. cut its workforce by 25% and prioritized activities to focus on a BLA filing for INZ-701 for ENPP1 deficiency, interim phase III data from its Energy 3 trial showed consistent safety and immunogenicity and increased phosphate levels in patients treated with the rare disease enzyme replacement therapy. http://www.bioworld.com/articles/720171 Thu, 15 May 2025 11:00:00 -0400 https://www.bioworld.com/articles/720171-inozymes-inz-701-shows-promise-for-rare-disease-enpp1-deficiency After dropping development in December of its lead program, Spruce Biosciences Inc. has found new life by acquiring a BLA-ready enzyme replacement therapy for the rare genetic neurodegenerative disease Sanfilippo syndrome type B. If approved, the therapy, tralesinidase alfa, could bring Spruce a priority review voucher. http://www.bioworld.com/articles/719212 Tue, 15 Apr 2025 11:00:00 -0400 https://www.bioworld.com/articles/719212-spruce-acquires-biomarins-sanfilippo-drug-tralesinidase-alfa Alteogen Inc. signed its first billion-dollar partnership this year for ALT-B4 technology through two separate contracts with Astrazeneca plc’s U.K.- and U.S.-based Medimmune subsidiaries worth up to $1.35 billion combined. http://www.bioworld.com/articles/718433 Tue, 18 Mar 2025 07:00:00 -0400 https://www.bioworld.com/articles/718433-alteogen-in-135b-medimmune-deal-astrazeneca-bids-1b-for-esobiotec Rendering of Alteogen’s ALT-B4 Rending of ALT-B4, a human recombinant enzyme. Credit: Alteogen Inc. Alteogen Inc. signed its first billion-dollar partnership this year for ALT-B4 technology through two separate contracts with Astrazeneca plc’s U.K.- and U.S.-based Medimmune subsidiaries worth up to $1.35 billion combined. http://www.bioworld.com/articles/718332 Mon, 17 Mar 2025 07:00:00 -0400 https://www.bioworld.com/articles/718332-alteogen-in-135b-medimmune-deal-astrazeneca-bids-1b-for-esobiotec Rendering of Alteogen’s ALT-B4 Rending of ALT-B4, a human recombinant enzyme. Credit: Alteogen Inc. Ascentage Pharma Group Corp. Ltd. has priced the first IPO of the year in the U.S. The offering of 7.32 million shares has the Suzhou, China, and Rockville, Md.-based company looking for gross proceeds of about $126.4 million, assuming a price of $17.25 per share. http://www.bioworld.com/articles/716992 Tue, 04 Feb 2025 12:00:00 -0500 https://www.bioworld.com/articles/716992-ascentage-prices-first-us-ipo-of-the-year Ascentage Pharma Group Corp. Ltd. has priced the first IPO of the year in the U.S. The offering of 7.32 million shares has the Suzhou, China, and Rockville, Md.-based company looking for gross proceeds of about $126.4 million, assuming a price of $17.25 per share. http://www.bioworld.com/articles/716596 Fri, 24 Jan 2025 12:00:00 -0500 https://www.bioworld.com/articles/716596-ascentage-prices-first-us-ipo-of-the-year Data from two out of three positive studies faced the U.S. FDA’s Anesthetic and Analgesic Drug Products Advisory Committee as members examined the package submitted by Seikagaku Corp., of Tokyo, for SI-6603 (condoliase), a chemonucleolytic drug for radicular leg pain associated with lumbar disc hernia. http://www.bioworld.com/articles/716341 Tue, 14 Jan 2025 12:00:00 -0500 https://www.bioworld.com/articles/716341-seikagakus-ferring-partnered-condoliase-for-pain-wins-at-adcom Medical art concept for radicular leg pain or sciatic nerve pain Data from two out of three positive studies faced the U.S. FDA’s Anesthetic and Analgesic Drug Products Advisory Committee as members examined the package submitted by Seikagaku Corp., of Tokyo, for SI-6603 (condoliase), a chemonucleolytic drug for radicular leg pain associated with lumbar disc hernia. http://www.bioworld.com/articles/716168 Fri, 10 Jan 2025 12:00:00 -0500 https://www.bioworld.com/articles/716168-seikagakus-ferring-partnered-condoliase-for-pain-wins-at-adcom Medical art concept for radicular leg pain or sciatic nerve pain The U.S. FDA’s Anesthetic and Analgesic Drug Products Advisory Committee will scrutinize Jan. 10 the safety and efficacy of Ferring Pharmaceuticals Inc.’s SI-6603 (condoliase), a chemonucleolytic drug for radicular leg pain associated with lumbar disc hernia. http://www.bioworld.com/articles/716117 Thu, 09 Jan 2025 12:00:00 -0500 https://www.bioworld.com/articles/716117-ferring-faring-ok-in-briefing-docs-for-condoliase-leg-pain-adcom Genentech Inc. didn’t need to wait until Thanksgiving for the U.S. FDA to make up its mind. More than a month ahead of its PDUFA date, the agency approved the firm’s first-line breast cancer treatment, Itovebi (inavolisib), providing the oral therapy a place with other niched therapies from Astrazeneca plc and Novartis AG. Itovebi is to be combined with Pfizer Inc.’s palbociclib (Ibrance) and Faslodex (fulvestrant, Astrazeneca) for adults with endocrine-resistant, PIK3CA-mutated, hormone receptor (HR)-positive, HER2-negative, locally advanced or metastatic breast cancer. http://www.bioworld.com/articles/713455 Fri, 11 Oct 2024 12:00:00 -0400 https://www.bioworld.com/articles/713455-fda-gives-early-nod-to-genentechs-first-line-breast-cancer-drug Breast cancer illustration The U.S. FDA has granted accelerated approval to Novartis AG’s Fabhalta (iptacopan) for reducing proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression. The approval strengthens the company’s renal disease presence as it puts two other IgAN treatments through clinical trials. http://www.bioworld.com/articles/711432 Thu, 08 Aug 2024 12:00:00 -0400 https://www.bioworld.com/articles/711432-yes-igan-novartis-proteinuria-therapy-receives-fda-approval Researchers in Japan have discovered that a phage-derived enzyme called endolysin, which targets highly resistant biofilm-forming bacteria, could help restore the gut microbiota to mitigate acute graft-vs.-host disease. Acute graft-vs.-host disease (aGVHD) is a common complication for patients who undergo allogeneic hematopoietic cell transplantation (allo-HCT). Recent studies have highlighted the significant role of the microbiome in aGVHD, with dysbiosis contributing to its pathogenesis. http://www.bioworld.com/articles/710541 Tue, 16 Jul 2024 09:00:00 -0400 https://www.bioworld.com/articles/710541-phage-derived-enzyme-can-help-fight-acute-graft-vs-host-disease Microbiome illustration Credit: Darryl Leja, NHGRI Disappointing top-line data from two failed phase III studies of soticlestat for Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) have crumpled the stock of co-developer Ovid Therapeutics Inc., which lost three-quarters of its value June 17. http://www.bioworld.com/articles/709733 Tue, 18 Jun 2024 12:00:00 -0400 https://www.bioworld.com/articles/709733-ovid-takes-hit-on-phase-iii-epilepsy-failures-in-takeda-venture Epileptic brain and abnormal EEG wave discharges Disappointing top-line data from two failed phase III studies of soticlestat for Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) have crumpled the stock of co-developer Ovid Therapeutics Inc., which lost three-quarters of its value June 17. http://www.bioworld.com/articles/709521 Mon, 17 Jun 2024 12:00:00 -0400 https://www.bioworld.com/articles/709521-ovid-takes-hit-on-phase-iii-epilepsy-failures-in-takeda-venture Epileptic brain and abnormal EEG wave discharges Focused on oral therapies for obesity, diabetes and rare diseases, Boston-based Syntis Bio Inc., which raised $15.5 million through seed funding last year, emerged from stealth to advance its synthetic tissue-lining technology and a pipeline of candidates. http://www.bioworld.com/articles/709551 Wed, 12 Jun 2024 12:00:00 -0400 https://www.bioworld.com/articles/709551-syntis-bio-launches-for-oral-obesity-rare-disease-drugs Rahul Dhanda, president and CEO, Syntis Bio Rahul Dhanda, president and CEO, Syntis Bio Focused on oral therapies for obesity, diabetes and rare diseases, Boston-based Syntis Bio Inc., which raised $15.5 million through seed funding last year, emerged from stealth to advance its synthetic tissue-lining technology and a pipeline of candidates. http://www.bioworld.com/articles/709451 Tue, 11 Jun 2024 12:00:00 -0400 https://www.bioworld.com/articles/709451-syntis-bio-launches-for-oral-obesity-rare-disease-drugs Rahul Dhanda, president and CEO, Syntis Bio Rahul Dhanda, president and CEO, Syntis Bio Syntis Bio Inc. has announced its launch, with a focus on advancing a pipeline of oral therapies engineered for optimization in the small intestine and a portfolio of enzyme replacement therapies to treat orphan metabolic and broad digestive disorders. http://www.bioworld.com/articles/709368 Tue, 11 Jun 2024 09:00:00 -0400 https://www.bioworld.com/articles/709368-syntis-bio-advances-oral-enzyme-replacement-therapies-toward-ind-filing AI-generated illustration of enzyme activity The U.S. FDA has granted orphan drug status to HM-15421 (GC-1134A, LA-GLA) for the treatment of Fabry disease. http://www.bioworld.com/articles/708978 Tue, 28 May 2024 09:00:00 -0400 https://www.bioworld.com/articles/708978-us-orphan-drug-status-granted-to-hanmi-and-gc-biopharma-candidate-for-fabry-disease Test tube, dropper, DNA illustration Zai Lab Ltd., of Shanghai and Cambridge, Mass., has been on a regulatory roll in China, gaining approval of Bristol Myers Squibb Co.’s Augtyro (repotrectinib) for lung cancer on May 12 and NMPA acceptance of subcutaneous Vyvgart‘s (efgartigimod alfa SC) supplemental BLA with priority review on May 14. http://www.bioworld.com/articles/708792 Tue, 21 May 2024 12:00:00 -0400 https://www.bioworld.com/articles/708792-zai-lab-exec-buys-stock-as-augtyro-vyvgart-sc-advances-in-china Joshua Smiley, president and chief operating officer, Zai Lab Joshua Smiley, president and chief operating officer, Zai Lab South Korea’s HLB Co. Ltd. saw its stock (KOSDAQ:028300) drop nearly 30% on May 17 as CEO Jin Yang-gon announced the U.S. FDA’s complete response letter (CRL) to its NDA seeking approval of its rivoceranib and camrelizumab (Airtuika, Jiangsu Hengrui Pharmaceutical Co. Ltd.) combo for liver cancer. http://www.bioworld.com/articles/708791 Tue, 21 May 2024 12:00:00 -0400 https://www.bioworld.com/articles/708791-fda-issues-crl-on-rivoceranib-camrelizumab-combo-in-liver-cancer To strengthen its cancer pipeline, South Korea’s Dong-A ST Co. Ltd. made a strategic investment of ₩25 billion (US$18.45 million) in Seocho-gu, Seoul-based Idience Co. Ltd., a cancer-focused subsidiary of Ildong Pharmaceutical Co. Ltd. on May 20. http://www.bioworld.com/articles/708790 Tue, 21 May 2024 12:00:00 -0400 https://www.bioworld.com/articles/708790-dong-a-st-invests-25b-in-ildong-subsidiary-inks-cancer-deal To strengthen its cancer pipeline, South Korea’s Dong-A ST Co. Ltd. made a strategic investment of ₩25 billion (US$18.45 million) in Seocho-gu, Seoul-based Idience Co. Ltd., a cancer-focused subsidiary of Ildong Pharmaceutical Co. Ltd. on May 20. The equity buyout makes Dongdaemun-gu, Seoul-based Dong-A ST the second largest shareholder of Idience, following Ildong Pharmaceutical. The two companies also shook on a co-development deal for Idience’s lead cancer asset, venadaparib (IDX-1197). http://www.bioworld.com/articles/708781 Mon, 20 May 2024 12:00:00 -0400 https://www.bioworld.com/articles/708781-dong-a-st-invests-25b-in-ildong-subsidiary-inks-cancer-deal