Day two of the J.P Morgan Healthcare Conference rolled on with positive data from Sling Therapeutics Inc. that is leading the company to a phase III study in treating thyroid eye disease. The privately held company posted top-line efficacy and safety results from a phase IIb/III study of its lead candidate, linsitinib, which hits its primary endpoint with statistical significance in the twice-daily, 150-mg oral dose. Sling said its confirmatory phase III study of the small molecule will begin sometime in 2025. The 43rd annual conference has more than 8,000 attendees with about 500 companies presenting through this Thursday, Jan. 16.

Alliance for Regenerative Medicine: Field growing, cost still an issue

The accelerating pace of U.S. FDA approvals for cell and gene therapies is “great for the field and great news for the patients,” but questions remain over commercialization, with “costs remaining stubbornly high.” That was the glass half-full summary of Tim Hunt, president of the industry group, the Alliance for Regenerative Medicine, reprising progress in 2024, and looking forward to the prospects for further growth and the potential impact of the incoming Trump administration in 2025.

Regenxbio, Nippon in $810M deal for Hunter and Hurler gene therapies

In a deal potentially worth $810 million for Regenxbio Inc., Nippon Shinyaku Co. Ltd. is partnering on the development and commercialization of RGX-121 for Hunter syndrome and RGX-111 for Hurler syndrome. Terms call for Regenxbio to receive $110 million at closing and up to $700 million in milestone payments, plus double-digit royalties on net sales in the licensed territories. Nippon Shinyaku will commercialize both products in the U.S. and Asia, while Regenxbio will lead manufacturing, clinical and commercial supply, and all future clinical development. It also retains all rights to, and potential proceeds from, a priority review voucher it may attain upon U.S. FDA approval of RGX-121, and it reserves the right to develop and commercialize RGX-121 and RGX-111 in countries outside of the U.S. and Asia.

CSPC wins China clearance of new DPP-4 inhibitor

CSPC Pharmaceutical Group Ltd. on Jan. 13 gained the National Medical Products Administration’s approval of Shanzeping (prusogliptin tablets; DBPR-108) as a novel oral dipeptidyl peptidase-IV (DPP-4) inhibitor to treat adult patients with type 2 diabetes. CSPC’s Shanzeping is now cleared as a monotherapy or combination therapy with metformin to improve glycemic control in adults with type 2 diabetes who failed to achieve adequate control with metformin alone.

Roche, Prothena forge ahead in MTBR; others, too

Microtubule binding region (MTBR)-targeting candidates are coming to the fore, even as the market digests Roche AG’s news in December that prasinezumab, partnered with Prothena plc, fell short of its primary phase IIb endpoint in Parkinson’s disease. The pair isn’t giving up, though. Firms busy separately in the MTBR space include such names as Eisai Co. Ltd. and UCB SA.

In monkeys, enhanced TB vaccine dissolves after inducing protection

An experimental tuberculosis vaccine with a dual mission – self-destruction after inducing immunity – improved the design of the Bacillus Calmette-Guérin immunotherapy, a vaccine also used against cancer. Scientists at the University of Pittsburgh engineered this strain with a double break, which is effective and safer after an intravenous administration, according to their results in nonhuman primates and mice.

FDA eyes update to 2014 guidance on sex-specific study considerations

The U.S. FDA posted a draft guidance that would revisit a 2014 final guidance on sex-specific data drawn from medical device clinical trials, expanding the scope to include considerations of gender. However, the draft states that the scientific question addressed by the study may determine whether both sex- and gender-specific data will be collected for the study as opposed to just one of those variables.

The BioWorld Insider podcast: a year of interviews and insights

The BioWorld Insider podcast is a monthly dive into the business and science of drug development. Conversations with CEOs and BioWorld analysts in the past 12 months have included a wide range of chats with experts who are shaping the future. Subjects included quarterly reviews for M&As and financings, the slow but steady growth of funding and partnering for therapies to treat women, the impact of artificial intelligence and quantum computing on drug development, rare disease drug development, the costs of delays in clinical trials, and an overview of the one of the year’s biggest conferences, the annual American Society of Clinical Oncology gathering in Chicago.

2024 BioWorld Year in Review

BioWorld’s staff recaps the therapeutic trends and breakthroughs of 2024, the economic highs and lows facing the industry throughout the past year and the regulatory actions that are likely to have implications as biopharma forges ahead in 2025.

2024 BioWorld MedTech Year in Review

BioWorld MedTech’s staff recaps the med-tech and diagnostic trends and breakthroughs of 2024, the economic highs and lows facing the industry throughout the past year and the regulatory actions that are likely to have implications as med tech forges ahead in 2025.

2024 BioWorld Science Year in Review

Taking a look back at the achievements in research this year, including brain maps, pandemic breakthroughs and advances in pregnancy health.

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Adaptimmune, Arsenalbio, Ashvattha, Banner, Bausch Health, Bicycle, Biomea Fusion, Bristol Myers Squibb, Calcimedica, Calidi, Cycle, Formycon, George Medicines, Immunis, Immunovant, Jiangsu Hengrui, Jeune Aesthetics, Jupiter, Naya, Norgine, Normunity, Ose, Phio, Rappta, Regeneron, Sanofi, Springworks, Telix, Teva, Tune, Verastem, Whitecap, X4