Belite Bio Inc. unveiled positive top-line data from the 104-subject phase III Dragon study with tinlarebant in Stargardt disease type 1 (STGD1). The trial met its primary efficacy endpoint, turning up a statistically significant and clinically meaningful 36% reduction in the growth rate of retinal lesions vs. placebo, measured as definitely decreased autofluorescence by fundus autofluorescence imaging. STGD1 leads to progressive vision loss, usually starting in childhood or young adulthood. No treatment has been approved. Tinlarebant works by reducing and maintaining levels of serum retinol binding protein 4, the sole carrier protein for retinol transport from the liver to the eye. Shares of the San Diego-based firm (NASDAQ:BLTE) were trading at $144.64, up $7.20.

China approves Innovent’s picankibart as first domestic IL-23p19

China’s National Medical Products Administration (NMPA) gave the nod to Innovent Biologics Inc.’s picankibart (IBI-112), branded as Pecondle, for treating moderate to severe plaque psoriasis in adults who are candidates for systemic therapy. A recombinant anti-interleukin-23p19 subunit (IL-23p19) monoclonal antibody, picankibart is the first domestic IL-23p19 to be approved in China. It specifically targets the IL-23p19 subunit, preventing IL-23 from binding to cell surface receptors. The approval comes more than a year after the NMPA accepted Innovent’s NDA in October 2024.

Q32 sells rare kidney disease drug to Akebia for $12M up front

Shares of Q32 Bio Inc. (NASDAQ:QTTB) were trading up by more than 100% at midday on news that the firm has extended its cash runway by selling its phase II-stage complement inhibitor, ADX-097, to Akebia Therapeutics Inc. for $12 million in up-front and near-term payments, with as much as $592 million to come in development, regulatory and commercial milestones. Q32 now has sufficient cash to sustain operations into the second half of 2027, well beyond the expected top-line readout from the phase IIa trial testing lead candidate bempikibart in alopecia areata. For Akebia, the deal continues its efforts to build a rare kidney disease pipeline, which also includes praliciguat, an oral soluble guanylate cyclase candidate licensed from Cyclerion Therapeutics Inc. in 2021.

Protego secures $130M in series B for its AL amyloidosis program

Protego Biopharma Inc. closed an oversubscribed $130 million series B financing that will be used to advance PROT-001, the company’s treatment for amyloid light (AL) chain amyloidosis. The drug, which works by stabilizing immunoglobulin light chains to prevent amyloid buildup, is currently being tested in a phase I study with data expected in the early part of next year. Assuming the safety, pharmacokinetics and pharmacodynamics in healthy volunteers look favorable, the capital will allow the San Diego-based company to start a pivotal phase II study in patients with AL amyloidosis the latter part of next year.

HS/CSU space enlivened by Inflarx findings

Jena, Germany-based Inflarx NV’s recent positive data from a phase IIa basket study exploring INF-904 in both indications highlighted efforts across the board in hidradenitis suppurativa (HS) and chronic spontaneous urticaria (CSU). Efficacy data were reported from 29 of 31 HS patients and from 30 of 31 subjects with CSU. The Inflarx experiment is continuing toward the finish of the four-week post-treatment observation period, with the final outcomes due to roll out at scientific meetings. Among HS players is Moonlake Therapeutics AG, which turned in results from two identical phase III trials – Vela-1 and Vela-2 – of sonelokimab in September that were plagued by what the company called “placebo wobble.”

HIV remission after heterozygous CCR5Δ32 stem cell transplant

2025 has been the most challenging year in the efforts to fight HIV since at least the advent of antiretroviral therapy. In a report on “Overcoming disruption, transforming the AIDS response,” released last week ahead of World AIDS Day on Dec. 1, the Joint United Nations Program on HIV/AIDS described “a global system in shock” by sharply reduced funding from the U.S. and other wealthy nations. According to the report, “although the second part of 2025 has provided welcome signs of continued commitment from international donors to ending AIDS as a public health threat, including the United States of America, it is clear that the era of steady, year-on-year support through international HIV assistance is over,” and that this new era could see more than 3 million excess new HIV infections by 2030.

Also in the news

Adial, Anges, Ascendis, Astrazeneca, Beone, Biomx, Biosidus, Celltrion, CG Oncology, Mural, Neurizon, Novo Nordisk, Otsuka, Pfizer, Prestige, Renalys, RK, Roche, Scilex, Sun, Valneva, Vasomune, Vistagen, Xoma, Zydus