Intriguing science: where HIV remains hidden?
Even when patients respond well to antiretroviral therapies and have below detection level of HIV, dosing cannot be stopped. If patients stop the treatment, HIV, hiding in latent infected CD4 T cells in several organs, will rapidly restart multiplying. Finding out these reservoirs is the aim of Last Gift, an initiative by University of San Diego, by conducting a rapid autopsy few hours after death of a volunteer before post-death processes start. The analyses of these samples, including intact DNA and RNA, can unravel the hidden reservoirs and guide science on new therapeutic approaches.
For more information, check out The Last Gift Study.
European Medicine Agency (EMA)
The EMA’s Committee for Pharmacovigilance Risk Assessment Committee (PRAC) held its monthly meeting from September 1 to 4, 2025. Here are the key takeaways:
- PRAC initiated a review of medicines containing levamisole, used to treat parasitic worm infections in adults and children, following concerns about the risk of leukoencephalopathy.
- PRAC endorsed a direct healthcare professional communication (DHPC) warning against the use of polyacrylonitrile(PAN)-based membranes during continuous renal replacement therapy (CRRT) in critically ill patients receiving caspofungin.
- PRAC discussed a DHPC about the risk of severe hypercalcemia in patients treated with Crysvita (burosumab).
- PRAC reviewed a DHPC on a new intravenous formulation of Remsima (infliximab), which must not be administered to patients with hereditary fructose intolerance (HFI) as it contains sorbitol.
- PRAC discussed a DHPC restricting the use of Tegretol (carbamazepine) oral suspension in neonates. PRAC meeting highlights.
US Food and Drug Administration (FDA)
- Vonvendi ([von Willebrand factor (Recombinant)], Takeda) received label expansion to include routine prophylaxis for reducing bleeding episodes in adults with von Willebrand disease (VWD). Takeda press release.
- Intercept Pharmaceuticals announced its decision to voluntarily withdraw Ocaliva (obeticholic acid) from the US market for the treatment of primary biliary cholangitis (PBC) following a request from the FDA. Intercept press release. The FDA has placed on clinical hold all Intercept Pharmaceutical trials involving obeticholic acid.
- Inlexzo (gemcitabine intravesical system, Johnson & Johnson) was approved for the treatment of adult patients with Bacillus Calmette-Guérin (BCG)-unresponsive, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ. J&J press release.
- Koselugo (selumetinib, AstraZeneca) was approved for pediatric patients ≥1 year with neurofibromatosis type 1 (NF1) and symptomatic, inoperable plexiform neurofibromas (PN). FDA press release.
New data from clinical trials (Phase 2, Phase 3)
Here are the recent updates on Phase 2 and Phase 3 clinical trials with published data between September 1 and 14, 2025:
- BioNTech and BMS shared positive data from the Phase 2 trial evaluating pumitamig (BNT327 or BMS986545) plus chemotherapy in patients with extensive-stage small cell lung cancer. BioNTech press release.
- Rapport Therapeutics announced positive data of the Phase 2a open label trial of RAP-219 in patients with drug-resistant focal onset seizures met its primary endpoint. Rapport press release. The company will progress directly to phase 3 trials.
- Sanofi announced that the Phase 3 study of amlitelimab in adults and adolescents with atopic dermatitis met all primary and key secondary endpoints. Sanofi press release.
- Ionis Pharmaceuticals reported positive results from two Phase 3 studies of olezarsen in people with severe hypertriglyceridemia (sHTG). Ionis press release.
- Upstream Bio shared positive results from the Phase 2 trial of verekitug in patients with chronic rhinosinusitis with nasal polyps. Upstream Bio press release.
- AbbVie reported positive results from the Phase 2 trial evaluating the outpatient setting for the first full dose of epcoritamab monotherapy in adult patients with relapsed/refractory (R/R) diffuse large B-cell lymphoma (DLBCL). AbbVie press release.
- AL-S Pharma announced that the Phase 2 trial of AP-101 for amyotrophic lateral sclerosis (ALS) met its primary endpoint. Neurimmune press release.
- Amgen and Kyowa Kirin reported positive results from the Phase 3 study of rocatinlimab in adults and adolescents with moderate to severe atopic dermatitis (AD). Amgen press release.
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