Belumosudil Mesilate

Orca Bio has announced that the US FDA extended the review timeline for its Biologics License Application (BLA) for Orca-T, an investigational allogeneic T-cell immunotherapy targeting hematologic malignancies including acute leukemias and myelodysplastic syndromes. The revised PDUFA target action date is July 6, 2026, three months beyond the original deadline, following the FDA’s classification of a recent company submission as a Major Amendment to the BLA. The extension was triggered by Orca Bio’s submission of updated chemistry, manufacturing and controls (CMC) information provided in response to FDA requests during the standard review process, the company said. The agency has not requested additional clinical data, and Orca Bio stated it believes the updated CMC information does not affect the benefit-risk conclusions of the BLA. The filing carries Priority Review designation, and Orca-T has previously received Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug Designation for the prevention of graft-versus-host disease (GVHD) or death in patients eligible for hematopoietic stem cell transplant (HSCT). Orca-T is composed of highly purified hematopoietic stem cells, regulatory T-cells, and conventional T-cells derived from matched related or unrelated donors. The inclusion of regulatory T-cells is central to the product’s design rationale: by modulating the post-transplant alloreactive immune response, the approach aims to reduce GVHD without eliminating the graft-versus-leukemia effect that makes allogeneic transplantation therapeutically relevant. This positions Orca-T not as an adjunctive drug but as a redefined graft — intended to replace conventional unmanipulated cell products at the transplant step itself. The clinical program supporting the BLA includes the Phase III Precision-T trial (NCT05316701), which enrolled patients with hematologic malignancies and used moderate or severe chronic GVHD-free survival at 12 months as its primary endpoint, assessed by an independent Endpoint Adjudication Committee. An earlier Phase Ib study (NCT04013685) focused on safety endpoints including the incidence of grade 3 or 4 acute GVHD and primary graft failure. The filing enters review at a time when GVHD remains a substantial source of morbidity and non-relapse mortality in the allogeneic transplant setting. Approved prophylactic agents include abatacept, which received FDA approval in 2021 for use in unrelated-donor transplants, while treatment options for established GVHD include ruxolitinib, belumosudil, and axatilimab, the latter approved in 2024 for recurrent or refractory chronic GVHD. Despite these additions, no approved intervention has demonstrated the capacity to simultaneously reduce GVHD and preserve the graft-versus-leukemia effect — the core biological tension that precision graft engineering approaches such as Orca-T are designed to address. Your email address will not be published. Required fields are marked * Comment * Name * Email * Website

Novo cutting 400 jobs in IndianaAlltrna's first-in-human study of tRNA candidate will take place in AustraliaAstellas lets AviadoBio gene therapy option lapseSanofi's Rezurock bags conditional EU nod for chronic GvHD Novo cutting 400 jobs in IndianaNovo Nordisk confirmed to FirstWord Tuesday that it will reduce its workforce in Bloomington, Indiana by approximately 400 positions. Impacted staff will be notified in early May.The cuts primarily affect manufacturing roles, but extend across multiple functions, according to a company spokesperson. The layoffs will leave about 1400 employees at the site.The Bloomington site is "a critical part of our US manufacturing network" and the Danish drugmaker continues to invest there, the spokesperson said, adding the downsizing "reflects the business realities we face in today's competitive environment."The facility "requires a focused, right-sized team rather than the workforce size inherited when we acquired this site," the spokesperson explained. "We're aligning our staffing accordingly."In September, Novo said it would shrink its global workforce by 9000 positions, most in its home base of Denmark, as it looks to better respond to "a more dynamic and consumer-driven obesity market."-Anna BratulicAlltrna's first-in-human study of tRNA candidate will take place in AustraliaAlltrna said Tuesday that Australian regulators have cleared it to begin a Phase I trial of AP003 in healthy volunteers, marking the first therapeutic based on transfer RNA (tRNA) to enter the clinic. The trial, which will evaluate the safety and pharmacokinetics of single ascending doses of AP003 and inform future development, "begins to establish the clinical foundation to evaluate a single engineered tRNA across diseases that share the same premature termination codon," said Alltrna Chief Medical Officer Nerissa Kreher.In development for patients with liver Stop Codon Disease who carry an arginine-to-TGA (Arg-TGA) nonsense mutation, AP003 is a chemically modified, engineered tRNA therapy encapsulated within a liver-directed nanoparticle. The experimental treatment is designed to restore full length protein production by inserting the correct amino acid at an Arg-TGA premature termination codon.The study's impending launch comes nearly three years after the biotech raised $109 million in a series B to develop its tRNA-based machine learning platform (see – ViewPoints: How Alltrna is leveraging tRNA biology to address genetic diseases).-Elizabeth EatonAstellas lets AviadoBio gene therapy option lapseAstellas has stepped back from its deal with AviadoBio, allowing its option to license the AVB-101 gene therapy expire and returning full global rights to the biotech. The move unwinds a 2024 agreement that carried nearly $2.2 billion in potential milestones tied to the programme for frontotemporal dementia with progranulin mutations, though Astellas remains a shareholder in the UK company.AviadoBio said it continues to advance AVB-101 in the clinic, recently initiating a fourth dose-escalation cohort in the Phase I/II ASPIRE-FTD trial.The study has so far tested AVB-101 in 12 patients across the first three dose levels. Early data have shown dose-dependent increases in levels of cerebrospinal fluid progranulin alongside a clean safety profile, with no need for prophylactic or reactive immunosuppression, and no serious adverse events reported to date, according to the company.The move follows a similar retreat in October, when Astellas passed on licensing Taysha Gene Therapies' TSHA-102 for Rett syndrome before its pivotal trial. Meanwhile, an earlier gene therapy effort that stemmed from its purchase of Audentes Therapeutics was plagued with setbacks.-Anna BratulicSanofi's Rezurock bags conditional EU nod for chronic GvHDThe European Commission granted a conditional marketing authorisation for Sanofi's Rezurock (belumosudil) in chronic graft-versus-host disease (GvHD), clearing the selective ROCK2 inhibitor to treat adults and adolescents aged 12 and older when previous therapies have failed or are unsuitable.Having issued a negative opinion last October, the EMA's Committee for Medicinal Products for Human Use (CHMP) reached a positive conclusion in January after Sanofi requested a re-examination. At the time, Sanofi noted that it committed to conducting a new post-approval confirmatory study.Rezurock — gained through Sanofi's acquisition of Kadmon Pharmaceuticals — was first approved by the FDA in 2021. The drug is also being investigated in paediatric chronic GvHD and chronic lung allograft dysfunction.-Pavan Kamat