NEW YORK – Australia's Therapeutic Goods Administration this week confirmed its decision not to register Eisai and Biogen's anti-amyloid Alzheimer's disease drug Leqembi (lecanemab), reaffirming a decision from last year, after which Eisai had requested it reconsider the decision in December 2024. However, the TGA confirmed its initial verdict that the demonstrated efficacy of the drug did not outweigh the safety risks associated with it, such as the risk of amyloid-related imaging abnormalities (ARIA), particularly in patients with certain APOE variants, including APOE4 heterozygotes.
The TGA said it proposed limiting the drug to APOE4 noncarriers, in which it felt safety and efficacy were satisfactorily established, but Eisai was not willing to seek that indication. Eisai this week said it had proposed maintaining an indication for APOE4 noncarriers and heterozygotes, while suggesting that APOE4 heterozygotes should be treated in specialist centers and supervised by physicians with expertise in monitoring ARIA. The TGA did not agree with this proposal. Eisai said it "remains committed" to ensuring access to Leqembi in Australia and is exploring options to achieve this, potentially including seeking review by an administrative review tribunal.
Health Canada has issued conditional marketing authorization for Biogen's Qalsody (tofersen), which previously has been approved in the US and EU, as a treatment for amyotrophic lateral sclerosis (ALS) with a mutation in the SOD1 gene, the company said this week, making Qalsody the first treatment targeting a genetic form of ALS to be approved in Canada. The authorization is conditional and pends results from clinical trials to verify the drug's clinical benefit.
China's National Medical Products Administration (NMPA) this week granted breakthrough therapy designation to Carsgen Therapeutics' autologous CAR T-cell therapy satricabtagene autoleucel (satri-cel) for the treatment of Claudin 18.2 (CLDN 18.2)-positive advanced gastric and gastroesophageal junction cancer patients who have failed at least two prior lines of therapy. Carsgen expects to submit a new drug application seeking approval of satri-cel in this setting to the NMPA in the first half of 2025.
VolitionRx this announced that its Nu.Q Discover epigenetic profiling assays would be used to measure disease progression and treatment response in a longitudinal PhaseI/IIb study sponsored by “a leading pharmaceutical company.” The company said that its assays were selected following a successful pilot study. The firm expects this study to generate “significant revenue” and is discussing further projects.
The European Commission has provided a positive opinion from the European Medicines Agency's Committee for Advanced Therapies for Ocugen's OCU410 and OCU410ST to receive advanced therapy medicinal product (ATMP) classification, which can accelerate the regulatory review timelines of certain advanced treatments, the Malvern, Pennsylvania-based company said this week. Ocugen is developing OCU410 as a gene therapy for vision loss due to geographic atrophy, with plans to file for regulatory approvals in 2028, while it's developing OCU410ST for Stargardt disease due to ABCA4-related retinopathies, with plans to file for regulatory approvals in 2027.
Rockville, Maryland-based Regenxbio this week said it has closed a deal for a strategic partnership with Japanese pharmaceutical company Nippon Shinyaku, which it announced in January and said was valued at $810 million, under which the firms will develop and commercialize gene therapies for mucopolysaccharidosis diseases. The companies will advance Regenxbio's RGX-111 for Hurler syndrome and RGX-121 for Hunter syndrome, which Regenxbio said could potentially garner market approval in the US as early as later this year.
Guardant Health said this week that it has entered a deal with Dak Prescott’s Faith Fight Finish Foundation and Feist-Weiller Cancer Center’s Partners in Wellness to expand access to the company's Shield blood-based colorectal cancer screening test. The foundation has provided a grant that will allow Partners in Wellness to offer Guardant's test as part of its mobile cancer screening program to patients it serves across more than 30 communities in northern and central Louisiana, regardless of their ability to pay. Details about the funding amount were not disclosed.
Avacta Therapeutics announced this week that it has sold Launch Diagnostics and its subsidiaries for £12.9 million ($16.7 million) in cash to Duomed Belgium, a subsidiary of Palex Healthcare Group. Completion of the sale is expected by the end of April 2025 and proceeds will be used to further Avacta's PreCision platform. After the divestment, Avecta's cash runway will extend into Q1 2026.
Avacta purchased the diagnostics company in October 2022 for up to £37 million. It is also in discussions to sell its remaining and much smaller Coris BioConcept diagnostics unit.
Avacta also said this week that initial proof-of-concept data for its AVA6000 clinical stage asset – a PreCision-enabled form of doxorubicin – have demonstrated the potential of its platform. According to Avacta, AVA6000 has shown a significant reduction in the observed toxicities associated with conventional doxorubicin, the capacity to shrink tumors with multiple, durable responses observed in different disease settings with sensitivity to doxorubicin, and the profound concentration of the active payload in the tumor versus the plasma.
Its AVA6103 program is a PreCision-enabled peptide drug conjugate that combines the PreCision peptide linked to exatecan. The program has completed candidate selection and is in the preparation stage for the Phase I trial, which is expected to begin early in 2026.
Avacta noted that it continues to explore the possibility of attaining a dual listing on Nasdaq.
In Brief This Week is a selection of news items that may be of interest to our readers but had not previously appeared on Precision Medicine Online.