NEW YORK – The US Food and Drug Administration this week granted breakthrough therapy designation for Bayer's HER2 and EGFR inhibitor BAY 2927088 for previously treated patients with unresectable or metastatic non-small cell lung cancer whose tumors have activating HER2 (ERBB2) mutations. The designation was based on preliminary results from a Phase I trial in NSCLC patients harboring mutations in HER2 or EGFR. The FDA grants breakthrough therapy designation for treatments that preliminary clinical evidence indicates may demonstrate substantial improvement over available therapy on a clinically significant endpoint. Breakthrough therapy designation allows the sponsor to meet with the FDA more frequently for guidance on the drug development program along with the ability to submit data on a rolling basis and apply for accelerated approval or priority review.
Bethesda, Maryland-based Gain Therapeutics this week said it has started the multiple ascending dose portion of its Phase I study of GT-02287, the company's lead drug candidate, which is being studied as a treatment for slowing or halting the progression of Parkinson's disease associated with GBA1 gene mutations. A human research ethics committee in Australia approved the start of this part of the study in parallel with the completion of the last single ascending dose cohorts based on safety and tolerability data observed so far.
ImmPact Bio USA this week said it has been awarded an $8 million grant from the California Institute for Regenerative Medicine to support the company's ongoing open-label Phase Ib/II trial evaluating its autologous, bispecific CD19/CD20 CAR T-cell therapy IMPT-514 as a treatment for refractory lupus nephritis and systemic lupus erythematosus. ImmPact Bio expects to share initial efficacy and safety data from the dose-escalation trial in the second half of the year.
Taysha Gene Therapies this week said the adolescent and adult Phase I/II REVEAL trial of TSHA-102, the company's Rett syndrome gene therapy candidate, will proceed into the high-dose cohort earlier than expected, and the firm anticipates dosing the first patient in Q2. It will also expand the trial, currently launched in Canada, to the US. Taysha also plans to dose the second patient in the low-dose cohort of the Phase I/II trial in pediatric patients.
Columbus, Ohio-based Andelyn Biosciences this week announced that it has been selected as a viral vector manufacturing partner for the Bespoke Gene Therapy Consortium, a public-private project launched by the Foundation for the National Institutes of Health to develop resources to support gene therapy development and shepherd eight rare disease gene therapies through first-in-human trials. Andelyn, a cell and gene therapy contract development and manufacturing organization, will manufacture adeno-associated vectors for two therapies being developed for CNGB1 retinitis pigmentosa 45 and NPHP5 retinal degeneration.
Viracta Therapeutics said this week that it has finished the second stage of enrollment into one of the cohorts of its Phase II NAVAL-1 clinical trial, through which the firm is studying its investigational treatment regimen, Nana-val (nanatinostat plus valganciclovir), in certain lymphoma patients. The cohort includes patients with relapsed or refractory Epstein-Barr virus (EBV)-positive peripheral T-cell lymphoma. The firm said it plans to report top-line data for this cohort during the second quarter of 2024 and to meet with the US Food and Drug Administration in mid-2024 to discuss requirements for seeking accelerated approval for Nana-val.
The Council of the European Union last week approved the extension of certain deadlines of the In Vitro Diagnostic Regulation. It also approved moving up the date when diagnostic test developers would need to supply data about their tests to the Eudamed medical database. In January, the European Commission proposed giving companies more time to apply the IVDR under certain circumstances after finding a "considerable number" of IVDs on the EU market don't meet the new rules, which began rolling out in 2022. In particular, the situation is "critical" for high-risk IVDs, the EC said. The deadlines to comply with the IVDR were May 26, 2025, for high-risk IVDs and May 26, 2027, for lower-risk IVDs. The new proposal pushes those deadlines to December 2027 for high individual and public health risk devices, December 2028 for high individual and moderate public health risk devices, and December 2029 for lower-risk devices. The proposal also moves up the deadline by which test and device manufacturers will need to upload data about their devices to Eudamed to late 2025 from 2026. The European Parliament is expected to vote on the Commission's plan in April.
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.