NEW YORK – The US Food and Drug Administration this week granted breakthrough therapy designation to AstraZeneca and Daiichi Sankyo's Enhertu (trastuzumab deruxtecan) for the treatment of unresectable or metastatic hormone receptor (HR)-positive HER2-low or HER2-ultralow breast cancer. Low HER2 expressed was defined as IHC staining result of 1+ or 2+ with negative results for HER2 gene amplification by in situ hybridization. HER2 ultralow was defined as an IHC result greater than 0 and less than 1. The FDA granted breakthrough therapy designation based on results presented earlier this year from the Phase III DESTINY-Breast06 trial, which showed survival outcomes were similar between HER2-low and HER2-ultralow patient groups. 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.
The European Medicines Agency this week validated Bristol Myers Squibb's Type II variation application to expand the indication of CAR T-cell therapy Breyanzi (lisocabtagene maraleucel) to include treatment of adult patients with relapsed or refractory follicular lymphoma who have received two or more prior lines of systemic therapy. Validation of the application by the EMA confirms the submission is complete and marks the start of the review process. The application was based on data from the Phase II TRANSCEND FL study, which demonstrated a 97 percent response rate to Breyanzi in the third-line setting for relapsed or refractory follicular lymphoma patients. BMS has also submitted applications in the US and Japan seeking approval in the same follicular lymphoma indication.
Research Triangle Park, North Carolina-based Opus Genetics this week said it has received rare pediatric disease designation from the US Food and Drug Administration for OPGx-LCA5, a gene therapy it's developing for Leber congenital amaurosis type 5, an inherited disease and early-onset form of retinal degeneration. The company is evaluating the safety and preliminary efficacy of the gene therapy in an ongoing open-label Phase I/II trial.
Mindera Health this week said it has completed the credentialing process to become a provider under Medi-Cal, California's Medicaid program, so that its laboratory test to personalize prescribing for psoriasis biologics can be made available to Medicaid beneficiaries in the state. The company's transcriptomics test, Mind.Px, is designed to predict which class of biologic a patient is likely to respond to based on an analysis of mRNA extracted from the skin.
Accenture, through its Accenture Ventures arm, has made a strategic investment for an undisclosed amount in Earli, a biotechnology company developing a synthetic biology-based early cancer detection technology. Earli's technology uses genetic constructs that switch on only in cancer cells and force them to produce any signal protein of choice. The proteins are then used for diagnostic imaging or activating the immune system against the cancer, according to the company's website. Earli, based in Redwood City, California, will use the new funding to expand its collaborations with global health and pharma companies. Accenture Ventures said this week that the company is also joining Project Spotlight, its engagement and investment program for companies developing disruptive technologies.
The Maritime Newborn Screening program in Canada's Nova Scotia province this week expanded to include genetic testing for spinal muscular atrophy, a progressive neuromuscular disorder caused by mutations in the SMN1 gene. By screening for the disorder at birth, the government hopes infants diagnosed with spinal muscular atrophy will be able to receive treatment and care management earlier. The Maritime Newborn Screening program now tests for two dozen conditions.
iHope Genetic Health said this week that it has selected the first clinical sites participating in its initiative to increase genomic testing access globally. The program, run by the Genetic Alliance and funded by Illumina, provides free genomic testing, including clinical exome and genome sequencing, to underserved children with suspected genetic disorders. Testing will be provided through the iHope Genetic Health laboratory network, currently consisting of three labs. The program expects to provide testing to hundreds of patients this year and thousands in the next two years. The clinical sites include Child and Youth Care Zimbabwe; Hospital Edgardo Reba gliati Martins-ESSALUD, Instituto Nacional de Ciencias Neurológicas, and Instituto Nacional de Salud del Niño in Peru; Padrino Children's Foundation in Mexico; the Rare Disease Ghana Initiative; Al Jalila Children's Specialty Hospital in the United Arab Emirates; Children's Hospital of Philadelphia; and Mendel – Centre for Genetic Education in the Republic of North Macedonia. The plan is to expand the network to at least 20 clinical sites and six laboratories in 2025, iHope said.
Burning Rock Biotech this week reported a 7 percent decline year over year in its second quarter revenues to RMB 135.5 million ($19.0 million) RMB 146.3 million. For the three months ended June 30, its central laboratory channel revenues dropped to RMB 48.8 million from RMB 66.2 million, in-hospital channel revenues rose to RMB 59.9 million from RMB 53.8 million, and pharma research and development channel revenues grew to RMB 26.9 million from RMB 26.2 million. The Guangzhou, China-based company had a net loss of RMB 108.0 million for Q2 2024 compared to a net loss of RMB 131.2 million a year ago. For the recently completed quarter, Burning Rock said its Class A ordinary shares had a net loss of RMB 1.05 per share compared to a net loss of RMB 1.28 a year ago. Its Class B ordinary shares had a net loss of RMB 1.05 per share in Q2 2024 compared to a net loss of RMB 1.28 per share a year ago.
Its R&D costs retreated to RMB 65.0 million in Q2 2024 from RMB 95.8 million a year ago, while its SG&A costs rose to RMB 141.7 million from RMB 140.4 million. The firm had RMB 532.5 million in cash and cash equivalent at the end of the recently completed quarter and RMB 505,000 in restricted cash.
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.