Adverum Biotechnologies Inc. shares (NASDAQ:ADVM) dipped 64 cents, or 20%, to trade midday at $2.42 after an update on the ADVM-022 development program. The company reviewed data available from the phase II Infinity trial in diabetic macular edema (DME) and the phase I Optic experiment in wet age-related macular degeneration (AMD). Data show marked differences in the safety profile between the two patient populations and between the low (2 x 10^11 vg/eye) vs. high (6 x 10^11 vg/eye) doses. Adverum is quitting development for DME after an incident of dose-limiting toxicity – a signal not seen before in ocular gene therapy or anti-VEGF treatment – surfaced at the high dose. But the Redwood City, Calif.-based company aims to evaluate ADVM-022, a single intravitreal injection gene therapy, at low doses and with alternative prophylactic regimens in a new phase II study in wet AMD.
Alzamend posts positive preclinical Alzheimer’s data
Alzamend Neuro Inc.’s positive preclinical data sent the Tampa, Fla.-based company’s stock (NADAQ:ALZN) 72% higher before the market opened, though shares had settled to a far more modest 4% increase by midday. The company is researching AL-002, a mutant-peptide sensitized cell for treating Alzheimer’s disease. The positive data came from a GLP toxicology study using a transgenic mouse model. The cell-based vaccine is designed to restore a patient’s immunological system and reduce beta-amyloid plaque by activating the immune system to stop beta amyloids from forming and from breaking down. The company said AL-002 has the potential to reverse the effects of Alzheimer’s.
Function follows form: Predicted protein structures for human proteome now
Researchers at Google AI company Deepmind and the European Molecular Biology Laboratory/European Bioinformatics Institute (EMBL/EBI) have developed and published an open-access database with predicted structures of 98.5% of proteins in the human proteome.
NHS England tackles pricey gene therapies with £340M fund
NHS England has announced a new £340 million ($481 million) Innovative Medicines Fund (IMF), as the country grapples with the issue of bringing ultra-pricey specialist medicines to patients on the country’s taxpayer-funded health care system. The IMF sits alongside the reformed Cancer Drugs Fund, which also provides up to £340 million each year to provide interim reimbursement for oncology medicines where NICE thinks further data are needed to support a final decision. The IMF is targeted at new medicines, in particular cutting-edge gene therapies, although the finer details of how the fund will work will be consulted upon.
BIO Asia-Taiwan: Cell and gene therapy development changing biopharma ecosystem
Cell and gene therapy have seen much progress in recent times, with the product pipelines in those areas bursting with more than 1,200 therapies. Naturally, the challenges, opportunities and essential development strategies in those fields were the focus on the last day of the BIO Asia-Taiwan Conference 2021.
COVID-19 efforts drive activity, but volume falls for nonprofit deals and grants
While 2020 was clearly a busier year for grants and nonprofit deals with biopharma companies, pandemic efforts continue to drive the activity in these two areas. Through mid-July, there have been 204 grants valued at $1.75 billion, and 516 bio/nonprofit deals worth more than $8 billion, up from $4.66 billion only a month ago.
Also in the news
Abeona, Abivax, Acorda, Acticor, Adverum, Astrazeneca, Azurrx, Biogen, Biontech, Boehringer Ingelheim, Brickell, Capsida, Caribou, Cortexyme, Eisai, Endo, EV Biologics, HCW, Icosavax, Imaginab, Immixbio, Kyowa Kirin, Lonza Cell & Gene Therapy, Moderna, NGM, Norgine, Novartis, Oncimmune, Pfizer, Plexxikon, Rallybio, Rhythm, Ribon, Sigilon, Synlogic, US Worldmeds