LONDON – CEOs of five leading pharma companies have hit back against any suggestion COVID-19 vaccines and therapies could be approved in advance of phase III data, saying it is imperative the highest standards of quality, safety and efficacy are upheld everywhere. The comments were made in an online briefing organized by the industry body, International Federation of Pharmaceutical Manufacturers and Associations (IFPMA), to update on progress in developing vaccines and therapies, and warn against any regulatory shortcuts, after recent reports that the FDA is being leant on to approve a vaccine before the U.S. presidential election.

Dueling plasma guidelines a sign of the times

While the emergency use authorization (EUA) the FDA granted last week for convalescent plasma remains a political talking point, the agency moved ahead with the EUA yesterday, issuing immediately effective guidance on the use of convalescent plasma to treat COVID-19 under the EUA, in ongoing clinical trials and on an individual investigational new drug (IND) basis. Just a day earlier, the NIH’s COVID-19 Treatment Guidelines Panel had updated its COVID-19 treatment guidelines, saying there is insufficient data to recommend either for or against the use of convalescent plasma and stressing that it should not be considered standard of care for treating coronavirus infections.

Akebia crashes on safety miss in phase III CKD study with vadadustat

Shares of Cambridge, Mass.-based Akebia Therapeutics Inc. (NASDAQ:AKBA) crashed by 72%, or $7.28, to trade midday at $2.72 on top-line data from the trial called Pro2tect, the second of two cardiovascular (CV) outcomes programs. The two Pro2tect studies evaluated the efficacy and safety of vadadustat, Akebia's oral hypoxia-inducible factor prolyl hydroxylase inhibitor vs. Aranesp (darbepoetin alfa, Amgen Inc.) for the treatment of anemia due to chronic kidney disease in adult patients not on dialysis. Vadadustat achieved the primary and key secondary efficacy endpoint in each of the two Pro2tect studies, demonstrating noninferiority to Aranesp as measured by a mean change in hemoglobin between baseline and the primary evaluation period (weeks 24-36) and secondary evaluation period (weeks 40-52). But the drug missed the primary safety endpoint of the program, defined as noninferiority of vadadustat vs. the comparator in time to first occurrence of major adverse CV events, which is the composite of all-cause mortality, non-fatal myocardial infarction and non-fatal stroke across both experiments.

Rain Therapeutics raises $63M to advance newly licensed liposarcoma asset

Rain Therapeutics Inc., a California company developing targeted cancer therapies with a biomarker-based strategy, has raised a $63 million series B round led by Boxer Capital to support its development of the small-molecule MDM2 inhibitor RAIN-32 (milademetan) for the potential treatment for liposarcoma and other cancers.

Lodo blooms as it acquires Hibiskus

In its second acquisition in the four weeks, Lodo Therapeutics Corp., of New York, has acquired Hibiskus Biopharma Inc. plus exclusive worldwide rights from the University of California Riverside and Michigan State University to preclinical proteasome and immunoproteasome inhibitors developed by Hibiskus’ co-founders. Hibiskus’ lead preclinical molecule is an irreversible and hybrid cyclic peptide proteasome inhibitor from the syrbactin natural product family. Preclinical studies conducted at the National Cancer Institute suggest the small molecule may have utility in treating solid tumors. On Aug. 7, Lodo completed the acquisition of Doylestown, Pa.-based Conifer Point Pharmaceuticals LLC, which has proprietary tools and expertise in computational structural biology and cheminformatics.

Oncolytic virus, CAR T boost each other in solid tumor treatment

Researchers at City of Hope have used a combination of oncolytic virus and CD19-targeting CAR T cells to first force expression of CD19 on tumor cells and then hunt down those cells, eradicating tumors in immunocompetent mouse models and endowing them with immunity to later re-administration of tumor cells. They reported their results in the Sept. 2, 2020, issue of Science Translational Medicine.

Biofourmis raises $100M to expand digital care footprint

Biofourmis Inc. scooped up $10 million in a series C round that was led by Softbank Vision Fund 2. The funds are earmarked to accelerate U.S. and global expansion of Biofourmis’ artificial intelligence (AI)-powered health analytics platform and to advance its pipeline of digital therapeutics. The company focuses software-based therapeutics and AI tools for personal predictive care and has U.S. FDA-cleared products that aim to boost clinicians’ ability to remotely monitor and treat patients. Existing investors Edbi, Massmutual Ventures, Openspace Ventures and Sequoia Capital participated in the financing. With this latest round, Biofourmis has raised a total of $145 million to date.

Also in the news

89bio, Aeon, Akebia, Allergy, Arbor, Astrazeneca, Auris, Biocryst, Bio-Thera, Celecor, Chi-Med, CNS, Enesi, Exact, Glaxosmithkline, Hifibio, Incyte, Innovent, Kite, Lattice, Madrigal, Novan, Novavax, NS Pharma, Oncolix, Pharnext, Redhill, Sanofi, Sol-Gel, Sutro, Taysha Gene Therapies, Tonix, Vistagen, Xencor