In its second big pharma deal to date, Shape Therapeutics Inc. drew Otsuka Pharmaceutical Co. Ltd. to the table in a gene therapy collaboration that combines the former’s AI-driven AAV platform with the latter’s expertise in ophthalmology. Under the terms, Shape gets an undisclosed up-front payment and is eligible for milestones exceeding $1.5 billion, plus royalties. The agreement comes two years after Shape inked a potential $3 billion-plus deal with Roche Holding AG targeting neurodegenerative diseases.

Twice as nice: With Alexion, Verge cuts another big AI deal

Verge Genomics Inc. has entered a second big AI deal with a large drug company. Verge will receive up to $42 million, including up-front, equity and near-term payments from Alexion, Astrazeneca Rare Disease, to identify multiple targets for rare neurodegenerative and neuromuscular diseases. The deal could top out at $840 million. There also is potential for downstream royalties. Alexion will select potential targets and keeps an option to license and advance targets through the clinic and into commercialization. In June 2021, Eli Lilly and Co. agreed to pay Verge $25 million in up-front, equity and potential near-term payments, plus as much as $694 million in milestones, to discover and validate up to four new targets for treating the disease over the course of a three-year collaboration.

Cymabay to visit regulators with upbeat phase III data in PBC

In the aftermath of Cymabay Therapeutics Inc.’s positive phase III data with seladelpar, the peroxisome proliferator-activated receptor delta agonist in primary biliary cholangitis (PBC), speculation percolated regarding where the drug will fit in the competitive landscape. Newark, Calif.-based Cymabay disclosed the upbeat news Sept. 6 regarding the study called Response in PBC, which met its primary and all key secondary endpoints. The company will be taking the results to regulators globally for advice about next steps. Shares (NASDAQ: CBAY) were trading midday on Sept. 8 at $17, up $1.99, or 13%.

Biopharma financings up from last year with $42.75B raised through August 2023

In August 2023 biopharma companies raised a collective $4.02 billion through 86 transactions, down from the previous seven months’ average of $5.53 billion. The amount raised in August is higher than the $3.93 billion raised in January and $3.74 billion in February and comparable to April’s $4.19 billion. 2023 financings compare most similarly to 2019, when totals averaged $4.82 billion, and 2022, with $5.07 billion monthly averages.

Interim results show Brii’s therapeutic HBV vaccine induces immune response 

Brii Biosciences Ltd.’s therapeutic vaccine for hepatitis B (HBV), BRII-179 (VBI-2601), induced functional immune responses – inducing broad antibody and T-cell responses – in patients with chronic HBV in a phase II trial that evaluated the vaccine in combination with pegylated interferon-alfa, according to interim results. BRII-179 is a recombinant protein-based HBV immunotherapeutic candidate that expresses the pre-S1, pre-S2 and S HBV surface antigens and is designed to induce enhanced B-cell and T-cell immunity. “In this latest study we were looking at interferon-treated patients with hepatitis B and looking at the additive effect of BRII-179 on top of standard-of-care interferon, and what we saw is that when we gave seven doses of the therapeutic vaccine, on top of the interferon, that we had a higher rate of surface antigen loss, a higher rate of antibody production, and a strong correlation between the presence of an antibody and the surface antigen loss,” Brii’s chief medical officer, David Margolis, told BioWorld.

Curocell clears preliminary Kosdaq review, preps November IPO

South Korean CAR T developer Curocell Inc. said on Sept. 8 that it passed a preliminary screening to list on Kosdaq – news that comes four months after its application was made to the Korea exchange on May 2, 2023, and a year after its first failed application in January 2022. “The next steps are to submit a securities report and, upon approval, an IPO in November 2023,” a Curocell official confirmed to BioWorld. In June 2023, when the Daejeon-based firm reported positive interim phase II results of its independently developed CAR T therapy, anbalcabtagene-autoleucel, Curocell announced intentions to press on with the Kosdaq listing with Samsung Securities and Mirae Asset Securities as the joint bookrunners. 

BioWorld Insider Podcast – AI: driving drug development from effective to remarkable

Google’s Scott Penberthy joins the podcast for a visionary discussion that scans the horizon for startling changes artificial intelligence will bring to drug development in the relatively near future. Among the gems and eyebrow raisers is talk of dramatic reductions in the time it takes to identify the right molecule for development and how digital clinical trials in the not-too-distant future will substantially shrink study times. This episode also provides a preview of the annual Biofuture conference. Each year, a group of trailblazers, disruptors and forward-thinking executives converge to evaluate and forecast the future of health care. This year, BioWorld is a gold sponsor of the Oct. 4-6 event in New York. If you attend, you'll have the chance to hear panels and join workshops and fireside chats with key opinion leaders like Penberthy.

Also in the news

4D Molecular, Actimed, Actinium, Adamis, Alector, Alerje, Alnylam, Amneal, Anheart, Arch, Arcutis, Ariceum, Artelo, Carrick, Celularity, Coherus, Corvus, Cymabay, DMK, Durect, Eloxx, Fulcrum, Gilead, Grit, Harmony, Inovio, Intensity, Janssen, Lynch, Maat, Merck, Neuroplast, Palatin, Pepgen, Opus Genetics, Pfizer, Phanes, Promis, Roche, Scisparc, Sorrento, Surface Oncology, Valneva, Vaxart, VBI Vaccines