Q1 2017 – Advanced Therapies Make a Strong Showing Early in the Year

April 12, 2017


Guest post by Patricia Reilly, M.S., Head of Intelligence Alliances and Unification, Pharma Intelligence and Nancy Dvorin, Executive Editor, In Vivo

Advanced therapy companies started 2017 with a bang. One of the key events in Q1, following the passage of the 21st Century Cures Act, was the announcement that Humacyte received the first Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for its Phase III human acellular vessel HUMACYL.

APAC deals are on the rise. Kite and Shanghai Fosun entered a joint venture to develop KTE-C19 in China. The venture will include profit sharing and an upfront payment of $40 million to Kite, milestones and sales royalties. Additionally, the deal includes an option for several other TCR therapies that could bring the total deal to more than USD 140 million. Molmed announced a  USD 4.5 million agreement with TTY Biopharm for an exclusive license to commercialize Zalmoxis in select Asian territories. Caladrius has agreed to sell the remaining portion of holdings in its PCT subsidiary to Hitachi Chemical for USD 75 million with future potential milestone payments.

CRISPR and Casebia, its joint venture with Bayer AG, signed an agreement with Maxcyte for Maxcyte’s cell engineering platform to develop therapies for hemoglobin-related diseases and severe combined immunodeficiency. Allergan and Editas announced an agreement that would give Allergan the option to license five ocular programs from Editas’s gene-editing platform. Editas, which raised an USD 84.6 million FOPO in March, will receive an upfront payment of USD 90 million for the development of five candidate programs and will be eligible for milestone payments related to their program for Leber Congenital Amaurosis, a rare retinal degenerative childhood disease that leads to blindness.

Financings in a variety of forms continues to fuel this growing sector. Kite closed its USD 409.7 million public offering of common stock. Mustang Bio, a subsidiary of Fortress with a CAR-T therapy in Phase I for glioblastoma, raised USD 94.5 million in a private placement. Kadmon announced private placement equity financing this quarter as well, raising USD 23 million while BioTime and Biostage closed successful public offerings. Regenxbio sold 3.7 million shares of common stock for USD 75.9 million and Adaptimmune raised USD 60.1 million through the sale of 14.3 million ADSs. Cell Medica secured a USD 73.18 million Series C round. The company’s therapy baltaleucel-T is currently in Phase II for advanced lymphomas associated with EBV. Solid Biosciences raised USD 50 million in a Series C financing. Solid will use the financing to continue its focus on gene therapy programs for Duchenne muscular dystrophy. Exonics Therapeutics revealed that it will receive USD 5 million in seed money to move forward with preclinical research in Duchenne muscular dystrophy from CureDuchenne Ventures. Exonics hopes to use CRISPR/Cas 9 technology to identify and correct exon mutations that prevent the production of dystrophin.

Growth in the number of clinical trials added to the positive signs of confidence in this sector.

There were over 800 trials at the end of 2016 with the greatest number in cell therapies currently in Phase II compared to a total of 630 trials in 2015. However, the news was not all good in Q1: Juno’s Phase II Rocket trial for JCAR015 in ALL was closed after multiple holds because of patient deaths from cerebral edema. Juno and its partner Celgene have decided to move forward with several other treatments, including JCAR014 and JCAR017 that target B cell antigen CD19 and are being tested in other blood cancers. Juno is also looking to develop JCAR018, which targets CD22, and JTCR016, a T cell receptor product that targets WT-1.

To hear more on developments in the advanced therapies sector, and to meet and forge partnerships with decision makers from pharma, biotech and finance and patient advocacy groups, attend the Cell & Gene Exchange event taking place in Washington, DC, May 22–23, 2017.

Previous Video
BIO-Europe Spring® 2017: Venneos develops next generation label-free cell analysis
BIO-Europe Spring® 2017: Venneos develops next generation label-free cell analysis

Cell-based assays play a crucial role in drug discovery, toxicity testing and have the potential to contrib...

Next Article
Using Partnerships, Hua Medicine Brings Novel Diabetes Drug to Phase III
Using Partnerships, Hua Medicine Brings Novel Diabetes Drug to Phase III

Li Chen, PhD, co-founded Hua Medicine (Shanghai) six years ago with Ge Li, PhD, founder of WuXi AppTec. The...