Industry Update: Regulations, approvals, acquisitions, and capital market and finances
Latest developments compiled from 01 — 31 January 2015
Latest developments in the field of stem cell research and regenerative medicine compiled from publicly available information and press releases from non-academic institutions 01 — 31 January 2015, scheduled to be published in Volume 10 Issue 4 of Regenerative Medicine.
Regulations, approvals, acquisitions…
Acquisitions and mergers
Cardio3 and OnCyte
Cardio3 BioSciences has acquired OnCyte, the oncology division of Celdara Medical, and OnCyte’s portfolio of immuno-oncology product candidates, led by Phase I-bound CM-CS1. The deal could generate up to US$ 182 million for seller Celdara–including up to US$ 50 million if CM-CS1 makes it all the way to market, plus another up to US$ 42 million if the other two T cell products follow CM-CS1 to market. OnCyte’s immune-oncology portfolio includes three autologous CAR T-Cell cell therapy products and an allogeneic T-Cell platform, designed to target a broad range of cancer indications. Cardio3 agreed to pay Celdara US$ 10 million upfront–of which US$ 4 million will be paid in Cardio3 shares–plus up to US$ 50 million in payments tied to development and regulatory milestones for OnCyte’s lead product, the autologous CAR T-Cell drug candidate CM-CS1. CM-CS1 is an autologous CAR T-cell therapy which employs NKG2D, a “natural killer” cell receptor designed to target ligands present on most tumor types, including hematologic cancers as well as solid tumors. Last year, the FDA granted clearance of Celdara’s IND application for a Phase I trial of CM-CS1 in patients with acute myeloid leukemia/advanced myelodysplastic syndrome and multiple myeloma. Cardio3 said the company plans to begin enrolling patients during the first quarter of this year, and expects to complete the study in the second quarter of 2016, with interim data to be reported throughout the enrolment period. In addition, Cardio3 agreed to pay Celdara up to US$ 21 million per product in development and regulatory milestone payments for OnCyte’s two other CAR T-cell programs targeting other cancer cell ligands. Both are in preclinical development. Celdara also stands to gain up to US$ 80 million in sales milestone payments if net sales will exceed US$ 1 billion, as well as royalties ranging from 5 to 8%. Additional information about the clinical trial is available at http://clinicaltrials.gov (ID: NCT02203825).
Capital market and finances
Athersys
Athersys (OH, USA; www.athersys.com) has been awarded a grant from Innovate UK (www.innovateuk.org), formerly the Technology Strategy Board, which will support a Phase 2a clinical study evaluating the administration of MultiStem® cell therapy to acute respiratory distress syndrome (ARDS) patients. The grant is expected to provide up to approximately US$ 3.1 (£2.0) million in support over the course of the study, which will be conducted at leading clinical sites in the UK in conjunction with the Cell Therapy Catapult (UK; https://ct.catapult.org.uk).
CureDuchenne Ventures & Capricor
CureDuchenne Ventures (CA, USA; www.cureduchenne.org/cureduchenne-ventures.html) are investing US$ 1 million in Capricor Therapeutics (CA, USA; www.capricor.com) to advance promising research to treat heart disease associated with Duchenne muscular dystrophy. Capricor has shown positive pre-clinical data for Cardiosphere-derived cells (CDCs) on Duchenne muscular dystrophy cardiomyopathy. CureDuchenne is investing in Capricor to support the conduct of a first clinical trial (Phase I) in patients with Duchenne.
Japan
Japan is attracting companies from around the world that are working to develop regenerative medicine treatments, thanks to a regulatory change that shortens approval times significantly. Based on new laws that took effect in November, regenerative medicine treatments can be approved in as little as two to three years in Japan, compared with the roughly seven years required previously. This is the speediest in the world. In Western markets, approval takes about seven years. Israeli biotech company Pluristem Therapeutics (www.pluristem.com) will be developing in Japan a treatment for weak blood flow to extremities using cells taken from human placenta. Clinical trials will start as early as this year in collaboration with a Japanese partner. British company ReNeuron (www.reneuron.com) is also trying to expand into Japan, with a stroke treatment developed using stem cells. Clinical trials will likely begin as soon as this year. In some cases, new treatments developed here may become available in Japan first, ahead of the drugmaker’s home market. Medipost of South Korea (www.medi-post.com), meanwhile, plans to bring to Japan a treatment for damaged joint cartilage developed from umbilical cord cells. The company already sells the product back home but is now targeting the bigger Japanese market. Japanese companies such as JCR Pharmaceuticals (www.jcrpharm.co.jp/en/), Terumo (www.terumomedical.com) and a FUJIFILM Tissue Engineering unit (www.fujifilm.com/worldwide/) are also readying their own regenerative medicine treatments. The Japanese market for regenerative medicine, which stood at 9 billion yen (US$ 74.1 million) in 2012, is projected to expand to 95 billion yen in 2020 and 1 trillion yen in 2030, according to the industry ministry. The government of Prime Minister Shinzo Abe has positioned regenerative medicine as a pillar of its growth strategy. By attracting foreign companies with high-level skills, Japanese industry could get a technological boost, too.