Industry Update: Capital market and finances

Latest business developments compiled from October 01 — November 30 2015

Latest business developments in the field of stem cell research and
regenerative medicine compiled from publicly available information and
press releases from non-academic institutions October 01 — November 30 2015, scheduled to be published in Volume 11 Issue 2 of Regenerative Medicine.

Avita

Avita Medical Ltd. (Australia; www.avitamedical.com) announced the closing of a common stock placement to sophisticated and institutional investors through the issue of 107,727,359 shares at a price of US$ 0.066 (AU$ 0.093) per share. The company said the placement is part of its funding strategy to support its stated commercialization goals under a broader placement approval from shareholders. This US$ 7 (AU$ 10) million placement has been priced at a 20% discount to the 5-day volume weighted average price.

Cellectis, Servier and Pfizer

Following successful demonstration of their CAR T-cell technology UCART19, Cellectis (France; www.cellectis/com) announced that Servier (France; www.servier.com) had exercised an option to acquire the exclusive worldwide rights to UCART19, which is about to enter initial Phase I clinical tests, and Pfizer (NY, USA; www.pfizer.com) would work with Servier on the drug’s development. Pfizer will have rights to sell the treatment in the U.S., with Servier responsible for marketing elsewhere. The tie-up is separate from Pfizer’s collaboration with Cellectis announced in 2014, which did not include UCART19. Cellectis will get US$ 38.2 million upfront from Servier and is eligible for over US$ 300 million in milestone payments, research financing and royalties on sales. Financial terms for the Servier agreement with Pfizer were not disclosed. UCART19 is being tested for chronic lymphocytic leukaemia and acute lymphoblastic leukaemia. Cellectis is developing CAR-T immunotherapies using engineered cells from a single donor for use in multiple patients.

Emulate

Emulate, Inc. (MA, USA; www.emulatebio.com) has been awarded a research grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF; NY, USA; www.michaeljfox.org) to apply the company’s Organs-on-Chips to investigate the potential safety liabilities observed in leucine-rich repeat kinase 2 (LRRK2) inhibitors, a class of drug candidates for the treatment of Parkinson’s disease.

Mesoblast

Mesoblast (Australia; www.mesoblast.com) was listed on the Nasdaq Global Market on 13 November under the symbol ‘MESO’. Mesoblast expects to receive gross proceeds of approximately AU$ 95.8 million (US$ 68.3 million), before deduction of underwriting discounts and commissions and expenses, for the public offering of 8,535,059 American Depositary Shares (ADSs), which includes the underwriters partial exercise of their over-allotment option. This issue represents approximately 12.6% of Mesoblast’s issued share capital. The ADSs were issued at a public offering price of US$ 8.00 per ADS. Mesoblast intends to use the net proceeds of this offering for ongoing clinical programs, research and development

Novartis and Gamida Cell

Novartis (Switzerland; www.novartis.com) will invest up to an extra US$ 15 million in Gamida Cell (Israel; www.gamida-cell.com). Novartis last year invested US$ 35 million in the company for a 15 percent stake, in a deal that could reach US$ 600 million if Novartis exercises a buyout option that expires in 2016. The US$15 million investment will be used to advance Gamida Cell’s clinical programs, including the development of NiCord, an experimental treatment for patients with high risk hematological malignancies, or blood cancers such as leukemia and lymphoma and sickle cell disease. Gamida plans to initiate a Phase III clinical trial with NiCord in mid-2016. Novartis will not have rights or options to Gamida Cell products or technology under the terms of the agreement.

Regeneus

Regeneus Ltd (Australia; www.regeneus.com.au) has received a US$ 2.4 (AU$ 3.4) million from the Australian government to progress its regenerative medicine product pipeline, including the Progenza allogeneic stem cell treatment for knee osteoarthritis. Progenza is produced from MSC derived from the adipose tissue of a healthy donor who has been extensively screened. The clinical-stage regenerative medicine company received the funds as part of the government’s research and development tax incentive program for activities conducted during FY2015. Regeneus anticipates that it will be eligible to receive reimbursement for qualifying expenditure in its R&D programs during FY2016. The financial support will underpin product development work, which has recently included the treatment of the first patient under Phase I clinical trials of Progenza.

Stratatech

Stratatech (WI, USA; www.stratatechcorp.com) has been awarded a contract valued at up to US$ 247 million by the Biomedical Advanced Research and development Authority (BARDA; www.phe.gov/about/BARDA/Pages/default.aspx), part of the U.S. Department of Health and Human Services’ Office of the Assistant Secretary for Preparedness and Response. The contract is for the advanced development of StrataGraft® skin tissue, the Company’s flagship skin replacement product, as a medical countermeasure to treat patients with severe thermal burns. The planned clinical studies will enable Stratatech to file a Biologics License Application with the FDA for the commercial marketing of StrataGraft tissue, and confirm efficacy in all key patient populations. The contract also provides for the procurement of finished product by the U.S. government so as to establish an inventory of StrataGraft tissue for use in case of a natural or manmade mass casualty emergency.