Industry Update: Regulations, approvals, acquisitions

Latest business developments compiled from January 1 — February 29 2016

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 from January 1 — February 29 2016, scheduled to be published in Volume 11 Issue 4 of Regenerative Medicine.

Green light

Asterias Biotherapeutics

Asterias Biotherapeutics (CA, US; www.asteriasbiotherapeutics.com) successfully completed an End-of-Phase 2 meeting with the US FDA for AST-VAC1, its investigational therapy targeting acute myeloid leukemia. During the meeting, the FDA indicated general agreement with Asterias’ proposed development plan for registration of AST-VAC1 through a single Phase 3 trial to support an accelerated development pathway and BLA filing. In this study, Asterias will assess the impact of AST-VAC1 compared to placebo on the duration of relapse-free-survival as the primary endpoint, and on overall survival as the secondary endpoint in patients who have achieved complete remission using standard therapies. The proposed trial will include acute myeloid leukemia patients 60 years and older, along with younger individuals who are at high risk for relapse and are not candidates for allogeneic bone marrow transplantation. Pending positive results, this trial could be the basis for accelerated approval of AST-VAC1. Asterias currently plans to submit a request for a Special Protocol Assessment to the FDA to confirm the primary endpoint and other design elements of this pivotal Phase 3 trial.

Asterias has also received Orphan Drug Designation from the US FDA for its product development candidate, AST-OPC1, for the treatment of acute spinal cord injury. Orphan Drug Designation is granted by the FDA Office of Orphan Products Development to products that treat rare diseases. The FDA defines rare diseases as those affecting fewer than 200,000 people in the United States. Orphan Drug Designation may provide the sponsor certain benefits and incentives, including a period of marketing exclusivity for the first marketing application if regulatory approval is received for the designated indication, potential tax credits for certain activities and waiver of certain administrative fees.

Bellicum

Bellicum Pharmaceuticals (TX, US; www.bellicum.com) announced that the US FDA has granted orphan drug designation for BPX-501, the company’s lead product candidate. The FDA designation is for the combination of BPX-501 genetically modified T cells and activator agent rimiducid as “replacement T-cell therapy for the treatment of immunodeficiency and GvHD after allogeneic hematopoietic stem cell transplant.” BPX-501 is an adjunct T-cell therapy incorporating the Company’s proprietary CaspaCIDe safety switch. Following an allogeneic transplant, a lack of sufficient mature T cells constitutes immune deficiency that can contribute to infections, viral reactivation, and relapse. The ability to correct this immune deficiency through the add-back of mature donor T cells, without raising the risk of uncontrollable GvHD, has the potential to fundamentally change the risk profile of allogeneic transplantation.

Lion Biotechnologies

Lion Biotechnologies, Inc. (NY, US; www.lbio.com) announced that the US Food and Drug Administration (FDA) has allowed its IND application to conduct clinical trials of LN-145 in the treatment of cervical cancer and head and neck squamous cell carcinoma. Each of the single-arm studies will be conducted at up to six sites in a total of 10 patients with either recurrent or metastatic carcinoma. The objectives of the studies are to assess safety, tolerability and various indicators of efficacy. Lion Biotechnologies is a clinical-stage biotechnology company focused on the development of cancer immunotherapy products for the treatment of various cancers. The company’s lead product candidate is an adoptive cell therapy using tumor-infiltrating lymphocytes for the treatment of patients with refractory metastatic melanoma, and is based on a clinical Cooperative Research and Development Agreement with the National Cancer Institute. TIL therapy is also being evaluated in physician-sponsored clinical trials at MD Anderson Cancer Center and Moffitt Cancer Center.

Pending

Amarantus

Amarantus (VA, US; www.amarantus.com) filed for rare pediatric disease and orphan drug classifications for its engineered skin substitute (ESS). The ESS graft, developed by Amarantus BioScience, would be used to treat the rare skin disease giant congenital melanocytic nevi, which causes dark patches in infants and is linked to an increased chance of melanoma. If approved, the designations would place ESS at an advantage for a priority review voucher, as well as a seven-year marketing exclusivity period and tax credits.

Acquisitions

MiMedx Group and Stability

MiMedx Group (GA, US; www.mimedx.com) has signed a definitive agreement to acquire Stability Stability Biologics (TN, USA; www.stabilitybio.com), a provider of human tissue products to surgeons, facilities and distributors serving the surgical, spine and orthopedics sectors of the healthcare industry. Stability Biologics is privately held and was founded in 2010. MiMedx expects the transaction to be accretive to Full Year Adjusted EPS in 2016, and it projects the 2016 Stability Biologics revenues generated from its processed tissue and bone product sales and distribution to be at least US$ 15 million. Following the transaction, Stability Biologics will operate as a wholly-owned subsidiary of MiMedx.

OSE Pharma and Effimune

OSE Pharma (France, www.osepharma.com) and Effimune (France, www.effimune.com) signed a merger agreement. The Boards of Directors of both OSE Pharma and Effimune have approved the proposed terms of the merger, which will be submitted for approval to the shareholders of the two companies during Extraordinary General Meetings that are expected to take place at the end of the second quarter of 2016. The merger will result in the shareholders of OSE Pharma owning approximately 71% of the capital of the merged entity and the shareholders of Effimune, 29%. The objective of the merger is to create a new international enterprise that offers innovative immunotherapies based on the activation or regulation of the immune system.

Name change

Cell Therapy Catapult

The Cell Therapy Catapult (UK; www.ct.catapult.org.uk) officially changed its name to the Cell and Gene Therapy Catapult. The new name more accurately reflects the activities of the organization, the boom in gene modified cell therapies, and the wave of direct gene therapies currently in development.