Industry Update: Regulations, approvals, acquisitions

Written by Dusko ILIC

Latest business developments compiled from 1—31 December, 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 1—31 December 2015, scheduled to be published in Volume 11 Issue 3 of Regenerative Medicine.

Green light

Pluristem

Pluristem Therapeutics (Israel; www.pluristem.com) announced that the U.S. FDA has granted the Company’s PLX-PAD cells Orphan Drug Designation in the treatment of severe preeclampsia. Preeclampsia is among the most common medical complications of pregnancy and a leading cause of premature births, stillbirths and neonatal and maternal deaths. It is estimated by different sources that preeclampsia costs the global health care system about $3 billion annually. PLX-PAD cells improved several parameters of preeclampsia in animal models.

Pluristem also has reached an agreement with Japan’s Pharmaceuticals and Medical Devices Agency (PMDA; http://www.pmda.go.jp/english/) on the design of the final trial needed to apply for conditional approval of PLX-PAD cells in the treatment of critical limb ischemia (CLI). The approval of the protocol for the 75-patient trial was part of a larger agreement on the development of PLX-PAD via Japan’s new accelerated regulatory pathway for regenerative medicine. Pluristem expects to submit the formal Clinical Trial Notification (CTN) to the PDMA, based on the agreement reached with the regulatory body, in early 2016. The PMDA is expected to respond officially within 30 days. Earlier in 2015, the PMDA cleared PLX-PAD cells for use in clinical studies in Japan, a prerequisite to conducting this clinical trial.

Pending

BioLineRx

BioLineRx (Israel; www.biolinerx.com) filed the regulatory submissions required to commence a Phase 2 trial for BL-8040, as a novel approach for mobilization and collection of bone marrow stem cells from the peripheral blood circulation. The trial is expected to commence shortly after receipt of regulatory approval. The Phase 2 open-label study will be conducted as an investigator-initiated study in collaboration with Washington University School of Medicine (MO, USA: https://medicine.wustl.edu) and will enroll up to 24 donor/recipient pairs. The trial will evaluate the ability of BL-8040 to promote stem cell mobilization as a single agent in the allogeneic transplantation setting.

Red light

Pluristem

Pluristem (Israel; www.pluristem.com) has received a notice from United Therapeutics (MA, USA; www.unither.com) ending its licensing agreement for the development of PLX-PAD for the treatment of pulmonary arterial hypertension. According to the licensing agreement Pluristem will regain full rights to PLX-PAD in this indication, as well as all clinical data and regulatory submissions, allowing Pluristem to move forward with the clinical development of the program and seek other licensing partners.