Industry update: regulations, approvals, acquisitions…

Latest regulatory, approval and acquisition developments compiled from 1 April—31 May 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 1 April—31 May 2015, scheduled
to be published in Volume 10 Issue 6 of Regenerative Medicine.

Green light

jCyte

jCyte (CA, USA; www.jcyte.com) has received a green light from the US FDA for a first-of-its-kind stem cell-based treatment for retinitis pigmentosa. The Phase I/II study will begin enrolling up to 16 patients at University of California Irvine (www.uci.edu) and a possible second site. The primary purpose of the trial is to determine the safety of a single injection of retinal progenitor cells into the eyes of patients with advanced retinitis pigmentosa. The effect on ocular function will also be assessed.

Pluristem

Pluristem Therapeutics (Israel; www.pluristem.com) announced that Japan’s Pharmaceuticals and Medical Devices Agency (PMDA; www.pmda.go.jp/english/) agreed with the proposed quality and large-scale manufacturing methods for PLacental-eXpanded (PLX)-PAD cells for use in clinical trials. This agreement is an important milestone for initiation of a Phase I/II study in critical limb ischemia through Japan’s Accelerated Pathway for Regenerative Medicine. The new regulatory pathway could potentially significantly reduce time to market for cell therapies such as PLX cells. Japan’s Accelerated Pathway for Regenerative Medicine went into effect in November 2014. According to the law, regenerative medicine therapies can receive conditional, time-limited approval for marketing, and be eligible for reimbursement, upon proof of safety and initial proof of efficacy. Safety and effectiveness need to be confirmed within 7 years after the conditional approval.

Pluristem’s PLX cell program in critical limb ischemia has been selected for the European Medicines Agency’s Adaptive Pathways pilot project. The goal of the project is to improve timely access for patients to new medicines. It allows for early marketing authorization of a therapy in a restricted patient population, followed by additional assessments and the possibility of later approval for use in broader patient populations.

RepliCel

RepliCel Life Sciences (BC, Canada; www.replicel.com) has received approval from the University of British Columbia (UBC) Clinical Research Ethics Board (CREB) to conduct a Phase I/II clinical trial at the UBC Hospital. The trial, entitled, “A randomized, double-blind, placebo-controlled, single-centre study to evaluate the efficacy and safety of RCT-01 in men and women with unilateral, chronic Achilles tendinosis” (also known as the “ReaCT study”), will investigate the potential of RepliCel’s tendon repair product, RCT-01, to treat chronic tendon injury. CREB approval clears the way for site initiation and patient recruitment into the clinical trial. The study will include 28 participants with chronic Achilles tendinosis. Study participants will receive ultrasound-guided injections of either RCT-01 or placebo (on a 3:1 treatment-to-placebo ratio) directly into areas of injury within the Achilles tendon. Participants’ overall health and tendinosis will be monitored over a six-month period while they undergo post-treatment physiotherapy to help facilitate recovery from their Achilles tendinosis. RCT-01 is the first of two products from RepliCel’s non-bulbar dermal sheath fibroblast platform currently slated for clinical trials in 2015.

Regeneus

Regeneus (Australia; http://regeneus.com.au/) has received ethics approval for a clinical trial of its new off-the-shelf allogeneic stem cell treatment, known as Progenza, for patients with knee osteoarthritis. Progenza is produced from adipose tissue from a healthy donor who has been extensively screened. When Progenza cells are injected into the damaged joint or tissue, the MSCs have the potential to reduce pain and inflammation and slow the progression of disease.

Regeneus has also received ethics approval to commence a trial for a personalized therapeutic cancer vaccine that is aimed at harnessing the body’s own immune system to fight cancer cells. The vaccine, known as RGSH4K, is produced from a patient’s own cancer cells and, combined with a proprietary immunostimulant, is designed to activate the immune system against the cancer cells to initiate a body-wide response. The immune system’s memory should recognize and respond to both existing and new tumors.

Pending

Anika

Anika Therapeutics (MA, USA; www.anikatherapeutics.com) has filed an Investigational Device Exemption (IDE) with the US FDA to conduct a pivotal Phase III clinical trial for Hyalofast®, a biodegradable, 3D, hyaluronic acid-based scaffold with autologous bone marrow aspirate concentrate (BMAC). European clinical data demonstrated that patients who utilized Hyalofast with BMAC in a one-step arthroscopic procedure were able to naturally regenerate hyaline-like cartilage with a minimally invasive and cost-effective procedure. Hyalofast is CE Marked in Europe for the entrapment of mesenchymal stem cells in connection with the treatment of chondral and osteochondral lesions, and it is commercially available in Europe and certain other international markets. The Hyalofast pivotal trial, “FastTRACK,” will compare Hyalofast’s treatment of articular knee cartilage defect lesions to that of a control microfracture treatment. FastTRACK is a prospective, randomized, active treatment-controlled, evaluator-blinded, and multi-center study. Anika expects to enroll 200 patients at up to 30 sites in the United States and Europe beginning in the fourth quarter of 2015.

ReNeuron

ReNeuron (UK; www.reneuron.com) has filled an Investigational New Drug (IND) application with the US FDA to commence a Phase I/II clinical trial with its human Retinal Progenitor Cell (hRPC) therapy candidate for retinitis pigmentosa, a group of hereditary eye diseases that lead to progressive kiss of sight due to dying cells in the retina. The US FDA put the hRPC therapy for retinitis pigmentosa on the Fast Track after reviewing pre-clinical data ahead of granting permission for phase I/II trials. The Phase I/II clinical trial will be conducted at Massachusetts Eye and Ear Infirmary (MA, USA; www.masseyeandear.org). The trial design is an open-label, dose escalation study to evaluate the safety, tolerability and preliminary efficacy of the hRPC stem cell therapy candidate in up to 15 patients with advanced retinitis pigmentosa. The method of administration of the hRPCs will be a single sub-retinal injection. The primary endpoint of the study is safety, with patients being followed up for 12 months post-treatment with monitoring including measurements of visual acuity.