Industry Update: Regulations, approvals, acquisitions…
Latest developments compiled from 1 October until 30 November 2014
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 October until 30 November 2014, scheduled to be published in Volume 10 Issue 2 of Regenerative Medicine.
Regulations
EMA
After an 18-month saga, the European Medicines Agency (EMA) has approved the details of a new system allowing researchers to scrutinize unpublished data from clinical trials. Scientists and transparency campaigners have praised the decision as a big step forward but have voiced lingering concerns, in particular about information that could be redacted before clinical study reports are shared. EMA is the first entity in the world to introduce such rules, setting new standards for transparency. The plan, which was approved at a managing board meeting in London, represents a real shift in favor of ensuring research data is shared routinely and reused effectively in the public interest. EMA’s decision is part of a major battle over who has the right to see and analyze the data from clinical studies of drugs, vaccines, and other medical products. Published journal articles often contain the main outcomes of such studies but lack detailed data and information about study design, efficacy, and safety analysis, which might shed a different light on the results when analyzed by others; moreover, some trials are not published at all. The AllTrials campaign has argued that the details of every trial should be publicly available for anyone to study. Industry has balked at the idea, although some companies have recently committed to greater openness. The policy’s annex 3 lists items that could be redacted from clinical study reports because it may be considered “commercially confidential information.” This includes, for example, details about proprietary information about analytical methods, or so-called exploratory endpoints that could provide clues of potential off-label uses to competitors. The new rules will apply to data submitted as part of marketing authorization applications after 1 January 2015 [5]. The policy will be reviewed by June 2016 and will serve as a bridge until the rollout of the revamped EU clinical trial regulation–which provides a legal basis for the release of clinical trial results, but will enter into force no sooner than May 2016. This means that results of the first trials conducted under the new law will be publicly available from 2019 or 2020 on. At a later stage, EMA also plans to make available individual patient data.
Green light
BioCardia
BioCardia (CA, USA; www.biocardia.com) has received a green light from the FDA to begin a Phase III study of its combination bone marrow-derived CardiAMP therapy and in vitro diagnostic for heart failure. The product is the first heart stem cell therapy to go through the FDA’s medical device approval process rather than the biologics pathway. The trial will enroll 250 patients at up to 40 clinical sites.
BioTime
BioTime (CA, USA; www.biotimeinc.com) has received authorization to begin its pivotal human clinical trial of Reneviaâ„¢ in Europe. In the trial, Renevia will be used in combination with the patient’s own fat-derived cells and injected into portions of the patient’s face where there has been a loss of fat from under the skin (lipoatrophy). Lipoatrophy is estimated to occur in 35-50% of the 10 million HIV patients on antiretroviral therapy. This pivotal trial follows the previous successful safety trial of Renevia, the completion of which was announced earlier in 2014.
BrainStorm
BrainStorm (Israel; www.brainstorm-cell.com) have received the US FDA approval for commencement of their multi-center Phase II trial for Amyotrophic Lateral Sclerosis (ALS) in the US. NurOwnâ„¢ is autologous, adult stem cell therapy technology that differentiates bone marrow-derived mesenchymal stem cells (MSC) into specialized, neuron-supporting cells. These neuron-supporting cells (known as “MSC-NTF” cells) secrete neurotrophic, or nerve-growth, factors for protection of existing motor neurons, promotion of motor neuron growth, and re-establishment of nerve-muscle interaction. The ability to differentiate MSC into MSC-NTF cells, and confirmation of their activity and potency before transplantation, makes NurOwn a first-of-its-kind approach for treating neurodegenerative diseases.
Cell Cure Neurosciences
US FDA has cleared Cell Cure’s (Israel; www.cellcureneurosciences.com) Investigational New Drug (IND) application seeking to initiate a Phase I/IIa clinical trial of OpRegen® in patients with geographic atrophy, the severe stage of the dry form of age-related macular degeneration. OpRegen consists of animal product-free retinal pigment epithelial cells derived from hESC and is intended to be administered as a single dose into the subretinal space of patients’ eyes in order to treat this leading cause of blindness. Cell Cure will conduct the trial in Israel where OpRegen will be transplanted as a single dose into the subretinal space of the eye to test the safety and efficacy of the product. Patient enrollment is expected to begin in 2014 following approval of the trial by the Israel Ministry of Health.
ISCO
International Stem Cell Corporation (ISCO; CA, USA; www.internationalstemcell.com), announced that the US FDA has cleared ISCO’s human parthenogenetic stem cells line for investigational clinical use.
ReGen
Regen BioPharma (CA, USA; www.regenbiopharma.com) announced issuance of IND number 16200 from the FDA for a proposed Phase I/II clinical trial assessing safety with signals of efficacy of the dCellVax gene silenced dendritic cell immunotherapy for treating breast cancer. The proposed trial will recruit 10 patients with metastatic breast cancer and will involve 4 monthly injections of the dCellVax gene-silenced dendritic cell therapy. The trial will last one year, with tumor assessment before therapy and at 6 and 12 months.
Pending
Kadimastem
Kadimastem (Israel; www.kadimastem.com) had approached the US FDA regarding the cellular treatment it is developing for ALS. Based on the quality of the data presented, the FDA recommended rapidly moving forward in the approval process towards product development and safety (towards clinical trials). The company’s platform technology enables the differentiation of human pluripotent cells (hPSC) into mature oligodendrocytes, astrocytes, motoneurons, pancreatic beta cells and other cell types.
Mesoblast and JCR
Mesoblast’s (Australia; www.mesoblast.com) Japanese partner, JCR Pharmaceuticals Co (http://www.jcrpharm.co.jp/en/), has filed with the Japanese Pharmaceuticals and Medical Devices Agency (PMDA; www.pmda.go.jp/english/) to receive approval for manufacturing, marketing, and product registration of the allogeneic or “off-the-shelf” MSC product JR-031 for the treatment of acute graft versus host disease (GVHD) in children and adults. The product registration filing by JCR will be subject to a priority review as JR-031 has been granted orphan drug status. If the filing is successful, JR-031 will be the first allogeneic cell-based product approved in Japan. The product was initially licensed form Osiris Therapeutics (MD, USA; www.osiris.com). The licensor of the product has been changed to Mesoblast following the assignment of human MSCs-related rights from Osiris to Mesoblast in October 2013. Mesoblast plans a product registration filing with the US FDA for its allogeneic MSC product in children with steroid-refractory acute GVHD in 2016. Additionally, Mesoblast remains on track for commercial launch of its allogeneic MSC product in Canada and New Zealand in 2016 in children with GVHD.
TissueGene
TissueGene (MD, USA; www.tissuegene.com) has received recommendations from the US FDA to submit its Phase 3 protocol for review under the Special Protocol Assessment. These recommendations were in response to TissueGene’s request for an end-of-Phase 2 meeting to discuss plans for pivotal Phase 3 randomized controlled trials of TissueGene-C for treatment of osteoarthritis of the knee. TissueGene has completed Phase 2 trials of TissueGene-C for an allogeneic cell therapy for osteoarthritis of the knee. Further information about this clinical trial are available at http://clinicaltrials.gov (ID: NCT01671072).
Name Change
Aastrom into Vericell
The publicly traded company Aastrom Biosciences (MI, USA) moved its headquarters from Michigan to the Boston area and renamed itself Vericel (MA, USA; http://vcel.com). The firm would maintain its Michigan manufacturing operations. It was not immediately clear how many employees the move would affect. The company had 190 employees as of June 30, 2014, according to a SEC filing. The move comes less than a month after Aastrom raised US$ 40.3 million by offering 15.7 million shares of its stock at a price of US$ 2.55. The offering boosted the company’s liquidity amid concerns about its cash flow.
Advanced Cell Technology into Ocata
Advanced Cell Technology (MA, USA; www.advancedcell.com) changed its name into Ocata Therapeutics (MA, USA; www.ocata.com).
Stemgent into Asterand
Stemgent (MA, USA; www.stemgent.com) decided to diversify its business two years ago by buying Detroit-based Asterand, a tissue bank company. Stemgent’s investors liked the tissue bank business so well they have decided to un-diversify. In September, they announced the sale of the stem cell operation to ReproCELL (Japan; www.reprocell.com/en/), for US$ 8.5 million in cash. On Oct. 1, Stemgent announced the deal had closed and that the company had been renamed Asterand Bioscience (www.asterand.com) with headquarters moved back to Detroit.
Acquisitions
Bioventus
Bioventus (NC, USA; www.BioventusGlobal.com) has acquired the OsteoAMP® product line, intellectual property and commercial business from Advanced Biologics (CA, USA; www.advancedbiologics.com). OsteoAMP is a bone allograft made using a proprietary process designed to retain the bone’s natural growth factor complement. OsteoAMP comes in four formats and is a cost-effective alternative to recombinant growth factors and allograft-derived stem cells. The four distinctive formats of the product (granules, compressible sponges, putty, and structural grafts) provide an advantage by allowing surgeons to tailor delivery of the technology according to their needs in surgery.
References:
[5]http://www.ema.europa.eu/docs/en_GB/document_library/Other/2014/10/WC500174796.pdf