Industry Update: Regulations, approvals, acquisitions…
Latest developments compiled from 01 February — 31 March 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 01 February — 31 March 2015, scheduled to be published in Volume 10 Issue 5 of Regenerative Medicine.
Green light
Neurotech
US FDA gave a green light to Neurotech Pharmaceuticals (RI, USA; www.neurotechusa.com) to proceed with its Investigational New Drug application to conduct a Phase 2 clinical study of NT-503 Encapsulated Cell Therapy (ECT) for the treatment of recurrent subfoveal choroidal neovascularization secondary to age related macular degeneration (wet AMD). The Phase 2, randomized, active-controlled, masked study will commence immediately and enroll 150 patients. The safety and efficacy of one NT-503 ECT implant will be compared to aflibercept intravitreal injections every 8 weeks in patients who have been treated with at least 3 anti-vascular endothelial growth factor (VEGF) injections and still have active disease. Patients will be followed for 2 years with a 12-month interim assessment. ECT is an intravitreal implant that houses a proprietary retinal pigment epithelial cell line genetically engineered to produce several therapeutically active biologics for at least 2 years. NT-503 is a novel VEGF receptor protein that is continuously produced by the ECT platform.
ViaCyte
ViaCyte (CA, USA; www.viacyte.com), a privately-held regenerative medicine company with the first hESC-derived islet replacement therapy for the treatment of diabetes in clinical trials, has received a No Objection Letter from Health Canada providing clearance to proceed with sites in Canada for the Company’s Phase 1/2 clinical trial of its VC-01TM product candidate. The VC-01 product candidate is currently being evaluated in patients with type 1 diabetes who have minimal to no insulin-producing b-cell function. The location and enrollment start date of the first Canadian clinical trial site have not yet been disclosed. Further information about the ongoing clinical trial in the USA are available at http://clinicaltrials.gov (ID: NCT02239354).
Red light
WARF
A nine-year legal challenge to human embryonic stem cell patents ended, when the Supreme Court declined to hear the case. The decision means the Wisconsin Alumni Research Foundation (WARF; WI, USA; www.warf.org), will get to keep its patent rights for the hESC, which were discovered in 1998 by University of Wisconsin – Madison scientist James Thompson [2]. WARF maintains it has the right to license use of human embryonic stem cells, because Thompson developed the methods to isolate them from embryos, which had not been previously done even though the derivation is an obvious extension of methods used to derive non-primate embryonic stem cells, and therefore not patentable. However, the challengers succeeded in preventing WARF from gaining rights over iPSC. Two public interest groups, Consumer Watchdog (CA, USA; www.consumerwatchdog.org) and the Public Patent Foundation (NY, USA; www.pubpat.org), challenged the patents in 2006, and in 2007 succeeded in narrowing WARF’s claims to exclude the IPSC. The groups continued the challenge on the grounds that as a product of nature, hESC are not patentable. The U.S. Patent and Trade Office turned down that challenge, and the case reached the Supreme Court last year. By not hearing the case, the Supreme Court let that decision stand.
Acquisitions
Actavis and Allergan
Shareholders of Actavis (Ireland; www.actavis.com) and Allergan (CA, USA; www.alergan.com) approved all proposals related to Actavis’s planned acquisition of Allergan. The deal, valued at US$ 66 billion, is expected to close in the second quarter. The combined company would be one of the world’s largest drug makers by sales, with about US$ 23 billion in sales, led by Allergan’s anti-wrinkle treatment Botox. Actavis’s shares, up nearly 38% over the past 12 months, edged up to US$ 290.17 in recent after-hours trading, while Allergan’s shares, up 80% over the past 12 months, edged up to US$ 233.90.
Fuji Film and CDI
Fuji Film (Japan; www.fujifilmholdings.com/en/index.html) will acquire and Cellular Dynamics International (WI, USA; www.cellulardynamics.com), via an all-cash tender offer to be followed by a second step merger. Fujifilm aims to acquire all issued and outstanding shares of Cellular Dynamics’ common stock for US$ 16.5 per share or approximately US$ 307 million (on a fully diluted basis). The offer represents a premium of 108% to Cellular Dynamics’ closing price on March 27. Upon completion of the transaction, Cellular Dynamics’ will continue to run its operations in Madison, Wisconsin and Novato, California as a consolidated subsidiary of Fujifilm.
Kite and TCF
Kite Pharma (CA, USA; www.kitepharma.com) has acquired privately held T-Cell Factory (TCF; the Netherlands) for US$ 22.5 (€ 21.0) million million) upfront, and renamed it Kite Pharma EU, in a deal designed to establish a European presence for the buyer and strengthen its T Cell Receptor (TCR) gene therapy product platform. TCF has focused on discovery and development of tumor-specific TCRs for broad use in cancer treatment based on its TCR-GENErator™ platform. TCRs allow targeting of tumor antigens found inside cancer cells, as well as surface antigens. Kite agreed to make the upfront payment, of which US$ 4.0 (€3.8) million will be paid in Kite stock, as well as an undisclosed sum in payments tied to achieving clinical, regulatory, and sales milestones relating to TCR-based product candidates.
Orgenesis and MaSTherCell
Orgenesis (NY, USA; www.orgenesis.com) is acquiring MaSTherCell (Belgium; www.masthercell.com), a contract development and manufacturing organization. Orgenesis bought all issued and outstanding shares of MaSTherCell in exchange for US$ 24.593 million in shares of the acquiring company’s common stock. Orgenesis said MaSTherCell proved to be an attractive acquisition target because of its process development capabilities and fully closed and tightly controlled GMP systems required to deliver commercial-scale manufacturing. Before the acquisition, the two companies had already been working together under a strategic manufacturing agreement.
Valeant and Dendreon
Valeant Pharmaceuticals International (QC, Canada; www.valeant.com) has purchased bankrupt Dendreon (WA, USA; www.dendreon.com) and its prostate cancer vaccine Provenge® (sipuleucel-T) for US$ 415 million [3]. Dendreon filed for Chapter 11 bankruptcy protection in November 2014 battered by the failure of Provenge, which cost US$ 93,000 for a course of treatment, to make a dent in the prostate cancer markets. Provenge had sales of around US$ 300 million last year.
References:
[2]Thomson JA, Itskovitz-Eldor J, Shapiro SS et al. Embryonic stem cell lines derived from human blastocysts. Science. 282 (5391), 1145-7 (1998).
[3]Ledford H. Therapeutic cancer vaccine survives biotech bust. Nature. 519, 17-18 (2015).