Industry Update: Regulations, approvals, acquisitions…

Written by Dusko ILIC

Latest developments compiled from 1—30 September 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—30 September 2014, scheduled to be published in Volume 10 Issue 1 of Regenerative Medicine.

Green light

Bone Therapeutics

Bone Therapeutics (Belgium; www.bonetherapeutics.com) has received clearance from the Competent Authorities and Central Ethics Committee in Belgium for a phase II proof-of-concept study to assess safety and efficacy of its allogeneic osteoblastic cell therapy product ALLOB® in spinal fusion procedures for degenerative lumbar disc disease. ALLOB has been classified as a tissue engineered product under ATMP regulation 1394/2007EMA.In this pilot proof-of-concept study, 16 patients with symptomatic degenerative lumbar disc disease that require interbody fusion will be treated with a single dose of ALLOB mixed with bioceramic granules to promote bone formation and fusion at the degenerative disc level. Patients will be enrolled in 4 centers and safety and efficacy of the treatment will be monitored over 12 months by clinical (Oswestry Disability Index) and radiological (fusion progression) evaluation, with an additional 24-month post-study follow-up.

Cellular Dynamics International

US Patent and Trademark Office has awarded Cellular Dynamics International (WI, USA; www.cellulardynamics.com) the Patent No. 8,815,585 on the automated production of human pluripotent stem cells, including iPSC. This patent has broad-reaching effects, covering research, cellular therapy development and stem cell banking. Large-scale applications, such as cellular therapeutic development as well as stem cell banking, require automation for parallel processing of samples from many donors. In addition, the patent broadly covers automated iPSC culturing without the use of feeder cells or the addition of serum to culture media, factors important to cellular therapy development in a current Good Manufacturing Practice (cGMP) environment. This patent joins an Intellectual Property portfolio of over 800 patents awarded to, pending or in-licensed by Cellular Dynamics.

Medipost

Medipost America (MD, USA: www.medipostamerica.com) has received the US FDA approval of Phase 1/2 clinical trial for PNEUMOSTEM®. The Phase 1/2 trial will assess the safety and efficacy of PNEUMOSTEM on prematurely born infants who will be at high-risk of developing Bronchopulmonary Dysplasia. PNEUMOSTEM is made from allogeneic human umbilical cord blood-derived MSC, and is expected to have effect on regenerating the lung tissue and improving inflammatory responses in premature infants. Bronchopulmonary Dysplasia is the leading cause of mortality and severe complications in premature infants and currently no therapy or approved drug exists. Hence, the PNEUMOSTEM trial is expected to draw global attention in the field of neonatal medicine. PNEUMOSTEM received Orphan Drug designation in Korea by the Ministry of Food and Drug Safety (MFDS) and the Phase 2 study in Korea is 80% complete.

Northwest

Northwest Biotherapeutics (MD, USA; www.nwbio.com), a biotechnology company developing DCVax® personalized immune therapies for solid tumor cancers, announced that its DCVax-L is the first product to receive formal designation as a “Promising

Innovative Medicine” (PIM) under the new “Early Access to Medicines Scheme” (EAMS) launched in the UK in April 2014 [1]. A PIM is the first step in a 2-step process for early access approval under the EAMS. The PIM designation for DCVax-L covers all malignant gliomas, which would include both Glioblastoma multiforme (the most severe grade) as well as less malignant grades, and would include both newly diagnosed and recurrent gliomas. The EAMS is an important new initiative in the UK, launched by the Medicines and Healthcare Products Regulatory Agency (MHRA, the FDA of the UK), to lead the way in accelerating patients’ access to innovative new medicines for serious diseases. The first step under the EAMS is MHRA’s scientific evaluation of whether a product candidate meets three criteria for a PIM designation: (i) the product is for a serious disease or condition with high unmet medical need; (ii) the product is likely to offer a major advantage over treatments available today; and (iii) the potential adverse effects of the product are outweighed by the potential benefits. The second step under the EAMS is MHRA’s determination of a Scientific Opinion about the product candidate’s benefits and risks, based on available clinical data. A positive or negative Scientific Opinion will be judged by the same three criteria as for the PIM designation, as well as a fourth criterion: the Company’s ability to manufacture the product to rigorous “GMP” (clinical grade) standards. If the Scientific Opinion is positive, the product candidate may then be prescribed by physicians and provided to (and paid for by) patients before the product is formally licensed and while it is still in clinical development. MHRA aims to deliver the Scientific Opinion within 90 days after a party submits an application for Step 2 of the EAMS process. NW Bio’s DCVax-L has now become the first product to earn this PIM certification. There is no expiration on the PIM certification.

References

[1]http://www.mhra.gov.uk/Howweregulate/Innovation/EarlyaccesstomedicinesschemeEAMS/index.htm