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NW Bio’s Cancer Vaccine Is The First Drug To Be Designated By UK Authorities As A “Promising Innovative Medicine” (PIM)

On September 16, 2014 Northwest Biotherapeutics reported 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 (Press release Northwest Biotherapeutics, SEP 16, 2014, View Source [SID:1234500752]). 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. Information about the EAMS program can be found on the MHRA website at: View Source

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.

NW Bio’s DCVax-L has now become the first product to earn this PIM certification. There is no expiration on the PIM certification.

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.

In granting the first PIM designation under this new EAMS to DCVax-L, the UK Department Of Health has stated, “An innovative cell therapy for cancer has become the first to be certified as ‘promising’ as part of a new Government scheme aimed at getting new medicines to patients quicker.

The medicine, which has been developed by US-based pharmaceutical company Northwest Biotherapeutics Inc. (NW Bio), is the first drug to be awarded the UK’s new Promising Innovative Medicines (PIM) designation, the initial step in the Early Access to Medicines Scheme which aims to increase patient access to unlicensed treatments.”

UK Life Sciences Minister George Freeman noted that “The designation of the PIM is the first, crucial step in developing cutting-edge medicines sooner, giving real hope to patients and their families.”

Dr. Keyoumars Ashkan, a senior neuro-surgeon at Kings College Hospital in London, and the Principal Investigator for the clinical trial of DCVax-L in the UK, commented that “Brain cancers strike patients of all ages, and are rapidly lethal. New treatment options are urgently needed. DCVax-L offers an exciting new approach to treating these brain cancers, through personalized immune therapy. It is encouraging to see this innovative new product be the first to receive certification as a Promising Innovative Medicine under the new EAMS.”

Linda Powers, CEO of NW Bio, commented that “We are most grateful to the MHRA and Minister Freeman for spearheading this new EAMS. It strikes a very practical balance between clinical benefits and risks, through careful scientific evaluations, and will be of great help to doctors and patients in opening up new treatment options.”

“In prior Phase I/II clinical trials of DCVax-L for brain cancer, significant delays in disease progression and significant extensions of patients’ survival have been seen, with virtually no serious adverse effects,” Ms. Powers continued. “We believe that DCVax-L embodies the combination of innovation and beneficial balance of clinical benefits and risks that the EAMS is designed to accelerate. We are excited that DCVax-L has received the first PIM certification.”

(Press release, NantBioScience, SEP 15, 2014, View Source [SID:1234505862])

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(Press release, NantBioScience, SEP 15, 2014, View Source [SID:1234503331])

(Press release, NantBioScience, SEP 15, 2014, View Source [SID:1234503306])

SignalRx Presents at AACR Conference on its Dual Kinase-Epigenetic Inhibitors for Treating Cancer

On September 15, 2014 SignalRX Pharmaceuticals Inc., focused on developing more effective oncology drugs though molecular design imparting multiple target inhibition, reported the presentation of scientific data on the Company’s proprietary dual inhibitor program in oncology (Press release, SignalRx, SEPT 15, 2014, View Source [SID1234527331]). The presentation by Dr. Donald Durden, MD, PhD, founder and CEO of SignalRx was made at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Conference on Targeting the PI3K-mTOR Network in Cancer at the Sheraton Philadelphia Downtown Hotel, Philadelphia, Pennsylvania.

The presentation highlighted the discovery and early development of patented new molecules such as SF2523 that inhibit kinase targets such as PI3 kinase (PI3K) while also blocking epigenetic targets such as bromodomain proteins. SignalRx presented in vivo evidence of efficacy without toxicity in mouse cancer models with SF2523 alleviating potential theoretical safety concerns arising from inhibiting multiple key nodal cancer targets with one drug.

The ability to safely simultaneously inhibit multiple key targets is highly sought after in cancer treatment to maximize efficacy and prevent resistance. Replacing several combinations of drugs with one drug hitting multiple targets is also being driven by exploding oncology drug costs to patients and to the health care system. SignalRx compounds are being developed as next generation PI3K inhibitors and it has now been shown that they also potently inhibit certain bromodomain proteins such as BRD4. SF2523 is a lead dual inhibitor compound and has been demonstrated to: 1) inhibit proliferation across 18 different cancer cell lines; 2) induce apoptosis in neuroblastoma cells, renal cell carcinoma cells, multiple brain cancer cell lines including patient-derived samples; 3) block angiogenic signaling and blood vessel production in vivo; 4) inhibit cancer stem cells in breast and human medulloblastoma patient cells; 5) exhibit potent antitumor efficacy and anti-metastatic effects without toxicity in renal cell carcinoma xenograft models, neuroblastoma mouse models, orthotopic pancreatic cancer model and Lewis lung cancer models.

Bromodomain proteins bind to acetylated lysine groups on chromatin and promote gene transcription. SignalRx’s compounds act as acetyl lysine mimetics and prevent the bromodomain protein from binding to chromatin. This mechanism of action is distinct from the compound’s PI3K inhibition where the inhibitors bind in the ATP catalytic site of the kinase. This allows for the first time simultaneous control of both the PI3K pathway and the transcription of certain genes mediated by bromodomain proteins. An example of the utility of this approach to solve unmet medical needs is to block the tumor suppressor gene MYC which is a driver in many cancers including CLL and multiple myeloma and for which small molecule inhibitors have been elusive. Inhibition of PI3K increases the cellular degradation of MYC protein while BRD4 inhibition decreases the transcription (production) of MYC protein. Use of SignalRx’s dual PI3K/BRD4 inhibitors provides a unique and effective approach to block the action of MYC via two orthogonal mechanisms demonstrated with lead compound SF2523 to be efficacious without toxicity in mouse models.

These new compounds are broadly covered by composition-of-matter U.S. Patent No. 8,557,807 entitled "Thienopyranones as kinase inhibitors" issued October 15, 2013. Additional information on preparation and structure-activity-relationships of these compounds were recently published in the Journal of Medicinal Chemistry (February 14th 2013 issue, volume 56, pages 1922-1939).

"The discovery of SF2523 which inhibits both PI3K and BRD4 represents a major step forward in designing anticancer agents to be as effective as possible" said Donald L. Durden, MD, PhD,. "Additionally, the problem of early stage clinical trial evaluation of multiple combinations is solved by this approach by consolidating at least two of the combination partners into one drug. Moreover, because two separate drugs will always suffer from differing cell penetration, metabolism, and pharmacokinetics our approach of one molecule hitting two critical targets in the same cell is the only way to ensure that desired simultaneous blockage of multiple key signaling pathways is achieved in vivo."