Scimitar Equity Blog

ISCO.OB is focused on using hpSC to treat severe diseases of the eye, the nervous system and the liver where cell therapy has been proven … clinically … yet is limited by the availability of safe immune-matched human cells.  ISCO.OB is about to demonstrate the therapeutic applicability and potential immune rejection advantages of hpSC lines relative to other stem cell classes. Its new production facility for clinical-grade stem cell products has passed final building inspection consisting of separate suites to further develop and produce different cell types from the their human parthenogenic stem cell (hpSC) technology.  These parthenogenetic stem cells have the medical advantage of allowing immune-matching to large segments of the population and the ethical advantage of not requiring the use of human embryos.  

Reiterating, ISCO.OB has developed a new stem cell technology called “parthenogenesis” that promises to significantly advance the field of regenerative medicine by addressing the significant problem of immune-rejection.

• Human parthenogenetic stem cells have therapeutic potential,
• Parthenogenesis utilizes unfertilized human eggs to create “parthenogenetic” stem cells (hpSC) that could be immune-matched,
• Like hESCs, hpSCs are pluripotent yet avoid ethical issues associated with destruction or use of viable human embryos,
• Unlike iPSCs, hpSCs do not require manipulation of gene expression back to a less differentiated stage, which may prove to be a safety or regulatory obstacle,
• Unlike both ESCs and iPSCs, hpSCs can be created in a homozygous form such that each line can be an immunological match for millions of patients,
• While researchers are ecstatic with the new outlook on hESC-based research, many have voiced concerns over the decision to prevent funding for cell lines created through parthogenesis as well as another technique called somatic cell nuclear transfer,
• The NIH states that the guidelines reflect the fact that there is no clear consensus … yet … about the ethical process.

Stem cell surgery options and procedures continue to evolve at a rapid rate and companies such as Cytori Therapeutics are making preparations to serve more medical centers and cosmetic surgery facilities with stem cell orders in the oncoming years. Most recently, Cytori has received an order for a StemSource Cell Bank from Cosmetic Surgery Seishin in Japan.  The order request is for the patented Cell Bank which includes a complete StemSource processing system, equipment, software, a database, and protocols needed to freeze and preserve patient’s adipose-derived stem cells.  This StemSource Cell Bank allows doctors to use adult regenerative cells that are extracted directly from the patient’s adipose tissue, and use them in procedures such as stem cell breast augmentation, skin resurfacing procedures, and facial plastic surgery procedures that demand rapid regeneration of cells and skin tissue.

Since stem cell and regenerative cell preservation is such a delicate process, it is critical that the cells are stored under the perfect temperature and extracted using very special techniques and tool.

• The Cell Bank will be installed at Cosmetic Surgery Seishin within the next few months and the center will be using the stem cells derived from liposuction procedures that are performed at the practice on a regular basis.

Chief Medical Officer of Cosmetic Surgery Seishin,  Dr. Tatsuro Kamakura reports, “I have been using Cytori’s products for over three years and they have become an integral part of my practice … I have seen an increasing demand among my patients for the ability to bank their cells on their own future use. Having a StemSource Cell Bank will further distinguish our growing practice.”

Cytori is one of the world leaders in regenerative medicine and has been providing hundreds of physicians with the latest innovations in medical technology and stem cell resources such as the StemSource Cell Bank.

Access Pharma (OTC: ACCP.OB) is an emerging bio-pharmaceutical company which is focusing on the development of a late-stage, diversified oncology pipeline in addition to a treatment called MuGard that is cleared for marketing in the U.S., Europe, and other key global markets for a common side effect of some cancer treatments known as mucositis (painful sores in the mouth and GI mucosal lining).

In addition, the Company is developing earlier stage compounds in its pipeline and has an oral cobalamin nanopolymer drug delivery technology which has demonstrated positive results for the delivery of insulin by mouth in preclinical animal models.

Access expects to maintain a low cash burn rate of approximately $5 million during 2010, as compared to an operating budget of approximately $35 million – reflecting the significant benefit provided by partner funding such as the clinical development of ProLindac in the Asia-Pacific region. In addition, the current cash and expected milestone payments this year provides adequate funding for all development and commercialization plans through at least mid-2011, which does not take into account MuGard royalties from SpePharm in Europe and pending sales of the product in North America.

Last December, ACCP.OB provided an update on its European commercial launch of MuGard, an FDA approved treatment for oral mucositis, a debilitating side effect of radiation treatment and chemotherapy. MuGard is commercially launched by Access’ partner, SpePharm, in six European countries, including the UK, Germany, Italy, Norway, Greece and Sweden. Access is conducting pre-marketing activities, including ramping of commercial production, with the goal of a commercial launch during March / April 2010 in U.S.

Over 15,000 bottles of MuGard have been used by over 2,000 patients since launch. SpePharm is currently gathering feedback from clinicians in the UK, Germany and Italy that are participating in a patient assessment project and expects that out of a total of approximately 1,500 patients who will be given MuGard in this project, a consistent number of patient forms will be collected by year end, and the information will be quality checked for potential presentation at a scientific or medical conference or meeting during 1H10, such as the Multinational Association of Supportive Care in Cancer (MASCC) that will be held June 24-26 in Vancouver.

Thiarabine is ACCP’s next-generation nucleoside analogue (e.g. fludarabine, cladrabine) designed for the treatment of blood-based cancers such as lymphoma and leukemia. Access is currently working with leukemia and lymphoma specialists to initiate additional Phase 2 clinical trials in acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL), and B-Cell lymphomas. These pilot studies are expected to begin during 2Q10 and will seek to determine the optimal dosage regimen and most susceptible malignancies for future trials to serve as the basis for partnership discussions for further development and commercialization.

In early January, Access announced successful results in a small, pilot study that validates the new, scalable manufacturing process for ProLindac that is a key component for partnership discussions and large-scale production of the drug for late-stage / pivotal studies and potential commercialization. ProLindac is a next-generation DACH platinum anti-cancer compound which includes a proprietary nano-polymer drug delivery vehicle that allows for over 10X the dose of platinum to be delivered in a targeted manner to cancer cells with a much better safety profile compared to standard platinum-based drugs which cause significant and cumulative neurotoxicity.

Access will also conduct a combination study evaluating ProLindac plus Taxol (paclitaxel) for second-line treatment of platinum pre-treated patients with advanced ovarian cancer. This is a multi-center study being conducted in Europe in up to 25 evaluable patients with primary efficacy endpoint goal of achieving at least a 63% response rate and expects to begin patient dosing by March-April 2010. The initial cohort of about 10 patients will begin the study as an open-label, dose-escalation study that is expected to provide initial results during 3Q09 and a possible partnership or larger pivotal Phase 3 trial could begin during 2H10.

Access has developed a nano-polymer drug delivery system for the oral administration of large molecules that are currently administered as injections (e.g. insulin, human growth hormone/hGH, erythropoietin/EPO, fertility drugs, parathyroid hormone/PTH, RNA-based therapeutics, monoclonal antibodies). This drug delivery technology involves coating a nano-particle with a vitamin B-12 analog (cobalamin) that binds to intrinsic factor in the gut and triggers binding to cellular receptors which absorb the entire package, resulting in exponential increases in absorption through the gut of large molecule drugs / hormones typically administered by injection.

 In June 2009, Access announced that two bio-pharmaceutical companies (one North American, one European) would conduct preclinical, proof-of-concept studies in animals (rat and dog models of diabetes) before proceeding to more formal negotiations for the Company’s oral, long-acting (basal) insulin product candidate, seeking to validate the greater than 80% oral insulin bioavailability results achieved by Access in preclinical studies for its oral insulin formulation. Final results from the non-exclusive collaborators are possible during 1Q10 while Access may initiate proof-of-concept (Phase 1 equivalent) studies for oral insulin in humans in Eastern Europe or India during 2H10 (with expected duration of 3-5 months and cost of $250-300,000), and also plans to file an additional patent application reflecting improvements to the technology.

With the recently announced financing that included the sale of common stock / warrants at $3 per share, Access will have approximately $7 million in cash / equivalents and 25 million shares of common stock outstanding on a fully diluted basis (up to 30 million shares taking into account outstanding warrants, etc.) plus a $5.5 million note outstanding which is due at the end of 2011. The outstanding note is expected to be paid in part or full by possible upfront payments as part of co-promotion licensing deal(s) for MuGard in North America which could be combined with a reset of the current $27.50 conversion price.

Additional analyst coverage is also possible throughout 2010, including firms such as Rodman & Renshaw (NASDAQ: RODM), which served as the sole lead placement agent in the recently closed financing. Also, listing of Access shares is anticipated on the AMEX, which should provide greater liquidity and improved bid / ask spreads for trading of the stock. Finally, MuGard sales projections at 12-months post-launch include annualized revenue run rates of $30 million for Europe at 20-25% royalties (i.e. $2.5 million per month) and $50 million for North America (i.e. approximately $4 million per month.

Disclosure: Long ACCP.OB

Results emanated from an independent study evaluating the use of Hextend® in hemodynamically unstable trauma patients. Hextend (6% Hetastarch in Lactated Electrolyte Injection) is BTIM’s commercially-available blood plasma volume expander used to treat hypovolemia (low blood volume). The study reported that initial resuscitation with Hextend was associated with NO obvious coagulopathy and reduced mortality compared to fluid resuscitation without Hextend.  However, study principals cautioned that their “…study design has several limitations, but that the results are encouraging and warrant a randomized controlled clinical trial”. Results showed that overall mortality for the patients treated with Hextend was significantly lower at 5.2 % compared to 8.9 % for the patients receiving fluid resuscitation without Hextend.  NO other study has evaluated the safety and efficacy specifically in trauma patients even though there had been several other studies  in various other types of surgical patients.

Hextend is a formulation of 6% hetastarch combined with a physiologically balanced crystalloid carrier that more closely mirrors plasma electrolyte balance than 6% hetastarch in 0.9% sodium chloride.

• The hetastarch component creates oncotic pressure, which would normally be provided by blood proteins and permits retention of intravascular fluid,
• The crystalloid carrier provides electrolytes necessary for physiologic functions and has a composition resembling that of the principal ionic constituents of normal plasma,
• Hextend contains a normal physiological level of calcium.

The non-randomized observational trial was the largest involving Hextend in any group of surgical patients and was conducted by a team of physicians led by Kenneth G. Proctor, Ph.D. at the University of Miami Ryder Trauma Center.

• 1,714 patients were evaluated of which 805 patients received standard of care fluid resuscitation plus up to one liter of Hextend within the first 2 hours of arrival at the trauma center and 909 patients received standard of care fluid resuscitation without Hextend,
• While survival was improved in those receiving Hextend, a higher proportion of these patients required ICU admission and transfusions of blood or plasma, consistent with lower mortality and a higher percentage of patients surviving to require subsequent critical care. All results were confirmed using univariate analysis,
• Dr. Proctor’s team was one of the first to evaluate the potential use of Hextend as a resuscitation fluid in the laboratory.

The U.S. Army has deployed Hextend for initial resuscitation of battlefield casualties because less Hextend is required compared to commonly used saline solution to achieve the same plasma volume expansion effect. This offers a major logistic advantage for a combat medic who has to carry all his supplies in his backpack.

BioTime markets blood plasma volume expanders and related technology for use in surgery, emergency trauma treatment, and other applications. BTIM’s lead product, Hextend®, is a blood plasma volume expander manufactured and distributed in the U.S. by Hospira, Inc. and in South Korea by CJ CheilJedang Corp. under exclusive licensing agreements.

The turmoil in the last 2-3 weeks markets makes it hard sometimes to focus GERN’s short-term share price appreciation.  An expanding indication for GRNOPC1 is good news as the FDA placed a clinical hold on the early-stage trial of this compound. The outstanding warrant exchange accounting for approximately 3/4 of all warrants strengthened the balance sheet with GERN selling additional shares of common to investors at a premium for gross proceeds of $10 M, generating a 2.7 M shares lock-up provision with the new block of shares being purchased and issuance of a call option to the investors to purchase an additional $5 M of common stock.  Adding to the these events,  the GRN163L  compound is being tested on human cells and on rodents showing very striking results; attacking not only the bulk of the tumor cells, but also the rare cancer stem cells that are believed to be responsible for most of a cancer’s growth. Read more…

International Stem Cell Corporation (NASDAQ:ISCO.OB) is a biotech focused on therapeutic and research products.

• ISCO.OB develops pluripotent stem cells that are comparable in function to embryonic stem cells from which cells for human transplant are derived; and techniques to cause those cells to be differentiated into the specific cell types required for transplant, as well as manufactures protocols to produce these cells without contamination with animal by-products.

Focus: ISCO.OB creates human cells for the treatment diabetes, liver disease, corneal disease, and retinal disease through cell transplant therapy, as well as engages in the development of therapeutic products. Read more…

ReNeuron is a UK-based stem cell company. Their primary objective is the development of stem cell therapies targeting areas of significant unmet or poorly met medical need. RENE.L’s shares are traded on the London AIM market.

Focus: ReNeuron’s stem cell products are derived from non-embryonic human tissue sources. RENE.L’s stem cell therapy program have been built around stem cell expansion technology, c-mycER. This platform enables, from a single tissue sample, the growth of selected human stem cells into banks of quality-assured stem cell lines. Read more…

Results from Cytori Therapeutics’ preclinical fat grafting study have been published in the February issue of the Annals of Plastic Surgery. This article, entitled “Supplementation of Fat Grafts with Adipose-Derived Regenerative Cells (ADRCs) Improves Long-Term Graft Retention,” examines the science behind cell-enriched autologous fat grafting and its application to cosmetic and reconstructive surgery. Read more…

Cytori Therapeutics received U.S. Patent No. 7,651,684 (the ‘684 patent) which covers the CYTX’s methods for delivering adipose-derived stem and regenerative cell-enriched fat grafts into patients. The ‘684 patent specifically covers cell-enriched fat grafts to treat soft-tissue defects and urinary incontinence. In addition, the ‘684 patent covers methods for delivering cell-enriched fat grafts to a patient’s breast, wrinkles, lips, under the eyes, cheeks, chin, and other parts of the body and broadens current expansive patent portfolio covering adipose-derived cell therapies. Read more…

MultiCell Immunotherapeutics specializes in developing primary liver cell immortalization technologies to produce cell-based assay systems for use in drug discovery by leveraging patented technology and expertise to develop therapeutics to treat serious diseases including multiple sclerosis, type-1 diabetes, and infectious disease. Read more…