Henry McCusker's  Instablog

With ImmunoCellular Therapeutics (NYSE MKT: IMUC)

NBS' subsidiary, Progenitor Cell Therapy (PCT), and ImmunoCellular Therapeutics (NYSE MKT: IMUC) ("IMUC executed a Services Agreement under which PCT will provide cGMP manufacturing services to support R&D of IMUC's ICT-121 cell therapy product candidate, a dendritic cell vaccine targeting CD133 cells.

The Bottom Line: PCT currently provides manufacturing services for IMUC's lead product candidate, ICT-107, a dendritic cell-based vaccine targeting multiple tumor-associated antigens for glioblastoma, for its P2 clinical trial. With its East and West Coast facilities … PCT has unique expertise in manufacturing, regulatory, logistical transport and commercialization for therapeutics development.

NBS closed on 5/31/13 at $0.56; so expect a few cents <+$0.01 - $0.03 appreciation <look to the last such announcement and up-ward move> on 5/13/13 <Georgetown over 2 days>and as the short interest is at its lowest in over 18 months plus an UP market!

Disclosure: I have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.

Strategic Partnership Award from California Institute for Regenerative Medicine

SGMO receives $6.4 Million Strategic Partnership Award From California Institute for Regenerative Medicine (CIRM) to develop ZFP Therapeutic® for Beta-thalassemia funding for IND application and clinical trial of curative approach on the application of its zinc finger nuclease (ZFN) gene-editing technology in hematopoietic stem cells (HSCs).

The 4 year grant provides matching funds for preclinical work that will support an IND application and a P1 clinical trial in transfusion-dependent beta-thalassemia patients. The grant application entitled "A Treatment for Beta-thalassemia via High Efficiency Targeted Genome Editing of Hematopoietic Stem Cells" won the highest scientific score and was the only application recommended for funding in this round of CIRM's Strategic Partnership Awards.

SGMO is taking a different approach. During development, a fetal form of hemoglobin is made. In infancy, it fully protects beta-thalassemia patients from developing disease symptoms. Later in childhood however, production of fetal hemoglobin ceases and is replaced by synthesis of adult-type beta-globin chains that are defective in beta-thalassemia patients. SGMO's approach enables the permanent production of therapeutic fetal hemoglobin to achieve normal levels of hemoglobin and RBCs, with the goal of eliminating, or greatly reducing, the need for chronic blood transfusions. Moreover, by performing this genome editing in HSCs isolated and returned to the same patient (so called autologous BMT), SGMO's approach eliminates both the need for a matched donor and the risk of GvHD.

The Bottom Line: Adding to our coverage list as SGMO's ZFN-genome editing technology enables modification of a patient's own stem cells and potentially provides a safer approach to current therapies for hemoglobinopathies such as beta-thalassemia and sickle cell disease. the persistence of fetal hemoglobin beyond the newborn stage mitigates the severity of these hemoglobin disorders. If successful, this could eliminate the need for life-long medications and red blood cell transfusions that are currently the standard of care for these disorders."

SGMO closed on 5/23/13 at $7.43 on a low volume <450> day and had traded as high as $10.80 on 5/3/13. This is definitely an actionable event and I project a $0.10 to $0.20 jump for SGMO even in a DOWN and vacation weekend market on the non-dilutive CIRM funding - it might even spill over into Tuesday. The 50 day moving average is $9.28 followed by the 200 at a low of $8.10.

Beta-thalassemia is a genetic disease of the blood caused by mutations in the beta-globin gene. This gene defect leads to impaired production of hemoglobin, the iron-containing protein in red blood cells (RBCs) that carry oxygen from the lungs to the tissues. Individuals with thalassemia are dependent on blood transfusions for survival as they fail to make sufficient healthy RBCs. The unmet medical need in transfusion-dependent beta-thalassemia is significant, with reduced life expectancy due to multi-organ failure caused by iron overload, blood-borne infections and other disease complications. A bone marrow transplant (BMT) of HSCs from a "matched" related donor (allogeneicBMT) is curative.

Disclosure: I have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.

Net loss was $1.1 M or $0.07 per share

Net loss for Q3/13 was $1.1 M or $0.07 per share compared with $1.8 M or $0.11 per share in Q3/12.

Revenue was $3.2 M per share compared with $4.9 M in Q3/12. R&D expenses were $658 K compared to $959 K for Q3/12. G&A expenses were $1.5 M compared to $1.2 M in Q3/12. Total expense was $2.8 M, compared to a total expense of $4.3 M for Q3/12. Top line performance increased more than $700 K or 19% driven by several factors including sales increase of AutoXpress Platform disposables and manual bag sets, and disposables from products used to prepare stem cell concentrates from bone marrow. Growth was driven by several factors, including an increase in sales of AutoXpress(R) Platform (AXP) disposables and manual bag sets. KOOL benefited from increased disposable sales from products used to prepare stem cell concentrates from bone marrow, due to increased sales to its distributor, Celling Technologies.

• KOOL had a total of $7.5 million in cash, cash equivalents, and investments compared to $7.9 M in cash and cash equivalents at 12/31/12.

Q1/13 Highlights

• Added Crioestaminal, a leading cord blood stem cell bank in Portugal, as a customer for AXP System, replacing a competitor's product;
• Appointed Dan Bassey as CFO. Dan brings more than 20 years of finance and accounting experience, with strong expertise in operations, strategic planning and M&A.

The Bottom Line: KOOL struggles to break the $1.00 mark - last seen on 1/14/13. A "footprint" in China with a 5 year contract … could boost revenues <and other Asian countries>but by when? KOOL continues to better leverage its cost structure to increase core revenues year-over-year they reduced operating loss in Q1/13 by $500 K which excluded the gain on sales of assets and … their cash balance remained consistent.

Reduced net loss by $640 K through cost management strategies, despite $619 K in costs related to litigation and positioning initiatives. 22% of their $2.8 M in total operating expenses was non-core operating costs, including litigation related expenses and those incurred to support our longer term growth initiatives. In the conference call, Matt said - KOOL not infringe that there patents < the patent for the rescue system> were invalid and Harvest has requested more time to respond to KOOL counterclaim answers - the 1st bullet. Will this get thrown out?

My follow-on question is - how does KOOL maintain their software with their current abilities and capacities? Also the revenue regeneration is "not" theirs but the distributors - again issues of control and motivation. and for a 3rd bullet - is there … competitive trouble trouble brewing … with SynGen and their <KOOL's> former founder … concerning more superior harvesting capacity? See … @ syngeninc.com … who has been funded by Bay City Capital.

KOOL has completed a P1 and P1b study in CLI; the CLI P1 trial with the University of Naples was published last quarter. The data show that 1 year following administration of the cells that 10 of the 13 patients showed improve blood flow and as a whole, the cohorts blood flow doubled, which is compelling but - early. The P1b CLI study conducted with our other partner … TotipotentRX … in India treated 15 patients, which also compelling interim results. These were very sick patients, were specific or significant prior medical interventions had failed to achieve re-vascularization and each were within 24 hours of limb amputation. KOOL completed a P1 study of non-union fractures with its partner, TotipotentRX in US and India. This P1 study totaled 19 enrolled patients, of which 5 were last follow-up, each of whom had failed numerous prior surgical attempts at bone fusion. The study showed significant improvements in healing and pain scores as well as the 71% union rate within 18 weeks. Based on these results, we're planning a P2 study for non-union fractures in conjunction with our partner.

The 4th bullet … Why is KOOL in the "therapeutic" development business - a tremendous cash drain!!

KOOL closed UP +$0.03 to $on 5/13/12 on 65.5 K shares; was UP again +$0.05 to $0.95 on 5/14 with 32.4 K shares but, drifted DOWN to $0.91 on 5/15/15 on 22.6 K shares. A … HOLD based on revenue "pushes" otherwise KOOL will "drip" downward as 2013 "floats" on!

Disclosure: I have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.