Research « LifeTech Capital

17 May 2012

StemCells (STEM) Note 05-17-12

Posted in StemCells Inc. (STEM) | Comments Off

HuCNS-SC® Phase I/II in Spinal Cord Injury Data Shows Safety in AIS-A
Enrollment in Spinal Cord Injury Trial Now Expands to U.S., Canada and EU
HuCNS-SC® Phase I PMD Data in Children Shows Unprecedented Results
Gains in Motor and/or Cognitive Function in 3 out of 4 Children

Download Full 8-Page Note with Important Disclosures: Morning Note 05-17-12 STEM

StemCells Inc. announced completion of the first planned interim safety review in the first cohort of patients (the severe AIS-A patients) in their Phase I/II spinal cord injury clinical trial implanting their HuCNS-SC^® cells (purified human neural stem cells) demonstrating the surgery, immunosuppression and the cell transplants were been well-tolerated. This represents the first time that neural stem cells have been transplanted as a potential therapeutic agent for spinal cord injury.

Specifically, the AIS-A patients suffer from a complete spinal cord injury in which there is no neurological function below the level of the injury and were transplanted with a dose of 20 million cells at the site of injury in the thoracic spinal cord.

There were no abnormal clinical, electrophysiological or radiological responses to the cells

All patients were neurologically stable through the first four months following transplantation of the cells.

The independent Data Safety Monitoring Committee, has recommended that the study advance to enrollment of patients with incomplete neurological injury (AIS-B).

Enrollment is now underway and is open to patients in Europe, the United States and Canada with incomplete spinal cord injury.

OTHER RECENT NEWS
On March 31st, unprecedented results were announced by StemCells Inc. as they successful concluded the Phase I in Pelizaeus-Merzbacher Disease (PMD), a rare hypo-myelination disorder in children. A summary was presented March 31st at the 2012 European Leukodystrophy Association (ELA) Families/Scientists Meeting in Paris. StemCells Inc. now intends to proceed to Phase II clinical trials. Publication of the detailed data in a peer-reviewed journal will be a significant catalyst for StemCells Inc. Highlights of the results were:

Progressive and durable donor-cell derived myelination in all 4 patients
Small but measureable gains in motor and/or cognitive function in 3 of the 4 patients (the 4th patient remained clinically stable)
After 1 year, MRI showed changes compatible with increased myelination in the region of the transplantation. The MRI signs of myelination persisted even after immunosuppression was stopped at 9 months and in fact, were also found to progress over time.
The development of new myelin signals is unprecedented in patients with conatal PMD and is consistent with HuCNS-SC engraftment.

These results may also be applicable to other leukodystrophies, as well as more common myelin disorders including transverse myelitis, multiple sclerosis and periventricular white matter injury seen in Cerebral Palsy.

Download Full 8-Page Note with Important Disclosures: Morning Note 05-17-12 STEM

17 May 2012

GenSpera (GNSZ) Update 05-17-12

Posted in GenSpera Inc. (GNSZ) | Comments Off

G-202 Phase Ia MTD Clinical Trial Successfully Completed
Phase Ib Now Enrolling in Up To 18 Patients
Phase II Prostate Cancer Trial Planned for Q3
G-202 Unaffected by Changing Competitive Landscape

Download Full 24-Page Report with Important Disclosures: GNSZ Update 05-17-12

1.) G-202 Phase Ia MTD Trial Completed – Phase Ib Now Enrolling: On March 29^th, GenSpera announced the completion of the Phase Ia dose-escalation trial of G-202 and has now commenced enrollment of the Phase Ib trial in up to 18 patients to further refine the dosing regimen and determine a recommended dose for Phase II clinical studies. Specifically, 28 patients were treated in the Phase Ia at doses ranging from 1.2 mg/m²/dose (~2 mg/dose) up to 88 mg/m²/dose (~150 mg/dose). The drug exposure in patients receiving the higher doses of G-202 falls within the range associated with anti-tumor efficacy in animal models. Although the study was not designed to determine the anti-tumor effects of the drug, signs of potential positive effects were observed. GenSpera also stated that they expect to initiate a Phase II study in castrate-resistant, chemotherapy-naïve prostate cancer patients within the next few months. Data from the Phase Ib trial, expected in early 2013, should give additional insight into the efficacy of G-202 in humans. (see G-202 Phase Ia & Ib Human Clinical Trials). GenSpera is also planning on commencing a Phase II trial of G-202 in castrate-resistant chemotherapy-naive prostate cancer patients in the U.S. with additional sites in the U.K. in Q3 2012.

2.) G-202 Still Needed Despite Recent Advances in Prostate Cancer: Although several drugs have recently been approved in prostate cancer such as JEVTANA^®, ZYTIGA™ and PROVENGE^® they have different mechanisms-of-action to GenSpera’s G-202. Eventually, all patients will become refractory to these anti-androgen and chemotherapies while PROVENGE cellular therapy only extended survival by approximately 4.5 months. The new drugs under development also use mechanisms of action that inhibit androgen production or binding to receptors on the cancer cell surfaces such as Medivation’s MDV3100 (an androgen receptor signaling inhibitor) demonstrating a survival benefit of 4.8 months without the use of steroid co-administration (as seen with drugs like ZYTIGA™). However, MDV3100′s anti-cancer effect is still dependent on interrupting androgen signaling. Eventually patients will become resistant to the treatment and the disease will progress. In contrast, G-202 kills independently of androgen pathways and in theory could represent a curative option for advanced prostate cancer patients. (see Competition)

3.) Unique Mechanism of Action (MOA): GenSpera’s drug candidates are based on chemical derivatives of a plant cytotoxin, called thapsigargin, which is a potent inhibitor of the intracellular sarcoplasmic/endoplasmic reticulum calcium ATPase (SERCA) pump. The inhibition of the transport protein causes intracellular Ca2+ (calcium) to rise significantly and trigger apoptosis (cell death). For example, G-202 is a prodrug where the active cytotoxin, 12ADT, is masked by a peptide complex until it binds to and is cleaved by the targeted Prostate-Specific Membrane Antigen (PSMA) thus triggering apoptosis. (see Thapsigargin and Prodrug Delivery of 12ADT). Investors should note a video of GenSpera’s platform technology can be seen at: http://www.genspera.com/investors_mainvideo.html

4.) Attractive Space for M&A: We note that Cougar Biotechnology, a former bulletin board company formed by a reverse-merger in 2006 was acquired by Johnson & Johnson (NYSE:JNJ) in 2009 for $970M in cash based on Phase II data for their prostate cancer drug (ZYTIGA™) which ultimately received FDA approval in April 2011. While partnerships and acquisitions are highly unpredictable, we believe GenSpera could eventually be an attractive candidate as early as Phase II completion should they show strong clinical results.

5.) Capital Raise Expected: Although GenSpera believes they have sufficient cash through March 2013, we expect the company to raise additional funds in preparation for their planned Phase II clinical trials in multiple indications and we have included our estimates in the financial model.

6.) Maintaining Rating of Strong Speculative Buy and $4.00 Target: GenSpera has been successfully progressing “under the radar” for some time now and we believe the company will soon begin attracting wider investor attention. GenSpera’s G-202 with its unique mechanism of action in prostate cancer and other tumor types along with the strong preclinical data and intellectual property provides an intriguing opportunity for savvy investors wanting to get ahead of the curve. Our Strong Speculative Buy rating and 12-18 month Price Target of $4.00 is based on a 35x multiple on projected 2017 earnings and discounted 55% to adjust for risk with a 10% acquisition premium for Phase II results.

Download Full 24-Page Report with Important Disclosures: GNSZ Update 05-17-12

14 May 2012

Navidea (NAVB) Update 05-14-12

Posted in Navidea Biopharmaceuticals (NAVB) | Comments Off

Lymphoseek^® Data Versus Sulfur Colloid at ASCO
Lymphoseek^® FDA PDUFA Date September 10^th
Multiple Catalysts and Multiple Shots-on-Goal

Download Full 35-Page Report with Important Disclosures: NAVB Update 05-14-12

1.) Lymphoseek^® PDUFA Date is September 10^th: Navidea’s first commercial candidate, Lymphoseek^®, is expected to receive FDA approval by the PDUFA date of September 10^th and to be quickly followed by a U.S. launch with partner Cardinal Health (NYSE:CAH). We do not expect the anticipated interim data from Navidea’s ongoing NEO3-06 Phase III trial in Head and Neck squamous cell carcinoma to impact the NDA decision as the interim analysis would not be completed by the PDUFA date of September 10^th. (see Lymphoseek^® Background & Development Program)

2.) Lymphoseek European Filing on Track for Year-End: Navidea reiterated that they expect to file the Lymphoseek Marketing Authorization Application (MAA) to the European Medicines Agency’s (EMA) for review before year-end. Navida also expects to announce partnership(s) this year in order to prepare the European market for an anticpated Lymphoseek launch in 2013. (see Lymphoseek^® Background & Development Program)

3.) Additional Data to be Presented at ASCO: Navidea recently presented data comparing Lymphoseek to sulfur colloid in breast cancer patients showing Lymphoseek Localization Rate (LR) of 99.91% (meta-analysis) and 98.65% (pooled analysis) versus sulfur colloid LR (from peer-reviewed literature) of 94.13%. Lymphoseek’s Degree of Localization (DL) was 2.1 (meta-analysis) and 2.2 (pooled analysis) versus sulfur colloid DL (peer-reviewed literature) of 1.6. All results were statistically significant. Full data on the comparison of Lymphoseek and sulfur colloid will be presented at the American Society for Clinical Oncology (ASCO) Annual Meeting, June 1-5, 2012 in Chicago, IL.

4.) AZD4694 in Alzheimer’s Disease:
Navidea expects to begin Phase III clinical trials in early 2013 for AZD4694, which was in-licensed with worldwide exclusive rights from AstraZeneca (NYSE:AZN). AZD4694 is an Alzheimer’s disease (AD) diagnostic using a Fluorine-18 labeled radiopharmaceutical that binds to β-amyloid deposits in the brain that can then be imaged using a PET (Positron Emission Tomography) scanner. Since the initial indication will be to show negative for β-amyloid pathology and rule out Alzheimer’s disease, we believe AZD4694 will be quickly adopted for early-onset patient diagnosis. (see AZD4694 Alzheimer’s Imaging Background & Development Program)

5.) Possible Candidate for Parkinson’s Disease: Navidea also has an option agreement with Alseres Pharmaceuticals (Pink:ALSE) to license [¹²³I]-E-IAFCT Injection, an Iodine-123 radiolabeled imaging agent used with single photon emission computed tomography (SPECT) imaging, being developed as an aid in the diagnosis of Parkinson’s disease and movement disorders. The option agreement provides for exclusive rights until June 30, 2012 to perform final due diligence with a focus on the regulatory pathway. We note that both Mark Pykett, CEO and Dr. Thomas Tulip, EVP are previously from Alseres. (see CFT (E-IAFCT) Parkinson’s Imaging Background & Development Program)

6.) RIGScan Human MAb Work Continues: Navidea continues development and manufacturing work on a human CC49 monoclonal antibody for RIGScan™ CR, an Iodine-125 radiolabeled agent used with a hand-held gamma radiation detector to warn surgical oncologists, during surgery, to the existence of additional cancerous tissue that would otherwise be undetectable. However, we believe that Navidea will likely partner both the development and distribution of RIGScan before proceeding to Phase III clinical trials. (see RIGScan™ CR Background & Development Program)

Summary: We believe Navidea shares continue to represent a significant investment opportunity and we are maintaining Navidea with a Strong Speculative Buy rating and a 12-18 month price target of $5.75. Our valuation is based on a 35x multiple on projected fiscal year 2016 EPS and discounted 35% for cumulative risk.

Download Full 35-Page Report with Important Disclosures: NAVB Update 05-14-12

14 May 2012

StemCells (STEM) Update 05-14-12

Posted in StemCells Inc. (STEM) | Comments Off

PMD Data in Children Shows Unprecedented Results
Journal Publication Expected to be a Major Catalyst
Interim Spinal Cord Data in Severe Patients this Week
Enrollment to Begin Soon for Dry AMD Phase I/II
Potential CIRM Awards for $20M Each in Summer

Download Full 24-Page Report with Important Disclosures: STEM Update 05-14-12

1.) Unprecedented results were announced by StemCells Inc. as they successful concluded the Phase I in Pelizaeus-Merzbacher Disease (PMD), a fatal myelination disorder in children. Specifically, top-line data showed progressive and durable donor-cell derived myelination was found in all 4 children (investors should note that development of new myelin signals is unprecedented in patients with connatal PMD.) Furthermore, there were small but measureable gains in motor and/or cognitive function in 3 of the 4 children (the 4^th child remained stable). After 1 year, MRI showed changes compatible with increased myelination in the region of the transplantation. The MRI signs of myelination persisted even after immunosuppression was stopped at 9 months and in fact, were also found to progress over time. The detailed data has been submitted to a peer-reviewed journal and we expect publication will be a significant and high-visibility catalyst for StemCells Inc. (see Phase I Human Trial of HuCNS-SC® in Pelizaeus-Merzbacher Disease) StemCells Inc. has also filed for a Strategic Partnership Award with the California Institute for Regenerative Medicine (CIRM) for a Phase II clinical trial in PMD worth up to $10M in funding. Finally, investors should note that these results open the door for StemCells Inc. in more common myelination disorders, such as Multiple Sclerosis and certain forms of Cerebral Palsy.

2.) Interim AIS-A Results for Phase I/II Trial in Chronic Spinal Cord Injury: Armin Curt, MD, FRCPC, Professor and Chairman, Spinal Cord Injury Center at the University of Zurich, and Medical Director of the Paraplegic Center at the Balgrist University Hospital and principal investigator for StemCells Inc.’s Phase I/II clinical trial in chronic spinal cord injury is expected to present interim results for the 1st cohort of chronic spinal cord injury patients (AIS-A) on Thursday, May 17 at the Interdependence 2012 Global SCI Conference in Vancouver, British Columbia. Investors should note that AIS-A patients are the most severe with no motor or sensory function. The AIS-B patient cohort, with less severe injury, is currently being screened and we expect to begin enrollment shortly. (see Phase I Human Clinical Trial HuCNS-SC^® for Spinal Cord Injury) In addition, StemCells Inc. has submitted an application to the California Institute for Regenerative Medicine (CIRM) for development in Cervical Spinal Cord Injuries with UC Irvine for up to $20M million over 4 years with a decision expected this summer.

3.) Phase I/II in Dry AMD Expected to Begin Soon:
On February 2, 2012, the FDA approved StemCells Inc. IND for a Phase I/II trial in Dry Age-Related Macular Degeneration (Dry AMD) and the trial is expected to commence enrollment shortly. The Phase I/II is an open-label dose-escalating trial in 16 patients treating their worst eye with a single injection into the space beneath the retina and results evaluated over the course of 12 months. Investors should note that 85% of all AMD patients currently have the Dry form and 100% of patients with the more serious Wet form progressed from the initial Dry form. The dry form can also cause vision loss without turning into the wet form. (see HuCNS-SC^® for Dry AMD)

4.) Alzheimer’s Collaboration: StemCells Inc.and Frank LaFerla, Ph.D., a world renowned leader in Alzheimer’s disease research, are collaborating to study HuCNS-SC^® human neural stem cells in Alzheimer’s disease. Dr. LaFerla’s has already published research has shown that mouse neural stem cells enhance memory in a mouse model of Alzheimer’s disease and the goal of the collaboration is to replicate these results using StemCells Inc.’s HuCNS-SC human neural stem cells. Dr. LaFerla’s original paper titled “Neural stem cells improve cognition via BDNF in a transgenic model of Alzheimer disease” was published in August 2009 issue of Proceedings of the National Academy of Sciences (PNAS) and the full paper can be accessed free of charge at http://www.pnas.org/content/106/32/13594.full In September 2011, the California Institute of Regenerative Medicine (CIRM) awarded a grant totaling approximately $100,000 for their Alzheimer’s collaboration and they have submitted an application for a research award up to $20M over 4 years with a decision expected this summer.

5.) We are maintaining a Strong Speculative Buy with a Price Target of $4.50: We believe StemCells Inc. has now begun distinguishing itself as the most advanced player in the stem cell space. The extraordinary results showing myelination in children with PMD combined with their advancing clinical programs Chronic Spinal Cord Injury, Dry Age-Related Macular Degeneration and Alzheimer’s Disease provide significant milestones in unlocking the value of their HuCNS-SC^® (Human Central Nervous System Stem Cells). The publication in a peer-reviewed journal of the PMD results is expected to be a major and highly-visible catalyst for StemCells Inc.’s shares. In addition, StemCells Inc. has filed for 2 CIRM research awards, one in Cervical Spinal Cord Injury and one in Alzheimer’s Disease, and each award is worth up to $20M over 4 years with decisions expected this summer along with 2 new Strategic Partnership Award applications worth up to $10M each. We believe StemCells Inc. shares will become a significant leader in the publicly-traded stem cell space during 2012. Our Strong Speculative Buy rating and 12-18 month target price of $4.50 is based on 30x estimated 2016 EPS discounted 55% for cumulative risks in a first-in-class stem cell therapy.

Download Full 24-Page Report with Important Disclosures: STEM Update 05-14-12

02 May 2012

Echo (ECTE) Note 05-02-12

Posted in Echo Therapeutics (ECTE) | Comments Off

Echo Announces Very Strong Symphony^® tCGM Results in Critical Care
Final Feasibility Data Expected This Quarter at ADA Conference
Prelude^® SkinPrep Program to Refocus on Ex-US Markets

Download Full 7-Page Note with Important Disclosures: Morning Note 05-02-12 ECTE

Echo Therapeutics announced very strong results for their Symphony^® tCGM (transdermal continuous glucose monitoring) system from the Feasibility 3A study in Critical Care patients (their lead indication) conducted at Tufts Medical Center in Boston. Results from the ongoing 3B study site are expected to be announced at the American Diabetes Association 72nd Scientific Sessions conference June 8th-12th in Philadelphia.

Investors should note three key takeaways from the Feasibility 3A (Tufts) results:

1.) The Feasibility 3A data indicates that the new Symphony^® tCGM System can successfully monitor Critical Care patients, which is their lead indication. The CG-EGA was 99.6% clinically accurate with 0% benign errors for a combined A+B of 99.6%. These are the best results seen thus far during feasibility testing. In addition, the MARD remained in range at 12.3%.

2.) The Feasibility 3A data agreed with the data seen in the previous Feasibility studies in both healthy and diabetic patients using Prelude^® microabrasion. The Symphony tCGM results appear to be accurate and consistent. The data also indicates that Echo’s non-invasive transdermal biosensor and skin microabrasion technology is equivalent to the competition’s invasive sensor wire systems.

3.) The Symphony^® tCGM System appears safe as there were no adverse events reported.

Study	MARD	CG-EGA A	CG-EGA A+B	SkinPrep	Patients	# of Glucose Readings
Feasibility 3A (Tufts)	12.3%	99.6%	99.6%	Prelude^®Microabrasion	15 Critical Care (Cardiac Surgery)	540
Feasibility 2	12.6%	94.4%	96.9%	Prelude^®Microabrasion	20 Diabetic (Type I&II)	2,600
Feasibility 1	10.5%	98.3%	99.5%	Prelude^®Microabrasion	12 Healthy	1,600
Prototype Pilot 3	12.9%	n/r	97%	Prelude^®Microabrasion	Diabetic	900
Prototype Pilot 2	11.6%	86.4%	100%	Ultrasound	8 Critical Care	147
Prototype Pilot 1	12.5%	89.6%	98.7%	Ultrasound	10 Diabetic	220
Source: Echo Therapeutics

Competition / Product	MARD	CG-EGA A+B	SkinPrep
Echo Symphony^® tCGM	10.5%-12.6%	94.4%-99.6%	Prelude^®Microabrasion
Abbott Freestyle Navigator	9.3%-12.3%	98.0%	None
DexCom SEVEN PLUS	13.0% -15.9%	97.0%	None
Medtronic Guardian RT	15.6%-19.7%	98.9%	None
Source: Echo Therapeutics and LifeTech Capital

MARD: Mean Absolute Relative Difference – Error calculated as the average relative difference between Symphony and the reference measurements

CG-EGA A: Continuous Glucose-Error Grid Analysis A – the clinically accurate A zone of the Clarke error grid

CG-EGA A+B: includes the clinically relevant B zone benign errors of the Clarke error grid

FEASIBILITY 3 (Tufts)
Study Design: The study was performed at Tufts Medical Center and enrolled 15 adult patients scheduled for elective cardiac surgery. The skin of each patient was prepared using Prelude and a Symphony tCGM biosensor was applied to the skin site prior to surgery. Reference blood samples were taken from arterial line catheters at 30-minute intervals and measured on a YSI 2300 STAT Plus Glucose Analyzer. The data collected by Symphony was blinded to study subjects and Tufts clinical staff. At the conclusion of the study period, the test skin sites were inspected for redness or other undesirable effects.

Study Results: Using over 540 Symphony tCGM glucose readings from 15 study subjects paired with reference blood glucose measurements, CG-EGA showed that 99.6% of the readings were clinically accurate and 0% were benign errors with a combined A+B of 99.6%. The MARD for the study was 12.3%. There were no adverse events reported from the Prelude skin preparation or the Symphony tCGM biosensor.

FEASIBILITY 2
Study Design: 20 adult subjects with Type 1 or Type 2 diabetes were evaluated. The skin of each subject was prepared using Prelude and a Symphony tCGM biosensor was applied to the skin site. Venous reference blood samples were taken from intravenous lines at 15-minute intervals for 24 hours and measured on a YSI 2300 STAT Plus Glucose Analyzer. The study data was blinded to study subjects and study personnel. At the conclusion of the 24-hour study period, the test skin sites were inspected for redness or other undesirable effects.

Study Results: Using over 2,600 Symphony tCGM glucose readings from the 20 study subjects paired with reference blood glucose measurements, CG-EGA showed that 94.4% of the readings were clinically accurate and 2.5% were benign errors with a combined A+B of 96.9%. The MARD for the study was 12.6%. Values for blood glucose measurements ranged from 38 to 399 mg/dL. There were no adverse events reported from the Prelude skin permeation or the Symphony tCGM biosensor.

FEASIBILITY 1
Study Design: 12 adult subjects without a history of diabetes were evaluated. The skin of each subject was prepared using Prelude and a Symphony tCGM biosensor was applied to the skin site. Venous reference blood samples were taken from intravenous lines at 15-minute intervals for 24 hours and measured on a YSI 2300 STAT Plus Glucose Analyzer and a commercially available, professional-use glucometer. The study data was blinded to study subjects and study personnel. At the conclusion of the 24-hour study period, the test skin sites were inspected for redness or other undesirable effects.

Study Results: Using over 1,600 Symphony tCGM glucose readings from the 12 study subjects paired with reference blood glucose measurements, CG-EGA showed that 98.3% of the readings were clinically accurate and 1.2% were benign errors with a combined A+B of 99.5%. The MARD for the study was 10.5%. Values for blood glucose measurements ranged from 64 to 212 mg/dL. There were no adverse events reported from the Prelude skin permeation or the Symphony tCGM biosensor.

Download Full 7-Page Note with Important Disclosures: Morning Note 05-02-12 ECTE

Page 1 of 2812 3 4 5 6 7 8 9 10...20...»Last »

Research

StemCells (STEM) Note 05-17-12

GenSpera (GNSZ) Update 05-17-12

Navidea (NAVB) Update 05-14-12

StemCells (STEM) Update 05-14-12

Echo (ECTE) Note 05-02-12

Subscribe to Research Updates Using Twitter / RSS / Email

[ COMPANY INDEX ]

Search Website