Barron's on AVI BioPharma: "Bad Timing for a Promising Stock" $AVII

Shares of AVI BioPharma dropped after results of a key clinical trial disappointed Wall Street. A partnership with a big drug firm could revive the stock.

From Barron's:

"The ink had barely dried on our latest issue when AVI BioPharma (ticker: AVII), the subject of a bullish article ("Waiting and Hoping," April 2), released data early Monday morning from a key clinical trial for its drug to treat Duchenne muscular dystrophy. The news was good, but not good enough to meet elevated expectations on Wall Street.

AVI's lead drug, called eteplirsen, is doing better than the stock, which could reward patient investors.

See the full story @ Barron's.

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NeoStem Closes Public Offering for $6,800,000 in Gross Proceeds ($NBS)

NeoStem, Inc. (AMEX: NBS; Stock Twits: $NBS) is engaged in the development and manufacturing of cell-based therapies in the U.S.  Its January 2011 acquisition of Progenitor Cell Therapy, LLC ("PCT") is central to the Company's strategic mission of capturing the paradigm shift to cell therapy.  The acquisition of PCT gives NeoStem not only access to a world class contract manufacturing cell therapy company but provides a platform and expertise around the evaluation, development and regulatory requirements to develop autologous, allogeneic, immunomodulatory and vaccine-based therapeutics. NeoStem also holds the worldwide exclusive license to VSEL(TM) Technology, which uses very small embryonic-like stem cells, shown to have several physical characteristics that are generally found in embryonic stem cells, and is pursuing the licensing of other technologies for therapeutic use.

The Company has announced "the closing of its previously announced underwritten public offering of 15,000,000 units and the exercise of the over-allotment option by the underwriter for an additional 2,000,000 units, bringing the total units offered to 17,000,000. The offering was priced at $0.40 per unit. Each unit consists of one share of common stock and a warrant to purchase one share of common stock with a per share exercise price of $0.51. Maxim Group LLC acted as sole book-running manager."

Dr. Robin Smith, NeoStem's Chairman & CEO, commented, 

"NeoStem's management remains focused on our key objectives of expanding our stem cell therapeutic contract manufacturing business, enrolling the PreSERVE AMR-001 Phase 2 clinical trial for preserving heart function after a heart attack and monetizing our China pharmaceutical subsidiary through divestiture."

The offering resulted in gross proceeds of $6,800,000, "prior to deducting underwriting discounts and commissions and offering expenses payable by the Company. These funds will be used for working capital purposes, including research and development of cell therapeutic product candidates, expansion of business units, strategic transactions and other general corporate purposes."

Read the full release at Neostem.com. 

Aastrom ($ASTM): Molecular Therapy Publishes Data for RESTORE-CLI

Aastrom Biosciences (NASDAQ: ASTM) announced that final results from the company's RESTORE-CLI Phase IIb clinical trial for ixmyelocel-T were published in the peer-reviewed journal MolecularTherapy. The Phase IIb clinical results demonstrated that treatment with ixmyelocel-T improved time to treatment failure in patients with critical limb ischemia (CLI) compared to the control group, and in the subgroup of patients with wounds at baseline demonstrated an improvement in amputation free survival. A total of 48 patients were treated with ixmyelocel-T and 24 received a placebo. Adverse event rates in both groups were similar. Patients in the treatment arm showed a 62% reduction in risk relative to placebo in the primary efficacy endpoint of time to first occurrence of treatment failure. A post hoc analysis of the subgroup of 45 patients with wounds at baseline resulted in a 77% risk reduction in time to first occurrence of treatment failure and a positive trend in the Phase III endpoint of amputation-free survival.

Daily Dose Take-Away: This data clearly suggests that ixmyelocel-T (expanded bone marrow) is active and impact the course of the disease. The Phase 3 trial is now funded and well powered. While its been a long road, Aastrom fundamentals are moving in the right direction.

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Rodman and Renshaw update on $KERX

Perifosine Strikes Out – Zerenex at Bat – A Potential Home Run Opportunity

Rodman and Renshaw update on KERX (PDF)
 
Summary: X-PECT Trial Fails to Meet Primary Endpoint – Removing All Perifosine Revenues from Model Today, Keryx announced that the Phase 3 X-PECT trial for the treatment of metastatic colorectal cancer failed to meet its primary endpoint of overall survival (OS). During the conference call, management elaborated that the median OS observed in the control arm of capecitabine + placebo was significantly higher than any other historical control treatment investigated in other randomized trials for the similar patient population. Additionally, further analysis revealed no benefit of perifosine + capecitabine treatment in the KRAS wild type or mutant population, or any other subgroup of patients. No further details regarding the difference in the survival between the treatment and the control arm, or the toxicities related to the treatment were provided. The management further indicated that given the failure of the X-PECT trial, the recruitment in the Phase 3 multiple myeloma trial may be affected, and the company is contemplating whether to continue the Phase 3 trial of perifosine with Velcade and dexamethasone in relapsed / refractory multiple myeloma patients. In our opinion, the company will likely shut down all clinical programs involving perifosine in an effort to conserve cash.
 
Phase 3 Zerenex Trial for Hyperphosphatemia to Report in 4Q12 During the call, the management also reminded investors about the ongoing Phase 3 trial evaluating Zerenex for the treatment of hyperphosphatemia in end-stage renal disease (ESRD) patients. Keryx is conducting a 58-week long-term, multicenter, randomized, open-label, safety and efficacy Phase 3 trial evaluating Zerenex, a phosphate binder, in 440 patients with ESRD on dialysis. During the call today, management reiterated that the data from the Phase 3 trial is expected to be available in 4Q12, with potential filing of the NDA and MAA in 1H13. In our opinion, the long-term study of Zerenex has a higher than average likelihood to succeed given that the short-term Phase 3 trial of Zerenex in ESRD patients met its primary endpoint of demonstrating a dose response in the change of serum phosphorous from baseline to day 28 (p-value < 0.0001). Of additional significance, Zerenex may reveal additional benefits in combating anemia if the Phase 3 data demonstrate a reduced need for intravenous and/or EPO blood cell stimulating drugs such as Procrit, Epogen and Aranesp.
 
Zerenex Market Potential We would like to remind investors that the US market for phosphate binders in the dialysis setting is approximately $700 MM and worldwide the market is approximately $1.2 BN. The 25% year-over-year growth in the phosphate binding market over the past 5 years has been driven by an increased incidence of dialysis with a growing diabetic population, as well price increases from market leader Genzyme for Renagel and Renvela. We believe that Zerenex has the potential to address the deficiencies among marketed phosphate binders, and capture 14% of the hyperphosphatemia market, corresponding to peak US revenues of approximately $250 MM by 2018, if successful.
 
Quick Take We are reiterating our Market Outperform / Speculative Risk rating but lowering our target price of $8 to $3 based on a discounted 2015 revenues and earnings multiples analysis. In our opinion, the failure of perifosine is a temporary setback, and provides an opportunity to a value-oriented investor to invest in the potential of the Zerenex franchise. The potential cost-savings benefit, combined with previous efficacy data, makes Zerenex an attractive treatment option in the approximately $700 MM U.S. market for agents that lower phosphate in ESRD patients on dialysis. We believe that Keryx represents a significantly undervalued and underappreciated company and is potentially suitable for the risk-oriented investor.

Derma Sciences ($DSCI) Raises $19.7 million

Derma Sciences (NASDAQ: DSCI) raised $19.7 million through the sale of 2.1 million shares at $9.25 in a follow-on underwritten by Piper Jaffray. Derma plans to begin Phase III testing next half of DSC127 for diabetic foot ulcers. The compound is an angiotensin analog. Derma also markets dressings for wounds and burns.  

DSC127 is an analog of a naturally occurring peptide, Angiotensin. It has been shown to increase keratinocyte proliferation, increase extracellular matrix production, and increase vascularization. Additionally, histological examination has shown that DSC127 accelerated collagen deposition six-fold. All these help to accelerate dermal tissue repair. One potential method of action is the up-regulation of mesenchymal stem cells (MSCs) at the site of injury. MSCs originate in the human embryo and are considered to be multipotent — a type of stem cell that has not yet adopted a specific cellular phenotype. Such cells have the ability to differentiate into various types of cells found within the human body, including fibroblasts, adipose cells, muscle cells, bone cells, and skin cells.

The patented amino acid peptide DSC127 optimizes the well published wound healing capabilities of Angiotensin while removing all blood pressure effects of the compound.

Extensive pre-clinical studies have demonstrated the efficacy of the compound in accelerating healing and reducing scar formation. Pre-clinical studies thus far have shown:

• Improved in-growth of host tissue into artificial skins
• Accelerated healing in full thickness skin excision wounds in rats and diabetic mice
• Accelerated healing in partial thickness thermal injuries in guinea pigs
• Accelerated healing in a random flap skin model in rats Improved scar reduction in rats

Phase 1 is completed and top line Phase 2 data appears to have been presented last fall. The next step will be pivotal (phase III) studies.

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AlloCure (private) Raises $25 million in series B for Cell Therapy in Kideny Disease

Allocure Inc. (Burlington, Mass.) raised $25 million in a series B round. New investor Lundbeckfond Ventures joined existing investors SV Life Sciences and Novo A/S in the round. AlloCure plans to start Phase II testing this summer of AC607 to treat acute kidney injury.

AC607 is a novel biologic therapy under development for the treatment of AKI. AC607 also possesses potential applications in other grievous illnesses. AC607 comprises allogeneic bone marrow-derived mesenchymal stem cells that are harvested from healthy adult donors and then expanded via a mature and state-of-the art manufacturing process. AC607 homes to the site of injury where it mediates powerful anti-inflammatory and organ repair processes via the secretion of beneficial paracrine factors, without differentiation and repopulation of the injured kidney. Importantly, AC607 avoids recognition by the host's immune system, enabling administration in an "off the shelf" paradigm without the need for blood or tissue typing.

The capital just raised will be used to support the next step of clinical development: "Building on the encouraging AC607 Phase 1 results presented last fall at the American Society of Nephrology annual meeting, AlloCure is poised to conduct a multicenter, randomized, double-blind, placebo controlled trial of AC607 for the treatment of AKI beginning this Summer at a number of the leading academic institutions and hospitals in the United States.

StemCells ($STEM) Announces webcast to discuss Pelizaeus-Merzbacher disease clinical trial results

A summary of the trial results will be presented on Saturday, 31-Mar, at the 2012 European Leukodystrophy Association (ELA) Families/Scientists Meeting in Paris:

• Webcast: Monday, 2-Apr, at 11:00ET 
• http://investor.stemcellsinc.com/phoenix.zhtml?c=86230&p=irol-irhome

BackGround: What is Pelizaeus-Merzbacher Disease (PMD) ?

PMD is a myelination disorder that primarily affects young children. It is a rare central nervous system disorder in which coordination, motor abilities, and intellectual function are delayed to variable extents. In November of 2009 STEM began a trial at the University of California, San Francisco (UCSF) Benioff Children's Hospital. In February 2010 the cells were used to treat the first patient enrolled in the trial, marking the first time that neural stem cells have been transplanted as a potential treatment for a myelination disorder.  In February 2011, the fourth and final patient was enrolled and dosed.  Results of the trial will be reported on Monday (see link above).

This Phase I trial has been designed to assess the safety and preliminary efficacy of STEM's HuCNS-SC cells as a potential treatment for PMD. While the primary focus in this first trial is safety, the company is looking for evidence of new myelin formation in the patients’ brains following the transplantation of the cells, as well as any signs of improved neurological function.

The four enrolled patients have connatal PMD, the most severe form of the disease. All patients were transplanted with the HuCNS-SC cells and were evaluated regularly over a 12-month period in order to monitor and evaluate the safety and tolerability of the HuCNS-SC cells, the surgery and the immunosuppression. In addition, MRI examination of the brain post-transplant should enable the measurement of new myelin formation. The company is hoping to follow the effects of this therapy long-term, so,we expect a four-year observational study to be reviewed.

Daily Dose Conclusion: This is cutting edge science. Safety come first but STEM is moving in areas that represent totally unmet medical needs. The tough financing environment as hurt the whole stem cell sector but the science is marching forward. STEM remains a pioneer with their HuCNS-SC technology.

Myelination Disorders / PMD (continued)

The gene mutations responsible for PMD result in improperly produced or too much proteolipid protein (PLP), which proves toxic to oligodendrocytes, the CNS cells that produce myelin. Myelin, comprised of fats, cholesterol and protein, is critical to healthy functioning of the central nervous system because it provides the insulation needed for proper transmission of nerve impulses.

In myelination disorders, the deficient myelin sheath does not properly insulate the axon, so transmission of nerve impulses is impeded.

Neuron with deficient myelin

The StemCells Approach: Myelin Production to Protect Nerve Cells

When StemCells human neural stem cells are transplanted in animals, they migrate to the sites where myelin is deficient. They differentiate into oligodendrocytes, which form healthy myelin sheaths to protect axons, helping nerve cells communicate with each other. They do this by developing myelin appendages that wrap around the axons of nearby neurons to provide the insulation (myelin) needed for proper transmission of nerve impulses.

HuCNS-SC^® Cell

Oligodendrocyte forms healthy myelin sheath around axon.

Neuron

Oligodendrocyte

Normal transmission of nerve impulses.

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Islet Sciences Announces DiaKine Therapeutics' Novel Diabetes - $ISLT

Islet Sciences, Inc., (OTCBB: ISLT) is a biotechnology company engaged in the research, development and commercialization of patented technologies in the field of transplantation therapy for patients with diabetes.The company is based on prior technology that originated from islet biosciences. The company is pioneering the use of porcine (pig) islet cells encapsulated in an algenate bubble and transplanted into type 1 diabetics that will then function to produce insulin. The potential of this therapy to change the diabetes paradigm could be dramatic.

Today they announced that its subsidiary DiaKine Therapeutics Inc. received European patent protection. The drugs are small molecules that can be used to treat diabetes, atherosclerosis and other inflammatory diseases.

The European patent (#1919867), issued on March 16, 2012, covers the composition and methods of use of orally active drugs with a novel mechanism to reduce autoimmunity and inflammation. In particular, the small molecules have the potential to protect insulin-producing cells from inflammation, reduce insulin resistance, and prevent or treat cardiovascular disease, making them ideally suited for the treatment of type 1 and type 2 diabetes, atherosclerotic cardiovascular disease and other conditions associated with activation of the body's Interleukin 12/STAT4 Pathway.

"Inflammation is a major link associated with the development of diabetes and its complications," said Dr. Jerry L. Nadler, the company's chief science officer and chairman of the board. "This European patent strengthens DiaKine's ability to address diabetes and to meet a medical need for the treatment other inflammatory diseases, such as atherosclerosis and autoimmune disorders."

"This patent issuance is a clear testament to the value of our therapies which represents a large market opportunity for Islet Sciences," said John Steel, Chairman and CEO of Islet Sciences. We are pleased to announce this milestone which clearly strengthens our value proposition."

The compounds were first developed at the University of Virginia and licensed to DiaKine by the U.Va. Patent Foundation (now the U.Va. Licensing & Ventures Group) http://www.innovation.virginia.edu.

See the full press release @ Yahoo Finance