U.S. may speed approval of "breakthrough" drugs

Under a proposal likely to become law later this year, U.S. drug regulators would be able to  label promising treatments as "breakthorugh" therapies to speed their path to approval. 

At Reuters.com, journalist Anna Yukhananov writes that the proposal would allow drug regulators to "work with companies to speed up clinical trials, for example by testing the drugs for a shorter time or enrolling fewer patients...The plan fits with President Barack Obama's aim to foster innovation as a means of spurring job creation, and may pacify some critics who say the FDA lags European countries in approving new medicines or medical devices."

Yukhananov quoted Dr. Janet Woodcock, head of the FDA's drugs center, who in March asked reporters, "What happens when you have a breakthrough drug that shows an effect that's never been seen before? If we'd done business as usual during the AIDS epidemic, we would have never controlled that epidemic."

Also quoted was Jeff Allen, executive director of Friends of Cancer Research, who commented, "The most promising drugs show an effect early. [But] there's a mentality mong drug developers or FDA reviewers that you have to go through this multi-step approach" to get a drug to market. We're hoping to encourage getting away from that."

Read more at Reuters.com. 

6 Low Debt Pharmaceutical Stocks Rated Buy Or Better @ Seeking Alpha (KERX, VTUS)

At Seeking Alpha, contributor ZetaKap wrote an article asking, "Interested in pharmaceutical companies? In search of companies that can manage their debt well? Do you prefer companies that can manage their long term debt? Are you after stocks that analysts are calling 'buy' or 'strong buy'? If so, you'll probably like this list."

Two of the six companies listed were Keryx Biopharmaceuticals, Inc. (NASDAQ: KERX) and Ventrus Biosciences (NASDAQ: VTUS).

About Keryx, ZetaKap wrote,

"Keryx Biopharmaceuticals Inc. has a Debt/Equity Ratio of 0.00 and Long Term Debt/Equity Ratio of 0.00 and Analysts' Rating of 1.80. The short interest was 13.77% as of 05/12/2012. Keryx Biopharmaceuticals, Inc., a biopharmaceutical company, together with its subsidiaries, focuses on the acquisition, development, and commercialization ofpharmaceutical products for the treatment cancer and renal disease. The company's products under development include KRX-0401 (perifosine), an oral anti-cancer agent that inhibits Akt activation in the phosphoinositide 3-kinase (PI3K) pathway, as well as affects other pathways associated with programmed cell death, cell growth, cell differentiation, and cell survival. Its KRX-0401 is in Phase III clinical development stage for the treatment of refractory advanced colorectal cancer and multiple myeloma, as well as in Phase I and Phase II clinical development stages for the treatment of other tumor types."

About Ventrus Biosciences, ZetaKap writes,

"Ventrus Biosciences Inc. has a Debt/Equity Ratio of 0.00 and Long Term Debt/Equity Ratio of 0.00 and Analysts' Rating of 1.40. The short interest was 3.14% as of 05/12/2012. Ventrus Biosciences, Inc., a development stage specialty pharmaceutical company, focuses on the development and commercialization of late-stage prescription drugs for gastrointestinal disorders, primarily hemorrhoidal disease, anal fissures, and fecal incontinence. It is developing three late-stage product candidates, including Iferanserin ointment (VEN 309) for the topical treatment of symptomatic internal hemorrhoids; Diltiazem cream (VEN 307), a topical treatment for the relief of pain associated with anal fissures; and Phenylephrine gel (VEN 308) for the treatment of fecal incontinence associated with ileal pouch anal anastomosis, an FDA orphan indication. The company was formerly known as South Island Biosciences, Inc."

Read the full article at SeekingAlpha.com.  

FDA Advisory Committee Supports Approval of Gilead’s Truvada® for Reducing the Risk of Acquiring HIV (GILD)

Gilead Sciences, Inc. (Nasdaq:GILD) is a biopharmaceutical company that discovers, develops and commercializes innovative therapeutics in areas of unmet medical need. The company’s mission is to advance the care of patients suffering from life-threatening diseases worldwide. Headquartered in Foster City, California, Gilead has operations in North America, Europe and Asia Pacific.

The Company announced that the FDA's Antiviral Drugs Advisory Committee "has voted to support approval of once-daily oral Truvada® (emtricitabine and tenofovir disoproxil fumarate) to reduce the risk of HIV-1 infection among uninfected adults, an HIV prevention strategy called pre-exposure prophylaxis or PrEP. If the FDA decides to approve Truvada for PrEP, it would be the first agent indicated for uninfected individuals to reduce their risk of acquiring HIV."

In response to questions posed to the committee, "members voted 19 to 3 in favor of approval for Truvada for PrEP in men who have sex with men; 19 to 2 (with 1 abstaining) in support of use in HIV-uninfected partners in serodiscordant couples; and 12 to 8 (with 2 abstaining) in other individuals at risk for acquiring HIV through sexual activity."

Although the committee's recommendations are not binding, the FDA will consider them as the agency completes its six-month priority review of Gilead’s supplemental New Drug Application (sNDA) of Truvada for PrEP. Gilead submitted the sNDA on December 15, 2011 and the FDA has established a target review date under the Prescription Drug User Fee Act (PDUFA) of June 15, 2012.

The committee’s positive recommendation followed presentations today of efficacy and safety data from several clinical studies of Truvada for PrEP, including two large placebo-controlled Phase 3 trials sponsored by the U.S. National Institutes of Health and the University of Washington, respectively. Several other clinical studies support the use of Truvada for HIV risk reduction.

Truvada was approved by the FDA in 2004 for the treatment of HIV-1 infection and is currently the most-prescribed antiretroviral treatment in the United States. Truvada is not indicated for HIV prevention.

Read more at investors.gilead.com. 

Cytomedix says proposed National Coverage Determination memo by CMS includes proposal through CED for all three major wound categories; conference call May 10 (CMXI)

Cytomedix, Inc. (CMXI.OB) is a biotechnology company developing advanced tissue regeneration technologies. Its primary focus is on commercializing autologous cell based therapies that facilitate the body’s natural healing processes for enhanced healing and tissue repair.  Its commercial portfolio is based on autologous platelet rich plasma (“PRP”) platform technology, and includes the Angel® Whole Blood Separation System (“Angel®”) and the AutoloGelTM System (“AutoloGel”). 

The company has said that "the Centers for Medicare & Medicaid Services issued a proposed National Coverage Determination memo for autologous blood-derived products for chronic non-healing wounds. In its decision memo CMS has proposed coverage through its Coverage with Evidence Development program for all three major wound categories: diabetic, venous and pressure wounds."

In the CED process, CMS provides payment for items and services while generating additional clinical data to demonstrate their impact on health outcomes. Cytomedix says that the proposed decision by CMS bodes well for its ongoing discussions with a top 20 global pharmaceutical company for an exclusive U.S. supply and distribution agreement for AutoloGel in wound care.

A Company conference call was held 10-May at 9:00 ET to discuss the proposed NCD and the CED initiatives. A replay of the conference call will be available beginning two hours after its completion through May 17, 2012 by dialing 888-286-8010 (domestic) or 617-801-6888 international) and entering passcode 61561051. The call will also be archived for 90 days at www.streetevents.com, www.fulldisclosure.com and www.cytomedix.com.

Read more at CNN Money. 

Aastrom Biosciences Announces First Patient Enrolled in REVIVE Phase 3 Clinical Trial of Ixmyelocel-T (ASTM)

Aastrom Biosciences, Inc. (Nasdaq:ASTM) is the leader in developing patient-specific, expanded multicellular therapies for use in the treatment of patients with severe, chronic cardiovascular diseases. The company's proprietary cell-processing technology enables the manufacture of ixmyelocel-T, a patient-specific multicellular therapy expanded from a patient's own bone marrow and delivered directly to damaged tissues. Aastrom has advanced ixmyelocel-T into late-stage clinical development, including a Phase 3 clinical program to study patients with critical limb ischemia and a planned Phase 2b clinical trial in patients with ischemic dilated cardiomyopathy. 

The Company announced that it has enrolled its first patient in the REVIVE Phase 3 clinical trial of ixmyelocel-T. The REVIVE study is currently underway in the United States to assess the efficacy and safety of ixmyelocel-T in the treatment of no option patients with critical limb ischemia (CLI).

The REVIVE trial is "the largest randomized, double-blind, placebo-controlled, multicenter study ever conducted in patients with CLI. The Phase 3 trial has 80 treatment centers qualified to enroll patients and will include 594 CLI patients who have no option for revascularization and also have existing tissue loss due to ischemia. Patients will be followed for a total of 18 months, including 12 months from randomization for efficacy and an additional six months for safety. The primary endpoint of the trial will be amputation-free survival at 12 months."

Tim Mayleben, president and chief executive officer at Aastrom Biosciences, commented,

"The enrollment of the first patient in the REVIVE clinical trial represents another important milestone in our effort to advance the development program for ixmyelocel-T through the final stages of clinical development. We are grateful to our investigators for their commitment to enrolling patients as quickly as possible in this important trial."

Read more at investors.aastrom.com. 

Compassionate Use of Pluristem's PLX Cells Saves the Life of a Child After Bone Marrow Transplantation Failure (PSTI)

Pluristem Therapeutics Inc. (NasdaqCM: PSTI; TASE: PLTR) is a leading developer of placenta-based cell therapies. The company's patented PLX (PLacental eXpanded) cells drug delivery platform releases a cocktail of therapeutic proteins in response to a variety of local and systemic inflammatory diseases. PLX cells are grown using the company’s proprietary 3D micro-environmental technology and are an off-the-shelf product that requires no tissue matching or immune-suppression treatment prior to administration. PLX-PAD comprehensive clinical development plan has been recognized by both the EMA and FDA, targeting a sub-population of 20-million patients of Peripheral Artery Disease (PAD) market. 

The Company has announced that "a seven year-old girl suffering from an aplastic bone marrow whose condition was rapidly deteriorating is now experiencing a reversal of her condition with a significant increase in her red cells, white cells and platelets following the intramuscular injection of the company's PLacental eXpanded (PLX) cells."

Patients with Aplastic bone marrow have no blood-forming hematopoietic stem cells in their bone marrow.

Professor Reuven Or, Director of Bone Marrow Transplantation, Cell Therapy and Transplantation Research Center at Hadassah Medical Center and the child's physician, explained, 

"With her body rejecting all possible treatment -- and with no other options -- we finally turned to Pluristem's PLX cells, which literally saved her life. The results of this unique case indicate that PLX cells may be effective in treating other diseases that affect the bone marrow."

Zami Aberman, Chairman and CEO of Pluristem, commented,

"Pluristem is extremely happy that our PLX cells have helped this little girl. Remarkably, these beneficial effects were seen in the patient after our PLX cells were administered intramuscularly and correlates with the positive effects on the bone marrow when we administered our PLX cells intramuscularly (IM) in animals exposed to toxic levels of radiation. Pluristem now has several data points to indicate that our PLX cells may work for systemic diseases when given locally, away from the target organ, and without a need to give cells intravenously."

In February 2012, Pluristem announced the results of animal studies which suggest that "PLX cells can be potentially effective in treating the life threatening hematopoietic complications associated with Acute Radiation Syndrome (ARS)." In the experiments in question, animals were given PLX cells IM up to 24 hours post irradiation. They demonstrated a recovery of their red cells, white cells, platelets and bone marrow to almost normal levels. This announcement, along with his patient's significant deterioration following two bone marrow transplants, that led Professor Reuven Or to contact Pluristem about the possible compassionate use of PLX cells to treat his young patient.

Pluristem recently received U.S. FDA Clearance "to begin a Phase II clinical trial using the company's proprietary PLX-PAD cell product candidate intramuscularly for the treatment of Intermittent Claudication (IC), a subset of peripheral artery disease (PAD). In April, the Company was awarded a $3.1 Million grant by the Israeli Government, which will be used to help fund R&D and clinical trials."

This news could have positive implications for Athersys who is planning to pursue a phase II registrational style trial in GvHD prevent. There are also implications for Osiris. 

Read the full press release at Yahoo Finance. 

Islet Sciences Announces Exclusive License Agreement with Yale University to Commercialize an Early Beta Cell Destruction and Diabetes Diagnostic ($ISLT)

Islet Sciences, Inc. (OTCBB: ISLT) is a development-stage biotechnology company with patented technologies focused on transplantation therapy for people with insulin-dependent diabetes. The Company's transplantation technology includes methods for the culturing, isolation, maturation, and immuno-protection (microencapsulation) of islet cells. Islet Sciences’ mission includes the introduction of commercial products with applications to cell-based replacement therapy in the healthcare marketplace.

 The Company announced that it has exclusively licensed technology from Yale University.

Chairman and CEO of Islet Sciences John Steel stated, 

"In the course of research conducted under Yale auspices, Dr. Kevan Herold and his colleagues at Yale University School of Medicine have developed a technology entitled 'Circulating hypomethylated B cell-derived DNA' as a biomarker of B cell destruction. We are excited to utilize this groundbreaking invention as we look to commercialize it to benefit the growing worldwide diabetic community. This test has the potential to identify beta cell death and emerging diabetes far earlier than clinical presentation."

Dr. Kevan Herold added, 

"The opportunity to identify diabetes and islet dysfunction prior to clinical presentation could be beneficial therapeutically. I am looking forward to working with Islet Sciences to commercialize this technology that clearly holds so much potential."

Clusters of beta cells form islets within the pancreas. Diminished beta/islet cell function is one of the primary causes of insulin dependent diabetes and can be caused by numerous factors. Insulin dependent diabetes is the more difficult aspect of the disease.

Jonathan Soderstrom, director of the Office of Cooperative Research at Yale commented,

"We are pleased that Islet Sciences has made the decision to invest in this discovery and look forward to developing it into a valuable new tool for treating this debilitating disease."

Read more at Yahoo Finance. 

3-D printing, bioprinting pushing the edges of regenerative medicine ($ONVO)

Organovo Holdings, Inc. (OTC: ONVO) is focused on delivering breakthrough three-dimensional biology capabilities to create tissue on demand for research and surgical applications. As the first company with a three-dimensional tissue technology that works across tissue types, we are meeting the promise of regenerative medicine to fill unmet medical needs. Organovo's powerful NovoGen Bioprinting platform creates human tissues starting with any cell source. From disease models to tissue creation, bioprinting solves urgent needs in biological research. 

On Cleveland.com, contributor Brie Zeltner wrote an article about the idea behind bioprinting, its applications, and some the companies and researchers working on it. She writes,

"Imagine a custom-made knee replacement modeled to fit exactly from scans of your own bone, built one infinitesimal layer at a time by a printer that uses 'ink' made from living cells. Or a replacement heart valve, seeded with human stem cells, printed in a single functioning piece. It may sound like science fiction, but these feats of bioengineering are not far from being realized."

She continues,

"Organovo Inc., a San Diego-based tissue engineering company, is using printers to make small amounts of human tissue, including muscle and blood vessels. With the printer precisely placing cells, the team is able to create tissue that is almost indistinguishable from the real thing under a microscope. And pharmaceutical companies are using the printed tissue to test their drugs, saving money by identifying ones that would fail early in clinical trials. The collaboration will fund their continued research."

Read the full article at Cleveland.com.