Neuralstem, Inc. (NYSE Amex: CUR) announced that its Phase I trial to treat Amyotrophic Lateral Sclerosis (ALS or Lou Gehrig’s disease) with its spinal cord stem cells has been approved by the Institutional Review Board (IRB) at Emory University in Atlanta, GA. The trial, which was approved by the FDA in September, will take place at the Emory ALS Center, under the direction of Dr. Jonathan Glass M.D., Director of the Emory ALS Center, who will serve as the site Principal Investigator (PI).

The trial will study the safety of Neuralstem’s cells and the surgical procedures and devices required for multiple injections of Neuralstem’s cells directly into the grey matter of the spinal cord.

“The commencement of the first trial using our stem cells, and the first ALS stem cell trial in the U.S., represents a significant step in regenerative medicine,” said Richard Garr, Neuralstem CEO. “We look forward to working with the Emory ALS Center. We expect to begin treating patients with our stem cells in January. Again, patients who are interested should reach out directly to the Emory ALS Center.”

About the Trial

This Phase I trial, which will primarily evaluate safety of the cells and the surgery procedure, will ultimately consist of 18 ALS patients with varying degrees of the disease, who will be treated with spinal injections of Neuralstem’s patented human neural stem cells. The FDA has approved the first stage of the trial, which consists of 12 patients who will receive five-to-ten stem cell injections in the lumbar area of the spinal cord. The patients will be examined at regular intervals post-surgery, with final review of the data to come about 24 months later.

In addition to Dr. Glass, site PI at Emory, the overall PI for the Neuralstem ALS trial program is Dr. Eva Feldman, M.D., Ph.D., Director of the University of Michigan Health System ALS Clinic and the Program for Neurology Research & Discovery.

About Neuralstem, Inc.

Neuralstem’s patented technology enables, for the first time, the ability to produce neural stem cells of the human brain and spinal cord in commercial quantities, and the ability to control the differentiation of these cells into mature, physiologically relevant human neurons and glia. The company is targeting major central nervous system diseases including: Ischemic Spastic Paraplegia, Traumatic Spinal Cord Injury, Huntington’s disease and Amyotrophic Lateral Sclerosis (ALS), often referred to as Lou Gehrig’s disease. Neuralstem plans to initiate a Phase I clinical trial to treat ALS with its stem cells. ALS is a progressive fatal neurodegenerative disease that affects nerve cells in the brain, leading to the degeneration and death of the motor neurons in the spinal cord that control muscle movement. Pre-clinical work has shown Neuralstem’s cells to extend the life of rats with ALS (as reported in the journal TRANSPLANTATION, October 16, 2006, in collaboration with Johns Hopkins University researchers), and also reversed paralysis in rats with Ischemic Spastic Paraplegia, (as reported in NEUROSCIENCE, June 29, 2007, in collaboration with researchers at University of California San Diego).

Cautionary Statement Regarding Forward Looking Information

This news release may contain forward-looking statements made pursuant to the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Investors are cautioned that such forward-looking statements in this press release regarding potential applications of Neuralstem’s technologies constitute forward-looking statements that involve risks and uncertainties, including, without limitation, risks inherent in the development and commercialization of potential products, uncertainty of clinical trial results or regulatory approvals or clearances, need for future capital, dependence upon collaborators and maintenance of our intellectual property rights. Actual results may differ materially from the results anticipated in these forward- looking statements. Additional information on potential factors that could affect our results and other risks and uncertainties are detailed from time to time in Neuralstem’s periodic reports, including the annual report on Form 10-K for the year ended December 31, 2008 and the quarterly report on form 10-Q for the period ended September 30, 2009.

Source: Neuralstem, Inc

Celldex Therapeutics, Inc. (NASDAQ: CLDX) announced the results of a positive Phase 2 study of CDX-011 (formerly CR011-vcMMAE), in patients with heavily pre-treated, locally advanced or metastatic breast cancers. As presented today at the 32nd Annual CTRC-AACR San Antonio Breast Cancer Symposium, the primary efficacy endpoint for the study has been met with significant antitumor activity in patients whose tumors express the target GPNMB. In addition, encouraging results were seen in patients with “triple-negative disease” where treatment options are relatively limited due to lack of hormone receptor or HER2-neu expression.

CDX-011 is the first candidate from the Company’s antibody-drug conjugate (ADC) platform, which utilizes fully human monoclonal antibodies to deliver the potent cellular toxin, MMAE, directly to tumor cells by targeting GPNMB. GPNMB is a glycoprotein frequently expressed in a number of tumor types and associated with cancer progression and recurrence. CDX-011 uses the Seattle Genetics MMAE ADC technology, which has established a very promising antitumor effect in advanced clinical studies.

“As seen in this study, treatment with CDX-011 can induce disease regression and stabilization,” said Thomas Davis, M.D., Senior Vice President and Chief Medical Officer of Celldex Therapeutics. “We are very encouraged to see such positive results in patients with triple negative disease - or those with advanced, refractory and heavily pre-treated breast cancers — where there is a clear unmet medical need. Moving forward, we are planning expanded Phase 2 development focused on patients with tumors expressing GPNMB.”

The Phase 2 study investigated the safety, tolerability and efficacy of CDX-011 in locally advanced or metastatic breast cancer patients who were heavily pre-treated (median of seven prior regimens). The study confirmed the safety of CDX-011 at the pre-defined maximum dose level (1.88 mg/kg) in 6 patients. An additional 28 patients were enrolled as an expanded Phase 2 cohort (for a total of 34 treated patients at 1.88 mg/kg, the Phase 2 dose) to evaluate the progression-free survival (PFS) rate at 12 weeks. As previously seen in melanoma patients, the 1.88 mg/kg dose was well tolerated in this patient population. The primary activity endpoint, which called for at least 5 of 25 (20%) patients in the Phase 2 study portion to be progression-free at twelve weeks, has been met. To date, 9 of 26 (35%) evaluable patients are without progression of disease at twelve weeks.

In addition, at the Phase 2 dose level, 4 of 32 (13%) evaluable patients achieved confirmed or unconfirmed Partial Responses (PR) while 15 of 25 (60%) evaluable patients with measurable disease experienced some reduction in tumor size. GPNMB expression was identified in 10 of 14 (71%) of analyzed tumor samples and treatment with CDX-011 was associated with improved outcomes in all activity parameters in patients whose tumors expressed GPNMB. Notably, in patients who received the Phase 2 dose and whose tumors expressed GPNMB, 2 of 7 (29%) had confirmed Partial Responses, 5 of 7 (71%) had decreases in tumor size, and all 7 achieved at least stable disease with duration from 17.3 to 26.9 weeks. The median PFS in all patients was 9.1 weeks, but in patients whose tumors expressed GPNMB, median PFS was 18.3 weeks, compared to median PFS of 5.9 weeks for patients whose tumors did not express GPNMB. In patients with triple negative disease, 5 of 7 (71%) analyzed samples expressed GPNMB, 7 of 9 (78%) evaluable patients had tumor shrinkage, and the median PFS for these patients was 17.9 weeks.

About CDX-011

CDX-011 (formerly CR011-vcMMAE) is an antibody-drug conjugate (ADC) being developed by Celldex Therapeutics, Inc. that consists of a fully-human monoclonal antibody, CR011, linked to a potent cell-killing drug, monomethyl-auristatin E (MMAE). The ADC technology, comprised of MMAE and a stable linker system for attaching it to CR011, was licensed from Seattle Genetics, Inc. The ADC is designed to be stable in the bloodstream. Following intravenous administration, CDX-011 targets and binds to GPNMB, a specific protein that is predominantly expressed in cancerous tumors, including melanoma, breast cancer and gliomas. Upon internalization into the targeted cell, CDX-011 is designed to release MMAE from CR011 to produce a cell-killing effect. CDX-011 is currently completing two Phase 2 trials assessing the safety and efficacy in the treatment of melanoma and for the treatment of metastatic breast cancer, and in a Phase 1 trial to evaluate the safety and activity of alternate dosing schedules.

About Breast Cancer

Breast cancer is the most common cancer in women and a leading cause of death in the United States. According to the American Cancer Society, more than 180,000 women will be diagnosed with invasive breast cancer in 2009 with more than 40,000 deaths attributed to this disease.

Despite recent advances in therapy, the median survival of patients with metastatic breast cancer is 2 to 3 years, while patients with “triple-negative” or “basal-like” breast cancer have limited treatment options and poorer outcomes. Therefore, a significant unmet need remains for novel therapeutic approaches for patients with locally advanced and metastatic breast cancer who have failed other therapies.

Source
Celldex Therapeutics, Inc.

Boehringer Ingelheim announced today the initiation of a new phase III clinical trial to evaluate one of its two late-stage oncology pipeline compounds for the treatment of patients with advanced ovarian cancer. The clinical study, called LUME-Ovar-1 trial, investigates the compound BIBF 1120, a novel oral anti-angiogenic agent , for its efficacy and safety as first-line treatment in combination with standard chemotherapy compared to placebo in combination with standard chemotherapy in patients with advanced ovarian cancer.

The LUME-Ovar 1 trial / AGO-OVAR 12

AGO-OVAR 12 or LUME-Ovar 1 is an intergroup study conducted by an international consortium of study groups led by the AGO Study Group (Arbeitsgemeinschaft Gynaekologische Onkologie Study Group, Germany), and Boehringer Ingelheim. BIBF 1120 (planned brand name Vargatef™) is a novel oral compound that works by simultaneously inhibiting three receptors involved in the formation of blood vessels, a process also called angiogenesis, which is needed for tumours to grow and spread.

“Anti-angiogenic compounds are one of the most promising new approaches in treating cancer, and ovarian cancer appears to be particularly sensitive to these novel agents, as suggested by early clinical trials.” said Andreas du Bois, MD, PhD, of AGO Study group and International Coordinating Investigator of this trial. “BIBF 1120 inhibits several aspects of the pathway that controls the growth of new blood vessels, and it has already been shown to be both effective and well tolerated in previous trials, including a recently completed phase II trial of BIBF 1120 in ovarian cancer.”

The AGO Study group has already performed a phase I/II study and developed the combination now evaluated. “Based on our good experience with this combination,” said Dr. Philipp Harter MD , of AGO study group and principal investigator of the starting trial, “we look forward to continue investigating this novel compound in ovarian cancer patients in the first line setting.”

The decision to progress BIBF 1120 into a phase III trial in ovarian cancer was based on previous study results which indicate that the agent may be both efficacious and well tolerated as maintenance therapy in patients with relapsed ovarian cancer who had responded to prior chemotherapy. Data presented this year at the annual meeting of the American Society of Clinical Oncology (ASCO) showed that in the trial women with ovarian cancer treated with BIBF 1120 were less likely to experience progression of their disease compared to those treated with placebo: at 36 weeks, 14.3% of women taking BIBF 1120 were progression-free compared to 5% of women taking placebo. This phase II trial represents the first published randomised, placebo controlled data of an anti-angiogenic agent in ovarian cancer.

As angiogenesis plays a pivotal role in the growth of all solid tumours, BIBF 1120 is currently being investigated in a number of cancers including advanced non-small cell colorectal cancer (CRC), renal cell cancer (RCC) und hepatic cell cancer (HCC).

BIBF 1120 is one of Boehringer Ingelheim’s most advanced compounds along with BIBW 2992 (planned trade name Tovok™), both of which are currently in phase III development for the treatment of patients with advanced non-small cell lung cancer, a patient population with limited treatment choices.

LUX-Lung 1 Trial: Development progress with BIBW 2992
In the clinical development of the compound BIBW 2992, a milestone has been reached recently. The company completed patient recruitment for a Phase III Lung cancer trial, the LUX-Lung 1 trial. LUX-Lung 1 investigates BIBW 2992 (planned brand name Tovok™) plus best supportive care (BSC) versus placebo plus BSC in non-small cell lung cancer patients who have experienced a failure of treatment with erlotinib or gefitinib (EGFR TKI failures).

Boehringer Ingelheim believes in evidence-based, scientific progress; its extensive oncology clinical trial programme involves more than 800 study centers in 47 countries. Boehringer Ingelheim has a dedicated cancer research center in Vienna where scientists are focused on the discovery and development of new treatments to combat or alleviate cancer.

About Ovarian Cancer

Ovarian cancer often goes undetected until an advanced stage, and is sometimes referred to as the “silent killer”. According to the 2008 World Health Organization World Cancer Report, as of 2002, ovarian cancer was ranked as the 6th most common cancer in women, and is considered the most lethal of gynaecological malignancies. Approximately 204,000 new cases were diagnosed worldwide and 125,000 women died from the disease in 2002. The ACS estimates that about 21,550 new cases of ovarian cancer were diagnosed in the United States (U.S.) during 2008. Only forty-five percent of women with ovarian cancer are still alive five years after diagnosis in the U.S.

About AGO

The AGO Study group is a German cooperative study group active in clinical research in gynecological oncology for almost 20 years with great experience in developing and optimizing therapeutic standards in first and second line treatment of ovarian cancer. Together with other European and overseas cooperative study groups, AGO has developed the current standard regimens.

Source
Boehringer Ingelheim