Medivation and Pfizer : Results from Phase 3 Concert Trial of Dimebon in Alzheimer's Disease

January 17, 2012 - Dimebon Did Not Meet Primary Efficacy Endpoints - Medivation, Inc. (NASDAQ: MDVN) and Pfizer Inc. (NYSE: PFE) announced results from the CONCERT trial, which is a Phase 3 trial that evaluated dimebon (latrepirdine) when added to ongoing treatment with donepezil HCL tablets in patients with mild-to-moderate Alzheimer's disease. Dimebon did not achieve statistically significant results for either of the two co-primary endpoints, the Alzheimer's Disease Assessment Scale — cognitive subscale (ADAS-cog), which measures cognitive ability, or the Alzheimer's Disease Cooperative Study — Activities of Daily Living (ADCS-ADL), which measures self care and daily function.

"We are disappointed in the CONCERT results and the implications for Alzheimer's disease patients and their caregivers," said David Hung, M.D., president and CEO of Medivation. "I would like to extend my sincere thanks to the patients, their physicians and study teams involved in this trial."

Dimebon was generally well tolerated in the study. A full analysis of the results from CONCERT will be conducted and submitted for presentation at an upcoming scientific congress.

Medivation and Pfizer will discontinue development of dimebon for all indications and will terminate the ongoing open label extension study in Alzheimer's disease. The companies also announce that they will terminate their collaboration to co-develop and market dimebon pursuant to the terms of their Collaboration Agreement... Medivation's Press Release - Pfizer's Press Release -

Aphios® Awarded Phase II SBIR Grant From National Institute On Aging For Alzheimer's Disease Research

January 24, 2012— Aphios® Corporation has received a Phase II SBIR grant from the National Institutes of Health (NIH) amounting to $2.4 million over two years. The National Institute on Aging (NIA), part of the NIH, awarded the grant to the Aphios Corporation to develop an “Alzheimer’s Disease Therapeutic.”

Alzheimer’s Disease (AD) is the third leading cause of death in America and among the highest in the industrial world. AD is a devastating age-related neurological disorder that affects more than 4.5 million Americans and more than 10 million people worldwide. Experts estimate that 22 million people around the world and more than 8 million Americans will be afflicted with this disease by 2025.

“Aphios is developing APH-0703, a potent protein kinase C (PKC) modulator that activates the alpha-secretase pathway which can dramatically enhance the generation of soluble amyloid precursor protein (sAPP), diminishing plaques and cognitive deficits associated with Alzheimer’s Disease,” explains Dr. Trevor P. Castor, President and CEO, Aphios Corporation. “We are developing novel formulations of APH-0703 based on Aphios’ proprietary hydrophobic-based formulation and patented SFS-PNS polymer nanospheres technologies. After formulation selection, Aphios will manufacture and characterize the API and final drug product following cGMP guidelines, and conduct preclinical studies in preparation of filing an IND with the FDA to conduct human clinical trials.”... Aphios' Press Release -

Targacept : Decision by AstraZeneca to Advance AZD1446 in Alzheimer’s Disease

January 05, 2012 - Targacept, Inc. (NASDAQ: TRGT), a clinical-stage biopharmaceutical company developing novel NNR Therapeutics™, announced that AstraZeneca has informed the company that it plans to progress the development of Targacept’s product candidate AZD1446 as a treatment for Alzheimer’s disease.

AZD1446 is a selective modulator of the alpha4beta2 neuronal nicotinic receptor that arose out of a research collaboration conducted by Targacept and AstraZeneca. Under the terms of a 2005 collaborative research and license agreement, AstraZeneca is responsible for conducting and funding the development and potential commercialization of AZD1446. The next clinical trial of AZD1446 is expected to be a Phase 2 study as an adjunct treatment to donepezil in patients with mild to moderate Alzheimer’s disease... Targacept's Press Release -

Abbott Biotech Ventures Invests in NeuroGenetic Pharmaceuticals

January 4, 2012 - Advancing Research on Therapy for Alzheimer’s Disease -

NeuroGenetic Pharmaceuticals, Inc. (NGP), a privately held biopharmaceutical company focused on Alzheimer’s disease (AD) therapeutics, has received an investment from Abbott Biotech Ventures. The amount of the investment was not disclosed. Dr. William T. Comer, president and CEO of NGP said, “The funding will expedite the development of our lead candidate, NGP 555, for the prevention of AD, including achieving our immediate goal of initiating clinical trials.”

Earlier, NGP announced that it has been awarded a Small Business Innovation Research (SBIR) fast-track grant from the National Institutes of Health (NIH) for preclinical work on NGP 555. The first phase award is in the amount of $288,000 with future awards to approach $1 million for each subsequent phase, which can be up to three rounds of additional funding for milestone-based achievements.

About NeuroGenetic Pharmaceuticals, Inc. 

NeuroGenetic Pharmaceuticals, Inc. (NGP), a biopharmaceutical discovery and development company founded in 2009, is developing innovative drug therapies for Alzheimer’s disease. Based in San Diego, Calif., the company’s next objective is to obtain an Investigational New Drug approval for its clinical candidate, NGP 555. Future clinical trials will utilize specific amyloid biomarkers and/or brain scanning as an early diagnostic and to monitor drug efficacy in clinical trials. Combining early disease identification with a treatment capable of preventing AD-related pathology, such as NGP 555, would represent an important advance in our ability to prevent AD or hinder its progression to dementia. Clearly, the earlier AD is detected and treated, the better the likelihood of a good outcome.

NGP 555, a proprietary “first in class” molecule for the treatment/prevention of Alzheimer’s disease, is a gamma-secretase modulator targeting the γ-secretase complex, a key enzyme in the amyloid pathway. The compound has excellent brain penetration and is devoid of side-effects seen with other potential amyloid therapies such as gamma-secretase inhibitors and monoclonal antibodies. Based on the pre-clinical studies in rodents, this compound is expected to prevent the formation of Aβ42 and the deposition of amyloid plaques in the human brain, thereby precluding neuronal cell death and the dementia associated with AD... NeuroGenetic Pharmaceuticals' Press Release -

Neoprobe Licenses AstraZeneca Imaging Agent for Amyloid Detection to Aid Diagnosis of Alzheimer’s Disease

December 12, 2011 - Neoprobe Corporation (NYSE Amex: NEOP) announced that it has in-licensed the worldwide exclusive rights from AstraZeneca to the late-stage radiopharmaceutical imaging candidate, AZD4694, for aiding the diagnosis of Alzheimer’s disease (AD).

AZD4694 is a Fluorine-18 labeled precision radiopharmaceutical candidate for use in the imaging and evaluation of patients with signs or symptoms of cognitive impairment such as AD. It binds to Beta-amyloid deposits in the brain that can then be imaged in positron emission tomography (PET) scans. Amyloid plaque pathology is a required feature of AD diagnosis and the presence of amyloid pathology is a supportive feature for diagnosis of probable AD. Patients who are negative for amyloid pathology do not have AD.

Clinical studies in more than 70 patients suggest that AZD4694 has the ability to image patients quickly and safely with high sensitivity. Importantly, AZD4694 exhibits low background and white matter uptake, thereby providing clear images of Beta-amyloid deposits. Neoprobe intends to initiate a Phase III clinical program in early 2013, while simultaneously building the requisite safety and training database. Patents and patent applications filed around the world related to AZD4694 are effective until 2028... [PDF] Neoprobe's Press Release -

Dainippon Sumitomo Pharma : launch of Alzheimer's Disease Treatment Donepezil hydrochloride tablets/OD tablets 3mg/5mg “DSP”

November 28, 2011 - Dainippon Sumitomo Pharma Co., Ltd. (DSP) (Headquarters: Osaka, Japan; President: Masayo Tada) announces the launch of Alzheimer's disease treatment Donepezil hydrochloride tablets/OD tablets 3mg/5mg “DSP” in Japan, as of December 2, 2011.

In domestic pharmaceuticals, DSP designates the Central Nervous System field as one of its focus marketing areas, and offers various treatments such as atypical antipsychotics "LONASEN® " and "LULLAN® ", Parkinson's disease drug "TRERIEF® ", norepinephrine-activating neural function ameliorant "DOPS® ", anti-epileptic drug "EXCEGRAN® ", and serotonin-agonist antianxiety drug "SEDIEL® ". Approximately 230 CNS MRs in the CNS Sales & Marketing Division newly established in April 2011 are developing activities to provide information on these products.

DSP decided to handle a CNS generic drug as part of a plan to strengthen its product lineup in the CNS area. On June 24, 2011, DSP created “Established Products & Chain Pharmacies Groups” dedicated to generic drugs in the CNS area under CNS Sales and Marketing, each under the CNS Higashi-Nippon Region, CNS Capital Region, CNS Kinki-Tokai Region and CNS Nishi-Nippon Region with the aim to promote sales of generic drugs in the CNS area... [PDF] Dainippon Sumitomo Pharma's Press Release -

Probiodrug : Top-Line Results of the First Clinical Study of PQ912 for Treatment of Alzheimer’s Disease

November 14, 2011 - Phase 1 Study of the First Glutaminyl Cyclase (QC) Inhibitor, Which Blocks Formation of Highly Neurotoxic pyroGlu Aβ Variants, Shows that PQ912 is Well Tolerated With Therapeutically-Relevant Levels in Blood and Cerebrospinal Fluid –

Probiodrug AG (Probiodrug), a biotech company developing products for the treatment of neurodegenerative and inflammatory diseases, with a particular focus on Alzheimer's disease (AD), today announced top-line results of its Phase 1 single (SAD) and multiple ascending dose (MAD) study of PQ912 in healthy volunteers. PQ912 is a glutaminyl cyclase (QC) inhibitor for the treatment of AD and it is the first QC inhibitor to enter clinical development.

The Phase 1 trial, conducted in Switzerland, demonstrated that PQ912 is safe and well tolerated after oral dosing. Dose-proportional pharmacokinetics and a strong pharmacokinetic and pharmacodynamic relationship based on QC inhibition were observed in plasma and cerebrospinal fluid. The combined SAD/MAD study involved 100 volunteers in a blinded, placebo controlled randomized trial.

PQ912 is a small molecule that targets QC, an enzyme that catalyzes the formation pyroGlu amyloid-beta (Aβ) a highly toxic Aβ variant that is involved in the development and progression of AD. AD is a neurodegenerative disease characterized by deposits of extracellular Aβ plaques in the brain, intraneuronal tangles and cerebral neuronal loss. QC inhibitors address a major pathology associated with AD by inhibiting the formation of Aβ variants which lead to the assembly of highly neurotoxic Aβ-oligomers... Probiodrug's Press Release -

Astellas and RIKEN : Collaborative Research Launched to Explore Novel Drug Targets for Alzheimer’s Disease

November 8, 2011 - RIKEN Brain Science Institute and Astellas Pharma Form Strategic Alliance for New Drug Discovery -

RIKEN, Japan (President: Ryoji Noyori) and Astellas Pharma Inc. (President: Yoshihiko Hatanaka, hereafter called “Astellas”) have signed a five year joint research agreement for the purpose of “deciphering the pathogenic mechanism for and identifying novel drug targets for Alzheimer’s disease”. With the goal of conquering the social burdens of Alzheimer’s disease, synergies between the fundamental research function of RIKEN Brain Science Institute (RIKEN BSI, Director: Susumu Tonegawa) and drug discovery capabilities of Astellas will be leveraged to develop revolutionary new drugs.

1. Background

The number of patients within Japan suffering from Alzheimer’s disease and other forms of dementia is expected to rise along with the aging of the Japanese society and reach 3.25 million by the year 2020 (Source: “Everyone’s Mental Health” of the Ministry of Health, Labor and Welfare). About half of patients with dementia suffer from Alzheimer’s disease, and the burden upon their families and society is heavy. Consequently, the conquering of Alzheimer’s disease at an early stage is an important issue for Japan as a medically advanced country. However at the current time there are no fundamental treatments or preventative measures that have been established, and there are only symptomatic treatments. In order to establish fundamental treatments, it is critical to fully understand the pathogenic mechanisms of the disease and to translate the findings on important mechanisms into discovery of novel drugs for the disease.

Laboratory for Proteolytic Neuroscience at the RIKEN BSI conducts research designed to acquire fundamental knowledge for mechanisms of proteolysis responsible for pathological cascade in neurodegenerative diseases that can be used in the diagnosis, prevention, and treatment of patients with neurodegenerative disorders. In particular, the top priority given to Alzheimer’s disease has led to the acquisition of new information on the important causative agent (amyloid beta peptide) in July 2011... [PDF] Astellas Pharma's Press Release - RIKEN's Press Release -