Drug Companies Resist White House Call to Reduce Rights to Exclusive Drug Data
Posted by Pharma Service Provider | 2:25 PMMedSource Hires New Vice President of Clinical Operations
Posted by Pharma Service Provider | 2:23 PMAbbott Expands XIENCE V(R) USA Trial to Enroll Patients into Landmark Dual Anti-Platelet Therapy (DAPT) Trial
Posted by Pharma Service Provider | 2:22 PMLeading European Contract Research Organization Selects OmniComm to Provide eClinical Solutions for Important Phase III Study
Posted by Pharma Service Provider | 2:17 PMImmunoGen Announces Encouraging New Clinical Data with the Company’s IMGN901 Compound in the Treatment of Small-Cell Lung Cancer
Posted by Pharma Service Provider | 1:12 PMIMGN901 was created by ImmunoGen to kill tumors that express the CD56 protein targeted by the compound. It consists of a potent cancer-cell killing agent, DM1, attached to a CD56-targeting antibody, huN901, using an engineered linker. IMGN901 currently is in early clinical testing and is a potential treatment for SCLC, Merkel cell carcinomas, ovarian cancers, multiple myeloma, and other CD56+ cancers.
"We are encouraged by the initial efficacy and safety profile of IMGN901 in SCLC and Merkel cell carcinoma, especially as these cancers have proven highly challenging to treat,” stated Professor Penella J. Woll, MD, PhD, Weston Park Hospital, UK. "The data thus far indicate that IMGN901 offers potential for use in a number of indications, including several types of CD56-expressing solid tumors.”
The findings being presented at the 13th World Conference on Lung Cancer by Professor Woll come from the two IMGN901 trials that included SCLC patients. Study 001 (enrollment has closed) was open to patients with any type of CD56+ solid tumor, including SCLC, during its dose-escalation Phase I leg. In its Phase II leg, enrollment was limited to patients with SCLC or other CD56+ small-cell carcinoma. In this trial, IMGN901 was administered weekly for four weeks every six weeks. Study 002 (enrollment is ongoing) is a dose-escalation trial evaluating IMGN901 in patients with CD56+ solid tumors, including SCLC, when administered daily for three consecutive days every 21 days. In both trials, the SCLC must have recurred following previous treatment with standard therapies for a patient to qualify for enrollment.
Pfizer And Medivation Initiate Phase 3 Trial Of Dimebon In Patients With Huntington Disease
Posted by Pharma Service Provider | 1:11 PM"Based on the promising results of our Phase 2 trial of dimebon in Huntington disease, we are pleased to advance dimebon into late-stage clinical development," said Lynn Seely, M.D., chief medical officer for Medivation. "Huntington disease is a fatal genetic disease for which no medications are currently approved by the FDA to treat the cognitive impairment associated with the condition."
Orphan status is granted by the FDA to promote the development of products that demonstrate promise for the treatment of rare diseases affecting fewer than 200,000 Americans annually, such as Huntington disease, which affects 30,000 individuals in the United States, with another 150,000 at risk. Orphan drug designation entitles Pfizer and Medivation to a seven-year period of marketing exclusivity in the United States for dimebon if it is approved by the FDA for the treatment of Huntington disease. It also enables the companies to apply for research funding, tax credits for certain research expenses, and a waiver from the FDA's application user fee.
Latrepirdine is the proposed generic (nonproprietary) name for dimebon.
Design of the HORIZON Study
The double-blind, placebo-controlled Phase 3 trial will enroll approximately 350 patients with Huntington disease at approximately 50 sites in North America, Europe and Australia. Patients will be randomized to receive either dimebon (latrepirdine) 20 mg three times daily or placebo for six months.
The primary endpoints of the trial are the Mini Mental State Examination (MMSE), which measures cognition, and the Clinician's Interview-Based Impression of Change, plus caregiver input (CIBIC-plus), which measures global function. The trial will include only patients who have cognitive impairment, as subjectively assessed by an investigator and objectively by MMSE score.
Secondary endpoints include the Neuropsychiatric Inventory (NPI), which measures behavior; the Alzheimer's Disease Cooperative Study Activities of Daily Living (ADCS-ADL), which measures self-care and daily function; and the Unified Huntington Disease Rating Scale (UHDRS'99) Total Motor Score, which measures motor impairment; and safety.
The trial is being conducted in collaboration with the Huntington Study Group (HSG) and the European Huntington's Disease Network (EHDN). The HSG is a non-profit group of experienced clinical trial investigators from medical centers in the United States and abroad dedicated to clinical research of Huntington disease. The EHDN is a non-profit network of professionals providing an infrastructure for large scale Huntington disease clinical trials throughout Europe.
TB is the leading cause of death among people living with HIV in Africa. Seventy-three percent of South Africans infected with TB are also HIV-positive.
A new vaccine (AERAS-402/Crucell Ad35) could significantly advance the fight against TB in both HIV-positive and HIV-negative patients.
Health systems management organisation The Aurum Institute will conduct a trial in Johannesburg to determine the safety of the vaccine in HIV-positive adults. The trial will also provide the first indications of whether the vaccine is effective.
"If we find a vaccine, it will be a major contribution to helping reduce the risk to TB-infected, HIV-infected individuals, and for controlling TB in high HIV prevalence settings such as South Africa," said The Aurum Institute's CEO, Dr Gavin Churchyard.
If the vaccine was effective in HIV-positive people, it was likely that it would be even more effective in HIV-negative people, said Churchyard. They hope to start vaccinating next month.
The advantage of a vaccine over treatment is that it's easier to roll out on a large scale.
There's also no need to worry about adherence to chronic medication, or adverse drug interactions.
"We believe that an effective TB vaccine is the best hope for the achievement of the millennium goals for TB reduction and the eventual elimination of this scourge from our planet," said Churchyard.
The current vaccine, BCG, was developed more than 85 years ago.
It reduces the risk of severe forms of TB in early childhood, but is not very effective in preventing pulmonary TB in adolescents and adults - the populations with the highest rates of TB.
Preliminary clinical trial data has shown that the vaccine being tested by The Aurum Institute produces the highest levels of CD8 immune cells, a leading strategy in the pursuit of an effective TB vaccine.
AERAS-402/Crucell Ad35 was developed by Crucell, a biopharmaceutical company in the Netherlands, and is being funded by a non-profit organisation called the Aeras Global TB Vaccine Foundation.
It has previously been tested for safety on healthy adults in the US and South Africa, and on adults exposed to TB in South Africa and Kenya, but this is the first trial that will test safety in HIV-infected adults.
One of the reasons why the results of the trial will be important is because vaccinations are generally administered en masse.
"You don't want to have to worry about someone's HIV status," said Churchyard. Additionally, in low resource settings, many people do not know their HIV status. "We want to be sure that the vaccine doesn't cause HIV to progress more rapidly."
Volunteers in the trial will have already been infected with HIV and will be at high risk of acquiring TB.
"We hope that we will show it's safe, and that we will see the first glimpse of whether it may be effective," said Churchyard.
A Phase 1 Trial of pharmacologic interactions between transdermal selegiline and a 4-hour cocaine infusion
Posted by Pharma Service Provider | 1:07 PMMethods: Twelve nondependent cocaine-experienced subjects received deuterium-labeled cocained5 intravenously (IV) 0.5mg/kg over 10 minutes followed by 2 mg/kg over 4 hours before and after one week of transdermal selegiline 6 mg/24 hours.
Plasma and urine were collected for analysis of selegiline, cocaine, catecholamine and metabolite concentrations. Pharmacodynamic measures were obtained.
Results: Selegiline did not change cocaine pharmacokinetic parameters.
Selegiline administration increased phenylethylamine (PEA) urinary excretion and decreased urinary MHPG-sulfate concentration after cocaine when compared to cocaine alone. No serious adverse effects occurred with the combination of selegiline and cocaine, and cocaine-induced physiological effects were unchanged after selegiline.
Only 1 peak subjective cocaine effects rating changed, and only a few subjective ratings decreased across time after selegiline.
Conclusion: No pharmacological interaction occurred between selegiline and a substantial dose of intravenous cocaine, suggesting the combination will be safe in pharmacotherapy trials. Selegiline produced few changes in subjective response to the cocaine challenge perhaps because of some psychoactive neurotransmitters changing in opposite directions.
AVEO's Tivozanib Demonstrates Anti-Tumor Activity in Engineered Lung Tumors Exhibiting Treatment Resistant Mutations
Posted by Pharma Service Provider | 1:06 PMAVEO Pharmaceuticals, Inc., a biopharmaceutical company leveraging breakthrough discoveries in cancer biology to discover, develop and commercialize targeted oncology therapies, today announced data which demonstrates that tivozanib (AV-951) – the company’s oral, triple VEGF receptor inhibitor – exhibits potent anti-angiogenic and anti-tumor activity in AVEO’s proprietary in vivo lung cancer models. Specifically, treatment with tivozanib resulted in complete tumor growth inhibition or tumor regression (shrinkage) in lung tumors driven by EGFR or KRAS mutations, which are especially difficult to treat. These data are being presented today at the 13th World Conference on Lung Cancer (WCLC) in San Francisco, abstract number PD10.1.5.
"Non-small cell lung cancer is a highly heterogeneous disease and one of the most difficult cancers to treat. Common genetic alterations responsible for the disease, such as those driven by KRAS or certain EGFR mutations, typically render anticancer treatments powerless,” stated Murray O. Robinson, Ph.D., senior vice president, oncology at AVEO. “Utilizing our proprietary cancer biology platform, we have created and treated genetically-engineered lung cancer mouse models driven by treatment resistant EGFR or KRAS, resulting in a much more realistic, human-like environment in which we can evaluate tivozanib efficacy. We are very pleased with and encouraged by the robust activity demonstrated by tivozanib in these difficult-to-treat tumor alleles."
Tivozanib is a novel, oral triple VEGF receptor inhibitor which recently completed a Phase 2 clinical study and is expected to enter a Phase 3 clinical trial later this year in patients with advanced kidney cancer. Tivozanib is being studied in several ongoing Phase 1b/2a clinical trials. Clinical results to date suggest tivozanib is a potent, well tolerated anticancer agent with broad potential as a treatment for solid tumors.
"Our innovative, proprietary cancer biology platform helps us create more relevant models of cancer in which we can evaluate the efficacy of our drug candidates preclinically, thereby increasing the probability of success in the clinic,” stated Tuan Ha-Ngoc, president and chief executive officer of AVEO Pharmaceuticals. “The robust anti-angiogenic and anti-tumor activity of tivozanib in AVEO-engineered models of TKI-resistant lung tumors, paired with the early results observed with tivozanib in patients with lung cancer from the previously reported Phase 1 trial, highlights the potential role of tivozanib in the treatment of this difficult-to-treat cancer.”
In this study, AVEO developed genetically engineered lung adenocarcinoma models driven by either treatment resistant EGFRT790M or KRAS mutant proteins by creating chimeric mice incorporating genetically modified ES cells. Tumors driven by EGFR or KRAS were propagated in vivo, to examine tivozanib activity in both subcutaneous and orthotopic settings. Treatment of subcutaneous lung KRAS tumors with tivozanib 2mg/kg and 5mg/kg daily PO for two weeks resulted in complete tumor growth inhibition and 44 percent tumor regressions respectively. Lung EGFR tumors were tested in an orthotopic setting by intravenous seeding. Tivozanib treatment was initiated after tumor establishment was detected by in vivo imaging; 5 mg/kg daily dosing for more than 10 days resulted in prolonged survival. Histologic analysis showed the same dramatic anti-angiogenic changes as in the subcutaneous KRAS tumors, indicating that tivozanib is effective in killing orthotopic lung tumors that are resistant to EGFR targeted kinase inhibitors (TKIs).
In a previously reported Phase 1 dose-escalation study evaluating tivozanib in 41 patients with advanced solid tumors clinical benefit (i.e., partial response or stable disease lasting for three months or longer) was achieved across multiple tumor types, including in those patients with lung cancer. Overall, tumor shrinkage was observed in 33 percent of all patients during treatment with tivozanib. A Phase 1b/2a trial evaluating tivozanib as monotherapy in patients with non-small cell lung cancer was initiated in March 2009 and is ongoing.
In May 2009, researchers presented complete Phase 2 data evaluating tivozanib in metastatic renal cell carcinoma (mRCC) at the 45th Annual Meeting of the American Society for Clinical Oncology (ASCO), which demonstrated prolonged progression-free survival of 11.8 months and an excellent safety profile in patients treated with tivozanib. The company expects to initiate a Phase 3 trial of tivozanib in advanced kidney cancer later this year.
Neurocrine Biosciences Reports Second Quarter 2009 Results
Posted by Pharma Service Provider | 1:06 PMNeurocrine Biosciences, Inc. today announced its financial results for the quarter ended June 30, 2009. For the second quarter of 2009, the Company reported a net loss of $15.3 million, or $0.39 per share, compared with a net loss of $21.0 million, or $0.55 per share, for the same period in 2008. For the six months ended June 30, 2009, the Company reported a net loss of $34.9 million, or $0.90 per share, as compared to $42.0 million, or $1.10 per share, for the same period last year.
Revenues for the second quarter of 2009 and 2008 were $0.7 million. Revenues for the six months ended June 30, 2009 were $1.5 million, compared with $2.5 million for the same period in 2008. The decrease in revenues is primarily due to milestones recognized in 2008 under our collaboration agreement with GlaxoSmithKline (GSK) related to the clinical advancements of our CRF program. During both six month periods ended June 30, 2009 and 2008, we recognized $1.5 million in revenue under our collaboration agreement for indiplon with Dainippon Sumitomo Pharma Co. Ltd. (DSP) from amortization of up-front licensing fees.
Research and development expenses decreased to $10.8 million during the second quarter of 2009 compared with $16.2 million for the same period in 2008. For the six months ended June 30, 2009, research and development expenses were $21.7 million, compared to $30.4 million for the same period last year. The decrease in research and development expenses is primarily due to expense management efforts and decreasing external clinical development expenses related to the elagolix program.
General and administrative expenses were $4.8 million for the second quarter of 2009 and $4.7 million during the same period last year. For the six months ended June 30, 2009, general and administrative expenses were $9.0 million, compared to $13.0 million for the first half of 2008. We incurred a $2.2 million restructuring charge in the first half of 2008 compared to a $0.7 million charge in the first half of 2009. Additionally, other non-personnel cost savings have resulted in six month over six month savings of approximately $1.2 million.
The Company's balance sheet on June 30, 2009 reflected total assets of $86.9 million, including cash and investments of $74.0 million compared with balances at December 31, 2008 of $118.2 million and $101.5 million, respectively.
"We are in a very good financial position with our burn well controlled and within the guidance we gave at the beginning of the year," said Kevin C. Gorman, President and Chief Executive Officer. "At the same time, we are moving forward with our clinical programs, the most advanced of which is elagolix, and have just put another compound, VMAT2 inhibitor into Phase I trials. We also continue to make progress on several preclinical projects."
Pipeline Highlights
Elagolix Update
The Week 24 results of the recently completed Lilac Petal Study (0702) were released earlier today. This study assessed elagolix in subjects with confirmed endometriosis over a six-month period. The first three months of the study included three arms; elagolix 150 mg, elagolix 250 mg, and placebo. After the initial three months, the placebo arm was re-randomized into one of the two elagolix arms. These 24 Week results of the Lilac Petal Study again confirmed that elagolix has clinically meaningful efficacy coupled with a favorable safety profile.
The Tulip Petal Study (0703) has completed subject randomization in Central Eastern Europe (n=174). This study is designed as a randomized, double-blind, placebo and active controlled trial with four treatment arms; elagolix 150 mg, elagolix 250 mg, leuprolide depot, and placebo. We expect top-line data (first three months of placebo and active controlled treatment) to be available in the fourth quarter of this year.
Petal Study (0603) bone data were presented at the Endocrine Society meeting in Washington, D.C. in June 2009 and the clinical efficacy and safety abstract from this study has been accepted for presentation at the American Society for Reproductive Medicine in Atlanta, November 2009.
Urocortin 2 Update
The Christchurch Cardioendocrine Research Group at University of Otago, Christchurch School of Medicine and Health Sciences, New Zealand, in collaboration with Neurocrine, has obtained regulatory approval to begin a pilot study in patients with Acute Decompensated Heart Failure. These patients are the target population for the Urocortin 2 mechanism of action and the investigational intervention will be compared to standard-of-care treatment; enrollment of 50 subjects is planned.
VMAT2 Update
The highly selective blockade of the Vesicular Monoamine Transporter 2 (VMAT2) with NBI-98854 should be of clinical benefit in patients with a variety of CNS diseases, especially those with involuntary hyperkinetic movements such as Tardive Dyskinesia. A Clinical Trial Application has been approved by Health Canada and we will initiate a single ascending dose Phase 1 study in August 2009.
Conference Call and Webcast Thursday, July 30, 2009 at 8:30 a.m. EDT
Neurocrine will hold a live conference call and webcast tomorrow morning, Thursday, July 30, 2009 at 8:30 a.m. Eastern Daylight Time (5:30 a.m. Pacific Daylight Time). Participants can access the live conference call by dialing 1-800-895-0198 (US) or 785-424-1053 (International) using the conference passcode 7NEURO. The call can also be accessed via the webcast through the Company's website at http://www.neurocrine.com/
If you are unable to attend the webcast and would like further information on this announcement, please contact the Investor Relations Department at Neurocrine Biosciences at (858) 617-7600. A replay of the conference call will be available approximately one hour after the conclusion of the call by dialing 1-800-723-0479 (US) or 402-220-2650 (International) using the passcode 7NEURO. The call will be archived for two weeks.
Neurocrine Biosciences, Inc. is a biopharmaceutical company focused on neurological and endocrine diseases and disorders. Our product candidates address some of the largest pharmaceutical markets in the world including endometriosis, anxiety, depression, pain, diabetes, benign prostatic hyperplasia (BPH), irritable bowel syndrome (IBS) and other neurological and endocrine related diseases and disorders. Neurocrine Biosciences, Inc. news releases are available through the Company's website via the internet at http://www.neurocrine.com/
In addition to historical facts, this press release may contain forward-looking statements that involve a number of risks and uncertainties. Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements are risks and uncertainties associated with Neurocrine's business and finances in general, as well as risks and uncertainties associated with the Company's GnRH program, R & D pipeline and Company overall. Specifically, the risks and uncertainties the Company faces with respect to the Company's GnRH program include risk that the elagolix clinical trials will fail to demonstrate that elagolix is safe and effective; risk that elagolix will not proceed to later stage clinical trials; and risks associated with the Company's dependence on corporate collaborators for development, commercial manufacturing and marketing and sales activities. In addition, the Company faces risks and uncertainties with respect to the Company's R & D pipeline including risk that the Company's urocortin 2, and VMAT2 clinical candidates will not proceed to later stage clinical trials, and risk that the Company's research programs will not identify pre-clinical candidates for further development. With respect to its pipeline overall, the Company faces risk that it will be unable to raise additional funding required to complete development of all of its product candidates; risk relating to the Company's dependence on contract manufacturers for clinical drug supply; risks associated with the Company's dependence on corporate collaborators for commercial manufacturing and marketing and sales activities; uncertainties relating to patent protection and intellectual property rights of third parties; risks and uncertainties relating to competitive products and technological changes that may limit demand for the Company's products; and the other risks described in the Company's report on Form 10-K for the year ended December 31, 2008 and Form 10-Q for the quarter ended March 31, 2009. Neurocrine undertakes no obligation to update the statements contained in this press release after the date hereof.
NEUROCRINE BIOSCIENCES, INC.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
(in thousands, except loss per share data)
Three Months Ended Six Months Ended
June 30, June 30,
-------- --------
2009 2008 2009 2008
---- ---- ---- ----
(unaudited) (unaudited)
Revenues:
Sponsored research
and development $3 $4 $20 $16
License fees and milestones 730 730 1,460 2,460
Grant revenue - - - 9
--- --- --- ---
Total revenues 733 734 1,480 2,485
Operating expenses:
Research and development 10,808 16,186 21,656 30,413
General and administrative 4,827 4,665 9,022 12,951
Cease use expense 941 - 5,769 -
--- --- ----- ---
Total operating expenses 16,576 20,851 36,447 43,364
Loss from operations (15,843) (20,117) (34,967) (40,879)
Other income and (expense):
Interest income and other
income 563 1,060 22 2,666
Interest expense - (1,914) - (3,835)
--- ------- --- -------
Total other income (expense)
net 563 (854) 22 (1,169)
Net loss $(15,280) $(20,971) $(34,945) $(42,048)
======== ======== ======== ========
Net loss per common share:
Basic and diluted $(0.39) $(0.55) $(0.90) $(1.10)
====== ====== ====== ======
Shares used in the calculation
of net loss per common share:
Basic and diluted 39,046 38,421 38,858 38,376
====== ====== ====== ======
NEUROCRINE BIOSCIENCES, INC.
CONDENSED CONSOLIDATED BALANCE SHEETS
(in thousands)
June 30, December 31,
2009 2008
---- ----
(unaudited)
Current assets:
Cash and investments $52,767 $80,473
Other current assets 1,017 950
----- ---
Total current assets 53,784 81,423
Property and equipment, net 4,183 6,191
Long-term investments 21,242 21,057
Restricted cash 6,414 6,409
Other non-current assets 1,246 3,102
----- -----
Total assets $86,869 $118,182
======= ========
Current liabilities $33,017 $26,094
Long-term liabilities 46,504 55,314
Stockholders' equity 7,348 36,774
----- ------
Total liabilities and stockholders' equity $86,869 $118,182
======= ========
Source: Neurocrine Biosciences, Inc.
Sangamo BioSciences Reports Second Quarter 2009 Financial Results
Posted by Pharma Service Provider | 1:04 PMSangamo BioSciences, Inc. today reported second quarter 2009 financial results and accomplishments.
For the second quarter ended June 30, 2009, Sangamo reported a consolidated net loss of $4.5 million, or $0.11 per share, compared to a consolidated net loss of $7.4 million, or $0.18 per share, for the same period in 2008. As of June 30, 2009, the company had cash, cash equivalents, marketable securities and interest receivable of $52.6 million.
Revenues for the second quarter of 2009 were $4.7 million, compared to $2.8 million for the same period in 2008. Second quarter 2009 revenues were from the Company's collaboration agreements with Dow AgroSciences (DAS) and Sigma-Aldrich Corporation, enabling technology agreements in protein production and research grants. The revenue recognized for the second quarter of 2009 consisted of $4.2 million in collaboration agreements and $0.5 million in research grants.
Research and development expenses were $6.9 million for the second quarter of 2009, compared to $8.3 million for the same period in 2008. The decrease in research and development expenses for the second quarter of 2009 was primarily due to decreased manufacturing and preclinical expenses partially offset by increased clinical trial expenses. Non-cash employee stock-based compensation included in research and development expenses totaled $0.7 million for the second quarters of both 2009 and 2008.
General and administrative expenses were $3.0 million for the second quarter of 2009, compared to $2.5 million for the same period in 2008. The increase in general and administrative expenses was primarily due to increased professional services and personnel costs including non-cash employee stock-based compensation which totaled $0.8 million in the second quarter of 2009 compared to $0.6 million in the same period in 2008.
Total operating expenses for the second quarter of 2009 were $9.9 million, compared to $10.8 million for the same period in 2008.
Net interest and other income was $0.6 million for the second quarters of both 2009 and 2008.
Six Month Results
For the six months ended June 30, 2009, the consolidated net loss was $11.3 million, or $0.28 per share, compared to a consolidated net loss of $15.4 million, or $0.38 per share, for the six-month period ended June 30, 2008. Revenues were $7.9 million for the first half of 2009, compared to $5.6 million in the same period in 2008. Total operating expenses were $20.1 million for the first half of 2009 and $22.4 million in the first half of 2008. The decrease in operating expenses for 2009 was primarily associated with decreased manufacturing, preclinical and decreased lab supply expenses partially offset by increased clinical trial expenses.
Recent Highlights
-- Presentation of positive Phase 2 ZFP Therapeutic data at ADA 2009.
Sangamo announced the presentation of previously unreleased positive
Phase 2 clinical data from its ZFP Therapeutic(TM) program to develop
SB-509 as a treatment for diabetic neuropathy (DN) at the 69th Annual
Scientific Sessions of the American Diabetes Association (ADA). Data
from Sangamo's SB-509-601 and SB-509-701A Phase 2 clinical trials
demonstrated that SB-509 treatment resulted in statistically
significant and clinically relevant improvements in subjects with
moderate and severe DN as compared to placebo. The data have enabled
the definition of a responder population for SB-509 for future
clinical trials.
-- Achievement of key milestone in cell engineering agreement with
Genentech. A key research milestone was achieved in Sangamo's
Research and License Agreement with Genentech, Inc., a wholly-owned
member of the Roche Group. Genentech scientists demonstrated the
successful knockout of two pre-selected genes in a Chinese hamster
ovary (CHO) cell line using Sangamo's proprietary zinc finger
DNA-binding protein nuclease (ZFN) technology. Genentech is using ZFN
technology to generate cell lines with novel characteristics for
pharmaceutical protein production purposes. The milestone achievement
triggered a payment from Genentech to Sangamo.
-- Publication in Nature and Science magazines of data demonstrating the
successful application of ZFN technology in plant agriculture and in
the generation of novel transgenic animals. Data were published in
April in the scientific journal Nature by licensing partner Dow
AgroSciences and Sangamo demonstrating the successful application of
ZFP technology for the efficient generation of maize plants with
multiple, commercially important traits. The techniques described are
broadly applicable across plant species and traits and establish a new
method for rapid and precise crop development. A second publication in
July in the journal Science described a method for the rapid and
efficient production of transgenic rats in which specific genes are
deleted or knocked out. The technology opens the door for the
production of knockout animals in species other than mouse and
broadens the possibilities for new animal models of human disease.
-- Sangamo awarded Grand Challenges Explorations Grant for Innovative
Global Health Research from the Bill & Melinda Gates Foundation. The
grant will support an innovative global health research project
conducted by Sangamo scientists titled "Zinc Finger Nucleases for In
Vivo Treatment of HIV Infection."
-- Promotion of Philip D. Gregory, D. Phil. to Chief Scientific Officer
in addition to his role as Vice President, Research. Dr. Gregory's
promotion became effective July 1, 2009. He has served as Sangamo's
Vice President, Research since October 2005 and joined the company in
December 2000.
Conference Call
Sangamo will host a conference call today, July 29, 2009 at 5:00 p.m. ET, which will be open to the public. The call will also be webcast live and can be accessed via a link on the Sangamo BioSciences website in the Investor Relations section under "Events and Presentations" http://investor.sangamo.com/events.cfm. The webcast replay will also be available for two weeks after the call. During the conference call, the company will review these results, discuss other business matters, and provide forward-looking guidance with respect to the remainder of 2009.
The conference call dial-in numbers are 877-795-3638 for domestic callers and 719-325-4858 for international callers. The passcode for the call is 9591497. For those unable to listen in at the designated time, a conference call replay will be available for one week following the conference call, from approximately 8:00 p.m. ET on July 29, 2009 to midnight ET on August 5, 2009. The conference call replay numbers for domestic and international callers are 888-203-1112 and 719-457-0820 respectively. The conference ID number for the replay is 9591497.
About Sangamo
Sangamo BioSciences, Inc. is focused on researching and developing zinc finger DNA-binding protein (ZFP) technology for therapeutic gene regulation and modification. By engineering ZFPs that recognize a specific DNA sequence Sangamo has created ZFP transcription factors (ZFP TF(TM)) that can control gene expression and, consequently, cell function. Sangamo is also developing sequence-specific ZFP Nucleases (ZFN(TM)) for gene modification. The most advanced ZFP Therapeutic(TM) development program is currently in Phase 2 clinical trials for evaluation of safety and clinical effect in patients with diabetic neuropathy and ALS. Sangamo also has a Phase 1 clinical trial to evaluate safety and clinical effect of a ZFP Therapeutic for the treatment of HIV/AIDS. Other therapeutic development programs are focused on cancer, neuropathic pain, nerve regeneration, Parkinson's disease and monogenic diseases. Sangamo has established strategic partnerships with companies in non-therapeutic applications of its ZFP technology, including Dow AgroSciences, Sigma-Aldrich Corporation Genentech and Pfizer. For more information about Sangamo visit www.sangamo.com.
This press release contains forward-looking statements regarding Sangamo's current expectations. These forward looking statements include, without limitation, references to the research and development of ZFP TFs and ZFNs, clinical trials, therapeutic and non-therapeutic applications of Sangamo's ZFP technology platform, achievement of research milestones and objectives, strategic partnership with collaborators and anticipated amount of cash and cash equivalents. These statements are not guarantees of future performance and are subject to certain risks, uncertainties and assumptions that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, the early stage of ZFP Therapeutic development, uncertainties related to the timing of initiation and completion of clinical trials, whether clinical trial results will validate and support the safety and efficacy of ZFP Therapeutics, and the ability to establish strategic partnerships. Further, there can be no assurance that the necessary regulatory approvals will be obtained or that Sangamo will be able to develop commercially viable ZFP-based therapeutics. Actual results may differ from those projected in forward-looking statements due to risks and uncertainties that exist in the company's operations and business environments. These risks and uncertainties are described more fully in Sangamo's Annual Report on Form 10-K and Quarterly Reports on Form 10-Q as filed with the Securities and Exchange Commission. Forward-looking statements contained in this announcement are made as of this date and will not be updated.
SELECTED CONSOLIDATED FINANCIAL DATA
(in thousands, except per share data)
(unaudited)
STATEMENT OF OPERATIONS DATA:
Three Months Ended Six Months Ended
June 30, June 30,
----------- ----------
2009 2008 2009 2008
---- ---- ---- ----
Revenues
Collaboration agreements $4,213 $2,378 $7,370 $4,462
Research grants 513 464 513 1,145
----- ----- ----- -----
Total revenues 4,726 2,842 7,883 5,607
Operating expenses:
Research and development 6,877 8,286 14,133 16,929
General and administrative 3,007 2,545 5,933 5,472
Total operating expenses 9,884 10,831 20,066 22,401
------ ------ ------ ------
Loss from operations (5,158) (7,989) (12,183) (16,794)
Interest and other income, net 647 570 840 1,406
Net loss $(4,511) $(7,419) $(11,343) $(15,388)
======= ======= ======== ========
Basic and diluted net loss per
common share $(0.11) $(0.18) $(0.28) $(0.38)
Shares used in computing basic and
diluted net loss per common share 41,123 40,858 41,094 40,673
====== ====== ====== ======
SELECTED BALANCE SHEET DATA:
June 30, 2009 December 31, 2008
------------- -----------------
Cash, cash equivalents, marketable
securities and interest receivable $52,586 $65,025
Total assets 54,938 67,850
Total stockholders' equity 47,176 55,396
Source: Sangamo BioSciences, Inc.
Micromet, Inc. , a biopharmaceutical company developing novel, proprietary antibodies for the treatment of cancer, inflammation and autoimmune diseases, today announced that it intends to offer shares of its common stock in a public offering. Piper Jaffray & Co. is acting as the sole book running manager, with RBC Capital Markets as co-manager in this offering. The offering is being made pursuant to a shelf registration statement filed with the Securities and Exchange Commission on June 19, 2009, which became effective on July 2, 2009. A prospectus supplement relating to the offering will be filed with the Securities and Exchange Commission.
This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of, these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or jurisdiction. When available, copies of the prospectus supplement relating to this offering may be obtained by contacting Piper Jaffray & Co. by mail at 800 Nicollet Mall, Suite 800, Minneapolis, MN 55402, or by telephone at (800) 747-3924.
Money for startup biotechs sure is hard to come by in this economy. And that means more companies are being forced to consider being acquired in order to survive and bring their products to market.
“Fairly new companies, particularly those in early stage development, are stuck in the position of having to be more resourceful,” said Imran Nasrullah, the Massachusetts Biotechnology Council’s chief business officer.
“Increasingly, everybody is conserving cash and deciding whether to cut back on programs.”
Tight Market
In biotech, medical device and life sciences companies, that is problematic. Their programs are research into drugs or medical devices and are the only things these companies have, he said. If they cut back on them, the chances of them succeeding are greatly reduced.
“It’s not a sustainable strategy to put things on hold,” Nasrullah said.
Despite the tightened purse strings of venture capitalists, investors remain very interested in biotechnology. After all, the recently released quarterly Price-waterhouseCoopers MoneyTree report on VC funding for the second quarter of 2009 shows that biotechnology was still the largest single industry category for that period.
The report showed that $1.5 billion went into 260 deals across the country.
Based on current trends, the report projects VC funding for the whole year will probably mirror the levels seen in 1996 and 1997, when funding ranged from $11 billion to $14 billion.
So what options are available to young companies?
For some it means focusing less on interesting angel or venture capital money and more on partnering with or being acquired by a bigger pharmaceutical or biotechnology company.
“In a go-go economy, financing may be a better choice than partnering in the short-term with a bigger company to develop a drug,” said Robert “Duffy” DuFresne, CEO of Ischemix LLC, a small biotech company in Maynard. “It’s a challenging financial environment right now. The impact of that is that it changes the balance between the desirability of partnering with another company or selling the company versus finding the next stage of financing.”
Ischemix researchers have developed a small molecule that acts as a dual-action drug. It blocks oxygen-free radicals and calcium overload, which are the primary causes of muscle damage during and following a heart attack.
DuFresne said his company has talked with larger companies about partnering with them to bring the drug to market, or to be acquired by them. But either of those scenarios is possible only if the company sees a positive outcome of its Phase II clinical trials. If the trial goes well, the results make the company more attractive, he said.
“We’re living in more economically constrained times, but there is a huge need for pharma companies to add attractive programs to their drug pipelines,” DuFresne said.
Patent periods coming due may be driving some of that acquisition and partnering as well.
“Driving the move for a lot of acquisitions of smaller biotechs by big pharma is the fact that a lot of their billion-dollar drugs are coming off patent and that means revenues are falling,” said the MBC’s Nasrullah. Once the patent for a drug ends, generic drug makers can begin entering the market, and they usually do, he said.
The council has instituted a matchmaking program to help both large biotech and pharma companies as well as the smaller biotech companies.
“It’s sort of like speed dating,” Nasrullah said. “We bring all the buyers and sellers together in one place. It’s much more cost effective for big companies than sending teams out all across the country. It’s one stop shopping.”
Nonprofit Route
Very early stage companies have found another way to keep going while awaiting grant money.
“Another option is for companies to partner with disease foundations which are actively investing in small biotech companies,” Nasrullah said.
Local entrepreneur Bradley L. Hodges, with a small biotech called Prothelia Inc. in Milford, has gotten help from a patient advocacy organization to keep going before federal grants kick in this summer. He is working to commercialize a small molecule protein that he believes will help children with muscular dystrophy.
He knew long before he quit his job as a researcher at Genzyme Corp. in Framingham that he would pursue a muscular dystrophy treatment and began communicating early on with nonprofits like the Muscular Dystrophy Association and the Parent Project Muscular Dystrophy, both advocacy and education groups.
Late last year the Parent Project agreed to fund his salary from January to May this year. He could then focus on writing additional grants as he waited for his first two Small Business Innovation Research grants from the federal National Institute of Health. Those two grants total $500,000 and should start any week, he said.
“Because I had my own skin in the game I think they saw me as a good risk,” Hodges said, adding that they knew he had almost exhausted his 401(k) retirement money to get the company started. “It may not have been a lot of money, but it let me keep going.”
Some organizations like the Muscular Dystrophy Association Venture Philanthropy, a spinoff of the MDA, are investing more in small biotech companies to get potential treatments to market more quickly.
While these alternatives to venture capital are necessary to keep small biotech companies going, it doesn’t mean that there isn’t any VC activity. It just means that there is less of it.
Long River Ventures, a small venture capital fund with offices in Worcester and Hadley, participated with other investors in a second quarter, $8 million investment in a Worcester company, Verax Biomedical Inc.
Verax produces a test that quickly determines whether blood supplies are contaminated.
“We’re very busy investing; we think this is a great time to be investing,” said Will Cowen, a Long River partner.
“And there are scrappy entrepreneurs who will find a way to advance their company regardless of the economy.”
Women have been using eye makeup at least as far back as ancient Egypt if not longer. Dark, thick, long eyelashes have always been attractive and using makeup to achieve that beauty is part of many women's daily routine.
Now you can make your eyes more beautiful a different and easier way. Latisse is a new product offered by Allergan that has FDA approval for treating sparse eyelashes (a condition known as hypotrichosis). It is a prescription treatment and provides very noticeable, even dramatic results.
How Latisse Works
Latisse has an active ingredient called bimatoprost. The exact way Latisse works is not known, but it is thought that bimatoprost lengthens the growth phase of the eyelashes. All hair has a three-phase growth cycle:
1. Growth phase
2. Transition phase
3. Dormant phase
By prolonging the growth phase, Latisse causes hairs to grow longer, and more hairs to grow. It must be applied once a day, preferably in the evening, to the base of the upper eyelashes.
How to Use Latisse
Latisse comes in a pack with sterile applicators and a supply of the Latisse solution. Holding the applicator horizontally, you put a drop of the solution near its tip, then draw the applicator along the upper eyelash baseline. It is done much as you would use a liquid eyeliner. If you get some of the solution in your eye, it will not cause any problem.
Each applicator should be used only once and then discarded. This is to guard against any eye infection arising from contaminated applicators. Use a separate applicator for each eye. If there is any excess solution, carefully blot it and next time put slightly less solution on the applicator.
• If you forget to use Latisse one evening, don't use extra to make up for that. Just wait till the next evening's regular dose.
• Avoid touching the tip of the applicator or bumping it against any surfaces
• When you start applying Latisse, mark it on your calendar so that you can document your progress. You might even want to take a few photos for a Before and After set.
Latisse Results
You will start to see the Latisse difference after about four weeks of daily use. After two months and three months you will have longer, darker and thicker eyelashes, and after four months you will see the full results.
The process is gradual and cannot be rushed because the natural growth cycle of hair cannot be rushed. In the clinical trials conducted for FDA approval, 78 percent of the participants achieved significant increase in their eyelash length, thickness and darkness by the end of 16 weeks.
Latisse is not FDA-approved for use on the lower eyelashes, as this was not included in the FDA testing process. So for safety's sake, do not apply it to your lower eyelashes until further research has been done. However, the upper lashes touch the lower lashes when we sleep, so there might be some effect on the lower lashes even though you do not apply it there directly.
Some Possible Side Effects
You might find that your body needs a little time to adjust to Latisse use. At first there might be some redness and itchiness, but typically it will resolve by itself within one or two weeks. In the FDA tests, just four percent of participants had these side effects for a while. It does not mean that your eyes are being harmed.
In rare cases, skin darkening may occur where Latisse is applied. The pigment which creates skin and hair color is melanin and while increasing melanin in the lashes, Latisse could possibly increase it also in the nearby skin. Perhaps that would simply look like a little eyeliner. It would disappear if Latisse use was discontinued.
If you are in the Seattle area and would like to know more about Latisse and whether it would be a good option for you, please contact Bellevue LASIK & Cornea. Dr. Kent Leavitt offers a full range of laser vision correction procedures and is now offering Latisse as an extra service for residents of Seattle, Bellevue, Everett, Olympia, and Tacoma, Washington.

