ACAD
34.93
-0.08
-0.23%
AEMD
3.65
-0.32
-8.13%
APRI
2.26
+0.04
+1.80%
ARNA
1.43
-0.01
-0.69%
ATEC
2.68
-0.12
-4.29%
CNAT
4.4
-0.02
-0.45%
CRXM
0.21
0.00
0.00%
CYTX
1.61
-0.03
-1.83%
DXCM
76.9
+0.41
+0.54%
GNMK
13.02
+0.14
+1.09%
HALO
13.49
+0.2
+1.50%
ILMN
167.79
+4.29
+2.62%
INNV
0.104
+0.001
+0.971%
INO
5.92
+0.06
+1.02%
ISCO
1.75
+0.15
+9.38%
ISIS
57.56
0.00
0.00%
LGND
104.07
+0.02
+0.02%
LPTN
2.93
0.00
0.00%
MBVX
2.45
-0.07
-2.78%
MEIP
1.54
-0.04
-2.53%
MNOV
5.8
-0.01
-0.17%
MRTX
5.15
-0.05
-0.96%
MSTX
0.098
-0.005
-4.6921%
NBIX
41.72
-0.05
-0.12%
NUVA
74.55
+0.42
+0.57%
ONCS
1.26
-0.03
-2.33%
ONVO
3.01
0.00
0.00%
OREX
4
-0.08
-1.96%
OTIC
13.15
+0.9
+7.35%
QDEL
21.31
-0.17
-0.79%
RCPT
231.96
0.00
0.00%
RGLS
1.25
+0.05
+4.17%
RMD
71.29
+0.57
+0.81%
SPHS
2.55
+0.01
+0.39%
SRNE
4.15
0.00
0.00%
TROV
1
-0.08
-6.98%
VICL
2.18
+0.02
+0.93%
VOLC
18
0.00
0.00%
ZGNX
10.35
0.00
0.00%
ACAD
34.93
-0.08
-0.23%
AEMD
3.65
-0.32
-8.13%
APRI
2.26
+0.04
+1.80%
ARNA
1.43
-0.01
-0.69%
ATEC
2.68
-0.12
-4.29%
CNAT
4.4
-0.02
-0.45%
CRXM
0.21
0.00
0.00%
CYTX
1.61
-0.03
-1.83%
DXCM
76.9
+0.41
+0.54%
GNMK
13.02
+0.14
+1.09%
HALO
13.49
+0.2
+1.50%
ILMN
167.79
+4.29
+2.62%
INNV
0.104
+0.001
+0.971%
INO
5.92
+0.06
+1.02%
ISCO
1.75
+0.15
+9.38%
ISIS
57.56
0.00
0.00%
LGND
104.07
+0.02
+0.02%
LPTN
2.93
0.00
0.00%
MBVX
2.45
-0.07
-2.78%
MEIP
1.54
-0.04
-2.53%
MNOV
5.8
-0.01
-0.17%
MRTX
5.15
-0.05
-0.96%
MSTX
0.098
-0.005
-4.6921%
NBIX
41.72
-0.05
-0.12%
NUVA
74.55
+0.42
+0.57%
ONCS
1.26
-0.03
-2.33%
ONVO
3.01
0.00
0.00%
OREX
4
-0.08
-1.96%
OTIC
13.15
+0.9
+7.35%
QDEL
21.31
-0.17
-0.79%
RCPT
231.96
0.00
0.00%
RGLS
1.25
+0.05
+4.17%
RMD
71.29
+0.57
+0.81%
SPHS
2.55
+0.01
+0.39%
SRNE
4.15
0.00
0.00%
TROV
1
-0.08
-6.98%
VICL
2.18
+0.02
+0.93%
VOLC
18
0.00
0.00%
ZGNX
10.35
0.00
0.00%
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Invitae announces expansion of its genetic testing offering to include unique test for Spinal Muscular Atrophy (SMA) during the 2017 ACMG Annual Clinical Genetics Meeting

March 22, 2017 – 3:30 am

SAN FRANCISCO, March 22, 2017 /PRNewswire/ — Invitae Corporation (NYSE: NVTA), one of the fastest growing genetic information companies, today announced the availability of a new genetic test for the diagnosis of Spinal Muscular Atrophy (SMA), a neuromuscular disease that is one of the leading lethal genetic disorders among infants as well as a significant cause of progressive neuromuscular disease in childhood. The new test, announced during the American College of Medical Genetics (ACMG) Annual Clinical Genetics Meeting, features a novel, custom methodology that offers significant improvements over current testing approaches.

SMA is an autosomal recessive disorder and the second leading genetic disease in infancy behind cystic fibrosis affects. SMA affects approximately one in every 10,000 infants, while one in 50 people is a carrier for the disorder. The majority of SMA cases are caused by loss of the gene SMN1, with variation in the number of copies of related gene, SMN2, playing a role in mitigating the severity of the disease. Loss of SMN1 results in the absence of a protein necessary for the normal function of nerves governing movement. Without adequate nerve function, patients with SMA experience progressive muscle weakness and atrophy, impacting the ability to crawl, sit, or stand and eventually to breathe or swallow.

Accurate testing of both genes is critical for the diagnosis and treatment of SMA. Testing for the absence of functional SMN1 is the basis of diagnosis and can distinguish the condition from other neuromuscular diseases such as muscular dystrophy. Understanding the number of copies of the SMN2 gene can provide prognostic information and help guide therapeutic choices and clinical trial participation. However, due to the similarities between the two genes, testing is technically challenging and reliably accurate tests have not previously been widely available.

“Determining SMN2 copy number accurately has, in the past, been a challenge. Improvement in the SMN2 copy number assay will provide better diagnostic and prognostic information about SMA patients that will guide clinicians with appropriate disease management,” said Perry Shieh, MD, associate professor and director of the neuromuscular program at the University of California Los Angeles.

Invitae’s unique approach leverages advanced next generation sequencing and a customized bioinformatics solution to accurately identify sequence changes and copy number changes in both genes from a single test. Analysis of SMN1 and SMN2 is now available from Invitae as a stand-alone test, and it has been added to a number of the company’s comprehensive neuromuscular and neuropathy panels, allowing physicians the ability to test for SMA alongside a number of other neurological disorders for no additional cost.

“In addition to the use of the Invitae SMA test in the diagnosis of infants and children with early onset neuromuscular disease, the recent development of new therapies directed at SMA is leading to the possible inclusion of SMN1 testing in universal newborn screening panels, which will mean in turn that having an accurate, cost-effective confirmatory test is essential,” said Robert Nussbaum, MD, chief medical officer at Invitae. “We’re proud to bring this advancement in testing to the SMA community.”

Affordable, transparent pricing
The new SMA test will be provided at the same pricing as all other Invitae tests. Invitae offers a transparent pricing structure independent of the number of genes required to provide an accurate diagnosis within a single clinical area. Invitae has secured in-network status with the major national health insurance payers. For third-party payers with whom Invitae is out-of-network and for non-contracted institutions, the price per test in a clinical area is $1,500. In addition, for patients without insurance coverage or who do not meet insurance criteria for coverage, Invitae offers its full test offerings for $475 per test in a clinical area.

About Invitae
Invitae Corporation’s (NYSE: NVTA) mission is to bring comprehensive genetic information into mainstream medical practice to improve the quality of healthcare for billions of people. Invitae’s goal is to aggregate most of the world’s genetic tests into a single service with higher quality, faster turnaround time, and lower price than many single-gene and panel tests today. The company currently provides a diagnostic service comprising hundreds of genes for a variety of genetic disorders associated with oncology, cardiology, neurology, pediatrics, and other rare disease areas. Additionally, the company has created a Genome Network to connect patients, clinicians, advocacy organizations, researchers, and drug developers to accelerate the understanding, diagnosis, and treatment of hereditary disease. For more information, visit our website at invitae.com.

Safe Harbor Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to the company’s belief that its new genetic test for the diagnosis of Spinal Muscular Atrophy (SMA) offers significant improvements over current testing approaches, which will provide better diagnostic and prognostic information about SMA patients that will guide clinicians with appropriate disease management; and that the new therapies directed at SMA is leading to the possible inclusion of the testing of the gene SMN1 in universal newborn screening panels, which in turn will mean that having an accurate, cost-effective confirmatory test is essential. Forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially, and reported results should not be considered as an indication of future performance. These risks and uncertainties include, but are not limited to:  the company’s history of losses; the company’s need to scale its infrastructure in advance of demand for its tests and to increase demand for its tests; the company’s ability to develop and commercialize new tests and expand into new markets; the risk that the company may not obtain or maintain sufficient levels of reimbursement for its tests; risks associated with the company’s ability to use rapidly changing genetic data to interpret test results accurately, consistently, and quickly; the company’s ability to compete; and the other risks set forth in the company’s filings with the Securities and Exchange Commission, including the risks set forth in the company’s Annual Report on Form 10-K for the year ended December 31, 2016. These forward-looking statements speak only as of the date hereof, and Invitae Corporation disclaims any obligation to update these forward-looking statements.

NOTE: Invitae and the Invitae logo are trademarks of Invitae Corporation. All other trademarks and service marks are the property of their respective owners.

Contact:
Laura D’Angelo
pr@invitae.com
314-920-0617

 

SOURCE Invitae Corporation

Join David Baltimore, Nicole Glaros, Nathan Myhrvold, & More at Our Napa Summit

March 22, 2017 – 3:15 am

Year after year, Xconomy has gathered exemplary business leaders, investors, and far-seeing technologists to our most prestigious event—The Napa Summit. This year is no different. Come join us…

[[Click headline to continue reading.]]

Imprimis Pharmaceuticals Announces Fourth Quarter and Year-End 2016 Financial Results

March 21, 2017 – 12:08 pm

SAN DIEGO, March 21, 2017 /PRNewswire/ — Imprimis Pharmaceuticals, Inc. (NASDAQ: IMMY), an ophthalmology-focused pharmaceutical company, today reported financial results for the fourth quarter and year ended December 31, 2016.  

Key Fourth Quarter Financial Highlights and Recent Developments

  • Total revenue of $5.8 million in the fourth quarter of 2016, up 65% compared to revenue of $3.5 million reported for the same quarter a year ago, and representing a 19% increase compared to revenue of $4.9 million in third quarter 2016. Total revenue for the year 2016 was $19.9 million, a 105% increase compared to $9.7 million reported for the full-year 2015.
  • Ophthalmology-related sales were $3.6 million in the fourth quarter, representing a 20% quarterly growth rate compared to $3.0 million reported in the third quarter 2016. For the year 2016, ophthalmology-related sales were $11.0 million, an over 200% increase compared to $3.1 million reported for full-year 2015.
  • Loss from operations of $3.5 million and net loss of $6.1 million in fourth quarter of 2016, compared to loss from operations of $4.7 million and net loss of $5.1 million reported the same quarter of the prior year.
  • Adjusted EBITDA loss for the fourth quarter of 2016 was $2.4 million, an improvement compared to an Adjusted EBITDA loss of $3.0 million reported in the fourth quarter of 2015.
  • Gross margin reported in the fourth quarter 2016 was 47% compared to 44% for the same quarter the prior year. The company expects gross margins to increase during 2017 as a result of increased production, labor and ordering efficiencies at the New Jersey outsourcing facility, and implementation of previously-announced cost-reduction programs to decrease cash-based expenses by $3 million annually.
  • Completed a private placement transaction of common stock for a total of approximately $10.1 million in gross proceeds, before deducting underwriting and other customary expenses. The company’s CEO, CFO, Sr. Director of Corporate Development and a Board member participated in the offering.
  • With a compounded annual growth rate from 2014-2016 of 245%, Imprimis was recently ranked the 4th fastest growing biotech/pharmaceutical company and the 12th overall fastest growing company on Deloitte’s Technology Fast 500™ ranking of North American companies.
  • Commenced production of Imprimis’ core sterile ophthalmic formulations from the company’s new state-of-the-art FDA-registered outsourcing facility complying with current good manufacturing practice (cGMP) requirements for outsourcing facilities and enabling a simplified customer ordering process.

Mark L. Baum, CEO of Imprimis, stated, “We are pleased with our record growth in the fourth quarter and for the fiscal year 2016.  Last year was a strategically critical time as we completed our New Jersey 503B outsourcing facility and registered it with the FDA and also assembled a seasoned senior management team to lead us into 2017 and beyond.  We expect our new 503B facility will play an important role as our customer network expands and we enter new ophthalmic markets.  Our goal is to continue growing market share in our core ophthalmology markets, cataract and refractive surgeries, while selectively targeting new larger markets within ophthalmology.  In the first half of the year, we expect to launch our glaucoma strategy, which will include a series of preservative-free combination drops never before available in the U.S.  We are also planning to make repackaged Avastin for wet age-related macular degeneration (Wet AMD) and diabetic macular edema (DME), and new mydriatics and anesthetics for ocular surgery.  As we end the first half, we expect to launch new formulations for infection and inflammation targeted at the ophthalmic and optometric markets.  During the second half of 2017, we anticipate launching our dry eye disease (DED) program which our team has been developing over the past year.” 

“We have an exciting future as we continue to innovate new products and create value for our shareholders from our growing base of drug assets.  I am confident the investments we have made to date will drive meaningful financial progress and allow us to reach our near-term goal of profitability, while ensuring a stable platform for our long-term growth.  The entire Imprimis team remains steadfast in our commitment to the company’s mission and vision of providing high-quality innovative medications to physicians and their patients at affordable prices while building value for our shareholders.” 

Recent Commercialization and Corporate Developments

  • Dropless Therapy® and LessDrops® combination topical drops are capturing an estimated 10 percent market share from large eye drop companies.
    • Serviced over 600,000 ocular surgeries since launch in April 2014.
    • Expanded customer base to over 1,500 ophthalmologists.
    • Growing a strong library of published clinical data. A recently-announced peer-reviewed paper provided a retrospective review of the efficacy of Dropless® and its benefits to patients, physicians and their staff.
  • Over 120 physicians are now prescribers of the IV Free MKO Melt sublingual conscious sedation formulation, an option to intravenous anesthetic for patients undergoing ocular and other surgical procedures. Imprimis intends to add the MKO Melt to its 503B facility portfolio in 2017 eliminating the need for patient-specific prescriptions and allowing for volume orders. During the year, the company plans to support two MKO Melt investigator-initiated studies at major U.S. teaching organizations focused in ophthalmology and dentistry. Additional surgical market opportunities for the MKO Melt include dental procedures, colonoscopies, prostatectomies, women’s health, dermatology procedures and vasectomies, representing over 70 million procedures performed annually in the U.S.
  • Entered into an agreement with the specialty pharmacy division of one of the largest pharmacy benefit managers in the country to supply Imprimis’ complete formulary through its national network of specialty pharmacies. This renowned PBM serves more than 65 million patients and dispenses one billion prescriptions annually.
  • Strengthened intellectual property (IP) portfolio in ophthalmology and other technologies. Imprimis now owns 27 key domestic and international patents or pending patent applications and over 150 U.S. and international trademarks have been issued or pending supportive of the company’s commercial sales and marketing activities.

ImprimisRx Pharmacy Operations

  • In February 2017 began shipments of core sterile ophthalmic formulations from the company’s New Jersey FDA-registered outsourcing facility in compliance with cGMP requirements for outsourcing facilities. Customers can register for an ImprimisRx 503B account to purchase the company’s flagship Dropless and LessDrops medications in convenient 20-unit boxes without the need for patient-specific prescriptions at http://www.imprimisrx.com/503b-prereg/. The facility is fully equipped with automated filling and labeling robotics and a new integrated order and fulfillment system that bypasses customer service and moves orders directly to the facility’s fulfillment center. The investments made to increase production and ordering efficiencies are expected to increase the customer experience and drive sales growth and increase margins.

Financial Summary

Selected highlights regarding operating results for the three months and full year ended December 31, 2016 and for the same periods in 2015 are as follows (in thousands, except per share data):

For the three months
ended December 31, 2016

For the three months
ended December 31, 2015

Total Revenues

$5,793

$3,503

Cost of Sales

3,071

1,947

  Gross Profit

2,722

1,556

Selling & Marketing Expenses

1,415

2,041

General & Administrative Expenses

4,213

4,177

Research & Development Expenses

601

33

Total Other Expense, net

2,554

429

Net Loss

$(6,102)

$ (5,124)

Net Loss per Common Share

$(0.44)

$ (0.53)

 

For the year ended
December 31, 2016

For the year ended
December 31, 2015

Total Revenues

$19,942

$9,716

Cost of Sales

9,831

5,206

  Gross Profit

10,111

4,510

Selling & Marketing Expenses

7,382

6,496

General & Administrative Expenses

17,569

12,504

Research & Development Expenses

739

332

Impairment of intangible assets and goodwill

303

Total Other Expense, net

3,205

1,077

Net Loss

$(19,087)

$(15,899)

Net Loss per Common Share

$(1.50)

$(1.66)

 

Adjusted EBITDA

In addition to the company’s results of operations determined in accordance with U.S. generally accepted accounting principles (GAAP), which are presented and discussed above, management also utilizes adjusted EBITDA, an unaudited financial measure that is not calculated in accordance with GAAP, to evaluate the company’s financial results and performance and to plan and forecast future periods. Adjusted EBITDA is considered a “non-GAAP” financial measure within the meaning of Regulation G promulgated by the SEC.  Management believes that this non-GAAP financial measure reflects an additional way of viewing aspects of the company’s operations that, when viewed with GAAP results, provides a more complete understanding of the company’s results of operations and the factors and trends affecting its business.  Management believes adjusted EBITDA provides meaningful supplemental information regarding the company’s performance because (i) it allows for greater transparency with respect to key metrics used by management in its financial and operational decision-making; (ii) it excludes the impact of non-cash or, when specified, non-recurring items that are not directly attributable to the company’s core operating performance and that may obscure trends in the company’s core operating performance; and (iii) it is used by institutional investors and the analyst community to help analyze the company’s results.  However, adjusted EBITDA and any other non-GAAP financial measures should be considered as a supplement to, and not as a substitute for, or superior to, the corresponding measures calculated in accordance with GAAP. Further, non-GAAP financial measures used by the company and the manner in which they are calculated may differ from the non-GAAP financial measures or the calculations of the same non-GAAP financial measures used by other companies, including the company’s competitors.

The company defines adjusted EBITDA as net income (loss) excluding the effects of interest, taxes, depreciation, amortization, stock-based compensation, other income (expense) and, if any and when specified, other non-recurring income or expense items.  The company believes that the most directly comparable GAAP financial measure to adjusted EBITDA is net loss. Adjusted EBITDA has limitations and should not be considered as an alternative to gross profit or net loss as a measure of operating performance or to net cash provided by (used in) operating, investing or financing activities as a measure of ability to meet cash needs.

The following is a reconciliation of adjusted EBITDA, a non-GAAP measure to the most comparable GAAP measure, net loss, for the three months ended December 31, 2016 and 2015 (in thousands):

For the three months ended
December 31, 2016

Net Loss

$ (6,102)

  Stock-based compensation

684

  Interest expense, net

782

  Taxes

(111)

  Depreciation

356

  Amortization of intangible assets

89

  Non-recurring expenses(1)

1,966

  Impairment of intangible assets and goodwill

  Other income, net

(42)

  Adjusted EBITDA

$ (2,378)

(1)

Non-recurring expense items include one-time cost for extinguishment of debt.

 

For the three months ended
December 31, 2015

Net Loss

$ (5,124)

  Stock-based compensation

1,124

  Interest expense, net

429

  Taxes

  Depreciation

69

  Amortization of intangible assets

91

  Non-recurring expenses(2)

442

  Other income, net

  Adjusted EBITDA

$ (2,969)

(2)

Non-recurring expense items include certain transactional expenses and expenses related to restructuring the company’s sales and marketing efforts, including severance expenses.

 

Conference Call and Webcast

The company’s management team will host a conference call and audio-only webcast today at 4:30 p.m. EST (1:30 p.m. PST) to discuss the financial results and recent developments.  To participate in the call, please dial (877)-407-8035 for domestic callers or (201)-689-8035 for international callers.  To listen to the webcast, please click here or visit the investor relations section of the Imprimis website at www.ImprimisRx.com.  A replay of the call will be available until April 21, 2017.  To access the replay, dial (877)-481-4010 domestically or (919)-882-2331 internationally and reference Conference ID: 10212. 

About Imprimis Pharmaceuticals

Imprimis Pharmaceuticals, Inc. (NASDAQ: IMMY) is a pharmaceutical company dedicated to producing and dispensing high quality innovative medications in all 50 states.  The company’s unique business model increases patient access and affordability to many critical medicines.  Headquartered in San Diego, California, Imprimis owns and operates three production and dispensing facilities located in California, New Jersey and Pennsylvania. For more information about Imprimis, please visit the corporate website at www.ImprimisRx.com

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the U.S. Private Securities Litigation Reform Act of 1995. Any statements in this release that are not historical facts may be considered such “forward looking statements.” Forward-looking statements are based on management’s current expectations and are subject to risks and uncertainties which may cause results to differ materially and adversely from the statements contained herein. Some of the potential risks and uncertainties that could cause actual results to differ from those predicted include our ability to make commercially available our compounded formulations and technologies in a timely manner or at all; physician interest in prescribing our formulations; risks related to our compounding pharmacy operations; our ability to enter into other strategic alliances, including arrangements with pharmacies, physicians and healthcare organizations for the development and distribution of our formulations; our ability to obtain intellectual property protection for our assets; our ability to accurately estimate our expenses and cash burn, and raise additional funds when necessary; risks related to research and development activities; the projected size of the potential market for our technologies and formulations; unexpected new data, safety and technical issues; regulatory and market developments impacting compounding pharmacies, outsourcing facilities and the pharmaceutical industry; competition; and market conditions. These and additional risks and uncertainties are more fully described in Imprimis’ filings with the Securities and Exchange Commission, including its Annual Report on Form 10-K and its Quarterly Reports on Form 10-Q. Such documents may be read free of charge on the SEC’s web site at www.sec.gov. Undue reliance should not be placed on forward looking statements, which speak only as of the date they are made. Except as required by law, Imprimis undertakes no obligation to update any forward-looking statements to reflect new information, events or circumstances after the date they are made, or to reflect the occurrence of unanticipated events.

Other than drugs compounded at a registered outsourcing facility, all Imprimis compounded formulations may only be prescribed pursuant to a physician prescription for an individually identified patient consistent with federal and state laws.

Media Contact
Deb Holliday
deb@pascalecommunications.com
412-877-4519

Investor Contact
Bonnie Ortega
bortega@imprimispharma.com
858-704-4587

 

SOURCE Imprimis Pharmaceuticals, Inc.

Viking Therapeutics Reports Fourth Quarter and Year-End 2016 Financial Results and Provides Corporate Update

March 21, 2017 – 12:05 pm

– VK5211 Phase 2 study in hip fracture proceeding, with results expected mid-year- VK2809 Phase 2 study in hypercholesterolemia and fatty liver disease proceeding, with results expected in late 2017- New program initiated to evaluate VK2809 in patients with glycogen storage disease type Ia (GSD Ia)

SAN DIEGO, March 21, 2017 /PRNewswire/ — Viking Therapeutics, Inc. (“Viking”) (NASDAQ: VKTX), a clinical-stage biopharmaceutical company focused on the development of novel therapies for metabolic and endocrine disorders, today announced its financial results for the fourth quarter and year ended December 31, 2016, and provided an update on its clinical pipeline and other corporate developments.

Highlights from, and Subsequent to, the Quarter Ended December 31, 2016
“We made excellent progress with multiple pipeline programs in 2016.  We continued enrolling patients into the Phase 2 trial of our selective androgen receptor modulator VK5211 for hip fracture, initiated a Phase 2 trial with our novel thyroid receptor beta agonist VK2809 in fatty liver disease and hypercholesterolemia, began planning for a proof-of-concept trial with VK2809 in GSD Ia, a new orphan indication, and reported positive proof-of-concept data from our second thyroid receptor agonist, VK0214, in an in vivo model of the orphan disease X-linked adrenoleukodystrophy.  We expect this momentum to continue in 2017 as we plan to announce the results from the hip fracture study mid-year, followed later in the year by the results from the fatty liver and hypercholesterolemia trial.  Each of these events will mark a key milestone for the company, providing clinical data in indications that each represent significant potential revenue opportunities,” stated Brian Lian, Ph.D., chief executive officer of Viking.  “We are also continuing our strategic partnerships with the Kennedy Krieger Institute and Duke University, which together provide valuable support as we work to advance our two orphan programs into clinical development.  We believe VK2809 in GSD Ia and VK0214 in X-ALD each represent important opportunities to apply our novel science to areas of high unmet medical need.  Finally, we were able to accomplish all of this while maintaining a lean operating model, ending the year with a strong cash position at over $13 million, and retaining access to approximately $14 million in additional funding through our current equity line and ATM program.”

Pipeline and Corporate Highlights

  • Phase 2 clinical trial of VK5211 in patients recovering from hip fracture surgery nearing complete enrollment.  VK5211 is a novel, orally available, non-steroidal small molecule selective androgen receptor modulator (SARM) which has been shown to have a stimulatory effect on lean body mass and bone mineral density, and may offer significant benefits to patients recovering from hip fracture surgery.  The ongoing international trial continues enrolling patients with recent fractures, targeting 120 total subjects across four treatment arms.  Patients receive once daily oral doses of VK5211 for 12-weeks, and will be assessed for changes in lean body mass, the trial’s primary endpoint, as well as various exploratory endpoints evaluating functional and quality of life measures.  Viking currently expects data from this study to be available in mid-2017.
  • Phase 2 clinical trial of VK2809 in hypercholesterolemia and fatty liver disease is progressing.  VK2809 is a novel, orally available small molecule thyroid receptor agonist that possesses selectivity for liver tissue as well as the beta receptor subtype, suggesting promise in this patient population.  The ongoing trial is enrolling up to 80 patients with hypercholesterolemia and fatty liver disease.  The company currently expects to complete this trial in late 2017. 
  • New program initiated to evaluate VK2809 for the treatment of glycogen storage disease type Ia (GSD Ia).  GSD Ia is an orphan genetic disease that results in an excess accumulation of glycogen and lipids in the liver, potentially leading to hepatic steatosis, hepatic adenomas, and hepatocellular carcinoma.  Initial results from an in vivo proof-of-concept study showed that treatment with VK2809 produced rapid and substantial reductions in liver triglyceride content, liver weight and liver weight as a percentage of body weight compared with vehicle-treated controls.  Mean liver triglyceride content was reduced by more than 60% in VK2809-treated animals relative to vehicle-treated control animals, while average liver weight was reduced by more than 30% vs. controls.  Complete results from this study, which is being conducted under a sponsored research agreement between Duke University and Viking, will be presented at a future scientific meeting.
  • Evaluation of VK0214 in X-linked adrenoleukodystrophy (X-ALD) advancing.  VK0214 is a novel, orally available small molecule thyroid receptor agonist that possesses selectivity for the beta receptor subtype.  Encouraging initial results from an in vivo study of VK0214 in the ABCD1 knockout model of X-ALD were presented at the American Thyroid Association meeting in September 2016.  This study successfully achieved its primary objective, demonstrating the ability of VK0214 to lower plasma very long chain fatty acids (VLCFAs) after six weeks of treatment.  The accumulation of VLCFAs is believed to contribute to the underlying pathology of X-ALD.  Following this initial study, in Q4 2016, the company, in collaboration with the Kennedy Krieger Institute, initiated a longer-tem study to evaluate the effects of VK0214 in this model.  Initial results from this study are expected to be available in 2Q17. 
  • Funding secured to advance GSD Ia program.  In February 2017, Viking entered into an agreement with a dedicated healthcare investment fund to support the initial clinical development of the company’s GSD Ia program.  With this funding, the company plans to file an Investigational New Drug (IND) application for VK2809 for the treatment of patients with GSD Ia and initiate a human proof-of-concept study in the second half of 2017. 

Financial Highlights
Quarter Ended December 31, 2016 and 2015

Research and development expenses for the three months ended December 31, 2016 were $2.6 million compared to $3.2 million for the same period in 2015. The decrease was primarily due to decreased activities related to third party manufacturing of our clinical-stage drug candidates. 

General and administrative expenses for the three months ended December 31, 2016 were $1.1 million compared to $1.4 million for the same period in 2015. The decrease was primarily due to decreases in stock-based compensation expense. 

For the three months ended December 31, 2016, Viking reported a net loss of $3.6 million, or $0.18 per share, compared to a net loss of $5.1 million, or $0.56 per share, in the corresponding period in 2015.

Year Ended December 31, 2016 and 2015

Research and development expenses for the year ended December 31, 2016 were $9.0 million compared to $7.0 million for the same period in 2015. The increase in research and development expenses was primarily related to increases in expenses related to clinical trial activity for our VK5211 and VK2809 programs and preclinical efforts for our VK0214 program. 

General and administrative expenses for the year ended December 31, 2016 were $4.8 million compared to $5.0 million for the same period in 2015. The decrease in general and administrative expenses was primarily related to a decrease in stock-based compensation expense, offset by an increase in costs related to being a publicly traded company.

For the year ended December 31, 2016, Viking reported a net loss of $14.7 million, or $0.90 per share, compared to a net loss of $23.4 million, or $3.68 per share, in the comparable period in 2015. The decrease in net loss in the year ended December 31, 2016 was primarily due to a change in fair value of accrued license fees expense of $9.4 million recorded in 2015 with no comparable expense in 2016.

Balance Sheet as of December 31, 2016

At December 31, 2016, Viking held cash, cash equivalents and investments totaling $13.2 million.  As of February 28, 2017, Viking had 23,825,425 shares of common stock outstanding.

Conference Call
To participate on the conference call, please dial (877) 870-4263 from the U.S. or (412) 317-0790 from outside the U.S. In addition, following the completion of the call, a telephone replay will be accessible until April 3, 2017 by dialing (877) 344-7529 from the U.S. or (412) 317-0088 from outside the U.S. and entering conference ID # 10102405.  Those interested in listening to the conference call live via the internet may do so by visiting the Investor Relations section of Viking’s website at www.vikingtherapeutics.com. An archive of the webcast will be available for 30 days on the company’s website at www.vikingtherapeutics.com.

About Viking Therapeutics, Inc. 
Viking Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on the development of novel, first-in-class or best-in-class therapies for metabolic and endocrine disorders.  The company’s research and development activities leverage its expertise in metabolism to develop innovative therapeutics designed to improve patients’ lives.  Viking has exclusive worldwide rights to a portfolio of five therapeutic programs in clinical trials or preclinical studies, which are based on small molecules licensed from Ligand Pharmaceuticals Incorporated.  The company’s clinical programs include VK5211, an orally available, non-steroidal selective androgen receptor modulator, or SARM, in Phase 2 development for the treatment and prevention of lean body mass loss in patients who have undergone hip fracture surgery, VK2809, a small molecule thyroid beta agonist in Phase 2 development for hypercholesterolemia and fatty liver disease, and VK0612, a first-in-class, orally available drug candidate in Phase 2 development for type 2 diabetes.  Viking is also developing novel and selective agonists of the thyroid beta receptor for GSD Ia and X-linked adrenoleukodystrophy, as well as two earlier-stage programs targeting metabolic diseases and anemia.

Forward-Looking Statements
This press release contains forward-looking statements regarding Viking Therapeutics, including statements about Viking’s expectations regarding its development activities, expected timing for clinical trial screening, enrollment and completion and the announcement of clinical trial data, VK5211’s, VK2809’s and VK0214’s potential to produce therapeutic benefits and create significant revenue opportunities for the company, expectations regarding an IND application for VK2809, and Viking’s ability to use its equity line and ATM program for raising capital. Forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially and reported results should not be considered as an indication of future performance. These risks and uncertainties include, but are not limited to: risks associated with the success, cost and timing of Viking’s product candidate development activities and clinical trials; and risks regarding regulatory requirements, among others discussed in the “Risk Factors” section of our most recent periodic reports filed with the Securities and Exchange Commission (SEC), including our most recent Form 10-K and Form 10-Q, all of which you may obtain for free on the SEC’s website at www.sec.gov. These forward-looking statements speak only as of the date hereof.  Viking disclaims any obligation to update these forward-looking statements.

Viking Therapeutics, Inc.

Statements of Operations and Comprehensive Loss

(Unaudited)

Three Months Ended December 31,

Year Ended December 31,

2016

2015

2016

2015

Revenues

$

$

$

$

Operating expenses:

Research and development

2,647,469

3,219,414

9,000,499

6,966,842

General and administrative

1,090,054

1,400,889

4,846,776

5,029,636

Total operating expenses

3,737,523

4,620,303

13,847,275

11,996,478

Loss from operations

(3,737,523)

(4,620,303)

(13,847,275)

(11,996,478)

Other income (expense):

Change in fair value of accrued license fees

(9,381,848)

Change in fair value of debt conversion feature liability

620,087

(216,841)

1,064,170

(1,043,478)

Amortization of debt discount

(431,227)

(240,516)

(1,788,088)

(893,502)

Amortization of financing costs

(92,849)

(138,701)

Interest expense, net

(3,098)

(13,303)

(21,928)

(88,682)

Total other income (expense)

92,913

(470,660)

(884,547)

(11,407,510)

Net loss

(3,644,610)

(5,090,963)

(14,731,822)

(23,403,988)

Other comprehensive loss, net of tax:

Unrealized gain (loss) on securities

(755)

(4,848)

478

(7,370)

Comprehensive loss

$

(3,645,365)

$

(5,095,811)

$

(14,731,344)

$

(23,411,358)

Basic and diluted net loss per share

$

(0.18)

$

(0.56)

$

(0.90)

$

(3.68)

Weighted-average shares used to compute basic
  
and diluted net loss per share

19,930,096

9,012,768

16,278,292

6,355,869

 

Viking Therapeutics, Inc.

Balance Sheets

December 31,

2016

(unaudited)

December 31,

2015

Assets

Current assets:

Cash and cash equivalents

$

3,075,502

$

768,550

Short-term investments – available for sale

10,075,058

13,335,499

Prepaid expenses and other current assets

824,269

1,097,599

Total current assets

13,974,829

15,201,648

Deferred public offering and other financing costs

521,538

157,455

Deposits

39,341

80,000

Total assets

$

14,535,708

$

15,439,103

Liabilities, convertible notes and stockholders equity (deficit)

Current liabilities:

Accounts payable

$

1,203,888

$

592,414

Other accrued liabilities

1,237,122

1,384,398

Accrued interest

34,894

Convertible notes payable, current portion (net of discount of $675,589 and $0 at December 31, 2016 and 2015, respectively)

3,269,582

Debt conversion feature liability

731,048

Total current liabilities

6,476,534

1,976,812

Accrued interest, non-current

183,611

Convertible notes payable (net of discount of $0 and $348,460 at December 31, 2016 and 2015, respectively)

2,151,540

Debt conversion feature liability

2,370,903

Deferred rent

16,307

31,239

Total long-term liabilities

16,307

4,737,293

Total liabilities

6,492,841

6,714,105

Commitments and contingencies

Stockholders’ equity:

Preferred stock, $0.00001 par value: 10,000,000 shares authorized at December 31, 2016 and 2015; no shares issued and outstanding at December 31, 2016 and 2015

Common stock, $0.00001 par value: 300,000,000 shares authorized at December 31, 2016 and 2015; 20,823,873 shares issued and outstanding at December 31, 2016 and 9,683,741 shares issued and outstanding at December 31, 2015

208

97

Additional paid-in capital

68,326,818

54,277,716

Accumulated deficit

(60,277,267)

(45,545,445)

Accumulated other comprehensive loss

(6,892)

(7,370)

Total stockholders’ equity

8,042,867

8,724,998

Total liabilities and stockholders’ equity

$

14,535,708

$

15,439,103

Follow Viking on Twitter @Viking_VKTX.

 

SOURCE Viking Therapeutics, Inc.

goBalto Awarded Top 20 Most Promising Biotech Technology Solution Providers 2017

March 21, 2017 – 9:00 am

Industry proven study startup process optimization key to selection by CIOReview Magazine

SAN FRANCISCO, March 21, 2017 /PRNewswire/ — goBalto, Inc., the leading provider of cloud-based clinical study startup solutions, has been selected by CIOReview for the second consecutive time as one of the 20 Most Promising Biotech Technology Solution Providers 2017.

The award selection by honorary industrial experts is based on evaluation of goBalto‘s specialties in helping pharmaceutical and clinical research organizations (CRO) optimize study startup processes and reduce waste; optimization that supports seamless integration into these companies’ eClinical ecosystems, thereby offering the flexibility and robustness needed to accelerate the clinical trials process.

Study Startup (SSU) refers to a series of steps performed before a clinical trial begins. These steps typically include activities such as country selection, pre-study visits, site selection and initiation, regulatory document submission, contract and budget negotiations, and enrolling the first patient. Since each of these steps has multiple components, delays in study timelines are commonplace.

“It’s a great honor to include goBalto, in our annual ranking list of 20 Most Promising Biotech Technology Solution Providers 2017,” said Jeevan George, Managing Editor of CIOReview. “goBalto is the only company offering a complete end-to-end platform for starting clinical trials, from site identification, feasibility assessment and selection through to activation, with comprehensive metrics to track adherences to timeline and budget.”

“We are honored that our technology has been acknowledged among the best solutions in the industry for the second year in a row,” said Sujay Jadhav, goBalto‘s CEO. “The CIOReview Awards’ recognition of our solutions for accelerating the end-to-end processes for starting clinical trials supports our continued efforts, and that of our customers and partners, to advance SSU.”

With recent multiple enterprise expansions goBalto now services more than two-thirds of the top 25 global pharmas and three of the top five CROs, having the largest industry-proven set of country specific regulatory business process workflows and associated in-depth activation metrics.

“Our growth momentum reflects adoption of our innovative efforts by life science organizations engaged in running trials to mitigate risk, ensure compliance, reduce cycle times, minimize costs, and ultimately enable valuable therapies to get to patients sooner,” added Jadhav.

About CIOReview
Published from Fremont, California, CIOReview is a print magazine that explores and understands the plethora of ways adopted by firms to execute the smooth functioning of their businesses. A distinguished panel comprising of CEOs, CIOs, VCs, analysts including CIOReview editorial board finalized the “20 Most Promising BioTech Technology Solution Providers 2017” in the U.S. and shortlisted the best vendors and consultants. For more info: www.cioreview.com.

About goBalto
goBalto is the industry leader in cloud-based study startup software for the global life sciences industry, offering the only complete end-to-end platform for starting clinical trials, from site identification, feasibility assessment and selection through to activation, with comprehensive metrics to track adherence to timelines and budget. Committed to accelerating clinical trials through innovation, product excellence, and customer success, goBalto works with three of the top five CROs and more than two-thirds of the top 25 pharmas. Our customers include: Allergan, Covance, CMIC HOLDINGS, Genentech Roche, ICON, INC Research, Novartis, and PSI CRO. goBalto is headquartered in San Francisco, with offices in Pennsylvania and Singapore. For more information, visit www.gobalto.com.

Press contact:
Craig Morgan
415.671.4372
cmorgan@gobalto.com

 

SOURCE goBalto, Inc.

Tarrex Biopharma Announces FDA Acceptance of IND Application for TX803 to Commence Phase I Clinical Trials in Colorectal Cancer Patients

March 21, 2017 – 5:00 am

SAN DIEGO, March 21, 2017 /PRNewswire/ — Tarrex Biopharma, Inc. today announced that the U.S. Food and Drug Administration (FDA) has accepted the Company’s Investigational New Drug (IND) application for TX803, a novel treatment for colorectal cancer. “This marks the first significant milestone for Tarrex in our efforts to develop innovative cancer therapeutics to address unmet medical needs and we are very excited that we will be able to start testing TX803 in advanced colorectal cancer patients,” said Kanyin E. Zhang, Ph.D. CEO of Tarrex.

TX803 is a first-in-class small molecule therapeutic that inhibits the PI3K/AKT pathway through a novel mechanism – by targeting, not the kinase itself, but a PI3K activating onco-protein, the truncated retinoid X receptor alfa (tRXRα). Moreover, tRXRα is found in high levels in tumor tissues obtained from a significant portion of colorectal, gastric and breast cancer patients. Preclinical studies have demonstrated that TX803 is orally bioavailable, well tolerated, and efficacious in several animal tumor models including KRAS mutated colorectal cancer. Tarrex has licensed the worldwide rights of TX803 and related intellectual properties from the Sanford Burnham Prebys Medical Discovery Institute in San Diego, California.

“Approximately 50% of colorectal cancer patients have tumors with a KRAS or NRAS mutation, which renders them resistant to anti-EGFR therapies, such as cetuximab and panitumumab. If clinically proven, TX803 has the potential to address a significant unmet need in treating RAS mutated colorectal and possibly other type of cancers,” said Dr. Kimmie Ng, MD, MPH, Director of Clinical Research, Center for Gastrointestinal Oncology at Dana Farber Cancer Institute, Harvard Medical School, and Principal Investigator for TX803 clinical trials.

About Tarrex Biopharma

Tarrex Biopharma is a privately-owned drug development company with an office in San Diego, California and a research facility in Xiamen, China. Tarrex is committed to developing novel cancer therapeutics that seek to address significant unmet medical needs in major commercial markets worldwide. In addition to the lead candidate TX803 for colorectal cancer, the company is partnered with Aranda Pharma, a Finnish drug discovery company, to develop a new generation of small molecule androgen receptor (AR) antagonist for the treatment of castration resistant prostate cancer (CRPC), targeting specifically enzalutamide resistant CRPC.

Contact:

Kanyin E. Zhang, Ph.D., CEO of Tarrex Biopharma,
kanyin.zhang@tarrexbio.com 
858.395.6918 (US) / +86 18150116193 (China)

SOURCE Tarrex Biopharma

Neurocrine Announces American Journal of Psychiatry Publication of Positive Results from Kinect 3 Phase III Study of INGREZZA TM (valbenazine) for the Treatment of Tardive Dyskinesia

March 21, 2017 – 4:59 am

– Phase III study conducted in patients with tardive dyskinesia shows significant and meaningful reduction in symptoms with INGREZZA- INGREZZA clinical development program is largest ever in tardive dyskinesia with over 1,000 persons having participated in 20 clinical trials

SAN DIEGO, March 21, 2017 /PRNewswire/ — Neurocrine Biosciences, Inc. (NASDAQ: NBIX), a biotechnology company focused on neurologic, psychiatric and endocrine related disorders, announced today that positive results from the Kinect 3 Phase III study of INGREZZA (valbenazine) for the treatment of tardive dyskinesia (TD) were published online by the American Journal of Psychiatry (DOI: 10.1176/appi.ajp.2017.16091037). Once-daily INGREZZA, a novel, selective vesicular monoamine transporter 2 (VMAT2) inhibitor, demonstrated a significant and meaningful reduction in TD symptoms compared with placebo in participants with underlying schizophrenia, schizoaffective disorder or mood disorder. INGREZZA was found to be generally well tolerated with adverse events consistent with those of prior studies.

Tardive dyskinesia is thought to affect at least 500,000 people in the U.S. and is characterized by uncontrollable, abnormal and repetitive movements of the trunk, extremities and/or face.  These symptoms are associated with chronic exposure to dopamine receptor blockers such as antipsychotic medications and can be severe, persistent and irreversible. In some cases, they can even interfere with speech, walking, swallowing and breathing.

“The unprecedented results from the Kinect 3 study demonstrate the potential of INGREZZA to fill a significant unmet need in this underserved patient population,” said Kevin C. Gorman, Chief Executive Officer of Neurocrine. “This novel, new chemical entity was developed by Neurocrine chemists and biologists who devoted themselves to identifying a treatment for those living with the disruptive impact of tardive dyskinesia.” 

“There are currently no medications indicated for the treatment of tardive dyskinesia approved by the U.S. Food and Drug Administration. A common approach to address the condition has been to discontinue antipsychotic treatment or reduce the dosage, which may often have a negative impact on the psychiatric status of individuals suffering from TD,” said Christopher F. O’Brien, Chief Medical Officer of Neurocrine. “The results of the Kinect 3 study indicate that INGREZZA significantly reduced tardive dyskinesia symptoms while maintaining stability of psychiatric status during treatment.”

Neurocrine has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for INGREZZA and has been granted Priority Review with a Prescription Drug User Fee Act (PDUFA) action date of April 11, 2017. Neurocrine received Breakthrough Therapy Designation from the FDA in 2014 for INGREZZA for the treatment of TD.

Kinect 3 Study Results

The study met its primary endpoint of change-from-baseline in the Abnormal Involuntary Movement Scale (AIMS) at week six in the 80mg once-daily dosing group compared to placebo as assessed by expert central blinded video raters. The mean change from baseline to week six in the AIMS rating was -3.2 for the 80mg once-daily group as compared to -0.1 in the placebo group (p>0.0001).

In addition, the percentage of participants who achieved an AIMS response (defined in the study as a reduction greater than or equal to 50 percent from baseline in dyskinesia score) was higher in the INGREZZA 80mg/day group compared to placebo at all study visits. At week six, 40 percent (p<0.001) of participants receiving 80mg/day of INGREZZA had at least a 50% improvement in AIMS dyskinesia score as compared to only 8.7 percent of those who received placebo.

The Kinect 3 study was a Phase III, randomized, double-blind, placebo-controlled, parallel-group, fixed-dose study in which 234 subjects with TD and underlying schizophrenia, schizoaffective disorder or mood disorder (including bipolar disorder or major depressive disorder) receive six weeks of once-daily INGREZZA (40mg or 80mg capsules) or placebo.  Subsequent to the completion of the six week placebo-controlled dosing, all subjects were placed on once-daily 40mg or once-daily 80mg of INGREZZA through week 48.

Safety Profile

During the six-week placebo-controlled treatment period INGREZZA was generally well tolerated and the most common adverse reactions were somnolence and drooling. The frequency of adverse events was similar among all treatment groups and treatment emergent adverse effects were consistent with those of prior studies.  There were no drug-drug interactions identified in subjects who were utilizing a wide range of psychotropic and other concomitant medications.

About Tardive Dyskinesia

Tardive dyskinesia is caused by treatments that block dopamine receptors in the brain, such as antipsychotics and other medications. In patients with TD, these treatments are thought to result in excessive dopamine signaling in the region of the brain that controls movement. Tardive dyskinesia is characterized by uncontrollable, abnormal and repetitive movements of the trunk, extremities and/or face. These can include hand or foot movements, rocking of the trunk, lip smacking, grimacing, tongue protrusion, facial movements or blinking, as well as puckering and pursing of the lips. Tardive dyskinesia can cause significant impairment and may lead to social withdrawal, reduced workplace productivity or loss of employment, feeling embarrassed in public, or making others feel uncomfortable. 

About INGREZZA

VMAT2 is a protein concentrated in the human brain that is primarily responsible for re-packaging and transporting monoamines (dopamine, norepinephrine, serotonin, and histamine) in pre-synaptic neurons. INGREZZA (valbenazine) capsules, developed in the Neurocrine laboratories, is a novel, selective VMAT2 inhibitor that modulates dopamine release during nerve communication, showing little or no affinity for VMAT1, other receptors, transporters and ion channels. INGREZZA is designed to provide low, sustained, plasma and brain concentrations of active drug to allow for once-daily dosing. The proprietary name INGREZZA has been conditionally accepted by the FDA.

Modulation of neuronal dopamine levels in diseases such as tardive dyskinesia, Tourette syndrome, Huntington’s chorea, schizophrenia, and tardive dystonia, which are characterized, in part, by a hyperdopaminergic state, may provide symptomatic benefits for patients with these diseases.

The Company has a pending NDA under review by the FDA to utilize INGREZZA for the treatment of tardive dyskinesia. The Company also has another ongoing placebo-controlled Phase II Tourette syndrome study evaluating INGREZZA in pediatrics, the T-Force GREEN study. Additionally, the Company has an ongoing open-label, fixed-dose rollover study of INGREZZA in up to 180 subjects with Tourette syndrome.

About Neurocrine Biosciences

Neurocrine Biosciences, Inc. discovers and develops innovative and life-changing pharmaceuticals in diseases with high unmet medical needs through its novel R&D platform, focused on neurologic, psychiatric and endocrine based diseases and disorders. The Company’s three late-stage clinical programs are: INGREZZA, a VMAT2 inhibitor for the treatment of movement disorders; elagolix, a gonadotropin-releasing hormone antagonist for women’s health that is partnered with AbbVie Inc.; and opicapone, a novel, once-daily, peripherally-acting, highly-selective catechol-o-methyltransferase inhibitor under investigation as adjunct therapy to levodopa in Parkinson’s patients. Neurocrine plans to commercialize INGREZZA in the United States upon approval by the FDA.

Neurocrine Biosciences, Inc. news releases are available through the Company’s website via the internet at http://www.neurocrine.com.

Forward Looking Statements

In addition to historical facts, this press release contains 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 INGREZZA development for both tardive dyskinesia and/or Tourette syndrome. Specifically, the risks and uncertainties the Company faces include risks that the INGREZZA NDA may not obtain regulatory approval from the FDA for tardive dyskinesia or such approval may be delayed or conditioned; risks that additional regulatory submissions, for Tourette syndrome or otherwise, may not occur or be submitted in a timely manner; risks that the FDA or regulatory authorities outside the U.S. may make adverse decisions regarding INGREZZA; risks that INGREZZA development activities may not be completed on time or at all; risks that ongoing INGREZZA development activities may be delayed for regulatory or other reasons; risks that ongoing or future INGREZZA clinical trials may not be successful or replicate previous clinical trial results, may fail to demonstrate that INGREZZA is safe, tolerable or effective, or may not be predictive of real-world results or of results in subsequent clinical trials; risks that INGREZZA may be precluded from commercialization by the proprietary rights of third parties, or have unintended side effects, adverse reactions or incidents of misuse; risks associated with the Company’s dependence on third parties for development and manufacturing activities related to INGREZZA; risks that the Company will be unable to raise additional funding, if required, to complete development of or commercialize INGREZZA; risks and uncertainties relating to competitive products and technological changes that may limit demand for INGREZZA; risks that the Company may not successfully commercialize INGREZZA; and other risks described in the Company’s annual report on Form 10-K for the year ended December 31, 2016. Neurocrine disclaims any obligation to update the statements contained in this press release after the date hereof.

 

SOURCE Neurocrine Biosciences, Inc.

Rosa & Co. Celebrates 15th Anniversary, Launches New Website

March 21, 2017 – 4:35 am

SAN CARLOS, Calif., March 21, 2017 /PRNewswire/ — Rosa and Co. LLC, founded in January 2002, celebrates its 15-year milestone as an employee-owned drug-development advisory firm. In its first 15 years, Rosa has grown to become the worldwide commercial leader in PhysioPD™ Research and helped researchers to make more confident decisions at all stages in drug research and development by elucidating the mechanistic connection between direct drug effect and relevant preclinical and clinical outcomes.

“I am humbled by the opportunity we have had over these past 15 years to collaborate with industry leaders worldwide and proud of the positive impact that our expertise has had on their product development pipelines. I am especially grateful that our successes have allowed Rosa to grow and thrive, especially as a privately held company. As we look to the future, we will continue to build upon the foundation of our basic philosophy: to be a science-focused and client-friendly firm that provides scientific insight that is otherwise very difficult and expensive to achieve.”

Ron Beaver, PhD
Founder, Chairman, and CEO        

As part of it’s anniversary celebration, Rosa has launched a new website (www.rosaandco.com) to better reflect its industry-leading scientific capabilities and support the development of the broader PhysioPD-style modeling community.

Over the last 15 years, Rosa has expanded its comprehensive basic biology and therapeutic knowledge base, developed industry-leading modeling capabilities, and engaged clients that include most of the top-20 global pharmaceutical firms and dozens of emerging biotechnology and pharmaceutical firms. Expert scientific knowledge and unique modeling capability have been key in retaining satisfied clients, and Rosa’s staff bring more than a century of aggregate experience applying PhysioPD Research to the inherent challenges faced by the drug development industry.

About Rosa

Rosa supports clients with their critical decisions – from preclinical through clinical development – by creating and using customized PhysioPD Research Platforms that connect mechanisms to outcomes and that are used to research and simulate normal and disease physiology, drug action, patient variability, and trial outcomes. Rosa’s clients are involved in Platform creation, testing, and biological simulation research. Clients retain the Platforms as a program asset to support decisions and future research. Rosa’s staff has unparalleled professional experience applying biological modeling and simulation research to accelerate drug development and has over 15 years of client engagements in virtually all therapeutic areas.

Rosa is unique in its breadth and depth of disease area experience, including metabolic diseases, immuno-oncology, oncology, cardiovascular, inflammation, immune dysfunction, central nervous system disorders, dermatology, and antibacterial/antiviral biology. For more information, visit http://www.rosaandco.com.

Rosa and the Rosa logo are trademarks of Rosa & Co. LLC.

Media Contact:
Rebecca Baillie
530-383-2845
150859@email4pr.com

SOURCE Rosa & Co. LLC

Lin BioScience Licenses Novel Therapeutic Program for Brain Cancer from the University of Sydney

March 21, 2017 – 4:00 am

LBS-002’s ability to cross the blood-brain barrier shows promise for treating both primary and metastatic brain cancers

SAN DIEGO, March 21, 2017 /PRNewswire/ — Lin BioScience, a drug development company specializing in innovative therapies for oncology, ophthalmology, and cardiology, today announced an exclusive licensing agreement with the University of Sydney for a microtubule-targeting agent to treat brain cancers.

The therapeutic candidate, LBS-002, is a small molecule that disrupts the division of cancer cells by preventing the formation of microtubules (the cell structures responsible for the segregation of chromosomes in cell division).

“To date, treating primary glioblastomas and metastatic brain cancers has been challenging because the blood-brain barrier prevents effective drugs from reaching their targets,” stated Dr. Tom Lin, CEO of Lin Bioscience. “LBS-002’s ability to cross the blood-brain barrier enables it to overcome the permeability limitations experienced by traditional treatments, making it a promising therapeutic candidate. We’re excited to work with the expertise of Dr. Munoz, Dr. Kassiou and their teams at the University of Sydney to bring new cancer therapies to the clinic.”

Under the terms of the agreement, Lin BioScience will gain exclusive global rights to the development and commercialization of intellectual property developed at University of Sydney by Drs. Lenka Munoz and Michael Kassiou. Further terms of the agreement are not disclosed.

“Our research has passed the discovery stage and now our goal is to progress into clinical testing,” stated Drs. Munoz and Kassiou. “LBS-002 provides an exciting basis for a program that will lead to new treatments for brain tumors and we are very excited to be working with an innovative biotech like Lin BioScience in translating our fundamental research into clinical studies,” said Drs. Munoz and Kassiou.

About Lin BioScience

Lin BioScience is a drug development company specializing in innovative therapies for diseases with unmet medical needs. Lin BioScience’s diverse and novel pipeline consists of first-in-class drugs aimed at treating life-threatening or disabling diseases in oncology, ophthalmology, and cardiology.

 

SOURCE Lin Bioscience Inc.

Phase 1/2 Clinical Trial of Apexigen’s APX005M in Combination with Pembrolizumab (Keytruda®) Opens for Metastatic Melanoma Patients

March 21, 2017 – 3:00 am

SAN CARLOS, Calif., March 21, 2017 /PRNewswire/ — Apexigen, Inc., a clinical-stage biopharmaceutical company focusing on discovering and developing antibody-based therapeutics for the treatment of cancer with an emphasis on new Immuno-Oncology (I-O) agents, today announced the initiation of patient enrollment in a Phase 1/2 clinical trial at The University of Texas MD Anderson Cancer Center.  This trial is designed to evaluate Apexigen’s CD40 agonistic antibody APX005M in combination with pembrolizumab (Keytruda®) in patients with metastatic melanoma.

“APX005M has demonstrated robust immune system activation and an excellent safety profile and we are eager to explore the therapeutic benefit of combining our antibody with pembrolizumab for patients living with melanoma,” said Ovid Trifan, M.D., Ph. D., Chief Medical Officer at Apexigen. “We expect that APX005M’s anti-tumor immune response through antigen-presenting cells will become a cornerstone of many new therapeutic I-O combinations that can help treat cancer patients in the coming years.”

“The field of immuno-oncology is making significant advances in the treatment of cancer patients. We are excited that APX005M may play a pivotal role in driving even more advances in I-O treatment to benefit cancer patients,” said Xiaodong Yang, M.D., Ph.D., President and Chief Executive Officer of Apexigen.

This Phase 1/2 clinical trial plans to enroll patients with metastatic melanoma and will evaluate the safety, tolerability and preliminary efficacy of APX005M, administered locally into the tumor, combined with systemic administration of pembrolizumab. During the initial dose-escalation part of this trial, the maximum tolerated dose of APX005M in combination with pembrolizumab will be determined.  In the subsequent dose-expansion part of this trial, the primary endpoint will measure overall response rate for patients using the immune-related Response Criteria (irRC) criteria and the secondary endpoint will assess the immune-related best overall response.  Additional information on this clinical trial is available at www.clinicaltrials.gov (identifier: NCT02706353).

About APX005M
APX005M is a novel, humanized monoclonal antibody that stimulates the anti-tumor immune response in the tumor microenvironment through antigen-presenting cells (APC) of the immune system. APC activation complements the action of immune checkpoint inhibitors, such as pembrolizumab, a PD-1 inhibitor, which releases T cells from immunosuppression. In a recently completed clinical trial, APX005M demonstrated robust immune activation and an excellent safety profile. Preclinical data strongly suggest that combination of a CD40 agonistic antibody with an immune checkpoint inhibitor, such as an anti-PD-1, anti-PD-L1 and/or anti-CTLA4 antibody, will result in synergistic anti-tumor efficacy.

About Metastatic Melanoma
According to the American Cancer Society’s estimates, approximately 87,110 individuals will be diagnosed with melanoma and 9,730 people will die from this form of cancer in 2017 in the United States. Patients diagnosed with metastatic melanoma, or Stage IV melanoma, have a 5-year survival rate of between 15% and 20%.

About Apexigen, Inc.
Apexigen is a clinical-stage biopharmaceutical company discovering and developing a new generation of antibody therapeutics for oncology, with an emphasis on new immuno-oncology agents that could harness the patient’s immune system to combat and eradicate cancer. APX005M and the Company’s additional preclinical programs were discovered using APXiMABTM, Apexigen’s proprietary product discovery platform. This platform has enabled the Company and its collaboration partners to discover and develop high-quality therapeutic antibodies against a variety of molecular targets, including targets that are difficult to drug with conventional antibody technologies. Seven product candidates discovered using APXiMABTM are currently in clinical development, either internally by Apexigen or by its partners. For more information, please visit www.apexigen.com.

Apexigen Contact:
Mark Nevins
Vice President, Business Development
650-931-6236
mnevins@apexigen.com

 

SOURCE Apexigen, Inc.