On May 9, 2016 Neuralstem, Inc. (Nasdaq: CUR), a biopharmaceutical company focused on the development of central nervous system therapies based on its neural stem cell technology, reported its financial results for the three months ended March 31, 2016 and provided a business update (Press release, Neuralstem, MAY 9, 2016, View Source [SID:1234512122]).

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Business Highlights
On May 6, 2016, the Company closed a public offering resulting in net proceeds of $7.42 million. The net proceeds will be used for general corporate purposes, including the Phase 2 NSI-189 major depressive disorder (MDD) clinical trial and ongoing research and development activities.

In March 2016, we commenced the NSI-189 Phase 2 clinical trial for the treatment of Major Depressive Disorder (MDD).
In February 2016, we strengthened our management team with the appointment of Richard Daly as our President and Chief Executive Officer.

In January 2016, we announced an initiative to pursue collaborations for our stem cell therapy programs in order to utilize additional expertise, expedite clinical and regulatory pathways and secure alternative funding.

"The Company’s ability to raise significant capital from institutional investors provides us a cash runway to continue to fund our clinical development programs, specifically the NSI-189 Phase 2 MDD clinical trial," said Richard Daly, CEO. "We recently commenced our Phase 2 MDD trial which confirms the Company is on track to have results in the second half of 2017. We are committed to continue to execute our clinical and corporate strategy to create additional stakeholder value."

Small Molecule Pharmaceutical Compounds Clinical Development
Lead asset, NSI-189 Phase 2 clinical trial for the treatment of major depressive disorder (MDD)
In March 2016, we commenced our NSI-189 Phase 2 clinical trial for the treatment of MDD, a double-blind, randomized, placebo-controlled, 220 subject study. For information on the trial please visit View Source

Cell Therapy Platform Clinical Developments
In January 2016, Karl Johe, Founder and Chief Scientific Officer, presented at the Phacilitate Cell & Gene Therapy World Conference. He concluded that the collective trial data analysis showed that our proprietary neural stems cells consistently demonstrated biological activity in all three program indications: amyotrophic lateral sclerosis (ALS), chronic spinal cord injury (cSCI), and motor deficits due to ischemic stroke.

NSI-566 Phase 1 safety trial for the treatment of cSCI
In January 2016, the Company reported preliminary six-month follow-up Phase 1 safety data on all four subjects in the chronic spinal cord injury trial. The stem cell treatment demonstrated feasibility and safety. A self-reported ability to contract some muscles below the level of injury was confirmed via clinical and electrophysiological follow-up examinations in one of the four patients treated. This study was completed with the collaboration of the UCSD School of Medicine, supported by the UCSD Sanford Stem Cell Clinical Center; substantially all of the clinical costs of this study have been funded by grants arranged through the University.

NSI-566 Phase 1 and 2 safety trials for the treatment of amyotrophic lateral sclerosis (ALS)
In September 2015, nine-month Phase 2 and combined Phase 1 and Phase 2 data on the NSI-566 trial in amyotrophic lateral sclerosis (ALS) was presented at the American Neurological Association Annual Meeting by the principal investigator, Eva Feldman, MD, PhD, Director of the A. Alfred Taubman Medical Research Institute and Director of Research of the ALS Clinic at the University of Michigan Health. The data showed that the intraspinal transplantation of the cells was safe and well tolerated.

In January 2016, the Company announced that it is in discussions with various governmental, state and non-profit organizations regarding funding grants for the next trial. Initiation of the trial will be dependent upon significant funding from such sources.

NSI-566 Phase 1 safety trial for the treatment of motor deficits in stroke
During the three months ended March 31, 2016, the company completed dosing the third of three planned cohorts, each cohort included three patients, for a total of nine patients in a Phase 1 open label, dose-escalation trial evaluating safety and the maximum tolerated dose. The trial is being conducted by Neuralstem China, at BaYi Brain Hospital in Beijing, China.

Results of Operations for the Quarter Ended March 31, 2016:
Cash, cash equivalents and short-term investments on hand was approximately $7.6 million at March 31, 2015, compared to approximately $12.2 million at December 31, 2015. The decrease was primarily due to our ongoing operating expenses primarily related to preparations for the initiation of our NSI-189 Phase 2 clinical trial for the treatment of MDD.

On May 06, 2016, we closed a public offering of 20,000,000 shares of common stock and 20,000,000 common stock purchase warrants at a public offering price of $0.40 per each share and common stock purchase warrant. We received aggregate gross proceeds of $8.0 million and net proceeds of approximately $7,420,000 from the offering. Based upon our cash at March 31, 2016, and the proceeds from our May public offering, we expect to be able to fund our operations through December 31, 2016.

In the quarter ended March 31, 2016, we reported a net loss of approximately $6.6 million or $0.07 per share, compared to a loss of approximately $5.1 million or $0.06 per share in the first quarter of 2015. Our operating loss in the quarter ended March 31, 2016 was approximately $6.2 million compared to a loss of $4.6 million in the same quarter of 2015. The increase in operating loss was primarily attributable to the severance accrual and acceleration of stock based compensation expense related to the departure of our previous CEO. A decrease of approximately $0.1 million in research and development expense was offset by an increase of approximately $1.7 million in general and administrative expenses.

Research and development expenses decreased approximately $117,000 or 4% for the period ended March 31, 2016 compared to the comparable period of 2015 primarily as a result of a decrease in pre-clinical and clinical costs partially offset by an increase in headcount and stock based compensation.

General and administrative expenses increased approximately $1,737,000 or 121% for the period ended March 31, 2016 compared to the comparable period of 2015 primarily due to a severance accrual and increased stock based compensation resulting from the accelerated vesting of options, both related to the termination of our former Chief Executive Officer.

In addition, in the first quarter of 2016 we recognized approximately $0.4 million of other expenses primarily comprised of interest expenses related to our long-term debt.
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Neuralstem, Inc.

Unaudited Condensed Consolidated Balance Sheets

March 31,
2016

December 31,
2015

ASSETS

CURRENT ASSETS

Cash and cash equivalents

$
7,620,746

$
4,716,533

Short-term investments

–

7,517,453

Trade and other receivables

39,167

37,316

Prepaid expenses

1,077,762

1,159,782

Total current assets

8,737,675

13,431,084

Property and equipment, net

336,974

343,200

Patents, net

1,066,577

1,103,467

Other assets

57,692

71,797

Total assets

$
10,198,918

$
14,949,548

LIABILITIES AND STOCKHOLDERS’ (DEFICIT) EQUITY

CURRENT LIABILITIES

Accounts payable and accrued expenses

$
2,627,137

$
1,455,826

Accrued bonuses

287,046

161,362

Current portion of long term debt, net of fees and discount

4,466,081

4,634,742

Other current liabilities

241,008

173,542

Total current liabilities

7,621,272

6,425,472

Long term debt, net of fees, discount and current portion

2,546,296

3,391,808

Other long term liabilities

200,739

164,990

Total liabilities

10,368,307

9,982,270

STOCKHOLDERS’ EQUITY (DEFICIT)

Preferred stock, 7,000,000 shares authorized, zero shares issued and outstanding

–

–

Common stock, $0.01 par value; 300 million shares authorized, 92,044,042 and 92,005,705 shares outstanding in 2016 and 2015, respectively

920,440

920,057

Additional paid-in capital

177,473,335

176,002,832

Accumulated other comprehensive income

1,298

3,071

Accumulated deficit

(178,564,462)

(171,958,682)

Total stockholders’ equity (deficit)

(169,389)

4,967,278

Total liabilities and stockholders’ equity (deficit)

$
10,198,918

$
14,949,548

Neuralstem, Inc.

Unaudited Condensed Consolidated Statements of Operations and Comprehensive Loss

Three Months Ended March 31,

2016

2015

Revenues

$
2,500

$
2,917

Operating expenses:

Research and development expenses

3,065,590

3,182,823

General and administrative expenses

3,170,522

1,433,074

Total operating expenses

6,236,112

4,615,897

Operating loss

(6,233,612)

(4,612,980)

Other income (expense):

Interest income

11,136

13,569

Interest expense

(386,506)

(453,734)

Other income

3,199

–

Total other income (expense)

(372,171)

(440,165)

Net loss

$
(6,605,783)

$
(5,053,145)

Net loss per share – basic and diluted

$
(0.07)

$
(0.06)

Weighted average common shares outstanding – basic and diluted

92,009,782

89,654,634

Comprehensive loss:

Net loss

$
(6,605,783)

$
(5,053,145)

Foreign currency translation adjustment

(1,773)

13

Comprehensive loss

$
(6,607,556)

$
(5,053,132)
– See more at: View Source#sthash.mDCb8Mlo.dpuf

On May 9, 2016 Kite Pharma, Inc. (Nasdaq: KITE), a clinical-stage biopharmaceutical company focused on developing engineered autologous cell therapy (eACT) products for the treatment of cancer, reported a corporate update and reported first quarter 2016 financial results for the period ended March 31, 2016 (Press release, Kite Pharma, MAY 9, 2016, View Source [SID:1234512151]).

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"Our ZUMA-1 update at AACR (Free AACR Whitepaper) last month highlighted the potential of KTE-C19, our breakthrough immunotherapy candidate, to put patients with chemorefractory aggressive non-Hodgkin lymphoma (NHL), a patient population burdened with a short life expectancy and limited treatment options, into a durable complete remission," noted Arie Belldegrun, M.D., FACS, Chairman, President, and Chief Executive Officer. "We remain on track to provide interim data from the pivotal phase 2 portion of ZUMA-1 later this year and plan to submit the KTE-C19 registration filing to the U.S. Food and Drug Administration (FDA) by the end of 2016."

"Our commitment to delivering a breakthrough personalized cell therapy to cancer patients now extends fully to our manufacturing and market planning areas. Validation and qualification activities at our state-of-the-art commercial manufacturing facility are underway. The commercial team is actively evaluating a broad range of market and payor strategies for making KTE-C19 available to patients with a significant unmet need."

First Quarter 2016 and Recent Highlights

At AACR (Free AACR Whitepaper), reported rapid and durable responses in patients with chemorefractory diffuse large B cell lymphoma treated with KTE-C19 in the phase 1 portion of ZUMA-1.
Ongoing complete responses were observed in 3 of 7 patients at 9-month study follow-up (1 patient) and 6-month study follow-up (2 patients).
KTE-C19-related adverse events consisted predominantly of cytokine release syndrome and neurotoxicity, which were generally reversible.
Partnered with Genentech to study KTE-C19 in combination with the checkpoint inhibitor atezolizumab. Kite expects to initiate a Phase 1b/2 combination study in patients with chemorefractory diffuse large B cell lymphoma in the second half of 2016.
Expanded our clinical and research partnership with the National Cancer Institute (NCI) by entering into a new Cooperative Research and Development Agreement (CRADA) with James (Jim) N. Kochenderfer, M.D., and the NCI’s Experimental Transplantation and Immunology Branch.
Phase 1 study of human anti-CD19 chimeric antigen receptor for treating B-cell malignancies currently ongoing.
Also at AACR (Free AACR Whitepaper), William Lu, Ph.D., a collaborator of Kite’s at the NCI, reported data from a Phase 1 study of a T cell receptor (TCR) product candidate targeting MAGE-A3 that was advanced under Kite’s CRADA with the Surgery Branch at the NCI.
Data reported at AACR (Free AACR Whitepaper) support Kite’s plan to file later this year an investigational new drug (IND) application for a TCR product candidate that targets a MAGE-A3 antigen expressed on solid tumors.
Entered into a research and license agreement with Leiden University Medical Center in the Netherlands to identify and develop additional TCR product candidates targeting solid tumors that are associated with the human papillomavirus (HPV) type 16 infection.
Appointed Tim Moore, a biopharma executive with more than 30 years of global operations experience, as Executive Vice President, Technical Operations, to lead product development, manufacturing, supply chain, quality assurance, and end-to-end process optimization for all aspects of Kite’s engineered T cell product candidates.
Augmented Kite’s commercial function, under the leadership of Chief Commercial Officer Shawn Tomasello, with the appointment of an integrated executive team responsible for all aspects of commercial and medical affairs strategy, planning, and analysis for the potential launch of KTE-C19.
First Quarter 2016 Financial Results

Revenue was $5.1 million for the first quarter of 2016.
Research and development expenses were $34.4 million for the first quarter of 2016, and include $8.5 million of non-cash stock-based compensation expense.
General and administrative expenses were $16.5 million for the first quarter of 2016, and include $6.4 million of non-cash stock-based compensation expense.
Net loss was $43.9 million, or $0.90 per share, for the first quarter of 2016.
Non-GAAP net loss for the first quarter of 2016 was $29.1 million, or $0.60 per share, which excludes non-cash stock-based compensation of $14.9 million.
As of March 31, 2016, Kite had $577.4 million in cash, cash equivalents, and marketable securities.
Kite continues to expect the full year 2016 net cash burn to be $235 to $250 million dollars, which includes approximately $20 million in capital expenditures, but excludes any inflows or outflows from business development activities. The estimated full year 2016 net cash burn is primarily driven by an estimated net loss of $295 to $310 million, which includes an estimated $80 million of non-cash stock-based compensation expense.

Promedior’s drug candidates are based on Pentraxin-2, an endogenous human protein that is specifically active at the site of tissue damage and works as an agonist to initiate a resolution process for prevention and potential reversal of fibrosis (Company Pipeline, Promedior, MAY 9, 2016, View Source [SID:1234512083])). Promedior’s Pentraxin-2 therapeutics harness the innate healing power of the immune system, acting as a master regulator upstream in the fibrosis cascade.

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Normal wound healing consists of 3 distinct pathways: (1) inflammation, (2) proliferation, and (3) resolution. Fibrosis occurs when the normal wound healing response gets locked in the proliferation pathway, resulting in a cascade of excessive scar tissue formation that leads to tissue damage and organ dysfunction.

Pentraxin-2 directs the immune system to turn on the resolution pathway and simultaneously turn off the proliferation pathway, and works specifically in areas of tissue injury.

Pentraxin-2-based therapeutics have several advantages over other experimental approaches to treating fibrotic diseases:
· Potent Upstream Agonist: Fibrosis, the formation of dysregulated scar tissue, is a highly conserved process with many downstream redundancies in the pathway. By turning on a resolution pathway and thereby turning off the proliferation pathway upstream of significant redundancies, Pentraxin-2-based therapeutics offer the potential for a more robust approach to efficacy. In contrast, most competitive approaches target single downstream enzymes or cytokine targets, making it difficult to achieve efficacy without combination therapy.
· Specificity: Pentraxin-2’s natural mechanism of action involves specificity that targets activity to the damaged tissue microenvironment, ensuring that the drug effect occurs in areas of disease. Less specific approaches could lead to unintended side-effects and toxicity.
· Potential to Reverse Fibrosis and Recover Function: By simultaneously promoting resolution and turning off the proliferation pathway, Pentraxin-2-based therapeutics offer the potential to break down scar tissue and promote recovery of organ function. Preclinical results have demonstrated the ability to reverse fibrosis. Such reversal of fibrosis is possible due to the promotion of factors in the resolution pathway such as enzymes that break down the extracellular matrix that comprise the scar tissue in fibrotic tissue.
Extensive studies conducted by Promedior and its collaborators have demonstrated the ability of Pentraxin-2 to act as an upstream agonist that is specific for the damaged tissue microenvironment across many major tissue types and in several models of fibrotic disease, strongly supporting its potential as a novel anti-fibrotic agent. Promedior and its collaborators have published many of their findings in peer-reviewed journals and presented them at medical and scientific meetings.

On May 09, 2016 Oncothyreon Inc. (NASDAQ:ONTY), a clinical-stage biopharmaceutical company, reported a corporate update and reported financial results for the quarter ended March 31, 2016 (Press release, Oncothyreon, MAY 9, 2016, View Source [SID:1234512124]).

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"During the first quarter, the Company continued to advance its portfolio of oncology product candidates and achieved a major milestone with the commencement of the Phase 2 combination trial of ONT-380 with Herceptin and Xeloda in patients with HER2-positive, third-line metastatic breast cancer," said Scott Myers, President and CEO of Oncothyreon.

"In my short tenure so far, it is already clear the Company is committed to making an impact on the lives and outcomes of cancer patients, and I look forward to building upon that foundation," continued Mr. Myers. "In the coming months, the Company plans to review all programs and implement a development strategy for the optimal allocation of resources, with the goal of delivering novel therapies to cancer patients."

First Quarter and Recent Highlights:

Clinical Development:

ONT-380 Phase 2 Combination Trial Underway in Patients with HER2-Positive Breast Cancer. This randomized, double-blind, placebo-controlled trial is evaluating ONT-380 in combination with Herceptin (trastuzumab) and Xeloda (capecitabine). ONT-380 is an oral, HER2-selective, central nervous system (CNS)-active tyrosine kinase inhibitor. The Phase 2 trial is targeted to enroll approximately 180 heavily pretreated patients with advanced HER2-positive breast cancer who present with or without brain metastases. Building on encouraging Phase 1b results, the primary and secondary endpoint objectives are designed to measure ONT-380’s contribution on both systemic and CNS disease, an area of unmet need for patients.

Presentation of data highlighting the preclinical development of orally bioavailable, potent and selective checkpoint kinase 1 (Chk1) inhibitors. Results presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2016 highlighted preclinical data demonstrating that select Oncothyreon Chk1 inhibitors display cellular potency against Chk1, are active against a diverse range of cancer cell lines and demonstrate synergistic activity in combination with certain chemotherapeutic drugs. The pharmaceutical properties also indicate good metabolic stability and pharmacokinetic properties, with good oral bioavailability in preclinical models. The research was conducted pursuant to a research collaboration agreement with Sentinel Oncology.
Corporate Update:

Appointment of Scott Myers, President and CEO and member of the Board of Directors.
Scott Myers joined Oncothyreon on April 4, 2016. Mr. Myers most recently served as Chief Executive Officer and President for Aerocrine AB, a life sciences company based in Stockholm, Sweden and Morrisville, NC. Aerocrine was acquired by Circassia Pharmaceuticals, a UK-based immunotherapy company, in July of 2015. Mr. Myers also served as an independent Director for Orexo, AB, a pharmaceutical company and is currently an industry advisor to EQT, a large Scandinavian investment fund. Prior to joining Aerocrine in 2011, he served as Vice President, Head of European Mid-Markets for UCB Pharma, SA, a Belgian-based, global biopharmaceutical company. In this role, Mr. Myers oversaw commercial operations and medical affairs for 32 countries in the EU. Prior to UCB, he served in senior roles responsible for business development, strategic marketing and pharmaceutical portfolio management at several leading companies including Johnson & Johnson.

New board members appointed. In January 2016, Oncothyreon appointed new board members Mark Lampert and Gwen Fyfe, M.D. Mr. Lampert brings extensive biotechnology and finance experience to the board. He is the founder of BVF Partners L.P., affiliates of which, including Biotechnology Value Fund, L.P., have been stockholders of Oncothyreon since 2011. Dr. Fyfe previously served as vice president of Genentech’s oncology development group and later as vice president, Avastin Franchise Team at Genentech.
First Quarter 2016 Financial Highlights

Cash, cash equivalents and investments totaled $46.5 million as of March 31, 2016, compared to $56.4 million at December 31, 2015, a decrease of $9.9 million, or 17.6%. The decrease was primarily attributable to cash used to fund operations of $9.8 million.

Net loss for the quarter ended March 31, 2016 was $12.9 million, or $0.14 per basic and diluted share, compared with a net loss of $7.9 million, or $0.08 per basic and diluted share, for the comparable period in 2015. The increase in net loss for the quarter was primarily attributable to increases in general and administrative expenses of $4.3 million primarily related to the retirement and separation agreement that Oncothyreon entered into with its former CEO in January 2016 and increases in research and development expenses of $0.5 million primarily due to greater activity related to the development of the product candidates.
Financial Guidance

Oncothyreon believes the following financial guidance to be correct as of the date provided. Oncothyreon is providing this guidance as a convenience to investors and assumes no obligation to update it.

Oncothyreon currently expects operating expenses in 2016 to be higher than in 2015. This increase will primarily be related to expenditures associated with the Phase 2 trial of ONT-380. Oncothyreon currently expects cash used in operations in 2016 to be approximately $38.0 million to $40.0 million. With cash, cash equivalents and investments of $46.5 million as of March 31, 2016, Oncothyreon estimates that its cash, cash-equivalents and investments will be sufficient to fund operations for at least the next 12 months.

Upcoming Events

ONT-380 Poster Presentation at ASCO (Free ASCO Whitepaper) Annual Meeting:

Abstract 513: "Efficacy results of a phase 1b study of ONT-380, a CNS-penetrant TKI, in combination with T-DM1 in HER2+ metastatic breast cancer (MBC), including patients (pts) with brain metastases." Virginia F. Borges, MD from the University of Colorado Cancer center will discuss the poster at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting in Chicago on Sunday, June 5, 2016, from 11:30 a.m. – 12:45 p.m. CT in Hall D2, with a poster session preceding from 8 a.m. to 11:30 a.m. CT in Hall A.
R & D Day:

Oncothyreon will host an R & D Day in New York City on June 14, 2016. Company management will discuss an overview of the company’s lead product candidate, ONT-380. Several distinguished oncology thought leaders will also participate. This event will be webcast.

On May 9, 2016 Novocure (NASDAQ: NVCR), a commercial stage oncology company pioneering a novel therapy for solid tumors, reported financial results for the first quarter ended March 31, 2016, highlighting strong growth in prescriptions, active patients and reported revenues (Press release, NovoCure, MAY 9, 2016, View Source [SID:1234512153]).

First quarter 2016 highlights include:
Three months ended March 31,
2016 2015 % change
Non-financial
Prescriptions received in period(1) 755 437 73 %
Active patients at period end(2) 797 372 114 %

Financial, in millions (unaudited)

Revenues(3) $ 13.1 $ 5.2 151 %

Net loss $ (35.4 ) $ (23.3 )

Cash and cash equivalents at the end of period $ 115.9 $ 74.2
Short-term investments at the end of period $ 119.8 $ 22.0

(1) A "prescription received" is a commercial order for Optune that is received from a physician certified to treat patients with Tumor Treating Fields (TTFields) therapy for a patient not previously on TTFields therapy. Orders to renew or extend treatment are not included in this total. In the future, Novocure may have regulatory approvals and commercial programs for multiple clinical indications, at which time Novocure will recognize a commercial order as a prescription for the same patient for each clinical indication treated. For example, in the future, a patient may have a prescription for the treatment of lung cancer and a prescription for the treatment of brain metastases from the lung cancer.
(2) An "active patient" is a patient who is on TTFields therapy under a commercial prescription order as of the measurement date, including patients who may be on a temporary break from treatment and who plan to resume treatment in less than 60 days.
(3) For the reported periods, all revenues were recognized when cash was collected and all other revenue recognition criteria had been met.

"I am pleased to see commercial momentum building following the October 2015 FDA approval of Optune for the treatment of newly diagnosed GBM and the December 2015 publication of EF-14 phase 3 pivotal trial results in the Journal of the American Medical Association," commented Asaf Danziger, Chief Executive Officer. "Our sales and marketing teams are intensely focused on the education of oncologists as we introduce a completely new treatment modality to the market. I am extremely proud of the hard-earned progress we have made in the two quarters since approval." Mr. Danziger continued, "I believe that we are well positioned to deliver substantial year-over-year growth in 2016. We expect the incorporation of Optune for newly diagnosed GBM into treatment guidelines, the launch, pending FDA approval, of our second generation Optune system in the United States, and the continued expansion of our U.S. and German sales forces to provide additional catalysts to support our commercial execution. We are committed to bringing Optune to all GBM patients who may benefit from it." Wilco Groenhuysen, Chief Financial Officer, stated, "2015 started a transformational phase for Novocure with the October FDA approval of Optune for newly diagnosed GBM. I am pleased to report triple digit growth in both active patients and revenues in the first quarter of 2016 compared to the same period in 2015 and believe we will continue to see significant year-over-year growth in operating statistics and revenues throughout 2016." "As our commercial teams focus on execution within the glioblastoma indication, our R&D team continues to make progress toward developing TTFields for a variety of additional solid tumors," added William Doyle, Chairman. "We have five ongoing or completed phase 2 pilot trials in brain metastases, non-small cell lung cancer, ovarian cancer, pancreatic cancer and mesothelioma. We plan to open phase 3 pivotal trials in brain metastases and non-small cell lung cancer in 2016. In addition to our clinical development activities, our engineering team continues its work to improve all aspects of TTFields delivery to enhance ease-of-use for patients." 2016 First Quarter Operating Statistics and Financial Update Prescriptions in the quarter ended March 31, 2016, increased by 318 prescriptions, or 73 percent, compared to the same period in 2015. The increase in prescriptions was driven primarily by commercial activities in the United States after the October 2015 U.S. Food and Drug Administration (FDA) approval of Optune for the treatment of newly diagnosed glioblastoma (GBM), increased commercial activities in Germany, and enhanced awareness of Optune following the December 2015 publication of EF-14 phase 3 pivotal trial results in the Journal of the American Medical Association.
In the United States, 684 prescriptions were received in the quarter ended March 31, 2016, an increase of 273 prescriptions, or 66 percent, compared to the same period in 2015.
In Germany and other EMEA markets, 71 prescriptions were received in the quarter ended March 31, 2016, an increase of 45 prescriptions, or 173 percent, compared to the same period in 2015.
The conversion of prescriptions to new patients is driven by the prescription fill rate and the time to fill the prescription. The prescription fill rate for the 12 months ended March 31, 2016, was 75 percent. The increase or decrease in active patients in any given period reflects the number of new patients less the number of patients discontinuing therapy. The rate of patients discontinuing therapy is determined by the treatment duration for patients starting therapy in prior periods. Novocure expects average treatment duration of its total active patient population to increase as the ratio of newly diagnosed GBM to recurrent GBM active patients increases. The portion of Optune prescriptions for newly diagnosed GBM was more than 50 percent in the first quarter of 2016. There were 797 active patients on Optune therapy at March 31, 2016, an increase of 425 active patients, or 114 percent, compared to March 31, 2015. The increase in active patients was driven both by prescription growth and by an increase in the percentage of newly diagnosed GBM patients who started Optune in prior periods.
In the United States, there were 699 active patients on Optune therapy at March 31, 2016, an increase of 352 active patients, or 101 percent, compared to March 31, 2015.
In Germany and other EMEA markets, there were 98 active patients on Optune therapy at March 31, 2016, an increase of 73 active patients, or 292 percent, compared to March 31, 2015.
For the 12 months ended March 31, 2016, the average cash payment received was more than $14,000 per charged month in the United States. The difference between billed and paid amounts consists of patient financial assistance, charitable care, discounts, disputed underpayments and indirect taxes. Generally, Novocure’s average time to collect on billed charges ranges between four and five months in the United States. The "payment amount" and "average time to collect" metrics do not include Novocure’s experience with patients covered by the Medicare fee-for-service program, as Novocure has not received material payments from the Medicare fee-for-service program, or Novocure’s experience with patients outside of the United States. For the three months ended March 31, 2016, revenues increased to $13.1 million compared to $5.2 million for the same period in 2015, representing 151 percent growth. This growth was primarily driven by increased Optune adoption. For the three months ended March 31, 2016, cost of revenues increased to $8.0 million compared to $3.9 million for the same period in 2015, representing 105 percent growth. This was primarily driven by an increase in the cost of transducer array shipments due to an increase in active Optune patients as well as increased personnel costs to establish infrastructure necessary to support an increasing volume of shipments to patients. Research, development and clinical trials expenses for the three months ended March 31, 2016, were $11.4 million compared to $9.9 million for the same period in 2015, representing growth of 15 percent. This growth was primarily due to increased personnel costs and increased expenses related to clinical education and investigator-sponsored trials, partially offset by reduced expenses related to the development of the second generation Optune system. Sales and marketing expenses for the three months ended March 31, 2016, were $13.3 million compared to $6.4 million for the same period in 2015, representing growth of 109 percent. This growth was primarily due to increased personnel costs as well as increased marketing expenses to expand commercial operations in the United States and Germany and to establish commercial operations in Switzerland and Japan. General and administrative expenses for the three months ended March 31, 2016, were $12.3 million compared to $7.0 million for the same period in 2015, representing growth of 76 percent. This growth was primarily due to increased personnel costs as well as increased expenses related to professional services and activities associated with being a public company. Personnel costs for the three months ended March 31, 2016, included $5.5 million in non-cash share-based compensation expenses, comprised of $0.1 million in cost of revenues; $0.8 million in research, development and clinical trials; $1.3 million in sales and marketing; and $3.2 million in general and administrative expenses. Total non-cash share-based compensation expenses for Q1 2015 were $1.8 million. Net losses for the three months ended March 31, 2016, were $35.4 million compared to $23.3 million for the same period in 2015. As of March 31, 2016, we had $115.9 million in cash and cash equivalents and $119.8 million in short-term investments for a total balance of $235.8 million in cash, cash equivalents and short-term investments. Other first quarter 2016 corporate achievements
Expanded coverage: Novocure expanded coverage of Optune for the treatment of newly diagnosed and/or recurrent GBM to include more than 63 million additional lives through new policies with Anthem, Cigna, Humana, Regence Blue Cross Blue Shield, Preferred One, Univera Healthcare, and Asuris Northwest Health. This brought the total number of covered lives to more than 107 million in the United States as of March 31, 2016. Subsequent to the quarter end, positive coverage polices were issued by Group Health Cooperative Washington and Idaho, and Blue Cross Blue Shield of Michigan, bringing the total number of covered lives to more than 112 million in the United States as of May 1, 2016.
Second generation Optune system European launch: All Optune patients in Europe are now on the second generation of Optune, which is less than half the weight and half the size of the original commercial system.
PANOVA phase 2 pilot data in pancreatic cancer: Results from the first cohort of the PANOVA trial were presented at ASCO (Free ASCO Whitepaper) GI suggesting TTFields therapy plus chemotherapy may be safe as first-line treatment and improve survival of patients with advanced pancreatic cancer. Progression free survival and overall survival of patients treated with TTFields combined with gemcitabine were more than double those of gemcitabine-treated historical controls. Of the evaluable tumors, 30 percent had partial response and another 30 percent had stable disease.
Recognition as a clinical cancer advance by ASCO (Free ASCO Whitepaper): Phase 3 pivotal trial results of TTFields in combination with temozolomide for the treatment of newly diagnosed GBM were selected for inclusion in the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper)’s Clinical Cancer Advances 2016: Annual Report on Progress against Cancer. The report reviews the recent top advances and emerging trends in clinical cancer research that are leading to improved cancer treatments for patients.
Anticipated milestones over the next nine months
NCCN guidelines update: With the October 2015 FDA approval of Optune for the treatment of newly diagnosed GBM, Novocure expects that Optune should be added to the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology for Central Nervous System Cancers for newly diagnosed GBM in 2016.
FDA approval for the second generation Optune system: In December 2015, Novocure filed a premarket approval (PMA) supplement application with the FDA seeking approval of the second generation of Optune for the approved indications. The company subsequently received and, in April 2016, responded to questions from the FDA on the PMA supplement application. Assuming no further FDA comments or requests for additional information, the company hopes to begin marketing the second generation Optune system in the United States in the third quarter of 2016.
PANOVA phase 2 pilot trial last patient in: Following the approval of nab-paclitaxel, a taxane-based chemotherapy, for the treatment of advanced pancreatic cancer, the PANOVA study was expanded to include 20 additional patients treated with TTFields in combination with nab-paclitaxel and gemcitabine. Novocure expects to finish enrollment of the second patient cohort in 2016 and, with an expected six months follow-up, anticipates phase 2 pilot data will be available for presentation in 2017.
INNOVATE phase 2 pilot trial last patient in: In October 2014, the INNOVATE trial opened to study TTFields together with weekly paclitaxel for patients with recurrent ovarian cancer. The trial is expected to finish enrollment in 2016 and, with an expected six months follow-up, Novocure anticipates phase 2 pilot data will be available for presentation in 2017.
METIS phase 3 pivotal trial first patient in: Based upon pre-clinical data showing TTFields can prevent metastatic seeding in vivo and the established safety and efficacy of TTFields in GBM, Novocure believes TTFields could be an effective treatment for patients with brain metastases. Novocure submitted a pre-submission package to the FDA for discussion and feedback in December 2015 and submitted a full investigational device exemption (IDE) application in April 2015. The open-label randomized study will test the effectiveness of TTFields following stereotactic radiosurgery (SRS) compared with watchful waiting alone in patients with brain metastases stemming from non-small cell lung cancer. Novocure plans to open the trial in mid-2016 subject to FDA approval of the IDE.
LUNAR phase 3 pivotal trial first patient in: Novocure has completed a phase 2 pilot trial in advanced non-small cell lung cancer and is planning a randomized phase 3 pivotal trial for the treatment of non-small cell lung cancer. Given recent preclinical research of TTFields in combination with PD-1 inhibitors, the company is currently reviewing the protocol design to possibly capitalize on the potential synergies with taxane-based chemotherapies and the potential additivity with PD-1 inhibitors. The company plans to open the trial in late 2016 subject to FDA approval of the IDE.
Japanese approval for newly diagnosed GBM: Novocure submitted a partial amendment application to the Japanese Pharmaceuticals and Medical Devices Agency for the treatment of newly diagnosed GBM patients in December 2015 and hopes to receive Japanese Ministry of Health, Labour and Welfare (MHLW) approval in late 2016. The MHLW approved the use of Optune in patients with recurrent GBM in March 2015. The company plans to wait until MHLW approval for newly diagnosed GBM before submitting an application for public reimbursement in Japan.

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