Cellular Biomedicine Group Reports First Quarter 2016 Financial Results and Provides Business Highlights

On May 9, 2016 Cellular Biomedicine Group, Inc. (Nasdaq:CBMG) ("CBMG" or the "Company"), a clinical-stage biomedicine firm engaged in the development of effective stem cell therapies for degenerative diseases and immunotherapies for cancer, reported financial results for the first quarter ended March 31, 2016 and provided business highlights (Press release, Cellular Biomedicine Group, MAY 9, 2016, View Source [SID:1234512205]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"In the first quarter of 2016, we made several key clinical and business advancements that we expect will promote long-term growth and strengthen our presence in the cancer and osteoarthritis markets," commented Tony (Bizuo) Liu, CBMG’s Chief Executive Officer. "We reported encouraging Phase IIb data from our most advanced stem cell program for autologous adipose-derived mesenchymal progenitor cell (haMPC) ReJoin therapy for Knee Osteoarthritis (KOA), and launched a Phase I clinical trial for an off-the-shelf allogeneic haMPC AlloJoinTM therapy for KOA. The closing of a $43.13 million strategic investment through April 2016 brings us closer to reaching our objectives of launching multi-site clinical trials in immuno-oncology cell therapies in China, to file INDs and launch clinical trials for AlloJoinTM therapy for KOA patients in the US and to further strengthen our pipeline. We are determined to build on the momentum from the first quarter and believe that we can accomplish meaningful progress on our 2016 operating objectives."

First Quarter 2016 Financial Performance

Cash Position: Cash and cash equivalents as of March 31, 2016 were $15.7 million compared to $14.9 million as of December 31, 2015. We had an increase in cash generated from financing activities due to a private placement financing in February 2016 for gross proceeds of approximately $5 million.
Net Cash Used in Operating Activities: Net cash used in operating activities for the first quarter of 2016 was $3.58 million, compared to $2.41 million for the same period in 2015.
Revenue: Revenues in the first quarter of 2016 were $488,491 compared to $603,390 for the same period in 2015. The revenue in first quarter 2016 is solely comprised of the hospital’s technology services related to cell therapy treatments.
G&A Expenses: General and administrative expenses for the first quarter of 2016 were $2.8 million compared to $2.7 million for the same period in 2015.
R&D Expenses: Research and development expenses for the first quarter of 2016 were $2.40 million, compared to $1.46 million for the same period a year ago. The increase was primarily attributable to the increase of the Company’s immunotherapy research and development team and expenses related to clinical trials.
Net Loss: Net loss allocable to common stock holders was $4.2 million, compared to $4.3 million for the same period in 2015.
Business & Technology Highlights of 2016 To Date

Announced encouraging 48-week clinical data from the Phase IIb trial of the Company’s ReJoin haMPC therapy for Knee Osteoarthritis (KOA), revealing increase of patient’s knee cartilage volume and relief of pain;
Launched an investigator initiated Phase I clinical trial of an off-the-shelf allogeneic adipose-derived mesenchymal progenitor cell (haMPC) AlloJoinTM therapy for KOA patients in China;
Advanced the Company’s cash position following the closing of an agreement with Wuhan Dangdai Science & Technology Industries Group Inc. to invest up to $43.13 million for 2.27 million shares of the Company’s common stock, representing a 16.2% post-money stake investment as of April 15, 2016.

PRM-151

PRM-151: Lead Product Candidate

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Promedior’s lead product, PRM-151, is a recombinant form of human pentraxin-2 protein (rhPTX-2) formulated for intravenous injection (Company Pipeline, Promedior, MAY 9, 2016, View Source [SID:1234512082]). Promedior is initially focusing the clinical development of PRM-151 on rare systemic fibrotic diseases, such as Idiopathic Pulmonary Fibrosis (IPF) and myelofibrosis. Highlights of PRM-151’s clinical development include:

· A Phase 1a clinical study in healthy subjects and IPF patients demonstrated that PRM-151 was safe and well-tolerated
· A Phase 1b randomized, double-blind, placebo-controlled, multiple ascending dose study in IPF patients demonstrated that PRM-151 was generally safe and well‐tolerated and resulted in a mean improvement in Forced Vital Capacity (FVC) at 8 weeks after dosing for only two weeks, whereas patients receiving placebo had a decline in FVC. These data were · presented at the American Thoracic Society Annual Meeting on May 22, 2013.

· A Phase 2 clinical trial to evaluate PRM-151 in patients with myelofibrosis is ongoing. This trial is a multi-center, two-stage, adaptive design study to determine the efficacy and safety of PRM-151 as a single agent or added to a stable dose of ruxolitinib in patients with Primary Myelofibrosis (PMF), Post-Polycythemia Vera MF (post-PV MF), or Post-Essential Thrombocythemia MF (post-ET MF). Data were presented at the 2014 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) and the 2014 European Hematology Association (EHA) (Free EHA Whitepaper) meetings in June. Positive preliminary data demonstrated biologic activity with improvements across clinically relevant measures, including bone marrow fibrosis, hemoglobin, platelets, spleen volume, and symptoms. Clinical data showed improvements in four independent treatment groups of myelofibrosis patients who received PRM-151 weekly or monthly, either as a single agent or in patients with no further improvements on a stable dose of ruxolitinib1. Importantly, PRM-151 demonstrated safety and tolerability both alone and in combination with ruxolitinib, with no evidence of the myelosuppression commonly observed with other treatments. This recent data in myelofibrosis demonstrates the potential of Promedior’s immuno-oncology approach in fibrotic cancers.

PRM-151 has demonstrated efficacy in multiple preclinical models of fibrotic disease, including the reduction of established pulmonary fibrosis.

Neuralstem Reports Fiscal First Quarter 2016 Results and Business Update

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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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.
View News Release Full Screen
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

Kite Pharma Reports First Quarter 2016 Financial Results

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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"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.

Pentraxin-2 Platform

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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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.