On March 14, 2016 Ignyta, Inc. (Nasdaq: RXDX), a precision oncology biotechnology company, reported company highlights and financial results for the full year ended December 31, 2015(Press release, Ignyta, MAR 14, 2016, View Source [SID:1234509529]).

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"2015 was a transformational year for Ignyta in terms of the substantial progress we made towards becoming a leading precision oncology biotechnology company focused on the development of first-in-class and best-in-class precision medicines," said Jonathan Lim, M.D., Chairman and CEO of Ignyta. "We significantly grew our pipeline of molecularly targeted therapies through our transactions with Teva and Lilly, and we made substantial strides on the pre-clinical, clinical and regulatory fronts with our development programs, including initiating a potentially registration-enabling Phase 2 clinical trial of our lead product candidate, entrectinib. Furthermore, we achieved CAP accreditation of our in-house diagnostics laboratory, bolstered our balance sheet, and strengthened our team. We look forward to continuing our efforts to develop compelling therapies for the benefit of cancer patients."

Company Highlights

Strategic Positioning

In February 2016, Ignyta announced plans to prioritize certain "core" pipeline programs that have generated the most promising data to date, and to deprioritize certain "non-core" pipeline programs that fall outside of Ignyta’s focus on molecularly targeted therapies. This strategic positioning prioritizes key research and development activities, improves efficiencies and reduces operating expenses.

These plans include:

An ongoing commitment to drive value from Ignyta’s core Rx/Dx business model by prioritizing the continued development of the company’s molecularly targeted entrectinib, taladegib, RXDX-105 and RXDX-106 programs, in conjunction with complementary Dx development and laboratory operations;

Continued activities to advance Ignyta’s immuno-oncology and cancer stem cell programs that relate to its current molecularly targeted pipeline (e.g., potential immunotherapy applications of the RXDX-105 and RXDX-106 programs, and potential molecularly targeted cancer stem cell applications of the taladegib and Spark programs); and

Cessation of all development activities relating to the company’s RXDX-107, RXDX-103 and RXDX-108 programs, and certain Spark discovery programs.

Entrectinib Clinical Progress

In September 2015, Ignyta announced the initiation of its Phase 2 clinical trial of entrectinib, the company’s proprietary oral tyrosine kinase inhibitor targeting solid tumors that harbor activating alterations to NTRK1, NTRK2, NTRK3, ROS1 or ALK. This clinical trial, called STARTRK-2, the second of the "Studies of Tumor Alterations Responsive to Targeting Receptor Kinases," is a global, multicenter, open label, potentially registration-enabling Phase 2 clinical trial of entrectinib that utilizes a basket design with screening of patient tumor samples for gene rearrangements of the relevant targets. Such a basket design takes full advantage of entrectinib’s demonstrated preliminary clinical activity across a range of different tumor types that harbor a rearrangement of one of the genes encoding any one of entrectinib’s five protein targets.

Also in September 2015, the company announced updated interim results of its Phase 1 clinical trials of entrectinib, which were presented in an oral presentation session at the 2015 European Cancer Congress (ECC 2015) in Vienna, Austria.

The clinical trials included the ALKA-372-001 study and the STARTRK-1 study. Both trials were designed to determine the maximum tolerated dose (MTD) and/or recommended Phase 2 dose (RP2D), as well as preliminary anti-cancer activity, of single agent entrectinib in patients with solid tumors with the relevant molecular alterations: NTRK1 (encoding TrkA), ROS1 or ALK for ALKA-372-001 and NTRK1/2/3 (encoding TrkA/B/C), ROS1 or ALK for STARTRK-1.

As of the August 15, 2015, data cut-off for the presentation, the findings showed:

A total of 92 patients with a range of solid tumors were dosed across both clinical trials, with nine patients treated at or above the RP2D beyond six months and one patient beyond one year;

Entrectinib was well-tolerated;

The fixed daily dose RP2D was determined to be 600 mg, taken orally once per day (QD);

18 patients across both clinical trials met the company’s Phase 2 eligibility criteria, which include:
Presence of an NTRK1/2/3, ROS1 or ALK gene rearrangement, as opposed to other types of molecular alterations (e.g., SNPs, amplifications, deletions);

ALK-inhibitor and/or ROS1-inhibitor naïve; and

Treatment at or above the RP2D;

The response rate in the 18 patients who met these criteria across both studies was 72% (13 responses out of 18 treated patients, as assessed by the clinical sites). Nine of these responders remained on study treatment with durable responses of up to 21 treatment cycles. An additional 3 patients remained on study with stable disease. The responses included:

3 responses out of 4 patients with an NTRK1, NTRK2 or NTRK3 gene rearrangement, including patients with non-small cell lung cancer (NSCLC), colorectal cancer and salivary gland cancer;

6 responses, including one complete response, out of 8 patients with a ROS1 gene rearrangement, all of which were in NSCLC; and

4 responses out of 6 patients with an ALK gene rearrangement, including two NSCLC patients and two patients with other solid tumors.

Entrectinib had demonstrated objective tumor response in the central nervous system (CNS), a frequent site of metastases and progression of advanced solid tumors.

In July 2015, the company announced a clinical collaboration with the University of California, San Francisco (UCSF), which is conducting a multicenter, open label umbrella trial to obtain proof of concept data in patients with metastatic melanoma that is positive for molecular alterations in specific tyrosine kinase receptors. UCSF will exclusively use entrectinib for patients enrolled in the clinical trial having activating molecular alterations to NTRK1/2/3 (encoding TrkA/B/C) or ROS1.

Taladegib License Transaction with Eli Lilly and Company

In November 2015, Ignyta announced it had exclusively licensed worldwide rights relating to Eli Lilly’s taladegib oncology development program in exchange for an upfront payment of $2.0 million in cash and the issuance to Lilly of approximately 1.2 million shares of Ignyta’s common stock. Taladegib is a potent, orally bioavailable small molecule hedgehog/smoothened antagonist that has achieved compelling clinical proof of concept and an RP2D in a Phase 1 dose escalation clinical trial.

Teva Asset Acquisition

In March 2015, Ignyta acquired worldwide rights and assets relating to four targeted oncology development programs from Teva in exchange for 1.5 million shares of Ignyta’s common stock. The programs currently under active development include:

RXDX-105, the company’s orally available, small molecule multikinase inhibitor with potent activity against such targets as RET and
BRAF, that is currently in a Phase 1/1b dose escalation clinical trial; and

RXDX-106, the company’s small molecule, pseudo-irreversible inhibitor of Tyro-3, Axl and Mer (TAM) and cMET that is in late preclinical development.

Presentation of RXDX-105 Clinical Data

In November 2015, Ignyta announced interim results from the ongoing Phase 1/1b clinical trial of RXDX-105, which were presented at the 27th EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium on Molecular Targets and Cancer Therapeutics in Boston, Massachusetts.

The dose escalation clinical trial was designed to determine the MTD and/or RP2D, as well as preliminary anti-cancer activity, of single agent RXDX-105 in patients with advanced or metastatic solid tumors that were not selected based on any molecular alteration.

As of the October 26, 2015, data cut-off for the presentation, the findings showed:

A total of 41 patients with a range of solid tumors were dosed in the clinical trial;

RXDX-105 was well-tolerated;

The MTD and RP2D had not yet been determined;

Pharmacokinetic measurements showed increased exposure with increasing dose, with a half-life compatible with once-daily dosing. Dosing in the fed state appears to further increase exposure;

Exposure was reaching levels expected to be efficacious based on tumor growth inhibition in animal models of RET- and BRAF-driven tumors; and

Tumor regression was observed in six patients treated with 275 mg, including one confirmed partial response (40% reduction) in a patient with NSCLC with a KRAS G12C mutation. Two additional patients with thyroid cancer and squamous cell lung cancer exhibited reductions of 20% and 27%, respectively. In patients with tumor regression, there appears to be an exposure/response correlation.

In March 2016, Ignyta announced the selection of the RP2D and initiation of the Phase 1b portion of the Phase 1/1b clinical trial of RXDX-105. The Phase 1b portion of the study utilizes a basket design focusing on patients with solid tumors that contain molecular alterations of RET or BRAF.

Dx Laboratory

In December 2015, the company announced that its San Diego diagnostic laboratory had been accredited by the College of American Pathologists (CAP) based on results of an on-site inspection. This accreditation is awarded to facilities that meet the highest standards of quality in clinical laboratory services. The U.S. Centers for Medicare & Medicaid Services (CMS) has approved CAP as an accreditation organization for clinical laboratories under CLIA (Clinical Laboratory Improvement Amendments). Ignyta had previously announced CLIA registration of its diagnostic laboratory, and CAP accreditation results in full CLIA certification for Ignyta’s diagnostic laboratory.

In June 2015, the company announced the release for clinical use of its first clinical trial assay, called Trailblaze PharosTM, to support patient identification and enrollment into its STARTRK clinical development program for entrectinib. The proprietary assay was co-developed with ArcherDx and validated within Ignyta’s San Diego diagnostic laboratory. This lab is utilizing this assay in acting as the central testing lab for patient screening for the STARTRK-2 Phase 2 clinical study of entrectinib.

Financing Transactions

Concurrently with the Lilly license transaction in November 2015, Ignyta entered into a stock purchase agreement with Lilly under which Lilly purchased 1.5 million shares of Ignyta common stock at a price of $20 per share in a private placement, which resulted in aggregate gross proceeds of $30 million.

In September 2015, Ignyta borrowed an additional $10 million under its term loan facility from Silicon Valley Bank.

In June 2015, the company issued an aggregate of approximately 4.3 million shares of its common stock in an underwritten public offering at a purchase price of $17.50 per share, which resulted in aggregate gross proceeds of approximately $75 million.

Concurrently with the Teva asset acquisition transaction, Ignyta sold to Teva and selected healthcare investors a total of approximately 4.2 million shares of its common stock at a price of $10 per share in a registered direct offering, which resulted in gross aggregate proceeds of approximately $42 million.

Financial Results

For the 2015 fiscal year, net loss was $92.5 million, or $3.44 per share, compared with $40.0 million, or $2.18 per share, for the 2014 fiscal year.

Ignyta had no material revenues during 2015 or 2014.

Research and development expenses for 2015 were $73.5 million, compared with $30.5 million for 2014. The majority of the increase was due to non-cash costs incurred in connection with the acquisition of rights to development programs from Teva and Lilly during 2015. The remaining increase was primarily attributable to an increase in activities relating to development of entrectinib and the company’s other product candidates, increased personnel expenses related to hiring and engaging additional employees and consultants, and facilities related expenses as a result of the expansion of the company’s leased facilities space.

General and administrative expenses were $17.1 million for 2015, compared with $9.5 million for 2014. The increase was primarily attributable to increases in personnel costs and additional investor relations, audit, legal and intellectual property costs.

At December 31, 2015, the company had cash, cash equivalents and available-for-sale securities totaling $172.1 million and current and long-term debt of $31.0 million. At December 31, 2014, the company had cash and cash equivalents totaling $76.6 million and current and long-term debt of $21.0 million.

On March 14, 2016 Cellular Biomedicine Group Inc. (NASDAQ: CBMG) ("CBMG" or the "Company"), a clinical-stage biomedicine firm engaged in the development of effective treatments for degenerative and cancerous diseases, reported business highlights and financial results for the full year ended December 31, 2015 (Press release, Cellular Biomedicine Group, MAR 14, 2016, View Source [SID:1234509527]).

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"2015 proved to be a transformative year for Cellular Biomedicine Group with the Company’s entrance into the dynamic field of immuno-oncology. We now operate under dual technology platforms: Immuno-oncology (I/O) and Stem Cells. We expect to advance several of our CAR-T candidates including CBM.CD19 and CBM.CD20 into multiple indications of hematological cancer. Eventually, our effort to sponsor multi-center clinical trials in the near future should lead to servicing the large cancer market in China. We also reported encouraging Phase IIb data from our most advanced stem cell program for Knee Osteoarthritis (KOA), and we will explore the possibility of entrance into the U.S. market," commented Tony (Bizou) Liu, CBMG’s Chief Executive Officer. "The expansion of our GMP facilities into Beijing allows us to prepare for anticipated manufacturing demands from our immuno-oncology and stem cell platforms for clinical trials and future commercialization opportunities. We strengthened our operating and management capabilities with the addition of key talents, which will better position the Company to monetize our growing cell therapy pipeline. We look forward to an exciting 2016 as we leverage these strengths to execute on our clinical milestones, build an innovative pipeline and move our clinical assets into later stage clinical development."

2015 and Recent Clinical Developments

Immuno-Oncology Platform

Announced positive clinical data from Phase I of its CAR-T immuno-oncology clinical development programs of:
CBM-CD19.1 for Acute Lymphoblastic Leukemia (B-cell ALL)
CBM-CD20.1 for Advanced Diffuse Large B Cell Lymphoma (DLBCL)
CBM-CD30.1 for Stage III and IV Hodgkin’s lymphoma
CBM-EGFR.1 for the treatment of patients with EGFR expressing advanced relapsed/refractory solid tumors.
In all trials the assets were shown to be safe, feasible and efficacious.
The participants enrolled in the studies were advanced, relapsed, and/or refractory to other standard-of-care therapies. This patient population has substantial unmet medical needs.
Stem Cell Therapies Platform

Announced encouraging 48-week clinical data from the Phase IIb trial of its 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) AlloJoinTMtherapy for KOA patients in China;

Recruited patients for a clinical study on ReJoin therapy for Cartilage Damage (CD) resulting from sports injury, which also serves as a supporting study of ReJoinfor KOA with arthroscopic evidences. We plan to release results from this study in 1H 2017.

2015 and Recent Corporate Highlights

Completed two acquisitions, which substantially increased CBMG’s immuno-oncology platform, including:
PLA General Hospital’s ("PLAGH", Beijing, also known as "301 Hospital") Chimeric Antigen Receptor T cell (CAR-T) therapies, redirected T cells against CD19, CD20, CD30 and Human Epidermal Growth Factor Receptor (EGFR or HER1), their patents (all pending), and Phase I/II clinical data of the aforementioned therapies and manufacturing knowledge;

Blackbird Bio Finance and University of South Florida’s ("Licensor") next generation GVAX vaccine’s ("CD40LGVAX") related technologies, technical knowledge and FDA IND approved clinical trial protocol
Expanded the Company’s cell manufacturing capabilities with the opening of the Company’s third GMP facility, a 15,000 square feet site in Beijing, China, approximately half of which has been designed as a GMP equipped facility to support clinical batch production and commercial scale manufacturing;

Commenced revenue generation through the Company’s T Cells Receptor ("TCR") clonality analysis and CentrixTTM adoptive cell transfer technology services provided to 9 cooperative hospitals located in Beijing, Shandong and Anhui provinces in China;
Strengthened the leadership team with the appointments of Richard L. Wang, Ph.D., MBA, PMP, formerly with GSK, as Chief Operating Officer and Yihong Yao, Ph.D., B.S., formerly with Astrazeneca/Medimmune as Chief Scientific Officer;

Formed a Scientific Advisory Board (SAB) with the appointment of Alan List, M.D. as Chair of the SAB, the appointment of Scott J. Antonia, M.D., Ph.D. to advise the company on immuno-oncology and Guoping Fan, Ph.D. to advise the Company on stem cell technology and its applications;

Continued to demonstrate good corporate governance by meeting the required higher listing standards to successfully upgrade the listing of the Company’s securities from the NASDAQ Capital Market to the NASDAQ Global Market and being selected into the broad-market Russell 3000 Index;

Advanced the Company’s cash position:
Total private placement transactions of approximately $19.6 Million in 2015
Announced agreement of 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 19.4% stake investment in Q1 2016 with an initial closing of $5 million in February 2016, and the remaining $38.13 million by April 15, 2016.

Full Year 2015 Financial Results

Cash Position: The Company had working capital of $13.7 million as of December 31, 2015 compared to $12.0 million as of December 31, 2014. Cash position increased to $14.9 million at December 31, 2015 compared to $14.8 million at December 31, 2014, due to an increase in cash generated from financing activities as a result of a private placement financing in 2015 for aggregate net proceeds of approximately $19.0 million, partially offset by an increase in cash used in operating and investing activities.

Net Cash Used in Operating Activities: Full-year 2015 net cash used in operating activities was $11.8 million compared to $9.7 million in 2014. The change in operating assets and liabilities was primarily due to an increase in accounts receivables, long-term prepaid expenses combined with decreases in tax payables and non-current liabilities, partially offset by an increase in accrued expenses.

Revenue: Full-year 2015 revenue was $2.5 million compared to $0.6 million in 2014. All revenue for the year ended December 31, 2015 was derived from TCR technology services.

G&A Expenses: Full-year 2015 general and administrative expenses were $13.1 million compared $7.9 million in 2014. Increased expenses in 2015 were associated with increased corporate activities related to the management and the development of the Company’s biomedicine business, which were primarily attributed to:

An increase in stock-based compensation expense of $3.7 million, which primarily resulted from the new grants and higher fair value of unvested options in 2015 after the Company listed on Nasdaq in June 2014 compared with those unvested options as of December 31, 2014;
An increase in payroll of $0.3 million in line with the headcount increase in management in 2015;
An increase in depreciation and amortization of $0.2 million, which was mainly attributed to the technical knowledge and patents obtained from the acquisition of AG in the third quarter 2014;
R&D Expenses: Full-year 2015 research and development expenses were $7.6 million compared to $3.1 million in 2014, the increase mainly attributable to the increase of our immunotherapy research and development team, which resulted in an increase in payroll expenses of $1.2 million and an increase in stock-based compensation expenses of $1.8 million.

Net Loss: Full-year 2015 net loss allocable to common stock holders was $19.4 million compared to $15.5 million in 2014. Changes in net loss were primarily attributable to changes in operations of our biomedicine segment.

Primary 2016 Operating Objectives
The Company’s key 2016 operational objectives are to seek early possibilities of conducting multi-center Phase IIb trials with its CAR-T constructs after confirming their safety and tolerability profile, to evaluate feasibility of sponsoring a registration trial-like clinical study to support a New Drug Application (NDA) for an allogeneic haMPC Knee Osteoarthritis therapy ("Allo KOA") study in the United States, and continue to build a pipeline of advanced technologies to bolster the Company’s CAR-T China market position.

On March 14, 2016 Bellicum Pharmaceuticals, Inc. (Nasdaq:BLCM), a clinical stage biopharmaceutical company focused on discovering and developing novel cellular immunotherapies for cancers and orphan inherited blood disorders, reported financial results for the fourth quarter and full year ended December 31, 2015, and provided an update on the Company’s recent progress (Press release, Bellicum Pharmaceuticals, MAR 14, 2016, View Source;p=RssLanding&cat=news&id=2148302 [SID:1234509523]).

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"In 2015, Bellicum made significant progress across all of our T-cell immunotherapy programs, reporting positive interim results with our lead product candidate BPX-501, and advancing our CAR T and TCR programs," said Tom Farrell, Bellicum’s President and Chief Executive Officer. "Data presented at ASH (Free ASH Whitepaper) from the BP-004 clinical trial with BPX-501 demonstrated disease-free outcomes in pediatric patients with genetic blood diseases. We believe that BPX-501, which was recently granted orphan drug designation by the FDA, could represent a vital treatment option for the many patients for whom a transplant is recognized as the preferred treatment, but who are not treated because they lack a perfect match donor."

Continued Mr. Farrell, "Building on this momentum, we look forward to achieving important milestones in 2016, with data updates from the BPX-501 program expected to be presented at several medical meetings. We also expect to launch Phase 1 clinical trials of three novel product candidates: BPX-701 and BPX-601 in mid-2016, and BPX-401 in the second half of 2016. The BPX-701 and BPX-601 programs were reviewed at the March 10th meeting of the National Institutes of Health (NIH) Recombinant DNA Advisory Committee (RAC) and we were pleased with the engaging discussion and meeting outcomes."

2015 HIGHLIGHTS AND CURRENT UPDATES

DEVELOPMENT PROGRAMS

BPX-501

Adjunct T-cell therapy administered after allogeneic hematopoietic stem cell transplantation (HSCT), using genetically modified donor T cells incorporating our CaspaCIDe safety switch, is being evaluated in malignant and nonmalignant blood diseases.

Enrollment in Phase 1/2 BP-004 clinical trial continues at strong pace, with 63 pediatric patients enrolled in the E.U. and 12 patients enrolled in the U.S. to date.
Granted orphan drug designation by the FDA for the combination of BPX-501 genetically modified T cells and activator agent rimiducid as "replacement T-cell therapy for the treatment of immunodeficiency and Graft versus Host Disease after allogeneic hematopoietic stem cell transplant."
Reported interim data from ongoing BP-004 trial, demonstrating disease-free outcomes in pediatric patients with genetic blood diseases who had undergone HSCT followed by BPX-501 donor T-cell replacement. Presented at the 57th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in December 2015, the data showed that treated patients achieved immune recovery significantly faster than historical control subjects not given BPX-501, as well as a significant reduction in time to hospital discharge (21 days sooner vs. historical controls) and reduced re-hospitalizations. In addition, none of the patients had developed chronic GvHD and no patient died of transplantation-related complications. Of the 39 patients treated (as of Nov. 30, 2015), 21 had genetic blood diseases, including Fanconi anemia, beta thalassemia, SCID and Wiskott-Aldrich Syndrome, and 18 had blood cancers.
DOTTI study clinical data published in BLOOD highlighted safety and effectiveness of CaspaCIDe-modified T-cell add-back. Results of a 12-patient investigator-sponsored trial conducted by Baylor College of Medicine demonstrated that the add-back led to improved immune reconstitution and infection control. The data also showed that GvHD can be rapidly controlled and resolved by administration of rimiducid, and that the productive anti-viral cells remain, repopulate and maintain immunity.
U.S. patent issued to Baylor College of Medicine for technology exclusively licensed to Bellicum. The patent, issued for methods of inducing selective apoptosis of cells, extends Bellicum’s proprietary rights to the use of its lead product candidate until at least 2031.
BPX-701

High affinity T cell receptor (TCR) product candidate, incorporating our CaspaCIDe safety switch, is designed to target malignant cells expressing the preferentially-expressed antigen in melanoma (PRAME).

Bellicum continues to advance its next-generation, proprietary TCR product candidate targeting PRAME. The Company licensed the PRAME-specific TCR technology from Leiden University Medical Center in April 2015 and is preparing for the start of clinical trials for the initial planned indications of Refractory or Relapsed Acute Myeloid Leukemia (AML) and Myelodysplastic Syndromes (MDS), with an additional clinical trial planned for metastatic uveal melanoma. Each of these are orphan indications in which PRAME is highly expressed and for which current treatment options are limited.
BPX-601

GoCAR-T product candidate, containing proprietary iMC (inducible MyD88/CD40) activation switch, is designed to treat solid tumors expressing prostate stem cell antigen (PSCA).

Continued to advance the Company’s first GoCAR-T product candidate toward the clinic. The planned indication for the initial Phase 1 study is non-resectable pancreatic cancer. Preclinical data reported at ASH (Free ASH Whitepaper) 2015 showed robust anti-tumor activity, and enhanced T-cell proliferation and persistence compared to traditional CAR T constructs.
Obtained an exclusive global license from Agensys for adoptive cell therapies targeting tumors expressing PSCA.
BPX-401

CIDeCAR product candidate, incorporating novel, proprietary MC costimulatory domain and CaspaCIDe safety switch, is designed to target blood cancers expressing CD19.

Presented preclinical data at ASH (Free ASH Whitepaper) 2015 highlighting the potent anti-tumor effects of BPX-401. The preclinical in vivo results showed that tumors can be eliminated quickly and safely with CIDeCAR cells. Notably, BPX-401 elicited dose-dependent elevation of cytokines, analogous to cytokine release syndrome, but cytokine levels were rapidly normalized upon administration of rimiducid, without loss of tumor control.
CORPORATE UPDATES

Committed to build-out of in-house U.S. manufacturing capabilities. The Company leased an additional 27,000 square feet at its current location and completed the design phase for the build-out of manufacturing space. The facility is designed to support the efficient manufacturing of our novel cellular immunotherapies for clinical trials and early commercial requirements.
Recently closed on a debt financing agreement with Hercules Capital to support the build-out of the Company’s U.S. manufacturing facilities. Under the loan terms, Bellicum can borrow up to $30 million, of which the final $10 million tranche is contingent upon achievement of specified milestones and approval by Hercules’ investment committee.
ANTICIPATED 2016 MILESTONES

BPX-501

Expect to provide an update on the nonmalignant patient cohort from the BP-004 clinical trial, as well as initial data for patients with blood cancers treated at the lead European clinical trial site, during the 42nd Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT) which takes place April 3 – 6, 2016.
Expect to meet with regulators in Europe and the U.S. in the second quarter of 2016, with the goal of defining the path to regulatory filing and approval initially for nonmalignant pediatric genetic diseases.
Anticipate presenting updated data from the BPX-501 program at ASH (Free ASH Whitepaper) in December 2016.
BPX-701

Expect to begin enrolling patients in a Phase 1 clinical trial for refractory or relapsed acute AML and MDS in mid-2016.
BPX-601

Expect to begin enrolling patients in a Phase 1 clinical trial in mid-2016 for non-resectable pancreatic cancer.
BPX-401

Expect to move a CIDeCAR product candidate directed to the CD19 antigen into the clinic in the second half of 2016.
Corporate

Expect to complete build-out of the U.S. cGMP viral vector and cellular therapy manufacturing facility by the end of 2016.
Expect to establish a European presence and initiate activities in anticipation of the potential commercialization of BPX-501 in future years.
Fourth Quarter and Full Year 2015 Financial Results

Cash Position and Guidance: Bellicum ended the year on December 31, 2015 with cash and investments totaling $150.4 million, compared to $191.6 million at December 31, 2014. Based on current operating plans and capital available under its loan facility, Bellicum expects to end 2016 with approximately $80 to $90 million in cash, cash equivalents and investments, and anticipates that current cash resources will be sufficient to meet operating requirements through 2017. The Company also expects that in 2016 it will invest approximately $25 to $30 million for capital projects to enable in-house U.S. manufacturing and to support the Company’s growth.

Grant Revenues were $34,000 and $282,000 for the fourth quarter and year ended December 31, 2015, respectively, and $14,000 and $1,780,000 during the comparable periods in 2014. The decrease in full year 2015 grant revenues was primarily due to the June 2014 expiration of the Company’s grant award from the Cancer Prevention and Research Institute of Texas.

R&D Expenses: Research and development expenses were $10.2 million and $33.6 million for the fourth quarter and year ended December 31, 2015, respectively, compared to $4.2 million and $12.1 million during the comparable periods in 2014. The higher expenses in the 2015 periods were primarily due to an increase in BPX-501 clinical and manufacturing costs as a result of increased patient enrollment in clinical trials, an increase in costs related to preclinical product candidates BPX-701, BPX-601 and BPX-401’s IND-enabling activities, and an increase in general research and development costs including personnel costs, and allocated overhead.

License fees were $3.0 million and $3.2 million for the fourth quarter and year ended December 31, 2015, respectively, compared to no license fees in 2014. The increase in fees was primarily due to a new license agreement with Agensys, an affiliate of Astellas, as consideration for rights granted to Bellicum under the agreement related to its BPX-601 product candidate, whereby Agensys was paid a non-refundable upfront fee of $3.0 million.

G&A Expenses: General and administrative expenses were $3.8 million and $12.7 million for the fourth quarter and year ended December 31, 2015, respectively, compared to $2.0 million and $4.3 million during the comparable periods in 2014. The increased G&A expenses in 2015 were primarily due to overall growth and public company related costs, including an increase in personnel, legal and accounting expenses and costs related to facilities, insurance and travel.

Net Loss: Bellicum reported a net loss of $16.8 million for the fourth quarter of 2015 and $48.5 million for the year ended December 31, 2015, compared to a net loss of $74.3 million and $84.0 million for the comparable periods in 2014. The net loss amounts for the 2014 periods included a charge of $43.2 million incurred in the fourth quarter of 2014 in conjunction with the ARIAD license restructure transaction and a non-cash accounting charge of $24.4 million recorded for the change in fair value of warrants that were exercised in conjunction with Bellicum’s December 2014 initial public offering, of which $23.2 million was a fourth quarter expense. The results also included non-cash, share-based compensation charges of $2.5 million and $8.4 million for the fourth quarter and year ended December 31, 2015, respectively, and $0.7 million and $0.9 million for the comparable periods in 2014.

Shares Outstanding: At December 31, 2015, Bellicum had 26,931,881 shares of common stock outstanding.

On March 14, 2016 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, reported financial results for the fourth quarter and full year 2015 and commented on recent accomplishments and clinical development plans for its pipeline of several SINE-based therapeutics including selinexor, its lead product candidate (Press release, Karyopharm, MAR 14, 2016, View Source [SID:1234509521]).

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"During 2015, we continued to expand our leadership position in the development of oral SINE-based oncology and non-oncology therapies with clinical advancement of selinexor across a number of hematologic and solid tumor indications and the presentation of encouraging data on our earlier-stage pipeline programs," said Michael G. Kauffman, MD, PhD, Chief Executive Officer of Karyopharm. "Our broad and aggressive oncology clinical development program continues in 2016 with both company- and investigator-sponsored trials underway or planned to evaluate selinexor both as a single-agent and in combination with other existing and emerging oncology therapies. In addition, we made important progress with our non-oncology focused SINE compounds in a number of disease areas in which XPO1 is critically involved. We look forward to several potentially value-creating data readouts over the next 18 months."

Clinical Development Plans:
Selinexor in hematologic malignancies. Karyopharm is actively enrolling patients in several later phase clinical studies evaluating single-agent selinexor in hematologic malignancies, including patients with relapsed/refractory multiple myeloma (MM) (STORM study), older patients with relapsed/refractory acute myeloid leukemia (AML) (SOPRA study) and patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL) (SADAL study). Karyopharm expects data from the first 80 patients in the STORM study to be available in mid-2016, at which point, it will evaluate whether to expand the study. Karyopharm also expects to provide an interim analysis update from the SOPRA study in late-2016 with top-line data expected in mid-2017. Based on discussions with the U.S. Food and Drug Administration (FDA), Karyopharm amended the protocol for the ongoing SADAL study to remove dexamethasone from the regimen and to evaluate selinexor as a single-agent in this patient population. The study will continue to compare 60 mg and 100mg twice weekly doses of selinexor with 100 patients per arm and at least 50% of patients with GCB-type DLBCL. Based on these amendments, the company expects to report top-line data from this study in early 2017.

Single-agent selinexor in solid tumors. Karyopharm is currently conducting company-sponsored trials of single-agent selinexor in three solid tumor indications including advanced unresectable dedifferentiated liposarcoma (SEAL study), heavily pretreated patients with gynecologic malignancies (SIGN study) and recurrent glioblastoma multiforme (KING study). Karyopharm is planning to present updated data from the SIGN and KING studies at an appropriate medical meeting in mid-2016. The Phase 2/3 SEAL study, evaluating single-agent oral selinexor versus placebo, was initiated earlier this year based on promising clinical data presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2015 Annual Meeting in which durable stable disease and improvement in progression free survival compared to previous chemotherapies were observed. Top-line data from the Phase 2 portion of this study are expected in mid-2017.

Selinexor combinations. A number of investigator-sponsored and company-sponsored trials evaluating selinexor in combination with either chemotherapy or targeted agents in hematologic and solid tumor indications are currently ongoing or planned. In mid-2016, Karyopharm plans to initiate a Phase 2/3 study (SCORE study) evaluating the combination of selinexor, carfilzomib and dexamethasone in patients with refractory MM who were previously treated with a proteasome inhibitor and an immunomodulatory agent. In addition, Karyopharm expects to report top line data from the Phase 1 portion of a Phase 1b/2 study (STOMP study) evaluating selinexor in combination with commonly used treatments, including bortezomib, pomalidomide, or lenalidomide, for relapsed/refractory MM in late-2016.

KPT-8602, second generation SINE compound. In January 2016, Karyopharm initiated a Phase 1/2 study of oral KPT-8602, a novel, second generation, SINE compound, in patients with relapsed/refractory MM. Data from preclinical studies presented at the 2015 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting demonstrated single-agent anti-MM activity of KPT-8602 along with the potential for higher and/or more frequent dosing compared with selinexor. Top-line safety and tolerability data from this Phase 1/2 study are expected in late 2016.

KPT-9274, oral dual inhibitor of PAK4 and NAMPT. In mid-2016, Karyopharm plans to initiate clinical development in patients with heavily pretreated solid tumors or lymphoma for its oral dual inhibitor of PAK4 and NAMPT, KPT-9274.

Verdinexor (KPT-335). In May 2015, Karyopharm began clinical testing of verdinexor in a randomized, double-blind, placebo-controlled, dose-escalating Phase 1 clinical trial in healthy human volunteers in Australia. Verdinexor was found to be generally safe and well tolerated. Karyopharm is preparing to advance verdinexor for certain viral indications with an initial focus on influenza. Preclinical data provide strong support for other potential indications for verdinexor, including human immunodeficiency virus (HIV).
KPT-350. KPT-350 is an investigational new drug application-ready oral compound with a preclinical data package supporting potential efficacy in a number of neurological, autoimmune and inflammatory conditions. We plan to partner with a collaborator to undertake the clinical development and potential commercialization of KPT-350 in one or more mutually agreed indications.

Scientific Presentations and Publications:
Positive clinical data demonstrating the activity and tolerability of selinexor across multiple hematologic malignancies including MM and AML were presented at the 2015 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting held in December 2015 in Orlando, Florida.

Updated data from an ongoing investigator-sponsored trial of selinexor in combination with carfilzomib and dexamethasone in refractory MM were presented by Andrzej Jakubowiak, MD, PhD, from The University of Chicago. The data demonstrated encouraging efficacy of selinexor in combination with carfilzomib and dexamethasone in heavily pretreated patients with highly refractory MM, including patients whose disease is refractory to previous carfilzomib-based combinations, suggesting the potential of this combination to overcome carfilzomib resistance. Based on these data, Karyopharm expects to initiate a Phase 2/3 clinical study (SCORE study) in mid-2016.

Karyopharm researchers and collaborators also presented preclinical data demonstrating the potential of selinexor to synergize with proteasome inhibitors to overcome drug resistance in MM.

Karyopharm presented data demonstrating the activity and tolerability of selinexor in combination with standard of care agents in the AML setting, including data from a Phase 2 trial investigating the efficacy and tolerability of selinexor in combination with arabinoside cytosine (Ara-C) and idarubicin in "fit" patients with relapsed and/or refractory AML. Karyopharm also presented data from a Phase 1 study determining the safety and efficacy of selinexor in combination with fludarabine and cytarabine in pediatric patients with relapsed and/or refractory acute leukemia.

Karyopharm researchers presented data establishing 60mg as the most appropriate selinexor dose for both efficacy and tolerability across many hematologic and solid cancers. These data support the selinexor first-in-human Phase 1 clinical trial solid tumor data published in Journal of Clinical Oncology in March 2016, which recommended a dose of 60mg given twice a week as the Phase 2 dose. This recommendation was based on superior patient tolerability, longer duration of therapy, and no demonstrable improvement in radiologic response or disease stabilization compared with higher doses.

Fourth Quarter and Year Ended December 31, 2015 Financial Results

Cash, cash equivalents and investments as of December 31, 2015, including restricted cash, totaled $210.0 million, compared to $214.8 million as of December 31, 2014.

For the year ended December 31, 2015, research and development expense was $97.7 million compared to $60.1 million for the year ended December 31, 2014. For the year ended December 31, 2015, general and administrative expense was $21.6 million compared to $15.9 million for the year ended December 31, 2014. The increase in research and development expense resulted primarily from the increase in expenses related to the continued clinical development of selinexor. The increase in general and administrative expense resulted primarily from an increase in personnel costs, including stock-based compensation expense.

Karyopharm reported a net loss of $118.2 million, or $3.32 per share, for the year ended December 31, 2015, compared to a net loss of $75.8 million, or $2.43 per share, for the year ended December 31, 2014. Net loss includes stock-based compensation expense of $17.1 million and $14.2 million for the years ended December 31, 2015 and 2014, respectively.

For the quarter ended December 31, 2015, research and development expense was $24.1 million compared to $20.0 million for the quarter ended December 31, 2014. For the quarter ended December 31, 2015, general and administrative expense was $5.3 million compared to $5.9 million for the quarter ended December 31, 2014. The increase in research and development expenses resulted primarily from the increase in expenses related to the continued clinical development of selinexor.

Karyopharm reported a net loss of $29.0 million, or $0.81 per share, for the quarter ended December 31, 2015, compared to a net loss of $25.9 million, or $0.79 per share, for the quarter ended December 31, 2014. Net loss includes stock-based compensation expense of $5.4 million and $4.6 million for the quarters ended December 31, 2015 and December 31, 2014, respectively.

Financial Outlook
Based on current operating plans, Karyopharm expects that its existing cash and cash equivalents will fund its research and development programs and operations into the middle of 2018, including advancing the four later-stage clinical studies to their next data inflection points. Karyopharm expects to end 2016 with at least $120 million in cash, cash equivalents and investments.

On March 14, 2016 Stemline Therapeutics, Inc. (Nasdaq:STML) reported financial results for the quarter ended December 31, 2015 (Press release, Stemline Therapeutics, MAR 14, 2016, View Source [SID:1234509512]).

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Ivan Bergstein, M.D., Stemline’s Chief Executive Officer, commented, "During the fourth quarter, we made significant progress across all three of our development programs. Key achievements included 1) generating and presenting data from our ongoing Phase 2 trial of SL-401 in blastic plasmacytoid dendritic cell neoplasm (BPDCN) which we have designed to support potential registration, 2) treating our first patient in the second stage of our Phase 2 program of SL-701 in advanced brain cancer, and 3) the opening of our IND for SL-801 on schedule. At the American Society of Hematology (ASH) (Free ASH Whitepaper) meeting in December, we presented very strong data from our ongoing SL-401 trial in BPDCN, demonstrating a high overall response rate in BPDCN, with multiple complete responses. We are also very pleased with the pace of enrollment in BPDCN, and note that we are tracking in-line with our expectations. We look forward to sharing key clinical and regulatory updates on SL-401 throughout this year."

Dr. Bergstein continued, "We continue to evaluate SL-401 in several additional malignancies that overexpress IL-3R. Specifically, we have single agent trials enrolling AML patients with minimal residual disease and patients with advanced high-risk myeloproliferative neoplasms. We also expect our first clinical studies that combine SL-401 with other therapies will begin this year."

Dr. Bergstein concluded, "Our other pipeline candidates, SL-701 and SL-801, continue to advance. Our SL-701 trial is currently enrolling patients and we expect updates from this program in the second half of the year. The SL-801 IND is open, and we are on track to treat our first patient this quarter. With our strong cash position, we believe we have sufficient financial resources to achieve significant clinical and regulatory milestones this year and beyond."

Fourth Quarter 2015 Financial Results Review

Stemline ended the fourth quarter of 2015 with $97.5 million in cash, cash equivalents and investments, as compared to $104.0 million as of September 30, 2015, which reflects a cash burn of $6.5 million for the quarter. The company ended the fourth quarter of 2015 with 18.2 million shares outstanding.

For the fourth quarter of 2015, Stemline had a net loss of $10.2 million, or $0.58 per share, compared with a net loss of $6.9 million, or $0.53 per share, for the same period in 2014. The net loss for full year 2015 was $37.2 million, or $2.15 per share, as compared with a net loss of $28.8 million, or $2.23 per share, in the prior year.

Research and development expenses were $7.9 million for the fourth quarter of 2015, which reflects an increase of $2.8 million, or 56%, compared with $5.1 million for the fourth quarter of 2014. The increase in expenses during the current quarter was primarily attributable to the ramp up in our clinical trial activities for SL-401 and SL-801.

General and administrative expenses were $2.6 million for the fourth quarter of 2015, which reflects an increase of $0.6 million, or 30%, compared with $2.0 million for the fourth quarter of 2014. The increase in expenses during the current quarter was primarily attributable to an increase in compensation expense relating to administrative employees.